OpCT-001, Stem Cell-Derived Therapy for Primary Photoreceptor Diseases, Receives FDA Fast Track Designation

The FDA has granted a fast track designation to OpCT-001 (Blue Rock Therapeutics), an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases, according to a news release from Bayer Pharmaceuticals. OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for this subgroup of diseases, which includes retinitis pigmentosa and cone-rod dystrophy.¹

Primary photoreceptor diseases can cause loss of vision over time. | Image Credit: © sakurra - stock.adobe.com

“We are thrilled to receive fast track designation from the FDA for OpCT-001,” Amit Rakhit, MD, MBA, chief development and medical officer of BlueRock Therapeutics, said in a news release. “People living with primary photoreceptor diseases need new treatments that have the potential to restore vision, and we look forward to working closely with the FDA to advance this program through clinical trials.”¹

In patients with primary photoreceptor diseases, the structure and function of photoreceptor cells in the retina are impacted, causing irreversible vision loss among children and adults. Unfortunately, limited treatment options are currently available for treating these diseases, which affect approximately 110,000 patients in the US.¹

By replacing degenerated cells in the retina of patients with functional cells, OpCT-001 aims to restore vision. The degeneration and loss of photoreceptor cells—rods and cones—in the retina, whether because of age, injury, or inherited retinal diseases (IRD), is one of the leading causes of permanent blindness or vision impairment across the world. Critically, these photoreceptors cannot regenerate in humans, allowing for cellular therapies such as OpCT-001 to play a role in replacing them.²

OpCT-001’s mechanism of action allows for it to aid in the recovery of vision for patients with primary photoreceptor diseases. The therapy is comprised of allogeneic human iPSC-derived photoreceptor cells; once these are transplanted, the cells mature into cones—which help recover color vision and central high acuity—and rods—which recover proper vision at low light levels and peripheral vision, according to experts.²

Given the unmet need of the thousands of patients with these diseases, the FDA's fast track designation is designed to expedite the development and review of promising therapeutic candidates for serious conditions that help fill an urgent unmet health need. Drugs that receive this designation can receive more frequent interaction with the FDA, accelerating their development and putting them on the path for approval if proper criteria are met.¹

The FDA has cleared OpCT-001 to proceed to a phase 1/2a clinical trial. BlueRock has also had OpCT-001’s investigational new drug application approved by the agency, and they plan for the clinical trial to assess the safety, tolerability, and impact on visual function with the therapy in patients with these diseases. Additionally, the trial will evaluate OpCT-001 at multiple dose levels and enroll participants across multiple US-based sites.^1,3

Primary photoreceptor diseases constitute health burdens that are often difficult to diagnose and treat while presenting a major impact on patient quality of life. It will be essential for specialty pharmacists, health care professionals, and relevant regulatory authorities to follow the clinical development of OpCT-001, as the therapy shows significant promise as a treatment for these conditions.¹

REFERENCES

1. Bayer Pharmaceuticals. BlueRock Therapeutics receives FDA fast track designation for OpCT-001 for the treatment of primary photoreceptor diseases. News Release. Released February 27, 2025. Accessed March 4, 2025. https://www.bayer.com/en/us/news-stories/treatment-of-primary-photoreceptor-diseases

2. FujiFilm. FujiFilm Cellular Dynamics and Opsis Therapeutics announce license with BlueRock Therapeutics for iPSC-derived cell therapy candidate. News Release. Released January 23, 2024. Accessed March 5, 2025. https://www.fujifilm.com/us/en/news/FCDI_Opsis_BlueRock_Option

3. BlueRock Therapeutics. BlueRock Therapeutics announces FDA clearance of IND application for investigational iPSC-derived cell therapy OpCT-001 for the treatment of primary photoreceptor diseases. News Release. Released September 3, 2024. Accessed March 5, 2025. https://www.bluerocktx.com/bluerock-therapeutics-announces-fda-clearance-of-ind-application-for-investigational-ipsc-derived-cell-therapy-opct-001-for-the-treatment-of-primary-photoreceptor-diseases/