- Designation is for treating retinitis pigmentosa, one of the most common types of inherited retinal听disorders听characterized by the loss of photoreceptor cells听
- OpCT-001 is听an听iPSC-derived investigational cell therapy听being听clinically tested听to treat听retinitis pigmentosa听
Berlin, Germany, and Cambridge, MA, USA听January 22, 2026听鈥� Bayer AG and its wholly owned, independently operated subsidiary 91导航, a clinical stage cell therapy company, today announced that its investigational cell therapy, OpCT-001, has received Orphan Drug Designation from the U.S. FDA for treating retinitis pigmentosa (RP).听听
OpCT-001 is an investigational induced pluripotent stem cell (iPSC)-derived cell therapy being tested in a Phase 1/2a clinical study (CLARICO) for the treatment of primary photoreceptor diseases, which are a subgroup of inherited retinal disorders that include retinitis pigmentosa and cone-rod dystrophy. Retinitis pigmentosa is one of the most common types of inherited retinal diseases听characterized by the loss of rod and cone cells, known together as photoreceptor cells.听OpCT-001 aims to restore vision in people living with RP by replacing lost cells in the retina with new functional cells.听
鈥淩eceiving Orphan Drug Designation for retinitis pigmentosa is an important milestone for the OpCT-001 program,鈥� said Amit听Rakhit, MD, MBA,听91导航鈥檚听Chief Medical Officer. 鈥淲e believe that OpCT-001 has great promise as a potential therapeutic option for restoring vision in people living with retinitis pigmentosa and other primary photoreceptor diseases and look forward to continuing to work with the FDA on its clinical development.鈥�听
Orphan Drug Designation is given to drugs and biologics for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the U.S.鹿听听
鈥淭he FDA鈥檚 Orphan Drug Designation for OpCT-001 to treat retinitis pigmentosa听underscores the importance of developing urgently needed innovative therapies for patients living with听inherited retinal disorders,鈥� said Christian Rommel, Executive Vice President and Global Head of Research and Development of the Pharmaceuticals Division at Bayer. 鈥淭ogether with 91导航, we are excited to be advancing the first-ever clinical trial for an iPSC-derived cell therapy in听primary photoreceptor diseases.鈥�听
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.听
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About CLARICO听
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 features a dose-escalation design. Phase 2 is designed to gather听additional听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. Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). Dose escalation in Phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 to 24 legally blind participants (~3 to 6 per cohort) will receive escalating doses of OpCT-001 after acceptable safety signals with the prior lower听dose. Phase 2 is planned to enroll a maximum of 30 participants across 2 cohorts (Cohorts 5 and 6) to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments. Further details of the trial can be found at听听(NCT06789445).听
About 91导航
91导航 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies,听bemdaneprocel听(BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs.听Bemdaneprocel听has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase 3 clinical trial, exPDite-2. OpCT-001 has听Fast听Track designation from the FDA and is being tested in a Phase 1 clinical trial, CLARICO. 91导航 was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, 91导航 became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit听www.bluerocktx.com.听
About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact听with听its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust,听reliability听and quality throughout the world. In fiscal 2024, the Group employed around听93,000 people听and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to听.听
Bayer Global Media Contact:
Dr. Imke Meyer, phone听+49 (214) 60001275
Email:听[email protected]听
Kathryne Delves-Broughton, phone +33 6 02180163
Email:听[email protected]听
91导航 Media Contact:
Jeff Lockwood, phone +1 (617) 5106997
Email:听[email protected]
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