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Archive for the ‘Crispr’ Category

Quest to Use CRISPR Against Disease Gains Ground – Scientific American

The prospect of using the popular genome-editing tool CRISPR to treat a host of diseases in people is moving closer to reality.

Medical applications of CRISPRCas9 had a banner year in 2019. The first results trickled in from trials testing the tool in people, and more trials launched. In the coming years, researchers are looking ahead to more sophisticated applications of CRISPR genome editing that could lay the foundation for treating an array of diseases, from blood disorders to hereditary blindness.

But although the results of clinical trials of CRISPR genome editing so far have been promising, researchers say that it is still too soon to know whether the technique will be safe or effective in the clinic.

Theres been a lot of appropriate caution in applying this to treating people, says Edward Stadtmauer, an oncologist at the University of Pennsylvania in Philadelphia. But I think were starting to see some of the results of that work.

It has only been seven years since researchers discovered thata molecular defence system called CRISPRCas9, which microbes use to fend off viruses and other invaders, could beharnessed to rewrite human genes.

Since then gene-editing has attracted attention for its potential to modify embryosan application that is ethically and legally fraught if those embryos are destined tobecome human beings. But in parallel, scientists have been testing CRISPR's much less controversial ability to disable or correct problematic genes in other cells in order to treat a host of diseases.

In 2016, Chinese researchers announced that they hadtreated the first person with a CRISPRCas9 therapydesigned to fight cancer. In cells extracted from a participant's blood, the researchers disabled the gene that codes for a protein called PD-1,which holds the immune system in checkbut can shield cancer cells in the process. The scientists then reinjected the cells.

By 2019, the US governments clinicaltrials.gov database listed more than a dozen active studies that are testing CRISPRCas9 as a treatment for a range of diseases from cancer to HIV and blood disorders.

So far, too few people have been treated in these trials to draw any firm conclusions about the safety of CRISPRCas9 therapies or how well they work. Preliminary results from two trialsone in which gene-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancershowed no signs of clinical improvement.

In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. In the man treated for HIV, the researchers attempted to use CRISPR to disable a protein that many strains of HIV use to enter cells. But only 5% of the transplanted cells were editednot enough to cure disease, the researchers reported in September. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University in Beijing and a lead author of the work.

There are early hints that another trial might meet with more success. CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, have treated two people with the genetic disorders sickle-cell anaemia and -thalassaemia. Both deplete oxygen-carrying haemoglobin molecules in the blood: the idea is to use CRISPR to disable a gene that otherwise shuts off production of another form of haemoglobin. Early results suggest that the treatment might have eased some symptoms of the disorders, but the participants will need to be followed for a longer period to be sure.

Other researchers are already itching to move beyond editing cells in a dish. The challenge is in finding ways to transport the gene-editing machinery to where it is needed in the body, says John Leonard, chief executive of Intellia Therapeutics, a biotechnology company in Cambridge, Massachusetts, that is focused on CRISPRCas9 genome editing. The delivery approach is so important.

Last July, the pharmaceutical companies Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin launched a trial to treat the genetic disorder Leber congenital amaurosis 10, which can cause blindness, by editing eye cells. Researchers will inject into the eye a virus containing DNA that encodes the CRISPR genome-editing machinery, bypassing the need to guide those tools through the bloodstream to the specific tissues. The virus will be responsible for carrying the genome-editing tools into cells. It is the first trial to attempt CRISPRCas9 gene editing inside the body, and early results could be reported this year.

That would be a landmark moment for the field, and could pave the way for future trials targeting other organs, says Charles Gersbach, a bioengineer at Duke University in Durham, North Carolina. But he and others say that they hope researchers will eventually move away from using viruses to shuttle genome-editing machinery into cells. Deactivated viruses can still sometimes provoke immune responses, and can only carry a limited amount of DNA.

What's more, some gene-editing tools are currently too large to fit inside commonly used gene-therapy viruses, says chemical biologist Andrew Anzalone at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. These include the souped-up CRISPR systems calledprime editorsthat were first reported in late 2019and might prove to be more precise and controllable than CRISPRCas9.

Intellia is looking for a way around the viruses. The company has partnered with Swiss pharmaceutical giant Novartis to develop fatty nanoparticles that can protect genome-editing molecules as they travel through the bloodstream, but also pass through the membranes of target cells.

These particles tend to accumulate in the liver, and researchers are working to develop particles that infiltrate other tissues, such as muscle or the brain. But for now, Intellia will focus on liver diseases, says Leonard, and plans to launch its first trial of the technology this year. Its crawl before you walk, so to speak, he says.

None of the technologies currently being tested is what researchers envision for the long-term applications of genome-editing, says Gersbach. The approaches that people are taking are the things that we can do today, he says, but not what we would do if we could design the ideal drug.

Leonard says that when he meets with investors, they often demand to know what medical advances will be made in the next six months. We do our best to describe that, but I always end it by saying, Can you imagine a future without gene editing? he says. I have yet to meet the person who says, yes.

This article is reproduced with permission and wasfirst publishedon January 6, 2020.

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Quest to Use CRISPR Against Disease Gains Ground - Scientific American

Welcome to the bioengineering culture clash – STAT

Bioengineering, once viewed primarily as an academic discipline, is growing up.

Our ability to engineer biology is on the verge of changing the landscape of health and health care. Tools and treatments that are engineered, not discovered CAR-T therapies for cancer, CRISPR for gene editing, stem cell therapies, and more are now making their way not just into new startups but into established industry. Just look at the first-generation CAR-T companies that have been acquired by major biopharma companies, like Bristol-Myers Squibb/Celgene acquiring Juno or Gilead acquiring Kite.

The acquirers, massive organizations built on the foundations of discovery, are now ingesting companies built with engineering DNA. These are two extremely different mindsets. For decades, biopharma companies essentially used scientists to build products, because there was no means to engineer them. The intersection of these worlds is driving us into the future.

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Here come the culture clashes.

These emerge every time major new technologies disrupt an industry. Think of the early days of oil and the evolution as the industry matured from wildcatters one man, one rig, one highly risky and unreliable process to Rockefellers and Rothschilds and a highly sophisticated engineering and discovery process that used every advanced technology and enterprise tool available.

In biopharma and health care today, the old culture of discovery the idea that science is driven by discovering new knowledge (hypothesis > test > repeat) is clashing with the new culture of engineering (design > test > iterate). This clash encompasses how everything is handled, from identifying biological targets to designing clinical trials and even to how we access health care.

Knowing that these clashes are coming will help smooth the way as the biopharma industry integrates bioengineering deeper and more broadly. I see four key clashes worth noting.

We cant yet take for granted a common understanding that we can engineer biology. In spite of bioengineering departments flourishing at revered institutions like Harvard and MIT and Stanford and Berkeley, in spite of the success of new tools like CAR-T and CRISPR, some think that bioengineering is either hype or a passing fad. Thats OK; every new field struggles with the old guard.

Bioengineering still needs to come from a place of clearly and repeatedly explaining its worth with evidence. Lets just get used to this. That said, naysayers are predisposed to cling to old-school approaches. After all, thats the value they have to offer. Ultimately, they will need to adapt to an engineering approach or get engineered out of the process.

The culture of discovery and the culture of engineering value progress differently. Discovery, for example, prioritizes the Eureka! moment above all. One of the most challenging aspects of drug development is that you cant establish a key performance indicator for such moments. Pure discovery is a lottery ticket business that exists in the biopharma industry only because of its incredibly high value and the potential to save millions of lives.

Engineering, on the other hand, values a repeatable process, one that can be stacked or adjusted the way we build with Legos, aiming for compounding results a consistent fractional improvement year over year, leading to exponential improvement over time.

Health care needs both approaches. Todays great discovery will be engineerable tomorrow (OK, maybe 50 years from now). CRISPR, for example, began with discoveries in Haloferax mediterranei, a species of salt-tolerant bacteria. That was pure scientific discovery. But using CRISPR as a tool, as a therapeutic, or as a platform for future innovations is squarely in the world of bioengineering.

The choice of staying or leaving is now at the heart of the debate about what engineering can or cant handle, and what should remain pure, unfettered empirical discovery.

The truth is that engineering can handle empirical approaches. For example, is an A/B test discovery or is it engineering? Its actually both discovery done via an engineering process with iteration. Because biology is so incredibly sophisticated and complex, there will always be discovery risk the risk that some heretofore unknown aspect of biology will lead to failure. But part of engineering is, and should be, handling discovery and failure, and discovery can and should be engineered.

Tools like artificial intelligence and machine learning allow us to introduce to the world of discovery faster throughput, faster iteration, and greater reproducibility. We need to know how and where to apply engineering and discovery frameworks, and where the two worlds meet. Is your discovery risk one where you must wait for serendipity, or can you improve your odds by engineering some part of it?

The discovery and engineering cultures speak something that sounds like the same language, but really isnt. Words like discovery and platform mean very different things in science than they do in engineering. Even success doesnt directly translate: Does it work and we know how we got there, or did we get there in a repeatable process we can tweak?

Getting lucky with a serendipitous discovery is not success in an engineering discipline, nor is getting unlucky a failure in engineering, since you can learn something valuable from failure with which to tweak the process. Like any language issue, we need to recognize the different meanings in those core concepts and know when to use which depending on the world you are in.

Integrating these cultures requires each side to understand core tools, language, and mindsets in both worlds, and knowing where to leverage the differences. Where can discovery yield new empirical information for an engineered process? Where can an engineered process increase the odds of success over a more traditional discovery route?

In health care, as more and more products and tools become engineerable, the world of discovery will need to transition toward integrating engineering. This will be bumpy and uncomfortable and lets be real, there will be blood. But there will also be many bright spots. There will be people trained in science who, when introduced to engineering, feel they can tap into a new world of possibility. There will be engineers who maybe started their careers because as kids they dreamed of engineering trains who suddenly feel they have the potential to help cure cancer.

The audacious dream of engineering biology on a molecular scale is finally being realized not just in practice but commercially if we can surmount the culture clashes.

Vijay Pande, Ph.D., is a general partner at Andreessen Horowitz, a Silicon Valley venture capital firm, and an adjunct professor of bioengineering at Stanford University. He serves on the boards of Apeel Sciences, Asimov, BioAge, Ciitizen, Devoted Health, Freenome, Insitro, Omada, and PatientPing.

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Welcome to the bioengineering culture clash - STAT

Here’s Why CRISPR Therapeutics Stock Jumped 113.2% in 2019 – Motley Fool

What happened

Shares of CRISPR Therapeutics (NASDAQ:CRSP) rose over 113% last year, according to data provided by S&P Global Market Intelligence. The pharma stock built momentum throughout much of the year, but surged in October ahead of an important data presentation that ultimately lived up to the hype. That allowed the gene-editing stock to easily outperform the 28.8% gain of the S&P 500 in 2019.

The end-of-year rally was driven by promising clinical results for its lead drug candidate. The first two individuals, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), dosed with CTX001 achieved functional cures after receiving an initial dose of the gene-editing product. The results need to be proven durable and replicated in a larger number of patients, but the update was about as good as investors could have hoped for at the current stage of development.

Image source: Getty Images.

Both SCD and TDT are caused by structural abnormalities in red blood cells. But these are one of the few cells in the human body that don't contain DNA. That means CRISPR Therapeutics has to harvest stem cells from the bone marrow of patients, apply gene editing to those extracted cells, and then inject the engineered stem cells back into patients (the ex vivo method). If the therapy works, then the engineered stem cells should produce functional red blood cells and potentially result in a cure.

In the early study, the ex vivo approach of CTX001 appeared to do just that. The TDT patient required an average of 16.5 blood transfusions per year in the two years before the clinical trial. Nine months after receiving the gene-editing treatment, the individual was transfusion independent (compared with an expected 12 transfusions) and expressed working copies of hemoglobin on 99.8% of red blood cells.

The SCD patient experienced an average of seven vaso-occlusive crises (painful blockages of blood vessels caused by abnormally shaped red blood cells) per year in the two years before the clinical trial. Four months after receiving the gene-editing treatment, the individual reported no vaso-occlusive crises (compared with an expectation for two such episodes) and expressed working copies of hemoglobin on 94.7% of red blood cells.

The early success of CTX001 bodes well for the ex vivo approach of CRISPR Therapeutics and its partner Vertex Pharmaceuticals(NASDAQ:VRTX), but investors should be careful not to extrapolate the results too broadly. Gene-editing tools that are applied inside the body (in vivo) face significantly steeper obstacles, such as the difficulty of delivering gene-editing payloads to specific tissue types inside the body. There's also the elephant in the room: Scientists are beginning to realize that current-generation CRISPR gene-editing tools don't work all that well.

Nonetheless, CRISPR Therapeutics is the top CRISPR-based gene-editing stock on the market. It has the cash, the partnerships, and the early results to back up its claim to that label.

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Here's Why CRISPR Therapeutics Stock Jumped 113.2% in 2019 - Motley Fool

CRISPR Cuts Through Layers of Butterfly Wing-Pattern Evolution – Entomology Today

The bright wing patterns in butterflies in the genus Heliconius make for a highly visible palette to use CRISPR gene-editing techniques to investigate the evolution of wing patterns and the genes that influence them, say researchers at the Smithsonian Institute of Tropical Research. (Photo credit: Sebastian Menas/Instagram @mena_sebas)

To help shed light on some of these largest questions remaining in evolutionary biology, the authors of a paper published recently in Current Biology paper narrowed in on one evolutionary storyMllerian mimicry in Heliconius butterflies.

Laura Kraft

Heliconius butterflies synthesize compounds from the family of the cyanides and also feed on plants that have these compounds. So, they are very distasteful for predators, especially birds, says Carolina Concha, Ph.D., Biodiversity Genomics Fellow at the Smithsonian Institute of Tropical Research. In order to advertise their toxicity, these butterflies have evolved bright red, orange, yellow and blue warning colors with strong black banding. Unfortunately for the butterflies, birds typically have to bite and kill a few butterflies before learning that bright colors mean bad tastes. So over time, distantly related yet equally distasteful species of these butterflies have converged to have the exact same wing color and pattern so that both convergent species can benefit from an increased warning signal. This causes birds to learn faster by feeding on fewer individuals from either species.

These bright color patterns in Heliconius butterflies have evolved and diverged into a number of species found in Central and South America over the last 12 to 14 million years, but it was only within very recent evolutionary historythe last 2.5-4.5 million yearsthat some distantly related distasteful butterflies converged to have the same patterns. Says Concha, The wing patterns have diversified so much in such a short time, so you have these really diverse forms within a single species and at the same time, you have very distantly related species that converge in a single phenotype.

Carolina Concha, Ph.D., shows off the stack of display boxes of CRISPR-mutation butterflies that she and her collaborators have created in the Smithsonian Institute of Tropical Research in Gamboa, Panama. (Photo credit: Laura Kraft)

CRISPR allows us to make the same butterflies, with one gene missing. Its a way to interrogate nature and just ask What is the function of this gene? and Did it change during evolution?' says Arnaud Martin, author on the Current Biology paper and Assistant Professor at George Washington University. One of the earliest genes to be expressed during butterfly wing development is called WntA (pronounced WIN- tah), and it is thought to be a major gene controlling the paint by number system used by butterflies to color and pattern their wings. WntA was once expressed during embryonic development of many different types of organisms, including vertebrates, but has been lost in most. Perhaps after its role in embryonic development had become downplayed in butterflies, WntA developed a new role in wing patterning, defining the boundaries of black and color banding patterns on adult Heliconius butterfly wings.

The researchers proposed to use CRISPR as scissors to precisely cut out the section of DNA that codes for the WntA gene in a few Mllerian co-mimic pairs. They had two complimentary hypotheses: If mimetic wing patterns developed in different butterflies from using a highly similar gene regulatory mechanism involving WntA, then knocking out the gene should result in the same wing pattern changes in mutant butterflies of the different species. If, on the other hand, identical wing patterns were caused by highly different pathways involving WntA, then the wings of two species of mutant butterflies may exhibit different patterns.

Carolina Concha, Ph.D., carefully injects CRISPR into Heliconius butterfly eggs. If she can inject the eggs within two hours after they are laid, she gets less mosaicism in the CRISPR mutation than with three- or four-hour-old eggs. (Photo credit: Luca Livraghi)

After injecting thousands of eggs, Concha finally got full CRISPR knockout of three co-mimetic pairs of Heliconius butterflies. I honestly thought that co-mimetic Heliconius species were generated by similar tweaks of the same developmental pathways. I was wrong, says Owen McMillan, Ph.D., Dean of Academic Programs at STRI. In all three co-mimetic pairs, the resulting mutant butterflies had dramatically different wing color patterning after removing the WntA gene, supporting the second hypothesis that even highly different pathways can lead to the same wing pattern.

CRISPR allowed us to push our basic understanding of the pathways underlying wing pattern formation in entirely new directions. We have made great progress in identifying the key genes underlying pattern formation, but CRISPR allows us, for the first time, to understand how they work. It is a remarkably cool tool for discovery, says McMillan.

While major changes have been made to the regulatory pathway of WntA, resulting in these wing patterning differences, they do all focus on the same gene. But the really surprising part of this paper is that despite these butterflies developing under different conditions and experiencing different evolutionary histories over millions of years, they still converged upon the same phenotype with a completely different network of gene regulation. This really highlights that the genome has a few favorite genetic tools that drive the evolution of specific parts of the anatomy, but the way these tools can be used is flexible, says Martin

If youd like to learn more, here is a video showing the method of using CRISPR to change butterfly wing patterning:

Laura Kraftis a Ph.D. student at North Carolina State University and a National Science Foundation Graduate Research Fellow. When she isnttraveling the world, she spends her time making science more accessible through science writing and outreach. Email:ljkraft@ncsu.edu.

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CRISPR Cuts Through Layers of Butterfly Wing-Pattern Evolution - Entomology Today

CRISPR Technology Market Insights and Forecast, 2019-2025 – Analysis of Biomedical, Agricultural and Industrial Applications – Yahoo Finance

Dublin, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The "Global CRISPR Technology Market 2019-2025" report has been added to ResearchAndMarkets.com's offering.

The global CRISPR technology market is expected to witness a significant growth rate during the forecast period.

Industry giants are working in CRISPR technology development and are investing in the R&D of the technology. Such investments are expected to create an opportunity for the growth of the market in the near future.

The global CRISPR technology market is segmented on the basis of application and end-user. Based on the application, the market is segmented into biomedical applications, agricultural applications, and industrial applications. In fruit crops, CRISPR technology has numerous applications as it improves the important agronomic traits such as biotic and abiotic stress tolerance and fruit quality.

Further, on the basis of end-user, the market is segmented into pharmaceutical & biopharmaceutical companies, and academic & research institutes. CRISPR being a really new technology seeks the interest of everyone from doctors to academic & research institutes. CRISPR holds a lot of hidden potentials to cure many rare and incurable diseases that are still to be discovered and is driving the academic & research institutes as an end-user segment to grow with a significant rate in the market.

Geographically, the market is segmented into four major regions; North America, Europe, Asia-Pacific and Rest of the world (RoW). Among these, North America is expected to hold a prominent position in the global CRISPR technology market. The presence of major pharma companies in the region tends to enhance the growth of the global CRISPR market.

Further, the report covers the analysis of several players operating in the market. Some of the players include Thermo Fisher Scientific Inc., Merck KGaA, GenScript Biotech Corp., Horizon Discovery Group PLC, CRISPR Therapeutics AG, and others.

The report covers:

Key Topics Covered

1. Report Summary 1.1. Research Methods and Tools1.2. Market Breakdown 1.2.1. By Segments1.2.2. By Geography

2. Market Overview and Insights2.1. Scope of the Report 2.2. Analyst Insight & Current Market Trends2.2.1. Key Findings2.2.2. Recommendations2.2.3. Conclusion2.3. Rules & Regulations

3. Competitive Landscape3.1. Company Share Analysis3.2. Key Strategy Analysis3.3. Key Company Analysis 3.3.1. Thermo Fisher Scientific, Inc. 3.3.1.1. Overview3.3.1.2. Financial Analysis3.3.1.3. SWOT Analysis3.3.1.4. Recent Developments3.3.2. Merck KGaA3.3.2.1. Overview3.3.2.2. Financial Analysis3.3.2.3. SWOT Analysis3.3.2.4. Recent Developments3.3.3. GenScript Biotech Corp.3.3.3.1. Overview3.3.3.2. Financial Analysis3.3.3.3. SWOT Analysis3.3.3.4. Recent Developments3.3.4. Horizon Discovery Group PLC3.3.4.1. Overview3.3.4.2. Financial Analysis3.3.4.3. SWOT Analysis3.3.4.4. Recent Developments3.3.5. CRISPR Therapeutics AG3.3.5.1. Overview3.3.5.2. Financial Analysis3.3.5.3. SWOT Analysis3.3.5.4. Recent Developments

4. Market Determinants4.1. Motivators4.2. Restraints4.3. Opportunities

5. Market Segmentation5.1. CRISPR Technology Market by Application5.1.1. Biomedical Applications5.1.2. Agricultural Applications5.1.3. Industrial Applications5.2. Global CRISPR Technology Market by End-User5.2.1. Pharmaceutical and Biopharmaceutical Companies5.2.2. Academic & Research Institutes

6. Regional Analysis6.1. North America6.1.1. United States6.1.2. Canada6.2. Europe6.2.1. UK6.2.2. Germany6.2.3. Italy6.2.4. Spain6.2.5. France6.2.6. Rest of Europe6.3. Asia-Pacific6.3.1. China6.3.2. India6.3.3. Japan6.3.4. Rest of Asia-Pacific6.4. Rest of the World

7. Company Profiles7.1. AstraZeneca PLC7.2. BASF SE7.3. Beam Therapeutics Inc.7.4. Bio-Rad Laboratories, Inc.7.5. Caribou Bioscience Inc.7.6. Cellectics SA7.7. Cibus, Ltd.7.8. CRISPR Therapeutics AG7.9. Danaher Corp.7.10. Editas Medicine7.11. GeneCopoeia inc.7.12. GenScript Biotech Corp.7.13. Horizon Discovery Group PLC7.14. Intellia Therapeutics Inc.7.15. Lonza Group Ltd.7.16. Merck KGaA7.17. New England Biolabs, Inc.7.18. Origene Technologies, Inc.7.19. Pairwise Plants7.20. Precision Bioscience, Inc.7.21. Sangamo Therapeutics Inc.7.22. Thermo Fisher Scientific, Inc.7.23. Transposagen Biopharmaceuticals, Inc.7.24. Tropic Biosciences UK LTD.7.25. Yield10 Bioscience, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/11g9aw

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CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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CRISPR Technology Market Insights and Forecast, 2019-2025 - Analysis of Biomedical, Agricultural and Industrial Applications - Yahoo Finance

CRISPR-edited crops reveal gene responsible for salt tolerance in rice – Genetic Literacy Project

Scientists from China National Rice Research Institute reported that FLN2, a gene that encodes fructokinase-like protein2, influences sugar metabolism as well as rice plants response to salinity stress. The results of their study are published in Biomolecules.

Several mutagenized rice lines were grown under high salinity conditions to pinpoint the genes needed for the expression of salinity tolerance. Some rice lines with mutation in FLN2 showed susceptibility to salinity stress. Wild-type rice lines exposed to salinity stress showed up-regulated FLN2, while CRISPR-Cas9-generated lines with dysfunctional FLN2 exhibited hypersensitivity to salinity stress. Furthermore, sugar metabolism was reduced in the knockout line than in wild-type plants. This may imply that the compromised salinity tolerance in FLN2 knockout plants was caused by the shortage in assimilate needed for growth.

The researchers concluded that FLN2 is vital in seedling growth as well as in tolerance to salinity stress.

Read full, original article: Crop Biotech Update January 8, 2020

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CRISPR-edited crops reveal gene responsible for salt tolerance in rice - Genetic Literacy Project

Shareholders Are Thrilled That The CRISPR Therapeutics (NASDAQ:CRSP) Share Price Increased 170% – Simply Wall St

CRISPR Therapeutics AG (NASDAQ:CRSP) shareholders might be concerned after seeing the share price drop 11% in the last month. But that doesnt undermine the rather lovely longer-term return, if you measure over the last three years. In three years the stock price has launched 170% higher: a great result. To some, the recent share price pullback wouldnt be surprising after such a good run. The fundamental business performance will ultimately dictate whether the top is in, or if this is a stellar buying opportunity.

View our latest analysis for CRISPR Therapeutics

Because CRISPR Therapeutics made a loss in the last twelve months, we think the market is probably more focussed on revenue and revenue growth, at least for now. Generally speaking, companies without profits are expected to grow revenue every year, and at a good clip. Thats because its hard to be confident a company will be sustainable if revenue growth is negligible, and it never makes a profit.

In the last 3 years CRISPR Therapeutics saw its revenue grow at 87% per year. Thats much better than most loss-making companies. Along the way, the share price gained 39% per year, a solid pop by our standards. But it does seem like the market is paying attention to strong revenue growth. Thats not to say we think the share price is too high. In fact, it might be worth keeping an eye on this one.

You can see below how earnings and revenue have changed over time (discover the exact values by clicking on the image).

While the share price may move with revenue, other factors can also play a role. For example, weve discovered 4 warning signs for CRISPR Therapeutics (of which 1 is major) which any shareholder or potential investor should be aware of.

Were pleased to report that CRISPR Therapeutics rewarded shareholders with a total shareholder return of 64% over the last year. That gain actually surpasses the 39% TSR it generated (per year) over three years. The improving returns to shareholders suggests the stock is becoming more popular with time. Shareholders might want to examine this detailed historical graph of past earnings, revenue and cash flow.

If you like to buy stocks alongside management, then you might just love this free list of companies. (Hint: insiders have been buying them).

Please note, the market returns quoted in this article reflect the market weighted average returns of stocks that currently trade on US exchanges.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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Shareholders Are Thrilled That The CRISPR Therapeutics (NASDAQ:CRSP) Share Price Increased 170% - Simply Wall St

SNACS: The FAANG Of The Roaring 20s – Yahoo Finance

The roaring 20s are upon us, and the investment opportunities set in front of us are exhilarating. This new decade has a lot in store for us with tech as the driving force behind it.

FAANG was the acronym that drove the stock market to continuously new highs over the past decade: Facebook (FB), Amazon (AMZN), Apple (AAPL), Netflix (NFLX), and Google aka Alphabet (GOOGL). This is an acronym that I am sure you are familiar with. These stocks exponential returns may be exhausted, and a new set of equities are ready to take their place. It is time to look for the new FAANG.

When assessing market-shifting companies, you need to look for firms with an exciting product offering characterized by longevity and a substantial total addressable market (TAM). Firms with savvy management teams that are able to navigate through both the best and worst times nimbly.

I have chosen a new acronym of stocks that I believe could change the world in the roaring 20s. The companies include Crispr (CRSP), Sea Limited (SE), Alibaba (BABA), Nvidia (NVDA), and Splunk (SPLK) or SCANS, as I like to call it.

Here I will give a brief introduction of each stock and explain why I believe these shares will drive the market in this new decade.

Sea Limited (SE)

Sea is the leading internet company in Southeast Asia and Taiwan. These economies are digitalizing at an exponential rate, and Sea is well-positioned to take on the quickly expanding addressable market. The company operates three market leader segments, including an ecommerce platform, a digital entertainment division, and a digital payment company (Shopee, Garena, and AirPay, respectively).

The internet economy in Southeast Asia has tripled in the past 5 years to $100 billion and is expected to triple again by 2025 to $300 billion. Sea is growing at an even faster rate, with year-over-year topline appreciation in the high triple-digit percentages as the company continues to take an increasing amount of market share.

Sea Limited is going to be the tech powerhouse that helps turn the third world economies of Southeast Asia and Taiwan into digitalized world markets.

CRISPR (CRSP)

CRISPR is a biomedical firm that is on the verge of changing the world. This company can edit an individuals DNA, an achievement that is going to change modern medicine. This technology could be used to cure almost any disease if it is successfully implemented. What CRISPRs gene therapy does is splice out the bad or disease driving DNA and add healthy strands. The company is also a leader in regenerative stem-cell medicine, which could save the lives of 100s of thousands.

CRISPR has an established portfolio of life-changing therapies in its pipeline at various stages of development. Hemoglobinopathy is the closest to commercially viable and is currently in clinical trials. If it passes clinical trials, I see this stock jumping substantially.

These shares are still a risky asset considering the possibility that none of its gene-therapies make it past the clinical stage. Based on early trials, it appears that the therapy does indeed work, and this potential has begun to be priced into CRSP. The stock has appreciated 350% since it went public in late 2016, and I believe that this is just the beginning of its growth. The ability to change an individuals DNA is going to change the world of medicine.

Alibaba (BABA)

The Amazonof the East has been driving substantial growth, but I dont believe that investors are correctly valuing Alibabas fundamentals. BABA is trading at roughly 1/3rd of Amazons forward P/E valuation (seen below), despite achieving wider margins, stronger profitable, and a greater growth outlook. Alibaba is operating in one of the worlds largest and fastest-growing consumer markets (China).

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Alibaba controls not only Chinas ecommerce market but also its cloud computing space with a 47% market share. Its cloud computing space has the most room to run as Chinas cloud infrastructure continues to expand at an exponential rate high double-digit to triple-digit percentages.

Alibaba still has some geopolitical risk due to the US-China trade war, but as far as this next decades biggest equity drivers, I would replace AZMN with BABA in my portfolio.

Nvidia (NVDA)

This is the most exciting chip maker in the world today. Nvidia is known for the invention of the GPU, which is a chip original purposed for image rendering, but Nvidia has taken its capabilities far beyond this. Nvidias chips are hyper-fast and slowly becoming smarter as the technology develops. Its chips are becoming a necessity in data centers and are an essential element of AI development. I believe that one of Nvidias integrated circuits will be apart of the first true AI, which is going to change the world.

Nvidia is also leveraging 5G with its anticipated cloud gaming platform. Like cloud computing is the future of business data and analytics, cloud gaming is the future of gaming. Nvidia is making a big bet in this field with its cloud platform, GeForce NOW. This platform allows gamers to use their Macs or PCs for gaming anywhere with the high-speed, low-latency technology of Nvidias GPUs without needing Nvidias hardware locally.

Nvidia is undoubtedly a company of the future, and despite its 4-digit gains over the past decade, I believe that this stock still has legs to run. I dont think that the company has scratched the surface of what its chips could do.

Splunk (SPLK)

Splunk is a platform that helps companies utilize real-time machine data for collection, indexing, and alerts, allowing companies to uncover actionable insight from this data no matter the source or format. The company is leveraging AI and machining learning for forecasting and anticipative decision making.

Real-time data management is becoming increasingly necessary in business across industries as this digital age makes speed a competitive advantage. Splunk is well-positioned to take on the massive addressable market that is yet to recruit Splunks services. This firm is well-suited to transform the way our economy utilizes real-time data.

Take Away

The market driving stocks will undoubtedly make excellent long-term investments for the roaring 20s. SCANS will be a force to be reckoned with in this next decade. Short term volatility in these stocks shouldnt cause you to shy away from their long-term potential. I believe we may be on the edge of a market correction, so if you are worried about short-term earnings, I may wait for a pullback. If you are a long-term investor that is willing to ride this decades waves, I wouldnt hesitate to pull the trigger on these stocks.

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Click to get this free report Splunk Inc. (SPLK) : Free Stock Analysis Report Sea Limited Sponsored ADR (SE) : Free Stock Analysis Report NVIDIA Corporation (NVDA) : Free Stock Analysis Report Netflix, Inc. (NFLX) : Free Stock Analysis Report Alphabet Inc. (GOOGL) : Free Stock Analysis Report Facebook, Inc. (FB) : Free Stock Analysis Report CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report Alibaba Group Holding Limited (BABA) : Free Stock Analysis Report Amazon.com, Inc. (AMZN) : Free Stock Analysis Report Apple Inc. (AAPL) : Free Stock Analysis Report To read this article on Zacks.com click here. Zacks Investment Research

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SNACS: The FAANG Of The Roaring 20s - Yahoo Finance

Why This Thematic Healthcare Could be a January Winner – ETF Trends

Due in large part to the J.P. Morgan Health Care conference in San Francisco, the biotechnology industrys marquee yearly confab, January is often a strong month for related equities and ETFs.

That conference can serve as a springboard for mergers and acquisitions activity and with the genomic space currently in the spotlight, an uptick in consolidation in that arena could benefit the Global X Genomics & Biotechnology ETF (Nasdaq: GNOM).

GNOM tracks the Solactive Genomics Index and seeks to invest in companies that potentially stand to benefit from further advances in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics, and biotechnology, according to Global X.

Companies are only eligible for inclusion if they generate at least 50% of their revenues from genomics related business operations. The index is market cap-weighted with a single security cap of 4.0% and a floor of 0.3%. The ETF provides exposure to CRISPR, gene editing and therapeutics companies. CRISPR, in particular, is an area to watch.

January is disproportionately represented both by a number of deals and dollar value over the past 5 years, Evercore ISI analyst Josh Schimmer wrote in a note out Wednesday morning, reports Josh Nathan-Kazis for Barrons. January has seen as high as 33% of a years total deals (5/15 in Jan 2018) and as high as 48% of a years total dollar value ($36bn/$76bn in Jan 2017).

GNOM tries to help investors take on a thematic multi-capitalization exposure to innovative elements that cover advancements in gene therapy bio-informatics, bio-inspired computing, molecular medicine, and pharmaceutical innovations. These advancements can also translate over to growth potential, potentially providing investors with long-term alpha with low correlation relative to traditional growth strategies.

Entering 2020, will companies look to keep their heads down with modest guidance? Schimmer wrote, according to Barrons. If so, we might see another choppy month, although the macro setup is quite different this time around with expectations around conservative price hikes already in sentiment.

For more thematic investing ideas, visit our Thematic Investing Channel.

The opinions and forecasts expressed herein are solely those of Tom Lydon, and may not actually come to pass. Information on this site should not be used or construed as an offer to sell, a solicitation of an offer to buy, or a recommendation for any product.

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Why This Thematic Healthcare Could be a January Winner - ETF Trends

Intellia Therapeutics Highlights Recent Progress and Anticipated 2020 Milestones – GlobeNewswire

CAMBRIDGE, Mass., Jan. 09, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today provided an update on recent progress and the Companys 2020 priorities and expected milestones.

2020 will be a significant year for Intellia, as we execute on our full-spectrum strategy. With milestones anticipated across our pipeline, we are making important progress towards the development of curative treatments for severe diseases. In particular, we expect to dose ATTR patients with the first-ever systemically delivered CRISPR/Cas9-based therapy this year, and we are beginning IND-enabling activities for our newly announced development candidate, NTLA-5001, a WT1-TCR-directed engineered cell therapy, for treatment of AML, said Intellia President and Chief Executive Officer, John Leonard, M.D. We are focused on developing a robust platform with modular genome editing capabilities that enable a fast and reproducible path to development. Todays update reflects this strategy, and it also features the announcement of our third development program, an in vivo knockout approach for HAE. Importantly, this program leverages the infrastructure and insights from NTLA-2001 and underscores our ability to produce a rapid succession of new clinical candidates. We are excited by the strong momentum across our diverse pipeline and look forward to providing updates on our development programs in the upcoming year.

Program Updates and Anticipated 2020 Milestones:

Cash Position and Financial Guidance:

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020; its plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; its plans to nominate a development candidate for its hereditary angioedema (HAE) program in the first half of 2020; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; its presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML and HAE programs, in any future studies, including human clinical trials; its ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; its business plans and objectives for its preclinical studies and clinical trials, including the therapeutic potential and clinical benefits thereof, as well as the potential patient populations that may be addressed by its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; the impact of its collaborations on its development programs, including but not limited to its collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron) and Regenerons ability to enter into a Co/Co agreement for the HAE program; statements regarding the timing of regulatory filings for its development programs; its use of capital, including expenses, future accumulated deficit and other financial results during 2019 or in the future; and the ability to fund operations through the end of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Excerpt from:
Intellia Therapeutics Highlights Recent Progress and Anticipated 2020 Milestones - GlobeNewswire

genOway and Merck Strengthen CRISPR/Cas9 Strategic Alliance by Extending Their Partnership to All Animal Cell Models – PRNewswire

LYON, France, Jan. 7, 2020 /PRNewswire/ --genOway, a public company and leader in developing genetically modified research models, today announced an important milestone in extending its strategic alliance with Merck, a leading science and technology company and leader in genome editing.

In December 2018, genOway acquired from Merck exclusive worldwide rights on its foundational CRISPR/Cas9 portfolio in the rodent field (all applications involving rodent cells or animals). Today, the two companies have further strengthened their collaboration by entering into an additional license providing genOway with non-exclusive rights to commercialize the development and use of all other animal cell models for its customers' internal research uses as well as commercial exploitation.

"We are delighted to extend our relationship with Merck. The Merck IP is growing and broadening. Merck is now recognized as a leading provider of foundational CRISPR IP. This additional license will enable genOway to serve our customers better, by offering them broad and versatile solutions and the necessary intellectual property rights to help accelerate their research," says Alexandre Fraichard, founder and Chief Operating Officer of genOway.

Both Merck and genOwayhave identified research fields where they can combine their respective technologies and expertise to develop and validate new CRISPR/Cas9-related products and solutions. Merck's patented CRISPR integration technology isa strong entry point through which innovation can be developed and launched.

About genOway

genOway (Euronext Growth: ALGEN; ISIN: FR0004053510) is a biotechnology society that operates in 28 countries in Europe, Asia and North America, and more than 260 research institutes and 80 biopharmaceutical companies. genOway's development is based on a broad and exclusive technology platform, as well as on strong intellectual property rights, combining patents and licensing agreements. The company has signed many commercial contracts with the leaders of the pharmaceutical industry (BMS, Janssen, Novartis, Pfizer, etc.), and with the most prestigious academic research centers, including the King's College and the University of Manchester in England; Harvard, Caltech and the National Institutes of Health in the United States; the Pasteur Institute in France; the German National Genome Research Network and the Max Planck Institute in Germany.

http://www.genOway.com

Logo - https://mma.prnewswire.com/media/1057708/genOway_Logo.jpg

Contact: Sandrine Carteaulicensing@genoway.com+33-43-76-54-100

SOURCE genOway

https://www.genoway.com

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genOway and Merck Strengthen CRISPR/Cas9 Strategic Alliance by Extending Their Partnership to All Animal Cell Models - PRNewswire

Viewpoint: In 100 years, we’ll be honoring controversial CRISPR scientist He Jiankui – Genetic Literacy Project

When I saw the news that He Jiankui and colleagues had beensentenced to three years in prisonfor the first human embryo gene editing and implantation experiments, all I could think was, How will we look back at what they had done in 100 years?

I imagine that the scientists, medical doctors, and biotechnologists reading this essay will almost unanimously proclaim that He Jiankui will never be viewed in a positive way. What they fail to see is that societal ethics change, especially over long time frames.

In the next 100 years, thousands of edited embryos will be implanted and become children. I believe that embryo editing and implantation will someday be viewed much as how IVF is viewed today. When a human embryo being edited and implanted is no longer interesting enough for a news story, will we still view He Jiankui as a villain?

I dont think we will. But even if we do, He Jiankui will be remembered and talked about more than any scientist of our day. Although that may seriously aggravate many scientists and bioethicists, I think he deserves that honor.

Read full, original post: CRISPR babies scientist He Jiankui should not be villainized or headed to prison

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Viewpoint: In 100 years, we'll be honoring controversial CRISPR scientist He Jiankui - Genetic Literacy Project

CRISPR Therapeutics to Present at the Goldman Sachs 12th Annual Healthcare CEOs Unscripted: A View from the Top Conference – Yahoo Finance

ZUG, Switzerland and CAMBRIDGE, Mass., Jan. 06, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, is scheduled to present at the Goldman Sachs 12th Annual Healthcare CEOs Unscripted: A View from the Top conference on Thursday, January 9, 2020, at 1:10 p.m. ET.

A live webcast of the fireside chat will be available on the "Events & Presentations" page in the Investors section of the Company's website at https://crisprtx.com/events. A replay of the webcast will be archived on the Company's website for 14 days following the presentation.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

Investor Contact:Susan Kimsusan.kim@crisprtx.com

Media Contact:Jennifer PaganelliWCG on behalf of CRISPR347-658-8290jpaganelli@wcgworld.com

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CRISPR Therapeutics to Present at the Goldman Sachs 12th Annual Healthcare CEOs Unscripted: A View from the Top Conference - Yahoo Finance

CRISPR Technology Market Emerging Trends to Achieve Significant Growth in the Coming Years – Pro News Time

The Insight Partners dedicated research and analysis team consist of experienced professionals with advanced statistical expertise and offer various customization options in the existing study.

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology is a simple but powerful tool for genome editing. This tool enables life science researchers to easily edit DNA sequences and modify gene function. It has many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. By delivering the CRISPR enzyme Cas9 nuclease coupled with synthetic guide RNA (gRNA) into a cell, the cells genome can be cut at a desired location, that allows existing genes to be removed or add new ones.

Increasing usage of CRISPR systems in microbiology, growing government and private investments on research and development of genome editing, rising prevalence of genetic disorders, and increases application of CRISPR/Cas9 technology to improve crop production drives the global CRISPR technology market. However, ethical issues associated with CRISPR and lack of skilled personnel restrain the global CRISPR technology market over the forecast period.

Download a sample report Explore further @ https://www.theinsightpartners.com/sample/TIPRE00002853

Top Dominating Key Players:

The global CRISPR technology market is segmented on the basis of product and services, application, end user. Based product and services, the market is segmented as, enzymes, kits, services and others. The CRISPR technology market is categorized based on application into, genetic engineering, cell line engineering and others. Based on end user, the CRISPR Technology market is classified into biotechnology & pharmaceutical companies, contract research organizations (CROS), and academic & government research institutes.

The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global CRISPR Technology market based product and services, application, end user. It also provides market size and forecast till 2027 for overall market with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The CRISPR Technology Market by each region is later sub-segmented by respective countries and segments. The report covers analysis and forecast of 13 countries globally along with current trend and opportunities prevailing in the region.

North America held over major share in the CRISPR Technology market in 2017 owing to significant research carried out in order to develop novel therapeutics for disease targeting and high adoption of genome editing technique for germline modifications. North America is expected to collectively contribute towards the growth of CRISPR Technology market owing to the presence of major market players and also the development of technologically advanced products of CRISPR technology is expected to influence the CRISPR technology market growth.

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The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are a specialist in Technology, Healthcare, Manufacturing, Automotive and Defense.

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CRISPR Technology Market Emerging Trends to Achieve Significant Growth in the Coming Years - Pro News Time

UC’s 20th US patent on CRISPR-Cas9 gene-editing technologies – Health Gazette

A New Years Eve gift has been granted by the federal government to the University of California its 20th U.S. patent on CRISPR-Cas9 gene-editing technologies. The addition extends a large patent portfolio that is already being used to develop crop and seed breeding and human and animal health.

The new patent is the 18th concerning CRISPR-Cas9 technology granted this year by the University of Vienna and Emmanuelle Charpentier, who co-invented the technology with Jennifer Doudna of UC Berkeley, professor of molecular and cell biology and chemistry. Charpentier is currently the head of the Berlin-based Max Planck Institute for Infection Biology.

2019 has been an incredibly important and successful year in our ongoing efforts to maintain UC as the pioneer of CRISPR-Cas9 Intellectual Property in the United States, said Eldora L. Ellison, CRISPR-Cas9 University Patent Strategist and President of Sterne, Kessler, Goldstein & Fox, an intellectual property law firm. We are inspired by this years USPTO (U.S. Patent and Trademark Office) recognition of the leadership of the Doudna-Charpentier team on CRISPR-Cas9 and look forward to working to grow our portfolio by 2020.

In accordance with the UCs long-standing dedication to developing and applying its proprietary inventions to human enhancement, the university allows non-profit institutions, including academic institutions, to use the groundbreaking CRISPR-Cas9 technology for non-commercial research and educational purposes.

The UC also encouraged the widespread marketing of CRISPR-Cas9 technology through an exclusive license with Berkeley, Californias Caribou Biosciences Inc., which has sub-licensed the patent family to numerous companies around the world. The technology is currently being used to modify cattle, sheep and pig genomes to help fend off disease, create screens for human disease medications, generate updated human and mouse cell lines to help researchers understand and manage these disorders in humans, and manufacture research reagents.

In addition, Caribou licenses the technology for human medical uses to Intellia Therapeutics Inc., specifically cancer treatments, genetic disorders, viral infections and inflammatory diseases. The new patent (U.S. Patent 10,519,467), which proposes a method of generating a genetically engineered cell using CRISPR-Cas9 gene editing, is part of a collection of foreign and domestic patents that includes multiple CRISPR-Cas9 formulations and methods, such as controlling and editing genes and modulating transcription in any environment, even within plant, animal and human cells. The UCs 20 patents are the largest portfolio of CRISPR-Cas9 patents in the U.S. The UC has received notices of allowance for the issuance of five additional patents in early 2020.

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UC's 20th US patent on CRISPR-Cas9 gene-editing technologies - Health Gazette

‘Strange’ decade gave us CRISPR, gene therapy advances and a Neanderthal genome – Genetic Literacy Project

[H]ere, we present some of the innovations, both conceptual and technological, that stood out throughout the past decade.

In 2010, an international group of scientists published thefirst draft of the Neanderthal genome. And three years later, another large group of coauthors published ahigh-coverage, complete Neanderthal genome.

Beyond the remarkable advances concerning the sequencing of DNA, the 2010s saw the rapid establishment and development of a revolutionary genome editing strategy: CRISPR.

The 2010s were also momentous for personalized medicine and gene therapy. In 2017, the FDA approved the gene therapy Luxturna, which treats a single-gene disease that causes childhood blindness, making it the first such therapeutic to receive FDA approval.

I cant help feeling that the 2020s have big things in store for several areas of life science. The excitement that has built around personalized medicine, CRISPR as a therapeutic tool, and AI presage wide applications for these still-young technologies. As they have been throughout the decade, bioethicists in the 2020s will need to remain ever vigilant, considering continuing developments such as the creation of human/animal chimeras, the genomic modification of human embryos, and the potential of more-accessible genome sequencing.

Read full, original post: What A Long, Strange Decade Its Been

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'Strange' decade gave us CRISPR, gene therapy advances and a Neanderthal genome - Genetic Literacy Project

Promega to work with MilliporeSigma on advancing drug development using gene-editing tools – EPM Magazine

Life sciences group Promega has signed a license agreement with MilliporeSigma to advance drug development through new research products created using gene-editing technology.

The agreement will give Promega access to MilliporeSigmas foundational CRISPR genome-editing technology. Promega will aim to create new research products for investigating endogenous biology, including those for drug development. This will give scientists the tools needed to better read the physiological or natural levels of protein expression, providing a more accurate understanding of protein behavior.

This license further expands the potential of CRISPR, and, more importantly, gives scientists a new view into natural cell activity, said Bill Linton, president and CEO, Promega Corporation. This is quite a meaningful contribution to many areas of applied research in such fields as cancer and neuroscience.

Research papers such as those in ACS Chemical Biology have detailed how the Promega HiBiT Protein Tagging System can be combined with CRISPR-Cas9-mediated gene editing to tag endogenous proteins and simplify their study under natural expression conditions.

Under this licensing agreement, Promega plans to use our intellectual property to develop CRISPR- edited cell lines, which can play a major role in determining drug efficacy, toxicity and overall development, said Udit Batra, CEO, MilliporeSigma.

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Promega to work with MilliporeSigma on advancing drug development using gene-editing tools - EPM Magazine

The Final Numbers Now Have The Rebound: CRISPR Therapeutics AG (CRSP) and Coupa Software Incorporated (COUP) – BOV News

GRAUBNDNER KANTONALBANK (PRIVAT bought a fresh place in CRISPR Therapeutics AG (NASDAQ:CRSP). The institutional investor bought 32.00 shares of the stock in a transaction took place on 11/29/2019. In another most recent transaction, which held on 11/30/2019, NIKKO ASSET MANAGEMENT AUSTRALIA sold approximately 983.00 shares of CRISPR Therapeutics AG. In a separate transaction which took place on 11/29/2019, the institutional investor, MEDICAL STRATEGY GMBH sold 1.9 thousand shares of the companys stock. The total Institutional investors and hedge funds own 46.90% of the companys stock.

In the most recent purchasing and selling session, CRISPR Therapeutics AG (CRSP)s share price increased by 0.20 percent to ratify at $59.44. A sum of 1391345 shares traded at recent session and its average exchanging volume remained at 1.08M shares. The 52-week price high and low points are important variables to concentrate on when assessing the current and prospective worth of a stock. CRISPR Therapeutics AG (CRSP) shares are taking a pay cut of -19.68% from the high point of 52 weeks and flying high of 116.15% from the low figure of 52 weeks.

CRISPR Therapeutics AG (CRSP) shares reached a high of $60.28 and dropped to a low of $57.36 until finishing in the latest session at $58.01. Traders and investors may also choose to study the ATR or Average True Range when concentrating on technical inventory assessment. Currently at 3.24 is the 14-day ATR for CRISPR Therapeutics AG (CRSP). The highest level of 52-weeks price has $74.00 and $27.50 for 52 weeks lowest level. After the recent changes in the price, the firm captured the enterprise value of $2.69B. The liquidity ratios which the firm has won as a quick ratio of 8.30, a current ratio of 8.30 and a debt-to-equity ratio of 0.00.

Having a look at past record, were going to look at various forwards or backwards shifting developments regarding CRSP. The firms shares fell -10.95 percent in the past five business days and shrunk -18.72 percent in the past thirty business days. In the previous quarter, the stock rose 53.24 percent at some point. The output of the stock increased 24.90 percent within the six-month closing period, while general annual output gained 112.13 percent. The companys performance is now negative at -2.41% from the beginning of the calendar year.

According to WSJ, CRISPR Therapeutics AG (CRSP) obtained an estimated Overweight proposal from the 16 brokerage firms currently keeping a deep eye on the stock performance as compares to its rivals. 2 equity research analysts rated the shares with a selling strategy, 2 gave a hold approach, 12 gave a purchase tip, 0 gave the firm a overweight advice and 0 put the stock under the underweight category. The average price goal of one year between several banks and credit unions that last year discussed the stock is $77.50.

Coupa Software Incorporated (COUP) shares on Fridays trading session, jumped 3.91 percent to see the stock exchange hands at $159.58 per unit. Lets a quick look at companys past reported and future predictions of growth using the EPS Growth. EPS growth is a percentage change in standardized earnings per share over the trailing-twelve-month period to the current year-end. The company posted a value of -$1.35 as earning-per-share over the last full year, while a chance, will post $0.47 for the coming year. The current EPS Growth rate for the company during the year is -16.40% and predicted to reach at 31.67% for the coming year. In-depth, if we analyze for the long-term EPS Growth, the scenario is totally different as the current prediction is 58.37% for the next five year.

The last trading period has seen Coupa Software Incorporated (COUP) move -0.24% and 173.82% from the stocks 52-week high and 52-week low prices respectively. The daily trading volume for Coupa Software Incorporated (NASDAQ:COUP) over the last session is 2.92 million shares. COUP has attracted considerable attention from traders and investors, a scenario that has seen its volume jump 64.87% compared to the previous one.

Investors focus on the profitability proportions of the company that how the company performs at profitability side. Return on equity ratio or ROE is a significant indicator for prospective investors as they would like to see just how effectively a business is using their cash to produce net earnings. As a return on equity, Coupa Software Incorporated (NASDAQ:COUP) produces -21.60%. Because it would be easy and highly flexible, ROI measurement is among the most popular investment ratios. Executives could use it to evaluate the levels of performance on acquisitions of capital equipment whereas investors can determine that how the stock investment is better. The ROI entry for COUPs scenario is at -9.60%. Another main metric of a profitability ratio is the return on assets ratio or ROA that analyses how effectively a business can handle its assets to generate earnings over a duration of time. Coupa Software Incorporated (COUP) generated -7.40% ROA for the trading twelve-month.

Volatility is just a proportion of the anticipated day by day value extendthe range where an informal investor works. Greater instability implies more noteworthy benefit or misfortune. After an ongoing check, Coupa Software Incorporated (COUP) stock is found to be 4.65% volatile for the week, while 3.70% volatility is recorded for the month. The outstanding shares have been calculated 61.40M. Based on a recent bid, its distance from 20 days simple moving average is 8.43%, and its distance from 50 days simple moving average is 11.88% while it has a distance of 24.45% from the 200 days simple moving average.

The Williams Percent Range or Williams %R is a well-known specialized pointer made by Larry Williams to help recognize overbought and oversold circumstances. Coupa Software Incorporated (NASDAQ:COUP)s Williams Percent Range or Williams %R at the time of writing to be seated at 18.12% for 9-Day. It is also calculated for different time spans. Currently for this organization, Williams %R is stood at 13.07% for 14-Day, 13.07% for 20-Day, 8.24% for 50-Day and to be seated 8.24% for 100-Day. Relative Strength Index, or RSI(14), which is a technical analysis gauge, also used to measure momentum on a scale of zero to 100 for overbought and oversold. In the case of Coupa Software Incorporated, the RSI reading has hit 66.33 for 14-Day.

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The Final Numbers Now Have The Rebound: CRISPR Therapeutics AG (CRSP) and Coupa Software Incorporated (COUP) - BOV News

Better Buy: CRISPR Therapeutics vs. Sangamo Therapeutics – The Motley Fool

If you're considering investing in the gene editing sector, it's worth taking some time to look through all the main players in this small but promising biotech market. At the moment, there are just a few noteworthy companies in this space, all of them still at early clinical stages despite commanding market valuations well into the billions of dollars.

CRISPR Therapeutics (NASDAQ:CRSP) is likely the first gene editing stock to come to mind, and it's considered by many to be the leading company in the market, if only by market cap. However, smaller companies, like Sangamo Therapeutics (NASDAQ:SGMO), also have plenty of promise.

If you're wondering which of these two stocks is the better buy, then read below to find out all the details.

Image source: Getty Images.

While different gene editing companies target their own specific conditions, investors will notice that many tend to coalesce around the area of blood disorders. Both CRISPR and Sangamo are working on drug candidates that target sickle cell disease and transfusion-dependent beta thalassemia (TDT), which are disorders that hinder the ability of hemoglobin to carry oxygen around the body.

CRISPR is working on CTX001, which has been used to treat two different patients, one with sickle cell disease and the other with beta thalassemia. Both patients have shown a complete reversal of all key symptoms, with more patients now undergoing CTX001 treatment.

Unlike CTX001, Sangamo has two separate drug candidates, each targeting only one of the blood disorders mentioned above. ST-400 is Sangamo's beta thalassemia drug, while BIVV003 is its sickle cell candidate. Both are being developed alongside Sanofi, which has partnered with Sangamo to develop these drugs.

While BIVV003 is still undergoing early clinical testing, with investors still waiting to see the preliminary results, ST-400 has proven to be an early success so far. Sangamo released data in early December regarding the first three patients treated with ST-400 for TDT, with all of them showing encouraging results with few side effects. Further results are expected to come out in 2020.

Sangamo has a pretty diverse portfolio of drug candidates that are either in preclinical or clinical stages of development, totaling 15 separate projects in comparison to CRISPR's nine. Five of those are in early phase 1/2 trials. Besides Sangamo's sickle cell and beta thalassemia treatments, Sangamo is working on treatments for Fabry disease, Hemophilia A, and Hunter syndrome (also known as mucopolysaccharidosis type 2 or MPS II).

The Hemophilia A treatment, SB-525, showed strong results in its phase 1/2 study earlier this year. Patients with this blood disorder, who experience a lack of a key blood-clotting factor, showed significant improvements in levels of this clotting factor after taking SB-525.

Even patients with severe cases of hemophilia A, which is extremely hard to treat, showed impressive improvements in the levels of this clotting factor. Pfizer, which is partnered with Sangamo to develop SB-525, is now moving toward a new phase 3 trial, which is expected to begin sometime in 2020.

Sangamo's Fabry treatment, ST-920, is still undergoing its own early-stage clinical trials, with little information available at present. The only setback for Sangamo has been in its MPS II drug, SB-913, which ended up failing to significantly help patients with the rare genetic disorder. While the company hasn't given up on SB-913 yet, it's definitely the weak link in an otherwise strong drug portfolio.

CRISPR's drug portfolio is a bit narrower, with only two drugs in clinical testing in comparison to Sangamo's five. Besides the previously mentioned CTX001, CRISPR has a fairly strong cancer immunology lineup. CTX110, CTX120, and CTX130 are its selection of immunology candidates, although CTX110 is the only one in clinical testing at the moment.

Cancer immunology is a massive market that's estimated to reach $127 billion by 2026, and a home run in this area would be a major win for CRISPR. CTX110 is a CAR-T (chimeric antigen receptor T-cell) therapy, a type of treatment in which immune cells are extracted from a patient, retrained outside the body, and later reintroduced into the patient's system in hopes they will perform better. While it's not the only CAR-T therapy being developed, CRISPR's treatment could prove to be much cheaper than current treatments, which cost hundreds of thousands of dollars for a single patient.

CRISPR has had a strong fiscal third quarter, reporting $138.4 million in net income on revenues of $211.9 million. But in 2019, CRISPR has so far only reported $36.3 million in net income, as the earlier quarters reported losses. While it's nice that CRISPR is reporting a profit, something very few early-stage biotech companies can boast, it's still a very small figure considering CRISPR's $3.9 billion market cap.

Sangamo's financials look a lot different. Besides being a fraction of CRISPR's size with a market cap of $970 million, Sangamo's Q3 2019 revenues came in at $21.9 million, while reporting a net loss of $27.4 million for the quarter. However, the company has an impressive $408.3 million in cash and equivalents, enough to last for around four years at the current rate of expenses.

In terms of traditional valuation metrics, it's hard to evaluate clinical-stage biotech stocks by looking at ratios, as their financial figures can change dramatically if a drug candidate receives approval. Currently, CRISPR trades at 16.7 its price to sales (P/S) ratio, but in July, the company was trading at an astronomical 1,800 P/S ratio, meaning that investors were willing to pay extraordinarily high amounts for what little revenue it was making that quarter. In comparison, Sangamo is more moderately priced, with a 12.2 P/S ratio.

Both companies are compelling investments if you're looking for exposure to the gene editing sector. While Sangamo has a broader pipeline of projects, I still think CRISPR is the better choice if you had to pick just one of these companies. CRISPR has not only shown positive clinical results for CTX001 and CTX110, but is also reporting a profit for this most recent quarter, which is pretty rare for early-stage biotech stocks. Meanwhile, Sangamo isn't expected to turn a profit anytime soon.

However, gene-editing drugs are still at an early stage of clinical development, and plenty of things can change over the coming years. Both CRISPR and Sangamo are promising investments for someone who's comfortable buying into early-stage biotech stocks.

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Better Buy: CRISPR Therapeutics vs. Sangamo Therapeutics - The Motley Fool

Duke Researchers Garner Over $6 Million in NIH Funding to Fight Genetic Diseases – Duke Today

Hemophilia. Cystic fibrosis. Duchenne muscular dystrophy. Huntingtons disease. These are just a few of the thousands of disorders caused by mutations in the bodys DNA. Treating the root causes of these debilitating diseases has become possible only recently, thanks to the development of genome editing tools such as CRISPR, which can change DNA sequences in cells and tissues to correct fundamental errors at the sourcebut significant hurdles must be overcome before genome-editing treatments are ready for use in humans.

Enter the National Institutes of Health Common Funds Somatic Cell Genome Editing (SCGE) program, established in 2018 to help researchers develop and assess accurate, safe and effective genome editing therapies for use in the cells and tissues of the body (aka somatic cells) that are affected by each of these diseases.

Todaywith three ongoing grants totaling more than $6 million in research fundingDuke University is tied with Yale University, UC Berkeley and UC Davis for the most projects supported by the NIH SCGE Program.

In the 2019 SCGE awards cycle, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering, and collaborators across Duke and North Carolina State University received two grants: the first will allow them to study how CRISPR genome editing affects engineered human muscle tissues, while the second project will develop new CRISPR tools to turn genes on and off rather than permanently alter the targeted DNA sequence. This work builds on a 2018 SCGE grant, led by Aravind Asokan, professor and director of gene therapy in the Department of Surgery, which focuses on using adeno-associated viruses to deliver gene editing tools to neuromuscular tissue.

There is an amazing team of engineers, scientists and clinicians at Duke and the broader Research Triangle coalescing around the challenges of studying and manipulating the human genome to treat diseasefrom delivery to modeling to building new tools, said Gersbach, who with his colleagues recently launched the Duke Center for Advanced Genomic Technologies (CAGT), a collaboration of the Pratt School of Engineering, Trinity College of Arts and Sciences, and School of Medicine. Were very excited to be at the center of those efforts and greatly appreciate the support of the NIH SCGE Program to realize this vision.

For their first grant, Gersbach will collaborate with fellow Duke biomedical engineering faculty Nenad Bursac and George Truskey to monitor how genome editing affects engineered human muscle tissue. Through their new project, the team will use human pluripotent stem cells to make human muscle tissues in the lab, specifically skeletal and cardiac muscle, which are often affected by genetic diseases. These systems will then serve as a more accurate model for monitoring the health of human tissues, on-target and off-target genome modifications, tissue regeneration, and possible immune responses during CRISPR-mediated genome editing.

Currently, most genetic testing occurs using animal models, but those dont always accurately replicate the human response to therapy, says Truskey, the Goodson Professor of Biomedical Engineering.

Bursac adds, We have a long history of engineering human cardiac and skeletal muscle tissues with the right cell types and physiology to model the response to gene editing systems like CRISPR. With these platforms, we hope to help predict how muscle will respond in a human trial.

Gersbach will work with Tim Reddy, a Duke associate professor of biostatistics and bioinformatics, and Rodolphe Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University, on the second grant. According to Gersbach, this has the potential to extend the impact of genome editing technologies to a greater diversity of diseases, as many common diseases, such as neurodegenerative and autoimmune conditions, result from too much or too little of certain genes rather than a single genetic mutation. This work builds on previous collaborations between Gersbach, Barrangou and Reddy developing both new CRISPR systems for gene regulation and to regulate the epigenome rather than permanently delete DNA sequences.

Aravind Asokan leads Dukes initial SCGE grant, which explores the the evolution of next generation of adeno-associated viruses (AAVs), which have emerged as a safe and effective system to deliver gene therapies to targeted cells, especially those involved in neuromuscular diseases like spinal muscular atrophy, Duchenne muscular dystrophy and other myopathies. However, delivery of genome editing tools to the stem cells of neuromuscular tissue is particularly challenging. This collaboration between Asokan and Gersbach builds on their previous work in using AAV and CRISPR to treat animal models of DMD.

We aim to correct mutations not just in the mature muscle cells, but also in the muscle stem cells that regenerate skeletal muscle tissue, explainsAsokan. This approach is critical to ensuring long-term stability of genome editing in muscle and ultimately we hope to establish a paradigm where our cross-cutting viral evolution approach can enable efficient editing in multiple organ systems.

Click through to learn more about the Duke Center for Advanced Genomic Technologies.

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Duke Researchers Garner Over $6 Million in NIH Funding to Fight Genetic Diseases - Duke Today

The Advances that Will Shape Life Sciences in the 2020s – The Scientist

The 2010s brought major advancements in every aspect of the life sciences and ushered in an era of collaboration and multidisciplinary approaches. The Scientist spoke with Steven Wiley, a systems biologist at Pacific Northwest National Laboratory and member of TSs editorial board, about what he thinks the recent past indicates about the upcoming decade of research.

Wiley: The next year will be a continuation of the scientific breakthroughs that were present the last couple of years, and whats happened the last couple of years will fundamentally transform the next decade. There are two areas that I think are really posed for an explosive growth, and one is single-cell biology. . . . The second one people know is transformative . . . is CRISPR technologies.

Wiley: Single-cell sequencing, single-cell proteomics, single-cell imagingthese are all part of this new area of single-cell biology which is really going to impact a whole slew of different fields. We'll see a lot of breakthroughs driven by that in the next year, but well see the full impact of this playing out over the next decade.

[Single-cell biology] started [out] driven by single-cell sequencing, and very near on the horizon is going to single-cell proteomics. And then, of course, that complements a lot of the imaging work thats been done, developing a new generation of probes to be able to query whats happening at the single-cell level.

This really brings to the fore the idea that cells in a population are very heterogeneous, and what we see at the population level is a reflection of what the individual cells are doing. And until we understand what the individual cells in a population are doing, we cant deal with issues of, for example, mathematically modeling whats going on in cells.

[Researchers developing] both sequencing technologies and proteomics technology in the last decade have been working on increased sensitivity and speed and precision. This increase in speed and precision and increasingly small sample size has gotten down to a point where now we can look at things like cancer heterogeneity. That is . . . when you treat a cancer you can kill 95 percent of the tumor but theres 5 percent left and that is whats going to come back, and you have a recurrence of the cancer or metastasis. So its the small parts that really cause the problem, and until you can actually understand why those resistant cells are different, youre never going to do things like develop a completely effective cancer treatment.

Now the technology is thereboth sequencing technology and mass spectrometry technologies. It opens up new worlds of what we can look at, and I think thats why this is really being very transformative. Were now at the level where we can look at individual cells. Thats amazing.

Wiley: CRISPR technologieseveryone touts them as a way of editing the genome, which is true. But the true power of that, I believe . . . is the fact that it provides a way of tagging endogenous genes. So for example, you see a number of different papers come out in which people have used CRISPR technologies to insert fluorescent markers into genes. You can look at the dynamics and localization and expression of individual genes and individual cells.

The second thing that CRISPR is really good at is perturbations, being able to turn up genes and down genes, altering the expression of individual genes up and down in a cell with incredible specificity. For example, [with] a genetic disease or in cancer, most of the really significant impactful genetic changes are at the level of increased expression or decreased expression. So the way we think about changing gene expression is: [in] one cell type, the gene is off, [and in] another cell type, the gene is on. But thats not actually true. There are subtle changes in abundance and localization and disposition of individual genes that have enormous regulatory impact on the cells. But weve lacked good tools to [investigate] that.

The ability to manipulate the expression level of genes, to tag them, to make modifications in the individual genes and cells opens up a toolbox of experimental technologies that are just revolutionary.

Editors note: Answers have been edited for length and clarity.

Emma Yasinski is a Florida-based freelance reporter. Follow her on Twitter@EmmaYas24.

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The Advances that Will Shape Life Sciences in the 2020s - The Scientist

CRISPR Therapeutics AG [CRSP] A Healthcare stock that is highly – The Dwinnex

CRISPR Therapeutics AG [NASDAQ: CRSP] gained by 1.21% on the last trading session, reaching $60.04 price per share at the time. CRISPR Therapeutics AG represents 57.50M in outstanding shares, while the company has a total market value of $3.41B with the latest information.

The CRISPR Therapeutics AG traded at the price of $60.04 with 803450 shares were bought and sold during the latest trading session. Over the period of the last 3 months, the average trading volume of CRSP shares recorded 1.08M.

Its stock price has been found in the range of 27.50 to 74.00. This is compared to its latest closing price of $59.32.

Pay attention to the next-scheduled financial results for this company to be released, which is slated for Mon 24 Feb (In 52 Days).

Now lets turn to look at profitability: with a current Operating Margin for CRISPR Therapeutics AG [CRSP] sitting at -5087.80, this companys Net Margin is now -5.30%. These metrics indicate that this company is not generating as much profit, after accounting for expenses, compared to its market peers.

This companys Return on Total Capital is -54.81, and its Return on Invested Capital has reached -40.70%. Its Return on Equity is -56.89, and its Return on Assets is -43.40. These metrics suggest that this CRISPR Therapeutics AG does a poor job of managing its assets, and likely wont be able to provide successful business outcomes for its investors in the near term.

What about valuation? This companys Enterprise Value to EBITDA is -22.31. The Enterprise Value to Sales for this firm is now 16.31. CRISPR Therapeutics AG [CRSP] has a Price to Book Ratio of 3.78.

Shifting the focus to workforce efficiency, CRISPR Therapeutics AG [CRSP] earns $16,617 for each employee under its payroll. Similarly, this companys Receivables Turnover is 2.30 and its Total Asset Turnover is 0.01. This publicly-traded organizations liquidity data is also interesting: its Quick Ratio is 16.81 and its Current Ratio is 16.81. This company, considering these metrics, has a healthy ratio between its short-term liquid assets and its short-term liabilities, making it a less risky investment.

CRISPR Therapeutics AG [CRSP] has 57.50M shares outstanding, amounting to a total market cap of $3.41B. Its stock price has been found in the range of 27.50 to 74.00. At its current price, it has moved down by -18.86% from its 52-week high, and it has moved up 118.33% from its 52-week low.

This stocks Relative Strength Index (RSI) is at 42.19. This RSI score is good, suggesting this stock is neither overbought or oversold.

Shares of CRISPR Therapeutics AG [CRSP], on the whole, present investors with both positive and negative signals. Wall Street analysts have mixed reviews when it comes to the 12-month price outlook, and this companys financials show a combination of strengths and weaknesses. Based on the price performance, this investment is somewhat risky while presenting reasonable potential for ROI.

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CRISPR Therapeutics AG [CRSP] A Healthcare stock that is highly - The Dwinnex

Year in Review: The Top Science Stories of 2019 – WTTW News

2019 has seen some astonishing scientific breakthroughs, from the creation of the first ever image of a black hole a feat once thought impossible to the use of DNA splicing technology to treat sickle cell disease.

As the year comes to an end, three of our regular science contributors Daniel Holz of the University of Chicago, Rabiah Mayas of the Museum of Science and Industry and Mark Hammergren of the Adler Planetarium share what they regard as the most significant science stories of the year.

Here are the stories they selected.

Daniel Hotz: First ever image of a black hole

Using the Event Horizon Telescope, scientists obtained an image of the black hole at the center of galaxy M87, outlined by emission from hot gas swirling around it under the influence of strong gravity near its event horizon. (Credits: Event Horizon Telescope collaboration et al.)

In April, an international astronomical team called the Event Horizon Telescope Collaboration produced an image of a black hole for the first time.

Because not even light can escape from the immense gravity well that is a black hole, the idea of imaging a one was once thought impossible. Holz explained that the image the Event Horizon Telescope team produced is actually of super-heated matter about to fall into the black hole.

We are not looking at the black hole itself, what we are doing is seeing stuff fall into the black hole and that stuff gets very, very hot superheated because of the strong gravity and then it glows, said Holz. And what we are seeing is that glow but in the center nothing is glowing and thats because theres a black hole there.

Mayas noted the years of hard work and international collaboration that created what has already become an iconic image. The team used telescopes around the world to, in effect, create one huge telescope the size of the Earth to create the image.

There were scientists and engineers and astronomers from across the globe that came together to generate this image, said Mayas. The shear technology and the instrumentation and the collaboration that led to that is another example of what a career in STEM can look like for young people.

Rabiah Mayas: Gene-edited cells used to treat sickle cell disease

Researchers at the Sarah Cannon Research Institute in Nashville, Tennessee, announced in November that they had used genetically edited cells to treat sickle cell disease a painful and until now incurable condition that impacts millions of people in the United States and around the world.

Doctors used cells from a patients bone marrow that had been modified using CRISPR cas9 gene-splicing technology and reintroduced the cells back into the patients body.

CRISPR cas9 is something that was identified in bacteria as part of a bacterias natural immunity, said Mayas. The way that CRISPR works is that it looks for specific regions of DNA so the genetic information in the cells of many organisms and recognizes the particular sequence. And cas9 is an enzyme that can cut it. So it cuts the DNA, makes a break, and then your cell can put those ends back together.

CRISPR in this case was used to genetically modify the version of hemoglobin which is the protein that is malformed in Sickle Cell and turn it into a different form that is functional, said Mayas.

Within a month, those cells were producing healthy hemoglobin.

Mark Hammergren: Artemis moon mission

NASA is returning to the moon much sooner than it originally planned.

At the direction of President Donald Trump, NASA has been asked to accelerate its Artemis mission and return humans to the moons surface by 2024. The original Artemis schedule would have put humans back on the moon by 2028.

President Trump and his administration have proposed accelerating this return to the moon and came out and said we are going to land humans on the moon a man and a woman on the moon by 2024. And that is the directive given to NASA, said Hammergren. Regardless of what you think of these plans this is a directive to NASA that NASA has to follow.

Mayas noted that although the Artemis mission is to the moon, it is also regarded as a way to answer key questions and test and prove technology that could one day take humans to Mars.

What does it mean to spend time on another solar body? What does it mean to look for water and develop systems on a place that is not Earth in preparation for Mars?

All: Climate change

Activists on the evening of Monday, Oct. 7, 2019 closed down the streets in front of Chicago City Hall and the James R. Thompson Center as they called on Mayor Lori Lightfoot and Gov. J.B. Pritzker to declare a climate emergency. (WTTW News)

Climate change is the defining challenge of our time, according to United Nations Secretary-General Antonio Guterres.

Guterres noted in an introduction to the latest report of the UNs Science Advisory Group, released in September, that the climate is already changing and highlights the far-reaching and dangerous impacts that will unfold for generations to come.

All three of our scientific contributors believe that climate change is one of the top science stories of the year.

I think young people have been telling us for years that they have been concerned about climate change, said Mayas. Young people from indigenous cultures around the world, from black and other marginalized communities in this country and elsewhere have been screaming for a while about climate change in part because we know from data that certain communities suffer the consequences of climate change more than others.

Hammergren said that as a planetary scientist we have to consider the Earth as a system as a whole and that he had seen directly the increase in carbon dioxide in the atmosphere in his astronomical observations.

Holz noted the evidence for global warming was overwhelming at this point.

The last few years have shown just look at the news the wildfires, the storms, the rising sea levels its just this whole parade of disasters. And this is just the beginning, said Holz. I fear for the future.

But Holz also noted that its not yet too late to try and address some of the worst impacts of climate change, particularly as young people around the world have rallied around this issue.

Its not too late, we can all get involved theres lots of things to do and the fact that young people are rising up. It really impacts them the most and we should listen to them, said Holz. Its somewhat embarrassing that we have to have the young, the next generation, to hold us to task.

Related stories:

Field Museums New Meteorite Contains Stardust That Predates the Solar System

Astronomers Take First-Ever Picture of a Black Hole

Climate Simulations Are Mostly Accurate, Study Finds

Climate Activists: The Oceans Are Rising, And So Are We

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Year in Review: The Top Science Stories of 2019 - WTTW News

Will Prime Editors be the New CRISPR? – Discover Magazine

CRISPR may have generated a lot of buzz this year, but some researchers are already looking beyond it to the next new gene-editing technique. Say hello to prime editing.

If CRISPR is like scissors then you can think of prime editors like word processors, said chemist David Liu in an October press briefing. He spoke days ahead of the first-ever prime editing study, published in Nature and authored by Liu and his team at the Broad Institute of MIT and Harvard University. Liu explained that, while CRISPR cuts through DNAs double helix to snip out genes, prime editing searches for and replaces targeted genes without such slicing and dicing, reducing the risk of unintended changes to the genetic code.

The team was able to correct mutations associated with both sickle cell and Tay-Sachs diseases. Liu believes the technique ultimately might be able to correct almost 90 percent of such mutations, but stressed additional studies are needed to gauge prime editings full potential.

This is the beginning, rather than the end, said Liu.

[This story appeared in print as "It's Prime Time for a New Gene Editor."]

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Will Prime Editors be the New CRISPR? - Discover Magazine

These 6 Incredible Discoveries From The Past Decade Have Changed Science Forever – ScienceAlert

From finding the building blocks for life on Mars to breakthroughs in gene editing and the rise of artificial intelligence, here are six major scientific discoveries that shaped the 2010s - and what leading experts say could come next

We don't yet know whether there was ever life on Mars - but thanks to a small, six-wheeled robot, we do know the Red Planet was habitable.

Shortly after landing on 6 August 2012, NASA's Curiosity rover discovered rounded pebbles - new evidence that rivers flowed there billions of years ago.

The proof has since multiplied, showing there was in fact a lot of water on Mars - the surface was covered in hot springs, lakes, and maybe even oceans.

A crater on the Red Planet filled with water ice. (ESA/DLR/FU Berlin, CC BY-SA 3.0 IGO)

Curiosity also discovered what NASA calls the building blocks of life, complex organic molecules, in 2014.

And so the hunt continues for signs that Earth-based life is not (or wasn't always) alone.

Two new rovers will be launched next year - America's Mars 2020 and Europe's Rosalind Franklin rovers, looking for ancient microbes.

"Going into the coming decade, Mars research will shift from the question 'Was Mars habitable?' to 'Did (or does) Mars support life?'" said Emily Lakdawalla, a geologist at The Planetary Society.

We had long thought of the little corner of the Universe that we call home as unique, but observations made thanks to the Kepler space telescope blew apart those pretensions.

Launched in 2009, the Kepler mission helped identify more than 2,600 planets outside of our Solar System, also known as exoplanets - and astronomers believe each star has a planet, meaning there are billions out there.

Kepler's successor TESS was launched by NASA in 2018, as we scope out the potential for extraterrestrial life.

Expect more detailed analysis of the chemical composition of these planets' atmospheres in the 2020s, said Tim Swindle, an astrophysicist at the University of Arizona.

We also got our first glimpse of a black hole this year thanks to the groundbreaking work of the Event Horizon Telescope collaboration.

(Event Horizon Telescope Collaboration)

"What I predict is that by the end of the next decade, we will be making high quality real-time movies of black holes that reveal not just how they look, but how they act on the cosmic stage," Shep Doeleman, the project's director, told AFP.

But one event from the decade undoubtedly stood above the rest: the detection for the first time on September 14, 2015 of gravitational waves, ripples in the fabric of the universe.

The collision of two black holes 1.3 billion years earlier was so powerful it spread waves throughout the cosmos that bend space and travel at the speed of light. That morning, they finally reached Earth.

The phenomenon had been predicted by Albert Einstein in his theory of relativity, and here was proof he was right all along.

Three Americans won the Nobel prize in physics in 2017 for their work on the project, and there have been many more gravitational waves detected since.

Cosmologists meanwhile continue to debate the origin and composition of the universe. The invisible dark matter that makes up its vast majority remains one of the greatest puzzles to solve.

"We're dying to know what it might be," said cosmologist James Peebles, who won this year's Nobel prize in physics.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) - a family of DNA sequences - is a phrase that doesn't exactly roll off the tongue.

(Meletios Verras/iStock)

But the field of biomedicine can now be divided into two eras, one defined during the past decade: before and after CRISPR-Cas9 (or CRISPR for short), the basis for a gene editing technology.

"CRISPR-based gene editing stands above all the others," William Kaelin, a 2019 Nobel prize winner for medicine, told AFP.

In 2012, Emmanuelle Charpentier and Jennifer Doudna reported that they had developed the new tool that exploits the immune defense system of bacteria to edit the genes of other organisms.

It is much simpler than preceding technology, cheaper and easy to use in small labs.

Charpentier and Doudna were showered in awards. but the technique is also far from perfect and can create unintended mutations.

Experts believe this may have happened to Chinese twins born in 2018 as a result of edits performed by a researcher who was widely criticized for ignoring scientific and ethical norms.

Still, CRISPR remains one of the biggest science stories of recent years, with Kaelin predicting an "explosion" in its use to combat human disease.

For decades, doctors had three main weapons to fight cancer: surgery, chemotherapy drugs, and radiation.

The 2010s saw the rise of a fourth, one that was long doubted: immunotherapy, or leveraging the body's own immune system to target tumor cells.

(Design Cells/iStock)

One of the most advanced techniques is known as CAR T-cell therapy, in which a patient's T-cells - part of their immune system - are collected from their blood, modified and reinfused into the body.

A wave of drugs have hit the market since the mid-2010s for more and more types of cancer including melanomas, lymphomas, leukemias and lung cancers - heralding what some oncologists hope could be a golden era.

For William Cance, scientific director of the American Cancer Society, the next decade could bring new immunotherapies that are "better and cheaper" than what we have now.

The decade began with a major new addition to the human family tree: Denisovans, named after the Denisova Cave in the Altai Mountains of Siberia.

Scientists sequenced the DNA of a female juvenile's finger bone in 2010, finding it was distinct both from genetically modern humans and Neanderthals, our most famous ancient cousins who lived alongside us until around 40,000 years ago.

The mysterious hominin species is thought to have ranged from Siberia to Indonesia, but the only remains have been found in the Altai region and Tibet.

We also learned that, unlike previously assumed, Homo sapiens bred extensively with Neanderthals - and our relatives were not the brutish simpletons previously assumed but were responsible for artworks, such as the handprints in a Spanish cave they were credited for crafting in 2018.

They also wore jewelry, and buried their dead with flowers - just like we do.

Next came Homo naledi, remains of which were discovered in South Africa in 2015, while this year, paleontologists classified yet another species found in the Philippines: a small-sized hominin called Homo luzonensis.

Advances in DNA testing have led to a revolution in our ability to sequence genetic material tens of thousands of years old, helping unravel ancient migrations, like that of the Bronze Age herders who left the steppes 5,000 years ago, spreading Indo-European languages to Europe and Asia.

"This discovery has led to a revolution in our ability to study human evolution and how we came to be in a way never possible before," said Vagheesh Narasimhan, a geneticist at Harvard Medical School.

One exciting new avenue for the next decade is paleoproteomics, which allows scientists to analyze bones millions of years old.

"Using this technique, it will be possible to sort out many fossils whose evolutionary position is unclear," said Aida Gomez-Robles, an anthropologist at University College London.

"Neo" skull of Homo naledi from the Lesedi Chamber. (John Hawks/University of the Witwatersrand)

Machine learning - what we most commonly mean when talking about "artificial intelligence" - came into its own in the 2010s.

Using statistics to identify patterns in vast datasets, machine learning today powers everything from voice assistants to recommendations on Netflix and Facebook.

So-called "deep learning" takes this process even further and begins to mimic some of the complexity of a human brain.

It is the technology behind some of the most eye-catching breakthroughs of the decade: from Google's AlphaGo, which beat the world champion of the fiendishly difficult game Go in 2017, to the advent of real-time voice translations and advanced facial recognition on Facebook.

In 2016, for example, Google Translate - launched a decade earlier - transformed from a service that provided results that were stilted at best, nonsensical at worst, to one that offered translations that were far more natural and accurate.

At times, the results even seemed polished.

"Certainly the biggest breakthrough in the 2010s was deep learning - the discovery that artificial neural networks could be scaled up to many real-world tasks," said Henry Kautz, a computer science professor at the University of Rochester.

"In applied research, I think AI has the potential to power new methods for scientific discovery," from enhancing the strength of materials to discovering new drugs and even making breakthroughs in physics, Kautz said.

For Max Jaderberg, a research scientist at DeepMind, owned by Google's parent company Alphabet, the next big leap will come via "algorithms that can learn to discover information, and rapidly adapt and internalize and act on this new knowledge," as opposed to depending on humans to feed them the correct data.

That could eventually pave the way to "artificial general intelligence", or a machine capable of performing any tasks humans can, rather than excelling at a single function.

Agence France-Presse

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These 6 Incredible Discoveries From The Past Decade Have Changed Science Forever - ScienceAlert

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