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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 new interim clinical data from its Phase 1/2 study of NTLA-2002, showcasing safety, kallikrein reduction, and attack rate information. This investigational CRISPR/Cas9 therapy aims to prevent angioedema attacks in hereditary angioedema (HAE) patients. Key presentation details include:
- Date: Nov 12, 2022
- Presenter: Dr. Hilary Longhurst
A live webcast to discuss the data will be held on Nov 14, 2022. NTLA-2002 represents a novel approach to a rare genetic disorder affecting 1 in 50,000 people.
Intellia Therapeutics, Inc. (NASDAQ:NTLA) will present its Q3 2022 financial results and operational highlights on a conference call scheduled for November 3, 2022, at 8 a.m. ET. Investors can participate by dialing 1-833-316-0545 (U.S.) or 1-412-317-5726 (international). A live webcast will also be available on the company’s website. Intellia focuses on developing genome editing therapies through CRISPR technology, aiming to address various diseases with its in vivo and ex vivo programs.
Intellia Therapeutics (NASDAQ:NTLA) announced upcoming presentations at two investor conferences in October 2022. The Chardan’s 6th Annual Genetic Medicines Conference is scheduled for October 3, 2022, at 8:30 a.m. ET in a fireside chat format. The 2022 Truist Securities Genetic Medicine Summit will include a genome editing panel on October 20, 2022, at 9:00 a.m. ET. A live webcast of the Chardan presentation will be available on Intellia's website, with a replay accessible for 30 days.
Intellia Therapeutics announced promising interim results from the Phase 1 study of NTLA-2001, a CRISPR/Cas9 therapy for transthyretin (ATTR) amyloidosis. At doses of 0.7 mg/kg and 1.0 mg/kg, mean serum transthyretin (TTR) reductions of 93% and 92% were observed at day 28. The treatment was well-tolerated, with transitory infusion reactions as the only adverse events. These outcomes suggest NTLA-2001 may provide a single-dose treatment option for patients with cardiomyopathy manifestations of ATTR amyloidosis. Further discussions on this data are scheduled for an investor event today.
Intellia Therapeutics and Regeneron Pharmaceuticals announced promising interim results from the Phase 1 study of NTLA-2001, a CRISPR therapy for transthyretin (ATTR) amyloidosis. The trial exhibited significant mean serum transthyretin reductions of 93% and 92% at doses of 0.7 mg/kg and 1.0 mg/kg, respectively, on day 28. The treatment was well-tolerated, with minimal adverse reactions. Follow-ups indicated sustained reductions over two to six months. Intellia plans to expand the study with a fixed dose of 0.7 mg/kg and aims for a pivotal trial, potentially including U.S. patients.
Intellia Therapeutics (NASDAQ:NTLA) announced positive interim results from its Phase 1/2 study of NTLA-2002, a CRISPR-based treatment for hereditary angioedema (HAE). A single dose of 25 mg resulted in a 91% reduction in HAE attacks over 16 weeks, with two patients remaining attack-free. At 75 mg, the treatment achieved a 92% reduction in plasma kallikrein levels. NTLA-2002 was well-tolerated, with mild adverse events. The company plans to proceed with a Phase 2 dose-expansion in H1 2023. These findings validate Intellia's genome editing platform, highlighting its potential to address various genetic diseases.
SparingVision, a French biotech focused on genomics in ophthalmology, has secured Series B financing co-led by Jeito Capital and the University of Pittsburgh Medical Center Enterprise. The $XX million funding will accelerate development of its lead treatments, SPVN06 and SPVN20, targeting retinitis pigmentosa. The investment supports first-in-human studies and further innovations in their pipeline. SparingVision aims to bring mutation-agnostic genomic therapies to patients suffering from inherited retinal diseases and dry age-related macular degeneration.
Intellia Therapeutics announced a virtual investor event on September 16, 2022, to present interim data from its ongoing Phase 1/2 studies of NTLA-2002 and NTLA-2001. NTLA-2002 aims to treat hereditary angioedema and will provide initial safety and kallikrein protein reduction data. NTLA-2001 targets ATTR amyloidosis, and an update on its cardiomyopathy arm will also be shared. Both candidates utilize CRISPR technology for gene editing, showing promise in their respective clinical trials. This event will be accessible online, and a replay will be available for 30 days.
Intellia Therapeutics, Inc. (NASDAQ:NTLA) announced that the FDA has granted orphan drug designation for its CRISPR-based genome editing candidate, NTLA-2002, aimed at treating hereditary angioedema (HAE). This candidate seeks to prevent HAE attacks after a single dose by knocking out the KLKB1 gene, reducing plasma kallikrein activity. Currently in a Phase 1/2 study, interim data will be presented on September 16, 2022, at the Bradykinin Symposium. Orphan designation provides several incentives, including tax credits and potential marketing exclusivity.
Intellia Therapeutics (NASDAQ:NTLA) announced the first clinical data on NTLA-2002, their novel CRISPR candidate, set for oral presentation at the Bradykinin Symposium on September 16, 2022. NTLA-2002 aims to prevent angioedema attacks in patients with hereditary angioedema (HAE) through a single-dose treatment strategy. The presentation will cover interim safety data, kallikrein reduction, and attack rates from the Phase 1/2 study. This innovative therapy represents a significant advancement in tackling the challenges associated with HAE, a rare genetic disorder affecting approximately 1 in 50,000 individuals.