BlueRock Starts OpCT-001 Trial for Retinal Diseases

Bayer and its subsidiary BlueRock Therapeutics have 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.

A First in Clinical Testing for iPSC-Derived Therapy in PR Diseases

OpCT-001 is the first iPSC-derived investigational cell therapy to be evaluated in humans for the treatment of primary photoreceptor (PR) diseases. These diseases are a subgroup of inherited retinal disorders that include retinitis pigmentosa and cone-rod dystrophy, which affect the structure and function of retinal photoreceptor cells, leading to irreversible vision loss in both children and adults. Currently, treatment options for these conditions are extremely limited, with an estimated 110,000 individuals affected in the U.S.

OpCT-001 is designed to restore vision by replacing degenerated photoreceptor cells in the retina with functional cells. The therapy remains investigational and has not been approved by any regulatory authority; its safety and efficacy have not yet been established or fully evaluated.

Details of the CLARICO Clinical Trial

CLARICO is a phase 1/2a, first-in-human, multi-center, two-part interventional study that plans to enroll up to approximately 54 adults with primary photoreceptor disease.

• Phase 1 will concentrate on safety using a dose-escalation approach.

• Phase 2 is structured to gather additional safety data and to assess the impact of OpCT-001 on visual function, functional vision, and anatomical measures of engraftment across various clinical subgroups.

Leadership Perspectives on Advancing OpCT-001

“The initiation of the CLARICO trial represents a key milestone for the OpCT-001 program,” said Amit Rakhit, MD, MBA, Chief Development and Medical Officer at BlueRock Therapeutics. “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.”

Christian Rommel, Executive Vice President and Global Head of Research and Development at Bayer Pharmaceuticals, added:

“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. 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.”