Drug intelligence / Profile preview

M052

Development stage
Preclinical
Lead developer
M6P Therapeutics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravenous
01

Overview

M052 is an adeno-associated virus (AAV) gene therapy candidate being developed by M6P Therapeutics for the treatment of Fabry disease. The therapy is designed to deliver a functional copy of the *GLA* gene to enable the sustained production of the alpha-galactosidase A (GLA) enzyme. M052 utilizes the company's proprietary bicistronic vector platform, which co-expresses the GLA enzyme along with a GlcNAc-1-phosphotransferase. This approach is intended to enhance the mannose 6-phosphate (M6P) tagging of the expressed enzyme, thereby improving its lysosomal targeting and facilitating cross-correction in affected tissues. By optimizing the phosphorylation of the enzyme, M052 aims to provide a more effective therapeutic option compared to conventional gene therapies or standard enzyme replacement therapies. It is currently in the preclinical stage of development.

02

Targets

GLA (Alpha-galactosidase A)

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