Genetically Modified People Are Walking Among Us – The New …

It felt as if humanity had crossed an important line: In China, a scientist named He Jiankui announced on Monday that twins had been born in November with a gene that he had edited when they were embryos.

But in some ways this news is not new at all. A few genetically modified people already walk among us.

In the mid-1990s, fertility doctors in New Jersey got an idea for how to help women have children. They suspected that some women struggled to become pregnant because of defective material in their eggs.

To rejuvenate them, the doctors drew off some of the jellylike filling in eggs donated by healthy women and injected it into the eggs of their patients before performing in vitro fertilization.

The researchers did not ask the Food and Drug Administration for permission to try out the procedure. Only after their patients started having healthy children did they share the news that it seemed to work. Once the word got around, would-be parents streamed into clinics to try the procedure themselves.

But other people reacted with shock rather than excitement. Our cells generate fuel in miniature factories called mitochondria. And each mitochondrion carries its own small set of genes. The New Jersey fertility doctors might have created children with the DNA of three people, not two.

It turned out that this was indeed the case. The doctors discovered that some of the children carried mitochondrial DNA from the donors in addition to their parents. In their 2001 report on this discovery, they called it the first case of human germ-line genetic modification resulting in normal healthy children. The germ line is a lineage of cells that gives rise to a new person.

The F.D.A. was not pleased. It sent the clinics letters demanding that they apply to test the method as if it was a new experimental drug. Those bureaucratic hurdles were so daunting that the clinics stopped injecting eggs.

By then, perhaps a dozen children had been born with a mixture of DNA. Maybe there are more no one knows for sure.

The New Jersey doctors later tracked down some of these children and didnt find anything unusual about their health as teenagers. Meanwhile, some biologists had realized that a variation on their procedure might be able to do something else: prevent diseases that are otherwise incurable.

Like the DNA in our chromosomes, the DNA in our mitochondria can mutate. Mutations can cause symptoms ranging from blindness to early death, and women pass them down to their children. An estimated one in 5,000 people suffer from a mitochondrial disease, and for a vast majority, there are no effective treatments. Scientists wondered if they could erase these diseases by swapping mitochondria.

The procedure they envisioned began with taking a patients chromosomes out of one of her eggs. Next, they got an egg from a healthy donor and removed her chromosomes as well. Finally, they inserted the patients chromosomes into the donor egg and fertilized it with sperm.

Tests of this so-called mitochondrial replacement therapy, carried out on mice and monkeys, offered encouraging results. But when scientists approached the United States government about trying it out on human eggs, they got shut down.

It wasnt just the possible medical risks that worried people. Many saw it as an affront to human dignity.

It is a macabre form of eugenic human cloning, declared a Nebraska congressman, Jeff Fortenberry, at a hearing in 2014.

Two years later a provision was mysteriously slipped into a congressional budget bill that barred the F.D.A. from even considering mitochondrial replacement therapy. So researchers went underground.

In 2016, an American fertility doctor named John Zhang announced that he had gone to Mexico to quietly carry out the procedure on a woman from Jordan with a neurological disease called Leigh syndrome. She gave birth to a boy who appeared healthy. But she and her husband had no interest in letting scientists track the health of their child. We know nothing more of his fate.

This history echoed loudly this week, when Dr. He, an assistant professor at the Southern University of Science and Technology in Shenzhen, told the world that he had made gene-edited babies by altering the DNA of human embryos with a new technology called Crispr.

He cut out a small portion of DNA from a gene called CCR5. People who are missing this chunk of genetic material appear to be resistant to infections with H.I.V. Dr. He reasoned that genetically modified babies would resist the virus, too.

On Sunday, MIT Technology Review broke the news, followed by a lengthy story by The Associated Press. Dr. He posted a series of triumphant videos online, and on Wednesday, he went to a major gene-editing conference in Hong Kong to show slides with some details of his work.

Like the New Jersey fertility doctors before him, Dr. He was roundly condemned for his secretive recklessness. The organizers of the Hong Kong meeting issued a statement Thursday calling the birth of the twins irresponsible. They said Dr. He had designed the study poorly, and they labeled his ethical considerations a failure. Some scientists who watched Dr. He's talk wondered if he might have actually removed the wrong chunk of the CCR5 gene. The Chinese government called the procedure illegal and opened an investigation.

I got in touch with Glenn Cohen, a professor at Harvard Law School who studies reproductive technologies, to ask him to guess what happens next. His forecast sounded like a repeat of the mitochondrial replacement story.

My sense of what will happen is that across the world there will be strong regulatory action, Professor Cohen told me. He predicted a blanket ban of the technology. People are scared, and when they are scared they make decisions that are not so subtle.

On Wednesday, the commissioner of the F.D.A., Scott Gottlieb, appeared to give some credence to Professor Cohens prediction. In an interview with BioCentury, he criticized the scientific community for failing to stop Dr. He and warned of potential regulations and laws that could be far more restrictive than they might otherwise be if there were more confidence that the community was able to self-impose appropriate standards.

That would be a shame. There may be times when editing human embryos would make medical sense. Last year, the National Academy of Sciences and the National Academy of Medicine issued detailed guidelines about what sort of cases might qualify. While they didnt point to any particular disease, they argued that it should be considered only when no other treatment could allow parents to have a healthy child.

Fortunately, history offers us a different path. We need only look at what happened to mitochondrial replacement therapy in Britain.

When British scientists raised the idea of using the procedure on human eggs, the country conducted a serious, open conversation about the pros and cons. The health department conducted a long investigation. Parliament held a public debate. And in 2015 it passed a law approving the procedure.

The British government wasnt creating a medical Wild West, where doctors were free to use the procedure whenever they wanted. Clinics had to get a license from Britains Human Fertilization and Embryology Authority, which would monitor the procedures and track the children throughout their lives to check for unexpected side effects.

This February the authority announced that it was for the first time approving the use of mitochondrial replacement therapy on two women at a fertility clinic in Newcastle. On Thursday, a representative at the authority declined to say whether children had yet been born as a result.

Its only natural for the world to focus its attention on the two babies born in China. But these babies in Britain deserve our attention, too. We can choose which ones represent the future.

Carl Zimmer writes the Matter column for The New York Times and is the author of She Has Her Mothers L
augh: The Powers, Perversions, and Potential of Heredity.

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Genetically Modified People Are Walking Among Us - The New ...

Spatial Genomics & Transcriptomics Market Demand is Expanding at a Stellar Pace in the Years to Follow – BioSpace

The demand within the global spatial genomics and transcriptomics market is expanding at a stellar pace in the years to follow. Advancements in molecular biology have paved the way for revenue inflow into the global spatial genomics and transcriptomics market. The need for studying genetic patterns in humans, animals, and plants has generated new opportunities for market expansion, Genetic engineering has emerged as a robust domain within nascent biological sciences, creating room for experimentation and analysis. The applications of genomics in molecular biology and genetic studies has given a thrust to market expansion.

In this custom review, TMR Research delves into the extrinsic and intrinsic trends that are shaping the growth graph of the global spatial genomics and transcriptomics market. The domain of biological sciences has encapsulated new technologies for studying sizes, compositions, and archetypes of human genes. This is playing a vital role in driving sales across the global spatial genomics and transcriptomics market. This review also assesses the impact of advancements in genetic engineering to decode market growth.

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Spatial Genomics & Transcriptomics Market: Notable Developments

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Spatial Genomics & Transcriptomics Market: Growth Drivers

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The importance of microbiology in genetic studies has created a boatload of opportunities for growth and expansion across the global spatial genomics and transcriptomics market. The use of spatial genomics to understand the structure and composition of genes has enabled the inflow of fresh revenues into the global market. Besides, the use of genetic studies in the domain of veterinary care has also generated humongous opportunities for market expansion. The study of human and animal genes often goes hand-in-hand for the purpose of core research and analysis.

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Spatial Genomics & Transcriptomics Market Demand is Expanding at a Stellar Pace in the Years to Follow - BioSpace

Roy Curtis: ‘If Mayo are to achieve immortality, it will require the greatest act of defiance Croke Park has ever known’ – Independent.ie

Ernst Jngerhad most likely never heard of Mayo nor Gaelic football when, exactly 100 years ago, he drafted perhaps the most savage and lyrically striking account of war ever committed to print.

et the stark, three-word title of the German World War One veteran's visceral recollection of life in the trenches feels like a perfect fit when considering what James Horan's side will encounter when they gallop over Croke Park's brow and into the eye of Dublin's murderous efficiency.

Storm of Steel.

If Mayo are to locate unmatchable glory, if they are to make matchwood of so many dire predictions, if they are to finally touch fantasy, it will require the greatest act of defiance the GAA's old house of worship has ever known.

They are required to defy gravity and logic and the apparently immutable laws of nature.

Only the kind of wild and admirable contrariness that is their calling card will convince Mayo they can bend the rules of physics.

Their most potent asset is that they stride onto a rectangle of grass that Dublin pitiless, incomparable, insatiable, magnificent Dublin - have transformed into a killing field, armed with the absolute conviction that they will win.

Aidan OShea and Cillian OConnor and Patrick Durcan will not doubt for even a nanosecond that they can take down Brian Fenton, Ciarn Kilkenny and Dean Rock.

It doesnt matter that such a mindset might seem illogical, the same kind of unhinged optimism that persuades a lion-tamer certain he will emerge unscathed from sticking his head between the jaws of a hungry jungle cat.

The very fact they wear a cape of defiance rather than the cloak of inferiority that is the uniform of choice for so many of Dublins trembling opponents offers Mayo an initial shield against the storm of steel enjoyed by few of their peers.

Is it enough to counter the forest of statistics insisting those green and red ambitions are bound once more for the boneyard?

Almost certainly not.

Mayo have not beaten Dublin in 16 attempts spanning more than eight years.

The odds-makers think it is twice as likely that Dublin will win by more than 10 points than lose by one.

Meath, like Mayo, relegated from Division One in late autumn, were pulverised by Dessie Farrell's Sky Blue junkyard car crusher in the Leinster final. Long before the end of a 21-point slaughter, the more squeamish among the TV audience felt obliged to avert their eyes.

Horan understands that he faces an opponent propelled by the kind of ambition that knows no bounds, that it is Dublins way to keep throwing dizzying punches long after their concussed opponent has been counted out.

Close

Mayo manager James Horan. Photo: Sportsfile

SPORTSFILE

He knows too that Fenton, Kilkenny, Rock, Stephen Cluxton, James McCarthy and Con OCallaghan all long ago secured residency in footballs hall of fame.

Somehow, still, Horan will not permit himself to surrender to futility as he prepares for one of the great challenges in all of sport.

His county's back catalogue in this match-up is one vaccine against despair.

In 2016 and 2017, the teams contested three All-Ireland finals in 12 months. One of those convulsive contests finished in a draw, the other two were settled by a point. At the end of nearly four hours of superior, compelling football, the aggregate score was 4-41 (53) to 2-45 (51) in Dublins favour.

On one level, the strength of Mayos conviction, that unbreakable belief that can plant their standard on football's highest peak, can seem like a kind of madness.

It is, after all, 69 years since they were champions of Ireland. Some 13 counties among them Louth, Offaly and Derry have lifted Sam since 1951.

If national silverware was the only measure, then Mayo could be accused of marching with a strut that is wildly out of proportion to their achievements.

But then, there is more than a single yardstick to calculate greatness.

Competitive courage, a superhuman defiance, a Terminator-like capacity, even when ruinously disabled, to reform and regather and return the following year to battle againthese are the qualities that underpin Mayo.

And have earned them the affection of so many neutrals.

Even more than Kerry, though the latter bagged the only All-Ireland not won by Dublin since 2013, they have found a way to go toe-to-toe time and again with the greatest force the game has ever known.

Beyond that history of insolence, the bedrock on which to construct any kind of solid argument the underdogs can bark loudest on Saturday is non-existent.

Examine the relative strengths of both teams and this can be viewed as a contest between an electric chair and a doomed death row convict.

Dublin's third quarter eruption of brilliance in last year's semi-final, a detonation of shock and awe pyrotechnics that made a mushroom cloud of Mayo hopes, seemed to radically reset the parameters of their decade-long rivalry.

Close

Mayo's Tommy Conroy

SPORTSFILE

Any wargaming of likely outcomes based on that game would have Mayo risking not only defeat but destruction.

Dublin, it is true, will not face Mayo's broken class of 2019.

Horan has re-seeded, adding youth, pace and forward potency. Eoghan McLaughlin, Oisin Mullin and Tommy Conroy offer a fresh line of resistance.

Even still, the sense is that Mayo are no longer the force of old certainly not defensively.

Tipperary, a Division Three team, created near double-figure goal chances in the All-Ireland semi-final.

Every time they ran at Mayo, the green and red gates seemed to slide invitingly open.

Offer Dublin a similar scent of blood and their carnivore instincts will take over, inflicting instant annihilation.

The kind visited on even those warriors of rare conviction, who go eyeball-to-eyeball with something as ferocious and unceasing as Dublin's storm of steel.

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Roy Curtis: 'If Mayo are to achieve immortality, it will require the greatest act of defiance Croke Park has ever known' - Independent.ie

Covid Is Reshaping Death. And Maybe Life. – The Wall Street Journal

The desire to die in the presence of those we love is so deeply ingrained that during the Civil War, soldiers dying on battlefields pulled out family photographs to create the experience in their imaginations. In 2020, the face-to-face family death vigil largely became an impossible luxury. While relatives wept on sidewalks, people with Covid died in isolation by the tens of thousands, attended only by masked nurses and aides holding iPads and dressed in hazmat suits.

Just as deeply honored, for millennia, is our unspoken promise to handle the bodies of our dead with reverence. So visceral is this obligation that in the powerful Greek tragedy Antigone, the heroine knowingly courts imprisonment when she ventures onto a battlefield to give her disgraced brother a proper funeral, rather than leaving his body to be chewed up by birds and dogs and violated. Now, during Covid surges, the dead are zipped into body bags and stacked in refrigerator trucks.

During the Great Plague of London in the mid-1600s, church bells tolled day and night, each peal announcing a single death to everyone within earshot. Today the death toll arrives quietly, in a daily email from my county health department.

The novel coronavirus is the third-leading cause of death in the U.S. this year and responsible for roughly a tenth of all American deaths in 2020. We are in for some horrifying months before widespread mass vaccination reduces these grim statistics. Death, which once seemed tamable, hidden away in nursing homes, and pushed into the upper reaches of the lifespan, came out of the closet in 2020: contagious, capricious and striking people of every age. This is forcing a profound reckoning with the limits of free will, American exceptionalism and technological utopianismin short, with human powerlessness in the face of death.

This is a profound surprise for many people in the U.S. and the wealthy developed world. For three-quarters of a century, deadly contagions (with the exception of polio and AIDS) had mainly been problems for another century or continent. Average lifespans for the comfortably off were lengthening. Futurists, misreading the science, wistfully imagined that ordinary people might someday live to be 120 or even 150. Alphabet Inc.s Calico division and Oracle Corp. founder Larry Ellison sank millions of dollars into research on extending longevity. One of the stated goals of Mark Zuckerberg and Priscilla Changs $3 billion foundation is to cure, prevent, or manage all diseases by the end of the 21st century.

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Covid Is Reshaping Death. And Maybe Life. - The Wall Street Journal

Excerpts From Edwin Wilsons Magic Time – The Wall Street Journal

Dec. 17, 2020 6:13 pm ET

As a schoolboy in the early 1940s, Edwin Wilson saw Al Jolson drop to his knees in the footlights of the Shubert Theatre and belt out Mammy. As a young man he thrilled to the original Broadway productions of A Streetcar Named Desire, Death of a Salesman, Oklahoma!the standard American repertoire aborning. In his memoir Magic Time, Mr. Wilson, now 93, writes fondly of how these and other electrifying moments of New York playgoing led him, circuitously, to Yale Drama School and a lifetime of teaching and making theater happenwriting scripts, directing and producing plays, encouraging young talent, and writing or cowriting three of the most enduring college theater textbooks. At the heart of the book, however, is an account of his long tenure as The Wall Street Journals theater critic, from 1972 to 1994. It was work that, with its first-night tickets and aisle seats, returned him to what drew him to the dramatic arts in the first placean audience members experience of living theater. More than a reminiscence, Magic Time is also a mini-anthology of Mr. Wilsons favorite reviews and other writings for this paper. The critic excelled at profiles, interviews and memorial tributes, four of which are adapted here.

On composer Richard Rodgers, who died at 77 in 1979: For the first 22 years of his professional life, Rodgers collaborated with Lorenz Hart. With Hart, he always wrote the music first, spinning out a waltz, a ballad or a patter song, then Hart would add his intricate, urbane lyrics: Well go to Greenwich / where modern men itch / to be free. When Hart, due to personal problems, could no longer serve as his lyricist, Rodgers turned to Oscar Hammerstein II, and began a second remarkable collaboration. Beginning with Oklahoma! in 1943, Rodgers and Hammerstein turned out hit shows year after year: Carousel, South Pacific, The King and I, The Sound of Music. Where Harts lyrics had been sophisticated, often bittersweet, Hammersteins were sincere, straightforward and sometimes even overly sentimental. But Rodgers adjusted: his music took on a warmer, down-to-earth quality. He even changed his composing habits; instead of writing the music first, he set his tunes to Hammersteins completed lyrics. Through all of these changes one thing remained constant: Rodgerss great gift for melody and his solid musicianship. Alec Wilder, whose book American Popular Song is probably the definitive study of the subject, made a detailed analysis of more than a hundred Rodgers tunes. Afterward he wrote: I am more than impressed and respectful: I am astonished. His songs, Mr. Wilder said, revealed a higher degree of excellence, inventiveness and sophistication than any other writer I have studied. Sir Thomas Beecham once remarked, if an opera cannot be played by an organ-grinder, it is not going to achieve immortality. Richard Rodgerss songs have been played by organ-grinders, sung by Ella Fitzgerald, and performed by a thousand dance bands. They have more than met the test and have achieved their own form of immortality.

From a 1987 piece on George Abbott, an insight into the directors ear for talent, elicited from the deadpan comic actress Nancy Walker: I was 19 when I went to tryouts for Best Foot Forward. The last thing in the world I wanted to be was funny; no girl 19 wants to be funny. But Mr. Abbott listened to me sing and later cast me. In his response to my singing those 32 bars he told me who I was. Beginning right there he defined my career and, in fact, my whole life. And I am eternally grateful. . . . Mr. Abbotts genius is that he never let me know I was funny.

A memory of seeing the 1949 London production of Christopher Frys verse play The Ladys Not for Burning, starring John Gielgud: It just so happened that in the Fry play two young actors were making their stage debuts: Richard Burton and Claire Bloom. Some 40 years later, when I was interviewing Claire for a television series, I told her I had seen that production. Every night we were in that show, she said, there was one scene where I could not resist looking at the audience. It was a scene in which John, in that sonorous, unmistakable voice, would be declaiming blank verse . . . but the audience would be staring, not at Gielgud but at Richard silently scrubbing the floor.

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Excerpts From Edwin Wilsons Magic Time - The Wall Street Journal

The pivotal moment in 2018 that led Waterford on the path to possible 2020 immortality – Independent.ie

There was a sliding-door moment in hurling in September 2018 which led to Waterfords surprise appearance in todays All-Ireland final.

ichael Ryan had stepped down as Tipperary boss after a torrid summer. Liam Cahill appeared in pole position to succeed him

As a player Cahill never realised the potential he displayed as a 19-year-old, when he won an All-Star in 1996 after a sensational rookie season.

When Tipperary won the All-Ireland five years later, Cahill, who ought to have been at his prime, was a non-playing substitute.

But as a coach he had built up an impressive CV by 2018. He first became involved with Declan Ryans minor management team in 2007 when Tipperary won the All-Ireland. He spent two more years as a minor selector before returning as its boss in 2013.

After the team was beaten by Clare in the 2014 championship, Cahill decided he needed to share the workload. He recruited Toomevara native Mickey Bevans to his management team.

They had played together on under-age Tipperary teams and though Bevanss inter-county career never got off the ground, he became a pivotal player on the Toomevara club team which won 10 county titles.

Bevanss attention to detail had impressed everybody he worked with. His first senior coaching job in Tipperary was with the Upperchurch Drombane club in 2013.

Before he met the players for the first time, he asked the club chairman for a photograph of each player so he could greet them by name at their first meeting.

In an interview with the Sunday Times, club chairman James Barry said Bevans made a lasting impact during his two years. The whole culture of the club changed. He set standards. The impression he made was huge.

Cahill and Bevans hit it off straight away and during the next four years Tipperary won one All-Ireland minor title (2016), one All-Ireland U-21 (2018) and one All-Ireland U-20 title (2019).

Ultimately, Cahill didnt get the Tipperary senior gig which was hardly surprising. Once Liam Sheedy who managed the team when they ended Kilkennys bid for five-in-row in 2010 indicated he was interested in returning, he was a shoo-in for the job.

Cahill stayed on as the Tipp U-20 manager for another season. But when Waterford came calling last autumn, he jumped at the chance to manage a county senior team for the first time.

His home in Ballingarry is less than an hours drive from Waterford and his right-hand man Bevans was familiar with hurling in the Deise due to his involvement with Waterford IT teams in the Fitzgibbon Cup.

It was still a risky move. Waterford had contested the 2017 All-Ireland final but their stock had plummeted in the meantime. Cahill was their third manager in 13 months.

Derek McGrath had departed after the 2018 Munster campaign, during which his side had failed to win a game.

Initially, they did well under his successor Praic Fanning and reached the league final but were well beaten by Limerick (1-24; 0-19). The championship turned into a car crash after they suffered a one-point loss to Clare at Walsh Park in the first round. They lost their other three games by an aggregate of 51 points.

Fanning opted not to see out the second year of his term. Nonetheless, his role in the development of the current team should not be dismissed Conor Prunty, Calum Lyons and Jack Prendergast all made their championship debuts in 2019.

There was a sense of shock and awe about Cahills arrival in Waterford last October. He dropped long-time captain Noel Connors as well as forward Maurice Shanahan. It had the desired impact, as 2017 Hurler of the Year Austin Gleeson acknowledged.

When I heard that Noel and Maurice were cut from the panel, it was a signal of intent of what way the lads wanted to go. Its after making us train harder, a lot harder," he said.

The fear is there that hes laying down his own marker. Its something that maybe we needed, needed big time, to make us just as hungry again, said Gleeson, who in his own words cancelled Christmas to focus on training.

Outside the group John Mullane was one of the first to realise that things might be different with Cahill.

Just after Christmas he dropped down to Carriganore to watch them train. Writing in the Irish Independent on the day of their semi-final against Kilkenny, Mullane recalled what he saw on that freezing night.

What unfolded in the 90 minutes was a high-tempo session based around tackling and movement with Cahill and Mikey Bevans proactive throughout every bit of it. Then came the running with Offaly man Martin Bennett, whose name mightnt be too familiar to many, but he put them through their paces.

It was a session where I turned around to one chap taking notes and said: Thank God Im well retired. My first thought returning to the car was that Waterford had two unbelievable men in charge that any other county would give their right arm for.

The odds still looked stacked against them. Three of Waterfords most experienced players Michael Brick Walsh, Philip Mahony and Barry Coughlan retired, while the cruellest blow of all came on the eve of the championship when free-taker Pauric Mahony damaged his cruciate ligament for the second time in his career.

By then Meath native Jack Fagan and former Brighton and Hove Albion soccer player Dessie Hutchinson had established themselves as first-team players.

Cahill didnt insist on the team adhering to a defined system, which had been the hallmark of Derek McGraths reign.

Instead, he trusted the players to rely on their instincts and they responded magnificently and none better than Stephen Bennett.

When he missed a couple of early frees against Cork in the Munster semi-final the prophets of doom were ready to pounce. But once he got his eye in, everything has flowed culminating in his tour-de-force performance against Kilkenny.

But it is Limerick rather than the Cats who are setting the standards in hurling nowadays.

Waterford pushed them in the Munster final, though they never looked like winning the game. They failed to raise a green flag and scored just 11 points from play.

But, regardless of whether they can complete the fairy tale this afternoon, Cahill and Bevans have announced themselves as a formidable force in hurling management. Their day in the sun will come.

Online Editors

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The pivotal moment in 2018 that led Waterford on the path to possible 2020 immortality - Independent.ie

Roy Curtis: ‘If Mayo are to achieve immortality, it will require the greatest act of defiance Croke Park has ever known’ – Sunday World

rnst Jnger had most likely never heard of Mayo nor Gaelic football when, exactly 100 years ago, he drafted perhaps the most savage and lyrically striking account of war ever committed to print.

Yet the stark, three-word title of the German World War One veteran's visceral recollection of life in the trenches feels like a perfect fit when considering what James Horan's side will encounter when they gallop over Croke Park's brow and into the eye of Dublin's murderous efficiency.

Storm of Steel.

If Mayo are to locate unmatchable glory, if they are to make matchwood of so many dire predictions, if they are to finally touch fantasy, it will require the greatest act of defiance the GAA's old house of worship has ever known.

They are required to defy gravity and logic and the apparently immutable laws of nature.

Only the kind of wild and admirable contrariness that is their calling card will convince Mayo they can bend the rules of physics.

Their most potent asset is that they stride onto a rectangle of grass that Dublin pitiless, incomparable, insatiable, magnificent Dublin have transformed into a killing field, armed with the absolute conviction that they will win.

Aidan OShea and Cillian OConnor and Patrick Durcan will not doubt for even a nanosecond that they can take down Brian Fenton, Ciarn Kilkenny and Dean Rock.

It doesnt matter that such a mindset might seem illogical, the same kind of unhinged optimism that persuades a lion-tamer certain he will emerge unscathed from sticking his head between the jaws of a hungry jungle cat.

Close

Michael Fitzsimons of Dublin battles with Mayo talisman Cillian O'Connor

SPORTSFILE

The very fact they wear a cape of defiance rather than the cloak of inferiority that is the uniform of choice for so many of Dublins trembling opponents offers Mayo an initial shield against the storm of steel enjoyed by few of their peers.

Is it enough to counter the forest of statistics insisting those green and red ambitions are bound once more for the boneyard?

Almost certainly not.

Mayo have not beaten Dublin in 16 attempts spanning more than eight years.

The odds-makers think it is twice as likely that Dublin will win by more than 10 points than lose by one.

Meath, like Mayo, relegated from Division One in late autumn, were pulverised by Dessie Farrell's Sky Blue junkyard car crusher in the Leinster final. Long before the end of a 21-point slaughter, the more squeamish among the TV audience felt obliged to avert their eyes.

Horan understands that he faces an opponent propelled by the kind of ambition that knows no bounds, that it is Dublins way to keep throwing dizzying punches long after their concussed opponent has been counted out.

He knows too that Fenton, Kilkenny, Rock, Stephen Cluxton, James McCarthy and Con OCallaghan all long ago secured residency in footballs hall of fame.

Somehow, still, Horan will not permit himself to surrender to futility as he prepares for one of the great challenges in all of sport.

His county's back catalogue in this match-up is one vaccine against despair.

In 2016 and 2017, the teams contested three All-Ireland finals in 12 months. One of those convulsive contests finished in a draw, the other two were settled by a point. At the end of nearly four hours of superior, compelling football, the aggregate score was 4-41 (53) to 2-45 (51) in Dublins favour.

On one level, the strength of Mayos conviction, that unbreakable belief that can plant their standard on football's highest peak, can seem like a kind of madness.

It is, after all, 69 years since they were champions of Ireland. Some 13 counties among them Louth, Offaly and Derry have lifted Sam since 1951.

If national silverware was the only measure, then Mayo could be accused of marching with a strut that is wildly out of proportion to their achievements.

But then, there is more than a single yardstick to calculate greatness.

Competitive courage, a superhuman defiance, a Terminator-like capacity, even when ruinously disabled, to reform and re-gather and return the following year to battle againthese are the qualities that underpin Mayo.

And have earned them the affection of so many neutrals.

Even more than Kerry, though the latter bagged the only All-Ireland not won by Dublin since 2013, they have found a way to go toe-to-toe time and again with the greatest force the game has ever known.

Beyond that history of insolence, the bedrock on which to construct any kind of solid argument the underdogs can bark loudest on Saturday is non-existent.

Examine the relative strengths of both teams and this can be viewed as a contest between an electric chair and a doomed death row convict.

Dublin's third quarter eruption of brilliance in last year's semi-final, a detonation of shock and awe pyrotechnics that made a mushroom cloud of Mayo hopes, seemed to radically reset the parameters of their decade-long rivalry.

Any war-gaming of likely outcomes based on that game would have Mayo risking not only defeat but destruction.

Dublin, it is true, will not face Mayo's broken class of 2019.

Horan has re-seeded, adding youth, pace and forward potency. Eoghan McLaughlin, Oisin Mullin and Tommy Conroy offer a fresh line of resistance.

Even still, the sense is that Mayo are no longer the force of old certainly not defensively.

Tipperary, a Division Three team, created near double-figure goal chances in the All-Ireland semi-final.

Every time they ran at Mayo, the green and red gates seemed to slide invitingly open.

Offer Dublin a similar scent of blood and their carnivore instincts will take over, inflicting instant annihilation.

The kind visited on even those warriors of rare conviction, who go eyeball-to-eyeball with something as ferocious and unceasing as Dublin's storm of steel.

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Roy Curtis: 'If Mayo are to achieve immortality, it will require the greatest act of defiance Croke Park has ever known' - Sunday World

The meaning of our holiday traditions – San Lorenzo Valley Press-Banner

We all celebrate the holidays in a different way. Each family has its own traditions and warm memories from years gone by. Some of us celebrate Christmas, some Hanukkah, some Kwanzaa. Many of our traditional Christmas customs originate from Winter Solstice celebrations. The plants associated with each are an important part of tradition and symbolism.

Winter solstice is Dec. 21. Solstice literally means Sun Stands Still and for a few days around this time of year the sun appears to stand still in the sky. Nearly all cultures and faiths have some sort of winter solstice celebration. They have been with us for thousands of years starting at the beginning of agriculture among people who depended on the return of the sun. We have incorporated many of the plants from traditional winter solstice celebrations into our ownholly, ivy, evergreens, rosemary and mistletoe. How did this come about?

Holly remains green throughout the year when deciduous trees like the oak shed their leaves. Decorating with it throughout the home has long been believed to bring protection and good luck. Placing a ring of holly on doors originated in Ireland since holly was one of the main plants that was green and beautiful with its red berries at this time of year. Norseman and Celts planted a holly tree near their homes to ward off lightning strikes. The crooked lines of holly leaves gave rise to its association with lightning and in fact holly does conduct lighting into the ground better than most trees.

Like other evergreens, ivy symbolizes immortality and eternal life. In England it is traditionally used in kissing balls with holly and mistletoe. It has also stood for fidelity, healing and marriage. Ancient Romans thought it brought good luck and joy. It was worn as a crown or fashioned into a wreath or garland.

Evergreen trees play a role in solstice celebrations. Early Romans and Christians considered the evergreen a symbol of the continuity of life. Fir, cedar, pine boughs and wreaths were used to decorate homes. Small gifts were hung from the branches. This may have been where the Christian tradition of decorating an evergreen tree or Yule tree in December originated. Other sacred trees of the solstice are yew, birch, arborvitae and ash.

We often see rosemary plants trained into a Christmas tree shape. Rosemary is evergreen in the winter and blooms at the same time making it the perfect plant for the holidays. Traditionally rosemary was spread on floors at Christmas as people walked over the herb releasing the fragrant scent and filling the home with blessings and protection.

How did our fascination with mistletoe get started? From earliest times it has been one of the most magical, mysterious and sacred plants of Greeks, Celts, Scandinavia, England and European folklore in general.The Druids believed the mistletoes magical powers extended beyond fertility. It was believed to cure almost any disease and was known as the all healer. Sprigs fixed above doorways of homes were said to keep away lightning and other types of evil. Because the plant is parasitic and has no roots it was believed that it grew from heaven.

Kissing under the mistletoe probably came from the Greek/Roman belief that it bestowed fertility and had life-giving power. In Scandinavia it was considered a plant of peace under which enemies could declare a truce or fighting spouses could kiss and make up. However this tradition originated, its a good one.

The Yule log dates back to the Saxons and Celts. Oak trees represented strength, endurance, protection and good luck. It was the most sacred tree of Europe. On the eve of the winter solstice, the longest night of the year, people would keep a huge oak log burning for 12 hours. They would toss oak twigs and acorns into the fire, shout out their hopes and resolutions for the coming New Year and sing Yuletide carols. A piece of the Yule log was saved to start the fire the following year.

Its traditional for us to have some poinsettias in the house for the holidays but they dont have a very long history of European tradition like other plants because poinsettia is a native of Mexico. In the 1820s President Andrew Jackson appointed Joel Roberts Poinsett as the first U.S. Ambassador to Mexico. In 1828 he found a beautiful shrub with large red flowers growing next to a road. He took cuttings and brought them back to his greenhouse in South Carolina. Because the leaves or bracts turn bright red around Christmas time they have been used as decorations for the holidays ever since.

Traditional plants symbolic of Hanukkah are the citron, myrtle twigs, willow twigs and palm fronds. The Four Species are waved together along with special blessings as part of the synagogue service or at home.

Kwanzaa is a Swahili word that means first and signifies the first fruits of the harvest. With ears of corn, fruit and nuts it is observed for seven days during the last week of December and celebrates the fruit or accomplishments coming out of the year of labor. Each family celebrates Kwanzaa in its own way, but celebrations often include songs and dances, African drums, storytelling, poetry reading and a large traditional meal. Observed by people of all faiths it is a celebration of African roots.

Around the world, holiday celebrations have their own special meaning. So whether you Zoom with friends and distant family or celebrate with your Pod, embrace your own traditions and have a wondrous holiday.

Jan Nelson, a landscape designer and California certified nursery professional, will answer questions about gardening in the Santa Cruz Mountains. Email her at [emailprotected], or visit jannelsonlandscapedesign.com.

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The meaning of our holiday traditions - San Lorenzo Valley Press-Banner

Many rebirths continue to live within us – The Times of India Blog

The other night I had a dream about my mother, who died over 30 years ago. She looked the same as she had done then, and my dream filled me with happiness, not the sorrow of loss.

Like most people I often dream of the dead. But on this particular occasion I had a moment of half-wakefulness that limbo between sleep and consciousness when the mind is strangely lucid in which a thought came to me: Such dreams are reminders that our dead live on within us.

The next morning, fully awake, I remembered that thought, and tried to figure out what it meant, if anything at all.

Freud interpreted dreams as a psychic mechanism by which the subconscious processes unresolved impulses and issues and brings them to the light of day for resolution.

So, what meaning would I give, in a non-Freudian sense, as to the way my personal dead, those I have known, live again in my dream state, in my subconscious? Could this be a metaphor for a different take of the doctrine of reincarnation, central to much of what is called Hinduism, and in Buddhist philosophy?

Ive always been a bit chary of the belief that the cosmic wheel of karmic consequence causes us to be reborn, over and over, until we finally attain moksha, or nirvana.

To me the idea of reincarnation has always smacked of a sort of spiritual charity shop where a succession of physical forms, like discarded clothes, are passed on from person to person until they become totally outworn and are no longer needed.

But there could be another, simpler view of reincarnation: that we are reborn not in individual physical terms, not as ourselves in the cast-off clothing of different mortal flesh, but in the thoughts and deeds of those we have encountered and who have influenced us, for good or ill, during our lives.

Schopenhauer in his The World as Will and Idea reinterprets reincarnation as a continuum of consciousnesses, each assimilating and subsuming others, like a baton passed on from runner to runner in a relay race.

In Boris Pasternaks novel, the eponymous Dr Zhivago expounds his take on spiritual immortality by suggesting that all of us live on, are reborn, in the ways in which we continue to mould, consciously or otherwise, the lives of others who come after us, not just as our genetic descendants but those who are the offspring of our memes, our mental genes, which we leave behind like footprints on the shifting sands of time for others to follow.

Spiritual masters like Christ, Prophet Mohammed, Guru Nanak, Mahavira and others, have left behind a memetic legacy as a foundation on which their followers have built faith systems. Mohandas Gandhis active philosophy of ahimsa and satyagraha was derived from Tolstoy and Thoreau, and in turn passed on to Martin Luther King Jr, and others.

Our parents, teachers, friends, even strangers we come into tangential contact with, live on in us by being the wellspring of what we think and do, often without our being aware of our source of motivation.

When we dream of those who are no more, they are not spectres of the past, but are an indispensable part of our own living selves, as we ourselves will be to others, in the flowing river of life called reincarnation.

Views expressed above are the author's own.

END OF ARTICLE

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Many rebirths continue to live within us - The Times of India Blog

Chinese TV Animation Is Starting to Corner the Global Boys Love Market – CBR – Comic Book Resources

Thanks to the likes of Mo Dao Zu Shi and Heaven Official's Blessing, Chinese animation is breaking through in a big way -- particularly BL series.

Donghua, Chinese animation, has rocketed in popularity in part thanks to the BL series Mo Dao Zu Shi (The Grandmaster of Demonic Cultivation in English). Another series based on novels by author Mo Xiang Tong Xiu, Heaven Official's Blessing, is also gaining popularity, being the donghua streamed by Funimation. These popular series have opened the world to danmei (Chinese BL) and have become phenomenons on Tumblr and Twitter. Their global popularity seems to have put danmei on the radar -- giving Chinese comics and animation a wider platform, similar to the popularity Korean comics (manhwa) have received over the years as well.

Danmei, similar to Japan's yaoi genre, is somewhat controversial. Male characters are often put in heteronormative "masculine" and "feminine" roles, perpetuating harmful stereotypes. Danmei is usually made by heterosexual women for the straight female gaze, rather than by LGBTQ creators. However, the genre is also considered a positive step in representation as it pushes LGBTQ romance into the Chinese mainstream.

RELATED: Heaven Official's Blessing Sheds More Light on Xie Lian's Underdog Status

Though homosexuality is legal in China, media with LGBTQ elements tends to be censored because of the conservative government. Danmei series, which contain subtle elements of boys' love, are small but significant breakthroughs -- especially as the live-action danmei The Untamed became one of China's biggest dramas. As danmei becomes increasingly popular, to the point of a global sensation, it may both increase LGBTQ acceptance and lessen China's censorship laws.

Mo Dao Zu Shi and Heaven Official's Blessing are also both part of the Xianxia genre, a genre influenced by traditional Chinese mythology. The protagonists are often "cultivators," people trained in martial and mystical arts, that seek immortality (the closest translation for the concept of "Xian") and supernatural powers. Mo Dao Zu Shi's protagonist, Wei Wuxian, attains Xian only to be killed -- but is then summoned into the world once again, taking over host Mo Xuanyu's body and eventually being involved in a complex conspiracy. Meanwhile, in Heaven Official's Blessing, the character Xie Lian is shunned by the Heavens twice and attempts to ascend a third time.

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Chinese TV Animation Is Starting to Corner the Global Boys Love Market - CBR - Comic Book Resources

Latest Overwatch Patch Features D.Va and Reinhardt Buffs – Hotspawn

A brand new patch was released for the Overwatch retail servers on Thursday, which included a small nerf for Baptiste and a moderate buff to both D.Va and Reinhardt. All of the new changes were tested on the most recent experimental card over the past week before being finalized and released for all modes.

D.Va received a buff in Thursdays patch which changed her health pool from 200 armor with 400 health to 300 armor with 300 health. Armor blocks five damage from any attack that deals more than ten damage, and it halves the damage of any attack that deals less than ten damage. This means that armor is an effective tool for countering shotgun pellets, damage-over-time beams, and weapons that rely on hitting a lot of shots quickly, because armor has a more significant effect on lower damage pellets than high damage hits. By increasing her armor, this change should make D.Va a more durable hero, which is good since she has been feeling pretty squishy for a tank in recent months.

The developers gave Reinhardt a similar buff, increasing his armor from 200 to 250. Post change, Reinhardt has a total health pool of 550 health, 250 of which is armor and 300 of which is health. They also increased the damage of his Rocket Hammer from 75 to 85 damage per hit. The buff to his damage means he can kill a 250 health enemy in three hits instead of four. When combined, these two buffs should make a significant improvement on Reinhardts frontline abilities, since he will be able to deal more damage and take more damage.

Finally, the developers nerfed Baptiste in this Overwatch patch by lowering the health on his Immortality Field from 200 to 150 and increasing the cost of his ultimate, Amplification Matrix, by 15%. He recently received a buff to his Biotic Launcher and Amplification Matrix in the last patch on November 18th. Those adjustments made Baptiste much more effective than the developers had originally intended, so they decided to nerf him by making his Immortality Field slightly worse and making it harder to earn his ultimate.

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Latest Overwatch Patch Features D.Va and Reinhardt Buffs - Hotspawn

#ComicBytes: Who is Gorr the God Butcher? | NewsBytes – NewsBytes

Marvel fans around the world recently celebrated the announcement of several new projects and additions to the MCU.

However, the best revelation till now has been about the villain of Thor: Love and Thunder, Gorr the God Butcher, who will be portrayed by Christian Bale.

As the name suggests, Gorr kills Gods.

Read ahead to know why and how he does it.

A young Gorr grew up on an unnamed planet with a crippled leg.

Although he knew that he must pay homage to Gods to keep his family safe, he questioned his faith when both of his parents die.

Years later, when his partner Arra and their kids also perish, Gorr loses all faith in the Gods, which results in his tribe disowning him.

After being shunned by his tribe, Gorr wanders around the deserts where he witnesses two Gods on the ground.

When the surviving God asks Gorr for help, he asks, "Where were you when my children were starving? Where were you when we needed our Gods?!"

Then suddenly, the dead God's weapon binds itself to Gorr that enables him to stab the injured God.

The weapon that binds itself to Gorr as a symbiote is the All-Black the Necrosword. In fact, all symbiotes of Marvel Comics (like Venom) descend from this weapon.

Necrosword allows for immortality, flight, regeneration, conjuring weapons, and other superhuman abilities.

Gorr uses his new-found abilities to create versions of his dead wife and son, along with his own guards called the Black Berserkers.

After his first kill, Gorr decides to become the God Butcher, and eventually faces the Asgardian God of Thunder, Thor.

On the first encounter, he almost kills young Thor. During the second time, Thor teams with two other versions of himself from different eras to defuse his Godbomb and kill him.

On their final encounter, Thor defeats a resurrected Gorr.

In comics, Gorr is a mortal without his memories after the destruction of the All-Black symbiote.

He is living his life in peace with the Sky Lords of Indigarr.

Gorr has proved to be a mighty adversary for Thor and has had a huge impact on his life.

It would be interesting to see how Bale brings this character to life on-screen.

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#ComicBytes: Who is Gorr the God Butcher? | NewsBytes - NewsBytes

What is Genetic Medicine

Genetic medicines are genetic materials such as DNA and RNA delivered into the body as a therapeutic. They are a promising new class of medicine that was not possible even a short time ago.

Advancements in science and technology are changing the way we define disease, develop drugs and prescribe treatments with an explosion of insights into the role of genetics in infectious diseases, cancer and rare diseases. Genetic medicines are an emerging technology with the potential to be developed as personalized medicines and for mass administration, by teams with a wide range of capabilities.

2020 Nobel Laureate Dr. Michael Houghton on Lipid Nanoparticle RNA Vaccines

Genetic vaccines are a new class of medicine that introduces new genes to the body to produce key antigens that help the body fight infections. Genetic vaccines can be broken down into two broad categories, viral vectors and non-viral vectors that use synthetic methods such as lipid nanoparticles deliver genes into cells. Non-viral vectorsoffer some advantages of viral vectorsincluding the ability to deliver larger genes, reduced biosafety concerns, and simplified synthetic production.

Watch the Full Webinar

Particularly, mRNA vaccines are a recent innovation in genetic medicine but are now at the forefront of the many vaccine technologies for the COVID-19 pandemic. mRNA vaccines use non-viral vectors to deliver the drug into the body which leads to safer and fast-to-develop vaccines. The mRNA vaccines at the forefront of the COVID-19 pandemic show how genetic vaccines have not only the potential for high potency, low-cost manufacturing and safe administration but also the capacity for rapid development.

A promising application for genetic vaccines is self-amplifying mRNA (saRNA), a vaccine platform that uses the human body toamplifythe vaccine and produce vaccine antigens. saRNA can offer a potent vaccine in extremely small doses due to its ability to amplify itself inside the body.

All these advantages of the mRNA vaccines translate into the development of other genetic medicines. The search for a pandemic vaccine has accelerated all genetic medicines by highlighting their advantages on the world stage.

Learn More About Genetic Vaccines

Samuel Clark, PNI Director of R&D, on how an mRNA gene therapy can work.

Gene therapy is the introduction of genetic material into the body to modify how proteins are expressed to treat a disease. Gene therapy is a promising application for genetic medicines for a number of diseases such as inherited disorders, viral infection and cancers. This technique allows doctors to treat a disorder by delivering a gene into a patient instead of using drugs or surgery.

Researchers are testing several approaches to gene therapy, including:

Replacing a disease-causing mutated gene with a healthy gene

Inactivating, or knocking out, a malfunctioning mutated gene

Introducing a new gene to help fight a disease

The amount of gene therapies being developed have continued to grow with 352 gene therapy in clinical trialsglobally at the end of last year. The FDA expects to see a doubling of new gene therapy applications each year. Scott Gottlieb, the former FDA commissioner, predicts that by 2025 the US would be approving between 10 and 20 gene therapies each year.

Learn More About Gene Therapy

In 1989, A National Institutes of Health (NIH) approved study provided evidence for the first time that human cells could be genetically modified and returned to the patient without harm. Since then, the industry has grown significantly with 22 gene and gene-modified cell therapies approved by regulatory bodies from various countries as of August 2019.

Precision NanoSystems can provide a customized, end-to-end pathway for your drug development programs, from lead candidate selection through early phase clinical trials to commercialization. Our proprietary technology platforms and comprehensive expertise enable researchers to work with a single, integrated partner to translate disease biology insights into non-viral genetic medicines. Minimizing handoffs throughout the process results in faster timelines and reduced costs.

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Publication - Abstract

July 01, 2020

Journal of Controlled Release

G. Lou, G. Anderluzzi, S.T. Schmidt, S. Woods, S. Gallorini, M. Brazzoli, F. Giusti, I. F...

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May 08, 2020

Vaccines

G. Anderluzzi, G. Lou, S. Gallorini, M. Brazzoli, R. Johnson, D.T. O'Hagan, B.C. Baudner a...

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April 02, 2020

Journal of Controlled Release

D. Chatzikleanthous, S.T. Schmidt, G. Buffi, I. Paciello, R. Cunliffe, F. Carboni, M.R. Ro...

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December 31, 2019

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C.B. Roces, M.T. Hussain, S.T. Schmidt, D. Christensen and Y. Perrie

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May 31, 2019

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R.A. Feldman, R. Fuhr, I. Smolenov, A. Ribeiro, L. Panther, M. Watson, J.J. Senn, M. Smith...

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May 18, 2019

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N. Jyotsana, A. Sharma, A. Chaturvedi, M. Heuser et al.

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What is Genetic Medicine

Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program, Two Preclinical Assets, and Novel Capsid…

Round was led by Sofinnova Investments with participation from Abingworth, Lightstone Ventures and all existing investors

Company expands board of directors and plans to build out team

DURHAM, N.C. and BOSTON, Dec. 16, 2020 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced it has closed an oversubscribed $55 million Series A financing led by Sofinnova Investments with participation from additional new investors Abingworth and Lightstone Ventures. Founding investors Hatteras Venture Partners and the Foundation Fighting Blindness RD Fund, along with existing investors Osage University Partners, University of Florida, and Manning Family Foundation, also participated in the round. Sarah Bhagat, PhD, Partner at Sofinnova, Jackie Grant, PhD, Principal at Abingworth, and Jason Lettmann, General Partner at Lightstone, will join Atsenas board of directors.

Proceeds will be used to advance Atsenas ongoing Phase I/II clinical trial evaluating a gene therapy for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children, as well as complete manufacturing development for Phase 3. In addition, the funds will enable Atsena to expand its team to support the research and development of novel gene therapies, including the progression of two existing preclinical programs in inherited retinal diseases toward the clinic and advancement of the companys innovative adeno-associated virus (AAV) technology platform.

We are grateful for the support of our new and existing investors and are encouraged by their enthusiasm for the potential of our technology to overcome the unique hurdles of inherited retinal diseases to prevent or reverse blindness, said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. The Series A financing provides financial runway to reach the key inflection point of reading out efficacy data from our LCA1 clinical trial. While we continue expeditiously advancing this trial and our preclinical programs, we are excited to accelerate our growth as a leading ophthalmic gene therapy company.

The Phase I/II LCA1 clinical trial is currently enrolling patients in the second dosing cohort. Atsena exclusively licensed the rights to the gene therapy from Sanofi, which originally licensed it from University of Florida. LCA is the most common cause of blindness in children. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease.

We believe Atsenas foundation in ocular gene therapy and potentially game-changing novel AAV vectors position the company to become a partner of choice, said Dr. Bhagat. Sofinnova is delighted to support Atsena and we look forward to helping the team further its mission to develop life-changing gene therapies for patients with inherited retinal diseases.

About Atsena TherapeuticsAtsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The companys ongoing Phase I/II clinical trial is evaluating a potential therapy for one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and has offices in Boston, MA and North Carolinas Research Triangle, environments rich in gene therapy expertise. For more information, please visit atsenatx.com.

About Sofinnova InvestmentsSince our founding in 1974, Sofinnova has been active in life science investing. We are a clinical-stage biopharmaceutical investment firm with approximately $2.3B in assets under management and committed capital. We invest in both private and public equity of therapeutics-focused companies. Our goal is to actively partner with entrepreneurs in both the U.S. and Europe, across all stages of company formation. From drug development and navigating the regulatory process to company building and IPO, we strive to be collaborative, meaningful board members, and excellent partners at every level. We seek to build world class companies that aspire to dramatically improve the current state of medical care today and ultimately, the lives of patients. Sofinnova has expertise investing in gene therapy companies, including investments in Spark, which developed the first approved gene therapy, Akouos, and Audentes, and Xylocor. For more information, please visit http://www.sofinnova.com.

About Abingworth Abingworth is a leading transatlantic life sciences investment firm. Abingworth helps transform cutting-edge science into novel medicines by providing capital and expertise to top caliber management teams building world-class companies. Since 1973, Abingworth has invested in approximately 168 life science companies, leading to more than 44 M&A/exits and close to 70 IPOs. Our therapeutic focused investments fall into 3 categories: seed and early-stage, development stage, and clinical co-development. Abingworth supports its portfolio companies with a team of experienced professionals at offices in London, Menlo Park (California) and Boston. For more information, visit abingworth.com.

About Lightstone VenturesLightstone Ventures is a leading venture capital firm investing in therapeutic-oriented companies across the life science spectrum, from breakthrough medical devices to novel drugs and biopharmaceuticals. Founded in 2012, Lightstone has been part of many successful new ventures from inception through commercialization and plays a critical role guiding and building successful healthcare companies. With a proven strategy and global footprint, the Lightstone team has been involved in several of the largest venture-backed life science exits over the last decade including: ALX Oncology, Acceleron, Ardian, Calithera, Claret Medical, Disarm, MicroVention, Nimbus, Plexxikon, Portola, Promedior, Proteolix, Ra Pharma, Tizona, Twelve and Zeltiq. For more information, visithttps://www.lightstonevc.com.

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Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program, Two Preclinical Assets, and Novel Capsid...

They thought their gene therapy failed. Instead, it spawned a medical mystery – Endpoints News

Jos-Alain Sahel was on a rare vacation in Portugal in the spring of 2018 when his phone rang with grim news: The gene therapy he had worked on for a decade, a potential cure for a rare form of blindness, had failed in a pivotal trial.

In the first minute, I was very disappointed, Sahel says. I said, well OK, its not working.

A failed trial in drug development is crushing but not unexpected, a tradeoff of doing business in biology. You examine the full data, go back to the drawing board and either abandon the effort or tweak and try again. Sahel, founder of four companies and the longtime head of the Vision Institute of Paris, was used to the process. But this time, when the full data came, he was bewildered.

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They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News

NeuBase Therapeutics Announces Positive Preclinical In Vivo Data for PATrOL-enabled Anti-gene for the Treatment of Myotonic Dystrophy Type 1 -…

In vivo data after single-dose IV administration demonstrate engagement with DMPK mRNA and broad rescue of mis-splicing across key transcripts

Findings provide support for hypothesized mechanism of action of anti-gene, which is designed to not degrade the DMPK transcript

Data further validate the potential of the PATrOL platform to develop highly targeted therapies that increase, decrease or change causal protein function

NeuBase management to hold conference call and webcast today, December 16, at 8:00 a.m. EST

PITTSBURGH, Pa., Dec. 16, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, today announced positive in vitro and in vivo preclinical data for its PATrOL-enabled anti-gene therapies for the treatment of myotonic dystrophy type 1 (DM1). These new data show that PATrOL-enabled Compound A can rapidly resolve mis-splicing without negatively impacting DMPK protein levels. They also support the potential of NeuBases anti-gene approach to comprehensively treat the underlying cause of DM1.

Despite the fact that the genetic basis of DM1 is well understood today, there is still an urgent need to find the first genetically-targeted, disease-modifying treatment option for affected patients, said Curt Bradshaw, Ph.D., Chief Scientific Officer of NeuBase. DM1 is caused by a genetic mutation in the DMPK gene leading to mis-splicing of a broad spectrum of genes and DMPK protein insufficiency. A treatment option that addresses mis-splicing while retaining functional DMPK protein levels may be key to treating all aspects of DM1.

Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, added, Using our proprietary PATrOL platform, we have designed a first-in-class anti-gene candidate that selectively binds mutant DMPK mRNA and opens its hairpin secondary structure, as opposed to a mechanism of action that explicitly degrades the mutant and wild-type transcripts indiscriminately, making it a unique option for the treatment of DM1. These in vitro and in vivo data both support our hypothesized mechanism of action and demonstrate rapid and broad resolution of the mis-splicing that is the primary cause of DM1.

This is the second set of positive data that weve announced in 2020 for our PATrOL-enabled therapies, which we believe serves as proof of concept that further validates our technologic foundation. With a single unified platform, we believe we can increase, decrease or change protein function of potentially any nucleic acid target, unique among genetic medicine approaches. We are excited by the progress we have made and look forward to providing additional updates on our platform and pipeline of programs at an R&D day in the first half of 2021.

In vitro data highlights in DM1 patient-derived fibroblasts:

In vivo data highlights in the HSALR transgenic mouse model of DM1 that expresses high levels of mutant CUG-repeat-containing mRNA (HSA) in skeletal muscle:

DM1 is a rare, autosomal dominant repeat expansion disorder characterized by progressive muscle wasting and weakness. It also affects the central nervous system (CNS) and heart. DM1 is caused by CTG nucleic acid repeats in the DMPK gene that produce a hairpin structure in the transcribed DMPK mRNA. The hairpin structure sequesters critical splice regulators and results in the mis-splicing of multiple gene transcripts. Furthermore, the binding of splice regulators traps the mutant DMPK mRNA in the nucleus, resulting in DMPK protein haploinsufficiency, or half the level of protein that is needed for normal function, which is thought to exacerbate the CNS and cardiac symptoms that are characteristic of DM1 (as knock-out mice for Dmpk show both severe cardiac conduction defects as well as issues with neuronal cytoskeletal remodeling manifesting in aberrant long-term potentiation). The prevalence of DM1 is >5/100,000 in the general population. There are currently no approved treatments for DM1.

Conference Call and Webcast Details

NeuBase Therapeutics, Inc. will discuss these data during a webcasted conference call with slides today, December 16, 2020, at 8:00 a.m. EST. To access the webcast, please click here. An archived recording of this presentation will be available following the call through the IR Calendar page on the Investors section of the Companys website, http://www.neubasetherapeutics.com.

About NeuBase Therapeutics, Inc.NeuBase is accelerating the genetic revolution using a new class of synthetic medicines. NeuBase's designer PATrOL therapies are centered around its proprietary drug scaffold to address genetic diseases at the source by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions. To learn more, visit http://www.neubasetherapeutics.com.

Use of Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential significance and implications of the Companys positive in vitro and in vivo preclinical data for its PATrOL-enabled anti-gene therapies for the treatment of myotonic dystrophy. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the risks that prior data will not be replicated in future studies; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCdaniel@lifesciadvisors.com OP: (617) 430-7576

NeuBase Media Contact:Cait Williamson, Ph.D.LifeSci Communicationscait@lifescicomms.com OP: (646) 751-4366

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NeuBase Therapeutics Announces Positive Preclinical In Vivo Data for PATrOL-enabled Anti-gene for the Treatment of Myotonic Dystrophy Type 1 -...

An atlas of S. pneumoniae and host gene expression during colonization and disease – The Mix

Streptococcus pneumoniae is an opportunistic pathogen that commonly infects young children and the elderly. This atlas will help researchers better understand how to treat these infections.

Streptococcus pneumoniae is an opportunistic pathogen that commonly infects young children and the elderly. This atlas will help researchers better understand how to treat these infections.The bacteria Streptococcus pneumoniae colonizes the nasopharynx and can cause pneumonia. Then, it can spread from the lungs to the bloodstream and cause organ damage. This opportunistic pathogen commonly infects young children, those who are immunocompromised and the elderly. In 2015, S. pneumoniae infections worldwide killed an estimated 192,000 to 366,000 children under age 5.

To understand how this pathogen adapts to different locations in the body, and also how the host responds to the invading microbe, researchers at the University of Alabama at Birmingham, the University of Maryland School of Medicine and Yale University School of Medicine measured bacterial and host gene expression at five different sites in a mouse model the nasopharynx, lungs, blood, heart and kidneys using three genetically different strains of S. pneumoniae.

Their resulting in vivo atlas of host-pathogen interactions at disease-relevant anatomical sites is now published in Proceeding of the National Academy of Sciences. The researchers identified shared and organ-specific transcriptomes of S. pneumoniae, and they showed that the bacterial and host gene expression profiles are highly distinct during asymptomatic colonization versus disease-causing infection.

This means the bacterium behaves differently, depending on which site it infects, and that the mouse organs, in turn, also respond differently to the presence of bacteria. Additionally, certain S. pneumoniae genes were found to always be highly expressed by all three strains of bacteria at all anatomical sites, which makes them ideal targets for new vaccines or therapies.

This was the first time that gene expression profiles during colonization and at multiple host infection sites were mapped from both the host and the pathogen perspectives.

We believe that the atlas of transcriptional responses during host-pathogen interactions presented here, the authors wrote, will constitute an essential resource for the pneumococcal and microbial pathogenesis research communities and serve as a foundation for identification and validation of key host and pneumococcal therapeutic targets in future studies.

Carlos J. Orihuela, Ph.D., professor in the UAB Department of Microbiology, and Herv Tettelin, Ph.D., professor in the University of Maryland School of Medicine Department of Microbiology and Immunology, are co-senior authors.

Carlos J. Orihuela, Ph.D.Besides a descriptive analysis of the transcriptomes, researchers confirmed their findings using bacterial mutants, in vivo challenge experiments and host treatments. In challenge experiments, the researchers found that an interferon beta anti-inflammatory treatment prevented the bacteria from invading vital organs and promoted host survival. This finding offers potentially new therapeutic avenues.

Symptoms of pneumococcal infection include fever, cough, shortness of breath, chest pain, stiff neck, confusion, increased sensitivity to light, joint pain, chills, ear pain, sleeplessness and irritability. While advances in antibiotics and the use of pneumococcal conjugate vaccines since 2000 have lowered deaths attributable to S. pneumoniae, the pathogen continues to show an increase in antibiotic resistance, and it also can switch to capsule types that are not covered by the current United States Food and Drug Administration-approved vaccines. Thus, pneumococcal infections continue to be a significant cause of illness and death.

Co-authors with Tettelin and Orihuela, in the study, An in vivo atlas of host-pathogen transcriptomes during Streptococcus pneumoniae colonization and disease, are Adonis DMello, Department of Microbiology and Immunology, University of Maryland School of Medicine; Ashleigh N. Riegler, Eriel Martnez, Sarah M. Beno and Tiffany Ricketts, UAB Department of Microbiology; and Ellen F. Foxman, Department of Laboratory Medicine, Yale University School of Medicine.

Support came from the Merck, Sharpe & Dohme Corp. Merck Investigator Studies Program award IISP ID#: 57329 and from National Institutes of Health grant AI114800.

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An atlas of S. pneumoniae and host gene expression during colonization and disease - The Mix

Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) – Canada NewsWire

Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defectiveSMN1gene1.It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein1.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking.Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa."The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy.The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mre Enfant Soleil, Universit Laval in Qubec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3. Without a functionalSMN1gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90%of cases4.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene1.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular AtrophySMA is the leading cause of genetic infant death2. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities5.This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression6.Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly5. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta7,8.

About Novartis in Gene Therapy and Rare DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for thein vivogene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon forin vivodelivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

Excerpt from:
Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire

CRISPR Therapeutics Receives Grant to Advance In Vivo CRISPR/Cas9 Gene Editing Therapies for HIV – GlobeNewswire

-Funding from the Bill & Melinda Gates Foundation will support research to enable CRISPR/Cas9-based therapies for HIV that can benefit patients worldwide-

ZUG, Switzerland and CAMBRIDGE, Mass., Dec. 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the receipt of a grant from the Bill & Melinda Gates Foundation to research in vivo gene editing therapies for the treatment of HIV.

While we have demonstrated the promise of CRISPR/Cas9 gene editing ex vivo in sickle cell disease and beta thalassemia, an in vivo approach to editing hematopoietic stem cells could allow the transformative benefit of CRISPR/Cas9 to reach a broader array of patients, including those in low resource settings that lack sufficient infrastructure for stem cell transplantation, said Tony Ho, M.D., Executive Vice President and Head of Research & Development at CRISPR Therapeutics. We look forward to working on new therapies that could contribute to the global effort to reduce the burden of HIV.

The grant builds upon CRISPR Therapeutics proprietary CRISPR/Cas9 gene editing technology and expertise in editing hematopoietic stem cells and contributes to efforts to accelerate transformative medicines for global health.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Ho in this press release, as well as regarding CRISPR Therapeutics expectations about any or all of the following: (i) the expected benefits of CRISPR Therapeutics research funded by the Bill & Melinda Gates Foundation and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be favorable and predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; potential impacts due to the coronavirus pandemic, such as the timing and progress of preclinical studies; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR THERAPEUTICS word mark and design logo are registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

Media Contact:Rachel EidesWCG on behalf of CRISPR+1-617-337-4167reides@wcgworld.com

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CRISPR Therapeutics Receives Grant to Advance In Vivo CRISPR/Cas9 Gene Editing Therapies for HIV - GlobeNewswire

Genetic Analysis Services Market: Uptake of Next-generation Sequencing and Multi-gene Tests to Drive Market – BioSpace

Genetic Testing to Establish Strong Foothold in Current and Future Healthcare System

The notable rise in the demand for hereditary genetic testing over the past few years is one of the major factors that is expected to fuel the growth of the global genetic analysis services market in the upcoming decade. Technological advancements coupled with the drive to discover new and innovative genetic analysis techniques are set to shape the overall growth trajectory of the global genetic analysis services market during the forecast period. Over the past decade, the genome testing sector has witnessed consistent developments due to which, the global genetic analysis services market is anticipated to expand at an impressive rate during the assessment period.

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Hereditary genetic testing has emerged as ideal, and a rapidly evolving technology within the genetic analysis services market. This is likely to continue, owing to advancements in technology and findings of research activities. The increasing demand for improved and cutting-edge prediction and diagnostic tools and services coupled with surge in demand for disease monitoring is anticipated to play a key role in the overall growth of the global genetic analysis services market during the assessment period.

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Healthcare experts and credible researchers around the world are of the opinion that genetic testing is expected to be the future of the healthcare ecosystem. Advancements in the biomedical field coupled with the notable rise in the number of companies that are developing new genetic-testing kits are expected to augment the global genetic analysis services market during the forecast period. Moreover, as interest levels for precision medicine continues to witness sizeable growth around the world, as a result of which the demand for genetic analysis services is projected to grow at an impressive pace.

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Uptake of Next-generation Sequencing and Multi-gene Tests to Drive Market

Advancements in the genetic technology are likely to play an instrumental role in shaping the growth trajectory of the global genetic analysis services market during the forecast period. Furthermore, due to advancements in technology, the scope of genetic testing has widened by a considerable margin due to which, the demand for genetic analysis services is increasing. While genetic analysis services in the past were largely time-consuming and cumbersome, at present, increasing speed and availability of genomic testing are anticipated to present a plethora of opportunities to the players involved in the current market landscape for genetic analysis services.

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In addition, the gradual shift in the point of access to testing is evolving, as more number of consumers can avail genetic analysis services outside the healthcare setting. Advancements in genetic medicine at the back of advancements in technology are likely to bolster the growth of the global genetic analysis services market during the assessment period.

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Research and Development Activities in Full Swing amid COVID-19 Pandemic

Research and development activities are expected to continue in full swing amid the ongoing COVID-19 pandemic. The significant rise in the demand for genetic counseling services during the ongoing COVID-19 crisis is anticipated to generate consistent revenue for the players involved in the genetic analysis services market. Furthermore, researchers and scientists are increasingly focusing on discovering genetic mechanisms that are required to prevent the spread and transmission of the novel coronavirus disease. Genetic research is estimated to unlock various intricate details of the novel coronavirus, thereby opening up new opportunities for mitigation. The ongoing research pertaining to genetics and its correlation with the ongoing pandemic is expected to provide a detailed and microscopic understanding of the overall cellular mechanisms of the virus.

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Genetic Analysis Services Market: Uptake of Next-generation Sequencing and Multi-gene Tests to Drive Market - BioSpace