Expanded Intellia CRISPR data set up showdown with Pfizer, Ionis and Alnylam in not-so-rare disease
Eight months after delivering landmark results with one of the first CRISPR clinical trials of its kind, Intellia announced the experimental therapy held up in a larger group of patients and over a longer period of time.
In the initial study, Intellia showed a single infusion of its gene-editing therapy could lower the amount of toxic protein six patients with a rare and fatal genetic disease produce by an average of 52% or 87%, depending on the dose. It was the second trial to use CRISPR directly in patients, and the first to deliver data, clearing a major test for the Nobel-winning technology.
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