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Scientific Publications

Below please find a few of the most recent scientific publications developed by members of the M6PT clinical development team. The findings published below have been central to the creation, development, and optimization of our S1S3 co-expression platform technology. Our scientists are working to discover and develop next generation therapies for individuals with lysosomal storage disorders (LSDs), a family of more than 50 rare inherited diseases.
  1. A novel S1S3 phosphotransferase co-expression gene therapy platform for lysosomal disorders. Poster session at WORLDSymposium, Feb. 11, 2022.
  2. 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.
  3. 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.
  4. Co-expression of S1S3 phosphotransferase in production cell line improves mannose 6-phosphorylation and cellular uptake of alpha-N-acetylglucosaminidase (Sanfilippo syndrome type B). Poster session at WORLDSymposium, Feb. 11, 2022.
  5. 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.
  6. 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.
  7. 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.
  8. 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.
  9. 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.
  10. 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.