NTLA-3001
Hereditary Alpha-1 Antitrypsin Deficiency (AATD)
Phase 1/2Active
Key Facts
Indication
Hereditary Alpha-1 Antitrypsin Deficiency (AATD)
Phase
Phase 1/2
Status
Active
Company
About Intellia Therapeutics
Intellia Therapeutics is a leader in translating Nobel Prize-winning CRISPR/Cas9 technology into transformative genomic medicines. The company's strategy leverages a dual-platform approach—in vivo and ex vivo editing—to advance a pipeline targeting rare genetic diseases like hereditary angioedema (HAE) and ATTR amyloidosis, with key programs in late-stage development. Backed by a strong intellectual property foundation and a strategic partnership with Regeneron, Intellia is building toward becoming a fully integrated, commercial-stage biopharmaceutical company.
View full company profile