rugonersen

Rugonersen (OHB-724) is an investigational *antisense oligonucleotide* being developed for the treatment of **Angelman syndrome**. It works by targeting and suppressing the UBE3A-antisense transcript (UBE3A-ATS), which allows restoration of UBE3A expression from the normally silent paternal allele in neurons, addressing the genetic basis of Angelman syndrome. Rugonersen was discovered through a high-throughput screen of approximately 2,500 LNA-modified antisense oligonucleotides in patient-derived neurons. It has shown favorable potency, efficacy, and safety in preclinical studies, and initial human data have been published. The therapy is being advanced by Oak Hill Bio, after in-licensing from Roche.[1][4][8][10][12]

Other names

rugonersenOHB-724OHB724OHB 724RO 7248824RO7248824RO-7248824RG 6091RG6091RG-6091UBE3A-LNAUBE-3A-LNAUBE 3A-LNA