StemCell Therapy

TEL AVIV, Israel, Sept. 08, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) announced today the successful completion of a Type B pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding the Company's development plan for VB-601. VB-601 is the Companys lead anti-MOSPD2 antibody for immune-inflammatory indications, for which a briefing package for the pre-IND meeting was submitted in June. Based on the FDA's feedback, VBL plans to advance its IND-enabling activities for VB-601 as planned.

We are very pleased with the outcome of the pre-IND meeting with the FDA, said Dror Harats, M.D., CEO of VBL Therapeutics. VB-601 is a first-in-class product candidate that has a completely novel mechanism of action. It can block the ability of monocyte to migrate to inflammatory sites, no matter which molecules try to attract them in. Therefore, it is important that we have reached alignment with the FDA on the path forward for VB-601. With this green light, we plan to continue to advance VB-601, aiming to start a first-in-human study in the second half of 2021.

About VB-601VBL is advancing a drug development program that is exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases. VB-601 is a proprietary anti-MOSPD2 monoclonal antibody which has potential for treatment of multiple chronic inflammatory diseases.

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for cancer. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every two months. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers phase 1 trial as well as in three VBL-sponsored tumor-specific phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding VBLs programs, including VB-601, including their pre-clinical and clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. VBL Therapeutics undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:

Michael RiceLifeSci Advisorsmrice@lifesciadvisors.com(646) 597-6979

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VBL Therapeutics Announces Successful Completion of Pre-IND Meeting with FDA Regarding Proposed Clinical Development of VB-601 - BioSpace

Seeing a child debilitated by illness is never easy.

When doctors tell youthere is nothing they nor you can do to help ease yourbaby's suffering, well, parents who know that type of helplessness often find it hard to describe.

We were told to take my son home and love him, becausehe probably wouldnt live past his second birthday, said Pace father Todd Hamrick. But, were way past that birthday now.

Hamricks son, Alek, was diagnosed at six months old with spinal muscular atrophy and not expected to live long enough to toddle. ButAlek, now 3, has beaten the odds and outlived that initial, bleak prognosis.

His parents attribute much of his success to his doctor, Richard Finkel, who entered Alek into a clinical trial for what they believe has been a wonder drug for their little boy, Evrysdi, which was recently approved by the Food and Drug Administration.

Whats hard to even get around is that people even bothered to research it, Todd Hamrick said. Its just a small amount of the population that has SMA. Its not like researching a blood pressure medication.

Spinal muscular atrophy, or SMA,is a genetic disorder caused by a loss of nerve cells that effect human motor function.

Essentially, those afflicted by SMA are made weak. Their muscleswaste away. In many cases, eventually, a person loses their ability to walk, to eat andevento breathe, and they die.

Aleks mother, Iwona Hamrick, is a nurse at a local hospital and wellremembers the moment she heardher sons diagnosis.

It was unimaginable. His pregnancy was normal, she said. We did genetic testing and it was negative. Unfortunately, at that time, they were not screening for his disease. So, from a healthy baby to a dying baby, you know?

The parents felt they had to travel, in more ways than one, to find the places and help that they could for their Alek.

Todd Hamrick said that he and wife decided to move from Gulf Breeze to Pace after Aleks diagnosis after feeling ostracized by many of their former acquaintances. The parents felt like some people who they used to know were made uncomfortable by their son's illness.

The area is very in-the-dark when it comes to children with issues, Todd Hamrick said. If your kids are healthy and you'rehealthy, its a great area. But, it doesnt attract the greatest talent or best and biggest facilities or endowments.

So, Alek traveled with his family to meet his future doctor, Finkel, at the Nemours Children's Hospital in Orlando.

Alekwas first treated with a gene therapy drug, and he made some response with that, Finkel told the News Journal. But more recently, he started on a second drug, which seems to be having an enhanced effect, I must say.

Finkel, an expert in the field of pediatric neurologic disorders, left Florida in March for a position leading the new Center for Experimental Neurotherapeutics at St. Jude Childrens Research Hospital in Memphis, Tennessee. Buthe has continued to monitor Aleks progress via video.

Both of the drugs he has received, the gene therapy and this new drug, Evrysdi, are designed to increase a certain protein in Aleks body that is deficient because of his genetic disorder, he explained. But they do it in different ways. The potential advantage of his new drug, Evrysdi because its an oral drug; you take it by mouth it goes into the stomach and into the bloodstream, and from there, it goes to all the tissues of the body.

And, we think that there is an enhanced effect, becauseit gets into the muscle tissue, Finkel continued. "These are very, very, early daysin trying to make assessments, soI dont want to say that we can come to any kind of conclusions yet."

However the cutting-edge drug works doesnt matter to a mother, whos just glad that it isworking.

It gave us hope. Thats for sure, Iwona Hamrick said. Becausewe felt helpless.

Since Alek started his new treatment last November, his strength has increased tremendously.

His muscle tone got better, Iwona Hamrick said. He is much stronger in the upper body, so much so, he is pushing his little wheelchair.

Alek can now cruise around his Pace home in an extraordinarily lite-weight wheelchair designed by a Swedish inventor who alsohas a child with SMA.

Alek had a lot of trouble before starting this medication even pushing it, Iwona Hamrick said. Sonowhe is just rolling around the house. Also, he is barring more weight on his legs.

Recently, Alek has started to be able to walk in a pool a huge milestone for the toddler.

But some worries remain the same.

Every day, Alek must use a type of breathing machine.

Its a cough assist machine, Todd Hamrick explained. We use it two times a day, when hes healthy. Becausewhere we can just clear our throats when we cough, he doesnt have that strength no lung strength.

Alek attends physical therapy, aqua-therapy,hippotherapy, occupational therapy and speech therapy sessions every week to try and ensure he remains healthy and continues to properly develop.

My worries have changed a lot, Todd Hamrick said. I used to worry my child was going to die. Now, Im worrying about if other kids will bully him at school.

"But that'sa great worry to have," he continued. "Compared to how it use to be, getting picked on is a great thing to worry about.

Colin Warren-Hicks can be reached at colinwarrenhicks@pnj.com or 850-435-8680.

Read or Share this story: https://www.pnj.com/story/news/2020/09/08/pace-florida-toddler-spinal-muscular-atrophy-helped-drug-evrysdi/5681766002/

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Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal

NEW YORK and CLEVELAND, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company will participate in the following upcoming investor conferences.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona’s clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company’s portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona’s novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona’s fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Greg Gin VP, Investor Relations Abeona Therapeutics +1 (646) 813-4709 ggin@abeonatherapeutics.com

Media Contact: Scott Santiamo Director, Corporate Communications Abeona Therapeutics +1 (718) 344-5843 ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics to Participate in Upcoming Investor Conferences - Stockhouse

Roots Analysis has done a detailed study on Gene Therapy Market (3rd Edition), 2019-2030, covering key aspects of the industrys evolution and identifying potential future growth opportunities.

Key Market Insights

For more information, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

Table of Contents

4.2.1 Types of Viral Vectors

11.2 Analysis by Special Designations Awarded

11.3.1. Analysis by Target Indication

11.3.2. Analysis by Type of Vector Used

11.4.1. Analysis by Target Indication

11.4.2. Analysis by Type of Vector Used

11.5.1. Analysis by Target Indication

11.5.2. Analysis by Type of Vector Used

11.6.1. Analysis by Target Indication

11.6.2. Analysis by Type of Vector Used

11.7.1. Analysis by Target Indication

11.7.2. Analysis by Type of Vector Used

11.8.1. Analysis by Target Indication

11.8.2. Analysis by Type of Vector Used

11.9.1. Analysis by Target Indication

11.9.2. Analysis by Type of Vector Used

11.10.1. Analysis by Target Indication

11.10.2. Analysis by Type of Vector Used

11.11.1. Analysis by Target Indication

11.11.2. Analysis by Type of Vector Used

11.12.1. Analysis by Target Indication

11.12.2. Analysis by Type of Vector Used

11.13.1. Analysis by Target Indication

11.13.2. Analysis by Type of Vector Used

For more information, please click on the following link:

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About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

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The Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 - Scientect

[190+ Pages PDF Report] Facts & Factors (FnF) published a market research report onGene Therapy Market: By Size, Share, Growth Factors, Top Players, COVID-19 Analysis and Forecast, 2020-2026that includes a research report with TOC including a list of tables and figures in its research offerings.

Gene Therapy Market Report: Industry Insights, 2020-2026

This multi-client research study on theGene Therapy marketprovides in-depth research and analysis into Gene Therapy industry trends, market developments and technological insights. The report provides data and analysis of Gene Therapy penetration across application segments across countries and regions. The report presents a strategic analysis of the Gene Therapyy market through key drivers, challenges, opportunities, and growth contributors.

The global Gene Therapy market delivers value to customers through reliable market size for 2020 on the basis of demand and price analysis. The report presents near term and long term forecast of the addressable Gene Therapy market size to 2026.

The report presents an introduction to the Gene Therapy market in 2020, analyzing the COVID-19 impact both quantitatively and qualitatively.

Request an Exclusive FREE SAMPLE REPORT of Gene Therapy Market:https://www.fnfresearch.com/sample/gene-therapy-market-by-type-germ-line-gene

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The FREE PDF Sample Report Includes:

(Note: The sample of this report is updated with COVID-19 impact analysis before delivery)

Some of Following Top Market Players Profile Included in This Report:

2020 Gene Therapy Market: COVID-19 Impact Analysis

The global rise of COVID-19 has many businesses struggling and confused about what steps to take to minimize the economic impact. A simple look at the stock market will tell you that coronavirus has led to a volatile economy, but there are numerous other factors at play.

The long term COVID-19 impact on the business industry is largely still unknown; hence,Facts and Factors market researchanalysts have already covered the effects of COVID-19 on the business industry at a large level, as well as global and regional levels. Through our coverage below, get a better understanding of the business and economic implications coronavirus has on trends like remote working, consumer shopping behavior, global advertising spend, and essential industries like food, medical, travel, and transportation.

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Gene Therapy Market 2020: Research Scope & Coverage

The report covers Gene Therapy market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the markets historic and forecast market growth by geography. The market size analysis gives the market size covering both the historic growth of the market, the impact of the COVID 19 virus, and forecasting its recovery. The report also provides a comprehensive analysis of current & future trends and emerging avenues for the growth of this market along with this research also offers an insight into the competitive landscape in terms of new technological developments, untapped segments, and value chain analysis.

The report, Gene Therapy Market, provides up-to-date information about market size, share shifts, and potential risks, as well as in-depth knowledge of products and services, which can help in planning and in executing category management activities. It focuses on the cost-saving aspects of procurement and on providing insights that can lead to the optimization of category spend.

Get Free Customized Copy of Gene Therapy Market Report @https://www.fnfresearch.com/customization/gene-therapy-market-by-type-germ-line-gene

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Competitive Landscape

The market appears to be fragmented and with the presence of several. This Gene Therapy market research report will help clients identify new growth opportunities and design unique growth strategies by providing a comprehensive analysis of the markets competitive landscape and offering information on the products offered by companies.

Key Takeaways from this Gene Therapy Market Report

About Us:

Facts & Factorsis a leading market research company and offers customized research reports and consulting services. Facts & Factors aims at management consulting, industry chain research, and advanced research to assist our clients by providing planned revenue model for their business. Our report and services are used by prestigious academic institutions, start-ups, and companies globally to understand the international and regional business background.

Contact Us (Corporate Office):Facts & FactorsA 2108, Sargam,Nanded City,Sinhagad Road,Pune 411041, IndiaUSA: +1-347-989-3985Email:[emailprotected]Web:https://www.fnfresearch.com

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Know Top Players and Growing Trends in Gene Therapy Market 2020 Research Report and Industry Forecast till 2026 - Galus Australis

MarketsandResearch.biz has published the latest market research study on Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 by Company, Type and Application, Forecast to 2025 which investigates a few critical features of the market such as industry condition, division examination, market insights. The report studies the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market share, competition landscape, market share, growth rate, future trends, market drivers, opportunities and challenges, sales channels. The report has referenced down to earth ideas of the market in a straightforward and unassuming way in this report. The research contains the categorization of the market by top players/brands, region, type, and end-user. The report exhaustive essential investigation of current market trends, opportunities, challenges, and detailed competitive analysis of the industry players in the market.

The research report has comprehensively included numbers and figures with the help of graphical and pictorial representation which embodies more clarity on the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market. Then the report delivers key information about market players such as company overview, total revenue (financials), market potential, global presence, as well as market share, prices, production sites and facilities, products offered, and strategies adopted by them. Market status and outlook of global and major regions, from angles of players, countries, product types, and end industries have been analyzed.

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

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/83910

Key strategic manufacturers included in this report: BioReliance, Richter-Helm, UniQure, Cobra Biologics, MassBiologics, Oxford BioMedica, Lonza, MolMed, FinVector, FUJIFILM Diosynth Biotechnologies, Brammer Bio, bluebird bio, Aldevron, Spark Therapeutics, VGXI, Biovian, Eurogentec, Novasep, PlasmidFactory, Cell and Gene Therapy Catapult, Vigene Biosciences

Market Potential:

Key market vendors have been predicted to obtain the latest opportunities as there has been an increased emphasis on spending more on the work of research and development by many of the manufacturing companies. Also, many of the market contenders are forecasted to make a foray into the emerging economies to find new opportunities. The global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market has gone through rapid business transformation by good customer relationships, drastic and competitive growth, significant changes within the market, and technological advancement in this market.

Geographically, this report is segmented into several key countries, with market size, growth rate, import and export of in these countries from 2015 to 2020, which covering: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

The market can be segmented into product types as: AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors

The market can be segmented into applications as: Cancers, Inherited Disorders, Viral Infections, Others

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/83910/global-viral-vectors-non-viral-vectors-and-gene-therapy-manufacturing-market-2020-by-company-type-and-application-forecast-to-2025

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Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 | Scope of Current and Future Industry 2026 - Good Night, Good...

Notice: This Content doesnt contain all the Information of the Report please fill the form (via link) and get all interesting information just one click in PDF with the latest update with chart and Table of Content

The report offers a systematic presentation of the existing trends, growth opportunities, market dynamics that are expected to shape the growth of the Cell Therapy Manufacturing Market. The various research methods and tools were involved in the market analysis, to uncover crucial information about the market such as current & future trends, opportunities, business strategies and more, which in turn will aid the business decision-makers to make the right decision in future.

This Report Covers Leading Companies Associated in Worldwide Cell Therapy Manufacturing Market: Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

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The report begins with a brief introduction and market overview of the Cell Therapy Manufacturing industry followed by its market scope and size. Next, the report provides an overview of market segmentation such as type, application, and region. The drivers, limitations, and opportunities for the market are also listed along with current trends and policies in the industry.

The key players profiled in this report include:Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

Regions included:

o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

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Key Benefits:

o This study gives a detailed analysis of drivers and factors limiting the market expansion of Cell Therapy Manufacturing

o The micro-level analysis is conducted based on its product types, end-user applications, and geographies

o Porters five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players

o By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this Cell Therapy Manufacturing market

The research study can answer the following Key questions:

What will be the progress rate of the Cell Therapy Manufacturing Market for the conjecture period, 2020 2027?What are the prominent factors driving the Cell Therapy Manufacturing Market across different regions?Who are the major vendors dominating the Cell Therapy Manufacturing industry and what are their winning strategies?What will be the market scope for the estimated period?What are the major trends shaping the expansion of the industry in the coming years?What are the challenges faced by the Cell Therapy Manufacturing Market?

Table of Contents

Report Overview: It includes the Cell Therapy Manufacturing market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary: This section of the report gives information about Cell Therapy Manufacturing market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players: Here, key players of the Cell Therapy Manufacturing market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study: All of the regions and countries analyzed in the Cell Therapy Manufacturing market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

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Cell Therapy Manufacturing Market Growth, Demand, And Value Is Increasing In The Upcoming Years - The Daily Chronicle

Chicago, United States:The global Cell and Gene Therapy Market report offers a complete research study that includes accurate estimations of market growth rate and size for the forecast period 2020-2025. It offers a broad analysis of market competition, regional expansion, and market segmentation by type, application, and geography supported by exact market figures. The all-inclusive market research report also offers Porters Five Forces Analysis and profiles some of the leading players of the global Cell and Gene Therapy Market. It sheds light on changing market dynamics and discusses about different growth drivers, market challenges and restraints, and trends and opportunities in detail. Interested parties are provided with market recommendations and business advice to ensure success in the global Cell and Gene Therapy Market.

Top Key players cited in the report:Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell Science, Inc., Human Stem Cells Institute, Kite Pharma, Inc., Kolon TissueGene, Inc., Novartis AG, Orchard Therapeutics plc., Organogenesis Holdings Inc., Pfizer, Inc., RENOVA THERAPEUTICS, .

Get Free PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart): https://www.reporthive.com/request_sample/2369809

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

Cell and Gene Therapy Marketreports offers important insights which help the industry experts, product managers, CEOs, and business executives to draft their policies on various parameters including expansion, acquisition, and new product launch as well as analyzing and understanding the market trends.

Each segment of the global Cell and Gene Therapy market is extensively evaluated in the research study. The segmental analysis offered in the report pinpoints key opportunities available in the global Cell and Gene Therapy market through leading segments. The regional study of the global Cell and Gene Therapy market included in the report helps readers to gain a sound understanding of the development of different geographical markets in recent years and also going forth. We have provided a detailed study on the critical dynamics of the global Cell and Gene Therapy market, which include the market influence and market effect factors, drivers, challenges, restraints, trends, and prospects. The research study also includes other types of analysis such as qualitative and quantitative.

Global Cell and Gene Therapy Market: Competitive Rivalry

The chapter on company profiles studies the various companies operating in the global Cell and Gene Therapy market. It evaluates the financial outlooks of these companies, their research and development statuses, and their expansion strategies for the coming years. Analysts have also provided a detailed list of the strategic initiatives taken by the Cell and Gene Therapy market participants in the past few years to remain ahead of the competition.

Global Cell and Gene Therapy Market: Regional Segments

The chapter on regional segmentation details the regional aspects of the global Cell and Gene Therapy market. This chapter explains the regulatory framework that is likely to impact the overall market. It highlights the political scenario in the market and the anticipates its influence on the global Cell and Gene Therapy market.

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Report Highlights

Table of Contents

Report Overview:It includes six chapters, viz. research scope, major manufacturers covered, market segments by type, Cell and Gene Therapy market segments by application, study objectives, and years considered.

Global Growth Trends:There are three chapters included in this section, i.e. industry trends, the growth rate of key producers, and production analysis.

Cell and Gene Therapy Market Share by Manufacturer:Here, production, revenue, and price analysis by the manufacturer are included along with other chapters such as expansion plans and merger and acquisition, products offered by key manufacturers, and areas served and headquarters distribution.

Market Size by Type:It includes analysis of price, production value market share, and production market share by type.

Market Size by Application:This section includes Cell and Gene Therapy market consumption analysis by application.

Profiles of Manufacturers:Here, leading players of the global Cell and Gene Therapy market are studied based on sales area, key products, gross margin, revenue, price, and production.

Cell and Gene Therapy Market Value Chain and Sales Channel Analysis:It includes customer, distributor, Cell and Gene Therapy market value chain, and sales channel analysis.

Market Forecast Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

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COVID-19 Impact on Cell and Gene Therapy Market Research, Size, Growth And Trends 2020 to 2025: Keyplayer-Amgen Inc., bluebird bio, Inc. - Scientect

AVROBIO, a US-based company, developing lentiviral-based gene therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Companys investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease.

Get a complete list of the presentations,here.

What is AVR-RD-02: AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. It is an ex vivo lentiviral-based investigational gene therapy. The stem cells are genetically modified to express functional glucocerebrosidase (GCase), which reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease.

How advanced is AVR-RD-02 development program: The Company is actively recruiting in Canada for its Phase 1/2 clinical trial of AVR-RD-02, which seeks to evaluate the safety and efficacy of the therapy in patients with Type 1 Gaucher disease.

What is an orphan drug designation: Orphan-drug designation provides special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDAs implementing regulations at 21 CFR Part 316. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

What is the gene therapy pipeline like for the treatment of Gaucher disease: There are only a few gene therapy candidates that are being developed for Gaucher disease.

For further information, check out the report here

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Gene Therapy Pipeline Update: AVROBIO Receives Orphan-Drug Designation | Roots Analysis - The News Brok

The report covers the forecast and analysis of the Cell and Gene Therapy Consumables market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the Cell and Gene Therapy Consumables market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the Cell and Gene Therapy Consumables market on a global level.

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In order to give the users of this report a comprehensive view of the Cell and Gene Therapy Consumables market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service & product launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the Cell and Gene Therapy Consumables market by segmenting the market based on product type, application/therapeutics, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

A rise in the awareness about the gene & cell therapies will propel the market growth during the period from 2019 to 2027. Nevertheless, conducting randomized control tests will inhibit the expansion of the market during the forecast timeline. However, the growing trend for treating neurodegenerative ailments through the use of gene treatment will proliferate the market growth over the forecast period.

The expansion of the market during the forecast timespan is owing to the high frequency of chronic ailments including cancer and heart disorders. Apart from this, inflation in the occurrence of these disorders produces lucrative demand for enhanced therapies and this will culminate in the market demand over the forecast timespan.

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Based on the product type, the market is sectored into Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media. Application/ Therapeutics- wise, the market for cell and gene therapy consumables are classified into Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology.

Some of the key players in the market include Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife.

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Cell and Gene Therapy Consumables Market 2020-2027/ Size, Segments and Supply Demand Forecast - StartupNG

The Global Gene Therapy Market report focuses on market size, status, and forecast 2020-2024, along with this, the report also focuses on market opportunities and threats, tactical decision-making, and evaluating the market. The Gene Therapy market report delivers data and information on changing investment structure, technological advancements, market tendencies and developments, capacities, and detailed information about the key players of the global market. In addition to this, the report also involves the development of the Gene Therapy market in the major regions across the world.

Cutting-edge released the research study on Global Gene Therapy Market, which deals a exhaustive overview of the factors influencing the global business scope. Gene Therapy Market research report shows the latest market insights, current situation analysis with upcoming trends, and breakdown of the products and services. The Gene Therapy Industry Report delivers key statistics on the market status, size, share growth factors of the Gene Therapy .

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Top Leading players of Gene Therapy Market Covered in the Report:

Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.Advantagene

The report has enclosed key geographic regions such as Europe, Japan, United States, India, Southeast Asia and Europe. As far as the sub-regions, North America, Canada, Medico, Australia, Asia-Pacific, India, South Korea, China, Singapore, Indonesia, Japan, Rest of Asia-Pacific, Germany, United Kingdom, France, Spain, Italy, Rest of Europe, Russia, Central & South America, Middle East & Africa are included.

Key Market Segmentation of Gene Therapy :

On the basis of types, the Gene Therapy Market from 2020 to 2024 is primarily split into:

Ex vivoIn Vivo

On the basis of applications, the Gene Therapy Market from 2020 to 2024 covers:

CancerMonogenicInfectious diseaseCardiovascular diseaseOther

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The Gene Therapy Market Research Report furthermore delivers a local examination of the market with a high focus on showcase development, development rate, and development potential. The research report calculates marketplace length estimate to analyze investment potentials and growth.

In this study, the years considered to estimation the market size of the Gene Therapy Industry Market: History Year: 2014-2018Base Year: 2018Estimated Year: 2019Forecast Year 2019 to 2024

The Gene Therapy market report provides answers to the following key questions:

Major Points Covered in Table of Contents:

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Global Gene Therapy Market 2020-2024 Expected To Witness The Highest Growth During Forecast period - Scientect

The global Gene Therapies for Cancer Treatment market study covers the projection size of the market both in terms of value (Mn/Bn US$) and volume (x units). The report estimates the lookup of different local distributors in the overall market and provides the market size of the Gene Therapies for Cancer Treatment market using both bottom-up and top-down approaches. To investigate the key players and their market contribution, primary and secondary research has been comprehensively performed. In addition, all the figures, subdivisions, and shares have been collected with the help of trustworthy sources.

In the Gene Therapies for Cancer Treatment market research study, 2018 is considered as the base year, and 2019-2029 is considered as the forecast period to predict the market size. The report identifies each Gene Therapies for Cancer Treatment market player on the basis of market share, production portfolio, and growth rate. In addition, the research study analyzes the strengths, weaknesses, opportunities and threats of the players.

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Global Gene Therapies for Cancer Treatment market report on the basis of market players

The following players are covered in this report:Takara BioTocagenVBL TherapeuticsCold GenesysGenprexMomotaro-GeneMultiVirSynerGene TherapeuticsZiopharm OncologyAnchiano TherapeuticsCelgeneCelsionBluebird BioGene Therapies for Cancer Treatment Breakdown Data by TypeSomatic Cell Gene Therapy (SCGT)Germline Gene Therapy (GGT)Gene Therapies for Cancer Treatment Breakdown Data by ApplicationCancer Research CentersDiagnostic LaboratoriesCancer HospitalsOthers

The report provides market share, consumption pattern, and influencing factors of each region. Prominent countries driving the regional growth are also covered in the report.

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The Gene Therapies for Cancer Treatment market report answers the following questions:

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Gene Therapies for Cancer Treatment Market Projected to Witness Vigorous Expansion by 2019-2025 - Scientect

Adenovirus vectors have become a popular delivery platform for gene therapy and vaccines. However, many people have preexisting immunity against adeno-associated viruses (AAVs), which could significantly reduce vector uptake and therefore render the therapeutic cargo they carry ineffective. Even in individuals that lack preexisting immunity, the first inoculation of a vector could induce immune responses that can thwart repeat administration.

To overcome that obstacle, scientists at the University of Pittsburgh created a CRISPR-based system that they say could effectively prevent immunity to the viral vector. The CRISPR tool briefly suppressedgenes that are related to anti-adenovirus antibody production and helped achieve better gene therapy uptake in mice. The results werepublished in Nature Cell Biology.

Samira Kiani, the studys co-senior author, has co-founded SafeGen Therapeutics with the goal to bring this platform to the clinic to enable safer gene therapy.

Accelerate Clinical Operations Across Sponsors, CROs, and Partners

The most advanced life sciences organizations know that digital innovation and multi-platform integrations are essential for enabling product development. New platforms are providing the life sciences industry with an opportunity to improve the efficiency of clinical trials and reduce costs while remaining compliant and reducing risk.

Many clinical trials fail because of the immune response against AAV gene therapy, Kiani said in a statement. And then you cant re-administer the shot because people have developed immunity.

One recent case in point is CanSino Biologics adenovirus type 5 (Ad5)-vectored COVID-19 vaccine. In the shots phase 2 trial, about half of the participants had high levels of preexisting Ad5 neutralizing antibody. These individuals were found to produce significantly lower immune responses to the vaccine than did those with low preexisting anti-Ad5 immunity.

Kiani and colleagues set out to modify the gene expression that's associated with an immune response against AAV, but only temporarilyjust long enough for the therapeutic content to do its jobbecause they didnt want to tamper with normal immune functions.

RELATED:Hopkins team invents non-viral system for getting gene therapy into cells

CRISPR, which can edit out a defective gene or add in a functional one, is already being explored as a gene therapy strategy. So the University of Pittsburgh researchers figured they could hit two birds with one stone by utilizing CRISPR to also control the immune response.

They treated mice with the CRISPR-based system and then re-exposed them to AAV, and the animals didnt make more antibodies against the virus. The treated mice were more receptive to subsequent AAV-delivered gene therapy than were the control animals, the team reported.

Gene therapies hold promise as potential cures to many hard-to-treat diseases that would otherwise require chronic treatment. So scientists have been trying to perfect their transport vehicles for better safety and efficacy.

A team led by scientists at the San Raffaele Telethon Institute for Gene Therapy in Italy, for example,attached the CD47 protein to the surface of lentiviral vectors so they could escape detection and destruction by the immune system. And researchersfrom Johns Hopkins University developed a nonviral delivery system that uses a polymer nano-container, whichreleases the therapy once inside the cell.

Now, Kianihopes to validate the CRISPR-based technology at SafeGen. In addition to its potential as a gene therapy delivery platform, the researchers also found that their method can prevent or treat sepsis in mice, suggesting it might also be useful for treating inflammatory conditions.

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Using CRISPR to improve viral vectors for gene therapy - FierceBiotech

Gene therapy generally relies on viruses, such as adeno-associated virus (AAV), to deliver genes into a cell. In case of CRISPR-based gene therapies, molecular scissors can then snip out a defective gene, add in a missing sequence or enact a temporary change in its expression, but the bodys immune response to AAV can thwart the whole endeavor.

To overcome that obstacle, researchers at the University of Pittsburgh created a system that uses CRISPR in a different way. Their system briefly suppresses genes that are related to AAV antibody production so the virus can deliver its cargo unimpeded. These results published today inNature Cell Biology.Many clinical trials fail because of the immune response against AAV gene therapy, said study co-senior author Samira Kiani, associate professor of pathology in Pitt'sSchool of Medicineand member of thePittsburgh Liver Research Center(PLRC) andMcGowan Institute for Regenerative Medicine. And then you cant readminister the shot because people have developed immunity.

So Kiani and her long-time collaborator Mo Ebrahimkhani, associate professor of pathology at Pitt, member of PLRC and the McGowan Institute, set out to modify gene expression related to the bodys immune response to AAV. But this gene is important for normal immune function, so the researchers didnt want to shut it down forever, just tamp it down momentarily.

Since CRISPR is such a convenient system for editing the genome, the pair figured they would put it to use for altering the master switches that orchestrate genes involved in immune response.

Were hitting two birds with one stone, said Ebrahimkhani. You can use CRISPR to do your gene therapy, and you can also use CRISPR to control the immune response.

When the researchers treated mice with their CRISPR-controlled immune suppression system and then exposed them to AAV again, the animals didnt make more antibodies against the virus. These animals were more receptive to subsequent AAV-delivered gene therapy compared to controls.

Beyond gene therapy, the study also shows that CRISPR-based immune suppression can prevent or treat sepsis in mice, highlighting the potential for this tool to be broadly useful for a range of inflammatory conditions, including cytokine storm andacute respiratory distress syndrome, both of which can crop up with COVID-19, though more studies are needed to engineer safety features.

The main goal of this study was to develop CRISPR-based tools for inflammatory conditions, said study lead author Farzaneh Moghadam, a PhDstudent in Kianis lab. But when we looked at bone marrow samples, we saw that the group treated with our tool showed a lower immune response to AAV compared to the control group. That was very interesting, so we started exploring how this tool contributes to antibody formation against AAV and could potentially address safety and efficacy concerns with gene therapy trials.

Kiani co-founded SafeGen Therapeutics with the goal of bringing this technology to the clinic.

This study was supported by National Institute of Biomedical Imaging and Bioengineering, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, and a DARPA Young Faculty Award.

Reference: Moghadam F, LeGraw R, Velazquez JJ, et al. Synthetic immunomodulation with a CRISPR super-repressor in vivo. Nature Cell Biology. 2020;22(9):1143-1154. doi:10.1038/s41556-020-0563-3.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Editing the Immune Response To Aid the Delivery of Gene Therapy Cargo - Technology Networks

TipRanks

As fears of a tech bubble and stretched valuations become the talk of the town, investors are turning to Wall Street titans for guidance, namely Ken Griffin. Founding hedge fund Citadel in 1990, the firm now boasts over $35 billion worth of assets under management.As a 19-year-old sophomore at Harvard University, Griffin began trading from his dorm room with a fax machine, computer and phone. Now, the CEO of Citadel, whose net worth stands at $15.5 billion, is known as one of the Wall Street greats. Looking at the funds performance during the COVID crisis, its even more clear why Griffin has legendary status.Unlike the average hedge fund, which had a negative return of between 3-4% in the first half of 2020, Citadels flagship Wellington fund saw its returns land between 13-14% for the same period.Bearing this in mind, we wanted to take a closer look at three stocks Citadel snapped up recently. Using TipRanks database, we found out that each ticker has earned a Strong Buy consensus rating from the analyst community. Not to mention all three of them boast massive upside potential.AVEO Pharmaceuticals (AVEO)Hoping to provide better outcomes for patients, AVEO Pharmaceuticals advances targeted medicines for oncology and other unmet medical needs. Following an important regulatory milestone, its no wonder all eyes are on this healthcare name.Griffin is among those singing AVEOs praises. Increasing its holding by a whopping 2,357%, Citadel bought up 383,720 shares in Q2. With the total position now landing at 400,003 shares, it is valued at $1,824,013.H.C. Wainwright analyst Swayampakula Ramakanth reminds investors that on June 1, the FDA accepted the NDA for tivozanib, the company's lead candidate, for review, based on the fact that the TIVO-3 study reported positive final overall survival (OS) data. In the study, AVEOs therapy was compared to sorafenib, marketed as Nexavar by Bayer, for the treatment of advanced renal cell carcinoma (RCC) in the third and fourth-line settings.Looking more closely at the data, which was presented at the ASCO 2020 virtual meeting, the final OS analysis resulted in an overall hazard ratio (HR) of 0.97, which favored tivozanib. Ramakanth was encouraged by the OS results as they suggest tivozanib at least has a similar overall relative risk of deaths compared to sorafenib.Considering that TIVO-3 study met both the primary endpoint of progression free survival (PFS) and the secondary endpoint of overall response rate (ORR), with comparable OS to the active comparator, we believe tivozanib would likely get a green light for the U.S. approval, which could be a major catalyst in the next 12 months, Ramakanth opined.Adding to the good news, the dose escalation for the Phase 1b/2 DEDUCTIVE study, evaluating tivozanib in combination with durvalumab, a monoclonal antibody against PD-L1 marketed as Imfinzi by AstraZeneca in hepatocellular carcinoma (HCC), has been wrapped up, with it progressing to Phase 2. As the CDC estimates about 33,000 patients suffer from liver cancer every year in the U.S., Ramakanth sees an additional opportunity.To this end, Ramakanth rates AVEO a Buy rating along with a $12 price target. Should his thesis play out, a potential twelve-month gain of 163% could be in the cards. (To watch Ramakanths track record, click here)Other analysts dont beg to differ. 3 Buy ratings and no Holds or Sells have been assigned in the last three months. So, the word on the Street is that AVEO is a Strong Buy. The $15 average price target is more aggressive than Ramakanths and implies 229% upside potential. (See AVEO stock analysis on TipRanks)IDEAYA Biosciences (IDYA)Next up we have IDEAYA Biosciences, an oncology-focused precision medicine company that develops targeted therapeutics by using molecular diagnostics. Based on the strength of its technology, this name has scored several fans.Reflecting a new position for Griffin's Citadel, the fund pulled the trigger on 248,005 shares in Q2. As for the value of this holding, it comes in at $2,881,818. Writing for Northland Capital, analyst Tim Chiang believes shares are undervalued based on the future potential of its precision medicine oncology pipeline, which targets specific biomarkers. Expounding on this, he stated, IDEAYA is applying its capabilities across multiple classes of precision medicine, including direct targeting of oncogenic pathways and synthetic lethality which represents an emerging class of precision medicine targets.Part of what makes IDYA a stand-out, in Chiangs opinion, is the fact that its preclinical programs use its synthetic lethality (SL) platform, which targets tumors with MTAP gene deletion and homologous recombination deficiency (HRD) including BRCA mutations.We believe the longer-term upside potential with IDYA shares is significant given the potential utility of SL. The first clinically validated SL gene pair was PARP-BRCA1/2, and based on the efficacy of PARP inhibitors, the SL approach to treating cancer has achieved substantial commercial validation, the analyst explained.To back this up, Chiang points out that several PARP inhibitors have already been approved for the treatment of tumors with BRCA and other DNA damage repair alterations, including ovarian, breast and pancreatic cancers. These inhibitors include AstraZeneca's olaparib, GlaxoSmithKline's niraparib, Pfizer's talazoparib and Clovis rucaparib. He added, We estimate these four drugs generated over $1.6 billion in worldwide sales in 2019 and are expected to reach over $6 billion in sales by 2024.It should be noted that multiple IND filings are set to come within the next 4-12 months, with IDYAs lead SL candidate, IDE397, which was designed to inhibit MTAP and MAT2A and thus cause the death of cancerous tumor cells, entering the clinic in 2021.It should come as no surprise, then, that Chiang joined the bulls. To start off his IDYA coverage, he puts an Outperform rating and $28 price target on the stock. This target implies a possible twelve-month rise of 141% could be on the horizon. (To watch Chiangs track record, click here)Similarly, the rest of the Street is getting onboard. 5 Buy ratings assigned in the last three months add up to a Strong Buy analyst consensus. In addition, the $25.20 average price target puts the potential twelve-month gain at 116%. (See IDYA stock analysis on TipRanks)Ocular Therapeutix (OCUL)Using its patented bioresorbable hydrogel-based formulation technology, Ocular Therapeutix develops cutting-edge therapies for diseases and conditions of the eye. The progress of its clinical programs has caught Wall Streets attention, with some arguing that now is the time to get in on the action.Griffin and Citadel didnt want to miss out on an opportunity. Snapping up 161,032 shares during Q2, the hedge fund gave the holding a 272% boost. The total position is now comprised of 220,269 shares and is valued at $1,718,098.Representing Raymond James, 5-star analyst Dane Leone cites the potential to address the unmet needs in the dry eye disease indication as a key component of his bullish thesis. The company boasts two assets targeting the condition, OTX-CSI (chronic) and OTX-DED (acute). OTX-CSI incorporates the FDA-approved immunomodulator cyclosporine as the active drug in the intracanalicular insert, which is released for an estimated three months to increase tear production.When it comes to OTX-DED, Leone argues OCUL was clever in pursuing the development of OTX-DED (a low dose form of DEXTENZA, a corticosteroid intracanalicular insert placed in the punctum, a natural opening in the eye lid, and into the canaliculus to deliver dexamethasone to the ocular surface for up to 30 days without preservatives) for the treatment of episodic dry eye, as the prior safety data from the DEXTENZA approval enabled management to file a Phase 2-enabling IND by YE20.Speaking to the possible opportunity here, both products could provide revenue generation for physicians in the treatment of dry eyes using procedure CPT code 0356T, which could provide incentive for rapid adoption in the dry eye space that is currently valued at $5.1 billion, in Leones opinion.As for the clinical and regulatory pathway, the Phase 2 trial for OTX-DED is set to kick off in 2H21, slightly after OTX-CSI, which will see a Phase 2 clinical trial evaluating two different formulations initiated by 4Q20. Based on the short treatment duration of OTX-DED, management thinks it will still be brought to market first.To this end, Leone remains optimistic about the companys long-term growth narrative. As a result, he rates OCUL a Strong Buy along with a $15 price target. This figure implies shares could rise 89% in the year ahead. (To watch Leones track record, click here) Turning now to the rest of the Street, other analysts are on the same page. With 100% Street support, or 3 Buy ratings to be exact, the consensus is unanimous: OCUL is a Strong Buy. The $13.50 average price target brings the upside potential to 70%. (See OCUL stock analysis on TipRanks)To find good ideas for stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Gene Therapy Market is Forecast to Reach US$ 6.42 billion in 2024, Witnessing Growth at a CAGR of 19.29% Over 2020-2024 - Yahoo Finance

Gyros Protein Technologies AB has announced the introduction of Gyrolab AAVX Titer Kit for adeno-associated virus (AAV) vector titer determination in AAV vector manufacturing. The new Gyrolab AAVX Titer Kit for physical titer determination, one of the critical quality attributes (CQAs) of the FDA process validation guidelines, has a broad applicability across numerous AAV serotypes and adds to the Companys wide range of ready-to-use kits that are already used by scientists and bioengineers in the rapidly growing cell and gene therapy market, where AAV vectors are commonly used.

Gyrolab AAVX Titer Kit supports scientists in the development and production of cell and gene therapies to maximize performance and productivity, and reduces the time needed to produce patient treatments. With compressed development timelines for these therapies, the new kit generates results 4 times faster and requires 10 times less sample compared to traditional ELISA methods, accelerating AAV vector workflows and enabling high quality data to be produced from small sample volumes. Featuring an analytical range of >3 logs, Gyrolab AAVX Titer Kit also minimizes repeat testing compared to ELISA.

Gyrolab AAVX Titer Kit is based on a highly selective AAVX affinity ligand developed with the Thermo Scientific CaptureSelect technology by Thermo Fisher Scientific. These ligands are also the basis of POROS CaptureSelect AAVX Affinity Resin, which is frequently used to purify AAV viral vectors. The AAVX ligand binds with high affinity and selectivity to native and recombinant AAV particles of various serotypes (AAV1 to AAV8, AAVrh10).

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Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks

The development prediction report titled Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 by Company, Regions, Type and Application, Forecast to 2025 focuses on thoughtful insights and facts relating to the market. The report investigates the most recent market patterns such as market development openings, size, share, and drivers. The report throws light on the markets historical data, key vendors, region-wise market, and projections for 2020 to 2025 time-period. Report authors have categorized global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market segments, regions, and product types and distribution channels to provide market analysis and information. All the relevant points of interest market product type, producing price, scope, applications are covered in the report. In the later section, market dynamics are covered including market growth factors, limitations, market opportunities, and challenges are mentioned.

NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

Competitive Rivalry:

The report offers a comprehensive study of prime players operating in this global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market by highlighting their product description, business outline, and business strategy. The report conjointly encompasses the amount of production, future demand, and also the health of the organization. Later, the report highlights all the recent developments, product launches, joint ventures, merges, and accusations by the top players. The leading players are also covered with product description, business outline, and production, company profile, product portfolio, product/service price, capacity, sales, and cost.

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The report analyses every sub-segment regarding the individual growth trends, contribution to the total market, and the upcoming forecasts. For the forecast period, the report offers estimates of global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market value and volume. The report discusses key drivers, market challenges, cost structure, barriers, technology, regions, and applications. It spotlights on effective manufacturing methods, production volume, manufacturing facilities, capacities, pricing analysis, product description.

Top key players involved in the industry are: BioReliance, Richter-Helm, UniQure, Cobra Biologics, MassBiologics, Oxford BioMedica, Lonza, MolMed, FinVector, FUJIFILM Diosynth Biotechnologies, Brammer Bio, bluebird bio, Aldevron, Spark Therapeutics, VGXI, Biovian, Eurogentec, Novasep, PlasmidFactory, Cell and Gene Therapy Catapult, Vigene Biosciences

Segmentation by type and analysis of the market: AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors

Segmentation by application and analysis of the market: Cancers, Inherited Disorders, Viral Infections, Others

The report offers an exhaustive geographical analysis of the global market, covering important regions such as: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

Moreover, the report analyzes the worlds main regions contributing to the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market development. These regions are assessed the product price, profit, capacity, production, supply, demand, and market growth rate and forecast, etc. Additionally, manufacturing processes, cost structures, import/export consumption, supply and demand figures, cost, price, revenue, and gross margins are analyzed.

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Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 Analysis, Types, Applications, Forecast and COVID-19 Impact...

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

Originally posted here:
Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - Scientect

LONDON Gyroscope Therapeutics Ltd. is poised to move the field of ocular gene therapy on from the treatment of inherited rare diseases to address more common eye conditions, after receiving FDA 510(k) clearance for its Orbit SDS subretinal delivery device.

Orbit SDS uses a cannula to deliver a therapeutic payload to the subretinal space, which it accesses via a suprachoroidal approach route. A microneedle inside the cannula enables targeted dose delivery.

That eliminates the need for a vitrectomy, and is both faster, easier and safer than the surgical procedure, removing concerns about gene therapy vectors leaking from the subretinal space.

Although vitrectomy has been used to deliver several gene therapies, it is not practical for treating hundreds of thousands of patients with dry age-related macular degeneration (AMD) and other common eye diseases. FDA 510(k) clearance of Orbit SDS now opens the way to scale up administration of ocular gene therapies to much larger indications.

The FDA clearance creates the potential to speed up development and widen patient access. In addition to a replacing a defective gene with a functional one, it will be possible to expand into the routine delivery of therapeutic proteins.

The company says it will use the device in its own clinical trials and make it available to other companies developing cell and gene therapies for eye diseases.

Charlotte Arnold, vie president of corporate affairs at Stevenage, U.K.-based Gyroscope told BioWorld the company now plans to integrate Orbit SDS into its GT005 program in dry AMD. We anticipate starting to generate data delivering GT005 using the Orbit SDS in a planned future cohort in the ongoing phase I/II Focus trial, she said.

Unlike the wet form of AMD that can be treated with anti-VEGF antibodies to prevent neovascularization, there is no therapy approved for dry AMD, which is both a cause of significant visual impairment and a precursor to wet AMD.

GT005 uses an adeno-associated virus to deliver the gene encoding an endogenous anti-inflammatory factor, to restore the balance of a complement system known to be hyperactivated in AMD. The aim is that a one-off treatment will prevent further degeneration.

The FDA nod is an important component of developing gene therapies to help preserve sight, said Khurem Farooq, CEO of Gyroscope. The Orbit [device] is exquisitely designed to target the subretinal space, with the aim of providing precise and consistent dosing, Farooq said.

The clearance for Orbit SDS rests on a phase IIb study of palucorcel (CNTO-2476), in the treatment of geographic atrophy of age-related AMD, an advanced stage of AMD, characterized by progressive loss of retinal pigment epithelium. That leads to loss of photoreceptors, resulting in visual disturbance and difficulty adjusting to low light conditions. Central vision is lost progressively.

The Orbit device was used in the phase I/II trial after initial studies in which palucorel was administered by retinotomy showed there was a risk of retinal detachment. A further study used subretinal delivery, but the technique was judged too technically difficult to take forward.

That led on to the development of Orbit SDS, which was purpose designed for subretinal delivery.

In the 21-patient phase I/II palucorel study the investigators reported that administration to the subretinal site was achieved successfully in most patients and [was] not associated with any moderate or severe, or serious ocular adverse events. The investigators were unable to deliver cells in 3 of 21 participants. Most adverse events were mild and resolved within one month.

Vitrectomy can be associated with an increase in intraocular pressure and accelerated formation of cataracts. No such effects were seen in the Orbit SDS study.

Palucorel is a cell therapy derived from human umbilical tissue that expresses anti-inflammatory factors including interleukin-6, brain-derived growth factor, fibroblast growth factors, and anti-angiogenic thrombospondins. While it showed positive effects in two earlier trials, there were no significant improvements in vision in the Orbit SDS-administered trial.

However, the investigators say, This first in human trial of a suprachoroidal approach for delivery of subretinal liquids was successful from a surgical perspective and clearly demonstrates that subretinal delivery via the suprachoroidal space is feasible.

Gyroscope acquired the Orbit SDA device when it merged with its developer, Orbit Biomedical of Philadelphia in April 2019, to become a fully integrated retinal gene therapy company, with clinical, manufacturing and delivery capabilities.

Orbit Biomedical, now renamed Gyroscope USA Inc., is an ISO 13485 medical device company certified to design, manufacture and distribute ophthalmic surgical instruments.

Following on from the FDA clearance, Arnold said Gyroscope is pursuing a CE mark in Europe for Orbit SDS as well. We also plan to enter into licensing and collaboration arrangements for use of the Orbit SDS by other companies for delivery of gene and cell therapies being developed to treat eye diseases, she said.

Read the original:
Gyroscope Therapeutics FDA clearance puts subretinal delivery device in Orbit - BioWorld Online

Non-Hodgkin lymphoma (NHL) is among the most common cancers in the United States. In 2020, an estimated 77,240 Americans will be diagnosed with the disease and about 20,000 people will die from it.1 Seventy-two percent of patients live for at least 5 years after diagnosis.2 However, the success of treatment varies widely across the many subtypes of NHL. Patients diagnosed with diffuse large B-cell lymphoma (DLBCL), the most common subtype, have a 5-year survival rate of 63% for all disease stages combined. By contrast, follicular lymphoma (FL) has a 5-year survival rate of 88% for all stages combined. Patients with mantle cell lymphoma, one of the more difficult-to-treat subtypes, have an average survival time of less than 5 years.3

But the treatment landscape is transforming across the entire spectrum of NHL. Novel immunotherapies and small molecule inhibitors are offering both previously treated and untreated patients entirely new options and new combinations. At the American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program, researchers led by Jeremy Abramson, of Massachusetts General Hospital in Boston, provided a compelling overview of these new and upcoming treatments.4 Their overview, published in the ASCO Educational Book, offered a detailed and vital look at the present and future of NHL treatment.

CAR T-cell immunotherapy has been hailed as a major game-changer for some blood cancers.5 For aggressive B-cell lymphomas, such as DLBCL, anti-CD19 chimeric antigen receptor T cells (CAR-T) are a new option for patients who have relapsed following chemoimmunotherapy or autologous stem cell transplant (ASCT). For this patient group, which has a median overall survival of about 4 months, anti-CD19 CAR-T agents have elicited durable remissions in about 40% of patients.5-7 Clinical trial data has led to the approval of axicabtagene, ciloleucel, and tisagenlecleucel by both the US Food and Drug Administration and the European Medicine Agency. CAR T-cells offer curative intent therapy to patients with relapsed DLBCL who are not eligible for stem cell transplant, Dr Abramson told Cancer Therapy Advisor. Previously these patients only had palliative options available.

Some clinicians envision CAR-T as a potential first-line therapy for patients with DLBCL who relapsed after first-line chemoimmunotherapy. These patients, noted Anton Hagenbeek, MD, PhD, professor of Hematology at Amsterdam University Medical Centers, who was not involved with the review paper, represent one of the highest unmet needs in the treatment of lymphoma to date. And Helen Heslop, MD, who directs the Center for Cell and Gene Therapy at Baylor College of Medicine in Houston, Texas, noted that trials for first-line CAR-T therapy are already underway in acute lymphoblastic leukemia. Not everyone sees this potential. First-line treatment in NHL is generally very effective and much less expensive than CAR T-cells, explained Edward Copelan, MD, who chairs the Department of Hematologic Oncology and Blood Disorders at Carolinas Healthcare System in Charlotte, North Carolina, and who was not an author of the review. Though Dr Copelan emphasized that patients at extremely high risk of relapse following standard immunochemotherapy may respond well to CAR-T therapy.

Dr Abramson and co-authors highlighted a difficult conundrum with regard to DLBCL. Although the addition of rituximab to cyclophosphamide, doxorubicin, hydrochloride, and vincristine sulfate (CHOP) chemotherapy is curative for most patients, the number of patients who may be cured after relapse has declined. Thus improvements in second-line therapy are desperately needed. Several clinical trials are currently evaluating anti CD19 CAR-T for primary refractory or early relapsed aggressive B-cell lymphoma compared with traditional salvage therapy or ASCT. The same approach is being studied for relapsed DLBCL patients who are not transplant candidates.

Whether CAR-T therapy will find a place on the first line of care is another current question. The cost and logistics of CAR-T therapy make it unlikely that this approach will replace R-CHOP as the standard initial treatment, Dr Abramson and colleagues noted. I do not think CAR T-cells will replace frontline chemotherapy, Dr Abramson said. However, the authors note that for patients who do not respond well to initial treatment, such an approach may make sense a possibility that is now being examined in the clinical trial setting. Dr Hagenbeek is more certain about the first-line role for CAR-T therapy, based on the dismal prognosis of DLBCL that is refractory to R-CHOP.

On the subject of CAR-T therapy as first-line therapy, the authors pay special attention to so-called double-hit lymphomas (DHLs), also known as double expressor lymphomas. According to a multicenter study published in 2017, R-CHOP is curative for more patients than conventional wisdom has held.9 Clinical trials are currently investigating the use of anti-CD19 CAR-T early for patients who consistently test positive for disease on PET scans during initial therapy, though Dr Abramson and colleagues call for caution with this approach because the prognostic value of PET/CT scans is controversial. Dr Hagenbeek is optimistic about the potential for CAR-T therapy to improve the prognosis for patients with double- and even triple-hit lymphomas, if applied in the first complete, PET-negative metabolic remission. Because these patients have a relatively small tumor load, this approach could, said Dr. Hagenbeek, completely eradicate minimal residual disease. Its the double-hit patients who Dr Copelan sees as the likeliest candidates for first-line CAR T-cell treatment.

Read more here:
Targeted Therapy for Non-Hodgkin Lymphoma: Current Progress and Future Plans - Cancer Therapy Advisor