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Archive for the ‘Gene therapy’ Category

UK Startup to Manufacture Cell and Gene Therapies with… – Labiotech.eu

Tuesday, April 28th, 2020

The UK company MicrofluidX has closed a 1.6M (1.4 M) seed funding round to develop a microfluidic platform that could produce cell and gene therapies more cheaply than conventional cell cultures.

The funding round was led by UKI2S, a national seed investment fund targeting early-stage companies, as well as Longwall Ventures and Cambridge Angels.

MicrofluidX will use the funding to establish a prototype of its technology. With this prototype, the company then aims to compare the performance of its microfluidics approach to current cell culture techniques used to produce gene and cell therapies.

In the dynamic cell therapy space, one of the major bottlenecks facing the field is the manufacture and scaling process, as manual cell culturing techniques are often required. Applying microfluidic technology to the manufacturing process may be an answer to this issue.

Although it is less developed at the manufacturing level, microfluidics technology has long been part of research in cell biology. It has several advantages over conventional cell cultures. For example, it allows cell cultures to be controlled more precisely on chips, increases automation, and can reduce the consumption of expensive ingredients in the cell culture process by a factor of twenty.

According to MicrofluidX, its platform could scale up microfluidics far beyond just biology research. The aim is to run dozens of cell cultures in parallel, with the capacity to produce cells more cheaply and with a higher yield than with current manufacturing techniques.

The result is that we can leverage all the inherent advantages of microfluidic cell culture at a scale never seen before, MicrofluidXs founder and CEO, Antoine Espinet, told me. This leads to much lower bioprocessing costs, better control over the final product, and faster translation from research to commercialization.

In particular, the company is investigating its technologys capacity to produce immune T-cells a common type of cell used in immunotherapies such as CAR T-cell therapies and other cell types.

Whilst the regulatory agencies are now warming up to cell and gene therapies, there are still growing pains, especially around manufacturing, Pablo Lubroth, an investor with UKI2S, told me.

It is essential to not only support companies that produce the therapies themselves, but also companies that are developing enabling technologies to ensure these therapies can be effectively commercialized and therefore have a tangible benefit to the patient.

As well as manufacturing, microfluidics is gaining traction in diagnostics and screening. For example, another UK startup, Lightcast Discovery, was founded last year to screen cells using microfluidics and beams of light. Additionally, the Belgian nanofluidics company miDiagnostics last month raised 14M to commercialize its silicon chip diagnostics in collaboration with Johns Hopkins University in the US.

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Vertex Pharmaceuticals and Affinia Therapeutics Establish Multi-Year Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies |…

Tuesday, April 28th, 2020

DetailsCategory: More NewsPublished on Monday, 27 April 2020 15:42Hits: 258

Affinia Therapeutics proprietary AAV vector technology to be used in Vertexs genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis

BOSTON, MA & WALTHAM, MA, USA I April 27, 2020 I Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and Affinia Therapeutics announced today that the two companies have entered into a strategic research collaboration to engineer novel adeno-associated virus (AAV) capsids to deliver transformative genetic therapies to people with serious diseases. Affinia Therapeutics proprietary AAVSmartLibrary and associated technology provides capsids for improved tissue tropism, manufacturability and pre-existing immunity. The collaboration will leverage Affinia Therapeutics capsid engineering expertise and Vertexs scientific, clinical and regulatory capabilities to accelerate the development of genetic therapies for people affected by Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and cystic fibrosis (CF).

This collaboration with Affinia Therapeutics will enhance our existing capabilities in discovering and developing transformative therapies for people with serious diseases, said Bastiano Sanna, Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. Affinia Therapeutics innovative approach to the discovery and design of AAV capsids brings yet another tool to our Vertex Cell and Genetic Therapies toolkit, and were excited to partner with them to bring together their technology platform with our research and development expertise.

At Affinia Therapeutics, were setting a new standard in genetic therapy by leveraging our platform to methodically engineer novel AAV vectors that have unique therapeutic properties, said Rick Modi, Chief Executive Officer. Vertex is an established leader in developing transformative medicines for genetic diseases and renowned for its scientific rigor. We are thankful for the scientific validation this partnership brings and look forward to working closely with them to advance life-changing, differentiated genetic therapies and make a meaningful difference to those affected by these diseases.

About the Collaboration

Under the terms of the agreement, Affinia Therapeutics will apply its vector design and engineering technologies to develop novel capsids with improved properties. The agreement provides Vertex an exclusive license under Affinia Therapeutics proprietary technology and intellectual property (IP) in DMD and DM1 with an exclusive option to license rights for CF and an additional undisclosed disease. The scope of the agreement covers all genetic therapy modalities in these diseases. Affinia Therapeutics will be eligible to receive over $1.6 billion in upfront and development, regulatory and commercial milestones, including $80 million in upfront payments and research milestones that will be paid during the research term, plus tiered royalties on future net global sales on any products that result from the collaboration. Affinia Therapeutics will be responsible for the discovery of capsids that meet certain pre-determined criteria. Vertex will be responsible for and will fund the design and manufacturing of genetic therapies incorporating the selected capsids, preclinical and clinical development efforts, and commercialization of any approved products in the licensed diseases.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need. http://www.affiniatx.com.

About Vertex Pharmaceuticals

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

SOURCE: Vertex Pharmaceuticals

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CEVEC Pharmaceuticals GmbH : Announces the Launch of the ELEVECTA(R) Platform – the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors…

Tuesday, April 28th, 2020

DGAP-News: CEVEC Pharmaceuticals GmbH / Key word(s): Product LaunchCEVEC Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors

28.04.2020 / 08:30 The issuer is solely responsible for the content of this announcement.

CEVEC Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors

Cologne, Germany, April 28th, 2020

CEVEC Pharmaceuticals GmbH (CEVEC), the leading provider of high-performance cell technology for the manufacturing of advanced biotherapeutics from research to production scale, today announced the launch of the ELEVECTA(R) platform, a unique technology to manufacture AAV (Adeno-associated viral) vectors at large scale. With the launch, CEVEC is the first and only provider on the market to offer AAV gene therapy vector manufacturing technology on the basis of fully stable producer cell lines.

The patent-protected technology has been proven by CEVEC in pilot projects with several partners and is now commercially available for Pharma and Biotech companies running gene therapy programs from late research to all clinical phases.

ELEVECTA(R) takes AAV vector manufacturing to the next level

With the ELEVECTA(R) technology, CEVEC has taken a unique approach based on producer cell lines, which have all necessary elements for AAV production stably integrated in one cell. Up to now, manufacturing of AAV - the most widely used vector for in vivo gene therapy - has required expensive transfection reagents and cGMP-grade plasmids. ELEVECTA(R) now overcomes these limitations and enables efficient, high-performance AAV production from one cell line in consistent quality. The technology can easily be implemented in bioprocess development and large-scale GMP manufacturing facilities running standard suspension bioreactor equipment widely used in industry for monoclonal antibody and recombinant protein production.

"The launch of ELEVECTA(R) forms a milestone on the way into a new era of viral vector manufacturing as it provides a solution for one of the major challenges in gene therapy development. By closing the production gap and providing the necessary quantities of viral vectors, it paves the way to address more common indications such as Alzheimer's, Parkinson's, or Rheumatoid Arthritis as well as therapies that require larger doses," said Nicole Faust, CEO of CEVEC Pharmaceuticals. "With this launch, we provide pharmaceutical and biotechnology companies with the opportunity to benefit right from the beginning from what we believe is the future for viral vector production. The superior features in terms of scalability, production efficiency and robustness of our stable producer cell lines position CEVEC at the leading edge in the rapidly growing field of gene therapy vector manufacturing."

ELEVECTA(R) closes the production gap in gene therapy viral vector production

Gene therapy is considered to be the most effective and often only treatment option for many severe and life-threatening diseases. Moreover, in a growing number of indications, gene therapies for the first time offer the opportunity to cure a disease. In addition, as gene therapy is moving from rare and ultra-rare to more common indications with larger patient numbers and systemic treatments, larger amounts of vector material are required. Consequently, production processes are needed that are fit for this purpose, enabling the manufacture of the necessary quantities of viral vectors with high yield and consistent quality.

ELEVECTA(R) - A concept to match the customers' needs

CEVEC's newly launched ELEVECTA(R) technology overcomes the limitations of existing manufacturing methods such as restricted scalability, time-consuming and cost-intensive material sourcing and complex production processes. ELEVECTA(R) technology is based on a patent-protected, fully documented human suspension cell line developed and optimized by CEVEC.

The ELEVECTA(R) technology works for any combination of serotype-specific capsid and therapeutic gene of interest. The stable integration of these components into the cell results in cell lines designed to produce highly functional AAV vectors. Custom-made ELEVECTA(R) Producer Cell Lines are available as research cell banks or as fully tested cGMP Master Cell Banks for manufacturing of clinical and commercial material.

CEVEC launches the technology in various formats matching the needs of customers in different development phases, with focus on gene therapy programs from late research to any clinical phases. CEVEC offers cell line development services to match the specific needs of gene therapy programs and various license packages from research and development to commercial manufacturing.

Further information:

To get a deeper insight into the ELEVECTA(R) technology register for a webinar on May 5th, at 5:00 p.m. CEST (11:00 a.m. EST) featuring CEVEC's CSO, Dr. Silke Wissing, talking about "Stable AAV producer cell lines: elevating vector manufacturing" and/or visit the ELEVECTA(R) web page.

In addition, you can meet CEVEC's management at the 23rd ASGCT virtual Annual Meeting taking place from May 12 - 15, 2020. During this event, interested parties can listen to the ELEVECTA(R) poster presentations and visit the CEVEC virtual booth. Please request a virtual meeting through the ASGCT webpage or directly contact CEVEC under bizdev@cevec.com.

About CEVEC:

CEVEC is a leading provider of high-performance cell technology for the manufacturing of advanced bio-therapeutics from R&D to manufacturing scale. The company's product portfolio comprises platform technologies for gene therapy viral vectors (AAV, Adenoviral vectors, Lentiviral vectors,), vaccines and complex recombinant proteins. With the ELEVECTA(R) Technology, CEVEC offers a unique solution for large-scale production of AAV vectors using producer cell lines with all necessary components stably integrated into the cell. The technology is based on suspension cells and does not require any expensive transfection reagents and cGMP plasmids. CEVEC's CAP(R) Technology based on human suspension cells is the ideal production platform for RCA-free Adenoviral vectors, Lentiviral vectors, Oncolytic viruses, Viral vaccines and exosomes. With the CAP-Go(R) Technology CEVEC provides a solution to the increasing need for recombinant production of complex and highly glycosylated protein molecules, including laminins, coagulation factors and plasma proteins.

For more information, please visit the Company's website.

Follow CEVEC on LinkedIn and Twitter.

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28.04.2020 Dissemination of a Corporate News, transmitted by DGAP - a service of EQS Group AG.The issuer is solely responsible for the content of this announcement.

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CEVEC Pharmaceuticals GmbH : Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors...

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MicrofluidX to take on single-use with microfluidics – Bioprocess Insider – BioProcess Insider

Tuesday, April 28th, 2020

MicrofluidX will use fundraising money to advance its cell bioprocessing technology, which uses microfluidics to tackle challenges associated with bioprocessing advanced therapies.

The London, UK-based firm has raised 1.4 million ($1.7 million) in seed funding to advance its microfluidics-based technology, which aims to combat challenges associated with the production of cell and gene therapies (CGTs) such as cost of goods, batch variability, and scalability.

Cell and gene therapies have demonstrated significant therapeutic potential. However, these treatments are now at a critical juncture, Antoine Espinet, CEO of MicrofluidX, told Bioprocess Insider. Complex manufacturing challenges remain and impact the development and accessibility of therapies to patients.

Image: iStock/barbol88

According to Espinet, current technologies lead to high treatment cost of manufacturing, driven by high reagent volumes, logistics-heavy centralized manufacturing in large specialized facilities, and low automation; product variability, both batch-to-batch and within each batch due to low control over all the parameters of the bioprocess; and long process re-engineering to scale from experimental batches to manufacture of therapeutics doses.

MicrofluidXs technology is based on microfluidics cell culture, which Espinet said holds several advantages over conventional single-use technologies. These include a 10-20 times lower reactant consumption, increased process efficiency, up to 50 times more cells per unit volume/surface area, and a high level of precision and control over all process parameters.

MicrofluidX leverages the power of microfluidics thanks to its highly parallelizable microfluidic bioprocessing system that combines multiple cell culture capabilities in one closed system and is fully scalable from thousands to several hundred million cells without the need to re-engineer your process: same yield, same quality, same results.

The financing will be used to build a working prototype that can be used to generate comparative biological data between this platform and conventional single-use technologies.

MicrofluidX is working to bring its products to the market quickly, Espinet said, and is looking to partner with academics, biotechs, pharmas and Contract Manufacturing Organizations (CMO) to develop and test its products on various cell and gene therapy processes.

In particular, an important piece of work is for those partners to run side-by-side comparisons with existing SUTs on their cells and processes. With these comparisons expected to show break-through improvements, MicrofluidX will enter into its industrialization and marketing phase, to make its technology available to the bioprocessing world.

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MicrofluidX to take on single-use with microfluidics - Bioprocess Insider - BioProcess Insider

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Inclusive Comprehension 2020: Gene Therapy Market (Trending PDF) Addressing Structure, Scope, Potential, and Growth Prospects Till 2029 – Jewish Life…

Tuesday, April 28th, 2020

Worldwide Gene Therapy Market has been thoroughly elaborated in a research report put on sale by Marketresearch.biz, bearing the title Gene Therapy MarketGlobal Industry Analysis, Size, Share, Growth, Trends, and Forecast 20202029. According to the report, the market is expected to be driven by a wide range of macroeconomic and industry-related factors. The key players in this market are focusing on product strategies and developments to increase their customer base and maintain their position.

The Gene Therapy Market is an intrinsic study of the current status of this business vertical and encompasses a brief synopsis about its segmentation. This industry report is inclusive of a nearly accurate prediction of the market scenario over the forecast period 20202029 market size with respect to valuation as sales volume. The study lends focus to the top magnates comprising the competitive landscape of Gene Therapy market, as well as the geographical areas where the industry extends its horizons, in magnanimous detail.

|| Access insightful study with over 100+ pages, list of tables & figures, profiling 10+ companies. Ask for Free Sample Copy(PDF) @https://marketresearch.biz/report/gene-therapy-market/request-sample

Operational and Emerging Players: Gene Therapy Market- Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

According to the current market situation, this report continuously observing promising growth of the global Gene Therapy market. The report further suggests market appears to progress at an accelerating rate over the forecast period. Also, the major players are elaborated on the basis of the proprietary technologies, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, report also examines R&D developments, legal policies, SWOT Analysis and strategies of Gene Therapy market players.

This Gene Therapy market research report surrounds importance on:

Manufacturing process and technology used in Gene Therapy market, key developments and trends changing in the development

Complete examination, including an evaluation of the parent market

Detailed account of market, volume and forecast, by leading key players, product type and end users applications

Environmental spread, development designs, pieces of the overall industry, key methodologies, and different financials systems of Gene Therapy market

Industrial analysis by upstream raw materials, downstream industry, current market dynamics and ensuing consumers analysis

|| Place Inquiry for further Details or Customization of Report:https://marketresearch.biz/report/gene-therapy-market/#inquiry

Gene Therapy Market Segmentation Outlook:

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

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Gene Therapy Market Section by Region:

ASIA-PACIFIC MARKET: China, Southeast Asia, India, Japan, Korea, Western Asia

THE MIDDLE EAST & AFRICA MARKET: GCC, North Africa, South Africa

NORTH AMERICA MARKET: United States, Canada, Mexico

EUROPE MARKET: Germany, Netherlands, UK, France, Russia, Spain, Italy, Turkey, Switzerland

SOUTH AMERICA MARKET: Brazil, Argentina, Columbia, Chile, Peru

Our Research Methodology is based on the following main points:

i. Data Collections and Interpretation

ii. Analysis

iii. Data Validation

iv. Final Projections and Conclusion

The latest research on the Gene Therapy Market fundamentally delivers insights that can empower stakeholders, business owners, and field marketing executives to make effective investment decisions driven by facts and extremely thorough research. The study aims to provide an evaluation and deliver essential information on the competitive landscape to meet the unique requirements of the companies and individuals operating in the Gene Therapy Market for the forecast period, 20202029. To help firms comprehend the Gene Therapy industry in multiple ways, the report exhaustively assesses the share, size, and growth rate of the business worldwide.

There are 13 Sections to show the global Gene Therapy market:

Chapter 1: Market Overview, Drivers, Segmentation overview, Restraints and Opportunities

Chapter 2: Market competition by key Manufacturers

Chapter 3: Production by Regions

Chapter 4: Consumption by Regions

Chapter 5: Production By Types, Revenue and Market share by Types

Chapter 6: Market share (%) and Growth Rate by Applications, Consumption By Applications

Chapter 7: Complete profiling and analysis of leading Manufacturers

Chapter 8: Region-wise manufacturing expenses, Manufacturing cost analysis, Raw materials analysis

Chapter 9: Sourcing Strategy, Industrial Chain and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Market Effect Factors Analysis, Impact Analysis

Chapter 12: Market Forecast 2020-2029

Chapter 13: Gene Therapy Research Findings and Conclusion, methodology and data source, Appendix

CLICK HERE, To Get Complete TOC

Conclusively, this report will provide you a clean view of each and every truth of the market without a need to consult another research report or a statistics supply. Our report will offer you with all of the facts about the past, present, and future of the Market.

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Glaucoma can be successfully treated with gene therapy – International Business Times, Singapore Edition

Tuesday, April 28th, 2020

Hackers leak data related to WHO, Wuhan lab and Gates Foundation

A common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients, say researchers.

Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight.

Current treatments include either eye drops, laser or surgery, all of which have limitations and disadvantages. "At present, there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early," said study researcher Dr Colin Chu from the University of Bristol in the UK.

For the findings, published in the journal Molecular Therapy, the research team tested a new approach that could provide additional treatment options and benefits. The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.

The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye. Using the latest gene-editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure.

"We hope to advance towards clinical trials for this new treatment in the near future. If it's successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money," Chu said. The researchers are currently in discussion with industry partners to support further laboratory work and rapidly progress this new treatment option towards clinical trials.

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Lysogene S.A. (EPA:LYS): When Will It Breakeven? – Simply Wall St

Tuesday, April 28th, 2020

Lysogene S.A.s (EPA:LYS): Lysogene S.A. engages in the research and clinical development of gene therapy for neurodegenerative disorders. The 64m market-cap company announced a latest loss of -4.3m on 31 December 2019 for its most recent financial year result. Many investors are wondering the rate at which LYS will turn a profit, with the big question being when will the company breakeven? Below I will provide a high-level summary of the industry analysts expectations for LYS.

Check out our latest analysis for Lysogene

According to the industry analysts covering LYS, breakeven is near. They expect the company to post a final loss in 2021, before turning a profit of 69m in 2022. LYS is therefore projected to breakeven around 2 years from now. In order to meet this breakeven date, I calculated the rate at which LYS must grow year-on-year. It turns out an average annual growth rate of 50% is expected, which is extremely buoyant. If this rate turns out to be too aggressive, LYS may become profitable much later than analysts predict.

Im not going to go through company-specific developments for LYS given that this is a high-level summary, however, keep in mind that by and large a biotech has lumpy cash flows which are contingent on the product type and stage of development the company is in. This means that a high growth rate is not unusual, especially if the company is currently in an investment period.

One thing Id like to point out is that LYS has managed its capital judiciously, with debt making up 13% of equity. This means that LYS has predominantly funded its operations from equity capital,and its low debt obligation reduces the risk around investing in the loss-making company.

There are too many aspects of LYS to cover in one brief article, but the key fundamentals for the company can all be found in one place LYSs company page on Simply Wall St. Ive also put together a list of relevant aspects you should further research:

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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Lysogene S.A. (EPA:LYS): When Will It Breakeven? - Simply Wall St

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Personalized Gene Therapy Treatment Market Analysis By Industry Size, Share, Revenue Growth, Development And Demand Forecast To 2029 – Germany English…

Tuesday, April 28th, 2020

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

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

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

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

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

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

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

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

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

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BRIEF-Novartis CEO Says Still Need Proper Blinded Studies To Determine Which Drugs Have Benefit for COVID-19 – Reuters UK

Tuesday, April 28th, 2020

April 28 (Reuters) - Novartis AG:

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

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

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

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

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

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

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

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

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

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BRIEF-Novartis CEO Says Still Need Proper Blinded Studies To Determine Which Drugs Have Benefit for COVID-19 - Reuters UK

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Gene Therapy For CNS Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 – Latest Herald

Tuesday, April 28th, 2020

Bluebird Bio

Global Gene Therapy For CNS Disorders Market Segmentation

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

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

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

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

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

South America (Brazil, Argentina etc.)

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

Some Notable Report Offerings:

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

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

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

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

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

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

Table of Contents:

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

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

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

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

About Us:

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

Contact Us:

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Market Research Intellect

New Jersey ( USA )

Tel: +1-650-781-4080

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

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

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

Tuesday, April 28th, 2020

Analysis Report on Gene Therapy Market

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

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

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

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

COVID-19 Impact on Gene Therapy Market

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

The global Gene Therapy market segment by manufacturers include

segmented as follows:

Global Gene Therapy Market, by Product

Global Gene Therapy Market, by Application

Global Gene Therapy Market, by Region

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Important queries addressed in the report:

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

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

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

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Future Prospects of Plasmid DNA Manufacturing Market by 2026 with Top Key Players Cobra Bio, Richter-Helm, Eurogentec, Cell and Gene Therapy…

Tuesday, April 28th, 2020

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

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

Leading companies Influencing in this Market are:

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

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

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

Global Plasmid DNA Manufacturing Market Segmentation:

Segmentation by Type:

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

Segmentation by Application:

CancersInherited DisordersViral InfectionsOthers

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Key questions answered in the report include:

Table of Contents

Global Plasmid DNA Manufacturing Market Research Report 2020 2026

Chapter 1 Plasmid DNA Manufacturing Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

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

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

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Plasmid DNA Manufacturing Market Forecast

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

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Gene Therapy For Inherited Genetic Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 – Latest Herald

Tuesday, April 28th, 2020

Spark Therapeutics Inc.

Global Gene Therapy For Inherited Genetic Disorders Market Segmentation

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

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

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

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

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

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

South America (Brazil, Argentina etc.)

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

Some Notable Report Offerings:

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

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

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

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

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

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

Table of Contents:

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

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

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

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

About Us:

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

Contact Us:

Mr. Steven Fernandes

Market Research Intellect

New Jersey ( USA )

Tel: +1-650-781-4080

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

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Gene Therapy For Inherited Genetic Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald

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Gene Therapy – The Future Is Here! – MedicineNet

Saturday, April 11th, 2020

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

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

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

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

Uses of gene therapy

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

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

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

Delivery of genes into cells

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

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

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

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

A "bionic chip"

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

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

Route of administration of gene therapy

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

The potential power of gene therapy

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

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

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

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

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

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Gene Therapy - The Future Is Here! - MedicineNet

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Evotec Expands into Gene Therapy – Associated Press

Saturday, April 11th, 2020

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

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

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

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

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

About Gene Therapy

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

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

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

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

Contact Evotec SE:

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

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

SOURCE: Evotec AG via EQS Newswire

View source version on accesswire.com:

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

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Evotec Expands into Gene Therapy - Associated Press

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Evotec allies with Takeda to move into gene therapy R&D – FierceBiotech

Saturday, April 11th, 2020

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

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

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

Virtual Clinical Trials Online

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

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

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

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

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Evotec allies with Takeda to move into gene therapy R&D - FierceBiotech

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Evotec Makes Move into Gene Therapy with Takeda… – Labiotech.eu

Saturday, April 11th, 2020

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

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

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

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

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

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

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

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

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

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

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Small molecules, biologics and now gene therapies: Germany’s Evotec adds another feather to its R&D cap – Endpoints News

Saturday, April 11th, 2020

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

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

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

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

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

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Symptoms in ALS Mouse Model Improve with CRISPR Base Editing – The Scientist

Saturday, April 11th, 2020

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

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

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

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

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

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

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

Colin Lim, University of Illinois

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

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

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

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

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

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

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

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

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

Saturday, April 11th, 2020

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

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

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

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Gene Therapy Market - Key Restraints

Impact of COVID-19 on Gene Therapy Market

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

Competitive Landscape

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

About the Report

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

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

SOURCE: FactMR

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