GENEration Hope

GENEration Hope - Ron Kleiman
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In-depth conversations with scientists, clinicians, advocates, and families at the front lines of rare disease, gene therapy, and genomic medicine. Hosted by rare disease parent and filmmaker Ron Kleiman, GENEration Hope explores the science, the ethics, and the human stories behind the fight to give children with genetic disorders a better future.

Episódios

  1. há 4 dias

    Allyson Berent on Angelman Syndrome, FAST, GTX-102 & Parent-Led Drug Development

    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

    1h 29min

  2. 5 de mai.

    How RNA Therapies Are Changing Duchenne | Dr. Yoshitsugu Aoki

    Dr. Yoshitsugu Aoki is a physician-scientist at the forefront of RNA therapeutics and genetic medicine.As Director of the Department of Molecular Therapy at the National Center of Neurology and Psychiatry in Tokyo, Dr. Aoki has helped lead the development of antisense oligonucleotide (ASO) therapies for Duchenne muscular dystrophy—including viltolarsen, an exon-skipping therapy approved in both Japan and the United States.In this conversation, we explore how RNA therapies became a reality, what they’re doing for patients today, and where the field is heading next.⸻🔬 What we cover: What exon skipping is (in simple terms)How ASO therapies are helping Duchenne patientsBreakthrough clinical results and what they meanDelivering therapies to the brainThe future of RNA, gene therapy, and combination treatmentsHow AI and robotics could accelerate drug development⸻🌐 GENEration HopeI’ve launched the new GENEration Hope website along with a newsletter and weekly news updates covering the latest breakthroughs in genetic medicine.👉 Visit: generationhope.co👉 Subscribe to stay up to date on what’s coming nextIf you find this valuable, liking and subscribing really helps support the project.⸻⚠️ DisclaimerThis content is for informational purposes only and is not medical advice.

    37 min

  3. 8 de abr.

    Dr. Guoping Feng (MIT) on SHANK3 Gene Therapy, Brain Disorders, and What’s Coming Next

    Welcome — I’m Ron Kleiman, and this is GENEration Hope.In this episode, I’m joined by Dr. Guoping Feng, Professor of Neuroscience at MIT, affiliated with the McGovern Institute, the Yang Tan Collective, and the Broad Institute. We talk about: • Why he left medicine to pursue research that could lead to treatments for kids  • The urgency of moving faster — because “kids are growing up every day”  • Gene therapy delivery, the blood–brain barrier, and what’s changing with new vectors  • How AI and machine learning are speeding up vector design and behavioral testing  • The miniSHANK3 approach: why the full SHANK3 gene is too large, how a mini gene is designed, and how it’s delivered  • Where genome editing (base editing / prime editing) may fit in the future If conversations like this help, please subscribe, like, and share — it’s the best way to support GENEration Hope. This content is for informational purposes only and is not medical advice.Chapters00:00 Cold open: the urgency + “that’s our hope” 01:07 Intro: state of genetic medicine (April 2026) + guest setup 04:29 Who Guoping is (MIT / Yang Tan Collective) + mission 04:54 Hangzhou med school → why he shifted to research 06:12 From fruit flies to mouse models (Drosophila → mammals) 06:40 PhD at SUNY Buffalo + moving into mouse genetics 07:20 Postdoc at WashU + “benefit patients” as the goal 07:52 Conditions his lab targets: PMS, Rett, SYNGAP1, Dravet 09:23 Can AI speed discovery? What actually moves things faster 10:15 Donors + centers at MIT + enabling translation to clinic 11:26 “Kids are growing up every day” + the role of young scientists 15:20 Why primate studies matter + safety confidence 16:25 Ben Deverman vectors + BBB is species-specific 17:39 ICV vs IV delivery + combining gene therapy with better capsids 20:14 AI protein design + ML behavior tracking + trial design 22:25 Lisa Yang’s story + SHANK3 connection 25:01 Why rare monogenic disorders need resources + “technical problem” 28:50 What a treatable world could look like for families 31:36 Brain plasticity + what changes might look like over time 32:46 Why foundations matter + speeding recruitment and development 36:20 Mini gene + safer genome editing (base editing) 40:30 Timeline: getting things into clinic (optimism + under 5 years) 41:53 Jaguar license: miniSHANK3 and why SHANK3 is too big 42:25 How they chose what to keep/remove in miniSHANK3 44:48 Why not split into two vectors? the delivery problem 46:33 Base editing limits + prime editing + Prime Medicine 48:54 Who owns IP at MIT/Broad + licensing basics 50:29 Explain it simply: what vectors are + how AAV9/ICV works 53:04 CSF basics + neuron counts + how much coverage is needed 55:26 Distribution challenges + future vectors + re-dosing & antibodies 58:13 Wrap-up + thanks + outro 

    59 min

  4. 2 de mar.

    How a Parent-Led Movement Grows: Sue Lomas (Phelan-McDermid Syndrome Foundation)

    Sue Lomas helped build the Phelan-McDermid Syndrome community in the earliest days—when families were scattered, information was scarce, and finding “your people” took persistence. In this conversation, Sue and I talk about how rare-disease communities form, why early diagnosis and genetics matter, and how parent-led foundations can accelerate research by connecting families, clinicians, and scientists.We also explore the emotional side of the journey: the turning points that reshape what you believe is possible, the role of family support behind the scenes, and what purpose looks like after stepping away from a long mission.If you’re a rare disease parent, advocate, researcher, or someone who cares about the future of gene-based medicine—this one is for you.If this resonates, please like, subscribe, and share it with a family who needs hope today.Chapters 00:00 Cold Open: A moment that captures the mission01:33 Connecting with other foundations (learning by reaching out)03:42 Turning point: realizing more is possible (communication)06:40 The feeling of acceleration in research and progress09:53 The early days: “by luck” and finding the right people11:49 First conferences and building real community13:49 The support system: partnership and what it takes16:19 Early diagnosis & genetics (why it matters)19:09 Growing a foundation while staying cohesive22:03 “Shank3” in a classroom: when awareness spreads25:08 How this journey changes parents over time29:43 Advice for parents: planning, siblings, and support33:17 Retirement, purpose, and what comes next36:50 Final reflections and gratitude

    40 min

  5. 1 de fev.

    Ep 5. Dr. Katy Phelan: The Origins of Phelan-McDermid Syndrome + Why Hope Is Growing

    Welcome to GENEration Hope — I’m Ron Kleiman.Today’s guest is Dr. Katy Phelan, a pioneering human geneticist and one of the founders of the Phelan-McDermid Syndrome Foundation (PMSF). This conversation was recorded at the PMSF Annual Conference (2024).Dr. Phelan takes us back to the earliest days of how Phelan-McDermid syndrome was first identified, how families found each other long before social media, and why community became a turning point for so many parents.We also break down diagnostics in plain language — including microarray, sequencing, and why chromosome analysis can still matter — and we talk about what’s giving families real hope as research and treatments move closer.If conversations like this are helpful, please subscribe and turn on notifications so you’re alerted each month when a new interview drops, along with updates from GENEration Hope.Stay Hopeful,Ron⸻Chapters (paste into YouTube)00:01:21 Who is Dr. Katy Phelan (geneticist + PMSF founder)00:01:44 “Girls aren’t built for math” — and proving that wrong00:03:17 Why human genetics (and the Celtics + PMS colors story)00:04:39 The first 22q13 deletion case and the start of collaboration00:06:04 Community and siblings00:07:41 How close are we to treatments? (optimism + perspective)00:09:26 Microarray explained (and what it can’t tell you)00:10:22 Why chromosome analysis still matters (ring chromosomes)00:12:10 Publishing → families calling → learning from parents00:13:05 “They wanted a name like Down syndrome”00:14:26 The first meeting (grant, logistics, and rapid growth)00:18:03 Early whole genome sequencing + diagnostic guidelines00:20:11 US insurance vs Canada timelines/access realities00:21:34 Gene therapy: what families worry about most00:24:38 Misdiagnoses and missed chromosome tests00:29:37 Toronto: why SHANK3 variants may be undercounted00:31:38 Shank3-related vs Shank3-unrelated PMS (inclusion)00:35:41 Deletions vs variants: what it can mean clinically

    39 min

  6. 6 de jan.

    Inside the Fight to Cure Rett Syndrome – Monica Coenraads Interview

    Monica Coenraads, founder and CEO of the Rett Syndrome Research Trust (RSRT), has led the charge to cure Rett Syndrome—raising over $123 million and driving multiple gene therapy programs now in clinical trials.In this in-depth conversation, Monica shares her personal journey from her daughter Chelsea’s diagnosis to building one of the world’s most impactful rare disease research organizations. She explains the evolution of Rett research—from the discovery of MECP2 to today’s cutting-edge genetic medicines including gene therapy, base and prime editing, and RNA trans-splicing.We discuss: • The founding of RSRT and early breakthroughs • The reversibility discovery that changed everything • The Taysha and Neurogene gene therapy trials • The future of Rett research (Roadmap to Cures 2.0) • How parents can catalyze scientific progress • The power of early diagnosis and newborn screeningStay tuned after the interview for updates on the latest Rett, STXBP1, and Phelan-McDermid Syndrome gene therapy trials.👉 Subscribe for more interviews with scientists, researchers, and advocates advancing gene therapy for rare genetic disorders.🌐 Visit GENErationHope.co for global updates and resources.🕒 Chapters / Timestamps00:00 – Introduction00:28 – About Monica Coenraads & RSRT01:53 – Chelsea’s Diagnosis and Early Signs05:04 – Discovering MECP2 and Rett Syndrome’s Cause07:22 – Founding the Rett Syndrome Research Foundation08:29 – The Breakthrough Reversibility Experiment11:10 – Turning to Gene Therapy (2008–2010)13:08 – Parental Perspective: Asking Naive but Important Questions15:23 – What is Rett Syndrome? Symptoms and Progression18:13 – Prevalence and Underdiagnosis: Newborn Screening Data22:07 – Diagnostic Delays and Barriers for Families25:58 – Founding RSRT: A Focus on Genetic Medicines27:24 – Major Milestones in 25 Years of Rett Research30:31 – Trials and Setbacks: Novartis, Avexis, and Lessons Learned32:20 – Advice for Newly Diagnosed Families36:22 – How RSRT Raised $100 Million Through Families38:05 – Why Funding Still Matters Most Today39:13 – RSRT’s Team and Family-Driven Urgency44:07 – The Importance of Early Diagnosis47:05 – Debating Newborn Screening Policy48:41 – The Current Gene Therapy Trials (Taysha & Neurogene)53:29 – Roadmap to Cures 2.0: What’s Next for Rett Research57:09 – The Future: Base Editing, Prime Editing, and RNA Approaches1:00:27 – The Challenges of Delivery (AAV9 and Beyond)1:02:05 – Ranking the Modalities & FDA Collaboration1:07:32 – Collaboration Across Rare Disease Communities1:08:47 – Final Thoughts & Message to Families1:09:30 – Closing Monologue: Updates on Rett & STXBP1 Trials

    1h 12min

  7. 6 de jan.

    Dr. Alex Kolevzon on Gene Therapy, SHANK3 & the Future of Treatment | Generation Hope

    Welcome to Generation Hope. In this wide-ranging interview, Dr. Alex Kolevzon (Mount Sinai) sits down with Ron to discuss the science, the trials, and the human side of gene therapy for rare neurodevelopmental disorders.In this episode we cover:• Dr. Kolevzon’s path into autism research and why focusing on genetics (like SHANK3) matters.• The evolution from symptom-based treatments to gene-targeted approaches.• Clinical progress: what early trials look like (design, dosing, age cohorts) and how behavioural therapies will work alongside gene therapy.• Biomarkers and outcome measures — EEG, imaging, and how those tools help stratify patients.• Neonatal sequencing and the ethical questions around early genetic screening.• Real family perspectives and why these advances could be transformative for children and adults living with Phelan-McDermid, Rett, STXBP1, Angelman and more.• A cautiously optimistic take on timelines and the potential societal impact — what a future with disease-modifying therapies might look like.If you care about gene therapy, rare genetic disorders, or the future of neurodevelopmental medicine, this conversation offers both scientific insight and real-world hope.📌 Visit our site for more interviews, updates, and resources: GENERationHope.co👍 Like this video if you found it helpful and subscribe to be notified when new interviews drop.🔔 Hit the bell to get updates about the documentary and Gene Therapy breakthroughs.

    50 min

  8. 6 de jan.

    Dr. Curtis Rogers on Phelan-McDermid: Diagnosis, Newborn Screening, AI & Gene Therapy Delivery

    Dr. Curtis Rogers is a Senior Clinical Geneticist at the Greenwood Genetic Center and a long-time leader in the Phelan-McDermid syndrome (PMS) community.In this wide-ranging conversation recorded at the 2024 Phelan-McDermid community conference, we talk about: • What inspired Dr. Rogers to pursue medicine and genetics • How Phelan-McDermid syndrome was first recognized • How families built a community even before the internet • How genetic testing evolved from early chromosome studies to today’s sequencing • Why access, wait times, and insurance can slow diagnosis • The promise—and ethical complexity—of newborn screening • Where AI is beginning to help in clinical genetics • Why delivering genetic medicines to the brain is one of the biggest real-world challengesGENEration Hope shares stories and science from the rare disease community—focused on what’s changing now, and what’s coming next.▶️ Subscribe for more interviews:  @GENErationHope_1  🌐 Learn more: generationhope.co#PhelanMcDermid #SHANK3 #RareDisease #Genetics #GeneTherapy

    56 min

  9. 01/12/2025

    Sequencing Every Baby? Newborn Genomes, GUARDIAN & Rett – Dr. Wendy Chung

    Newborn genome sequencing isn’t science fiction anymore. It’s already changing which babies get diagnosed, when they get treated, and how many families spend years in diagnostic limbo.When our daughter Evie started missing her milestones, we spent five years bouncing between specialists, MRIs, EEGs, blood tests, microarrays and more—with no answers. It took whole exome sequencing to finally reveal a point mutation in SHANK3, now known as Phelan-McDermid syndrome. Our story is heartbreakingly common for families with genetic disorders—and the emotional and economic cost is huge. In this episode of GENEration Hope, I sit down with Dr. Wendy K. Chung—clinical and molecular geneticist, Chair of Pediatrics at Boston Children’s Hospital and Harvard Medical School, and Principal Investigator of the GUARDIAN newborn genome screening study. We talk about: • How whole-genome sequencing (gWGS) is changing newborn diagnosis • What the GUARDIAN study is finding in over 13,000 babies so far • Why Rett syndrome is far more common than we thought (around 1 in 1,500 girls) • The week she diagnosed a 72-year-old and a 3-week-old with Rett • The ethics of telling parents about serious conditions when babies still look “perfectly fine” • How families can “stand up and be counted” in registries so they’re not left out of future trials • Why early diagnosis is essential for gene-targeted therapies—and why timing may one day mean treating even before birth

    48 min
  • 9 episódios

Sobre

In-depth conversations with scientists, clinicians, advocates, and families at the front lines of rare disease, gene therapy, and genomic medicine. Hosted by rare disease parent and filmmaker Ron Kleiman, GENEration Hope explores the science, the ethics, and the human stories behind the fight to give children with genetic disorders a better future.

Informações

  • Criado por
    GENEration Hope - Ron Kleiman
  • Anos de atividade
    2025 - 2026
  • Episódios
    9
  • Classificação
    Livre
  • Copyright
    © GENEration Hope - Ron Kleiman
  • Site do podcast
    GENEration Hope