M043

M043 is an adeno-associated virus (AAV) gene therapy being developed by M6P Therapeutics for the treatment of Sanfilippo syndrome type B (Mucopolysaccharidosis type IIIB; MPS IIIB). The therapy utilizes M6P Therapeutics' proprietary S1S3 co-expression platform, which involves the co-expression of the lysosomal enzyme alpha-N-acetylglucosaminidase (NAGLU) with the GlcNAc-1-phosphotransferase subunits S1 and S3. This engineering is designed to enhance the mannose-6-phosphate (M6P) glycosylation of the NAGLU enzyme, thereby improving its targeting to the mannose-6-phosphate receptor (M6PR) and subsequent lysosomal delivery and cellular uptake. By increasing the efficiency of enzyme delivery to affected tissues, M043 aims to reduce the accumulation of heparan sulfate and address the neurodegenerative symptoms of MPS IIIB.