Precision BioSciences, Inc. (DTIL) announced Wednesday that the U.S. Food and Drug Administration or FDA has granted Rare Pediatric Disease Designation for PBGENE-DMD, the company's investigational gene editing therapy for Duchenne muscular dystrophy or DMD.
PBGENE-DMD is a preclinical-stage in vivo gene editing program using Precision's proprietary ARCUS platform.
The therapy is designed to excise exons 45-55 of the dystrophin gene through a single AAV-delivered dose, with the goal of restoring near full-length dystrophin protein expression.
According to the company, the therapy has the potential to benefit up to 60 percent of DMD patients, a rare and life-threatening neuromuscular disorder that affects approximately 15,000 individuals in the U.S., primarily boys under 18.
In preclinical studies, PBGENE-DMD demonstrated durable dystrophin expression in skeletal and cardiac muscle, as well as satellite muscle stem cells, which are believed to be important for long-term therapeutic benefit.
With this designation, Precision may be eligible to receive a Priority Review Voucher or PRV upon FDA approval of PBGENE-DMD.
The PRV can be used to expedite FDA review of another therapy or sold to other sponsors as a source of non-dilutive funding.
Precision BioSciences noted that IND-enabling toxicology studies are ongoing, and initial clinical data is expected in 2026.
Chief Development and Business Officer Cindy Atwell stated that the designation highlights the significant unmet need in DMD and supports the differentiated capabilities of the ARCUS platform.
Cindy confirmed that PBGENE-DMD is designed to permanently edit the patient's own DNA to enable production of a functional dystrophin protein.
Currently, DTIL, is trading at $4.43, down by 2.84 percent on the Nasdaq.
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