AXO-AAV-GM2
Phase 1Gene therapy
AXO-AAV-GM2 is one of 6 small molecule programs targeting Sandhoff disease. It is currently in Phase 1.
1
Indications
2
Total Trials
1
Active Trials
5
Competitors
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
5 competitors in Sandhoff Disease
#DrugCompanyPhaseTrials
1Cytoxan
BBaxter Healthcare Corporation
Approved
Busulfex
Akero TherapeuticsApproved
Wellcovorin
GGlaxo Dba Glaxosmithkline
Phase 2
Daraprim
FFondazione Matilde Tettamanti Menotti De Marchi Onlus
Phase 1
Wellcovorin
EEastern Cooperative Oncology Group
Phase 1
AXO-AAV-GM2
TTerence Flotte
Phase 1
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for AXO-AAV-GM2. EMBL-EBI & Wellcome Sanger Institute.
- [4]
Details
Drug Info
Quick Facts
- Modality
- Gene therapy[2]
- Highest Phase
- Phase 1[1]
- Company
- TTerence Flotte