Intellia Therapeutics moves HAE treatment closer to approval

Intellia Therapeutics moves HAE treatment closer to approval

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Key Points:

  • Intellia Therapeutics reported that a single dose of its gene-editing treatment, lonvo-z, significantly reduced swelling attacks in patients with hereditary angioedema (HAE) during a Phase 3 trial.
  • The therapy demonstrated an 87% reduction in attack rates compared to placebo, with over 60% of treated patients remaining attack-free, versus 11% in the placebo group.
  • Lonvo-z, if approved, would be the second CRISPR-based medicine on the market and the first in vivo treatment that edits DNA directly within the patient's body.
  • Intellia has already begun a rolling submission with regulatory agencies, moving closer to potential approval of this novel gene-editing therapy.

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