VCBeat Health Contact Us
Home BlueRock Therapeutics Doses First Patient in Phase 1 Trial of DA01, the First-in-Class Pluripotent Stem Cell-Derived Dopaminergic Neuron Therapy for Parkinson’s Disease

BlueRock Therapeutics Doses First Patient in Phase 1 Trial of DA01, the First-in-Class Pluripotent Stem Cell-Derived Dopaminergic Neuron Therapy for Parkinson’s Disease

Jan 23, 2022 03:35 CST Updated 03:35
Bayer

Pharmaceutical Product R&D Developer

BlueRock Therapeutics

Induced Pluripotent Stem Cell (iPSC) Therapy Developer

University Health Network

University Health Network (UHN) is a significant milestone in Canada’s healthcare system and serves as a teaching hospital affiliated with the University of Toronto. UHN’s vision is to achieve global impact, while its mission is to provide exemplary patient care, medical research, and education. Its purpose is to create a compassionate, innovative, and accountable teaching hospital that promotes health for patients, society, and the world. Its core values include care, excellence, teamwork, innovation, and respect. In October 2008, UHN was named one of “Canada’s Top 100 Employers” by Mediacorp Canada Inc., and in November of the same year, it was recognized as a “Toronto Star Top Employer” by the Toronto Star. The UHN logo features three profile silhouettes, representing the three constituent hospitals as well as three commitments: patient care, medical research, and medical education. Together, they are interconnected like leaves to form a cohesive circle, symbolizing collaboration and completeness. The gold color of the graphic signifies quality, reflecting a commitment to continuous exploration through a successful mindset and medical excellence to meet growing challenges and realities. The blue color of the text represents the hospital’s traditional color.


News on January 22, 2022 /BioValleyBIOON/ --BayerBayer’s wholly-owned subsidiary BlueRock Therapeutics recently announced that the first patient has been dosed in an open-label Phase 1 study (Ph1) evaluating DA01 for the treatment of advanced Parkinson's disease (PD), in collaboration with the University Health Network (UHN).

DA01 is a multi-potentStem CellsDerived dopaminergic neurons are the first-of-their-kind cell therapy, with the potential to regenerate lost function and possibly improve and reverse Parkinson's disease.Currently, DA01 is undergoing clinical evaluation in a subset of advanced Parkinson's disease patients for whom conventional drug therapy is no longer fully effective.DA01 is transplanted into the patient's brain through surgery, integrating into the brain and restoring lost physiological functions.. If successful, this treatment method will bring significant progress to Parkinson's disease.

The primary objective of the Phase 1 trial is to evaluate the safety and tolerability of DA01 cell transplantation one year post-transplant. Secondary objectives include assessing evidence of transplanted cell survival and motor effects at 1 and 2 years post-transplant, evaluating continued safety and tolerability over a 2-year period, and assessing the feasibility of the transplantation.

Dopaminergic neurons (Image source: medicalnewstoday.com)

Parkinson's disease (PD) is a progressive neurodegenerative disorder caused by damage to nerve cells in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is due to the loss of dopamine-producing neurons.It is estimated that patients have already lost 60-80% of their dopaminergic neurons at the time of diagnosis.

Parkinson's disease usually begins with a tremor in one hand. Other symptoms include stiffness, spasms, and slow movement (bradykinesia). According to the Parkinson’s Foundation, more than 10 million people worldwide are living with Parkinson’s disease, including one million in the United States.

BlueRock Therapeutics is a leading engineered cell therapy company with a mission to create authentic cellular medicines to reverse devastating diseases and a vision to improve the human condition. The company’s Cell+Gene platform harnesses the power of cells for neurology, cardiology,ImmunologyDeveloping innovative therapies for ophthalmic indications.

BlueRock Therapeutics' cell differentiation technology recapitulates the developmental biology of cells to produce truly authentic cells, some of which are engineered for additional therapeutic functions. Using these cell therapies to replace damaged or degenerated tissues holds the potential to restore lost function. BlueRock Therapeutics was founded in 2016 by Versant Ventures and Bayer and capitalized with one of the largest Series A financings in biotech history by Bayer and Versant Ventures. The company was fully acquired by Bayer in 2019. (Bioon.com)