- Astellas is collaborating with Harvard's Dr. Constance L. Cepko and her team to investigate the underlying pathogenesis of retinitis pigmentosa (RP) in a gene therapy pact.
- RP is an inherited disease that affects peripheral vision, leading to progressive vision loss, as well as night blindness. It affects 1/4,000 people in the U.S. All told, one in 100 people in the US are genetic carriers.
- This deal was initiated as part of the Astellas Innovation Management (AIM) initiative.
In October 2013, Astellas developed the AIM with the goal of developing external relationships with academic experts and others to collaborate on preclinical development. Joining forces with Dr. Cepko, Professor of Genetics and Professor of Ophthalmology, is part of the AIM initiative.
By using adeno-associated virus vectors—the genes that preserve vision in people who inherit retinitis pigmentosa—Dr. Cepko will focus on how retinal degeneration occurs and progresses in RP patients. Astellas is aiming high with AIM, determined to address unmet medical needs by combining experts from both industry and academia, and providing the support needed to expedite meaningful outcomes.