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Lonvo Z

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In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE
biotech2 months ago

In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE

Intellia Therapeutics’ Phase 3 study of lonvo-z, its in vivo CRISPR-based treatment for hereditary angioedema, showed an 87% reduction in attack rates versus placebo after a single dose, with over 60% of patients attack-free, prompting an FDA rolling submission and positioning lonvo-z as the second approved CRISPR medicine and the first in vivo gene-editing therapy.

technology2 months ago

Intellia’s One-Time CRISPR Therapy Delivers Phase 3 Win for Hereditary Angioedema

Intellia Therapeutics reported positive topline Phase 3 HAELO results for lonvo-z (NTLA-2002), a one-time in vivo CRISPR therapy for hereditary angioedema that achieved its primary endpoint with an 87% reduction in attacks over six months, 62% attack- and therapy-free, and favorable safety; the company has initiated a rolling FDA Biologics License Application with a potential U.S. launch in the first half of 2027, marking a global first for in vivo gene editing.