Intellia Therapeutics Highlights its Three-Year Strategic Priorities and Anticipated 2024 Key Milestones - Seite 2

1. Complete patient enrollment for pivotal studies of NTLA-2001 and NTLA-2002, including planned biologics license application (BLA) submission for NTLA-2002 in 2026;
2. Launch clinical trials for next wave of in vivo and ex vivo programs, validating CRISPR-based in vivo targeted gene insertion and an allogeneic cell engineering solution designed to avoid NK cell-mediated rejection; and
3. Expand the range of diseases that can be targeted by deploying new editing and delivery innovations, including advancing gene editing in tissues outside the liver and DNA writing technology.
   

Anticipated 2024 Milestones

• NTLA-2001 for ATTR Amyloidosis:
  • Dose the first patient in MAGNITUDE trial of NTLA-2001 for the treatment of ATTR-CM in Q1 2024.
  • Continue to open new sites and enroll patients, globally, in the MAGNITUDE study.
  • Prepare for the Phase 3 study of NTLA-2001 for the treatment of ATTR amyloidosis with polyneuropathy (ATTRv-PN) in 2024.
  • Present updated clinical data from the ongoing Phase 1 study of NTLA-2001 in 2024.
• NTLA-2002 for HAE:
  • Initiate the global pivotal Phase 3 trial of NTLA-2002 in 2H 2024, subject to regulatory feedback.
  • Present updated data from Phase 1 and new data from Phase 2 portion in 2024.
• NTLA-3001 for AATD-Associated Lung Disease:
  • Dose first patient in Phase 1 study of NTLA-3001 in 2024.

Presentation at the 42^nd Annual J.P. Morgan Healthcare Conference

John Leonard, M.D., President and Chief Executive Officer of Intellia, will present a company overview at the 42^nd Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 9, at 5:15 p.m. PT (8:15 p.m. ET). A live webcast will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website, www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for a limited time following the conference.

About Intellia Therapeutics

Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.