Bayer and BlueRock’s lemiretprocel receives FDA and EMA orphan drug designations for inherited retinal disorders, including retinitis pigmentosa.
Written By: Rishabh Sonawane, BPharm
Reviewed By: Pharmacally Editorial Team
Bayer and BlueRock Therapeutics have received U.S. FDA orphan drug designations for lemiretprocel (OpCT-001) in retinitis pigmentosa and cone-rod dystrophy, while the EMA has granted designations covering broader rod- and cone-dominant inherited retinal disorders.
Regulatory Designations Expand Development Scope
The designations cover an investigational induced pluripotent stem cell (iPSC)-derived cell therapy being evaluated in the Phase 1/2a CLARICO study (NCT06789445). The FDA designations apply specifically to retinitis pigmentosa and cone-rod dystrophy. The EMA designations cover syndromic and non-syndromic inherited retinal disorders (IRDs) with a rod-dominant phenotype and non-syndromic IRDs with a cone-dominant phenotype.
Inherited retinal disorders comprise a diverse group of genetic diseases that progressively damage photoreceptor cells, including rods and cones, and can lead to severe visual impairment or blindness. Available treatment options remain limited for many primary photoreceptor diseases.
iPSC-Derived Cell Therapy Targets Photoreceptor Loss
Lemiretprocel is an iPSC-derived investigational cell therapy intended to replace lost retinal cells with functional cells. The therapy is being developed for primary photoreceptor diseases, a subgroup of IRDs that includes retinitis pigmentosa, Usher syndrome and cone-rod dystrophies.
Rather than treating a single causative genetic mutation, the cell-replacement approach seeks to address photoreceptor cell loss across multiple disease settings. However, the clinical ability of transplanted cells to survive, integrate into the retina and restore visual function remains under investigation.
CLARICO Study Evaluates Safety and Visual Outcomes
The first-in-human CLARICO study (NCT06789445) is a multisite Phase 1/2a interventional trial expected to enroll up to approximately 54 adults with primary photoreceptor disease.
Phase 1 focuses primarily on safety and tolerability using a dose-escalation design. Four planned dose levels will be evaluated across four cohorts, with approximately three to six legally blind participants per cohort. The study uses a standard 3+3 escalation approach, with progression to higher doses based on acceptable safety findings at the preceding dose.
Phase 2 will enroll up to 30 additional participants across two cohorts. Two dose levels selected from Phase 1 safety and tolerability findings will be evaluated, with participants randomized 1:1 between the cohorts. Participants and investigators outside the surgical team will remain masked to dose assignment.
The Phase 2 assessment will include safety as well as visual function, functional vision and anatomic measures of cell engraftment. No efficacy conclusions can yet be drawn because the therapy remains investigational and clinical evaluation is ongoing.
Regulatory Recognition Supports Further Development
Amit Rakhit, BlueRock’s chief medical officer, said the FDA and EMA designations represent an important development milestone and support continued engagement with health authorities during lemiretprocel’s clinical development.
Christian Rommel, Bayer’s global head of research and development for pharmaceuticals, said the designations recognize the potential application of the therapy across multiple inherited retinal disorders.
The regulatory designations do not establish efficacy or safety and do not constitute marketing authorization. Lemiretprocel has not been approved by the FDA, EMA or any other regulatory authority.
Next Steps in Clinical Development
The immediate priority is completion of Phase 1 dose escalation and generation of safety and tolerability data to inform Phase 2 dose selection. Subsequent evaluation will determine whether cell engraftment translates into measurable improvements in visual function and functional vision across relevant patient subgroups.
As an iPSC-derived cell replacement therapy, lemiretprocel could potentially be evaluated across multiple inherited retinal diseases, but its clinical benefit will depend on evidence from controlled clinical development.
Reference
BlueRock Therapeutics’ investigational cell therapy lemiretprocel (OpCT-001) for primary photoreceptor diseases granted orphan drug designations by FDA and EMA, BlueRock Therapeutics, 30 September 2026
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO) (CLARICO), ClinicalTrials.gov ID NCT06789445
About the Writer
Rishabha Sonawane, B.Pharm (LinkedIn) is healthcare writer with a strong interest in medical writing, regulatory affairs, clinical research, and AI-driven drug discovery. He has completed specialized training from the NIH and ICMR in clinical pharmacology, clinical research, and scientific writing. Passionate about evidence-based healthcare communication, he focuses on translating complex scientific research into clear, accurate, and engaging medical content.