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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 presented key findings at the 29th Annual Congress of the European Society of Gene & Cell Therapy. The company showcased its proprietary allogeneic T cell therapy platform designed to address immune rejection in therapeutic candidates for cancer and autoimmune diseases. Significant advancements include a lipid nanoparticle-based CRISPR/Cas9 delivery system and the first reported consecutive in vivo gene insertion and knockout in non-human primates aimed at treating alpha-1 antitrypsin deficiency (AATD). This work positions Intellia at the forefront of genome editing therapeutics.
Intellia Therapeutics has received authorization from New Zealand's MEDSAFE to initiate a Phase 1/2 clinical study for NTLA-2002, a CRISPR/Cas9-based therapeutic aimed at treating hereditary angioedema (HAE). NTLA-2002 is designed to inactivate the KLKB1 gene, potentially providing a one-time treatment to prevent debilitating attacks associated with HAE. Patient enrollment is expected to start by year-end, with further regulatory applications planned globally. The program builds on insights gained from previous trials on NTLA-2001 for ATTR amyloidosis.
Intellia Therapeutics (NASDAQ:NTLA) will present at Chardan’s 5th Annual Genetic Medicines Conference on October 4, 2021, at 10:30 a.m. ET. This clinical-stage genome editing company is focused on developing curative therapeutics using CRISPR/Cas9 technology through in vivo and ex vivo methods. A live webcast of the presentation will be available on their website approximately 15 minutes prior to the start. A replay will be accessible for 14 days following the event.
Intellia Therapeutics (NASDAQ:NTLA) announced the FDA's acceptance of the IND application for NTLA-5001, its first ex vivo CRISPR-engineered therapy targeting Wilms’ Tumor 1 (WT1) for acute myeloid leukemia (AML). This milestone paves the way for a Phase 1/2a clinical study, expected to start patient screening by year-end, focusing on AML patients post-first-line therapy. The therapy aims to improve the five-year survival rate, currently below 30%. The study will involve up to 54 participants across two dosing categories, assessing safety and anti-tumor efficacy.
Intellia Therapeutics (NASDAQ:NTLA), a clinical-stage genome editing company, will participate in two virtual healthcare investor conferences in September 2021. The first will be at the Wells Fargo Healthcare Conference on September 9 at 10:40 am ET, followed by the H.C. Wainwright 23rd Annual Global Investment Conference on September 13 at 7:00 am ET. Live webcasts of the presentations will be available on Intellia's website, with replays accessible for 14 days post-event. Intellia focuses on curative therapeutics powered by CRISPR/Cas9 technology.
Intellia Therapeutics has made significant strides in its CRISPR gene-editing programs, with promising interim Phase 1 data for NTLA-2001 showing a maximum 96% reduction in serum TTR levels for ATTR amyloidosis. The company is poised to initiate Phase 1 trials for NTLA-2002 and NTLA-5001 this year. Financially, Intellia ended Q2 2021 with $551.3 million in cash and raised an additional $648.1 million from a follow-on offering. Despite a net loss of $68.8 million, Intellia's strong financial position supports ongoing and future development efforts.
Intellia Therapeutics (NASDAQ:NTLA) will present its Q2 2021 financial results and operational highlights on August 5, 2021, at 8 a.m. E.T. The conference call will provide insights into its progress in developing CRISPR/Cas9-based therapeutics. Interested participants can join via U.S. or international dialing, with a live webcast available. A replay will be accessible on Intellia's website starting noon E.T. on the same day. The company focuses on curative therapies using both in vivo and ex vivo genome editing techniques.
Intellia Therapeutics (NASDAQ:NTLA) has completed a public offering of 4,758,620 shares at $145.00 each, raising approximately $690 million, following the full exercise of the underwriters' option for 620,689 additional shares. The offering was managed by Goldman Sachs, Jefferies, SVB Leerink, and Barclays. Shares were offered under a shelf registration statement effective with the SEC. This capital is expected to support the development of Intellia's CRISPR/Cas9-based therapeutics. The press release includes forward-looking statements subject to market uncertainties.
Intellia Therapeutics (NASDAQ:NTLA) announced the pricing of a public offering of 4,137,931 shares at $145.00 per share, aiming to raise approximately $600 million before expenses. The offering includes a 30-day option for underwriters to purchase up to an additional 620,689 shares. The offering is set to close around July 2, 2021, subject to conditions. Goldman Sachs, Jefferies, SVB Leerink, and Barclays are the joint managers for this offering, which is conducted under an effective SEC shelf registration statement.
Intellia Therapeutics (NASDAQ:NTLA) announced a public offering of $400 million in common stock, with an option for underwriters to purchase an additional 15%. The company aims to utilize proceeds for advancing its CRISPR/Cas9 genome editing technology. Goldman Sachs & Co. LLC, Jefferies, SVB Leerink, and Barclays are joint book-running managers for the offering. The offering is contingent on market conditions and may change. The shares will be sold under an effective registration statement filed with the SEC, ensuring compliance with securities laws.