BlueRock Therapeutics Announces First Patient Dosed with DA01 in Phase 1 Study in Patients with Advanced Parkinson's Disease

CAMBRIDGE, Mass., June 8, 2021 /PRNewswire/ -- BlueRock Therapeutics, a clinical stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, announced today the dose administration for the first patient in a Phase 1 (Ph1), open-label study of pluripotent stem cell-derived dopaminergic neurons in patients with advanced Parkinson's disease (PD). The purpose of the Ph1 clinical trial is to evaluate the safety, tolerability, and preliminary efficacy of DA01 in patients with PD.

"It's both exciting and humbling to be a part of delivering this first-of-its-kind medicine to Parkinson's patients, with the hope of re-innervating the human brain to make profound and lasting improvements to the quality of life for PD patients," said Emile Nuwaysir, Ph.D., President and Chief Executive Officer of BlueRock. "Our objective is to use authentic cells, to have them integrate entirely into the brain and restore lost physiologic function. If successful, this new therapeutic modality could have implications for the Parkinson's community and beyond."

About the DA01 Phase 1 Trial
The initial study site is Memorial Sloan Kettering Cancer Center and Weill Cornell Medical Center, under the guidance of Dr. Sarva, M.D., Principal Investigator (PI). The trial will enroll ten patients at sites in the US and Canada. In this study, subjects undergo surgical transplantation of the dopamine-producing cells into the putamen. The first surgery was performed by Dr. Viviane Tabar, Chair of the Department of Neurosurgery and Theresa Feng Chair in Neurosurgical Oncology at Memorial Sloan Kettering Cancer Center. The primary objective of the Ph1 study is to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant. The secondary objectives of the study are to assess the evidence of transplanted cell survival and motor effects at one- and two-years post-transplant, to evaluate continued safety and tolerability at two years, and to assess the feasibility of transplantation.

Additional sites will be activated at the University of California, Irvine (PI: Dr. Henchcliffe, M.D., D.Phil.), and the University of Toronto (UHN) (PI: Dr. Lozano, O.C., M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S.).

More information about this trial is available at (NCT#04802733).

About Parkinson's Disease
Parkinson's disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons. At diagnosis, it is estimated that patients have already lost 60-80% of their dopaminergic neurons. Parkinson's disease often starts with a tremor in one hand. Other symptoms are rigidity, cramping and slowness of movement (bradykinesia). Parkinson's disease is the second most common neurodegenerative disorder, impacting more than 7.5 million people, including 1.3 million people in North America.

About BlueRock Therapeutics
BlueRock Therapeutics is an engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. The company's cell+gene platform enables the creation, manufacture, and delivery of authentic cell therapies with engineered functionality by simultaneously harnessing pluripotent cell biology and genome editing. This enables an approach where, in theory, any cell in the body can be manufactured and any gene in the genome can be engineered for therapeutic purposes. The platform is broadly applicable, but the company is focused today in neurology, cardiology, and immunology. In August 2019, the company was acquired by Bayer AG, for an enterprise value of $1B in upfront and milestone payments. For BlueRock this marks the next step in the journey to prove degenerative disease is reversible, and to bring our revolutionary new medicines to the patients who desperately need them. For more information, visit

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate 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 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses before special items amounted to 4.9 billion euros. For more information, go to

Dr. Tabar has financial interests related to BlueRock. Researchers at Memorial Sloan Kettering Cancer Center (MSK) developed stem cell-derived dopaminergic neurons for the treatment of neurodegenerative diseases, and MSK licensed this intellectual property to BlueRock. MSK has institutional financial interests related to this intellectual property and BlueRock.

Forward-Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements 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 timing of our clinical trial for DA01; our results regarding the safety, tolerance and efficacy of DA01 cell transplantation for patients with Parkinson's disease; and ongoing FDA and other regulatory requirements regarding the development of DA01. 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 the Company'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.

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SOURCE BlueRock Therapeutics

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