Bayer advances cell and gene therapies in Parkinson’s

Bayer announced progress of two potential therapies against Parkinson’s disease — a cell therapy being developed with BlueRock Therapeutics and a gene therapy being developed with AskBio.

A first participant received randomized treatment in the pivotal phase 3 clinical trial, exPDite-2, of bemdaneprocel, an investigational allogeneic pluripotent stem cell-derived therapy for PD. Bemdaneprocel is designed to replace the dopamine producing neurons that are lost in Parkinson’s disease. The dopaminergic neuron precursors, derived from human embryonic pluripotent stem cells, continue developing into mature dopamine neurons after implantation. In a surgical procedure, the neuron precursors are implanted into the brain of a person with Parkinson’s disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson’s disease and to potentially restore motor and non-motor function to the patient.

At the same time, Bayer announced that the first European participants have been randomized in REGENERATE-PD, a phase 2 clinical trial of AB-1005, an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2). The therapy contains the human glial cell line-derived neurotrophic factor (GDNF) transgene which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery. Recombinant GDNF has long been evaluated as a potential treatment for diseases, such as PD, marked by progressive degeneration of midbrain dopaminergic neurons.

Both therapies are focused on treating moderate-stage PD.