StemCell Therapy

Playing through the greenery and litter of a mini forest's undergrowth for just one month may be enough to change a child's immune system, according to a small new experiment.

When daycare workers in Finland rolled out a lawn, planted forest undergrowth such as dwarf heather and blueberries, and allowed children to care for crops in planter boxes, the diversity of microbes in the guts and on the skin of young kids appeared healthier in a very short space of time.

Compared to other city kids who play in standard urban daycares with yards of pavement, tile and gravel, 3-, 4-, and 5-year-olds at these greened-up daycare centres in Finland showed increased T-cells and other important immune markers in their blood within 28 days.

"We also found that the intestinal microbiota of children who received greenery was similar to the intestinal microbiota of children visiting the forest every day," saysenvironmental scientist Marja Roslund from the University of Helsinki.

One daycare before (left) and after introducing grass and planters (right). (University of Helsinki)

Prior research has shown early exposure to green space is somehow linked to a well-functioning immune system, but it's still not clear whether that relationship is causal or not.

The experiment in Finland is the first to explicitly manipulate a child's urban environment and then test for changes in their micriobiome and, in turn, a child's immune system.

While the findings don't hold all the answers, they do support a leading idea - namely that a change in environmental microbes can relatively easily affect a well-established microbiome in children, giving their immune system a helping hand in the process.

The notion that an environment rich in living things impacts on our immunity is known as the 'biodiversity hypothesis'. Based on that hypothesis,a loss of biodiversity in urban areascould be at least partially responsible for the recent rise in immune-related illnesses.

"The results of this study support the biodiversity hypothesis and the concept that low biodiversity in the modern living environment may lead to an un-educated immune system and consequently increase the prevalence of immune-mediated diseases," the authors write.

The study compared the environmental microbes found in the yards of 10 different urban daycares looking after a total of 75 kids between the ages of 3 and 5.

Some of these daycares contained standard urban yards with concrete and gravel, others took kids out for daily nature time, and four had their yards updated with grass and forest undergrowth.

Over the proceeding 28 days, kids in these last four daycares were given time to play in their new backyard five times a week.

When researchers tested the microbiota of their skin and gut before and after the trial, they found improved results compared to the first group of kids that played in daycares with less greenery for the same amount of time.

Even in that short duration of the study, researchers found microbes on the skin and guts of children who regularly played in green spaces had increased in diversity - a feature which is tied to an overall healthier immune system.

Their results largely matched the second group of kids at daycares who had outings for daily nature time.

Among kids who got outside, playing in the dirt, the grass and among the trees, an increase in a microbe called gammaproteobacteria appeared to boost the skin's immune defence, as well as increase helpful immune secretions in the blood and reduce the content of interleukin-17A, which is connected to immune-transmitted diseases.

"This supports the assumption that contact with nature prevents disorders in the immune system, such as autoimmune diseases and allergies," says Sinkkonen.

The results aren't conclusive and they will need to be verified among larger studies around the world. Still, the benefits of green spaces appear to go beyond our immune systems.

Researchshows getting outside is also good for a child's eyesight, and being in nature as a kid is linked to better mental health. Some recent studies have even shown green spaces are linked to structural changes in the brains of children.

What's driving these incredible results is not yet clear. It could be linked to changes to the immune system, or something about breathing healthy air, soaking in the sun, exercising more or having greater peace of mind.

Given the complexities of the real world, it's really hard to control for all the environmental factors that impact our health in studies.

While rural children tend to have fewer cases of asthma and allergies, the available literature on the link between green spaces and these immune disorders is inconsistent.

The current research has a small sample size, only found a correlation, and can't account for what children were doing outside daycare hours, but the positive changes seen are enough for scientists in Finland to offer some advice.

"It would be best if children could play in puddles and everyone could dig organic soil," encouragesenvironmental ecologist Aki Sinkkonen, also from the University of Helsinki.

"We could take our children out to nature five times a week to have an impact on microbes."

The changes are simple, the harms low, and the potential benefits widespread.

Bonding with nature as a kid is also good for the future of our planet's ecosystems.Studiesshow kids who spend time outdoors are more likely to want to become environmentalists as adults, and in a rapidly changing world, that's more important than ever.

Just make sure everyone's up to date on their tetanus vaccinations, Sinkkonen advises.

The study was published in the Science Advances.

Continue reading here:
Daycares in Finland Built a 'Forest Floor', And It Changed Children's Immune Systems - ScienceAlert

Across the world, immunologists who retooled their labs to join the fight against SARS-CoV-2 are furiously trying to explain why some people get so sick while others recover unscathed. The pace is dizzying, but some clear trends have emerged.

One area of focus has been the production of antibodies powerful proteins capable of disabling and killing invading pathogens like viruses. Of great concern has been the sporadic identification of so-called autoreactive antibodies that, instead of targeting disease causing microbes, target the tissues of individuals suffering from severe cases of COVID-19.

Early studies implicated these autoantibodies in dangerous blood clots forming in patients admitted to intensive care. More recently, they have been linked to severe disease by inactivating critical components of viral immune defenses in a significant fraction of patients with severe disease.

As an immunologist within the Lowance Center for Human Immunology at Emory University, I have been investigating the immune response responsible for producing antibodies in COVID-19. Under the direction of Dr. Ignacio Sanz, our group has previously investigated immune responses contributing to autoantibody production in autoimmune disorders like lupus, and more recently in severe cases in COVID-19. However, while we were able to characterize the response in COVID-19 patients as autoimmunelike, we could not confirm the production of autoantibodies hidden within their antiviral responses.

Now we can.

In a newly released study awaiting peer-review, we describe the alarming finding that in the sickest patients with COVID-19, autoantibody production is common a finding with large potential impact on both acute patient care and infection recovery.

Autoantibodies come in flavors that are usually associated with specific disease types. Patients with lupus, for example, will often have antibodies that target their own DNA the molecules that make up the human genome.

Patients with the autoimmune disorder rheumatoid arthritis are less likely to have those antibodies, but more likely to show positive tests for rheumatoid factor antibodies that target other antibodies.

In this study, the Lowance Center group analyzed the medical charts of 52 patients in intensive care who were diagnosed with COVID-19. None of them had a history of autoimmune disorders. However, they were tested during infection for autoantibodies found in a variety of disorders.

The results are stark. More than half of the 52 patients tested positive for autoantibodies. In patients with the highest levels of c-reactive protein (a marker of inflammation) in the blood, more than two-thirds displayed evidence that their immune system was producing antibodies attacking their own tissue.

While these findings raise concerns, there are things that our data dont reveal. Although patients with severe disease clearly display autoantibody responses, the data dont tell us to what extent these autoantibodies contribute to the most severe symptoms of COVID-19.

It could be that severe viral illness routinely results in the production of autoantibodies with little consequence; this could just be the first time were seeing it. We also dont know how long the autoantibodies last. Our data suggest that they are relatively stable over a few weeks. But, we need follow-up studies to understand if they are persisting routinely beyond infection recovery.

Importantly, we believe that the autoreactive responses we have identified here are specific to the SARS-CoV-2 infection there is no reason to believe that similar results would be expected through vaccination against the virus.

However, while it is possible that these autoantibodies are benign, or even helpful in a yet-unidentified manner, its also possible that they arent. Maybe these self-targeted antibody responses do indeed contribute to disease severity, helping explain the delayed onset of severe symptoms in some patients that may correlate with antibody production.

This could be a reason that treatment with dexamethasone, an immunosuppressant often used to quell flare-ups of autoimmune disorders, might be effective in treating patients with only the most severe disease. It is also possible that these responses are not short lived, outlasting the infection and contributing to ongoing symptoms now experienced by a growing number of long-hauler COVID-19 patients.

Most concerning, it is possible that these responses could self-perpetuate in some patients, resulting in the emergence of new, permanent autoimmune disorders.

My colleagues and I sincerely hope that this is not the case rather, that the emergence of autoantibodies in these patients is a red herring, a quirk of a viral immune response in some patients that will resolve on its own. But we need to do better than hope we need to ask the right questions and figure out the answers. Fortunately, this study also gives us the tools to do that.

The tests that were run on these patients to determine their autoreactive profile are not specialized. They are available to most hospital labs across the country. Indeed, the two most common antibodies that we find in these patients, antinuclear antibodies and rheumatoid factor, are detected by common tests used by rheumatologists.

Our study shows that by testing for just these two autoantibodies, and the inflammatory marker c-reactive protein, we may be able to identify patients more likely to be experiencing potentially dangerous immune responses that might benefit from more aggressive immune modulation.

[Get facts about coronavirus and the latest research. Sign up for The Conversations newsletter.]

Further, autoreactivity testing might help identify patients who might benefit from rheumotological follow-up to monitor recovery, and help us understand whether some cases of long-hauler COVID-19 might be related to persisting autoantibodies. If so, these patients might respond to the same immune-targeted therapies that have been successful in MIS-C where autoantibody production has now been documented.

Finally, by testing patients immediately following COVID-19 recovery, we can establish baselines and begin to track the possible emergence of new cases of autoimmunity following this terrible disease, and plan early rheumatological intervention if needed.

We now have the tools. Its time to start using them.

Original post:
COVID-19 causes some patients' immune systems to attack their own bodies, which may contribute to severe illness - The Conversation US

Winter is coming and a second wave of Covid-19 is already with us, so it has never been more important to strengthen your immunity.

Along with hand-washing, masks and social distancing, its the best defence we have against the coronavirus.

A vaccine is still some way off and scientists warn that without one there is no hope of achieving the herd immunity needed to end the pandemic.

Boosting your own immunity makes sense, and studies show there is lots you can do.

A balanced diet is key, but there are certain nutrients that are particularly important and scientists have been working hard to find the stars that can help in the Covid battle.

Take these simple steps to strengthen your immune system and keep you well through the tough winter ahead:

Vitamin D, which is essential for healthy bones and muscles, is often known as the sunshine vitamin giving a big clue as to why we dont get enough in winter.

While it can be found in some foods, including oily fish, red meat and eggs, our bodies grab the majority of what they need from exposure to sunlight.

One in five Brits is deficient, with the number increasing as the nights draw in.

And thats why the official advice is to consider taking a supplement containing 10mcg of vitamin D (the equivalent of 400IU) during winter.

But as well as keeping bones strong, this simple vitamin could also play a key role in the fight against Covid-19.

A super-study published earlier this year, which analysed results from 39 trials, found that taking 10mcg of the sunshine vitamin reduced the risk of respiratory infections by up to 23 per cent.

More recently, researchers at the University of Birmingham, who tested blood samples from 392 healthcare staff, found that 72per cent of those who were deficient in vitamin D had also caught Covid-19.

By contrast, only 51 per cent of those with good levels carried antibodies confirming they had been infected.

Covidence-UK, a study that uses monthly surveys of UK citizens to identify potential risk factors for Covid-19, has also identified a link.

Researchers are launching a randomised control trial to see if providing free vitamin D to people with low levels reduces their odds of catching the virus. To sign up and support the Covidence-UK search for ways to reduce the risk of Covid-19, visit qmul.ac.uk/covidence.

Some scientists believe its no accident that countries where cabbage and fermented vegetables, such as sauerkraut and kimchi, are popular, have reported lower coronavirus death rates.

Dr Jean Bousquet, Honorary Professor of Pulmonary Medicine at Montpellier University, France, has calculated that every gram of fermented vegetables eaten each day reduces the risk of dying of Covid-19 by 35 per cent.

A similar pattern was seen in countries where a lot of cabbage is consumed, and Professor Bousquet believes the benefits are down to high levels of the antioxidant sulforaphane in them.

He adds: Nutrition may play a role in the immune defence against Covid and may explain some of the differences seen in Covid across Europe. Ive now changed my diet, and it includes raw cabbage three times a week, sauerkraut once a week and pickled vegetables.

Dozens of studies have underlined the importance of zinc for strong immunity. And the World Health Organisation confirms it, saying: Zinc is thought to help decrease susceptibility to acute lower respiratory tract infections by regulating various immune functions.

Zinc can be found in shellfish, beans and lentils, but eight per cent of adults here in the UK do not get the recommended intake.

Research has shown that taking zinc within 24 hours of cold symptoms appearing cuts the duration of infections by a third.

Excitingly, studies have confirmed zinc inhibits Covid-19 and there is evidence it may boost levels of interferon, a protein that helps our immune system identify threats.

The balance of good and bad bacteria in our gut has a big impact on immunity. Dr Bousquets research has also identified a link between fermented dairy products, such as yoghurt and kefir, and a lower rate of deaths from the virus.

A recent Chinese study discovered that probiotic drinks that contained a combination of lactobacillus bacteria reduced the risk of respiratory infections by 59 per cent.

Another study, published in the European Journal of Public Health, found that children who were given daily probiotic supplements werearound a third less likely to needantibiotics.

A 150ml glass of orange juice provides more than 80 per cent of the immune-strengthening vitamin C that we need each day.

But there is growing scientific interest around another nutrient in orange juice, which could be even more important for our immunity.

Hesperidin, a micronutrient found in citrus fruit, slows the rate that viruses can replicate. Laboratory tests showed such strong antiviral activity that some scientists think it could be used to develop new drugs against dreaded influenza.

A recent study showed it locks on to key proteins on the Covid-19 virus, which could make it harder for infection to take hold.

Because the highest concentrations are found in the pith of fruit, juice will have more hesperidin in it than whole fruit. Shop-bought orange juice contains three times more hesperidin than juice squeezed at home.

Read the original post:
Strengthen your immune system against Covid with help of Vitamin D, zinc and pickles - Mirror Online

Immune boosting is a phrase that I really cant get along with, says leading nutritional therapist and healthy eating expert, Amelia Freer, when asked about the best immune-boosting advice for the coming winter months. In fact, according to Freer, weve been approaching it all wrong and when it comes to our immune system, the aim isnt to boost, but rather to balance it.

Read more: Feeling Low? Here Are 8 Ways To Ease Seasonal Affective Disorder

An overactive immune system can result in auto-immune disease, or a significant widespread inflammatory state, while an under-active or otherwise compromised immune system can increase our risk of infection neither of which is ideal. In the simplest of terms, says Freer, we want to be able to switch our immune function on appropriately, and then switch it off again when the infection risk has passed.

As for how we can do this best, Freer suggests nurturing and supporting our overall health and wellbeing. There are various nutrients that our body requires to mount and suppress an appropriate immune response, she comments. The best way to get these is through eating a balanced, nutritious, and abundant diet, so including a wide variety of different whole foods into our diets throughout the winter is a great place to start.

A variety of fresh green vegetables is key.

SCIENCE PHOTO LIBRARY

First up is dark-green vegetables such as kale, chard, spinach, rocket, Brussels sprouts, sprouting broccoli. They all provide a variety of beneficial phytonutrients, fibre, vitamin A, magnesium, folate and more. If there is one thing to add to our diets, it is this group of vegetables. Aim for at least one portion per day (remembering that when cooked, they tend to shrink considerably in terms of volume, making it easier to achieve this target).

Citrus fruits are a good source of vitamin C and perfect for the coldest winter months a little bit of concentrated sunshine just when we need it most. I particularly love the month or two that blood oranges are available [around December to April] I have one almost every day when I can, as the most deliciously simple dessert.

Citrus fruits such as oranges, grapefruits, and mandarins can provide, a little bit of concentrated sunshine just when we need it most, according to Freer.

voloshin311

This is a cheap and readily available oily fish and a source of long-chain omega-3 fatty acids, which can help to regulate inflammation in the body. It also contains some food-based vitamin D, as well as protein, and its a great speedy choice for lunches. Top tip: look out for unsmoked, frozen mackerel fillets in the freezer section of some supermarkets. Ive found that its the best fish to cook from frozen, and contains less salt than the smoked version.

There are a huge variety of orange-fleshed pumpkins to enjoy over winter. They provide a source of vitamin A, which, as a fat-soluble vitamin, is best absorbed alongside some healthy fats. I therefore tend to slow-roast my squash and pumpkins in a little olive oil, and then enjoy as they come, blended into sauces or soups, or tossed into salads with rocket, radicchio, some toasted hazelnuts and crumbled feta.

Brazil nuts are a key source of the micronutrient selenium, which is an important mineral for optimal immune response. Just four or five Brazil nuts per week can meet our selenium requirements. It is, however, one of the few whole food nutrients that we can over-consume, so its best to mix things up and eat just a few each week alongside a variety of other nuts and seeds, too.

Protein and pulses are important for enabling the body to mount an appropriate immune response.

Adl Bkefi

I adore pulses in all shapes and sizes, and I find them a convenient source of protein I aim to have roughly a palm-sized portion of protein at each meal of the day. Protein is important for enabling the body to mount an appropriate immune response, as well as for repair and growth of our bodys tissues, and for appetite and blood-sugar regulation. I buy pulses in bulk in jars and add them to soups, make them into hummus and other dips, throw them into curries and stews, or eat them cold with some olive oil, lemon and a few chopped herbs.

Shellfish is a good source of zinc and vitamin B12, and mussels and scallops are in season over the winter months. They are a bit of a treat, but its worth making the effort to cook them once in a while. Do check that they are sustainably sourced and if youre unsure about cooking them yourself, it might be a good option to consider ordering if eating out.

Rolled oats are a great choice and can provide not only a warming and delicious porridge breakfast, but also a hefty dose of fibre, too. Soaking oats overnight can help to make the nutrients they contain more absorbable, as well as speeding up the cooking time.

Eating eggs regularly is a simple way to introduce immunity balancing benefits into your diet.

Jody Louie took this picture

Eggs are such a versatile and useful ingredient to have on hand for quick meals and speedy snacks. They are also a source of vitamin B12, a little vitamin D, vitamin A, protein and some are even fortified with omega-3 fatty acids, all of which are important for balanced immune function.

Maintaining a good level of hydration can help to keep our mucosal barriers moist, such as those in our mouth and the lining of our nose. This might sound strange, but hydration of these tissues helps to support the natural immune function that exists within them, warding off infection before it has a chance to enter the body. Plus water wont spoil your appetite for the abundance of nourishing whole foods awaiting you at your next meal.

Good hydration supports muscles and skin tissues, enabling them to better fight off infection.

Dulin

The only supplement that is recommended for everyone to consider over the winter months is vitamin D. In some countries, the sunlight is not strong enough between October and early March for our skin to make enough vitamin D to meet our requirements.

I know its been said a thousand times before, but it really is what works: wash your hands, prioritise sleep, actively respond to and manage stress, move regularly, exercise, moderate alcohol and avoid smoking. Its not original, but it is effective.

More from British Vogue:

See original here:
Immunity Boosting Is Over, Its All About Balance - British Vogue

Findings from a new study showed an association between high serum immunoglobulin G (IgG) and immunoglobulin A (IgA) levels and milder cases of Clostridium difficile (C. difficile).

Specifically, this association was modulated by the two serum antibodies targeting of both toxins A (TcdA) and B (TcdB), which play an essential role in the pathogenesis of the disease.

Investigators from Tel Aviv University and Tel Aviv Sourasky Medican Center conducted a case-control study to determine the risk factors of C. difficile infection (CDI) and evaluate the link between humoral immune response and CDI severity.

Thus, their analysis consisted of a total of 50 patients with CDI, 62% of whom were female. This represented a subset of 140 total CDI patients who were enrolled in their study.

They categorized CDI patients according to severitysevere disease was defined as leukocytosis with a white blood cell count of 15,000 cells/L, decreased blood albumin (<30 g/L) or a rise in serum creatinine level 1.5 times the premorbid level. Any patient who did not fulfill any of these requirements were considered to have a mild case.

Additionally, they analyzed 52 patient controls who were not suffering from diarrhea, where 56% were female. In total, they had enrolled 140 controls.

Both CDI patients and controls were matched by age, sex, hospitalization ward (medical or surgical), and number of hospitalization stays.

The mean age between the CDI and control groups were 79.2% years and 82.7%, respectively.

The investigators collected stool specimens from the both groups to test for C. difficile. Blood samples were also collected, and the levels of serum IgG and IgA antibodies against TcdA and TcDB were measured.

Overall, they found that patients with CDI presented with higher geometric mean titers (GMT) values of serum IgG antibody against TcdA when compared with the control group (20.1 EU vs 11.6 EU, respectively; P = .0001).

The GMT values of serum IgG against TcdB were also higher for the CDI group than for the control (18.0 EU vs 12.0 EU, respectively; P = .04).

They also noted that similar trends were observed for IgA antibodies, but the differences were not statistically significant.

In terms of associations for C. diff severity, they found that GMT values of serum IgA against TcdB was significantly higher among CDI patients with mild disease as compared with patients with severe disease (9.2 EU vs 4.9 EU, respectively; P = .023).

Similar but non-statistically significant trends were noted found for IgA and IgG levels against TcdA, as well as for IgG against TcdB.

Limiting the analysis to sera that were collected at days 714 following the diagnosis of C. difficile showed significantly higher IgG levels against TcdA and TcdB in patients with mild CDI compared to patients with severe CDI, they wrote.

Furthermore, they found significant correlations between serum IgG levels and TcdA and TcdB (Spearmans r = 0.31). Other strong correlations included IgA levels against TcdA and TcDB (r = 0.53) and IgG and IgA levels against TcdB (r = 0.43).

Although there remains great uncertainty behind the mechanism that can explain the protective effect of serum IgA and IgG antibodies against C. diff toxins, the investigators nonetheless suggested implications for the findings.

Based on the current evidence from observational studies and our new findings, the concept of presenting antigens that can prime or boost the immune system towards the production of antitoxin circulating antibodies seems a sensible approach for developing preventive and therapeutic vaccines and technologies for CDI, the team wrote.

The study, "Enhanced Humoral Immune Responses against Toxin A and B of Clostridium difficile is Associated with a Milder Disease Manifestation," was publshed online in Journal of Clinical Medicine.

Read more here:
Increased Humoral Immune Response Against C. Diff Toxins Linked to Mild Disease - MD Magazine

We are still working to figure out why low levels of vitamin D are associated with worse outcomes in critical illness, noted Todd W. Rice, MD, FCCP, associate professor of medicine at Vanderbilt University Medical Center in Nashville, Tennessee.

Studies have shown that low levels of vitamin D are associated with worse outcomes in critical illnessfor instance, needing to be in the intensive care unit (ICU) morebut we are still working to figure out why, noted Todd W. Rice, MD, FCCP, associate professor of medicine at Vanderbilt University Medical Center in Nashville, Tennessee, in an interview for this years CHEST Annual Meeting.

Transcript

Tell us about your presentation on day 1 of CHEST, Vitamin D in Critical Illness: Helpful Fact or Hopeless Fiction.

This is a presentation in a session about vitamins and vitamins in the ICU. And obviously, based off of the title, Im going to talk about vitamin D. The short, brief story of vitamin D is that weve had a number of studies that have shown that low levels of vitamin D are associated with worse outcomes, including needing to be into the ICU more, having more infections in the ICU, and staying in the ICU longer, having higher mortality while youre in the ICU. And so the thought process is that repleting peoples vitamin D to normal levels may prevent some of these bad outcomes. And weve tried this in a number of trials and we havent had great success in improving outcomes by giving patients vitamin D. And the question then becomes, why is that the case? Is it that low vitamin D level is not the reason that people do worse, its just kind of associated with being sick and doing worse? Or is there something about vitamin D metabolism and vitamin D absorption that we dont yet understand? So were not really supplementing it right, were not really targeting the right levels, those sorts of things. And I think were still working to figure that out.

How do the pleiotropic effects of micronutrients affect critical illness?

Vitamin D has a number of kind of positive effects in the body. Obviously, its involved in bone metabolismthats probably not that relevant in critical illnessbut its very, very, very much involved in the immune system. And its sort of a cofactor for our immune cells in fighting infection. And its been shown in a number of other studies, not in the ICU, that low vitamin D levels result in less robust immune systems, more prone to get infections, and worse outcomes.

The other effects of it are that it seems to have some anti-inflammatory effects. We dont entirely understand exactly how it decreases inflammation, but it seems to decrease inflammation and also kind of has some effects on the endothelial lining of the lungs and the vasculature. So all of those effects kind of together are pleiotropic in the fact that they sort of grow these areas, stimulate these areas, and are beneficial to the body in that regard.

Read more:
Dr Todd Rice Explains the Anti-inflammatory Benefits of Vitamin D to Our Immune Systems - AJMC.com Managed Markets Network

People wear face masks in Times Square as the city continues the re-opening efforts following restrictions imposed to slow the spread of coronavirus on October 22, 2020 in New York City.

Noam Galai | Getty Images

Mandy Elmore, 47, has been wearing masks for more than 20 years. That's because she has cystic fibrosis, a hereditary disease that affects her lungs and digestive system.

Because of her illness, a cold or flu can land her in the hospital. Prior to the pandemic, Elmore, who lives in Dallas, Texas, had to stop going to church or traveling on planes to avoid strangers coughing and sneezing directly on her.

"Masks offer freedom for those of us who are sick," she said. "I would feel comfortable going to church in the winter or to movies or to birthday parties if people could think about those like me who truly suffer as a result of a simple cold virus."

For millions of Americans like Elmore, it would change their lives for the better if it became more of a cultural norm in the U.S. for people to wear masks when they're under the weather or in crowded areas. The West has stigmatized mask-wearing, but in countries like Japan or South Korea, residents might get dirty looks if they hop on a subway with a sniffle and no mask.

Still, there's reason for skepticism. Not everyone in America is wearing masks, even now, when public health officials are strongly encouraging them to do so. Rallies to protest masks have popped up across the country, with many Americans pointing out that it's a violation of their personal freedoms.

But for others, who potentially represent a less vocal majority, it could become the new normal. Since the start of the pandemic, many people bought a handful of masks for the first time and have gotten used to wearing them in public. Doctors and public health experts believe that American culture could fundamentally shift to embrace new hygiene practices.

"I think we do need a new culture of masks, at least any time not feeling well, and I think masks are in and handshakes out for the indefinite future," said Dr. Tom Frieden, the former director of the C.D.C. during the Obama Administration and the president of global health initiative Resolve to Save Lives.

"Post pandemic, there will be new social norms," added Dr. Panagis Galiatsatos, a pulmonary and critical care doctor, who treats patients with chronic respiratory conditions like COPD and cystic fibrosis, as well as Covid-19.

"I think face masks will continue to be used by the general public in times when they don't feel well, and honestly we're realizing that no one feels slighted without a handshake," he said.

Sometimes, it takes a pandemic to change behavior. Across East Asia, mask wearing really took off in the aftermath of the SARS outbreak in 2003. The U.S. was largely spared from SARS. But in Hong Kong, where more than 280 people died, there was widespread panic. All of that led to many countries developing practices around how to tamp down on potential disease outbreaks early, with measures like social distancing, travel bans, and masks.

Almost two decades later, this advanced planning gave the region an edge when it came to Covid-19. Many people already had a mask or two at home and had become used to wearing one. In countries like Taiwan, those who did not wear a mask were occasionally even publicly shamed on social media for failing to take proper precautions.

William Hsiao, emeritus professor of economics inthe Department of Health Policy and Management at the Harvard T.H. Chan School of Public Health, told CNBC that in these cultures, there's a strong feeling that sometimes people have to sacrifice their "individual desires and benefits" for the sake of their community. That helped countries like Taiwan and Vietnam, which have experienced relatively few cases of Covid-19, come together to face a threat in a more unified way. In Taiwan, only 7 people have died from the virus, while Vietnam has reported just 35 deaths.

In the West, it hasn't been as easy to disseminate public health measures. Not all Americans have eagerly embraced mask-wearing, even in the height of the pandemic. Research organizations like Brookings say that "a culture of individualism" is an obstacle. Americans aren't as used to putting the needs of the community ahead of themselves.

Still, some Americans say their perspective has fundamentally shifted. Rather than powering through illness by going to school or work as normal, they say they'd take extra precautions or stay home. And some say that they'd be comfortable wearing a mask in a crowded setting from now on.

"I grew up going to school, even if I was sick" said Spencer Guthrie, 45, from San Francisco. "I never missed a single day of school from K-12."

But Guthrie said his views have changed since the start of the pandemic. He would take greater precautions now if he felt under the weather. "Masks are not the slightest bit inconvenient for long periods of time if you find one that fits well," he said.

Others say they discovered unexpected benefits to wearing a mask. Hope King, a New York-based journalist, has found comfort in that moment on the street when people pull their mask up as they approach. She sees it as a sign of respect.

King has experienced the rise of anti-Asian racism during the pandemic. So wearing a mask makes her feel safer.

"I think maybe you can't tell immediately that I'm Asian from far away, especially if I'm wearing glasses," she said.

King envisions keeping her mask on as long as people around her continue to do so. She also plans to wear one if she starts to feel sick, as a signal to others that she's invested in protecting them.

For some young Americans, wearing a mask just isn't big deal. Gurdane Bhutani, 29, suspects that face coverings won't be perceived as a "strange thing to do" in the future at times when people are packed together. He already refrains from eating peanuts on an airplane in case someone has an allergy. So a mask doesn't seem any different.

"It's not hard to do, and it could make a difference for someone else," he said. "I know people with autoimmune conditions are feeling really relieved that everyone is wearing masks now."

Doctors like Krishna Komanduri, chief of the division of transplantation and cellular therapy at the University of Miami, has noticed an increased empathy for immunocompromised patients in recent months. People with weakened immune systems have long feared getting sick and that prospect is never far from their thoughts.

And now, with Covid-19, even young and healthy people are concerned about avoiding germs and spreading illness.

"For our cancer patients, nothing has really changed," he said. "Rather we have been drawn into their world."

Cancer patients, he said, or really anyone with a compromised immune system, are no strangers to masks and social distancing. Some received strange looks prior to the pandemic, he said. But not anymore. "More widespread adoption of these measures would be good in general," he explained.

For her part, Elmore, who suffers from cystic fibrosis, has never felt safer. And ironically, that's during a respiratory pandemic.

"Now, if someone coughs or sneezes with a mask on in the store, I'm not trying to run to get away," she said. "Pre-Covid, I would literally breathe out and walk as fast as possible to get away from the airflow."

Elmore said her entire family would keep their ears alerted for any hint of sickness at a time when very few people wore masks. "My husband used to say, 'cougher on the left,' to prevent me from breathing as they walked by," she said.

Elmore isn't expecting that everyone will wear masks at all times in the future. She is hoping that people will take more precautions, however, when they start feeling sick. "I do hope those that are having symptoms will consider them," she said.

Read the rest here:
Will masks become the 'new normal' even after the pandemic has passed? Some Americans say so - CNBC

Summary

Two MSK-led studies published in the journal Nature support the idea of using immunotherapy drugs to treat the environment surrounding a tumor as an indirect way to combat cancer.

Despite the remarkable successes of immune-based treatments for cancer, not everyone responds to these approaches, and relapses do occur. Researchers around the world are racing to find ways to improve outcomes for people receiving immunotherapy. But new findings from scientists at Memorial Sloan Kettering suggest they may be focusing too narrowly on the problem.

According to Ming Li, an immunologist in the Sloan Kettering Institute, most existing immunotherapy approaches, including both checkpoint blockade and CAR T therapy, aim to prod the immune system into finding and killing cancer cells a kind of frontal attack on the disease.

But tumors also need supportive environments safe harbors in which to grow and thrive. They need the support of blood vessels that provide them with nutrients, Dr. Li says.

Could destroying these safe harbors be an indirect way to fight this internal enemy? Dr. Li thinks the answer is yes, and on October 21, he published two papers in the journal Nature in support of the concept.

We know that the immune system is incredibly adept at recognizing harmful invaders and attacking them with precision, he says. But thats not the only way our immune system protects us from threats. It also promotes healing of damaged tissue so that pathogens cant take root in the body. This latter role, we now show, can also be enlisted in the fight against cancer.

Dr. Li and his colleagues found they could thwart cancers in mice by encouraging immune cells to begin the process of wound repair around a tumor. In the process, blood vessels that feed the cancer are pruned away and cancer cells starve and die. Its an approach that Dr. Li has dubbed cancer environment immunotherapy.

To understand how wound healing can help curb cancer, consider what happens when someone gets an injury, say a cut from a knife. Early on, the site becomes inflamed red, hot, and swollen. During this phase of wound healing, blood vessels dilate and immune cells rush in to fight enemies that may cause infection and clean up the debris. But later on, the wound fills in with new tissue, and inflammation resolves.

An important player in the wound healing process is a molecule called TGF-beta, whose presence waxes and wanes with the inflammation cycle. In the context of a cancerous wound, TGF-beta persists and makes cancer growth worse. By contrast, blocking its action inhibits tumor development. This latter effect is dependent upon immune cells called T cells, previous research has shown.

Blocking the action of a molecule called TGF-beta in immune cells (right) triggers cell death (blue) in tumors in mice.

Dr. Li and his team wanted to find out more about which T cells were involved in curbing cancer growth when TGF-beta is blocked. They initially suspected that a subset of T cells called CD8 T cells, or killer T cells, were responsible for restraining tumor development. But when they genetically removed the receptor for TGF-beta from CD8 cells in mice, it had no effect on cancer growth.

Next, they asked whether a different subset of T cells, called CD4 T cells, or helper T cells, could explain the phenomenon of cancer suppression. Indeed, genetically removing the receptor for TGF-beta in CD4 T cells dramatically reduced cancer growth in mice.

How do CD4 T cells contribute to cancer control in this context? Dr. Li and his colleagues found that these cells promote wound healing around a tumor. As part of this process, the blood vessels supplying nutrients to a tumor are dramatically remodeled, and a kind of protective wall is formed around the tumor, depriving it of sustenance.

These results, reported in the first Nature paper, showed that blocking TGF-beta signaling in CD4 T cells could activate a powerful wound healing response that directly opposes cancer development.

But what about tumors that have already been growing? Could blocking TGF-beta restrain them? Dr. Li and his team explored this question in a second set of experiments, published in a second Nature article.

By blocking TGF-beta inhelper T cells, we allow the wound healing to run to completion. We heal the wound that is cancer.

They designed an antibody-based drug that can bind to both TGF-beta andhelper T cells. They found that this drug, which they called 4T-Trap, could dramatically reduce cancer in mice.

Previous attempts at blocking TGF-beta as a cancer treatment have not been successful, likely because this molecule has many effects in the body, and therefore blocking it completely can cause severe side effects such as heart problems or even the appearance of new cancers. But 4T-Trap targets the TGF-beta-blocking-molecule directly to CD4 T cells, so side effects are reduced. In other words, its a more targeted approach.

That fact that CD4helper T cells were the key players rather than CD8 killer T cells came as something of a surprise to the researchers. These days, CD8 cytotoxic T cells that recognize cancer cells are in the spotlight. Dr. Li says, Its almost become dogma that if its T cell mediated, then it must be CD8 T cells. That was our original hypothesis, too. But that turns out not to be the case.

Yet the findings are not completely unprecedented. In fact, the discovery that the promotion of wound healing can dramatically curb cancer progression meshes nicely with older work. In the mid-1980s, cancer researcher Harold Dvorak published a now-famous article in the New England Journal of Medicine, in which he argued that tumors are essentially wounds that do not heal. Tumors enlist normal wound healing to help themselves grow. They thrive by enlisting the early stages of the immune responses to tissue damage growth of new blood vessels, for example but then never get to later stages of wound healing when these blood vessels are normally pruned away.

By blocking TGF-beta inhelper T cells, we allow the wound healing to run to completion, Dr. Li says. We heal the wound that is cancer.

Reflecting on the way his results echo these earlier findings, Dr. Li says, Its an exciting homecoming.

He proposes that such cancer environment immunotherapy could be a powerful addition to current immune-based treatments for cancer. His lab is currently collaborating with physician-researchers at MSK to translate these new findings to patients.

Original post:
MSK-led Studies Support the Concept of Cancer Environment Immunotherapy - On Cancer - Memorial Sloan Kettering

Many of the symptoms experienced by people infected with SARS-CoV-2 involve the nervous system. Patients complain of headaches, muscle and joint pain, fatigue and brain fog, or loss of taste and smellall of which can last from weeks to months after infection. In severe cases, COVID-19 can also lead to encephalitis or stroke. The virus has undeniable neurological effects. But the way it actually affects nerve cells still remains a bit of a mystery. Can immune system activation alone produce symptoms? Or does the novel coronavirus directly attack the nervous system?

Some studiesincluding a recent preprint paper examining mouse and human brain tissueshow evidence that SARS-CoV-2 can get into nerve cells and the brain. The question remains as to whether it does so routinely or only in the most severe cases. Once the immune system kicks into overdrive, the effects can be far-ranging, even leading immune cells to invade the brain, where they can wreak havoc.

Some neurological symptoms are far less serious yet seem, if anything, more perplexing. One symptomor set of symptomsthat illustrates this puzzle and has gained increasing attention is an imprecise diagnosis called brain fog. Even after their main symptoms have abated, it is not uncommon for COVID-19 patients to experience memory loss, confusion and other mental fuzziness. What underlies these experiences is still unclear, although they may also stem from the body-wide inflammation that can go along with COVID-19. Many people, however, develop fatigue and brain fog that lasts for months even after a mild case that does not spur the immune system to rage out of control.

Another widespread symptom called anosmia, or loss of smell, might also originate from changes that happen without nerves themselves getting infected. Olfactory neurons, the cells that transmit odors to the brain, lack the primary docking site, or receptor, for SARS-CoV-2, and they do not seem to get infected. Researchers are still investigating how loss of smell might result from an interaction between the virus and another receptor on the olfactory neurons or from its contact with nonnerve cells that line the nose.

Experts say the virus need not make it inside neurons to cause some of the mysterious neurological symptoms now emerging from the disease. Many pain-related effects could arise from an attack on sensory neurons, the nerves that extend from the spinal cord throughout the body to gather information from the external environment or internal bodily processes. Researchers are now making headway in understanding how SARS-CoV-2 could hijack pain-sensing neurons, called nociceptors, to produce some of COVID-19s hallmark symptoms.

Neuroscientist Theodore Price, who studies pain at the University of Texas at Dallas, took note of the symptoms reported in the early literature and cited by patients of his wife, a nurse practitioner who sees people with COVID remotely. Those symptoms include sore throat, headaches, body-wide muscle pain and severe cough. (The cough is triggered in part by sensory nerve cells in the lungs.)

Curiously, some patients report a loss of a particular sensation called chemethesis, which leaves them unable to detect hot chilies or cool peppermintsperceptions conveyed by nociceptors, not taste cells. While many of these effects are typical of viral infections, the prevalence and persistence of these pain-related symptomsand their presence in even mild cases of COVID-19suggest that sensory neurons might be affected beyond normal inflammatory responses to infection. That means the effects may be directly tied to the virus itself. Its just striking, Price says. The affected patients all have headaches, and some of them seem to have pain problems that sound like neuropathies, chronic pain that arises from nerve damage. That observation led him to investigate whether the novel coronavirus could infect nociceptors.

The main criteria scientists use to determine whether SARS-CoV-2 can infect cells throughout the body is the presence of angiotensin-converting enzyme 2 (ACE2), a protein embedded in the surface of cells. ACE2 acts as a receptor that sends signals into the cell to regulate blood pressure and is also an entry point for SARS-CoV-2. So Price went looking for it in human neurons in a study now published in the journal PAIN.

Nociceptorsand other sensory neuronslive in discreet clusters, found just outside the spinal cord, called dorsal root ganglia (DRG). Price and his team procured nerve cells donated after death or cancer surgeries. The researchers performed RNA sequencing, a technique to determine which proteins a cell is about to produce, and they used antibodies to target ACE2 itself. They found that a subset of DRG neurons did contain ACE2, providing the virus a portal into the cells.

Sensory neurons send out long tendrils called axons, whose endings sense specific stimuli in the body and then transmit them to the brain in the form of electrochemical signals. The particular DRG neurons that contained ACE2 also had the genetic instructions, the mRNA, for a sensory protein called MRGPRD. That protein marks the cells as a subset of neurons whose endings are concentrated at the bodys surfacesthe skin and inner organs, including the lungswhere they would be poised to pick up the virus.

Price says nerve infection could contribute to acute, as well as lasting, symptoms of COVID. The most likely scenario would be that the autonomic and sensory nerves are affected by the virus, he says. We know that if sensory neurons get infected with a virus, it can have long-term consequences, even if the virus does not stay in cells.

But, Price adds, it does not need to be that the neurons get infected. In another recent study, he compared genetic sequencing data from lung cells of COVID patients and healthy controls and looked for interactions with healthy human DRG neurons. Price says his team found a lot of immune-system-signaling molecules called cytokines from the infected patients that could interact with receptors on neurons. Its basically a bunch of stuff we know is involved in neuropathic pain. That observation suggests that nerves could be undergoing lasting damage from the immune molecules without being directly infected by the virus.

Anne Louise Oaklander, a neurologist at Massachusetts General Hospital, who wrote a commentary accompanying Prices paper in PAIN, says that the study was exceptionally good, in part because it used human cells. But, she adds, we dont have evidence that direct entry of the virus into [nerve] cells is the major mechanism of cellular [nerve] damage, though the new findings do not discount that possibility. It is absolutely possible that inflammatory conditions outside nerve cells could alter their activity or even cause permanent damage, Oaklander says. Another prospect is that viral particles interacting with neurons could lead to an autoimmune attack on nerves.

ACE2 is widely thought to be the novel coronaviruss primary entry point. But Rajesh Khanna, a neuroscientist and pain researcher at the University of Arizona, observes that ACE2 is not the only game in town for SARS-CoV-2 to come into cells. Another protein, called neuropilin-1 (NRP1), could be an alternate doorway for viral entry, he adds. NRP1 plays an important role in angiogenesis (the formation of new blood vessels) and in growing neurons long axons.

That idea came from studies in cells and in mice. It was found that NRP1 interacts with the viruss infamous spike protein, which it uses to gain entry into cells. We proved that it binds neuropilin and that the receptor has infectious potential, says virologist Giuseppe Balistreri of the University of Helsinki, who co-authored the mouse study, which was published in Sciencealong with a separate study in cells. It appears more likely that NRP1 acts as a co-factor with ACE2 than that the protein alone affords the virus entry to cells. What we know is that when we have the two receptors, we get more infection. Together, its much more powerful, Balistreri adds.

Those findings piqued the interest of Khanna, who was studying vascular endothelial growth factor (VEGF), a molecule with a long-recognized role in pain signaling that also binds to NRP1. He wondered whether the virus would affect pain signaling through NRP1, so he tested it in rats in a study that was also published in PAIN. We put VEGF in the animal [in the paw], and lo and behold, we saw robust pain over the course of 24 hours, Khanna says. Then came the really cool experiment: We put in VEGF and spike at the same time, and guess what? The pain is gone.

The study showed what happens to the neurons signaling when the virus tickles the NRP1 receptor, Balistreri says. The results are strong, demonstrating that neurons activity was altered by the touch of the spike of the virus through NRP1.

In an experiment in rats with a nerve injury to model chronic pain, administering the spike protein alone attenuated the animals pain behaviors. That finding hints that a spike-like drug that binds NRP1 might have potential as a new pain reliever. Such molecules are already in development for use in cancer.

In a more provocative and untested hypothesis, Khanna speculates that the spike protein might act at NRP1 to silence nociceptors in people, perhaps masking pain-related symptoms very early in an infection. The idea is that the protein could provide an anesthetic effect as SARS-CoV-2 begins to infect a person, which might allow the virus to spread more easily. I cannot exclude it, says Balistreri. Its not impossible. Viruses have an arsenal of tools to go unseen. This is the best thing they know: to silence our defenses.

It still remains to be determined whether a SARS-CoV-2 infection could produce analgesia in people. They used a high dose of a piece of the virus in a lab system and a rat, not a human, Balistreri says. The magnitude of the effects theyre seeing [may be due to] the large amount of viral protein they used. The question will be to see if the virus itself can [blunt pain] in people.

The experience of one patientRave Pretorius, a 49-year-old South African mansuggests that continuing this line of research is probably worthwhile. A motor accident in 2011 left Pretorius with several fractured vertebrae in his neck and extensive nerve damage. He says he lives with constant burning pain in his legs that wakes him up nightly at 3 or 4 A.M. It feels like somebody is continuously pouring hot water over my legs, Pretorius says. But that changed dramatically when he contracted COVID-19 in July at his job at a manufacturing company. I found it very strange: When I was sick with COVID, the pain was bearable. At some points, it felt like the pain was gone. I just couldnt believe it, he says. Pretorius was able to sleep through the night for the first time since his accident. I lived a better life when I was sick because the pain was gone, despite having fatigue and debilitating headaches, he says. Now that Pretorius has recovered from COVID, his neuropathic pain has returned.

For better or worse, COVID-19 seems to have effects on the nervous system. Whether they include infection of nerves is still unknown like so much about SARS-CoV-2. The bottom line is that while the virus apparently can, in principle, infect some neurons, it doesnt need to. It can cause plenty of havoc from the outside these cells.

Read more about the coronavirus outbreak from Scientific American here. And read coverage from our international network of magazines here.

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What We Know So Far about How COVID Affects the Nervous System - Scientific American

Dive Brief:

Sarepta is best known for its RNA technology platform, which has led to two approved though also controversial drugs for Duchenne muscular dystrophy.

Yet, Sareptais also deeply invested in gene therapy, having developed an extensive list of more than two dozen experimental treatments, six of which have reached human testing.

Rare diseases have been an early target in this rapidly growing field. The two gene therapies approved in the U.S., Roche's Luxturna and Novartis' Zolgensma, are respectively used to treat an uncommon form of blindness and a muscle disease that occurs in about 1 in every 10,000 births.

Sarepta'stherapies target a wide variety of rare diseases, including Duchennemuscular dystrophy, Pompe disease, and types of Limb-girdle muscular dystrophy. Taking a stake in AavantiBio, with its work in Friedreich'sataxia, could expand Sarepta'sreach even further. The disease affects approximately 1 in every 40,000 people, according to the National Organization for Rare Disorders, which would equate to around 8,200 patients in the U.S.

"Our equity participation in AavantiBio serves our strategy to build our gene therapy engine through targeted investment in potentially life-enhancing therapies,"Doug Ingram, Sarepta'sCEO, said in a statement Thursday.

AavantiBio joins a couple large, powerful companies in the hunt for a gene therapy to treat Friedreich's ataxia. Pfizer and Novartis are each working on their own programs.

Outside of gene therapy, Reata Pharmaceuticals disclosed last year positive datafrom a study that tested an oral drug, known as omaveloxolone,in patients with Friedreich's ataxia. Reata said it intends to file the drug for approval based on those results.

AavantiBiowill be headquartered in the greater Boston area, putting it close by Cambridge, Massachusetts-based Sarepta. Cumbo, along with his role as CEO, will take one of AavantiBio'seight board of directors seats. Cumbohas, during the span of his career, helped launch 11 specialty products across multiple drug companies, including Sarepta, Gilead and Vertex, where he built a sales team for the hepatitis C drug Incivek.

Co-founders Byrne and Corti will be on the board too, alongside two independent directors and representatives from the investor group of Perceptive, Bain and RA.Louise Rodino-Klapac, senior vice president of gene therapy for Sarepta, will serve as a board observer.

Read more here:
Sarepta, continuing its gene therapy push, helps launch a startup - BioPharma Dive

Some technologies that have emerged and altered the landscape in recent years include immunotherapy, CRISPR-Cas9 gene editing, and chimeric antigen receptor (CAR) T-cell therapies. Now, another platform technology is maturing from the research laboratory to commercial viability. In 2017, the U.S. Food and Drug Administration (FDA) approved the first directly administered gene therapy for mutations of a specific, disease-related gene. That product, Luxturna, marketed by Spark Therapeutics, delivers a functioning copy of the RPE65 gene to retinal cells using an adeno-associated virus (AAV) as a vector to treat a genetic form of blindness.

This advance has injected new energy into biotech startups seeking to capitalize on gene therapy.

As the field matures, gene therapy companies will eventually need to find a way to sell once-in-a-lifetime cures in a market built around chronic therapies. In some ways, its been easier for our European counterparts and other single-payer economies, [research analyst Mani] Foroohar comments. [They] can rest more assured that the financial benefits of reducing future hospitalizations due to expensive future illness will be captured by the same payor thats providing the upfront outlay.

Just as biotechnology has transformed every aspect of our healthcare system over the last 40 years, gene therapy will challenge, disrupt, and overturn our healthcare pricing and reimbursement paradigms as it becomes an increasingly common and routine treatment approach.

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Resurgence of gene therapy has dramatically altered the the biomedicine revolution - Genetic Literacy Project

Paris-based SparingVision, a genomic medicine company focused on ocular diseases, raised 44.5 million (approximately $52.2 million) in a financing round. Funds will be used to advance the development of the companys treatment for a genetic eye disorder that can lead to vision loss.

SparingVision is developing SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa, the most common inherited retinal degeneration that affects about two million people globally. There is currently no approved treatment to treat all genetic forms of this rare retinal disease that leads to blindness, the company said. According to SparingVision, SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting synergistically, aim to slow or stop the degeneration of photoreceptors. Loss of photoreceptors leads to blindness in retinitis pigmentosa. In June, the European Commission granted Orphan Drug designation to SPVN06

In addition to advancing its gene therapy treatment, funds from the financing round will be used to support SparingVisions GMP activities, including the manufacturing of a first clinical batch of the product, as well as regulatory activities. Funds will also be used to begin human trials of the gene therapy, which are set to begin in 2021.

Gene therapy has already been approved by regulatory to approve a type of genetic blindness. Spark Therapeutics, now a division of Roche, won regulatory approval for Luxturna (voretigene neparvovec), a gene therapy for a rare, genetic form of blindness. Luxturna is approved for the treatment of pediatric and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The disease can lead to vision loss and may cause complete blindness in certain patients. The approval marked the first time the U.S. Food and Drug Administration approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene.

In addition to developing its gene therapy for retinitis pigmentosa, SparingVision said it intends to establish a toehold in the United States and will expand its management team.

The financing round was led by 4BIO Capital and UPMC Enterprises. It was supported by Jeito Capital and Ysios Capital. Current investors Bpifrance and Foundation Fighting Blindness also participated in the round. Torreya Capital, LLC served as exclusive placement agent for the offering.

In addition to the financing, Stphane Boissel, who currently serves as chairman of the board of directors, was named chief executive officer of the company. He takes over from Florence Allouche, a cofounder of the company. Boissel, who previously served as head of corporate strategy at Sangamo Therapeutics, said the support SparingVision received in the financing round demonstrates the excitement about the potential of SPVN06.

With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals, Boissel said in a statement.

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SparingVision Nabs 44.5 Million to Support Gene Therapy, Adds New CEO - BioSpace

Luke Timmerman interviews Sana Biotechnology CEO Steve Harr at the GeekWire Summit this week.

Sana Biotechnology CEO Steve Harr shed more light on one of most secretive, heavily funded startups in Seattle and the global biotech industry detailing its plans to create tools that replace and repair human body cells, with the potential to treat various diseases and create new medicines.

Harr spoke with biotechnology journalist Luke Timmerman, founder of The Timmerman Report, this week at the GeekWire Summit. Sana raised more than $700 million this summer in one of the largest venture financing deals in the life sciences industry and one of the biggest rounds on record in Seattle.

Founded in 2019, the 250-person company has an ambitious goal of both repairing cells in the body (gene therapy) and also replacing damaged cells (cell therapy). Its led by several former executives from Juno Therapeutics, another Seattle biotech company that went public in 2014 and sold to Celgene for $9 billion in 2018.

Sana has kept a relatively low profile since launching. It is competing with much larger entities that have deeper pockets and more robust logistics capabilities. But Harr said a startup such as Sana has a key differentiator.

We have one competitive advantage: we can make faster and better decisions, he said. We get there because we have better people, we have greater focus, and we have better communication.

Read on for key takeaways from the conversation.

How Sana started:Harr and his former colleagues at Juno learned a lot about engineering cells and manipulating genes during their startup journey. Juno was among a handful of U.S. companies making cutting-edge cancer immunotherapy treatments.

But they also knew there was more opportunity in a nascent industry of gene and cell therapy.

We wanted to build the transformative or winning company of this next era, of the next 20 years, Harr said. To do that, we had to break the model of what biotech is, which is typically taking an idea and figuring out where to apply it best.

Sana instead is trying to build the platform that can engineer cells and fix them, much like building a computer.

There are a whole host of component parts that go into it, Harr said. We have to aggregate the right technologies.

Harr said too many biotech companies sell solutions in search of problems. He likened it to someone showing up with a tiny screwdriver and looking for a loose screw to fix. Harr sees Sana more as a toolbox that can help build the right medicine for the right patient.

Sana is targeting various disease areas, including cancer, diabetes, genetic disorders, and more. They are relatively diverse, but there are some really fundamental underlying platform and strategy principles that drive each of those, Harr said.

Sanas secret sauce:One key focus for the company is reimagining the delivery system for these therapies how to get DNA, RNA, proteins, etc. into a cell. Ultimately at the core, what were trying to do is really improve delivery, and really figure out how to hide cells from the immune system, Harr said.

Hiding re-engineered or replaced cells from ones immune system is important to prevent the possibility of the body rejecting the new cells.

Harr also talked about delivering therapy via injection, with the body becoming the bioreactor. Its similar to technology built by Moderna and others. You deliver the tools to enable your body to make its own medicine, Harr said.

Manufacturing:Sana is also aiming to innovate how gene and cell therapies are produced and distributed at scale. They are typically expensive Timmerman said CAR T-cell immunotherapies for cancer ran in the $300,000-to-$400,000 range per patient. Figuring out manufacturing costs at scale and making it less than current alternative methods of care for patients will be key to the strength of Sanas business. Harr added that you have to do it in a way thats constructive for the system.

Headcount: Sana employs 250 people spread across offices in Seattle, the Bay Area, and Cambridge, Mass. Having three outposts helps the company attract the best talent, Harr said. One advantage to raising so much capital is being able to hire the best folks. Last month Sana added top scientists Ed Rebar and Terry Fry to the executive team.

If you really hire one of the true world leaders in something, it is pretty amazing how quickly teams form around them, Harr noted.

Money matters: Having more than $700 million in the bank helps Sana in various other ways. Harr said a lot of biotech companies often run what amount to experiments to justify raising more capital. We have the privilege of running experiments to find truth as fast as you want to, he said. And then we want to have the balance sheet, technologies, and people to be able to grapple with whatever the truth is.

Timeline: Harr said the company is on track with its original strategy but does not plan on selling medicine in the next two years. It will progress with multiple medicines in parallel, not one at a time, Harr said.

Leadership advice:During the pandemic and remote work, Harr said hes started to reach out to four-to-six people at Sana each week that he wouldnt normally talk with. He holds half-hour meetings to chat about what they are working on, and what leadership could do to make their life or job better. I found that to be just such an invigorating way to learn whats going on, he said.

[The full interview with Harr, and other GeekWire Summit sessions, are available on-demand exclusively to attendees of the virtual event.Learn more and register here.]

Link:
Sana CEO reveals details about stealthy gene therapy startup that has raised more than $700M - GeekWire

NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that it will host a virtual R&D Day on Friday, October 30, 2020 at 11:30 AM Eastern time, to discuss the Companys AXO-Lenti-PD gene therapy for Parkinsons disease.

Axovants Parkinsons disease R&D Day will be moderated by Chief R&D Officer, Gavin Corcoran, M.D., and will feature presentations on the current treatment landscape and unmet medical need for people living with Parkinsons disease from the following key opinion leaders:

In addition, the Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including:

Drs. Adler, Palfi, and Eberling will be joined by Dr. Corcoran to answer questions following the formal presentations.

AXO-Lenti-PD is the only investigational gene therapy for Parkinsons disease that delivers three genes via a lentiviral vector to encode a set of critical enzymes required for endogenous dopamine synthesis, with the goal of improving motor function and restoring steady, tonic levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

To register for the R&D webcast, please click here.

A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.

Biographies of R&D Day Panelists:

Dr. Adler has received numerous grants to investigate experimental treatments for Parkinson's disease, essential tremor, dystonia, restless legs syndrome, and chronic traumatic encephalopathy (CTE). He serves as an advisory member to many different international medical societies such as the International Parkinson and Movement Disorder Society, MDS Industry Education and Services Committee, and the American Academy of Neurology Section of Movement Disorders. Dr. Adler has a commitment to education having trained residents, 14 fellows and graduate students, and has given many invited lectures. Dr. Adlers main research interests are investigating tissue diagnostic tests for Parkinsons disease, biomarkers for an early diagnosis of Parkinsons disease and PD with dementia, and identification of new treatments for PD and PD with dementia. He also has been investigating essential tremor, restless legs syndrome, and dystonia. He has published over 400 research papers and reviews, and edited a book entitled Parkinson's Disease and Movement Disorders: Diagnosis and Treatment Guidelines for the Practicing Physician. In 2006, Dr. Adler was awarded the Mayo Clinic Distinguished Investigator of the Year Award.

Dr. Palfi has published extensively on trophic factor- and enzyme-based gene therapy in Parkinsons disease and Huntingtons disease. He is a principal investigator on numerous preclinical and clinical studies and has been involved in studies of many novel agents including implanted brain devices, optogenetic, homeoprotein, trophic factors GDNF, CNTF and dopamine lentiviral vectors.

Dr. Eberling earned undergraduate and graduate degrees in biological psychology from the University of California at Berkeley, later moving to the Lawrence Berkeley National Laboratory where she developed expertise in neuroimaging techniques and gene therapy approaches for Parkinsons disease.

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:

Investors

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

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.commedia@axovant.com

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Axovant Gene Therapies to Host Virtual Parkinson's Disease R&D Day on October 30, 2020 - BioSpace

Biogen is cutting its experimental multiple sclerosis (MS) drug opicinumab as well as an spinal muscular atrophy (SMA) candidate amid a third-quarter clear-out.

First, to its MS drug. In its third-quarter financials posted Wednesday morning, the biopharma said: In October 2020 Biogen announced that the phase 2 AFFINITY study of opicinumab in MS did not meet its primary or secondary endpoints and that Biogen has discontinued development of opicinumab.

The anti-LINGO monoclonal antibody has experience with failure: All the way back in 2016, it posted a similar phase 2 flop after not hitting its primary and secondary endpoints for MS in the so-called SYNERGY trial.

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Back then, opicinumab was seeing whether it could have an effect on a multicomponent primary endpoint designed to assess ambulation, upper extremity function and physical disability in patients with relapsing forms of MS. Opicinumab, however, failed to outperform placebo in this regard.

A secondary efficacy endpoint, intended to evaluate the slowing of disability progression, also came up negative. But Biogen dug through the data, and, instead of culling it then and there, decided to carry on, saying: While we missed the primary endpoint, the SYNERGY study results suggest evidence of a clinical effect of opicinumab.

RELATED: Biogen tumbles on PhII MS flop, but refuses to write off drug

Biogen believed the data showed an increased clinical effect of opicinumab versus placebo (when used at the same time as interferon beta-1a intramuscular injection).

That led to the AFFINITY study, started in 2017, which looked at opicinumab as an add-on therapy in patients who are adequately controlled on their anti-inflammatory disease-modifying therapy (DMT), versus the DMT alone.

The primary endpoint of the study, overall response score, looked to assess the improvement and worsening of disability over time. Clearly, it failed to achieve this, though in its third-quarter update it did not reveal further details.

It also quietly announced it was throwing out BIIB089, an SMA gene therapy hopeful that had been on an IND hold due to dorsal root ganglion toxicity. Again, it did not any extra color to this cull in its financials.

The biopharma is now pinning its hopes on another failed drug, aducanumab, which it hopes can get past an FDA advisory committee early next month and a potential approval next year, though this controversial Alzheimers disease asset has been given low odds by analysts of managing that feat.

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Biogen finally culls MS dud opicinumab, adds SMA gene therapy to the garbage heap - FierceBiotech

WATERTOWN, Mass. and RESEARCH TRIANGLE PARK, N.C., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations. The FDA grants Rare Pediatric Disease Designation to incentivize development of new treatments for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the U.S. The Rare Pediatric Disease designation program allows for a Sponsor who receives an approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

This Rare Pediatric Disease designation from the FDA highlights the significant unmet medical need that Selecta and AskBio are seeking to address with MMA-101 for this rare metabolic disorder, said Carsten Brunn, Ph.D., chief executive officer of Selecta Biosciences. When used with AAV gene therapy vectors, Selectas ImmTOR aims to inhibit the immune response to the AAV vector, potentially allowing re-dosing of gene therapies. Ongoing clinical programs will focus on evaluating product candidate performance in patients who may have been underdosed or those who may lose transgene expression over time. Were honored to receive this recognition and look forward to advancing this program in hopes of helping young patients affected by MMA and their families.

MMA is a serious and potentially life-threatening inherited metabolic disorder that presents in patients from newborns to adulthood, said Sheila Mikhail, J.D., CEO and co-founder of AskBio. AskBio is committed to delivering transformative genetic medicines for rare diseases like this one, and the Rare Pediatric Disease designation helps us continue development of MMA-101.

AskBio and Selecta expect to initiate a Phase 1 clinical trial of MMA-101 and ImmTOR for patients with MMA in 1H 2021.

About Methylmalonic AcidemiaMethylmalonic Acidemia (MMA) is a rare monogenic disorder in which the body cannot break down certain proteins and fats. This metabolic disease may lead to hyperammonemia and is associated with long-term complications including feeding problems, intellectual disability, chronic kidney disease and inflammation of the pancreas. Symptoms of MMA usually appear in early infancy and vary from mild to life-threatening. Without treatment, this disorder can lead to coma and in some cases death.

About Selecta Biosciences, Inc.Selecta Biosciences, Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the bodys natural self-tolerance in autoimmune diseases. The companys first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit http://www.selectabio.com. About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. Its pipeline includes clinical-stage programs in Pompe disease and congestive heart failure and a diverse preclinical portfolio of therapeutics targeting neuromuscular, CNS and other diseases, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics, acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics, acquired by Pfizer). AskBios gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the company), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy and MMA, the companys plans to initiate a clinical trial for a product candidate to treat MMA, the ability of the company and AskBio to develop gene therapy products using ImmTOR and AskBios technology, any development plans of the company and AskBio have for product candidates to treat serious and life-threatening diseases and the intention to seek regulatory approval thereof, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the companys intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AskBio Forward-Looking StatementsThis press release contains forward-looking statements regarding AskBio. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans, expects, will, intends, potential, possible and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding MMA-101, including the potential timing of the Phase 1 clinical trial for patients with MMA, AskBios pipeline of development candidates; AskBios goal of developing life-saving medicines aimed at curing genetic diseases; the potential benefits of AskBios development candidates to patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond AskBios control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

Any of the foregoing risks could materially and adversely affect AskBios business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

For more information please contact:

Selecta: For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

For Media: Meredith Sosulski, Ph.D.LifeSci Communications, LLC+1-929-469-3851msosulski@lifescicomms.com

AskBio:Robin FastenauVice President, Communications+1-984-275-2705rfastenau@askbio.com

Link:
Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia - GlobeNewswire

In anticipation of our Pfizer Gene Therapy Career Event scheduled for Wednesday, October 28, 2020, BioSpace spoke with Alison Ricci, Pfizers Senior Business HR Partner- Global Sciences, Business HR for Bioprocess Research and Development, and HR Site Lead for the companys four locations in Chapel Hill and Morrisville, North Carolina, Chesterfield, Missouri and Lake Forest, Illinois.

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. This is in addition to the companys continuing investment into gene therapy research and development and other areas going on throughout the company, but specifically in its North Carolina sites in Chapel Hill and Kit Creek. The companys presence in North Carolina currently exceeds 3,600 people, with 650 in Sanford. The expanded facility is expected to add over 100 new jobs.

Ricci told BioSpace the company has been investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area. They have three facilities in and around the area that focus specifically on gene therapy, including a research facility in Morrisville, its existing clinical manufacturing site in Chapel Hill, and a very large manufacturing site in Sanford.

And in recognition of the continued investment and commitment to gene therapy, the company acquired a 60,000-square-feet building and 16 acres in Durham, which they are renovating into a clinical manufacturing site, essentially doubling our clinical manufacturing capacity.

*Rendering of Durham location

As such, the company has numerous positions in the area currently open with many more expected as the new site comes online.

Right now, we have 30 positions that were going to be advertising for that directly support gene therapy in those three different campuses, Ricci said.

The roles they will be looking to fill include what Ricci calls a multitude of individual contributor, team lead and management roles."

"For instance, weve got our R&D and analytical scientist roles; we have manufacturing engineering roles; we have technical specialist roles; and different types of manufacturing support and technology transfer roles, Ricci said.

They also have training specialist positions because there are a variety of supporting functions that go into the magic of making gene therapy work.

Ricci seemed excited about gene therapy and the part it is playing increasingly in Pfizers core mission. Speaking of Duchenne muscular dystrophy (DMD), increasingly a target of gene therapies, she noted how exciting and important it is to be able to provide a therapy that extends quality of life, that doesnt just treat symptoms, but that potentially marks a minimization of those symptoms and the elongation of life. Thats one of the central core components of who Pfizer is. Our patients are our North Star.

She also emphasized not only the science and motivation of Pfizer as an employer, but its culture.

People typically equate large organizations with a level of bureaucracy, that results in less risk and less innovation opportunities, Ricci said. In my five years with Pfizer Ive seen the culture shift to focus on innovation, inclusion, quality and smart resourcing figuring out how to free up resources so we can focus on bold moves and exciting new therapies allowing our scientists to stretch and grow, and make a difference for the patients that count on us.

The company has also structured rewards and recognition around those types of approaches, not only for people theyre bringing into the organization, but with its current staff.

Were still maintaining our focus on a combination of productivity and doing it right the first time while encouraging innovation, taking bold moves and trying new things," Ricci said. "Because thats the best way that good science is done, and patients win in the process.

She also notes that the Pfizer Gene Therapy Career Event is a great opportunity to have direct contact not only with Pfizer hiring managers, but with colleagues who are working in those same roles theyre hiring for.

Its a unique experience in terms of really understanding who Pfizer is and what we have to offer, Ricci said.

And, of course, the company is hiring throughout its worldwide operations in support of not only its COVID-19 clinical research trials and manufacturing processes, but in the numerous other areas the company focuses on.

The expansion in Sanford, North Carolina is only a part of the companys focus on building out capacity. It has committed about $5 billion in U.S.-based capital projects over the next several years.

But the North Carolina area is expanding end-to-end capabilities in gene therapy. The Kit Creek facility focuses on small scale production, from 2L flasks up to 250L bioreactors to develop the early processes that will later be used in larger scale manufacturing. The Chapel Hill facility is where the process is optimized, and staffers work at a 250L scale while developing and implementing quality control measures that include Good Manufacturing Practice (GMP) standards. The Sanford facility is designed for high-quality, efficient supply of gene therapies at clinical- and commercial-scale.

Ricci also points to Paul Mensah, Pfizers Vice President of Bioprocess Research and Development.

He has a nice, pragmatic style and approach in terms of how he continues to foster and leverage innovation, but also the science of how everyone works together to collaborate and deliver. He instills that in the leaders on the team. Ricci said.

Were continuing to focus in terms of career development and the aspirations of each colleague. There are opportunities for colleagues at Pfizer to do anything they want within the realms of the organization." Ricci said. "You can be on the frontlines of early stage development, you can work on the late stage, you can have opportunities for clinical manufacturing. You can run the gamut in terms of different roles with the scientific profile within Pfizer.

Originally posted here:
Pfizer: Expanding North Carolina Gene Therapy Facility and Adding Jobs - BioSpace

Irvine, Calif., October 19, 2020 A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing, illustrates the use of a new generation CRISPR technology and lays the foundation for the development of a new therapeutic modality for a wide range of inherited ocular diseases caused by different gene mutations.

In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function, said Krzysztof Palczewski, PhD, the Irving H. Leopold chair and a distinguished professor in the Gavin Herbert Eye Institute, Department of Ophthalmology at the UCI School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, said first author Susie Suh, assistant specialist in the UCI School of Medicine Department of Ophthalmology. We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, said co-first author Elliot Choi, also an assistant specialist in the UCI Department of Ophthalmology.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the UCI team successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases. Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu, PhD.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast and spatial and temporal frequency, said Palczewski. These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases.

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy. Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

This research was supported in part by grants from the National Institutes of Health, the Research to Prevent Blindness Stein Innovation Award, Fight for Sight, the Eye and Tissue Bank Foundation (Finland), The Finnish Cultural Foundation, the Orion Research Foundation, the Helen Hay Whitney Foundation, US Department of Veterans Affairs, and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

About the University of California, Irvine: Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visit http://www.uci.edu.

Media access: Radio programs/stations may, for a fee, use an on-campus ISDN line to interview UCI faculty and experts, subject to availability and university approval. For more UCI news, visit news.uci.edu. Additional resources for journalists may be found at communications.uci.edu/for-journalists.

About the UCI School of Medicine: Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI-led study reveals significant restoration of retinal and visual function following gene therapy - UCI News

MALVERN, Pa., Oct. 22, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen, will speak on the importance of strategic partnerships in gene therapy including product consistency, identifying the right partner as well as providing examples of successful partnerships at the 4th Annual Gene Therapy for Rare Disorders Europe Conference from October 26-28, 2020 presented digitally.

Presentation Details:Title:The Importance of Strategic Partnerships in Gene TherapyDate: Wednesday, October 28, 2020Time: 11:00 AM (GMT)Location: DigitalRegister: https://genetherapy-europe.com/take-part/register/

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact: LaVoieHealthScience Katie Gallagherkgallagher@lavoiehealthscience.com+1 617-792-3937

Original post:
CEO Dr. Shankar Musunuri to Discuss the Importance of Gene Therapy Strategic Partnerships at 4th Annual Gene Therapy for Rare Disorders Europe...

Wishing on a star is one of the most magical rites of childhood:

Star light, star bright,The first star I see tonightI wish I may, I wish I might,Have the wish I wish tonight.

But for one little Canadian boy named Sam, that simple activity was beyond his reach. Thats because 8-year-old Sam couldnt see the stars.

Sam suffers from the rare disorder retinitis pigmentosa, a form of progressive blindness caused by genetic retinal degeneration that results from mutations in the RPE65 gene.

You lose perception of light, Dr. Elise Heon, of Sick Kids Hospital, explained to CTV News. You end up in darkness and [its] slowly progressive, its relentless, your visual field shrinks and shrinks and shrinks and shrinks.

Sams sight was extremely limited, especially at night. Images most people take for grantedthe stars, an airplane streaking across the sky, or even our own shoeswere beyond the scope of his vision. Until recently, there was no effective treatment for his condition.

RELATED:Scientists Use New Holy Grail Gene Therapy to Heal Damage Caused By Heart Attacks and it Could Save Millions

Now, however, thanks to a new form of gene therapy, many patients, including Sam, are seeing huge improvements in their eyesight. The science behind the protocol is impressive.

After being modified with a healthy copy of the gene, an inactivated virus is injected directly into the retina. (Each eye is injected only once.) The healthy gene then goes to work, enabling cells to produce a protein that converts light into electrical signals, which in turn, facilitates improved vision and prevents further progression of the disease.

The targeted gene therapy protocol, developed in the U.S., was recently green-lit for use in Canada, but with Sams sight failing, he and his mom, Sarah Banon, traveled to America last year to get him treatment.

Within a weeks time, Banon began to notice progress and says Sams condition has continued to improve over the course of the year since he underwent the procedure.

She reports her son has gained incredible confidence. He dresses without help. Hes able to see, even when its dark, and he no longer requires lights on when its cloudy outside.

MORE: In World First, Gene Therapy Trial Restores Vision in Patients With Genetic Form of Blindness

Now he is able to function as a normal child, she told CTV. This is a story of hope A child told it is what it is, and now, when he looks up at night, he can see stars.

And when Sam wishes on those stars, hell know in his heart that sometimes, wishes really can come true.

Cure Your Friends Of Negativity By Sharing The Good News To Social Media

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8-Year-old Sees Stars for the First Time After His Blindness is Treated With Gene Therapy - Good News Network