Genetic Engineering additionally called genetic modification or genetic manipulation is the immediate control of a living being's genes using biotechnology. It is an arrangement of innovations used to change the hereditary forms of cells, including the exchange of qualities inside and across species limits to create enhanced or novel living beings.

Genetic Engineering has been connected in various fields including research, medicine, industrial biotechnology and agriculture. In research, GMOs are utilized to contemplate quality capacity and articulation through loss of function, gain of function, tracking and expression experiments. By thumping out genes responsible for specific conditions it is possible to create animal model organisms of human diseases. And in addition to producing hormones, immunizations and different drug genetic engineering can possibly fix hereditary diseases through quality treatment. Similar strategies that are utilized to create medications can likewise have mechanical applications, for example, producing enzymes for detergents, cheeses and different products.

The ascent of commercialised genetically modified crops has given a financial advantage to agriculturists in a wide range of nations, however, has additionally been the wellspring of a large portion of the debate encompassing the innovation. This has been available since its initial implementation, the primary field trials were destroyed by anti-GM activists. In spite of the fact that there is a logical accord that at presently accessible sustenance got from GM crops represents no more serious hazard to human wellbeing than regular nourishment, GM sustenance security is the main concern with critics.

Genetic engineering is the study of genes and the science of heredity. Genetic engineers or geneticists study living organisms ranging from human being to crops and even bacteria.

These professionals also conduct researches which is a major part of their work profile. The experiments are conducted to determine the origin and governing laws of a particular inherited trait. These traits include medical condition, diseases etc. The study is further used to seek our determinants responsible for the inherited trait.

Genetic engineers or Geneticists keep on finding ways to enhance their work profile depending on the place and organization they are working with. In manufacturing, these professionals will develop new pharmaceutical or agricultural products while in a medical setting, they advise patients on the diagnosed medical conditions that are inherited and also treat patients on the same.

Skill sets for Genetic engineers or Geneticists

Strong understanding of scientific methods and rules

complex problem solving and critical thinking

ability to use computer-aided design (CAD)

graphics or photo imaging

PERL, Python

word processing software programs

excellent mathematical, deductive and inductive reasoning, reading, writing, and oral comprehension skills

ability to use lasers spectrometers, light scattering equipment, binocular light compound microscopes, bench top centrifuges, or similar laboratory equipment

Typical responsibilities of a Genetic Engineering or Geneticist includes:

When a genetic engineer gains a year of experience, one of the regions they can indulge into is hereditary advising, which includes offering data, support and counsel on hereditary conditions to your patients.

An individual aspiring to pursue a professional degree in Genetic Engineering can begin the BTech course after his/her 10+2 Science with Physics, Chemistry, Maths and Biology.

Admission to BTech in Genetic Engineering is made through entrance tests conducted in-house by various universities or through the scores of national engineering entrance examination like JEE for IITs/NITs & CFTIs across the country.

Genetic Engineering professionals require a bachelors or masters degree in Genetic Engineering or Genetic Sciences for entry-level careers. In any case, a doctoral qualification is required for those looking for free research professions. Important fields of study in Genetic Engineering incorporate natural chemistry, biophysics or related fields.

Genetic Engineers require a solid comprehension of logical techniques and guidelines, and in addition complex critical thinking and basic reasoning aptitudes. Phenomenal scientific, deductive and inductive thinking aptitudes, and in addition perusing, composing, and oral cognizance abilities are additionally expected to work in this field.

A semester- wise breakup of the course is tabulated below

SEMESTER I

SEMESTER II

Mathematics 1

Mathematics 2

English

Material Science

Physics

Principles of Environmental Science

Chemistry

Biochemistry

Basic Engineering 1

Basic Engineering 2

-

Cell Biology

-

Value Education

SEMESTER III

SEMESTER IV

Enzyme Technology

Basic Molecular Techniques

Genetics & Cytogenetics

Molecular Biology

Immunology

Stoichiometry and Engineering Thermodynamics

Microbiology

Bio-press Principles

Mechanical Operations & heat Transfer

Biostatistics

German Language Phase 1/French Language Phase 1/Japanese Language Phase 1

German Language Phase 2/Japanese Language Phase 2/French Language Phase 2

-

SEMESTER V

SEMESTER VI

Advanced Molecular Techniques

Recombinant DNA Technology

Functional Genomics and Microarray Technology

Bioinformatics

Momentum Transfer

Chemical Reaction Engineering

Bioprocess Engineering

Gene Therapy

Biophysics

Biosensors and Biochips

Plant Tissue Culture and Transgenic Technology

-

Personality Development

-

SEMESTER VII

SEMESTER VIII

Bio-separation Technology

Project Work

Animal Cell Culture and Transgenic Technology

Bio-Safety, Bio-ethics, IPR & Patients

Nano-biotechnology in Healthcare

-

Stem Cell Biology

-

Aspirants who wish to join the engineering industry as a genetic engineer can apply for the following jobs profiles available:

JOB PROFILE

JOB DESCRIPTION

Genetic Engineer

They apply their knowledge ofengineering, biology, and biomechanical principles into the design, development, and evaluation of biological and health systems and products, such as artificial organs, prostheses, instrumentation, medical information systems, and health care and management.

Lecturer/Professor

They teach at undergraduate and graduate level in areas allocated and reviewed from time to time by the Head of Department.

Research Scientist

They are responsible for designing, undertaking and analyzing information from controlled laboratory-based investigations, experiments and trials.

Scientific/Medical Writer

The research, prepare and coordinate scientific publications. The medical writer is responsible for researching, writing and editing clinical/statistical reports and study protocols, and summarizing data from clinical studies.

Most of the engineering educational institutes shortlist candidates for admission Into BTech in Genetic Engineering course on the basis of engineering entrance exams. These entrance exams are either conducted at the national level like JEE or held in-house by various engineering institutes in the country.

Some of the popular engineering entrance examinations aspirants should consider appearing for admissions to UG and PG level Automobile engineering courses are:

Q. Which college is best for genetic engineering?

A. SRM University Chennai Tamil Nadu, Bharath University Chennai Tamil Nadu, Aryabhatta Knowledge University Patna Bihar, Jawaharlal Nehru Centre for Advanced Scientific Research Bangalore are some of the institutes offering genetic engineering

Q. Is Jee required for genetic engineering?

A. NITs and IITs across India does not offer genetic engineering. But there are 23 collages which take admission on the basis of JEE main

Q. What is the qualification for genetic engineering?

A. For admission to BTech Genetic Engineering course, the candidate is needed to have passed the Higher Secondary School Certificate (10+2) examination from a recognized Board of education with Biology, Physics and Chemistry as main subjects with a minimum aggregate score of 60%.

Q. Does IIT offer genetic engineering?

A. No, IIT directly does not offer genetic engineering. Candidates have to take Life Sciences in graduation or Biotechnology from any engineering college in India.

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Genetic Engineering - Courses, Subjects, Eligibility ...

Human genetic enhancement or human genetic engineering refers to human enhancement by means of a genetic modification. This could be done in order to cure diseases (gene therapy), prevent the possibility of getting a particular disease[1] (similarly to vaccines), to improve athlete performance in sporting events (gene doping), or to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence.These genetic enhancements may or may not be done in such a way that the change is heritable (which has raised concerns within the scientific community).[2]

Genetic modification in order to cure genetic diseases is referred to as gene therapy. Many such gene therapies are available, made it through all phases of clinical research and are approved by the FDA. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.[3] As of 2017, Spark Therapeutics' Luxturna (RPE65 mutation-induced blindness) and Novartis' Kymriah (Chimeric antigen receptor T cell therapy) are the FDA's first approved gene therapies to enter the market. Since that time, drugs such as Novartis' Zolgensma and Alnylam's Patisiran have also received FDA approval, in addition to other companies' gene therapy drugs. Most of these approaches utilize adeno-associated viruses (AAVs) and lentiviruses for performing gene insertions, in vivo and ex vivo, respectively. ASO / siRNA approaches such as those conducted by Alnylam and Ionis Pharmaceuticals require non-viral delivery systems, and utilize alternative mechanisms for trafficking to liver cells by way of GalNAc transporters.

Some people are immunocompromised and their bodies are hence much less capable of fending off and defeating diseases (i.e. influenza, ...). In some cases this is due to genetic flaws[clarification needed] or even genetic diseases such as SCID. Some gene therapies have already been developed or are being developed to correct these genetic flaws/diseases, hereby making these people less susceptible to catching additional diseases (i.e. influenza, ...).[4]

In November 2018, Lulu and Nana were created.[5] By using clustered regularly interspaced short palindromic repeat (CRISPR)-Cas9, a gene editing technique, they disabled a gene called CCR5 in the embryos, aiming to close the protein doorway that allows HIV to enter a cell and make the subjects immune to the HIV virus.

Athletes might adopt gene therapy technologies to improve their performance.[6] Gene doping is not known to occur, but multiple gene therapies may have such effects. Kayser et al. argue that gene doping could level the playing field if all athletes receive equal access. Critics claim that any therapeutic intervention for non-therapeutic/enhancement purposes compromises the ethical foundations of medicine and sports.[7]

Other hypothetical gene therapies could include changes to physical appearance, metabolism, mental faculties such as memory and intelligence.

Some congenital disorders (such as those affecting the muscoskeletal system) may affect physical appearance, and in some cases may also cause physical discomfort. Modifying the genes causing these congenital diseases (on those diagnosed to have mutations of the gene known to cause these diseases) may prevent this.

Also changes in the mystatin gene[8] may alter appearance.

Behavior may also be modified by genetic intervention.[9] Some people may be aggressive, selfish, ... and may not be able to function well in society.[clarification needed] There is currently research ongoing on genes that are or may be (in part) responsible for selfishness (i.e. ruthlessness gene, aggression (i.e. warrior gene), altruism (i.e. OXTR, CD38, COMT, DRD4, DRD5, IGF2, GABRB2[10])

There is some research going on on the hypothetical treatment of psychiatric disorders by means of gene therapy. It is assumed that, with gene-transfer techniques, it is possible (in experimental settings using animal models) to alter CNS gene expression and thereby the intrinsic generation of molecules involved in neural plasticity and neural regeneration, and thereby modifying ultimately behaviour.[11]

In recent years, it was possible to modify ethanol intake in animal models. Specifically, this was done by targeting the expression of the aldehyde dehydrogenase gene (ALDH2), lead to a significantly altered alcohol-drinking behaviour.[12] Reduction of p11, a serotonin receptor binding protein, in the nucleus accumbens led to depression-like behaviour in rodents, while restoration of the p11 gene expression in this anatomical area reversed this behaviour.[13]

Recently, it was also shown that the gene transfer of CBP (CREB (c-AMP response element binding protein) binding protein) improves cognitive deficits in an animal model of Alzheimers dementia via increasing the expression of BDNF (brain-derived neurotrophic factor).[14] The same authors were also able to show in this study that accumulation of amyloid- (A) interfered with CREB activity which is physiologically involved in memory formation.

In another study, it was shown that A deposition and plaque formation can be reduced by sustained expression of the neprilysin (an endopeptidase) gene which also led to improvements on the behavioural (i.e. cognitive) level.[15]

Similarly, the intracerebral gene transfer of ECE (endothelin-converting enzyme) via a virus vector stereotactically injected in the right anterior cortex and hippocampus, has also shown to reduce A deposits in a transgenic mouse model of Alzeimers dementia.[16]

There is also research going on on genoeconomics, a protoscience that is based on the idea that a person's financial behavior could be traced to their DNA and that genes are related to economic behavior. As of 2015, the results have been inconclusive. Some minor correlations have been identified.[17][18]

George Church has compiled a list of potential genetic modifications based on scientific studies for possibly advantageous traits such as less need for sleep, cognition-related changes that protect against Alzheimer's disease, disease resistances, higher lean muscle mass and enhanced learning abilities along with some of the associated studies and potential negative effects.[19][20]

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Human genetic enhancement - Wikipedia

THE IDEA

A team of Vanderbilt researchers led byJamey Young, Cornelius Vanderbilt Professor of Engineering,John McLean, department chair and Stevenson Professor of Chemistry, and Carl Johnson, Cornelius Vanderbilt Professor of Biological Sciences, has described a novel method for rapidly characterizing the biological impact of genetic editing on bacteria. The new approach enables researchers to take the consequences of specific genetic edits into account as they engineer the production of specific chemicals from bacteria.

Ultimately the goal of this work is to discover the best way to genetically modify blue-green algae called cyanobacteria to produce fatty acid molecules that are used as a source of clean energy.Youngled this component of the work. The advanced analytical techniques developed in the McLean lab will enable our project team to rapidly engineer cyanobacteria and other microbes for high-yield production of medium-chain free fatty acids, which are readily converted into fuels, he said.

Rather than probing one gene edit, we are probing multiple bacterial strains with different gene edits to discern which are productive for what we want to accomplishand to understand all the consequences of gene editing on the bacterias overall biology, McLean said. We can characterize many gene editsor strainsin one experiment.

The research could lead to much shorter timeframes for green fuel production, drug discovery and the translation of research from the lab to the public in a variety of disciplines.

The societal, environmental and economic benefits of this type of energy production are massive, McLean said.

Further, the researchers were able to rapidly examine the consequences of gene editing on the target and on the complete biology of the organism, instead of the traditional examination of one molecule at a time. As a result, researchers can look at multiple gene edits at once, cutting down the time for synthetic experiments.

Imagine growing algae at your home in a resealable zipper storage bag that is really making fuel for your energy needs, McLean said. That is a very long-term outcome of this research, but we are one step closer toward optimizing how bacterial strains produce this kind of fuel.

With expertise and access to myriad state-of-the-art microscopy instruments, McLean and his team are continuing to develop new approaches that enhance the technologys performance and outputs.

This work was supported by U.S. Department of Energy, Office of Science, Biological and Environmental Research Division under award number DE-SC00019404 and the U.S. Department of Energy, Office of Science, Office of Biological and Environmental Research, Genomic Science Program under award number DE-SC0022207. Financial support for aspects of this research was also provided by the National Institutes of Health grants NIH NIGMS R01GM092218, NIGMS R37GM067152, NCI R03CA222-452-01 and NCI 1F32GM128344-01, the U.S. Environmental Protection Agency under Assistance Agreement 83573601, and the U.S. Army Research Office and the Defense Advanced Research Projects Agency under Cooperative Agreement W911 NF-14-2-0022.

With the McLean laboratorys status as aWaters Center of Innovation, the team had access to instrumental and early-stage technology that made this work possible.

The article, Accelerating Strain Phenotyping with Desorption Electrospray Ionization-Imaging Mass Spectrometry and Untargeted Analysis of Intact Microbial Colonies,waspublished inthejournalProceedings of the National Academy of SciencesonDec. 7.

The papers co-authorsincludeJody C. May, research assistant professor of chemistry, andBrian F. Pfleger, professor of chemical and biological engineering at University of WisconsinMadison. The papers first authors,Piyoosh BabeleandBerkley M. Ellis, are graduate students in the Young and McLean labs, respectively.

Read more:

Research Snapshot: Producing green fuel, and more rapid determination of the biological consequences of gen... - Vanderbilt University

UNIGE Scientists have discovered how the absence of a genetic switch can lead to malformations during embryonic development.

Embryonic development follows delicate stages: for everything to go well, many genes must coordinate their activity according to a very meticulous scheme and tempo. This precision mechanism sometimes fails, leading to more or less disabling malformations. By studying the Pitx1 gene, one of the genes involved in the construction of the lower limbs, a team from the University of Geneva (UNIGE), in Switzerland, has discovered how a small disturbance in the activation process of this gene is at the origin of clubfoot, a common foot malformation. Indeed, even a fully functional gene cannot act properly without one of its genetic switches. These short DNA sequences provide the signal for the transcription of DNA into RNA, and are essential for this mechanism. And when just one of these switches is missing, the proportion of cells where the gene is active decreases, preventing the lower limbs from being built properly. These results, which can be read in the journal Nature Communications, highlight the hitherto largely underestimated role of genetic switches in developmental disorders.

During embryonic development, hundreds of genes must be precisely activated or repressed for organs to build properly. This control of activity is directed by short DNA sequences that, by binding certain proteins in the cell nucleus, act as true ON/OFF switches. When the switch is turned on, it initiates the transcription of a gene into RNA, which in turn is translated into a protein that can then perform a specific task, explains Guillaume Andrey, professor in the Department of Genetic and Developmental Medicine at the UNIGE Faculty of Medicine, who led this research. Without this, genes would be continuously switched on or off, and therefore unable to act selectively, in the right place and at the right time.

In general, each gene has several switches to ensure that the mechanism is robust. However, could the loss of one of these switches have consequences? This is what we wanted to test here by taking as a model the Pitx1 gene, whose role in the construction of the lower limbs is well known, says Raquel Rouco, a post-doctoral researcher in Guillaume Andreys laboratory and co-first author of this study.

To do this, the scientists modified mouse stem cells using the genetic engineering tool CRISPR-CAS 9, which makes it possible to add or remove specific elements of the genome. Here, we removed one of Pitx1s switches, called Pen, and added a fluorescence marker that allows us to visualize the gene activation, explains Olimpia Bompadre, a doctoral student in the research team and co-first author. These modified cells are then aggregated with mouse embryonic cells for us to study their early stages of development.

Usually, about 90% of cells in future legs activate the Pitx1 gene, while 10% of cells do not. However, when we removed the Pen switch, we found that the proportion of cells that did not activate Pitx1 rose from 10 to 20%, which was enough to modify the construction of the musculoskeletal system and to induce a clubfoot, explains Guillaume Andrey. Indeed, the proportion of inactive cells increased particularly in the immature cells of the lower limbs and in the irregular connective tissue, a tissue that is essential for building the musculoskeletal system.

Beyond the Pitx1 gene and clubfoot, the UNIGE scientists have discovered a general principle whose mechanism could be found in a large number of genes. Flawed genetic switches could thus be at the origin of numerous malformations or developmental diseases. Moreover, a gene does not control the development of a single organ in the body, but is usually involved in the construction of a wide range of organs. A non-lethal malformation, such as clubfoot for example, could be an indicator of disorders elsewhere in the body that, while not immediately visible, could be much more dangerous. If we can accurately interpret the action of each mutation, we could not only read the information in the genome to find the root cause of a malformation, but also predict effects in other organs, which would silently develop, in order to intervene as early as possible, the authors conclude.

Reference: Cell-specific alterations in Pitx1 regulatory landscape 1 activation caused 2 by the loss of a single enhancer 13 December 2021, Nature Communication.DOI: 10.1038/s41467-021-27492-1

Original post:

Missing Genetic Switch at the Origin of Malformations During Embryonic Development - SciTechDaily

Life goes on as gene-edited foods begin to hit the market. Japanese consumers have recently started buying tomatoes that fight high blood pressure, and Americanshave been consuming soyengineered to produce high amounts of heart-healthy oils for a little over two years. Few people noticed these developments because, as scientists have said for a long time, the safety profile of a crop is not dictated by the breeding method that produced it. For all intents and purposes, it seems that food-safety regulators have done a reasonablejob of safeguarding public health against whatever hypothetical risks gene editing may pose.Credit: Karuchibe

But this has not stopped critics of genetic engineering from advocating for more federal oversight of CRISPR and othertechniquesused to make discrete changes to the genomes of plants, animals and other organisms we use for food or medicine. Over at The Conversation, a team of scientists recently made the case for tighter rules inCalling the latest gene technologies natural is a semantic distraction they must still be regulated.

Many scientists have defended gene editing, in part, by arguing that it simply mimics nature. A mutation that boosts the nutrient content of rice, for example, is the same whether it was induced by a plant breeder or some natural phenomenon. Indeed, the DNA of plants and animals we eatcontains untold numbersof harmless, naturally occurringmutations. But The Conversation authors will havenone of this:

Unfortunately, the risks from technology dont disappear by calling it natural Proponents of deregulation of gene technology use the naturalness argument to make their case. But we argue this is not a good basis for deciding whether a technology should be regulated.

They have written a very longpeer-reviewed articleoutlining a regulatory framework based on scale of use.The ideais that the more widely a technology is implemented, the greater risk it may pose to human health and the environment, which necessitates regulatory control points to ensure its safe use. Its an interesting proposal, but its plagued by several serious flaws.

The most significant issue with a scale-based regulatory approachis that its a reaction to risks that have never materialized. This isnt to say that a potentially harmful genetically engineered organism will never be commercialized. But if were going to upend our biotechnology regulatory framework, we need to do so based on real-world evidence. Some experts have actually argued, based on decades of safety data, that the US over-regulates biotech products. As biologist and ACSHadvisorDr. Henry Miller and legal scholar John Cohrssen wroterecently in Nature:

After 35 years of real-world experience with genetically engineered plants and microorganisms, and countless risk-assessment experiments, it is past time to reevaluate the rationale for, and the costs and benefits of, the case-by-case reviews of genetically engineered products now required by the US Environmental Protection Agency (EPA), US Department of Agriculture (USDA) and US Food and Drug Administration (FDA).

Real-world data aside for the moment, there are some theoretical problems with the scalabilitymodel as well. Theargument assumes thatrisks associated with gene editing proliferate as use of the technology expands, because each gene edit carries a certain level of risk. This is a false assumption, as plant geneticist Kevin Folta pointed out on a recentepisode of the podcastwe co-host (21 minute mark).

Scientists have a variety of tools with which to monitor and limit the effects of specific gene edits. For example,proteins known asanti-CRISPRs can be utilized to halt the gene-editing machinery so it makes only the changes we want it to. University of Toronto biochemist Karen Maxwell has explained how this couldwork in practice:

In genome editing applications, anti-CRISPRs may provide a valuable off switch for Cas9 activity for therapeutic uses and gene drives. One concern of CRISPR-Cas gene editing technology is the limited ability to control its activity after it has been delivered to the cell . which can lead to off-target mutations. Anti-CRISPRs can potentially be exploited to target Cas9 activity to particular tissues or organs, to particular points of the cell cycle, or to limit the amount of time it is active

Suffice it to say that these and other safeguards significantly alter the risk equation and weaken concerns about a gene-edits-gone-wild scenario. Parenthetically, scientists design these sorts of preventative measures as they developmoregenetic engineering applications for widespread use. This is why the wide variety of cars in production todayhave safety featuresthat would have been unheard of in years past.

To bolster their argument, The Conversation authors made the following analogy:

Imagine if other technologies with the capacity to harm were governed by resemblance to nature. Should we deregulate nuclear bombs because the natural decay chain of uranium-238 also produces heat, gamma radiation and alpha and beta particles? We inherently recognize the fallacy of this logic. The technology risk equation is more complicated than a supercilious its just like nature argument

If someone has to resort to this kind of rhetoric, the chances are excellent that their argument is weak. Fat Man and Little Boy,the bombs droppedon Japan in 1945, didnt destroy two cities because a nuclear physicist in New Mexico made a technical mistake. These weapons are designed to wreak havoc. Tomatoes bred to produce more of an amino acid, in contrast, are not.

The point of arguing that gene-editing techniques mimic natural processes isnt to assert that natural stuff is good; therefore, gene editing is also good. Instead, the point is to illustrate that inducing mutations in the genomes of plants and animals is not novel or uniquely risky. Even the overpriced products marketed as all-naturalhave been improvedby mutations resulting from many years of plant breeding.

Nonetheless, some scientistshave arguedthat reframing the gene-editing conversation in terms of risk vs benefit would be a smarter approach than making comparisons to nature. I agree with them, so lets start now. The benefits of employing gene editing to improve our food supply and treat disease far outweigh the potential risks, which we can mitigate. Very little about modern life is naturaland its time we all got over it.

Cameron J. English is the director of bio-sciences at theAmerican Council on Science and Health. Visithis websiteand follow ACSH on Twitter@ACSHorg

A version of this article was originally posted at theAmerican Council on Science and Healthand is reposted here with permission. The American Council on Science and Health can be found on Twitter@ACSHorg

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Viewpoint: An argument for CRISPR crops 'Very little about modern life is natural and it's time we all got over it' - Genetic Literacy Project

image:From left to right: Marc Gell, Dimitrije Ivani, Avencia Snchez-Mejas and Maria Pallars. view more

Credit: UPF

An international, multidisciplinary team of researchers from theTranslational Synthetic Biology Laboratoryat Pompeu Fabra University (Barcelona, Spain), led by Dr.Marc Gell, has published an article in the scientific journalNature Communicationsshowing the potential of Find Cut-and-Transfer (FiCAT) technology as a state-of-the-art tool forgene writingto develop advanced therapies that are safer and more effective in their future clinical application in patients withgenetic and oncological diseasesthat have few treatment options.

The UPF Translational Synthetic Biology Laboratory has been working on gene editing and synthetic biology applied to gene therapies since 2017. FiCAT technology is an important scientific breakthrough to overcome the current limitations of the technology used today for genome editing and gene therapy.

Human genome engineering has significantly progressed in the last decade with the development of new editing tools, but there was still a technology gap that would allow therapeutic genes to be transferred efficiently with few size limitations, comments Dr. Marc Gell, supervisor of the study.

In this work, the researchers develop an efficient and precise programmable gene writing technology based on the combination of modified proteins CRISPR-cas and piggy Bac transposase (PB), succeeding in inserting small and large fragments. Dr.Maria Pallars, co-first author of the study explains that: CRISPR stands out for its precision when editing small fragments. However, transposases allow us to insert large fragments but in an uncontrolled manner. We have combined the best of each technology.

In this way, FiCAT technology allows us to precisely insert large fragments of DNA into the genome. This means we can develop therapeutic solutions to diseases that currently have no treatment, such as Duchenne muscular dystrophy, or some cases of hereditary blindness, in which the affected gene is large in size, asserts Dr.Avencia Snchez-Mejas, a senior researcher with the group and co-supervisor of the work.

They tested the technology in human and mouse cell lines achieving efficiencies of 522% with minimal off-target insertions and have demonstrated on-target gene transfer in vivo in mouse liver and germline cells in mouse models. Lastly, they performed a directed evolution of FiCAT and further improved efficiency by 25-30%. We have been progressively modifying enzymes so that they acquire the function we were looking for, selecting the ones that displayed a better function, detailsDimitrije Ivani, co-first author of the article. Our work is a clear example that enzyme engineering in the context of genome editing has great potential, he concludes.

UPF has transferred FiCAT technology via the spinoffIntegra Therapeutics, founded in 2020 by the researchers Marc Gell and Avencia Snchez-Mejas, seeking to get this scientific knowledge and technological capacity to reach the biopharmaceutical industry to develop safe and efficient advanced technologies that reach patients. Recently, Integra Tx has obtained 4.5 million euros in funding from Advent France Biotechnology (France), Invivo Capital (Spain) and Takeda Ventures (USA).

The work published in Nature Communications was carried out with funding from the Societal challenges AEI, AGAUR- PRODUCTE, UPGRADE-Horizon 2020 of the European Commission; la Caixa CaixaImpulse Validate and CaixaImpulse Consolidate programmes; the Ramn Areces Foundation; and the Ramn y Cajal programme of the Spanish Ministry of Economy, Industry and Competitiveness.

Reference article:

Pallars-Masmitj, M; Ivani, D; Mir-Pedrol, J; Jaraba-Wallace, J; Tagliani, T; Oliva, B; Snchez-Mejas, A; Gell, M. Find and cut-and-transfer (FiCAT) mammalian genome engineering. Nature Communications (2021). DOI: 10.1038/s41467-021-27183-x.

Nature Communications

Experimental study

Cells

Find and cut-and-transfer (FiCAT) mammalian genome engineering

3-Dec-2021

A.S.-M., D.I., M.G. and M.P.-M. have filed patent applications on FiCAT technology. Patent applicant: Pompeu Fabra University; application number: PCT/IB2020/055507; status of application: pending; specific aspect of manuscript covered in patent application: this patent application covered the general aspects of DNA binding proteins fused to integrases and transposases. Specifically, Fig. 1b, Supplementary Fig. 2a and preliminary data on activity characterization of some of the hyPB mutants included in Fig. 1c, d were disclosed in this patent application. A.S.-M. and M.G. are shareholders of Integra Therapeutics, company that licensed FiCAT technology. The remaining authors declare no competing interests.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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New gene-writing technology to obtain more effective and safe therapies developed - EurekAlert

In a new mouse study, scientists at the Icahn School of Medicine at Mount Sinai demonstrate how the activity of one gene, turned on in a newly discovered group of bone-bordering cells, may play an important role in shaping the skull.

The findings are published in the journalNature Communications in a paper titled, Single-cell analysis identifies a key role for Hhip in murine coronal suture development, and led by Greg Holmes, PhD, assistant professor of genetics and genomic sciences at Icahn Mount Sinai.

Craniofacial development depends on formation and maintenance of sutures between bones of the skull, the researchers wrote. In sutures, growth occurs at osteogenic fronts along the edge of each bone, and suture mesenchyme separates adjacent bones. Here, we perform single-cell RNA-seq analysis of the embryonic, wild type murine coronal suture to define its population structure.

Researchers focused on the cells of the coronal suture, a fibrous joint that connects the front and middle bone plates.

The Holmes lab worked with researchers in the labs of Bin Zhang, PhD, Harm van Bakel, PhD, and Ethylin Wang Jabs, MD, of Icahn Mount Sinai. Together they studied how the genetic activity in the cells of the coronal suture changes during early development.

Their findings suggested that a gene encoding a molecule called hedgehog interacting protein (HHIP) plays a unique role in coronal suture development. The researchers observed the gene was more active in a novel group of mesenchyme cells than it was in osteoblasts.

Using single-cell with bulk RNA-seq analysis we have better defined the distinctive composition of the coronal suture at the transcriptional and cell population levels, the researchers wrote.

Looking toward the future, the researchers hope that advanced single-cell genetic studies like this one will pave the way for a more thorough understanding of how a skull is shaped under healthy and disease conditions.

Our transcriptomic approach greatly expands opportunities for hypothesis-driven research in coronal and other suture development, concluded the researchers.

See more here:

Researchers Dig Up Genes and Cells Related to Skull Formation in Mice - Genetic Engineering & Biotechnology News

Figure 1. Leading researchers in the collaborative project. The full list of the co-authors in the Nature Communication paper: Zhong Guo, Oleh Smutok, Wayne A. Johnston, Patricia Walden, Jacobus P. J. Ungerer, Thomas S. Peat, Janet Newman, Jake Parker, Tom Nebl, Caryn Hepburn, Artem Melman, Richard J. Suderman, Evgeny Katz, Kirill Alexandrov.

The best and most efficient way to perform multi-disciplinary research is by doing it in collaboration. One of such research programs, including synthetic biology, materials science, bioelectrochemistry, bioelectronics, and biosensors, has been performed in a close collaboration between scientists at the Department of Chemistry and Biomolecule Science, Clarkson University, Dr. Oleh Smutok, Dr. Artem Melman (deceased on November 25, 2021), and Dr. Evgeny Katz, with a team of Australian scientists led by Dr. Kirill Alexandrov, Queensland University of Technology (Figure 1). This collaboration being active for several years has been supported with grants from Human Frontiers Science Program (HFSP) and US Department of Defense with the total funding over 1 million dollars. The results from the collaborative efforts have been published in numerous scientific papers and covered by several patents. The most recent and impressive publication was a paper in Nature Communications one of the top scientific journals (Impact Factor 14.92). The paper entitled Design of a methotrexate-controlled chemical dimerization system and its use in bio-electronic devices (Nature Commun. 2021, 12 article No. 7137) reports on a novel artificial enzyme produced by genetic engineering that can be activated with a drug (methotrexate) molecules. The artificial enzyme was immobilized at an electrode surface and used for the drug biosensing with extremely high sensitivity and specificity (Figure 2).

In addition to the fundamental novelty of using the artificial signal-activated enzyme, the study is highly relevant for practical biomedical application. Methotrexate is a toxic drug used in anti-cancer chemotherapy and its overdose has serious, life-threatening side effects. Thus, the methotrexate analysis in biological fluids is important for keeping the drug at the optimal concentration. The study opens the future options for biomedical applications of the developed biosensor and possibilities for other biosensing systems based on the same concept. It should be noted that the success of this project was based on the collaboration of scientists with expertise in different areas, synthetic biology, synthetic organic chemistry, and bioelectrochemistry. This is an exemplary collaboration that serves as a model of performing multi-disciplinary research. While the artificial enzyme preparation was carried out by the Australian team led by Dr. Alexandrov, the bioelectrochemical study of the developed biosensor was performed by Dr. Smutok at Clarkson University. Both US and Australian teams are continuing their successful work combining synthetic biology and bioelectronics and are expecting many more interesting and practically important results. The scientific efforts are combined with the education of graduate and undergraduate students participating in the project.

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Nature Communications paper published by two collaborating teams at Clarkson University (NY, USA) and Queensland University of Technology (Australia)...

P-BCMA-101 demonstrated strong anti-tumor activity and was well tolerated in nearly 100 patients with R/R multiple myeloma at the time of data cutoff

P-BCMA-101 in combination with rituximab achieved an improved overall response rate of 78% and 100% overall survival

Learnings from P-BCMA-101 informed development of the Company's first fully allogeneic CAR-T product candidate, P-BCMA-ALLO1

SAN DIEGO, Dec. 13, 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 reported interim results from its Phase 1/2 PRIME clinical trial of P-BCMA-101 for the treatment of relapsed/refractory multiple myeloma (R/R MM) at the 2021 American Society of Hematology (ASH) Annual Meeting.

Poseida Therapeutics (PRNewsfoto/Poseida Therapeutics, Inc.)

The results show that P-BCMA-101, a non-viral transposon-based autologous CAR-T, was well tolerated and demonstrated strong anti-tumor activity in advanced, late line R/R MM patients. The learnings from P-BCMA-101 informed the development of the Company's first allogeneic program, P-BCMA-ALLO1 which is also being evaluated in R/R MM patients. The Company previously announced that it is winding down the P-BCMA-101 autologous program in favor of the allogeneic program, P-BCMA-ALLO1.

"We are encouraged by the outcomes seen from our clinical trial of P-BCMA-101, results that continue to validate our approach and that have informed P-BCMA-ALLO1, our first fully allogeneic CAR-T program for patients with multiple myeloma, as well as our other programs. Our focus is on creating differentiated product candidates with a high percentage of T stem cell memory (Tscm) cells," said Eric Ostertag, M.D., Ph.D., chief executive officer of Poseida Therapeutics. "Looking ahead, we continue to advance P-BCMA-ALLO1 and P-MUC1C-ALLO1 and look forward to presenting data in 2022 for both of these allogeneic programs."

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The PRIME trial is a Phase 1/2, open label 3+3 single dose escalation of P-BCMA-101 CAR-T cells. The primary objective of the study is to determine the safety and maximum tolerated dose of P-BCMA-101 based on dose limiting toxicities (DLT), and the key secondary objective is to assess the anti-myeloma effect of the product. The median patient age was 62, with a median time since diagnosis of approximately 5.8 years. Patients were heavily pre-treated, with a median of 7 prior lines of therapy (2-18). As of the data cut-off date of October 15, 2021, a total of 98 patients have been dosed with P-BCMA-101.

The best observed treatment regimen was a combination with rituximab (n=14), with an overall response rate (ORR) of 78%, a VGPR/sCR rate of 43% and 100% overall survival at the time of the data cutoff. Progression free survival was also improved with rituximab, with median overall survival rates not yet reached in several cohorts including the rituximab combination cohorts. Response rates for other cohorts are consistent with results previously reported.

Across the study, no dose-limiting toxicities were observed. 28% of patients developed cytokine release syndrome (CRS) and 7% of patients developed neurotoxicity. None of the patients developed Grade 3 or higher CRS, and 2% of patients developed Grade 3 neurotoxicity. There were no treatment-related deaths among the patient population and no patients needed ICU admission as a result of CAR-T related toxicities. 28 patients were treated on a fully outpatient basis.

"P-BCMA-101 demonstrated strong anti-tumor activity in advanced multiple myeloma patients, and cohorts to date have shown minimal CRS and neurotoxicity, which allows for safe administration in an outpatient environment and combinations with other therapies," said Caitlin Costello, M.D., Associate Clinical Professor of Medicine and member of the Division of Blood and Marrow Transplantation at the University of California, San Diego. "These data indicate that the piggyBac transposon-based platform is an attractive option for allogeneic CAR-T cells, which has led to a first-in-human Phase 1 study."

The Company's first fully allogeneic CAR-T cell product, P-BCMA-ALLO1 utilizes Poseida's proprietary piggyBac DNA delivery system and Cas-CLOVER site-specific gene editing system to create an allogeneic product that prevents both graft-vs-host and host-vs-graft diseases and also incorporates a next-generation BCMA binder. P-BCMA-ALLO1 manufacturing involves a proprietary "booster" molecule that allows for numerous doses to be produced from a single manufacturing run, while maintaining desirable Tscm cells, which can reach percentages in the 60-80% range.

The Investigational New Drug (IND) application for P-BCMA-ALLO1 was given a safe to proceed designation by the FDA in August 2021. The Phase 1 study is an open label, dose escalation study following a 3+3 design of dose escalation in subjects with R/R MM. The study will assess the safety and maximum tolerated dose of P-BCMA-ALLO1 based on dose limiting toxicities. Key secondary objectives of the study include the anti-myeloma effect and safety of P-BCMA-ALLO1.

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 Delivery System, Cas-CLOVER Site-specific Gene Editing System and 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.

Forward-Looking StatementStatements 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, among other things, the potential benefits of Poseida's technology platforms and product candidates, Poseida's plans and strategy with respect to developing its technologies and product candidates, and anticipated timelines and milestones with respect to Poseida's development programs and manufacturing activities. 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, risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry and the other risks described in Poseida's filings with the Securities and Exchange Commission. All forward-looking statement 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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Poseida Therapeutics Provides Update on BCMA-Targeted CAR-T Clinical Trials at the 2021 American Society of Hematology (ASH) Annual Meeting - Yahoo...

The global genome editing market is expected to reach US$ 10,691.0 million by 2025 from US$ 3,210.1 million in 2017; it is estimated to grow at a CAGR of 17.0% from 2018 to 2027.

According The Insight Partners study on Genome Editing Market Forecast to 2027 COVID-19 Impact and Global Analysis by Technology, Application, End User, The report highlights trends existing in the market, and drivers and hindrances pertaining to the market growth. Factors such as Increase in funding for the genome editing, rising prevalence of the genetic disorders, rise in the advancements for genome editing technology and rise in the production of genetically modified crops are the driving factors for the growth of the market.

Genome editing is a technique that is utilized for the changes that are to be done in the DNA of a cell or an organism. The technique involves cutting DNA sequences for the addition or removing the DNA in the genome. The changes in the genome are done for the required characteristics of the cell. Genome editing is done for the research purpose, the treatment of the diseases, and the biotechnological purpose.

Get Sample PDF Copy of Genome Editing Market at: https://www.theinsightpartners.com/sample/TIPHE100000853/

Market Insights

Increase in Funding for the Genome Editing

The market for genome editing is expected to grow in the coming near future due to the growth factor that is driving the market is the increase in the funding. The different government in the different regions are increasing their funds and grants to develop genome editing research. Owing to genome editings advantages, the various government is supporting their public and private research and academic institutes for increasing the research activities for the genome editing and genetic engineering.

Across the world, funding is being provided by every nation. However, the more funds, for instance, in January 2018 US government announced donating US$ 190 million for research for the next six years. Also, the government is hoping to develop therapies to treat cancer and other diseases using gene editing. In addition, the National Institutes of Health (NIH) has kept approximately US$ 45.5 million aside for the next four fiscal years for the Somatic Cell Genome Editing program. Moreover, in the Asia Pacific region, the countries are also investing more in the development of genome editing technology for two-three years back. For instance, in April 2016, Japan invested approximately US$76million for the five years for the creation of Japanese owned genome editing technologies.

Furthermore, the investments are made for private companies operating for genome editing. For instance, in August 2015, Editas Medicine is a company at the forefront of developing the gene-editing technology CRISPR has received US$ 120 million to create a new treatment for the conditions which include cancer, retinal diseases, and sickle cell anemia. Therefore, the rise in the funding for genome editing is likely to drive the market for genome editing in the forecast period. The rise in the funding will enhance the research and development of the gene-editing technologies and products for the researchers for efficient and effective genome editing. The funding will also enable the biopharmaceutical and pharmaceutical companies to develop technologies for the therapies using gene editing to treat and diagnose chronic diseases.

It also includes the impact of the COVID-19 pandemic on the market across all the regions. The Genome Editing Market , by region, is segmented into North America, Europe, Asia Pacific (APAC), Middle East and Africa (MEA), and South and Central America (SAM).

COVID-19 first began in Wuhan (China) during December 2019 and since then it has spread at a fast pace across the globe. The US, India, Brazil, Russia, France, the UK, Turkey, Italy, and Spain are some of the worst affected countries in terms confirmed cases and reported deaths. The COVID-19 has been affecting economies and industries in various countries due to lockdowns, travel bans, and business shutdowns.

Download the Latest COVID-19 Analysis on Genome Editing Market Growth Research Report at: https://www.theinsightpartners.com/covid-analysis-sample/TIPHE100000853

Based on technology, the genome editing market is segmented into transcription activator-like effector nucleases (TALENS), clustered regularly interspaced short palindromic repeats (CRISPR), zinc finger nucleases (ZFNs), antisense RNA and others. In 2017, the CRISPR segment held the largest share of the market, by technology owing to the applications and its benefits offered. The TALENs segment is expected to grow at the fastest rate during the coming years.

Based on application, the genome editing market is segmented into genetic engineering, cell line engineering and others. In 2017, cell line segment held the largest share of the market, by application. Moreover, the genetic engineering segment is expected to grow at the fastest rate during the coming years owing to its sub segments such as animal genetic engineering and plant genetic engineering that are being carried out extensively.

Based on end user, the genome editing market is segmented into biotechnology & pharmaceutical companies, contract research organizations, academic & government research organization and other end users. The market is dominated by the biotechnology & pharmaceutical companies and is expected to surge significantly during the forecast period from 2017 to 2025. The biotechnology & pharmaceutical companies segment is expected gain its market share during the forecast period. Also, biotech & pharmaceutical companies is expected to show a prime CAGR owing to the increasing government funding and partnerships between the various organizations in all the regions.

Genome Editing Market : Competitive Landscape and Key Developments

Transposagen Biopharmaceuticals, Inc.,Integrated DNA Technologies, Inc.,Thermo Fisher Scientific Inc.,GenScript,Lonza,Horizon Discovery Group plc,Sangamo Therapeutics, Inc.,New England Biolabs,Editas Medicine,Merck KGaA.

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The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We specialize in industries such as Semiconductor and Electronics, Aerospace and Defense, Automotive and Transportation, Biotechnology, Healthcare IT, Manufacturing and Construction, Medical Device, Technology, Media and Telecommunications, Chemicals and Materials.

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Genome Editing Market to hit US$ 10691.0 Million, Globally, by 2025 at 17.0% CAGR: The Insight Partners - Digital Journal

Like many of the technologies that are driving innovation in the alternative protein space, plant molecular farming has traditionally been used in the pharmaceutical industry. The practice which involves genetically editing a crop so that its cells produce a desired protein is being discussed as a way to rapidly produce proteins for COVID-19 vaccines.

In the food industry, molecular farming is one route to producing the animal proteins that give egg, dairy, and meat products their visual, taste, and functional properties. Molecular farming allows you to use the exact same protein that would normally be produced by a chicken or cow, without the need for any actual animals.

Moolec Science, a spinoff of Argentina-based agtech company Bioceres Crop Solutions, is probably the most prominent name in molecular farming for the food industry. Moolec already sells chymosin, a cheesemaking enzyme, which the company grows in safflower plants. Theyve also successfully grown meat proteins in soybean and pea plants.

The Moolec team believes that molecular farming can help to bring down the end costs of alternative meat products. (Theres nothing better than low-tech farming to produce at an enhanced scale and low cost, company CEO and co-founder Gastn Paladini told The Spoon back in October.) And they may be right.

Molecular farming can help producers to avoid some of the costly and tricky problems of growing proteins in traditional bioreactors. When you use a plant as your bioreactor, as food scientist and thought leader Tony Hunter pointed out in an article this year, you dont need to worry about maintaining sterile conditions: Plants have built-in immune systems.

Moolec plans to launch its first animal-free meat protein in late 2022 or early 2023. The company is currently working toward regulatory approval for its products and its progress will be an interesting test of regulatory tolerance of Moolecs brand of genetic engineering.

One potential concern for regulators as they scrutinize molecular farming processes will be the possibility of gene flow from modified crops to related plants. Tiamat Sciences, a Belgium-based molecular farming startup, is limiting that possibility by growing its crops in a contained vertical farming system.

Tiamat has plans to expand alongside the cell-based meat industry. By targeting nascent markets on the verge of scale-up, weve already demonstrated significant traction for our solutions and an early revenue potential that is outstanding for a biotech startup, said Tiamats founder and CEO France-Emmanuelle Adil in a recent press release. The company currently produces GRAS-certified, animal-free growth factors for cultivated meat, and also manufactures proteins for the pharmaceutical industry.

Last month, Tiamat announced that it had raised a $3 million seed funding round led by Silicon Valley venture capital firm True Ventures. The company is using those funds to construct a pilot facility in Durham, N.C. so we may see them boost their capacity in the year to come.

Molecular farming startups still have some issues to work out. As Tony Hunter noted in his piece on molecular farming, plant tissue has larger and fewer protein-producing cells compared to the same volume of mammal tissue, making plants less productive as protein factories. And there are costs associated with extracting protein molecules from plants at the cellular level.

Still, the same upsides of molecular farming that make it attractive to the pharmaceutical industry will likely continue to spark interest from alternative protein producers especially as those producers seek ways to bring down the retail prices of their products.

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In 2022, Molecular Farming Startups Will Move Toward Commercialization of Animal-Free Proteins - The Spoon

One 2021 study looked at the genome of Solanum sitiens a wild tomato species which grows in the extremely harsh environment of the Atacama Desert in Chile, and can be found at altitudes as high as 3,300m (10,826ft). The study identified several genes related to drought-resistance in Solanum sitiens, including one aptly named YUCCA7 (yucca are draught-resistant shrubs and trees popular as houseplants).

They are far from the only genes that could be used to give the humble tomato a boost. In 2020 Chinese and American scientists performed a genome-wide association study of 369tomato cultivars, breeding lines and landraces, and pinpointed a gene called SlHAK20 as crucial for salt tolerance.

Once the climate-smart genes such as these are identified, they can be targeted using Crispr to delete certain unwanted genes, to tune others or insert new ones. This has recently been done with salt tolerance, resistance to various tomato pathogens, and even to create dwarf plants which could withstand strong winds (another side effect of climate change). However, scientists such as Cermak go even further and start at the roots they are using Crispr to domesticate wild plant species from scratch, "de novo" in science speak. Not only can they achieve in a single generation what previously took thousands of years, but also with a much greater precision.

De novo domestication of Solanum pimpinellifolium was how Cermak and his colleagues at the University of Minnesota arrived at their 2018 plant. They targeted five genes in the wild species to obtain a tomato that would be still resistant to various stresses, yet more adapted to modern commercial farming more compact for easier mechanical harvesting, for example. The new plant also had larger fruits than the wild original.

"The size and weight was about double," Cermak says. Yet this still wasn't the ideal tomato he strives to obtain for that more work needs to be done. "By adding additional genes, we could make the fruit even bigger and more abundant, increase the amount of sugar to improve taste, and the concentration of antioxidants, vitamin C and other nutrients," he says. And, of course, resistance to various forms of stress, from heat and pests to draught and salinity.

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The tomatoes at the forefront of a food revolution - BBC News

GAITHERSBURG, Md., Dec. 13, 2021 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced that it has submitted a regulatory filing to the Ministry of Health and Prevention (MoHaP) for emergency use of its COVID-19 vaccine in the United Arab Emirates (UAE).

"The rapid emergence and continuedspread of variants is a stark reminder that no one is safe until everyone is safe in the fight against COVID-19," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "We remain committed to delivering our vaccine, which is based on a proven, well understood platform, to countries around the world as we anticipate that ongoing vaccination will be necessary over the long term to end the pandemic."

Novavax made the submission for the regulatory evaluation by MoHaP of NVX-CoV2373, the company's recombinant nanoparticle protein-based COVID-19 vaccine with Matrix-M adjuvant. The filing includes clinical data from two pivotal Phase 3 clinical trials: PREVENT-19, which included 30,000 participants in the U.S. and Mexico and demonstrated 100% protection against moderate and severe disease, 93.2% efficacy against the predominantly circulatingvariants of concern and variants of interest, and 90.4% efficacy overall; and a trial of 15,000 participants in the U.K. that demonstrated efficacy of 96.4% against the original virus strain, 86.3% against the Alpha (B.1.1.7) variant and 89.7% efficacy overall. In both trials, NVX-CoV2373 demonstrated a reassuring safety and tolerability profile.

Novavax and Serum Institute of India Pvt. Ltd. (SII) recently received Emergency Use Authorization (EUA) for the vaccine inIndonesiaand thePhilippines, and the companies have filed for EUA inIndiaand for Emergency Use Listing (EUL) with theWorld Health Organization(WHO). Novavax also announced regulatory filings for its vaccine in theUnited Kingdom,Australia,New Zealand,Canada, theEuropean Union, Singapore and with theWHO.Additionally, Novavax and SK bioscience announced a Biologics License Application (BLA)submission to MFDSinSouth Korea. Novavax expects to submit the complete package to the U.S. FDA by the end of the year.

The chemistry, manufacturing and controls (CMC) data package submitted to MoHaP and other global regulatory agencies leverages Novavax' manufacturing partnership with SII, the world's largest vaccine manufacturer by volume. It will later be supplemented with data from additional manufacturing sites in Novavax' global supply chain.

About the NVX-CoV2373 Phase 3 trials

NVX-CoV2373 is being evaluated in two pivotal Phase 3 trials: a trial in the U.K. that demonstrated efficacy of 96.4% against the original virus strain, 86.3% against the Alpha (B.1.1.7) variant and 89.7% efficacy overall; and the PREVENT-19 trial in the U.S. and Mexico that demonstrated 100% protection against moderate and severe disease, 93.2% efficacy against the predominantly circulatingvariants of concern and variants of interest, and 90.4% efficacy overall. It was generally well-tolerated and elicited a robust antibody response.

About NVX-CoV2373

NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax' patented saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19.

Novavax' COVID-19 vaccine is packaged as a ready-to-use liquid formulation in a vial containing ten doses. The vaccination regimen calls for two 0.5 ml doses (5 mcg antigen and 50 mcg Matrix-M adjuvant) given intramuscularly 21 days apart. The vaccine is stored at 2- 8 Celsius, enabling the use of existing vaccine supply and cold chain channels.

About Matrix-M Adjuvant

Novavax' patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About Novavax

Novavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development and commercialization of innovative vaccines to prevent serious infectious diseases. The company's proprietary recombinant technology platform harnesses the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. NVX-CoV2373, the company's COVID-19 vaccine, received Emergency Use Authorization in Indonesia and the Philippines and has been submitted for regulatory authorization in multiple markets globally. NanoFlu, the company's quadrivalent influenza nanoparticle vaccine, met all primary objectives in its pivotal Phase 3 clinical trial in older adults. Novavax is currently evaluating a COVID-NanoFlu combination vaccine in a Phase 1/2 clinical trial, which combines the company's NVX-CoV2373 and NanoFlu vaccine candidates. These vaccine candidates incorporate Novavax' proprietary saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visitwww.novavax.comand connect with us on Twitter,LinkedIn, Instagram and Facebook.

Forward-Looking Statements

Statements herein relating to the future of Novavax, its operating plans and prospects, its partnerships, the ongoing development of NVX-CoV2373, the scope, timing and outcome of future regulatory filings and actions, Novavax' plans to submit a complete package to the U.S. FDA by the end of the year, and Novavax' plan to supplement the CMC data submitted to the MoHaP with data from the additional manufacturing sites in Novavax' global supply chain are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include challenges satisfying, alone or together with partners, various safety, efficacy, and product characterization requirements, including those related to process qualification and assay validation, necessary to satisfy applicable regulatory authorities; difficulty obtaining scarce raw materials and supplies; resource constraints, including human capital and manufacturing capacity, on the ability of Novavax to pursue planned regulatory pathways; challenges meeting contractual requirements under agreements with multiple commercial, governmental, and other entities; and those other risk factors identified in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Novavax' Annual Report on Form 10-K for the year ended December 31, 2020 and subsequent Quarterly Reports on Form 10-Q, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at http://www.sec.gov and http://www.novavax.com, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

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Novavax Files for Emergency Use of COVID-19 Vaccine in the United Arab Emirates - KPVI News 6