Drug Profile

M 012 - M6P Therapeutics

Alternative Names: AAV-GBA-S1S3; M-012 - M6P Therapeutics

Latest Information Update: 18 Jul 2025

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At a glance

Originator M6P Therapeutics
Class Gene therapies
Mechanism of Action Gene transference; Glucosylceramidase replacements

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Gaucher's disease
New Molecular Entity No
Available For Licensing Yes

Highest Development Phases

Preclinical Gaucher's disease

Most Recent Events

18 Jul 2025 M 012 is still in preclinical development in Gaucher's-disease in USA (Parenteral) (M6P therapeutics pipeline, July 2025)
18 Jul 2025 M6P Therapeutics plans to file IND for Gaucher's disease in second quarter of 2026 (M6P Therapeutics pipeline, July 2025)
18 Jul 2025 Pharmacodynamics data from a preclinical trial in Gaucher disease released by M6P Therapeutics

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