Intellia doses first patient with anti-HAE gene therapy; Sobi announces new R&D chief
Gene therapy biotech Intellia Therapeutics has dosed its first patient with genome editing candidate NTLA-2002, its drug candidate for hereditary angioedema, a rare genetic condition that causes swelling under the skin.
The drug candidate has been under development as a single-dose gene therapy to prevent HAE attacks by inactivating the target gene kallikrein B1 (KLKB1). That would, Intellia hopes, reduce plasma kallikrein activity and thus prevent HAE attacks.
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