25 November 2020 - Polaryx Therapeutics announced today that it has received from the U.S. FDA both rare paediatric disease and orphan drug designations for the treatment of GM2 gangliosidosis with PLX-300.
GM2 gangliosidosis, also known as Tay-Sachs and Sandhoff diseases, are ultra-rare and fatal paediatric neurodegenerative disorders caused by defects in hexosaminidase A and hexosaminidase B, key enzymes in the lysosome, respectively.