We are leading experts in lysosomal storage disorders.

Below please find a few of the most recent scientific publications developed by members of the M6P clinical development team:

• A novel S1S3 phosphotransferase co-expression gene therapy platform for lysosomal disorders. Poster session at WORLDSymposium, Feb. 11, 2022.

 

• M021- A uniquely glycosylated, highly phosphorylated acid-alpha glucosidase enzyme replacement therapy for the treatment of Pompe disease. Poster session at WORLDSymposium, Feb. 11, 2022.

 

• M011 A novel highly phosphorylated β-glucocerebrosidase enzyme with broader tissue biodistribution for the treatment of Gaucher disease. Poster session at WORLDSymposium, Feb. 11, 2022.

 

• Recombinant human NAGLU with improved mannose 6-phosphorylation for Sanfilippo B syndrome. Poster session at the 16th International Symposium on MPS and Related Diseases, July 23-25, 2021.

 

• M002, a novel AAV9-mediated gene therapy in a mouse model of mucolipidosis type II. Poster session at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 11, 2021.

 

• Mucolipidosis type II AAV9 gene therapy pilot study (M002): In vivo safety of over-expressing modified GlcNAc-1-phosphotransferase (S1S3) in wild-type mice. Poster session at WORLDSymposium, Feb. 12, 2021.

 

• Phosphorylated acid β-glucosidase (M011, GCaseM6P) enzyme replacement therapy leads to better tissue distribution, cellular uptake, and efficacy in the Gaucher D409A mouse model compared to conventional α-mannosyl terminated β-glucosidase. Poster session at WORLDSymposium, Feb. 12, 2021.

 

• A new platform technology for next generation lysosomal enzyme replacement and potential gene therapy in the treatment of lysosomal diseases. Poster session at WORLDSymposium, Feb. 11, 2020.

 

• Engineering Of GlcNAc-1-Phosphotransferase For Production Of Highly Phosphorylated Lysosomal Enzymes For Enzyme Replacement Therapy.  Mol Ther Methods Clin Dev. 2017 Jun 16; 5: 59–65.