Intellia Therapeutics

🇺🇸United States

Country: 🇺🇸United States

Ownership: Public

Established: 2014-01-01

Employees: 526

Market Cap: $2.3B

Website: http://www.intelliatx.com

Introduction: Intellia Therapeutics, Inc. is a clinical stage genome editing company, which engages in the development of curative therapeutics using the CRISPR/Cas9 system. Its CRISPR/Cas9 system transforms medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creates engineered cells that can treat oncological and immunological diseases. The company was founded by Andrew May, Luciano Marraffini, Rodolphe Barrangou, Nessan Bermingham, Rachel Haurwitz, Erik Sontheimer, Jennifer Doudna, and Derrick Rossi in May 2014 and is headquartered in Cambridge, MA.

Clinical Trials

A Phase 3 Study of NTLA-2001 in ATTRv-PN

Phase 3

Recruiting

Conditions: Neuromuscular Disease
Neuromuscular Diseases (NMD)
Neurodegenerative Disease
Neurodegenerative Disease, Hereditary
Neurodegenerative Diseases
Neuromuscular Diseases
Nerve Disorders
Nervous System Disease
Nervous System Diseases
Genetic Disease, Inborn

Interventions: Biological: nexiguran ziclumeran
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

and more 6 locations

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
Biological: NTLA-2002

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

and more 27 locations

NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease

Phase 1

Withdrawn

Conditions: Lung Disease
Pulmonary Disease
AATD
Alpha-1 Antitrypsin Deficiency
Alpha-1 Antitrypsin Deficiency-associated Lung Disease

Interventions: Biological: Biological NTLA-3001

First Posted Date: 2024-10-02

Last Posted Date: 2025-01-17

Lead Sponsor: Intellia Therapeutics

Target Recruit Count: 30

Registration Number: NCT06622668

Locations: 🇳🇿
New Zealand Clinical Research, Aukland, New Zealand

Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002

Conditions: Hereditary Angioedema

First Posted Date: 2024-02-16

Last Posted Date: 2025-04-30

Lead Sponsor: Intellia Therapeutics

Target Recruit Count: 100

Registration Number: NCT06262399

Locations: 🇫🇷
Hôpital Claude Huriez, Lille, France
🇫🇷
Centre National de Reference - Grenoble, Grenoble, France
🇳🇱
University of Amsterdam Academic Medical Center, Amsterdam, Netherlands

and more 2 locations

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: Biological: NTLA-2001
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

and more 99 locations

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Recruiting

Conditions: Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Wild-Type Transthyretin Cardiac Amyloidosis

First Posted Date: 2023-01-26

Last Posted Date: 2025-05-20

Lead Sponsor: Intellia Therapeutics

Target Recruit Count: 72

Registration Number: NCT05697861

Locations: 🇬🇧
Clinical Trial Site, London, United Kingdom

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Phase 1

Active, not recruiting

Conditions: Hereditary Angioedema

Interventions: Biological: Biological NTLA-2002
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

Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

Phase 1

Terminated

Conditions: Acute Myeloid Leukemia

First Posted Date: 2021-10-04

Last Posted Date: 2023-12-28

Lead Sponsor: Intellia Therapeutics

Target Recruit Count: 6

Registration Number: NCT05066165

Locations: 🇬🇧
Research Site 8, London, United Kingdom
🇬🇧
Research Site 9, London, United Kingdom
🇺🇸
Research Site 2, Los Angeles, California, United States

and more 7 locations

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 Amyloidosis
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy

Interventions: Biological: NTLA-2001

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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