Glafabra Therapeutics Receives FDA Orphan Drug Designation for GT-GLA-S03, Advancing a Redosable Cell Therapy for Fabry Disease Ready to Announce with Confidence?

Park City, Utah--(Newsfile Corp. - March 12, 2026) - Glafabra Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to GT-GLA-S03, its lead cell therapy candidate for classic Fabry disease.

To view the full announcement, including downloadable images, bios, and more, click here.

Key Takeaways:

FDA Orphan Drug Designation unlocks the path to market. Seven years of exclusivity, $4.68M in fee exemptions, and tax credits -- de-risking the program and signaling FDA validation of the science.

One treatment. Five years. Already proven in humans. Five years of clinical data shows GT-GLA-S03 is safe, effective, and durable -- replacing 130 clinic visits over five years with a single dose, and redosable when needed.

Built to scale beyond Fabry. The same Live-cel platform is already in preclinical development for Pompe and Gaucher diseases -- a combined patient population of 2 million with no durable treatment options today.

Click image above to view full announcement.

Media Contact

Dr. Chris Hopkins, PhD, MBA -- CEO, Glafabra Therapeutics

chris@glafabra.com - 801-631-9114 - www.glafabra.com

ABOUT GLAFABRA THERAPEUTICS

Glafabra Therapeutics is a Park City, Utah biotechnology company developing cell therapies for LSDs using its proprietary Live-cel platform. GT-GLA-S03, its lead program for classic Fabry disease, is backed by five years of human clinical data, FDA Orphan Drug Designation, and issued patent 12,540,336. The pipeline includes preclinical programs for Pompe disease (GT-GAA-S04) and Gaucher disease (GT-GBA1-S05). Co-founded by Dr. Chris Hopkins, Dr. Jeffrey Medin, and Dr. Ronan Foley.

Scientific References: PMID 39794302 (Clin Transl Med. 2025); PMID 33633114 (Nat Commun. 2021); NCT02800070

Note to Editors: Orphan Drug Designation does not indicate that GT-GLA-S03 is safe or effective for the treatment of Fabry disease. GT-GLA-S03 is an investigational therapy not approved by the FDA or any regulatory authority. Clinical data are from a pilot study. This release contains forward-looking statements.

Contacts:

Dr. Chris Hopkins

801-631-9114

chris@glafabra.com

Source: Glafabra

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/288262

Source: Reportable, Inc.

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