MYR-101

MYR-101 (rAAV-Olig001-ASPA) is a recombinant adeno-associated virus (rAAV) vector-based gene therapy intended to treat Canavan disease. Canavan disease is a fatal childhood genetic disorder characterized by white matter degeneration in the brain. It is caused by a mutation in the aspartoacylase gene (ASPA), which leads to a deficiency of the aspartoacylase enzyme (ASPA). ASPA is produced in oligodendrocytes, and participates in the metabolism of N-acetylaspartate (NAA). When not properly metabolized , NAA accumulates in the brain and negatively affects myelin production. MYR-101 targets oligodendrocytes and delivers functional ASPA. The FDA has granted MYR-101 Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for the treatment of patients with Canavan disease.