Rugonersen
Modify Date: 2025-08-27 12:15:09
Rugonersen structure
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Common Name | Rugonersen | ||
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| CAS Number | 2591587-57-2 | Molecular Weight | 6530.00 | |
| Density | N/A | Boiling Point | N/A | |
| Molecular Formula | N/A | Melting Point | N/A | |
| MSDS | N/A | Flash Point | N/A | |
Use of RugonersenRugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense 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]. |
| Name | Rugonersen |
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| Description | Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense 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]. |
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| Related Catalog | |
| Target |
ubiquitin-protein ligase E3A (UBE3A)[1] |
| In Vitro | 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]. |
| In Vivo | 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]. Animal Model: Male cynomolgus monkey[1] Dosage: 24 mg per monkey Administration: Intrathecal injection; single dose or twice dose with 2 weeks apart; sacrificed at 8, 15, 29, 57, and 85 days after the last dose Result: Resulted a long duration of action on paternal UBE3A reactivation in NHP brains after IT delivery. Animal Model: WT and AS Ube3a m-/p+ mice adult mice (10-12 weeks old)[1] Dosage: 150 μg per mice Administration: Intracerebroventricular injection; single dose; harvested at 2 weeks post injection Result: Revealed a steep relationship between UBE3A-ATS knock-down and UBE3A mRNA/protein upregulation, whereby an almost 90% downregulation was needed to achieve a 50% upregulation, respectively. |
| References |
[2]. World Health Organization · 2021: WHO Drug Information. |
| Molecular Weight | 6530.00 |
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