Latest news with #Intellia
Yahoo
08-07-2025
- Business
- Yahoo
The Next Moderna? This Clinical-Stage Biotech Has Breakout Potential.
Moderna (MRNA), an undervalued name in the biotech space, rose to prominence during the COVID-19 pandemic. This occurred when its mRNA-based COVID-19 vaccine was one of the first to be granted emergency-use authorization and distributed globally. This opportunity also validated Moderna's mRNA platform's effectiveness and scalability, boosting the company's stock price by more than 1,200% between 2020 and 2022. Another such clinical-stage contender generating growing excitement is Intellia Therapeutics (NTLA), a CRISPR/Cas9-based gene-editing company. Intellia aims to provide one-time, curative treatments for serious genetic diseases. Intellia's stock has fallen 1.9% year-to-date, while the broader market, as tracked by the S&P 500 Index ($SPX), has risen 6%. This Analyst Just Raised His Broadcom Stock Price Target by 70%. Should You Buy AVGO Now? Why Alibaba Stock Looks Like a Screaming Buy After Falling 27% From Its 2025 Highs 2 ETFs Offering Juicy Dividend Yields of 20% or Higher Markets move fast. Keep up by reading our FREE midday Barchart Brief newsletter for exclusive charts, analysis, and headlines. Let's dig deeper to see if this could be the next transformative biotech story that reshapes medicine — and markets — in the same way Moderna did. Intellia employs a dual-platform strategy, focusing on both in vivo (inside the patient's body) and ex vivo (outside the patient's body) approaches to treating conditions such as transthyretin amyloidosis (ATTR) and hereditary angioedema (HAE). Intellia's lead in vivo therapies include NTLA-2001 (Nexiguran ziclumeran or nex-z), a treatment for ATTR amyloidosis that is in the Phase 3 MAGNITUDE trial. Nex Z was also granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA), indicating its potential for accelerated development. NTLA-2002, a treatment for hereditary angioedema (HAE), is now in late-stage clinical trials. In the first quarter of 2025, Intellia administered the first dose in the HAELO trial. This event marks a critical inflection point, bringing Intellia closer to regulatory filings and possible approval. However, the company recently halted its NTLA-3001 lung disease candidate, as well as several other research programs. It also reduced its workforce by about 27%, causing a short-term disruption. The goal of this strategic reorganization is to increase focus on late-stage assets while conserving capital. While Intellia does not yet generate product revenue, it did generate $16.6 million in collaboration revenue during the first quarter. As of Q1 end, the company held $707.1 million in cash and executed a 27% workforce reduction, ensuring a runway until the first half of 2027. Intellia's first-mover advantage in in vivo gene editing could propel it to great success if its candidates are approved. The company has also collaborated with heavyweights like Regeneron (REGN), gaining expertise, resources, and a broader clinical reach. However, as a clinical-stage biotech company, the company faces the risk of clinical trials failing or delays in regulatory approvals because gene editing is a high-stakes game. Furthermore, increased cash burn may necessitate dilution. Long-term growth investors who believe in CRISPR's transformative potential may view NTLA stock as an enticing but high-risk investment opportunity. Overall, Wall Street has assigned Intellia stock a 'Moderate Buy' rating. Of the 27 analysts covering NTLA stock, 19 have a 'Strong Buy' recommendation, one suggests a 'Moderate Buy,' six say it is a 'Hold,' and one suggests a 'Strong Sell.' The average price target of $38.04 implies the stock has upside potential of 245% over current levels. Plus, the Street-high price estimate of $106 suggests the stock can rally over 860% over the next 12 months. While the upside appears out of reach, Moderna stock is a prime example of how a single successful product can propel a biotech stock toward the skies. Intellia, like Moderna, focuses on diseases with high clinical need and, if successful, has the potential to transform medicine. On the date of publication, Sushree Mohanty did not have (either directly or indirectly) positions in any of the securities mentioned in this article. All information and data in this article is solely for informational purposes. This article was originally published on
Yahoo
03-07-2025
- Business
- Yahoo
Citizens JMP Maintains Neutral Stance on Intellia Despite Positive Lonvo-z Data
Intellia Therapeutics, Inc. (NASDAQ:NTLA) ranks among the best CRISPR stocks to buy. Citizens JMP reaffirmed its Market Perform rating on Intellia Therapeutics, Inc. (NASDAQ:NTLA) on June 17. The update came in response to encouraging three-year follow-up data from the company's Phase I trial of NTLA-2002, now known as Lonvoguran ziclumeran (Lonvo-z), in patients with hereditary angioedema (HAE). The treatment possesses a solid safety record, with only brief side effects being noted. According to Citizens JMP, the findings 'underline durability and shows the value proposition to patients that can potentially live disease free.' However, despite the encouraging long-term results from the HAE therapy research, the firm believes that Intellia's stock is 'fairly valued' at current levels. Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a biotech company at the forefront of CRISPR-Cas9 gene-editing technology, developing cutting-edge treatments for both in vivo and ex vivo applications. Its main initiative, NTLA-2001, aims to treat transthyretin amyloidosis, a disease that is currently incurable. Targeting hereditary angioedema (HAE), NTLA-2002 is another promising medication in development. While we acknowledge the potential of NTLA as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. Read More: and Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
21-06-2025
- Business
- Yahoo
Will Intellia Therapeutics (NASDAQ:NTLA) Spend Its Cash Wisely?
Just because a business does not make any money, does not mean that the stock will go down. For example, although made losses for many years after listing, if you had bought and held the shares since 1999, you would have made a fortune. Nonetheless, only a fool would ignore the risk that a loss making company burns through its cash too quickly. Given this risk, we thought we'd take a look at whether Intellia Therapeutics (NASDAQ:NTLA) shareholders should be worried about its cash burn. In this report, we will consider the company's annual negative free cash flow, henceforth referring to it as the 'cash burn'. First, we'll determine its cash runway by comparing its cash burn with its cash reserves. We've found 21 US stocks that are forecast to pay a dividend yield of over 6% next year. See the full list for free. A cash runway is defined as the length of time it would take a company to run out of money if it kept spending at its current rate of cash burn. In March 2025, Intellia Therapeutics had US$504m in cash, and was debt-free. Looking at the last year, the company burnt through US$381m. Therefore, from March 2025 it had roughly 16 months of cash runway. Importantly, analysts think that Intellia Therapeutics will reach cashflow breakeven in 5 years. Essentially, that means the company will either reduce its cash burn, or else require more cash. Depicted below, you can see how its cash holdings have changed over time. Check out our latest analysis for Intellia Therapeutics Intellia Therapeutics reduced its cash burn by 8.8% during the last year, which points to some degree of discipline. Unfortunately, however, operating revenue declined by 13% during the period. Considering both these factors, we're not particularly excited by its growth profile. While the past is always worth studying, it is the future that matters most of all. For that reason, it makes a lot of sense to take a look at our analyst forecasts for the company. Intellia Therapeutics seems to be in a fairly good position, in terms of cash burn, but we still think it's worthwhile considering how easily it could raise more money if it wanted to. Companies can raise capital through either debt or equity. Commonly, a business will sell new shares in itself to raise cash and drive growth. By comparing a company's annual cash burn to its total market capitalisation, we can estimate roughly how many shares it would have to issue in order to run the company for another year (at the same burn rate). Since it has a market capitalisation of US$994m, Intellia Therapeutics' US$381m in cash burn equates to about 38% of its market value. That's not insignificant, and if the company had to sell enough shares to fund another year's growth at the current share price, you'd likely witness fairly costly dilution. On this analysis of Intellia Therapeutics' cash burn, we think its cash runway was reassuring, while its cash burn relative to its market cap has us a bit worried. Shareholders can take heart from the fact that analysts are forecasting it will reach breakeven. Looking at the factors mentioned in this short report, we do think that its cash burn is a bit risky, and it does make us slightly nervous about the stock. Its important for readers to be cognizant of the risks that can affect the company's operations, and we've picked out 2 warning signs for Intellia Therapeutics that investors should know when investing in the stock. Of course, you might find a fantastic investment by looking elsewhere. So take a peek at this free list of companies with significant insider holdings, and this list of stocks growth stocks (according to analyst forecasts) — Investing narratives with Fair Values Vita Life Sciences Set for a 12.72% Revenue Growth While Tackling Operational Challenges By Robbo – Community Contributor Fair Value Estimated: A$2.42 · 0.1% Overvalued Vossloh rides a €500 billion wave to boost growth and earnings in the next decade By Chris1 – Community Contributor Fair Value Estimated: €78.41 · 0.1% Overvalued Intuitive Surgical Will Transform Healthcare with 12% Revenue Growth By Unike – Community Contributor Fair Value Estimated: $325.55 · 0.6% Undervalued View more featured narratives — Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Sign in to access your portfolio
Business Upturn
15-06-2025
- Business
- Business Upturn
Intellia Therapeutics Announces Positive Three-Year Data from Phase 1 Trial of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema (HAE) at the European Academy of Allergy and Clinical Immunology Congress
With up to three years of follow-up, a single dose of lonvo-z led to a 98% mean reduction in monthly HAE attack rate in all 10 patients All 10 patients were attack-free and treatment-free for a median of 23 months through the latest follow-up, demonstrating the potential of lonvo-z to become the first one-time therapy for most HAE patients Lonvo-z was well tolerated and continues to demonstrate a favorable safety profile The global Phase 3 HAELO trial of lonvo-z has concluded screening ahead of schedule with more than half screened from U.S. sites; Intellia to provide an update on enrollment in the future CAMBRIDGE, Mass., June 15, 2025 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced three-year follow-up data from the Phase 1 portion of the ongoing Phase 1/2 study in patients with HAE after receiving a single dose of lonvoguran ziclumeran (lonvo-z, also known as NTLA-2002). Results were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held June 13-16 in Glasgow, United Kingdom. 'Today's results underscore the promising potential of Intellia's approach to gene editing therapy – a one-time treatment that was well tolerated and offered a highly differentiated, durable effect for patients suffering from a serious disease,' said Intellia President and Chief Executive Officer John Leonard, M.D. 'Seeing all 10 patients in the Phase 1 portion of this study free from both HAE attacks and chronic therapy at nearly two years of median follow-up is incredibly encouraging. These data fuel our optimism for the outcomes of our ongoing Phase 3 HAELO study, which we expect to report in the first half of 2026, and highlight the strong value we believe it will offer patients, physicians and payers.' 'People living with HAE often report a reduced quality of life because they worry about the likelihood of their next attack, either because they still experience attacks or are reminded of it by their use of chronic therapy,' said Dr. Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research, Inc. 'Based on the data, it is reasonable to expect lonvo-z could offer patients the potential to be free from both physical HAE attacks and the burden of managing chronic HAE treatment.' In the Phase 1 portion of the study, a one-time dose of 25 mg (N=3), 50 mg (N=4) or 75 mg (N=3) of lonvo-z was administered via intravenous infusion and plasma kallikrein protein levels were measured along with HAE attacks. At the time of the February 12 data cutoff, patients were attack-free and treatment-free for a median of nearly two years. With up to three years of follow-up, a single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% over the study period, compared to pre-treatment baseline. For all 10 patients, deep, dose-dependent and durable reductions in plasma kallikrein protein continued to be observed through the latest assessment. Safety Across all three dose levels, lonvo-z has been well tolerated and continues to demonstrate a favorable safety profile consistent with earlier data presented at EAACI in 2024. The most frequent adverse events during the study period were infusion-related reactions (IRRs). IRRs were mostly Grade 1 and resolved with all patients receiving the full dose. With up to 3 years of follow-up, no treatment-emergent serious adverse events were observed, and no treatment-related adverse events were observed during the period following 28 days after dosing. Clinical Development Plans Intellia's global Phase 3, randomized, double-blind, placebo-controlled HAELO trial is ongoing to assess the safety and efficacy of lonvo-z at the 50 mg dosage. The Company announced today the HAELO trial has successfully completed screening ahead of schedule, with over half of the patients being screened in the United States. The study is no longer recruiting and Intellia will provide an update on enrollment in the future. New and longer-term data from the Phase 2 portion of the ongoing Phase 1/2 study is planned to be presented in the second half of 2025. Intellia expects to submit a biologics license application (BLA) in 2026 to support the Company's plans for a U.S. launch in 2027. For more information on HAELO (NCT06634420), please visit About the Lonvoguran Ziclumeran (lonvo-z, also known as NTLA-2002) Clinical Program Intellia's ongoing Phase 1/2 study is evaluating the safety and efficacy of lonvo-z in adults with Type I or Type II hereditary angioedema (HAE). The Phase 1 portion of the study is an international, open-label study designed to identify the dose level of lonvo-z selected for further evaluation in the Phase 2 portion of the study. Enrollment in both portions of the Phase 1/2 study is complete. Intellia dosed the first patient in the global Phase 3, randomized, double-blind, placebo-controlled HAELO trial in January of 2025. Visit (NCT05120830) for more details. About Lonvo-z Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an investigational in vivo CRISPR-based gene editing therapy designed to prevent HAE attacks by inactivating the kallikrein B1 ( KLKB1 ) gene, which encodes for prekallikrein, the kallikrein precursor protein. Interim Phase 1/2 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission. About Intellia Therapeutics Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia's deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at and follow us @intelliatx. Forward-Looking Statements This press release contains 'forward-looking statements' of Intellia Therapeutics, Inc. ('Intellia' or the 'Company') within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia's beliefs and expectations concerning: the safety, efficacy, success and advancement of its clinical programs for lonvoguran ziclumeran or 'lonvo-z' (also known as NTLA-2002) for hereditary angioedema ('HAE'), including the ability to successfully complete its global Phase 3 HAELO study; its expectation to present additional data regarding lonvo-z, including reporting outcomes of the Phase 3 HAELO study in the first half of 2026 and presenting new and longer-term data from the Phase 2 portion of the ongoing Phase 1/2 study of lonvo-z in the second half of 2025; and its expectation to be able to support a biologics license application for lonvo-z for the treatment of HAE by 2026 for a U.S. launch in 2027. Any forward-looking statements in this press release are based on management's current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to Intellia's relationship with third parties, including its contract manufacturers, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to Intellia's relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to our product candidates, including lonvo-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for our product candidates, including uncertainties related to regulatory approvals to conduct clinical trials, including our ability to complete the Phase 3 HAELO study for HAE; the risk that any one or more of Intellia's product candidates, including lonvo-z, will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies for the same product candidate or Intellia's other product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia's actual results to differ from those contained in the forward-looking statements, see the section entitled 'Risk Factors' in Intellia's most recent annual report of Form 10-K and quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Intellia's other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law. Intellia Contacts: Investors:Brittany ChavesSenior Manager, Investor Relations [email protected]
Yahoo
30-05-2025
- Business
- Yahoo
Intellia stock dips following safety event in Phase III gene therapy trial
Intellia Therapeutics' stock has fallen nearly 23% following an announcement that a patient in its Phase III trial for a gene therapy targeting transthyretin amyloid (ATTR) cardiomyopathy suffered a serious adverse event (AE). In a filing by the Securities and Exchange Commission (SEC), the company reported that one patient experienced Grade 4 liver transaminase elevations after treatment with nexiguran ziclumeran (nex-z) in the Phase III MAGNITUDE trial (NCT06128629). According to Intellia, the event is resolving without the need for hospitalisation or medical intervention. Upon the announcement, Intellia's stock, listed on the Nasdaq exchange, dropped by 22.87% from $9.66 a share at market close on 28 May to $7.45 at market close on 29 May. Intellia emphasised that this is a single event from more than 200 patients dosed with nex-z in the MAGNITUDE study. Other AEs have been similar to those seen in the Phase I study, including infusion-related reactions and asymptomatic liver transaminase elevations. The SEC filing added that Intellia said it will continue to monitor these events as the study progresses. The trial has so far enrolled 365 patients out of an expected total enrollment of approximately 765 patients, which Intellia says is on track. The company aims to complete enrollment by early 2027. Intellia is also investigating nex-z in the Phase III MAGNITUDE-2 study (NCT06672237), which is enrolling patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Dosing has started, with investigators seeking data on whether the therapy will lead to a reduction in serum TTR. Intellia will be waiting for data from MAGNITUDE-2 before filing a biologics licence application (BLA) to the US Food and Drug Administration (FDA) in 2028, with hopes for a 2029 commercial launch. It has been a challenging time for gene therapy development, with several severe AEs and, in some instances, fatalities seeming to plague investors' views on the therapeutic approach. On 27 May, Rocket Pharmaceuticals announced that a patient died in its Phase II trial for a Danon disease gene therapy after suffering a serious AE. In March 2025, Sarepta and Roche reported the death of a teenage patient dosed with its AAV gene therapy for Duchenne muscular dystrophy (DMD), Elevidys (delandistrogene moxeparvovec-rokl), causing agencies to pause ongoing clinical studies. This pause has since been lifted. Last year, a fatality in Pfizer's Phase II DMD gene therapy trial, as well as low efficacy in a Phase III study, caused the big pharma to drop the development of the AAV candidate, named fordadistrogene movaparvovec. Earlier this month, the FDA shared that American haematologist oncologist Vinay Prasad will lead its Center for Biologics Evaluation and Research (CBER), the division responsible for regulating gene therapies and vaccines. Prasad has openly criticised Elevidys, primarily about the clinical evidence and the FDA's decision to approve the therapy, which raises questions about his overall opinion of the gene therapy approach. "Intellia stock dips following safety event in Phase III gene therapy trial" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
