By Chris Wack
Precision BioSciences said Tuesday that its partner, iECURE, has received Fast Track designation from the Food and Drug Administration for ECUR-506.
ECUR-506 is iECURE's in vivo gene insertion program designed to treat ornithine transcarbamylase, or OTC, deficiency in newborns using a PCSK9-specific ARCUS nuclease, licensed from Precision, that enables insertion of a functional copy of the OTC gene.
OTC deficiency is the most common urea cycle disorder and is caused by a genetic defect in a liver enzyme responsible for the detoxification of ammonia.
The Fast Track designation is designed to begin the development and expedite the review of therapeutics to treat serious conditions that fill an unmet medical need. Therapeutics that receive Fast Track designation may be eligible for more frequent meetings with and written communication from the FDA to discuss a development plan and ensure collection of appropriate data to support potential approval.
ECUR-506 previously received rare pediatric disease and orphan drug designations from the FDA, and orphan designation from the European Commission to treat OTC deficiency.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
05-07-24 1003ET
