The Future Is Now: Dr. Jim Burns of Locanabio On How Their Technological Innovation Will Shake Up Healthcare
“Working smarter is better than working harder.” Sometimes I think that it would have been smarter for me to work as hard as I have on how to do things better, rather than just do more of what I was doing.
As a part of our series about cutting edge technological breakthroughs, I had the pleasure of interviewing Dr. Jim Burns.
Jim Burns, PhD, joined Locanabio in December 2019 as CEO and Board member. Prior to joining Locanabio, Dr. Burns served as the CEO of Casebia Therapeutics and led the team in discovering and developing new CRISPR/Cas9-based breakthrough therapeutics to treat blood disorders, blindness and heart disease. Prior to Casebia, Dr. Burns spent the bulk of his career at Sanofi-Genzyme, where he held several leadership roles with increasing responsibility, including North America Site Head for R&D where he coordinated R&D operations across key therapeutic areas, and head of Sanofi-Genzyme R&D. Dr. Burns is a former board member of MassBio, a member of the National Academy of Engineering and a member of the External Advisory Committee for the BioMedical Engineering Department at Boston University. He also served as the industry representative for the Food and Drug Administration General Plastic Surgery Panel. Dr. Burns earned a Bachelor of Science degree in biology from Purdue University and Master of Science and Doctorate degrees in bioengineering from the University of Illinois-Chicago, where his thesis work focused on drug delivery. Following his graduate studies, he was a post-doctoral researcher at the University of Florida.
Thank you so much for doing this with us! Can you tell us a story about what brought you to this specific career path?
In 1984, I was interviewing for a postdoctoral position at the University of Florida which unfortunately, was less than impressive upon further examination and ultimately would not have given me the experience that I wanted to further my career. I could not stop thinking about another postdoctoral position I had previously applied for with Eugene P. Goldberg, PhD, Director of the Biomaterials Center in the Department of Materials Science and Engineering at the University of Florida. At the time, Dr. Goldberg said he simply did not have space for me on his team. But I was determined to meet with him and convince him to hire me. I remember walking up to his office, knowing that he was not expecting me and that he may not even be there. There were two things that were remarkable about that day: 1) that same day Dr. Goldberg’s father, unfortunately, had passed away, and 2) Dr. Goldberg was standing at his desk holding a piece on paper with my name and number, looked up and said, “I was just going to call you because a position just opened up.”
The reason I wanted to work with Dr. Goldberg was because I wanted to work with someone who could help me understand how I could take basic science and engineering principles and apply them in a translational way to products that could actually get into people and help them. Some of those skills and mentoring opportunities I got during my two years with Dr. Goldberg at the University of Florida, learning about product development and translating early ideas into products that can help people, were in some ways how I ended up working at Genzyme (now Sanofi Genzyme) for as long as I did. Through Dr. Goldberg’s mentoring and his professional relationships, I was able to start my journey at Genzyme and in the field of pharmaceutical and biotechnology product development.
Can you share the most interesting story that happened to you since you began your career?
I am not sure how interesting this is to others, but early in my Genzyme career there was a situation that shaped how I think about leadership. During my first year at Genzyme, my lab sponsored work with an external collaborator on an important project that we hoped would lead to a significant corporate partnership. However, the potential partner could not reproduce the collaborator’s results, which we were relying on to close the deal, and they walked away from further discussions. There was a lot at stake so you can imagine this caused much consternation within the R&D leadership, the board of directors, and with our CEO Henri Termeer. Henri called a meeting and asked each of us — starting with me — if we believed the technology really worked. At this point, I had nothing to lose so of course I said, “Yes.” Henri then went around the room asking each in turn the same question and all said, “No,” except for the last person he asked who happened to be my boss — the head of research for Genzyme — and who did not like the project. To my surprise, he said, “Yes, and give Jim three months to prove it.”
Three months later, my lab produced results showing the technology worked, and most importantly a completely independent testing facility using the same study protocol under good laboratory practice (GLP) conditions showed exactly the same results. With these studies in hand, we were able to raise $35 million to fund projects that ultimately generated two commercial products. This episode showed me an important leadership feature by two different people in the same meeting. Sometimes support of the person is more important than doubt about a project. My boss did not believe the technology would work (most things do not in our business, after all, so this was not a crazy position), but he realized that I did believe, and it was important to him to support me. And Henri knew it was important to support his head of research and the new kid, even if there were questions about the technology. After that moment, I had the greatest respect for Henri and my boss as leaders, and always tried to incorporate an element of people impact in my decisions.
Can you tell us about the cutting edge technological breakthroughs that you are working on? How do you think that will help people?
At Locanabio, we are focused on correcting disease-causing mutations at the RNA level. While gene therapy continues to be a focus among many pharmaceutical and biotechnology companies, including those focused on rare diseases, in recent years RNA has been in the spotlight, thanks in part to the emergence of nucleotide-based RNA targeting platforms. We are advancing a new class of gene therapies that target dysfunctional RNA. The combination of RNA modification and gene therapy delivery via one-time administration makes Locanabio’s platform, called the CORRECTX™ platform, unique. It is positioned as a promising therapeutic approach that could transform the treatment of many genetic and even non-genetic diseases, many of which currently have limited or no approved treatment options.
How do you think this might change the world?
We believe that like antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), gene editing, gene therapy and now mRNA vaccines, engineered RNA-binding protein systems have the potential to be a significant new advance in medicine because of the inherent modularity of the technology and because of the breadth of diseases for which a one-time RNA targeted therapy can be the best approach. Through creative manipulation of the basic systems, we can effect a number of different types of RNA manipulations, each of which has a number of genetic and non-genetic disease applications. We live in an amazing time in which many genetic medicines have either been approved or are making significant clinical advances. All have a place in the armamentarium of tools to apply to human disease, and we believe our approach with CORRECTX™ will as well.
Using the CORRECTX™ platform, we are pursuing applications in devastating neurodegenerative, neuromuscular and retinal diseases including myotonic dystrophy type 1 (DM1), Huntington’s disease, spinocerebellar ataxia type 1 (SCA1) and familial amyotrophic lateral sclerosis (ALS).
We recently published promising preclinical study data on our platform in the journal Nature Biomedical Engineering. Based on our platform, we developed what is called an RNA-targeting CRISPR Cas9 system (RCas9), which is comprised of an RNA-binding protein and RNA-modifying enzyme, that is delivered via a gene therapy adeno-associated virus (AAV) vector to destroy disease-causing RNA. We assessed whether the CRISPR RCas9 system could provide molecular and functional rescue of dysfunctional RNA processing in mice with DM1. Results from the preclinical study showed, for the first time ever, that the molecular and physiological features of DM1 could be reversed, showing the significant potential of our platform technology to treat this devastating neuromuscular disorder.
Keeping “Black Mirror” in mind can you see any potential drawbacks about this technology that people should think more deeply about?
As more gene therapy and gene editing technologies emerge, it is important to think carefully about how we leverage these tools to best treat genetic diseases and to recognize that there is no single approach or treatment that will be right for every patient. It is incredibly exciting to see the gene therapy and editing landscape rapidly progress and hopefully one day we will reach a point where physicians can ask themselves, “What is the right genetic approach or genetic tool I should use to treat my patient in the safest possible way?”
Was there a “tipping point” that led you to this breakthrough? Can you tell us that story?
Our unique CORRECTX™ platform technology was born out of research conducted by Locanabio founder and world leader in RNA biology, Gene Yeo, PhD, at the University of California, San Diego (UCSD). The “tipping point” was the moment he realized that gene therapy could be used to deliver proteins that bind to RNA, rather than DNA (as with CRISPR Cas9). This new approach, called RNA directed Cas9 (RCas9), is the basis of our platform and allows us to combine two validated technologies in medicine — RNA modification and gene therapy.
What do you need to lead this technology to widespread adoption?
We recently completed a $100 million Series B financing to support preclinical and clinical development of our platform technology for treating diseases such as Huntington’s disease, DM1, genetic ALS and retinal disease. With new funding we hope to accelerate our efforts to advance multiple promising development programs, initiate what we call investigational new drug (IND)-enabling studies, and further develop our platform, which has the potential to be a major new advance in medicine.
What have you been doing to publicize this idea? Have you been using any innovative marketing strategies?
We have been and continue to speak with a range of industry stakeholders who are important to us, including investors, analysts, potential partners, key opinion leaders, physicians and patient advocates, about the significant potential of our platform technology and its unique approach to addressing disease areas of unmet need. We have also been promoting important company and development milestones throughout the year in press releases and speaking with a number of business and medical trade media to build broader awareness of our technology. In just one example, Locanabio was recently featured in a leading medical trade publication called Genetic Engineering & Biotechnology News about technological advances and breakthroughs in medicine, which was subsequently featured in an article in The New York Times Magazine.
None of us are able to achieve success without some help along the way. Is there a particular person who you are grateful towards who helped get you to where you are? Can you share a story about that?
I have already mentioned an example of Henri Termeer influencing my thinking on leadership. But he also had an impact in a very simple way on how I think about innovation. About six months after the situation I described earlier, I was walking with Henri and my boss, the head of R&D at Genzyme, across the Boston Common on our way back from a visit at Massachusetts General Hospital. I was talking about all the ideas I had for products that we should work on in my lab. And it must have been irritating to listen to, but Henri did not say anything until we got across the Common when he stopped, looked at me and said, “Ideas are a dime a dozen,” and then he kept walking. Those six words had a profound impact on me because I, of course, immediately realized that I had to deliver, not just come up with ideas. I became highly focused on learning the process of product research and development. It became a science to me; how to define goals, deliverables, develop plans, run meetings, hire great people, and assemble effective teams. I realized that great ideas were necessary but not sufficient for innovation. One had to execute effectively to ultimately deliver products to patients, which is when you can only say innovation has really happened.
How have you used your success to bring goodness to the world?
I feel that the hardest thing humans do is develop drugs to try to fool biology to do something that a specific type of cell is not programmed to do. We are developing a completely new class of gene therapies that are able to target the root cause of certain genetic diseases by correcting mutated or dysfunctional RNA in patients. The diseases we are targeting are serious, devastating and underserved, largely because they are rare diseases, but we are leading the way in creating a new advance in medicine that could bring patients much-needed treatments that can truly make a significant impact on their health and quality of life.
But 10 years or so from now, I hope that we can leverage our platform to go beyond genetic medicines and treat other types of diseases, even non-genetic diseases. Our team is very passionate about transforming our platform technology into something that can help as many patients as possible.
What are your “5 Things I Wish Someone Told Me Before I Started” and why. (Please share a story or example for each.)
- “Working smarter is better than working harder.” Sometimes I think that it would have been smarter for me to work as hard as I have on how to do things better, rather than just do more of what I was doing.
- “It is ok to say no.” The art of people and project leadership is to balance genuinely listening to people with making decisions firmly even if they are not welcome. Every time you make a decision you are saying “no” to another option, so embrace saying “no,” or you will never get anywhere as a leader.
- “It is ok to be wrong or make mistakes as long as you learn from them.” We go through life and school getting bombarded with negative feedback if we do something wrong, get a poor test score, etc., so we avoid admitting mistakes. This is a disaster from a personal growth and continuous improvement standpoint. Making mistakes and learning from them is key to becoming better.
- “Listening is more important than talking.” I read somewhere that a person who talks a lot is more often thought a fool than someone who listens and is thoughtful about what they say. I have learned to like this way of thinking because by listening you gather information and when talking the information is only going out. As a leader you have to balance this with the need for people to know where you stand, even if you agree with what is being said.
- “People will remember about 10% of what you say in a presentation, 10% of how you said it, and 80% how you made them feel.” When I heard this from a former TV news reporter it changed how I prepared and delivered my presentations. It made me much more connected to the audience, and I believe helped me give better presentations.
You are a person of great influence. If you could inspire a movement that would bring the most amount of good to the most amount of people, what would that be? You never know what your idea can trigger. 🙂
I wonder how much energy we would save and how much positive impact on the environment we would have if once a month the world turned its lights off for one hour, something similar to the citywide energy conservation and awareness campaign Lights Out San Francisco that started in 2007, only taking it to the next level.
Can you please give us your favorite “Life Lesson Quote”? Can you share how that was relevant to you in your life?
It is a quote from Henry David Thoreau’s Walden: “The mass of men lead lives of quiet desperation.” My mother shared this with me when I was about 15 years old as a lesson that you never know what someone else is going through, so keep that in mind when you deal with people and judge their actions.
Some very well known VCs read this column. If you had 60 seconds to make a pitch to a VC, what would you say? He or she might just see this if we tag them ?
Locanabio is developing the next advance in genetic medicine by combining both gene therapy and RNA modification for treating diseases. Our proprietary CORRECTX™ platform consists of RNA-targeting systems that are delivered using a gene therapy vector to modify dysfunctional RNA in a one-time administration. The platform is versatile in that we can perform different RNA manipulations — destruction, replacement, splicing, editing, and translational enhancement — which offers the potential to treat many other types of diseases, even non-genetic diseases, that other RNA-targeting therapies cannot. Our unique and highly targeted approach can also reduce the risk of DNA modifications and off-target effects associated with traditional gene therapies. We are developing therapeutic options targeting neurodegenerative, neuromuscular and retinal diseases including DM1, Huntington’s disease and retinitis pigmentosa. We recently completed a $100 million Series B financing in Q4 2020.
How can our readers follow you on social media?
Locanabio Twitter page: https://twitter.com/locanabio
Locanabio LinkedIn page: https://www.linkedin.com/company/locanabio/
My personal LinkedIn page: https://www.linkedin.com/in/jim-burns-b101aa9/
Thank you so much for joining us. This was very inspirational.
The Future Is Now: Dr Jim Burns of Locanabio On How Their Technological Innovation Will Shake Up He was originally published in Authority Magazine on Medium, where people are continuing the conversation by highlighting and responding to this story.
