Harvard and Astellas Pharma collboarate on gene therapy for Retinitis Pigmentosa

Astellas Pharma Inc. has announced a research collaboration with Constance L Cepko, Ph.D., an investigator at Harvard Medical School, Boston, Massachusetts, focused on discovering the pathologic mechanism for retinitis pigmentosa and identification of  new therapeutic targets. Astellas and Harvard will work with the aim to provide a new treatment option with a view of a 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 is still not fully understood and an effective treatment has yet to be established.

This collaborative research will be led by Dr Cepko, Professor of Genetics and of Ophthalmology at Harvard, who is also an investigator of the Howard Hughes Medical Institute, and is 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.
 
The collaborators are hopeful that some of their candidates will help 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.