Astellas Pharma is collaborating with Constance L. Cepko, Ph.D., professor of genetics and of ophthalmology at Harvard Medical School, to discover the pathologic mechanism for retinitis pigmentosa and idenitify new therapeutic targets for it. Astellas and Harvard say their aim will be to provide a new treatment option with a view of a gene therapy to retinitis pigmentosa patients who have difficulty maintaining good vision.

Dr. Cepko, who is also an investigator with the Howard Hughes Medical Institute, will lead a research team that will use adeno-associated virus vectors to identify genes that prolong vision in people who inherit retinitis pigmentosa. Astellas says it will support the collaboration for up to three years; if successful, the Japanese firm plans to conduct further drug discovery on such candidates as well as develop and commercialize them 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.” Astellas' svp and CSO Kenji Yasukawa, Ph.D., said in a statement. “This collaboration with Professor Cepko at Harvard is a part of this strategy.”

“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,” Dr. Cepko commented, adding that these treatments may also be effective in patients with other eye conditions such as glaucoma or AMD.








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