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.

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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