NTLA-2002
- Generic Name
- NTLA-2002
- Drug Type
- Biotech
- Background
NTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9. It is an investigational therapy designed to knock out the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Phase 3
Recruiting
- Conditions
- Hereditary Angioedema
- Interventions
- Biological: Normal Saline IV Administration
- First Posted Date
- 2024-10-09
- Last Posted Date
- 2025-05-02
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 60
- Registration Number
- NCT06634420
- Locations
- 🇫🇷
CHU de Lille - Hopital Claude Huriez, Lille, France
🇫🇷AP-HM - Hopital de la Timone, Marseille, France
🇩🇪Charite Universitaetsmedizin Berlin, Berlin, Germany
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Phase 1
Active, not recruiting
- Conditions
- Hereditary Angioedema
- Interventions
- Other: Normal Saline IV Administration
- First Posted Date
- 2021-11-15
- Last Posted Date
- 2024-07-11
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 37
- Registration Number
- NCT05120830
- Locations
- 🇬🇧
Clinical Trial Site, Cambridge, United Kingdom
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