Oligonucleotide (Antisense oligonucleotide) · Gene silencing: CEP290 · developed by ProQR Therapeutics
Sepofarsen is an RNA antisense oligonucleotide for intravitreal injection. It is being studied for Leber congenital amaurosis 10 and other retinal diseases causing vision disorders and blindness.
Summary written with AI from the sources listed at the end of this page.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT03913143 | Phase 2/3 | Leber Congenital Amaurosis 10 | Active not recruiting | PRQR |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
BlindnessEye DiseasesEye Diseases, HereditaryEye Disorders CongenitalLeber Congenital AmaurosisLeber Congenital Amaurosis 10Neurologic ManifestationsRetinal DiseaseSensation DisordersVision Disorders
Updated Oct 3, 2026. Something wrong or missing? Report it on the PRQR page.
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