Intellia Therapeutics, Inc.

Good morning. My name is Drew, and I will be your conference operator today. And welcome to the Intellia Therapeutics first quarter 2022 earnings conference call. At this time, all participants are in a listen-only mode. Following the formal remarks, we will open the call up for your questions. Please be advised that this call is being recorded at the company's request. If you require operator assistance, press star then zero. At this time, I would like to turn it over to Ian Karp, Senior Vice President of Investor Relations and Corporate Communications at Intelia. Please proceed.

Thank you, Operator, and good morning, everyone. Welcome to Intelia Therapeutics' first quarter 2022 earnings call. Earlier this morning, Intelia issued a press release outlining the company's progress this quarter, as well as topics for discussion on today's call. This release can be found on the Investors and Media section of of Intelia's website at inteliatx.com. This call is being broadcast live and a replay will be archived on the company's website. At this time, I would like to take a minute to remind listeners that during this call, Intelia management may make certain forward-looking statements and ask that you refer to our SEC filings available at sec.gov for discussion of potential risks and uncertainties. All information presented on this call is current as of today. and Intelia undertakes no duty to update this information unless required by law. Joining me from the Intelia side are Dr. John Leonard, Chief Executive Officer, Dr. David Levwall, Chief Medical Officer, Dr. Laura Sepp-Lorenzino, Chief Scientific Officer, and Glenn Goddard, Chief Financial Officer. John will begin with an overview of recent business highlights. David will recap the recent update from our first in human study of NTLA-2001, as well as progress across our clinical program. Laura will then recap the company's R&D progress, and Glenn will review Intelia's financial results for the first quarter. John will then offer some concluding remarks before we open the call up for Q&A. With that, I'll turn the call over to our CEO, John.

Thank you, Ian, and thank you all for joining us this morning. At Intelia, we're building a full-spectrum genome editing company. We're deploying the industry's broadest and deepest toolbox, including novel editing and delivery solutions, to harness the immense power of CRISPR-based technologies for in vivo and ex vivo therapeutic applications, each with the potential to revolutionize the future of medicine. At the beginning of this year, we laid out three core priorities, accelerating the clinical validation of our in vivo pipeline, expanding our pipeline, and building on our scientific leadership by driving forward key platform innovations. We're making excellent progress against these objectives. During the first quarter, we shared updated interim data from our landmark study of NTLA-2001. These results demonstrated that treatment was generally well-tolerated and delivered rapid, consistent dose-dependent reductions in serum TTR in people with hereditary ATTR amyloidosis with polyneuropathy. Among the three patients in the 0.7 milligrams per kilogram dose group, Treatment with NTLA-2001 led to a mean serum TTR reduction of 86% by day 28. In six patients treated with a single one make per gig dose, a 93% mean and 98% maximum reduction was achieved by day 28. Further, the reduction of TTR levels has been sustained through the observation period, which provides the first evidence that a single dose CRISPR investigational therapy may provide a lifelong effect. David will review these data in greater detail, including selection of the fixed dose to be evaluated in the expansion portion of the polyneuropathy arm. We plan to present additional data from the polyneuropathy arm of the study at the upcoming EASL International Liver Congress being held in June. In addition, we hope to share the first interim data from the cardiomyopathy arm later this year. These updated data not only bolster confidence in NTLA-2001 as a potential treatment for ATTR amyloidosis, but also reinforce our belief in the power of our platform for in vivo gene editing. Notably, we look forward to sharing the initial clinical data from our second in vivo program, NTLA-2002, for HAE later this year. Switching to our ex vivo pipeline, in March, we dosed the first patient in our clinical study of NTLA-5001 for the treatment of AML. Altogether, the modular nature of our platform is on full display as we've advanced and expanded our pipeline considerably, in both the in vivo and ex vivo settings, and initiated multiple strategic business development collaborations that extend the reach of our technology beyond our core focus. Finally, earlier this week, we were pleased to welcome Muna Banji to our board of directors. She brings over three decades of experience in commercial strategy and market access. Her history of successfully navigating global healthcare systems to improve patient access to innovative medicines will be invaluable to Intelia. In addition, her appointment is an important step forward as we believe that it's essential that our board members represent diverse backgrounds and have experiences and skills in areas that are most relevant to the company's strategic plans. With that introduction, I'll hand the call over to our Chief Medical Officer, David Lebwall, who will review the NCLA 21 data in greater detail and detail progress across our pipeline. David?