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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 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.
Intellia Therapeutics (NASDAQ:NTLA) has received the Innovation Passport from the U.K. Medicines and Healthcare products Regulatory Agency for NTLA-2002, a genome editing therapeutic candidate aimed at treating hereditary angioedema (HAE). This designation allows entry into the Innovative Licensing and Access Pathway (ILAP), facilitating faster market access for innovative medicines. The Phase 2 portion of NTLA-2002's clinical study is expected to start in the first half of 2023, following ongoing Phase 1/2 trials focusing on the drug's safety and effectiveness.
Intellia Therapeutics (NASDAQ: NTLA) outlines its strategic priorities for 2023-2024, emphasizing late-stage development of CRISPR-based therapies NTLA-2001 and NTLA-2002. Key milestones include submitting IND applications for these treatments and initiating pivotal global studies by the end of 2023. The company ended 2022 with approximately $1.3 billion in cash, indicating a strong financial position. Intellia aims to advance novel gene editing technologies and maintain its leadership in genome editing through high-impact opportunities.
Intellia Therapeutics (NASDAQ:NTLA) has priced a public offering of 6,550,219 shares at $45.80 each, aiming to raise approximately $300 million. The underwriter, Goldman Sachs, has a 30-day option for an additional 982,532 shares. The offering is set to close around December 2, 2022, based on standard conditions. The shares are offered under an existing SEC registration statement. Intellia specializes in CRISPR-based genome editing therapies, focusing on potential cures for diseases through innovative therapeutic approaches.
Intellia Therapeutics (NASDAQ: NTLA) announced a public offering of $250 million in common stock, with a potential additional 15% option for underwriters. Goldman Sachs & Co. LLC is the sole underwriter. The offering is subject to market conditions and follows an effective shelf registration with the SEC. A preliminary prospectus will be available soon. The funds raised are intended to enhance Intellia's development of CRISPR-based therapies. However, there is no guarantee on the offering's completion or terms.
Intellia Therapeutics has released promising interim data from its Phase 1/2 clinical trial of NTLA-2002, a CRISPR-based therapy for hereditary angioedema (HAE). Results show robust reductions in plasma kallikrein levels and HAE attack rates across all tested doses. Notably, patients in the 25 mg and 75 mg cohorts remain attack-free through the latest follow-up, with intervals lasting between 5.5 to 10.6 months. The treatment was well-tolerated, with no serious adverse effects reported. An investor event discussing these findings is scheduled for November 14, 2022.
Intellia Therapeutics (NASDAQ:NTLA) has presented interim results from its Phase 1 trial of NTLA-2001, a CRISPR-based treatment for ATTR amyloidosis with cardiomyopathy. Results showed a remarkable 90% mean serum TTR reduction following a single dose, sustained for four to six months. NTLA-2001 was generally well-tolerated, with minimal adverse reactions. This study marks a significant step in exploring the potential of NTLA-2001 to permanently inactivate the TTR gene, offering a promising future for patients with this life-threatening condition.
Intellia Therapeutics (NTLA) reported significant interim results from its clinical trials for NTLA-2001 and NTLA-2002 in the third quarter of 2022. NTLA-2001 showed mean serum transthyretin protein reductions of 93% and 92% at doses of 0.7 mg/kg and 1.0 mg/kg, respectively. The firm has strong cash reserves of $848.7 million and expects to initiate Phase 2 of NTLA-2002 by early 2023. Collaboration revenue increased to $13.3 million, while R&D expenses rose to $96.7 million. The net loss was reported at $113.2 million, indicating an increase compared to the previous year.
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