GALYA-1: Gene Therapy for Niemann-Pick Type C
How GALYA-1 Works
GALYA-1 works like a delivery truck: the truck is a non-pathogenic virus and the package it carries is a working copy of the NPC1 gene. GALYA-1 is expected to be administered via a lower back (intrathecal) injection into the central nervous system, or intravenously. From the injection site, it can travel to the brain to deliver the working NPC1 gene to cells. (Last updated: March 29, 2018)
With VTS-270 (Cyclodextrin)
GALYA-1 is not intended to compete with the drug VTS-270 (a specific, well-characterized composition of hydroxypropyl-beta-cyclodextrin). Rather, GALYA-1 is expected to work in combination with VTS-270 for a more complete treatment strategy.
After diligent testing in multiple animal models followed by guidance from the FDA, we are moving forward with manufacturing and remaining preclinical studies necessary to support a Phase I clinical trial. In our March 2018 Update, we laid out three estimates for timeline to file an IND. (Last updated: March 29, 2018)
In order to enroll in the Phase I trial for GALYA-1, a person must...(We will update this space once we have received regulatory approval for an IND.)