MYR-101

MYR-101 (rAAV-Olig001-ASPA) is an investigational recombinant adeno-associated virus (rAAV) vector-based gene therapy developed for the treatment of Canavan disease, a rare and fatal childhood genetic disorder characterized by white matter degeneration in the brain. The disease is caused by mutations in the ASPA gene, leading to deficiency of aspartoacylase enzyme and accumulation of N-acetylaspartate (NAA), which impairs myelin production. MYR-101 delivers a functional ASPA gene specifically to oligodendrocytes via intracerebroventricular administration, enabling restoration of aspartoacylase activity, reduction in NAA levels, and improvement in myelination. Clinical data from phase 1/2 trials show significant reductions (>80%) in NAA levels and increases in brain white matter volume with associated functional improvements. The therapy has received FDA Fast Track, Rare Pediatric Disease, Orphan Drug designations, RMAT designation, and was selected for the FDA START pilot program[1][2][3][4][5][7].

Other names

rAAV-Olig001-ASPArAAV-Olig-001-ASPArAAV-Olig 001-ASPAMYR101MYR-101MYR 101