First CRISPR Gene Therapy Reduces Seizures by 87% in Phase 3, Targeting Commercial Launch in 2027

Intellia Therapeutics, Inc. (NTLA) announced that in its global Phase 3 HAELO trial in hereditary angioedema (HAE) patients, a single intravenous dose of the candidate lonvo-z reduced attack rates by 87% versus placebo, and 62% of patients experienced no attacks during the observation period without any long-term prophylactic therapy. The company reported a favorable safety profile, with no serious adverse events or grade 3 or higher events. Intellia outlined a regulatory and commercialization strategy targeting completion of sequential BLA submissions by late 2026 and a U.S. launch in the first half of 2027. As of Q1 2026, its cash, cash equivalents and short-term investments totaled $517.2 million—approximately KRW 720 billion—including roughly $207 million (about KRW 290 billion) raised in an April follow-on offering, securing funding into at least 2028.

In the same quarter, the company reported partnership revenue of $15 million, R&D expenses of $80.7 million, SG&A expenses of $34.8 million and a net loss of $96.2 million. Intellia also announced that the FDA has lifted the clinical hold on the MAGNITUDE and MAGNITUDE-2 Phase 3 trials for its transthyretin amyloidosis (ATTR) candidate nex-z, which had been paused due to liver toxicity concerns late last year, allowing patient enrollment and dosing to resume.

Intellia first released global top-line data from the HAELO Phase 3 on April 27, emphasizing it as the first Phase 3 results for an in vivo CRISPR gene-editing therapy. On the same day, the company separately announced the start of its sequential BLA submissions for lonvo-z and outlined plans to expand its commercialization infrastructure across medical affairs, sales, distribution and pricing.

Headquartered in Massachusetts, Intellia Therapeutics is a clinical-stage biotech company developing in vivo CRISPR-based gene-editing therapies designed to directly target and cut disease-driving genes. Focusing on serious rare diseases such as hereditary angioedema and transthyretin amyloidosis, the company is considered a leading player in the next-generation gene-editing therapy market, with single-dose treatments aimed at delivering long-lasting effects.