Can rewriting DNA change the future for families living with rare genetic disease? Dr. David Liu joins Ron Kleiman to discuss base editing, prime editing, patients already benefiting from these technologies, and the work needed to reach more families.Recorded in Boston the day after Prime Medicine's October 5, 2026 announcement that the first patient had been dosed in its Phase 1/2 trial of PM577a, an investigational in vivo prime-editing treatment for Wilson disease.David's laboratory pioneered base editing and prime editing. He is a Harvard professor, director of the Merkin Institute for Transformative Technologies in Healthcare at the Broad Institute, a Howard Hughes Medical Institute investigator, and a co-founder of Prime Medicine. His honors include the 2025 Breakthrough Prize in Life Sciences and the 2026 Franklin Institute Bower Award and Prize for Achievement in Science.In this conversation:• How base editing and prime editing work, and why correcting DNA is different from disrupting a gene.• Patient stories involving leukemia, chronic granulomatous disease, sickle cell disease, and Baby K.J.'s personalized treatment.• Prime Medicine's Wilson disease milestone and why clinical results are the next important step.• Newborn sequencing, early diagnosis, and the Center for Therapeutic Genetics.• Research toward gene editing in the brain for Dravet syndrome and alternating hemiplegia of childhood (AHC).• How AI and OptiPrime help researchers design and improve gene-editing tools.David identifies 32 base- or prime-editing clinical trials at the time of this interview. Treatments discussed are at different stages of research and clinical development; animal results and early patient outcomes do not establish effectiveness for every patient or condition.GENEration Hope brings families conversations and breakthrough news at the intersection of rare disease, genetic medicine, and AI.Subscribe, like, and share to help these conversations reach more families. Visit https://generationhope.co for more interviews, our newsletter, and weekly video updates highlighting the most important breakthrough news.Listen to the GENEration Hope podcast on Apple Podcasts, Spotify, iHeartRadio, Amazon Music, or wherever you get your podcasts.CHAPTERS00:00 Highlights: gene editing reaches patients00:45 Introducing Dr. David Liu03:01 From chemistry to genetics06:21 Why cutting DNA is not enough07:59 How base editing began10:31 Evolving the adenine base editor12:21 Prime editing: search and replace for DNA15:39 Prime Medicine news and 32 clinical trials17:54 Alyssa: the first base-edited patient19:19 Cholesterol, PCSK9 and disease prevention22:01 Protective mutations and the wider potential of editing24:25 Correcting a disease-causing mutation: BEAM-30225:37 Prime editing for chronic granulomatous disease28:18 Base editing for sickle cell disease29:19 Baby K.J. and a personalized genetic medicine32:22 Newborn sequencing and actionable findings34:14 The Center for Therapeutic Genetics35:38 In vivo prime editing for Wilson disease36:58 Gene editing in the brain: Dravet syndrome and AHC40:35 Making genetic medicines repeatable and scalable41:15 Regulatory pathways and treatment access44:59 AI, OptiPrime and the future of discovery51:25 Closing and more from GENEration HopeLEARN MOREDavid Liu / Broad Institute: https://www.broadinstitute.org/bios/david-liuPrime Medicine's Wilson disease announcement: https://investors.primemedicine.com/news-releases/news-release-details/prime-medicine-announces-first-patient-dosed-global-phase-12Center for Therapeutic Genetics: https://sites.broadinstitute.org/ctgOptiPrime: https://www.broadinstitute.org/news/ai-model-streamlines-prime-editing#GeneEditing #RareDisease #GENErationHope

Update for the week of September 28, 2026A historic week for rare disease families — Sanfilippo syndrome just got its first-ever approved treatment, the Angelman community has a bold new gene therapy program entering the pipeline, and an ASO milestone is bringing real hope to FUS-ALS families. But approval doesn't always mean access, and we're not shying away from that conversation.**In this week's update:**- [0:00] Intro & what's covered this week- [0:45] FDA approves Fayuvi — the first-ever gene therapy for Sanfilippo Syndrome Type A- [2:10] The $3.95M price tag: what it means for families trying to access this treatment- [3:20] Skylark Bio enters the Angelman arena with a new UBE3A restoration program- [4:35] Ionis reports positive Phase 3 results for ulefnersen in FUS-ALS- [5:40] Claude AI discovers a novel enzyme system — and what that could mean for biology- [6:30] Closing thoughts & where to find usGENEration Hope is a documentary and media platform covering rare genetic disorders, gene therapy, and the families navigating this world. Created by Ron Kleiman, a filmmaker and rare disease dad whose daughter Evie has Phelan-McDermid Syndrome. We cover what matters to families — not just investors.Subscribe and hit the bell 🔔 for weekly genetic medicine updates.**Find us here:**🌐 Website: https://generationhope.co📺 YouTube: @GENErationHope_1📸 Instagram: @GenerationHopeCo**Sources:**🔗 FDA Approval — Fayuvi / Sanfilippo Type A: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type🔗 Ultragenyx Price Announcement: https://www.cnbc.com/2026/09/18/ultragenyx-sets-3point95-million-price-for-rare-disease-gene-therapy.html🔗 Skylark Bio / Angelman Pipeline: [Business Wire link]🔗 Ionis / FUS-ALS Phase 3 Results: https://ir.ionis.com/news-releases/news-release-details/ionis-announces-positive-topline-results-phase-3-fusion-study🔗 Claude AI Enzyme Discovery: https://www.anthropic.com/news/claude-discovers-novel-enzyme-system---```0:00 Intro — A Historic Week for Rare Disease Families0:45 FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A2:10 The $3.95 Million Question: Who Can Access Fayuvi?3:20 Skylark Bio Enters the Angelman Arena with New UBE3A Program4:35 Ionis Phase 3 Win: A Meaningful Milestone for FUS-ALS Families5:40 Claude AI Discovers a Novel Enzyme System6:30 Closing Thoughts — Till Next Time, Stay Hopeful```

In this GENEration Hope interview, Ron Kleiman speaks with Dr. Allyson Berent — rare disease mom, veterinarian, interventional specialist, researcher, biotech founder, and Chief Science Officer of FAST, the Foundation for Angelman Syndrome Therapeutics.After her daughter Quincy was diagnosed with Angelman syndrome, Allyson became one of the leading voices in parent-led drug development. Through FAST, she helped drive research programs that have moved from family urgency to real clinical development, including GTX-102, which is now in a fully enrolled global Phase 3 study, with data expected in the second half of 2026.In this conversation, we talk about Angelman syndrome, FAST, GTX-102, AS2 Bio, gene-targeted therapies, the realities of building treatments for rare diseases, and what happens when families refuse to wait for someone else to move the science forward.We also discuss the broader future of rare disease treatment — including ASOs, gene replacement, gene editing, stem-cell-based approaches, clinical trials, access, urgency, and the role families play in changing what is possible.If conversations like this are important to you, please subscribe, like, and share. It is the best way to support GENEration Hope and help us reach more families, researchers, clinicians, and advocates.Visit GENErationHope.co to watch more interviews, sign up for the newsletter, and follow our weekly updates on gene therapy, RNA therapy, newborn screening, clinical trials, and rare disease research.Subscribe to GENEration Hope on YouTube for more interviews and weekly updates.Disclaimer: This interview is for educational and informational purposes only and should not be taken as medical advice. Families should speak with their own physicians, specialists, or clinical trial teams about any treatment or research questions.#AngelmanSyndrome #RareDisease #GeneTherapy #RNATherapy #GTX102 #FAST #GenerationHope