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Intellia's Gene-Editing Candidate Shows Durability In ATTR Amyloidosis

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  • Intellia Therapeutics Inc (NASDAQ: NTLA) and Regeneron Pharmaceuticals Inc (NASDAQ: REGNannounced additional interim data from the Phase 1 study of NTLA-2001, in vivo genome editing candidate for transthyretin (ATTR) amyloidosis.

  • Intellia had said that at the highest dose level (1.0mg/kg), six patients had shown a 93% mean reduction in the toxic protein. In the new data, three patients reached the nine-month follow-up point and maintained that level of TTR reduction.

  • Related: Intellia Shares Updated Data On Gene Editing Durability In Rare Disease.

  • The mean reduction for the three patients at the second-highest dose (0.7mg/kg) was 86%. For the longest-tracked group, dose level two at 0.3mg/kg, the three patients had a mean reduction of 89%.

  • Headaches, infusion-related reactions, back pain, rash, and nausea were the most frequent adverse events across all four dose levels.

  • Eleven of the 15 patients reported a maximal adverse event of grade 1. The most serious adverse event, which Intellia said was “possibly related” to its treatment, was Grade 3 vomiting in a patient with a medical history of gastroparesis.

  • Price Action: NTLA shares are down 1.87% at $49.81, and REGN shares are down 0.39% at $605.00 during the market session on the last check Friday.

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