What's Happening?
Precision BioSciences, Inc. has announced a late-breaking poster presentation at the 30th Annual International Congress of the World Muscle Society, showcasing preclinical data for PBGENE-DMD, a gene editing therapy for Duchenne Muscular Dystrophy (DMD). The data highlights PBGENE-DMD's ability to improve muscle function through increased dystrophin expression and satellite stem cell editing. The company plans to file an IND and/or CTA by the end of 2025, with clinical data expected in 2026.
Why It's Important?
DMD is a severe genetic disorder with limited treatment options, affecting muscle function and leading to early mortality. PBGENE-DMD represents a potential breakthrough in gene editing therapy, offering a first-in-class approach to restore dystrophin protein expression in up to 60% of DMD patients. The promising preclinical results suggest significant improvements in muscle function, which could transform the treatment paradigm for DMD. Successful development and commercialization of PBGENE-DMD could provide a much-needed therapeutic option for patients and families affected by this debilitating disease.
What's Next?
Precision BioSciences is advancing final toxicology studies and preparing for regulatory filings by the end of 2025. The company anticipates initial clinical data in 2026, which will be crucial for further development and potential approval. Precision will continue to engage with the scientific community and regulatory bodies to ensure the successful progression of PBGENE-DMD. The company is committed to addressing the unmet needs in DMD treatment and leveraging its ARCUS platform for broader applications.
