M072

M072 is an adeno-associated virus (AAV) gene therapy being developed by M6P Therapeutics for the treatment of Tay-Sachs disease. It utilizes an engineered hexosaminidase enzyme, HexM, which is designed to restore the catabolism of GM2 gangliosides in the central nervous system. Tay-Sachs disease is a lysosomal storage disorder caused by mutations in the HEXA gene, leading to a deficiency in the HexA enzyme and subsequent toxic accumulation of GM2 gangliosides in neurons. M072 aims to deliver the genetic instructions for HexM to cells, thereby bypassing the deficiency and preventing neurodegeneration. The program is currently in the preclinical stage.