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The Cell and Gene Therapy Market to Reach Revenues of Over $6.6 billion by 2024 – Market Research by Arizton – PRNewswire

CHICAGO, Nov. 13, 2019 /PRNewswire/ -- According to Arizton's recent research report, Cell and Gene Therapy Market - Global Outlook and Forecast 2019-2024 is expected to grow at a CAGR of more than 24% during the forecast period.

Key Highlights Offered in the Report:

Key Offerings:

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Cell and Gene Therapy Market Segmentation

Market Segmentation by Products

Market Segmentation by Distribution Channel Type

Market Segmentation by End-users

Cell and Gene Therapy Market Dynamics

CAR T-cell therapy has gained significant traction in recent years. It is the single most rapidly growing type of product in the market that generates revenue at a phenomenal rate. At present, it is the fastest advancing technology in cancer treatment and has the capability to replace many existing therapies. CAR T-cell therapy addresses current challenges in cancer care through superior efficacy, safety, and delivery mechanisms. CAR T-cell therapy has brought itself into focus due to the personalized nature of this therapy and the utilization of advanced genetic engineering technology. The wide acceptance and use of CAR T-cell therapy is fueling the growth of the global cell and gene therapy market.

Key Drivers and Trends fueling Market Growth:

Cell and Gene Therapy MarketGeography

The US dominates the cell and gene therapy market in North America due to the high prevalence of chronic diseases and other conditions. There is also comparably high utilization and wide accessibility of these therapies. In Europe, cell and gene therapy products are considered to be part of the Advanced Therapy Medicinal Products (ATMPs), which are commonly known as regenerative medicine globally. The major factors leading to the growth in APAC region are the growing prevalence of cancers, osteoarthritis, burns, and other chronic wounds, the introduction of advanced products in Japan, advanced R&D activities in countries such as South Korea, India.

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Market Segmentation by Geography

Major Vendors

Other vendors include - Anterogen, Tego Sciences, Japan Tissue Engineering, JCR Pharmaceuticals, Medipost, MolMed, AVITA Medical, CollPlant, Corestem, Biosolution, Stempeutics Research, Orchard Therapeutics, Takeda Pharmaceutical Company, CHIESI Farmaceutici, CO.DON, AnGes, GC Pharma, JW CreaGene, APAC Biotech, Nipro Corp., Terumo, Orthocell, and bluebird bio.

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About Arizton:

Arizton Advisory and Intelligence is an innovation and quality-driven firm, which offers cutting-edge research solutions to clients across the world. We excel in providing comprehensive market intelligence reports and advisory and consulting services.

We offer comprehensive market research reports on industries such as consumer goods & retail technology, automotive and mobility, smart tech, healthcare, and life sciences, industrial machinery, chemicals and materials, IT and media, logistics and packaging. These reports contain detailed industry analysis, market size, share, growth drivers, and trend forecasts.

Arizton comprises a team of exuberant and well-experienced analysts who have mastered in generating incisive reports. Our specialist analysts possess exemplary skills in market research. We train our team in advanced research practices, techniques, and ethics to outperform in fabricating impregnable research reports.

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The Cell and Gene Therapy Market to Reach Revenues of Over $6.6 billion by 2024 - Market Research by Arizton - PRNewswire

The Cell and Advanced Therapies Supply Chain Management Market is Anticipated to Grow at an Annualized Rate of 11% till 2030, Claims Roots Analysis -…

Over time, biopharmaceutical companies have realized the importance of integrating advanced software into the cell and advanced therapies supply chain. Such upgrades have demonstrated the ability to offer both time and cost saving

LONDON, Nov. 19, 2019 /PRNewswire/ -- Roots Analysishas announced the addition of the "Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions (Cell Orchestration Platforms, Enterprise Manufacturing Systems, Inventory Management Systems, Laboratory Information Management Systems, Logistics Management Systems, Patient Management Systems, Quality Management Systems, Tracking & Tracing Systems, and Other Software), 2019-2030" report to its list of offerings.

The cell and advanced therapies supply chain is complex, with several legacy challenges, such as those related to patient scheduling, resource planning, inventory management, and deliverable tracking. A number of innovative, software-enabled systems are available / under development to mitigate the aforementioned concerns and simplify the management of biopharmaceutical supply chains.

To order this 420+ page report, which features 150+ figures and 350+ tables, please visit this link

Key Market Insights

Over 160 software-enabled supply chain management systems are currently available

Of these, more than 25% are inventory management systems (IMS), which are primarily used for tracking inventory, orders, sales and deliveries. Examples include (in alphabetical order) ATiM Software, Benchling Inventory, CryoTrackIMS, Cryotrax, CTM-STAR, Lynx Mobile, Mosaic FreezerManagement, and Stafa Apheresis.

~80% of marketed solutions are used in collection centers and sample storage warehousesFurther, about 49% of such systems are being implemented to streamline manufacturing operations of cell and advanced therapies.

Cloud-based deployment is gradually gaining popularity

Stakeholders claim that such deployment methods can be rapidly provisioned with minimal management effort (often over the internet), allowing for faster implementation. At present, about 47% of the software systems are being deployed via cloud; examples include (in alphabetical order) Chronicle automation software, Cryoportal, evo.is, PAS-X MES, STARLIMS, tempmate-CLOUD, and TrakCel.

Around 57% of the stakeholders in this industry are based in the US

This can be attributed to the increasing interest in cell and advanced therapies in this region, making North America the current hub of innovation in this field. It is followed by players in the EU (35%) and Asia Pacific (8%).

2,600+ tweets focused on the increasing interest and ongoing efforts of industry stakeholders

Social media analysis revealed that many biopharmaceutical developers are adopting software-enabled supply chain management solutions with increasing enthusiasm. In fact, many of the tweets were related to the ability of such systems to resolve the challenges associated with large volumes of supply chain data.

Over 10 supply chain orchestration solutions are currently available in the market

These solutions have demonstrated the ability to efficiently integrate core software systems, in order to offer needle-to-needle traceability within complex supply chains.

~USD 650 million has been invested by both private and public investors, since 2014

Of this, close to USD 300 million was raised through venture capital funding rounds, representing 47% of the total capital raised. Further, there were five instances of IPOs / secondary offerings, accounting for USD 280 million in raised capital.

Partnership activity has increased at an annualized rate of 14%

In fact, around 55% of the reported deals were established post 2016; the maximum partnership activity was observed in 2018. Majority of these agreements (75+) were observed to be focused on platform integration or the establishment of service alliances.

North America and Europe are anticipated to capture over 85% market share by 2030

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

Key Questions Answered

The financial opportunity within the cell and advanced therapies supply chain management market has been analyzed across the following segments:

The report features inputs fromeminent industry stakeholders, according to whom software-enabled supply chain management systems have the potential to eliminating risks, and reducing time and capital investment in the cell and advanced therapy development process. The report includes detailed transcripts of discussions held with the following experts:

The research includes elaborate profiles of key stakeholders (listed below), featuring a brief company overview, its financial information (if available), and a detailed description of its platform(s), recent developments and an informed future outlook.

For additional details, please visit

https://www.rootsanalysis.com/reports/view_document/cell-therapies-supply-chain/260.htmlor email sales@rootsanalysis.com

You may also be interested in the following titles:

1. Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA and Other Vectors)

2. Global T-Cell (CAR-T, TCR, and TIL) Therapy Market (4th Edition), 2019 2030

3. Stem Cell Therapy Contract Manufacturing Market, 2019-2030

4. Cell Encapsulation: Focus on Therapeutics and Technologies, 2019-2030

5. Cell and Gene Therapy CROs Market, 2018-2030

Contact:

Gaurav Chaudhary+1(415)-800-3415Gaurav.Chaudhary@rootsanalysis.com

Excerpt from:
The Cell and Advanced Therapies Supply Chain Management Market is Anticipated to Grow at an Annualized Rate of 11% till 2030, Claims Roots Analysis -...

Cell Therapy Market 2019 In-depth Analysis with Inputs from Key Industry Participants – Research Writeups

Cell therapy (also called cellular therapy or cytotherapy) is therapy in which cellular material is injected into a patient; this generally means intact, living cells. For example, T cells capable of fighting cancer cells via cell-mediated immunity may be injected in the course of immunotherapy.

This report on the globalCell Therapy Marketis a detailed research study that helps provides answers and related questions with respect to the emerging trends and growth opportunities in this particular industry. It also highlights each of the prominent factors related to the growth of the market are; growing GDP, demographics, increasing purchasing power, increasing demand, government incentives, government policies, regulatory policies, product standards.

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Top Companiesin the Global Cell Therapy Market (Sales, Price, Revenue, value, volume, market share)-:Dendreon, Mesoblast, Vericel, Novartis AG, GlaxoSmithKline plc, MEDIPOST, Osiris, PHARMICELL, NuVasive, Inc., JCR Pharmaceuticals Co., Ltd, ANTEROGEN.CO.,LTD., Cynata, CELLECTIS, BioNTech IMFS, EUFETS GmbH, Cognate, Pluristem, Grupo Praxis, Genzyme Corporation, Advanced Tissue,and others.

Market Segment by Type

AutologousAllogeneic

Market Segment by Application

Hospitals and clinicsASCsRegenerative medicine centers

Cell therapy products, which are derived from stem cells, tissues, and organs grown in laboratories, are injected into patients. The growing number of clinical trials, government and private funding, and increasing number of partnerships between companies are driving the growth of the global cell therapy market. Cell therapy products that are available in the market are based on autologous and allogenic cells. The demand of cell therapy treatment is increasing. This is because cell therapy products can be used for personalized treatment.

Market Segment by Regions, regional analysis covers:

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

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

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

South America (Brazil, Argentina, Colombia, etc.)

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

You will be able to rely on the comprehensive country coverage, including:

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All of the segments studied in the research study are analyzed on the market share, revenue, and other important factors. Our research study shows how different segments are contributing to the growth of the global Cell Therapy market. It also provides information on key trends related to the segments included in the report. This helps market players to concentrate on high-growth areas of the global Cell Therapy market. The research study also offers separate analysis on the segments on the basis of absolute dollar opportunity.

What is present in the report?

Detailed analysis of data center for specific country

Current opportunity and future potential identification

Most exhaustive and updated report

Helps to identify the current trends, challenges and market drivers

Covers value chain evolution and changing distribution dynamics

Know what your competitors are doing in the market

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Cell Therapy Market 2019 In-depth Analysis with Inputs from Key Industry Participants - Research Writeups

Global CAR T-Cell Therapy for Multiple Myeloma Market 2019 Company Business Overview – Juno Therapeutics, Kite Pharma, Novartis, Collectis – Hitz…

Fior Markets has offered a new market study research namely Global CAR T-Cell Therapy for Multiple Myeloma Market 2019 by Manufacturers, Countries, Type and Application, Forecast to 2024, providing an overall analysis of the CAR T-Cell Therapy for Multiple Myeloma market. The goal of analyzing the complete market was achieved with the help of the previously collected chronological data, the exhaustive qualitative insights, and the statistical data of the market. This study is an effort to provide valuable intelligence for globalbusinesses that are taking effort to obtain dominance over the industry and maximum revenue outcome. It investigates substantial market occurrences from 2014 to 2018 and offers authentic and reliable predictions of up to 2024 that help market players make informed business decisions accordingly.

The report revolves around a historic and present market status to obtain valuable forecast analysis based on market size, share, trends, sales volume, revenue, and growth rate. Various statistics included in the report are made using technical data and industry figures sourced from the most reputable databases. Other aspects of the report that are beneficial to reader include investment feasibility analysis, recommendations for growth, investment return analysis, trends analysis, opportunity analysis, and SWOT analyses of competing companies.

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Market share of global CAR T-Cell Therapy for Multiple Myeloma industry is dominated by companies likeJuno Therapeutics, Kite Pharma, Novartis, Collectis,

On the basis of product, this report displays the production, revenue, price, market share, and growth rate of each type, primarily split into Monotherapy, Combination Therapy

On the basis on the end users/applications, this report focuses on the status and outlook for major applications/end users, sales volume, market share, and growth rate for each application, including Multiple Myeloma, Refractory or Relapsed Multiple Myeloma,

The market is analyzed on the basis of regions namely North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

The report divides the market into diverse segments such as types, applications, regions, end-users, technologies.CAR T-Cell Therapy for Multiple Myeloma market competitive scenario covers a study of participants core values, niche markets, missions, objectives, strengths, and weaknesses. The report mainly focuses on delivering competitive advantages to market players which will help them compete vigorously in the ever-changing business environment.

Furthermore, information related to current and future policies and regulations is organized in the report as well as the regulatory environment is an essential part of the CAR T-Cell Therapy for Multiple Myeloma market which needs special focus. The report presents substantial insights for forthcoming business opportunities, challenges, risks, obstacles, and restraints.Industry leading tools and techniques has been used to evaluate these market dynamics.

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You will come to know a thorough CAR T-Cell Therapy for Multiple Myeloma analysis of the growth sector and able to design and improve your product development and sales strategies accordance to it. You can develop market-entry strategies to sustain competition as the competitors are performing out of the way through precise and updated strategies. You will be able to analyze business frameworks of the known players.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team (sales@fiormarkets.com), who will ensure that you get a report that suits your needs.

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Global CAR T-Cell Therapy for Multiple Myeloma Market 2019 Company Business Overview - Juno Therapeutics, Kite Pharma, Novartis, Collectis - Hitz...

Former Novartis CEO Cell And Gene Therapies Will Drive Innovation – Clinical Leader

By Ed Miseta, Chief Editor, Clinical LeaderFollow Me On Twitter @EdClinical

When Joe Jimenez was promoted to CEO of Novartis in 2010, many in the industry probably thought, Joe who? Jimenez himself chuckles when telling the story, which he did earlier this year at the 2019 Veeva R&D Summit. Jimenez had been at Novartis for less than three years and had a background in the consumer-packaged goods industry. He was working in London when AstraZeneca asked him to sit on the board of the company. In that capacity he learned about the pharmaceutical industry and decided he wanted to be part of it.

When he joined Novartis as division head of Consumer Health, Jimenez took his skillsets from the consumer-packaged goods industry and applied them to pharma. After heading Novartis Consumer Health for a few months, Jimenez was asked to run the pharmaceutical division. That surprised me, he says. I was neither a physician nor a scientist. The board told me they had physicians and scientists. What they wanted was somebody who could look at how the world was changing and position the company to win in that emerging environment."

Joe Jimenez

I said this is nuts, notes Jimenez. We can generate double digit growth if we just start investing in the commercial sales and marketing in China and in parts of Southeast Asia. By doing that, by making those allocation changes, the business took off and then three years later the board made me the CEO.

Partnerships Will Drive Innovation

When Jimenez joined Novartis, the company was launching a lot of products, but many were unsuccessful once they hit the marketplace. He felt the company was not thinking about the commercial potential of company products in the pipeline. To better oversee the income potential of treatments, Jimenez created a group called early commercial development, which he calls the commercial conscience of research.

That was a little nerve wracking, he now says. I was the only individual on the Innovation Management Board with an equal vote who was not an MD or a PhD. This got our scientists to realize that it's not good enough to create a molecule. You have to think about when you're going to launch that molecule and what the competitive landscape is going to look like.

When Jimenez made the move from Big Pharma to small biotech, he also learned about how quickly companies can move. At Novartis, he remembers being in meetings that would last hours simply trying to make a decision that should have taken five minutes. He is now an advisor for several small biotech companies where those long meetings do not take place.

Bureaucracy doesn't necessarily have to be a bad word, he admits. Bureaucracy can certainly mean red tape, but if you have a huge pharmaceutical company without a clear decision making structure, what you are left with is chaos.

One successful strategy that was used at Novartis was in creating a Venture Group, which Jimenez describes as a successful and independent effort. The company put money into a venture fund and told the fund to do its thing. It had access to the resources of Novartis but was not linked to the company.

One thing that makes pharma different from a small biotech is deep resources and the scale and type of people available, says Jimenez. There is an R&D infrastructure and the ability to work on many different projects. In a small biotech we struggle to determine if we are going to focus on one or two projects. A portfolio review at Novartis would discuss 100 different programs. When you are making a small number of bets, you really need to think carefully about those bets. If you make the wrong bet, you will be out of business. A VC firm will help pharma find the good bets by looking for innovation outside their four walls even before those therapies reach the clinical stage."

Jimenez believes we will see more partnering models emerge between pharma and biotech companies, particularly with the explosion of innovation going on in the industry. He believes new treatments being developed in the cell and gene space will drive a lot of the innovation. Those treatments will be assisted by advanced analytics that allow the industry to target disease like never before. That combination will create a new ecosystem of innovation and partnering between Big Pharma and biotech companies.

CAR T Therapy Opens Doors

Shortly after he was promoted to CEO at Novartis, the company was the first to pursue a CAR T therapy. When Novartis invested in the therapy, it still appeared to be science fiction.

We were discussing the concept of taking blood from a cancer patient, genetically engineering the T-cells in that blood to recognize and go after the tumor, and then reinfuse it back into the patient, says Jimenez. This was in 2012, and I remember my oncology people coming into my office with stunned looks on their faces. They had just become aware of Carl June's work at the University of Pennsylvania. His early work was with chronic lymphocytic leukemia, and his CAR T therapy was being used on patients who had no other hope.

Novartis looked at Junes work and decided they had to be involved with it. At the time, no other large pharma companies opted to make an investment. Kite and Juno were two biotechs that emerged in the space and were quickly purchased by Big Pharma.

We were the original pioneers and decided we would do whatever it takes to get those treatments to the patients that needed them, states Jimenez. There were patients who had no other options. We knew this type of therapy had the potential to save thousands of lives. That may not have been our most successful endeavor, but I do think it is the one that has had the biggest impact.

Now that gene editing is a reality, Jimenez believes regenerative medicine holds huge hope for patients. The U.S. population is aging, and Novartis has been investigating how treatments can increase life expectancy and prolong vision, hearing, and muscle as humans age. These are areas where there is significant degeneration.

We can look at muscle as an example, says Jimenez. When humans age they lose a lot of muscle. That makes us frail, which leads to injuries, hip fractures, and eventual death. If we can build muscle among the elderly by finding a way to increase muscle mass, we could potentially extend life. There are currently several small biotech companies looking at muscle, as well as hearing and sight. I believe we may see significant increases in life expectancy in our lifetime.

A Revolution Of Advanced Analytics

Looking towards the future, Jimenez believes advanced analytics and other emerging technologies will revolutionize all elements of the pharma industry, including research, drug development, and commercialization. New technologies can help the entire drug development process work faster, easier, and less expensively.

Jimenez is also a big believer in decentralized trials, which are taking trials to where the patients are instead of to the trial sites that have been used in the past. That model will speed clinical development but also enable patients who would otherwise not be able to participate in a trial. Now anyone can participate even if they dont live near a clinic or medical center.

All of this is happening because of technology, data, and data science, he states. I believe that in the next 10 years we are going to see radical changes in the way that research, development, and commercial are executed. Soon everything will be driven by data.

Today around 40 percent of all investment is going into the area of immuno-oncology. Jimenez believes thats good, since patients will now have new treatment options that they never thought possible. He notes there are around 3,500 immuno-oncology therapies currently being investigated. Still, he worries about what that might mean for more traditional diseases that still have a large unmet medical need. For example, he sees some companies almost completely abandoning respiratory disease, rheumatology, dermatology, GI, and cardiovascular disease. While the news is good for oncology patients, the swing in funding could leave other patients in need.

The patients with respiratory disease deserve new medicines as well, says Jimenez. We have to make sure we prevent what happened in anti-infectives, where there is currently very little activity. We are on the verge of turning several cancers into chronic diseases. That is very exciting news, but we will need companies to pick up the slack in these other disease areas.

Finally, for everyone involved in drug development, Jimenez recommends that you disrupt. Look at what you're working on today and find new ways to radically change the status quo. Data, data science, and advanced analytics are going to be key, he states. The early adopters of technologies that improve efficiency are going to be the ones who win. We are living in an amazing time for healthcare. Our focus is on patients and human health. I just don't think there's a greater mission and purpose.

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Former Novartis CEO Cell And Gene Therapies Will Drive Innovation - Clinical Leader

Global CAR T-Cell Therapy Market 2019 Development Status, Competition Analysis and Application 2024 – News Adopt

GlobalCAR T-Cell TherapyMarket Growth 2019-2024is the definitive study of the globalCAR T-Cell Therapyindustry.Fiormarketsreport helps you prepare to better ride the business cycles while anticipating the future of the market. The report largely focuses on market competition, segmentation, geographical expansion, and other important aspects regarding the market. It contains a deep analysis of microeconomic and macroeconomic factors impacting the growth of the market. The industry outlook report helps you anticipate upcoming trends. Then the research has covered industry drivers, geographic trends, market statistics, market forecasts, producers, and raw material/equipment suppliers. It further highlights leading companies, types, applications, and factors affecting the positive future forecast.

DOWNLOAD FREE SAMPLE REPORT :https://www.fiormarkets.com/report/global-car-t-cell-therapy-market-growth-status-and-382329.html#sample

The market currently witnesses the presence of several major and other key vendors, contributing toward the market growth. The major players functioning the market were identified through the second survey and the market rankings were determined through the first and second surveys. New product development and technological advancements remain essential for competitors to capitalize upon in the industry across the globe. A comprehensive understanding of the market is essential to understanding and facilitating the complete value chain.

For competitor segment, the report includes globalkey playersofCAR T-Cell Therapyas well as some small players covering:Celgene (Juno Therapeutics), Novartis, Gilead (Kite Pharma), Pfizer, CARsgen Therapeutics, Autolus Therapeutics, Aurora BioPharma, Sorrento Therapeutics, Mustang Bio, Bluebird Bio, Collectis, Allogene Therapeutics, Celyad

For geography segment, regional supply, application-wise, and type-wise demand, major players, price is presented from 2014 to 2024. This report covers the following regions: [Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia, Spain), Middle East & Africa (Egypt, South Africa, Israel, Turkey, GCC Countries).

Important Aspects of Report:

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Other Insights Covered In The Market Report:

The research report has analyzed the worlds main regionCAR T-Cell Therapymarket conditions, including the product price, profit, capacity, production, supply, demand, and industry growth rate and forecast, etc. In addition, new project SWOT analysis, investment feasibility analysis, and investment return analysis are also provided. As the market is growing with significant growth potential, our report looks not only at the market today but also at how it will develop over the next five years and the trends and developments that will drive growth. The report delivers a complete overview of market segments and the regional outlook of the market.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team(sales@fiormarkets.com), who will ensure that you get a report that suits your needs.

Arash is the chief editor of News Adopt. He handles the responsibility of covering Technology news. Arash has a rich experience of 15 years in covering technology news. He is also an Adjunct Professor at the University of Southern California.

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Global CAR T-Cell Therapy Market 2019 Development Status, Competition Analysis and Application 2024 - News Adopt

Early Results Are Positive for Experimental CRISPR Therapies – The Scientist

The first patients in each of two early-stage clinical trials testing CRISPR-based treatments for inherited blood disorders have been symptom free for several months with normalized hemoglobin levels, the companies developing the therapies announced today (November 19). Both participants experienced only treatable, temporary side effects, according to the data.

Last fall, Switzerland-headquartered CRISPR Therapeutics and Vertex Pharmaceuticals in Boston teamed up to launch a trial in Germany testing a CRISPR-based approach to treating -thalassemia. The therapy, known as CTX001, is applied to blood stem cells removed from the patient to cleave the BCL11A gene that represses the production of fetal hemoglobin. These cells are then reinfused to provide a healthy supply this protein, normally only produced in infancy, to overcome problems associated with mutations in the gene encoding the adult version. A few months later, the two companies launched a parallel trial in Nashville, Tennessee, to test CTX001 in sickle cell patients, who also suffer from mutations in the gene for adult hemoglobin.

The data released this week pertains to the first patients treated, one in each of these trials. In both participants, the CRISPRed cells successfully homed to the bone marrow. In the nine months since she received the treatment, the -thalassemia patient has required no blood transfusions, which she had needed regularly for 16 years, and her total hemoglobin levels are near-normal, STATreports. Meanwhile, the sickle-cell patient has not suffered any of the painful and organ-damaging events known as vaso-occlusive crises, where the misshapen blood cells characteristic of the disease stick to and clog small blood vessels, since she received her treatment in July, and her total hemoglobin levels have also normalized, according to STAT.

We are very, very excited, Haydar Frangoul, the treating physician at the Sarah Cannon Research Institute in Nashville, tells NPR. This preliminary data shows for the first time that gene editing has actually helped a patient with sickle cell disease. This is definitely a huge deal.

Both patients experienced only minimal side effects, which the treating physicians attributed to the drug busulfan, used to wipe out the patients mutant bone marrow cells before receiving the infusion of CRISPRed blood stem cells.

These results are remarkable because they represent the first clinical evidence that CRISPR-Cas9 has real curative potential, Jeff Leiden, the president and chief executive officer of Vertex Pharmaceuticals, tells STAT. Vertex and CRISPR Therapeutics say they will now proceed with enrolling a total of 45 patients in each trial.

Jef Akst is managing editor ofThe Scientist. Email her atjakst@the-scientist.com.

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Early Results Are Positive for Experimental CRISPR Therapies - The Scientist

CHMP recommends EMA approval of polatuzumab vedotin for the treatment of adult patients with R/R DLBCL – Lymphoma Hub

On the 14th November 2019, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended the approval of polatuzumab vedotin, in combination with bendamustine and rituximab (BR), for the treatment of adult patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who are unsuitable for a hematopoietic stem cell transplant.1 Polatuzumab vedotin is a first-in-class anti-CD79b antibody-drug conjugate, which kills malignant B cells by delivering anti-mitotic monomethyl auristatin E.2

The combination was approved earlier this year by the U.S. Food and Drug Administration(FDA) for the treatment of patients with DLBCL (read more here).

The CHMPs positive opinion was based on data from the global phase Ib/II study GO29365 (NCT02257567) that evaluated the safety, tolerability, and activity of polatuzumab vedotin in combination with BR or obinutuzumab in R/R follicular lymphoma or DLBCL. The polatuzumab vedotin-BR combination achieved a complete response rate of 40% (n= 16/40) compared with 17.5% (n= 7/40) with BR alone, and a median survival of 12.4 months versus 4.7 months, respectively.3,4

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CHMP recommends EMA approval of polatuzumab vedotin for the treatment of adult patients with R/R DLBCL - Lymphoma Hub

CStone announces first patient dosed in the Phase I bridging registrational study of ivosidenib – PRNewswire

SUZHOU, China, Nov. 19, 2019 /PRNewswire/ -- CStone Pharmaceuticals ("CStone" or the "Company", HKEX: 2616)today announced that the first patient has been dosed in the Phase I bridging registrational study of ivosidenib (TIBSOVO) in China. This stand-alone trial is designed to validate the efficacy, safety, and pharmacokinetics of ivosidenib in patients with IDH1 mutant relapsed or refractory acute myeloid leukemia (R/R AML).

Developed by CStone's partner, Agios Pharmaceuticals (NASDAQ: AGIO), ivosidenib was approved by the U.S. FDA in July 2018 for the treatment of adult patients with R/R AML with a susceptibleIDH1 mutation as detected by an FDA-approved test. In May 2019, CStone submitted a new drug application (NDA) for ivosidenib in Taiwan for the treatment of adult patients with IDH1 mutant R/R AML.

Current standard of care treatment for newly diagnosed AML patients mainly includes intensive induction chemotherapy (IC), followed by consolidation therapy such as allogeneic hematopoietic stem cell transplantation (Allo-HSCT) in order to attain durable remission. Approximately 35% to 40% of those treated patients achieve complete remission, while only about 25% achieve 3 years or longer survival. The majority of AML patients develop acquired resistance to treatment or eventually relapse, leading to R/R AML, which has a very poor prognosis in the absence of standard of care treatment options globally. With the emergence of DNA sequencing technology, the detection of genetic mutations has presented new opportunities and challenges in AML treatment. IDH1 mutations are associated with around 6% to 10% of all AML cases.

Dr. Frank Jiang, Chairman and CEO of CStone, commented: "AML is the most common acute leukemia affecting adults with over 30,000 new cases estimated in China every year. AML is characterized by its rapid progression witha five-year survival rate below 20%. We are faced with the urgent need for clinical development, particularly for IDH1 mutant R/R AML patients, due to the lack of any effective treatment in China. We will rigorously press ahead with the clinical development of ivosidenib to achieve its regulatory approval in China which will allow more AML patients in Greater China to benefit from this precision therapy."

CStone's Chief Medical Officer, Dr. Jason Yang, noted: "Ivosidenib is a potent and highly selective IDH1 inhibitor, and the only targeted therapy currently approved by the U.S. FDA for IDH1 mutant AML. It is very encouraging that we have already initiated two registrational studies of ivosidenib in China, including the global Phase III AGILE study of ivosidenib in combination with azacitidinein adult patients with newly diagnosed IDH1 mutant AML who are not eligible for intensive chemotherapy."

About TIBSOVO (ivosidenib)

TIBSOVO is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 25% (7/28) of patients with newly diagnosed AML and 19% (34/179) of patients with relapsed or refractory AML treated with TIBSOVO experienced differentiation syndrome. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal if not treated. Symptoms of differentiation syndrome in patients treated with TIBSOVO included noninfectious leukocytosis, peripheral edema, pyrexia, dyspnea, pleural effusion, hypotension, hypoxia, pulmonary edema, pneumonitis, pericardial effusion, rash, fluid overload, tumor lysis syndrome, and creatinine increased. Of the 7 patients with newly diagnosed AML who experienced differentiation syndrome, 6 (86%) patients recovered. Of the 34 patients with relapsed or refractory AML who experienced differentiation syndrome, 27 (79%) patients recovered after treatment or after dose interruption of TIBSOVO. Differentiation syndrome occurred as early as 1 day and up to 3 months after TIBSOVO initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, initiate dexamethasone 10 mg IV every 12 hours (or an equivalent dose of an alternative oral or IV corticosteroid) and hemodynamic monitoring until improvement. If concomitant noninfectious leukocytosis is observed, initiate treatment with hydroxyurea or leukapheresis, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms and administer corticosteroids for a minimum of 3 days. Symptoms of differentiation syndrome may recur with premature discontinuation of corticosteroid and/or hydroxyurea treatment. If severe signs and/or symptoms persist for more than 48 hours after initiation of corticosteroids, interrupt TIBSOVO until signs and symptoms are no longer severe.

QTc Interval Prolongation:Patients treated with TIBSOVOcan develop QT (QTc) prolongation and ventricular arrhythmias. One patient developed ventricular fibrillation attributed to TIBSOVO. Concomitant use of TIBSOVOwith drugs known to prolong the QTc interval (e.g., anti-arrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) and CYP3A4 inhibitors may increase the risk of QTc interval prolongation. Conduct monitoring of electrocardiograms (ECGs) and electrolytes. In patients with congenital long QTc syndrome, congestive heart failure, or electrolyte abnormalities, or in those who are taking medications known to prolong the QTc interval, more frequent monitoring may be necessary.

Interrupt TIBSOVOif QTc increases to greater than 480 msec and less than 500 msec. Interrupt and reduce TIBSOVOif QTc increases to greater than 500 msec. Permanently discontinue TIBSOVOin patients who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.

Guillain-Barr Syndrome:Guillain-Barr syndrome occurred in <1% (2/258) of patients treated with TIBSOVO in the clinical study. Monitor patients taking TIBSOVO for onset of new signs or symptoms of motor and/or sensory neuropathy such as unilateral or bilateral weakness, sensory alterations, paresthesias, or difficulty breathing. Permanently discontinue TIBSOVO in patients who are diagnosed with Guillain-Barr syndrome.

ADVERSE REACTIONS

DRUG INTERACTIONS

Strong or Moderate CYP3A4 Inhibitors:Reduce TIBSOVOdose with strong CYP3A4 inhibitors. Monitor patients for increased risk of QTc interval prolongation.

Strong CYP3A4 Inducers:Avoid concomitant use with TIBSOVO.

Sensitive CYP3A4 Substrates:Avoid concomitant use with TIBSOVO.

QTc Prolonging Drugs:Avoid concomitant use with TIBSOVO. If co-administration is unavoidable, monitor patients for increased risk of QTc interval prolongation.

LACTATION

Because many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed children, advise women not to breastfeed during treatment with TIBSOVOand for at least 1 month after the last dose.

Please see full Prescribing Information, including Boxed WARNING.

About CStone

CStone Pharmaceuticals (HKEX:2616) is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 15 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, five late-stage candidates are at or near pivotal trials. With an experienced team, a rich pipeline, a robust clinical development-driven business model and substantial funding, CStone's vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.

For more information about CStone Pharmaceuticals, please visit: http://www.cstonepharma.com.

Forward-looking Statement

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

SOURCE CStone Pharmaceuticals

http://www.cstonepharma.com

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CStone announces first patient dosed in the Phase I bridging registrational study of ivosidenib - PRNewswire

These Scientists May Have Found a Cure for ‘Bubble Boy’ Disease – Smithsonian.com

On the morning of April 25, 2018, in Fort Wayne, Indiana, Omarion Jordan came into the world ten-fingers-and-toes perfect. His mother, Kristin Simpson, brought her dark-haired newborn home to a mostly empty apartment in Kendallville, about 30 miles to the north. Shed just moved in and hadnt had time to decorate. Her son, however, had everything he needed: a nursery full of toys, a crib, a bassinet and a blue octopus blanket.

Still, within his first couple of months, he was plagued by three different infections that required intravenous treatments. Doctors thought he had eczema and cradle cap. They said he was allergic to his mothers milk and told her to stop breastfeeding. Then, not long after he received a round of standard infant vaccinations, his scalp was bleeding and covered with green goop, recalled the first-time mother, who was then in her late teens. She took him to the hospital emergency room, where, again, caregivers seemed puzzled by the babys bizarre symptoms, which didnt make any sense until physicians, finally, ordered the right blood test.

What they learned was that Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the bubble boy disease. Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system (hence the unusual reaction to vaccination). The bubble boy name is a reference to David Vetter, a Texas child born with SCID-X1 in 1971, who lived in a plastic bubble and ventured out in a NASA-designed suit. He died at 12, but his highly publicized life inspired a 1976 TV movie starring John Travolta.

Today, technological advances in hospitals provide a kind of bubble, protecting SCID-X1 patients with controlled circulation of filtered air. Such safeguards are necessary because a patient exposed to even the most innocuous germs can acquire infections that turn deadly. As soon as Omarion tested positive for the disorder, an ambulance carried him to Cincinnati Childrens Hospital in nearby Ohio and placed him in isolation, where he remained for the next few months. I had no idea what would happen to him, his mother recalled.

Approximately one in 40,000 to 100,000 infants is born with SCID, according to the Centers for Disease Control and Prevention. Only about 20 to 50 new cases of the SCID-X1 mutationwhich accounts for about half of all SCID casesappear in the United States each year. For years, the best treatments for SCID-X1 have been bone marrow or blood stem cell transplantations from a matched sibling donor. But fewer than 20 percent of patients have had this option. And Omarion, an only child, was not among them.

As it happened, medical scientists at St. Jude Childrens Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patients own stem cells, which then go on to produce the immune system components needed to fight infection. Simpson enrolled Omarion in the clinical study and Cincinnati Childrens Hospital arranged a private jet to transport her and her son to the research hospital, where they stayed for five months.

St. Jude wasnt the first to try gene therapy for SCID-X1. Nearly 20 years ago, researchers in France reported successfully reconditioning immune systems in SCID-X1 patients using a particular virus to deliver the correct gene to cells. But when a quarter of the patients in that study developed leukemia, because the modified virus also disrupted the functioning of normal genes, the study was halted and scientists interested in gene therapy for the disorder hit the brakes.

At St. Jude, experts led by the late Brian Sorrentino, a hematologist and gene therapy researcher, set out to engineer a virus delivery vehicle that wouldnt have side effects. They started with a modified HIV vector emptied of the virus and its original contents, and filled it with a normal copy of the IL2RG gene. They engineered this vector to include insulators to prevent the vector from disturbing other genes once it integrated into the human genome. The goal was to insert the gene into stem cells that had come from the patients own bone marrow, and those cells would then go on to produce working immune system cells. It was crucial for the viral vector to not deliver the gene to other kinds of cellsand thats what the researchers observed. After gene therapy, for example, brain cells do not have a correct copy of the gene, explained Stephen Gottschalk, who chairs St. Judes Department of Bone Marrow Transplantation and Cellular Therapy.

In the experimental treatment, infants received their re-engineered stem cells just 12 days after some of their bone marrow was obtained. They went through a two-day, low-dose course of chemotherapy, which made room for the engineered cells to grow. Within four months, some of the babies were able to fight infections on their own. All eight of the initial research subjects left the hospital with a healthy immune system. The remarkably positive results made news headlines after being published this past April in the New England Journal of Medicine. Experimental gene therapy frees bubble boy babies from life of isolation, the journal Nature trumpeted.

So far, the children who participated in that study are thriving, and so are several other babies who received the treatmentincluding Omarion. As a physician and a mom, I couldnt ask for anything better, said Ewelina Mamcarz, lead author of the journal article and first-time mother to a toddler nearly the same age as Omarion. The children in the study are now playing outside and attending day care, reaching milestones just like my daughter, Mamcarz says. Theyre no different. Mamcarz, who is from Poland, came to the United States to train as a pediatric hematologist-oncologist and joined St. Jude six years ago.

Other medical centers are pursuing the treatment. The University of California, San Francisco Benioff Childrens Hospital is currently treating infant patients, and Seattle Childrens Hospital is poised to do the same. Moreover, the National Institutes of Health has seen success in applying the gene therapy to older patients, ages 3 to 37. Those participants had previously received bone marrow transplants from partially matched donors, but theyd been living with complications.

In the highly technical world of medicine today, it takes teamwork to achieve a breakthrough, and as many as 150 peoplephysicians, nurses, regulators, researchers, transplant coordinators and othersplayed a role in this one.

Sorrentino died in November 2018, but hed lived long enough to celebrate the trial results. In the early 90s, we thought gene therapy would revolutionize medicine, but it was kind of too early, said Gottschalk, who began his career in Germany. Now, nearly 30 years later, we understand the technology better, and its really starting to have a great impact. We can now develop very precise medicine, with very limited side effects. Gottschalk, who arrived at St. Jude a month before Sorrentinos diagnosis, now oversees the hospitals SCID-X1 research. Its very, very gratifying to be involved, he said.

For now the SCID-X1 gene therapy remains experimental. But with additional trials and continued monitoring of patients, St. Jude hopes that the therapy will earn Food and Drug Administration approval as a treatment within five years.

Simpson, for her part, is already convinced that the therapy can work wonders: Her son doesnt live in a bubble or, for that matter, in a hospital. He can play barefoot in the dirt with other kids, whatever he wants, because his immune system is normal like any other kid, she said. I wish there were better words than thank you.

Excerpt from:
These Scientists May Have Found a Cure for 'Bubble Boy' Disease - Smithsonian.com