Research Objective

A novel AAV-based gene therapy candidate for use in treatment of inherited ocular disease.

Impact

AAV vectors have a limited cargo capacity (<5kb), preventing their use in the treatment of many untreatable genetic diseases caused by large genes.

Major Proposed Activities

• In vitro optimization and selection of gene therapy candidates
• In vivo testing to establish efficacy of gene therapy candidates
• Demonstration of disease-modifying activity in mouse genetic model of ocular disease
• Evaluation of safety for a dual vector AAV approach
• Testing activity in human photoreceptors

Of the 280 genetic mutations that cause inherited retinal disease (IRD), there is an approved treatment for just one. Our innovative technology provides a solution for challenges encountered by conventional AAV-based gene therapy approaches. Because our technology is agnostic to gene, the advancement of this therapy will be an important step forward for developing treatment for a wide variety of genetic diseases, including those that impact the diverse California population.