Avista Therapeutics, a gene therapy developer for rare ophthalmic conditions, has formed a partnership with Roche to develop novel AAV gene therapy vectors for the eyes. Avista recently launched as a spinout from the University of Pittsburgh Medical Center (UPMC).

The partnership with Roche aims to apply Avista’s single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology to develop 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 these novel capsids, which will be distinct from Avista’s internal pipeline.

“We are excited to enter into this collaboration with Roche, a global leader in health care,” said Robert Lin, CEO, Avista Therapeutics. “This collaboration will complement our in-house pipeline and will accelerate the delivery of transformative therapies to patients.”

Avista will receive an upfront payment of $7.5 million and, if successful, is eligible to receive additional payments during the research phase of the partnership, as well as clinical and sales milestone payments and royalties for resulting products with a total potential deal value that may exceed $1 billion.

Built on the research of leading experts in viral vector development and clinical ophthalmology, Avista was founded by: Leah Byrne, assistant professor of ophthalmology at the University of Pittsburgh School of Medicine; José-Alain Sahel, M.D., chair of the department of ophthalmology at the University of Pittsburgh School of Medicine, who is known worldwide for his expertise in vision restoration techniques and is an expert in AAV vector generation; and Paul Sieving, M.D., an internationally recognized physician-scientist and former director of the National Eye Institute.

Avista’s computationally guided, in vivo scAAVengr platform leverages a high-throughput approach with built-in quantitative validation of novel cell-specific AAVs, enabling the rapid translation of transformative gene therapies to the clinic for diseases impacting people’s vision. The company will develop a proprietary pipeline built on a toolkit of AAV variants that can target gene delivery to individual retinal cell types.

“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,” added Dr. Lin, who is also a vice president at UPMC Enterprises, the innovation, venture capital and commercialization arm of UPMC. “Avista was founded to solve this problem, and 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 $10 million in seed funding and years of foundational support from UPMC Enterprises, which invests in translational science with the potential to radically transform health care.

Jeanne Cunicelli, president of UPMC Enterprises, said, “UPMC is thrilled to support the launch of Avista, a team with unmatched expertise in AAV technology and clinical ophthalmology who are addressing a diverse group of blinding disorders that have a profound impact on patients’ quality of life. We believe that Avista is uniquely positioned to address the high unmet medical need in inherited retinal diseases.”