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# Intellia Rolling BLA for Hereditary Angioedema CRISPR Therapy
- URL: https://www.fdaweb.com/intellia-rolling-bla-for-hereditary-angioedema-crispr-therapy/
- Published: 2026-04-27T12:00:00.000Z
- Updated: 2026-09-14T13:38:07.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5161044

Intellia Therapeutics has begun a rolling BLA submission for its experimental gene-editing therapy lonvoguran ziclumeran, or lonvo-z, as a one-time treatment for hereditary angioedema (HAE). The company expects to complete submission in the second half of the year and, if approved, launch the therapy in the first half of 2027.

Lonvo-z is designed as an in vivo CRISPR/Cas9 gene-editing treatment administered in an outpatient setting. The therapy works by inactivating the KLKB1 gene, with the goal of permanently reducing kallikrein and bradykinin — key drivers of the painful and potentially life-threatening swelling attacks that characterize HAE, the company says.

The regulatory filing follows positive topline results from the Phase 3 HAELO trial, in which a single dose of lonvo-z met its primary and all key secondary endpoints. According to the company, most patients receiving the therapy were free from both HAE attacks and the need for ongoing treatment during the six-month primary observation period.

The company says it is leveraging FDA’s Regenerative Medicine Advanced Therapy designation, which allows for a rolling review of application components and is intended to accelerate development and evaluation of promising therapies. Intellia also participated in the agency’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot program, which facilitates earlier and more frequent interaction with regulators on manufacturing issues.

Hereditary angioedema affects an estimated 1 in 50,000 people and is typically managed with lifelong preventive or on-demand therapies, often requiring frequent intravenous, subcutaneous, or oral dosing, the company says.

If approved, lonvo-z could represent a shift away from chronic disease management toward a potentially curative, single-dose approach — advance that would also mark a milestone for in vivo CRISPR therapeutics, a field long viewed as promising but still emerging in late-stage clinical development.