Astellas & Harvard Take On Retinitis Pigmentosa

Astellas will fund a study of genetic retinal degenerative disease run by Dr. Constance Cepko from Harvard University.

AsianScientist (Oct. 24, 2014) – Astellas Pharma Inc. will collaborate with an investigator at Harvard Medical School in a study focused on discovering the pathologic mechanism for retinitis pigmentosa and identification of new therapeutic targets.

Retinitis pigmentosa is a retinal degenerative disease caused by genetic mutations and characterized by loss of peripheral and night vision, followed by loss of color vision and complete blindness. The pathogenesis of the retinal degeneration is still not fully understood and an effective treatment has yet to be established.

This collaborative research will be led by Dr. Constance L. Cepko, Professor of Genetics and of Ophthalmology at Harvard University, who is also an investigator of the Howard Hughes Medical Institute, a world-renowned authority on ophthalmology and gene therapy. Using adeno-associated virus vectors, genes that prolong vision in those who inherit retinitis pigmentosa will be identified and verified.

Astellas will support the collaboration for up to three years. If the collaboration is successful in identifying genes that lead to promising treatment options for patients, Astellas will conduct further drug discovery on such promising candidates, and subsequent development and commercialization under an exclusive license from Harvard.

“For the purpose of enhancing the ability to generate new drugs, Astellas is looking for challenges in new therapeutic areas and novel technology platforms as well as new drug discovery opportunities that satisfy unmet medical needs through collaboration with external partners. It is our hope that this collaboration to advance drug discovery will bring light to patients who struggle with retinitis pigmentosa,” said Dr. Kenji Yasukawa, Senior Vice President and Chief Strategy Officer, Astellas.

Dr. Cepko had the following to say: “We are hopeful that some of our candidates will preserve the color and daylight vision of RP patients, regardless of the disease gene in their family. These treatments may also be effective in people that have other types of ocular disease, such as age-related macular degeneration, or glaucoma.”

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Source: Astellas.
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