“We’re off to a very strong start in 2024 as we execute against our strategic priorities to realize the full potential of CRISPR-based gene editing,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are focused on rapidly enrolling patients in the pivotal Phase 3 MAGNITUDE trial of NTLA-2001 for the treatment of ATTR amyloidosis with cardiomyopathy and expect to dose the first patient in the first quarter of this year. We also remain on track to begin the Phase 3 trial for our second in vivo CRISPR-based therapy, NTLA-2002 for hereditary angioedema, later in the year. At the same time, we continue to expand both the technical approaches for CRISPR-based therapies, as well as the range of diseases they can potentially address. We plan to initiate two first-in-human studies for product candidates leveraging our modular gene insertion platform to produce a deficient protein – one wholly owned program focused on alpha-1 antitrypsin deficiency-associated lung disease and, together with Regeneron, a second program focused on hemophilia B. Finally, we are progressing our editing capabilities, including DNA writing, and applying them to diseases that originate outside of the liver. We announced last week a new collaboration with ReCode Therapeutics to advance novel gene editing treatments directly to the lung in patients with cystic fibrosis. These pipeline and platform efforts move us closer to setting a new standard of care for people living with serious diseases and expanding Intellia’s impact as the leading gene editing company.”
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