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.
Intellia Therapeutics Announces Initiation of HAELO Phase 3 Study of NTLA-2002
Intellia Therapeutics initiates HAELO Phase 3 study of NTLA-2002, a single-dose CRISPR-based gene editing therapy for hereditary angioedema (HAE), following positive Phase 1/2 data.
Related Clinical Trials:
Intellia Therapeutics Announces Initiation of HAELO Phase 3
Intellia Therapeutics initiates HAELO, a global Phase 3 study of NTLA-2002, a CRISPR-based gene editing therapy for hereditary angioedema (HAE). NTLA-2002 aims to prevent HAE attacks by inactivating the KLKB1 gene, with a single-dose treatment potential. The study follows positive Phase 1/2 data, showing significant attack rate reductions and durable kallikrein level reductions.
Related Clinical Trials:
2 Innovative Cathie Wood Stocks to Buy If You Can Stomach the Risk
Cathie Wood's Ark Innovation ETF added Intellia Therapeutics (gene-editing) and Recursion Pharmaceuticals (AI-driven drug development) to its portfolio, highlighting their potential despite risks.
Jennifer Doudna: 'Curiosity-driven, fundamental science' underpins Nobel Prize-winning work
Jennifer Doudna, Nobel Prize winner and CRISPR pioneer, discussed improving CRISPR-based gene therapies at Fred Hutch Cancer Center. Her lab aims to make CRISPR technologies more efficient and easier to deliver, focusing on enhancing Cas9 DNA editing and developing better delivery methods. Doudna highlighted the potential of miniEDVs, a novel delivery system, and emphasized the need for diverse genetic data to ensure therapy effectiveness across different populations.
Top three trends in precision medicine
Precision medicine uses genetic, environmental, and lifestyle data to tailor treatments, with AI, CRISPR-Cas9, and mRNA as key trends. AI accelerates genomic analysis and drug discovery, CRISPR-Cas9 offers precise gene editing, and mRNA vaccines expand beyond COVID-19, driving personalized healthcare.
Gene Editing Therapeutics Market Report 2024, Featuring Profiles of Key Players Allogene
Gene editing therapeutics market estimated at $11 million in 2024, forecasted to grow at 147% CAGR to reach $1 billion by 2029, driven by chronic diseases, rare disorders, strategic initiatives, and precision medicine.
Future of CRISPR: Gene Editing Technologies Herald Landmark Clinical Trials
CRISPR technology, including CRISPR-Cas9, CRISPR-Cas12, and CRISPR-Cas13, is revolutionizing genome editing. FDA approved the first CRISPR-Cas9 drug, Casgevy®, for sickle cell disease and beta thalassemia. Other applications include treating urinary tract infections, hereditary transthyretin amyloidosis, hereditary angioedema, cardiovascular diseases, type 1 diabetes, systemic lupus erythematosus, HIV, and blood cancers. Challenges include cost, regulatory standards, and ethical considerations.
Related Clinical Trials:
Intellia Therapeutics Announces First Quarter 2024 Progress and Plans
Intellia Therapeutics advances CRISPR-based therapies, with NTLA-2001 Phase 3 trials for ATTR amyloidosis progressing ahead of schedule. Plans to initiate NTLA-2001 Phase 3 for polyneuropathy by year-end. NTLA-2002 Phase 3 for HAE and NTLA-3001 Phase 1 for AATD to start in 2024. Ended Q1 2024 with $953M in cash.
Best Biotech Stocks to Buy in 2025
The golden age of biotechnology is marked by scientific advances in disease treatment and prevention, offering investment opportunities. Key companies like Axsome Therapeutics, Exelixis, Intellia Therapeutics, Regeneron Pharmaceuticals, Vertex Pharmaceuticals, and Twist Bioscience are leading with innovative drugs and technologies. The COVID-19 pandemic has further accelerated biotech advancements, especially in treatments and vaccines.
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