Sharing is caring!


The Phase 1 (Ph1), open-label study aims to evaluate the safety, tolerability and preliminary efficacy of pluripotent stem cell-derived dopaminergic neurons in patients with advanced Parkinson’s disease (PD), and has dosed its first patient.

“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,” said Emile Nuwaysir, Ph.D., President and Chief Executive Officer of BlueRock.

About the DA01 Phase 1 Trial

Ten patients will be enrolled at sites in the US and Canada. Dopamine-producing cells are surgically transplanted into the putamen as part of the trial, and 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 Centre. The primary objective of the study is to evaluate the safety and tolerability of DA01 cell transplantation after one year. The secondary objectives are to assess the evidence of transplanted cell survival and motor effects at one- and two-year intervals, as well as continued safety and tolerability, and feasibility of transplantation. 

More information on the trial can be found at clinicaltrials.gov.

About Parkinson’s Disease

Parkinson’s disease is the second most common neurodegenerative disorder, affecting over 7.5 million people. Nerve cell damage in the brain leads to a drop in dopamine levels, and the loss of dopamine-producing neurons leads to worsening motor and non-motor symptoms. 60-80% of dopaminergic neurons are estimated to have already been lost at diagnosis. 

About BlueRock Therapeutics

A clinical-stage biopharmaceutical company and wholly owned subsidiary of Bayer AG, BlueRock Therapeutics aims to develop regenerative cell therapies for intractable diseases. The company’s revolutionary 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. The company is currently focused on neurology, cardiology and immunology. For more information, visit bluerocktx.com.

About Bayer

A global enterprise focused on the fields of healthcare and nutrition, Bayer’s products and services are designed for the benefit of people and the planet, while the Group’s goal is also to increase earning power and create value through innovation and growth. For more information, visit www.bayer.com.

(Source: Cision, 8th June 2021)