Category Archives: Gene Medicine

Global Gene Editing Market: Snapshot

Gene editing or genome editing is the targeted insertion or modification of cells in living organism or cells and the method has come to occupy a crucial part of biomedical researches, constantly transforming various disciplines of life sciences. Over the past few years, continuous advancements in gene-editing technologies have led to the advent of several versatile methods, which have enabled investigators to introduce a number of sequence-specific changes into the genomes of a variety of cell types. This has facilitated the discovery of promising human gene therapies proving useful for treating various diseases. The use of targeted nucleases or engineered nucleases in laboratories has provided researchers potential tools to economically and rapidly manipulate almost any genomic sequence for a broad range of cell types.

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In recent years, gene editing techniques have been witnessed a paradigmatic shift with the advent of methods such as clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), zinc-finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs). Using these technologies, investigators have been successful in generating a wide spectrum of outcomes, which are likely to prove useful in diverse areas as synthetic biology, disease modeling, neurosciences, and drug discovery. For instance, targeted nuclease have enabled the insertion of targeted DNA double-strand breaks (DSBs). This has led to the activation of DNA repair pathways in various cells. In vivo applications of various gene editing tools, however, suffer from noticeable constraints. For instance, nuclease delivery or expression can be enabled only in diseased cells, thereby limiting potential of the market to an extent. Nevertheless, constant engineering advances are being made which will expectedly lay robust groundwork for expanding the current array of genome-modifying tools which may drive gene editing market.

Gene editing involves the insertion, deletion, or replacement of DNA at specific sites in the genome of a cell or an organism. It is usually achieved in a laboratory environment using molecular scissors.

Regulations for the security of life, well-being of plants and animals, human health, and environmental compliance will influence market players for increased focus on import, export, and commercialization of genetically modified organisms (GMOs).

The report is an all-important tool to comprehend the various factors and growth trends that will influence the growth of the gene editing market between 2017 and 2025. The market study is a collective of facts and factoids that are associated with the global gene editing market in a chronological order. The analysis of past data and current market trends enable research analysts to present a satisfactory conclusion regarding the markets future. Thus, the analysis presented in the report can be used to devise successful business strategies for the future. Using standard analytical tools such as Porters Five Forces and SWOT analysis, the report presents the indices of strength, weakness, opportunities, and threats of the global gene editing market until the end of the forecast period in 2025.

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The increasing expenditures on research and development, growth of the biotechnology and pharmaceutical companies, and rising demand for synthetic genes are the major factors driving the global gene editing market.

The increasing prevalence of infectious diseases, cancer, and other genetic disorders is steering the growth of the gene editing industry. Moreover, the increasing demand for personalized medicine and advancement of medical science is propelling the industrys demand.

However, strict government regulations to receive approval for mutation undertakings and lack of public awareness will challenge the growth of the gene editing market. Government regulations for assessing the medical benefits as well as the potential hazards of gene editing procedure will benefit the growth of this market.

The global gene editing market can be analyzed on the basis of technology, end user, application, and region. In terms of technology, the market can be segmented into CRISPR, ZFN, TALEN and others. On the basis of application, the global gene editing market can be divided into cell line engineering, plant genetic engineering, animal genetic engineering, and others. By end user, the market can be segmented into biotechnology and pharmaceutical companies, contract research organizations, and academic and government institutes.

The global gene editing market can be divided into the regional segments of North America, Europe, Asia Pacific, and Rest of the World. The U.S. gene editing market is expected to display robust growth due to growth trend manifested by biotechnology and pharmaceutical companies and adoption of advanced technologies such as CRISPR for treating chronic hereditary diseases.

In Europe, the U.K. is expected to contribute significantly to the growth of the gene editing market in this region. This is mainly due to the rising geriatric population and increasing incidence of chronic diseases. The Asia Pacific gene editing market is expected to display fast growth rate in the coming years. The rising geriatric population, modernization of healthcare practices, technological advancements, and government initiatives for controlling diseases are fuelling the growth of the Asia Pacific gene editing market.

South Africa is expected to contribute significantly to the revenue of its regional market. The rising prevalence of sickle cell anemia, HIV, hemophilia and several forms of cancer will drive the industrys growth.

The report mentions and profiles some of the top companies in the global gene editing market, namely Agilent Technologies, AstraZeneca, Cellectis, Editas Medicine, Dharmacon, Qiagen, Sigma-Aldrich, Allele Biotech, Bio Rad, CRISPR Therapeutics, GE Healthcare, Lonza, Recombinetics, and Thermo Fisher Scientific.

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Originally posted here:
Gene Editing Market Poised to Expand at a Robust Pace Over 2025 - 3rd Watch News

New test may improve COVID-19 diagnosis

By Alexis Kessenich

Many unknowns remain with the novel coronavirus, including diagnostic testing. Some tests have reported a false negative rate as high as 40%, incorrectly informing patients that they do not have a SAR-CoV-2 infection the virus that causes COVID-19 when they actually do. Without accurate testing, clinicians cant properly treat their patients, and the public may not take additional protective measures, like self-isolating, to keep others safe.

Before the coronavirus pandemic hit, the RADICAL (Rapid Diagnostics in Categorizing Acute Lung Infections) study team, led byEphraim Tsalik, was developing a new diagnostic test based on host gene expression to allow clinicians to differentiate between bacterial and viral infections. This test would allow clinicians to better distinguish who has a bacterial infection and more accurately prescribe antibiotics, leading to a decrease in overall antibiotic usage and antibiotic resistance.

Now, thanks to a $250,000 grant from theAntibacterial Resistance Leadership Group, the team has shifted its focus to examine whether their host gene expression test works for patients with COVID-19.

Tsalik and his team will enroll patients with COVID-19 in their study to evaluate whether the host gene expression test works in that population and generate new gene expression data to answer other questions, such as how the biology of the novel coronavirus compares to other infections.

Were finding that there are some really unique elements of COVID-19 compared to other respiratory viral infections, said Tsalik. But if we can identify it using a complementary strategy focusing on the host response, it could significantly improve our ability to accurately identify people who have the infection.

The team has already shown that using a host response strategy for other viruses can accurately detect people who are pre-symptomatic, which is difficult to identify otherwise. If their test works in patients with COVID-19, it could not only help decrease the rate of false negatives, but could also give clinicians a new tool to help with contact tracing and quarantining.

Current tests also cannot determine who is at risk for a severe case of the disease. While some will only have a mild case, others will progress into a much more severe illness. These questions, though, could be answered using the host gene expression approach.

Even if the team finds their current test isnt applicable to COVID-19 patients, they will explore other modifications. It may not be the current iteration of the RADICAL test, Tsalik said, but the strategies we used to develop the test will be essential to answering some of the pressing questions associated with this very unique disease.

The team is still enrolling participants in their study. If you or someone you know is interested in joining, visit theMESSI study pageto learn more.

New translational research: Blood test can tell if antibiotics are needed

Originally posted here:
Uncovering the Unknowns to COVID-19 Testing | Center for Applied Genomics and Precision Medicine - Duke Today

The Latest Research Report on Genetic Engineering Market size | Industry Segment by Applications, by Type, Regional Outlook, Market Demand, Latest Trends, Genetic Engineering Industry Share & Revenue by Manufacturers, Company Profiles, Growth Forecasts 2025. Analyzes current market size and upcoming 5 years growth of this industry.

The report presents a highly comprehensive and accurate research study on the globalGenetic Engineering market. It offers PESTLE analysis, qualitative and quantitative analysis, Porters Five Forces analysis, and absolute dollar opportunity analysis to help players improve their business strategies. It also sheds light on critical Genetic Engineering Marketdynamics such as trends and opportunities, drivers, restraints, and challenges to help market participants stay informed and cement a strong position in the industry. With competitive landscape analysis, the authors of the report have made a brilliant attempt to help readers understand important business tactics that leading companies use to maintainGenetic Engineering market sustainability.

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Global Genetic Engineering Market to reach USD XX billion by 2025.

Global Genetic Engineering Market valued approximately USD XX billion in 2017 is anticipated to grow with a healthy growth rate of more than XX% over the forecast period 2018-2025. The major driving factor of global Genetic Engineering market are surging utility of technologies such as CRISPR, Talen & ZNF and rising focus on innovation in Gene Therapy in Genetic Engineering. In addition, increasing funding for research and development of medical products is the some other driving factor that drives the market. However, one of the major restraining factors of Genetic Engineering market is high amount of investment. Genetic engineering is also known as genetic modification or genetic manipulation. It is the direct manipulation of an organisms genes using biotechnology. It is a set of technologies used to change the genetic makeup of cells, including the transfer of genes within and across species boundaries to produce improved or novel organisms. Genetic engineering allows of plant or animals to be modified so their maturity can occur at a quicker pace. Genetic modification can also help to create resistance to common forms of forms of organism death. Genetic engineering can also change the traits of plants or animals so that they produce greater yield per plant. Any genetic mutation caused by environmental mutagens may also be corrected through genetic engineering.

The regional analysis of Global Genetic Engineering Market is considered for the key regions such as Asia Pacific, North America, Europe, Latin America and Rest of the World. North America has dominate the market of total generating revenue with 40% across the globe in 2016 due to increasing use of genetic engineering for use of gene therapy, high incidence of cancer and increasing awareness for the use of stem cells. Europe is also contributing second largest major share in the global market of Genetic Engineering. Asia-Pacific region is also anticipated to exhibit higher growth rate / CAGR over the over the coming years due to presence of developing countries, companies grabbing these opportunities and extracting their presence in the region. The Middle East and Africa holds the least share in global genetic engineering market owing to limited availability of medicine facilities.

The major market player included in this report are:

Thermo Fisher Scientific Inc.

Merck KGAA

Horizon Discovery Group Plc

Transposagen Biopharmaceuticals Inc.

New England Biolabs

Genscript Biotech Corporation

Lonza Group Ltd.

Origene Technologies Inc.

Integrated DNA Technologies Inc.

Amgen Inc.

The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall also incorporate available opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and product offerings of key players. The detailed segments and sub-segment of the market are explained below:

By Devices:

oPCR

oGene Gun

oGel Assemblies

oOthers

By Techniques:

oArtificial Selection

oGene Splicing

oCloning

oOthers

By End-User:

oResearch Institutes

oAcademic Institutes

oPharmaceutical Industries

oOthers

By Application:

oAgriculture

oMedical Industry

oForensic Science

oOthers

By Regions:

oNorth America

oU.S.

oCanada

oEurope

oUK

oGermany

oAsia Pacific

oChina

oIndia

oJapan

oLatin America

oBrazil

oMexico

oRest of the World

Furthermore, years considered for the study are as follows:

Historical year 2015, 2016

Base year 2017

Forecast period 2018 to 2025

Target Audience of the Global Genetic Engineering Market in Market Study:

oKey Consulting Companies & Advisors

oLarge, medium-sized, and small enterprises

oVenture capitalists

oValue-Added Resellers (VARs)

oThird-party knowledge providers

oInvestment bankers

oInvestors

Have Any Query Or Specific Requirement?Ask Our Industry Experts!

Table of Contents:

Study Coverage:It includes study objectives, years considered for the research study, growth rate and Genetic Engineering market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary:In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Genetic Engineering market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Genetic Engineering Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region:It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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Genetic Engineering Market Growing Rapidly with Significant CAGR, Leading Players, Innovative Trends and Expected Revenue by 2025 - Cole of Duty

News Release

Thursday, July 9, 2020

Comparing epigenetic differences between humans and domestic dogs provides an emerging model of aging.

One of the most common misconceptions is that one human year equals seven dog years in terms of aging. However, this equivalency is misleading and has been consistently dismissed by veterinarians. A recent study, published in the journalCell Systems, lays out a new framework for comparing dog-to-human aging. In one such comparison, the researchers found the first eight weeks of a dogs life is comparable to the first nine months of human infancy, but the ratio changes over time. The research used epigenetics, a process by which modifications occur in the genome, as a biological marker to study the aging process. By comparing when and what epigenetic changes mark certain developmental periods in humans and dogs, researchers hope to gain specific insight into human aging as well.

Researchers performed a comprehensive analysis and quantitatively compared the progression of aging between two mammals, dogs and humans. Scientists at the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and collaborators at the University of California (UC) San Diego, UC Davis and the University of Pittsburgh School of Medicine carried out the research.

All mammals experience the same overarching developmental timeline: birth, infancy, youth, puberty, adulthood and death. But researchers have long sought specific biological events that govern when such life stages take place. One means to study such a progression involves epigenetics gene expression changes caused by factors other than the DNA sequence itself. Recent findings have shown that epigenetic changes are linked to specific stages of aging and that these are shared among species.

Researchers focused on one type of epigenetic change called methylation, a process in which molecules called methyl groups are attached to particular DNA sequences, usually parts of a gene. Attaching to these DNA regions effectively turns the gene into the "off" position. So far, researchers have identified that in humans, methylation patterns change predictably over time. These patterns have allowed the creation of mathematical models that can accurately gauge the age of an individual called "epigenetic clocks."

But these epigenetic clocks have only been successful in predicting human age. They do not seem to be valid across species, such as in mice, dogs, and wolves. To see why the epigenetic clocks in these other species differed from the human version, researchers first studied the epigenetic changes over the lifetime of a domestic dog and compared the resultsobtained with humans.

Dogs are a useful model for such comparisons because much of their environment, diet, chemical exposure, and physiological and developmental patterns are similar to humans.

"Dogs experience the same biological hallmarks of aging as humans, but do so in a compressed period, around 10 to 15 years on average, versus over 70 years in humans. This makes dogs invaluable for studying the genetics of aging across mammals, including humans," said Elaine Ostrander, Ph.D., NIH Distinguished Investigator and co-author of the paper.

Dr. Ostrander and her colleagues in Trey Ideker's laboratory at UC San Diego took blood samples from 104 dogs, mostly Labrador retrievers, ranging from four weeks to 16 years of age. They also obtained previously published methylation patterns from 320 people, whose ages ranged from 1 to 103 years. The researchers then studied and compared the methylation patterns from both species.

Based on the data, researchers identified similar age-related methylation patterns, specifically when pairing young dogs with young humans or older dogs with older humans. They did not observe this relationship when comparing young dogs to older humans and vice versa.

The study also found that groups of specific genes involved in development can explain much of the similarity, which had similar methylation patterns during aging in dogs and humans.

"These results suggest that aging can, in part, be explained by a continuum of changes beginning in development," said Dr. Ideker. "The programs of development are expressed incredibly strongly at defined periods when the pup is in the womb and childhood. But equally strongly are systems that clamp down to stop it. In a sense, we are looking at aging as the residual 'afterburn' of those powerful forces."

The researchers also attempted to correlate the human epigenetic clock with dogs, using this as a proxy for converting dog years to human years.

The new formula is more complicated than the "multiply by seven" method. When dogs and humans experience similar physiological milestones, such as infancy, adolescence and aging, the new formula provided reasonable estimates of equivalent ages. For example, by using the new formula, eight weeks in dogs roughly translates to nine months in humans, which corresponds to the infant stage in both puppies and babies. The expected lifespan of senior Labrador retrievers, 12 years, correctly translates to 70 years in humans, the worldwide average life expectancy.

The group acknowledges that the dog-to-human years formula is largely based on data from Labrador retrievers alone. Hence, future studies with other dog breeds will be required to test the formula's generalizability. Because dog breeds have different life spans, the formula may be different among breeds.

Dr. Ostrander noted, "It will be particularly interesting to study long-lived breeds, a disproportionate number of which are small in size, versus breeds with a shorter lifespan, which includes many larger breeds. This will help us correlate the well-recognized relationship between skeletal size and lifespan in dogs."

The study also demonstrates that studying methylation patterns may be a useful method to quantitatively translate the age-related physiology experienced by one organism (e.g., humans) to the age at which physiology in a second organism is most similar (e.g., dogs). The group hopes that such translation may provide a useful tool for understanding aging and identifying ways to maximize healthy lifespans.

"This study, which highlights the relevance of canine aging studies, further expands the utility of the dog as a genetic system for studies that inform human health and biology," said Dr. Ostrander.

This press release describes a basic research finding. Basic research increases our understanding of human behavior and biology, which is foundational to advancing new and better ways to prevent, diagnose, and treat disease. Science is an unpredictable and incremental process each research advance builds on past discoveries, often in unexpected ways. Most clinical advances would not be possible without the knowledge of fundamental basic research.

NHGRI is one of the 27 institutes and centers at the National Institutes of Health. The NHGRI Extramural Research Program supports grants for research, and training and career development at sites nationwide. Additional information about NHGRI can be found at https://www.genome.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

###

Excerpt from:
NIH researchers reframe dog-to-human aging comparisons - National Institutes of Health

July 08, 2020 -In the largest study of its kind, researchers discovered hundreds of novel genetic variants linked to type 2 diabetes, potentially improving care for millions living with this disease.

A team from the Perelman School of Medicine at the University of Pennsylvania and the Veterans Health Administrations (VHA) Corporal Michael J. Crescenz Veterans Affairs Medical Center (CMCVAMC) examined the genes of more than 200,000 people around the world with type 2 diabetes.

In addition to uncovering new genetic variants linked to the condition, researchers identified gene variants that vary by ethnicity, as well as variants tied to conditions related to type 2 diabetes like coronary heart disease and chronic kidney disease.

The group used data from the worlds largest biobank, the Million Veteran Program (MVP) in the VHA, as well as data from the DIAGRAM Consortium, the UK Biobank, the Penn Medicine Biobank, and Biobank Japan. Researchers analyzed a study population of 1.4 million people around the world, of whom almost 230,000 had type 2 diabetes.

The team then broke down the genetic makeup of those hundreds of thousands with type 2 diabetes and found 558 independent genetic variants that are differentially distributed between people with and without type 2 diabetes. Twenty-one of these variants were specific to European ancestry while seven were specific to African American ancestry. Of the 558 variants found, 286 had never been discovered.

Researchers set out to discover if certain genetic variants among this group of people could be linked to specific type 2 diabetes-related conditions.

Ultimately, three were linked to coronary heart disease, two to acute ischemic stroke, four to retinopathy, two to chronic kidney disease, and one to neuropathy, saidMarijana Vujkovic, PhD, a biostatistician at both the Perelman School of Medicine at the University of Pennsylvania, VHAs CMCVAMC and a co-leader for the VHAs national MVP Cardiometabolic Working Group.

Building on this research, the scientific community can assess which of the surrounding genes nearby the identified genetic variants is likely to be the causal gene that alters the risk of type-2 diabetes, and that could lead to early interventions to limit controllable risks of developing the condition.

While the researchers found many genetic variants in people with type 2 diabetes, no one variant was labeled as the worst or most dangerous.

However, just like heart disease, schizophrenia, or obesity, it is the accumulation of a large number of these variants that can add up to a considerable increase in risk, said co-senior authorBenjamin F. Voight, PhD, an associate professor of Systems Pharmacology and Translational Therapeutics at Penn, and a co-leader for the VHAs national MVP Cardiometabolic Working Group.

We hope this study can not only help find that subset of patients with substantial risk, but also to motivate new, future studies for treatments based on these findings.

Knowing more about the genetic variants linked to type 2 diabetes could help identify potential therapeutic targets for type 2 diabetes. Researchers also noted that this information could help guide treatment plans for people with the disease who may be susceptible to specific diabetes complications.

Going forward, the researchers plan to conduct a long-term examination of how genetics influence disease progression among patients with type 2 diabetes and associated metabolic disorders. The group is also leveraging the list of newly-discovered genes to investigate medication interactions.

Knowing the genetic susceptibility for diabetes complications in a patient already diagnosed with type-2 diabetes, for example through a cumulative genetic risk score, could help guide that patients care, said co-senior-authorKyong-Mi Chang, MD, a professor of Medicine at Penn, Associate Chief of Staff for Research at VHAs CMCVAMC and the Co-PI for the VHAs MVP Merit Award that supported this work.

As clinicians, we hope that these findings can ultimately be applied to improve the health outcomes for our patients including veterans.

Go here to read the rest:
Researchers Discover Genetic Variants Linked to Type 2 Diabetes - HealthITAnalytics.com

Part 2 of 3. Read Part 1.

Humanity has been on a very steep learning curve since, reacting to a 31 December 2019 report from the Wuhan Municipal Health Commission that they were seeing an atypical pneumonia of unknown cause, the World Health Organisation (WHO) published (5 January, 2020) the first Disease Outbreak Bulletin to alert the public health community and the global media to the disease we call COVID-19. From the outset, both the WHO and the local virologists were primed by the 2002/3 SARS-CoV-1 experience to think that a coronavirus might be involved.

That was confirmed when Chinese investigators isolated the SARS-CoV-2 virus that causes COVID-19 on 7 January and made the gene sequence available globally a couple of days later. Knowing the viral RNA sequence enabled those who already had appropriate platform technologies to start right away with the job of making vaccines. It also allowed Mike Catton and Julian Druce of The Royal Melbourne Hospitals Victorian Infectious Disease Reference Laboratory (VIDRL) that is part of our Institute to add a sensitive PCR (polymerase chain reaction) test specific for SARS-CoV-2 to the more broadly reactive assay established earlier to detect the SARS-CoV-1 and MERS viruses, neither of which caused disease in Australia. Variants of the SARS-CoV-2 PCR are the tests that, as we all understand, have allowed the identification of currently infected individuals that facilitates targeted control strategies.

Arriving in Melbourne from China on 19 January, a returned resident very responsibly alerted authorities several days later to the fact that he was symptomatic and could be infected with this new virus. The diagnosis was immediately confirmed by PCR and, by Australia Day (26 January), Mike, Julian and teams had recovered infectious SARS-CoV-2 from him and announced that that the virus would be made available globally to legitimate laboratories. That was important: having fully infectious virus meant that researchers and diagnosticians could, for example, do virus neutralisation tests to measure protective antibodies in serum, screen chemical compounds (small molecules) for possible antiviral effects in tissue culture and challenge immunised animals to test for vaccine efficacy.

So, here are a couple of very big lessons that those of us who work in science understood but some in government and many in the broader community may not have fully appreciated.

The international co-operation that is essential for all science, and especially for public health science, functioned for COVID-19 from the outset and has, in fact, continued to work. The WHO did its job of alerting responsible individuals and agencies to the fact that there could be a problem, the Chinese gave out the gene sequence as soon as they had it, and our Institute was the first to provide the infectious virus globally for key laboratory tests. Locally, the communication mechanisms between public health professionals and elected officials proved fit for purpose. The Australian government was immediately aware of the potential threat and the Minister for Health, Greg Hunt, made a public announcement on 25 January that we had recorded our index case.

Additional to that, the very big lesson we should all take on board here is that modern science protects and serves us. Though everyone understood that the catastrophic influenza pandemic of 1918/19 was caused by a virus, diagnosis back then was all symptomatic, no human influenza virus was isolated until 1933 and it was only during World War 2 (1939-45) that the first, primitive influenza vaccines were rolled out to protect the troops against the possibility of a repeat pandemic that, thankfully, did not occur. When it comes to SARS-CoV-2 and COVID-19 we had a specific diagnostic test within days and, I will personally be very surprised if large-scale human vaccination is not in full swing by the second half of 2021.

Even so, the big lesson for the public is that, no matter how wonderful the laboratory science, actually getting products out there to protect people is a much more cumbersome process. Ensuring that a novel drug or vaccine is safe and efficacious takes time. Even though regulatory authorities have been comfortable with the idea that preliminary trials in animals and small numbers of human volunteers (Phase 1) can be conducted simultaneously, all that information must be evaluated before any product can be given to substantial numbers of people. Every possible effort is being made to ensure that all participants in large, closely monitored Phase 2 then Phase 3 trials will be protected, or at least safe, following community exposure to SARS-CoV-2.

Much of what had to be done over this first six months of the COVID-19 challenge was just plain hard work. An enormous effort was, for example, made within VIDRL to build testing capacity by helping other private and public laboratories get up to speed. And the Institute is still in the process of evaluating rapid person-side antibody tests that can be used for large-scale serological surveys. The obvious lesson here is that we are protected by having well-funded, high quality public laboratories and Institutions that can rapidly build capacity in the face of any pandemic threat. Next week, Ill finish my assessment of the lessons weve learned so far, to return to that six months from now.

This article is the latest in theSetting it Straightseries written by Laureate ProfessorPeter Dohertyfrom Australias University of Melbourne and Doherty Institute to explain aspects of the evolving COVID-19 pandemic. You can read them allhere.

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COVID-19: The lessons learned to date - Cosmos

NEW YORK and ROME, July 13, 2020 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID, hereinafter Ovid), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, and Angelini Pharma S.p.A. (hereinafter Angelini Pharma), an Italian family-owned pharmaceutical company committed to helping patients with a constant and prevalent focus on Mental Health, Rare Diseases and Consumer Health, announced an agreement in which Angelini Pharma will be responsible to develop, manufacture and commercialize OV101 (gaboxadol) for the potential treatment of Angelman syndrome in the European Union and other countries in the European Economic Area (Switzerland, Turkey and the United Kingdom) and Russia. Angelini Pharma will execute the agreement through its new affiliate Angelini Pharma Rare Diseases AG. OV101 is believed to be the only delta ()-selective GABAA receptor agonist in development and is currently being evaluated in the pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline results expected in the fourth quarter of 2020.

Under the terms of the agreement, Ovid will receive an upfront payment of $20 million and is eligible to receive up to an additional $212.5 million in payments upon the achievement of development, manufacturing and sales milestones for the initial indication (Angelman syndrome), as well as double-digit royalties on net sales if OV101 is successfully commercialized. Ovid will retain all U.S. and rest-of-world commercial rights to OV101.

We are excited to enter into a strategic collaboration with Angelini Pharma with the goal of bringing OV101, if approved, to the Angelman community in Europe as quickly as possible. Angelini Pharma is an ideal partner for Europe as they have deep regional knowledge, an established infrastructure with a history of successful product launches, and a commitment to improving the quality of life of the patient communities they serve, said Jeremy Levin, DPhil, MB, BChir, Chairman and Chief Executive Officer of Ovid Therapeutics. Finding the right partners to bring OV101 to the Angelman community as rapidly as possible is a core part of our global strategy. We believe this partnership with Angelini will help to maximize the potential commercial value of OV101 and achieve our strategic objectives in this important geography."

Today is a day that we will remember. Through our collaboration with Ovid Therapeutics, we are laying the foundation to developing innovative health solutions for rare diseases, in line with Angelini Pharmas new strategy, said Pierluigi Antonelli, Angelini Pharma CEO. The new business unit Angelini Pharma Rare Diseases AG will contribute to the development, registration, production and, if approved, commercialization in Europe of OV101, Ovid Therapeutics very promising drug being evaluated in a Phase 3 clinical trial for the treatment of Angelman syndrome. As of now, there is no effective treatment for this rare genetic disease, characterized by severe psychomotor disability, which manifests itself from childhood. Delivering on our commitment makes us proud both from a scientific and social impact perspectives.

"As shareholders and executives of Angelini Holding we continue to invest in the pharma area, which today represents half of our Group's turnover, commented the executive vice president Thea Paola Angelini and the CEO Sergio Marullo di Condojanni. Our global development and internationalization strategy focuses on business areas with high growth potential. Particularly, we look closely at all the opportunities that can open up, not only in healthcare, but also in the consumer and machinery sector."

Rothschild & Co acted as an advisor to Ovid on the collaboration agreement.

About Angelman Syndrome Angelman syndrome is a rare genetic condition that is characterized by a variety of signs and symptoms. Characteristic features of this condition include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. Individuals with Angelman syndrome typically have normal lifespans but are unable to live independently. Therefore, they require constant support from a network of specialists and caregivers. Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000 people globally.

There are no approved therapies by the U.S. Food and Drug Administration (FDA), European Medicines Agency or restof-world for Angelman syndrome, and treatment primarily consists of behavioral interventions and pharmacologic management of symptoms.

Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta ()-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.

About OV101 (gaboxadol)OV101 is believed to be the only delta ()-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has demonstrated in laboratory studies and animal models to selectively activate the -subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through modulation of tonic inhibition.

Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and thereby address several core symptoms of these conditions. In both these syndromes, the underlying pathophysiology includes disruption of tonic inhibition modulated through the -subunit of GABAA receptors. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in more than 4,000 patients (more than 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles. Ovid is conducting a pivotal Phase 3 clinical trial with OV101 in Angelman syndrome (NEPTUNE) and has completed a Phase 2 signal-finding clinical trial with OV101 in Fragile X syndrome (ROCKET).

OV101 has received Rare Pediatric Disease Designation from the FDA for the treatment of Angelman syndrome. The FDA has also granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. In addition, the European Commission (EC) has granted orphan drug designation to OV101 for the treatment of Angelman syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035 without regulatory extensions.

Ovid Conference Call and Webcast InformationOvid Therapeutics will host a live conference call and webcast today at 8:15 a.m. Eastern Time. The live webcast can be accessed by visiting the Investors section of the Companys website at https://investors.ovidrx.com/news-events/presentations-events. Alternatively, please call 866-830-1640 (U.S.) or 210-874-7820 (international) to listen to the live conference call. The conference ID number for the live call is 5579257. A replay of the webcast will be available on the Companys website following the live conference call.

About Ovid TherapeuticsOvid Therapeutics Inc. is a New York-based biopharmaceutical company using its BoldMedicine approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The Companys most advanced investigational medicine, OV101 (gaboxadol), is currently in clinical development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935 (soticlestat) in collaboration with Takeda Pharmaceutical Company Limited for the potential treatment of rare developmental and epileptic encephalopathies (DEE). For more information on Ovid, please visit http://www.ovidrx.com.

About Angelini PharmaAngelini Pharma, owned by Angelini Holding, is a pharmaceutical Company committed to helping patients with a constant and prevalent focus on Mental Health, including Pain, Rare Diseases and Consumer Health. Angelini Pharma has an extensive and recognized R&D programs, "World Class" production plants and international commercialization activities of active ingredients and market-leading drugs. For further information, please visit http://www.angelinipharma.com

About Angelini HoldingAngelini Holding is the parent company of an international group operating in the pharmaceutical and consumer goods sectors. Founded in Italy in 1919, today Angelini group operates in 17 countries with a staff of 5,600 and a turnover of 1,7 billion. In addition to the Pharmaceutical sector, Angelini group operates in Personal and Home Care business area through Fater, a joint venture with Procter & Gamble, in the Machinery field, again in joint venture with P&G, with the group operating in automation and robotics for the consumer goods industry Fameccanica, in Perfumery and Skincare and Suncare with Angelini Beauty and in the Wine sector through Bertani Domains. Angelini Holding has recently entered the Baby food market as well through MadreNatura, a joint venture with Hero Group, which offers 100% organic baby food products.

Ovid Therapeutics Forward-Looking StatementsThis press release includes certain disclosures that contain forward-looking statements, including, without limitation, statements regarding: advancing development of and commercializing OV101, the potential benefits and value of OV101; the anticipated reporting schedule of clinical data for OV101; and the potential benefits and outcome from this collaboration. You can identify forward-looking statements because they contain words such as will, appears, believes and expects. Forward-looking statements are based on Ovids current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include uncertainties in the development and regulatory approval processes, and the fact that initial data from clinical trials may not be indicative, and are not guarantees, of the final results of the clinical trials and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovids filings with the Securities and Exchange Commission under the caption Risk Factors. Such risks may be amplified by the COVID-19 pandemic and its potential impact on Ovids business and the global economy. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Ovid Therapeutics Contacts

Investors and Media:Ovid Therapeutics Inc.Investor Relations & Public Relationsirpr@ovidrx.com

Or

Investors: Steve KlassBurns McClellan, Inc.sklass@burnsmc.com(212) 213-0006

Media:Katie Engleman1ABkatie@1abmedia.com

Angelini Pharma Contact:Daniela PoggioHead of Global Communications Angelini Pharma+39 348 6558882daniela.poggio@angelinipharma.com

Angelini Holding Contact:Institutional & External Relations Director Angelini Holding+39 348 6707240alessandra.favilli@angeliniholding.com

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Ovid Therapeutics and Angelini Pharma Enter into Exclusive License Agreement to Develop, Manufacture and Commercialize OV101 for the Treatment of...

The global market for DNA read, write and edit applications should grow from $17.0 billion in 2019 to $43.1 billion by 2024 with a compound annual growth rate (CAGR) of 20.5% during the period of 2019-2024.

Report Scope:

The scope of the report includes DNA read, write and edit technologies, applications, industries, initiatives, patents and companies. The markets for read, write and edit products and services are given for 2017, 2018, 2019 (estimated) and 2024 (forecast).

This report reviews the main read, write and edit technologies and explains why genetic variation is important in clinical testing and disease. It then discusses significant large-scale research initiatives that impact read, write and edit applications. Of particular interest is a discussion of population-scale sequencing projects throughout the world, and their likely impact. The main market driving forces for read, write and edit products and services are listed and discussed.

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The report quantifies each of the main market segments. The read (sequencing) market is quantified by delivered format, including sequencing workflow products (sample preparation kits and reagents, sequencing instruments and consumables, and informatics) and sequencing services (clinical diagnostics and sequencing services to applied market customers).

The sequencing workflow products market is quantified by type, that is, DNA isolation and extraction; target enrichment; library preparation; and informatics/ecosystems. The sequencing instruments and consumables market is given by platform (Sanger, NGS and 3GS).

The sequencing services market is analyzed by end user application (applied, clinical, and R&D). Within sequencing services, the applied market is analyzed by end-user application (agriculture, biopharma, consumer, microbiology, population-scale genomics, synthetic biology and other).

Also within sequencing services, the clinical market is analyzed and quantified by disease category (cardiovascular, clinical microbiology and infectious diseases, Mendelian disorders, metabolic/immune disorders, neurology, oncology, reproductive health and transplant medicine).

The DNA write (synthesis) market is quantified by product type (oligonucleotides, synthetic biology parts, genes and RNA therapeutics). The oligonucleotide market is analyzed by application (gene editing, sequencing, PCR, FISH, microarray, gene synthesis and other). The gene market is quantified by gene type (standardized, value-added). Finally, the RNA therapeutics market is quantified by platform (RNA interference, antisense oligos, micro RNA modulation and mRNA) and by disease category (cancer, hematology, musculoskeletal, neurology and rare diseases).

The DNA edit (gene editing) market is quantified by application (agriculture, biopharma, diagnostics and therapeutics); editing platform (CRISPR, meganuclease, TALEN, ZFN). The gene editing agriculture market is analyzed by product type (crop/seeds, livestock). The gene editing biotechnology market is analyzed by product type (kits and reagents, cell line engineering, animal models and services). The gene editing therapeutics market is analyzed by disease category (eye and rare diseases).

Specific geographic markets discussed include North America, Europe, Asia-Pacific, and the rest of the world (ROW).

Industry sectors analyzed include next-generation sequencing; long read sequencing; DNA synthesis; RNA therapies; and gene editing.

More than 320 companies in the read, write and edit industry are profiled in this report.

BCC Research also provides a summary of more than 180 of the main industry acquisitions and strategic alliances that took place from January 2018 through June 2019, including key alliance trends.

Report Includes:

28 data tables and 77 additional tables An overview of the global market for DNA read, write and edit technologies, applications, and industries Analyses of global market trends, with market data from 2017, 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 Discussion on sequencing technologies, market applications, industry structure, and important clinical sequencing initiatives Information pertaining to several significant large-scale research initiatives that are contributing to sequencing services, write synthesis and gene editing technologies market development A look at the innovations in pharmaceutical and biotechnology companies and research & development programs in stem cell-based therapies and gene therapies Coverage of significant patents and their allotments in each category, as well as major industry acquisitions and strategic alliances data Company profiles of over 320 major global players within the industry, including 3Billion Inc., 23Andme Inc., Bayer AG, Becton, Dickinson and Co. (BD), Bio-Rad Laboratories, Pacific Biosciences, Qiagen NV, Roche Holding AG and Thermo Fisher Scientific Inc.

Summary:

DNA (and RNA) read, write and editing includes the primary methods in which nucleic acids are analyzed (sequencing), created (synthesis) and modified (gene editing). It is becoming increasingly important in the life sciences industry to be expert in all aspects of nucleic acids in order to exploit significant opportunities within this industry. The end users for these technologies include any industry that works with biological systems, and even some that dont (e.g., DNA data storage).

The DNA read, write and edit industry is at the beginning stages of its growth story; penetration of the key markets is still at an early stage. For example, the cumulative number of human genomes sequenced reached REDACTED as of January 2019 (less than REDACTED of the global human population); we estimate that population-scale projects alone will increase that figure to more than REDACTED genomes sequenced during the next five years. The percentage of non-human species sequenced as of January 2019 was less than REDACTED of all species. These data indicate that there is significant future upside for sequencing across research, metagenomics, agriculture, synthetic biology and clinical applications,among others.

The situation is similar for DNA writing and editing technologies, with clinical therapeutic applications in particular providing an enormous total available future market that is yet to be significantly penetrated. Major successes in this industry include the adoption of next-generation sequencing (NGS) for noninvasive prenatal testing; enabling the roles of synthetic DNA oligonucleotides and genes in the rise of the synthetic biology industry; and rapid adoption of CRISPR gene editing by research institutions and biopharma industries.

There is increasing interplay among the three DNA technology platforms, giving rise to innovative corporate strategies. For example, Arbor Biotechnologies employs sequencing, gene synthesis and artificial intelligence to perform high-throughput discovery of biomolecules, including new CRISPR proteins.

More Info of Impact Covid19 @ https://www.trendsmarketresearch.com/report/covid-19-analysis/12558

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DNA Read, Write And Edit Market Sales Volume, Status, Growth, Opportunities and World the COVID-19 - 3rd Watch News

Researchers in the Jacobs School of Medicine and Biomedical Sciencescontinue to spearhead a number of projects related to the COVID-19 global health pandemic.

Peter L. Elkin, professor and chair of biomedical informatics, says several current studies are focused on data collection that can be used to better understand how to combat COVID-19.

Much of the work is being completed through the Clinical and Translational Science Awards (CTSA) consortium, of which UB is a member. It is one of more than 50 medical research institutions across the nation currently receiving CTSA program funding from the National Institutes of Health.

One such project is the launch of the National COVID Cohort Collaborative (N3C), a joint program between the National Center for Data to Health and the National Center for Advancing Translational Sciences.

Elkin says the projects aim is to build a warehouse of COVID-19 data for the entire CTSA consortium and for otherinterested contributing health care organizations.

This is intended to hold all patient data (inpatient and outpatient) on COVID-tested patients from all of the CTSA hubs, he says. It entails a cloud-based method for data collection on the COVID-19 pandemic.

We are working closely with N3C to see how this can be designed and implemented in astandardized and timely fashion.

The goal of developing a national-level COVID-19 database is to facilitate research and improve recruitment to clinical trials, he says.

N3C is looking to address the many difficult questions raised by the COVID-19 global emergency, such as:

UB is also a member of COMBATCOVID, a New York State initiative to save case report formson all hospital admissions for upper respiratory infections,including all patients tested for COVID-19 or patients who are suspected to have COVID-19.

The statewide consortium will collect and analyze the results from all the CTSA institutions in the state.

It is being run out of New York University, and I am participating from our site as our CTSA informatics core director, Elkin says. I am working on the design and data governance.

The data use agreements are being signed, and the database design and data definitions are being built, he adds. This larger row-level dataset will allow us to ask questions that would notbe possible at any one institution.

In UBs Department of Biomedical Informatics, Elkin and Frank D. LeHouillier, senior programmer and analyst, are involved in the project.

Clinical researchers in the Jacobs School who are involved include:

Researchers in the Department of Biomedical Informatics have also developed a validated microbiome platform that finds infected persons with COVID-19 whether symptomatic or not using deep sequencing of stool microbiome samples.

Elkin is working with postdoctoral associate Sapan Mandloi in using a National Center for Biotechnology Information (NCBI) Sequence Read Archive (SRA) database to collect and process metagenomics data for the organism classified as human gut metagenome.

The more than 300,000 samples are divided into 3,464 projects, according to Mandloi.

We are performing comparison of all samples raw sequences with SARS-Cov-2 genome using a NCBI SRA Taxonomy Analysis Tool (STAT), which utilizes precomputed k-mer dictionary databases and gene-specific profiling, Mandloi says. This allows us to perform geographic mapping of samples identified across the world.

Some 9,720 samples were identified as potential cases of colonization for COVID-19, which were mostly from the U.S., China, Australia and the U.K., he adds.

The ability to identify and track this trafficking of genetic material is vital as a public health topic, he says. As of now, this large pool of genetic data remains largely untapped for clinical surveillance using the combined strategy of gene-based profiling and k-mer-based classification on raw genomic data.

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Jacobs School researchers collecting COVID-19 data - UB Now: News and views for UB faculty and staff - University at Buffalo Reporter

Finding vaccines or drugs against COVID-19 is certainly one of the main current objectives of medical research centers worldwide. At Childrens National Medical Center, researchers are deploying technology tools from Amazon Web Services Inc. to combine hundreds of data sets to identify genes that might be targeted to treat many diseases, including COVID-19.

We know that there are a lot of drugs that target different genes,and we are particularly interested in, for example, can we repurpose some of these drugs to treatdifferent types of viruses, including COVID-19? said Wei Li (pictured), principal investigator at the Center for Genetic Medicine Research & Center for Cancer and Immunology Research at Childrens National Medical Center.

Li spoke with Stu Miniman, host of theCUBE, SiliconANGLE Medias livestreaming studio, during the AWS Public Sector Summit event. They discussed how the genome project can help combat COVID-19, as well as the role of AWS technology tools in scientific research. (* Disclosure below.)

The Childrens National Medical Center has been using computational biology and gene editing approaches to understand humangenome and disease, and it is particularly interested in a gene-editingtechnology called CRISPR screening, according to Li, who has a research background in computer science.

This is a fascinating technology because it tells you whether one of the 20,000human genes are connected with some certain disease phenotype in one single experiment, he said. We are tryingto, for example, perform machine-learning and data-mining approaches to find new clues of human diseasefrom the original mix and screening big data.

CRISPR screening and other similar screening methods have been widely used in recent years by several research laboratories to study virus infections, such as those related to HIV, Ebola, influenza and now coronavirus, according to Li. Then, the team at the Childrens National Medical Center had an idea: to connect all the sets of screening data related to these viruses to try to extract new information that cannot be identified in a single study.

Can we identify new patterns or new human genes that are commonly responsible for many different virus types? Or can we find some genes that work only from some certain type of viruses? he asked.

Researchers use AWS technology to process and analyze huge amount of data sets, in addition to creating an integrated database in the cloud, so that research results can be freely accessed around the world. It is estimated that AWS technology can reduce the time to process screening data from months to days, according to Li.

Two major benefits are expected from the outcome of this research project.

The first thing is that we hope to find some genes thatcan be potentially drug targets. So, if there are existing drugs that target the genes, then that would be perfect, because we dont need to do anything about this, he explained. And,in the end, we hope that these drugs can have the broad antiviral activity; that means that these drugs can be potentially used to treat COVID-19 and in the future if theres a new virus coming out.

Watch the complete video interview below, and be sure to check out more of SiliconANGLEs and theCUBEs coverage of the AWS Public Sector Summit event. (* Disclosure: TheCUBE is a paid media partner for the AWS Public Sector Summit Online event. Neither Amazon Web Services Inc., the sponsor for theCUBEs event coverage, nor other sponsors have editorial control over content on theCUBE or SiliconANGLE.)

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Children's National Medical Center and AWS partner for genome project targeting COVID-19 - SiliconANGLE