Aiming to make the impossible, possible
Our
Mission
Transforming lives of people with severe diseases by developing potentially curative genome editing treatments.
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Positive Results from the Phase 2 Study of NTLA-2002
NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy for hereditary angioedema. Data was published in The New England Journal of Medicine and presented at the 2024 ACAAI Scientific Meeting.
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PATIENT STORIES
Milton
Living with ATTR Amyloidosis with Cardiomyopathy
PATIENT STORIES
Nancy
Living with ATTR Amyloidosis with Polyneuropathy
PATIENT STORIES
Shanna, Oren and Damian
Living with Hereditary Angioedema Type 1
Our
