Bayer promotes two first cell and gene therapies to treat Parkinson's disease

Bayer Group announced on June 8 that its wholly-owned clinical trial phase biopharmaceutical company BlueRock (BlueRock Therapeutics) has successfully completed the administration of a Parkinson’s disease patient in an open-label phase I clinical study.
The first dose of dopaminergic neurons DA01 produced by stem cells is administered
.
At the same time, the adeno-associated virus (AVV) gene therapy company AskBio (Asklepios BioPharmaceutical Inc.
), a wholly-owned subsidiary of Bayer, is also advancing a gene therapy project to provide advanced treatments for Parkinson's disease
.
Currently, this project is conducting patient recruitment and evaluation for a phase Ib clinical study
.

Wolfram Carius, Global Head of Cell and Gene Therapy at Bayer, said: “BlueRock and AskBio’s clinical candidate therapies have great potential for the treatment of Parkinson’s disease
.
For the first time, it is possible for us to prevent and reverse this degenerative disease, thereby truly helping those who There are patients with high unmet medical needs
.
Although the project is still in the early stages of clinical trials, we will firmly promote breakthrough scientific development and look forward to bringing significant improvements to the lives of patients who urgently need innovative drugs
.
"

Parkinson's disease is the most common neurodegenerative movement disorder, affecting more than 10 million people worldwide
.
It is caused by damage to nerve cells in the brain, resulting in lower levels of dopamine (a neurotransmitter involved in processes such as memory or exercise)
.
The disease usually begins as a tremor of one hand
.
Other symptoms include stiffness, cramps, and movement disorders (involuntary, unstable, writhing-like state of the face, arms, legs, or trunk)
.
For example, dopamine substitutes for levodopa are usually used to relieve the symptoms of the disease, but its effect will be weakened as the disease progresses, and there is currently no treatment that can improve the disease
.
By targeting the root causes of diseases, cell and gene therapy aims to achieve the goal of transcending symptomatic treatment
.

BlueRock uses real dopaminergic neurons to re-innervate the affected areas of the human brain and reverse the degeneration process, thereby realizing the possibility of restoring motor function
.
BlueRock's clinical trial will recruit ten patients at research centers in the United States and Canada
.
In this study, patients underwent surgery to transplant dopamine-producing cells into the putamen, which is the deep structure of the brain affected by Parkinson's disease
.
The main goal of the Phase I study (NCT04802733) is to evaluate the safety and tolerability of DA01 cells in the one-year period after transplantation
.
The secondary goals of the study are to evaluate the evidence of the survival and movement effects of transplanted cells in one and two years after transplantation, to evaluate the continued safety and tolerability of the two-year period, and to evaluate the feasibility of transplantation
.

AskBio's method consists of AAV, which delivers the human glial cell-derived neurotrophic factor (GDNF) gene to neurons in the putamen, allowing the brain regions affected by Parkinson's disease to express and secrete GDNF protein
.
Long-term experiments using AAV-GDNF have shown that in rodents and non-human primate models, the continuous expression of GDNF can promote the regeneration of midbrain neurons and a significant recovery of motor function
.
AskBio's clinical research is currently recruiting and evaluating patients for phase Ib clinical trials in the United States to evaluate safety and preliminary efficacy
.
Since its launch in August 2020, a total of ten patients have been recruited into the Phase Ib study (NCT04167540)
.

About Bayer

As a multinational company, Bayer has core competitiveness in health and agriculture in the field of life sciences
.
The company is committed to helping people overcome the major challenges brought about by the growing and aging global population through products and services, and benefiting the prosperity and development of mankind and the planet
.
Bayer is committed to promoting sustainable development and having a positive impact on its business
.
At the same time, the group also enhances profitability and creates value through technological innovation and business growth
.
Globally, the Bayer brand stands for credibility, reliability and quality
.
In fiscal year 2020, Bayer has approximately 100,000 employees and sales of 41.
4 billion euros
.
Excluding special projects, the investment in research and development is 4.
9 billion euros
.
For more information, please visit
.

About AskBio

After being acquired in 2020, AskBio (Asklepios BioPharmaceutical, Inc.
), a wholly-owned and independently operated subsidiary of Bayer, is a gene therapy company that fully integrates adeno-associated viruses, focusing on the development of drugs that have the potential to cure diseases and save lives
.
AskBio has a portfolio of clinical projects in the field of indications including neuromuscular, central nervous system, cardiovascular and metabolic diseases
.
The company's clinical product development lines include Pompe disease, Parkinson's disease and congestive heart failure, and authorization to develop treatments for clinical indications such as hemophilia and Duchenne muscular dystrophy
.
AskBio's gene therapy platform includes Pro10™, an industry-leading patented cell line production process, and an extensive adeno-associated virus (AAV) capsid and promoter library
.
AskBio's global headquarters is located in Research Triangle Park, North Carolina, USA, and its European headquarters is located in Edinburgh, UK
.
The company has successfully developed hundreds of patented third-generation AAV capsids and promoters, some of which have entered clinical trials
.
AskBio was founded in 2001.
As an early innovator in the field of gene therapy, AskBio has more than 500 patents in the fields of AAV production, chimeric and self-complementary capsids
.
For more information, please visit or follow the company's LinkedIn account
.

About BlueRock

BlueRock is an engineered cell therapy company whose mission is to develop regenerative drugs for intractable diseases
.
The company's "gene + cell" platform can simultaneously use pluripotent cell biology and gene editing technology to produce, manufacture and provide real cell therapy with engineered functions, thereby realizing a method: theoretically any cell in the human body All can be made, and any gene in the genome can be edited for therapeutic purposes
.
This platform has broad application prospects, but the company currently focuses on neurology, cardiology and immunology
.
In August 2019, the Bayer Group acquired the company with a total value of US$1 billion including advance payments and milestone payments
.
For BlueRock, this marks a new stage in its development, which aims to prove that degenerative diseases are reversible and bring its revolutionary new drugs to patients in urgent need
.
(Bayer official website)