A confirmed diagnosis may take time.

Diagnosis represents the first step on this rare disease journey. Sometimes doctors will notice something off about the child during a newborn screening, and a genetic test will identify a known mutation in the DNA. But not all conditions are so quickly detected, and it can take several years for parents to get a confirmed diagnosis.

About half of all children never get that far, according to Marshall Summar, M.D., the director of the Rare Disease Institute at Childrens National Hospital in Washington, D.C. When you sequence someones DNA, you are going to find a lot of changes, Dr. Summar said. Figuring out which change might be the one that is causing it is a tremendous challenge.

Genetic counselors warn parents beforehand that they may not get a definitive answer as to what condition their child could have. They may have to check back each year. Dr. Summar estimates that between five and 10 new rare diseases are described in the scientific literature every week, making it challenging for the medical field to keep up.

Meanwhile, the realization that a child may have a debilitating, lifelong condition weighs heavily. Some parents, particularly mothers, blame themselves, said Lemuel Pelentsov, Ph.D., a nurse who studies the needs of rare disease families at the University of South Australia, in Adelaide. In a 2016 study by Dr. Pelentsov and his colleagues, about 40 percent of the 300 rare parents surveyed reported being treated for depression and an equal number for anxiety. One of the things they do to combat that, he said, is get very invested in the childs disease.

When parents reach out to other parents, they are not simply looking for emotional support or advice. They are rebuilding a social life, one that will revolve around their childs disease. Many rare diseases have their own support groups. Global Genes is an umbrella group that supports 600 disease-specific foundations, as well as parents of children whose diseases are so rare they have no foundation.

We encourage folks to work together, said Kimberly Haugstad, the organizations executive director whose son has a rare form of hemophilia, a condition in which the blood doesnt clot normally. The parent is going to come from such different places in their own walk of life.

Each year, Global Genes hosts a Rare Boot Camp to mentor and teach parents how to set up a nonprofit, create patient registries and fund research. After attending the boot camp, the Van Wyks and other parents founded GACI Global, an organization that connects families affected by GACI, along with medical professionals.

Continued here:
The Wilderness of Rare Genetic Diseases and the Parents Navigating It - The New York Times

Recommendation and review posted by Bethany Smith

New Jersey, United States,- Market Research Intellect sheds light on the market scope, potential, and performance perspective of the Global Hemophilia Gene Therapy Market by carrying out an extensive market analysis. Pivotal market aspects like market trends, the shift in customer preferences, fluctuating consumption, cost volatility, the product range available in the market, growth rate, drivers and constraints, financial standing, and challenges existing in the market are comprehensively evaluated to deduce their impact on the growth of the market in the coming years. The report also gives an industry-wide competitive analysis, highlighting the different market segments, individual market share of leading players, and the contemporary market scenario and the most vital elements to study while assessing the global Hemophilia Gene Therapy market.

The research study includes the latest updates about the COVID-19 impact on the Hemophilia Gene Therapy sector. The outbreak has broadly influenced the global economic landscape. The report contains a complete breakdown of the current situation in the ever-evolving business sector and estimates the aftereffects of the outbreak on the overall economy.

Leading Hemophilia Gene Therapy manufacturers/companies operating at both regional and global levels:

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The Hemophilia Gene Therapy market report provides successfully marked contemplated policy changes, favorable circumstances, industry news, developments, and trends. This information can help readers fortify their market position. It packs various parts of information gathered from secondary sources, including press releases, web, magazines, and journals as numbers, tables, pie-charts, and graphs. The information is verified and validated through primary interviews and questionnaires. The data on growth and trends focuses on new technologies, market capacities, raw materials, CAPEX cycle, and the dynamic structure of the Hemophilia Gene Therapy market.

This study analyzes the growth of Hemophilia Gene Therapy based on the present, past and futuristic data and will render complete information about the Hemophilia Gene Therapy industry to the market-leading industry players that will guide the direction of the Hemophilia Gene Therapy market through the forecast period. All of these players are analyzed in detail so as to get details concerning their recent announcements and partnerships, product/services, and investment strategies, among others.

Sales Forecast:

The report contains historical revenue and volume that backing information about the market capacity, and it helps to evaluate conjecture numbers for key areas in the Hemophilia Gene Therapy market. Additionally, it includes a share of each segment of the Hemophilia Gene Therapy market, giving methodical information about types and applications of the market.

Reasons for Buying Hemophilia Gene Therapy Market Report

This report gives a forward-looking prospect of various factors driving or restraining market growth.

It renders an in-depth analysis for changing competitive dynamics.

It presents a detailed analysis of changing competition dynamics and puts you ahead of competitors.

It gives a six-year forecast evaluated on the basis of how the market is predicted to grow.

It assists in making informed business decisions by performing a pin-point analysis of market segments and by having complete insights of the Hemophilia Gene Therapy market.

This report helps the readers understand key product segments and their future.

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In the end, the Hemophilia Gene Therapy market is analyzed for revenue, sales, price, and gross margin. These points are examined for companies, types, applications, and regions.

To summarize, the global Hemophilia Gene Therapy market report studies the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and the trends observed in the market that can either propel or curtail the growth rate of the industry. The market factors impacting the global sector also include provincial trade policies, international trade disputes, entry barriers, and other regulatory restrictions.

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Hemophilia Gene Therapy Market Growth By Manufacturers, Type And Application, Forecast To 2026 - 3rd Watch News

Recommendation and review posted by Bethany Smith

LOS ANGELES, United States: QY Research has recently published a report, titled Global Cell and Gene Therapy Market Size, Status and Forecast 2020-2026. The research report gives the potential headway openings that prevails in the global market. The report is amalgamated depending on research procured from primary and secondary information. The global Cell and Gene Therapy market is relied upon to develop generously and succeed in volume and value during the predicted time period. Moreover, the report gives nitty gritty data on different manufacturers, region, and products which are important to totally understanding the market.

Key Companies/Manufacturers operating in the global Cell and Gene Therapy market include: 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.

Get PDF Sample Copy of the Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) :

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Segmental Analysis

Both developed and emerging regions are deeply studied by the authors of the report. The regional analysis section of the report offers a comprehensive analysis of the global Cell and Gene Therapy market on the basis of region. Each region is exhaustively researched about so that players can use the analysis to tap into unexplored markets and plan powerful strategies to gain a foothold in lucrative markets.

Global Cell and Gene Therapy Market Segment By Type:

Rare DiseasesOncologyHematologyCardiovascularOphthalmologyNeurologyOther Therapeutic Classes Cell and Gene Therapy

Global Cell and Gene Therapy Market Segment By Application:

Pharmaceutical and Biotechnology CompaniesResearch and Academic InstitutionsContract Research Organizations (CROs)HospitalOthers Based on

Competitive Landscape

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the global Cell and Gene Therapy market.

Key companies operating in the global Cell and Gene Therapy market include 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.

Key questions answered in the report:

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TOC

1 Report Overview1.1 Study Scope1.2 Market Analysis by Type1.2.1 Global Cell and Gene Therapy Market Size Growth Rate by Type: 2020 VS 20261.2.2 Rare Diseases1.2.3 Oncology1.2.4 Hematology1.2.5 Cardiovascular1.2.6 Ophthalmology1.2.7 Neurology1.2.8 Other Therapeutic Classes1.3 Market by Application1.3.1 Global Cell and Gene Therapy Market Share by Application: 2020 VS 20261.3.2 Pharmaceutical and Biotechnology Companies1.3.3 Research and Academic Institutions1.3.4 Contract Research Organizations (CROs)1.3.5 Hospital1.3.6 Others1.4 Study Objectives1.5 Years Considered 2 Global Growth Trends2.1 Global Cell and Gene Therapy Market Perspective (2015-2026)2.2 Global Cell and Gene Therapy Growth Trends by Regions2.2.1 Cell and Gene Therapy Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Cell and Gene Therapy Historic Market Share by Regions (2015-2020)2.2.3 Cell and Gene Therapy Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Market Restraints 3 Competition Landscape by Key Players3.1 Global Top Cell and Gene Therapy Players by Market Size3.1.1 Global Top Cell and Gene Therapy Players by Revenue (2015-2020)3.1.2 Global Cell and Gene Therapy Revenue Market Share by Players (2015-2020)3.2 Global Cell and Gene Therapy Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.3 Players Covered: Ranking by Cell and Gene Therapy Revenue3.4 Global Cell and Gene Therapy Market Concentration Ratio3.4.1 Global Cell and Gene Therapy Market Concentration Ratio (CR5 and HHI)3.4.2 Global Top 10 and Top 5 Companies by Cell and Gene Therapy Revenue in 20193.5 Key Players Cell and Gene Therapy Area Served3.6 Key Players Cell and Gene Therapy Product Solution and Service3.7 Date of Enter into Cell and Gene Therapy Market3.8 Mergers & Acquisitions, Expansion Plans 4 Cell and Gene Therapy Breakdown Data by Type (2015-2026)4.1 Global Cell and Gene Therapy Historic Market Size by Type (2015-2020)4.2 Global Cell and Gene Therapy Forecasted Market Size by Type (2021-2026) 5 Cell and Gene Therapy Breakdown Data by Application (2015-2026)5.1 Global Cell and Gene Therapy Historic Market Size by Application (2015-2020)5.2 Global Cell and Gene Therapy Forecasted Market Size by Application (2021-2026) 6 North America6.1 North America Cell and Gene Therapy Market Size (2015-2026)6.2 North America Cell and Gene Therapy Market Size by Type (2015-2020)6.3 North America Cell and Gene Therapy Market Size by Application (2015-2020)6.4 North America Cell and Gene Therapy Market Size by Country (2015-2020)6.4.1 United States6.4.2 Canada 7 Europe7.1 Europe Cell and Gene Therapy Market Size (2015-2026)7.2 Europe Cell and Gene Therapy Market Size by Type (2015-2020)7.3 Europe Cell and Gene Therapy Market Size by Application (2015-2020)7.4 Europe Cell and Gene Therapy Market Size by Country (2015-2020)7.4.1 Germany7.4.2 France7.4.3 U.K.7.4.4 Italy7.4.5 Russia7.4.6 Nordic7.4.7 Rest of Europe 8 China8.1 China Cell and Gene Therapy Market Size (2015-2026)8.2 China Cell and Gene Therapy Market Size by Type (2015-2020)8.3 China Cell and Gene Therapy Market Size by Application (2015-2020)8.4 China Cell and Gene Therapy Market Size by Region (2015-2020)8.4.1 China8.4.2 Japan8.4.3 South Korea8.4.4 Southeast Asia8.4.5 India8.4.6 Australia8.4.7 Rest of Asia-Pacific 9 Japan9.1 Japan Cell and Gene Therapy Market Size (2015-2026)9.2 Japan Cell and Gene Therapy Market Size by Type (2015-2020)9.3 Japan Cell and Gene Therapy Market Size by Application (2015-2020)9.4 Japan Cell and Gene Therapy Market Size by Country (2015-2020)9.4.1 Mexico9.4.2 Brazil 10 Southeast Asia10.1 Southeast Asia Cell and Gene Therapy Market Size (2015-2026)10.2 Southeast Asia Cell and Gene Therapy Market Size by Type (2015-2020)10.3 Southeast Asia Cell and Gene Therapy Market Size by Application (2015-2020)10.4 Southeast Asia Cell and Gene Therapy Market Size by Country (2015-2020)10.4.1 Turkey10.4.2 Saudi Arabia10.4.3 UAE10.4.4 Rest of Middle East & Africa 11 Key Players Profiles11.1 Amgen Inc.11.1.1 Amgen Inc. Company Details11.1.2 Amgen Inc. Business Overview11.1.3 Amgen Inc. Cell and Gene Therapy Introduction11.1.4 Amgen Inc. Revenue in Cell and Gene Therapy Business (2015-2020))11.1.5 Amgen Inc. Recent Development11.2 bluebird bio, Inc.11.2.1 bluebird bio, Inc. Company Details11.2.2 bluebird bio, Inc. Business Overview11.2.3 bluebird bio, Inc. Cell and Gene Therapy Introduction11.2.4 bluebird bio, Inc. Revenue in Cell and Gene Therapy Business (2015-2020)11.2.5 bluebird bio, Inc. Recent Development11.3 Dendreon Pharmaceuticals LLC.11.3.1 Dendreon Pharmaceuticals LLC. Company Details11.3.2 Dendreon Pharmaceuticals LLC. Business Overview11.3.3 Dendreon Pharmaceuticals LLC. Cell and Gene Therapy Introduction11.3.4 Dendreon Pharmaceuticals LLC. Revenue in Cell and Gene Therapy Business (2015-2020)11.3.5 Dendreon Pharmaceuticals LLC. Recent Development11.4 Fibrocell Science, Inc.11.4.1 Fibrocell Science, Inc. Company Details11.4.2 Fibrocell Science, Inc. Business Overview11.4.3 Fibrocell Science, Inc. Cell and Gene Therapy Introduction11.4.4 Fibrocell Science, Inc. Revenue in Cell and Gene Therapy Business (2015-2020)11.4.5 Fibrocell Science, Inc. Recent Development11.5 Human Stem Cells Institute11.5.1 Human Stem Cells Institute Company Details11.5.2 Human Stem Cells Institute Business Overview11.5.3 Human Stem Cells Institute Cell and Gene Therapy Introduction11.5.4 Human Stem Cells Institute Revenue in Cell and Gene Therapy Business (2015-2020)11.5.5 Human Stem Cells Institute Recent Development11.6 Kite Pharma, Inc.11.6.1 Kite Pharma, Inc. Company Details11.6.2 Kite Pharma, Inc. Business Overview11.6.3 Kite Pharma, Inc. Cell and Gene Therapy Introduction11.6.4 Kite Pharma, Inc. Revenue in Cell and Gene Therapy Business (2015-2020)11.6.5 Kite Pharma, Inc. Recent Development11.7 Kolon TissueGene, Inc.11.7.1 Kolon TissueGene, Inc. Company Details11.7.2 Kolon TissueGene, Inc. Business Overview11.7.3 Kolon TissueGene, Inc. Cell and Gene Therapy Introduction11.7.4 Kolon TissueGene, Inc. Revenue in Cell and Gene Therapy Business (2015-2020)11.7.5 Kolon TissueGene, Inc. Recent Development11.8 Novartis AG11.8.1 Novartis AG Company Details11.8.2 Novartis AG Business Overview11.8.3 Novartis AG Cell and Gene Therapy Introduction11.8.4 Novartis AG Revenue in Cell and Gene Therapy Business (2015-2020)11.8.5 Novartis AG Recent Development11.9 Orchard Therapeutics plc.11.9.1 Orchard Therapeutics plc. Company Details11.9.2 Orchard Therapeutics plc. Business Overview11.9.3 Orchard Therapeutics plc. Cell and Gene Therapy Introduction11.9.4 Orchard Therapeutics plc. Revenue in Cell and Gene Therapy Business (2015-2020)11.9.5 Orchard Therapeutics plc. Recent Development11.10 Organogenesis Holdings Inc.11.10.1 Organogenesis Holdings Inc. Company Details11.10.2 Organogenesis Holdings Inc. Business Overview11.10.3 Organogenesis Holdings Inc. Cell and Gene Therapy Introduction11.10.4 Organogenesis Holdings Inc. Revenue in Cell and Gene Therapy Business (2015-2020)11.10.5 Organogenesis Holdings Inc. Recent Development11.11 Pfizer, Inc.10.11.1 Pfizer, Inc. Company Details10.11.2 Pfizer, Inc. Business Overview10.11.3 Pfizer, Inc. Cell and Gene Therapy Introduction10.11.4 Pfizer, Inc. Revenue in Cell and Gene Therapy Business (2015-2020)10.11.5 Pfizer, Inc. Recent Development11.12 RENOVA THERAPEUTICS10.12.1 RENOVA THERAPEUTICS Company Details10.12.2 RENOVA THERAPEUTICS Business Overview10.12.3 RENOVA THERAPEUTICS Cell and Gene Therapy Introduction10.12.4 RENOVA THERAPEUTICS Revenue in Cell and Gene Therapy Business (2015-2020)10.12.5 RENOVA THERAPEUTICS Recent Development11.13 Shanghai Sunway Biotech Co., Ltd.,10.13.1 Shanghai Sunway Biotech Co., Ltd., Company Details10.13.2 Shanghai Sunway Biotech Co., Ltd., Business Overview10.13.3 Shanghai Sunway Biotech Co., Ltd., Cell and Gene Therapy Introduction10.13.4 Shanghai Sunway Biotech Co., Ltd., Revenue in Cell and Gene Therapy Business (2015-2020)10.13.5 Shanghai Sunway Biotech Co., Ltd., Recent Development11.14 Sibiono GeneTech Co. Ltd.,10.14.1 Sibiono GeneTech Co. Ltd., Company Details10.14.2 Sibiono GeneTech Co. Ltd., Business Overview10.14.3 Sibiono GeneTech Co. Ltd., Cell and Gene Therapy Introduction10.14.4 Sibiono GeneTech Co. Ltd., Revenue in Cell and Gene Therapy Business (2015-2020)10.14.5 Sibiono GeneTech Co. Ltd., Recent Development11.15 Spark Therapeutics, Inc.10.15.1 Spark Therapeutics, Inc. Company Details10.15.2 Spark Therapeutics, Inc. Business Overview10.15.3 Spark Therapeutics, Inc. Cell and Gene Therapy Introduction10.15.4 Spark Therapeutics, Inc. Revenue in Cell and Gene Therapy Business (2015-2020)10.15.5 Spark Therapeutics, Inc. Recent Development11.16 Vericel Corporation10.16.1 Vericel Corporation Company Details10.16.2 Vericel Corporation Business Overview10.16.3 Vericel Corporation Cell and Gene Therapy Introduction10.16.4 Vericel Corporation Revenue in Cell and Gene Therapy Business (2015-2020)10.16.5 Vericel Corporation Recent Development11.17 ViroMed Co., Ltd.10.17.1 ViroMed Co., Ltd. Company Details10.17.2 ViroMed Co., Ltd. Business Overview10.17.3 ViroMed Co., Ltd. Cell and Gene Therapy Introduction10.17.4 ViroMed Co., Ltd. Revenue in Cell and Gene Therapy Business (2015-2020)10.17.5 ViroMed Co., Ltd. Recent Development 12 Analysts Viewpoints/Conclusions 13 Appendix13.1 Research Methodology13.1.1 Methodology/Research Approach13.1.2 Data Source13.2 Disclaimer13.3 Author Details

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Trending: Cell and Gene Therapy Market by Application, Type, Region 2020 | Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell...

Recommendation and review posted by Bethany Smith

Global Cell and Gene Therapy Market: Trends Estimates High Demand by 2027

The Cell and Gene Therapy Market 2020 report includes the market strategy, market orientation, expert opinion and knowledgeable information. The Cell and Gene Therapy Industry Report is an in-depth study analyzing the current state of the Cell and Gene Therapy Market. It provides a brief overview of the market focusing on definitions, classifications, product specifications, manufacturing processes, cost structures, market segmentation, end-use applications and industry chain analysis. The study on Cell and Gene Therapy Market provides analysis of market covering the industry trends, recent developments in the market and competitive landscape.

It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the global Cell and Gene Therapy market. All findings and data on the global Cell and Gene Therapy market provided in the report are calculated, gathered, and verified using advanced and reliable primary and secondary research sources. The regional analysis offered in the report will help you to identify key opportunities of the global Cell and Gene Therapy market available in different regions and countries.

The final report will add the analysis of the Impact of Covid-19 in this report Cell and Gene Therapy industry.

Some of The Companies Competing in The Cell and Gene Therapy Market are: 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.

Get a Sample Copy of the [emailprotected] https://www.reportsandmarkets.com/sample-request/covid-19-impact-on-global-cell-and-gene-therapy-market-report-history-and-forecast-2015-2026-breakdown-data-by-companies-key-regions-types-and-application?utm_source=primefeed&utm_medium=46

The report scrutinizes different business approaches and frameworks that pave the way for success in businesses. The report used Porters five techniques for analyzing the Cell and Gene Therapy Market; it also offers the examination of the global market. To make the report more potent and easy to understand, it consists of info graphics and diagrams. Furthermore, it has different policies and improvement plans which are presented in summary. It analyzes the technical barriers, other issues, and cost-effectiveness affecting the market.

Global Cell and Gene Therapy Market Research Report 2020 carries in-depth case studies on the various countries which are involved in the Cell and Gene Therapy market. The report is segmented according to usage wherever applicable and the report offers all this information for all major countries and associations. It offers an analysis of the technical barriers, other issues, and cost-effectiveness affecting the market. Important contents analyzed and discussed in the report include market size, operation situation, and current & future development trends of the market, market segments, business development, and consumption tendencies. Moreover, the report includes the list of major companies/competitors and their competition data that helps the user to determine their current position in the market and take corrective measures to maintain or increase their share holds.

What questions does the Cell and Gene Therapy market report answer pertaining to the regional reach of the industry?

The report claims to split the regional scope of the Cell and Gene Therapy market into North America, Europe, Asia-Pacific, South America & Middle East and Africa. Which among these regions has been touted to amass the largest market share over the anticipated duration

How do the sales figures look at present how does the sales scenario look for the future?

Considering the present scenario, how much revenue will each region attain by the end of the forecast period?

How much is the market share that each of these regions has accumulated presently

How much is the growth rate that each topography will depict over the predicted timeline

A short overview of the Cell and Gene Therapy market scope:

Global market remuneration

Overall projected growth rate

Industry trends

Competitive scope

Product range

Application landscape

Supplier analysis

Marketing channel trends Now and later

Sales channel evaluation

Market Competition Trend

Market Concentration Rate

Reasons to Read this Report

This report provides pin-point analysis for changing competitive dynamics

It provides a forward looking perspective on different factors driving or restraining market growth

It provides a six-year forecast assessed on the basis of how the market is predicted to grow

It helps in understanding the key product segments and their future

It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors

It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments

TABLE OF CONTENT:

Chapter 1:Cell and Gene Therapy Market Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3:Cell and Gene Therapy Market Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Cell and Gene Therapy Market Effect Factors Analysis

Chapter 12: GlobalCell and Gene Therapy Market Forecast to 2027

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Research On Global Cell and Gene Therapy Market (impact of COVID-19) with Top Players: Amgen Inc.,bluebird bio, Inc.,Dendreon Pharmaceuticals LLC. and...

Recommendation and review posted by Bethany Smith

Global Cancer Gene Therapy Market Report 2019-Market Size, Share, Price, Trend and Forecast report is based on comprehensive analysis conducted by experienced and professional experts. The report mentions, factors that are influencing growth such as drivers, restrains of the market. The report offers in-depth analysis of trends and opportunities in the Cancer Gene Therapy Market. The report offers figurative estimations and predicts future for upcoming years on the basis of the recent developments and historic data. For the gathering information and estimating revenue for all segments, researchers have used top-down and bottom-up approach. On the basis of data collected from primary and secondary research and trusted data sources the report offers future predictions of revenue and market share.

The Leading Market Players Covered in this Report are : Adaptimmune,GlaxoSmithKline,Bluebird bio,Merck,Celgene,Shanghai Sunway Biotech .

Get Free Sample PDF Of Cancer Gene Therapy Market @ https://www.researchmoz.us/enquiry.php?type=S&repid2271992

Impact of Covid-19 in Cancer Gene Therapy Market:The utility-owned segment is mainly being driven by increasing financial incentives and regulatory supports from the governments globally. The current utility-owned Cancer Gene Therapy are affected primarily by the COVID-19 pandemic. Most of the projects in China, the US, Germany, and South Korea are delayed, and the companies are facing short-term operational issues due to supply chain constraints and lack of site access due to the COVID-19 outbreak. Asia-Pacific is anticipated to get highly affected by the spread of the COVID-19 due to the effect of the pandemic in China, Japan, and India. China is the epic center of this lethal disease. China is a major country in terms of the chemical industry.

Key Businesses Segmentation of Cancer Gene Therapy MarketOn the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, sales volume, Cancer Gene Therapy market share and growth rate of Cancer Gene Therapy foreach application, including-

On the basis of product,this report displays the sales volume, revenue (Million USD), product price, Cancer Gene Therapy market share and growth rate ofeach type, primarily split into-

Cancer Gene Therapy Market Regional Analysis Includes: Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia) Europe(Turkey, Germany, Russia UK, Italy, France, etc.) North America(the United States, Mexico, and Canada.) South America(Brazil etc.) The Middle East and Africa(GCC Countries and Egypt.)

Key Highlights of the Table of Contents:Cancer Gene Therapy Market Study Coverage:It includes key market segments, key manufacturers covered, the scope of products offered in the years considered, global Cancer Gene Therapy market and study objectives. Additionally, it touches the segmentation study provided in the report on the basis of the type of product and applications.Cancer Gene Therapy Market Executive summary:This section emphasizes the key studies, market growth rate, competitive landscape, market drivers, trends, and issues in addition to the macroscopic indicators.Cancer Gene Therapy Market Production by Region:The report delivers data related to import and export, revenue, production, and key players of all regional markets studied are covered in this section.Cancer Gene Therapy Market Profile of Manufacturers:Analysis of each market player profiled is detailed in this section. This segment also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

Do You Have Any Query Or Specific Requirement? Ask to Our Industry [emailprotected]https://www.researchmoz.us/enquiry.php?type=E&repid2271992

Key Questions Answered: To gain insightful analyses of the Cancer Gene Therapy market and have a comprehensive understanding of the global market and its commercial landscape. Assess the production processes, major issues, and solutions to mitigate the development risk. To understand the most affecting driving and restraining forces in the market and its impact on the global Cancer Gene Therapy market. Learn about the Cancer Gene Therapy market strategies that are being adopted by leading respective organizations. To understand the future outlook and prospects for the market. Besides the standard structure reports, we also provide custom research according to specific requirements.

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]Media Release @ https://www.researchmoz.us/pressreleaseFollow me on Blogger: https://trendingrelease.blogspot.com/

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Cancer Gene Therapy Market Present Scenario, the Growth Prospects with Forecast 2024 - 3rd Watch News

Recommendation and review posted by Bethany Smith

Global Cell and Gene Therapy Market Report, History and Forecast 2015-2026, Breakdown Data by Companies, Key Regions, Types and Application

In 2019, the global Cell and Gene Therapy Market size was US$ xx million and it is expected to reach US$ xx million by the end of 2026, with a CAGR of xx% during 2021-2026.

Cell and Gene Therapy Market is segmented by Type, and by Application. Players, stakeholders, and other participants in the global Cell and Gene Therapy Market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast by Type and by Application in terms of revenue and forecast for the period 2015-2026.The Report scope furnishes with vital statistics about the current market status and manufacturers. It analyzes the in-depth business by considering different aspects, direction for companies, and strategy in the industry.

After analyzing the report and all the aspects of the new investment projects, it is assessed the overall research and closure offered. The analysis of each segment in-detailed with various point views; that include the availability of data, facts, and figures, past performance, trends, and way of approaching in the market. The Cell and Gene Therapy Market report also covers the in-depth analysis of the market dynamics, price, and forecast parameters which also include the demand, profit margin, supply and cost for the industry.

The report additionally provides a pest analysis of all five along with the SWOT analysis for all companies profiled in the report. The report also consists of various company profiles and their key players; it also includes the competitive scenario, opportunities, and market of geographic regions. The regional outlook on the Cell and Gene Therapy Market covers areas such as Europe, Asia, China, India, North America, and the rest of the globe.

In-depth analysis of expansion and growth strategies obtained by Key players and their effect on competition market growth. The research report also provides precise information on your competitors and their planning. All of the above will help you to make a clear plan for top-line growth.

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Top key players @ 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., and

The main goal for the dissemination of this information is to give a descriptive analysis of how the trends could potentially affect the upcoming future of Cell and Gene Therapy Market during the forecast period. This markets competitive manufactures and the upcoming manufactures are studied with their detailed research. Revenue, production, price, market share of these players is mentioned with precise information.

Global Cell and Gene Therapy Market: Regional Segment Analysis

This report provides pinpoint analysis for changing competitive dynamics. It offers a forward-looking perspective on different factors driving or limiting market growth. It provides a five-year forecast assessed on the basis of how they Cell and Gene Therapy Market is predicted to grow. It helps in understanding the key product segments and their future and helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments.

Key questions answered in the report include:

What will the market size and the growth rate be in 2026?

What are the key factors driving the Global Cell and Gene Therapy Market?

What are the key market trends impacting the growth of the Global Cell and Gene Therapy Market?

What are the challenges to market growth?

Who are the key vendors in the Global Cell and Gene Therapy Market?

What are the market opportunities and threats faced by the vendors in the Global Cell and Gene Therapy Market?

Trending factors influencing the market shares of the Americas, APAC, Europe, and MEA.

The report includes six parts, dealing with:

1.) Basic information;

2.) The Asia Cell and Gene Therapy Market;

3.) The North American Cell and Gene Therapy Market;

4.) The European Cell and Gene Therapy Market;

5.) Market entry and investment feasibility;

6.) The report conclusion.

All the research report is made by using two techniques that are Primary and secondary research. There are various dynamic features of the business, like client need and feedback from the customers. Before (company name) curate any report, it has studied in-depth from all dynamic aspects such as industrial structure, application, classification, and definition.

The report focuses on some very essential points and gives a piece of full information about Revenue, production, price, and market share.

Cell and Gene Therapy Market report will enlist all sections and research for each and every point without showing any indeterminate of the company.

Reasons for Buying this Report

This report provides pin-point analysis for changing competitive dynamics

It provides a forward looking perspective on different factors driving or restraining market growth

It provides a six-year forecast assessed on the basis of how the market is predicted to grow

It helps in understanding the key product segments and their future

It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors

It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments

TABLE OF CONTENT:

1 Report Overview

2 Global Growth Trends

3 Market Share by Key Players

4 Breakdown Data by Type and Application

5 United States

6 Europe

7 China

8 Japan

9 Southeast Asia

10 India

11 Central & South America

12 International Players Profiles

13 Market Forecast 2019-2025

14 Analysts Viewpoints/Conclusions

15 Appendix

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COVID-19 Impact on Global Cell and Gene Therapy Market Trends Analysis, Top Manufacturers, Shares, Growth Opportunities, Statistics & Forecast to...

Recommendation and review posted by Bethany Smith

FRAMINGHAM, Mass.--(BUSINESS WIRE)--SCIEX, a global leader in life science analytical technologies, honors its 50th anniversary with the digital launch of pioneering products. On July 10th, the first of its kind SCIEX virtual product experience will introduce products that enable customers to Run Fast, Go Beyond and Power Up.

Half a century ago, SCIEX set out with a mission to develop analytical technologies for aerospace. Today, our technologies cover analysis of environmental hazards, biomarkers discovery, clinical diagnostics, drug development, food safety and more, says Inese Lowenstein, President of SCIEX. Our 20K install base spans innovative labs and companies around the world. I am incredibly proud of the progress SCIEX has achieved and look forward to continued innovation in the next 50 years.

Since 1970, the portfolio has grown through significant development and acquisitions, transitioning through the years from MDS SCIEX to Applied Biosystems to AB Sciex to SCIEX. Along the way, key integrations such as capillary electrophoresis from Beckman Life Sciences, have also strengthened the portfolio.

The Run Fast, Go Beyond and Power Up experience on July 10th is as much the celebration of 50 years of progress as it is the answer to urgent needs voiced by customers. The challenging environment posed by the last few months has amplified these needs and we are proud to launch new products when customers need them most.

To highlight a few insights from our customers:

Lieve Dillen, Senior Principal Scientist for Assay Development and Analytical Support within the Development Bioanalysis group of Janssen R&D in Belgium: One of the critical aspects for me is sensitivity. Within our department, the most challenging applications independent of the analyte almost all require increased sensitivity. Often, intrinsic sensitivity is compromised by carryover and/or contamination.

Shane R. Needham, Co-Founder at Alturas Analytics: The next 10 years I see that we are going to have an improvement in sensitivity and that will improve our efficiency and productivity. We are definitely going to see more large molecule bioanalysis to support gene therapy programs with LC-MS.

Alex Krotulski, Research Scientist at The Center for Forensic Science Research & Education: Ten years from now, high resolution mass spec is really going to be the way forensic labs go. Labs will also need to consider the complexity of the science that they are using for their screening capabilitiesstreamlined workflows, good software, good hardware to make these instruments robust is going to be really important. Software is the translator for scientists. Finding user friendly software is keymaking sure it is easy to learn, easy to use, robust and not going to crash.

Bernard Bajema, Project Lead at Vitens: You have to look for different substances in water and some substances are difficult to measure because of the sensitivity of the instruments. A very sensitive instrument can reduce your work and you can get cheaper tests developed.

To learn more and register for Run Fast, Go Beyond and Power Up, visit https://sciex.com/showcase-2.

About SCIEX

SCIEX delivers solutions for the precision detection and quantification of molecules, empowering our customers to protect and advance the wellness and safety of all. We have led the field of mass spectrometry for 50 years. From the launch of the first ever commercially successful triple quadrupole in 1981, we have developed groundbreaking technologies and solutions that influence life-changing research and outcomes.

Today, as part of the Danaher (NYSE: DHR) family of global life science and technology innovators, we continue to pioneer robust solutions in mass spectrometry and capillary electrophoresis. Our customers are able to quickly respond to environmental hazards, better understand biomarkers relevant to disease, improve patient care in the clinic, bring relevant drugs to market faster and keep food healthier and safer.

Thats why thousands of life science experts around the world choose SCIEX to get the answers they can trust to better inform critical decisions that positively impact lives.

For more information, visit sciex.com.

Lets connect: Twitter, LinkedIn, Facebook, and Instagram.

Advances in human wellness depend on the power of precise science.

The SCIEX clinical diagnostic portfolio is For In Vitro Diagnostic Use. Rx Only. Product(s) not available in all countries. For information on availability, please contact your local sales representative or refer to https://sciex.com/diagnostics. All other products are For Research Use Only. Not for use in Diagnostic Procedures.

Trademarks and/or registered trademarks mentioned herein, including associated logos, are the property of AB Sciex Pte. Ltd. or their respective owners in the United States and/or certain other countries.

2020 DH Tech. Dev. Pte. Ltd. RUO-MKT-12-11920-A.

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SCIEX Honors 50 Years of Innovation With Virtual Product Experience Launch - Business Wire

Recommendation and review posted by Bethany Smith

New Jersey, United States,- Market Research Intellect sheds light on the market scope, potential, and performance perspective of the Global Gene Therapy in Oncology Market by carrying out an extensive market analysis. Pivotal market aspects like market trends, the shift in customer preferences, fluctuating consumption, cost volatility, the product range available in the market, growth rate, drivers and constraints, financial standing, and challenges existing in the market are comprehensively evaluated to deduce their impact on the growth of the market in the coming years. The report also gives an industry-wide competitive analysis, highlighting the different market segments, individual market share of leading players, and the contemporary market scenario and the most vital elements to study while assessing the global Gene Therapy in Oncology market.

The research study includes the latest updates about the COVID-19 impact on the Gene Therapy in Oncology sector. The outbreak has broadly influenced the global economic landscape. The report contains a complete breakdown of the current situation in the ever-evolving business sector and estimates the aftereffects of the outbreak on the overall economy.

Leading Gene Therapy in Oncology manufacturers/companies operating at both regional and global levels:

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The Gene Therapy in Oncology market report provides successfully marked contemplated policy changes, favorable circumstances, industry news, developments, and trends. This information can help readers fortify their market position. It packs various parts of information gathered from secondary sources, including press releases, web, magazines, and journals as numbers, tables, pie-charts, and graphs. The information is verified and validated through primary interviews and questionnaires. The data on growth and trends focuses on new technologies, market capacities, raw materials, CAPEX cycle, and the dynamic structure of the Gene Therapy in Oncology market.

This study analyzes the growth of Gene Therapy in Oncology based on the present, past and futuristic data and will render complete information about the Gene Therapy in Oncology industry to the market-leading industry players that will guide the direction of the Gene Therapy in Oncology market through the forecast period. All of these players are analyzed in detail so as to get details concerning their recent announcements and partnerships, product/services, and investment strategies, among others.

Sales Forecast:

The report contains historical revenue and volume that backing information about the market capacity, and it helps to evaluate conjecture numbers for key areas in the Gene Therapy in Oncology market. Additionally, it includes a share of each segment of the Gene Therapy in Oncology market, giving methodical information about types and applications of the market.

Reasons for Buying Gene Therapy in Oncology Market Report

This report gives a forward-looking prospect of various factors driving or restraining market growth.

It renders an in-depth analysis for changing competitive dynamics.

It presents a detailed analysis of changing competition dynamics and puts you ahead of competitors.

It gives a six-year forecast evaluated on the basis of how the market is predicted to grow.

It assists in making informed business decisions by performing a pin-point analysis of market segments and by having complete insights of the Gene Therapy in Oncology market.

This report helps the readers understand key product segments and their future.

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In the end, the Gene Therapy in Oncology market is analyzed for revenue, sales, price, and gross margin. These points are examined for companies, types, applications, and regions.

To summarize, the global Gene Therapy in Oncology market report studies the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and the trends observed in the market that can either propel or curtail the growth rate of the industry. The market factors impacting the global sector also include provincial trade policies, international trade disputes, entry barriers, and other regulatory restrictions.

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Gene Therapy in Oncology Market Growth By Manufacturers, Type And Application, Forecast To 2026 - 3rd Watch News

Recommendation and review posted by Bethany Smith

We are unleashing our nations scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldnt describe the study in any further detail because, they said, its disclosure would be market-moving.

NBC News, July 3

Somethings cooking. And its not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that wont tamp down on the hurricane of criticism thats been tearing at the White House, and buffeting the presidents most stalwart core defenders as the economy suffers.

Weve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trumps initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness killing trials that arent likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Flagship execs take a lesson from nature to master 'gene writing,' launching a star-studded biotech with big ambitions to cure disease - Endpoints...

Recommendation and review posted by Bethany Smith

The global Gene Therapy market study presents an all in all compilation of the historical, current and future outlook of the market as well as the factors responsible for such a growth. With SWOT analysis, the business study highlights the strengths, weaknesses, opportunities and threats of each Gene Therapy market player in a comprehensive way. Further, the Gene Therapy market report emphasizes the adoption pattern of the Gene Therapy across various industries. The global Gene Therapy market has seen a historical CAGR of nearly XX% during the period (2020-2026) and is projected to create a valuation of about US$ XX Mn/Bn by 2026.

The Gene Therapy market report highlights the following players:

The Gene Therapy market report examines the operating pattern of each player new product launches, partnerships, and acquisitions has been examined in detail.

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The Covid-19 (corona virus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries, and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Gene Therapy Market globally. This report on Gene Therapy market provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcase market trends and forecast, factoring the impact of Covid -19 Situation.

Important regions covered in the Gene Therapy market report include:

The Gene Therapy market report takes into consideration the following segments by product type:

The Gene Therapy market report contain the following end uses:

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The Gene Therapy market report offers a plethora of insights which include:

The Gene Therapy market report answers important questions which include:

The Gene Therapy market report considers the following years to predict the market growth:

Why Choose Fact.MR?

Fact.MR follows a multi- disciplinary approach to extract information about various industries. Our analysts perform thorough primary and secondary research to gather data associated with the market. With modern industrial and digitalization tools, we provide avant-garde business ideas to our clients. We address clients living in across parts of the world with our 24/7 service availability.

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Global Gene Therapy Market on a Steady Growth Trail; Fact.MR Provides Projections in Light of COVID-19 Pandemic - The Cloud Tribune

Recommendation and review posted by Bethany Smith

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Gene TherapyMarket which would mention How the Covid-19 is Affecting the Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Key Regions and Proposal for Gene Therapy Market Players to battle Covid-19 Impact.

Theglobal Gene Therapy market has been remarkable momentum in the recent years. The Gene TherapyMarket report is one of the most comprehensive and important data about business strategies, qualitative and quantitative analysis of Global Market. It offers detailed research and analysis of key aspects of the Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the Gene Therapy market.

Top Leading players covered in the Gene Therapy market report: Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene and More

Get PDF Sample Report With Impact of COVID-19 on Gene Therapy [emailprotected] https://www.marketinforeports.com/Market-Reports/Request-Sample/101868

The Gene Therapy market report specifically highlights the market share, regional outlook, company profiles, product portfolio, a record of the recent developments, strategic analysis, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene in the market, sales, distribution chain, manufacturing, production, new market entrants as well as existing market players, advertising, brand value, popular products, demand and supply, and other important factors related to the market to help the new entrants understand the market scenario better. the global Gene Therapy market will showcase a steady CAGR in the forecast year 2020 to 2025.

Market Segment by Type covers:Ex vivoIn vivo

Market Segment by Application covers:Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

Our Complimentary Sample Gene Therapy market Report Accommodate a Brief Introduction of the research report, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology.

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Regions Covered in the Global Gene Therapy Market: The Middle East and Africa (GCC Countries and Egypt) North America (the United States, Mexico, and Canada) South America (Brazil etc.) Europe (Turkey, Germany, Russia UK, Italy, France, etc.) Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

Years Considered to Estimate the Gene Therapy Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year: 2020-2025

Highlights of the Report: Accurate market size and CAGR forecasts for the period 2019-2025 Identification and in-depth assessment of growth opportunities in key segments and regions Detailed company profiling of top players of the global Gene Therapy market Exhaustive research on innovation and other trends of the global Gene Therapy market Reliable industry value chain and supply chain analysis Comprehensive analysis of important growth drivers, restraints, challenges, and growth prospects

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COVID-19 Update: Global Gene Therapy Market is Expected to Grow at a Healthy CAGR with top players Sangamo, Spark Therapeutics, Dimension...

Recommendation and review posted by Bethany Smith

New York, July 06, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Biologics Safety Testing Market Research Report by Test Type, by Application - Global Forecast to 2025 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p05913604/?utm_source=GNW

On the basis of Test Type, the Biologics Safety Testing Market is studied across Adventitious Agent Detection Tests, Bioburden Tests, Cell Line Authentication and Characterization Tests, Endotoxin Tests, Residual Host Contaminant Detection Tests, and Sterility Tests.

On the basis of Application, the Biologics Safety Testing Market is studied across Blood and Blood-Related Products Testing, Cellular and Gene Therapy, Stem Cell Research, Tissue and Tissue-Related Products Testing, and Vaccine & Therapeutics Development.

On the basis of Geography, the Biologics Safety Testing Market is studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas region is studied across Argentina, Brazil, Canada, Mexico, and United States. The Asia-Pacific region is studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, South Korea, and Thailand. The Europe, Middle East & Africa region is studied across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Company Usability Profiles:The report deeply explores the recent significant developments by the leading vendors and innovation profiles in the Global Biologics Safety Testing Market including Avance Biosciences Inc., Charles River Laboratories International, Inc., Cytovance Biologics, Inc., Eurofins Scientific Se, Lonza Group Ltd., Merck KGaA, Pace Analytical Services Inc., Sartorius AG, Source Bioscience, Thermo Fisher Scientific Inc., Toxikon Corporation, and Wuxi Apptec.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Biologics Safety Testing Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, so for and, the long-term effects projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlaying COVID-19 issues and potential paths forward. The report is delivering insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecast, considering the COVID-19 impact on the market.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on sulfuric acid offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:1. What is the market size and forecast of the Global Biologics Safety Testing Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Biologics Safety Testing Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Biologics Safety Testing Market?4. What is the competitive strategic window for opportunities in the Global Biologics Safety Testing Market?5. What are the technology trends and regulatory frameworks in the Global Biologics Safety Testing Market?6. What are the modes and strategic moves considered suitable for entering the Global Biologics Safety Testing Market?Read the full report: https://www.reportlinker.com/p05913604/?utm_source=GNW

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The Global Biologics Safety Testing Market is expected to grow from USD 3,410.53 Million in 2019 to USD 6,802.68 Million by the end of 2025 at a...

Recommendation and review posted by Bethany Smith

A new report titledGlobal Gene Therapy Marketpublished by Fior Markets gives an in-depth overview of the market, detailing the latest product/services/industry coverage and market forecasts and status by 2025. The total report is divided by company, by country, and by application/types for the competitive landscape analysis. This report is categorized as a key area for accelerating the marketization. The report then estimates 2020-2025 market development trends of global Gene Therapy Industry. This comprehensive research report comprises a whole market scenario along with the dynamics affecting it. The study uncovers the gaps and opportunities to derive the most relevant insights from this research document to gain market size.

NOTE: Our report highlights the major issues and hazards that companies might come across due to the unprecedented outbreak of COVID-19.

DOWNLOAD FREE SAMPLE REPORT:https://www.fiormarkets.com/report-detail/376052/request-sample

The major components analyzed in the report are the markets product type, application, end-use, the solution, and the services that are offered. Both numerical data and subjective information about every segment are featured for better understanding. Major players in the global Gene Therapy market are identified by determining percentage splits, market shares, growth rate, and breakdowns of the product markets. The research document includes analysis of growing competition, key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, main regions, demand, and developments.

REQUEST FOR CUSTMIZATION:https://www.fiormarkets.com/enquiry/request-customization/376052Geographically, the report includes the research on production, consumption, revenue, market share and growth rate, of the following regions:North America, Europe, Asia Pacific, South America, and the Middle East and Africa.

Top players listed in the market report are:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

Moreover in the report, based on product and the end users/applications, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share, and growth rate for each type and application. The competitive landscape chapter of the global Gene Therapy market report provides detailed information about market players such as company overview, total (financial) revenue, market potential, global presence, market share, production sites and facilities, products offered and strategies adopted. Noteworthy patterns, drivers, impact factors worldwide, and areas are further recognized in the report.

BROWSE COMPLETE REPORT AND TABLE OF CONTENTS:https://www.fiormarkets.com/report/global-gene-therapy-market-by-type-germline-gene-376052.html

Major Advantages of Market:

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team (sales@fiormarkets.com), who will ensure that you get a report that suits your needs.

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COVID-19 After Effects on the Global Gene Therapy Market 2020 - NJ MMA News

Recommendation and review posted by Bethany Smith

The report on the Cancer Gene Therapy market provides a birds eye view of the current proceeding within the Cancer Gene Therapy market. Further, the report also takes into account the impact of the novel COVID-19 pandemic on the Cancer Gene Therapy market and offers a clear assessment of the projected market fluctuations during the forecast period. The different factors that are likely to impact the overall dynamics of the Cancer Gene Therapy market over the forecast period (2020-2026) including the current trends, growth opportunities, restraining factors, and more are discussed in detail in the market study.

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Cancer Gene Therapy Market Is Expected to Foresee an Outstanding Growth by 2024 - Apsters News

Recommendation and review posted by Bethany Smith

Carmine Therapeutics and Takeda Collaborate to Discover and Develop Rare Disease Gene Therapies Using Novel Red Blood Cell Extracellular Vesicles Platform

Singapore based startup Carmine Therapeutics has announced that it has signed a research collaboration agreement with Japanese firm Takeda Pharmaceutical Company Limited o discover, develop and commercialize transformative non-viral gene therapies for two rare disease targets using Carmines REGENT(TM) technology, based on red blood cell extracellular vesicles. In addition, Takeda has committed a $5M convertible loan in support of the development of Carmines novel REGENT platform.

Under the terms of the agreement, Carmine will receive an upfront payment, research funding support, and is eligible for over $900M in total milestone payments plus tiered royalties.

Takeda has an option to license the programs following the completion of pre-clinical proof of concept studies and would be responsible for clinical development and commercialization.

Carmine Therapeutics is pioneering a novel class of therapeutics based on its REGENT(TM) technology which leverages red blood cell extracellular vesicles (RBCEVs), first published in Nature Communications in 2018.

An initial focus is non-viral gene therapies. Compared to adeno-associated virus (AAV)-based gene therapy, RBCEV-based gene therapy has the potential for repeat dosing, a significantly larger transgene payload capacity in excess of 11KB, and enhanced bio-distribution in selected tissues through RBCEV surface modification.

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Carmine Therapeutics teams up with Takeda for non-viral gene therapies - BSA bureau

Recommendation and review posted by Bethany Smith

By the time the FDA finally granted an accelerated OK for Immunomedics Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

Thats because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

We do not know the totality of their decision other than its pretty evident that the primary endpoint was met; otherwise they could not request to halt the study, Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.

Three months later, Immunomedics has detailed the results from ASCENT, including progression-free survival and overall survival data that the principal investigator said set a new benchmark in mTNBC.

PFS was the primary endpoint, and Trodelvy an antibody-drug conjugate known as sacituzumab govitecan kept the cancer at bay (and patients from dying) for a median of 5.6 months, compared to 1.7 months on chemotherapy (p<0.0001). The hazard ratio registered at 0.41.

Shares $IMMU rose 6.67% to $40.04.

Immunomedics stopped short of spelling out the OS win, but noted that and other key secondary endpoints such as objective response rate were met.

Importantly, the ASCENT topline data also validate the manageable safety profile of sacituzumab govitecan, rendering it a good partner candidate for combination with other therapies, including immunotherapy, Aditya Bardia, the principal investigator and director of precision medicine at Mass Generals Center for Breast Cancer, said.

Trodelvy was approved, with a black box warning for severe neutropenia and severe diarrhea, as a third-line treatment for mTNBC, a tough-to-treat cancer associated with poor prognosis. The new data are expected to support a full OK.

It is gratifying to see the final confirmatory results of Trodelvy in a randomized study supporting the previously reported Phase 2 data that formed the basis of the accelerated approval of Trodelvy, Aghazadeh, who has been running the show since Michael Pehl left the helm early 2019 in the wake of an FDA rejection, said in a statement.

Novartis Oncology vet and Ipsen alum Harout Semerjian came on board briefly as CEO in April, only to depart less than two months later as the pandemic created insurmountable challenges for moving his family from London.

The hiccup hasnt dented Cowen analyst Phil Nadeaus high expectations of the therapy, which his consultants said will be rapidly adopted as the standard in relapsed or refractory mTNBC, bringing in $1 billion in worldwide revenue by 2025 (Everest Medicines is handling the development and commercialization in China). According to Nadeau, the numbers hit the upper end of expectations.

Investors anticipated that Trodelvy would produce a median PFS of 5-5.5 months consistent with its Ph. I/II, but our sense is that most anticipated a median PFS for chemotherapy of 2.5-3 months, he wrote.

And thats not taking into account its potential in ER+/HER2- metastatic breast cancer or urothelial carcinoma, which now seems even more likely given the positive ASCENT data.

Investors underestimate the sales potential of Trodelvy, he wrote in a recent note. Trodelvys data suggest it is a similar improvement over the standard of care in r/r ER+/HER2- mBC as it is in mTNBC, and there are approx. 3-4x as many women with r/r ER+/HER2- mBC. This market is a $3-4B opportunity for Trodelvy, not yet fully reflected in estimates or in IMMUs valuation, despite potentially pivotal data anticipated around YE.

See the original post here:
UPDATED: Immunomedics spells out PFS benefit of Trodelvy in mTNBC, hunting a full OK just weeks after accelerated approval - Endpoints News

Recommendation and review posted by Bethany Smith

Putting your employees and company culture first keeps the focus on inclusion and innovation, giving the company an incredible competitive advantage. At least thats the mantra at the heart of MyoKardia, a California-based biotech company that is developing precision medicine for cardiovascular diseases (CVDs).

We want to change the world for people with cardiovascular disease by taking a patient-focused, scientifically driven approach, Tassos Gianakakos, MyoKardias CEO, told BioSpace. When youre addressing hard problems, you need different opinions, approaches, and expertise at the table. That is the only way to effectively deliver on the mission.

Companies are at risk of getting it wrong when they dont focus on culture early on you need to launch out of the gate with a culture mindset, Gianakakos added. You get back what you put out there, so being a mission-driven, culture-valuing company will help draw in likeminded employees. That group genius is what enables remarkable improvements to health outcomes for society.

CVD, also called heart disease, is a blanket term used to describe many diseases that affect the heart or blood vessels. Globally, heart diseases are by far the number one killer in the world, with CVDs responsible for 17.9 million deaths worldwide. These conditions are highly prevalent throughout the population 30.3 million US adults have been diagnosed with CVDs.

Credit: WHO

We lose more people in the U.S. and around the world to cardiovascular conditions than any other disease, Gianakakos. MyoKardias entire purpose is to change that. We want to be the leading company developing precision medicine for CVDs. Our approach is different; were subtyping patient populations within these large, heterogeneous conditions so that we can identify effective, targeted therapeutics. The idea is to discover and develop medicines that have transformative potential for people.

MyoKardias late-stage pipeline focuses on two CVDs: hypertrophic cardiomyopathy (HCM), where the heart muscle becomes abnormally thick (hypertrophied), making it harder for the heart to pump blood; and dilated cardiomyopathy (DCM), where the hearts main pumping chamber (called the left ventricle) stretches and thins (dilates), making it harder for the heart to pump blood.

HCM is frequently caused by gene mutations in heart muscle proteins that cause the heart muscle to squeeze with more force than needed, leading to abnormal thickening over time. It is the most common inherited heart disease, occurring in about 1 in 500 people (over 650,000 people in the US). HCM is the most common cause of cardiac arrest (where the heart suddenly stops beating), in younger people. Although certain medications, like beta blockers and blood thinners, are used to treat some HCM symptoms, there arent any drugs that specifically address the underlying problem in HCM the genetic mutation-induced thickened heart muscle.

Positive results from a Phase III clinical trial of mavacamten, MyoKardias lead drug candidate for HCM, were announced in May. MyoKardia aims to submit a New Drug Application (NDA) submission with the FDA in the first quarter of 2021 and is planning for its first product launch.

DCMs causes may be varied in addition to genetics, a number of diseases are linked to left ventricle dilation, including diabetes, obesity, high blood pressure, infections, and drug and alcohol abuse. It is a common cause of systolic heart failure (where the heart isnt pumping blood as well as it should be). Medications such as angiotensin-converting enzyme (ACE) inhibitors, beta blockers, and blood thinners can successfully treat heart failure, but none of them are specific to the heart and have systemic side effects.

MyoKardias investigational drug danicamtiv is intended to increase heart contractions without interfering with the hearts ability to fill. The company recently reported encouraging data from their Phase IIa study of danicamtiv in chronic heart failure patients. They plan to advance into two new Phase II studies in specific patient populations: genetic DCM patients and systolic heart failure patients with paroxysmal or persistent atrial fibrillation (AFib).

BioSpace spoke to Gianakakos and Ingrid Boyes, MyoKardias Senior Vice President of Human Resources, about the companys pipeline, culture, and why building a culture of diversity and inclusion is foundational to a company.

(Boyes previously spoke to BioSpace in 2015 about what MyoKardia is looking for when theyre hiring.)

COMPANY CULTURE, DIVERSITY & INCLUSION

BioSpace: Why is company culture and diversity so important to a successful company? How do you promote diversity and inclusion at MyoKardia?

Gianakakos: The diseases we are tackling know no ethnic, gender or socioeconomic boundaries. So our company culture needs to reflect this. Our teams need to reflect this and the patients we are working to help. Its hard for us to see doing good science and achieving our mission any other way. And it goes beyond the science. To have a successful and meaningful company, we need to innovate more broadly in growth strategies, commercial models, and new ways to more effectively get our therapies to patients who need it around the world.

Im proud of how we embrace each others differences gender, ethnicity and race, orientation, socioeconomic status and beliefs -- and highlight the importance of company culture. Everyone at MyoKardia shares the same mission, the same values, but we embrace and value each persons differences. We want our employees to feel safe sharing their own voice and know that different points of view are valued and respected.

Boyes: Tassos passion for company culture is a large part of why I joined the company five years ago. As a Hispanic woman, its really important to me to create an environment where people can thrive and grow. We have fun while creating a valuable community. As employee number 50, I was able to focus on how to help build a company culture with Tassos that values diversity by building on employees experiences. We were very intentional about company culture and how we evolve it. Every voice at MyoKardia counts and every person plays an important role in improving CVD patients lives.

We actively seek input from our employees and encourage them to challenge the status quo. We also invite employees to lead activities and bring their unique perspectives to work.

Gianakakos: We want to bring great people who are passionate to the company and play to their strengths. Focusing on increasing their engagement and creating an energizing work environment allows employees to do their most creative and best work. Having people build the skills they want and need by cross-training and encouraging lifelong learning improves the connectivity and the innovation within the company.

We believe this is one of the key competitive advantages at MyoKardia connecting and supporting people to engage and excite them and ensuring they have a voice that is valued. Having diverse perspectives and a commitment to listening leads us to much better decisions and results.

What kind of diversity and engagement activities do you do both within MyoKardia and externally with the general public?

Boyes: We always strive to improve the culture by actively soliciting feedback from our employees though a number of channels, including engagement surveys. Implementing employee-led initiatives has brought great features into the companys culture, such as a womens forum that brings in external women speakers and identifies female role models, a green team focused on being more sustainable, and a community volunteer team that actively supports our community. All of these activities also help to develop valuable leadership skills regardless of title within our organization.

Gianakakos: Based on employee feedback, weve also implemented several policy changes, such as increasing the companys 401k match and giving each employee a six-week sabbatical once they have been with the company for six years.

Boyes: We want to be connected with diverse organizations and participate as much as possible externally connecting with others in the community with culture-focused passion. We are always looking to connect with driven people who share our company values.

Switching gears to the science, what does CVD drug development look like right now?

Gianakakos: In many ways, CVD is where oncology was 20 years ago there were no precision medicines and non-specific treatments such as chemotherapy and radiation were used regardless of cancer type. The number of drugs in development for CVDs is woefully low relative to its global burden. There are over 1,100 oncology drugs in development, but only 200 for cardiovascular diseases, despite CVDs killing more people annually than all cancers combined. In oncology today, precision medicine approaches have given us countless targeted therapies that have completely transformed patient care. We are making this happen today in CVD, where we feel may even have advantages over oncology given the many tools now available to monitor the heart, such as wearables and patches that measure the heart rate and rhythm.

What made you focus on precision cardiac medicine? Why now?

Gianakakos: Momentum around precision medicine in other disease areas was clearly growing and resulting in important advances when MyoKardia started eight years ago. The first cystic fibrosis drug that treated the underlying cause rather than the symptoms (ivacaftor) was just launched by Vertex and a few years prior to MyoKardia our founding investors were involved in launching several exciting new companies like Foundation Medicine, Agios and bluebird bio who were developing potentially game-changing targeted therapies.

Traditionally, CVD clinical trials are massive, expensive, and often fail. When there is a lack of understanding of the underlying disease biology and its unclear exactly what the drug is doing, that can result in a large signal-to-noise ratio. This in turn, requires larger studies which are more expensive, and the therapies have to benefit large numbers of patients for the investment to make sense. This is a recipe that doesnt lead to innovative or efficient drug discovery. Identifying smaller, more homogenous subgroups of patients who all share the same disease pathology, and targeting them with drugs designed specifically to address the underlying disease biology is so powerful. Were matching the tailored treatment to address each persons underlying condition understanding how to identify the right drugs for the right patients.

CARDIOVASCULAR DISEASE DRUG DEVELOPMENT & MYOKARDIAS PIPELINE

What are the major knowledge gaps that need to be addressed to make precision cardiac medicine achievable for many patients? What does the landscape look like right now for precision cardiac medicine?

Gianakakos: There needs to be a cultural shift in the CVD field to move away from grouping broad heterogenous patients together, to focusing on smaller, well defined patient groups treated with targeted therapies and learning as much as we can from those that respond very well and, as importantly, those that do not.

Matching patient profiles to drugs that specifically address their underlying disease is key. Leaning on existing technology, such as wearables, genetic sequencing, imaging, and biomarker profiles to subtype CVD patients and deeply understand the biological drivers of disease will lead to critically important targeted therapies and much more effective clinical trials.

In terms of other precision cardiac medicine approaches in development, gene therapies are being explored. While that technology is maturing, most gene therapies for CVDs are still in early-stage research, but eventually could be helpful for certain sub-groups of patients with CVD.

Relative to other disease areas, like oncology, it has been challenging for companies to invest in new approaches to drug discovery and development in areas like CVD and neurology. However, given the staggering medical need, and with progress being made by companies like ours, I expect interest in CVD precision medicine to increase over the next 3-5 years.

What does MyoKardias pipeline look like?

Gianakakos: Our Phase III drug, called mavacamten (MYK-461), is for HCM. HCM is a genetic disease where the heart thickens due to excessive force of contraction cause by mutations in the heart muscle proteins. There are two common subtypes of HCM: obstructive, where the thickening also occurs near the base of the aorta and prevents (obstructs) blood from flowing well out of the heart; and non-obstructive, where the thick muscle makes it challenging for the heart to relax and fill, reducing the amount of blood flow out of the heart without physically obstructing blood flow. About one-third of HCM patients have the non-obstructive type.

Mavacamten is a small molecule that targets the heart muscle protein myosin reducing the excessive force of contraction, directly addressing the underlying cause of HCM. We announced positive data from our Phase III trial (EXPLORER-HCM) of mavacemten in about 250 symptomatic obstructive HCM patients and we are now able to move full steam ahead on our first regulatory submission for approval. Encouraging results from a Phase II trial (MAVERICK-HCM) of mavacamten in about 60 participants with symptomatic non-obstructive HCM were recently presented and we are going to be moving mavacamten forward in non-obstructive patients. We are also conducting a long-term extension study is also ongoing for patients who participated in either EXPLORER-HCM or MAVERICK-HCM.

We started hyper focused in a disease with a defined genetic background and will expand in a deliberate way into adjacent diseases with similar problems, such as heart failure with preserved ejection fraction. About 3 million people in the U.S. have problems filling and relaxing their hearts and we estimate that approximately 10% of them share similar pathology to HCM. Are these disease subtypes related? Do they have similar genetic mutations? We plan to start a Phase II trial in the next few months to explore if mavacamten can help that specific heart failure population and learn much more about this devastating form of heart failure.

We also have a Phase II molecule, called danicamtiv (MYK-491), for DCM that is designed to increase the force of contraction in the heart - the opposite of what mavacamten has been created to do. Danicamtiv is a small molecule that selectively increases the number of myosin-actin cross bridges, supporting heart muscle contractions to help the heart pump more efficiently. It has recently completed a Phase Ib/IIa trial in DCM or stable heart failure patients and has shown very promising early results. We are now moving into a separate Phase II study in DCM patients with certain genetic mutations. Among the most interesting new findings from our clinical study of danicamtiv is that it appears to have a direct effect on the performance of the left atrium. We were able to confirm and learn more about these findings in nonclinical studies, which is leading us to explore danicamtiv in patients with systolic dysfunction and atrial fibrillation.

MyoKardia has gone from startup to successfully completing our first Phase III trial in eight years. In the coming months, we will be submitting our first drug to the FDA this year, which if approved will bring the first every therapy designed specifically for HCM to people with this debilitating condition.

We design our therapies with the aim of targeting the underlying disease mechanism to treat and, in some cases, reverse the problem, actually slowing down or reversing disease progression. That allows patients to live full lives, free from fear and complications. We are very excited and remain super ambitious. The magic and special sauce is really our employees and our culture.

Go here to see the original:
MyoKardia: The Precision Cardiac Medicine Company with Diversity and Inclusion at its Heart - BioSpace

Recommendation and review posted by Bethany Smith

Dear Dr. Roach I am 80 years old. Two years ago, I was diagnosed with hyperthyroidism. I am taking methimazole 10 milligrams, two tabs daily. Whenever the endocrinologist reduces the tablets to once daily, my TSH remains low. I am also taking metoprolol. I read that beta blockers interfere with the absorption of methimazole. Should I take a different medication? M.W.

Answer Hyperthyroidism is when the thyroid gland releases too much hormone into the blood. This may cause many symptoms, such as fast heart rate, tremor, anxiety, weight loss, excess bowel movements and shortness of breath. Methimazole blocks the synthesis of thyroid hormone, but often beta blockers such as metoprolol are also used to treat some symptoms, especially the fast heart rate, anxiety and tremor. Beta blockers work quickly and are usually prescribed as soon as hyperthyroidism is diagnosed.

Since they are so often used together, I was surprised to hear you read beta blockers block absorption of methimazole nothing in medical literature supports that. It is true that once a person has gone from hyperthyroid to normal thyroid, the body can absorb metoprolol better, so the dose may need to be decreased.

TSH is thyroid stimulating hormone, the bodys signal to release thyroid hormone. In people with hyperthyroidism due to a problem with the gland Graves disease, an autoimmune thyroid disease the TSH level is very low. More methimazole would be expected to raise the TSH, while decreasing the dose should make TSH lower.Dear Dr. Roach I am 84 years old and in good health. I recently read that most Americans dont get enough potassium, which lowers blood pressure. Assuming I was one of the majority, I started taking a potassium supplement. Wise or not? C.H.

Continued here:
Beta blockers work with other hyperthyroidism meds | To Your Good Health - STLtoday.com

Recommendation and review posted by Bethany Smith

On June 7, the Los Angeles County Board of Supervisors will vote on a new motion co-authored by Supervisors Mark Ridley-Thomas and Janice Hahn, that, if passed, will reinstate the fundingfor the long-planned and much-anticipated Probation Oversight Commission or POC,that was slashedat last weeks board meeting.

(We wrote here about last weeks budget-slashing, and about the importance of funding the POC.)

Then, over the weekend, WitnessLA received a copy of a newly-filed complaint against LA County, which described a teenage boy who was in one of probations juvenile hallswhen he was allegedly forced against his will, and without his parents knowledge, into an extremely unusual form of treatment, by various medical professionals who work inside probations youth facilities.

The story is an alarming one, and it illustrates one more timewhy so many youth advocates and othersbelieve that civilian oversight of the nations largest probation department is urgent.

Heres the deal.

Hormones for teenage boys

On June 25, 2019, just a little over a year ago, a 16-year-old boy (whom well call Javier, to protect his anonymity) had just been detained and sent toCentral Juvenile Hall, one of the countys two remaining juvenile halls, located northeast of downtown LA, on Eastlake Avenue.

At Eastlake, as the hall is often known, Javier was givena physical examination by some of the facilities medical personnel.

According to the complaint, during the June 25, 2019, exam,medical stafffirst administereda blood test, and a urine test, after which Dr. Danny Wang, reportedlydetermined that Javier had slightly elevated testosterone levels.

Javier was, after all, a teenager, which meanthis hormones were likely to be bouncing quite a bit.

But in Javiers case, the physician who examined him reportedly diagnosed him with Oppositional Defiance Disorder, or ODD, then elected to prescribe an unusual form of medication, with the idea that it would make the boyless aggressive.

(Dr. Wang and any other medical professionals dealing with mental health in probationshalls and camps, dont work for probation directly, but for the LA County Department of Mental Health.)

ODD, as it is known, is not an uncommon diagnosis for boys and girls who come in contact with the juvenile justice system.In thedeeply-researched Los Angeles County Probation Outcomes Report of 2015, lead author Dr. Denise Herz, and her co-authors,found that 92 percentthe kids in LA County Probations carehad some kind of mental health diagnosis out of the American Psychiatric Associations Diagnostic and Statistical Manual of Mental Disorders. In the case of probations kids,the report found that Disruptive Behavior Disorder, Oppositional Defiant Disorder (ODD), and a couple of other unpleasantly named mood disorders, were diagnosed 58 percentof the time.

And of course, once there is a diagnosis, treatment often follows and, in juvenile facilities that treatment is often medication.

In Javiers case, themedication was Estradiol, which is a form of the female hormone estrogenthatis most commonlyused by women to help reduce symptoms of menopause.

Wang reportedly prescribed 30 doses of the stuff, to be taken daily, all without Javiers parents knowledge or permission. Javier was reportedly given his first estrogen treatment in the form of three vaccinations.

Worried, Javier told the medical people that hed not given permission to be given any drugs or medicine, and asked what the stuff was for. The nurse reportedly falsely told the teenager that it was to treat a small nodule on his chest.

Javier was still uncomfortable with taking the mystery medication. At first, he said he would refuse to take it. Then reportedly,one of the medical staff told him he could not in fact refuse. Javiers probation officer was also standing nearby when the conversation took place, and reiterated that Javier didnt have a choice in the matter.

Later, Javier would tell his parents, and his lawyer,Wesley G Ouchi,that he was afraid of getting a negative write up, by staff members that could be reported to the judge and negatively affect his case, as the judge had threatened to make his sentence longer and harsher, possibly even send him to the states Department of Juvenile Justice system (DJJ), unless he ran a good program, and got zero write-ups.

Hormone storm

Fearful about giving staff any reason to criticize his behavior, Javier took the Estradiol, as he was instructed.

Replacement hormones like Estradiol are powerful and almostmost immediately, Javier began to feel strange and sick. According to the complaint, the effects were physical, emotional, cognitive, and psychological.

And then he began to grow breasts. Really, it wasmore of a swelling on his chest than actual boobs. But the weird swellings were visible to other kids, said attorney, Wes Ouchi, when WitnessLA spoke to him. Andthen, of course, the teasing began.

Javieralso broke out in pimples on his face, scalp, and body.

He became depressed, and couldnt concentrate. He was highly anxious, had a hard time controlling his thoughts. The teasing from other boys didnt help.

Finally, after 13 doses, a desperate Javier refused the treatment, write-ups, or no write-ups and, this time, he stuck to it.

After he refused treatment, Dr. Wang andhis boss Dr. David Oh, told his parents about the treatment.

But didJavier really receive any kind of accepted treatment?

Asfar as attorney Ouchi has been able to determine, the answer is no.

There isno any kind of indication that providing Estrodial to an adolescent male is any kind of accepted treatment for anything, he said. We think it was an experiment. And we think itslikely not the only time they used the treatment, experimentally.

He and hisfirmhavent done discovery yet, Ouchi said. So they are just at the beginning stages in probing the case more deeply. Yet, he believes theres lots more to be uncovered.

Ouchiisinterested, he said, to find out if there are other teenage boys in probations system who were given estrogen in this potentially purely experimental manner, which he described as potentially criminal.

UCLAs Dr. Jorja Leap, who was part of the planning for the POC, and one of the co-authors ofthe deeply researchedLA County Probation Governance Report, was very disturbed by the implications of the new lawsuit.

These troubling developments point to the need for real oversightwith powerto hold probation, andthose fromother county agencies, whowork inside probations facilities,fullyaccountable when they harm those in their care, she told us.

(You can read the full complaintright here.)

So, yes, fund the POC!

Later this week, WitnessLA willhave a report onanother disturbinglawsuit having to do with LA County Probation, this time pertaining to something that happened during the evacuation of the kids at Barry J. Nidorf Juvenile to the then deserted Las Padrinos Juvenile hall.

This second lawsuit also pointsto the critical need for oversight.

These recent allegations and many others speak to the vital necessity of robust, credible, and meaningful civilian oversight over the Los Angeles County Probation Department, said Cyn Yamashiro after reading the complaint having to do with Javiers case, and discussing the second lawsuit.

The county cannot continue to formulate policy by responding to litigation. Reacting to crises and civil claims as a means of developing public policy ignores the profound harm suffered by victims of this type of abuse and costs the county desperately needed scarce resources.

Yamashiro is the Directing Attorney of the LA County Bar Associations Independent Juvenile Defender Program. He is also on the existing Probation Commission, and on the temporary blue-ribbon commission that designed the POC.

Probation Department reform has been at the top of the list in terms of difficulty in bringing about change in LA County, Supervisor Ridley-Thomas said of the PUC vote, when WLA asked him about the matter. Here it is a full decade-plus, and were just now getting around to establishing an oversight entity with teeth. I never would have imagined it would be this difficult. But I want to give props to the advocates, to the juvenile probationers, to the parents, to the community at large who never gave up, to some of the nonprofit providers, interventionists and a whole host of individuals who know theres a better way to speak the language of reform, rehabilitation, andreentry.

Yep.

Go here to see the original:
New Lawsuit Tells of 16-Yr-old Boy Allegedly Forced By County Officials to Take Estrogen as Medication to Control His Behavior | - witnessla.com

Recommendation and review posted by Bethany Smith

By Maddy Zee | July 6th, 2020

As research into Alzheimers disease progresses and awareness grows, one fact remains consistent: women are disproportionately affected by the disease. Several theories exist, but a recent study suggests that the link between Alzheimers and menopause, a part of aging for most women, could be the answer.

Being Patient spoke with Dr. Richard Isaacson, a neurologist and director of Weill Cornell Universitys Alzheimers Prevention Clinic, about his involvement in the study on menopause and the role of the link between Alzheimers and menopause in future research.

Being Patient: What is it about our hormones thats related to the brain? What do we know about the connection between estrogen and the brain?

Dr. Richard Isaacson: Well, well take one little step back. If you were to ask me this question ten years ago, I would have said I have no idea. Five years ago, we started the Alzheimers prevention clinic at Weill Cornell and New York Presbyterian back in 2013. It took a couple years to kind of get my feet wet and understand not just how people are doing, but how sex differences go in pre-clinical normal patients who are at risk for Alzheimers.

It started becoming much more clear like you said that its not just because women live longer. Two out of every three brains affected by Alzheimers is a womans brain, and we used to say we dont know why. Honestly, I dont believe that thats the case anymore. Im pretty confident that we have significant clues that can not just understand the risk factors, but also how we can maybe intervene to reduce risk. All that being said, there are, I believe, several reasons and its not just hormones.

I think hormones are very important and well talk about that. Im a physician, Im a neurologist, Im a practicing clinician in the Alzheimers prevention clinic, so we get a lot of questions: should I be on hormones? What should I take? Should I take a pill? Should I take a cream? When should I start? Im not menopausal yet, Im just starting to have pre-menopausal symptoms, Im done with menopause, should I start now?

These are very common questions, and while I cant say we have the perfect road map or instruction manual of answers, there are multiple reasons why a woman could be on a different trajectory than a man. Hormones we will definitely talk about, but its more than that.

During the pre-menopause transition there are bio-energetic shifts, and what that means is that the brains energy pathways change. When that shift happens in certain women they will be on a different fast forwarding road to Alzheimers disease, and our goal as a clinician, my goal in the clinic, is to get them off of that road using evidence-based and safe interventions. Is hormone replacement one of those interventions? Im fairly sure it is, but its not a one-size fits all answer. When to start, what to start, theres just so many questions here and I think I understand it much more now than five years ago.

Being Patient: Estrogen traditionally begins dropping in a womens mid-thirties. Is it true that different women have different degrees of an estrogen drop? And is there evidence that sharper drops of estrogen, like falling off a cliff, are worse for the brain?

Dr. Richard Isaacson: Sure, so the falling off the cliff analogy is a good one and the thing that immediately makes me think of what would make a women fall of the cliff in terms of a precipitous and immediate drop in estrogen levels, the number one thing I can think of is surgical menopause, which basically means a hysterectomy where they take out the ovaries.

Now, in the past 20 years ago and earlier when women got hysterectomies, and theres a variety of reasons why women have to get hysterectomies, but when you take the uterus out and you take the ovaries out with it and prior to that the ovaries were working and there were some degree of regularity of ups and downs of estrogen which is normal throughout the cycle, when you take that out its like falling off a cliff. I dont know that we have the perfect answers and the perfect evidence, but both the study that we just published and also other studies basically suggest that when you fall off of that cliff, that could be a problem in certain women, in many women, but not all women. And thats a complicated thing.

Does the womans genetics play a role? I think yes. The other thing thats really important is, did that woman go on hormonal replacement therapy immediately? Okay, thats important. What was that hormonal replacement therapy? What was the dose? Was there progesterone? Was there just estrogen? What type of estrogen was it? Was it a pill? A patch? A cream? How long did they continue the estrogen replacement for?

If I had to say a yes or no and you boxed me into a corner, is estrogen protective on the brain? Sure, I would lean towards yes. I shake the eight ball, and all signs are pointing to yes. But its just not that simple, you know, a woman just cant just say that I heard this in this video, Dr. blah blah blah prescribed me estrogen, I want to prevent Alzheimers. Its, unfortunately, a little more complicated than that. What this study showed is that we kind of understand more now about how hormone replacement therapy, how the pre-menopause transition, how a hysterectomy for example, all these three things might not just affect Alzheimers risk but really how they affect brain pathology.

So in the brain we can look at characteristics from the size of the brain if theres shrinkage in parts of the brain that basically cause or are associated with Alzheimers, the hippocampus the memory center of the brain we look at the grey matter and the white matter, we look at the size of the brain or atrophy, we look at something called glucose metabolism. If we see hypo-metabolism, well thats a clue that there could be a problem especially if those are hypo-metabolic reduced energy consumption, and really reduced energy metabolism is a better term in parts of the brain related to Alzheimers.

And finally is there amyloid in the brain, amyloid is the pathologic protein that builds up in the brain of people with Alzheimers. Is it the cause of Alzheimers? I dont really think so. Is it related to Alzheimers? Absolutely, its a biomarker that accumulates in the brains of people with Alzheimers.

In that study we really showed that women who are in the pre-menopause transition and during menopause and also women that do hormone replacement therapy and other things, we understand now the relationship between those characteristics and brain pathology related to Alzheimers. So long story short, it takes a long time to figure this out and I think this study is important because its a little bit more clear now.

Being Patient: Do we just think that we are losing our memory, or are we actually losing our memory because were reaching menopause?

Dr. Richard Isaacson: Im going to give a collective apology on behalf of most physicians out there because when I was in med school almost twenty years ago we never learned that menopause causes cognitive changes, like that was not even on the radar, like zero. And then we kind of learned that there were some cognitive aberrations but its fine, its just due to, I dont know, hormones or whatever, lack of sleep, its not real.

And the problem is that I said its not real. Well absolutely thats not correct. These are real symptoms, these are absolutely real, but how Im going to characterize it is different from what you just said. These are absolutely real, tangible cognitive changes. You used the term memory, memory is a loaded term.

When Im talking the word memory, I know its memory because I do the tests. The problem with cognitive function, is that if your attention is less than it used to be, if your processing speed is lower than it used to be, if your ability to learn new information is less than it used to be, the person may say that theyre having a memory problem, but not necessarily. We test all these different factors, you know theres downstream and upstream right?

If someone cant consolidate information and get it into the memory banks, Papez circuit, long-term, short-term, if you cant consolidate the memory that may be an attention thing or a processing speed thing, thats not memory. So what I would say here is its essential to characterize the exact cognitive change related to the perimenopause transition before jumping on memory and saying, Oh, I have Alzheimers, I think.

All that being said, theres certainly some memory dysfunction, because a lot of the time its not memory. In the women that have a memory problem during the perimenopause transition, who have multiple family members with Alzheimers that have an ApoE4 variant one or two, thats when I start getting a little worried.

Thats when I say, How is your exercise? What is your cholesterol? I need to know everything about you. Not just how many days a week you are exercising, what are you doing? What is your average heart rate during your exercise? Oh, youre on an elliptical but youre texting, no thats not intense exercise enough. Oh youre not doing strength training, what do you mean, you need to build muscle, because if you dont build muscle youre not going to increase your metabolism.

I get really serious with women who truly have an objective memory problem, we do like multiple different cognitive tests in our cognitive battery 45-55 minutes, if its a memory problem, Im legitimately concerned and and I will predict that there is a bio-energetic change in the brain that we can really detect. So, I completely buy it. I think its totally real, but we just have to be careful how we characterize it.

When a woman is having these symptoms I also really dive deep into sleep. Sleep disturbances through the perimenopause transition are like absolutely so essential at characterizing in our clinic, we have over fifty people wearing this risk biosensor and we just published on this in the journal of Prevention of Alzheimers Disease where we can use biosensor information and put it in an algorithm to predict how the brain is functioning.

Its actually pretty cool stuff but we can track sleep, and not just total sleep, its how long does it take to fall asleep, deep sleep, how much REM sleep there is, we look at cardiovascular measures like something called heart rate variability, is a woman ruminating about their symptoms, are they having repetitive negative thinking which fast forwards amyloid and tau deposition which fast forwards shrinkage of the memory center of the brain.

So in women during the perimenopause transition, its not just about the hormones, its about everything thats downstream from the sleeping disturbed by the hormones, so Im ruminating about it, is this a serotonin problem, is it an estrogen problem, we need to talk about pharmacological and non-pharmacological approaches, sleep hygiene, could we take a supplement, are you exercising, are you having caffeine before bed? Theres so many different things we need to address for a precision approach.

Edited for clarity

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Dr. Richard Isaacson: The Link Between Alzheimer's and Menopause - Being Patient

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CARLSBAD, Calif., July 7, 2020 /PRNewswire/ --Qualigen, Inc.(NASDAQ: QLGN) ("Qualigen" or the "Company") announces it has engaged Pro Football Hall of Fame and College Football Hall of Fame inductee Mike Haynes as an advisor to the Company and as spokesman for Qualigen's FastPack rapid diagnostic system. After receiving an elevated PSA test result using a FastPack immunoassay test at a 2008 Hall of Fame event sponsored by Qualigen and the American Urological Association, Mr. Haynes has been a prominent advocate for prostate cancer testing.

Mr. Haynes will assist Qualigen's outreach efforts to support utilization and distribution of the FastPack system, as well as representing the Company at health events and with other sponsorship opportunities with the NFL and other U.S. professional sports leagues and organizations.

FastPack has been used worldwide for almost 20 years to assist in diagnosing many diseases, from elevated PSA levels and hormone deficiencies to other men's health conditions. Qualigen also recently announced the launch of its reliable, rapid FastPack test for antibodies against the virus that causes COVID-19, slated for initial distribution this month.

"I often tell people that Qualigen's FastPack testing system saved my life, and that is why I am thrilled to join the Qualigen team," stated Mike Haynes. "FastPack is a reliable, convenient and rapid test system that gives physicians and other point-of-care medical professionals the power of large reference laboratory testing on location for their patients. I recommend all men over the age of 55 to get a PSA test and know their family history. It's simple and an early diagnosis could save their lives too."

Mike Haynes starred for the New England Patriots and Los Angeles Raiders from 1976-1989. Considered by some to be the greatest cornerback of all time, he was named to the NFL's 100th Anniversary and 75th Anniversary All-Time Teams. In order to encourage men to know their prostate cancer risk and to get tested, Mr. Haynes has been a spokesperson for the Know Your Stats About Prostate Cancer campaign, a combined effort between the NFL and the Urology Care Foundation, the official foundation of the American Urological Association.

"I've known Mike for many years and strongly support his message to inform men about the need to get tested for prostate cancer. We welcome Mike to the Qualigen team and look forward to advancing the FastPack message together," stated Michael Poirier, President and Chief Executive Officer of Qualigen.

About the FastPack SystemThe FastPack System is a rapid and highly accurate immunoassay testing system consisting of the FastPack Analyzer and the FastPack test pouch (a single-use, disposable, foil packet that includes the FastPack reagent chemistry). This "Laboratory in a Pouch" is installed in physician offices, clinics and small hospitals around the world, and quickly detects diseases and medical conditions at the point-of-care. Since the conception of the system, the Company has expanded its assay menu to 10 tests including tests for prostate cancer, thyroid function, metabolic disorders, antibodies against SARS-CoV-2, and research applications. Over the past 20 years, FastPack has generated more than $100 million in commercial sales. Qualigen's worldwide distributor for FastPack is Sekisui Diagnostics, LLC, a subsidiary of a multibillion-dollar Japanese chemical and technology company; Sekisui, in turn, works with national distributors including McKesson Corporation and Henry Schein Inc.

About Qualigen Therapeutics, Inc.Qualigen Therapeutics, Inc. is a biotechnology company focused on developing novel therapeutics for the treatment of cancer and infectious diseases, as well as maintaining and expanding its core FDA-approved FastPack System, which has been used successfully in diagnostics for almost 20 years. The FastPack menu includes tests for cancer, men's health, hormone function, vitamin D status and antibodies against SARS-CoV-2. The Company's cancer therapeutics pipeline includes ALAN (AS1411-GNP), RAS-F and STARS. ALAN (AS1411-GNP) is a DNA coated gold nanoparticle cancer drug candidate that has the potential to target various types of cancer with minimal side effects. The foundational aptamer of ALAN, AS1411, is also being studied for use in treating viral-based infectious diseases, including COVID-19. RAS-F is a small molecule RAS oncogene protein-protein inhibitor for blocking RAS mutations that lead to tumor formation, especially in pancreatic, colorectal and lung cancers. STARS is a DNA/RNA-based treatment device for removal from circulating blood of precisely targeted tumor-produced and viral compounds. Qualigen's facility in Carlsbad, California is FDA and ISO Certified and its FastPack product line is sold worldwide by its commercial partner Sekisui Diagnostics, LLC. For more information on Qualigen Therapeutics, Inc., please visit https://www.qualigeninc.com/.

Qualigen Forward-Looking StatementsThis news release contains forward-looking statements by the Company that involve risks and uncertainties and reflect the Company's judgment as of the date of this release. These statements include those related to potential future development, testing and launch of product candidates. Actual events or results may differ from our expectations. For example, there can be no assurance that advisor and advocacy efforts will improve FastPack sales; that adoption and placement of FastPack Pro System instruments (which are the only FastPack instruments on which the Company's SARS-CoV-2 IgGtest kits can be run) will be widespread; that the Company will be able to manufacture the FastPack Pro System instruments and SARS-CoV-2 IgGtest kits successfully; that any commercialization of the FastPack Pro System instruments and SARS-CoV-2 IgGtest kits will be profitable; that the Company will successfully develop any drugs or therapeutic devices; that preclinical or clinical development of the Company's drugs or therapeutic devices will be successful; that future clinical trial data will be favorable or that such trials will confirm any improvements over other products or lack negative impacts; that any drugs or therapeutic devices will receive required regulatory approvals or that they will be commercially successful; that the Company will be able to procure or earn sufficient working capital to complete the development, testing and launch of our prospective therapeutic products; or that we will be able to maintain or expand market demand and/or market share for our diagnostic products. Our stock price could be harmed if any of the events or trends contemplated by the forward-looking statements fails to occur or is delayed or if any actual future event otherwise differs from expectations. Additional information concerning these and other risk factors affecting the Company's business (including events beyond the Company's control, such as epidemics and resulting changes) can be found in the Company's prior filings with the Securities and Exchange Commission, available at http://www.sec.gov. The Company disclaims any intent or obligation to update these forward-looking statements beyond the date of this news release, except as required by law. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

View original content:http://www.prnewswire.com/news-releases/qualigen-therapeutics-engages-nfl-hall-of-famer-mike-haynes-as-advisor-to-the-company-and-spokesman-for-the-fastpack-rapid-diagnostic-system-which-saved-my-life-301088889.html

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Qualigen Therapeutics Engages NFL Hall of Famer Mike Haynes as Advisor to the Company and Spokesman for the FastPack Rapid Diagnostic System...

Recommendation and review posted by Bethany Smith

Dear Doctor: Our dad was diagnosed with lung cancer and will be getting chemo and radiation. Im not really sure how they work, or why he has to have both. Should we get a second opinion?

Dear Reader: We use the word cancer to refer to a group of diseases in which abnormal cells within the body begin to divide uncontrollably. They are able to evade the bodys natural defenses, and, over time, they can spread throughout the body. The goal of cancer treatment is to prevent these rogue cells from dividing. This is achieved either by killing the cancer cells outright, or disrupting their DNA, and thus their ability to divide. Radiation and chemotherapy are two of the most common cancer treatments. Although each works to stop the progress of the disease, they act in different ways.

Chemotherapy is a systemic treatment. That means that the specialized drugs used in chemotherapy are administered via the bloodstream so that they can reach and act upon cells throughout the body. More than 100 different chemotherapy medications fall into five major categories. Each of these drug categories behave in a different way, but all share the same objective. That is, to disrupt the life cycle of cells within the body that divide rapidly, which includes cancer cells.

Chemotherapy drugs are quite powerful. Because they circulate to all parts of the body, they act on all of the rapidly dividing cells in the body, including those that are not cancer. This includes hair roots, which is why hair loss occurs in many cancer patients undergoing chemo. Other common side effects include nausea, vomiting, fatigue, anemia and infection.

Radiation therapy, by contrast, is a localized treatment. It targets a specific part of the body where cancer cells are found. The powerful energy delivered via X-rays, gamma rays, electron beams or protons destroys cancer cells and damages their DNA as well. This prevents the rogue cells from dividing and growing, and thus stops their spread.

Some radiation treatments involve ingesting, injecting or implanting a radioactive substance, which either locates or binds to cancer cells. Although the effect on noncancerous cells isnt as severe in radiation therapy as it is in chemotherapy, healthy cells do get damaged. This results in side effects such as skin problems at the delivery site and fatigue.

Depending on the type of cancer and how far it has spread, its not unusual for several treatments to be used together. This includes chemotherapy and radiation therapy, as well as surgery, immunotherapy, hormone therapy and targeted drug therapies.

In certain types of lung cancer, studies have found that using chemotherapy along with radiation therapy can improve survival rates. While radiation is effective at targeting solid tumors, chemotherapy can act upon cancer cells throughout the body, including those that havent yet been identified.

Lung cancer treatment is a rapidly advancing field. For that reason, its often helpful for patients to have their case and treatment plan reviewed by a physician or medical team that specializes in the disease. To avoid unexpected costs, check with your insurer for guidance on which doctors and facilities are covered by your plan.

Eve Glazier, M.D., MBA, is an internist and associate professor of medicine at UCLA Health. Elizabeth Ko, M.D., is an internist and assistant professor of medicine at UCLA Health.

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Ask the Doctors: Many cancers require more than one kind of treatment - Eureka Times-Standard

Recommendation and review posted by Bethany Smith

Cushing disease is caused by tumour in the pituitary gland which leads to excessive secretion of a hormone called adrenocorticotrophic (ACTH), which in turn leads to increasing levels of cortisol in the body. Cortisol is a steroid hormone released by the adrenal glands and helps the body to deal with injury or infection. Increasing levels of cortisol increases the blood sugar and can even cause diabetes mellitus. However the disease is also caused due to excess production of hypothalamus corticotropin releasing hormone (CRH) which stimulates the synthesis of cortisol by the adrenal glands. The condition is named after Harvey Cushing, the doctor who first identified the disease in 1912. Cushing disease results in Cushing syndrome. Cushing syndrome is a group of signs and symptoms developed due to prolonged exposure to cortisol. Signs and symptoms of Cushing syndrome includes hypertension, abdominal obesity, muscle weakness, headache, fragile skin, acne, thin arms and legs, red stretch marks on stomach, fluid retention or swelling, excess body and facial hair, weight gain, acne, buffalo hump, tiredness, fatigue, brittle bones, low back pain, moon shaped face etc. Symptoms vary from individual to individual depending upon the disease duration, age and gender of the patient. Disease diagnosis is done by measuring levels of cortisol in patients urine, saliva or blood. For confirming the diagnosis, a blood test for ACTH is performed. The first-line treatment of the disease is through surgical resection of ACTH-secreting pituitary adenoma, however disease management is also done through medications, Cushing disease treatment market comprises of the drugs designed for lowering the level of cortisol in the body. Thus patients suffering from Cushing disease are prescribed medications such as ketoconazole, mitotane, aminoglutethimide metyrapone, mifepristone, etomidate and pasireotide.

Cushings disease treatment market revenue is growing with a stable growth rate, this is attributed to increasing number of pipeline drugs. Also increasing interest of pharmaceutical companies to develop Cushing disease drugs is a major factor contributing to the revenue growth of Cushing disease treatment market over the forecast period. Current and emerging players focuses on physician education and awareness regarding availability of different drugs for curing Cushing disease, thus increasing the referral speeds, time to diagnosis and volume of diagnosed Cushing disease individuals. Growing healthcare expenditure and increasing awareness regarding Cushing syndrome aids in the revenue growth of Cushings disease treatment market. Increasing number of new product launches also drives the market for Cushings disease Treatment devices. However availability of alternative therapies for curing Cushing syndrome is expected to hamper the growth of the Cushings disease treatment market over the forecast period.

The Cushings disease Treatment market is segment based on the product type, technology type and end user

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Cushings disease Treatment market is segmented into following types:

By Drug Type

By End User

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Cushings disease treatment market revenue is expected to grow at a good growth rate, over the forecast period. The market is anticipated to perform well in the near future due to increasing awareness regarding the condition. Also the market is anticipated to grow with a fastest CAGR over the forecast period, attributed to increasing investment in R&D and increasing number of new product launches which is estimated to drive the revenue growth of Cushings disease treatment market over the forecast period.

Depending on geographic region, the Cushings disease treatment market is segmented into five key regions: North America, Latin America, Europe, Asia Pacific (APAC) and Middle East & Africa (MEA).

North America is occupying the largest regional market share in the global Cushings disease treatment market owing to the presence of more number of market players, high awareness levels regarding Cushing syndrome. Healthcare expenditure and relatively larger number of R&D exercises pertaining to drug manufacturing and marketing activities in the region. Also Europe is expected to perform well in the near future due to increasing prevalence of the condition in the region.

Asia Pacific is expected to grow at the fastest CAGR because of increase in the number of people showing the symptoms of Cushing syndrome, thus boosting the market growth of Cushings disease treatment market throughout the forecast period.

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Some players of Cushings disease Treatment market includes CORCEPT THERAPEUTICS, HRA Pharma, Strongbridge Biopharma plc, Novartis AG, etc. However there are numerous companies producing branded generics for Cushing disease. The companies in Cushings disease treatment market are increasingly engaged in strategic partnerships, collaborations and promotional activities to capture a greater pie of market share.

The research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to categories such as market segments, geographies, types, technology and applications.

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Global Cushing's Disease Treatment Market to Witness Rapid Development During the Period 2017 2025 - Jewish Life News

Recommendation and review posted by Bethany Smith

By William Petri, The Conversation

I am a physician and a scientist at the University of Virginia. I care for patients and conduct research to find better ways to diagnose and treat infectious diseases, including COVID-19. Here Im sharing what is known about which treatments work, and which dont, for the new coronavirus infection.

Keep in mind that this field of medicine is rapidly evolving as our understanding of the SARS-CoV-2 virus improves. So what I am writing today may change within days or weeks.

Below are the treatments that have been tried and for which we have the best knowledge.

There are three randomized controlled trials of hydroxychloroquine, all of which have failed to prove or disprove a beneficial or harmful effect on COVID-19 clinical course or clearance of virus. Given this current lack of evidence, these drugs, which normally are used to treat arthritis, should only be used within the context of a controlled clinical trial.

The drug lopinavir is an inhibitor of an enzyme called HIV protease which is involved in the production of viral particles. Protease inhibitors for HIV were revolutionary, leading to our current ability to effectively treat HIV. Lopinavir also can inhibit enzymes that perform similar functions as the HIV protease in the SARS and MERS coronaviruses. Ritonavir increases the level of lopinavir in the blood so the lopinavir/ritonavir combination was tested in a randomized controlled clinical trial for COVID-19.

Unfortunately, there was no impact on the levels of virus in the throat or duration of viral shedding, nor did patients clinical course or survival change. There therefore is no role for lopinavir/ritonavir in the treatment of COVID-19.

When a synthetic steroid hormone, called dexamethasone, was given to patients with COVID-19 the drug decreased 28-day mortality by 17 percent and hastened hospital discharge.

This work was performed in a randomized and controlled clinical trial of over 6,000 patients, and while not replicated in another study or yet peer reviewed, is certainly enough evidence to recommend its use.

Tocilizumab is an antibody, that blocks a protein, called IL-6 receptor, from binding IL-6 and triggering inflammation. Levels of IL-6 are higher in many patients with COVID-19, and the immune system in general seems to be hyper-activated in those with the most severe disease. This leads many physicians and physicians to think that inhibiting the IL-6 receptor might protect patients from severe disease.

Tocilizumab is currently FDA approved for the treatment of rheumatoid arthritis and several other collagen-vascular diseases and for cytokine storma harmful overreaction of the immune system that can be caused by certain types of cancer therapy and COVID-19.

A retrospective observational study found that COVID-19 patients treated with tocilizumab had a lower risk of mechanical ventilation and death. But we lack a randomized controlled clinical trial so there is no way to ascertain if this apparent improvement was due to tocilizumab or from the imprecise nature of retrospective studies.

Convalescent plasma, the liquid derived from blood after removing the white and red blood cells, contains antibodies from previous infections that the plasma donor had. This plasma has been used to prevent infectious diseases including pneumonia, tetanus, diphtheria, mumps and chickenpox for over a century. It is thought to benefit patients because antibodies from the plasma of survivors bind to and inactivate pathogens or their toxins of patients. Convalescent plasma has now been used in thousands of COVID-19 patients.

However, the only randomized clinical trial was small and included just 103 patients who received convalescent plasma 14 days after they became ill. There was no difference in the time to clinical improvement or mortality between those who did and did not receive treatment. The encouraging news was that there was a significant decrease in virus levels detected by PCR.

It is therefore too early to tell if this will be beneficial and controlled clinical trials are needed.

A nurse is collecting convalescent plasma from a recovered COVID-19 patient to help the healing process of other COVID-19 patients in Indonesia. Budiono,/ Sijori images/Barcroft Media via Getty Images

Remdesivir is a drug that inhibits the coronavirus enzyme that makes copies of the viral RNA genome. It acts by causing premature stoppage or termination of the copying and ultimately blocks the virus from replicating.

Remdesivir treatment, especially for patients who required supplemental oxygen before they were placed on a ventilator reduced mortality and shortened the average recovery timefrom 15 to 11 days.

There was a concern that drugs called ACE inhibitors or angiotensin receptor blockers (ARBs), which are used to treat high blood pressure and heart failure, could increase levels of the ACE2 proteins, the receptor for SARS-CoV-2, on the surface of cells in the body. This would, physicians hypothesized, allow more entry points for the virus to infect cells and would therefore boost the severity of new coronavirus infections.

However, there is no evidence that this is the case. The American Heart Association, the Heart Failure Society of America and the American College of Cardiology all recommend that patients continue to take these medications during the pandemic as they are beneficial in the treatment of high blood pressure and heart failure.

We have made amazing progress in the treatment of COVID-19. Two therapiessteroids and Remdesivirhave already been shown to help. Those who benefit from these treatments owe thanks to patients who volunteered to participate in controlled clinical trials, and the physicians and pharmaceutical companies that lead them.

William Petri is a professor of medicine at the University of Virginia

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Surveying COVID-19 Treatments From Hydroxychloroquine and Remdesivir to Steroids and Plasma - The Daily Beast

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DetailsCategory: AntibodiesPublished on Monday, 06 July 2020 14:45Hits: 328

Trodelvy significantly improved progression-free survival (PFS) and overall survival (OS) in previously-treated brain metastasis negative patients with advanced mTNBC

The safety profile of Trodelvy was consistent with the FDA-approved label and no new safety signals were observed

Trodelvy is the first antibody-drug conjugate (ADC) shown to improve clinical outcomes in people with relapsed or refractory mTNBC who have received two prior therapies

sBLA submission seeking full approval planned for later this year

MORRIS PLAINS, NJ, USA I July 06, 2020 IImmunomedics, Inc. (NASDAQ: IMMU) (Immunomedics or the Company), a leading biopharmaceutical company in the area of antibody-drug conjugates, today announced that the confirmatory Phase 3 ASCENT study of Trodelvy (sacituzumab govitecan-hziy) met its primary endpoint of progression-free survival (PFS), as well as key secondary endpoints in brain metastasis negative patients with mTNBC who have previously received at least two prior therapies for metastatic disease.

The results of the global Phase 3 ASCENT study confirm our initial observations that sacituzumab govitecan has the potential to change the standard management of mTNBC. Based on these data, sacituzumab govitecan has set a new benchmark in scientific and clinical innovation for patients with mTNBC by offering a novel alternative to the common drugs currently in clinical practice. Importantly, the ASCENT topline data also validate the manageable safety profile of sacituzumab govitecan, rendering it a good partner candidate for combination with other therapies, including immunotherapy, stated the study principal investigator, Aditya Bardia, MD, MPH, Director of Precision Medicine at the Center for Breast Cancer, Mass General Cancer Center and Assistant Professor of Medicine at Harvard Medical School.

In the ASCENT study, Trodelvy demonstrated a statistically significant improvement in the primary endpoint of PFS compared to chemotherapy, with a hazard ratio of 0.41 (95% confidence interval (CI), 0.32-0.52). The median PFS for patients treated with Trodelvy was 5.6 months (95% CI, 4.3-6.3), compared to 1.7 months (95% CI, 1.5-2.6) for chemotherapy (p<0.0001). Trodelvy also met key secondary endpoints of the study, including overall survival and objective response rate. The safety profile of Trodelvy observed in the ASCENT study remained consistent with the U.S. Food and Drug Administration (FDA)-approved label, with neutropenia and diarrhea as the most common Grade 3 or 4 adverse events and no new safety signals were observed. Full results will be presented at an upcoming medical conference.

Given the poor prognosis associated with mTNBC, we are excited that Trodelvy demonstrated improved clinical outcomes for these patients, said Dr. Loretta M. Itri, Chief Medical Officer of Immunomedics. We are grateful to all the patients, their families and healthcare providers, as well as our colleagues at Immunomedics, who participated in the ASCENT study and overcame many obstacles, including the COVID-19 pandemic, to complete the collection and analyses of these important data. We look forward to presenting full results at an upcoming medical conference, as well as sharing them with the FDA in support of the full approval of Trodelvy in this difficult-to-treat cancer.

Trodelvy was recently approved by the FDA as a third-line treatment for adult patients with mTNBC under the agencys Accelerated Approval Program based on the objective response rate and duration of response observed in a single-arm, multicenter Phase 2 study. The Phase 3 confirmatory ASCENT study was designed under an FDA Special Protocol Assessment (SPA) to validate the promising safety and efficacy activity of Trodelvy that supported its accelerated approval.

It is gratifying to see the final confirmatory results of Trodelvy in a randomized study supporting the previously reported Phase 2 data that formed the basis of the accelerated approval of Trodelvy. These results provide important additional information for patients and their treating physicians to understand the definitive benefits they are deriving, commented Dr. Behzad Aghazadeh, Executive Chairman of Immunomedics. Importantly, the strong ASCENT data reinforce the promise of our unique ADC technology and embolden us to continue our work to change the treatment paradigm for patients with difficult-to-treat cancers.

Trodelvy carries a black box warning for severe neutropenia and severe diarrhea. The most common adverse reactions occurring in 25 or more percent of patients included nausea, neutropenia, diarrhea, fatigue, anemia, vomiting, alopecia, constipation, decreased appetite, rash and abdominal pain. The most common Grade 3 or 4 adverse events occurring in more than 5 percent of patients were neutropenia, white blood cell count decreased, anemia, hypophosphatemia, diarrhea, fatigue, nausea and vomiting. Two percent of patients discontinued treatment due to adverse events. There were no deaths related to treatment and no severe cases of neuropathy or interstitial lung disease.1

Conference Call

The Company will host a conference call and live audio webcast today at 8:00 a.m. Eastern Time to discuss the ASCENT data. To access the conference call, please dial (877) 303-2523 or (253) 237-1755 using the Conference ID 8872919. The conference call will be webcast via the Investors page on the Companys website at https://immunomedics.com/investors/. Approximately two hours following the live event, a webcast replay of the conference call will be available on the Companys website for approximately 30 days.

About ASCENT

The international, open-label confirmatory Phase 3 study enrolled more than 500 patients with metastatic triple-negative breast cancer who had received at least two prior therapies for metastatic disease. Patients were randomized to receive either Trodelvy or a physicians choice of chemotherapy. The primary endpoint of the study was progression-free survival. Secondary endpoints include overall survival, objective response rate, duration of response, time to onset of response, and other measures of safety and tolerability. More information about ASCENT is available at http://clinicaltrials.gov/show/NCT02574455.

About TRODELVY

Trodelvy (sacituzumab govitecan-hziy) is the lead product and the most advanced program in Immunomedics unique antibody-drug conjugate (ADC) platform. Trodelvy is an ADC that is directed against Trop-2, a cell-surface protein expressed in many solid cancers. Trodelvy binds to Trop-2 and delivers the anti-cancer drug, SN-38, to kill cancer cells. Immunomedics has an extensive development program for Trodelvy, including multiple ongoing studies in triple-negative breast cancer, metastatic urothelial cancer, hormone receptor-positive/human epidermal growth factor receptor 2-negative metastatic breast cancer, and metastatic non-small cell lung cancer, either as a monotherapy or in combination with other agents. Visit https://www.trodelvy.com/ for more information.

About triple-negative breast cancer (TNBC)

TNBC is an aggressive type of breast cancer, accounting for up to 20 percent of all breast cancers. The disease is diagnosed more frequently in younger and premenopausal women and is highly prevalent in African American and Hispanic women. TNBC cells do not have estrogen or progesterone hormone receptors, or very much of the human epidermal growth factor receptor 2 hence the term triple negative. This means that medicines that target these receptors are not typically effective in TNBC. There is currently no approved standard of care for people with previously-treated mTNBC.

About Immunomedics

Immunomedics is a leader in next-generation antibody-drug conjugate (ADC) technology, committed to help transform the lives of people with hard-to-treat cancers. Our proprietary ADC platform centers on using a novel linker that does not require an enzyme to release the payload to deliver an active drug inside the tumor cell and the tumor microenvironment, thereby producing a bystander effect. Trodelvy, our lead ADC, is the first ADC the FDA has approved for the treatment of people with metastatic triple-negative breast cancer and is also the first FDA-approved anti-Trop-2 ADC. For additional information on the Company, please visit its website at https://immunomedics.com/. The information on its website does not, however, form a part of this press release.

Reference

1. TRODELVY Prescribing Information. Morris Plains, NJ: Immunomedics Inc, 2020.

SOURCE: Immunomedics

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Immunomedics Announces Positive Results from Phase 3 ASCENT Study of TRODELVY in Previously-Treated Patients with Metastatic Triple-Negative Breast...

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