Avista Therapeutics Partners with Roche to Develop Advanced AAV Gene Therapy Vectors for Ocular Diseases

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.
  • Avista leverages its computationally guided single-cell adeno-associated virus engineering (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.
  • Avista’s quantitative, in vivo-based approach and clinical ophthalmology expertise enable it to rapidly translate new gene therapies to the clinic.

Built on the research of leading experts in viral vector development and clinical ophthalmology, Avista was founded by the following Scientists:

Leah Byrne, Ph.D., Assistant Professor of Ophthalmology at the University of Pittsburgh School of Medicine, and expert in AAV vector generation,

José-Alain Sahel, M.D., Chair of the Department of Ophthalmology at the University of Pittsburgh School of Medicine. Dr. Sahel  is known worldwide for his expertise in vision restoration techniques,

Paul Sieving, M.D., Ph.D., 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.
  • Avista Therapeutics was recently launched as a spinout of leading health system UPMC, aims to develop innovative gene therapies for rare ophthalmic conditions.

Avista Therapeutics in the NEWS

Avista announced a partnership with Roche (RHHBY) that aims at developing novel AAV gene therapy vectors for the eyes. The partnership intends 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 the novel capsids, which will be distinct from Avista’s internal pipeline.

Avista Therapeutics

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.

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 healthcare.

From Avista Therapeutics

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

Dr. Lin added “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. 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, President of UPMC Enterprises. “We believe that Avista is uniquely positioned to address the high unmet medical need in inherited retinal diseases.”

Contact Avista Therapeutics:

Chris Stern
[email protected]


MacDougall Advisors
Lauren Arnold
[email protected]

Prohost Observations

Thanks to the advancement of gene therapy and of AAV technologies, the time has come to address the unmet needs in treating retinal diseases.

We will follow up on Avista’s future accomplishments and the firm’s possible attraction for more collaboration through its advanced technological capabilities.

Did you see Prohost Comprehensive Letter #451?

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