obudanersen

Obudanersen (formerly ION582) is an investigational antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of Angelman syndrome. Angelman syndrome is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited *UBE3A* gene in the brain. In neurons, the paternal *UBE3A* allele is normally silenced by a long non-coding RNA called the *UBE3A* antisense transcript (*UBE3A-ATS*). Obudanersen is designed to target and promote the degradation of *UBE3A-ATS*, thereby unsilencing the paternal *UBE3A* allele and restoring UBE3A protein expression in the central nervous system. The drug is administered via intrathecal injection and is currently being evaluated in the pivotal Phase III REVEAL clinical trial.

Other names

BIIB121BIIB-121BIIB 121ION582ION-582ION 582