Bayer moves forward with cell and gene therapies for Parkinson's disease

Bayer announced today that its BlueRock Therapeutics subsidiary successfully administered the first dose of its Parkinson’s disease therapies.

Berlin-based Bayer said in a news release that BlueRock successfully administered the dose of its investigational pluripotent stem cell-derived dopaminergic neruons, called DA01, to a Parkinson’s disease patient in an open-label Phase 1 clinical safety and tolerability study.

Additionally, Bayer said its wholly-owned, clinical-stage adeno-associated virus (AAV) gene therapy company, Asklepios BioPharmaceutical (AskBio), is currently recruiting and evaluating patients in an ongoing Phase 1b clinical study to assess the safety and preliminary efficacy of a parallel gene therapy program for Parkinson’s disease.

BlueRock will enroll 10 patients across the U.S. and Canada. Those enrolled will undergo surgical transplantation of the dopamine-producing cells into the putamen deep brain structure, with a primary objective of assessing safety and tolerability at one year post-transplant and secondary objectives that include assessing the evidence of transplanted cell survival and motor effects at one and two years post-transplant. Additionally, the secondary endpoints include continued safety and tolerability at two years and the feasibility of transplantation.

AskBio’s AAV delivers human glial cell line-derived neurotrophic factor (GDNF) gene to the neurons within the putamen to provide expression and secretion of GDNF protein in brain regions impacted by Parkinson’s. The trial is currently recruiting and evaluating U.S. patients to assess safety and preliminary efficacy, with 10 patients enrolled so far since August 2020.

“The potential of BlueRock and AskBio’s clinical candidates to treat Parkinson’s disease and truly help patients with their high unmet medical need could be immense,” Bayer head of cell & gene therapy Wolfram Carius said in the release. “We are still in early phases of clinical trials, but passionately committed to advancing breakthrough science to improve the lives of patients who have been waiting for way too long for innovative treatments.”