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
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
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.
Why Choose FirstWord FutureViews?
FirstWord FutureViews reports analyse in detail significant emerging technology and market trends that pharma executives need to understand if they are to manage the opportunities and challenges that lay ahead. These concise and highly focused reports:
All Contents Copyright © 2021 Doctor's Guide Publishing Limited All Rights Reserved