Aimee Donald, MBChB, PhD, on Assessing PR001 in Children With Type 2 Gaucher Disease

“We're all kind of waiting desperately to see the outcome of this trial, because I can't think of another trial that has approached GD2 specifically. Certainly, there's nothing else out there at the moment for this patient population. So, it's a really important study, and not just important for GD2 or Gaucher disease, but across lysosomal storage disorders.”

PR001, (Prevail Therapeutics), a single dose, potentially disease-modifying gene therapy, is being evaluated for the potential treatment of infants with type 2 Gaucher disease (GD2), also known as neuronopathic GD, in the phase 1/2 PROVIDE clinical trial (NCT04411654). PR001 is an adeno-associated virus (AAV9) gene therapy administered via intracisternal magna injection.

PR001 aims to address the mutations in the GBA1 gene responsible for Gaucher disease that disrupt the production of beta-glucocerebrosidase and therefore the metabolism of glycolipids, which aggregate α-Synuclein and lead to inflammation and neurodegeneration. The multicenter, open-label study initiated dosing in 2020 and is continuing to enroll up to its target enrollment of 15 participants. Its primary completion date is September 2028.

CGTLive spoke with Aimee Donald, MBChB, PhD, pediatrician, Royal Manchester Children’s Hospital, and professor, University of Manchester, to learn more about the PROVIDE trial. She also discussed the current treatment landscape of GD2 and unmet needs that remain for this population.

REFERENCE

Prevail Therapeutics provides PR001 program update. News release. Prevail Therapeutics. January 9, 2020. https://www.globenewswire.com/en/news-release/2020/01/09/1968680/0/en/Prevail-Therapeutics-Provides-PR001-Program-Update.html