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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 (NTLA) has announced the dosing of the first patient with NTLA-5001, an investigational T cell receptor (TCR) therapy aimed at treating acute myeloid leukemia (AML). This CRISPR/Cas9 engineered therapy targets the Wilms' Tumor 1 (WT1) antigen, frequently found in AML and other cancers. The Phase 1/2a study will assess the therapy's safety, efficacy, and anti-tumor activity. The trial has two arms, focusing on AML patients with varying disease burdens. With over 20,000 new AML cases annually in the U.S., NTLA-5001 represents a significant advancement in cancer treatment.
On February 28, 2022, Intellia Therapeutics and Regeneron Pharmaceuticals announced promising interim results from a Phase 1 clinical trial of NTLA-2001 for treating transthyretin amyloidosis (ATTR). The study demonstrated a mean serum TTR reduction of 93% at the highest dose (1.0 mg/kg) by day 28, with durable reductions lasting between two to twelve months. The treatment was generally well tolerated, with mild adverse effects reported. The companies plan to advance the therapy into a dose-expansion cohort in Q1 2022, marking a significant step in CRISPR-based genomic medicine.
Intellia Therapeutics announced promising interim results from a Phase 1 trial of NTLA-2001, aimed at treating transthyretin (ATTR) amyloidosis. A mean serum TTR reduction of 93% was achieved at the 1.0 mg/kg dose by day 28, with durable effects sustained for 2 to 12 months. The treatment was generally well tolerated, with most adverse events mild. NTLA-2001 is the first CRISPR-based therapy administered systemically, marking a significant advancement in genome editing. Intellia plans to initiate a polyneuropathy dose-expansion cohort in Q1 2022 and will host an investor event to discuss these findings.
Intellia Therapeutics (NASDAQ: NTLA) reported its Q4 2021 results, showcasing significant growth and strategic advancements in its CRISPR-based pipeline. The company plans to present additional interim data from its Phase 1 study on NTLA-2001 for ATTR amyloidosis on February 28, 2022. Notably, Intellia announced two new candidates, NTLA-6001 for hematologic cancers and NTLA-2003 for liver-related AATD. With $1.086 billion in cash as of year-end 2021, up from $597.4 million in 2020, Intellia is well-positioned for future developments. Net loss rose to $81.2 million, emphasizing the costs of advancing multiple clinical programs.
Intellia Therapeutics (NASDAQ:NTLA) has announced a lease agreement for a new 140,000-square-foot GMP manufacturing facility in Waltham, Massachusetts, expected to be operational in 2024. This facility will support the production of components for its CRISPR-based investigational therapies, enhancing manufacturing capacity and capability as the company advances its pipeline. Intellia's Chief Technical Officer emphasized the strategic importance of this investment for efficient production, while the Chief Financial Officer noted the benefits of a long-term lease over building a facility.
Intellia Therapeutics (NASDAQ:NTLA) has announced two virtual investor events in February 2022. The first is the Fourth Quarter and Full-Year 2021 Financial Results, scheduled for February 24 at 8:00 a.m. ET. U.S. callers can dial 1-833-316-0545 to join. The second event is an update on interim clinical data for NTLA-2001, set for February 28 at 4:30 p.m. ET, featuring presentations from leading experts. NTLA-2001 aims to treat ATTR amyloidosis using CRISPR technology, potentially allowing for a single-dose treatment option.
Intellia Therapeutics (NTLA) and ONK Therapeutics have entered a collaboration to develop CRISPR-edited NK cell therapies for cancer treatment. ONK receives non-exclusive rights to Intellia's ex vivo genome editing and delivery technologies for up to five therapies, while Intellia is eligible for $184 million per product in milestone payments and royalties. The agreement allows for co-development of two therapies, with Intellia holding lead commercialization rights in the U.S. This collaboration aims to enhance NK cell efficacy against various cancers.
Intellia Therapeutics (NASDAQ:NTLA) announced its acquisition of Rewrite Therapeutics for $45 million upfront, plus up to $155 million in milestones. This strategic acquisition enhances Intellia's genome editing capabilities, integrating Rewrite's novel DNA writing platform. Rewrite’s technology promises precise gene editing solutions, including targeted corrections and edits in non-dividing cells, facilitating potential advancements in genomic medicine. The merger is expected to expand Intellia’s therapeutic applications, aligning with its mission to develop curative CRISPR-based therapeutics.
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.
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