For the ailing gene editing industry, hope came earlier this month in the tiny, smiling, fuzzy-headed form of KJ Muldoon.
At just 6 months old, KJ received a gene-editing treatment custom-built to correct his unique mutation. He’s not cured, researchers explained at the annual American Society of Gene & Cell Therapy meeting in New Orleans. But he has been able to resume a normal diet and is no longer on the path to a liver transplant.
The news could not have come at a more welcome or more jarring time for the field. For three years, gene editing has seemed in free fall, riven by layoffs, closures, shuttered programs, and sinking stock prices. Now here, finally, in striped pajama form, was a reminder of what a decade of advances could deliver. “How awesome is it that we’re at this point?” said Nessan Bermingham, who co-founded and led Intellia, one of the first CRISPR companies, until 2017.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.
Already have an account? Log in
