POPULARITY
3 episodes with casgevy
4 episodes with casgevy
3 episodes with casgevy
2 episodes with casgevy
3 episodes with casgevy
2 episodes with casgevy
2 episodes with casgevy
2 episodes with casgevy
From baby blood to breakthrough cures — we explore how unlocking fetal hemoglobin through gene editing (exa-cel/Casgevy) is transforming treatment for sickle cell disease and β-thalassemia.
Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Novo Nordisk predicts a brighter future for Wegovy with the end of the semaglutide shortage, but analysts remain skeptical as Eli Lilly's Zepbound gains ground in sales. The new HHS vaccine requirement has raised questions about its true intentions, with leading vaccine physician Paul Offit criticizing the lack of clarity. Meanwhile, pharmaceutical imports from Ireland are on the rise, biotech stocks fell after Vinay Prasad was named to succeed Marks at CBER, and Lotte Biologics' ADC facility in Syracuse offers end-to-end services for antibody manufacturing. CRISPR's Casgevy is gaining traction with more gene therapy proof of concept expected in 2025, impacting M&A and IPOs in the biotech industry. Summit Pharmaceuticals is nearing the first global phase III data for Keytruda, set to lose exclusivity in 2028 and potentially face competition from biosimilars. A report suggests that low-price drug nations are benefiting from US innovation, undervaluing innovative medicines by 90%. VC financing in biopharma declined by 20% in Q1, but megarounds kept the median deal size high. M&A and IPOs faced challenges due to policy issues, leading to an increase in licensing deals. Novo vows to improve market access for Wegovy, while Trump orders FDA to ease US plant expansion and increase inspections of foreign facilities. BMS pledges a $40 billion investment in the US, Novartis makes M&A moves, and Lilly remains unfazed by CVS's decision to side with Novo in the obesity market battle. Some drugmakers are stockpiling products in the US amid Trump's trade war. NGM terminates half of its staff as its lead asset moves through a registrational study. Job opportunities in clinical quality assurance audit, RBQM central statistical monitoring, and field medical capabilities are available.
In a special co-branded episode between Oncology On theGo hosted by CancerNetwork® and the American Society for Transplantation and Cellular Therapy (ASTCT)'s program ASTCT Talks, Nora M. Gibson, MD, MSCE, and Taha Al-Juhaishi, MD, spoke about real-world applications of betibeglogene autotemcel (beti-cel; Zynteglo) as a treatment for patients with beta (β)-thalassemia.They spoke in the context of a study that Gibson presented at the 2025 Tandem Meetings, which evaluated patients who received commercial beti-cel in a single-center cohort following the agent's FDA approval in August 2022. ¹ ²Nora is a fourth-year fellow in bone marrow transplant andcellular therapy at the Children's Hospital of Philadelphia (CHOP), with a background in clinical research and epidemiology. Al-Juhaishi is the Associate Director of the Hematopoietic Stem Cell Transplantation and Cell TherapyProgram at Oklahoma University Health Stephenson Cancer Center and an assistant professor of Medicine at the University of Oklahoma College of Medicine.Findings from Gibson's study revealed that among 10 patients who underwent stem cell collection at CHOP from 2022 to 2024, beti-cel yielded consistent red blood cell transfusion independence, with investigators notingprolonged platelet engraftment time and high platelet transfusion requirements. Beyond these findings, the conversation focused on how beti-cel compares withother currently available gene therapies for patients with hemoglobin disorders as well as non-curative therapies like allogeneic stem cell transplantation. Gibson and Al-Juhaishi also discussed strategies for mitigating occlusive disease and other potential toxicities associated with beti-cel.“It's a really exciting time to be working in this fieldwhere we finally have really good options for these patients. From our experience and from clinical trials, beti-cel and likely exagamglogene autotemcel [Casgevy]...are very effective, curative therapies for thalassemia in the real-world setting, and we've seen very similar results in sickle celldisease,” said Gibson. “These therapies have been really life-changing for our patients, and they've had a huge reduction in their symptoms and a huge reduction in their burden of health care that's required.”References1. Gibson NM, Friedman DF, Elgarten CW, et al. Post-approval, real-world experience with betibeglogene autotemcel for transfusion-dependent betathalassemia. Transplantation and Cellular Therapy. 2025;31(2):S254. doi:10.1016/j.jtct.2025.01.386.2. FDA approves first cell-based gene therapy totreat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News Release. FDA. August 17, 2022. Accessed April 21, 2025.https://tinyurl.com/3vrkk8kz
In a special co-branded episode between Oncology On the Go hosted by CancerNetwork® and the American Society for Transplantation and Cellular Therapy (ASTCT)'s program ASTCT Talks, Nora M. Gibson, MD, MSCE, and Taha Al-Juhaishi, MD, spoke about real-world applications of betibeglogene autotemcel (beti-cel; Zynteglo) as a treatment for patients with beta (β)-thalassemia. They spoke in the context of a study that Gibson presented at the 2025 Tandem Meetings, which evaluated patients who received commercial beti-cel in a single-center cohort following the agent's FDA approval in August 2022. Nora is a fourth-year fellow in bone marrow transplant and cellular therapy at the Children's Hospital of Philadelphia (CHOP), with a background in clinical research and epidemiology. Al-Juhaishi is the Associate Director of the Hematopoietic Stem Cell Transplantation and Cell Therapy Program at Oklahoma University Health Stephenson Cancer Center and an assistant professor of Medicine at the University of Oklahoma College of Medicine. Findings from Gibson's study revealed that among 10 patients who underwent stem cell collection at CHOP from 2022 to 2024, beti-cel yielded consistent red blood cell transfusion independence, with investigators noting prolonged platelet engraftment time and high platelet transfusion requirements. Beyond these findings, the conversation focused on how beti-cel compares with other currently available gene therapies for patients with hemoglobin disorders as well as non-curative therapies like allogeneic stem cell transplantation. Gibson and Al-Juhaishi also discussed strategies for mitigating occlusive disease and other potential toxicities associated with beti-cel. “It's a really exciting time to be working in this field where we finally have really good options for these patients. From our experience and from clinical trials, beti-cel and likely exagamglogene autotemcel [Casgevy]...are very effective, curative therapies for thalassemia in the real-world setting, and we've seen very similar results in sickle cell disease,” said Gibson. “These therapies have been really life-changing for our patients, and they've had a huge reduction in their symptoms and a huge reduction in their burden of health care that's required.” References 1. Gibson NM, Friedman DF, Elgarten CW, et al. Post-approval, real-world experience with betibeglogene autotemcel for transfusion-dependent beta thalassemia. Transplantation and Cellular Therapy. 2025;31(2):S254. doi:10.1016/j.jtct.2025.01.386. 2. FDA approves first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News Release. FDA. August 17, 2022. Accessed April 21, 2025. https://tinyurl.com/3vrkk8kz
This week, GSK and Gilead and Arcellx presented key data at the American Society of Hematology (ASH) annual meeting as they vie for a competitive advantage in multiple myeloma. Meanwhile, Vertex unveiled positive long-term data for its CRISPR Therapeutics–partnered gene therapy Casgevy in sickle cell disease—results BMO Capital Markets analysts said should help Casgevy keep its edge over bluebird bio's Lyfgenia. On that note, Casgevy and Lyfgenia have a new outcomes-based payment model after the Centers for Medicare and Medicaid Service (CMS) said both companies have agreed to participate in a voluntary program to improve access to the gene therapies. Speaking of access, Eli Lilly and Novo Nordisk both announced significant manufacturing investments aimed at shoring up production of their diabetes and weight loss blockbusters tirzepatide and semaglutide. And in related news, the European Commission gave its blessing to Novo Holdings' controversial acquisition of contract manufacturing firm Catalent. Elsewhere, AbbVie got a much-needed win for Cerevel-acquired Parkinson's disease therapy tavapadon—a month after the deal's cornerstone asset emraclidine failed in schizophrenia—while uniQure announced it has aligned with the FDA on “key elements” of the accelerated approval pathway for its investigational gene therapy for Huntington's, AMT-130.
In a special co-branded episode between Oncology On the Go hosted by CancerNetwork® and the American Society for Transplantation and Cellular Therapy (ASTCT)'s program ASTCT Talks, Alexis K. Kuhn, PharmD, BCOP, spoke with Katie Bruce, PharmD, BCPPS, and Susie Long, PharmD, about the use of approved cell-based gene therapies for patients with sickle cell disease, beta thalassemia, adrenoleukodystrophy (ALD), and metachromatic leukodystrophy (MLD). These panelists shared the pharmacist's perspective on ensuring quality care with these ex vivo gene therapies across all treatment phases, including mobilization, conditioning, and infection prophylaxis. Kuhn is an ambulatory Pediatric Hematology/Oncology/BMT Pharmacist at the Mayo Clinic in Rochester, Minnesota, and an assistant professor of Pharmacy at the Mayo Clinic College of Medicine. Bruce is a pediatric clinical pharmacy specialist at the Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy program of Tristar Centennial Medical Center in Nashville, Tennessee. Long is a pediatric clinical pharmacist in the Blood and Marrow Team at the University of Minnesota Masonic Children's Hospital. Specifically, the panelists spoke about the use of agents like elivaldogene autotemcel (Skysona) and atidarsagene autotemcel (Lenmeldy), which are FDA-approved for ALD and MLD, respectively. They also discussed the use of exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia), which the FDA approved for treating patients 12 years and older with sickle cell disease in December 2023. The conversation broke down each stage of treatment, detailing optimal strategies for the cell manufacturing and storing processes as well as the management of toxicities like cytopenias. They also reviewed key considerations during the post-infusion period that may help maximize the quality of life for patients after they complete their therapy. “It has been so amazing to be able to be a part of gene therapy and gene editing,” Bruce stated regarding the potential long-term impacts of these treatments. “We have patients who are able to hold full-time jobs they never were able to have before. We have patients who are climbing mountains and backpacking through Europe, which would have never been an option before because their sickle cell disease would have prevented them from [doing] that…. It's not an easy process, and it has a lot of steps for the patient to go through, but the reward at the end of it all is worth it.” References 1. bluebird bio receives FDA accelerated approval for SKYSONA® gene therapy for early, active cerebral adrenoleukodystrophy (CALD). News release. bluebird bio, Inc. September 16, 2022. Accessed October 7, 2024.https://tinyurl.com/mp8crxes 2. FDA approves first gene therapy for children with metachomatic leukodystrophy. New release. FDA. March 18, 2024. Accessed October 7, 2024. https://tinyurl.com/mrh659yk 3. FDA approves first gene therapies to treat patients with sickle cell disease. News release. FDA. December 8, 2023. Accessed October 7, 2024. https://tinyurl.com/3zbdnf4c
In a special co-branded episode between Oncology On the Go hosted by CancerNetwork® and the American Society for Transplantation and Cellular Therapy (ASTCT)'s program ASTCT Talks, Alexis K. Kuhn, PharmD, BCOP, spoke with Katie Bruce, PharmD, BCPPS, and Susie Long, PharmD, about the use of approved cell-based gene therapies for patients with sickle cell disease, beta thalassemia, adrenoleukodystrophy (ALD), and metachromatic leukodystrophy (MLD). These panelists shared the pharmacist's perspective on ensuring quality care with these ex vivo gene therapies across all treatment phases, including mobilization, conditioning, and infection prophylaxis. Kuhn is an ambulatory Pediatric Hematology/Oncology/BMT Pharmacist at the Mayo Clinic in Rochester, Minnesota, and an assistant professor of Pharmacy at the Mayo Clinic College of Medicine. Bruce is a pediatric clinical pharmacy specialist at the Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy program of Tristar Centennial Medical Center in Nashville, Tennessee. Long is a pediatric clinical pharmacist in the Blood and Marrow Team at the University of Minnesota Masonic Children's Hospital. Specifically, the panelists spoke about the use of agents like elivaldogene autotemcel (Skysona) and atidarsagene autotemcel (Lenmeldy), which are FDA-approved for ALD and MLD, respectively. They also discussed the use of exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia), which the FDA approved for treating patients 12 years and older with sickle cell disease in December 2023. The conversation broke down each stage of treatment, detailing optimal strategies for the cell manufacturing and storing processes as well as the management of toxicities like cytopenias. They also reviewed key considerations during the post-infusion period that may help maximize the quality of life for patients after they complete their therapy. “It has been so amazing to be able to be a part of gene therapy and gene editing,” Bruce stated regarding the potential long-term impacts of these treatments. “We have patients who are able to hold full-time jobs they never were able to have before. We have patients who are climbing mountains and backpacking through Europe, which would have never been an option before because their sickle cell disease would have prevented them from [doing] that…. It's not an easy process, and it has a lot of steps for the patient to go through, but the reward at the end of it all is worth it.” References bluebird bio receives FDA accelerated approval for SKYSONA® gene therapy for early, active cerebral adrenoleukodystrophy (CALD). News release. bluebird bio, Inc. September 16, 2022. Accessed October 7, 2024. https://tinyurl.com/mp8crxes FDA approves first gene therapy for children with metachomatic leukodystrophy. New release. FDA. March 18, 2024. Accessed October 7, 2024. https://tinyurl.com/mrh659yk FDA approves first gene therapies to treat patients with sickle cell disease. News release. FDA. December 8, 2023. Accessed October 7, 2024. https://tinyurl.com/3zbdnf4c
2023 ended with an exciting biotech breakthrough for sickle cell patients. A gene-editing therapy using the revolutionary CRISPR technology provides new hope and options for the nearly 100,000 Americans with sickle cell disease. In this episode, we talk with the company behind one of the life-changing therapies, CASGEVY, and speak with two advocates for sickle cell patients.
Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Superbabies: Putting The Pieces Together, published by sarahconstantin on July 12, 2024 on LessWrong. This post was inspired by some talks at the recent LessOnline conference including one by LessWrong user "Gene Smith". Let's say you want to have a "designer baby". Genetically extraordinary in some way - super athletic, super beautiful, whatever. 6'5", blue eyes, with a trust fund. Ethics aside[1], what would be necessary to actually do this? Fundamentally, any kind of "superbaby" or "designer baby" project depends on two steps: 1.) figure out what genes you ideally want; 2.) create an embryo with those genes. It's already standard to do a very simple version of this two-step process. In the typical course of in-vitro fertilization (IVF), embryos are usually screened for chromosomal abnormalities that would cause disabilities like Down Syndrome, and only the "healthy" embryos are implanted. But most (partially) heritable traits and disease risks are not as easy to predict. Polygenic Scores If what you care about is something like "low cancer risk" or "exceptional athletic ability", it won't be down to a single chromosomal abnormality or a variant in a single gene. Instead, there's typically a statistical relationship where many genes are separately associated with increased or decreased expected value for the trait. This statistical relationship can be written as a polygenic score - given an individual's genome, it'll crunch the numbers and spit out an expected score. That could be a disease risk probability, or it could be an expected value for a trait like "height" or "neuroticism." Polygenic scores are never perfect - some people will be taller than the score's prediction, some shorter - but for a lot of traits they're undeniably meaningful, i.e. there will be a much greater-than-chance correlation between the polygenic score and the true trait measurement. Where do polygenic scores come from? Typically, from genome-wide association studies, or GWAS. These collect a lot of people's genomes (the largest ones can have hundreds of thousands of subjects) and personal data potentially including disease diagnoses, height and weight, psychometric test results, etc. And then they basically run multivariate correlations. A polygenic score is a (usually regularized) multivariate regression best-fit model of the trait as a function of the genome. A polygenic score can give you a rank ordering of genomes, from "best" to "worst" predicted score; it can also give you a "wish list" of gene variants predicted to give a very high combined score. Ideally, "use a polygenic score to pick or generate very high-scoring embryos" would result in babies that have the desired traits to an extraordinary degree. In reality, this depends on how "good" the polygenic scores are - to what extent they're based on causal vs. confounded effects, how much of observed variance they explain, and so on. Reasonable experts seem to disagree on this.[2] I'm a little out of my depth when it comes to assessing the statistical methodology of GWAS studies, so I'm interested in another question - even assuming you have polygenic score you trust, what do you do next? How do you get a high-scoring baby out of it? Massively Multiplexed, Body-Wide Gene Editing? Not So Much, Yet. "Get an IVF embryo and gene-edit it to have the desired genes" (again, ethics and legality aside)[3] is a lot harder than it sounds. First of all, we don't currently know how to make gene edits simultaneously and abundantly in every tissue of the body. Recently approved gene-editing therapies like Casgevy, which treats sickle-cell disease, are operating on easy mode. Sickle-cell disease is a blood disorder; the patient doesn't have enough healthy blood cells, so the therapy consists of an injection of the patient's own blood cells which h...
Link to original articleWelcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Superbabies: Putting The Pieces Together, published by sarahconstantin on July 12, 2024 on LessWrong. This post was inspired by some talks at the recent LessOnline conference including one by LessWrong user "Gene Smith". Let's say you want to have a "designer baby". Genetically extraordinary in some way - super athletic, super beautiful, whatever. 6'5", blue eyes, with a trust fund. Ethics aside[1], what would be necessary to actually do this? Fundamentally, any kind of "superbaby" or "designer baby" project depends on two steps: 1.) figure out what genes you ideally want; 2.) create an embryo with those genes. It's already standard to do a very simple version of this two-step process. In the typical course of in-vitro fertilization (IVF), embryos are usually screened for chromosomal abnormalities that would cause disabilities like Down Syndrome, and only the "healthy" embryos are implanted. But most (partially) heritable traits and disease risks are not as easy to predict. Polygenic Scores If what you care about is something like "low cancer risk" or "exceptional athletic ability", it won't be down to a single chromosomal abnormality or a variant in a single gene. Instead, there's typically a statistical relationship where many genes are separately associated with increased or decreased expected value for the trait. This statistical relationship can be written as a polygenic score - given an individual's genome, it'll crunch the numbers and spit out an expected score. That could be a disease risk probability, or it could be an expected value for a trait like "height" or "neuroticism." Polygenic scores are never perfect - some people will be taller than the score's prediction, some shorter - but for a lot of traits they're undeniably meaningful, i.e. there will be a much greater-than-chance correlation between the polygenic score and the true trait measurement. Where do polygenic scores come from? Typically, from genome-wide association studies, or GWAS. These collect a lot of people's genomes (the largest ones can have hundreds of thousands of subjects) and personal data potentially including disease diagnoses, height and weight, psychometric test results, etc. And then they basically run multivariate correlations. A polygenic score is a (usually regularized) multivariate regression best-fit model of the trait as a function of the genome. A polygenic score can give you a rank ordering of genomes, from "best" to "worst" predicted score; it can also give you a "wish list" of gene variants predicted to give a very high combined score. Ideally, "use a polygenic score to pick or generate very high-scoring embryos" would result in babies that have the desired traits to an extraordinary degree. In reality, this depends on how "good" the polygenic scores are - to what extent they're based on causal vs. confounded effects, how much of observed variance they explain, and so on. Reasonable experts seem to disagree on this.[2] I'm a little out of my depth when it comes to assessing the statistical methodology of GWAS studies, so I'm interested in another question - even assuming you have polygenic score you trust, what do you do next? How do you get a high-scoring baby out of it? Massively Multiplexed, Body-Wide Gene Editing? Not So Much, Yet. "Get an IVF embryo and gene-edit it to have the desired genes" (again, ethics and legality aside)[3] is a lot harder than it sounds. First of all, we don't currently know how to make gene edits simultaneously and abundantly in every tissue of the body. Recently approved gene-editing therapies like Casgevy, which treats sickle-cell disease, are operating on easy mode. Sickle-cell disease is a blood disorder; the patient doesn't have enough healthy blood cells, so the therapy consists of an injection of the patient's own blood cells which h...
Send us a Text Message.We Love Science podcast celebrates our 50th episode with a discussion all about the hopes and progress of CRISPR clinical trials. Before a new drug, therapy, vaccine, or medical device is approved for general use by the public, its safety and effectiveness are evaluated in a series of tests on volunteers. These Clinical Trials progress in 3 successive phases with each phase designed to answer specific questions about the product under evaluation. Questions can include: What side effects does it cause? What is the lowest effective dose? What is the highest safe dose? Does it improve patients' quality of life? Is it an effective cure for the disease in question? How does it compare to the current treatments (if any are available) for that particular disease?Currently, only one CRISPR gene therapy, Casgevy, is approved for public use. It is approved in several countries to treat the blood disorders, Sickle Cell Disease and Beta Thalassemia. However, many CRISPR clinical trials are still ongoing; they span ~ 10 major disease categories and occupy every clinical trial phase. In this episode, we discuss the successes and challenges of CRISPR clinical trials to cure HIV/AIDS, Cardiovascular disease, and others. Tune in to learn more about:· What questions do successive clinical trial phases answer?· How can CRISPR technology cure HIV/AIDS?· The results for the hereditary amyloidosis treatment, which is now in phase III of clinical trials· Listener question: An update for Sickle Cell Disease CRISPR treatmentsFor more information on this topic, visit our website: welovesciencepodcast.comUseful Links: US and EU clinical trials databases: https://clinicaltrials.gov/https://euclinicaltrials.eu/?lang=en Catch up on the previous CRISPR episodes:Ep 35: The first CRISPR Chronicles episode: How CRISPR gene therapy is bringing hope to the worldEp 38: A foundational discussion explaining the secret behind CRISPR gene therapy Ep 41: A look into the ups and downs of how the CRISPR saga developedEp 47: The CRISPR Cure for Sickle Cell Disease Reach out to Fatu:www.linkedin.com/in/fatubmTwitter: @thee_fatu_band LoveSciencePodcast@gmail.com Reach out to Shekerah:www.linkedin.com/in/shekerah-primus and LoveSciencePodcast@gmail.com Music from Pixabay: Future Artificial Intelligence Technology 130 by TimMoorMusic from https://freemusicarchive.org/music/Scott_Holmes: Hotshot by ScottHolmesMusic
In this edition of The Weekly, Managing Editor Jef Akst and News Editor Greg Slabodkin share their insights from the American Society of Gene & Cell Therapy's 27th annual conference, including advances around adeno-associated viruses and the ongoing discussion about the FDA's accelerated approval program and how it relates to rare diseases. Also, on Tuesday, Eli Lilly announced that an advisory committee meeting will be held for its Alzheimer's drug donanemab on June 10. Lilly is aiming for full approval of the anti-amyloid antibody after accelerated approval was denied in January 2023. And the race between Vertex and bluebird bio's gene therapies Casgevy and Lyfgenia is heating up.
In this special edition of The Weekly, we discuss the much-anticipated annual meeting of the American Society of Gene & Cell Therapy in Baltimore. It starts Tuesday and will hit on wide-ranging issues facing the cell and gene therapy (CGT) space, from new therapeutic advances to safety concerns and regulatory considerations to manufacturing and commercialization. Big hitters in the CGT space will be there. Sarepta, whose gene therapy for Duchenne muscular dystrophy Elevidys received accelerated approval in June 2023, will be presenting, as will CRISPR Therapeutics, which in the last few months brought to market Casgevy, its Vertex-partnered CRISPR-based therapy, for sickle-cell disease and beta thalassemia. There will also be plenty of big names, including Peter Marks, director of the FDA's Center for Biologics Evaluation and Research. Marks will be speaking in two sessions at ASGCT 2024, one on global regulatory convergence and the other on the regulation of CGTs, from IND to BLA. Stay tuned for this week's regular episode on Wednesday when we discuss what we're seeing and hearing in Baltimore.
With its potential to expand the horizons of what's possible for patients, CRISPR is the new darling of biotech. Reaching an important milestone in 2023 with the FDA approval of a sickle cell treatment using CRISPR, the technology is poised to break new barriers for treating patients in the near future. In this episode we talk with two companies working together on the next generation of CRISPR: editing cells within the body. We discuss the progress and the challenge in making this breakthrough a reality. Follow us on LinkedIn, X, Facebook and Instagram. Visit us at https://www.bio.org/
Professor Doudna was awarded the 2020 Nobel Prize in Chemistry with Professor Emmanuelle Charpentier for their pioneering work in CRISPR genome editing. The first genome editing therapy (Casgevy) was just FDA approved, only a decade after the CRISPR-Cas9 editing system discovery. But It's just the beginning of a much bigger impact story for medicine and life science.Ground Truths podcasts are now on Apple and Spotify. And if you prefer videos, they are posted on YouTubeTranscript with links to audio and relevant external linksEric Topol (00:06):This is Eric Topol with Ground Truths, and I'm really excited today to have with me Professor Jennifer Doudna, who heads up the Innovative Genomics Institute (IGI) at UC Berkeley, along with other academic appointments, and as everybody knows, was the Nobel laureate for her extraordinary discovery efforts with CRISPR genome editing. So welcome, Jennifer.Jennifer Doudna (00:31):Hello, Eric. Great to be here.Eric Topol (00:34):Well, you know we hadn't met before, but I felt like I know you so well because this is one of my favorite books, The Code Breaker. And Walter Isaacson did such a wonderful job to tell your story. What did you think of the book?My interview with Walter Isaacson on The Code Breaker, a book I highly recommendJennifer Doudna (00:48):I thought Walter did a great job. He's a good storyteller, and as you know from probably from reading it or maybe talking to others about it, he wrote a page turner. He actually really dug into the science and all the different aspects of it that I think created a great tale.Eric Topol (01:07):Yeah, I recommended highly. It was my favorite book when it came out a couple years ago, and it is a page turner. In fact, I just want to read one, there's so many quotes out of it, but in the early part of the book, he says, “the invention of CRISPR and the plague of Covid will hasten our transition to the third great revolution of modern times. These revolutions arose from the discovery beginning just over a century ago, of the three fundamental kernels of our existence, the atom, the bit, and the gene.” That kind of tells a big story just in one sentence, but I thought I'd start with the IGI, the institute that you have set up at Berkeley and what its overall goals are.Jennifer Doudna (01:58):Right. Well, let's just go back a few years maybe to the origins of this institute and my thinking around it, because in the early days of CRISPR, it was clear that we were really at a moment that was quite unique in the sense that there was a transformative technology. It was going to intersect with lots of other discoveries and technologies. And I work at a public institution and my question to myself was, how can I make sure that this powerful tool is first of all used responsibly and secondly, that it's used in a way that benefits as many people as possible, and it's a tall order, but clearly we needed to have some kind of a structure that would allow people to work together towards those goals. And that was really the mission behind the IGI, which was started as a partnership between UC Berkeley and UCSF and now actually includes UC Davis as well.The First FDA Approved Genome EditingEric Topol (02:57):I didn't realize that. That's terrific. Well, this is a pretty big time because 10 years or so, I guess starting to be 11 when you got this thing going, now we're starting to see, well, hundreds of patients have been treated and in December the FDA approved the first CRISPR therapy for sickle cell disease, Casgevy. Is that the way you say it?Jennifer Doudna (03:23):Casgevy, yeah.Eric Topol (03:24):That must have felt pretty good to see if you go from the molecules to the bench all the way now to actually treating diseases and getting approval, which is no easy task.Jennifer Doudna (03:39):Well, Eric, for me, I'm a biochemist and somebody who has always worked on the fundamentals of biology, and so it's really been extraordinary to see the pace at which the CRISPR technology has been adopted, and not just for fundamental research, but also for real applications. And Casgevy is sort of the crowning example of that so far, is that it's really a technology that we can already see how it's being used to, I think it's fair to say, effectively cure a genetic disease for the first time. Really amazing.Genome Editing is Not the Same as Gene TherapyEric Topol (04:17):Yeah. Now I want to get back to that. I know there's going to be refinements about that. And of course, there's beta thalassemia, so we've got two already, and our mutual friend Fyodor Urnov would say two down 5,000 to go. But I think before I get to the actual repair of the sickle cell defect molecular defect, I think one of the questions I think that people listeners may not know is the differentiation of genome editing with gene therapy. I mean, as you know, there was recently a gene therapy approval for something like $4.25 million for metachromatic leukodystrophy. So maybe you could give us kind of skinny on how these two fundamental therapies are different.Jennifer Doudna (05:07):Right. Well, it's a great question because the terminology sounds kind of the same, and so it could be confusing. Gene therapy goes back decades, I can remember gene therapy being discussed as an exciting new at the time, direction back when I was a graduate student. That was little while ago. And it refers to the idea that we can use a genetic approach for disease treatment or even for a cure. However, it fundamentally requires some mechanism of integrating new information into a genome. And traditionally that's been done using viruses, which are great at doing that. It's just that they do it wherever they want to do it, not necessarily where we want that information to go. And this is where CRISPR comes in. It's a technology allows precision in that kind of genetic manipulation. So it allows the scientist or the clinician to decide where to make a genetic change. And that gives us tremendous opportunity to do things with a kind of accuracy that hasn't been possible before.Eric Topol (06:12):Yeah, no question. That's just a footnote. My thesis in college at University of Virginia, 1975, I'm an old dog, was prospects for gene therapy in man. So it took a while, didn't it? But it's a lot better now with what you've been working on, you and your colleagues now and for the last decade for sure. Now, what I was really surprised about is it's not just of course, these hemoglobin disorders, but now already in phase two trials, you've got hereditary angioedema, which is a life-threatening condition, amyloidosis, cancer ex vivo, and also chronic urinary tract infections. And of course, there's six more others like autoimmune diseases like lupus and type 1 diabetes. So this is really blossoming. It's really extraordinary.Eric Topol (07:11):I mean, wow. So one of the questions I had about phages, because this is kind of going back to this original work and discovery, antimicrobial resistance is really a big problem and it's a global health crisis, and there's only two routes there coming up with new drugs, which has been slow and not really supported by the life science industry. And the other promising area is with phages. And I wonder, since this is an area you know so well, why haven't we put more, we're starting to see more trials in phages. Why haven't we doubled down or tripled down on this to help the antimicrobial resistance problem?Jennifer Doudna (08:00):Well, it's a really interesting area, and as you said, it's kind of one of those areas of science where I think there was interest a while ago and some effort was made for reasons that are not entirely clear to me, at least it fizzled out as a real focused field for a long time. But then more recently, people have realized that there's an opportunity here to take advantage of some natural biology in which viruses can infect and destroy microbes. Why aren't we taking better advantage of that for our own health purposes? So I personally am very excited about this area. I think there's a lot of fundamental work still to be done, but I think there's a tremendous opportunity there as well.CRISPR 2.0Eric Topol (08:48):Yeah, I sure think we need to invest in that. Now, getting back to this sickle cell story, which is so extraordinary. This is kind of a workaround plan of getting fetal hemoglobin built up, but what about actually repairing, getting to fixing the lesion, if you will?Eric Topol (09:11):Yeah. Is that needed?Jennifer Doudna (09:13):Well, maybe it's worth saying a little bit about how Casgevy works, and you alluded to this. It's not a direct cure. It's a mechanism that allows activation of a second protein called fetal hemoglobin that can suppress the effect of the sickle cell mutation. And it's great, and I think for patients, it offers a really interesting opportunity with their disease that hasn't been available in the past, but at the same time, it's not a true cure. And so the question is could we use a CRISPR type technology to actually make a correction to the genetic defect that directly causes the disease? And I think the answer is yes. The field isn't there quite yet. It's still relatively difficult to control the exact way that DNA editing is occurring, especially if we're doing it in vivo in the body. But boy, many people are working on this, as you probably know. And I really think that's on the horizon.Eric Topol (10:19):Yeah. Well, I think we want to get into the in vivo story as well because that, I think right now it's so complicated for a person to have to go through the procedure to get ultimately this treatment currently for sickle cell, whereas if you could do this in vivo and you could actually get the cure, that would be of the objective. Now, you published just earlier this month in PNAS a wonderful paper about the EDVs and the lipid nanoparticles that are ways that we could get to a better precision editing. These EDVs I guess if I have it right, enveloped virus-like particles. It could be different types, it could be extracellular vesicles or whatnot. But do you think that's going to be important? Because right now we're limited for delivery, we're limited to achieve the right kind of editing to do this highly precise. Is that a big step for the future?Jennifer Doudna (11:27):Really big. I think that's gating at the moment. Right now, as you mentioned, somebody that might want to get the drug Casgevy for sickle cell disease or thalassemia, they have to go through a bone marrow transplant to get it. And that means that it's very expensive. It's time consuming. It's obviously not pleasant to have to go through that. And so that automatically means that right now that therapy is quite restricted in the patients that it can benefit. But we imagine a day when you could get this type of therapy into the body with a one-time injection. Maybe someday it's a pill that could be taken where the gene editors target the right cells in the body. In diseases like that, it would be the stem cells in the bone marrow and carry out gene editing that can have a therapeutic benefit. And again, it's one of those ideas that sounds like science fiction, and yet already there's tremendous advance in that direction. And I think over the next, I don't know, I'm guessing 5 to 10 years we're going to see that coming online.Editing RNA, the Epigenome, and the MicrobiomeEric Topol (12:35):Yeah, I'm guessing just because there's so much work on the lipid nanoparticles to tweak them. And there's four different components that could easily be made so much better. And then all these virus-like proteins, I mean, it may happen even sooner. And it's really exciting. And I love that diagram in that paper. You have basically every organ of the body that isn't accessible now, potentially that would become accessible. And that's exciting because whatever blossoming we're seeing right now with these phase two trials ongoing, then you basically have no limits. And that I think is really important. So in vivo editing big. Now, the other thing that's cropped up in recent times is we've just been focused on DNA, but now there's RNA editing, there's epigenetic or epigenomic editing. What are your thoughts about that?Jennifer Doudna (13:26):Very exciting as well. It's kind of a parallel strategy. The idea there would be to, rather than making a permanent change in the DNA of a cell, you could change just the genetic output of the cell and or even make a change to DNA that would alter its ability to be expressed and to produce proteins in the cell. So these are strategies that are accessible, again, using CRISPR tools. And the question is now how to use them in ways that will be therapeutically beneficial. Again, topics that are under very active investigation in both academic labs and at companies.Eric Topol (14:13):Yeah. Now speaking of that, this whole idea of rejuvenation, this is Altos. You may I'm sure know my friend here, Juan Carlos Belmonte, who's been pushing on this for some time at Altos now formerly at Salk. And I know you helped advise Altos, but this idea of basically epigenetic, well using the four Yamanaka factors and basically getting cells that go to a state that are rejuvenated and all these animal models that show that it really happens, are you thinking that really could become a therapy in the times ahead in patients for aging or particular ideas that you have of how to use that?Jennifer Doudna (15:02):Well, you mentioned the company Altos. I mean, Altos and a number of other groups are actively investigating this. Not I would say specifically regarding genome editing, although being able to monitor and probably change gene functions that might affect the aging process could be attractive in the future. I think the hard question there is which genes do we tweak and how do we make sure that it's safe? And better than me I mean, that's a very difficult thing to study clinically because it takes time for one thing, and we probably don't have the best models either. So I think there are challenges there for sure. But along the way, I feel very excited about the kind of fundamental knowledge that will come from those studies. And in particular, this question of how tissues rejuvenate I think is absolutely fascinating. And some organisms do this better than others. And so, understanding how that works in organisms that are able to say regrow a limb, I think can be very interesting.Eric Topol (16:10):And that gets me to that recent study. Well, as you well know, there's a company Verve that's working on the familial hypercholesterolemia and using editing with the PCSK9 through the liver and having some initial, at least a dozen patients have been treated. But then this epigenetic study of editing in mice for PCSK9 also showed results. Of course, that's much further behind actually treating patients with base editing. But it's really intriguing that you can do some of these things without having to go through DNA isn't it?Jennifer Doudna (16:51):Amazing, right? Yeah, it's very interesting.Reducing the Cost of Genome EditingEric Topol (16:54):Wild. Now, one of the things of course that people bring up is, well, this is so darn expensive and it's great. It's a science triumph, but then who can get these treatments? And recently in January, you announced a Danaher-IGI Beacon, and maybe you can tell us a bit about that, because again, here's a chance to really markedly reduce the cost, right?Jennifer Doudna (17:25):That's right. That's the vision there. And huge kudos to my colleague Fyodor Urnov, who really spearheaded that effort and leads the team on the IGI side. But the vision there was to partner with a company that has the ability to manufacture molecules in ways that are very, very hard, of course, for academic labs and even for most companies to do. And so the idea was to bring together the best of genome editing technology, the best of clinical medicine, especially focused on rare human diseases. And this is with our partners at UCSF and with the folks in the Danaher team who are experts at downstream issues of manufacturing. And so the hope there is that we can bring those pieces together to create ways of using CRISPR that will be cost effective for patients. And frankly, we'll also create a kind of roadmap for how to do this, how to do this more efficiently. And we're kind of building the plane while we're flying it, if you know what I mean. But we're trying to really work creatively with organizations like the FDA to come up with strategies for clinical trials that will maintain safety, but also speed up the timeline.Eric Topol (18:44):And I think it's really exciting. We need that and I'm on the scientific advisory board of Danaher, a new commitment for me. And when Fyodor presented that recently, I said, wow, this is exciting. We haven't really had a path to how to get these therapies down to a much lower cost. Now, another thing that's exciting that you're involved in, which I think crosses the whole genome editing, the two most important things that I've seen in my lifetime are genome editing and AI, and they also work together. So maybe before we get into AI for drug discovery, how does AI come into play when you're thinking about doing genome editing?Jennifer Doudna (19:34):Well, the thing about CRISPR is that as a tool, it's powerful not only as a one and done kind of an approach, but it's also very powerful genomically, meaning that you can make large libraries of these guide RNAs that allow interrogation of many genes at once. And so that's great on the one hand, but it's also daunting because it generates large collections of data that are difficult to manually inspect. And in some cases, I believe really very, very difficult to analyze in traditional ways. But imagine that we have ways of training models that can look at genetic intersections, ways that genes might be affecting the behavior of not only other genes, but also how a person responds to drugs, how a person responds to their environment and allows us to make predictions about genetic outcomes based on that information. I think that's extremely exciting, and I definitely think that over the next few years we'll see that kind of analysis coming online more and more.Eric Topol (20:45):Yeah, the convergence, I think is going to be, it's already being done now, but it's just going to keep building. Now, Demis Hassabis, who one of the brilliant people in the field of AI leads the whole Google Deep Mind AI efforts now, but he formed after AlphaFold2 behaving to predict proteins, 200 million proteins of the universe. He started a company Isomorphic Labs as a way to accelerate using AI drug discovery. What can you tell us about that?Jennifer Doudna (21:23):It's exciting, isn't it? I'm on the SAB for that company, and I think it's very interesting to see their approach to drug discovery. It's different from what I've been familiar with at other companies because they're really taking a computational lens to this challenge. The idea there is can we actually predict things like the way a small molecule might interact with a particular protein or even how it might interact with a large protein complex. And increasingly because of AlphaFold and programs like that, that allow accurate prediction of structures, it's possible to do that kind of work extremely quickly. A lot of it can be done in silico rather than in the laboratory. And when you do get around to doing experiments in the lab, you can get away with many fewer experiments because you know the right ones to do. Now, will this actually accelerate the rate at which we get to approved therapeutics? I wonder about your opinion about that. I remain unsure.Editing Out Alzheimer's Risk AllelesEric Topol (22:32):Yeah. I mean, we have one great success story so far during the pandemic Baricitinib, a drug that repurposed here, a drug that was for rheumatoid arthritis, found by data mining that have a high prospects for Covid and now saves lives in Covid. So at least that's one down, but we got a lot more here too. But it, it's great that Demis recruited you on the SAB for Isomorphic because it brings in a great mind in a different field. And it goes back to one of the things you mentioned earlier is how can we get some of this genome editing into a pill someday? Wow. Now, one of the things that for personal interest, as an APOE4 carrier, I'm looking to you to fix my APOE4 and give me APOE2. How can I expect to get that done in the near future?Jennifer Doudna (23:30):Oh boy. Okay, we'll have to roll up our sleeves on that one. But it is appealing, isn't it? I think about it too. It's a fascinating idea. Could we get to a point someday where we can use genome editing as a prophylactic, not as a treatment after the fact, but as a way to actually protect ourselves from disease? And the APOE4 example is a really interesting one because there's really good evidence that by changing the type of allele that one has for the APOE gene, you can actually affect a person's likelihood of developing Alzheimer's in later life. But how do we get there? I think one thing to point out is that right now doing genome editing in the brain is, well, it's hard. I mean, it's very hard.Eric Topol (24:18):It a little bit's been done in cerebral spinal fluid to show that you can get the APOE2 switch. But I don't know that I want to sign up for an LP to have that done.Jennifer Doudna (24:30):Not quite yet.Eric Topol (24:31):But someday it's wild. It's totally wild. And that actually gets me back to that program for coronary heart disease and heart attacks, because when you're treating people with familial hypercholesterolemia, this extreme phenotype. Someday and this goes for many of these rare diseases that you and others are working on, it can have much broader applicability if you have a one-off treatment to prevent coronary disease and heart attacks and you might use that for people well beyond those who have an LDL cholesterol that are in the thousands. So that's what I think a lot of people don't realize that this editing potential isn't just for these monogenic and rare diseases. So we just wanted to emphasize that. Well, this has been a kind of wild ride through so much going on in this field. I mean, it is extraordinary. What am I missing that you're excited about?Jennifer Doudna (25:32):Well, we didn't talk about the microbiome. I'll just very briefly mention that one of our latest initiatives at the IGI is editing the microbiome. And you probably know there are more and more connections that are being made between our microbiome and all kinds of health and disease states. So we think that being able to manipulate the microbiome precisely is going to open up another whole opportunity to impact our health.Can Editing Slow the Aging Process?Eric Topol (26:03):Yeah, I should have realized that when I only mentioned two layers of biology, there's another one that's active. Extraordinary, just going back to aging for a second today, there was a really interesting paper from Irv Weissman Stanford, who I'm sure you know and colleagues, where they basically depleted the myeloid stem cells in aged mice. And they rejuvenated the immune system. I mean, it really brought it back to life as a young malice. Now, there probably are ways to do that with editing without having to deplete stem cells. And the thought about other ways to approach the aging process now that we're learning so much about science and about the immune system, which is one of the most complex ones to work in. Do you have ideas about that are already out there that we could influence the aging process, especially for those of us who are getting old?Jennifer Doudna (27:07):We're all on that path, Eric. Well, I guess the way that I think about it is I like to think that genome editing is going to pave the way to make those kinds of fundamental discoveries. I still feel that there's a lot of our genetics that we don't understand. And so, by being able to manipulate genes precisely and increasingly to look at how genes interact with each other, I think one fundamental question it relates to aging actually is why do some of us age at a seemingly faster pace than others? And it must have to do at least in part with our genetic makeup and how we respond to our environment. So I definitely think there are big opportunities there, really in fundamental research initially, but maybe later to actually change those kinds of things.Eric Topol (28:03):Yeah, I'm very impressed in recent times how much the advances are being made at basic science level and experimental models. A lot of promise there. Now, is there anything about this field that you worry about that keeps you up at night that you think, besides, we talked about that we got to get the cost down, we have to bridge health inequities for sure, but is there anything else that you're concerned about right now?Jennifer Doudna (28:33):Well, I think anytime a new technology goes into clinical trials, you worry that things may get out ahead of their skis, and there may be some overreach that happens. I think we haven't really seen that so far in the CRISPR field, which is great. But I guess I remain cautious. I think that we all saw what happened in the field of gene therapy now decades ago, but that really put a poll on that field for a long time. And so, I definitely think that we need to continue to be very cautious as gene editing continues to advance.Eric Topol (29:10):Yeah, no question. I think the momentum now is getting past that point where you would be concerned about known unknowns, if you will, things that going back to the days of the Gelsinger crisis. But it's really extraordinary. I am so thrilled to have this conversation with you and to get a chance to review where the field is and where it's going. I mean, it's exploding with promise and potential well beyond and faster. I mean, it takes a drug 17 years, and you've already gotten this into two treatments. I mean, I'm struck when you were working on this, how you could have thought that within a 10-year time span you'd already have FDA approvals. It's extraordinary.Jennifer Doudna (30:09):Yeah, we hardly dared hope. Of course, we're all thrilled that it went that fast, but I think it would've been hard to imagine it at the time.Eric Topol (30:17):Yeah. Well, when that gets simplified and doesn't require hospitalizations and bone marrow, and then you'll know you're off to the races. But look, what a great start. Phenomenal. So congratulations. I'm so thrilled to have the chance to have this conversation. And obviously we're all going to be following your work because what a beacon of science and progress and changing medicine. So thanks and give my best to my friend there at IGI, Fyodor, who's a character. He's a real character. I love the guy, and he's a good friend.Jennifer Doudna (30:55):I certainly will Eric, and thank you so much. It's been great talking with you.*******************************************************Thanks for listening and/or reading this edition of Ground Truths.I hope you found it as stimulating as I did. Please share if you did!A reminder that all Ground Truths posts (newsletter and podcast( are free without ads. Soon we'll set it up so you can select what type of posts you want to be notified about.If you wish to be a paid subscriber, know that all proceeds are donated to Scripps Research, and thanks for that—it greatly helped fund our summer internship program for 2023 and 2024.Thanks to my producer Jessica Nguyen and to Sinjun Balabanoff for audio/video support. Get full access to Ground Truths at erictopol.substack.com/subscribe
Monica Bertagnolli went from a childhood on a cattle ranch to a career as a surgeon to a top post in the Biden administration. As director of the National Institutes of Health, she's working to improve the way we find new treatments — despite regulatory constraints and tight budgets. SOURCE:Monica Bertagnolli, director of the National Institutes of Health. RESOURCES:"Steven Levitt and John Donohue Defend a Finding Made Famous by 'Freakonomics,'" by Steven Levitt and John Donohue (The Economist, 2024)."Why 'Freakonomics' Failed to Transform Economics," (The Economist, 2024)."Steven D. Levitt (Freakonomics Co-Author and U Chicago Econ Prof) on His Career and Decision to Retire From Academic Economics," by Jon Hartley (The Capitalism and Freedom in the Twenty-First Century Podcast, 2024)."Why Autoimmune Disease Is More Common in Women: X Chromosome Holds Clues," by Elie Dolgin (Nature, 2024)."Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease," by Carrie MacMillan (Yale Medicine News, 2023)."Fact Sheet: President Biden Reignites Cancer Moonshot to End Cancer as We Know It," (2022)."Mini-Antibodies Discovered in Sharks and Camels Could Lead to Drugs for Cancer and Other Diseases," by Mitch Leslie (Science, 2018). EXTRAS:"Who Pays for Multimillion-Dollar Miracle Cures?" by Freakonomics, M.D. (2023)."What's Stopping Us From Curing Rare Diseases?" by Freakonomics, M.D. (2023)."Abortion and Crime, Revisited (Update)," by Freakonomics Radio (2022)."John Donohue: 'I'm Frequently Called a Treasonous Enemy of the Constitution,'" by People I (Mostly) Admire (2021).
Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Astrazeneca acquired a biotech startup called Amolyt for $800 million, adding a late-stage drug prospect for parathyroidism to its rare disease portfolio. Bio, a lobbying group for the biopharmaceutical industry, changed its stance and now supports a bill to limit China's role in US biotech. German biotech Tubulis secured €128 million in financing to capitalize on the momentum in antibody-drug conjugates (ADCs). J&J and Novo are supporting Asgard's efforts to develop personalized cancer therapies that reprogram tumor cells in the body. The FDA is focusing on early deaths in a meeting regarding broader CAR-T use in myeloma, particularly in trials from Bristol Myers Squibb and Johnson & Johnson. Additionally, there are five questions facing emerging biotech companies as they navigate through the industry's current landscape. The newsletter also covers various topics including the use of AI-enabled digital twins to secure the pharmaceutical supply chain, Medicare coverage of weight-loss drugs, and shifts in the clinical trial landscape.The EPA has issued a final rule limiting ethylene oxide emissions from medical device sterilizers, giving companies two years to comply. Exactech received a warning letter from the FDA regarding faulty implant packaging analysis. US hospitals expect an increase in procedures as staffing pressures ease, potentially benefiting companies like Boston Scientific, Medtronic, and Stryker. The FDA is seeking feedback on expanding premarket cybersecurity guidance. Additionally, AI and digital health trends are becoming more prominent in the medical device industry.AstraZeneca has acquired Amolyt Pharma for $1.05 billion, adding to its rare disease portfolio with late-stage candidate eneboparatide for hypoparathyroidism. The deal also includes ownership of azp-3813, being assessed for acromegaly in a phase I trial. In other news, Vertex failed to convince the UK watchdog of the value of its CRISPR therapy Casgevy, and Wuxi AppTec has been removed from a trade group amid US national security concerns. ADC Biotech Tubulis closed a $138 million financing round to support its lead solid tumor antibody-drug conjugate candidates.The Biden administration has proposed the implementation of "march-in rights" to seize patents for drugs that are priced unreasonably high. This move has sparked debate among stakeholders, with concerns about the potential negative impact on innovation in drug development. Despite efforts to bring down drug prices, critics believe that federal intervention in patent protection could have devastating consequences.The biosimilars market in the U.S. has been limited by patent thickets and insurance contracting, but there are signs of momentum. CVS has launched a new venture in biosimilar drug experimentation, while biosimilar makers are using different strategies to compete with top-selling drugs like Humira. Sandoz has spun out of Novartis to become a standalone generic drugmaker.Thank you for listening to Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.
I'm very sorry for my absence. My son and I fell very sick with a stomach bug and we are finally back rejoining the world. That was rough. Thanks for your patience. Whenever I'm sick I am reminded of the quote: “A healthy person wants a million things. A sick person wants just one.” This made me want to tell the story of the medical therapy ATA188. A month ago I shared the story of an incredible invention that will cure the terrible pain from Sickle Cell Disease. Miraculously, there are now two drugs approved by the FDA that completely cure this disease. It is truly incredible. But we also discussed that the drugs Casgevy and Lyfgenia are priced at $2 million to $3 million per treatment! A shocking price tag to say the least. It's understandable why someone might look at that price tag and get angry with the company that is selling the products. How dare they charge millions of dollars for something that is supposed to help cure people? This brings up a big hairy question: How much should medical treatments cost? How should they be priced? In the case of these two drugs, the alternative treatment for the patients costs $4 million over a lifetime and doesn't provide relief from pain. So even at $2-$3million, the new treatment is less expensive, better for the patient, and saves the patient from a lot of ongoing pain. But even so, I can totally understand why someone would still look at the $3 Million price tag and think that the pharmaceutical company that is selling the drug is a money hungry monster. I get it. But not all drugs that are tested are successful like Casgevy and Lyfgenia. Take for instance, ATA188 from Atara Biotherapeutics. ATA188 is an immune therapy designed to treat multiple sclerosis (MS). This therapy is working on the newly developed theory that MS might be caused by the Epstein-Barr virus (EBV) in someway. This really cool study of over 10,000,000 military personnel showed a strong correlation between EBV and MS. ATA188 hoped to use healthy t-cells to target EBV cells to reduce or halt the progression of MS, offering a new approach to treating this chronic disease.I have two close family members that suffer with MS so I have been following this treatment fairly closely in hopes that it could offer some answers and potentially a solution for my loved one. Year Of The Opposite - Travis Stoliker's Substack is a reader-supported publication. To receive new posts and support my work, consider becoming a free or paid subscriber.A little history on drug/therapy development: Medical treatments like drugs and immunotherapies like ATA188 usually involve many partners and organizations that work together to test and develop these medical inventions. For ATA188, Memorial Sloan-Kettering Cancer Center and QIMR Berghofer Medical Research Institute were involved. Atara Biotherapeutics was the company that was commercializing the technology. What does this mean? Well basically the universities are often the ones that invent the new therapy or drug but they aren't that great at testing, getting FDA approval, and bringing the drug/therapy to market so that patients can buy the product to help them. So usually the Universities partner with a company or a bunch of investors to help get the innovation from the lab into the real world. That's where a company like Atara Biotherapeutics comes in. Atara enters into a licensing deal with the University and the people that invented ATA188 where, in most cases, the University/Inventor give the company a license to commercialize the invention, and in exchange they get fees and royalties if the product is sold to customers and patients. But, the catch for Atara Biotherapeutics is that now they must pay for the testing and trials that go into getting the drug/therapy approved. This can take a very long time and cost a hell of a lot of money. For instance, the phase 1 trial for ATA188 was way back in 2017. As you may recall, in most cases there are 3 phases of trials. I'm greatly simplifying this but in general here is what the trials do: * Phase 1: Get a small group of people, give them the drug/treatment, make sure it's safe and test what dosage to give. * Phase 2: Get a larger group of people and make sure it's safe and figure out what the best dosage to give is. * Phase 3: Get an even larger group to test that the drug is safe and evaluate how it compares to the alternative treatments on the market and placebo. All of us in the MS community were thrilled when we saw the results of the Phase 1 trial. 9 of the 24 people in the trial had improvement in their disability! This got a lot of us very excited that we may finally have some relief for MS patients. Of course, this early trial data got Atara Biotherapeutics excited too! With this result they wanted to put more money into funding Phase 2 trials. Here is how they designed the Phase 2 study: “The Phase 2 EMBOLD study for ATA188 is a randomized, double-blind, placebo-controlled dose-expansion trial designed to evaluate the efficacy and safety of ATA188 in patients with progressive multiple sclerosis (PMS). The study, which began with the enrollment of the first patient in June 2020, aimed to measure changes in disability measures compared to baseline, particularly sustained disability improvement (SDI) over time. Additionally, the study included multiple measures of patients' function as well as various biomarkers.”So to recap the timeline, the phase 1 study started in 2017 and the phase 2 portion of the study didn't start until mid 2020. This is one of the things that contributes to the cost of drugs. It just takes a long time to test them and make sure they are safe. Not only did it take 3 years from phase 1 to phase 2, but phase two itself was designed to last 2 years. Sadly, in November of 2023, we got the first results from the Phase 2 trials and they were bad. The therapy had failed. Here is the technical jargon: “ATA188 did not meet its primary endpoint of change in confirmed disability improvement among patients with non-active progressive multiple sclerosis after 12 months of treatment. A 6% disability improvement was observed in the ATA188 group at the 12-month findings, which was significantly lower than the 33% observed in the Phase 1 study. The expected rate of confirmed disability improvement for patients on placebo was 16% at 12 months, exceeding the 4% to 6% expected rate.”Basically, after 6 years of testing and a lot of hope and promise, the people that received the treatment in the study did WORSE than those that received a placebo. Somehow 16% of the people that received the placebo got better but only 6% of the people that received the treatment. How could that be? How could people that didn't get any treatment (placebo) do better than those that got the treatment? — We don't know. But this is exactly why we do these tests. Not only do we have to make sure they are safe, but we also have to make sure that they perform better than a placebo. So what happens with ATA188? It's not certain yet. Atara Biotherapeutics has fired 25% of their staff including a VP and their Chief Medical Officer. The severance payments to those employees alone, just for firing them, was more than $4million. So how much did Atara Biotherapeutics spend on ATA188? It's not easy to tell exactly how much was spent on ATA188, but what we do know is that Atara spent a lot on research and development: * In 2020, the total R&D expenses were $244.65 million.* In 2021, the total increased slightly to $282.001 million.* In 2022, the total slightly decreased to $272.533 million Now of course, not all of that money was spent on ATA188 and these are just 2020, 2021 and 2022 numbers. So lets be very conservative, let's say that $150,000,000 was spent to test ATA188. I think that is a very conservative number, but I can't be sure. That money is now gone. $150,000,000 out the door, never to return. Not only is the money gone, but several medical professionals spent their time and effort for almost 7 years working on the testing for this drug. That time and effort is all gone now. Where did the $150 million come from? Atara is a publicly traded company meaning that anyone of the public can purchase shares in the company. The owners of the company are the ones that lost the $150,000,000. Take a look at their stock price: Anyone that invested in Atara for the past 5 years, most likely lost a lot of money. So why did they invest? They invested because they believed ATA188 would work. Maybe they were suffering with MS so they wanted to put their money into a company that might provide a cure. Maybe they were just money seeking investors that read the Phase 1 study, got excited, and thought they could invest in the next Novo Nordisk stock that was going to the moon! Whatever their rational, the investors that put money into Atara were the ones that took the financial risk to test ATA188. They risked their own capital to see if this treatment could help other humans. On this bet, they lost. This drug failed and it probably cost them about $150,000,000! An incredible amount of money. Hard to imagine really. It's estimated that only 1 in 5,000 drugs/therapies that are tested make it to human clinical trials. And of the drugs / therapies that make it to human clinical trials, only 1 in 10 will ever get regulatory approval. That's about a 0.002% chance that a therapy may be approved! So why would anyone take the risk with their own money when there is only a 0.002% chance of success? It's easy to answer: They invest for the chance to make a lot of money. Think about it like this…Let's say you are in vegas and you walk past a slot machine that says there is a .002% chance of winning. If you put in $1 you have a .002% chance of winning $10. Would you do it? Probably not. But what about $1 bet for a .002% chance to win $1,000? Well now it's a bit more exciting. What about $1 bet for a .002% chance at winning $1,000,000,000? That's a great bet to take! As much as we like to think of medical inventions as being altruistic inventions that are solely accomplished by the brilliant scientist in a lab - that's not how it actually works. The drug and therapy discovery and invention process is a beautiful dance between several parties. The universities and government providing foundational support and investment, the government evaluating the safety and effectiveness of the treatments, business people establishing companies to commercialize these inventions, and investors providing the financial support to fund the testing. So how does this all relate to the $2-$3 million Sickle Cell Therapy treatments? Thanks for sticking with me as I got to the point. While I can see how someone would look at a $3million treatment and be shocked and outraged - we need to keep in mind how many ATA188's are out there. How many drugs were tested that never saw the light of day? How much money was spent on developing drugs that didn't get approved? We must keep that in mind when thinking of healthcare costs and drug costs. There is a delicate balance that needs to be struck for a healthy society. We don't want our health care costs to run out of control and eat up our economy. But we also want to make sure that we keep incentivizing new invention and drug discovery. In order to do this we need: * scientists that are incentivized financially to keep inventing new treatments* investors that are incentivized financially to keep investing in new treatments. I firmly believe that we need both of these things in order to maximize our society's ability to invent new medical breakthroughs. As much as we like to hope that our medical inventions are done by noble scientists that don't care about money - that doesn't seem to be the reality. Some people argue that we need to remove the “profit motive” from healthcare in order to serve patients better. I can sympathize with this view because it makes emotional sense. You don't, for instance, want someone deciding who lives or dies based on how much money they have. I totally understand that argument. But when it comes to medical invention and discovery, the profit motive is a very important component in my mind. To me personally, I think the investors that risked $150,000,000 of their own money with the hopes that Atara would invent a cure for MS are heroes! They risked a lot of their own capital in hopes that it would help other humans. It didn't work out this time and they lost all or most of their money. But if that investment had worked out, for me personally, I would hope that every single investor in ATA188 would be rewarded for their risk by getting filthy rich. Not because they are “profiting off the sick” but because they were the ones that invested in making the sick healthy. And to me, that's the kind of investments we should be rewarding as a society. Year Of The Opposite - Travis Stoliker's Substack is a reader-supported publication. To receive new posts and support my work, consider becoming a free or paid subscriber. Get full access to Year Of The Opposite - Travis Stoliker's Substack at www.yearoftheopposite.com/subscribe
2023 ended with an exciting biotech breakthrough for sickle cell patients. A gene-editing therapy using the revolutionary CRISPR technology provides new hope and options for the nearly 100,000 Americans with sickle cell disease. In this episode, we talk with the company behind one of the life-changing therapies, CASGEVY, and speak with two advocates for sickle cell patients. Follow us on LinkedIn, X, Facebook and Instagram. Visit us at https://www.bio.org/
In today's episode, intense weather resulted in scenes dubbed as "biblical destruction". Meanwhile, the European Commission has greenlighted the use of Casgevy gene therapy to treat beta thalassaemia and sickle cell disease. Elsewhere, the "pay-as-you-throw" scheme is set to hit households in Cyprus in the pocket. All this and more in the Cyprus Beat briefing brought to you by the Cyprus Mail.
Last month the FDA approved a new treatment for sickle cell disease, the first medical therapy to use CRISPR gene editing technology. It works by identifying the gene or genes causing the disorder, modifying those genes and then returning them to the patient's body.There are now two gene therapies offered by pharmaceutical companies for sickle cell disease: Casgevy from Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia from BlueBird Bio. But prices for these one-time treatments are steep: Casgevy costs $2.2 million per patient and Lyfgenia $3.1 million.Both promise a full cure, which would be life-changing for patients with this debilitating condition. Over 100,000 Americans, mostly of African descent, have sickle cell disease.This milestone raises more questions: What will be the next disease that CRISPR can help cure? And is it possible to reduce the costs of gene therapy treatments?Ira talks with Dr. Fyodor Urnov, professor of molecular and cell biology and scientific director of technology and translation at the Innovative Genomics Institute, based at the University of California, Berkeley, about the future of CRISPR-based cures.Transcripts for this segment will be available the week after the show airs on sciencefriday.com. To stay updated on all things science, sign up for Science Friday's newsletters.
What does the body of evidence say on microplastics and their impact on our health? Plus: we talk about what we have learned about long COVID since 2020, and we discuss the very first approval for a clinical application of the gene-editing technology known as CRISPR. A Block: Microplastics (0:58) What are micro- and nanoplastics; microplastics in the environment and in our bodies; how they get into humans and animals; the infamous study that was retracted; occupational exposure to microplastics versus casual exposure; eating microplastics by consuming seafood; can they cause infertility; BPA and BPS; the latest study on microplastics; are plastic water filters bad; should you avoid bottled water; alternatives to single-use plastic bottles. B Block: Long COVID Update (37:55) The problems with diagnosing it; how to treat it; the involvement of the mitochondria; probiotics for long COVID; should people with long COVID exercise; the COVID vaccine protects against long COVID; direct-to-participant digitized clinical trials. C Block: CRISPR Therapy Approved (56:22) CRISPR therapy approved in the UK and US for sickle cell disease and transfusion-dependent beta thalassemia. Jonathan initially mischaracterizes the therapy as repairing the mutation. Chris corrects him. Don't email us, thank you! * Theme music: “Fall of the Ocean Queen“ by Joseph Hackl * Assistant researcher: Aigul Zaripova To contribute to The Body of Evidence, go to our Patreon page at: http://www.patreon.com/thebodyofevidence/. To make a one-time donation to our show, you can now use PayPal! https://www.paypal.com/donate?hosted_button_id=9QZET78JZWCZE Patrons get a bonus show on Patreon called “Digressions”! Check it out! References: 1) Background on micro- and nanoplastics: https://montrealgazette.com/opinion/columnists/christopher-labos-is-bottled-water-bad-for-your-health 2) The recent study published in PNAS: https://doi.org/10.1073/pnas.2300582121 3) The retracted study: https://www.nature.com/articles/nature.2017.21929 4) The WHO 2022 report on microplastics: https://www.who.int/publications/i/item/9789240054608 5) What kind of reusable bottle is best: https://wapo.st/3O1yy3R 6) Review article on long COVID from a Chinese team: https://www.nature.com/articles/s41392-023-01640-z 7) Mitochondria are involved in long COVID: https://doi.org/10.1126/scitranslmed.abq1533 8) Dr. Eric Topol's commentary on the mitochondria paper: https://erictopol.substack.com/p/long-covid-mitochondria-the-big-miss 9) The positive clinical trial on probiotics for long COVID: https://doi.org/10.1016/S1473-3099(23)00685-0 10) Exercising can be bad for long COVID: https://www.nature.com/articles/s41467-023-44432-3 11) At least 3 doses of the COVID vaccine seem to provide a 70% reduction in the risk of getting long COVID: https://erictopol.substack.com/p/sotp-state-of-the-pandemic 12) What happened to the NIH RECOVER Long COVID initiative funds: https://erictopol.substack.com/p/long-covid-mitochondria-the-big-miss 13) Direct-to-participant digitized clinical trials: https://erictopol.substack.com/p/long-covid-mitochondria-the-big-miss 14) Information on CASGEVY: https://pi.vrtx.com/files/patientpackageinsert_exagamglogene_autotemcel.pdf It's Not Twitter, But It'll Do: 1) Jonathan's article on mindfulness meditation: https://www.mcgill.ca/oss/article/critical-thinking-health-and-nutrition/mindfulness-meditation-often-fails-scientific-test 2) Jonathan's article on the graveyard of skeptical projects: https://www.skeptic.org.uk/2024/01/the-graveyard-of-skeptical-projects-suggests-its-funding-not-talent-that-were-lacking/ 3) Chris featured at Darwin Day on February 10th for the Humanist Association of Toronto: https://www.humanisttoronto.ca/events-1/hat-forum-240127-g3ja3-3g8g9 4) Chris being interviewed at Indigo Pointe-Claire by Ken Connors on February 17: https://www.facebook.com/drlabos/posts/926877516110207
Sickle Cell Disease impacts about 100,000 people in the US every year. It's an incredibly painful disease where the cells become misshapen like a crescent moon or a sickle. This shape causes the cells to get trapped and restrict the blood flood which causes chronic pain, organ damage, strokes, and shortened life expectancy. Bone marrow transplant has been the best treatment for patients, but it was very challenging to find a potential donor. Only 15% of siblings are a suitable match to be a donor and the chances of finding a match in the general population is about 10%. This means that only about 25% of patients suffering with Sickle Cell Disease had an option for treatment. Year Of The Opposite - Travis Stoliker's Substack is a reader-supported publication. To receive new posts and support my work, consider becoming a free or paid subscriber.The FDA has now approved two new treatments, Casgevy and Lyfgenia. “Both products are made from the patients' own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, a patients' own stem cells are collected, and then the patient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in Casgevy and Lyfgenia. Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product's safety and effectiveness.” (Link to FDA release)“bone marrow transplant was the first potential cure for sickle cell disease, but trying to find a good match for a transplant is a big barrier. This new technology uses gene therapy to allow patients to be their own match.” - Cece Calhoun, MD, MBA, a Yale Medicine hematologist-oncologist.This is a huge advancement and this same type of gene therapy could be offering hope for other diseases in the near future. But what about the cost? Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million. Lyfgenia the competing genetic medicine is priced at $3.1 million.Those price points are hard to imagine. But let's put some context around them.. Lets compare the cost of the new treatments versus the current standard of care. Insurance companies and the industry estimates the cost of managing sickle cell over a lifetime for someone with recurrent pain is between $4 - $6 million. So $4-6 million for the current treatment vs $2-$3million for the new treatment. How much did it cost to develop Casgevy? That has been a bit hard for me to track down exactly. Vertex (the maker of Casgevy) and CRISPR Therapeutics have an interesting development agreement where they split the cost and profits 60:40. Vertex made a $200,000,000 payment to CRISPR Therapeutics to cover some of the R&D expense as they crossed the milestone of getting FDA approval. However, this doesn't really represent the entire cost of creating Casgevy, because it relies upon the technology created with CRISPR itself. CRISPR alone has had more than $1billion in R&D expense in the past 3-4 years. So how much did it cost to develop Casgevy? It's hard to know exactly, but well into the billions of dollars for sure. So should Casgevy cost $2.2million per treatment? I don't know. But it's something we are going to have to think about as a society. On one hand, we want to be able to fund research and development that invents new technologies that could help hundreds of thousands of people every year. But we also don't want a health care system that is so large that the budget eats into the entire US economy. It's going to be interesting to see how this plays out over the coming years. This isn't the last drug that is going to cost millions of dollars to get. Thank you for reading Year Of The Opposite - Travis Stoliker's Substack. This post is public so feel free to share it. Get full access to Year Of The Opposite - Travis Stoliker's Substack at www.yearoftheopposite.com/subscribe
The first surge of IPO activity this year sees Alto Neuroscience, ArriVent Biopharma, CG Oncology, Kyverna Therapeutics and Metagenomi making announcements. Lori, Greg and Tyler also discuss gene therapy pricing including Vertex/CRISPR's Casgevy.
For the free course "ChatGPT4 for Medical Writers and Editors," go to learnAMAstyle.com Visit Nascentmc.com/podcast for detailed show notes and links. Erdafitinib for Urothelial Carcinoma: The FDA has fully approved erdafitinib (Balversa) for advanced or metastatic urothelial carcinoma with FGFR3 genetic alterations in adults. This follows its initial accelerated approval and is based on the phase 3 THOR trial results, showing improved survival rates and manageable side effects. Erdafitinib reduced death risk by 36% and had a lower treatment discontinuation rate compared to chemotherapy. TTFields in NSCLC: The FDA is reviewing a premarket approval application for tumor treating fields (TTFields) combined with standard therapies for platinum-resistant non–small cell lung cancer. TTFields, first approved in 2011, disrupt cancer cell division and showed enhanced survival in NSCLC patients when combined with immune checkpoint inhibitors or docetaxel, without increasing systemic toxicities. The FDA's decision is expected in the second half of 2024. AI-Powered Device to Detect Skin Cancer: The FDA has approved the first AI-powered handheld device by DermaSensor for assisting in skin cancer detection. It uses AI-driven spectroscopy for analyzing skin lesions and is based on a study involving over 1000 patients. While not a primary screening tool, it aids in detecting melanoma and other skin cancers, especially in patients over 40, and requires further validation testing. Casgevy for Transfusion-Dependent Beta Thalassemia: The FDA has approved Casgevy (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, for treating transfusion-dependent beta-thalassemia. This follows its approval for sickle cell disease and marks the first CRISPR gene-editing technology application for this condition. The approval came ahead of the anticipated date and follows Bluebird Bio's 2022 approval for a similar gene therapy. HyQvia for CIDP: HyQvia, an immune globulin infusion 10%, has been approved by the FDA for chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. Initially approved for primary immunodeficiency, HyQvia is the only product combining immunoglobulin with hyaluronidase, allowing for monthly subcutaneous infusions. The approval is based on its demonstrated efficacy in preventing neuromuscular disability relapse. Physicians' Understanding of FDA Approval Process: A national survey reveals that many physicians have limited understanding of the FDA's drug and medical device approval processes. Only 41% of the surveyed physicians reported moderate or better comprehension of the drug approval process. Despite believing in the adequacy of FDA standards, there's a call for more rigorous post-marketing studies and enhanced education on FDA processes to avoid misconceptions and inaccurate patient advice.
Gene therapy approved for beta thalassemia; treatment for preventing of chronic inflammatory demyelinating polyneuropathy relapse; device to aid dermatologist referrals; vertebral strength device for low-bone mass; Narcan shelf-life extension
Episode 2 of The Issue on the ASGCT podcast network is an in-depth interview with Dr Tom McCauley, CSO of Omega Therapeutics. Tom and Emily talk about his path from condensed matter physics to leading teams of scientists exploring the next generation of molecular therapeutics. They also dive into their mutual experience in the early days of in vivo gene editing and the challenges one faces with the complexities of these products, particularly before there is much of a roadmap for development. Finally, they dive into the recent public Ad Comm and ultimate approval of Casgevy and how we hope the industry continues to evolve and set standards as we move forward novel approaches. Music by: https://www.steven-obrien.net/--------------------------Bright New Morning - Steven O'Brien (Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.
Ron Robins Transcript & Links, Episode 121, January 12, 2024 Hello, Ron Robins here. So, welcome to this podcast episode 121 titled “The Most Profitable Clean Energy Stocks. Plus…” It's presented by Investing for the Soul. I do hope that you had a wonderful time over the holidays! Now investingforthesoul.com is your site for vital global ethical and sustainable investing mentoring, news, commentary, information, and resources. And look at my newly revised website at investingforthesoul.com! Tell me what you think. Now, remember that you can find a full transcript, and links to content – including stock symbols and bonus material – on this episode's podcast page located at investingforthesoul.com/podcasts. Also, a reminder. I do not evaluate any of the stocks or funds mentioned in these podcasts, nor do I receive any compensation from anyone covered in these podcasts. Furthermore, I will reveal to you any personal investments I have in the investments mentioned herein. Additionally, quotes about individual companies are brief. Please go to this podcast's webpage for links to the actual articles for more company and stock information. Also, some companies might be covered more than once and there are also 12 article links below that time didn't allow me to review them here. ------------------------------------------------------------- 1) The Most Profitable Clean Energy Stocks. Plus… Now in this edition, I'm featuring articles covering a broad range of sustainable industries. They include more articles in the renewable energy, tech, and health sectors. The first article I'm covering is titled 13 Most Profitable Renewable Energy Stocks by Ramish Cheema. It's seen on finance.yahoo.com. Here are some quotes. Please forgive me regarding the improper pronunciation of some of these companies! “To make our list of the most profitable renewable energy stocks, we ranked the top 30 holdings of BMO Global Asset Management's BMO Clean Energy Index ETF (ZCLN.TO) by their latest trailing twelve month net income and selected the top 13 stocks. For these stocks we have also mentioned hedge fund sentiment. 13. First Solar, Inc. (NASDAQ:FSLR) Latest Trailing Twelve Month Net Income: $473 million First Solar is an American company that sells products used in solar power generation systems. Unlike other renewable energy stocks, its shares are up 15.14% year to date. During Q3 2023, 49 out of the 910 hedge funds part of Insider Monkey's database had held a stake in First Solar, Inc. 12. Avangrid, Inc. (NYSE:AGR) Latest TTM Net Income: $536 million Avangrid is a diversified American utility that generates power from both traditional and renewable power sources. It hasn't been doing well financially as of late by having missed analyst EPS estimates in three out of its fourth latest quarters. By the end of September 2023, 19 out of the 910 hedge funds tracked by Insider Monkey were the firm's shareholders. 11. Enphase Energy, Inc. (NASDAQ:ENPH) Latest TTM Net Income: $571 million Enphase Energy makes and sells power systems that work with solar panels… By the end of this year's third quarter, 40 out of the 910 hedge funds profiled by Insider Monkey had held a stake in Enphase Energy. 10. Constellation Energy Corporation (NASDAQ:CEG) Latest TTM Net Income: $774 million Constellation Energy is an American renewable energy company that uses solar, nuclear, and other clean sources to generate power… During this year's third quarter, 45 out of the 910 hedge funds part of Insider Monkey's database had held a stake in the company. 9. Energias de Portugal, S.A. (ELI:EDP.LS) Latest TTM Net Income: $784 million Energias de Portugal, S.A. is a Portuguese diversified renewable energy company with a presence all over Europe and North America. It is one of the most strongly rated stocks on our list. 8. Corporación Acciona Energías Renovables, S.A. (BME:ANE.MC) Latest TTM Net Income: $847 million Corporación Acciona Energías Renovables, S.A. is a Spanish renewable energy firm headquartered in Alcobendas, Spain. It uses solar, hydro power, biomass, and other clean energy sources to generate power. 7. EDP Renováveis, S.A. (ELI:EDPR.LS) Latest TTM Net Income: $852 million EDP Renováveis, S.A. is another Spanish renewable energy company. The firm has thousands of megawatts of power generation capacity in the U.S., Brazil, Spain, and other countries. Despite a broader slowdown in the renewable energy industry, it has done well on the financial front. 6. Centrais Elétricas Brasileiras S.A. - Eletrobrás (NYSE:EBR) Latest TTM Net Income: $940 million Centrais Elétricas Brasileiras S.A. - Eletrobrás is a Brazilian renewable energy utility with dozens of dams and two nuclear power plants in its power generation portfolio… Insider Monkey dug through 910 hedge fund portfolios for their September quarter of 2023 shareholdings and found seven shareholders. 5. CPFL Energia S.A. (BVMF:CPFE3.SA) Latest TTM Net Income: $1 billion CPFL Energia S.A. is one of the oldest companies on our list since it was set up in 1912. Headquartered in Campinas, SP, Brazil, the firm provides electricity to all kinds of users in its home country… Its shares are rated Buy on average. 4. Companhia Energética de Minas Gerais – CEMIG (NYSE:CIG) Latest TTM Net Income: $1 billion Companhia Energética de Minas Gerais is a Brazilian utility headquartered in Belo Horizonte. It is one of the biggest energy companies in Brazil and accounts for more than ten percent of the country's power generation capacity. During Q3 2023, 15 out of the 910 hedge funds covered by Insider Monkey's research had bought and owned [the company]. 3. Brookfield Renewable Corporation (NYSE:BEPC) Latest TTM Net Income: $1.5 billion Brookfield Renewable Corporation is an American renewable energy utility with a presence in several countries and a power generation capacity of more than twelve thousand megawatts… the shares are rated Strong Buy. 2. Chubu Electric Power Company, Incorporated (OTC:CHUEF) Latest TTM Net Income: $2.73 billion Chubu Electric Power Company, Incorporated is a Japanese utility headquartered in Nagoya, Japan… the firm has been in business [since] 1889. Chubu Electric Power Company, Incorporated generates electricity through nuclear, hydroelectric, wind, and other clean sources. December 2023 has been a controversial month for the firm as a Japanese regulator is fining it for engaging in bribery. 1. China Yangtze Power Co., Ltd. (SHA:600900.SS) Latest TTM Net Income: $3 billion China Yangtze Power Co., Ltd. is a Chinese utility company with a presence in several Asian and South American countries. It is one of the biggest renewable energy companies in the world, known particularly for generating power through hydroelectric plants.” End quotes. ------------------------------------------------------------- 2) The Most Profitable Clean Energy Stocks. Plus… Next, we have this article. It's titled Green Giants: 7 ESG Stocks Leading the Way in Sustainable Investing by Muslim Farooque and found on investorplace.com. Here are some brief quotes by Mr. Farooque on each company. “1. Microsoft (NASDAQ:MSFT) The tech giant's early and substantial investments in generative AI have positioned it at the vanguard of the AI revolution… Simultaneously, Microsoft's dedication to ESG principles has earned it accolades as one of the top ESG stocks. 2. Ormat Technologies (NYSE:ORA) is a giant in the field of renewable geothermal energy technology… The company's achievements include constructing more than 190 power plants and installing more than 3,200 megawatts (MW) of capacity. Ormat stock has fallen out of favor with investors… However, the future looks promising. 3. Fluence Energy (NASDAQ:FLNC) is… bolstering the burgeoning electric vehicle (EV) market through its powerful energy storage solutions and AI-driven energy management systems. These competencies are critical for the development of efficient EV charging infrastructure and the integration of renewable energy into the power grid… This optimistic outlook underscores Fluence's potential as a key player in the energy storage and EV market, making it a compelling, sustainable option. 4. Bunge (NYSE:BG) Operating in agribusiness, refined & specialty oils, and milling, the company profits mainly from agribusiness. With a presence in 40 countries, Bunge is positioned remarkably well amidst growing global food shortage concerns, and its business model champions sustainable food supply chains by supporting agricultural communities efficiently… It offers a forward dividend yield of 2.61%, with a notable 22-year history of consistent dividend payments. 5. Altus Power (NYSE:AMPS) is a key player in the solar energy space, which is elevating its game with Atlus IQ, an AI-powered, cloud-based tool revolutionizing energy usage insights and solar savings. 6. NextEra Energy (NYSE:NEE) with its unique positioning as both a leading utility company and a solar and wind energy pioneer, presents a fascinating investment opportunity… Recent economic shifts, particularly high bond yields, have cast a shadow over utility firms and their dividends. However, this situation presented NextEra Energy as a strong contrarian choice, especially given its dividend yield consistently above the 2.5% mark… 7. Host Hotels & Resorts (NASDAQ:HST) It's a distinguished real estate investment trust (REIT), which continues to make strides in the luxury and upper-upscale hotel market. With an enviable portfolio boasting names such as Grand Hyatt, Hilton, Marriott, and others, the company has established itself as a heavyweight in the hospitality sphere. What sets this company apart, however, is its unwavering commitment to ESG principles… In addition to its ESG credentials, HST has demonstrated impressive financial performance… Additionally, its forward yield stands at an attractive 2.9%.” End quotes. ------------------------------------------------------------- 3 Healthcare Technology Stocks to Improve Lives in 2024 Now to the article on healthcare. It's titled 3 Healthcare Technology Stocks to Improve Lives in 2024. It's by Jeremy Flint and found also on investorplace.com. Here are some quotes by Mr. Flint on each of his picks. “1. Crispr Therapeutics (NASDAQ:CRSP) made waves earlier this month as its sickle cell treatment, Casgevy, became the first gene-editing therapy approved by the FDA. The implications… are staggering. FDA approval for an ailment as serious as sickle cell opens the floodgates for long-term gene editing and CRISPR tech treatments, ranging from complex chronic diseases to routine wound repair. Investors' lack of enthusiasm is primarily due to Casgevy's short-term operational and financial implications rather than what the approval stands for conceptually. 2. Teladoc Health (NYSE:TDOC) Teladoc… trades more than 90% below past highs. But that fall from grace isn't an indictment of Teladoc's model or viability… Teladoc's market share has lots of room to improve… Teladoc is neck-and-neck with direct competitors Amwell (NYSE:AMWL) and Doxy.me. As medical needs evolve, so do delivery and interaction mechanisms, making Teladoc one of the best healthcare technology stocks for 2024. 3. Intuitive Surgical (NASDAQ:ISRG) captures another long-term healthcare trend, increased reliance on robotics during surgical procedures. Intuitive Surgical… is a healthcare technology giant in the NASDAQ-100 and S&P 500. Despite its relative overvaluation, analysts are nearly all bullish on Intuitive Surgical… EquitySet, staking $409 as a fair price target, putting shares 18% undervalued at current levels… If you want to anchor a basket of healthcare technology stocks, a stable but innovative giant like Intuitive Surgical stands as a strong cornerstone.” ------------------------------------------------------------- Other Honorable Mentions – not in any order. 1) Title: Jefferies initiates coverage on solar energy stocks on yahoo.com. By Rad Smith and Nicholas Jacobino. 2) Title: 3 Renewable Energy Stocks to Buy Hand Over Fist Right Now on yahoo.com. By Travis Hoium. 3) Title: 5 ESG Funds That Rebounded in 2023 on morningstar.com. By Mahi Roy. 4) Title: 5 Best Solar Energy Stocks For 2024 on forbes.com. By Jason Kirsch. 5) Title: 3 Renewable Energy Growth Stocks to Buy Hand Over Fist Before 2024 on fool.com. By Matthew DiLallo, Tyler Crowe, and Jason Hall. 6) Title: Eclipsing Expectations: 3 Solar Stocks Offering Hidden Value for 2024 on investorplace.com. By Terel Miles. 7) Title: 12 Best Solar Power Stocks To Invest In According to Financial Media on finance.yahoo.com. By Usman Kabir. 8) Title: Alternative Energy Explorers: 3 Stocks Investing in a Greener Future investorplace.com. By Faisal Humayun. 9) Title: Top 10 renewable energy companies Energy Magazine on energydigital.com. By Maya Derrick. 10) Title: 3 ESG Stocks That Are Sustainable and Profitable on investorplace.com. By Steve Booyens. 11) Title: 3 Eco-Friendly Stocks Leading in Sustainable Supply Chains on investorplace.com. By Muslim Farooque. 12) Title: 8 Best Energy Stocks to Buy in 2024 on usnews.com. By Matt Whittaker. ------------------------------------------------------------- Ending Comment Well, these are my top news stories with their stock and fund tips -- for this podcast titled: The Most Profitable Clean Energy Stocks. Plus…” Now, please be sure to click the like and subscribe buttons on Apple Podcasts, Google Podcasts, or wherever you download or listen to this podcast. That helps bring these podcasts to others like you. And please click the share buttons to share this podcast with your friends and family. Let's promote ethical and sustainable investing as a force for hope and prosperity in these very troubled times! Contact me if you have any questions. Thank you for listening. And, again, please look at my new totally revised website at investingforthesoul.com. Tell me what you think! I'll talk to you next on January 26th. Bye for now. © 2023 Ron Robins, Investing for the Soul
Summary: Visit learnAMAstyle.com for the downloadable AMA Style cheat sheet Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast A summary of Eleven Potentially Practice-Changing Drugs Approved by the FDA in 2023 https://www.pharmacytimes.com/view/eleven-potentially-practice-changing-drugs-were-approved-by-the-fda-in-2023 Also added Casgevy and Lyfgenia for Sickle Cell Disease Podcast episode was from December 11, 2023. Happy new year! Intro and outro music Garden Of Love by Pk jazz Collective
In this episode of Health411, host Dr. Jonathan Karp and producers Daniel Geller and Josh Brewer delve into the groundbreaking world of Casgevy, the first FDA-approved CRISPR/Cas9-based treatment for sickle cell anemia. They explore the science behind this revolutionary gene-editing therapy, its potential implications for patients, and the ethical considerations surrounding genetic interventions in healthcare.
In this annual round-up episode, we welcome back Dr. Veera Rajagopal to cover some of the biggest stories in genetics and precision medicine from 2023! Veera is a scientist at Regeneron with an interest in human genetics and drug target discovery in neuroscience and psychiatry. He is a prolific writer as well, both on his substack, GWAS Stories, and on twitter, @doctorveera. Join us as Patrick and Veera cover the highlights of 2023, from Veera's personal achievements to the approval of Casgevy and understanding the impacts of studying rare variants for drug development!
In today's Real Physician Reacts episode, we delve into a groundbreaking development in the treatment of sickle cell disease – the FDA's approval of Casgevy, a revolutionary gene editing therapy. This significant advancement opens new doors for those who have long battled the challenges of sickle cell disease, offering hope and a potential paradigm shift in treatment. Please SUBSCRIBE for new videos every Monday afternoon and Thursday Evening https://www.drberrypierre.com/YTsubscription Let's Connect: Instagram TikTok Lunch and Learn Patreon Family Key Discussion Points: The Plight of Sickle Cell Patients: We'll explore the struggles faced by individuals with sickle cell disease and the historical context of their treatment. Gene Editing Therapy Revolution: Delving into the science behind gene editing therapy, we'll discuss how Casgevy represents a new frontier in medical treatment. FDA's Approval of Casgevy: Analyzing the implications of this approval, we'll examine what it means for the future of sickle cell disease management and for patients worldwide. This episode is an insightful journey into one of the most promising medical advancements of our time, signifying a major step forward in the fight against sickle cell disease. LINKS: FDA Approves 2 Sickle Cell Treatment
La tecnología CRISPR de edición genética ya es una realidad en una terapia que permite terminar con dos enfermedades potencialmente letales. Una puerta abierta a un cambio de paradigma en los fármacos. Lo analizamos con Cristina G. Lucio, periodista de la sección de Salud de EL MUNDO, y escuchamos la historia de Antonio, a quien podría salvar la vidaSee omnystudio.com/listener for privacy information.
Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. Glaukos, a medical technology company, has received FDA approval for its drug-releasing eye implant to treat glaucoma. This product is expected to revolutionize glaucoma treatment by addressing noncompliance with eye drops. Freenome, another medical technology company, has started a study of a blood test for lung cancer. The test could potentially serve as an alternative to CT imaging, which exposes patients to radiation. Tandem, a diabetes management platform, has completed the US launch of its revamped platform called Tandem Source. The platform combines the features of the company's legacy offerings with new data reports. In the wake of the Philips recall, two US senators are urging a review of FDA medical device oversight. The senators claim that Philips did nothing while patients suffered due to their sleep apnea devices. Medtronic has received approval for its pulsed field ablation (PFA) treatment for atrial fibrillation. PFA is considered a safer alternative to radiofrequency and cryoablation, and other companies like Boston Scientific and Johnson & Johnson are also pursuing the PFA market.The North Carolina Attorney General has sued HCA over degraded care quality at Mission Health. The lawsuit blames HCA for staffing and service shortages at the hospital.CMS has sent a letter to health plans and pharmacy benefit managers (PBMs) urging them to ease up on independent pharmacies. The letter asks payers to implement special payment arrangements with pharmacies before a new policy takes effect that could reduce upfront payments to pharmacies.CVS, Geisinger, and other healthcare organizations have signed a pledge committing to responsible use of artificial intelligence (AI) in healthcare.Nasdaq has ruled that Veradigm will not be delisted from the exchange as long as it complies with financial reporting requirements.Biogen receives positive recommendation from European regulators for its gene editing treatment Casgevy, while the clearance for Biogen's Skyclarys is uncertain.Novartis veteran Jay Bradner joins Amgen as its new chief scientific officer and head of R&D.The FDA expands the use of cancer drug Welireg to treat advanced kidney cancer.Fewer biotech companies are going public after a record run, but Fractyl Health becomes the first company in nearly a month to join the IPO queue.Moderna's cancer vaccine data leads to a share rally, with speculation that the company and partner Merck could seek approval before completing phase 3 testing.Patent protection will expire for many multibillion-dollar medicines this decade, prompting large pharma companies to find new products and markets.European regulators push for approval of CRISPR drugs.AstraZeneca and AbbVie are criticized by Elizabeth
In this episode of “Answers From the Lab,” host Bobbi Pritt, M.D., chair of the Department of Laboratory Medicine and Pathology at Mayo Clinic, is joined by William Morice II, M.D., Ph.D., CEO and president of Mayo Clinic Laboratories, to discuss the U.S. Food and Drug Administration's (FDA) recent approval of two gene therapies for sickle cell disease.Their discussion includes: The cause and characteristics of sickle cell disease. How the new cell-based gene therapies, Casgevy and Lyfgenia, work in the body. Bioethical and accessibility considerations of innovative treatments like gene-editing therapies. A brief update on the status of the FDA's proposed rule on laboratory-developed tests.
FDA-approved Casgevy & Lyfgenia made from patients' blood stem cells which are modified and given back as single-dose infusion. India ‘in preclinical stage' of trial of similar therapy.----more----https://theprint.in/health/genetic-scissors-india-using-same-tech-to-find-sickle-cell-cure-as-gene-therapy-approved-by-us-fda/1884468/
A daily news briefing from Catholic News Agency, powered by artificial intelligence. Ask your smart speaker to play “Catholic News,” or listen every morning wherever you get podcasts. www.catholicnewsagency.com - Under certain circumstances, it may be permissible for a Catholic to keep a small portion of a deceased loved one's ashes in a personal place of significance if some conditions are met, according to the Vatican's Dicastery for the Doctrine of the Faith. https://www.catholicnewsagency.com/news/256253/vatican-small-part-of-cremated-ashes-can-be-kept-in-personal-place-in-certain-cases Illinois has agreed to halt its enforcement of a law that restricts the speech and advertising of pro-life crisis pregnancy centers following a lawsuit that challenged its constitutionality on First Amendment and other grounds. https://www.catholicnewsagency.com/news/256256/illinois-agrees-to-halt-speech-restrictions-on-pro-life-pregnancy-centers-amid-lawsuit A new gene-editing therapy called Casgevy, which is designed to help treat patients suffering from sickle cell disease, has been endorsed by the National Catholic Bioethics Center and its president, Doctor Joseph Meaney. https://www.catholicnewsagency.com/news/256248/groundbreaking-gene-editing-therapy-receive-ethical-thumbs-up Today, the Church celebrates Saint Lucy, a third century consecrated virgin who was brutally tortured and martyred by the local governor. Owing to a miracle related to her eyes, she is the patroness of the blind, eye trouble, and other eye ailments. https://www.catholicnewsagency.com/saint/st-lucy-83
Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights: 1. Iptacopan (Fabhalta) for PNH: The FDA approved iptacopan (Fabhalta), the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH), a condition characterized by the destruction of red blood cells. This approval, based on the APPLY-PNH and APPOINT-PNH studies, marks a significant advancement over existing infusion-based treatments, with most patients experiencing increased hemoglobin levels without the need for blood transfusions. 2. Casgevy and Lyfgenia for Sickle Cell Disease: The FDA approved two gene therapies, Casgevy and Lyfgenia, for sickle cell disease treatment. Casgevy, using CRISPR/Cas9 technology for a one-time stem cell transplant, showed significant effectiveness in reducing severe vaso-occlusive crises in trials. Lyfgenia, employing lentiviral vector gene addition, demonstrated a high rate of resolution in severe vaso-occlusive events, marking a substantial progression in treating this condition. 3. SGT-003 for Duchenne Muscular Dystrophy: SGT-003, a gene therapy for Duchenne muscular dystrophy (DMD), received FDA fast track designation. Utilizing a novel capsid to deliver microdystrophin, this one-time intravenous therapy aims to address the underlying cause of DMD. It follows the accelerated approval of another gene therapy, Elevidys, also for DMD, highlighting advancements in the treatment of this muscular disorder. 4. New Fingerstick Blood Collection Device: The FDA has cleared a new fingerstick blood collection device, MiniDraw™, by Becton Dickinson. This less invasive method for obtaining blood samples aims to transform diagnostic testing by allowing lab-quality results for common blood tests without the need for traditional venous blood draws, offering a more patient-friendly alternative. See the full write ups for today's episode at nascentmc.com/podcast Intro and outro music Garden Of Love by Pk jazz Collective
Good morning from Pharma and Biotech Daily: the podcast focusing on essential news in the Pharma and Biotech world. Today, we'll cover multiple stories.Gilead Sciences aims to be a key oncology player while maintaining its focus on infectious diseases, particularly HIV. Its drug, Sunleca, approved in 2022 for treating multidrug-resistant HIV, is also being explored as a biannual preventive injection. Gilead is dedicated to tackling HIV's global health challenges.The FDA advises oncology drugmakers to establish optimal doses early in development. Astrazeneca is advancing in blood cancer care with novel scientific developments. Cell therapies are transforming healthcare, offering solutions to various diseases. Biopharma Dive explores the journey of the first CRISPR medicine, including its challenges and milestones. Moderna and Merck have begun a late-stage mRNA cancer therapy study. Experts at ASH respond cautiously to the approval of sickle cell gene therapies. There's also a call to increase RSV vaccination rates among older adults.The FDA has greenlit two gene therapies for sickle cell disease (SCD), including a pioneering CRISPR-based treatment. These mark a significant progress in SCD treatments, offering hope beyond bone marrow transplants. However, long-term safety and efficacy of gene-editing treatments remain under scrutiny. The FDA plans to continue evaluating these therapies' outcomes.Gene therapies are being researched for various genetic disorders, promising targeted treatments. Yet, these therapies require further study to ensure their safety and effectiveness.The FDA has approved a new CRISPR therapy, Casgevy, for SCD developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This marks a significant step in gene editing, though there are hurdles for its broad adoption.Merck faces challenges as trials involving Keytruda, a major revenue source, fail. Meanwhile, Spyre Therapeutics raised $180 million for its anti-inflammatory drug pipeline. Biotech venture firms experienced increased funding interest.In healthcare, the White House investigates private equity's role. The AHA and FAH oppose a bill affecting outpatient drug payments. Ardent Health Services recovers from a ransomware attack. Kroger introduces a benefits program for Medicare Advantage, Medicaid, and employer benefits recipients. A KLAS Research survey shows growing interest in generative AI among healthcare executives.Lastly, the FDA is poised to approve the first CRISPR gene editing treatment. This could be a milestone in gene editing, setting a precedent for treating genetic diseases. The therapy's launch will be crucial for its long-term success. Companies in gene therapy have learned from past experiences and are now better positioned to navigate the healthcare system.
Die Themen in den Wissensnachrichten: +++ Umwege mit dem Flugzeug könnten das Klima schützen +++ Riesen-Goldfische werden zum Problem im Ontariosee +++ Was Baby T-Rex so gegessen hat +++**********Weiterführende Quellen zu dieser Folge:Can we successfully avoid persistent contrails by small altitude adjustments of flights in the real world?, DLR, 07.12.2023Surface paving more important than species in determining the physiology, growth and cooling effects of urban trees, Landscape and Urban Planning, Dezember 2023Life outside the fishbowl: Tracking an introduced population of goldfish (Carassius auratus) in an embayment on the Laurentian Great Lakes, Journal of Great Lakes Research, 111.11.2023Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia, Vertex Pharmaceuticals, 16.11.2023Exceptionally preserved stomach contents of a young tyrannosaurid reveal an ontogenetic dietary shift in an iconic extinct predator, Science Advances, 08.12.2023Die Geminiden: Sternschnuppen in der Vorweihnachtszeit, Haus der Anstronomie, 09.12.2023**********Ihr könnt uns auch auf diesen Kanälen folgen: Tiktok und Instagram.
Proxy trojan targets macOS users for traffic redirection Indoor navigation has had a slow start Krasue RAT uses cross-kernel Linux rootkit to attack telecoms U.S. approves first gene-editing treatment, Casgevy, for sickle cell disease The DNS Deep-Drive continues with guests Josh Kuo, DNS expert, and Ross Gibson, Principal Solutions Architect of Infoblox, to talk about external authoritative DNS - whether enterprises should fully manage their own external DNS or use managed services, threats like domain hijacking, using load balancers, and more. Hosts: Curtis Franklin and Brian Chee Guests: Josh Kuo and Ross Gibson Download or subscribe to this show at https://twit.tv/shows/this-week-in-enterprise-tech. Get episodes ad-free with Club TWiT at https://twit.tv/clubtwit Sponsors: canary.tools/twit - use code: TWIT lookout.com vanta.com/ENTERPRISE
Proxy trojan targets macOS users for traffic redirection Indoor navigation has had a slow start Krasue RAT uses cross-kernel Linux rootkit to attack telecoms U.S. approves first gene-editing treatment, Casgevy, for sickle cell disease The DNS Deep-Drive continues with guests Josh Kuo, DNS expert, and Ross Gibson, Principal Solutions Architect of Infoblox, to talk about external authoritative DNS - whether enterprises should fully manage their own external DNS or use managed services, threats like domain hijacking, using load balancers, and more. Hosts: Curtis Franklin and Brian Chee Guests: Josh Kuo and Ross Gibson Download or subscribe to this show at https://twit.tv/shows/this-week-in-enterprise-tech. Get episodes ad-free with Club TWiT at https://twit.tv/clubtwit Sponsors: canary.tools/twit - use code: TWIT lookout.com vanta.com/ENTERPRISE
Proxy trojan targets macOS users for traffic redirection Indoor navigation has had a slow start Krasue RAT uses cross-kernel Linux rootkit to attack telecoms U.S. approves first gene-editing treatment, Casgevy, for sickle cell disease The DNS Deep-Drive continues with guests Josh Kuo, DNS expert, and Ross Gibson, Principal Solutions Architect of Infoblox, to talk about external authoritative DNS - whether enterprises should fully manage their own external DNS or use managed services, threats like domain hijacking, using load balancers, and more. Hosts: Curtis Franklin and Brian Chee Guests: Josh Kuo and Ross Gibson Download or subscribe to this show at https://twit.tv/shows/this-week-in-enterprise-tech. Get episodes ad-free with Club TWiT at https://twit.tv/clubtwit Sponsors: canary.tools/twit - use code: TWIT lookout.com vanta.com/ENTERPRISE
Proxy trojan targets macOS users for traffic redirection Indoor navigation has had a slow start Krasue RAT uses cross-kernel Linux rootkit to attack telecoms U.S. approves first gene-editing treatment, Casgevy, for sickle cell disease The DNS Deep-Drive continues with guests Josh Kuo, DNS expert, and Ross Gibson, Principal Solutions Architect of Infoblox, to talk about external authoritative DNS - whether enterprises should fully manage their own external DNS or use managed services, threats like domain hijacking, using load balancers, and more. Hosts: Curtis Franklin and Brian Chee Guests: Josh Kuo and Ross Gibson Download or subscribe to this show at https://twit.tv/shows/this-week-in-enterprise-tech. Get episodes ad-free with Club TWiT at https://twit.tv/clubtwit Sponsors: canary.tools/twit - use code: TWIT lookout.com vanta.com/ENTERPRISE
Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Black Box Biology, published by GeneSmith on November 29, 2023 on LessWrong. Suppose you want to decrease your risk of heart disease. The conventional advice goes something like this: Eat a healthier diet with less LDL-cholesterol raising foods Exercise more Keep your blood sugar under control Don't smoke, don't sit too much and don't take 400mg of methamphetamine on a regular basis An alternative strategy might be some kind of genetic intervention. For example, an active clinical trial by Verve Therapeutics aims to treat individuals with inherited high cholesterol by editing the PCSK9 gene. These trials almost always start the same: there's some rare disorder caused by a single gene. We have a strong mechanical understanding of how the gene causes the disorder. We use an animal model with an analogous disorder and show that by changing the gene we fix or at least ameliorate the condition. This is the traditional approach. And despite being slow and limited in scope, it occasionally produces results like Casgevy, a CRISPR-based treatment for sickle cell and beta thallasemia which was approved by the UK in mid-November. It might cost several million dollars. But it cures sickle cell! That has to count for something. Most diseases, however, are not like sickle cell or beta thalassemia. They are not caused by one gene. They are caused by the cumulative effects of thousands of genes plus environmental factors like diet and lifestyle. If we actually want to treat these disorders, we need to start thinking about biology (and genetic treatments) differently. Black Box Biology I think the conventional approach to genes and disorders is fundamentally stupid. In seeking absolute certainty about cause and effect, it limits itself to a tiny niche with limited importance. It's as if machine learning researchers decided that the best way to build a neural network was to hand tune model parameters based on their intricate knowledge of feature representations. You don't need to understand the mechanism of action. You don't need an animal model of disease. You just need a reasonable expectation that changing a genetic variant will have a positive impact on the thing you care about. And guess what? We already have all that information. We've been conducting genome-wide association studies for over a decade. A medium-sized research team can collect data from 180,000 diabetics and show you 237 different spots in the genome that affect diabetes risk with a certainty level of P < 5*10^-9! In expectation, editing all those variants could decrease someone's diabetes risk to negligible levels. I predict that in the next decade we are going to see a fundamental shift in the way scientists think about the relationship between genes and traits. The way treatments change outcomes is going to become a black box and everyone will be fine with it because it will actually work. We don't need to understand the mechanism of action. We don't need to understand the cellular pathway. We just need enough data to know that when we change this particular base pair from an A to a G, it will reduce diabetes risk by 0.3%. That's enough. Thanks for listening. To help us out with The Nonlinear Library or to learn more, please visit nonlinear.org
Link to original articleWelcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Black Box Biology, published by GeneSmith on November 29, 2023 on LessWrong. Suppose you want to decrease your risk of heart disease. The conventional advice goes something like this: Eat a healthier diet with less LDL-cholesterol raising foods Exercise more Keep your blood sugar under control Don't smoke, don't sit too much and don't take 400mg of methamphetamine on a regular basis An alternative strategy might be some kind of genetic intervention. For example, an active clinical trial by Verve Therapeutics aims to treat individuals with inherited high cholesterol by editing the PCSK9 gene. These trials almost always start the same: there's some rare disorder caused by a single gene. We have a strong mechanical understanding of how the gene causes the disorder. We use an animal model with an analogous disorder and show that by changing the gene we fix or at least ameliorate the condition. This is the traditional approach. And despite being slow and limited in scope, it occasionally produces results like Casgevy, a CRISPR-based treatment for sickle cell and beta thallasemia which was approved by the UK in mid-November. It might cost several million dollars. But it cures sickle cell! That has to count for something. Most diseases, however, are not like sickle cell or beta thalassemia. They are not caused by one gene. They are caused by the cumulative effects of thousands of genes plus environmental factors like diet and lifestyle. If we actually want to treat these disorders, we need to start thinking about biology (and genetic treatments) differently. Black Box Biology I think the conventional approach to genes and disorders is fundamentally stupid. In seeking absolute certainty about cause and effect, it limits itself to a tiny niche with limited importance. It's as if machine learning researchers decided that the best way to build a neural network was to hand tune model parameters based on their intricate knowledge of feature representations. You don't need to understand the mechanism of action. You don't need an animal model of disease. You just need a reasonable expectation that changing a genetic variant will have a positive impact on the thing you care about. And guess what? We already have all that information. We've been conducting genome-wide association studies for over a decade. A medium-sized research team can collect data from 180,000 diabetics and show you 237 different spots in the genome that affect diabetes risk with a certainty level of P < 5*10^-9! In expectation, editing all those variants could decrease someone's diabetes risk to negligible levels. I predict that in the next decade we are going to see a fundamental shift in the way scientists think about the relationship between genes and traits. The way treatments change outcomes is going to become a black box and everyone will be fine with it because it will actually work. We don't need to understand the mechanism of action. We don't need to understand the cellular pathway. We just need enough data to know that when we change this particular base pair from an A to a G, it will reduce diabetes risk by 0.3%. That's enough. Thanks for listening. To help us out with The Nonlinear Library or to learn more, please visit nonlinear.org
On the version of Hot off the Wire posted Nov. 24 at 6 a.m. CT: A new large study finds that the popular weight-loss drug Wegovy reduced the risk of serious heart problems by 20% in certain patients. The international trial showed the weekly injections prevented heart attacks, strokes and heart-related deaths. Wegovy is a high-dose version of the diabetes treatment Ozempic. The latest study tested Wegovy in people without the disease. It's being praised as a landmark study that could change the way doctors treat heart disease in some patients. The findings could also shift perceptions that a new class of obesity drugs are cosmetic treatments and put pressure on insurers to cover them. Federal regulators say a new version of the popular diabetes treatment Mounjaro can be sold as a weight-loss drug. The Food and Drug Administration approved drug named Zepbound on Wednesday. The drug has been shown to help dieters lose 40 to 60 pounds in testing. It is the latest diabetes drug approved for weight loss, joining Novo Nordisk's Wegovy, a high-dose version of Ozempic. Short supply and high costs have limited access to both medications. The FDA approved the new drug for people considered obese or for those who are overweight and have a weight-related health condition. A new report says progress against childhood cancers has stalled in recent years for Black and Hispanic youth. Thursday's report from the Centers for Disease Control and Prevention looked at cancer death rates for children and teens over two decades. The rates for white, Hispanic and Black children started about the same and all went down during the first decade. But then over the next 10 years, only the rate for white children dipped a little lower. Overall, the cancer death rate for children and teenagers declined 24% over the two decades, with the biggest drop in deaths from leukemia. NEW YORK (AP) — Syphilis cases in U.S. newborns again are on the rise. It has health officials calling for new measures to stop the increase, including encouraging millions of sexually active women of childbearing age and their partners to get tested. The Centers for Disease Control and Prevention says more than 3,700 babies were born with congenital syphilis in 2022. That's 10 times more than a decade ago and a 32% increase from 2021. CDC officials say there needs to be expanded testing. The agency also suggests starting syphilis treatment as soon as a pregnant woman tests positive. LONDON (AP) — Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling illness in the U.K. In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it had approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020. The medicine was approved for patients with sickle cell and thalassemia. Both diseases are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen. —The Associated Press About this program Host Terry Lipshetz is a senior producer for Lee Enterprises. Besides producing the daily Hot off the Wire news podcast, Terry conducts periodic interviews for this Behind the Headlines program, co-hosts the Streamed & Screened movies and television program and is the producer of Across the Sky, a podcast dedicated to weather and climate. Lee Enterprises produces many national, regional and sports podcasts. Learn more here.See omnystudio.com/listener for privacy information.
En entrevista con Pamela Cerdeira, para MVS Noticias, Carol Perelman, divulgadora de la ciencia, explicó, ¿se puede editar el ADN con precisión?See omnystudio.com/listener for privacy information.
The world's first approval of a CRISPR-based gene editing therapy, granted by the U.K.'s MHRA, was a milestone for the technology that was just discovered about 12 years ago. On the latest BioCentury This Week podcast, BioCentury's editors discuss the implications of the landmark and the challenges that still face partners Vertex Pharmaceuticals and CRISPR Therapeutics in launching and scaling the rollout of Casgevy exagamglogene autotemcel (exa-cel) in the U.K. and beyond. The editors also discuss the unintended consequences of the Inflation Reduction Act (IRA) on the development of orphan drugs and new legislation that aims to address the problem by providing an exemption to price negotiations for therapies that are approved only for orphan indications, regardless of how many. The team also reflects on the legacy of FDA's Janet Woodcock ahead of her planned retirement next year; the sentiment coming out of last week's Jefferies Healthcare Conference in London; and the aftermarket performance of two market debuts last week from antibody-drug conjugate CMO WuXi XDC Cayman and Mural Oncology.Music for the 24th Bio€quity Europe promo produced by:Thomas de Paula Eby, Andreas Unge, Epidemic Sound via Getty Images
The gene-editing therapy, called Casgevy, uses Crispr to prevent debilitating pain in patients with sickle cell disease. It also eliminates the need for regular blood transfusions in people with beta thalassemia. Read this story here.
The latest episode of the DDW Highlights podcast is now available to listen to below. DDW's Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates. The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field. You can listen below, or find The Drug Discovery World Podcast on Spotify, Google Play and Apple Podcasts.
Three news stories summarized & contextualized by analytic journalist Colin Wright.UK medicines regulator approves gene therapy for two blood disordersSummary: The UK's Medicines and Healthcare Products Regulatory Agency has approved a CRISPR-based treatment called Casgevy which edits the genes that cause two common blood disorders, sickle cell anemia and beta thalassemia, and which in clinical trials seemingly cured almost all the patients who participated.Context: This is the first CRISPR-based, faulty gene-editing treatment to be approved by a regulator, and that approval could impact the around 15,000 people in the UK who have sickle cell and the 1,000-ish people who have beta thalassemia; in clinical trials, 28 of the 29 sickle cell patients suffered no major pain episodes for at least a year after treatment, and 39 of the 42 participating beta thalassemia patients did not need red blood cell transfusions for at least a year following their treatment; this is still a wild west sort of therapy, so patients will be watched closely and everyone's on guard for potential, unexpected long-term consequences associated with editing genes in this way, but if the successes we've seen in clinical trials so far continue, it's likely this treatment will be approved in more jurisdictions in the near-future and that more treatments based on the same general principle, using CRISPR and CRISPR-like tools to edit patients' genes to address health issues, will be forthcoming.—The GuardianOne Sentence News is a reader-supported publication. To support my work, consider becoming a free or paid subscriber.US to resume food aid across Ethiopia next monthSummary: The US Agency for International Development has said that it will start sending food aid to Ethiopia again beginning in December, after halting such shipments in June due to their interception by, reportedly, regional governments and military groups.Context: This aid will be sent on a one-year, trial basis, now that the agency has implemented reforms meant to prevent the theft of goods meant for non-military civilians by the very groups causing much of their suffering; Ethiopia has been plagued by conflict in recent years, much of that conflict the result of a revolt by the TPLF against the central government, which led to widespread violence and starvation across the country—that conflict mostly ended in November of 2022, and the TPLF began disarming in early 2023.—ReutersFrance issues 'historic' arrest warrant for Syria's AssadSummary: In what's being called an historic move, the French government has issued an international arrest warrant for Syrian President Bashar al-Assad for his alleged complicity in crimes against humanity and war crimes.Context: Assad has been accused of using chemical weapons, including sarin gas, against his own people in 2013 when the ongoing Syrian civil war was just getting started; three other arrest warrants were also issued by the French government against Assad's brother, the head of Syria's elite military unit, and two Syrian generals; this theoretically puts heightened pressure on Assad, though it's unclear whether this will have any practical impact on him in the foreseeable future, as the principle this type of warrant is based on, universal jurisdiction, allows national governments to claim jurisdiction beyond their borders, but has never been successfully applied to the current leader of a sovereign nation.—Al-MonitorThis week's inflation data continues to be heralded as a cause for celebration across economic circles, and has triggered a huge surge in stock investment, capping a long slump in the same.—Axios2,786 megahertzSize of spectrum the Biden administration is studying to see if it should be reallocated for wireless broadband purposes.These bands show promise for use-cases ranging from next-generation satellite (space-to-space) and autonomous/unmanned vehicle communications, to more conventional utilities for defense-related or consumer-grade wireless broadband networks.The US government is taking its time, though, as previous reallocations of spectrum have led to interference issues and public battles between industries, like the ongoing conflict between the airline industry and wireless carriers over the deployment of 5G.—Ars TechnicaTrust Click Get full access to One Sentence News at onesentencenews.substack.com/subscribe
Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. ## The world's first CRISPR medicine, Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, has been approved in the UK for the treatment of sickle cell disease and beta thalassemia. However, a gene editing therapy like CRISPR may not be as simple as it seems. While Casgevy can mute the damaging symptoms of sickle cell disease, treatment may not be straightforward. Red Tree Venture Capital plans to expand into San Diego's biotech pipeline and build a competitor to Boston-area investors. The FDA has approved Augtyro, a new drug developed by Bristol Myers, for the treatment of lung cancer. Astellas has published a paper detailing a gene therapy study that led to patient deaths. The company is working with regulators to lift a clinical hold on the treatment.## The Biden administration has finalized a rule requiring nursing homes to disclose their ownership. CommonSpirit, a nonprofit operator, has started its 2024 fiscal year with a $738 million loss. CMS has proposed stricter network adequacy standards for plans sold in state-run ACA exchanges. Centene, a health insurer, has named Susan Smith as its new Chief Operating Executive. UnitedHealth is being sued for using an algorithm to deny care for Medicare Advantage members. There is an increasing trend of electronic health record (EHR) adoption, which has facilitated the rise of big data in healthcare.## The UK's Medicines and Healthcare Products Regulatory Agency (MHRA) has granted conditional marketing authorization for gene-edited therapy ExA-Cel, developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This marks the world's first approval for a CRISPR-edited therapy. Bristol Myers Squibb's (BMS) drug Augtyro has received FDA approval to treat ROS1-positive non-small cell lung cancer (NSCLC). Astellas sees a path forward for its gene therapy AT132 despite four patient deaths in a clinical trial.## The traditional approach to health marketing is becoming inadequate and less effective compared to biotech and pharmaceutical marketers' expectations. Quad offers next-generation solutions that aim to deliver superior results at a lower cost. The text mentions that Quad has a downloadable resource called "next-gen solutions for health marketing" that covers various topics. Quad also offers advertising opportunities to reach over 96,000 biopharma industry executives.## Influencer marketing is growing at a faster rate than traditional paid social media and is projected to reach $21.2 billion worldwide by 2023. Top retailers are taking note of this trend and using it to shape their strategies for the future. The trendline highlights several examples of how brands are leveraging influencer marketing.## Cognizant's Life Sciences Manufacturing Practice offers resources such as web content, whitepapers, videos, and thought leadership articles on their website. They specialize in creating connected GMP manufacturing and lab systems using digital technology adoption and support.## An infographic discusses the factors and macro-trends that different types of medical device companies need to consider when developing a location strategy. The infographic highlights several macro-trends that companies should take into account when developing their manufacturing strategies.## Finance and procurement professionals in the healthcare industry are encouraged to stay ahead by embracing change and utilizing automation. There is an e-book available that provides tools for success and offers a custom demo for transforming operations management in healthcare organizations.## Novo Nordisk plans to invest $6 billion in expanding its manufacturing capabilities in Denmark. Valneva has received regulatory approval from the FDA for its chikungunya vaccine. The American Medical Association (AMA) has called for broader health insurance coverage f
In today's episode, health authorities in the United Kingdom have approved a drug which aims to cure beta thalassaemia and sickle cell disease. The drug is called Casgevy and utilises a gene-editing tool as Crispr to manipulate the DNA of beta thalassaemia and sickle cell disease sufferers. Meanwhile, Parliament passed a new law on Thursday which sets out the rights and responsibilities of both employees who work remotely and their employers. Elsewhere, the forestry department in a written report has blamed the electricity authority (EAC) for the near catastrophic fires in Paphos forest, as well as that in Palaichori, this past summer. All this and more in the Cyprus Beat briefing brought to you by the Cyprus Mail.
© 2020-2025 Ivy Podcast Discovery LLC
