In a recent press release, Precision BioSciences announced that their novel therapy, PBGENE-DMD, had received U.S. Food and Drug Administration (FDA) Orphan Drug Designation for the treatment of Duchenne muscular dystrophy (DMD).
PBGENE-DMD utilizes gene editing technology to restore near full-length dystrophin protein within the human body with the goal of improving functional outcomes. The therapy uses Precision BioSciences’ proprietary ARCUS genome editing platform, which has a unique method of permanently editing the dystrophin gene.
The Orphan Drug Designation for PBGENE-DMD is a status granted by the FDA to therapies that are intended to treat rare conditions impacting fewer than 200,000 people in the U.S. The designation carries several benefits, including a period of market exclusivity, tax credits and a faster approval process.
PBGENE-DMD was also recently granted Rare Pediatric Disease Designation from the FDA, which also carries incentives for the development of new treatments.
Read more about DMD testing and diagnosis
“This regulatory milestone builds on our recent receipt of Rare Pediatric Disease designation and, together with our preclinical body of evidence, gives us tremendous confidence as we move this program towards the clinic,” Cindy Atwell, clinical development and business officer at Precision BioSciences, said. “Looking ahead, we remain in active dialogue with the FDA as we advance PBGENE-DMD toward regulatory milestones, with clinical data anticipated in 2026.”
Precision BioSciences now intends to work with leading DMD clinicians to design a human trial with the aim of testing human response.
Early studies have demonstrated that PBGENE-DMD can target key muscle types that are implicated in the disease progression of DMD. Testing involving a humanized DMD mouse model indicated substantial and persistent functional improvements. PBGENE-DMD could be used to manage up to 60% of the patient population affected by DMD.
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