Clinical Research

Intellia Therapeutics Inc.

Address
40 Erie Street

Sponsor Overview

assignment
7
sponsored trials
science
11
molecules
location_city
55
research centres
public
15
countries
person_search
53
investigators

Specializations

Hereditary Angioedema

Clinical research includes hereditary angioedema, with studies focused on hereditary angioedema due to C1 esterase inhibitor deficiency and related attack prevention. The programme also includes long-term follow-up in treated participants and evaluation of disease control in adults with recurrent swelling episodes.

  • HAE attacks
  • C1 esterase inhibitor deficiency
  • Long-term safety

Research activity in this area is centred on reducing disease burden in patients with recurrent angioedema episodes.

Transthyretin Amyloidosis

The sponsor supports trials in transthyretin amyloidosis, including both hereditary transthyretin amyloidosis with polyneuropathy and transthyretin amyloidosis-related cardiomyopathy. These studies address neurological and cardiac manifestations of the disease, with follow-up in previously treated participants.

  • ATTRv-PN
  • ATTR-CM
  • Cardiac muscle disease

Work in this field reflects a strong focus on inherited amyloid disorders affecting the peripheral nerves and the heart.

Alpha-1 Antitrypsin Deficiency

Clinical investigation also extends to alpha-1 antitrypsin deficiency-associated lung disease, including pulmonary emphysema. The research programme examines lung-related disease in adults with inherited deficiency states and associated respiratory impairment.

  • AATD-associated lung disease
  • Pulmonary emphysema
  • Adult respiratory disease

This area adds a pulmonary dimension to the sponsor’s portfolio of inherited disease research.

Genetic Medicine

The overall portfolio is concentrated in genetic medicine and rare disease research, with activity across inherited disorders that affect the immune, neurological, cardiac, and respiratory systems. The sponsor’s clinical work spans multiple countries and research sites, supporting studies in severe monogenic conditions.

  • Rare inherited disorders
  • Neurological disease
  • Cardiovascular disease

Medical affairs activity is directed toward clinical development in genetically defined patient populations.

Sponsored Trials

Trial Name Country Status
Evaluation of NTLA-2001 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Hereditary Transthyretin Amyloidosis with PolyneuropathyFrance FranceSweden SwedenNot Recruiting
Evaluation of NTLA-2002 Efficacy and Safety in Hereditary Angioedema Patients: A Phase 3 Randomized, Double-Blind, Placebo-Controlled TrialFrance FranceGermany GermanyThe Netherlands The NetherlandsNot Recruiting
Evaluation of NTLA-2002, a CRISPR-Cas9 Gene Editing Therapy, for Safety and Efficacy in Adults with Hereditary Angioedema (HAE)France FranceGermany GermanyThe Netherlands The NetherlandsNot Recruiting
Evaluation of NTLA-3001 Safety and Pharmacokinetics in Adults with Alpha-1 Antitrypsin Deficiency-Associated Pulmonary EmphysemaIreland IrelandNot Recruiting
Long-Term Safety Evaluation of NTLA-2001 in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy and Transthyretin Amyloidosis-Related CardiomyopathyFrance FranceSweden SwedenRecruiting
Long-Term Safety Evaluation of NTLA-2002 in Patients with Hereditary Angioedema: A Follow-Up Study on Ziclumeran and LonvoguranFrance FranceGermany GermanyThe Netherlands The NetherlandsRecruiting
Phase 3 Randomized Double-Blind Study of NTLA-2001 Efficacy and Safety in Transthyretin Amyloidosis with Cardiomyopathy PatientsAustria AustriaBelgium BelgiumCzechia CzechiaDenmark DenmarkFrance FranceGermany GermanyHungary HungaryItaly ItalyNorway NorwayPoland PolandPortugal PortugalSpain SpainSweden SwedenThe Netherlands The NetherlandsRecruiting

Data & Insights

science
Molecule Portfolio
Compounds under investigation
4
Exclusive Molecules
out of 11 total (36% not shared with other sponsors)

This sponsor works with 11 molecules, of which 4 are not being studied by other sponsors.

hub
Site Partnerships
Collaboration with research centres
13%
Return partnership rate
7 of 55 sites collaborated multiple times

This sponsor has worked with 55 sites across their trial portfolio.

Therapeutic Focus

  • 1.
    Congenital, Hereditary, and Neonatal Diseases and Abnormalities5 trials
    165 other sponsors
  • 2.
    Cardiovascular Diseases1 trial
    359 other sponsors
  • 3.
    Respiratory Tract Diseases1 trial
    277 other sponsors

Partnered Research Centres

55 centres
location_city
assignment352
Aarhus University Hospital
Address
Palle Juul-Jensens Boulevard 99, 8200 Aarhus
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location_city
assignment155
Amsterdam UMC
Address
Meibergdreef 9, 1105 AZ Amsterdam
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location_city
assignment680
Amsterdam UMC
Address
De Boelelaan 1117, 1081 HV Amsterdam
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location_city
assignment966
Assistance Publique Hopitaux De Marseille
Address
264 Rue Saint Pierre, 13005 Marseille
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location_city
assignment191
Az St-Jan Brugge-Oostende A.V.
Address
Ruddershove 10, 8000 Bruges
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location_city
assignment624
Azienda Ospedaliero Universitaria Careggi
Address
Largo Giovanni Alessandro Brambilla 3, 50134 Florence
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