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Developing Next Generation Therapies for Lysosomal Storage Disorders

A New Path

Unlocking the potential of mannose 6-phosphate (M6P), our breakthrough platform technology drives the development of next generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs). Our mission is to translate the M6PT innovation into more effective next generation therapies for individuals affected by LSDs – to relieve suffering, to restore health, to increase hope, and to improve lives.

A New Path

Unlocking the potential of mannose 6-phosphate (M6P), our breakthrough platform technology drives the development of next generation enzyme replacement and for lysosomal storage disorders (LSDs). Our mission is to translate the M6PT innovation into more effective next generation therapies for individuals affected by LSDs – to relieve suffering, to restore health, to increase hope, and to improve lives.

Our Main Focus

Our innovative S1S3 co-expression platform technology enables us to develop next generation, best-in-class, targeted enzyme replacement therapy (ERT) or gene therapy products to treat lysosomal storage disorders (LSDs).

What Are Lysosomal Storage Disorders (LSDs)?

Lysosomal storage disorders (LSDs) are a group of more than 50 rare inherited diseases that are associated with life-threatening, life-shortening, and life-sapping complications. Most LSDs do not have treatments. While some do, the treatments may not adequately address the signs, symptoms, or progression of the disease for all individuals affected. Read More

Mannose 6-Phosphate (M6P)

Mannose 6-phosphate (M6P) is a specialized carbohydrate structure found on lysosomal enzymes. M6P enables newly synthesized lysosomal enzymes to bind to M6P receptors in Golgi for delivery of the lysosomal enzymes to lysosome within cells. There are also M6P receptors on the cell surface of most, if not all cells, that can bind exogenous M6P-bearing lysosomal enzymes to enable their cellular uptake and delivery to lysosomes.

S1S3 Co-expression Platform Technology

Our innovative S1S3 co-expression platform technology enhances mannose 6-phosphate (M6P) content on lysosomal enzymes to increase lysosomal targeting to develop both enzyme replacement therapies (ERTs) and gene therapies. Read More

Pipeline

We are developing next generation enzyme replacement therapies (ERTs) and gene therapies to treat LSDs and are working as quickly and efficiently as possible to meet the urgent needs of those living with these serious, life-threatening rare diseases.

M6P Therapeutics Presents Promising Preclinical Data in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ 2022

February 11, 2022

– Study Results Highlight Potential Utility of Targeting Mannose 6-Phosphate Pathway to Treat Multiple Lysosomal Storage Disorders – ST. LOUIS, Mo., – February 11, 2022 – M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of promising preclinical …

M6P Therapeutics to Participate in Key Event Highlights at WORLDSymposium™ 2022 and Present Preclinical Data Across Multiple Lysosomal Diseases

February 2, 2022

– M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company Co-founder of M6PT, Dr. Stuart Kornfeld, to receive the 2022 Roscoe O. Brady Award for Innovation and Accomplishment – – Preclinical data across three lysosomal disorders demonstrate the broad potential of the Company’s therapies developed under its novel S1S3 co-expression technology platform …

Update from Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) on Mucolipidosis Type II

October 9, 2021

The Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) announced that they will be able to initiate the Natural History studies on a model of mucolipidosis type II (ML II) in 2022. M6PT met with both the MCRN and Cure ML to provide an update on the status of M6PT’s preclinical development programs for …

M6P Therapeutics to Present at the 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis

October 8, 2021

ST. LOUIS, Mo., – October 8, 2021 – M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced the Company’s participation at the 17th International Congress on Neuronal Ceroid Lipofuscinosis (NCLs), or NCL2021, being held from October 6-10, 2021, …

Dr. Stuart Kornfeld, M6P Therapeutics’ Co-Founder, to Receive the Roscoe O. Brady Award For Innovation and Accomplishment at WORLDSymposium™ 2022

September 30, 2021

– The Roscoe O. Brady Award is granted to individuals for their substantial body of innovation and achievement in the field of lysosomal storage disorder (LSDs) research and therapy – ST. LOUIS, Mo., – September 30, 2021 – M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for …

M6P Therapeutics Presented Data on M041, A Recombinant Enzyme Therapy, for the Treatment of Sanfilippo B Syndrome at MPS 2021

July 28, 2021

– Preclinical data provide proof of concept for recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) demonstrating improved mannose 6-phosphorylation – – Leveraging the Company’s proprietary S1S3 bicistronic expression platform, M041 is co-expressed human NAGLU and S1S3, resulting in NAGLUM6P – – With increased efficiency to phosphorylate lysosomal enzymes, M041 is a potential novel recombinant enzyme therapy for Sanfilippo B …

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Individuals & families

We Are Dedicated to the Individuals We Serve.

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Working at M6PT

We are passionate about science and its ability to improve health for and give hope to humans affected by disease. This is a unique opportunity to join an innovative, dynamic, and entrepreneurial company committed to developing therapies to treat individuals affected by LSDs.

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