The Future of CRISPR

Where will CRISPR deliver real value?

Investors and pharma are enthusiastic about CRISPR gene editing technology: not only can it be used to develop new therapeutics, it has become a powerful tool in drug discovery, clinical trial stratification, diagnostics, and may even beneficially impact the manufacturing cost of cell based biologics. But the commercial potential of CRISPR has yet to be fully realised: patent disputes, safety concerns and, many argue, the need for suitable regulatory frameworks to ensure technical and ethical requirements are met.

So, where is CRISPR delivering now and what direction will the technology go in? In The Future of CRISPR, we interviewed leading advocates to help you evaluate where—and how—CRISPR is transforming drug discovery, clinical research and therapy development.

Experts explore CRISPR opportunities and challenges

Why are pharma and biotechs interested in CRISPR and where are they using it?
Why is CRISPR gaining credence over other gene editing techniques?
What companies are leading the field in CRISPR technology and why?
What therapeutic areas are pharma exploring with CRISPR and where might this lead in the future?
What impact could patent disputes have on freedom to operate in this field now and in the future?
How are companies looking to improve the specificity of gene editing and extend the duration of action?
Are there sufficient regulations and incentives in place to encourage innovation in the CRISPR space now and in the future?

What our experts say…

"Genome engineering's been around for a while with TALENS and using ZNC fingers since 2005 with good success, but its use is limited. The informatics tools needed to build a ZNC finger or TALENS are very complicated and the barriers for adoption are high. There have been some very successful gene editing programs in pharma and biotech but it requires huge investments. Six years ago to contract the generation of a ZNC finger could cost hundreds of thousands of dollars. CRISPR on the other hand is much simpler and cheaper, all you need is a guide RNA, Cas9 and some informatics about where you're going to cut the best sequence and the rest of the tools are in the lab."
Ted Tisch,
Synthego

"The big pharma guys have all taken, in our experience, a slightly different approach to CRISPR. All of them clearly saw the value, but they all took a different kind of approach in terms of whether or not they wanted to in-license, or outsource the work, acquire or de novo develop their own tools. I think some of the people who have made the most impressive impact early on are people like Novartis and AstraZeneca who were early adopters and developing things with their own tools. But, all of them have their own take on it."
Benedict Cross,
Horizon Discovery

"The natural marriage between CRISPR technology and disease is for genetic disease, a disease that's inherited like recessive conditions such as cystic fibrosis, dominant conditions like Huntington's or amyloidosis, or a poisoned or a missing gene. A second natural marriage here is editing the genome of cell therapies, to do a more comprehensive reprogramming of a cell. We have editing that we do inside the body, where we use delivery vehicle such as lipid nanoparticles and then there's the engineered cell therapies which I think all the companies are doing because of the simplified delivery challenge."
Thomas Barnes,
Intellia Therapeutics

What to expect

A detailed report, supported with case studies, examining the current status and future trends in CRISPR gene editing technology
An examination of 7 key issues which drug developers need to understand and respond to
30 targeted questions put to CRISPR advocates
Their perceptive responses that provided 35 insights supported by 67 directly quoted comments

What is CRISPR?

Clustered Regularly Interspaced Short Palindromic Repeats– CRISPR associated protein 9 Cas9 (CRISPR-cas9) (CRISPR) is a gene editing technology discovered in 2012 by Jennifer Doudna (Berkeley, California), Emmanuelle Charpentier (Max Planck Institute for Infection Biology, Berlin) and Feng Zhang (Broad MIT). CRISPR technology has been widely applied by the pharma and biotech industry to accelerate drug discovery, identify new targets and develop novel therapeutics. In the US, more than 20 clinical trials are currently underway evaluating CRISPR therapeutics in a range of diseases, including rare monogenic haematological and ocular diseases and polygenic cancers.

Expert contributors

The report harnesses critical insights and opinions from front-line industry experts who completely understand the potential for CRISPR and the key areas where pharma can benefit.

Benedict C S Cross, Head of Functional Genomics at Horizon Discovery. Ben joined Horizon in 2013 to expand and advance Horizon's functional genomic screening capability and to lead a major research alliance in synthetic lethal target discovery. Ben led the establishment of pooled CRISPR-Cas9 screening at Horizon and co-developed the Dual Screening methodology with CRISPRi and CRISPRa to target complex mechanism of action analysis. Ben now heads up the teams applying these functional genomic screening platforms in target discovery, target validation and other research programmes.
Ted Tisch, COO of Synthego. Ted is responsible for product development, operations and commercial functions, as the company develops full-stack engineering platforms delivering innovative genome engineering solutions. Before joining Synthego, Ted spent 25 years in at Bio-Rad Laboratories developing leading reagent, instruments and software products in Research & Development, running Reagent Operations and serving six years as Vice President and General Manager at Bio-Rad Laboratories of the Protein Function Division.
Thomas Barnes, Senior Vice President, Innovative Sciences, Intellia Therapeutics. Tom 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.. He has wide-ranging knowledge of biological systems through his work across diverse platforms, including genomics and gene discovery, small molecule drug repositioning, and protein engineering. Previously, as Vice President of Discovery at Eleven Biotherapeutics, Tom led his team in creating a novel chimeric cytokine antagonist, as well as two novel technologies. As Senior Vice President and site head of GeneLogic's drug repositioning division, he oversaw technology platforms in metabolomics, toxicogenomics, gene expression informatics, and the genetics of drug metabolism and transport.

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The Future of CRISPR

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