Glafabra Therapeutics, Uncommon Cures Collaborate to Advance Fabry Disease Therapy
September 25, 2025
PARK CITY, UT – September 3, 2025 – Glafabra Therapeutics, a clinical-stage biotechnology company developing long-lasting cellular gene therapies, and Uncommon Cures, a trailblazing rare disease clinical trial program, today announced a collaboration to advance Glafabra’s lead asset for Fabry disease into IND-enabled clinical trials in the United States.
“We are very excited to have selected Uncommon Cures to guide us through regulatory and into the clinic with our Live-cel technology,” said Chris Hopkins, CEO of Glafabra. “We are excited to bring to patients with Fabry Disease a new therapy that lasts for years, not weeks.”
Chuck Bucklar, Chief Business Officer of Uncommon Cures, stated, “The Fabry therapy at Glafabra is a compelling opportunity to advance our mission to accelerate the pace at which safe and effective therapies are brought to the rare disease patient community. We are thrilled to join Glafabra on their journey to help make the lives of the Fabry patients better.”
About Glafabra Therapeutics Glafabra is a clinical-stage biotechnology company focused on developing long-lasting cellular gene therapies for patients with rare enzyme-deficiency disorders like Fabry, Gaucher, and Pompe disease.
Glafabra’s main technology, known as Live-cel Therapy, uses a patient’s own (autologous) stem cells to restore gene function. The process involves several steps: harvesting a patient’s hematopoietic stem cells (HSCs) from their bone marrow; genetically engineering the cells in a lab to produce a missing or deficient enzyme; and reintroducing the modified cells back into the patient, where they then secrete the therapeutic protein. Unlike traditional enzyme replacement therapy, which requires frequent infusions, Glafabra’s “one-and-done” approach aims to provide years of relief from a single treatment.
ABOUT UNCOMMON CURES Based in Chevy Chase, Maryland, Uncommon Cures is revolutionizing the way that clinical trials for rare disease therapies are designed and conducted – combining scientifically rigorous designs tailored to the unique features of rare diseases, with a global site network centered on the patient experience. Uncommon Cures’ mission is to accelerate the development and delivery of life-changing treatments for patients with rare diseases by streamlining the clinical trial process, reducing costs, prioritizing patient well-being, and maximizing the probability of regulatory success.
Media Contacts
Chris Hopkins, Chief Executive Officer, chris@glafabra.com, 801-631-9114
Chuck Bucklar, Chief Business Officer, cbucklar@uncommoncures.com, 206-303-9308