In a medical first, an infusion of the CRISPR gene editor into the blood of three people with a rare genetic disease is easing their symptoms.
, the company hasn’t yet revealed whether the patients’ symptoms improved. For the trial in hereditary angioedema, however, the benefits emerged quickly, Intellia reports.
Drugs can help prevent these attacks by blocking a protein, kallikrein, that ramps up bradykinin levels—this essentially counteracts the effects of the loss of C1-esterase inhibitor. But CRISPR could allow patients to avoid lifelong use of those drugs by permanently knocking out the gene for kallikrein. Intellia took on that challenge by pairing CRISPR’s DNA-cutting enzymes with a strand of RNA that guides them to the gene.
In the hereditary angioedema trial, three patients who received a low dose of the CRISPR treatment saw their kallikrein blood levels drop by 65% on average at 8 weeks, clinical immunologist Hilary Longhurst reported at the 2022 Bradykinin Symposium. Longhurst, of the University of Auckland, leads the New Zealand arm of the study. Two patients who were having one to three swelling attacks per month have had none since the treatment.
Kallikrein levels dropped even more, by 91%, in three patients treated more recently with a higher dose of CRISPR nanoparticles. The results suggest the company’s early success with in vivo CRISPR is “not a fluke; it’s reproducible,” Leonard says.
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