Feb. 3, 2021 12:30 UTC

ST. LOUIS--(BUSINESS WIRE)-- M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced its scientific advisory board (SAB) that will support the Companys mission of translating its innovative bicistronic-S1S3 technology platform into best-in-class therapies that address unmet needs within the LSD community. The Companys platform enables improved biodistribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies.

As we work to advance our robust pipeline, we seek the input and support of a world-class team of scientific advisors with deep expertise in genetics, rare diseases, and lysosomal storage and metabolic disorders in particular, said Pawel Krysiak, president and chief executive officer of M6P Therapeutics. The collective insights, knowledge, commitment, and expertise of our scientific advisory board will help us translate this high science into potential medical benefit for the individuals affected by these serious conditions.

By combining the substantial expertise of the SAB with the expertise of the Companys internal R&D team in recombinant enzyme and gene therapies, M6P Therapeutics is well positioned to rapidly advance its deep pipeline of LSD programs. The members of the SAB are:

M6P Therapeutics bicistronic-S1S3 technology platform enhances mannose 6-phosphate content on lysosomal enzymes for both recombinant enzyme and gene therapies, which improves enzyme uptake across target tissues, said Stuart Kornfeld, MD, M6P Therapeutics co-founder and chairman of its SAB. With promising pre-clinical data across numerous LSD programs, this innovation can potentially translate into new and more efficacious treatments, reduced immunogenicity, and more efficient dosing regimens.

About M6P Therapeutics

M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its bicistronic-S1S3 platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

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M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board - BioSpace