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sonpiretigene isteparvovec
- Development stage
- Phase 2
- Lead developer
- Nanoscope Therapeutics
- Modality
- Gene Therapies
- Administration
- Ophthalmic, Intravitreal
01
Overview
Sonpiretigene isteparvovec (MCO-010) is an investigational, ambient-light activatable optogenetic gene therapy developed by Nanoscope Therapeutics for the treatment of advanced retinitis pigmentosa and Stargardt disease. It utilizes an adeno-associated virus serotype 2 (AAV2) vector to deliver a proprietary multi-characteristic opsin (MCO) gene cassette into retinal bipolar cells. By expressing light-sensitive proteins in these downstream neurons, the therapy aims to restore vision in patients with severe photoreceptor degeneration, regardless of the underlying genetic mutation. It is administered as a single intravitreal injection and is designed to work in ambient light conditions without the need for external light-amplification devices.
- Brand names
- MCO-010MCO010MCO 010
- Other names
- Multi-Characteristic Opsin 010
02
Targets
MCO (Multi-characteristic opsin)
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