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Intellia Therapeutics, Inc. (NASDAQ: NTLA) is a pioneering clinical-stage gene editing company dedicated to revolutionizing medicine through the use of CRISPR-based therapies. Founded in 2014 by Caribou Biosciences and Atlas Venture, along with leading scientists in the field, Intellia harnesses the potential of CRISPR/Cas9 technology to develop curative treatments for a variety of genetically defined diseases.
The company's core mission is to leverage CRISPR/Cas9—an innovative technology adapted from a natural cellular process that allows for precise and flexible gene editing. This technology enables the correction of specific DNA sequences within any organism, opening new frontiers in biomedical research and clinical interventions.
Intellia's portfolio includes both in vivo and ex vivo gene editing programs. The in vivo programs aim to edit disease-causing genes directly within the human body, targeting conditions such as ATTR amyloidosis, hereditary angioedema (HAE), and alpha-1 antitrypsin deficiency. Notable achievements include the interim results from their NTLA-2002 program for HAE, which showed a 95% reduction in monthly attack rates, with most patients remaining attack-free.
The ex vivo programs focus on engineering human cells outside the body to treat cancer and autoimmune diseases. These programs include collaborations with prominent companies like Regeneron and Novartis, which help advance their therapeutic pipeline.
Intellia's financial condition reflects its robust pipeline and strategic partnerships. The company recently reported strong enrollment progress in their pivotal Phase 3 MAGNITUDE trial for NTLA-2001 targeted at ATTR amyloidosis with cardiomyopathy. They have also formed a strategic collaboration with ReCode Therapeutics to develop treatments for cystic fibrosis, utilizing ReCode's Selective Organ Targeting (SORT) lipid nanoparticle delivery platform.
Committed to advancing the field of gene editing, Intellia continues to expand its CRISPR-based platform with novel editing and delivery technologies. The company remains at the forefront of a new era in medicine, as evidenced by its ongoing clinical trials and promising preliminary data.
For more detailed information and the latest updates, investors and stakeholders are encouraged to visit www.intelliatx.com and follow Intellia on Twitter @intelliatx.
Intellia Therapeutics, a clinical-stage gene editing company, announced the appointment of Brian Goff to its board of directors. Goff, who is the current CEO of Agios Pharmaceuticals, brings over three decades of experience in commercialization, operations, and sales and marketing. He has held senior roles at Alexion Pharmaceuticals, Neurovance, Baxalta, Baxter Healthcare, Novartis Pharmaceuticals, and Johnson & Johnson. Intellia's CEO, John Leonard, highlighted Goff's expertise in rare disease product launches as critical for the company's transition to a commercial organization, especially as it prepares to commercialize its first CRISPR-based therapies.
Intellia Therapeutics announced positive long-term data from the Phase 1 study of NTLA-2002, a CRISPR-based gene editing therapy for hereditary angioedema (HAE). The data showed that eight out of ten patients remained attack-free following a 16-week primary observation period and continued to show positive results for over two years. A single dose of NTLA-2002 led to a 98% reduction in monthly HAE attacks. All patients who stopped prophylaxis treatment remained free from chronic treatment. The therapy demonstrated a favorable safety profile with mild adverse events. The company plans to begin a Phase 3 trial in the second half of 2024.
Intellia Therapeutics, Inc. announced strong financial results and progress in gene editing therapies for various diseases. They are rapidly enrolling patients in Phase 3 trials for ATTR amyloidosis, planning new Phase 3 trials for other conditions, and presenting clinical data in 2024. The company ended Q1 2024 with approximately $953 million in cash.
Intellia Therapeutics, a gene editing company, will discuss its first quarter 2024 financial results and operational highlights on May 9, 2024. The conference call will focus on revolutionizing medicine with CRISPR-based therapies. Investors can join the call via phone or webcast.
Intellia Therapeutics, Inc. (NASDAQ: NTLA) will present updated data from the Phase 1/2 study of NTLA-2002 for the treatment of Hereditary Angioedema (HAE) at the EAACI Congress 2024. The presentation will cover safety, kallikrein reduction, and attack rate data. The company will also host an investor webcast on June 3, 2024.
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