Category Archives: Genetic Therapy

Garda Mary Gardiner is a name you might remember from a video which did the rounds online back in April.

In it, Tralee Community Garda Mary Gardiner led locals in a recreation of the famous Is This The Way To Amarillo to raise awareness for the Do It For Dan campaign.

Well, Officer Gardiner is back with a larger cast in order to help out another amazing cause. The latest clip sees Mary and her emergency service colleagues dance to Uptown Funk by Bruno Mars. This time, the aim is to help raise vital funds for A Better Life For Livie, a campaign set up to help nine-month-old Livie receive treatment in the USA.

Livie has been diagnosed with a rare and serious genetic neuromuscular condition called spinal muscular atrophy (SMA) type 1 and she requires once-off gene therapy called Zolgensma, which is currently only available in USA.

Without treatment, it's unlikely that Livie will live past her second birthday but it's expected to cost around 2m. So far, a GoFundMe page has raised nearly 400,000 so there's still a lot more work to do. However, with the Gardai doing their bit like this, word is bound to spread fast. Check out the video in full below...

Some decent moves there it has to be said but Bruno can probably rest easy for now. You can find out more about A Better Life For Livie and donate if you wish to do so via this link.

More here:
WATCH: Gardai dance to Uptown Funk to raise funds for baby girl's treatment in USA - Lovin.ie

NEW YORK, May 28, 2020 /PRNewswire/ --

Global Viral Vector and Plasmid Manufacturing Market to Reach $5.86 Billion by 2030

Read the full report: https://www.reportlinker.com/p05902571/?utm_source=PRN

Market Report Coverage - Viral Vector and Plasmid Manufacturing

Market Segmentation

Vector Type Plasmid DNA and Viral Vector Viral Vector Type Adenovirus, Adeno-Associated Virus, Retrovirus, Lentivirus, Vaccinia Virus, and Other Viral Vectors Disease Type Cancer, Genetic Disease, Infectious Disease, Cardiovascular Disease, and Other Diseases Application Gene Therapy, Cell Therapy, Vaccinology, and Other Applications

Regional Segmentation North America U.S., Canada Europe Germany, U.K., France, Italy, Switzerland, Belgium, Spain, and Rest-of-Europe Asia-Pacific China, Australia, Japan, India, South Korea, Singapore, and Rest-of-Asia-Pacific Rest-of-the-World Latin America and Middle-East and Africa

Growth Drivers Rising Prevalence of Cancer, Genetic Disorders, and Infectious Diseases Rapid Uptake of Viral and Plasmid Vectors for the Development of Innovative Therapies Increasing Number of Clinical Studies for the Development of Gene Therapy Favorable Funding Scenario for Vector-Based Therapies

Market Challenges Unaffordable Cost of Gene Therapies High Manufacturing Costs of Viral Vectors and Plasmids Complications Associated with Large-Scale Production of Vectors

Market Opportunities Rising Demand for Synthetic Genes Emergence of Next-Generation Vectors

Key Companies ProfiledFUJIFILM Holdings Corporation, GENERAL ELECTRIC, Lonza, Merck KGaA, MolMed S.p.A., Novasep Holding, Oxford Biomedica plc, Catalent, Inc., Thermo Fisher Scientific, Inc., GenScript, Boehringer Ingelheim, Wuxi AppTec Co., Ltd., Sartorius AG, Takara Bio Inc., and Aldevron, L.L.C.

Key Questions Answered: What is a vector, and what is its importance in the medical industry? What are the major characteristics and types of vectors? What are the areas of application of vectors? What are the major advancements in the viral vector and plasmid manufacturing sector? What are the key trends of the global viral vector and plasmid manufacturing market? How is the market evolving and what is its future scope? What are the major drivers, challenges, and opportunities of the global viral vector and plasmid manufacturing market? What are the key developmental strategies implemented by the key players of the global viral vector and plasmid manufacturing market to sustain the competition of the market? What is the percentage share of each of the key players in different key developmental strategies? What is the regulatory scenario of the global viral vector and plasmid manufacturing market? What are the initiatives implemented by different governmental bodies and guidelines put forward to regulate the commercialization of viral vector and plasmid manufacturing products? What are major milestones in patenting activity in the global viral vector and plasmid manufacturing market? What was the market size of the global viral vector and plasmid manufacturing market in 2019, and what is the market size anticipated to be in 2030? What is the expected growth rate of the global viral vector and plasmid manufacturing market during the period between 2020 and 2030? What is the global market size for manufacturing plasmids and different types of viral vectors available in the global viral vector and plasmid manufacturing market in 2019? What are the key trends of the market with respect to different vectors and which vector type is expected to dominate the market during the forecast period 2020-2030? What are the different disease areas where plasmids and viral vectors are employed in the global viral vector and plasmid manufacturing market? Which disease type dominated the market in 2019 and is expected to dominate in 2030? What are the different applications associated with viral vector and plasmid manufacturing? What was the contribution of each of the application areas in the global viral vector and plasmid manufacturing market in 2019, and what is it expected in 2030? Which region is expected to contribute the highest sales to the global viral vector and plasmid manufacturing market during the period between 2019 and 2030? Which region and country carry the potential for significant expansion of key companies in the viral vector and plasmid manufacturing market? What are the leading countries of different regions that contribute significantly toward the growth of the market? Which are the key players of the global viral vector and plasmid manufacturing market, and what are their roles in the market? What was the market share of the key players in 2019?

Market OverviewThe ability of vectors to carry out genetic modification through the introduction of therapeutic DNA/gene into a patient's body or cell has enabled its application in a wide range of modern therapies, including cell and gene therapies.Growing prominence of these therapies in different medical applications has therefore resulted in an increased demand for both viral and non-viral vectors.

Vector-based therapies are currently being used for the treatment of a large number of diseases, including cancer, infectious diseases, genetic diseases, and cardiovascular diseases, among others.Viral vectors and plasmid reduce the cost of treatment and help in decreasing repeated administrations of medications.

Moreover, vectors are also increasingly being used in the field of vaccinology for the development of vaccines owing to the advantage offered by them in inducing a wide range of immune response types. Several players, including biopharmaceutical companies, research institutes, contract manufacturing organizations, and non-profit organizations, have therefore focussed their interest on the development and production of viral vectors and plasmids.

Our healthcare experts have found viral vector and plasmid manufacturing industry to be one of the most rapidly evolving markets, and the global market for viral vector and plasmid manufacturing is predicted to grow at a CAGR of 16.28% over the forecast period of 2020-2030. The market is driven by certain factors, which include success of vector-based cell and gene therapies in treating various therapeutic conditions, increasing number of clinical studies in the field of gene therapy and availability of funding for vector-based gene therapy development, technological advancements in the biomanufacturing sector, and growing investments for expanding vector manufacturing facilities.The market is favoured by the rising prevalence of genetic disorders, cancer, and infectious diseases that has raised the demand for advanced therapeutics and increasing acceptance for comparatively newer treatment options in developing countries.However, the growth of the market is also affected by several factors.

Exorbitant manufacturing cost and highly regulated processes for large-scale vector production are the key challenges cited by industry experts.In addition, lack of required infrastructure and the shortfall of expertise in terms of scale, complexities, and quality assurance for vector production are some of the factors restraining the market growth.

However, rise of contract manufacturers has effectively addressed the above-articulated manufacturing challenges by offering a wide range of vector manufacturing services that offer lucrative opportunities for the growth of the market. Further, increase in research and developmental activities in vector engineering offers strong promise to drive the growth of the viral vector and plasmid manufacturing market in the upcoming years.

Within the research report, the market is segmented on the basis of vector type, application, disease, and region. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Competitive LandscapeThe exponential rise in the application of viral vector and plasmid in various therapies on the global level has created a buzz among companies to invest significantly in viral vector and plasmid manufacturing market.The market is highly competitive, marking the presence of several contract manufacturing organizations and biopharmaceutical companies, who are engaged in in-house vector manufacturing.

Among the different players of the market, Lonza and Thermo Fisher Scientific hold majority of the market share. Other companies contributing significantly toward the growth of the global viral vector and plasmid manufacturing market include GE Healthcare, Fujifilm Holding Corporation, Merck KGaA, Oxford Biomedica plc, Sartorius AG, and Catalent, Inc., among others. On the basis of region, North America holds the largest market share, while Asia-Pacific is anticipated to grow at the fastest CAGR during the forecast period.

Countries Covered North America U.S. Canada Europe U.K. Germany France Spain Italy Switzerland Belgium Rest-of-Europe Asia-Pacific China Japan Australia South Korea India Singapore Rest-of-Asia-Pacific Rest-of-the-World

Read the full report: https://www.reportlinker.com/p05902571/?utm_source=PRN

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Excerpt from:
global market for viral vector and plasmid manufacturing is predicted to grow at a CAGR of 16.28% over the forecast period of 2020-2030 - Olean Times...

This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new era of highly individualized therapies where traditional paradigms of drug development are being significantly challenged.IntroductionBeginning in the mid-1980s and through the last decade, several scientific discoveries have paved the way for ultra-personalized therapeutics. Among these discoveries were the chemical synthesis of oligonucleotides (short bits of DNA and/or RNA), the mapping of the human genome, and the mechanisms of RNA interference (RNAi) and CRISPR (clustered regularly interspace short palindromic repeats) technologies.Having access to the map of the human genome and powerful genetic tools, such as RNAi and CRISPR, has allowed scientists and healthcare and regulatory professionals to forge a new therapeutic frontier. The first step toward this frontier came with the development of personalized medicines, more commonly referred to as gene therapies. In personalized medicines, a gene known to be the cause of a disease state for a significant population of individuals is targeted for therapeutic intervention. The second step pertained to the development of bespoke therapies, often referred to as the next chapter in personalized medicine.A bespoke therapy is a medical treatment that has been tailored to the specific individual characteristics, symptoms, and responses of a patient during all stages of care. The genetic tools developed in recent years have made these individualized therapies a reality. This approach translates into the treatment of a small patient population suffering from rare or ultra-rare genetic diseases for which the therapeutic agent is designed specifically for an individual.In March 2020, the FDA held a workshop on the development of individualized therapeutics to connect with the scientific and regulatory communities to better understand the needs, challenges, and opportunities in the bespoke therapies landscape.1 Based on its insights from the workshop, the agency plans to develop guidance to foster progress in the nascent field while recognizing and protecting the rights of the individual patients who will benefit from these therapies.Traditional models of regulation, clinical trial design, and business development are no longer valid for ultra-personalized therapies. Several questions arise:

Read the original:
Bespoke therapies opportunities, challenges, and hope - Regulatory Focus

Latest market study on Global Gene Therapy Market Forecast to 2027 Covid-19 Impact and Global Analysis By Cell Type (Somatic Gene Therapy, Germline Gene Therapy); By Application (Genetic Disorder, Cancer, Neurological Disorder, and Others).The research report provides deep insights into the global market revenue, parent market trends, macro-economic indicators, and governing factors, along with market attractiveness per market segment. The report provides an overview of the growth rate of the Gene Therapy market during the forecast period, i.e., 20202027. Most importantly, the report further identifies the qualitative impact of various market factors on market segments and geographies. The research segments the market on the basis of product type, application, technology, and region. To offer more clarity regarding the industry, the report takes a closer look at the current status of various factors including but not limited to supply chain management, niche markets, distribution channel, trade, supply, and demand and production capability across different countries.

Request Sample Copy of Gene Therapy Market at: https://www.theinsightpartners.com/sample/TIPHE100001165/

Gene therapy is the introduction of DNA into a patient to treat a genetic disease or a disorder. The newly inserted DNA contains a correcting gene to correct the effects of a disease, causing mutations. Gene therapy is a promising treatment for genetic diseases and also includes cystic fibrosis and muscular dystrophy. Gene therapy is a suitable treatment for infectious diseases, inherited disease and cancer.

The growth of the gene therapy market is regulated due to various reason which includes the rapid involvement of synthetically modified gene to treat various diseases, it helps in designing the personalized medicine, rise in the research and development of the gene therapy among the others. The gene therapy requires less doses of medicines and is one time treatment, this factor is likely to show growth opportunity for gene therapy market in coming near future.

Some of the key players profiled in the study are Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Inc., Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, HORAMA S.A., etc.

The research provides answers to the following key questions:

The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including Medical Device, Pharmaceutical, Healthcare and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Gene Therapy Market globally. This report on Gene Therapy Market provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcase market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

The report profiles the key players in the industry, along with a detailed analysis of their individual positions against the global landscape. The study conducts SWOT analysis to evaluate strengths and weaknesses of the key players in the Gene Therapy market. The researcher provides an extensive analysis of the Gene Therapy market size, share, trends, overall earnings, gross revenue, and profit margin to accurately draw a forecast and provide expert insights to investors to keep them updated with the trends in the market.

Competitive scenario:

The study assesses factors such as segmentation, description, and applications of Gene Therapy industries. It derives accurate insights to give a holistic view of the dynamic features of the business, including shares, profit generation, thereby directing focus on the critical aspects of the business.

Scope of the Report

The research on the Gene Therapy market focuses on mining out valuable data on investment pockets, growth opportunities, and major market vendors to help clients understand their competitors methodologies. The research also segments the Gene Therapy market on the basis of end user, product type, application, and demography for the forecast period 20212027. Comprehensive analysis of critical aspects such as impacting factors and competitive landscape are showcased with the help of vital resources, such as charts, tables, and infographics.

Gene Therapy Market Segmented by Region/Country: North America, Europe, Asia Pacific, Middle East & Africa, and Central & South America

Major highlights of the report:

All-inclusive evaluation of the parent market

Evolution of significant market aspects

Industry-wide investigation of market segments

Assessment of market value and volume in past, present, and forecast years

Evaluation of market share

Study of niche industrial sectors

Tactical approaches of market leaders

Lucrative strategies to help companies strengthen their position in the market

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Original post:
How Gene Therapy Market: Impact Of COVID-19 On Biotechnology Industry Sangamo Therapeutics, Vineti, Solid Biosciences - 3rd Watch News

Double dose: Mice with mutations in both copies of SHANK3 have more behavioral differences than animals with mutations in one copy of the gene.

tiripero / iStock

Correcting a mutation in the autism gene SHANK3 in fetal mice lessens some autism-like behaviors after birth, according to a new study1. The work adds to evidence that gene therapy may help some people with SHANK3 mutations.

In people, mutations in SHANK3 can lead to Phelan-McDermid syndrome, a condition that causes developmental delays and often autism. Up to 2 percent of people with autism have a mutation in SHANK32.

Our findings imply that early genetic correction of SHANK3 has the potential to provide therapeutic benefit for patients, lead investigator Craig Powell, professor of neurobiology at the University of Alabama at Birmingham, wrote in an email.

A 2016 study showed that correcting mutations in SHANK3 in both young and adult mice can decrease excessive grooming, which is thought to correspond to repetitive behaviors in people with autism.

Last year, Powell and his team also showed that correcting SHANK3 mutations in adult mice eliminates some autism-like behaviors3. But the results were difficult to interpret. The team reversed the mutation using an enzyme called Cre-recombinase that could edit SHANK3 if the animals were given a drug called tamoxifen. Control mice in that study that did not receive tamoxifen but had the gene for Cre still showed behavior changes, raising the possibility that the enzyme affected their brains.

In the new work, Powells team used a different approach. They engineered mice with a mutation in both or only one copy of SHANK3 the latter more closely mirrors what happens in people. Some animals had the Cre gene, but some also had another gene for a Cre-activating protein that is naturally expressed when the animals are in utero. By using this protein, the researchers could avoid using tamoxifen, which some studies have shown may also cause behavioral changes in mice4.

The control mice had either the gene for Cre-recombinase or fortheCre-activating protein, but not both, allowing the researchers to isolate any effects from the method itself.

They found that correcting the mutation lowers some but not all of the animals autism-like behaviors, a finding Powell says is surprising. The mice groom less and are more social by some measures, but they still prefer interacting with an object than with another mouse.

We dont really know why some behaviors are affected and not others, Powell says.

Mice with one mutated copy of SHANK3 have fewer behavioral differences than mice with two, they also found, which indicates the value of using both kinds of animals in gene-reversal studies, experts say.

The fact that they did analyze both side by side, and they did see some differences, I find quite intriguing, says Gaia Novarino, professor of neuroscience at the Institute of Science and Technology in Klosterneuburg, Austria.

The team originally planned to consider when and where in the brain SHANK3 was corrected. But the Cre-activating protein involved in the study was expressed throughout the brain, preventing region-specific findings.

The team gave some mice the antibiotic doxycycline to suppress Cre expression, in hopes of also testing the effects of correcting SHANK3 in adulthood. But the method failed, for unknown reasons.

It is also important to publish experiments that do not work out exactly as planned, Powell says.

The teams openness about the studys shortcomings could help others design their own studies or re-evaluate previous work, says Yong-Hui Jiang, chief of medical genetics at Yale University.

People will learn from the difficulties and the experience, Jiang says.

It would still be helpful to test whether correcting SHANK3 mutations can reverse autism-like behaviors in adult mice without using tamoxifen, other researchers say.

Its beneficial to do experiments in such a way where you leave very little room for alternative interpretations, says Gavin Rumbaugh, professor of neuroscience at the Scripps Research Institute in Jupiter, Florida. He suggests using a mouse that does not express Cre until the animal is administered doxycycline, rather than trying to suppress Cre with the drug.

The work lends credence to the idea that gene therapy might alleviate some difficulties associated with autism in people with SHANK3 mutations, researchers say. Further studies could also investigate in how many cells the gene needs to be restored to change behavior, and what would be the safest and most effective stage of development to intervene with a gene therapy.

The impression is you have a quite large window, Novarino says. Thats quite positive.

More:
Reversing SHANK3 mutations in mice mitigates autism-like traits - Spectrum

Gene therapy is an experimental treatment that involves introducing genetic material into a persons cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinsons disease, cancer and even HIV, through a number of different approaches. A gene can be delivered to a cell using a carrier known as a vector. The most common types of vectors used in gene therapy are viruses.

Scope of the Report:This report focuses on the Gene Therapy in global market, especially in North America, Europe, Asia-Pacific, South America, Middle East and Africa. This report categorizes the market based on manufacturers, regions, types and applications.

The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points within the industrys value chain.

No. Of Pages 130

Report Covers Market Segment by Manufacturers:

Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.Advantagene

Market Segment by Type, covers:

Ex vivoIn Vivo

Market Segment by Applications, can be divided into:

CancerMonogenicInfectious diseaseCardiovascular diseaseOther

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There are 15 Chapters to deeply display the global Gene Therapy market.

Chapter 1: Describe Gene Therapy Introduction, product scope, market overview, market opportunities, market risk, market driving force.

Chapter 13, 14 and 15: Describe Gene Therapy sales channel, distributors, traders, dealers, appendix and data source.

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Excerpt from:
Gene Therapy Market 2020 Industry Size, Regions,Trends and Top Manufacturers Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics,...