Gene Therapy
44 videos · last 6 months shuffled daily, then older videos newest first
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21:15 A New Era for FOXG1: The Race to First‑in‑Human Trials
FOXG1 Research Foundation CEO Nasha Fitter on FDA Fast Track designation, IND clearance, and the first clinical trial of a FOXG1 gene therapy.
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22:15 Delivering Advanced Neurotherapy Trials
Professor Liam Gray, director of the BRAIN Unit, and Professor Anne Rosser of Cardiff University discuss delivering trials of gene, RNA and cell therapies for Huntington's and Parkinson's diseases, for the Centre for Trials Research.
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1:02:09 Cystic Fibrosis State of the Science: Emily's Entourage
Emily's Entourage's scientific team reviews the research and drug development landscape for the final 10% of people with cystic fibrosis who do not benefit from mutation-targeted therapies, and presents its new Research Roadmap.
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59:57 Congressional Briefing on Gene Therapy for N-of-1 Diseases
ASGCT's Congressional briefing brings together academic researchers, industry leaders and patient advocates on cell and gene therapy and platform-based development for ultrarare and N-of-1 genetic diseases.
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57:55 Neurogene Update on NGN-401 Gene Therapy for Rett
Rachel McMinn, PhD, Founder and CEO of Neurogene, presents updates on NGN-401, its investigational gene therapy for Rett syndrome, covering the Phase 1/2 program and the fully dosed Embolden Phase 3 trial, for the International Rett Syndrome Foundation.
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4:02:40 Engaging Stakeholders in Cell and Gene Therapy Research
ASGCT and NORD host a workshop at the ASGCT 2026 Annual Meeting on engaging patients and other stakeholders in cell and gene therapy translational research.
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3:45:23 Cell and Gene Therapy Development: Regulatory Considerations
A Friends of Cancer Research meeting covers regulatory and operational considerations in cell therapy development, modernising early-phase clinical development, risk-based CMC flexibility and predictability in drug development policy.
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8:52 Full Interview with Dr. Shannon Boye from Atsena Therapeutics
Dr. Shannon Boye, Founder and Chief Scientific Officer at Atsena Therapeutics, on her background and the company's two ongoing clinical trials, recorded at the Visions 2026 conference.
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33:57 Sickle Cell Gene Therapy and Trials: Patient Session
Suzi Lowi, a clinical trials research nurse at Imperial College Healthcare NHS Trust, explains what is involved in gene therapy at a West London HCC sickle cell patient event, with audience questions.
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1:06:09 Affinia BAG3 DCM - UPBEAT Clinical Trial Webinar Recording 6.10.26
The Genetic Cardiomyopathy Awareness Consortium on AFTX-201, an investigational one-time gene therapy for dilated cardiomyopathy caused by BAG3 mutations, and the UPBEAT trial.
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37:41 Wilson Disease Symposium: Clinical Trials and Gene Therapy
The second session of the Wilson Disease Association and University of Michigan 2026 Spring Support Symposium covers improving neurological symptoms, understanding clinical trials, working with a trial support team, and gene therapy clinical trials.
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55:59 Gene Therapies Demystified: Policy, Access, and the Road Ahead
John Feore, Director of Health Policy & Advocacy for the Institute for Gene Therapies, reviews gene therapy policy at the FDA, HHS and Congress and how patient groups work on access, for the CMT Research Foundation.
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14:42 SCGE Resources: Gene Therapy Trial Browser and IND Toolkit
Karen Clark, PhD, a Research Scientist at the Medical College of Wisconsin, describes free resources on the SCGE platform, including a gene therapy clinical trial browser, shared regulatory documents and an IND application toolkit for gene therapy.
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39:17 FRF-001 Gene Therapy Trial Update, June 2026
The FOXG1 Research Foundation's gene therapy team updates the FOXG1 community on the first-in-human FRF-001 trial, covering how enrollment works, what happens at each stage and frequently asked questions.
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36:16 The Science Behind Parkinson's Clinical Trials, Part 1
Dr. Kevin McFarthing, a biochemist and expert in Parkinson's clinical trials, joins the APDA to explain the biology behind therapies in Parkinson's trials, from gene therapies and antisense oligonucleotides to cell-replacement and antibody therapies.
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40:08 Stevie Ringel of Nome on AI in Ultra-Rare Drug Development
Stevie Ringel, co-founder of Nome and founder of the Kizuna Foundation, tells The Genetics Podcast how AI agents handle the operational work of small-batch ultra-rare drug development, and discusses FDA signals and funding models.
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42:26 Solid Biosciences: FALCON SGT-212 Phase 1b Update
Solid Biosciences' Matthew Harmelink, MD, Therapeutic Area Head of Neuromuscular, and Seth Rotberg, Senior Manager of Patient Advocacy, update the Friedreich's Ataxia Research Alliance community on the FALCON phase 1b study of the gene therapy SGT-212.
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9:34 Long Term Followup of Intravitreal Gene Therapy for Neovascular AMD
Retina Synthesis discusses three-year results from the Phase 2 trial of Adverum's intravitreal gene therapy with Dr. Dilsher Dhoot of California Retina Consultants.
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15:25 Optogenetics for Treating Inherited Retinal Degenerations
Retina Synthesis discusses Phase 2 results from Nanoscope regarding the use of optogenetics for treatment of retinal degenerations with Allen Ho, MD, Professor of Ophthalmology, Wills Eye Hospital.
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10:42 Interview with Dr. Mark Pennesi of the Retina Foundation - Visions 2026 Conference
Dr. Mark Pennesi of the Retina Foundation, an inherited retinal degeneration specialist who both treats patients and runs trials, interviewed at the Visions 2026 conference.
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3:27 Gene Therapy Weekly: FOXG1's First Trial Site Is Open
GENEration Hope reports that the FOXG1 gene therapy trial has activated its first clinical site, at Texas Children's Hospital and Baylor College of Medicine.
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1:10:01 Expanding Access to Cell and Gene Therapies Across Africa
Speakers from Makerere University, Moi University and Benjamin Mkapa Hospital describe the regulatory, logistical and infrastructure challenges of establishing cell and gene therapy programmes in African countries, in an ASGCT webinar.
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4:08 First Patient Dosed in FOXG1 Gene Therapy Trial
The FOXG1 Research Foundation announces that the first patient has received FRF-001, its investigational FOXG1 gene replacement therapy, in a first-in-human clinical trial the foundation sponsors.
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1:56 FDA Approval of a Duchenne Gene Therapy Developed at Nationwide
Nationwide Children's Hospital reports FDA approval of a gene therapy for Duchenne muscular dystrophy developed there, with principal investigator Jerry Mendell, MD, describing the single-infusion clinical trial in boys with DMD.