State of the Regenerative Medicine Industry
August 4, 2017

"You are morally obliged to find something new and then you are morally obliged to tell everyone about it" -Dr. Arnold Caplan, Business of Regenerative Medicine 2017

From July 17-19, I attended the Business of Regenerative Medicine conference in Toronto, ON. There was a diverse range of people attending this conference, from early RM entrepreneurs, CEOs of RM-related companies, RM-focused investment firms and professors whose work focuses on advancing RM. This conference was broken down into a series of talks regarding a variety of topics in the regenerative medicine (RM) field. In this series, we heard from top leaders in their respective fields about where the regenerative medicine industry is currently situated and glimpses into what is to come.

Dr. Phil Vanek from GE Healthcare kicked off this series. Dr. Vanek emphasized the importance of encouraging clinicians to administer and choose RM strategies. We can transition the current state of the RM industry to the required state by achieving operational excellence and compliance through safe and effective means. For example, automated robotics will need to replace the typical manual labour done by humans in order to achieve a production capable of supplying the demand of RM technologies. To accomplish this, the industry will need to adopt a “hands-off” approach to reduce the risk per dose as opposed to the cost per dose over the next 10 to 15 years. These advances are not necessary to treat millions of patients, but the projected hundreds of thousands of patients who will be able to benefit from RM strategies.

Dr. Vanek reinforced the fact that on average it takes 3 to 7 years to bring an idea to a product state in the RM industry. This is much slower than the IT industry where no specialized equipment is needed to and rapid prototyping can occur. The RM industry also requires approximately $1 to $25 million to progress from the idea to product stage. Each RM therapy can cost hundreds of thousands of dollars. With the example of T-cell therapy, there are extensive costs associated to consumables and reagents required to treat one patient with cell therapy. To reduce this cost, we can produce more cells effectively OR we can use fewer of the "right" cells. No one knows which one will be more cost efficient but in my opinion, strategies that pursue each route will eventually weed out the more cost efficient method.

Dr. Vanek focused on the development of technologies that can enable major changes in the RM industry. One of these technologies will need to allow devices from various manufacturers to "talk" to each other. There will need to be a standardized technology that enables both the hardware (i.e. tubing) and software to be compatible with various manufacturers. CCRM is investigating this with the BridGE initiative, which employs engineers and scientists to tackle the manufacturing workflow problem. The IT industry can take advantage of this deficit of enabling technology in the biotech and RM industry. Custom software distributed on an open source platform will have the greatest advantage of being adopted by multiple companies to allow hardware to "talk" to each and improve workflow in a continuous manner.

Next up to discuss the science of the RM industry was Dr. Peter Zandstra, Chief Scientific Officer of CCRM and Post-Executive Director of the Medicine by Design program.

He introduced his talk by stating that RM strategies can either engineer the cell or engineer the niche to achieve the desired outcome. Engineering the cell would include strategies such as genetic engineering to express certain biomarkers to make the cell hone in on their target. Engineering the niche may include changing the physical and chemical nature around the cell through various biomaterials, topographies, or pharmaceutical agents. Dr. Zandstra explained that there is a disconnect between research and product that needs to be filled through commercialization and manufacturing efforts. Dr. Zandstra's talk focused on engineering the cell. He hoped that one day we could design cells like we currently do microchips. We want to engineer biology in a controllable and predictable behaviour through synthetic biology.

What interested me the most was during the Q&A portion where someone asked: What kind of team is needed for these advances in RM to happen? Dr. Zandstra stated that an interdisciplinary team skilled in everything from biomedical engineering to quantitative biology will aid in enabling these advances. These teams will focus on generalized problems rather than a specific field, something that the Medicine by Design at the University of Toronto initiative is already implementing.

Dr. Reni Benjamin of Raymond James Financial Inc. covered the state of investment in the RM industry. His talk focused on the economic risk of investing in the RM industry, with increases and decreases depending on certain events or publications in the cell and gene therapy field. What I liked most about the presentation was his 7 lessons that can be applied to cell and gene therapy (as well as other biotech fields):

1.Ignore the noise and focus on the fundamentals

Data, both clinical and pre-clinical, is the most important aspect of convincing all parties invested in the technology that it will be successful.

  1. Be realistic regarding valuations

I immediately thought of Dragon's Den where companies consistently overestimate their worth. Companies need to be as realistic as possible when appraising their worth.

  1. Raise capital when you can, not when you have to

Raising money is the only way for smaller companies to continue operations until a profit can be made. Dr. Benjamine encouraged start-ups in the cell and gene therapy space to always be looking to raise money and not leaving it to when it is absolutely crucial.

  1. Watch the markets closely and understand where you are in the cycle

Companies have failed and succeeded in the cell and gene therapy field before. It is important to learn from these mistakes and progress forward to a successful future.

  1. Have multiple shots on goal

Do not only have one technology that has the potential for commercialization. It is important to diversify your investment platform to ensure that a failed or rejected technology does not cripple the company.

  1. Don't drink your own Kool-Aid and try to be objective

Try to look at your company through the eyes of someone not within the company. This will help you and your employees understand where potential problems may arise and be realistic with what you already have and what is feasibly possible within a reasonable amount of time.

  1. Listen - especially to the people who don't like your story

Those who think your technology is not going to succeed can often provide insights from another view that you may not have considered before. This can aid a young company in ensuring that they do not make the similar mistakes of another failed company or technology.

Dr. Michael May closed off this series with a talk about increasing the "stickiness" of the RM industry. Dr. May is the CEO of CCRM, a Canadian government funded initiative to help RM based technologies commercialize. He mentioned how important it was for the creation of RM focused risk capital to ensure that young companies with successful technology will have the same opportunities for growth as multi-million dollar corporations. Canada is set to take the stage as global leader in RM and needs to solidify that position with global investment in RM focused Canadian companies.

I hinted at synthetic biology earlier in the post, but I will distill what Andrew Hessel, a researcher with Autodesk Life Sciences, talked to us about how synthetic biology can potentially save the world. Stay tuned!

Written by
Nathan Holwell
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