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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 significant advancements in its clinical development pipeline for 2022. The company is progressing NTLA-2001 for transthyretin amyloidosis, with additional Phase 1 study data expected in Q1 2022. NTLA-2002 is also in development for hereditary angioedema, with interim data anticipated in 2H 2022. The firm is establishing the safety profile of NTLA-5001 for acute myeloid leukemia and plans to nominate new development candidates. Intellia ended 2021 with a robust cash position of $1.1 billion.
Intellia Therapeutics, Inc. (NASDAQ:NTLA) will present virtually at the 40th Annual J.P. Morgan Healthcare Conference on January 12, 2022, at 2:15 p.m. ET. This leading clinical-stage genome editing company focuses on developing CRISPR-based therapeutics. Interested parties can access a live audio webcast of the presentation on Intellia’s website, with a replay available for two weeks afterward.
Intellia is advancing innovative therapies through in vivo and ex vivo CRISPR programs, demonstrating its leadership in genetic medicine.
Intellia Therapeutics (NTLA) has entered a collaboration with Kyverna Therapeutics to develop KYV-201, an allogeneic CD19 CAR T-cell therapy targeting autoimmune diseases. Under the agreement, Kyverna will lead preclinical and clinical development, while Intellia will gain an equity stake and potential royalties on sales. Intellia retains the option to co-develop KYV-201 in the U.S., sharing development costs and revenue. The partnership aims to leverage Intellia's CRISPR technology, allowing for improved safety and efficacy in autoimmune treatments.
Intellia Therapeutics (NASDAQ: NTLA) has appointed Derek Hicks as the new Executive Vice President, Chief Business Officer. With over 25 years of experience, Hicks previously led business development at Spark Therapeutics and held significant roles at Pfizer. Intellia's President John Leonard expressed confidence that Hicks will enhance their growth and collaboration strategies in genome editing, emphasizing the company's commitment to developing innovative therapeutics using CRISPR/Cas9 technology. Hicks is eager to join Intellia at this pivotal moment in its evolution.
Intellia Therapeutics (NTLA) reports significant progress with NTLA-2002, the first single-dose CRISPR gene-editing therapy for hereditary angioedema (HAE). The first patient has been successfully dosed in a Phase 1/2 study designed to evaluate safety, tolerability, and pharmacodynamics. The therapy aims to inactivate the kallikrein B1 (KLKB1) gene, reducing plasma kallikrein activity to prevent HAE attacks. This innovative treatment has the potential to drastically lower treatment burdens for patients with HAE.
Intellia Therapeutics has received regulatory approval from the UK Medicines and Healthcare products Regulatory Agency for a protocol amendment in its Phase 1 study of NTLA-2001, a CRISPR/Cas9-based therapy. The amendment allows enrollment of patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study aims to assess safety and efficacy, expanding its participant pool to include up to 36 adults with different forms of ATTR-CM. NTLA-2001 is pioneering as a potentially curative treatment, with previous interim data showing significant TTR level reductions.
Intellia Therapeutics (NASDAQ: NTLA) announced it will present data from its ex vivo research and development at the 63rd American Society of Hematology Annual Meeting, from December 11-14, 2021. Key presentations include a novel allogeneic technology that mitigates host cell rejection and details on the clinical-scale manufacturing of NTLA-5001, a T cell therapeutic for acute myeloid leukemia. The data aims to advance engineered cell therapies for life-threatening diseases, showcasing Intellia's commitment to CRISPR/Cas9 technology.
Intellia Therapeutics (NTLA) announced significant advancements in its clinical pipeline. They initiated dosing of Cohort 4 in the Phase 1 study of NTLA-2001, targeting ATTR amyloidosis, with data expected in Q1 2022. The company accelerated plans for NTLA-2001 in patients with ATTR-CM and is on track to launch first-in-human studies for NTLA-2002 (HAE) and NTLA-5001 (AML) by year-end. Intellia also nominated NTLA-3001 for AATD and reported a robust cash position of $1.1 billion. However, the company faced a net loss of $71.6 million in Q3 2021.
Intellia Therapeutics (NASDAQ: NTLA) will present its third quarter 2021 financial results and operational highlights on November 4, 2021, at 8 a.m. ET. The call can be accessed via U.S. dial-in at 1-833-316-0545 or international at 1-412-317-5726. It will feature crucial insights on the company's advancements in developing curative CRISPR/Cas9 therapeutics. Additionally, a replay will be available on the company's Investors & Media section after the call.
Intellia Therapeutics (NASDAQ: NTLA) announced that the FDA granted orphan drug designation to NTLA-2001 for treating ATTR amyloidosis. This investigational therapy is the first CRISPR treatment designed for systemic use. NTLA-2001 aims to be a potential single-dose treatment, possibly halting the disease’s complications. Intellia is committed to advancing its genome editing platform, with NTLA-2001 currently in Phase 1 trials. The FDA's designation provides development incentives, echoing a similar approval from the European Commission earlier this year.
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