Sepofarsen

DB16327InvestigationalGene Therapies

Sepofarsen is under investigation in clinical trial NCT03913143 (A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)).

Mechanism: Pharmacologically active on Centrosomal protein of 290 kDa
Curator reviewed
Code names: QR-110

Resolves to

Compound

UNII7CAZ46X8ELCAS2227173-68-2

Clinical / RWD

ATCS01XA29

Targets

O15078CEP290

What you can answer from here — as of October 05, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

5Clinical trialsPhase, status and sponsor resolved per trial

1ATC codeIncluding every combination product, plus 2 drug categories

5+ReferencesStructured and connected to the statements they support

Which drugs share a target with Sepofarsen, and which of those have an active Phase 3 trial?

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