FDA grants FAST-TRACK STATUS FOR CUTX-101 TREATMENT OF MD

MD, also known as curly-hair syndrome, a rare congenital copper metabolism abnormality caused by a mutation in the copper-transferred protein ATP7A gene, is genetically known as X-linked recessive genetics, with a incidence of about one in 100,000 in newbornsrecently, Cyprium Therapeutics(
FDA
has granted the rapid access status of the treatment of THE typical Menkes syndrome (MD) to theFDA treatment that has not shown significant clinical progressabout CUTX-101
CUTX-101 is a subcutaneous injection of copper histopackpreparation, designed to supplement blood and brain levels in Patients with MDData from Phase I/II clinical studies show that early treatment of MD patients using CUTX-101 can effectively improve neurodevelopmental outcomes and survivalCurrently, CUTX-101 is in Phase III clinical developmentIn addition to CUTX-101, Cyprium is also developing a gene therapy based on adeno-related virus (AAV) AAV-ATP7A, which is designed to deliver the correct version of the ATP7A gene to Patients with MDIn the United States, the FDA has qualified for the orphan drug cut cutX-101 and AAV-ATP7A