NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830 ↗

Phase 1/2 Active not recruiting 37 enrolled Intellia Therapeutics

RandomizedSequentialDouble-blindTreatment

Summary

This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).

Timeline

Start: 2021-12-10
Primary completion: 2024-04-04
Completion: 2026-07

Outcome

Met primary endpoint

release “Phase 2 study of NTLA-2002 for hereditary angioedema (HAE) met its primary and all secondary endpoints;” intelliatx.com ↗

Publications

Cohn DM, Gurugama P, Magerl M, Katelaris CH, Launay D, Bouillet L, Petersen RS, Lindsay K, Aygoren-Pursun E, Maag D, Butler JS, Shah MY, Golden A, Xu Y, Abdelhady AM, Lebwohl D, Longhurst HJ. CRISPR-Based Therapy for Hereditary Angioedema. N Engl J Med. 2025 Jan 30;392(5):458-467. doi: 10.1056/NEJMoa2405734. Epub 2024 Oct 24.
Longhurst HJ, Lindsay K, Petersen RS, Fijen LM, Gurugama P, Maag D, Butler JS, Shah MY, Golden A, Xu Y, Boiselle C, Vogel JD, Abdelhady AM, Maitland ML, McKee MD, Seitzer J, Han BW, Soukamneuth S, Leonard J, Sepp-Lorenzino L, Clark ED, Lebwohl D, Cohn DM. CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema. N Engl J Med. 2024 Feb 1;390(5):432-441. doi: 10.1056/NEJMoa2309149.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	NTLA-2002	CRISPR / gene editing	25 mg	Intravenous
Subject	NTLA-2002	CRISPR / gene editing	50 mg	Intravenous
Subject	NTLA-2002	CRISPR / gene editing	75 mg	Intravenous