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CRISPR Therapeutics DMD program
- Development stage
- Preclinical
- Lead developer
- Vertex
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous, Intramuscular, Ex Vivo
01
Overview
The CRISPR Therapeutics DMD program is an investigational CRISPR/Cas9-based gene-editing therapy being developed for the treatment of Duchenne muscular dystrophy (DMD). Developed in collaboration with Vertex Pharmaceuticals, the program utilizes CRISPR/Cas9 technology to target the DMD gene, aiming to restore the production of functional dystrophin protein. The approach typically involves precisely editing the genetic mutations that cause DMD—such as by skipping specific exons to restore the reading frame—thereby addressing the underlying genetic cause of the muscle-wasting disease. Vertex Pharmaceuticals leads the development and commercialization of this program under an expanded collaboration agreement with CRISPR Therapeutics. The program is currently in the preclinical research stage.
- Other names
- CRISPR/Vertex DMD program
02
Targets
DNM1L (Dynamin-related protein 1)
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