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Intellia Shares Updated Data On Gene Editing Durability In Rare Disease

Eight months after delivering promising biomarker data from one of the first CRISPR trials, Intellia Therapeutics Inc (NASDAQ:NTLA) presented additional data from the Phase 1 trial for NTLA-2001 in transthyretin, or ATTR, amyloidosis.

  • In the initial study, Intellia showed that a single infusion of its gene-editing therapy could lower the amount of toxic protein in six patients by an average of 52% or 87%, depending on the dose
  • The new results showed that a slightly higher dose produced similar results. An even higher one could reduce protein expression by an average of 93% across six patients, with minimal side effects. 
  • The most common adverse events across 15 patients in the study were mild symptoms such as rash and nausea. 
  • One dose-limiting toxicity was observed in a patient who experienced vomiting, rated as a grade 3 adverse event.
  • Intellia confirmed that the reduction in serum levels of the transthyretin (TTR) was maintained in patients after follow-up ranging from two months to 12 months post-treatment.
  • Intellia leads the development and commercialization of NTLA-2001 as part of a collaboration with Regeneron Pharmaceuticals Inc (NASDAQ:REGN).
  • Price Action: NTLA shares traded 8.32% lower at $90.63 during pre-market trading on Tuesday.