Sepofarsen

Names

[ CAS No. ]:
2227173-68-2

[ Name ]:
Sepofarsen

Biological Activity

[Description]:

Sepofarsen (QR-110) is an RNA antisense oligonucleotide targeting to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene.

[Related Catalog]:

Research Areas >> Others
Signaling Pathways >> Others >> Others

[References]

[1]. Russell SR, Drack AV, Cideciyan AV, et al. Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial. Nat Med. 2022;28(5):1014-1021.  

[2]. Cideciyan AV, Jacobson SG, Ho AC, et al. Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report. Nat Med. 2021;27(5):785-789.  

Chemical & Physical Properties

No Any Chemical & Physical Properties

Related Compounds

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