The Food and Drug Administration (FDA) has granted Fast Track designation to DA01 for the treatment of advanced Parkinson disease.
DA01 is an investigational pluripotent stem cell-derived dopaminergic neuron therapy designed to replace the midbrian dopaminergic neurons lost in Parkinson disease using the Company’s cell+gene platform. By replacing these neurons, DA01 may rebuild the neural circuit and restore motor control, thereby potentially reversing the disease.
The Company is currently investigating DA01 in a phase 1 study (ClinicalTrials.gov Identifier: NCT04802733). Patients (N=10) will receive DA01 through surgical transplantation into the putamen of the brain under general anesthesia. The primary outcome measure is an assessment of safety and tolerability at 1 year post-transplant. Key secondary endpoints include evidence of cell survival and effect on Parkinson disease symptoms.
The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to therapies that are expected to have an impact on factors such as survival and daily functioning.
BlueRock Therapeutics receives FDA Fast Track designation for DA01 in the treatment of advanced Parkinson’s disease. News release. BlueRock Therapeutics. Accessed July 19, 2021. https://www.prnewswire.com/news-releases/bluerock-therapeutics-receives-fda-fast-track-designation-for-da01-in-the-treatment-of-advanced-parkinsons-disease-301336295.html.