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LOS ANGELES, United States: QY Research has recently published a report, titled Global CRISPR And CRISPR-Associated Genes Market Size, Status and Forecast 2020-2026. The research report gives the potential headway openings that prevails in the global market. The report is amalgamated depending on research procured from primary and secondary information. The global CRISPR And CRISPR-Associated Genes market is relied upon to develop generously and succeed in volume and value during the predicted time period. Moreover, the report gives nitty gritty data on different manufacturers, region, and products which are important to totally understanding the market.

Key Companies/Manufacturers operating in the global CRISPR And CRISPR-Associated Genes market include: Thermo Fisher Scientific, Editas Medicine, Caribou Biosciences, CRISPR therapeutics, Intellia therapeutics, Inc., Cellectis, Horizon Discovery Plc, Sigma Aldrich, Precision Biosciences, Genscript, Sangamo Biosciences Inc., Lonza Group Limited, Integrated DNA Technologies, New England Biolabs, Origene Technologies CRISPR And CRISPR-Associated Genes

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Segmental Analysis

Both developed and emerging regions are deeply studied by the authors of the report. The regional analysis section of the report offers a comprehensive analysis of the global CRISPR And CRISPR-Associated Genes market on the basis of region. Each region is exhaustively researched about so that players can use the analysis to tap into unexplored markets and plan powerful strategies to gain a foothold in lucrative markets.

Global CRISPR And CRISPR-Associated Genes Market Segment By Type:

CRISPR is a type of gene-editing technology that lets scientists more rapidly and accurately cut and paste genes into DNA. Market Analysis and Insights: Global CRISPR And CRISPR-Associated Genes Market The global CRISPR And CRISPR-Associated Genes market size is projected to reach US$ XX million by 2026, from US$ XX million in 2020, at a CAGR of XX%% during 2021-2026. Global CRISPR And CRISPR-Associated Genes Scope and Market Size CRISPR And CRISPR-Associated Genes market is segmented 7, and 4. Players, stakeholders, and other participants in the global CRISPR And CRISPR-Associated Genes market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast 7 and 4 in terms of revenue and forecast for the period 2015-2026. The following players are covered in this report:Thermo Fisher ScientificEditas MedicineCaribou BiosciencesCRISPR therapeuticsIntellia therapeutics, Inc.CellectisHorizon Discovery PlcSigma AldrichPrecision BiosciencesGenscriptSangamo Biosciences Inc.Lonza Group LimitedIntegrated DNA TechnologiesNew England BiolabsOrigene Technologies CRISPR And CRISPR-Associated Genes

Global CRISPR And CRISPR-Associated Genes Market Segment By Application:

Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

Competitive Landscape

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the global CRISPR And CRISPR-Associated Genes market.

Key companies operating in the global CRISPR And CRISPR-Associated Genes market include Thermo Fisher Scientific, Editas Medicine, Caribou Biosciences, CRISPR therapeutics, Intellia therapeutics, Inc., Cellectis, Horizon Discovery Plc, Sigma Aldrich, Precision Biosciences, Genscript, Sangamo Biosciences Inc., Lonza Group Limited, Integrated DNA Technologies, New England Biolabs, Origene Technologies CRISPR And CRISPR-Associated Genes

Key questions answered in the report:

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TOC

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by CRISPR And CRISPR-Associated Genes Revenue1.4 Market 71.4.1 Global CRISPR And CRISPR-Associated Genes Market Size Growth Rate 7: 2020 VS 20261.4.2 Genome Editing1.4.3 Genetic Engineering1.4.4 GRNA Database/Gene Librar1.4.5 CRISPR Plasmid1.4.6 Human Stem Cells1.4.7 Genetically Modified Organisms/Crops1.4.8 Cell Line Engineering1.5 Market by Application1.5.1 Global CRISPR And CRISPR-Associated Genes Market Share 4: 2020 VS 20261.5.2 Biotechnology Companies1.5.3 Pharmaceutical Companies1.5.4 Academic Institutes1.5.5 Research and Development Institutes1.6 Study Objectives1.7 Years Considered 2 Global Growth Trends2.1 Global CRISPR And CRISPR-Associated Genes Market Perspective (2015-2026)2.2 Global CRISPR And CRISPR-Associated Genes Growth Trends by Regions2.2.1 CRISPR And CRISPR-Associated Genes Market Size by Regions: 2015 VS 2020 VS 20262.2.2 CRISPR And CRISPR-Associated Genes Historic Market Share by Regions (2015-2020)2.2.3 CRISPR And CRISPR-Associated Genes Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 CRISPR And CRISPR-Associated Genes Market Growth Strategy2.3.6 Primary Interviews with Key CRISPR And CRISPR-Associated Genes Players (Opinion Leaders) 3 Competition Landscape by Key Players3.1 Global Top CRISPR And CRISPR-Associated Genes Players by Market Size3.1.1 Global Top CRISPR And CRISPR-Associated Genes Players by Revenue (2015-2020)3.1.2 Global CRISPR And CRISPR-Associated Genes Revenue Market Share by Players (2015-2020)3.1.3 Global CRISPR And CRISPR-Associated Genes Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global CRISPR And CRISPR-Associated Genes Market Concentration Ratio3.2.1 Global CRISPR And CRISPR-Associated Genes Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by CRISPR And CRISPR-Associated Genes Revenue in 20193.3 CRISPR And CRISPR-Associated Genes Key Players Head office and Area Served3.4 Key Players CRISPR And CRISPR-Associated Genes Product Solution and Service3.5 Date of Enter into CRISPR And CRISPR-Associated Genes Market3.6 Mergers & Acquisitions, Expansion Plans 4 Market Size 7 (2015-2026)4.1 Global CRISPR And CRISPR-Associated Genes Historic Market Size 7 (2015-2020)4.2 Global CRISPR And CRISPR-Associated Genes Forecasted Market Size 7 (2021-2026) 5 Market Size 4 (2015-2026)5.1 Global CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020)5.2 Global CRISPR And CRISPR-Associated Genes Forecasted Market Size 4 (2021-2026) 6 North America6.1 North America CRISPR And CRISPR-Associated Genes Market Size (2015-2020)6.2 CRISPR And CRISPR-Associated Genes Key Players in North America (2019-2020)6.3 North America CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)6.4 North America CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 7 Europe7.1 Europe CRISPR And CRISPR-Associated Genes Market Size (2015-2020)7.2 CRISPR And CRISPR-Associated Genes Key Players in Europe (2019-2020)7.3 Europe CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)7.4 Europe CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 8 China8.1 China CRISPR And CRISPR-Associated Genes Market Size (2015-2020)8.2 CRISPR And CRISPR-Associated Genes Key Players in China (2019-2020)8.3 China CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)8.4 China CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 9 Japan9.1 Japan CRISPR And CRISPR-Associated Genes Market Size (2015-2020)9.2 CRISPR And CRISPR-Associated Genes Key Players in Japan (2019-2020)9.3 Japan CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)9.4 Japan CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 10 Southeast Asia10.1 Southeast Asia CRISPR And CRISPR-Associated Genes Market Size (2015-2020)10.2 CRISPR And CRISPR-Associated Genes Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia CRISPR And CRISPR-Associated Genes Market Size by Type (2015-2020)10.4 Southeast Asia CRISPR And CRISPR-Associated Genes Market Size by Application (2015-2020) 11 India11.1 India CRISPR And CRISPR-Associated Genes Market Size (2015-2020)11.2 CRISPR And CRISPR-Associated Genes Key Players in India (2019-2020)11.3 India CRISPR And CRISPR-Associated Genes Market Size by Type (2015-2020)11.4 India CRISPR And CRISPR-Associated Genes Market Size by Application (2015-2020) 12 Central & South America12.1 Central & South America CRISPR And CRISPR-Associated Genes Market Size (2015-2020)12.2 CRISPR And CRISPR-Associated Genes Key Players in Central & South America (2019-2020)12.3 Central & South America CRISPR And CRISPR-Associated Genes Market Size by Type (2015-2020)12.4 Central & South America CRISPR And CRISPR-Associated Genes Market Size by Application (2015-2020) 13 Key Players Profiles13.1 Thermo Fisher Scientific13.1.1 Thermo Fisher Scientific Company Details13.1.2 Thermo Fisher Scientific Business Overview13.1.3 Thermo Fisher Scientific CRISPR And CRISPR-Associated Genes Introduction13.1.4 Thermo Fisher Scientific Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020))13.1.5 Thermo Fisher Scientific Recent Development13.2 Editas Medicine13.2.1 Editas Medicine Company Details13.2.2 Editas Medicine Business Overview13.2.3 Editas Medicine CRISPR And CRISPR-Associated Genes Introduction13.2.4 Editas Medicine Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.2.5 Editas Medicine Recent Development13.3 Caribou Biosciences13.3.1 Caribou Biosciences Company Details13.3.2 Caribou Biosciences Business Overview13.3.3 Caribou Biosciences CRISPR And CRISPR-Associated Genes Introduction13.3.4 Caribou Biosciences Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.3.5 Caribou Biosciences Recent Development13.4 CRISPR therapeutics13.4.1 CRISPR therapeutics Company Details13.4.2 CRISPR therapeutics Business Overview13.4.3 CRISPR therapeutics CRISPR And CRISPR-Associated Genes Introduction13.4.4 CRISPR therapeutics Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.4.5 CRISPR therapeutics Recent Development13.5 Intellia therapeutics, Inc.13.5.1 Intellia therapeutics, Inc. Company Details13.5.2 Intellia therapeutics, Inc. Business Overview13.5.3 Intellia therapeutics, Inc. CRISPR And CRISPR-Associated Genes Introduction13.5.4 Intellia therapeutics, Inc. Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.5.5 Intellia therapeutics, Inc. Recent Development13.6 Cellectis13.6.1 Cellectis Company Details13.6.2 Cellectis Business Overview13.6.3 Cellectis CRISPR And CRISPR-Associated Genes Introduction13.6.4 Cellectis Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.6.5 Cellectis Recent Development13.7 Horizon Discovery Plc13.7.1 Horizon Discovery Plc Company Details13.7.2 Horizon Discovery Plc Business Overview13.7.3 Horizon Discovery Plc CRISPR And CRISPR-Associated Genes Introduction13.7.4 Horizon Discovery Plc Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.7.5 Horizon Discovery Plc Recent Development13.8 Sigma Aldrich13.8.1 Sigma Aldrich Company Details13.8.2 Sigma Aldrich Business Overview13.8.3 Sigma Aldrich CRISPR And CRISPR-Associated Genes Introduction13.8.4 Sigma Aldrich Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.8.5 Sigma Aldrich Recent Development13.9 Precision Biosciences13.9.1 Precision Biosciences Company Details13.9.2 Precision Biosciences Business Overview13.9.3 Precision Biosciences CRISPR And CRISPR-Associated Genes Introduction13.9.4 Precision Biosciences Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.9.5 Precision Biosciences Recent Development13.10 Genscript13.10.1 Genscript Company Details13.10.2 Genscript Business Overview13.10.3 Genscript CRISPR And CRISPR-Associated Genes Introduction13.10.4 Genscript Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.10.5 Genscript Recent Development13.11 Sangamo Biosciences Inc.10.11.1 Sangamo Biosciences Inc. Company Details10.11.2 Sangamo Biosciences Inc. Business Overview10.11.3 Sangamo Biosciences Inc. CRISPR And CRISPR-Associated Genes Introduction10.11.4 Sangamo Biosciences Inc. Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.11.5 Sangamo Biosciences Inc. Recent Development13.12 Lonza Group Limited10.12.1 Lonza Group Limited Company Details10.12.2 Lonza Group Limited Business Overview10.12.3 Lonza Group Limited CRISPR And CRISPR-Associated Genes Introduction10.12.4 Lonza Group Limited Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.12.5 Lonza Group Limited Recent Development13.13 Integrated DNA Technologies10.13.1 Integrated DNA Technologies Company Details10.13.2 Integrated DNA Technologies Business Overview10.13.3 Integrated DNA Technologies CRISPR And CRISPR-Associated Genes Introduction10.13.4 Integrated DNA Technologies Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.13.5 Integrated DNA Technologies Recent Development13.14 New England Biolabs10.14.1 New England Biolabs Company Details10.14.2 New England Biolabs Business Overview10.14.3 New England Biolabs CRISPR And CRISPR-Associated Genes Introduction10.14.4 New England Biolabs Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.14.5 New England Biolabs Recent Development13.15 Origene Technologies10.15.1 Origene Technologies Company Details10.15.2 Origene Technologies Business Overview10.15.3 Origene Technologies CRISPR And CRISPR-Associated Genes Introduction10.15.4 Origene Technologies Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.15.5 Origene Technologies Recent Development 14 Analysts Viewpoints/Conclusions 15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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Trending: CRISPR And CRISPR-Associated Genes Market Research Key Players, Industry Overview and forecasts to | Thermo Fisher Scientific, Editas...

In January, vaccine researchers lined up on the starting blocks, waiting to hear a pistol. That shot came on January 10, when scientists in China announced the complete genetic makeup of the novel coronavirus. With that information in hand, the headlong race toward a vaccine began.

As the virus, now known as SARS-CoV-2, began to spread like wildfire around the globe, researchers sprinted to catch up with treatments and vaccines. Now, six months later, there is still no cure and no preventative for the disease caused by the virus, COVID-19, though there are glimmers of hope. Studies show that two drugs can help treat the sick: The antiviral remdesivir shortens recovery times (SN: 4/29/20) and a steroid called dexamethasone reduces deaths among people hospitalized with COVID-19 who need help breathing (SN: 6/16/20).

But the finish line in this race remains a safe and effective vaccine. With nearly 180 vaccine candidates now being tested in lab dishes, animals and even already in humans, that end may be in sight. Some experts predict that a vaccine may be available for emergency use for the general public by the end of the year even before it receives expedited U.S. Food and Drug Administration approval.

Velocity might come at the expense of safety and efficacy, some experts worry. And that could stymie efforts to convince enough people to get the vaccine in order to build the herd immunity needed to end the pandemic.

Were calling for transparency of data, says Esther Krofah, executive director of FasterCures, a Washington, D.C.-based nonprofit. We want things to accelerate meaningfully in a way that does not compromise safety or the science, but we need to see the data, she says.

Traditionally, vaccines are made from weakened or killed viruses, or virus fragments. But producing large amounts of vaccine that way can take years, because such vaccines must be made in cells (SN: 7/7/20), which often arent easy to grow in large quantities.

Getting an early good look at the coronaviruss genetic makeup created a shortcut. It let scientists quickly harness the viruss genetic information to make copies of a crucial piece of SARS-CoV-2 that can be used as the basis for vaccines.

That piece is known as the spike protein. It studs the viruss surface, forming its halo and allowing the virus to latch onto and enter human cells. Because the spike protein is on the outside of the virus, its also an easy target for antibodies to recognize.

Researchers have copied the SARS-CoV-2 version of instructions for making the spike protein into RNA or DNA, or synthesized the protein itself, in order to create vaccines of various types (see sidebar). Once the vaccine is delivered into the body, the immune system makes antibodies that recognize the virus and block it from getting into cells, either preventing infection or helping people avoid serious illness.

Using this approach, drugmakers have set speed records in devising vaccines and beginning clinical trials. FasterCures, which is part of the Milken Institute think tank, is tracking 179 vaccine candidates, most of which are still being tested in lab dishes and animals. But nearly 20 have already begun testing in people.

Some front-runners have emerged, leading the pack in a neck-and-neck race. Some have been propelled by an effort by the U.S. federal government, called Operation Warp Speed, which has picked a handful of vaccine candidates to fast-track.

First out of the starting gate was one developed by Moderna, a Cambridge, Mass.based biotech company. It inoculated the first volunteer with its candidate vaccine on March 16, just 63 days after the viruss genetic makeup was revealed. The company has since reported preliminary safety data, and some evidence that its vaccine stimulates the immune system to produce antibodies against the coronavirus (SN: 5/18/20).

That company and several others now have vaccines entering Phase III clinical trials. Moderna and the National Institute of Allergy and Infectious Diseases, in Bethesda, Md., will begin inoculating 30,000 volunteers with either the vaccine or a placebo in July to test the vaccines efficacy in large numbers of people.

Modernas vaccine requires two doses; a prime and a boost. That means it will take 28 days to get any individual person vaccinated, NIAID director Anthony Fauci said June 26 during a Milken Institute webinar. It will take weeks and months to give the full set of shots to all those people. Then it will take time to determine whether more people in the placebo group get COVID-19 than those in the vaccine group a sign that the vaccine works. Those results could come in late fall or early winter.

NIAID launched a clinical trials network July 8 to recruit volunteers at sites across the United States for phase III testing of vaccines and antibodies to prevent COVID-19. Modernas vaccine will be the first in line for testing.

Some researchers propose accelerating clinical trials even further by trying controversial challenge trials, in which vaccinated volunteers are intentionally exposed to the coronavirus (SN: 5/27/20). None of those studies have gotten the green light yet.

Three other global drug and vaccine companies have announced plans to launch similarly sized trials this summer: Johnson & Johnson; AstraZeneca, working with the University of Oxford; and Pfizer Inc., which has teamed up with the German company BioNTech. Like Moderna, all are part of Operation Warp Speed, or will be joining it.

Usually, Phase III trials are about determining efficacy. But the rush to get through earlier stages designed to make sure a drug doesnt cause harm means that scientists also will be keeping a keen eye on safety, Fauci said. Researchers will be watching, in particular, for any suggestion that antibodies generated by the vaccine might enhance infection.

That can happen when antibodies stimulated by the vaccine dont fully neutralize the virus and can aid it getting into cells and replicating, or because the vaccine alters immune cell responses in unhelpful ways. Vaccines against MERS and SARS coronaviruses made infections with the real virus worse in some animal studies.

Such enhanced infections are a worry for any unproven vaccine candidate, but some experimental vaccines in the works may be more concerning than others, says Peter Pitts, president of the Center for Medicine in the Public Interest, a nonprofit research and education organization headquartered in New York City.

For instance, China-based CanSino Biologics Inc. has developed a hybrid virus vaccine: Its made by putting the coronavirus spike protein into a common cold virus called adenovirus 5. That virus can infect humans but has been altered so that it can no longer replicate.

In a small study, reported June 13 in the Lancet, CanSinos vaccine triggered antibody production against the spike protein. But many volunteers already had preexisting antibodies to the adenovirus, raising concerns that that could weaken their response to the vaccine. A weakened response might make an infection worse when people encounter the real coronavirus, Pitts says.

Thats of particular concern because CanSino said in a June 29 statement to the Hong Kong stock exchange that its vaccine was approved by the Chinese government for temporary use by the Chinese military. Thats essentially turning soldiers into guinea pigs, Pitts says.

The type of antibodies stimulated by the vaccine will be important in determining whether the vaccine protects against disease or makes things worse, Yale University immunologists Akiko Iwasaki and Yexin Yang, warned April 21 in Nature Reviews Immunology. Some types of antibodies have been associated with more severe COVID-19.

And it will be important to monitor the ratio of neutralizing antibodies and non-neutralizing antibodies, as well as activity of other immune cells triggered by the vaccines, an international working group of scientists recommended in a conference report in the June 26 Vaccine.

Public health officials will also be tracking side effects closely. As big as the vaccine trials may be, we cant be sure that there arent rare side effects, Anne Schuchat, principal deputy director of the Centers for Disease Control and Protection, said June 29 during a question-and-answer session with the Journal of the American Medical Association. Thats why even when we get enough to vaccinate large numbers, were going to need to be following it.

In 1976 for instance, it turned out that Guillain-Barr syndrome, a rare neurological condition in which the immune system attacks parts of the nervous system, was a rare side effect of the swine flu influenza vaccine. That didnt become obvious until the vaccine had already been rolled out to 45 million people in the United States.

Scientists and journalists share a core belief in questioning, observing and verifying to reach the truth. Science News reports on crucial research and discovery across science disciplines. We need your financial support to make it happen every contribution makes a difference.

Early on, it was unclear whether scientists could devise a vaccine against the coronavirus at all. Its now a question of when rather than if well have a vaccine.

But some researchers have expressed concern that rushing clinical trials might lead federal regulators to approve a vaccine based on its ability to trigger antibody production alone. Its still unclear how well antibodies protect against reinfection with the coronavirus and how long any such immunity may last (SN: 4/28/20). The measure of whether the vaccine works should be its ability to protect against illness, not antibody production, Fauci said.

I really want to make sure that we dont have a vaccine thats distributed among the American people unless we know its safe and we know it is effective, he said. Not that we think it might be effective, but that we know its effective.

So far though, companies are measuring success by the antibody. For instance, INOVIO, a biotechnology company based in Plymouth Meeting, Pa., announced June 30 that 94 percent of participants in a small safety trial made antibodies against the coronavirus. The data, delivered via news release like that from numerous other companies rushing to show progress, had not been peer-reviewed and other details about the companys DNA-based vaccine were sparse.

Despite still having much to prove, companies are gearing up manufacturing without knowing if their product will ever reach the market. By the end of the year, companies promise they can have hundreds of millions of doses. We keep saying, Are you sure? And they keep saying yes, Fauci said. Thats pretty impressive if they can do it.

For instance, if everything goes right, a vaccine in testing now from Pfizer might be available as soon as October, Pfizer chairman and chief executive Albert Bourla said during the Milken Institute session. If we are lucky, and the product works and we do not have significant bumps on our way to manufacturing, he said, the company expects to be able to make 1 billion doses by early next year.

Pfizer released preliminary data on the safety of one of four vaccine candidates it is evaluating July 1 at medRxiv.org. In the small study of 45 people, no severe side effects were noted. Vaccination produced neutralizing antibodies at levels 1.8 to 2.8 times levels found in blood plasma from people who had recovered from COVID-19, researchers reported.

Novavax Inc., a Gaithersburg, Md.-based biotechnology company, announced July 7 that it was being award $1.6 billion from Operation Warp Speed to conduct phase III trials and to deliver 100 million doses of its vaccine as early as the end of the year.

If manufacturers can deliver a vaccine as promised, there could be another big hurdle: Theres no guarantee people will line up for shots. About a quarter of Americans said in recent polls that they would definitely or probably not get a coronavirus vaccine if one were available. Thats a pending public health crisis, Pitts says.

Krofah agrees. We need to think about the post-pandemic world in the midst of all of this, she says. We need to start building that public trust now. Tackling issues of vaccine hesitancy shouldnt be left until a vaccine is available, she says.

Whether with vaccines or treatments, we need to expedite, but not rush, Pitts says. Theres a perception that therapeutics or vaccines will be approved willy-nilly because of politics, and thats a dangerous misperception. The FDA laid out guidelines, including an accelerated approval process, on June 30 that should ensure any approved vaccines work, he says.

There is good news for those who are eagerly awaiting vaccines, Krofah and Pitts say: There wont be just one winner in the race. Instead, there may be multiple options to choose from. Thats not a luxury; it may be a necessity. Multiple vaccines may be needed to protect different segments of the population, Krofah says. For instance, elderly people may need a vaccine that prods the immune system harder to make antibodies, and children may need different vaccines than adults do.

Whats more, long-term investments in development will be needed so that vaccines can be altered if the virus mutates. We need to stay the front and not declare victory once a vaccine has been approved for emergency use, she says.

For now, vaccine makers are moving both as quickly and as carefully as possible, Bourla said. I am aware that right now that billions of people, millions of businesses, hundreds of governments are investing their hope for a solution in a handful of pharma companies.

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A COVID-19 vaccine may come soon. Will the blistering pace backfire? - Science News

Illumina announced today the launch of its TruSight Software Suite, a solution that aids in creating efficient workflows to help increase adoption of whole-genome sequencing and comes with the promise of significantly reducing the time from sample to answer from daysor even weeksto hours.

Developed in collaboration with researchers and clinicians at the Mayo Clinic, and other partners, Illumina says TruSight provides a turn-key solution to tackle the most critical, and challenging piece of incorporating whole-genome sequencing for the identification of rare genetic diseasesthe interpretation of millions of variants to rapidly identify the handful of relevant variants that are contributing to an individuals disease.

The new software suite can pull together the power of a range of offering from Illumina including the NovaSeq 6000, its DRAGEN Bio-IT Platform, and Illumina DNA PCR-Free Prep, which when taken together provides a complete whole-genome sequencing analysis workflow for curation and reporting of rare variants.

This combination of products will set the standard for scalable and swift interpretation of genomic information, enabling whole-genome sequencing to become the standard of care in rare diseases, said Ryan Taft, vice president of scientific research at Illumina in a press release. By enabling users to quickly sift through millions of variants to find an answer, we will make it easier for rare disease patients to benefit from valuable genomic insights.

The launch of the new workflow software comes as rare disease diagnosis and treatment is rapidly establishing itself as the second prominent area of precision medicine alongside cancer care. It is thought there could be as many as 7,000 rare diseases and, when considered as a group, these are estimated to affect between 25 million and 30 million people in the U.S. alone and more than 200 million globally.

While some rare genetic diseases require almost immediate attention after birth in order to provide any chance at effective treatment, as evidenced by the ongoing work of Dr. Stephen Kingsmore and colleagues at Rady Childrens Institute of Genomic Medicine, many more rare conditions are not life threatening. In these cases, the patients and their families often embark on a diagnostics odyssey one marked by referrals from one medical specialist to another and can often take as long as seven years before a diagnosis.

Between needing regular care and the battery of testing done for rare disease patients, it is estimated that in the U.S. alone the cost of pediatric genetic diseases total more than $57 billion every year. Broadening availability to whole-genome testing for patients with a suspect rare genetic disorder can help shorten the time to diagnosis and potentially save billions of dollars of healthcare costs.

The future of pediatric medicine will include whole-genome sequencings for suspectedgeneticdisorders, said William Morice, M.D., Ph.D., president, Mayo Clinic Laboratories, and department chair, laboratory medicine and pathology at Mayo Clinic. Enabling laboratories and physicians with access toefficient, clinical-gradewhole-genome sequencingsolutionsis essential.

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Illumina Intros Genomic Analysis Workflow Software to Speed Diagnosis of Genetic Conditions - Clinical OMICs News

For 25 years, researchers have explored an idea that, by regulating certain genes, they could treat one of the world's most debilitating neurological diseases. That work has led to encouraging data, with the latest coming Wednesday from two studies published in the New England Journal of Medicine.

"It's a really exciting time for the field," said Orla Hardiman, clinical professor of neurology at Trinity College in Dublin and co-author to a NEJM editorial published alongside the studies.

Previously, scientists discovered the risk of developing amyotrophic lateral sclerosis,also known as ALS or Lou Gehrig's disease, is higher if a select group of genes mutate. The newly published studies each tested an experimental drug meant to silence one such gene, called SOD1, that encodes an eponymous protein. While both studies were designed to evaluate safety, researchers also looked at protein levels to see if the drugs were working as intended.

One of these drugs uses a virus to deliver a small strip of genetic material into patients' spinal areas. In theory, the material would block the gene from making its protein, but results from two patients showed that neither had a substantial change in protein levels in their cerebrospinal fluid.

However, after one of the patients died, an autopsy showed SOD1 protein levels in his spinal cord tissue were lower than in untreated patients with the same form of ALS. The study investigators concluded that additional trials with a larger number of patients are necessary to better understand the drug's effects.

The other study had more clearly positive results. It tested four doses of Biogen's tofersen against placebo, and found lower SOD1 protein levels in the cerebrospinal fluid of patients who received the drug. Compared to those in the placebo group, protein concentrations were about 20 to 25 percentage points lower for patients given the two middle tofersen doses and 33 percentage points lower for patients on the highest, 100 mg dose.

Biogen, which announced summary data from the trial last year, has since moved the high dose into a larger, efficacy-focused trial that aims to recruit around 100 patients. Enrollment has been "reasonable," albeit with slight delays due to the coronavirus pandemic, according to Toby Ferguson, head of the company's neuromuscular development unit.

Though tofersen will likely need positive late-stage results to support an approval, the currently available data offer a confidence boost for Biogen. Like other ALS drug hunters, the biotech has hit setbacks the most damaging of which came in 2013 when its small molecule medicine dexpramipexole failed a Phase 3 study.

"It's not fully shown to work yet, but at least the biology seems to be going in the right way," Ferguson said of tofersen. "It fundamentally says to me that if we pick the right targets, ALS can be a treatable disease. And we need to push forward both with genetic targets and appropriate targets for the broader population."

The tofersen study may also fuel optimism in the broader ALS research community. While two drugs are approved for ALS, there remains an urgent demand for more treatments. Most patients live just three to five years after they're diagnosed, according to the Centers for Disease Control and Prevention.

Following decades of research, genetic medicine has, in recent years, proven itself to be a valuable weapon against hard-to-treat neurological conditions. In 2016, for example, Biogen's drug Spinraza became the first ever approved treatment for spinal muscular atrophy, a rare and often life-threatening condition that impairs muscle growth. Spinraza, like tofersen, is a type of gene-silencing medicine called antisense oligonucleotides, or ASOs.

Sarepta Therapeutics also has two ASO products approved for a different muscular disorder, and research on other gene-based treatments is advancing for difficult neurological diseases like Huntington's and Rett syndrome.

In ALS, several companies are working on genetic medicines. Novartis and Voyager Therapeutics each have plans for a SOD1-targeting ALS gene therapy, while MeiraGTx and the partners Pfizer and Sangamo Therapeutics are developing gene therapies not specific to SOD1.

With tofersen, though, Biogen holds a leading and potentially tone-setting position.

As the drug progresses through late-stage testing, Hardiman said it would be "fantastic" if the drug demonstrates not just reductions in SOD1 protein levels, but also the ability to slow or stabilize the disease. Biogen's smaller study hinted that tofersen's effect on SOD1 protein levels might translate to slower functional declines, but the data aren't proof it actually does.

"If we can show that gene-silencing in SOD1 is effective, it opens the way for other gene-silencing approaches in other genetic forms of ALS," she said, pointing to several other mutations associated with ALS.

"We are in a new era now where we have a much better understanding of genomic regulation, and we're getting to a place where it's really possible to modulate these pathways in a way that's genuinely therapeutic," Hardiman added.

ALS drug research also extends beyond genes, since estimates hold that only 5% to 10% of cases are inherited and, within that fraction, SOD1 mutations account for 15% to 20% of cases.

Currently, the Sean M. Healey & AMG Center for ALS Research is running a first-of-its-kind platform trial to test five experimental therapies, including ones from Biohaven Pharmaceutical and Cambridge, Massachusetts-based Ra Pharmaceuticals, now owned by Belgium's UCB.

Privately held Amylyx Pharmaceuticals, meanwhile, is working separately with the Healey Center. The company said in December its experimental treatment slowed ALS progression in a mid-stage study, although no actual data was released.

Alexion Pharmaceuticals, a large rare disease drugmaker, also recently began exploring whether one of its approved therapies could work in ALS too.

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Experts propose decentralized system to monitor wildlife markets, other hotspots

A juvenile saddleback tamarin is measured as part of an annual health check of a population of three primate species in southeastern Peru. In a perspective article published July 9 in Science, a team of wildlife biologists, infectious disease experts, and others propose a decentralized, global wildlife biosurveillance system to identify before the next pandemic emerges animal viruses that have the potential to cause human disease.

The virus that causes COVID-19 probably originated in wild bats that live in caves around Wuhan, China, and may have been passed to a second animal species before infecting people, according to the World Health Organization. Many of the most devastating epidemics of recent decades including Ebola, avian influenza and HIV/AIDS were triggered by animal viruses that spilled over into people. Despite the ever-present danger of a new virus emerging and sparking a worldwide pandemic, there is no global system to screen for viruses in wild animals that eventually may jump to humans.

In a perspective article published July 9 in Science, a diverse group of infectious disease experts, ecologists, wildlife biologists and other experts argue that a decentralized global system of wildlife surveillance could and must be established to identify viruses in wild animals that have the potential to infect and sicken people before another pandemic begins.

Its impossible to know how often animal viruses spill over into the human population, but coronaviruses alone have caused outbreaks in people three times in the last 20 years, said co-author Jennifer A. Philips, MD, PhD, referring to the SARS, MERS and COVID-19 epidemics. Philips is an associate professor of medicine and co-director of theDivision of Infectious Diseasesat Washington University School of Medicine in St. Louis. Even a decade ago it would have been difficult to conduct worldwide surveillance at the human-wildlife interface. But because of technological advances, it is now feasible and affordable, and it has never been more obvious how necessary it is.

Every animal has its own set of viruses, with some overlap across species. Often, an animal species and its viruses have lived together for so long that theyve adapted to one another, and the viruses cause either no symptoms or only mild to moderate disease. But when different animal species that dont normally have much contact are brought together, viruses have the opportunity to jump from one species to another. Most viruses dont have the genetic tools to infect another species. But viruses with such tools can be lethal to a newly infected species with no natural immunity.

Human activity is making such spillover events more and more likely. As the population of the world continues to grow, the demand for natural resources skyrockets. People push into wild areas to make space for new homes and businesses, and to access resources to fuel their economies and lifestyles. Wild animals are caught and sold for consumption, or as exotic pets at wildlife markets, where diverse species are jumbled together under crowded and unsanitary conditions. Wild-animal parts are shipped around the world as trinkets or ingredients for traditional or alternative medicines.

And yet there is no international system set up to screen for disease-causing viruses associated with the movement of wildlife or wildlife products.

In the lead up to this article, I spoke with friends and colleagues around the world who do wildlife research in Madagascar, Indonesia, Peru, Ecuador and asked them, Where do you take your samples for screening? said co-author Gideon Erkenswick, PhD, a postdoctoral research associate in Philips lab. Erkenswick is also the director of Field Projects International, a nonprofit organization dedicated to the study and conservation of tropical ecosystems. In almost every situation, the answer was Nowhere. Locally, there is nobody with dedicated time and resources to do this work. To find new disease-causing viruses, we have to find willing foreign collaborators, then get samples out of the country, which is difficult and expensive.

Philips, Erkenswick, and colleagues in the Wildlife Disease Surveillance Focus Group that authored the Science paper, suggest the establishment of a global surveillance network to screen wild animals and their products at hotspots such as wildlife markets. The idea would be to have local teams of researchers and technicians extract viral genomes from animal samples, rapidly sequence them on site and upload the sequences to a central database in the cloud. The cost and size of the necessary scientific equipment has dropped in recent years, making such screening affordable even in resource-limited settings where most such hotspots are located.

Theres now a genetic sequencer available that is literally the size of a USB stick, Erkenswick said. You could bring that and a few other supplies into a rainforest and analyze a sample for sequences associated with disease-causing viruses on site in a matter of hours. I mean, if you do chance upon something like the virus that causes COVID-19, do you really want to be collecting it, storing it, transporting it, risking further exposure, sample degradation, and adding months or years of delay, before you figure out what youve got? There are people with the expertise and skills to do this kind of work safely pretty much everywhere in the world, they just havent been given the tools.

Once viral sequences are uploaded, researchers around the world could help analyze them to identify animal viruses that may be a threat to people and to develop a better understanding of the universe of viruses that thrive in different environments. By comparing genomic sequence data, researchers can identify what family an unknown virus belongs to and how closely it is related to any disease-causing viruses. They can also identify whether a virus carries genes associated with the ability to cause disease in people.

By knowing the diversity out there, and tracking its evolution, we can ensure that we stay ahead of whats in wildlife populations and at the wildlife-human interface, Philips said. In the past, before modern transportation, spillover events would have been local and spread slowly, giving people elsewhere time to respond. But now the world is so small that an event in one place puts the whole world at risk. This is not someone elses problem. Its everyones problem.

Watsa M and the Wildlife Disease Surveillance Focus Group. Rigorous wildlife disease surveillance: A decentralized model could address global health risks associated with wildlife exploitation. Science. July 10, 2020. DOI: 10.1126/science.abc0017

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Artificial intelligence (AI) is a growing market engulfing all the sectors, is showing no signs of slowing down, especially in healthcare.

FREMONT, CA: The usage of AI in healthcare is predicted to reach $6.8 billion by 2021. Companies and countries are seeing the value of focusing on AI research. Healthcare databases are usually complicated but are full of useful information that can be utilized for drug discovery and precision medicine. Adopting AI in healthcare can help in improving the organization of data as well as fast-paced research breakthroughs.

It is a well-known fact that personalized medicine creates better patient outcomes when treating cancer. Additionally, to detect cancer, AI technology shows promising possibilities when it comes to differentiating genetic mutations to allow for precision medicine. By being able to evaluate and pinpoint genetic mutations, oncologists can provide better treatment for their patients. Personalized, or precision medicine demands constant analysis of genetic mutations to discover new treatments. Leveraging AI tools like Machine Learning and big data can help streamline this data collection and even improve with drug discovery.

AI technology helps differentiate healthy versus cancer cells, determine genetic mutations, and help researchers develop cancer treatment drugs. The pharmaceutical company is teaming up with technology giants to apply AI tools to drug discovery efforts. Using big data and Machine Learning technology, researchers can now analyze vast amounts of data seeking new ways to apply gene therapy. By having these tools, the time it takes to make these discoveries could be drastically shortened, leading to faster drug discovery and development.

It is transparent that the adoption of AI in healthcare can provide many benefits to researchers, healthcare organizations, and patients. AI tools can aid in making breakthroughs in cancer diagnostics, as well as treatment in real-time. But precaution must be ensured so that the data is shared safely and accurately. There must always be a delicate balance of human touch and machine to help prescribe with care.

See Also:Top Drug Discovery and Development Solution Companies

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How Can AI Help in Oncology Assisting Diagnostics and Drug Discovery? - Healthcare Tech Outlook

LOS ANGELES, July 7, 2020 /PRNewswire/ -- The Prostate Cancer Foundation (PCF) has collaborated with aconsortium of 41 leading patient advocacy organizations, professional societies and industry partners to publish a white paper detailing recommendations for the use of testing terminology in precision medicine for patient education throughout the cancer community. Use of consistent language will significantly improve patient awareness and understanding of potentially life-saving testing options available for both new cancer diagnoses and progression or recurrence of disease. In prostate cancer, testing is a crucial tool that may reveal additional treatment options and/or information for a man's family about their own cancer risk.

Research shows that despite widespread acceptance of the importance of testing, actual testing rates lag far behind best-practice recommendations for both biomarker testing for somatic (acquired) mutations and other biomarkers, and for germline genetic testing for identifying germline (inherited) mutations (also known as variants). Analysis by The Consistent Testing Terminology Working Group(Working Group) indicates that language disparity is a primary obstacle to patient communication with providers about testing for their specific cancer type. Further, development of consistent language can increase patient understanding and communication, facilitate shared decision making, support value-based care and assure concordance in policy development.

"Both types of testing biomarker testing and genetic testing for inherited cancer risk are important in the care of prostate cancer patients," said Dr. Andrea Miyahira, Director of Global Research and Scientific Communications at PCF. "One example is the very recent approval of medications for men with advanced prostate cancer and certain mutations in their tumor or inherited mutations that would be revealed through testing. Therefore, clear terminology and understanding between patients and providers is all the more vital. PCF supports this valuable collaboration across cancer types."

The Working Group is a consortium of 20 cancer patient advocacy groups representing solid tumor and hematologic malignancies, three professional societies, and 18 pharmaceutical and diagnostic companies and testing laboratories. Over the course of many years, multiple activities, led by numerous individual patient advocacy organizations and professional societies have developed the groundwork for this effort. The Working Group has launched a multi-faceted dissemination and communications effort to ensure that its recommendations and supporting materials are widely available among all key stakeholders within the cancer ecosystem, including providers, patient advocacy organizations, guidelines agencies, payers, and policymakers.

In developing its recommendations, the Working Group, first convened in 2019 by LUNGevity Foundation, identified 33 terms related to biomarker, genetic and genomic testing that were being used in patient education and clinical care within the different cancer communities. In many cases, multiple terms were used to describe the same test. Various testing modalities, the source of testing samples, and the multiplicity of gene mutations currently identifiable by testing, were contributing factors in this often-confusing overlap.

In the final analysis, three umbrella descriptor terms emerged as recommendations from the Working Group's milestone exploration: "Biomarker testing"was selected as the preferred term for tests that identify characteristics, targetable findings or other test results originating from malignant tissue and blood; "genetic testing for an inherited mutation" and "genetic testing for inherited cancer risk" were selected as consensus terms for tests used to identify germline (inherited) mutations.

"Far too many patients across all cancer types are still missing out on essential tests for biomarkers and inherited mutations indicating cancer risk," said Michelle Shiller, DO, AP/CP, MGP, Co-Medical Director of Genetics at Baylor Sammons Cancer Center and Staff Pathologist at Baylor University Medical Center. "With rates of biomarker testing and genetic testing for an inherited mutation at sub-optimal levels for numerous patient populations, patients are not benefiting from biomarker-directed care or not learning about their inherited cancer risk. Confusion around testing terms is a driving factor in this undertesting and ultimately has a detrimental impact on patient care."

"When someone is diagnosed with cancer, they're swept into a whirlwind of bewildering words and complex, pressing decisions. Our Working Group's goal is to help calm that storm of confusion with clear and consistent language that facilitates communication and medical decision-making. A unified voice and message from providers, industry and the patient advocacy community about testing is absolutely vital to optimal cancer care," saidNikki Martin, Director of Precision Medicine Initiatives at LUNGevity Foundation.

An abstract on the Working Group's recommendations was published in May 2020 as part of the American Society of Clinical Oncology (ASCO) Annual Meeting Virtual Library. The White Paper can be viewed in its entirety athttp://www.commoncancertestingterms.org/.

About LUNGevity Foundation LUNGevity Foundation is the nation's leading lung cancer organization focused on improving outcomes for people with lung cancer through research, education, policy initiatives, and support and engagement for patients, survivors, and caregivers. LUNGevity seeks to make an immediate impact on quality of life and survivorship for everyone touched by the diseasewhile promoting health equity by addressing disparities throughout the care continuum. LUNGevity works tirelessly to advance research into early detection and more effective treatments, provide information and educational tools to empower patients and their caregivers, promote impactful public policy initiatives, and amplify the patient voice through research and engagement. The organization provides an active community for patients and survivorsand those who help them live better and longer lives.

Comprehensive resources include a medically vetted and patient-centric website, a toll-free HELPLine for support, the International Lung Cancer Survivorship Conference, and an easy-to-use Clinical Trial Finder, among other tools. All of these programs are to achieve our visiona world where no one dies of lung cancer. LUNGevity Foundation is proud to be a four-star Charity Navigator organization. Please visit http://www.LUNGevity.org to learn more.

About the Prostate Cancer Foundation The Prostate Cancer Foundation (PCF) is the world's leading philanthropic organization dedicated to funding life-saving prostate cancer research. Founded in 1993 by Mike Milken, PCF has raised more than $830 million in support of cutting-edge research by more than 2,200 research projects at 220 leading cancer centers in 22 countries around the world. Thanks in part to PCF's commitment to ending death and suffering from prostate cancer, the death rate is down more than 50% and countless more men are alive today as a result. PCF research now impacts more than 73 forms of human cancer by focusing onimmunotherapy, the microbiome, and food as medicine. For more information, visit PCF.org.

Media Contact: Donald Wilson Prostate Cancer Foundation (310) 428-4730 [emailprotected]

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Genomic sequencing explained

Genomic sequencing creates a genetic fingerprint of organisms and maps the order of how chemical building blocks of a genome are organised.

The researchers looked at how the virus genetic sequence was organised by detecting and translating minute differences in each new infection. A genetic family tree was created showing which COVID-19 positive cases were connected and to track clusters.

The more fingerprints we took, and the critical information collected from the contact tracers, the easier it became to identify if someone contracted COVID-19 from a known cluster or case, said Dr Rockett.

Very early on we were able to discover cases which werent linked to a known cluster or case. This informed state and federal governments that community transmission was happening, and led to the border closures, revision of testing policies and other measures that stopped further spread of the virus.

Dr Rockett and her team managed to produce these genomic data so quickly because they leveraged years of experience in using genome sequencing to track down food-borne pathogens such as salmonella, during food poisoning outbreaks, and transmission of tuberculosis.

The study is a behind the scenes look at the complex and coordinated effort by virologists, bioinformaticians and mathematical modellers alongside clinicians and public health professionals.

Dr Rocketts lab is the dedicated facility hosted by NSW Health Pathology providing genomic sequencing data to NSW Health professionals working at the frontline of managing the pandemic.

Genome sequencing is the key to unlocking the puzzle of local transmission, and its critical that we continue to invest in this research to advance our ability to contain the virus in the long-term not just to trace locally acquired cases, but also to identify new cases once border restrictions are lifted and travel resumes, says Dr Rockett.

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Genetic fingerprints of first COVID19 cases help manage pandemic - News - The University of Sydney

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

The roulette wheel that decides who lives and dies from the coronavirus is weighted by the type of blood coursing through the veins of victims, gifting some with innate resistance and dooming others to misery and torment.

Infectious disease specialists say the worldwide pandemic is especially cruel to people with type A blood, which apparently lacks certain compounds that help fight off the disease.

A study published June 17 in the New England Journal of Medicine found that people with type A blood have a higher risk of contracting the disease and suffering complications. The analysis, conducted by an international team of scientists, also showed that people with type O blood were at least partially protected from the virus.

It was one of several recent reports on the phenomenon, which epidemiologists say is not unique to COVID-19.

People with Type A blood... are more likely to have severe disease and death than people with other types, said John Swartzberg, an infectious-disease specialist at UC Berkeley. It doesn't surprise me because we know that blood types are associated with other infectious diseases.

Blood type is determined by a gene that tells the body what blood cell proteins to make. The different types, A, B, AB or O, have different antigens, which determine their properties, including weaknesses and strengths. A blood type that is positive means that persons red blood cells carry a protein called Rh, also known as the RhD antigen. Negative blood type does not.

Epidemiologists have long known that blood type plays a role in how peoples bodies react to infectious diseases, and type A positive and negative appears to be among the most problematic.

For example, people with type A blood have a higher chance of developing certain cancers, particularly stomach cancer. All the different types of blood have agreeable and disagreeable qualities, but type A is associated with higher levels of the stress hormone cortisol, according the National Institutes of Health.

Swartzberg said people with type A blood are also more likely to contract the most virulent form of malaria, known as plasmodium falciparum. The protozoan parasite is transmitted through the bite of a female mosquito.

On the other hand, people with type O blood are less likely to develop inflammation during infections, suffer from heart disease, pancreatic cancer or contract parasitic diseases like falciparum.

The Journal of Medicine study sequenced the genomes of 1,980 COVID-19 patients in Spain and Italy who had suffered respiratory failure and compared their results with an approximately equal number of people who were not sick. The researchers concluded that people with type A blood had as much as a 45 percent higher risk of getting severely ill from the coronavirus.

Another study, of more than 2,000 people in China last March, also found that blood group A had a significantly higher risk of coronavirus infection. That information aligns with other studies, most of them not yet peer reviewed.

In each case, type O blood was linked to lower risk and less severe illness. A study by the genomics site 23andMe calculated that people with blood type O were 9% to 18% less likely to contract COVID-19 than people with other types of blood.

Type O blood is handy in other ways. O positive is the most common blood type, and O negative is compatible with all other types of blood. Because O negative blood can be given to anybody, it is commonly used for transfusions.

Studies have shown that people with type O blood also get fewer blood clots, a serious problem among COVID-19 patients.

SARS-CoV-2, the specific coronavirus that causes COVID-19, is essentially a tiny parasite that uses its tell-tale spike proteins to latch onto the much larger human cells, like pepper on an egg. The virus uses the cells receptors to worm its way inside, where it replicates itself billions of times and spreads throughout the body.

There are a variety of factors that influence vulnerability to COVID-19 infection, including old age, underlying medical conditions and possibly race, although the high mortality rate among minorities is more likely related to poverty and a lack of medical care. A study, published Wednesday in Nature, said Latino and African Americans are three times more likely than white people to be infected by the coronavirus and nearly twice as likely to die.

Men are hospitalized and die from the virus more often than women, a disparity that researchers have linked to testosterone, the male sex hormone.

Researchers know that the coronavirus targets ACE2 receptors, a protein on the surface of human cells that normally helps regulate blood pressure. Peter Chin-Hong, a professor of medicine and infectious diseases at UCSF, said the genes that make the ACE2 receptors are next to the genes that provide the blood type codes.

Because they are so close to each other they influence each other in ways we don't understand, Chin-Hong said. Things are next to each other for a reason.

Nobody knows exactly how the coronavirus operates, but some scientists believe the virus, when it infects a new host, carries with it genetic coding blood type antigens from its last victim. Apparently, type O blood adapts better to the coronavirus coding.

Swartzberg said this may have something to do with the types of carbohydrates, or sugars, on the surface of red blood cells.

The type A carbohydrate may facilitate the entrance of the protozoan into the red blood cell, causing more severe infection, Swartzberg said. People with type O blood, which doesnt have any of those carbohydrates, may be somewhat protected.

George Rutherford, a UCSF infectious disease specialist, said caucasians of Mediterranean descent have the highest percentage of type A blood.

Most of these (blood type) observations are from Italy and Spain, which have had horrendous COVID outbreaks, Rutherford said.

A big puzzle is that blood type doesnt seem to matter when it comes to African Americans and other people of color. Type O blood is more common among African Americans a little more than half carry that type yet African Americans have disproportionately high infection rates. The same goes for Latinos, 57 percent of whom carry type O blood.

Its an indication, Rutherford said, that socioeconomic problems like poverty, obesity and stress may be bigger factors in who gets the disease and how ill they become than blood type.

Peter Fimrite is a San Francisco Chronicle staff writer. Email: pfimrite@sfchronicle.com Twitter: @pfimrite

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If you have this blood type, studies show youre at higher risk for the coronavirus - San Francisco Chronicle

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

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

We may have mapped the human genome in 2003, but a new worldwide study has discovered the links between our genes and the conditions that ail us.

In this time of extra focus on health, allow me one more story from the brave new world of medical research. Every day, twenty American war veterans kill themselves. That is fifteen percent of the total amount of Americans who take their own lives each year; a disproportionally high number. A few weeks ago, the American Department of Veteran Affairs learnt a little more about why that might be the case.

While trauma is involved, surprisingly so is genetics. Research that looked at an astounding 200,000 veterans, concluded that quite a few of them were susceptible to anxiety and depression even before they were sent to Afghanistan or another warzone. In fact, in an astonishing number, there was a problem with a gene called MAD1C1, that is also implicated in bipolar disease and schizophrenia. On top of that, there were five other genetic variants that are linked to anxiety that were more prevalent in this group. Obviously, this is important information. Not only will it now be possible to better predict who should and shouldnt go to war, but deaths can also be prevented by teaching people how to cope before the shit hits the fan.

With 200,000 participants, this research is the largest ever study into anxiety in the world. But it is not the only mass investigation into illness that is going on at the moment. In fact, bigger is definitely better at almost all laboratories on the planet. For instance, a few months ago researchers looked closer at insomnia than had ever been possible before: 1.3 million people were involved, and 956 genes were found that could hold the key to solving a problem that a third of the general population suffers from. An issue, too, that is implicated in all manner of mental health issues, as well as diabetes and cardiovascular disease.

This kind of research is part of Genome-Wide Association Studies that are taking place from New York to Melbourne and Cape Town to Oslo. As you may remember, in 2003 the Human Genome Project was completed, and that meant that suddenly researchers could look into genetic contributions to common diseases better than ever before.

Until the human genome was mapped, the only way to look at the role genes played in illnesses was to study families. That was relatively successful if they were suffering from a single gene disorder, but not so much if it was more complicated than that. But after humanity cracked the gene code in 2003, Biobanks started springing up everywhere. At the moment, weve got forty-five in NSW alone, and the largest in the southern hemisphere is at the RPA in Camperdown. It is run by NSW Health and stores more than three million human samples for use in research. Usually, that is left-over tissue from an operation, biopsy or blood test, of course, donated with written consent. At Camperdown, researchers can apply for access to those samples, so they can investigate whatever illness they are looking at at a much larger scale than pre-2003.

These studies, as usual, involve one group of people with an illness and a control group without. But because so many samples are available, it is possible to look at enormous populations. That means you are casting a wide net, but because there is no hypothesis before you start, anything can happen. The focus, of course, is finding the genes that are associated with a particular disease. And once youve found those, you can zoom in and look a little closer. This has two consequences: first of all, that you can know more about more illnesses much faster than before. Secondly, it is laying the groundwork for personalised medicine.

In the near future, it will no longer be one size fits all (like one type of chemo for everybody with bowel cancer, for instance). Treatments will be tailored to one individual patient, because when we know more about one persons particular gene make-up it is easier to design something that will be just right for them. Not just when they are already sick, but even in the prevention of that illness. Less guesswork, less adverse reactions to treatments, fewer mistakes.

Of course, there are limitations. Not everything can be explained by looking at genes, for instance, and every person responds differently to disease, which makes treatment still complicated. Also, completing a complete genome sequencing is still expensive. And the problem with quite a few of the Biobanks is that the owners of the samples are generally white and Western. Apart from that, just knowing which genes are associated with a disease is only the beginning.

The challenge is the road from that knowledge to new drugs, diagnostics and maybe prevention. Nevertheless, so far over three thousand GWA studies have been done, into almost two thousand different diseases. We now know more about what causes heart attacks (from a study started in 2004), have found a protein that is involved in producing macular degeneration and can pinpoint genes that are related to risky behaviour, like driving too fast, smoking, drinking and having high-risk sex. We have found the genes connected to intelligence, obesity, schizophrenia, childhood aggression, antisocial behaviour, depression and all manner of other things.

There are Biobanks in NSW that specialise in melanoma, stroke, sleep, childrens cancer, gynaecological issues and problems with the brain. I know it is a little brave new world, and we need to be careful it doesnt turn into an Orwellian nightmare. But limitless possibilities, and hope for those who are sick: there is something to be said for that, isnt there?

For this story I have used the following sources:

https://nsw.biobanking.org/locator

https://www.smh.com.au/healthcare/biggest-biobank-in-the-southern-hemisphere-to-revolutionise-medical-research-in-nsw-20171113-gzk5os.html

NSW Health Statewide Biobank

https://www.researchgate.net/publication/331328430_Genome-wide_analysis_of_insomnia_in_1331010_individuals_identifies_new_risk_loci_and_functional_pathways

https://www.researchgate.net/publication/330368016_Genome-wide_association_analyses_of_risk_tolerance_and_risky_behaviors_in_over_1_million_individuals_identify_hundreds_of_loci_and_shared_genetic_influences

https://www.researchgate.net/publication/330368016_Genome-wide_association_analyses_of_risk_tolerance_and_risky_behaviors_in_over_1_million_individuals_identify_hundreds_of_loci_and_shared_genetic_influences

https://edition.cnn.com/2020/01/09/health/anxiety-genetic-association-wellness-trnd/index.html?utm_source=twCNN&utm_content=2020-01-10T05%3A09%3A03&utm_medium=social&utm_term=link

https://www.mentalhealth.va.gov/suicide_prevention/data.asp

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Worldwide genome research could change the course of medical history - The Big Smoke Australia

Genetic tests, also called DNA tests, are used to identify changes in DNA sequences or chromosomal structures. Genetic testing also includes measuring the consequences of genetic alterations, such as RNA analysis as an output of gene expression, and biochemical analysis to measure specific protein outputs.

The Europe Genetic testing services market is expected to reach US$ 5,840.9 Mn in 2027 from US$ 2,521.6 Mn in 2019. The market is estimated to grow with a CAGR of 11.4% from 2020-2027.

Genetic testing is a type of medical test that identifies changes in chromosomes, genes, or proteins. The results of genetic tests can help identify or rule out suspicious genetic conditions or determine the likelihood of someone developing or inheriting a genetic disorder.

A medical device is any device intended to be used for medical purposes. Medical devices benefit patients by helping health care providers diagnose and treat patients and helping patients overcome sickness or disease, improving their quality of life.

The healthcare industryis undergoing rapid transformations since a few years now. Various technological improvementshave been witnessedin the segments including diagnosis and treatment options for chronic diseases. The increase in incidences of chronic illnesses and the increasing ageing population are the primary factors fuelling the growth of healthcare segment.

The Europe Genetic Testing Servicesmarketis growing along with the healthcare industry, but the market is likely to slow down its growth due to the shortage of skilled professionals, suggests the Business Market Insights report.

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France has well-developed policies and strategies in place for improving the prevention of hereditary cancers. Also, France is planning to develop a national plan for personalized medicine. Genomic Medicine France 2025, which was published in 2016, which appeals for healthcare and manufacturing firms to pilot genomic sequencing platforms. By 2020 the aim is to establish a network of centers able to process around 235,000 samples for whole genome sequencing.

These factorsare expectedto offer broad growth opportunities in the healthcare industry and this is expected to cause the demand forimmunochemistryassays in the market.

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EUROPE GENETIC TESTING SERVICES MARKET SEGMENTATION

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Europe Genetic Testing Services Market is expected to reach US$ 5840.9 Million by 2027 with CAGR of 11.4%. - Owned

Theres encouraging and not so encouraging news about COVID-19 testing.

The most common tests used to diagnose an infection with the novel coronavirus are almost 100 percent effective if administered correctly.

However, the same cant be said of tests to determine if youve already had the disease and have developed antibodies.

Experts say diagnostic testing is one of the most powerful public health tools for fighting the spread of the coronavirus.

The tests identify people who may need treatment. Results also trace those who have been in contact with other individuals to help prevent the transmission of the disease further. This can assist epidemiologists in determining how widely the virus has spread.

Testing makes the enemy visible, said Dr. Emily Volk, an assistant professor of pathology at the University of Texas-Health in San Antonio and president-elect of the College of American Pathologists (CAP).

There are two basic types of tests for the novel coronavirus. One type diagnoses an infection and the other tests for antibodies.

Diagnostic tests detect active infections. This is the test you want if you think youve been exposed to the coronavirus or are exhibiting symptoms of COVID-19.

There are currently two types of diagnostic tests available.

The RT-PCR nasopharyngeal tests are more widely used and more familiar. Most involve sticking a 6-inch swab deep into your nose to collect virus samples to test.

However, some more recently approved RT-PCR tests seek to avoid the discomfort associated with the nasopharyngeal swab tests by allowing samples to be collected via a shallow swab of the nose or by testing saliva for the presence of the virus.

If performed correctly, RT-PCR swab tests would be pretty close to 100 percent accurate, Volk told Healthline.

We should be diagnosing people with PCR tests because they are the most accurate, added Dr. Christina Wojewoda, a pathologist at the University of Vermont and vice chair of CAPs microbiology committee.

To get the most accurate results, RT-PCR tests should be conducted 8 days after suspected exposure or infection, to ensure that enough viral material is present to detect.

Some clinicians know that, but people who are swabbing may not be passing that information along, Wojewoda told Healthline.

Its also possible to administer the test too late, after the body has successfully fought off the disease, according to Dr. William Schaffner, professor of medicine in the division of infectious diseases at the Vanderbilt University School of Medicine in Tennessee and medical director of the National Foundation for Infectious Diseases.

The test must also be administered properly, which means inserting the swab 3 inches or so to reach the cavity where the nasal passages meet the pharynx.

If youve had this test and it wasnt uncomfortable, it wasnt done correctly, Schaffner told Healthline.

False-positive results, while rare, can occur with PCR tests, said Wojewoda, because the coronavirus genetic material may linger in the body long after recovery from an infection.

You cant tell if the person [had an infection] 3 days ago or 5 months ago, she said.

Swabs are also used to collect samples for antigen testing. These tests have the advantage of yielding faster results (hours rather than several days).

Theyre also less accurate than RT-PRC tests, mostly because they require test samples to contain large amounts of virus proteins to yield a positive result.

False-negative results from antigen tests may range as high as 20 to 30 percent.

If an antigen test is positive, you can believe it, said Wojewoda. If its negative, you have to question that.

As the name suggests, these tests look for antibodies made by your immune system in response to an infection with the new coronavirus.

Antibody tests are not diagnostic tests.

Antibodies can take several days or weeks to develop after you have an infection and may stay in your blood for several weeks after recovery, according to the Food and Drug Administration (FDA). Because of this, antibody tests should not be used to diagnose an active coronavirus infection.

Antibody tests also arent terribly useful.

Ideally, a positive antibody test would tell you that youve recovered from COVID-19 or a coronavirus infection and have immunity from future infections, allowing you to return to work, travel, and socialization without the risk of transmitting the infection or becoming sick again yourself.

However, researchers dont yet know whether the presence of antibodies means that you have immunity, whether you could still get sick from a different strain of the virus, or how long immunity lasts.

Antibody tests are problematic because they can be misused easily, said Volk. You may think if you have a positive antibody test that you dont have to wear a mask or conform to social distancing, but antibodies dont tell us that you have immunological armor against future infections.

Antibody tests also are subject to false-positive results.

The job of antibodies is to stick to things, so they can create a positive test result if they react to a different type of coronavirus, said Wojewoda.

Antibody tests show the most promise if the way the human body controls the coronavirus is with an antibody response, Wojewoda added. If not, it doesnt make any difference.

For example, she said, its T cells, not antibodies, that help the body fight an HIV infection.

Thats another piece of data that needs to be figured out before testing can be figured out, Wojewoda said.

Every COVID-19 test currently (and legally) available in the United States has been approved by the FDA under the agencys Emergency Use Authorization (EUA) protocol.

The EUA permits the FDA to allow unapproved medical products or unapproved uses of approved medical products to be used in an emergency to diagnose, treat, or prevent serious or life threatening diseases or conditions caused by chemical, biological, radiological and nuclear threat agents when there are no adequate, approved, and available alternatives.

That has allowed novel coronavirus tests to quickly hit the market without the research and testing normally required for FDA approval.

To date, the FDA has approved 130 different RT-PCR, antigen, and antibody tests for the new coronavirus.

Doing a full clinical trial takes a long time, but we need tests now, said Sherry Dunbar, PhD, senior director of global scientific affairs for Luminex Corporation, which manufactures a pair of PRC tests and has submitted an application to the FDA for emergency approval of a new antigen test.

Experts generally agree that the RT-PCR tests are more accurate and useful than antigen and antibody tests, which are better used as confirmatory tools.

Dunbar told Healthline that some testing labs are using multiple tests to anticipate shortages on testing products. Theyre also using the quicker tests when demand is high and the slower but more accurate tests on weekends or during slower times.

Wojewoda said that while some tests promise quicker results than others, the biggest limiting factor to turnaround results is shortages of reagents the chemicals used to do the testing.

Im not looking for a new test, she said. Those on the market are as accurate and fast as they need to be. We have the instruments we need to test. We just need more stuff to do it with.

As with most other things regarding the novel coronavirus, pathologists and testing labs are learning about COVID-19 on the fly, said Dunbar.

Never in my career have I seen anything like this, where the public is discussing and analyzing the data at the same time as the researchers, she said. Were basing our response on past knowledge of other viruses, but as we like to say, the bugs dont read the book. What happened in the past can help us prepare, but things will continue to evolve.

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How Accurate Are the Coronavirus Diagnostic and Antibody Tests? - Healthline

NEW YORK & OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (Takeda) (TSE:4502/NYSE:TAK) and the New York Academy of Sciences announced today the Winners of the third annual Innovators in Science Award for their excellence in and commitment to innovative science that has significantly advanced the field of rare disease research. Each Winner receives a prize of US $200,000.

The 2020 Winner of the Senior Scientist Award is Adrian R. Krainer, Ph.D., St. Giles Foundation Professor at Cold Spring Harbor Laboratory. Prof. Krainer is recognized for his outstanding research on the mechanisms and control of RNA splicing, a step in the normal process by which genetic information in DNA is converted into proteins. Prof. Krainer studies splicing defects in patients with spinal muscular atrophy (SMA), a devastating, inherited pediatric neuromuscular disorder caused by loss of motor neurons, resulting in progressive muscle atrophy and eventually, death. Prof. Krainers work culminated notably in the development of the first drug to be approved by global regulatory bodies that can delay and even prevent the onset of an inherited neurodegenerative disorder.

Collectively, rare diseases affect millions of families worldwide, who urgently need and deserve our help. Im extremely honored to receive this recognition for research that my lab and our collaborators carried out to develop the first approved medicine for SMA, said Prof. Krainer. As basic researchers, we are driven by curiosity and get to experience the thrill of discovery; but when the fruits of our research can actually improve patients lives, everything else pales in comparison.

The 2020 Winner of the Early-Career Scientist Award is Jeong Ho Lee, M.D., Ph.D, Associate Professor, Korea Advanced Institute of Science and Technology (KAIST). Prof. Lee is recognized for his research investigating genetic mutations in stem cells in the brain that result in rare developmental brain disorders. He was the first to identify the causes of intractable epilepsies and has identified the genes responsible for several developmental brain disorders, including focal cortical dysplasias, Joubert syndromea disorder characterized by an underdevelopment of the brainstemand hemimegalencephaly, which is the abnormal enlargement of one side of the brain. Prof. Lee also is the Director of the National Creative Research Initiative Center for Brain Somatic Mutations, and Co-founder and Chief Technology Officer of SoVarGen, a biopharmaceutical company aiming to discover novel therapeutics and diagnosis for intractable central nervous system (CNS) diseases caused by low-level somatic mutation.

It is a great honor to be recognized by a jury of such globally respected scientists whom I greatly admire, said Prof. Lee. More importantly, this award validates research into brain somatic mutations as an important area of exploration to help patients suffering from devastating and untreatable neurological disorders.

The 2020 Winners will be honored at the virtual Innovators in Science Award Ceremony and Symposium in October 2020. This event provides an opportunity to engage with leading researchers, clinicians and prominent industry stakeholders from around the world about the latest breakthroughs in the scientific understanding and clinical treatment of genetic, nervous system, metabolic, autoimmune and cardiovascular rare diseases.

At Takeda, patients are our North Star and those with rare diseases are often underserved when it comes to the discovery and development of transformative medicines, said Andrew Plump, M.D., Ph.D., President, Research & Development at Takeda. Insights from the ground-breaking research of scientists like Prof. Krainer and Prof. Lee can lead to pioneering approaches and the development of novel medicines that have the potential to change patients lives. Thats why we are proud to join with the New York Academy of Sciences to broadly share and champion their workand hopefully propel this promising science forward.

Connecting science with the world to help address some of societys most pressing challenges is central to our mission, said Nicholas Dirks, Ph.D., President and CEO, the New York Academy of Sciences. In this third year of the Innovators in Science Award we are privileged to recognize two scientific leaders working to unlock the power of the genome to bring innovations that address the urgent needs of patients worldwide affected by rare diseases.

About the Innovators in Science Award

The Innovators in Science Award grants two prizes of US $200,000 each year: one to an Early-Career Scientist and the other to a well-established Senior Scientist who have distinguished themselves for the creative thinking and impact of their research. The Innovators in Science Award is a limited submission competition in which research universities, academic institutions, government or non-profit institutions, or equivalent from around the globe with a well-established record of scientific excellence are invited to nominate their most promising Early-Career Scientists and their most outstanding Senior Scientists working in one of four selected therapeutic fields of neuroscience, gastroenterology, oncology, and regenerative medicine. Prize Winners are determined by a panel of judges, independently selected by the New York Academy of Sciences, with expertise in these disciplines. The New York Academy of Sciences administers the Award in partnership with Takeda.

For more information please visit the Innovators in Science Award website.

About Takeda Pharmaceutical Company Limited

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

About the New York Academy of Sciences

The New York Academy of Sciences is an independent, not-for-profit organization that since 1817 has been committed to advancing science, technology, and society worldwide. With more than 20,000 members in 100 countries around the world, the Academy is creating a global community of science for the benefit of humanity. The Academy's core mission is to advance scientific knowledge, positively impact the major global challenges of society with science-based solutions and increase the number of scientifically informed individuals in society at large. Please visit us online at http://www.nyas.org.

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Takeda and the New York Academy of Sciences Announce 2020 Innovators in Science Award Winners - Business Wire

Hansa grants Sarepta exclusive license to develop and promote imlifidase as a potential pre-treatment prior to the administration of gene therapy in Duchenne muscular dystrophy and Limb-girdle muscular dystrophy, for patients with neutralizing antibodies (NAbs) to adeno-associated virus (AAV).

Under the terms of the license: Hansa will receive a USD 10 million upfront payment and is eligible for up to USD 397.5 million in development, regulatory and sales milestone payments. Hansa will book all sales of imlifidase and would be eligible for royalties in the high single-digits to mid-teens on any gene therapy sales enabled through pre-treatment with imlifidase in NAb-positive patients.

Lund, Sweden July 2, 2020. Hansa Biopharma (Hansa), the leader in immunomodulatory enzyme technology for rare IgG mediated diseases, announced today that it has entered into an agreement with Sarepta Therapeutics Inc. (Sarepta), the leader in precision genetic medicine for rare diseases, through which Sarepta is granted an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy treatment in Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD). The pre-treatment is intended for patients with pre-existing neutralizing antibodies (NAb-positive patients) to adeno-associated virus (AAV), the technology that is the basis for Sareptas gene therapy products.

Sarepta will be responsible for conducting pre-clinical and clinical studies with imlifidase and any subsequent regulatory approvals. Sarepta will also be responsible for the promotion of imlifidase as a pre-treatment to Sareptas gene therapies following potential approval.

Under the terms of the agreement, Hansa will receive a USD 10 million upfront payment, and is eligible for a total of up to USD 397.5 million in development, regulatory and sales milestone payments. Hansa will book all sales of imlifidase, and earn high single-digit to mid-teens royalties on Sareptas incremental gene therapy sales when treating NAb-positive patients enabled through pre-treatment with imlifidase.

Sren Tulstrup, President & CEO of Hansa Biopharma comments,We see significant potential for our enzyme technology in the gene therapy space overall, and we are excited to partner with Sarepta, a leading player in the field, to use the unique features of imlifidase to potentially enable gene therapy treatment in patients who today arent eligible for these breakthrough therapies due to pre-existing neutralizing antibodies in two conditionswith a very high unmet medical need.

Doug Ingram, President & CEO, Sarepta Therapeutics said,As we expand our leadership position in genetic medicine and build out our gene therapy engine, one of Sareptas central ambitions is to find scientific solutions that bring our potentially life-saving therapies to the greatest number of the rare disease patients we serve. One of the current limitations of gene therapy is the inability to treat patients who have pre-existing neutralizing antibodies to the AAV vector. While our AAVrh74 vector has been associated with a low screen out rate for neutralizing antibodies, even that low rate is inconsistent with our mission.

In pre-clinical and clinical models, Hansas technology has shown the ability to clear the IgG antibodies that prevent dosing AAV-based gene therapies. If successful, this could offer the potential of extending our gene therapy treatments to DMD and LGMD patients who would otherwise have been denied access due to pre-existing antibodies.

Hansa Biopharma will be hosting a conference call with President & CEO Sren Tulstrup, CSO & COO Christian Kjellman and CFO Donato Spota.

Conference Call Partnership agreement with Sarepta TherapeuticsA conference call will take place July 2nd, 2020 at 10:00am CET. The audio cast will be recorded and subsequently be available on the Hansa website https://hansa.eventcdn.net/202007

Participants dial-in numbersSE: + 46 81 241 09 52UK: + 44 203 769 6819US: + 1 646 787 0157

This is information that HansaBiopharma AB is obliged to makepublic pursuant to the EU MarketAbuse Regulation.

About imlifidaseImlifidase is a unique antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets IgG and inhibits IgG-mediated immune response. It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration. CHMP/EMA has adopted a positive opinion, recommending conditional approval of imlifidase for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. Endorsement of the positive opinion by the European Commission is expected in the third quarter of 2020.Hansa has also reached an agreement with the FDA on a regulatory path forward for imlifidase in kidney transplantation of highly sensitized patients in the U.S. and has three ongoing phase 2 trials in autoimmune diseases and post-transplant indications.

About gene therapy and neutralizing antibodiesGene therapy is a growing and revolutionizing treatment technology in which healthy gene sequences are inserted into cells of a patient. The treatments are potentially curative in monogenic diseases like hemophilia and muscular dystrophy through a single dose. Harmless recombinant viruses are used to carry the healthy genes into the cell. Due to the partial viral origin of the gene therapy constructs, a certain subset of patients carry neutralizing anti-AAV antibodies towards gene therapy products, depending on what AAV serotype being used, forming a barrier for treatment eligibility.Antibodies prevent effective transfer of healthy gene sequence and can be a safety concern. Imlifidase as a pre-treatment may have the potential to eliminate neutralizing antibodies prior to gene therapy. Similarly, imlifidase may have the potential to enable any potentially necessary re-dosing of gene therapy for all patients.

About Duchenne Muscular Dystrophy (DMD)Duchenne muscular dystrophy is a rare genetic disease caused by mutation in the DMD gene, encoding for the protein dystrophin. Duchenne is an irreversible, progressive disease that causes the muscles in the body to become weak and damaged over time. It is eventually fatal and there is no cure. DMD affects one in 3,500 to 5,000 males born worldwide (approximately 400-500 annual cases in the US) and causes muscles in the body to become weak and most patients use wheelchair by the age of 12.

About Limb-Girdle Muscular Dystrophy (LGMD)Limb-girdle muscular dystrophy or (LGMD) is a genetically and clinically heterogeneous group of rare muscular dystrophies. It is characterised by progressive muscle wasting which affects predominantly hip and shoulder muscles. LGMD has an autosomal pattern of inheritance and currently has no known cure or treatment. It can be caused by a single gene defect that affects specific proteins within the muscle cell, including those responsible for keeping the muscle membrane intact. LGMD has a global prevalence of approximately 1.63 per 100,000 individuals worldwide.

For further information, please contact:Klaus Sindahl, Head of Investor RelationsHansa Biopharma Mobile: +46 (0) 709-298 269E-mail: klaus.sindahl@hansabiopharma.com

About Hansa BiopharmaHansa Biopharma is leveraging its proprietary immunomodulatory enzyme technology platform to develop treatments for rare immunoglobulin G (IgG)-mediated autoimmune conditions, transplant rejection and cancer.The Companys lead product candidate, imlifidase, is a unique antibody-cleaving enzyme that potentially may enable kidney transplantation in highly sensitized patients with potential for further development in other solid organ transplantation and acute autoimmune indications. CHMP/EMA has adopted a positive opinion, recommending conditional approval of imlifidase for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. Endorsement of the positive opinion by the European Commission is expected in the third quarter of 2020. Hansas research and development program is advancing the next generation of the Companys technology to develop novel IgG-cleaving enzymes with lower immunogenicity, suitable for repeat dosing in relapsing autoimmune diseases and oncology.Hansa Biopharma is based in Lund, Sweden and also has operations in Europe and US.

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Hansa Biopharma announces exclusive agreement with Sarepta Therapeutics to develop and promote imlifidase as pre-treatment ahead of gene therapy in se...

There was a time when modern medicine was primitive. There were no antibiotics, so every infection took its own course, leading to decline in health. Hypertension and diabetes were largely untreatable. X-ray was new, and remedies had changed but little from medieval times. No one ever embarked on the goodness of preventative treatment, not to speak of predictive medicine, beyond taking a distasteful cod liver oil capsule.

During the last hundred years, modern medicine has undergone a sea change. Just think of it an ever-expanding repertoire of medicines, high-tech procedures, therapies and reams of clinical data to employ when one gets sick. Yet modern medicine remained (in)complete, notwithstanding the therapeutic advances.

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Things are now changing thanks to the integration of all such advances, from how a persons diet interacts with ones unique genetic profile to how environmental pollutants affect our thinking, not to speak of preventative medical approaches in health and wellness. The bigperestroikahas begun, and it is poised to transform health care for a growing number of people in the near future. Welcome to a whole new world of personalized, bespoke medicine.

Personalized medicine is, in essence, tailored or customized medical treatment. It treats while keeping in mind the unique, individual characteristics of each patient, which are as distinct as ones fingerprint or signature. It also includes scientific breakthroughs in our understanding of how a persons unique molecular and genetic profile makes them susceptible to certain illnesses. Personalized medicine expands our ability to envisage medical treatments that would not only be effective but also safe for each patient while excluding treatments that may not provide useful objectives.

Personalized medicine is, in simple terms, the use of new methods of molecular scrutiny. It is keyed to help better manage a patients illness or their genetic tendency toward a particular illness or a group of diseases. In so doing, it aims to achieve optimal therapeutic outcomes by helping both clinicians and patients choose a disease management approach that is likely to work best in the context of the patients unique genetic and environmental summary. In other words, it allows to accurately diagnose diseases and their sub-types while prescribing the best form and dose of medication most suited to the given patient.

Personalized, or precision, medicine is not rocket science it is, in essence, an extension of certain traditional approaches to understanding and treating disease. What jazzed up the therapeutic fulcrum of personalized medicine are tools that are more precise. This is what also offers clinicians better insights for selecting a treatment protocol based on a patients molecular profile. Such a patient-specific methodology, as has been practiced for long in certain complementary and alternative medical (CAM) or integrative approaches, not only curtails harmful side effects but also leads to more successful outcomes, including reduced costs in comparison to the current trial-and-error approach to treatment, which has distressingly come to the fore during these extraordinary and unprecedented times of COVID-19.

It is still early days, but the fact remains that personalized medicine has changed the old ways of how we all thought about, identified and managed health issues. As personalized medicine increasingly bids fair to an exciting journey in terms of clinical research and patient care, its impact will only further expand our understanding of medical technology.

What personalized medicine has done is bring about a paradigm shift in our thinking about people in general and also specifically. We all vary from one another what we eat, what others eat, how we react to stress or experience health issues when exposed to environmental factors. It is agreed that such variations play a role in health and disease. It is also being incrementally accepted that certain natural variations found in our DNA can influence our risk of developing a certain disease and how well we could respond to a particular medicine.

All of us are unique individuals, perhaps with the exemption of identical twins, albeit the genomes are unique in them, too. While we are genetically similar, there are small differences in our DNA that are unique, which also makes us distinctive in terms of health, disease and our response to certain medicinal treatments.

Personalized medicine is poised to tap natural variations found in our genes that may play a role in our risk of getting or not getting certain illnesses, along with numerous external factors, such as our environment, nutrition and exercise. Variations in DNA can, likewise, lead to differences in how medications are absorbed, metabolized and used by the body. The understanding of such genetic variations and their interactions with environmental factors are elements that will help personalized medicine clinicians to produce better diagnostics and drugs, and select much better treatments and dosages based on individual needs not as just fixing a pill or two, as is the present-day conventional medical practice.

It is established that a majority of genes function precisely as intended. This gives rise to proteins that play a significant role in biological processes while allowing or helping an individual to grow, adapt and live in their environment. It is only in certain unusual situations, such as a single mutated or malfunctioning gene, that our apple cart is disturbed. This leads to distinct genetic diseases or syndromes such as sickle cell anemia and cystic fibrosis. In like manner, multiple genes acting together can impact the development of a host of common and complex diseases, including our response to medications used to treat them.

New advances will revolutionize bespoke medical treatment with the inclusion of drug therapy as well as recommendations for lifestyle changes to manage, delay the onset of disease or reduce its impact. Not surprisingly, the emergence of new diagnostic and prognostic tools has already raised our ability to predict likely outcomes of drug therapy. In like manner, the expanded use of biomarkers biological molecules that are associated with a particular disease state has resulted in more focused and targeted drug development.

Molecular testing is being expansively used today to identify breast cancer and colon cancer patients who are likely to benefit from new treatments and to preempt recurrences. A genetic test for an inherited heart condition is helping clinicians to determine which course of treatment would maximize benefit and minimize serious side effects while bringing about curative outcomes.

Such complexities exist for asthma and other disorders too. This is precisely where molecular analysis of biomarkers can help us to identify sub-types within a disease while enabling the clinician to monitor their progression, select appropriate medication, measure treatment outcomes and patients response. Future advances may make biomarkers and other tools affordable and allow clinicians to screen patients for relevant molecular variations prior to prescribing a particular medication.

It is already clear that personalized medicine promises three strategic benefits. In terms of preventative medicine, personalized medicine will improve the ability to identify which individuals are predisposed to develop a particular condition. A better understanding of genetic variations could also help scientists identify new disease subgroups or their associated molecular pathways and design drugs to target them. This could also help select patients for inclusion, or exclusion, in late-stage clinical trials. Finally, it will allow to work out the best dosage schedule or combination of drugs for each individual patient.

Yet not everything is hunky-dory for personalized medicine. Critics of precision medicine believe that the whole idea is too much of overhyped razzmatazz, among other things. Proponents, however, argue that when it comes to managing our own health, most of us are used to the idea of taking a one-size-fits-all approach be it medicines, supplements, diets and diagnoses. This may be wrong.

What works, as they put it, for one may be a gaffe for another. As the award-winning oncologist and medical technology innovator, Dr. David B. Agus, author of the groundbreaking bookThe End of Illness, puts it, each patients individual risk factors are based on ones DNA, the environment and a preventative lifestyle plan in response. He begins with simple, profound pointers: How is your sense of smell? and Is your ring finger longer than your middle finger? He explains with statistics-backed guidelines that moving and walking regularly is mandatory because exercising and then sitting is equivalent to smoking cigarettes, while eating and sleeping at consistent hours is imperative because irregularity causes inflammation.

The inference is obvious: We should all understand our physiology and quiz doctors with the thorough, exploratory frame of mind of a gadget buyer. This holds the key to making medicine truly personal, more humane, effective and safe while keeping in mind the individual in us all as unique and distinctive, the sum of the whole not just the parts.

The views expressed in this article are the authors own and do not necessarily reflect Fair Observers editorial policy.

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The Future of Medicine Is Bespoke - Fair Observer

CAMBRIDGE, Mass., June 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the retirement of Sandy Mahatme, Sareptas executive vice president, chief financial officer and chief business officer, from the company effective July 10, 2020. The company has commenced a search process to identify the future chief financial officer. During the interim period, the finance and accounting functions will report directly to Sareptas Chief Executive Officer, Doug Ingram, and other departments reporting to Mr. Mahatme will be overseen by members of Sareptas executive committee.

The Sarepta from which Sandy retires is a very different one from the organization he joined as our chief financial officer some eight years ago. And the Sarepta of today a financially solid biotechnology organization with perhaps the industrys deepest and most valuable pipeline of genetic medicine candidates with the potential to extend and improve lives would not have been possible without Sandys business acumen and dedication, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. On behalf of our board of directors and the entire organization, I want to wish Sandy all the best in his next journey and thank him for his invaluable and numerous contributions to our success and for having built a strong team of finance leaders who will continue to perform as he departs.

Said Mr. Mahatme, It has been a privilege to serve as Sareptas CFO and CBO for almost eight years and to have participated in its remarkable transformation and extraordinary growth. Working with this leadership team and our talented colleagues, we have built a strong foundation for Sareptas ongoing success in achieving its goal of changing the lives of patients with rare diseases around the world. Having built a strong team of finance, IT, facilities, manufacturing and business development professionals, I feel confident that this is a good time to transition to other opportunities, knowing that Sarepta is well-positioned to continue to lead the industry.

Sandy will continue to serve on the Board of Directors for Flexion Therapeutics, Inc., Aeglea BioTherapeutics, Inc., and Idorsia Pharmaceuticals Ltd.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking StatementThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the search process to identify the future chief financial officer, the reporting structure during the interim period and the performance of the finance team; Sareptas potential to extend and improve lives; Sareptas goal of changing the lives of patients with rare diseases around the world; and Sarepta being well-positioned to continue to lead the industry.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Announces Retirement of Sandy Mahatme, Chief Financial Officer and Chief Business Officer - BioSpace

Researchers identified a protein that can not only worsen skin inflammation but also plays a key role in damaging joints and bones of patients with psoriasis.

Patients with psoriasis show higher rates of diverse comorbid conditions, such as psoriatic arthritis (PsA), which occurs in one-third of patients with psoriasis and can cause severe, disabling joint disease. However, the reason why so many people with psoriasis develop PsA hasnt been clear.

Since the damage that occurs as a result of PsA is irreversible, identifying patients with PsA early, before too much damage is done to bones, tendons, and joints, is an important consideration, researchers noted.

A team led by Case Western Reserve University School of Medicine researchers discovered that normalizing KLK6 can eliminate skin inflammation and reduce the arthritis-like damage.

"To discover that turning down KLK6 eliminated the skin inflammation and even improved the arthritis-like changesthat was unbelievable," Nicole Ward, PhD, the study's principal investigator and a professor of nutrition and dermatology at the medical school, said in a statement. "This suggests that clinicians need to aggressively treat patients with psoriasis to prevent the arthritis changes, which generally occur after the skin disease presents itself. Since the joint and bone damage are largely irreversible in patients, prevention becomes critical."

In previous research, Ward found that the skin of patients with psoriasis had 6 times more KLK6 than normal. In addition, the PAR1 receptor protein, which causes cellular/tissue responses like inflammation when activated, is overproduced in these patients skin and immune cells. The theory that came from these findings was that KLK6 drove inflammation through signaling of PAR1.

In this new study, the researchers overproduced KLK6 through genetic engineering to develop psoriasis-like skin disease. When PAR1 was deleted, there was a reduction in skin inflammation, as well as an improvement in bone and joint problems.

"These findings suggest that chronic inflammation originating in the skin has the capacity to cause distant joint and bone destruction seen in arthritis, according to Ward.

Reference

Billi AC, Ludwig JE, Fritz Y, et al. KLK6 expression in skin induces PAR1-mediated psoriasiform dermatitis and inflammatory joint disease. J Clin Invest. 2020;130(6):3151-3157. doi:10.1172/JCI133159

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Connection Between Psoriasis and Joint Disease Indicates Early Treatment Can Be Key - AJMC.com Managed Markets Network

During the coronavirus pandemic, it's unlikely that AI doctors would work at all: the depth of moral decisions that need to be made simply can't be accommodated by a program.Vidal Balielo Jr.

By now, its almost old news that artificial intelligence (AI) will have a transformative role in medicine. Algorithms have the potential to work tirelessly, at faster rates and now with potentially greater accuracy than clinicians.

In 2016, it was predicted that machine learning will displace much of the work of radiologists and anatomical pathologists. In the same year, a University of Toronto professor controversially announced that we should stop training radiologists now. But is it really the beginning of the end for some medical specialties?

AI excels in pattern identification in determining pathologies that look certain ways, according to Elliot Fishman, a radiology and oncology professor at Johns Hopkins University and a key proponent of AI integration into medicine. Ultimately, specialties that rely heavily on visual pattern recognition notably radiology, pathology, and dermatology are those believed to be at the greatest risk. With the advent of virtual primary care services, such as Babylon, General Practice may also have to adapt in the future.

Pattern recognition functions

In January of this year, an article in Nature reported that AI systems outperformed doctors in breast cancer detection. This was carried out by an international team, including researchers from Google Health and Imperial College London on mammograms obtained from almost 29,000 women. Screening mammography currently plays a critical role in early breast cancer detection, ensuring early initiation of treatment and yielding improved patient prognoses. False negatives are a significant problem in mammography. The study found AI use was associated with an absolute reduction of 9.4% and 2.7% reduction in false negatives, in the USA and UK, respectively. Similarly, use of the AI system led to a reduction of 5.7% and 1.2% in the USA and UK respectively for false positives. The study suggested that AI outperformed the six radiologists individually, and was equivalent to the current double-reading system of two doctors currently used in the UK. These developments have already had perceptible consequences in practice: algorithms eliminate the need for a second radiologist when interpreting mammograms. However, critically, one radiologist remains responsible for the diagnosis.

AI can also be deployed to predict the cognitive decline that leads to Alzheimers disease... allowing early intervention and treatment

Earlier studies have also yielded similar results: a 2017 study published in Nature examined the use of algorithms in dermatology. The study, from Stanford University, involved an algorithm developed by computer scientists using an initial database of 130,000 skin disease images. When compared to the success rates of 21 dermatologists, the algorithm was almost equally successful. Likewise, in a study conducted by the European Society for Medical Oncology, it was found that AI exceeded the performance of 58 international dermatologists. A system reliant on a form of machine learning known as Deep Learning Convolutional Neural Network (CNN) missed fewer melanomas (the most lethal form of skin cancer), and misdiagnosed benign moles (or nevi) as malignant less often than the group of dermatologists.

Further applications in medicine

However, the prospects of AI technology extend beyond the clear applications in cancer diagnosis and radiology: recent studies have also demonstrated that AI may be able to detect genetic diseases in infants by rapid whole-genome sequencing and interpretation. Considering that time is critical in treating gravely ill children, such automated techniques can be crucial in diagnosing children who are suspected of having genetic diseases.

In addition, AI can also be deployed to predict the cognitive decline that leads to Alzheimers disease. Such computational models can be highly valuable at the individual level, allowing early intervention and treatment planning. FDA approval has also been granted to a number of companies for such technologies; these include Imagens OsteoDetect, an algorithm intended to aid wrist fracture detection. In addition, algorithms may have functions in other specialties such as anaesthesiology in monitoring and responding to physiological signs.

Limitations of AI

Despite the benefits that AI integration into clinical practice can provide, the technology is not without limitations. Machine learning algorithms are highly dependent on the quality and quantity of the data input, typically requiring millions of observations to function at suitable levels. Biases in data collection can heavily impact performance; for instance, racial or gender representation in the original data set can lead to differences in diagnostic abilities of the system for different groups, consequently leading to disparities in patient outcomes. Considering that certain pathologies, including melanoma, present differently between races and with different incidences, this can often lead to both later diagnoses and poorer outcomes for racial minorities, as found in a number of studies. Volunteer bias of the data collected is also a pertinent consideration; for example, although lactate concentration is a good predictor of death, this is not routinely measured in healthy individuals.

Considering the magnitude of what is at stake raises the question of whether it is appropriate to rely solely on machines without any human input.

Other key problems which may arise include how algorithms overfit predictions based on random errors in the data, resulting in unstable estimates which vary between data samples. In addition, clinicians may take a more cautious approach when making a diagnosis. Therefore, it may appear that a human underperforms compared to an algorithm since their actions may yield a lower accuracy in tumour identification, however this approach could lead to a lower number of critical cases missed.

Ultimately, the tendency for humans to favour propositions given by automated systems over non-automated ones, known as automation bias, may exacerbate these problems.

Attempts to replace GPs with AI have been unsuccessful

The success of AI integration into clinical practice crucially depends on the receptiveness of patients. Babylon, a start-up company based in the UK, was developed to give medical advice to patients using chat services. Although Babylon has been referred to as the biggest disruption in medical practice in years and a game-changer in UK media as quoted on Babylons website it is questionable how successful the service has been so far Babylon has been slow in recruiting patients and this month, it came under fire for data breaches. The fact that patients lose access to their regular GP if they sign up to Babylon is perhaps a key contributing factor for Babylons slow take-off. Therefore, it appears that human contact is highly valued by patients, after all, at least for some medical specialties.

Potential effect of COVID-19

The COVID-19 pandemic, with its requirements for social distancing, could potentially accelerate the use of AI. COVID-related restrictions could change the perception of patients about remote medical consultations, paving the way for increased receptiveness to primary healthcare apps including Babylon. The pandemic has also highlighted the inadequacies in fast internet access throughout the country. This may encourage increased government investment into broadband infrastructure, which may, in turn, facilitate broader penetration of AI technology. The increased pressure on the NHS may also encourage greater use of algorithms to delegate menial tasks as seen in specialties such as radiology already.

The future

AI will likely become an indispensable tool in clinical medicine, facilitating the work of professionals by automating mundane, albeit essential tasks. By reducing the medical workload, this could allow healthcare professionals to dedicate greater efforts to other aspects of their work, including patient interaction. As emphasised by the President of the Royal College of Radiologists, radiologists can instead focus more of their time on interventional radiology and in managing more complex cases to a much greater extent. Indeed, innovation may aid clinicians and augment their decision-making capabilities to improve their efficiency and diagnostic accuracy, however it remains doubtful whether technology can fully replace these roles. After all, considering the magnitude of what is at stake human life raises the question of whether it is appropriate to rely solely on machines without any human input. Therefore, it remains likely that human involvement will need to continue across medical specialties, although this may be in a reduced or adapted form.

Varsity is the independent newspaper for the University of Cambridge, established in its current form in 1947. In order to maintain our editorial independence, our newspaper and news website receives no funding from the University of Cambridge or its constituent Colleges.

We are therefore almost entirely reliant on advertising for funding, and during this unprecedented global crisis, we have a tough few weeks and months ahead.

In spite of this situation, we are going to look at inventive ways to look at serving our readership with digital content for the time being.

Therefore we are asking our readers, if they wish, to make a donation from as little as 1, to help with our running cost at least until we hopefully return to print on 2nd October 2020.

Many thanks, all of us here at Varsity would like to wish you, your friends, families and all of your loved ones a safe and healthy few months ahead.

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The rise of AI in medicine - Varsity Online