Product Description
AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the beta-hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors. (Sourced from: http://investors.axovant.com/news-releases/news-release-details/axovant-gene-therapies-announces-fda-clearance-ind-axo-aav-gm2)
Mechanisms of Action: Gene Therapy,HEXA,HEXB
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: Fast Track - Gangliosidosis, GM2 *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Terence Flotte
Company Location:
Company CEO:
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
Highest Development Phases
Phase 1: Tay-Sachs Disease|Sandhoff Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| STUDY00001894 | N/A |
Active, not recruiting |
Gangliosidosis, GM2|Gangliosidoses|Tay-Sachs Disease|Sandhoff Disease |
2028-12-01 |
2024-09-27 |
Primary Endpoints |
|
| AXO-GM2-001 | P1 |
Completed |
Tay-Sachs Disease|Sandhoff Disease |
2024-05-23 |
71% |
2025-01-10 |
Patient Enrollment|Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments|Trial Status |
Recent News Events
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