Roche and Avista partner on AAV gene therapy vectors for eye disease

The partnership aims to apply Avista’s single-cell adeno-associated virus (AAV) engineering platform (scAAVengr) technology in the development of intravitreal capsids of AAV that match a capsid profile defined by Roche.

Under the terms of the partnership, Roche has the right to evaluate and license Avista’s novel capsids, and will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy programs using them.

Avista will receive an upfront payment of $7.5 million. Roche will also make other payments to the technology provider during the research phase of the collaboration as well as sales-based milestone and clinical royalty payments for the resulting products. The deal could ultimately be worth more than $1 billion to Avista.

Avista’s computer-guided in vivo scAAVengr platform leverages a high-throughput approach with built-in quantitative validation of novel cell-specific AAVs. The company said the technology could advance transformative gene therapies targeting eye disease to clinical trials.

“Traditional therapies for retinal dystrophies only address symptoms and complications, neglecting the underlying disease biology, and while current vector-based technologies show promise, they have been significantly limited in their ability to target cell types keys through the retina.Avista was founded to solve this problemsaid its CEO, Robert Lin. “Our innovative scAAVengr platform enables us to deliver gene therapy payloads by intravitreal injection to treat a full range of retinal diseases with reduced immunogenicity.

Avista received $10 million in seed funding and foundational support from UPMC Enterprises. According to Jeanne Cunicelli, president of UPMC Entreprises, Avista is “uniquely positioned to address the high unmet medical need in the area of ​​inherited retinal diseases.

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