Archive for the ‘Gene Therapy Research’ Category

The market research firm Facts and Factors have recently added the latest study on, titled "Cancer Gene Therapy Market By Type (Ex-vivo and In-vivo) and By Product (Viral Vectors, Non-viral Vectors, and Others): Global Industry Outlook, Market Size, Business Intelligence, Consumer Preferences, Statistical Surveys, Comprehensive Analysis, Historical Developments, Current Trends, and Forecasts, 20202026", consisting of 190+ research pages which examine the overview of the various factors enabling growth and trends in the global industry.

This report is well crafted with a combination of the crucial data associated with the worldwide market, along with key factors liable for the demand for its services and merchandise. The report highlights the newest technological developments and new launches that assist our customers to set up their future-based potential products, make wise business selections to meet the projected demand ratio.

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

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Vigene Biosciences, Sirion Biotech, Bluebird bio, Cellectis, Ziopharm, Cobra, Uniqure, Finvector, Sarepta Therapeutics

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

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

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

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

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

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The 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 the US, Asia Pacific, Middle East, and Europe. Various aspects such as production capability, demand, product value, material parameters and specifications, distribution chain and provision, profit and loss, are explained in a comprehensive way in the Cancer Gene Therapy market report.

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

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

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

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Cancer Gene Therapy Market CAGR Status, Segmentation by Revenue, Gross margin, Analysis, Research, Forecast - Northwest Trail

A new coronavirus was spreading in the U.S., and with social distancing orders going into place, talk immediately turned to finding a treatment that could stop the spread.

In Maryland, companies jumped into action. Work began on a vaccine at Gaithersburg-based Novavax, and the company partnered with Emergent Biosolutions on initial development and manufacturing. Gaithersburgs Altimmune is also developing a vaccine, and announced plans last week for a preclinical testing collaboration with the University of Alabama Birmingham.

In all, Maryland Tech Council President Marty Rosendale now counts 28 Maryland companies working on the response in some form, whether they are developing new vaccines, therapeutics and tests, or providing lab space and clinical expertise thats key to bringing those forward. And a collaborative spirit is taking root.

This is about getting the job done, said Rosendale, who is connecting companies whether they are members of his organization or not. This is about making sure that we build these vaccines and diagnostics and get them cleared by the FDA as fast as we can and get out there and serve the public.

As the race against the virus is on among scientists and CEOs, its worth it for the rest of us to take a moment to remember that the ability to spin up operations that can develop life-saving treatments doesnt get built overnight. That manufacturing center from Emergent Biosolutions in Baltimores Bayview? It was already established as one of three federal centers to make new products to stop public health threats, and doubled in size in 2017.

With the need for years of research, lab space and stringent requirements to meet safety since theyre creating things that are put into peoples bodies, life sciences and biotech work takes longer to roll up to the general public. Equally, the region is home to work in biotech and life sciences that has developed over decades, and it has been gaining more and more influence in recent years.

Its also at the center of some of the most groundbreaking work to advance human ingenuity in the region and save lives. And, economically, its been home to some of the biggest news about M&A activity, venture capital and job growth.

When it comes to growing industries, the life sciences sector is among the most impactful in the area. Spanning hospital research labs and manufacturing centers where production takes place, harnessing living cells to create new treatments and other biotech, biomedical and pharma work is happening in a footprint that knits together Baltimore, the Maryland suburbs, D.C. and Northern Virginia.

Its an area that is home to key assets. That includes federal government institutions where research needs are identified such as the Bethesda-based National Institutes of Health, the White Oak-based U.S. Food and Drug Administrationand the Gaithersburg-based National Institute of Standards and Technology (NIST), while Frederick-based Fort Detrick is home to a base of biomedical work.

Funding from those institutions flows to universities such as Johns Hopkins Universityand the University of Maryland, where experts and faculty advance research. The response to the crisis offered an example of the leadership role, as noted Hopkins infectious disease expert Arturo Casadevall is leading a collaboration among research centers to explore the potential use of blood plasma from COVID-19 survivors as a therapeutic. The effort was funded to the tune of $4 million by Bloomberg Philanthropies and the State of Maryland, officials said in late March. By last week, the FDA approved a clinical trial that will allow researchers to tell whether the plasma is effective in protecting healthcare workers and first responders.

We are very fortunate that Maryland has some of the top health research facilities in the world, and I am confident in our states ability to be a leader in developing treatments and perhaps even a vaccine for COVID-19, Gov. Larry Hogan said in a statement issued when the announcement was made.

Emergent, too, is working on a plasma-derived therapy that collects donations of plasma from people who recovered. On April 2, the company was awarded $14.5 million from the U.S. Biomedical Advanced Research and Development Authority and was entering into a formal partnership to advance its treatment for severe hospitalized patients and at-risk people. Going forward, NIHs National Institute of Allergy and Infectious Diseases has agreed to incorporate the candidate into future clinical studies of COVID-19 treatments.

Though it is applying to a new coronavirus, the companys work on plasma therapy didnt just start. It is drawing on 40 years of experience working with plasma-derived therapies, said CEO Robert G. Kramer Sr.

Universities also help to get those discoveries to the public, establishing patents and nurturing startups and helping hospital systems offer places to gain new insight in the clinic. And at a collection of publicly traded companies born inside the state and big firms that moved here like GSK and AstraZeneca, work goes on to turn innovations hatched at research labs into products that can be made available widely to the public.

The biohealth industry is one of the top industrial clusters and very important to the Maryland economy, said Richard A. Bendis, president and CEO of Rockville-based BioHealth Innovation Inc.

The collective activity has already identified a name: the BioHealth Capital Region. And theres an ambitious goal: to be one of the top three hubs in the nation for biohealth by 2023. (For context, it was #4 last year, according to a ranking released by GEN.)

But even in the shorter term, there are signs of a new wave of activity thats having an impact everywhere in one metric that matters locally on the ground: job growth. Specifically, thats playing out as companies seek the facilities to make products in the state, and the people who can run the operation.

As the Maryland life sciences industry matures and moves further into clinical development and commercialization, the demand for manufacturing is growing, Rosendale said.

One of the largest exits of the decade arrived heralded from the sector in April 2019, when Catalent acquired Baltimore-based Paragon Bioservices. The $1.2 billion deal came just days after Paragon opened a new manufacturing center near BWI and made big plans to grow hundreds of jobs locally, and the newly branded Paragon Gene Therapy has continued to hire there and at other facilities like its University of Maryland BioPark HQ.

The same month, GileadsKite Pharma announced plans of its own to create jobs in the region with a forthcoming biologics manufacturing facility in Frederick County.

Longtime companies in the area have also opened their own manufacturing facilities. Gaithersburg-based Emergent Biosolutions has a pair of plants in Baltimore where it makes vaccines and recently expanded, while Precigen (formerly Intextron) opened its own gene and cell therapy-focused space in Germantown last year.

The maturity can also be measured in investment: Frederick-based Rooster Bio and BioFactura each made plans to ramp up production with second rounds of funding.

At the same time, Rosendale said, the next generation of life science companies are now beginning to move out of the incubators and into clinical development. Viela Bio spun out of longtime regional stalwart MedImmune (owned by AstraZeneca) and made a splash with a debut on the public market in 2019.

And there are signs that it makes sense to plant seeds for future growth in facilities like the two-year-old Johns Hopkins Fast Forward 1812 in East Baltimore and LifeBridge Health Bioincubator at Sinai Hospital in North Baltimore, as well as plans for a new BioPark building along MLK Boulevard in Southwest Baltimore and the forthcoming JLABS space from Johnson & Johnson Innovation and the Childrens National Health System in D.C.

The wave of growth has brought a job dynamic that has folks looking to fill roles, as well as orders.

Unemployment in the sector is at an all-time low, while the demand for skilled workforce in the sector continues to grow, Rosendale said.

Life sciences is often listed alongside cybersecurity as a growing industry in the state, and they face similar situations when it comes to workforce needs.

With more and more companies advancing their pipelines and seeing success, there is demand across the life science continuum for talent, said Chris Frew, CEO of BioBuzz Media and Workforce Genetics. Especially in the later stage clinical and commercialization fields. Biomanufacturing is in great demand and there are lots of jobs, process engineers are also in demand and hard to find.

The state draws bonafides in the industry from a large cluster of Ph.D.s. And, to be sure, theres room for highly educated experts to have a role in shaping a company. Bendis said tighter alignment between industry and academia to shape curricula and more education on what jobs are available for postdocs who want to stay in the region can help.

And when it comes to the demands of processing and manufacturing, educated folks are needed at many levels.

The running joke with my colleagues is if you can tell the difference between a eukaryote and prokaryote, youre hired, Ben Woodard, who leads an effort to train students at the Biotechnology Research and Education Program at the University of Maryland in College Park, told us prior to the pandemic in early March. (The program is currently working to roll out remote offerings since most lab research at the university has been halted). There are more jobs than people and the ultimate goal is to help healthcare.

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The DMV region's life sciences sector jumped into the fight against COVID-19. Here's why it could move quickly - Technical.ly

CAMBRIDGE, Mass., April 03, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today provided an update on the RESTORE-1 clinical trial of VY-AADC (NBIb-1817) for Parkinsons disease. Voyager and its collaborator Neurocrine Biosciences have temporarily paused screening of new patients in the RESTORE-1 trial to allow clinical trial sites to assess the impact of the COVID-19 pandemic on both the implementation of previously disclosed protocol amendments and on the safety of study participants. The companies plan to resume patient screening and enrollment as soon as the assessment is completed and Neurocrine, as the study sponsor, and Voyager, in consultation with clinical sites, determine that screening can safely and effectively resume. Voyager and Neurocrine Biosciences continue preparations for the initiation of the RESTORE-2 registrational study in Parkinsons disease planned for the second half of 2020.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly-owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, a monogenic form of ALS called SOD1, Friedreichs ataxia, Alzheimers disease, and other neurodegenerative diseases related to defective or excess aggregation of tau and alpha-synuclein proteins in the brain. Voyager has strategic collaborations with AbbVie and Neurocrine Biosciences. Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience, Voyager is headquartered in Cambridge, Massachusetts. For more information, please visit http://www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, plan, anticipate, believe, estimate, undoubtedly, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the assessment of the impact of the COVID-19 pandemic on the RESTORE-1 and RESTORE-2 trials, the resumption of screening and enrollment in the RESTORE-1 trial and planned initiation of the RESTORE-2 trial. All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. These forward-looking statements are subject to risks and uncertainties, including the risk that the assessment of the impact of the COVID-19 pandemic impact on the RESTORE-1 and RESTORE-2 trials may not be completed or may be substantially delayed, the risk that the consequences of the COVID-19 pandemic may not allow Neurocrine Biosciences to resume screening and enrollment of patients for the RESTORE-1 trial or to commence the RESTORE-2 trial, the risk that the impact of the COVID-19 pandemic on the RESTORE-1 and RESTORE-2 trials could affect Voyagers existing collaboration with Neurocrine Biosciences, or the risk that the COVID-19 pandemic could affect Voyagers ability to develop and commercialize products currently in Voyagers research and development pipeline. These statements are also subject to a number of material risks and uncertainties that are described in Voyagers Annual Report on Form 10-K filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investors: Paul CoxVP, Investor Relations917-754-0207 pcox@vygr.com

Media: Sheryl Seapy W2Opure949-903-4750 sseapy@purecommunications.com

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Voyager Therapeutics Provides Update on the RESTORE-1 Parkinson's Disease Trial - Associated Press

BioMarin Pharmaceutical Inc. BMRN announced that it is planning to file regulatory applications in the third quarter of 2020, seeking approval for achondroplasia candidate, vosoritide, in the United States and Europe following its meetings with health authorities.

The regulatory applications will be filed based on data from successfully completed phase III study, which evaluated vosoritide as a treatment for achondroplasia, the most common form of disproportionate short stature in humans or dwarfism, in children aged 5 to 14 for 52 weeks. In December 2019, BioMarin announced positive top-line data from the study, which showed that patients treated with the candidate achieved placebo-adjusted change from baseline in growth velocity of 1.6 cm/year after treatment duration of one year.

The filings will be further supported by long-term safety and efficacy from a phase II study and ongoing extension studies on vosoritide in dwarf children.

A potential approval will make the drug the first and only approved treatment for achondroplasia in the United States and Europe. BioMarin estimates that around 25,000 children suffer from this disorder in its commercial territories, which represents decent sales growth opportunity.

Meanwhile, Ascendis Pharma ASND is developing an achondroplasia candidate in a phase II study.

BioMarins shares have declined 2.1% so far this year compared with the industrys decrease of 11.6%.

The companys key drugs, Vimizim and Kuvan, enjoy strong demand trends, which drove its revenues higher in 2019. Its newest product, Palynziq is also witnessing strong commercial uptake in the United States.

Apart from vosoritide, the company has a strong rare disease pipeline with focus on developing gene therapies.

BioMarins lead gene therapy candidate, valoctocogeneroxaparvovec (valrox) is under review in the United States and Europe as a treatment for severe hemophilia A. Decisions from the concerned regulatory authorities are expected in the second half of 2020.

A phase I/II study is evaluating another gene therapy candidate, BMN-307, as a treatment for phenylketonuria (PKU). Please note that Kuvan and Palynziq are also approved for treating PKU.

The companys PKU franchise generates the majority of the companys revenues. Another drug will likely boost the sales of this franchise.

Meanwhile, Kuvan is set to face generic competition as early as October 2020. This will likely dent the companys revenues in 2021. A few other companies are also developing treatments for PKU in early-stage studies, which include Synlogics SYBX small molecule candidate, SYNB1618; Homology Medicines FIXX gene therapy, HMI-102; and Rubius Therapeutics red cell therapeutic candidate, RTX-134. Potential approval to vosoritide may partially offset loss in Kuvan sales.

BioMarin Pharmaceutical Inc. Price

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Biomarin to File Regulatory Applications for Vosoritide in Q3 - Yahoo Finance

WESTBURY, N.Y., April 6, 2020 /PRNewswire/ --BioIVT, a leading provider of research models and services for drug and diagnostic development, today announced the opening of its new blood donor center on the Tufts University campus in Medford, MA to support academic and pharmaceutical researchers involved in COVID-19, cell and gene therapy research.

"BioIVT wants to play a leading role in supporting COVID-19 research efforts and blood donations are a vital resource for the research and development of new therapies, vaccines, and diagnostics. We have many years' experience developing blood products, including blood-derived immune cells for cell and gene therapy research, and we want to make that expertise count," said BioIVT CEO Jeff Gatz. "Researchers recognize and appreciate BioIVT's rapid response and commitment to high quality, fresh blood products and this new donor center will allow us to offer those attributes and services to additional US clients."

BioIVT's new Boston blood donor center is its seventh. The company has similar facilities located in California, Tennessee and Pennsylvania to serve US clients and in London, UK for EU-based clients.

"While the initial focus at our Boston donor center will be on delivering fresh blood, leukopaks and buffy coats within hours of collection, we plan to add more capabilities and donors over time," said Jeff Widdoss, Vice President of Donor Center Operations at BioIVT.

Leukopaks, which contain concentrated white blood cells, are used to help identify promising new drug candidates, assess toxicity levels, and conduct stem cell and gene therapy research. They are particularly useful for researchers who need to obtain large numbers of leukocytes from a single donor.

BioIVT blood products can be supplied with specific clinical data, such as the donor age, ethnicity, gender, BMI and smoking status. Its leukopaks are also human leukocyte antigen (HLA), FC receptor and cytomegalovirus typed. HLA typing is used to match patients and donors for bone marrow or cord blood transplants. FC receptors play an important role in antibody-dependent immune responses.

COVID-19-related Precautions Blood donor centers are considered essential businesses and will remain open during the COVID-19 quarantine. BioIVT is taking additional safety measures to protect both blood donors and its staff during this difficult time. It has instituted several social distancing measures, including increasing the space between chairs in the waiting room and between donor beds, and limiting the entrance of non-essential personnel. The screening rooms are disinfected between donors and all areas of the center continue to be cleaned at regular intervals.

As soon as each blood donor signs their informed consent form, their temperature is taken. If they have a fever, their appointment is postponed, and they are referred to their physician. Any donor who develops COVID-19 symptoms after donating blood is required to inform the center immediately.

All BioIVT blood collections are conducted under institutional review board (IRB) oversight and according to US Food and Drug Administration (FDA) regulations and American Association of Blood Banks (AABB) guidelines.

Those who would like to donate blood at BioIVT's new Boston-area donor center should call 1-833-GO-4-CURE or visit http://www.biospecialty.com to make an appointment.

Further information about the products available from BioIVT's new donor center can be found at https://info.bioivt.com/ma-donor-ctr-req.

About BioIVTBioIVT is a leading global provider of research models and value-added research services for drug discovery and development. We specialize in control and disease-state biospecimens including human and animal tissues, cell products, blood and other biofluids. Our unmatched portfolio of clinical specimens directly supports precision medicine research and the effort to improve patient outcomes by coupling comprehensive clinical data with donor samples. Our PHASEZERO Research Services team works collaboratively with clients to provide target and biomarker validation, phenotypic assays to characterize novel therapeutics, clinical assay development and in vitro hepatic modeling solutions. And as the premier supplier of hepatic products, including hepatocytes and subcellular fractions, BioIVT enables scientists to better understand the pharmacokinetics and drug metabolism of newly-discovered compounds and their effects on disease processes. By combining our technical expertise, exceptional customer service, and unparalleled access to biological specimens, BioIVT serves the research community as a trusted partner in elevating science. For more information, please visit http://www.bioivt.com or follow the company on Twitter @BioIVT.

BioIVT Contact: Courtney Noah, SVP, Marketing & Client Services, 516-483-1196Media Contact: Lisa Osborne, Rana Healthcare Solutions, 206-992-5245, [emailprotected]

SOURCE BioIVT

http://www.bioivt.com

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BioIVT Opens New Blood Donor Center to Support Boston-area Research into COVID-19 Therapies, Vaccines and Diagnostics - PRNewswire

Cancer Gene Therapy Market size is expected to exceed USD 2.5 billion by 2025. Rising cancer prevalence across the globe will positively impact the cancer gene therapy market growth.

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Some of the major industry players include: Sirion Biotech, Vigene Biosciences, bluebird bio, Ziopharm, Cellectis, Cobra, Finvector, Uniqure, Sarepta Therapeutics, Caribou among others. These industry players adopt several strategic initiatives to maintain their market position.

Technological advancements in the biotechnology sector have led to developments and innovations in cancer gene therapy. Recently developed cancer therapies have been incorporated with genetically modified genes that blocks the growth of tumor. Moreover, efficient PCR technology and isothermal amplification technologies have been introduced that transformed the way of detecting mutations in the genes. Moreover, introduction of CRISPR gene editing tools have modified the process of developing gene therapy. Hence, advancements in technology has ensured availability of advanced cancer gene therapy that will boost the industry growth. However, high cost of cancer gene therapy may impede its demand, thereby restraining the industry growth to certain extent.

Type segment of cancer gene therapy market includes ex-vivo and in-vivo. In-vivo segment is projected to experience around 22% growth throughout the analysis timeframe owing to various advantages associated with it. In-vivo gene therapy involves direct delivery of therapeutic gene into the target cell and has shown effective results in treatment of cancer. The viral vectors that are delivered utilizing in-vivo gene therapy help in inhibiting the activity of tumor inducing genes and has also shown positive results during clinical trials. Aforementioned factors will elevate the segmental growth.

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Products of cancer gene therapy include viral vectors and non-viral vectors. Viral vectors segment was valued over USD 310 million in 2018. Viral vectors are highly preferred during gene transfer process as they have showcased high transfection efficiency. Viral vectors possess safety profile as compared to other vectors. Moreover, viral vectors expresses the desired antigen in accurate conformation enhancing the immune system.

End-users of cancer gene therapy are biopharmaceutical companies, research institutes and others. Biopharmaceutical companies segment accounted for around 48% revenue share in 2018 and is expected to experience exceptional growth in the forthcoming years. Companies such as Novartis and Roche are developing cancer gene therapies that have high adoption of viral as well as non-viral vectors, thereby proving beneficial for the segmental growth. These companies also conduct clinical trials that raises the demand for vectors, hence fostering the segmental growth.

China cancer gene therapy market will experience over 26% CAGR throughout the forecast years. Significant country growth can be attributed to the rising awareness regarding the availability of advanced therapies for treating cancer. Furthermore, increasing government initiatives and funds motivate the researchers and scientists for carrying out extensive research activities associated with cancer gene therapy that will positively influence the country growth. Above mentioned factors coupled with increasing prevalence of cancer will further stimulate the industry growth.

Cancer gene therapy industry is dominated by few major players. Cancer gene therapy industry is still in the developing phase, therefore, players involved in thie market focus on integrating advanced technology to promote developments in the therapies. The players also implement certain strategic initiatives such as merger, acquisitions and product launches for acquiring competitive advantage. For instance, in 2013, Celgene and bluebird bio collaborated to introduce innovations in gene therapies. Such collaborations will provide both the companies to gain competitive advantage over others.

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Cancer Gene Therapy Market trends research and projections - GroundAlerts.com

Below, we look at 14 stocks and partnerships that are trying to produce a vaccine, treatment or diagnostic test for the COVID-19. All of them have seen their share prices experience sharp and volatile movements as the coronavirus has grown into a pandemic.

Although investors have got excited by the prospect of investing in a game-changing breakthrough, not all of them will succeed and will see their price fall back down as their valuations become over-stretched.

For example, London-listed Synairgen has risen over 750% since the start of the year, while Inovio Pharmaceuticals, Novavax, Vaxart and Vir Biotechnology have all more than doubled in value. On the other hand, some big-name stocks fighting the coronavirus have seen their share prices slump as the outbreak worsens, such as Pfizer, GlaxoSmithKline and Johnson & Johnson, all of which could rise if they are successful in their efforts.

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Trading a stock allows you to speculate on the future share price movement of a stock, allowing you to take a position on whether you believe it will fall (going short) or will rise (going long). You do not own the underlying shares and wont receive any dividends, but you can use leverage. This can be done using either an IG CFD or spread betting account.

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What stocks are trying to make a coronavirus vaccine? - IG

Takeda Pharmaceutical and a subsidiary of Germany-based Evotec SE forged a long-term research collaboration that will support the Japanese companys growing number of research stage gene therapy discovery programs.

Evotec GT, an Austria-based subsidiary of Evotec SE, will support multiple Takeda programs that target conditions aligned with the Japanese pharma companys four core therapeutic areas in oncology, rare diseases, neuroscience and gastroenterology. Evotec GT will use its growing gene therapy capabilities and drug discovery platform to support the research. No financial details were announced as part of the collaboration and neither Takeda nor Evotec gave any hints about the scope of the project, nor did they hint at any particular targets the companies would go after.

The collaboration is an expansion of a partnership the two companies struck last year to develop at least five drug discovery programs across a broad range of indications.in that deal, worth more than $850 million, Evotec will use its platform to validate development theories and advance small molecule programs along Takedas core therapeutic areas.

Craig Johnstone, chief operating officer of Evotec, said the latest collaboration expands the scope of the companys relationship with Takeda into gene therapy. He said the alliance with Takeda is a demonstration of the value of our multimodality platform with innovative technologies and best-in-class execution for addressing the most urgent requirements of our partners. Evotecs operations cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development. Johnstone added that relationships like the one with Takeda will transform the industrys approach to discovering and developing new therapies for devastating diseases.

We are excited to be broadening and expanding our discovery efforts with the Evotec team. Gene therapy is a growing therapeutic approach in our portfolio and this alliance with Evotec will help us further accelerate our delivery of transformative therapies for patients, particularly those with rare diseases, Steven Hitchcock, global head of research at Takeda, said in a statement.

For Evotec, the expanded deal with Takeda comes a few days after the company struck a licensing and development agreement with Canadas panCELLa Inc. Under the terms of that agreement, Evotec licensed panCELLas proprietary iPS cell lines iACT Stealth Cells, which are genetically modified to prevent immune rejection of derived cell therapy products. Evotec will also have access to a new-generation cloaking technology known as hypoimmunogenic cells. Evotec intends to use the cell lined to develop iPSC-based, off-the-shelf cell therapies with long-lasting efficacy that can be safely administered to a broad population of patients without the use of medication to suppress a patients' immune system.

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Takeda and Evotec Enter into Gene Therapy Research Alliance - BioSpace

Located on the Tufts University Medford, MA campus, this new donor center will enable delivery of fresh blood, leukopaks and buffy coats for COVID-19, cell and gene therapy research within hours of collection

WESTBURY, N.Y., April 6, 2020 /PRNewswire/ --BioIVT, a leading provider of research models and services for drug and diagnostic development, today announced the opening of its new blood donor center on the Tufts University campus in Medford, MA to support academic and pharmaceutical researchers involved in COVID-19, cell and gene therapy research.

"BioIVT wants to play a leading role in supporting COVID-19 research efforts and blood donations are a vital resource for the research and development of new therapies, vaccines, and diagnostics. We have many years' experience developing blood products, including blood-derived immune cells for cell and gene therapy research, and we want to make that expertise count," said BioIVT CEO Jeff Gatz. "Researchers recognize and appreciate BioIVT's rapid response and commitment to high quality, fresh blood products and this new donor center will allow us to offer those attributes and services to additional US clients."

BioIVT's new Boston blood donor center is its seventh. The company has similar facilities located in California, Tennessee and Pennsylvania to serve US clients and in London, UK for EU-based clients.

"While the initial focus at our Boston donor center will be on delivering fresh blood, leukopaks and buffy coats within hours of collection, we plan to add more capabilities and donors over time," said Jeff Widdoss, Vice President of Donor Center Operations at BioIVT.

Leukopaks, which contain concentrated white blood cells, are used to help identify promising new drug candidates, assess toxicity levels, and conduct stem cell and gene therapy research. They are particularly useful for researchers who need to obtain large numbers of leukocytes from a single donor.

BioIVT blood products can be supplied with specific clinical data, such as the donor age, ethnicity, gender, BMI and smoking status. Its leukopaks are also human leukocyte antigen (HLA), FC receptor and cytomegalovirus typed. HLA typing is used to match patients and donors for bone marrow or cord blood transplants. FC receptors play an important role in antibody-dependent immune responses.

COVID-19-related Precautions Blood donor centers are considered essential businesses and will remain open during the COVID-19 quarantine. BioIVT is taking additional safety measures to protect both blood donors and its staff during this difficult time. It has instituted several social distancing measures, including increasing the space between chairs in the waiting room and between donor beds, and limiting the entrance of non-essential personnel. The screening rooms are disinfected between donors and all areas of the center continue to be cleaned at regular intervals.

As soon as each blood donor signs their informed consent form, their temperature is taken. If they have a fever, their appointment is postponed, and they are referred to their physician. Any donor who develops COVID-19 symptoms after donating blood is required to inform the center immediately.

All BioIVT blood collections are conducted under institutional review board (IRB) oversight and according to US Food and Drug Administration (FDA) regulations and American Association of Blood Banks (AABB) guidelines.

Those who would like to donate blood at BioIVT's new Boston-area donor center should call 1-833-GO-4-CURE or visit http://www.biospecialty.com to make an appointment.

Further information about the products available from BioIVT's new donor center can be found at https://info.bioivt.com/ma-donor-ctr-req.

About BioIVTBioIVT is a leading global provider of research models and value-added research services for drug discovery and development. We specialize in control and disease-state biospecimens including human and animal tissues, cell products, blood and other biofluids. Our unmatched portfolio of clinical specimens directly supports precision medicine research and the effort to improve patient outcomes by coupling comprehensive clinical data with donor samples. Our PHASEZERO Research Services team works collaboratively with clients to provide target and biomarker validation, phenotypic assays to characterize novel therapeutics, clinical assay development and in vitro hepatic modeling solutions. And as the premier supplier of hepatic products, including hepatocytes and subcellular fractions, BioIVT enables scientists to better understand the pharmacokinetics and drug metabolism of newly-discovered compounds and their effects on disease processes. By combining our technical expertise, exceptional customer service, and unparalleled access to biological specimens, BioIVT serves the research community as a trusted partner in elevating science. For more information, please visit http://www.bioivt.com or follow the company on Twitter @BioIVT.

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BioIVT Opens New Blood Donor Center to Support Boston-area Research into COVID-19 Therapies, Vaccines and Diagnostics - Yahoo Finance

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

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

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

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

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

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

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Gene Therapy in Oncology Market Share Analysis and Research Report by 2025 - Express Journal

WRAL TechWire keeps tabs on the latest and greatest meetups, panels, workshops, conferences, application deadlines and all things happening in the North Carolina startup/tech world. The Headliners is a multi-part weekly roundup of upcoming events to add to your calendar.

Below is a list of April events in Raleigh, Durham, Chapel Hill and the greater Triangle area. To find out whats happening this month in cities outside of the Triangle, check out part two of the Headliners column. Another post highlights events in May. Meetups that occur regularly are listed here.

If youd like your event to be included, feel free to send me an email.

Also, check out a full range of events on TechWires interactive calendar, along with our comprehensive resource guide for startups in the Triangle.

Note: The following list is our lineup of Triangle events through the end of Aprilmost have been switched to a virtual format in light of social distancing recommendations.

UNC Kenan-Flagler Business Schools Entrepreneurs Lab series features lectures revealing the secrets behind successful entrepreneurs. Chris LeGrand and Bill Spruill will speak this week.

Wake Forest University is hosting a virtual panel with experts discussing climate solutions in the state of North Carolina.

Join this event to see first-hand software demonstrations from the latest cohort of engineers in Project Shifts immersive full-stack development program.

Join the Code for Chapel Hill meetup to network with like-minded individuals and work on civic hacking projects. Meetings are held every two weeks on Tuesdays.

1 Million Cups, presented by Kauffman, is a weekly informal pitch event for the startup community. Join for free coffee and entrepreneurial support as local startups deliver their presentations.

In this webinar, NC TECH will facilitate a discussion with NC State economist Dr. Michael Walden on the economic impact of COVID-19.

In this virtual workshop, participants will collaborate with others in imagining their future and mapping out a plan to make their ideas come to fruition.

Allstacks is hosting a webinar covering how software developers and product leaders can use predictive forecasting and risk management solutions to meet their goals.

Join the Research Triangle Cleantech Cluster and regional thought leaders for a webinar on how energy markets are responding to COVID-19, how businesses are adapting, and what the energy sector may look like in a post-pandemic world.

LaunchBios Larger Than Life Science event (usually held in Durham) is going virtual, allowing life science entrepreneurs from anywhere in the world to join and hear from experts discussing the latest research and innovations in their fields. Join this webinar to learn about gene therapy technology.

LaunchBios Larger Than Life Science event (usually held in Durham) is going virtual, allowing life science entrepreneurs from anywhere in the world to join and hear from experts discussing the latest research and innovations in their fields. Join this webinar to learn about bringing gene therapy products to market.

Join Lucid Dream for a webinar on how remote teams can use VR to adapt to a new virtual business environment.

Capitol Broadcasting Company and WRAL are Here to Help our local businesses. Tune in to get advice from industry experts and hear ideas from other local business owners. More coverage here.

Join Raleigh Chamber to hear from experts on accounting and employment law. The speakers are Kevin Ceglowski of Poyner Spruill, LLP and Marshall Stein of Elliott Davis.

During this virtual event, UNC Gillings School of Global Public Health students will deliver 10-15 minute TEDx-style presentations to highlight inspiring projects, findings and experiences they have had in advancing global health.

For its next meeting (held virtually), AITP-RTP will host Scott Gerard for a discussion on important AI concepts and applications.

This free bi-monthly event offers a space for local tech professionals to build connections and find potential job opportunities.

This weekly meetup brings together developers, IT professionals and tech enthusiasts who are interested in the Google Cloud Platform.

During this event, students will gather to pitch their ventures before a panel of judges, receive feedback and celebrate the end of their work at BLUE.

The Duke Incubation Fund provides funding for innovative projects that demonstrate potential for future financial support, company formation licensing and not-for-profit partnering.

In a series of online sessions, Momentum Learnings seventh cohort of Immersive Web Development students will present the projects theyve created during the 12-week full-stack web development program.

In this webinar, reacHIRE CEO Addie Swartz will share how companies can increase engagement, community and connection among early-career women.

Code for Durham brings together technologists, designers, developers, data scientists, map makers and activists to collaborate on civic technology projects. Meetings are held every two weeks on Tuesdays. Pizza will be provided.

All Things Open is now seeking talks from technologists to be featured at the 2020 conference, set for October 18-20 in Raleigh.

1 Million Cups, presented by Kauffman, is a weekly informal pitch event for the startup community. Join for free coffee and entrepreneurial support as local startups deliver their presentations.

In this webinar, the Institute for Emerging Issues is bringing together representatives from Service Year organizations, nonprofits and government agencies to discuss the innovative ways in which communities are using service years to tackle problems and add opportunities.

Join this webinar to hear from Duke University Rubenstein Fellow Eric Perakslis on how health systems are using informatics and technology during pandemic outbreaks.

The Regional Transportation Alliances Innovations and Solutions Forum series will highlight new innovations to improve intersections, freeways and transit systems.

Hosted by the Carolina Small Business Development Fund, this webinar will cover the many funding opportunities and other resources for small businesses that are affected by the COVID-19 pandemic.

NC IDEA LABS is a four-week customer discovery program that helps idea-stage entrepreneurs take their first steps toward building a successful startup. The next cohort will run virtually from May 26 to June 19.

LaunchBios Larger Than Life Science event (usually held in Durham) is going virtual, allowing life science entrepreneurs from anywhere in the world to join and hear from experts discussing the latest research and innovations in their fields. Join this webinar to learn about the work behind a coronavirus vaccine.

The Regional Transportation Alliances Innovations and Solutions Forum series will highlight automated mobility and connected transportation.

In this months C-Suite Perspectives, WakeMed Health & Hospitals CEO Donald Gintzig will discuss the characteristics that make a great leader.

This free monthly interactive webinar provides participants with an overview of NC TECHs activities, resources and member offerings.

This weekly meetup brings together developers, IT professionals and tech enthusiasts who are interested in the Google Cloud Platform.

This monthly webinar series hosts a global health professional who will share insights and advice for advancing your career. This months webinar features Andrew Herrera, executive director of Curamericas.

NC TECHs Government Vendor Network is a forum for member companies who are interested in doing business with state government.

UNC Kenan-Flagler Business Schools Entrepreneurs Lab series features lectures revealing the secrets behind successful entrepreneurs. Amy Cosper and Sophia Woo will speak this week.

Join the Code for Chapel Hill meetup to network with like-minded individuals and work on civic hacking projects. Meetings are held every two weeks on Tuesdays.

1 Million Cups, presented by Kauffman, is a weekly informal pitch event for the startup community. Join for free coffee and entrepreneurial support as local startups deliver their presentations.

The Research Triangle Cleantech Cluster and Joules Accelerator have selected the latest cohort of the Cleantech Catalyst program. Join this webinar to meet the eight cleantech startups in the 2020 class.

For the third cycle of its Flash Grant program, the North Carolina Biotech Center is searching for innovative projects aiming to address the global coronavirus (COVID-19) outbreak, precision health, and digital and data-driven life science technologies.

Join this webinar to learn how you can benefit from the SBIR/STTR program to help small businesses commercialize their products and services.

This free bi-monthly event offers a space for local tech professionals to build connections and find potential job opportunities.

This weekly meetup brings together developers, IT professionals and tech enthusiasts who are interested in the Google Cloud Platform.

Code for Durham brings together technologists, designers, developers, data scientists, map makers and activists to collaborate on civic technology projects. Meetings are held every two weeks on Tuesdays. Pizza will be provided.

Bring your ideas and opinions to the next Midtown Techies meetup. Events are held on the last Tuesday of every month.

1 Million Cups, presented by Kauffman, is a weekly informal pitch event for the startup community. Join for free coffee and entrepreneurial support as local startups deliver their presentations.

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Triangle headliners: 48 webinars & events to add to your April calendar - WRAL Tech Wire

Q. You and Burns have worked together in the past. Could you describe the collaborative process of turning a book into a documentary?

A. First of all, its an incredible collaboration. When Ken and I launch a film we begin with talks about the book, both of us excited and aware that we are wading into the unknown. We take long walks through Central Park and ask questions outside the nitty gritty of the film: What is the mood of the film? What are the primary messages were trying to convey? I was lucky to work on The Gene with largely the same production team that adapted my first book, The Emperor of All Maladies: A Biography of Cancer, including the incredible filmmaker Barak Goodman (JRN86).

A film is different from a book. In The Gene we wereable to include a trove of historical footage and humanize the inspiring stories of patients and their families seeking cures for their genetic disease. People like Nancy Wexler, professor of neuropsychology at Columbias medical center, who has spent most of her life on an odyssey to find the gene for Huntingtons, a disease that killed her mother. Luke Rosen and Sally Jackson, parents on a tireless quest to raise awareness for their daughters rare degenerative disease. These personal stories help genetics come to life, but they also highlight how much we still do not know. I hope people will find the mood of our film somber, thoughtful and hopeful.

Q. For $200 a person can order a profile of his or her genome that provides ancestral information, as well as genetic health risks. Do you see this as a positive societal development?

A. The question you have to ask is do we want to live in a world where you can send a sample of saliva and find out that you have a 10 percent or 20 percent risk of developing breast cancer in the next 30 years. This information can be useful, motivating you to adopt more positive health behaviors. But it also marks you, changes you. It can change your relationship with yourself, your body. When you decide to test for future risk you are also, inevitably, asking yourself, what kind of future am I willing to risk?

Q. Could genetics play a role in how vulnerable a person is to contracting COVID-19, and whether that person is more at risk of dying from the illness?

A. This is one of the great mysteries of this infection. Young, healthy people are dying, even if most serious cases occur in the elderly and those with pre-existing conditions. There are multiple studies trying to unravel why some people infected with SARS-Cov2, the virus that causes COVID-19, fall seriously ill, while others show only mild or nonexistent symptoms.We are finding a correlation between high viral loadthe amount ofvirus present in any sample taken from a patientand more severe illness.

As I argued recently in The New Yorker, we have done a good job measuring the spread of the virus across populations, but it is now time we learn more about how SARS-Cov2 behaves in the body. This requires large-scale efforts to collect the DNA of people and the virus that they are infected with. One example of a study might be taking the DNA of those with serious underlying disease and comparing it to the DNA of those with mild or asymptomatic cases. We need to determine whether genetic variations among humans affect how susceptible individuals are to COVID-19 infections as rapidly as possible.

Q. What would you like audiences will take away from the film?

A. We hope The Gene will help people understand that the story of the revolution in genetics that is transforming medical science is also the story of what makes us who we are. Wed like to see the film spark a national conversation. The National Institute of Healths National Human Genome Research Institute, our outreach and education partner, is planning many activities. We are in conversations with people in cities across the country, including policymakers and science educators, right down to the primary school level, to take part in discussions and host screenings.

In the next few weeks NIH will launch an interactive digital platform that will go beyond the book and film, adding discussion of the COVID-19 pandemic. After you watch the film, please keep up with us on Twitter to learn more about these activities. Visit @DrSidMukherjee, @KenBurns and @WETA (our producing public media station). Stay tuned.

More here:
Siddhartha Mukherjee and Ken Burns Present PBS Premiere of 'The Gene: An Intimate History' - Columbia University

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Global Gene Therapy Market 2019 Valuable Growth Prospects and Upcoming Trends till 2025 - Curious Desk

TORONTO, April 02, 2020 (GLOBE NEWSWIRE) --

QuestCap Inc. (QuestCap or the Company) (CSE: QSC; OTCMKTS: COPRF; FRA: 34C1) is pleased to announce an investment of $1 million in the Sunnybrook Research Institutes COVID-19 research. With these funds, Sunnybrook will establish the Sunnybrook Translational Research Group for Emerging and Respiratory Viruses (SERV) and QuestCap will receive a 3.5% royalty on any revenues earned by Sunnybrook from the commercialization of any of the research done by SERV.

Led by infectious diseases physician and microbiologist Dr. Samira Mubareka, SERVs work will focus on three crucial streams of research: vaccines and therapeutics, virus biology and transmission prevention. Dedicated investment in SERV means Dr. Mubareka and her team will be able to accelerate the pace of further research into COVID-19 at a crucial time.

Dr. Lawrence Steinman and Mike McCarthy Appointed as Advisors

QuestCap is pleased to announce the appointment of Dr. Lawrence Steinman and Mike McCarthy to the board of advisors. Dr. Lawrence Steinman is Professor of Neurology, Neurological Sciences and Pediatrics at Stanford University and Chair of the Stanford Program in Immunology from 2001 to 2011.

Mike McCarthy has more than 14 years of experience with the Ontario Ministry of Health and Long-Term Care and 24 years in health policy and delivery. He was appointed Senior Policy Advisor to the Progressive Conservative Minister of Health of Ontario, advising on physician, nursing and primary care, public health, laboratories, OHIP, organ transplant, HIV and blood issues. In 2003, he provided strategic support and counsel to the government of Ontario during the SARS outbreak.

Lawrence Steinman stated that, The Sunnybrook technology will be a key tool for the development of both powerful and safe vaccines against COVID-19, and for the development of point of care and even home diagnostics to test whether one is immune to the COVID-19 virus. Those who are immune can return to work and can provide safe and needed duties to the community. It would be especially important to know if you are a health care worker, grocer or delivery person whether or not you have immunity. You could then be safe and carry on as a SuperWomen or SuperMan in these times, knowing you are immune! Mike McCarthy adds I look forward to advising QuestCap in its visionary efforts to bring innovative testing and vaccine technologies to Canada and the world to combat COVID-19.

The Company has granted 500,000 stock options to Mr. Steinman pursuant to the Companys stock option plan. The stock options shall vest in four equal instalments every three months such that all stock options fully vest by the date that falls 12 months from the date of grant and may be exercised at a price of $0.305 per common share for a period of five years from the date of grant. This grant of options is subject to the approval of the Canadian Securities Exchange.

Sunnybrook Translational Research Group

Sunnybrook has been at the global forefront of the COVID-19 pandemic since the first Canadian case was identified, both clinically and on the research front. Dr. Mubareka, along with clinical microbiologist Dr. Robert Kozak and a team of close collaborators, has led Sunnybrooks research response efforts since the earliest days of the pandemic. Their work has been globally significant, leading to early implementation of a hospital-based COVID-19 test. Most crucially, on March 10, 2020, their team isolated the agent responsible for the ongoing outbreak of COVID-19. The isolated virus is helping researchers in Canada and elsewhere to develop better diagnostic testing, treatments and vaccines, and gain a better understanding of the viruss biology, evolution and clinical shedding.

This is extremely time-sensitive research, says Dr. Mubareka. As a scientist on the frontlines, I have seen the devastation this virus can cause. We all feel the effects of physical distancing as our lives are fundamentally changed. This is why support from our community is so important right now. The more we understand how this virus behaves, the better we can limit its spread as we race to find a treatment or vaccine. This investment will be put to work immediately toward our human and operational funding needs to rapidly accelerate research into COVID-19.

Now is the time to dedicate as many resources as possible to stopping the spread of this highly contagious virus, explains Dr. Andy Smith, Sunnybrooks President and CEO. On behalf of Sunnybrook, I would like to extend my deepest thanks to QuestCap for stepping up with this inspiring investment. Your support will have a direct impact on the lives of countless people in our communities, across Canada and around the world.

With these crucial tools in hand, QuestCap saw an opportunity to give this research a major boost at a critical juncture. As countries grapple with the effects of COVID-19, there has been a collective global effort to help minimize the impacts of this deadly virus, says Stan Bharti, Co-Chairman of QuestCap. QuestCap, through its investment platform, is looking to do its part by providing a critical investment to help fund the vital research being conducted by Dr. Mubareka, Dr. Kozak and their teams.

The investment to Sunnybrook will help researchers in Canada and across the world develop better diagnostic testing, treatments and vaccines, and gain a better understanding of SARS-CoV-2 biology, evolution and clinical shedding for the development of commercial applications (Commercial Applications). As consideration for its investment, QuestCap will receive a 3.5% royalty on any revenue earned by Sunnybrook from the any Commercial Applications resulting from the research conducted by SERV.

At a time of global uncertainty as COVID-19 continues to devastate people, families and entire countries, the urgency of research has never been greater, says Dr. Kullervo Hynynen, Vice President of Research & Innovation at Sunnybrook. Yet, often times, there simply isnt enough funding for research. I am deeply grateful to QuestCap for this clear statement on supporting the power of research to save and change lives.

About the Research

SERV is a three-pronged COVID-19 research response program, consisting of the following critically important areas of focus:

1. Virus biology (genomics): Identifying the genetic sequence of a virus is an essential step for identifying key targets for treatments and vaccines. It also tells the story of origin and its relation to other viruses. This approach provides precision genomic data, which will be essential for outbreak investigation. Funds for SERV allow Sunnybrook scientists to turn around whole genome viral sequences at the site of care: Sunnybrook.

2. Vaccines and therapeutics: Our ability to identify and isolate the virus that causes COVID-19, based on samples from infected patients, allows us to collaborate on antiviral research and conduct our own vaccine and transmission work. With SERV, Sunnybrook will share our findings about the virus within the Canadian research and diagnostic community, thereby driving further innovative solutions to the COVID-19 pandemic.

3. Transmission prevention: As part of a World Health Organizations effort to evaluate risk to health-care workers of providing certain support measures, such as high-flow oxygen use, Sunnybrook is building a simulation space for live virus experiments using mannequins with funds for SERV. This is important because protecting care providers is key to preventing the spread of the virus within hospitals.

About Lawrence Steinman

Steinman is Professor of Neurology, Neurological Sciences and Pediatrics at Stanford University and Chair of the Stanford Program in Immunology from 2001 to 2011. His research focuses on antigen specific tolerance in autoimmune disease and in gene therapy for degenerative neurologic diseases. He has elucidated what provokes relapses and remissions in multiple sclerosis (MS). He is taking forward a pivotal clinical trial with antigen specific tolerization therapy for type 1 diabetes. He serves as attending neurologist at Stanfords Lucille Packard Childrens Hospital. Steinman is an elected member of the US National Academy of Sciences and the National Academy of Medicine.

Steinman was senior author on the 1992 Nature article that led to the drug Tysabri, approved for MS and Crohns disease. Tysabri has been taken by over 200,000 individuals with MS.

Dr. Steinman graduated from Dartmouth College, Magna Cum Laude in Physics. His MD is from Harvard Medical School. He was a post-doctoral fellow in chemical immunology fellow at the Weizmann Institute of Science. After neurology residency he remained on the faculty in 1980. He has received numerous honors, including the John M. Dystel Prize in 2004, the Javits Neuroscience Investigator Award from the NINDS twice, the Charcot Prize in MS research, and the Cerami Prize in Translational Medicine. Steinman is a member of both the National Academy of Sciences, and the National Academy of Medicine.

Dr. Steinman co-founded several biotech companies, including Neurocrine, Atreca, 180 Therapeutics, and Tolerion. He was a Director of Centocor from 1988 until its sale to Johnson and Johnson.

About Mike McCarthy

Mike is widely recognized across Canada as a stellar health care advocate and system expert. As a volunteer Vice-President of the Canadian Hemophilia Society, Mike was the national spokesperson for Canadians infected by blood tainted with Hepatitis C. His tireless efforts resulted in a landmark $2 billion settlement with the federal government in 2006, and the Queens Golden Jubilee Award presented by the Governor General of Canada.

Presently Mike is a Principal at Grosso McCarthy and provides counsel to clients in both the not-for-profit and for-profit sectors. He helps clients navigate provincial, federal and international governments at the levels of the civil service and elected officials. Mike has also been on the front lines of health care, working for 18 years as a psychiatric nurse.

As a principal with Grosso McCarthy, Mike builds on more than 14 years of experience with the Ontario Ministry of Health and Long-Term Care and 24 years in health policy and delivery. He was appointed Senior Policy Advisor to the Progressive Conservative Minister of Health of Ontario, advising on physician, nursing and primary care, public health, laboratories, OHIP, organ transplant, HIV and blood issues.

In 2003, he provided strategic support and counsel to the government of Ontario during the SARS outbreak.

About QuestCap

QuestCap is an investment company that seeks to enhance shareholder value over the long term by opportunistically making various investments that may include, without limitation, the acquisition of equity, debt or other securities of publicly traded or private companies or other entities, financing in exchange for pre-determined royalties or distributions and the acquisition of all or part of one or more businesses, portfolios or other assets.

For additional information, please contact:

G Scott Moore

Co-Chairmansmoore@forbesmanhattan.com1-416-861-5903

Media contact:

Wynn TheriaultThirty Dash Communications34 King St East #400Toronto M5C 2X8416.710.3370

Cautionary Note Regarding Forward-lookingInformation

This press release contains "forward-looking information" within the meaning of applicable Canadian securities legislation. Forward-looking information includes, but is not limited to, statements with respect to the investment with SERV; the grant of options; and Sunnybrook and the appointment of advisory board members. Generally, forward-looking information can be identified by the use of forward-looking terminology such as "plans", "expects" or "does not expect", "is expected", "budget", "scheduled", "estimates", "forecasts", "intends", "anticipates" or "does not anticipate", or "believes", or variations of such words and phrases or state that certain actions, events or results "may", "could", "would", "might" or "will be taken", "occur" or "be achieved". Forward-looking information is subject to known and unknown risks, uncertainties and other factors that may cause the actual results, level of activity, performance or achievements of the Company, as the case may be, to be materially different from those expressed or implied by such forward-looking information. Although the Company has attempted to identify important factors that could cause actual results to differ materially from those contained in forward-looking information, there may be other factors that cause results not to be as anticipated, estimated or intended. There can be no assurance that such information will prove to be accurate, as actual results and future events could differ materially from those anticipated in such statements. Accordingly, readers should not place undue reliance on forward-looking information. The Company does not undertake to update any forward-looking information, except in accordance with applicable securities laws.

NEITHER THE CANADIAN SECURITIES EXCHANGE NOR ITS REGULATION SERVICES PROVIDER HAS REVIEWED OR ACCEPTS RESPONSIBILITY FOR THE ADEQUACY OR ACCURACY OF THIS RELEASE.

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Negative Pressure Wound Therapy market report:

The Negative Pressure Wound Therapy markets business intelligence research comprehensively provides a quick of crucial facts consisting of the merchandise catalogue, analytical elaboration, and other industry-linked information.

The study also covers the key aspects related to the on-going events such as mergers & acquisitions, new product launches, and synergisms. The study further harmonies a rigid preliminary for gaining loads of insights that potential buyers can use for ensuring better profits at low capitals. The demonstration of information on market segmentation by type, application, and geography delivers a critical viewpoint of, what manufacturers are seeking for the stipulated timeframe, 2020 2026.

Scope of the Report:

North America is the largest market of negative pressure wound therapy, both in production and consumption. In 2015, North America supplied more than 60% and consumed more than 50% of the global negative pressure wound therapy.

Europe is the second largest market, following North America. Asia area is the emerging market with a higher growth rate. While the negative pressure wound therapy industry in Asia market is still underdevelopment with many hospitals and patients are still using common gauze to cure wound.

In recent years, disposable negative pressure wound therapy is more and more popular. Among them, PICO single use product provided by Smith & Nephew is outstanding.

The worldwide market for Negative Pressure Wound Therapy is expected to grow at a CAGR of roughly 6.1% over the next five years, will reach 2000 million US$ in 2024, from 1400 million US$ in 2019, according to a new Globalmarketers.biz Research study.

This report focuses on the Negative Pressure Wound Therapy in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the market based on manufacturers, regions, type and application.

This article will help the Negative Pressure Wound Therapy manufacturers identify the volume inflation prospect with affecting trends.

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An in-depth list of key vendors in Negative Pressure Wound Therapy market includes:

Acelity (KCI)Smith & NephewCardinal HealthDevonMedelaTriage MeditechWuHan VSDTalley Group

Negative Pressure Wound Therapy Market segment by Type, the merchandise are often split into

Conventional NPWT DevicesDisposable NPWT Devices

Market segment by Application, split into

HospitalsClinicsHomecare

Market segment by Regions/Countries, this report covers

North America

Europe

China

Japan

Southeast Asia

India

Central & South America

Make an Inquiry About This Report @https://www.globalmarketers.biz/report/medicine/global-gene-therapy-market-2019-by-company,-regions,-type-and-application,-forecast-to-2024/130926#inquiry_before_buying

The study objectives of this report are:

In this study, the years considered to estimate the market size of Negative Pressure Wound Therapy are as follows:

For the info information by region, company, type and application, 2019 is taken into account because the base year. Whenever data information was unavailable for the bottom year, the prior year has been considered.

The market study discusses the highlighted segments on the idea of BPS, market share, profit, and other vital factors. Our business report elaborates the impact of various subdivisions to the growth of the global Negative Pressure Wound Therapy market. It also delivers information on key trends associated with the subdivisions covered in the report. This aids market participants to address worthwhile areas of the global Negative Pressure Wound Therapy market. The marketing study also delivers analysis on the subdivisions supported absolute dollar opportunity.

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Ask for Detailed Table of Content with Table of Figures:

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Negative Pressure Wound Therapy Market : Growth, Demand Analysis, Size, Global Industry Research Trends, Manufacturers, Developments in Performances...

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 31 March 2020 10:51Hits: 317

CAMBRIDGE, MA & SAN FRANCISCO, CA, USA I March 30, 2020 IGeneration Bio and Vir Biotechnology (Nasdaq: VIR) today announced a collaborative research agreement to explore the potential for Generation Bios non-viral gene therapy platform to extend the impact and reach of Virs current or future human monoclonal antibodies (mAb) against SARS-CoV-2, the virus responsible for COVID-19. Generation Bios technology has the potential to deliver genetic information directly to cells without the use of adeno-associated viruses (AAV), in effect instructing the patients body to produce the antibody itself.

The companies believe that this technology, coupled with Virs potent neutralizing antibodies, has the potential to provide effective, long-lasting protection against SARS-CoV-2. Virs leading antibody was isolated from a SARS-CoV-1 recovered patient and potently neutralizes SARS-CoV-2. Vir believes that this approach can potentially provide broad and longer-lasting protection.

Together, we believe we can develop long-lasting therapies suitable for population-wide prevention and treatment, said Generation Bio President and CEO Geoff McDonough, M.D. We are moving with urgency to explore leveraging our platform to build protection against COVID-19 for the long term.

Generation Bios proprietary non-viral gene therapy platform is designed to enable production of target proteins from a patients own cells. This approach may allow the patient to maintain stable levels of antibody expression for years, providing continuous protection against the target virus. In addition, the companies intend to leverage Generation Bios scalable manufacturing process to potentially extend the reach of Virs monoclonal antibodies to a greater number of patients.

We are eager to bring our antibodies to patients as quickly as possible, and should they work, to make them available to as many patients as quickly as possible. We are excited to explore the potential of ceDNA in an infectious disease setting and our anti-SARS-CoV-2 program offers a way to do that, said George Scangos, Ph.D., CEO of Vir. Both companies are highly motivated to make meaningful contributions to stopping this disease and we look forward to a productive collaboration with Generation Bio.

About Generation Bio

Generation Bio is an innovative genetic medicines company focused on creating a new class of gene therapy to provide durable, redosable treatments for patients suffering from both rare and prevalent diseases. The companys non-viral platform incorporates a proprietary high-capacity DNA construct called closed-ended DNA, or ceDNA; a novel cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The company is designing therapies to provide targeted delivery of genetic payloads that include large and multiple genes across a broad array of tissues, and to be redosable for individualized and extended treatment throughout a patients life. The platform is designed to expand access to treatments for rare diseases and to address prevalent diseases through efficient, scalable manufacturing.

About Virs Antibody Platform

Vir has a robust method for capitalizing on unusually successful immune responses naturally occurring in people who are protected from, or have recovered from, infectious diseases. The platform is used to identify rare antibodies from survivors that have the potential to treat and prevent rapidly evolving and/or previously untreatable pathogens via direct pathogen neutralization and immune system stimulation. Vir engineers the fully human antibodies that it discovers to enhance their therapeutic potential. This platform has been used to identify and develop antibodies for pathogens including Ebola (mAb114, currently in use in the Democratic Republic of Congo), hepatitis B virus, influenza A, SARS-CoV-2, malaria, and others.

About Vir Biotechnology

Vir Biotechnology is a clinical-stage immunology company focused on combining immunologic insights with cutting-edge technologies to treat and prevent serious infectious diseases. Vir has assembled four technology platforms that are designed to stimulate and enhance the immune system by exploiting critical observations of natural immune processes. Its current development pipeline consists of product candidates targeting hepatitis B virus, influenza A, SARS-CoV-2, human immunodeficiency virus, and tuberculosis. For more information, please visit http://www.vir.bio.

SOURCE: Generation Bio

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Generation Bio and Vir Biotechnology to Collaborate on Research to Leverage Scalable Non-Viral Gene Therapy Platform for Durable Production of...

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A programme investigating the potential of gene therapy for the treatment of osteoarthritis in horses, dogs and cats, has begun at the Royal Veterinary College (RVC), led by Skeletal Regeneration expert, Dr Scott Roberts.

Dr Scott Roberts, who joined the RVC as Senior Lecturer in Translational Skeletal Research in the Department of Comparative Biomedical Sciences, has recently initiated the programme of investigative research.

The research is inspired by Dr Roberts interest in skeletal stem cell signalling and the effect of signal modulation on skeletal tissue regeneration. He initiated this work during his time at KU Leuven (Belgium) but has subsequently researched this topic as Principal Investigator at University College London and as Senior Principal Scientist at UCB Pharma. During his time at UCB, Dr Roberts identified musculoskeletal drug targets and led the subsequent drug discovery projects. His research has resulted in several patent applications on the use of cells and therapeutic antibodies to promote the regeneration of bone and cartilage.

The new gene therapy research programme in osteoarthritis will be undertaken in partnership with the LEP funded Vaccinology and Cell Therapy Hub at the RVC. This will not only provide an environment for further research on regenerative medicine but also the facilities to create reagents for clinical translation.

The Hub with its close connections to both scientists and veterinary clinicians is also well-placed to take the science from bench-to-bedside and facilitate clinical trials in veterinary patients. This will include horses at the Equine Referral Hospital, and dogs and cats at the Queen Mother Hospital for Animals at the RVC.

It is proposed that this treatment would transition into human clinical studies, exemplifying the RVCs commitment to the One Health approach which recognises and facilitates the synergy between animal and human health.

Dr Scott Roberts said:

This research has the potential to change the way that we approach degenerative joint disease and I am delighted to have access to the Vaccinology and Cell Therapy Hub while we undertake this work. We hope that this science will lead to a ground-breaking treatment for osteoarthritis in animals, and eventually humans. We are optimistic about the future of this research, particularly given its contribution to the RVCs One Health ethos. I look forward to taking advantage of the RVCs world-leading facilities, as well as the scientists and clinicians who will help us take this research forward.

Professor Jonathan Elliott, Vice Principal of Research and Innovation at the RVC, said:

We are delighted that Scott Roberts has joined the RVC and will pursue his translational research into novel therapies that stimulate repair of cartilage for osteoarthritis in horses, dogs and people. This appointment builds on the RVCs expertise in Skeletal Biology. Scotts links to the Pharma Industry are very important for his translational science to have impact. His work fits perfectly with the goals of our soon to open Vaccinology and Cell Therapy Hub, made possible by the funding from the Hertfordshire Local Enterprise Partnership.

The research Dr Roberts is undertaking is critical as cartilage has a very poor capacity to repair itself, with cartilage injuries often progressing to osteoarthritis. There is also currently no approved evidence-driven therapy for the treatment of this disease.

In addition to osteoarthritis, Dr Roberts research aims to create regenerative therapeutics for non-healing bone fractures. This is based on a comprehensive understanding of tissue development, as tissue repair is now regarded as a re-emergence of embryonic signalling cascades. Dr Roberts has used knowledge in this area to identify developmentally inspired methodologies to create laboratory grown tissue implants that have the capacity to drive bone fracture repair.

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Investigation Begins into Gene Therapy for Osteoarthritis - Everything Horse UK

Covington-based CTIClinical Trial and Consulting Services has partnered with California-based Humanigen, Inc. on a study for the treatment of novel coronavirus 2019 (COVID-19).

Humanigen, Inc., (HGEN) (Humanigen), a clinical-stage biopharmaceutical company focused on preventing and treating cytokine storm with lenzilumab, the companys proprietary Humaneered anti-human-granulocyte-macrophage colony-stimulating factor (GM-CSF) monoclonal antibody, announced that the company has submitted an initial protocol synopsis to the FDA in support of the companys plans to initiate a multi-center, US, Phase III study in COVID-19 patients.

The study, if approved, will be a randomized, controlled, clinical trial with lenzilumab for the prevention of ARDS and/or death in hospitalized patients with pneumonia associated with coronavirus 2 (SARS-CoV-2) infection in COVID-19.

The study will be conducted in partnership with CTI, which specializes in complex programs for critically and chronically ill patients.

Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of cancers and infectious diseases via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms.

Humanigen has pioneered the field of GM-CSF neutralization and, unlike others, has already conducted two Phase I and two Phase II studies, including in patients with severe respiratory conditions, with excellent safety results, said Dr. Cameron Durrant, chief executive officer of Humanigen. We have been working on prevention of cytokine storm for nearly three years. Lenzilumab has an excellent safety and tolerability profile and has not been associated with serious adverse events, including in patients who are immunosuppressed or with severe asthma. Subject to discussion and agreement with regulatory authorities, we intend to progress as quickly as possible with this study.

More details on the companys programs in COVID-19 can be found on the companys websiteunder the COVID-19 tab.

CTI Clinical Trial and Consulting Services is a global, privately held, full-service contract research organization (CRO).

It delivers a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization.

CTIs focused therapeutic approach provides pharmaceutical, biotechnology, and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine/gene therapy, immunology, transplantation, nephrology, hematology/oncology, neurology, infectious diseases, hepatology, cardiopulmonary, and pediatric populations.

Schroeder

The COVID-19 pandemic demands an unprecedented response from the pharmaceutical and biotechnology communities, requiring innovation, collaboration, and prioritization of speed without the cost of safety, remarked Tim Schroeder, founder and CEO of CTI. We are proud to have the opportunity to collaborate on such a response with the Humanigen team and to work together to develop an effective treatment solution for those hardest hit by COVID-19.

CTI also offers a fully integrated multi-specialty clinical research site that conducts phase I-IV trials. CTI has a passion for helping life-changing therapies succeed in chronically and critically ill patient populations. With clinical trial experience across 6 continents, CTI partners with research sites, patients, and sponsors to fulfill unmet medical needs. CTI is headquartered in the Greater Cincinnati, OH area, with operations across North America, Europe, Latin America, and Asia-Pacific.

For more information on CTI, click here.

Study to tackle leading cause of death in COVID-19 patients

Lenzilumab, a monoclonal antibody that neutralizes granulocyte macrophage colony stimulating factor (GM-CSF)

GM-CSF is up-regulated in COVID-19 patients and may cause a harmful immune response leading to acute respiratory distress syndrome (ARDS)

Clinical evidence suggests that this harmful immune response contributes to cytokine storm in COVID-19 patients at risk of developing ARDS

Company plans to study lenzilumab in COVID-19 patients to prevent ARDS and mortality with CTI Clinical Trial and Consulting Services (CTI)

COVID-19 is an infectious disease caused by SARS-CoV-2. COVID-19 has become a global pandemic, with over 460,000 confirmed cases and over 21,000 deaths reported to date. Patients with severe cases of COVID-19 experience severe viral pneumonia that can progress to acute respiratory distress syndrome (ARDS) and death.

ARDS is an acute, life-threatening inflammatory lung injury characterized by hypoxia a lack of oxygen to the tissue and stiff lungs due to increased pulmonary vascular permeability. ARDS necessitates hospitalization and mechanical ventilation. A rapid increase in patients with ARDS presents a major challenge for the global public health system given limited hospital beds and ventilators. When implementing standard of care, including mechanical ventilation, ARDS has an overall mortality rate of greater than 40 percent.

Humanigen, Inc. study is focused on the belief that its GM-CSF neutralization and gene-editing platform technologies have the potential to reduce the inflammatory cascade associated with coronavirus infection as well as the serious and potentially life-threatening CAR-T therapy-related side effects while preserving and potentially improving the efficacy of the CAR-T therapy itself, thus breaking the efficacy/toxicity linkage.

The companys immediate focus is to prevent or minimize the cytokine storm that precedes severe lung dysfunction and ARDS in cases of pneumonia-associated SARS-CoV-2 infection and also in combining FDA-approved and development stage CAR-T therapies with lenzilumab, the companys proprietary Humaneered anti-human-GM-CSF immunotherapy, which is its lead product candidate.

CTI

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Covington-based CTI partners with Humanigen for study of Lenzilumab for treatment of Coronavirus - User-generated content

WALTHAM, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies and a mission to develop transformative medicines for devastating diseases, today announced it has closed an oversubscribed $60 million Series A financing. Seed venture investors F-Prime Capital and New Enterprise Associates (NEA) co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza and Partners Innovation Fund.

The proceeds will be used to advance the companys platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

The company is led by recently appointed Chief Executive Officer Rick Modi, who has a proven track record of building value at companies including AveXis, InterMune, MedImmune and Centocor. Joining the board of directors as part of the financing are Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, NEA; and Robert Weisskoff, Ph.D., Partner, F-Prime Capital. Industry veteran and gene therapy leader Sean Nolan will chair the board.

At Affinia Therapeutics, were setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors and gene therapies that have remarkable targeting properties, said Modi, CEO of Affinia. We are pleased to partner with such a distinguished syndicate to advance our platform and investigational product candidates toward the clinic for patients in need.

The companys technology was licensed from Lonza and Massachusetts Eye and Ear. It was developed at the Grousbeck Gene Therapy Center and further advanced under a sponsored research agreement with Lonza led by Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

In addition to Vandenberghe, joining Affinia Therapeutics as scientific co-founders are Botond Roska, M.D., Ph.D., Director, Institute of Molecular and Clinical Ophthalmology Basel; Aaron Tward, M.D., Ph.D., Assistant Professor, University of California, San Francisco; and Eric Zinn, Ph.D. student, Mass. Eye and Ear and Harvard University. Together, these scientists have authored more than 200 papers and filed more than 20 patents in the field of gene therapy.

Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies. By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine, said Vandenberghe, Director, Grousbeck Gene Therapy Center at Mass. Eye and Ear.

The Series A financing comes after Vandenberghe and his team successfully developed AAVSmartLibraries comprising thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vectors structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. Affinia Therapeutics has potentially the worlds largest library of patented functional AAV vectors.

Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines, said Mathers, General Partner at NEA. We are pleased to accelerate the impact of this exciting field.

About Affinia Therapeutics

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

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Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies - GlobeNewswire

Demonstrates broad biodistribution, including across the blood-brain barrier into the central nervous system, and into skeletal muscle, in non-human primates (NHPs) after systemic administration

Durable and therapeutically relevant drug concentrations achieved in NHPs after single intravenous dose

Potent cell-based activity and allele-specific enrichment in patient-derived cell lines

Platform validation data supports expansion of the therapeutic pipeline into new organ systems previously unreachable with first-generation antisense oligonucleotide technology

Management to hold a conference call today at 8 a.m. ET

PITTSBURGH, March 31, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq:NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies to address genetic diseases, today announced positive preclinical data from its pharmacokinetics studies in non-human primates (NHPs) and in vitro pharmacodynamics data in patient-derived cell lines. NeuBase believes these data validate the key advantages of the proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform and support the Companys decision to advance the development of its Huntingtons disease (HD) and myotonic dystrophy type 1 (DM1) programs, as well as the potential expansion of its therapeutic pipeline into other indications.

Dr. George Church, professor of genetics at Harvard Medical School and member of the National Academy of Sciences, stated, Given the activity and broad biodistribution observed in these studies and the potential for easier target definition, I believe the PATrOL technology may have a potent impact on the future of drug development and treatment of genetic diseases.

Non-Human Primate Pharmacokinetic Study

Quantitative whole-body autoradiography was performed on NHPs.A PATrOL-enabled compound was radio-labeled, and theresulting material was injected into NHPs at 5 mg/kg via a bolus tail vein injection. At four hours, twelve hours, and seven days post-dosing, NHPs were sacrificed andsectioned into 40 m slices.Slices were exposed to autoradiography imaging plates alongside a dilution series of radioactive PNA in whole blood.Upon imaging, the dilution series enabled an analysis of the amount of compound in each of the tissues. In addition, prior to sacrifice, whole blood, urine, and feces were collected from the NHPs at specified timepoints.The major conclusions from this study include:

Rapid uptake of compound out of the bodys circulation after systemic intravenous administration, with a half-life in circulation of approximately 1.5 hours;

Compound penetrates every organ system studied, including the central nervous system and skeletal muscle;

Compound crosses the blood-brain barrier and into the key deep brain structures, including the caudate, supporting a key capability for the development of the Companys lead program in HD; Delivery of the compound to skeletal muscle, the primary organ system that is affected in DM1;Because both HD and DM1 have manifestations outside of the primary affected organ, the broad biodistribution of the compounds may enable a potential whole-body therapeutic solution in both indications.

96% of administered compound remained in vivo after a one-week period (latest timepoint tested);Redistribution over one week after administration between organ systems enriches concentrations in key brain regions up to two-fold, including in those deep brain structures most relevant for HD;Retention of ~90% of compound concentrations achieved in skeletal muscle over the course of one-week post-single-dose administration; and

Patient-Derived Huntingtons Cell Line Pharmacodynamic Studies

Multiple Huntingtons disease candidate compounds were incubated with HD-derived cells and assayed for their toxicity and their ability to selectively knock down mutant huntingtin protein (mHTT) expression by engaging with the CAG repeat expansion in the huntingtin (HTT) gene transcript. Multi-well plates were seeded with cells and candidates were added to the culture at various concentrations.Cells were grown for three days and thereafter assayed for cell death.Cell pellets were also collected, lysed, and run on gradient SDS-PAGE gels.Following the transfer of the proteins to a membrane, the membrane was probed with anti-huntingtin and anti-beta-actin antibodies.Secondary antibodies were used to image the immunoblots.The beta-actin bands were used to normalize the amount of protein across the wells.The amounts of mutant and wild type huntingtin protein in treated cells were compared to untreated cells to determine the level of knockdown.The major conclusions from this study include:

Story continues

Activity in engaging target disease-causing transcripts and knocking-down resultant malfunctioning mHTT protein levels preferentially over normal HTT protein knock-down; and

Dose limiting toxicities were not observed relative to a control either at or above the doses demonstrating activity in human cells in vitro.

In addition, PATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month.

We believe the PATrOL platform has the potential to create drugs that are easy for patients to take at infrequent intervals after they have tested positive for a genetic disease but before symptoms emerge, said Dietrich Stephan, Ph.D., chief executive officer of NeuBase. We believe the best way to effectively manage degenerative genetic diseases is to get ahead of the disease process, and we believe that can only be achieved with early diagnosis coupled with well-tolerated, effective, and easily administered therapies.

Dr. Robert Friedlander, chief medical officer of NeuBase and member of the National Academy of Medicine, stated, An allele specific approach that can be systemically administered and cross the blood brain barrier would be an ideal drug profile for many untreatablegenetic diseases.I believe that NeuBase is moving towards realizing this goal.

The intersection of the NHP pharmacokinetic data and the in vitro patient-derived pharmacodynamic data provides a roadmap to create a pipeline of therapeutic candidates which can reach target tissues of interest after systemic administration and achieve the desired activity at that dose. NeuBase believes the data from these studies support the advancement of the Companys HD and DM1 programs into lead optimization and subsequent IND-enabling studies, as well as provide a roadmap for the future expansion of the Companys therapeutic pipeline into other indications, including oncology.

Dr. Sam Broder, former Director of the National Cancer Institute of the National Institutes of Health and member of the National Academy of Sciences, stated, I believe that the NeuBase strategy of targeting transcripts before they become dangerous mutant proteins has the potential to deliver a dramatic improvement in our collective capabilities to effectively treat a wide range of genetic diseases, including some of the most deadly cancers, by targeting driver mutations and accelerating immunotherapy capabilities.

Conference Call

NeuBase Therapeutics, Inc. will discuss these data and next steps for development during a webcasted conference call with slides today, March 31, 2020, at 8:00 a.m. ET. The live and archived webcast of this presentation can be accessed through the IR Calendar page on the Investors section of the Companys website, http://www.neubasetherapeutics.com. The dial-in details for the call are 877-451-6152 (domestic) or +1-201-389-0879 (international), and conference ID: 13701118. The archived webcasts will be available for approximately 30 days following the presentation date.

About NeuBase Therapeutics

NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform is designed to permit the rapid development of targeted drugs, thereby potentially increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements include, among other things, statements regarding the Companys goals and plans and the Companys pharmacokinetics and pharmacodynamics studies. These forward-looking statements are distinguished by use of words such as will, would, anticipate, expect, believe, designed, plan, or intend, the negative of these terms, and similar references to future periods. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those described in the risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Companys current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Companys plans to develop and commercialize its product candidates; the Companys plans to commence clinical trials in Huntingtons disease and myotonic dystrophy type 1 and to potentially expand the pipeline into other indications; the utility of the preclinical data generated in existing studies performed by the Company in determining the results of potential future clinical trials and of the potential benefits of the PATrOL platform technology; the timing of initiation of the Companys planned clinical trials; the timing of the availability of data from the Companys clinical trials; the timing of any planned investigational new drug application or new drug application; the Companys plans to research, develop and commercialize its current and potential future product candidates; the clinical utility, potential benefits and market acceptance of the Companys current and potential future product candidates; the Companys commercialization, marketing and manufacturing capabilities and strategy; the Companys ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCDaniel@lifesciadvisors.comOP: (617) 535-7746

NeuBase Media Contact:Travis Kruse, Ph.D.Russo Partners, LLCtravis.kruse@russopartnersllc.comOP: (212) 845-4272

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NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL Platform - Yahoo Finance

Gene Therapy Market industry development trends and strategic plan for positive Growth discuss in research report.The global gene therapy market is estimated to be over US$ 411.2 Mn in 2018. It is anticipated to grow at a double-digit CAGR of 33.1% from 2019 to 2030.

This press release was orginally distributed by SBWire

New York, NY -- (SBWIRE) -- 03/31/2020 -- As per research findings of Market Industry Reports (MIR), increasing number of product approvals & R&D activities, supportive government initiatives in the gene therapy arena are set to open new avenues for stakeholders.

Major Players Include:

The global gene therapy market is largely governed by prominent players such as Novartis AG, UniQure N.V., Regenxbio Inc., Sibiono GeneTech Co. Ltd., Amgen Inc., Voyager Therapeutics, Sarepta Therapeutics, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, and Orchard Therapeutics plc, among others.

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Growth Factors:

Growth opportunities in the global gene therapy market looks promising during the forecast period. Rising incidences of chronic diseases as well as genetic disorders globally, favorable initiatives from governments to support research & development of gene therapy products and technological advancements are some of the key factors driving the global gene therapy market.

Similarly, increased focus of players in this market for getting approvals for gene therapy products is anticipated to boost the gene therapy market significantly during the forecast period. For instance, in May 2019, AveXis, a U.S.-based company received FDA's approval for Zolgensma. Zolgensma is used for the treatment of a rare genetic disorder and Spinal Muscular Atrophy (SMA) in pediatric patients below the age of 2 years."Companies are striving to discover, develop, and deliver gene therapies for rare genetic disorders,"

From a regional perspective, Europe is expected to witness the highest CAGR in the coming years due to presence of several gene therapy products in the development stage. Whereas, North America accounts for the largest share in the gene therapy market. The large share of this region can be attributed to rising incidences of chronic diseases & genetic disorders such as cancer, hemophilia, Multiple Sclerosis (MS), and others.

For deep-rooted insights on this topic, Go for Interesting Discount @ https://www.marketindustryreports.com/discount/149

MARKET ECOSYSTEM/CLASSIFICATION

By Vector Type

Viral VectorsAdenovirusLentivirusRetrovirusAdeno-associated virusHerpes simplex virusPoxvirusVaccinia virusOthersNon-viral VectorsNaked plasmid vectorGene gunElectroporationLipofectionOthers

By Gene TypeAntigenCytokineTumor SuppressorSuicideDeficiencyGrowth FactorsReceptorsOthers

By ApplicationOncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious DiseasesOther Diseases

REASONS TO BUY THE REPORT:

Deep-rooted quantitative and qualitative insights at segment & sub-segment levelGlobal insights with country levels informationComprehensive insights on market dynamics, and business environment Insights on 'Key Strategies' followed by leading players of the marketAccess to 'Latest Strategic Developments' registered by leading players of the market

CUSTOMIZATION OPTIONS:

Key Distributors ListFree addition of 'Company Profiles'Clientele Base Assessment

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About Market Industry ReportsMarket Industry Reports is a global leader in market measurement and advisory services. It is a 100% subsidiary of ExlTech. The firm has always been at the forefront of innovations to address worldwide industry trends and opportunities. We offer our clients a unique depth of market intelligence in an actionable format to move their business forward. Our analysis incorporates consumer study in more than 100 countries, providing a tactical approach to drive sustained business growth. We continue to pioneer state-of-the-art approach in research & analysis that will help you to overcome complexities and stay ahead of the curve. By nurturing the perception of genius and optimized market intelligence, we unfold contingencies for our clients in the evolving world of technology, mega-trends, and industry convergence. We inspire our clients to empower and shape their businesses and to build world-class products.

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For more information on this press release visit: http://www.sbwire.com/press-releases/gene-therapy-market-companies-outlook-and-trends-likely-to-witness-cagr-of-331-from-2019-to-2030-1285124.htm

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Gene Therapy Market Companies Outlook and Trends Likely to Witness CAGR of 33.1% from 2019 to 2030 - Press Release - Digital Journal

A new research study titled Global Hemophilia Gene Therapy Market Growth (Status and Outlook) 2020-2025 has been presented by MRInsights.biz offering a comprehensive analysis of the market through which users can benefit from the complete market research report with all the required useful information about this global Hemophilia Gene Therapy market. The report covers the latest and upcoming industry trends and offers a global spectrum of the market and future forecast from 2020 to 2025 years. The report offers a detailed examination of opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, and developments.

The market is bifurcated into product type, application, key manufacturers and key regions and countries. The report assists users to achieve competitive leverage with acquiring and preserving market position in the global Hemophilia Gene Therapy industry. The report provides an investigation into the global market status, shares, supply-demand, market drivers, challenges and opportunities, and geological areas. The report then serves information on sales and market share estimates by-product as well as a profile of the companys business. The study emphasizes on consumption, market share and growth rate of the industry. The research helps readers to understand strategies to make sound investments.

DOWNLOAD FREE SAMPLE REPORT: https://www.mrinsights.biz/report-detail/233493/request-sample

On the basis of types, the market is primarily split into: Hemophilia A, Hemophilia B

On the basis of applications, the market covers: Hemophilia A Gene Therapy, Hemophilia B Gene Therapy

Additionally company basic information, manufacturing base, and competitors list is being provided for each listed manufacturers: Spark Therapeutics, Ultragenyx, Sangamo Therapeutics, Bioverativ, Shire PLC, Freeline Therapeutics, BioMarin, uniQure

All key regions and countries are assessed here on the basis of company, type of product, and application covering Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia, Spain), Middle East & Africa (Egypt, South Africa, Israel, Turkey, GCC Countries)

The study presents in-depth insights into each of the leading end-user verticals along with annual forecasts to 2025. The report includes global Hemophilia Gene Therapy market consumption analysis by application as well as analysis of value, product utility, market percentage, and production market share by type. Key attributes of the leading companies are displayed that includes company overview, financial overview, recent developments, development strategies, and SWOTs.

ACCESS FULL REPORT: https://www.mrinsights.biz/report/global-hemophilia-gene-therapy-market-growth-status-and-233493.html

The Report Addresses The Following Queries Related To The Market:

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. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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Global Hemophilia Gene Therapy Market 2020 Industry Size, Development Status, Estimated CAGR, and Forecast to 2025 - Galus Australis

DUBLIN--(BUSINESS WIRE)--The "Hemophilia Treatment Market to 2027 - Global Analysis and Forecasts by Product; Disease; Treatment Type; Therapy; End User, and Geography" report has been added to ResearchAndMarkets.com's offering.

The global hemophilia treatment is expected to reach US$ 44,089.71 million in 2027 from US$ 14,454.81 Mn in 2019. The market is estimated to grow with a CAGR of 15.9% from 2020-2027.

The growth of the hemophilia treatment is primarily attributed to the factors such as increasing prevalence of hemophilia and rise in favorable government initiatives leading to launch of new products in the market. However, high cost related with hemophilia treatment is likely to pose a negative impact on the market growth. On the other hand, growth opportunities in emerging regions are likely to have a positive impact on the growth of the global hemophilia treatment during the forecast period.

According to data from the National Hemophilia Foundation in 2020, Hemophilia A happens in 1 in 5,000 live male births. Hemophilia A is about four times as common as hemophilia B. The number of people with hemophilia in the United States is anticipated to be around 20,000 individuals. Moreover, the incidence of hemophilia is not known across the globe but projected at above 400,000 people. Almost 75% of people with hemophilia worldwide are either undiagnosed or receive disparate treatment.

In Italy, according to the data of the National Center for Biotechnology Information (NCBI) in 2017, the number of registered people with bleeding disorders increased from about 7000 in 2000 to around 8500 in 2011 and more than 11,000 in 2015. The trend is due to an upsurge in the number of patients who are recorded, mainly in those with vWD type 1, mild hemophilia, or other factor deficiencies. Thus, increasing prevalence of hemophilia, high cases of under-diagnosed hemophilia cases, and improving healthcare infrastructure will eventually grow the market for hemophilia treatment in the forecast period.

Global hemophilia treatment, based on the product, is segmented into plasma derived coagulation factor concentrates plasma derived coagulation factor concentrates, desmopressin, antifibrinolytic agents. In 2019, the recombinant coagulation factor concentrates held the largest share of the market, by product. Also, recombinant coagulation factor concentrates segment is expected to grow at the fastest rate during the coming years owing to increasing adoption of integrated systems across the globe.

Reasons to Buy

Key Topics Covered

1. Introduction

1.1 Scope of the Study

1.2 Report Guidance

1.3 Market Segmentation

1.3.1 Global Hemophilia Treatment Market - By Product

1.3.2 Global Hemophilia Treatment Market - By Disease

1.3.3 Global Hemophilia Treatment Market - By Treatment Type

1.3.4 Global Hemophilia Treatment Market - By Therapy

1.3.5 Global Hemophilia Treatment Market - By End User

1.3.6 Global Hemophilia Treatment Market - By Geography

2. Global Hemophilia Treatment Market - Key Takeaways

3. Research Methodology

3.1 Coverage

3.2 Secondary Research

3.3 Primary Research

4. Hemophilia Treatment- Market Landscape

4.1 Overview

4.2 PEST Analysis

4.2.1 North America - PEST Analysis

4.2.2 Europe: PEST Analysis

4.2.3 APAC: PEST Analysis

4.2.4 MEA: PEST Analysis

4.2.5 South & Central America - PEST Analysis

4.3 Expert Opinion

5. Hemophilia Treatment Market - Key Market Dynamics

5.1 Market Drivers

5.1.1 Increasing Prevalence of Hemophilia

5.1.2 Rise in Favorable Government Initiatives

5.2 Market Restraints

5.2.1 High Cost Related with Hemophilia Treatment

5.3 Market Opportunities

5.3.1 Growth in the Global Healthcare Market

5.4 Future Trends

5.4.1 Research Activities and Product Innovations

5.5 Impact Analysis

6. Hemophilia Treatment Market - Global Analysis

6.1 Global Hemophilia Treatment Market Revenue Forecast And Analysis

6.2 Global Hemophilia Treatment Market, By Geography - Forecast And Analysis

6.3 Positioning of Key Players

6.3.1 Bayer AG

6.3.2 Pfizer Inc.

7. Global Hemophilia Treatment Market Analysis- by Product

7.1 Overview

7.2 Hemophilia Treatment Market Revenue Share, by Product (2019 and 2027)

7.3 Plasma-Derived Coagulation Factor Concentrates

7.4 Recombinant Coagulation Factor Concentrates

7.5 Desmopressin

7.6 Antifibrinolytic Agents

8. Hemophilia Treatment Market Analysis - by Disease

8.1 Overview

8.2 Hemophilia Treatment Market, by Disease 2019 and 2027 (%)

8.3 Hemophilia A

8.4 Hemophilia B

8.5 Hemophilia C

9. Hemophilia Treatment Market Analysis - by Treatment Type

9.1 Overview

9.2 Hemophilia Treatment Market Revenue Share, by Treatment Type (2019 and 2027)

9.3 Prophylaxis

9.4 On-demand

10. Hemophilia Treatment Market Analysis - by Therapy

10.1 Overview

10.2 Hemophilia Treatment Market, by Therapy 2019 and 2027 (%)

10.3 Replacement Therapy

10.4 ITI Therapy

10.5 Gene Therapy

11. Hemophilia Treatment Market Analysis - by End User

11.1 Overview

11.2 Hemophilia Treatment Market, by End User 2019 and 2027 (%)

11.3 Hospitals & Clinics

11.4 Hemophilia Treatment Centers

11.5 Ambulatory Surgical Centers

12. Hemophilia Treatment Market - Geographic Analysis

12.1 North America: Hemophilia Treatment Market

12.2 Europe: Hemophilia Treatment Market

12.3 APAC: Hemophilia Treatment Market

12.4 Middle East & Africa: Hemophilia Treatment Market

12.5 South And Central America: Hemophilia Treatment Market

13. Global Hemophilia Treatment Market - Industry Landscape

13.1 Overview

13.2 Growth Strategies Done By the Companies in the Market, (%)

13.3 Organic Developments Done By The Companies in The Market

13.4 Inorganic Developments Done by The Companies In The Market

14. Company Profiles

14.1 Bayer AG

14.2 Sanofi

14.3 F. Hoffmann-La Roche Ltd.

14.4 Kedrion S.p.A.

14.5 CSL Limited

14.6 Biotest AG.

14.7 Pfizer Inc.

14.8 Novo Nordisk A/S

14.9 Octapharma AG

Continued here:
Hemophilia Treatment Market Study & Forecast to 2027 by Product, Disease, Treatment Type, Therapy, End-user and Geography - ResearchAndMarkets.com...

A leading market research firmFacts & Factors (FnF)added a research report onGene Therapy Market By Type (Germ Line Gene Therapy and Somatic Gene Therapy), By Vector Type (Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome), and By Therapy Area (Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027includes 190+ pages research report with TOC included in its research database. The research report portraying a wide-ranging assessment of theGene Therapy marketincludes the rate of expansion of the Gene Therapy market over the predicted duration. Providing a succinct outline, it concludes the valuation and size of the Gene Therapy market in the near future. Additionally, it also comprises the foremost contributing aspects to the development of the Gene Therapy market as well as the leading players in the market along with their market share. The top outstanding industry players/manufacturers are also included in this report to understand the companys business strategies, sales, and factor of growth.

The Gene Therapy market research report offers valuable insights pertaining to the revenue segmentation, business summary, and product offerings of the foremost market players. The study also estimates the expansion of the well-known market players through SWOT study. Also, it considers the most recent developments in the market whilst estimating the growth of major market players. Furthermore, in the Gene Therapy market size report, the main product category and segments along with its sub-segments of the Keyword market are elaborated.

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This free report sample includes: The report covers present status and future prospects. The report analyses market trends, size, and forecast in different geographically. The report provides market competition overview among the Top companies. The report provides a complete analysis of the current and emerging market trends and opportunities. Example pages from the report. FnF research methodology.

The Gene Therapy market research report also states the latest floats in the Gene Therapy market and numerous opportunities for the growth of the Gene Therapy market in the upcoming period. The study utilizes many methodological tools to estimate market expansion in the predicted period.

Thesize of Gene Therapy marketis divided based on the product type, purchaser, and application segments. The industry growth of each segment is assessed along with the prediction of their growth in the near future. The relevant data and statistics gathered from the regulatory authorities have been portrayed in the report to assess the development of segments. Further, the trends in the Gene Therapy market are also branched out on the basis of regions such as the Middle East & Africa, Asia Pacific, Latin America, North America, and Europe.

To Know What is Size, Share of the Gene Therapy Market, Ask a Free Sample Report:https://www.fnfresearch.com/sample/gene-therapy-market-by-type-germ-line-gene

Major Company Profiles Covered in This Report:

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The Gene Therapy market analysis research report is drafted on the basis of a brief assessment and huge data collected from the Gene Therapy market. The data collected include current industry trends and requirements associated with services & manufacturing goods.

The report on Gene Therapy market worth segments and defines the Gene Therapy market considering the in-detailed forecasts and study of the market size. The report further includes various figures, data tables, and in-depth TOC on the Gene Therapy market.

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The major region covered in this report:

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An in-depth analysis of the most recent developments and novel technological advancements provides the users with a free hand to introduce their exceptional products and processes to update the service contribution. The global share of Gene Therapy market report emphasizes the most recent trends, growth, and new business opportunities to provide a comprehensive review of the global market. The demand proportion and development of innovative technologies also are revealed in the Gene Therapy market report.

The statistics in the data collected are graphically presented in theGene Therapy market size and trendsresearch report. It also includes key performers, vendors, and suppliers. The report highlights the command and drivers influencing the market.

What Gene Therapy Market research report offers?

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Global Gene Therapy Market to Reflect Impressive Growth Rate by 2026 - Global Industry News 24