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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 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.
Intellia Therapeutics (NTLA) recently announced significant progress in its Phase 1 study of NTLA-2001 for transthyretin (ATTR) amyloidosis. The company completed the dose-escalation portion for cardiomyopathy, with interim results showing deep, sustained TTR reductions for up to 12 months following a single dose. Intellia plans to focus solely on allogeneic cell therapies moving forward, including NTLA-5001. Financially, it ended Q2 2022 with $907 million in cash but reported a net loss of $100.7 million, up from $68.8 million in Q2 2021.
Intellia Therapeutics (NASDAQ:NTLA) will announce its Q2 2022 financial results on August 4, 2022, at 8 a.m. ET. The conference call will allow participants to discuss the operational highlights and financial metrics from the quarter. Interested parties can join via U.S. and international calling options, with a webcast available online. After the call, a replay will be accessible on Intellia's website. The company specializes in genome editing using CRISPR technologies, aiming to develop curative treatments for diseases.
Intellia Therapeutics and Regeneron Pharmaceuticals announced positive interim results from the Phase 1 study of NTLA-2001, a CRISPR-based therapy for ATTR amyloidosis. The study showed sustained serum transthyretin (TTR) reductions of 93% by day 28 at the 1.0 mg/kg dose, remaining durable through six months. Follow-up data from patients indicate a favorable safety profile, with the majority of adverse events being mild. The companies plan further development, aiming for an 80 mg fixed dose in the next study phase.
Intellia Therapeutics (NTLA) and Regeneron Pharmaceuticals (REGN) released promising interim data from a Phase 1 study of NTLA-2001, a CRISPR-based treatment for transthyretin amyloidosis. Results showed sustained reductions in serum transthyretin (TTR) levels, with a 93% mean reduction at the highest dose by day 28, maintained over six months. The treatment was well-tolerated, mostly causing mild adverse events. An investor event is set to discuss the findings further. The study continues to explore NTLA-2001's potential as a one-time therapeutic solution.
Intellia Therapeutics (NASDAQ:NTLA) plans to present interim data from the Phase 1 study of NTLA-2001 at the International Liver Congress 2022. This investigational therapy targets hereditary ATTR amyloidosis, aiming to demonstrate a durable response from a single dose. Initial results showed up to 98% reduction in serum TTR levels in some patients. The presentation on June 24 will include new durability data and fixed dose selection for the ongoing expansion cohort. NTLA-2001 could become the first single-dose treatment for ATTR amyloidosis, highlighting Intellia's advancements in CRISPR technology.
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