Exhilarating possibilities in transformative health care

One of BlueRock Therapeutics' main claims to fame in the biotech world today is the size of the funding it launched with last year: US$225 million – one of the largest series A financings in biotech history. But the company's founders expect it will soon be known for much more than that. Their vision is to transform medicine and health care by harnessing the power of the cell to repair the human body.

If BlueRock's regenerative medicine platform evolves as planned, the resulting therapeutics will be able to restore natural biological functions that have been lost to degenerative diseases, such as Parkinson's or heart disease – the two conditions they plan to target first. The anticipated induced pluripotent stem cell (iPSC) therapies will work by replacing neurons in the brains of Parkinson's patients and heart muscle cells in heart attack survivors.

Bayer AG and Versant Ventures founded BlueRock in December 2016 and together contributed the US$225 million to support the new firm's platform and pipeline. This amount is expected to give the company at least four years of runway to advance several programs without having to contend with the usual treadmill of venture capital financing.

The power of super cells

In embryos, stem cells begin undifferentiated (that is, unprogrammed) and eventually grow into specialized cells – such as bone marrow cells or nerve cells. At the embryonic stage, when they still have the potential to become many different things, they're known as pluripotent. Once specialized, they can no longer become another type of tissue.

Adult cells are already specialized. But recent breakthroughs have resulted in the discovery of iPSCs: adult stem cells that have been “induced” to revert to a pluripotent state. These cells hold great promise in treating degenerative and other diseases.

The right time

The timing is right for BlueRock, says CEO Emile Nuwaysir, for several reasons. One is that iPSC technology has recently matured to a point where it's possible to specify what cell you want a pluripotent stem cell to become – for example, a cardiomyocyte (cardiac muscle cell) or a midbrain dopamine-producing neuron. The upshot is it's now possible to make a therapeutic product from a stem cell.

Secondly, the regulatory picture is rapidly coming into focus. "The FDA is working on an act and Congress is working on a new framework. There are also more advanced regulatory frameworks in other parts of the world," says Nuwaysir. "It's becoming more obvious how these products may be regulated and how they should be developed."

The third factor is the money—not just the sizable cash infusion that BlueRock launched with, but growing investor interest in stem cell technologies broadly.

The confluence of these factors led Bayer and Versant to conclude that the time to launch BlueRock was now.

Emile Nuwaysir, CEO, BlueRock

… And the right place

While BlueRock's head office is in Boston, the bulk of its scientific activity will be in Toronto, says Nuwaysir – for several significant reasons.

"At our root, we are an iPSC company," he says, "and Toronto happens to be one of the world's leading centres for expertise in stem cells."

From the discovery of insulin in the 1920s to the reinvention of pharma when Genentech commercialized the first recombinant insulin in the late 1970s, Toronto has long been a hub for medical discovery, Nuwaysir notes. The city continued to blaze trails throughout the 1990s and 2000s, when it became one of the most productive places in the world in stem cell research.

"The connection between this basic research and the transformative medicine that BlueRock envisions is real."

Drawing on Ontario's medtech ecosystem

On top of that, notes BlueRock CTO Robert Dean, Toronto offers a web of supportive organizations, from world-class universities to the Centre for Commercialization of Regenerative Medicine (CCRM) and the McEwen Centre for Regenerative Medicine at the University Health Network (UHN). BlueRock recently formalized a manufacturing partnership with CCRM and a research collaboration with the McEwen Centre. CCRM has the capacity to produce a variety of stem cell types for therapeutics in the considerable quantities needed for major clinical trials, and will support BlueRock's manufacturing platform.

Toronto has other assets as well, says Nuwaysir, including an "incredible talent pool, a very livable city and the opportunity to partner with world-class leaders" – such as Dr. Gordon Keller, Director of the McEwen Centre and a world leader in stem cell biology, and Michael A. Laflamme, a cardiac cell therapy pioneer and Senior Scientist at UHN.

On a practical level, Nuwaysir and Dean appreciate how easy it is to travel in and out of Toronto, given the fact that they often need to be in Boston. "Linking with the big financial centres on the East Coast is easy from here," says Dean. "I haven't experienced any issues in synchronizing or keeping our teams up to date."

Asking the big questions

BlueRock logo

BlueRock is scheduled to begin clinical trials as early as 2018, but Nuwaysir isn't speculating on when treatments may be commercialized. "What we're trying to do here is not incremental," he emphasizes. "We think these are transformative therapies."

BlueRock's main challenge is hiring, says Nuwaysir – a job made easier by the tremendous talent pool in southern Ontario, yet still complicated by the need to choose carefully. "We're getting great responses, but it takes a lot of effort to make sure you pick the right people and build the right organization."

Toronto's appeal may help attract the big guns Nuwaysir and Deans are looking for. In fact, BlueRock recently succeeded in recruiting a new Senior Vice-President of product development: Dr. Michael Scott, who was successfully enticed to return to Canada after a distinguished career in biotechnology in the U.S.

But perhaps the most exciting news of all is the company's recent decision to expand its presence in Toronto with a 10,000 sq. ft., state-of-the-art R&D and manufacturing facility in the MaRS Discovery District just steps away from the CCRM. The BlueRock research team is expected to move into the space later in 2017.

As for what happens next – the possibilities are exhilarating to contemplate.

"You start asking very big questions," says Nuwaysir. "You start thinking about what the implications are if you can reverse degenerative disease, and where we'll go from here. We think this is the foundation of a new branch of medicine – and think the world will see it that way too, when we prove it's possible to reverse the motor control symptoms of Parkinson's. It's going to be an incredibly exciting journey."

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