Manufacturers: Oak Hill Bio, Roche
Rugonersen (RO7248824) is a locked nucleic acid antisense oligonucleotide initially developed by Roche and later acquired by Oak Hill Bio as a potential disease-modifying treatment for Angelman syndrome. It is designed to increase expression of the paternal UBE3A gene by targeting the UBE3A-antisense transcript. Following encouraging findings from the Phase I TANGELO study, Oak Hill Bio plans to commence a pivotal Phase III clinical trial in the second quarter of 2026. Rugonersen is being positioned as a potential best-in-class therapy for Angelman syndrome.
Rugonersen (RO7248824) is a locked nucleic acid antisense oligonucleotide initially developed by Roche and later acquired by Oak Hill Bio as a potential disease-modifying treatment for Angelman syndrome. It is designed to increase expression of the paternal UBE3A gene by targeting the UBE3A-antisense transcript. Following encouraging findings from the Phase I TANGELO study, Oak Hill Bio plans to commence a pivotal Phase III clinical trial in the second quarter of 2026. Rugonersen is being positioned as a potential best-in-class therapy for Angelman syndrome.
Rugonersen is an antisense oligonucleotide that increases expression of the paternal UBE3A gene by targeting the UBE3A-antisense transcript, thereby addressing the underlying genetic cause of Angelman syndrome.
1 associated conditions
