StemCell Therapy

April 28 (Reuters) - Novartis AG:

* NOVARTIS CEO SAYS CORONAVIRUS IMPACTS ARE GREATEST ON NEW CLINICAL STUDY STARTS, SAYS MANAGING SITUATION VIA DIGITAL TECHNOLOGY

* NOVARTIS CEO SAYS INCREASING REMOTE MONITORING OF PATIENTS IN CLINICAL STUDIES

* NOVARTIS CEO SAYS REGULATORS HAVE COMMITTED TO MAINTAINING TIMELINES FOR NEW MEDICINES

* NOVARTIS CEO SAYS BEOVU WAS OFF TO OUTSTANDING LAUNCH, HAS IDENTIFIED RARE SAFETY SIGNAL FOR RETINAL VASCULITIS, STUDYING THE REASON FOR SAFETY SIGNAL, CONTINUES TO BELIEVE MEDICINE WILL EVENTUALLY EXCEED $1 BILLION IN SALES

* NOVARTIS CEO SAYS GUIDANCE ASSUMES HEALTH CARE SYSTEMS WILL RESUME NORMAL OPERATIONS IN Q2

* NOVARTIS CEO SAYS HAS PAUSED ENROLLMENT IN UK INCLISIRAN TRIAL DUE TO CORONAVIRUS PANDEMIC, HOPEFUL TO RESTART IN COMING QUARTERS

* NOVARTIS CEO SAYS SEES SOME SLOWDOWN IN HOSPITAL VISITS IMPACTING LUTATHERA, KYMRIAH, ZOLGENSMA, SAYS DOING BEST TO REACH OUT TO PATIENTS WHO NEED GENE THERAPY

* NOVARTIS CEO SAYS DIFFICULT TO PREDICT HOW PANDEMIC WILL UNFOLD IN COMING YEAR, AS THINGS EVOLVE WILL UPDATE GUIDANCE AS NEEDED

* NOVARTIS CEO SAYS FEW OF STUDIES SO FAR ON THERAPIES FOR CORONAVIRUS HAVE MET GOLD STANDARD OF RANDOMIZED, BLINDED CLINICAL STUDIES, STILL NEED PROPERLY POWERED BLINDED STUDIES TO REALLY DETERMINE MEDICINES HAVE BENEFIT Further company coverage: (Reporting by John Miller)

Go here to see the original:
BRIEF-Novartis CEO Says Still Need Proper Blinded Studies To Determine Which Drugs Have Benefit for COVID-19 - Reuters UK

The research study on Global Personalized Gene Therapy Treatment market 2019 presents an extensive analysis of current Personalized Gene Therapy Treatment market size, drivers, trends, opportunities, challenges, as well as key Personalized Gene Therapy Treatment market segments. Further, it explains various definitions and classification of the Personalized Gene Therapy Treatment industry, applications, and chain structure.In continuation of this data, the Personalized Gene Therapy Treatment report covers various marketing strategies followed by key players and distributors. Also explains Personalized Gene Therapy Treatment marketing channels, potential buyers and development history. The intent of global Personalized Gene Therapy Treatment research report is to depict the information to the user regarding Personalized Gene Therapy Treatment market forecast and dynamics for the upcoming years. The Personalized Gene Therapy Treatment study lists the essential elements which influence the growth of Personalized Gene Therapy Treatment industry. Long-term evaluation of the worldwide Personalized Gene Therapy Treatment market share from diverse countries and regions is roofed within the Personalized Gene Therapy Treatment report. Additionally, includes Personalized Gene Therapy Treatment type wise and application wise consumption figures.

Download Sample of This Strategic Report:https://www.kennethresearch.com/sample-request-10225585

After the basic information, the global Personalized Gene Therapy Treatment Market study sheds light on the Personalized Gene Therapy Treatment technological evolution, tie-ups, acquisition, innovative Personalized Gene Therapy Treatment business approach, new launches and Personalized Gene Therapy Treatment revenue. In addition, the Personalized Gene Therapy Treatment industry growth in distinct regions and Personalized Gene Therapy Treatment R;D status are enclosed within the report.The Personalized Gene Therapy Treatment study also incorporates new investment feasibility analysis of Personalized Gene Therapy Treatment. Together with strategically analyzing the key micro markets, the report also focuses on industry-specific drivers, restraints, opportunities, and challenges in the Personalized Gene Therapy Treatment market.

Global Personalized Gene Therapy Treatment Market Segmentation 2019: Personalized Gene Therapy TreatmentThe study also classifies the entire Personalized Gene Therapy Treatment market on basis of leading manufacturers, different types, various applications and diverse geographical regions. Overall Personalized Gene Therapy Treatment market is characterized by the existence of well-known global and regional Personalized Gene Therapy Treatment vendors. These established Personalized Gene Therapy Treatment players have huge essential resources and funds for Personalized Gene Therapy Treatment research as well as developmental activities. Also, the Personalized Gene Therapy Treatment manufacturers focusing on the development of new Personalized Gene Therapy Treatment technologies and feedstock. In fact, this will enhance the competitive scenario of the Personalized Gene Therapy Treatment industry.

The Leading Players involved in global Personalized Gene Therapy Treatment market are:

By Therapy (Targeted Treatments and Pharmacogenomics),By Application (Breast Cancer, Brain Cancer, Colorectal Cancer, Certain Childhood Cancers, Gastrointestinal Stromal Tumor, Kidney Cancer, Leukemia, Lymphoma, Melanoma, Lung Cancer, and Multiple Myeloma)By Region (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)

Download Sample of This Strategic Report:https://www.kennethresearch.com/sample-request-10225585

Worldwide Personalized Gene Therapy Treatment Market Different Analysis:Competitors Review of Personalized Gene Therapy Treatment Market: Report presents the competitive landscape scenario seen among top Personalized Gene Therapy Treatment players, their company profile, revenue, sales, business tactics and forecast Personalized Gene Therapy Treatment industry situations. Production Review of Personalized Gene Therapy Treatment Market: It illustrates the production volume, capacity with respect to major Personalized Gene Therapy Treatment regions, application, type, and the price. Sales Margin and Revenue Accumulation Review of Personalized Gene Therapy Treatment Market: Eventually explains sales margin and revenue accumulation based on key regions, price, revenue, and Personalized Gene Therapy Treatment target consumer. Supply and Demand Review of Personalized Gene Therapy Treatment Market: Coupled with sales margin, the report depicts the supply and demand seen in major regions, among key players and for every Personalized Gene Therapy Treatment product type. Also interprets the Personalized Gene Therapy Treatment import/export scenario. Other key reviews of Personalized Gene Therapy Treatment Market: Apart from the above information, correspondingly covers the company website, number of employees, contact details of major Personalized Gene Therapy Treatment players, potential consumers and suppliers. Also, the strengths, opportunities, Personalized Gene Therapy Treatment market driving forces and market restraints are studied in this report.

Highlights of Global Personalized Gene Therapy Treatment Market Report:* This report provides in detail analysis of the Personalized Gene Therapy Treatment and provides market size (US$ Million) and Cumulative Annual Growth Rate (CAGR (%)) for the forecast period: 2019 ; 2029. * It also elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for world Personalized Gene Therapy Treatment market. * This study also provides key insights about Personalized Gene Therapy Treatment market drivers, restraints, opportunities, new product launches, approvals, regional outlook, and competitive strategies adopted by the leading Personalized Gene Therapy Treatment players. * It profiles leading players in the worldwide Personalized Gene Therapy Treatment market based on the following parameters ; company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies and future plans. * Insights from Personalized Gene Therapy Treatment report would allow marketers and management authorities of companies to make an informed decision with respect to their future product launches, market expansion, and Personalized Gene Therapy Treatment marketing tactics. * The world Personalized Gene Therapy Treatment industry report caters to various stakeholders in Personalized Gene Therapy Treatment market. That includes investors, device manufacturers, distributors and suppliers for Personalized Gene Therapy Treatment equipment. Especially incorporates government organizations, Personalized Gene Therapy Treatment research and consulting firms, new entrants, and financial analysts. *Various strategy matrices used in analyzing the Personalized Gene Therapy Treatment market would provide stakeholders vital inputs to make strategic decisions accordingly.

Global Personalized Gene Therapy Treatment Market Report Provides Comprehensive Analysis of Following: ; Personalized Gene Therapy Treatment Market segments and sub-segments ; Industry size ; Personalized Gene Therapy Treatment shares ; Personalized Gene Therapy Treatment Market trends and dynamics ; Market Drivers and Personalized Gene Therapy Treatment Opportunities ; Supply and demand of world Personalized Gene Therapy Treatment industry ; Technological inventions in Personalized Gene Therapy Treatment trade ; Personalized Gene Therapy Treatment Marketing Channel Development Trend ; Global Personalized Gene Therapy Treatment Industry Positioning ; Pricing and Brand Strategy ; Distributors/Traders List enclosed in Positioning Personalized Gene Therapy Treatment Market.

Request For Full Report:https://www.kennethresearch.com/sample-request-10225585

Moreover, the report organizes to provide essential information on current and future Personalized Gene Therapy Treatment market movements, organizational needs and Personalized Gene Therapy Treatment industrial innovations. Additionally, the complete Personalized Gene Therapy Treatment report helps the new aspirants to inspect the forthcoming opportunities in the Personalized Gene Therapy Treatment industry. Investors will get a clear idea of the dominant Personalized Gene Therapy Treatment players and their future forecasts.

About Kenneth Research:

Kenneth Research provides market research reports to different individuals, industries, associations and organizations with an aim of helping them to take prominent decisions. Our research library comprises of more than 10,000 research reports provided by more than 15 market research publishers across different industries. Our collection of market research solutions covers both macro level as well as micro level categories with relevant and suitable market research titles. As a global market research reselling firm, Kenneth Research provides significant analysis on various markets with pure business intelligence and consulting services on different industries across the globe. In addition to that, our internal research team always keep a track on the international and domestic market for any economic changes impacting the products demand, growth and opportunities for new and existing players.

Contact Us

Kenneth ResearchEmail: [emailprotected]Phone: +1 313 462 0609

More:
Personalized Gene Therapy Treatment Market Analysis By Industry Size, Share, Revenue Growth, Development And Demand Forecast To 2029 - Germany English...

Bluebird Bio

Global Gene Therapy For CNS Disorders Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

To get Incredible Discounts on this Premium Report, Click Here @ https://www.marketresearchintellect.com/ask-for-discount/?rid=175792&utm_source=LHN&utm_medium=888

Gene Therapy For CNS Disorders Market Region Coverage (Regional Production, Demand & Forecast by Countries etc.):

North America (U.S., Canada, Mexico)

Europe (Germany, U.K., France, Italy, Russia, Spain etc.)

Asia-Pacific (China, India, Japan, Southeast Asia etc.)

South America (Brazil, Argentina etc.)

Middle East & Africa (Saudi Arabia, South Africa etc.)

Some Notable Report Offerings:

-> We will give you an assessment of the extent to which the market acquire commercial characteristics along with examples or instances of information that helps your assessment.

-> We will also support to identify standard/customary terms and conditions such as discounts, warranties, inspection, buyer financing, and acceptance for the Gene Therapy For CNS Disorders industry.

-> We will further help you in finding any price ranges, pricing issues, and determination of price fluctuation of products in Gene Therapy For CNS Disorders industry.

-> Furthermore, we will help you to identify any crucial trends to predict Gene Therapy For CNS Disorders market growth rate up to 2026.

-> Lastly, the analyzed report will predict the general tendency for supply and demand in the Gene Therapy For CNS Disorders market.

Have Any Query? Ask Our Expert@ https://www.marketresearchintellect.com/need-customization/?rid=175792&utm_source=LHN&utm_medium=888

Table of Contents:

Study Coverage: It includes study objectives, years considered for the research study, growth rate and Gene Therapy For CNS Disorders market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Gene Therapy For CNS Disorders market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Gene Therapy For CNS Disorders Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

About Us:

Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage and more. These reports deliver an in-depth study of the market with industry analysis, market value for regions and countries and trends that are pertinent to the industry.

Contact Us:

Mr. Steven Fernandes

Market Research Intellect

New Jersey ( USA )

Tel: +1-650-781-4080

Tags: Gene Therapy For CNS Disorders Market Size, Gene Therapy For CNS Disorders Market Growth, Gene Therapy For CNS Disorders Market Forecast, Gene Therapy For CNS Disorders Market Analysis

Our Trending Reports

U.S. Pest Control Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Battery Monitoring System Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

The rest is here:
Gene Therapy For CNS Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald

Analysis Report on Gene Therapy Market

A report on global Gene Therapy market has hit stands. This study is based on different aspects like segments, growth rate, revenue, leading players, regions, and forecast. The overall market is getting bigger at an increased pace due to the invention of the new dynamism, which is making rapid progress.

The given report is an excellent research study specially compiled to provide latest insights into critical aspects of the Global Gene Therapy Market.

Request Sample Report @https://www.mrrse.com/sample/17781?source=atm

Some key points of Gene Therapy Market research report:

Strategic Developments: The custom analysis gives the key strategic developments of the market, comprising R&D, new product launch, growth rate, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

Market Features: The report comprises market features, capacity, capacity utilization rate, revenue, price, gross, production, production rate, consumption, import, export, supply, demand, cost, market share, CAGR, and gross margin. In addition, the report offers a comprehensive study of the market dynamics and their latest trends, along with market segments and sub-segments.

Analytical Tools: The Global Gene Therapy Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, feasibility study, and many other market research tools have been used to analyze the growth of the key players operating in the market.

COVID-19 Impact on Gene Therapy Market

Adapting to the recent novel COVID-19 pandemic, the impact of the COVID-19 pandemic on the global Gene Therapy market is included in the present report. The influence of the novel coronavirus pandemic on the growth of the Gene Therapy market is analyzed and depicted in the report.

The global Gene Therapy market segment by manufacturers include

segmented as follows:

Global Gene Therapy Market, by Product

Global Gene Therapy Market, by Application

Global Gene Therapy Market, by Region

Request For Discount On This Report @ https://www.mrrse.com/checkdiscount/17781?source=atm

Important queries addressed in the report:

Moreover, the report highlighted revenue, sales, manufacturing cost, and product and the States that are most competitive in the lucrative market share idea. There is a discussion on the background and financial trouble in the global Gene Therapy economic market. This included the CAGR value during the outlook period leading to 2025.

Buy This Report @ https://www.mrrse.com/checkout/17781?source=atm

Benefits of Purchasing Gene Therapy Market Report:

Inimitable Expertise: Analysts will provide deep insights into the reports.

Analyst Support: Get your query resolved from our team before and after purchasing the report.

Customers Satisfaction: Our team will assist with all your research needs and customize the report.

Assured Quality: We focus on the quality and accuracy of the report.

Conclusively, this report will provide you a clear view of each and every fact of the market without a need to refer to any other research report or a data source. Our report will provide you with all the facts about the past, present, and future of the concerned Market.

Read more here:
COVID-19 impact: Gene Therapy Market 2019 Business Insights,Top Companies,Growth, Market Size Global Market Share, Trends, Outlook, Opportunity and...

Spark Therapeutics Inc.

Global Gene Therapy For Inherited Genetic Disorders Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

To get Incredible Discounts on this Premium Report, Click Here @ https://www.marketresearchintellect.com/ask-for-discount/?rid=170144&utm_source=LHN&utm_medium=888

Gene Therapy For Inherited Genetic Disorders Market Region Coverage (Regional Production, Demand & Forecast by Countries etc.):

North America (U.S., Canada, Mexico)

Europe (Germany, U.K., France, Italy, Russia, Spain etc.)

Asia-Pacific (China, India, Japan, Southeast Asia etc.)

South America (Brazil, Argentina etc.)

Middle East & Africa (Saudi Arabia, South Africa etc.)

Some Notable Report Offerings:

-> We will give you an assessment of the extent to which the market acquire commercial characteristics along with examples or instances of information that helps your assessment.

-> We will also support to identify standard/customary terms and conditions such as discounts, warranties, inspection, buyer financing, and acceptance for the Gene Therapy For Inherited Genetic Disorders industry.

-> We will further help you in finding any price ranges, pricing issues, and determination of price fluctuation of products in Gene Therapy For Inherited Genetic Disorders industry.

-> Furthermore, we will help you to identify any crucial trends to predict Gene Therapy For Inherited Genetic Disorders market growth rate up to 2026.

-> Lastly, the analyzed report will predict the general tendency for supply and demand in the Gene Therapy For Inherited Genetic Disorders market.

Have Any Query? Ask Our Expert@ https://www.marketresearchintellect.com/need-customization/?rid=170144&utm_source=LHN&utm_medium=888

Table of Contents:

Study Coverage: It includes study objectives, years considered for the research study, growth rate and Gene Therapy For Inherited Genetic Disorders market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Gene Therapy For Inherited Genetic Disorders market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Gene Therapy For Inherited Genetic Disorders Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

About Us:

Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage and more. These reports deliver an in-depth study of the market with industry analysis, market value for regions and countries and trends that are pertinent to the industry.

Contact Us:

Mr. Steven Fernandes

Market Research Intellect

New Jersey ( USA )

Tel: +1-650-781-4080

Tags: Gene Therapy For Inherited Genetic Disorders Market Size, Gene Therapy For Inherited Genetic Disorders Market Growth, Gene Therapy For Inherited Genetic Disorders Market Forecast, Gene Therapy For Inherited Genetic Disorders Market Analysis

Our Trending Reports

Catalyst Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Diabetes Drugs Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Read more from the original source:
Gene Therapy For Inherited Genetic Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald

Plasmid DNA Manufacturing Market research report is the professional study with the premium insights which includes the size of the business, the ongoing patterns, drivers, dangers, conceivable outcomes and primary segments. The report encompasses the competition landscape entailing share analysis of the key players in the Plasmid DNA Manufacturing market based on their revenues and other significant factors. Report analyzes changing trends and competitive analysis which becomes essential to monitor performance and make critical decisions for growth and development. It also provides market information in terms of development and its capacities.

Get Sample Copy of this report @: https://www.a2zmarketresearch.com/sample?reportId=231682

Leading companies Influencing in this Market are:

Cobra Bio, Richter-Helm, Eurogentec, Cell and Gene Therapy Catapult, VGXI, PlasmidFactory, Kaneka Corporation, Nature Technology Corporation, Waisman Biomanufacturing, FUJIFILM Diosynth Biotechnologies, LakePharma.

In this Plasmid DNA Manufacturing Market research report, the prominent factors driving the advancement of this market were recorded and the business accomplices and end administrators were indulgent. The setup of the business division, examples, and challenges monitoring the market comprehensively are in like manner a bit of this wide examination. Different meetings and social events were driven by the distinguishable pioneers of this industry to get persisting and revived encounters concerned to the market.

The report gathers the essential information including the new strategies for growth of the industry and the potential players of the global Plasmid DNA Manufacturing Market. It enlists the topmost industry player dominating the market along with their contribution to the global market. The report also demonstrates the data in the form of graphs, tables, and figures along with the contacts details and sales of key market players in the global Plasmid DNA Manufacturing Market.

Global Plasmid DNA Manufacturing Market Segmentation:

Segmentation by Type:

HQ Grade Plasmid DNAGMP Grade Plasmid DNANon-GMP Grade Plasmid DNA

Segmentation by Application:

CancersInherited DisordersViral InfectionsOthers

Buy Exclusive Report @: https://www.a2zmarketresearch.com/buy?reportId=231682

Key questions answered in the report include:

Table of Contents

Global Plasmid DNA Manufacturing Market Research Report 2020 2026

Chapter 1 Plasmid DNA Manufacturing Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global 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 Market Effect Factors Analysis

Chapter 12 Global Plasmid DNA Manufacturing Market Forecast

For more Information! Ask to Experts @: https://www.a2zmarketresearch.com/enquiry?reportId=231682

If you have any special requirements, please let us know and we will offer you the report as you want.

Here is the original post:
Future Prospects of Plasmid DNA Manufacturing Market by 2026 with Top Key Players Cobra Bio, Richter-Helm, Eurogentec, Cell and Gene Therapy...

From Our Archives Medical Author: Frederick Hecht, MD, FAAP, FACMG

Gene therapy is the treatment of disease by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is responsible for the disease. Gene therapy may use the genetic material, DNA, itself as the means of treatment.

DNA or deoxyribonucleic acid is the very long molecule that encodes the genetic information. A gene is a stretch of DNA required to make a functional product such as part or all of a protein. People have about 100,000 to 150,000 genes. During gene therapy, DNA that codes for specific genes is delivered to individual cells in the body.

Most, if not all, diseases have a genetic factor. The genetic factor can be wholly or partially responsible for the disease. For example, in disorders such as cystic fibrosis, hemophilia, and muscular dystrophy, changes in a gene directly result in the condition. In other conditions such as high cholesterol and high blood pressure, genetic and environmental factors interact to cause disease. Disorders associated with aging often involve the loss of gene activity in specific types of cells. Even infections can be related to genes. In fact, they have two sets of genetic determinants: the genes of the infective agent and the genes of the person with the infection.

Uses of gene therapy

Gene therapy is being used in many ways. For example,to:

A large variety of genes are now being tested for use in gene therapy. Examples include: a gene for the treatment of cystic fibrosis (a gene called CFTR that regulates chloride); genes for factors VIII and IX, deficiency of which is responsible for classic hemophilia (hemophilia A) and another form of hemophilia (hemophilia B), respectively; genes called E1A and P53 that cause cancer cells to undergo cell death or revert to normal; AC6 gene which increases the ability of the heart to contract and may help in heart failure; and VEGF, a gene that induces the growth of new blood vessels (angiogenesis) of use in blood vessel disease.

A short synthetic piece of DNA (called an oligonucleotide) is being used by researchers to "pre-treat" veins used as grafts for heart bypass surgery. The piece of DNA seems to switch off certain genes in the grafted veins to prevent their cells from dividing and thereby prevent atherosclerosis.

Delivery of genes into cells

Gene delivery can be used in cells that have been removed from the body (ex vivo gene therapy) or in cells that are still in the body (in vivo gene therapy). Genes can be delivered into cells in different ways. The selection of a gene delivery system depends on the target cell, the duration of gene expression required for therapeutic effect, and the size of the piece of DNA to be used in the gene therapy.

Genes can be carried into cells by viruses. Viral vectors or carriers take advantage of the natural ability of a virus to enter a cell and deliver genetic material to the nucleus of the cell that contains its DNA. In developing virus carriers, the DNA coding for some or all of the normal genes of the virus to be used as a carrier are removed and replaced with a treatment gene. Most of these virus carriers are engineered so that they are able to enter cells, but they cannot reproduce themselves and so are innocuous.

Genes can also be delivered within tiny synthetic "envelopes" of fat molecules. Cell membranes contain a very high concentration of fat molecules. The fat molecule "envelope" can carry the therapeutic gene into the cell by being admitted through the cell membrane as if it were one of its own molecules.

Genes can also gain entrance into cells when an electrical charge is applied to the cell to create tiny openings in the membrane that surrounds a cells. This technique is called electroporation.

A "bionic chip"

A new "bionic chip" has been developed to help gene therapists using electroporation to slip fragments of DNA into cells. Electroporation was originally a hit-or-miss technique because there was no way to determine how much of an electrical jolt it took to open the cell membrane.

The "bionic chip" solves this problem. It contains a single living cell embedded in a tiny silicon circuit. The cell acts as a diode, or electrical gate. When it is hit with just the right charge, the cell membrane opens, allowing the electricity to pass from the top to the bottom of the bionic chip. By recording what voltage caused this phenomenon to occur, it is now posssible to determine precisely how much electricity it takes to pry open different types of cells.

Route of administration of gene therapy

The choice of route for gene therapy depends on the tissue to be treated and the mechanism by which the therapeutic gene exerts its effect. Gene therapy for cystic fibrosis, a disease which effects cells within the lung and airway, may be inhaled. Most genes designed to treat cancer are injected directly into the tumor. Proteins such as factor VIII or IX for hemophilia are also being introduced directly into target tissue (the liver).

The potential power of gene therapy

Most gene therapy for diseases such as cystic fibrosis and hemophilia has been designed only to ease, not to cure, the disease. However, the delivery of functional copies of genes provides a potential method to correct a disease at its most basic level.

Gene therapy also holds the potential to provide "patient-friendly" treatment regimens for a variety of diseases. Today, many patients with hemophilia and diabetes must have repeated injections in order to manage their disease because proteins exist in the blood stream for a limited period of time before they are degraded or eliminated. Since DNA is more stable and functions inside the cell, the delivery of genes may result in longer-term expression of the necessary proteins. SLIDESHOW Heart Disease: Causes of a Heart Attack See Slideshow

Because of its accuracy, gene therapy has the potential to eliminate cancer cells without damaging normal, healthy tissue. Furthermore, cancer gene therapies may provide alternatives when a disease does not respond to other older treatments.

The potential of gene therapy is great but, compared to its promise, the results to date are still quite limited. However, the benefits of gene therapy are believed to be on the near horizon. Gene therapy is one of the hottest areas of medical research today. (And gene therapy companies have been among the hottest in the stock market.)

The remarkable advances in genetics, including the human genome project, have opened new doors for the exploration of gene therapy. New technologies are needed to speed the progress of gene therapy. As these new technologies such as the "bionic chip" arrive, we believe that, without a doubt, gene therapy will play an increasingly important and prominent part in medicine in the decades to come.

CONTINUE SCROLLING FOR RELATED SLIDESHOW

View post:
Gene Therapy - The Future Is Here! - MedicineNet

HAMBURG, GERMANY / ACCESSWIRE / April 6, 2020 / Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) today announced that the Company has established a dedicated site for research and development of gene therapy-based projects: Evotec Gene Therapy (Evotec GT) which will start operations with a strong team of gene therapy experts at an R&D site in Orth/Donau, Austria.

Evotec GT is an integral part of Evotecs integrated drug discovery platform and complements the Companys existing expertise. This strategic addition marks an important step towards Evotecs long-term vision of becoming a fully modality-agnostic drug discovery and development partnership company.

The team in Austria have worked together for many years and applied their research within gene therapy to different gene therapy-related technologies as well as various indications. The scientists have deep expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases. Evotec GTs fully operational site will enable the Company to perform dedicated services in the field of gene therapy along the value chain of its customers from Pharma and biotech as well as foundations and academia.

Dr Werner Lanthaler, Chief Executive Officer of Evotec, commented: We are delighted to initiate our new gene therapy platform and step into this field, which perfectly fits into our business strategy going forward. In recent years, precision medicines based on cell and gene therapies have emerged and are predicted to grow significantly. Gene therapy is a promising approach in the development of genetic medicines for patients, especially for inherited and rare diseases. Finding the best candidate agnostic of modality for any given disease biology will ultimately bring forward the best medicine for patients.

Dr Friedrich Scheiflinger, EVP Head of Gene Therapy at Evotec, said: We are proud to join the growing Evotec team to add the highly promising modality of gene therapy to drug discovery projects. Our team has performed research in the field across different technologies and therapeutic areas for many years and we look forward to leveraging our expertise as part of the truly impressive, modality-agnostic Evotec platform.

About Gene Therapy

Gene therapy is a technique that modifies a persons genes to treat or prevent disease by introduction, removal or editing of genetic material, specifically DNA or RNA, within the cells of a patient. Gene therapies aim to replace a disease-causing gene with a healthy copy, inactivate a disease-causing gene, introduce a new or modified gene or interfere on an expression-regulatory level to support treatment of a disease. Through this modification of gene expression, gene therapies can increase levels of disease-fighting proteins or reduce levels of disease-causing proteins within the cell. Since direct insertion of genes into cells is still very inefficient, gene delivery is facilitated by vehicles which are most often of viral origin. The structure of these viral vectors has been modified to accommodate for the therapeutic gene and to render the vector non-infectious. Depending on the indication and the affected tissue, the technique can be either applied ex-vivo or in-vivo, i.e. with or without removing the cells from the patients body for the therapeutic procedure.

According to various analyst reports, the gene therapy market was valued at approx. $ 500 m in 2018 and the market is expected to reach > $ 5 bn by 2025 with an impressive CAGR of ~34% over the forecast period. Furthermore, rapid and significant progress in the molecular and cellular biology arena, driven by technological advancements in genomics and gene-editing tools, has contributed to an increasing number of approved gene therapies as well as an expanding pipeline. According to the Alliance for Regenerative Medicine (ARM), by the end of the second quarter of 2019, there were more than 700 clinical trials ongoing globally.[1]

ABOUT EVOTEC SE Evotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,000 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industrys need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and womens health. On this basis, Evotec has built a broad and deep pipeline of approx. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to http://www.evotec.com and follow us on Twitter @Evotec.

FORWARD LOOKING STATEMENTS Information set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

Contact Evotec SE:

Gabriele Hansen, SVP Corporate Communications, Marketing & Investor Relations, Phone: +49.(0)40.56081-255,

[1] Sources: https://www.grandviewresearch.com/industry-analysis/gene-therapy-market; https://www.marketwatch.com/press-release/at-339-cagr-gene-therapy-market-size-to-surpass-usd-518-billion-by-2025-2019-09-16; https://www.prnewswire.com/news-releases/gene-therapy-market-to-garner-6-21-bn-globally-by-2026-at-34-8-cagr-says-allied-market-research-300975194.html

SOURCE: Evotec AG via EQS Newswire

View source version on accesswire.com:

https://www.accesswire.com/583999/Evotec-Expands-into-Gene-Therapy

View post:
Evotec Expands into Gene Therapy - Associated Press

Evotec has allied with Takeda to expand into gene therapy research. The move sees Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.

Takeda acquired a gene therapy center in Orth an der Donau, Austria, through its acquisition of Shire, which picked up the site two years earlier in its takeover of Baxalta. Throughout the series of changes in ownership, which began when Baxter spun out Baxalta in 2014, a team at the site has worked on gene therapies.

Now, Evotec is set to start playing a role in those efforts. The German drug discovery shop has set up a gene therapy unit, Evotec GT, staffed by a team of more than 20 scientists in Orth an der Donau who have worked together for many years.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

Evotec disclosed news of the move into gene therapies alongside details of a multiyear partnership with Takeda. The deal, which features an undisclosed upfront fee and other payments over time, tasks Evotec with applying its new gene therapy capabilities and broader drug discovery platform to Takedas cancer, rare disease, neuroscience and gastroenterology programs.

Neither Evotec nor Takeda referred directly to a transfer of employees in the statement to disclose the deal. However, Evotec did reveal that Friedrich Scheiflinger is leading its gene therapy unit. Until recently, Scheiflinger headed up drug discovery for Takeda in Austria, with a particular focus on gene therapies. In light of Evotecs comments about its new gene therapy team having worked together for years, it is likely that other gene therapy researchers made the move from Takeda with Scheiflinger.

The agreement gives Evotec a beachhead in the fast-growing gene therapy sector. In explaining(PDF) the rationale for moving into the space, Evotec expressed a desire to be "modality agnostic" and develop wholly and co-owned candidates.

Here is the original post:
Evotec allies with Takeda to move into gene therapy R&D - FierceBiotech

German biotech giant Evotec has moved into gene therapy and partnered with Takeda to develop new therapies in oncology, rare diseases, neuroscience, and gastroenterology.

The two companies did not reveal financial details of the collaboration, but did say that it will be a multi-year alliance that adds to Evotecs existing deal with the big pharma that stretches back more than 10 years. The four therapy areas they will concentrate on align with Takedas core therapeutic focus.

The alliance with Takeda gives us a head start, Evotec CEO Werner Lanthaler told me. Takeda has strong experience in gene therapy and we are proud that they have chosen us as a partner.

Evotec also announced it will be opening a 20-person R&D site in Austria, in Orth an der Donau, to work on its gene therapies. While this will be run independently from the Takeda deal, according to Lanthaler, he said the discussions around the collaboration happened at the same time as the gene therapy site was being set up.

The site known as Evotec GT will be led by Friedrich Scheiflinger, who worked for Takeda before moving to Evotec. According to a statement from Evotec, the team has worked together for many years and as well as a focus on gene therapy techniques, has expertise in virology and blood disorders, as well as metabolic and muscle diseases.

Gene therapy has undergone a revolution in recent years and has changed dramatically since the 90s. With approvals of therapies such as the eye gene therapy Luxturna in 2018, many companies are now investing in and working to develop similar therapies.

One reason Evotec cites for moving into this area is the potential for rapid market growth. At present, there are only a few gene therapies on the market, but there are many in development. The value of the market is expected to reach 4.6B by 2025, a dramatic increase from 459M in 2018.

Evotec is also working on various other advanced therapies, including cell therapy with its EVOcells platform. The company announced last week that it made a licensing and investment agreement with Canadian biotech panCELLa, a company modifying cell therapies to evade the immune system and prevent rejection. Evotec additionally has a PanOmics platform that combines transcriptomics and proteomics data to find new drug targets.

After expanding our biologics capabilities through the acquisition of Just Evotec Biologics in 2019, Evotec GT is yet another important piece to complete the multimodality puzzle, explained Lathaler.

The timing was ideal, as Evotec GT brings our existing expertise in the gene therapy field to a new level. It also has close technological ties especially to our induced pluripotent stem cells and PanOmics platforms, so these platforms can grow and gain traction together.

Image from Shutterstock

See original here:
Evotec Makes Move into Gene Therapy with Takeda... - Labiotech.eu

German drug discovery company Evotec which has a thriving rolodex of biopharma partners such as Bayer, Boehringer Ingelheim, Novartis, Novo Nordisk, Pfizer, Sanofi, and Takeda is now venturing into gene therapies.

The company swallowed Seattle-based Just Biotherapeutics, a company focused on reducing the cost of manufacturing protein therapies last year. It is now setting up a dedicated R&D site for gene therapies in Austria, in an effort to achieve a modality-agnostic repertoire small molecules, biologics and now gene therapies.

The site will be run by a team of twenty researchers led by Friedrich Scheiflinger, who worked for Baxalta, Shire and Takeda. The scientists have expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases.

In recent years, precision medicines based on cell and gene therapies have emerged and are predicted to grow significantly. Gene therapy is a promising approach to the development of genetic medicines for patients, especially for inherited and rare diseases, said Evotec chief Werner Lanthaler in a statement.

Cell and gene therapies are not easy to develop and are prohibitively expensive to manufacture.A combined $2 billion has been set aside by nearly a dozen drugmakers including Pfizer and Novartis for gene therapy manufacturing since 2018, according to an analysis by Reuters in November.

Read more:
Small molecules, biologics and now gene therapies: Germany's Evotec adds another feather to its R&D cap - Endpoints News

Base editors, which convert one nucleotide to another without a double-strand DNA break, have the potential to treat diseases caused by mutant genes. One drawback, though, is that the DNA that encodes CRISPR base editors is longtoo long to fit in the adeno-associated viruses (AAVs) most commonly used for gene therapy. In a study published in Molecular Therapy on January 13, researchers split the DNA encoding a base editor into two AAV vectors and injected them into a mouse model of inherited amyotrophic lateral sclerosis (ALS). The strategy disabled the disease-causing gene, improving the animals symptoms and prolonging their lives.

Wed like to be able to make gene editing tools that can fit inside an AAV vector. Unfortunately, some of the tools are so big that they cant fit inside, so in this study, they were able to come up with a solution to that by using a split protein, says David Segal, a biochemist at the University of California, Davis, who was not involved in the work. Its not the first time that that system has been used, but its the first time its been applied to this kind of base editor.

Pablo Perez-Pinera, a bioengineer at University of Illinois at Urbana-Champaign, and colleagues developed a strategy to split the base editor into two chunks. In a study published in 2019, they generated two different AAV vectors, each containing a portion of coding DNA for an adenine-to-thymine base editor. They also included sequences encoding so-called inteinsshort peptides that when they are expressed within proteins stick together and cleave themselves out, a bit like introns in RNA. The researchers built the inteins into the vectors such that when the inteins produced by the two vectors dimerized, bringing the two base editor parts together, and then excised themselves, they left behind a full-length, functional base editor.

When Perez-Pinera told Thomas Gaj, also a bioengineer at the University of Illinois at Urbana-Champaign, about the strategy, Gaj tells The Scientist,they immediately set out to test it in a mouse model of ALS. The transgenic mice have about 25 copies of the human gene, superoxide dismutase 1(SOD1), with mutations that cause ALS in people. The animals display motor neuron loss and muscle atrophy, plus their neurons accumulate inclusionsdense spots in the gray and white matter of their spinal cords that include SOD1 proteinbefore dying at about four months of age on average. The symptoms and life expectancy in the 20 percent of ALS patients with mutations in SOD1vary based on which mutation they have, but most have muscle weakness and motor neuron death, as well as inclusions containing SOD1 protein.

Instead of using the adenine-to-thymine base editor, the researchers developed a cytosine-to-thymine converter using the coding sequence of Streptococcus pyogenes Cas9 and a guide RNA that targets both wild type and mutant human SOD1 to create an early stop codon. This doesnt affect the mouse SOD1. In human cells, the split base editor seemed to be even more efficient than when the editor was transfected at full length, hitting about 29 percent of the target sites, compared to the full-length editors 19 percent.

Next the authors packaged their split base editor into two AAV backbones and injected them or a control AAV into the animals lumbar cerebrospinal fluid when they were around two months old. The vectors ended up primarily in astrocytes, as well as in neurons and microglia. While the researchers didnt see a difference in symptom onset at around three months, the mice that received the base editor maintained their weight and lived about 10 percent longer than controls. The treated mice also had fewer SOD1-positive inclusions and healthier motor neurons.

In this cross section of the spinal cord of a mouse model of amyotrophic lateral sclerosis (ALS), researchers delivered a CRISPR base editing system (yellow) to astrocytes (red) in order to disable the expression of a mutant gene and reduce symptoms.

Colin Lim, University of Illinois

Using base editors to disable the mutant SOD1 gene in astrocytes (a cell type that normally supports healthy nervous system function but in SOD1-ALS exerts toxicity onto motor neurons) led to a marked slowing in disease progression, Gaj writes in an email to The Scientist. Since many persons with ALS are diagnosed following the onset of symptoms, pre-clinical strategies that can meaningfully slow the disease are especially important and should be further studied.

This is a good indication that base editing actually can be used to treat ALS, says Baisong Lu, a gene therapy researcher at Wake Forest School of Medicine who did not participate in the work. He cautions that off-target effectsthe base editor can edit both DNA and RNAand how long the AAV delivery method lasts are both in need of more work before this technique would be safe for people.

The dual AAV strategy could also be expensive, says Mimoun Azzouz, a neuroscientist at the University of Sheffield in the United Kingdom. Thinking about the clinical development and marketing and the commercialization of this product, you need to manufacture two viruses, and you need to assess these two viruses for safety, so the cost can be extremely high.

Despite the challenges, the strategy shows promise for translation to humans, Perez-Pinera writes in an email to The Scientist.AAVs are already approved by the Food and Drug Administration for gene therapy, he explains. Plus, using a humanized model of the diseasea mouse that contains the human sequence of the target genemeans that the method validated in mouse models can be translated to people without adapting them to target a different sequence. People who develop ALS due to a mutation in SOD1also have one good copy of the gene, just like the mice, which have a functioning mouse copy.

We injected animal models shortly before disease onset. While injecting the animals earlier could improve the outcome of the disease as demonstrated in other studies, the reality is that ALS is not typically diagnosed until the patient experiences symptoms. Our study predicts what can be expected from treating a patient recently diagnosed with the disease, Perez-Pinera writes.

We still have some distance to travel before the results in our current study can benefit ALS patients, Gaj acknowledges. The researchers are working on minimizing off target effects and on developing new delivery methods that could improve efficacy. We still have a number of important questions to answer and technological hurdles to address before we begin thinking about clinical translation.

C.K.W. Lim et al., Treatment of a mouse model of ALS by in vivo base editing,Molecular Therapy,doi:10.1016/ j.ymthe.2020.01.005, 2020.

Abby Olena is a freelance journalist based in Alabama. Find her on Twitter@abbyolena.

Read more from the original source:
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing - The Scientist

Niche players in the global gene therapy market must leverage greater government expenditure and upgrade their existing infrastructure along with expanding their gene therapy centers for sustaining their market hegemony.

ROCKVILLE, MD / ACCESSWIRE / April 8, 2020 / Global gene therapy market is poised for robust growth with net revenue pool set to exceed approximately US$ 5 Bn by 2026 end. The market is receiving tailwinds from advancements in synthetic biology. On that premise, the gene therapy market will expand 3X through over the forecast period, projects Fact.MR (2020-2026).

"Certain types of cancer such as Diffuse Large B-cell Lymphoma (DLBCL) and lymphoblastic leukemia are contributing to high mortality rates across the world. Gene therapy is gaining increasing recognition in its immense potential for treating rare diseases. Continued research and development in the area of gene therapy is supporting market growth as well," states Fact.MR.

Request PDF sample of the 170-page report on the Gene Therapy Market-

https://www.factmr.com/connectus/sample?flag=S&rep_id=4648

Gene Therapy Market - Key Findings

Gene Therapy Market - Key Driving Factors

Explore 59 tables and 79 figures of the study. Request TOC of the report at-

https://www.factmr.com/report/4648/gene-therapy-market

Gene Therapy Market - Key Restraints

Impact of COVID-19 on Gene Therapy Market

In view of ongoing onslaught of COVID-19 pandemic, the focus of major healthcare authorities of the world has gravitated towards developing vaccines for the deadly respiratory disease. Gene therapy is one such area of research which could help boost antibodies required to treat patients infected with Coronavirus. For instance, Generation Bio is exploring the potential role of gene therapy in treating COVID-19 patients. Hence, the global gene therapy market will benefit from the outbreak in that market players are rushing to develop multiple therapeutic approaches for SARS-CoV-2. Growing fears of similar Coronavirus outbreaks in the future will continue accelerating the development of gene therapy as well.

Competitive Landscape

Prominent players profiled in this Fact.MR study include, but are not limited to, Orchard Therapeutics Limited, CELGENE CORPORATION, Spark Therapeutics, Inc., Sibiono GeneTech Co. Ltd., Spark Therapeutics Inc., Gilead Sciences Inc., and Novartis AG. Developed regions remain the key focus area of major stakeholders in the global gene therapy market. Existing gene therapy centers are being prioritized by market players in order to utilize the full extent of their resources. Moreover, they are benefitting from success rates associated with gene therapy and faster drug approvals. Gilead Sciences Inc. expanded their gene therapy centers to a total of 90 recently.

About the Report

This 170-page study offers in-depth commentary on the gene therapy market. The study provides compelling insights on the gene therapy market on the basis of product (yescarta, kymriah, luxturna, strimvelis, and gendicine), application (ophthalmology, oncology, Adenosine Deaminase Deficiency- Severe Combined Immunodeficiency) across three regions (The United States, Europe, and Rest of the World).

Explore Fact.MR's Comprehensive Coverage on Healthcare Landscape

Foot Orthotic Insoles Market - Get Fact.MR's comprehensive insights on the global foot orthotic insoles market covering segmental analysis, region-wise data, and market players for the predefined projection period (2020-2026).

Automated External Defibrillators Market - Fact.MR's latest analysis on the global automated external defibrillators market encompasses qualitative and quantitative insights for the course of forecast period (2020-2025).

Foley Catheters Market - Know more about the key influencing factors, major drivers, opportunities, and challenges for the global foley catheters market through Fact.MR in-depth study during 2020-2027.

About Fact.MR

Expert analysis, actionable insights, and strategic recommendations of the veteran research team at Fact.MR helps clients from across the globe with their unique business intelligence requirements. With a repository of over thousand reports and 1 million+ data points, the team has scrutinized the Healthcare industry across 50+ countries for over a decade. The team provides unmatched end-to-end research and consulting services. Fact.MR's healthcare market reports and industry analysis help businesses navigate challenges and take critical decisions with confidence and clarity amidst breakneck competition.

Contact:

Fact.MR11140 Rockville PikeSuite 400Rockville, MD 20852United StatesEmail: sales@factmr.comWeb: https://www.factmr.com/PR- https://www.factmr.com/media-release/1409/global-gene-therapy-market

SOURCE: FactMR

View source version on accesswire.com: https://www.accesswire.com/584395/Gene-Therapy-Market-Set-for-3X-Expansion-Between-2020-and-2026-COVID-19-Stimulating-Development-of-Multiple-Therapeutic-Approaches-FactMR

See more here:
Gene Therapy Market Set for 3X Expansion Between 2020 and 2026; COVID-19 Stimulating Development of Multiple Therapeutic Approaches: Fact.MR -...

The genetic code to alllife on Earth, both simple and complex, comes down to four basic letters: A, C,T and G.

Untangling the role thatthese letters play in lifes blueprint has allowed scientists to understandwhat makes everything from bacteria to people the way they are. But as researchershave learned more, they have also sought ways to tinker with this blueprint,bringing ethical dilemmas into the spotlight. The Gene, a two-part PBS documentary from executive producer Ken Burnsairing April 7 and 14, explores the benefits and risks that come withdeciphering lifes code.

The film begins with oneof those ethical challenges. The opening moments describe how biophysicist HeJiankui used the gene-editing tool CRISPR/Cas9 to alter the embryos of twin girls who were born in China in 2018 (SN: 12/17/18). Worldwide, criticscondemned the move, claiming it was irresponsible to change the girls DNA, asexperts dont yet fully understand the consequences.

This moment heraldedthe arrival of a new era, narrator David Costabile says. An era in whichhumans are no longer at the mercy of their genes, but can control and evenchange them.

Headlines and summaries of the latest Science News articles, delivered to your inbox

The story sets the stagefor a prominent theme throughout the documentary: While genetics holdsincredible potential to improve the lives of people with genetic diseases,there are always those who will push science to its ethical limits. But thedriving force in the film is the inquisitive nature of the scientistsdetermined to uncover what makes us human.

The Gene, based on the book of the same name by Siddhartha Mukherjee (SN:12/18/16), one of the documentarys executive producers, highlights many ofthe most famous discoveries in genetics. The film chronicles Gregor Mendels classicpea experiments describing inheritance and how experts ultimately revealed inthe 1940s that DNA a so-called stupid molecule composed of just four chemicalbases, adenine (A), thymine (T),cytosine (C) and guanine (G) is responsible for storing geneticinformation. Historical footage, inBurns typical style, brings to life stories describing the discovery of DNAshelical structure in the 1950s and the success of the Human Genome Project indecoding the human genetic blueprint in 2003.

The film also touches ona few of the ethical violations that came from these discoveries. The eugenicsmovement in both Nazi Germany and the United States in the early 20th century aswell as the story of the first person to die in a clinical trial for genetherapy, in 1999, cast a morbid shadow on the narrative.

Interwoven into thistimeline are personal stories from people who suffer from genetic diseases.These vignettes help viewers grasp the hope new advances can give patients asexperts continue to wrangle with DNA in efforts to make those cures.

In the documentarysfirst installment, which focuses on the early days of genetics, viewers meet a family whose daughter is grappling with arare genetic mutation that causes her nerve cells to die. The family searchesfor a cure alongside geneticist Wendy Chung of Columbia University. The secondpart follows efforts to master the human genome and focuses on AudreyWinkelsas, a molecular biologist at the National Institutes of Health studyingspinal muscular atrophy, a disease she herself has, and a family fighting tosave their son from a severe form of the condition.

For science-interested viewers, the documentary does not disappoint. The Gene covers what seems to be every angle of genetics history from the ancient belief that sperm absorbed mystical vapors to pass traits down to offspring to the discovery of DNAs structure to modern gene editing. But the stories of the scientists and patients invested in overcoming diseases like Huntingtons and cancer make the film all the more captivating.

Originally posted here:
The PBS documentary The Gene showcases genetics promise and pitfalls - Science News

A group of Korean researchers said they have confirmed a gene responsible for inherited retinal disorders (IRD) among Koreans.

IRD is a combination of several rare diseases that usually develops at a young age and progresses slowly over the lifetime. The patients gradually lose their sight, and most of them eventually lose their vision entirely due to continuous retinal cell degeneration.

The Seoul National University Bundang Hospital (SNUBH) Department of Ophthalmology and Seoul National University Hospital (SNUH) Department of Laboratory Medicine jointly conducted the study.

Currently, antioxidant therapy, artificial retinal transplantation, and stem cell therapy are being used to treat the disorder regardless of mutations, but the only viable treatment is gene therapy. Even when gene therapy is possible, only less than 1 percent of all IRD patients can be treated with it.

In the West, genetic abnormalities of these retinal diseases have been studied and known well. However, researches on Korean cases are still lacking, and the joint research team tackled the subject to find the causative gene for IRDs with 86 domestic patients, the team said in a news release on Wednesday.

The team studied and identified the gene responsible for the disorders by using the latest technique of gene analysis with the most number of patients who have been reported so far.

The study revealed that only 44 percent of the patients, 38 out of 86, possessed the causal gene for IRDs. Even among the patients with retinitis pigmentosa, the most common disorder among the IRDs, only 41 percent had the causative gene.

The causative genes could be quite diverse even in the same disorder. The patients can find a responsible gene only when they receive genetic counseling very actively and can receive gene counseling, too, the research team explained.

Differences were found in the type and frequency of causal gene mutations between Korean and Western cases. However, there were similarities between those of Korean and other Asian nations, including Japan.

The research and diagnosis environment for IRDs has been very poor until now, and our study has significance as a basic data for diagnosis and treatment for Korean patients with IRDs, SNUH Department of Ophthalmology Professor Woo Se-joon said.

Patients need to receive causal gene tests actively to provide the domestic medical communities with sufficient data, and a list of patients who can be treated. By doing so, clinical trials and new drug development in gene therapy will progress smoothly, he added.

Previously, only a few hospitals could diagnose the causative gene for IRDs and afford to test and treat IRD patients due to the high cost of genetic testing. Recently, however, the chance of diagnosis has increased as more hospitals are conducting genetic tests amid the lowered cost thanks to insurance benefits.

Also, the therapeutic opportunity for IRD patients is likely to get broadened, as the retinal pigment epithelium 65 gene (RPE65) therapy won approval from the U.S. Food and Drug Administration for the first time in the world.

Although we do not have a clear way to prevent IRDs at the moment, the prediction of risk and their early detection are developing through the discovery of family history and causative genes, Professor Woo said. Early diagnosis can prevent impaired vision by gene therapy and vision correction, and the patients will be able to choose appropriate jobs with social activities.

Also taking part in the research team were Professors Joo Kwang-sic and Park Kyu-hyung of SNUBH and Professors Seong Moon-woo and Park Sung-sup of SNUH.

The results of this study were published in the Journal of Korean Medical Science.

shim531@docdocdoc.co.kr

< Korea Biomedical Review, All rights reserved.>

View post:
SNUH team finds causal gene of inherited retinal disorder - Korea Biomedical Review

LOS ANGELES, April 09, 2020 (GLOBE NEWSWIRE) -- Enochian Biosciences, a company focused on gene-modified cellular therapy in infectious disease and cancer,announces that all three submissions to the 23rd Annual Meeting of the ASCGT were accepted for presentation. The Conference will be held virtually May 12-15, 2020.

Dr. Serhat Gumruku, inventor and co-founder, will have two presentations: Hijacking HBV Pol to Induce Apoptosis Specifically in Infected HepatocytesIn Vivo: A Novel Approach for Potential Treatment or Cure; and Increased Engraftment of Gene Modified HSPCs Overexpressing ALDH1 UsingIn VivoChemoselection. Dr. Ramesh Halder, Senior Staff Scientist, will present: Gene Modified CD34+Cells With Increased ALDH1 Expression ConfersIn VitroProtection Against Cyclophosphamide.

Forward-Looking StatementsStatements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties, including but not limited to the success or efficacy of our pipeline. All statements other than historical facts are forward-looking statements, which can be identified by the use of forward-looking terminology such as believes, plans, expects, aims, intends potential, or similar expressions. Actual events or results may differ materially from those projected in any of such statements due to various uncertainties, including as set forth in Enochians most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Enochian undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

Contact: ir@enochianbio.com

More here:
Enochian Biosciences Announces Scientific Presentations at the American Society of Cell and Gene Therapy (ASCGT) Annual Meeting - Yahoo Finance

Global Gene Therapy Market report underlines the specific study of the Biotechnology industry which explains what the market definition, classifications, applications, engagements, and global industry trends are. The market data analyses and evaluated in this Gene Therapy market report makes you achieve the business goals and objectives in preset time frame. It highlights a wide-ranging evaluation of the markets growth prospects and restrictions. This report is very useful to all sizes of business which makes it simpler to take informed decisions regarding different facets of industry. Gene Therapy market report truly acts as a backbone to the business.

A finest market research report acts as an innovative solution for the businesses in todays changing market place and hence this Gene Therapy report is generated. Key players in the market, major collaborations, merger and acquisitions along with trending innovation and business policies are also reviewed in this Gene Therapy report. The report has a list of key competitors with the required specifications and also provides the strategic insights and analysis of the key factors influencing the industry. The data and information of the Gene Therapy report not only helps business make data-driven decisions but also guarantees maximum return on investment (ROI).

Get the inside scope of the Sample report @https://www.theinsightpartners.com/sample/TIPHE100001165/

MARKET INTRODUCTION

Gene therapy is introduction of DNA into a patient to treat a genetic disease or a disorder. The newly inserted DNA contains a correcting gene to correct the effects of disease causing mutations. Gene therapy is promising treatment for the genetic diseases and also includes cystic fibrosis and muscular dystrophy. Gene therapy is suitable treatment for the infectious diseases, inherited disease and cancer.

Key Competitors In Market are Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, and HORAMA S.A.

MARKET SCOPE

The Global Gene Therapy Market Analysis to 2025 is a specialized and in-depth study of the gene therapy industry with a focus on the global market trend. The report aims to provide an overview of global gene therapy market with detailed market segmentation by cell type, application, and geography. The global gene therapy market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading market players and offers key trends and opportunities in the market.

Market segmentation:

By Cell Type (Somatic Gene Therapy, Germline Gene Therapy);

By Application (Genetic Disorder, Cancer, Neurological Disorder, and Others)

By Geography North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. And 13 countries globally along with current trend and opportunities prevailing in the region.

REASONS TO BUY

Save and reduce time carrying out entry-level research by identifying the growth, size, leading players and segments in the wound irrigation system market.

Highlights key business priorities in order to assist companies to realign their business strategies.The key findings and recommendations highlight crucial progressive industry trends in the global wound irrigation system market, thereby allowing players across the value chain to develop effective long-term strategies.

Develop/modify business expansion plans by using substantial growth offering developed and emerging markets.

Scrutinize in-depth global market trends and outlook coupled with the factors driving the market, as well as those hindering it.

Enhance the decision-making process by understanding the strategies that underpin security interest with respect to client products, segmentation, pricing and distribution.

Click to buy full report with all description:-https://www.theinsightpartners.com/buy/TIPHE100001165/

About Us:

The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are committed to provide highest quality research and consulting services to our customers. We help our clients understand the key market trends, identify opportunities, and make informed decisions with our market research offerings at an affordable cost.We understand syndicated reports may not meet precise research requirements of all our clients. We offer our clients multiple ways to customize research as per their specific needs and budget

Contact Us:The Insight Partners,Phone: +1-646-491-9876Email: [emailprotected]

Read more here:
Gene Therapy Market 2020: Competitive Landscape and Recent Industry Development Analysis by Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure...

University of Georgia Honors student Landon Clark is among 396 undergraduates across the nation to be recognized as Barry Goldwater Scholars, earning the highest undergraduate award of its type for the fields of the natural sciences, mathematics and engineering.

Clark, from Leesburg, is a third-year CURO Honors Scholar who is triple majoring in biochemistry and molecular biology, genetics and biology.

Since 1995, 61 UGA students have received the Goldwater Scholarship, which recognizes exceptional sophomores and juniors across the United States.

The University of Georgia is proud of Landon and pleased that he is extending our record of success in this prestigious competition, said President Jere W. Morehead. Already as an undergraduate student, he is helping UGA fulfill our commitment to conduct research that helps people lead longer, healthier lives.

This year, recipients were selected from a field of more than 5,000 college sophomores and juniors who were nominated by 461 academic institutions nationwide. Each awardee will receive up to $7,500 toward the cost of tuition, fees, books, and room and board.

Of this years Goldwater Scholars, 50 are mathematics and computer science majors, 287 are majoring in the natural sciences and 59 are majoring in engineering.

I am thrilled for Landon, who is absolutely one of our very best students, said David S. Williams, associate provost and director of the Honors Program, who serves as the UGA campus faculty representative for the Goldwater Scholarship. As a CURO Honors Scholar, Landon was recruited to come to UGA because of his promise as a researcher. This Goldwater award and recognition proves that his hard work is paying off. I look forward to watching his career continue to blossom in the future.

Clarks long-term plans include pursuing an MD/PhD in immunology, performing translational research on immune diseases using gene and immunotherapy techniques, and teaching at a university. As a translational researcher specializing in immunology, he plans to use gene therapy techniques to treat and cure immune disorders in humans.

Since his first weeks as a UGA freshman in August 2017, Clark has conducted research in the lab of Michael Terns, Distinguished Research Professor of Biochemistry and Molecular Biology. He researches the complex processes of CRISPR-Cas immune systems through a model archaeal organism, Pyrococcus furiosus. Now a junior, he has collaborated on three different projects, co-authored a paper, and mentors other undergraduates in this field.

Last summer, he worked in the translational research lab of H. Trent Spencer, a professor of pediatrics and director of the gene and cell therapy program at Emorys Winship Cancer Institute. There, Clark researched immune diseases and potential gene therapy techniques. In 2017, he also conducted research on sociological variables influencing educational inequality with Dawn Robinson, a professor of sociology at UGA.

Clark is a Crane Leadership Scholar, director of academic outreach for the Student Government Association, director of internal communications for UGA Red Cross, an exam writer for UGA Science Olympiad Outreach, co-president of the Biochemistry Undergraduate Society, co-president of the Omicron Delta Kappa National Honor Society, and treasurer of the Honors Program Student Council. He also volunteers in the neuroscience and cardiology units at Piedmont Athens Regional Medical Center.

Clark has presented his research at 10 conferences. He also studied neurology and cancer biology through UGAs Studies Abroad Program in Cortona, located in Tuscany, Italy.

The scholarship honoring Sen. Barry Goldwater was created to encourage outstanding students to pursue careers in the fields of mathematics, natural sciences and engineering. Since its first award in 1989, the Barry Goldwater Scholarship and Excellence in Education Foundation has bestowed 8,628 scholarships worth more than $68 million.

See the article here:
UGA's Landon Clark named 2020 Goldwater Scholar - University of Georgia

Detailed market survey on the Global Hemophilia Gene Therapy Market Research Report 2020-2026. It analyses the vital factors of the Hemophilia Gene Therapy market supported present business Strategy, Hemophilia Gene Therapy market demands, business methods utilised by Hemophilia Gene Therapy market players and therefore the future prospects from numerous angles well. Business associatealysis could be a market assessment tool utilized by business and analysts to grasp the quality of an business. Hemophilia Gene Therapy Market report It helps them get a sense of what is happening in an industry, i.e., demand-supply statistics, Hemophilia Gene Therapy Market degree of competition within the industry, Hemophilia Gene Therapy Market competition of the business with different rising industries, future prospects of the business.

NOTE: Hemophilia Gene Therapy reports include the analysis of the impact of COVID-19 on this industry. Our new sample is updated which correspond in new report showing impact of Covid-19 on Industry trends. Also we are offering 20% discount

Obtain sample copy of Hemophilia Gene Therapy market report: https://calibreresearch.com/report/global-hemophilia-gene-therapy-market-4387#request-sample

The Global Hemophilia Gene Therapy Market report is a fully analyzed and intelligent study of the international industry that focuses on a wide range of significant elements such as market size in terms of value and volume, regional growth analysis, competition and segmentation. It is considered as extraordinary findings that accountable to offer insightful details into some essential attributes related to the global Hemophilia Gene Therapy Market 2020. The detailed investigation of this report has been carried out by the list of skillful researchers and investigators with a deep analysis of current industry trends, availability of distinct opportunities, drivers, openings and limitation that influence the Hemophilia Gene Therapy Market on the global scale.

The Global Hemophilia Gene Therapy market worth about xx billion USD in 2020 and it is expected to reach xx billion USD in 2026 with an average growth rate of x%. United States is the largest production of Hemophilia Gene Therapy Market and consumption region in the world, Europe also play important roles in global Hemophilia Gene Therapy market while China is fastest growing region.

Checkout Inquiry For Buying or Customization of Hemophilia Gene Therapy Market Report: https://calibreresearch.com/report/global-hemophilia-gene-therapy-market-4387#inquiry-for-buying

Geographically, Hemophilia Gene Therapy market report is segmented into several key Regions, with production, consumption, revenue. The major regions involved in Hemophilia Gene Therapy Market are (United States, EU, China, and Japan).

Leading companies reviewed in the Hemophilia Gene Therapy report are:

Spark TherapeuticsFreeline TherapeuticsSangamo TherapeuticsUltragenyxuniQureShire PLCBioMarinBioverativ

Hemophilia Gene Therapy Market Product Type Segmentation As Provided Below:The Hemophilia Gene Therapy Market report is segmented into following categories:

The product segment of the report offers product market information such as demand, supply and market value of the product.

The application of product in terms of USD value is represented in numerical and graphical format for all the major regional markets.The Hemophilia Gene Therapy market report is segmented into Type by following categories;Hemophilia AHemophilia B

The Hemophilia Gene Therapy market report is segmented into Application by following categories;Hemophilia A Gene TherapyHemophilia B Gene Therapy

Reportedly, the massive growth graph in the research and development sectors will be liable to generate plenty of excellent opportunities in the upcoming years. The Hemophilia Gene Therapy market is a valuable resource of insightful information for specific business strategists. Apart from this, it also offers an in-depth summary of the Hemophilia Gene Therapy Market along with growth assessment, revenue share, demand & supply data, historical as well as futuristic amount etc. A group of research analysts offers a detailed description of the value chain and its distributors info. Moreover, the Hemophilia Gene Therapy market study report delivers comprehensive information regarding the global industry that enhances the scope, understanding and application of the same.

Checkout FREE Report Sample of Hemophilia Gene Therapy Market Report for Better Understanding: https://calibreresearch.com/report/global-hemophilia-gene-therapy-market-4387#request-sample

Industry analysis, for an entrepreneur or a company, is a method that helps it to understand its position relative to other participants in the Hemophilia Gene Therapy Market. It helps them to identify both the opportunities and threats coming their way and gives them a strong idea of the present and future scenario of the Hemophilia Gene Therapy industry. The key to extant during this changing business setting is to know the variations between yourself and your competitors within the Hemophilia Gene Therapy Market. The deep research study of Hemophilia Gene Therapy market based on development opportunities, growth limiting factors and feasibility of investment will forecast the Hemophilia Gene Therapy market growth.

Finally, The global research document on the Hemophilia Gene Therapy Market discovers a large set of information regarding the competitive business environment and other substantial components. The prime aim of these major competitors is to focus on improved technologies and newer innovations.

More:
Research on Hemophilia Gene Therapy Market (impact of COVID-19) 2020-2026 Spark Therapeutics, Freeline Therapeutics, Sangamo Therapeutics, Ultragenyx...

Welcome to the latest edition of our weekly EuroBiotech Report. We start this week with AstraZeneca, which revealed it aims to have a COVID-19-neutralizing antibody in the clinic in the next three to five months. AstraZeneca's compatriot GlaxoSmithKline also shared an update on its COVID-19 activities. GSK added Xiamen Innovax Biotech to the list of organizations it is working with on a COVID-19 vaccine. While those are just two of the many initiatives targeting COVID-19, Wellcome is worried about a funding shortfall. Wellcome wants businesses to commit $8 billion this month to keep programs moving forward quickly. Researchers working outside of COVID-19 are faring worse still, with Cancer Research UK responding to the pandemic with deep cuts. Away from COVID-19, Evotec entered the gene therapy space through a deal with Takeda. And more. Nick Taylor1. AstraZeneca targets summer start for COVID-19 antibody trial

AstraZeneca has outlined its multipronged approach to the development of antibodies against the pandemic SARS-CoV-2 virus. The Big Pharma is applying its own discovery capabilities to the task and helping to evaluate candidates identified by academic partners in China and the U.S.2. GSK allies with Innovax for COVID-19 vaccine R&D project

GlaxoSmithKline has teamed up with Xiamen Innovax Biotech to evaluate a vaccine against the novel coronavirus behind the COVID-19 pandemic. The agreement gives Innovax access to a GSK adjuvant to enhance the immune response triggered by its recombinant protein-based vaccine.3. Wellcome targets $8B raise in weeks to fix COVID-19 funding gap

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

Wellcome is calling on businesses to invest in an $8 billion (7 billion) fund focused on reducing cases of COVID-19 to zero as soon as possible. Almost half the money is earmarked for the development of drugs and vaccines that Wellcome sees as the worlds best exit strategy from the pandemic.4. COVID-19 claims Cancer Research UK funding cuts, forecasting major shortfall

Cancer Research UK (CRUK) said it has been forced to take the difficult decision to make deep funding cuts across its grants and institutes as the fallout from the pandemic continues to affect all areas of biomedical research.

5. Evotec allies with Takeda to move into gene therapy R&D

Evotec has allied with Takeda to expand into gene therapy research. The move sees Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.And more articles of note>>

Go here to see the original:
EuroBiotech ReportAZ, GSK in COVID-19, $8B funding call, CRUK cuts and Evotec - FierceBiotech