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Gene Therapy Trial Report

Summary

NTLA-2002 in Adults With Hereditary Angioedema (HAE)


NCTID NCT05120830 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hereditary Angioedema
Disease Ontology Term DOID:14735
Compound Name NTLA-2002
Sponsor Intellia Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 37 (ACTUAL)
Results Posted Not Available

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 (pivotal dose)
Dose 3 75 mg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2021-11-03
Completion Date 🔄 2026-07
Last Update 🔄 2026-03-24

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Age \>18 years 2. Diagnosis of HAE Types I or II 3. Ability to provide evidence of HAE attacks to meet the screening requirement 4. Subjects must have access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks. 5. Adequate chemistry and hematology measures at screening 6. Subjects must agree not to participate in another interventional study for the duration of this trial. 7. Subjects must be capable of providing signed informed consent Exclusion Criteria: 1. Concurrent diagnosis of any other type of recurrent angioedema 2. Subjects who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component. 3. Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject. 4. Unwilling to comply with study procedures.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


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

Regulatory Information


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

Resources/Links