BlueRock - Cell+Gene Platform
With our cell+gene platform, BlueRock is aiming to redefine the very way we treat disease, moving from addressing mere symptoms to restoring health lost to cell damage or degeneration. Instead of prescribing a pill to treat tremors in patients with Parkinson’s, we might restore the chemical messengers that were destroyed, addressing tremors at the source. Those who have suffered a heart attack may be able to have their damaged heart cells replaced rather than be burdened with constant medications that cause debilitating side effects or serve only to address limited symptoms. And, further, we have the potential to engineer immune cells that could deliver unique therapeutic payloads to treat autoimmunity and cancer.
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Neurology
Lead focus: Parkinson’s disease – The current standard of care for Parkinson’s disease has remained unchanged for more than 60 years. We now know that dopaminergic neurons are lost in Parkinson’s disease-mediated neurodegeneration. With our cell+gene platform we can differentiate our iPSCs into authentic dopamine neurons possessing the same properties of the neurons that were previously lost. These cells have the potential to reform the neural networks that were destroyed, restoring motor function and improving quality of life.
In collaboration with Memorial Sloan Kettering Cancer Center, we have received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) and permission from Health Canada to proceed with a Phase 1 study in patients with advanced Parkinson's disease. This is the first trial in the United States and Canada to study pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson's disease.
Cardiology
After a cardiac event, a person can lose billions of heart cells within minutes. Once lost, those cells are gone for good. As a result, many heart attack survivors must take medications for the rest of their lives to increase life expectency and manage their symptoms, which can include pain, shortness of breath and fatigue. Using our cell+gene platform, BlueRock creates authentic cardiac muscle cells with the aim to replace lost cells and restore cardiac function to help people live longer, more fulfilling lives.
Immunology
Lost T cell and/or macrophage function plays a central role in many immune-related diseases. BlueRock is a leader in differentiating pluripotent stem cells into T cells and macrophages for immunological applications. BlueRock’s authentic immune cells have the potential to carry out an array of functions within the body, such as delivering a cytokine payload or rallying other components of the immune response. By turning on the process that nature has provided for fighting disease, our cell+gene platform has the potential to address a variety of devastating diseases, including cancer, in a new way.
Ophthalmology
Millions of patients around the world today are suffering from permanent vision loss as a result of incurable ocular diseases. In order to address the unmet need of a treatment for blinding disorders of the retina, BlueRock entered into a strategic R&D alliance with FUJIFILM Cellular Dynamics and Opsis Therapeutics to discover and develop off-the-shelf iPSC therapies for ocular diseases. The alliance brings together the necessary technologies and expertise to develop cell replacement therapies for both age-related macular degeneration (AMD) and inherited retinal diseases (IRDs).
What is cell+gene?
BlueRock has developed an exquisite and powerful platform, cell+gene, that reprograms mature, differentiated cells back to iPSC’s and applies advanced engineering to enhance the cells’ therapeutic capabilities. We then use advanced bioprocesses to re-differentiate these iPSCs into new, authentic replacements of cells damaged or lost to disease.
- Cell: highly specific authentic cells, manufactured from iPSCs under rigorous process control
- Gene: genetic engineering to enhance those authentic cells with specific functions to improve their therapeutic effect
The BlueRock Difference – Authentic Cells
BlueRock has a superior understanding and know-how of the intricate requirements needed to differentiate pluripotent cells into specific cells in the body – neuronal cells, cardiac cells, immune cells and more. Using our proprietary bioprocess and manufacturing techniques, we create stable master cell banks capable of virtually unlimited expansion and differentiation into any cell type.
Our advanced gene editing capabilities further allow us to engineer iPSCs into authentic cells to enable new therapeutic modalities. BlueRock scientists can program those replacement cells to carry out specific functions, whether that be delivering lost signals in the brain, pumping blood in the heart, or activating a therapeutic immune response.
Our expertise in producing authentic cells opens the door to creating medicines that raise the standard of care from outdated “band-aid” fixes to replacing cells that have been lost, and engineering them to work seamlessly with the surrounding healthy cells to restore function.
Key Advantages
BlueRock’s expertise in building master cell banks creates endless possibilities for what diseases we can treat – we just need to know the precise recipe to make the cell type we desire. We can grow these cells to high numbers and freeze them down so that they’re readily available when needed and can be quickly scaled to meet industrial needs.
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