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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, Inc. (NASDAQ:NTLA), a leader in genome editing, will announce its first quarter 2023 financial results and operational highlights on May 4, 2023, at 8 a.m. ET. Investors can participate via a conference call or a live webcast. The company focuses on developing potentially curative therapeutics using CRISPR-based technologies through its in vivo and ex vivo programs, targeting specific disease-causing genes. Intellia aims to harness the full potential of genome editing for transformative genetic therapies, supported by its robust intellectual property portfolio and clinical development experience.
Intellia Therapeutics (NASDAQ:NTLA) announced the appointment of Bill Chase to its board of directors, effective immediately. Chase will also chair the audit committee, succeeding Caroline Dorsa, who is retiring on June 15, 2023, following her appointment as chair of Biogen's board. Bill Chase has over 30 years of experience in financial management, having served as CFO at AbbVie and held various roles at Abbott. This leadership change is aimed at strengthening Intellia's governance as it advances its CRISPR-based therapies into late-stage clinical development. CEO John Leonard emphasized Chase's financial acumen as a valuable asset in the company's mission to develop novel gene therapies.
Intellia Therapeutics (NASDAQ: NTLA) announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational therapy NTLA-2002, aimed at treating hereditary angioedema (HAE). This CRISPR-based treatment targets the kallikrein B1 (KLKB1) gene to prevent life-threatening swelling attacks. The RMAT designation facilitates accelerated development and review, including early FDA interactions and potential priority review. NTLA-2002 has also received Orphan Drug Designation and Innovation Passport status. Intellia is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of NTLA-2002.
Intellia Therapeutics (NASDAQ: NTLA) announced that the FDA has cleared its IND application for NTLA-2002, a genome editing candidate aimed at treating hereditary angioedema (HAE). This approval allows the inclusion of the United States in the ongoing Phase 1/2 clinical trial. NTLA-2002 employs CRISPR technology to permanently inactivate the kallikrein B1 (KLKB1) gene, significantly reducing the frequency of HAE attacks. Intellia will rapidly begin patient enrollment for Phase 2 and anticipates data release from the Phase 1 study later this year. The company aims to revolutionize HAE treatment and has reported promising interim results from early trials.
Intellia Therapeutics (NTLA) announced key milestones in early 2023, including the initiation of a global Phase 2 study for NTLA-2002, a CRISPR-based treatment for hereditary angioedema. The IND application for NTLA-2002 has been submitted to enable patient enrollment in the U.S. Furthermore, the company plans to file an IND application for NTLA-2001 for ATTR amyloidosis by mid-2023, with a global pivotal trial expected by year-end. Intellia ended 2022 with $1.3 billion in cash. Major R&D expenses rose to $100 million, contributing to a net loss of $113.4 million for Q4 2022. Upcoming clinical data presentations in 2023 will support these developments.
Intellia Therapeutics (NASDAQ:NTLA) will announce its fourth quarter and full-year 2022 financial results on February 23, 2023, at 8 a.m. ET. Investors can join the conference call by dialing 1-833-316-0545 (U.S.) or 1-412-317-5726 (international) five minutes prior. A live webcast will also be available through their website. Intellia is a leader in developing CRISPR-based genome editing therapeutics, focusing on innovative treatments for various diseases through in vivo and ex vivo programs. The company aims to unlock the full potential of genetic medicine.
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