Category Archives: Genetic Engineering

Over the past decade there has been a small cottage industry of published books that address the ethical issues arising from new developments in biotechnology. They cover genetically modified food, transgenic animals, biological weapons, and a subject that accounts for the most volumes, the genetic modification of human beings. As a matter of historical interest, the ethical discussion around creating an uber mensch or in the contemporary jargon, a trans-human or genetically enhanced person, preceded the genetic engineering revolution of the early 1970s. French biologist Jean Rostand's book, Can Man be Modified? was translated into English in 1959.[1] Without knowing anything about cloning or stem cells, Rostand wrote:

If a biologist takes any fragment of tissue from the freshly dead body there is no absolute reason why we should not imagine the perfect science of the future remaking from such a culture, the complete person, strictly identical to the one who had furnished the principle.[2]

Rostand was speculating about the powers of science 50 years into the future and inquiring about the ethical conundrums they would create. Now we are there. In many respects, the science is still ahead of the ethics. Take, for example, the decision of the U.S. Patent & Trademark Office (USPTO) to deny a patent for the production of a chimera by cell fusion that combines the chromosomes of a human and a non-human animal into a viable embryo. The USPTO denied the patent on the grounds that the hybrid organism was too similar to a human being. Even though the chimera had not been created, to be seriously considered, the patent design had to have been sufficiently persuasive so that anyone familiar with the art of making animal chimeras would be able to make the human-animal embryo. While human beings cannot be patented, the processes used to genetically modify humans can be and have been patented. Whether and how those processes should be used is the subject of The Ethics of Genetic Engineering.

Roberta M. Berry has written a creative book on how ethics can inform individual decisions and social policy on human genetic engineering. The book focuses primarily on germline genetic modification. The discussions and analyses are largely aimed at prospective parents who wish to bring into the world a "more perfect" child, not by education or through nurturance of the child's creativity, but by engineering the child's genomes at the point of gestation. Perfection means being more resistant to disease or having other phenotypes that provide more than average advantages in society. Berry applies her skills as a trained philosopher to analyze issues that have been discussed largely by bioethicists in think tanks, university seminars, and professional journals, as well as among NGOs seeking to warn society about the new science of reproductive genetics looming on the horizon.

Whimsically, we might speak about this book as a philosopher's guide to Gattaca, the 1997 film about a future when genetic engineering makes possible the creation of biologically superior humans (known as "valids"), who enter positions of power and prestige.

When the human genome project got underway in the early 1990s and personalized genetics was seen as the next medical frontier, scientists and many bioethicists constructed an ethical firewall between somatic cell and germline gene therapy. The former was seen as a practical extension of drug therapy, although in this case the therapeutics is in the form of genetic materials that are delivered into the patient's cells. Germline gene therapy (or enhancement) was connected to eugenics because it involved planned genetic changes to future generations. This was a short-lived distinction as scientists broke away from any constraints on research. Berry's book, which assumes that germline gene enhancement of humans will eventually take place, prepares readers for new personal and societal choices that will be available to prospective parents. Beyond that the book offers readers a useful exegesis in practical ethics.

The book is divided into five chapters. Chapter 1 offers a broad brushstroke look into the early developments of genetic engineering leading to what the author refers to as "fractious problems", complex and divisive ethical problems resulting from breakthroughs in biomedical science. Chapters 2, 3 and 4 explore how the classical philosophies (utilitarianism, Kantianism, and virtue ethics) provide clarity and wisdom to the ethical choices associated with human germline genetic engineering or the genetic selection of embryos. In Chapter 5 the author explores the viewpoints of modern philosophers including John Rawls, Robert Nozick, Alasdair MacIntyre, Charles Taylor and Ronald Dworkin on changing the genetic architecture of humans. Berry also argues the case that virtue ethics provides the best framework for addressing the issues. "But it does not follow that a utilitarian calculus of welfare maximization or a deontological assessment of duties or rights is well-suited to parental or policy decision-making about revising the genomes of our future children."[3] When science is capable of circumventing the genetic lottery of biological meiosis between sperm and egg, we are faced with new personal and normative reproductive decisions, which become the focus of the book.

There are two qualities that distinguish this book from the pack. First, the author has a richer understanding of ethical theory than most writing in the field of "genome ethics." She uses a broad tapestry of ethical theories as lenses for analyzing problems. And second, Berry applies a creative form of dialogue between one person who personifies a physician, bioethicist and/or genetics counselor and two other individuals who are prospective parents. The dialogues are reminiscent of the Platonic and Galilean dialogues where different philosophical or scientific perspectives appear in the personages who question each other on issues of great public concern. From a teaching standpoint, Berry's dialogues will be useful in reaching students who may have difficulty in applying ethical theory to contemporary problems.

For example, the Kantian counselor explores the parents' right to do whatever is in their power (including genetic modification) to produce a superior child. The counselor says: "Although [your] purpose would be to gain additional opportunities for [your] children, the result of everyone extending their children's lives in an effort to gain a greater share of available opportunities would remain constant and even diminish over time."[4] Applying Kant's Categorical Imperative, the counselor concludes that the prospective parents cannot, without reaching a contradiction to their goals, universalize the maxim "I should act to genetically modify the ovum of my future child to gain additional opportunities." If, for example, height were the phenotype desired, and everyone was afforded the same opportunity to modify the ovum for greater height, there would be no advantage. While this is good Kantianism, it may not convince most people who wish to exercise every available advantage for their children. There are other considerations that are subsumed to the authority of science, namely, that safe genetic modification of the human genome is a myth that, if attempted, is likely to result in dangerous human pathologies.

The conclusion reached by Berry as to how society will resolve the problems brought on by the expected scientific capacity to engineer the human genome is optimistic but philosophically weak. It is based on the faith that a society which devotes itself to virtue (in education and practical life) will use appropriate forms of casuistry to navigate safely through the bramble bush of ethical conflicts. Berry writes that

Virtue ethics invites us to embrace all [ways of understanding] and it trusts that this will enable us to see not just a booming buzzing confusion, but what practical wisdom requires under all the facts and circumstances so we can be as accomplished at acting from the virtues in making choices about genetic engineering as we are in making choices about other practical problems that we confront in daily life.[5]

Some would argue this is what currently exists as we cross the frontier of genetics and reproductive technology, namely ethical and social anarchism.

[1] Jean Rostand. Can Man be Modified? Translated from the French by Jonathan Griffin. New York: Basic Books, 1959.

[2] Rostand, 1959, pp. 13-14.

[3] Robert M Berry. The Ethics of Genetic Engineering. New York: Routledge, 2007, p. ix.

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The Ethics of Genetic Engineering | Reviews | Notre Dame ...

Every year between 2004 and 2013, swathes of cabbage grown in fields and greenhouses across New York were attacked by a lethal bacteria that severely wilted the leaves, sometimes making the vegetables appear scorched. For over a century, little was known about this untreatable plant epidemic called black rot, which threatens food security worldwide.

But a group of scientists in Singapore has, for the first time, identified how this "crop killer" bacteria hijack plants at the molecular level and cripple their immune systems.

Their findings will pave the way for plant biologists to better treat infected plants and find ways to rear bacteria-resistant crops without using genetic engineering, said the study's lead, Associate Professor Miao Yansong from Nanyang Technological University's (NTU) School of Biological Sciences.

"For some of the devastating disease in agriculture, the whole field has to be burnt," he said.

Prof Miao and his team found that the black rot-causing bacteria, called Xanthomonas, inject toxic proteins into plant cells. The surface of plant cells contains substances that activate an immune response against diseases.But the toxic proteins form a sticky network, adhering to the cell surface and hijacking the plant's defense mechanisms.

Read the complete article at http://www.straitstimes.com.

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Singapore scientists uncover secret of the black rot in vegetable crops - hortidaily.com

New research by scientists at North Carolina State University (NC State) has demonstrated that genes are capable of identifying and responding to coded information in light signals, as well as filtering out some signals entirely. Their study findings showed how a single mechanism can trigger different behaviors from the same gene. The fundamental idea here is that you can encode information in the dynamics of a signal that a gene is receiving, said Albert Keung, PhD, an assistant professor of chemical and biomolecular engineering at NC State. So, rather than a signal simply being present or absent, the way in which the signal is being presented matters.

The researchers say there are practical applications for their work in the pharmaceutical and biotech sectors. In biomanufacturing, you often want to manage both the growth of cells and the rate at which those cells are producing specific proteins, said Jessica Lee, PhD, research assistant at NC State. Our work here can help manufacturers fine-tune and control both of those variables. Lee is first author, and Keung is corresponding author of the teams published paper in Cell Systems, which is titled, Mapping the dynamic transfer functions of eukaryotic gene regulation, and in which they concluded, This work directly demonstrates thesignal processing potential of a single individual gene and develops molecular and computational tools that can be used to harness it.

There is plenty of evidence that biological information can be encoded in the dynamics of signaling components, and not just in their biochemical identities, the authors noted. This has been implicated in a range of physiological processes, such as the stress response, stem cell differentiation, and oncogenesis. Cells, with a limited number of components, utilize dynamic signal processing to perform sophisticated functions in response to complex environments, the researchers stated. Transcription factors (TFs) may be a particularly important archetype for this typeof information transmission, as they are relatively low in diversity but must command many distinct and complex geneexpression programs.

For their reported study, the researchers developed a platform that combined optogenetics and flow cytometry to map the protein expression response to different dynamic inputs. They modified a yeast cell to express a gene that produces fluorescent proteins when the cell is exposed to blue light. The promoter region of the gene is responsible for controlling the genes activity, and in the modified yeast cells, a specific protein binds to the promoter region of the gene. When blue light is shone on that protein, it becomes receptive to a second protein. When the second protein binds to the first protein, the gene becomes active. And thats easy to detect, because the activated gene produces proteins that glow in the dark.

The researchers exposed these yeast cells to 119 different light patterns. Each light pattern differed in terms of the intensity of the light, how long each pulse of light was, and how frequently the pulses occurred. The researchers then mapped out the amount of fluorescent protein that the cells produced in response to each light pattern.

We may tend to think of genes being turned either on or off, butless like a light switch and more like a dimmer switcha gene can be activated a little bit, a lot, or anywhere in between. So, if a given light pattern led to the production of a lot of fluorescent protein, that meant the light pattern made the gene very active. If the light pattern led to the production of just a little fluorescent protein, that meant the pattern only triggered mild activity of the gene.

We found that different light patterns can produce very different outcomes in terms of gene activity, said Lee. The big surprise, to us, was that the output was not directly correlated to the input. Our expectation was that the stronger the signal, the more active the gene would be. But that wasnt necessarily the case. One light pattern might make the gene significantly more active than another light pattern, even if both patterns were exposing the gene to the same amount of light.

The researchers found that all three light pattern variablesintensity of the light, frequency of the light pulses, and how long each pulse lastedcould influence gene activity, but they also found that controlling the frequency of light pulses gave them the most precise control over gene activity.

We also used the experimental data here to develop a computational model that helped us better understand why different patterns produce different levels of gene activity, said Leandra Caywood, co-author of the paper and a PhD student at NC State. For example, we found that when you bunch rapid pulses of light very closely together, you get more gene activity than you would expect from the amount of light being applied. Using the model, we were able to determine that this is happening because the proteins cant separate and come back together quickly enough to respond to every pulse. Basically, the proteins dont have time to fully separate from each other between pulses, so are spending more time connected meaning that the gene is spending more time activated. Understanding these sorts of dynamics is very useful for helping us figure out how to better control gene activity using these signals.

Our finding is relevant for cells that respond to light, such as those found in leaves, Keung added. But it also tells us that genes are responsive to signal patterns, which could be delivered by mechanisms other than light.

So how might this work in cells? A cell may receive a chemical signal. The presence of the chemical cant be patternedits either present or it is not. However, the cell can respond to the presence of the chemical by creating a patterned signal for the target gene. The cell does this by controlling the rate at which the protein that binds to the promoter region enters and exits the nucleus of the cell. We could think of controlling the presence and absence of this protein as sending a Morse code message from the cell to the gene. Depending on a suite of other variablessuch as the presence of other chemicalsthe cell can fine-tune the message it sends to the gene in order to modulate its activity.

This tells us that you can use the same protein to give different messages to the same gene, Keung said. So the cell can use one protein to have a gene respond differently to different chemicals.

In a separate set of experiments, the researchers found that genes were also able to filter out some signals. The mechanics of this are both straightforward and mysterious. The researchers could tell that when a second protein attached to the promoter region of the gene, some frequencies of light pulses did not trigger the production of fluorescent proteins. In short, the researchers know the second protein ensured that a gene responds only to a specific suite of signalsbut they dont know exactly how the second protein accomplishes that.

The researchers also found that they could control the number of distinct signals a gene could respond to by manipulating the number and type of proteins attached to the promoter region of the gene.

For example, you could attach proteins to the promoter region that serve as filters to limit the number of signals that activate the gene. Or you could attach proteins to the promoter region that trigger different degrees of activation of the gene.

One additional contribution of this work is that weve determined we can communicate about 1.71 bits worth of information through the promoter region of a gene with just one protein attachment, Lee said. As the authors explained, This system revealed tunable gene expression and filtering behaviors and provided a quantification of the limit to the amount of information that can be reliably transferred across a single promoter as ~ 1.7 bits. Lee continued, In practical terms that means that the gene, without a complex network of protein attachments, is able to distinguish between more than three signals without error. Previous work had set that baseline at 1.55 bits, so this study advances our understanding of whats possible here. Its a foundation we can build on.

The researchers say their work will enable future studies that help scientists to understand the dynamics of cell behavior and gene expression. This work directly demonstrates the signal processing potential of a single individual gene and develops molecular and computational tools that can be used to harness it, they wrote. There are many avenues to expand into and explore. In our work, we relied on endpoint measurements that could be rapidly measured by flow cytometry. However, information can also be stored in the dynamics of the output signal, e.g., the production rate, time delay of repression/activation, or oscillatory behavior. High throughput approaches that can track the output dynamics of thousands of cultures would unlock this potential space for investigation.

And while the reported study focused on a single promoter, different promoter structures would likely confer distinct transfer functions, the investigators further noted. Continued advances in experimental and computational systems that can handle the large parameter space of dynamic signals will unlock our ability to measure, quantify, and understand information transmission in biological systems and reveal the underpinnings of how limited numbers of components can give rise to the rich complexity of biological functions.

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Findings Show Gene Behavior Depends on Coded Info in Signals and Could be Harnessed to Fine-Tune Biotech - Genetic Engineering & Biotechnology...

OTS 2021 Virtual Conference

OTS 2021 Virtual Conference

SAN DIEGO, Sept. 04, 2021 (GLOBE NEWSWIRE) -- The Oligo Meeting is purposefully designed to bring people together to share incredible advancements in the field of oligonucleotide therapeutics. While still unable to come together in person, OTS leadership believes in the power of sharing science and the dedicated organizing committee has planned a professional, outstanding, and exciting event in which attendees will join leading scientists from around the world.

Last year's virtual meeting was extraordinarily successful, and this year's virtual conference has been carefully planned to be even more seamlessly engaging and productive. All the components of the in-person annual meeting will be included: sessions, short talks, posters, exhibitors, and networking, which will be accessed through one online platform. Recorded talks and posters can be viewed on-demand through December 31, 2021, for all registered delegates. A fun and interactive networking tool will be available throughout the entire four days of the meeting, 24 hours a day.

Session topics feature Nucleic Acid Chemistry, Rare Diseases, 20th Anniversary of Mammalian RNAi, Delivery, Genome and RNA Editing, Bob Letsinger, PhD - 100 Years of History, and the Awards Presentation. The final sessions include two Oligonucleotide Preclinical sessions and finish with the highly anticipated Clinical Studies session.

This year's featured event speakers include an outstanding lineup of leading experts covering a broad range of oligonucleotide-based disciplines.

Stanley T. Crooke, MD, PhD, Founder and CEO of n-Lorem Foundation and founder, former CEO, and Chairman of the Board at Ionis led the scientific development of a new platform for drug discovery: antisense technology. He engineered the creation of one of the largest, more advanced development pipelines in the biotechnology industry.

John Maraganore, PhD, is the CEO and Director of Alnylam Pharmaceuticals, which has led the translation of RNA interference from Nobel Prize-winning discovery into an innovative, entirely new class of medicines.

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Marie Wikstrm Lindholm, PhD, SVP and Head of Molecular Design at Silence Therapeutics built and leads a skilled team at Silence focusing on fine-tuning the design of their proprietary GalNAc-conjugated siRNA technology and exploring siRNA delivery outside the hepatocyte.

Craig Mello, PhD, is a joint winner of the 2006 Nobel Prize in Physiology or Medicine for the discovery of RNA interference. He has been involved in several RNAi-based biotechnology companies and recently co-founded Atalanta Therapeutics.

Kelvin K. Ogilvie, PhD, a leading expert on biotechnology, bioorganic chemistry, and genetic engineering, invented the drug Ganciclovir and developed a general method for the chemical synthesis of large RNA molecules, which is still the basis for RNA synthesis worldwide.

Laura Sepp-Lorenzino, PhD oversees all drug research across in vivo and engineered cell therapy areas as Chief Scientific Officer at Intellia Therapeutics, Inc., a company developing curative genome editing treatments to positively transform the lives of people with genetic diseases.

These are just a few of the many experts that attendees will hear from as they present interesting and cutting-edge topics. Last year's virtual conference received rave reviews from participants and this year's virtual meeting is expected to be even more spectacular.

Those wishing to attend can register here.

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Geri Beaty

Phone: (619)795-9458

Email: info@oligotherapeutics.org

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The Oligonucleotide Therapeutics Society Presents the 2021 Virtual Conference - Yahoo Finance

DBMR has added a new report titled CRISPR Gene-Editing Market with data Tables for historical and forecast years represented with Chats & Graphs spread through Pages with easy to understand detailed analysis. . Report is structured with the meticulous efforts of an innovative, enthusiastic, knowledgeable and experienced team of analysts, researchers, industry experts, and forecasters. This market report also offers an in-depth overview of product specification, technology, product type and production analysis considering major factors such as revenue, cost, and gross margin. Two of these major tools of market analysis are SWOT analysis and Porters Five Forces Analysis. The finest CRISPR Gene-Editing Market report is generated with a nice combination of advanced industry insights, practical solutions, talent solutions and the use of latest technology which gives an excellent user experience.

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Global CRISPR Gene-Editing Market By Therapeutic Application (Oncology, Autoimmune/Inflammatory), Application (Genome Engineering, Disease Models, Functional Genomics and Others), Technology (CRISPR/Cas9, Zinc Finger Nucleases and Others), Services (Design Tools, Plasmid and Vector, Cas9 and g-RNA, Delivery System Products and Others), Products (GenCrispr/Cas9 kits, GenCrispr Cas9 Antibodies, GenCrispr Cas9 Enzymes and Others), End-Users (Biotechnology & Pharmaceutical Companies, Academic & Government Research Institutes, Contract Research Organizations and Others), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2026

Global CRISPR gene-editing market is rising gradually with a healthy CAGR of 23.35 % in the forecast period of 2019-2026. Growing prevalence of cancer worldwide and expanding the application of CRISPR technology by innovative research from the different academic organizations are the key factors for market growth.

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Few of the major competitors currently working in the global CRISPR gene-editing market are Applied StemCell, ACEA BIO, Synthego, Thermo Fisher Scientific Inc, GenScript, Addgene, Merck KGaA, Intellia Therapeutics, Inc, Cellectis, Precision Biosciences, Caribou Biosciences, Inc, Transposagen Biopharmaceuticals, Inc, OriGene Technologies, Inc, Novartis AG, New England Biolabs among others

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Market Drivers

Market Restraints

Complete report is available (TOC) @https://www.databridgemarketresearch.com/toc/?dbmr=global-crispr-gene-editing-market

Key Developments in the Market:

In April 2019, GenScript has launched Single-stranded DNA Service for CRISPR-based Gene Editing which help the key researchers to have access on the high quality, pure ssDNA for CRISPR-based gene insertion and hence can accelerate the development of gene as well as cell therapy for cancer immunotherapy

In February 2018, Cellectis has received two U.S. patents (US#9,855,297 and US#9,890,393) entiled as Methods for engineering T cells for immunotherapy by using RNA-guided CAS nuclease system for CRISPR Use in T-Cells. The U.S. grant of these patents, the company can generate revenue by out-licensing the products to the pharma companies that are ready to use CRISPR technologies in T-cells

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Global CRISPR gene-editing market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of CRISPR gene-editing market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

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CRISPR Gene-Editing Market Report- Growth in Future with Size, Share, Growth, and Key Companies Analysis Eudaemonia - Eudaemonia

Genetic engineering using the new Crispr technology is just hitting the US stock market. This brand new sector has the potential to explode in price and keep on growing for decades.

Most people are aware that the dotcom boom was technology-based, but few realise that since the very start of stock markets in the 1700s, the whole shooting match has been driven by technological advancement. The history of the stock market is the history of funding and the speculation in technology: sail, navigation, canals, railways, radio, automobiles, electronic, computing, the internet and finally the internet applications like Facebook, Google and Amazon.

So the way to invest is to catch new technology at a stage where it is exciting enough to be incorporated into listed enterprises, but not so late as to be a later entrant buying at increasingly lofty valuations.

Some people call this investing, but it is in the end speculation, a less respectful way of typifying someone putting their money down to back an opinion. Some will call it gambling, but the thing to recall about gambling is there are groups of people who make huge sums gambling, namely bookmakers and casinos, and the only difference between them and the customer is that for the house the odds are skewed in their favour. Picking to invest in technology before it breaks into the mainstream is investing with an edge, because skill is the edge in the stock market and spotting technology is certainly a skill.

So I have written about the coming hydrogen economy and why the platinum group metals are a place to invest alongside other producers and IP holders of exotic metals, but one should never stop looking for the next big thing.

For sure a next big thing is genetic engineering and it is just hitting the US stock market. Like nuclear power, genetic engineering has been a taboo since my childhood; sci-fi monster movies are embedded in the minds of many, with evil mutations causing havoc. So the good old trick of a rebrand seems to be doing the job of slipping in this unstoppable technology under the radar.

It is now called gene editing and offers hope and potential almost beyond limit. Fixing genetic diseases, the terrible consequences of which plague millions, is only the tip of a massive iceberg of positive possibilities, and in an incredible breakthrough the ability to fix and change DNA has been slung into a new era by a new technology.

This technology is Crispr. Im no biogeneticist, but my finance guy understanding is that Crispr allows for a cheap, fast and accurate way of cutting out and/or adding in new bits of DNA code to existing living cells. Jennifer Doudna and Emmanuelle Charpentier were given the Nobel Prize for the development of the gene-editing tools that led from Rodolphe Barrangous yogurt research to the development of phage-resistant yogurt bacteria. (Blessed are the cheese makers.)

By the time you read this I will probably own all the Crispr technology companies listed, which include: Crispr Therapeutic, Editas Therapeutics, Intellia Therapeutics and Beam Therapeutics, all in the US, but while this is an exciting cohort today, just like the hydrogen economy theme and for that matter cryptocurrency, this is just the start of a new explosive segment that will grow for decades and provide those investors who care to skill up on the nuances of the field an opportunity to build wealth for much of the rest of their lives.

Right now, these companies are comparative minnows with market caps of $4-$11bn. These might seem a lot, but when you consider Tesla can hold a value of $700bn and merely replaces the internal combustion engine in one form of transport, you can imagine that a technology that transforms the engine of life itself can command some interesting valuations given enough time to penetrate the mainstream investor imagination.

People always want names as tips. Its an entropic method that is as inadvisable as it is popular. The key tip is that this is a new segment that will explode in value and that speculators/investors can adopt it as a platform for long-term gains.

Biotech companies and the so called pharmas are a core segment for investors, especially in the US, and it is only a matter of time before the Crispr-Cas9 revolution breaks out of the specialist press and into the mainstream. Things never go as fast as you expect, especially if you see the future clearly. This shortening of the field of view is a drawback of seeing what is going to happen next, but in the end it does come to pass. You could have been receiving email in 1990 and be clearly expecting online systems to turn everything upside down, yet still have to wait a few years to jump on board the stocks that would then go vertical.

The key idea is that its worth getting in early, hanging on tight while constantly winnowing the sector down to the small sub-group that will be the colossuses in 20 years time. The earlier you can find such a sector, the more upside there is. As such, its a great time to be investing, as there are crypto, hydrogen economy and gene-editing segments that will have a long and lucrative future for those gritty enough to dig in and become experts in these technologies.

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Money & Markets: The latest technology to invest in gene editing - E&T Magazine

Medical science is starting to license and use drugs and procedures that change the genetic code inside the bodys cells, and to correct the bad code that can give rise to conditions such as cancer and the auto-immune diseases. Since HIV is a disease that results from a virus inserting such a piece of bad code into our genes, such therapies could be used to snip out that code and effect a cure.

This was what attendees at last months International AIDS Society Conference on HIV Science (IAS 2021) heard at the workshop on curing HIV. The workshop opened with two introductory talks by Professor Hans-Peter Kiem, the chair of gene therapy at the Fred Hutchinson Cancer Research Center in Seattle in the US (the Fred Hutch) and, in a joint presentation, by the Fred Hutchs Dr Jennifer Adair and Dr Cissy Kityo of the Joint Clinical Research Centre (JCRC) in Kampala, Uganda.

The latter talk was a sign of acknowledgement that, while the prospects for genetic medicine are brighter than ever before, their expense and sophistication do not fit well with the global epidemiology of HIV, which mainly affects the worlds poorest and most disadvantaged communities. Despite this, Fred Hutch and JCRC have embarked upon a joint research programme to develop within the next few years a genetic therapy treatment for HIV that could be realistically scaled up for use in lower-income settings.

A unit of heredity, that determines a specific feature of the shape of a living organism. This genetic element is a sequence of DNA (or RNA, for viruses), located in a very specific place (locus) of a chromosome.

A type of experimental treatment in which foreign genetic material (DNA or RNA) is inserted into a person's cells to prevent or fight disease.

To eliminate a disease or a condition in an individual, or to fully restore health. A cure for HIV infection is one of the ultimate long-term goals of research today. It refers to a strategy or strategies that would eliminate HIV from a persons body, or permanently control the virus and render it unable to cause disease. A sterilising cure would completely eliminate the virus. A functional cure would suppress HIV viral load, keeping it below the level of detection without the use of ART. The virus would not be eliminated from the body but would be effectively controlled and prevented from causing any illness.

The body's mechanisms for fighting infections and eradicating dysfunctional cells.

In cell biology, a structure on the surface of a cell (or inside a cell) that selectively receives and binds to a specific substance. There are many receptors. CD4 T cells are called that way because they have a protein called CD4 on their surface. Before entering (infecting) a CD4 T cell (that will become a host cell), HIV binds to the CD4 receptor and its coreceptor.

HIV cure research pioneer Dr Paula Cannon of the University of Southern California, chairing the session, said: After several decades of effort and false starts, gene therapies now hold out promise for diseases that were previously untreatable.

Hans-Peter Kiem acknowledged the pivotal role of community advocacy in supporting cure research, noting that his project, defeatHIV, was one of the first beneficiaries of a grant from the Martin Delaney Collaboratories, named after the celebrated US treatment activist who died in 2009.

The other factor that gave impetus to HIV cure research was, of course, the announcement that someone had been cured: Timothy Ray Brown, whose HIV elimination was first announced in 2008 and who came forward publicly in 2010. He died in 2019 from the leukaemia whose treatment led to his HIV cure but by then had had 13 years of post-HIV life. He had survived long enough to talk with Adam Castillejo, the second person cured of HIV, and encourage him to come forward too.

Timothy and Adams stories showed that HIV could be cured, and with a crude form of gene therapy too: cancer patients, they were both given bone marrow transplants from donors whose T-cells lacked the gene for the CCR5 receptor, which is necessary for nearly all HIV infection.

But there have only been two cures for two reasons: firstly, bone marrow transplant is itself a very risky procedure involving deleting and replacing the entire immune system of already sick patients. In 2014 Browns doctor, Gero Hutter, reported that Timothy Ray Brown was only one of out of eight patients on whom the procedure had been tried, but that all the others had died.

Secondly, compatible bone marrow donors are hard to come by as it is, and restricting them to the 1% or so of people who lack the CCR5 receptor, all of them of northern European ancestry, means very few people could benefit from this approach. Attempting transplant with T-cells that do not lack CCR5, in the hope that replacing the immune system with cells from a person without cancer will also get rid of their HIV anyway, has produced temporary periods of undetectable HIV off therapy, but the virus has always come back.

(People like Brown and Castillejo, whose HIV infection was cured by medical intervention, need to be distinguished from people who seem to have spontaneously cured themselves, such as Loreen Willenberg: such people are of course of great interest to cure researchers, but the trick is to make it happen consistently in other people.)

Brown and Castillejos cures, as transplants, were so-called allogenic, meaning that the HIV-resistant cells came from another person. Better would be autogenic transplants, in which immune system cells are taken from a person with HIV, genetically altered in the lab dish to make them resistant to HIV, and then re-introduced. This type of procedure written about for aidsmap as long ago as 2011 by treatment advocate Matt Sharp, who underwent one.

The repertoire of gene therapies is not restricted to CCR5 deletion. Gene therapy is immensely versatile, and could be used in a number of ways.

Instead of using gene therapy to make cells resistant to HIV, it could directly repair defective genes in cells by means of cut-and-paste technology such as CRISPR/Cas9. This is already being used in trials for some genetic conditions such as cystic fibrosis and sickle-cell anaemia. Given that HIV-infected cells are also defective in the sense that they contain lengths of foreign DNA that shouldnt be there, they are amenable to the same molecular editing. Early trials have produced promising results but the challenge, as it has been in a lot of gene therapy, is to ensure that the cells containing DNA are almost entirely eliminated.

One way of doing this is not to delete the HIV DNA from infected cells but to preferentially kill off the cells themselves by creating so-called chimeric antigen receptor (CAR) T-cells. These are T-lymphocytes whose genes have been modified so that their usual receptors such as CD4 or CD8 have been replaced with receptors attuned very specifically to antigens (foreign or unusual proteins) displayed by infected cells and cancer cells. A couple of CAR cell therapies are already licensed for cancers; the problem with HIV is that the reservoir cells do not display immune-stimulating antigens on their surfaces. This means that CAR T-cells would have to be used alongside drugs such as PD-1 inhibitors that stop the cells retreating into their quiescent reservoir phase, an approach demonstrated at IAS 2021.

A couple of other approaches could be used to produce either vaccines or cures. One is to engineer B-cells so they produce broadly neutralising antibodies. A way of tweaking them to do this, called germline targeting, is covered was also discussed at IAS 2021, but if we manage to generate B-cells that can do this, we could then in theory directly edit their genes to make them do the same thing.

"Timothy Ray Brown and Adam Castillejo were both given bone marrow transplants from donors whose T-cells lacked the gene for the CCR5 receptor."

The other way is to induce cells to make viral antigens or virus-like particles that the immune system then reacts to. Scientists have been working on this technique for 20 years and it triumphed last year when the Pfizer and Moderna vaccines against the SARS-CoV-2 virus had over 90% success in suppressing symptomatic COVID-19. These vaccines are not genetic engineering in the sense of altering the genome of cells; rather, they introduce a product of the genetic activation in cells, the messenger RNA that is produced when genes are read and which is sent out into the rest of the cell to tell it to make proteins.

However because HIV is more variable and less immunogenic than SARS-CoV-2, the vaccine induced by the RNA would have to be something that looked much more like a whole virus than just the bare spike protein induced by the Pfizer and Moderna vaccines. If there was such a vaccine could be used both therapeutically as well as in prevention, by stimulating an immune reaction to activated HIV-infected cells. Moderna have announced they will now resume the HIV vaccine research they were working on when COVID-19 hit.

The problem with all these more gentle procedures is that it has proved difficult to replace all the HIV-susceptible cells with the HIV-resistant or HIV-sensitised ones: although engraftment takes place, meaning that the autologous cells are not rejected by the body and are able to establish a population for some time (in some animal experiments, replacing as much as 90% of the native immune cells), eventually the unaltered immune cells tend to win out because the introduced cells lack the deep reservoir of replenishing cells.

Kiem said that the way scientists have been trying to get round this is to only select and alter so-called haematopoeic stem cells (HSCs). These rare and long-lived cells, found in the bone marrow, are the replenishing reservoir of the immune system. They differentiate when they reproduce and give rise to all the immune cells that do different things: CD4 and CD8 T-lymphocytes, B-cells that make antibodies, macrophages that engulf pathogens, dendritic cells, monocytes, natural killer cells, and others.

Altering HSCs genetically so that they are able to fight HIV in one way or another could in theory give rise to a persistent, HIV-resistant immune system. They could in theory lie in wait and be ready to produce effector cells of various types. They would be ready when a new HIV infection comes along (if used as a vaccine) or when HIV viral rebound happens and there is detectable virus in the body (if used as part of a cure). If a person with CAR-engineered stem cells could have repeated cycles of treatment interruption, their HIV reservoir could in theory slowly be deleted.

"Gene therapies are astonishingly expensive."

As mentioned above, although genetic medicine shows enormous promise, the complexity and expense of its techniques means that at present it is unlikely to benefit most people who really need it.

Hans-Peter Kiem said that currently about 60 million people have conditions that could benefit from gene therapy. The vast majority of these either have HIV (37 million) or haemoglobinopathies blood-malformation diseases such as sickle-cell anaemia and thalassaemia that are also concentrated in the lower-income world (20 million).

Dr Jennifer Adair, one of the first researchers to have proposed collaboration on gene therapies for HIV with African institutes, said that gene therapies have already been licensed for conditions such as thalassaemia, spinal muscular atrophy, T-cell lymphoma and a form of early-onset blindness.

But they are astonishingly expensive. The worlds most expensive drug tag goes, depending on which source you read, either to Zynteglo, a genetic medicine correcting malformed beta-haemoglobin and licensed in the US for thalassaemia, or Zolgensma, a drug licensed in Europe and given to children to correct the defective gene that results in spinal muscular atrophy.

Both cost about 1.8 million for a single dose. The price is not just due to the cost of the complex engineering used to make them, but because they are used to treat rare diseases and so have a small market.

At present the technology need to engineer autogenic genetically engineered cells is, if anything, even more expensive and complex than that needed to introduce allogenic cells. It can involve in the region of ten staff and a workspace of 50 square metres per patient. Recently a so-called gene therapy in a box has been made available that can reduce the area needed to produce autogenic genetically-engineered cells from 50 to less than one square metre, and the staff need to one or two, But what is really needed is genetic engineering in a shot; a therapy similar to a vector or RNA vaccine that can be introduced as an injection and produces the genetic changes needed within the body.

Undaunted by the challenges, the US National Institutes of Health are collaborating with the Bill and Melinda Gates foundation to work on a combined programme of HIV and sickle-cell-anaemia genetic therapy (given that something that works for one could be adapted to work with the other).

And the Fred Hutchinson Center has teamed up with the Joint Clinical Research Centre in Uganda with the very ambitious goal of making a genetic therapy that would be at least ready for human testing within two years in an African setting, and that could be scaled up to be economical for Africa if successful.

Dr Cissy Kityo of JCRC in Uganda told the conference that as of 2020, there were 373 trials of gene therapy products registered, of which 35 were in phase III efficacy trials. The global budget for regenerative medicine, which includes genetic therapy and related techniques, was $19.9 billion, having jumped by 30% since the previous year. The US Food and Drug Administration projects that based on the current rate of progress and the development pipeline, they may be licensing around 100 gene-therapy products a year by 2025.

This branch of medicine is no longer exotic, she said. Now steps have to be taken to trial gene therapies in the people who needed them most, and to turn the exotic into the affordable, she added.

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Could gene therapies be used to cure more people with HIV? - aidsmap

Upon an otherwise unruly landscape of choppy sea and craggy peaks, the salmon farms that dot many of Norways remote fjords impose a neat geometry. The circular pens are placid on the surface, but hold thousands of churning fish, separated by only a net from their wild counterparts. And that is precisely the conundrum. Although the pens help ensure the salmons welfare by mimicking the fishs natural habitat, they also sometimes allow fish to escape, a problem for both the farm and the environment.

In an attempt to prevent escaped fish from interbreeding with their wild counterparts and threatening the latters genetic diversity, molecular biologist Anna Wargelius and her team at the Institute of Marine Research in Norway have spent years working on ways to induce sterility in Atlantic salmon. Farmed salmon that cannot reproduce, after all, pose no threat to the gene pool of wild stocks, and Wargelius has successfully developed a technique that uses the gene-editing technology Crispr to prevent the development of the cells that would otherwise generate functioning sex organs.

In fact, Wargelius team was a little too successful. To be financially viable, commercial fish farms need at least some of their stock to reproduce. So the scientists went a step further, developing a method of temporarily reversing the modification they had already made. Theyve created what they call sterile parents.

The term may sound like an oxymoron, but the sterile parents have the potential to solve one of the most pressing problems facing salmon aquaculture, both in Norway and around the world. Wargelius says it could be up to a decade before the results of her work are commercially available, but once they are, they have the potential to make an already burgeoning food source markedly more friendly on the environment. And by prioritizing environmental concerns and employing a technique that simply turns off a gene rather than introducing one from a different species, Wargelius and her team may contribute to a shift in how genetic engineering is perceived in Norway, a country with some of the strictest regulations regarding genetically modified organisms on the books.

Aquaculture, the cultivation of saltwater and freshwater organisms under controlled conditions, has long been a controversial industry. On the one hand, its been hailed as an answer to overfishing and to a growing global populations demands for protein; on the other, it canpollute the water and spread diseaseamong both farmed and wild fish. Despite these drawbacks, around the world, aquaculture is booming. In the two decades of this century, production has increased an average of 5.3 percent per year, and as of 2018, more than 126 million tons of the seafood consumed annually came from a farm. Of those 126 million, 1.4 million were Norwegian salmon.

In fact, Norway is the worlds largest producer of farmed salmon, raising millions of the fish in sea pens scattered throughout its jagged fjords. Aquaculture is the countrys second-largest industry, and those pens are an important part of its reputation among major seafood producers for relatively sustainable production methods. Up to 260 feet wide and 160 feet deep, they are open-net, allowing the chilly waters of the North Atlantic to circulate freely and more closely replicate part of the salmons habitat.

Exact numbers are difficult to pin down, butone estimateputs the total number of escaped fish at 2.1 million in the last decade, and anotherstudyfound evidence of genes from farmed fish in wild specimens that were caught in 109 out of 147 Norwegian salmon rivers. Already under threat from overfishing the population has been cut in half in the last 30 years the threat to wild salmon biodiversity from this aquaculture gene stock is so great that farms are fined for fish that escape. Inone casethis year, Mowi, a company based in Bergen, Norway, was fined $450,000 for a 2018 incident in which 54,000 fish escaped.

In her early work, Wargelius focused on vaccines as a means of inducing the sterility that would minimize that threat. But her strategy changed soon after she learned about Crispr, the gene-editing technique that functions kind of like a Swiss Army knife, with different tools that make it possible to both insert material into a gene and snip it out.

Dorothy Dankel, a researcher at the University of Bergen who collaborates with Wargelius, recalled when Crispr first popped up. Anna was saying, Wow, theres this new paper that just came out with something called Crispr, Dankel said. She felt like the vaccine approach, its really kind of hit or miss. With Crispr, Wargelius thought, the team could hedge their bets.

But some scientists were skeptical, said Dankel. Genetically modified crops have encountered varying degrees of consumer resistanceglobally, in part because some modification techniques involve inserting genes from one species into another a process that has provoked fears ofFrankenstein foods. People were freaking out, saying, No, its GMO, we cant do that in Norway, said Dankel.

But an exception exists for lab experiments. By using Crispr to treat newly fertilized fish eggs, Wargeliuss team was able to knock out a specific gene calleddead-endordnd that is responsible for the migration of germ cells to the gonad. Germ cells eventually give rise to gametes, or sexual reproductive cells, and without them, the thinking went, the fish in this initial cohort would not reach sexual maturity.

At the same time, the scientists also turned off a gene that controls for pigmentation, because the resulting albinism would make it significantly easier to keep track of which fish had been modified. Sure enough, many of the Crispr-treated embryos grew into yellow-hued salmon that lacked germ cells.

Salmon grow slowly, so it would take just over a year before the biologists could confirm the impact of the missing cells, but by 2016, it was clear: 100 percent of the albino fish failed to reach sexual maturity. They were all sterile.

Using Crispr to change a gene that causes sex organs to develop, scientists have created salmon that are sterile. But, with the right treatment, those same fish may have their fertility returned, and thus breed sterile offspring that still contain the edited gene. Video: Institute of Marine Research

It was a very elegant experiment, says Yonathan Zohar, a professor of marine biotechnology at the University of Maryland, Baltimore County and an expert in aquaculture and fish reproduction technologies. Linking thedead-endgene to an albino gene provided a very good visual indication of which fish had been treated, he said. Her approach made a lot of sense.

There was only one problem: The element of Crispr they were using to produce the sterile salmon required a technician to manually inject each embryo with a protein that cut thedndgene a labor-intensive method that is hardly viable for commercial fisheries. Wargelius wanted to find a way to reverse the impact of the genetic modification without removing it from the salmons DNA. After all, the goal was pass sterility along to the next generation. A fish farm could then keep its brood stock separate from the rest of the salmon.

We thought, okay, maybe the simplest way to produce enough sterile salmon is to enable some of the sterile fish to reproduce, Wargelius explained.

She was skeptical the idea would work. But a year after her team injected a certain mRNA from wild salmon into newly fertilized eggs in an effort to effectively turn the fishs fertility back on, Wargelius received a text from the research station with photos that proved the technique had worked. It read: We have many fish with germ cells here!

Eventually, the treated fish developed gonads and reached sexual maturity, producing offspring that inherited their parents genetic sterility. The scientists wont have the full results until this fall, after the first generation is 8 to 10 months old the age at which salmon normally develop gonads. But so far, they say, everything is on track. Theoretically, yes we should get 100 percent sterility, Wargelius says.

Of course, certain safeguards need to be in place. A Crispr experiment to breedhornless cattlein the U.S. was initially hailed as a major success, but was laterdiscoveredto have introduced an unintended stretch of bacterial DNA into the cows genome. The producers thought that only their edit was being introduced, said Jennifer Kuzma, a professor and co-director of the Genetic Engineering and Society Center at North Carolina State University. You have to be cautious that youre not getting any off-target or unintended effects, she said. One way guard against this: Sequence the offsprings entire genome and look carefully for unintended changes in the DNA.

The Norwegian team is taking care to do this, and Kuzma sees their work as, in many ways, exemplary. The work has a societal benefit, it has biosafety mechanisms in place, and its being done in collaboration with ethicists, she says. Its being done under a pretty solid, good governance model.

The Norwegian scientists havent yet sequenced the salmons genomes to look for any secondary effects, and its still relatively early in the salmons lifespan, so they wont know about behavioral changes until the sterile offspring are transferred to the sea pens; currently the juveniles are living in tanks in the lab. Anna will have to demonstrate that when you take those fish to the net pens, they perform as well as the non-treated ones, says Zohar. And, he adds, shell have to scale everything up.

Those are significant hurdles,but the biggest hurdle, Zohar points out, is regulatory. From the time that genetically engineered crops first became widely available in the 1990s, their production has been regulated to different degrees, with some countries, such as the United States, merely demanding that the crops meet the same health and environmental standards as their conventionally bred counterparts.

Other countries have imposed stricter regulations on selected crops. In Mexico, for example, genetically engineered corn is banned because it poses a threat to the biodiversity of native maize. And other countries especially those in Europe have banned all genetically engineered crops intended for human consumption, as a food safety precaution. (To date, the safety concerns associated with GMOs have not been borne out.)

Norway has some of the most stringent restrictions in the world when it comes to genetically modified organisms: Farmers are barred from cultivating GMO crops and no genetically modified food products can be imported. Those policies, codified in the 1993 Gene Technology Act, were a reflection of both a powerful and fiercely protectionist agricultural sector and a public that is deeply conservationist and prides itself on its close connection to nature.

It was black or white, says Aina Bartmann, CEO of GMO-Network, an umbrella organization of nongovernmental organizations and corporations that represents 1.7 million consumers. It was so obvious, I think, for everyone in Norway, in Scandinavia, and also in the European Union, she said, that GMOs offered no contribution to anything we want.

Under the current legislation in Europe, Crispr is considered a gene modification technique, and no products created through it can be sold in Norway. (It is, however, authorized for research, and is being tested on lettuce and strawberries, in addition to salmon.) But that may be changing. Bjrn Kre Myskja, a professor of ethics at Norwegian University of Science and Technology, is working on a study of the conditions that would make gene editing technology socially and morally acceptable to Norwegians.

His research is currently in progress, but hes already seeing evidence, both in his work and anecdotally, that attitudes are changing particularly when it comes to technologies like Crispr, which dont always involve inserting the genes of one species into another. When you do something that might happen in an ordinary naturally-occurring kind of mutation, he said, then there seems to be a larger percentage that will find that acceptable.

Myskja has also observed in his research that opposition varies depending upon the perceived purpose of the modification. A modification that is intended to increase yields or to make an organism grow faster and therefore increase the profits of the producer is generally frowned upon in Norway. But a modification that achieves a broader good by increasing sustainability, for example, or improving animal welfare, might be tolerated. Therefore, a modification that benefits salmon, such as sterility or resistance to sea lice, may fall on the acceptable part of the scale, says Myskja.

His early findings are echoed in asurveyconducted by GENEinnovate, a collaboration of private companies, research institutions, and the Norwegian Biotechnology Advisory Board, an independent committee made up of 15 members appointed by the Norwegian government. It found that a majority of Norwegian consumers had a positive attitude toward gene editing if it carried clear social benefits and was carefully labelled. Bartmann, of GMO-Network, has noticed the same even among her organizations members. There are a lot of uncertainties associated with many aspects of gene editing, she said, and her members remain concerned about possible risks of releasing genetically modified crops or animals into the wild. We support the research going on now in Norway, she said, and we think that the more knowledge we get about the new methods, the better.

In the U.S., Kuzma has noted similar trends. In surveys, people say they see edits or genes inserted from the same species as slightly more acceptable than transgenic, she said, referring to genes inserted from different species. In the marketplace, in part because there are so few products in the market, a significant proportion dont really care. But there are still years of distrust to get over, and theres a segment of around 20 percent that will reject GMOs in any form.

For the moment, the aquaculture industry in Norway is hedging its bets. Historically, the industry has taken a hardline position against GMOs, conscious that the appeal of their products rests on a public perception of genetic purity. AquaGen, a breeding company that supplies fertilized Atlantic salmon eggs, sent a statement to Undark, writing that producing sterile salmon by Crispr may be a future solution, but many technical, ethical, legislative, and commercial issues need to be solved before commercial implementation. Cermaq, an international salmon farming company, similarly wrote to Undark that farming sterile salmon may have advantages, and research in this area is very interesting, but noted that the company is currently not planning to farm the gene-edited fish.

Yet Dankel has seen change among industry representatives. In 2014, she interviewed a senior manager at AquaGen, and asked if she saw a future for Crispr in her company. Dankel received a hard no: This is playing with fire, Dankel recalls being told. Our customers expect pure genetics; they dont want anything modified. Just a few years later, she says, the company told her the technique is part of their research strategy.

The speed with which Crispr technology is developing and being adopted in laboratories around the world helps explain some of that transformation. But locally, Dankels own work plays a role, too. Within the Wargelius lab, Dankel is the representative forResponsible Research and Innovation, a position devoted to ensuring that ethical and social considerations are embedded into the research.

This involves doing outreach explaining the research to the public and what Dankel calls inreach getting people who arent used to collaborating on a subject to work together. When it comes to something as complex as Crispr, she finds that with these interdisciplinary teams that combine biochemistry and molecular biology with social and economic assessments, it is essential to create a common language for what the goals are, and what success and failure might look like.

Dankel too is noticing a change in the discourse in the wake of the Crispr salmon. The pendulum has swung and now people even the Biotechnology Council of Norway are only saying the good things about Crispr and not anything about off-target effects, Dankel said, or that once you start this technology you can never put it back.

Yet perhaps the clearest indication that Norway may soon adjust its legislation to make room for Crispr is the governments creation, in November 2020, of anew committeeto review the field of genetic technologies. Headed by Wargelius, it will report on its findings and recommendations in June 2022. Norway really wants to promote a public debate about the law that is based in science, says Dankel. They could have chosen a law professor to lead that. They could have chosen someone whos not a biologist.

And Wargelius still has a lot of biology to do. She and her team are just now beginning to work on their second generation of fish and also are planning to sequence the genome of the sterile fish to ensure there arent any unintended edits. Wargelius estimates that any commercial licensing for this application, provided it is approved, is five to 10 years away.

But shes in no hurry. With Crispr, she suspects, Norway is moving toward an application-based process, where the technology will be approved in cases where the need for or benefit from a specific use is sufficient to outweigh the risks. Which is why, she says, she chooses an open and thorough approach for her own research. We also now are trying to start a collaboration with both economic and ethical researchers to see what is the potential in the market, what will people think, said Wargelius. I would like to have a quite slow process, where we really have all the documentation that we need to be certain that its a solid product.

Lisa Abend is a journalist and food writer based in Copenhagen and Madrid. Her work has been published in Bon Appetit, Food and Wine, Time Magazine, The Atlantic, Wired, The New York Times, and Slate, among other outlets. Find Lisa on Twitter @LisaAbend

A version of this article was originally posted atUndarkand is reposted here with permission. Undark can be found on Twitter@undarkmag

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How can we protect wild salmon from interbreeding with farmed salmon? CRISPR gene editing is a solution - Genetic Literacy Project

Zolgensma which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay is currently the most expensive drug in the world. A one-time treatment of the life-saving drug for a young child costs US$2.1 million.

While Zolgensmas exorbitant price is an outlier today, by the end of the decade therell be dozens of cell and gene therapies, costing hundreds of thousands to millions of dollars for a single dose. The Food and Drug Administration predicts that by 2025 it will be approving 10 to 20 cell and gene therapies every year.

Im a biotechnology and policy expert focused on improving access to cell and gene therapies. While these forthcoming treatments have the potential to save many lives and ease much suffering, health care systems around the world arent equipped to handle them. Creative new payment systems will be necessary to ensure everyone has equal access to these therapies.

Currently, only 5% of the roughly 7,000 rare diseases have an FDA-approved drug, leaving thousands of conditions without a cure.

But over the past few years, genetic engineering technology has made impressive strides toward the ultimate goal of curing disease by changing a cells genetic instructions.

The resulting gene therapies will be able to treat many diseases at the DNA level in a single dose.

Thousands of diseases are the result of DNA errors, which prevent cells from functioning normally. By directly correcting disease-causing mutations or altering a cells DNA to give the cell new tools to fight disease, gene therapy offers a powerful new approach to medicine.

There are 1,745 gene therapies in development around the world. A large fraction of this research focuses on rare genetic diseases, which affect 400 million people worldwide.

We may soon see cures for rare diseases like sickle cell disease, muscular dystrophy and progeria, a rare and progressive genetic disorder that causes children to age rapidly.

Further into the future, gene therapies may help treat more common conditions, like heart disease and chronic pain.

The problem is these therapies will carry enormous price tags.

Gene therapies are the result of years of research and development totaling hundreds of millions to billions of dollars. Sophisticated manufacturing facilities, highly trained personnel and complex biological materials set gene therapies apart from other drugs.

Pharmaceutical companies say recouping costs, especially for drugs with small numbers of potential patients, means higher prices.

The toll of high prices on health care systems will not be trivial. Consider a gene therapy cure for sickle cell disease, which is expected to be available in the next few years. The estimated price of this treatment is $1.85 million per patient. As a result, economists predict that it could cost a single state Medicare program almost $30 million per year, even assuming only 7% of the eligible population received the treatment.

And thats just one drug. Introducing dozens of similar therapies into the market would strain health care systems and create difficult financial decisions for private insurers.

[Over 110,000 readers rely on The Conversations newsletter to understand the world. Sign up today.]

One solution for improving patient access to gene therapies would be to simply demand drugmakers charge less money, a tactic recently taken in Germany.

But this comes with a lot of challenges and may mean that companies simply refuse to offer the treatment in certain places.

I think a more balanced and sustainable approach is two-fold. In the short term, itll be important to develop new payment methods that entice insurance companies to cover high-cost therapies and distribute risks across patients, insurance companies and drugmakers. In the long run, improved gene therapy technology will inevitably help lower costs.

For innovative payment models, one tested approach is tying coverage to patient health outcomes. Since these therapies are still experimental and relatively new, there isnt much data to help insurers make the risky decision of whether to cover them. If an insurance company is paying $1 million for a therapy, it had better work.

In outcomes-based models, insurers will either pay for some of the therapy upfront and the rest only if the patient improves, or cover the entire cost upfront and receive a reimbursement if the patient doesnt get better. These models help insurers share financial risk with the drug developers.

Another model is known as the Netflix model and would act as a subscription-based service. Under this model, a state Medicaid program would pay a pharmaceutical company a flat fee for access to unlimited treatments. This would allow a state to provide the treatment to residents who qualify, helping governments balance their budget books while giving drugmakers money upfront.

This model has worked well for improving access to hepatitis C drugs in Louisiana.

On the cost front, the key to improving access will be investing in new technologies that simplify medical procedures. For example, the costly sickle cell gene therapies currently in clinical trials require a series of expensive steps, including a stem cell transplant.

The Bill & Melinda Gates Foundation, the National Institute of Health and Novartis are partnering to develop an alternative approach that would involve a simple injection of gene therapy molecules. The goal of their collaboration is to help bring an affordable sickle cell treatment to patients in Africa and other low-resource settings.

Improving access to gene therapies requires collaboration and compromise across governments, nonprofits, pharmaceutical companies and insurers. Taking proactive steps now to develop innovative payment models and invest in new technologies will help ensure that health care systems are ready to deliver on the promise of gene therapies.

The Bill & Melinda Gates Foundation has provided funding for The Conversation US and provides funding for The Conversation internationally.

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New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many - The Conversation US

SOUTH SAN FRANCISCO, Calif., Sept. 2, 2021 /PRNewswire/ --The National Science Foundation (NSF) awarded Felix Biotechnology a Small Business Innovation Research (SBIR) Phase Igrantfor$256,000 to further enhance their machine learning-based approach for engineering bacteriophage as a novel treatment for drug-resistant bacterial infections.

Bacteriophage, or phage, are viruses that infect and kill bacteria. Phage have key benefits relative to small molecule antibiotics, but there are several hurdles to turning phage into a true biotherapeutic. First, like traditional antibiotics, bacteria can become resistant to phage. Second, due to their specificity, finding the right phage to treat an infection can be challenging. Felix has made significant headway in addressing the first hurdle. The NSF SBIR grant will support Felix's efforts to address the second hurdle by engineering phage for improved efficacy and utility, starting with proof-of-concept work in Pseudomonas aeruginosa, a pathogen of high clinical need.

"NSF is proud to support the technology of the future by thinking beyond incremental developments and funding the most creative, impactful ideas across all markets and areas of science and engineering," said Andrea Belz, Division Director of the Division of Industrial Innovation and Partnerships at NSF.

"The NSF funding will supercharge our high-throughput methodologies for fast and economical identification and characterization of phage and phage-bacteria interactions. These rich datasets power our ML and genetic engineering tools, creating a platform for tuning phage specificity and producing phage products targeting dangerous human pathogens, like P. aeruginosa and Mycobacterium abscessus," said Felix Biotechnology co-founder and principal investigator on the grant, Natalie Ma, PhD.

Phase I SBIR awardees are eligible to apply for Phase II awards of up to $1 million. Small businesses with Phase II funding are eligible for $500,000 in additional matching funds with qualifying third-party investment or sales.

"Felix is tackling the most challenging technical hurdles limiting the broad application of phage therapy to the clinic. With the antimicrobial resistance crisis only growing, new solutions to treat bacterial infections are absolutely essential for ensuring our global future health," said Dr. Ma.

For more on Felix Biotechnology, please visit: https://www.felixbt.com/

For more on the National Science Foundation's Small Business Programs, please visit: https://seedfund.nsf.gov/

SOURCE Felix Biotechnology, Inc.

https://www.felixbt.com/

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Felix Biotechnology Awarded Competitive Grant from the National Science Foundation to Power Phage Engineering Platform - PRNewswire