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Intellia's in vivo CRISPR therapy first to succeed in Phase 3

An experimental treatment that uses CRISPR to edit genes directly inside the body has just succeeded in a Phase 3 trial — a first for the technology — putting it on track for a potential FDA approval in the first half of next year.

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An experimental treatment that uses CRISPR to edit genes directly inside the body has just succeeded in a Phase 3 trial — a first for the technology — putting it on track for a potential FDA approval in the first half of next year.

The therapy, made by Intellia Therapeutics, uses the molecular scissors of CRISPR to break a gene that contributes to disfiguring and dangerous swelling attacks in people with hereditary angioedema. The one-time infusion reduced those attacks by 87% compared to placebo, the company announced Monday morning.

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