Product Description
FLT201 is an investigational liver-directed AAV gene therapy in preclinical development for the treatment of Gaucher disease Type 1. FLT201 contains a liver-specific promoter and a GBA1 sequence that expresses FreelineÕs novel, proprietary GCase variant 85, which has a 20-fold longer half-life at lysosomal pH conditions than wild-type GCase protein. (Sourced from: https://www.freeline.life/investors/newsroom/freeline-announces-orphan-drug-designations-for-flt201-for-the-treatment-of-gaucher-disease/)
Mechanisms of Action: Gene Therapy,GBA1
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: Orphan Drug - Gaucher Disease *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Freeline
Company Location: HERTFORDSHIRE X0 SG1 2FX
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Brazil, Germany, Israel, Spain, United Kingdom, United States
Active Clinical Trial Count: 4
Highest Development Phases
Phase 2: Gaucher Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| GALILEO-2 | P2 |
Recruiting |
Gaucher Disease |
2029-05-01 |
|
| FLT201-02 | P2 |
Unknown Status |
Gaucher Disease |
2029-03-31 |
|
| Galileo-1 | P2 |
Active, not recruiting |
Gaucher Disease |
2025-03-02 |
|
| GALILEO-1 | P1 |
Active, not recruiting |
Gaucher Disease |
2024-12-04 |