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AVA-311

Development stage: Discontinued
Lead developer: Adverum Biotechnologies
Modality: Gene Therapies
Administration: Intravitreal

Overview

AVA-311 is a gene therapy candidate developed for the treatment of X-linked retinoschisis (XLRS), a genetic retinal disease. The therapy uses a novel adeno-associated virus (AAV) vector designed through directed evolution to deliver therapeutic genes directly to retinal cells, aiming to correct the underlying genetic defect responsible for XLRS. The drug was developed through a collaboration between Adverum Biotechnologies and Regeneron, with preclinical studies conducted but no evidence of progression to clinical trials. Its current development status is discontinued[1][9].

Targets

RS1 (Retinoschisin)

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