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.
Product Specifications
CAS Number
2227173-68-2
Product Name Alternative
QR 110
UNSPSC
12352208
Target
Others
Type
Oligonucleotides
Related Pathways
Others
Field of Research
Others
Assay Protocol
https://www.medchemexpress.com/sepofarsen.html
Solubility
10 mM in H2O
Smiles
[Sepofarsen]
Molecular Formula
C180H235N67O101P16S
Molecular Weight
5962.00
References & Citations
[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.
Shipping Conditions
Room temperature
Scientific Category
Oligonucleotides
Clinical Information
Phase 3
Frequently Asked Questions
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