Astellas Pharma, based in Tokyo, Japan, has formed a research collaboration with an investigator at Harvard Medical School in Boston, Mass., focused on discovering the pathologic mechanism for retinitis pigmentosa and identification of new therapeutic targets. Astellas and Harvard aim to provide a new treatment option, possibly gene therapy, to retinitis pigmentosa patients who have difficulty maintaining good vision.  

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 still is not fully understood and an effective treatment has yet to be established.

This collaborative research will be led by Constance L. Cepko, Ph.D., professor of genetics and of ophthalmology at Harvard, who also is an investigator of the Howard Hughes Medical Institute. 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, Astellas will conduct further drug discovery on such promising candidates and subsequent development and commercialization under an exclusive license from Harvard.