Ultevursen sodium  (Synonyms: QR-421a sodium)

Ultevursen sodium (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen sodium binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen sodium exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa^[1][2].

Ultevursen sodium induces concentration-dependent USH2A exon 13 skipping in WERI-Rb1 cells^[1].
Ultevursen sodium induces concentration-dependent USH2A exon 13 skipping in patient-derived iPSC-derived photoreceptor progenitor cells^[1].
Ultevursen (QR-421a) (50 ng; 3 days) sodium delivered as a U7 snRNA skipper induces USH2A exon 13 skipping in stable HEK293T USH2A-eGFP reporter cells, leading to detectable eGFP expression^[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Ultevursen sodium (QR-421a) related PMO restored visual function by inducing exon skipping in ush2a-exon13 mutant zebrafish^[2].
Ultevursenassociated AAV-U7 dual skipper (1 μL viral vector suspension; intratympanic + utricle microinjection; P1-P2 neonatal Ush2a KO-1/KO-1 mice; 16 weeks in vivo incubation period) sodium achieves robust hair cell transduction and mediates sustained Ush2a exon skipping within mouse inner ear tissue^[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

8372.24 (free acid)

C₂₇₃H₃₇₀N₈₁Na₂₁O₁₄₄P₂₀S₂₀

Solid

White to off-white

[Ultevursen (sodium)]

Room temperature in continental US; may vary elsewhere.

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

• [1]. International Nonproprietary Names for Pharmaceutical Substances (INN). WHO Drug Information, Vol. 36, No. 2, 2022.