Category Archives: Human Genetic Engineering

The drought monitor remains essentially unchanged. The areas of abnormally dry and moderate drought are slowly expanding east through central Kansas and deeper into Northwest Kansas. Barton County remains abnormally dry while all of Stafford and the southern two-thirds of Pawnee are on moderate drought. This is being written on the 26th and hopefully the predicted rainfall happened. This wouldnt eliminate dry conditions but would at least prevent them from deepening as would more seasonal temperatures. Last week focused on major stories in agriculture for 2019. This week, lets peer into the crystal ball for potentially important stories for ag in 2020. This isnt a comprehensive list and isnt in any particular order.

Naturally, weather must be on this list and not just weather in our area but nationally and across the world. The entire planet is experiencing more extremes in temperature and precipitation which are having major impacts on agricultural production worldwide. This is especially true in many parts of Africa and Asia. So far the more developed agricultural producers are coping. The key is so far. And this spills over into more than just feeding people but leads to civil unrest within countries and between countries.

Water issues continue to dominate many regions with some planners predicting that if these climate extremes continue, there could actually be armed conflict over water. Within the U.S. conflict concerns water usage urban areas and industry versus agriculture, and water quality for human consumption agricultural and industrial pollutants.

Trade issues remain front and center with the need for all countries involved to pass the USMCA. Additionally, while Phase One is supposedly in place between the U.S. and China, nothing has been signed and many are skeptical China will impost the dollar amount they are promising. And U.S. producers are facing increasing competition from Brazil, Chile, Argentina, and other producers.

The overall farm economy also appears weak. This has resulted in a huge spike in farm bankruptcies. Causes include the trade wars, large surpluses with attendant lower commodity prices, and input costs. This spills into all aspects of farm country where personal income and growth lag far behind more urban areas.

After a bit of a respite, farm consolidation is picking up and we are on track for fewer, large farms. And this consolidation isnt simply with producers but also within all aspects of agriculture from chemical and seed companies to processors.

Help wanted signs will continue to increase as the shortage of skilled necessary labor in all aspects of agriculture continues to worsen. We need tens of thousands of workers from skilled farm and ranch hands to agronomists, crop protection specialists, and in all aspects of input and output of food, fiber, and fuel.

The pace of technological change will continue to accelerate. This includes genetic engineering, drones, and all aspects of precision farming. This is also an area where a lack of skilled educated labor will slow down potential progress and efficiency.

The mental health of those involved in food, fiber, and fuel production will be a major story with the alarming increase in farmer suicides. Private and public agencies are starting to react but the problem still appears to begrowing.

Naturally, there are many others. To all a safe and Happy New Year.

Dr. Victor L. Martin is the agriculture instructor/coordinator for Barton Community College. He can be reached at 620-792-9207, ext. 207.

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2020 - The Year in Agriculture - Great Bend Tribune

As the 2010s draw to a close and we look forward to the new 2020s, Bronwyn Williams takes a quick trip in a hypothetical time machine back to the start of the last decade of disruption and the technological milestones that defined it.

2010

NEW HIEROGLYPHICS

Although, thanks to Brexit and that wall, the utopian globalist agenda may be showing strain at the moment, we can find comfort in the emergence of our new favourite international language the emoji.

In 2010, the first emoji character library was accepted into Unicode, thereby recognising the little symbols we are all so familiar with today as an official universal internet language.

Our contemporary hieroglyphics unite generations and nations in a common visual tongue.

They could also, however, mark the beginning of the end of the age of literacy as we know it today. After all, who needs to text letters when a picture says a thousand words?

Emojis

2011

WHO NEEDS A CAR ANYWAY?

In 2011, the first Uber drivers took to the streets, accelerating both the on-demand and the sharing economy. Commuters could catch a ride at the touch of a button on their smartphones and no longer needed to own their own vehicles to benefit from the convenience of having their own cars.

Today, the e-economy has evened out to allow us to share anything from housing to handbags thanks to companies such as Airbnb and Rent. The take away is that we no longer need to own an object to enjoy it. Also worth considering is how Uber and its fellow gig economy firms are work and law.

Society is still grappling with how to deal with gig workers who report to an app, rather than a human boss, and are not covered by traditional labour laws.

2012

TO THE LEFT, TO THE LEFT

Tinder

The world is in the middle of a sex recession. Teenagers and young adults are more likely to be virgins than their parents and grandparents were at the same age. This phenomenon may or may not be linked to the way interactions are increasingly taking place online, rather than in person.

Tinder, the infamous dating app which launched in 2012, is just one example of how people are turning to technology to help connect with each other with varying degrees of success.

Clearly, though, we are missing something from our fellow humans in our digitally connected world. More and more people are turning to artificially intelligent chat bots, such as Microsofts Xiaoice, which has more than 100 million friends for companionship.

2013

HOLD THE BEEF

The move towards veganism and vegetarianism is a growing global trend. In the US, for example, one in four 25- to 34-year-olds do not eat meat.

Then in 2013, science gave us a way to have our cow and eat it too in the form of synthetic, cruelty-free lab-grown burger patties that look and taste just like the real deal.

Looking ahead, as startups such as Future Meat Technologies make high-tech foods become more accessible, acceptable and affordable, it is likely future generations will view killing animals for food to be a barbaric, embarrassing relic of human history.

Other faux food firms such as Perfect Day and Clara Foods are replacing milk and eggs with artificial imitations indistinguishable from the real product.

2014

WHATS YOUR NUMBER?

In 2014 China started piloting its ambitious, ubiquitous social credit score system to track and rank citizens based on online and offline behaviour.

Built around a national surveillance network, the system rewards good citizens and punishes offenders. Individuals with low scores are denied access to services and freedoms such as using public transport or attending top schools.

Similar human quantification systems can be found in capitalist countries, where consumers are tracked, rated and rewarded by the companies that serve and sell to them. South Africans are familiar with behavioural rewards (and punishment) systems employed by medical and vehicle insurers. Rule by behavioural economics, or nudge is set to grow.

2015

HELLO COMPUTER

Alpha Go

In October 2015, Alphabets artificially intelligent computer programme AlphaGo, beat a professional human Go player for the first time.

This impressive feat of computing prowess reignited the global conversation around the future of artificial intelligence (AI), and the possibility of the so-called Singularity that is when an AI becomes smarter than the entirety of human intelligence. It also reawakened concerns about artificially intelligent machines and algorithms replacing human jobs and perhaps leading to a global post-work economy.

Since then, AI and machine learning have progressed to the point that the worlds top Go player, Lee Se-dol, has retired in defeat, stating that AI cannot be defeated.

2016

FAKE NEWS

In 2016, the website BuzzFeed coined the term fake news in response to a spate of plainly inaccurate, yet intriguingly titled, web articles originating from Macedonia.

Since then, the lies have continued to spread around the world, influencing elections from the US to the UK and South Africa, while the truth limps along behind trying to clean up the fallout.

Fake news, spread via viral clickbait articles shared on social media, has become a global phenomenon with wide-reaching consequences.

Its impact can be felt everywhere from the growing anti-vaccination movement to blame for the re-emergence of once-eradicated measles outbreaks, to the spread of dangerous populist political ideas and the rise of extremist political parties globally.

2017

DEEPFAKE

If fake news was problematic, it was only the start. In 2017 a Reddit user came up with the term deepfake to describe a series of videos he had edited, using a machine-learning algorithm, to transpose famous peoples faces on to porn footage to create convincingly realistic fake movies. In the age of the deepfake we can no longer trust our eyes or ears, as sitting presidents and corporate leaders have discovered to their detriment. Unscrupulous agents can now literally place fake words into real peoples mouths, and put real people into really compromising situations.

Seeing is no longer believing.

2018

SUPERHUMANS

Last year, the first genetically engineered human babies, twin girls, were born in China, ushering in the age of intelligent designer babies.

The girls had been edited using CRISPR Cas-9 technology while still embryos. As the technology progresses, and as more and more governments allow genetic engineering of humans and human embryos, we are sitting on the precipice between natural selection (evolution) and intelligent design.

The ethics of what should be allowed (for example, the eradication of genetic illnesses) and what should be restricted (such as selecting and editing human embryos for good looks or superior intelligence) will be some of the most important questions the human race needs to answer in the years ahead.

2019

REAL WEIRD, REAL FAST

If you thought the last decade was disruptive, just wait until you see what comes next.

This year, Google announced that it had achieved quantum supremacy in other words that the company had managed to demonstrate a successful application of quantum computing.

Should the technology continue to progress from this early sign of success, quantum computing could dramatically increase the processing power and speed of computers as we know them today.

Then add 5G speed internet which is set to roll out in China in early next year to the mix and we can look forward to another decade of superspeed disruptions.

Bronwyn Williams is trend translator at FluxTrends.com

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The decade that disrupted us - News24

Cell and gene therapy manufacturing may never be standardized but the whole industry would gain if firms collaborated to develop common methods for some processes according to an expert.

Manufacturing cell and gene therapies is an expensive business, partly because no two products are made the same way.

A recent study in the journal Nature suggested the average cost of making an autologous cell therapy is between $100,000 and $300,000 per patient.1

The authors attributed the high cost to the use of novel and specialized manufacturing processes [which] make scaling to meet commercial demand a significant challenge for all.

A separate study in the Journal of Clinical Oncology also concluded that difficulties scaling-up the bespoke manufacturing processes and technologies used to make cell and gene therapies significantly increases production costs.2

Market tensions

And high costs are a problem, according to Maria Whitman, managing principal at consulting firm, ZS Associates, who said cell and gene therapy firms need to find more economic ways of making products.

Standardization in manufacturing across the industry is not likely to be the priority for standardization in the short termHowever, the in-market cell and gene therapies have illuminated a number of tensions in the U.S. healthcare system which was designed for pills and biologics.

With over 200 CAR-TCR trials alone in the United States, there is need for standardization of aspects of the process to enable scale and commercial viability of these technologies. The challenge is that, today, each manufacturer is in part by necessity establishing their own process and protocols, she said.

The key is to look for similarities in processes, according to Whitman.

Potential areas for manufacturing and logistical standardization include apheresis protocols, labeling and information management, tracking processes, and training certifications, she said.

Whitman suggested contract manufacturers could help to identify common manufacturing challenges if customers are willing to work together and share information about noncompetitive areas of production.

The process question we should be asking as an industry is this: what is really competitive IP, and what is not? If we answer that, we can identify and solve for more systemic needs.

Logistics is another area where standardization would benefit the sector, Whitman added, citing developers of autologous therapies as the obvious example.

Autologous cell therapies are produced from the patients own cells. Typically the cells are harvested at a clinic and transported to the manufacturing facility before being returned to the patient. Ensuring such therapies are delivered in a timely fashion is vital.

According to Whitman, Manufacturers are trying multiple approaches to streamline the logistics of distance between manufacturing and patient administration. Some are developing in-house solutions and technology or leveraging partnerships to minimize risks and timing.

There is also a new industry emerging of companies forming to solve specific issues including apheresis networks, product manufacturers, as well as companies that create ordering portals, supply chain management systems.

One approach is to localize manufacture. Whitman said, There are already a number of manufacturers working on technologies to make point-of-care cell therapy a reality. Some academics are also creating their own CAR-TCRs, for example, and running trials in parallel with traditional manufacturer trials.

Ultimately the growth of the cell and gene therapy sector will depend on manufacturers ability to balance production and logistics costs with product prices. And the desire to find such a balance is clear, Whitman said.

Manufacturers will look for ways to optimize and automate the process where possible to reduce the cost of skilled human labor and continue to remove risk and drive efficiency in the system.

References1. http://www.nature.com/articles/s41434-019-0074-72. hascopubs.org/doi/10.1200/JCO.18.02079

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IP or Not IP: That Is the Question for Cell and Gene Therapy Sector - Genetic Engineering & Biotechnology News

Science and technology are the great drivers of innovation in the worldaround us.Technological and scientific breakthroughs help people every day, bringing drinking water to the needy, access to information through the internet to remote villages, cures for obscure diseases.

Many aspects of scientific discovery are under no ethical questions. But there are alsoa number of scientific endeavors that push the ethical lines of what science should revolve around. While all the areas of controversy we'll look into have great benefits, they also come with a lot of ethical burdens, like harm to animals, people, or the environment.

It all should make us stop and think, at what point do the negatives of innovation overshadow the good that it brings. And is there ever an innovation so beneficial to the world and mankind that it would be worth ethical tragedy on the road to scientific and technological progress? Ponder these questions as we look into 7 ethically controversial areas of science and technology...

Artificial intelligence is at the forefront of techno-jargon these days. Every company that has anything to do with technology is using it as a buzzword to sell their product. "New dog collar with built-in AI to detect when your dog is in distress! Install our simple computer plug-in and we'll optimize your workday."

AI certainlyhas its applications and benefits, but there are areas where it has some extensive drawbacks. Take two key AI technologies that have questionable benefits, or rather extensive drawbacks: deep fakes and Neuralink.

You've probably heard of deep fakes, the face-swapping technology that is used to make world leaders say things they never didor for less family-friendly things.

You might not know about Neuralink though. It's one of Elon Musk's technological endeavors that aims to improve brain-machine interfaces, record memories, and other technological advancements with the brain.

Focusing in on Neuralink first, questions surround the ethics of connecting human brains to machines and utilizing AI to make human brains function better. Ethical questions primarily focus on the development of said technology and potential side-effects. The company's goal is to optimize human brain function, but the testing that will be needed to get there will be extensive. This means human testing, on human brains, with unknown consequences. At what point is the potential promise of drastic technological advancement not worth the potential human loss in the development of the technology?

RELATED: AI CONTINUES TO ACT IN UNPREDICTABLE WAYSSHOULD WE BE CONCERNED?

Moving from Neuralink, we're met with technology, deep fakes, that pose less benefit to humanity. There's arguably little reason that anyone needs to replace someone's face with another's in a videoat least, little reason that isn't nefarious.

Yet, technology exists thanks to artificial intelligence and machine learning. It continues to be researched under the guise of benefits through improved video editing technology, but at the end of the day,there's no way to keep it from being used for negative purposes.

At the end of the day, artificial intelligence has the potential to completely change how we interact with the worldbut are there too many negatives? Time will tell...

Through CRISPR, scientists are able to edit human genomes. That means researchers can alter DNA sequences and alter how our genes function. That means the potential to correct genetic defects, preventing the spread of diseaseorrr for making designer babies.

CRISPR is short for CRISPR-Cas9, a gene-editing tool that utilizes the Cas9 enzyme to cut strands of DNA. It's basicallylike molecular scrapbooking.

The idea and implementation of CRISPR came from how bacteria defend themselves, by chopping up and destroying the DNA of foreign invaders before they are able to take hold of the organism.

CRISPR was just a theory until in 2017, a paper was published demonstrating just how CRISPR worked.

Chinese scientists have started using CRISPR to engineer designer babies or create human babies withedited genes, primarily lacking any tendency towards genetic defects. All of this seems noble and can potentiallyimprove humanity's quality of life, but at what cost? We largely don't know any potential side-effects and if there are any, we're talking about human life.

Designing humans also brings into question what exactly a human is. Are we naturally occurring beings, or does being a human just mean thinking like we do and form or process doesn't matter?

Moving on from human gene editing in CRISPR, we can examine the ethical issues with gene editing on other beings, like plants. Gene editing encompasses anytime a scientist intervenes in an organism's genetics.

This intervention creates GMOs or genetically modified organisms. This results in stronger, more drought-resistant crops. Or crops that have higher yields per acre, among a bounty of other things.

Today, gene editing happens across the world and it is done on both plants and animals, mostly in the pursuit of better food production. Looking into the animal realm,gene editing has been usedto create pigs that aren't susceptible to Porcine Reproductive and Respiratory Syndrom, or PRRS. Gene editing has been used to create pigs that are naturally very resistant to the disease, improving animal welfare.

The gene-editing process for all organisms is overseen by various federal agencies, obviously depending upon the country you're practicing this science in. It raises many ethical concerns, primarily along with the side-effects that might be caused by it, and it is still a much-debated topic by ethicists.

Animal testing is likely the most controversial area of scientific research on this list. Many people couldn't care less while others vehemently oppose it. For years, animal testing has been used to create newer and better pharmaceuticals, better makeup, better shampoos, etc.

The keyword here is "better" as it means better for humans. At the end of the day, animal testing places the prevention of human suffering over the importance of the prevention of animal suffering. In certain cases, the ethical argument for animal testing is easier, i.e. cancer research, or other pursuits that would prevent human death. In other cases, the argument is harder, as the development of a better lipstick.

The ethical debate around animal testing is essentially a real-life trolley problem. On one hand, you have human suffering and on the other, you have animal suffering. And we seem to have no problem with animal suffering as long as it is for a greater cause.

In introducing the subject, we've made it seem fairly cut and dry, but as science goes, it rarely ever is. An increasing number of scientists are starting to question the relevance of continued animal testing at a time where AI and other tech is starting to be able to accurately model and predict biological interfaces. A great deal of animals are harmed in the creation of many of the chemicals, and we musk as ourselves, is it worth it?

The natural progression from animal testing is human testing or trials of medication on human test subjects. Human subject research is often necessary to get drugs to the final phase of regulatory approval. It serves as the final check of how a givenmedicine or chemical will interact with the human system. Yet, time and time again it has hurt, maimed, or killed individuals. And we have to ask ourselves again, at what point does it become not worth it?

History hasn't been kind to the reputation of human trials, though scientists are making aconstant effort to create safety standardsin the process.

In 1947, it was discovered that two German physicians conducted deadly experiments on concentration camp prisoners during WWII. They were prosecuted as war criminals in the Nuremberg Trials. The Allies then established the Nuremberg Code, being the first international document for voluntary human consent for research.

With human testing today, the testing proceeds onlyif the patient consents to the study. Though this often leads to people with lesser fortune signing up for human trials to earn some extra cash. The ethics of the entire research situation can still be hotly debated.

Military weapon development is another major crossroad of science and ethics. Take, for example, the development of the atomic bombs during the Manhattan Project during WWII. In many ways, the research conducted during these experiments furthered humanity's understanding of atoms, molecules, and quantum. In other ways, this research killed tens of thousands of people.

Military power and weapon technology pose an ethical dilemma largelydue to the nature ofhumankind. If a givencountry doesn't invest resources into developing the best weapons technology, then another more powerful country will simplyswoop in and overpower them. That's the way it works nowadays. It's the unfortunate truth of the interaction of global superpowers. And once again, we're met with a real-life trolley problem.

Do we invest scientific resources into developing better weapons to protect ourselves and thus kill others, or do we let ourselves be killed and "protect" others? We would certainlynot opt for the latter, would we?

Since it seems like the earth has seen better days, maybe it's time to just abandon our planet and move to a new clean slate, like Mars. We know that there is flowing liquid water on Mars somewhere, and we know there are also other resources to help us survive.

So, why not just up and move humanity there?

The biggest ethical questions around Mars colonization are presented when you consider the potential of life on Mars or the potential of future life on Mars. We can't state with absolute certainty that there is life on the planet. Moving humanity there could harm it. We also don't definitively know that life won't occur on the planet through natural means. If humanity moving there interrupts the natural progression of Mars life, isn't that an ethical issue?

RELATED: SPACEX IS PREPARING A MISSION TO COLONIZE MARS BY 2026

The answers to those suppositions largely have to do with how humanity in total should approach its ethical responsibility. If you believe humanity's only ethical responsibility is to themselves, then it's likely not an issue. If you believe that we're responsible for all lesser life forms, then you'll run into countless ethical dilemmasin the process.

Closing out this discussion of ethical dilemmas in science and technology we're left again wonderingwhat are innovation and the betterment of humanity worth? The answer to that question will vary depending upon who you ask... but ask yourself, what is innovation worth?

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7 Ethically Controversial Research Areas in Science and Technology - Interesting Engineering

CRISPR, a tool used to edit genes, has the power to shape the future of the human genome. The international community is wary of the technology, but a lone Russian scientist says we should already be doing more tests...on human embryos.

Denis Rebrikov would have been following the news with great interest when CRISPR, a method for editing the genes of living organisms, made international headlines last year.

It was reported that a Chinese geneticist named He Jiankui had edited the genomes of twin girls without consulting the global scientific community. Known to the world as Lulu and Nana, the babies had their CCR5 gene altered in the womb in the hope of improving their resistance to HIV. When Hes experiments were made public, the Chinese authorities cracked down on his research, and the resulting international uproar led to further restrictions on human testing using CRISPR.

As it turned out, Rebrikov had already been planning his own tests for quite some time.

A geneticist himself, Rebrikov worked for years in relative obscurity at the Pirogov Russian National Research Medical University that is, until he went public with his intentions last summer to pick up the torch where He left off. When I see a new technology come forward, he says, I want to see how it works and how I can improve it. Where Rebrikovs vision differs from Hes is in whether or not experiments should be conducted openly or not. The Russian scientist believes everything should be done in the public eye, and with the involvement of the state.

Conversations over genome editing are nothing new for Russia. In fact, a public conversation on its national importance has been taking place over the past two years.

A watershed moment came in 2017 when President Vladimir Putin addressed a youth forum in Sochi. In some of his first public comments on the subject, the president described the technologys potential applications, from the medicinal to the military, calling its use (and potential misuse) as fearsome as the atomic bomb. Elsewhere he confirmed it as a technology that will determine the future of the whole world.

Accordingly, Russia has been investing heavily into genetic research. $2 billion was reportedly spent on establishing official research programs in 2017, with an additional $3.3 billion invested this past April. The payoffs when they come will be enormous. Not only may the health of the nation be improved: as with any technology, innovation brings with it a geopolitical edge. Words like biodefense have circulated within the upper echelons of Russian society, and major figures like Mikhail Kovalchuk (director of the Kurchatov Institute, made famous by this years Chernobyl series) have pushed for Russia to become a global leader in genetics.

This kind of environment is encouraging for figures like Rebrikov, who decided to go public in June with his intention to continue working with genes affecting HIV transmission. But there was difficulty in finding parents who were willing to participate in such a study, so Rebrikov changed course and decided to work with genes connected to hearing loss in children. He found five couples who would qualify for the experiment; one of which met with the scientist to discuss potential risks and benefits. The couple as of yet has not decided on whether they want to participate, even in theory.

Rebrikov hadnt gotten as far as He Jiankui before becoming an international sensation, but this was intentional: he may want the scientific community to know there are no secrets in his lab. For him, comparisons to nuclear weapons can be taken in his stride. The situation is completely analogous to developing an atomic bomb, he says. Can bad people use technology for bad purposes? Of course, but did ethical concerns stop the Soviet Union from doing so?

While international the reaction has not been as heated as it was with He, there have been numerous articles published in major journals like Nature and Science demanding that the international community pressure Rebrikov to stop any future applications of the technology. Some have gone as far as to call him rogue.

In contrast with policies in China and the United States, though, Russias response has been more cautiously optimistic. Whereas other global powers have placed effective moratoriums on embryonic genetic editing (with little chance of these policies changing any time soon), an official panel including leading Russian experts met in July to discuss the question. Figures ranging from Kovalchuk to prominent endocrinologist Maria Vorontsova were invited to speak at the gathering.

Human embryo

The scientist also receives support from the Pirogov Institute. Sergey Lukyanov, Rebrikovs colleague and former PhD advisor, says that his intentions are admirable: [He] is one of those people who takes action towards any imperfection of the universe that can, from his point of view, be corrected. For him, this is an opportunity to bring happiness to parents to have healthy children.

Rebrikov is not without his critics, however. Prominent researchers like Pavel Tishchenko, a bioethicist at the Russian Academy of Sciences (RAS) Institute of Philosophy, have called for increased restrictions. Tishchenko organized an ethics panel in October 2019 to review the case and is concerned that parents might not be aware of all the risks involved, or that ethics and regulatory committees might not be as rigorous as necessary.

One of the main questions that needs answering, Tishchenko has said, is who will bear responsibility for possible complications down the line. The edited genes in the Chinese twins might have effects beyond HIV resilience (especially as the CCR5 gene is linked to memory formation), and he claims that todays scientists are not equipped to make the necessary judgment calls.

The Russian Ministry of Health has since come out with an official statement calling genetic experimentation on humans premature. Interesting enough, however, no concrete regulations have been introduced that would definitively prohibit experiments like the ones Rebrikov suggests. Under the current rules, a grey zone exists that may allow for certain experiments depending on whether or not the embryos were created for research purposes or previously discarded, or on whether the experiments are conducted for research purposes or for a clinical trial.

For now, it seems like Rebrikov has put some of his plans on hold. He has said publicly that he will definitely not transfer an edited embryo without the permission of the regulator, but all the same has expressed frustration with the delays. I want the rules to be set, he said, but nobody is doing this. Moreover, the couple which consulted with him has not yet expressed interested in progressing further, and the global attention paid to his research may make any future missteps into a potential international incident.

But temporary setbacks are no guarantee that the status quo is going to last. The current regulatory limbo, despite the rhetoric from Rebrikovs critics, still allows for dramatic steps to be made in the future. And given the potential for genetic engineering to change the world, it may be that Russia may still allow Rebrikov and his team to develop their research further than any other scientist on the planet.

Only time will tell.

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Meet the Russian geneticist who wants to edit your children - Russia Beyond

For all of the wondrous potential of immunotherapies, there have been some notable obstacles in the early goings. Engineering immune cells to attack cancerous tumors can lead to solid results shortly after administering a dose, but for many patients the effects wear off once rapidly mutating tumor cells acquire new defense mechanisms.

Cellectis (NASDAQ:CLLS) thinks it may have a partial solution. In mid-November, the gene editing company published the results from a proof of concept study for its "smart" immunotherapy approach. Is the technique the future of cellular medicine?

Image source: Getty Images.

Today, cellular oncology therapies genetically engineer immune cells to bolster their safety and efficacy as a cancer treatment. There are T cells, natural killer (NK) cells, tumor infiltrating lymphocytes (TILs), and others. They're often engineered with chimeric antigen receptors (CARs) or T cell receptors (TCRs), which allow them to home in on and suppress specific genes in cancer cells.

While current-generation CAR T cells or CAR NK cells are capable of mounting formidable attacks on tumors at first, treatment responses aren't durable for all patients. That's because cancer cells mutate to rely on different proliferation genes, or secrete new molecules into the tumor microenvironment that neutralize immune cells. Meanwhile, overstimulating the immune system can reduce the potency of immune cells and lead to devastating side effects, such as cytokine release syndrome.

That prompted Cellectis to design "smart" CAR T cells capable of adapting to changes in the tumor microenvironment. In a proof of concept study, the company utilized synthetic biology concepts to rewire genetic circuits in three different genes of the initial T cells.

One edit made the immunotherapy more potent, but in a controlled manner to reduce off-target toxicity. The other two edits imbued CAR T cells with the ability to secrete inflammatory proteins inside the tumor microenvironment in proportion to the concentration of cancer cells.

In other words, the smart CAR T cells only asked for help from the rest of the immune system when it was needed most, which increased the anti-tumor activity of treatment and made native immune cells less likely to become neutralized. That should reduce the likelihood of triggering cytokine release syndrome, the most common (and potentially fatal) side effect of cellular medicines, which is caused by high concentrations of immune cells.

The study was conducted in mice, which means the safety and efficacy observations can't be extrapolated into humans. But that wasn't the point. The proof of concept demonstrates that the basic idea of engineering tightly controlled genetic circuits into immunotherapies is feasible. It could even allow multiple genetic circuits of the same drug candidate to be tested against one another in parallel, hastening drug development and lowering costs. Is it the inevitable future of cellular medicine?

Image source: Getty Images.

Gene editing tools are required to engineer immune cells. In fact, immunotherapies are the lowest hanging fruit for gene editing technology platforms today. It's simply easier to engineer immune cells in the lab (ex vivo) than it is to engineer specific cell types in the complex environment of the human body (in vivo).

That explains why nearly every leading gene editing company has immunotherapy programs in its pipeline. Coincidentally, all of the leading drug candidates in the industry pipeline are off-the-shelf CAR T cells engineered to treat CD19 malignancies such as non-Hodgkin's lymphoma (NHL) and B-acute lymphoblastic leukemia (B-ALL), regardless of the gene editing approach used. The smart CAR T cells designed by Cellectis targeted CD22 malignancies, but the approach could be adapted to CD19 antigen.

Developer(s)

Drug Candidate

Gene Editing Approach

Development Status

Cellectis and Servier

UCART19

TALEN

Phase 2

Precision BioSciences (NASDAQ:DTIL)

PCAR0191

ARCUS gene editing

Phase 1/2

CRISPR Therapeutics (NASDAQ:CRSP)

CTX110

CRISPR-Cas9

Phase 1/2

Sangamo Therapeutics (NASDAQ:SGMO) and Gilead Sciences (NASDAQ:GILD)

KITE-037

Zinc finger nuclease

Preclinical

Data source: Company websites.

Will these companies eventually turn to "smart" immunotherapies with regulated genetic circuits? It does seem inevitable, especially if the approach can reduce or eliminate cytokine release syndrome and enable more durable responses.

For example, Cellectis reported that all seven patients taking part in the phase 1 trial of UCART19 suffered from at least grade 1 cytokine release syndrome, which caused complications that led to the death of one patient. Five of the seven patients achieved molecular remission, but one relapsed (and remained alive) and one died. To be fair, all patients taking part in the trial had advanced, heavily pretreated B-ALL.

Precision BioSciences has encountered similar obstacles in an ongoing phase 1/2 trial of PBCAR0191. The company's lead drug candidate was administered to nine patients with NHL or B-ALL. Three cases of cytokine release syndrome were reported, but all were manageable. Seven responded to treatment, including two that achieved a complete response, but three eventually relapsed.

CRISPR Therapeutics recently began dosing patients with CTX110 in a phase 1/2 trial that will eventually enroll up to 95 individuals, but initial results won't be available until 2020. Sangamo Therapeutics and Kite Pharma, a subsidiary of Gilead Sciences, are plowing ahead with zinc fingers,but are still in preclinical development.

Investors seem pleased with most of these gene editing stocksright now. After all, despite the obstacles, current-generation cellular medicines are delivering impressive results in patient populations with relatively few options. But upcoming data readouts could easily differentiate the pack. That could increase the need to invest in augmented capabilities, such as smart immunotherapies.

There's plenty of untapped potential in cellular medicine. Today, companies are developing drug candidates with engineered CARs and TCRs designed to test hypotheses about the function of immunotherapies. As approaches find success, measured in safer and more durable responses, the next layer of complexity will be added in an effort to find even more successful therapies. And the cycle will continue.

Therefore, it seems inevitable that the field of cellular medicine will turn to smart immunotherapies with more complex genetic edits, much like the field quickly embraced the need for engineered immune cells and off-the-shelf manufacturing processes. That said, the immediate focus for Cellectis and its peers is building a stable foundation -- and those efforts have only just begun.

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Did Cellectis Just Provide a Glimpse of the Future of Cellular Medicine? - The Motley Fool