Our cell+gene platform creates a fundamental new way to impact disease
Through the convergence of state-of-the-art technologies in cell biology and gene editing, we have created a fundamentally new way to treat a broad range of diseases that overcomes the limitations of current methods that rely on harvested primary cells to develop therapeutics.
What is unique about the BlueRock cell+gene platform?
We start with healthy donor cells that we then engineer to create a truly universal source for cell therapy. These universal pluripotent stem cells (PSCs) form the basis of our platform, which has two distinct advantages over others:
First, using our proprietary bioprocessing and manufacturing techniques, we create stable master cell banks capable of virtually unlimited expansion and differentiation into any cell type in the body.
Second, we can then direct the differentiation of these universal cells into almost any cell type in the human body, recapitulating natural developmental processes. Through exclusive licenses from our scientific founders and other collaborators, we have access to the world’s leading methods for the creation of authentic cell types in the areas of neurology, cardiology, and immunology. BlueRock is in the process of developing these advances to create clinical-grade, authentic cell therapy product candidates with high potency, purity, and specificity at commercial scale.
In addition, our advanced gene editing capabilities further allow us to engineer these authentic cells to enable new therapeutic modalities. These engineered cell therapies have disruptive advantages, being able to locally deliver a high regional concentration of payload with no systemic toxicity. This avoids many of the challenges associated with systemic, non-targeted delivery, and cells can be engineered to perform a broad range of functionalities including protein production, antibody or cytokine secretion, or local enzyme replacement.
BlueRock’s founders have pioneered the derivation of dopaminergic neurons that have increased dopamine release as they mature. Our lead program is in Parkinson’s disease, where we have established robust pre-clinical proof of concept data to restore motor function. Here, we aim to reinnervate the human brain and reverse degenerative disease, while we explore additional applications of our best-in-class capabilities across other neurological conditions.
BlueRock’s founders have mastered the creation of PSC-derived cardiovascular cell subtypes with potential utility in treating degenerative heart disease. Having successfully derived ventricular cardiomyocytes, initial preclinical proof of concept has been established.
Our universal macrophage platform enables multiple modalities to treat autoimmune disorders. To date, we have successfully generated PSC-derived macrophages that functionally respond to external stimuli similar to adult-derived macrophages. These and other immune regulatory cells can be further engineered to be immuno-suppressive or to deliver a range of biotherapeutic payloads.