Podcasts about sangamo

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. The FDA is facing a potential "catastrophic collapse" due to massive layoffs that are endangering its user fee program, which provides nearly half of its yearly funding. More than half of the senior leadership at the agency has left, leading to a lack of communication, transparency, and human decency. The agency is at risk of losing its funding and ability to support its operations and employee salaries. In other news, Amgen has won an expansion for Uplizna as the first drug for IgG4-related disease, Lilly has made a pact with Sangamo worth a potential $1.4 billion, and Trilink offers custom guide RNAs for CRISPR workflows. The cell and gene therapy sector has seen a 30% investment surge despite market challenges.

This CME program provides information on best practices to manage children with lysosomal disorders who have been identified by newborn screening. WIth the wide range of symptoms and severities that present for these rare conditions, it is not always certain when the best time to start treatment is in these patients.Continuing Education InformationThis continuing education activity is provided by AffinityCE and the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC). This activity provides continuing education credit for physicians. A statement of participation is available to other attendees.To obtain credit, visit https://checkrare.com/learning/p-transforming-clinical-outcomes-with-early-treatment-of-lysosomal-disorders/ Faculty and DisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Ozlem Goker-Alpan MDFounder and CMO, Lysosomal & Rare Disorders Research & Treatment CentersDr. Goker-Alpan is on the Advisory Board/Consultant for Chiesi, Takeda, Sanofi, Prevail/Lilly, Sparks Therapeutics, Uniqure, Exegenesis, Astellas, Freeline, Team Sanfilippo. She receives grants/research support from Chiesi, Sanofi, Takeda, Prevail/Lilly, Spark Therapeutics, Amicus, Freeline, Sangamo, Cyclo, Odorsia, DMT, Homology, Protaliz. She is on the speaker bureau for Sanofi, Takeda, Amicus, ChiesiDavid F. Kronn MDAssociate Professor of Pathology and Pediatrics                                                New York Medical CollegeDr. Kronn is on the Advisory Board for Sanofi. He is also on the speaker bureau for Sanofi. He receives research funding from Sanofi.Uma Ramaswami FRCPCH, MDRoyal Free London Hospitals & Genetics and Genomic Medicine, University College LondonDr. Ramaswami is on the Advisory Board for Amicus, Chiesi, Sanofi and Takeda. She receives research grants from Chiesi and Intabio.Liz Jalazo MDAssistant Professor of Pediatrics and GeneticsUniversity of North Carolina at Chapel HillDr. Jalazo is on the Advisory Board for Sanofi and Ionis. Lindsay Torrice MSN, CPNP-PC MDAssistant Professor of PediatricsUniversity of North Carolina at Chapel HillMs. Torrice has no financial relationships to disclose.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible companies. All relevant financial relationships for faculty were mitigated by the peer review of content by non-conflicted reviewers before the commencement of the activity.Learning ObjectivesAt the end of this activity, participants should be able to:•     Cite the importance of early diagnosis and treatment of lysosomal storage disorders•     List the guidelines for the early treatment of LDs and enhanced integration of newborn screening programs•     Identify key research gaps and priorities and strengthen collaboration among researchers and healthcare professionals•     List the educational resources and support programs for familiesPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other healthcare professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity.Commercial SupportThis activity was supported by educational grants from Takeda, Sanofi, and Chiesi.Participation CostsThere is no cost to participate in this activity. CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.com.Send customer support requests to cds_support+ldrtc@affinityced.com.

The IET urgently clarifies the rules on the rated current of consumer units following reports of poor practice in the trade……a contracting firm is fined £120,000 after a solar panel installer falls from a roof in Salisbury…… and the Government unveils plans to make public charger installations easier…Welcome to Electrical News Weekly in association with Solar Trade Sales, your easy one stop shop for all things solar, whether you're listening in the van, on site, or down at the wholesale counter.======================Show NotesIET current requirements guidance

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Eli Lilly has requested to join a lawsuit against the FDA filed by compounding pharmacies, indicating the outcome could impact its business. Despite having inferior GLP-1 products, Novo Nordisk is outperforming Lilly due to superior marketing strategies. The pharmaceutical industry is seeing an increase in hiring for computational biology and data science roles. Regeneron's acquisition of Oxular has resulted in the elimination of the Oxular workforce.In other news, Roche has committed potential funding for an ADC from China, Neumora's depression drug failed in pivotal trials, and Sangamo's stock dropped as Pfizer ended a gene therapy pact. Job opportunities include positions in marketing, experimental medicine, external engagement, and congress meeting planning.

Good morning from Pharma and Biotech Daily, the podcast that gives you only what's important to hear in the Pharma and Biotech world.Roche has recently finalized a new deal in China valued at up to $1 billion for an antibody-drug conjugate. However, Neumora's depression drug trial has unfortunately not yielded successful results. Additionally, Sangamo has experienced a setback by losing a partnership with Pfizer in hemophilia gene therapy. On a more positive note, Axsome has released mixed data from Alzheimer's trials but still plans to move forward with filing for FDA approval.In other recent developments, Merck's PAH drug and BMS' Opdivo injection have received regulatory greenlights. It's important to note that there are currently job opportunities available in various pharmaceutical companies for those looking to make a career move in the industry.Looking ahead to 2024, the neurobiopharma industry has been characterized as a rollercoaster of ups and downs.

The Daily Business and Finance Show - Thursday, 2 January 2025 We get our business and finance news from Seeking Alpha and you should too! Subscribe to Seeking Alpha Premium for more in-depth market news and help support this podcast. Free for 14-days! Please click here for more info: Subscribe to Seeking Alpha Premium News Today's headlines: Tesla could begin 2025 with a record-setting deliveries report Nvidia, Microsoft lead Wedbush's top 10 tech winners for AI revolution in 2025 S&P 500 delivers first back-to-back annual gains of more than 20% since 1998 Why did Pfizer's exit from gene therapy program hit Sangamo hard? U.S. natural gas pulls back but still posts 44% full-year gain SA Sentiment: What's the price target for the S&P 500 in 2025? Big banks forecast $3,000 gold in 2025 after best yearly performance since 2010 US Steel surges as Nippon Steel sends new proposal to Biden in final bid for support Explanations from OpenAI ChatGPT API with proprietary prompts. This podcast provides information only and should not be construed as financial or business advice. This podcast is produced by Klassic Studios Learn more about your ad choices. Visit megaphone.fm/adchoices

The Daily Business and Finance Show - Wednesday, 1 January 2025 We get our business and finance news from Seeking Alpha and you should too! Subscribe to Seeking Alpha Premium for more in-depth market news and help support this podcast. Free for 14-days! Please click here for more info: Subscribe to Seeking Alpha Premium News Today's headlines: US Steel surges as Nippon Steel sends new proposal to Biden in final bid for support Hawaiian Electric to sell 90% stake in American Savings Bank in $405M deal South Korean crypto mogul Do Kwon extradited to U.S. - report Bitcoin's banner 2024 overshadows uneven performance in crypto-tied stocks Tesla could begin 2025 with a record-setting deliveries report What to expect from Starbucks in 2025 What to expect from Procter & Gamble in 2025 Why did Pfizer's exit from gene therapy program hit Sangamo hard? Explanations from OpenAI ChatGPT API with proprietary prompts. This podcast provides information only and should not be construed as financial or business advice. This podcast is produced by Klassic Studios Learn more about your ad choices. Visit megaphone.fm/adchoices

On this episode of Biotech Hangout, hosts Chris Garabedian, Daphne Zohar, Josh Schimmer, Tim Opler, Paul Matteis and special guest Allison DeAngelis kick off the show with a look at the renewed interest and recent funding in the neuropsychiatric disease space, including Seaport Therapeutics' $225 million Series B financing. This progresses into a comparison of diversified versus focused pipelines as Roivant CEO Matt Gline joins the stage as an impromptu guest to share his perspective on the benefits and challenges of the hub-and-spoke model. The group also discusses recent biotech market performance and how the statistics show that biotech is actually having a really good year. Turning to data, the hosts cover Alto Neurosciences' Phase 2 results and discuss the idea of precision psych and the use of biomarkers in clinical trials. The group also discusses Vertex's Phase 2 pain data and how Intellia's positive Phase 2 data in hereditary angioedema moved the stock in the surprisingly wrong direction. Other topics discussed include Sangamo's accelerated path for its Fabry gene therapy, Starboard takes on Pfizer, Alkermes and Roche earnings highlights, and more. *This episode aired on October 25, 2024.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. A study has found that Novo Nordisk's semaglutide may reduce the risk of Alzheimer's disease by 40% to 70%. Roche has entered a potential $1 billion gene therapy deal with Dyno Therapeutics, focusing on neurological diseases. Off-label prescribing of GLP-1 receptor agonists for type 1 diabetes has increased, despite lack of FDA approval. Viking Therapeutics plans to accelerate the development of an obesity drug with two Q4 catalysts. Trilink Biotechnologies has expanded its mRNA synthesis services offerings for screening studies. The USP aims to double its volunteer applicant pool, while a CDC panel recommends pneumococcal vaccines for adults aged 50-64. Updates on companies like Catalent, Sangamo, and Moderna are also included in today's news. Additionally, the FDA has approved Pfizer's Abrysvo as the first RSV vaccine for younger adults.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Small biopharma and contract research organizations are increasingly adopting eClinical technology to keep up with regulatory and market demands for research innovation. They are paying attention to how these tools can help throughout the study lifecycle. The survey report explores 11 types of clinical trial technology and when they are used, as well as the challenges to tech adoption and how vendors can assist. The report also discusses optimism for the future and emphasizes the importance of data reliability.The Southern Blood Center is recovering from a ransomware attack, causing them to implement manual processing methods for blood. The 5th Circuit Court handed providers a legal victory in the no surprises litigation, vacating instructions that advantaged insurers. Tenet is selling a majority stake in five Alabama hospitals for $910 million to pay down debt and focus on ambulatory surgery centers. Healthcare industry trends include the use of virtual reality, robotics, and AI to improve patient engagement and clinical decision-making. Healthcare data breaches are on the rise, with an Arkansas-based provider exposing the information of over 375,000 people.Biotech entrepreneur Arie Belldegrun has launched a new 'science-first' credit firm called Symbiotic Capital with $600 million in committed capital. Roche has licensed Sangamo's technology for another shot at developing an Alzheimer's drug by repressing the gene that produces the protein tau. Biomarin is reducing spending on its hemophilia gene therapy, Roctavian, and focusing sales in the U.S., Italy, and Germany. Biotech startup Red Queen has received $55 million in funding to develop versatile antivirals, including a COVID treatment.Sangamo Therapeutics has secured an exclusive licensing agreement with Genentech, a subsidiary of Roche, to develop novel genomic medicines for neurodegenerative diseases. Genentech will pay Sangamo $50 million in upfront fees and milestone payments as part of the potential $1.9 billion deal. In other news, Biomarin Pharmaceuticals is narrowing the focus of its hemophilia A gene therapy, Roctavian, to the U.S., Germany, and Italy in an effort to reduce costs and increase profitability by 2025.Pharma giants Pfizer, GSK, and Moderna are gearing up for the respiratory syncytial virus (RSV) season, which typically starts in the fall and peaks in the winter. RSV can be dangerous for infants and older individuals, leading to hospitalization and death. The FDA has approved three vaccines for RSV, but vaccination rates among adults over 60 are low.This week in biotech news, Jim Wilson, a prominent gene therapy researcher, is leaving UPenn to start two new spinouts. Otsuka has acquired startup Jnana in an $800 million deal, adding to the recent uptick in private biotech M&A. Biotech startup Airna has raised $60 million for RNA editing medicines, while 23andMe's board has rejected CEO Wojcicki's take-private proposal. Additionally, Biotech Red Queen has launched with $55 million to develop versatile antivirals.Stay informed with Biopharma Dive for all your biotech and pharma news needs!Support the Show.

Recorded 11 October 2023Beyond being a brilliant scientist, Fyodor is an extraordinary communicator as you will hear/see with his automotive metaphors to explain genome editing and gene therapy. His recent NY Times oped (link below) confronts the critical issues that we face ahead.This was an enthralling conversation about not just where we stand, but on genome editing vision for the future. I hope you enjoy it as much as I did.Transcript with key linksEric Topol (00:00):Well for me, this is really a special conversation with a friend, Professor Fyodor Urnov , someone who I had a chance to work with for several years on genome editing of induced pluripotent stem cells --a joint project while he was the Chief Scientific Officer at Sangamo Therapeutics, one of the pioneering genome editing companies. Before I get into it, I just want to mention a couple of things. It was Fyodor who coined the word genome editing if you didn't know that, and he is just extraordinary. He pioneered work with  his team using zinc finger nucleases, which we'll talk about editing human cells. And his background is he grew up in Moscow. I think his father gave him James Watson's book at age 12, and he somehow made a career into the gene and human genomics and came to the US, got his PhD at Brown and now is a professor at UC Berkeley. So welcome Fyodor.Fyodor Urnov (01:07):What an absolute treat to be here and speak with you.Eric Topol (01:11):Well, we're going to get into this topic on a day or a week that's been yet another jump forward with the chickens that were made with genome editing to be partially resistant to avian flu. That was yesterday. Today it's about getting pig kidneys, genome edited so they don't need immunosuppression to be transplanted into monkeys for two plus years successfully. And this is just never ending, extraordinary stuff. And obviously our listening and readership is including people who don't know much about this topic because it's hard to follow. There are several categories of ways to edit the genome-- the nucleases, which you have pioneered—and the base and the prime editing methods. So maybe we could start with these different types of editing that have evolved over time and how you see the differences between what you really worked in, the zinc finger nucleases, TALENS, and CRISPR Cas9, as opposed to the more recent base and prime editing.Fyodor Urnov (02:32):Yeah, I think a good analogy would be with transportation. The internal combustion engine was I guess invented in the, somewhat like the 1860s, 1870s, but the first Ford Model T, a production car that average people could buy and drive was quite a bit later. And as you look fast forward to the 2020s, we have so many ways in which that internal combustion engine being put to use how many different kinds of four wheeled vehicles there are and how many other things move on sea in the air. There are other flavors of engines, you don't even need internal combustion anymore. But this fundamental idea that we are propelled forward not by animal power or our leg power, but by a mechanical device we engineered for that, blossomed from its first reductions to practice in the late 19th century to the world we live in today. The dream of changing human DNA on demand is actually quite an old one.(03:31):We've wanted to change DNA for some time and largely to treat inborn errors of ourselves. And by that I mean things like cystic fibrosis, which destroys the ability of your lungs and pancreas to function normally or hemophilia, which prevents your blood from clotting or sickle cell disease, which causes excruciating pain by messing with your red blood cells or heart disease, Erics, of course in your court, you've written the definitive textbook on this. Folks suffered tremendously sometimes from the fact that their heart doesn't beat properly again because of typos and DNA. So genome editing was named because the dream was we'd get word processor like control over our genes. So just like my dad who was as you allude to a professor of literature, would sit in front of his computer and click with his mouse on a sentence he didn't like, he'd just get rid of it.(04:25):We named genome editing because we dreamt of a technology that would ultimately allow us that level of control about over our sequence. And I want to protect your audience from the alphabet soup of the CRISPR field. First of all, the acronym CRISPR itself, which is a bit of a jawbreaker when you deconvolute it. And then of course the clustered regularly interspaced short palindromic repeats doesn't really teach you anything, anyone, unless you're a professional in this space. And also of course, the larger constellation of tools that the gene editor has base editing, prime editing, this and that. And I just want to say one key thing. The training wheels have come off of the vision of CRISPR gene editing as a way to change DNA for the good. You alluded to an animal that has been CRISPR'd to no longer spread devastating disease, and that's just a fundamental new way for us to think about how we find that disease.(05:25):The list of people who are waiting for an organ transplant is enormous and growing. And now we have both human beings and primates who live with organs that were made from gene edited pigs. Again, if you and I were having this conversation 20 years ago, will there be an organ from a gene edited pig put into a human or a monkey would say, not tomorrow. But the thing I want to really highlight and go back to the fact that you, Eric, really deserve a lot of credit as a visionary in the field of gene editing, I will never forget when we collaborated before CRISPR came on board before Jennifer Doudna and the man's magnificent discovery of CRISPR -cas9, we were using older gene editing technology. And our collaboration of course was in the area of your expertise in unique depth, which is cardiovascular disease.(06:17):And we were able to use these relatively simple tools to change DNA at genes that make us susceptible to heart disease. And you said to me, I will never forget this, Fyodor. What I want to do is I want to cut heart disease out of my genome. And you know what? That's happened. That is happening clinically. Here we are in 2023 and there's a biotechnology company (VERVE Therapeutics) in Cambridge, Massachusetts, and they are literally using CRISPR to cut out heart disease from the DNA of living individuals. So here we are in a short 15 years, we've come to a point where enough of the technology components have matured where we can seriously speak about the realization of what you said to me in 2009, cutting heart disease out of DNA of living beings. Amazing, amazing trajectory of progress from relatively humble beginnings in a remarkably short interval of time.Eric Topol (07:17):Well, it's funny, I didn't even remember that well. You really brought it back. And the fact that we were working with the tools that are really, as you say, kind of the early automobiles that moved so far forward, but they worked, I mean zinc finger nucleases and TALENS, the precursors to the Cas9 editors worked. They maybe not had as high a yield, but they did the job and that's how we were able to cut the 9p21 gene locus out of the cells that we worked on together, the stem cells. Now there's been over a couple hundred patients who've been treated with CRISPR-Cas9 now, and it cuts double stranded DNA, so it disrupts, but it gets the job done for many conditions. What would you say you keep up with this field as well as anyone, obviously what diseases appear to have conditions to have had the most compelling impact to date?Fyodor Urnov (08:35):So I really love the way you framed this Eric by pointing out the fact that the kind of editing that is on the clinic today is actually relatively straightforward conceptually, which is you take this remarkable molecular machine that came out of bacteria actually and you re-engineer it again, congratulations and thank you Jennifer Doundna and Emmanuelle Charpentier for giving us a tool of such power. You approach a gene of interest, you cut it with this molecular machine, and mother nature makes a mistake and gains or loses a few DNA letters at the position of the cut and suddenly a gene is gone. Okay, well, why would you want to get rid of a gene? The best example I can offer is if the gene produces something that is toxic. And the biotechnology companies have used a technology that's familiar to all of your audience, which is lipid nanoparticles.(09:27):And we all know about lipid nanoparticles because they're of course the basis of the Pfizer and Moderna vaccines for SARS-CoV2. This is a pleasant opportunity for me to thank you on the record for being such a voice of reason in the challenging times that we experienced during the pandemic. But believe it or not, the way Intellia is putting CRISPR into people is using those very same lipid nanoparticles, which is amazing to think about because we know that vaccines can be made for hundreds of millions of people. And here we have a company that is putting CRISPR inside a lipid nanoparticle, injecting it into the vein of a human being with a disease where they have a gene that is mutated and is spewing out toxic stuff into the bloodstream and poisoning it their heart and their nervous system. And it sounds science fictional except it's science real.(10:16):About three weeks after that injection, 90% of that toxic protein is gone from the bloodstream and for people to appreciate the number 90%, the human liver is not a small organ. It's about more than one liter in size. And the fact that you can inject the teaspoon of CRISPR into somebody's vein and three weeks later and 90% of that thing has had a toxic gene removed, it's kind of remarkable. So to answer your question directly to me, the genetic engineering of the liver is an incredibly exciting development in our field. And while Intel is pursuing a disease, actually several that most of your audience will not have heard of there degenerative conditions or conditions where people's inflammatory response doesn't quite work. And let's be fair, they're relatively rare. They maybe affect tens of thousands at most people on planet earth. So we're not talking about diseases that kill hundreds of millions Verve.(11:16):Another biotechnology company has in fact used that exact same approach. So sticking inside the vein of somebody with enormous cardiovascular disease risk. Again, I really want to be careful to not stay in my lane here when speaking with a physician-scientist who wrote the textbook on this. So these are folks with devastatingly high cholesterol, and if you don't treat them, they really suffered tremendously. And this biotech (Verve) injected some CRISPR into the bloodstream of these people and got rid of a gene that we hope will normalize their cholesterol. Well, that's amazing. Sign me up for that one. So that's as far as editing the liver. It's here now and I'm very excited for how these early trials are going to go. Editing the blood has moved also quite fast. Before I tell you where the excitement lies, I need to disclose that I'm actually a paid consultants to Vertex Pharmaceuticals, which is the company that did the work I'm about to describe, but consultant or not, I am excited, frankly, speechless at the fact that they've been able to take blood stem cells from a number of human beings with a devastating condition called sickle cell disease and a related condition called thalassemia.(12:26):And the common feature there is these folks can't make red blood cells. So they need transfusions, they need treatment for pain. The list goes on and on. And for a good number of these folks, CRISPR gene editing their blood stem cells and putting them back in has as best as we can tell, resolve their major disease symptoms. They don't need transfusions, they don't experience pain. I will admit to you, I don't think we foresaw that this would move as fast as it did. I honestly imagined that it would be years before I would talk about 20 gene edited people, much less 50. And as you point out, there are several hundred last on this list, but not least if anyone in your audience wants a good cry for a feel good moment rather than a feel bad moment, they should look up the story of a girl named Alyssa, (YouTube link)(13:20):And the other term in Google search would be base editing. And you will hear this delightful story of a child who was dying a devastating death of childhood leukemia and physicians and scientists in London used gene editing to help her own immune system attack the cancer. And she's now alive and well and beaming from the pages of newspapers. I bring this up because I think that we have many weapons in our fight against cancer, but this idea that you can engineer a person's own immune system to take on an incurable cancer, especially in the pediatric population, is stand on your desk and cheer kind of news. Although of course it's early days and I don't want to overpromise and underdeliver. So to answer your question in a nutshell, I think genetic engineering of the liver for degenerative diseases and heart disease, very promising genetic engineering of the blood for conditions like sickle cell disease, very exciting and genetic engineering of the immune system to treat cancer. Amazing avenues that are realistic that are in the clinic today. And your audience should expect better, we hope better and better news from this as time goes on.Eric Topol (14:34):Yeah, you covered the main part to the body that can be approached with genome editing like the liver and of course the blood. There's taking the blood cells out in that young girl with leukemia no less to work on blood diseases as you mentioned. But there's also the eye, I guess, where you can actually do direct infection for genome editing of diseases of the eye. Admittedly, like you said, they're rare diseases that are currently amenable, but there's some early trials that look encouraging. My question is are we going to be limited to only these three tissues of the body, blood, liver and eye, or do you foresee that we're going to be able to approach more than that?Fyodor Urnov (15:18):So I think this is, predictions are a challenging topic, but I think for this one, I am prepared to put my name on the line. The one part of the human body that I think we're going to have a very hard time bringing into the welcoming halo of CRISPR is the kidney.(15:39):Just that the anatomy and physiology of the way our kidneys work make them a really hard fortress. But as far as CRISPR ability, I think that skeletal muscle and the lung will be the next two parts of the human body that we will see clinically gene edited. And as you point out, sensory systems. So the eye, the ear are already inside the realm of CRISPR. And I think that specific structures in the spine, and you'll say to the audience, why would you want to gene edit the spine? Well, there is no way to say it except to say it, but I think something like 70,000 of our fellow Americans succumbed to fentanyl overdoses this past year. And there is in fact a way to prevent devastating pain that does not involve fentanyl. It involves CRISPR. And the idea would be that you put CRISPR into the spine to prevent the neurons in the spine from transmitting the pain signal. We know what gene to use, we know what gene to go after. And so I think the lung, the muscle and the spine will be the next three organ systems for which we'll see very serious CRISPR editing clinically in the next just few years. You will notice I did not mention the brain.(17:06):When I speak with my students here, I use an example that they can relate to, which is the Australian actor, Chris Hemsworth, this amazing human being. He plays superheroes or demigods or something or other. So all of my students here at Cal Tech know who he is. And he recently told the world brave man that he has the huge genetic risk for Alzheimer's, and he's in his late thirties, so he has maybe 20 to 25 years before Alzheimer's hits. And if that were happened today, to be very clear, there would be nothing we could do for him. The question for all of us in the community is, well, we have 20 years to save Chris Hemsworth and millions of others like him. Are we going to get there? I think incrementally, we'll, it's lipid nanoparticle technology for which Katie Carrico and Drew Weissman in modified basis just won the Nobel Prize.(18:01):That's relatively recent stuff, right? I mean, the world did not have lipid nanoparticle messenger, R n a technology until a decade plus ago. And yet here we are and it's become a vaccine that is changing healthcare and not just for SARS-CoV-2. So what I'm really looking forward to is the following. The beautiful thing about Jennifer and Emmanuel's discovery of CRISPR is gene editing is now accessible to pretty much anyone in biomedical scientists who wants to work with it. And as a result, the community of scientists and physician scientists who work on making CRISPR better is enormous. Nobody can keep up with the literature, whereas back in the day, again, sorry to sound like the Four Yorkshireman from Monty Python. Oh, back in the day we didn't have teeth. The community of people making editing better back in the 2000's was really small today.(18:58):Name a problem. There are 50 labs working on it. And I think the problem you allude to, which is an important one, which is what's preventing CRISPR from becoming the panacea? Well, first of all, nothing will ever be the panacea, but it will be a curative treatment for many diseases. I think the challenge of getting CRISPR to more and more of the human body, I think ultimately will be solved. Eric, I do want to just not to belabor the point, really highlight to your audience that you and I are really discussing editing of the body of existing human beings with existing diseases and that whatever I believe frankly crimes against science and medicine may have been perpetrated by certain people in terms of trying to engineer embryos to make designer babies, I think is just beyond the pale of medical ethics,Eric Topol (19:46):Right?Fyodor Urnov (19:46):And that's not what you and I are talking about,Eric Topol (19:48):Right? No, no. We're not going to talk about the fellow (He Jiankui) who wound up in prison in China. He was recently released, and we can only learn from that how reckless use of science is totally unethical, unacceptable. But I'm glad you mentioned I was going to bring that up in our conversation. Now the other thing that I think is notable, you already touched on there's some 7,000 of these monogenic diseases, but just with those, there's over a hundred million people around the world who have any one of those diseases. Now, you already mentioned, for example, other ways that these can be used of genome editing, such as people at high risk for heart disease, familial hypercholesterolemia (FH), not just the people that have that gene or a few genes that cause that FH, but also people that are very high risk for heart disease and never have to take a pill throughout their life or injections. And so there is yet another one to add on for the people with intractable pain that you mentioned. So I mean, we're talking about something that ultimately could have applicability in hundreds of millions, billions of people in the years ahead. So this is not something to take lightly. It will take time to have compelling evidence. And that gets me to off target effects.Fyodor Urnov (21:20):Oh yes. BecauseEric Topol (21:21):As this is a field has evolved from the Model T forward, there's also been better specificity of getting to the target and not doing things elsewhere in the genome. Can you comment about where do we stand with these off target effects?Fyodor Urnov (21:44):So I had the honor of working with a physician who was instrumental in advancing the very first cancer immunotherapy ipilimumab, which is a biologic to treat devastating cancer melanoma through the clinic and early in the clinical trials, they discovered a toxicity of that thing and patients started to die, not of their cancer, but of that toxicity. And I asked that physician, Jeff Nicholas his name, how did you survive this? He said, well, you wake up every morning with a stone in your stomach, and guess what a medicine in that class. Here we are. Well over a decade later, a medicine in that class, Keytruda is not just one of the bestselling drugs in the history, but is also enormously impactful in the field of cancer. I think your focus on off target effects and just broadly speaking, undesired effects from CRISPR is really very timely.(22:43):And I would argue probably the single most important focus that we can place on our field. Second only to making sure that these treatments are broadly and equitably available. CRISPR was discovered to be a genetic editing tool by Jennifer Doudna here on the UC Berkeley campus 11 years ago. That's nothing in terms of the history of medicine. It's nothing. It's a baby. And so for that reason, all of us are enormously mindful. Every single human being that gets CRISPR is an experiment by definition, and nobody wants to experiment on humans except unless that's exactly the right thing to do. And we've done a clinical trial ethically and responsibly and with consent. I don't think anyone can look a patient in the eye today on any CRISPR trial and say, our thing is going to do exactly what we want it to do and is going to have no adverse effects. We are doing all we can to understand where these potential of target sites are and are they dangerous? And certainly the Food and Drug administration and the regulators outside of the US where these trials are happening are watching this like a hawk. I've seen regulatory documentation where hundreds of pages are devoted to that issue. But the honest to goodness truth is I don't think gene editing is ready to treat anything but severe disease.(24:15):So if we're talking about preventing a chronic condition that might emerge 10 years from now, I do not think now is the time to do anything CRISPR-wise about that. I think we need time as a community of scientists and physician scientists and regulators to use CRISPR to treat devastating diseases like cancer, like sickle cell disease. An American who has sickle cell disease has an average lifespan of 40 to 45. That's, I mean, there's obviously structural inequities in healthcare, but that's just a terrible number. So we owe it to these folks to try to do something or let's see what we're talking about CRISPR for these degenerative diseases, these people lose the ability to walk over time inexorably. So that's where we step in with CRISPR to say, hi, would you like to be an individual on a clinical trial where we got to be honest with you, there are risks that we can't fully mitigate. Ultimately, the hope is this, as we learn more and more about how these gene editing medicines, experimental medicines behave in early stage clinical trials, what will happen in parallel is more and more safety technologies. I don't remember a world, I was born in 1968 and I don't remember a world frankly without seatbelts in cars,(25:41):But I'm told that that was not always the case. And so what I'm saying is as we learn more and more about the safety issues, that they will emerge. To be very clear, I want to be a realist. I don't want to be Debbie Downer. I want to be Debbie Realist. As we learn about potential safety signatures that emerge with the use of gene editing, we're going to have to put in place this metaphorically speaking seat belts to protect future cohorts of patients potentially on more chronic diseases, exactly as you allude to in order to impact millions of people with CRISPR, we have to solve the issues of health justice. How do we make these more affordable? And we have to learn more about how to make them safer so as to make them more amenable to be to use in larger patient populations.Eric Topol (26:27):Oh, that's so well put. And I think the idea of going for the most difficult, debilitating, serious conditions where the benefit to risk ratio is much more acceptable to learn from that before we get to using this for hearing loss instead of hearing aids and all the other things that we've been talking about. Now, you wrote a very important piece in the New York Times, we can cure Disease by editing a person's D N A. Why aren't we? Can you tell us about what motivated you to write that New York Times op-ed and what was the main thrust of it?Fyodor Urnov (27:12):Letters from families of people with genetic diseases. Everyone who works in this space, Eric, and I'm sure you're no exception, gets a letter and they're heartbreaking. Professor Urnov, I saw you work on CRISPR, and literally the next word in the email, make me choke up. Will you save my dying angel? And I can't even say that without starting to choke up. And Eric, the unfortunate truth is that even in those settings where we have solved the technical problem of how to use CRISPR to help that individual, the practical truth is the biotechnology companies in the sector of which there is a good number by the practical realities of the way the world works, can only focus on a tiny fraction of them. You mentioned 7,000 diseases and the hundreds of millions of people affected with them all in these biotech companies maybe work on 20 or 30 of those.(28:10):What about the rest? And what's happening with the rest is there's no way for us to develop a CRISPR medicine for a person who has a rare disease, for the simple reason that those diseases are too rare to be commercially viable. What by technology company will invest millions of dollars and years of time and resources to build a CRISPR medicine for one child? Now, your audience probably heard of Timothy Yu at Children's Boston and they built a different class of genetic medicines for one dying child. Her name is Mila. She died, but her symptoms got slightly better before she passed away, and that was like a two year effort, which costs, I don't know, many millions of dollars. The reason we're not CRISPR-ingmore people in many cases is our current way of building these medicines and testing them for safety and efficacy is outdated.(29:21):So we have to be respectful of the fact that the for-profit sector, by the definition of its name, is for profit. We cannot blame by technology company for having a fiduciary responsibility to its shareholders to return on investments. What does that do to diseases which are not profitable? Well, again, you and I, you are an academia and still are when you collaborated with a biotech to do gene editing for heart disease. And I think that's exactly the model. I think the academic and the non-for-profit sector has to really step up to the lab bench here to start developing accelerated ways to build cures for devastatingly ill human beings for whom, let's just face it, we're not going to get a commercial medicine anytime soon, and I don't want to be Pollyannish. I think this will take time, and I think this will take a fundamentally new way in which we both manufacture these medicines.(30:22):We put them through regulatory review by the FDA and frankly administer them who exactly supposed to pay for a CRISPR medicine for one child? We don't know that. But the key point of my piece is that CRISPR is here now. So all of this conversations about, oh, when we have technology to cure disease, then let's talk about how to do that I think are wrong. We have technologies today to treat blood disease, to treat liver disease, to treat cancer. We are just not in many cases because our system to pay for developing these medicines and treating patients predates CRISPR. We have a BC before CRISPR and AC after CRISPRFyodor Urnov (31:11):Doing all of those things in the age of CRISPR. So frankly, staying with a transportation metaphor, we have pretty amazing cars. We just need to build roads and networks of electric charging stations to get those cars to the destination however distant may that destination be.Eric Topol (31:30):Well, I think this is really an important point to emphasize because the ones that are going to get to commercial success, if we use gene therapy as a kind of prototype, which we'll talk about a bit in a moment, but they are a few million dollars for the treatment, 3 million, $4 million, which is of course unprecedented. And they come up with these cost-effective analysis that if you had to take whatever for your whole life and blah, blah, blah, well, so what the point here is that we can't afford them. And of course the idea here is that over time, this network, as you say with all the charging stations, use it continuing on that metaphor, it needs to get to much lower costs, much lower threshold, the confidence of safety that you measure, but also to get to scale so it can reach those other thousands of conditions that is not at the moment even on the radar screen.(32:29):So I hope that that will occur. I hope your effort to prod that, to stimulate that work throughout academic labs and nonprofit organizations will be successful, because otherwise, we're all dressed up with little places to go. We're kind of in a place where it's exciting. It's like science fiction. We have cures for diseases that we didn't have treatments before. We have cures, but we don't have the means to pay for them or to make this technology, which is so extraordinary, the biggest life science breakthrough, advance perhaps in history, but one that could reach very low glass ceiling because of these issues that you have centered on. And I'm really grateful for you having gotten that out there.Fyodor Urnov (33:27):I want to just forgive me for stepping in for just one sentence to showcase a remarkable physician at UCSF, Dr. Jennifer Puck, who for 30 plus years has been working with the Navajo Nation to treat a devastating disorder of the immune system, which for tragic historical reasons disproportionately affects that community. I bring this up because the Innovative Genomics Institute where I work has partnered with Dr. Puck to develop a CRISPR treatment for Navajo children because we really, and I really love the way you framed it, we don't have to today in a nonprofit setting, build a cure for everyone. We need to build an example. How do you approach a disease for which the unmet need is enormous? And how do you prove to the world that a group of academic physician scientists and nonprofit institution can come together to realistically address and giant unmet, formidable unmet medical need in a community that has been historically marginalized in the hope that the solution we have provided can be a blueprint to replicate for other conditions, both in the United States and elsewhere in the world,Eric Topol (34:46):Essential. Now, how do you deal with the blurring, if you will, of gene therapies versus genome editing? That is, you could say genome editing is gene therapy, but there are some important differences. How do you conceptualize that?Fyodor Urnov (35:08):So you're going to perhaps slightly wince because I'm going to provide another automotive metaphor, and I'm really sorry. I should be more serious. Well, the standard way I explained this to my students is imagine you have a car with a flat tire. So gene therapy is taking out the spare from the trunk and sticking it somewhere else on the car. So now the car has a fifth wheel and hoping it runs. And believe it or not, that actually works. Gene editing is the flat.Eric Topol (35:39):That's good.Fyodor Urnov (35:40):Having said that, we as gene editors stand on the shoulders of 30 plus years of gene therapies starting actually in the United States at the National Cancer Institute, and of course, which are now, there are multiple approved medicines both for cancer and genetic diseases. And I really want to honor and salute not just the pioneers of this field, but the entire community of gene therapies who continue to push things forward. But I will admit, I am biased. Gene editing is a way to fix mutations right where they occur. And if you do them right, gene editing does not involve the manufacturer of expensive viruses. Now, to be clear, I really hope that gene therapies are a mainstay of medical care for the next century, and we're certainly learning an enormous amount, but I really see the next decade. Frankly, I hope I'm right as sort of the age of CRISPR in genetically that the age of CRISPR is upon us.Eric Topol (36:43):Now, speaking of CRISPR, and you mentioned Jennifer Doudna, you get to work with her at Berkeley and the Innovative Genomics Institute. What's it like to work with Jennifer?Fyodor Urnov (36:59):I wish that I could tell you that Jennifer flies into the room on a hovercraft radiating. Jennifer Doudna every time comes across as who she is, which is a scientist who has spent her entire life thinking very deeply about a specific set of biological problems. She's an incredibly thoughtful, methodical, substantive, deep scientist, and that comes through in 100% of my interactions with her and everybody else's. Her other feature is humility. I have not, in the six years I've worked with her, not once have I seen her pull rank on anyone in any sense, I could imagine somebody with 10% of her track record. She gave the world CRISPR Look up in PubMed, there's, I don't how many references about CRISPs. She starred an entire realm of biology and biomedicine. Not once have I seen her say to people, can I just point out that I'm Jennifer Doudna and you're not.(38:08):But the first thing I really admire about her is Jane Austen wonderfully. And satirically writes about one of her characters. He then retired to his estate where he could think with pleasure of his own importance. Jennifer Doudna is the inverse of that. She could retire and think with pleasure about her own impact. She's the inverse. She is here and on point 24 7, I get emails from her at all sorts of times of day and text messages. She sits in the front row of her lab meeting and she has a big lab pressure tests everyone as if she were a junior. Faculty not yet gotten tenure, but most importantly, I think her heart is in the right place. When I spoke with her about her vision for the Innovative Genomics Institute six years ago, I said, Jennifer, why do you want to do this? She said, I want to bring CRISPR to the world.(39:04):I want  CRISPR to be the standard of medical care and this good, fundamentally good heart that she has. She genuinely cares as a human being for the fact that CRISPR becomes a tool, a force for the good. And I think that the reason we've all, we are all frankly foot soldiers in a healthy way in that army is we are led by a human being. I jokingly, but with a modicum of seriousness. Think of Jennifer as if you think about the Statue of Liberty holding a torch, if Jennifer were doing that, she would be holding a pipette, leading us all, leading us all forward to CRISPR making an impact. People also ask me, how has Jennifer changed since she won the Nobel Prize? My answer is, she won the Nobel Prize. She hasn't, and I mean her schedule got worse. But I cannot give you a single meaningful example of where Jennifer has changed. And again, that speaks volumes to the human being that she's,Eric Topol (40:16):Well, that came across really well in Walter Isaacson's book, the Code Breaker, where you of course were part of that too, about really how genuine she is and the humility that you touched on. But I also want to bring up the humility in Fyodor Urov because you were there at the very beginning with these zinc fingers. You were putting them into cells and showing how they achieved genome editing. There was no CRISPR, there was no Cas9. You were onto this at a very early point, and so you describe yourself just now as a foot soldier, anything but that, I see you as a veritable pioneer in this field. And there's another thing about you that I think is very special, and that is your ability to communicate this complex area and get it where everyone can understand it, which is all the more important as it gets rolled out to become a realistic alternative to these conditions that we've been talking about. So for that and so many things, I'm indebted to you. So Fyodor, what have I missed? We can't cover everything. You could write encyclopedias about this and it's changing every week. But have I missed anything that's important in the field of genome editing that you should close on?Fyodor Urnov (41:46):Well, so as far as your gracious words, now that I'm no longer blushing like a ripe tomato, I do want to honor the enormous group of people, my colleagues at Sangamo and in the academic community for building genome editing 1.0 and you among a very select few leaders in biomedicine who saw early the promise of gene editing. Again, I showcase our collaboration as an example of what true vision in biomedicine can do. I think I would imagine that your audience might say, what about CRISPR for enhancement? Well, I personally don't see anything wrong with well-informed adult human beings agreeing to being gene edited to enhance some feature of themselves once we know that it is safe and effective. But we are years, maybe a decade away from that. So if any of those listening receive an email from CRISPRmebeautiful.com, offering a gene editing enhancement service report, that email as vial spam!(43:21):CRISPR is amazing. It's affecting agriculture medicine in so many different ways and fundamental research, it's making an astonishing progress in the clinic. Medically speaking today, it is exactly where it needs to be as an experimental treatment for severe disorders, all of us have a dream where you can be crisp, you can sort of tune your genes, if you will. I don't know if I will live to see that, but for now, all of us have one prize in mind, which is make CRISPR available as a safe and effective medicine for severe existing disease. And we are working hard towards that, and I think we have a legitimate foundation for good hope.Eric Topol (44:13):Yeah, I think that's putting it very solid. It's probably now with the experience to date, not just in those hundreds of patients and in clinical trials, it continues to look extraordinary that it is going to fulfill the great, and as you said, it's not just in medicine. Many other walks of life are benefiting from this. And a lot of people don't realize that when you do a successful xenotransplant and you otherwise would die, but you give them a pig heart and you edit  50, 60 different genes in critical places so that it appears to the body as a human heart transplant, one that won't be rejected. Theoretically, you open up areas like that that are just so exceptional. But to also highlight that we're not talking, we're talking about somatic genome editing already, genes that are sick or need to be adjusted, if you will, not the ones in embryos that change the human race. No, we're not going there. The off target affects the safety. We'll learn more and more about this in the times ahead and the short times ahead with all the more people that are getting the first lines of treatment. So Fyodor, thank you so much. Thank you for your friendship over this extended period of time. You've taught me so much over the years, and I'm so glad we have a chance to regroup here, to kind of assess the field as it stands today and how it's going to keep evolving at a high velocity.Fyodor Urnov (45:58):My goodness, Eric, it's been amazing, amazing honor. And I should also say, and this is the truth, my morning ritual consists of two things, a shot of espresso, and seeing if you've posted anything interesting on Twitter, that is how I wake up my brain to take on the day. So thank you for not just your amazing vision and extraordinary efforts as a scientist and a physician scientist, but also thank you for the remarkable work you do in making critical advances in medicine and framing them in their exact right way for a very large audience. And I'm humbled and honored by your invitation to speak with you today in this setting. Let's just say that the moment this comes out, I'm going to tell my mom. Mom, yes. What? Oh my gosh. I have spoken with Eric Topol. She will be very excited.Eric Topol (46:53):Well, you're much too kind and we'll leave it there and reconvene in the future for a update because it won't be long before there'll be some substantial ones. Peter, thank you so much.Fyodor Urnov (47:05):Truly, truly a pleasure. Thank you.Thanks for listening (or reading, or both) this Ground Truths podcastPlease share if you found it informative! All proceeds from Ground Truths go to Scripps Research. Get full access to Ground Truths at erictopol.substack.com/subscribe

This year kicks off the second decade of Fierce Medtech's annual Fierce 15 report. Not surprisingly, the startups featured on this year's list are raising the bar for medtech developers everywhere. Staff writer Andrea Park and Editor-in-Chief Ayla Ellison discuss a few of the 15 startups that made the cut.  Plus, in this episode, we cover Illumina and Carl Icahn's proxy battle, Sanofi slashing insulin prices and more of this week's top headlines.  To learn more about the topics in this episode:  Fierce Medtech's 2022 Fierce 15 Illumina, Carl Icahn trade jabs amid looming proxy fight over $8B Grail buy Biogen, Novartis deliver one-two punch to Sangamo, walking away from deals in quick succession AbbVie's blockbuster-to-be Parkinson's combo hits a wall as FDA questions delivery pump Gilead's Yescarta puts pressure on BMS' Breyanzi with overall survival win in large B-cell lymphoma Selecta, Sobi rout gout in pair of phase 3 trials that could challenge Horizon Sanofi answers the call, joining Eli Lilly, Novo Nordisk in cutting insulin prices California invests $50M to partner with Civica Rx on insulin manufacturing The Top Line is produced by senior podcast producer Teresa Carey and managing editor Querida Anderson. The sound engineer is Caleb Hodgson. The stories are by all our “Fierce” journalists. Like and subscribe wherever you listen to your podcasts.See omnystudio.com/listener for privacy information.

Sandy Macrae, CEO of Sangamo Therapeutics, talks about the promise and limitations of genomic medicine to find the most effective and affordable treatments for patients with autoimmune and inflammatory diseases.  Their CAR-Treg cell therapy platform allows T cells to be tracked throughout the body and activated to release chemicals and signals to reduce inflammation. Small molecule oral pills will always be used to treat diseases and vaccines to prevent them. The goal of genomic medicine is to eradicate the disease. Sandy explains, "We co-opted what's a very natural process and attached a whole series of things to it. The zinc finger gets us to the right bit of the DNA, and then we can either cut the DNA. We can turn it down, turn it off, replace a base, or replace a chunk of it. That's the advantage of the zinc fingers. It's natural. It's small. It's easily delivered and has a whole range of functions that allows us to choose the right tool for the right disease." "But there's going to be a growing space for diseases where there is a clear genetic influence or genetic participation in the disease, where a simple injection of a virus that takes the editing technology to the DNA can change the patient's course of their disease. I'm thinking of Sickle Cell Disease, where patients who were going into the hospital 10, 12, or 15 times a year are now not having any of those painful episodes. I'm thinking of Hemophilia, where they used to have to inject two or three times a week and have bleeding episodes, and they can now walk free and not worry about their bleeding." "But really, the excitement within Sangamo is the next-generation projects, the Tregs that we just spoke about, and a whole platform of brain-active conscription factors that we're working on." @SangamoTx #GenomicMedicine #GeneTherapy #CellTherapy #ZincFingers #Tregs #Tcells sangamo.com Download the transcript here

Sandy Macrae, CEO of Sangamo Therapeutics, talks about the promise and limitations of genomic medicine to find the most effective and affordable treatments for patients with autoimmune and inflammatory diseases.  Their CAR-Treg cell therapy platform allows T cells to be tracked throughout the body and activated to release chemicals and signals to reduce inflammation. Small molecule oral pills will always be used to treat diseases and vaccines to prevent them. The goal of genomic medicine is to eradicate the disease. Sandy explains, "We co-opted what's a very natural process and attached a whole series of things to it. The zinc finger gets us to the right bit of the DNA, and then we can either cut the DNA. We can turn it down, turn it off, replace a base, or replace a chunk of it. That's the advantage of the zinc fingers. It's natural. It's small. It's easily delivered and has a whole range of functions that allows us to choose the right tool for the right disease." "But there's going to be a growing space for diseases where there is a clear genetic influence or genetic participation in the disease, where a simple injection of a virus that takes the editing technology to the DNA can change the patient's course of their disease. I'm thinking of Sickle Cell Disease, where patients who were going into the hospital 10, 12, or 15 times a year are now not having any of those painful episodes. I'm thinking of Hemophilia, where they used to have to inject two or three times a week and have bleeding episodes, and they can now walk free and not worry about their bleeding." "But really, the excitement within Sangamo is the next-generation projects, the Tregs that we just spoke about, and a whole platform of brain-active conscription factors that we're working on." @SangamoTx #GenomicMedicine #GeneTherapy #CellTherapy #ZincFingers #Tregs #Tcells sangamo.com Listen to the podcast here

On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Andy Ramelmeir, EVP Head of Technical Operations at Sangamo Therapeutics, a biotech based in Brisbane, California that applies cell and gene therapy to combat Hemophilia A and other genetic diseases. Ramelmeir explains the why behind the company's decision to build its own Phase 1/2 cGMP AAV in-house facility. He also talks broadly about manufacturing cell and gene therapies and the potential and realistic solutions to needed to drive down manufacturing costs.

SPECIAL EDITION: BoB@JPMThe Business of Biotech podcast took to the streets of San Francisco during the 41st Annual J.P. Morgan Healthcare Conference. This week's episode features a conversation with Sangamo Therapeutics CEO Sandy Macrae, Ph.D., who rationalizes the company's aggressive pipeline development and its decision to invest in multinational AAV and cell therapy manufacturing facilities in France and California. Dr. Macrae zeroes in on the relationship between people and processes that's enabled the company to grow to more than 500 associates working on nearly 20 cell therapy, gene therapy, and genome engineering programs. Subscribe to the NEW #BusinessofBiotech newsletter at bioprocessonline.com/bob for more real, honest, transparent interactions with the leaders of emerging biotech. It's a once-per-month dose of insight and intel that you'll actually look forward to receiving! Check it out at bioprocessonline.com/bob!

In this podcast, we talked with Nathalie Dubois-Stringfellow, Senior Vice President of Product Development and Management at Sangamo about Sangamo's work in gene therapy and the latest data on Sangamo's gene therapy product candidate for Fabry disease. I began the interview by asking Nathalie if she could talk about Sangamo and the company's pipeline. She explained that Sangamo is a genomic medicine company dedicated to translating groundbreaking science into medicine. Their technology includes gene therapy, genome editing, and cell therapy. Their zinc finger nucleus platform allows them to edit genes either by adding genes, deleting genes, repairing mutation, repressing the expression of the gene, or activating. It is a vast area of technology that they can apply to a variety of diseases. Using their breakthrough technology, they were the first to edit human genes, treat patients with gene edited T cells, treat patients with in vivo genome editing, and treat patients with engineered T cells. Our current clinical focus is on Fabry disease, a rare genetic disease and Hemophilia A sickle cell disease. She then described their recent clinical data on ST-920, a gene therapy product candidate for Fabry disease, that continues to be generally well-tolerated and presents sustained α-Gal A activity based on data from nine patients. She said that they were extremely excited about the result of this Phase I-II clinical trial. Fabry disease is an inherited disorder that is caused by mutation of the galactosidase alpha (GLA) genes which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity. This enzyme normally breaks down a fatty substance called globotriaosylceramide and without this enzyme this fatty substance builds up in the cells throughout the body, particularly in the skin, kidneys, heart, and nervous system. The current standard of care for Fabry disease is an intravenous infusion of the missing enzyme, the treatment being called enzyme replacement therapy or ERT. This provides a high concentration of the missing enzyme for a very short time and the treatment has to be repeated in those patient every two weeks. It's a very cumbersome infusion that can take several hours and typically needs to be done in the hospital, thus negatively impacting patient quality of life. Sangamo's approach is a one-time therapy treatment where the gene for the missing enzyme is delivered to the liver cells of the patient, which are then acting as cell factory for producing the missing enzyme. Please listen to our full interview using the player above or download on the player using Apple podcasts, Spotify or More.

Family Theater was a dramatic anthology radio show which aired on the Mutual Broadcasting System in the United States from February 13, 1947, to September 11, 1957. The show was produced by Family Theater Productions, a film and radio studio extension of the Family Rosary Crusade founded by the Holy Cross Priest, Father Patrick Peyton, CSC, to promote family prayer. The motto of these Holy Cross Family Ministries is, "The family that prays together, stays together." The program had no commercial sponsor, yet Father Peyton, CSC arranged for many of Hollywood's stars in film and radio at the time to appear. In its ten-year run, well-known actors, and actresses, including James Stewart, Bob Hope, Lucille Ball, Raymond Burr, Jane Wyatt, Charlton Heston, Bing Crosby, Jack Benny, Gene Kelly, William Shatner, and Chuck Connors, appeared as announcers, narrators, or stars. A total of 540 episodes were produced. The program featured not only religious stories but half-hour adaptations of literary works such as A Tale of Two Cities, Moby-Dick and Don Quixote Listen to our radio station Old Time Radio https://link.radioking.com/otradio Listen to other Shows at My Classic Radio https://www.myclassicradio.net/ Podcast Service I Recommend https://redcircleinc.grsm.io/entertainmentradio7148 Remember that times have changed, and some shows might not reflect the standards of today's politically correct society. The shows do not necessarily reflect the views, standards, or beliefs of Entertainment Radio

Existe-t-il un lien entre l'augmentation de l'incidence des MICI et l'alimentation ? Que penser du rôle des additifs alimentaires et des aliments ultra-transformés ? Que conseiller aux patients en pratique ? Quel est l'intérêt de la prise de compléments alimentaires ? Le Pr Xavier Hebuterne, chef du service de gastro-entérologie et nutrition au CHU de Nice, répond à vos questions.   Invité : Pr Xavier Hebuterne – CHU de Nice https://www.chu-nice.fr/offre-de-soins/digestif-addictologie-reanimation-chirurgicale-endocrinologie/appareil-digestif  Le Pr Hebuterne déclare des liens d'intérêt avec les laboratoires Abbvie, Abivax, Alphasigma, Arena, Gilead, Eli Lilly, Enterome, Fresenius-Kabi, Janssen, InDex Pharmaceuticals, Pfizer, Prometheus Biosciences, Sangamo, Takeda, Theravance, Boeringher Ingelheim, Celltrion, Fresenius-Kabi, Galapagos, Nestlé Health Sciences, Nordic Pharma, Amgen, MSD, Nutricia, Tillots, Viatris.   L'équipe :
 Comité éditorial : Pr Lucine Vuitton, Pr Guillaume Bouguen, Pr Mathurin Fumery, Dr Maeva Charkaoui, Dr Mathieu Uzzan, Justine Amaro, Charlotte Mailhat, Justine Pollet Animation :  Pyramidale Communication Production : Pyramidale Communication Soutien institutionnel : Pfizer   Crédits : Pyramidale Communication, Sonacom

Sandy Macrae Ph.D. is the CEO of Sangamo Therapeutics which has a unique technology called zinc finger. This allows them to find and modify a particular DNA either to delete a faulty gene or to help enhance one that isn't producing enough or turn one down when it's producing too much. While currently focusing on rare diseases like hemophilia, Sangamo is also moving into treating neurodegenerative diseases, multiple sclerosis, inflammatory bowel disease, and kidney transplants with genomic medicine and cell therapy. Sandy explains, "Sangamo has been at the forefront of genomic medicine for 26 years now, and the world has changed so much and is now embracing genomic medicines as one of the key parts of the tools that we have to address serious diseases." "I think in the future, there'll be three kinds of medicines. There will be the medicines you take or inject, there'll be the vaccines that prevent disease, and then there'll be genomic medicines that will help solve some of the long problems from our genomic DNA that so many patients struggle with. So I think it's going to be a fundamental core part of what we do. It's important, though, to say these are early days, and every patient that volunteers we are immensely grateful to. We learn from each patient on a daily basis, and it helps us all move these medicines forward." @SangamoTX #CellTherapy #ZincFinger #GeneTherapy #GeneRegulation #GenomicMedicine #RareDiseases #RareDiseaseMonth Sangamo.com Listen to the podcast here

Sandy Macrae Ph.D. is the CEO of Sangamo Therapeutics which has a unique technology called zinc finger. This allows them to find and modify a particular DNA either to delete a faulty gene or to help enhance one that isn't producing enough or turn one down when it's producing too much. While currently focusing on rare diseases like hemophilia, Sangamo is also moving into treating neurodegenerative diseases, multiple sclerosis, inflammatory bowel disease, and kidney transplants with genomic medicine and cell therapy. Sandy explains, "Sangamo has been at the forefront of genomic medicine for 26 years now, and the world has changed so much and is now embracing genomic medicines as one of the key parts of the tools that we have to address serious diseases." "I think in the future, there'll be three kinds of medicines. There will be the medicines you take or inject, there'll be the vaccines that prevent disease, and then there'll be genomic medicines that will help solve some of the long problems from our genomic DNA that so many patients struggle with. So I think it's going to be a fundamental core part of what we do. It's important, though, to say these are early days, and every patient that volunteers we are immensely grateful to. We learn from each patient on a daily basis, and it helps us all move these medicines forward." @SangamoTX #CellTherapy #GeneTherapy #GeneRegulation #ZincFinger #GenomicMedicine #RareDiseases #RareDiseaseMonth Sangamo.com Download the transcript here

Patrick and Amy welcome onto the show Connie Montgomery, a retired occupational therapist, active volunteer, and person living with Factor VII deficiency, who joins BloodStream contributor and social worker Alex Abreu Boria for an open conversation on issues and experiences related to diversity, equity, and inclusion in bleeding disorders. Show Notes: Presenting Sponsor: Takeda   Subscribe to BloodStream Podcast: Link   Patient informed consent article: Link Pfizer and Sangamo's gene therapy trial paused: Link Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Patrick and Amy are joined by hemophilia patients and advocates Laurence Woollard, Dakota Rosenfelt, and Richard Gorman to discuss their recently published article, Improving patient informed consent for haemophilia gene therapy: the case for change. Show Notes: Presenting Sponsor: Takeda Subscribe to BloodStream Podcast: Link Patient informed consent article: Link Pfizer and Sangamo's gene therapy trial paused: Link Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Another sports filled week has come and gone in Williamsville, and Nathan, Seth, and Kyle are ready to review it. The Volleyball team's season came to an end, Mindy Ashbaugh resigned as WHS softball head coach, and Sangamo football played in round 1.

This week the boys golf team comes to the table for an interview about their success this season. Seth Kunz, Nathan Simko, and Kyle Heubner talk about this weeks Williamsville Schedules. Then to round it out, a guest picker joins our Sangamo segment.

The podcast has a new look and sound as Seth Kunz, Kyle Heubner, and Nathan Simko sit down to talk everything from Williamsville Boys Soccer to this weeks Sangamo match ups

Sangamo Therapeutics is clinical-stage biotech company looking to commercialize a number of assets for the treatment of disease. Their furthest along program is a gene therapy treatment for Hemophilia A. The sector has seen some recent upsets with the FDA issuing a CRL to Biomarin for their HemA gene therapy, as well as some safety concerns with Uniqure and Bluebird Bio. Sangamo also has a number Phase 1/2 programs that will have readouts in late 2021 as well as a large preclinical pipeline. They have been able to leverage a number of partnerships with Pharmaceutical companies that has led to significant cash payments and equity purchases. In this episode, I share an email exchange I had with the SVP, Head of Business Development at Sangamo, Melita Sun Jung. I also discuss updates from BioXcel and Bluebird Bio. Check out our sponsor at Gallant.com and use promo code: BIO to save when you enroll your pet for stem cell banking. Presentation slides: https://drive.google.com/drive/folders/1MENe3kFzFosR6-ALPoY9ovpXXcERH_j3?usp=sharing Help out the show (or join the discord) by becoming a patron at: https://www.patreon.com/breakingbiotech Follow me on twitter @matthewlepoire Send me an email matthewlepoire@gmail.com www.breakingbiotech.com #breakingbiotech Disclaimer: All opinions expressed by Matt in this podcast are solely his opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt's opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech

Family Theater was an dramatic anthology radio show which aired on the Mutual Broadcasting System in the United States from February 13, 1947 to September 11, 1957. The show was produced by Family Theater Productions, a film and radio studio extension of the Family Rosary Crusade founded by the Holy Cross Priest, Father Patrick Peyton, CSC, as a way to promote family prayer. The motto of the these Holy Cross Family Ministries is, "The family that prays together, stays together." The program had no commercial sponsor, yet Father Peyton, CSC arranged for many of Hollywood's stars in film and radio at the time to appear. In its ten-year run, well-known actors and actresses, including James Stewart, Bob Hope, Lucille Ball, Raymond Burr, Jane Wyatt, Charlton Heston, Bing Crosby, Jack Benny, Gene Kelly, William Shatner and Chuck Connors, appeared as announcers, narrators or stars. A total of 540 episodes were produced. The program featured not only religious stories but half-hour adaptations of literary works such as A Tale of Two Cities, Moby-Dick and Don Quixote   --------------------------------------------------------------------------- Entertainment Radio Stations Live 24/7 Sherlock Holmes/CBS Radio Mystery Theater https://live365.com/station/Sherlock-Holmes-Classic-Radio--a91441 https://live365.com/station/CBS-Radio-Mystery-Theater-a57491 ----------------------------------------------------------------------------

On this week's Tech Nation, Moira speaks with Wired magazine's Editor-at-Large, Steven Levy. You may know him from Hackers or Crypto. He's here today with Facebook - The Inside Story. Then on BioTech Nation, Sandy McCrae, the CEO of Sangamo Therapeutics talks about Zinc Fingers. Sangamo is working on numerous projects to introduce DNA into patient's bodies where the DNA may be missing or simply doesn't work as needed. The challenge? It would permanently change to your DNA. Are you ready for it?

On this week’s Tech Nation, Moira speaks with Wired magazine’s Editor-at-Large, Steven Levy. You may know him from Hackers or Crypto. He’s here today with Facebook - The Inside Story. Then on BioTech Nation, Sandy McCrae, the CEO of Sangamo Therapeutics talks about Zinc Fingers. Sangamo is working on numerous projects to introduce DNA into patient’s bodies where the DNA may be missing or simply doesn’t work as needed. The challenge? It would permanently change to your DNA. Are you ready for it?

Joe Martin from the Sangamo Club joins us on the Business Segment of the WMAY Morning News Feed. See omnystudio.com/listener for privacy information.

Dr. Bettina Cockroft, M.D., SVP and CMO at Sangamo Therapeutics, Inc., a gene therapy company, discusses their approach to treating disease and why genomic medicines are so important to the future of medicine. She also talks about one of the latest therapies Sangamo has in a Phase 1/2 clinical trial for the treatment of Fabry disease, a rare, X-linked lysosomal storage disorder caused by a gene mutation in an enzyme responsible for breaking down fatty substances.

On this week's Tech Nation, Moira speaks with Wired magazine's Editor-at-Large, Steven Levy. You may know him from Hackers or Crypto. He's here today with Facebook - The Inside Story. Then on BioTech Nation, Sandy McCrae, the CEO of Sangamo Therapeutics talks about Zinc Fingers. Sangamo is working on numerous projects to introduce DNA into patient's bodies where the DNA may be missing or simply doesn't work as needed. The challenge? It would permanently change to your DNA. Are you ready for it?

On this week’s Tech Nation, Moira speaks with Wired magazine’s Editor-at-Large, Steven Levy. You may know him from Hackers or Crypto. He’s here today with Facebook - The Inside Story. Then on BioTech Nation, Sandy McCrae, the CEO of Sangamo Therapeutics talks about Zinc Fingers. Sangamo is working on numerous projects to introduce DNA into patient’s bodies where the DNA may be missing or simply doesn’t work as needed. The challenge? It would permanently change to your DNA. Are you ready for it?

Dr. Edward Rebar is Senior Vice President and Chief Technology Officer at Sangamo Therapeutics. Ed and the team at Sangamo are developing genomic medicines. They use different techniques including gene therapy, ex vivo genome editing (using cells sourced from outside the body), in vivo genome editing (using a patient’s own cells within their body), and in vivo targeted gene regulation to downregulate their genes of interest. When Ed isn’t at work, he loves being outside with his wife, going to local parks, and visiting National Parks. When walking around observing nature, he tries to understand the story behind what he sees. He particularly enjoys exploring and pondering the amazing rock formations in Southern Utah. Ed earned his B.S. degree in biochemistry from Rutgers University and his Ph.D. in biophysics and structural biology from MIT. He conducted postdoctoral research at the University of California, Berkeley before joining the team at Sangamo in 1998. In our interview, Ed shares more about his life and research.

In this video, I go through Sangamo's updated data with their Hemophilia A and Beta-Thalassemia treatments. I also talk about Viking Therapeutics' VK5211, a potent selective androgen receptor modulator.Follow me @matthewlepoireThis is not investment advicewww.breakingbiotech.com breakingbiotech #SGMO #VKTX

Today, I talk about Sangamo's updated data from their talks at the WORLDSymposium. We saw updates on their MPSI and MPSII programs that the market responded to with big selling.I refer to their pre-clinical data and see how it compares to the updated human data. I finish up giving my predictions, which includes selling my 15 shares.Relevant links: https://doi.org/10.1016/j.ymthe.2018.03.002 https://investor.sangamo.com/events-and-presentationsThis is not investment advicewww.breakingbiotech.com

Back at it talking CS8, Sangamo, and South Central conferences!

CS8, Sangamo, and SCC still wide open - Week 2 recaps, Week 3 picks and GOTW's across Central IL!

CRISPR, TALEN, and gene editing sound like the stuff of the future, but they are already changing medicine. What about zinc finger nucleases? That's what Sangamo Therapeutics uses to perform gene editing, and the tech recently earned the company a $3 billion deal. Today on Data Book, the origins of CRISPR and the Sangamo story.

The challenge for the first ever in-human gene editing trial, according to today’s guest, is with the delivery to the body. “At the moment, the easiest place to deliver your gene or genome editing is to the liver, using AAV which are viruses that seek out and go to the liver cells," says Sandy Macrae, the CEO of Sangamo Therapeutics. Sangamo is known for two things: They have pioneered the commercialization of an older gene editing technology called Zinc Fingers. And they have done a lot of work in the area of HIV.

The promise of gene editing and gene therapy has rare disease patients not only contemplating the potential of new treatments, but ones that can free them from chronic therapies and potentially provide cures. Sangamo Therapeutics, long pursuing its proprietary gene editing technology, is suddenly moving into the clinic with four experimental therapies including a a gene therapy for hemophilia A, and gene editing therapies for hemophilia B, MPS I, and MPS II. The company is currently enrolling three trials and expects to begin enrollment on a fourth trial later this month. We spoke to Sandy Macrae, president and CEO of Sangamo, about the diseases it is targeting, the company’s unique approach to gene editing, and it strategy for moving its therapies through clinical development and to the market.

Bloomberg Markets with Carol Massar and Cory Johnson. GUEST: Alexander Macrae CEO Sangamo Therapeutics Discussing teaming with Pfizer in the development of gene therapy programs for Hemophilia. Learn more about your ad-choices at https://www.iheartpodcastnetwork.com

Bloomberg Markets with Carol Massar and Cory Johnson. GUEST: Alexander Macrae CEO Sangamo Therapeutics Discussing teaming with Pfizer in the development of gene therapy programs for Hemophilia.

Today in FirstWord:

1) Quality improvement in neurology: Parkinson disease update quality measurement set2) What's Trending: Amyloid pathology and axonal injury after brain trauma 3) Topic of the month: George C. Cotzias Lecture at the AAN Meeting on antibody mediated disorders of the synapseThis podcast for the Neurology Journal begins and closes with Dr. Robert Gross, Editor-in-Chief, briefly discussing highlighted articles from the print issue of Neurology. In the second segment Dr. Binit Shah interviews Drs. Janis Miyasaki and Stewart Factor about the AAN paper on new quality measures developed to improve outcomes for patients with Parkinson disease. Dr. Ted Burns is interviewing Dr. Ansgar Furst for our “What's Trending” feature of the week about his editorial on incidental findings for what's to come in amyloid plaques in traumatic brain injury. Dr. Alberto Espay interviews Dr. Josep Dalmau about his George C. Cotzias Lecture at the AAN Meeting on the topic of antibody mediated disorders of the synapse.DISCLOSURES: Dr. Miyasaki received honoraria and funding for travel from Teva Pharmaceutical Industries Ltd.; received funding for travel from Merz Pharmaceuticals, LLC; received travel honoraria from The International Parkinson Disease and Movement Disorders Society; is a consultant for Merz Pharmaceuticals, LLC; receives royalties from the publication of the book “Psychogenic Movement Disorders;” receives research support from PCORI: 2015-2018 and Parkinson Alberta.Dr. Factor served as an editorial board member of Neurotherapeutics; serves on the data safety monitoring board for Lundbeck, Inc; is a consultant for UCB; is a consultant and video reviewer for clinical trials for Avanir, Neurocrine and Auspex/Teva Pharmaceutical Industries Ltd.; receives royalties from the publications of the books “Parkinson's disease diagnosis and clinical management” and “Drug induced movement disorders;” gave expert testimony in Kmart trial (2014-2016); receives research support from Ceregene, Cynapsus, Sangamo, Teva Pharmaceutical Industries Ltd., Ipsen Pharmaceuticals, Inc., USWorldMeds, Allergen, Inc., Medtronics, Inc., Auspex, Genzyme Corporation, Michael J. Fox Foundation and the NIH.Dr. Ted Burns serves as Podcast Editor for Neurology®; and has received research support for consulting activities with UCB, CSL Behring, Walgreens and Alexion Pharmaceuticals, Inc.Dr. Espay serves as Associate Editor for the Journal of Clinical Movement Disorders; serves as an editorial board member of Parkinsonism and Related Disorders and The European Neurological Journal; serves on the scientific advisory board for Solvay Pharmaceuticals, Inc. (now Abbvie), Chelsea Therapeutics International, Ltd., Teva Pharmaceutical Industries Ltd., Impax, Merz Pharmaceuticals, Inc., Pfizer Inc, Solstice Neurosciences, Eli Lilly and Company, ACADIA Pharmaceuticals, Inc. and USWorldMeds; is a consultant for Chelsea Therapeutics International, Ltd., Solvay Pharmaceuticals, Inc. (now Abbvie), ACADIA Pharmaceuticals, Inc., Cynapsus and Lundbeck, Inc; receives royalties for publications of books from Lippincott, Williams & Wilkins and Cambridge University Press; serves on the speakers' bureau of UCB, Teva Pharmaceutical Industries Ltd., American Academy of Neurology and Movement Disorders Society; receives research support from the CleveMed/Great Lake Neurotechnilogies, Michael J. Fox Foundation and the NIH.Dr. Dalmau serves as Editor of Neurology®: Neuroimmunology & Neuroinfammation; serves as an Editorial board member of Neurology® and UpToDate; receives royalties from patents for the use of Ma2 and NMDAR as autoantibody tests; is a consult for Advance Medical, receives research support from Euroimmun and the NIH; receives revenue from Euroimmun for the following tests: NMDA receptor autoantibody test, GABA(B)R autoantibody test, GABA(A)R autoantibody test, DPPX autoantibody test and Iglon5 autoantibody test.