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Clinical Trial Report

Gene Therapy Trial Report

Summary

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCTID NCT05120830 (View at clinicaltrials.gov)
Description
Indication Hereditary Angioedema
Compound Name NTLA-2002
Sponsor Intellia Therapeutics
Funder Type Industry
Status
Active not recruiting
Enrollment Count 37

Therapy Information

Target Gene/Variant KLKB1
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Intravenous
Drug Product Type MRNA, LNP
Target Tissue/Cell Hepatocyte
Delivery System Lipid encapsulation
Vector Type LDLR
Editor Type Cas9 mRNA
Dose 1 25 mg
Dose 2 50 mg
Dose 3 75 mg
Dose 4 50 mg (pivotal dose)
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2021-11-03
Completion Date 2026-03-31
Last Update 2024-09-19

Participation Criteria

Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations

No.of Trial Sites 9
Locations New Zealand,Netherlands,United Kingdom,Australia,France,Germany

Regulatory Information

Has US IND False
Recent Updates BLA submission planned 2026; Phase II study met all primary and secondary endpoints, selected pivotal dose

Resources/Links

Patents
  • US20230203480A1
  • US20240018496A1
  • WO2024011206A1
Clinical Publications
News and Press Releases
Protocol