Archive for the ‘Gene Therapy Research’ Category

The gene therapy company sold 7.6 million shares of stock at a price of $1.05 per share

Genprex Inc () announced an $8 million offering of stock through securities purchase agreements made with influential investors.

The gene therapy company sold 7.6 million shares at a price of $1.05 per share, in accordance with Nasdaq rules governing registered direct offerings.

Genprex, based in Austin, Texas and Cambridge, Massachusetts, plans to use the proceeds to advance its clinical programs in non-small cell lung cancer, as well as for working capital and general corporate purposes.

The company is developing a new approach to treating cancer, including its initial product candidateOncoprex, which is an immunogene therapy for non-small cell lung cancer.

Contact Andrew Kessel at [emailprotected]

Follow him on Twitter @andrew_kessel

Add related topics to MyProactive

Create your account: sign up and get ahead on news and events

The Company is a publisher. You understand and agree that no content published on the Site constitutes a recommendation that any particular security, portfolio of securities, transaction, or investment strategy is...

In exchange for publishing services rendered by the Company on behalf of Genprex, Inc. named herein, including the promotion by the Company of Genprex, Inc. in any Content on the Site, the Company receives from said...

Go here to see the original:
Genprex rakes in $8M in at-the-market stock offering - Proactive Investors USA & Canada

The Gene Therapy and Antisense Drugs Market research report added by Market Study Report, LLC, provides a succinct analysis on the recent market trends. In addition, the report offers a thorough abstract on the statistics, market estimates and revenue forecasts, which further highlights its position in the industry, in tandem with the growth strategies adopted by leading industry players.

The Gene Therapy and Antisense Drugs market study is a well-researched report encompassing a detailed analysis of this industry with respect to certain parameters such as the product capacity as well as the overall market remuneration. The report enumerates details about production and consumption patterns in the business as well, in addition to the current scenario of the Gene Therapy and Antisense Drugs market and the trends that will prevail in this industry.

Request Sample Report @ https://www.tmrresearch.com/sample/sample?flag=B&rep_id=500&source=atm

What pointers are covered in the Gene Therapy and Antisense Drugs market research study?

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the regional landscape of the industry:

The geographical reach of the Gene Therapy and Antisense Drugs market has been meticulously segmented into United States, China, Europe, Japan, Southeast Asia & India, according to the report.

The research enumerates the consumption market share of every region in minute detail, in conjunction with the production market share and revenue.

Also, the report is inclusive of the growth rate that each region is projected to register over the estimated period.

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the competitive landscape of the industry:

The competitive expanse of this business has been flawlessly categorized into companies such as

Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

Request For Discount On This Report @ https://www.tmrresearch.com/sample/sample?flag=D&rep_id=500&source=atm

Exclusive details pertaining to the contribution that every firm has made to the industry have been outlined in the study. Not to mention, a brief gist of the company description has been provided as well.

Substantial information subject to the production patterns of each firm and the area that is catered to, has been elucidated.

The valuation that each company holds, in tandem with the description as well as substantial specifications of the manufactured products have been enumerated in the study as well.

The Gene Therapy and Antisense Drugs market research study conscientiously mentions a separate section that enumerates details with regards to major parameters like the price fads of key raw material and industrial chain analysis, not to mention, details about the suppliers of the raw material. That said, it is pivotal to mention that the Gene Therapy and Antisense Drugs market report also expounds an analysis of the industry distribution chain, further advancing on aspects such as important distributors and the customer pool.

The Gene Therapy and Antisense Drugs market report enumerates information about the industry in terms of market share, market size, revenue forecasts, and regional outlook. The report further illustrates competitive insights of key players in the business vertical followed by an overview of their diverse portfolios and growth strategies.

Customize This Report @ https://www.tmrresearch.com/sample/sample?flag=CR&rep_id=500&source=atm

Some of the Major Highlights of TOC covers:

Go here to see the original:
R&D Activities to Fast-track the Growth of the Gene Therapy and Antisense Drugs Market Between 2017 2025 - Fusion Science Academy

This report presents the worldwide In Situ Hybridization market size (value, production and consumption), splits the breakdown (data status 2018 and forecast to 2025), by manufacturers, region, type and application.

This study also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market.

Request Sample Report @ https://www.tmrresearch.com/sample/sample?flag=B&rep_id=782&source=atm

Top Companies in the Global In Situ Hybridization Market:

key drivers, restraints, recent trends, and growth opportunities in the global market for in situ hybridization. The market shares, product portfolios, technological developments, and business strategies of the major players are discussed in detail. The report also sheds light on factors such as market size, supply and demand ratio, market attractiveness, and key segments of the in situ hybridization market.

Global In Situ Hybridization Market: Drivers and Restraints

Technological advancements in the field of in situ hybridization, such as development of the cytogenetic technique, is one of the key factors driving the market. The rising prevalence of cancer has necessitated clinical research, which is another major market driver. The introduction of new therapeutic agents has led to a high rate of adoption of companion diagnostics, boosting the overall market expansion. Besides these factors, the growth of the pharmaceutical and biotechnology industry has improved the prospects of the in situ hybridization market worldwide.

Florescence in situ hybridization (FISH) can be used in the detection of genetic abnormalities such as aneuploidy, characteristic gene fusion, or loss of a chromosomal region. It is also useful for research in the fields of gene mapping and identification of genetic aberrations, which are responsible for cancer. As this technique is simple yet effective, FISH will ensure the growth of the global in situ hybridization market.

On the contrary, strict regulatory policies will obstruct the growth of the in situ hybridization market. However, the emergence of molecular cytogenetics will present significant opportunities due to unmet needs in accurate disease diagnosis, rising number of chromosomal disorders, and surge in population.

Global In Situ Hybridization Market: Regional Outlook

On the basis of geography, the global market for in situ hybridization can be segmented into Europe, Asia Pacific, Latin America, North America, and the Middle East and Africa. North America holds a large share in the overall market, with Europe and Asia Pacific also exhibiting promising growth. Extensive research activities in countries such as Canada and the U.S., increased adoption of companion diagnostics, and beneficial government policies have been aiding the in situ hybridization market in North America.

Over the forecast period 2017-2025, Asia Pacific will undergo tremendous growth on account of growing incidence of cancer and its diagnosis, increased healthcare expenditure, and increasing health awareness among people. The presence of international companies in countries such as India and China will further provide an impetus to the market.

Companies Mentioned in the Report

The major companies operating in the market for in situ hybridization include Bio Sb, Inc., Advanced Cell Diagnostics, Inc., Merck KGaA, Abbott Laboratories, Inc., Leica Biosystems Nussloch GmbH, Danaher Corporation, and Agilent Technologies. Several companies are using business strategies such as product enhancement, collaborations, acquisitions, and partnerships with a view to increasing profit.

Request For Discount On This Report @ https://www.tmrresearch.com/sample/sample?flag=D&rep_id=782&source=atm

The report provides a valuable source of insightful data for business strategists and competitive analysis of In Situ Hybridization Market. It provides the In Situ Hybridization industry overview with growth analysis and futuristic cost, revenue and many other aspects. The research analysts provide an elaborate description of the value chain and its distributor analysis. This Tire In Situ Hybridization study provides comprehensive data which enhances the understanding, scope and application of this report.

Influence of the In Situ Hybridization market report:

-Comprehensive assessment of all opportunities and risk in the In Situ Hybridization market.

In Situ Hybridization market recent innovations and major events.

-Detailed study of business strategies for growth of the In Situ Hybridization market-leading players.

-Conclusive study about the growth plot of In Situ Hybridization market for forthcoming years.

-In-depth understanding of In Situ Hybridization market-particular drivers, constraints and major micro markets.

-Favorable impression inside vital technological and market latest trends striking the In Situ Hybridization market.

Customize This Report @ https://www.tmrresearch.com/sample/sample?flag=CR&rep_id=782&source=atm

The report has 150 tables and figures browse the report description and TOC:

Table of Contents

1 Study Coverage

1.1 In Situ Hybridization Product

1.2 Key Market Segments in This Study

1.3 Key Manufacturers Covered

1.4 Market by Type

1.4.1 Global In Situ Hybridization Market Size Growth Rate by Type

1.4.2 Hydraulic Dredges

1.4.3 Hopper Dredges

1.4.4 Mechanical Dredges

1.5 Market by Application

1.5.1 Global In Situ Hybridization Market Size Growth Rate by Application

2 Executive Summary

2.1 Global In Situ Hybridization Market Size

2.1.1 Global In Situ Hybridization Revenue 2014-2025

2.1.2 Global In Situ Hybridization Production 2014-2025

2.2 In Situ Hybridization Growth Rate (CAGR) 2019-2025

2.3 Analysis of Competitive Landscape

2.3.1 Manufacturers Market Concentration Ratio (CR5 and HHI)

2.3.2 Key In Situ Hybridization Manufacturers

2.3.2.1 In Situ Hybridization Manufacturing Base Distribution, Headquarters

2.3.2.2 Manufacturers In Situ Hybridization Product Offered

2.3.2.3 Date of Manufacturers Enter into In Situ Hybridization Market

2.4 Key Trends for In Situ Hybridization Markets & Products

3 Market Size by Manufacturers

3.1 In Situ Hybridization Production by Manufacturers

3.1.1 In Situ Hybridization Production by Manufacturers

3.1.2 In Situ Hybridization Production Market Share by Manufacturers

3.2 In Situ Hybridization Revenue by Manufacturers

3.2.1 In Situ Hybridization Revenue by Manufacturers (2019-2025)

3.2.2 In Situ Hybridization Revenue Share by Manufacturers (2019-2025)

3.3 In Situ Hybridization Price by Manufacturers

3.4 Mergers & Acquisitions, Expansion Plans

More Information.

Read more:
Global Cake Mix Market Industry: A Latest Research Report to Share Market Insights and Dynamics - Fusion Science Academy

The Global Gene Therapy Market is growing at an exciting pace driven by changing dynamics and risk ecosystem, a study of which forms the crux of the report. The study on the global Gene Therapy market takes a closer look at several regional trends and the emerging regulatory landscape to assess its prospects. The critical assessment of the numerous growth factors and breaks in the global Gene Therapy market offered in the analyses helps in assessing the lucrativeness of its key segments.

Download Free PDF Brochure for Latest Research Study of Gene Therapy Market:: https://www.globalmarketers.biz/report/life-sciences/global-gene-therapy-industry-market-research-report/7517 #request_sample

This Report Covers Leading Companies Associated in Worldwide Market:

UniQure NVBluebird Bio,Celladon,Avalanche Bio,Sangamo,Shanghai Sunway Biotech Co. LtdAdvantagene,Sibiono GeneTech,Spark Therapeutics,Dimension Therapeutics,

Summary of Market: The global Gene Therapy market is valued at xx million US$ in 2019 is expected to touch xx million US$ by the close of 2025, growing at a CAGR of xx% during 2020-2025.

The report emphases on Gene Therapy Market volume and value at Global Level, Regional Level And Company Level. From a global standpoint, this report embodies overall market size by studying historical data and future outlook.

The report is bifurcated into product type, applications, and regions worldwide. The above areas are further bifurcated into country-level data statistics for the below countries.

The key regions and countries covered in this report are:

Please note, the regional and country level data can be altered and provided as per clients custom requirements.

Global Gene Therapy Market Segmentation, By Product Type:

Viral vectorNon-viral vector

Global Gene Therapy Market Segmentation, By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Inquire Before Buying: https://www.globalmarketers.biz/report/life-sciences/global-gene-therapy-industry-market-research-report/7517 #inquiry_before_buying

Research objectives:

To study and estimate the market size of Gene Therapy , in terms of value.

To find development and challenges for the global market.

To observe worthwhile expansions including expansions, new services presents in worldwide industry.

To classify and assess the side view of important companies of Global Gene Therapy industry.

Key Questions Answered in the Report:

How is the Gene Therapy market expected to Grow In Terms Of Value during the study period?

What are the Competition Developments and Trends in the Gene Therapy market?

What are the core Macro-Economic and Industry Factors impacting the growth of the Gene Therapy market?

What are the Key Challenges, Opportunities, and Improvements faced by market players in the global Gene Therapy market?

Table of Content

1 Report Outline

1.1 Research Opportunity

1.2 Major Industrialists

1.3 Market Segment by Type

1.4 Market Segment by Application

1.5 Study Objectives

1.6 Years Considered

2 Global Evolution Trends

2.1 Production and Volume Analysis

2.1.1 Global Gene Therapy Production Value 2015-825

2.1.2 Global Gene Therapy Production 2015-2025.

2.1.3 Global Gene Therapy Capacity 2015-2025.

2.1.4 Global Gene Therapy Marketing Pricing and Trends

2.2 Major Producers Growth Rate (CAGR) 2020-2025.

2.2.1 Global Gene Therapy Market Size CAGR of Major Regions

2.2.2 Global Gene Therapy Market Share of Major Regions

2.3 Industry Trends

2.3.1 Market Top Trends

2.3.2 Market Operators

3 Market Share by Industrialists

3.1 Capacity and Production by Industrialists

3.1.1 Global,Gene Therapy Capacity by Industrialists

3.1.2 Global Gene Therapy Production by Industrialists

3.2 Revenue by Industrialists

3.2.1. Gene Therapy Revenue by Industrialists (2015-2020)

3.2.2. Gene Therapy Revenue Share by Industrialists (2015-2020)

3.2.3 Global Gene Therapy Market Concentration Ratio (CR5 and HHI)

3.3. Gene Therapy Price by Industrialists

3.4 Major Industrialists of Gene Therapy Plants/Factories Distribution and Area Served

3.5 Date of Major Industrialists Enter into Gene Therapy Market

3.6 Major Industrialists Gene Therapy Product Offered

3.7 Mergers & Acquisitions, Expansion Plans

4 Market Dimensions by Type

4.1 Production and Production Rate for Each Type

4.2 Global Gene Therapy Production Market Share by Type

4.3 Global Gene Therapy Production Value Market Share by Type

4.4. Gene Therapy Ex-factory Price by Type

5 Market Size by Application

5.1 Overview

5.2 Global Gene Therapy Consumption by Application

6 Production by Regions

6.1 Global Gene Therapy Production (History Data) by Regions 2015-2020.

6.2 Global Gene Therapy Production Value (History Data) by Regions

6.3 North America

6.3.1 North America Production Growth Rate 2015-2020.

6.3.2 North America Production Value Growth Rate 2015-2020.

6.3.3 Major Players in North America

6.3.4 North America Import & Export

6.4 Europe

6.4.1 Europe Production Growth Rate 2015-2020.

6.4.2 Europe Production Value Growth Rate 2015-2020.

6.4.3 Major Players in Europe

6.4.4 Europe Import & Export

6.5 China

6.5.1 China Production Growth Rate 2015-2020.

6.5.2 China Production Value Growth Rate 2015-2020.

6.5.3 Major Players in China

6.5.4 China Import & Export

6.6 Japan

6.6.1 Japan Production Growth Rate 2015-2020.

6.6.2 Japan Production Value Growth Rate 2015-2020.

6.6.3 Major Players in Japan

6.6.4 Japan Import & Export

7. Gene Therapy Consumption by Regions

7.1 Global Gene Therapy Consumption (History Data) by Regions

7.2 North America

7.2.1 North America Consumption by Type

7.2.2 North America Consumption by Application

7.2.3 North America Consumption by Countries

7.2.4 United States

7.2.5 Canada

7.2.6 Mexico

7.3 Europe

7.3.1 Europe Consumption by Type

7.3.2 Europe Consumption by Application

7.3.3 Europe Consumption by Countries

See the original post here:
Gene Therapy Market Is Thriving Worldwide with major players Like: UniQure NV Bluebird Bio, Celladon, Avalanche Bio - Expedition 99

A recent market study published by Marketresearch.biz in its upcoming report outlook titled, Gene Therapy Market: Global Industry Analysis and Astonishing Growth [2020-2029] offers key market insights and emerging trends on the global Gene Therapy market.

The report has offered an exhaustive analysis of the Gene Therapy Market taking into consideration all the crucial aspects like growth factors, constraints, market developments, and future prospects. Market researchers and industry experts have pointed out the key market trends and prospects that may impact the overall Gene Therapy Market growth. This will help players to leverage the opportunities to strengthen their position. Also, the report throws light on the important factors that are contributing to the Gene Therapy Market growth. Additionally, challenges and impeding factors that could hamper the growth of the Gene Therapy Market in the years to come are mentioned in the report.

The Gene Therapy market report includes comprehensive information about the markets major competitors, including various organizations, companies, associations, suppliers and manufacturers competing for production, supply, sales, revenue generation, and after-sales performance expectations. The bargaining power of numerous vendors and buyers have also been included in the research report.

Download Free PDF Brochure with Full Analysis of Key Players:https://marketresearch.biz/report/gene-therapy-market/request-sample

Following Key Players are Analysed in this Report: Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

Market Dynamics:

Set of qualitative informative data that incorporates PORTER Five Forces Model, Macro-Economic factors, PESTEL Analysis, SWOT Analysis, Value Chain Analysis, Regulatory Framework along with Industry Background and Overview.

Worldwide Gene Therapy Research Methodology

Marketresearch.biz presents a detailed picture of the market by way of study, synthesis, and summation of information from various sources. The information thus presented is reliable, comprehensive, and the result of extensive research, both primary and secondary. The analysts have presented the different features of the market with a particular focus on identifying the key business influencers.

Gene Therapy Market Data Break Down is illuminated below by vector type, gene type, application, and region:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

The report cast light on an extensive analysis based on the most prominent Gene Therapy manufacturers operating in the industry. Activities performed by robust Gene Therapy manufacturers/companies are product development, research, and innovation as well as technology adoptions that are intensifying the competitive intensity and companys ability to offer better product lineup. The companies are also performing strategic acquisitions, ventures, mergers, and partnerships to enlarge their serving area and strengthen their existence worldwide.

The study delivers an exact evaluation of the financial operations of companies covering Gene Therapy sales volume, capital investment, gross margin, profitability, revenue, cash flow, and growth rate. Their manufacturing capacity, production volume, product specifications, import-export activities, production processes, raw material sourcing, and distribution networks are also elaborated in this Gene Therapy report. Companies are also engaged in product launches, promotional activities, and brand developments as part of strategic planning.

How will the report help new companies to plan their investments in the Gene Therapy market?

The Gene Therapy market research report classifies the competitive spectrum of this industry in elaborate detail. The study claims that the competitive reach spans the companies.

The report also mentions about the details such as the overall remuneration, pricing trends, product sales figures, gross margins, etc.

Information about the sales & distribution area alongside the details of the company, such as company overview, product specifications, buyer portfolio, etc., are provided in the Gene Therapy market study.

Inquire/Speak To Expert for Further Detailed Information About Gene Therapy Report :https://marketresearch.biz/report/gene-therapy-market/#inquiry

Regional Outlook: Regional analysis is another important part of the report which is segregated into different sections. One section of the report is entirely dedicated to regional consumption analysis whereas another for regional production analysis. It includes North America, Europe, China, Japan, Southeast Asia, India.

Some of the Major Highlights of TOC covers in Gene Therapy Market Report:

Chapter 1: Methodology & Scope of Gene Therapy Market

Definition and forecast parameters

Methodology and forecast parameters

Data Sources

Chapter 2: Executive Summary of Gene Therapy Market

Business trends

Regional trends

Product trends

End-use trends

Chapter 3: Gene Therapy Industry Insights

Industry segmentation

Industry landscape

Vendor matrix

Technological and innovation landscape

Chapter 4: Gene Therapy Market, By Region

Chapter 5: Company Profile

Business Overview

Financial Data

Product Landscape

Strategic Outlook

SWOT Analysis

And CONTINUE

Analysts with high skill in information gathering and governance use industry strategies to collate and examine data at all stages. Our analysts are trained to combine superior research methodology, modern data collection techniques, subject expertise and years of collective experience to deliver useful and accurate research reports.

We will be happy to attend to your queries regarding the above-market study. Kindly contact us atinquiry@marketresearch.bizto learn more about the market report.

Getin Touch with Us:

Mr. Benni Johnson

Prudour Pvt. Ltd.

420 Lexington Avenue,

Suite 300 New York City, NY 10170,

United States

Tel:+ 1-347-826-1876

Website:https://marketresearch.biz/

Thanks a million for reading! You can also request custom information like chapter-wise or specific region-wise study as per your interest.

Link:
Gene Therapy Market Segmentation 2020 | Analyzing the Impact Followed by Restraints till 2029 - News Monitoring

Coherent Market Insights has recently announced the addition of a new research report to its repository named, Global Cell and Gene Therapy Market Status and Forecast 20192026, covering top-line subjective and quantitative synopsis data. The market review provides an eccentric tool for analyzing the market in terms of strengths, and weakness, marking opportunities, and supporting strategic and proficient decision-making. The key drivers and restraints affecting the growth of the Cell and Gene Therapy are stated.

A comprehensive analysis of the Cell and Gene Therapy market is presented in this document, along with a brief overview of the segments in the industry. The study presents a feasible estimate of the current market scenario, including the Cell and Gene Therapy market size with regards to the volume and renumeration. The report is a collection of significant data related to the competitive landscape of the industry. It also contains data with regards to several regions that have successfully established its position in the Cell and Gene Therapy market.

Get Free Sample Copy of this Report: https://www.coherentmarketinsights.com/insight/request-sample/2475

The Top players including:Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

The report also points out the latest trends in the market and the various opportunities for the market to grow in the near future. Insightful information about the key players such as business market overview, product offerings, and industry revenue segmentation has been provided in the report. It also serves an extensive analysis of different sections and sub-segments which offers market insights toward the historic market scenarios along with future growth and prospect.

Our competitor profiling includes evaluation of distribution channels and products and services offered by and financial performance of companies operating in the global Cell and Gene Therapy market. We also provide Porters Five Forces, PESTLE, and SWOT analysis to assess competitive threat and examine other aspects of the global Cell and Gene Therapy market. The report offers strategic recommendations, competitor benchmarking for performance measurement, and analysis of partnership, merger, and acquisition targets and industry best practices. It also provides analysis of profitability and cost across the industry value chain.

Competitive Rivalry: The Cell and Gene Therapy report incorporates the detailed analysis of the leading organizations and their thought process and what are the methodologies they are adopting to maintain their brand image in this market. The report aides the new bees to understand the level of competition that they need to fight for to strengthen their roots in this competitive market.

Principal Research:

The research team works with industry experts from the Global Cell and Gene Therapy industry including the management organizations, processing organizations, value chain analytics by service providers of the Cell and Gene Therapy market.

Subordinate Research:

In the Secondary research vital information about the Cell and Gene Therapy industries value chain, total pool of key players, and application areas. Market separation is done as per the industrial drifts to the deepest level, terrestrial markets and key developments from both market place and technology-oriented viewpoints.

Get Free PDF Brochure of this Report: https://www.coherentmarketinsights.com/insight/request-pdf/2475

This report forecasts revenue growth at a global, regional & country level, and provides an analysis of the market trends in each of the sub-segments from 2019 to 2026.

North America (USA, Canada, Mexico, etc.)

Asia-Pacific (China, Japan, India, Korea, Australia, Indonesia, Taiwan, Thailand, etc.)

Europe (Germany, UK, France, Italy, Russia, Spain, etc.)

Middle East & Africa (Turkey, Saudi Arabia, Iran, Egypt, Nigeria, UAE, Israel, South Africa, etc.)

South America (Brazil, Argentina, Colombia, Chile, Venezuela, Peru, etc.)

In addition, the Cell and Gene Therapy market research will help solve the following problems:

Ultimately, Cell and Gene Therapy reports provide detailed information and expert analysis on key consumer trends and behaviours in the market, as well as an overview of market data and key brands. Cell and Gene Therapy Market Reports provide all data with easy-to-understand information

Purchase Copy of This Business Report: https://www.coherentmarketinsights.com/insight/buy-now/2475

About us:

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

Contact Us:

Name: Mr. Raj Shah

Phone: US +12067016702 / UK +4402081334027

Email:[emailprotected]

Visit our Blog:https://hospitalhealthcareblog.wordpress.com/

Continued here:
Cell and Gene Therapy Market to Exceed Revenues Worth US$ By the End of 2020 2027 - Melanian News

Experts at Newcastle University have shown that the rate of kidney disease in people with Joubert syndrome is determined by the genetic makeup of the individual and each patient may respond differently to the treatment.

Joubert syndrome is a complex disorder, affecting approximately one in 80,000 newborns, causing varying degrees of physical, mental and sometimes visual impairments. It is often associated with severe kidney disease that requires dialysis and ultimately transplantation.

The study, published online in the Proceedings of the National Academy of Sciences, is the first time that an explanation has been given for the difference of disease progression in Joubert syndrome patients.

Significant breakthrough

The Newcastle research has identified a second gene called BSND a 'modifier gene' which determines the severity of kidney disease in patients with CEP290 mutations of Joubert syndrome.

It has been assumed that these modifier genes exist, but they have never been found before in rare genetic conditions until now.

Professor John Sayer and Dr Colin Miles, from the Translational and Clinical Research Institute, Newcastle University, led the Medical Research Council-funded research.

Professor Sayer said: "We have shown, using mouse and human DNA samples, that BSND is a modifier gene for the severity of kidney disease in Joubert syndrome.

"This is the first time that a modifier gene for inherited kidney disease has been identified, and this information will improve diagnoses and will be used to develop therapies to reduce the severity of kidney disease in affected patients.

"Our research is a major step forward and, in the future, we may be able to offer a therapy that switches on the protective modifier gene and reduces the development of genetic kidney disease.

"This work paves the way towards personalised therapies in patients with inherited kidney disease."

The international study used mouse models and DNA samples from patients with Joubert syndrome to progress the research.

Scientists used mouse models of disease and genetic manipulation to see how the kidney disease responded to modifier gene manipulation, cross-referenced with DNA sequencing data from patients around the world to prove the modifier gene was relevant in humans.

Challenging disease

Professor Sayer, a Consultant Nephrologist at Newcastle Hospitals NHS Foundation Trust, said: "The treatment of genetic kidney disease is challenging, as this requires both the correction of the underlying gene defect and the delivery of the treatment.

"We have shown that the kidney disease in a mouse can be dramatically changed by switching on or off a modifier gene.

"This will mean that we can use this information to carry out treatments, including genetic therapies, to lessen the effects of inherited kidney diseases, such as Joubert syndrome.

"We are testing these treatments further in our model systems before we move into patient studies."

Within the next three years, research will start to test the treatment of patients with modifier genes in the hope of developing personalized treatment plans.

Patient story

Siblings Emma, 11, and Ben Buckley, eight, have Joubert syndrome and both developed kidney failure before the age of eight.

They were diagnosed with Joubert syndrome from a few months of age and both have required dialysis and a kidney transplant.

They suffer from a range of medical issues due to Joubert syndrome, including visual impairment, communication problems, and developmental delay.

The two children, of Whitley Bay, North Tyneside, have been instrumental in helping further the research over the years, allowing the Newcastle scientists to study the mutation in detail.

Parents Leanne and Michael say they welcome the findings of the Newcastle University-led study as it will help to give patients a chance of preventing kidney failure in the future.

Leanne said: "It is very important that research is done into Joubert syndrome and the linked kidney damage, as this will hopefully prevent patients in the future needing a kidney transplant.

"All throughout Ben and Emma's lives, they have lived with the effects of Joubert syndrome and scientists found they had a problem with the CEP290 gene.

"Both Ben and Emma have needed dialysis and kidney transplants because of their kidney problems and I would like to hope this research will help prevent kidney failure for other affected children.

"We were happy for Ben and Emma to provide samples for the study as anything that helps further understanding into the condition is well worth doing, so it's great to see the study's positive results."

Download The Devdiscourse News App for Latest News.

Continue reading here:
Discovery of gene that modifies severity of inherited kidney disease - Devdiscourse

About a decade ago, the ill-fated therapeutic cancer vaccine Provenge was approved, eventually bankrupting its developer Dendreon. Since then, a number of drugmakers have seen similar efforts splutter and fizzle, although the emergence of immunotherapies checkpoint inhibitors and CAR-T drugs offered a glimmer of hope in resuscitating the field. Banking on that promise is PDC*line Pharma, which secured a 20 million injection on Wednesday.

Spun out of the French Blood Bank, the Belgian-French biotech is developing off-the-shelf cancer vaccines that are based on a therapeutic cell line of plasmacytoid dendritic cells, which are sometimes referred to as the Swiss army knife of immune cells due to their diverse range of function. PDC*line Pharmas technology is being developed to be synergistic with checkpoint inhibitors.

Scientists initially hoped that turbocharging the immune system to battle cancer cells would be enough to shrink tumors. But that didnt quite occur since cancer cells possess the ability to put brakes on that immune assault.

In the case of Provenge, data showed the product helped prostate cancer patients live longer but there was no evidence of tumor shrinkage or cancer cell death. Still,Wall Street tagged it with blockbuster expectations. But its adoption was restricted by its complex autologous therapeutic administration, high price tag and changing treatment landscape. Since then, a number of other therapeutic cancer vaccine makers have crashed and burned including Argos Therapeutics and Bavarian Nordic.

A few years ago, the emergence of checkpoint inhibitors which are engineered to unleash the immune system emerged as the perfect partner in crime for the therapeutic cancer vaccine. That is what PDC*line Pharma hopes its vaccine will accomplish.

Its lead product is currently in a Phase I/II study in patients with the most common form of lung cancer. Enrollment is expected to be completed by 2022, chief Eric Halioua told Endpoints News.

Provenges complicated autologous administration, cost of goods, batch-to-batch variability and underwhelming efficacy all contributed to its tepid sales, Halioua suggested, indicating that PDC*line Pharmas plan to incorporate a checkpoint inhibitor into the equation would likely be beneficial.

You need a good vaccine to activate (the) immune system, and you need something to break the defense mechanisms of the tumor. And all the first generations of vaccines were not in these situations.

Although the company is initially testing its off-the-shelf vaccine as a monotherapy, eventually a checkpoint inhibitor will be added to the mix.

But caution is warranted: Efforts to combine therapeutic cancer vaccines and checkpoint inhibitors have also met with failure. Last month, French biotech Transgene abandoned its therapeutic lung cancer vaccine after mid-stage data showed that when tested alongside Bristol-Myers Opdivo, the combination did not significantly shrink tumors.

Altogether PDC*line Pharma which recently signed a 108 million licensing deal (plus royalties) for its lung cancer vaccine in South Korea and other Asian regions has raised more than 30 million since its inception in 2014.

In this latest round of Series B funding, PDC*line Pharma lured five new investors: Korean Investment Partners, as well as two South Korean funds Shinhan-Cognitive Start-up Fund and UTC 2019 BIOVENTUREFUND in addition to two Belgian funds, SRIW (The Regional Investment Company of Wallonia) and Sambrinvest (the investment fund of Charleroi).

The new investors joined existing investors SFPI-FPIM, the Belgian Federal Holding and Investment Company, Noshaq Group, the Financire Spin-off Luxembourgeoise/INVESTSUD Group, among other undisclosed names.

Read more:
French-Belgian biotech banks 20M to break ground in blood-splattered field of therapeutic cancer vaccines - Endpoints News

New research has found that the cell and gene therapy clinical environment in the UK has encouraged commercial sponsorship from around the world.

Researchers have found that the UK accounts for over 12 percent of global cell and gene therapy clinical trials. Another finding revealed that these trials have increased by approximately 45 percent in the UK compared to 2018.

According to a report published by Cell and Gene Therapy Catapult (CGT Catapult), international companies are also recognising the appeal of the UK cell and gene therapy environment. The findings show they are sponsoring the majority of UK commercial clinical trials, which account for 77 percent of the total 127 ongoing trials. This is an increase from the 25 percent of commercially sponsored trials in 2013.

The researchers suggest that the National Health Service (NHS) and UK ecosystem are providing the right platforms to allow innovative therapies to progress through to the clinic in ever increasing numbers.

Keith Thompson, CEO of CGT Catapult said: The total number of cell and gene therapy clinical trials in the UK has been increasing consistently by an average of 25 percent year-on-year since 2013. This has been enabled by the development of the UKs fantastic ecosystem to support the development and clinical adoption of cell and gene therapies. The infrastructureand initiatives that have been put in place, with strong backing by the government, including the Advanced Therapy Treatment Centrenetwork, are giving companies the confidence to setup and run their innovative clinical studies here. The result is that we are now seeing therapies moving from academic projects towards becoming commercial products that can be delivered at scale by the NHS.

The report highlights that the main indication for cell and gene therapy clinical trials remains oncology at 39 percent, followed by 13 percent ophthalmology and 12 percent haematology.

Health Minister Baroness Nicola Blackwood said: These extraordinary figures show the UKs life sciences sector is leading the world in getting cutting-edge treatments to NHS patients as quickly as possible.

Go here to see the original:
UK accounts for 12 percent of global cell and gene therapy clinical trials - European Pharmaceutical Review

GenScript Biotech Global Forum Highlights Advances in Cell and Gene Therapy and Opportunities in China

GenScript Biotech Corp., a leading global biotechnology group and a pioneer in the field of gene synthesis, held its inaugural "Global Forum on Cell & Gene Therapy and the Booming China Market," during the JP Morgan Healthcare Conference week, attracting hundreds of industry leaders, investors and others to address the challenges and opportunities in this innovative field.

"As an industry, we are on the brink of achieving some extraordinary breakthroughs in cell and gene therapy for cancer and other diseases," said GenScript Biotech CEOFrank Zhang, PhD. "Four gene and cell therapies have recently been approved by the FDA, bringing new hope to patients, and this is only the beginning. Our vision is to make cancer a chronic or curable disease rather than a deadly one, and to transform the treatment of cancer, autoimmune and other diseases by leveraging the advantages of cell and gene therapy."

While significant advances are being made, the Forum also tackled some of the more pressing challenges, such as mitigating treatment side effects, improving treatment efficacy in solid tumors and scaling up manufacturing. Panelists from Kite Pharma, GE Healthcare Life Sciences, Ziopharm Oncology, Oxford Biomedica, Genethon, CARsgen Therapeutics, J&J Innovation Asia Pacific, the American Society of Gene & Cell Therapy, Loncar Investment, Lilly Asia Ventures, and many others participated in the event.

In the U.S. alone, the U.S. Food and Drug Administration is expected to approve 40-60 cell and gene therapies by 2030. During a panel discussion focused on regulatory issues, experts considered what regulators will need to do to keep up with the rapid pace of innovation, the new hospital-based regulatory pathway inChina, how to ensure quality through the manufacturing process, and the challenges and opportunities that come with regulatory harmonization among different countries.

Chinacontinues to attract significant attention from industry and investors and is poised to grow even more. During his welcoming remarks, Zhang notedChina'semergence as a global economic leader, with a projected$1.1 trillionspend on healthcare this year, as well as the growing disease burden inChina. By 2030, an estimated 4.3 million Chinese will be diagnosed with one of the 14 major cancers, according to research from IMS Health. Panelists addressed issues such as the amount of capital required to achieve scale inChina, and advantages of the market inChina.

"The drug development business is changing rapidly andChinais at the fore in a number of ways," Zhang said. "Biotech and pharma companies do not need or desire to have the infrastructure to scale their drugs through commercialization. With lower costs,Chinais a natural place for companies to contract out costly development and manufacturing to organizations that have the expertise and experience to collaborate with them through the entire discovery to development lifecycle."

For its part, GenScript has put significant resources into its Contract Development and Manufacturing Organization (CDMO) business to meet the increasing demand. In 2018, the company officially launched its biologics CDMO segment, and last year opened a new GMP compliant biologics research center. GenScript is also leading the way in cell therapy through its antibody discovery service and plasmid and virus production capabilities.

View post:
Advances in Cell and Gene Therapy and Opportunities in China - BSA bureau

A new team at Medical College of Georgia is taking aim at defective gene sequences that cause disease using the revolutionary CRISPR method that allows precise editing of specific sequences to create animal models of those diseases.

Drs. Lin Gan and Joseph Miano sit at a conference table outside their new lab at Augusta University, and in the blink of an eye the discussion goes from how they began using a certain technology in its early days of 2013 to how they are applying the latest iteration that came out only last month.

Thats how fast they, and their field of genome editing and manipulation, are moving.

Gan and Miano and colleague Xiaochun Long were recently recruited as a team to the Medical College of Georgia at AU, where Gan became the founding director of the Transgenic and Genome Editing Core. He is a Georgia Research Alliance Eminent Scholar in Neuroscience, and Miano is a J. Harold Harrison Distinguished University Chair in Vascular Biology.

The floor of their lab shows they are only a few months into the new stint in Augusta.

Were still living out of boxes, Miano said as he stepped around cardboard boxes.

The two have been together since the early 1990s in Houston and spent the past 20 years working together at the University of Rochester, though they had no idea they were both headed there initially.

Unbeknownst to me, he was being recruited at the same time, Miano said.

Were meant to be together and we didnt even coordinate our move, Gan said jokingly.

The move to Augusta was deliberate. Miano had interviewed at MCG in 1995 and wanted no part of it then and was not interested again after recently giving a talk at the school and receiving some initial offers.

But then I came down for a second visit and I thought, Hmm, this is interesting, Miano said. They have really built it up well. Theres an opportunity here to make a difference.

But the three were a package deal Long and Miano are now married, and Gan would not have come without them.

I also see this as an opportunity, Gan said. I can definitely have more support here for the Core.

He was quite familiar with MCG and in particular the eye researchers there, which is also an interest for him.

There is a very strong eye group here, Gan said, and he has been supplying them with animal models of disease for more than a decade. Creating those animals models, where a gene may be knocked out or silenced, got a lot easier for him with the advent of CRISPR, or clustered regularly interspaced short palindromic repeats.

It is the ability to take a short length of genetic material known as a guide RNA, which will match up with a highly specific target in DNA and in many instances attach an enzyme that then precisely cuts the DNA at that spot, allowing researchers to target very specific sections of that DNA and either silence genes or in some cases begin the replacement of defective sections.

Gan and Miano use CRISPR to create animal models in mice of human disease that other researchers can use to study those diseases. They often involve a small mutation in the genetic material that gives rise to the condition, known as a single nucleotide polymorphism or SNP.

CRISPR is very effective for SNPs, for mutations, Gan said. It is a little quicker.

There are a lot of them, and more are being added all of the time, Miano said.

The latest database of SNPs, that last time I looked was over 600 million, he said.

Miano was at the conference in 2012 when Dr. Jennifer Doudna of the University of California, Berkley made what he called a jaw-dropping revelation that a bacterial defense system against viruses allows for the precise targeting and cutting of DNA. CRISPR soon became a hot new technique that he and Gan adopted the following year.

Though they are primarily working in mice, CRISPR can be used in other animal models and outside the field as well, Miano said.

I think what is exciting, as Lin says, anything with a genome can be edited, he said. The agricultural field is blowing up. There are all kinds of stuff going on in agriculture with editing.

It has already reached the human level. The National Library of Medicines database of clinical trials involving CRISPR-aided therapy or testing lists 20 active studies, although only a handful are in the U.S. and most are in China. Therapies are most likely to focus first on what Miano called the low-hanging fruit, the 7,000 or so diseases that arise from a single gene defect, such as cystic fibrosis.

More complex conditions, such as heart disease, are going to be tough, he said.

There is no silver bullet for those, but CRISPR will be at the center of that work, he said.

For his part, Gan would like to focus on the eye. The tissue there is thinner and more concentrated, so correcting a defect in vision in, for instance, the macular area of the retina at the back of the eye involves manipulating much fewer cells than trying to correct something in the brain or elsewhere.

This is great for manipulation because a lot of our vision comes from the macular region, Gan said. Its a very small region. That is why he believes in terms of therapies, the eye will be one of the first.

See the article here:
Genome editing heralds new era of disease research, therapy - The Augusta Chronicle

All News

January 13, 2020

We expect that a satellite cell with the corrected DMD gene would quite quickly and continuously propagate the edited gene throughout the muscle tissue, said Prof. Amy Wagers, who leads the research. (Photo credit: Jon Chase/Harvard Staff Photographer.)

Cambridge, Mass. January 13, 2020 Harvard University stem cell researchers led by Amy Wagers, PhD, are embarking on a major study of Duchenne muscular dystrophy (DMD). Supported by research funding from Sarepta Therapeutics, under a multi-year collaboration agreement coordinated by Harvards Office of Technology Development (OTD), the project aims to use in-vivo genome editing, in mouse models of DMD, to fully and precisely restore the function of the dystrophin protein, which is crucial for proper muscular growth and development. Approaches validated by this work may point the way to an eventual therapeutic strategy to reverse DMD in humans.

Duchenne muscular dystrophy is a genetic disease caused by the lack of a protein called dystrophin that normally helps to support the structural integrity of muscle fibers, including those in the heart. Without the dystrophin protein, cells are weaker and degenerate more quickly. Over time, affected individuals boys, typically, as it is a recessive X-linked disorder lose their capacity to move independently.

Its really a devastating disease; it robs young boys of their capacity to be young boys, said Wagers, who is the Forst Family Professor of Stem Cell and Regenerative Biology, Co-Chair of the Department of Stem Cell & Regenerative Biology, and an Executive Committee Member of Harvard Stem Cell Institute. Though it is early days, Im hopeful that through this work we may identify and validate new avenues for therapy to completely rescue the proper expression and function of the dystrophin protein and regenerate healthy muscle tissue.

Researchers worldwide have pursued a variety of promising approaches such as cell and gene therapies, small-molecule therapies, and others to lessen or prevent the disease and improve patients quality of life.

The strategy pursued by the Wagers Lab at Harvard aims to fully correct the genetic template for dystrophin at its source, in the DNA of stem cells (satellite cells) that create and regenerate muscle cells. Combining cutting-edge CRISPR/Cas9 genome editing technologies with a deep knowledge of stem cell science and regenerative biology, this approach if successful might offer a permanent restoration of muscular function.

In skeletal muscle, muscle fibers are terminally post-mitotic, meaning they cannot divide and they cannot reproduce themselves, Wagers explains. If you lose muscle fibers, the only way to produce new muscle is from stem cells, specifically the satellite cells. The satellite cells are self-renewing, self-repairing, and ready to spring into action to create new muscle fibers. So we expect that a satellite cell with the corrected DMD gene would quite quickly and continuously propagate the edited gene throughout the muscle tissue.

At present, research conducted in mice has shown promising results. In June, the Wagers Lab published the results of editing stem cells in vivo, demonstrating that stem cell genes can be edited in living systems, not only in a dish. In that work, Wagers and her team delivered genome editing molecules to the cells using adeno-associated viruses (AAVs). Her lab has also successfully used gene editing in heart, muscle, and satellite cells to partially restore the function of the DMD gene that encodes dystrophin, by chopping out faulty sequences of code that are disrupting the proper reading frame.

The new stem-cell approach pursued in collaboration with Sarepta would build on these achievements and use more precise genome editing approaches, in animal models of DMD, to entirely replace genetic mutations in the DMD gene with correctly encoded sequences. The project will also explore alternate delivery methods and strategies to mitigate immune effects of in vivo genome editing.

This ambitious project will benefit greatly from the resources and insights of a company with deep clinical experience in the development of therapeutics for muscular dystrophy, said Vivian Berlin, Managing Director of Strategic Partnerships at Harvard OTD. Preclinical discoveries by Harvard researchers may open entirely new possibilities for lifesaving treatments in the long run, offering much-needed hope to patients and families in the future. Were grateful to be able to sustain the important momentum already established in Prof. Wagers lab, through this collaboration.

As we work to bring forward new treatments for patients with DMD, Sarepta is excited to support Prof. Wagers and her lab to accelerate the development of a gene editing approach, which has shown significant potential in early studies, said Louise Rodino-Klapac, Sareptas Senior Vice President of Gene Therapy. This multi-year collaboration is part of Sareptas broader commitment to pursuing all therapeutic modalities and advancing our scientific understanding of gene editing in order to maximize the potential of this approach to help patients.

Under the terms of the agreement between Harvard and Sarepta, the company will have the exclusive option to license any arising intellectual property for the purpose of developing products to prevent and treat human disease. As with any research agreement facilitated by OTD, the right of academic and other not-for-profit researchers to use the technology in further scholarly work is preserved.

All News

Read the original:
Muscular dystrophy collaboration aims to correct muscle stem cells' DNA - Harvard Office of Technology Development

SAN FRANCISCO Novartis sparked a new round of drug pricing criticism last May when the Swiss pharma revealed its new gene therapy Zolgensma would cost $2.1 million per patient.

In the eight months since then, however, insurers have reimbursed treatment for all but one eligible infant who have received the one-time therapy, according to company CEO Vas Narasimhan, speaking Monday at the J.P. Morgan Healthcare Conference.

Commercially, it seems, Novartis' launch has gone according to plan.

But the drugmaker is still awaiting the Food and Drug Administration's judgment on a scandal that erupted last summer following revelations preclinical testing data for Zolgensma had been inappropriately altered. And a clinical hold from the regulator on another study of the gene therapy has slowed plans for expanding treatment into older children.

Resolving both will be key tests in 2020, as will be expanding newborn screening for spinal muscular atrophy, the genetic disease that Zolgensma treats.

David Lennon

Novartis

This year could also feature the start of human testing for two new gene therapies from AveXis, the biotech developer of Zolgensma that Novartis bought in 2018 to gain access to its drug pipeline.

AveXis President David Lennon spoke with BioPharma Dive about the challenges the business is facing, along with how Novartis' ownership has accelerated its ambitions.

This interview has been condensed and edited for clarity.

What's the latest progress on newborn screening for spinal muscular atrophy?

LENNON: It's still a key priority with the states, as it was approved last July. There's been a lot of progress. Sixteen states have implemented newborn screening that represents about 32% of new births in the U.S. that are screened. That's our internal estimate of what we see. We estimate that number will get to 70% over the course of this year as more states implement it.

There's an amazing impact when you have newborn screening. In states where we have newborn screening, [there is] a two- to three-fold higher utilization of gene therapy for newborns than in states where we don't. A lot of that has to do with, when newborn screening gets set up, a dedicated referral pathway to the best institutions also gets created. The parents are informed of the diagnosis and get referred to a geneticist to get counsel.

Do you think the newborn screening process should be reformed, given the state-to-state disparities?

LENNON: It's a travesty. You're just waiting for these kids to present clinically. The bureaucracy that limits the adoption of these kinds of policies is silly in many cases.

RUSP [The Recommended Uniform Screening Panel] recommends and has a two-year implementation requirement that's soft, there's no enforcement around it. It's up to the states to put it in place. Implementation often takes active patient groups and an active physician to advocate for it getting into the state.

How do you grade Novartis' job on communicating Zolgensma's price to payers and the public?

LENNON: With payers, which were the folks we had to really convince, we did an outstanding job. Payers understand the value it provides, they readily wrote policies to cover the product even with a broad label at launch, very positive discussions and good coverage in general very early. I'd give us strong marks on the payer side.

In the public domain, I think it's more problematic. Healthcare cost transparency and understanding of healthcare costs in the general public is not great. It's very easy to just go to the extreme and say, '$2.1 million, I don't understand; that must be ridiculous.' If I was to say it cost $2 million to do a heart transplant, people might say 'I totally get that.'

We anticipate about 300 or 400 [will be treated with Zolgensma], basically the same number of pediatric heart transplants that get done every year. When you start to try to draw some comparisons, it quickly gets lost in the 'but it's $2.1 million dollars.' We have to do a better job of helping people generate relevant transparency that allows us to put these things in context.

How did you choose the pipeline products to prioritize?

LENNON: Our next two programs take on different challenges and opportunities. One is Rett syndrome, where organic [gene] expression needs to be much more targeted. If we overexpress this protein, you actually create a similar syndrome to the one we are trying to address. There's a kind of 'Goldilocks' area of expression. That program is using a promoter that is more controlled and has a natural feedback mechanism that limits the amount of expression.

The third program, called AVXS-301, is for a genetic form of Lou Gehrig's disease amyotrophic lateral sclerosis or ALS. That is actually an inhibitory construct. The first two programs we've had are gene replacement the gene's defective, we're adding back protein. In this case, the defect leads to overexpression or active expression of a mutated form of SOD1 that drives inflammation and degradation of the neurons of these patients. We are inhibiting that by introducing a short hairpin RNA that actually inhibits the expression of the defective gene.

Those programs existed as part of the acquisition of AveXis. Since then, we started partnering with [the Novartis Institutes of Biomedical Research] to apply this platform to different diseases. The first one we announced is a project for Friedreich's ataxia, a muscle-wasting disease.

You took over AveXis in 2018, coming from Novartis. What was AveXis particularly skilled at doing, and what's an area that Novartis' ownership could help in?

LENNON: Technically, this organization was very skilled at taking a problem, breaking it down, developing options to address it and agreeing on the best solution forward. It was an organization that was optimistic it could continually find that. Whether you believe in optimism or luck, the organization was very effective at finding that.

Where the organization really benefits from the Novartis backbone is then: How do you take the process that you developed and make that an industry-leading, highly reproducible and globally scalable system? That's where Novartis is brilliant. That experience has allowed us to bring in quality systems, management talent that knows how to scale, and financial wherewithal that allows us to scale to now multiple sites around the country.

When you look at AveXis, we could do one thing at a time. Now we can start to do seven or eight things at a time, because Novartis has the backbone and scale because they're used to managing 143 projects at once.

Ned Pagliarulo contributed to this article.

Read the rest here:
Novartis' David Lennon on next steps for AveXis, selling Zolgensma's price to the public - BioPharma Dive

19 January 2020 11min read

But ethical and economic challenges could limit the benefits medtech brings to healthcare.

The Nobel Prize in Medicine 2019 was awarded to three men for their discoveries of how cells sense and adapt to oxygen availability. The Nobel Prize in Medicine 2018 was given to two men for their discovery of cancer therapy by inhibition of negative immune regulation. The Nobel Prize in Chemistry 2017 went to a trio for developing cool microscope technology that revolutionises biochemistry by allowing researchers to study three-dimensional structures of biomolecules in their search for a cure for the Zika and other viruses. One of the three chemistry winners of 2017, Joachim Frank of the US, said at the time that he thought the chance of winning a Nobel Prize was minuscule because there are so many innovations and discoveries happening.

Frank is still right about that. Technological leaps in medicine dubbed medtech are accelerating as researchers find better ways to treat more diseases, in more ways, for more people. Advances are occurring in biotechnology, immunotherapy, surgery, and foetal and neonatal care to name just some areas. Artificial-intelligence software trained on data from digitalised health records and devices can spot problems faster and more reliably than can humans. HCA Healthcare, the largest for-profit hospital operator in the US, for instance, now uses algorithms trained on 31 million cases to detect the sepsis infection that kills about 270,000 people a year in the US.

More medtech advances are certain. Money is pouring into research and development overseen by regulators and doctors to ensure benefits outweigh risks. Common sights soon might be robot physicians, remote surgery and mini 3D-printed organs. Bacteria genetically reprogrammed to destroy tumours in mice could one day work on humans. Genome scans and gene therapies could become routine.

For all this promise, however, medtech comes with two certain and one likely drawback. The first definite disadvantage is that medtech is raising ethical issues that could stop the deployment of key advances. The two most sensitive are the gene-editing of foetuses (superbabies) that could alter human experience and protecting the privacy of patient data, an issue highlighted in November when it emerged that US healthcare provider Ascension had secretly handed over the records of tens of millions of patients to data crunchers at Google. Medtechs other certain shortcoming is the cost. Many advancements might never become mainstream because they could prove too expensive for governments burdened with budget deficits and heavy debt loads that are already facing rising healthcare costs as their populations age.

Medtechs contentious disadvantage is doctors are finding that self-monitoring via devices, which often detects harmless abnormalities and fuels hypochondria, is leading to unwarranted anxiety, incorrect diagnoses and unneeded treatments. All up, medtechs value to society will be tied to the extent to which these disadvantages limit the spread of its unquestionable benefits.

Many of medtechs ethical issues could be resolved, to be sure, but that wont be easy. Some medtech advancements, especially those based on AI, are economical. Medtech needs to be assessed with the perspective that there is much it is not solving. Medtech advances, for example, arent enough to avert the recent decline in life expectancy in western countries due to heart attacks tied to obesity. Medtech pharmaceutically does little for autoimmune diseases such as arthritis that afflict one in four US adults though it is improving joint-replacement surgery. Researchers are yet to find a cure for infections made drug-resistant due to the overuse of antimicrobial drugs that the World Health Organisation says could kill 10 million people a year by 2050. Nothing medtech has come up with is usurping MRI scans and X-rays.

Be these as they may, medtech advancements are ushering in treatments that produce better outcomes for patients. Only time will tell how much ethical, economic and other possible drawbacks limit mainstream access to medtechs benefits.

The biotech era

Eras become known for their medical advancements. From the 1920s to the 1950s, for example, the key medical leaps were vaccines and antibiotics. Later epochs might regard todays advances to be centred on cell and gene therapy, robotic surgery and perhaps AI.

Hope for cures from gene therapy, an area of research that emerged from the late 1980s, accelerated in the early 2000s when the human genome was sequenced. And treatments are underway now and more are likely. Biopolymers (nucleic acid) are injected into cells to treat inherited eye diseases and immune deficiencies while researchers are studying how gene therapy could treat cancer, heart disease and diabetes. A stellar example of gene therapy improving lives is that a Novartis subsidiary has developed a one-time gene-based treatment (Zolgensma) that is a curing treatment for children born with spinal muscular atrophy (who without this advance constantly need treatment over their short lives). The problem is one dose costs US$2.1 million.

Aside from the costs, gene therapy comes with other challenges too. The finicky nature of genes has made progress slow. Other hindrances are rejection, side effects such as cancer, and the risk that other genes might be delivered to a cell. Some treatments are so risky authorities have halted them. Some breakthroughs have proved false a recent study debunks that a certain gene causes depression. That the ethical issues surrounding gene therapy are unresolved became an urgent issue in 2018 when two Chinese babies were born with modified genes.

Inventions to assist surgeons have proved faster to everyday use (and less problematic). Robots have aided orthopaedic surgeons since the mid-1980s and now help with general, transplant, urological and other procedures. One measure of their widespread use is that Intuitive, the US-based maker of the 1999-launched da Vinci surgical system, counts that tens of thousands of surgeons have conducted more than six million procedures in at least 66 countries using its equipment. The benefits of robotic-assisted surgery are less invasive, more precise and safer procedures due to fewer and tinier incisions (microsurgery) and reduced human error.

While less-invasive surgery shortens hospital stays and robotic surgerys lower margins of error reduce the need and costs of further treatment, robotic-assisted procedures are expensive. Assuming cost issues can be overcome, technology will expand its role in surgery and robots could use AI more extensively to help surgeons make more decisions.

AIs use in healthcare goes well beyond surgery too. AI programs including chatboxes are diagnosing heart disease and cancer, identifying retinal damage, analysing suicide risk, streamlining drug-development processes, proposing remedies for multiple sclerosis, even helping the dumb speak. AI s promise is more timely, economical, convenient and streamlined treatments.

AIs usual drawbacks apply, however. Personal data needs privacy protection, which can impede research. Data can be dodgy and data-training algorithms can be flawed and biased, which could lead to misdiagnosis. AI is vulnerable to hacking, whereby malicious tweaks lead to errors. AIs deployment often runs ahead of peer review and ethical considerations.

A neurotic world?

One medtech achievement is to elevate the practitioner Doctor Me. The term (sometimes stated as Doctor You) is for when people use devices and self-testing to monitor their health or genetic risks.

Self-monitoring comes with many advantages. It can save lives. The unwell can gain comfort if their vital signs are normal. The data collected can help everyones health and allow people to find others with similar issues, which could provide clues for treatments and moral support.

The problem, however, is that Doctor Me has ushered in the nocebo effect, essentially a form of hypochondria. The nocebo effect occurs when patients think they are experiencing a side effect to a greater degree than possible or when people fret they are suffering from an ailment that a test showed they are at risk of say people self-tested as prone to Alzheimers imagine they have the affliction when they forget something.

A Stanford study of 2018 found the nocebo effect is ripe in self-testing genetics, a flagship area of medtech that is not foolproof. The expression could become ubiquitous soon because more people are testing their disposition to Alzheimers, cancer and obesity by 2017, already one in 25 in the US knew their genetic data. If the nocebo effect becomes widespread, authorities may need to limit self-testing.

While future Nobel Prizes await those making medtech advances, perhaps others lie ahead for those who find ways to resolve medtechs ethical, economic and hypochondriac challenges.

Published by Michael Collins, Investment Specialist, Magellan Group

See the original post here:
Technology is reshaping modern medicine - TheBull.com.au

As the annual JP Morgan Healthcare Conference draws to a close, lets celebrate the remarkable innovation that drives the biopharma industry. Heres a look at the top 10 companies noted in the BioSpace Ideal Employer 2019 survey for being the most innovative and what theyve been up to recently.

Regeneron Pharmaceuticals. Ranked as the top innovator in the survey, Regeneron has long had a reputation for innovation, and is often cited as Sanofis innovation engine because of its numerous collaborations with the French-based company. Regeneron has seven marketed products, including Arcalyst for rare autoinflammatory disease, Eylea for a common cause of blindness, Praluent for high cholesterol, and Dupixent for atopic dermatitis. It also has REGN-EB3, a three-antibody therapy used to treat Ebola.

On January 9, Regeneron announced results from LUMINA-1, a Phase II trial of garetosmab in patients with fibrodysplasia ossificans progressive (FOP), an ultra-rare genetic disorder that leads to abnormal bone formation. After 28 weeks, the drug decreased total lesion activity compared to placebo by 25%.

Verily Life Sciences. Formerly known as Google Life Sciences, Verily is Alphabets life science research company. It was originally a division of Google X. At this point, the company doesnt have any marketed products and its not completely clear on the companys overall focus. It has numerous partnerships, such as one with Sanofi to develop products for managing diabetes, a disease-detecting nanoparticle platform called project Tricorder, and a partnership with Johnson & Johnson on surgical robotics. It also has partnerships with Alon, 3M, Allergan, Biogen, Dexcon, GlaxoSmithKline, Mayo Clinic, Brigham and Womens Hospital and many others.

On December 23, 2019, Verily partnered with Emory Healthcare to deploy new solutions to help improve cost-effectiveness, operational efficiency and quality. This deal with utilize Emorys academic medical center and partner with Verilys expertise in data science, analytics, user experience and product development. The initial focus is a deep analysis of existing drugs and lab-ordering patterns at Emory.

Illumina. Illumina develops, manufactures, and markets laboratory devices, with particular emphasis on DNA sequencing, genotyping, gene expression and proteomics. On January 4, 2020, Illumina announced a 15-year, non-exclusive deal with Roche. Not only will that increase the availability of next-generation sequencing-based in vitro diagnostic (IVD) tests on Illuminas systems, but the two companies will collaborate to complement Illuminas pan-cancer assay TruSight Oncology 500 (TSO 500) with new companion diagnostic (CDx) claims.

That deal came only a short time after Illumina canceled a $1.2 billion merger with another next-generation sequencing company, Pacific Biosciences (PacBio). Illumina decided the deal was not likely to be approved by antitrust regulators in the U.S. and UK. Illumina holds about 80% of the global DNA sequencing market.

bluebird bio. Bluebird bio focuses on the nascent field of gene therapy. Currently its sole approved product is Zynteglo. It was approved by the European Commission (EC) on June 14, 2019 for patients 12 years or older with transfusion-dependent beta-thalassemia who did not have a 0/0 genotype and for patients where hematopoietic stem cell (HSC) transplantation wasnt appropriate, but a human leukocyte antigen (HLA-matched related HSC donor isnt available. Its a little difficult to mention bluebird without mentioning the price of Zynteglo, which is $1.8 million in Europe. In addition to its scientific innovation, bluebird bio is innovative in terms of pricing structure. Zynteglos price is spread out over five years, with an initial upfront price of 315,000 euros with the four additional yearly payments due only if the treatment continues to work.

The product launched in Germany on Jan. 13, 2020. Bluebird initiated the rolling Biologics Licensing Application for Zynteglo in the U.S. and is currently in discussions with the U.S. Food and Drug Administration (FDA) on the timing and various components of the submission. They hope to complete the BLA submission in the first half of this year.

Biogen. Biogen specializes in therapies for central nervous system disorders. Some of its most well-known products are Alprolix for hemophilia B, Avonex, Fampyra, Tecfidera and Tysabri for multiple sclerosis, and Spinraza for spinal muscular atrophy (SMA). However, most recently, the company has been in the headlines for its aducanumab for Alzheimers disease. The drug was declared a failure in March 2019, but was resurrected this year after some of the later trial data showed effectiveness at the highest dose. Still, its not a slam dunk to be approved by the FDA and there are plenty of skeptics.

Analysts expect Biogen to submit aducanumab to the FDA in a matter of weeks, although the company is being tight-lipped about the timetable.

Bayer. Based in Germany, Bayer is one of the largest pharma companies in the world. In 2018, Bayer acquired U.S.-based Monsanto, which no longer exists under the Monsanto name. The companys business units include Bayer Crop Science, Consumer Health, Pharmaceuticals, Animal Health and Business Services.

On Jan. 16, 2020, Bayer sold one of its last Germany-based manufacturing facilities to Shanghai, China-based WuXi Biologics. The plant will be run by WuXi Biologics and act as a backup site for the manufacture of Bayers Kovaltry (antihemophilic factor). The primary site for Kovaltry product is Bayers facility in Berkeley, California.

Novartis. Based in Switzerland, Novartis has a well-known portfolio of drugs, including Clozaril, Voltaren, Tegretol, Diovan, Gleevec, and Ritalin. Its Sandoz Division is a global leader in generic drugs and biosimilars.

Last year, the FDA approved Zolgensma, a gene therapy for SMA, which was developed by its subsidiary, AveXis. Although there was some controversy over data manipulation in preclinical studies, it was determined not to affect the safety or efficacy of the therapy.

In late November 2019, Novartis acquired The Medicines Company for $9.7 billion, only a week after The Medicines Company announced positive data from its ORION-10 Phase III trial for inclisiran for lowering cholesterol.

GlaxoSmithKline. Headquartered in London, GSK markets drugs for numerous major diseases, such as asthma, cancer, infections, diabetes and mental health. Its best-known drugs include Advair, Augmentin, Flovent, Lamictal and others.

At the recent JP Morgan Healthcare Conference, Emma Walmsley, GSKs chief executive officer, predicted it will have six regulatory approvals in the U.S. this year.

I am pleased with the progress and the momentum that weve been able to make over the past couple years, she told CNBCs Jim Cramer.

Walmsley noted positive data for a number of programs, including ones gained from its $5.1 billion acquisition of Tesaro Oncology in 2018. She also noted a two-drug regimen for HIV that the companys subsidiary ViiV Healthcare has been developing.

Genentech. Generally viewed as the first modern biotechnology company, Genentech is a subsidiary of Swiss-based Roche, although Genentech is based in South San Francisco. It has a laundry list of successful drugs, often in the oncology market, such as Avastin, Tarceva, Zelboraf, Kadcyla, Alecansa, Venclexta and Tecentriq. It also has antivirals, such as Xofluza, Hemlibra for hemophilia A, and Esbriet for idiopathic pulmonary fibrosis.

On Dec. 12, 2019, Genentech announced that its Phase III IMspire150 trial in patients with previously untreated BRAF V600 mutation-positive advanced melanoma, hit its primary endpoint of progression-free survival (PFS). It showed adding Tecentriq to Cotellic and Zeleboraf decreased the risk of the disease getting worse or death, compared to placebo plus Cotellic and Zelboraf.

Amgen. Based in Thousand Oaks, California, Amgens best-selling products are Neulasta, an immunostimulatory for patients undergoing chemotherapy, and Enbrel, used to treat rheumatoid arthritis and other autoimmune diseases. Other products include Epogen, Aranesp, Prolia and XGeva.

On Nov. 1, 2019, the company expanded its presence in China by taking a 20.5% stake in China-based BeiGene Co. Amgen paid $2.7 billion in cash for the stake. As a result of the deal, BeiGene will commercialize Xgeva, Kyprolis and Blincyto in China.

On Jan. 13, 2020, the company inked strategic collaborations with Guardant Health and QIAGEN to develop blood- and tissue-based companion diagnostics for investigational cancer treatment AMG 510. AMG 510 is the first KRASG12C inhibitor to advance to the clinic for multiple cancer types.

Read more from the original source:
Who are the 10 Most Innovative Biopharma Companies? - BioSpace

(MENAFN - Ameliorate Solutions)

The report presents an in-depth assessment of the Global Red Biotechnology including enabling technologies, key trends, market drivers, challenges, standardization, regulatory landscape, deployment models, operator case studies, opportunities, future roadmap, value chain, ecosystem player profiles and strategies. The report also presents forecasts for Global Red Biotechnology investments from 2020 till 2025.

Industry Overview-

The Red Biotechnology Market is expected to register a CAGR of 5.7% during the forecast period. Red biotechnology is a process that utilizes organisms to improve health and helps the body to fight against diseases. Red biotechnology has become a very important part of the field of diagnostics, gene therapy, and clinical research and trials. Genetic engineering and the development and production of various new medicinal products to treat life-threatening diseases are also part of the benefits of red biotechnology. Severe Combined Immune Deficiency (SCID) and Adenosine deaminase (ADA) deficiency are genetic disorders that were successfully treated with gene therapy. Several promising gene therapies are under development for the treatment of cancer and genetic disorders. According to the World Health Organization (WHO), approximately 6,000 to 8,000 rare diseases found and out of them, nearly 80% are genetic disorders. Rising incidence and prevalence of chronic and rare diseases and increased funding in the healthcare industry are the key driving factors in the red biotechnology market.

Click the link to get a free Sample Copy of the Report:

https://www.marketinsightsreports.com/reports/01091744865/red-biotechnology-market-growth-trends-and-forecast-2020-2025/inquiry?Mode=21

Top Leading Manufactures-

Pfizer Inc, AstraZeneca PLC, F. Hoffmann-La Roche Ltd, Celgene Corporation, Takeda Pharmaceutical Company Limited, Biogen Inc, Amgen Inc, Gilead Sciences Inc, Merck KGaA, CSL Limited

Biopharmaceutical Industry Segment is Expected to Hold a Major Market Share in the Red biotechnology Market

- Biopharmaceuticals are medical drugs that are produced by using biotechnology. Biopharmaceuticals are proteins, antibodies, DNA, RNA or antisense oligonucleotides used for therapeutic or diagnostic purposes, and these products are produced by means other than direct extraction from a native (non-engineered) biological source.- The first biopharmaceutical product approved for therapeutic use was recombinant human insulin (Humulin), which was developed by Genentech and marketed by Eli Lily in the year 1982 and in the year 2019, Novartis received FDA approval for gene therapy product in the treatment of spinal muscular atrophy (SMA) condition. Using an AAV9 viral vector, called Zolgensma, which delivers SMN protein into the motor neurons of afflicted patients.- According to the World Health Organization (WHO), globally Cancer is the second leading cause of death and an estimated 9.6 million deaths in the year 2018.- Increasing incidence and prevalence of chronic and rare diseases and rapid expansion of the biopharmaceutical industries are the key driving factors in the biopharmaceutical industry segment.

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period

North America expected to hold a major market share in the global red biotechnology market due to the rising prevalence of chronic and rare diseases, increased expenditure in the healthcare industry in this region. According to the National Institutes of Health (NIH), in the year 2019, approximately 1.8 million people will be diagnosed with cancer in the United States and estimated 268,600 women and 2,670 men will be diagnosed with breast cancer. Moreover, the rise in the adoption of advanced technologies in gene therapy and increasing investments in research and development is fueling the growth of the overall regional market to a large extent.

Inquire for Discount:

https://www.marketinsightsreports.com/reports/01091744865/red-biotechnology-market-growth-trends-and-forecast-2020-2025/discount?Mode=21

Key Strategic Developments : The study also includes the key strategic developments of the market, comprising R & D, new product launch, M & A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a Global and regional scale.

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

Analytical Tools: Global Red Biotechnology 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 Porter's five forces analysis, feasibility study, and investment return analysis have been used to analyzed the growth of the key players operating in the market.

The research includes historic data from 2014 to 2020 and forecasts until 2025 which makes the reports an invaluable resource for industry executives, marketing, sales and product managers, consultants, analysts, and other people looking for key industry data in readily accessible documents with clearly presented tables and graphs.

Media Contact Us:

Irfan Tamboli (Head of Sales) Market Insights Reports

Phone: + 1704 266 3234 | +91-750-707-8687

|

MENAFN18012020007010643ID1099570077

See original here:
Red Biotechnology Market Size, Status and Recent Advancements, Forecast 2020 to 2025 - MENAFN.COM

Axovant Gene Therapies (NASDAQ:AXGT) was upgraded by stock analysts at ValuEngine from a hold rating to a buy rating in a report released on Friday, January 3rd, ValuEngine reports.

A number of other equities research analysts have also commented on the company. Chardan Capital increased their price target on Axovant Gene Therapies from $10.00 to $15.00 and gave the company a buy rating in a research report on Monday, October 28th. Zacks Investment Research raised Axovant Gene Therapies from a hold rating to a strong-buy rating and set a $6.00 price target for the company in a research report on Wednesday, November 13th. One equities research analyst has rated the stock with a sell rating, one has issued a hold rating and nine have assigned a buy rating to the companys stock. The company currently has a consensus rating of Buy and a consensus target price of $24.66.

Shares of NASDAQ:AXGT opened at $4.43 on Friday. The company has a quick ratio of 1.41, a current ratio of 1.41 and a debt-to-equity ratio of 0.69. Axovant Gene Therapies has a twelve month low of $3.81 and a twelve month high of $19.60. The stocks 50 day moving average is $5.14 and its 200-day moving average is $6.11. The firm has a market capitalization of $105.98 million, a price-to-earnings ratio of -1.00 and a beta of 1.18.

Large investors have recently made changes to their positions in the business. Tower Research Capital LLC TRC grew its stake in shares of Axovant Gene Therapies by 955.3% during the 2nd quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after acquiring an additional 3,821 shares during the period. Barclays PLC bought a new position in shares of Axovant Gene Therapies during the 3rd quarter worth $65,000. Jane Street Group LLC grew its stake in shares of Axovant Gene Therapies by 28.8% during the 2nd quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after acquiring an additional 10,375 shares during the period. Woodstock Corp bought a new position in shares of Axovant Gene Therapies during the 4th quarter worth $83,000. Finally, BlackRock Inc. bought a new position in shares of Axovant Gene Therapies during the 2nd quarter worth $1,482,000. Hedge funds and other institutional investors own 14.80% of the companys stock.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

Featured Story: What is the price-sales ratio?

To view ValuEngines full report, visit ValuEngines official website.

Receive News & Ratings for Axovant Gene Therapies Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Axovant Gene Therapies and related companies with MarketBeat.com's FREE daily email newsletter.

More here:
Axovant Gene Therapies (NASDAQ:AXGT) Upgraded to Buy by ValuEngine - Slater Sentinel

Zacks Investment Research downgraded shares of Axovant Gene Therapies (NASDAQ:AXGT) from a hold rating to a sell rating in a report released on Wednesday morning, Zacks.com reports.

According to Zacks, Axovant Sciences Ltd. is a biopharmaceutical company which focuses on the acquisition, development and commercialization of therapeutics for the treatment of neurodegenerative disorders. Its product candidate includes RVT-101 which is in different clinical trial for the treatment of Alzheimers disease and other forms of dementia. Axovant Sciences Ltd. is based in Hamilton, Bermuda.

Other equities research analysts also recently issued reports about the stock. ValuEngine raised shares of Axovant Gene Therapies from a hold rating to a buy rating in a report on Friday, January 3rd. Chardan Capital upped their price objective on shares of Axovant Gene Therapies from $10.00 to $15.00 and gave the company a buy rating in a report on Monday, October 28th. One investment analyst has rated the stock with a sell rating, one has issued a hold rating and nine have issued a buy rating to the company. The stock currently has a consensus rating of Buy and an average price target of $24.66.

NASDAQ AXGT traded down $0.22 during trading on Wednesday, hitting $4.43. 227,466 shares of the companys stock traded hands, compared to its average volume of 146,417. Axovant Gene Therapies has a 12-month low of $3.81 and a 12-month high of $19.60. The company has a debt-to-equity ratio of 0.69, a current ratio of 1.41 and a quick ratio of 1.41. The business has a 50 day moving average of $5.14 and a 200-day moving average of $6.11.

Axovant Gene Therapies (NASDAQ:AXGT) last released its quarterly earnings data on Friday, November 8th. The company reported ($0.61) EPS for the quarter, beating the Thomson Reuters consensus estimate of ($1.15) by $0.54. Research analysts predict that Axovant Gene Therapies will post -3.58 earnings per share for the current year.

A number of hedge funds and other institutional investors have recently added to or reduced their stakes in the business. BlackRock Inc. purchased a new stake in shares of Axovant Gene Therapies in the second quarter worth about $1,482,000. Tower Research Capital LLC TRC lifted its holdings in shares of Axovant Gene Therapies by 955.3% in the second quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after buying an additional 3,821 shares in the last quarter. Jane Street Group LLC lifted its holdings in shares of Axovant Gene Therapies by 28.8% in the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after buying an additional 10,375 shares in the last quarter. Barclays PLC purchased a new stake in shares of Axovant Gene Therapies in the third quarter worth about $65,000. Finally, Woodstock Corp purchased a new stake in shares of Axovant Gene Therapies in the fourth quarter worth about $83,000. 14.80% of the stock is currently owned by institutional investors and hedge funds.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

Further Reading: What are the qualifications of a portfolio manager?

Get a free copy of the Zacks research report on Axovant Gene Therapies (AXGT)

For more information about research offerings from Zacks Investment Research, visit Zacks.com

Receive News & Ratings for Axovant Gene Therapies Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Axovant Gene Therapies and related companies with MarketBeat.com's FREE daily email newsletter.

Visit link:
Axovant Gene Therapies (NASDAQ:AXGT) Downgraded to Sell at Zacks Investment Research - Riverton Roll

The Electrical Shielding Tape market is an intrinsic study of the current status of this business vertical and encompasses a brief synopsis about its segmentation. The report is inclusive of a nearly accurate prediction of the market scenario over the forecast period market size with respect to valuation as sales volume. The study lends focus to the top magnates comprising the competitive landscape of Electrical Shielding Tape market, as well as the geographical areas where the industry extends its horizons, in magnanimous detail.

The market report, titled Global Electrical Shielding Tape Market Research Report 2019 By Manufacturers, Product Type, Applications, Region and Forecast to 2025, recently added to the market research repository of details in-depth past and present analytical and statistical data about the global Electrical Shielding Tape market. The report describes the Electrical Shielding Tape market in detail in terms of the economic and regulatory factors that are currently shaping the markets growth trajectory, the regional segmentation of the global Electrical Shielding Tape market, and an analysis of the markets downstream and upstream value and supply chains.

Request Sample Report @ https://www.marketresearchhub.com/enquiry.php?type=S&repid=2560202&source=atm

The report offers the market growth rate, size, and forecasts at the global level in addition as for the geographic areas: Latin America, Europe, Asia Pacific, North America, and Middle East & Africa. Also, it analyses, roadways and provides the global market size of the main players in each region. Moreover, the report provides knowledge of the leading market players within the Electrical Shielding Tape market. The industry-changing factors for the market segments are explored in this report. This analysis report covers the growth factors of the worldwide market based on end-users.

The key manufacturers covered in this Electrical Shielding Tape market report:

Daimler AGSchmitz CargobullGreat DaneUtility TrailerPaccarVolvoWabash National CorporationFontaineHyundai TransleadNavistarKogel Trailer GmbhSchwarzmuller GroupFAW SipingHuida HeavyCIMCSinotrukLiangshan Huayu

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypeUp To 50 T50-100 TAbove 100 T

Segment by ApplicationCement IndustryFood IndustryChemical IndustryLogistics IndustryOil and Gas IndustryOther

Make An EnquiryAbout This Report @ https://www.marketresearchhub.com/enquiry.php?type=E&repid=2560202&source=atm

In accordance with a competitive prospect, this Electrical Shielding Tape report dispenses a broad array of features essential for measuring the current Electrical Shielding Tape market performance along with technological advancements, business abstract, strengths and weaknesses of market position and hurdles crossed by the leading Electrical Shielding Tape market players to gain leading position. Other aspects such as customer base, sales reach, local coverage, production price trends, and production cost layout are also analyzed to bestow accurate rivalry perspective.

Pivotal highlights of Electrical Shielding Tape market:

The Electrical Shielding Tape market report includes a brief about the cost analysis, key raw material used, as well as the fluctuating price trends of the war material.

The suppliers of the raw material and their market concentration rate have also been enlisted.

The manufacturing cost structures, encompassing details about the raw material, manufacturing process analysis, as well as labor costs have been enumerated in the study.

Substantial details about the industry chain analysis, downstream buyers, and sourcing strategies have been elucidated.

A separate section has been designated for the analysis of the marketing strategy adopted, as well details about the distributors that are a part of the supply chain.

The report is inclusive of information regarding the channels adopted for the product marketing, marketing channel development trends, pricing and brand strategies, as well as target clientele.

You can Buy This Report from Here @ https://www.marketresearchhub.com/checkout?rep_id=2560202&licType=S&source=atm

View post:
Gene Therapy Market 2020 Industry Size, Regions,Trends and Top Manufacturers Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics,...

Global Gunshot Detection System Marketwas valued US$ 920.2 Mn in 2017 and is expected to reach US$ 7120.7 Mn by 2026, at CAGR of 29.2% during forecast period.

The major growth drivers of the gunshot detection system market include the rise in demand for advanced security systems, increasing demand for security in various industry verticals, and rising criminal activities in urban areas. In addition, Increased incidences of gun-firing, especially at school premises, have led to various security measures, such as warning people in close proximity and alerting authorities to the location of the shooting all these factors will drive the growth of Gunshot Detection system market during the forecast period. Increasing government initiative side has led to the growth of the Gunshot Detection system across the globe. The latest trend for smart cities across the globe is resulting in the quantitative increase in demand of Gunshot across the nation.

Growing investment in border security is also likely to drive the market growth in the coming years, as gunshots systems can help border security forces to detect and stop any attempts of smuggling, infiltration, and illegal immigration. Also, with a little amendment of the system, it might be possible for the manufacturers to help forces identify criminals and detect illegal entry even in the absence of gunshots. Various law enforcement agencies across global are now deploying various strategies in order to control the increased incidents related to the mass shooting, which will further increase the demand and interest in advanced gunshot detection technology.

REQUEST FOR FREE SAMPLE REPORT:https://www.maximizemarketresearch.com/request-sample/29340

At the same time, the factors that are limiting the market are the scarcity of gunshot detection professionals, the high installation cost of gunshot detection systems. Also, worldwide financial uncertainty and macroeconomic situations such as currency exchange rates and economic difficulties are some of the major challenges which are hampering the growth of Gunshot Detection System Market.

Based on the installation, the vehicle installations segment is expected to account for the largest share of the gunshot detection system market over the period. The growth in the vehicle installations segment is due to the upgradation of existing armored vehicle fleets. The increasing delivery of new armored vehicles is also leading to increasing demand for armored vehicles. Increasing soldier modernization programs are also contributing to the demand for overall GDS.

The global gunshot detection systems market is mainly dominating by Raytheon products, both in military and civilian applications. Shotspotter, which sells civilian versions of Raytheons gunshot detection system through the license, generated a revenue of around XX million in 2017. The product obtaining cost for gunshot detection systems is very low. The civilian usage of gunshot detection systems is gradually increasing across various countries, with the threat from random shooters and terrorists augmenting the demand from civilians.

North America is expected to drive the growth of the gunshot detection system market during the forecast period, 2017 to 2026. The gunshot detection system market in North America is expected to witness growth, because of the largest civilian gunfire deaths in the region, especially in the US where the firearm-related death rate is 25 times higher than other countries. Major US cities are planning to install indoor gunshot detection systems at universities, corporate office locations, and financial facilities, among others, over the next few years.

The objective of the report is to present a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, industry-validated market data and projections with a suitable set of assumptions and methodology. The report also helps in understanding the Global Gunshot Detection System Market dynamics, structure by identifying and analysing the market segments and project the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. The report also provides PEST analysis, PORTERs analysis, and SWOT analysis to address questions of shareholders to prioritizing the efforts and investment in the near future to the emerging segment in the Global Gunshot Detection System Market.

DO INQUIRY BEFORE PURCHASING REPORT HERE:https://www.maximizemarketresearch.com/inquiry-before-buying/29340

Scope of Global Gunshot Detection System Market:

Global Gunshot Detection System Market, By Type:

Indoor OutdoorGlobal Gunshot Detection System Market,By Installation:

Fixed Installation Wearable Installation Vehicle Mounted InstallationGlobal Gunshot Detection System Market, By Application:

Military Law EnforcementGlobal Gunshot Detection System Market, By Region:

North America Europe Asia-Pacific South America Middle East & AfricaKey Players Operated in Market Include:

Raytheon Company Thales Group Battelle Memorial Institute Rafael Safran Electronics & Defense Rheinmetall AG ELTA Systems Ltd

MAJOR TOC OF THE REPORT

Chapter One: Gunshot Detection System Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Gunshot Detection System Market Competition, by Players

Chapter Four: Global Gunshot Detection System Market Size by Regions

Chapter Five: North America Gunshot Detection System Revenue by Countries

Chapter Six: Europe Gunshot Detection System Revenue by Countries

Chapter Seven: Asia-Pacific Gunshot Detection System Revenue by Countries

Chapter Eight: South America Gunshot Detection System Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Gunshot Detection System by Countries

Chapter Ten: Global Gunshot Detection System Market Segment by Type

Chapter Eleven: Global Gunshot Detection System Market Segment by Application

Chapter Twelve: Global Gunshot Detection System Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Gunshot Detection System Market Report at:https://www.maximizemarketresearch.com/market-report/global-gunshot-detection-system-market/29340/

About Us:

Maximize Market Research provides B2B and B2C market research on 20,000 high growth emerging technologies & opportunities in Chemical, Healthcare, Pharmaceuticals, Electronics & Communications, Internet of Things, Food and Beverages, Aerospace and Defense and other manufacturing sectors.

Contact info:

Name: Lumawant Godage

Organization: MAXIMIZE MARKET RESEARCH PVT. LTD.

Email: [emailprotected]

Contact: +919607065656/ +919607195908

Website: http://www.maximizemarketresearch.com

Go here to read the rest:
Global Gene Therapy Market 2019 exhibiting a CAGR of 22.07% till 2025 - Fusion Science Academy

From our Obsession

New thinking is required to serve an aging population.

Scientists know a lot about the hallmarks of different types of dementia. Alzheimers disease is characterized by buildups of amyloid and tau proteins. Vascular dementia is the result of gnarled and broken blood vessels that normally supply oxygen to the brain. Parkinsons disease and other Lewy Body dementias are caused by misshapen alpha-synuclein proteins in the brain.

Conventional wisdom has it that each of these dementias needs its own treatment. An anti-amyloid drug probably wouldnt work for someone who doesnt have amyloid buildups in their brain.

But to this day, there are no definitive treatmentsor preventive measuresfor any of the dozens of dementias out there. Which has led some researchers to take a more systematic approach: What if there were a single mechanism in the brain that, when faulty, leads to all kinds of dementias? And what if this mechanism, like a switch, could be flipped off?

Thats the thinking of Michael Fossel, the founder of the Michigan-based biotech startup Telocyte, which is developing treatments for Alzheimers. Today (Jan. 14), Fossel published a review articlepostulating that Alzheimers and other dementias are caused by a failing of a workhorse class of brain cells called glia. He also proposes that he and his colleagues at Telocyte, founded in 2015, have a solution: a gene therapy that could target these cells to keep dementia at bay.

The paper is theoreticalits a review, so its not presenting any original data. Its a new way of thinking, and a bold proposition. Its encouraging to see individuals like Dr. Fossel pulling together research and trying to come up with new theories, says Rebecca Edelmayer, the director of scientific engagement at the Alzheimers Association. The Alzheimers Association is a nonprofit and publisher of Alzheimers and Dementia, the journal in which Fossels review article appeared.

But while theories are important, Edelmayer says, they also need to be tested. Gene therapies are still relatively new. And theres reason to wonder about the safety of the gene the therapy would introduce: one that codes for the enzyme telomerase. Before scientists can even begin to test Fossels systematic theory of dementia, theyll need a lot of data demonstrating its safety.

Telomerase has been a focus of longevity research for years. Its an enzyme that lengthens telomeres, which are the genetic caps on the end of our chromosomes. Every time cells divide, telomeres shortenand when telomeres have been sufficiently shaved away, cells enter a state called senescence and stop dividing. Then, they self-destruct.

Shorter telomeres have been correlated with a whole host of age-related health issues: cancer, diabetes, and even forms of dementia. But its not the telomeres themselves that cause these issues, Fossel suggests. As my telomeres shorten, there are a lot of other things going on, too, he says.

The relative length of telomeres, we know, sends a signal to the rest of the cells DNA. As telomeres shorten with cell replication, cells change the way they carry out other genetic instructions, which can result in shoddy protein production. Its a process called the telomere positron effect (paywall), and scientists still dont fully understand it.

Fossel posits that when telomeres shrink in microglial cells, part of the brains immune system, other critical parts of their DNA degrade, tooand that genetic damage can result in many different dementias.

Telocytes gene therapy would aim to rebuild those glial telomeres. That would involve sending an active copy of the telomerase gene, TERT, into the cerebrospinal fluid, carried by a virus. The virus, which should be otherwise benign, isnt great at getting genetic material into specific cells: in mouse models, about 5% of the total therapy winds up in neurons, to no lasting effect, and about 1% winds up in microglial cells, Fossel says. But even with the TERT gene just floating around in the glial cell for a few weeks or months, it might be enough for telomerase to lengthen those end caps and trick the cell into expressing genes like it did in its younger days.

Usually, gene therapies work by introducing new genetic material that replaces a persons faulty or missing genetic code. Telocytes gene therapy, however, wouldnt be replacing a gene: Itd just be giving glial cells another copy of one they already have. All of our cells have the TERT gene embedded in their chromosomes. But the vast majority of cells (save for red blood cells, sperm or egg cells, and cells along parts of the digestive tract) have the gene switched permanently off.

Thats for good reason: Telomerase is active in most forms of cancer. Which is why many scientists fear that inserting a gene that codes for telomeraselike Telocytes gene therapyrisks causing cancer

My main concern is its safety, says Jue Lin, molecular biologist at the University of California San Francisco whose work focuses on studying telomere length and stress levels over time. We dont know whether the over-expression of telomerase will increase the risk of cancer. In the brain, particularly in the glial cells that Fossels proposed gene therapy would target, the cancer in question would likely be glioblastoma, a ravenously growing brain tumor.

Mouse models using telomerase gene therapy in the brain have been promising, with no notable incidence of cancerbut those experiments are imperfect. Mice express telomerase differently than humans do, Lin explains: They have a lot more telomerase, in more tissues than humans. Mice also dont live as long as we do, and cancer takes a long time to develop, Lin says.

And gene therapies carry the risk of a dangerous immune reaction to the virus carrying the therapeutic gene. The viruses used in gene therapy todayand the one Fossel proposes usingshould be safer than the ones used in the early days of gene therapy. Adeno-associated virus, or AAV for short, should elicit only the tiniest of immune responses. But scientists have recently voiced concerns about the long-term safety of gene therapies using AAV.

Given the risks, theres disagreement over whether the telomerase approach is worth pursuing. Its important and interesting to have an additional hypothesis, says Diego Forero, a researcher at the School of Health Sciences at the Fundacin Universitaria del rea Andina in Colombia. His work, which is independent of Fossels, focuses on exposing astrocytes, a type of glial cell in the brain, to telomerase, to see how theyll react. Hes found that telomerase is involved in other cellular functions, like a cells metabolism. In his opinion, its too early to say that Fossels theory should be tested.

Rather than focusing on the potential therapeutic application of telomerase in brain cells, Forero is interested in more basic, exploratory research. He thinks that applying it to a specific targetlike a cure for dementiawouldnt tell scientists enough about all the ways telomerase could affect brain cells.

Those calls for prudence can be frustrating for dementia patients facing a dearth of options. Even with no immediate plans to conduct clinical trials, Fossel says he has already had some 200 people with mild to moderate dementia reach out to him as willing participants. Theyre ineligible for most other clinical trials for dementia therapies, which tend to seek out participants who have risk factors of the disease but minimal symptomsor none at all.

People have faced terrible disease and said Im going to take my chances, says Arthur Caplan, a bioethicist at New York Universitys Langone Medical Center. With vulnerable populations desperate for treatment, peer review from independent scientists becomes even more important. Its critical that the data and research are conducted by parties that dont have a vested financial interest in a certain outcome.

These studies also need to have strong institutional review boards, Caplan says. These boards are required any time researchers are conducting experiments with human subjectsespecially when the risks are so high.

Libella Gene Therapeutics, a Kansas-based biotech startup, is beginning a clinical trial for a telomerase gene therapy to treat broad aging this year. However, its taken its work to Colombia, where the standards for institutional review boards arent as high as they are in the US. Its a tactic informally known as IRB shopping, and it raises eyebrows in the research community.

Were always open to new ideas and novel ways [to treat dementia, Edelmayer says. We have to leave no stone unturned. But, she continued, one of the biggest things we want to see is not just theories. We want to see them tested.

Continued here:
Could a gene therapy cure dementia? - Quartz

After a manufacturing specification delay, Bluebird Bio has officially launched Zynteglo, its first gene therapy, andtapped a new leader to run the operation.

Nicola Heffron, a former exec with Celgene, Shire and GlaxoSmithKline, has joined Bluebird as its European chief, Bloomberg reported, just as the company gets its rollout underway there. She is replacing Andrew Obenshain, whos moving up to the global leadership team.

Heffron is tasked with charting the course for Bluebirds first commercial launch of its first product, Zynteglo, to treatbeta thalassemia, a rare inherited disease marked by reduced production of oxygen-carrying hemoglobin in red blood cells.

Artificial Intelligence in the Pharma Industry

How far has the adoption of AI technology come in Life Sciences? Share your thoughts in this brief, 8-minute survey. Let your voice be heard.

And Heffron knows a thing or two about blood disorders. Before Bluebird, she headed up global marketing for Celgenes myeloid portfolio, her LinkedIn profile shows.

Wednesday, Bluebird revealed that the $1.76 million-per-treatment gene therapy is now available in Germany. The University Hospitalof Heidelberg serves as the drugs first qualified treatment center inthe country, and Bluebird said its working with institutions that have expertise in stem cell transplant as well as in treating patients with [beta thalassemia] to establish more centers.

RELATED:Bluebird Bio readies Zynteglo launch as EU approves 'refined' manufacturing

During a presentation at the annual J.P. Morgan Healthcare Conference on Tuesday, Bluebird CEO Nick Leschly confirmed the companysvalue-based payment model for the costly onetime therapy. Under that arrangement,payments of 315,000 ($351,000) each are made in five installments over five years. Except for the first round of expenses, payers only pay the rest if Zynteglo delivers on its therapeutic promise.

So far, the reimbursement agreements Bluebird has penned using that model can coverabout half of Germanys patients, according to Leschly.

Novartis has also rolled out a similar program for its $2.1 million spinal muscular atrophy gene therapy Zolgensma.

Bluebird won its European nod for Zynteglo last year but delayed the launch after a manufacturing specification hiccup. The Cambridge, Massachusetts-based biotech has established a manufacturing network that includes both internal facilitiesand contract partners for itslentiviral vector and drug product, Leschly said Tuesday.

RELATED:ASH: Bluebird's multiple myeloma CAR-T follow-up shows promise in phase 1

In the U.S., Bluebird has started itsrolling submission for approval and is in talks with the FDA regarding the requirements and timing of the various components of the application. Its expecting to finish the process in the first half of 2020.

Outside of beta thalassemia, Bluebird is testing the same drug, also known as LentiGlobin, in sickle cell disease, which is also marked by an abnormality in hemoglobin.

According to phase 1/2 data presented at last years American Society of Hematology annual meeting in December, none of the 17 patients enrolled in group Cwhich used an improved stem cell harvest technique and a new manufacturing processrequired regular blood transfusions post-treatment. Nine patients had beenfollowed for at least six months at that data cutoff.

More:
Bluebird Bio taps ex-Celgene exec Heffron to lead its first gene therapy launch - FiercePharma

SAN RAFAEL, Calif., Jan. 13, 2020 /PRNewswire/ --BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, announced today that both the U.S. Food and Drug Administration (FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. have granted the Company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

The Company expects to start dosing patients in PHEARLESS, a Phase 1/2 study, in the first quarter of 2020 with product made at commercial scale from its award-winning gene therapy manufacturing facility. The Company is actively preparing regulatory submissions to open additional clinical sites in other countries.BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Status.

BMN 307 follows BioMarin's first investigational gene therapy clinical program: valoctocogene roxaparvovec to treat severe hemophilia A, currently in Phase 3. Based on data from an interim analysis of the ongoing Phase 3 study, the European Medicines Agency validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec, and the review process has begun. The Company also has submitted a Biologics License Application to the FDA and anticipates the review to begin in February 2020.

"With BMN 307, we are joining together our expertise in PKU biology and the knowledge we have gained from developing the only two approved therapies for PKU with our understanding of gene therapy clinical development and manufacturing from our valoctocogene roxaparvovec experience," said Hank Fuchs, President, Worldwide Research and Development at BioMarin."BioMarin has stood with the PKU community for over 15 years and remains dedicated to continuing to increase the body of medical knowledge in this devastating disease."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

BioMarin's clinical program is composed of two key studies. PHEARLESS, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, PHENOM, which has already started enrolling patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has treated approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 40 clinical studies in PKU and has sponsored 38 external clinical studies. BioMarin researchers have authored 54 publications in medical and scientific journals on PKU and supported another 52 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307. Manufacturing capabilitiesare an essential driver for our gene therapy programs and allows us to control quality, capacity, costs and scheduling enabling rapid development. Manufacture of BMN 307 was performed with a commercial ready process at scale in this facility. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and will speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet both commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well in to the future.

About Phenylketonuria

PKU, or PAH deficiency, is a genetic disorder affecting approximately 50,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's BMN 307 program generally, BioMarin's planned submissions to regulatory authorities for BMN 307, BioMarin's gene therapy manufacturing capabilities, the impact of using material manufactured at commercial scale in a clinical trial, the timing and results of BioMarin's planned Phase 1/2 trial of BMN 307, and the review of marketing applications for valoctocogene roxaparvovec. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:results and timing of current and planned preclinical studies and clinical trials of BMN 307; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

Contact:

Investors:

Media:

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

home

Go here to read the rest:
BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy - PRNewswire

Precision BioSciences, a Durham-based genome editing company, has reached two regulatory milestones for its potential therapy against multiple myeloma, a chronic cancer of white blood cells.

The U.S Food and Drug Administration (FDA) has accepted the companys Investigational New Drug (IND) application and also granted the therapy Orphan Drug Designation, a status that gives companies tax reductions and other incentives to develop treatments for rare diseases.

The therapy, designated as PBCAR269A, is Precisions third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate.

FDA acceptance of the IND for PBCAR269A further underscores the ongoing progress in our allogeneic CAR T pipeline, said Matt Kane, co-founder and chief executive officer of Precision BioSciences. We have now moved three CAR T programs from preclinical to clinical stage development since April 2019, and we look forward to continuing to advance our allogeneic CAR T portfolio to bring these novel therapeutic candidates to patients.

Matthew Kane

The company plans to begin a Phase 1 clinical trial of the therapy this spring at multiple sites using material produced at its own manufacturing facility in Durham. About 48 patients are expected to be enrolled.

For more information about the trial, visitwww.clinicaltrials.gov, and enter study identifier number NCT04171843.

In preclinical disease models, PBCAR269A has demonstrated no evidence of graft-versus-host disease at doses that resulted in potent anti-tumor activity, said Chris Heery, M.D., chief medical officer of Precision BioSciences. There remains significant unmet need in the treatment of relapsed/refractory multiple myeloma, and we are excited to begin clinical trials with an off-the-shelf CAR T therapy candidate in this setting.

The IND for PBCAR269A builds on the initial clinical data Precision presented in late 2019 for its lead program, a CAR T therapy for treating non Hodgkins lymphoma and B-cellacute lymphoblastic leukemia, and the FDAs acceptance of an IND for another CAR T therapy for treating non-Hodgkins lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma.

Precision BioSciences harnesses T cells, a type of white blood cell that is vital to the adaptive immune systems ability to identify specific antigens and destroy pathogens. Through the companys CAR T technology, the T cells can be directed to kill cancer cells.

Precision produces the CAR T therapies by selecting T cells derived from healthy donors as starting material. Then, using its proprietary ARCUS genome-editing technology, the company modifies the donor T cells.

Scientists insert the CAR gene at the T cell receptor locus, enabling the T cell to target a specific marker on a cancer cell, while knocking out the T cell receptor to prevent the patients immune system from recognizing and attacking the T cells.

The company optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of certain types of CAR T cells throughout the manufacturing process and in the final product.

The process creates a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease, normally a major challenge when inserting foreign or altered cells or tissues into the body.

The company has posted a four-minutevideoon its website to explain CAR T therapy, using Samurai warriors as an analogy.

Last July Precision opened its Manufacturing Center for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor CAR T cell therapy products.

Precision Bio facility

Given the potential output of our platform, weve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients, Kane said when the facility opened. In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.

The facility can produce three different drug substances: allogeneic CAR T cells, messenger RNA and adeno-associated viral vectors. It was designed to meet regulatory requirements in the United States, Europe and Japan.

In addition to health care, Precisions ARCUS genome-editing platform has applications in food and agriculture.

In 2018 the company created a new name and brand identity, Elo Life Systems, for its food and agriculture business, previously known as Precision PlantSciences, based in Research Triangle Park.

Elo is using the ARCUS platform and other new technologies for applications in crop improvement, animal genetics, industrial biotechnology and sustainable agriculture.

Since it was spun out of Duke University in 2006, Precision raised about $300 million in venture capital, government grants and collaboration agreements. The company went public in March 2019, grossing $145.4 million in an initial public offering of stock.

The companys shares are listed on the Nasdaq Global Select Market under the ticker symbol DTIL, shorthand for the companys marketing tagline, Dedicated to improving life.

(C) N.C. Biotech Center

View original post here:
Precision BioSciences hits two key FDA milestones in advancing gene therapy for cancer - WRAL Tech Wire

SAN FRANCISCO Pharmaceutical companies and their investors have grown accustomed to big news kicking off the year, specifically multibillion-dollar deals.

The last three J.P. Morgan Healthcare Conferences, considered a sort of Opening Day for the industry, were hallmarked by the acquisitions of Ariad Pharmaceuticals, Impact Biomedicines and Loxo Oncology. (And that's not including Bristol-Myers Squibb's $74 billion deal for Celgene the week before the meeting last year.)

In fact, Eli Lilly pressed Loxo for a quick buyout to have something flashy to announce at last year's conference. Such enthusiasm was noticeably absent this time around, though, resulting in a quieter first day than biotech shareholders had hoped for.

The Nasdaq Biotechnology Index fell almost 2%, with Sage Therapeutics, Clovis Oncology and other potential takeover targets trading down by market's close. Brad Loncar, a founder of biotech exchange-traded funds, noted on Twitter how even shares of MorphoSys fell despite the German drugmaker having the most positive news of the day.

While major M&A announcements seem unlikely for the rest of the week, industry experts still expect the challenges facing bigger companies will result in a healthy number of deals in 2020. In the meantime, biotechs will be busy trying to deliver on their development plans some of which were provided in more detail during Monday's presentations.

Connecticut-based Alexion Pharmaceuticals is best known for its high-priced rare disease drugs Soliris and Ultomiris. On Monday, the company gave an early look at full-year financials, reporting a top line revenue increase of more than 20% between 2018 and 2019. That growth correlates to, at the very least, roughly $4.96 billion in annual revenue, which would be slightly higher than the average analyst estimate.

For Stifel's Paul Matteis, more surprising than the revenue beat was Alexion's plan to treat four times as many U.S. neurology patients with Soliris and Ultomiris by 2025. If successful, the plan would create a "substantial upside" to revenue estimates, according to the analyst.

"This of course raises a number of natural questions," Matteis wrote in a note to investors, "such as where will this growth come from, and what does it assume (if anything) for additional neuro indications where Soliris/Ultomiris isn't derisked."

Shareholders, however, responded positively to the updates, sending Alexion shares up 4%.

Soliris is approved to treat several diseases, including a chronic neuromuscular illness known as gMG and a type of central nervous system inflammation abbreviated as NMOSD. Alexion says that, in less than two years time, these neurology indications have become its largest franchise by patient volume. By the end of 2019, almost 1,900 U.S. neurology patients were taking Soliris.

Ultomiris, a follow-on to Soliris, is under investigation as a treatment for gMG and NMOSD across a couple of late-stage studies. And on Tuesday, Alexion announced it will soon begin a Phase 3 study of the drug in ALS, with plans to enroll 350 adults in a 50-week trial.

Selling new paths to growth is particularly important for Alexion now, as the company has come under pressure from activist investor Elliott Advisors to seek a sale.

BioMarin could bring the first hemophilia gene therapy to market later this year. While waiting for regulators to confirm its approval application is under review, the California biotech announced Monday it has more than doubled capacity at a gene therapy plant. Altogether, the facility can make up to 10,000 doses each year of either the hemophilia treatment or a separate BioMarin gene therapy that's about to begin human testing.

That capacity level, according to executives, would allow the company to treat all U.S. hemophilia A patients in roughly two year's time. The update increases the competitive pressure on drugmakers with marketed products, such as Takeda and Novo Nordisk, as well as those working on rival hemophilia gene therapies. Swiss pharma giant Roche falls into both buckets, and could lose out on many patients because of BioMarin, according to a recent doctor survey from Citi Research.

Sarepta Therapeutics also had a manufacturing update, announcing that production for its experimental micro-dystrophin gene therapy is now large enough to be considered commercially viable.

RBC Capital Markets analyst Brian Abrahams called this a "critical manufacturing milestone" for Sarepta, one that shores up the timeline for a pivotal study scheduled to start sometime in the middle of the year.

Despite launching one of the industry's largest initial public offerings in 2018, Rubius Therapeutics has struggled out of the gate in getting its first clinical data. While its ambitions are large, the Flagship Pioneering-backed biotech failed to meet expectations it set for delivering early Phase 1 data from its lead asset, a PKU drug, by the end of 2019.

"We understand what we didn't do right in 2019, and we are doing it differently in 2020," CEO Pablo Cagnoni told a half-filled breakout room at the JPM conference. "We will deliver in 2020."

While Cagnoni and other executives emphasized cancer therapies set to enter clinical testing in 2020 and speedy progress on its own manufacturing plant, the biotech also admitted it still has not dosed a single PKU patient to date, calling into question the company's ability to execute.

Pressuring Rubius further is BioMarin's announcement that it will develop a PKU gene therapy, with plans to dose its first patient this quarter. BioMarin already sells two PKU drugs and could be a formidable competitor.

Shares in Rubius ticked down by about 5% Monday and have shed two-thirds of their value since the company went public.

See the original post here:
Alexion's neuro bet, Rubius' trial troubles and biotech's gene therapy milestones - BioPharma Dive