On April 27, 2026, Intellia Therapeutics (“Intellia”) announced that it has begun a rolling submission of a biologics license application (BLA) to the FDA for lonvoguran ziclumeran (lonvo-z), an investigational therapy for hereditary angioedema (HAE). Lonvo-z, previously known as NTLA-2002, is designed as a one-time, outpatient-administered treatment using in vivo CRISPR-based gene editing technology. The candidate therapy targets the KLKB1 gene, with the aim of reducing kallikrein and bradykinin levels, which are implicated in HAE attacks. If approved, lonvo-z could represent the first in vivo CRISPR-based gene editing therapy to reach the market.
In parallel with the regulatory update, Intellia reported positive topline results from its Phase 3 HAELO clinical trial. According to the company, the study met its primary and key secondary endpoints. Most patients receiving a single dose of lonvo-z experienced freedom from HAE attacks and discontinued ongoing therapy during the six-month primary observation period.
The BLA submission is being conducted under a rolling review framework enabled by the therapy’s Regenerative Medicine Advanced Therapy (RMAT) designation. This pathway permits sponsors to submit completed sections of an application incrementally, allowing for earlier engagement with the FDA. Intellia also participated in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot, which facilitates ongoing dialogue with the agency regarding manufacturing and development considerations.
The company expects to complete the BLA in the second half of 2026. If accepted, the FDA will determine review priority and assign a target action date. A potential launch is anticipated in the first half of 2027, subject to approval.
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