Genetic Engineering & Biotechnology News

DEC 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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6 | DECEMBER 2017 | Malorye Allison Branca It's one of the highest-stakes races in all of biopharma. Top scientists around the globe are jockeying to develop the first approved medical treatment based on clustered regu- larly interspaced short palindromic repeats (CRISPR), or more precisely, CRISPR/nuclease gene-editing tech- nology, which arrived on the scene just a few years ago. Initially, a hand- ful of intriguing initial human stud- ies, including some in embryos, have helped fire up the field. Now, dozens of CRISPR-based trials are planned worldwide and, in a new twist, China seems to be ahead of the pack, with the U.S. trying to catch up. "Gene editing is exploding," says Rhonda Bassel-Duby, Ph.D., professor of molecular biology, UT Southwestern Medical Center, whose group has done groundbreaking work with CRISPR and Duchenne muscular dystrophy models. The rise of CRISPR has also revived interest in older gene-editing techniques, such as those based on zinc finger nucle- ases (ZFNs) and transcription activa- tor-like nucleases (TALENs). Start-up Homology Medicines recently raised $127 million for a little known edit- ing technique based on viruses. Faster, cheaper, easier to use, CRISPR is already a clear winner on the lab bench. "The CRISPR revolution has been one of the most monumental technical advances in the gene-editing field," said Christos Georgiadis, Ph.D., of the Great Ormond Street Institute of Child Health, University College Lon- don (UCL), U.K. It provides targeted genomic knockout efficiencies that are "unprecedented," he adds. But many also think CRISPR can be used to create a host of new cures. It also offers a means to tweak other therapies, such as those based on T cells or stem cells, including the breakout chimeric antigen T cell (CAR-T) cancer treatments, the first of which (Novartis' Kymriah [tisa- genlecleucel]) was just approved. The market for CAR-T is expected to be worth more than $8 billion within ten years, according to research by Coherent Market Insights. However, those therapies are hampered by the fact that they require removing, altering, then re- administering a patients' own cells, which is a lot of handling and added expense. CRISPR, experts think, might be able to solve this prob- lem. "Could CRISPR make these less individualized?" asks Rachel Haurwitz, Ph.D., president and CEO of Caribou Biosciences. That would turn an autologous treatment into an allogeneic one—a big win. The first CRISPR-based trial was conducted by a team lead by Lu You at Sichuan University, Chengdu, China in October 2016. Those researchers injected a lung cancer patient with immune cells edited to knockout the activity of PD-1—a A Dose of CRISPR B E S T O F C R I S P R 2 017 Poseida researchers in the lab. Can Gene Editing Cut It in the Clinic? ➜

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