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Category Archives: Genetic Engineering
Posted: October 16, 2021 at 2:13 am
Genetic engineering opens new possibilities for biomedical enhancement requiring ethical, societal and practical considerations to evaluate its implications for human biology, human evolution and our natural environment. In this Commentary, we consider human enhancement, and in particular, we explore genetic enhancement in an evolutionary context. In summarizing key open questions, we highlight the importance of acknowledging multiple effects (pleiotropy) and complex epigenetic interactions among genotype, phenotype and ecology, and the need to consider the unit of impact not only to the human body but also to human populations and their natural environment (systems biology). We also propose that a practicable distinction between therapy and enhancement may need to be drawn and effectively implemented in future regulations. Overall, we suggest that it is essential for ethical, philosophical and policy discussions on human enhancement to consider the empirical evidence provided by evolutionary biology, developmental biology and other disciplines.
Lay Summary: This Commentary explores genetic enhancement in an evolutionary context. We highlight the multiple effects associated with germline heritable genetic intervention, the need to consider the unit of impact to human populations and their natural environment, and propose that a practicable distinction between therapy and enhancement is needed.
There are countless examples where technology has contributed to ameliorate the lives of people by improving their inherent or acquired capabilities. For example, over time, there have been biomedical interventions attempting to restore functions that are deficient, such as vision, hearing or mobility. If we consider human vision, substantial advances started from the time spectacles were developed (possibly in the 13th century), continuing in the last few years, with researchers implanting artificial retinas to give blind patients partial sight . Recently, scientists have also successfully linked the brain of a paralysed man to a computer chip, which helped restore partial movement of limbs previously non-responsive [4, 5]. In addition, synthetic blood substitutes have been created, which could be used in human patients in the future .
The progress being made by technology in a restorative and therapeutic context could in theory be applied in other contexts to treat non-pathological conditions. Many of the technologies and pharmaceutical products developed in a medical context to treat patients are already being used by humans to enhance some aspect of their bodies, for example drugs to boost brain power, nutritional supplements, brain stimulating technologies to control mood or growth hormones for children of short stature. Assistive technology for disabled people, reproductive medicine and pharmacology, beside their therapeutic and restorative use, have a greater potential for human enhancement than currently thought. There are also dual outcomes as some therapies can have effects that amount to an enhancement as for example, the artificial legs used by the South African sprinter Oscar Pistorius providing him with a competitive advantage.
This commentary will provide general ethical considerations on human enhancement, and within the several forms of so-called human biomedical enhancement, it will focus on genetic engineering, particularly on germline (heritable) genetic interventions and on the insights evolutionary biology can provide in rationalizing its likely impact. These insights are a subject often limited in discussions on genetic engineering and human enhancement in general, and its links to ethical, philosophical and policy discussions, in particular . The rapid advances in genetic technology make this debate very topical. Moreover, genes are thought to play a very substantial role in biological evolution and development of the human species, thus making this a topic requiring due consideration. With this commentary, we explore how concepts based in evolutionary biology could contribute to better assess the implications of human germline modifications, assuming they were widely employed. We conclude our brief analysis by summarizing key issues requiring resolution and potential approaches to progress them. Overall, the aim is to contribute to the debate on human genetic enhancement by looking not only at the future, as it is so often done, but also at our evolutionary past.
The noun enhancement comes from the verb enhance, meaning to increase or improve. The verb enhance can be traced back to the vulgar Latin inaltiare and late Latin inaltare (raise, exalt), from altare (make high) and altus (high), literally grown tall. For centuries human enhancement has populated our imagination outlined by stories ranging from the myths of supernormal strengths and eternal life to the superpowers illustrated by the 20th century comic books superheroes. The desire of overcoming normal human capacities and the transformation to an almost perfect form has been part of the history of civilization, extending from arts and religion to philosophy. The goal of improving the human condition and health has always been a driver for innovation and biomedical developments.
In the broadest sense, the process of human enhancement can be considered as an improvement of the limitations of a natural version of the human species with respect to a specific reference in time, and to different environments, which can vary depending on factors such as, for example, climate change. The limitations of the human condition can be physical and/or mental/cognitive (e.g. vision, strength or memory). This poses relevant questions of what a real or perceived human limitation is in the environment and times in which we are living and how it can be shifted over time considering social norms and cultural values of modern societies. Besides, the impact that overcoming these limitations will have on us humans, and the environment, should also be considered. For example, if we boost the immune system of specific people, this may contribute to the development/evolution of more resistant viruses and bacteria or/and lead to new viruses and bacteria to emerge. In environmental terms, enhancing the longevity of humans could contribute to a massive increase in global population, creating additional pressures on ecosystems already under human pressure.
Two decades ago, the practices of human enhancement have been described as biomedical interventions that are used to improve human form or functioning beyond what is necessary to restore or sustain health . The range of these practices has now increased with technological development, and they are any kind of genetic, biomedical, or pharmaceutical intervention aimed at improving human dispositions, capacities, or well-being, even if there is no pathology to be treated . Practices of human enhancement could be visualized as upgrading a system, where interventions take place for a better performance of the original system. This is far from being a hypothetical situation. The rapid progress within the fields of nanotechnology, biotechnology, information technology and cognitive science has brought back discussions about the evolutionary trajectory of the human species by the promise of new applications which could provide abilities beyond current ones [12, 13]. If such a possibility was consciously embraced and actively pursued, technology could be expected to have a revolutionary interference with human life, not just helping humans in achieving general health and capabilities commensurate with our current ones but helping to overcome human limitations far beyond of what is currently possible for human beings. The emergence of new technologies has provided a broader range of potential human interventions and the possibility of transitioning from external changes to our bodies (e.g. external prosthesis) to internal ones, especially when considering genetic manipulation, whose changes can be permanent and transmissible.
The advocates of a far-reaching human enhancement have been referred to as transhumanists. In their vision, so far, humans have largely worked to control and shape their exterior environments (niche construction) but with new technologies (e.g. biotechnology, information technology and nanotechnology) they will soon be able to control and fundamentally change their own bodies. Supporters of these technologies agree with the possibility of a more radical interference in human life by using technology to overcome human limitations , that could allow us to live longer, healthier and even happier lives . On the other side, and against this position, are the so-called bioconservatives, arguing for the conservation and protection of some kind of human essence, with the argument that it exists something intrinsically valuable in human life that should be preserved [18, 19].
There is an ongoing debate between transhumanists  and bioconservatives [18, 19, 23] on the ethical issues regarding the use of technologies in humans. The focus of this commentary is not centred on this debate, particularly because the discussion of these extreme, divergent positions is already very prominent in the public debate. In fact, it is interesting to notice that the moderate discourses around this topic are much less known. In a more moderate view, perhaps one of the crucial questions to consider, independently of the moral views on human enhancement, is whether human enhancement (especially if considering germline heritable genetic interventions) is a necessary development, and represents an appropriate use of time, funding and resources compared to other pressing societal issues. It is crucial to build space for these more moderate, and perhaps less polarized voices, allowing the consideration of other positions and visions beyond those being more strongly projected so far.
Ethical and societal discussions on what constitutes human enhancement will be fundamental to support the development of policy frameworks and regulations on new technological developments. When considering the ethical implications of human enhancement that technology will be available to offer now and in the future, it could be useful to group the different kinds of human enhancements in the phenotypic and genetic categories: (i) strictly phenotypic intervention (e.g. ranging from infrared vision spectacles to exoskeletons and bionic limbs); (ii) somatic, non-heritable genetic intervention (e.g. editing of muscle cells for stronger muscles) and (iii) germline, heritable genetic intervention (e.g. editing of the CC chemokine receptor type 5 (CCR5) gene in the Chinese baby twins, discussed later on). These categories of enhancement raise different considerations and concerns and currently present different levels of acceptance by our society. The degree of ethical, societal and environmental impacts is likely to be more limited for phenotypic interventions (i) but higher for genetic interventions (ii and iii), especially for the ones which are transmissible to future generations (iii).
The rapid advances in technology seen in the last decades, have raised the possibility of radical enhancement, defined by Nicholas Agar, as the improvement of human attributes and abilities to levels that greatly exceed what is currently possible for human beings . Genetic engineering offers the possibility of such an enhancement by providing humans a profound control over their own biology. Among other technologies, genetic engineering comprises genome editing (also called gene editing), a group of technologies with the ability to directly modify an organisms DNA through a targeted intervention in the genome (e.g. insertion, deletion or replacement of specific genetic material) . Genome editing is considered to achieve much greater precision than pre-existing forms of genetic engineering. It has been argued to be a revolutionary tool due to its efficiency, reducing cost and time. This technology is considered to have many applications for human health, in both preventing and tackling disease. Much of the ethical debate associated with this technology concerns the possible application of genome editing in the human germline, i.e. the genome that can be transmitted to following generations, be it from gametes, a fertilized egg or from first embryo divisions . There has been concern as well as enthusiasm on the potential of the technology to modify human germline genome to provide us with traits considered positive or useful (e.g. muscle strength, memory and intelligence) in the current and future environments.
To explore some of the possible implications of heritable interventions we will take as an example the editing (more specifically deletion using CRISPR genome editing technology) of several base pairs of the CCR5 gene. Such intervention was practised in 2018 in two non-identical twin girls born in China. Loss of function mutations of the CCR5 had been previously shown to provide resistance to HIV. Therefore, the gene deletion would be expected to protect the twin baby girls from risk of transmission of HIV which could have occurred from their father (HIV-positive). However, the father had the infection kept under control and the titre of HIV virus was undetectable, which means that risk of transmission of HIV infection to the babies was negligible .
From an ethical ground, based on current acceptable practices, this case has been widely criticized by the scientific community beside being considered by many a case of human enhancement intervention rather than therapy [29, 30]. One of the questions this example helps illustrate is that the ethical boundary between a therapy that corrects a disorder by restoring performance to a normal scope, and an intervention that enhances human ability outside the accepted normal scope, is not always easy to draw. For the sake of argument, it could be assumed that therapy involves attempts to restore a certain condition of health, normality or sanity of the natural condition of a specific individual. If we take this approach, the question is how health, normality and sanity, as well as natural per se, are defined, as the meaning of these concepts shift over time to accommodate social norms and cultural values of modern societies. It could be said that the difficulty of developing a conceptual distinction between therapy and enhancement has always been present. However, the potential significance of such distinction is only now, with the acceleration and impact of technological developments, becoming more evident.
Beyond ethical questions, a major problem of this intervention is that we do not (yet?) know exactly the totality of the effects that the artificial mutation of the CCR5 may have, at both the genetic and phenotypic levels. This is because we now know that, contrary to the idea of one gene-one trait accepted some decades ago, a geneor its absencecan affect numerous traits, many of them being apparently unrelated (a phenomenon also known as pleiotropy). That is, due to constrained developmental interactions, mechanisms and genetic networks, a change in a single gene can result in a cascade of multiple effects . In the case of CCR5, we currently know that the mutation offers protection against HIV infection, and also seems to increase the risk of severe or fatal reactions to some infectious diseases, such as the influenza virus . It has also been observed that among people with multiple sclerosis, the ones with CCR5 mutation are twice as likely to die early than are people without the mutation . Some studies have also shown that defective CCR5 can have a positive effect in cognition to enhance learning and memory in mice . However, its not clear if this effect would be translated into humans. The example serves to illustrate that, even if human enhancement with gene editing methods was considered ethically sound, assessing the totality of its implications on solid grounds may be difficult to achieve.
Beyond providing the opportunity of enhancing human capabilities in specific individuals, intervening in the germline is likely to have an impact on the evolutionary processes of the human species raising questions on the scale and type of impacts. In fact, the use of large-scale genetic engineering might exponentially increase the force of niche construction in human evolution, and therefore raise ethical and practical questions never faced by our species before. It has been argued that natural selection is a mechanism of lesser importance in the case of current human evolution, as compared to other organisms, because of advances in medicine and healthcare . According to such a view, among many others advances, natural selection has been conditioned by our niche-construction ability to improve healthcare and access to clean water and food, thus changing the landscape of pressures that humans have been facing for survival. An underlying assumption or position of the current debate is that, within our human species, the force of natural selection became minimized and that we are somehow at the end-point of our evolution . If this premise holds true, one could argue that evolution is no longer a force in human history and hence that any human enhancement would not be substituting itself to human evolution as a key driver for future changes.
However, it is useful to remember that, as defined by Darwin in his book On the Origin of the Species, natural selection is a process in which organisms that happen to be better adapted to a certain environment tend to have higher survival and/or reproductive rates than other organisms . When comparing human evolution to human genetic enhancement, an acceptable position could be to consider ethically sound those interventions that could be replicated naturally by evolution, as in the case of the CCR5 gene. Even if this approach was taken, however, it is important to bear in mind that human evolution acts on human traits sometimes increasing and sometimes decreasing our biological fitness, in a constant evolutionary trade-off and in a contingent and/or neutralin the sense of not progressiveprocess. In other worlds, differently from genetic human enhancement, natural selection does not aim at improving human traits . Human evolution and the so-called genetic human enhancement would seem therefore to involve different underlying processes, raising several questions regarding the implications and risks of the latter.
But using genetic engineering to treat humans has been proposed far beyond the therapeutic case or to introduce genetic modifications known to already occur in nature. In particular, when looking into the views expressed on the balance between human evolution and genetic engineering, some argue that it may be appropriate to use genetic interventions to go beyond what natural selection has contributed to our species when it comes to eradicate vulnerabilities . Furthermore, when considering the environmental, ecological and social issues of contemporary times, some suggest that genetic technologies could be crucial tools to contribute to human survival and well-being . The possible need to engineer human traits to ensure our survival could include the ability to allow our species to adapt rapidly to the rate of environmental change caused by human activity, for which Darwinian evolution may be too slow . Or, for instance, to support long-distance space travel by engineering resistance to radiation and osteoporosis, along with other conditions which would be highly advantageous in space .
When considering the ethical and societal merits of these propositions, it is useful to consider how proto-forms of enhancement has been approached by past human societies. In particular, it can be argued that humans have already employedas part of our domestication/selective breeding of other animalstechniques of indirect manipulation of genomes on a relatively large scale over many millennia, albeit not on humans. The large-scale selective breeding of plants and animals over prehistoric and historic periods could be claimed to have already shaped some of our natural environment. Selective breeding has been used to obtain specific characteristics considered useful at a given time in plants and animals. Therefore, their evolutionary processes have been altered with the aim to produce lineages with advantageous traits, which contributed to the evolution of different domesticated species. However, differently from genetic engineering, domestication possesses inherent limitations in its ability to produce major transformations in the created lineages, in contrast with the many open possibilities provided by genetic engineering.
When considering the impact of genetic engineering on human evolution, one of questions to be considered concerns the effects, if any, that genetic technology could have on the genetic pool of the human population and any implication on its resilience to unforeseen circumstances. This underlines a relevant question associated with the difference between health and biological fitness. For example, a certain group of animals can be more healthyas domesticated dogsbut be less biologically fit according to Darwins definition. Specifically, if such group of animals are less genetically diverse than their ancestors, they could be less adaptable to environmental changes. Assuming that, the human germline modification is undertaken at a global scale, this could be expected to have an effect, on the distribution of genetically heritable traits on the human population over time. Considering that gene and trait distributions have been changing under the processes of evolution for billions of years, the impact on evolution will need to be assessed by analysing which genetic alterations have been eventually associated with specific changes within the recent evolutionary history of humans. On this front, a key study has analysed the implications of genetic engineering on the evolutionary biology of human populations, including the possibility of reducing human genetic diversity, for instance creating a biological monoculture . The study argued that genetic engineering will have an insignificant impact on human diversity, while it would likely safeguard the capacity of human populations to deal with disease and new environmental challenges and therefore, ensure the health and longevity of our species . If the findings of this study were considered consistent with other knowledge and encompassing, the impact of human genetic enhancements on the human genetic pool and associated impacts could be considered secondary aspects. However, data available from studies on domestication strongly suggests that domestication of both animals and plans might lead to not only decreased genetic diversity per se, but even affect patterns of variation in gene expression throughout the genome and generally decreased gene expression diversity across species . Given that, according to recent studies within the field of biological anthropology recent human evolution has been in fact a process of self-domestication , one could argue that studies on domestication could contribute to understanding the impacts of genetic engineering.
Beyond such considerations, it is useful to reflect on the fact that human genetic enhancement could occur on different geographical scales, regardless of the specific environment and geological periods in which humans are living and much more rapidly than in the case of evolution, in which changes are very slow. If this was to occur routinely and on a large scale, the implications of the resulting radical and abrupt changes may be difficult to predict and its impacts difficult to manage. This is currently highlighted by results of epigenetics studies, and also of the microbiome and of the effects of pollutants in the environment and their cumulative effect on the development of human and non-human organisms alike. Increasingly new evidence indicates a greater interdependence between humans and their environments (including other microorganisms), indicating that modifying the environment can have direct and unpredictable consequences on humans as well. This highlight the need of a systems level approach. An approach in which the bounded body of the individual human as a basic unit of biological or social action would need to be questioned in favour of a more encompassing and holistic unit. In fact, within biology, there is a new field, Systems Biology, which stresses the need to understand the role that pleiotropy, and thus networks at multiple levelse.g. genetic, cellular, among individuals and among different taxaplay within biological systems and their evolution . Currently, much still needs to be understood about gene function, its role in human biological systems and the interaction between genes and external factors such as environment, diet and so on. In the future if we do choose to genetically enhance human traits to levels unlikely to be achieved by human evolution, it would be crucial to consider if and how our understanding of human evolution enable us to better understand the implications of genetic interventions.
New forms of human enhancement are increasingly coming to play due to technological development. If phenotypic and somatic interventions for human enhancement pose already significant ethical and societal challenges, germline heritable genetic intervention, require much broader and complex considerations at the level of the individual, society and human species as a whole. Germline interventions associated with modern technologies are capable of much more rapid, large-scale impacts and seem capable of radically altering the balance of humans with the environment. We know now that beside the role genes play on biological evolution and development, genetic interventions can induce multiple effects (pleiotropy) and complex epigenetics interactions among genotype, phenotype and ecology of a certain environment. As a result of the rapidity and scale with which such impact could be realized, it is essential for ethical and societal debates, as well as underlying scientific studies, to consider the unit of impact not only to the human body but also to human populations and their natural environment (systems biology). An important practicable distinction between therapy and enhancement may need to be drawn and effectively implemented in future regulations, although a distinct line between the two may be difficult to draw.
In the future if we do choose to genetically enhance human traits to levels unlikely to be achieved by human evolution, it would be crucial to consider if and how our understanding of humans and other organisms, including domesticated ones, enable us to better understand the implications of genetic interventions. In particular, effective regulation of genetic engineering may need to be based on a deep knowledge of the exact links between phenotype and genotype, as well the interaction of the human species with the environment and vice versa.
For a broader and consistent debate, it will be essential for technological, philosophical, ethical and policy discussions on human enhancement to consider the empirical evidence provided by evolutionary biology, developmental biology and other disciplines.
This work was supported by Fundao para a Cincia e a Tecnologia (FCT) of Portugal [CFCUL/FIL/00678/2019 to M.A.].
Conflict of interest: None declared.
Post-implantation impedance spectroscopy of subretinal micro-electrode arrays, OCT imaging and numerical simulation: towards a more precise neuroprosthesis monitoring tool
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Human enhancement: Genetic engineering and evolution
Posted: at 2:13 am
Genetic engineering is a new and controversial process. With its medicinal, therapeutic, and agricultural applications, many scientists view it as a scientific beacon leading to a new era of discovery and solutions. However, others view the novelty of genetic engineering as one of its downfallsthere are so many unknown aspects of this new branch of science that it is not possible to know that it is safe.
For students and professionals interested in sustainability or resource management, this tension is a pertinent one to explore. Read on for some of the most important pros and cons of genetic engineering for the field of environmental science and sustainability.
According to the National Academy of sciences, genetic engineering could help make crops more resistant to the effects of climate change by introducing traits to plants that give them a wider range of temperature tolerance and make them more likely to survive drought conditions. This development holds great potential for those who study and build sustainable food systems and agricultural initiatives, as well as those developing policy to manage the effects of less ecological options.
One of the most recent advances in genetic engineering is the development of CRISPR gene editing technology. Derived from the anti-viral defense mechanisms of bacteria, CRISPR technology allows gene modification to be conducted more efficiently than ever before due to its precise gene targeting and cost-effectiveness. This can boost speed, helping to resolve the bottleneck problem facing many geneticiststhe processing of genetic data and experiments has generally been very time consuming, meaning many important experiments and scientific answers have been backlogged due to issues of efficiency rather than possibility. The impact of this technological acceleration is relevant to those pursuing an Environmental Science Degree online, as the potential ecological effects of genetic engineering multiply with increased gene editing capacity.
The effects of genetic engineering go beyond the lab: genetic transference and mutation take place within and between organisms in the natural environment. A recent bioethical study from the University of Chile suggests that strict regulatory norms be established in order to control the potential effects of the intergenerational passing of gene mutations between organisms, as these can have unpredictable environmental impacts. These concerns must be carefully considered by sustainability students. For example, students pursuing an M.S. in Professional Science at Unity College may address issues like this when studying Conservation Ecology, weaving together social sciences and ecological principles to develop system-level solutions to complex ecological problems.
Finally, some environmental science and sustainability experts argue that genetic engineering must be conducted with restraint due to technological limitations. For instance, although CRISPR technology boasts incredible efficiency, it also results in a significant (though decreasing) number of off-target effectsthat is, of accidental gene edits. Sustainability students and professionals must maintain a realistic understanding of the actual precision of genetic engineering technology so as to best anticipate its real-world effects.
Do you want to contribute to the responsible management of genetic engineering?
Contact Unity College to find out more about our online Masters Degree in Environmental Science!
Posted: at 2:13 am
In 1967, an up-and-coming scientist parted ways with his mentor. They had been working together for over ten years. Nobel Laureate Arthur Kornberg urged the more junior man to stay: You have a gift for doing enzyme research. The only true path to knowledge is E. coli.
Paul Berg thought otherwise. After spending a year learning mammalian cell culture at the Salk Institute, Berg returned to Stanford. Working with the renowned Kornberg refrigerator, which was stocked with enzymes essential to the projects success, Berg successfully spliced DNA from the bacterial virus lambda together with DNA from the mammalian virus SV40.
Public fear of the technology kept Berg from introducing the plasmid to an organism, but Recombinant DNA had nonetheless arrived.
It was not long until another team of scientists took over where Berg had left off. In 1973, Herbert Boyer and Stanley Cohen created a new type of recombinant DNA, an E.coli plasmid in which resistance to the antibiotic tetracycline had been added. This time they transformed E.coli, adding this new DNA to the organism. The experiment was a success: transformed E.coli demonstrated resistance to tetracycline. The scientists wondered what else was possible.
They added genes from a toad, to find out whether genes from higher order animals would also transfer. The newly transformed E.coli and their predecessors began producing ribosomal RNA. Many tiny E.coli could easily produce large amount of product.
Once scientists realized that the common workhorse of biotechnology could read and implement mammalian genetic instructions they began to think of medical applications. Might these tiny bacteria be capable of producing much-needed biological components of the human variety?
By 1979, E.coli was producing human insulin. It was only the beginning. In the 1980s, scientists began genetically engineering mice. These transgenes, or knockout mice, were often created to mirror a human illness and are still heavily used in scientific research today. The tools of biotechnology are now widespread in research, agriculture, and medicine.
Though the tools of genetic engineering impact our lives in innumerous ways, the sexier science involves direct human application. Fixing genetic abnormalities in utero, engineering babies with artificially heightened intelligence, and making human clones are some of the mad-scientist concepts the come to mind. They arent mad because theyre necessarily beyond our capabilities, but because they bring up ethical questions. Our capabilities in these areas are further along than you might think.
For most of human history babies were born with very little knowledge of their genetics beforehand. In the advent of In Vitro Fertilization (IVF), Preimplantation Genetic Diagnosis (PGD) is offered to ensure an embryo is healthy before implantation. As a bonus, parents who have eggs of both genders can choose which to implant. The Fertility Institutes Clinic in Los Angeles will even test for eye color.
Technically, however, this is selection. Engineering is new.
In 2016, a group of international scientists used CRISPR, a new technology that allows for more effective and efficient gene editing, to edit a human embryo. Their edit corrected a gene that causes heart disease. Since then, other scientists have also successfully edited embryo genes. Though none of these embryos have been implanted, they theoretically could have been.
Scientists are working to make the process more efficient and reliable, but once the technology is established there is little difference between correcting genetic errors and enhancing genes. This potential jump will depend on gene identification and public will.
In 1996, we cloned a sheep. Dolly was a first, but she would not be the last. The next year, 23 mouse clones confirmed reproducibility. Some were even clones of clones. Today, for a price, you can clone a beloved pet. Since our first attempts at cloning, which were inefficient and led to very low levels of success, scientist have come a long way.
For a non-reproductive cell to create new life, genes that had been turned off during differentiation of the cell have to be turned back on through a process called reprogramming. Cells are hardy little beasts and they do the best they can, but the task is monumental.
Fast forward to the work of researcher Yi Zhang at Howard Hughes Medical Institute. Zhang was determined to increase cloning efficiency. Like most cell and molecular biology, processes depend on the various factors involved. Working diligently to figure out which factors can unlock the reprogramming potential in non-reproductive cells, Zhang increased the success of cloning from about 1 percent to 10 percent.
Then he turned to humans.
In 2015, Zhang inserted skin cells from one set of people into eggs donated by a small group of volunteers. When the extra reprogramming factors were added, a quarter of the eggs developed into embryos.
Zhang isnt trying to build a human army of clones, or even clone a great thinker for the betterment of our race. Both would be illegal. He is instead interested in therapeutic cloning. Stem cells taken from these nascent embryos would be a perfect match to the donor. Organs or tissue grown could theoretically be transplanted with little risk of rejection, saving a patients life.
In many ways, genetic engineering is still in its infancy. The future depends largely on what the public will accept. Today it seems a cloned dog and genetic modifications to prevent or mitigate disease are acceptable, while human cloning and true enhancement are not. It will be interesting to see what is considered acceptable twenty years from now.
See the original post here:
The History of Genetic Engineering - Genetics Digest
Posted: at 2:13 am
The following page shows the career & education requirements, salary and job outlook for Genetic Engineering around the country.
A genetic engineer is a highly educated expert who uses a range of molecular technologies and tools in order to take fragments of DNA and rearrange them to come up with a breed that has certain advantages. The goal is to take out or add to the genetic makeup of a specific organism, thereby improving it. These professionals may also used DNA codes and transfer them between species. This is done in an effort to make sure that organisms become stronger, and to enable them to survive in different environments. For instance, they may work with plants to ensure that they continue to bear fruit even if a drought were to occur. Alternatively, they could change the DNA of certain bacteria that produces a certain compound that can be used as a drug to enhance the drugs capabilities.
It is very rare to find a genetic engineer anywhere other than a laboratory. Most of these professionals work in labs and will have infrequent office work, something they will most often complete within their labs. Office work includes doing things like writing papers and drafting reports, or even coming up with publications. Usually, they work for private companies, including research organizations and pharmaceutical companies. They may also be found within universities or hospitals, as well as, within government organizations. Usually, genetic engineers will have the opportunity to specialize their skills as well.
Becoming a genetic engineer requires a lot of education. While legally the minimum is to complete a bachelors degree in a field such as molecular genetics, molecular biology, biophysics, or biochemistry, it is very rare for this to be sufficient to land a good job. Instead, employers look for candidates with masters degrees, or even doctorate degrees, emphasizing molecular biology or molecular genetics. While an undergraduate degree is good as an entry point, completing a Ph.D. is generally the best option of all.
There are no legal requirements in terms of licensing and certification for genetic engineers. However, to demonstrate that you are committed to maintaining the standard of your profession, you may want to consider certification through the Biomedical Engineering Society, which is a nationally recognized organizations that provides members with events, resources, networking opportunities, education, training, and more.
According to Indeed.com, the following career/job titles with salary figures are most closely related to Genetic Engineering.
According to the U.S. Bureau of Labor Statistics (BLS), all biomedical engineers or genetic engineers, earned $86,220 per year in May 2015.
The BLS has reported that biomedical engineers can expect to see a 23% growth in demand for years between 2014 and 2024, which is one of the fasted rates.
According to Indeed.com, the average national salary of jobs for Genetic Engineering was $69,000.00 with a high confidence ranking based on over 250 sources. Average Genetic Engineering salaries for job postings nationwide are 19% higher than average salaries for all job postings nationwide.
The following lists Genetic Engineering salaries in each state around the country. The figures are based on the total number of job postings by employers through Indeed.com. For example, DC had the largest quoted salary of $87,000 while Hawaii had the smallest quoted salary of $43,000.
Poseida Therapeutics Announces Research Collaboration with Takeda for Novel Non-Viral In Vivo Gene Therapies – Yahoo Finance
Posted: at 2:13 am
Collaboration to leverage Poseida's non-viral piggyBac DNA Modification System, Cas-CLOVER Site-Specific Gene Editing System, biodegradable DNA and RNA nanoparticle delivery technology and other proprietary genetic engineering platforms
Collaboration to initially include up to six liver- and hematopoietic stem cell (HSC)- directed indications with an option to add two additional programs
In addition to an upfront payment, Poseida is eligible to receive preclinical, development and commercial milestone payments plus tiered royalties into the double digits
Poseida to host conference call today at 8:00am ET
SAN DIEGO, Oct. 12, 2021 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, today announced that it has entered into a research collaboration and exclusive license agreement with Takeda Pharmaceutical Company Limited ("Takeda") to utilize Poseida's piggyBac, Cas-CLOVER, biodegradable DNA and RNA nanoparticle delivery technology and other proprietary genetic engineering platforms for the research and development of up to eight gene therapies. The collaboration will focus on developing non-viral in vivo gene therapy programs, including Poseida's Hemophilia A program.
Poseida Therapeutics (PRNewsfoto/Poseida Therapeutics, Inc.)
"We are excited to partner with Takeda, a global biopharmaceutical leader whose commitment to the development of novel therapies for rare diseases complements our innovative platform technologies and robust gene therapy pipeline," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida. "Our technologies offer highly efficient gene delivery, fully integrated non-viral genome insertion and ultra-precise site-specific gene editing. Together with Takeda, we look forward to developing potential cures for a number of genetic diseases with high unmet need."
Under the terms of the agreement, the parties will collaborate to initially develop up to six in vivo gene therapy programs utilizing Poseida's novel technology platforms including piggyBac, Cas-CLOVER and biodegradable nanoparticle technology, as well as certain emerging technologies. Takeda also has an option to add two additional programs to the collaboration and is obligated to provide funding for all collaboration program R&D costs.
Poseida will receive an upfront payment of $45 million and preclinical milestones that together could potentially exceed $125 million in the aggregate, if milestones for six programs are achieved. Poseida is also eligible to receive future clinical development, regulatory, and commercial milestone payments with a total potential value over the course of the partnership of up to $2.7 billion if milestones for all six programs are achieved, and up to $3.6 billion if the milestones related to the two optional programs are also achieved. Poseida will lead research activities up to candidate selection, after which Takeda will assume responsibility for further development and commercialization.
"Poseida's differentiated platform technologies show great promise in developing non-viral in vivo gene therapies using their novel genetic engineering and delivery technologies that complement our existing collaborations," said Takeda Rare Diseases Drug Discovery Unit Head, Madhu Natarajan. "This partnership reinforces Takeda's commitment to investing in next-generation gene therapy approaches that have the potential to deliver functional cures to patients with rare genetic and hematologic diseases. We look forward to partnering with Poseida where we can apply our broad development capabilities to help progress several early stage preclinical programs."
Poseida Therapeutics Conference Call and Webcast Information
Poseida's management team will host a conference call and webcast at 8:00am ET today, October 12, 2021 to discuss the collaboration. The dial-in numbers for domestic and international callers are (866) 939-3921 and (678) 302-3550, respectively. The conference ID number for the call is 50242119.
Participants may access the live webcast on the Investors & Media Section of the Poseida website, http://www.poseida.com. An archived replay of the webcast will be available for approximately 30 days following the event.
About Poseida Therapeutics, Inc.
Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary genetic engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBac DNA Modification System, Cas-CLOVER Site-Specific Gene Editing System and biodegradable nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics. To learn more, visit http://www.poseida.com and connect with us on Twitter and LinkedIn.
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding potential payments and activities under the collaboration agreement with Takeda, the potential benefits of Poseida's technology platforms and product candidates and Poseida's plans and strategy with respect to developing its technologies and product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon Poseida's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the fact that the collaboration agreement with Takeda may be terminated early, the fact that Poseida will have limited control over the efforts and resources that Takeda devotes to advancing development programs under the collaboration agreement, risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry, the fact that future preclinical and clinical results could be inconsistent with results observed to date and the other risks described in Poseida's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Poseida undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
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Posted: at 2:13 am
Genetic engineering : the deliberate modification of the characteristics of an organism by manipulating its genetic material.
Genetic engineering is something that has been used in science fiction to scare people for decades. We typically end up with either tyrants who use their super intelligence and super strength to recreate the world in a manner or their choosing, or with monsters. (Or both.)
Genetic engineering has roots in the eugenics movement of the early 1900s. During this time, individuals deemed unfit were sterilized (For more, read about Buck v. Bell). Also, the American Eugenics Movement provided impetus for the German "Law for the Prevention of Progeny with Hereditary Diseases" in the 1930s.
But with the advent of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) the science fiction aspects of genetic engineering are quickly becoming a reality, and as a society we need to decide now the ethics and morality of genetic engineering in humans.
But first, what is CRIPSR? CRISPR works like a pair of molecular scissors, cutting DNA strands and allowing for specific gene editing. (For more on DNA, see this article by the NIH. For information about CRISPR listen to this excellent Make Me Smart podcast: CRISPR for Beginners)
We can get into the weeds with the technical details, but for the purposes of considering the ethics, let's consider broadly the two primary types of genetic manipulation: human germline editing and somatic cell editing.
Somatic cell editing is what is used when doctors and researchers use genetic editing to attempt to cure disease like cancer. Genetic engineering is used in this case, to go in an fix and existing problem. These changes end with the individual.
Germline editing is when genetic changes are made that may be passed along to succeeding generations.
The ethics of these two types of procedures are distinct and different, yet overlapping.
Germline editing makes many people squeamish, because it affects changes that would be inherited and passed down. It could allow a government to create a race of super warriors with increased strength and stamina and decreased fear (See: Many many science fiction story lines). But it could also allow society to wipe out inherited diseases such as sickle cell anemia.
The ethical problem lies in the fact that allowing for the treatment of diseases opens the door for cosmetic or vanity use of germline editing, since if the technique itself is ethical, it should be allowed for all types of uses.
Most people see somatic cell editing, which is not inherited, as a good when it is used to cure a disease like cancer. The ethics are less clear if somatic cell editing is used to "improve" an individual by as increasing their intelligence or strength and speed..
Is it ethical for people who can afford to do so to "improve" their children? Will this create two separate classes of people: Those who can afford improvements and those who cannot? Will this widen the gulf between the rich and the poor?
Science fiction has spent years depicting these ethical morasses. From Star Trek: The Wrath of Khan to Jurassic Park to The Fly, there are depictions of genetic engineering gone wrong. But the reality is that these dilemmas are less likely to create monsters than they will be to create cases like Carrie Buck who was involuntarily sterilized in the 1920 or the character of Dr Julian Bashir on Star Trek: Deep Space Nine.
"I'm still your father, Jules, and I will not have you talk to me like that.""No. You used to be my father. Now you're my architect, a man who designed a better son, to replace the defective one he was given. Well, your design has a built-in flaw. It's illegal."-- Doctor Bashir, I Presume (ST:DS9)
If you are unfamiliar with Dr Bashir, he was genetically engineered as a child, and then spent his teenage years and adulthood hiding the fact, because what was done to him was illegal. His story poses the moral and ethical questions of whether he can remain a Starfleet officer because of this.
We also see Dr. Bashir working with adults whose childhood manipulations were not successful, in one case using surgery to help a young woman who was mute and withdrawn from her engineering--because not all changes would be successful, and there would be individuals who suffered negative consequences as a result.
First gene-edited babies may be at risk of early death
The reason I bring up the science fiction examples is that these procedures are complicated, and it's extremely difficult to contemplate ethics when you don't understand the basics of the science. When presented with these ethical dilemmas in story form, we can consider the possible results without having to fully understand the science underpinning these changes.
These changes are coming. There is no way to put the genie back in the bottle. Which is why I believe it's so important to contemplate the ethical issues that have arisen in the past and will arise in the future from genetic engineering.
Because there are, of course, no easy answers.
Genetic engineering could have tremendous benefits, with the possibility of wiping out deadly inherited diseases and cancers. It could also create monsters or widen the gap between the rich and the poor beyond recovery.
What we decide as yet unknown. The only known is that these changes are coming, like it or not, and we are better off facing them prepared with as much knowledge and thought as we can manage.
Make Me Smart: CRISPR for Beginners Live Science: What Is CRISPR?Why Treat Gene Editing Differently In Two Types Of Human Cells?Somatic Cell Genome EditingFirst gene-edited babies may be at risk of early deathStar Trek: Deep Space Nine - "Doctor Bashir, I Presume", "Statistical Probabilities", "Chrysalis"19 Best Genetic Engineering Science Fiction BooksBest Movies and TV Shows Featuring CRISPR and Genetic Engineering
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The Ethics of CRISPR and Genetic Engineering | Osher Lifelong ...
Posted: at 2:13 am
In the modern world, the lack of food and the search for solutions to this problem is one of the main tasks of developing science and engineering. The leading causes of this problem are planetary overpopulation, rapid urbanization, and of course, climate change. One of the solutions to this problem is GMOs organisms created with the help of genetic engineering primarily to increase crop yield.
Changes in the quantity and quality of genes and mutations are found everywhere in nature. Mutations can be both useful and harmful. Beneficial mutations are at the heart of the evolution of any species. Genetic engineering allows for altering the genetic code of animals and plants, endowing them with new properties or improving the existing ones. But how does that help in tackling climate change? Lets try to find out, starting with defining what GMOs are.
GMO (genetically modified organism) is an organism in which the set of genes has been artificially altered. Most GMOs are different types of plants that are used in agriculture and the food industry. When food is made from such organisms, it is called genetically modified food.
In traditional breeding, new plant varieties are developed by crossing plants with the desired traits and selecting the best results. In the genetic modification of organisms, the genes of non-crossed species are used. Such changes in genes cannot occur as a result of reproduction or natural recombination of genes.
The wars of supporters and opponents of genetically modified organisms have been going on for decades. Some are sure that GMO foods cause tumors and affect the human genetic code, while others believe that modified food is no different from ordinary food and even surpasses it in quality. The problem is complex, but research in recent years has clarified a lot.
So far, there are only two likely risks associated with the use of GMOs:
With the help of genetic engineering methods, scientists create high-yielding crops and resistant to different conditions. Such crops can be grown on dry, saline, and degrading soils, which is essential due to high rates of environmental pollution.
With the help of high-yielding GM crops, people can solve the problem of hunger, especially in developing countries. In addition, the population of the Earth is constantly growing, so the issue of hunger may affect other countries as well in the future.
Besides, genetically modified crops can be less harmful to the environment during cultivation. For example, scientists have successfully tested higher-yielding genetically modified rice, significantly reducing methane emissions, one of the leading greenhouse gases that cause climate change.
In fact, GMO plants can also be watered and cultivated less frequently. This will save water and reduce the greenhouse effect by reducing the thermal radiation of arable land. In addition, fewer agricultural machinery in the fields will help control carbon dioxide emissions.
Breeding plants that bear fruit more often require minimal cultivation and even absorb CO2. This would help to significantly reduce the greenhouse effect and improve the environmental situation around the world.
Overall, despite all the difficulties with the development and safety testing, scientists are confident that humanity cannot do without transgenic plants and products in the future. GMOs will help humanity prevent hunger or mass crop failure and minimize the harm agriculture poses to the environment.
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For Climate Change and Agriculture, Are GMOs the Future? - Amico Hoops
Posted: at 2:13 am
image:Most up-to-date information and perspectives on exciting new research findings and discoveries emanating from interplanetary exploration and terrestrial field and laboratory research programs. view more
Credit: Mary Ann Liebert, Inc., publishers
The potential habitability of the Venusian cloud layer based on hints of past water on Venus is detailed in a special collection of articles in the peer-reviewed journal Astrobiology. Click here to read the articles now.
Sanjay Limaye (Guest Editor of this special issue), from the University of Wisconsin-Madison, and Lev Zelenyi and Ludmilla Zasova, from the Space Research Institute, Russian Academy of Sciences, introduce the Venus Collection, a series of papers from the first workshop on habitability of the cloud layer. The workshop was held in Moscow and organized by the Russian Space Agency, Space Research Institute, and NASA. The workshop can be traced to a paper, Was Venus the First Habitable Planet, by Michael Way (NASA/Goddard Institute for Space Studies) and colleagues in 2016, which provoked a fresh look at the possibility that life existed on the planet in the past if it indeed had liquid water for a long time, including the possibility that microorganisms could be responsible for absorption of sunlight, and contrasts that of Limaye and colleagues described in a paper published in Astrobiology in 2018.
This special issue on Venus is coming out at just the right time. NASA just announced two new missions to Venus and ESA announced one as well. Venus is going to be in the scientific focus for some years to come, says Christopher McKay, PhD, Deputy Editor of Astrobiology, from NASA Ames Research Center.
In this issue, Sanjay Limaye and colleagues coauthored Venus, an Astrobiology Target. They present a case for the exploration of Venus as an astrobiology target, based on the likelihood that liquid water existed on the surface in the past, leading to the potential for the origin and evolution of life. They propose investigations into the potential for habitable zones within Venus present-day clouds and Venus-like exo atmospheres.
The detection of phosphine (PH3) in Venus atmosphere has contributed to the hypothesis that there may be life in the Venusian clouds. Matthew Pasek, from University of South Florida, and coauthors, present two related abiotic routes for phosphine generation within the atmosphere of Venus. Corrosion of large impactors as they ablate near Venus cloud layer, and the presence of reduced phosphorous compounds in the subcloud layer could result in production of phosphine and could explain the phosphine detected in Venus atmosphere. A paper led by William Bains of MIT concludes that phosphine cannot be produced in the Venus atmosphere by conventional processes.
A paper by Sara Seager (Massachusetts Institute of Technology) and colleagues provides a possible life cycle for the putative microorganisms. Other papers explore other aspects of habitability of the Venus cloud layer, including the potential for phototrophy by Rakesh Mogul (California Polytechnic Institute).
Finding answers to this question is like putting together a jigsaw puzzle except that we do not show what the picture of the pieces put together should look like. The data and inferences from decades of observations do not seem to fit together nicely and many pieces are missing. The ten papers in this collection from the first workshop on the Venus cloud layer habitability represent a beginning of solving the puzzle. The pace is quickening, with more than twice as many papers to be presented at the second workshop on cloud habitability of Venus later this year.
It is important to find out if Venus was ever habitable and whether its thick, global cloud cover can harbor life today, not just for understanding the origins of life, but also to have any confidence regarding what we infer about habitable exoplanets. The ten papers in the collection are hopefully the first steps in this quest, says Sanjay Limaye, PhD, Guest Editor, from the University of Wisconsin-Madison.
About the JournalAstrobiology, led by Editor-in-ChiefSherry L. Cady, Ph.D., at the Pacific Northwest National Laboratorys Marine and Coastal ResearchLaboratory(MCRL), and a prominent international editorial board comprised of esteemed scientists in the field, is the authoritative peer-reviewed journal for the most up-to-date information and perspectives on exciting new research findings and discoveries emanating from interplanetary exploration and terrestrial field and laboratory research programs. The Journal is published monthly online with Open Access options and in print. Complete tables of content and a sample issue may be viewed on theAstrobiologywebsite.The 2020 Journal Impact Factor is 4.335.
About the PublisherMary Ann Liebert, Inc., publishersis known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industrys most widely read publication worldwide. A complete list of the firms more than 100 journals, books, and newsmagazines is available at theMary Ann Liebert, Inc., publishers website.
Venus' Spectral Signatures and the Potential for Life in the Clouds
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Searching for habitable zones within Venus' clouds - EurekAlert
Posted: at 2:13 am
WASHINGTON Every now and then, a sliver of sanity seeps through the barricade of national lunacy.
Last week, a handful of bipartisan lawmakers introduced two bills aimed at ending one of our nations most-barbaric practices mandatory animal testing of new pharmaceuticals destined for human trials.
Its been a while since Ive performed a midair, double-heeled click, but I managed a reasonable facsimile upon hearing this news. The Senates FDA Modernization Act and the Houses H.R. 2565 set the stage for a groundbreaking move to end animal suffering while advancing timelier, more efficient drug development.
In part, the measures result from lessons learned during development of the coronavirus vaccine: We dont need to wait so long to develop human therapies if we bypass some outdated laws' archaic demands, including a 1930s-era law requiring animal testing before human trials.
When the pandemic demanded swift action on a vaccine, the Food and Drug Administration worked with government officials and pharmaceutical companies to create lifesaving drugs in record time. This happened because Moderna and Pfizer were allowed to run animal testing and early trials on humans at the same time, rather than completing separate animal trials first.
The best reason to stop using animals in drug tests is the fact that animals dont respond to drugs the same way people do. (If they did, we might as well all go to veterinarians for our shots.)
Although the use of animals in science and medicine has benefited human beings, theres significant evidence that human subjects have been harmed in the clinical testing of drugs that were deemed safe by animal studies, as Gail A. Van Norman wrote in the journal JACC: Basic to Translational Science.
Alarmingly, adverse drug reactions are the fourth leading cause of death in the United States after heart disease. It does not sound to me like using animals normally mice and monkeys is worth the price in cruelty we pay for our health.
Besides, other ways of conducting research are available and in use.
The first is a technique that performs a procedure in a controlled environment outside of a living organism, which sounds a lot better than the alternative. Such tests are being used and typically involve tests or experiments performed on computers or via computer simulation. This method also is being used in studies that predict how drugs interact with the body and with pathogens.
Nevertheless, drug companies and the scientific community likely will fight this initiative, just as they have in past years, if only because they dont want to change how they do business. Several important animal rights victories, including President Trumps ban on using dogs in experiments, has some firms and many scientists worried about the future of such research.
Cultural trends also seem to suggest that public opinion is shifting on animal research. A 2018 Pew Research Center study found that a slight majority of Americans (52%) oppose animal testing.
But it is not without exceptions: When asked about genetic engineering of animals, the numbers shift toward the survival of our species over others. Only 21% think that engineering aquarium fish to glow is an appropriate use of technology, for example, while 57% approve of using animals to grow organs and tissue for humans in need of a transplant.
Though there didnt seem to be any significant partisan alignments, there was evidence that support for animal testing rises with education. Americans with postgraduate degrees support animal experimentation to a greater degree because, theoretically, theyve likely had greater exposure to science. The less educated more often oppose animal experimentation.
Still, some in the scientific community are getting worried about the future of animal research. Ken Gordon, executive director of a Seattle biomedical research firm, has tracked U.S. attitudes toward animal research using 17 years of Gallup polls. Extrapolating, he predicts that the portion of the public that finds animal testing morally wrong will exceed the portion that finds it morally acceptable within the next two to three years.
When that happens, he said, funding will dry up, and our work will get a lot more difficult.
Thats probably an overstatement. Id like to think that science and humane research can coexist. Much of what we do in research today is because of how weve always done it ever since the 4th century B.C. when Aristotle was performing animal experiments to learn about anatomy.
Several millennia later is time enough to liberate our animal hostages along with our better angels and put technology to its highest and best uses. Besides, given what we know, it just makes sense.
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Kathleen | Opinion | heraldbulletin.com - The Herald Bulletin
Posted: at 2:13 am
Takeda is aggressively partnering with biotech companies this week. Seattle-based Immusoft announced it hadinked a research pact and license option deal with Takeda Pharmaceutical Company to create, develop and market cell therapies in rare inherited metabolic diseases with central nervous system (CNS) manifestations and complications. It will leverage Immusofts Immune System Programming (ISP) technology platform.
Takeda is paying Immusoft an undisclosed fee upfront and research funding support. Immusoft will be eligible for option fees and milestones of more than $900 million. Takeda picks up options to exclusively license the programs before the clinic. Immusoft will be eligible for tiered royalties on commercial products coming out of the partnership. Takeda will handle continued preclinical and clinical development and commercialization.
We are excited to enter this collaboration with Takeda, a recognized global leader in rare disease therapies, stated Sean Ainsworth, Immusofts chief executive officer. This advances our leadership position in B cells as biofactories for therapeutic protein delivery, a novel approach that Immusoft has pioneered. This partnership provides Immusoft with significant resources to further develop our Immune System Programming (ISP) technology platformand therapies in diseases for which patients have limited options.
Immusofts pipeline, primarily in the IND-enabling or discovery phases, has compounds for Hurler syndrome (MPS I and MPS II), ALS, Duchenne muscular dystrophy, Pompe, and Gaucher diseases.
The companys ISP platform modifies B cells and instructs them to deliver gene-encoded therapies. The partnership will focus on developing therapies that can be delivered across the blood-brain barrier. Immusoft is also working on new approaches to modify cell therapies to be more durable and redosable genetically.
We continue to build our internal capabilities as well as partner with innovative companies early on in the discovery process to advance our next-generation gene and cell therapy ambitions for rare genetic and hematologic diseases, said Madhu Natarajan, Takeda Rare Diseases Drug Discovery Unit head. Working together with Immusoft, we hope to validate their ISP technology for CNS delivery of innovative therapeutics for rare neurometabolic diseases.
Just yesterday, Takeda signed a research deal with San Diegos Poseida Therapeuticsworth up to $3.6 billion or more. Under the terms of that deal, the companies will collaborate to develop up to six in vivo gene therapy programs using Poseidas tech platforms, including piggyBac, Cas-CLOVER, and biodegradable nanoparticles. Takeda picked up an option to add two more programs to the deal andfund all collaboration program R&D costs.
Takeda is paying Poseida $45 million upfront and preclinical milestones that could exceed $125 millionif all six program milestones are hit. Throughout the partnership, Poseida will also be eligible for future milestonesfor up to $2.7 billion, which could reach $3.6 billion if all milestones are achieved and the two optional programs.
They will focus on non-viral in vivo gene therapies, including Poseidas Hemophilia A program.
Natarajan said, Poseidas differentiated platform technologies show great promise in developing non-viral in vivo gene therapies using their novel genetic engineering and delivery technologies that complement our existing collaborations. This partnership reinforces Takedas commitment to investing in next-generation gene therapy approaches that have the potential to deliver functional cures to patients with rare genetic and hematologic diseases.
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