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Clinical Trial Report

Gene Therapy Trial Report

Summary

HAELO: a Phase 3 Study to Evaluate NTLA-2002 in Participants with Hereditary Angioedema (HAE)

NCTID NCT06634420 (View at clinicaltrials.gov)
Description
Indication Hereditary Angioedema
Compound Name NTLA-2002
Sponsor Intellia Therapeutics
Funder Type Industry
Status
Recruiting
Enrollment Count 60

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 50 mg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase3
Submit Date 2024-10-07
Completion Date 2027-09
Last Update 2024-10-22

Participation Criteria

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

Locations

No.of Trial Sites 1
Locations United States

Regulatory Information

Has US IND True
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