Illustration of Gene Therapy Vectors for Ocular DiseasesAvista Therapeutics, which recently launched as a spinout of leading health system University of Pittsburgh Medical Center (UPMC), aims to develop innovative gene therapies for rare ophthalmic conditions. The new company announced a partnership with Roche to develop novel AAV gene therapy vectors for the eyes.

The partnership 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, PhD, Chief Executive Officer of 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, PhD, Assistant Professor of Ophthalmology at the University of Pittsburgh School of Medicine, and 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, PhD, internationally recognized physician-scientist and former director of the National Eye Institute. Both Drs. Byrne and Sahel are affiliated faculty members of the McGowan Institute for Regenerative Medicine.

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.

“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,” said Jeanne Cunicelli, MBA, President of UPMC Enterprises. “We believe that Avista is uniquely positioned to address the high unmet medical need in inherited retinal diseases.”

About Avista Therapeutics

Avista Therapeutics’ mission is to develop innovative gene therapies for retinal diseases, including rare ophthalmic conditions that have a profound impact on patients’ quality of life. We leverage our computationally guided scAAVengr platform to generate and validate a toolkit of proprietary AAV vectors that target specific cell types using minimally invasive intravitreal delivery with reduced dosages. Our quantitative, in vivo-based approach and clinical ophthalmology expertise allow us to rapidly translate new gene therapies to the clinic.

Illustration:  Avista Therapeutics.

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