We elevate the fundamentals.
M6P Therapeutics is a privately held biopharmaceutical company developing the next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs) by unlocking the potential of mannose 6-phosphate (M6P). Our first-in-class technology platform creates lysosomal enzymes that harness high levels of mannose 6-phosphate (M6P), which restore the enzymatic activity within the natural pathway for exogenous enzymes and gene delivery.
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 even 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 enzyme replacement therapy (ERT) 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, Pompe disease, Krabbe disease, mucopolysaccharidosis IIIB, Fabry disease, and alpha-mannosidosis. Our mission is to develop a customized therapy option for all patients with LSDs. We expect to initiate two clinical programs by early 2022.