Prevail Therapeutics’ Phase 1/2 PROCEED clinical study, which will assess our PR001 experimental gene therapy for Gaucher disease Type 1 is now open for patient enrollment.
The initial site, located at the Lysosomal & Rare Disorders Research and Treatment Center in Fairfax, Virginia, U.S. is now screening potential participants and a second U.S. location is expected to begin enrollment in the coming months.
About Prevail Therapeutics
Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company, strives to develop disease-modifying, potential one-time gene therapies for people living with rare conditions, including Gaucher disease, and neurodegenerative diseases.
Prevail is now recruiting for a Phase 1/2 clinical trial, called PROCEED, which will evaluate an AAV-gene therapy candidate delivered by IV infusion (i.e., in vivo, directly into the body), for people living with Type 1 Gaucher disease. If you are interested in learning more about PROCEED, please visit ClinicalTrials.gov (NCT05487599).
In addition, Prevail is also conducting PROVIDE, a Phase 1/2 clinical trial evaluating the same gene therapy candidate delivered directly to the brain for the potential treatment of children diagnosed with Type 2 Gaucher disease. More information about this trial can also be found at ClinicalTrials.gov (NCT04411654).
What is gene therapy?
Gene therapy is one approach to treating genetic diseases and there are several gene therapy methods. The goal of gene therapy is to introduce one or more genes into a person’s cells to make a healthy copy of a missing or lacking protein. The majority of gene therapies in development are intended to be one-time treatments.
What approach does Prevail Therapeutics take to gene therapy?
Prevail Therapeutics is researching gene therapies using an adeno-associated virus (AAV), a vector not known to cause disease in humans, in vivo (directly into the body), to transport genetic material to specific cells and potentially restore the function of a disease-causing gene. In the case of Gaucher disease, the gene therapy will deliver a healthy copy of the GBA1 gene.
What can I expect if I am to pursue participation in the PROCEED trial?
In the PROCEED trial, PR001 is administered as a slow, single-dose IV infusion through a venous catheter, which is a small tube placed into a peripheral vein (e.g., a vein in the arm) to allow the treatment to flow into the body, to treat the peripheral manifestations of GD1 by delivering a healthy copy of the GBA1 gene.
Eligible participants will undergo standard pre-screening and genetic testing.
In advance of the gene therapy administration, a prophylactic medication is given to make sure the body is receptive to the gene therapy by reducing inflammation or immune response. These FDA approved medications are given in a relatively low dose for a short duration and are generally well-tolerated.
The IV infusion takes up to approximately 90 minutes. Overall, an individual should expect their stay in the treatment facility to last approximately 8-12 hours.
Following dosing, the participant and their study doctor will determine the best course of action and timeline for phasing off of ERT/SRT, as outlined in the clinical trial protocol and will participate in standard follow-up visits for an additional four years.
How can I learn more?
Additional information on Prevail’s approach and the PROCEED trial can be found at: