CRISPR-UBE3A-ATS

CRISPR-UBE3A-ATS is an experimental CRISPR-based gene editing therapy being developed by AS²Bio in collaboration with CourageGT for the treatment of Angelman syndrome. The disorder is primarily caused by the loss of function of the maternally inherited UBE3A gene in neurons, while the paternal copy remains intact but is epigenetically silenced by a long non-coding RNA known as the UBE3A antisense transcript (UBE3A-ATS). This therapeutic program utilizes CRISPR technology to target and disrupt the UBE3A-ATS, thereby unsilencing and reactivating the paternal UBE3A gene to restore functional protein levels in the central nervous system. It is currently in the preclinical stage of development.