Product Description
4D-125 drug product developed for gene therapy, which comprises an AAV capsid variant (4D-R100) carrying a codon-optimized human Retinitis Pigmentosa GTPase Regulator transgene. (Sourced from: https://clinicaltrials.gov/ct2/show/NCT04517149)
Mechanisms of Action: Gene Therapy,RPGR
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: Injection
FDA Designation: Fast Track - Retinitis Pigmentosa|Retinitis *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: 4D Molecular
Company Location: EMERYVILLE CA 94608
Company CEO: David Kirn
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: United States
Active Clinical Trial Count: 1
Highest Development Phases
Phase 2: Retinitis Pigmentosa
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
---|---|---|---|---|---|
XLRP | P2 |
Active, not recruiting |
Retinitis Pigmentosa |
2026-06-01 |
24% |