In The Know: Analyst answers — The Future of CRISPR

With interest on the rise regarding CRISPR (clustered regularly interspaced short palindromic repeats)-Cas9, we spoke to subject expert and analyst Dr Cheryl Barton to dive into recent research. From core issues to surprising insights, here she shares her takeaways from discussions with leading industry experts.


FirstWord: What makes CRISPR an important area for pharmaceutical companies right now?

Cheryl Barton: CRISPR is a gene editing technology that was discovered in 2012 and taken research by storm as it offers a cheap, easy, reliable and scalable means of editing genes.


FW: What experts have you spoken with on the subject?

CB: We interviewed leading advocates to help you evaluate where - and how - CRISPR is transforming drug discovery, clinical research and therapy development.

  • Benedict Cross, Head of Functional Genomic Screening, Horizon Discovery

  • Thomas Barnes, Senior Vice President of Innovative Sciences & eXtellia, Intellia Therapeutics, one of three companies working in this field with products about to go into the clinic.

  • Chief Operating Officer, Product Development, Manufacturing, Commercial, Synthego, involved in developing CRISPR-based tools for drug discovery and diagnostics.


FW: Which key issues generated the most interest during conversations with experts?

CB: The application of CRISPR in drug discovery – tools are already being used by pharma to accelerate development and more effectively choose drug development targets. Likewise, while it is still in early stages, pharma companies like Novartis and Regeneron Pharmaceuticals have began looking at both in vivo for sickle cell anaemia and ocular diseases, and ex vivo in stem cell cancer therapies. Experts also spoke to the possibility of future applications being used in diagnostics.


FW: What were some of the most insightful interview quotes?

CB: Regarding the speed of the CRISPR revolution, Benedict Cross from Horizon Discovery said:

“There has been no technology that I've ever seen that's developed so rapidly and become so embedded in every laboratory, with perhaps the exception of something like PCR [polymerase chain reaction] technology, but it's in no way as therapeutically applicable or as dynamic as CRISPR.”

On the topic of CRISPR gaining momentum, Ted Tisch from Synthego said:

“It's almost impossible to predict the future because of the speed of what's happened over the last two years. CRISPR is gaining momentum. Firstly, companies will focus on rare heritable diseases because that population doesn’t have any alternatives and so that’s the perfect place to start. But that's not where it will have its greater impact. That’s going to come from more common diseases like cancer and hemophilia; high-risk patients where there’s a broader patient base. But in order to achieve this we need better informatics, better controls and the regulatory environment needs to be acceptable to that.”

And lastly, Thomas Barnes of Intellia Therapeutics was quoted:

“CRISPR lends itself to a very modular approach. Essentially you can use the same setup, the same construct, the same delivery to treat a number of different diseases which all originate in the same organ, but for different patients who have those respective diseases. … It'd be nice to get to a point where you could agree with regulators on an approach that would enable you to pursue those sorts of conditions with a universal application package where you can just do the things everybody knows you can do from a therapeutic perspective.”


FW: What was the most notable takeaway from researching CRISPR?

CB: The most notable takeaway was explained via a quote from Thomas Barnes of Intellia Therapeutics, discussing where CRISPR might lead in the future: “At some point this century people will routinely have their genomes sequenced, maybe at birth. One driver for doing so will be that knowledge of your genome will have become actionable, because of approaches like the ones Intellia is pioneering. So the technology we are working on represents a frontier of a world where we think about managing the health of individuals very differently.”


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