Precision BioSciences received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD, a gene-editing therapy in development for Duchenne muscular dystrophy (DMD), announced a press release published recently by the company.
This designation marks a step forward in potential new treatment options and recognizes the significant need for new therapies to address DMD. For patients and families, this news may offer hope that more targeted and effective treatments could become available in the coming years.
“The receipt of Rare Pediatric Disease Designation highlights the significant unmet need that necessitates new therapeutic options for boys living with DMD,” said Cindy Atwell, chief development and business officer at Precision BioSciences.
PBGENE-DMD uses Precision Bioscience’s proprietary ARCUS platform to edit the dystrophin gene in a permanent way, aiming to restore the body’s ability to produce a near full-length, functional version of the dystrophin protein. The approach involves excising exons 45 through 55 from the gene, a strategy that could be relevant to more than 60% of people living with DMD. By addressing such a large segment of patients, the therapy could offer a meaningful option where few currently exist.
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In preclinical studies, PBGENE-DMD demonstrated the ability to reach and edit key muscle types involved in DMD’s progression. Research in a humanized mouse model showed significant, lasting improvements across multiple muscle groups, including the heart and critical skeletal muscles.
The therapy also edited muscle stem cells, which are thought to be important for sustaining benefits over time. These results suggest the treatment could help maintain muscle function and slow progression of disease.
The Rare Pediatric Disease Designation not only highlights the urgent medical need but also may enable Precision BioSciences to receive a Priority Review Voucher if PBGENE-DMD eventually earns FDA approval. Such vouchers are meant to encourage the development of treatments for serious pediatric diseases by offering faster review of another product in the future. They can also be sold, providing potential funding for further research.
Precision BioSciences expects to finish the final required toxicology studies soon, with plans to generate initial clinical data in 2026. Company leaders say this timeline represents important progress toward offering patients and caregivers another therapeutic option. While challenges remain before the therapy becomes available, the FDA’s designation is seen as an important milestone on that path.
For patients living with DMD, which currently has limited treatment choices, news of PBGENE-DMD’s progress and regulatory support offers cautious optimism. If successful in trials, the therapy may help slow progression of disease and improve daily life for many children and families affected by this condition.
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