M6P Therapeutics

What M6P Therapeutics does

M6P Therapeutics is a privately held, venture-backed biotechnology company founded in 2017 and headquartered in St. Louis, Missouri, developing next-generation enzyme replacement therapies (ERTs) and gene therapies for lysosomal storage disorders (LSDs). The company's scientific foundation is its proprietary S1S3 co-expression platform technology, which uses a truncated variant of GlcNAc-1-phosphotransferase discovered by co-founders Dr. Stuart Kornfeld and Dr. Lin Liu at Washington University to predictably enhance mannose 6-phosphate (M6P) content on lysosomal enzymes. This elevated M6P content improves binding to CI-M6P receptors, enabling more efficient cellular uptake and broader tissue biodistribution than conventional ERTs.

The pipeline spans six disclosed programs across both modalities: M021 (Pompe disease ERT), M011 (Gaucher disease ERT), M012 (Gaucher gene therapy), M052 (Fabry gene therapy), M002 (Mucolipidosis II/III gene therapy), and M041 (Sanfilippo B syndrome ERT). The company has secured six Rare Pediatric Disease Designations and two Orphan Drug Designations from the FDA, presented preclinical data at conferences including WORLDSymposium and ASGCT, and targeted 2023 for entry into the clinic. Research collaborations with Washington University and patient-advocacy partnerships with organizations such as the Mucolipidosis Collaborative Network and Cure Mucolipidosis support both platform development and future commercialization planning.

M6P Therapeutics is pre-commercial with no approved products and no disclosed revenue. Its revenue model is described as a one-time license/biopharmaceutical product revenue structure, with future go-to-market execution expected via specialty pharmacy and rare-disease specialty distributor channels. The leadership team includes President & CEO Pawel Krysiak (formerly of BioMarin and Genzyme), CSO Hung Do (formerly CSO of Amicus Therapeutics), and Chairman Cuong Do (former Samsung Global Strategy Group President).

Name

M6P Therapeutics

Legal name

M6P Therapeutics, Inc.

Website

https://m6ptherapeutics.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

M6P Therapeutics is a privately held biotechnology company developing next-generation enzyme replacement therapies and gene therapies for lysosomal storage disorders using its proprietary S1S3 co-expression platform that enhances mannose 6-phosphate content on lysosomal enzymes.

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Enzyme Replacement & Metabolic Disorder Therapies (HLAIAIAB)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Recombinant Protein Therapeutics (enzymes, hormones, growth factors) (HLAAAAAB), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)

Keywords

• Lysosomal storage disorders
• Enzyme replacement therapies
• Gene therapy pipeline
• Rare disease therapeutics
• Biopharmaceutical development

Headquarters

HQ city

St Louis

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Others

Revenue model

1. Biopharmaceutical Product Revenue: M6P Therapeutics is a privately held, venture-backed biotechnology company developing next-generation enzyme replacement therapies (ERTs) and gene therapies for lysosomal storage disorders (LSDs). As a pre-commercial company in the clinical development stage, they do not currently have approved products or revenue. The company plans to initiate their first clinical program in 2023.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

M6P Therapeutics is a privately held, venture-backed biotechnology company developing next-generation enzyme replacement therapies (ERTs) and AAV gene therapies for lysosomal storage disorders (LSDs) such as Pompe, Gaucher, Fabry, Mucolipidosis II/III, and Sanfilippo B. Its products leverage the proprietary S1S3 co-expression platform that enhances mannose 6-phosphate content on lysosomal enzymes to improve cellular uptake via the CI-MPR pathway and enable efficient cross-correction. The company is pre-commercial with no approved products on the market and planned to initiate its first clinical program in 2023.

Product overview

M6P Therapeutics is a privately held biotechnology company developing a portfolio of next-generation enzyme replacement therapies (ERTs) and gene therapies for lysosomal storage disorders (LSDs). The company's core asset is its proprietary S1S3 co-expression platform technology, which enhances mannose 6-phosphate (M6P) content on lysosomal enzymes. Lead programs include M021 (Pompe disease ERT), M011 (Gaucher disease ERT), M012 (Gaucher gene therapy), M052 (Fabry gene therapy), M002 (Mucolipidosis II/III gene therapy), and M041 (Sanfilippo B syndrome ERT). The platform enables improved biodistribution and cellular uptake of therapeutic proteins.

Differentiator

Problem solved

Functional benefit

Products and services

• M021
• M011
• M012
• M052
• M002
• M041

Quantifiable outcome

• M021 demonstrated significantly better muscle targeting in Pompe mice than standard of care ERT
• +3 more outcomes

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered core.

• Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML)coreStrategic or Co-development Partner · 1 October 2021M6P Therapeutics met with both the Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) to provide updates on the status of M6PT's preclinical development programs for ML II/ML III, and to discuss collaboration efforts to develop and foster the discovery and development of new therapies for mucolipidosis.
• Washington University Medical SchoolcoreStrategic or Co-development Partner · 1 February 2021Washington University is the home institution of co-founders Dr. Stuart Kornfeld and Dr. Lin Liu, who discovered the S1S3 technology. The company maintains ongoing research collaboration with Washington University scientists including Dr. Patricia Dickson and Dr. Mark Sands.

Scale indicators4 records

Recent moves6 records

Expansion highlights5 records

Emerging players

• 4D Molecular Therapeutics:
• Denali Therapeutics: Denali Therapeutics develops engineered enzyme replacement therapies using its proprietary transport vehicle (TV) platform for crossing the blood-brain barrier to address CNS manifestations of LSDs. Closely comparable to M6P's mission of improving lysosomal enzyme delivery to difficult-to-reach tissues.
• Regenxbio: Regenxbio develops AAV gene therapy platforms (NAV Technology) and has active rare disease programs including for mucopolysaccharidoses. Relevant comparator for M6P's M002 (AAV9 ML II/III) gene therapy approach given shared AAV delivery infrastructure.
• Prevail Therapeutics: Prevail Therapeutics (acquired by Eli Lilly in 2021) develops AAV-based gene therapies for lysosomal diseases including Gaucher (PR001) and other CNS-targeted programs. Direct overlap with M6P's gene therapy strategy targeting CNS manifestations of LSDs via AAV delivery.

Broad incumbents

• Sanofi (Genzyme): Sanofi, through its Genzyme Rare Disease franchise, markets the dominant standard-of-care ERTs that M6P Therapeutics aims to improve upon: Cerezyme (Gaucher), Myozyme/Lumizyme (Pompe), Fabrazyme (Fabry), and Aldurazyme (MPS I). Broad incumbent with established commercial infrastructure and physician relationships across all LSDs.
• BioMarin Pharmaceutical: BioMarin develops and commercializes enzyme and gene therapies for rare diseases including Vimizim (MPS IVA), Naglazyme (MPS VI), Aldurazyme co-promote, and Pompe disease programs. M6P's CEO Pawel Krysiak previously served as Area Director & General Manager at BioMarin, providing direct operational overlap.
• Takeda Pharmaceutical (Shire): Takeda, through its acquisition of Shire, markets Replagal (agalsidase alfa, Fabry disease) and Elaprase (Hunter syndrome/MPS II). Major LSD franchise owner with global commercial reach and active gene therapy investments in rare diseases.

Direct peers

• Amicus Therapeutics: Amicus Therapeutics develops therapies for rare diseases including Pompe (AT-GAA, in late-stage development), Fabry (migalastat), and other LSDs. Direct competitor to M6P Therapeutics across multiple LSD programs and previously acquired Callidus Biopharma (founded by M6P's founder Cuong Do) for its Pompe disease drug.
• Ultragenyx Pharmaceutical: Ultragenyx develops therapies for rare and ultra-rare diseases including MPS VII (Mepsevii), gene therapy programs for rare metabolic diseases, and enzyme replacement candidates. Direct competitor pursuing next-generation approaches to LSDs and is comparable in pipeline stage and strategy.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about M6P Therapeutics