Cells, Made to Order: BlueRock and the Stem Cell Therapy Revolution

Few realms of biotechnology promise as much as gene and cell therapy. These fields aim to edit cells to correct disease states and to replace cells killed by degenerative diseases, directly reversing some of the most debilitating and seemingly untreatable conditions humans can have. These promises have been difficult to realize, but technology is reaching the point where some of them are entering clinical use. Firms like BlueRock Therapeutics are at the forefront of this new arena of science.

BlueRock’s proposition is simple, as Ainslie Little, their Senior Vice President of Corporate Strategy, presents it: they want to restore health to people whose tissues are degenerating due to genetic disease. To do this, they establish pluripotent stem cell lines, edit their genomes to remove disease-causing mutations, and use those stem cells to rebuild healthy version of the degenerating tissue. When this kind of stem cell therapy works as desired, it physically replaces the dead, damaged, or dying cells that cause the disease with new, healthy cells that do not contain the disease-causing gene variants, which both reverses the disease’s existing effects and cures it going forward. BlueRock’s approach is already showing clinical success, with early trials of bemdaneprocel (BRT-DA01), a cell therapy for Parkinson’s disease, looking positive enough to warrant additional, larger trials. BlueRock’s ambitions are far grander, and Little offers a list of ongoing projects: “We also have programs directed to treatment of lysosomal storage disorders and demyelination diseases…we have a cardiac program where we generate cardiomyocyte cells from pluripotent stem cells in an effort to create a therapeutic that would replace the cells lost in patients with heart failure.” This shows the flexibility of what BlueRock Therapeutics has managed to create: in theory, it amounts to a radical extension of the human body’s natural healing process, able to regenerate tissues lost to virtually any event or condition.

One other item shows the extent to which BlueRock’s efforts could be developed: “We also have a couple of programs in our pipeline that target diseases that are not degenerative diseases per se, but diseases where we think delivery of a cell or use of a cell as a delivery vehicle for a payload, for instance, might be advantageous over other more traditional modalities of therapeutic treatment.”

BlueRock achieved its success, and builds its ambitions on, a foundation of technologies cleverly developed in-house or licensed from their current owners. In particular, their proprietary system for creating pluripotent stem cells from ordinary cells and maintaining the resulting cell lines is the core of their business plan. Licensing use of CRISPR-Cas9 gene editing technology from Editas Medicine enabled them to form their current core model: creating healthy versions of diseased cells to introduce into patients whose cells are sick.

Ainslie Little’s path to BlueRock Therapeutics was as winding as BlueRock’s medical advancements are revolutionary, and is explored in our podcast. Listen to this interview and other stories of biotechnology pioneers changing the world at our landing page.