Guess the Therapy Answer May 15, 2026

Intellia Therapeutics recently submitted a rolling BLA to the FDA for which CRISPR-based gene editing therapy for hereditary angioedema?

Answer: lonvoguran ziclumeran (lonvo-z, formerly known as NTLA-2002)

See below for further reading on lonvo-z:

Lonvo-z Advances Toward Approval as First In Vivo CRISPR Therapy for Hereditary Angioedema

April 27, 2026 — Intellia Therapeutics has initiated a rolling biologics license application (BLA) submission to the FDA for lonvoguran ziclumeran (lonvo-z), an investigational one-time CRISPR-based gene editing therapy for hereditary angioedema (HAE), following phase 3 data showing substantial reductions in attack rates and treatment burden.

The application is supported by topline results from the global phase 3 HAELO trial, which met its primary and all key secondary endpoints, marking what could become the first regulatory submission for an in vivo CRISPR-based therapy.

“If approved, lonvo-z will become the world’s first in vivo CRISPR-based gene editing therapy,” said John Leonard, MD, president and chief executive officer of Intellia, in a statement.

First Patient Dosed in Phase 3 Trial for Intellia Therapeutics’ Hereditary Angioedema Gene Editing Therapy NTLA-2002

January 23, 2025 — The first patient has been dosed in Intellia Therapeutics’ pivotal phase 3 HAELO clinical trial (NCT06634420) evaluating NTLA-2002, an investigational CRISPR/Cas9-based gene-editing therapy that is delivered systemically as a single-dose, for the treatment of HAE.

HAELO takes the form of a double-blind, placebo-controlled study, and will randomly assign participants in a 2:1 ratio to receive either a 50mg dose of NTLA-2002 or placebo. The trial is expected to enroll 60 patients in total, and includes adults with both type 1 and type 2 HAE. Participants who are assigned to the placebo group may cross over to receive the gene-editing therapy at 28 weeks posttreatment. The number of HAE attacks and the number of patients who are attack-free from weeks 5 through 28 posttreatment will serve as key end points for the study. HAELO is currently recruiting patients at sites in Arizona, California, Colorado, and Ohio in the United States and at a site in Ontario, Canada. Intellia anticipates that enrollment in the study will be finished in the second half of this year.