Drugs / AAV2-REP1
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 2 | NCT03507686 | Nov 2017 → Jun 2022 | choroideremia | Biogen | Completed | No outcome recorded |
| Phase 2 | NCT02407678 | Aug 2016 → Jul 2021 | choroideremia | University of Oxford | Completed | No outcome recorded |
| Phase 2 | NCT02553135 | Sep 2015 → Feb 2018 | choroideremia | Byron Lam | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2021-06-14 | Biogen | Results Biogen Announces Topline Results from Phase 3 Gene Therapy Study in Choroideremia biogen.com ↗ |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2021-06-14 | Biogen | Biogen Announces Topline Results from Phase 3 Gene Therapy Study in Choroideremia biogen.com ↗ |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AAV2-REP1 | “The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants...” NCT02407678 ↗ |
| Known as | timrepigene emparvovec | “Timrepigene emparvovec is an adeno-associated virus serotype 2 vector encoding the cDNA of Rab escort protein 1, augmented by a downstream woodchuck hepatitis virus...” PMID 38970425 ↗ Jul 2024 |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)” NCT02407678 ↗ |
| Route | Other | “AAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection” NCT02407678 ↗ |
| Target | RABEP1 | “encoding the cDNA of Rab escort protein 1” PMID 38970425 ↗ Jul 2024 |