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About Us

We Elevate the Fundamentals.

M6P Therapeutics, or M6PT, is a life sciences company whose mission is clear and simple: to translate the M6PT innovation into more effective next generation therapies for individuals affected by lysosomal storage disorders (LSDs) – to relieve suffering, to restore health, to increase hope, and to improve lives.

M6PT has the team to do it. We are skilled and experienced in the discovery, non-clinical, clinical, and commercial development of drugs that treat rare diseases.

M6PT has the tools to do it. Our S1S3 co-expression platform technology enables consistently well phosphorylated next generation lysosomal enzymes with mannose 6-phosphate (M6P) for developing LSD treatments.

M6PT has the drive to do it. As stewards of a breakthrough technology, we push forward to develop and deliver next generation treatments to individuals affected, caregivers, healthcare providers, physicians, and global health systems.

Company Statistics

Founded In

Dec. 20 0

Status

Private, Venture Backed

Employess

0

Founded In

St. Louis, Mo

M6PT: Building a Biotech Company Efficiently

2017
Breakthrough Discovery:
Drs Lin Liu and Stuart Kornfeld at University of Washington in St. Louis created the first modified GlcNAc-1-phosphotransferase, named S1S3 variant, that efficiently phosphorylates lysosomal enzymes. The results were published in Mol Ther Methods Clin Dev.
2017
2017
M6PT's Founding:
Dr. Kornfeld and Cuong Do co-founded M6PT following the breakthrough research conducted by Dr. Kornfeld and his team at Washington University.
2017
2019
S1S3 Co-Expression Platform Creation:
M6PT team successfully created a platform technology based on the breakthrough finding of S1S3 phosphotransferase.
2019
2020
Series A Financing Round:
M6PT raises Series A financing.
2020
2020
Regulatory Updates:
M6PT announced that the U.S. Food and Drug Administration (FDA) granted six rare pediatric disease designations (RPDDs) for various programs within its development pipeline for LSDs, including four enzyme replacement therapy and two gene therapy programs. In addition, the FDA granted two orphan drug designations (ODDs) for its gene therapy programs for Gaucher disease and mucolipidosis. Read the full release here.
2020
2021
2021 Preclinical Data:
M6PT presented in vivo preclinical data for enzyme replacement therapy and gene therapy product at four scientific and family conferences

  • WORLDSymposium™ 2021 Researchers presented promising preclinical data in both Gaucher disease and mucolipidosis type II (ML II). Read the full release here
  • American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting. 2021: M6PT presented preclinical proof-of-concept data for M002, its gene therapy candidate for ML II. Read the full release
  • 16th Annual International Symposium of MPS and Related Diseases (MPS 2021): M6PT presented pre-clinical proof-of-concept data on M041, an enzyme replacement therapy in development, for the Treatment of Sanfilippo B Syndrome. Read the full release
  • 17th International Congress on Neuronal Ceroid Lipofuscinosis (NCLs): M6PT presented preclinical data on Batten disease. Read the full release
2021
2021
2021 Series A+ Financing Round:
M6PT adds additional funds in a Series A+ financing round.
2021
2022
Prestigious Award for Dr. Kornfeld:
Cofounder Dr Stuart Kornfeld is awarded the 2022 Roscoe O Brady Innovation & Accomplishment Award at WORLD
2022
2022
2022 Preclinical Data:
M6PT researchers presented promising preclinical data at the 18th Annual WORLDSymposium™ 2022. In four poster presentations, including two Contemporary Forum presentations, M6PT reported preclinical efficacy results for LSDs, including Sanfilippo B syndrome, Gaucher disease, and Pompe disease. Read the full release
2022