sepofarsen
Nucleic acidexperimental
Clinical trial activity
3 trials · 3 clinical orgs · 0 marketing orgs
Phase 1
0
Phase 2
1
Phase 3
3
Phase 4
0
Earliest trial started Apr 4, 2019 (NCT03913143)
Timeline
Indications
Studied for
- Blindness · Phase 3
- Eye Diseases · Phase 3
- Eye Diseases, Hereditary · Phase 3
- Leber Congenital Amaurosis · Phase 3
- Neurologic Manifestations · Phase 3
- Optic Atrophy, Hereditary, Leber · Phase 3
- Retinal Degeneration · Phase 2/Phase 3
- Retinal Diseases · Phase 3
- Retinal Dystrophies · Phase 2/Phase 3
- Sensation Disorders · Phase 3
- Vision Disorders · Phase 3
Mechanism of action
- CEP290 mRNAINHIBITOR
CEP290 mRNA inhibitor
- CEP290 mRNAPOSITIVE MODULATOR
CEP290 mRNA positive modulator
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Multi-specific
- No
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL4594497 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06891443 | Phase 3 | Jun 4, 2025 | Laboratoires Thea, Sepul Bio |
| NCT04855045 | Phase 2/Phase 3 (Phase 3) | Mar 23, 2021 | ProQR Therapeutics |
| NCT03913143 | Phase 2/Phase 3 (Phase 3) | Apr 4, 2019 | ProQR Therapeutics |
Showing 3 of 3 trials
Page 1 / 1
Organizations
Research & Development (3)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| ProQR Therapeutics | For profit | 2 | 2 | 1 | 2019 |
| Laboratoires Thea | For profit | 1 | 1 | 1 | 2025 |
| Sepul Bio | For profit | 1 | 0 | 1 | 2025 |
3 organizations
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