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elsunersen   Click here for help

GtoPdb Ligand ID: 14130

Synonyms: PRAX-222 | PRAX222
Compound class: Nucleic acid
Comment: Elsunersen (PRAX-222) is a 20-mer anti-sense oligonucleotide that is designed to selectively decrease expression of the gene for the voltage-gated sodium channel Nav1.2 (SCN2A). This is proposed as a mechanism to treat early onset developmental and epileptic encephalopathy (DEE) [1-3], that is driven by gain-of-function mutations in SCN2A. We include the linear nucleotide sequence in this entry, without the chemical modifications that are included in the administered version of elsunersen. A full descriptor is d([2'-O-(2-methoxyethyl)](m5rC-sp-m5rC-rA-5rC-rG-rA)-5C-sp-A-sp-T-p-A-p-T-p-T-p-T-p-T-p-T-p-m5C-sp-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA).
References
1. Li N, Yan L, Peng A, Fu X, Qin H, Yao K. (2025)
Voltage-gated sodium channels in the nervous system: Molecular physiology to therapeutic interventions.
Neural Regen Res, [Epub ahead of print]. [PMID:40808385]
2. Wagner M, Berecki G, Fazeli W, Nussbaum C, Flemmer AW, Frizzo S, Heer F, Heinen F, Horton R, Jacotin H et al.. (2025)
Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy.
Nat Med, 31 (7): 2174-2178. [PMID:40263630]
3. Zhao T, Chen F, Wang L, Xie Y, Yang M, Feng F. (2025)
SCN2A gene mutations with epilepsy: single center experience.
Ital J Pediatr, 51 (1): 170. [PMID:40462216]