Search jobs 11-May-2021 M6P Therapeutics Presents M002 Gene Therapy Preclinical Proof-of-Concept Data for the Treatment of Mucolipidosis Type II at the ASGCT 24th Annual Meeting
- Preclinical data provide proof of concept for M002 to restore cellular uptake and lysosomal delivery and attenuate phenotype in mouse models of mucolipidosis type II -
- M002 leverages the Company’s proprietary S1S3 bicistronic platform technology in an AAV9 gene therapy to restore mannose 6-phosphate recognition marker on lysosomal enzymes -
ST. LOUIS, Mo. (BUSINESS WIRE) M6P Therapeutics (“the Company”), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of preclinical proof-of-concept data for M002, its gene therapy candidate for mucolipidosis type II (MLII), at the American Society of Gene & Cell Therapy (ASGCT) 24