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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 (NASDAQ:NTLA) announced the appointment of Edward Dulac as the new Executive Vice President, Chief Financial Officer (CFO), and Treasurer, effective July 22, 2024. Dulac will replace Glenn Goddard, who steps down on June 30, 2024. Dulac brings over 20 years of experience in finance, business development, and corporate strategy from roles at Fate Therapeutics, Celgene (now Bristol Myers Squibb), Barclays Capital, Lehman Brothers, and Pfizer. Intellia's CEO, John Leonard, expressed confidence in Dulac's ability to drive the company's future commercial success and thanked Goddard for his contributions. This leadership transition marks a significant step as Intellia continues to advance its CRISPR-based therapies.
Intellia Therapeutics has announced positive clinical data demonstrating the potential for redosing with its proprietary CRISPR-based therapy, NTLA-2001. In a Phase 1 study, a follow-on 55 mg dose led to a 90% median reduction in serum TTR at day 28 among three patients who previously received the lowest dose. This redosing capability, enabled by Intellia's non-viral lipid nanoparticle (LNP) delivery platform, is not currently planned for the NTLA-2001 program but shows promise for future therapies requiring additive effects. The 55 mg follow-on dose was well tolerated, with only one mild infusion-related reaction reported. The NTLA-2001 program is part of a collaboration with Regeneron. The promising results will inform Phase 3 trials for ATTR-CM and ATTRv-PN.
Intellia Therapeutics (NASDAQ: NTLA) will present groundbreaking clinical data on redosing patients with its investigational CRISPR gene editing therapy, NTLA-2001, at the Peripheral Nerve Society Annual Meeting from June 22-25, 2024, in Montreal, Canada.
The Phase 1 study data, featuring redosing of three patients initially administered 0.1 mg/kg followed by a 55 mg dose, will provide insights into the safety and pharmacodynamics of this approach.
While repeat dosing is not planned for NTLA-2001 in treating transthyretin amyloidosis (ATTR), it could benefit future therapies using Intellia’s lipid nanoparticle (LNP) delivery platform by enabling additional dosing where needed.
Dr. Jorg Taubel will present these findings on June 25, 2024.
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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