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Intellia Therapeutics, Inc. (NTLA) is a leading clinical-stage genome editing company focused on developing potentially curative therapies using CRISPR/Cas9 technology. The company operates in the rapidly growing gene editing market, targeting rare diseases, oncology, and autoimmune conditions. Intellia has established a strong position in the field through its proprietary non-viral delivery platforms, including lipid nanoparticles (LNPs) and adeno-associated virus (AAV) vectors, which enhance precision and scalability. Its most advanced candidates, NTLA-2001 for transthyretin (ATTR) amyloidosis and NTLA-2002 for hereditary angioedema (HAE), are in clinical trials, demonstrating early proof-of-concept in humans. The company collaborates with Regeneron, providing financial backing and validation of its platform.
CRISPR/Cas9 in vivo and ex vivo therapies; 100+ patents; first-ever clinical data showing systemic CRISPR editing in humans (NTLA-2001).
Intellia offers high-risk, high-reward exposure to CRISPR-based therapies, with a validated platform and strong partnerships offsetting near-term losses. The upcoming NTLA-2001 Phase 3 readout is a key inflection point, but dilution risk and competition warrant caution. Suitable for investors with a 5+ year horizon and high risk tolerance.
Intellia 2023 10-K, Q4 2023 investor presentation, Regeneron collaboration press releases, ClinicalTrials.gov (NCT05697861, NCT05120830).