Category Archives: Human Genetic Engineering

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

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

CHINA has built an isolation hospital for coronavirus sufferers in six days. In the spirit of socialist emulation, the team building the Wuhan facility aimed to beat the seven-day record set in Beijing during the 2003 Sars emergency.

As this weekends weather events have shown, Britain is yet to set in place effective flood controls.

China is a big economy and can mobilise very considerable human and material resources. But Britain is, by comparison, a mature economy the fifth-largest in the world and has yet to lay the first sleeper in the second high-speed railway.

The Chinese have offered to build this disputed bit of infrastructure by the middle of the decade. It might be a good idea to suggest they take the contract to build a decent system of flood defences first and then have a crack at building HS2 top-down from the north andupgrade the regional rail system while they are at it.

Two crises, two systems.

It is impossible for the lay person, from outside the charmed circles of experts, to make informed decisions about the feasibility or the costs of infrastructure projects of this scale.

That is why governments have to take leadership responsibility, make the people charged with these tasks accountable and keep a sharp eye on the costs and commercial advantages that accrue for the people and enterprises involved.

The starting point for any project at the scale required to modernise Britains rail network (and for that matter our coastal and flood defence systems) is the human and social needs measured against the environmental and social costs of not getting on with it.

By the same token, the starting point for any response to a medical emergency of the kind presented by this new mutation of the coronavirus must be the public health priorities that it raises.

It is hard to imagine that Britain, relying on an increasingly privatised health system, a civil engineering sector dominated by large, larcenous and frequently failing firms in many cases owned and managed by dynasties of extremely reactionary hue to be able to either conceive of such projects, let alone carry it out to such a tight timetable.

The reason China can do this is not due to any inherent characteristics of the Chinese people, any unexplained genetic predisposition, but simply the nature of the actually existing social system.

China can mobilise these enormous social forces, can direct these immense human resources, and gain the enthusiastic human engagement of the necessary millions precisely because the commanding heights of the economy and the decisive levers of power are, in essence, socially owned and directed.

This is not to say that every aspect of the way China goes about things would go down particularly well in Britain. That being said, there is a fairly substantial constituency of opinion who might give very serious consideration to implementing the Chinese policy of shooting corrupt bankers.

And the practice of imposing powerful sanctions including long prison sentences on political and government officials, or the managers of enterprisesguilty of negligence and corruption in their public roles is something that would represent a sea change in the way Britain deals with these matters.

Capitalism, as a system for running complex modern economies and managing advanced and modern states, continually demonstrates its redundancy.

We have powerful examples of different ways of doing things. Britain needs to find its own way to ensure that the system of ownership and control corresponds to the real needs of our people and the harmonious and productive development of our economy. It is demonstrably clear that this is not capitalism.

Link:
There's much to learn from China's mobilisation in the face of crisis - Morning Star Online

The power of speculative fiction often lies in its ability to make us look at the world around us with fresh eyes. Mundane acts have a way of becoming extraordinarily beautiful when we are faced with the prospect of their vanishing. Here, baseball becomes a site of resistance, an emblem of humanity, an antidote to the automation and artificial intelligence that controls every other aspect of life in AutoAmerica. After all, what would be the point of automating such a thing as nine human players throwing and catching balls to the best of their physical abilities? What significance could there possibly be in a robot pitching a perfect game? We are here, one coach says late in the novel, because we believe anything can happen in a ballgame. You can get a guy and all his stats but give him a stick to swing, and you still dont know what will happen. Its a marvelously refreshing concept in a world that is otherwise dominated by algorithms.

The Resisters is a book that grows directly out of the soil of our current political moment, and much of the books unsettling pleasure lies in Jens ingenious extrapolation (or, in some cases, redescription) of contemporary problems. The book brims with EnforceBots (police robots), ThoughtCommand (next-level voice command), PermaDerms (permanent skin whitening) and SmartGuns. AutoAmerica is a nation shaped by policies like ShipEmBack, a mass deportation of immigrants, and the One Chance Policy, wherein Surplus families are permitted only one pregnancy, no matter the outcome.

Jen has such a gifted ear for the manipulative languages of tech, marketing and government that at times the sheer abundance of clever details threatens to overwhelm the stories of her characters. But perhaps this overabundance is part of the novels method, a way of swallowing the characters and the reader into AutoAmericas reality. The Resisters is aimed at many catastrophes at once: surveillance technology, government overreach, authoritarianism, automation, economic inequality, racism, sexual assault and the institutional mishandling of it, geopolitical conflict and climate change.

The central thread of the book, though, or perhaps the most lingering, is its obsession with the threats of artificial intelligence. The Resisters is full of characters who voluntarily hand over their humanity by agreeing to GenetImprovement or by mindlessly following the orders of Aunt Nettie. In one unnerving section, the narrator recounts the incremental steps that led to this all-encompassing control first, he let Aunt Nettie keep his calendar, then respond to emails on his behalf. (The Resisters might make you stop and actually read your user agreements.)

In the most devastating moment of this ultimately quite tender novel, one characters mind is surgically merged against her will with Aunt Nettie, so that the line between human and internet is no longer clear, even to herself. Crucially, it is other human beings who carry out this dreadful procedure, which suggests that even in a dystopian world dominated by artificial intelligence, people are still the ones who carry out the most atrocious acts.

We live in a moment when The Handmaids Tale is a hit television show, and Kellyanne Conways use of the term alternative facts reminded so many readers of the double talk in George Orwells classic 1984 that the novel hit the best-seller list seven decades after its original publication. The public seems to feel that the worst speculative fictions are coming true. Of course, Margaret Atwood would contend that The Handmaids Tale was true even as it was written. Perhaps Gish Jen could make a similar argument about much of The Resisters. The hope she offers, though, lies in the books title, and in the heroism of its family of Bartlebys, who resist both the lure of conveniences and the threats of the powerful, with one phrase: I would prefer not to.

See the original post here:
The Future Is Here, and Uncomfortably Close to Home - The New York Times

Editors note:Dr. Shedingeris a Professor of Religion at Luther College in Decorah, Iowa. He is the author of a recent book critiquing Darwinian triumphalism,The Mystery of Evolutionary Mechanisms: Darwinian Biologys Grand Narrative of Triumph and the Subversion of Religion.

Why should advocates of intelligent design care about a French Jesuit priest who died more than 60 years ago? Pierre Teilhard de Chardin (1881-1955) along with being a Jesuit priest was also a geologist and paleontologist who made several trips to China to participate in geological and paleontological work (he was part of the team that discovered Piltdown Man, later revealed to be a hoax). But Teilhard is best known for his book The Phenomenon of Man, published in French in the 1930s and in English in 1955. In this book Teilhard lays out a vision for the evolutionary process that is at odds with the established scientific view but is consistent with his own religious convictions.

Teilhard argued that the science of his time had a truncated view of evolution. Scientists studied the evolutionary process as if it were a movie playing on a screen in front of them with the scientists themselves as mere passive observers. Teilhard thought that evolution needed to be viewed from the inside, viewing humans not only as observers of evolution but also as its products. As such, Teilhard conceived evolution as occurring on four levels, only two of which were acknowledged by establishment scientists.

The first of these levels he called cosmogenesis, the evolution of the physical universe. The second level he labeled biogenesis, the evolution of life in the physical universe. According to Teilhard, this is where evolutionary biologists had traditionally stopped. But a full accounting of the evolutionary process, he believed, required two additional levels: psychogenesis, the evolution of consciousness in biological organisms, and noogenesis, the evolution of reflective thought, a characteristic unique to humans. With the evolution of humans, Teilhard believed evolution had crossed what he called a threshold of reflection that would fundamentally alter the very course of evolution. Rather than a billion-fold trial and error, evolution would now proceed more intentionally through the exercise of the human mind. We should remember that Teilhard formulated these ideas in the 1930s, long before anyone had conceived of the possibility of genetic engineering. Teilhard was prescient.

Having fully accounted for the evolutionary process, Teilhard went on to articulate his most controversial idea. He argued that over time, human minds would eventually form a web of reflective consciousness enveloping the Earth (what would he think of the Internet?!). He called this the noosphere. In time, the noosphere would reach an omega point where consciousness would completely fuse with the God who created it. Teilhards view of evolution was thus highly teleological. The evolutionary process existed for the purpose of creating beings with the ability of reflective thought so that they could commune with their Creator. No Darwinian contingency here!

Not surprisingly, most Darwinians howled with derision at Teilhard. In response to The Phenomenon of Man, Nobel Laureate Peter Medawar published one of the most devastating book reviews ever written. Medawar called Teilhards book nonsense, tricked out with a variety of metaphysical conceits, and its author can be excused of dishonesty only on the grounds that before deceiving others he has taken great pains to deceive himself. For Medawar, reading Phenomenon brought on feelings of real distress, even despair. Despite this, many philosophers and theologians found Teilhards book of great interest. But according to arch-Darwinian Daniel Dennett, the esteem with which non-scientists held the book is nothing more than a testimony to their depth of loathing of Darwins dangerous idea, a loathing so great that it will excuse any illogicality and tolerate any opacity in what purports to be an argument. The Darwinian reaction to Teilhards explicit evolutionary theology is of course to be expected. What we dont expect is to find that this disdain was not shared universally within the Darwinian establishment.

Enter Theodosius Dobzhansky, perhaps the most important figure in the history of evolutionary theory after Darwin. In his oft-cited essay Nothing in Biology Makes Sense Except in the Light of Evolution, Dobzhansky unexpectedly calls Teilhard one of the great thinkers of our age. As a man of deep Christian faith himself, Dobzhansky clearly resonated with Teilhards attempt to create a synthesis between evolution and religious thought. In fact, Dobzhansky appears to have been so taken with Teilhards work that he served for a year as president of the North American Teilhard Society (1969). Of course, we will never learn from the textbooks that a figure as central to the modern evolutionary synthesis as Dobzhansky seemed to embrace an explicitly teleological and even theological understanding of evolution. I suppose Dobzhansky was deceived (according to Medawar) or prone to illogicality (according to Dennett)!

Of course, neither Teilhard nor Dobzhansky appears to have made an explicit design argument. They would be better categorized as theistic evolutionists. For Dobzhansky this is confirmed when in his previously cited essay he states, There is, of course, nothing conscious or intentional in the action of natural selection. Here Dobzhansky adheres to the standard Darwinian story. Yet just a few lines later he notes humans ability to make conscious, intentional decisions, and concludes, This is why the species Homo sapiens is the apex of evolution. The incompatibility between these two statements seems not to have occurred to Dobzhansky. Clearly, a process with no direction or larger purpose by definition has no apex. His attempt to hold to both an orthodox Darwinian viewpoint and an orthodox Christian viewpoint simultaneously dissolves into incoherence. Theistic evolutionary schemes seem to be a logical dead end.

While Pierre Teilhard de Chardin may not have been a forerunner of intelligent design thinking per se, the significance of his pointing out the incomplete nature of the evolutionary theory of his day should not be underestimated. As Thomas Nagel would argue today, any theory of evolution that excludes the origin of mind and consciousness from consideration is at best half a theory. Teilhard noticed this weakness of Darwinian evolutionary theory nearly a century ago, and at least one very prominent Darwinian may well have agreed, even if he never admitted it in public.

Photo: Pierre Teilhard de Chardinin 1947, viaArchives des jsuites de France [CC BY-SA].

Go here to read the rest:
Teilhard de Chardin and the Incomplete Nature of Evolutionary Theory - Discovery Institute

Imagine a world where a mosquito bite is just an itchy annoyance. No malaria. No dengue fever.

Last month, scientists announced they had taken one more step toward that vision. A paper in the journal PLOS Pathogens described how they synthetically engineered mosquitoes to stop the spread of dengue fever, a viral tropical disease that sickens as many as 100 million people each year.

Now imagine genetically tweaking an invasive species of mosquito to save native Hawaiian birds from extinction, or transferring genes from one species of endangered chestnut tree to another to help the latter resist blight. Employing the same sort of genetic engineering used to make a plant-based burger bleed, scientists are beginning to explore the ways synthetic biology could help protect biodiversity and conserve species.

Synthetic biology, or synbio, employs the latest and greatest gene-editing tools, such as the cut-and-paste technology known as CRISPR-Cas9. Combined with new techniques to digitize and automate the design and modeling of various genetic elements, scientists can now engineer organisms to produce novel food ingredients or to rewire the switches that express genes that control certain functions.

In the case of those dengue-carrying mosquitoes, scientists genetically tweaked members of the Aedes aegypti species by transferring genes from the human immune system that create an antibody to suppress dengue fever into the blood-sucking insect. The antibody is activated and expressed once the female mosquito draws blood. In effect, the mosquito is cured of dengue fever before it can transmit the disease.

The next step would be to propagate the new genetic element to confer dengue immunity through a population. Thats where a gene drive comes in. Gene drive systems, which can be natural or synthetically engineered, skew inheritance of a certain genetic element so that it will spread more quickly through generations.

The idea is to bypass normal inheritance rulesthat classic Darwinian concept that inheritance is driven by genetic variations that improve an organisms ability to compete in a dog-eat-dog worldso that re-engineered traits become dominant.

In terms of conservation, synbio could potentially address several areas of concern, such as curbing invasive species, reducing pressures from wildlife trade, improving resistance to disease, and even bringing a species back from the brink of extinction.

Biologists at the University of California San Diego (UCSD), who also led the team that wrote the PLOS Pathogens paper on mosquitoes, developed a novel gene drive system for manipulating genetic inheritance in Drosophila suzukii, a fruit fly with the common name spotted-wing drosophila.

This particular pest, native to Japan and first discovered in the US in 2008, injects its eggs into soft ripening fruit like berries. Current practices to defend against spotted-wing drosophila rely on either heavy insecticide use or early harvesting. Its estimated the pest costs the US economy as much as $700 million each year in losses.

The engineered gene drive from UCSD, dubbed Medea after the character in Greek mythology that killed her offspring, uses a synthetic toxin and a corresponding antidote function to achieve 100 percent inheritance bias in less than 20 generations.

This genetic Trojan Horse could then be used to spread elements that confer susceptibility to certain environmental factors, such as triggering the death of the modified fruit flies at a certain temperature.

UC San Diego associate professor Omar Akbari told Singularity Hub that his team is getting close to field testing some of our technologies. The furthest along for our group would be the use of [precision guided sterile insect technique] to control wild populations of D. Suzuki.

A number of companies are turning to synbio to create ingredients where the natural product is expensive, rare, or threatened. Take the well-known example of vanilla. Most products on the market use a synthetic version of vanillas main ingredient, vanillin, made from petrochemicals.

Swiss company Evolva has developed a genetically modified yeast to produce vanillin in a manner similar to brewing beer. Modern Meadow also uses DNA editing tools to engineer specialized collagen-producing yeast cells for making leather products.

In a case more directly related to wildlife conservation, Singaporean scientists engineered a synthetic replacement for horseshoe crab blood cells, which have been used in biomedical applications for decades. All four species of horseshoe crabs are considered imperiled by the International Union for Conservation of Nature (IUCN).

However, while a replacement product for horseshoe crab blood has been commercially available for more than 15 years, it has yet to be broadly adopted for various reasons. Thats finally changing, as new studies have confirmed that available synthetics are just as reliable as horseshoe crab blood for detecting endotoxins in biomedical manufacturing.

The long-lived American chestnut was once one of the dominant tree species of forests in the eastern US. A blight from Asia introduced in the late 1800s has all but wiped them out. Efforts to breed American chestnuts with disease-resistant chestnut trees in China have had limited success, as its not easy to propagate the desired traits from several genes through succeeding generations.

A project led by the College of Environmental Science and Forestry in Syracuse, New York is using synbio to produce a blight-resistant American chestnut without even harming the fungus.

The researchers have copied a single gene from wheat and transferred it into American chestnuts. The gene produces an enzyme called oxalate oxidase that doesnt kill the fungus. Instead, it breaks down the fungus toxin that attacks the trees tissue properties.

The bonus is that the fungus itself is left untouched, so the blight remains dormant and doesnt evolve resistance over time.

While bringing the dead back to life is one trick that will likely elude scientists in our lifetime, synbio researchers have been actively working to resurrect the woolly mammoth and other extinct species such as the passenger pigeon, which disappeared for good more than a century ago.

These projects arent strictly creating pure examples of these long-gone species. Rather, scientists are inserting sections of ancient DNA code into modern relatives. In the case of the woolly mammoth, researchers are attempting to create a mammoth-elephant hybrid using the Asian elephant.

Proponents of this sort of resurrection science say its less about trying to revive extinct species than about saving those that are currently at risk of disappearing. The Asian elephant (Elephas maximus) is on the IUCN Red List of Threatened Species.

A team led by George Church out of Harvard University hopes that by transferring genes in the mammoth genome to the Asian elephant it will be able to survive in the Arctic; relevant genes might include those that code for extra fat and dense hair. That would extend the animals range into regions that are already changing due to a warming climate.

Like geoengineeringmanipulating the environment to stave off the effects of climate changebioengineering has its critics and detractors. Some react viscerally to the idea of altering natural systems in any way.

One of the main arguments revolves around the concern that introducing a genetically modified species could have unintended consequences. While no one expects a Jurassic Park scenario where genetically enhanced monsters chase Jeff Goldblum through the jungle, there is a chance that genetically tweaked traits could jump species or otherwise go off script.

Kent Redford believes fostering a conversation about the possible advantages and disadvantages of the role of synbio in conservation is important regardless of where one stands on the divide.

My mission is to make sure that the conservation community knows about these technologies and has taken a considered and informed opinion on them, and tried to influence [these] technologies for the good of biodiversityto minimize harm and to increase positive outcomes, he told Singularity Hub during a phone interview.

A conservation expert who has served at the The Nature Conservancy and Wildlife Conservation Society, Redford is the chair of an IUCN task force on synthetic biology and biodiversity conservation. He was the lead editor on an assessment report, Genetic Frontiers for Conservation, which will be presented this summer at the IUCN World Conservation Congress in France.

The opinion of the IUCN matters. Its 1,300 member organizations include governments, non-governmental organizations, business associations, and scientific and academic institutions.

Redford declined to speculate as to what sort of recommendations may come out of the IUCN meeting. He did note that the intersection of synbio and conservation remains on the periphery for many in the conservation community.

Most of my colleagues dont see why they should be paying much attention to this, he said. Some of those who are aware of these emerging technologies consider them to be relevant tools to help solve some of the intractable problems in conservation. Others believe these genetic techniques have the potential to completely ruin the natural world and the lives of poor people.

Akbari agreed that the biggest challenge for synbio in conservation isnt the technology but securing regulatory approvals and public support. I think we need time, he said. As more technologies are developed and tested with positive outcomesI believe the resistance will lessen.

While the scientific community debates the potential and the pitfalls of synbio, biodiversity will continue to decline.

A report last year by the United Nations Intergovernmental Science-Policy Platform on Biodiversity and Ecosystem Services issued a number of disturbing statistics. For example, the average abundance of native species in most major land-based habitats has fallen by at least 20 percent, mainly since 1900. And nearly 10 percent of all domesticated breeds of mammals humans have used for food and agriculture throughout history were extinct by 2016, with at least 1,000 more breeds still threatened.

I think the natural world is in serious trouble, Redford said. Whether synbio can be part of the answer to that problem remains a big question.

Image Credit: Image by RayNight from Pixabay

Read the original post:
Engineering Bugs, Resurrecting Species: The Wild World of Synthetic Biology for Conservation - Singularity Hub

After a week at the World Economic Forum in Davos this month, I'm convinced the globe faces two existential threats that demand disruption of our businesses, our policies and indeed our lifestyles: climate change and health assurance for all.

Both will require disruption in our way of thinking, creative partnerships with entities that have not worked together to create new ecosystems, as well as artificial intelligence and other new technologies that may be game-changers if constructed properly.

Just as climate change cannot be solved by the energy industry alone, health assurance cannot be solved by the healthcare delivery industry alone. At Davos, I served as a distinguished fellow of the World Economic Forum, charged with developing equitable and sustainable business models for the transition to a digital economywhat World Economic Forum founder Klaus Schwab called the "fourth industrial revolution."

The fourth industrial revolution can be defined in a pretty nonthreatening way as the blurring of boundaries between the digital, physical and biological worlds, as a fusion of advances in artificial intelligence, robotics, the internet of things, genetic engineering, quantum computing, 5G and the kitchen sink of exciting new technologies that will blossom in the next decade.

But the impact of a digital economy on healthcare will be immense. Which is why at Davos I advocated that we talk less about the technology of self-driving cars, and more about self-healing humans.

After presenting at 10 sessions at the forum, here's my framework for equitable and sustainable models of change:

Start with ethics. Trust is more important than technology. Ethics must be injected into product development at the very earliest stage, when values are being assessed. Don't wait until a product is ready for market and then ask marketing to make it trustworthy.

Reach across industry. We talk and plan in silos, but health assurance only comes when our industry talks with those involved in food, transportation, education, policy and the creation of jobs.

There's no such thing as "non-disruptive" disruption. By definition, disruption will be painful to those who don't want to think differently as new ecosystems are built.

Data is not the new gold, but intellectual property is. We must understand how intellectual property is derived from the personal data of our patients, and ensure there are bright lines for enhanced consent in the use of this data.

Never forget the human in the middle. As online meets offline, the excitement tends to focus on the technology. But what's exciting is focusing on humanson new roles for clinicians in the online-meets-offline world, on new services for patients. And on what I call health assuranceconstructing a system where the primary goal is a healthy and happy life for all.

As in the climate change crisis, it's the humans who will create the revolution. There is, in fact, an army of Greta Thunbergs in healthcare. You see them in the patient empowerment movement. You see them among our students, who are deeply concerned about social justice in a world that doesn't reimburse for it. You see them in the calls for gender and racial equality. And you can find them in the frontiers of the digital health movement.

I've long believed that if you want to see the future, find good people who are uncomfortable with the status quo. Our job is to go to places like Davos and advocate for those peoplefor a system that can be brilliant when caring for the sick, but also enhances health assurance across all boundaries.

Originally posted here:
Do we need a Greta Thunberg in healthcare? - ModernHealthcare.com