Intellia Therapeutics, Inc. (NASDAQ:NTLA) Barclays 27th Annual Global Healthcare Conference March 11, 2025 3:00 PM ET
Company Participants
John Leonard – President and CEO
Conference Call Participants
Gena Wang – Barclays
Gena Wang
Good afternoon, everyone. My name is Gena Wang. I’m SMID cap biotech analyst at the Barclays. It is my great pleasure to introduce our next presenting company, Intellia Therapeutics. On the stage with me is John Leonard, the President and also Chief Executive Officer. John, maybe before we dive into the questions, could you maybe give a quick overview of Intellia?
John Leonard
Sure. Intellia is a CRISPR based gene editing company. We’re now in our 11th year, one of the three original pioneers in the space. We’ve focused primarily on in vivo based therapies where we’ve pioneered some of the work with lipid nanoparticles. Now we have three different programs in Phase 3 for two different targets. I’m sure we’ll be talking about that. And then continue to build the technology base to pursue other targets within the liver and outside the liver as well as a circumscribed ex vivo program.
Question-and-Answer Session
Q – Gena Wang
Okay, great. So maybe I will start with your ATTR program. I know this is already moving very fast to Phase 3. So regarding the Phase 3 trial design, I’m pretty sure this is one of the key questions given we have so many other Phase 3 trials ongoing and we have a readout. So maybe regarding the Phase 3 trial design in the ATTR cardiomyopathy. Now what is the latest thoughts regarding the trial design? And I will ask a specific question.
John Leonard
Sure. I mean, just the basic design is a placebo controlled randomized study. Patients are randomized two to one drug to placebo on top of standard of
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