PBGENE-DMD
Duchenne Muscular Dystrophy (DMD)
Phase 1/2Active - IND Cleared; Clinical trial initiation
Key Facts
Indication
Duchenne Muscular Dystrophy (DMD)
Phase
Phase 1/2
Status
Active - IND Cleared; Clinical trial initiation
Company
About Precision BioSciences
Precision BioSciences leverages its novel ARCUS genome editing platform to create precise gene insertion, knockout, and repair therapies. The company has achieved significant clinical milestones, including FDA Fast Track and Orphan Drug designations for its lead programs, and has demonstrated early clinical safety and antiviral activity for PBGENE-HBV. Its strategy focuses on advancing its wholly-owned pipeline in genetic diseases while maintaining partnerships, such as with TG Therapeutics, to validate its platform in other therapeutic areas.
View full company profileTherapeutic Areas
Other Duchenne Muscular Dystrophy (DMD) Drugs
| Drug | Company | Phase |
|---|---|---|
| AOC Program | Avidity Biosciences | Clinical |
| AGAMREE® (vamorolone) | Catalyst Pharmaceuticals | Approved/Commercial |
| SRP-9001 (delandistrogene moxeparvovec) | Sarepta Therapeutics | Approved |
| Deramiocel (CAP-1002) | Capricor Therapeutics | Phase 3 |
| RGX-202 | REGENXBIO | Phase 1/2 |
| DT-DEC01 | Dystrogen Therapeutics | Phase 1 |