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AAV.7m8.CaMeRaOpsin GTx
- Development stage
- Preclinical
- Lead developer
- Adverum Biotechnologies
- Modality
- Gene Therapies
- Administration
- Ophthalmic
01
Overview
AAV.7m8.CaMeRaOpsin GTx is a preclinical optogenetic gene therapy being developed by Adverum Biotechnologies for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The therapy utilizes the AAV.7m8 vector, a proprietary adeno-associated virus variant engineered through directed evolution for efficient retinal transduction following intravitreal injection. The payload is "CaMeRaOpsin," a light-sensitive opsin protein designed to confer photosensitivity to surviving retinal cells, such as bipolar cells or retinal ganglion cells, in areas where photoreceptors have been lost. This approach aims to restore visual function by bypassing damaged photoreceptors and creating a new light-sensing layer within the retina, potentially offering a restorative treatment for patients with advanced retinal degeneration.
- Other names
- CaMeRaOpsin GTx
02
Targets
Chimeric optogenetic opsin
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