Using a stem cell-derived therapeutic developed over more than a decade in Dr. David Gamm’s lab at UW-Madison, BlueRock Therapeutics has treated the first patient in a new Phase 1/2a clinical trial aimed at a group of inherited retinal disorders (including RP and cone-rod dystrophy). The initiative is the result of a strategic research alliance formed in 2021 between the Gamm laboratory, Opsis Therapeutics, Fujifilm Cellular Dynamics Inc., BlueRock Therapeutics, and Bayer to bring these therapies to patients. The induced pluripotent stem cell (iPSC)-derived photoreceptor cell product OpCT-001 is designed to restore functional photoreceptors to patients with RP and other retinal diseases leading to primary photoreceptor loss. In response to news of this first treatment, Dr. Gamm noted that “…this is an important and necessary first step in testing this first-of-its-kind therapy, and we are grateful to all of the people participating and monitoring the trial. I am particularly pleased that this strategy and human iPS cell technology in general was born, developed, and refined here at UW-Madison.”
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