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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) reported first-quarter 2021 results, ending March 31 with $600.8 million in cash. The company plans to announce initial safety data for NTLA-2001, a CRISPR-based therapy for transthyretin amyloidosis (ATTR), in mid-2021 and to submit IND applications for NTLA-5001 (acute myeloid leukemia) and NTLA-2002 (hereditary angioedema) in 2021. Research and development expenses increased to $39.3 million due to advancements in key programs. The company recorded a net loss of $46.2 million, up from $31.8 million year-over-year.
Intellia Therapeutics (NASDAQ:NTLA) announced it will present its first quarter 2021 financial results and operational highlights on May 6, 2021, at 8 a.m. ET. The conference call will allow investors to understand the company's progress in developing proprietary therapeutics using CRISPR/Cas9 technology. U.S. callers can dial 1-833-316-0545; international callers should use 1-412-317-5726. A replay will be available on the company's website later that day.
Intellia Therapeutics (NASDAQ:NTLA) announced updated preclinical data on CRISPR/Cas9-mediated gene insertion for treating alpha-1 antitrypsin deficiency (AATD) at the ASGCT Annual Meeting, held virtually from May 11-14, 2021. The findings suggest a potential to restore normal AAT protein levels after a single dose, showcasing Intellia's commitment to innovative treatments. Presentations from key executives will cover topics such as genome editing strategies and on-target safety assessments. Further information is expected to be released during the meeting.
Intellia Therapeutics (NASDAQ: NTLA) has appointed Dr. Georgia Keresty to its Board of Directors, enhancing its leadership team. Dr. Keresty brings over 35 years of pharmaceutical experience, having held senior roles in leading companies like Takeda, Johnson & Johnson, and Bristol-Myers Squibb. Her expertise encompasses transitioning therapeutic programs to clinical development and improving manufacturing processes. Intellia aims to leverage her knowledge as it advances its CRISPR/Cas9 technology towards curative therapies.
Intellia Therapeutics (NASDAQ:NTLA) has received orphan drug designation from the European Commission for NTLA-2001, aimed at treating transthyretin amyloidosis (ATTR). This investigational CRISPR therapy is the first of its kind to be delivered systemically via intravenous dosing, targeting the TTR gene responsible for producing misfolded proteins. This milestone is significant for the ATTR community, where no existing cure is available. The therapy shows potential to halt and reverse ATTR progression and is part of a co-development agreement with Regeneron.
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.
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