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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) has announced two upcoming virtual investor events in November 2024. The first event, scheduled for November 7 at 8 a.m. ET, will present third quarter 2024 financial results and operational highlights. The second event, on November 16 at 11 a.m. CT/12 p.m. ET, will showcase new clinical data from the Phase 1 study of nexiguran ziclumeran (nex-z), their investigational CRISPR-based gene editing therapy for transthyretin (ATTR) amyloidosis. The nex-z data presentation follows a late-breaking oral presentation at the 2024 American Heart Association Scientific Sessions in Chicago. This therapy is being developed in collaboration with Regeneron as a single-dose treatment.
Intellia Therapeutics announced positive Phase 2 results for NTLA-2002, their investigational CRISPR gene editing treatment for hereditary angioedema (HAE). The study showed that a single 50 mg dose resulted in a 77% and 81% mean monthly attack rate reduction compared to placebo during weeks 1-16 and 5-16, respectively. Eight of 11 patients in the 50 mg arm remained completely attack-free following a one-time infusion. The treatment demonstrated an encouraging safety profile, with most adverse events being Grade 1 or 2. Based on these results, Intellia has selected the 50 mg dose for their global pivotal Phase 3 HAELO study.
Intellia Therapeutics (NASDAQ:NTLA) has announced a new date for its investor webcast to review NTLA-2002 Phase 2 data. The webcast will now be held on Thursday, October 24 at 8:30 a.m. ET, instead of the previously announced date of October 28, 2024.
This change does not affect the planned oral presentation at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Boston, Massachusetts, scheduled for October 24 – 28.
Investors can join the webcast via a provided link or through the Events and Presentations page on Intellia's website. A replay of the webcast will be available on the company's website for at least 30 days following the call.
Intellia Therapeutics (NASDAQ:NTLA) has initiated HAELO, a global Phase 3 study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for hereditary angioedema (HAE). NTLA-2002 is designed as a single-dose treatment to prevent potentially life-threatening swelling attacks in HAE patients. The study will evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE, with patients randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo.
The primary endpoint is the change in number of HAE attacks from week 5 through week 28. This initiation follows positive safety and efficacy data from the ongoing Phase 1/2 study, which showed dramatic reductions in attack rate and consistent, deep, and durable reductions in kallikrein levels. Detailed results from the Phase 2 portion will be presented at the 2024 ACAAI Annual Scientific Meeting in October.
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on CRISPR-based therapies, has announced the granting of inducement awards to nine new employees under its 2024 Inducement Plan. The awards, granted on October 1, 2024, consist of time-based restricted stock units (RSUs) for 54,944 shares of Intellia's common stock.
The RSUs will vest in three equal installments on October 1 of 2025, 2026, and 2027, subject to the employees' continued service. These awards were granted outside of Intellia's stockholder-approved equity incentive plans, as part of the 2024 Inducement Plan adopted by the board of directors in June 2024. The compensation committee approved the awards as a material inducement for employment, in accordance with Nasdaq Listing Rule 5635(c)(4).
Intellia Therapeutics (NASDAQ:NTLA) announced that new interim data from the ongoing Phase 1 study of nexiguran ziclumeran (nex-z) will be presented at the 2024 American Heart Association (AHA) Scientific Sessions in Chicago. Nex-z is an investigational in vivo CRISPR-based gene editing therapy designed as a single-dose treatment for transthyretin (ATTR) amyloidosis.
The presentation will include safety data, reduction in serum TTR, biomarkers of disease progression, and functional capacity data in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). Dr. Marianna Fontana from University College London will present the findings on November 16, 2024, as part of the Late-Breaking Science session.
Intellia is developing nex-z in collaboration with Regeneron as part of a multi-target discovery, development, and commercialization partnership.
Intellia Therapeutics (NASDAQ:NTLA) has announced that detailed results from the Phase 2 study of NTLA-2002, their investigational in vivo CRISPR-based gene editing therapy for hereditary angioedema (HAE), will be presented at the 2024 ACAAI Annual Scientific Meeting. The presentation, titled "Results From a Phase 2, Randomized, Placebo-Controlled Trial of CRISPR-Based Therapy NTLA-2002 for Hereditary Angioedema," will take place on October 26, 2024. This follows a previous positive topline announcement that the study met its primary and all secondary endpoints. Intellia will also host an investor webcast on October 28, 2024, to review the new data.
Intellia Therapeutics (NASDAQ:NTLA), a leading clinical-stage gene editing company, has announced inducement grants for five new employees under its 2024 Inducement Plan. The grants include:
- Non-qualified stock options for 49,751 shares
- Time-based RSUs for 44,154 shares
- Performance-based RSUs for 17,064 shares linked to total stockholder return
- Performance-based RSUs for 12,500 shares tied to development milestones
These equity awards, granted outside of Intellia's stockholder-approved plans, were approved by the compensation committee as a material inducement for employment, in accordance with Nasdaq Listing Rule 5635(c)(4).
Intellia Therapeutics (NASDAQ:NTLA) reported positive Phase 2 results for NTLA-2002 in hereditary angioedema (HAE), meeting primary and secondary endpoints. The company plans to initiate a Phase 3 trial in 2H 2024 with the selected 50 mg dose. Rapid enrollment continues in the Phase 3 MAGNITUDE trial of NTLA-2001 for ATTR amyloidosis with cardiomyopathy. Intellia expects to dose the first patient in the Phase 1/2 study of NTLA-3001 for alpha-1 antitrypsin deficiency in 2H 2024. The company ended Q2 2024 with $939.9 million in cash, expected to fund operations into late 2026. Q2 financial results show collaboration revenue of $7.0 million, R&D expenses of $114.2 million, and a net loss of $147.0 million.
Intellia Therapeutics (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on CRISPR-based therapies, has announced its upcoming second quarter 2024 earnings call. The conference call is scheduled for August 8, 2024, at 8 a.m. ET. During this call, the company will present its Q2 2024 financial results and provide operational highlights.
Investors and interested parties can join the call by dialing 1-833-316-0545 (U.S.) or 1-412-317-5726 (international). A live webcast will also be available. A replay of the call will be accessible through Intellia's website after 12 p.m. ET on the same day.