updated: Aug 30, 2021
August 30, 2021 (Newswire.com) –
Polaryx Therapeutics, Inc. (“Polaryx”), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.
GM2 Gangliosidoses, also known as Tay-Sachs and Sandhoff diseases, are ultra-rare and fatal pediatric neurodegenerative disorders caused by defects in Hexosaminidase A (HEXA) and Hexosaminidase B (HEXB), key enzymes in the lysosome, respectively. These genetic defects lead to abnormal accumulation of gangliosides, resulting in severe progressive neurodegeneration, seizures, loss of mobility, hearing, and vision, and early death. There is no cure for these diseases and the only treatment is…