- Area
- Neurology
- Clinical Indication
- Parkinson's disease bemdaneprocel (BRT-DA01)
- Cell Type
- Dopaminergic neuron progenitor
Program overview
Cell replacement therapy
BlueRock Therapeutics is developing innovative cell replacement therapies for Parkinson’s disease (PD) and other neurological disorders. Leveraging patented pluripotent stem cell (PSC) technology, BlueRock’s neurology program aims to reshape the way these disorders are managed.
The most advanced investigational candidate is bemdaneprocel, a PSC-derived dopaminergic neuron progenitor cell replacement therapy currently being developed for the treatment of people with Parkinson’s disease.
Parkinson's disease and cell replacement
Disease burden
Parkinson's disease leads to motor and non-motor symptoms that substantially affect quality of life for people with Parkinson's disease and their care partners.
Pathophysiology
The loss of dopaminergic neurons in the substantia nigra pars compacta (SNpc) is a key driver in the pathophysiology of Parkinson's disease, leading to striatal dopamine depletion and progressive multisystem involvement.
Unmet need
There is a need for therapies that address the loss of dopaminergic neurons and maintain efficacy as Parkinson's disease progresses.
Mechanism of action
Bemdaneprocel comprises allogeneic, PSC-derived dopaminergic neuron progenitor cells designed to engraft and replace the lost dopaminergic inputs in the posterior putamen.
Neurology pipeline
- Area
- Neurology
- Clinical Indication
- Parkinson's disease (BR-DA02)
- Cell Type
- Dopaminergic neuron progenitor
- Area
- Neurology
- Clinical Indication
- Neurodegenerative diseases
- Cell Type
- Microglia
- Area
- Neurology
- Clinical Indication
- Genetic or acquired myelin defects
- Cell Type
- Oligodendrocyte
Development status
Clinical development
Neurology | Phase 1
Long-term follow-up
exPDite | Parkinson's disease
exPDite was a first-in-human, multisite, open-label Phase 1 trial to assess the safety and tolerability of bemdaneprocel in people with Parkinson's disease. The trial met its primary objective of safety and tolerability at 12 months post transplantation. Secondary and exploratory efficacy assessments showed improvement or stability in motor function through 3 years post transplantation. Because this small, open-label Phase 1 trial was not designed or powered to establish efficacy, these findings remain exploratory. At 18 months, 18F-DOPA PET imaging suggested that the transplanted cells continued to survive and engraft in the brain after discontinuation of immunosuppression. Participants remain engaged in long-term follow-up.
Trial IDs:
Neurology | Phase 3
Active, recruiting
exPDite-2 | Parkinson's disease
BlueRock Therapeutics is enrolling patients in a Phase 3, multicenter, randomized, sham surgery-controlled, double-blind trial to assess the efficacy and safety of bemdaneprocel in approximately 102 adults with Parkinson's disease.
Trial IDs: