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sepofarsen
- Development stage
- Phase 3
- Lead developer
- ProQR Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravitreal
01
Overview
Sepofarsen is a first-in-class investigational RNA-based antisense oligonucleotide designed to treat Leber congenital amaurosis type 10 (LCA10), a severe inherited retinal disease that causes childhood blindness. It specifically targets the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene, which leads to aberrant splicing and non-functional CEP290 protein. Sepofarsen binds to the mutated pre-mRNA at this site, blocking access to an aberrant splice site and restoring normal splicing, resulting in increased production of functional CEP290 protein. The drug is administered via intravitreal injection directly into the eye. Clinical trials have shown improvements in visual function and retinal structure following treatment with sepofarsen[1][3][4][5][6][7].
- Other names
- sepofarsen
02
Targets
CEP290 IVS26 (CEP290 IVS26 mutant allele)
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