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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, Inc. (NASDAQ:NTLA) is set to present its first preclinical data on a new cytosine deaminase base editor technology at the Cold Spring Harbor Laboratory virtual meeting, running from March 24-26, 2021. The data highlights the ability of the company’s proprietary base editors to enable multiple gene knockouts without increasing translocation rates. CEO John Leonard emphasizes the expansion of their genome editing platform for developing therapies for severe diseases. The presentation details include an impressive T cell editing efficiency of over 90% while maintaining standard translocation levels.
Intellia Therapeutics (NASDAQ: NTLA) showcased promising preclinical data on March 10, 2021, regarding non-viral genome editing of hematopoietic stem cells (HSCs) in mouse models. This innovative approach aims to address inherited blood disorders such as sickle cell disease (SCD). Key findings included durable, dose-dependent gene editing in bone marrow and HSCs, with effects lasting over a year. Intellia's proprietary lipid nanoparticles facilitated the safe delivery of CRISPR/Cas9, potentially revolutionizing treatment options for patients globally without the need for invasive procedures.
Intellia Therapeutics (NASDAQ:NTLA) reported operational and financial results for Q4 2020, ending with a cash position of $597.4 million. The company successfully dosed its first patient with NTLA-2001, a CRISPR-based therapy for transthyretin amyloidosis (ATTR). It expects to report initial clinical data on NTLA-2001 this year and plans to submit IND applications for NTLA-5001 (AML) and NTLA-2002 (HAE) in mid-2021. R&D expenses increased to $38.2 million, while net loss was $42.2 million for Q4 2020, compared to $28.3 million in Q4 2019.
Intellia Therapeutics (NASDAQ:NTLA) will announce its fourth quarter and full-year 2020 financial results on February 25, 2021, at 8 a.m. ET. The company focuses on developing curative therapeutics utilizing CRISPR/Cas9 technology for both in vivo and ex vivo applications. Interested participants can join the conference call by dialing the provided numbers. A replay of the call will be available later that day on the company's website. Intellia's mission is to produce permanent gene edits and engineered cells to treat various diseases.
Intellia Therapeutics (NASDAQ:NTLA) is advancing its global Phase 1 study of NTLA-2001, a single-course therapy for transthyretin amyloidosis (ATTR). The company plans to submit an IND for NTLA-5001, targeting acute myeloid leukemia (AML), by mid-2021, and another IND for NTLA-2002 for hereditary angioedema (HAE) in the second half of 2021. Intellia also aims to nominate at least one new candidate in 2021. With a strong cash position of $597 million at the end of 2020, the company is well-positioned to fund operations for the next 24 months.
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.
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