Mar 25, 2024

BlueRock Therapeutics and Foundation Fighting Blindness announce collaboration to expand the Uni-Rare natural history study of patients living with inherited retinal diseases

Press Releases

Collaboration will add a new multi-gene cohort of patients living with inherited retinal diseases. Data insights from the new study cohort will inform the future clinical trial design for BlueRock’s pipeline of cell therapies for treating blindness.

CAMBRIDGE, MA USA and COLUMBIA, MD, March 25, 2024 – BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG, and Foundation Fighting Blindness today 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,” said Ahmed Enayetallah, Senior Vice President and Head of Development for BlueRock Therapeutics. “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 Dr. José-Alain Sahel, 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.


Media Contacts
Jeff Lockwood
Vice President, Communications

Chris Adams
Vice President, Marketing & Communications

About BlueRock Therapeutics LP
BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to reverse devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for patients suffering from neurological, cardiovascular, immunological, and ophthalmic diseases. Our lead clinical program, bemdaneprocel, (BRT-DA01) is in Phase I clinical trials for Parkinson’s disease. We were 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, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy Platform. 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 us on LinkedIn.

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, “Health for all, Hunger for none,” the company’s 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 with 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, reliability, and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to

Follow us on Facebook: Follow us on Twitter: @BayerPharma

About the Foundation Fighting Blindness
Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $891 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit for more information.

Forward-Looking Statements
Certain statements in this press release are forward-looking which may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate” and “intend,” among others. These forward-looking statements are based on BlueRock’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the outcomes from our clinical trials and ongoing FDA and other regulatory requirements.

As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of BlueRock’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof.