M161

M161 is an enzyme replacement therapy (ERT) being developed by M6P Therapeutics for the treatment of Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. MPS IVA is a lysosomal storage disorder caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase (GALNS), which leads to the accumulation of keratan sulfate and chondroitin-6-sulfate in various tissues, particularly bone and cartilage. M161 is designed to replace the deficient GALNS enzyme, thereby facilitating the degradation of these glycosaminoglycans and potentially alleviating the skeletal and systemic manifestations of the disease. The drug utilizes M6P Therapeutics' proprietary platform to enhance the phosphorylation of the enzyme, aiming for improved cellular uptake and lysosomal targeting.