PBGENE-DMD
Duchenne Muscular Dystrophy (DMD)
Phase 1/2IND Cleared, Trial Initiation Pending
Key Facts
Indication
Duchenne Muscular Dystrophy (DMD)
Phase
Phase 1/2
Status
IND Cleared, Trial Initiation Pending
Company
About Precision BioSciences
Precision BioSciences is a clinical-stage biotech leveraging its proprietary ARCUS genome editing platform to develop potentially curative in vivo therapies for genetic diseases. The company has advanced two wholly-owned programs into Phase 1/2 trials: PBGENE-HBV for chronic Hepatitis B and PBGENE-DMD for Duchenne Muscular Dystrophy, both supported by key regulatory designations. Its strategy combines advancing these core assets with strategic partnerships to validate and fund the ARCUS platform, positioning it in the competitive but high-potential gene editing landscape.
View full company profileTherapeutic Areas
Other Duchenne Muscular Dystrophy (DMD) Drugs
| Drug | Company | Phase |
|---|---|---|
| DT-DEC01 | Dystrogen Therapeutics | Phase 1 |
| Skeletal Muscle Regeneration Program | IPS HEART | Pre-clinical |
| KYMBEE (deflazacort) | Upsher-Smith | Approved |
| ALY688ER | Allysta Pharmaceuticals | Phase 1 |
| AVGN7 + AVGND | AAVogen | Pre-clinical |
| Myomatrix Stabilization Program | Strykagen | Preclinical |
| RIPPLE™ (implied) | SonoThera | Preclinical |
| Vamorolone (AGAMREE) | ReveraGen BioPharma | Approved |
| AGAMREE (vamorolone) | Catalyst Pharmaceuticals | Marketed |
| EDG-5506 | Edgewise Therapeutics | Phase 2 |
| ELEVIDYS (delandistrogene moxeparvovec-rokl) | Sarepta Therapeutics | Marketed / Phase 3 |
| SRP-5051 (vesleteplirsen) | Sarepta Therapeutics | Phase 2 |