Podcasts about Gene therapy

What if an Alzheimer's therapy did not just slow decline, but restored memory and physical health back to baseline?In this episode of Spark Time, we sit down with Dan Salain, President and CEO of Recall Therapeutics, and Dr. Atul Chopra, Co-founder and CSO, to unpack one of the boldest ideas in neuroscience today: RTX-100, a gene therapy designed to restore cognition and systemic health in Alzheimer's disease. Dan and Atul walk us through the science behind targeting memory circuits rather than just clearing amyloid, how a liver-expressed protein naturally crosses the blood–brain barrier, and why their preclinical data show full recovery of associative, recognition, and spatial memory in advanced mouse models.We also explore the overlooked systemic side of Alzheimer's cachexia, sarcopenia, frailty, UTIs, pneumonia and how RTX-100 appears to normalize body weight, muscle strength, endurance, and fluid balance alongside cognitive recovery. From serendipity in the lab to regulatory strategy and potential paths to market, this conversation opens up a very different vision for what treating Alzheimer's and even aging itself could look like.

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we'll explore the latest advances and strategic moves shaping the industry, providing you with insights into how these developments might influence drug development and patient care.The pharmaceutical and biotech sectors are currently experiencing a wave of transformative changes. A significant development is the trade agreement between the United States and the United Kingdom, which excludes medicines from import tariffs. This strategic move, orchestrated by the Trump administration, is set to reduce costs and bolster investments in pharma sectors across both nations. By enhancing market accessibility, it aims to stimulate cross-border investment in pharmaceutical research and production.On the clinical front, Eli Lilly is making headlines by joining Novo Nordisk in reducing self-pay prices for its GLP-1 receptor agonist, Zepbound. This reflects a broader industry trend towards patient-centric pricing models aimed at improving affordability. With healthcare costs on the rise, these measures could ease financial burdens for patients requiring long-term medication regimens.Regulatory updates are also making waves, with the FDA planning stricter vaccine regulations under Dr. Vinay Prasad's leadership at the Center for Biologics Evaluation and Research (CBER). These updates come amid concerns over vaccine safety during COVID-19-related incidents, underscoring a commitment to maintaining public trust in vaccines. Regulatory scrutiny continues as Prasad focuses on COVID-19 vaccine safety in children amid reports linking 10 child deaths to vaccines via VAERS—emphasizing challenges interpreting safety data while highlighting the need for robust methodologies ensuring reliable causality conclusions.In corporate restructuring news, Valneva is streamlining its operations by closing a site and eliminating 30 roles. This move highlights an industry focus on optimizing resources to bolster vaccine development pipelines. Meanwhile, Microsize and Schedio's acquisition of Lonza's Swiss micronization plant underscores ongoing investments in advanced manufacturing technologies critical for high-quality pharmaceuticals.In ophthalmology, Belite Bio is advancing with promising Phase 3 results for tinlarebant in treating Stargardt disease—a rare genetic eye disorder. This success positions Belite to file for FDA approval, potentially expanding treatment options for this underserved patient population. Such advancements in targeted therapies emphasize the need for ongoing research in genetic disorders.Regeneron is betting $150 million on Tessera's gene writing technology targeting alpha-1 antitrypsin deficiency (AATD). This collaboration highlights the industry's growing interest in gene therapy as a frontier for treating rare diseases, marking a shift towards precision medicine where tailored genetic interventions offer hope for previously untreatable conditions. Regeneron's strategic move investing $275 million partnering with Tessera exploring gene editing capabilities—focusing on TSRA-196 targeting the SERPINA1 gene linked to AATD showcases potential advancing therapeutic options for genetic disorders through gene editing technologies offering new disease treatment avenues.AI-driven platforms were spotlighted at RSNA 2025 by industry leaders such as GE Healthcare, Philips, and Siemens. These innovations promise to revolutionize radiological workflows by enhancing diagnostic accuracy and operational efficiency through AI integration. As AI continues to permeate healthcare technologies, its potential to transform diagnostic processes marks a significant leap towards personalized medicine.Akebia Therapeutics' acquisition ofSupport the show

In this uplifting episode, hosts Beth Glassman and Cathy Gildenhorn sit down with Leanna Scaglione, a powerhouse rare disease advocate and marathon runner living with NF2-Related Schwannomatosis (NF2-SWN), formerly known as neurofibromatosis type 2. Diagnosed at just 16 years old, Leanna's life changed dramatically when tumors were discovered in her nervous system, a hallmark of NF2-SWN. Many would have slowed down. Instead, Leanna sped up. Today, she has: Completed multiple marathons, including Berlin and New York City Participated in clinical trials Become an advocate and 2025 Ambassador for the Children's Tumor Foundation Set her sights on becoming the first person with NF2 to complete all 7 Abbott World Marathon Majors Her message is powerful: a diagnosis doesn't define the finish line. In This Episode, We Discuss: What NF2-SWN is and how it impacts the nervous system The emotional toll of receiving a life-altering diagnosis as a teenager Losing a dream — and finding a new one Running marathons through surgeries, treatments, and uncertainty How advocacy and visibility can change the rare disease landscape Building a life rooted in resilience, purpose, and possibility About Our Guest: Leanna Scaglione is 34 years old living with NF2-Related Schwannomatosis (NF2-SWN). Ever since being diagnosed at 16 years old, she has refused to live defined by her diagnosis. From relearning to walk, participating in immunotherapy drug trials, and going through numerous surgeries she has defied the odds against her. Most recently she has undergone surgery to remove her right acoustic neuroma. She continues to live her best life as an endurance runner, sharing her story and experiences living with NF2 in hopes to spread awareness, motivate her community and rally for a cure. Resources:  Children's Tumor Foundation NF2 Accelerator: A Strategic Portfolio Approach to End NF2 Articles Explaining NF2 MedlinePlus GeneReviews National Organization for Rare Disorders (NORD) Research from China that Leanna reference for a gene therapy for NF2 Yuan R, Wang B, Wang Y, Liu P. Gene Therapy for Neurofibromatosis Type 2-Related Schwannomatosis: Recent Progress, Challenges, and Future Directions. Oncol Ther. 2024 Jun;12(2):257-276. doi: 10.1007/s40487-024-00279-2. Epub 2024 May 17. PMID: 38760612; PMCID: PMC11187037. Additional Research Articles Alexandra K. O'Donohue, Samantha L. Ginn, Gaetan Burgio, Yemima Berman, Gabriel Dabscheck, Aaron Schindeler, The evolving landscape of NF gene therapy: Hurdles and opportunities, Molecular Therapy Nucleic Acids, Volume 36, Issue 1, 2025, 102475 ,ISSN 2162-2531, https://doi.org/10.1016/j.omtn.2025.102475. Connect With Us:   Stay tuned for the next new episode of “It Happened To Me”! In the meantime, you can listen to our previous episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “It Happened To Me”.  “It Happened To Me” is created and hosted by Cathy Gildenhorn and Beth Glassman. DNA Today's Kira Dineen is our executive producer and marketing lead. Amanda Andreoli is our associate producer. Ashlyn Enokian is our graphic designer. See what else we are up to on Twitter, Instagram, Facebook, YouTube and our website, ItHappenedToMePod.com. Questions/inquiries can be sent to ItHappenedToMePod@gmail.com. 

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today we dive into some of the most significant and exciting advancements shaping the industry.Let's start with a breakthrough coming out of recent clinical trials. A novel approach to treating Alzheimer's disease is making waves. Researchers have been focusing on a monoclonal antibody designed to target amyloid-beta plaques in the brains of patients suffering from this debilitating condition. The latest Phase 3 trial results have shown a promising reduction in cognitive decline among participants, offering a glimmer of hope for millions affected by Alzheimer's worldwide. The study, conducted over 18 months, involved more than 1,500 patients and demonstrated a statistically significant slowing of disease progression compared to placebo. This could mark a pivotal moment in Alzheimer's treatment, shifting the paradigm from symptomatic relief to disease modification.In regulatory news, the FDA has granted approval to a new gene therapy for hemophilia B. This therapy, developed using cutting-edge CRISPR technology, offers a potential cure for patients who have long relied on regular infusions to manage their bleeding disorder. By directly editing the genes responsible for clotting factor production, this therapy can potentially provide long-term relief with just a single administration. The approval follows extensive clinical trials that showed sustained increases in clotting factor levels and a dramatic reduction in bleeding episodes among participants. This development not only underscores the growing impact of genomic medicine but also highlights the regulatory body's commitment to advancing innovative treatments that meet unmet medical needs.Shifting focus, there's an intriguing trend emerging in oncology drug development. The industry is increasingly adopting personalized medicine approaches, tailoring treatments based on individual genetic profiles. This shift is particularly evident in the rise of targeted therapies and combination regimens designed to tackle cancer at its genetic roots. A recent study demonstrated how combining targeted therapies with immune checkpoint inhibitors can enhance treatment efficacy in certain types of cancer, such as melanoma and non-small cell lung cancer. By understanding the specific mutations driving tumor growth and leveraging the body's immune response, these combinations are setting new standards for cancer care.In another significant development, researchers have unveiled promising results from an innovative mRNA vaccine trial aimed at combating infectious diseases beyond COVID-19. The technology, which gained prominence during the pandemic, is now being applied to other viral threats like influenza and RSV. Early-phase clinical trials have shown robust immune responses and favorable safety profiles, suggesting mRNA vaccines could soon revolutionize how we approach vaccination for a variety of pathogens. This versatility and rapid development timeline make mRNA platforms particularly attractive for addressing emerging infectious diseases swiftly.Meanwhile, a new study has raised awareness about antibiotic resistance—a growing concern globally. Scientists have developed an advanced diagnostic tool capable of rapidly identifying bacterial infections and their resistance profiles within hours instead of days. This innovation can significantly impact how clinicians prescribe antibiotics, ensuring targeted treatments that minimize resistance development. By providing real-time insights into bacterial genetics and resistance mechanisms, this tool empowers healthcare providers to make informed decisions that preserve antibiotic efficacy for future generations.Lastly, let's toSupport the show

Happy Thanksgiving… Thursday, November 27, 2025. Week 48.   Continued from #S10e189…   And the AAV Paper (https://pubmed.ncbi.nlm.nih.gov/40988338/) from #S10e187… https://curesyngap1.org/podcasts/syngap10/clinical-research-ai-dx-nl47-survey-autism-press-6-days-to-register-for-syngap1conf-s10e187/   https://curesyngap1.org/blog/ Issac's story, Transmitter reprint, Scramble 4 write up and JK on #Autism, #MustRead   https://curesyngap1.org/resources/webinars/ 119 - 112 Register for livestream of the conference, AAV from Allen Inst., dos en espanol, Missense, Unlock and Rare-X for ProMMiS.   https://curesyngap1.org/podcasts/syngap1-stories/  A gold mine have you listened to #38, the Virginie Pod, really must listen, she is our leader. https://www.linkedin.com/posts/graglia_syngap1stories-syngap1-syngap1storiesty-activity-7387203351907708928-liNL    CLINICAL TRIAL & GENETIC MEDICINE CORNER Example of Ultragenyx FAST Angelman follow on trial to look at other ages and genotypes, key message, never give up. https://www.linkedin.com/posts/cureangelman_the-global-aurora-study-will-enroll-approximately-activity-7389647402690957312-Bihi Congrats to Novartis on approval of the first Gene Therapy to Cure SMA! https://www.linkedin.com/posts/graglia_sma-fdaapproval-rarediseaseinnovation-activity-7398939783005347840-Ocd_ Remember Spinraza was approved in December 2019.   TODOS Sign up for Citizen Health: https://www.citizen.health/partners/srf USE YOUR ICD-10 F78.A1 #S10e185 https://www.youtube.com/watch?v=dale0NbxDpU Go to CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/   SOCIAL MATTERS 4,468 LinkedIn.  https://www.linkedin.com/company/curesyngap1/  1,480 YouTube.  https://www.youtube.com/@CureSYNGAP1    11.2k Twitter https://twitter.com/cureSYNGAP1  45k Insta https://www.instagram.com/curesyngap1/    $CAMP stock is at $3.62 on 26 Nov. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ Episode 190 of #Syngap10 #CureSYNGAP1

This week, a world first gene therapy treats rare Hunter syndrome. Could these personalised medicines be used more widely? We speak to Claire Booth, professor in Gene Therapy at Great Ormond Street Hospital. And high in the Chilean desert, the last bit of 13 billion year old light has hit the mirror of the Atacama Cosmology Telescope for the last time. Dr Jenifer Millard, a science communicator and host of the Awesome Astronomy podcast, tells us what it's been up to for the past 20 years.And Penny Sarchet, managing editor at New Scientist brings her pick of the latest new discoveries.Think you know space? Head to bbc.co.uk, search for BBC Inside Science, and follow the links to the Open University to try The Open University Space Quiz. Presenter: Tom Whipple Producers: Alex Mansfield, Ella Hubber, Jonathan Blackwell, Tim Dodd and Clare Salisbury Editor: Martin Smith Production Co-ordinator: Jana Bennett-Holesworth

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve deep into a series of transformative events that underscore the dynamic nature of our industry, where scientific innovation meets regulatory evolution and market adaptation.We begin with significant regulatory news from Medicare, which recently announced price reductions for 15 prescription drugs, including Novo Nordisk's semaglutide products, Ozempic and Wegovy. This initiative is part of the Inflation Reduction Act aimed at making essential medications more affordable. By potentially increasing accessibility to these treatments, this move highlights a growing trend towards cost containment in drug pricing within the U.S. healthcare system. It reflects a broader effort to ensure that life-saving treatments remain within reach for more patients, emphasizing the need for balance between innovation and affordability.Turning to approvals, Otsuka has secured FDA clearance for Voyxact, a first-in-class treatment targeting IgA nephropathy (IgAN). This positions Otsuka in an increasingly competitive market space populated by major players like Novartis and Vertex. The entry of Voyxact could pave the way for innovative therapeutics in kidney diseases, offering new hope to patients who have had limited treatment options until now.On the other side of the Atlantic, French authorities have conducted a raid on Sanofi's headquarters as part of a tax fraud investigation. This development sheds light on ongoing scrutiny in the pharmaceutical sector regarding financial practices and regulatory compliance. Such investigations can have far-reaching implications on corporate governance and transparency, reminding us of the importance of ethical practices in maintaining industry trust.Novo Nordisk has strategically used its FDA national priority voucher to expedite the review process for a high-dose formulation of Wegovy. This move underscores the importance of regulatory incentives in accelerating drug development timelines, allowing for quicker patient access to potentially life-changing therapies. It's a testament to how strategic navigation through regulatory pathways can significantly impact drug availability.In clinical trials, Sarepta Therapeutics received FDA clearance to conduct a study combining its gene therapy Elevidys with sirolimus in patients with Duchenne muscular dystrophy. The study aims to address liver safety issues associated with Elevidys, which had led to previous label restrictions. This reflects the industry's commitment to enhancing therapeutic safety profiles while expanding treatment indications.In oncology advancements, AstraZeneca's Imfinzi received FDA approval for use in early-stage stomach cancer, marking its third perioperative indication. This approval underscores the expanding role of immunotherapy across various cancer types and stages, offering new treatment paradigms that could improve surgical outcomes and long-term patient survival.Despite these advances, there is skepticism regarding artificial intelligence's role in regulatory compliance submissions among pharmaceutical professionals. A survey reveals that 65% express distrust towards AI-generated outputs, highlighting challenges that AI technologies face in gaining acceptance within highly regulated environments such as pharmaceuticals. However, federal recommendations to revamp U.S. biotechnology research emphasize incorporating AI into scientific processes to maintain global competitiveness. This call reflects concerns over potential declines in innovation leadership and underscores the need for strategic investment in research infrastructure.In antitrust news, the Federal Trade Commission (FTC) outlined its case agaiSupport the show

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a landscape rich with scientific innovation, regulatory scrutiny, and strategic business decisions shaping the future of healthcare.Let's begin with Novartis, which has achieved a significant milestone by securing FDA approval for Itvisma. This is an intrathecal formulation of its gene therapy Zolgensma, designed to treat older patients with spinal muscular atrophy (SMA). Priced at $2.59 million, this approval is a critical advancement in gene therapy for SMA, broadening the treatment horizon for a wider patient demographic. Gene therapies are increasingly crucial in addressing rare genetic disorders, offering transformative potential in patient care. Novartis's SMA market expansion post-FDA approval of Itvisma not only enhances its SMA portfolio but also signifies growing acceptance towards gene therapies as viable treatment options for genetic disorders. Meanwhile, Novartis is strategically restructuring, planning to cut 550 jobs at a Swiss plant by 2027 while expanding its workforce in North Carolina. This move reflects broader industry trends toward optimizing global operations and investing in regions with strategic manufacturing capabilities.In the sphere of regulatory scrutiny, lawmakers are questioning the FDA's National Priority Voucher Program amid concerns about corruption and expedited reviews. This situation highlights ongoing challenges within regulatory frameworks to balance innovation speed with rigorous safety assessments. An investigation into the FDA's new priority review voucher program has been initiated due to concerns over corruption and expedited review processes potentially compromising drug safety. This inquiry could influence future regulatory frameworks and underscores balancing accelerated drug approvals with rigorous safety standards. Richard Pazdur expressed concerns about expedited drug approval programs' safety and legality as he takes on his new role as director of the FDA's Center for Drug Evaluation and Research. These initiatives aim to accelerate drug reviews but spark debate over patient safety implications—underscoring an ongoing tension between innovation speed and regulatory diligence.Turning to Novo Nordisk, their expansion of the Amycretin program demonstrates a commitment to tackling chronic conditions like diabetes. Following promising Phase 2 data showcasing dual agonist capabilities, Novo Nordisk is advancing pivotal trials focused on obesity. This strategic pivot aligns with market needs and scientific discoveries that could significantly enhance diabetes management options. Further emphasizing Novo Nordisk's commitment to diabetes management, their expansion of the amycretin program after promising Phase 2 results demonstrates the efficacy of a dual agonist originally focused on obesity. This underscores a trend toward multifunctional biologics addressing metabolic disorders by targeting multiple pathways—indicative of broader industry shifts towards integrated therapeutic approaches. Novo Nordisk's recent mid-stage clinical trial results for Amycretin—a weight loss treatment—are noteworthy as they demonstrated sustained efficacy over 36 weeks in type 2 diabetes patients without a plateau in weight loss. Analysts highlight its potential as a superior therapeutic option in the burgeoning weight loss market due to its durable solution for weight management.On a contrasting note, SK Life Science encounters regulatory hurdles as the FDA scrutinizes advertising practices related to its antiseizure medication Xcopri. This scenario underscores the complex interplay between marketing strategies and regulatory compliance within the pharmaceuticSupport the show

New Crash Test Dummies for women…  Freddie Mercury old boyfriend writes book…  I Wool Survive…  Email: Chewingthefat@theblaze.com   www.blazetv.com/jeffy   $20 off annual plan right now ( limited time )   New Years Eve hosts on ABC…  Chelsea Handler to host Critics Choice again…  All Reality AMC+ thru Prime…  Social Media usage..  .Gene Therapy breakthrough…  Who Died Today: Jimmy Cliff 81 /  Udo Kier 81 / John Eimen 76 / Dharmendra 89…  Joke(s) of The Day… Learn more about your ad choices. Visit megaphone.fm/adchoices

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a host of transformative events reshaping the landscape, from strategic acquisitions and funding infusions to regulatory maneuvers and scientific breakthroughs.Johnson & Johnson has taken a decisive step in its oncology strategy with the $3 billion acquisition of Halda's cell death technology. This acquisition, focusing on the "hold and kill" bifunctional small molecule platform, is poised to enhance J&J's prostate cancer pipeline significantly. It underscores J&J's commitment to expanding its oncology portfolio through innovative platforms designed to improve therapeutic outcomes. The move highlights a broader industry trend toward personalized medicine and targeted cancer therapies, which are becoming pivotal in improving patient care.In another domain of cancer treatment, Nuvalent has unveiled promising Phase 1/2 data for its candidate neladalkib, which could position the company as a formidable competitor to Pfizer's established lung cancer drug, Lorbrena. The promising data might expedite regulatory discussions with the FDA, potentially leading to an accelerated approval process. This development illustrates the competitive landscape in oncology, where firms strive to introduce novel therapies with improved efficacy and safety profiles.The field of antibody-drug conjugates (ADCs) is also experiencing significant advancements. A San Diego-based biotech has secured $120 million in funding to develop a best-in-class ADC formula, with support from Merck & Co. This initiative aims to refine the precision and efficacy of ADCs by delivering cytotoxic agents directly to cancer cells while minimizing collateral damage to healthy tissues. Such innovations are crucial as they represent a new frontier in targeted cancer therapy.In terms of financial activities, Artios Pharma's successful $115 million Series D funding round is set to bolster its clinical efforts in exploring DNA damage response inhibitors for cancer treatment. These inhibitors target cancer cells' ability to repair DNA damage, holding potential for more effective therapies against resistant cancer types. Meanwhile, Sofinnova Partners' €650 million raise for biotech and medtech investments amid a volatile economic environment underscores continued investor confidence in life sciences despite market uncertainties.Bayer is making strategic moves in China by opening an incubator in Beijing. This facility will host local biopharma companies such as Suzhou Puhe Biopharma and Beijing Youngen Technology, fostering innovation and collaboration within China's burgeoning biotech landscape. Such initiatives reflect global efforts to leverage regional strengths and foster cross-border collaborations.On the operational side, Nxera Pharma is restructuring its workforce by laying off 15% of its staff as part of a strategic pivot towards profitability. This decision mirrors broader industry trends where companies refocus resources on core projects to streamline operations and enhance financial stability.A recent study has highlighted the impact of NIH grant cuts on clinical trials across the United States. Over 383 trials involving more than 74,000 patients have been disrupted due to funding terminations under the current administration. This situation raises concerns about the sustainability of clinical research funding and its implications for ongoing medical advancements.Jazz Pharmaceuticals has reported practice-changing Phase 3 results for its HER2-targeted drug Ziihera for gastroesophageal adenocarcinoma. These findings reaffirm Jazz's confidence in positioning Ziihera as a preferred first-line treatment option for HER2-positive cancers, poSupport the show

In this episode of the Parkinson's Podcast, learn about gene therapy for Parkinson's, including common misunderstandings and ongoing research about it.  This episode contains some technical terms and abbreviations. For your convenience, here is a short list of definitions that might be helpful.   AADC – Aromatic L-Amino Acid Decarboxylase: An enzyme that helps make dopamine (not a rock and roll band)  AAV – Adeno-Associated Virus: A harmless virus used to deliver gene therapy  CDNF – Cerebral Dopamine Neurotrophic Factor: A protein that helps keep dopamine-producing cells healthy  DBS – Deep Brain Stimulation: A surgical treatment that uses electrical signals to help control symptoms  GAD – Glutamic Acid Decarboxylase: An enzyme that helps rebalance brain circuits.   GBA – A gene linked to Parkinson's risk  GCH1 – GTP Cyclohydrolase 1: An enzyme that supports dopamine production.   GDNF – Glial Cell Line-Derived Neurotrophic Factor: A protein that helps protect brain cells  LRRK2 – A gene linked to Parkinson's risk  PARK2 – A gene linked to Parkinson's risk  PET – Positron Emission Tomography: A brain scan that shows chemical activity   PINK1 – A gene linked to Parkinson's risk   SNCA – A gene linked to Parkinson's risk  More information about Gene Therapy for Parkinson's is available here:  https://davisphinneyfoundation.org/blog/gene-therapy-parkinsons/   https://youtu.be/n2KNtRK3SIk  https://youtu.be/v9mJiO4EEag    Sign up for updates on webinars, events, and resources for the Parkinson's community: https://dpf.org/newsletter-signup

The Foundation funded preclinical studies for the approach and launched Opus.

Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]

Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]

Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]

Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]

Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]

Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]

We discuss  the early results of gene therapy of neovascular AMD by intravitreal administration of the vector with Dr. Glenn Yiu, Professor of Ophthalmology, University of California, Davis.

In this episode Rafael sits down with Michelle Chen, President and CEO of Form Bio, a techbio company using AI to advance genome engineering and the development of genetic medicines. Michelle shares her zigzag journey from scientist to executive, how her team is tackling the biggest bottlenecks in gene therapy, and why AI has become essential in designing safer, more effective treatments. ⁠⁠TPM E51 highlights >⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Episode 51 links:Michelle Chen on LinkedInForm Bio

Cody Hodgson and The RYR-1 Foundation 11/11/25Diagnosed in 2016 with RYR-1-related malignant hyperthermia, Cody Hodgson's promising NHL playing career was cut short when the effects of his disease made it impossible for him to push his body to the extent necessary in pro hockey. However, what initially looked like a major setback gave way to a very different, equally powerful calling. Now an active ambassador for the Pittsburgh, PA-based RYR-1 Foundation, Hodgson is precisely the kind of figure in the hockey world that our production staff loves connecting with.With mobile recording gear in tow, our producer and co-host, Jeff Taylor recently joined the RYR-1 Foundation in Pittsburgh for their 10th anniversary conference. A handful of in-depth interviews were conducted that day, including with former NHL'er Cody Hodgson himself, as well as Toronto-based anesthesiologist Dr. Sheila Riazi, Hodgson's diagnosing physician. Reading: The RYR-1 Foundation's Clinical Care Guidelines text:https://ryr1.org/clinical-care-guidelinesVideo: Watch Cody Hodgson score an incredible shorthanded goal, beating all five Carolina Hurricanes players:https://www.youtube.com/watch?v=1pyGSyfkO6IVideo: Wim Hof Breathing Method (YouTube):https://www.youtube.com/watch?v=tybOi4hjZFQToday's episode was co-produced by Jeff Taylor and Ashley Taylor.Very special thanks to Espresso A Mano, Pittsburgh's finest coffee roasters and cafe, for partnering with Fly Penguins Fly podcast. Visit an Espresso A Mano location near you:https://espressoamano.com/Thank you for listening!! Follow the podcast on X/Twitter: @penspodJeff Taylor: @penspod_JT // Jordan DeFigio: @fidgenewtonLETS GO PENS.JEFF TAYLOR + JORDAN DEFIGIOFly Penguins Fly Podcast is produced by Jeff TaylorOriginal theme and outro music composed and recorded by Jeff TaylorMark Guiliana played drums, Ashley Taylor sang.Interested in becoming a sponsor of the podcast?Email the Fly Penguins Fly Podcast at: penspod@gmail.comShane Taylor designed and delivered our artwork.Hire Shane: shanetaylordesigns@gmail.comThis episode includes portions of the following musical selections:Fly Penguins Fly Theme composed and produced by Jeff TaylorKid Kodi by Blue Dot SessionsHammers and Tape composed and produced by Jeff TaylorArp Landing composed and produced by Jeff TaylorInside the Paper Crane by Blue Dot SessionsHardboil by Blue Dot SessionsCamphor Kingdom by Blue Dot SessionsDiatom by Blue Dot Sessions

Host Matt Burgess speaks with Assistant Professor Barbara Harrison of Howard University about APOL1-related kidney disease, sickle cell disease, and the role of cultural awareness in genetic counselling. They discuss health inequities, community trust around new therapies like gene editing, and efforts to increase diversity in the profession through the GOLDEN mentorship program.

For the first time in human history, we can diagnose thousands of genetic diseases—often for under $1,000—but we still can't treat most of them. The problem isn't understanding what's broken; it's delivering the fix to the right cells.Eric Kelsic, CEO of Dyno Therapeutics, joins a16z's Jorge Conde to explain how AI-designed protein shells are solving gene therapy's delivery crisis. They explore why Huntington's patients can now get 15 extra years of healthy life, how Dyno inverted the liver-to-brain delivery ratio by 1000x, and why capsids evolved by nature are now being designed by machine learning models trained on millions of variants.Eric introduces the concept of genetic agency—humanity's first-ever ability to take action at the DNA level—and details why solving delivery for common diseases will make ultra-rare disease treatments economically viable. Plus: what happens when gene therapy requires neurosurgery today but could be a simple injection tomorrow, why recent deaths in clinical trials prove we need better technology now, and how genetic medicine could become as routine as surgery within our lifetimes. Resources:Follow Eric on X: https://x.com/ekelsicFollow Jorge on X: https://x.com/JorgeCondeBioLearn more about GATC 2025: https://www.dynotx.com/gatc2025 Stay Updated:If you enjoyed this episode, be sure to like, subscribe, and share with your friends!Find a16z on X: https://x.com/a16zFind a16z on LinkedIn: https://www.linkedin.com/company/a16zListen to the a16z Podcast on Spotify: https://open.spotify.com/show/5bC65RDvs3oxnLyqqvkUYXListen to the a16z Podcast on Apple Podcasts: https://podcasts.apple.com/us/podcast/a16z-podcast/id842818711Follow our host: https://x.com/eriktorenbergPlease note that the content here is for informational purposes only; should NOT be taken as legal, business, tax, or investment advice or be used to evaluate any investment or security; and is not directed at any investors or potential investors in any a16z fund. a16z and its affiliates may maintain investments in the companies discussed. For more details please see a16z.com/disclosures.  Hosted by Simplecast, an AdsWizz company. See pcm.adswizz.com for information about our collection and use of personal data for advertising.

Alicia Silver, senior director at ADVI Health, highlights the evolving landscape of cell and gene therapy and the need to improve patient access and payment for these treatments. Availability of these therapies for solid tumors and genetic diseases like sickle cell disease is expanding due to the transition from inpatient to outpatient and community settings. The FDA's decision to remove REMS requirements for specific therapies has accelerated the growth of facilities to provide care, particularly for vulnerable populations. Alicia explains, "We work with a number of different cell and gene therapy clients throughout the sector. So we work with manufacturers who have commercialized cell and gene therapy products. So they have products that are currently on the market, manufacturers who are going through the process of clinical trials right now, working with the FDA to get approved products. But we also work with trade organizations that are working behind the scenes at the sector level, trying to get different policies and access changes for patients."   "To date, there's probably close to a couple of dozen FDA-approved cell and gene therapies, and they treat everything from blood cancers, which were the first approvals in something called CAR T. We saw blood cancers as the first approvals, and then everything through to solid tumors in oncology. But also, we have newer gene therapies for conditions like sickle cell disease. And that's an area that's been incredibly underserved and definitely will benefit from a durable gene therapy that hopefully corrects some of the issues that patients with sickle cell disease have, like pain crises that end up in a hospital. So from that perspective, we see a really wide range of treatments available to patients today and many more on the horizon." "I think the price tag is definitely somewhat of sticker shock for people who don't understand how cell and gene therapy products are valued. And so what we do a lot of times, educating on, is helping payers understand that it's not necessarily $2 million for a treatment that's going to be a recurrent payment, but something that's kind of an investment in the patient's and the plan's future." #ADVIHealth #CellTherapy #GeneTherapy #AcesstoCellGeneTherapy #ClinicalTrials  advi.com Download the transcript here

Alicia Silver, senior director at ADVI Health, highlights the evolving landscape of cell and gene therapy and the need to improve patient access and payment for these treatments. Availability of these therapies for solid tumors and genetic diseases like sickle cell disease is expanding due to the transition from inpatient to outpatient and community settings. The FDA's decision to remove REMS requirements for specific therapies has accelerated the growth of facilities to provide care, particularly for vulnerable populations. Alicia explains, "We work with a number of different cell and gene therapy clients throughout the sector. So we work with manufacturers who have commercialized cell and gene therapy products. So they have products that are currently on the market, manufacturers who are going through the process of clinical trials right now, working with the FDA to get approved products. But we also work with trade organizations that are working behind the scenes at the sector level, trying to get different policies and access changes for patients."   "To date, there's probably close to a couple of dozen FDA-approved cell and gene therapies, and they treat everything from blood cancers, which were the first approvals in something called CAR T. We saw blood cancers as the first approvals, and then everything through to solid tumors in oncology. But also, we have newer gene therapies for conditions like sickle cell disease. And that's an area that's been incredibly underserved and definitely will benefit from a durable gene therapy that hopefully corrects some of the issues that patients with sickle cell disease have, like pain crises that end up in a hospital. So from that perspective, we see a really wide range of treatments available to patients today and many more on the horizon." "I think the price tag is definitely somewhat of sticker shock for people who don't understand how cell and gene therapy products are valued. And so what we do a lot of times, educating on, is helping payers understand that it's not necessarily $2 million for a treatment that's going to be a recurrent payment, but something that's kind of an investment in the patient's and the plan's future." #ADVIHealth #CellTherapy #GeneTherapy #AcesstoCellGeneTherapy #ClinicalTrials  advi.com Listen to the podcast here

In this episode of Speaking of Mol Bio, Dr. Andre Ghetti, CEO of AnaBios, offers a deep dive into the world of translational preclinical research. AnaBios is redefining early human insights by using ethically sourced, functional human tissues and cells to generate actionable data before compounds ever enter clinical trials. Ghetti walks us through the company's approach of offering human-relevant safety and efficacy data, validating drug targets, and supporting everything from small startups to major pharma groups.We learn how AnaBios engages with clients to customize assays, especially in high-need areas like non-opioid pain therapies, fibrosis, and cardiac safety, and how they use a blend of standardized and novel functional assays, some of which required building their own hardware. He also discusses their integration of RT-PCR, RNA-seq, and calcium imaging, including genetically encoded sensors to monitor neuronal activity at scale.From their use of machine learning to analyze massive data sets, to collaborations with the FDA, to their unique ability to preserve tissue viability across the U.S., AnaBios offers a powerful glimpse into the future of translational biology. Dr. Ghetti also shares advice for young scientists and reflects on what's next for AnaBios, including oncology and stem-cell model integration. Subscribe to get future episodes as they drop and if you like what you're hearing we hope you'll share a review or recommend the series to a colleague.  Visit the Invitrogen School of Molecular Biology to access helpful molecular biology resources and educational content, and please share this resource with anyone you know working in molecular biology. For Research Use Only. Not for use in diagnostic procedures.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a series of transformative events reshaping the landscape of healthcare, from strategic mergers and regulatory shifts to groundbreaking advancements in drug development.Let's begin with a monumental merger that signals a shift towards more integrated healthcare solutions. The $48.7 billion acquisition of Kenvue, a consumer health spinout from Johnson & Johnson, by Kimberly-Clark illustrates the growing convergence between consumer health products and traditional pharmaceuticals. This strategic move highlights a trend towards expanding product portfolios and enhancing distribution networks, aiming to better address comprehensive patient needs. Mergers like these could redefine how healthcare products are marketed and delivered, emphasizing holistic approaches to patient care.Turning to regulatory news, the resignation of Dr. George Tidmarsh from the FDA due to controversial communications has spotlighted the ongoing challenges within regulatory oversight. This incident underscores the delicate balance regulators must maintain in ensuring transparency while safeguarding sensitive information. Such developments are crucial as they directly affect public trust in drug approval processes and the industry's ability to navigate complex regulatory landscapes.In scientific advancements, Roche is making significant progress with its drug Gazyva for autoimmune diseases. Following FDA approval for lupus-related kidney disease, promising Phase 3 trial results for systemic lupus erythematosus (SLE) are propelling Roche closer to offering new hope for patients with this chronic condition, which currently has limited effective treatments. This success underscores the potential of targeted therapies in transforming treatment paradigms for autoimmune diseases.Eli Lilly's $3 billion investment in a new manufacturing facility in the Netherlands marks a strategic effort to bolster oral medicine production globally. This expansion not only reinforces Lilly's commitment to meeting global demand but also reflects an industry-wide trend towards investing in scalable manufacturing capabilities. Such moves are critical for ensuring supply chain resilience and addressing rising healthcare needs worldwide.In legal news, Pfizer's fierce competition with Metsera over Novo Nordisk's counteroffer highlights the high stakes involved in securing promising assets within the biotech sector—a sector particularly focused on obesity treatment due to its substantial market growth potential. The outcome of this legal battle could influence future strategic partnerships and acquisitions, demonstrating the intense competition among pharmaceutical giants.Meanwhile, UniQure faces challenges as its gene therapy for Huntington's disease encounters regulatory hurdles with the FDA. Despite initial expectations as a groundbreaking treatment, this setback emphasizes the rigorous scrutiny gene therapies undergo to ensure safety and efficacy. Such hurdles highlight the complexities of advancing novel therapies through regulatory pathways.Policy developments are also reshaping drug pricing structures as evidenced by HRSA's approval of eight drugmakers' plans for a 340B rebate model pilot program. This initiative aims at optimizing pricing structures while balancing cost containment with access to essential medications for underserved populations—a critical concern in today's healthcare landscape.International collaborations continue to play a pivotal role in accelerating drug discovery and development. Neurocrine Biosciences' $880 million deal with China's TransThera Sciences exemplifies such partnerships, focusing on emerging therapeutic areas like immunology. These collaborations are vital in leveraging global expertise and resources to drive innovatSupport the show

From childhood curiosity and early passions to her son's life-changing diagnosis, Liz shares the deeply personal journey that turned her into one of the most daring figures in longevity science — even becoming the first human to test her own gene therapy.Together, Len and Liz dive into:The truth about telomeres, stem cells, and the hallmarks of agingHow AAV gene therapy could rewrite the human lifespanThe clash between innovation, regulation, and Big PharmaWhy mental health, music, and creativity are essential to longevityAnd what the future of aging gracefully — or not at all — might look likeBy the end, you'll be questioning what it really means to live a long life… and whether immortality is closer than we think.  EndoDNA: Where Genetic Science Meets Actionable Patient CareEndoDNA bridges the gap between complex genomics and patient wellness. Our patented DNA analysis platforms and AI technology provide genetic insights that support and enhance your clinical expertise.Click here to check out to take control over your Personal Health & Wellness Connect with EndoDNA on SOCIAL: IG | X | YOUTUBE | FBConnect with host, Len May, on IG Hosted by Simplecast, an AdsWizz company. See pcm.adswizz.com for information about our collection and use of personal data for advertising.

Could optogenetics represent the innovative thinking needed to restore some function in retinitis pigmentosa (RP) patients? Allen Ho, MD, joins us to discuss the state of play in optogenetics, specifically regarding MCO-010 (Nanoscope Therapeutics). What have we seen in the several years since the first patients with RP were dosed with MCO-010 in the RESTORE study? And Charles Wykoff, MD, PhD, joins us to review data assessing the safety and efficacy of sura-vec (Regenxbio/AbbVie) for the treatment of non-proliferative diabetic retinopathy. What do patients look like at 2 years after a single suprachoroidal injection? Listen in to find out.  

Vidcast:  https://www.instagram.com/p/DQdnYr9jZwV/An exciting, ground-breaking clinical study now shows that children born with a defective gene coding for a vital inner ear protein can have that gene  repaired and hearing restored. This phenomenally successful preliminary clinical trial was recently published in the New England Journal of Medicine.Genetic bioengineers at New York's Regeneron Pharmaceuticals loaded a normal copy of the otoferlin gene into a dual adeno-associated virus acting as a Trojan horse.  Twelve children, born without the ability to synthesize otoferlin protein, received the gene injection, dubbed DB-OTO therapy, into their inner ears at 3 clinical centers: Harvard's Mass. Eye and Ear Infirmary, UC San Diego's Children's Hospital, and University College London.  Otoferlin is necessary for the inner ear's ability to convert sound vibrations into electrical impulses.  At 24 weeks post-injection, 9 of the 12 children, 75%, regained measurable hearing.  Three, 25%, developed near normal hearing.  The gene therapy was well-tolerated without any significant side effects.This gene therapy, with further refinement and after larger clinical trials, may be a one-and-done treatment for one common form of congenital deafness. Cochlear implants will continue to be essential therapy for other types of genetic and acquired severe hearing losses pending development of other genetic and/or chemical cochlear modifications.https://www.nejm.org/doi/full/10.1056/NEJMoa2400521#deafness #children #congenital #otoferlin #dboto 

How far has gene therapy and genetics medicine advanced?  How accessible is that healthcare to Montanans? Click on the podcast to learn more about how Shodair Children’s Hospital‘s  world-class genetics program has served Montana families for 50 years, and how […] The post Making Gene Therapy Accessible in Montana first appeared on Voices of Montana.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a dynamic landscape of scientific breakthroughs, regulatory shifts, and strategic maneuvers reshaping the industry.BioMarin's recent decision to divest from its hemophilia A gene therapy, Roctavian, has garnered significant attention. Despite being the sole approved gene therapy for hemophilia A, Roctavian has struggled with sales since its launch two years ago. This move underscores the complex challenges in commercializing gene therapies, highlighting that even groundbreaking treatments can face hurdles in market penetration. It reflects broader implications for the commercialization strategies of innovative therapies and emphasizes that market acceptance is as crucial as clinical efficacy.In manufacturing and regulatory affairs, Regeneron is navigating hurdles with its Eylea HD due to persistent manufacturing issues. The FDA's complete response letter points to ongoing problems at a Novo Nordisk plant. This situation illustrates the critical role of manufacturing standards in securing regulatory approvals and ensuring consistent product availability. Regeneron's efforts to seek alternative manufacturing solutions emphasize the importance of compliance and quality assurance in the pharmaceutical landscape.Roche is advancing its kidney disease portfolio with a Phase 3 trial success for Gazyva against idiopathic nephrotic syndrome. Building on previous approvals for lupus nephritis, this achievement underscores Roche's strategic focus on expanding indications for existing biologics. It highlights the value of lifecycle management strategies in maximizing therapeutic potentials and extending the reach of established drugs.A significant shift in pharmacy benefit management is underway as Cigna's Evernorth division moves away from PBM rebates through Express Scripts. This transition towards a rebate-free model may influence industry-wide practices, addressing growing scrutiny over rebate structures criticized for their lack of transparency and their impact on drug pricing.CSL's decision to delay the spinoff of its flu vaccine unit amid declining U.S. immunization rates illustrates market challenges in vaccine uptake. The anticipated drop, particularly among older populations, raises public health concerns and underscores the necessity for enhanced outreach and education to improve immunization coverage.On the investment front, AbbVie, Regeneron, and Sanofi have collectively invested $80 million in ZAG Bio's Series A funding round. This company is developing thymus-targeted medicines for autoimmune diseases, reflecting continued interest in novel therapeutic approaches addressing unmet medical needs within the biotech space.Catalent's rebranding initiative signifies a strategic effort to align corporate identity with mission-driven objectives, emphasizing "missions that matter" as it approaches an anniversary milestone with Novo Nordisk's acquisition. Such rebranding efforts are critical for differentiating service offerings and reinforcing corporate values within competitive markets.The competitive landscape within diabetes and obesity treatment markets is experiencing a potential paradigm shift following the results from Innovent and Eli Lilly's Phase 3 trial of mazdutide. This dual GLP-1/glucagon receptor agonist outperformed Novo Nordisk's semaglutide, offering improved outcomes in weight reduction and glycemic control. Mazdutide's dual mechanism could redefine treatment protocols, offering patients enhanced therapeutic benefits.MapLight Therapeutics has successfully raised $250 million through an IPO to advance its schizophrenia treatment candidate, Cobenfy. This funding supports further clinical development and potential commercialization efforts, reflecting investor confidence in innovative neurologSupport the show

Gene therapy isn't science fiction anymore, it's becoming one of the most exciting frontiers in diabetes care. In this episode, Dr. Jeremy Pettus and Dr. Steve Edelman sit down with Dr. Fraser Wright, career gene therapist and Co-Founder/Chief Gene Therapy Officer at Kriya Therapeutics, to explore how gene therapy could one day help the body make its own insulin.Together, they unpack what gene therapy actually is, Dr. P's involvement as a medical consultant for Kriya Therapeutics, how AAV (adeno-associated virus) vectors work, and what makes this “one-and-done” approach so different from traditional treatments. From success stories in blindness and hemophilia to emerging type 1 diabetes studies, the trio breaks down the science, the safety, and the hope behind this revolutionary research.You'll learn how gene therapy has moved from rare diseases to more common ones, why the first human trials in diabetes are on the horizon, and what and what this could mean for long-term blood sugar control and independence from injections.Key Topics:Gene Therapy 101: How gene therapy differs from protein-based drugs like insulin, and why AAV vectors act as safe, engineered delivery vehicles rather than infectious viruses.Why AAV, Why Now: Seven FDA-approved AAV-based therapies have proven the potential of long-term, single-dose treatments.From Eyes to Endocrine: Lessons learned from retinal gene therapy are now guiding approaches to metabolic conditions like diabetes.The Type 1 Diabetes Approach: A muscle-targeted program aims to help the body naturally produce insulin and stabilize blood sugar levels.Control and Safety: Built-in glucose sensing and the ability to turn off gene expression ensure precision and reversibility.Real-World Considerations: How exercise, treatment site, and existing technologies like pumps or CGMs could work alongside this therapy.0:00 – Intro: Jeremy and Steve introduce gene therapy and why this topic is a game-changer2:15 – What gene therapy is and how it's different from standard protein-based treatments4:54 – Understanding AAV: safety, engineering, and why it's the preferred delivery method8:50 – Real-world success: seven FDA-approved AAV therapies for genetic diseases12:20 – Lessons learned from treating blindness and how they apply to diabetes17:40 – The Type 1 Diabetes model: using muscle tissue to produce insulin22:15 – Managing control, glucose sensing, and preventing hypoglycemia25:50 – Safety measures and how treatment can be reversed locally if needed28:30 – Exercise, durability, and what animal studies reveal about real-life performance31:10 – Timelines, trials, and what's next for gene therapy in diabetes. What's Ahead: Clinical trials expected to begin around 2026, offering cautious but real optimism for the future of diabetes treatment.Visit TCOYD's Website for more diabetes edutainment for people living with diabetes: tcoyd.org**Tune in for two new episodes each month! Like what you hear and want to help us grow? Please rate and review this podcast so we can reach more people living with diabetes!**Follow our social media channels to empower yourself with the essential areas of diabetes knowledge led by two endocrinologists living with type 1 diabetes: Facebook  |  Instagram  |  YouTube ★ Support this podcast ★

Most founders dream of raising millions. Few survive the 153 “no's” it takes to get there.Behind every biotech breakthrough lies exhaustion — late-night calls, failed rounds, and investors who walk away at the finish line.What separates the ones who make it isn't luck or timing — it's resilience built into process.In this episode, Jason Foster, CEO of Ori Biotech, shares how he transformed relentless rejection into a billion-dollar trajectory. From rebuilding cell-therapy manufacturing to leading global teams through economic storms, Jason reveals how founders can systematize grit, master storytelling, and survive when everything seems to fall apart.You'll learn how to navigate fundraising winters, why leadership begins with self-care, and how to build companies that endure long after the hype fades. If you've ever doubted your path as a builder, this conversation will remind you that resilience is not a trait — it's a practice.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into several significant shifts in the industry, marked by scientific advancements, regulatory changes, and strategic corporate maneuvers.Starting with a major acquisition, Novartis has strategically purchased Avidity Biosciences, a San Diego-based biotech company specializing in muscular dystrophy treatments, for a striking $12 billion. This substantial investment underscores Novartis's dedication to expanding its neuroscience portfolio. Avidity's innovative RNA-based therapies show great promise for treating neuromuscular diseases, highlighting a broader industry trend where large pharmaceutical companies are investing heavily in late-stage biotech firms to bolster their pipelines with cutting-edge technologies. Such moves are pivotal as they align with the growing emphasis on precision medicine and the development of novel therapeutic options for conditions with limited existing treatments.In other acquisition news, Eli Lilly has expanded its gene therapy portfolio through acquiring Adverum Biotechnologies for up to $262 million. This acquisition is expected to bolster Eli Lilly's position in the gene therapy space, particularly in ophthalmology. Gene therapy offers transformative potential by directly addressing underlying genetic causes of diseases, with Adverum's focus on ophthalmic conditions potentially offering innovative solutions for unmet medical needs in eye-related disorders. The acquisitions by Novartis and Eli Lilly reflect broader trends within the pharmaceutical industry where companies actively seek to diversify their pipelines through mergers and acquisitions. These transactions emphasize strategic incorporation of advanced biotechnologies such as RNA therapeutics and gene therapy into development portfolios aiming to deliver breakthroughs in patient care.On the regulatory front, Bayer has achieved a milestone with the U.S. FDA approval of Lynkuet (elinzanetant), a nonhormonal medication designed to manage menopause symptoms. This approval represents a significant step forward in providing alternative treatment options to a traditionally hormone-reliant segment, emphasizing the industry's shift towards diversifying therapeutic solutions and addressing unmet medical needs. This move highlights continuous efforts to address women's health issues through new pharmacological interventions.Meanwhile, Merck's Winrevair has received an updated FDA label following successful results from the Phase 3 Zenith trial. This label expansion is anticipated to enhance its market position, potentially propelling Winrevair to blockbuster status. These developments highlight the critical role of rigorous clinical trials in validating drug efficacy and safety, which ultimately influence regulatory decisions and market dynamics.BridgeBio has also made headlines with its successful Phase 3 trial for a rare disease candidate. By demonstrating significant improvements in clinical outcomes and biomarkers, BridgeBio is poised to file for FDA approval. This reflects an increasing focus on precision medicine within the industry, particularly in addressing rare and genetic disorders.In diabetes management news, Innovent and Eli Lilly's mazdutide has outperformed Novo Nordisk's semaglutide in a head-to-head study focused on glucose regulation and weight loss. As a GLP-1/glucagon dual receptor agonist, mazdutide offers broader therapeutic effects, showcasing the competitive landscape in metabolic disorders where novel mechanisms are vying for superiority.Regulatory activities remain pivotal, as demonstrated by Syndax receiving a second indication for its leukemia drug Revuforj. Such expansions underscore the importance of ongoing clinical research and regulatory engagement in maximizing a drug's therapeutic reach.NSupport the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into some of the most significant shifts and strategies shaping our industry.Novartis's acquisition of Avidity Biosciences for a staggering $12 billion marks a pivotal moment in the pharmaceutical landscape this year. With this acquisition, Novartis underscores its commitment to bolstering its neuromuscular disease pipeline. Avidity Biosciences has made a name for itself with its cutting-edge RNA therapeutic technologies, particularly its Antibody Oligonucleotide Conjugates (AOCs). This platform uniquely combines monoclonal antibodies with oligonucleotides, enhancing precision in targeting specific cell types. The integration of Avidity's technology into Novartis's research efforts could accelerate the development of new therapies, potentially transforming patient care with more effective and targeted treatment options. This move not only highlights the industry's focus on specialized therapeutic areas but also anticipates future advances in RNA therapeutics, extending beyond neuromuscular disorders to areas like oncology.In a similar vein, the FDA has shown its willingness to reconsider drugs that previously faced setbacks. GSK's Blenrep has made a return to the U.S. market after receiving approval for treating certain myeloma patients. This approval is particularly noteworthy given the drug's earlier negative advisory committee vote and postponed decision. It marks a significant rebound for GSK's oncology portfolio and reflects the FDA's dynamic approach towards drugs that show potential in specific therapeutic combinations.Meanwhile, Sanofi continues to make waves with Dupixent, achieving over €4 billion in quarterly sales due to its expanded indications. This success contrasts with a decline in Sanofi's vaccine sales, demonstrating shifting dynamics within pharmaceutical portfolios where biologics and specialty drugs are increasingly pivotal. Sanofi's recent financial report highlighted a notable 17% drop in vaccine sales due to reduced demand and pricing challenges in Europe. In response, companies must navigate fluctuating public health demands and economic pressures effectively.On the global stage, efforts to make transformative therapies like Vertex's Trikafta more accessible are gaining momentum through innovative trade-policy workarounds. A buyers club aims to introduce a lower-cost alternative produced by Bangladesh's Beximco, highlighting ongoing challenges and creative strategies in global drug accessibility.Roche's expansion through Chugai's $200 million M&A deal for an IgA nephropathy asset underscores the strategic importance of regional markets in driving growth. Similarly, Lonza's acquisition of a California biologics site aligns with its goals to meet increasing biomanufacturing demands.The industry is also adapting to technological advancements, with AI integration into life sciences commercialization being touted as a frontier for growth. Despite this potential, many organizations remain unprepared to harness AI fully. Leading companies embedding AI solutions aim for measurable outcomes that could significantly drive strategic decision-making and operational efficiencies.Eli Lilly's acquisition of Adverum Biotechnologies aligns with its strategic interests in gene therapy, focusing on promising therapeutic programs that address unmet medical needs. This acquisition centers around Ixo-vec for wet age-related macular degeneration (AMD), highlighting broader industry trends towards investing heavily in innovative therapies that address unmet needs.Conversely, Sanofi's halt on an RSV vaccine development highlights the inherent risks in vaccine development pipelines. Meanwhile, Regeneron's decision to discontinue a CAR T candidate acquired from 2seventy bio showcases ongoing reassessment witSupport the show

In this episode, we speak with Associate Professor Leszek Lisowski, Head of the Translational Vectorology Unit at the Children's Medical Research Institute (CMRI), about the cutting-edge world of gene therapy. He explains how viral vectors are engineered to deliver life-saving treatments for rare genetic diseases - many of which are so uncommon, they're only known by the name of the affected gene. Despite being often overlooked, genetic diseases impact 10% of adults and 30% of children in hospitals. Leszek sheds light on the challenges of diagnosis, the promise of emerging genetic testing, and how his work is paving the way for more accurate, effective therapies. Linkedin: Children's Medical Research Institute, Leszek Lisowski Facebook: @jeansforgenes Instagram: @jeansforgenesau

The journey to a healthier body can sometimes be a struggle. Some foods help, some don't. Certain exercises are supposed to be good but don't show everyone the same rewards. That's where our genes could unlock the path to personalized health.Kyler Ockey shares some of the breakthroughs that Shed is working on to help people individualize and personalize their health journey. Turns out our genes play an important role in how food, exercise, and our environment affect our physical body. Without knowing how to optimize our genes, we could be doing "everything right" and not get the results we want. Kyler Ockey is the Chief Strategy & Product Officer at Shed, a leader in science-backed wellness solutions. Shed created Genetic Testing Kits, designed to help individuals understand how their bodies respond to nutrition, fitness, sleep, stress, and more — and make choices that work with their biology, not against it. Available in four targeted options — Total Wellness, Nutrition + Fitness, Nutrition, and Fitness — the kits provide personalized insights and actionable recommendations for fueling smarter, training effectively, and supporting long-term health. Follow Shed on Instagram @tryshed Visit ConfidenceThroughHealth.com to find discounts to some of our favorite products.Follow me via All In Health and Wellness on Facebook or Instagram.Find my books on Amazon: No More Sugar Coating: Finding Your Happiness in a Crowded World and Confidence Through Health: Live the Healthy Lifestyle God DesignedProduction credit: Social Media Cowboys

What if your skin cells could help you have a baby? Could the future of fertility mean having genetically related children even if a woman is in menopause or lost fertility due to cancer treatments? In this fascinating episode of Brave & Curious, Dr. Lora Shahine sits down with Dr. Paula Amato, reproductive endocrinologist and researcher at Oregon Health & Science University, to unpack the groundbreaking study that made headlines around the world — creating human embryos from skin cells. Dr. Amato explains the science behind this breakthrough, known as in vitro gametogenesis (IVG), and how her team combined genetic material from skin cells and eggs to explore what could one day help people with infertility. She describes the painstaking process of somatic cell nuclear transfer, the ethical oversight behind this research, and the incredible collaboration that led to this first step forward.  Together, Dr. Shahine and Dr. Amato break down complex genetics, discuss the difference between this and “three-parent IVF,” and reflect on what it all means for the future of reproductive medicine. In this episode you'll hear: [2:06] Ripped from the headlines: Human Embryos Made From Skin Cells [4:15] A refresher on chromosomes [7:55] Step-by-step explanation of the science [11:27] Introducing IVG (in vitro gametogenesis ) [15:24] IVG vs IVF [19:38] Who does this benefit? [22:20] How might it change the future? [25:37] Ethical and societal questions [28:03] Balancing progress with responsibility [33:45] What is next in fertility science? Resources mentioned:  Research Study in Nature Communications OHSU Center for Embryonic Cell and Gene Therapy @paula-amato on LinkedIn   Dr. Shahine's Weekly Newsletter on Fertility News and Recommendations Follow @drlorashahine Instagram | YouTube | Tiktok | Her Books

Genetic counsellors Matt and Sandra explore Huntington disease, prenatal and lab-based testing ethics, and how lab genetic counsellors act as gatekeepers. They discuss exclusion testing, the challenges of writing clear lab reports, and recent advances in gene therapy. The episode also covers the growing role of AI and automation in genetic labs, the evolving responsibilities of lab GCs, and how testing impacts life choices like career planning and family decisions.

We're live at BDC!  Host Amy Board take you straight to the exhibit hall floor, chatting with doctors like Drs. Quon, Sidonio, and Radak, plus patient advocates Collin Johnson and Matt Capsel. From man-on-the-street interviews to a touching story from Greg, this episode reminds us all to run—not walk—towards science. Join us for real conversations, patient perspectives, and insights on gene therapy, joint health, and life in the bleeding disorders community. Next up: An inspiring interview with Patty Weltin and the “Beyond the Diagnosis” art exhibit.   Don't miss this insightful, energizing episode that dives into the future of bleeding disorders care.   Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   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. Show Notes:   Subscribe: The BloodStream Podcast   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter  BloodStream on Instagram BloodStream on LinkedIn BloodStream on TikTok  

Program notes:0:40 New drug for SLE1:41 Monitored for a year2:43 Only one pathway limited3:00 AI and health care4:00 How to assure compliance?5:00 Policy changes needed6:01 Pessimistic about oversight6:50 Genetic treatment for ADA deficiency7:50 Using a viral vector8:50 A curative treatment9:30 Social media and cognitive performance in adolescents10:30 Low and high increasing social media use11:30 Good enough evidence12:31 End

From CAR-T therapies to viral vectors, cell and gene treatments are redefining the boundaries of pharmacy practice—but with innovation comes complexity. Host Carolyn Liptak welcomes Dr. Mark Wiencek, Principal Microbiologist with the Technical Services Group at Contec, and Dr. Amanda Frick, Senior Clinical Manager of Market Intelligence at Vizient, to break down the challenges of compounding these advanced therapies.   Listen in as they discuss real-world risk assessments, biosafety considerations, and how hospital pharmacies can safely manage these groundbreaking yet high-risk treatments.   Guest speakers:  Mark Wiencek, PhD Principal Microbiologist, Technical Services Group Contec Amanda Frick, PharmD, BCPS Senior Clinical Manager, Market Intelligence Vizient   Host:  Carolyn Liptak, MBA, RPh  Pharmacy Executive Director  Vizient   Show Notes:  [01:02-01:51] Mark shares his background and experience in microbiology [01:52-04:04] Overview of the types of cell and gene therapies (CGT) currently used in clinical practice [04:05-05:14] Which CGT therapies are most applicable to pharmacy compounding and why [05:15-10:29] Things not on the NIOSH list and the risks [10:30-12:03] Evaluating whether viral vectors can penetrate intact skin and the true occupational exposure risks [12:04-13:18] If hazards are not defined by the NIOSH list, how should these CGT hazards be classified [13:19-15:03] Determining the safest environment for compounding CGT therapies [15:04-20:14] Best practices for decontamination, disinfection, and viral vector handling [20:15-20:59] Do you need a dedicated biosafety cabinet for CGT therapies [21:00-22:55] Recommended resources for further learning   Links | Resources:   Blind and colleagues (Nationwide): Click here Wang and colleagues (Stanford): Click here CONTEC HEALTHCARE WEBINAR Using Bugs as Drugs: Compounding Viral Vectors in Cell & Gene Therapy for Hospital Pharmacies, Mark Wiencek, May 13, 2025: Click here Blind, J.E., Ghosh, S., Niese, T.D., Gardner, J.C., Stack-Simone, S., Dean, A. and Washam, M., 2024. A comprehensive literature scoping review of infection prevention and control methods for viral-mediated gene therapies. Antimicrobial Stewardship & Healthcare Epidemiology, 4(1), p.e15. Click here Deramoudt, L., Pinturaud, M., Bouquet, P., Goffard, A., Simon, N. and Odou, P., 2024. Method for the detection and quantification of viral contamination during the preparation of gene therapy drugs in a hospital pharmacy. Occupational and Environmental Medicine, 81(12), pp.615-621. Click here Korte, J., Mienert, J., Hennigs, J.K. and Körbelin, J., 2021. Inactivation of adeno-associated viral vectors by oxidant-based disinfectants. Human Gene Therapy, 32(13-14), pp.771-781. Click here (abstract only; full article available for purchase) Martino, J.G., McConnell, K., Greathouse, L., Rosario, B.D. and Jaskowiak, J.M., 2024. Cellular therapy site-preparedness: Inpatient pharmacy implementation at a large academic medical center. Journal of Oncology Pharmacy Practice, 30(8), pp.1442-1449. Click here Penzien, C., 2023. Safe handling of BioSafety drugs and live virus vaccines. Pharm Purch Prod, 20(4), p.12. Click here Petrich, J., Marchese, D., Jenkins, C., Storey, M. and Blind, J., 2020. Gene replacement therapy: a primer for the health-system pharmacist. Journal of Pharmacy Practice, 33(6), pp.846-855. Click here Wang, A., Ngo, Z., Yu, S.J. and MacDonald, E.A., 2025. Implementing standard practices in the safe handling of gene therapy and biohazardous drugs in a health-system setting. American Journal of Health-System Pharmacy, p.zxaf026. Click here   VerifiedRx Listener Feedback Survey: We would love to hear from you - Please click here   Subscribe Today! Apple Podcasts Spotify YouTube RSS Feed

When baby KJ was diagnosed with a rare metabolic disorder, the team at Children's Hospital of Philadelphia (CHOP) had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On this episode, Madeline speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ's first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients. 

What if solving tomorrow's bioprocessing challenges meant questioning the very physics of chromatography and reimagining downstream processing from the ground up?For years, large biomolecules like viral vectors and exosomes have squeezed through the limitations of traditional chromatography, leaving scientists to wrestle with capacity trade-offs and slow mass transfer. But what if a fundamental shift could unlock breakthroughs for gene therapies, vaccines, and advanced biologics?In this thought-provoking episode, host David Brühlmann sits down with Alois Jungbauer, professor emeritus of downstream processing at BOKU University (Vienna) and scientific advisor at BioChromatographix International. Together, they examine the future of chromatography and downstream processing, exploring innovations that tackle the challenges of modern gene therapy, advanced therapeutics, and sustainability in manufacturing.Alois shares his perspective on anticipating industry needs, the importance of mentorship, and why the physical limits for purification of large biomolecules are yet to be reached.Episode Highlights:The difference between solving current problems and anticipating the needs of the next generation in biotech (00:00)The promise and technical details of monolithic chromatography and its application for large modalities like gene therapy vectors and exosomes (03:01)Explanation of “inverted morphology" and AXISFLOW™ in new chromatography materials (03:18)Challenges and opportunities in continuous gene therapy vector production, and why it's not widespread yet (07:40)The critical role of sustainability in downstream processing, particularly water usage and its impact on the viability and costs of distributed manufacturing (10:30)The relationship between reducing water consumption, manufacturing footprint, and cost of goods (12:16)Advice on scientific career development: listening, reading, understanding future industry needs, finding a mentor, and having persistence (13:50)Broader reflections on the value of science, societal perceptions, and science communication in today's world (17:34)Alois's key takeaway: downstream processing for gene therapy has not yet reached its physical limits—there is still significant room for improvement (20:26)If you're looking for fresh strategies to tackle the bottlenecks of large-molecule purification - and a candid perspective on where the real opportunities and societal responsibilities lie - you won't want to miss this conversation.Connect with Alois Jungbauer at leading conferences, including Bioprocessing Summit and Bioprocessing International, or find the next integrated continuous biomanufacturing event where he's co-chairing.Connect with Alois Jungbauer:LinkedIn: www.linkedin.com/in/alois-jungbauer-14984811Website : www.biochromatographix.comNext step:Book a 20-minute call to help you get started on any questions you may have about bioprocessing analytics: https://bruehlmann-consulting.com/callPreparing for your IND? We're building a CMC Dashboard in Excel to help biotech founders track tasks, timelines, and risks in one place. Join the waitlist for early access at

Ladies, it's Breast Cancer Awareness Month, so go get your boobies checked! We're also talking family lineage, growing up on a farm, and the groundbreaking treatment for Huntington's disease.00:00:00 - Introduction & Getting Started 00:02:04 - Fall Season & Halloween Decorations Debate 00:05:27 - Big Tea & Do Dog (Parent Nicknames) 00:15:27 - Big Family Reflections 00:20:08 - Family Genealogy & Lineage00:24:11 - Pop Culture & Science Discussion00:30:16 - Gene Therapy & Medical Research 00:33:07 - Eric Dane & ALS Awareness 00:37:11 - mRNA Vaccine Technology 00:38:36 - Wrapping Up & Superpower TalkMORE PODCAST EPISODES: https://youtube.com/playlist?list=PLTGuNbPgq2EartAwwgs_H-LVho3FvWnXpJUST LISTEN TO THE PODCAST: https://link.chtbl.com/imomsohardSEE US ON TOUR: (FALL DATES COMING SOON)https://www.imomsohard.com/WATCH OUR AMAZON PRIME SPECIAL: https://www.amazon.com/IMomSoHard-Live/dp/B07VBJ34DTIf you are interested in advertising on this podcast email ussales@acast.comTo request #IMOMSOHARD to be on your Podcast, Radio Show, or TV Show, reach out to talent@pionairepodcasting.comFOLLOW US: Facebook: https://www.facebook.com/imomsohardInstagram: https://www.instagram.com/imomsohard/Twitter: https://twitter.com/imomsohardGet our sponsor DISCOUNT CODES here!https://linktr.ee/imshpodcastABOUT US Female comedy duo Kristin Hensley and Jen Smedley have been performing, teaching, and writing comedy internationally for a combined 40+ years. They have been moms for one quarter of that time and it shows. How do they cope? They laugh about all of the craziness that comes with being a mom and they want you to laugh about it too! From snot to stretchmarks to sleepless nights, Kristin and Jen know firsthand that parenting is a hard job and they invite you to join them in taking it all a little less seriously (even if for a few short minutes a day). After all, Jen currently has four days of dry shampoo in her hair and Kristin's keys are still in her front door. They try, they fail, they support each other, and they mom as hard as they can.Disclaimer: This podcast is for entertainment purposes only. Views expressed on this podcast solely reflect those of the host and do not reflect the views of Pionaire. Hosted on Acast. See acast.com/privacy for more information.

In this episode, host David Brühlmann is joined by Alois Jungbauer, Professor Emeritus at BOKU University, Vienna, and Scientific Advisor at BioChromatographix International. With over 40 years in bioprocess engineering and more than 400 published papers, Jungbauer offers a unique perspective on how downstream processing and purification technologies have evolved and where they're headed next.Alois shares his unconventional journey into the world of biotech, starting from humble beginnings on an Austrian farm, and discusses key turning points that have shaped the industry. Together, they explore the latest challenges triggered by high cell culture titers, new therapeutic modalities, and the drive for automation and process integration in manufacturing.In this episode:Why the gap between upstream and downstream processes remains a core bottleneck (03:27)How rigid and convective chromatography materials changed throughput and efficiency (06:56)The shift in purification strategies with skyrocketing titers in cell culture (08:43)Automation and integrated biomanufacturing: their role in making processes more robust and continuous (10:20)Misconceptions around resin costs in full-scale versus clinical-scale manufacturing (12:10)What automation and process monitoring mean for real-time release and factory scheduling (14:05)The resurgence and application of displacement chromatography for gene therapy vectors (15:49)Whether you're deep into process development or just curious about the next wave in biotech manufacturing, Alois Jungbauer's insights offer valuable context for the road ahead.Connect with Alois Jungbauer:LinkedIn: www.linkedin.com/in/alois-jungbauer-14984811Website : www.biochromatographix.comNext step:Book a 20-minute call to help you get started on any questions you may have about bioprocessing analytics: https://bruehlmann-consulting.com/callPreparing for your IND? We're building a CMC Dashboard in Excel to help biotech founders track tasks, timelines, and risks in one place. Join the waitlist for early access at https://scale-your-impact.notion.site/27dd9c6ba679804b80a7ce439d56c91a?pvs=105

Drs. Marianeli Rodriguez and Kat Talcott join to discuss the October 2025 edition of Retinal Physician, including inherited retinal disease treatment pipeline, gene therapy, and systemic therapy for sickle cell disease and retinopathy.

On "This Week in Pharmacy" for September 26th, we're talking about Trust in Healthcare, World Pharmacists Day, and Cell & Gene Therapy. Today's co-host is Dr. Melissa Smith, PharmD CPH with Florida PGx Consulting LLC. https://www.floridapgxconsulting.com/  First, #TWIRx News - the FDA approves Inluriyo imlunestrant on 9/25/2025 which is to treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, estrogen receptor-1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. (https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025)  Second, Nested-Knowledge - is going to revolutionize information access and leverage. This is a powerful tool with evidence synthesis tools for medical researchers. This will help accelerate, collaborate, automate and share the latest and personalized treatment plans. (https://nested-knowledge.com/) Next, announcing "Evidence based Podcasting", the new standard in audio learning for Providers and next level in podcast Continuing Medical Education (CME). (https://pharmacypodcast.com/2025/09/26/evidence-based-podcasting-and-the-future-of-medical-education/ ) Shout out to Nick Calla with Orsini, a leader in rare disease pharmacy solutions, has been chosen by Crinetics Pharmaceuticals, Inc., as a specialty pharmacy partner for PALSONIFY™ (paltusotine), a nonpeptide, selectively-targeted somatostatin receptor type 2 nonpeptide (SST2) agonist. (https://www.wvnews.com/news/around_the_web/partners/pr_newswire/subject/licensing_marketing_agreements/orsini-selected-as-specialty-pharmacy-partner-for-crinetics-palsonify-paltusotine/article_9b42bfe7-69a6-58be-98ff-5e2245145415.html) Today we have two guest Pharmacists to dig into the "Trust in Healthcare" theme of today's #TWIRx -- the godfather of Functional Pharmacy Robert Kress RPh., and Dr. Lauren Castle, PharmD, MS, AFMC - Founder & CEO of Functional Medicine Pharmacists Alliance. Who do you trust? What information is trustworthy and why?  Our special feature is updates on 'Cell & Gene Therapy' with McKesson's Head of Cell Gene Advanced Therapies, Joe DePinto at InspiroGene!  (https://inspirogene.com/)  Today's TWIRx is sponsored by 'Sykes & Company' the Pharmacy Accounting Jedi Masters and Independent Pharmacy Cooperative (IPC) and how they're revolutionzing digital health and new revenues with the iCare+ System!    Trust Matters, Cell and Gene, & World Pharmacists Day | TWIRx

My guest is Dr. Sergiu Pașca, MD, professor of psychiatry and behavioral sciences at Stanford University. We discuss the biology and genetics of autism, why autism diagnoses are increasing and recent progress in using stem cells to understand and treat profound autism and other brain disorders. Dr. Pașca explains “organoids and assembloids”—human stem cell–derived tools he pioneered to study, treat and cure complex brain diseases. We also discuss ethical and safety issues with using gene editing and stem cells in humans. Read the episode show notes at hubermanlab.com. Thank you to our sponsors AG1: https://drinkag1.com/huberman David: https://davidprotein.com/huberman Helix: https://helixsleep.com/huberman BetterHelp: https://betterhelp.com/huberman Function: https://functionhealth.com/huberman Timestamps (00:00) Sergiu Pașca (02:08) Autism Spectrum Disorder, Incidence, Genetics (07:16) Is Autism More Common in Males? (09:35) Sponsors: David & Helix Sleep (11:56) Eye Contact in Babies, Fever; Proposed Causes of Autism; Genes (18:48) Genetic or Idiopathic Autism Diagnoses, Timothy Syndrome (21:37) Rise in Autism Diagnoses (26:46) Cause, Correlation & Neurological Disease; Schizophrenia, Do Vaccines Cause Autism? (31:34) Global Increase in Autism; Gene Therapy, CRISPR, Follistatin (41:05) Sponsors: AG1 & BetterHelp (43:41) Stem Cells, Ethics, Yamanaka Factors, Human Stem Cell Models (52:03) Umbilical Stem Cells; Stem Cell Injections & Dangers, Autistic Kids (59:30) Organoids, Modeling Brain Development, Intrinsic Development Timer (1:12:22) Assembloids, Brain Cell Migration & Circuit Formation, Self-Organization (1:21:22) Four-Part Assembloid, Sensory Assembloid, Pain Conditions (1:25:45) Sponsor: Function (1:27:33) Future Medical Therapies, Cell Banking, Immortalize Tissues, Rejuvenate Cells (1:34:56) Assembloids & Ethics, Importance of Nomenclature, Science Collaboration & Self-Correction (1:45:38) Cell Transplantation & Ethics, Timing (1:55:05) Genetic Testing for Parents, Genetic Penetrance (2:02:36) Assembloids, Timothy Syndrome, Epilepsy, Schizophrenia, Dystonia (2:14:30) Scientific Career, Walking, Art, Medical School (2:20:44) Zero-Cost Support, YouTube, Spotify & Apple Follow & Reviews, Sponsors, YouTube Feedback, Protocols Book, Social Media, Neural Network Newsletter Disclaimer & Disclosures Learn more about your ad choices. Visit megaphone.fm/adchoices