BlueRock Therapeutics begins Phase 1/2a trial of OpCT-001 for primary photoreceptor diseases with first patient treated

BERLIN, Germany & CAMBRIDGE, MA, USA | July 08, 2025

Bayer AG, along with its wholly owned subsidiary, BlueRock Therapeutics LP, has initiated a groundbreaking clinical trial named CLARICO, which tests an investigational therapy, OpCT-001. This therapy is being explored as a potential treatment for primary photoreceptor diseases, a category of inherited retinal disorders. OpCT-001 is a pioneering induced pluripotent stem cell (iPSC)-derived therapy. This marks the first instance of such a therapy undergoing clinical evaluation for primary photoreceptor diseases, which encompass conditions like retinitis pigmentosa and cone-rod dystrophy.

BlueRock's Chief Development and Medical Officer, Dr. Amit Rakhit, emphasized the importance of this trial, stating, "The launch of the CLARICO trial is a pivotal step for the OpCT-001 program. We believe this therapy has immense potential in restoring vision for those affected by primary photoreceptor diseases. Our goal is to evaluate its safety and tolerability as we move forward with this crucial initiative."

The CLARICO trial, which is a Phase 1/2a study, aims to examine the safety, tolerability, and clinical impact of OpCT-001 on up to 54 adults suffering from primary photoreceptor diseases. The first phase will concentrate on safety using a dose-escalation model, while the second phase will collect further safety data and assess the therapy's impact on visual function and structural integration in various patient subgroups.

Christian Rommel, Executive Vice President and Global Head of Research and Development at Bayer's Pharmaceuticals Division, commented, "Our goal is to revolutionize treatment options for patients experiencing vision loss. OpCT-001 has the potential to restore sight in individuals with primary photoreceptor disease. We are thrilled to announce the first patient in this trial, which is the inaugural clinical evaluation of an iPSC-derived treatment in this domain."

Primary photoreceptor diseases impair the structure and function of retinal photoreceptor cells, resulting in irreversible vision loss in both children and adults. The therapeutic aim of OpCT-001 is to replace the degenerated cells with functional ones to restore vision. Currently, treatment options for these conditions are limited, affecting approximately 110,000 people in the United States.

OpCT-001 remains an investigational therapy that has not yet received approval from regulatory bodies, and its safety and efficacy are still under evaluation.

CLARICO, as a first-in-human, multisite, two-part study, seeks to evaluate OpCT-001’s safety and effectiveness in adults with primary photoreceptor disease. The first phase includes a dose-escalation approach, where four planned dose levels will be tested across four cohorts. Using a conventional 3+3 model, the first phase will involve 12 to 24 legally blind participants. Phase 2 plans to enroll up to 15 participants in two cohorts, with doses determined by the safety and tolerability findings from Phase 1. Participants will be randomly assigned to either cohort, and those outside the surgical team will remain unaware of the dose assignments. Detailed trial information is available under identifier NCT06789445 at clinicaltrials.gov.

BlueRock Therapeutics is a clinical-stage company focused on developing cell therapies for treating severe diseases, including neurological and ophthalmic conditions. As a subsidiary of Bayer AG, BlueRock is driven by a commitment to innovation and delivering hope to patients in need.