Intellia Therapeutics Shares Are Gaining After Positive Data From Second CRISPR Candidate - Intellia Therapeutics (NASDAQ:NTLA)

Intellia Therapeutics Inc NTLA announced interim results from an ongoing Phase 1/2 study of NTLA-2002, its second in vivo genome editing candidate.
NTLA-2002 is a systemically administered CRISPR candidate being developed for hereditary angioedema (HAE) and is designed to knock out the KLKB1 gene in liver cells, thereby reducing the production of kallikrein protein.
The uncontrolled activity of kallikrein is responsible for the overproduction of bradykinin, which leads to fatal swelling attacks in HAE patients.
NTLA-2002 led to dose-dependent reductions in plasma kallikrein and achieved maximal reductions by week eight, with mean reductions of 65% and 92% in the 25 mg and 75 mg dose cohorts, respectively.
Furthermore, these reductions were sustained through at least 16 weeks in the 25 mg cohort and eight weeks in the 75 mg cohort, for which complete cohort biomarker data were available.
Treatment with a single dose of 25 mg of NTLA-2002 resulted in a mean reduction in HAE attacks of 91% throughout the 16-week observation period. Additionally, two of the three patients have not had a single HAE attack since treatment, and all three patients have been attack-free since week 10.
Patients in the 75 mg cohort have not completed the primary 16-week observation period.
NTLA-2002 was generally well-tolerated at both dose levels, and the majority of adverse events were mild in severity. The most frequent adverse events were infusion-related reactions, primarily mild and resolved within one day.
Price Action: NTLA shares are up 7.20% at $73.52 during the premarket session on the last check Friday.

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