What You Need to Know About BlueRock Therapeutics

BlueRock Therapeutics

BlueRock Therapeutics, which has locations in Toronto, New York City and Cambridge, Mass., rocketed to existence in December 2016 with $225 million in venture funding to focus on regenerative medicine, using induced pluripotent stem cell (iPSC) therapies.

Emile Nuwaysir, BlueRock’s president and chief executive officer, told BioSpace, “We’re targeting previously intractable degenerative diseases, like Parkinson’s and heart failure. There are hundreds of millions of patients worldwide that suffer from degenerative diseases like these where there is a known mechanism of action and that action is attributable to the loss of a specific cell. What we intend to do is to address the loss of cells by replacing the cell that was lost and by doing that, restore the native biologic function and, we expect, reversing the symptoms of the degenerative disease.”

There are many factors that differentiate BlueRock from other startups, including its financial backers, Bayer AG and Versant Ventures, but the science is a bit different than most stem-cell approaches. Most companies approach this field using embryonic stem cells, which requires destruction or manipulation of pre-implantation stage embryos, which have had issues with creating patient-matching embryonic stem cell lines, as well as ethical concerns over use of embryos. iPSC can be developed directly from adult tissues, can grow indefinitely, and are capable of growing into any cells in the body.

“We are addressing fundamental cell loss,” Nuwaysir says. “In a heart failure patient, the first step is a heart attack, a myocardial infarction, and within hours of that heart attack, the patient will lose a billion heart muscle cells. And after that, medicine is really not very good at dealing with this situation, no matter what drugs you use—small molecules, chemistry, large molecules, devices, intervention—the heart simply does not have the muscle to pump correctly. We’ve tried a lot of ways to make the patient experiences better, to try and reduce their pain, to reduce the symptoms of the disease, but almost nothing we do addresses the root cause of the disease, which is that cell loss. What we intend to do is remuscularize the heart and improve the heart muscle function.”

Company Leadership

Emile Nuwaysir – president and chief executive officer. Nuwaysir previously served as president and chief operating officer of Cellular Dynamics International, a Fujifilm Company. He was also president of Opsis Therapeutics, CDI’s ocular cell therapy subsidiary. Before CDI, Nuwaysir worked for NimbleGen Systems.

Robert Deans – chief technology officer. Prior to BlueRock, Deans was chief scientific officer of Rubius Therapeutics. Before Rubius, he was executive vice president at Athersys. He also served as vice president of research at Osiris Therapeutics.

Eric Soller – vice president, corporate development and strategy. Soller led the launch of BlueRock as an entrepreneur-in-residence at Versant Ventures. Prior to joining Versant in 2016, Soller was a junior partner in McKinsey & Company’s New York office.

Gordon Keller – scientific co-founder. Keller is director of the McEwen Centre for Regenerative Medicine at the University Health Network in Toronto. Prior to that, he was director of the Black Family Stem Cell Institute at the Mnt. Sinai School of Medicine in New York.

Michael Laflamme – founding investigator. Laflamme is a senior scientist at Toronto General Hospital Research Institute.

Lorenz Studer – scientific co-founder. Studer is director of the Center for Stem Cell Biology and member of the Developmental Biology Program at Memorial Sloan Kettering Cancer Center in New York.

Viviane Tabar – founding investigator. Tabar is Theresa Feng chair in neurosurgical oncology at Memorial Sloan Kettering Cancer Center in New York, and holds a joint appointment at Weill Cornell Medical College in the departments of Neurosurgery and Neuroscience.

Company Financing

In December 2016, the company launched with $225 million in Series A financing from Bayer AG and Versant Ventures.

"We are just like any other startup in one sense, in that we have investors and those investors are supporting us, but we have complete freedom to pursue our strategic vision just like any other startup," Nuwaysir says. “We are not, for example, partnered in any way with anybody. Having said that, we have two great, phenomenal investors. Versant Ventures, a top tier venture capital firm, in my opinion, one of the best in my space, cell and gene therapies. They’re a forward-thinking and accomplished group of people and I’m honored to have them both as financiers and advisors. The other financial investor is Bayer, one of the largest cardiovascular franchises in the world, one of the broadest, deepest organizations, they provide tremendously valuable strategic insight. Those two groups represent our financial backing, they are our board of directors, but we are an independent company, we go in the direction we like.”

Pipeline

BlueRock has at least six pipeline programs in neurodegenerative and cardiovascular disorders. The furthest along is in Parkinson’s disease. “We’ve publicly disclosed that our first program is in Parkinson’s disease and we expect to be in the clinic treating our first patients next year,” Nuwaysir says. “We’re excited about what we think we’ll show. We have a great partnership with Sloan Kettering and the Weill Medical Center, and have a series of other neurodegenerative indications we haven’t disclosed yet, but will in time. In cardiovascular, we’re broadly focused on heart failure. We haven’t announced an IND target date yet, but we know it’ll be after Parkinson’s. Again, our goal there is to remuscularize the heart. It’s well under way and we’re building GMP access and preparing for that kind of definitive preclinical-stage work.”

Market Competition

As mentioned earlier, quite a few companies are working with stem cells, but most work on adult or primary cells that have been isolated from fetal sources, cadavers or patients. That’s quite different from what BlueRock is doing. “What we do is use an iPSC as a manufacturing platform to manufacture the terminal cell that’s lost, that midbrain dopaminergic neuron, and put it back in the brain,” Nuwaysir says. “Within that category, there’s no other company like us that has a broad platform that’s addressing multiple therapeutic categories of medical need.”

He does mention Viacyte and Semma Therapeutics, but notes Viacyte is using embryonic stem cells. “They’re kind of in our space,” he says, “but not a competitor, more like an industry colleague, I would say. It’s a pretty small list. That’s why I think it’s so exciting, because what we’re doing really hasn’t been tested yet.”

Dollars and Deals

Another thing that’s unusual about BlueRock, quite unusual for a startup, is the number of partnerships already in place at launch. It has a strategic partnership with Memorial Sloan Kettering Cancer Center, a manufacturing platform with a partnership with Toronto-based CCRM, and the foundation of the company’s approach is iPSC intellectual property invented by Shinya Yamanaka of Kyoto University in Japan and licensed from iPS Academia Japan, which manages iPSC IP. Additional programs are also being advanced in collaboration with the Toronto-based McEwen Centre for Regenerative Medicine and University Health Network and the Weill Cornell Medical Center.

“I’m just a tremendously lucky guy to have walked into this,” Nuwaysir says. “I was hired after the founding and a lot of these deals were part of the founding of BlueRock. We’re extremely well-positioned, uncommonly well-positioned, for success, in part by Versant and Bayer, but in Toronto with the Regeneration Medicine Network, all these world-class organizations, people like Gordon Keller and Mike LaFlamme, leading researchers in stem cell and translational biology, and in New York and Sloan Kettering, with Lorenz Studer and Viviane Tabar. We have access to facilities and people and know-how and it’s really sped us up the curve very quickly.”

What to Expect

The company plans to have its Parkinson’s program into the clinic in 2018, so in 2019 it will hopefully have good safety data, with a final readout in 2020. “I think we’ll show in that trial that we’ve reversed the symptoms of degenerative disease by restoring the cells that were lost to degeneration,” Nuwaysir says. “That’s not a small moment for science; that’s not a small moment for medicine. You start asking very big questions where that leads us, what we can do with the ability to repair the body when it can’t repair itself. In five years, it’ll be common understanding that this is possible, that you cannot only do it in a disease as complex as Parkinson’s and heart failure, that it opens the whole industry to the possibility of restoring function to these diseases, and then to things like rebuilding organs, which becomes possible.”

Nuwaysir expects 2018 will be a big year for BlueRock as it moves into the clinic. “2018 will be a transformational year for BlueRock as we advance our first pluripotent stem cell therapeutic into the clinic for Parkinson’s disease. Our recognition as BioSpace’s top of the 2018 class is a tremendous honor that underscores the joint effort between our fantastic team, collaborators and scientific partners to develop therapeutics that are designed to restore the body’s native function in degenerative diseases,” Nuwaysir says.

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