M6P Therapeutics is an early stage biotech research and development company founded by leaders in M6P research. The company is located in St. Louis, Missouri aims to develop new gene therapies and enzyme replacement therapies for lysosomal storage disorders based on a recent scientific breakthrough by researchers Stuart Kornfeld and Lin Liu.
Drs. Kornfeld and Liu recently achieved a feat no one thought possible — they successfully created a mechanism to regulate the content of M6P on lysosomal enzymes. This first-ever platform to enhance M6P content for all lysosomal enzymes means it may soon be possible to treat even LSDs where the required enzymes have naturally low M6P levels, such as MPS IIIB and alpha-mannosidosis. The approach can also improve treatment options for other LSDs.
Our initial research programs are focused on Gaucher disease, Pompe disease, Krabbe disease, mucopolysaccharidosis IIIB, Fabry disease, and alpha-mannosidosis. We aim to expand to additional lysosomal diseases over time. All of our programs are currently in the preclinical stage. We expect to have programs ready for clinical development by 2021.