This Week’s CGT News: CRISPR Hits Phase 3 Milestone - Summary - MDSpire
Clinical Guidelines

This Week’s CGT News: CRISPR Hits Phase 3 Milestone

Intellia’s one-time HAE therapy delivers strong results as Cabaletta strikes CAR-T manufacturing deal

  • May 4, 2026

  • 7 min

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Intellia Therapeutics has achieved significant results in a Phase 3 trial for its CRISPR gene editing therapy, lonvoguran ziclumeran (lonvo-z), aimed at treating hereditary angioedema (HAE). This therapy reduced HAE attacks by 87% and allowed 62% of patients to be attack-free without ongoing medication over a six-month period. The therapy functions by silencing the KLKB1 gene, thus providing a durable solution against the disease. With favorable safety results, a biologics license application to the FDA is imminent, targeting a 2027 market launch.

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This Week’s CGT News: CRISPR Hits Phase 3 Milestone

  • May 4, 2026

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