aav-rpgr
Gene therapyexperimental
Clinical trial activity
3 trials · 6 clinical orgs · 0 marketing orgs
Phase 1
2
Phase 2
3
Phase 3
0
Phase 4
0
Earliest trial started Mar 16, 2017 (NCT03116113)
Timeline
Indications
Studied for
Mechanism of action
- X-linked retinitis pigmentosa GTPase regulatorSupplementation
supplementation - RPGR
Chemistry & pharmacology
Oral
No
Parenteral
No
Topical
No
Also known as
- aav-rpgr
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07174726 | Phase 2 | Sep 10, 2025 | Beacon Therapeutics |
| NCT03252847 | Phase 1/Phase 2 (Phase 2) | Jul 14, 2017 | Emas Pharma Ltd., MeiraGTx, Syne Qua Non Limited, University of London |
| NCT03116113 | Phase 1/Phase 2 (Phase 2) | Mar 16, 2017 | NightstaRx Limited |
Showing 3 of 3 trials
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Organizations
Research & Development (6)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Beacon Therapeutics | For profit | 1 | 1 | 1 | 2025 |
| Emas Pharma Ltd. | For profit | 1 | 0 | 1 | 2017 |
| MeiraGTx | For profit | 1 | 1 | 1 | 2017 |
| NightstaRx Limited | For profit | 1 | 1 | 1 | 2017 |
| Syne Qua Non Limited | For profit | 1 | 0 | 1 | 2017 |
| University of London | Academic/Hospital | 1 | 0 | 1 | 2017 |
6 organizations
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