Poseida researchers in the lab.
Poseida researchers in the lab.

It’s one of the highest-stakes races in all of biopharma. Top scientists around the globe are jockeying to be first with an approved CRISPR-based treatment. The breakthrough gene editing technique, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), arrived on the scene just a few years ago. But already a handful of intriguing initial human studies, 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, 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 zinc fingers and TALENs. Start-up Homology Medi­cines recently raised $127 million for a little known editing 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 of the Great Ormond Street Institute of Child Health, University College London (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 CAR-T cancer treatments, the first of which (Novartis’ Kymria) was just approved.


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