Astellas, Harvard Partner On Gene Therapy For Retinitis Pigmentosa

Astellas Pharma and researchers at Harvard intend to partner and research treatments for retinitis pigmentosa. The collaboration, announced in a press release, is headed by Constance L. Cepko, Ph.D., a professor of Genetics and Ophthalmology at Harvard. The researchers will identify genes that support vision in patients with inherited retinitis pigmentosa using adeno-associated virus vectors.

Kenji Yasukawa, Ph.D., SVP and Chief Strategy Officer at Astellas, said, "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 and Harvard Agreement

Astellas states that it will support the research at Harvard for three years. It hopes to be successful at identifying genes that prolong vision in those who inherit retinitis pigmentosa. If those discoveries identify treatments, Astellas will continue developing promising candidates under licensing agreements with Harvard. Dr. Cepko, also affiliated with the Hughes Medical Institute, said, “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."

Adeno-Associated Virus Vectors

Astellas and Harvard intend to use adeno-associated virus vectors in gene research on retinitis pigmentosa, a degenerative disease that causes a loss of peripheral vision, night vision, and color vision, followed by total blindness. Adeno-associated virus (AAV) vectors are being used more frequently as delivery vehicles for gene therapy applications because AAV vectors can infect non-dividing and dividing cells persistently without pathogenicity. Researchers have found that AAV vectors perform sufficiently in animal models and have demonstrated safety in humans. However, it still is being proven that AAV vectors can be effective in human.  More work is needed on AAV basic biology, efficient AAV vectors, and novel applications.

Astellas Ophthalmology

Astellas does not list Ophthalmology in its therapeutic areas, nor does it have ophthalmology candidates in its pipeline. However, as Dr. Yasukawa said, the company is looking at new treatment areas. He also said that Astellas seeks partnerships for new ventures. In August, Astellas partnered with Cancer Research UK and its commercial arm, Cancer Research Technology, to find treatments for pancreatic cancer.