Long-acting Cell-Based Gene Therapy, Fabry Disease and Beyond with Glafabra CEO, Dr. Chris Hopkins
About this episode
The episode features a discussion with Dr. Chris Hopkins on the advancements in cell-based gene therapies for rare diseases, particularly Fabry disease.
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…
More episodes of BioTalk Unzipped
- Active Machine Learning for Drug Discovery & Nanomedicine with Dr. Daniel Reker
- Why Haven’t We Cured Cancer Yet? | Dr. Bob Liu Explains
- The Intersection of Biotech, Patent Law, and AI with Dr. Kate Neville, IP Attorney
- Fail Fast, Learn Faster: Drug Development in Rare Disease with Dr. Binodh DeSilva
- Unlocking Antibody Development with AI with Dr. Pin-Kuang Lai
- The Science and Hope of Brain Cancer with Dr. Robert Thorne, Denali Therapeutics
Explore listener stats, chart rankings, contacts and more on the BioTalk Unzipped podcast page.