Intellia Therapeutics, Inc. (NASDAQ:NTLA) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. It presented encouraging three-year follow-up data from their Phase 1 trial of lonvoguran ziclumeran (lonvo-z) in patients with hereditary angioedema (HAE) at the EAACI Congress 2025.
A lab scientist peering into a microscope focused on gene editing technology.
All 10 patients were free of attacks and treatment for a median of 23 months after receiving a single dose of lonvo-z, which led to a 98% mean reduction in monthly HAE attacks. Positive safety results were maintained across all treatment dosages. The current Phase 3 HAELO trial has a majority of U.S. enrollment and has completed screening ahead of schedule. Intellia Therapeutics, Inc. (NASDAQ:NTLA) plans to launch in 2027 after submitting a BLA in 2026.
Lonvo-z (NTLA-2002) is an experimental in vivo CRISPR-based gene editing therapy that targets the KLKB1 gene to prevent HAE attacks. The FDA has designated it as an Orphan Drug and RMAT, while the EMA has designated it as PRIME, among other regulatory classifications.
Administered intravenously at doses ranging from 25 to 75 mg, the therapy demonstrated dose-dependent kallikrein protein decrease and long-term efficacy, with no major treatment-related side effects recorded.
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