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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), a clinical-stage gene editing company specializing in CRISPR-based therapies, has announced it will host a conference call on February 27, 2025, at 8 a.m. ET to discuss its fourth quarter and full-year 2024 financial results and operational highlights.
U.S. participants can dial 1-833-316-0545, while international callers should use 1-412-317-5726. A live webcast will be available, and a replay will be accessible through the company's website starting at 12 p.m. ET on the same day.
Intellia Therapeutics (NASDAQ:NTLA) has announced the award of inducement grants to three new employees under its 2024 Inducement Plan. The grants consist of time-based restricted stock units (RSUs) for 21,863 shares of common stock, which will vest in three equal installments on February 1 of 2026, 2027, and 2028. The vesting is contingent upon continued service with Intellia.
The awards were granted outside of Intellia's stockholder-approved equity incentive plans through the 2024 Inducement Plan, which was adopted by the board in June 2024. The compensation committee approved these grants as material inducement for employment in accordance with Nasdaq Listing Rule 5635(c)(4).
Intellia Therapeutics (NASDAQ:NTLA) has announced the dosing of the first patient in its global Phase 3 HAELO study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for hereditary angioedema (HAE). The company expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026, aiming for a U.S. launch in 2027.
NTLA-2002 is being developed as a single-dose treatment for this potentially life-threatening disease. The company plans to present longer-term data from the ongoing Phase 1/2 study later this year to demonstrate the treatment's durability of effect.
Intellia Therapeutics (NASDAQ:NTLA) announced strategic reorganization and 2025 milestones, focusing on two priority programs: NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for ATTR amyloidosis. The company plans to complete enrollment for the Phase 3 HAELO study in H2 2025 and aims to submit a Biologics License Application in H2 2026.
The reorganization includes discontinuing NTLA-3001 development and implementing a 27% workforce reduction in 2025, resulting in approximately $8 million in charges. With approximately $862 million in cash and investments at Q4 2024, combined with cost savings, the company expects to maintain operations into H1 2027.
Key 2025 objectives include enrolling over 550 patients in the MAGNITUDE study for nex-z, completing commercial leadership team buildout, and advancing medical education initiatives. The company also announced leadership changes, with CSO Laura Sepp-Lorenzino retiring and Birgit Schultes promoted to Executive VP and CSO.
Intellia Therapeutics (NASDAQ:NTLA) has granted inducement awards to three new employees under its 2024 Inducement Plan. The grants consist of time-based restricted stock units (RSUs) for 23,155 shares of common stock, which will vest in three equal installments on January 1 of 2026, 2027, and 2028. The vesting is contingent upon continued employment with Intellia.
These awards were granted outside of Intellia's stockholder-approved equity incentive plans through the 2024 Inducement Plan, which was adopted by the board in June 2024. The compensation committee approved these grants as material inducement for employment in compliance with Nasdaq Listing Rule 5635(c)(4).
Intellia Therapeutics (NASDAQ:NTLA) has announced an inducement grant award to a new employee under its 2024 Inducement Plan. The grant consists of 3,521 restricted stock units (RSUs) of Intellia's common stock, which will vest in three equal installments on December 1 of 2025, 2026, and 2027. The award was granted outside of Intellia's stockholder-approved equity incentive plans and was approved by the company's compensation committee in accordance with Nasdaq Listing Rule 5635(c)(4).
Intellia Therapeutics (NASDAQ:NTLA) announced that its investigational therapy nexiguran ziclumeran (nex-z) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treating hereditary transthyretin amyloidosis with polyneuropathy. This single-dose CRISPR-based treatment aims to inactivate the TTR gene to prevent TTR protein production. The designation was granted based on promising Phase 1 data showing rapid, deep, and durable TTR reduction. This marks the third special regulatory designation for nex-z, following Orphan Drug Designations from both the FDA and European Commission.
Intellia Therapeutics announced positive clinical data from its ongoing Phase 1 trial of nexiguran ziclumeran (nex-z), a CRISPR-based gene editing therapy for ATTR amyloidosis. The one-time treatment showed consistently rapid, deep, and durable reduction in serum TTR, with evidence of disease stabilization or improvement. In the ATTR-CM arm, patients demonstrated favorable trends across cardiac disease markers at month 12, with a mean 90% serum TTR reduction. The ATTRv-PN arm showed similar positive trends with 91% TTR reduction at month 12. The therapy maintained a favorable safety profile, with mainly mild to moderate infusion-related reactions reported.
Intellia Therapeutics (NASDAQ:NTLA) reported Q3 2024 financial results and pipeline progress. Key highlights include FDA clearance for MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran in ATTR amyloidosis, strong enrollment in MAGNITUDE Phase 3 study, and active patient screening in HAELO Phase 3 study for hereditary angioedema. The company ended Q3 with $944.7 million in cash. Financial results show $9.1 million in collaboration revenue, R&D expenses of $123.4 million, and a net loss of $135.7 million. Cash runway extends into late 2026.
Intellia Therapeutics (NASDAQ:NTLA) has granted inducement awards to ten new employees under its 2024 Inducement Plan. The grants include 73,459 restricted stock units (RSUs) of common stock, which will vest in three equal installments on November 1, 2025, 2026, and 2027. These awards were granted outside the company's stockholder-approved equity incentive plans and were approved by Intellia's compensation committee in accordance with Nasdaq Listing Rule 5635(c)(4).
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