Gene Editing

Editas loses its lead in the biotech race to launch the first CRISPR/Cas9 human study

Katrine Bosley, Editas

Editas was the first of three gene editing biotechs to make a splash on Nasdaq and up until this week it also looked like it was in the lead in the race to be the first in the group to make it into a groundbreaking human study.

No more.

On Monday Editas $EDIT CEO Katrine Bosley said that a “manufacturing delay related to production of input materials for AAV manufacturing” forced the company to delay their planned IND filing to the middle of next year. As execs explained it, one of the input materials used in making their delivery vehicle failed a quality control inspection, and the delay in fixing that caused them to lose their time slot with the CMO.

CRISPR Therapeutics CEO Rodger Novak

It’s a significant delay. Editas, which saw its stock drop 5% in pre-market trading Tuesday, had planned to file late this year. And in the process of slipping behind, the biotech lost its lead to CRISPR Therapeutics. In CRISPR’s Q1 update $CRSP a few days ago CEO Rodger Novak noted that his group plans to file its CTA — clinical trial authorization in Europe — in late 2017 for its lead therapeutic product to treat beta-thalassemia.

Intellia $NTLA, meanwhile, is still focused more on animal studies, with IND enabling studies for its ATTR program slated to run through the second half of 2017 and the first half of 2018.

These three biotechs have been slugging it out over control of the patents behind one of the most dramatic new openings in drug R&D of the decade. By using CRISPR/Cas9 tech to edit genomic DNA, these companies believe that they are starting down a path that will lead to once-and-done cures for a legion of ailments. And the scientists behind the launch of CRISPR Therapeutics and Intellia — Emmanuelle Charpentier and Jennifer Doudna — have worked to get Editas’ patents from the Broad thrown out, saying they invented the technique. So far, though, they have failed at that.

However the patent fight turns out, they’re all years away from proving who can take the lead in commercializing the technology. But these early plays are already highlighting how significant changes in initial clinical timelines can ultimately affect the outcome of the bigger late-stage race to come.

None of the biotechs can claim to be the first to try it in humans. That distinction already fell to a group in China that pulled off an early exploratory study. And Sean Parker has agreed to fund a human study led by Carl June at the University of Pennsylvania that could be the first to get started in the U.S.

There are bound to be plenty of big surprises ahead.


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