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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)
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