It’s the time of year when science journalists are scouring labs around the globe to compile lists of the hottest breakthroughs from 2015, and the technologies that will change our world in the coming year. Many of these will impact healthcare and medicines development, from machine learning to 3D printing. But one which has the potential to revolutionize the pharmaceutical pipeline, and was named Science magazine’s breakthrough of 2015, is CRISPR genome editing technology.
The CRISPR-Cas 9 system is a technology based on a naturally occurring bacterial process that allows scientists to make very precise changes in the genome of virtually any type of cell or species of animal. Whilst other technologies were available previously, the development of CRISPR has made genome editing significantly cheaper and quicker, allowing it to be applied to an incredible range of situations, from cell lines to gene therapies.
A recent Wellcome Trust Sanger Institute-AstraZeneca conference highlighted some of the incredible advances in this field, and how scientists from academia and industry are already using genome editing technologies to accelerate the translation of scientific breakthroughs from bench to patient benefit. For example, cell lines modelling a specific patient's cancer with their specific genetic mutations can now be generated in just a matter of months, accelerating understanding of the disease and drug discovery. Others are using the technology to carry out very high throughput screens of potential drug targets, identifying or eliminating potential hits rapidly.
However, much of the excitement around genome editing comes from the potential to use it directly in new therapies. Results from early studies using genetically engineered immune cells to target cancer have shown great promise, and genome editing techniques may improve the development of these therapies. The potential also exists to directly edit the genome in humans, opening the possibility of therapies for previously untreatable rare genetic conditions, such as Duchenne Muscular Dystrophy. Such studies are already underway in animal models, although it will be several years before they reach the clinic.
The potential of the field is difficult to deny. As with all new technologies though, a note of caution is important. It is vital that the bioscience sector, including our industry, is fully engaged in considering and discussing the societal impacts of the technology we develop and use. The discussion of regulatory and ethical challenges must keep pace with technological development, which is no easy feat. Genome editing techniques raise challenging ethical questions. This is particularly the case in relation to the potential to modify the human germline, which was addressed at the recent Washington International Summit on Human Genome Editing. Beyond this though, the regulatory, societal, and patient implications are broad. We particularly welcome the Nuffield Council for Bioethics' ongoing work on these, and look forward to contributing to this important conversation.
Developing new technologies to bring treatments to patients is at the heart of what our industry does, and there is a wealth of possibilities for how new science will shape industry's R&D in 2016…organs on a chip, big data, genomic sequencing. CRISPR is just one of these, but certainly has the potential to be a game changer for medicines development this year.
We will be responding to the Nuffield Call for Evidence and we would welcome member comments and input. Please contact me for more information.