Scribe Therapeutics, a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced its participation in the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place May 13-17, 2025, at the Ernest N. Morial Convention Center in New Orleans, LA.
Scribe Therapeutics will deliver two presentations that showcase the company’s ongoing advances enabled by its CRISPR approach to engineer novel in vivo genetic medicines. In one demonstration of potent therapeutic application, Scribe Therapeutics will present novel genome editing strategies using its X-Editor (XE) technology to target mutations in Duchenne Muscular Dystrophy (DMD), a severe neuromuscular disorder that can lead to respiratory and cardiac failure. Findings from this study will showcase XE as an effective and versatile platform for the treatment of DMD and other inherited muscle disorders.
The joint presentation will describe work by Scribe Therapeutics and Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, to engineer self-inactivating adeno-associated viral (AAV) vectors featuring Scribe Therapeutics’s CRISPR-based XE with self-targeting guide RNAs and results demonstrating potent, transient, and controllable on-target in vivo editing in the central nervous system of mice. The companies recently reached a success milestone in their collaboration to address neurological and neuromuscular diseases.
Read More: New Gene Therapies for Duchenne Muscular Dystrophy
