Precision BioSciences (NASDAQ: DTIL) gets rare pediatric designation granted from FDA

Precision BioSciences, Inc. (NASDAQ: DTIL) stock surged 17% on Wednesday after the company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for its PBGENE-DMD treatment for Duchenne muscular dystrophy (DMD).

The designation highlights the significant unmet need for new therapeutic options for boys living with DMD, a rare genetic disorder that affects approximately 15,000 people in the United States. DMD primarily affects individuals under the age of 18 and is characterized by progressive muscle weakness.

Precision’s PBGENE-DMD program leverages the company’s proprietary ARCUS gene editing platform to excise exons 45-55 of the dystrophin gene. According to the company, this approach has the potential to benefit up to 60% of patients with DMD by permanently editing a patient’s DNA sequence to produce naturally occurring, near full-length dystrophin protein.

“Our approach is designed to permanently edit a patient’s own DNA sequence, resulting in naturally produced, near full-length dystrophin protein known to be functional in humans,” said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences.

With this designation, Precision may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD. This voucher can be used to receive FDA priority review for a different product or can be sold to another sponsor, potentially providing non-dilutive capital.

The clinical stage gene editing company is currently completing final IND-enabling toxicology studies and expects to generate initial clinical data in 2026.