BlueRock Therapeutics Secures $225 Million to Advance iPSC-Based Therapies

On December 12, BlueRock Therapeutics, a company dedicated to developing induced pluripotent stem cell (iPSC) therapies, secured $225 million in funding—a rare feat of such magnitude. This round of financing was jointly provided by Bayer and Versant Ventures, underscoring their strong confidence in the potential of this stem cell technology to cure diseases. Currently, BlueRock’s primary focus is on treating patients with myocardial infarction or chronic heart failure by helping them regenerate cardiomyocytes. The $225 million investment is sufficient to cover BlueRock’s R&D expenses for the next four years. Versant Ventures revealed that BlueRock’s clinical trials are scheduled to begin in 2018.

BlueRock Therapeutics is known as the “next-generation regenerative medicine company,” with its competitive edge stemming from exclusive access to Professor Shinya Yamanaka’s patented induced pluripotent stem cell (iPSC) technology. This technology was initially developed in 2006 by Japanese scientist Shinya Yamanaka, who introduced a combination of four transcription factors (Oct4, Sox2, Klf4, and c-Myc) into differentiated somatic cells using viral vectors, thereby reprogramming them into a cell type resembling embryonic stem cells and embryonic pluripotent stem cells. Professor Yamanaka was awarded the Nobel Prize in Physiology or Medicine in 2012 for this breakthrough.

Subsequently, scientists around the world successively discovered other methods capable of generating these cells. On March 10, 2016, a research team led by Professor Koji Nishida of Ophthalmology at Osaka University in Japan successfully cultured cells from major ocular structures—including parts of the cornea, lens, and retina—from induced pluripotent stem cells (iPS cells) for the first time worldwide. These findings were published in the online edition of the British scientific journal Nature on the 9th of this month. Earlier this year, the world’s leading journals Nature and Cell each published articles commemorating the first decade since the invention of iPSC technology.

Unlike technologies such as oogonial stem cells, this groundbreaking innovation directly reprograms a person’s skin cells, blood cells, or other somatic cells into induced pluripotent stem cells (iPSCs), which can then differentiate into stem cells, neurons, or any other cell types required for regeneration. This approach not only mitigates the risk of immune rejection but also circumvents ethical controversies.

What Is the Potential of iPSC Technology in Disease Treatment? In 2008, George Daley’s laboratory at Harvard University used induced cell reprogramming technology to convert skin cells from patients with ten different genetic disorders into induced pluripotent stem (iPS) cells. These cells will play a significant role in establishing disease models and drug screening.

In 2009, Chinese scientists successfully generated mice from induced pluripotent stem (iPS) cells in November 2008. The collaborative work led by Researcher Zhou Qi from the Institute of Zoology, Chinese Academy of Sciences, and Researcher Zeng Fanyi from Shanghai Jiao Tong University School of Medicine demonstrated that viable, fertile mice could be produced using iPS cells, thereby providing the first proof worldwide that iPS cells possess pluripotency similar to that of embryonic stem cells. The findings suggest that iPS cells may serve as a potential source for treating various diseases, akin to embryonic stem cells.

In clinical-grade trials, only one case involving iPSCs was conducted in Japan in 2014 over a ten-year period, successfully restoring vision to a patient with macular degeneration. However, the trial was unfortunately halted in 2015 due to two minor genetic mutations.

This project is led by the team’s two founders: Dr. Gordon Keller, a leading figure in the field of stem cells, and Dr. Michael A. Laflamme, a pioneer in cardiac cell therapy. Additionally, they have recruited Dr. Lorenz Studer from Memorial Sloan Kettering Cancer Center as their scientific co-founder.

Dr. Studer is also a leading figure in the field; he was awarded the 2015 MacArthur “Genius Grant.” He published an article in Nature demonstrating that induced pluripotent stem cells could be differentiated into dopamine-producing neurons, which successfully cured Parkinson’s disease in mice. The other corresponding author of this study, Dr. Viviane Tabar, is also a scientific co-founder of BlueRock Therapeutics.

R&D activities will be conducted in Toronto, New York, and Boston. The company has also partnered with CCRM in Boston, which boasts extensive experience in the development and commercialization of regenerative medicine technologies, providing significant support to BlueRock’s R&D efforts and future market launch. BlueRock maintains extensive collaborative partnerships in the United States, Canada, and Japan.

Dr. Axel Bouchon of the Bayer Life Science Center (BLSC) commented on the collaboration, stating, “Research in the field of cell therapy is a strategic component of Bayer’s portfolio. While induced pluripotent stem cell (iPSC) technology holds the potential to successfully address some of the most challenging diseases, numerous obstacles must still be overcome before its full clinical application can be realized. Nevertheless, by leveraging the world’s top talent and resources, I am confident that we can achieve our ultimate goal of curing diseases.”