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.
First Patient Dosed in Cardiac Gene Therapy Trial
Roche plans to acquire Poseida Therapeutics for $1 billion to advance off-the-shelf CAR-T cell therapies. Tenaya Therapeutics dosed its first patient with TN-401 gene therapy for ARVC. The FDA granted RMAT designation to Intellia's nex-z CRISPR-based therapy for hereditary transthyretin amyloidosis.
Gene Switch Market Impacts on Medicine and Biotechnology Sector
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Intellia's Single-Dose Gene Editing Treatment for ATTR Amyloidosis Gets FDA Fast-Track
Intellia Therapeutics announces FDA's RMAT designation for nexiguran ziclumeran (NTLA-2001) to treat hereditary ATTR amyloidosis with polyneuropathy, based on interim Phase 1 data showing rapid, deep, and durable TTR reduction.
CF Foundation invests $15M in Recode Therapeutics to boost CF gene editing
The Cystic Fibrosis Foundation is investing up to $15 million in Recode Therapeutics to develop gene-editing medicines for CF patients without effective treatment options. Recode is collaborating with Intellia Therapeutics to create a healthy version of the CFTR gene, potentially working for all patients regardless of mutation. This is the second major investment in Recode by the CF Foundation, following support for RCT2100, an inhaled mRNA therapy currently in clinical testing.
ATTR-CM Approval for BridgeBio Could Trigger Tight Race with Pfizer
BridgeBio's acoramidis, a transthyretin stabilizer, may soon compete with Pfizer's tafamidis for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Acoramidis, with an FDA action date of Nov. 29, could see approval, potentially challenging Pfizer's established position in the $5.2 billion ATTR-CM market. BridgeBio's Phase III trial showed acoramidis improved survival rates and reduced hospitalizations, though it faces challenges like twice-daily dosing and lack of mortality benefit data compared to tafamidis. Emerging RNA therapies, like Alnylam's Amvuttra and Intellia's nex-z, also show promise in treating ATTR-CM.
Intellia's gene editing therapy shows early potential in rare heart condition
Intellia Therapeutics reports early-stage data indicating its gene editing treatment, NTLA-2001, may stabilize or improve outcomes for ATTR-CM patients, showing a median improvement in the six-minute walk test and 92% maintaining or improving NYHA class. The treatment uses CRISPR to reduce TTR production, with a 90% mean reduction observed. Intellia is now in Phase III trials for NTLA-2001, with potential revenue of $1.2bn by 2030.
Related Clinical Trials:
Intellia Announces First Clinical Evidence from Ongoing Phase 1 Study that Nexiguran ...
Intellia Therapeutics announced positive Phase 1 trial data for nex-z in ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction, disease stabilization or improvement, and favorable safety profile. Data presented at the 2024 AHA Scientific Sessions and published in the New England Journal of Medicine.
Novel CRISPR-Cas9-Based Promising in ATTR Cardiomyopathy
A CRISPR-Cas9-based therapy, nexiguran ziclumeran, reduced serum transthyretin (TTR) levels by 89% at 28 days and 90% at 12 months in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The therapy, targeting TTR production in the liver, showed potential to halt disease progression, with minimal change in biomarkers and functional tests. The phase III MAGNITUDE trial will further assess its safety and efficacy.
Intellia Therapeutics Reports Promising Phase 1 Trial Results For Nexiguran Ziclumeran In ...
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Intellia says CRISPR therapy shows superior gain for cardiomyopathy patients
Intellia Therapeutics CEO John Leonard faces stock market challenges despite positive gene editing trial data. New year-long data from a CRISPR drug trial for ATTR cardiomyopathy aims to address concerns about its effectiveness compared to older technologies.
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