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Bayer to advance two first-of-its-kind cell and gene therapies for Parkinson’s disease

Bayer AG announced today that BlueRock Therapeutics (BlueRock), a clinical stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, successfully administered the first dose of its pluripotent stem cell-derived dopaminergic neurons, named DA01, to a Parkinson’s disease patient in their open-label Phase 1 clinical study. In parallel a gene therapy program also targeted at providing advanced therapies for Parkinson’s disease is driven forward by Bayer’s wholly-owned clinical-stage adeno-associated virus (AAV) gene therapy company Asklepios BioPharmaceutical, Inc. (AskBio). This program is currently recruiting and evaluating patients in an ongoing Phase 1b clinical study.
“The potential of BlueRock and AskBio’s clinical candidates to treat Parkinson’s disease could be immense,” said Wolfram Carius, Head of Cell and Gene Therapy at Bayer. “For the first time, it might be possible to stop and reverse this degenerative disease and truly help patients with their high unmet medical need. The start of clinical trials represents the beginning towards a truly breakthrough treatment option to dramatically improve the lives of patients.”
Parkinson’s disease is the most common neurodegenerative movement disorder, impacting more than 10 million people worldwide. It is caused by nerve cell damage in the brain, leading to decreased levels of dopamine (a neurotransmitter involved in processes such as memory or movement). The disease often starts with a tremor in one hand. Other symptoms include rigidity, cramping and dyskinesias (involuntary, erratic, writhing movements of the face, arms, legs or trunk). Dopamine substitutes, such as levodopa, are commonly used to mitigate the symptoms of the disease, but their effect diminishes as the disease progresses and there is currently no disease-modifying treatment available. By targeting the disease at its root cause, cell and gene therapies aim to go beyond symptomatic treatments.
Using authentic dopaminergic neurons, BlueRock aims to re-innervate the affected regions of the human brain and reverse the degenerative process, potentially restoring motor function. BlueRock’s clinical trial will enroll ten patients at sites in the United States of America (US) and Canada. In this study, patients undergo surgical transplantation of the dopamine-producing cells into the putamen, a deep brain structure affected by Parkinson’s disease. The primary objective of the Phase 1 study (NCT04802733) 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 feasibility of transplantation.
AskBio’s approach consists of an AAV that delivers human glial cell line-derived neurotrophic factor (GDNF) gene to the neurons within the putamen, resulting in expression and secretion of GDNF protein in brain regions impacted by Parkinson’s disease. Long-term experiments using AAV-GDNF showed that sustained expression of GDNF can promote regeneration of midbrain neurons and significant motor recovery in rodents and non-human primate models. AskBio’s clinical study is currently recruiting and evaluating patients in Phase 1b in the US to assess safety and preliminary efficacy. A total of 10 patients have been enrolled since the start of the Phase 1b study (NCT04167540) in August 2020.