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.
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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/
Glafabra - https://www.glafabra.com/
Environmental Defense Fund - https://www.edf.org/
Dr. Chad Briscoe
https://www.linkedin.com/in/chadbriscoe/
Celerion - https://www.celerion.com/
Gregory Austin
https://www.linkedin.com/in/gregoryaustin1/
Celerion - 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/
Gene therapy trial saves boy, 4, from 'death sentence'
https://www.thetimes.com/uk/healthcare/article/gene-therapy-trial-great-ormond-street-70l2sgqw
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
Key Takeaways1. 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
