scAAVengr Platform
How does scAAVengr work?
Despite progress in gene therapy, ensuring that viral vectors effectively deliver therapeutic genes to the targeted cells in the retina remains a key obstacle. To address this challenge, the in-vivo scAAVengr computational platform utilizes single-cell RNA sequencing to identify various AAV vectors, rapidly and quantitatively, for their suitability in delivering gene therapy to specific parts of the retina.
How is it different?
The traditional approach to evaluating AAVs is slow and imprecise, often taking years and necessitating several studies to identify promising candidates for delivering gene therapy. In contrast, scAAVengr offers a more rapid and precise method for evaluating AAVs as it directly measures the efficacy of AAV vectors in both cell entry and gene cargo expression. Avista is also utilizing intravitreal injections to broaden gene therapy accessibility beyond sub-retinal delivery, which requires invasive surgery of the retina. The Company’s novel AAVs have the potential to be administered in-office at lower doses, delivering the same or improved results as the current surgical procedure.