- Striving to establish a new treatment using adeno-associated virus (AAV)-based gene therapy -

TOKYO and PITTSBURGH, PA, September 24, 2020 – Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) and University of Pittsburgh (Chancellor: Patrick Gallagher, Ph.D., “Pittsburgh”) have entered into a research collaboration agreement focused on the discovery and optimization of clinical candidates for the treatment of dry age-related macular degeneration (dry AMD), a back-of-the-eye disease, utilizing the AAV-based gene therapy approach.

Among back-of-the-eye diseases, age-related macular degeneration is one of the leading causes of acquired blindness in the elderly. Dry AMD, in particular, is a disease in which retinal pigment epithelial cells gradually degenerate in the macula of the retina, and visual acuity deteriorates significantly as the disease advances to a severe stage. However, the mechanism leading to such lesions has not been fully elucidated, and no clinically effective treatment has yet to be established.

In this collaboration, Debasish Sinha, Ph. D., Jennifer Salvitti Davis, M.D. Chair in Ophthalmology Research and Professor of Ophthalmology, Cell Biology and Developmental Biology in University of Pittsburgh School of Medicine, a world-renowned authority in the research field of back-of-the-eye diseases, and Astellas, who has capabilities in drug discovery, will work jointly to evaluate and optimize the drug candidates with an aim to provide new treatment options that help recover and maintain vision for the patients suffering from dry AMD.  Astellas has the exclusive negotiation right for further development and commercialization pertaining to clinical candidates obtained in this collaboration.

“With the population aging, the negative health impacts and costs of the disease will increase dramatically over the next decade. While effective treatment has been developed for advanced wet AMD, currently there is no treatment or prevention for dry AMD. We look forward to working with Astellas to advance the development of innovative treatments,” said Dr. Sinha.
“At Astellas, Blindness & Regeneration and Genetic Regulation are Primary Focus of our research and development strategy. We are engaged in drug discovery research with new modalities such as cell and gene therapies. In addition to the cell therapy program that is currently being developed, we are confident that this collaboration with Pittsburgh will enable us to accelerate our efforts to deliver innovative treatments for patients with back-of-the-eye diseases who have high unmet medical needs. Through alliances and partnerships with academia and bioventures, and more, Astellas will proactively incorporate various superior capabilities and aim to create innovative medical solutions that will turn innovative science into VALUE for patients,” said Akihiko Iwai, Ph.D., President of Drug Discovery Research at Astellas.


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