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M092
- Development stage
- Preclinical
- Lead developer
- M6P Therapeutics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
01
Overview
M092 is an adeno-associated virus (AAV) gene therapy being developed by M6P Therapeutics for the treatment of CLN2 disease (late infantile neuronal ceroid lipofuscinosis type 2), a form of Batten disease. CLN2 is caused by mutations in the TPP1 gene, leading to a deficiency in the tripeptidyl peptidase 1 (TPP1) enzyme. M092 utilizes AAV-mediated delivery of a functional TPP1 gene to restore enzyme activity. The therapy leverages M6P Therapeutics' proprietary platform designed to enhance the mannose 6-phosphate (M6P) glycosylation of the lysosomal enzyme, potentially improving cellular uptake and distribution within the central nervous system. It is currently in the preclinical stage of development.
02
Targets
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