The FDA is unveiling a faster approval pathway for custom gene-editing therapies, aiming to spark major investment and accelerate cures for rare diseases through Crispr-based innovations.
Intellia Therapeutics (NTLA) shares drop 15% after FDA halts late-stage gene therapy trials following serious liver safety concerns in patient.
Intellia Therapeutics halts MAGNITUDE trials of nex-z gene therapy after patient hospitalization with Grade 4 liver toxicity. Over 650 patients enrolled in ATTR amyloidosis studies.
Intellia completed enrollment for lonvo-z, a gene-editing drug for severe swelling (HAE). It aims to be the first one-time treatment for HAE, a rare genetic disease. Top study data is expected in early 2026, with a potential U.S. launch in early 2027. Intellia's shares surged on the news.
