From Yahoo Finance: 2025-06-23 01:41:00

Intellia Therapeutics presented positive 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. All 10 patients were free of attacks and treatment for a median of 23 months after receiving a single dose of lonvo-z.

Lonvo-z is an experimental in vivo CRISPR-based gene editing therapy that targets the KLKB1 gene to prevent HAE attacks. 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.

Intellia Therapeutics plans to launch in 2027 after submitting a BLA in 2026 for their Phase 3 HAELO trial. Lonvo-z has been designated as an Orphan Drug and RMAT by the FDA, and PRIME by the EMA. The therapy showed a 98% mean reduction in monthly HAE attacks with positive safety results maintained across all treatment dosages.

Read more: Intellia Reports Positive 3-Year Lonvo-z Data in HAE Trial at EAACI Congress