Long-acting Cell-Based Gene Therapy, Fabry Disease and Beyond with Glafabra CEO, Dr. Chris Hopkins

Active Machine Learning for Drug Discovery & Nanomedicine with Dr. Daniel Reker

Can artificial intelligence help make cancer therapies safer, more targeted, and more effective? In this episode of BioTalk Unzipped, Gregory Austin sits down with Dr. Daniel Reker, Assistant Professor at Duke University, for a wide-ranging conversation on active machine learning, nanomedicine, drug delivery, and the future of AI in biomedical research. This episode is brought to you by Leucentra. Inspired by Science Empowered by IT https://leucentra.com/ [https://leucentra.com/] Dr. Reker works at the intersection of AI, chemistry, biomedical engineering, pharmacology, and molecular medicine. His lab develops computational and experimental approaches to better understand small molecules, nanoformulations, and drug delivery systems. The conversation explores how machine learning can support drug discovery and development, especially in areas where datasets are small and the biology is complex. Dr. Reker explains why nanoformulations may be able to improve targeted drug delivery, reduce toxicity, and potentially revive therapeutic agents that previously failed because of safety or tolerability issues. Gregory and Dr. Reker also discuss explainable AI, the risks of black box thinking, AI bias, predictive modeling, FDA considerations, non-animal models, and the responsible use of AI in education and science. Topics include: • Active machine learning in drug discovery • AI and nanomedicine • Cancer therapy and targeted drug delivery • How nanoformulations may reduce toxicity • Small datasets in biomedical AI • Explainable AI and scientific trust • AI bias and model limitations • Regulatory implications for predictive models • The role of AI in education and cognitive development • The future of integrated data in drug development Guest bio: Dr. Daniel Reker is an Assistant Professor at Duke University. His research focuses on computational and experimental approaches to molecular medicine, including active machine learning, drug delivery, nanoformulations, small molecules, and translational pharmacology. He was named to Forbes 30 Under 30 Europe in Science and Healthcare. Guest contact: Dr. Daniel Reker Email: daniel.reker@duke.edu LinkedIn: https://www.linkedin.com/in/danielreker/ [https://www.linkedin.com/in/danielreker/] Duke website: https://rekerlab.pratt.duke.edu/ [https://rekerlab.pratt.duke.edu/] Connect with BioTalk Unzipped: Gregory Austin https://www.linkedin.com/in/gregoryaustin1/ [https://www.linkedin.com/in/gregoryaustin1/] Dr. Chad Briscoe https://www.linkedin.com/in/chadbriscoe/ [https://www.linkedin.com/in/chadbriscoe/] BioTalk Unzipped uncovers the stories behind medical progress through conversations with innovators across biotech, pharma, medtech, bioanalysis, clinical research, regulatory science, and drug development.

Why Haven’t We Cured Cancer Yet? | Dr. Bob Liu Explains

Why haven’t we cured cancer yet? A Genentech scientist explains the real reason. In this episode of BioTalk Unzipped, Gregory Austin and Dr. Chad Briscoe sit down with Dr. Bob Liu, Senior Principal Scientist at Genentech Roche, to unpack one of the most important and misunderstood questions in modern medicine. This is a rigorous, scientifically grounded conversation on cancer biology, immunotherapy, and the real constraints shaping oncology drug development today. Dr. Liu brings over a decade of experience across antibody drug conjugates (ADCs), bispecific antibodies, and CAR-T therapies, offering a rare, insider perspective on why a universal cure remains elusive and where meaningful progress is actually being made. Thanks to our Founding Sponsor: LEUCENTRA: Helping teams evaluate, implement, and get real value from IT solutions that support innovation, not slow it down. https://leucentra.com/ [https://leucentra.com/] WHAT YOU’LL LEARN 1. Why cancer is not one disease, but more than 200 biologically distinct conditions 2. What “curing cancer” actually means in clinical oncology 3. How immunotherapies like checkpoint inhibitors and CAR-T are changing outcomes 4. The biological limits of eliminating every cancer cell 5. How tumors evade immune detection and adapt over time 6. Why only about 20% of patients respond to immuno-oncology therapies 7. The role of biomarkers, molecular profiling, and precision medicine 8. Why early detection remains one of the biggest unsolved challenges 9. The economic and regulatory pressures shaping next-generation therapies KEY INSIGHT Cancer is not simply something to eliminate. It is a dynamic, adaptive system evolving within the human body. The future of oncology is not just eradication, but control, personalization, and intelligent engagement of the immune system. NOTABLE QUOTES “Cancer is a collection of more than 200 diseases, each requiring its own specific approach.” “The cure for some cancers is within reach, but for many others, early detection remains the critical challenge.” “Our immune system is constantly surveilling. The key is learning how to harness it effectively.” TIMESTAMPS 00:00 – Introduction 02:42 – Bob’s passion - AACR 04:28 – Why we haven’t cured cancer 07:09 – Defining a cancer cure 10:26 – Cancer classification and molecular signatures 14:16 – Methylation profiling in diagnosis 17:22 – Patient resources and navigation 21:14 – FDA shifts toward randomized trials for CAR-T 24:15 – Cost and access challenges 26:33 – Cancer vs cardiovascular disease progress 33:39 – The challenge of early detection 37:48 – Biomarker limitations 39:14 – Immune system dynamics in cancer 45:19 – Bioanalytical challenges in modern therapies 51:08 – Progress and future outlook ABOUT THE GUEST Dr. Bob Liu is a Senior Principal Scientist at Genentech Roche specializing in bioanalytical sciences and immunogenicity assessment for advanced oncology therapies, including T-cell bispecifics and CAR-T. RESOURCES & LINKS FDA to tighten approval requirements for CAR-T therapies https://www.raps.org/news-and-articles/news-articles/2025/12/fda-to-tighten-approval-requirements-for-car-t-cel [https://www.raps.org/news-and-articles/news-articles/2025/12/fda-to-tighten-approval-requirements-for-car-t-cel] American Association for Cancer Research (AACR) https://www.aacr.org/ [https://www.aacr.org/] National Cancer Institute – Molecular diagnostics and biomarkers https://www.cancer.gov/about-cancer/diagnosis-staging/diagnosis [https://www.cancer.gov/about-cancer/diagnosis-staging/diagnosis] Pattern recognition technologies in diagnostics https://toby.health [https://toby.health] CONNECT Dr. Bob Liu https://www.linkedin.com/in/bob-liu-42b8b278/ [https://www.linkedin.com/in/bob-liu-42b8b278/] Dr. Chad Briscoe https://www.linkedin.com/in/chadbriscoe/ [https://www.linkedin.com/in/chadbriscoe/] Gregory Austin https://www.linkedin.com/in/gregoryaustin1/ [https://www.linkedin.com/in/gregoryaustin1/] FINAL THOUGHT The path to curing cancer is not a single breakthrough. It is a long, complex progression of scientific advances, better diagnostics, and deeper biological understanding. The progress is real. But the work is far from finished.

Innovation: The Intersection of Biotech, Patent Law, and AI with Dr. Kate Neville, IP Attorney

Recorded October 31, 2025 In this episode of BioTalk Unzipped, Gregory Austin and Dr. Chad Briscoe sit down with Dr. Kate Neville, immunologist turned seasoned biotech patent attorney at Marshall, Gerstein & Borun, to unpack one of the most misunderstood and mission-critical areas of life sciences: intellectual property. If you are a biotech founder, scientist, executive, or investor, this conversation is essential listening. We explore what patent prosecution really means, when startups should begin thinking about IP protection, how “freedom to operate” can determine commercial viability, and how emerging AI tools are reshaping the patent landscape. Dr. Neville brings 25+ years of experience guiding university spin-outs, biotech startups, and global pharmaceutical companies through complex patent strategy. She has helped secure patents for FDA-approved drugs and offers a rare dual perspective as both scientist and attorney. IN THIS EPISODE WE DISCUSS: • The difference between patent prosecution and patent litigation • Why it is never too early for biotech startups to think about IP • The U.S. one-year grace period vs. Europe’s stricter disclosure rules • What “Freedom to Operate” really means for commercialization • Antibody patents, CDR regions, and the doctrine of equivalents • How premature disclosure can impact global patent strategy • The real-world back-and-forth of patent office “office actions” • AI-assisted prior art search at the USPTO — opportunity or risk? • How funding cycles influence patent filing decisions • Women in biotech leadership and venture funding disparities • The most rewarding part of protecting life-changing therapies We also break down the USPTO’s new AI pilot programs designed to modernize patent examination and discuss how artificial intelligence may impact biotech patenting over the next several years. WHY THIS MATTERS Intellectual property is often the single most valuable asset in a biotech company. Strong IP strategy can unlock funding, partnerships, and market exclusivity. Weak or mistimed IP decisions can permanently limit global opportunity. For founders and scientists: timing, geography, and disclosure discipline matter more than most people realize. ABOUT OUR GUEST Dr. Kate Neville Partner, Marshall, Gerstein & Borun PhD in Immunology, JD LinkedIn: https://www.linkedin.com/in/kate-neville-phd/ [https://www.linkedin.com/in/kate-neville-phd/] Firm Bio: https://www.marshallip.com/katherine-l-neville-ph-d/ [https://www.marshallip.com/katherine-l-neville-ph-d/] Charity Highlight: Girls on the Run Chicago An organization building confidence and resilience in young girls through mentorship and athletic achievement. https://www.girlsontherun.org/ [https://www.girlsontherun.org/] HOSTS Dr. Chad Briscoe Bioanalytical Scientific Leader https://www.linkedin.com/in/chadbriscoe/ [https://www.linkedin.com/in/chadbriscoe/] Gregory Austin Director, Business Development | Bioanalysis https://www.linkedin.com/in/gregoryaustin1/ [https://www.linkedin.com/in/gregoryaustin1/] If you enjoyed this episode, subscribe to BioTalk Unzipped on Apple Podcasts, Spotify, or your preferred platform and share with a colleague in biotech, pharma, or life sciences innovation.

Long-acting Cell-Based Gene Therapy, Fabry Disease and Beyond with Glafabra CEO, Dr. Chris Hopkins

In this episode of BioTalk Unzipped, hosts Gregory Austin and Dr. Chad Briscoe sit down with Glafabra CEO: Dr. Chris Hopkins, geneticist, biochemist, and biotech entrepreneur, to explore the science and strategy behind next generation cell-based gene therapies for rare diseases. With more than 25 years of experience spanning gene augmentation, rare disease biology, CRISPR licensing, and biotech formation, Dr. Hopkins shares how autologous, ex vivo engineered cell therapies may overcome key limitations of current enzyme replacement and viral gene therapies, particularly for Fabry disease. The conversation dives deep into: • How lentiviral gene augmentation in patient derived cells enables sustained enzyme production • Why redosing matters and where one time AAV therapies fall short • The scientific rationale for early intervention, including potential newborn treatment • Differences between autologous and emerging allogeneic approaches • Regulatory pathways for rare disease therapies and recent FDA developments • The role of non animal models in translational research • Montana’s early access therapy law and its broader implications • Building biotech platforms amid a challenging funding environment Topics include cell based gene therapy, Fabry disease, lentiviral vectors, stem cell engineering, rare disease drug development, regulatory science, and translational medicine. Subscribe to BioTalk Unzipped for in depth conversations with the scientists and leaders shaping the future of biomedical innovation. 00:00 - Intro 00:53 – Welcome to BioTalk Unzipped, Guest intro: Dr. Chris Hopkins 02:10 – Guest charity: Environmental Defense Fund 03:12 – His journey into rare-disease therapeutics and Glafabra 05:58 – Discovering a new enzyme-deficiency therapy 06:39 – Current standard of care 07:42 – How the new autologous cell therapy works 09:40 – Treating patients earlier (even newborns) 10:33 – Emerging therapies - AAV gene therapy vs. cell-based therapy 12:16 – Long-term results & repeat dosing 14:30 – Future plans: T-cells & allogeneic approaches 18:08 – New News: FDA resubmission for rare disease 20:00 – Navigating FDA pathways 22:06 – Non-animal testing & alternative models 25:50 – Montana’s early-access therapy law & medical tourism 29:03 – Could other states follow? 31:31 – Biotech’s current funding challenges 33:46 – New News: Gene therapy trial saves 4-year-old 37:09 – Long-term vision for expanding therapies 39:53 – Personal segment: outdoor life & skiing 44:43 – Guest question on international trade Dr. Christopher Hopkins https://www.linkedin.com/in/christopherehopkins/ [https://www.linkedin.com/in/christopherehopkins/] Glafabra - https://www.glafabra.com/ [https://www.glafabra.com/] Environmental Defense Fund - https://www.edf.org/ [https://www.edf.org/] Dr. Chad Briscoe https://www.linkedin.com/in/chadbriscoe/ [https://www.linkedin.com/in/chadbriscoe/] Celerion - https://www.celerion.com/ [https://www.celerion.com/] Gregory Austin https://www.linkedin.com/in/gregoryaustin1/ [https://www.linkedin.com/in/gregoryaustin1/] Celerion - https://www.celerion.com/ [https://www.celerion.com/] New News Articles: US FDA asks Stealth BioTherapeutics to resubmit application for rare genetic condition therapy https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-asks-stealth-biotherapeutics-resubmit-application-rare-genetic-condition-2025-05-29/ [https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-asks-stealth-biotherapeutics-resubmit-application-rare-genetic-condition-2025-05-29/] Gene therapy trial saves boy, 4, from 'death sentence' https://www.thetimes.com/uk/healthcare/article/gene-therapy-trial-great-ormond-street-70l2sgqw [https://www.thetimes.com/uk/healthcare/article/gene-therapy-trial-great-ormond-street-70l2sgqwj] Montana, revolutionary law passed: unlimited research for longevity https://en.ilsole24ore.com/art/montana-approved-revolutionary-law-researching-longevity-without-limits-AHmDI7BB?refresh_ce=1 [https://en.ilsole24ore.com/art/montana-approved-revolutionary-law-researching-longevity-without-limits-AHmDI7BB?refresh_ce=1] KEY TAKEAWAYS 1. A NEW CELL THERAPY COULD REPLACE LIFELONG ENZYME TREATMENTS FOR FABRY PATIENTS. 2. UNLIKE GENE THERAPY, THIS TREATMENT CAN BE REDOSED — NO ONE-AND-DONE LIMIT. 3. EARLY INTERVENTION, EVEN IN NEWBORNS, MAY BECOME POSSIBLE. 4. MONTANA’S NEW LAW COULD OPEN A FAST LANE FOR EXPERIMENTAL THERAPIES IN THE U.S. 5. DESPITE A TOUGH FUNDING CLIMATE, BREAKTHROUGHS SHOW GENE-EDITED CELL THERAPIES ARE RESHAPING THE FUTURE. #CELLTHERAPY #GENEEDITING #RAREDISEASE #BIOTECHINNOVATION #GENETICMEDICINE #HEALTHCAREFUTURE #MEDICALBREAKTHROUGH #CellBasedGeneTherapy #RareDisease #FabryDisease #GeneAugmentation #LentiviralVectors #Biotech #TranslationalScience #GeneticMedicine #BioTalkUnzipped https://youtu.be/VcPXZmK-XU8 [https://youtu.be/VcPXZmK-XU8]

Fail Fast, Learn Faster: Drug Development in Rare Disease with Dr. Binodh DeSilva

In this episode of BioTalk Unzipped, Gregory Austin and Dr. Chad Briscoe sit down with Dr. Binodh DeSilva, Senior Vice President of Bioanalysis at Ultragenyx Pharmaceutical, to explore the science and soul behind rare-disease drug development. From her early days studying electrochemistry at the University of Kansas to leading cutting-edge bioanalytical programs at Ultragenyx, Dr. DeSilva shares how curiosity and community shaped her four-decade career. She discusses the profound responsibility of working with limited, often irreplaceable patient samples with care. A special thanks to AAPS (https://www.aaps.org/ [https://www.aaps.org/]) for their help and support of this episode. The conversation dives into: * Balancing rigor and agility in small-population clinical studies * Leveraging entrepreneurial mindsets from biotech within big pharma frameworks * The promise of dried blood spots (DBS) and patient-centric sampling * Mentorship, curiosity, and the future of scientific leadership * Her return to Sri Lanka with KU faculty to recruit the next generation of scientists Throughout the discussion, DeSilva underscores a recurring theme: science thrives when curiosity meets compassion. This episode is a masterclass in both. Guest Links Dr. Binodh DeSilva https://www.linkedin.com/in/binodh-desilva/ [https://www.linkedin.com/in/binodh-desilva/] Ultragenyx Pharmaceuticals - https://www.ultragenyx.com/ [https://www.ultragenyx.com/]  Hosts Dr. Chad Briscoe https://www.linkedin.com/in/chadbriscoe/ [https://www.linkedin.com/in/chadbriscoe/]  Celerion - https://www.celerion.com/ [https://www.celerion.com/]   Gregory Austin https://www.linkedin.com/in/gregoryaustin1/ [https://www.linkedin.com/in/gregoryaustin1/]  Celerion - https://www.celerion.com/ [https://www.celerion.com/]   Keywords: BioTalk Unzipped, Binodh DeSilva, Ultragenyx, rare disease research, bioanalysis, dynamic drug development, dried blood spots, DBS sampling, biologics, AAPS NBC 2025, Gregory Austin, Chad Briscoe, Celerion, scientific leadership, mentorship in science, biopharma innovation, curiosity in research, Sri Lanka scientists, analytical chemistry, pharma innovation, drug development ethics.