Archive for the ‘Gene Therapy Research’ Category

DUBLIN, Dec. 17, 2019 /PRNewswire/ -- The "Red Biotechnology Market - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2019-2027" report has been added to ResearchAndMarkets.com's offering.

This study on the red biotechnology market provides readers with a holistic market overview, through an extensive analysis of the market.

The report analyzes the market with regards to the historical and current data to provide a forecast for the period of 2019-2027. Actionable insights and findings pertaining to the red biotechnology market help report readers take major business decisions that support their long-term business growth.

The study includes the compilation of the assessment of significant market dynamics such as key industry trends and major developments carried out by leading players, along with a detailed competitive assessment. The study is divided into key sections to provide readers with an individual understanding of the various aspects of the red biotechnology market.

Key Questions Answered

Key Topics Covered

1. Global Red Biotechnology Market - Preface1.1. Market Definition and Scope1.2. Market Segmentation1.3. Key Research Objectives1.4. Research Highlights

2. Assumptions and Research Methodology2.1. Red Biotechnology Market Definition2.2. Red Biotechnology Market Taxonomy

3. Executive Summary : Global Red Biotechnology Market3.1. Introduction3.1.1. Definition3.1.2. Industry Evolution/Developments3.2. Overview3.3. Market Dynamics3.3.1. Drivers3.3.2. Restrains3.3.3. Opportunities3.4. Global Red Biotechnology Market Analysis and Forecast, 2017-20273.5. Market Revenue Projections (US$ Bn)4. Market Outlook4.1. Pipeline Analysis4.2. Mergers & Acquisitions

5. Global Red Biotechnology Market Analysis and Forecast, by Application 5.1. Introduction5.2. Key Findings/Developments, by Type 5.3. Global Red Biotechnology Market Value (US$ Bn) Forecast, by Application, 2017-20275.3.1. Biopharmaceutical Production5.3.2. Gene Therapy5.3.3. Pharmacogenomics5.3.4. Genetic Testing5.4. Global Red Biotechnology Market Attractiveness Analysis, by Application

6. Global Red Biotechnology Market Analysis and Forecast, by End-user 6.1. Introduction6.2. Key Findings/Developments, by End-user 6.3. Global Red Biotechnology Market Value (US$ Bn) Forecast, by End-user, 2017-20276.3.1. Pharmaceutical Industry 6.3.2. CMO & CRO6.3.3. Research Institutes6.3.4. Others 6.4. Global Red Biotechnology Market Attractiveness Analysis, by End-user

7. Global Red Biotechnology Market Analysis and Forecast, by Region/sub-Region7.1. Key Findings/Developments 7.2. Global Red Biotechnology Market Value (US$ Bn) Forecast, by Region/Sub-Region, 2017-20277.2.1. North America7.2.2. Europe7.2.3. Asia-Pacific 7.2.4. Latin America7.2.5. Middle East & Africa (MEA)7.3. Global Red Biotechnology Market Attractiveness Analysis, by Region/Sub-Region

8. North America Red Biotechnology Market Analysis and Forecast

9. Europe Red Biotechnology Market Analysis and Forecast

10. Asia-Pacific Red Biotechnology Market Analysis and Forecast

11. Latin America Red Biotechnology Market Analysis and Forecast

12. Middle East & Africa Red Biotechnology Market Analysis and Forecast

13. Competition Landscape13.1. Market Player - Competition Matrix 13.2. Market Share Analysis, by Company (2018)13.3. Company Profiles13.3.1. Pfizer Inc.13.3.2. Biogen Inc.13.3.3. Amgen Inc.13.3.4. AstraZeneca PLC13.3.5. Gilead Sciences Inc.13.3.6. Celgene Corporation13.3.7. F. Hoffmann-La Roche Ltd.13.3.8. Merck KGaA13.3.9. Regeneron Pharmaceuticals Inc.13.3.10. Takeda Pharmaceutical Company Limited

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Insights Into the World's Red Biotechnology Market, 2017-2019 & 2027 - Emerging Opportunities, Revenue Projections, Leading Players - PRNewswire

In an effort to understand aging, researchers from Stanford University and colleagues around the world analyzed almost 3,000 proteins in the blood of more than 4,000 people ages 18 to 94. They published their research in the journal Nature Medicine.

When we went into this, we assumed you aged gradually, so we would see these changes taking place relatively steadily as individuals get older, said Tony Wyss-Coray, professor of neurology at Stanford University, senior author of the study.

That isnt what they found, however. They found that the proteins appear to change in three distinct waves, with the first occurring during our 30s, peaking around age 34.

Then we found a second wave around 60, and then we found a third one, the most prominent one, really around 80 years of age, Wyss-Coray said.

The researchers are dubbing this a proteomic clock, that somewhat accurately predicts an individuals age.

One of the reasons for the research was the insight that by placing the blood of younger mice into older mice, the older mice appeared to grow youngerthe reverse happened, toothe blood from older mice aged younger mice. So, whats in the blood of different-aged animals (including people) that is making these changes for better or worse?

Another fact Wyss-Coray and his team knew was that in the blood proteins of people with Alzheimers disease, the biggest differences werent between Alzheimers patients and healthy people of the same age, but between people of different ages.

The researchers winnowed the thousands of proteins they were looking at down to 373 whose levels appeared to be predictive of age.

Wyss-Coray notes that most of the proteins found in the blood derive from other tissues. So we can start to ask where these proteins come from and if they change with age.

For example, proteins that trace back to the liver would suggest the liver is aging. Or if they derive from the kidney, the kidney is aging. Wyss-Coray hopes to eventually be able to analyze blood protein patterns to create a personalized aging clock where I can tell you, based on the composition of your blood, your kidney seems to be aging faster than it should.

And perhaps, if enough information is found, similar to the mouse aging experiments, it might be possible to isolate proteins that contribute to the effectpreferably the one that keeps you young, not makes you old.

Some companies went into the business of selling blood transfusion plasma from young people as an anti-aging potion. The U.S. Food and Drug Administration (FDA), in February 2019, issued warnings to consumers and health care providers against this, pointing out that the FDA did not test these in order to confirm therapeutic benefit or safety.

We strongly discourage consumers from pursuing this therapy outside of clinical trials under appropriate institutional review board and regulatory oversight, the agency said in a statement from then-Commissioner Scott Gottlieb.

Gottlieb added, Our concerns regarding treatments using plasma from young donors are heightened by the fact that there is no compelling clinical evidence on its efficacy, nor is there information on appropriate dosing for treatment of the conditions for which these products are being advertised. Plasma is not FDA-recognized or approved to treat conditions such as normal aging or memory loss, or other diseases like Alzheimers or Parkinsons disease.

The agency warned of possible risks of infectious, allergic, respiratory and cardiovascular problems from the treatments.

Meanwhile, groups like Wyss-Corays and others are attempting to identify which proteins are most effective and whether the proteins themselves are active in staying young or just markers of something else.

Irina Conboy, a researcher at the University of California, Berkeley, published research in the journal Aging in August 2019, that analyzed a protein known as TGF-beta, which is associated with aging. Her experiments in laboratory mice hint that by blocking TGF-beta, aging effects can be slowed.

Wyss-Coray has founded a biotech company, Alkahest, that is researching blood plasma infusions in Alzheimers disease, as well as other applications of blood plasma infusions. At the recent 12th Clinical Trials on Alzheimers Disease conference the company gave an oral presentation of Phase II trial of GRF6019 in mild-to-moderate Alzheimers disease. The Phase IIa trial was completed and patients were randomized and treated with 100mL or 250mL of GRF6019 for five days during Week 1 and again for five consecutive days until Week 13 with a treatment-free interval of 11 weeks after each dose.

GRF6019 and another of its products, GRF6021, are proprietary plasma fractions developed and provided by Grifols. In animal models the plasma fractions improved neurogenesis as well as age-related learning and memory deficits. They also decreased neuroinflammation.

Toshiko Tanaka, an investigator at the National Institute on Aging was the lead author of a study published in 2018 that leveraged similar techniques and also found proteins associated with aging. One of the great things about these advancements, she told NPR, is its becoming a lot cheaper to measure a lot of these molecules, so bigger studies and more studies can assess the same proteins.

At this time, the research is a way off from specifically identifying proteins associated with aging, but its a very good first step.

Paola Sebastiani, a biostatistician at Boston University who has also conducted research on aging and blood proteins, told NPR that, For a long time we have focused, in the field of healthy aging, on genetics.

However, we generally cant modify genesalthough gene therapies and genome editing techniques such as CRISPR are starting to do just that. Sebastiani points out that one of the interesting aspects of these blood proteins is they can be inhibited or blocked or modified using a variety of biochemical and small molecule process that theoretically be used to improve health and slow the aging process.

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Aging in Waves: New Findings on Aging-Related Proteins in the Blood - BioSpace

As 2019 comes to a close, Charles River Laboratories (CRL) has been busy. Following a partnership with Bit Bio announced earlier this month, this morning, CRL announced the acquisition of HemaCare for approximately $380 million in cash.

California-based HemaCare is a provider of human-derived cellular products for the cell therapy market. The company provides biomaterials, including a wide range of human primary cell types. Also, HemaCare supplies cell processing services to support the discovery, development, and manufacture of cell therapies, including allogeneic and autologous programs. Combined with CRLs integrated, early-stage portfolio of discovery, safety assessment, and manufacturing support services, the acquisition of HemaCare will create a comprehensive solution for cell therapy developers and manufacturers worldwide to help accelerate their critical programs from basic research and proof-of-concept to regulatory approval and commercialization, the company said in its announcement. For its clients, CRL believes the addition of HemaCare will enhance client retention and accelerate biopharmaceutical clients speed-to-market.

As more and more cell and gene therapies are expected to be approved over the next several years, CRL believed now was the time to push into the space. James C. Foster, president and chief executive officer of CRL, said that in order to continue to enhance the companys abilities to support the research efforts of its clients, CRL needed to expand its scientific capabilities into this high-growth market.

HemaCare advances the development of life-saving cell therapies through the use of its high-quality cellular products that represent critical inputs to these therapeutics. The addition of HemaCares innovative cell therapy products and services to our integrated, early-stage solutions will create a unique, go-to partner for clients to work with Charles River across a comprehensive cell therapy portfolio from idea to novel therapeutic, Foster said in a statement.

Foster added that HemaCares expertise is well-recognized and pointed to the fact the company has worked on all of the cell therapy drugs approved by the U.S. Food and Drug Administration to date. He said the addition of HemaCare will enhance CRLs value proposition for its clients and will also drive profitable revenue and generate value for the companys shareholders.

The proposed purchase price of $380 million equates to $25.40 per share of HemaCare. That represents a 27% premium to HemaCares closing price on Dec. 13. The acquisition is expected to close early in the first quarter of 2020.

For CRL, the acquisition of HemaCare follows a strategic partnership forged with Bit Bio earlier this month. Bit Bio reprograms human cells for use in research, drug discovery, and cell therapies. Through its partnership with Bit Bio, CRL said it will offer clients access to an expanding suite of authentic human cells through their use in target discovery, validation and screening services.

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Charles River Laboratories Moves into Cell Therapies with $380 Million Acquisition of HemaCare - BioSpace

Gene therapy, once dismissed as too dangerous, has made a comeback, with two products approved in the U.S. since December 2017 and hundreds more in the pipeline. STATs latest report takes a deep dive into a crucial component of these new treatments: the viral vectors used to deliver gene therapies to cells and organs.

As dozens of new gene therapies near the market, we spoke with academic experts, pioneers in the field, and executives with 18 companies, large and small, to identify the most important challenges surrounding the engineering of better vectors, their safety, effectiveness, efficiency, production, and cost and how key players are thinking about overcoming those hurdles.

These engineered viruses are difficult to manufacture, particularly at the massive scale needed for some indications. Scientists are working hard to bring down the cost and speed up the process of making viral vectors, so that all the patients that could benefit from gene therapy will have access to it.

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Beyond the introduction, this report has four major components:

The basics of viral vectors and the history of their development;

Major challenges in the development, manufacturing, and testing of viral vectors, and possible solutions;

A close look at the status of gene therapies in 10 disease categories that are advancing through preclinical studies or are being tested in early-stage clinical trials;

And perspective on the U.S. Food and Drug Administrations approach to regulating viral vectors.

The report The STAT guide to viral vectors, the linchpin of gene therapy is intended for anyone with a strong interest in gene therapy, including biotech executives, investors, scientists, lawyers, policymakers, and patients and families interested in learning more. Our aim is to make the problems, stakes, and possibilities clear to everyone.

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New STAT report explores viral vectors, the linchpin of gene therapy - STAT

The research study on Global Gene Therapy market 2019 presents an extensive analysis of current market size, drivers, trends, opportunities, challenges, as well as key market segments. Further, it explains various definitions and classification of the Gene Therapy industry, applications, and chain structure.

In continuation of this data, the Gene Therapy report covers various marketing strategies followed by key players and distributors. Also explains marketing channels, potential buyers and development history. The intent of global Gene Therapy research report is to depict the information to the user regarding market forecast and dynamics for the upcoming years.

The study lists the essential elements which influence the growth of Gene Therapy industry. Long-term evaluation of the worldwide market share from diverse countries and regions is roofed within the report. Additionally, includes type wise and application wise consumption figures.

After the basic information, the global Gene Therapy Market study sheds light on the technological evolution, tie-ups, acquisition, innovative business approach, new launches and revenue. In addition, the Gene Therapy industry growth in distinct regions and R&D status are enclosed within the report.

The study also incorporates new investment feasibility analysis of Gene Therapy. Together with strategically analyzing the key micro markets, the report also focuses on industry-specific drivers, restraints, opportunities, and challenges in the Gene Therapy market.

Highlights of Global Gene Therapy Market Report:

Table of Content:01: Gene Therapy Market Overview02: Global Gene Therapy Sales, Revenue (value) and Market Share by Players03: Gene Therapy Market Sales, Revenue (Value) by Regions, Type and Application (2014-2018)04: Region wise Top Players Gene Therapy Sales, Revenue and Price05: worldwide Gene Therapy Industry Players Profiles/Analysis06: Gene Therapy Manufacturing Cost Analysis07: Industrial Chain, Gene Therapy Sourcing Strategy and Downstream Buyers08: Gene Therapy Marketing Strategy Analysis, Distributors/Traders09: Gene Therapy Industry Effect Factors Analysis10: Global Gene Therapy Market Forecast (2019-2029)11: Gene Therapy Research Findings and Conclusion12: Appendix

This post was originally published on Market Research Sheets

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Gene Therapy Market by Industry Size, Share, Competitors' Landscape, And Segmentation Including: Regional Segment, Type Segment, Industry Segment,...

Sarepta Therapeutics (SRPT) recently publicized that the FDA approved Vyondys 53. This approval came only a few months after the drug received a CRL due to concerns over the risk of infection at the infusion site and renal toxicity. The company filed an appeal and it looks like the matters raised in the CRL were quickly resolved. SRPT has shot up over 30% following the news, and but is still trading well under its 52-week high of $158.80. Not only is this approval important for the companys commercial outlook, but it also reinstalls some confidence in the companys gene therapy pipeline and technology. Despite the recent big move to the upside, I still see SRPT to be discounted at this current price and could be a lucrative long-term investment.

I intend to provide a brief background on Vyondys 53 and its journey through the FDA. In addition, I discuss why I still think SRPT is still worth a speculative investment. Finally, I reveal my plans for my SRPT position for 2020.

Vyondys 53 is similar to Sareptas Exondys 51, which allows the skipping over gene mutation that inhibits patients with DMD from producing dystrophin. Exondys 51 works in roughly 13% of the roughly 15K boys in the U.S. who have DMD. Although Vyondys 53 will only take aim at 8% of the DMD population, it is still the second most common DMD mutation. Vyondys 53 will be priced at $300K a year per 20kg, so some patients could be paying over $1M a year for the treatment.

Figure 1: DMD and Exon Skipping (Source: SRPT)

Vyondys 53 was an accelerated approval, so Sarepta will need to conduct post-marketing studies to confirm its safety and efficacy. Sarepta is already enrolling patients in their confirmatory trial and should have the results in 2024.

Back in August, Vyondys 53 received a CRL primarily due to renal toxicity concerns from pre-clinical data...at a dose 10 times greater than what was submitted in the NDA. I have seen some ridiculous reasons for the FDA sending a CRL, but citing preclinical data at 10x dosage was by far the most outrageous. I have to imagine almost every drug or therapy is toxic at a 10x dosage. To be honest, I was convinced the FDAs CRL was more than suspicious, but it appears they have backtracked and granted Sarpetas appeal.

Not only is this a win for Sarepta, but it is also a win for gene therapy and the companies who are attempting to develop gene therapeutics. Most of the leading gene therapy stocks took a hit following the CRL as the prospects for gene products making through the FDA's scrutiny seemed to be degrading. Now that the FDA has recognized their error, investors should have renewed confidence in the regulatory and commercial outlook for gene therapies in the US.

As I mentioned before, Vyondys 53 approval has reassured the market that Sarepta's platform and technology are capable of producing more marketable gene therapies. Looking at Sarepta's pipeline (Figure 2) the company can see a great amount of potential value.

Figure 2: SRPT Pipeline (Source: SRPT)

Sarepta is now pushing their next-generation PPMO RNA platform. The company will have safety and dosing insight in 2020. If Sareptas PPMO shows encouraging results they plan to conduct additional research on new therapeutic targets using the PPMO platform.

In terms of microdystrophin manufacturing, the company has completed building out of Sareptas single-use microdystrophin manufacturing facility in Lexington, Massachusetts. In addition, the company also has dedicated suites with Paragon in Maryland, which is expected to have a greater capacity than the Lexington facility. So, it appears the company will have the capacity to support more commercial products as they continue to cross the FDA finish line.

Not only is the company have a strong R&D department, but their commercial team has demonstrated some success with payers, physicians, and prospective patients. The company has recorded revenue growth since Q4 of 2016 and is expected to continue to record sequential revenue growth for the next 7 years (Figure 3).

Figure 3: SRPT Estimated Annual Revenue Growth (Source: Seeking Alpha)

Exondys 51 continues to perform well with Q3 sales above consensus at $99M, which was a 26% increase over Q3 of last year. This prompted the company to boost the 2019 revenue guidance range from $365M to $375M to a range of $370M to $380M for Exondys 51. In fact, Exondys 51 numbers have grown quarter-over-quarter for over the past 3 years.

Another reason to be bullish on SRPT is the companys current financial position, which over $1B. Their healthy cash position will allow the company to fast-track Sareptas growing pipeline and support company operations. Indeed, the company revealed a net loss of $126.3M in Q3, so the company is still incinerating cash. However, Sarepta recently announced an agreement with Pharmakon Advisors, LP, for $500M to be given two $250M tranches. The first will be obtainable after closing and the second will be available at Sareptas option until December 31st of next year. This recent funding not only allows the company to keep the pedal down on R&D, but it was a non-dilutive fundraising event.

What is more, one of Sarepta's potential competitors Wave Life Sciences (WVE) recently announced that it has decided to drop its DMD exon skipping programs. This decision comes after some disappointing data for their exon 51 and exon 53 skipping product candidates. Not only has Sarepta lost a potential competitor, but Wave's exit from DMD shows how advanced Sarepta's exon-skipping products really are.

As a result, I believe Sarepta has the products, pipeline, commercial ability, manufacturing capacity, and the financials to execute on their long-term growth strategy. Returning to figure 3, I expect Sarepta to follow this growth trajectory for the next several years, and will rapidly grow into its current valuation. Sareptas aspiring strategy has created one of the biggest multi-platform genetic medicine pipelines in biotech, which contains over 25 active programs across Sareptas RNA and gene therapy platforms that will be providing multiple catalysts in the coming years. Considering the points above, I still see SRPT to be worthy of speculative buy that could become a lucrative long-term investment.

What's My Plan?

I took a leap of faith following the CRL and added to my speculative SRPT position. Now that I am in the green, I plan on holding my shares for at least four more years and will consider holding if the company is able to get their current pipeline across the finish line. In the meantime, I am going to take advantage of SRPT's volatility and will wait for a pull-back to add to my position. However, I am not going to go all-in until the company has completed their confirmatory studies.

What is more, Sareptas approval has bolstered my confidence in my other gene therapy investments and will look to add to those positions in the near future.

Disclosure: I am/we are long SRPT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Sarepta Therapeutics: FDA Approval Reinstalls Confidence In The Pipeline - Seeking Alpha

The research report Cancer Gene Therapy Market Global Industry Analysis 2019 2025 offers precise analytical information about the Cancer Gene Therapy market. The report identifies top players in the global market and divides the market into several parameters such as major drivers market strategies and imposing growth of the key players. Worldwide Cancer Gene Therapy Industry also offers a granular study of the market dynamics, segmentation, revenue, share forecasts and allows you to make superior business decisions. The report serves imperative statistics on the market stature of the prominent manufacturers and is an important source of guidance and advice for companies and individuals involved in the Cancer Gene Therapy industry.

This Cancer Gene Therapy market report bestows with the plentiful insights and business solutions that will support our clients to stay ahead of the competition. This market report contains categorization by companies, region, type, and application/end-use industry. The competitive analysis covered here also puts light on the various strategies used by major players of the market which range from new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and many others that leads to increase their footprints in this market. The transparent research method carried out with the right tools and methods makes this Cancer Gene Therapy market research report top-notch.

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Competitive Landscape

Global Cancer Gene Therapy market is highly split and the major players have used numerous tactics such as new product launches, acquisitions, innovation in products, expansions, agreements, joint ventures, partnerships, and others to increase their footprints in this market.

Key players profiled in the report include: Adaptimmune, GlaxoSmithKline, Bluebirdbio, Merck, Celgene, ShanghaiSunwayBiotech, BioCancell, ShenzhenSiBionoGeneTech, SynerGeneTherapeutics, OncoGenexPharmaceuticals, GeneluxCorporation, CellGenesys, Advantagene, GenVec, BioCancell, Celgene, EpeiusBiotechnologies, IntrogenTherapeutics, ZiopharmOncology, ShenzhenSiBionoGeneTech, AltorBioscience

Market Segmentation

Cancer Gene Therapy Market report segmentation on Major Product Type:GeneInducedImmunotherapy, OncolyticVirotherapy, GeneTransfer

Market by Application: Here, various application segments of the global Cancer Gene Therapy market are taken into account for the research study.

Hospitals, DiagnosticsCenters, ResearchInstitutes

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Regional Analysis

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

Key Questions Answered in Global Cancer Gene Therapy Market Report:-

What will the market growth rate, overview, and analysis by type of global Cancer Gene Therapy Market in 2026?

What are the key factors driving, analysis by applications and countries Global Cancer Gene Therapy Market?

What are dynamics, this summary includes analysis of the scope and price analysis of top players profiles of Global Cancer Gene Therapy Market?

Who are the opportunities, risk and driving forces of the global Cancer Gene Therapy Market?

Who are the opportunities and threats faced by the vendors in the Global Cancer Gene Therapy Market?

What are the Global Cancer Gene Therapy market opportunities, market risk and market overview of the Market?

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Cancer Gene Therapy Market Expected to Deliver Dynamic Progression until 2028| Adaptimmune, GlaxoSmithKline, Bluebirdbio, Merck - E-Industry News

The global Cancer Gene Therapy market report offers a significant assistant that helps the reader to get a thorough understanding of the value chain analysis. The latest trends, developments, promotion, strategies, and many more provide an uphold success. To reveal the general market trends coupled with conditions and variable tendencies the global Cancer Gene Therapy market report acts as a bible for the reader. The report offers reliable information in relation to the market with proper planning techniques. This report is presented in a precise fashion that records state-of-art information regarding preferences, consumers demands, attitudes, and variable tendencies about the specific product pipelines. The report also aims to offer an open discussion about the global Cancer Gene Therapy market.

About Cancer Gene Therapy Market

Gene therapy is the insertion of a functional gene into the cells of a patient to correct an inborn error of metabolism, in order to alter or repair an acquired genetic abnormality, and to provide a new function to a cell. Owing to high success rate throughout the preclinical and clinical trials, cancer gene therapy is gaining popularity. Improvised technological advancements, increased adoption of developing genomic technologies such as next generation sequencing (NGS) and high density micro array are some of the major factors that will drive the global cancer gene therapy market in next upcoming years. Cancer cells can be differentiated from their usual neighbors on the basis of specific phenotypic changes, such as rapid division rate, attack of new cellular areas, high metabolic rate, and altered shape.

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The insights of the report cover the wide spectrum of the global Cancer Gene Therapy market. With deep insights the reader gets the feasibility while reading that focuses upon the market dynamics governing the trajectory. The in-depth analytical study conducted by the researchers offers strengthens the decision making of the specific market and provoke the analysts to come up with the solution. The report further includes figures and stats coupled with significant compound growth. The compound growth rate directs the reader or analyst to envisage the market growth in base year and forecast time frame.

Competitive Landscapes:

The competitive landscapes are a must-include chapter involving the global players that withstand the competition for the global Cancer Gene Therapy market. This assures the market participants to develop effective strategies to set a benchmark to adopt a significant market position. Further, a competitive environment helps them to determine not only potential advantages but also varied obstacles for the global Cancer Gene Therapy market. In this chapter, the players can examine various strategies and analyze the competitiveness among the players.

List of the Key Players Cited in the Report:

OncoGenex PharmaceuticalsSynerGene TherapeuticsShenzhen SiBiono GeneTechBioCancellShanghai Sunway BiotechCelgeneMerckBluebird bio Inc.GlaxoSmithKlineAdaptimmune

Market Segmentation

On the basis of types, the global Cancer Gene Therapy market is fragmented into

Product 1Product 2

Based on applications, the global Cancer Gene Therapy market is split into:

HospitalsDiagnostics CentersResearch Institutes

With the successive chapters of the Cancer Gene Therapy market, the report further throws the spotlight upon the thorough assessment of the segments at the global outlook. This supports the reader to get a view about the products pipelines, technology, services, end-users, and regions in the overall market. The segment analysis chapter further involves the factors responsible for driving the market on one side while restraining the market on the other side.

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Regional Segmentation

With a wide trend and factors influencing the market that directs regional as well as the primary direction of growth are swung by local market players and unique market drivers. The market study is uplifted at regional as well as country level. This helps to determine the past record and future records through revenue coupled with volume price analysis to involve the region-wise leaders based on the market share and revenue.

Major geographies covered in the report includeNorth America, Europe, Asia-Pacific, South America, and the Middle East & Africa.

Sub-regions includes

The years that were considered for the study of this report are the following:

The study objectives are:

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Cancer Gene Therapy Market 2019| Key Players, Industry Share, Trends, Growth Analysis, Regional Demand and Future Insights by 2026 - Techi Labs

Coherent Market Insights released a new market study on 2018-2026 Cell and Gene Therapy Market with 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. At present, the market is developing its presence. Report offering you more creative solutions that combine our deep geographic experience, intimate sector knowledge and clear insights into how to create value in your business. The research study provides estimates for 2018-2026 Cell and Gene Therapy Market Growth Forecast till 2026*.

The Cell and Gene Therapy Market research report covers the present scenario and the growth prospects of the global Cell and Gene Therapy industry. The report enlists several important factors, starting from the basics to advanced Market intelligence which plays a crucial part in strategizing.

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

Key companies (manufacturing situations, size and production, product specifications etc.) Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

Cell and Gene Therapy Market Report provides key statistics on the market status of the Cell and Gene Therapy manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the Cell and Gene Therapy industry. The Cell and Gene Therapy Market report also presents the vendor landscape and a corresponding detailed analysis of the major vendors operating in the market.

Cell and Gene Therapy Market report analyses the Market potential for each geographical region based on the growth rate, macroeconomic parameters, consumer buying patterns, and Market demand and supply scenarios.

Regions of Cell and Gene Therapy Market:

North America: United States, Canada, Mexico

Europe: Germany, France, UK, Russia, Italy, Rest of Europe

Middle East Africa: Turkey, Egypt, South Africa, GCC Countries, Rest of Middle East & Africa

Asia-Pacific: China, India, Australia, Japan, South Korea, Indonesia, Malaysia, Philippines, Thailand, Vietnam

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In the end, the report makes some important proposals for a new project of Cell and Gene Therapy Industry before evaluating its feasibility. Overall, the report provides an in-depth insight into the global Cell and Gene Therapy industry covering all important parameters.

The Report provides a detailed Cell and Gene Therapy Industry overview along with the analysis of industrys gross margin, cost structure, consumption value, and sale price, Processing Techniques, Network Management, Services Offered, Related Software Market, Social Media Marketing, Cost Structure, Supply Chain, Development Management Techniques, Retailers Analysis, Financial Support, business Strategies, Marketing Channels, Market Entry Strategies, Industry Development Challenges and Opportunities, Investment Plans, Economic Impact on Cell and Gene Therapy Market.

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Rapid Expansion Projected for Cell and Gene Therapy Market by 2026 - Galus Australis

"Our impressive portfolio of life sciences companies underscores the tremendous opportunity for this industry in Central Ohio."

COLUMBUS, Ohio (PRWEB) December 17, 2019

Today, Rev1 Ventures, the investor startup studio that combines capital and strategic services to help startups scale and corporations innovate, is announcing the launch of its second fund focused on life sciences companies. The $15MM Rev1 Catalyst Fund II with investment from The Ohio State University, Nationwide Childrens Hospital and Rev1 is three times the size of the first Life Sciences fund and aims to deliver critical investment capital and support for research-based spinouts and healthcare innovators. As a result of the success of the Rev1 Life Sciences Fund I, which has helped industry trailblazers commercialize technologies and build successful companies bringing novel therapies and solutions to the market, this fund will continue to help Rev1 fulfill its mission to propel the creation of high growth companies in the region.

"Our impressive portfolio of life sciences companies underscores the tremendous opportunity for this industry in Central Ohio, said Tom Walker, president and CEO of Rev1 Ventures. We are proud to help innovators fulfill their potential and bring life-saving technologies and therapies to more people. By aligning champions of healthcare innovation, including prominent research institutions like The Ohio State University and Nationwide Childrens Hospital, we are helping to foster life sciences innovation and support entrepreneurs and scientists as they build and grow new companies. The Rev1 Catalyst Fund II is dedicated to expanding the infrastructure to support life sciences and attracting talent and like-minded investors to fuel commercialization.

The Rev1 Life Sciences Fund I supports an impressive portfolio of companies driving the creation of innovative therapies and solutions, and has generated more than $250MM in exits, including the recent acquisitions of Celenex and Myonexus.

Ohio State is committed to supporting our accomplished researchers at every point along the innovation pipeline, said Cheryl Turnbull, senior director for new ventures at Ohio State. Catalyst Fund II supports Ohio States land grant mission and will help advance the promising life sciences technologies being developed at Ohio State and in Columbus, accelerating medical breakthroughs to market where they can have a positive impact on people throughout the world.

We have many significant success stories about the power of this approach--from the worlds first digital pathology cloud-based platform, Deep Lens, to Milo Biotechnology, a startup working to reverse muscular dystrophy using gene therapy, said Matt McFarland, vice president of commercialization and industry relations at Nationwide Children's Hospital. Myonexus, a Nationwide Childrens spinout that is developing the first-ever corrective gene therapy for limb-girdle muscular dystrophies, was recently acquired by Sarepta Therapeutics. Columbus has the foundational elements in place, so we anticipate this hotbed of activity will only get more robust.

Rev1 and Nationwide Childrens Hospital have been paramount to our success as well as to our ability to take a development and build a business around it, said Dave Billiter, co-founder and CEO of Deep Lens. In this field, it is critical that your investors and partners understand the industry, your potential, your market. The participation of these institutions and the fact that they are in our own backyard, is truly impacting lives and changing healthcare.

For more information about the fund and to learn more about the life sciences startups working with Rev1 Ventures, visit: https://www.rev1ventures.com/our-companies/.

About Rev1 VenturesRev1 Ventures is the investor startup studio that combines capital and strategic services to help startups scale and corporates innovate. Based in the Midwest, and in the number one city for scaling startups, Rev1 aligns innovators and founders with corporate and research partners to access customers and markets, helping entrepreneurs build great companies. With a proven track record of identifying, guiding and investing in high potential startups, Rev1 helps companies solve real problems for markets in need of real solutions. Rev1 has $100MM in capital under management, providing a capital continuum from corporate and community partners, as well as the Ohio Third Frontier. Rev1 is the most active seed investor in Ohio five years running, according to Pitchbook. For more information, visit http://www.rev1ventures.com.

About The Ohio State UniversityThe Ohio State University was founded in 1870 following the Land-Grant College Act of 1862. Classes began in the fall of 1873 with 24 students. The first class of six men graduated in 1878, followed by the first woman graduate in 1879. Today, Ohio State is among the largest and most respected public research universities in the nation, with more than 66,000 students on six campuses. The spring 2017 graduating class of over 11,500 was the largest in school history. Ohio State is known best for its vibrant student experience, research excellence, athletic prowess and highly engaged Buckeye family. The true strength of Ohio State is its people. Buckeye Nation includes more than 45,000 faculty and staff as well as 550,000 alumni living and working in 170 countries across the globe.

About Nationwide Childrens HospitalNamed to the Top 10 Honor Roll on U.S. News & World Reports 2019-20 list of Best Childrens Hospitals, Nationwide Childrens Hospital is one of Americas largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Childrens has a staff of more than 13,000 providing state-of-the-art pediatric care during more than 1.5 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Childrens physicians train the next generation of pediatricians and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Childrens Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.

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Rev1 Ventures Launches $15MM Fund with The Ohio State University and Nationwide Children's Hospital to Support Research-based Healthcare Innovation -...

Understanding Real-World Evidence to Advance Patient-Centric Innovation in Bleeding Disorders

Real-world evidence from studies across many of Takedas portfolio of treatments for hemophilia demonstrate the cost savings and patient benefits resulting from ongoing personalized treatment. However, in von Willebrand disease, real-world evidence highlights the ongoing unmet clinical need for personalization, as it may enable improved treatment outcomes. Insights presented at ASH include:

Real-world evidence plays a crucial role in understanding patterns of care that happen in day-to-day medical practice outside of rigorous clinical studies, said Jonathan Roberts, MD, Associate Medical Director, Bleeding & Clotting Disorders Institute, Peoria, Ill. The U.S. medical claims data show improvement is needed around management of von Willebrand disease in children and adolescents to optimize treatment and reduce the amount of bleeding episodes following diagnosis.

Preclinical Scientific Studies Address Challenges of Current AAV Gene Therapies Takeda also presented data from preclinical scientific studies regarding certain known limitations of AAV gene therapies. These studies will inform Takeda's approach to its own investigational AAV gene therapy programs; TAK-754, an investigational AAV gene therapy for hemophilia A is currently in Phase 1 clinical study, soon to be followed by other potential gene therapies including TAK-748, an investigational gene therapy for hemophilia B.

The treatment goal of gene therapy for hemophilia is to provide sustained therapeutic levels of endogenous clotting factor over multiple years. Hemophilia gene therapies have the potential to provide prolonged, high-level expression of factor, and limit the need for frequent factor infusion.1,2 To deliver gene therapy to a patient, a normal copy of a missing gene is packaged into a delivery vehicle, called a vector.3 Recombinant AAV, particularly those delivered by AAV5 and AAV8 capsid serotypes, serve as the vector in most of the ongoing hemophilia studies.3 The vector delivers the functional gene into a patients liver cells, which can then properly produce blood-clotting proteins. 4,5 However, patients pre-existing immunity to AAV8 capsid, and other AAV serotypes, can impact the safety and efficacy of these therapies.3

To better understand the prevalence of pre-existing immunity against commonly used AAV2, AAV5 and AAV8 capsid in adult patients with hemophilia, Takeda conducted an international prospective and ongoing epidemiological study, Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia, (abstract 3349) that found 50% of patients with hemophilia have neutralizing antibodies to AAV2, AAV5 or AAV8 capsid with 40% demonstrating co-prevalence to all three evaluated serotypes. As a result, these patients are not likely to respond to gene therapies based on AAV vectors.3

As we continue to advance our hemophilia A and hemophilia B investigational gene therapy programs, Takeda is also investigating approaches to overcome the challenges of current AAV gene therapies that could potentially be applied to hemophilia and other rare monogenic diseases, said Dan Curran, M.D., Head, Rare Diseases Therapeutic Area Unit at Takeda. Developing new gene therapy approaches including those capable of treating pre-existing immunity to AAV, enabling re-dosing, lowering doses, enhancing biodistribution and developing alternative gene delivery vehicles are critical to one day providing functional cures to patients.

The poster AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies, (abstract 5922) reported pre-clinical data on one potential approach to overcoming pre-existing AAV immunity.6 In the study, an AAV8-specific immune adsorption column (IAC) was used to mimic the processing of patients plasma in an in vitro setting by applying different treatment cycles to plasma reservoirs which shows anti-AAV8 titers could be depleted.6 Insights from this study will be applied to Takedas research to determine if an IAC could enable the administration of AAV8 gene therapies to patients with pre-existing immunity and potentially facilitate the re-administration of gene therapy.6

ADYNOVATE Professional Important Information

ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Important Information

Indications and Limitation of Use ADYNOVATE is a human antihemophilic factor indicated in children and adults with hemophilia A (congenital factor VIII deficiency) for:

ADYNOVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS Prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).

WARNINGS & PRECAUTIONSHypersensitivity Reactions Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

Neutralizing Antibodies Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.

ADVERSE REACTIONS The most common adverse reactions (1% of subjects) reported in the clinical studies were headache and nausea.

Click here for Full Prescribing Information https://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf

FEIBA [Anti-Inhibitor Coagulant Complex] Indications and Detailed Important Risk Information

Indications for FEIBA

FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for:

FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX.

Detailed Important Risk Information for FEIBA

WARNING: EMBOLIC AND THROMBOTIC EVENTS

CONTRAINDICATIONS

FEIBA is contraindicated in patients with:

WARNINGS AND PRECAUTIONS

Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur, particularly following the administration of high doses (>200 units/kg/day) and/or in patients with thrombotic risk factors.

Patients with DIC, advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with recombinant factor VIIa have an increased risk of developing thrombotic events due to circulating tissue factor or predisposing coagulopathy. Potential benefit of treatment should be weighed against potential risk of these thromboembolic events.

Infusion should not exceed a single dose of 100 units/kg and daily doses of 200 units/kg. Maximum injection or infusion rate must not exceed 2 units/kg/minute. Monitor patients receiving >100 units/kg for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue FEIBA and initiate appropriate diagnostic and therapeutic measures.

Safety and efficacy of FEIBA for breakthrough bleeding in patients receiving emicizumab has not been established. Cases of thrombotic microangiopathy (TMA) were reported in a clinical trial where subjects received FEIBA as part of a treatment regimen for breakthrough bleeding following emicizumab treatment. Consider the benefits and risks with FEIBA if considered required for patients receiving emicizumab prophylaxis. If treatment with FEIBA is required for patients receiving emicizumab, the hemophilia treating physician should closely monitor for signs and symptoms of TMA. In FEIBA clinical studies TMA has not been reported.

Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur. Symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. Reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue FEIBA and provide appropriate supportive care.

Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

FEIBA contains blood group isohemagglutinins (anti-A and anti-B). Passive transmission of antibodies to erythrocyte antigens, e.g., A, B, D, may interfere with some serological tests for red cell antibodies, such as antiglobulin test (Coombs test).

ADVERSE REACTIONS

Most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting.

Serious adverse reactions seen are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.

DRUG INTERACTIONS

Consider possibility of thrombotic events when systemic antifibrinolytics such as tranexamic acid and aminocaproic acid are used with FEIBA. No adequate and well-controlled studies of combined or sequential use of FEIBA and recombinant factor VIIa, antifibrinolytics, or emicizumab, have been conducted. Use of antifibrinolytics within approximately 6 to 12 hours after FEIBA is not recommended.

Clinical experience from an emicizumab clinical trial suggests that a potential drug interaction may exist with emicizumab.

Please see FEIBA full Prescribing Information, including BOXED WARNING on Embolic and Thrombotic Events

About Hemophilia Hemophilia is a challenging chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.7 Hemophilia A is more common than hemophilia B;7 hemophilia A affects about 158,225 people, whereas hemophilia B affects about 31,247 people worldwide.8

People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.7 Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.7,8

About Takeda Hematology Following its recent acquisition of Shire, Takeda is a leader in hemophilia with the longest heritage and market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years driving innovation for patients9 and a broad portfolio of 11 products across multiple bleeding disorders.10 Our experience as leaders in hematology means we are well prepared to meet todays needs as we pursue future developments in the care of bleeding disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and full protection against bleeds, and more personalized patient care.

About Takeda Pharmaceutical Company Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Neuroscience and Rare Diseases. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

For more information, visit https://www.takeda.com.

References 1. National Hemophilia Foundation. Future Therapies. Accessible at: https://www.hemophilia.org/Bleeding-Disorders/Future-Therapies. Accessed: November 2019.2. Wong, T. & Recht, M. Drugs (2011) 71: 305.3. Rajavel, K et al. Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia. Data on File.4. Doshi BS, Arruda VR. Gene Therapy for Hemophilia: What Does the Future Hold? Therapeutic Advances in Hematology. 2018; 9(9):273-293.5. Pipe, SW. Gene therapy for hemophilia. Pediatric Blood Cancer. 2018; 65(2): e26865.6. Kruzik, A et al. AAV8-specific immune adsorption column: A treatment option for patients with pre- existing anti-AAV8 neutralizing antibodies. Data on File.7. World Federation of Hemophilia. What is hemophilia? World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646. Last Accessed April 2019.8. World Federation of Hemophilia. Report on the Annual Global Survey 2017. World Federation of Hemophilia website. http://www1.wfh.org/publications/files/pdf-1714. pdf Last Accessed April 2019.9.World Federation of Hemophilia. About Bleeding Disorders: Treatment. World Federation of Hemophilia website. https://www.wfh.org/en/page.aspx?pid=642. Last Accessed April 2019.10. Shire Website. Product List. Website: https://www.shire.com/products/product-list?t=. Last Accessed June 2019.

Important Notice

For the purposes of this notice, press release means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (Takeda) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, Takeda is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words we, us and our are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. In particular, this press release contains forecasts and management estimates related to the financial and operational performance of Takeda, including statements regarding forecasts for Revenue, Operating profit, Adjusted EBITDA, Profit before income taxes, Net profit attributable to owners of Takeda, Basic earnings per share, Amortization and impairment and other income/expense, Underlying Revenue, Underlying Core Earnings margin, Underlying Core EPS and Net Debt. Without limitation, forward looking statements often include the words such as targets, plans, believes, hopes, continues, expects, aims, intends, will, may, should, would, could anticipates, estimates, projects or words or terms of similar substance or the negative thereof. Any forward-looking statements in this document are based on the current assumptions and beliefs of Takeda in light of the information currently available to it. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takedas business, including general economic conditions in Japan, the United States and worldwide; competitive pressures and developments; applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; changes in exchange rates; claims or concerns regarding the safety or efficacy of marketed products or products candidates; and post-merger integration with acquired companies, any of which may cause Takedas actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takedas results, performance, achievements, or financial position, see Item 3. Key InformationD. Risk Factors in Takedas Registration Statement on Form 20-F filed with the

U.S. Securities and Exchange Commission, available on Takedas website at: https://www.takeda.com/investors/reports/sec-filings/ or at http://www.sec.gov. Neither Takeda nor its management gives any assurances that the expectations expressed in these forward-looking statements will turn out to be correct, and actual results, performance or achievements could materially differ from expectations. Persons receiving this press release should not place undue reliance on forward looking statements. Takeda undertakes no obligation to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make. Past performance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takedas future results.

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Takeda Demonstrates Its Long-Standing Commitment to Advancing Treatments for Rare Bleeding Disorders with Studies Highlighting Real-World Evidence and...

On the heels of an FDA speedy review for Keytrudas potential use in non-muscle invasive bladder cancer (NMIBC), its close rival, a gene therapy by Ferring Pharmaceuticals spinout FerGene, has posted late-stage data. By the looks of it, the two drugs are up for a fight.

Among patients with high-risk NMIBC superficial disease thats unresponsive to standard-of-care Bacillus Calmette-Gurin (BCG), nadofaragene firadenovec eliminated tumors in 53%, or 55 of 103 patients,at month three in a phase 3 study, FerGene unveiled Thursday at the Society of Urologic Oncology meeting.

By comparison, in Keytrudas own registrational trial on the same target patient population, the Merck & Co. PD-1 completely cleared tumors in 41.2%, or 42 of 102 patients, after three months, according to an update at the European Society for Medical Oncology annual meeting in September.

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The length of time responses lasted appeared similar between the two drugs in their separate studies. For Keytruda, 24 patients (23.5%) continued to show no signs of disease after a year. As for nadofaragene firadenovec, 24.3%, or 25 patients, were still tumor-free at month 12.

In terms of safety, Keytruda recorded Grade 3/4 side effects in12.7% of patients, while FerGene said there were no Grade 4/5 events in its study.

We are pleased with these Phase 3 data results, including the complete response rates and favorable safety profile seen with nadofaragene firadenovec, Nigel Parker, scientific founder of FKD Therapies, said in a statement. The data have also helped FKD'snew drug application earn an FDA priority review.

RELATED:Merck's Keytruda is bound for new bladder cancer territory. But can it hold up against gene therapy?

Ferring recently gained commercial rights to the gene therapy from FKD, and, with $400 million in help from Blackstone Life Sciences, spun it into FerGene. Interestingly, it was Merck that licensed the drugout to FKD in the first place in 2011 in return for an equity stake in the then-newly formed Finnish company.

Priority reviews in hand, the two companies could be looking at FDA approvals soon. The burning question is, how does FerGene plan to price a gene therapy, which belongs to a class of drug thats notoriously costly? In a statement sentto FiercePharma, Ferring said it's too early to discuss pricing, that its top priority is still to get nadofaragene firadenovec approved andinvest into R&Dto study the product in more indications.

Keytruda is meant to be given ata fixed dose every three weeks. Nadofaragene firadenovec, which uses an adenovirus vector to deliver the gene interferon alfa-2b to stimulate an innate immune response to fight cancer, is administered into the bladder every three months.

Merck does have an upper hand against FerGene. The Big Pharma has been the sole supplier of BCG in the U.S. and several other key markets globally for several years now. So, it could offer BCG and Keytruda as a one-two punch for NMIBC, similar to the wayBayer is billing Nexavar and Stivarga as a part of the same continuumin first- and second-line liver cancer.

RELATED:Merck limits orders for bladder cancer drug as demand outstrips supply

There are other players eyeing the same patient population. Sesen Bio has Vicinium, an antibody-drug conjugate that targets epithelial cell adhesion molecule antigens on the surface of tumor cells to deliver a toxin payload. In its own phase 3 trial dubbed Vista also in high-risk, BCG-unresponsive NMIBC, Vicinium eradicated tumors in 40% of 89 patients at month three, according to an update the company provided in August. However, its response seems to wane over time more quickly than its rivals', as only 17% of patients showed no signs of tumor activity after 12 months.

The Cambridge, Massachusetts-based biotech recently held two meetings with the FDA and confirmed a submission process, including the design for a post-marketing confirmatory trial. It would enroll BCG-refractory patients who, because of supply constraints, haven't received an optimal BCG dose, which the company said represents a broader patientpopulation in light of anongoing shortage.

Sesen now expects to submit a biologics license application under rolling review by year-end with potential approval in 2020.

As for its pricing, during a presentation at the H.C. Wainwright investor conference in September, Sesens president and CEO Thomas Cannell pointed out that PD-1/L1s would cost about $150,000 to $200,000 per patient per year in NMIBC.

Weve done two rounds of market research with payers, and they think thats reasonable, he said. They think at those levels, there will probably be minimal prior authorization or step edits in terms of restricting a treatments use.

Assuming an official launch in 2021, Jefferies analysts, in a Nov. 12 note to clients, pegged $167.5 million for Viciniums U.S. sales in 2024. Before the priority designation, SVB Leerinks Daina Graybosch predicted a Keytruda launch in NMIBC in 2022 and forecastU.S. sales of $250 million in the indication for the Merck PD-1 inhibitor in 2025.

Editor's Note: The story has been updated with a statement from Ferring Pharma.

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Watch out, Keytruda. Ferring's bladder cancer gene therapy rival has new dataand they look competitive - FiercePharma

Cancer immunology drugs, which harness the bodys immune system to better attack cancer cells, have significantly changed the face of cancer treatment. People with aggressive cancers are now living longer, healthier lives. Unfortunately, cancer immunology therapy only works in a subset of patients.

Now, a new study from scientists at the UCLA Jonsson Comprehensive Cancer Center helps explain why some people with advanced cancer may not respond to one of the leading immunotherapies, PD-1 blockade, and how a new combination approach may help overcome resistance to the immunotherapy drug.

The UCLA study, published today in the inaugural issue of the new scientific journal Nature Cancer, showed that genetic and pharmacological inhibition of the oncogene PAK4 overcomes resistance to anti-PD-1 therapy in preclinical models.

One of the main reasons patients do not respond to PD-1 blockade is because the T cells never make it into the tumor to attack the cancer cells, said lead author Gabriel Abril-Rodriguez, a doctoral candidate in the departments of pharmacology and medicine in the David Geffen School of Medicine at UCLA. We found that biopsies of patients who did not respond to PD-1 blockade showed an overexpression of PAK4, so that led us to believe it played a role in suppressing the immunotherapy treatment.

PAK4 has been known previously to be involved in cell migration and proliferation. The new research from UCLA demonstrates that high expression of this oncogene also correlates with a lack of immune cells migrating into the tumors to destroy the cancer cells.

Using biopsies from people with advanced melanoma who received the immune checkpoint blocking antibody pembrolizumab, UCLA researchers performed RNA sequencing to characterize the phenotype of the tumors. They saw that the tumors that did not respond to PD-1 blockade had a high expression of PAK4 and were not infiltrated by immune cells, meaning that the immune cells had not found their way to the tumor to attack the cancer cells.

The team then inhibited PAK4 in cell lines by either using a drug inhibitor or a gene editing technique called CRISPR-Cas9. The scientists found that deleting PAK4 increased the migration of tumor-specific immune cells and sensitized tumors to PD-1 blockade immunotherapy,reversing the resistance.

Developing new and improved combination treatments like this one for people who do not initially respond to anti-PD-1 treatment is the next step forward in our efforts to make immunotherapy work better for more people, said Dr. Antoni Ribas, the studys senior author, a professor of medicine at the Geffen School and director of the Jonsson cancer centers Tumor Immunology Program. The results from this study could also be expanded to other tumor types that are notoriously resistant to PD-1 blockade, such as pancreatic cancer.

The PAK4 inhibitor used in the study is already being tested in a phase one trial. The combination treatment with anti-PD-1 will be tested in a clinical trial setting in the near future.

The study was funded in part by the National Institutes of Health and the Parker Institute for Cancer Immunotherapy.

Excerpt from:
UCLA study shows inhibition of gene helps overcome resistance to immunotherapy - UCLA Newsroom

To ensure that CRISPR gene editing technology is not misused as was done by a Chinese scientist who created the gene-edited babies recently that attracted global criticism, the Indian Council of Medical Research (ICMR) has framed national guidelines and regulations regarding the procedures and requirements to be followed for performing Gene Therapy in India.

CRISPR stands for clustered regularly interspaced short palindromic repeats are DNA sequences found within the genome of prokaryotes, and are used in genome editing along with enzymes called CRISPR-associated nucleases (most commonly Cas9).

Gene Therapy refers to the process of introduction, removal or change in content of an individuals genetic material with the goal of treating the disease and a possibility of achieving long term cure.

Since this nascent field is emerging in India, the Government has proactively have come up with the National Guidelines for Gene Therapy Product Development and Clinical Trials to promote further research and streamline regulatory processes for future clinical trials using gene therapeutic products (GTPs).

As per the New Drugs and Clinical trial Rules (2019) the GTPs falls under new drug and shall always be deemed to be new drug. Thus as per these rules framed jointly by Indian Council of medical Research and Department of Biotechnology (DBT), academic trials are not applicable to clinical trials using GTPs.

"India has large burden of genetic disorders and unmet medical needs and gene therapy can prove to be a turning point in treatment of such disorders. However, it also brings along with it unique technical risks and ethical challenges," said an official from the ICMR.

She cited the creation of babies using germline gene editing by a Chinese scientist recently that has fuelled a debate on ethical concerns regarding applications of gene therapy technologies.

"This also brought to forefront the requirement of stringent guidelines and regulations to prevent misuse and premature commercialization.

"Many countries around the world have developed rules and guidelines to regulate gene therapy trials.

Taking cognizance of situation, it was felt necessary to frame national guidelines and regulations to direct scientists and clinicians including industry regarding the procedures and requirements to be followed for performing gene therapy in India," she added.

It is proposed to establish Gene Therapy Advisory and Evaluation Committee (GTAEC) anchored at ICMR. GTAEC shall be an independent body of experts representing diverse areas of biomedical research, concerned government agencies and other stakeholders.

"This committee will be composed of a core group of scientists and clinicians in the sector, as well as representation of the government agencies (ICMR, DGHS, CDSCO, DBT, DST, MCI). For each disease area in GTP trials, specific clinical consultants with extensive disease specific expertise will be co-opted to aid in the decision-making process," as per the guidelines.

It says that biological material from humans can be procured only from clinics/hospitals that have an evaluation committee.

The EC must ensure that the Standard Operating Procedures (SOPs) are in compliance with the national guidelines. "Investigators should treat the biological material with utmost respect and adequate care to avoid its misuse. The institute needs to define SOPs for development, production; storage and disposal of the GTPs or its components should be as per the Regulations and Guidelines on Bio-safety of Recombinant DNA Research and Bio-containment 2017," says the guidelines.

Until 2017, almost 2600 gene therapy and 6 clinical trials have been conducted worldwide in 38 countries, of which 64.9 per cent were in USA, 23.2 per cent in Europe and approximately 6.5 per cent were in Asia.

Within Asia, China has reported about 84 gene therapy clinical trials, followed by Japan (44 trials) and South Korea (14 trials). The vast majority of gene therapy trials have addressed cancer (66.6 per cent), monogenic diseases (11.5 per cent), cardiovascular diseases (6.2 per cent) and infectious diseases (6.3 per cent)

Link:
ICMR issues guidelines for gene therapy in India - Daily Pioneer

BOSTON and LONDON, Dec. 08, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, will be presenting new registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, FL.

On Sunday, December 8, 2019, investigators will describe ongoing clinical progress for two lead development programs in the companys primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome (WAS) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy; and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

In addition, on Monday, December 9, 2019, investigators will deliver an oral presentation featuring updated data from the ongoing clinical proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at SR-Tiget.

To learn more about Orchards approach to ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapy, conference attendees can visit booth #2228 in the Exhibition Hall.

Full presentation details are below:

Poster Presentation Details

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010Publication Number: 3346Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded CohortPublication Number: 3345Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Oral Presentation Details

Extensive Metabolic Correction of Hurler Disease by Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II TrialPublication Number: 607Session: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersDate and time: Monday, December 9, 7:00am ET

About ADA-SCID and OTL-101Severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4 The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births in the United States and between one in 200,000 and one in 1 million in Europe.3 OTL-101 is an autologous, ex vivo, hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID being investigated in multiple clinical trials in the United States and Europe, including a registrational trial at the University of California, Los Angeles (UCLA). OTL-101 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of ADA-SCID, and Breakthrough Therapy Designation from the FDA.

About WAS and OTL-103Wiskott-Aldrich Syndrome (WAS) is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age. Treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018 and has been developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

About MPS-I and OTL-203Mucopolysaccharidosis type I (MPS-I) is a rare inherited neurometabolic disease caused by a deficiency of the IDUA (alpha-L-iduronidase) lysosomal enzyme required to break down glycosaminoglycans (also known as GAGs or mucopolysaccharides). The accumulation of GAGs across multiple organ systems results in the symptoms of MPS-I including neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births.5 There are three subtypes of MPS-I; approximately 60 percent of MPS-I patients have the severe Hurler subtype and, when untreated, these patients rarely live past the age of 10.Id Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the UK and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, including any cryopreserved formulations of such product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Orphanet. SCID due to ADA deficiency. 2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314. 3Kwan A, et al. JAMA. 2014;312:729-738. 4Sauer AV, et al. Front Immunol. 2012;3:265. 5Beck et al. The Natural History of MPS I: Global Perspectives from the MPS I Registry. Genetics in Medicine 2014, 16(10), 759.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Showcases Clinical Data at the 61st American Society of Hematology Annual Meeting - BioSpace

The National Academy of Inventors (NAI) has named University of Iowa cystic fibrosis and gene therapy researcher John Engelhardt, PhD, a 2019 Fellow.

Engelhardt, who is professor and head of anatomy and cell biology in the UI Carver College of Medicine and director of the UI Center for Gene Therapy, is recognized for his work in developing gene therapies to treat cystic fibrosis (CF). He will receive the award during an induction ceremony at the Heard Museum in Phoenix, Arizona, on April 10, 2020.

Engelhardts research primarily focuses on the molecular basis of CF, a progressive, inherited disease that causes persistent lung infections and other complications. CF is caused by well-studied mutations in a single gene, and Engelhardt has worked to develop gene therapy and gene editing methods to help treat the condition.

He also develops viral vector systems and animal models to test these methods and ultimately improve gene delivery. The animal models his laboratory has created are used by over 80 CF researchers, and he recently renewed a Research and Resource Center, funded by the National Institutes of Health (NIH), to continue this service to the research community and biotechnology companies that are developing therapies for CF and other lung diseases.

Engelhardt additionally studies airway stem cell niches, or the regulatory mechanisms that control stem cell growth and repair in the lungs, and has developed stem cell therapies for CF.

He currently holds 12 issued US patents, 41 issued foreign patents, and has 23 active patent applications. His patents and applications have been licensed to six companies, including two start-ups and a Fortune 100 company. Engelhardt provides critical tools and assistance to other researchers and companies in the field of CF research, and he is sponsored by the Cystic Fibrosis Foundation.

Engelhardt co-founded the gene therapy company Talee Bio, which was sold and is now Spirovant Sciences. The Philadelphia-based company was recently a part of a $3 billion deal to enhance the development of gene therapies for CF and other genetic diseases. Engelhardt remains on the scientific advisory board for Spirovant Sciences and serves as a key advisor as new therapies are created and tested.

NAI President Paul Sanberg says Engelhardt was selected for induction as he has demonstrated a highly prolific spirit of innovation in creating or facilitating outstanding inventions that have made a tangible impact on the quality of life, economic development, and welfare of society.

The University of Iowa Research Foundation (UIRF) nominated Engelhardt for this award to recognize his impact on creating and broadly commercializing gene therapies and his mentoring of other entrepreneurs on campus.

John has an extensive portfolio of intellectual property for advancing the commercialization of gene therapies, said Marie Kerbeshian, executive director of UIRF and an assistant vice president in the Office of the Vice President for Research. Not only is he a successful entrepreneur, as a UI researcher he is a key supporter of other researchers and other companies as they seek cures for cystic fibrosis.

He is one of 168 distinguished academic inventors across 136 research universities and institutes worldwide to join the academy this year. To date, NAI Fellows hold more than 41,500 issued U.S. patents, and the 2019 class includes six recipients of the U.S. National Medal of Technology & Innovation and U.S. National Medal of Science, four Nobel Laureates, among other honors.

We are very proud to see Dr. Engelhardts innovative and groundbreaking work recognized nationally, said Brooks Jackson, MD, MBA, UI vice president for medical affairs and the Tyrone D. Artz Dean of the UI Carver College of Medicine. He is a pioneer in his field and has set a prime example of how dedication and collaboration can lead to major advances in finding treatments for debilitating diseases.

Engelhardt is the second UI faculty member to join the academy, after UI neurosurgeon Matthew Howard, MD, was named a 2018 fellow for his work in developing brain and spinal cord neuromodulation devices.

Engelhardt joined the UI faculty in 1997 and is the Roy J. Carver Chair in Molecular Medicine and director of the Center for Gene Therapy of Cystic Fibrosis, which has received funding from the NIH continuously over the past 20 years. He earned a doctoral degree in human genetics from Johns Hopkins University and was a post-doctoral fellow at the University of Michigan. He has published 263 articles and book chapters, and has received over $74 million dollars in NIH grant support for his research.

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Engelhardt named 2019 Fellow of the National Academy of Inventors - Iowa Now

WASHINGTON--(BUSINESS WIRE)--FerGene, a new gene therapy company formed by Ferring Pharmaceuticals and Blackstone Life Sciences, announced today positive results from the pivotal Phase 3 clinical trial evaluating nadofaragene firadenovec (rAd-IFN/Syn3), an investigational gene therapy, for the treatment of high-grade, Bacillus Calmette-Gurin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). FKD Therapies Oy (FKD) has led the development and regulatory filing of nadofaragene firadenovec, which has been studied in 33 centers across the U.S. in collaboration with the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). The results were presented during the bladder cancer session at the Society of Urologic Oncology 20th Annual Meeting in Washington D.C.

The Phase 3 study of 157 patients from the U.S. met its primary endpoint with 53% of CIS Ta/T1 patients (carcinoma in situ; bladder cancer that is confined to the superficial layer, with or without concomitant high-grade Ta or T1 papillary disease) achieving a CR at three months, and 24% continuing to show a CR at 12 months. Moreover, the study also demonstrated broad efficacy in this difficult to treat patient population with a 73% HGRF survival in patients with papillary disease at three months and 44% HGRF survival at 12 months. In the study, nadofaragene firadenovec was instilled directly into the patients bladder every three months. All responses at 12 months were confirmed by protocol-mandatory five-point biopsies.

Bladder cancer is one of the most frequently occurring cancers with an estimated 699,450 people living with bladder cancer and more than 80,000 new cases diagnosed each year in the U.S. alone.1 In high-grade NMIBC patients, BCG is the standard treatment, and, although effective, over 60% of these tumors eventually re-occur. 2,3

Currently, patients living with high-grade NMIBC who are unresponsive to BCG have few treatment options and often face bleak outcomes, including complete bladder removal, known as cystectomy, said Colin P. N. Dinney, MD, Professor and Chairman of the Department of Urology at The University of Texas M.D. Anderson Cancer Center. Cystectomy is a complex and life-altering surgical procedure for patients, so these positive results from the Phase 3 trial of nadofaragene firadenovec are highly promising for patients. It would be gratifying to provide an alternative that addresses the critical unmet need for effective second-line therapy for patients facing radical cystectomy.

Efficacy Analysis*

AssessmentPeriod

CIS Ta/T1 Disease (n=103) CR(% [n])

High-Grade Ta/T1 Papillary Disease (non CIS)

(n=48) HGRF Survival (% [n])

Month 3

53.4% (55)

72.9% (35)

Month 6

40.8% (42)

62.5% (30)

Month 9

35.0% (36)

58.3% (28)

Month 12

24.3% (25)

43.8% (21)

*151 patients

In the Phase 3 trial, the most common adverse events (AEs) included fatigue, bladder spasm and discharge around the catheter, micturition urgency, hematuria, chills, fever, headache, painful urination, urinary tract infection, and diarrhea. No grade 4 or 5 treatment-related AEs were reported in the study. Study drug-related AEs were transient and local in nature, with a median duration of less than two days, with the exception of fatigue, which had a median duration of 11 days and urinary frequency which had a median duration of 41 days. There was a 1.9% percent rate of discontinuations due to study drug-related AEs.

We are pleased with these Phase 3 data results, including the complete response rates and favorable safety profile seen with nadofaragene firadenovec, said Nigel R. Parker4, PhD, of FKD Therapies Oy. These data were part of our submission package to the FDA, and we look forward to continuing to work with the agency to potentially bring nadofaragene firadenovec to patients with BCG unresponsive disease.

As a practicing urologist and trial investigator, its encouraging to see these types of efficacy and safety results in patients with high-grade NMIBC, an area thats been in need of new innovative treatment options for more than 20 years, said Neal Shore, MD, FACS, Medical Director, Carolina Urologic Research Center. These robust clinical results further demonstrate the potential of nadofaragene firadenovec as a valuable treatment option for NMIBC patients.

The U.S. Food and Drug Administration (FDA) has validated FKDs Biologics License Application (BLA) and granted Priority Review for nadofaragene firadenovec, which previously received Fast Track and Breakthrough Therapy Designations.

About nadofaragene firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational gene therapy being developed as a treatment for patients with high-grade, BCG unresponsive, NMIBC. It is an adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder every three months. The vector enters the cells of the bladder wall, where, it breaks down, releasing the active gene to do its work. The internal gene/DNA machinery of the cells picks up the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient's own bladder wall cells into multiple interferon microfactories, enhancing the body's natural defenses against the cancer.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is an early form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.5 It is estimated that there will be 80,000 new cases of bladder cancer in the U.S. in 2019; more than 70% of these cases present as NMIBC.2,6 In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, between 30% and 50% cases with high-grade disease will recur.7 The outcome for BCG unresponsive patients is poor, with total cystectomy (complete removal of the bladder) often being the next treatment option.8

About FerGene

FerGene, a new gene therapy company and Ferring subsidiary, has been created to potentially commercialize nadofaragene firadenovec in the U.S. and to advance the global clinical development. FerGenes goal is to bring this promising therapy to a patient population which has seen little improvement in their standard of care over the past twenty years. Blackstone Life Sciences will invest $400 million USD and Ferring will invest up to $170 million USD in FerGene. Ferring will also potentially launch and commercialize nadofaragene firadenovec outside of the U.S.

About FKD Therapies Oy

FKD Therapies Oy is a specialist gene therapy company based in Kuopio, Finland originally conceived by scientific and medical founders, Dr Nigel R Parker and Professor Seppo Yla-Herttuala4, for the specific purpose of undertaking the development of adenovirus mediated interferon alfa-2b. FKD has led the overall development of nadofaragene firadenovec through manufacturing at FinVector Oy, late stage clinical trials and the current BLA filing. FinVector Oy and FKD Oy are part of the Trizell Group.

About Ferring Pharmaceuticals

Ferring Pharmaceuticals is a research-driven, specialty biopharmaceutical group committed to helping people around the world build families and live better lives. Headquartered in Saint-Prex, Switzerland, Ferring is a leader in reproductive medicine and maternal health, and in specialty areas within gastroenterology and urology. Founded in 1950, Ferring now employs approximately 6,500 people worldwide, has its own operating subsidiaries in nearly 60 countries and markets its products in 110 countries.

1 National Cancer Institute. Cancer Stat Facts: Bladder Cancer. Available at: https://seer.cancer.gov/statfacts/html/urinb.html. Last accessed: December 2019.

2 Maruf, M et al., Non invasive bladder cancer: a primer on immunotherapy. Cancer Biol Med. 2016;13(2):194-205.

3 Derr, L et al., Intravesical Bacillus Calmette Guerin Combined with a Cancer Vaccine Increases Local T-Cell Responses in Non-muscle-Invasive Bladder Cancer Patients. Clin Cancer Res. 2017;23(3):717-725.

4 AIV Institute for Molecular Sciences, Kuopio, Finland.

5 Anastasiadis A, de Reijke TM. Best practice in the Treatment of Nonmuscle Invasive Bladder Cancer. Ther Adv Urol. 2012;4(1):13-32

6 Ghatalia, Pooja et al. Approved checkpoint inhibitors in bladder cancer: which drug should be used when?. Therapeutic advances in medical oncology vol. 10 1758835918788310. 30 Jul. 2018, doi:10.1177/1758835918788310.

7 Cambier S et al. EORTC Nomograms and Risk Groups for Predicting Recurrence, Progression, and Disease-specific and Overall Survival in NonMuscle-invasive Stage TaT1 Urothelial Bladder Cancer Patients Treated with 13 Years of Maintenance Bacillus Calmette-Gurin. European Urolology. 2016, Vol. 69(1): 60-69.

8 Cookson, M et al.,Use of intravesical valrubicin in clinical practice for treatment of nonmuscle-invasive bladder cancer, including carcinoma in situ of the bladder. Therapeutic Advances in Urology. 2014, Vol. 5(5):181-191.

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FerGene Announces Pivotal Phase 3 Study of Nadofaragene Firadenovec Met Its Primary Endpoint With More Than Half of Patients With High-Grade...

Sarepeta Therapeutics and StrideBio will collaborate to advance novel gene therapies for four genetic neurological disorders, including Rett syndrome, the companies have announced.

Under the agreement, StrideBio, which specializes in viral-based delivery systems for gene therapy, will conduct initial research, development, and manufacturing for the first four gene targets in these neurological disorders: MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Pick).

The main goal of the early development stage is to obtain investigational new drug approvals from the U.S. Food and Drug Administration, which are mandatory to start clinical trials.

In turn, Sarepta, a company focusing on precision genetic medicine, expands its gene therapy pipeline by gaining exclusive licenses on the selected targets. It also gains the possibility to extend licensing to four additional targets (for a total of eight) in neuromuscular and central nervous system diseases.

StrideBio owns aplatform to create adeno-associated viral (AAV) vectors, a class of modified viruses that are used to deliver gene therapy. They are engineered to be harmless (non-infectious) and work to deliver functioning genes directly into specific cells and tissues.

One of the current challenges in gene therapy is that some people carry a natural immunity against AAVs, in the form of neutralizing antibodies that react against these vectors and prevent gene therapies from working. In addition, immune reactions against AAVs can also become toxic for patients. This limits the number of patients who can benefit from AAV-based gene therapies, as carriers of neutralizing antibodies are excluded from gene therapy trials and treatments.

StrideBios platform addresses this problem by creating novel AAV capsids, or protein shells, that enclose the genetic material to be delivered and are able to escape pre-existing neutralizing antibodies.

As such, the platform holds promise for gene therapies to be used in a greater number of patients, the company says.

These new capsids can also be engineered to improve specific delivery of gene therapy to tissues of interest in a particular condition.

Sarepta and StrideBio plan to address re-dosing challenges as well in patients who have received some sort of AAV-based gene therapy.

With our partnership with StrideBio, Sarepta continues to build on its leadership position in gene therapies to treat rare diseases. We are excited to work with StrideBio and access its innovative AAV platform for next-generation capsids, Doug Ingram, Sareptas president and CEO, said in a press release.

Our partnership with StrideBio expands our research portfolio by up to eight new targets and ensures that we gain access to new technology and targets while not distracting Sarepta from its near-term priorities, he added.

Sapan Shah, PhD, StrideBios CEO, said: This partnership will provide significant resources and expertise to enable StrideBios continued rapid expansion of our research and manufacturing platform, as well as accelerate the development of AAV gene therapies for multiple rare disease targets.

We are looking forward to working together with Sarepta to bring novel treatments to patients as quickly as possible, he added.

Sarepta will pay StrideBio $48 million as upfront payment, in addition to future payments for development, regulatory, and commercial milestones for the four programs. StrideBio will also receive royalties on potential worldwide sales.

If the collaboration is expanded to the four additional targets, Sarepta will pay up to $42.5 million along with future milestone payments.

Ana is a molecular biologist with a passion for discovery and communication. As a science writer she looks for connecting the public, in particular patient and healthcare communities, with clear and quality information about the latest medical advances. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in genetics, molecular biology, and infectious diseases.

Total Posts: 2

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

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Rett Among Disorders Targetted for Gene Therapies in Partnership Deal - Rett Syndrome News

GERMANTOWN, Md., Dec. 06, 2019 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS)(Orgenesis or the Company), a leading cell and gene therapy enabling company providing centralized CDMO manufacturing and development services through its subsidiary Masthercell Global, Inc., as well as localized point-of-care(POCare) development and processing centers for therapeutic treatments, today announced a strategic partnership agreement (Partnership) between the HYGEIA Group and the TheracellOrgenesis joint venture (JV). Under the Agreement, the JV will implement Orgenesis POCare cell therapy platform for clinical development and commercialization of cell and gene therapies within HYGEIA Groups network of three hospitals in Greece. As previously announced, Orgenesis and TheraCell Advanced Biotechnology formed a JV to advance Orgenesis POCare platform in Greece, Cyprus, the Balkan region and selected Middle Eastern countries.

The POCare platform is designed to collect, process and supply cells within the patient care setting for various therapeutic treatments. The goal of the platform is to reduce the cost and complexity of supplying cell and gene therapies, as well as elevate quality standards by integrating automated processing units and proprietary technologies.

HYGEIA is the first hospital network in this region to implement Orgenesis POCare cell therapy platform. The Partnership is intended to provide HYGEIA Group with resources to advance clinical development and deliver personalized, advanced therapies across its network for a wide range of diseases in oncology, hematology, orthopedics, nephrology, dermatology and diabetes.

This partnership with the HYGEIA Group further validates the significant value proposition of our POCare platform, as it enables the development and delivery of cell and gene therapies onsite at hospitals. We believe this platform has the potential to transform the cell and gene therapy market, by bringing life-saving therapies to market in a much more time and cost-effective manner, said Vered Caplan, CEO of Orgenesis. Theracell has proven to be an ideal partner with extensive experience and capabilities in autologous cell therapy and regenerative medicine, with operations in Greece and strong relationships throughout the region. We are in active discussions to establish PoCare locations and partnerships with hospitals and healthcare networks in other countries and regions across the world.

Andreas Kartapanis, CEO, HYGEIA Group, commented, HYGEIA Group is honored to work with Theracell and Orgenesis to become the first hospital network in Greece to provide advanced cell and gene therapies for both clinical research and patient treatment utilizing the POCare platform. We believe this Partnership will provide us a strong competitive advantage in this rapidly developing field. More importantly, this Partnership will benefit patients that will now have greater access to these important therapies.

About HYGEIA Group

HYGEIA Group operates three hospitals in Greece, with a total capacity of 1,261 beds, 52 operating rooms, 19 delivery rooms and 10 intensive care units. More than 3,100 employees and approximately 3,900 associate physicians offer their services to the HYGEIA Group, which was founded in 1970 by medical doctors, most of which were professors at the University of Athens and have since been active in providing primary and secondary care services. The following hospitals are also part of the HYGEIA Group: MITERA General, Obstetrics - Gynecology & Pediatrics Hospital and LITO Obstetrics, Gynecology & Surgical Center, licensed for 459 and 100 hospital beds, respectively.

About Theracell

TheraCell is a regenerative biotechnology company with operations in Greece, where its laboratories and primary facilities are located. The Company focuses in the areas of autologous cell therapy and regenerative medicine. TheraCell has extensive experience in the isolation, processing and application of adipose derived stem cells (ADSCs), as well as somatic cells and has developed a patented platform for tissue engineering and cell therapies in the areas of Dermatology, Chondral Defects and Chronic Kidney Injury.

About Orgenesis

Orgenesis is a biopharmaceutical company specializing in the development, manufacturing and processing of technologies and services in the cell and gene therapy industry. The Company operates through two platforms: (i) a point-of-care (POCare) cell therapy platform (PT) and (ii) a Contract Development and Manufacturing Organization (CDMO) platform conducted through its subsidiary, Masthercell Global. Through its PT business, the Companys aim is to further the development of Advanced Therapy Medicinal Products (ATMPs) through collaborations and in-licensing with other pre-clinical and clinical-stage biopharmaceutical companies and research and healthcare institutes to bring such ATMPs to patients. The Company out-licenses these ATMPs through regional partners to whom it also provides regulatory, pre-clinical and training services to support their activity in order to reach patients in a point-of-care hospital setting. Through the Companys CDMO platform, it is focused on providing contract manufacturing and development services for biopharmaceutical companies. Additional information is available at: http://www.orgenesis.com.

Notice Regarding Forward-Looking StatementsThis press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, the success of our reorganized CDMO operations, the success of our partnership with Great Point Partners, our ability to achieve and maintain overall profitability, the sufficiency of working capital to realize our business plans, the development of our transdifferentiation technology as therapeutic treatment for diabetes which could, if successful, be a cure for Type 1 Diabetes; our technology not functioning as expected; our ability to retain key employees; our ability to satisfy the rigorous regulatory requirements for new procedures; our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended November 30, 2018, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

Investor contact for Orgenesis:David WaldmanCrescendo Communications, LLCTel: 212-671-1021Orgs@crescendo-ir.com

Media contact for Orgenesis:Image Box CommunicationsNeil Hunter / Michelle BoxallTel +44 20 8943 4685neil@imageboxpr.co.uk/michelle@imageboxpr.co.uk

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Orgenesis and Theracell to Launch Point of Care Cell and Gene Therapy Centers within HYGEIA Group's Hospital Network in Greece - GlobeNewswire

The similar efficacy and safety results seen across this post-approval analysis and the ZUMA-1 registrational trial are extremely encouraging for appropriate patients with relapsed or refractory large B-cell lymphoma who may benefit from Yescarta, said Marcelo Pasquini, MD, MS, co-lead investigator and Senior Scientific Director of the CIBMTR; Cellular Therapy Registry Director; and Associate Professor of Medicine, Division of Hematology / Oncology at the Medical College of Wisconsin. The comparable early outcomes, including side effects, support the potential of Yescarta in older, higher risk and more difficult-to-treat patients that physicians often see in the clinic.

In October 2017, Yescarta became the first CAR T cell therapy to be approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma (PMBCL), high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma. The U.S. Prescribing Information for Yescarta contains a Boxed Warning regarding the risk of cytokine release syndrome (CRS) and neurological toxicities; see below for Important Safety Information.

The post-approval study population (n=533) included a larger proportion of older patients (65 years; 37 percent versus 25 percent) and more patients with double and triple hit lymphoma (36 percent versus 11 percent) compared to ZUMA-1.

At follow-up of at least six months after a single infusion of Yescarta, the best objective response via investigator assessment among the 326 patients evaluable for efficacy showed an objective response rate (ORR) of 84 percent, with 66 percent of patients having achieved a complete response (CR). ORR was similar among older and younger patients (92 percent in patients 65 years versus 80 percent in patients <65 years). Patients 65 years (n=108) achieved a CR rate of 72 percent versus 62 percent in patients <65 years (n=218).

Among all patients evaluable for safety (n=533), those 65 years or older had comparable rates of CRS (Any Grade: 84 percent vs. 80 percent; Grade 3: 10 percent vs. 8 percent) and neurologic toxicity (Any grade: 64 percent vs. 55 percent; Grade 3: 22 percent vs. 19 percent) as those under 65 years. Four patients died due to CRS, four patients died from neurologic toxicity and one patient died from both CRS and neurologic toxicity. This Grade 5 AE rate of approximately 1 percent for CRS and neurologic toxicity each is comparable to ZUMA-1.

With more than 85 centers authorized to treat patients with Yescarta, these post-approval results reinforce its potentially transformative role in third line or later relapsed or refractory large B-cell lymphoma, said Christi Shaw, Chief Executive Officer of Kite. The demonstrated efficacy and safety of Yescarta in the real-world setting, coupled with our industry-leading manufacturing that has delivered for thousands of patients so far, means physicians have a real opportunity to bring the potential benefits of CAR T to their patients in need.

U.S. Important Safety Information for Yescarta

BOXED WARNING: CYTOKINE RELEASE SYNDROME AND NEUROLOGIC TOXICITIES

CYTOKINE RELEASE SYNDROME (CRS): CRS occurred in 94% of patients, including 13% with Grade 3. Among patients who died after receiving Yescarta, 4 had ongoing CRS at death. The median time to onset was 2 days (range: 1-12 days) and median duration was 7 days (range: 2-58 days). Key manifestations include fever (78%), hypotension (41%), tachycardia (28%), hypoxia (22%), and chills (20%). Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), cardiac arrest, cardiac failure, renal insufficiency, capillary leak syndrome, hypotension, hypoxia, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome. Ensure that 2 doses of tocilizumab are available prior to infusion of Yescarta. Monitor patients at least daily for 7 days at the certified healthcare facility following infusion for signs and symptoms of CRS. Monitor patients for signs or symptoms of CRS for 4 weeks after infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES: Neurologic toxicities occurred in 87% of patients. Ninety-eight percent of all neurologic toxicities occurred within the first 8 weeks, with a median time to onset of 4 days (range: 1-43 days) and a median duration of 17 days. Grade 3 or higher occurred in 31% of patients. The most common neurologic toxicities included encephalopathy (57%), headache (44%), tremor (31%), dizziness (21%), aphasia (18%), delirium (17%), insomnia (9%) and anxiety (9%). Prolonged encephalopathy lasting up to 173 days was noted. Serious events including leukoencephalopathy and seizures occurred with Yescarta. Fatal and serious cases of cerebral edema have occurred in patients treated with Yescarta. Monitor patients at least daily for 7 days at the certified healthcare facility following infusion for signs and symptoms of neurologic toxicities. Monitor patients for signs or symptoms of neurologic toxicities for 4 weeks after infusion and treat promptly.

YESCARTA REMS: Because of the risk of CRS and neurologic toxicities, Yescarta is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta REMS. The required components of the Yescarta REMS are: Healthcare facilities that dispense and administer Yescarta must be enrolled and comply with the REMS requirements. Certified healthcare facilities must have on-site, immediate access to tocilizumab, and ensure that a minimum of 2 doses of tocilizumab are available for each patient for infusion within 2 hours after Yescarta infusion, if needed for treatment of CRS. Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense or administer Yescarta are trained about the management of CRS and neurologic toxicities. Further information is available at http://www.YESCARTAREMS.com or 1-844-454-KITE (5483).

HYPERSENSITIVITY REACTIONS: Allergic reactions may occur. Serious hypersensitivity reactions including anaphylaxis may be due to dimethyl sulfoxide (DMSO) or residual gentamicin in Yescarta.

SERIOUS INFECTIONS: Severe or life-threatening infections occurred. Infections (all grades) occurred in 38% of patients, and in 23% with Grade 3. Grade 3 or higher infections with an unspecified pathogen occurred in 16% of patients, bacterial infections in 9%, and viral infections in 4%. Yescarta should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after Yescarta infusion and treat appropriately. Administer prophylactic anti-microbials according to local guidelines. Febrile neutropenia was observed in 36% of patients and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad spectrum antibiotics, fluids and other supportive care as medically indicated. Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure and death, can occur in patients treated with drugs directed against B cells. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS: Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and Yescarta infusion. Grade 3 or higher cytopenias not resolved by Day 30 following Yescarta infusion occurred in 28% of patients and included thrombocytopenia (18%), neutropenia (15%), and anemia (3%). Monitor blood counts after Yescarta infusion.

HYPOGAMMAGLOBULINEMIA: B-cell aplasia and hypogammaglobulinemia can occur. Hypogammaglobulinemia occurred in 15% of patients. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis and immunoglobulin replacement. The safety of immunization with live viral vaccines during or following Yescarta treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during Yescarta treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES: Patients may develop secondary malignancies. Monitor life-long for secondary malignancies. In the event that a secondary malignancy occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

EFFECTS ON ABILITY TO DRIVE AND USE MACHINES: Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following Yescarta infusion. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, during this initial period.

ADVERSE REACTIONS: The most common adverse reactions (incidence 20%) include CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections-pathogen unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.

About Kite

Kite, a Gilead Company, is a biopharmaceutical company based in Santa Monica, California. Kite is engaged in the development of innovative cancer immunotherapies. The company is focused on chimeric antigen receptor and T cell receptor engineered cell therapies. For more information on Kite, please visit http://www.kitepharma.com.

About Gilead Sciences

Gilead Sciences, Inc. is a research-based biopharmaceutical company that discovers, develops and commercializes innovative medicines in areas of unmet medical need. The company strives to transform and simplify care for people with life-threatening illnesses around the world. Gilead has operations in more than 35 countries worldwide, with headquarters in Foster City, California. For more information on Gilead Sciences, please visit the companys website at http://www.gilead.com.

About the CIBMTR

The CIBMTR (Center for International Blood and Marrow Transplant Research) is a research collaboration between the National Marrow Donor Program (NMDP)/Be The Match and the Medical College of Wisconsin (MCW). Through a collaboration with Be The Match BioTherapies, a subsidiary of NMDP/Be The Match, the organizations offer end-to-end solutions for cell and gene therapy developers, including cell sourcing and collection support, clinical trial services, supply chain and logistics, manufacturing and commercialization support, and outcomes management. The CIBMTR collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide to increase survival and enrich quality of life for patients. The CIBMTR has developed a dedicated cellular therapy registry which now serves as the infrastructure for the National Cancer Institute -funded Cellular Immunotherapy Data Resource (CIDR) and further expands the utilization of this resource by the general biomedical community. The CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of transplant centers, and a unique and extensive clinical outcomes database.

For more information on the CIBMTR, please visit http://www.cibmtr.org.

Forward-Looking Statement

This press release includes forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the possibility of unfavorable results from other ongoing and additional clinical trials involving Yescarta. There is also the risk that physicians may not see the benefits of the use of Yescarta for relapsed or refractory large B-cell lymphoma in the older and more difficult-to-treat patient population as described herein. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. The reader is cautioned not to rely on these forward-looking statements. These and other risks are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, as filed with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation to update any such forward-looking statements.

U.S. Prescribing Information for Yescarta, including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com.

Yescarta is a registered trademark of Gilead Sciences, Inc., or its related companies.

For more information on Kite, please visit the companys website at http://www.kitepharma.com or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

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Kite and the CIBMTR Present Positive Findings From Real-World Use of Yescarta (Axicabtagene Ciloleucel) in Relapsed or Refractory Large B-Cell...

BRISBANE, Calif. & NEW YORK--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, and Pfizer, Inc. (NYSE: PFE), today announced updated follow-up results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy in patients with severe hemophilia A. The data showed that SB-525 was generally well tolerated and demonstrated sustained increased Factor VIII (FVIII) levels following treatment with SB-525 through to 44 weeks, the extent of follow-up for the longest treated patient in the 3e13 vg/kg dose cohort. Data from 11 patients treated with SB-525 will be featured in a poster presentation today, December 7, 2019, at the 61st Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL. The SB-525 ASH poster, which includes the full set of data, is available on Sangamos website in the Investors and Media section under Events and Presentations.

I am pleased that all five patients in the high dose (3e13 vg/kg) cohort rapidly achieved normal levels of Factor VIII, and that Factor VIII levels have been stable and durable in the normal range for the first two patients up to 44 and 37 weeks following treatment respectively, with no bleeding events or factor usage up to a follow up of 44 weeks in the longest treated patient, said Barbara Konkle, M.D., Bloodworks Northwest, Professor of Medicine at University of Washington and a Principal Investigator of the Alta study. It is important to continue to follow these patients to determine whether these results are sustained in the longer term as the combination of a favorable safety profile coupled with sustained expression at a level that prevents bleeding and allows normal activity will be the hallmark of a successful gene therapy for hemophilia A.

Alta study data presented at ASH included 11 patients treated across four ascending dose cohorts: 9e11 vg/kg (2 patients), 2e12 vg/kg (2 patients), 1e13 vg/kg (2 patients) and 3e13 vg/kg (5 patients). The data cutoff date was October 17, 2019.

An analysis of plasma FVIII antigen was assessed by ELISA and demonstrated antigen concentrations consistent with the FVIII activity measured by the chromogenic assay. Dose dependent increases in FVIII activity over baseline were observed across the dose cohorts. The lower-dose cohorts indicate durable FVIII activity with up to 52 weeks of follow-up.

In the 3e13 vg/kg dose cohort, patients achieved normal range FVIII activity within 5-7 weeks of treatment with SB-525. The first two patients treated in this cohort (Patients 7 and 8) have achieved stable FVIII levels, demonstrating durability in the normal range through 44 and 37 weeks, respectively, as measured by the chromogenic assay. The two patients most recently treated in this cohort (Patients 10 and 11), with 22 and 12 weeks of follow-up, respectively, demonstrated a similar pattern of FVIII expression. The FVIII expression pattern observed in Patient 9 differed from that of other patients in the cohort. Seven weeks following treatment, Patient 9 achieved normal range FVIII levels. Beginning at week 13, FVIII levels in that patient fluctuated in a range below normal, but still well above the level needed to prevent spontaneous bleeding. At week 18, FVIII levels in Patient 9 began to increase, and as of the latest measurement at week 24, continued to rise. No patient in the 3e13 vg/kg dose cohort has experienced bleeding events up to 44 weeks of follow-up, and no patient in this dose cohort required factor replacement following initial use of prophylactic factor.

SB-525 was generally well tolerated across all dose cohorts. The treatment-related adverse events include: alanine aminotransferase (ALT) elevation (36.4%, n=4), pyrexia (27.3%, n=3), increased aspartate aminotransferase (18.2%, n=2), tachycardia (18.2% n=2), fatigue (9.1%, n=1), hypotension (9.1%, n=1) and myalgia (9.1%, n=1). Treatment-related serious adverse events (SAEs) of hypotension (grade 3) and fever (grade two) occurred in one patient in the 3e13 vg/kg cohort six hours following dosing with SB-525 that fully resolved within 24 hours. No similar events were reported in the other patients dosed in that cohort. No patients treated with SB-525 experienced an ALT elevation associated with loss of Factor VIII expression. In the 3e13 vg/kg dose cohort, four patients experienced transient low grade ALT elevations (>1.5 x baseline) that were managed with a tapering course of oral steroids. The study does not use corticosteroids prophylactically, initiating them only in the event of an ALT elevation that is greater than 1.5x baseline.

The updated results from the Alta study suggest that SB-525 may represent a differentiated gene therapy for patients with severe hemophilia A, said Bettina Cockroft, M.D., Chief Medical Officer of Sangamo. The results continue to suggest that if sustained over a longer duration, SB-525 has the potential to be a predictable, reliable, and safe treatment that may bring clinical benefits to patients with severe hemophilia A.

Sangamo has completed the manufacturing technology transfer and initiated the transfer of the Investigational New Drug (IND) Application to Pfizer, which is expected to be completed in the first quarter 2020. Pfizer is enrolling patients in the Phase 3 lead-in study, the data from which is expected to provide a baseline for patients who are subsequently enrolled into the Phase 3 study (ClinicalTrials.gov Identifier: NCT03587116).

We are pleased with the progress that we have made in progressing SB-525 gene therapy toward a Phase 3 registrational study, including enrolling the first patient in the 6-month lead-in study. We expect to dose the first patient in the Phase 3 registrational study in 2020, said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizers Rare Diseases Research Unit. We continue to believe that if the observed safety and efficacy results are sustained, this gene therapy has the potential to transform the treatment paradigm of severe hemophilia A.

About the Alta study

The Phase 1/2 Alta study is an open-label, dose-ranging, multicenter clinical trial designed to assess the safety and tolerability of SB-525 in patients with severe hemophilia A. The mean age of the 11 patients assessed is 30 years (range 18-47 years). All 11 patients are male. The U.S. Food and Drug Administration has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer.

About SB-525 Gene Therapy

SB-525 comprises a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. The SB-525 vector cassette was designed to optimize both the vector manufacturing yield and liver-specific FVIII protein expression. The SB-525 transcriptional cassette incorporates multi-factorial modifications to the liver-specific promoter module, FVIII transgene, synthetic polyadenylation signal and vector backbone sequence.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Pfizer Inc: Working together for a healthier world

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Sangamo Forward Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the investigational hemophilia A gene therapy, SB-525, including its potential therapeutic benefits; the potential long-term durability of SB-525 gene therapy; SB-525 having the potential to be a predictable and reliable treatment that may bring clinical benefit to patients with hemophilia A and to potentially represent a transformative treatment paradigm; plans to advance SB-525 into a potential registrational study; the potential benefits of the RMAT and Orphan medicine designation for SB-525; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the research and development process; additional data, including the risk that the data reported from the Alta to date may not be indicative of the final results from the Alta study or that such final results may not validate and support the safety and efficacy of SB-525; the completion of the Alta study; the possibility of unfavorable new clinical data from the Alta study and further analyses of existing clinical data from the study that may material change clinical outcomes; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clincal studies relating to SB-525, any potential registrational studies or any other clinical studies of SB-525; whether Sangamo will be able to maintain or receive the benefits associated with RMAT, Orphan Drug, Fast Track and Orphan Medicinal Product designations for SB-525; Sangamo's reliance on Pfizer and other third-parties to meet their clinical and manufacturing obligations; Sangamos ability to maintain strategic partnerships; and the potential for technological developments by Sangamo's competitors that will obviate Sangamo's gene therapy technology. Further, there can be no assurance that the necessary regulatory approvals will be obtained for SB-525 or that Sangamo and its partners will be able to develop commercially viable product candidates. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission and Sangamo's most recent Quarterly Report on Form 10-Q. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

Pfizer Disclosure Notice: The information contained in this release is as of December 7, 2019. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about an investigational hemophilia A agent, SB-525, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications for any potential indications for SB-525 may be filed in any jurisdictions; whether and when regulatory authorities in any jurisdictions may approve any such applications, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether SB-525 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of SB-525; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

Link:
Sangamo and Pfizer Announce Updated Phase 1/2 Results Showing Sustained Increased Factor VIII Activity Through 44 Weeks Following SB-525 Gene Therapy...

One of the most popular methods for the insertion of therapeutic genes into cells to treat the disease is to transport using a virus that has been stripped of their infectious properties. But non-infectious virus can still sometimes trigger immune responses dangerous.A team of Johns Hopkins Medicine proposes an alternative method for transporting large therapies cellsincluding genes and even gene-CRISPR system edition. It is a nano-container made of a polymer that biodegrades once their inside the cell, triggering therapy. The researchers describe the invention in the journal Advances Science.The team, led by biomedical engineer Jordan Green, Ph.D., was inspired by viruses, which have many properties that make them ideal vehicles. They have both negative and positive charges, for example, allowing the cells to approach them. Thus Green and his colleagues developed a polymer containing four molecules with positive and negative charges. I used to make a container that interacts with the cell membrane and eventually enveloped by it.How ICON, Lotus and Bioforum are improving the efficiency of modern studio with EDCCRO are often at the forefront of adopting new technologies to make clinical trials more efficient. Listen how ICON, Lotus Clinical Research, and Bioforum are accelerating the basis of building data and automating tasks management information data.RELATED: Could a grape-based compound to improve the efficiency of gene therapy?Hopkins researchers conducted four experiments to test the nanocontainers travel in cells and deliver complex therapies once inside. First, a small protein packaged in the polymeric material and mixed with mouse kidney cells in a laboratory dish. The use of fluorescent tags confirmed that the protein made in the cell. Experiment with immunoglobulinand noted a medicinehuman much larger than the 90% of renal cell received treatment was then repeated.From there, they made the payload, even bulkier packaging the nanocontainers with gene-CRISPR system edition. With the help of fluorescent signals that were able to confirm that CRISPR was to work once inside the cells, the inactivation of a gene 77% of the time.Thats pretty effective considering with other genes editing systems, it is possible to obtain the result of cutting right genes less than 10 percent of the time, said graduate student Rui Yuan said in a statement.Finally, the Hopkins researchers injected CRISPR components in mouse models of brain cancer using polymer nanocontainers. Again evidence that had taken place editing gene was successful.The development of improved methods for gene therapy is a priority in the field. In October, for example, scientists at Scripps Research describes a way to use a small molecule called caraphenol A to reduce levels of transmembrane induced by interferon (IFITM) proteins, which could, in turn, will allow viral vectors to pass more easily into cells. And earlier this year, an Italian equipment described a method for including the CD47 protein lentiviral vectors to improve the transfer of therapeutic genes into cells of the liver.The next step for researchers Hopkins Rui and green is to improve the stability of nanocontainers so they can be injected into the bloodstream. They hope to be able to direct them to cells that have certain genetic markers, it was reported.

Read more:
Hopkins invented equipment nonviral system for gene therapy in cells - Market Research Feed

The "Gene Therapy Market" report includes an in-depth analysis of the global Gene Therapy market for the present as well as forecast period. The report encompasses the competition landscape entailing share analysis of the key players in the Gene Therapy market based on their revenues and other significant factors. Further, it covers the several developments made by the prominent players of the Gene Therapy market. The well-known players in the market are Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

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The company profiles presented in the report include company synopsis, business tactics adopted, and major developments. Furthermore, The report presents a detailed segmentation Ex vivo, In vivo, Market Trend by Application Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others of the global market based on technology, product type, application, and various processes and systems. Additionally, the report provides competition al circumstances within the major players in the Gene Therapy market. The report also includes the companies active in product expansions and innovating new advanced technology intending to develop huge opportunities for the Gene Therapy market.

The report also provides the market dynamics such as drivers, restraints, strategies & guidelines, trends, avenues, and technological improvements anticipated to have an impact on the Gene Therapy Market growth in the projected period. The study gives a detailed analysis of the development of the market during the forecast period. Further, the report also reviews the market in terms of value [USD Million] and size [k. MT] across diverse regions.

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Moreover, the report comprises major developments made in the Gene Therapy market. Porters five force analysis is used to determine the competition in the Gene Therapy market along with new entrants and their strategies & tactics. The report involves the value chain analysis which denotes workflow in the Gene Therapy market. Furthermore, the market has been classified on the basis of category, processes, end-use industry, and region. On the basis of geography, the report bifurcates the market.

Thus, this report is a compilation of all the data necessary to understand the Gene Therapy market in every aspect.

There are 15 Chapters to display the Global Gene Therapy market

Chapter 1, Definition, Specifications and Classification of Gene Therapy, Applications of Gene Therapy, Market Segment by Regions;Chapter 2, Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, Technical Data and Manufacturing Plants Analysis of Gene Therapy, Capacity and Commercial Production Date, Manufacturing Plants Distribution, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, Regional Market Analysis that includes United States, China, Europe, Japan, Korea & Taiwan, Gene Therapy Segment Market Analysis (by Type);Chapter 7 and 8, The Gene Therapy Segment Market Analysis (by Application) Major Manufacturers Analysis of Gene Therapy ;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type Ex vivo, In vivo, Market Trend by Application Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, The Consumers Analysis of Global Gene Therapy ;Chapter 12, Gene Therapy Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, Gene Therapy sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

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Reasons for Buying Gene Therapy Market

This report provides pin-point analysis for changing competitive dynamicsIt provides a forward looking perspective on different factors driving or restraining market growthIt provides a six-year forecast assessed on the basis of how the market is predicted to growIt helps in understanding the key product segments and their futureIt provides pin point analysis of changing competition dynamics and keeps you ahead of competitorsIt helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments

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Global Gene Therapy Market Executive Summary and Analysis by Top Players 2019-2025: Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche...

The Cancer Gene Therapy research report is a valuable source of data for business strategists. It provides the Cancer Gene Therapy overview with growth analysis and historical and futuristic cost revenue demand and supply data. The research analysis provides an elaborative description of the value chain and distributor analysis.

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The Cancer Gene Therapy market study provides comprehensive data that enhance the understanding, scope and application of this report.

The report provides a basic overview of the industry including definitions and classifications. The Cancer Gene Therapy analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status.

Prominent players of Cancer Gene Therapy market:

Product Type Coverage (Market Size & Forecast, Major Company of Product Type etc):

Application Coverage (Market Size & Forecast, Different Demand Market by Region, Main Consumer Profile etc.):

Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins.

This report studies the Cancer Gene Therapy status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyses the top players in global market, and splits the Cancer Gene Therapy By product type and applications/end industries.

Read Detailed Index of full Research Study at @ https://99marketresearch.com/global-cancer-gene-therapy-market-size-status-and-forecast-2019-2025/172976/

To comprehend 2018-2026 Cancer Gene Therapy dynamics in the world mainly, the worldwide 2018-2026 Cancer Gene Therapy is analyzed across major global regions. Cancer Gene Therapy Also provides customized specific regional and country-level reports for the following areas.

North America: United States, Canada, and Mexico.

South & Central America: Argentina, Chile, and Brazil.

Middle East & Africa: Saudi Arabia, UAE, etc

The study objectives of this report are:

To study and forecast the market size of Cancer Gene Therapy

To analyze the global key players, SWOT analysis, value and global market share for top players.

To define, describe and forecast the market by type, end-use and region.

To analyses and compare the market status and forecast among global major regions.

To analyses the global key regions market potential and advantage, opportunity and challenge, restraints and risks.

To identify significant trends and factors driving or inhibiting market growth.

TOC-

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

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Cancer Gene Therapy Market 2019, Trend, CAGR Status, Growth, Analysis and Forecast to 2025 - Drnewsindustry

This market research report provides a big picture on Gene Therapy Market, on a Global basis, offering a forecast and statistic in terms of revenue during the forecast period. The report covers a descriptive analysis with detailed segmentation, complete research and development history, latest news and press releases. Furthermore, the study explains the future opportunities and a sketch of key players involved in the Gene Therapy Markets hike in terms of revenue.

Every crucial and decisive detail for the development of the market and the restraining factors that may hamper the market in near future is mentioned with solutions in the report. Segmentations of the market are studied specifically to give profound knowledge for supplementary market investments.

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Some of the leading key players areAdmedus, Aziyo Biologics Inc., Coloplast Group, COOK BIOTECH INC, DSM Biomedical, Lattice Biologics Ltd., Medtronic, Merck KGaA, Darmstadt, MTF Biologics, and Smith & Nephew

The report aims to provide an overview of the Extracellular matrix market with detailed market segments by application, raw materials and geography. The global extracellular matrix market is expected to witnessing massive growth during the forecast period. The report provides important statistics on the market conditions of the leading extracellular matrix market players and offers key trends and opportunities in the market.

The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global extracellular matrix patches market based on various segments. It also provides market size and forecast estimates from year 2017 to 2027 with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The extracellular matrix patches market by each region is later sub-segmented by respective countries and segments. The report covers analysis and forecast of 18 countries globally along with current trend and opportunities prevailing in the region.

The report analyzes factors affecting extracellular matrix patches market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the extracellular matrix patches market in these regions.

Market Segmentation:

The global extracellular matrix market is segmented on the basis of application and raw material.

Based on application, the market is classified as vascular repair and reconstruction, dural repair, wound healing, cardiac repair, pericardial repair and soft tissue repair.

Based on raw material, the extracellular matrix market is divided as bovine, porcine and other raw material.

Geographically, the global market fragmented across several regions such as North America, Latin America, Asia-Pacific, Africa, and the Middle East on the basis of different business verticals. In the era of developing and developed countries Extracellular Matrix Market are holding the highest share. A clear picture of these market has been provided by presenting the market with distinctive info graphics.

North America is expected to hold significant share in the Extracellular Matrix market, owing to favorable reimbursement and regulatory policies in the United States. The demand for Extracellular Matrix in Asia Pacific is anticipated to grow at a steady rate during the forecast period, due to increased healthcare expenditure in China and India.

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This report studies comprehensively and discusses the prominent features of the Extracellular Matrix market in terms of its structure, landscape, demand factors, challenges and very significantly, the estimated market performance.

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Scrutinize in-depth global market trends and outlook coupled with the factors driving the market, as well as those hindering it.

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Biggest Innovations In Gene Therapy Market Technological Advancement, Emerging Trends And Forecast To 2025 Including Top Key Players Sangamo...