M 013
Alternative Names: Lenti-GBA-S1S3; M-013Latest Information Update: 12 Apr 2023
At a glance
- Originator M6P Therapeutics
- Class Gene therapies
- Mechanism of Action Gene transference; Glucosylceramidase replacements
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
- Available For Licensing Yes
Highest Development Phases
- Discontinued Gaucher's disease