Archive for the ‘Gene Therapy Research’ Category

New York, Oct. 10, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Ewings Sarcoma Treatment Market by Type and Geography - Global Forecast and Analysis 2019-2023" - https://www.reportlinker.com/p05821758/?utm_source=GNW In 2018, the combination therapy segment had a significant market share, and this trend is expected to continue over the forecast period. Factors such as the high efficacy of combination therapy in treating Ewings sarcoma will play a significant role in the combination therapy segment to maintain its market position. Also, our global Ewings sarcoma treatment market report looks at factors such as the growing awareness about and funding for Ewings sarcoma, regulatory incentives, and financial assistance programs and reimbursement schemes. However, lack of approved therapies for Ewings sarcoma, absence of predictive biomarkers in Ewings sarcoma, and side-effects of chemotherapy may hamper the growth of the Ewings sarcoma treatment industry over the forecast period.

Global Ewings Sarcoma Treatment Market: Overview

Financial assistance programs and reimbursement schemes

The increasing cost of therapeutics for the treatment of various indications is encouraging governments of various countries and pharmaceutical vendors to introduce several financial assistance programs and reimbursement schemes. There are several drug assistance programs that offer payment limitation programs for costly medications such as chemotherapeutics. GlaxoSmithKline, Merck, and Pfizer are some of the prominent vendors providing prescription drug assistance programs to patients. Such assistance programs increase the patient adherence to drug treatment which will eventually lead to the expansion of the global Ewings sarcoma treatment market at a CAGR of almost 6% during the forecast period.

Emergence of regenerative therapies

Regenerative medicines, including gene therapy, are gaining traction in the market. This is encouraging several pharmaceutical companies to study and develop a gene therapy to treat metastatic Ewings sarcoma and recurrent Ewings tumors. The new therapeutic methods help in minimizing tumor cell proliferation in advance Ewings sarcoma. For instance, Gradalis is conducting a Phase I clinical trial to evaluate pbi-shRNA EWS/FLI1 Type 1 LPX, an anti-stathmin gene therapy.The growing focus on advancing regenerative medicines in clinical phases is expected to have a positive impact on the overall market growth.

Competitive Landscape

With the presence of several major players, the global Ewings sarcoma treatment market is fragmented. This robust vendor analysis is designed to help clients improve their market position, and in line with this, this report provides a detailed analysis of several leading Ewings sarcoma treatment manufacturers, that include Amneal Pharmaceuticals Inc., Baxter International Inc., Bristol-Myers Squibb Co., Eli Lilly and Co., Merck & Co. Inc., Mylan NV, Novartis AG, Pfizer Inc., Sun Pharmaceutical Industries Ltd., and Teva Pharmaceutical Industries Ltd.

Also, Ewings sarcoma treatment market analysis report includes information on upcoming trends and challenges that will influence market growth. This is to help companies strategize and leverage on all future growth opportunities.Read the full report: https://www.reportlinker.com/p05821758/?utm_source=GNW

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The global Ewing's sarcoma treatment market at a CAGR of almost 6% during the forecast period - GlobeNewswire

The Progress 1000 is compiled every year. The theme of this years list is the future and technology, and embraces whole new sectors including augmented and virtual reality and cyber security, as well as a wide range of activists challenging inequality and helping the environment.

Dr Prasanna Sooriakumaran, a consultant prostate cancer surgeon at UCLH, was included for his pioneering robotic surgery. He is investigating new techniques to spare men with prostate cancer the potentially debilitating effects of surgery. Early trials show that his new technique has drastically reduced recovery time for up to 94 per cent of patients.

Prasanna said: It is a great honour to top the list of Londons most influential doctors in the Progress 1000 after having also made the list last year. This is a testament to the wonderful staff at UCLH, who provide world class care to men with prostate cancer.

Professor Bryan Williams is director of one of the UKs leading NIHR Biomedical Research Centres at UCLH, director of research at UCLH and Chair of Medicine at UCL. He is a clinician at UCLH and is recognised as one of the worlds leading authorities on high blood pressure.

He said: It is good to see recognition of the influence that staff at UCLH have in driving forward medical research and innovation in London and beyond.

Professor Charles Swanton, UCLs professor of personalised cancer medicine with a lab at UCL Cancer Institute and the Francis Crick Institute in Kings Cross, a consultant at UCLH and chief clinician at Cancer Research UK, is leading pioneering research on lung cancer. Professor Swanton studies how cancers evolve in the body to spread and become resistant to therapy. He is also researching ways to treat tumours more effectively.

Charlie said: This is a great testament to the hospital, university, Crick and CRUK and the team for making TRACERx possible.

Professor Tariq Enver, director at UCL Cancer Institute and professor of stem cell biology at UCL, leads a grand coalition in the war on cancer by encouraging closer working relations between UCL, Kings College London, Queen Mary University of London and the Francis Crick Institute, creating a centre of excellence for biotherapeutics.

Tariq said: It is fantastic that the vital work being done by the team in London - which has the potential to transform cancer treatment in the long run is being recognised and encouraged. Being nominated is a massive boost for all of us who have worked so hard to reach this point.

Professor Ravi Gupta, until recently an infectious diseases clinician at UCLHs Hospital for Tropical Diseases, studies the evolution and spread of HIV drug resistance globally. He, along with colleagues at Imperial College London, recently published a report on how a patient with HIV and lymphoma is now free from both conditions after an allogeneic stem cell transplant using cells from a donor lacking a critical receptor protein for HIV infection, CCR5. This work has rejuvenated the field of HIV gene therapy.

He said: It has been humbling to work at UCLH alongside such dedicated staff, both clinical and academic. I hope that recognition of the London Patient HIV cure in the Progress 1000 list will serve as an inspiration in London and beyond in the fight against HIV/AIDS.

Professor Chris Whitty, the chief medical officer for England and a UCLH physician, was included on the list for his work which focuses on the diagnosis and management of infectious diseases in children and adults.

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Six people with links to UCLH listed among most influential people in London - University College London Hospitals

Oct. 10, 2019 09:16 UTC

AJDOVINA, Slovenia--(BUSINESS WIRE)-- BIA Separations, a leading bio-chromatography development and manufacturing company, today announced the introduction of its CORNERSTONE Exosome Process Development Solution, designed to help drug-developers overcome critical development bottlenecks in the preparation of exosomes for clinical use.

Introducing two proprietary new technologies central to highly pure and scalable purification, BIA Separations Exosome Process Development Solution represents the latest extension of its CORNERSTONE programs for the manufacture of gene therapy vectors, following the launch of its AAV program earlier this month.

Exosomes fulfil a critical role as communicators among cells, with targeting and message content depending on their surface receptors and payload. Their unique ability to deliver that payload to the right target makes them obvious candidates for an extensive range of therapeutic applications in the fields of cancer and regenerative medicine, including for neurodegenerative and cardio-pulmonary disorders.

For such clinical applications to be realised, thoroughly purified exosomes are required, with key contaminant classes such as host-cell proteins, host-cell DNA, and non-exosomal vesicles reduced to trace levels. BIA Separations new technology brings important new tools for this purification, whilst ensuring fast and efficient process development. The technology is easily scalable from lab to manufacturing, and is able to provide better product quality than traditional purification systems.

CORNERSTONE Exosome Development Solution is built on the capabilities of two novel technologies. Kryptonase is an integrated enzymatic treatment that reduces host-cell DNA and facilitates the removal of host-cell protein contaminants. The CIMmultus EV monolithic column is a chromatography device that eliminates non-exosomal vesicles, and concentrates exosomes in a low shear environment. The technologies are available as part of a comprehensive Process Development Service, in which BIA Separations experts design and optimize processes to support clinical trials and scale-up to industrial manufacturing. Translational scientists and process developers can also access both new products as a Process Development Pack for exosome research purposes.

Ale trancar, Chief Executive Officer at BIA Separations, said: Exosomes represent the next generation delivery strategy for nucleic acids and other therapeutics, as well as being evaluated as a platform for regenerative medicine with high hopes of bringing more therapeutic options to chronically ill patients. We are all looking forward to seeing the impact of our tools on this novel therapy space.

Pete Gagnon, Chief Scientific Officer at BIA Separations, added: Every new product class presents a unique new set of challenges and exosomes are no exception. Our new CORNERSTONE program embodies the decades of experience weve developed with the purification of viruses, DNA plasmids, mRNA, and other gene therapy products, and extends it to include exosomes. This gives our customers a head-start that will help them be first to the clinic and first to market.

To learn more about BIA Separations services and technology for therapeutic vectors, visit http://www.biaseparations.com/en/featured/cornerstone-process-solutions and http://www.biaseparations.com/en/featured/evs

Notes to Editors

For high resolution images, please email sarah.jeffery@zymecommunications.com

About BIA Separations: http://www.biaseparations.com

BIA Separations is the leading developer and manufacturer of CIM (Convective Interaction Media) monolithic chromatographic columns, and development of integrated processes for the production, purification, and analysis of large biomolecules such as gene therapy vectors.

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BIA Separations introduces CORNERSTONE Exosome Process Development Solution to Enable Industrial Scale Manufacture of Therapeutic Exosomes - BioSpace

Scientists have discovered a new way to treat cystic fibrosis (CF) that involves delivering artificial proteins to patients lung cells to replace the faulty cystic fibrosis transmembrane conductance regulator (CFTR) protein.

The finding was reported in the study, Anion carriers as potential treatments for cystic fibrosis: transport in cystic fibrosis cells, and additivity to channel-targeting drugs, published recently in the journal Chemical Science.

CF is a genetic disorder caused by mutations in the CFTR gene, which provides instructions to make the CFTR protein.This protein works as a channel that transports in and out of cells molecules with a negative electrical charge (called anions), such as chloride, which have a direct impact on the regulation of water transport and the production of mucus.

In patients with CF, the transport of anions is impaired, leading to a buildup of fluid in the lungs and other organs.

One approach to restore anion transport to CF cells [utilizes] alternative pathways for transmembrane anion transport, including artificial anion carriers (anionophores), the researchers wrote.

The basic idea is to deliver these anionophores to patients lung cells, so that these artificial molecule transporters may replace the faulty CFTR protein, and restore the transport of water and salt in and out of cells, alleviating the symptoms of CF.

According to the researchers, these anionophores, unlike other types of targeted therapies that have been developed to correct specific defects in CFTR, may be used as a form of treatment for all CF patients, either alone or in combination with other CF therapies.

In their study, researchers from the University of Bristol in collaboration with investigators from the University of Southampton, both in the U.K., designed and tested 22 different anionophores.

Researchers discovered that four of these compounds (compound 11, 12, 15, and 19) were able to effectively transport anions in and out of lung cells cultured in a lab dish in a dose-dependent manner.

Moreover, they showed that all four compounds improved the transport of anions in cells that contained the F508 deletion, the most common CFTR mutation associated with CF, when used together with lumacaftor and ivacaftor, the active ingredients of two ofVertex Pharmaceuticals approved CF therapies, Orkambi and Kalydeco.

We also show that the compounds need not be toxic, which is an important issue, and that their activity can supplement the effects of recently introduced drugs, which help some CF patients through a different mechanism, Anthony Davis, from the University of Bristol and corresponding author of the study, said in a press release.

Overall, the results provide further evidence that anionophores, by themselves or together with other treatments that restore anion transport, offer a potential therapeutic strategy for CF, the researchers wrote.

Theres still a long way to go, but if the research proceeds as hoped it might lead to a genuinely effective and general treatment for CF patients over this timescale, Davis said.

Joana holds a MSc in Biology and a MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. She is currently finishing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that made up the lining of blood vessels found in the umbilical cord of newborns.

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Scientists Hope They Have Found New Form of Cystic Fibrosis Therapy - Cystic Fibrosis News Today

LONDON Chances are, youve heard little or nothing about Rick Klausners startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today hes officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million hes raised in the past year to make that vision a reality.

Weve being staying stealth, Klausner tells me, then adding with a chuckle: and going back to stealth after this.

Cell therapy has a lot of challenges, notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. Over the years Rick and I talked about how it would be wonderful to take that on as a mission.

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A new player is taking the field in a push for a hemophilia A gene therapy, and it's a big one - Endpoints News

Hemophilia Gene therapy is introduction of healthier gene into the body of the patient which replaces the damaged gene. Due to the progress and technology advancement Hemophilia gene therapy is soon expected to be a possibility. Hemophilia is one disease which is expected to be a target for treatment with hemophilia gene therapy. People suffering from hemophilia have difficulty and lack in forming blood clots which brings them at hug risk of uncontrolled bleeding during minor injuries and internal bleeding into muscles and joints. There are no approved Hemophilia Gene therapy presently however the treatment involve injections which clot the proteins, these proteins are expensive and cost about 1 million per year. Manufacturers and scientists with continuous Research and development are making efforts for successful treatment of Hemophilia by Gene therapy. Patients suffering from hemophilia A has a mutation for factor VIII in the gene and patients suffering from hemophilia B have a mutation due to the absence of clotting factor IX. The final goal of hemophilia gene therapy is the restoration of a corrected gene for the rest of the life of the patient which is a challenging and yet not accomplished. Hemophilia gene therapy treatment has a low brink for success. The hemophilia gene therapy uses viruses as a vector however the problem is the vector is not large enough which can carry all the genes and the other problem occurs during the insertion of the vector as the vector is a virus there is a possibility that it can interfere with immune system and react to it. Moreover hemophilia gene therapy is more likely to interact with the immune system if the doses are increased. The scientists and the biopharmaceutical industries are trying their best in order to cure Hemophilia A and B by gene therapy.

Tentatively,Hemophilia Gene therapyMarkethas been segmented on the basis of product type.

On the basis of Indication, Hemophilia Gene therapy Market can be segmented as:

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Global Hemophilia Gene therapy Market will witness advancement due to the rising population suffering from hemophilia. According to national institute of hemophilia approximately 20,000 people in U.S. and 400,000worldwide are suffering from hemophilia. The National hemophilia foundation is awarding grants to further support the research for Hemophilia Gene therapy which will in turn help in the treatment of hemophilia. The biopharmaceutical manufacturers and research centers are working together to understand the genetics of hemophilia and improve Hemophilia Gene therapy which could help in treatment of hemophilia in the future. Manufacturers such as Roche has recently acquired Spark Therapeutics for its long term investment hemophilia A gene therapy market. Also many drugs for hemophilia gene therapy are in clinical trials. The continuous investment and research by the manufacturers is expected to improve the hemophilia gene therapy market in the coming future. Also Hemophilia Gene therapy assures to address the unmet needs by one time administration which will further improve its severity. However the arrival of Hemophilia Gene therapy is a concern over its affordability and accessibility.

Geographically, global Hemophilia Gene therapy Market is split into regions viz. North America, Latin America, Middle East & Africa, Asia Pacific, Western Europe and Eastern Europe. North Americas Hemophilia Gene therapy Market is expected to grow because of evolution and progression in the technology and advancements to improve the patients health. However the willingness of payers and government to arrange funding or insurance coverage for Hemophilia Gene therapy is not well established. If the manufacturers that bring Hemophilia Gene therapy to the market have conventional and older hemophilia therapies within their product portfolio their consideration to offer gene therapy for low price may lack as the new technology would disrupt their present market.

Some of the major market members in the Global Hemophilia Gene therapy Market identified across the value chain includes: F. Hoffmann-La Roche AG, Pfizer Inc., BioMarin Pharmaceuticals, uniQure, Shire PLC, Sangamo Therapeutics, among others.

The report provides in-depth analysis of parent market trends, governing factors and macro-economic indicator and along with market attractiveness as per segments. Also the report is a compilation of qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report also outlines the qualitative impact of different market factors on market segments and geographies.

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The report Haemophilia Gene therapy Market covers exhaustive analysis on:

Gene therapy Market Regional analysis includes:

Report Highlights:

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Hemophilia Gene Therapy Market Segments and Key Trends 2018 to 2028 - My People Times

New York City, NY: Oct 09, 2019 Published via (Wired Release) The global Gene Therapy market is comprehensively researched and analyzed in the report by MarketResearch.Biz to help out market players to improve their business plans and ensure long-term success.

The study includes of 100+ market data Graphs & Figures, Gene Therapy Pie Chart, and Tables. The report has an in-depth analysis and is easy to understand. Currently, the Gene Therapy market is increasing its presence. The Research report shows a complete evaluation of the market and has ongoing growth factors, upcoming trends, facts, attentive opinions, and market data verified by the Gene Therapy industry. The report offers a forecast for global Gene Therapy Market till 2028.

Gene Therapy market report offers a comprehensive outlook of current trends and new product launch in the global Gene Therapy market. Featuring worldwide and regional data and over leading key players profiles, this report serves the ultimate guide to exploring opportunities in the Gene Therapy industry globally. The author of the report has used simple language and uncomplicated statistical figures that comprised thorough information and complete information on the global Gene Therapy market. The report provides market players with useful information and suggests result-oriented tactics to gain a competitive edge in the Gene Therapy market. This shows how distinct players are competing in the worldwide market and discusses strategies to distinguish themselves from other competitors.

For Detailed Insights and Better Understanding of Gene Therapy Market, Request Free Sample PDF Here: https://marketresearch.biz/report/gene-therapy-market/request-sample

Key highlights and crucial features of the report:

1) Which leading players are presently included in the report?

Here are the companies that are presently included in the report: Amgen Inc, NewLink Genetics Corp., Bluebird bio Inc, Kite Pharma Inc, Novartis, Transgene SA, Genethon, Spark Therapeutics Inc, GlaxoSmithKline PLC, Applied Genetic Technologies Corporation and Oxford BioMedica PLC.

** List of the companies stated above might differ in the final report dependent on a merger, name change, and other factors.

2) Can you list or add the latest firms as per client requirement?

Yes, we can add a new firm as per your requirement. The final confirmation regarding the same must be provided by the research team subject to the difficulty of the survey.

** Availability of information will be confirmed after research in the case of a privately held firm.

3) Which regions are covered in the report? Is it possible to list any specific country?

Currently, our research report provides special focus and attention on the following areas:

Europe, China, United States, Japan, India, and Central & South America, Southeast Asia.

** Instead Of the above countries if the client required any regional customize report we do provide. The final confirmation regarding the same must be provided by our research team.

4) Is it possible to include extra Market breakdown or segmentation in the report?

Yes, it is possible depending on the difficulty of the survey and availability of market data. However, a detailed sharing of your requirements with our research team is a must before providing final confirmation to the client.

** Deliverable time and quote might vary depending upon the requirements.

Have Any Query Or Specific Requirement? Feel Free To Ask Our Industry Experts: https://marketresearch.biz/report/gene-therapy-market/#inquiry

Segmentation Analysis:

By Vector: Viral vector, Retroviruses, Lentiviruses, Adenoviruses, Adeno Associated Virus, Herpes Simplex Virus, Poxvirus, Vaccinia Virus, Non-viral vector, Naked/Plasmid Vectors, Gene Gun, Electroporation, Lipofection. By Gene Therapy: Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth factors, Receptors, Other. By Application: Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious disease, Other Diseases

So as to get a more intense view of the Gene Therapy market size, competitive scenario is offered. This includes revenue Gene Therapy market share (%) by major players and revenue (in Mn USD) by major companies. Moreover, a qualitative study is made towards Gene Therapy market product/service differences, concentration rate, development of future trends, and new entrants. The Gene Therapy industry overviews, SWOT analysis and business strategies of each vendor in the market give understanding about the Gene Therapy market forces and how those can be utilized to create future growth opportunities.

Production Analysis: SWOT analysis of Gene Therapy industry key players based on Strengths, Weaknesses, Challenges, Companys internal & external environments. , Market Opportunities and Risks. It also contains Production, Revenue, and product price and market shares of key players. That information are further subject to Manufacturing Base Distribution, Gene Therapy Production Area and Product Type. Major points like Competitive landscape and latest Trends, Industry Mergers & Acquisitions, Expansion which are crucial information to grow/establish a business is also comprised.

Browse More Insight Of Gene Therapy Market Research Report Enabled with Respective Tables and Figures at: https://marketresearch.biz/report/gene-therapy-market/

In this research, the years considered to predict the market size of Gene Therapy Market are as follows:-

History Year: 2013-2017

Base Year: 2018

Estimated Year: 2019

Forecast Year: 2019 to 2028

Detailed analysis, actual numbers, market size evaluation, and Gene Therapy business opportunities are available in the full report.

Thank you for reading this article, you can also get a separate customize chapter-wise section or region-wise report editions.

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Gene Therapy Market 2028 Forecasts and Analysis with Top Key Players like Novartis, Kite Pharma, GlaxoSmithKline PLC - TheLoop21

New York City, NY: October 9, 2019- WorldGene Therapy MarketResearch Report is classified by key Gene Therapy manufacturers, regions, and various segmentation to offers all important details to the readers. A thorough analysis of Gene Therapy market based on product portfolio, applications, price, production processes included in Gene Therapy are calculated deeply. The market is hoped to have additional upcoming players which may lean to gigantic worldwide market development. Gene Therapy industry growth,scope, with Gene Therapy revenue are specifiy in this report.

Detailed study of Gene Therapy market competition, advancement, Gene Therapy development opportunities and factors restraining the market growth are study in detail. All the elementary market information like Gene Therapy consumer volume, market size, demand/supply analysis, and Gene Therapy gross margin study are included in this report. The changing competitive environment will lead to accretion of revenue in Gene Therapy market.

Worldwide Gene Therapy study will offers as a worthwhile guide for studying business opportunities and building the strategic business judgment which will gives benefit in Gene Therapy. The latest Gene Therapy trend and updated marketing strategies will foreseen the Gene Therapy market presentation in upcoming years.

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This information relevant to innovations proceed,technical advancement, in Gene Therapy, press release, Gene Therapy marketing strategies are expanded in this report. The report also manage Gene Therapy market inspection on a global and regional basis to provide worldwide market projection and market share for the upcoming period 2019 to 2028.

Purview of the Gene Therapy Market Report:

The foreseen Gene Therapy market trends, industry development, sales margin estimated over the forecast period are included in this report. The process containing in Gene Therapy manufacturing, end users, sellers, buyers, manufacturers are analysed deeply in this report.

Key developments, supply chain static of Gene Therapy, innovations will guide the market players to build up the strategies for business. Obtaining crucial Gene Therapy information and structured it in a separate way will help enhnaced the decisions.

Global Gene Therapy Market Classification:

This report illustrate the competitive landscape sight of all the key players depend on their company profile, Gene Therapy sales profit, development stature, Gene Therapy import/export scheme and consumption rate. The Gene Therapy improvement and market revenue are provided for each and every region, manufacturer and Gene Therapy application of product. The leading manufacturers of global Gene Therapy market contains Amgen Inc, Oxford BioMedica PLC, Genethon, Applied Genetic Technologies Corporation, GlaxoSmithKline PLC, NewLink Genetics Corp., Spark Therapeutics Inc, Transgene SA, Kite Pharma Inc, Bluebird bio Inc and Novartis.

The Gene Therapy manufacturing regions like Asia-Pacific, Canada, United States, Italy, South America, Africa, Japan, Mexico, Brazil, Southeast Asia, UK, Australia, Germany, Russia, Europe, Middle East & Africa, France, India,North America, Korea, and China are included in this Gene Therapy report. Gene Therapy industry scope and statistical data related to past, current and future Gene Therapy market are analysed in this report study.

Eccentricity Of The Global Gene Therapy Market Report:

Study of latest and future Gene Therapy industry trends will give boost to growth opportunities. The upcoming information relevant to market share, revenue, and growth will beneficial while taking the business decisions. The separate study depend on regions, and various segments will provides all the minuscule details to the readers. Growth endorsement, study of company profiles of Gene Therapy major manufacturers and strategies endorse by major players will propel business vision. Usefulness of investment anaylsis, market existence, and upcoming Gene Therapy sections will determine the market scope in forecasting years.

Enquire about the report, talk with our specialist at:https://marketresearch.biz/report/gene-therapy-market/#inquiry

TOC of Gene Therapy Report:

Part 1 of the report offers data related to Gene Therapy product scope, industry outlook, growth opportunities, challenges to the Gene Therapy market growth and major propeling forces.

Part 2 provides overall detailing related to key Gene Therapy manufacturers, their sales revenue, and product cost structure forecast over 2019-2028.

Part 3 lists the competitive sight of the Gene Therapy market depend on the company profile, volume and market share forecast from 2019-2028.

Part 4 analysis the major regions giving contribution to the market growth, their sales margin, size and leading manufacturing countries includes with these regions.

Part 5,6 gives details related to Gene Therapy industry size and share of each manufacturers existing within the region, trends, Gene Therapy scope, and application, forecast from 2019-2028.

Part 7,8 serves global market study based on various segments, Gene Therapy sales volume, forecast from 2019-2028.

Part 9 provides the futuristic market data relevant to Gene Therapy like the projected development, revenue share, market scope, emerging regions and the growth prospects of the industry.

Part 10 covers the study of Gene Therapy marketing channels, vendors, traders and finally beneficial research conclusions are served.

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Gene Therapy Market Growth Prospects, Traders, Opportunities with Growth Factor By 2028 - Healthcare News

Zion Market Research published a new 110+ pages industry researchGene Therapy Market: by Type (Somatic Cell Gene Therapy and Germ Cell Gene Therapy) and by Application (Genetic Disorders, Cancer, Cardiovascular Disease, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 20172024.

TheGlobal Gene Therapy Market Is Expected To Reach Around USD 2,269 Million By 2024complete outline is crystal clear penned down in the GlobalGene Therapy Marketresearch report such that not only an unskilled individual but also a professional can easily extrapolate the entire Gene Therapy Market within a few seconds.The research study covers research data which makes the document a handy resource for managers, analysts, industry experts, and other key people get ready-to-access and self-analyzed study along with TOC, graphs and tables to help understand the market size, share, trends, growth drivers and market opportunities and challenges.

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The Gene Therapy Market research report covers major industry player profiles that include:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

This report employs the SWOT analysis technique for the assessment of the development of the most remarkable market players. It additionally considers the latest upgrades while assessing the development of leading market players. Moreover, in the global Gene Therapy Market report, the key product categories of the global Gene Therapy Market are included. The report similarly demonstrates supportive data related to the dominant players in the market, for instance, product offerings, revenue, segmentation, and business synopsis. The global Gene Therapy Market is as well analyzed on the basis of numerous regions.

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Global Gene Therapy Market: Regional Analysis

To understand the competitive landscape in the market, an analysis of Porters five forces model for the market has also been included. The study encompasses a market attractiveness analysis, wherein all segments are benchmarked based on their market size, growth rate, and general attractiveness. This report is prepared using data sourced from in-house databases, secondary and primary research team of industry experts.

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The report answers important questions that companies may have when operating in the Global Gene Therapy Market. Some of the questions are given below:

What is the current CAGR of the Global Gene Therapy Market?

Which product is expected to show the highest market growth?

Which application is projected to gain a lions share of the Global Gene Therapy Market?

Which region is foretold to create the most number of opportunities in the Global Gene Therapy Market?

Will there be any changes in market competition during the forecast period?

Which are the top players currently operating in the global market?

How will the market situation change in the coming years?

What are the common business tactics adopted by players?

What is the growth outlook of the Global Gene Therapy Market?

Also, Research Report Examines:

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Gene Therapy Market to exceed USD 2269 Million By 2024 - The Industry Today News

Cancer Gene Therapy Market SWOT Analysis And Forecast 2024

The Cancer Gene Therapy Market report has been added to Qurate Business Intelligence offering. The global Cancer Gene Therapy Market report offers the important data to help the firms cope up with the knowledge gap due to the advancements in the industry and effectively utilize the opportunities that present itself into the ever-changing market.

Cancer Gene Therapy Market highlights the information about the dominant players industries and market, technologies, and abilities over the trends and the developments of the industries. After deep research and analysis by the experts, they also disclosed the data about the strong contenders contributing in the market growth and expansion and challenging one another in terms of demand, supply, production, value estimation, revenue, and sales.

The latest report offers wide-ranging coverage of several important industry verticals along with key market players. It as well offers consistency on the part of analysis or estimation across a range of coverage areas and geographies.

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Major Key players of Cancer Gene Therapy Market:

Adaptimmune, GlaxoSmithKline, Bluebird bio, Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, OncoGenex Pharmaceuticals, Genelux Corporation, Cell Genesys, Advantagene, GenVec, BioCancell, Celgene, Epeius Biotechnologies, Introgen Therapeutics, Ziopharm Oncology, Shenzhen SiBiono GeneTech, Altor Bioscience,

Type

Gene Induced Immunotherapy, Oncolytic Virotherapy, Gene Transfer

Application

Hospitals, Diagnostics Centers, Research Institutes

Cancer Gene Therapy MarketPromising Regions & Countries Mentioned In The Cancer Gene Therapy Market Report: North America (U.S and Canada and the rest of North America)Europe (Germany, France, Italy and Rest of Europe)Asia-Pacific (China, Japan, India, South Korea and Rest of Asia-Pacific)LAMEA (Brazil, Turkey, Saudi Arabia, South Africa and Rest of LAMEA)

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AbstractThe report covers the forecast and analysis for the Cancer Gene Therapy Market on a global and regional level.The report includes the positive and the negative factors that are influencing the growth of the market.The revenue generated by the prominent industry players has been analyzed in the report.The market numbers have been calculated using top-down and the bottom-up approaches.The Cancer Gene Therapy Market has been analyzed using S.T.E.E.P.L.E. Analysis (Social, Technological, Environmental, Economical, Political, Legal, Ethical) .The market is segmented on the basis of Cancer Gene Therapy Market type, software type, service type, solution type, and application type, which in turn is bifurcated on a regional level as well.All the segments have been evaluated based on the present and the future trends.The report deals with in-depth quantitative and qualitative analyses of the Cancer Gene Therapy Market.The report includes the detailed company profiles of the prominent market players.

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The global Cancer Gene Therapy Market report conveys the information regarding the prcised escalation or decline in market growth due to several key factors. The analysts, using various analytical methodologies such as SWOT analysis, S.T.E.E.P.L.E. Analysis, etc. among others to generate the precise forecast belonging to the growth rate and upcoming opportunities in the market growth at the global level. The global Cancer Gene Therapy Market report represents the complete information of the market in an eye-catching and easily understandable way with examples, figures, graphs, and flowcharts.

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Cancer Gene Therapy Market 2019 In-Depth Analysis of Industry Share, Size, Growth Outlook up to 2024 - Space Market Research

DUBLIN, Oct. 10, 2019 /PRNewswire/ -- The "Global DNA Read, Write and Edit Market" report has been added to ResearchAndMarkets.com's offering.

The scope of the report includes DNA read, write and edit technologies, applications, industries, initiatives, patents, and companies. The markets for read, write and edit products and services are given for 2017, 2018, 2019 (estimated) and 2024 (forecast).

This report reviews the main read, write and edit technologies and explains why genetic variation is important in clinical testing and disease. It then discusses significant large-scale research initiatives that impact read, write and edit applications. Of particular interest is a discussion of population-scale sequencing projects throughout the world, and their likely impact. The main market driving forces for read, write and edit products and services are listed and discussed.

The report quantifies each of the main market segments. The read (sequencing) market is quantified by delivered format, including sequencing workflow products (sample preparation kits and reagents, sequencing instruments and consumables, and informatics) and sequencing services (clinical diagnostics and sequencing services to applied market customers).

The sequencing workflow products market is quantified by type, that is, DNA isolation and extraction; target enrichment; library preparation; and informatics/ecosystems. The sequencing instruments and consumables market is given by platform (Sanger, NGS, and 3GS).

The sequencing services market is analyzed by end-user application (applied, clinical, and R&D). Within sequencing services, the applied market is analyzed by end-user application (agriculture, biopharma, consumer, microbiology, population-scale genomics, synthetic biology and other).

Also within sequencing services, the clinical market is analyzed and quantified by disease category (cardiovascular, clinical microbiology and infectious diseases, Mendelian disorders, metabolic/immune disorders, neurology, oncology, reproductive health, and transplant medicine).

The DNA write (synthesis) market is quantified by product type (oligonucleotides, synthetic biology parts, genes, and RNA therapeutics). The oligonucleotide market is analyzed by application (gene editing, sequencing, PCR, FISH, microarray, gene synthesis and other). The gene market is quantified by gene type (standardized, value-added). Finally, the RNA therapeutics market is quantified by platform (RNA interference, antisense oligos, micro RNA modulation, and mRNA) and by disease category (cancer, hematology, musculoskeletal, neurology, and rare diseases).

The DNA edit (gene editing) market is quantified by application (agriculture, biopharma, diagnostics, and therapeutics); editing platform (CRISPR, meganuclease, TALEN, ZFN). The gene-editing agriculture market is analyzed by product type (crop/seeds, livestock). The gene-editing biotechnology market is analyzed by product type (kits and reagents, cell line engineering, animal models and services). The gene-editing therapeutics market is analyzed by disease category (eye and rare diseases).

Specific geographic markets discussed include North America, Europe, Asia-Pacific, and the rest of the world (ROW).

Industry sectors analyzed include next-generation sequencing; long-read sequencing; DNA synthesis; RNA therapies; and gene editing.

More than 320 companies in the read, write and edit industry are profiled in this report.

The author also provides a summary of more than 180 of the main industry acquisitions and strategic alliances that took place from January 2018 through June 2019, including key alliance trends.

Market Summary

The DNA read, write and edit industry is at the beginning stages of its growth story; penetration of the key markets is still at an early stage. The data indicates that there is a significant future upside for sequencing across research, metagenomics, agriculture, synthetic biology, and clinical applications, among others.

The situation is similar for DNA writing and editing technologies, with clinical therapeutic applications, in particular, providing an enormous total available future market that is yet to be significantly penetrated. Major successes in this industry include the adoption of next-generation sequencing (NGS) for noninvasive prenatal testing; enabling the roles of synthetic DNA oligonucleotides and genes in the rise of the synthetic biology industry; and rapid adoption of CRISPR gene editing by research institutions and biopharma industries.

There is increasing interplay among the three DNA technology platforms, giving rise to innovative corporate strategies. For example, Arbor Biotechnologies employs sequencing, gene synthesis, and artificial intelligence to perform high-throughput discovery of biomolecules, including new CRISPR proteins.

Report Scope

Key Topics Covered

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Overview

Chapter 4 Technology Background

Chapter 5 DNA Read, Write and Edit Initiatives

Chapter 6 DNA Read, Write and Edit Applications

Chapter 7 DNA Read, Write and Edit Industries

Chapter 8 Acquisitions and Strategic Alliances

Chapter 9 DNA Read, Write and Edit Markets

Chapter 10 Patents

Chapter 11 Nucleic Acid Read, Write and Edit Company Profiles

Companies Mentioned

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Analysis on the Global DNA Read, Write & Edit Market, 2017-2019 and Forecast to 2024 - PRNewswire

Zion Market Research published a research report containing 110+ pages titled Gene Therapy Market: by Type (Somatic Cell Gene Therapy and Germ Cell Gene Therapy) and by Application (Genetic Disorders, Cancer, Cardiovascular Disease, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 20172024 in its research offering. This report is well crafted with a combination of the crucial data associated with the worldwide market, along with key factors liable for the demand for its services and merchandise. The report highlights the newest technological developments and new launches that assist our customers to set up their future-based potential products, make wise business selections to meet the projected demand ratio.

This report is an excellent presentation of critical dynamics, regional growth, competition, and other important aspects of the global Gene Therapy Market. This report will provide accurate market figures and statistics that include market CAGR, revenue, volume, consumption, production, market shares, price, and gross margin. Each regional market analysis in the report is carefully analyzed to explore key opportunities and business prospects they are expected to offer in the near future. This report also includes major market players in the global Gene Therapy market on the basis of various factors including their past market performance, market size & share, by product analysis, distribution channels, marketing strategies, etc. This equips players with crucial information and data to improve their business tactics and ensure a strong foothold in the global Gene Therapy market.

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How this report helps you?

Major Company Profiles Included in This Report:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

Key Highlights of TOC

Industry Overview: This starts with a product overview and scope of the global Gene Therapy market. It also gives consumption and production growth rate comparisons by application and product respectively. It also includes a glimpse of the regional study and market size analysis for the review for the forecasted period.

Major Company Profiles: Each company profiled in the report is assessed for its market growth keeping in view vital factors such as price, gross margin, revenue, production, markets served, main business, product specifications, applications, and introduction, areas served, and production sites.

Market Dynamics: The readers are provided with a comprehensive analysis of market challenges, demand, market influence factors, growth drivers & restraints, global opportunities, and trends.

Market Forecast: The research report will provide forecast by application, price, revenue, and production forecast by product, consumption forecast by region, production forecast by region, and production and revenue forecast.

Report Methodology and Data Source Used: This report includes the publishers disclaimer, list of authors, major primary and secondary sources, and research approach.

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The Gene Therapy market report keenly emphasizes on industrial affairs and developments, approaching policy alterations and opportunities within the market. The regional development methods and its predictions are explained in every key point that specifies the general performance and issues in key regions such as the US, Asia Pacific, Middle East, and Europe. Various aspects such as production capability, demand, product value, material parameters and specifications, distribution chain and provision, profit and loss, are explained comprehensively in the Gene Therapy market report.

The report hands in-depth segmentation of the worldwide market based on supported technology, product type, application, and numerous processes and systems. The report attains economical competitive analysis, business trends within the market, and alternative key characteristics of the worldwide Gene Therapy market. Our experts have genuinely concatenated the Gene Therapy market report by alluding the lists and figures, primary sources, with an intention to boost the understanding of the associated procedural terms and conditions.

Some of the major regions covered in this Gene Therapy Market report:

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The Gene Therapy market report includes the leading advancements and technological up-gradation that engages the user to inhabit with fine business selections, define their future-based priority growth plans, and to implement the necessary actions. The global Gene Therapy market report also offers a detailed summary of key players and their manufacturing procedure with statistical data and a profound analysis of the products, contribution, and revenue.

Every information given in the report is sourced and verified by our expert team and is collated with precision. To give a broad overview of the current global market trends and strategies led by key businesses, we present the information in a graphical format such as graphs, pie-charts with a superior illustration.

About Us:

Zion Market Research is an obligated company. We create futuristic, cutting-edge, informative reports ranging from industry reports, company reports to country reports. We provide our clients not only with market statistics unveiled by avowed private publishers and public organizations but also with vogue and newest industry reports along with pre-eminent and niche company profiles. Our database of market research reports comprises a wide variety of reports from cardinal industries. Our database is been updated constantly in order to fulfill our clients with prompt and direct online access to our database. Keeping in mind the clients needs, we have included expert insights on global industries, products, and market trends in this database. Last but not the least, we make it our duty to ensure the success of clients connected to usafter allif you do well, a little of the light shines on us.

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Global Gene Therapy Market Revenue Will Grow Over USD 2269 Million By 2024 - Industry PressRelease

Heres our recent analysis on Global Gene Therapy for Age-related Macular Degeneration Market research report 2019-2025 which primarily explains the crucial growth factors of the market that are impacting the Gene Therapy for Age-related Macular Degeneration industry along with the upcoming possibilities that are expected to influence the Gene Therapy for Age-related Macular Degeneration market during the forecast session between 2019 to 2025. In this study, the global Gene Therapy for Age-related Macular Degeneration market is evaluated through distinct industry elements such as restraints, opportunities, and drivers to help the users to have a better understanding about the fundamental overview of the Gene Therapy for Age-related Macular Degeneration market.

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In 2019, the global Gene Therapy for Age-related Macular Degeneration market size is valued at million US$ and it will be anticipated to gain million US$ by the end of the year 2025, which is boosting at a CAGR during the predicted period. While, in the Chinese continent, the Gene Therapy for Age-related Macular Degeneration market size is estimated at xx million US$ and it will grow about xx million US$ in 2025, with a CAGR of xx % from 2019-2025. The research report evaluates 2018 as the base year and 2019-2025 has been determined as the forecast timespan to estimate the Gene Therapy for Age-related Macular Degeneration market size.

Leading Players of the Gene Therapy for Age-related Macular Degeneration market are:

RetroSense TherapeuticsREGENXBIOAGTC

Gene Therapy for Age-related Macular Degeneration Market segmented by product types:

SubretinalIntravitrealUnspecified

Applications can be divided as:

MonotherapyCombination Therapy

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The research study accumulates the global Gene Therapy for Age-related Macular Degeneration market size on the basis of major topographical regions like Unites States, China and other zones including Japan, India, Korea, and Southeast Asia. Furthermore, the report firmly explains plenty of key factors including revenue share, Gene Therapy for Age-related Macular Degeneration market size, sales volume, and growth rate of each company. It also describes the statistical information regarding the revenue, Gene Therapy for Age-related Macular Degeneration market share and sales by regions, types, and applications. Meanwhile, it represents historical data from 2014 to 2019 and forecast to 2025.

This report analyzes essential details like price, production volume, market share, growth rate, and Gene Therapy for Age-related Macular Degeneration market value for the key players and collects informative data from 2014 to 2019 for the leading companies in USA, China, and European Union. Therefore, this study offers a brief introduction of the worldwide Gene Therapy for Age-related Macular Degeneration market along with in-depth evaluations for the revenue from 2019 to 2026 and determines market dimensions with respect to the provided aspects. The current industry vendors, stakeholders, and consultants are also included in the Gene Therapy for Age-related Macular Degeneration market in order to articulate the several paths and also implements gathered choices.

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Gene Therapy for Age-related Macular Degeneration Market Growth Analysis Reports by Companies RetroSense Therapeutics, REGENXBIO - The Chicago...

Zion Market Researchpublished a new industry research reportGlobal Gene Therapy Market Is Expected To Reach Around USD 2,269 Million By 2024is all around created with a blend of the significant information related with overall Gene Therapy Market, alongside key components obligated for the interest for its administrations and product.(Sample Copy Here)Gene Therapy Marketreport also provide a thorough understanding of the cutting-edge competitive analysis of the emerging market trends along with the drivers, restraints, challenges, and opportunities in the Market to offer worthwhile insights and current scenario for making right decision. The report covers the prominent players in the market with detailed SWOT analysis, financial overview, and key developments of the products/services from the past three years. Moreover, the report also offers a 360 outlook of the market through the competitive landscape of the global industry player and helps the companies to garnerGene Therapy Marketrevenue by understanding the strategic growth approaches.

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Table of Contents

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The Gene Therapy Market report includes the leading advancements and technological up-gradation that engages the user to inhabit with fine business selections, define their future-based priority growth plans, and to implement the necessary actions. The global Gene Therapy Market report also offers a detailed summary of key players and their manufacturing procedure with statistical data and profound analysis of the products, contribution, and revenue.

This report focuses on price, sales, revenue and growth rate of each type, as well as the types and each type price of key manufacturers, through interviewing key manufacturers. Second on basis of segments by manufacturers, this report focuses on the sales, price of each type, average price ofGene Therapy Market, revenue and market share, for key manufacturers.

Browse Press Release@www.zionmarketresearch.com/news/gene-therapy-market

This report focuses on Time and Expense Management System volume and value at global level, regional level and company level. From a global perspective, this report represents overall Time and Expense Management System market size by analyzing historical data and future prospect. Regionally, this report focuses on several key regions: North America, Europe, China and Japan. At company level, this report focuses on the production capacity, ex-factory price, revenue and market share for each manufacturer covered in this report.

Available Array of Customizations:

The classification of the global Gene Therapy Market is done based on the product type, segments, and end-users. The report provides an analysis of each segment together with the prediction of their development in the upcoming period. Additionally, the latest research report studies various segments of the global Gene Therapy Market in the anticipated period.

Also, Research Report Examines:

Lastly, with a team of vivacious industry professionals, we offer our clients with high-value market research that, in turn, would aid them to decipher new market avenues together with new strategies to take hold of the market share.

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Global Gene Therapy Market to Accumulate Revenues Worth US $2269 Million By 2024 - Market News Network

AUSTIN, Texas & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genprex, Inc. (NASDAQ: GNPX), a clinical-stage company and leader in gene therapy using non-viral vector transfection delivery, wishes to draw attention to additional research in the field validating non-viral vector delivery as the next evolution in gene therapy.

A recently published paper by researchers in Australia, Spain and Austria supports the belief that non-viral delivery could be safer for patients than viral vectors and could speed up the production time while reducing the costs of production. The paper titled, Encapsulation, Visualization and Expression of Genes with Biomimetically Mineralized Zeolitic Imidazolate Framework-8 (ZIF-8) published in the September 4, 2019 issue of the scientific journal Small, presents data from a biomolecule-metal-organic framework (nano MOF) in zeolitic imidazolate framework-8 (ZIF-8) and found it to be a viable vehicle for intracellular transfection and gene delivery. Genprex was not involved in the study, which used a different nanotechnology.

One of the biggest differentiators between Genprex and other gene therapy companies developing technologies to treat cancer and other serious diseases is our proprietary non-viral nanoparticle delivery system, which has already been used to safely treat more than 50 patients to date, said Genprexs Chairman and Chief Executive Officer, Rodney Varner. Most gene therapy research has been focused on using viral delivery systems to deliver genes to cancer cells, and today most approved gene therapies for non-blood cell therapies use a viral vector to deliver the gene to the patient. Our proprietary non-viral delivery system enables us to potentially treat patients with a system that may be safer, with lower production costs and better scalability.

Based on the shortcomings that viral vectors have historically had, including severe adverse reactions, high production costs, difficulty in scaling and high immunogenicity responses, Genprexs founders partnered with the National Institutes of Health (NIH) during the companys inception to develop its proprietary non-viral delivery system.

Specifically, Genprexs platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The nanovesicles are non-immunogenic, allowing repetitive therapeutic dosing. Genprexs nanovesicles are also clinically proven to effectively deliver molecular kinase inhibitors effectively.

A Phase I human clinical trial showed that Genprexs lead drug candidate, Oncoprex immunogene therapy, which is delivered through its nanovesicle non-viral delivery system, selectively and preferentially targeted primary and metastatic tumor cells, resulting in clinically significant anticancer activity. Genprexs clinical trials have also demonstrated that its delivery system is well tolerated in humans and can safely deliver high therapeutic doses.

About Genprex, Inc.

Genprex, Inc. is a clinical stage gene therapy company developing potentially life-changing technologies for cancer patients, based upon a unique proprietary technology platform, including Genprexs initial product candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC). Genprexs platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. Oncoprex has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for apoptosis, or programmed cell death, in cancer cells, and modulates the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. For more information, please visit the companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effects of Oncoprex on cancer and the safety, production cost and scalability of Oncoprex and its non-viral delivery system. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of Oncoprexs effect on cancer, the safety, cost and scalability of Oncoprex and its delivery system, as well as the timing and success of our clinical trials and planned clinical trials, Oncoprex and our other potential product candidates. These and other risks and uncertainties associated with Genprex and its lead product candidate Oncoprex are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex's Pioneering Use of Non-Viral Delivery for Gene Therapy is Gaining Industry Support - Business Wire

Scientists have developed a fast, efficient and economical method to create viral delivery vectors used in gene therapy to deliver modified versions of genes to treat disorders caused by genetic defects, including spinal muscular atrophy (SMA).

The findings were reported in the study, Production of adeno-associated virus vectors for in vitro and in vivo applications, and published in Scientific Reports.

Gene therapy is a relatively new approach that has been gaining popularity in clinical practice as a way to treat diseases caused by genetic mutations. It involves delivering a functional version of a gene to correct or replace a faulty gene within specific cells in the body.

In order to deliver the corrected version of the gene to cells, researchers use special viral vectors that work as carriers. Adeno-associated viruses, or AAVs, are one of the most used viral vectors in gene therapy due to their ability to infect and deliver the corrected gene to both dividing and non-dividing cells, without causing any harm to patients.

Gene therapy already is being used as a form of SMA treatment. The recent approval of Zolgensma (AVXS-101), a gene therapy co-developed by AveXis and Novartis, for the treatment of all types of SMA in newborns and toddlers up to the age of 2, was a historical landmark that highlighted the potential of AAV-based gene therapies to treat rare genetic disorders.

Most protocols recommend AAV purification from producer cells, grown in large cell stacks or cell culture factories to obtain sufficient AAVs for animal experiments. However, producer cells also release large quantities of AAV into the culture medium, which often remains unused, the investigators said.

In this study, researchers from the Boston University School of Medicine (BUSM) described a new protocol that allows them to maximize the quantity of AAVs that can be purified from producer cells and their culture medium in a fast, efficient and economic way.

With the new protocol, which involved several separation and purification laboratory techniques, they managed to obtain up to one milliliter of AAVs at a concentration of 10101011 viral genome copies per microliter, using five times less the number of producer cells normally used in conventional protocols.

They also showed the AAVs produced with the new protocol were viable and retained their ability to efficiently infect and deliver modified genes to cells in vitro and in vivo (outside and inside an organism, respectively).

Our protocol helps to produce AAVs efficiently and economically in regular laboratories so that researchers can easily conduct pre-clinical trialsforgene therapy, Markus Bachschmid, PhD, assistant professor of medicine at BUSM and corresponding author of the study, said in a press release.

Several labs in the Boston area and Japan have already tested this new protocol and found it useful, said Reiko Matsui, MD, assistant professor of medicine at BUSM and co-corresponding author of the study. Our hope is that many laboratories can adapt these procedures to accelerate research and promote gene therapy.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 85

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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New Method Produces High Quantities of Viral Vectors for Gene Therapy - SMA News Today

Food and Drug Administration leaders are concerned the agency won't be able to recruit and retain sufficient numbers of experts in fields that have undergone rapid scientific advances, such as cell and gene therapy or oncology.

In cell and gene therapy, the relevant FDA review unit aims to double in size over the next three to five years, competing with biopharmaceutical companies and venture firms for the same talent.

"Sometimes they're competing to actually pick people from our agency that's always fun," Peter Marks, director of the FDA's Center for Biologics Evaluation and Review, said Monday at the Biopharma Congress in Washington. "We're dealing with the issue of a very competitive job market."

Former agency head Scott Gottlieb, speaking at the same event, called recruitment and retention "an unrecognized challenge for the FDA."

In some respects, the FDA finds itself disadvantaged in enticing candidates who are in demand to fill high-paying private sector jobs. Congress acknowledged as much in the 21st Century Cures Act, a law passed in December 2016 that, among many other things, granted the regulator authority to pay some positions annual salaries of up to $400,000.

Marks said that flexibility will partially help address hiring problems, but doesn't see the issue going away, particularly given the anticipated expansion of the FDA's cell and gene therapy unit.

"We'll use that to the maximum, but it's going to continue to be a bit of a challenge," he added.

Other FDA leaders at Monday's event brought up alternative ways they hope to keep talent at the agency.

Gideon Blumenthal, the deputy director of the FDA's Oncology Center of Excellence, said they've shifted to a more academic model for the unit's roughly 100 medical oncologists.

A scientific liaison program encourages those FDA employees to take on more beyond the day-to-day review work. With it, those reviewers can become the agency's point person for a specific topic, such as breast cancer, melanoma or a research topic like health disparities, Blumenthal said.

"This is something we are really trying to push," he added. "We think it'll benefit not only the external community in understanding the FDA and oncology, but it'll help retain and attract the base talent, because not only are they reviewers, but they are really thought leaders in their fields."

In fast-moving fields, the FDA has also had difficulty filling its advisory committees with relevant, non-conflicted experts.

Janet Woodcock, a longtime FDA official and director of the Center for Drug Evaluation and Research, pointed to conflicts of interest policies as a sticking point.

"The ACs just have so many rules and lawyers ... it's really difficult to get people with the greatest expertise," she said Monday.

CBER's Marks agreed, adding that growth in rare disease drug development has made finding the right people more onerous.

"The conflict rules become so difficult that what you end up putting together is something that gets watered down ... to the point that you really can't have a discussion that's actually meaningful."

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FDA leaders worry agency will lose out on talent in gene therapy, cancer fields - BioPharma Dive

Clustered Regularly InterSpaced Palindromic Repeats (CRISPR) is a new technology for gene editing, with promising potential in medicine and basic research. CRISPR can be found in the DNA of bacteria. There are many different types of CRISPR DNA, but they all make proteins that protect bacteria against viruses, much like the human immune system functions to kill off viruses. The way CRISPR proteins do this is to cut the DNA of viruses think of them as molecular scissors preventing the virus from growing.

CRISPR has been in the news a lot recently. Some of the news coverage has been positive (promising new therapies) and some of it has been negative (CRISPR babies).

Scientists discovered that CRISPR proteins could be adapted to cut the DNA of other organisms, including humans. This led to demonstrations that CRISPR could be used for gene editing and gene therapy. The most commonly used CRISPR protein is called Cas9.

As with all game-changing technologies, attention has focused on ethically responsible uses of the technology, and rightly so. What is often missing from many CRISPR debates is an understanding of technical limitations that could impact how and when CRISPR is used in human gene-editing applications.

Read more: Opening Pandora's Box: Gene editing and its consequences

My laboratory became interested in CRISPR because we were working on technologies for gene editing. It quickly became evident that CRISPR and Cas9 were the future of gene editing because Cas9 was significantly easier to use than other technologies but, like every new technology, was ripe for improvement.

One of the biggest issues when trying to modify human DNA is the problem of so-called off-target effects. Off-target effects arise when Cas9 cuts a piece of DNA that it wasnt programmed to do.

In describing off-target effects, I like the analogy of a programming a car GPS unit with the address Tim Hortons. In any Canadian city or town, this search will result in multiple individual Tims locations. But what is the correct location?

In much the same way, Cas9 is guided to its DNA target by a small piece of ribonucleic acid (RNA), appropriately called a guideRNA. If the address specified by the guideRNA is not unique, Cas9 will be guided to multiple locations where it will cut the DNA. The address in this case is a continuous DNA sequence in the human genome, typically 20 base pairs in length.

Why are off-target effects an issue? Not all 20 base pair stretches are unique in the genome, and Cas9 has the added problem of being able to recognize sites that are not perfect matches to the 20 base pair address (these are called off-target sites). This means that there is always the potential for Cas9 to cut DNA at off-target sites that could lead to unwanted and serious side effects, including cancer. For any human therapeutic application using gene editing, minimizing the potential for off-target effects is paramount.

Off-target effects are not a new issue in the gene-editing field, and the potential for Cas9 to cause off-target cuts was recognized as a serious issue soon after Cas9 was identified.

Significant efforts by multiple research groups resulted in the development of engineered versions of Cas9 with greater specificity and fewer off-target effects. Scientists are finding new CRISPR-related proteins with novel applications in gene therapy.

A related problem is predicting off-target sites and identifying where Cas9 cuts in the human genome. In theory, finding potential Cas9 off-target sites should be straightforward. Simply search the three billion base pairs of the human genome for matches to the 20 base pair target (this is easier than it sounds and can be done on a laptop with basic programming experience).

Some studies have found that predicted off-target sites dont always match up with experimentally identified Cas9 off-target sites, whereas other studies have found low numbers of Cas9 off-target sites. These findings underscore the need to understand how Cas9 recognizes DNA so that improvements can be made to software that predicts Cas9 sites.

For gene editing and gene therapy applications done in somatic tissues, such as blood cells, DNA changes are not heritable. In these cases, a higher amount of off-target effects may be tolerated. Any off-target effects are unacceptable simply because they are difficult to detect and reverse. Until these issues are resolved, the question of off-target effects should not be ignored in debates about CRISPR gene editing.

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Gene editing needs to become more precise to live up to its promise - The Conversation CA

By Curtis Sprung, freelance reporter

This article was written for our sponsor, the Sanford Area Growth Alliance.

It's not every day a major corporation decides to invest half a billion dollars in your community, but that's what happened for the town of Sanford in August.

Pharmaceutical giant Pfizer announced plans for a state-of-the-art gene therapy manufacturing facility in Sanford as part of the company's ongoing investment in the area. Pfizer already has approximately 650 employees in Sanford, and the new facility is expected to add around 300 new jobs to the area.

"This investment will further strengthen Pfizer's leadership in gene therapy manufacturing technology," said Mike McDermott, president of Pfizer Global Supply, in a press release. "The expansion of the Sanford site is expected to create hundreds of highly skilled jobs which would increase Sanford's high-tech manufacturing environment and is part of our overall plan to invest approximately $5 billion in U.S.-based capital projects over the next several years."

The investment is just the next step in a long history of life science successes in the area. That history has primed Sanford to be a major player in the life sciences field for years to come.

"It speaks to the future of manufacturing realm here in Sanford," said Sanford Area Growth Alliance Existing Industry Development Manager Jimmy Randolph. "It's sure to attract other folks active in that space because of the significant life sciences activity in the Research Triangle area and Sanford is so proximate to that facility. It's a wonderful affirmation of the activity we've been engaged in."

Pfizer has locations across the globe, but ultimately, Sanford was able to provide a proven workforce, dedicated acreage, and a community college educating potential future employees in life sciences and manufacturing to close the deal.

Such a heavy investment means positive changes for the community and other companies that call Sanford home.

"Beyond the obvious things, I think what this will do is be a catalyst for further growth," Randolph said. "If you look at Sanford on the map, I think you can expect to see the rate of population growth and additional investment from an industrial manufacturing standpoint. Folks are already contacting us about additional space near the campus."

The continued investment in Sanford has resulted in quality of life improvements for residents ranging from new charter schools to the recently opened splash pad to the renovated downtown. All of this stems from a plan put in place nearly a decade ago to revitalize Sanford and continue to attract modern businesses and create a kind of economic development snowball effect, with improvements across many community aspects.

The Pfizer investment marks a major milestone for Sanford, but local leadership and SAGA board members are confident the growth will continue at a manageable pace. With their strong history of manufacturing, Sanford and Lee County remains open for business with an engaged community that's excited to see what happens next.

"The reality is, [we] are fairly far sighted," Randolph said. "The community around Sanford has a very favorable attitude toward manufacturing. I can't imagine a better place to live or work. The businesses that make it that way it really is a testament to the character of the citizens of this community."

This article was written for our sponsor, the Sanford Area Growth Alliance.

Continued here:
Pfizer to build a $500M gene-therapy facility in Sanford - WRAL.com

ST. PAUL, Minn., Oct. 7, 2019 /PRNewswire/ -- Recombinetics today announced a collaborative research project with Mayo Clinic to advance Osteogenesis Imperfecta (OI) research by developing the first-ever swine models of OI. These models will help preclinical researchers better understand disease etiology and progression and provide a reliable preclinical model for establishing the safety and efficacy of new therapies that patients with OI desperately need.

Also known as "brittle bone disease", clinical manifestations of OI vary from a mild increase in fractures to severe bone deformities, hearing loss and death in the neonatal period. To date, no treatment corrects the underlying cause or alleviates the complications of OI. Currently, research is hampered by mouse models that do not fully recapitulate the disease seen in patients and have been poor predictors of clinical efficacy. Providing the research community with gene-edited swine models that more closely mimic the human disease, could lead to better treatments, including in vivo gene-editing and gene therapy, orthopedic devices for intramedullary rodding, noninvasive techniques for malocclusion, and understanding the natural progression of bone mineralization after currently available therapeutics.

Project efforts will be led by Adrienne Watson, Ph.D., Recombinetics' Vice President of Research & Development and Mayo Clinic's David Deyle, MD, partially funded by an SBIR grant from the National Institutes of Health.

About RecombineticsFounded in 2008, Recombinetics (RCI) is producing gene-edited animals for biomedical and food production purposes and is generating commercial and collaborative revenues. RCI's technology platform supports three business lines: Acceligen (precision breeding to enhance health, well-being and productivity in food animals and aquaculture); Surrogen (gene-edited swine models of human diseases for biomedical research and pre-clinical trials by pharmaceutical and medical device companies); and Regenevida (development of human regenerative products including cells, tissues and organ products in swine models for exotransplantation to humans). Learn more at Recombinetics.com.

Contact:Kris Huson(651) 757-0427224755@email4pr.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/recombinetics-announces-collaboration-with-mayo-clinic-to-develop-the-first-ever-swine-models-of-osteogenesis-imperfecta-300931565.html

SOURCE Recombinetics

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Recombinetics Announces Collaboration with Mayo Clinic to Develop the First-Ever Swine Models of Osteogenesis Imperfecta - BioSpace

-- Improvements on functional measures seen in all three participants --

-- Significant reduction in creatine kinase maintained over nine months --

-- Results follow positive and robust expression and biomarker data presented earlier in 2019 --

CAMBRIDGE, Mass., Oct. 04, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (SRPT), the leader in precision genetic medicine for rare diseases, today announced the nine-month functional results from three Limb-girdle muscular dystrophy Type 2E (LGMD2E) clinical trial participants who received SRP-9003. SRP-9003 is an investigational gene therapy intended to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protein, the lack of which is the sole cause of LGMD2E.

In Cohort 1 of the SRP-9003 study, three participants ages 4-13 were treated with an infusion of SRP-9003 at a dose of 5x1013vg/kg. Improvements in functional outcomes were observed at day 270 (nine months) for all three participants.

We have now observed consistent functional improvements, in addition to high levels of expression of the missing protein of interest and strong results in related biomarkers, in both of our first cohorts for Duchenne muscular dystrophy (SRP-9001) and LGMD2E (SRP-9003). We intend to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible, said Doug Ingram, Sareptas president and chief executive officer. With the results of our first LGMD2E cohort, Sarepta continues to build its gene therapy engine, an enduring model created to design, develop and bring to the medical and patient community transformative therapies for those living with, and too often dying from, rare genetic disease.

At Day 270, mean creatine kinase (CK) was significantly reduced compared to baseline. CK is an enzyme biomarker strongly associated with muscle damage.

At Day 270, all three participants showed improvements from baseline across all functional measures, including the North Star Assessment for Dysferlinopathy (NSAD), time to rise, four-stair climb, 100-m walk test and 10-meter walk test. These results are distinctly different from what an age-matched, natural history group would predict.

No new safety signals were observed and the safety profile seen to date supports the ability to dose escalate in the next cohort of the study. As previously disclosed, two participants in the study had elevated liver enzymes, one of which was designated a serious adverse event (SAE), as the participant had associated transient increase in bilirubin. Both events occurred when the participants were tapered off oral steroids and, in both instances, elevated liver enzymes returned to baseline and symptoms resolved following supplemental steroid treatment.

LGMD2E is a devastating neuromuscular disease with no current treatment options so we are very pleased to observe a functional improvement in study participants who received SRP-9003, said Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Childrens Hospital and lead investigator for the study.

Sarepta had previously shared expression results from the study, which found that in two-month post-treatment muscle biopsies, clinical trial participants showed a mean of 51% beta-sarcoglycan positive fibers, as measured by immunohistochemistry (IHC), substantially exceeding the pre-defined 20% measure for success. Mean fiber intensity, as measured by IHC, was 47% compared to normal control.

About SRP-9003 and the Phase I/IIa Gene Transfer Clinical Trial

SRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle without promiscuously crossing the blood brain barrier, making it an ideal candidate to treat peripheral neuromuscular diseases. As a rhesus monkey-derived AAV vector, AAVrh74 has lower immunogenicity rates than reported with other common human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with LGMD2E, many of whom die from pulmonary or cardiac complications.

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This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of four and 15 years with significant symptoms of disease.

About Limb-Girdle Muscular Dystrophy

Limb girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with LGMD2E begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to death before age 30. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

About Sarepta Therapeutics

Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding our intention to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible; Sarepta continuing to build an enduring gene therapy model created to design, develop and bring to the medical and patient community transformative therapies for those living with rare genetic disease; the safety profile of SRP-9003 seen to date supporting the ability to dose escalate in the next cohort of the study; SRP-9003 being an ideal candidate to treat peripheral neuromuscular diseases; the potential benefits of the AAVrh74 vector and the MHCK7 promoter; and our mission to profoundly improve and extend the lives of patients with rare genetic-based diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: success in preclinical testing and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and initial results from a clinical trial do not necessarily predict final results; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications Sarepta utilizes to assess particular safety or efficacy parameters may yield different statistical results, and even if Sarepta believes the data collected from clinical trials of its product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; Sareptas ongoing research and development efforts may not result in any viable treatments suitable for clinical research or commercialization due to a variety of reasons, some of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve any significant revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2018, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website at http://www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

Excerpt from:
Sarepta Therapeutics Announces Positive Functional Results from the SRP-9003 (MYO-101) Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type...

Vancouver, British Columbia--(Newsfile Corp. - October 7, 2019) - PreveCeutical Medical Inc.(CSE: PREV) (OTCQB: PRVCF) (FSE: 18H)(the "Company" or "PreveCeutical"), is pleased to announce that it has received a cash refund of $616,802 AUD from the Australian Taxation Office under the Research and Development ("R&D") Tax Incentive Program. The cash refund is related to expenditures on eligible R&D activities conducted in Australia during the Company's 2018 financial year. The R&D activities included work done in areas across PreveCeutical's portfolio, including the Sol-gel nose-to-brain drug delivery system and the non-addictive analgesics programs, which are being conducted at the University of Queensland, Australia.

The refund received by the Company will support and reinforce the Company's continued investments in its R&D programs.

The R&D Tax Incentive Program encourages companies to engage in R&D programs, including ones that have the potential to improve global health outcomes, which boosts competitiveness and generates economic benefits locally.

PreveCeutical's President and Chief Science Officer, Dr. Mak Jawadekar stated, "We are highly supportive of the Australian Government's R&D Tax Credit incentive which recognises the critical role of R&D involved in potentially developing life-saving drugs, therapies and delivery devices. This refund would help enable PreveCeutical to further advance our proprietary therapeutic alternatives for preventive and curative therapies for some of the unmet medical needs."

About PreveCeutical

PreveCeutical is a health sciences company that develops innovative options for preventive and curative therapies utilizing organic and nature identical products.

PreveCeutical aims to be a leader in preventive health sciences and currently has five research and development programs, including: dual gene therapy for curative and prevention therapies for type 2 diabetes and obesity; a soluble gel drug delivery program; Nature Identical peptides for treatment of various ailments; non-addictive analgesic peptides as a replacement to the highly addictive analgesics such as morphine, fentanyl and oxycodone; and a therapeutic product for treating athletes who suffer from concussions (mild traumatic brain injury).

For more information about PreveCeutical, please visit http://www.PreveCeutical.com, follow us on Twitter: http://twitter.com/PreveCeuticals and Facebook: http://www.facebook.com/PreveCeutical.

On Behalf of the Board of Directors

"Dr. Makarand (Mak) Jawadekar"President & Chief Science Officer

For further information, please contact:

Deanna KressDirector of Corporate Communications & Investor Relations+1-778-999-6063deanna@PreveCeutical.com

Forward-Looking Statements:

This news release contains forward-looking statements and forward-looking information (collectively, "forward-looking statements") within the meaning of applicable Canadian and U.S. securities legislation, including the United States Private Securities Litigation Reform Act of 1995. All statements in this news release that are not purely historical are forward-looking statements and include any statements regarding beliefs, plans, expectations and orientations regarding the future including the Company's anticipated business plans, the intended use of the R&D Tax Incentive Program cash refund, and the prospect of its ability and success in executing its proposed plans. Often, but not always, forward-looking statements can be identified by words such as "pro forma", "plans", "expects", "may", "should", "budget", "schedules", "estimates", "forecasts", "intends", "anticipates", "believes", "potential", "will" or variations of such words including negative variations thereof and phrases that refer to certain actions, events or results that may, could, would, might or will occur or be taken or achieved. Forward looking statements are based on certain assumptions regarding the Company, including expected growth, results of operations, including the Company's research and development activities, performance, industry trends, growth opportunities, and that the Company will be able to obtain the financing required to carry out its planned future activities, retain and attract qualified research personnel and obtain and/or maintain the necessary intellectual property rights it needs to carry out its future business activities. Actual results could differ from those projected in any forward-looking statements due to numerous factors including risks and uncertainties relating to the inability of the Company, to, among other things, obtain any required governmental, regulatory or stock exchange approvals, permits, consents or authorizations required, including Canadian Securities Exchange acceptance of any planned future activities, commercialise therapeutic and diagnostic technologies, pursue business partnerships, complete its research programs as planned, and obtain the financing required to carry out its planned future activities. Other factors such as general economic, market or business conditions or changes in laws, regulations and policies affecting the biotechnology or pharmaceutical industry, may also adversely affect the future results or performance of the Company. These forward-looking statements are made as of the date of this news release and, unless required by applicable law, the Company assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in these forward-looking statements. Although the Company believes that the statements, beliefs, plans, expectations, and intentions contained in this news release are reasonable, there can be no assurance that those statements, beliefs, plans, expectations, or intentions will prove to be accurate. Readers should consider all of the information set forth herein and should also refer to other periodic reports provided by the Company from time-to-time. These reports and the Company's filings are available at http://www.sedar.com.

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Readers are cautioned that forward-looking statements are not guarantees of future performance or events and, accordingly, are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty of such statements.

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/48515

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PreveCeutical Receives $616,802 AUD Research and Development Tax Incentive Cash Refund - Yahoo Finance

For the past 15 years the University of Oxfords Simon Davis has been mapping the surface of T cells, exploring and examining the structures of surface proteins while determining what it takes to manage specific immune cell signaling. And now a group of UK investors says its ready to advance that research toward the clinic, inside a new biotech vehicle theyre setting up to take the tech into the commercial sphere.

Enter MiroBio, stage left. The newly crafted company has raised $34 million to launch their antibodies into the clinic. Oxford Sciences Innovation and Samsara Biocapital co-led the round, joined by Advent Life Sciences and SR One.

The plan is to use Davis insights and preclinical antibodies to hijack the natural mechanisms used to control immune cells for the purpose of targeting relevant diseases. And while the research has had obvious applicability in oncology where cell therapy evangelists are looking to develop the next wave of more sophisticated therapies MiroBio is starting out in autoimmune diseases, where errant attacks on healthy tissue trigger some major market ailments.

If you look at things from an investors perspective against a backdrop of intense research activity in oncology, says Executive Chairman Eliot Charles, the real opportunity near term was autoimmune disease.

Not that they arent interested in oncology.

For now, though, the emphasis is using the expertise at the venture groups to guide the company while they build out their team in the UK and follow up on the in vivo models that Davis worked with. And the first goal is resetting the immune system when it goes awry, as happens in autoimmune diseases.

Charles an Amgen vet whos now a venture partner at SR One, jumped on the phone with me early Monday to discuss the work with VP Operations Tim Funnell. Samsaras Bob Stein has stepped in as interim CSO as they follow up on 4 programs in-licensed from Davis lab, building out the initial team of 6 staffers to 15 or so.

The investor group has worked together on various projects through the years, says Charles, which is how the transatlantic syndicate came together to back Davis work in a startup.

On his website, Davis notes:

Our present goals are (1) to show that the kinetic segregation model does indeed explain T-cell receptor triggering, and (2) to use the idea to develop new types of therapeutic antibodies. The signaling concept is being tested using structural approaches and super-resolution imaging techniques, such as dSTORM. For this, the behavior of T-cell surface proteins is being studied at contacts with glass surfaces and supported lipid bilayers, in collaboration with Professor Klenerman. New, potentially therapeutic superagonistic antibodies are being developed and licensed to industry in collaboration with Professor Richard Cornall.

Its early days, of course, but that process has inspired a spinout with big plans.And they have enough cash to get to human studies on the lead, while building up a pipeline behind it.

See the article here:
Antibody research graduates from a top Oxford lab into the biotech world with $34M to fund R&D work - Endpoints News

Seven months after Oyster Point Pharma bridged its way into a Phase III study with $93 million in venture cash, the biotech is stepping back up to see if public investors are in a mood to back their play to jump into a big league market with a minor league team.

The Princeton, NJ-based biotech teed up an IPO on Friday, outlining their case on going after dry-eye disease a blockbuster market that accounts for tens of millions of patients. On the drug side, thats a market that has been dominated by Allergans cash cow Restasis now being carved up by generics as AbbVie buys out the company and Xiidra, a therapy which Takeda sold off to Novartis in a $5.3 billion deal in the wake of the Shire acquisition. Most patients get eye drops for the condition.

Tiny Oyster Points plan involves taking a mostly positive Phase IIb study where their drug, varenicline, a formulation of Chantix, managed to get the tear ducts to flow in a statistically significant manner and proving it works in a second registrational, confirmatory story. The FDA, according to the S-1, has signed off on using the Phase IIb as their first or 2 required pivotals. And that study, they maintain, is the first ever to show statistical significance against signs and symptoms of dry eye disease.

If they do get an approval, they believe that they can hire a sales force of 150 to 200 to get it out on the market. The biotech currently has a staff of 18, putting it in the leanest and meanest category of late-stage companies.

Whether they mean that or plan to use it as a classic bluff in preparation for a possible buyout deal is the big question. Startups traditionally have faced a mountainous challenge selling the drugs they manage to get approved, and thats even true in specialty cancer markets. For major markets like dry eye disease, the climb against Big Pharma and generics is distinctly more challenging.

The biotech has burned through $53 million and had $84 million banked at the end of H1.

New Enterprise Associates and Versant are the 2 big investors, with 32% of equity each. Jeffrey Nau, the CEO, has 2.1% of the shares going in.

Read the original:
Ophthalmology biotech upstart is asking investors to bet $85M-plus on their late-stage rival to Restasis and Xiidra - Endpoints News

Liver disease patients could one day benefit from a new cell therapy that has just completed its first clinical trial.

Researchers who tested the potential treatment in patients with liver cirrhosis where long term damage produces scarring found the therapy had no significant adverse effects.

Now the team, based at the Universitys MRC Centre for Regenerative Medicine, is to gauge the effectiveness of the treatment which is based on white blood cells called macrophages, that are key to normal liver repair.

The next stage of the trial will measure whether the therapy helps the liver to reduce scarring and stimulate regeneration. The results should be known within the next two years.

At present the only successful treatment for end-stage liver cirrhosis which claims around 14,000 lives in the UK each year (British Liver Trust) is an organ transplant. The safety trial is a vital step forward in finding an alternative therapy.

During the trial scientists took cells from the blood of nine patients with the disease and turned them into macrophages, in the Scottish National Blood Transfusion Services (SNBTS) cell therapy facility.

The new cells were then re-injected into the patient with the hope of repairing the damaged organ from within.

Causes of liver cirrhosis include infections such as hepatitis C, obesity, alcohol excess and some genetic and immune conditions.

Liver cirrhosis is a major healthcare issue in the UK and is one of the top five killers. The results from this first safety trial are encouraging and we can now progress to testing how effective it is in a larger group of people. If this was found to be effective it would offer a new way to tackle this important condition.

The research which was published in the journal Nature Medicine, received funding from the Medical Research Council and was conducted in partnership with the SNBTS and the Cell and Gene Therapy Catapult.

Excerpt from:
Cell therapy safe for liver patients, trial shows - Mirage News