German pharma company Bayer has announced that it has made progress in its ‘two-pronged’ approach to deliver cell and gene therapy candidates for the treatment of Parkinson’s disease.
BlueRock Therapeutics, a wholly-owned subsidiary of Bayer, has administered the first dose of DA01 – stem cell-derived dopaminergic neurons – to a Parkinson’s disease patient in a Phase I clinical study.
This trial will enrol ten patients at sites in the US and Canada, with patients involved set to undergo surgical transplantation of the dopamine-producing cells into the putamen brain structure.
It will primarily aim to assess the safety and tolerability of DA01 cell transplantation one-year post-transplant, while secondary objectives will assess the evidence of transplanted cell survival and motor effects at the same timepoint.
Another wholly-owned subsidiary of Bayer, Asklepios Biopharmaceutical (AskBio) has also begun recruiting and evaluating patients in an ongoing Phase Ib study for a gene therapy targeted for the treatment of Parkinson’s.
Meanwhile, AskBio’s study will aim to assess the safety and preliminary efficacy of its approach, which consists of an adeno-associated virus (AAV) that delivers human glial cell line-derived neurotrophic factor (GDNF) gene to the neurons in the putamen.
“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,” he added.