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, but it has also 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 the 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 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). The 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.
- 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 the 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.