The BioInsights Podcast
The BioInsights Podcast: discussing the challenges of translating novel biologics from bench to clinic to market.
The BioInsights Podcast
Beyond one and done: engineering durable, redosable cell‑based gene therapies
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BioInsights
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Season 5
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Episode 3
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The one‑and‑done model has defined gene therapy for a generation. Chris Hopkins thinks it is time to move on. At ASGCT 2026, Abi Pinchbeck spoke with the Co‑founder and CEO of Glafabra Therapeutics on redoseable cell‑based gene therapy, a platform spanning Fabry, Pompe, and Gaucher diseases, and what durable, if not permanent, treatment could mean for patients. A podcast version of this discussion is also available.
Fabry disease has traditionally been managed with enzyme replacement therapy. What led you to pursue a cell‑based gene therapy approach for this indication?
Redosability is central to your platform. How central was it as a design principle from the outset, and what does it mean for how clinicians are likely to schedule treatment?
How do you think about the trade‑offs between gene addition and in situ editing for lysosomal storage diseases, and does a redosable design change that decision‑making?
After Fabry disease, you have identified Pompe and Gaucher diseases as the next targets. What is the rationale for that expansion?
Where do each of your three programs currently stand, and what is your regulatory strategy for each?
What does the right pharma or biotech partnership look like, and at what stage does that conversation become most productive?
Finally, where do you see the company in the next five years, and what would it mean for patients?