Centrosomal protein 290. Every medicine in our data that ChEMBL or the Guide to Pharmacology records as acting on this protein, with the source for each and its current clinical trials on this site.
| Medicine | How it acts | Source | Current trials here |
|---|---|---|---|
| Sepofarsen CHEMBL4594497 |
CEP290 mRNA positive modulator Positive modulator Oligonucleotide |
ChEMBL only | 2 |
“Both” means ChEMBL and the Guide to Pharmacology each record this medicine acting on this protein; “only” means one source records it and the other does not (often because it lists fewer secondary targets). The Guide to Pharmacology adds a medicine here only when this protein is its primary target. Medicines without a link have no page on this site yet.
| Disease | Medicines tested | Furthest trial phase |
|---|---|---|
| Leber congenital amaurosis | 1 | Phase 3 |
| Leber congenital amaurosis 10 | 1 | Phase 2/3 |
From ChEMBL's drug-disease records, which draw on trial registries. A trial phase is not an approval; approved uses are on each medicine's page from the regulators' own records.
Which medicines act on this protein comes from two independent, expert-curated sources: ChEMBL's mechanism-of-action records (via the Open Targets Platform) and the IUPHAR/BPS Guide to PHARMACOLOGY. Each row says which of them lists it. Data from them is shared here under their share-alike licences (CC BY-SA 3.0 and 4.0).
A job re-reads both sources every 30 days. Medicines are matched to our pages by exact name or a ChEMBL-curated synonym, and to the Guide to Pharmacology by ChEMBL ID or exact name, never by a close spelling. No AI drafting. See our methodology. The Open Targets data on this page was last retrieved on 30 Sep 2026. How every register is built: methodology · fixes we have made: corrections log.
