Core Viewpoint - Intellia Therapeutics (NTLA) has demonstrated significant stock performance, with a 29.8% increase over the past month, outperforming the industry and the S&P 500 index [1][7]. Company Performance - The company's stock performance is attributed to advancements in its pipeline development and increased interest in gene therapies following Eli Lilly's acquisition of Verve Therapeutics [3][4]. - Intellia is transitioning towards becoming a commercial-ready organization by the end of 2026 [9]. Pipeline Development - Intellia is advancing two late-stage in vivo candidates: nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis and lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (HAE) [4][8]. - Nex-z is currently undergoing two phase III studies, with enrollment in the MAGNITUDE 2 study expected to complete by 2026 and a potential biologics license application planned for 2028 [5][6]. - Lonvo-z is in the pivotal phase III HAELO study, with enrollment expected to complete by Q3 2025 and a regulatory filing planned for the second half of 2026 [8]. Competitive Landscape - Intellia faces competition from other companies utilizing CRISPR/Cas9 technology, such as CRISPR Therapeutics, which recently received approval for its gene-edited therapy, Casgevy [12][13]. - The company has decided to halt the development of its in vivo gene insertion candidate, NTLA-3001, and plans to reduce its workforce by nearly 27% in 2025 [13]. Financial Outlook - NTLA shares currently trade at a price/book ratio of 1.43, below the industry average of 3.13 [14]. - The Zacks Consensus Estimate for NTLA's loss per share has narrowed for both 2025 and 2026, indicating a potential improvement in financial performance [16]. Conclusion - Intellia's recent stock rally is promising, but the lack of an approved product remains a significant challenge [17]. - The successful development of its pipeline candidates is crucial for the company's future [17][18].

NTLA-2002 (Hereditary Angioedema - HAE) - NTLA-2002 aims to be the first to offer lifelong freedom from attacks and prophylaxis after a single dose for HAE, with a BLA submission planned in 2026[8] - Phase 2 study results showed 73% of patients were attack-free and off chronic prophylaxis after a single dose of NTLA-2002 at 16 weeks[37, 41] - The global market for HAE is projected to reach approximately $5 billion by 2028[8, 44] Nex-z (Transthyretin Amyloidosis - ATTR) - Nex-z aims to be the first to stabilize or reverse disease progression with a single dose for ATTR, with a BLA submission planned in 2028 for polyneuropathy[8] - Phase 1 data showed deep, rapid, and durable reductions in serum TTR, with an 89% reduction in ATTR-CM and a 90% reduction in ATTRv-PN at day 28[63] - The global market for ATTR is projected to reach approximately $12 billion by 2028[8, 86] Clinical Development and Milestones - Intellia expects three commercial launches starting in 2027[8] - The company plans to complete enrollment in the Phase 3 HAELO trial for NTLA-2002 in the second half of 2025 and the MAGNITUDE-2 trial for ATTRv-PN in 2026[2] - Intellia plans to enroll at least 550 patients in the Phase 3 MAGNITUDE trial for ATTR-CM in 2025 and complete enrollment in early 2027[2]

An updated edition of the May 21, 2025, article.The promising field of genomics has undergone rapid evolution over the past decade. Genomics includes the study of a complete set of genes, their work process and way of interacting with each other and the environment. The primary focus is to evaluate all the genes of an organism rather than individual genes.This genetic information is being used to evaluate individual responses to drugs, leading to the development of targeted therapies. This, in turn, has pav ...

Core Insights - The recent personnel changes at the FDA's CBER have caused fluctuations in the stock prices of gene editing companies, with a notable decline in Capricor Therapeutics due to its association with the suspended officials [1][2] Group 1: Personnel Changes - The CBER's cell and gene therapy department experienced significant personnel changes, with the director Nicole Weldon and her deputy Rachel Anatol being placed on administrative leave [1] - These changes occurred shortly after the appointment of the new CBER director, Vinay Prasad, who replaced the previously departed Peter Marks [1] - The HHS spokesperson indicated that there were management philosophy disagreements between the suspended officials and Prasad [1] Group 2: Market Reactions - Despite the turmoil at CBER, leading gene editing companies such as Editas Medicine, Intellia Therapeutics, Beam Therapeutics, and CRISPR Therapeutics saw their stock prices rise initially [2] - Conversely, Capricor Therapeutics, which is advancing the cell therapy deramiocel for Duchenne muscular dystrophy, experienced a stock price drop of over 10% due to the involvement of the suspended officials in its drug application process [2]

Core Insights - Intellia Therapeutics announced promising three-year follow-up data from the Phase 1 portion of its ongoing Phase 1/2 study for lonvoguran ziclumeran (lonvo-z) in patients with hereditary angioedema (HAE) [1][2] - All 10 patients in the Phase 1 study were attack-free and treatment-free for a median of nearly two years, demonstrating the potential of lonvo-z as a one-time therapy [2][6] - The global Phase 3 HAELO trial has completed screening ahead of schedule, with over half of the patients screened in the United States [4][6] Clinical Results - A single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% compared to pre-treatment baseline [2][6] - Patients showed deep, dose-dependent, and durable reductions in plasma kallikrein protein levels throughout the study [2][6] - The treatment was well tolerated, with the most frequent adverse events being infusion-related reactions, which were mostly Grade 1 and resolved without complications [3][6] Development Plans - The ongoing Phase 3 HAELO trial is randomized, double-blind, and placebo-controlled, assessing the safety and efficacy of lonvo-z at the 50 mg dosage [4][5] - Intellia plans to submit a biologics license application (BLA) in 2026, aiming for a U.S. launch in 2027 [4][8] - New data from the Phase 2 portion of the ongoing Phase 1/2 study is expected to be presented in the second half of 2025 [4][10] About Lonvo-z - Lonvo-z is based on CRISPR/Cas9 technology and aims to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene [8] - The therapy has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the FDA [8][9] - Intellia is focused on leveraging gene editing technology to develop novel therapies that address unmet medical needs [9]

Intellia Therapeutics (NTLA) FY 2025 Annual General Meeting June 11, 2025 09:00 AM ET Speaker0 Good day, and welcome to the Intellia Therapeutics Incorporated twenty twenty five Annual Meeting of Stockholders. I would now like to turn the conference over to Doctor. Frank Furweil, Chairperson of the company's Board of Directors, to proceed with the twenty twenty five Annual Meeting of Stockholders. Doctor. Verwil, please go ahead. Speaker1 Thank you. Good morning. I'm Doctor. Frank Verwil, Chairperson of the ...

Shares of Intellia Therapeutics, Inc. (NTLA) have gained 6.1% over the past four weeks to close the last trading session at $7.69, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $38.32 indicates a potential upside of 398.3%.The average comprises 25 short-term price targets ranging from a low of $7 to a high of $106, with a standard deviation of $24.94. While the lowest estimate ...

Core Viewpoint - Intellia Therapeutics' shares fell by 22.9% following an update from its phase III study on the investigational gene-editing candidate, nexiguran ziclumeran (nex-z), for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM) [1] Group 1: Study Updates and Safety Concerns - A patient in the phase III MAGNITUDE study experienced grade 4 liver transaminase elevations, indicating a significant increase in liver enzymes, although the patient was asymptomatic and the issue resolved without hospitalization [2] - Investor concerns regarding the long-term safety of the gene therapy candidate have contributed to the stock's decline, with shares down 36.1% year-to-date compared to the industry's decline of 5.4% [3] Group 2: Pipeline Developments - Intellia is also developing nex-z for ATTR amyloidosis with polyneuropathy (ATTRv-PN), with the first patient dosed in the phase III MAGNITUDE 2 study in April, and enrollment expected to be completed by 2026 [5] - Upon successful completion of the MAGNITUDE 2 study, the company plans to submit a biologics license application for nex-z in ATTRv-PN by 2028, in collaboration with Regeneron Pharmaceuticals, which shares 25% of development costs and commercial profits [6] Group 3: Other Pipeline Candidates - Intellia is developing NTLA-2002 for hereditary angioedema (HAE), with the first patient dosed in the pivotal phase III HAELO study in January 2025, and enrollment expected to be completed by Q3 2025 [7][8] - A potential biologics license application for NTLA-2002 in HAE is planned for submission in the second half of 2026, although the complexity of developing these CRISPR-based therapies poses challenges [8]

Intellia Therapeutics Inc NTLA said on Wednesday that a single, recent, asymptomatic patient has had grade 4 (severe) liver transaminase elevations (liver enzymes) based on laboratory tests in its Phase 3 MAGNITUDE study.The trial is evaluating nexiguran ziclumeran (nex-z, also known as NTLA-2001) in transthyretin amyloidosis with cardiomyopathy.These elevations appear to be resolving without hospitalization or medical intervention and have fallen to grade 3 ALT and grade 2 AST elevations.The company has en ...

Core Viewpoint - The S&P 500 has experienced persistent volatility in 2025, leading to cautious investor sentiment, but this environment may present opportunities for significant gains for those willing to take moderate risks [1]. Group 1: Septerna Inc. - Septerna Inc. is a clinical-stage biotechnology firm focused on developing G protein-coupled receptor (GPCR) oral small molecule drug candidates for various diseases [4][6]. - The company has secured a major partnership with Novo Nordisk, which could enhance its growth potential, with an immediate payment of approximately $200 million and a deal potentially worth over $2 billion [5][6]. - Analysts have a consensus price target of $27 per share for Septerna, indicating a potential upside of 184.81% from the current price of $9.48, despite the stock having fallen by about 58% year-to-date [7][4]. Group 2: Intellia Therapeutics Inc. - Intellia Therapeutics is a genome editing firm developing therapies for conditions such as hemophilia and cancers, with promising trial data reported for its ongoing Phase 1 trial [9][10]. - The company has a strong cash position of $707 million as of March 31, 2025, which is expected to fund operations through early 2027 [10]. - The consensus price target for Intellia shares is $36.90, suggesting a potential quadrupling of the stock's current price, with 14 out of 21 analysts rating the stock as a Buy [11]. Group 3: Novavax Inc. - Novavax has received full FDA approval for its protein-based COVID-19 vaccine, unlocking a $175 million milestone payment from Sanofi, positioning it as a non-mRNA option [12][13]. - Despite the approval, the FDA has imposed significant restrictions that may limit the vaccine's availability, contributing to mixed market reactions [13]. - The consensus price target for Novavax shares is $19, indicating a potential upside of 160.63%, with four out of seven analysts rating the stock as a Buy [12][13].