Cell and Gene Therapy Market Global Industry Analysis from 2020 to 2027 Explored in Latest Research – Illadel Graff Supply
A complete research offering of comprehensive analysis of the market share, size, recent developments, and trends can be availed in this latest report by Big Market Research.
As per the report, theGlobal Cell and Gene Therapy Marketis anticipated to witness significant growth during the forecast period from 2020to 2027.
The report provides brief summary and detailed insights of the market by collecting data from the industry experts and several prevalent in the market. Besides this, the report offers a detailed analysis of geographical areas and describes the competitive scenario to assist investor, prominent players, and new entrants to obtain a major share of the global Cell and Gene Therapy market.
Our analysis involves the study of the market taking into consideration the impact of the COVID-19 pandemic. Please get in touch with us to get your hands on an exhaustive coverage of the impact of the current situation on the market. Our expert team of analysts will provide as per report customized to your requirement. For more connect with us at help@bigmarketresearch.com or call toll free: +1-800-910-6452
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The report presents a summary of each market segment such as type, end-user, applications, and region. With the help of pie charts, graphs, comparison tables, and progress charts a complete overview of the market share, size, and revenue, and growth patterns areaccessible in the report.
Additionally, an outline of each market segments such as end user, product type, application, and region are offered in the report.The market across various regions is analyzed in the report which includes North America, Europe, Asia-Pacific, and LAMEA.The report explains future trends and growth opportunities in every region. These insights help in understanding the global trends in the market and form strategies to be implemented in the future. Moreover, the research report profiles some of the leading companies in the global Cell and Gene Therapy industry. It mentions their strategic initiatives and offers a brief about their business. Some of the players profiled in the global Cell and Gene Therapy market include:
Key players in the Cell and Gene Therapy covers :CHIESI FarmaceuticiTego SciencesMolMedCollPlantAmgenTakeda Pharmaceutical CompanyAVITA MedicalStempeutics ResearchOsiris TherapeuticsAPAC BiotechBiosolutionVericelGilead SciencesCorestemMedipostCO.DONNovartis AGOrganogenesisOrchard TherapeuticsDendreonSpark TherapeuticsJW CreaGeneAnGesJapan Tissue EngineeringGC PharmaJCR Pharmaceuticals
Analysts have also stated the research and development activities of these companies and provided complete information about their existing products and services. Additionally, the report offers a superior view over different factors driving or constraining the development of the market.
The Cell and Gene Therapy can be split based on product types, major applications, and important countries as follows:
The basis of applications, the Cell and Gene Therapy from 2015 to 2025 covers:HospitalsWound Care CentersCancer Care CentersAmbulatory Surgical CentersOthers
The basis of types, the Cell and Gene Therapy from 2015 to 2025 is primarily split into:Cell TherapyGene Therapy
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The report clearly shows that the Cell and Gene Therapy industry has achieved remarkable progress since 2025 with numerous significant developments boosting the growth of the market. This report is prepared based on a detailed assessment of the industry by experts. To conclude, stakeholders, investors, product managers, marketing executives, and other experts in search of factual data on supply, demand, and future predictions would find the report valuable.
The report constitutes:Chapter 1 provides an overview of Cell and Gene Therapy market, containing global revenue, global production, sales, and CAGR. The forecast and analysis of Cell and Gene Therapy market by type, application, and region are also presented in this chapter.Chapter 2 is about the market landscape and major players. It provides competitive situation and market concentration status along with the basic information of these players.Chapter 3 provides a full-scale analysis of major players in Cell and Gene Therapy industry. The basic information, as well as the profiles, applications and specifications of products market performance along with Business Overview are offered.Chapter 4 gives a worldwide view of Cell and Gene Therapy market. It includes production, market share revenue, price, and the growth rate by type.Chapter 5 focuses on the application of Cell and Gene Therapy, by analyzing the consumption and its growth rate of each application.Chapter 6 is about production, consumption, export, and import of Cell and Gene Therapy in each region.Chapter 7 pays attention to the production, revenue, price and gross margin of Cell and Gene Therapy in markets of different regions. The analysis on production, revenue, price and gross margin of the global market is covered in this part.Chapter 8 concentrates on manufacturing analysis, including key raw material analysis, cost structure analysis and process analysis, making up a comprehensive analysis of manufacturing cost.Chapter 9 introduces the industrial chain of Cell and Gene Therapy. Industrial chain analysis, raw material sources and downstream buyers are analyzed in this chapter.Chapter 10 provides clear insights into market dynamics.Chapter 11 prospects the whole Cell and Gene Therapy market, including the global production and revenue forecast, regional forecast. It also foresees the Cell and Gene Therapy market by type and application.Chapter 12 concludes the research findings and refines all the highlights of the study.Chapter 13 introduces the research methodology and sources of research data for your understanding.
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Cell and Gene Therapy Market Global Industry Analysis from 2020 to 2027 Explored in Latest Research - Illadel Graff Supply
Recommendation and review posted by Bethany Smith
CRISPR/Cas9 Gene-Editing Therapy CTX001 for Severe Hemoglobinopathies Accepted for Plenary Presentation at the 62nd American Society of Hematology…
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 04, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data in seven patients from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition, which will take place virtually from December 5-8, 2020. Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center, will deliver the presentation on behalf of all the authors on December 6, 2020.
An abstract posted online today includes data from five patients with three months to 15 months of follow-up after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and data from two patients with three months and 12 months of follow-up in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Additional data will be presented at ASH, including longer-duration follow-up data for the patients included in the abstract and data for additional patients with greater than three months of follow-up.
CTX001 is being investigated in these two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.
The accepted abstract is now available on the ASH conference website.
About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.
Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.
CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.
About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.
About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.
About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.
CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data and plans to present data at the annual ASH meeting and exposition; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
CRISPR THERAPEUTICS word mark and design logo and CTX001 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.
About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the expectations and plans to present data at the annual ASH meeting and exposition, the development, including expected timeline for development, and potential benefits of CTX001, our plans and expectations for our clinical trials and clinical trial sites, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com
CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com
Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187
Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275
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CRISPR/Cas9 Gene-Editing Therapy CTX001 for Severe Hemoglobinopathies Accepted for Plenary Presentation at the 62nd American Society of Hematology...
Recommendation and review posted by Bethany Smith
Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 – PRNewswire
Avantor executives joined other industry leaders at virtual Cell & Gene Therapy Bioprocessing & Commercialization Conference
Panels discussed critical topics including process efficiency and scaling solutions
RADNOR, Pa., Nov. 2, 2020 /PRNewswire/ --Executives from Avantor Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries, recently provided expert insight at the Cell & Gene Therapy Bioprocessing & Commercialization Virtual Conference. Cell and gene therapy (C>) are two of the most revolutionary applications driving the biopharmaceutical industry.
In separate panel discussions with other global industry leaders, Dr. Ger Brophy, EVP, Biopharma Production at Avantor and Claudia Berrn, SVP, Business Development and Commercial Operations at Avantor, each addressed novel considerations and challenges facing the biopharmaceutical industry, including the impact of COVID-19. They highlighted innovation to help drive the creation and commercialization of life-changing, personalized C> treatments.
In a panel on the evolution of C> and the path toward scalability and manufacturability of these treatments, Dr. Brophy gave his perspective on this growing need for the bioprocessing industry.
"Genuine progress is being made in the long-standing battle to effectively treat and control disease, and cell & gene therapies will only continue to unlock new frontiers in medicine," said Dr. Brophy. "We're beginning to see more clearly the issues that need to be addressed, and know that if products are to scale and become more accessible to patients worldwide, there needs to be efficiency in operations. There is an absolute requirement for automation, both to reduce variability and to generate process efficiencies. At Avantor, we are ready to make the essential materials and technologies available to companies leading the charge and treating patients. Innovation and agility are central to how we're partnering with the industry to resolve these issues."
On a separate panel, Ms. Berrn highlighted how the industry is navigating the challenges and impact of the global pandemic, from the fragmentation of supply chains to clinical trial disruption.
"In this critical moment for the industry and the world, it is more important than ever for leaders in the cell & gene therapy community to come together to strategize and share ideas," said Ms. Berrn. "Avantor is actively working with the world's leading pharmaceutical and biotechnology companies to accelerate the production of novel treatments. Collectively, our goal is to mitigate any challenges in the process of taking a groundbreaking treatment from its initial scientific discovery to delivery in treating patients."
Avantor provides products for biopharma production workflows, including cell and gene therapy offering. Search 'Avantor biopharma' in your browser.
About AvantorAvantor, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. One of our greatest strengths comes from having a global infrastructure that is strategically located to support the needs of our customers. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries.We set science in motion to create a better world. For information, visit avantorsciences.com and find us on LinkedIn, Twitter and Facebook.
Robert DonohoeSenior Director, Corporate CommunicationsAvantorM: 484-688-4730[emailprotected]
Source: Avantor and Financial News
SOURCE Avantor and Financial News
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Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 - PRNewswire
Recommendation and review posted by Bethany Smith
Cell and Gene Therapy Market Reviewed in a New Study Eurowire – Eurowire
This report studies the Cell and Gene Therapy to get Covid-19 marketplace with Many details of the industry such as the market size, market standing, marketplace trends and prediction, the report also provides brief advice of their opponents as well as the particular growth opportunities with key market drivers. Locate the complete Cell and Gene Therapy to get Covid-19 market evaluation segmented by firms, area, type and software in the document.
New sellers from the marketplace are facing tough competition from Established foreign vendors as they fight with technological inventions, quality and reliability problems. The report will answer questions regarding the present market changes and the reach of competition, opportunity cost and much more.
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The report discusses the various Kinds of options for While the areas considered in the range of the report include North America, Europe, and assorted others. The analysis also highlights on how climbing digital security dangers is altering the industry situation.
Development policies and strategies are discussed along with Manufacturing processes and cost structures will also be examined. This report also claims import/export consumption, supply and demand Statistics, price, cost, earnings and gross earnings.
This report concentrates on the international Cell and Gene Therapy for Covid-19 Status, future prediction, growth opportunity, key marketplace and players.
The following players are covered in this report:
Amgen Inc.
bluebird bio, Inc.
Dendreon Pharmaceuticals LLC.
Fibrocell Science, Inc.
Human Stem Cells Institute
Kite Pharma, Inc.
Kolon TissueGene, Inc.
Novartis AG
Orchard Therapeutics plc.
Organogenesis Holdings Inc.
Pfizer, Inc.
RENOVA THERAPEUTICS
Shanghai Sunway Biotech Co., Ltd.,
Sibiono GeneTech Co. Ltd.,
Spark Therapeutics, Inc.
Vericel Corporation
ViroMed Co., Ltd.
The Cell and Gene Therapy for Covid-19 marketplace is a comprehensive record Which supplies a meticulous summary of the market share, size, trends, demand, product evaluation, program analysis, regional perspective, competitive strategies, predictions, and strategies affecting the Cell and Gene Therapy for Covid-19 Industry. The report contains a thorough analysis of this marketplace competitive landscape, with the assistance of comprehensive business profiles, SWOT analysis, project feasibility analysis, and a lot of other specifics about the essential companies working on the marketplace.
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The analysis aims Covid-19 in global sector.
Breakdown Data by Type
Rare Diseases
Oncology
Hematology
Cardiovascular
Ophthalmology
Neurology
Other Therapeutic Classes
Cell and Gene Therapy Breakdown Data by Application
Pharmaceutical and Biotechnology Companies
Research and Academic Institutions
Contract Research Organizations (CROs)
Hospital
Others
Based on regional and country-level analysis, the Cell and Gene Therapy market has been segmented as follows:
North America
United States
Canada
Europe
Germany
France
U.K.
Italy
Russia
Nordic
Rest of Europe
Asia-Pacific
China
Japan
South Korea
Southeast Asia
India
Australia
Rest of Asia-Pacific
Latin America
Mexico
Brazil
Middle East & Africa
Turkey
Saudi Arabia
UAE
Rest of Middle East & Africa
In the competitive analysis section of the report, leading as well as prominent players of the global Cell and Gene Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.
The following players are covered in this report:
Amgen Inc.
bluebird bio, Inc.
Dendreon Pharmaceuticals LLC.
Fibrocell Science, Inc.
Human Stem Cells Institute
Kite Pharma, Inc.
Kolon TissueGene, Inc.
Novartis AG
Orchard Therapeutics plc.
Organogenesis Holdings Inc.
Pfizer, Inc.
RENOVA THERAPEUTICS
Shanghai Sunway Biotech Co., Ltd.,
Sibiono GeneTech Co. Ltd.,
Spark Therapeutics, Inc.
Vericel Corporation
ViroMed Co., Ltd.
The Cell and Gene Therapy for Covid-19 market study report entirely Covers the very important data of their capacity, manufacturing, value, cost/profit, supply/demand import/export, further separated by country and company, and from application/type for the best possible upgraded information representation from the statistics, tables, pie graph, and charts. These data representations give predictive information concerning the potential estimations for persuasive market development. The detailed and in depth understanding concerning our publishers makes us from the box in the event of market evaluation.
Key questions Answered within this report
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Table of Contents
Chapter 1: Global Cell and Gene Therapy for Covid-19 Market Overview
Chapter 2: Cell and Gene Therapy for Covid-19 Market Data Analysis
Chapter 3: Cell and Gene Therapy for Covid-19 Technical Data Analysis
Chapter 4: Cell and Gene Therapy for Covid-19 Government Policy and News
Chapter 5: Global Cell and Gene Therapy for Covid-19 Market Manufacturing Process and Cost Structure
Chapter 6: Cell and Gene Therapy for Covid-19 Productions Supply Sales Demand Market Status and Forecast
More:
Cell and Gene Therapy Market Reviewed in a New Study Eurowire - Eurowire
Recommendation and review posted by Bethany Smith
$129.96 Billion Non-Oncology Precision Medicine Market, 2019-2020 & 2030 – Research Alliance Across Industry and Academia Accelerating Market…
DUBLIN, Nov. 4, 2020 /PRNewswire/ -- The "Global Non-Oncology Precision Medicine Market: Focus on Application Area, Ecosystem Type, Country Data (15 Countries) - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.
The global non-oncology precision medicine market was valued at $41.1 billion in 2019, and it is expected to grow at an impressive double-digit rate of 11.03% and reach a value of $129.96 billion in 2030.
The precision medicine approach is used for several disease management, such as oncology, immunology, neurology, and infectious diseases. In the non-oncology precision medicine market, the application of molecular biology is for studying the cause of a patient's disease at the molecular level for diseases other than cancer, so that target-based therapies or individualized therapies can be applied to cure the patient's health-related problems.
The existing non-oncology precision medicine market is favored by multiple factors, which include rising government initiatives, coupled up with the general population's growing awareness pertaining to molecular level diagnosis and treatment. Additionally, the increasing number of advancements in molecular diagnostics, more key players interested in investing for the development of novel personalized therapies, and increasing FDA approvals for such drugs among others.
Competitive Landscape
The non-oncology precision medicine market witnessed 59 collaborations and partnerships, four fundings and investments, 44 product launches and enhancements,26 regulatory and legal activities,18 mergers and acquisitions, and six business expansions during the period January 2016-August 2020.
Key Topics Covered:
1 Product Definition
2 Scope of the Research Study
3 Research Methodology3.1 Primary Data Sources3.2 Secondary Data Sources3.3 Market Estimation Model3.4 Selection Criteria for Company Profiles
4 Market Overview4.1 Introduction4.2 Enabling Technologies of Precision Medicine Emphasizing Focus on Prevention4.3 Promising Potential and Role of Precision Medicine
5 Impact of COVID-19 Pandemic on Global Non-Oncology Precision Medicine Market5.1 Unprecedented Demand for Non-Oncology Precision Medicine Due to COVID-195.2 COVID-19 Affecting Supply Chain of Non-Oncology Precision Medicine-Based Therapies5.3 Measures Taken to Address Current Problem
6 Global Non-Oncology Precision Medicine Market: Industry Analysis6.1 Legal and Regulatory Framework and Requirements6.1.1 North America6.1.1.1 Regulatory Requirements in U.S.6.1.2 Europe6.1.3 Asia-Pacific6.2 Patent Landscape
7 Global Non-Oncology Precision Medicine Market: Market Dynamics7.1 Overview7.2 Market Driving Factors7.2.1 Improved Availability of Targeted Therapies and Decreasing Trial and Error-Based Prescription7.2.2 Reduced Chances of Adverse Drug Reactions7.2.3 Decreasing Overall Cost of Genome Sequencing7.2.4 Rising Prevalence of Chronic Diseases7.3 Market Restraining Factors7.3.1 Unclear Regulatory Framework and Reimbursement Scenario for Personalized Medicine7.3.2 Limited Knowledge About Molecular Mechanism/Interaction7.3.3 Integrating and Securing Electronic Health Records7.4 Market Opportunities7.4.1 Research Alliance Across Industry and Academia Accelerating Market Entry7.4.2 Strong Pipeline and Increasing FDA Approvals
8 Global Non-Oncology Precision Medicine Market: Competitive Landscape8.1 Key Strategies and Developments8.1.1 Product Offerings8.1.2 Partnerships and Alliances8.1.3 Regulatory and Legal8.1.4 Mergers and Acquisitions8.1.5 Funding and Investments8.2 Market Share Analysis8.3 Growth-Share Analysis (by Company), 2019
9 Global Non-Oncology Precision Medicine Market (by Application), $Billion, 2019-20309.1 Overview9.2 Infectious Diseases9.2.1 Respiratory Infections9.2.2 Gastrointestinal Infections9.2.3 Sexually Transmitted Infections9.2.4 Others9.3 Neurology/Physiatry9.3.1 Neurodegenerative Disorders9.3.2 Neuropsychiatric Disorders9.3.3 Others9.4 Lifestyle and Endocrinology9.5 Cardiovascular9.5.1 Cardiac Myopathies and Arrhythmia9.5.2 Others9.6 Gastroenterology9.7 Other Applications
10 Global Non-Oncology Precision Medicine Market (by Ecosystem), $Billion, 2019-203010.1 Overview10.2 Applied Sciences10.2.1 Genomics10.2.2 Global Non-Oncology Precision Medicine Genomics Market (by Technology)10.2.2.1 Polymerase Chain Reaction (PCR)10.2.2.2 Precision Medicine Next-Generation Sequencing (PM NGS)10.2.2.3 Genome Editing10.2.2.4 Other Technologies10.2.3 Pharmacogenomics10.2.4 Other Applied Sciences10.3 Precision Diagnostics10.3.1 Molecular Diagnostics (MDx)10.3.2 Medical Imaging10.4 Digital Health and Information Technology10.4.1 Clinical Decision Support Systems (CDSS)10.4.2 Big Data Analytics10.4.3 IT Infrastructure10.4.4 Genomics Informatics10.4.5 In-Silico Informatics10.4.6 Mobile Health10.5 Precision Therapeutics10.5.1 Clinical Trials10.5.2 Cell Therapy10.5.3 Drug Discovery and Research10.5.4 Gene Therapy
11 Global Non-Oncology Precision Medicine Market (by Region), $Billion, 2019-2030
12 Company Profiles12.1 Company Overview12.2 Role of Abbott Laboratories in Global Non-Oncology Precision Medicine Market12.3 Financials12.4 SWOT Analysis
For more information about this report visit https://www.researchandmarkets.com/r/j2b11m
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$129.96 Billion Non-Oncology Precision Medicine Market, 2019-2020 & 2030 - Research Alliance Across Industry and Academia Accelerating Market...
Recommendation and review posted by Bethany Smith
The Cancer Gene Therapy Market to get on to the elliptical growth mode in the next decade – Eurowire
The gene therapy market has undergone a series of transformation from past few years. Initially gene therapy of monogenetic diseases was popular; however now the gene therapy market is gaining popularity for treatment of cancer. Cancer gene therapy refers to a process of treatment of cancer by inserting therapeutic DNA into patient. Cancer gene therapy is gaining popularity as a result of its success rate in preclinical and clinical trial stages. The most common technique for cancer gene therapy involves replacing a mutated gene that is causing cancer with a healthy copy of gene. Other technique involves inactivation of a gene that is not working properly. A new technique that is being introduced to cancer gene therapy market involves insertion of new genes into the body that would help to fight against tumor cells.
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North America followed by Europe dominates the global market for cancer gene therapy market due to large number of aging population and technological advancement in the region. Asia is expected to witness high growth in cancer gene therapy market, due to increasing government initiatives, rising economy and improvement in healthcare infrastructure in the region. Some of the key driving forces for cancer gene therapy market in emerging countries are increasing R&D investment, large pool of patients and rising government funding.
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Several unmet medical needs for treatment of cancer have encouraged R&D of cancer gene therapy. Various factors, such as increasing prevalence of cancer, rising government initiatives, increasing funding from various government and non-government organizations, are driving the global cancer gene therapy market. In addition, ethical acceptance of gene therapy for treatment of diseases and growing popularity of DNA vaccines is driving the global cancer gene therapy market. However, less awareness and high cost involved in treatment are restraining the growth of global market for cancer gene therapy.
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Innovation of some innovative therapy with better success rate is expected to offer good opportunity for cancer gene therapy. In addition, despite of high cost involved in R&D, pharmaceutical companies are showing increased interest in this field. This is expected to offer good potential for cancer gene therapy market. Currently, most of the cancer gene therapy products are in clinical trial phases. The market is expected to rise after clearance or success of these products from clinical trials. Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech
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The Cancer Gene Therapy Market to get on to the elliptical growth mode in the next decade - Eurowire
Recommendation and review posted by Bethany Smith
Gene Therapy Size Inclined to Project Higher Revenue Share during the Stated Forecast Period 2020 2026 – The Cloud Tribune
Fact.MR has recently announced the addition of a new study on the global Gene Therapy market to its document repository. This report is intended to provide a detailed analysis of all vital factors impacting negatively or positively on the development of the Gene Therapy market during the assessment period of 20AA to 20YY. Apart from this, the latest study highlights historical and current trends together with forecast on future trends shaping the growth of the global Gene Therapy market in the years ahead. This aside, the assessment of Gene Therapy Market presents analysis on challenges and opportunities during the assessment period.
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This growth is attributed to a plethora of factors. Increased research and development activities, growing investments in the Gene Therapy, and favorable regulatory policies by government bodies of several countries are some of the important factors driving the Gene Therapy market growth.
The recent COVID-19 (Coronavirus) pandemic has affected the revenues of companies engaged in almost every industrial sector. The companies operating in the global Gene Therapy market are also not an exception to this. The recently published Gene Therapy market research report sheds light on various strategies employed by industry leaders to sustain during this critical scenario of COVID-19 pandemic. In addition to this, the study includes various tactics used by market players to resolve many challenges related to the distribution of their products during the lockdown period.
The latest research report gives trustworthy data on different consumption patterns in various geographical regions.
Important regions covered in this report on the global Gene Therapy market include:
Based on product type, the global Gene Therapy market is classified into:
The research report profiles important players working in the Gene Therapy market. In addition to this, it covers data on the competitive landscape and various strategies utilized by these players to maintain their leading position in the market for Gene Therapy. Mergers, acquisitions, partnerships, collaborations, joint ventures, and new product launches are some of the key strategies utilized by vendors in the Gene Therapy market. Apart from this, the assessment gives important data on weaknesses, strengths, threats, and opportunities for all vendors working in the market for Gene Therapy.
The list of prominent players in the global Gene Therapy market includes the following names:
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Gene Therapy Size Inclined to Project Higher Revenue Share during the Stated Forecast Period 2020 2026 - The Cloud Tribune
Recommendation and review posted by Bethany Smith
BRIEFNovasep and Handl sign gene therapy product development and manufacturing deal – The Pharma Letter
Belgian companies Novasep and Handl Therapeutics have signed an agreement for the development and manufacturing of an adeno associated virus (AAV)-based gene therapy drug candidate for the treatment of neurodegenerative diseases.
Handl is developing multiple therapeutic AAV-based programs in collaboration with the Katholieke Universiteit Leuven, with the Center for Applied Medical Research of the University of Navarra, Spain, and with the Biomedical Neuroscience Institute of the University of Chile. The company is currently conducting invesrigational new drug (IND)-enabling pre-clinical studies.
Under the terms of the agreement, Novasep will develop and manufacture AAV vectors designed for these programs and will supply drug substance and drug product to support Handl Therapeutics preclinical and clinical studies.
Michael Linden, co-founder and head of research and development at Handl, said: We are excited to engage with Novasep to develop GMP manufacture capabilities for our novel gene therapies and are happy to have identified an outstanding partner right on our doorstep here in Belgium.
Cedric Volanti, Novaseps president of biopharma solutions, said: This new agreement recognizes Novaseps expertise in the field of viral vectors and will contribute to the important development of the cell and gene therapy market in Belgium.
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BRIEFNovasep and Handl sign gene therapy product development and manufacturing deal - The Pharma Letter
Recommendation and review posted by Bethany Smith
Cancer Gene Therapy Market Report Delivering Growth Analysis With Key Trends Of Top Companies (2020-2026) – PRnews Leader
A new research study has been presented by dataintelo offering a comprehensive analysis on the Global Cancer Gene Therapy Market where user can benefit from the complete market research report with all the required useful information about this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. The report discusses all major market aspects with expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments.
The Cancer Gene Therapy Market report provides a detailed analysis of the global market size, regional and country-level market size, segment growth, market share, competitive landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunity analysis, strategic market growth analysis, product launches, and technological innovations.
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Major Players Covered in this Report are: AdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene
Global Cancer Gene Therapy Market SegmentationThis market has been divided into Types, Applications, and Regions. The growth of each segment provides an accurate calculation and forecast of sales by Types and Applications, in terms of volume and value for the period between 2020 and 2026. This analysis can help you expand your business by targeting qualified niche markets. Market share data is available on the global and regional level. Regions covered in the report are North America, Europe, Asia Pacific, the Middle East & Africa, and Latin America. Research analysts understand the competitive strengths and provide competitive analysis for each competitor separately.
By Types:Oncolytic VirotherapyGene TransferGene-Induced Immunotherapy
By Applications:HospitalsDiagnostics CentersResearch Institutes
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Global Cancer Gene Therapy Market Regions and Countries Level AnalysisRegional analysis is a highly comprehensive part of this report. This segmentation sheds light on the sales of the Cancer Gene Therapy on regional- and country-level. This data provides a detailed and accurate country-wise volume analysis and region-wise market size analysis of the global market.
The report offers an in-depth assessment of the growth and other aspects of the market in key countries including the US, Canada, Mexico, Germany, France, the UK, Russia, Italy, China, Japan, South Korea, India, Australia, Brazil, and Saudi Arabia. The competitive landscape chapter of the global market report provides key information about market players such as company overview, total revenue (financials), market potential, global presence, Cancer Gene Therapy sales and revenue generated, market share, prices, production sites and facilities, products offered, and strategies adopted. This study provides Cancer Gene Therapy sales, revenue, and market share for each player covered in this report for a period between 2016 and 2020.
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Table of Contents1. Executive Summary2. Assumptions and Acronyms Used3. Research Methodology4. Market Overview5. Global Market Analysis and Forecast, by Types6. Global Market Analysis and Forecast, by Applications7. Global Market Analysis and Forecast, by Regions8. North America Market Analysis and Forecast9. Latin America Market Analysis and Forecast10. Europe Market Analysis and Forecast11. Asia Pacific Market Analysis and Forecast12. Middle East & Africa Market Analysis and Forecast13. Competition Landscape
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Cancer Gene Therapy Market Report Delivering Growth Analysis With Key Trends Of Top Companies (2020-2026) - PRnews Leader
Recommendation and review posted by Bethany Smith
The life and death of a boy whose diagnosis brought hope to other patients – STAT
No boy should have a last stretch of days. But Bertrand Might lived his as well as any boy could: There was a Star Trek marathon with his brother and sister, sunrises on the lakeshore, and visits with family in parks, beaches, and backyards anywhere they could safely gather during the pandemic.
His father, Matt Might, said it ended up being an unplanned farewell for 12-year-old Bertrand, whose health had always been precarious. He was the first person in the world diagnosed with a particular neurodegenerative condition that causes developmental delays, seizure-like activity in the brain, and frequent infections.
One of those infections, unrelated to Covid-19, led to his death on Oct. 23 after he spiraled into septic shock. But if his passing came too soon, it did not come before his life led to crucial discoveries for dozens of children with his condition.
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What he did with NGLY1 alone was pretty powerful, said Matt Might, referring to the gene involved in his sons disease. After years of research, it was the discovery of a double mutation in Bertrands NGLY1 gene, and the constellation of symptoms linked to it, that explained the cause of the illness and built a worldwide community around it.
There are 70 families on the patient mailing list right now for a disease that eight years ago didnt exist, Might said.
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Bertrand also inspired a quest by his father, an artificial intelligence expert and computer programmer, to employ precision medicine on a wider scale, using genetic data to help tailor treatments to patients with rare and hard-to-treat diseases like his sons.
Might began that work initially to help Bertrand, but it led to a stint on President Obamas precision medicine initiative and the creation of a new precision medicine institute (PMI) he now leads at the University of Alabama, Birmingham.
PMI was founded on this algorithm that Bertrand taught me, Might said. How do you try to therapeutically modulate a specific genetic target? There is a central game plan we use every time somebody comes in.
Might and his team examine what gene is involved in a persons condition and whether it is under-reactive, over-reactive, toxic, or missing altogether. The answers to those questions form the basis for a scientific process that often gives patients hope when conventional medicine has failed to provide an accurate diagnosis or effective treatments. A permanent endowment has been established at UAB in Bertrands name to fund advanced diagnostics and research to identify novel therapies for patients with no other options.
In Bertrands case, the double mutation in NGLY1 left him without an enzyme that facilitates the recycling of cellular waste. It severely limited his mobility, requiring him to use a wheelchair, and also impaired his liver function and ability to communicate.
Still, Bertrand drove the science of his condition while enduring countless hospitalizations, often due to infections that made it difficult to breathe.
Throughout his life, he developed a love for dolphins and an aquarium his parents set up in his bedroom. He spent hours learning words and reading with his father and mother, Cristina, and he bonded with his younger brother and sister over movies and video games.
Im proud of Bertrand in multiple ways, Might said. I would often tell people to imagine a being created without the ability to even feel malice. He was just a pure being, and I loved that about him.
In recent years, the science that led to his diagnosis has also begun to unravel the biology of NGLY1 deficiency and its impact on patients. A project sponsored by the National Institutes of Health is underway to screen hundreds of thousands of molecules for therapeutic potential against the illness, while Might has used computational methods to identify treatments that showed efficacy in animal subjects.
On Bertrands last day in the hospital, as his condition continued to deteriorate, his father read him an email from the father of another patient with his illness. It said that the Food and Drug Administration seemed pleased with pre-clinical studies of a gene therapy for NGLY1 and outlined a series of steps toward a clinical trial.
It was so meaningful to know the community that Bertrand formed has spawned efforts well beyond my own, Might said. And in the end, he died in a world where the hope of a cure existed.
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The life and death of a boy whose diagnosis brought hope to other patients - STAT
Recommendation and review posted by Bethany Smith
Gene Therapy For Heart Failure Market Report Provide the Development Strategies Adopted by Key Industry Players to Understand Competitive Scenario of…
This report studies the Gene Therapy For Heart Failure to get Covid-19 marketplace with Many details of the industry like the market size, market standing, market trends and forecast, the report also provides brief information of their opponents and the specific growth opportunities with key market drivers. Find the complete Gene Therapy For Heart Failure to get Covid-19 market evaluation segmented by firms, region, type and applications in the document.
New sellers in the market are facing tough competition from Established international vendors as they fight with technological innovations, quality and reliability problems. The report will answer questions regarding the current market changes and the reach of competition, opportunity cost and much more.
This Press Release will help you to understand the Volume, growth with COVID19 Impact Analysis. Click HERE To get SAMPLE PDF (Including TOC, Table & Figures) at: @ https://chronicalmarketresearch.com/request-for-sample-report/64529
The report discusses the various types of options for While the regions considered in the scope of the report include North America, Europe, and assorted others. The study also emphasizes on how climbing digital security dangers is changing the market scenario.
Development policies and strategies are discussed along with Manufacturing processes and cost structures are also analyzed. This report also claims import/export consumption, supply and demand Figures, price, cost, earnings and gross earnings.
This report focuses on the international Gene Therapy For Heart Failure for Covid-19 Status, future prediction, growth opportunity, key marketplace and key players.
The following players are covered in this report:
Askbio
RENOVACORINC
Uniqure
DINAQOR
PRECIGEN
Heartseed
Cardior
NOVARTIS
Blue Rock
The Gene Therapy For Heart Failure for Covid-19 market is a comprehensive record Which offers a meticulous overview of the market share, size, trends, demand, product evaluation, application analysis, regional perspective, competitive strategies, forecasts, and strategies impacting the Gene Therapy For Heart Failure for Covid-19 Industry. The report includes a thorough analysis of the market competitive landscape, with the assistance of comprehensive business profiles, SWOT analysis, project feasibility analysis, and several other specifics about the essential companies working on the marketplace.
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The study aims Of this report are:
To examine and forecast the market size of Gene Therapy For Heart Failure for Covid-19 in global sector.
To analyze the International Important players, SWOT analysis, value and International market share for best players.
To specify, describe and forecast the market by type, end use And area.
To analyze and compare the market standing and forecast among Global significant regions.
To analyze the International key regions market potential and Benefit, opportunity and challenge, restraints and risks.
To identify Substantial trends and factors driving or Inhibiting the market development.
To analyze the opportunities in the market for stakeholders
To strategically analyze each submarket with regard to Individual growth tendency and their contribution to the industry
To analyze competitive developments such as expansions,
To profile the key players and comprehensively Analyze their growth plans.
Breakdown Data by Type
Gene Modification
Gene Replacement
Others
Gene Therapy For Heart Failure Breakdown Data by Application
Hospital
Laboratory
Others
Based on regional and country-level analysis, the Gene Therapy For Heart Failure market has been segmented as follows:
North America
United States
Canada
Europe
Germany
France
U.K.
Italy
Russia
Nordic
Rest of Europe
Asia-Pacific
China
Japan
South Korea
Southeast Asia
India
Australia
Rest of Asia-Pacific
Latin America
Mexico
Brazil
Middle East & Africa
Turkey
Saudi Arabia
UAE
Rest of Middle East & Africa
The Gene Therapy For Heart Failure for Covid-19 market research report completely Covers the vital statistics of their capacity, production, value, cost/profit, supply/demand import/export, further divided by company and country, and by application/type for the best possible updated data representation from the figures, tables, pie chart, and graphs. These data representations give predictive data concerning the future estimations for persuasive market growth. The detailed and comprehensive understanding about our publishers makes us out of the box in case of market evaluation.
Key questions Answered in this report
What is going to the market size be in 2026 and what will the Growth rate be?
What will be the key market trends?
Whats driving this economy?
Which will be the challenges to promote growth?
Who will be the key vendors in this market space?
What are the market opportunities and threats faced by the Key vendors?
What are the strengths and weaknesses of the key vendors?
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Table of Contents
Chapter 1: Global Gene Therapy For Heart Failure for Covid-19 Market Overview
Chapter 2: Gene Therapy For Heart Failure for Covid-19 Market Data Analysis
Chapter 3: Gene Therapy For Heart Failure for Covid-19 Technical Data Analysis
Chapter 4: Gene Therapy For Heart Failure for Covid-19 Government Policy and News
Chapter 5: Global Gene Therapy For Heart Failure for Covid-19 Market Manufacturing Process and Cost Structure
Chapter 6: Gene Therapy For Heart Failure for Covid-19 Productions Supply Sales Demand Market Status and Forecast
Chapter 7: Gene Therapy For Heart Failure for Covid-19 Key Manufacturers
Chapter 8: Up and Down Stream Industry Analysis
Chapter 9: Marketing Strategy -Gene Therapy For Heart Failure for Covid-19 Analysis
Chapter 10: Gene Therapy For Heart Failure for Covid-19 Development Trend Analysis
Chapter 11: Global Gene Therapy For Heart Failure for Covid-19 Market New Project Investment Feasibility Analysis
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Originally posted here:
Gene Therapy For Heart Failure Market Report Provide the Development Strategies Adopted by Key Industry Players to Understand Competitive Scenario of...
Recommendation and review posted by Bethany Smith
LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA) – GlobeNewswire
LEXINGTON, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need.
Commenting on the announcement, Daniel Gruskin, M.D., Senior Vice President, Head of Clinical Development of LogicBio, said, We are pleased the FDA has granted Fast Track designation to LB-001 in recognition of the importance of our efforts to bring a durable treatment to the children suffering from MMA. With Fast Track status, we plan to continue to work closely with the FDA to fully utilize the opportunities presented by this designation to make LB-001 available to patients as quickly as possible.
About Fast Track Designation
The FDAs Fast Track designation is a process designed to expedite or facilitate the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It may also allow for priority or rolling review of a companys Biologics License Application (BLA).
About LogicBio Therapeutics
LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration withTakedato research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.
LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.
LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the timing, progress and results of the Companys research and development activities, including those related to LB-001, and the significance and benefits of receiving the FDAs Fast Track designation for LB-001 in MMA. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements inthe United Statesand in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with theU.S. Securities and Exchange Commission(SEC), including, without limitation, the Companys Annual Report on Form 10-K filed onMarch 16, 2020with theSEC, the Companys Quarterly Report on Form 10-Q filed onMay 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with theSEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.
Contact:
Matthias JaffeChief Financial OfficerMjaffe@logicbio.com617-245-0399
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LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA) - GlobeNewswire
Recommendation and review posted by Bethany Smith
Mustang Bio Announces MB-106 Data Selected for Presentation at the 62nd American Society of Hematology (ASH) Annual Meeting – GlobeNewswire
WORCESTER, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphoma (NHL), have been selected for a poster presentation at the 62nd American Society of Hematology (ASH) Annual Meeting, which is being held virtually from December 5 8, 2020. MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Research Center (Fred Hutch).
In the abstract posted today on the ASH website, Fred Hutch reported on four patients treated following a major revision in the cell manufacturing process. Complete remissions were observed in two follicular lymphoma patients (one each at dose levels 1 and 2), as well as a partial remission in a mantle cell lymphoma patient at dose level 2 and progressive disease in a follicular lymphoma patient at dose level 1. Dose level 1 was 3.3 x 105 CAR-T cells/kg and dose level 2 was 1 x 106 CAR-T cells/kg. As previously disclosed, no responses were seen in the 7 patients treated prior to cell process revision. Among the 11 total patients reported in the abstract, there was one occurrence of cytokine release syndrome (grade 3 unexplained alkaline phosphatase elevation in the setting of fever in a patient treated prior to cell process revision) and no occurrences of immune effector cell-associated neurotoxicity syndrome (any grade). No dose-limiting toxicity was observed.
Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are pleased that Fred Hutch will present interim data at ASH from the ongoing Phase 1/2 trial of MB-106, and at that time we expect to disclose data on at least eight total patients treated since the major cell process revision. In February 2020, we reported that the first patient treated in the trial with the revised MB-106 manufacturing process achieved a complete response at the lowest starting dose. The additional data disclosed today further indicate that MB-106 has an extremely favorable safety profile with evidence of promising clinical activity, even at low dose levels. We look forward to continuing progress on this CD20-targeted CAR T cell therapy program for patients with relapsed or refractory B-cell non-Hodgkin lymphomas.
Details of the presentation are as follows:
Title: Third Generation CD20 Targeted CAR T-Cell Therapy (MB-106) for Treatment of Patients with Relapsed/Refractory B-Cell Non-Hodgkin LymphomaSession: 704. Immunotherapies: Poster IAbstract: 1443Date and Time: Saturday, December 5, 2020, 10:00 a.m. - 6:30 p.m. ETPresenter: Mazyar Shadman, M.D., M.P.H., Associate Professor, Clinical Research Division, Fred Hutch, Seattle, WA
For more information, please visit the 62nd ASH Annual Meeting and Exposition website at https://www.hematology.org/meetings/annual-meeting/abstracts.
About B-cell Non-Hodgkin Lymphoma (NHL)There are several forms of NHL, including follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma, lymphoplasmacytic lymphoma and small lymphocytic lymphoma, which account collectively for about 45% of all cases of NHL. Most types of NHL are incurable with available therapies, except for allogenic hematopoietic stem cell transplant (allo-SCT). More than 70,000 new cases of B-cell NHL are diagnosed each year in the United States, and more than 19,000 patients die annually due to this group of diseases.
About MB-106 (CD20-targeted CAR T Cell Therapy)CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustangs research partner, Fred Hutchinson Cancer Research Center (Fred Hutch), in the laboratory of Oliver Press, M.D., Ph.D., and Brian Till, M.D., in the Clinical Research Division and exclusively licensed to Mustang Bio in 2017. MB-106 has been optimized as a third-generation CAR derived from a fully human antibody and is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in B-cell non-Hodgkin lymphoma patients. Additional information on the trial can be found at http://www.clinicaltrials.gov using the identifier NCT03277729.
About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.
ForwardLooking Statements
This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com
Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com
Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com
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Mustang Bio Announces MB-106 Data Selected for Presentation at the 62nd American Society of Hematology (ASH) Annual Meeting - GlobeNewswire
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LogicBio Therapeutics Announces Appointment of Veteran Biotech Executive Mariana Nacht, Ph.D., as Chief Scientific Officer and Kyle Chiang, Ph.D.,…
LEXINGTON, Mass., Nov. 03, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today announced the appointment of Mariana Nacht, Ph.D., as chief scientific officer, effective Nov. 30, 2020, and the promotion of Kyle Chiang, Ph.D., to chief operating officer, effective Nov. 2, 2020.
Dr. Nacht brings more than 20 years of experience in both large and small biotech companies to her role at LogicBio. Most recently, she served as CSO and was a founding executive team member of Cereius, where she led a small internal research team and a group of collaborators to develop radiolabeled proteins for the treatment of brain metastases. Before that, she served as CSO of Vivid Biosciences, a functional precision medicine company, where she was also a founding executive team member. Dr. Nacht has also served in key scientific roles at Padlock Therapeutics (acquired by Bristol Myer Squibb in 2014) and Avila Therapeutics, a platform company that developed covalent irreversible inhibitors and was acquired by Celgene in 2012. Earlier in her career, she spent a decade working at Genzyme (now Sanofi Genzyme), where she led anti-angiogenesis and oncology target discovery efforts. Dr. Nacht received her B.S. in biology from Tufts University and her Ph.D. from the University of Pennsylvania.
We are proud to expand our leadership team as we prepare to launch our first clinical trial in pediatric patients with methylmalonic acidemia (MMA) and continue to advance both our GeneRide and Next Generation Capsid platforms, said Fred Chereau, CEO of LogicBio. Mariana brings terrific expertise in novel therapeutic platforms as well as deep experience in building and leading strong scientific teams to her position as CSO. Were thrilled to welcome her to LogicBio as we move into this exciting next phase of progress. Im also delighted to have Kyle promoted to our core leadership team. He has provided essential guidance on pipeline strategy and program development from the early days of LogicBio and he will continue to be an important voice in shaping our future growth.
I am very enthusiastic about the potential for the GeneRide platform to transform care for pediatric patients with rare genetic diseases, Dr. Nacht said. We all enter this field to make a difference for patients, and I am excited to be joining LogicBio just as LB-001, our lead program for children with MMA, is about to enter the clinic with the Phase 1/2 SUNRISE trial. Beyond LB-001, I look forward to further advancing LogicBios pipeline with the goal of bringing more durable and transformational therapies to people living with devastating genetic diseases.
Dr. Chiang was the second employee at LogicBio and has held positions of increasing responsibility since joining the team as director of translational science in 2016. Most recently, he served as vice president, product strategy, where he led LB-001 through early regulatory interactions and managed LogicBios collaboration with the Childrens Medical Research Institute to develop more potent and more easily manufacturable AAV capsids for gene therapy and genome editing applications. Before joining LogicBio, Dr. Chiang led aTyr Pharmas ATYR1940 program from discovery through early clinical development for patients with facioscapulohumeral muscular dystrophy. Dr. Chiang received his B.S. in biochemistry and cell biology from the University of California, San Diego and his Ph.D. in macromolecular cellular structure and chemistry from the Scripps Research Institute.
LogicBio also announced today that Bryan Yoon, Esq., the companys chief administrative officer, general counsel and corporate secretary, will be departing from the company effective Nov. 6, 2020. I want to thank Bryan for his contributions to LogicBio and we wish him the best in his next challenge, Mr. Chereau said.
AboutLogicBioTherapeuticsLogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration withTakedato research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.
LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.
LogicBio is headquartered in Lexington, Mass. For more information, please visit http://www.logicbio.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the timing, progress and results of the Companys strategic directives and its research and development activities, including those related to LB-001 and its pipeline. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.
Contacts:
Investors:Matthew LaneGilmartin Investor Relationmatt@gilmartinir.com
Media:Stephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333
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LogicBio Therapeutics Announces Appointment of Veteran Biotech Executive Mariana Nacht, Ph.D., as Chief Scientific Officer and Kyle Chiang, Ph.D.,...
Recommendation and review posted by Bethany Smith
Global Viral Vector and Plasmid Manufacturing Market Insights and In-Depth Analysis 2020-2027 | Brammer Bio, Cobra Biologics, Cell, and Gene Therapy…
The Global Viral Vector and Plasmid Manufacturing Market Report, published by Emergen Research, is an all-encompassing study of the Viral Vector and Plasmid Manufacturing market, which emphasizes the estimated market size, share, value, volume, and futuristic outlook. The report details the major products and application rangesavailable in this market, highlighting the revenue, price, sales, production, growth rate, and market share of each segment and sub-segment. The essential market presented in the report in a tabular format is aimed at helping readers interpret the global market dynamics. The market intelligence report delivers a profound study of the Viral Vector and Plasmid Manufacturing business domain, discussing its principal aspects, such as the import & export dynamics, production, consumption, sales channels, and consumer bases in the major regional segments. The global Viral Vector and Plasmid Manufacturing market, which held a significant value of USD 358.4 Million in 2019, is projected to reach a whopping market value of USD 1.12 billion by 2027 at a stunning CAGR of 14.3%.
The latest research report expounds on the profound impact of the COVID-19 pandemic on the global Viral Vector and Plasmid Manufacturing market and its crucial segments. The report thoroughly examines the vital market-influencing factors and considers the COVID-19 pandemic as a major contributing element to this business verticals potential downturn. As per industry analysts, the Viral Vector and Plasmid Manufacturing industry is currently reeling from the pandemics gripping effects, and remarkable changes have been observed in the market dynamics and demand trends over recent months.The financial strains being suffered by each business organization in this industry have significantly slowed down their progress. Additionally, the report assesses the pandemics overall impact on the global market growth and involves a future COVID-19 impact assessment to help readers make prudent business decisions.
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Key Players Participating in the Industry:
Brammer Bio, Cobra Biologics, Cell, and Gene Therapy Catapult, FinVector Vision Therapies, Fujifilm Diosynth Biotechnologies, MassBiologics, SIRION Biotech, Merck KGaA Inc., Thermo Fisher Scientific, and Unique NV,
For the purpose of this report, Emergen Research has segmented into the global Viral Vector and Plasmid Manufacturing Market on the basis of Vector Type, Workflow, Disease, Application, End-User, and region:
Regional Fragmentation:
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The report succinctly analyzes the potential revenue growth of the Viral Vector and Plasmid Manufacturing market, its development patterns, and the future market trends. Moreover, the research report specializes in the meticulous analysis of the present market scenario. It forecasts the market value, volume, drivers, restraints, demand and supply ratio, production capacity, import/export status, growth rate, and other critical aspects over the projected period.
The report offers a panoramic view of the Viral Vector and Plasmid Manufacturing market on both global and regional levels. The study is further supported by key statistical data and industry-verified facts.The study strives to carefully gauge the present and future market growth prospects, untapped avenues, demand and consumption patterns, and the crucial factors poised to impact each market regions revenue potential. Therefore, the report scrutinizes the numerous growth trends & prospects and the significant challenges and threats that the market players might face in the upcoming years.
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Our expertise umbrellas the technological environment of all major industries, and our services help you map your actions to ensure optimal yield. Our analysts utilize their market proficiency to offer actionable insights that help our clients implement profitable strategies and optimize their return on investment. Our services are wide-ranging, right from technological environment analysis to technological profiling that highlights the existing opportunities in the market you can capitalize on to stay ahead of your competitors.
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Hemophilia Gene Therapy Market Analysis Of Growth, Size, Trends Progress And Challenges Till Upcoming Year 2020 2027 – Eurowire
Hemophilia Gene Therapy Market Overview
The Hemophilia Gene Therapy Market is rapidly undergoing changes. These changes are indicators of market growth. This year-over-year uptrend of the market is pointing towards a steady yet elliptical rise for the next septennial 2020-2027.
The Hemophilia Gene Therapy Market report imparts a detailed insight into the forecast period (2018-2027). The assessment contains different sections that act as the pillars for the market. Factors such as market patterns help businesses in laying out a blueprint of actions to be taken over the course of the specified time frame. The report also brushes over the other components market drivers, limitations, growth opportunities, and hurdles to highlight the effect of these variables over the market. The report also delineates the key markets that can be targeted for starting a business venture. The market drivers and restrictions are intrinsic components while opportunities and hurdles are extrinsic factors of the market. The Hemophilia Gene Therapy Market Report gives a point-of-view of the cyclic development of the market, in the specified time frame, in the offing.
Hemophilia Gene Therapy Market: Competitive Landscape
The report covers the competitive analysis of the market. As the demand is driven by a shoppers paying capacity and the pace of item development, the report shows the important regions that will drive growth. This step is necessary to maintain the balance between invested capitals with profit generated. The report indicates the benefits of the efficient assembly line and powerful promotion and dispersion tactics.
Moreover, the market report has a dedicated section covering the existing market players. This section exclusively shares insight into the budget reports of big-league members of the Hemophilia Gene Therapy Market. A survey was conducted to impart crucial knowledge about product benchmarking and SWOT examination. A brief profile segment likewise incorporates the business framework and capital-related data (inflow and outflow). The organizations mentioned in the report can be altered as per the customers prerequisites.
Following key players have been profiled with the help of proven research methodologies:
The Hemophilia Gene Therapy Market has been examined into different global market segments such as type, applications and global geographies. Each and every global market segment has been studied to get informative insights into various global regions.
Hemophilia Gene Therapy Market Segmentation:
Hemophilia Gene Therapy Market Segment by Type:
Hemophilia Gene Therapy Market Segment by Application:
Hemophilia Gene Therapy Market Segment by Global Presence:
North America Latin America Middle East Asia-Pacific Africa Europe
The report has been aggregated by using a couple of research methodologies such as primary and secondary research techniques. It helps in collecting informative pieces of professional information for deriving effective insights into the market. This informative report helps in making well informed and strategic decisions throughout the forecast period.
Hemophilia Gene Therapy Market: Scope of the Report
This report gives a comprehensive understanding of the Hemophilia Gene Therapy Market, under investigation. The market recommendations pointed out in the report are the outcomes of in-depth exploration and interviews. This crucial step helped in understanding the expectations of the market. Our dedicated team of experts examined the social, political, and financial factors alongside the current market elements influencing the Hemophilia Gene Therapy Market. This way, organizations can align their businesses, according to the latest trends, for generating profits and building a client base.
For getting a detailed understanding of the market growth, our experts have also added the section incorporating Porters Five Forces. The five powers that drive the investigation- purchasers bartering power, suppliers dealing power, the threat from newbies and substitutes, and the level of rivalry in the Hemophilia Gene Therapy Market. The report also encompasses the role of participants (intermediaries and end-users) who fuel the market. The focal point of the report is the market landscape, filled with competitors, of the Hemophilia Gene Therapy Market.
Key questions answered through this analytical market research report include:
What are the latest trends, new patterns and technological advancements in the Hemophilia Gene Therapy Market? Which factors are influencing the Hemophilia Gene Therapy Market over the forecast period? What are the global challenges, threats and risks in the Hemophilia Gene Therapy Market? Which factors are propelling and restraining the Hemophilia Gene Therapy Market? What are the demanding global regions of the Hemophilia Gene Therapy Market? What will be the global market size over the coming future? What are the different effective business strategies followed by global companies?
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Hemophilia Gene Therapy Market Analysis Of Growth, Size, Trends Progress And Challenges Till Upcoming Year 2020 2027 - Eurowire
Recommendation and review posted by Bethany Smith
Cancer Gene Therapy Market Business Opportunities, Segmentation Analysis, Top Companies and Forecast to 2028 – Eurowire
Beathan Report has published the Global report on The Cancer Gene Therapy market, which is made up of advice about each of the important parameters of this market like consumption and the manufacturing patterns coupled with all the earnings patterns for the forecast period. Concerning creation aspect, the report provides complete detailed analysis about the manufacturing processes combined with the gross financials accumulated by the top most manufacturers working within this business. The primary facet of this Cancer Gene Therapy market thats covered in the report helps the clients and the associations to better understand the business profile concerning drivers, restraints, challenges, and opportunities pertaining and affecting the marketplace dynamics.
Request Sample Report @ https://beathanreports.com/request-for-sample-report/7828
Key market players
Major competitors identified in this market include Adaptimmune, Bluebird bio, Celgene, Shanghai Sunway Biotech, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Altor BioScience, Amgen, Argenx, BioCancell, GlaxoSmithKline, Merck, OncoGenex Pharmaceuticals, Transgene, etc.
Based on the Region:
Asia-Pacific (China, Japan, South Korea, India and ASEAN)
North America (US and Canada)
Europe (Germany, France, UK and Italy)
Rest of World (Latin America, Middle East & Africa)
According to the Cancer Gene Therapy report, the
COVID-19 has majorly affected the Overall worldwide companies and itll take a enormous time for the company recovery. Vast majority of the business sectors have realigned their company strategies, priorities, and have amended their economic planning in order to remain in the company and keep their standing on the global platform. The thorough evaluation of this Cancer Gene Therapy market will enable the new market entrants to obtain reliable market approaches and strategy strong action plans for the prediction period.
Based on the Type:
Oncolytic Virotherapy
Gene Transfer
Gene-Induced Immunotherapy
Based on the Application:
Hospitals
Diagnostics Centers
Research Institutes
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Major highlights of this Cancer Gene Therapy market report:
* COVID-19 effect on the earnings Streams of the Cancer Gene Therapy market players.
* Statistics of the overall sales quantity And general market revenue.
* Industry trends breakdowns.
* Estimated expansion rate of this Cancer Gene Therapy Market.
* Pros and cons of the direct and
* In-depth information about the important Distributors, traders, and dealers.
Table of Contents : Cancer Gene Therapy Market
Part 01: Executive Summary
Part 02: Scope Of The Report
Part 03: Research Methodology
Part 04: Market Landscape
Part 05: Pipeline Analysis
Part 06: Market Sizing
Part 07: Five Forces Analysis
Part 08: Market Segmentation
Part 09: Customer Landscape
Part 10: Regional Landscape
Part 11: Decision Framework
Part 12: Drivers And Challenges
Part 13: Market Trends
Part 14: Vendor Landscape
Part 15: Vendor Analysis
Part 16: Appendix
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Cancer Gene Therapy Market Business Opportunities, Segmentation Analysis, Top Companies and Forecast to 2028 - Eurowire
Recommendation and review posted by Bethany Smith
CRISPR Genome Editing Market Research Report- Opportunities & Challenges With Totally Different Segments, Forecast- 2026 – The Think Curiouser
CRISPR Genome Editing Market report examines Product Specification, Major Segments in Focus, Geographic Focus, Production Capacity, Production, Sales Performance of key players in market which gives you deep understanding of competitive scenario of CRISPR Genome Editing market. CRISPR Genome Editing industry research report enables reader to dive into consumers mind.
Download Free Sample PDF of CRISPR Genome Editing [emailprotected] https://www.researchmoz.us/enquiry.php?type=S&repid=2602918
CRISPR Genome Editing market competition by top manufacturers, with production, price, and revenue (value) and market share for each manufacturer; the top players including:
Editas MedicineCRISPR TherapeuticsHorizon DiscoverySigma-AldrichGenscriptSangamo BiosciencesLonza GroupIntegrated DNA TechnologiesNew England BiolabsOrigene TechnologiesTransposagen BiopharmaceuticalsThermo Fisher ScientificCaribou Biosciences
Goal Audience of CRISPR Genome Editing Market 2019 Forecast to 2026 Market:
Raw material suppliers->>Distributors/traders/wholesalers/suppliers->>Regulatory bodies, including government agencies and NGO->>Commercial research & development (R&D) institutions->>Importers and exporters->>Government organizations, research organizations, and consulting firms->>Trade associations and CRISPR Genome Editing industry bodies->>End-use industries
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Based onProduct Type, CRISPR Genome Editing market report displays the manufacture, profits, value, and market segment and growth rate of each type, covers:
Genetic EngineeringGene LibraryHuman Stem Cells
Based onend users/applications, CRISPR Genome Editing market report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate for each application, this can be divided into:
Biotechnology CompaniesPharmaceutical Companies
CRISPR Genome Editing Market 2019 forecast to 2026 Market Segment by Regions, regional analysis covers
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Some of the important topics in CRISPR Genome Editing Market Research Report:
For More Information Kindly Contact:ResearchMozMr. Rohit Bhisey,90 State Street,Albany NY,United States 12207Tel: +1-518-621-2074USA-Canada Toll Free: 866-997-4948Email:[emailprotected]Follow us on LinkedIn @http://bit.ly/1TBmnVGMedia Release:https://www.researchmoz.us/pressreleaseFollow me on :https://nextgenmarketresearch.blogspot.com/
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CRISPR Genome Editing Market Research Report- Opportunities & Challenges With Totally Different Segments, Forecast- 2026 - The Think Curiouser
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Latest News 2020: CRISPR Technology Market by Coronavirus-COVID19 Impact Analysis With Top Manufacturers Analysis | Top Players: Thermo Fisher…
The latest CRISPR Technologymarket report estimates the opportunities and current market scenario, providing insights and updates about the corresponding segments involved in the global CRISPR Technologymarket for the forecast period of 2020-2026. The report provides detailed assessment of key market dynamics and comprehensive information about the structure of the CRISPR Technologyindustry. This market study contains exclusive insights into how the global CRISPR Technologymarket is predicted to grow during the forecast period.
The primary objective of the CRISPR Technology market report is to provide insights regarding opportunities in the market that are supporting the transformation of global businesses associated with CRISPR Technology. This report also provides an estimation of the CRISPR Technologymarket size and corresponding revenue forecasts carried out in terms of US$. It also offers actionable insights based on the future trends in the CRISPR Technologymarket. Furthermore, new and emerging players in the global CRISPR Technologymarket can make use of the information presented in the study for effective business decisions, which will provide momentum to their businesses as well as the global CRISPR Technologymarket.
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The study is relevant for manufacturers, suppliers, distributors, and investors in the CRISPR Technologymarket. All stakeholders in the CRISPR Technologymarket, as well as industry experts, researchers, journalists, and business researchers can influence the information and data represented in the report.
CRISPR Technology Market 2020-2026: Segmentation
The CRISPR Technology market report covers major market players like 1. Thermo Fisher Scientific 2. Merck KGaA 3. GenScript 4. Integrated DNA Technologies (IDT) 5. Horizon Discovery Group 6. Agilent Technologies 7. CellectaInc. 8. GeneCopoeiaInc. 9. New England Biolabs 10. Origene TechnologiesInc. 11. Synthego Corporation 12. ToolgenInc.
CRISPR Technology Market is segmented as below:
By Product Type: By Product & Service: 1. CRISPR Products 1.1. CRISPR Enzymes1.2. CRISPR Libraries 1.3. CRISPR Kits 1.4. Other CRISPR Products 2. CRISPR Services 2.1. gRNA Design & Vector Construction2.2. Cell Line Engineering 2.3. Screening Services 2.4. Other CRISPR Services
Breakup by Application:Application: 1. Biomedical 2. Agricultural 3. Industrial 4. Biological Research End-use: 1. Pharmaceutical and Biopharmaceutical Companies 2. Biotechnology Companies 3. Academic & Research Institutes 4. Contract Research Organizations
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Impact of COVID-19:CRISPR TechnologyMarket report analyses the impact of Coronavirus (COVID-19) on the CRISPR Technologyindustry.Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the CRISPR Technologymarket in 2020.
The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; emergency declared in many countries; massive slowing of the supply chain; stock market unpredictability; falling business assurance, growing panic among the population, and uncertainty about future.
COVID-19 can affect the global economy in 3 main ways: by directly affecting production and demand, by creating supply chain and market disturbance, and by its financial impact on firms and financial markets.
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Global CRISPR Technology Market Report Answers Below Queries:
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Key Questions Answered in this Report:
What is the market size of the CRISPR Technology industry?This report covers the historical market size of the industry (2013-2019), and forecasts for 2020 and the next 5 years. Market size includes the total revenues of companies.
What is the outlook for the CRISPR Technology industry?This report has over a dozen market forecasts (2020 and the next 5 years) on the industry, including total sales, a number of companies, attractive investment opportunities, operating expenses, and others.
What industry analysis/data exists for the CRISPR Technology industry?This report covers key segments and sub-segments, key drivers, restraints, opportunities and challenges in the market and how they are expected to impact the CRISPR Technology industry. Take a look at the table of contents below to see the scope of analysis and data on the industry.
How many companies are in the CRISPR Technology industry?This report analyzes the historical and forecasted number of companies, locations in the industry, and breaks them down by company size over time. The report also provides company rank against its competitors with respect to revenue, profit comparison, operational efficiency, cost competitiveness, and market capitalization.
What are the financial metrics for the industry?This report covers many financial metrics for the industry including profitability, Market value- chain and key trends impacting every node with reference to companys growth, revenue, return on sales, etc.
What are the most important benchmarks for the CRISPR Technology industry?Some of the most important benchmarks for the industry include sales growth, productivity (revenue), operating expense breakdown, the span of control, organizational make-up. All of which youll find in this market report.
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Latest News 2020: CRISPR Technology Market by Coronavirus-COVID19 Impact Analysis With Top Manufacturers Analysis | Top Players: Thermo Fisher...
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Global CRISPR And CRISPR-Associated (Cas) Genes Market Views: Taking A Nimble Approach To Explores Huge Growth In Near Future With Eminent Players -…
Scope of the Report:
CRISPR And CRISPR-Associated (Cas) Genes industry is relatively concentrated, manufacturers are mostly in the Europe and North America. Among them, North America region accounted for more than 45.70% of the total market of global CRISPR And CRISPR-Associated (Cas) Genes.Although this market has great potential for future development, we do not recommend entering the market for investors who do not have strong capital or do not have key technology.
The worldwide market for CRISPR And CRISPR-Associated (Cas) Genes is expected to grow at a CAGR of roughly 39.8% over the next five years, will reach 2640 million US$ in 2024, from 350 million US$ in 2019, according to a new Globalmarketers.biz Research study.
This report focuses on the CRISPR And CRISPR-Associated (Cas) Genes in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the market based on manufacturers, regions, type and application.
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Global CRISPR And CRISPR-Associated (Cas) Genes report portrays the fundamental details of the dominant market players elaborating their business profiles, CRISPR And CRISPR-Associated (Cas) Genes market revenue, sales volume, press releases, technical developments taking place in this industry.
Report is segmented into different parts as below:
Global CRISPR And CRISPR-Associated (Cas) Genes Market Details Based On Key Players:
Caribou BiosciencesAddgeneCRISPR THERAPEUTICSMerck KGaAMirus Bio LLCEditas MedicineTakara Bio USAThermo Fisher ScientificHorizon Discovery GroupIntellia TherapeuticsGE Healthcare Dharmacon
Global CRISPR And CRISPR-Associated (Cas) Genes Market Details Based on Product Category:
Genome EditingGenetic engineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem CellsGenetically Modified Organisms/CropsCell Line Engineering
Global CRISPR And CRISPR-Associated (Cas) Genes Market Details Based On Key Product Applications:
Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes
Global CRISPR And CRISPR-Associated (Cas) Genes Market Details Based On Regions
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The first part of the report portrays the information related to the basic CRISPR And CRISPR-Associated (Cas) Genes introduction, key market players, their company profiles, sales ratio, demand and supply volume, CRISPR And CRISPR-Associated (Cas) Genes market gains during 2018 and 2019. The second part of the CRISPR And CRISPR-Associated (Cas) Genes report extracts more details stating the sales revenue of each CRISPR And CRISPR-Associated (Cas) Genes industry player, the business strategies followed by them. The third part of the report displays the competitive scenario of all the CRISPR And CRISPR-Associated (Cas) Genes market players on basis of the revenue gains.
The fourth part of the report enlists the CRISPR And CRISPR-Associated (Cas) Genes details based on key producing regions and CRISPR And CRISPR-Associated (Cas) Genes market gains during the period from 2015 to 2019. Fifth, sixth, seventh, eighth and ninth part of the CRISPR And CRISPR-Associated (Cas) Genes report enlists the major countries within the regions and the CRISPR And CRISPR-Associated (Cas) Genes revenue generated during the period from 2012 to 2017. Tenth and eleventh part of the CRISPR And CRISPR-Associated (Cas) Genes report mentions the variety of CRISPR And CRISPR-Associated (Cas) Genes product applications, CRISPR And CRISPR-Associated (Cas) Genes statistics during 2015 to 2019.
Part number twelve, thirteen, fourteen and fifteen provides information regarding the futuristic CRISPR And CRISPR-Associated (Cas) Genes market trends expected during the forecast period from 2020 to 2024, CRISPR And CRISPR-Associated (Cas) Genes marketing strategies, CRISPR And CRISPR-Associated (Cas) Genes market vendors, facts and figures of the CRISPR And CRISPR-Associated (Cas) Genes market and vital CRISPR And CRISPR-Associated (Cas) Genes business conclusion along with data collection sources and appendix.
What CRISPR And CRISPR-Associated (Cas) Genes Market Report Contributes?
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Global CRISPR And CRISPR-Associated (Cas) Genes Market Views: Taking A Nimble Approach To Explores Huge Growth In Near Future With Eminent Players -...
Recommendation and review posted by Bethany Smith
3D Culture Models with CRISPR Screens Reveal Hyperactive NRF2 as a Prerequisite for Spheroid Formation via Regulation of Proliferation and Ferroptosis…
This article was originally published here
Mol Cell. 2020 Oct 26:S1097-2765(20)30693-6. doi: 10.1016/j.molcel.2020.10.010. Online ahead of print.
ABSTRACT
Cancer-associated mutations that stabilize NRF2, an oxidant defense transcription factor, are predicted to promote tumor development. Here, utilizing 3D cancer spheroid models coupled with CRISPR-Cas9 screens, we investigate the molecular pathogenesis mediated by NRF2 hyperactivation. NRF2 hyperactivation was necessary for proliferation and survival in lung tumor spheroids. Antioxidant treatment rescued survival but not proliferation, suggesting the presence of distinct mechanisms. CRISPR screens revealed that spheroids are differentially dependent on the mammalian target of rapamycin (mTOR) for proliferation and the lipid peroxidase GPX4 for protection from ferroptosis of inner, matrix-deprived cells. Ferroptosis inhibitors blocked death from NRF2 downregulation, demonstrating a critical role of NRF2 in protecting matrix-deprived cells from ferroptosis. Interestingly, proteomics analyses show global enrichment of selenoproteins, including GPX4, by NRF2 downregulation, and targeting NRF2 and GPX4 killed spheroids overall. These results illustrate the value of spheroid culture in revealing environmental or spatial differential dependencies on NRF2 and reveal exploitable vulnerabilities of NRF2-hyperactivated tumors.
PMID:33128871 | DOI:10.1016/j.molcel.2020.10.010
Recommendation and review posted by Bethany Smith
The Impact of the Coronavirus on the Global CRISPR and Cas Genes Market Fact.MR Study 2020 2026 – The Cloud Tribune
The study presents an in-depth analysis of the important growth avenues and existing growth dynamics in the estimation year of 2020, and key prospects over the analysis period of 2020 2026.The study on the global CRISPR and Cas Genes Market offers insights and analysis into the potential and current opportunities amongst various end-users. It also provides a detailed picture of the trends of the changing structure in the industry and the difficulties faced by various industry participants. The report elaborates on the challenges of utmost concern so as to prepare the participants and stakeholders and place them in a better position to face the challenges.
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Experienced analysts at Fact.MR has put together all the available strategies, methods, and resources pertaining to the CRISPR and Cas Genes market so as to come up with a crisp view. The report also makes a vivid explanation of the existing dynamics of growth in the market over the assessment period, 2020 2026. Global businesses are suffering from uncertainties and turmoil as the coronavirus Covid-19 spreads at an unprecedented rate across the world. The super spread of Covid-19, caused by the Sars-Cov-2 virus, comes with profound implications for the global CRISPR and Cas Genes market and is highly consequential. As such, business leaders, owners, and strategists across the globe are making great efforts to find out how Covid-19 will change the industrial forecasts and estimates. The study also discusses in detail the impact that it will leave on the global economy in the few months to come.
The global CRISPR and Cas Genes market report offers an analysis of the current opportunities in various regions and assesses their shares of revenue. Key regions covered in the report include the following:
With an offering of valuable insight into the profile of each of the major vendors in the market and the technological innovations in the said industry that could emerge as the cornerstone of their futuristic strategies and moves, the report seeks to facilitate better decision making by the companies. Some of the leading players comprise:
In terms of type of product, the global CRISPR and Cas Genes market can be segmented into:
In addition to understanding and discussing the demand patterns of several end users, this report by Fact.MR on the global CRISPR and Cas Genes market also sums up the trends that are expected to attract investments by other various ancillary industries.
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The study offers a clear and accurate analysis of the consumption and demand patterns of several services and products found in the global CRISPR and Cas Genes market. In addition to that, this very assessment by the experts of Fact.MR stresses the potential opportunities, market figures, and the effect of potential opportunities on the market figure of the future.
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The Impact of the Coronavirus on the Global CRISPR and Cas Genes Market Fact.MR Study 2020 2026 - The Cloud Tribune
Recommendation and review posted by Bethany Smith
California Proposition 14 is about pikuach nefesh heres why – Forward
Only two decades ago, stem cell therapy was highly regulated in the United States and other countries but it was well underway in Hadassah Hospitals labs in Jerusalem. Never would we have imagined that the US expansion of one of the key clinical trials conducted in our labs in Israel would be later funded by Californias Stem Cell Institute.
In 2004, California had the foresight to advance this critical area of research, and Hadassah advocates played a major role in the passing of an unprecedented statewide ballot initiative that authorized state funding for stem cell research. This marked the passage of Proposition 71 in 2004.
Fast forward to today. Hadassahs commitment to stem cell research led the Californians for Stem Cell, Research, Treatments and Cures Initiative effort in 2020 to reach out for help with their grassroots effort to qualify the latest stem cell funding initiative for the November ballot. It qualified with your help, and I hope that in a few short weeks well be celebrating the passage of Proposition 14, which will provide $5.5 billion to help accelerate development of treatments and cures for life-threatening diseases and conditions.
The power of stem cells is mind-blowing: We are able to use these cells to replace damaged or diseased tissue, and in this way, treatments or cures for diseases like age-related macular degeneration, ALS, MS, Parkinsons, Alzheimers and diabetes could be a reality in the foreseeable future.
I come to this subject from a place of personal sorrow.
I watched my father-in-law, Irv, suffer for 12 and a half years with ALS, a man I loved as if he were my own father. He fought and fought, he made every minute of his battle meaningful, soaking as much life as he could, until he couldnt. Irv is the reason why I became involved in Hadassah because of their cutting-edge medical research and he is the reason that Im writing to you now.
Today, Hadassah is doing incredible things in stem cell research, and the stunning results of their clinical trials have riveted the worldwide medical community.
Of course, the research most personal to me is the ALS research. Hadassah researchers conducted the worlds first clinical trial using the patients own bone marrow stem cells to treat ALS.Pikuach nefesh, the preservation of life, is the most important obligation in Judaism, and the one that drives Hadassah. With the potential of stem research, we have the ability to save millions of lives throughout the world.
And, with the promise of stem cells, we can accelerate the development of treatments and cures for life-threatening diseases and conditions that affect someone in nearly half of all California families.
I believe that stem cell research is going to allow our children to look at the major diseases that plague our modern world the way we now view polio.That potential will not reveal itself on its own, nor did it with polio. It took decades of research and funding for a polio vaccine to be fully developed, tested and made available widely.
There are no medical miracles. Medical advancement happens because of research. Research takes will, knowledge, chutzpah and, of course, money. The path to get a therapy approved by the Food and Drug Administration can take 12 to 15 years, requires thousands of patients for clinical trials and costs billions of dollars. from life-saving vaccines, to pioneering cancer treatments, to the sequencing of the human genome.w When research stalls for lack of funding, opportunities are missed. Promising avenues go unexplored.
The passage of Proposition 14 would help to overcome those hurdles and create a streamlined process that delivers much-needed treatments to patients who have few options. How amazing would it be to be part of making medical history.I am so proud to be a member of Hadassah, which is not only leading the way in stem cell research but also doing its research in service to humanity. Together, we can make medical discoveries happen and continue to set the pace for the worldwide medical community.
I wish that my father-in-law was here to give you his final thumbs up.
Stacey Dorenfeld is the National State Advocacy Co-Chair and the Hadassah Southern California Advocacy Chair.
The views and opinions expressed in this article are the authors own and do not necessarily reflect those of the Forward.
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California Proposition 14 is about pikuach nefesh heres why - Forward
Recommendation and review posted by Bethany Smith
Cynata looking to revolutionise stem cell therapy – The West Australian
Ongoing studies of Cynata Therapeutics Cymerus stem cell products are beginning to reveal a wide range of commercial possibilities for the ASX-listed companys cutting edge biotechnology that it is looking to apply to a multitude of ailments from the treatment of osteoarthritis and heart attacks through to COVID-19.
In its most advanced trials to date, Cynata will soon embark on a Phase 3 trial of its CYP-004 product, the companys mesenchymal stem cell or MSC product developed to treat osteoarthritis. The 448 person trial is being sponsored by The University of Sydney and will be funded by a project grant from the Australian Government National Health and Medical Research Council.
The company is also progressing on multiple other fronts developing a range of Cymerus MSC therapeutics with the CYP-001 product being another lead candidate. CYP-001 is being developed to treat acute graft-versus-host disease, or GVHD an affliction suffered by bone marrow transplant recipients. GVHD can develop from donated bone marrow that does not take well to a recipients body which triggers an immune response, attacking the host.
Presently, GVHD is treated with steroid therapy however sufferers tend to have a very low survival rate, with less than 20 per cent of patients living for more than two years and few alternate treatment pathways are available.
This looks set to change following Phase 1 trial of Cynatas CYP-001 product on a cohort of patients which saw the survival rate of sufferers of GVHD triple to 60 per cent over a two-year period. The company is now moving CYP-001 into Phase 2 testing and towards commercialisation with partner and shareholder, Fujifilm Corporation.
The matchup with the Japanese-based multi-national is already paying dividends with Cynata receiving an upfront US$3 million payment with further staged payments and royalties to follow in a licensing deal potentially worth more than US$50 million in the longer term.
Stem cells are the building blocks of the human body - essentially the cells from which all other cells are derived and under the right conditions, they can divide to produce more cells sometimes known as Daughter cells. These Daughter cells can become new stem cells or more specialised cells such as blood, bone or even the cells that make up brain or heart tissue.
When appropriately manipulated, stem cells have the potential to treat a range of diseases and aid in the healing and recovery of patients suffering both disease and trauma.
There are a limited number of sources of stem cells - embryonic stem cells, perinatal stem cells and adult stem cells.
Embryonic stem cells are thought to be the most useful and versatile but only harvestable in very small quantities. Perinatal stem cells found in amniotic fluid and umbilical cord blood are also only harvestable in limited quantities although their potential is yet to be fully understood.
Adult stem cells, found in bone marrow or fat, were previously thought to be only useful in producing a limited range of specialised cells with multiple donors required to generate practical amounts of therapeutical medicines.
However, ongoing research shows that by utilising a form of genetic reprogramming, mature cells can be re-programmed to behave like embryonic stem cells. These manipulated cells are called induced pluripotent stem cells, or iPSCs which is where Cynatas Cymerus technology comes into the picture.
Cynatas proprietary Cymerus technology uses iPSCs and a precursor cell called a mesenchymoangioblast to manufacture MSC therapies at a commercial scale without the need for multiple donors. This is where the Cymerus platform diverges from similar therapies, doing away with the need for multiple donors and overcoming a bottleneck in the generation of its product.
Other Cynata MSC products in development include a therapy to assist in the treatment and recovery of heart attacks, which is also showing promise according to the company. Another Cynata product undergoing pre-clinical trials with potential application in the treatment of lung disease is idiopathic pulmonary fibrosis, or IPF. Cynatas research in lung diseases has an unexpected spin-off in that its MSCs may assist in a patients recovery of COVID-19 according to the company. This application is being pursued in a clinical trial in COVID-19 patients presently being conducted in NSW.
These latest results with Cymerus MSCs add to the large body of evidence on the potency of these cells and their potential utility in treating a wide range of devastating diseases. IPF represents an enormous unmet medical need, as existing treatment options have only modest effects on disease progression and survival rates.
Cynatas is now modelling potential MSC therapies to treat various other afflictions too including critical limb ischemia, asthma, sepsis, cytokine release syndrome and diabetic wounds.
In the world of biotechnology, you really only have to produce one winner to attract a longing stare from the big biotechs who can swallow you whole with their massive cheque books with a range of targets and opportunities in its armoury that look to be developing well, dont be surprised if Cynata eventually disappears under the giant footprint of one of the big biotechs.
Is your ASX listed company doing something interesting? Contact: matt.birney@wanews.com.au
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Cynata looking to revolutionise stem cell therapy - The West Australian
Recommendation and review posted by Bethany Smith
BioLineRx Announces Positive Results from Interim Analysis of GENESIS Phase 3 Trial of Motixafortide (BL-8040) in Stem Cell Mobilization – PRNewswire
TEL AVIV, Israel, Oct. 30, 2020 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, today announced positive results from a planned interim analysis of the ongoing GENESIS Phase 3 trial of motixafortide for stem cell mobilization (SCM) in multiple myeloma patients.
At a meeting of the study's independent Data Monitoring Committee (DMC), a planned interim analysis of the study's primary endpoint was conducted independently by the DMC. Based on the statistically significant evidence favoring treatment with motixafortide, the DMC issued a recommendation to the Company that patient enrollment may be ceased immediately, without the need to recruit all 177 patients originally planned for the study.
In accordance with the DMC's recommendation, study enrollment is now complete at 122 patients. Full results for the study, including secondary and exploratory efficacy endpoints, as well as extended safety data, will be announced after the last patient enrolled reaches 100 days of follow-up post-transplantation, which is expected to occur in the first half of 2021.
"The compelling results of this planned interim analysis are a very significant milestone for our Company, as our SCM program is the Company's most efficient path to registration for motixafortide," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Stem cell mobilization represents a significant unmet medical need in multiple myeloma, as between 50% and 70% of patients are poor mobilizers. We eagerly await the final results of the study, expected in the first half of next year, which we hope will support our goal of changing the treatment paradigm in autologous stem-cell mobilization, thus positioning motixafortide in combination with G-CSF as the new standard of care in this indication."
The GENESIS trial was initiated in December 2017. GENESIS is a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of HSCs for autologous transplantation in multiple myeloma patients. The primary objective of the study is to demonstrate that only one dose of motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize 6x106 CD34+ cells in up to two apheresis sessions. Secondary objectives include time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters.
About BioLineRx
BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.
The Company's lead program, motixafortide (BL-8040), is a cancer therapy platform currently being evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with MSD. Motixafortide is also being evaluated in a Phase 2b study in consolidation AML and a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation.
BioLineRx is developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.
For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook,Twitter, and LinkedIn.
Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the coronavirus outbreak; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 12, 2020. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.
Contact:
Tim McCarthy LifeSci Advisors, LLC +1-212-915-2564 [emailprotected]
or
Moran Meir LifeSci Advisors, LLC +972-54-476-4945 [emailprotected]
SOURCE BioLineRx Ltd.
Recommendation and review posted by Bethany Smith