ST. LOUIS– M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced today the presentation of promising preclinical data at the 17th Annual WORLDSymposium™, a research conference dedicated to lysosomal diseases being held virtually Feb. 8-12, 2021. In two separate poster presentations, M6P Therapeutics’ researchers report preclinical efficacy and safety results for Gaucher disease and mucolipidosis II (MLII) respectively.
M6P Therapeutics developed its novel bicistronic-S1S3 technology platform to maximize the natural mechanism for trafficking enzymes to lysosomes by enhancing phosphorylation of lysosomal enzymes for both recombinant enzyme and gene therapies. By increasing levels of mannose 6-phosphate, a specialized carbohydrate structure found on the surfaces of lysosomal enzymes, therapies created through the Company’s proprietary platform are designed to improve biodistribution and cellular uptake of therapeutic enzymes to target tissues and to enable efficient cross-correction for gene therapies.
“Targeting the mannose 6-phosphate pathway for recombinant enzymes and gene therapies shows tremendous promise because it may lead to better tissue distribution, enhanced cellular uptake, and greater efficacy, compared to current therapeutic approaches for lysosomal storage disorders,” commented Lin Liu, Ph.D., Director of R&D at M6P Therapeutics. “The preclinical data we are presenting at the WORLDSymposium suggest that increasing the mannose 6-phosphate content on lysosomal enzymes may enable targeting of more cells within the body, possibly easing the disease burden for patients living with these devastating disorders.”
