BlueRock Therapeutics and Foundation Fighting Blindness Partner for IRD Study
BlueRock Therapeutics LP, a clinical stage cell therapy company and subsidiary of Bayer, and Foundation Fighting Blindness announced a collaboration to add a new cohort to the Foundation’s 'Uni-Rare natural history study' of people living with inherited retinal diseases (IRDs) caused by rare single gene mutations. The new cohort will include patients living with IRDs caused by mutations in multiple genes. BlueRock plans to use the data generated by the Uni-Rare study to support the identification and development of sensitive, reliable outcome measures for clinical trials and to enable the discovery and development of new therapies for treating IRDs.
“BlueRock is developing a pipeline of cell therapies that we believe has great potential for restoring vision in people living with blindness caused by retinal disease,” Ahmed Enayetallah, Senior Vice President and Head of Development for BlueRock Therapeutics, said in a news release. “We are excited to be working with the team at the Foundation Fighting Blindness to expand their study which will help inform the design of future clinical trials as our ophthalmology pipeline advances.”
Launched in 2022, the Uni-Rare study is a first-of-its-kind natural history study for approximately 1,500 people with one of more than 300 rare genes associated with IRDs including retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and a broad range of other conditions. The study is led by José-Alain Sahel, MD, Distinguished Professor and Chairman of the Department of Ophthalmology at the University of Pittsburgh School of Medicine.
“The Uni-Rare study is an ideal platform for the Foundation to collaborate with life science companies, like BlueRock, that share our goal of better-informed clinical trial designs for treatments of IRDs,” said Todd Durham, Senior Vice President of Clinical and Outcomes Research at the Foundation Fighting Blindness. “We are thrilled to partner with BlueRock on the addition of this multigene cohort that will provide more affected individuals in the IRD community with an opportunity to make a meaningful research impact.”
In January, BlueRock announced that it had exercised its option to exclusively license OpCT-001, an induced pluripotent stem cell (iPSC) derived cell therapy candidate being explored for the treatment of Primary Photoreceptor disease, from FUJIFILM Cellular Dynamics and Opsis Therapeutics. Primary photoreceptor diseases are a subgroup of inherited retinal diseases that includes retinitis pigmentosa and cone-rod dystrophies. These diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. No current treatment options exist for this patient population. OpCT-001 aims to restore vision loss caused by these diseases by replacing degenerated tissue in the retina with functional cells. An IND filing is planned for this year.