Dr. Tom Barnes, Chief Scientific Officer at Intellia Therapeutics discusses his work on CRISPR/Cas9, and the opportunities and challenges it offers those developing therapeutic oligos and peptides.
Dr Barnes has led platform-based research and drug discovery teams for over 20 years, and is responsible for extending the reach of Intellia’s CRISPR platform into new areas. A veteran entrepreneur who has helped launch several companies, he has turned creative scientific visions into successful businesses for both startups and established organizations.
How can oligo and peptide drug developers benefit from advances in CRISPR/Cas9 technology?
The straightforward way that CRISPR can help traditional drug modalities is by providing the ability to turn any cell system into a genetic system – essentially permanent RNAi. This is useful in target validation as well as for engineering cell lines to doing screens in. Of course, CRISPR is itself a new drug modality, when combined with the right delivery system such as lipid nanoparticles or viral delivery.
What opportunities and challenges do you see for CRISPR/Cas9 technology over the next 5 years?
I’ll split this into two – editing ex vivo, and in vivo. For ex vivo, I see the greatest opportunity in doing much more sophisticated engineering of cell therapies than has been done to date, effectively optimizing the cell’s behavior for its intended purpose. One of those improvements, creating allogeneic cells, could be broadly enabling for the whole class of engineered cell therapies. For in vivo editing, the challenge will be firstly to show that we can safely and effectively edit human tissues; secondly, it will be to show that we can repair as well as knock out genes. The key opportunity also lies here, namely to bring forward therapies for patients suffering from genetic disease, who typically have had few therapeutic options.
You’ve helped launch several successful companies and are currently Senior VP at Intellia Therapeutics. What advice can you share with our readers working to get their early stage start ups off the ground?
The elements of a successful startup are: a high-quality idea; committed investors; and a great team to execute. The idea should be more than something that’s do-able; it should be compelling. It should be able to outcompete other ideas that the investor may be considering. You’ll need a business plan, not just a research plan, that also suggests how you think the investors will exit. Having said that, different investors are looking for different things, so one “no” is not a condemnation of your idea. But be willing to hear what people tell you – a “no” with a good reason can help you see a better way. You believe in yourself, which is good, but you’ll need a team. Don’t be put off by the suggestion that people will need to be brought in above you, if you have limited experience. Biotech is a team sport where experience matters a lot, and investors know that.