Sepofarsen

Modify Date: 2025-08-26 15:51:26

Sepofarsen structure	Common Name	Sepofarsen
	CAS Number	2227173-68-2	Molecular Weight	N/A
	Density	N/A	Boiling Point	N/A
	Molecular Formula	N/A	Melting Point	N/A
	MSDS	N/A	Flash Point	N/A

Use of Sepofarsen

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.

Name	Sepofarsen

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.

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.

No Any Chemical & Physical Properties

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Sepofarsen

Use of Sepofarsen

Names

Sepofarsen Biological Activity

Chemical & Physical Properties

The content on this webpage is sourced from various professional data sources. If you have any questions or concerns regarding the content, please feel free to contact service1@chemsrc.com.