Precision BioSciences (NASDAQ:DTIL) shares are in focus after the U.S. Food and Drug Administration granted Fast Track designation to PBGENE-DMD, an investigational in vivo gene editing therapy designed to treat Duchenne muscular dystrophy (DMD).
The designation follows the recent clearance of the company’s Investigational New Drug (IND) application, clearing the path for the Phase 1/2 FUNCTION-DMD clinical study.
Unlike traditional gene therapies that deliver a functional copy of a gene via a viral vector, Precision’s candidate utilizes its proprietary ARCUS platform to edit the genome directly, potentially offering a more durable solution for boys living with the degenerative muscle disease.
The Fast Track program is designed to accelerate the development and review of drugs that treat serious conditions and fill unmet medical needs.
For Precision BioSciences, this status allows for more frequent communication with the FDA and eligibility for Rolling Review or Priority Review if supported by clinical data.