Microsoft co-founder Bill Gates makes a point during a Q&A with Margaret Hamburg, board chair for the American Association for the Advancement of Science. (GeekWire Photo / Alan Boyle)

Microsoft co-founder Bill Gates has been working to improve the state of global health through his nonprofit foundation for 20 years, and today he told the nations premier scientific gathering that advances in artificial intelligence and gene editing could accelerate those improvements exponentially in the years ahead.

We have an opportunity with the advance of tools like artificial intelligence and gene-based editing technologies to build this new generation of health solutions so that they are available to everyone on the planet. And Im very excited about this, Gates said in Seattle during a keynote address at the annual meeting of the American Association for the Advancement of Science.

Such tools promise to have a dramatic impact on several of the biggest challenges on the agenda for the Bill & Melinda Gates Foundation, created by the tech guru and his wife in 2000.

When it comes to fighting malaria and other mosquito-borne diseases, for example, CRISPR-Cas9 and other gene-editing tools are being used to change the insects genome to ensure that they cant pass along the parasites that cause those diseases. The Gates Foundation is investing tens of millions of dollars in technologies to spread those genomic changes rapidly through mosquito populations.

Millions more are being spent to find new ways fighting sickle-cell disease and HIV in humans. Gates said techniques now in development could leapfrog beyond the current state of the art for immunological treatments, which require the costly extraction of cells for genetic engineering, followed by the re-infusion of those modified cells in hopes that theyll take hold.

For sickle-cell disease, the vision is to have in-vivo gene editing techniques, that you just do a single injection using vectors that target and edit these blood-forming cells which are down in the bone marrow, with very high efficiency and very few off-target edits, Gates said. A similar in-vivo therapy could provide a functional cure for HIV patients, he said..

The rapid rise of artificial intelligence gives Gates further cause for hope. He noted that that the computational power available for AI applications has been doubling every three and a half months on average, dramatically improving on the two-year doubling rate for chip density thats described by Moores Law.

One project is using AI to look for links between maternal nutrition and infant birth weight. Other projects focus on measuring the balance of different types of microbes in the human gut, using high-throughput gene sequencing. The gut microbiome is thought to play a role in health issues ranging from digestive problems to autoimmune diseases to neurological conditions.

This is an area that needed these sequencing tools and the high-scale data processing, including AI, to be able to find the patterns, Gates said. Theres just too much going on there if you had to do it, say, with paper and pencil to understand the 100 trillion organisms and the large amount of genetic material there. This is a fantastic application for the latest AI technology.

Similarly, organs on a chip could accelerate the pace of biomedical research without putting human experimental subjects at risk.

In simple terms, the technology allows in-vitro modeling of human organs in a way that mimics how they work in the human body, Gates said. Theres some degree of simplification. Most of these systems are single-organ systems. They dont reproduce everything, but some of the key elements we do see there, including some of the disease states for example, with the intestine, the liver, the kidney. It lets us understand drug kinetics and drug activity.

The Gates Foundation has backed a number of organ-on-a-chip projects over the years, including one experiment thats using lymph-node organoids to evaluate the safety and efficacy of vaccines. At least one organ-on-a-chip venture based in the Seattle area, Nortis, has gone commercial thanks in part to Gates support.

High-tech health research tends to come at a high cost, but Gates argues that these technologies will eventually drive down the cost of biomedical innovation.

He also argues that funding from governments and nonprofits will have to play a role in the worlds poorer countries, where those who need advanced medical technologies essentially have no voice in the marketplace.

If the solution of the rich country doesnt scale down then theres this awful thing where it might never happen, Gates said during a Q&A with Margaret Hamburg, who chairs the AAAS board of directors.

But if the acceleration of medical technologies does manage to happen around the world, Gates insists that could have repercussions on the worlds other great challenges, including the growing inequality between rich and poor.

Disease is not only a symptom of inequality, he said, but its a huge cause.

Other tidbits from Gates talk:

Read Gates prepared remarks in a posting to his Gates Notes blog, or watch the video on AAAS YouTube channel.

Visit link:
Why Bill Gates thinks gene editing and artificial intelligence could save the world - GeekWire

The world is watching with alarm as China struggles to contain a dangerous new virus, now being called SARS-CoV-2. It has quarantined entire cities, and the US has put a blanket ban on travellers whove been there. Health officials are scrambling to understand how the virus is transmitted and how to treat patients.

But in one University of North Carolina lab, theres a different race. Researchers are trying to create a copy of the virus. From scratch.

Led by Ralph Baric, an expert in coronaviruseswhich get their name from the crown-shaped spike they use to enter human cellsthe North Carolina team expects to recreate the virus starting only from computer readouts of its genetic sequence posted online by Chinese labs last month.

The remarkable ability to boot up viruses from genetic instructions is made possible by companies that manufacture custom DNA molecules, such as Integrated DNA Technology, Twist Bioscience, and Atum. By ordering the right genes, which cost a few thousand dollars, and then stitching them together to create a copy of the coronavirus genome, its possible to inject the genetic material into cells and jump-start the virus to life.

The ability to make a lethal virus from mail-order DNA was first demonstrated 20 years ago. Its enough of a bioterrorism concern that companies carefully monitor who is ordering which genes. But its also an important way to respond to a sudden outbreak, since synthetic virus recipes give researchers powerful ways to study treatments, vaccines, and how mutations could make it more dangerous.

When a synthetic virus is better than the real thing

Barics North Carolina lab, which specializes in engineering viruses, has previously butted heads with Washington agencies over the work, which has included synthesizing new, never before seen coronaviruses that can infect mice. In 2014, the National Institutes of Health froze funding to several labs, including Barics, over concerns that such research was too risky. The funding was later reinstated.

For the China virus, Baric said in a telephone interview, his team placed an order for matching DNA from a manufacturer last month. Their first step was to go online and look at genetic sequences of the virus. They then compared several available sequences, which differ slightly, and picked a consensus version to have manufactured.

Once Baric gets his DNA, something that could take a month, he plans to inject the genetic instructions into cells. If things go as planned, the cells should begin making actual infectious viral particles.

CDC

By rolling their own germs, scientists can get hold of viruses even if they cant obtain them directly from a country, especially one thats in the grip of an epidemic. Baric says so far samples of the live virus from patients have not been made widely available from China. This is the future in terms of how the medical research community responds to a new threat, says Baric.

The real virus and the synthetic one should be basically identical. But with the synthetic one, we have a DNA copy that we can go back to over and over and over again, to make genetically identical viruses, says Timothy Sheahan, a researcher at UNC who works with Baric. Starting from these copies, scientists can remove genes, add others, and figure out things like what makes the germ spread and how it gains access to human cells. Sheahan wants to try infecting mice with the virus and giving them various drugs to see what stops it.

Artificial copies may also help scientists keep up with the outbreaks unpredictable path. I worry this virus is going to mutate in the course of the epidemic, and this would allow me to study what effects those mutations have, says Stanley Perlman, a microbiologist who works on coronaviruses at the University of Iowa. The synthetic virus is just a substitute for the actual virus, but with the DNA clone you can manipulate it and find the weak points and develop a therapy.

During past outbreaks, scientists would have had to wait months or years to get a look at the germ behind an outbreak. But with SARS-CoV-2 it took only weeks until its genetic sequence was posted online. Immediately, some scientists began analyzing the genetic data, comparing it to viruses from bats, snakes and pangolins; they concluded it could have begun circulating last November.

Biotech companies, governments, and universities also quickly started ordering physical copies of particular genes found in the virus. DNA manufacturers say they have been deluged with orders for virus parts, including those useful for verifying diagnostic tests and others needed to make potential vaccines.

Its been a pretty dramatic uptick, starting with the publication of the genome, says Adam Clore, technical director of synthetic biology at IDT, based in Iowa, and one of the worlds largest sellers of DNA. Its high priority. There are a number of institutions that are devoting nearly all their energy working on detection or vaccines.

Still, most researchers need only one or two genes from the virus to carry forward work on tests and vaccines. Barics lab in North Carolina is the only one in the US known to be trying to re-create the virus completely from ordered DNA parts.

How to keep deadly viruses out of the wrong hands

It was in the early 2000s that scientists first showed that synthetic DNA strands could be used to resurrect viruses just from their genetic code. A team in New York State did it with polio, producing infectious material from DNA they ordered online.

The technology immediately created bio-weapon worries. What if terrorists used the technique to resurrect smallpox? That hasnt happened, but it does mean that scourges like polio, smallpoxand now the Chinese coronaviruscannot now ever be truly wiped out. Researchers at the US Centers for Disease Control and Prevention (CDC) drove that point home in 2005 when they resurrected the influenza virus that killed tens of millions in 1918-1919.

To keep the technology out of the hands of evil-doers, companies that manufacture DNA banded together a few years ago to limit access to dangerous genes. The big US players have all agreed to compare incoming DNA orders to a database of about 60 lethal germs and toxins called select agents so that only authorized labs can ever obtain the DNA needed to resurrect them.

CDC

At our request, Battelle, a scientific R&D company whose software ThreatSEQ can make those comparisons, ran the scenario of someone trying to order a copy of SARS-CoV-2. According to Craig Bartling, a senior research scientist at Battelle, the software flagged both the entire virus, and most of its genes individually, at the highest threat level. Bartling says the alerts went off because the virus is highly similar to the original SARS, a related virus that sparked a global outbreak starting in 2002.

Research into the new virus is seen as risky enough that manufacturers of DNA hurried last week to meet and formulate a policy about who should be able buy complete versions of the new germs genome. In a statement released on February 11, the International Gene Synthesis Consortium, a trade group, struck a cautious position. It said it would treat the new Chinese virus as if it were SARS, a germ added to the select agent list in 2012 and whose possession is tightly monitored by the US government.

That means anyone who wants a complete synthetic copy of SARS-CoV-2 would need to undergo specific and detailed vetting and prove they are already registered by the CDC to work with SARS, as the North Carolina researchers are.

However, companies that manufacture DNA still have discretion over what they sell and to whom, and not all of them think they should make the whole genome of this virus. Claes Gustaffson, founder and chief commercial officer of Atum, a DNA supplier in California, says hes gotten orders from eight companies for parts of the virus genome and has personally approved a request by a US government agency to make 90% of its geneslikely
to create an attenuated (i.e., harmless) version of it.

They probably want to figure out how to make a vaccine as quickly as possible, says Gustaffson. But if someone wanted the whole thing, I wouldnt make it. Some things, like polio, you dont want to make, no matter who is asking.

UNC Gillings School of Public Health

Not everyone thinks synthesizing the new coronavirus is particularly dangerous. I dont really see a huge amount of risk, says Nicholas G. Evans, who studies biothreats at the University of Massachusetts, Lowell. Right now, a lot of people are spending a lot of time on how this coronavirus works. I think the risks are outweighed by the benefits.

The outbreak, which appears to have begun in a live animal market in the city of Wuhan, had caused more than 64,000 cases and 1,350 deaths in China by February 14, so its even worse than SARS, which killed 774 people.

Still, the US has not yet declared the new virus to be a select agent. According to Baric, the decision to add a new virus to the most-dangerous list is not made in the expanding outbreak, because it slows down research.

Scaring people

For now, only a very few sophisticated centers can actually re-boot a virus; theres no chance a nut working from a garage could do it. We are at the point where the best of the best can start to synthesize this new virus contemporaneously with the outbreak. But that is just a few labs, says Evans. Fortunately, we are still far from the point when lots of people can synthesize anything.

The advanced state of synthetic virus research, and the ability to genetically engineer germs, inevitably feeds fears, and conspiracy theories. Social media and some blog sites have been full of groundless speculation that the new virus was accidentally released from a Chinese bioweapon lab located outside of Wuhan. Theres no evidence that is the case, and substantial evidence it is not, but the rumor caused a diplomatic breach with China after it was repeated in the US Congress by a senator, Tom Cotton of Arkansas.

Baric says he doesnt see a particular danger to synthesizing the new virus at this stage of the outbreak, especially because the virus is still circulating in the wild. The important thing is to figure out what it does and stop it. Whether you get it from a cell or synthesize it, it ends up the same thing, says Baric.

Original post:
Biologists rush to re-create the China coronavirus from its DNA code - MIT Technology Review

Such is the extent of our dominion on Earththat the answers to questions around whether we are still part of nature and whether we even need some of it rely on an understanding of what we want as Homo sapiens. And to know what we want, we need to grasp what we are.

It is a huge question but they are the best. And as a biologist, here is my humble suggestion to address it, and a personal conclusion. You may have a different one, but what matters is that we reflect on it.

Perhaps the best place to start is to consider what makes us human in the first place, which is not as obvious as it may seem.

Sharing the full story, not just the headlines

Many years ago, a novel written by Vercors called Les Animaux Dnaturs (Denatured Animals) told the story of a group of primitive hominids, the Tropis, found in an unexplored jungle in New Guinea, who seem to constitute a missing link. However, the prospect that this fictional group may be used as slave labour by an entrepreneurial businessman named Vancruysen forces society to decide whether the Tropis are simply sophisticated animals or whether they should be given human rights. And herein lies the difficulty.

Human status had hitherto seemed so obvious that the book describes how it is soon discovered that there is no definition of what a human actually is. Certainly, the string of experts consulted anthropologists, primatologists, psychologists, lawyers and clergymen could not agree. Perhaps prophetically, it is a layperson who suggested a possible way forward.

She asked whether some of the hominids habits could be described as the early signs of a spiritual or religious mind. In short, were there signs that, like us, the Tropis were no longer at one with nature, but had separated from it, and were now looking at it from the outside with some fear.

Pluto has a 'beating heart' of frozen nitrogen that is doing strange things to its surface, Nasa has found.The mysterious core seems to be the cause of features on its surface that have fascinated scientists since they were spotted by Nasa's New Horizons mission."Before New Horizons, everyone thought Pluto was going to be a netball - completely flat, almost no diversity," said Tanguy Bertrand, an astrophysicist and planetary scientist at NASA's Ames Research Center and the lead author on the new study."But it's completely different. It has a lot of different landscapes and we are trying to understand what's going on there."

Getty

The ancient invertabrate worm-like species rhenopyrgus viviani (pictured) is one of over 400 species previously unknown to science that were discovered by experts at the Natural History Museum this year

PA

Jackdaws can identify dangerous humans from listening to each others warning calls, scientists say. The highly social birds will also remember that person if they come near their nests again, according to researchers from the University of Exeter. In the study, a person unknown to the wild jackdaws approached their nest. At the same time scientists played a recording of a warning call (threatening) or contact calls (non-threatening). The next time jackdaws saw this same person, the birds that had previously heard the warning call were defensive and returned to their nests more than twice as quickly on average.

Getty

The sex of the turtle is determined by the temperatures at which they are incubated. Warm temperatures favour females.But by wiggling around the egg, embryos can find the Goldilocks Zone which means they are able to shield themselves against extreme thermal conditions and produce a balanced sex ratio, according to the new study published in Current Biology journal

Ye et al/Current Biology

African elephant poaching rates have dropped by 60 per cent in six years, an international study has found. It is thought the decline could be associated with the ivory trade ban introduced in China in 2017.

Reuters

Scientists have identified a four-legged creature with webbed feet to be an ancestor of the whale. Fossils unearthed in Peru have led scientists to conclude that the enormous creatures that traverse the planets oceans today are descended from small hoofed ancestors that lived in south Asia 50 million years ago

A. Gennari

A scientist has stumbled upon a creature with a transient anus that appears only when it is needed, before vanishing completely. Dr Sidney Tamm of the Marine Biological Laboratory could not initially find any trace of an anus on the species. However, as the animal gets full, a pore opens up to dispose of waste

Steven G Johnson

Feared extinct, the Wallace's Giant bee has been spotted for the first time in nearly 40 years. An international team of conservationists spotted the bee, that is four times the size of a typical honeybee, on an expedition to a group of Indonesian Islands

Clay Bolt

Fossilised bones digested by crocodiles have revealed the existence of three new mammal species that roamed the Cayman Islands 300 years ago. The bones belonged to two large rodent species and a small shrew-like animal

New Mexico Museum of Natural History

Scientists at the University of Maryland have created a fabric that adapts to heat, expanding to allow more heat to escape the body when warm and compacting to retain more heat when cold

Faye Levine, University of Maryland

A study from the University of Tokyo has found that the tears of baby mice cause female mice to be less interested in the sexual advances of males

Getty

The Intergovernmental Panel on Climate Change has issued a report which projects the impact of a rise in global temperatures of 1.5 degrees Celsius and warns against a higher increase

Getty

The nobel prize for chemistry has been awarded to three chemists working with evolution. Frances Smith is being awarded the prize for her work on directing the evolution of enzymes, while Gregory Winter and George Smith take the prize for their work on phage display of peptides and antibodies

Getty/AFP

The nobel prize for physics has been awarded to three physicists working with lasers. Arthur Ashkin (L) was awarded for his "optical tweezers" which use lasers to grab particles, atoms, viruses and other living cells. Donna Strickland and Grard Mourou were jointly awarded the prize for developing chirped-pulse amplification of lasers

Reuters/AP

The Ledumahadi Mafube roamed around 200 million years ago in what is now South Africa. Recently discovered by a team of international scientists, it was the largest land animal of its time, weighing 12 tons and standing at 13 feet. In Sesotho, the South African language of the region in which the dinosaur was discovered, its name means "a giant thunderclap at dawn"

Viktor Radermacher / SWNS

Scientists have witnessed the birth of a planet for the first time ever. This spectacular image from the SPHERE instrument on ESO's Very Large Telescope is the first clear image of a planet caught in the very act of formation around the dwarf star PDS 70. The planet stands clearly out, visible as a bright point to the right of the center of the image, which is blacked out by the coronagraph mask used to block the blinding light of the central star.

ESO/A. Mller et al

Layers long thought to be dense, connective tissue are actually a series of fluid-filled compartments researchers have termed the interstitium. These compartments are found beneath the skin, as well as lining the gut, lungs, blood vessels and muscles, and join together to form a network supported by a mesh of strong, flexible proteins

Getty

Working in the Brazilian state of Mato Grosso, a team led by archaeologists at the University of Exeter unearthed hundreds of villages hidden in the depths of the rainforest. These excavations included evidence of fortifications and mysterious earthworks called geoglyphs

Jos Iriarte

More than one in 10 people were found to have traces of class A drugs on their fingers by scientists developing
a new fingerprint-based drug test.Using sensitive analysis of the chemical composition of sweat, researchers were able to tell the difference between those who had been directly exposed to heroin and cocaine, and those who had encountered it indirectly.

Getty

The storm bigger than the Earth, has been swhirling for 350 years. The image's colours have been enhanced after it was sent back to Earth.

Pictures by: Tom Momary

Pluto has a 'beating heart' of frozen nitrogen that is doing strange things to its surface, Nasa has found.The mysterious core seems to be the cause of features on its surface that have fascinated scientists since they were spotted by Nasa's New Horizons mission."Before New Horizons, everyone thought Pluto was going to be a netball - completely flat, almost no diversity," said Tanguy Bertrand, an astrophysicist and planetary scientist at NASA's Ames Research Center and the lead author on the new study."But it's completely different. It has a lot of different landscapes and we are trying to understand what's going on there."

Getty

The ancient invertabrate worm-like species rhenopyrgus viviani (pictured) is one of over 400 species previously unknown to science that were discovered by experts at the Natural History Museum this year

PA

Jackdaws can identify dangerous humans from listening to each others warning calls, scientists say. The highly social birds will also remember that person if they come near their nests again, according to researchers from the University of Exeter. In the study, a person unknown to the wild jackdaws approached their nest. At the same time scientists played a recording of a warning call (threatening) or contact calls (non-threatening). The next time jackdaws saw this same person, the birds that had previously heard the warning call were defensive and returned to their nests more than twice as quickly on average.

Getty

The sex of the turtle is determined by the temperatures at which they are incubated. Warm temperatures favour females.But by wiggling around the egg, embryos can find the Goldilocks Zone which means they are able to shield themselves against extreme thermal conditions and produce a balanced sex ratio, according to the new study published in Current Biology journal

Ye et al/Current Biology

African elephant poaching rates have dropped by 60 per cent in six years, an international study has found. It is thought the decline could be associated with the ivory trade ban introduced in China in 2017.

Reuters

Scientists have identified a four-legged creature with webbed feet to be an ancestor of the whale. Fossils unearthed in Peru have led scientists to conclude that the enormous creatures that traverse the planets oceans today are descended from small hoofed ancestors that lived in south Asia 50 million years ago

A. Gennari

A scientist has stumbled upon a creature with a transient anus that appears only when it is needed, before vanishing completely. Dr Sidney Tamm of the Marine Biological Laboratory could not initially find any trace of an anus on the species. However, as the animal gets full, a pore opens up to dispose of waste

Steven G Johnson

Feared extinct, the Wallace's Giant bee has been spotted for the first time in nearly 40 years. An international team of conservationists spotted the bee, that is four times the size of a typical honeybee, on an expedition to a group of Indonesian Islands

Clay Bolt

Fossilised bones digested by crocodiles have revealed the existence of three new mammal species that roamed the Cayman Islands 300 years ago. The bones belonged to two large rodent species and a small shrew-like animal

New Mexico Museum of Natural History

Scientists at the University of Maryland have created a fabric that adapts to heat, expanding to allow more heat to escape the body when warm and compacting to retain more heat when cold

Faye Levine, University of Maryland

A study from the University of Tokyo has found that the tears of baby mice cause female mice to be less interested in the sexual advances of males

Getty

The Intergovernmental Panel on Climate Change has issued a report which projects the impact of a rise in global temperatures of 1.5 degrees Celsius and warns against a higher increase

Getty

The nobel prize for chemistry has been awarded to three chemists working with evolution. Frances Smith is being awarded the prize for her work on directing the evolution of enzymes, while Gregory Winter and George Smith take the prize for their work on phage display of peptides and antibodies

Getty/AFP

The nobel prize for physics has been awarded to three physicists working with lasers. Arthur Ashkin (L) was awarded for his "optical tweezers" which use lasers to grab particles, atoms, viruses and other living cells. Donna Strickland and Grard Mourou were jointly awarded the prize for developing chirped-pulse amplification of lasers

Reuters/AP

The Ledumahadi Mafube roamed around 200 million years ago in what is now South Africa. Recently discovered by a team of international scientists, it was the largest land animal of its time, weighing 12 tons and standing at 13 feet. In Sesotho, the South African language of the region in which the dinosaur was discovered, its name means "a giant thunderclap at dawn"

Viktor Radermacher / SWNS

Scientists have witnessed the birth of a planet for the first time ever. This spectacular image from the SPHERE instrument on ESO's Very Large Telescope is the first clear image of a planet caught in the very act of formation around the dwarf star PDS 70. The planet stands clearly out, visible as a bright point to the right of the center of the image, which is blacked out by the coronagraph mask used to block the blinding light of the central star.

ESO/A. Mller et al

Layers long thought to be dense, connective tissue are actually a series of fluid-filled compartments researchers have termed the interstitium. These compartments are found beneath the skin, as well as lining the gut, lungs, blood vessels and muscles, and join together to form a network supported by a mesh of strong, flexible proteins

Getty

Working in the Brazilian state of Mato Grosso, a team led by archaeologists at the University of Exeter unearthed hundreds of villages hidden in the depths of the rainforest. These excavations included evidence of fortifications and mysterious earthworks called geoglyphs

Jos Iriarte

More than one in 10 people were found to have traces of class A drugs on their fingers by scientists developing a new fingerprint-based drug test.Using sensitive analysis of the chemical composition of sweat, researchers were able to tell the difference between those who had been directly exposed to heroin and cocaine, and those who had encountered it indirectly.

Getty

The storm bigger than the Earth, has been swhirling for 350 years. The image's colours have been enhanced after it was sent back to Earth.

Pictures by: Tom Momary

It is a telling perspective. Our status as altered or denatured animals creatures who have arguably separated from the natural world is perhaps both the source of our humanity and the cause of many of our troubles. In the words of the books author:

All mans troubles arise from the fact that we do not know what we are and do not agree on what we want to be

We will probably never know the timing of our gradual separation from nature although cave paintings perhaps contain some clues. But a key recent event in our relationship with the world around us is as well documented as it was abrupt. It happened on a sunny Monday morning, at precisely 8.15am.

A new age

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The atomic bomb that rocked Hiroshima on 6 August 1945 was a wake-up call so loud that it still resonates in our consciousness many decades later.

The day the sun rose twice was not only a forceful demonstration of the new era that we had entered buta reminder of how pa
radoxically primitive we remained: differential calculus, advanced electronics and almost godlike insights into the laws of the universe helped build, well a very big stick. Modern Homo sapiens seemingly had developed the powers of gods, while keeping the psyche of a stereotypical Stone Age killer.

We were no longer fearful of nature, but of what we would do to it, and ourselves. In short, we still did not know where we came from but began panicking about where we were going. We now know a lot more about our origins but we remain unsure about what we want to be in the future or, increasingly, as the climate crisis accelerates, whether we even have one.

Arguably, the greater choices granted by our technological advances make it even more difficult to decide which of the many paths to take. This is the cost of freedom. I am not arguing against our dominion over nature nor, even as a biologist, do I feel a need to preserve the status quo. Big changes are part of our evolution. After all, oxygen was first a poison which threatened the very existence of early life, yet it is now the fuel vital to our existence.

Similarly, we may have to accept that what we do, even our unprecedented dominion, is a natural consequence of what we have evolved into, and by a process nothing less natural than natural selection itself. If artificial birth control is unnatural, so is reduced infant mortality.

I am also not convinced by the argument against genetic engineering on the basis that it is unnatural. By artificially selecting specific strains of wheat or dogs, we had been tinkering more or less blindly with genomes for centuries before the genetic revolution. Even our choice of romantic partner is a form of genetic engineering. Sex is natures way of producing new genetic combinations quickly.

Even nature, it seems, can be impatient with itself.

Changing our world

Read the original:
Have humans evolved beyond nature? - The Independent

(KTSDESIGN/Science Photo Library)

Science issues whether connected with climate, childhood vaccines or new techniques in biotechnology are part of the fabric of civic life, raising a range of social, ethical and policy issues for the citizenry. As members of the scientific community gather at the annual meeting of the American Association for the Advancement of Science (AAAS) this week, here is a roundup of key takeaways from our studies of U.S. public opinion about science issues and their effect on society. If youre on Twitter, follow @pewscience for more science findings.

The data for this post was drawn from multiple different surveys. The most recent was a survey of 3,627 U.S. adults conducted Oct. 1 to Oct. 13, 2019. This post also draws on data from surveys conducted in January 2019, December 2018, April-May 2018 and March 2016. All surveys were conducted using the American Trends Panel (ATP), an online survey panel that is recruited through national, random sampling of residential addresses. This way nearly all U.S. adults have a chance of being selected. The survey is weighted to be representative of the U.S. adult population by gender, race, ethnicity, education and other categories. Read more about the ATPs methodology.

Following are the questions and responses for surveys used in this post, as well as each surveys methodology:

1Some public divides over science issues are aligned with partisanship, while many others are not. Science issues can be a key battleground for facts and information in society. Climate science has been part of an ongoing discourse around scientific evidence, how to attribute average temperature increases in the Earths climate system, and the kinds of policy actions needed. While public divides over climate and energy issues are often aligned with political party affiliation, public attitudes on other science-related issues are not.

For example, there are differences in public beliefs around the risks and benefits of childhood vaccines. Such differences arise amid civic debates about the spread of false information about vaccines. While such beliefs have important implications for public health, they are not particularly political in nature.

In fact, Republicans and independents who lean to the GOP are just as likely as Democrats and independents who lean to the Democratic Party to say that, overall, the benefits of the measles, mumps and rubella vaccine outweigh the risks (89% and 88% respectively).

2Americans have differing views about some emerging scientific and technological developments. Scientific and technological developments are a key source of innovation and, therefore, change in society. Pew Research Center studies have explored public reactions to emergent developments from genetic engineering techniques, automation and more. One field at the forefront of public reaction is the use of gene editing of babies or genetic engineering of animals. Americans have mixed views over whether the use of gene editing to reduce a babys risk of serious disease that could occur over their lifetime is appropriate (60%) or is taking medical technology too far (38%), according to a 2018 survey. Similarly, about six-in-ten Americans (57%) said that genetic engineering of animals to grow organs or tissues for humans needing a transplant would be appropriate, while four-in-ten (41%) said it would be taking technology too far.

When we asked Americans about a future where a brain chip implant would give otherwise healthy individuals much improved cognitive abilities, a 69% majority said they were very or somewhat worried about the possibility. By contrast, about half as many (34%) were enthusiastic. Further, as people think about the effects of automation technologies in the workplace, more say automation has brought more harm than help to American workers.

One theme running through our findings on emerging science and technology is that public hesitancy often is tied to concern about the loss of human control, especially if such developments would be at odds with personal, religious and ethical values. In looking across seven developments related to automation and the potential use of biomedical interventions to enhance human abilities, Center studies found that proposals that would increase peoples control over these technologies were met with greater acceptance.

3Most in the U.S. see net benefits from science for society, and they expect more ahead. About three-quarters of Americans (73%) say science has, on balance, had a mostly positive effect on society. And 82% expect future scientific developments to yield benefits for society in years to come.

The overall portrait is one of strong public support for the benefits of science to society, though the degree to which Americans embrace this idea differs sizably by race and ethnicity as well as by levels of science knowledge.

Such findings are in line with those of the General Social Survey on the effects of scientific research. In 2018, about three-quarters of Americans (74%) said the benefits of scientific research outweigh any harmful results. Support for scientific research by this measure has been roughly stable since the 1980s.

4The share of Americans with confidence in scientists to act in the public interest has increased since 2016.

Public confidence in scientists to act in the public interest tilts positive and has increased over the past few years. As of 2019, 35% of Americans report a great deal of confidence in scientists to act in the public interest, up from 21% in 2016.

About half of the public (51%) reports a fair amount of confidence in scientists, and just 13% have not too much or no confidence in this group to act in the public interest.

Public trust in scientists by this measure stands in contrast to that for other groups and institutions. One of the hallmarks of the current times has been low trust in government and other institutions. One-in-ten or fewer say they have a great deal of confidence in elected officials (4%) or the news media (9%) to act in the public interest.

5Americans differ over the role and value of scientific experts in policy matters. While confidence in scientists overall tilts positive, peoples perspectives about the role and value of scientific experts on policy issues tends to vary. Six-in-ten U.S. adults believe that scientists should take an active role in policy debates about scientific issues, while about four-in-ten (39%) say, instead, that scientists should focus on establishing sound scientific facts and stay out of such debates.

Democrats are more inclined than Republicans to think scientists should have an active role in science policy matters. Indeed, most Democrats and Democratic-leaning independents (73%) hold this position, compared with 43% of Republicans and GOP leaners.

More than four-in-ten U.S. adults (45%) say that scientific experts usually make better policy decisions than other people, while a similar share (48%) says such decisions are neither better nor worse than other peoples and 7% say scientific experts decisions are usually worse than other peoples.

Here, too, Democrats tend to hold scientific experts in higher esteem than do Republicans: 54% of Democrats say scientists policy decisions are usually better than those of other people, while two-thirds of Republicans (66%) say that scientists decisions are either no different from or worse than other peoples.

6Factual knowledge alone does not explain public confidence in the scientific method to produce sound conclusions. Overall, a 63% majority of Americans say the scientific method generally produces sound conclusions, while 35% think it can be used to produce any result a researcher wants. Peoples level of knowledge can influence beliefs about these matters, but it does so through the lens of partisanship, a tendency known as motivated reasoning.

Beliefs about this matter illustrate that science knowledge levels sometimes correlate with public attitudes. But partisanship
has a stronger role.

Democrats are more likely to express confidence in the scientific method to produce accurate conclusions than do Republicans, on average. Most Democrats with high levels of science knowledge (86%, based on an 11-item index of factual knowledge questions) say the scientific method generally produces accurate conclusions. By comparison, 52% of Democrats with low science knowledge say this. But science knowledge has little bearing on Republicans beliefs about the scientific method.

7Trust in practitioners like medical doctors and dietitians is stronger than that for researchers in these fields, but skepticism about scientific integrity is widespread. Scientists work in a wide array of fields and specialties. A 2019 Pew Research Center survey found public trust in medical doctors and dietitians to be higher than that for researchers working in these areas. For example, 48% of U.S. adults say that medical doctors give fair and accurate information all or most of the time. By comparison, 32% of U.S. adults say the same about medical research scientists. And six-in-ten Americans say dietitians care about their patients best interests all or most of the time, while about half as many (29%) say this about nutrition research scientists with the same frequency.

One factor in public trust of scientists is familiarity with their work. For example, people who were more familiar with what medical science researchers do were more trusting of these researchers to express care or concern for the public interest, to do their job with competence and to provide fair and accurate information. Familiarity with the work of scientists was related to trust for all six specialties we studied.

But when it comes to questions of scientists transparency and accountability, most Americans are skeptical. About two-in-ten or fewer U.S. adults say that scientists are transparent about potential conflicts of interest with industry groups all or most of the time. Similar shares (roughly between one-in-ten and two-in-ten) say that scientists admit their mistakes and take responsibility for them all or most of the time.

This data shows clearly that when it comes to questions of transparency and accountability, most in the general public are attuned to the potential for self-serving interests to skew science findings and recommendations. These findings echo calls for increased transparency and accountability across many sectors and industries today.

8What boosts public trust in scientific research findings? Most say its making data openly available. A 57% majority of Americans say they trust scientific research findings more when the data is openly available to the public. And about half of the U.S. public (52%) say they are more likely to trust research that has been independently reviewed.

The question of who funds the research is also consequential for how people think about scientific research. A 58% majority say they have lower trust when research is funded by an industry group. By comparison, about half of Americans (48%) say government funding for research has no particular effect on how much they trust the findings; 28% say this decreases their trust and 23% say it increases their trust.

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Key findings about Americans' confidence in science and their views on scientists' role in society - Pew Research Center

Reports from theFood and Agricultural Organization of the United Nations,World Health Organization, andothers emphasize the critical role of plant-based diets in creating a sustainable food future for all. Plant-based diets are also key for human nutrition, highlighted in diet guidelines the world over includingUS,Canada, andBrazil. Yet livestock remains essential to aroundone billionof the worlds indigent and theglobal demand for meat and dairy is expected toincrease by 70% by 2050.

Meat production and consumption habits must shift, and solutions are sorely needed to feed the appetite for meat in the US and abroad.

Enter plant-based burgers, which exploded onto the food scene in the 2010s. While eaters love them, questions followed: Are they healthier? More sustainable? And are they even real food?

Opinions are heated, but what does the science show?

A Brave New Burger thats Just Plain Better

Forget bland veggies burgers of yore that only appealed to die-hard vegetarians. Todays food technology methods have brought consumers a beefy patty that sizzlesand theyre a game-changer.

Beyond MeatandImpossible Foodslead the plant-based burger market, and are quite similarin nutrient content and ingredients. A key difference is the use of genetic engineering, used in Impossible to create its umami punch from soy leghemoglobin. Not surprisingly,Impossible eaters care not at all about the tech that made it tastynor should they, given the copious evidence of its safety. (Beyond, conversely, boasts theyre non-GMO.) Major food companies also offer their own plant-based burgers using a variety of techniques and ingredients, now available in supermarkets alongside Beyond and Impossible.

Critics questioned wondered whether plant-based burgers would take off; the marketplace already offers myriad vegetarian choices, after all. Yet contemporary consumersare increasingly seeking ecoconscious options that supplant meat, while delivering the pleasure of eating meatat least, some of the time. Ninety percent of plant-based meat and dairy consumers are omnivores, in fact, and Beyond reports that more than 70 percent of its consumers are meat-eaters seeking a more sustainable option. Importantly, Beyond and Impossible burgers are found on restaurant andfast foodmenus, a good thing since49% of eaters globallydine at restaurants at least weekly, and most choose fast food fare.

Public health and environmental benefits of plant-based burgers are plentiful. Research funded by Beyond Meat and conducted by independent scientists at the University of Michigan found that its burger used 99 percent less water, 93 percent less land, and 46 percent less energy and produced 90 percent fewer greenhouse gas emissions compared to a beef burger; similar results were found in a study of the Impossible Burger. While no peer-reviewed studies are yet available, a significant body of evidencelike this report of 40,000 farms in 119 countries and covering 40 food products that represent 90 percent of all that is eatenshows significantly higher environmental impacts of meat production on land, water, and air compared to plants. While grass-fed beef can be more sustainable, its complicatedand hardly the panacea supporters claim it to be.

And dont forget about antibiotic resistance, among the biggest threats to global health driven largely bymisuse of medicinesin livestock production.

Whatever the individual motivation to select a plant-based burger, the secret sauce is clear: When food tech delivers taste and convenience, health and sustainability win.

Burger Bloviating: Push Back on Plant-Based Meat

As with many food tech innovations, some folks in nutrition and activist circles began disparaging plant burgers as yet another ultra-processed food that consumers dont need. However, there is considerable variation in nutritional quality across the four-categoryNOVA classification(unprocessed and minimally processed foods, processed culinary ingredients, processed, ultra-processed). Andnumerous studiesincluding areportfrom several professional nutrition and food tech organizationsshow that (ultra-) processed foods like bread and canned goods are nutritionally beneficial; its thewhole dietthat matters.

Plus, beef burgers dont grow on trees; the industry employs an extensive set of ingredientsconsumers simply choose not to consider. A wide range ofadditives and preservativesand food processing methods were needed to get that cow ground up onto your bun, for instance, alongside atrocious conditions in industrial animal farming systems. And were you aware that meatpacking is among the most dangerous jobs in the world? The reality is that getting a burger to your table made from animals involves far more processing than one made with plants, facts its polystyrene package doesnt provide.

But is plant-based meat real food? The concept was popularized by journalist Michael Pollan, whose other pithy yet patronizing advice includes eat plants, not food made in plants. Food writer Mark Bittman recently opined, [w]e have to determine whether theyre actually food,likening plant-based burgers to Cheetos. (Seriously?) Other foodies jumped on the bandwagon, creatingnutrition confusionby preaching that meat from animals is inherently superior simply because its from an animal.

At the same time, some health professionals return to the dog-tired diet advice that consumers need to eat more vegetables and fruits, like fresh peas instead of burgers made from pea protein. Similarly, anivory-tower academiccalled plant-based burgers transitional en route to a whole foods diet, ignoring evidence that burgers can be part of a healthy diet, in moderationand are integral to American traditions.

Viewpoints like these reflect a lack of compassion for the realities most people face in just trying to get a meal on the table. They also undermine how difficult it is to change the way we eat, They also discount the vibrant role cuisine plays in culture and disregard the power of technology to meet food needs healthfully and sustainably.

For a Brighter Food Future, Vote With Your Fork

Addressing todays complex food challenges requires all the tools we have to curb climate change, address unsustainable and unjust practices in agriculture, and reduce diet-related chronic diseases. Though novel food technologies will always have haters, its a brave new world with a new generation of eaters.Millennials and Gen Zare highly motivated by health and sustainabilityand both are far more accepting offood technologythan previous generations. Scientific innovations like plant-based burgers will always play a role in shaping human diets,as they always haveand often for the better.

But lets not forget that a burger is a burger is a burgerand its especially tasty with all the fixins. (And fries. Obviously.) Most of us in high-income nations who strive to manage weight, stave off disease, and live longer are better off eating a vibrant salad loaded in fresh veggies, beans, and whole grains rather than a plant-based burger. At least, most of the time.

But you already know that, right?

So when that craving hits, grab a plant-based burger, and enjoy. Voting with your fork is a delicious way to support technologies that will help move forward the food revolution necessary to create a healthy and sustainable food future for all.

P.K. Newby, ScD, MPH, MS, is a nutrition scientist and author whose newest book is Food and Nutrition: What Everyone Needs to Know. Learn more about her at pknewby.com.

Related

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Burger Wars: Beyond Nutrition Idealism and Junk-Science Rhetoric, the Benefits of Choosing Plant-Based are Clear - The Spoon

While it seems increasingly difficult to find a crew to delve into role playing games these days, they hold a special place for me.

Few gaming experiences have been as well-remembered as the first months of playing Dungeons & Dragons, and the pure combination of wonder and amazement that provided.

There have been other RPGs since then of course, and in most every case they have been fun because you become immersed in the world of the game, and the character you play becomes near and dear to you.

As a result I often look at RPGs on Kickstarter, and on one such excursion GeneFunk 2090 from CRISPR Monkey Studios.

There was some great art to the game that was advertising itself as a Biopunk RPG so I looked a little deeper.

That led to the biggest discovery, that the studio doing the game is based in Saskatoon, which made me curious to learn more.

To begin with the game is a biopunk/cyberpunk RPG and setting made using the D&D 5E Open Gaming License. Players take on the role of elite mercenaries that specialize in investigation and violence. No magic or fantasy, but tons of cybernetics, genetic enhancements, nanobots, drones, hacking, androids, high tech guns and armor, and other amazing tech, explains a quick intro on the successful Kickstarter page.

Comparisons of course are natural, and this one immediately had me thinking a game in the same vein as Shadowrun, a long-running RPG, many will know.

So next I contacted James Armstrong regarding the game he has been involved in creating, to find out some information first hand.

He said the game is certainly Biopunk on nature.

I love biology, and the idea of genetic engineering, he replied via email. I actually have a M.Sc. in molecular biology, partially because I was interested in understanding the science behind genetic modification.

Also, Ive always loved speculative fiction, especially of the biopunk variety, from Brave New World, to Cronenberg movies. While I first started this game in 2001, I can tell its only now that biopunk is starting to come into the zeitgeist. Theres currently a Netflix special on biohacking, Jaimie Metzl is on Joe Rogan speaking about his Hacking Darwin book, and CRISPR is part of school curricula.

Theres been an open niche for biopunk RPGs, especially near-future ones and I wanted to address that, and see where I could take it. Endogenous DNA computers, genetic enhancement, mind-hacking, transgenic beasts, and anything else I could think of.

Not surprisingly Armstrong comes at creating an RPG based on his own long held interest.

Ive been an RPG fan since I was in Grade 3, he said. It was the Dungeons and Dragons box sets, red and blue. My older brother brought them home and I was immediately fascinated by the art, and the idea that I could be a character in a fantasy story.

From there, it was the Marvel Super Heroes game, T.M.N.T, WEG Star Wars, and whatever else I could get my hands on! Ive made plenty of my own systems along the way as well.

So where did the germ of the idea for GeneFunk come from.

It was really a convergence of creative influences, and an open niche! I grew up reading the Eastman and Laird T.M.N.T. graphic novels and RPG, loved cyberpunk fiction of every kind, and felt the Gattaca movie was well ahead of its time, related Armstrong. I wanted to play in a world filled will genetically enhanced humans and ubiquitous biotechnology.

Armstrong went into the creative process with a vision.

Create a modern take on the cyberpunk genre using the 5e ruleset, with a biopunk twist, he said. While I love the 80s vision of cyberpunk, most cyberpunk games I see tend to fit into this mold. It could use some updating, some new spice!

Its now apparent that a great deal of human enhancement will be at the genetic level, not necessarily grafted-on chrome arms and robot bodies. I want to show how the world might look if that genetic enhancement started before birth, and how biologically specializing humans might affect society, (and) an informal genetic caste system that emerges from a global market economy.

I also wanted to make some of the cyberpunk tropes a little more fluid. Rather than an explicitly dystopian world, I wanted to showcase a series of double-edged swords. Not technological and capitalistic doom-and-gloom, but something more ambiguous, with some parts being wonderful, and other parts being nightmarish, depending on your perspective. There are pros to living in a technological wonderland. Who needs Huntingtons disease? Alienation due to a collapse of a common human condition? Yes. Ultimate expression of personal identity and diversity through a fluid human form? Also yes.

With such a vast vision to capture the game took years to develop 18-years in fact.

I started in 2001, said Armstrong. I have homebrew versions of it in 3.5e and 4e as well, but once 5e came out, I knew it fit with the mechanics well and I wanted to take it to the next level. Granted, many of those years only had very part-time development, I really kicked it into high-gear over the last three years.

So what was the most difficult aspect of designing the game?

Capturing the powerful nature of genetic enhancement at character creation, said Armstrong. I wanted a characters base genome to provide a great deal of mechanical influence, much more so than a D&D race does. Genetic enhancement is unambiguously superior in GeneFunk, and I needed the mechanics to capture that. As such, starting characters are more powerful than they are in D&D. Theyre not close to X-Men level or anything like that, but they certainly wont be spending level 1 killing boars.

But the game is more than fights and battles.

Asked what is the best element of the game Armstrong said the biohacking, and the great variety of different genomes and upgrades.

There are 42 genetic enhancements and 58 upgrades. Theres even a tool included for players and GMs to make their own genomes.

Being able to play a character with completely different abilities at level 1, each time you make a character, is great for replayability. Ive always loved the meta-game of making characters, Ive probably made 10 characters for every 1 Ive played, regardless of system.

Also, diversity is fun! D&D groups are often a hodge-podge of dragon born, tieflings, gnomes, and goliaths, even if a campaign world might describe these races as rare. I built it so that there is baked-in fluff to support the fact that youre a party of genetic weirdos, stylishly exotic appearances and all.

The vibrancy of a new game world, and the built in diversity of characters to play make GeneFunk a game well worth looking into. Like any RPG the experience of course is only partly dictated by the ruleset, the game master, the one guiding things much as the director of a stage performance, being at least equally important to the overall experience.

It will help to know the base rules of 5e, the most recent incarnation of D&D and one admittedly turned me off the game completely. While I think 5e homogenized D&D into a world of overpowered clones, in a different world setting the core rules can shine.

So check out GeneFunk, it may not be the setting for every taste, but it offers an interesting vision of a future which may be closer than we think.

Check it out via the GeneFunk 2090 page on Facebook.

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New RPG from Sask. creators - Yorkton This Week

A University of Delaware professor is receiving grant funding to develop a new therapy for people with low blood platelet counts and other blood disorders.

When patients dont have enough blood platelets, they cant form clots and this can sometimes be a lethal condition. These patients rely on platelet donations as a treatment, but those are often in short supply since they cannot be frozen and are only good for a few days.

A few years ago, UDs professor of Chemical and Biomolecular Engineering Terry Papoutsakis discovered a way to get patients to produce more blood platelets in their own bloodstream by introducing microparticles made from cultured stem cells.

These microparticles can be frozen to be used on a large scale. And Papoutsakis says they could also be used to treat genetic blood disorders like primary immune deficiencies.

To be able to use those particles for applications in gene therapy for a variety of blood diseasesa lot of genetic diseases or acquired diseases for patients, said Papoutsakis. So thats a difficult problem to deal with and we think this has great promise.

Papoutsakis is being awarded $250,000 through a partnership between University City Science Center and CSL Behring meant to find new biotherapies. He says the award took a few years to secure as a result of contract negotiations around intellectual property rights.

It took a little while to do it, but that is to be understood that it would, because of the potential that this might develop into something quite bigger than we currently see, he said.

Papoutsakis says he has already had some success testing his discovery on mice. He says the award will fund a continuation of those tests, moving the therapy closer to clinical trials on human patients.

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UD professor working to use gene therapy to treat blood disorders - Delaware First Media

CAMBRIDGE, Mass.(GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, is presenting new data from two of its development programs at Keystone Symposias Engineering the Genome Conference, a joint meeting with the Emerging Cellular Therapies: Cancer and Beyond Conference, taking place Feb. 8-12, 2020, in Banff, Canada. Intellia researchers are presenting data in support of the companys lead engineered cell therapy development candidate, NTLA-5001 for the treatment of the hematological cancer, acute myeloid leukemia (AML). Intellia also is sharing preclinical results for its hereditary angioedema (HAE) program, which is Intellias third CRISPR/Cas9 development program, announced in January 2020.

Intellia continues to demonstrate strong progress across both our engineered cell therapy and in vivo pipelines, said Intellia President and Chief Executive Officer John Leonard, M.D. We are observing very favorable preclinical data with our engineered T cells, and we are moving ahead with IND-enabling studies and manufacturing for NTLA-5001, to enable a regulatory submission in the first half of 2021.

On the in vivo side, the data from our HAE development program reinforce the modularity of Intellia's non-viral delivery genome editing platform and how it is enabling independent, single-dose therapies for multiple monogenic diseases. For HAE, we expect to nominate a development candidate in the first half of this year, continued Dr. Leonard.

New Data from Intellias Engineered Cell Therapy Development Program for AML

NTLA-5001, which is Intellias first engineered T cell therapy development candidate and is wholly owned, utilizes a T cell receptor (TCR)-directed approach to target the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. The companys WT1-TCR T cell approach aims to develop a broadly applicable treatment for AML patients, regardless of mutational background of a patients leukemia.

The company is presenting data demonstrating that the selection of a natural, high-affinity TCR, in combination with CRISPR-enabled engineering and targeted insertion, results in an engineered T cell capable of specific and potent killing of primary AML blasts. Todays presentation at Keystone builds on data previously presented last fall at the Annual Congress of the European Society of Gene and Cell Therapy (ESGCT).

The data being presented at the Keystone conference substantiate the advantages that a homogeneous T cell product developed through CRISPR engineering, like NTLA-5001, may have over traditional T cell engineering approaches. In particular, traditional T cell engineering methods typically result in a T cell product that carries two different TCRs, one endogenous and one transferred, which can pair in various combinations of alpha and beta chains and form mixed TCRs with unknown specificities. Intellia researchers are sharing today that the precise replacement of the endogenous TCR with the transgenic TCR (tgTCR) resulted in T cells with improved tgTCR expression levels and in 95% of edited T cells carrying exclusively the desired pairs of the tgTCR alpha and beta chains. This therapeutic TCR profile is expected to yield improved T cell product homogeneity, as researchers showed that Intellias T cell editing approach results in superior function of the engineered T cells toward WT1-positive targets in vitro. This therapeutic TCR profile is also expected to result in lower reactivity against unwanted targets on normal tissues that could lead to toxicities, including graft-versus-host disease (GvHD).

Researchers identified that the selected lead WT1 TCR exhibits high avidity (in the nM range) to its target epitope and shows tight epitope specificity. Being a natural TCR isolated from a healthy donor, it may have a lower cross-reactivity risk than many affinity-matured TCRs. Cells engineered with Intellia's lead WT1 TCR also demonstrated no detectable cytotoxicity toward bone marrow CD34+ cells, which express WT1 at low levels. This is an advantage over current CAR-T cell approaches targeting CD33 or CD123 in AML, which have been shown to induce severe bone marrow toxicity.

Furthermore, the data demonstrate that specific and potent killing of WT1-positive primary AML blasts result from T cells expressing Intellias lead WT1 TCR when cocultured in vitro. This outcome was observed across multiple patient samples that harbor the frequent HLA-A*02:01 allele and that express different WT1 levels as well as AML characteristics. These data validate that the epitope targeted by the lead WT1 TCR, which is distinct from a previously evaluated RMF epitope, is presented efficiently and broadly by AML tumor cells that carry the correct human leukocyte antigen (HLA) restriction. Intellias lead WT1 TCR also has the potential to target WT1-positive solid tumors, such as ovarian cancer, glioblastoma, lung cancer and mesothelioma.

The company plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the first half of 2021 for NTLA-5001 for the treatment of AML. Details on todays presentations on WT1 TCR T cells, including data from ongoing collaborations with researchers at IRCCS Ospedale San Raffaele, Milan, at Keystone are as follows:

First Data Presented on Potential CRISPR/Cas9-Based Therapy for HAE, Intellias Third Development Program

Researchers presented yesterday at the Keystone conference the companys first dataset in support of Intellias development program for HAE. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. The disease is caused by increased levels of the bradykinin protein. Most patients with HAE have a C1 esterase inhibitor (C1-INH) protein deficiency, which normally prevents the unregulated release and buildup of bradykinin.

Intellias HAE treatment hypothesis involves knocking out the kallikrein B1 (KLKB1) gene to reduce kallikrein activity, which is involved in the biological pathway for release of bradykinin. Intellia expects this reduction to correlate with a decrease in bradykinin activity, thus, preventing the activation of endothelial cells that causes vascular leakage and angioedema in HAE patients. The data presented at the Keystone conference showed that the knockout of KLKB1 produces in non-human primates (NHPs) a 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates (Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least five months in an ongoing NHP study, in a highly reproducible manner observed across both rodent and NHP studies.

Similar to its lead in vivo program, for the treatment of transthyretin amyloidosis (ATTR), Intellias potential HAE therapy utilizes the companys modular non-viral lipid nanoparticle (LNP) system to deliver CRISPR/Cas9. Intellias proprietary LNP-based delivery system includes two basic components: Cas9 messenger RNA (mRNA) and a guide RNA (gRNA). The gRNA is the only variable portion of the LNP delivery system and is the sole component that needs to be changed from the LNP-based delivery system that forms the foundation of NTLA-2001, Intellias development candidate for the treatment of ATTR for which the company intends to submit an IND application in mid-2020.

Intellia continues to evaluate several potential guide RNAs and expects to nominate a development candidate for HAE in the first half of 2020. Intellias KLKB1 HAE program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party.

Yesterdays short talk, titled In Vivo Delivery of CRISPR/Cas9 to the Liver Using Lipid Nanoparticles Enables Gene Knockout Across Multiple Targets in Rodent and Non-Human Primates, was made
by Jessica Seitzer, director, genomics, Intellia. These data included results from ongoing collaborations with researchers at Regeneron.

All of Intellias presentations can be found here, on the Scientific Publications & Presentations page of Intellias website.

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.com and follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020; its plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; its plans to nominate a development candidate for its hereditary angioedema (HAE) program in the first half of 2020; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; its presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML and HAE programs, in any future studies, including human clinical trials; its ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; its business plans and objectives for its preclinical studies and clinical trials, including the therapeutic potential and clinical benefits thereof, as well as the potential patient populations that may be addressed by its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc.(Regeneron) and Regenerons ability to enter into a Co/Co agreement for the HAE program; statements regarding the timing of regulatory filings for its development programs; its use of capital, including expenses, future accumulated deficit and other financial results during 2019 or in the future; and the ability to fund operations through the end of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917lynnea.olivarez@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Presents New Data From Its Engineered Cell Therapy and In Vivo Programs at Keystone Symposia's Engineering the Genome Conference...