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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 progress in its gene therapy for alpha-1 antitrypsin (AAT) deficiency at the Alpha-1 Foundation’s workshop. The company demonstrated that a single course of its CRISPR/Cas9-based therapy can durably restore human AAT protein levels in non-human primates. This innovative approach could offer a potentially curative treatment for AAT deficiency, which causes liver and lung dysfunction. The stable AAT levels observed over 11 weeks indicate the treatment's promise, reinforcing Intellia's commitment to advancing its genome editing technologies for rare genetic disorders.
Intellia Therapeutics presented promising preclinical results for NTLA-5001, a TCR-T cell therapy targeting WT1 in acute myeloid leukemia (AML), at the 62nd ASH Annual Meeting. The lead candidate demonstrated superior anti-tumor activity in mouse models compared to standard methods. The company plans to submit an IND application in H1 2021 and initiate a first-in-human trial to assess safety and efficacy in AML patients. NTLA-5001 aims at treating AML patients irrespective of genetic subtypes, with potential future applications for WT1-positive solid tumors.
Intellia Therapeutics (NASDAQ:NTLA) announced the successful closing of a public offering of 5,513,699 shares at $36.50 per share, raising approximately $201 million before deductions. This includes the full exercise of the underwriters' option for an additional 719,178 shares. Goldman Sachs, Jefferies, and SVB Leerink were the joint book-running managers. The offering was conducted under a previously filed shelf registration statement with the SEC. The funds are expected to enhance Intellia's genome editing technology aimed at developing curative therapeutics.
Intellia Therapeutics (NASDAQ:NTLA) announced a public offering of 4,794,521 shares at $36.50 each, aiming to raise approximately $175 million before expenses. The underwriters, including Goldman Sachs, Jefferies, and SVB Leerink, hold a 30-day option to purchase an additional 719,178 shares. The offering is set to close around December 4, 2020. The shares are being offered under a previously filed shelf registration statement. This financing aims to support Intellia's development of CRISPR-based therapeutics.
Intellia Therapeutics (NASDAQ:NTLA) announced a public offering of $150 million in common stock, with an option for underwriters to purchase an additional 15% of shares. The offering is underwritten by Goldman Sachs, Jefferies, and SVB Leerink and is subject to market conditions. Shares are being sold under a previously filed shelf registration with the SEC. The company focuses on developing CRISPR/Cas9-based therapeutics. The completion of the offering depends on various factors, and the offering's size and terms may change.
Intellia Therapeutics (NASDAQ: NTLA) has received a grant from the Bill & Melinda Gates Foundation to develop non-viral treatments for sickle cell disease (SCD) using its CRISPR/Cas9 genome editing technology. This initiative aims to create safe and scalable solutions for SCD over the next 7-10 years, particularly addressing the disease's impact in developing countries. The funding will support preclinical validation of Intellia’s non-viral delivery systems for genome editing. SCD affects millions globally, with a high incidence in sub-Saharan Africa and India.
Intellia Therapeutics (NASDAQ:NTLA) announced the treatment of the first patient with NTLA-2001, a revolutionary potential therapy for transthyretin amyloidosis (ATTR). This groundbreaking single-course therapy utilizes CRISPR technology to potentially halt and reverse disease progression. The Phase 1 study focuses on hereditary ATTR with polyneuropathy, aiming for future trials across broader ATTR patient groups. Intellia's robust plans include rapid movement to pivotal studies, establishing NTLA-2001 as a leading candidate in curative treatments for ATTR.
Intellia Therapeutics (NTLA) announced significant progress in its clinical programs, notably the initiation of a Phase 1 trial for NTLA-2001, targeting transthyretin amyloidosis (ATTR), with patient dosing expected by year-end 2020. The company also aims to submit IND applications for NTLA-5001 (acute myeloid leukemia) in 1H 2021, and NTLA-2002 (hereditary angioedema) in 2H 2021. Financially, Intellia ended Q3 2020 with $407.9 million in cash, marking a healthy increase from the previous year, despite a net loss of $27.8 million. Collaboration revenue rose significantly, driven by the Regeneron partnership.
Intellia Therapeutics (NASDAQ:NTLA) will present its Q3 2020 financial results and operational highlights on November 5, 2020, at 8 a.m. ET. Interested participants can join the call by dialing 1-877-317-6789 in the U.S. or 1-412-317-6789 internationally. A replay will be available post-call on the company's website from 12 p.m. ET the same day. Intellia is focused on developing curative therapeutics using CRISPR/Cas9 technology, aiming to revolutionize medicine by permanently editing disease-related genes with a single treatment.
Intellia Therapeutics (NASDAQ: NTLA) announced the authorization of its Clinical Trial Application to commence a Phase 1 study of NTLA-2001, targeting hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). This therapy, potentially the first curative treatment for ATTR, utilizes in vivo CRISPR/Cas9 technology. The trial aims to assess safety, pharmacokinetics, and efficacy, with expectations to dose the first patient by the end of 2020. Intellia plans to expand the study after determining the optimal dose and further regulatory applications in other countries.
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