Intellia Therapeutics, Inc.

Good morning and welcome to Intellia Therapeutics' first quarter 2024 financial results conference call. My name is Drew and I will be your conference operator today. Following formal remarks, we will open the call up for a question and answer session. This conference is being recorded at the company's request and will be available on the company's website following the end of the call. As a reminder, all participants are currently in listen-only mode. If anyone requires operator assistance during the conference, please press star zero on your telephone keypad. I will now turn the conference 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 2024 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 Intellia's website at intelliatx.com. This call is being broadcast live, and a replay will be archived on the company's website. At this time, I'd like to take a minute to remind listeners that during this call, Intellia 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 Intelia are John Leonard, Chief Executive Officer, David Levwall, Chief Medical Officer, Laura Lorenzino, Chief Scientific Officer, and Glenn Godden, Chief Financial Officer. John will begin with an overview of recent business highlights. David will provide an update on our clinical pipeline progress. Laura will review our R&D updates. And Glenn will review our financials before we open up the call for your questions. With that, I'll now turn the call over to John, our Chief Executive Officer.

Thank you, Ian. Good morning, everyone, and thank you all for joining us today. We've made outstanding progress in the first quarter of 2024, with one ongoing and two soon-to-be-initiated pivotal phase three trials. Intelia is undoubtedly leading the gene editing revolution. and with well over 100 patients already dosed across our two lead programs, we have already amassed the largest set of safety and clinical activity data for any in vivo CRISPR-based therapies. Notably, we believe our one-time treatments for ATTR and HAE offering potentially unmatched clinical profiles could overcome key hurdles faced by patients in two large and rapidly growing commercial markets. Moreover, our first two investigational gene editing therapies are designed to be especially patient-friendly and convenient for physicians and caregivers. There's no extensive preconditioning regimen, no long-term steroid requirement, and no hospital stay, all of which can be very challenging for patients and limit commercial uptake associated with other gene therapies. And of course, with a one-time treatment, there's no annual insurance re-verification needed, which is typically required for chronic specialty therapies and can be a tremendous burden But this is just the beginning for industry-leading CRISPR-based technology. We're now entering the next stage of growth, pushing the boundaries of what we can do and expanding where we can go with CRISPR, from gene knockdown to gene insertion, from liver targets to a broader set of tissues. Our first wholly-owned CRISPR-based gene insertion program, NCLA-3001, is expected to enter human clinical development this year. NTLA-3001 holds the potential to provide normal alpha-1 levels after single-dose treatment for people with alpha-1 antitrypsin deficiency. We will also have a second clinical program utilizing our modular gene insertion platform run by Regeneron for hemophilia B expected to start patient dosing later this year. Building on our clinical success, we are now pursuing gene editing in five new tissue types outside the liver. As part of this expansion strategy, we have and will continue to establish collaborations with external innovators. These R&D efforts have already yielded at least a dozen potential drug candidates utilizing our technology. Further, we are advancing our modular platform by developing a diverse set of editing and delivery tools for in vivo and ex vivo applications. Whether it's our proprietary LMP formulations, novel gene editing tools, or differentiated allogeneic cell therapy approach, We emphasize safety and therapeutic activity at each step of development. Through our commitment to these principles, we are well on the path towards transforming cutting-edge scientific tools into real-world medical treatments. With this as a backdrop, we expect to end this year with five enrolling clinical studies, three of which are in Phase III. This includes a newly planned Phase III for NTLA-2001 in patients with ATTR polyneuropathy. Additionally, we plan to submit a BLA submission in 2026 for NTLA-2002, which we anticipate will lead to the first-ever approval for an in vivo CRISPR-based therapy. By that time, our expectation is we'll have accumulated safety, efficacy, and durability data for over seven years and have treated as many as 1,000 patients in our clinical studies. In summary, we will continue transforming medicine with gene editing therapies with at least three important clinical data readouts expected this year. I'll now hand the call over to our Chief Medical Officer, David Lebwal, who will provide an update on our clinical programs. David?