Intellia bolsters case for CRISPR-Cas9 durability, safety

Intellia Therapeutics Inc. (NASDAQ:NTLA) has reported that the efficacy of its in vivo CRISPR-Cas9 therapy for hereditary angioedema (HAE) persists up to three years, bolstering the case that gene editing can deliver on its curative promise.

The company said a single 25, 50 or 75 mg dose of lonvoguran ziclumeran (lonvo-z) led to a 98% mean reduction in HAE attack rates in 10 patients who were treated in a Phase I study and followed for a median of 23 months. The 10 patients had been free of HAE attacks for a median of 23 months at the latest follow-up...