BlueRock Begins First Human Trial of iPSC Cell Therapy for Primary Photoreceptor Diseases

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The Phase I/IIa CLARICO trial of OpCT-001 marks the first clinical trial of an investigational induced pluripotent stem cell-derived therapy for primary photoreceptor diseases.

Close-up of a biometric retina scanner. Image Credit: Adobe Stock Images/The prominence

Image Credit: Adobe Stock Images/The prominence

Key Takeaways

· First-in-human trial begins for iPSC therapy: BlueRock Therapeutics launches CLARICO, the first clinical study testing an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for primary photoreceptor diseases.

· OpCT-001 targets inherited retinal disorders: The investigational therapy aims to restore vision in patients with conditions such as retinitis pigmentosa and cone-rod dystrophy.

· Phased approach evaluates safety and vision outcomes: The two-part trial will assess safety, dose response, and potential improvements in visual function and retinal cell integration.

BlueRock Therapeutics, a Bayer subsidiary, announced that the first patient has been dosed in the Phase I/II CLARICO trial (NCT06789445), which is evaluating OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived therapy for primary photoreceptor diseases. According to the company, the trial is the first to test an iPSC-derived cell therapy in this patient population, which includes individuals with inherited retinal disorders such as retinitis pigmentosa and cone-rod dystrophy.1,2

What Makes This a First for iPSC-Derived Therapies in Retinal Disease?

“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, BlueRock Therapeutics, in a press 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.”1

CLARICO Trial Design and Dose Escalation Strategy

  • The first-in-human, multisite, two-part interventional CLARICO trial will evaluate the safety, tolerability, and efficacy of OpCT-001 in approximately 54 adults with primary photoreceptor disease.
  • Enrollment criteria include patients with a genetically confirmed diagnosis of primary photoreceptor disease BCVA at screening for Phase I: LogMAR 3.9 to LogMAR 1.0; BCVA at screening for Phase II: ETDRS letter score 20 and 60; and retinal structure examination in the study eye showing regions suitable for cell administration.
  • According to BlueRock, Phase I will focus on safety and includes a dose-escalation design across four cohorts, with a total of 12 to 24 legally blind participants receiving one of four planned dose levels.
  • Dosing will follow a standard 3+3 escalation model, enrolling approximately three to six patients per cohort.
  • Phase II will focus on gathering additional safety data, as well as evaluating the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.
  • Up to 15 participants will be enrolled in two randomized cohorts to receive one of two OpCT-001 doses selected based on Phase I data.1
  • Phase II participants will be randomly assigned in a 1:1 ratio to either cohort 5 or cohort 6.
  • Participants enrolled in the Phase II portion and the trial’s investigator and site personnel who are not part of the surgical team will be masked to OpCT-001 dose assignments.

Scope and Unmet Need in Primary Photoreceptor Diseases

According to BlueRock, primary photoreceptor diseases are a subgroup of inherited retinal diseases (IRDs). It is estimated that anywhere from 4.5 million to 6.8 million people are affected by an IRD globally. Notably, primary photoreceptor diseases account for an estimated 65% of all IRDs.3 In the United States, these diseases are estimated to affect approximately 110,000 people, including both children and adults.1

“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, EVP, global head, research and development, pharmaceuticals division, Bayer, in the press release. “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.”

References

1. BlueRock Therapeutics announces first patient receives investigational therapy in Phase 1/2a clinical trial of OpCT-001 for the treatment of primary photoreceptor diseases. Bayer. July 8, 2025. Accessed July 8, 2025. https://www.bayer.com/media/en-us/bluerock-therapeutics-announces-first-patient-receives-investigational-therapy-in-phase-12a-clinical-trial-of-opct-001-for-the-treatment-of-primary-photoreceptor-diseases/

2. A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO). ClinicalTrials.gov. Updated June 24, 2025. Accessed July 8, 2025. https://www.clinicaltrials.gov/study/NCT06789445.

3. About Primary Photoreceptor Disease. BlueRock. Accessed July 8, 2025. https://www.bluerocktx.com/the-science/programs/ophthalmology/#:~:text=Primary%20photoreceptor%20diseases%20are%20a,by%20photoreceptor%20degeneration%20or%20dysfunction.

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