Zilganersen, formerly known as ION373, is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) mRNA designed to inhibit the production of GFAP. Zilganersen is being developed as a potential therapy for Alexander disease (AxD).
About Alexander Disease
AxD is a rare, progressive and fatal neurological disease that affects the myelin sheath which protects nerve fibers. AxD is caused by a gain-of-function mutation in the GFAP gene and is characterized by progressive deterioration, including loss of skills and independence, generally leading to death in childhood or early adulthood. There are treatments that can relieve symptoms, but there is no disease modifying therapy yet available to patients.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.