We elevate the fundamentals.
M6P Therapeutics is a privately held, venture-backed life sciences company developing first-in-class, next-generation, targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs) by unlocking the potential of mannose 6-phosphate (M6P). Our first-in-class bicistronic-S1S3 technology platform enhances M6P content on lysosomal enzymes for both recombinant enzyme and gene therapies, which improves enzyme uptake across target tissues, allowing us to restore the enzymatic activity within this natural pathway.
Based on a scientific breakthrough by researchers Stuart Kornfeld and Lin Liu in 2017, we successfully created a technology platform to regulate the M6P levels on lysosomal enzymes, allowing us to potentially treat LSDs where the required enzymes have naturally low M6P levels, such as mucopolysaccaridosis IIIB (MPS IIIB) and alpha-mannosidosis. Because M6P has greater affinity to bind to the M6P receptors on the lysosome, we are able to develop targeted, individualized, and effective medicines, either recombinant enzyme therapy or gene therapy, to treat the specific LSD; we also have the opportunity to improve treatment options for other LSDs that currently have approved therapies.
With our ability to regulate M6P, we apply a rational approach and decades of battle-tested experience to identify the right modality and the right dosage at the right time for all patients with LSDs. We are translating high science into valuable investigational therapies to treat LSDs, with the potential to relieve suffering, restore health, and improve lives.
Our initial research programs are focused on Gaucher disease, Fabry disease, mucopolysaccharidosis IIIB, and mucolipidosis II (MLII), and plans to initiate its first clinical program in 2022. Below please find a full pipeline of our development programs.