M042

M042 is an adeno-associated virus (AAV) gene therapy candidate being developed by M6P Therapeutics for the treatment of Sanfilippo syndrome type B (Mucopolysaccharidosis type IIIB; MPS IIIB). The therapy is designed to deliver a functional copy of the NAGLU gene to cells, facilitating the sustained expression and secretion of the alpha-N-acetylglucosaminidase (NAGLU) enzyme. MPS IIIB is a rare, progressive lysosomal storage disorder caused by a deficiency in NAGLU, which leads to the accumulation of heparan sulfate and subsequent neurodegeneration. M042 utilizes M6P Therapeutics' proprietary platform to enhance the mannose 6-phosphate (M6P) phosphorylation of the expressed enzyme, potentially improving its uptake and targeting to the lysosomes of affected cells. The program is currently in the preclinical stage of development.