Bioregnum, Gene Therapy

While Jiankui He gets all the attention, don’t overlook the giant step that Editas is taking today on the CRISPR front

Bioregnum Opinion Column by John Carroll

As health officials the world over — and especially in China — breathe fire over Jiankui He’s apparently credible claims that he skated clear of regulators and genetically altered the embryos of two newborns to guard them against HIV, Editas $EDIT today is celebrating a critical milestone in the long, hard journey to launching its first human trial with major implications for the CRISPR field.

The FDA has accepted Editas’ IND for their first gene-editing trial in a handful of patients suffering from an inherited retinal degenerative disease called Leber Congenital Amaurosis type 10.

Normally, that’s not something that gets much attention in the biopharma world these days. As money has pumped into the system, creating a wave of new biotechs, INDs have become routine — even if, as in Editas’ case, it comes with a $25 million milestone payment.

Katrine Bosley, Editas

Editas, though, set out fully 5 years ago to widespread acclaim, one of several startups that were inspired by 3 scientists: Feng Zhang, Jennifer Doudna and Emmanuelle Marie Charpentier. Now, after the applause has died down, Editas and its partners at Allergan will finally have their shot to see whether gene editing tech — in this case CRISPR/Cas9 and the newer CRISPR/Cpf1 — can play a big role in correcting severe diseases. 

To get here Editas has had to fight off a stiff patent challenge, keep the money pumping in as a public company which necessarily had to spend years in preclinical development, bat back persistent questions about off-target effects and convince regulators that it had everything lined up properly before it shifted from animal studies to humans.

“The FDA’s acceptance of our IND for EDIT-101 is a significant moment in the field of genome editing,” noted CEO Katrine Bosley, “and importantly, a critical milestone for patients, as we are now one step closer to a treatment for LCA10.”

Jiankui He will end the week as one of the most notorious rogue scientists in the world. Editas will be better off financially and one big step closer to a dramatic test case to gauge the near-term potential of what is still a radically new technology. It won’t get as much attention, but in the long run it’s much more important than the headlines coming out of Hong Kong.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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