Avista Therapeutics Partners with Roche to Develop Therapies for Retinal Diseases

Avista News Translational Sciences

Avista Therapeutics, a UPMC Enterprises spinout that aims to develop innovative gene therapies for rare ophthalmic conditions, announced a partnership with Roche to develop novel AAV gene therapy vectors for ocular diseases.

The partnership aims to apply Avista’s single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology to develop AAV capsids for intravitreal injection 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. These programs will be distinct from Avista’s internal pipeline.

Built on the research of leading experts in viral vector development and clinical ophthalmology, Avista was founded by Leah Byrne, PhD, Assistant Professor of Ophthalmology at the University of Pittsburgh School of Medicine, an expert in AAV vector generation, José-Alain Sahel, MD, 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 Paul Sieving, MD internationally recognized physician-scientist and former director of the National Eye Institute.

Avista’s mission is to develop innovative gene therapies for retinal diseases, including rare ophthalmic conditions that have a profound impact on patients’ quality of life. Traditional therapies for retinal dystrophies address only symptoms and complications, neglecting the underlying biology of the diseases, and current vector technologies have been limited in their ability to target key cell types across the retina.

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 will use this technology to develop a proprietary pipeline built on a toolkit of AAV variants that can target gene delivery to individual retinal cell types. This toolkit will enable the rapid development and clinical translation of transformative gene therapies for diseases impacting people’s vision.

“We are excited to enter into this collaboration with Roche, a global leader in health care,” said Robert Lin, PhD, CEO of Avista, and VP at UPMC Enterprises. “We believe this collaboration will complement Avista’s in-house pipeline and accelerate the delivery of transformative therapies to patients.”

Avista received $10 million in seed funding and years of foundational support from the Translational Sciences team at UPMC Enterprises. With three full-time employees, Avista anticipates using this round of funding, in combination with their partnership with Roche, to expand the Avista team, establish laboratory and office space, and develop a preclinical therapeutic pipeline.

To learn more, visit the Avista website.

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