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Category Archives: Transhuman News

US government aims for better coordination in space weather campaign – SpaceNews

Posted: March 26, 2020 at 6:38 am

After decades of fighting to be taken seriously, meteorologists say space weather is beginning to get the attention it deserves.

The Trump administration continued the Space Weather Operations, Research and Mitigation (SWORM) working group established by the Obama Administration. SWORM is an interagency panel focused on coordination of federal work aimed at building resilience to the effects of space weather. In addition, space weather legislation is pending in the U.S. Senate and House of Representatives.

Everyone agrees this issue is important and it has to be addressed, Bill Murtagh, NOAA Space Weather Prediction Center program coordinator, said at the American Meteorological Society (AMS) conference in Boston earlier this year.

For years, space weather experts in government, industry and academia have been pointing out how vulnerable the electric power grid and Global Positioning System satellites are to solar storms.

The U.S. government has finally figured out that the potential for catastrophic economic impacts from a large space weather event is cause for concern, David Klumpar, director of Montana State Universitys Space Science and Engineering Lab, said by email.

Meanwhile, NASA is preparing to send people beyond low Earth orbit for the first time in 50 years, exposing astronauts to higher levels of the suns radiation. Sun activity is likely to rise in the 2020s as the current solar minimum makes way for solar maximum. And the Defense Department is creating a U.S. Space Force.

As solar maximum comes again and the Space Force stands up, we will start having a lot more specific requirements, Maj. Janelle Jenniges, Air Force Space Weather Integration chief, said at the AMS conference.

Even with the renewed attention, experts say, it will take years for the U.S. government to improve the coordination of space weather activities and to fill the gaps in its space weather observing systems.

The National Academies plans to hold a workshop in mid-2020 in the Washington area to examine the U.S. space weather infrastructure and proposals for improving it.

We want to take a holistic approach rather than an ad hoc opportunistic approach, Elsayed Talaat, director of the National Oceanic and Atmospheric Administrations Office of Projects, Planning and Analysis, said at AMS.

As those discussions take place, key U.S. and European space weather satellites are approaching the end of their lives. NASAs Advanced Composition Explorer, sent to Earth-Sun Lagrange Point 1 in 1997 to monitor solar wind and energetic particles, is expected to run out of propellant around 2024. NASAs remaining Solar Terrestrial Relations Observatory satellite, launched in 2006 to orbit the sun and provide imagery of coronal mass ejections and other phenomena, will detect solar activity days before it reaches Earth for about two more years. The solar panels on NASA-European Space Agency Solar and Heliophysics Observatory (SOHO) are set to stop working by 2025. SOHO has monitored coronal mass ejections from Lagrange Point 1 since 1995.

The job of replacing aging space weather satellites and launching new ones is shared by federal agencies. NASA and the National Science Foundation contribute to space weather research and modeling. NOAA issues space weather forecasts. The Defense Department creates additional classified and unclassified space weather analysis and forecasts.

U.S. Sens. Gary Peters (D-Mich.) and Cory Gardner (R-Colo.) introduced the Space Weather Research and Forecasting Act in 2019 to clarify the roles and responsibilities of each agency with respect to space weather. The bill also calls on NOAA to develop a replacement for SOHO and directs the Department of Homeland Security to identify critical infrastructure that could be disrupted by space weather. The Senate passed similar legislation in 2017.

In the House, Rep. Ed Perlmutter (D-Colo.) introduced a bill, Promoting Research and Observations of Space Weather to Improve the Forecasting of Tomorrow, to delineate federal agency roles and encourage greater information sharing among federal, academic or commercial space weather forecasters. The House Science, Space and Technology Committee approved the legislation in January.

Space weather can cause significant damage to our infrastructure and our economy, Perlmutter told SpaceNews by email. We need to make sure we are all working together to have the best research which informs the best modeling and forecasting possible.

Although the Senate and House bills are not identical, their intent is very much the same, said a congressional aide who asked not to be identified. Our goal over the coming months is to get together on the same text of the bill. Then, we have a good shot at getting it passed in both the House and the Senate.

Meanwhile, federal agencies are developing and launching new space weather sensors.

The Air Force Space and Missile Systems Center awarded contracts to Applied Technology Associates of Albuquerque New Mexico, and Teledyne Brown Engineering of Huntsville, Alabama, to build prototype Energetic Charge Particle sensors. In 2015, then-Air Force Secretary Deborah Lee James issued a memo calling for future Air Force satellites to include an energetic charged particle sensor. The sensors are designed to improve Air Force space weather models and pinpoint which anomalies are caused by the actions of an adversary versus space weather.

NOAA is developing Space Weather Follow On (SWFO), a satellite destined for Earth-Sun Lagrange Point 1 to house the Naval Research Laboratorys Compact Coronagraph and suite of instruments to measure solar wind. Scheduled for launch in 2024, SWFO is designed to carry on the work of SOHO and NOAAs Deep Space Climate Observatory launched in 2015.

NOAA also plans to send a second Compact Coronagraph into orbit in 2025 on Geostationary Operational Environmental Satellite-U.

The joint European Space Agency-NASA Solar Orbiter launched Feb. 9 to observe the suns poles, outer atmosphere and solar wind.

In 2024, NASA plans to send Interstellar Mapping and Acceleration Probe (IMAP) to Lagrange Point 1 to study particles and radiation streaming from the sun toward Earth. In addition to providing real-time solar wind data, IMAP is designed to host SWFO.

NASA plans to mount another space weather instrument, the Atmospheric Waves Experiment, on the exterior of the International Space Station in 2022 to observe the light in Earths atmosphere called airglow and determine how this combination of forces drives space weather in the atmosphere, Nicola Fox, NASAs Heliophysics Division director, said at AMS.

In 2022, NASA is set to launch the Polarimeter to Unify the Corona and Heliosphere mission to observe and track solar wind leaving the sun as well as coronal mass ejections. The PUNCH mission includes four small satellites.

Three act together to make a widefield imager and the fourth makes a narrow-field imager, Fox said. We are already talking to NOAA about how that can help with real-time aspects of space weather.

Tandem Reconnection and Cusp Electrodynamics Reconnaissance Satellites, a mission designed to ride into orbit as a secondary payload on PUNCH, is focused on gathering information on particles and fields in the region near the North Pole where magnetic field lines curve down toward Earth.

We are making tremendous progress, Thomas Zurbuchen, associate administrator for NASAs Science Mission Directorate, said at AMS. The most important thing is to take the measurements and start driving them toward predictive power in the space weather domain.

This article originally appeared in the March 16, 2020 issue of SpaceNews magazine.

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The Coronavirus Pandemic Shows Us The Importance Of Combatting Climate Change – Forbes

Posted: at 6:29 am

The common fruit fly which lives one to two months, suggesting insignificance has changed the world through medical research, leading to eight Nobel prizes in human genetics and disease prevention breakthroughs. Today an even smaller organism, Coronavirus, is changing the world even more significantly.

And confronting it with the same opportunity for breakthroughs as scientists treated fruit flies could hold the key to solving our greatest challenge climate change.

Of course, all of the coronaviruses impacts sickness, deaths, economic crises have been negative. But, like the scientists who saw something unique in the fruit fly instead of just an unwelcome pest, coronavirus offers us a unique opportunity: visceral lessons in how to approach future crises, and the horrible costs of not doing so.

First among those lessons is taking authoritative warnings seriously, even when that may result in tough decisions. We have been warned repeatedly over the last decade that a pandemic was an existential threat to our way of life. At the end of 2019, when the late Chinese doctor Li Wenliang first reported his alarm over a coronavirus outbreak, authorities detained him for spreading rumors. If they had acted on his warning, the spread in China would have been less severe.

But by January 21, 2020 China had 278 confirmed cases, other countries had 282, and the World Health Organization issued its first coronavirus advisory. Instead of preparing for the virus inevitable spread to the United States, President Donald Trump downplayed the risk, comparing it to a bad case of the flu. Two months later, tens of thousands of Americans have tested positive for the virus and millions more are under shelter-in-place rules, threatening to send the global economy into a devastating tailspin.

Unfortunately, weve consistently made these same mistakes of ignoring scientific warnings when dealing with other global crises, especially climate change. Beginning in June 1988, when climate scientist James Hansen warned Congress that global warming had begun, climate scientists predictions have repeatedly and increasingly warned of impending crises, and how climate change is accelerating faster than expected much like the Coronavirus. Sadly, the government response has ranged from non-existent to lacking.

Thirty years after Hansens warning, President Trump dismissed an official U.S. government assessment of climate changes risks in 2018, saying I dont believe it. As temperatures have risen, so too has the cost of inaction. From 1979 to 2017, the cost of global climate change-related disasters has increased 150%, costing $2.25 trillion, with the U.S. bearing the brunt of the financial pain at $945 billion nearly twice Chinas second-highest total of $492 billion.

Fortunately, in the battle against coronavirus, countries like South Korea that embrace science-based health warnings and act decisively are able to flatten the curve of the coronavirus spread to reduce infections and deaths. But when it comes to climate change, despite global accords such as the Paris Agreement, the world is still struggling to act decisively and in unison.

The Trump administration stands out with its rejection of science-based climate change policy, compounding decades of foot dragging by rolling back and undermining Obama administration efforts to rein in and reduce greenhouse gas emissions from coal, oil, and auto tailpipes. As of the end of 2019, a New York Times analysis identified 95 environmental rules that are being rolled back by the White House.

A key Trump environmental program roll back is expected to be finalized by the end of March. The administration is relaxing the auto greenhouse gas and fuel economy standards that President Obama announced in 2012. The first national program to reduce transportation greenhouse gas emissions, it was based on science, engineering capabilities, business capacities, as well as environmental and health benefits. It would have doubled fuel economy to 54.5 miles per gallon (mpg) by 2025, eliminated 6 billion tons of carbon dioxide, and saved consumers $1.7 trillion at the pump. It appeared the U.S. was finally listening to climate scientists.

But in early 2017 with Trump at the helm, the auto industry, amidst several years of record sales and profits, found an opportunity to renege on its commitment to the standards and asked the White House to relax the Obama administrations standards. After extensive analysis, the U.S. Environmental Protection Agencys scientists and auto engineers had recently re-affirmed the program. But facts were no longer in control of the process.

The final rule targets the standards for the 2021-2026 period. It is widely expected to pull back the standards to 37 mpg and reduce the annual fuel economy improvement to 1.5%, down from the current 5%.

Here is the rub. Transportation is now the fastest growing sector driving increased U.S. greenhouse gas emissions. Even the Obama administrations standards, which the Trump administration is trying to scale back, were never enough to address this gorilla in the room. A landmark study by the National Academy of Science in 2013 calculated that the worlds entire fleet of vehicles in 2025 would have to average around 180 mpg to limit warming to safe levels. As detailed in my book, Driving the Future, if we achieved the original 2025 target and enacted rules to continue the 5% annual improvement curve through 2050, we would only reach 80% of the target required to meet the Intergovernmental Panel on Climate Changes (IPCC) earlier target of 2C target of safe warming and the gap will be even greater to reach the new IPCC target of 1.5C.

The only pathway to reaching the IPCCs targets is transportation electrification. The administration should abandon the new rules they are developing, leave the current rules in place and begin work on the post 2025 standards. The auto industry has four to five year planning horizons and needs policy certainty. The world needs to avoid the scale of disruptions that climate change will bring even if the slow pace is deceiving.

The current coronavirus crisis has produced one near-miracle: The bitterly partisan U.S. Congress and federal government are quickly negotiating emergency legislation to deal with the public health and economic crises. Hopefully, reliance on science-based health measures will now guide the countrys approach to combatting coronavirus. And, while the world awaits the worst yet to come in coronavirus infections and deaths, the lessons from this pandemic could result in an approach to bi-partisan, scientifically driven commitment to combat climate change.

Like the seemingly insignificant fruit fly, confronting greenhouse gas and fuel economy standards could produce outsized breakthroughs on climate change. Like the coronavirus, listening to scientific warnings about climate change before it is too late could prevent outsized public health and economic tragedies.

And no, this is not a dream. The reality of global disruption is staring us all in the face. Blinking is not an option.

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Kallyope Inc. Announces $112M Series C Financing to Support First Clinical Trials and Advance Portfolio of Programs Targeting the Gut-Brain Axis – P&T…

Posted: at 6:29 am

NEW YORK, March 25, 2020 /PRNewswire/ -- Kallyope Inc., a leading biotechnology company focused on identifying and pursuing therapeutic opportunities involving the gut-brain axis, today announced a $112 million Series C financing. This financing will be used to advance its portfolio of programs and the company's first clinical trials, further establishing its leadership in the gut-brain axis field.

All investors from the Series B financing participated in the Series C round, including The Column Group, Lux Capital, Polaris Partners, Euclidean Capital, Two Sigma Ventures, Illumina Ventures, Alexandria Venture Investments, and Bill Gates. New investors include Casdin Capital, Greenspring Associates, and two unnamed leading institutional investors.

"Four years ago, we started our journey to build a preeminent biotech based in New York City as a first-mover in the gut-brain axis space. Now, this Series C financing will enable us to advance multiple programs to clinical development," said Kallyope CEO Nancy Thornberry.

The Series C financing comes after four highly productive years in which Kallyope has built a portfolio of programs directed to novel targets in a wide array of diseases. In support of these programs, the company has established industry-leading capabilities in designing oral small-molecule drugs that selectively target the gut but not the rest of the body.

The company today also announced its lead program targeting satiety circuits for weight loss, with clinical testing expected to begin later this year. A second program targeting gut barrier function with potential relevance for inflammatory bowel disease (IBD) and several other diseases is anticipated to enter the clinic soon after. In addition, the company continues to advance a broad portfolio of programs for gastrointestinal, CNS, and inflammatory disorders.

"Kallyope pursues programs where the company's platform provides an edge over other approaches and where we have an opportunity to deliver major clinical benefits rather than incremental improvements over current treatments. We are targeting neural and hormonal circuits, including novel vagal circuits, involved in a broad array of physiology and disease," said Thornberry.

"Kallyope has made significant progress since the company's inception in late 2015. Its platform is enabling a mechanistic understanding of the gut-brain axis, which in turn has revealed new, actionable biology that the company is now exploring in several promising programs. I believe that Kallyope's platform and rigorous approach to identifying, characterizing, and targeting gut-brain circuits with gut-restricted small molecules has greatly increased its odds of success in clinical studies," said Kallyope co-founder and board member Tom Maniatis, Ph.D.

About Kallyope Inc.

Kallyope, headquartered at the Alexandria Centerfor Life Science in New York City, is a biotechnology company dedicated to unlocking the therapeutic potential of the gut-brain axis. The company's cross-disciplinary team integrates advanced technologies in sequencing, bioinformatics, neural imaging, cellular and molecular biology, and human genetics to provide an understanding of gut-brain biology that leads to transformational therapeutics to improve human health. The company's founders are Charles Zuker, Ph.D., Lasker Award winner Tom Maniatis, Ph.D., and Nobel laureate Richard Axel, M.D. For more information visitwww.kallyope.com.

Contact

Morgan Warners (202) 337-0808mwarners@gpg.com

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Kallyope Inc. Announces $112M Series C Financing to Support First Clinical Trials and Advance Portfolio of Programs Targeting the Gut-Brain Axis - P&T...

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IN CONSERVATION: DR. JEFF STROVEL, CEO of VERALOX THERAPEUTICS – BioBuzz

Posted: at 6:29 am

Veralox Therapeutics is a startup currently headquartered at the Frederick Innovation Technology Center, Inc. (FITCI) in Frederick, Maryland. As a small molecule therapeutics company focused on developing medicines targeting the arachidonic acid pathway, they have two drug products in development for the treatment of a rare blood disorder known as heparin-induced thrombocytopenia and thrombosis and Type 1 Diabetes.

The BioBuzz team recently caught up with Veraloxs CEO Dr. Jeff Strovel, who spoke about the genesis of Veralox, its advancing pipeline, the companys recent milestones and its outlook for the future.

Tell our audience a bit about the founding of Veralox Therapeutics

Ill never forget the day a trusted colleague, David Maloney, called me when I was driving back to my office. He told me he had been working to develop a clinical candidate that selectively targeted the arachidonic acid (AA) pathway through inhibition of a protein called 12-Lipoxygenase. He believed the program was ready to be brought into a company, and there was potential for broad therapeutic utility of the inhibitor, which was intriguing. I have heard a lot of drug program pitches and usually get a sense of where the holes are pretty quickly. In this case, we talked for 30 minutes and afterward I knew this program was ready for company formation. Our first product in development, VLX-1005, is for treatment of the rare blood disorder heparin-induced thrombocytopenia & thrombosis (HITT) which also happens to be the same blood disorder that nearly killed my father years ago. We started the company in 2017 along with Daves colleague from the NCATS (National Center for Advancing Translational Sciences) at the NIH, Matt Boxer, with our rare disease blood disorder drug as our lead candidate. I feel the stars really aligned for us and we have been charging ahead ever since.

Dave, in collaboration with several other partners, led the development of our lead candidate, VLX-1005 while he was a chemistry Group Leader at NCATS.

What is your life science background and what did you do prior to co-founding Veralox?

I hold a Ph.D. in Human Genetics from the University of Maryland School of Medicine. I then went on to be a clinical cytogenetics fellow at the National Cancer Institute Department of Pathology at the NIH. While at NIH I developed a drug target ID platform that got the interest of companies doing cancer drug discovery and I eventually joined a company called Avalon, where I worked in drug discovery and development for about 9 years and served my last two years as Head of Translational Oncology. I learned a great deal about the drug discovery and development process during my time there.

After Avalon I was part of a group of local life science veterans that co-founded the Clinical Research Organization (CRO) Noble Life Sciences. The CRO also served as a kind of incubator for new companies. We incubated several new companies like Neximmune and Convergene Pharmaceuticals, where I served as Chief Scientific Officer and President from 2011 to 2015 before taking on the Chief Executive role from 2015 to 2017. During that time I had met and worked with Dave and I left Convergene to start Veralox with him and our other co-founder Matthew Boxer.

What is Veraloxs lead drug candidate, what disease does it target and where does it sit in the development process?

Our lead drug candidate is for the treatment of patients with HITT, which is a rare, life-threatening disease caused by an immune reaction to Heparin. This drug candidate is a small molecule product that inhibits the 12-lipoxygenase (12-LOX) enzyme which produces 12-HETE. Both 12-LOX and 12-HETE are implicated in numerous diseases. The only FDA-approved drug for HIT/HITT, argatroban, doesnt treat the underlying cause of the disease and has significant potential side effects leaving a significant unmet need.

There are approximately 50,000 people that will be diagnosed with HIT/HITT in the US this year but the number of those treated for this disease is approximately 150,000 annually. HIT takes time to diagnose and doctors cant wait for a definitive diagnosis to make treatment decisions. If patients are suspected of having the disease they must be treated immediately given the risk for deadly thrombosis. The current approved treatment, argatroban, can cause major bleeds which in and of itself can lead to death in these patients. Better drugs are needed that address the pathology of the disease without contributing potentially lethal side effects.

Our HIT treatment inhibits the underlying pathophysiology of HITT to halt the aberrant immune response driving the disease and essentially offering the potential of a curative modality.

Weve been strategically focused on HIT/HITT, but our approach has potential for a number of other indications. For our HIT/HITT drug candidate, weve submitted our Orphan Drug application and our pre-IND (Investigational New Drug) briefing book to the FDA; we expect to have our GMP material soon and to start GLP toxicology studies in the second quarter of this year. Our team expects to submit the IND for our lead candidate by the end of 2020. We are also co-developing a small molecule, orally administered drug product. We plan to advance this candidate for the treatment for Type 1 Diabetes, which is about a year behind our HIT/HITT clinical candidate.

What are some recent Veralox funding milestones and what are the companys next funding steps?

Weve had success raising capital from grants and venture capital groups. Veralox received about 300K from NIHs Small Business Innovation Research (SBIR) Fund, Phase I, and $300K from NIHs Small Business Technology Transfer (STTR) Fund. We also recently closed a $5.4M seed round that was co-led by Sanofi Ventures and the JDRF T1D Fund and included participation from the VTC Innovation and VTC Seed Fund, the Maryland Momentum Fund, the University of Vermont Health Network and TEDCO.

We are currently raising Series A funding to support clinical development of VLX-1005 for HITT and advance our second drug product for treatment of Type 1 Diabetes into clinical trials. Weve initiated discussions with several large venture capital firms all of which could lead our funding round and have the full support of our current investors in the process.

What can you tell us about your experience at FITCI and as a startup in the Frederick life science ecosystem?

Ive had experiences with other incubators, some of which no longer exist, but theres no comparison to FITCI. They offer great support to emerging companies that desperately need it at the early stages. I have been at other incubators that give you a physical place to do business, but FITCI really keeps an eye on you. The FITCI team helps you solve problems and they offer quarterly meetings for the CEOs to meet with groups of successful business people who can offer advice and provide mentorship for those in need. These mentors might not be in therapeutics but theyve done this before and can really help.

I really love Frederick because, to me, the area is just like a startup. Its really going somewhere. FITCI has two locations, but whenever we meet with people, we use the Market Street location in downtown Frederick. Its a nice face to show off to people from outside of the area. Our time at FITCI is nearing its end because of our funding success, but when we graduate from the incubator, the plan is to stay in Frederick. Weve already been looking at a few places.

What are some near term challenges for Veralox and where do you see the company being in three to five years?

Our biggest challenge is developing a completely novel first-in-class product. Theres no blueprint or recipe one can exactly follow, and it is challenging. This is, however, what the Veralox team is trained to do and we are well prepared and excited by the challenge and promise of developing a new treatment that can drastically improve and perhaps save the lives of people suffering from a terrible disease.

Were also currently looking to hire a Chief Medical Officer soon. Finding the best talent is always a challenge, but I know well be able to find the right person.

In three to five years, our goal is to have several products in the clinic and possibly an approved HIT/HITT drug on the market. Veralox has the potential to become an anchor life science company in the region. But for now, Id like Veralox to be known as the company that did what they said theyd do were community-minded and will continue to work hard to make a difference in the lives of patients with debilitating diseases.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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IN CONSERVATION: DR. JEFF STROVEL, CEO of VERALOX THERAPEUTICS - BioBuzz

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Patients with Severe Forms of Coronavirus Disease Could Offer Clues to Treatment – Howard Hughes Medical Institute

Posted: at 6:25 am

A new international project aims to enroll 500 COVID-19 patients to search for genetic mutations that make some people more vulnerable to severe infection.

HHMI scientists are joining many of their colleagues worldwide in working to combat the new coronavirus.Theyre developing diagnostic testing, understanding the viruss basic biology, modeling the epidemiology, and developing potential therapies or vaccines. Over the next several weeks, we will be sharing stories of some of this work.

Hundreds of clinicians worldwide are banding together in an effort to study some types ofseverecases of the new coronavirus disease.

The project, led by Howard Hughes Medical Institute (HHMI) Investigator Jean-Laurent Casanova at The Rockefeller University, seeks to identify genetic errors that make some younger patients especially vulnerable to the virus that causes COVID-19, the infectious respiratory illness also known as coronavirus disease 2019.

Casanova aims to enroll 500 patients internationally who meet three broad criteria: theyre less than 50 years old, have been diagnosed with COVID-19 and admitted to an intensive care unit, and have no serious underlying illnesses, such as diabetes, heart disease, or lung disease.

By studying these patients' DNA, scientists may pinpoint genetic mutations that make some people more susceptible to infection. Such information could one day help doctors identify people who are most at risk of developing severe coronavirus disease, says Casanova, a pediatrician at Rockefeller. It could also offer clues for scientists searching for new therapeutics. For example, if patients cells arent making enough of a particular molecule, doctors may be able to offer a supplement as treatment.

Were going to try to find the genetic basis of severe coronavirus infection in young people.

Jean-Laurent Casanova, HHMI Investigator at The Rockefeller University

That day may still be years away. This is not a short-term effort, Casanova says. Some scientists have hypothesized that COVID-19 might be a seasonal illness, with infections ebbing in the spring and summer, and then returning in the fall. But Casanovas team is optimistic. They have already begun enrolling patients and have started sequencing their exomes spelling out all of the DNA letters in every gene in a persons genome. Were going to try to find the genetic basis of severe coronavirus infection in young people.

Late last year, when the first coronavirus infections began cropping up in China, Casanova started reaching out to his colleagues there. Though the most severe cases seemed to concentrate among older adults and those with other conditions, Casanova was interested in the outliers kids and young adults hit hard by the illness who didnt have any of the usual risk factors, such as age or underlying illness.

His team kicked off a new project to study these mysterious cases, and in January just weeks after the Wuhan outbreak began enrolling patients. Clinicians mailed patient blood and DNA to his lab, and researchers there and elsewhere began processing samples the first steps needed for scientists to peer into patients genomes. Now, the project is global, and Casanova is collaborating with scientists and healthcare workers from Europe to Africa, Asia, and Oceania.

We will recruit children and adults <50 yo without risk factor admitted to ICU for idiopathic #COVID19. We will test the hypothesis that they carry inborn errors of immunity to this virus. Please refer patients to @casanova_lab and please RT. pic.twitter.com/DXPoFKieEy

Hunting for the genetic underpinnings of severe infectious diseases is nothing new for Casanovas team. What were doing with coronavirus is what my lab has been doing for 25 years with other infections, he says.

They look for weak spots in peoples immune systems small genetic changes that make people more vulnerable to disease. His group has previously searched the genomes of patients infected with viruses, bacteria, fungi, and even parasites. The infection closest to COVID-19 his team has studied is severe influenza pneumonitis, for which theyve discovered three genetic links. Theyve also identified specific genetic errors that can predispose patients with herpes to viral encephalitis. And theyve found that children with mutations in an immunity gene called IFN-gamma are vulnerable to the bacteria that cause tuberculosis. These children make low levels of the IFN-gamma protein, which is critical for fighting off bacterial infections.

Casanovas team has put these findings to use clinically. For example, the researchers have shown that tuberculosis patients with these genetic errors can benefit from treatment with IFN-gamma. Hes hoping to identify problematic genes in patients with severe coronavirus infection that can bring similar clinical gains. These genes could tell scientists which cellular defenses are crucial for warding off COVID-19 and pave the way for understanding whether such defenses are derailed in older adults or patients with an underlying medical condition.

In the US and around the world, severe coronavirus disease seems to hit older patients hardest, though scientists have reported some country-to-country variation. As of March 24, more than 44,000 confirmed and presumptive positive cases have been reported in the US. Fatality has been highest in people over 85 years old, according to a recent report from the Centers for Disease Control and Prevention (CDC). Though young people may be more susceptible than scientists once suspected,the older you are, the higher the likelihood you have a severe form of the disease, Casanova says.

Last week, Rockefeller closed all labs except those working on the coronavirus, and Casanova whittled his team to a skeleton crew of about eight people down from 35 who rotate so there is only one person per room at a time. He and his lab members are following CDC recommendations, and taking protective measures to keep themselves and others safe, including social distancing, washing hands, and disinfecting surfaces. Theyve also taken to Twitter to get the word out about their work. A tweet posted from Casanovas lab last week about recruiting new patients to their study has since been retweeted more than 400 times.

Soon, theyll be testing their genetic theory on a pandemic thats occurring in real time. Im grateful weve been able to start this new project so quickly, he says. God willing, it will be of clinical usein two or three years.

Follow the Casanova lab on Twitter (@casanova_lab) to learn the latest about their work. Doctors interested in enrolling patients in the study can contact Jean-Laurent Casanova at jean-laurent.casanova@rockefeller.edu.

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Clean your phone ‘at least once a day,’ medical experts say. Here’s howwithout damaging the screen – CNBC

Posted: at 6:25 am

The coronavirus pandemic has led to a spike in sales of products, includinghand sanitizer, toilet paper and pasta.

More than likely, however, there is no need to buy too much of anything, Dr. Amy Edwards, a pediatric infectious disease specialist at University Hospitals who works with the UH Roe Green Center for Travel Medicine & Global Health,tells Grow. "My advice would be to be vigilant, but calm, and not to panic."

To protect yourself from coronavirus, health professionals say the most important thing to do is wash your hands regularly. Don't forget to clean your phone regularly, too. "I clean my phone at least once a day," says Edwards. She advises others to do the same and many medical experts agree.

"It's often said that your phone is like a third hand because you're constantly touching it," says cleaning influencer Melissa Maker.

People take their phones out to eat, on the train, and to the bathroom. As a result, cellphones carry more than 17,000 bacterial gene copies each, according to a 2017 study. The report concluded that this "may play a role in the spread of infectious agents."

More from Grow:What to buy when your grocery store is out of pasta, beans, juiceHow a hairdresser plans to stretch her $4,000 savings while out of work3 smart ways to improve your finances while you're at home

Cleaning phones daily, at least, is smart, says Edwards."Certainly, if you are letting a lot of people use your phone, you would want to clean it to help prevent spread."

It's often said that your phone is like a third hand because you're constantly touching it.

Melissa Maker

Cleaning Influencer

If you want to clean your phone effectively, Maker says not to use a Lysolwipe or disinfectant wipe, as it may strip the coating of your phone over time. "The chemicals that are used in those disinfectant wipes are not meant to be used on electronics," she says.

Until recently, Apple advised against the use oftraditional cleaning products or compressed air.But earlier this month, Apple updated its instructions to say you can clean your phone with disinfectant wipes, as long as you wipe gently and avoid getting any liquid in charging ports.

GuidelinesforAndroidhandsets still advise steering clear of disinfectant wipes.

One alternative: Cleaning wipes that are specifically made for electronic devices. A 210-pack of individually wrapped lens- and screen-cleaning wipes is $16.99 on Amazon right now.

Maker suggests using a microfiber cloth. "Microfiber has the ability to pick up bacteria," Makers says. "Then you can launder the microfiber cloth."A six-pack of microfiber cleaning clothes is $9.99 on Amazon right now.

A damp microfiber cloth can remove microorganisms including viruses and bacteria and is more effective than a cotton rag, microbiologist Kristen Gibson told the The Wall Street Journal. It won't damage your phone the way a Lysol wipe might, either.

You can also pair onewith a homemade cleaner that is equal parts water and rubbing alcohol. Dip the cloth in the mixture, make sure it's not excessively wet, and then wipe down all parts ofyour phone. This will serve as an effective disinfectant.

And, of course, make sure you frequently wash your hands.

The article Clean Your Phone At Least Once a Day, Says Infectious Disease Specialist originally appeared on Grow by Acorns + CNBC.

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Castle Creek Biosciences Announces $75 Million Investment to Advance Development of Multiple Gene Therapy Candidates for Rare Diseases – Yahoo Finance

Posted: at 6:25 am

- Leveraging proprietary technology and manufacturing platform to develop transformativegene therapies for multiple rare diseases with high unmet needs -

- Pipeline led by Phase 3 gene therapy candidate for treatment of recessive dystrophic epidermolysis bullosa (RDEB), with a BLA filing targeted for 2021 -

- Backed by world-class group of biotech operators and investors -

EXTON, Pa., March 25, 2020 (GLOBE NEWSWIRE) -- Castle Creek Biosciences, Inc., a privately held, late-stage gene therapy company, announced that it has received a new investment of $75 million to support the advancement of its clinical development pipeline. Castle Creek Biosciences is a portfolio company of Paragon Biosciences, which led the $55 million equity investment from Fidelity Management & Research Company and Valor Equity Partners, along with a $20 million venture loan from Horizon Technology Finance Corporation (HRZN).

Castle Creek Biosciences is leveraging its proprietary technology platform and commercial-scale manufacturing infrastructure to develop personalized gene therapies for rare diseases with high unmet needs. The company plans to use the funding to advance and expand its gene therapy pipeline, led by the Phase 3 clinical development of FCX-007 (NCT04213261), its gene therapy candidate for the treatment of RDEB. It will also use the funding to expand its current good manufacturing practices (cGMP) infrastructure located in the greater Philadelphia region.

Clinical results from the ongoing Phase 1/2 clinical trial for FCX-007 continue to show positive trends in safety and wound healing in RDEB patients. Current data from this clinical trial were presented at the inaugural World Congress on Epidermolysis Bullosa held in London during January of 2020. FCX-007 was administered to 10 non-healing chronic wounds of which eight achieved complete wound closure 12 weeks post-administration (80%) vs. no wound closure in intra-patient, matched non-treated wounds (0%). FCX-007 continues to be well tolerated up to 52 weeks post administration.

We are proud to have the strategic support of world-class investors whose impact enables our efforts to transform the lives of patients and the future of medicine, said John Maslowski, Chief Executive Officer of Castle Creek Biosciences. We are steadfast in our commitment to the epidermolysis bullosa community and will continue to keep patients, caregivers and clinicians informed on the progress of our current programs, including FCX-007 and diacerein topical ointment, while we expand the scope of our gene therapy platform.

Castle Creek Biosciences is led by a strong executive leadership team with a proven record of developing innovative and potentially life-changing treatments for conditions with the greatest medical need, said Jeffery Aronin, Chairman and Chief Executive Officer of Paragon Biosciences. As investors, we are excited by the progress that the team has made and are committed to growing the Castle Creek Biosciences platform to address multiple rare genetic diseases.

About Castle Creek Biosciences, Inc. Castle Creek Biosciences is a privately held company that develops and commercializes gene therapies for patients with rare and serious genetic diseases. The companys lead gene therapy candidate, FCX-007, is being evaluated for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the most severe and debilitating form of epidermolysis bullosa (EB). The company is also advancing clinical research evaluating a diacerein topical ointment, CCP-020, for the treatment of epidermolysis bullosa simplex (EBS) and other forms of EB. In addition, Castle Creek Biosciences is developing FCX-013, a gene therapy for the treatment of moderate to severe localized scleroderma. Castle Creek Biosciences is a portfolio company of Paragon Biosciences. For more information, visit castlecreekbio.com or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon BiosciencesParagon is a life science innovator that invests in, builds, and advises bioscience companies. Our mission is to serve patients living with severe medical conditions which do not yet have adequate treatments. Paragons portfolio of independently-run bioscience companies focus on biopharmaceuticals, AI-enabled life science products, and advanced treatments such as cell and gene therapies. We help people live longer, healthier lives. For more information, please visit: ParagonBioSci.com.

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Bridging the gap study sequences Asian genomes to diversify genetic databases – University of Virginia The Cavalier Daily

Posted: at 6:25 am

Though the number of human genomes sequenced continues to rise rapidly since the completion of the Human Genome Project a scientific endeavor spanning multiple decades and countries aimed at detailing human DNA in 2003, less than 10 percent of those genomes to date correspond to individuals of Asian descent. The GenomeAsia 100K Project, a non-profit consortium, seeks to change this lack of knowledge surrounding a major portion of the worlds ethnicities. The conglomeration of researchers and private sector executives from around the world from Seoul, South Korea to the University plans to add 100,000 novel genomes from individuals of Asian ethnicity to new open-access databases.

Academic institutions and private sector companies came together in 2016 to launch the GenomeAsia 100K Project. While the research organization MedGenome and Nanyang Technological University in Singapore originally founded the non-profit consortium, representatives from other businesses and schools including Genentech, Macrogen and the University of California, San Francisco have joined the association.

Since genome sequencing can reveal the unique characteristics of each persons genetic material, it can help determine a persons ancestry and the propensity for certain medical conditions. According to GenomeAsia 100K, Asians constitute nearly half of the worlds population, and the distinct ethnicities and communities offer a relatively untapped repository of genetic diversity. The project hopes to provide new insights into inherited diseases as well as those caused by a combination of genetic and environmental factors.

Aakrosh Ratan, assistant professor of public health sciences and researcher for GenomeAsia 100K, explained that in particular, the information the initiative collects may help develop medical treatments based on peoples specific genetic makeup, instead of relying on traditional general treatments that may not target the unique root cause of each patients form of a disease.

The goal of precision medicine is to tailor treatment towards a persons genetic background, and that dream cannot be realized until you have the proper reference databases, Ratan said.

Mutations in humans DNA sequences lead to different copies of the same gene within a person and amongst ethnicities. These different versions of a gene can act as markers of diseases that are inherited or influenced by genetic makeup. For example, the disorder sickle cell anemia is caused by the change of a single point in the DNA sequence. When someone is born with copies of this particular gene from both parents contain the mutation, he or she will suffer from often debilitating pain resulting from red blood cells that cannot effectively transport oxygen.

Ratan explained that genome sequencing can highlight mutations in a persons DNA that may cause illnesses such as sickle cell anemia.

One of the ways we identify the mutations that drive a rare disease is by identifying the mutations and then prioritizing those mutations based on their prevalence in healthy populations, Ratan said. With the medical datasets we have compiled, we can actually improve such analyses for patients of Asian descent.

As of December 2019, the GenomeAsia 100K Project has completed the analysis of 1,739 genomes from 219 populations and 64 countries worldwide. Preliminary findings appeared that same month in the scientific journal Nature. The paper concluded that the sample provided a reasonable framework for sequencing practices and studying the history and health of Asian populations. Ratan and his lab at the University supervised the identification and contributed to the analysis of these genetic variants.

Once the 100,000 genomes have been collected and sequenced, the data will be publicly available as a controlled dataset. As a result, experts investigating topics from heart disease to human evolution can easily access the genome sequences.

One of the real gaps in human genetics studies of disease has been the underrepresentation of non-Europeans, Charles Farber, associate professor of public health sciences, said in an email to The Cavalier Daily. The work of the GenomeAsia 100K Consortium provided critical insight into the extent and nature of genome variation in individuals of Asian ancestry and will be critical in making disease genetic studies more inclusive of all global populations.

Ani Manichaikul, assistant professor of public health sciences in the Center for Public Health Genomics, expressed enthusiasm for the GenomeAsia 100K Project. She claimed that the additional genetic information could augment her research as part of the Multi-Ethnic Study of Atherosclerosis, a cardiovascular disease where fatty deposits accumulate and potentially block arteries. The study currently focuses on Caucasian, African American, Hispanic and Chinese American individuals.

The GenomeAsia project is very useful because there are some instances where particular genetic variants are only observed in particular genetic groups, Manichaikul said. Those markers can be unique to those sequenced through the project, which means we would not have necessarily have observed those particular variants otherwise.

Manichaikul also suggested that expanding existing repositories of hereditary statistics would improve methods of assigning people risk scores for diseases based on their DNA. The National Human Genome Research Institute describes polygenic risk score, which indicates a persons likelihood of certain diseases based on the presence of mutations known to be associated with a given disorder. Companies such as 23andMe have started to provide consumers with this metric, but without a comprehensive database of genomes from different populations, score reliability can decrease.

Since indicators of genetically-linked conditions often appear in certain alleles, or different versions of a gene, knowing whether one has a disease marker can help patients take preventative measures if need be. However, in the absence of comprehensive information on the range of disease markers that appear in different ethnicities, whole populations may lack the potential benefits of this burgeoning healthcare statistic.

The only way we can create risk prediction models that are accurate across populations is if we also have corresponding databases available with individuals that represent that diversity, Manichaikul said.

Following the findings in the preliminary study, GenomeAsia 100K Project collaborators will continue to sequence more genomes of Asian individuals. The hope is that, once researchers have access to the data, insights from 100,000 genomes will drive the development of new therapeutic strategies that will benefit people around the world.

I would like more researchers to have access to this data, Ratan said. This is a resource. Were working to establish these reference datasets, and we would definitely like them to be used.

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Coronavirus Treatment: Hundreds of Scientists Scramble to Find One – The New York Times

Posted: at 6:25 am

Working at a breakneck pace, a team of hundreds of scientists has identified 50 drugs that may be effective treatments for people infected with the coronavirus.

Many scientists are seeking drugs that attack the virus itself. But the Quantitative Biosciences Institute Coronavirus Research Group, based at the University of California, San Francisco, is testing an unusual new approach.

The researchers are looking for drugs that shield proteins in our own cells that the coronavirus depends on to thrive and reproduce.

Many of the candidate drugs are already approved to treat diseases, such as cancer, that would seem to have nothing to do with Covid-19, the illness caused by the coronavirus.

Scientists at Mount Sinai Hospital in New York and at the Pasteur Institute in Paris have already begun to test the drugs against the coronavirus growing in their labs. The far-flung research group is preparing to release its findings at the end of the week.

There is no antiviral drug proven to be effective against the virus. When people get infected, the best that doctors can offer is supportive care the patient is getting enough oxygen, managing fever and using a ventilator to push air into the lungs, if needed to give the immune system time to fight the infection.

If the research effort succeeds, it will be a significant scientific achievement: an antiviral identified in just months to treat a virus that no one knew existed until January.

Im really impressed at the speed and the scale at which theyre moving, said John Young, the global head of infectious diseases at Roche Pharma Research and Early Development, which is collaborating on some of the work.

We think this approach has real potential, he said.

Some researchers at the Q.B.I. began studying the coronavirus in January. But last month, the threat became more imminent: A woman in California was found to be infected although she had not recently traveled outside the country.

That finding suggested that the virus was already circulating in the community.

I got to the lab and said weve got to drop everything else, recalled Nevan Krogan, director of the Quantitative Biosciences Institute. Everybody has got to work around the clock on this.

Dr. Krogan and his colleagues set about finding proteins in our cells that the coronavirus uses to grow. Normally, such a project might take two years. But the working group, which includes 22 laboratories, completed it in a few weeks.

You have 30 scientists on a Zoom call its the most exhausting, amazing thing, Dr. Krogan said, referring to a teleconferencing service.

Viruses reproduce by injecting their genes inside a human cell. The cells own gene-reading machinery then manufactures viral proteins, which latch onto cellular proteins to create new viruses. They eventually escape the cell and infect others.

In 2011, Dr. Krogan and his colleagues developed a way to find all the human proteins that viruses use to manipulate our cells a map, as Dr. Krogan calls it. They created their first map for H.I.V.

That virus has 18 genes, each of which encodes a protein. The scientists eventually found that H.I.V. interacts, in one way or another, with 435 proteins in a human cell.

Dr. Krogan and his colleagues went on to make similar maps for viruses such as Ebola and dengue. Each pathogen hijacks its host cell by manipulating a different combination of proteins. Once scientists have a map, they can use it to search for new treatments.

In February, the research group synthesized genes from the coronavirus and injected them into cells. They uncovered over 400 human proteins that the virus seems to rely on.

The flulike symptoms observed in infected people are the result of the coronavirus attacking cells in the respiratory tract. The new map shows that the viruss proteins travel throughout the human cell, engaging even with proteins that do not seem to have anything to do with making new viruses.

One of the viral proteins, for example, latches onto BRD2, a human protein that tends to our DNA, switching genes on and off. Experts on proteins are now using the map to figure out why the coronavirus needs these molecules.

Kevan Shokat, a chemist at U.C.S.F., is poring through 20,000 drugs approved by the Food and Drug Administration for signs that they may interact with the proteins on the map created by Dr. Krogans lab.

Dr. Shokat and his colleagues have found 50 promising candidates. The protein BRD2, for example, can be targeted by a drug called JQ1. Researchers originally discovered JQ1 as a potential treatment for several types of cancer.

On Thursday, Dr. Shokat and his colleagues filled a box with the first 10 drugs on the list and shipped them overnight to New York to be tested against the living coronavirus.

The drugs arrived at the lab of Adolfo Garcia-Sastre, director of the Global Health and Emerging Pathogens Institute at the Icahn School of Medicine at Mount Sinai Hospital. Dr. Garcia-Sastre recently began growing the coronavirus in monkey cells.

Over the weekend, the team at the institute began treating infected cells with the drugs to see if any stop the viruses. We have started experiments, but it will take us a week to get the first data here, Dr. Garcia-Sastre said on Tuesday.

The researchers in San Francisco also sent the batch of drugs to the Pasteur Institute in Paris, where investigators also have begun testing them against coronaviruses.

If promising drugs are found, investigators plan to try them in an animal infected with the coronavirus perhaps ferrets, because theyre known to get SARS, an illness closely related to Covid-19.

Even if some of these drugs are effective treatments, scientists will still need to make sure they are safe for treating Covid-19. It may turn out, for example, that the dose needed to clear the virus from the body might also lead to dangerous side effects.

This collaboration is far from the only effort to find an antiviral drug effective against the coronavirus. One of the most closely watched efforts involves an antiviral called remdesivir.

In past studies on animals, remdesivir blocked a number of viruses. The drug works by preventing viruses from building new genes.

In February, a team of researchers found that remdesivir could eliminate the coronavirus from infected cells. Since then, five clinical trials have begun to see if the drug will be safe and effective against Covid-19 in people.

Other researchers have taken startling new approaches. On Saturday, Stanford University researchers reported using the gene-editing technology Crispr to destroy coronavirus genes in infected cells.

As the Bay Area went into lockdown on Monday, Dr. Krogan and his colleagues were finishing their map. They are now preparing a report to post online by the end of the week, while also submitting it to a journal for publication.

Their paper will include a list of drugs that the researchers consider prime candidates to treat people ill with the coronavirus.

Whoever is capable of trying them, please try them, Dr. Krogan said.

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AZ adds gene silencing tech to pipeline with Silence Therapeutics deal – – pharmaphorum

Posted: at 6:25 am

AstraZeneca has announced a collaboration with UK biotech Silence Therapeutics to develop gene silencing drugs for cardiovascular, renal, metabolic and respiratory diseases.

The deal could see AZ adding small interfering RNA (siRNA) technology to its pipeline in one of its main areas of research.

Silence Therapeutics says its technology can selectively inhibit any gene in the genome, specifically silencing the production of disease-causing proteins.

The UK biotech says it has achieved an additional level of accuracy by delivering its therapeutic RNA molecules exclusively to target cells.

siRNA is a technology that showed much promise after biologists Andrew Fire and Craig Mello received the Nobel Prize in Physiology or Medicine for discovering the technology in 2006.

But in 2010 it became apparent that it was harder to convert into a working therapy because of the challenge of delivering therapeutic RNA molecules to target tissues and big pharma quickly lost interest.

But companies like Silence have managed to overcome this hurdle and its rival Alnylam made history in 2018 when its siRNA drug Onpattro was approved by the FDA to treat hereditary transthyretin (hATTR) amyloidosis, which causes the build-up of amyloid protein in nerves and organs.

AZ will pay $60 million up front and invest $20 million in Silence, and will pay up to $400 million in milestone payments plus tiered royalties.

The companies expect to work on five targets within the first three years of the collaboration, and AstraZeneca has the option to extend it to a further five targets.

Silence has technology that can inhibit liver-expressed gene targets and the companies will collaborate to find siRNA molecules to other tissues including the heart, kidney and lung.

The UK biotech will design SiRNA molecules against gene targets selected by AZ, and will manufacture material to support toxicology and phase 1 clinical studies.

AZ and Silence will collaborate during the discovery phase, while AZ will lead clinical trials and marketing.

Silence will have the option to negotiate for co-development of two drugs of their choice starting from phase 2.

AZ will pay an option fee of $10 million for each selected target when a drug candidate is nominated, and Silence could receive up to $140 million in development milestones, and up to $250 million in market milestones.

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