Podcasts about Gene therapy

This episode is brought to you by State & Liberty, Blokes & Joi, Fatty15 and FLYKITT. Bbiochemist and biomolecular therapy expert Jay Spall joins us to unravel his transformative journey from spinal injury to becoming a leading figure in the world of biohacking and longevity. Jay shares groundbreaking insights into follistatin therapy, detailing its impressive effects on endurance, body composition, and overall well-being. His personal narrative, combined with scientific research and anecdotes, paints a vivid picture of how cutting-edge therapies can enhance human performance and extend the quality of life. Follow MiniCircle @minicircledna Follow Chase @chase_chewning ----- In this episode we discuss... (00:01) Introduction to Biochemical Therapy (10:07) Reclaiming Our Health Through (17:30) Hormone Optimization Therapies (32:09) Modern Progressive Interventions (42:28) Taking Healthcare Power Back by Our Choices (54:49) Neurological Therapy (01:07:45) Gene Therapy for Health and Longevity (01:14:55) How to Actually Measure Longevity (01:22:14) Maximizing Healthspan and Lifespan With Gene Therapy (01:31:09) Why Community Matters (01:36:05) Connecting With MiniCircle ----- Episode resources: Save 15% on the best-fitting men's clothes with code CHASE at https://www.StateAndLiberty.com  Save 10% on any diagnostic labs with code CHASE at https://www.JoiAndBlokes.com  Save an additional 15% on the 90-day starter kit of C15:0 essential fatty acid with code EVERFORWARD at https://www.Fatty15.com/everforward  Never get jet lag again and save 15% with code CHASE at https://www.FLYKITT.com  Watch and subscribe on YouTube Learn more at MiniCircle.io 

A baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment. - 米国のKJ（ケイジェイ）・マルドゥーンちゃんは、CPS1欠損症という命にかかわる稀な遺伝性疾患を持って生まれてきました。緩和ケアを含む今後が話されていた折、最新技術を使った遺伝子編集（gene-editing）治療を行うことになり希望が見えてきました。

A baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment. He's one of the first to be successfully treated with a bespoke therapy targeting a tiny but deadly error in his DNA. His doctors hope the technology could one day help the estimated 350 million people worldwide with rare diseases.

Baby KJ Muldoon was born with a rare genetic condition that is often fatal, but doctors used custom CRISPR gene therapy to target the exact mutation in his DNA. His family shares their emotional journey in their first TV interview with CBS News. Singer Cassie Ventura faced intense cross-examination in Sean "Diddy" Combs' sex crimes trial, with defense attorneys asking her to read aloud past text messages. Jericka Duncan reports on the disturbing details.In this week's "Kindness 101," Steve Hartman and his children revisit the story of a teenage athlete who started giving back to his community after a small suggestion inspired a profound shift in his outlook on life.In honor of Mental Health Awareness Month, Amazon Books Editorial Director Sarah Gelman shares titles focused on self-care, emotional wellness and navigating complex relationships. To learn more about listener data and our privacy practices visit: https://www.audacyinc.com/privacy-policy Learn more about your ad choices. Visit https://podcastchoices.com/adchoices

Despite missing the primary endpoint, the treatment improved vision for some patients.

At-home cervical cancer screening device gains clearance; the FDA indicates it wants to remove pediatric fluoride products from the market; the chikungunya vaccine is put on-pause for some individuals; a subcutaneous autoinjector is approved to treat migraines; and the FDA will review a gene therapy for Hunter syndrome.

In this episode, Ayesha speaks with Norman Putzki, MD, Senior Vice President and Global Development Head of Neuroscience and Gene Therapy at Novartis, about the complexities and real-world challenges of bringing new therapies to patients with neurological diseases. Dr. Putzki discusses the challenges and opportunities in developing treatments for complex brain disorders, and how gene therapy is being applied in neurological disease. Dr. Putzki transitioned from an academic career in clinical neurology, with a focus on diseases like multiple sclerosis (MS), to the pharmaceutical industry in 2010. Since then, he has taken on leadership roles in both medical and development functions, first at Biogen and later at Novartis, where he currently leads global development efforts in neuroscience and gene therapy. During his tenure at Novartis, he has been leading teams around the globe to various new indications, approvals and launches within the neuroscience portfolio. Dr. Putzki received his PhD from Essen University in Germany and completed residency training in Internal Medicine and Neurology in Essen, University College London (Institute of Neurology Queen's Square) and University of Berne (Switzerland). Dr. Putzki is an author/co-author of more than 50 peer reviewed publications and has authored 4 books. He is a fellow of the American Academy of Neurology. Tune into the episode to learn more about the current landscape of drug development in neuroscience and neurological diseases — from research and strategy to execution on a global scale. For more life science and medical device content, visit the Xtalks Vitals homepage. https://xtalks.com/vitals/ Follow Us on Social Media Twitter: https://twitter.com/Xtalks Instagram: https://www.instagram.com/xtalks/ Facebook: https://www.facebook.com/Xtalks.Webinars/ LinkedIn: https://www.linkedin.com/company/xtalks-webconferences YouTube: https://www.youtube.com/c/XtalksWebinars/featured

Part B of the trial will enroll nine adult patients as well as three pediatric patients.

The company is currently enrolling patients in its Phase 2/3 VISTA Clinical Trial.

In this episode of the Pain and Performance Podcast, I sit down with Mac Davis, the founder of Mini Circle, a cutting-edge company revolutionizing gene therapy with mini-circle plasmids. If you've ever wondered how science is pushing the boundaries of longevity, muscle growth, and inflammation control; this conversation is a must-listen. "Health feels better than convenient."What Makes Mini-Circle Plasmids Different?Most gene therapy sounds like sci-fi, but Mini Circle's approach is different; safer, cleaner, and non-invasive to your DNA. Unlike traditional methods that can alter your genetic code, mini-circle plasmids work alongside your body's natural systems without changing your original blueprint.No DNA modification – Just temporary instructions that help your body heal and optimize itself.Delivered into fat tissue – Simple injections (no scary viral vectors).Focused on real-world results – From reducing chronic pain to boosting muscle growth.The Power of Folistatin: More Than Just MuscleFolistatin isn't some synthetic lab chemical; it's a natural human hormone that regulates muscle growth, fat metabolism, and even bone health. But its real superpower? Fighting chronic inflammation, one of the biggest hidden killers in modern health.Patients with stubborn joint pain and injuries report dramatic improvements after Folistatin therapy.It's not just for athletes; anyone dealing with age-related muscle loss or inflammation could benefit.Clotho: The "Magic" Longevity GeneIf you haven't heard of Clotho, pay attention. This gene therapy is showing insane potential for:Cognitive function – Patients describe "brain fog" lifting within weeks.Kidney health – A major factor in how well we age.Overall vitality – Some say they feel "like their younger selves" again.One of the most striking takeaways? A patient who said:"This is how I was before. This is me."Stacking Therapies for Maximum ImpactWhy stop at one therapy when you can combine them? Gene therapy stacking (like using Folistatin + Clotho) could be the next big leap in health optimization. Think of it like building a personalized longevity protocol—tailored to your body's needs.People in their 50s and 60s regaining muscle and energy levels they hadn't seen in decades.Individuals prioritizing quality of life over just adding years.As Mac puts it: "People deserve to be healthy."Links: DERRICKTikTok:https://www.tiktok.com/@drderrickInstagram:https://www.instagram.com/derrickbhines/Youtube:https://www.youtube.com/@DrDerrickMac Davis:Website: https://minicircle.io/ Instagram:https://www.instagram.com/minicircledna/

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.

We review grog, we took some breaks and never addressed them hence the weird cuts sometimes. We talk older video games, i make funny and gross jokes she does not appreciate. Its all fun and games.

Drug development activities focused on the rare, neuromuscular condition Duchenne muscular dystrophy have translated into functional improvements and an extension of life expectancy. Regenxbio is among several companies pursuing a gene therapy to treat Duchenne. Regenxbio believes both its microdystrophin—a truncated form of the dystrophin gene­ small enough to fit in a vector­—and the vector it is using, give it a competitive advantage over other efforts. We spoke Curran Simpson, CEO of Regenxbio, about the company's platform technology, the advantages he sees with its experimental DMD gene therapy, and how a partnership announced at the start of 2025 focused on a pair of other gene therapies provided a welcome alternative to tapping the capital markets.

In this episode Gene and his Girlfriend talk about so much stuff that i forgot it all and i think you should just listen to the podcast maaannn, check it out.

Jake Lesnik, Co-Founder and CEO of DM Therapeutics, joins EisnerAmper's TechTalk host Janina Teoxon to discuss how his gene therapy company developed a novel, non-viral platform designed to improve the efficacy, safety, and accessibility of gene therapies for inherited diseases. In this episode, discover how its non-viral Dark Matter DNA™ technology overcomes the limitations of viral vector-based gene therapies by providing long-lasting therapeutic effects and the capacity to deliver larger, more complex genes. Tune in to learn more about the company's mission to turn its breakthrough into new medicines and curative therapies that could expand the range of treatable inherited diseases.

Event Objectives:Describe the different types of gene therapy.Describe the different vectors used.Describe some of the FDA approved products in pediatrics.Claim CME Credit Here!

This week's expert, Hepatologist and Key Opinion Leader Scott Friedman, joins Roger to discuss advances in acceptance of gene therapy and knowledge in other areas of basic liver science. When discussing science, he pays particular attention to findings on the diversity of stellate cells and his interest in CAR-T as a therapy for liver disease.This conversation starts with Scott discussing gene therapy. Specifically, he applauds the idea that gene therapy is  becoming accepted in many diseases after a faulty start years ago, due to an unfortunate patient death in a badly controlled trial.  He comments that this acceptance has unique benefits in liver disease because the liver can regenerate so much faster and more efficiently than other organs. He mentions some of the rare liver diseases in which patients are benefiting from gene therapy, and notes that we now have gene therapies and early-stage trials to target PNPLA3 and  other genes associated with MASH and MASH cirrhosis. Next, Scott discusses stellate cells, which he has discussed in earlier episodes of SurfingMASH. Science is increasingly demonstrating how many different types of heterogeneous stellate cells exist. As Scott puts it, these cells "come in many flavors," each of which plays a different role in cell generation or cell death. In fact, the specific therapeutic challenges that present themselves may vary as a patient moves along the pathway from F1 to F2 to F3 to F4. Further, we are learning that there may be several different forms of MASH to present differently at a cellular level. This makes tremendous sense, given that no one drug has proven successful in even a significant majority of patients yet. As the conversation winds down, Scott shares what he describes as a "sobering note" about the state of research funding in America in 2025. As he notes, there are certain kinds of applied and developmental research that private companies do well, but other kinds of basic research that only occur when funded in public and not-for-profit sectors. As a specific example, he cites CRISPR, initially funded publicly and now in the hands of biotech companies, which is used to treat a variety of diseases more effectively than they could have been treated before, if at all. He also comments that a poor early commercial decision slowed the development of statins. 

In this episode of Tales From the Heart host Lisa Salberg speaks with Dr. Steve Ommen about HCM: Family Screenings, Genetics + Gene Therapy.   This conversation was recorded Apr. 18, 2025.

Are new solutions for pediatric hearing loss finally within earshot? In this episode of the Back Table ENT Podcast, we talk with Dr. Daniela Carvalho, pediatric otolaryngologist and director of the Cochlear Implant Program at Rady Children's Hospital in San Diego. --- SYNPOSIS Dr. Carvalho discusses the latest advancements and practices in the field of pediatric hearing loss, including newer indications for cochlear implants and innovative gene therapies. They delve into the genetic causes of congenital hearing loss, current screening practices, and various gene therapies being tested for hearing deficiencies, such as those involving the OTOF gene and its application through viral vectors. The conversation also touches on the potential for future treatments, including completely implantable cochlear devices and the role of vaccines in preventing CMV-related hearing loss. --- TIMESTAMPS 00:00 - Introduction 01:54 - Understanding Congenital Hearing Loss and Screening 05:41 - Challenges with Older Children and Hearing Loss 08:48 - Cochlear Implants: Indications and Advances 15:59 - Exploring Gene Therapy for Hearing Loss 23:54 - Challenges and Considerations in Gene Therapy 25:23 - Counseling Families on Hearing Loss 27:04 - Future of Genetic Therapy and Cochlear Implants 32:46 - Addressing Congenital CMV 40:59 - Final Thoughts and Future Research --- RESOURCES Dr. Daniela Carvalho https://www.rchsd.org/doctors/daniela-carvalho-md-mmm/

Mac Davis never planned on revolutionizing human health through gene therapy. A serial biotechnology entrepreneur, he founded Minicircle after developing a groundbreaking transient genetic enhancement platform. In this episode, Davis shares his journey from scientific researcher to innovative startup founder, discussing the potential of hormones like statin to optimize muscle health, reduce inflammation, and potentially slow aging. He explores the challenges of building a gene therapy company, early investor support from Sam Altman and Peter Thiel, and his vision of making personalized health interventions as accessible as a routine medical treatment.

Asthik Biswas, Spyros Batzios, and Kshitij Mankad expand on their recent letter to the editor to explain why ushering in the new era of gene therapy treatments requires not just clinical but also imaging readiness. Imaging readiness in the gene therapy era-exploring standardized protocols for response assessment Asthik Biswas, et al https://doi.org/10.1002/jimd.12828

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.##New vaccine shows promising results in clinical trialsA new vaccine for a common virus has shown promising results in clinical trials, with a high efficacy rate and minimal side effects. This development could potentially revolutionize the way we prevent and treat this virus in the future.##FDA approves groundbreaking gene therapy for rare genetic disorderThe FDA has approved a groundbreaking gene therapy for a rare genetic disorder, marking a significant milestone in the field of genetic medicine. This therapy has the potential to transform the lives of patients suffering from this debilitating condition.##Big pharma company announces major mergerA major pharmaceutical company has announced a merger with another big player in the industry, creating a powerhouse in the global pharmaceutical market. This merger is expected to have far-reaching implications for the industry as a whole.##Biotech startup secures record-breaking funding roundA biotech startup has secured a record-breaking funding round, signaling strong investor confidence in their innovative approach to drug development. This funding will allow the startup to advance their research and bring potentially life-saving treatments to market.##Researchers discover new mechanism of action for cancer drugResearchers have discovered a new mechanism of action for a commonly used cancer drug, shedding light on how it effectively targets cancer cells. This discovery could lead to the development of more targeted and effective cancer treatments in the future.##FDA issues warning about potential side effects of popular arthritis medicationThe FDA has issued a warning about potential serious side effects associated with a popular arthritis medication, urging patients to consult their healthcare providers before starting or continuing treatment. This warning highlights the importance of monitoring and reporting adverse reactions to medications.##Biopharmaceutical company announces breakthrough in Alzheimer's researchA biopharmaceutical company has announced a major breakthrough in Alzheimer's research, identifying a promising new drug candidate that could potentially slow or even reverse the progression of the disease. This development offers hope for millions of patients and their families affected by Alzheimer's.##Pharma giant partners with leading research institute to accelerate drug discoveryA pharmaceutical giant has partnered with a leading research institute to accelerate drug discovery efforts and bring innovative therapies to market faster. This collaboration aims to leverage the strengths of both organizations to address unmet medical needs and improve patient outcomes.##Health authorities approve new treatment for rare autoimmune diseaseHealth authorities have approved a new treatment for a rare autoimmune disease, providing much-needed hope for patients who have struggled to find effective therapies. This approval represents a significant milestone in the field of autoimmune medicine.##Biotech company receives grant to develop novel antibioticA biotech company has received a grant to develop a novel antibiotic targeting drug-resistant bacteria, addressing a critical need in the fight against antibiotic resistance. This funding will support the company's research efforts and help bring this potentially life-saving treatment to market.##Innovative technology platform revolutionizes drug deliveryAn innovative technology platform has revolutionized drug delivery, enabling more precise targeting of drugs to specific cells or tissues in the body. This technology has the potential to improve treatment outcomes and reduce side effects for patients across various therapeutic areas.#End of podcast episode

00:00:00 - Surf's Up: Season 6 Episode 5Host Roger Green briefly describes this episode's three sections and introduces Roundtable guests. The Roundtable panel shares groundbreakers. 00:10:39 - Roundtable: A Deep Dive Into Drug Development, Part OneThe opening portion of this month's roundtable centers around two issues: exciting data for FGF-21s and, more generally, treating patients with cirrhosis. Naim Alkhouri sets the tone in his opening comments, which start by focusing on the exciting SYMMETRY data from efruxifermin and then hones in on FGF-21s and resmetirom in cirrhosis. The rest of the conversation features Jörh Schattenberg, Sven Francque and Naim discussing therapies in development for compensated and decompensating cirrhosis.00;24:44 - Newsmaker: Naga Chalasani on Real-World Experience Prescribing ResmetiromNaga joins Roger to discuss the paper Early Experience with resmetirom to treat Metabolic Dysfunction-Associated Steatohepatitis With Fibrosis in a Real-World Setting from his group at Indiana University, which his group authored and Hepatology Communications recently posted. The paper, based on IU Health's experience with its first 113 resmetirom patients, shares the group's practical experience developing processes to work closely with the specialty pharmacies dispensing resmetirom and, finally, concludes that a more engaged patient management strategy might reduce drug discontinuation to a level comparable with clinical trials.  00:47:21 - Expert: Scott Friedman on Gene Therapy, Diversity of Stellate Cell Types, Other Basic Liver ScienceScott and Roger cover a range of basis science topics in a fast-moving 19-minute discussion. It starts with Scott discussing the increasing acceptance that gene therapy is an acceptable way to treat a range of liver diseases, many of which are orphan or ultra-orphan but, in fact, include potential gene therapies for non-cirrhotic MASH and MASH cirrhosis. He notes that in addition to classic gene therapy, which introduces protective gene variants into the systems of patients with the risky variants, gene therapy is now looking to introduce FGF-21 into patients through genetic modification. From there, the conversation covers CAR-T therapy, the increasing ability to identify many different types of stellate cells and the idea that the most effective therapy for eary fibrosis, advanced fibrosis and cirrhosis might require fundamentally different kinds of interventions. The two final elements are the idea that what we now call "MASH" may be several diseases with different etiologies with similar manifestations and a passionate call for all of us to support maintaining NIH funding in whatever ways we can.01:06:45 - Business ReportAs Roger copes with his laryngitis, AI voices deliver an abbreviated business report 

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Trump has signaled support for removing the IRA's 'pill penalty', with analysts cautiously optimistic about the executive order. Tariffs are in focus as Q1 earnings get underway, with EU and US pharmas making demands of the European Commission. Former FDA officials warn of potential implications of workforce cuts at the FDA. J&J sets the tariff tone as Q1 earnings begin to roll in. Experts offer advice on optimizing process development and validation steps for cell and gene therapies. In other news, bluebird's second suitor, Ayrmid, fails to make an offer, and the top 6 highest-paid pharma CEOs in 2024 are revealed.The top 6 highest paid pharma CEOs in 2024 have been revealed, with Johnson & Johnson's Joaquin Duato no longer holding the top spot. Duato has urged for a tax fix rather than tariffs to drive US pharma manufacturing. Viking Therapeutics saw a share rally after rival Pfizer discontinued an obesity candidate. Johnson & Johnson's Q1 earnings beat analyst estimates, thanks to Tremfya and Carvykti. Novartis has pledged a $23 billion boost to US operations amid tariff threats. Trump has signaled support for removing IRA's pill penalty and opened a national security probe on pharma imports. The industry is facing uncertainty due to ongoing tariff drama. Trilink's grna for gene editing has been successful, offering high-purity custom guide RNAs for research purposes. Trump's tariff pause sparked a late-day rally for pharma stocks. Some companies, like Glycomine, Merck, and Boehringer Ingelheim, have received significant funding or made deals in the biopharma sector.Senior Editor Annalee Armstrong encourages readers to suggest topics for future coverage in the biopharma industry.

In the final episode of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss the abstract she presented at the AAN Annual Meeting and details on the upcoming gene therapy trial.  Show reference: https://www.neurology.org/doi/10.1212/WNL.0000000000208987 

In part two of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss current treatment options for Leber hereditary optic neuropathy (LHON). Show reference:   https://index.mirasmart.com/AAN2025/PDFfiles/AAN2025-002206.html   

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD information, and to apply for credit, please visit us at PeerView.com/YKX865. CME/NCPD credit will be available until April 18, 2026.Innovation Takes Shape in SCD: Rewriting Management Principles With Gene Therapy and Revisiting the Role of HCT In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Vertex Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD information, and to apply for credit, please visit us at PeerView.com/YKX865. CME/NCPD credit will be available until April 18, 2026.Innovation Takes Shape in SCD: Rewriting Management Principles With Gene Therapy and Revisiting the Role of HCT In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Vertex Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD information, and to apply for credit, please visit us at PeerView.com/YKX865. CME/NCPD credit will be available until April 18, 2026.Innovation Takes Shape in SCD: Rewriting Management Principles With Gene Therapy and Revisiting the Role of HCT In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Vertex Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The pharmaceutical industry, traditionally seen as recession-resistant, may face challenges due to Trump's tariffs and the dominance of GLP-1s. Lexeo is moving forward with a gene therapy for Friedreich's ataxia after promising phase I/II data, while Rallybio is pivoting to a new antibody for rare autoimmune disorders. HHS Secretary Robert F. Kennedy, Jr. will not be speaking to the Senate health committee this week as scheduled. In other news, Merida is entering the autoimmune and allergy arena with a $121 million series A funding. Lilly is suing an Indianapolis spa for selling knockoff versions of its migraine drug Zepbound, and biotech shares are affected by the departure of FDA's Marks. Cardiovascular disease is back in the spotlight.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD information, and to apply for credit, please visit us at PeerView.com/YKX865. CME/NCPD credit will be available until April 18, 2026.Innovation Takes Shape in SCD: Rewriting Management Principles With Gene Therapy and Revisiting the Role of HCT In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Vertex Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD information, and to apply for credit, please visit us at PeerView.com/YKX865. CME/NCPD credit will be available until April 18, 2026.Innovation Takes Shape in SCD: Rewriting Management Principles With Gene Therapy and Revisiting the Role of HCT In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Vertex Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD information, and to apply for credit, please visit us at PeerView.com/YKX865. CME/NCPD credit will be available until April 18, 2026.Innovation Takes Shape in SCD: Rewriting Management Principles With Gene Therapy and Revisiting the Role of HCT In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Vertex Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD information, and to apply for credit, please visit us at PeerView.com/YKX865. CME/NCPD credit will be available until April 18, 2026.Innovation Takes Shape in SCD: Rewriting Management Principles With Gene Therapy and Revisiting the Role of HCT In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Vertex Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

Gene therapy is rapidly emerging as a transformative approach for treating genetic hearing loss, with Regeneron advancing one of the field's most promising candidates: DB-OTO. In this conversation, Regeneron's Auditory Global Program Head Dr. Jonathon Whitton provides an in-depth look at the company's work developing a gene therapy targeting otoferlin-related hearing loss, a rare but significant condition causing congenital deafness in children. Whitton explains the scientific rationale behind targeting the OTOF gene, the challenges of delivering gene therapy to the inner ear, and why this therapy holds particular promise for pediatric patients. He also discusses how Regeneron is designing clinical trials with long-term outcomes in mind, as well as the broader implications for treating other forms of genetic hearing loss. This discussion offers valuable insights into the future of hearing therapeutics and the role large biotech companies may play in making genetic treatments more accessible. The following Regeneron pages highlight the company's research on gene therapies for hearing loss and provide information on genetic testing and navigating a hearing loss diagnosis:https://aboutgenetichearingloss.comhttps://www.regeneron.com/stories/auditory-gene-therapyBe sure to subscribe to our channel for the latest episodes each week and follow This Week in Hearing on LinkedIn and X (formerly Twitter).- https://twitter.com/WeekinHearing- https://www.linkedin.com/company/this-week-in-hearing- https://hearinghealthmatters.org/thisweek/

In part two of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss current treatment options for Leber hereditary optic neuropathy (LHON). Show reference:   https://index.mirasmart.com/AAN2025/PDFfiles/AAN2025-002206.html  

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.##FDA Approves New Cancer DrugThe FDA has approved a new cancer drug that has shown promising results in clinical trials. This drug is expected to provide a much-needed treatment option for patients with this type of cancer.##Major Biotech Company Announces MergerA major biotech company has announced a merger with another company in the industry. This merger is expected to create a powerhouse in the biotech world, with the potential for groundbreaking research and development.##COVID-19 Vaccine UpdateThere have been new developments in the race for a COVID-19 vaccine. Several pharmaceutical companies are in the final stages of clinical trials, and there is hope that a vaccine will be available soon.##Drug Pricing Legislation PassesLegislation aimed at addressing drug pricing has passed in Congress. This legislation is expected to lower the cost of prescription drugs for consumers and improve access to affordable medication.##Biotech Startup Receives FundingA promising biotech startup has secured funding to further its research and development efforts. This funding will allow the company to continue its work on innovative therapies and treatments.##FDA Issues Warning About Opioid UseThe FDA has issued a warning about the dangers of opioid use. This warning comes amid a growing opioid crisis in the United States, and highlights the importance of safe prescribing practices.##New Gene Therapy Shows PromiseA new gene therapy has shown promise in treating a rare genetic disorder. This therapy has the potential to revolutionize treatment for patients with this condition, offering hope for improved outcomes.##Pharma Company Launches Patient Assistance ProgramA pharmaceutical company has launched a patient assistance program to help those in need access necessary medications. This program aims to provide support for patients facing financial barriers to treatment.##Biotech Breakthrough in Alzheimer's ResearchA biotech company has made a breakthrough in Alzheimer's research, uncovering new insights into the disease. This discovery could lead to new treatment options for Alzheimer's patients in the future.

This week we are joined by Jason Foster, CEO of Ori Biotech to talk about the future of CGT and why scalable strategic manufacturing is in a make or break moment, his experience scaling Indivior from 5 to 1,100 people culminating in a £3B exit and the powerful culture behind that growth, the Bluebird Bio acquisition and what it means for CGT investment today, and why he is deeply optimistic about Ori Biotech's automated, closed platform. A must-listen for anyone building, investing in, or scaling the next era of biotech.

Guest Whitney Fallon, NP shares how your body works on an Individual basis with your own genetic processes understanding Neuro divergence with ADHD and Autism. How or why, you can or can't lose weight, insulin resistance, the best detox or nutrient absorption. Whitney Fallon Links: https://linktr.ee/woodedlakewellness https://Wellnessrenovation.com Feel the reassurance of a Psychiatric Service Dog—Train your own dog if they recognize and reduce any psychological symptoms, to fly, be in 'no-pet' housing, with no pet fees and access public places under ADA law. Training from Joanne S. Williams, LCSW. A 30-second free guide to see if you qualify at ServiceDogPro.com!   Free 30 minute focus call with Joanne to talk about what would work best for you for your emotional health. Free Cultivate patience worksheet to make the world a more patient place.  

This week on The Genetics Podcast, Patrick is joined by Salvador Rico, Chief Medical Officer at Encoded Therapeutics. They discuss Salvador's journey into drug development, his work on gene therapy for X-linked myotubular myopathy, and fundamental challenges and exciting advances in the genetics field.Show Notes: 0:00 Intro to The Genetics Podcast00:59 Welcome to Salvador and how he became involved in drug development11:01 Frustrations and rewards of the genetics field13:59 Salvador's study on gene therapy for patients with X-linked myotubular myopathy (XLMTM)19:46 Risk of liver issues in gene therapy trials and attempts to mitigate them24:22 Encoded Therapeutics‘ approach to drug discovery and what motivated Salvador to join the team27:22 Steps towards therapeutic targeting of gene regulatory elements 30:04 Advantages of different methods for drug delivery 32:31 DNA- versus RNA-based therapy 34:56 Insights from approaches in other fields, including psychiatry36:35 Considerations for using natural history studies40:32 Expectations and goals for Encoded Therapeutics' current and upcoming studies43:17 Closing remarksFind out moreEncoded Therapeutics (https://encoded.com/)Please consider rating and reviewing us on your chosen podcast listening platform! https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link

In part one of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss how Leber hereditary optic neuropathy (LHON) typically presents and outline the steps for diagnosing LHON in a clinical setting. Show reference:  https://index.mirasmart.com/AAN2025/PDFfiles/AAN2025-002206.html 

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Former FDA Commissioner Robert Califf has spoken out as layoffs continue at the FDA, with the latest cuts affecting the Center for Drug Evaluation and Research (CDER). The layoffs are part of a larger reduction of 10,000 jobs at the Department of Health and Human Services. In addition, biopharma markets are in chaos following the departure of Peter Marks from the FDA's Center for Biologics Evaluation and Research (CBER).Carisma Therapeutics has laid off most of its staff and is considering selling off assets and winding down fully. Analysts are calling for President Donald Trump to reconsider his appointment of Robert F. Kennedy Jr. as Secretary of Health and Human Services. The departure of Peter Marks from the FDA has caused shares in the biopharma markets to tumble.Trilink offers custom guide RNAs (grRNAs) for CRISPR-based therapy discoveries. Despite market challenges, the cell and gene therapy sector has seen a 30% investment surge. Companies like Tenaya are cutting their workforce to fund development of gene therapies.Overall, the biopharma industry is facing uncertainty and instability as layoffs continue and key figures depart from regulatory agencies.

This week's episode of Trambles is brought to you by pollen. Tim putzes around while he waits to speak with KISS bassist and cofounder Gene Simmons.

HFA Symposium is this weekend, and we have CEO Dan Kelsey on the podcast to tell us all about it! Plus, a gene therapy segment with the fantastic Brendan Hayes about what she's hearing in the community about gene therapy for hemophilia B.    Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   Show Notes: Subscribe: The BloodStream Podcast   It's a Whole New World Gene Therapy Segment brought to you by CSL Behring, which now has a first-of-its-kind hemophilia B treatment. Visit BeyondHemB.com or download B SUPPORT wherever you get your apps for more information. Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Physician Tina Stankovic is an ear, nose, and throat specialist and a lover of music whose seemingly disparate pursuits — medicine and music — have led her to a groundbreaking career in hearing research. She recently worked with music legend Paul Simon during his well-publicized battle with hearing loss and he has become a vocal advocate for hearing research. New understandings and new approaches like regenerative medicine have put the once-impossible dream of hearing restoration within reach, Stankovic tells host Russ Altman on this episode of Stanford Engineering's The Future of Everything podcast.Have a question for Russ? Send it our way in writing or via voice memo, and it might be featured on an upcoming episode. Please introduce yourself, let us know where you're listening from, and share your question. You can send questions to thefutureofeverything@stanford.edu.Episode Reference Links:Stanford Profile: Konstantina M. StankovicStanford Researchers Assist Paul Simon with his Return to the StageInside the Stanford Initiative to Cure Hearing Loss: Cutting-Edge Science and InnovationConnect With Us:Episode Transcripts >>> The Future of Everything WebsiteConnect with Russ >>> Threads / Bluesky / MastodonConnect with School of Engineering >>> Twitter/X / Instagram / LinkedIn / FacebookChapters:(00:00:00) IntroductionRuss Altman introduces guest Tina Stankovic, a professor of otolaryngology and neurosurgery at Stanford University.(00:03:36) Why Hearing LossWhat inspired Tina's lifelong journey into hearing science.(00:04:17) Treating Hearing LossLimits of current options and lack of FDA-approved therapies.(00:05:23) Causes of Hearing LossThe two main categories of hearing loss: conductive vs. sensorineural.(00:07:47) Inside the Inner EarThe complexity of the inner ear, and why diagnosis is so difficult.(00:09:22) Tinnitus & Hearing LossWhy ear damage can cause phantom sounds in the brain.(00:10:28) Emerging Technologies in Hearing ResearchNew technologies that are evolving treatment approaches.(00:15:19) Recreating the Ear in the LabChallenges the inner ear's unique composition pose to researchers.(00:20:02) AI Applications in Hearing DiagnosisThe ways AI is transforming diagnosis and genetic analysis.(00:21:31) Can Ears Regrow?Why humans don't regenerate ear cells—but mice might help.(00:23:55) Emotional & Social Toll of Hearing LossHow hearing loss can lead to isolation, stigma, and cognitive decline.(00:26:06) Born Deaf vs. Later Hearing LossExperiential differences between those with early and late hearing loss.(00:27:52) Paul Simon's Role and AdvocacyHow the artist got involved with Tina's work and the initiative at Stanford.(00:29:44) Protecting Your HearingBest practices for protecting your hearing.(00:33:21) Conclusion Connect With Us:Episode Transcripts >>> The Future of Everything WebsiteConnect with Russ >>> Threads / Bluesky / MastodonConnect with School of Engineering >>>Twitter/X / Instagram / LinkedIn / Facebook

We love to hear from our listeners. Send us a message.In this episode of Cell & Gene: The Podcast, Host Erin Harris dives into the world of retinal gene therapy with Kenji Fujita, M.D., Chief Medical Officer of Atsena Therapeutics. Atsena develops treatments for inherited retinal diseases, including X-linked retinoschisis (XLRS), a rare genetic condition that currently has no FDA-approved therapy. They talk through the impact of XLRS on patients, the challenges of gene delivery to the retina, and how Atsena's AAV vector technology is designed to overcome these hurdles. They also discuss the significance of the Fast Track designation recently granted to Atsena's XLRS program, the role of patient advocacy in rare disease research, and the key milestones to watch for in the months ahead.Subscribe to the podcast!Apple | Spotify | YouTube

Emerging cell and gene therapies represent areas of great promise for people with rare, genetic diseases, but the translation gap for these medicines can leave them stalled at the lab. Mass General Brigham in 2022 launched the Gene and Cell Therapy Institute, a research hub dedicated to advancing gene and cell therapies for various diseases to bridge the divide between academic labs and clinical development of therapies. The institute brings together more than 500 researchers and clinicians and boasts unique assets, such as its RNA Therapeutics Core, which enable it to produce cutting-edge circular RNA on-site. We spoke to Nathan Yozwiak, head of research at the Mass General Brigham Cell and Gene Therapy Institute, about the need it is seeking to address, how the institute operates, and what it might do to enable the development of bespoke therapies for ultra-rare diseases.

In this episode of Absolute Gene-ius, Dr. C. Dustin Rubinstein takes us inside the world of advanced genome editing, where cutting-edge tools like CRISPR and digital PCR are helping shape the future of biomedical research.As the Director of the Advanced Genome Editing Laboratory at the University of Wisconsin-Madison, Dr. Rubinstein shares how his lab develops genetically engineered pig models to study diseases like neurofibromatosis and cancer, providing researchers with more clinically relevant models than traditional small animals. He explains how digital PCR plays a crucial role in confirming genome edits with absolute precision, eliminating the ambiguity that often comes with qPCR and sequencing alone. The discussion dives into the advantages of dPCR for copy number variation analysis and gene editing confirmation, emphasizing the importance of multiple complementary technologies in modern molecular biology.Beyond the science, Dr. Rubinstein reflects on his career path, the value of mentors, and the unpredictable nature of scientific discovery. He also joins in on some lighthearted lab humor and shares his most embarrassing (and proudest) moments in research. Tune in for an insightful and entertaining look at the intersection of gene editing, career evolution, and the future of molecular biology.Visit the Absolute Gene-ius pageto learn more about the guests, the hosts, and the Applied Biosystems QuantStudio Absolute Q Digital PCR System. 

In this episode, we dive into the world of cryogenic storage and transportation of cell and gene therapy. Our guest, Krystal Haynes Senior Director International 3PL Business Development at ICS helps break down the complexities of cryogenic storage and the intricacies of CGT's. For more information subscribe to Running on Ice the newsletter or podcast. Follow the Running on Ice Podcast Other FreightWaves Shows Learn more about your ad choices. Visit megaphone.fm/adchoices

Episode 314.  James B and Eddie take on four Spider-Man Unlimited Books in this express episode containing twelve stories. Sponsored by Dr. Warren's Gene Therapy and Abraham Zimmers A to Z Solutions Theme Music by Jeff Kenniston.  This Episode Edited by James B using Audacity and Cleanfeed.  Summaries written by James B and Ollie Osnick.  Most Sound effects and music generously provided royalty free by www.fesliyanstudios.com and https://www.zapsplat.com/  Check out all the episodes on letsreadspiderman.podbean.com or wherever you get your podcasts. Check out our live meetup and Discord Channel here https://docs.google.com/document/d/1_mW6htjJUHOzlViEvPQqR-k68tClMGAi85Bi_xrlV7w/edit