The US Food and Drug Administration (FDA) has granted BlueRock Therapeutics a fast track designation for its cell therapy candidate DA01 for advanced Parkinsons disease.

BlueRock a Bayer subsidiary is currently evaluating the pluripotent stem cell-derived dopaminergic neuron therapy in a phase 1 study.

This early-stage trial is set to enrol ten patients across the US and Canada, with its primary objective to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant.

As a secondary objective, BlueRock will assess the evidence of transplanted cell survival and motoreffects at one- and two-years post-transplant and evaluate the continued safety and tolerability at two years, as well as the feasibility of transplantation.

Receiving fast track designation from the FDA is an important step, which will help us further accelerate clinical development of our DA01 cell therapy approach for Parkinsons disease, said Joachim Fruebis, chief development officer of BlueRock.

This is another critical step in the BlueRock mission to create authentic cellular medicines to reverse devastating diseases, with the vision of improving the human condition, he added.

In 2019, Bayer bought out its private equity partner Versant Ventures and founders in BlueRock Therapeutics for $240m, three years after setting up the company.

The decision gave Bayer complete control of BlueRocks cell therapy pipeline, headed by DA01 and spanning various diseases in the neurology, cardiology and immunology categories.

BlueRock was set up in late 2016 with $225m in start-up funding from Bayer and investment firm Versant, shortly after Bayer backed gene-editing specialist Casebia via its Leaps by Bayer investment arm.

BlueRocks induced pluripotent stem cells (iPSCs) platform is designed to encourage PSCs to differentiate into the dopaminergic neurons that are progressively destroyed in Parkinsons disease.

The hope is that introducing these neurons into areas of the brain where neurons are depleted will lead to increased dopamine release, restoring motor function.

The company is also developing iPSCs that differentiate into microglia, oligodendrocytes and eneic neurons for neurology applications, cardiomyocytes for heart failure, as well as macrophages and T regulatory cells for immunology applications including immune tolerance, fibrosis and graft-versus-host disease (GvHD).

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Bayer's BlueRock Therapeutics gains FDA fast track for Parkinson's disease cell therapy - PMLiVE