Bayer Advances First-in-Class Cell and Gene Therapies for Parkinson’s Disease into Clinical Trials

On June 8, 2021, Bayer announced that its subsidiary BlueRock Therapeutics (BlueRock) successfully transplanted DA01, a dopaminergic neuron therapy derived from pluripotent stem cells, into a patient with Parkinson’s disease for the first time in a Phase I clinical trial. Meanwhile, its subsidiary Asklepios Biopharmaceuticals (AskBio) is also advancing a gene therapy for Parkinson’s disease and is currently recruiting and evaluating patients in an ongoing Phase Ib clinical study.

Parkinson’s disease is the most common neurodegenerative movement disorder, affecting over 10 million people worldwide. It is caused by damage to nerve cells in the brain, leading to a decline in dopamine levels—a neurotransmitter involved in processes such as memory and movement. The disease typically begins with a tremor in one hand. Other symptoms include muscle rigidity, spasms, and dyskinesia (involuntary twisting movements of the face, arms, legs, or trunk). Dopamine replacement therapies, such as levodopa, are commonly used to alleviate symptoms; however, their efficacy diminishes as the disease progresses. By targeting the underlying cause of the disease, cell and gene therapies aim to go beyond purely symptomatic treatment paradigms.

Leveraging authentic dopaminergic neurons, BlueRock Therapeutics aims to reinnervate the affected regions of the human brain, thereby reversing the degenerative process and potentially restoring motor function in patients. BlueRock’s clinical trial will enroll 10 patients in the United States and Canada. In this study, patients will undergo surgery to transplant dopamine-producing cells into the putamen, a deep brain structure affected by Parkinson’s disease. The primary objective of this Phase I clinical trial is to evaluate the safety and tolerability of DA01 cell transplantation at 1 year post-transplantation. Secondary objectives include assessing the survival of the transplanted cells and their impact on motor function at 1 and 2 years post-transplantation, as well as the long-term safety and tolerability of the therapy.

AskBio's gene therapy utilizes an AAV vector to deliver the gene encoding human glial cell line-derived neurotrophic factor (GDNF) to neurons in the putamen, resulting in the expression and secretion of GDNF protein in brain regions affected by Parkinson's disease. Long-term studies using AAV-GDNF in rodent and non-human primate models have demonstrated that sustained GDNF expression can promote neuronal regeneration and significant motor recovery. AskBio's Phase 1b clinical trial is currently enrolling and evaluating patients in the United States to assess its safety and preliminary efficacy.

Note: The original text has been abridged.

References:

[1] Bayer to advance two first-of-its-kind cell and gene therapies for Parkinson’s disease. Retrieved June 8, 2021, from https://media.bayer.de/baynews/baynews.nsf/id/Bayer-to-advance-two-first-of-its-kind-cell-and-gene-therapies-for-Parkinsons-disease

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