Drugs / cotoretigene toliparvovec
cotoretigene toliparvovec
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial · 1 missed | ||||||
| Phase 1/2 | NCT03116113 | Mar 2017 → Nov 2020 | retinitis pigmentosa | Biogen | Completed | Missed primary |
| Phase 31 trial | ||||||
| Phase 3 | NCT03584165 | Jun 2018 → Jun 2026 | choroideremia, retinitis pigmentosa | NightstaRx Ltd, a Biogen Company | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2021-05-14 | Biogen | Results Biogen Announces Topline Results From Phase 2/3 Gene Therapy Study for XLRP biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced topline results from the Phase 2/3 XIRIUS study of cotoretigene toliparvovec (BIIB112), a gene therapy being investigated as a one-time therapy for patients with X-linked retinitis pigmentosa (XLRP). |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2021-05-14 | Biogen | Biogen Announces Topline Results From Phase 2/3 Gene Therapy Study for XLRP biogen.com ↗ |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | cotoretigene toliparvovec | “PURPOSE: Cotoretigene toliparvovec (BIIB112/AAV8-RPGR) is an investigational vector-based gene therapy designed to provide a full-length, codon-optimized retinitis pigmentosa...” PMID 38423215 ↗ Feb 2024 |
| Known as | AAV8-RPGR | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594321 ↗ |
| Known as | BIIB112 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03584165 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “subretinal delivery of an adeno-associated viral vector encoding codon-optimized human RPGR (AAV8-coRPGR)” PMID 32094925 ↗ Feb 2020 |
| Route | Other | “by sub-retinal injection” NCT03116113 ↗ |