Daily Archives: January 27, 2022

Opinion: Is There a Place For Spirituality in Space Science? – Undark Magazine

Posted: January 27, 2022 at 11:55 pm

It wasnt just that he mentioned a religious holiday. After all, NASA Administrator Bill Nelson wasnt the only person to observe, following the successful launch of the James Webb Space Telescope last month, that the long-awaited feat had occurred on Christmas Day. Rather, Nelsons comments raised eyebrows for their spiritual tone.

Its significant that we had the delays and it kept us all the way to today, Christmas Day, Nelson said in a video released by NASA shortly after the launch. He went on to quote a passage from Psalm 19: The heavens declare the glory of God. The firmament shows his handiwork.

To some viewers especially those who believe religion and science are incompatible the very mention of a religious text seemed to undercut the messaging of scientific achievement. The suggestion that the telescope served a Christian purpose, or that its use would reinforce a Christian worldview, also seemed to belie a commitment to inclusivity in science that NASA has claimed to value. (The agency is still reeling from the controversy over its decision to name the telescope after James Webb, a man alleged to have been complicit in the persecution of LGBTQ government workers.)

These are all valid concerns. But its also worth remembering that Nelsons biblical references follow in a long tradition of religious rhetoric in the U.S. space program. Theres a tendency to flatten this history to imagine that religious language is and always has been inappropriate in the scientific discourse. But one needs only look back a few decades to find a time when comments like Nelsons were not only acceptable in the American space culture they were a central part of Americas science identity.

From the 1950s, the United States was embroiled in a decades-long rivalry with the U.S.S.R. known as the Space Race a competition that turned the technological and military practicalities of space exploration into a sort of proxy battle for cultural, political, and economic validation. Each nations scientific successes were interpreted as triumphs of one national ideology over the other. Among those warring ideologies were the nations sharply contrasting attitudes toward religion.

The U.S.S.R. had officially embraced atheism (though some Soviet citizens were people of faith). In her recent history of Soviet atheism, Victoria Smolkin describes how Soviet leaders and cosmonauts used their victories in the Space Race as occasions to wave a banner of antipathy toward religion. During a 1962 visit to the U.S., Smolkin writes, Soviet cosmonaut German Titov, the second person in space, proclaimed his atheism, remarking that he had not seen God or angels during his 17 orbits of Earth. Later Soviet leader Nikita Khrushchev similarly joked to American reporters about Gods failure to show up in space. The brash rejection of God served to advance the Soviet effort to solidify state atheism and defuse religions threat to state authority.

But the Soviet Unions dismissal of religion also stirred a backlash on the other side of the Iron Curtain. In fields ranging from evolutionary biology to cosmology, American scientists criticized the ideological dogmatism of Marxism, claiming that it impaired free scientific inquiry. Whereas the Soviet regime was totalitarian and oppressive, the American scientific establishment, by embracing religious tolerance, projected an image of openness. Opposed to the strict atheism of the Soviets but wary of the perceived anti-science attitude of fundamentalist Christians, the American scientific establishment staked out a middle ground of respectable, generic but still Christian-leaning religiosity.

As public figures as well as scientists, NASA astronauts were frequently seen as exemplifying this milquetoast religious identity. Some astronauts were explicit about their own Christianity; others were more vague about the spirituality they experienced in the stars. Neil Armstrong, though he considered himself a deist, was nonetheless looked up to as a Christian role model who fulfilled a divine promise that humanity would someday reach the stars.

On Christmas Eve in 1968, the crew of Apollo 8 broadcast themselves from lunar orbit reading from the opening passages of Genesis as the Sun rose above the Moons horizon: In the beginning, God created the heaven and the earth. And God said, Let there be light: and there was light. The juxtaposition of those words with images of the lunar sunrise seemed to symbolize the convergence of religious and scientific values.

The Christmas Eve reading prompted Madalyn Murray OHair, the founder of the organization American Atheists, to file a lawsuit against NASA, arguing that the act abridged their First Amendment rights. But the lawsuit failed, and since then the tradition of astronauts expressing their personal faith, carrying objects of religious significance among their personal effects, even celebrating holidays in space, has largely been permitted and even incorporated into NASAs public outreach. American Presidents including John F. Kennedy, Lyndon B. Johnson, and Ronald Reagan all used religious language when talking about the Space Program, often with implicit or explicit criticism of the Soviets. Ultimately, NASA, American politicians of both parties, and the wider U.S. public created a narrative that Americas religiosity had helped the country succeed in the Space Race over its godless rival. This religiosity was effective in part because it avoided the messy specifics that might have created friction with science or between theologies.

One needs only look back a few decades to find a time when comments like Nelsons were not only acceptable in the American space culture they were a central part of Americas science identity.

Few people exemplify this melding of space exploration and spirituality more than Nelson himself. In 1986, decades before he became NASA Administrator, Nelson went to space on the shuttle Columbia, the last NASA mission before the Challenger disaster. His 1988 memoir described his extraterrestrial sojourn as an eye-opening religious experience that contrasted starkly with that of his Soviet counterparts. Yuri Gagarin, the first Russian cosmonaut, proudly proclaimed when he returned to earth that he had looked for God and had not found him, Nelson wrote (perhaps misattributing Titovs 1962 comments). I looked, and could see nothing else. The Soviets might have reached the heavens first, but the Americans were the first to find God up there.

Nelson also recalled reaching into his pocket and pulling out his Bible while on the Columbia:

I remembered when, as a student at Yale, I had read the ancient words of the 19th Psalm, written by a shepherd boy in Israel almost 3,000 years ago. My college mind had wondered, What could David possibly know about space? As I read those words again, I was amazed that they could express my feelings so perfectly: The heavens declare the glory of God. The firmament sheweth His handiwork.

More than 30 years later, Nelson uttered the same scripture nearly verbatim while reflecting on the launch of the the telescope. It is a passage that has long been invoked by scientists and theologians to express the idea that there are truths that can only be discovered outside of scripture truths that must be learned from the handiwork of nature. Its been quoted to argue against Biblical literalism and science denial. And, for Nelson, it seems to give voice to a certain sense of awe and spiritual wonder at nature that has abided in him since his time as an astronaut.

The scientific, religious, and political culture of the U.S., however, has evolved tremendously since then. Christian nationalism has become a widespread and antidemocratic political force one that has been deployed to attack government-supported, science-based efforts to stem the Covid-19 pandemic and curtail climate change. Cold War-era God-talk, and the embrace of generic religiosity, no longer exemplify Americas place in the modern geopolitical world. The words Nelson uses to capture his connection with the cosmos may not have changed since the 1980s, but its a different nation now.

Adam R. Shapiro is a historian of science and religion. He is the author of Trying Biology: The Scopes Trial, Textbooks and the Antievolution Movement in American Schools, and (with Thomas Dixon) the forthcoming Very Short Introduction to Science and Religion.

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An Awe-inspiring Space Odyssey Lifts Off in Houston – Texas Monthly

Posted: at 11:55 pm

Decades ago, before billionaires could hitch rides into orbit, space exploration seemed like a more poetic endeavor.

I grew up in Dickinson, less than ten miles from NASAs Manned Spacecraft Center, when it was still easy to name all the astronauts training there. I went to church and school with kids whose dads were among the pocket-protected brigade of genius engineers, including flight director Gene Kranz, the guy Ed Harris portrayed in Apollo 13.

As a college student in the 1970s, I had a summer gig as a proofreader at the Lunar Science Institute, in the Diamond Jim West Mansion just east of NASAs back gate. Jovial, unkempt, caffeine-fueled scientists toiled at night in a room a few steps from my desk, sharing time on a huge mainframe and producing papers about moon rocks and soil samples. I only vaguely understood their work, but it felt important, and I kept their nouns and verbs in agreement and their capitals and commas in place.

By then, everyone knew what Earth looked like from space; the Apollo 17 crews famous Blue Marble photograph, which will be fifty years old this December, had become ubiquitous. Today, closer views of our planet stream continuously from the International Space Station on cellphones and laptops, freely accessible, like so many other twenty-first century amenities born from our ability to launch gadgets into the sky. I wonder if anyone will remember how meaningful that first whole-Earth image was.

The Infinite, a new virtual reality encounter, aims to revive that old sense of wonder. It might even give you the overview effect, a visceral moment of clarity astronauts often experience when they view our planet from afara near-spiritual vision of humanity as a single species on a vaporous and vulnerable blue ball within the universe.

Set up at Houstons Sawyer Yards through February 20 (then heading to Seattle), The Infinite was filmed by astronauts aboard the International Space Station between January 2019 and September 2021. Using a custom video camera that produces ultra-high-definition, 3D, 360-degree views, they recorded short scenes outside (and inside, filmed with a smaller 360-degree camera) of the ISS to show how they live and work, as well as glimpses of Earth and the farther reaches of space from the orbiting laboratory. Some of that material was released last year as a four-part film series, Space Explorers: The ISS Experience, by Montreals Felix & Paul Studios (which also designed the outside camera) and Time Studios. The Space Explorers series won last years Emmy for Outstanding Interactive Program.

I love a good VR happening. One of the best Ive been to is fine art: Laurie Anderson and Hsin-Chien Huangs To The Moon delivers a fantastical lunarscape with dinosaurs that materialize from DNA strands and asteroids that morph into black diamonds. The Infinite is something elsetruly virtual reality.

The entry of the exhibit, though, is inspired by 2001: A Space Odyssey. With a small group of strangers, I walked a ramp through a glowing white vinyl room into a metal chamber where lights flashed through seams in the walls. As the sliding doors closed, we heard the voice of astronaut Anne McClain, describing how raw she felt just before she launched into space. A countdown further fired up our juices. When the doors opened into a huge, dark room, we plucked our sanitized headsets from eerily lit floor-to-ceiling tubes. A minute or so later, I dissolved into stardust and re-formed as a female avatar with a bright green grid for skin.

Left: Two exit galleries help ease the transition back down to Earth. Melissa Taylor Photography/Public Content

Top: Guests suit up before stepping into the International Space Station. Melissa Taylor Photography/Public Content

A transparent rendering of the ISS hovered in the rooms center. The stations ambient beeps and transmissions alternated with a celestial soundtrack through my headphones. I had to make myself walk through the renderings wallsa disconcerting feeling from the startto reach the interactive orb-portals inside. With each orb visitors touch, a different 360-degree filmed scene appears. The shows sixty portals are loosely organized into seven-minute chapters with inspirational names: Adaptation, Progress, Unity, Expansion. The portals are spaced around the room, roughly near the points of the stations floor plan where they were filmed, and visitors choose which ones they will open. No one can get to all sixty during the shows 35-minute duration, so each trip through is different. One of the organizers told me it took him six visits to find all the material. That would be costly (adult tickets are $36 to $65; kids, students, and seniors pay less).

I was literally lost in space, wandering cluelessly through the virtual laboratory during my first visit, and only slightly more oriented during a second. The Infinite dwells on the human aspects of life in the ISSwhat it means to live and work 260 miles above Earths surface in a contraption thats configured like a plumbing fixture under a kitchen sink and hangs from a truss structure the length of a football field. Even seemingly insignificant details absorbed me: rows of commemorative mission stickers affixed to surfaces; the Velcro strips everywhere, indispensable for holding virtually anything in place, including toothbrushes, laptops, and wrenches; the way long hair floats, Medusa-like, around faces.

The stations tubular modules are cramped, with no discernible floors or ceilings and no real up or down, just four walls covered with a mind-boggling mess of wires, monitors, consoles, cargo compartments, and bags of supplies. This is no place for minimalists or claustrophobes. And then theres the constant dance with microgravity. The astronauts drift gracefully through the stations tunnels like khaki-clad sea creatures, hooking their sock-covered feet under footholds or grabbing handholds when they need to be stationary or maneuver through modules.

I watched them toss a football, share a meal, jog on a treadmill, explain how their outhouse works, say goodbye to departing crewmates, and monitor the gazillion science experiments that hang out in the walls. I followed them into the cupola, an observation capsule that holds the stations only windows. Life on the ISS is a wild mix of the unimaginable and the mundane. The housecleaning never ends. The internet service is sketchy. Face time with families is sacred.

Preparing for an EVA, or extravehicular activityi.e., a space walktakes weeks: suits must be readied, every move planned. Just suiting up takes hours. Visitors to The Infinite, however, can step through the transparent walls of the virtual ISS to be outside in a few seconds. A few orbs beckoned to me from somewhere near the truss structure, and I went for them. I knew my boots were firmly planted on a thinly carpeted floor, but I still felt disembodied and dwarfed by a tower of panels that glinted in the sun. Just an arms length away, other parts of the ISS looked shockingly Rube Goldberglike, with exposed wires, sensors, satellite dishes, and the gangly robotic arms that grab arriving cargo and crew vehicles. I was close enough to read the logos on module exteriorsJAXA (Japanese Aerospace Exploration Agency), ESA (European Space Agency), SpaceX.

Then I looked down, and it knocked the wits out of me. Earth! Alive, immense, and bright blue beneath wispy brushstrokes of clouds, organic and palpably alive. My eyes teared up, then I went giddy. It wasnt just that it was beautiful: something about the wholeness of the planet transcended the mayhem I knew humans were inflicting on it and themselvessomething profound and hopeful. The overview effect.

I could have stayed all day, but a voice in the headset instructed me to follow a path of light to a chair where I would view the final scene. It took me outside the station again to participate in an EVA that was filmed last September. In real time, Japans Akihiko Hoshide and Frances Thomas Pesquet performed what would have been a mundane task if they hadnt been tethered to a machine moving at over 17,000 miles an hour. They were installing a bracket on the truss structure, a full days work. I was just along for a few minutes ride, mesmerized by their movements but free to gaze beyond them, to Earth. When the scene ended, I felt like I could still walk on air.

I did not expect The Infinite to flip any switches in my brain. It had been light-years since I breathed the early NASA air of the Mercury, Gemini, and Apollo missions, watching every nail-biter launch and landing on TV. I have binged on YouTube videos shot from the ISS for days now, hoping to recapture the sense of Earth as a miraculously contained sphere. Even when the views are awesome, on my ordinary screen, the world remains stubbornly flat.

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Silencing a faulty gene may uncover clues to rare forms of ALS – National Institutes of Health

Posted: at 11:55 pm

News Release

Monday, January 24, 2022

NIH-funded preclinical study identifies potential therapeutic approach to treat ALS.

Using an experimental drug, researchers were able to suppress a mutated amyotrophic lateral sclerosis (ALS) gene. Studies in mice demonstrate that the therapy could show promise in treating rare, aggressive forms of ALS caused by mutations in the fused in sarcoma (FUS) gene. The study, published in Nature Medicine, was funded in part by the National Institute for Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health.

The study models how promising gene-targeting therapies can move expeditiously from pre-clinical development to clinical testing, said Amelie Gubitz, Ph.D., program director at NINDS. There is a desperate need for innovative approaches to treating ALS.

ALS, also known as Lou Gehrigs disease, is a fatal neurological disorder that causes the degeneration of motor neurons in the brain and spinal cord. People with ALS rapidly lose muscle strength and eventually their ability to move, swallow, and breathe. Most cases of ALS are sporadic, but at least 10% of cases are familial, or due to mutations in various genes. Mutations in the gene FUS cause severe forms of ALS, referred to as FUS-ALS, including a rare type that begins in adolescence or young adulthood.

In the study, Neil Shneider, M.D., Ph.D., the Claire Tow Associate Professor of Motor Neuron Disorders and Director of the Eleanor and Lou Gehrig ALS Center at Columbia University, New York City, and his team delayed the onset of motor neuron degeneration in mice by using an antisense oligonucleotide drug designed to silence FUS by blocking cells from making specific proteins. Following encouraging results, they administered the drug to a patient with ALS.

Compared to normal mice, mice with a mutated FUS gene had higher levels of insoluble FUS and other ALS-related proteins in their brains and spinal cords. Mice with higher doses of mutant FUS in motor neurons experienced rapid neurodegeneration that began early in life, much like FUS-ALS patients.

The study establishes a mouse model that is highly disease-relevant, said Dr. Shneider. In mice, we found that FUS toxicity was due to a gain of function and was dose-dependent, suggesting that we could treat FUS-ALS by silencing the FUS gene.

In 2019 Dr. Shneider met an individual with ALS in search of therapies that may help her disease. Inspired by her story, Dr. Shneider teamed up with a pharmaceutical company to develop a personalized therapy designed to target the FUS mutation.

In mice, injecting a single dose of the drug into the ventricles, fluid-filled spaces surrounding the brain, delayed the onset of inflammation and motor neuron degeneration by six months. The drug also knocked down levels of FUS by 50% to 80% in the brain and spinal cord. Following drug administration, insoluble forms of other ALS-associated proteins were also cleared.

Under a compassionate use protocol reviewed by the U.S. Food and Drug Administration, Dr. Shneider administered the drug to the patient it had been designed for. The patient received repeated infusions of the drug into her spinal canal for 10 months. During the treatment, the patients rate of motor function deterioration slowed. The patient tolerated the treatment well and there were no medically adverse effects.

The study is an example of a precision medicine, bench-to-bedside effort, said Dr. Shneider. We began with the mouse model to establish a rationale for the drug, conducted efficacy studies in the mouse, moved the drug into a human, and collected valuable data that was ultimately used to support a larger Phase 3 clinical trial.

Treatment began more than six months after clinical onset, by which time the disease had already significantly advanced. As is typical with juvenile-onset FUS-ALS, the disease progressed rapidly, and the patient died from complications of the disease.

By studying the patients brain and spinal cord tissue, researchers found that the drug silenced FUS throughout the nervous system and reversed the toxic nature of FUS and other disease-related proteins. Compared to tissue from untreated FUS-ALS patients and healthy controls, FUS protein aggregatesa pathological hallmark of this form of ALSwere sparse, suggesting that they may have been cleared by the drug. Tissue samples were provided by the New York Brain Bank of Columbia University.

The protein made from the FUS gene has been shown to be important for various cellular processes. Prior studies in mice suggest that FUS mutations result in the production of an abnormal protein that forms clumps, or aggregates, leading to motor neuron damage. By targeting the faulty gene in a way that suppresses toxic FUS activity, gene silencing products like the antisense oligonucleotide drug could potentially reduce or prevent disease progression.

The results were used to support a clinical trial testing the drug in patients with FUS-ALS (NCT04768972).

This study was supported by grants from the NIH (NS106236), Nancy Perlman, Tom Klingenstein, and the Judith and Jean Pape Adams Charitable Foundation.

NINDSis the nations leading funder of research on the brain and nervous system.The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

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

NIHTurning Discovery Into Health

Korobeynikov, V.A., et al. Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nature Medicine, January 24, 2022. DOI: 10.1038/s41591-021-01615-z

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5 Explosive Space Stocks to Buy in 2022 and Beyond – Motley Fool

Posted: at 11:55 pm

Space is a new and exciting industry with potential both now and in the future. According to a report by Morgan Stanley, the space industry could grow from $400 billion to more than $1 trillion by 2040, which doesn't even factor in new markets like space tourism. How do investors capture this upside? Several companies are beginning to build their contributions to this new market.

Investors need to understand that such a young industry carries many risks, but these five companies can generate significant returns if things work out well over the long term.

Image source: Getty Images.

Sometimes it's hard to believe, but the day is coming when people can tour space. Virgin Galactic Holdings (NYSE:SPCE) is helping build the space tourism industry. Its spacecraft is designed for suborbital flights, leaving the Earth's atmosphere but not entering orbit. These flights produce a short duration of weightlessness before coming back down.

The company sells tickets for future flights at $454,000 per seat but has more test flights to complete before beginning customer flights. The company's most recent test was a successful flight in July 2021, when founder Richard Branson joined the ship's crew. The long-term potential for Virgin Galactic is a fleet expansion and mastery of the technology that can make the price to fly affordable for more customers, bringing space tourism into the mainstream. The stock's small $2.6 billion market cap leaves a lot of room for upside if Virgin Galactic is successful.

If there is a space industry, how will we process the payments that flow back and forth? Payment processing company Shift4 Payments (NYSE:FOUR) could help answer that question. Hotels, entertainment businesses, and restaurants across the country use Shift4's platform.

In its report, Morgan Stanley believes that satellite broadband, satellite-delivered internet, could contribute between 50%-70% of the space industry's projected growth. Elon Musk's SpaceX has invested heavily to build Starlink, its upcoming broadband business. Shift4 has partnered with SpaceX to support it with its payment services. Shift4's management estimates this addressable market as being worth between $100 billion and $500 billion globally. Shift4's satellite broadband exposure andgrowing payments business could fuel growth over the years ahead.

Space is a complex industry, and companies like the industrial technology business Trimble (NASDAQ:TRMB) can advance the development of space with its software solutions that help its customers use data information to run their operations. For example, it can use satellite data to help customers use their physical assets most effectively. Its primary businesses concentrate on applications like buildings and infrastructure, geospatial data, commodities and utilities, and transportation.

For instance, the company's technology helps farmers maximize their crop harvest. Trimble's ability to simplify parts of these complex industries and applicationscould make it a natural fit for the space industry as it grows over time. The immaturity of space could make Trimble a good stock for broad exposure, and it happens to be the top holding in ARK's space-focused ARK Space Exploration & Innovation ETF.

Aerospace and defense company Lockheed Martin (NYSE:LMT) is a natural fit in the space industry, thanks to its vast experience in rockets and propulsion gained from making various weapons systems and vehicles for the U.S. military. It has a space business segment -- including satellites, space transportation, and defense systems -- that accounted for almost a fifth of total revenue in 2020.

President Donald Trump created the Space Force in late 2019, a new division of the U.S. military. Suppose space eventually becomes a new arena for geopolitical tension. In that case, Lockheed Martin's existing relationships and experience in the space industry could help its space segment grow as a part of the company's overall business.

Satellites are gaining more use as technology advances, helping us transmit information between space and Earth at faster speeds. BlackSky Technology (NYSE:BKSY) is a geospatial-data company that's building a network of satellites to perform rapid surveillance of the planet's landscape through its software-as-a-service platform. It can generate and transmit imagery in approximately 90 minutes; its customers include U.S. intelligence agencies and commercial customers in commodities and insurance.

It's a young company competing against other satellite companies in the market. Still, BlackSky's $2.5 billion business pipeline gives the company predictable revenue over the next few years. Investors will want to keep an eye on execution to see that backlogged business converts to billed revenue, and that BlackSky successfully expands its satellite fleet over time. Its market cap is just $370 million, so the upside is sizable if things work out.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Their DNA Hides a Warning, but They Dont Want to Know What It Says – The New York Times

Posted: at 11:55 pm

I was like, wait a minute. Where is this information going? How will it be utilized? she later asked. I dont know if there are any implications for life and health insurance for me or my family, but why risk it?

What should happen when researchers, while sequencing a participants DNA as part of a large study, discover gene variants that increase the risk for conditions that might be prevented with medical treatment or surveillance? Some researchers believe they have an obligation to find the participants often years after they provided a DNA sample contact them, and tell them what they have found. But, some research subjects, like Ms. Konstadt, feel they have a right not to know. Is it ethical for doctors to let them insist they can opt out of learning more without first knowing the particular risk they are facing?

For Dr. Robert Green, an investigator for the biobank with Ms. Konstadts DNA, the Mass General Brigham Biobank, and author of a recent paper about its policies, the answers are clear. The consent form for the biobank tells participants that if the researchers find a worrisome variant, and if there is an intervention that can reduce risk, the participants will be contacted. There will be seven attempts to reach participants calls and letters before the team gives up.

We are offering the information, not forcing participants to accept it, said Dr. Green, who is also a geneticist and professor of medicine at Harvard. If you dont answer the phone or decide when offered that you dont want to hear anything more, or even hang up on us when we call, then thats your choice.

Dr. Green and his colleagues point out that the possibility of being contacted was in the consent form:

While you should not expect to receive any results from your participation in this research, if experts from the Biobank decide that research results from your sample are of high medical importance, we will attempt to contact you. In some situations, follow up testing might be needed in a certified clinical lab. You and your medical insurer may be responsible for the costs of these tests and any follow up care, including deductibles and co-payments.

But some, like Ms. Konstadt, did not notice that clause when signing the form.

Out of more than 36,000 participants, whose DNA its researchers analyzed, the Mass General Brigham Biobank found 425 with worrisome gene variants whose effects could be ameliorated by depending on the genes enhanced cancer surveillance or aggressive medical treatments to lower cholesterol levels, for example.

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Venture into the unknown – Los Angeles Times

Posted: at 11:55 pm

Everyone is always saying that we should get out of our comfort zones, but what does that actually mean? For me, it means to explore the unknown and its in the unknown that we can really grow and learn.

Through exploration of the unknown, humanity has been able to develop opportunities for success in every aspect of our society.

The subjects that students learn in science class today are derived from the exploration of the unknown. Without the great scientists and the research theyve committed themselves to, we would still be stuck in a state of perpetual stagnation.

For example, one of historys greatest scientists, first astronomers and initiator of our current space exploration programs was Galileo Galilei. Through careful planning and measurements along with a great deal of imagination, Galileo was able to create and perfect the telescope, a tool which has benefited us in space exploration for centuries.

Later he influenced many other leading scientists, such as Kepler, Newton, and our current NASA program (which has named a satellite after him), to continue his studies of the unknown universe. By taking the initiative and stepping into the investigation of the unknown, Galileo and many other noteworthy scientists were able to make a name for themselves and begin a new age of exploration, and human science will forever be in their debt.

However, the exploration of the unknown isnt limited to just academic values and knowledge. Revolutionists in both art and music have created entire eras of art, each being significantly different than the one before.

According to the National Gallery of Art, Pablo Picasso, founder of the art style Cubism, is a prime example of someone who spent their career changing what humanity knows of art. Although he was a conventional artist at the beginning of his career, his shift into what is now his most well known style of art is what made him influential.

Despite the fact that his art caused much controversy due to its unconventional depictions of what a normal painting should look like, by venturing into a new territory of art and exploring every corner of it, he was able to shape the concept of modern art.

An example of a pioneer in music is the well-known musician Louis Armstrong, who is credited with being one of the first and most prominent jazz musicians. As reported by the Louis Armstrong House, Armstrong started off rough with a difficult childhood, but he went on to become the first major jazz soloist and one of the most influential musicians of the twentieth century by producing a distinctly different style of trumpeting and incorporating a more formal yet loose style of swinging jazz that revolutionized the genre.

By breaking away from the constraints of normality, these artists and composers were able to bring about lasting changes and developments in the industry.

Not only can exploring the unknown benefit society as a whole, but it can also benefit one personally. When I was seven, I had taken an interest in rock climbing. On my eighth birthday, I asked my parents to hold my party at a local climbing gym. It would be my first time ever trying rock climbing.

Upon entering the facility with my friends, many of them decided that the feat was too scary for them. Being an adventurous third grader, I was determined to scale my first wall right then and there. However, while waiting in line for my turn, I began to get cold feet.

What if the rope snaps? What if I embarrass myself while trying to be cool?

Thoughts of doubt plagued my mind and soon it was my turn to start the climb. I was about to bail out on my chance when I realized that doubting myself into not partaking in an interest of mine was something that I was going to regret for years to come.

I hyped myself up for the climb and slowly started to scale the wall. About a quarter of the way through, I realized that I really enjoyed the experience and I started to climb faster. When I reached the top, I felt a certain sense of accomplishment that I wouldnt have felt if I had bailed out from the start.

To top my already proud self, the spotter who was handling the pulley gear told me that my climb was one of the best that shed ever seen from a junior and that I should consider continuing the sport.

From this experience, I learned that, although stepping out of my comfort zone and venturing out into the unknown was frightening, without the experience, I might have never found an interest that I was truly passionate about and even had a talent in.

The exploration of the unknown has provided the opportunity for the world to improve and augment itself in science, art, and beyond. Personally, it has definitely broadened my world and opened the doors to new experiences and growth.

What does the unknown have to offer you?

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BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH) -…

Posted: at 11:55 pm

-If successful, BridgeBios investigational gene therapy BBP-631 would be the first therapy for CAH to restore the bodys hormone and steroid balance by enabling people with CAH to make their own cortisol and aldosterone

-Initial Phase 1/2 data readout anticipated in the second half of 2022

-BridgeBios gene therapy portfolio also includes a clinical stage program for Canavan disease and preclinical programs for classic galactosemia, TMC1 hearing loss, tuberous sclerosis types 1 and 2, cystinuria and a genetic dilated cardiomyopathy

PALO ALTO, Calif., Jan. 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in ADventure, its Phase 1/2 clinical trial of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy for the treatment of classic congenital adrenal hyperplasia (CAH). CAH is one of the most prevalent genetic diseases, with more than 75,000 cases estimated in the United States and European Union.

Dosing the first patient in our CAH trial is a landmark milestone and we are grateful for the support from the medical and patient communities who helped us reach this moment. For more than 50 years people living with CAH have had the same limited standard of care lifelong daily steroid replacement treatment. Our investigational gene therapy offers patients a potential single-dose intervention designed to restore their bodys hormone and steroid balance by making their own cortisol and aldosterone, said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. This is the second gene therapy trial we have initiated in less than four months, and we are excited to advance this trial and our other gene therapy programs in the hope of improving patients lives.

Adults, children and families affected by CAH experience the daily burden of the disease and often, unfortunately, the side effects and morbidities associated with the current treatment regimens. As an endocrinologist, its incredibly exciting to reimagine a new approach to treating this disease, added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy. Adrenas Therapeutics, the affiliate company of BridgeBio focused on developing BBP-631 for CAH, is part of BridgeBio Gene Therapys portfolio.

The Phase 1/2 open-label study is designed to evaluate the safety, tolerability and pharmacodynamic activity of the companys AAV5 gene therapy, BBP-631, in adults with classic CAH. In the initial dose-finding phase of the study, each subject will receive a single intravenous (IV) infusion. The primary outcomes of the study are safety, as well as change from baseline in endogenous cortisol levels which BBP-631 has the unique potential to restore. Change from baseline in steroid biomarkers for hydroxyprogesterone (17-OHP) levels and androstenedione (A4) levels will also be measured. Preclinical proof-of-concept data have shown the approach provides efficient and persistent delivery of functional 21-hydroxylase (21-OH) enzyme to the adrenal gland.

We are honored to be the first site to administer gene therapy in a patient with CAH as it is a potential game-changing treatment option that targets the disease at its source, said Kyriakie Sarafoglou, M.D., associate professor and director of the Center for Congenital Adrenal Hyperplasia at the University of Minnesota. We are eager to see whethergene therapy can restore endogenous cortisol production, and also look forward to exploring its effect on thephysiological mechanisms that regulate the hypothalamic-pituitary-adrenal axis including circadian and ultradian hormonal profiles.

BridgeBios investigational AAV5 gene therapy for CAH is one of the Companys 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and cancers. Initial Phase 1/2 data readouts of the Companys AAV5 gene therapy for CAH and the Companys AAV9 gene therapy for Canavan disease are expected in the second half of 2022.

For more information about the ADventuretrial, visit ClinicalTrials.gov (NCT04783181).

About BBP-631BBP-631 is an AAV5 gene therapy developed to treat CAH due to 21-hydroxylase deficiency at its source. BBP-631 is designed to deliver a functional copy of the 21-hydroxylase gene and has been shown through multiple preclinical studies to result in efficient and persistent delivery to the adrenal gland, where hormones are naturally made. If successful, BBP-631 may restore the bodys hormone and steroid balance by enabling people with CAH to naturally make their own cortisol and aldosterone. It could also allow for people with CAH to eliminate or significantly reduce their daily glucocorticoid or mineralocorticoid doses, which is the current standard of care for patients.

About Congenital Adrenal Hyperplasia (CAH)Affecting approximately 75,000 people in the United States and European Union, CAH is a group of genetic disorders that affect the adrenal glands, which is caused by a mutation in the gene encoding for 21-hydroxylase, an enzyme essential for making the hormones cortisol and aldosterone which are critical for various physiologic functions. Cortisol is necessary for the body to respond to injury, stress or illness, and aldosterone is required to maintain proper blood pressure and sodium levels. Unable to produce cortisol and aldosterone, people with classic CAH cannot mount the healthy physiological response to stressors, such as illnesses, that allows their heart, lungs, kidneys and other organs to compensate for the stress, which can be life-threatening. These adrenal crises can be particularly dangerous for young children.

About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBios pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the companys first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking StatementsThis press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as anticipates, believes, estimates, expects, intends, may, plans, projects, seeks, should, will, and variations of such words or similar expressions.We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to the timing and success of BridgeBios Phase 1/2 clinical trial of BBP-631 for the treatment of CAH, expectations, plans and prospects regarding BridgeBios regulatory approval process for BBP-631, the ability of BBP-631 to treat CAH in humans, and the timing and success of initial top-line Phase 1/2 date of BBP-631, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBios ability to continue and complete its Phase 1/2 clinical trial of BBP-631 for the treatment of CAH, past data from preclinical studies not being indicative of future data from clinical trials, BridgeBios ability to advance BBP-631 in clinical development according to its plans, the ability of BBP-631 to treat CAH, the ability of BBP-631 to retain Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency, risks inherent in developing therapeutic products, the success, cost, and timing of the Companys product candidate research and development activities and ongoing and planned preclinical studies and clinical trials, the success and timing of preclinical study and clinical trial results, the success of its clinical trial designs, the fact that successful preliminary preclinical study or clinical trial results may not result in future clinical trial successes and/or product approvals, trends in the industry, the legal and regulatory framework for the industry, the success of current and future agreements with third parties in connection with the development or commercialization of the Companys product candidates and FDA-approved products, the size and growth potential of the market for the Companys product candidates and FDA-approved products, the Companys ability to access additional funding upon achievement of portfolio milestones, the accuracy of the Companys estimates regarding expenses, future revenue, future expenditures and needs for and ability to obtain additional financing, the Companys ability to be a sustainable genetic medicine innovation engine and to build the next great genetic medicine company, the Companys ability to obtain and maintain intellectual property protection for its product candidates and approved products, the competitive environment and clinical and therapeutic potential of the Companys product candidates and FDA-approved products, the Companys international expansion plans, and potential adverse impacts due to the global COVID-19 pandemic such as delays in clinical trials, preclinical work, overall operations, regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; as well as those set forth in the Risk Factors section of BridgeBios most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SECs website atwww.sec.gov.Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:Grace RauhGrace.rauh@bridgebio.com(917) 232-5478

BridgeBio Investor Contact:Katherine Yaukatherine.yau@bridgebio.com(516) 554-5989

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BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH) -...

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Gene therapy shows promise at treating severe form of epilepsy – Freethink

Posted: at 11:55 pm

Scientists at the University of Virginia (UVA) School of Medicine have developed a promising gene therapy to target the mutation behind a severe form of epilepsy.

The new Dravet syndrome treatment could help improve and extend the lives of people with the rare but debilitating disease.

The challenge: People born with Dravet syndrome start experiencing seizures when theyre still infants. Seizures continue throughout their lives and often lead to a host of health problems severe developmental delays, speech impairments, intellectual disability, and movement difficulties.

There is no cure for Dravet syndrome, and 10-20% of children born with it die before reaching adulthood.

Certain diets, medications, and therapies can help reduce the frequency and severity of seizures, but patients still require constant, lifelong care.

Between 10% and 20% of children born with Dravet syndome die before reaching adulthood.

The idea: In more than 80% of cases, people with Dravet syndrome have a mutation in a particular gene (called SCN1A) that results in reduced production of a critical protein in the brain.

This leads to a shortage of that protein in specialized brain cells, called interneurons, that protect against seizures.

No existing Dravet syndrome treatment directly addresses the underlying cause of the disease so the UVA team decided to make one that does.

The Dravet syndrome treatment: Dravet is usually caused by a single random mutation, but people generally carry two copies of every gene. The researchers aimed to use the second intact gene to treat the disease.

The treatment completely prevented seizures and premature death in infant mouse models of Dravet.

Using an approach they call Targeted Augmentation of Nuclear Gene Output (TANGO), the scientists developed a Dravet syndrome treatment that prompts the intact gene to increase protein production.

When tested in mouse models of Dravet syndrome, the treatment completely prevented seizures and premature death in infant mice.

The animals interneurons were more responsive, more active, and better able to do their jobs, according to a press release.

The big picture: Therapies that work in mice often fail in humans, but early results in human trials suggest this Dravet syndrome treatment has a shot.

More than 70% of children with Dravet in an ongoing phase 1/2A study experienced a reduction in seizure frequency following treatment, according to Stokes Therapeutics, the company developing the drug.

That study was small, with just 21 participants so far, and these are interim results, so its still too early to say whether the therapy (called STK-001) will ultimately make it to patients.

Still, if the results hold up in larger trials, the new treatment could help people with Dravet syndrome live longer, healthier lives.

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Scientists Surveyed People About Space. The Comments Are Out Of This World – VICE

Posted: at 11:55 pm

ABSTRACT breaks down mind-bending scientific research, future tech, new discoveries, and major breakthroughs.

The history of space exploration has never been dull, but we happen to be living through an especially eventful era of dazzling discoveries, ambitious missions, and emerging challenges.

To get a sense of how people feel about this multifaceted spacescape, Motherboard is exclusively publishing the results of a wide-ranging poll of the American public, conducted by a team at the Outer Space Institute, a transdisciplinary global network of space experts, and the Angus Reid Forum USA, an online public opinion community.

The poll, which is available to view in full at this link, queried more than 1,500 respondents about topics such as aliens, orbital debris, the United States Space Force, and human missions to the Moon and Mars. The team also invited participants to provide feedback, in their own words, on the poll, with verbatim responses that ranged in tone from wistful to conspiratorial to zany (one person, for instance, offered this simple yet incisive comment: Uranus).

The poll was conducted through an online opinion panel and was designed to be as statistically representative of the American public as possible in terms of demographics, geography, and even political affiliation. The end results offer an intriguing snapshot of public attitudes about space from the most influential spacefaring nation on Earth.

U.S. public opinion really matters in space because the United States really matters in space, said Michael Byers, a professor and Canada Research Chair in global politics and international law at the University of British Columbia (UBC), in a call with team members Aaron Boley, an associate professor and Canada Research Chair in planetary astronomy at UBC, and Gregor Sharp, senior manager of panel research and outreach at the Angus Reid Forum. (Byers and Boley also serve as co-directors of the Outer Space Institute).

As for why Canadian researchers conducted the poll, its because what [U.S. decision-makers] decide matters for everyone, added Byers.

On top of that, it's just super-interesting research that hasn't been done before, noted Sharp.

To that point, the poll revealed overwhelming consensus on a few issues. A whopping 81 percent of participants agreed that outer space should belong to everyoneno one country should be able to claim control over it, with 49 percent indicating they strongly agreed with that statement. The value of fundamental science, such as astronomy, was likewise broadly acknowledged, with 72 percent of respondents agreeing that it is important and deserves government funding (30 percent strongly agreed).

The participants also expressed high levels of optimism about the existence of alien life, as well as the odds that humans will one day encounter it. Seventy-one percent agreed it is likely that there is other intelligent life beyond Earth in our galaxy, the Milky Way, with 40 percent strongly agreeing with that statement.

Meanwhile, 75 percent said it is likely that we will detect microorganisms beyond Earth in our solar system (49 percent said very likely) while 77 percent said it is likely that we will detect microorganisms elsewhere in our galaxy (52 percent said very likely).

The broad recognition of the possibility of life in the solar system has implications for how we conduct space exploration, said Boley, who noted that it also underscores the need for planetary protection measures designed to prevent cross-contamination between Earth and other worlds.

The wide public recognition that this is something that is a real possibility is, I think, an important component of this poll, Boley added.

Byers said that some of the results were extremely helpful to the Outer Space Institutes advocacy efforts, which include a push to ban anti-satellite (ASAT) tests. These tests destroy satellites and create swarms of orbital debris, which is outer space junk made up of defunct or broken spacecraft parts.

The poll revealed strong opposition to ASAT tests and high levels of concern about orbital debris. Seventy-two percent agreed that there should be an international ban on conducting tests that create orbital debris, and 69 percent agreed that countries that create more orbital debris should be sanctioned.

These results show that the U.S. government would have public support to add its weight to the push for a test ban treaty, Byers said.

The poll also showed that the American public is wary of some commercial activities in space, and generally has nuanced opinions about the private space sector. Sixty-nine percent said there should be limits on the number of satellites that companies can launch, indicating awareness about megaconstellations, such as SpaceXs Starlink, that could add tens of thousands of satellites to orbit this decade. In addition, 54 percent of respondents said that companies should not be able to profit from resources extracted off of the planet, with 29 percent saying they should be able to profit and 17 percent saying they were unsure about this issue.

For the most part, the poll did not show a huge difference in opinion according to political affiliation. However, Americas notoriously polarized politics did manage to infect one topic: the United States Space Force.

Perhaps unsurprisingly, participants who supported Donald Trump in the 2020 presidential election were far more likely to have favorable views of this military agency, which was previously known as U.S. Air Force Space Command before Trump rebranded it. Seventy percent of those who voted for Trump in the last election agreed that the Space Force is essential for protecting American interests in outer space, compared to 33 percent of respondents who voted for Biden in 2020, and 41 percent of people who did not vote in the 2020 election.

There were other revealing nuggets lodged in the demographic details of the responses. Women were more likely than men to say they were unsure of their opinion on the presented issues, which is a gender-based trend that turns up in polls on many subjects. Sharp also pointed out that African-Americans were generally more skeptical of extravagant space missions, which may reflect, in part, the recognition of longstanding institutional racism within spaceflight, perhaps typified by the 1970 song Whitey on the Moon by Gil Scott-Heron.

Some respondents provided written feedback to the poll in a section the team called Verbatims. Sharp warned that these comments should not be viewed as representative of the American public, because they express the viewpoints of only a few individuals.

To the extent that a verbatim reflects data that we polled on, sometimes it's just a very good way to illustrate, in somebody else's words, how they think about a question that we may have abstracted or may have framed slightly differently, Sharp explained. There's the potential to link up verbatims to the overall results, but of course, proceed with caution because the way people infer or interpret things does vary.

Indeed, an interesting subset of responses referenced conspiracy theories and misinformation, though its difficult to parse whether those comments are genuine beliefs or playful trolls, according to the teamfollow-ups would be required to get a better sense of the real intent. Here are a few of those responses, some of which are edited for brevity but otherwise unaltered:

Did we really go to the Moon?

I believe its all a lie . Conspiracy theory 101

NASA is the biggest frauds besides the government. We havent been to the moon, outer space or mars. The earth is flat, not a globe. The earth being round is a massive deception that upon research is uncovered.

I just hope we really are going in outer space and the moon.Gov sure is wating alot of money in space.

I still find it strange that NASA kinda pretty much disappeared and then came back. Like what is really going on?

I think governments should be more truthful about all of it, like what's going on out there.

Was there actually a moon landing?

Humans destroy everything. We cant figure out to leave well enough alone. Lets fix Earth (and I dont even believe in the constructs of Global Warming )

China just wants to get us to spend billions....they haven't even been to the Moon......neither have we.....but I would think that anyone wishing to go to Mars would want to go to the Moon first.

Beyond mentions of conspiracies, the comments expose a kaleidoscopic array of interests and opinions about space. For instance, a significant chunk expressed concern that funding for space exploration would be better spent on social or environmental issues on Earth, while others were extremely enthusiastic about exploring outer space. Here are a few snippets of both perspectives.

I love space, space exploration, the idea of intelligent life-- I love all of it but it amounts to absolutely nothing when we have some pretty catastrophic issues here that could be solved with that money (military budgets, too!)

We should be looking for other habitable planets like earth and trying to figure out how to travel quickly, like warp drive, instead of wasting so much time on Mars that is not habitable.

Don't colonize Mars. Focus on this planet first. So many more people will suffer needlessly if we just take our societal fuck-ups to another planet, especially since so many are already suffering here. We cannot handle it.

There is no compelling reason to funnel billions of dollars into exploring space in any capacity when our planet will become unlivable within a few generations. Doing so is irresponsible and disgusting. Countries, companies, and individuals with wealth should spend their resources helping people and the earth, not getting into pissing wars over space.

The emergence of private space companies, such as SpaceX and Blue Origin, also generated a range of reactions. Some respondents welcomed the dawn of commercial spaceflight, while others voiced negative opinions of space sector billionaires like Elon Musk and Jeff Bezos. Here are some of those comments:

Billionaires are evil and have already destroyed this planet so they're looking for another to destroy, figures

I think the new private companies are doing much better than governments did to promote space exploration

I think we should spend our tax $$$ on people here on earth and stop wasting it on space projects. Let Elon Musk spend his billions on space projects if he wants to.

Limits on billionaire vanity project rocket launches- Jeff bezos should be taxed for every recreational rocket trip- perhaps to the scale of their greenhouse gas emissions.

Honestly I think if individual people are wealthy enough to go to space on their own, then however much money they spend getting there they should also have to donate to climate issues.

I hate the fact that we are now exploiting celebrities to go into space. They don't belong there. Leave space travel to the seasoned astronauts.

Another fascinating subsection of participants approached space issues through the lens of their religious beliefs:

We should be exploring Earth's waters instead of wasting so much time , money and energy on chasing outer space fantasies. If God wanted us to have other worldly neighbors he would have created them and maybe he already has!?

My biblical beliefs are that it All belongs to God, if we are to represent Him well, there needs to be a balance of protecting the USA and meeting the needs of the whole working class of earthly humans. Not just the rich assholes.

Naturally, many participants also offered their thoughts on UFOs and whether humans have already been contacted by alien life:

I've seen UFO's/UAP's 4 times in my life that I know no man had anything to do with creating or piloting. So I think more pointed questions as to one's personal experiences of whether or not we've ever witnessed a UFO/UAP would be interesting for you to know about us.

I believe that there are outer space beings. Mainly Aliens. I have seen UFO's in different areas of America.

Speaking of aliens, there was an interesting split in the comments between people who viewed extraterrestrial contact in a positive light and those that expressed concerns about the potential dangers of such an encounter:

We are not alone. We should have a global plan

Alien relations? We should decide as a planet how we interact with potentially intelligent alien life.

I think it is highly likely that we will find other intelligent life other than microorganisms out in our solar system! It is to vast and to large for us to be the only ones

If there is a race advanced enough to visit earth as often as we presume, how can we be foolish enough to think we could fight them in a battle.

Where are the smart aliens that can help us cure cancer and extend human life.

Some of the responses absolutely demand further clarification. One person suggested that people might be able to hear aliens when they smoke cannabis. Another dared to address the question on everyones mind: What about sex in zero gravity ??? Is getting pregnant there even possible ??? A participant also came up with a creative, if bittersweet, idea to immortalize humanitys existence: We should carve a carbon/carbon-dioxide molecule diagram into the moon in case humans die off because of climate change.

My personal favorite is a bit inscrutable, but I read it as a succinct suggestion: more planets. Agreed.

Overall, the combination of the poll and the verbatim responses suggest that plenty of Americans are curious about all facets of space explorationincluding completely unexpected onesand that many have strong opinions about how it should be conducted now, and in the future.

It was very pleasing to see the relatively high level of understanding of space issues, said Byers. People are plugged in.

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FDA Pulls on the Reins for Mustang’s Gene Therapy as Others Advance – BioSpace

Posted: at 11:55 pm

Shares of Mustang Biohave fallen more than 13% in premarket trading after the company announced the U.S. Food and Drug Administration had placed a hold on the companys Investigational New Drug application for its bubble boy gene therapy.

The Worcester, Mass.-based company said the regulatory agency placed a hold on the planned Phase II study pending Chemistry, Manufacturing and Controls (CMC) clearance for MB-207, Mustangs lentiviral gene therapy. Mustang intended to initiate a pivotal Phase II study to assess the gene therapys safety, tolerability, and efficacy as a potential treatment forX-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. The study would include patients who have been previously treated with a hematopoietic stem cell transplantation.

MB-207 has previously been granted Orphan Drug and Rare Pediatric Disease designations by the FDA, makingthe asset eligible for a rare pediatric disease voucher.

In addition to the planned MB-207 Phase II study, Mustang is conducting a Phase I/II XSCID study in newly diagnosed infants under the age of two with MB-107. Like MB-207, MB-107 has also been granted Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy designations.

Manuel Litchman, M.D., president and chief executive officer of Mustang Bio, said the company hopes to efficiently expedite the development of both MB-2017 and MB-107. He said they believe they are well positioned to address the FDAs CMC concerns.

While Mustangs gene therapy trial is temporarily prohibited from the beginning, the FDA did clear several other companies requests to begin clinical studies.

Mind Medicines LSD Formulation Moves into Phase IIb

New York-based Mind Medicines IND for a Phase IIb study of MM-120 for treatinggeneralized anxiety disorder (GAD) was given the go-ahead by the FDA. That trial had been on hold,but the hold was recently lifted after Mind Medicines address of participant monitoring protocols for the upcoming study. The Phase IIb trial is expected to begin later this year.

The company expects to enroll a total of 200 participants who will receive a single administration of up to 200 g of MM-120 or placebo. The study's primary endpoint is the reduction in anxiety symptoms for up to 12 weeks following a single administration of MM-120, a pharmacologically optimized form of LSD.

Imara Inc. Will Begin Cardiac Study in Second Quarter

Bostons Imara Inc. will begin assessing tovinontrine (IMR-687) in a Phase II studyto treatheart failure with preserved ejection fraction (HFpEF). The clinical trial is expected to begin in the second quarter of 2022. The trial will evaluate tovinontrine in approximately 170 patients 45 years of age or older with persistent HFpEF symptoms. The primary endpoint of the study will be change in NT-proBNP, with secondary endpoints that include safety and tolerability and the quality of life measures.

Tovinontrineis a highly selective and potent small-molecule inhibitor of phosphodiesterase-9 (PDE9).

Nanoscope Therapeutics Heads into Phase II with Stargardt Therapy

Texas-based Nanoscope received IND clearance from the FDA for a Phase II study of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy. MC)-010 is being assessed as a gene therapy to restore vision in Stargardt patients. Stargardt, an inherited retinal disease, is a form of macular degeneration affecting children and adults.

Nanoscopes MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive. It uses proprietary AAV2 vectors. The Phase II trial is expected to begin in the first half of 2022.

Nanoscope is currently conducting a PhaseIIbstudy of MCO-010 for retinal pigmentosa (RP) patients. MCO-010 has received orphan drug designations from the FDA for RP and Stargardt.

SwanBio Takes Rare Disease Gene Therapy into the Clinic

Philadelphia-based SwanBiosgene therapy for the treatment of adrenomyeloneuropathy (AMN) has been cleared for a Phase I/II study. SwanBios lead candidate SBT101 is the first AAV-based gene therapy in development designed to compensate for the disease-causing ABCD1 mutation in AMN patients.

Adrenomyeloneuropathy is the adult-onset degenerative spinal cord disease that affects people with adrenoleukodystrophy, a rare, genetic and metabolic condition. The Phase I/II study will assess the safety and efficacy of the gene therapy. It is expected to begin in the second half of 2022.

Preclinical data shows that treatment with SBT101 demonstrated dose-dependent improvement of AMN disease markers in animal models. The gene therapy was also well-tolerated in non-human primates at six months post-treatment. The company said the SBT101 program builds on its ongoing natural history study of AMN.

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