Intellia Therapeutics, Inc. (NTLA), a gene editing company, announced on Wednesday that the first patient has been dosed in its pivotal Phase 3 HAELO clinical trial of NTLA-2002, an investigational CRISPR-based gene therapy for hereditary angioedema or HAE.
The HAELO study aims to evaluate the efficacy and safety of NTLA-2002, a potential one-time treatment designed to prevent HAE attacks by inactivating the kallikrein B1 gene.
The trial will enroll 60 adults with Type I or Type II HAE, and patients will receive a single 50 mg infusion of NTLA-2002 or placebo.
The key endpoints of the study include the number of HAE attacks and the proportion of patients achieving attack-free status from week 5 through week 28.
Intellia expects to complete enrollment by the second half of 2025 and plans to submit a biologics license application or BLA in 2026, with a U.S. launch anticipated in 2027.
The initiation of the HAELO study marks a significant milestone in Intellia's development of NTLA-2002, which aims to provide long-term relief for patients suffering from this debilitating and life-threatening condition.
Cash Position:
The company had a cash, cash equivalents, and marketable securities were $944.7 million as of September 30, 2024.
NTLA is currently trading at $10.09, up 1.51%.
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