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Intellia is Leading the Gene Editing Revolution Corporate Overview August 2024
Intellia is Leading the
Gene Editing Revolution
August 20242Intellia Therapeutics’ Legal Disclaimer
This presentation contains “forward -looking statements” of Intellia Therapeutics, Inc. (“Intellia”, “we” or “our”) within the meaning of the Private Securities Litigation Reform Act of
1995 . These forward -looking statements include, but are not limited to, express or implied statements about Intellia’s beliefs and expectations regarding : our ability to build a
world -class gene editing toolbox to develop an unsurpassed gene editing pipeline ; the safety, efficacy and advancement of our clinical programs for NTLA -2001 , now known as
nexiguran ziclumeran or “nex-z”, for the treatment of transthyretin (“ATTR”) amyloidosis, NTLA -2002 for the treatment of hereditary angioedema (“HAE”) and NTLA -3001 for the
treatment of alpha -1 antitrypsin deficiency (“AATD”) -associated lung disease pursuant to our clinical trial applications (“CTA”) and investigational new drug (“IND”) submissions,
including the expected timing of data releases, regulatory filings, and the initiation and completion of clinical trials, such as initiating the Phase 3 study for the treatment of ATTR
amyloidosis with polyneuropathy in 2024 , presenting updated data from the ongoing Phase 1 study of NTLA -2001 in 2024 , initiating the Phase 3 clinical trial for NTLA -2002 for
HAE in 2024 , presenting additional data from the Phase 2 portion of the Phase 1/2 study of NTLA -2002 in 2024 , and dosing the first patient in the Phase 1 study of NTLA -3001 in
2024 ; the execution of its strategic priorities for 2024 -2026 , including the completion of patient enrollment for pivotal studies of NTLA -2001 and NTLA -2002 , the planned BLA
submission for NTLA -2002 for HAE in 2026 , demonstrating human proof -of-concept for targeted in vivo gene insertion, initiating clinical development for its allogeneic ex vivo
program, demonstrating preclinical proof -of-concept of editing in tissues outside the liver, and advancing DNA writing technology ; the ability to generate data to initiate clinical
trials and the timing of CTA and IND submissions ; the advancement, expansion and acceleration of our CRISPR/Cas 9 technology and related technologies, including DNA
writing, base editing, manufacturing and delivery technologies, to advance and develop additional candidates and treatments ; our ability to demonstrate our platform’s modularity
and replicate or apply results achieved in preclinical studies, including those in its NTLA -2001 , NTLA -2002 and NTLA -3001 programs, in any future studies, including human
clinical trials ; our ability to optimize the impact of our collaborations on our development programs, including, but not limited to, collaborations with Regeneron Pharmaceuticals,
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