Chemistry: Generation 2+
ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations in GFAP that lead to spontaneous overproduction and toxic accumulation of GFAP into abnormal protein deposits called Rosenthal fibers in the brain. ASO-mediated reduction of GFAP has the potential to ameliorate the underlying cause of disease pathology and reverse or prevent disease progression.
About Alexander disease
Alexander disease (AxD) is a rare neurological condition characterized as a leukodystrophy, or a disease affecting the myelin sheath (the fatty insulation that protects a nerve fiber and supports signal conduction). Two major types of AxD have been defined. Type I onset typically occurs before 4 years of age and patients can experience head enlargement, seizures, limb stiffness, delayed or declining cognition, and lack of growth. Type II onset typically occurs after the age of 4 and symptoms can include difficulty speaking, swallowing, and making coordinated movements. AxD is most often fatal. There are treatments that can relieve symptoms, but there is no disease modifying therapy yet available to patients.
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