Precision BioSciences Receives FDA Rare Paediatric Disease Designation for Duchenne Muscular Dystrophy Treatment
Thursday, June 26, 2025
Precision BioSciences has been granted Rare Paediatric Disease Designation by the US Food and Drug Administration (FDA) for its investigational therapy PBGENE-DMD, aimed at treating Duchenne muscular dystrophy (DMD).
Duchenne muscular dystrophy is a serious and life-limiting genetic condition affecting approximately 15,000 individuals in the United States, mostly boys under the age of 18. The FDA’s Rare Paediatric Disease Designation is given to therapies that target conditions affecting fewer than 200,000 people and that mainly impact children.
PBGENE-DMD is based on Precision’s proprietary ARCUS® gene editing platform and is designed to address a significant unmet need in children living with DMD.
The therapy works by excising exons 45–55 of the dystrophin gene using a one-time delivery of two ARCUS nucleases via a single AAV. This approach is intended to restore the production of near full-length, functional dystrophin protein, potentially benefiting over 60% of DMD patients.
Preclinical research with PBGENE-DMD has shown promising results. The therapy demonstrated the potential to edit muscle stem cells and improve function across key skeletal and cardiac muscles in a humanised DMD mouse model. It also showed signs of long-term durability, which may support sustained improvement in muscle function.
The company is currently completing final IND-enabling toxicology studies and expects to begin clinical trials, with initial data anticipated in 2026.
Source: precisionbiosciences.com
