Category Archives: Genetic Engineering

LOS ANGELES, Nov. 25, 2019 (GLOBE NEWSWIRE) -- Enochian Biosciences, a company focused on gene-modified cellular therapy in infectious disease and cancer, announces the expansion of its infectious disease pipeline by entering into an agreement in principle to acquire an exclusive, license for a treatment under development aimed to treat the Hepatitis B Virus (HBV) infections from G-Tech Bio, LLC. An abstract accepted for presentation at the HepDART meeting featuring in vivo and in vitro data from preclinical studies conducted with this novel HBV treatment candidate will be presented by Dr. Serhat Gmrkc, MD, PhD on December 10, 2019. Approximately 5 percent of the worlds population is infected with HBV, and around 1 million people per year die from the disease.

The widely respected and well-published collaborating scientist, Dr. Philippe Gallay, PhD, of the Scripps Institute said, I have been working in the hepatitis field for three decades. The mechanism of action conceived of by the brilliant scientist, Dr. Gmrkc, is remarkably innovative. The data generated thus far have exceeded my expectations. I look forward to the presentation at HepDART, an important conference for the field, and to advancing the work with Dr. Gmrkc and Enochian Biosciences.

Building on a Broad and Deep Pipeline

Based on the determinations from independent valuation specialists as contained in our audited financial statements for our fiscal year ended June 30, 2019, our intangible assets related primarily to our HIV pipeline are approximately $154.8 million. The addition of a treatment that we believe could have the potential to cure HBV expands an already exciting pipeline for Enochian.

Enochian licenses are for intellectual property that include proprietary know-how and pending patent applications covering aspects of our pipeline therapeutics. Additionally, Enochian intends to protect the therapeutics that may be approved for marketing in the future with regulatory exclusivity that is available in many jurisdictions around the world. These exclusivity strategies, which are based on pending patent applications, patents, if issued, and regulatory exclusivity, are common among biotech companies as therapeutic candidates proceed through pre-clinical and clinical stages leading to commercialization.

HIV

The cure for HIV has been demonstrated to be more than a theoretical possibility. The so called Berlin and London patients have been cured by undergoing a bone marrow transplant for cancer with cells from another person who had a naturally occurring genetic mutation that makes cells resistant to HIV infection. Unfortunately, this procedure causes a death rate of approximately one-third and causes significant and in some cases, life-long side effects for those who survive. Therefore, it can only be done in people who require a transplant for a life-threatening disease such as lymphoma. Several researchers and companies have attempted to genetically modify the cells of HIV-infected persons and return (transplant) them back into the same person, hoping to reduce the significant side effects with the approach used in the Berlin and London patients. However, the chemotherapy used is still rather toxic, and more significantly, there has not been significant uptake (engraftment) of the genetically modified cells, so the patients have not been cured.

HV-01: a New Approach Toward a Potential Cure for HIV

HV-01 was the original impetus for the conception of Enochian Biosciences HIV focus. It is based on an insight by Dr. Gmrkc, drawn from a non-HIV field, that by adding an additional genetic modification to cells beyond those that protect cells from HIV infection could give a competitive advantage to the survival of the cells in HIV patients, leading to enhanced engraftment of the cells and increase the potential for a cure. This approach could also significantly reduce the therapies needed, potentially allowing the procedure to be done on an outpatient basis.

The results from in vitro and in vivo pre-clinical studies being conducted by Dr. Gallays laboratory and Scripps Institute and Enochians lab have thus far exceeded expectations. Therefore, an Initial Targeted Engagement for Regulatory Advice on CBER ProducTs (INTERACT) meeting package, which we expect will assist us in initiating the Investigational New Drug (IND) process with the FDA, is being prepared. We are seeking to potentially submit that package in December, meeting our original goal of submission before the end of 2019.

HV-11 and HV-12: A Potential Preventive and Therapeutic Vaccine for HIV

Novel approaches to stimulate a persons immune response to more effectively respond to HIV could be used to prevent infection (preventive vaccine) or to allow an HIV-infected person to control HIV infection in the absence of any other treatment (therapeutic vaccine).

Enochian has partnered with one of the leading scientists and scientific institutions, Dr. Hans Peter Kiem, MD PhD, of the Fred Hutchinson Cancer Research Center, to test an innovative approach in non-human primates that we believe has the potential for development into a preventive and/or therapeutic/curative vaccine. Dr. Kiem said, We are very excited to be involved with this study. Dr. Gmrkcs scientific insight for the work is truly unique. I know of no similar approach being evaluated anywhere else in the world.

HIV 31 and 32: Potential new class of HIV treatment

Based on the innovative mechanism of action designed to be developed into a potential cure for HBV, Dr. Gmrkcs two novel approaches aimed at an alternative path to cure and/or treat HIV are in the discovery phase.

Oncology

Building on learning from peer-reviewed publications of Phase I/IIa trials, we are designing an innovative dendritic-cell based therapeutic vaccination platform that could potentially be developed into treatments to induce life-long remissions from some of the deadliest solid tumors. We plan to initially target pancreatic cancer, triple negative breast cancer, glioblastoma, and renal cell carcinoma with this platform. The platform might also allow for non-specific immune enhancement that could have the potential for impact against a broad array of solid tumors. As with HIV, our approach could potentially allow for outpatient therapy without ablating or significantly impairing the patients immune system, as many current approaches require.

Experimental designs have been created to develop our approach, including the procurement of vectors. We expect that the platform could move beyond the discovery phase in the coming months.

Building the Capital Foundation to Advance the Pipeline

Enochian has recently entered into agreements that upon satisfaction of certain closing conditions and closing will provide an additional $12 million in funding. Based on current projections, we believe these resources should be sufficient to advance the pipeline through pre-clinical phases into Phase I trials if IND approvals are secured.

The Inventor

After receiving his preclinical training at the Dokuz Eylul University in 2004, Dr. Serhat Gmrkc continued his training in various institutions and started working as a physician licensed by the Ministry of Health of Turkey in 2008. Later, after working under Dr. Suat Arusan in Turkey, Dr. Gmrkc decided to explore further studies in the field of cell and gene therapies. He is currently the director of Seraph Research Institute, a non-profit organization where he runs a research lab at the cutting edge of cell, gene and immunotherapy research. On agreeing to join the Institutes scientific board, one of the worlds leading experts and his mentor in immuno-oncology, Prof. Shimon Slavin said, Dr. Gmrkc was a young Turkish post-doc with interesting ideas when I first met him eight years ago. I am looking forward to our future collaboration in developing and implementing novel cancer treatments at Seraph Research Institute.

His interest in other fields of science, such as physics, has led him to pursue creating cross-disciplinary solutions to complex human diseases as a researcher. Based on his unique research training, Dr. Gmrkc has become a prolific inventor, submitting various patent/provisional patent applications, which include several approaches aimed to treat or potentially cure HIV/AIDS, Hepatitis B, major solid tumors, rare but deadly diseases, and for a novel vaccine for HIV among many others.

Dr. Gmrkc has licensed intellectual property related to HIV and several solid tumors to Enochian. Dr. Mark Dybul, MD, said, Dr. Gmrkc is one of those rare geniuses that is not bound by scientific discipline or dogma. He sees connections and opportunities often missed. His ideas are the purest kind: those that seem so obvious and simple once he has conceived of, and explained them.

About the Company

Enochian Biosciences is a pre-clinical stage biotechnology company committed to using its exclusive licenses for genetically modified cellular and immune-therapy technologies to seek to prevent or potentially cure HIV, to potentially provide life-long cancer remission of some of the deadliest cancers and to potentially cure HBV. We intend to do this by genetically modifying, or re-engineering, different types of cells, depending on the therapeutic area and then injecting or reinfusing the re-engineered cells back into the patient to provide treatment.

Forward-Looking Statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties, including but not limited to the success or efficacy of our pipeline or the sufficiency of our funding. All statements other than historical facts are forward-looking statements, which can be identified by the use of forward-looking terminology such as believes, plans, expects, aims, intends or similar expressions. Actual events or results may differ materially from those projected in any of such statements due to various uncertainties, including as set forth in Enochians most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and Enochian undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

Originally posted here:
Enochian Biosciences Expands its Infectious Disease Pipeline by Entering into an Agreement in Principle to Acquire an Exclusive License for a Novel...

Natural genetic engineering allowed plants to move from water to land, according to a new study by an international group of scientists from Canada, China, France, Germany and Russia.

This is one of the most important events in the evolution of life on this planetwithout which we as a species would not exist, said University of Alberta genomicist and study co-investigator Gane Ka-Shu Wong.

The movement of life from water to landcalled terrestrializationbegan with plants and was followed by animals and then, of course, humans. This study establishes how that first step took place.

The movement of plants from water to land was made possible when genes from soil bacteria were transferred to algae through a process called horizontal gene transfer. Unlike vertical gene transfer, such as the transfer of DNA from parent to child, horizontal gene transfer occurs between different species.

For hundreds of millions of years, green algae lived in freshwater environments that periodically fell dry, such as small puddles, riverbeds and trickling rocks, explained Michael Melkonian of the University of Duisburg-Essen in Germany. These algae mingled with and received key genes from soil bacteria that helped them and their descendants to cope with the harsh terrestrial environment and eventually evolve into the land plant flora that we see today.

The study is part of an international project focused on sequencing the genomes of more than 10,000 plant species. The discovery was made in the process of sequencing two particular algaeincluding a newly identified species called Spirogloea muscicola.

The approach that we used, phylogenomics, is a powerful method to pinpoint the underlying molecular mechanism of evolutionary novelty, said Shifeng Cheng, first author and principal investigator from the Agricultural Genome Institute at Shenzhen at the Chinese Academy of Agricultural Sciences.

The study, "Genomes of Subaerial Zygnematophyceae Provide Insights Into Land Plant Evolution, was published in Cell.

Read this article:
Genes borrowed from bacteria allowed plants to move from sea to land - Folio - University of Alberta

Opposition to genetically modified (GM) crops advanced by organic activist groups (and official organizations like the US National Organic Standards Board (NOSB) or the EUs European Court of Justice) is based on the claim that recombinant DNA technology introduces genes from one species into another. Thats not natural, these critics contend.

By this definition, though, gene-editing techniques like CRISPR/Cas9 are natural: Theyre part of the immune system in many species of bacteria. Scientists are now using these tools to make specific changes (or edits) to the DNA of food crops and animals to boost their nutritional content or protect them from disease, without adding foreign genes to their genomes.

Therefore, CRISPR-enhanced plants and animals could be utilized by organic growers and ranchers, right? So far, the answer is nobut some dissension in the ranks is starting to appear. While the organic industry generally remains opposed to all forms of genetic engineering, the sustainability benefits of gene-editing techniques like CRISPR have convinced several high-profile organic farmers to come out in support of the technology. Their opposition to the prevailing wisdom espoused by the NOSB suggests that organic agriculture could slowly begin to abandon its hard-line prohibition on biotechnology.

Not needed or wanted in organic agriculture

When CRISPR-Cas9 was introduced as a faster, easier way to edit genetic sequences (other techniques like TALENS and ZFN have been around but are more cumbersome), supporters of the technique in agriculture touted it as a way around organic farmings rules about foreign-ness. And many NGOs took a lets take a closer look approach, not immediately condemning the technique.

However, things changed after the US Department of Agriculture (USDA) in 2018 declared that:

USDA does not regulate or have any plans to regulate plants that could otherwise have been developed through traditional breeding techniques as long as they are not plant pests or developed using plant pests. This includes a set of new techniques that are increasingly being used by plant breeders to produce new plant varieties that are indistinguishable from those developed through traditional breeding methods. The newest of these methods, such as genome editing, expand traditional plant breeding tools because they can introduce new plant traits more quickly and precisely, potentially saving years or even decades in bringing needed new varieties to farmers.

Earlier this year, USDA Under Secretary of Agriculture Greg Ibach testified on Capitol Hill that organic growers could benefit from this development as well:

I think there is the opportunity to open the discussion to consider whether it is appropriate for some of these new technologies, that include gene editing, to be eligible to be used to enhance organic production.

These two statements didnt sit well with pro-organic groups. The Cornucopia Institute, a Wisconsin-based organic advocacy outfit, has been organizing a petition drive among similar groups in opposition to allowing any type of genetic modification in food. On its website, the Institute stated:

organic seed promotes biodiversity, democratizes collective resources, celebrates seed quality over quantity, and preserves agrarian tradition. GMO seeds are not needed or wanted in organic agriculture. In a 2017 survey conducted by Natural Grocers, 70% of respondents said they buy organic to avoid GMOs. Although advocates of GMOs claim that these crops will help farmers respond more quickly to environmental and pest threats, it takes years of testing to ensure the crops will perform as expected.

They have some support. The Organic Consumers Association, a trade group representing thousands of organic food retailers, asked its members to sign a letter asking the National Organic Standards Board to reject all forms of genetic engineering, and to continually update the NOSBs definition of excluded methods to keep up with the new forms of genetic engineering.

The NOSB in October voted against adopting gene editing (mutagenesis via intro methods), and previous decisions (most recently April 2019) have specifically excluded CRISPR, ZFN, TALENS and other gene-editing technologies from the organic designation.

Opposition in the ranks

But the traditional arguments put forth by organic, anti-GM NGOs are falling flat among some organically inclined farmers and scientists. Klaas Martens, an organic farmer of 1,600 acres of grains and vegetables in New York (and a supplier of Dan Barbers Blue Hill restaurant and Row 7 seed companywho was the subject of a recent New York Times op-ed), told attendees of the 2018 CRISPRcon gene-editing convention that he wouldnt have a problem with using gene editing, as long as the crops mimicked naturally occurring varieties. He told the New Food Economy:

If its used in the same way that current products are, then I wouldnt have any interest. (comparing gene-edited crops to Roundup-ready crops, which are genetically spliced with plant and bacterial DNA to resist herbicides) If it could be used in a way that enhanced the natural system, and mimicked it, then I would want to use it. But it would definitely have to be case by case.

Earlier, in 2017, Urs Niggli, director of the Research Institute of Organic Agriculture, told Greenpeace Magazine:

New techniques are currently revolutionizing genetic research. They allow extremely precise changes to the genome. This so-called genetic surgery changes the debate about the risks and chances of interventions in the genome.

For farmers including organic farmers the new method offers many opportunities: plans could be bred that better adapt to difficult environmental conditions such as drought, ground wetness or salinization. The fine root architecture could be improved so that roots absorb more nutrients such as phosphorous or nitrogen from the soil. Tolerance of resistance to diseases and pests, as well as storage and quality of food and feed could also be improved.

Scientists believe that the small changes made by CRISPR/Cas to the plants own genes, which are indistinguishable from a spontaneous or natural mutation, pose not risks. The situation is different when the method introduces foreign genes or when it causes entire populations to be eradicated.

Tom Willey, an organic farmer in California, also supports the organic industrys adoption of gene editing, as part of the effort to restore biodiversity. He told University of California, Berkeley postdoctoral scholar Rebecca Mackelprang:

I see circumstances under which it could be useful for short-cutting a process that for traditional breeding might take many plant generations.

Gene editing, then, could:

Reach back into genomes of the wild ancestors of crop species to recapture genetic materiallost due to breeding for other traits, mainly higher yields. In the light of the urgency posed by climate change, we might wisely employ CRISPR to accelerate such work.

While many organic advocates argue that adjusting to climate change, drought, salinity and pests can be done without GM (or synthetic chemicals), some organic farmers and industry participants obviously dont share this optimism. Currently, it costs more than $130 million and up to seven years to get a genetically engineered (or edited) crop approved for use in the United States (in Europe, its now essentially impossible thanks to the Green party influence on the EU and EU regulators strict adherence to the precautionary principle). This means that small businesses and academics not attached to large universities or industrial labs are shut out. It also means a host of developed crops that could be used to handle tomorrows challenges are waiting in the lab.

And thats a problem. CRISPR alone has resulted in the creation of a wide range of food thats more nutritious than conventionally (and organically) grown predecessors. These include soybean oil with less trans fat and and more oleic acids, a high-fiber wheat, a type of gluten-free wheat, as well as, as economist Steven Cerier wrote in a recent Genetic Literacy Project article:

Rice, wheat, legumes and several vegetables that have up to 60% more protein than existing varieties. Significantly, the amount of protein is increased at the expense of starch and other carbohydrates, thus increasing the nutritional density of foods made from these crops.

In addition to better nutrition, CRISPR and other techniques can produce these foods with fewer inputs (fertilizer, pesticides, even just plain raw land and water) than conventional and organic foods. CRISPR and other editing techniques are being used to produce crops that are more tolerant to drought, heat, and other symptoms of climate change. Relying solely on organic techniques has not resulted in any of these innovations.

Andrew Porterfield is a writer and editor, and has worked with numerous academic institutions, companies and non-profits in the life sciences.BIO. Follow him on Twitter@AMPorterfield

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Will CRISPR's promise force the organic industry to reconsider its opposition to gene-edited crops? - Genetic Literacy Project

SAN DIEGO--(BUSINESS WIRE)--CB Therapeutics is a leading synthetic biotechnology company focused on the research and development of cellular agriculture for the production of high-value molecules, compounds and rare ingredients. CB Therapeutics has been awarded a patent on Oct 10, 2019, titled Isolated codon optimized Nucleic Acids. This patent covers inventions related to the optimization of the entire biosynthetic cannabinoid pathway, from precursors to the final cannabinoid product.

We are excited about this patent award, as it covers the fundamentals of using genetic engineering tools for the production of cannabinoids in many different types of hosts and systems, ranging from bacteria, yeast, to cell-free expression systems, said Dr. Jacob Vogan, CSO of CB Therapeutics.

The patent allows CB Therapeutics to effectively use genetic engineering techniques and tools to enable high yield production of cannabinoids and their analogs while supporting the ability to integrate their platform into many hosts and systems seamlessly, with specific protocols for such techniques. The patent will also allow for the precise control of individual levels of cannabinoids and terpenes in the final product, ensuring the highest levels of quality and specificity for the needs of customers developing cannabinoid products and therapeutics. This cellular agriculture or fermentative biosynthesis technology replaces unsustainable agricultural techniques to grow hemp or cannabis and inefficient extraction processes to isolate CBD and other rare cannabinoids.

Our team of talented scientists and engineers are dedicated to developing new methods and systems to advance synthetic genomics and bio-engineering. Their serious dedication to innovative science is beginning to pay significant dividends for the advancement of our platform. This has resulted in a rapidly growing IP portfolio as we continue to file patent applications directed towards product recovery, optimization techniques to increase yields and other methods used to improve the overall production process, said Sher Butt, CEO of CB Therapeutics.

About CB Therapeutics

CB Therapeutics is currently producing high value molecules, compounds and rare ingredients from simple sugars utilizing yeast and the process of fermentation. CB Therapeutics expertise in synthetic genomics and bio-engineering has significantly advanced its proprietary production platform of yeasts, enzymes and production processes. After more than four years of research and development, the CB Therapeutics team is able to produce a broad range of phytochemicals faster, utilizing fewer resources, at greater yields and with more purity, consistency and efficiency than competing platforms. A 7000+ sf fully-licensed commercial batch facility in San Diego County, California includes research labs, offices and a production facility for laboratory scale and pilot production runs and is equipped with a suite of bench-top and large-scale fermenters for multiple biosynthetic production applications.

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CB Therapeutics Continues to Advance its Intellectual Property Portfolio with New Patent Awards - Business Wire

After a super-fast DNA test developed by scientists at Imperial College, Helen Rumbelow trialled their new gadget it lets people choose food to suit their genes

The Times,November 12 2019, 12:01am

Will it be my grandmothers cancer, or the family weakness for Alzheimers that will get me in the end? Our genes contain instructions for our death as well as our life, but they have always played dumb. Until now.

Now I can wear a wristband with my genetic vulnerability for fatal diseases coded into it. Which is by turns futuristic and kind of terrifying. For me, its like shaking hands with my heart attack scheduled for 2050: Nice to get to know you at last!

Weird, but I soon get used to it when I take the wristband shopping. Its the opposite experience to taking a toddler, endlessly pestering for sweeties, to the supermarket. When I aim the tiny scanner of the DNA Nudge wristband

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DNA Nudge app review: can this wristband tell you the best diet for your genes? - The Times

Doomsday is a beast everyone should fear. It is the creature that was able to kill the Man of Steel himself, Superman. Storm Collectibles is bringing this creature alive yet again with their newestInjustice 2figure it. This figure will be 1/12 scale and will come in at a whopping 10 inches tall. He is completely detailed and ready for action. He does come with two separate had portraits as well as three pairs of hands. He is decked out in spikes in the sporting some delightful green shorts. This figure looks quite articulated and it would go perfect with the otherInjustice 2figures already released. This Doomsday figure is a must-have for any Injusticefan or fans of the Kryptonian killing machine, Doomsday.

TheInjustice 21/12 scale Doomsday figure from Storm Collectibles will be priced at $95. This monster is set to roam the earth yet again in the first quarter of 2020. Pre-orders for him are already live and you can find him here. Dont forget to check out the otherInjustice 2figures already released from Storm Collectibles including Darkseid, Bane, and Lobo.

Superman. Im here to kill you. Is this a bad time?

DOOMSDAY is a rampaging, seemingly mindless, murdering monster who killed Superman. He is the result of Kryptonian genetic engineering gone awry. In The Death of Superman comic storyline in which he first appeared, Doomsday mysteriously bashed his way out of a metallic holding cell miles underground, dug his way up, and began senselessly killing and destroying everything he saw. His motives were initially unknown, but his nature was obvious; He was incredibly powerful, merciless, and seemingly unstoppable. He easily defeated the justice league before confronting Superman.

Features:

Release Date: Q1 2020

loves Marvel and the MCU. Has all of the Funko Pops for the MCU. He loves Tobey Spider-Man and insists The Phantom Menace is pure gold. He is still recovering from the aftermath of Avengers Endgame.

Continued here:
Doomsday Has Arrived with the New Storm Collectibles Figure - Bleeding Cool News

Bioethicist Jacob M. Appel wants the bioethics movement to educate your children about the policy and personal conundrums that involve medical care and health public policy. He claims that most of us give little thought to issues that may arise, such as end-of-life care and prenatal screening. Then, when an issue does come up, people are unprepared to make wise and informed decisions.

From, The Silent Crisis of Bioethics Illiteracy, published in Scientific American:

Change will only occur when bioethics is broadly incorporated into school curricula [at an early age] and when our nations thought leaders begin to place emphasis on the importance of reflecting meaningfully in advance upon these issues

Often merely recognizing such issues in advance is winning the greater part of the battle. Just as we teach calculus and poetry while recognizing that most students are unlikely to become mathematicians or bards, bioethics education offers a versatile skill set that can be applied to issues well outside the scientific arena. At present, bioethics is taught sporadically at various levels, but not with frequency, and even obtaining comprehensive data on its prevalence is daunting.

Is this really an appropriate field for children? Consider the issues with which bioethics grapples and whether elementary-, middle-, and high-school children have the maturity to grapple with them in a meaningful and deliberative way (not to mention, the acute potential that teachers will push their students in particular ideological directions):

Even if some students are mature enough to grapple with these issues thoughtfully, the next problem is that bioethics is extremely contentious and wholly subjective. Its not science, but focuses on questions of philosophy, morality, ideology, religion, etc.. Moreover, there is a dominant point-of-view among the most prominent voices in the field e.g., those who teach at leading universities and would presumably be tasked with writing the educational texts. These perspectives would unquestionably often stand in opposition to the moral values taught young students by their parents.

Appel is typical of the genus (if you will). He has called for paying women who plan to abort to gestate longer in their pregnancy so that more dead fetuses will be available sufficiently developed to be harvested for organs and used in experiments. He advocates mandatory termination of care for patients who are diagnosed as persistently unconscious to save resources for what he considers more important uses. He has also supported assisted suicide for the mentally ill.

Indeed, activists without a modifier like Catholic or pro-life before the term bioethicist are overwhelmingly very liberal politically and intensely secular in their approach. Most support an almost unlimited right to abortion, the legalization of assisted suicide, genetic engineering (once safe), and accept distinguishing between human beings and persons, that is, they deny universal human equality.

Some wish to repeal the dead donor rule that requires organ donors to be dead before their body parts are extracted an idea that admittedly remains somewhat controversial in the field. Most mainstream bioethicists deny the sanctity of human life and many think that an animal with a greater cognitive capacity has greater value than a human being with lower cognition. Add in the sectors general utilitarianish approach to health-care issues, such as supporting rationing, and the potential for propagandizing becomes clear.

With such opinions, often passionately held, how long would it be before early bioethics education devolved into rank proselytizing? But Wesley, Appel might say, the classes would be objective! Every side would be given equal and a respectful and accurate presentation.

Sure. If you believe that, you must think current sex education curricula and high school classes in social justice present all sides of those issues dispassionately and without attempt to persuade the students to particular points of view and cultural perspectives.

I have a deal for Appel: In-depth courses in bioethics should not be taught before college unless I get to write the textbooks! I promise to be objective and fairly present all sides. Honest!

Do you think he and his mainstream colleagues would approve of that deal?

Neither do I. And we shouldnt go along with his idea for the very same reason.

Photo credit:cherylt23 viaPixabay.

Cross-posted at The Corner.

Read more:
Bioethics Coming to Elementary and High Schools? - Discovery Institute

What happened

Shares of Precision BioSciences (NASDAQ:DTIL) rose as much as 19.5% today, as the company continues to benefit from its pipeline update that took place on Nov. 6. The gene-editing company released initial clinical data from its first drug candidate, PBCAR0191, demonstrating its potential to treat cancers of white blood cells, such as non-Hodgkin lymphoma (NHL).

Two of the three patients in the low-dose cohort achieved an objective tumor response, while the third showed signs of anti-tumor activity. That's about as good as the company could have hoped for in the early stages of development. It's also reminding investors that CRISPR gene editing isn't the only game in town -- and that Precision BioSciences' specific approach could prove valuable in engineering immunotherapies.

As of 1:40 p.m. EST, the stock had settled to a 16.9% gain. The gene-editing stock is up 70% since the beginning of November.

Image source: Getty Images.

Precision BioSciences is developing the ARCUS gene-editing platform, which offers several potential advantages over the more familiar CRISPR-Cas9 tools. The cutting enzymes are smaller, which makes it easier to deliver the genetic medicines. The enzymes don't make a double-stranded break in DNA, which could improve safety. It has also proven to be more accurate and more efficient at making genetic edits. And it's not subject to the bitter patent disputes and tangled web of licensing agreements affecting CRISPR tools.

In addition to those advantages, Precision BioSciences has wisely chosen to focus initially on engineering immunotherapies, rather than on directly editing cells in a patient's body. That gives researchers more control over the edits being made, and allows for a more homogeneous product.

The lead drug candidate, PBCAR0191, applies the ARCUS gene-editing platform to chimeric antigen receptor T cells. The idea is to engineer CAR-T cells to overcome their single largest limitation: strict patient matching requirements. By editing the surface of CAR-T cells, it may be possible to manufacture a product that can be used by any patient. The "off-the-shelf" approach could be combined with other genetic edits to make immunotherapy, already a promising treatment paradigm, the preferred treatment option for white-blood-cell cancers in the long run.

Investors shouldn't get too carried away; Precision BioSciences has only reported limited data from just three patients. It plans on presenting additional results from the ongoing phase 1/2 trial on Dec. 9 at the Annual Meeting of the American Society of Hematology (ASH), including results from the same three patients in the low-dose cohort and patients in the cohort with the next highest dose.

But that doesn't mean the current rally is unsustainable. Considering the gene-editing company had a market cap of just $325 million at the beginning of November, the stock's ascension could simply be a sign of investors nodding to the potential of the ARCUS platform.

Continue reading here:
Here's Why Precision BioSciences Is Soaring Today - The Motley Fool

Growing up in Tanzania, I knew that fruit trees were useful. Climbing a mango tree to pick a fruit was a common thing to do when I was hungry, even though at times there were unintended consequences. My failure to resist consuming unripened fruit, for example, caused my stomach to hurt. With such incidents becoming frequent, it was helpful to learn from my mother that consuming the leaves of a particular plant helped alleviate my stomach pain.

This lesson helped me appreciate the medicinal value of plants. However, I also witnessed my family and neighboring farmers clearing the land by slashing and burning unwanted trees and shrubs, seemingly unaware of their medicinal value, to create space for food crops.

But this lack of appreciation for the medicinal value of plants extends beyond my childhood community. As fires continue to burn in the Amazon and land is cleared for agriculture, most of the concerns have focused on the drop in global oxygen production if swaths of the forests disappear. But Im also worried about the loss of potential medicines that are plentiful in forests and have not yet been discovered. Plants and humans also share many genes, so it may be possible to test various medicines in plants, providing a new strategy for drug testing.

As a plant physiologist, I am interested in plant biodiversity because of the potential to develop more resilient and nutritious crops. I am also interested in plant biodiversity because of its contribution to human health. About 80% of the world population relies on compounds derived from plants for medicines to treat various ailments, such as malaria and cancer, and to suppress pain.

One of the greatest challenges in fighting diseases is the emergence of drug resistance that renders treatment ineffective. Physicians have observed drug resistance in the fight against malaria, cancer, tuberculosis and fungal infections. It is likely that drug resistance will emerge with other diseases, forcing researchers to find new medicines.

Plants are a rich source of new and diverse compounds that may prove to have medicinal properties or serve as building blocks for new drugs. And, as tropical rainforests are the largest reservoir of diverse species of plants, preserving biodiversity in tropical forests is important to ensure the supply of medicines of the future.

The goal of my own research is to understand how plants control the production of biochemical compounds called sterols. Humans produce one sterol, called cholesterol, which has functions including formation of testosterone and progesterone - hormones essential for normal body function. By contrast, plants produce a diverse array of sterols, including sitosterol, stigmasterol, campesterol, and cholesterol. These sterols are used for plant growth and defense against stress but also serve as precursors to medicinal compounds such as those found in the Indian Ayurvedic medicinal plant, ashwagandha.

Humans produce cholesterol through a string of genes, and some of these genes produce proteins that are the target of medicines for treating high cholesterol. Plants also use this collection of genes to make their sterols. In fact, the sterol production systems in plants and humans are so similar that medicines used to treat high cholesterol in people also block sterol production in plant cells.

I am fascinated by the similarities between how humans and plants manufacture sterols, because identifying new medicines that block sterol production in plants might lead to medicines to treat high cholesterol in humans.

An example of a gene with medical implications that is present in both plants and humans is NPC1, which controls the transport of cholesterol. However, the protein made by the NPC1 gene is also the doorway through which the Ebola virus infects cells. Since plants contain NPC1 genes, they represent potential systems for developing and testing new medicines to block Ebola.

This will involve identifying new chemical compounds that interfere with plant NPC1. This can be done by extracting chemical compounds from plants and testing whether they can effectively prevent the Ebola virus from infecting cells.

There are many conditions that might benefit from plant research, including high cholesterol, cancer and even infectious diseases such as Ebola, all of which have significant global impact. To treat high cholesterol, medicines called statins are used. Statins may also help to fight cancer. However, not all patients tolerate statins, which means that alternative therapies must be developed.

The need for new medicines to combat heart disease and cancer is dire. A rich and diverse source of chemicals can be found in natural plant products. With knowledge of genes and enzymes that make medicinal compounds in native plant species, scientists can apply genetic engineering approaches to increase their production in a sustainable manner.

Tropical rainforests house vast biodiversity of plants, but this diversity faces significant threat from human activity.

To help students in my genetics and biotechnology class appreciate the value of plants in medical research, I refer to findings from my research on plant sterols. My goal is to help them recognize that many cellular processes are similar between plants and humans. My hope is that, by learning that plants and animals share similar genes and metabolic pathways with health implications, my students will value plants as a source of medicines and become advocates for preservation of plant biodiversity.

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Across the US, more than 100,000 people are awaiting organ transplants. But there simply arent enough hearts, lungs, livers, and kidneys to meet demand, and 20 people die every day without the organs they need. For decades, scientists have dreamed of using animals to help fill the gap. Theyve been particularly interested in harvesting organs from pigs, whose physiology is similar to our own. Unfortunately, pigs also present some big biological challenges, including the fact that their genomes are chock full of genes that code for what are known as retroviruses, which could pose a serious threat to patients who receive porcine organs.

In 2015, George Church, a geneticist at Harvard University, announced a stunning breakthrough: Working with pig cells, he and his colleagues had managed to disable 62 copies of a retrovirus gene in one fell swoop. This would have been virtually impossible and a logistical nightmare with older forms of genetic modification, writes Nessa Carey in her new book, Hacking the Code of Life: How Gene Editing Will Rewrite Our Futures. But by using the new gene editing technology known as Crispr, the task was a relative cinch.

Nessa CareyHacking the Code of LifeIcon Books

Its just one example of how gene editing is giving us the power to alter the genome with unprecedented speed and precision. Carey, a biologist with a background in the biotech and pharmaceutical industry, offersa brisk, accessible primer on the fast-moving field, a clear-eyed look at a technology that is already driving major scientific advances and raising complex ethical questions

Its giving every biologist in the world the tools to answer in a few months questions that some scientists have spent half their careers trying to address, Carey writes. Its fueling new ways to tackle problems in fields as diverse as agriculture and cancer treatments. Its a story that began with curiosity, accelerated with ambition, will make some individuals and institutions extraordinarily wealthy, and will touch all our lives.

Though there are several different approaches to gene editing, the most prominent and the one that really supercharged the field is Crispr. The technique, based on an anti-viral defence system thats naturally present in bacteria, requires two pieces of biological material: an enzyme that acts as a pair of minuscule scissors, slicing strands of DNA in two; and a guide molecule that tells the enzyme where to cut.

In bacteria, these guide molecules direct the enzyme to chop up the genomes of invading viruses, preventing them from replicating.

But in 2012 and 2013, two teams of scientists reported that it was possible to hack this system to slice into any strand of DNA, at any complementary location they chose. Researchers could, for instance, create a guide molecule that steered the enzyme to one specific gene in the mouse genome and insert the editing machinery into a mouse cell; the enzyme would then make its cut at that exact spot.

Also Read: Is There More to Gene Editing Than Creating Designer Humans?

The cell would repair the severed DNA, but it would do so imperfectly, disabling the gene in question. In the years that followed, scientists refined the technique, learning to use it not only to inactivate genes but also to insert new genetic material at specific locations along the genome.

The approach is cheaper, easier, and faster than older methods of genetic engineering, which were first developed in the 1970s. In addition, as Carey explains, it can be used to create smaller modifications to the genome, and leaves fewer extraneous genetic elements. In its most technically exquisite form, gene editing leaves no molecular trace at all. It may just change, in a precisely controlled manner, one letter of the genetic alphabet.

But in 2012 and 2013, two teams of scientists reported that it was possible to slice into any strand of DNA. Photo: qimono/pixabay

The applications are almost endless. Gene editing has immense potential for basic research; scientists can learn a lot about what genes do by selectively disabling them. In addition, researchers have used the technology to create a wide variety of organisms that could become valuable agricultural commodities, including mushrooms that dont brown; wheat that produces fewer gluten proteins; drought tolerant, high-yield rice and corn; disease-resistant pigs; and super muscular goats.

How these products will do on the market if they ever reach it remains uncertain. Globally, gene-edited organisms are regulated by a patchwork of conflicting rules. For instance, in 2018, the US Department of Agriculture announced that it would not regulate gene-edited crops that could otherwise have been developed through traditional breeding techniques. A few months later, however, the European Union said that it would subject gene-edited plants to stringent restrictions.

Beyond agriculture, gene editing has enormous potential for medicine. It might, for instance, become a much-needed treatment for sickle cell disease. That painful, debilitating disease results from a genetic mutation that causes patients to produce a deformed version of haemoglobin, a protein that helps red blood cells transport oxygen. In a clinical trial currently underway, scientists are removing stem cells from the bone marrow of sickle cell patients, using Crispr to edit them, and then infusing the edited cells back into patients.

Also Read: Explainer: What Is CRISPR and How Does It Work?

Even if this trial succeeds, however, gene editing will not be a cure-all. It doesnt always work perfectly and can be challenging to administer directly to living humans (which is why some scientists are instead editing patients cells outside the body). Moreover, many diseases are caused by complex interactions between multiple genes, or genes and the environment. In fact, many of the most common and debilitating conditions arent likely to be good candidates for gene editing any time soon, Carey writes.

And, of course, the ethics of human gene editing can be enormously fraught. Thats especially true when scientists modify sperm cells, egg cells, or early embryos, making tweaks that could be passed down to subsequent generations. This kind of gene editing could theoretically cure some absolutely devastating genetic conditions, but we still have a lot to learn about its safety and effectiveness. It also raises a host of difficult questions about consent (an embryo obviously cannot give it), inequality (who will have access to the technology?), and discrimination (what will the ability to edit a gene related to deafness mean for deaf people, deaf culture, and the disability rights movement more broadly?).

Even in the face of these questions, at least one scientist has already forged ahead. In November 2018, He Jiankui, a researcher then at the Southern University of Science and Technology in China, shocked the world by announcing that the worlds first gene-edited babies twin girls, who He called Nana and Lulu had already been born. Months earlier, when Nana and Lulu were just embryos, He had edited their CCR5 genes, which code for a protein that allows HIV to infect human cells. By disabling the gene, He hoped to engineer humans who would be protected from HIV infection.

Also Read: How a Rogue Chinese Experiment Might Affect Gene-Based Therapies in India

The outcry was swift and harsh. Scientists alleged that Hes science was sloppy and unethical, putting two human beings at unnecessary risk. After all, there are already plenty of ways to prevent HIV transmission, and the CCR5 protein is known to have some benefits, including protecting against the flu. And He had raced ahead of the experts who were still trying to work out careful ethical guidelines for editing human embryos. He Jiankui has shot this measured approach to pieces with his announcement, and now the rest of the scientific community is on the back foot, trying to reassure the public and to create consensus rapidly, Carey writes.

Scientist He Jiankui attends the International Summit on Human Genome Editing at the University of Hong Kong on November 28, 2018. Photo: REUTERS/Stringer/File Photo

Hacking the Code of Lifedoesnt break much new ground, and for readers who have been paying attention to Crispr over the past few years, little in the book will come as a surprise. But it does provide a broad, even-handed overview of how much has already happened in a field that is less than ten years old.

Carey swats down the most dystopian dreams about Crispr, like the prospect that criminals might edit their own DNA to evade justice. Shes similarly skeptical that well end up using the technology to create super-beings with enhanced genomes that will make them taller, faster, more attractive.

We actually understand very little about the genetic basis of these traits and what we do know suggests that it will be very difficult to enhance humans in this way, she writes.

But she also acknowledges real risks, including the possibility that the technique could be used to create dangerous bioweapons, that gene-edited organisms could destabilise natural ecosystems, and that our new, hardy crops could prompt us to convert even more of the Earths undeveloped places into farmland.

None of this means that the technology should be abandoned; it has immense potential to improve our lives, as the book makes clear. But it does mean we need to proceed with caution. As Carey writes, Ideally, ethics should not be dragged along in the wake of scientific advances; the two should progress together, informing one another.

Emily Anthes, who has written for Undark, The New York Times, The New Yorker, Wired, and Scientific American, among other publications, is the author of the forthcoming book The Great Indoors.

This article was originally published on Undark. Read the original article.

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How Gene Editing Is Changing the World - The Wire