Roche and Avista team up on AAV gene therapy vectors for eye diseases

By Jane Byrne

- Last updated on GMT

© GettyImages/ljubaphoto
© GettyImages/ljubaphoto

Related tags Eye AAV Gene therapy Roche

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, is teaming up with Roche to develop novel AAV gene therapy vectors for eye diseases.

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

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

Avista will receive an upfront payment of $7.5m. Roche will also make further payments to the technology provider during the collaboration’s research phase as well as clinical, sales-based milestone and royalty payments for resulting products. The deal could ultimately be worth over $1bn for Avista.

Avista’s computationally 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 trial.

"Traditional therapies for retinal dystrophies address only symptoms and complications, neglecting the underlying biology of the diseases, and while current vector technologies hold promise, they have been greatly limited in their ability to target key cell types across the retina.Avista was founded to solve this problem​,” said its CEO Robert Lin. “Our innovative scAAVengr platform allows us to deliver gene therapy payloads through intravitreal injection to treat a full range of retinal diseases with reduced immunogenicity.”

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

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