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aguracingene cadoparvovec
- Development stage
- Phase 2
- Lead developer
- MeiraGTx
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Subretinal
01
Overview
Aguracingene cadoparvovec is an investigational gene therapy designed to treat achromatopsia, a rare inherited retinal disorder that causes color blindness and visual impairment. The therapy uses an adeno-associated virus (AAV) vector to deliver a functional copy of the CNGA3 gene directly into retinal cells via subretinal injection. By restoring CNGA3 function, the treatment aims to improve or restore cone photoreceptor activity in patients with achromatopsia caused by biallelic mutations in the CNGA3 gene. The drug is being developed by MeiraGTx and Johnson & Johnson Innovative Medicine (formerly Janssen), with UCL Business as the originator[1][2][3][6]. It has received orphan drug designation for color vision defects[1]. As of April 2024, it is in Phase I/II clinical trials for achromatopsia[1][7].
- Other names
- aguracingene cadoparvovec
02
Targets
CNGA3 (Cyclic nucleotide-gated channel subunit alpha 3)
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