Dr Eric Soller, Vice President of Corporate Development & Strategy, BlueRock Therapeutics
BlueRock Therapeutics was born from a fascinating collaborative model - and a particularly exciting one for the cell & gene therapy space, given the stature of the various partners involved. Can you just sketch out for us who the key stakeholders are, and how the collaboration initially came about?
BlueRock Therapeutics was founded in 2016 through a $225 million Series A investment from Bayer and Versant Ventures. We are very fortunate to have founding investors that saw the significant unmet medical need of patients with diseases of cell degeneration and wanted to ensure BlueRock has adequate resources to pursue our bold vision – to harness the power of the human cell to create cell therapies that repair the body when it cannot repair itself.
Versant had been exploring cell therapy as an investment theme for many years and realized it was the right time to found a future category leader given the confluence of a number of important recent advances across the value chain which we think will allow for the creation of well-defined cell products with regulatory & marketing success. The collaboration came together on the heels of a prior co-investment between CRISPR Therapeutics (a Versant company) and Bayer in a joint venture in gene editing called Casebia. Both groups realized they also shared a vision to create the premier regenerative medicine therapy company focused on cell therapy. At Versant we did the heavy lifting and utilized our global connections to identify what we consider some of the best scientific founders and programs in the world, and began building the company from the ground up with additional support from our Bayer colleagues. Critical to its formation was a large investment that would provide years of runway, so that BlueRock could focus on building a transformative company and technology platform, without the distraction of having to go out and raise money. The two firms brought all the elements together to form BlueRock, which is an independent biotech that is free to operate and forge its own strategic relationships.
BlueRock’s launch was also good news for a stem cell biotech field which has comparatively struggled for investment dollars and airtime over the past couple of years – so why iPSCs and why now?
We think that BlueRock is unlike any other company that has ever worked in the space and what we are doing has never been done before. We believe many of the earlier setbacks in the stem cell field can be attributed to a lack of understanding of the disease biology, and limitations in either technology or a lack of mastery of cell differentiation into authentic, well-defined cell types that can replace the lost function. That said, much has been learned and the field would not be where it is today without prior ground-breaking efforts.
BlueRock is building an industry-leading induced pluripotent stem cell (or iPSC) platform, incorporating the latest advances in cell technology. We have unique expertise in controlling differentiation of those pluripotent cells into specific cell types, and we have a deep understanding of our target disease-state biology and relevant translational models to demonstrate their efficacy. The cells we produce are the authentic cells needed to replace the cells that are dead, dying or defective in patients. For example, for patients with Parkinson’s disease, we can create authentic dopaminergic neurons and for congestive heart failure patients we can replace ventricular cardiomyocytes that were lost.
We saw the investment opportunity in iPSCs as being particularly ripe for a number of reasons, namely the enhanced control of your starting material that it provides over other approaches. While the technology is still relatively young, there has been an explosion in the science and the tech has stabilized significantly in recent years.
Can you tell us some more about the company’s priorities and goals for the 12 months ahead?
We are initially focused on cardiovascular and CNS conditions, exploring the potential of our cell therapy in patients with Parkinson’s disease, as well as patients with chronic heart failure. We anticipate testing our Parkinson’s disease product candidate in patients next year.
In addition to our R&D efforts, we are focused on building out our global presence and attracting top-talent to join our growing team. By the end of 2018 we plan to have a total workforce of 70-80 full time employees across New York, Toronto, and the Boston/Cambridge area.
Putting on your investor hat for a moment, what trends can the cell & gene therapy sector expect to see in investment terms over the coming year? Are some biotech’s current concerns around investor confidence just a reflection of the general investment climate, or the result of more specific concerns relating to cell & gene therapy?
Cell and gene therapies that could successfully replace or restore lost function are being actively developed but no one has really pulled it off yet -- so it's fair to say that some of the cautious investor sentiment is warranted. What we are attempting to do is not easy and failure is not at all uncommon when a new technology is being developed, and especially in an incredibly challenging field like this one.
That said, BlueRock has a very clear thesis and we are working with some of the best stem cell scientists and engineers in the world to advance our platform technology. Additionally, we are utilizing our connections to regulatory bodies who understand the significance of our efforts, and want to work with us to lay out the best approach to demonstrate effectiveness for patients.
Investors such as Versant Ventures and Bayer saw both the maturing state of pluripotent stem cell technology as well as its potential to be truly transformative, much in the same way combinatorial chemistry and molecular biology transformed therapeutics development, first with small molecules and then with biologics. They were willing to make a significant investment (one of the largest series A rounds in biotech history) in a potentially game-changing idea that could form the backbone of the future treatment paradigm for many patients.
It will be interesting to see if other investors and pharma companies try this approach. Looking back at the last year I’ve been pleased to see the size of the initial rounds for other single disease area-focused cell and gene therapy start ups and several of these companies seem to be on their way to raising sizable follow-on rounds. I would not be at all surprised to see investment appetite for the space increase in the coming years as we all continue to validate our efforts and we may be close to that tipping point.
As someone who can boast having both a leading business consultancy and a major VC company on your CV, can you give us 3 key learnings from the course of your career to date that you will be implementing in your new role of VP Corporate Development & Strategy at BlueRock?
People come first. If you follow this basic life rule you’ll never go wrong.
Carve out time for self-reflection – it will help you “connect the dots” looking backward.
Trust your instincts - as Steve Jobs said, you have to find a way to trust that the dots in your life will somehow connect in the future.
Finally, what are you hoping to get out of your participation in Phacilitate Cell & Gene Therapy Europe 2017?
I look forward to collaborating with some of the brightest minds in the industry as we discuss the trends driving the cell and gene therapy fields, the challenges and hurdles that we will have to overcome to deliver breakthrough treatments for patients, and the potential of these treatments for solving some of the most debilitating disease. Additionally, I am hoping to make important connections with talented people who want to work at a transformative company and might fill one of the many positions for which we are actively hiring in the coming months.