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NTLA-2002

Phase 3

Hereditary Angioedema | Monoclonal antibody | Immunology |Intellia Therapeutics, Inc.|Last Updated: Jul 29, 2026

Success Probability
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Market & Valuation
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment80
FDA Designations
RMATORPHAN_DRUG
Clinical trial landscape

NTLA-2002 · 1 trial · 1 indication

Phase 3 1
NCT06634420HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)Hereditary Angioedema
ACTIVE NOT_RECRUITING80 Analytics
PHASE3ACTIVE NOT_RECRUITING
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema
NCT0663442080
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Study Endpoints
Primary Endpoints
Time-normalized number of Investigator-confirmed HAE attacks
From Week 5 through Week 28
Secondary Endpoints
Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment
From Week 5 through Week 28
Time-normalized number of moderate or severe Investigator-confirmed HAE attacks
From Week 5 through Week 28
Investigator-confirmed HAE attack-free status
From Week 5 through Week 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Arm A: NTLA-2002ACTIVE_COMPARATORArm A: NTLA-2002 (50 mg; single IV infusion)
Arm B: PlaceboPLACEBO_COMPARATORArm B: Placebo (saline; single IV infusion)
Interventions
NameTypeDescription
NTLA-2002BIOLOGICALCRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
Normal Saline IV AdministrationBIOLOGICALThe administration of intravenous (IV) normal saline
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Eligibility Criteria
Age Range16 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites29

Inclusion Criteria: 1. Age ≥16 years 2. Clinical history consistent with HAE-C1INH-Type 1 or -Type 2 3. Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement 4. Must agree to refrain from the use of long-term prophylactic therapies from the sta...

Countries:United StatesAustraliaCanadaFranceGermanyNetherlandsNew ZealandSouth AfricaUnited Kingdom
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Competitive Landscape -Hereditary Angioedema 9 trials
CompanyTickerTrialsLead PhaseDrugs
Intellia Therapeutics, Inc.NTLA3PHASE3NTLA-2002, Normal Saline Administration
BioCryst Pharmaceuticals, Inc.BCRX2PHASE3Berotralstat
Ionis Pharmaceuticals, Inc.IONS1PHASE3Donidalorsen
Pharvaris N.V.PHVS1PHASE2deucrictibant
BioMarin Pharmaceutical Inc.BMRN1PHASE1Dose 1 of BMN 331
Astria Therapeutics, Inc.ATXS1PHASE2STAR-0215
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Recent Changes (Last 90 Days)
LOWJul 29, 2026NCT06634420lastUpdatePostDate: changed
LOWJul 29, 2026NCT06634420lastUpdatePostDate: changed
LOWMay 26, 2026NCT06634420Enrollment: 60 → 80
LOWMay 24, 2026NCT06634420studyFirstPostDate: changed