Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
cotoretigene toliparvovec
- Development stage
- Discontinued
- Lead developer
- Biogen
- Modality
- Gene Therapies
- Administration
- Subretinal
01
Overview
Cotoretigene toliparvovec is an investigational, adeno-associated virus serotype 8 (AAV8) vector-based gene therapy designed for subretinal administration. It delivers a full-length, codon optimized retinitis pigmentosa GTPase regulator (RPGR) gene to retinal cells in patients with X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. By replacing the defective or missing RPGR gene, this therapy aims to increase levels of functional RPGR protein and potentially slow, halt, or prevent further degeneration of photoreceptors in affected individuals. Cotoretigene toliparvovec was developed primarily for XLRP and has orphan drug designation for this indication[1][2][3][4][5].
- Other names
- Adeno-associated viral vector serotype 8 containing the human RPGR geneAdenovirus-associated viral vector serotype 8 containing the human RPGR gene
02
Targets
RPGR (Retinitis pigmentosa GTPase regulator)
Beyond the preview
Go deeper on cotoretigene toliparvovec.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on cotoretigene toliparvovec.