2/22/2024

speaker
Operator
Conference Operator

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.

speaker
Ian Karp
Senior Vice President of Investor Relations and Corporate Communications

Thank you, Operator, and good morning, everyone. Welcome to Intelia Therapeutics' fourth quarter and full year 2023 earnings call. Earlier this morning, Intellia 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 would like to take a minute to remind listeners that during the 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 Lebwall, Chief Medical Officer, Laura Sepp-Lorenzino, Chief Scientific Officer, and Glenn Goddard, Chief Financial Officer. John will begin with an overview of recent business highlights. David will provide an update on our clinical pipeline progress. Laurel will review our R&D updates, and Glenn will review our financials before we open up the call for questions. With that, I'll now turn the call over to John, our Chief Executive Officer. Thank you, Ian.

speaker
John Leonard
Chief Executive Officer

Good morning, everyone, and thank you all for joining us today. 2023 was an outstanding year for Intelia, and that strong momentum has carried into 2024. With our two lead programs either in or approaching Phase III clinical development, We're now closer than ever to the first in vivo CRISPR-based therapies reaching the market. We've made critical advances for the field of genome editing and for patients suffering from ATTR amyloidosis, or HAE. Notably, our one-time treatments offering potentially unmatched clinical profiles would address significant patient unmet need in two large and rapidly growing commercial markets. Having clinically validated our in vivo CRISPR gene editing technology by inactivating genetic targets in the liver, we're now bringing forth the next wave of innovation. This new frontier will come in two dimensions, broadening what we can do and expanding where we can go, all of which will allow us to increase the number of diseases we can pursue. The genome editing revolution is only made possible by the unique properties of CRISPR-Cas2. Intellia's expertise with CRISPR-Cas9 is unsurpassed and serves as a foundation for the diverse set of editing tools that we've developed and continue to advance. Whether using base editing or DNA writing tools, each of these technologies rely on the specificity and versatility of the CRISPR system. With our wide range of editing and delivering capabilities, we can apply the best tool for each therapeutic application. This allows us to address diseases where there is a meaningful opportunity to improve the standard of care. In some cases, we may even be able to pursue diseases that would otherwise be considered untreatable, if not for the power of CRISPR. It is this expansive and modular platform, coupled with our strong balance sheet, that will allow us to achieve the three-year strategic priorities announced earlier this year. Our focus remains on both near-term clinical execution as well as value-creating platform innovation. With this as a backdrop, we expect the following by the end of this year. Two in vivo knockout programs in active phase three studies, two in vivo gene insertion programs in first in human studies, and five different tissues outside the liver with active research programs. And additionally, we expect to have six or more collaborations with at least a dozen potential drug products utilizing our technology and research and development. We're confident in our ability to deliver on these ambitious goals. We have a proven track record of success with regulators in advancing CRISPR-based therapies and clinical trials. We've consistently delivered on our commitments to the scientific, patient, and investment communities. And finally, we have a world-class team of drug developers who have pioneered some of the most innovative and commercially successful medicines in history. In summary, Intellia is the company with the most advanced and expansive in vivo and ex vivo pipeline in the industry. With a potential BLA submission in 2026, we're well positioned to bring forth the first-ever in vivo CRISPR-based therapy. I'll now hand the call over to our Chief Medical Officer, David Levwall, who will provide an update on our clinical programs. David?

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