What are the key reasons that brought you to start this new journey with Intellia?

I had the wonderful opportunity to be there when small molecule drugs and antibody therapies were emerging as viable solutions for various diseases. However, there were instances where neither of these two worked and so we have had to go deeper at a cellular level in order to actually change the course of a particular disease.

The rise of genomic and especially CRISPR technology completely shifted the paradigm in pharma; barriers were crossed and a new pool of opportunity had developed.

As a result, we jumped on this emerging technology and founded Intellia, with the precise purpose to make our therapies as accessible as possible.

CRISPR has broad applicability but we have been focusing on genes that are active in diseases that emanate from the liver. For now, we are concentrating our efforts around Amyloidosis and Hereditary Angioedema but also on "ex vivo" work in the oncology and inflammation space. "Ex vivo" means that we can take out cells from the body and rewire them in such a way they can detect cancerous cells or turn off inflammatory pathways responsible for diseases such as Lupus. 

According to which criteria can you detect and solve certain diseases?

We are using a systemic delivery approach since in most genetic conditions it is impossible to take the tissue out and manipulate it. As a result, we needed to come up with ways to take the technology into the body. This approach opens up broad vistas for diseases that would have been otherwise overlooked. 

Every time we develop a new technology, we try to go to uncharted areas and solve really unique problems that have not been tackled before. We are always thinking about the nature of the genetic change that is implied and we are making sure that we can reach the cells that contain the gene that is defective. As technology expands, the goal is to increase the set of tissues that we will ultimately be able to reach and, thus, extend the list of diseases that will become solvable. The array of genetic changes that we are able to perform is continually evolving, but getting the technical apparatus to the affected cells is a challenge that we still need to work on. 

How have you perceived the pharma environment from a funding and regulations point of view?

Since we are in the pre-commercialization phase, we are attracting investors through demonstrating the progress that we have been making so far with the sets of products that we are

working on. At the same time, we are working with like-minded collaborators in the industry (like Novartis and Regeneron) because we have seen how teaming up usually leads to brilliant results. In this synergy, we are bringing the expertise regarding the inner workings of a cell and our partners are using this capability to gain insights about a particular biological outcome. 

When CRISPR technology emerged, there was an array of discussions regarding its boundaries and the way it should be regulated, but nowadays we are under the same legislative umbrella as classic drugs. With the exception of a few added essays that are necessary for us to show how the drug works, we found the environment really supports what we do and this is extremely encouraging. 

What are the main objectives that you would like to achieve in the next couple of years at Intellia?

The most ardent priority is to get our two lead products (for Amyloidosis and Hereditary Angioedema) approved and ready for the market. Along with that, we are also focused on finding new and creative ways of expanding the uses of technology in areas that are hard to reach, especially in the oncology space. Gene based medicine is here, it has tremendous potential and it is only going to grow in importance in the following years. The insights accumulated over the past few years are starting to bear fruit in a meaningful way and it is only a matter of time until there is a set of products that people will be able to get access to, in the near future. The beauty of it is that when I was in medical school, I could not even fathom that all these advancements would become a common place in medicine. All these new therapies are indeed very welcome to the market but the truth is that we still need to find viable solutions for broader funding and accessibility. In the end, we need to think about the perfect ecosystem where new technologies are largely available and also easy to reach (from a financial standpoint) by a wide patient population.