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ATX002 capsid
- Development stage
- Preclinical
- Lead developer
- Avista Therapeutics
- Modality
- Wild-type AAV → AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Engineered AAV → AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravitreal Injection
01
Overview
ATX002 capsid is a proprietary engineered adeno-associated virus (AAV) vector developed by Avista Therapeutics for gene therapy applications, specifically optimized for efficient panretinal transduction following intravitreal injection. The ATX002 capsid enables robust delivery of therapeutic genes to retinal cells, outperforming previous AAV variants such as 7m8 in nonhuman primate models. Its primary application is in the treatment of inherited retinal diseases, with current development focused on delivering the Retinoschisin protein for X-linked juvenile retinoschisis (XLRS). The technology leverages computational and machine learning approaches to optimize AAV vectors for targeted and minimally invasive ocular gene delivery[1].
- Other names
- Activated regulatory T cellsATregCD4-mediated activated regulatory T cellsCD-4-mediated activated regulatory T cellsCD 4-mediated activated regulatory T cellsGP120-activated regulatory T cellsGP-120-activated regulatory T cellsGP 120-activated regulatory T cells
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