Bayer & BlueRock Therapeutics: A First Neuronal Stem Cell Therapy to Show Positive Results in Phase I Clinical Study for Parkinson’s Disease    

Bayer and BlueRock Therapeutics

Berlin, Germany and Cambridge, M.A., U.S.A., June 28, 2023 – Bayer AG (BAYRY) and its subsidiary firm BlueRock Therapeutics LP announced positive top-line results from a Phase I clinical trial of the investigational drug bemdaneprocel (BRT-DA01), a potential first-in-class cell therapy for Parkinson’s disease.

Bemdaneprocel was well-tolerated in all 12 patients in the study to date with no major safety events.   

Secondary assessment of endpoints demonstrated feasibility of transplantation and evidence of cell survival and engraftment in the brain through one year.

Based on these results, a Phase II study is expected to begin enrolling patients in the first half of 2024. The topline data provide a strong rationale for initiating the next phase study, and we look forward to advancing this clinical program.”

Parkinson’s disease

Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. At diagnosis, it is estimated that patients have already lost 50-80% of their dopaminergic neurons. The loss of these neurons leads to a progressive loss of motor function and symptoms such as tremors, muscle rigidity, and slowness of movement (bradykinesia).

According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with around one million living in the United States.

No cure exists for Parkinson’s Disease and the efficacy of the current treatments decreases over time.  

Bemdaneprocel (BRT-DA01) – an investigational therapy dopamine producing neurons derived from pluripotent stem cells, are surgically implanted into the brain of a person with Parkinson’s disease. When transplanted, these cells have the potential to reform neural networks that have been destroyed by Parkinson’s disease in the hope of restoring motor and non-motor function to patients.

From Bayer

Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development said, “At Bayer, we are committed to advancing cell and gene therapy innovations for patients with Parkinson’s disease, a neurodegenerative disorder with debilitating effects on people’s lives for which there is currently no cure and only limited treatment options. The positive outcome of our first cell therapy clinical trial for Parkinson’s is encouraging not only for the bemdaneprocel development program but also our entire pluripotent stem cell-based platform and warrants further investigation in larger groups of patients.”

Detailed Phase I trial data from primary and secondary endpoints will be presented at the  International Parkinson’s Disease Congress and Movement Disorders in Copenhagen from Aug. 27 – 31. 2023.

About the Bemdaneprocel Phase I Trial

The primary objective of the Phase I trial is to assess the safety and tolerability of bemdaneprocel (BRT-DA01) transplantation at one-year post-transplant. The secondary objectives of the trial 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 feasibility of transplantation.  

The transplant surgeries were performed by the following physicians:

Dr. Viviane Tabar, MD, Chair of the Department of Neurosurgery at Memorial Sloan Kettering (MSK) Cancer Center

Dr. Andres Lozano, M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S., Neurosurgeon and Senior Scientist, Krembil Brain Institute, Alan & Susan Hudson Cornerstone Chair in Neurosurgery, Toronto Western Hospital, University Health Network and Chairman of the Division of Neurosurgery at the University of Toronto (UoT).

Participants were followed at clinical sites by Dr. Harini Sarva, M.D. at Weill Cornell Medicine, Dr. Claire Henchcliffe, M.D., D.Phil., F.A.A.N., F.A.N.A. at the University of California, Irvine, and Dr. Alfonso Fasano, M.D., PhD., Chair in Neuromodulation and Multi-Disciplinary Care at the University Health Network (UHN) and UoT.  


Memorial Sloane Kettering (MSK): Dr. Tabar has financial interests related to BlueRock. MSK has institutional financial interests related to BlueRock.

Note the foregoing institutional disclosure language is included because the referenced study relates to MSK technology licensed to BlueRock. 

University Health Network (UHN): UHN has institutional financial interests related to BlueRock.  

More information about the Phase I trial is available at (NCT04802733).

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. 

BlueRock Therapeutics was 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


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 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 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros.

R&D expenses before special items amounted to 6.2 billion euros.

For more information, go to

BlueRock Therapeutics Media Contact:

Jeff Lockwood:  Phone +1 617.510.6997                     Email: [email protected]

Bayer Media Contact:

Dr. Julia Schulze, Phone +49 175 5866432.                Email: [email protected]

Bayer U.S. Media Contact:

Sue Ann Pentecost, Phone +1 910.221.6446   Email: [email protected]

Bayer (BAYGn.DE) subsidiary BlueRock has become the first company to report initial success treating Parkinson’s disease in humans using an experimental stem cell therapy, the drugmaker said on Wednesday.

The hunt for Parkinson’s treatments has seen many setbacks over decades.

Bayer said that one year into a Phase I trial with 12 volunteers, BlueRock’s therapy was shown to be well-tolerated and that transplanted cells grew as intended in patients’ brains, which encouraged it to advance testing on humans to the second of three stages, with patient enrolment seen in the first half of 2024.

A slew of research projects around the globe have recently honed in on the approach to transplant modified cells to restore an area of the brain that normally produces dopamine.

Some of this work is done by Britain’s’ Cambridge University, South Korea’s Bundang CHA Hospital, International Stem Cell Corp’s (ISCO.PK) Cyto Therapeutics in Australia, the Chinese Academy of Sciences, Harvard University and Japan’s Kyoto University Hospital.

For BlueRock’s experimental therapy, researchers took induced pluripotent stem cells, which are modified to regain the ability to form any type of specialized tissue and transformed them into dopamine-producing nerve cells. When surgically implanted into the brain of a person with Parkinson’s disease, the therapeutic cells are designed to restore neural networks destroyed by the disease.

Initial trial results showed the cells multiplied and started making dopamine, an important brain signalling molecule which is lacking in Parkinson’s patients.

Parkinson’s, for which there is no cure, and which affects more than 10 million people worldwide, causes progressive brain damage. Common symptoms are loss of muscle control, tremors, muscle rigidity and slowness of movement while dementia is seen in some patients.

Bayer acquired BlueRock Therapeutics and Asklepios Biopharmaceutical in 2019 and 2020, respectively, in a push to build a cell and gene therapy business, Bayer last year struck a partnership deal with Mammoth Biosciences in the San Francisco Bay area, co-founded by Nobel laureate Jennifer Doudna, to develop therapeutic tools based on CRISPR/CAS9 gene editing.

Prohost Observation

A promising cure for Parkinson’s Disease would be a miracle that is badly needed. We are looking forward to Phase 2, which will start in the first half of 2024. 

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