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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 (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.
Intellia Therapeutics (NASDAQ:NTLA) announced positive interim data from its Phase 1 study of NTLA-2001 for ATTR amyloidosis, showing a 93% mean serum TTR reduction at 1.0 mg/kg by day 28. The company initiated Part 2 of the study and will present further data in June 2022. NTLA-2002 for hereditary angioedema is progressing with interim data expected in 2H 2022. The company reported a strong cash position of $995 million but posted a net loss of $146.9 million in Q1 2022, driven by R&D expenses. Intellia is on track to advance multiple candidates into regulatory submissions.
Intellia Therapeutics (NASDAQ:NTLA) has appointed Muna Bhanji, R.Ph., to its board of directors, enhancing its leadership team as it advances its genome editing initiatives. With over 30 years of experience in the biopharmaceutical industry, Bhanji brings valuable expertise in global commercialization and market access.
Her background includes senior roles at Merck & Co. and founding Tiba Global Access. This strategic appointment aims to bolster Intellia's efforts in developing CRISPR-based therapeutics for life-threatening diseases.
Intellia Therapeutics (NTLA) announced promising preclinical data supporting the development of NTLA-6001, an allogeneic CAR-T therapy targeting CD30-expressing hematologic cancers. Presenting at the Keystone Symposia, findings showed that their proprietary engineering platform produced immune-evading T cells, crucial for potent tumor responses. The preclinical data indicated NTLA-6001's potential in treating relapsed or refractory classical Hodgkin lymphoma. The candidate is progressing towards IND-enabling activities, reinforcing Intellia’s commitment to innovative genomic therapies.
Intellia Therapeutics (NASDAQ:NTLA) will announce its first quarter 2022 financial results on May 5, 2022, at 8 a.m. ET. The company, a leader in genome editing focused on CRISPR technology, will discuss operational highlights during a conference call. U.S. participants can dial 1-833-316-0545, while international callers should use 1-412-317-5726. A live webcast will also be available. Post-call, a replay will be accessible on Intellia’s website.
Intellia Therapeutics (NASDAQ: NTLA) announced that its investigational T cell receptor (TCR)-T cell therapy, NTLA-5001, has received orphan drug designation from the FDA for treating acute myeloid leukemia (AML). This therapy targets the Wilms’ Tumor 1 (WT1) antigen, prevalent in AML and other cancers. Currently in a Phase 1/2a clinical trial, NTLA-5001 aims to provide new treatment options for patients with recurrent AML. The orphan status supports development incentives, including potential market exclusivity and tax credits.
Intellia Therapeutics (NTLA) has announced the dosing of the first patient with NTLA-5001, an investigational T cell receptor (TCR) therapy aimed at treating acute myeloid leukemia (AML). This CRISPR/Cas9 engineered therapy targets the Wilms' Tumor 1 (WT1) antigen, frequently found in AML and other cancers. The Phase 1/2a study will assess the therapy's safety, efficacy, and anti-tumor activity. The trial has two arms, focusing on AML patients with varying disease burdens. With over 20,000 new AML cases annually in the U.S., NTLA-5001 represents a significant advancement in cancer treatment.
On February 28, 2022, Intellia Therapeutics and Regeneron Pharmaceuticals announced promising interim results from a Phase 1 clinical trial of NTLA-2001 for treating transthyretin amyloidosis (ATTR). The study demonstrated a mean serum TTR reduction of 93% at the highest dose (1.0 mg/kg) by day 28, with durable reductions lasting between two to twelve months. The treatment was generally well tolerated, with mild adverse effects reported. The companies plan to advance the therapy into a dose-expansion cohort in Q1 2022, marking a significant step in CRISPR-based genomic medicine.
Intellia Therapeutics announced promising interim results from a Phase 1 trial of NTLA-2001, aimed at treating transthyretin (ATTR) amyloidosis. A mean serum TTR reduction of 93% was achieved at the 1.0 mg/kg dose by day 28, with durable effects sustained for 2 to 12 months. The treatment was generally well tolerated, with most adverse events mild. NTLA-2001 is the first CRISPR-based therapy administered systemically, marking a significant advancement in genome editing. Intellia plans to initiate a polyneuropathy dose-expansion cohort in Q1 2022 and will host an investor event to discuss these findings.
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