M082

M082 is an adeno-associated virus (AAV) gene therapy being developed by M6P Therapeutics for the treatment of CLN1 disease, also known as infantile neuronal ceroid lipofuscinosis (INCL) or Batten disease. CLN1 is a severe neurodegenerative disorder caused by mutations in the *PPT1* gene, which leads to a deficiency in the enzyme palmitoyl-protein thioesterase-1. This enzyme is essential for the degradation of palmitoylated proteins within lysosomes. M082 is designed to deliver a functional copy of the *PPT1* gene to restore enzyme activity and address the underlying cause of the disease. The program utilizes M6P Therapeutics' proprietary platform, which is designed to enhance the lysosomal targeting of the expressed enzyme through improved mannose 6-phosphate (M6P) glycosylation, potentially increasing the therapeutic efficacy of the gene therapy.