Product Description
4D-310 is a novel adeno-associated virus (AAV) gene therapy comprised of two active components: the capsid (4D-C102) and the transgene cassette, which encodes a codon-optimized full length human GLA transgene driven by the CAG promoter. 4D-310 has been engineered so that it cannot replicate (replication incompetent). (Sourced from: https://clinicaltrials.gov/ct2/show/NCT04519749)
Mechanisms of Action: Gene Therapy,GLA
Novel Mechanism: No
Modality: Gene Therapy
Route of Administration: Intravenous
FDA Designation: Fast Track - Fabry Disease *
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: Australia, Taiwan, United States
Active Clinical Trial Count: 2
Highest Development Phases
Phase 2: Fabry Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
---|---|---|---|---|---|
4D-310-C001 | P2 |
Active, not recruiting |
Fabry Disease |
2026-01-01 |
32% |
4D-310-C002 | P2 |
Active, not recruiting |
Fabry Disease |
2026-01-01 |
32% |