BlueRock’s Photoreceptor Cell Therapy to Move into Clinical Trial

BlueRock Therapeutics, a cell therapy company and wholly owned subsidiary of Bayer AG, has received authorization from the US Food & Drug Administration to launch a Phase 1/2 clinical trial for OpCT-001, a photoreceptor cell therapy for people with inherited retinal diseases such as retinitis pigmentosa and cone-rod dystrophy. The study will assess several dose levels of OpCT-001 and is expected to enroll participants in sites across the US. Additional details of the trial are forthcoming.

The photoreceptors to be used in the study will be derived from induced pluripotent stem cells (iPSCs). To produce iPSCs, investigators take a small sample of blood or skin from an adult human donor. The cells are then genetically tweaked to revert to a stem-cell-like state. As stem cells, they can be coaxed to develop into virtually any cell type in the body, including photoreceptors. Furthermore, billions of cells (many therapy doses) can be produced from the cell sample.

OpCT-001 was licensed by FUJIFILM and Opsis Therapeutics, a company co-founded by David Gamm, MD, PhD, a world-renowned retinal cell therapy pioneer at the University of Wisconsin-Madison.

The Foundation Fighting Blindness provided significant funding over several years to Dr. Gamm and his team for the development of retinal and photoreceptor cell therapies derived from iPSC.