LL schreef op 16 september 2022 14:54:
Intellia Therapeutics Announces Positive Interim Clinical Data for its Second Systemically Delivered Investigational CRISPR Candidate, NTLA-2002 for the Treatment of Hereditary Angioedema (HAE)September 16, 2022 at 7:00 AM EDT
Positive interim clinical data further validate the modularity of Intellia’s industry-leading genome editing platform and its potential to target a multitude of genetic diseases
A single dose of NTLA-2002 led to a 65% and 92% mean plasma kallikrein reduction at 25 mg and 75 mg doses, respectively, at week eight
HAE attacks were reduced by 91% in the 25 mg dose cohort through week 16; two of three patients remain attack free since treatment with third patient attack free since week 10 through latest follow-up
NTLA-2002 was generally well-tolerated at both dose levels
Intellia plans to initiate the Phase 2 dose-expansion portion of the study in 1H 2023
Intellia to host investor event today, Friday, September 16, at 8:00 a.m. ET
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Intellia Therapeutics Investor Event and Webcast Information
Intellia will host a live webcast today, Friday, September 16, 2022, at 8:00 a.m. ET, to provide a clinical update from its in vivo portfolio, during which the company will review the presented clinical data at the 2022 Bradykinin Symposium alongside interim results from NTLA-2001. To join the webcast, please visit this link, or the Events and Presentations page of the Investors & Media section of the company’s website at
www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for at least 30 days following the call.