Avista Therapeutics Expands Its AI Gene Therapy Platform

Avista Therapeutics, a preclinical gene therapy company based in Bridgeside Labs at Hot Metal Campus, has expanded its gene therapy platform to take on some of the field's hardest delivery problems. The company recently announced that it is scaling up its capsid design program with artificial intelligence and adding a new Innovation Platform, work it says targets long-standing barriers in AAV capsid engineering, large gene delivery and manufacturing.

Image: Marc Schulte/Unsplash

The expansion grows out of the capsids Avista developed for retinal gene therapy. At the center of the new effort is ARTEMIS, an AI platform for capsid engineering. ARTEMIS draws on large non-human primate datasets with single-cell resolution that Avista generates in-house through its scAAVengr-HUnT workflow. Those datasets map millions of AAV variants across serotypes, tissues, routes of administration and animal models. ARTEMIS pairs that data with proprietary protein language models and the molecular dynamics of capsids, which lets the models account for the structural biology behind capsid fitness rather than sequence alone.

Image: MJH Shikder/Unsplash

The company also launched an Innovation Platform focused on AAV targeting, large gene delivery and AAV manufacturing. By combining structural biology, protein design and clinical experience, Avista aims to develop solutions to technical problems that have slowed the broader gene therapy field and kept promising treatments from reaching patients.

Robert Lin, PhD, Avista's chief executive officer, said the pairing of sequence and structural models positions the company to design safe, efficient delivery of genetic medicines to tissues throughout the body. "Building upon our best-in-class intravitreal vectors, Avista is leveraging these innovative technologies to create impactful therapies," Lin said, pointing to a preclinical pipeline that spans XLRS, PRPF31 and other retinal diseases along with central nervous system indications.

Avista's growth reflects the kind of research taking shape across the Hot Metal Campus, where university spinouts, established institutions and private industry all come together.

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