BlueRock Therapeutics Announces First Patient Treated in Phase 1/2a Trial of OpCT-001 for Primary Photoreceptor Diseases
Bayer and BlueRock Therapeutics announced that the first patient received the investigational therapy in CLARICO, a phase 1/2a clinical trial of OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for treating primary photoreceptor (PR) diseases, which are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy.
“The initiation of the CLARICO trial represents a key milestone for the OpCT-001 program,” Amit Rakhit, MD, MBA, BlueRock’s Chief Development and Medical Officer, said in a company news release. “We believe OpCT-001 holds significant promise as a novel therapeutic approach for restoring vision in people living with primary photoreceptor diseases, and we look forward to assessing its safety and tolerability profile as we advance this important program in our pipeline.”
CLARICO is a phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and includes a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.
“We aim to transform treatment options for patients facing irreversible vision loss. OpCT- 001, our investigational iPSC-derived cell therapy, has the potential to restore vision for individuals with primary photoreceptor disease,” said Christian Rommel, Executive Vice President and Global Head of Research and Development of the Pharmaceuticals Division at Bayer. “We are excited to announce the first patient in the CLARICO trial, the first-ever clinical trial for an iPSC-derived treatment in this field.”
Primary photoreceptor diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. OpCT-001 aims to restore vision in patients with primary photoreceptor diseases by replacing degenerated cells in the retina with functional cells. Limited treatment options currently exist for treating primary photoreceptor diseases which affect an estimated 110,000 people in the U.S.
OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.