Bayer’s BlueRock Therapeutics receives FDA Fast Track Designation for its iPSC OpCT-001 for primary photoreceptor diseases
This milestone positions BlueRock Therapeutics to accelerate clinical development of OpCT-001, aiming to restore vision in patients facing irreversible blindness.
BlueRock Therapeutics, a Bayer subsidiary, just announced that the FDA has granted Fast Track designation for its induced pluripotent stem cell (iPSC)-derived cell therapy, OpCT-001, for primary photoreceptor diseases.
iPSCs are adult cells that are reprogrammed into a stem cell, with the capability to differentiate into a wide variety of tissues. OpCT-001 is reportedly the first investigational therapy derived from iPSCs to enter clinical trials for primary photoreceptor diseases, a subgroup of inherited retinal disorders that include retinitis pigmentosa and cone-rod dystrophy.
Fast Track designation by the FDA is intended to accelerate the development and review process for drug candidates designed to treat serious conditions and unmet medical needs. Therapies receiving this designation can benefit from increased interaction with the FDA regarding clinical development plans and may be considered for accelerated approval and priority review, provided relevant criteria are met.
“We are thrilled to receive Fast Track designation from the FDA for OpCT-001,” said Amit Rakhit, MD, MBA, BlueRock’s Chief Development and Medical Officer. “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.”
Primary photoreceptor diseases affect photoreceptor cells within the retina, causing their degeneration and leading to irreversible vision loss in both children and adults. OpCT-001 seeks to restore visual function by replacing these degenerated retinal cells with healthy, functional photoreceptor cells derived from iPSCs. Currently, limited treatment options exist for primary photoreceptor diseases, which are estimated to affect approximately 110,000 people in the U.S.¹
“Receiving FDA Fast Track designation for OpCT-001 demonstrates the urgent need for innovative treatments in the field of primary photoreceptor diseases,” said Christian Rommel, Executive Vice President, Global Head of Research and Development, and Member of the Pharmaceuticals Leadership Team at Bayer. “We are proud to deliver on our commitment to advancing groundbreaking cell therapies by seeking to develop the first iPSC-derived investigational cell therapy for patients facing devastating consequences from primary photoreceptor diseases.”
OpCT-001 was exclusively licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics in January 2024 as part of a strategic research, development, and clinical manufacturing collaboration established in 2021. Under this collaboration, FUJIFILM Cellular Dynamics supported BlueRock Therapeutics in research, development, and execution of critical IND-enabling activities (studies and preparations required by the FDA before initiating human clinical trials), including the clinical manufacturing of OpCT-001.
OpCT-001 remains investigational and has not yet been approved by any regulatory authority. Its efficacy and safety have yet to be fully evaluated or established.
¹https://www.ncbi.nlm.nih.gov/books/NBK519518/;
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1808442/;
https://www.nidcd.nih.gov/health/usher-syndrome#b
About BlueRock Therapeutics LP
BlueRock Therapeutics LP is a clinical-stage company developing cell therapies aimed at treating serious diseases. The company’s pipeline includes investigational therapies targeting neurological, ophthalmic, cardiovascular, and immunological disorders. Currently, BlueRock is advancing two clinical-stage programs: bemdaneprocel (BRT-DA01)—an investigational cell therapy for Parkinson’s disease—and OpCT-001 for primary photoreceptor diseases. Bemdaneprocel has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA, has completed a Phase 1 clinical trial, and is advancing toward further clinical development. OpCT-001 has recently obtained FDA clearance to initiate Phase 1 clinical studies. BlueRock was founded in 2016 as a joint venture between Versant Ventures and Leaps by Bayer, Bayer AG’s impact investing arm focused on breakthrough innovation. Since 2019, BlueRock has operated as a wholly owned subsidiary of Bayer AG within its Cell & Gene Therapy platform. Additional information can be found at http://www.bluerocktx.com.
