StemCell Therapy

As the world awaits the arrival of a safe and effective coronavirus vaccine, a team of researchers has come forward with a provocative new theory: that masks might help to crudely immunize some people against the virus.

The unproven idea, described in a commentary published Tuesday in the New England Journal of Medicine, is inspired by the age-old concept of variolation, the deliberate exposure to a pathogen to generate a protective immune response. First tried against smallpox, the risky practice eventually fell out of favor, but paved the way for the rise of modern vaccines.

Masked exposures are no substitute for a bona fide vaccine. But data from animals infected with the coronavirus, as well as insights gleaned from other diseases, suggest that masks, by cutting down on the number of viruses that encounter a persons airway, might reduce the wearers chances of getting sick. And if a small number of pathogens still slip through, the researchers argue, these might prompt the body to produce immune cells that can remember the virus and stick around to fight it off again.

You can have this virus but be asymptomatic, said Dr. Monica Gandhi, an infectious disease physician at the University of California, San Francisco, and one of the commentarys authors. So if you can drive up rates of asymptomatic infection with masks, maybe that becomes a way to variolate the population.

That does not mean people should don a mask to intentionally inoculate themselves with the virus. This is not the recommendation at all, Dr. Gandhi said. Neither are pox parties, she added, referring to social gatherings that mingle the healthy and the sick.

The theory cannot be directly proven without clinical trials that compare the outcomes of people who are masked in the presence of the coronavirus with those who are unmasked an unethical experimental setup. And while outside experts were intrigued by the theory, they were reluctant to embrace it without more data, and advised careful interpretation.

It seems like a leap, said Saskia Popescu, an infectious disease epidemiologist based in Arizona who was not involved in the commentary. We dont have a lot to support it.

Taken the wrong way, the idea could lull the masked into a false sense of complacency, potentially putting them at higher risk than before, or perhaps even bolster the incorrect notion that face coverings are entirely useless against the coronavirus, since they cannot render the wearer impervious to infection.

We still want people to follow all the other prevention strategies, Dr. Popescu said. That means staying vigilant about avoiding crowds, physical distancing and hand hygiene behaviors that overlap in their effects, but cant replace one another.

The coronavirus variolation theory hinges on two assumptions that are difficult to prove: that lower doses of the virus lead to less severe disease, and that mild or asymptomatic infections can spur long-term protection against subsequent bouts of sickness. Although other pathogens offer some precedent for both concepts, the evidence for the coronavirus remains sparse, in part because scientists have only had the opportunity to study the virus for a few months.

Experiments in hamsters have hinted at a connection between dose and disease. Earlier this year, a team of researchers in China found that hamsters housed behind a barrier made of surgical masks were less likely to get infected by the coronavirus. And those who did contract the virus became less sick than other animals without masks to protect them.

A few observations in humans seem to support this trend as well. In crowded settings where masks are in widespread use, infection rates seem to plummet. And although face coverings cannot block all inbound virus particles for all people, they do seem to be linked to less illness. Researchers have uncovered largely silent, symptomless outbreaks in venues from cruise ships to food processing plants, all full of mostly masked people.

Data linking dose to symptoms have been gathered for other microbes that attack the human airway, including influenza viruses and the bacteria that cause tuberculosis.

But despite decades of research, the mechanics of airborne transmission largely remain a black box, said Jyothi Rengarajan, an expert in vaccines and infectious disease at Emory University who was not involved in the commentary.

That is partly because it is difficult to pin down the infectious dose required to sicken a person, Dr. Rengarajan said. Even if researchers eventually settle on an average dose, the outcome will vary from person to person, since factors like genetics, a persons immune status and the architecture of their nasal passages can all influence how much virus can colonize the respiratory tract.

And confirming the second half of the variolation theory that masks allow entry to just enough virus to prime the immune system might be even trickier. Although several recent studies have pointed to the possibility that mild cases of Covid-19 can provoke a strong immune response to the coronavirus, durable protection cannot be proven until researchers gather data on infections for months or years after these have resolved.

On the whole, the theory has some merits, said Angela Rasmussen, a virologist at Columbia University who was not involved in the commentary. But Im still pretty skeptical.

It is important to remember, she said, that vaccines are inherently less dangerous than actual infections, which is why practices like variolation (sometimes called inoculation) eventually became obsolete. Before vaccines were discovered, doctors made do by rubbing bits of smallpox scabs or pus into the skin of healthy people. The resulting infections were usually less severe than smallpox cases caught the typical way, but people definitely got smallpox and died from variolation, Dr. Rasmussen said. And variolation, unlike vaccines, can make people contagious to others.

Dr. Gandhi acknowledged these limitations, noting that the theory should not be construed as anything other than that a theory. Still, she said, Why not drive up the possibility of not getting sick and having some immunity while were waiting for the vaccine?

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A New Theory Asks: Could a Mask Be a Crude Vaccine? - The New York Times

Peng Y, Mentzer AJ, Liu G, Yao X, Yin Z, Dong D, Dejnirattisai W, Rostron T, Supasa P, Liu C, Lpez-Camacho C, Slon-Campos J, Zhao Y, Stuart DI, Paesen GC, Grimes JM, Antson AA, Bayfield OW, Hawkins DEDP, Ker DS, Wang B, Turtle L, Subramaniam K, Thomson P, Zhang P, Dold C, Ratcliff J, Simmonds P, de Silva T, Sopp P, Wellington D, Rajapaksa U, Chen YL, Salio M, Napolitani G, Paes W, Borrow P, Kessler BM, Fry JW, Schwabe NF, Semple MG, Baillie JK, Moore SC, Openshaw PJM, Ansari MA, Dunachie S, Barnes E, Frater J, Kerr G, Goulder P, Lockett T, Levin R, Zhang Y, Jing R, Ho LP, Cornall RJ, Conlon CP, Klenerman P, Screaton GR, Mongkolsapaya J, McMichael A, Knight JC, Ogg G, Dong T. Broad and strong memory CD4+ and CD8+ T cells induced by SARS-CoV-2 in UK convalescent individuals following COVID-19. Nat. Immunol.2020 Sep 04 [Epub ahead of print]. doi: 10.1038/s41590-020-0782-6. PMID: 32887977. View full text

Link:
Robust T Cell Response and Immune Memory in Recovered COVID-19 Patients - Medscape

One of the major challenges facing gene therapy - a way to treat disease by replacing a patients defective genes with healthy ones - is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the current available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses - from which dangerous disease-causing genes have been removed - as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts like a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up or genome. This means that researchers like me can leverage CRISPR technology to revolutionize gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

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A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the quantity of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibody against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology,provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

Here is the original post:
CRISPR can help combat the troubling immune response against gene therapy - The Conversation US

Since the first vaccine was developed in 1796, vaccinations have been phenomenally successful at preventing infectious diseases, and wiping out some altogether.

The latest video in our new YouTube series, Science with Sam, explains how vaccines work by training your immune system to recognise viruses and bacteria. Ever wondered how flu vaccines are made or why you need a new one every year? Click play to find out.

We also take a look at the unprecedented worldwide effort to develop a vaccine for the coronavirus, and consider the challenges involved in making, testing and distributing covid-19 vaccines.

Tune in every week toyoutube.com/newscientistfor a new episode, or check back tonewscientist.com

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Most of us have never had to worry about getting smallpox, polio or diphtheria. A hundred years ago, these diseases were common killers. Now, smallpox is a thing of the past, while polio and diphtheria are very rare in most of the world. The reason? Vaccines.

Vaccines are a way of training the immune system for a big fight, so that when it comes up against the same opponent in the future, it knows exactly how to defeat it.

When you encounter a virus or bacterium for the first time, your body has a hard time fighting it. But over time, it learns to recognise the danger. Your immune system produces powerful proteins called antibodies that target and eliminate disease-causing microbes.

After you recover from an infection, specialised cells remain in your blood and keep a memory of that pathogen theyre called memory cells so the next time you face the pathogen, your body can quickly produce the right antibodies to fight it off.

Vaccines are a clever way of harnessing this mechanism to make us immune to a disease. They are made of weakened or killed viruses or bacteria that trigger an immune response, without making us ill.

The first vaccine was developed in 1796 by Edward Jenner. At the time It was commonly believed that dairymaids were immune to smallpox because they were commonly exposed to cowpox, which is a related but less deadly virus. Jenner decided to test this idea by injecting an 8-year-old boy with pus from a dairy maids cowpox lesions. A few months later he injected the boy with smallpox and found the boy was indeed immune. Who said ever said science was pretty?

Since then, vaccination programmes have been extremely successful at preventing diseases, and even eliminating some altogether. Smallpox, a disease that killed around 300 million people in the 20th century, was finally eradicated in 1980.

What about the coronavirus? Right now, there are over 100 vaccines currently in development. Scientists have moved incredibly quickly, considering we knew almost nothing about this virus at the start of this year. It normally takes years to get to this point. The fastest vaccine ever developed before was for Ebola and that took five years.

There are several different ways to make a vaccine being tried. One is to use whole viruses that have been inactivated so they cant cause a full infection. This might be done by killing them with heat.

Then there are vaccines that are made from live viruses, but they are weakened so they wont grow well in the human body. These vaccines tend to generate a strong and long-lasting immune response. For example the MMR vaccine, for measles, mumps and rubella, contains three live viruses.

Some vaccines put viral molecules into the body the important bits that our immune cells can recognise rather than whole viruses. This is how the injected flu vaccine works, and several groups are working on coronavirus vaccines like this.

The flu virus, or influenza, is a particularly difficult virus to vaccinate against, because there are many variants, or strains, that circulate. Its like the virus has many hats, and changes them often, and that makes it hard for our immune system to recognise it when a new one comes around. To produce the vaccine, the virus has to be grown in chicken eggs in a process that takes many months and millions of eggs. Every year virologists have to predict which flu strains are going to dominate in the next season, so that companies can make enough doses to meet demand. Sometimes they get it wrong, and even when theyre right, the mutating virus might mean the vaccine doesnt work as well as we hoped.

What we really want is a universal flu vaccine based on parts of the virus that dont change. Research on that is ongoing, but unfortunately it doesnt get as much investment as it needs

A relatively new approach that is being investigated is to make a vaccine containing the genetic instructions for making viral proteins, in the form of DNA or RNA. Once inside the body, the genetic code causes a persons cells to produce distinctive proteins normally found on the virus. These proteins trigger an immune response, and that creates an immunological memory.

The University of Oxford is working on a vaccine that uses a harmless virus to deliver the viral genes into cells. Its based on a virus that causes colds in chimpanzees, but its been genetically modified so that it cant reproduce in humans. The first clinical trials have shown that it is safe and induces a strong immune response.

For all the latest news on vaccines and the coronavirus why not subscribe to New Scientist? Theres a special discount code for you: get a 20 per cent discount by entering the code SAM20.

Despite all the effort being put into coronavirus vaccines, theres no guarantee that this will give us the magic bullet were looking for. Theres so much we dont know about how our immune systems respond to the virus and it may not even be possible to generate long-term immunity. A vaccine might mean you just get a bit less sick from the disease, and you might need a new dose each year.

Then there is the challenge of manufacturing and distributing billions of doses to every country in the world. It may take years to make enough to go around, raising tricky questions about who should be first in line. Some countries including the UK have made deals to get their hands on the first shipments, before the vaccines have been proven to work.

To defeat the pandemic, we need to make sure everyone who needs vaccines has access to them. We need to think globally, cooperatively, and act less like children squabbling over cookies.

To successfully wipe out a disease, a large proportion of the population needs to be inoculated, creating what is known as herd immunity, meaning there are enough immune people in the population to stop a virus from circulating. That brings us to another problem: some people are deeply sceptical about vaccines, which could be an obstacle to getting vaccination rates up to the levels required.

This mistrust has been fuelled by unfounded scare stories, such as the false idea that the MMR vaccine causes autism. All vaccines have to be rigorously assessed for their safety before they are used widely, and continue to be monitored after they are rolled out. Large clinical trials have repeatedly found no link between the MMR vaccine and autism.

Despite this, rising anti-vaccination sentiment has led to a surge in measles in the US,with more than 1000 cases reported in 2019. And in a recent survey, 1 in 4 people in the US said they wouldnt take a coronavirus vaccine if it was available.

According to the World Health Organization, immunisations prevent an estimated 2-3 million deaths every year, in people from all age groups. By any measure, vaccines are one of the most successful innovations humankind has ever come up with. The coronavirus has reminded us just how vulnerable we are to infections, and that new diseases can emerge at any moment. Vaccines are our best hope for defeating them.

What is a black hole? And could you survive one?

Is our reality just one part of a multiverse?

More on these topics:

Read more from the original source:
What is a vaccine and how do they work? Find out in Science with Sam - New Scientist

In a season when we would usually be out cheering on our local sports teams, we are spending more time at home and repeating a new mantra: Wash your hands, practice social distancing, wear a mask.

But what if you could play offense instead of defense to fend off colds and viruses? What if adjusting your daily habits could build your immunity to help your body fend off illness, not only this year but every year?

Thats not only possible, says Katie Moksnes Bowman, its something she encourages her patients to do every day.

Stress is the number one way we increase inflammation in the body, says Moksnes Bowman, a licensed acupuncturist and Doctor of Acupuncture and Chinese Medicine (DACM) for Northwestern Health Sciences University. She says inflammation can affect digestion, sleep patterns, pain, and your bodys immunity.

The key to improving your immunity is to reduce inflammation in your body.

The amount of stress that has been created from the pandemic is causing issues for people physically and emotionally, she says. In Chinese medicine, your digestion matters, sleep matters, your immune system matters.

When I am in practice with a patient, we talk about sleep, bowels, diet and movement at every single treatment. I really want to work with them where theyre at.

She sees patients ranging from professional athletes to seniors with mobility issues and everyone in between, so there is no one-size-fits-all approach to treatment.

In Chinese medicine, we really view the body as a whole, she says. For example, if a patient has shoulder pain, Moksnes Bowman proceeds knowing the shoulder does not work independently from the rest of the body.

"The amount of stress that has been created from the pandemic is causing issues for people physically and emotionally. In Chinese medicine, your digestion matters, sleep matters, your immune system matters." Kate Moksnes Bowman, Northwestern Health Sciences University

If you are not digesting your foods properly, if youre not getting a good nights sleep, she says, I can do a ton of work on your shoulder, but its not going to repair well.

To help patients improve their health and build their immunity, she suggests small changes in diet and exercise, such as drinking enough water, reducing caffeine and sugar consumption, adding anti-inflammatory foods to their diet, and getting more movement every day.

I am not going to overhaul your whole diet, she says. If you do not want to stop eating pizza, I cannot make you stop eating pizza. But she might suggest that you try goat cheese on your pizza or sample a cauliflower crust.

I see myself as a reminder person, she says. I have patients come in and I say, How did your diet go this week? Did you eat something green? That means a plant, you know, not a green Jolly Rancher.

That question always gets a laugh, but the point is that little changes can make a difference in reducing inflammation and improving immunity.

When we are talking about diet and exercise, both of those things reduce inflammation and so does sleeping. Sleeping is a time to repair your body, Moksnes Bowman says. Asked what tops her list as the most important step, she says: Its not a hierarchy for me. Its more of a circle than a list, because all of those things are going to influence the next thing.

Small adjustments in diet and exercise are something patients do on their own between clinic visits, where Moksnes Bowman and other practitioners offer a range of therapies, from acupuncture and massage to cupping, Gua Sha, herbal medicine and even recipes to help improve your immunity.

If you have a lot of stress and are getting the common cold five times a winter, I would suggest you consider herbal medicine, she says. She advises against buying supplements in the grocery aisle. Seek a health professional who is specialized before taking Vitamin D, C or Elderberry syrup. They are all really good things, but theyre not always the right thing for everybody. Its always important to make sure you are taking the right amount.

Creating good sleep habits and a good sleeping environment are important, too. If you are on your phone or watching TV at night, the blue light from the device stimulates a part of the brain that doesn't allow you to fall asleep as well, she says.

Improved diet and exercise, combined with acupuncture or other types of Chinese medicine, can reduce inflammation over time by increasing blood flow and releasing endorphins, which Moksnes Bowman describes as that calm, happy hormone. That is our own bodys way of reducing pain in the body.

And that calm, happy hormone can lead to a good nights sleep, as described in a text from one of Moksnes Bowmans patients, who said: I cant believe how much my sleep improved by getting acupuncture.

The results arent anecdotal, she says. Sleep-tracking devices demonstrate that acupuncture can improve sleep; they record how well and deeply you are sleeping and if you are waking frequently during the night.

And while youre getting those extra ZZZs, your body is resting and fortifying its immunity.

One of the side effects of social distancing and working from home has been an increase in loneliness. Moksnes Bowman says that after a brief shutdown of the NWHSU Bloomington Clinic several months ago, she noticed two things when the clinic reopened: Patients who had missed appointments were in pain, and they were lonely.

People wanted to talk for so long, she says. I made my treatments a bit longer so patients could just talk, because people were feeling lonely.

She and other practitioners frequently refer patients to therapists, Tai Chi or Pilates instructors or others when they see an opportunity to help the patient move, relax or sort things out. Taking a deep breath and getting some release is also good for building a sense of well-being.

Think of amping up your immune system as the ultimate DIY project. Add some green to your diet, make sure you drink enough water, cut out some caffeine and get enough sleep for starters, and then add some acupuncture or massage. Together those steps can help fortify your immunity.

And keep in mind that this year, none of that replaces the need to frequently wash your hands, socially distance wherever possible and wear a mask when its not.

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Located in Bloomington,Northwestern Health Sciences Universityis a pioneer in integrative natural health care education, offering degree programs in chiropractic, acupuncture, Chinese medicine, massage therapy, medical assisting, medical laboratory programs, post-bac/pre-health, radiation therapy, and B.S. completion. At press time, itsBloomington clinicis open to the public and services include chiropractic care, Chinese medicine, massage therapy, naturopathic medicine, Bloomington Clinic offers integrative, natural care for the entire family in one location.

Each monththe Bloomington Clinic providers host a Provider Talks webinar that discusses topics from foot health to the ABZzzzs of Sleep to Promoting Health through the Seasons. Learn more about the webinar serieshere.

Telemedicine is a convenient way to care for yourself during these unprecedented times. Appointment times vary depending on the service. Providers are part ofNorthwestern Health Sciences University, a non-profit industry leader in integrative and natural healthcare education that provides access to the latest evidence and state-of-the-art technology so you get the natural solutions you truly need.

See more content fromNorthwestern Health Sciences University.

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Immune-Boosting Strategies to Stay Ahead of the Cold - Mpls.St.Paul Magazine

As we continue to navigate the global pandemic more attention is being focused on a long sought after vaccine. Dr. Greg talks withJerry Woodward, a professor of Microbiology, Immunology and Molecular Genetics who led a preclinical study at UK on one particular vaccine that shows promise. He also discusses the efficacy of producing a shot once a vaccine is tested and approved.

From UK Now:

PDS Biotechnology, a clinical stage immunotherapy company,has announcedpositive results from preclinical testing conducted at the University of Kentucky College of Medicine of its COVID-19 vaccine candidate, PDS0203.

PDS0203 stands out among COVID-19 vaccines currently in development because it includes a vaccine technology pioneered by PDS Biotech called Versamune, which stimulates important parts of the immune system to activate T cells.

Versamuneactivates an important immunological signaling pathway known to be essential in the induction of both anti-viral and anti-tumor immune responses.It also more efficient at presenting the disease-associated protein to immunological pathways that activate both CD8 (killer) and CD4 (helper) T cells that can recognize, kill and protect against a specific disease.

The most effective vaccines stimulate both antibody and T cells because both of those arms of the immune system are important to eliminate different viral infections, said Jerry Woodward, a professor of Microbiology, Immunology and Molecular Genetics who led the preclinical study at UK. While many of the vaccines out there stimulate a good antibody response, they dont always activate T cells. Thats one of the key advantages of the Versamunetechnology.

PDS0203 combines Versamunenanoparticles with a protein recognized by the human immune system that is derived from SARS-CoV-2, the virus that causes COVID-19. Prototype vaccines were tested at UK based on various recombinant SARS-CoV-2 proteins, including protein constructs created by UKs Protein Core lab.

In mouse models, PDS0203 showed strong activation of both protective antibodies as well as highly active and potent virus-specific CD8 killer and CD4 helper T cells within 14 days of treatment.PDS Biotech is submitting the findings to a peer-reviewed scientific journal and is expected to advance the PDS0203 vaccine to a phase 1 safety and immunogenicity clinical trial in humans.

To test the antibody arm of the immune response, mice are vaccinated and serum is separated from blood samples and tested for the amount of antibodies that will bind to the protein. To test the T cell arm of the immune response, T cells are isolated from the mice and tested for their ability to directly respond to peptides, or short pieces of protein, which are derived from the SARS-Cov-2protein. The results have been promising, Woodward says.

Vaccination with the recombinant proteins including the spike protein alone elicits lower levels of antibody response and almost no T cell response, Woodward said. When Versamuneis added, it dramatically increases both the antibody and T cell immune responses.

PDS Biotech contracts with expert labs like Woodwards for independent analysis of vaccine efficacy and research in the development of novel cancer therapies and infectious disease vaccines. Woodwards lab has been working with the company for several years, and recently received funding from the National Institutes of Allergy and Infectious Diseases to complete preclinical development of a Versamune-based universal influenza vaccine to provide broad protection against multiple strains of the flu virus. This spring, the focus of Woodwards collaboration with PDS Biotech expanded primarily from cancer to address the COVID-19 pandemic.

Versamunepresents unique potential for a vaccine to provide the level of immune response needed for protection against COVID-19, Woodward says.

Were optimistic an effective COVID-19 vaccine will be available relatively soon. There are probably over a hundred companies working on different vaccines and a lot of them are probably going to work," Woodward said."I believe that the Versamune-based vaccine due to its mechanism of action, simplicity and preliminary efficacy and human safety data, has excellent potential to be one of the more successful global vaccines.

Read more:
Meet A COVID Vaccine Researcher Here At UK This Week On Dr. Greg - WUKY

The medtech industry has borne the brunt of the COVID-19, and though strained for revenues, it has shown resilience and commitment by working unrelentingly to ensure availability of products (for both COVID and non-COVID emergencies) across the country. The medtech industry is in dire need of stimuli says Pavan Choudary, Chairman & Director General, Medical Technology Association of India and reiterates the policy changes required to reinvigorate the sector and ease some of the burden placed on this nascent industry, Chairman & Director General, Medical Technology Association of India narrates how the sector has also ensured that regular servicing and maintenance of capital equipment at the hospitals continues despite the financial blows it has taken.

The medtech industry is now in dire need of stimuli says Choudary and reiterates the policy changes required to reinvigorate the sector and ease some of the burden placed on this nascent industry

The unprecedented situation created by COVID-19 has resulted in alarm bells ringing across industries. After multiple lockdowns to curb the spread of infection, we are now in the phase of unlock 3.0 where businesses are cautiously starting to resume operations.

The corridor of uncertainty in the last six months has battered Indias economy which is projected to contract by 4.5 per cent this fiscal according to the International Monetary Fund (IMF). The collateral effect of the lockdowns has been sorely felt as several sectors including automobile, aviation, textiles, tourism, hospitality, real estate and electronics had almost completely come to a standstill. During this time, and in the absence of a vaccine or a reliable therapeutic remedy, the country relied on its healthcare and medical technology industry to spearhead the fight against COVID-19; and they have delivered, with uncanny grit and commitment.

The medtech sector has shown great determination in the face of adversity to ensure continued supply of critical medical devices and services, despite being bogged down by several operational issues like scarcity of contractual manpower for loading and un-loading, restrictive movement of service personnel and goods in several areas, stalling of customs clearance of imports, etc. The industry is also reeling under taxing financial challenges like escalation in costs due to the hike in freight charges, at the same time it has been hit by significant fall in revenue due to postponement and cancellation of elective procedures which drive a large part of the demand for medical devices.

It is estimated that medical technology industry has suffered a 50-85 per cent drop in revenue across categories in April-June due to the situation. Slicing the data category wise shows us that the cardiology category experienced a downfall in revenue up to 60 per cent in the first quarter of the financial year. The orthopaedic industry encountered an even bigger fall in revenue to an extent of up to 85 per cent. The opthalmology sector also recorded a similar impact as its revenue fell up to 75 per cent during April-June, 2020. Even the critical care device segment fell to nearly half. These challenges have been further compounded by the falling INR value, high basic customs duty rate and the newly imposed health cess on imported medical devices.

The medical device industry has borne the brunt of these issues, and has been strained for revenues, it is bleeding, yet has shown resilience and commitment by working unrelentingly to ensure availability of products (for both COVID and non-COVID emergencies) across the country. It has also ensured that regular servicing and maintenance of capital equipment at the hospitals continues despite the financial blows it has taken.

Several global medtech companies have even gone further by bringing COVID specific product designs and specifications (ventilators, face shields, medical beds) from the US into India to work with local manufacturers to manufacture in India and plug any potential holes in the supply.

Another significant impact of COVID-19 situation has been on the employment across sectors in India. According to data from the Center for Monitoring Indian Economy (CMIE), Indias overall unemployment rate as on August 13, 2020 stood at 7.9 per cent and is expected to settle at a higher level than seen before the COVID-19 induced lockdown. In the medical device sector, the effects of shrinking margins of hospitals can be seen at the channel and service dealership networks, and sub-dealers are already experiencing significant workforce downsizing. So far the global medtech fraternity have heeded to the governments request of protecting the jobs of its workforce, even while facing significant financial blows. However if the situation does not improve, these efforts may not be sustainable.

The medtech industry is in dire need of stimuli; to reinvigorate the sector the government must look towards easing some of the burden it has placed on this nascent industry. For the next steps, the government should consider removing the high customs duty on imported medical devices this would also increase healthcare affordability for patients at a time they need it the most, if this is not possible then at the minimum the government must roll back the additional burden of health cess imposed on imported medical devices, it must also look at ways to facilitate the resumption of elective procedures by issuing safety SOPs/ guidelines, this will go a long way in restoring the revenue streams of the medtech industry. The sector which is championing the fight against the pandemic cannot be allowed to crumble, for India will not be able to sustain its fall.

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How Indian medtech is battling the COVID-19 pandemic - Express Healthcare

Overview Of Absorbable and Non-Absorbable Sutures Industry 2020-2028:

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The Absorbable and Non-Absorbable Sutures Market analysis summary by Reports Insights is a thorough study of the current trends leading to this vertical trend in various regions. Research summarizes important details related to market share, market size, applications, statistics and sales. In addition, this study emphasizes thorough competition analysis on market prospects, especially growth strategies that market experts claim.

Absorbable and Non-Absorbable Sutures Market competition by top manufacturers as follow: B. Braun Melsungen AG, Ethicon, Smith & Nephew, Demetech Corporation, Conmed Corporation, W.L. Gore & Associates, Tepha, Meta Biomed, Zimmer Biomet, CP Medical, Miltex (A Integra LifeSciences Corporation Company), Assut Medical Sarl, DyNek Pty, Footberg, Stoelting, Unimed Medical Industries, Teleflex Incorporated, Boston Scientific Corporation, Medtronic, Surgical Specialties Corporation, Resorba Medical GmbH, Medline Industries, AD Surgical, Santec Medicalprodukte, Clinisut, Coreflon, Aspen Surgical, Medipac, Sutures, Pierson Surgical

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The global Absorbable and Non-Absorbable Sutures market has been segmented on the basis of technology, product type, application, distribution channel, end-user, and industry vertical, along with the geography, delivering valuable insights.

The Type Coverage in the Market are:

Absorbable SuturesNon-Absorbable Sutures

Market Segment by Applications, covers:

CardiovascularOrthopedicGynecologyOpthalmologyGeneral SurgeryOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

Major factors covered in the report:

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New Comprehensive Report on Absorbable and Non-Absorbable Sutures Market to Witness an Outstanding Growth during 2020 2028 with Top Players Like...

-Parkinsons Disease R&D Day to be hosted by Gavin Corcoran, MD, Chief R&D Officer, on October 30th, 2020, with leading movement disorder specialists

NEW YORK and BASEL, Switzerland, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the company will provide a corporate overview and participate in 1:1 meetings at upcoming virtual investor and patient conferences in September. Additionally, the company announced plans for their 1st Parkinsons Disease R&D Day with key opinion leaders.

Investor and Patient Conference Presentation details:

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. A replay will be available for approximately 30 days following the conference.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

Source:Axovant Gene Therapies

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Axovant Announces First Parkinson's Disease Gene Therapy R&D Day and Participation in Upcoming September Conferences" - GlobeNewswire

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the current available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts like a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up or genome. This means that researchers like me can leverage CRISPR technology to revolutionise gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the quantity of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibody against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology,provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

Samira Kiani, Associate Professor of Pathology, University of Pittsburgh

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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CRISPR Can Help Combat the Troubling Immune Response Against Gene Therapy - Gizmodo Australia

New Jersey, United States, The Gene Therapy Market report 2020 provides a detailed impression, describe the product industry scope and the market expanded insights and forecasts up to 2027. It shows market data according to industry drivers, restraints and opportunities, analyzes the market status, the industry share, size, future Trends and growth rate of the market. The Gene Therapy Market report is categorized by application, end user, technology, product / service types, and other, as well as by region. In addition, the report includes the calculated expected CAGR of chitosan acetate-market derivative from the earlier records of the Gene Therapy Market, and current market trends, which are organized with future developments.

Gene Therapy Market was valued at USD 3.69 Billion in 2019 and is projected to reach USD 24.78 Billion by 2027, growing at a CAGR of 26.9% from 2020 to 2027.

Gene Therapy Market, By Product

Viral Vectorso Adeno-associated virus vectorso Retroviral vectors Gammaretroviral vectors Lentiviral vectorso Other viral vectors (herpes simplex and adenoviral vectors) Non-viral Vectorso Oligonucleotideso Other non-viral vectors (plasmids and RNAi)

Gene Therapy Market, By Indication

Neurological Diseases Cancer Hepatological Diseases Duchenne Muscular Dystrophy Other Indications

Gene Therapy Market, By Delivery Method

Ex vivo In vivo

The report provides detailed coverage of the Gene Therapy Market, including structure, definitions, applications, and Industry Chain classifications. The Gene Therapy Market analysis is provided for the international markets including development trends, competitive landscape analysis, investment plan, business strategy, opportunities and development status of key regions. Development policies and plans are discussed and manufacturing processes and cost structures analyzed. This report also includes information on import / export consumption, supply and demand, costs, industry share, policy, Price, Sales and gross margins.

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Gene Therapy Market forecast up to 2027, with information such as company profiles, product picture and specification, capacity production, price, cost, revenue, and contact information. Upstream raw materials and equipment as well as downstream demand analyses are also carried out. The Gene Therapy Market size, development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and general research results are offered.

The Gene Therapy Market was created on the basis of an in-depth market analysis with contributions from industry experts. The report covers the growth prospects in the coming years and the discussion of the main providers.

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Gene Therapy Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 - The Daily Chronicle

The report covers the forecast and analysis of the gene therapy market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the gene therapy market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the gene therapy market on a global level.

In order to give the users of this report a comprehensive view of the gene therapy market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

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The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the gene therapy market by segmenting the market based on the type, vector type, therapy area, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

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Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market By Industry Type, By Brand And Major Players 2020-2027 - The Daily Chronicle

TEL AVIV, Israel, Sept. 08, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) announced today the successful completion of a Type B pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding the Company's development plan for VB-601. VB-601 is the Companys lead anti-MOSPD2 antibody for immune-inflammatory indications, for which a briefing package for the pre-IND meeting was submitted in June. Based on the FDA's feedback, VBL plans to advance its IND-enabling activities for VB-601 as planned.

We are very pleased with the outcome of the pre-IND meeting with the FDA, said Dror Harats, M.D., CEO of VBL Therapeutics. VB-601 is a first-in-class product candidate that has a completely novel mechanism of action. It can block the ability of monocyte to migrate to inflammatory sites, no matter which molecules try to attract them in. Therefore, it is important that we have reached alignment with the FDA on the path forward for VB-601. With this green light, we plan to continue to advance VB-601, aiming to start a first-in-human study in the second half of 2021.

About VB-601VBL is advancing a drug development program that is exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases. VB-601 is a proprietary anti-MOSPD2 monoclonal antibody which has potential for treatment of multiple chronic inflammatory diseases.

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for cancer. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every two months. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers phase 1 trial as well as in three VBL-sponsored tumor-specific phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding VBLs programs, including VB-601, including their pre-clinical and clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. VBL Therapeutics undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:

Michael RiceLifeSci Advisorsmrice@lifesciadvisors.com(646) 597-6979

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VBL Therapeutics Announces Successful Completion of Pre-IND Meeting with FDA Regarding Proposed Clinical Development of VB-601 - BioSpace

Seeing a child debilitated by illness is never easy.

When doctors tell youthere is nothing they nor you can do to help ease yourbaby's suffering, well, parents who know that type of helplessness often find it hard to describe.

We were told to take my son home and love him, becausehe probably wouldnt live past his second birthday, said Pace father Todd Hamrick. But, were way past that birthday now.

Hamricks son, Alek, was diagnosed at six months old with spinal muscular atrophy and not expected to live long enough to toddle. ButAlek, now 3, has beaten the odds and outlived that initial, bleak prognosis.

His parents attribute much of his success to his doctor, Richard Finkel, who entered Alek into a clinical trial for what they believe has been a wonder drug for their little boy, Evrysdi, which was recently approved by the Food and Drug Administration.

Whats hard to even get around is that people even bothered to research it, Todd Hamrick said. Its just a small amount of the population that has SMA. Its not like researching a blood pressure medication.

Spinal muscular atrophy, or SMA,is a genetic disorder caused by a loss of nerve cells that effect human motor function.

Essentially, those afflicted by SMA are made weak. Their muscleswaste away. In many cases, eventually, a person loses their ability to walk, to eat andevento breathe, and they die.

Aleks mother, Iwona Hamrick, is a nurse at a local hospital and wellremembers the moment she heardher sons diagnosis.

It was unimaginable. His pregnancy was normal, she said. We did genetic testing and it was negative. Unfortunately, at that time, they were not screening for his disease. So, from a healthy baby to a dying baby, you know?

The parents felt they had to travel, in more ways than one, to find the places and help that they could for their Alek.

Todd Hamrick said that he and wife decided to move from Gulf Breeze to Pace after Aleks diagnosis after feeling ostracized by many of their former acquaintances. The parents felt like some people who they used to know were made uncomfortable by their son's illness.

The area is very in-the-dark when it comes to children with issues, Todd Hamrick said. If your kids are healthy and you'rehealthy, its a great area. But, it doesnt attract the greatest talent or best and biggest facilities or endowments.

So, Alek traveled with his family to meet his future doctor, Finkel, at the Nemours Children's Hospital in Orlando.

Alekwas first treated with a gene therapy drug, and he made some response with that, Finkel told the News Journal. But more recently, he started on a second drug, which seems to be having an enhanced effect, I must say.

Finkel, an expert in the field of pediatric neurologic disorders, left Florida in March for a position leading the new Center for Experimental Neurotherapeutics at St. Jude Childrens Research Hospital in Memphis, Tennessee. Buthe has continued to monitor Aleks progress via video.

Both of the drugs he has received, the gene therapy and this new drug, Evrysdi, are designed to increase a certain protein in Aleks body that is deficient because of his genetic disorder, he explained. But they do it in different ways. The potential advantage of his new drug, Evrysdi because its an oral drug; you take it by mouth it goes into the stomach and into the bloodstream, and from there, it goes to all the tissues of the body.

And, we think that there is an enhanced effect, becauseit gets into the muscle tissue, Finkel continued. "These are very, very, early daysin trying to make assessments, soI dont want to say that we can come to any kind of conclusions yet."

However the cutting-edge drug works doesnt matter to a mother, whos just glad that it isworking.

It gave us hope. Thats for sure, Iwona Hamrick said. Becausewe felt helpless.

Since Alek started his new treatment last November, his strength has increased tremendously.

His muscle tone got better, Iwona Hamrick said. He is much stronger in the upper body, so much so, he is pushing his little wheelchair.

Alek can now cruise around his Pace home in an extraordinarily lite-weight wheelchair designed by a Swedish inventor who alsohas a child with SMA.

Alek had a lot of trouble before starting this medication even pushing it, Iwona Hamrick said. Sonowhe is just rolling around the house. Also, he is barring more weight on his legs.

Recently, Alek has started to be able to walk in a pool a huge milestone for the toddler.

But some worries remain the same.

Every day, Alek must use a type of breathing machine.

Its a cough assist machine, Todd Hamrick explained. We use it two times a day, when hes healthy. Becausewhere we can just clear our throats when we cough, he doesnt have that strength no lung strength.

Alek attends physical therapy, aqua-therapy,hippotherapy, occupational therapy and speech therapy sessions every week to try and ensure he remains healthy and continues to properly develop.

My worries have changed a lot, Todd Hamrick said. I used to worry my child was going to die. Now, Im worrying about if other kids will bully him at school.

"But that'sa great worry to have," he continued. "Compared to how it use to be, getting picked on is a great thing to worry about.

Colin Warren-Hicks can be reached at colinwarrenhicks@pnj.com or 850-435-8680.

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Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal

NEW YORK and CLEVELAND, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company will participate in the following upcoming investor conferences.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona’s clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company’s portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona’s novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona’s fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Greg Gin VP, Investor Relations Abeona Therapeutics +1 (646) 813-4709 ggin@abeonatherapeutics.com

Media Contact: Scott Santiamo Director, Corporate Communications Abeona Therapeutics +1 (718) 344-5843 ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics to Participate in Upcoming Investor Conferences - Stockhouse

Triplet therapies have become the accepted standard of care in multiple myeloma, according toMarc J. Braunstein, MD, PhD, who added that emerging quadruplet therapies are also generating excitement in the field.

As seen in the up-front setting, there has been an explosion of therapies, not just for patients with relapsed/refractory multiple myeloma who progress after induction therapy, but also for patients who progress after multiple lines of therapy, said Braunstein, an assistant professor in the Department of Medicine at NYU Long Island School of Medicine. In the field, we appreciate that clonal resistance is truly what leads to shorter remission over time. Now that we have more therapies to offer, we are seeing several new combinations.

In a special episode of OncLiveOn Air, Braunstein, who is also the course co-director of the Hematology-Oncology System and co-director of the Autologous Stem Cell Transplant Program at NYU Winthrop Hospital of NYU Langone Healths Perlmutter Cancer Center, highlighted pivotal research on the use of triplet and quadruplet regimens in the up-front and relapsed/refractory settings presented during the 2020 ASCO Virtual Scientific Program.

VRd Remains the Standard of Care in Newly Diagnosed Multiple Myeloma

Carfilzomib (Kyprolis), lenalidomide (Revlimid), and dexamethasone (KRd) did not improve progression-free survival (PFS) in patients with newly diagnosed multiple myeloma, according to results of the phase 3 ENDURANCE (E1A11) trial (NCT01863550).1

In the randomized analysis, investigators compared the use of KRd with that of bortezomib (Velcade), lenalidomide, and dexamethasone (VRd) in treatment-nave patients with multiple myeloma who had an ECOG performance score of 0, 1, or 2. Patients also had to have acceptable hematological parameters and organ function and measurable disease in serum, urine, or bone marrow to be included. If they had grade 2 or higher peripheral neuropathy or New York Heart Association III or IV heart failure or myocardial infarction less than 6 months before study start, they could not participate.

The first co-primary end point of the trial was PFS for the induction randomization and the second co-primary end point was overall survival (OS) for the second randomization. Key secondary end points included overall response rate, minimal residual disease negativity rate per flow cytometry, time to progression, OS, and toxicity.

In the field, this trial was patiently awaited for some time because we were eager to see the head-to-head comparison of a triplet regimen that includes either the protostome inhibitor bortezomib or carfilzomib in combination with lenalidomide and dexamethasone, the 2 most common up-front regimens in the space, explained Braunstein.

A total of 1087 patients were randomized 1:1 to receive VRd (n = 542) or KRd (n = 545). Patients on the VRd arm received 1.3 mg/m2of bortezomib on days 1, 4, 8, and 11 for cycles 1-8 and the same dose on days 1 and 8 for cycles 9-12; 25 mg of daily lenalidomide on days 1-14; and 20 mg of dexamethasone in on days 1, 2, 4, 5, 8, 9, 11, and 12 for cycles 1-4, and then 10 mg on days 1, 2, 4, 5, 8, 9, 11, and 12 for cycles 5-8, followed by 10 mg on days 1, 2, 8, and 9 for cycles 9-12. Treatment cycles were repeated every 3 weeks for 12 cycles.

Patients on the KRd arm received 20 mg/m2of carfilzomib on days 1 and 2 and 36 mg/m2on days 8, 9, 15, and 16 of cycle 1 followed by 36 mg/m2on days 1, 2, 8, 9, 15, and 16 for cycles 2-9; 25 mg of lenalidomide on days 1-21, and 40 mg of dexamethasone on days 1, 8, 15, and 22 for cycles 1-4 and 20 mg on days 1, 8, 15, and 22 for cycles 5-12. This treatment cycle was repeated every 4 weeks for 9 total cycles.

Results showed a median PFS of 34.4 months (95% CI, 30.1not evaluable) in the VRd arm compared with 34.6 months (95% CI, 28.8-37.8) in the KRd arm (HR, 1.04; 95% CI, 0.83-1.31; P = .742). In addition, on the VRd arm, 21 patients (4.0%) experienced a stringent complete response (sCR), 57 (10.8%) achieved a CR, 263 had a very good partial response (VGPR), and 103 patients had a partial response (PR). On the KRd arm, 31 patients (5.9%) achieved a sCR, 65 (12.4%) had a CR, 292 patients (55.5%) experienced a VGPR, and 68 (12.9%) had a PR. The median OS from induction randomization was not yet reached in either arm.

Moreover, treatment-related adverse effects (TRAEs) were mostly grade 3-5 and consisted of peripheral neuropathy, dyspnea, hypertension, heart failure, and acute kidney injury. The TRAEs of interest were cardiac, pulmonary, and renal, as well as peripheral neuropathy.

One of the criticisms of this study is that it did not include patients with high-risk multiple myeloma who, in prior studies, showed a benefit with carfilzomib, Braunstein noted. Notably, approximately 27% of patients in each group went on to receive autologous stem cell transplant, which was not planned according to the design of the study.

Based on these data, the investigators concluded that VRd should remain the standard of care.

Isatuximab/KRd for Newly Diagnosed, High-Risk Multiple Myeloma

Isatuximab-irfc (Sarclisa) plus KRd can be safely administered in patients with high-risk multiple myeloma, inducing deep responses, according to results from the interim analysis of the GMMG-concept trial presented during the meeting; this was the first study to investigate the quadruplet regimen in this patient population.2

In the multicenter, open-label, phase 2 analysis, investigators evaluated the efficacy of isatuximab, an anti-CD38 monoclonal antibody, in combination with KRd in patients with high-risk, newly diagnosed multiple myeloma. Patients could have received up to 1 cycle of anti-myeloma treatment prior to inclusion. They had to show acceptable organ function in order to participate.

The primary end point of the trial was minimal residual disease (MRD) negativity by flow cytometry to a sensitivity of 10-5and the secondary end point was PFS. Tertiary end points included ORR, duration of MRD negativity, OS, and quality-of-life (QOL) assessment.

This was an investigator-initiated study that ultimately evaluated a quadruplet regimen, which is the trend that we are pursuing these days in terms of up-front therapy, noted Braunstein.

In the analysis, 153 patients were randomized 1:1 based on transplant eligibility. In arm A, 117 transplant-eligible patients received 6 cycles of isatuximab plus KRd induction, 4 cycles of isatuximab plus KRd consolidation, and isatuximab plus KR maintenance. In arm B, 36 transplant-ineligible patients received the same course of treatment with 2 additional cycles of isatuximab plus KRd induction.

The interim analysis reported on a total of 50 patients: 46 in arm A and 4 in arm B. Results showed an ORR of 100% with the quadruplet, a VGPR or greater of 90%, a CR/sCR of 46%. In arm A, 41 of 46 patients achieved a VGPR or greater, while all patients in arm B achieved a VGPR. MRD was assessed in a total of 33 patients in arm A during induction and results showed that 20 patients were MRD negative, 11 were MRD positive, and 2 were not evaluable.

With regard to safety, the most common hematologic treatment-emergent AEs (TEAEs) reported were grade 3 or 4 and consisted of leukopenia (26%), neutropenia (34%), lymphopenia (28%), anemia (10%), and thrombocytopenia (14%).

Any-grade nonhematologic TEAEs included upper-respiratory tract infections (18%), pyrexia (12%), rash (16%), peripheral sensory neuropathy (16%), nasopharyngitis (10%), hypertension (12%), cardiac failure (4%), and infusion reaction (32%). No deaths were reported on the study.

We now have doublet, triplet, and quadruplet up-front regimens for patients with newly diagnosed multiple myeloma, Braunstein said. The more agents you combine synergistically to target the plasma cell clones, the deeper response rates you can achieve and that consistently correlates with longer remissions.

Belantamab Mafodotin Triplet for Relapsed/Refractory Multiple Myeloma

Belantamab mafodotin in combination with bortezomib and dexamethasone (B-Vd) demonstratedan acceptable safety profile, according to preliminary findings from a cohort of the phase 1/2 DREAMM-6 trial also presented during the meeting.3

In the 2-part, open-label, phase 1/2 study, investigators evaluated the safety and efficacy of the addition of belantamab mafodotin to B-Vd, 2 standard-of-care doublet regimens, in patients with relapsed/refractory multiple myeloma who have previously received at least 1 line of therapy. To be eligible for the trial, patients had to have measurable disease, acceptable organ function, and an ECOG performance status of 0 to 2. Those who underwent previous autologous stem cell transplant and those who were not candidates for transplant were permitted, along with those who were refractory to bortezomib.

The primary end points of the trial were safety, tolerability, and ORR as defined by the International Myeloma Working Group Uniform Response Criteria. Key secondary end points included preliminary clinical activity, further safety/tolerability examination, pharmacokinetic assessments, and the health-related QOL impact of the combination.

In arm A, belantamab mafodotin was combined with lenalidomide and dexamethasone (B-Rd) and, in arm B, belantamab mafodotin was combined with B-Vd.Part 1 was a dose-escalation phase and part 2 is an ongoing dose-expansion phase for each of the arms with either single (day 1) or split dosing (day 1 and 8) for belantamab mafodotin at 2.5 mg/kg or 3.4 mg/kg.

Results on 18 patients who received the 2.5-mg/kg dose of B-Vd experienced an ORR of 78% (95% CI, 52.4%-93.6%). In addition, patients demonstrated a clinical benefit rate of 83% (95% CI, 58.6%-96.4%) and the VGPR was 50%. The duration of response with the combination had not yet been reached.

Moreover, 89% of patients experienced grade 3 or 4 AEs. Dose reductions were required in 72% of patients because of AEs and all patients experienced a dose interruption or delay due to keratopathy and/or thrombocytopenia. Notably, all toxicities were found to be clinically manageable.

I believe belantamab mafodotin offers a unique therapeutic approach, which is very much needed in [patients with] refractory multiple myeloma, Braunstein concluded.

Read the original here:
Braunstein Reflects on the Rise of Quadruplet Therapies in Multiple Myeloma - OncLive

Basel, 8 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced U.S. Food and Drug Administration (FDA) 510k clearance for the cobas BKV Test on the cobas 6800 and 8800 Systems. The test was previously granted FDA Breakthrough Device designation demonstrating the improved treatment or diagnosis of life-threatening diseases or conditions for transplant patients. The test provides standardised, high-quality results that can help healthcare professionals better assess the risk of complications caused by the BK virus in transplant patients and identify effective treatment options.

BK virus (BKV) is a member of the polyomavirus family that can cause severe transplant-associated complications. Infection can occur without symptoms and happen early in life. After primary infection, the virus can remain inactive, only to possibly reactivate in immunocompromised individuals such as transplant recipients.

Our diagnostic tests can help clinicians greatly improve patient treatment plans and make quick adjustments for personalised healthcare, said Thomas Schinecker, CEO Roche Diagnostics. This FDA clearance allows Roche to offer healthcare professionals a transplant testing portfolio that includes Cytomegalovirus, Epstein-Barr virus and BK virus so they can simultaneously monitor and improve care for transplant patients who are at risk for these common infections or viral reactivations which can cause further illness or death.

The cobas BKV Test is a polymerase chain reaction (PCR) viral load test that runs on the fully automated and widely available cobas 6800 and cobas 8800 Systems. Along with the previously approved cobas EBV and CMV Tests, the cobas BKV Test has been calibrated to the World Health Organization (WHO) International Standard. This means that test results are reported in international units, making it possible for laboratories anywhere in the U.S. to obtain comparable results when measuring levels of BKV DNA.

About the cobas BKV TestThe cobas BKV Test was previously granted Breakthrough Device Designation by the FDA, together with the cobas EBV Test.

The cobas BKV Test is a real-time polymerase chain reaction (PCR) test with dual-target technology that provides quantitative accuracy and guards against the risk of sequence variations that may be present in the BK virus. The cobas BKV Test has robust coverage with a limit of detection of 21.5 IU/mL and an expanded linear range from 21.5 IU/mL to 1E+08 IU/mL in EDTA plasma.

The test offers an alternative to lab-developed tests (LDTs) or Analyte Specific Reagent (ASR) combinations, potentially minimising variability and complexity in testing, reducing workload and alleviating risk for laboratories. The test supports the goal of result standardisation across institutions by providing reproducible, high-quality results for clinical decision-making.

The fully automated cobas BKV Test and the cobas CMV and cobas EBV Tests can run on the cobas 6800/8800 Systems simultaneously, providing absolute automation with proven performance and flexibility, leading to time savings and increased efficiency.

About BK polyomavirus BK polyomavirus (BKV) is a member of the polyomavirus family that can cause transplant-associated complications including nephropathy in kidney transplantation and hemorrhagic cystitis in hematopoietic stem cell transplantation. Infection can occur early in life, often with no symptoms. After primary infection, the virus can remain inactive throughout life, only to possibly reactivate in immunocompromised individuals, such as patients who receive solid-organ transplants. For kidney transplant patients, BKV infection is considered the most common viral complication, causing polyomavirus nephropathy (PVN) in up to 10 percent of kidney transplant recipients, and about 50 percent of PVN-affected patients will experience transplant graft failure.1 BKV is also associated with hemorrhagic cystitis after allogeneic hematopoietic stem cell transplantation.2

About the cobas 6800/8800 SystemsWhen every moment matters, the fully automated cobas 6800/8800 Systems offer the fastest time to results with the highest throughput and the longest walk-away time available among automated molecular platforms. The systems provide up to 96 results in about three hours and 384 results for the cobas 6800 System and 1,056 results for the cobas 8800 System in an eight hour shift.*

Both systems make it possible for labs to perform up to three tests in the same run with no pre-sorting required. The systems also enable up to eight hours (cobas 6800 System) and four hours (cobas 8800 System) of walk-away time with minimal user interaction.*

These real-time PCR systems serve the areas of infectious disease, donor screening, sexual health, transplant, respiratory and antimicrobial stewardship.

Through an ever-increasing worldwide install base of cobas 6800/8800 Systems, labs are quickly and easily processing millions of tests per month to meet the changing demands of their communities, their customers, and the patients relying on the results of each assay. Globally, labs know and trust that a Roche assay guarantees high precision, accuracy, and traceability to World Health Organization standards.

Today, rapid advancements in healthcare technology, a shortage of skilled workers, industry-wide consolidation, and the proven need to be ready for the next outbreak have health systems looking to lay a reliable foundation for the future. With proven performance, absolute automation, and unmatched flexibility delivering unparalleled throughput 24/7cobas 6800/8800 Systems are designed to ensure a labs long-term sustainability and success now, more than ever.

Learn more now: http://www.cobas68008800.com or http://diagnostics.roche.com.*May vary based on workflow demands

About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve peoples lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the worlds largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the eleventh consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References[1] Jamboti, J. S. (2016) BK virus nephropathy in renal transplant recipients. Nephrology, 21: 647 654. doi: 10.1111/nep.12728. [2] Hirsch HH, Randhawa PS; AST Infectious Diseases Community of Practice. BK polyomavirus in solid organ transplantation-Guidelines from the American Society of Transplantation Infectious Diseases Community of Practice. Clin Transplant. 2019;33(9):e13528. doi:10.1111/ctr.13528

Roche Group Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com

See more here:
Roche receives FDA clearance for BK virus quantitative test on cobas 6800/8800 Systems to support better care for transplant patients - GlobeNewswire

A persuasive Exosome Therapeutic Market report can be explored more specifically in terms of breakdown of data by manufacturers, region, type, application, market status, market share, growth rate, future trends, market drivers, opportunities, challenges, emerging trends, risks and entry barriers, sales channels & distributors. The data and information have been derived from the reliable sources and include websites, annual reports of the companies, journals, and mergers which have been again checked & validated by the market experts. The estimations of CAGR values are also provided in this wide ranging Exosome Therapeutic Market report which helps businesses in deciding upon the investment value over the time period.

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This Exosome Therapeutic Market report takes into account the conception of business goals and requirements to bridge the gap by delivering the most proper and suitable solutions. The competitive analysis is also performed in this report which covers strategic profiling of key market players, their core competencies & competitive landscape of the market which aids businesses characterize their individual strategies. In this market document, market drivers and market restraints are studied carefully along with the analysis of market structure. To provide an absolute overview of the Healthcare industry, Exosome Therapeutic Market analysis report covers various aspects of market analysis, product definition, market segmentation, key developments & the existing vendor landscape.

Market Analysis and Insights:Global Exosome Therapeutic Market

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

The major players covered in theExosome Therapeutic Marketreport areevox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global.Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Get Full TOC, Tables and Figures of Market Report @https://www.databridgemarketresearch.com/toc/?dbmr=global-exosome-therapeutic-market&rp

Exosomes are used to transfer RNA, DNA, and proteins to other cells in the body by making alteration in the function of the target cells. Increasing research activities in exosome therapeutic is augmenting the market growth as demand for exosome therapeutic has increased among healthcare professionals.

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for anAnalyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

Global Exosome Therapeutic Market Scope and Market Size

Global exosome therapeutic market is segmented of the basis of type, source, therapy, transporting capacity, application, route of administration and end user. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

Based on type, the market is segmented into natural exosomes and hybrid exosomes. Natural exosomes are dominating in the market because natural exosomes are used in various biological and pathological processes as well as natural exosomes has many advantages such as good biocompatibility and reduced clearance rate compare than hybrid exosomes.

Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process. The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

Rising demand of exosome therapeutic across the globe as exosome therapeutic is expected to be one of the most prominent therapies for autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases treatment, according to clinical researches exosomes help to processes regulation within the body during treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases. This factor has increased the research activities in exosome therapeutic development around the world for exosome therapeutic. Hence, this factor is leading the clinician and researches to shift towards exosome therapeutic. In the current scenario the exosome therapeutic are highly used in treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases and as anti-aging therapy as it Exosomes has proliferation of fibroblast cells which is significant in maintenance of skin elasticity and strength.

Based on source, the market is segmented into dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, urine and others. Mesenchymal stem cells are dominating in the market because mesenchymal stem cells (MSCs) are self-renewable, multipotent, easily manageable and customarily stretchy in vitro with exceptional genomic stability. Mesenchymal stem cells have a high capacity for genetic manipulation in vitro and also have good potential to produce. It is widely used in treatment of inflammatory and degenerative disease offspring cells encompassing the transgene after transplantation.

Based on therapy, the market is segmented into immunotherapy, gene therapy and chemotherapy. Chemotherapy is dominating in the market because chemotherapy is basically used in treatment of cancer which is major public health issues. The multidrug resistance (MDR) proteins and various tumors associated exosomes such as miRNA and IncRNA are include in in chemotherapy associated resistance.

Based on transporting capacity, the market is segmented into bio macromolecules and small molecules. Bio macromolecules are dominating in the market because bio macromolecules transmit particular biomolecular information and are basically investigated for their delicate properties such as biomarker source and delivery system.

Based on application, the market is segmented into oncology, neurology, metabolic disorders, cardiac disorders, blood disorders, inflammatory disorders, gynecology disorders, organ transplantation and others. Oncology segment is dominating in the market due to rising incidence of various cancers such as lung cancer, breast cancer, leukemia, skin cancer, lymphoma. As per the National Cancer Institute, in 2018 around 1,735,350 new cases of cancer was diagnosed in the U.S. As per the American Cancer Society Inc in 2019 approximately 268,600 new cases of breast cancer diagnosed in the U.S.

Based on route of administration, the market is segmented into oral and parenteral. Parenteral route is dominating in the market because it provides low drug concentration, free from first fast metabolism, low toxicity as compared to oral route as well as it is suitable in unconscious patients, complicated to swallow drug etc.

The exosome therapeutic market, by end user, is segmented into hospitals, diagnostic centers and research & academic institutes. Hospitals are dominating in the market because hospitals provide better treatment facilities and skilled staff as well as treatment available at affordable cost in government hospitals.

Exosome therapeutic Market Country Level Analysis

The global exosome therapeutic market is analysed and market size information is provided by country by type, source, therapy, transporting capacity, application, route of administration and end user as referenced above.

The countries covered in the exosome therapeutic market report are U.S. and Mexico in North America, Turkey in Europe, South Korea, Australia, Hong Kong in the Asia-Pacific, Argentina, Colombia, Peru, Chile, Ecuador, Venezuela, Panama, Dominican Republic, El Salvador, Paraguay, Costa Rica, Puerto Rico, Nicaragua, Uruguay as part of Latin America.

Country Level Analysis, By Type

North America dominates the exosome therapeutic market as the U.S. is leader in exosome therapeutic manufacturing as well as research activities required for exosome therapeutics. At present time Stem Cells Group holding shares around 60.00%. In addition global exosomes therapeutics manufacturers like EXOCOBIO, evox THERAPEUTICS and others are intensifying their efforts in China. The Europe region is expected to grow with the highest growth rate in the forecast period of 2019 to 2026 because of increasing research activities in exosome therapeutic by population.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Huge Investment by Automakers for Exosome Therapeutics and New Technology Penetration

Global exosome therapeutic market also provides you with detailed market analysis for every country growth in pharma industry with exosome therapeutic sales, impact of technological development in exosome therapeutic and changes in regulatory scenarios with their support for the exosome therapeutic market. The data is available for historic period 2010 to 2017.

Competitive Landscape and Exosome Therapeutic Market Share Analysis

Global exosome therapeutic market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, concept cars, product approvals, patents, product width and breadth, application dominance, technology lifeline curve. The above data points provided are only related to the companys focus related to global exosome therapeutic market.

Many joint ventures and developments are also initiated by the companies worldwide which are also accelerating the global exosome therapeutic market.

For instance,

Partnership, joint ventures and other strategies enhances the company market share with increased coverage and presence. It also provides the benefit for organisation to improve their offering for exosome therapeutics through expanded model range.

Customization Available:Global Exosome Therapeutic Market

Data Bridge Market Researchis a leader in advanced formative research. We take pride in servicing our existing and new customers with data and analysis that match and suits their goal. The report can be customised to include price trend analysis of target brands understanding the market for additional countries (ask for the list of countries), clinical trial results data, literature review, refurbished market and product base analysis. Market analysis of target competitors can be analysed from technology-based analysis to market portfolio strategies. We can add as many competitors that you require data about in the format and data style you are looking for. Our team of analysts can also provide you data in crude raw excel files pivot tables (Factbook) or can assist you in creating presentations from the data sets available in the report.

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Continued here:
Exosome Therapeutic Market : SWOT Analysis of Top Key Player & Forecasts || Major Gaints Jazz Pharmaceuticals, Inc., Boehringer Ingelheim...

DBMR has added a new report titledExosome Therapeutic Marketwith data Tables for historical and forecast years represented with Chats & Graphs spread through Pages with easy to understand detailed analysis. This Exosome Therapeutic Market report is quite useful to find out the general market conditions and tendencies. It also estimates the probable market for a new product to be launched in the market. This also Report has compiled to provide various market aspects such as size, share, trends, dynamics, growth, sales, and industry analysis. The competitive analysis taken place in this Exosome Therapeutic Market report include strategic profiling of key market players, their core competencies, their strong and weak points, and competitive landscape of the market which supports businesses illustrate their individual strategies.

The Exosome Therapeutic Market 2020 research provides a basic overview of the industry including definitions, classifications, applications and industry chain structure. The Exosome Therapeutic Market Share analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed.

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

Get Sample Report + All Related Graphs & Charts (with COVID 19 Analysis) @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-exosome-therapeutic-market&pm

Exosomes are used to transfer RNA, DNA, and proteins to other cells in the body by making alteration in the function of the target cells. Increasing research activities in exosome therapeutic is augmenting the market growth as demand for exosome therapeutic has increased among healthcare professionals.

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for anAnalyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

Grab Your Report at an Impressive 30% Discount! Please click Here @https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-exosome-therapeutic-market&pm

The major players covered in the report are evox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global. Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Competitive Landscape and Exosome Therapeutic Market Share Analysis

Global exosome therapeutic market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, concept cars, product approvals, patents, product width and breadth, application dominance, technology lifeline curve. The above data points provided are only related to the companys focus related to global exosome therapeutic market.

Many joint ventures and developments are also initiated by the companies worldwide which are also accelerating the global exosome therapeutic market.

For instance,

Partnership, joint ventures and other strategies enhances the company market share with increased coverage and presence. It also provides the benefit for organisation to improve their offering for exosome therapeutics through expanded model range.

Global Exosome Therapeutic Market Scope and Market Size

Global exosome therapeutic market is segmented of the basis of type, source, therapy, transporting capacity, application, route of administration and end user. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

Based on type, the market is segmented into natural exosomes and hybrid exosomes. Natural exosomes are dominating in the market because natural exosomes are used in various biological and pathological processes as well as natural exosomes has many advantages such as good biocompatibility and reduced clearance rate compare than hybrid exosomes.

Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process. The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

Rising demand of exosome therapeutic across the globe as exosome therapeutic is expected to be one of the most prominent therapies for autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases treatment, according to clinical researches exosomes help to processes regulation within the body during treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases. This factor has increased the research activities in exosome therapeutic development around the world for exosome therapeutic. Hence, this factor is leading the clinician and researches to shift towards exosome therapeutic. In the current scenario the exosome therapeutic are highly used in treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases and as anti-aging therapy as it Exosomes has proliferation of fibroblast cells which is significant in maintenance of skin elasticity and strength.

Table of Contents:

For More Insights Get FREE Detailed TOC @https://www.databridgemarketresearch.com/toc/?dbmr=global-exosome-therapeutic-market&pm

Exosome therapeutic Market Country Level Analysis

The global exosome therapeutic market is analysed and market size information is provided by country by type, source, therapy, transporting capacity, application, route of administration and end user as referenced above.

The countries covered in the exosome therapeutic market report are U.S. and Mexico in North America, Turkey in Europe, South Korea, Australia, Hong Kong in the Asia-Pacific, Argentina, Colombia, Peru, Chile, Ecuador, Venezuela, Panama, Dominican Republic, El Salvador, Paraguay, Costa Rica, Puerto Rico, Nicaragua, Uruguay as part of Latin America.

Country Level Analysis, By Type

North America dominates the exosome therapeutic market as the U.S. is leader in exosome therapeutic manufacturing as well as research activities required for exosome therapeutics. At present time Stem Cells Group holding shares around 60.00%. In addition global exosomes therapeutics manufacturers like EXOCOBIO, evox THERAPEUTICS and others are intensifying their efforts in China. The Europe region is expected to grow with the highest growth rate in the forecast period of 2019 to 2026 because of increasing research activities in exosome therapeutic by population.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Huge Investment by Automakers for Exosome Therapeutics and New Technology Penetration

Global exosome therapeutic market also provides you with detailed market analysis for every country growth in pharma industry with exosome therapeutic sales, impact of technological development in exosome therapeutic and changes in regulatory scenarios with their support for the exosome therapeutic market. The data is available for historic period 2010 to 2017.

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Exosome Therapeutic Market 2020-2026 is Growing So Rapidly || Leading Players evox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United...

Roots Analysis has done a detailed study on Gene Therapy Market (3rd Edition), 2019-2030, covering key aspects of the industrys evolution and identifying potential future growth opportunities.

Key Market Insights

For more information, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

Table of Contents

4.2.1 Types of Viral Vectors

11.2 Analysis by Special Designations Awarded

11.3.1. Analysis by Target Indication

11.3.2. Analysis by Type of Vector Used

11.4.1. Analysis by Target Indication

11.4.2. Analysis by Type of Vector Used

11.5.1. Analysis by Target Indication

11.5.2. Analysis by Type of Vector Used

11.6.1. Analysis by Target Indication

11.6.2. Analysis by Type of Vector Used

11.7.1. Analysis by Target Indication

11.7.2. Analysis by Type of Vector Used

11.8.1. Analysis by Target Indication

11.8.2. Analysis by Type of Vector Used

11.9.1. Analysis by Target Indication

11.9.2. Analysis by Type of Vector Used

11.10.1. Analysis by Target Indication

11.10.2. Analysis by Type of Vector Used

11.11.1. Analysis by Target Indication

11.11.2. Analysis by Type of Vector Used

11.12.1. Analysis by Target Indication

11.12.2. Analysis by Type of Vector Used

11.13.1. Analysis by Target Indication

11.13.2. Analysis by Type of Vector Used

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https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

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Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

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The Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 - Scientect