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Novartis, BrainStorm Cell Therapeutics, Neuroplast, Rapa Therapeutics, and Longeveron Driving Innovations
DUBLIN--(BUSINESS WIRE)--Jun 27, 2025-- The 'Gene and Cell Therapies Targeting CNS Disorders Market - A Global and Regional Analysis: Focus on Drug and Region - Analysis and Forecast, 2025-2035" report has been added to offering. Global gene and cell therapies targeting central nervous system (CNS) disorders a market is on track for continued growth, driven by the aging population, and the development of more effective and comfortable solutions. The ongoing advancements in product technology and the expansion of care options will continue to shape the market's future. As demand for gene and cell therapies targeting central nervous system (CNS) disorders therapies rises, both global and regional players will play a key role in meeting the needs of individuals and healthcare systems alike, improving quality of life for people living with incontinence. The gene and cell therapies targeting central nervous system (CNS) disorders are rapidly advancing, offering potential treatments for conditions such as Parkinson's, Alzheimer's, Huntington's disease, and spinal cord injuries. Increasing incidences of CNS disorders one of the major driving factors of this market. The increasing number of patients with neurological disorders such as Alzheimer's, Parkinson's, and multiple sclerosis is creating an urgent need for innovative treatments. This drives demand for gene and cell therapies, as current treatments are often ineffective or only offer temporary relief. One of the significant drivers of the global gene and cell therapies targeting central nervous system (CNS) disorders market is the technological advancements in treatment delivery. The technological innovations in drug delivery systems, such as CRISPR and gene editing, stem cell therapy and AAV vectors for gene delivery. Furthermore, improved precision in treatment technologies such as CRISPR-Cas9 allow for precise editing of genes involved in neurological diseases. This level of precision offers hope for conditions that have a genetic root, such as Huntington's disease or spinal muscular atrophy have contributed to the market's growth. Despite the growth trajectory, several challenges continue to impact the global gene and cell therapies targeting central nervous system (CNS) disorders market. One of the primary challenges is high development and manufacturing costs. The developing and manufacturing gene and cell therapies, especially those targeting CNS disorders, is extremely expensive. The production of viral vectors for gene delivery and the preparation of cell-based therapies are both costly and complex processes. The high costs of developing these therapies can limit accessibility and affordability for patients. Additionally, these high costs can result in delayed market entry and restrictions on patient access, particularly in low- and middle-income countries. Leading players in the global gene and cell therapies targeting central nervous system (CNS) disorders market, such as Novartis, and BrainStorm Cell Therapeutics are continuously innovating to improve the effectiveness and comfort of gene and cell therapies targeting central nervous system (CNS) disorders. These companies are investing heavily in research and development to introduce new, technologically advanced therapies into the market. With a strong emphasis on user-friendly and environmentally sustainable products, these companies are shaping the future of gene and cell therapies targeting central nervous system (CNS) disorders while enhancing their market positions globally. The competitive landscape of the global gene and cell therapies targeting central nervous system (CNS) disorders market is diverse, with numerous players across different regions offering a wide range of products. Regional players and local manufacturers are expected to play an important role in the market's growth, especially as demand increases in emerging markets such as Asia-Pacific. As consumer preferences shift towards more discreet, comfortable, and affordable solutions, the gene and cell therapies targeting central nervous system (CNS) disorders market will continue to evolve, fostering new opportunities for both established and emerging companies. As the gene and cell therapies targeting central nervous system (CNS) disorders market evolves, emerging trends such expansion of cell-based therapies and focuses on personalized or precision medicine. This trend allows treatments to be more specific and effective for individual patients, reducing side effects and improving patient outcomes. Companies Featured Key Topics Covered: Executive Summary 1. Global Gene and Cell Therapies Targeting CNS Disorders Market: Industry Outlook 1.1 Overview 1.2 Regulatory Landscape 1.3 Global Gene and Cell Therapies Targeting CNS Disorders Market, Patent Landscape 1.3.1 By Country 1.3.2 By Year 1.4 Global Gene and Cell Therapies Targeting CNS Disorders Market, Clinical Trials Landscape 1.5 Key Trends 1.6 Market Dynamics 1.6.1 Overview 1.6.1.1 Impact Analysis 1.6.2 Market Drivers 1.6.3 Market Restraints 1.6.4 Market Opportunities 2. Global Gene and Cell Therapies Targeting CNS Disorders Market, by Drugs, $Million, 2023-2035 2.1 Gene Therapy Drugs 2.2 Cell Therapy Drugs 3. Global Gene and Cell Therapies Targeting CNS Disorders, by Region, $Million, 2023-2035 3.1 North America 3.1.1 Key Findings in North America 3.1.2 Market Dynamics 3.1.3 Market Sizing and Forecast 3.1.3.1 North America Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.1.3.1.1 U.S. 3.1.3.1.2 Canada 3.2 Europe 3.2.1 Key Findings in Europe 3.2.2 Market Dynamics 3.2.3 Market Sizing and Forecast 3.2.3.1 Europe Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.2.3.1.1 Germany 3.2.3.1.2 U.K. 3.2.3.1.3 France 3.2.3.1.4 Italy 3.2.3.1.5 Spain 3.2.3.1.6 Rest-of-Europe 3.3 Asia-Pacific 3.3.1 Key Findings in Asia-Pacific 3.3.2 Market Dynamics 3.3.3 Market Sizing and Forecast 3.3.3.1 Asia-Pacific Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.3.3.1.1 Japan 3.3.3.1.2 China 3.3.3.1.3 India 3.3.3.1.4 Rest-of-Asia-Pacific 3.4 Rest-of-the-World 3.4.1 Key Findings in Rest-of-the-World 3.4.2 Market Dynamics 3.4.3 Market Sizing and Forecast 4. Global Gene and Cell Therapies Targeting CNS Disorders Market: Competitive Landscape and Company Profiles 4.1 Competitive Landscape 4.1.1 New Offerings 4.1.2 Mergers and Acquisitions 4.1.3 Partnerships, Alliances, and Business Expansion 4.1.4 Funding Activities 4.1.5 Regulatory Approvals 4.2 Company Profiles 4.2.1 Overview 4.2.2 Product Portfolio 4.2.3 Target Customers 4.2.4 Key Professionals 4.2.5 Analyst View 5. Research Methodology For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. View source version on CONTACT: Laura Wood, Senior Press Manager [email protected] For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 KEYWORD: INDUSTRY KEYWORD: GENERAL HEALTH NEUROLOGY HEALTH GENETICS PHARMACEUTICAL SOURCE: Research and Markets Copyright Business Wire 2025. PUB: 06/27/2025 09:33 AM/DISC: 06/27/2025 09:33 AM
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[Latest] Global Cell and Gene Therapy Market Size/Share Worth USD 118.63 Billion by 2034 at a 18.79% CAGR: Custom Market Insights (Analysis, Outlook, Leaders, Report, Trends, Forecast, Segmentation, Growth Rate, Value, SWOT Analysis)
[220+ Pages Latest Report] According to a market research study published by Custom Market Insights, the demand analysis of Global Cell and Gene Therapy Market size & share revenue was valued at approximately USD 21.23 Billion in 2024 and is expected to reach USD 25.20 Billion in 2025 and is expected to reach around USD 118.63 Billion by 2034, at a CAGR of 18.79% between 2025 and 2034. The key market players listed in the report with their sales, revenues and strategies are Amgen, Inc., Biogen Inc., Biomarin Pharmaceutical Inc., Bluebird Bio, Inc. (Celgene Corporation), Bristol-Myers Squibb Company, Freeline Therapeutics Ltd., Gene Biotherapeutics, Gilead Sciences, Inc., Horama S.A., JCR Pharmaceuticals Co. Ltd., Kolon TissueGene, Inc., MeiraGTx Limited, Novartis International AG, Orchard Therapeutics Plc., and Organogenesis, Inc, and others. Austin, TX, USA, June 25, 2025 (GLOBE NEWSWIRE) -- Custom Market Insights has published a new research report titled 'Cell and Gene Therapy Market Size, Trends and Insights By Therapy Type (Cell Theray, Gene Therapy), By Therapeutic Class (Cardiovascular Disease, Cancer, Genetic Disorder, Rare Diseases, Oncology, Hematology, Ophthalmology, Infectious Disease, Neurological Disorders, Others), By Delivery Method (In Vivo, Ex Vivo), By End-User (Hospitals, Cancer Care Centers, Others), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025–2034' in its research database. 'According to the latest research study, the demand of global Cell and Gene Therapy Market size & share was valued at approximately USD 21.23 Billion in 2024 and is expected to reach USD 25.20 Billion in 2025 and is expected to reach a value of around USD 118.63 Billion by 2034, at a compound annual growth rate (CAGR) of about 18.79% during the forecast period 2025 to 2034.' Click Here to Access a Free Sample Report of the Global Cell and Gene Therapy Market @ Overview According to industry experts at CMI, the implementation of new strategies and technologies by the manufacturers presents lucrative opportunities for players in the Cell and Gene Therapy Market during the forecast period. Furthermore, we expect the growing significance of organized retailing to drive the future growth of the market. Key Trends & Drivers Advancements in Gene Editing Technologies: The development of precise gene editing tools such as CRISPR-Cas9, TALENs, and zinc finger nucleases is reshaping the gene therapy market. These technologies help in the accurate fixing of mutations in the genetic material, along with reducing off-target effects and enhancing safety. We expect CRISPR to gain popularity due to its efficiency, cost-effectiveness, and versatility in treating various genetic disorders. With the evolution of these tools, more clinical programs are now involved in integrating them, including therapies for inherited diseases and certain cancers. Continuous innovations in editing precision, delivery systems, and regulatory oversight are catapulting gene editing to the forefront of gene therapy applications in the future and expanding its commercial scope. Gene therapy: is rapidly expanding into rare and orphan diseases, many of which lack established treatments. These conditions often tend to have a well-defined genetic cause, making them apt candidates for gene intervention. Regulatory incentives such as orphan drug designations, priority review, and extended marketing exclusivity provide more encouragement for development in this arena. Emerging therapies are targeting spinal muscular atrophy, Duchenne muscular dystrophy, and inherited retinal disorders, while inroads made by their success have contributed to the further increase in research. This trend is addressing several pressing unmet medical needs while also providing for quicker entry into the marketplace with comparatively less competition, thereby fueling the rapid growth of the gene therapy sector. Increasing Regulatory Support and Fast-Track Approvals: Increasingly, worldwide regulatory bodies such as the FDA and EMA are bestowing fast-track designations, breakthrough therapy statuses, and conditional approvals for promising gene therapies. These pathways are meant to fast-track development and review of treatments for serious or life-threatening illnesses. Such a regulatory change indicates growing confidence in gene therapy regarding safety and efficacy, as well as the urgency of putting new treatment modalities on the market. With more therapies entering the clinical success phase, regulatory agencies are now actively working with developers to update and revise the guidance on manufacturing, trial design, and post-market surveillance. Hence, a forward regulatory approach accelerates approvals and enhances investor confidence in the field. Growth in Strategic Partnerships and M&A Activity: The gene therapy market is currently witnessing an increase in strategic collaborations, licensing deals, mergers, and acquisitions between biotech startups and large pharma companies. The partnerships then translate into the commercialization of innovation by bringing together innovative research and development with the regulatory, manufacturing, and distribution capabilities of big pharma. The big players are essentially looking to pump life into their pipeline while the smaller players are getting capital infusion and technical know-how. The notable M&A activities give a similar indication of confidence in the long-term value of gene therapies. Therefore, it promotes the efficiency of R&D, speeds up the arrival of products to the market, and shapes a competitive landscape, which in turn helps drive the growth of the market and increase the availability of new proposed treatments. Request a Customized Copy of the Cell and Gene Therapy Market Report @ Innovations in Delivery Mechanisms: The successful gene therapy needs the efficient delivery of the genetic materials inside the target cells. The innovations in the delivery systems, like adeno-associated viruses, lentiviral vectors, lipid nanoparticles, and non-viral systems are ushering in better therapeutic outcomes and have fewer side effects. These delivery systems are being fine-tuned for specific tissues and diseases, thereby increasing precision while reducing immune responses to the vector. Better platforms also consider vector re-dosing, increased tissue distribution, and efficacy, along with more straightforward manufacturing. As we develop better delivery technologies, the newer ones can treat more types of conditions and keep patients safer, making it easier to use gene therapy widely and helping it gain acceptance in clinics. Increasing Investment and Funding Activities: As there has been a surge in the venture capital, private equity, and public market investments coming into the fold, gene therapy is becoming more promising for the research and commercialization. The existence of several high-profile IPOs, funding rounds, and government grants serves as proof of investor confidence in gene therapy and in its ability to bring about a kind of transformation. The finances allow companies to further investigate the R&D, conduct clinical trials, and upgrade manufacturing facilities. Furthermore, there are also public and philanthropic organizations supporting early-stage innovations. This source of funding empowers developers to explore a broader range of diseases, invest in innovative technologies, and aim for global market penetration. Continued funding is a requisite to keep the momentum going and to ensure the long-term sustainability of the gene therapy sector. Report Scope Feature of the Report Details Market Size in 2025 USD 25.20 Billion Projected Market Size in 2034 USD 118.63 Billion Market Size in 2024 USD 21.23 Billion CAGR Growth Rate 18.79% CAGR Base Year 2024 Forecast Period 2025-2034 Key Segment By Therapy Type, Therapeutic Class, Delivery Method, End-User and Region Report Coverage Revenue Estimation and Forecast, Company Profile, Competitive Landscape, Growth Factors and Recent Trends Regional Scope North America, Europe, Asia Pacific, Middle East & Africa, and South & Central America Buying Options Request tailored purchasing options to fulfil your requirements for research. (A free sample of the Cell and Gene Therapy report is available upon request; please contact us for more information.) Our Free Sample Report Consists of the following: The updated report for 2024 includes an Introduction, an Overview, and an in-depth industry analysis. We have included the COVID-19 Pandemic Outbreak Impact Analysis in the package. About 220+ Pages Research Report (Including Recent Research) Provide detailed chapter-by-chapter guidance on the Request. Updated Regional Analysis with a Graphical Representation of Size, Share, and Trends for the Year 2025 Includes We have updated the tables and figures. The most recent version of the report includes the Top Market Players, their Business Strategies, Sales Volume, and Revenue Analysis Custom Market Insights (CMI) research methodology (Please note that the sample of the Cell and Gene Therapy report has been modified to include the COVID-19 impact study prior to delivery.) Request a Customized Copy of the Cell and Gene Therapy Market Report @ SWOT Analysis Strengths: A key advantage of cell and gene therapies is that they can go beyond just treating symptoms to tackle the root causes of diseases, providing the exciting chance for a single treatment for conditions that were once thought to be untreatable. The CGT market is experiencing exponential growth, with projections consistently indicating continued rapid expansion. The CGT market is propelled by ground-breaking technological advancements that enhance both the precision and potential of therapies. Weakness: A significant weakness of the Cell and Gene Therapy market is the exorbitant cost associated with manufacturing these advanced therapies, which currently renders them financially inaccessible for many patients. Scaling the manufacturing of cell and gene therapies to meet increasing global demand presents a formidable challenge. The supply chain for cell and gene therapies is exceptionally intricate and highly vulnerable, primarily due to the delicate nature of the products and strict logistical requirements. Opportunities: The increasing number of people suffering from long-term illnesses like different types of cancer, heart diseases, and diabetes, as well as a rise in genetic disorders such as sickle cell anemia and cystic fibrosis, is a strong reason for the growing need for new treatments. Governments worldwide are increasingly recognizing the transformative potential of cell and gene therapies and are actively supporting their development through significant initiatives and funding. The development and adoption of value-based payment models represent a crucial opportunity to mitigate the primary threat of high upfront costs. Threats: The most immediate and significant threat to the Cell and Gene Therapy market stems from the exorbitant upfront costs of these therapies, which create substantial reimbursement hurdles. A considerable threat to the CGT market's sustained growth and broader adoption is the inherent uncertainty surrounding the long-term efficacy and durability of these novel therapies. The intellectual property (IP) landscape surrounding cell and gene therapies presents a complex and formidable barrier to competition, potentially limiting market access and perpetuating high costs. Request a Customized Copy of the Cell and Gene Therapy Market Report @ Key questions answered in this report: What is the size of the cellular and gene therapy market, and what is its expected growth rate? What are the primary driving factors that push the Cell and Gene Therapy market forward? What are the Cell and Gene Therapy Industry's top companies? What are the different categories that the Cell and Gene Therapy Market caters to? What will be the fastest-growing segment or region? In the value chain, what role do essential players play? What is the procedure for getting a free copy of the Cell and Gene Therapy market sample report and company profiles? Key Offerings: Market Share, Size & Forecast by Revenue | 2025−2034 Market Dynamics – Growth Drivers, Restraints, Investment Opportunities, and Leading Trends Market Segmentation – A detailed analysis by Types of Services, by End-User Services, and by regions Competitive Landscape – Top Key Vendors and Other Prominent Vendors Buy this Premium Cell and Gene Therapy Research Report | Fast Delivery Available - [220+ Pages] @ Regional Analysis The Cell and Gene Therapy Market is segmented into various regions, including North America, Europe, Asia-Pacific, and LAMEA. Here is a brief overview of each region: North America: North America dominates cell and gene therapy (CGT) markets due to strong health infrastructure, substantial R&D investments, and a supportive regulatory landscape. Leading biotech entities present and contributed to the creation and growth of excellent clinical trial networks that stimulate innovation and commercialization. The U.S. is a pivotal market for early adoption of therapies like CAR-T. Strong governmental support, acceptance by the patient populace, and insurance reimbursements act as factors that augment market growth. The FDA's accelerated approval pathway and breakthrough therapy designations also allow the availability of treatments years before commercialization. Europe: Cell and gene therapy market in Europe is witnessing rapid growth with increasing investments in biotechnology and favorable regulatory initiatives such as the PRIME scheme by the European Medicines Agency. Countries such as Germany, the UK, and France are flower centers of research and clinical trials augmented by strong collaborative partnerships between public and private organizations. The rising incidence of chronic and rare diseases is further driving market demand. However, heterogeneous healthcare systems and reimbursement procedures act as hurdles in market access. Several initiatives have been set to harmonize the regulatory framework and set up an efficient infrastructure for manufacturing. Asia-Pacific: Considered an emerging and fast-growing market for cell and gene therapies, this region sees rising healthcare expenditure, development of biotechnology sectors, and an increasing prevalence of various diseases as high growth factors. Besides China, Japan has invested heavily in CGT research, clinical trials, and manufacturing setups; South Korea, too, is gradually forging ahead. Regulatory bodies have increasingly adapted to facilitate faster approvals, while growing awareness among clinicians and patients is also contributing to market growth. Although infrastructural and reimbursement constraints exist in some pockets, expanding government schemes and international collaborations further bolster the market growth. LAMEA: The LAMEA cell and gene therapy market is at an early stage but is expected to grow at a significant rate owing to the increasing burden of diseases along with the improving healthcare infrastructure. Latin America, the Middle East, and Africa have witnessed heightened activity in biotechnology and clinical research but remain limited in market penetration due to regulatory hitches, absence of specialized facilities, and reimbursement issues. The increasing partnership with the international biotech players along with the increasing initiatives from the government for better healthcare accessibility is expected to propel the market growth. Enhancements in awareness and infrastructure will establish LAMEA as a promising region for CGT, particularly in addressing unmet medical needs related to rare and genetic diseases. Request a Customized Copy of the Cell and Gene Therapy Market Report @ (We customized your report to meet your specific research requirements. Inquire with our sales team about customizing your report.) Still, Looking for More Information? Do you want data for inclusion in magazines, case studies, research papers, or media? Email Directly Here with Detail Information: support@ Browse the full 'Cell and Gene Therapy Market Size, Trends and Insights By Therapy Type (Cell Theray, Gene Therapy), By Therapeutic Class (Cardiovascular Disease, Cancer, Genetic Disorder, Rare Diseases, Oncology, Hematology, Ophthalmology, Infectious Disease, Neurological Disorders, Others), By Delivery Method (In Vivo, Ex Vivo), By End-User (Hospitals, Cancer Care Centers, Others), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025–2034' Report at List of the prominent players in the Cell and Gene Therapy Market: Amgen Inc. Biogen Inc. Biomarin Pharmaceutical Inc. Bluebird Bio Inc. (Celgene Corporation) Bristol-Myers Squibb Company Freeline Therapeutics Ltd. Gene Biotherapeutics Gilead Sciences Inc. Horama S.A. JCR Pharmaceuticals Co. Ltd. Kolon TissueGene Inc. MeiraGTx Limited Novartis International AG Orchard Therapeutics Plc. Organogenesis Inc. Others Click Here to Access a Free Sample Report of the Global Cell and Gene Therapy Market @ Spectacular Deals Comprehensive coverage Maximum number of market tables and figures We offer a subscription-based option. Best price guarantee We offer free 35% or 60 hours of customization. We also offer free post-sale service assistance. You will receive a 25% discount on your subsequent purchases. Service guarantees are available. The author will create a customized market brief for you. Browse More Related Reports: Advanced Genomics Market: Advanced Genomics Market Size, Trends and Insights By Offering (Products, Services), By Technology (Sequencing, PCR, Gene Editing, Flow Cytometry, Microarrays, In Situ Hybridization), By Application (Diagnostics, Drug Discovery and Development, Precision Medicine, Agriculture), By End-user (Hospitals and Clinics, Research Centers, Pharmaceutical and Biotechnology Companies), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025 – 2034 Battery-Free Implants Market: Battery-Free Implants Market Size, Trends and Insights By Product Type (Neural Implants, Cardiac Implants , Cochlear Implants, Orthopedic Implants, Others), By Technology (Energy Harvesting, Wireless Power Transfer, RFID/NFC-based Systems), By End User (Hospitals & Clinics, Ambulatory Surgical Centers, Research Institutions, Specialty Clinics), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025–2034 Duchenne Muscular Dystrophy Drugs Market: Duchenne Muscular Dystrophy Drugs Market Size, Trends and Insights By Drug Type (Exon skipping drugs, Corticosteroids, Gene therapy, Other drug types), By Route of Administration (Oral, Injectable), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025 – 2034 Psychiatric Digital Biomarkers Market: Psychiatric Digital Biomarkers Market Size, Trends and Insights By Type (Wearable, Mobile based Applications, Sensors, Other Type), By Clinical Practice (Diagnostic Psychiatric Digital Biomarkers, Monitoring Psychiatric Digital Biomarkers, Predictive and Prognostic Psychiatric Digital Biomarkers, Other Clinical Practice), By End Use (Healthcare Companies, Healthcare Providers, Payers, Other End Use), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025–2034 Electrophysiology Devices Market: Electrophysiology Devices Market Size, Trends and Insights By Type (Ablation Catheters, Diagnostic Catheters, Mapping System, Accessories, Others), By Application (Atrial Fibrillation, Supraventricular Tachycardia (SVT), Ventricular Tachycardia, Others), By End User (Hospitals & ASCs, Specialty Clinics & Diagnostic Centers), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025 – 2034 Vitreous Tamponades Market: Vitreous Tamponades Market Size, Trends and Insights By Product Type (Gaseous Tamponades, Liquid Tamponades, Silicone Oil, Perchloroethane, Others), By Application (Retinal Detachment, Macular Hole, Endophthalmis, Intraocular Tumours, Vitreous Haemorrhage, Others), By End User (Hospitals, Ophthalmology Clinics), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025 – 2034 Heart Valve Devices Market: Heart Valve Devices Market Size, Trends and Insights By Product Type (Mechanical Heart Valves, Biological Heart Valves, Transcatheter Heart Valves), By Treatment (Valve Repair, Valve Replacement), By End-User (Hospitals & Clinics, Ambulatory Surgical Centers, Cardiac Centers, Others), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025 – 2034 Infusion Therapy Market: Infusion Therapy Market Size, Trends and Insights By Product (Infusion Pump, Intravenous Sets, IV Cannulas, Needleless Connectors), By Application (Anti-infective, Hydration Therapy, Chemotherapy, Enteral Nutrition, Parenteral Nutrition, Specialty Pharmaceuticals, Other), By End-user (Hospitals, Ambulatory Surgical Centers, Clinics, Homecare Settings), and By Region - Global Industry Overview, Statistical Data, Competitive Analysis, Share, Outlook, and Forecast 2025 – 2034 The We have segmented the Cell and Gene Therapy Market as follows: By Therapy Type Cell Theray Gene Therapy By Therapeutic Class Cardiovascular Disease Cancer Genetic Disorder Rare Diseases Oncology Hematology Ophthalmology Infectious Disease Neurological Disorders Others By Delivery Method In Vivo Ex Vivo By End-User Hospitals Cancer Care Centers Others Click Here to Get a Free Sample Report of the Global Cell and Gene Therapy Market @ Regional Coverage: North America U.S. Canada Mexico Rest of North America Europe Germany France U.K. Russia Italy Spain Netherlands Rest of Europe Asia Pacific China Japan India New Zealand Australia South Korea Taiwan Rest of Asia Pacific The Middle East & Africa Saudi Arabia UAE Egypt Kuwait South Africa Rest of the Middle East & Africa Latin America Brazil Argentina Rest of Latin America This cell and gene therapy market research and analysis report contains answers to the following questions: Which Trends Are Causing These Developments? Who Are the Global Key Players in This Cell and Gene Therapy Market? What are the company profiles, product information, and contact details for these key players? What Was the Global Market Status of the Cell and Gene Therapy Market? What Was the Capacity, Production Value, Cost and PROFIT of the Cell and Gene Therapy markets? What Is the Current Market Status of the Cell and Gene Therapy industries? What's the market's competition in this industry, both company-wise and country-wise? What's the Market Analysis of Cell and Gene Therapy Market by Considering Applications and Types? What Are Projections of the Global Cell and Gene Therapy Industry Considering Capacity, Production, and Production Value? What Will Be the estimate of Cost and Profit? What Will Be Market Share, Supply, and Consumption? What about imports and exports? What Is Cell and Gene Therapy Market Chain Analysis by Upstream Raw Materials and Downstream Industry? What Is the Economic Impact On Cell and Gene Therapy Industry? What are Global Macroeconomic Environment Analysis Results? What Are Global Macroeconomic Environment Development Trends? What Are Market Dynamics of Cell and Gene Therapy Market? What Are Challenges and Opportunities? What Should Be Entry Strategies, Countermeasures to Economic Impact, and Marketing Channels for Cell and Gene Therapy Industry? Click Here to Access a Free Sample Report of the Global Cell and Gene Therapy Market @ Reasons to Purchase Cell and Gene Therapy Market Report Cell and Gene Therapy Market Report provides qualitative and quantitative analysis of the market based on segmentation involving economic and non-economic factors. Cell and Gene Therapy Market report outlines market value (USD) data for each segment and sub-segment. This report indicates the region and segment expected to witness the fastest growth and dominate the market. Cell and Gene Therapy Market Analysis by geography highlights the consumption of the product/service in the region and indicates the factors affecting the market within each region. The competitive landscape incorporates the market ranking of the major players, along with new service/product launches, partnerships, business expansions, and acquisitions in the past five years of companies profiled. The report includes extensive company profiles, which include company overviews, insights, product benchmarking, and SWOT analyses for the major market players. The Industry's current and future market outlook concerning recent developments (which involve growth opportunities and drivers as well as challenges and restraints of both emerging and developed regions). Cell and Gene Therapy Market Includes in-depth market analysis from various perspectives through Porter's five forces analysis and offers context for the market through Value Chain. Reasons for the Research Report The study provides a thorough overview of the global Cell and Gene Therapy market. Compare your performance to that of the market as a whole. Aim to maintain competitiveness while innovations from established leaders drive market growth. Buy this Premium Cell and Gene Therapy Research Report | Fast Delivery Available - [220+ Pages] @ What does the report include? Drivers, restrictions, and opportunities are among the qualitative elements covered in the worldwide Cell and Gene Therapy market analysis. The competitive environment of current and potential participants in the Cell and Gene Therapy market is covered in the report, as well as those companies' strategic product development ambitions. According to the component, application, and industry vertical, this study analyzes the market qualitatively and quantitatively. Additionally, the report offers comparable data for the important regions. We have provided actual market sizes and forecasts for each of the aforementioned segments. Who should buy this report? Participants and stakeholders in the worldwide Cell and Gene Therapy market should find this report useful. The research will be useful to all market participants in the Cell and Gene Therapy industry. Managers in the Cell and Gene Therapy sector are interested in publishing up-to-date and projected data about the worldwide Cell and Gene Therapy market. Governmental agencies, regulatory bodies, decision-makers, and organizations want to invest in Cell and Gene Therapy products' market trends. Market insights are sought by analysts, researchers, educators, strategy managers, and government organizations to develop plans. Request a Customized Copy of the Cell and Gene Therapy Market Report @ About Custom Market Insights: Custom Market Insights is a market research and advisory company delivering business insights and market research reports to large, small, and medium-scale enterprises. We assist clients with strategies and business policies and regularly work toward achieving sustainable growth in their respective domains. CMI offers a comprehensive solution, from data collection to investment advice. Our company's expert analysis digs out essential factors that help us understand the significance and impact of market dynamics. The professional experts utilize client insights to inform strategies for estimating future declines, forecasting opportunities for growth, and conducting consumer surveys. 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- Yahoo
CRISPR Therapeutics AG (CRSP): A Bull Case Theory
We came across a bullish thesis on CRISPR Therapeutics AG (CRSP) on Two Natural Cap's Substack. In this article, we will summarize the bulls' thesis on CRSP. CRISPR Therapeutics AG (CRSP)'s share was trading at $41.52 as of 11th June. CRSP's trailing and forward P/E were 22.55 and 23.36 respectively according to Yahoo Finance. A scientist peering into a microscope, researching the next gene therapy breakthrough. CRISPR has re-entered the spotlight thanks to a groundbreaking case where the technology was used to treat Baby KJ, born with a severe genetic liver disorder caused by a CPS1 enzyme deficiency. With only a 50% survival rate in infancy, traditional treatment included heavy medication and a strict low-protein diet. But researchers rapidly developed a personalized CRISPR therapy that KJ has now received three times, allowing him to eat a regular diet and significantly reduce medication use. This marks a major milestone not only for KJ but for the broader gene-editing landscape. At its core, CRISPR-Cas9 was originally a bacterial defense mechanism that evolved to recognize and cut viral DNA. Scientists have since repurposed this system, using modified guide RNAs and engineered Cas9 proteins to target human DNA with remarkable precision. In most therapeutic uses, CRISPR creates double-stranded breaks that are imperfectly repaired by the body—helpful for knocking out malfunctioning genes, as seen with CASGEVY, the FDA-approved therapy for sickle cell disease and beta thalassemia. KJ's case went further, employing 'base editing' to fix a single-letter DNA error using a deaminase-modified CRISPR complex that swapped an adenine for a guanine, correcting the mutation without a DNA break. This was delivered via lipid nanoparticles (LNPs), offering dosing flexibility over traditional viral vectors. The success not only validates the science behind base editing but also uplifts companies like Beam, CRISPR Therapeutics, and Intellia after years of market declines. It also spotlights Danaher's Aldevron and Acuitas Therapeutics, whose platforms were instrumental in the therapy's development, underscoring the growing viability of personalized gene medicine. We previously covered a bullish thesis on CRISPR Therapeutics (CRSP) from wallstreetbets by MADD-Scientis, emphasizing Casgevy's revenue potential, strong cash runway, and optionality in oncology and cardiology. Since the coverage, the stock price has appreciated by roughly 1.3%. Two Natural Cap's thesis adds depth by spotlighting CRSP's role in a groundbreaking base-editing therapy, reinforcing the platform's adaptability. Both cases point to CRSP's leadership in curative gene editing. CRISPR Therapeutics AG (CRSP) is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 29 hedge fund portfolios held CRSP at the end of the first quarter which was 27 in the previous quarter. While we acknowledge the risk and potential of CRSP as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the best short-term AI stock. READ NEXT: 8 Best Wide Moat Stocks to Buy Now and 30 Most Important AI Stocks According to BlackRock. Disclosure: None. This article was originally published at Insider Monkey. 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
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Business Standard
5 days ago
- Science
- Business Standard
Gene-edited rice may pose IPR risks, scientists say in letter to PM
A group of scientists calling themselves the 'Agricultural Scientists Manch' have written to the Prime Minister denouncing the recent release of India's first genetically edited rice varieties, stating that the technology used—CRISPR-Cas9—could face intellectual property rights (IPR) issues, potentially negating the real advantage Indian farmers could derive from genome-edited rice. The Centre has repeatedly clarified that all IPR issues related to gene editing are being thoroughly addressed, and a panel has been formed for this purpose. It has also assured that the government will acquire the necessary licences for gene-editing technologies and that no additional burden will fall on Indian farmers. The letter, signed by nearly 20 former and current scientists including Sharad Nimbalkar, former vice-chancellor of Panjabrao Deshmukh Agriculture University, Akola, stated that although CRISPR-Cas9 is a highly precise technology, it can still go off-track, potentially causing severe undesirable consequences. It also warned that large-scale use of gene-edited rice could contaminate India's native rice germplasm. The scientists further expressed concern that unresolved IPR issues could lead to Indian farmers becoming dependent on foreign seed technologies. They added that the newly released rice varieties were cleared without adequate evaluation. The letter was also marked to Agriculture Minister Shivraj Singh Chouhan, Environment Minister Bhupender Yadav, Health Minister Jagat Prakash Nadda and Science and Technology Minister Jitendra Singh. Last month, in a significant development, the government released two genome-edited rice varieties for the first time in the country. These varieties are expected to increase per-hectare yields by up to 30 per cent and mature 15–20 days earlier than existing varieties.
New York Post
19-06-2025
- Health
- New York Post
Could Down syndrome be eliminated? Scientists say cutting-edge gene editing tool could cut out extra chromosome
Cutting-edge gene editing technology could eradicate Down syndrome, according to Japanese scientists. Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in the United States, is caused by the presence of an extra copy of chromosome 21. The extra chromosome, also known as trisomy 21, causes cellular overactivity, compromises a range of processes within the body, and can manifest in distinctive physical traits, learning difficulties, and health concerns. Advertisement Now new research out of Mie University in Japan suggests that by using the DNA-modifying tech CRISPR, it is possible to remove the surplus chromosome in affected cells and bring cellular behavior closer to typical function. 3 Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in the United States, is caused by the presence of an extra copy of chromosome 21. Mongkolchon – CRISPR-Cas9 is a gene-editing system that utilizes an enzyme to identify specific DNA sequences. Once the enzyme locates a matching site, it snips through the DNA strands. Ryotaro Hashizume and his colleagues designed CRISPR guides to target only the trisomy 21 chromosome, a process called allele-specific editing, which directs the cutting enzyme to the desired spot. Advertisement When they used it on lab-grown cells, removing the extra copy of the gene normalized the way the genes expressed themselves in the body — suggesting that the genetic burden had been removed. They also found that after the extra chromosome was removed, genes tied to nervous system development were more active and those related to metabolism were less active. This backs up previous research that found extra copies of chromosome 21 disrupt brain development during early fetal growth. Researchers also tested their CRISPR guides on skin fibroblasts, which are mature, non-stem cells taken from people with Down syndrome. Advertisement In these fully developed cells, the editing method successfully removed the extra chromosome in a number of cases. 3 Hashizume and his team designed CRISPR guides to target only the trisomy 21 chromosome, a process called allele-specific editing, which directs the cutting enzyme to the desired spot. Gorodenkoff – After removal, these corrected cells grew faster and had a shorter doubling time than untreated cells, suggesting that removing the extra chromosome may help with the biological strain that slows down cell growth. Advertisement But the CRISPR can affect healthy chromosomes, too, and researchers are refining their program so that it only attaches to the extra copy of chromosome 21. This work proves that, rather than making small fixes, CRISPR can eliminate an entire chromosome. The scientists published their findings in PNAS Nexus. Hashizume and his team are hopeful that their work may be used to design regenerative therapies and treatments that address genetic surplus at its source. 3 Researchers also tested their CRISPR guides on skin fibroblasts, mature, non-stem cells taken from people with Down syndrome. Yakobchuk Olena – Researchers will continue to analyze the risks of DNA changes and monitor how modified cells function over time and their viability in real-world settings. A recent case study explored a medical mystery related to Down syndrome; the brain of an American woman with Down syndrome showed all the classic signs of Alzheimer's disease, yet she remained symptom-free throughout her lifetime. Advertisement People with Down syndrome face a much higher risk of developing Alzheimer's-related dementia as they age — an estimated three to five times higher than the general population. Scientists are still working to pinpoint the exact cause, but it's believed that the extra copy of chromosome 21 drives the overproduction of amyloid precursor protein. This excess production leads to the buildup of amyloid beta plaques in the brain, a hallmark of Alzheimer's disease.
