Monthly Archives: November 2020

Further guidance required for assessment of gene drive technology, says EFSA – EURACTIV

Posted: November 20, 2020 at 3:57 pm

Existing guidelines are adequate for evaluating risks associated with gene-drive modified insects, but further guidance is needed for some areas, most notably for environmental risk assessments, according to an opinion of the EUs food safety agency (EFSA).

After being mandated by the European Commission, EFSAs experts on genetically modified organisms (GMOs) published the scientific opinion related to engineered gene drives on Thursday (12 November), specifically focusing on gene drive modified disease-transmitting insects, primarily mosquitoes.

The evaluation was requested to explore the issue ahead of the consideration of any possible applications of the technology and is also designed to support the EU in discussions on the biosafety of GMOs in international fora such as the United Nations.

It found that while existing guidelines are sufficient for evaluating risks associated with technology, further guidance is needed for some areas, such as molecular characterisation, environmental risk assessment and post-market environmental monitoring.

Synthetic gene drives are a new form of genetic engineering, created via the genetic engineering method CRISPR/CAS9, and are intended to permanently modify or eradicate populations, or even whole species, in the wild.

The idea of gene drive technology is to force the inheritance of detrimental genetic traits. In this way, scientists hope to reprogramme or eradicate species such as disease-carrying insects and invasive species.

This is a key distinction between gene-drive organisms (GDOs) and genetically modified organisms (GMOs), which are explicitly designed to contain the spread of modified traits.

Scientists say that gene drive technology could play a key role in suppressing or modifying mosquito populations, thus potentially eradicating the life-threatening diseases they carry, such as malaria.

Recently, Imperial College London created a modification that was able to eliminate populations of malaria-carrying mosquitoes in lab experiments, funded by the Bill & Melinda Gates Foundation under the Target Malariaproject.

The technology is also being explored to control agricultural pests, eradicate invasive species, and rescue endangered species, with research rapidly evolving in this area.

After over 78 environmental and agricultural organisations signing a letter this week calling for a moratorium on gene drive technology, EURACTIV took a closer look at the controversial technology to find out about what it is and the implications it holds.

However, the report acknowledges there is concern that this emerging technology may have possible and irreversible side effects.

While Mareike Imken of the German association Save our Seeds welcomed EFSAs conclusion that existing guidelines for genetically engineered insects are insufficient for undertaking environmental risk assessments, she raised concerns that the report failed to acknowledge other key issues.

EFSA does not acknowledge a key challenge for the risk assessment and monitoring of genetically engineered gene drive organisms so-called next-generation effects, she highlighted.

These next-generation effects would encompass unintended changes to the biological characteristics in the offspring of GDOs, which she said would likely happen due to the repeated and uncontrollable process of genetic engineering that gene drives set in motion in nature.

The likely impossibility to model and predict next-generation-effects, as already observed in the offspring of genetically engineered plants, calls for the establishment of cut-off-criteria for risk assessment.

She added that decision-making about this technology needs to be informed by more than risk assessment, stressing that there is an urgent need for a broader political debate and processes for participatory and inclusive societal deliberation around the desirability, costs and benefits of this technology.

[Edited by Zoran Radosavljevic]

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Taking Cultured Meat to the Next Level – Technology Networks

Posted: November 20, 2020 at 3:57 pm

With its origins in the late 1990s, lab-grown or cultured meat, is produced by providing stem cells extracted from the muscle of an animal with a suitable growth medium and nutrients, enabling them to proliferate and then differentiate to form muscle tissue. Creating meat in this way could help to address some of the environmental and ethical issues associated with livestock farming, as well as offer health benefits to consumers.Pairing cellular agriculture with genetic engineering could also enable the development of novel foods, with non-native features, that may be nutritionally enhanced. In a study recently published in Metabolic Engineering, researchers from Tufts University engineered bovine cells to endogenously produce phytoene, lycopene and -carotene, and found a reduction in lipid oxidation levels when this cultured meat was cooked.

Technology Networks had the pleasure of speaking to Andrew Stout, lead author of the study, to learn how these cells were created and explore the benefits of engineering in the abilities to produce additional nutrients. Andrew also discussed some of the challenges that are so far limiting the wider commercialization of cultured meat and how this may change in the future.

Anna MacDonald (AM): Can you give us an overview of the process by which the cow cells were engineered to produce the carotenoids?Andrew Stout (AS): To do this, we inserted three genes into the cells which encode enzymes that convert native compounds into carotenoids. Specifically, the first gene in this pathway (phytoene synthase) takes a native chemical and turns it into the carotenoid phytoene. The second gene (phytoene desaturase) turns some of that phytoene into a second carotenoid called lycopene. And the third gene (lycopene cyclase) turns some of that lycopene into a third carotenoid called beta-carotene. In that way, we're able to get cow cells to produce three different carotenoids that aren't naturally produced in bovine tissue. To engineer the cells, we used a system called the Sleeping beauty transposon system. This system is essentially a "cut and paste" tool which randomly cuts open the cells' genomes and inserts new DNA which we provide (in this case, the genes for carotenoid-producing enzymes).

AM: Why were beta-carotene, phytoene and lycopene chosen in particular?AS: There were several reasons for this. The first and most important was their role as dietary antioxidants. A key mechanistic link between red meat consumption and colorectal cancer is through lipid oxidation. This oxidation leads to the production of free radicals that can interact with tissue in the colon, damage cellular DNA, and ultimately contribute to cancer formation. Antioxidants can act to "quench" those free radicals, thus potentially inhibiting their cancer-causing potential. As carotenoids are powerful antioxidants, they offer a promising target for improving the nutritional features of cell-cultured meat.

Other reasons include the importance of beta-carotene as a vitamin A precursor, previous demonstrations of phytoene synthase efficacy in mammalian cells, and also as a sort of homage to golden rice, the first major demonstration of using genetic engineering to nutritionally enhance a food product.

AM: Were there any side-effects as a result of the nutritional engineering?AS: There were a few. The most obvious was a reduction in growth rate in bovine satellite cells that were engineered with carotenoid-producing enzymes. This would have negative implications for production processes if it proves to be unavoidable. Interestingly, though, in immortalized mouse muscle cells, this reduction in growth rate wasn't seen. Instead, cells producing carotenoids actually grew faster than non-engineered cells. One explanation for this could be that immortalized cells are more "robust" and are more amenable to engineering than the primary (non-immortalized) cow muscle cells we used. It's possible that immortalized cow cells would show growth-effects more like those seen in the mouse cells, which would turn this production down-side into a production up-side. Another side effect we saw was a change in color of the cells -- they took on a reddish tinge with the production of the carotenoids. I don't think this is really a "positive" or "negative" effect, but it is pretty interesting. Other potential side-effects that would need to be looked into would be the effects of carotenoids on cell differentiation, on the prevalence of other nutrients (e.g., cholesterol, etc.), and on flavor, texture, aroma, etc.

Karen Steward (KS): Why do you think you saw lower levels of lipid oxidation when the cell cultured meat was cooked compared to conventional meat?AS: Since carotenoids are antioxidants, they act to quench oxidation in cells during storage, cooking, etc., so we would expect lower lipid oxidation if the cells are producing carotenoids and therefore increasing the total cellular level of antioxidants.

KS: What do you see are the benefits of engineering in the abilities to produce additional phytonutrients to beef cells, as opposed say to having a traditional steak with some vegetables? Is there a risk that in providing these nutrients through meat intake a diet would consequently lack fibre which could impact gut health?AS: This is a fun question! I think we're an extremely long way from actually being able to use this technology to replace vegetables on our plates (and anyways, what a culinarily boring world that would be!) I like to think of this technology not as a replacement of vegetables, but an enhancement of meat. For instance, not all vegetables are high in carotenoids, so if you can get those nutrients from another source in your meal, then your overall consumption of them can increase. Also, the roll of carotenoids in specifically inhibiting oxidation in meat can act to mitigate some of the negative health implications of meat consumption without aiming to reduce vegetable consumption. As a final note, I'd like to think of this work as really just the tip of the iceberg of what's possible. There are so many options for enhancing meat with this or similar technologies--enhanced flavor, therapeutic activity, enhanced smell, etc. I think there's a world of totally novel foods that are possible and that would expand our culinary palette, not reduce it.

KS: Is there any need to start with cow cells? Could you essentially start with any cell type or are there limitations?AS: No need at all! I think this would likely work for all mammalian cells, and there's a strong chance it would work for avian and fish cells as well. We wanted to work with bovine cells because beef is such a major contributor to meat-associated greenhouse gas production and is one of the main red meats consumed around the world. As such, I think it's a really important target for all cultured meat work, including nutritional engineering.

AM: What challenges are so far limiting the wider commercialization of cultured meat?AS: The key hurdles are cost and scale. The field needs to reduce the cost of growth media (likely by reducing the cost of growth factors, reducing cellular reliance on growth factors, finding growth-factor alternatives, or other creative solutions), and to increase the scalability of cell culture (increased growth rate, increased maximum cell density, etc.). There are certainly plenty of other challenges, such as regulatory and consumer reception, demonstration of nutritional and food-quality value, and demonstration of food-safety, but I think that right now cost and scale still reign supreme.

AM: Where do you see the future of cellular agriculture headed?AS: A good question! I'll answer for two slightly different technologies.

First, for cultured meat specifically:

I think in the near future, the field is heading towards a bit of a "realignment" or specification in terms of goals, expectations, hype, etc. I think that this can be seen in some of the ways that companies are starting to look at their products with a bit more nuance, such as looking at the possibility of hybrid cell-based/plant-based products, which could overcome some of the cost/scale barriers of a fully cell-cultured product. Beyond that, I like to think that there will be an expansion of creative solutions to problems, or creative new ways of thinking about cell cultured meat. This could come in the form of looking at agricultural waste products for cell culture components, exploring novel genetic strategies to improve growth / reduce cost, or looking into alternate culture strategies / bioreactors.

Then for cellular agriculture more generally:

I think cellular agriculture in general, while certainly offering its own challenges and hurdles, is a lot further along the developmental pathway than cultured meat. I'm thinking here of products that are already on the market and demonstrably feasible such as recombinant milk proteins (Perfect Day Foods), recombinant collagen proteins (Geltor, Inc.), or recombinant proteins to improve plant-based products (Impossible Foods). I think these technologies are going to continue coming out and coming down in cost, allowing a bunch of new awesome products to come out and accelerate the development of plant-based or fermentation-derived products.

Andrew Stout was speaking to Anna MacDonald and Dr Karen Steward, Science Writers for Technology Networks.

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‘A plague to be reckoned with’: UMN research creates a buzz with invasive fruit fly research – Minnesota Daily

Posted: November 20, 2020 at 3:57 pm

In breeding genetically sterile males, researchers aim to develop a new genetic pesticide.

In the Gortner Laboratory on the St. Paul campus, University of Minnesota graduate student Nate Feltman examines a fruit fly under a microscope. About three millimeters in diameter, the spotted wing drosophila has been wreaking havoc on many soft-skinned berries in almost all of the states in the U.S., including Minnesota.

With a taste for fruits like raspberries, strawberries, blueberries and cherries, the invasive species has hit Minnesota berry growers by storm in the peak of their harvest season since it was first observed in the U.S. in 2008. Laying eggs in ripening fruit, the flies make fruit spoil quicker and make it unmarketable for farmers.

A team of researchers at the University are looking for a way to introduce sterile male spotted wing drosophila flies into the population as a form of genetic pest control, helping farmers in a way that other pesticides and proactive measures have not.

In early November, assistant professor Mike Smanski published an article about a new breakthrough in this research, demonstrating for the first time this kind of genetic engineering was possible in the common fruit fly. This shows that researchers could engineer this work into spotted wing drosophila in the future.

The Universitys Smanski Lab has also studied this technique in mosquitoes, zebra fish and carp, but never with this type of fruit fly, he said.

These are all a new class of genetic pesticide, basically, that allow you to engineer the pest organism itself and convert that pest organism into the pesticide, Smanski said.

Through this work the researchers can create a pest that is biologically the same, but when the females mate with these genetically modified males, they will not produce viable offspring, he said.

This sterile insect technique can be helpful not only in reducing the population of insects, but in reducing the impacts of insecticides on surrounding species and nearby ecosystems, said Feltman, a second-year biochemistry, molecular biology and biophysics graduate student.

As part of the second phase of the research, they will be doing lab tests on the genetically modified spotted wing drosophila, he said. The hope is that the genetic work will prevent the flies from producing offspring.

Over the past decade, researchers have placed traps to study the flies in various farms around the state, including The Berry Patch in Forest Lake, Minnesota.

Kevin Edberg, an owner of the farm, said he first saw the spotted wing drosophila about five or six years ago. His farm is primarily composed of raspberries, strawberries and blueberries that customers pick themselves. Since the flies have come in and made the fruit rot prematurely, he has lost the last week of his harvest season, which is generally one of the most profitable weeks of the year.

Edberg started putting up traps filled with apple cider vinegar to capture the flies and monitor when they come out. In early July, he would catch up to four flies per trap. As the season goes on however, he will catch almost 600 per trap.

Of all the changes that Ive seen, and all of the challenges that have evolved over the last 40 years, [the spotted wing drosophila is] the one that has been the biggest game changer in how we manage and grow, he said.

Traditional avenues of pest management do not work either, he said, which makes it even harder to navigate how to stop them. And if they do not control it one year, they will come back worse the next.

Jerry Untiedt, owner of Untiedts Vegetable Farm Inc. in Waverly, Minnesota, is another berry farmer University researchers have been working with.

Because he uses a high tunnel an unheated greenhouse and sweeps, blows and vacuums the rows between his crops to get rid of rotting fruit that attracts the bugs, Untiedt said he has fared better than other farms in the industry. Anecdotally, Untiedt said that 40% to 50% of raspberry growers no longer grow raspberries because the pest management is too complicated and costly.

He said he is grateful for the Universitys work because without their research, there would not be much known about the spotted wing drosophila.

This is of utmost importance because most people dont understand the biology of this fruit fly and what it could do to this industry, Untiedt said. If youre not vigilant in your management, it can actually destroy your entire crop. Literally, a plague to be reckoned with.

Correction: A previous version of this story misstated the Smanski Labs progress with genetic engineering in some organisms.

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Influence of PSRC1, CELSR2, and SORT1 Gene Polymorphisms on the Variab | PGPM – Dove Medical Press

Posted: November 20, 2020 at 3:57 pm

Laith N AL-Eitan,1 Barakat Z Elsaqa,2 Ayah Y Almasri,1 Hatem A Aman,1 Rame H Khasawneh,3 Mansour A Alghamdi4,5

1Department of Biotechnology and Genetic Engineering, Faculty of Science and Arts, Jordan University of Science and Technology, Irbid 22110, Jordan; 2Faculty of Medicine, Jordan University of Science and Technology, Irbid 22110, Jordan; 3Department of Hematopathology, King Hussein Medical Center (KHMC), Royal Medical Services (RMS), Amman 11118, Jordan; 4Department of Anatomy, College of Medicine, King Khalid University, Abha 61421, Saudi Arabi; 5Genomics and Personalized Medicine Unit, College of Medicine, King Khalid University, Abha 61421, Saudi Arabia

Correspondence: Laith N AL-EitanDepartment of Biotechnology and Genetic Engineering, Faculty of Science and Arts, Jordan University of Science and Technology, P.O. Box 3030, Irbid 22110, JordanTel +962-2-7201000 ext 23464Email lneitan@just.edu.jo

Background: Cardiovascular disease is one of the most common causes of morbidity and mortality worldwide. Several cardiovascular diseases require therapy with warfarin, an anticoagulant with large interindividual variability resulting in dosing difficulties. The selected genes and their polymorphisms have been implicated in several Genome-Wide Association Study (GWAS) to be associated with cardiovascular disease.Objective: The goal of this study is to discover if there are any associations between rs646776 of PSRC1, rs660240 and rs12740374 of CELSR2, and rs602633 of SORT1 to coronary heart disease (CHD) and warfarin dose variability in patients diagnosed with cardiovascular disease undergoing warfarin therapy.Methods: The study was directed at the Queen Alia Hospital Anticoagulation Clinic in Amman, Jordan. DNA was extracted and genotyped using the Mass ARRAY system, statistical analysis was done using SPSS.Results: The study found several associations between the selected SNPs with warfarin, but none with cardiovascular disease. All 4 studied SNPs were found to be correlated to warfarin sensitivity during the stabilization phase except rs602633 and with warfarin dose variability at the initiation phase. CELSR2 SNPs also showed association with dose variability during the stabilization phase. Also, rs646776 and rs12740374 were linked to warfarin sensitivity over the initiation phase. Only rs602633 was associated with INR treatment outcomes.Conclusion: The findings presented in this study found new pharmacogenomic associations for warfarin, that warrant further research in the field of genotype-guided warfarin dosing.

Keywords: warfarin, SNPs, pharmacogenetics, Jordan

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Gene Therapy Market is Projected to Reach $4,402 million by 2023 | Leading key players are Novartis, Kite Pharma, GlaxoSmithKline , Spark Therapeutics…

Posted: November 20, 2020 at 3:57 pm

The global gene therapy market was valued at $584 million in 2016, and is estimated to reach $4,402 million by 2023, registering a CAGR of 33.3% from 2017 to 2023. Gene therapy is a technique that involves the delivery of nucleic acid polymers into a patients cells as a drug to treat diseases. It fixes a genetic problem at its source. The process involves modifying the protein either to change the genetic expression or to correct a mutation. The emergence of this technology meets the rise in needs for better diagnostics and targeted therapy tools. For instance, genetic engineering can be used to modify physical appearance, metabolism, physical capabilities, and mental abilities such as memory and intelligence. In addition, it is also used for infertility treatment. Gene therapy offers a ray of hope for patients, who either have no treatment options or show no benefits with drugs currently available. The ongoing success has strongly supported upcoming researches and has carved ways for enhancement of gene therapy.

Top Companies Covered in this Report: Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc., Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica, NewLink Genetics Corp.

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The gene therapy market is a widely expanding field in the pharmaceutical industry with new opportunities. This has piqued the interests of venture capitalists to explore this market and its commercial potential. Major factors that drive the growth of this market include high demands for DNA vaccines to treat genetic diseases, targeted drug delivery, and high incidence of genetic disorders. However, the stringent regulatory approval process for gene therapy and the high costs of gene therapy drugs are expected to hinder the growth of the market.

The global gene therapy market is segmented based on vector type, gene type, application, and geography. Based on vector type, it is categorized into viral vector and non-viral vector. Viral vector is further segmented into retroviruses, lentiviruses, adenoviruses, adeno associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others. Non-viral vector is further categorized into naked/plasmid vectors, gene gun, electroporation, lipofection, and others. Based on gene type, the market is classified into antigen, cytokine, tumor suppressor, suicide, deficiency, growth factors, receptors, and others. Based on application, the market is divided into oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious disease, and other diseases. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

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Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: GENE THERAPY MARKET, BY VECTOR TYPE

CHAPTER 5: GENE THERAPY MARKET, BY GENE TYPE

CHAPTER 6: GENE THERAPY MARKET, BY APPLICATION

CHAPTER 7: GENE THERAPY MARKET, BY REGION

CHAPTER 8: COMPANY PROFILE

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Gene Therapy Market is Projected to Reach $4,402 million by 2023 | Leading key players are Novartis, Kite Pharma, GlaxoSmithKline , Spark Therapeutics...

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Solve suffering by blowing up the universe? The dubious philosophy of human extinction – The Conversation UK

Posted: November 20, 2020 at 3:57 pm

At a time when humans are threatening the extinction of so many other species, it might not seem so surprising that some people think that the extinction of our own species would be a good thing. Take, for example, the Voluntary Human Extinction Movement, whose founder believes that our extinction would put an end to the damage we inflict on each other and ecosystems more generally.

Or theres the South African philosopher David Benatar, who argues that bringing people into existence always does them harm. He recommends we cease procreating and gradually desert the Earth.

But humans arent the only beings to feel pain. Non-human animals would continue suffering without us. So, driven by a desire to eliminate suffering entirely, some people have shockingly advocated taking the rest of nature with us. They recommend that we actively abolish the world, rather than simply desert it.

This disturbing and extremist position goes surprisingly far back in history.

Around 1600 years ago, Saint Augustine suggested that humans stop procreating. He endorsed this, however, because he wanted to hasten the Last Judgement and the eternity of joy thereafter.

If you dont believe in an afterlife, this becomes a less attractive option. Youd have to be motivated exclusively by removing suffering from nature, without any promise of gaining supernatural rewards. Probably the first person to advocate human extinction in this way was Arthur Schopenhauer. He did so 200 years ago, in 1819, urging that we spare the coming generations of the burden of existence.

Schopenhauer saw existence as pain so he believed we should stop bringing humans into existence. And he was clear about the result if everyone obeyed: The human race would die out.

But what about the pain of non-human animals? Schopenhauer had an answer, but it wasnt a convincing one. He was a philosophical idealist, believing that the existence of external nature depends on our self-consciousness of it. So, with the abolition of human brains, the sufferings of less self-aware animals would also vanish as they ceased to exist without us around to perceive them.

Even on Schopenhauers own terms, theres a problem. What if other intelligent and self-conscious beings exist? Perhaps on other planets? Surely, then, our sacrifice would mean nothing; existence and painful perception of it would continue. It fell to Schopenhauers disciple, Eduard von Hartmann, to propose a more complete solution.

Hartmann, born in Berlin in 1842, wrote a system of pessimistic philosophy that was almost as lengthy as his impressive beard. Infamous in his own time, but completely forgotten in ours, Hartmann proposed a shockingly radical vision.

Writing in 1869, Hartmann rebuked Schopenhauer for thinking of the problem of suffering in only a local and temporary sense. His predecessors vision of human extinction by sexual continence would not suffice. Hartmann was convinced that, after a few aeons, another self-conscious species would re-evolve on Earth. This would merely perpetuate the misery of existence.

Hartmann also believed that life exists on other planets. Given his belief that most of it was probably unintelligent, the suffering of such beings would be helpless. They wouldnt be able to do anything about it.

So, rather than only destroying our own kind, Hartmann thought that, as intelligent beings, we are obligated to find a way to eliminate suffering, permanently and universally. He believed that it is up to humanity to annihilate the universe: it is our duty, he wrote, to cause the whole kosmos to disappear.

Hartmann hoped that if humanity did not prove up to this task then some planets might evolve beings that would be, long after our own sun is frozen. But he didnt think this meant we could be complacent. He noted the stringency of conditions required for a planet to be habitable (let alone evolve creatures with complex brains), and concluded that the duty might fall exclusively on humans, here and now.

Hartmann was convinced this was the purpose of creation: that our universe exists in order to evolve beings compassionate and clever enough to decide to abolish existence itself. He imagined this final moment as a shockwave of deadly euthanasia rippling outwards from Earth, blotting out the existence of this cosmos until all its world-lenses and nebulae have been abolished.

He remained unclear as to exactly how this goal would be achieved. Speaking vaguely of humanitys increasing global unification and spiritual disillusion, he hinted to future scientific and technological discoveries. He was, thankfully, a metaphysician not a physicist.

Hartmanns philosophy is fascinating. It is also unimaginably wrong. This is because he confuses the eradication of suffering with the eradication of sufferers. Conflating this distinction leads to crazy visions of omnicide. To get rid of suffering you dont need to get rid of sufferers: you could instead try removing the causes of pain. We should eliminate suffering, not the sufferer.

Indeed, so long as there are intelligent beings around, theres at least the opportunity for a radical removal of suffering. Philosophers such as David Pearce even argue that, in the future, technologies like genetic engineering will be able to entirely phase it out, abolishing pain from the Earth. With the right interventions, Pearce contends, humans and non-humans could plausibly be driven by gradients of bliss, not privation and pain.

This wouldnt necessarily need to be a Brave New World, populated by blissed-out, stupefied beings: plausibly, people could still be highly motivated, just by pursuing a range of sublime joys, rather than avoiding negative feeling. Pearce even argues that, in the far future, our descendents might be able to effect the same change on other biospheres, throughout the observable universe.

So, even if you think removing suffering is our absolute priority, there is astronomical value in us sticking around. We may owe it to sufferers generally.

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Avrobio tracks improvements in first patient treated with Gaucher gene therapy – FierceBiotech

Posted: November 19, 2020 at 10:58 am

Avrobio has shared data on the first Gaucher disease patient to receive its gene therapy AVR-RD-02. The patient, who was stable on enzyme replacement therapy at baseline, experienced a 22% drop in a toxic metabolite after receiving AVR-RD-02 and stopping taking the standard of care.

Gaucher, like the Fabry disease targeted by Avrobios lead prospect, is currently treated using enzyme replacement therapies sold by Sanofi and Takeda, which entered the market through its takeover of Shire. However, a significant minority of patients experience physical limitations despite treatment. Negative outcomes include bone pain and spleen enlargement. Johnson & Johnsons Zavesca offers an oral alternative, but there remain unmet medical needs.

Avrobio is developing AVR-RD-02 to address those needs. The data shared as part of Avrobios R&D day mark the start of the effort to show AVR-RD-02 performs as hoped in the clinic.

The first patient to receive AVR-RD-02 discontinued enzyme replacement therapy one month before taking the gene therapy. Three months after receiving the gene therapy, levels of Gaucher biomarker lyso-Gb1 had fallen 22%. The patients level of plasma chitotriosidase, a biomarker of cells associated with severe organ damage, was down 17%. Hemoglobin and platelets were in the normal range.

AVR-RD-02 triggered those changes without causing serious adverse events. The data drop offers an early indication that Avrobio may be able to improve outcomes by harvesting hematopoietic stem cells, adding a gene that encodes for glucocerebrosidase and reinfusing the cells back into the same patient. With enzyme replacement therapies costing healthcare systems up to $400,000 a year per patient, there is scope for AVR-RD-02 to cut the cost of treating Gaucher disease.

Avrobio shared the early look at clinical data on AVR-RD-02 alongside updates about other assets. There is now more than three years of data on some Fabry patients treated with Avrobios lead asset, putting the company in a position to plot a path to accelerated approval. Avrobio plans to submit its briefing book to the FDA by the end of the year to align on an accelerated approval strategy.

The update also covered cystinosis candidate AVR-RD-04. The first patient to receive the candidate is off oral and eye drop cysteamine 12 months after receiving the gene therapy. The number of crystals in the patients skin are down 56%, leading Avrobio to posit they may have gained the ability to make their own functional cystinosin protein.

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Global Gene Therapy Industry – GlobeNewswire

Posted: November 19, 2020 at 10:58 am

New York, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Industry" - https://www.reportlinker.com/p05817594/?utm_source=GNW 6% in the year 2020 and thereafter recover and grow to reach US$3.3 billion by the year 2027, trailing a post COVID-19 CAGR of 19.5% over the analysis period 2020 through 2027. Governments worldwide are focusing all healthcare resources on fighting the global pandemic. Billions of dollars have poured into researching COVID-19 drugs, therapies and vaccines. Over US$8 billion globally excluding the U.S. has been pledged only for vaccine development. The U.S. has independently pumped billions of dollars into COVID-19 research and response. The massive reallocation of funds and reprioritization of efforts has left a glaring gap in other sectors of healthcare. Gene therapy which holds promise for treating cancer, cystic fibrosis, heart disease, diabetes, hemophilia & AIDS, is slumping due to lack of research funds & reduced footfall of patients seeking treatment. Given the complex and fragile manufacturing and delivery system along with funding models of the industry, COVID-19 has emerged as a black swan event. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites. There are concerns regarding administration of cell and gene therapies. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centres. Treatments intended to be delivered into ICUs are being impacted by bed reservations made for patients with COVID-19 infection.

R&D and preclinical activities are also affected by supply shortages as a result of strong demand for consumables like reagents and PPE from COVID-19 laboratories. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrolments for protecting participants from the risk of infection. These issues are delaying activation of new sites, prompting players to postpone new clinical trials. However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. While companies targeting paediatric diseases suspended trials, others dealing with oncology maintained the pace. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. These issues are attributed to confluence of various factors like travel ban, withdrawal of several services from healthcare sites and the risk of virus transmission. In addition, these disruptions are anticipated to threaten existence of certain cell and gene therapy companies, particularly small-scale biotech players that are in pre-commercial phase and rely on external funding. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.

In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Gene therapies hold promise to improve the condition of patients where traditional cancer treatments such as radiation and chemotherapy are not effective. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. Oncology will remain the key area of focus for gene therapy applications. Cancer therapies represent the leading category, as is gauged through robust rise in the number of molecules being tested across numerous clinical trials. Novartis which recently bagged the U.S. FDA approval for Kymriah, a gene therapy designed for the treatment of hematological cancer, is seeking to gain commercial approval in established and emerging countries. Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals. More developmental focus will also be shed on monogenic rare diseases which have clearer genomic targets and the unmet need in smaller patient populations. Majority gene therapies so far have come to market through accelerated review pathways of regulatory authorities. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed. In the coming years, there will be significant improvement in the number of approvals for new gene therapies. The growth is anticipated to emerge from different modalities including RNAi, ASOs and CRISPR gene editing based therapeutics which offer long term opportunities for growth. These technologies are generating much excitement for investors.

Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=GNW

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE I-1

II. EXECUTIVE SUMMARY II-1

1. MARKET OVERVIEW II-1 A Prelude to Gene Therapy II-1 Classification of Gene Therapies II-1 Impact of Covid-19 and a Looming Global Recession II-2 COVID-19 Causes Gene Therapy Market to Buckle & Collapse II-2 COVID-19 Impact on Different Aspects of Gene Therapy II-2 Manufacturing & Delivery II-2 Research & Clinical Development II-3 Commercial Operations & Access II-3 Managing Derailed Operations II-4 Focus on Clinical Development Programs II-4 Targeting Manufacturing & Delivery Strategies II-4 Securing Supplies II-4 Remote Working II-4 Gene Therapy Set to Witness Rapid Growth Post COVID-19 II-5 By Vector Type II-5 VIRAL VECTORS ACCOUNT FOR A MAJOR SHARE OF THE MARKET II-5 Adeno-Associated Virus Vectors II-6 Lentivirus II-6 NON-VIRAL VECTORS TO WITNESS FASTER GROWTH II-7 US and Europe Dominate the Gene Therapy Market II-8 Oncology Represents the Largest Indication for Gene Therapy II-9 Market Outlook II-9 WORLD BRANDS II-10

2. FOCUS ON SELECT PLAYERS II-16 Recent Market Activity II-18 Select Innovations II-24

3. MARKET TRENDS & DRIVERS II-25 Availability of Novel Therapies Drive Market Growth II-25 Select Approved Gene Therapy Products II-26 Adeno-associated Virus Vectors - A Leading Platform for Gene Therapy II-27 Lentiviral Vectors Witness Increased Interest II-27 Rising Cancer Incidence Worldwide Spurs Demand for Gene Therapy II-28 Exhibit 1: Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 II-28 Exhibit 2: Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 II-29 Exhibit 3: Number of New Cancer Cases and Deaths (in Million) by Region for 2018 II-30 Compelling Level of Technology & Innovation to Ignite Gene Therapy II-30 Promising Gene Therapy Innovations for Treatment of Inherited Retinal Diseases II-31 Gene Therapy Pivots M&A Activity in Dynamic Domain of Genomic Medicine II-31 M&As Rampant in Gene Therapy Space II-31 Gene Therapy Deals: 2018 and 2019 II-32 Emphasis on Formulating Robust Regulatory Framework II-33 Strong Gene Therapy Pipeline II-33 Gene Therapy: Phase III Clinical Trials II-33 OHSU Implements First-Ever LCA10 Gene Therapy Clinical Trial with CRISPR II-35 Growing Funding for Gene Therapy Research II-35 Market Issues & Challenges II-35

4. GLOBAL MARKET PERSPECTIVE II-37 Table 1: World Current & Future Analysis for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-37

Table 2: World Historic Review for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-38

Table 3: World 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2017, 2020 & 2027 II-39

Table 4: World Current & Future Analysis for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-40

Table 5: World Historic Review for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-41

Table 6: World 10-Year Perspective for Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-42

Table 7: World Current & Future Analysis for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-43

Table 8: World Historic Review for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-44

Table 9: World 10-Year Perspective for Non-Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-45

Table 10: World Current & Future Analysis for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-46

Table 11: World Historic Review for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-47

Table 12: World 10-Year Perspective for Oncological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-48

Table 13: World Current & Future Analysis for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-49

Table 14: World Historic Review for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-50

Table 15: World 10-Year Perspective for Rare Diseases by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-51

Table 16: World Current & Future Analysis for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-52

Table 17: World Historic Review for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-53

Table 18: World 10-Year Perspective for Neurological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-54

Table 19: World Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-55

Table 20: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-56

Table 21: World 10-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-57

III. MARKET ANALYSIS III-1

GEOGRAPHIC MARKET ANALYSIS III-1

UNITED STATES III-1 Table 22: USA Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-1

Table 23: USA Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-2

Table 24: USA 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-3

Table 25: USA Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-4

Table 26: USA Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-5

Table 27: USA 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-6

CANADA III-7 Table 28: Canada Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-7

Table 29: Canada Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-8

Table 30: Canada 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-9

Table 31: Canada Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-10

Table 32: Canada Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-11

Table 33: Canada 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-12

JAPAN III-13 Table 34: Japan Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-13

Table 35: Japan Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-14

Table 36: Japan 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-15

Table 37: Japan Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-16

Table 38: Japan Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-17

Table 39: Japan 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-18

CHINA III-19 Table 40: China Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-19

Table 41: China Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-20

Table 42: China 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-21

Table 43: China Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-22

Table 44: China Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-23

Table 45: China 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-24

EUROPE III-25 Table 46: Europe Current & Future Analysis for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 III-25

Table 47: Europe Historic Review for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-26

Table 48: Europe 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2017, 2020 & 2027 III-27

Table 49: Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-28

Table 50: Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-29

Table 51: Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-30

Table 52: Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-31

Table 53: Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-32

Table 54: Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-33

FRANCE III-34 Table 55: France Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-34

Table 56: France Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-35

Table 57: France 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-36

Table 58: France Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-37

Table 59: France Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-38

Table 60: France 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-39

GERMANY III-40 Table 61: Germany Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-40

Table 62: Germany Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-41

Table 63: Germany 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-42

Table 64: Germany Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-43

Table 65: Germany Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-44

Table 66: Germany 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-45

ITALY III-46 Table 67: Italy Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-46

Table 68: Italy Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-47

Table 69: Italy 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-48

Table 70: Italy Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-49

Table 71: Italy Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-50

Table 72: Italy 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-51

UNITED KINGDOM III-52 Table 73: UK Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-52

Table 74: UK Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-53

Table 75: UK 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-54

Table 76: UK Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-55

Table 77: UK Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-56

Table 78: UK 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-57

REST OF EUROPE III-58 Table 79: Rest of Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-58

Table 80: Rest of Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-59

Table 81: Rest of Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-60

Table 82: Rest of Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-61

Table 83: Rest of Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-62

Table 84: Rest of Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-63

ASIA-PACIFIC III-64 Table 85: Asia-Pacific Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-64

Table 86: Asia-Pacific Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-65

Table 87: Asia-Pacific 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-66

Table 88: Asia-Pacific Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-67

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Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR001 – GlobeNewswire

Posted: November 19, 2020 at 10:58 am

NEW YORK, Nov. 18, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) onNovember 17, 2020issued a composition of matter patent, U.S. Patent No. 10,837,028,with claims directed to the AAV vector used in PR001, Prevails experimental gene therapy program for the treatment of Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). The base patent term extends until October 3, 2038, excluding patent term extensions or coverage in additional related patent filings.

We are excited to make important progress this year with PR001, which is being evaluated in the Phase 1/2 PROPEL trial for patients with Parkinsons disease with GBA1 mutations and in the Phase 1/2 PROVIDE trial for patients with Type 2 Gaucher disease, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. We are advancing clinical development of PR001 to make a potentially transformative difference for these patients who currently have no approved treatment options.

The Company recently announced that patient dosing has continued in the Phase 1/2 PROPEL clinical trial of PR001 for PD-GBA patients, and it expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021. The Company expects to initiate enrollment of the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease patients in the fourth quarter of 2020 and currently anticipates it will provide the next update on PR001 biomarker and safety data for nGD in 2021.

The U.S. Food and Drug Administration has granted Fast Track designations for PR001 for the treatment of PD-GBA and nGD. In addition, the FDA granted PR001 Rare Pediatric Diseasedesignation for the treatment of nGD, and Orphan Drugdesignation for the treatment of patients with Gaucher disease.

About Prevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinsons disease with GBA1mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia withGRNmutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr.Asa Abeliovichin 2017, through a collaborative effort withThe Silverstein Foundationfor Parkinsons with GBA and OrbiMed, and is headquartered inNewYork, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevails gene therapy candidates to make a transformative difference for patients with neurodegenerative diseases; the expected timing of reporting additional interim data on a subset of patients from the PROPEL trial; and the anticipated timing of enrollment of and the next update on data from the PROVIDE trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors sections of the Companys most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Lisa QuTen Bridge Communications LQu@tenbridgecommunications.com678-662-9166

Investor Contact:investors@prevailtherapeutics.com

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Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR001 - GlobeNewswire

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Orgenesis CEO talks disruption: ‘We are the Uber of the cell and gene therapy space’ – BioPharma-Reporter.com

Posted: November 19, 2020 at 10:58 am

Maryland, US headquartered company, Orgenesis, is championing a model that aims to bring down those costs it works with partner hospitals throughout the commercialization process.

The companys CGT platform, consisting of a pipeline of licensed cell and gene therapies, scientific expertise, customised processing systems, and an ecosystem of healthcare providers and research institutes, is designed to provide a pathway for groundbreaking autologous therapies to become commercially available on an industrial scale and at prices accessible to large populations.

Orgenesis business model is one focused on decentralization, enabling precision medicines to be prepared on-site at hospitals. In this way, we can really expedite cell and gene therapy development, said Orgenesis CEO, Vered Caplan.

With operations in the US, Europe, Israel and South Korea, Orgenesis has now created an international network of point of care (POCare) centers to serve patients directly in the hospital setting.

Beyond the US, we have POCare centers in many countries in Europe such as Greece, the Netherlands, Belgium, Slovenia, Italy and Spain; we also have centers in Israel, in Korea and in India and we will be starting up soon in Dubai,said the CEO.

The goal is to make gene and cell therapies feasible for large numbers of patients, said Caplan. We used to work as a contract development and manufacturing organization (CDMO) but we sold that business to Catalent at the beginning of the year.

The centralized processing and supply chain model only served to create a frustrating working environment, with plenty of constraints, said the Orgenesis lead.

We realized very quickly that we couldnt really ramp up to large scale relying on that kind of centralized model, particularly for autologous products, which represent most of the market today. It takes six months to train someone to work in a high-grade cleanroom there is a lot of work and expense involved in that and there is a limited number of patients that can be treated in such cleanrooms the utilization rate is very low - it [centralized processing and supply] is a very inefficient and costly way to supply and to develop medicine there is so much manual work involved, she told BioPharma-Reporter.

The company had been working for a number of years, investing a huge amount of effort in developing a range of automation solutions to supplant those manual processes, as well as building its mobile CGT processing labs and units (OMPULs), she said.

We had been fielding so many requests from hospitals that wanted to collaborate with us, asking us to make or scale up their CAR-T and other therapies. We realized that in order to get this done, we needed to take a decentralized approach and that we needed to provide a solution, not only for one hospital, but for every hospital that wanted these type of therapies; and we saw that such a model brings down the price of the therapy tremendously.

A hospital gives Orgenesis a license to work on the therapy, on the processing; production of the final product is automated and supplied via an on-site point-of-care processing unit. Orgenesis then sets about democratizing the treatment,making it available to any hospital in its POCare network.

The company says the final customized, automated processing system it has developed, with the integrated specific therapy, solves a variety of processing and cost hurdles. It results in a lower required grade of cleanroom, it simplifies facility management requirements, it enables multi-batch processing per cleanroom, which means reduced technical staffing. Moreover, the localized processing eliminates the many logistical difficulties associated with traditional, centralized manufacturing and transport.

Overall, it is said to provide faster turnaround, increased safety, and improved quality control management on-site.

Hospitals really want to supply CGTs, while patients are reading about such treatments and making inquiries of healthcare providers, she added.

Ours is really a combined licensing and service model.

We are like Uber. If you have a car, you want to make some extra revenue, you call up Uber and it gives you the network, the technology and all the operating procedures to be a taxi driver. That is very much what we do in terms of hospitals we give them the ability to be biotech companies, because this is not the standard thing they do, they dont want to take responsibility for cell and gene therapy it is too much for them. They want to treat patients, but they want to have that local supply, so we give them the technology and the capabilities to do that. We give them regulatory support for clinical trials, we give them CRO support, we give them a network - so they can function and do what they need to do, which is to undertake research and treat patients.

Orgenesis intends to leverage its network of regional partners to advance the development and commercialization of its therapeutic pipeline. Towards this end, it said its partners have committed to funding the clinical programs. In turn, the company typically grants its partners geographic rights in exchange for future royalties, and a partnership with Orgenesis to support the supply of the targeted therapies. Through this model, Orgenesis has already signed contracts, which it expect to generate over US$40M in revenue over the next three years, if fully realized.

On the therapeutic front, Orgenesis is focused on several key verticals, including immuno-oncology, anti-viral, and metabolic/auto-immune diseases.

It recently acquired Koligo Therapeutics, with the aim of leveraging Koligos 3D-V bioprinting technology across its POCare Platform. That technology, which utilizes 3D bioprinting and vascularization with autologous cells to create biodegradable and shelf-stable three-dimensional cell and tissue implants, is being developed for diabetes and pancreatitis, with longer term applications for neural, liver, and other cell/tissue transplants.

In February this year, Orgenesis announced that it has entered into a collaboration agreement with the John Hopkins University to utilize the POCare platform to develop and supply a variety of CGTs including cell-based immunotherapy technologies.

And the University of California, Davis (UC Davis) joined its POCare network in January. The collaboration will involve the scale up and integration of UC Davis lentiviral vector process.

Today we are very much in validation mode. Most of the therapies in this space, and the ones we have licensed from the hospitals I think we have about 25 today are all at different stages of clinical development. Some have been used to treat patients but that has all been done under hospital exception.

When we adopt a therapy into the network, we run it through the entire R&D, formal clinical and regulatory processes as [our goal] is a harmonized process, to have the same standard [in our closed systems] at our [POCare] centers, whether that is in Germany or Korea, said the CEO.

The rest is here:
Orgenesis CEO talks disruption: 'We are the Uber of the cell and gene therapy space' - BioPharma-Reporter.com

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