Working Toward a Single Dose Gene Therapy for Type 1 Gaucher Disease


Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.

"The most recently activated study which we call PROCEED is designed to evaluate safety and tolerability of PR001 in people living with type 1 Gaucher disease, who have had a suboptimal response to the standard of care enzyme replacement therapies and substrate reduction therapies... These standard of care therapies are not effective in treating all symptoms of type 1 Gaucher disease, specifically, bone manifestations, and lung manifestations may still be treated in a suboptimal manner. Moreover, these treatments are repetitive and maybe burdensome to patients.”

Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly, is evaluating the gene therapy PR001 in type 1 Gaucher disease (T1GD) in the phase 1/2 PROCEED trial (NCT05487599). PR001 is an adeno-associated virus vector therapy that delivers the GBA1 gene, administered intravenously (IV) in this indication. The study is enrolling up to 15 participants with T1GD.

CGTLive spoke with Olga Uspenskaya, MD, PhD, vice president of clinical development, Prevail Therapeutics, to learn more about Prevail Therapeutics and the PROCEED trial. She discussed advantages of PR001 over standard of care options in the T1GD population.

Prevail Therapeutics has recently also engaged in a strategic collaboration with Capsida Biotherapeuitcs to develop other IV-administered gene therapies for central nervous system indications. The collaboration will see Capisida using its novel AAV engineering platform to develop capsids while Prevail will be responsible for preclinical and Investigational New Drug-enabling studies with therapeutic payloads.

Capsida Biotherapeutics Announces Strategic Collaboration with Prevail, a Wholly Owned Subsidiary of Lilly, to Develop Non-Invasive Gene Therapies for CNS Diseases. News release. Capsida Biotherapeutics. January 4, 2023.
Related Videos
Alfonso Sabater, MD, PhD, on Bringing Gene Therapy to Ophthalmology
Sharon Hesterlee, PhD
Thomas Crawford, MD, on Helping Patients With SMA Left Behind by Gene Therapy
J. Andrew Livingston, MD
Shankar Musunuri, PhD
© 2023 MJH Life Sciences

All rights reserved.