NTLA-2001
- Generic Name
- NTLA-2001
- Drug Type
- Biotech
- Background
NTLA-2001 is a clustered regularly interspaced short palindromic repeats CRISPR/ Cas9-based gene therapy consisting of a single guide RNA targeting the human TTR gene and a messenger RNA encoding Cas9.
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Phase 3
Recruiting
- Conditions
- Neuromuscular DiseaseNeuromuscular Diseases (NMD)Neurodegenerative DiseaseNeurodegenerative Disease, HereditaryNeurodegenerative DiseasesNeuromuscular DiseasesNerve DisordersNervous System DiseaseNervous System DiseasesGenetic Disease, Inborn
- Interventions
- Drug: Normal Saline as Placebo
- First Posted Date
- 2024-11-04
- Last Posted Date
- 2025-05-06
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 50
- Registration Number
- NCT06672237
- Locations
- 🇦🇷
Hospital Británico de Buenos Aires, Buenos Aires, Argentina
🇦🇺Westmead Hospital, Westmead, Australia
🇧🇷HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto, Ribeirao Preto, Brazil
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Phase 3
Recruiting
- Conditions
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
- Interventions
- Drug: Placebo
- First Posted Date
- 2023-11-13
- Last Posted Date
- 2025-05-13
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 765
- Registration Number
- NCT06128629
- Locations
- 🇫🇷
AP-HP Hopital Henri Mondor, Créteil, France
🇫🇷AP-HM - Hopital de la Timone, Marseille, France
🇺🇸Houston Methodist Hospital, Houston, Texas, United States
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
Phase 1
Active, not recruiting
- Conditions
- Wild-Type Transthyretin Cardiac AmyloidosisTransthyretin-Related (ATTR) Familial Amyloid PolyneuropathyTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
- Interventions
- First Posted Date
- 2020-10-23
- Last Posted Date
- 2025-05-01
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 72
- Registration Number
- NCT04601051
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
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