Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modifiedantisense oligonucleotides(ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch^[1][2].

ubiquitin-protein ligase E3A (UBE3A)^[1]

RO7248824 (0-10 μM) show a nanomolar potency against UBE3A-ATS (EC₅₀=26.3 nM), UBE3A mRNA upregulation (EC₅₀=15.4 nM) and UBE3A protein upregulation (EC₅₀=24.8 nM) in Angelman syndrome (AS) neurons^[1].

Rugonersen (RO7248824) (24 mg/monkey; i.t.; for 8-85 d) is well tolerated without adverse in-life effects or tissue pathology and produced a robust, long lasting (up to 3 months) paternal reactivation of UBE3A mRNA/protein across key monkey brain regions^[1].
Male cynomolgus monkeys^[1]Rugonersen (150 μg; i.c.v.; single dose) selectively and potently reduces UBE3A-ATS, while concomitantly upregulating the UBE3A mRNA and protein^[1].

6530.00

2591587-57-2

DNA, d(P-thio)((2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)rA-C-(2′-O,4′-C-methylene)rA-C-T-T-A-A-T-T-A-T-A-C-T-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)m5rC)

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