Dr. Michael Vellard, Ph.D., Vice President of Research at Ultragenyx Pharmaceutical.

Dr. Michael Vellard, Ph.D., Vice President of Research at Ultragenyx Pharmaceutical.

Research Background

Dr. Vellard joined Ultragenyx Pharmaceutical as Vice President of Research in May 2013. Prior to joining Ultragenyx, Dr. Vellard worked as Head of Lysosomal Biology at BioMarin Pharmaceutical Inc., a biopharmaceutical company, from October 1999 to May 2013. He was a postdoctoral fellow in the pediatric department at UCLA Harbor Medical Center from September 1992 to June 1995. Dr. Vellard received his B.S. in Natural and Life Sciences and M.S. in Molecular and Cellular Genetics from the University of Lyon I, France.  He obtained his Ph.D. in Virology from the Pasteur and Curie Institutes (Universities Paris VI, VII and XI), France.


Q: Why did you join BioMarin?

MV: Because I always wanted to work on something more practical. Even before I started my postdoc, I always wanted to treat actual patients. My postdoc work focused on lysosomal diseases. I came to BioMarin because I was able to continue working on the same types of disease I was interested in. Doing fundamental research is also important, but it might take 10 years before it can be applied to patients. For me, it is amazingly rewarding to see patients get a lot better after your treatment. For example, kids who have Morquio Sydrome are not able to walk. But after 6 months of our enzyme replacement treatment, they can go to bathroom by themselves. There is nothing better than to see real improvement in your patients.

(For more on BioMarin’s approach to treating Morquio Syndrome check out BioMarin’s Morquio website.)

Q: Why did you leave BioMarin and join Ultragenyx?

MV: After 15 years at the same company, sometimes you need change. When I joined BioMarin, I was the 35th or 36th employee, but right now, BioMarin has more than 1200 employees. I have the feeling that when a company gets bigger and bigger, the decision process might slow down. I like the fast speed pace, and I like always to have something happen. So I joined Ultragenyx also as the 35th employee in 2013 May. Right now the company has more than 100 employees.

Q: Both BioMarin and Ultragenyx focus on rare disease. In your opinion, what is the strategy to pick one rare disease to begin with?

MV: First we need to filter the information from academic research, and we need to know what exactly we need to fix. For example, right now we are only focused on monogenetic disease, because multigenetic disease would be too complicated to be applicable. After we decide what to fix, we will find out how to fix it, and whether it is easy to fix or not. So we always need to verify specific questions to be sure we can move on to the next step. Because we are limited in our number of failures, unlike big pharmaceutical companies who can screen 5 successful compounds out of 10,000 compounds, we really have to be sure that we are on the right track at every step. If we find something is wrong, we need to change direction as soon as possible. It is a very different mindset from academic research.

Q: How mature is enzyme replacement as a therapy? Can it be applied to other diseases? How expensive is it?

MV: Enzyme replacement therapy is a medical treatment replacing an enzyme in patients in whom that particular enzyme is deficient or absent. There are a lot of rare diseases that are treated using this therapy, and it is a highly established approach. I think the biggest challenge for enzyme replacement therapy is brain-related diseases because of the blood-brain barrier, so there is a challenge of how you treat the brain with this kind of approach. However, scientists are developing direct injections into the brain for enzyme replacement treatment. The price of enzyme replacement therapy is about $100,000 to $1,000,000 per patient. But you have to be aware that these diseases are rare, so compared to the total medical spending, it is still a small portion.

Q: What do you want to say to our postdocs who want to pursue an industry career?

MV: First of all, you have to be sure about what you want. As I said before, academia is very different from industry. If you are interested in something applicable, biotech might be a good choice. If you enjoy digging deep into the details to understand everything, then biotech might not be a good choice. One of the main differences is the pace: everything in biotech is a lot faster. We need to have result to make sure we are on the right track, and you need to show progress every day! We cannot spend 20 years to understand every detail or answer every tiny question. Another difference is that adaptability is much more important in biotech. For example, right now we are focused on lysosomal diseases, but maybe tomorrow, we will need to be experts in myopathy. So you cannot say, “I love lysosomes but I’m not interested in myopathy.” You need to be prepared to be an expert not just in your field but also other fields.

Q: When you hire a new staff, which is the most important thing that you look for?

MV: For me, most of the time I am looking for a motivated person who knows exactly what he or she wants. If the person who comes from academia just wants to make more money, I would not consider him or her. Adaptability is the key for me. So it doesn’t matter if the person was doing something that is not exactly the same of what I want him or her to do. Personality is more important than the specific techniques and expertise a person has. Also teamwork is very important. I need a person who will be able to easily integrate into a team, be able to work with a team, and be efficient to push a treatment as fast as possible to the patient.