This is exactly the kind of scrutiny a rare-disease gene therapy program should welcome before it reaches a larger trial.

Follow the whole series: https://bit.ly/Glafabra-TTW

@Brian Christie  @Neva West  @Brian Christie  @Tetsu Yung

Testing the Waters disclosure: We are considering a capital raise and are "Testing the Waters" under Regulation Crowdfunding. No money or other consider...more

Glafabra co-founder Dr. Jeff Medin presents Live-cel data at IFFD 2026, the international Fabry symposium at Jikei University in Tokyo, on October 2.

This puts trial data in front of the specialist community that diagnoses and manages Fabry patients directly -- a different, and in some ways more demanding, audience than investors or the general public.

We'll report what was actually discussed and ...more

Prepared by Putnam Insights LLC

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Dwayne is Professor Emeritus at the University of Toronto, author of more than 70 papers on hematopoietic signalling, leukemia, and lysosomal storage disorders, and served as project manager for the Phase 1 trial that first used gene therapy to treat Fabry disease -- the pilot work behind Glafabra's FACTS data.

He brings direct experience with the regulatory challenges specific to cell and gene th...more

Fabry, Pompe, and Gaucher disease are each caused by a different missing lysosomal enzyme, but treated with the same underlying approach: correct a patient's own hematopoietic stem cells ex vivo, then re-infuse them. FDA's INTERACT feedback confirmed CMC knowledge -- vector backbone, packaging system, process steps -- can carry across programs where those elements are conserved.

That's the basis f...more

The clinical data package FDA asked for in INTERACT feedback (alpha-Gal A activity, plasma lyso-Gb3, eGFR slope) is largely the same package Japan's PMDA would want to see for a parallel review. Glafabra is building Japan pharma and academic relationships (via IFFD, BIO Japan, and direct outreach) in parallel with the U.S. trial, rather than sequencing Japan after U.S. approval.

Done well, this co...more

Krista most recently served as Scientific Director at the Critical Path Institute, leading biomarker discovery for lysosomal storage disorders for inclusion in FDA trials.

She previously held research leadership roles at Florida Gulf Coast University and the University of Alabama at Birmingham's Department of Pediatrics.

At Glafabra, she leads the regulatory science translating our data into the e...more