FAST-AFFILIATED RESEARCH PROGRAM AT UCLA IS AWARDED $5.8M TO ADVANCE GENE THERAPY FOR ANGELMAN SYNDROME

AUSTIN, Texas, Jan. 30, 2025 /PRNewswire/ -- Dr. Roger Hollis from the University of California, Los Angeles, project scientist from the laboratory of Dr. Donald Kohn, a world leader in developing gene replacement therapies using blood stem cells, has been awarded a $5.8 million translational research award from the California Institute for Regenerative Medicine (CIRM) to further support the development of a hematopoietic stem cell gene therapy (HSC-GT) to treat Angelman syndrome (AS).

The team at UCLA is further developing this gene therapy approach in which a patient's own blood stem cells can be modified to add a healthy copy of the UBE3A gene that is generally either missing or not functioning in patients living with AS. The data to date, funded by TransformaTx Biotherapeutics, a biotechnology company founded by the Foundation for Angelman Syndrome Therapeutics (FAST), has demonstrated complete symptomatic correction in the commonly used adult mouse model of the disease.

"This program has consistently delivered remarkable animal data, reinforcing its potential as a groundbreaking treatment," said Dr. Donald Kohn, Distinguished Professor of Microbiology, Immunology and Molecular Genetics and Director of the UCLA Human Gene Medicine Program and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. "We are excited to collaborate with TransformaTx Biotherapeutics and FAST to advance this investigational gene therapy into clinical trials and bring more trial opportunities to those living with Angelman syndrome."

Chris Luthers, lead scientist for the molecular biology team at UCLA working on HSC-GT development, presented results of both in vitro and in vivo studies at the November 2024 FAST Global Summit on Angelman syndrome. The team will be using the new CIRM grant to prepare a pre-IND package submission to the U.S. Food and Drug Administration, which is the first step toward launching a Phase 1 clinical trial to evaluate the therapy in humans.

"Securing this critical funding from CIRM is a major step forward, accelerating our path to a Phase 1 clinical trial," said Dr. Allyson Berent, chief development officer at TransformaTx Biotherapeutics and chief science officer of FAST. "This achievement is a testament to the dedication of our incredible scientific team and the AS community, whose support has driven this program from the start."

Media Contact: Meghan Edberg, Director of Public Engagement (630-852-3278), [email protected] 

SOURCE Foundation for Angelman Syndrome Therapeutics (FAST)