Intellia Therapeutics
- 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
15
Trial Phases
3 Phases
Drug Approvals
0
Drug Approvals
No drug approvals found
This company may not have drug approvals in our database
Clinical Trials
Distribution across different clinical trial phases (12 trials with phase data)• Click on a phase to view related trials
A Phase 3 Study of NTLA-2001 in ATTRv-PN
- 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
- 2025-06-18
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 50
- Registration Number
- NCT06672237
- Locations
- 🇧🇷
Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP), Ribeirão Preto, Brazil
🇧🇷Instituto de Educacao, Pesquisa e Gestao em Saude, Rio de Janeiro, Brazil
🇦🇷Hospital Británico de Buenos Aires, Buenos Aires, Argentina
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
- Conditions
- Hereditary Angioedema
- Interventions
- Biological: Normal Saline IV Administration
- First Posted Date
- 2024-10-09
- Last Posted Date
- 2025-06-12
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 60
- Registration Number
- NCT06634420
- Locations
- 🇺🇸
University of California, San Diego (UCSD), San Diego, California, United States
🇺🇸Medical Research of Arizona, Scottsdale, Arizona, United States
🇺🇸Raffi Tachdjian MD, Inc, Santa Monica, California, United States
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
- Conditions
- Lung DiseasePulmonary DiseaseAATDAlpha-1 Antitrypsin DeficiencyAlpha-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
- 🇫🇷
Centre National de Reference - Grenoble, Grenoble, France
🇫🇷Hôpital Claude Huriez, Lille, France
🇳🇱University of Amsterdam Academic Medical Center, Amsterdam, Netherlands
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
- Conditions
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
- Interventions
- Drug: Placebo
- First Posted Date
- 2023-11-13
- Last Posted Date
- 2025-06-12
- Lead Sponsor
- Intellia Therapeutics
- Target Recruit Count
- 765
- Registration Number
- NCT06128629
- Locations
- 🇺🇸
University of Arizona, Tucson, Arizona, United States
🇺🇸Cedars-Sinai Medical Center, Los Angeles, California, United States
🇺🇸Western Greater Los Angeles VA Medical Center, Los Angeles, California, United States
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News
Intellia's CRISPR Gene Therapy Shows 98% Reduction in HAE Attacks After Three Years
Intellia Therapeutics reported three-year follow-up data showing lonvoguran ziclumeran achieved a 98% mean reduction in monthly hereditary angioedema attack rates across all 10 Phase 1 patients.
CRISPR Therapeutics Reports Breakthrough Results from In Vivo Gene Therapy CTX310 for Heart Disease
CRISPR Therapeutics reported initial top-line results from an early-stage study on CTX310, showing peak reductions of up to 82% in triglycerides and 81% in LDL cholesterol with a single dose.
Novel Gene-Editing Therapy Shows Promise for Rare Fatal Heart Disease ATTR-CM
Singapore researchers are leading Asia's first clinical trial of Nexiguran Ziclumeran (nex-z), a groundbreaking gene-editing therapy targeting transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and potentially fatal heart disease.
CRISPR Gene Editing Advances from Laboratory to Clinic with 250+ Active Trials Across Multiple Therapeutic Areas
CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas.
CRISPR-Cas9 Market Set to Surge: Over 25 Therapies in Clinical Trials Expected to Launch Within 5-7 Years
The global CRISPR-Cas9 therapy market is rapidly expanding with over 25 therapies currently in clinical trials, many of which are expected to receive regulatory approval and enter the market within the next 5-7 years.
Bayer and CRISPR Therapeutics Form $300 Million Joint Venture to Advance Gene-Editing Therapies
Bayer is investing $300 million over five years in a new joint venture with CRISPR Therapeutics, plus $35 million for a minority stake in the gene-editing pioneer.
Orphan Drug Market Growth Slowing but Remains Strategic Priority for Pharma
Orphan drug sales reached $168 billion globally in 2023, with growth expected to moderate to just under 10% annually through 2028, according to Evaluate's latest report.
Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments
Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.
Intellia Therapeutics Prioritizes Late-Stage CRISPR Therapies, Announces Workforce Reduction
Intellia Therapeutics is prioritizing NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis, focusing on late-stage development.
Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments
Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions.