M6P Therapeutics Presented Data on M041, A Recombinant Enzyme Therapy, for the Treatment of Sanfilippo B Syndrome at MPS 2021
Retrieved on:
Wednesday, July 28, 2021
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical trials, Enzymes, Branches of biology, NAGLU, Alpha-N-acetylglucosaminidase, Lysosomal storage disease, Glucocerebrosidase, Mannose 6-phosphate, Mannose, Lysosome, Glycoside hydrolase family 89, Mucopolysaccharidosis, Sanfilippo B Syndrome, M6P Therapeutics, SANFILIPPO B SYNDROME, M6P THERAPEUTICS
These data illustrate M041s potential as a recombinant enzyme therapy utilizing a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with improved mannose 6-phosphorylation.
Key Points:
- These data illustrate M041s potential as a recombinant enzyme therapy utilizing a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with improved mannose 6-phosphorylation.
- There are currently no approved therapies for Sanfilippo B syndrome, also known as MPS IIIB.
- Sanfilippo B syndrome is characterized by a defect in the NAGLU gene providing instructions for producing the enzyme alpha-N-acetylglucosaminidase.
- M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).