Clinical Trial

Disease: Hereditary Angioedema, HAE (NCT05120830)

Disease info:

Hereditary Angioedema (HAE) is a rare, genetic disorder characterised by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body. Currently available treatment options include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. 
 

Frequency:
It is estimated that one in 50,000 people are affected by HAE.
Official title:
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Who:

Intellia Therapeutics

Sponsor:

Intellia Therapeutics

Partners:
Locations:

Auckland, New Zealand

Study start:
Nov. 15, 2021
Enrollment:
55
Gene editing method:
CRISPR-Cas9
Type of edit:
Knock-out
Gene:
Kallikrein B1 (KLKB1)
Delivery method:
Lipid Nano Particles (LNPs) - In-vivo
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Last updated: Nov. 26, 2021
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