Moderna TodayMRNAModerna$25.80 -2.19 (-7.82%) 52-Week Range$23.15▼$170.47Price Target$53.95Add to WatchlistModerna Inc. NASDAQ: MRNA is down 35% in 2025, making it one of the worst-performing stocks among medical stocks and the broader market. Not surprisingly, the stock is down over 80% in the last 12 months. What may surprise investors is that the Moderna stock is down approximately 60% over the last five years. That means the stock has given back all the gains it made when the company’s COVID-19 vaccine ...

经济观察网讯据上海免疫与感染研究所消息，5月19日，中国科学院上海免疫与感染研究所、中国科学 院分子植物科学卓越创新中心、华东师范大学和康涅狄格大学等多个团队合作，提出了一种全新的 mRNA序列设计与优化平台——mRNAdesigner，为下一代核酸药物开发奠定了基础。 mRNA药物正成为现代生物医药领域的重要发展方向，但当前mRNA的设计面临着跨物种适应性不足、 忽略上下游非翻译区对表达的调控作用、免疫原性控制不足等多重挑战。对此，研究团队创新性地将 RNA翻译动力学的前沿研究成果融入设计理念，综合考虑了mRNA序列中的密码子偏好性、GC含量、 二级结构对蛋白翻译表达的影响，从而实现对mRNA主要结构区域(5'UTR、CDS、3'UTR)的全面优化设 计。经过该平台重新设计过的编码区序列，可在设定的GC比例下提高密码子自适应指数，优化碱基互 补配对模式的同时规避长茎环结构和GC富集区，降低mRNA在细胞中被天然免疫识别和降解的风险。 针对5'UTR区域，平台通过提高核糖体负载(MRL)优化设计，促使翻译起始和延伸更为高效。针对 3'UTR区域，通过调控元件注释算法，识别并减少与序列降解有关的调控元件的数量( ...

带状疱疹(herpes zoster)是由长期潜伏在人体内的水痘-带状疱疹病毒(varicella–zoster virus, VZV)经再启 动引起的感染性皮肤病。人群对VZV普遍易感，血清学证据显示，成人VZV感染率高达90%以上。带状 疱疹的发病率为3‰~5‰人╱年，亚太地区为3‰~10‰人╱年，并逐年递增2.5%~5.0%。50岁后随年龄 增长，VZV特异性细胞免疫功能逐渐降低，带状疱疹的发病率、住院率和病死率均逐渐升高。带状疱 疹的復发率为1%~10%,易患因素包括高龄、免疫力低下和慢性病患者。接种带状疱疹疫苗是抑制带状疱 疹发病的有效措施。带状疱疹疫苗接种的目标是抑制VZV再启动从而预防带状疱疹、带状疱疹后神经 痛(PHN)和其他并发症。 智通财经APP讯，艾美疫苗(06660)发布公告，继2025年3月该疫苗获美国食品和药品监督管理局(FDA) 批准开展临床试验后，集团研发的mRNA带状疱疹疫苗已于近日获得国家药品监督管理局《药物临床试 验批准通知书》。该疫苗中美双报并均获批准，充分验证了集团mRNA技术平台的强劲实力。 临床前试验中，第三方检测单位的检测结果显示：集团mRNA带状疱疹疫苗特异性 ...

Equity markets are full of bargains -- stocks that have performed terribly in recent months, whether due to trade-related marketwide issues or company-specific troubles, but could bounce back once things improve. There are also companies that are not performing well and are unlikely to rebound anytime soon; that's the kind investors should stay away from.Let's consider one stock in each category: Moderna (MRNA -1.50%), a beaten-down stock with decent prospects, and Teladoc Health (TDOC -1.02%), one whose fu ...

Maravai LifeSciences (MRVI) Q1 2025 Earnings Call May 12, 2025 05:00 PM ET Company Participants Deb Hart - Head of Investor RelationsTrey Martin - CEOKevin Herde - Executive VP & CFODan Arias - Managing DirectorBecky Buzzeo - Executive VP & Chief Commercial OfficerBrandon Couillard - Managing Director Conference Call Participants Conor McNamara - Equity Research AnalystDan Leonard - Managing Director & Research AnalystTejas Savant - Executive Director & Senior Healthcare Equity AnalystMatthew Stanton - Anal ...

Financial Data and Key Metrics Changes - The company reported $47 million in revenue for Q1 2025, exceeding expectations and showing a growth of over $4 million from Q4 2024 [6][27] - GAAP net loss before noncontrolling interests was $53 million for Q1 2025, compared to a loss of $23 million in Q1 2024 [27] - Adjusted EBITDA was negative $11 million for Q1 2025, down from a positive $8 million in Q1 2024 [28] - Basic and diluted EPS for Q1 was a loss of $0.21 per share, compared to a loss of $0.09 per share in Q1 2024 [30] Business Line Data and Key Metrics Changes - The Nucleic Acid Production (NAP) segment generated $29 million in revenue for Q1 2025, an increase of $1 million from Q4 2024 [6] - The Biologics Safety Testing (BST) segment reported $18 million in revenue for Q1 2025, up $3 million from Q4 2024 and flat compared to Q1 2024 [6][35] - The adjusted EBITDA margin for the BST segment was 70%, indicating strong performance [35] Market Data and Key Metrics Changes - Revenue by customer type in Q1 was 29% from biopharma, 28% from life science and diagnostics, 6% from academia, 7% from CDMO, and 30% through distributors [7] - Revenue by geography was 62% from North America, 15% from EMEA, 15% from Asia Pacific, and 8% from China [7] Company Strategy and Development Direction - The company is focused on a return to growth strategy and building a diversified franchise as a life science tool provider and clinical partner [10] - The strategy includes vertical integration in the NAP segment to enhance quality, speed, and cost efficiency [8] - The company aims to expand its product and service portfolio to drive long-term sustainable growth [10] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges of transitioning from a COVID-driven revenue model to a more diversified business [39] - The company is closely monitoring trade dynamics and working to mitigate potential impacts from tariffs [39] - The cash position of $285 million is seen as sufficient to manage the reset period while focusing on long-term growth [40] Other Important Information - The company plans to publish its 2024 sustainability report, highlighting its commitment to sustainability and long-term strategic objectives [25] - The integration of recent acquisitions is progressing well, enhancing operational capabilities and supply chain control [32] Q&A Session Summary Question: Focus areas for new trials and reasons for discontinuation - Management noted that while preclinical programs were flat, clinical programs showed growth, indicating a focus on later-stage projects due to funding conservatism [44][46] Question: Drivers of incremental demand in the next 12-18 months - Incremental demand is expected from clinical progression, new business wins, and the transition from bulk reagent supply to comprehensive service offerings [48] Question: Opportunities for market share due to U.S. manufacturing - The company sees potential for market share growth as interest in U.S.-based manufacturing increases, particularly in the mRNA space [51][54] Question: Impact of U.S. policy changes on customer buying patterns - Customers are experiencing mixed impacts from government funding changes, with some securing long-term funding while others delay decisions [60] Question: Government work and exposure - The company has no direct government work currently, with past contracts fulfilled, but remains open to future opportunities [66] Question: Seasonality in BST revenue - Q1 is typically a high point for BST revenue, influenced by manufacturing cycles and strong performance in China [70][72]

Financial Data and Key Metrics Changes - For Q1 2025, revenues were $29.4 million, down from $38 million in Q1 2024, primarily due to lower development milestone revenues from the CSL collaboration as the COVID vaccine transitions to commercialization [19][20] - Research and development expenses decreased to $34.9 million from $53.6 million year-over-year, driven by lower manufacturing costs, with expectations for further declines in the second half of fiscal year 2025 [20][21] - General and administrative expenses were $11.3 million, down from $14.9 million in the same period last year, attributed to reduced share-based compensation costs [21] - The net loss for Q1 2025 was approximately $14.1 million, or $0.52 per diluted share, compared to a net loss of $3 million, or $1 per diluted share, in Q1 2024 [21] Business Line Data and Key Metrics Changes - The mRNA therapeutics pipeline is being prioritized, with significant focus on the CF and OTC programs, while the COVID vaccine program is transitioning to commercialization [11][18] - The ARCT032 program for cystic fibrosis is in Phase II, with enrollment expected to complete by the end of 2025, and interim data anticipated in mid-2025 [7][10] - The ARCT810 program for OTC deficiency is also in Phase II, with interim data expected in Q2 2025 [8][10] Market Data and Key Metrics Changes - The company received an initial milestone payment from CSL related to the EU approval of the COVID vaccine, with further payments anticipated as the program progresses [11][19] - The company is preparing for a Marketing Authorization Application in the UK in Q2 2025 and a BLA filing in the US in Q3 2025 [12] Company Strategy and Development Direction - The company has made a strategic decision to focus resources on mRNA therapeutics, particularly CF and OTC programs, due to current market conditions and regulatory uncertainties [18][19] - The STAR self-amplifying mRNA platform continues to receive positive feedback from publications, enhancing the company's position in the market [12][14] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming clinical data from the CF and OTC programs, highlighting the potential for significant advancements in treatment options [22][23] - The company remains committed to maintaining a strong financial position, with a cash runway extended into 2028, allowing for the achievement of near-term milestones [21][22] Other Important Information - The company is utilizing a newly developed 15N ureogenesis assay to monitor the effects of ARCT810 in clinical development, which is expected to provide valuable data [10][18] - Long-term data from the COVID vaccine trial suggests a favorable safety profile, with no reports of myocarditis or pericarditis [14] Q&A Session Summary Question: Can you provide more details on the changes made to extend the cash runway and potential incoming cash flows? - Management indicated that tough decisions were made regarding cost reductions and focusing on critical programs, which contributed to extending the cash runway [27][28] Question: What milestones should we expect related to the UK and US approvals for the COVID vaccine? - There are no milestones associated with the UK or US approvals; however, a milestone is anticipated with the first US revenues from the COVID vaccine, expected in 2028 [30][31] Question: What is the expected size of the initial interim cohort for ARCT032 and the bar for success? - The interim data set is expected to include 6 to 9 subjects, with a bar for success set at a 3% improvement in lung function [35][36] Question: How does ARCT032 differ from competitor programs in terms of tolerability? - The company highlighted its exclusive LUNAR lipid nanoparticle delivery technology and the high purity of its mRNA constructs as key differentiators [42][43] Question: What are the expected biomarkers for the OTC program? - Management confirmed that glutamine, orotic acid, and other biomarkers will be measured to assess the effectiveness of the treatment [86][87]

Financial Data and Key Metrics Changes - For Q1 2025, revenues were $29.4 million, down from $38 million in Q1 2024, primarily due to lower development milestone revenues from the CSL collaboration as the COVID vaccine transitions to commercialization [17][18] - Research and development expenses decreased to $34.9 million from $53.6 million year-over-year, driven by lower manufacturing costs, partially offset by increased costs for CF and OTC programs [18][19] - General and administrative expenses were $11.3 million, down from $14.9 million in the same period last year, mainly due to reduced share-based compensation costs [19][20] - The net loss for Q1 2025 was approximately $14.1 million, or $0.52 per diluted share, compared to a net loss of $3 million, or $1 per diluted share, in Q1 2024 [20] Business Line Data and Key Metrics Changes - The mRNA therapeutics pipeline is being prioritized, with significant focus on CF and OTC programs, while the COVID vaccine program is transitioning to commercialization [10][16] - The company expects to complete Phase II enrollment for ARCT032 by the end of 2025 and provide interim data for the first two cohorts in mid-2025 [6][9] - The ARCT-810 program for OTC deficiency is also progressing, with interim data expected in Q2 2025 [7][9] Market Data and Key Metrics Changes - The company received an initial milestone payment from CSL related to the EU approval of the COVID vaccine, Costave, and anticipates further milestone payments as the program progresses [10][17] - The company is preparing for a Marketing Authorization Application (MAA) filing in the UK in Q2 2025 and a U.S. BLA filing in Q3 2025 [11] Company Strategy and Development Direction - The company has made a strategic decision to focus resources on mRNA therapeutics, particularly CF and OTC programs, due to current market conditions and regulatory uncertainties [10][16] - The STAR self-amplifying mRNA platform continues to receive positive feedback from publications, enhancing the company's position in the market [11][13] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming clinical data from CF and OTC programs, indicating a strong cash runway extending into 2028 [21] - The company remains committed to advancing its therapeutic programs and believes that the science will support its regulatory interactions [100] Other Important Information - The company has received FDA Fast Track designation for ARCT2304, its mRNA vaccine candidate for pandemic influenza A [14] - The 15N Ureogenesis Assay is expected to provide important data for monitoring the effect of ARCT-810 in clinical development [9][108] Q&A Session Summary Question: Can you provide more details on extending the cash runway and potential cash flows? - Management indicated that focusing on critical programs and making cost reductions contributed to extending the cash runway, with a conservative approach to cash burn forecasts [26][27] Question: What milestones should we expect related to UK and U.S. approvals for the COVID vaccine? - There are no milestones associated with UK or U.S. approvals; however, a milestone is anticipated with the first U.S. revenues from Costave, expected in 2028 [31][60] Question: What is the expected size of the interim cohort for ARCT032? - The interim data set is expected to include 6 to 9 subjects, with a bar for success set at a 3% improvement in lung function [36][37] Question: How does ARCT032 differ from competitor programs? - The key differentiators include the exclusive LUNAR lipid nanoparticle delivery technology and the high purity of the mRNA construct, which may enhance safety and tolerability [44][45] Question: What are the thresholds for FEV1 improvement in the CF program? - A 3% improvement in FEV1 is considered a significant threshold, with higher improvements potentially allowing for smaller Phase III trial sizes [86][87] Question: Will other biomarkers be measured in the OTC program? - In addition to glutamine, orotic acid and other amino acids will also be measured as part of the biomarker strategy [88][89]

中新网西安5月9日电 (记者 阿琳娜)记者9日从西安电子科技大学获悉，该校生命科学技术学院邓宏章教 授团队以创新性非离子递送系统，成功破解"毒性-效率"死锁，为基因治疗装上"安全导航"。 据介绍，在生物医药技术迅猛发展的今天，mRNA疗法以其巨大的潜力和迅猛的发展速度成为医学领域 的焦点，mRNA技术正逐步重塑现代医疗的版图。然而，这一领域的核心挑战——如何安全高效地递送 mRNA至靶细胞始终是制约其临床转化的关键瓶颈。传统脂质纳米颗粒(LNP)依赖阳离子载体的递送系 统虽广泛应用，却伴随毒性高、稳定性差等难题，亟需一场技术革命。 mRNA作为携带负电荷的亲水性大分子，需借助载体穿越细胞膜的静电屏障并抵御RNA酶的快速降解。 传统LNP依赖阳离子脂质与mRNA的静电结合，虽能实现封装，却因电荷相互作用引发炎症反应和细胞 毒性，且存在靶向性差、体内表达周期短等缺陷。邓宏章团队另辟蹊径，通过人工智能筛选出硫脲基团 作为关键功能单元，构建基于氢键作用的非离子递送系统(TNP)。 与传统LNP不同，TNP通过硫脲基团与mRNA形成强氢键网络，实现无电荷依赖的高效负载。实验表 明，TNP不仅制备工艺简便，更具备多项突破 ...

Core Viewpoint - Vertex Pharmaceuticals experienced a significant stock decline after missing Wall Street's revenue and earnings estimates for Q1, but this sell-off presents a buying opportunity for investors [1][2]. Financial Performance - Vertex reported Q1 revenue of $2.77 billion, a 3% increase year over year, but below the expected $2.83 billion [2]. - Adjusted earnings per share were $4.06, missing the consensus estimate of $4.29 [2]. Operational Updates - The company announced a temporary pause in the multiple ascending dose portion of its phase 1/2 study for mRNA therapy VX-522 due to a tolerability issue [3][5]. - Vertex attributed weaker-than-expected financial results partly to illegal copycat products in Russia, although it believes this issue is isolated to that market [3]. Guidance and Future Outlook - Vertex raised the lower end of its full-year revenue guidance for 2025 to between $11.85 billion and $12 billion, up from a previous lower end of $11.75 billion [4]. - The management remains optimistic about the investment thesis despite the setbacks with VX-522 [5]. Product Developments - Vertex's new cystic fibrosis therapy, Alyftrek, is receiving positive feedback from physicians and patients, with expectations for a switch from existing CF drugs [7]. - The company received a positive opinion from the CHMP for Alyftrek, aiming for European Commission approval in the second half of 2025 [8]. - Vertex's new non-opioid pain drug, Journavx, is expected to see increased sales volume in the coming months, despite minimal sales reported in Q1 [10]. Pipeline Prospects - Vertex plans to file for regulatory approvals for zimislecel in treating severe Type 1 diabetes next year, pending positive study results [11]. - The company may also seek U.S. accelerated approval for povetacicept in treating IgA nephropathy in the first half of 2026, contingent on favorable interim data [11]. Risk Management - Vertex's CFO indicated that the company has low exposure to tariffs due to its geographically diverse supply chain and manufacturing primarily in the U.S. [12]. Investment Sentiment - Despite the revenue and earnings miss, Vertex is viewed as a strong investment opportunity, with expectations for significant returns driven by the adoption of Alyftrek and the potential success of Journavx [13][14].