Product Description
LYS-SAF302delivers a functional copy of the human SGSH gene directly to brain cells using the adeno-associated virus carrier, AAVrh.10, which has a particular tropism for cells of the CNS. (Sourced from: https://www.lysogene.com/lys-saf302/)
Mechanisms of Action: Gene Therapy,SGSH
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: Fast Track - Mucopolysaccharidosis III *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Lysogene SA
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count: 1
Highest Development Phases
Phase 3: Mucopolysaccharidosis
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| 2018-000195-15 | P3 |
Active, not recruiting |
Mucopolysaccharidosis |
2025-04-22 |