Archive for the ‘Crispr’ Category

The CRISPR and CRISPR-associated (Cas) Genes market is anticipated to grow in the forecast, owing to the factors such as rising adoption of genome editing technique, growing adoption of CRISPR, and increasing prevalence of genetic disorders. Furthermore, increasing demand for drug discovery is likely to pose growth opportunities for the CRISPR and CRISPR-associated (Cas) Genes market to grow.

CRISPR & CRISPR-associated (Cas) Genes Market to 2027 Global Analysis and Forecasts By Product (Vector-based Cas, DNA-free Cas); Application (Genome Engineering, Disease models, Functional Genomics, Knockdown/activation, Others); End User (Biotechnology and Pharmaceutical Companies, Academic and Government Research Institutes, Contract Research Organizations) and Geography

CRISPR and CRISPR-Associated (Cas) Genes is a genome editing tool that enables the researchers to make changes in the DNA. CRISPR-Cas9 stands for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. In recent years the CRISPR and CRISPR-Associated (Cas) Genes has gained lot of popularity as it offers it is cheaper, faster, accurate, and more efficient genome editing methods.

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What the report features:-

Leading key market players mentioned in the report:-

The Global CRISPR and CRISPR-associated (Cas) Genes market Analysis to 2027 is a specialized and in-depth study of the medical device industry with a special focus on the global market trend analysis. The report aims to provide an overview of CRISPR and CRISPR-associated (Cas) Genes market with detailed market segmentation by product, application, end user and geography. The global CRISPR and CRISPR-associated (Cas) Genes market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading CRISPR and CRISPR-associated (Cas) Genes market players and offers key trends and opportunities in the market.

The global CRISPR and CRISPR-associated (Cas) Genes market is segmented on the basis of product, application, and end user. Based on product the market is segmented into vector-based Cas and DNA-free Cas. Based on application the market is segmented into genome engineering, disease models, functional genomics, knockdown/activation and others. Based on end user the market is segmented into biotechnology and pharmaceutical companies, academic and government research institutes, contract research organizations.

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2019 CRISPR & CRISPR-associated Genes Market to Emerge with Increasing Demand for Drug Discovery with Top Companies Insights - Thermo Fisher...

CRISPR Therapeutics AG (CRSP):

If you are considering getting into the day trading or penny stock market, its a legitimate and profitable method for making a living. Every good investor knows that in order to make money on any investment, you must first understand all aspects of it, so lets look at daily change, stock price movement in some particular time frame, volatility update, performance indicators and technical analysis and analyst rating. Picking a stock is very difficult job. There are many factors to consider before choosing a right stock to invest in it. If picking stock was easy, everyone would be rich right? This piece of financial article provides a short snap of CRISPR Therapeutics AG (CRSP) regarding Tuesday trading session and presents some other indicators that can help you to support yours research about CRISPR Therapeutics AG (CRSP).

CRISPR Therapeutics AG (CRSP) stock Trading Summary:

CRISPR Therapeutics AG (CRSP) stock changed position at -2.62% to closing price of $39 in recent trading session. The last closing price represents the price at which the last trade occurred. The last price is also the price on which most charts are based; the chart updates with each change of the last price. The stock registered Tuesday volume of 666406 shares. Daily volume is the number of shares that are traded during one trading day. High volume is an indication that a stock is actively traded, and low volume is an indication that a stock is less actively traded. Some stocks tend always to have high volume, as they are popular among day traders and investors alike. Other stocks tend always to have low volume, and arent of particular interest to short-term traders. The stock average trading capacity stands with 502.9K shares and relative volume is now at 1.33.

CRISPR Therapeutics AG (CRSP) Stock Price Movement in past 50 Days period and 52-Week period

CRISPR Therapeutics AG (CRSP) stock demonstrated 75.52% move opposition to 12-month low and unveiled a move of -27.64% versus to 12-month high. The recent trading activity has given its price a change of -22.68% to its 50 Day High and 9.98% move versus to its 50 Day Low. Prices of commodities, securities and stocks fluctuate frequently, recording highest and lowest figures at different points of time in the market. A figure recorded as the highest/lowest price of the security, bond or stock over the period of past 52 weeks is generally referred to as its 52-week high/ low. It is an important parameter for investors (as they compare the current trading price of the stocks and bonds to the highest/lowest prices they have reached in the past 52 weeks) in making investment decisions. It also plays an important role in determination of the predicted future prices of the stock.

CRISPR Therapeutics AG (CRSP) Stock Past Performance

CRISPR Therapeutics AG (CRSP) stock revealed -18.24% return for the recent month and disclosed -20.63% return in 3-month period. The stock grabbed 1.11% return over last 6-months and 8.21% return in yearly time period. To measure stock performance since start of the year, it resulted a change of 36.51%. Past performance shows you the funds track record, but do remember that past performance is not an indication of future performance. Read the historical performance of the stock critically and make sure to take into account both long- and short-term performance. Past performance is just one piece of the puzzle when evaluating investments. Understanding how performance fits in with your overall investing strategy and what else should be considered can keep you from developing tunnel vision.

Volatility in Focus:

The stock unfolded volatility at 4.71% during a week and it has been swapped around 5.62% over a month. Volatility is a rate at which the price of a security increases or decreases for a given set of returns. Volatility is measured by calculating the standard deviation of the annualized returns over a given period of time. It shows the range to which the price of a security may increase or decrease. Volatility measures the risk of a security. It is used in option pricing formula to gauge the fluctuations in the returns of the underlying assets. Volatility indicates the pricing behavior of the security and helps estimate the fluctuations that may happen in a short period of time. If the prices of a security fluctuate rapidly in a short time span, it is termed to have high volatility. If the prices of a security fluctuate slowly in a longer time span, it is termed to have low volatility.

The average true range is a volatility indicator. This stocks Average True Range (ATR) is currently standing at 2.06.

Overbought and Oversold levels

The stock has RSI reading of 43.34. RSI gives an indication of the impending reversals or reaction in price of a security. RSI moves in the range of 0 and 100. So an RSI of 0 means that the stock price has fallen in all of the 14 trading days. Similarly, an RSI of 100 means that the stock price has risen in all of the 14 trading days. In technical analysis, an RSI of above 70 is considered an overbought area while an RSI of less than 30 is considered as an oversold area. RSI can be used as a leading indicator as it normally tops and bottoms ahead of the market, thereby indicating an imminent correction in the price of a security. It is pertinent to note that the levels of 70 and 30 needs to be adjusted according to the inherent volatility of the security in question.

Analyst Watch: Analysts have assigned their consensus opinion on this stock with rating of 2.3 on scale of 1 to 5. 1 or 2 =>Buy view 4 or 5 => Sell opinion. 3 =>Hold. Analysts recommendations are the fountainhead of equity research reports and should be used in tangent with proprietary research and investment methodologies in order to make investment decisions.

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Attractive Stock of Yesterday : CRISPR Therapeutics AG (CRSP) - WEB NEWS OBSERVER

On Monday, shares of CRISPR Therapeutics AG (NASDAQ:CRSP) marked $40.05 per share versus a previous $37.91 closing price. With having a 5.64% gain, an insight into the fundamental values of CRISPR Therapeutics AG, investors would also find a great ally in the technical patterns of the stock movements showed in stock charts. CRSP showed a rise of 40.18% within its YTD performance, with highs and lows between $22.22 $53.90 during the period of 52 weeks, compared to the simple moving average of -1.84% in the period of the last 200 days.

Jefferies equity researchers changed the status of CRISPR Therapeutics AG (NASDAQ: CRSP) shares to a Buy rating in the report published on August 1st, 2019. Other analysts, including Canaccord Genuity, also published their reports on CRSP shares. Canaccord Genuity repeated the rating from the previous report, marking CRSP under Buy rating, in the report published on July 26th, 2019. Additionally, CRSP shares got another Buy rating from ROTH Capital, setting a target price of $50 on the companys shares, according to the report published in June 10th, 2019. On the other hand, William Blair Initiated the Mkt Perform rating for CRSP shares, as published in the report on March 14th, 2019. Goldman seems to be going bullish on the price of CRSP shares, based on the price prediction for CRSP. Another Sell rating came from Citigroup.

The present dividend yield for CRSP owners is set at 0, marking the return investors will get regardless of the companys performance in the upcoming period. In addition, the growth of sales from quarter to quarter is recording -72.70%, hinting the companys progress in the upcoming progress.

In order to gain a clear insight on the performance of CRISPR Therapeutics AG (CRSP) as it may occur in the future, there are more than several well-rounded types of analysis and research techniques, while equity is most certainly one of the more important indicators into the companys growth and performance. In this case, you want to make sure that the return on the present equity of -51.50% is enough for you to make a profit out of your investment. You may also count in the quick ratio of the company, currently set at 14.00 so you would make sure that the company is able to cover the debts it may have, which can be easily seen in annual reports of the company.

Set to affect the volatility of a given stock, the average volume can also be a valuable indicator, while CRSP is currently recording an average of 496.33K in volumes. The volatility of the stock on monthly basis is set at 5.34%, while the weekly volatility levels are marked at 4.30%with 9.19% of gain in the last seven days. Additionally, long-term investors are predicting the target price of $62.13, indicating growth from the present price of $40.05, which can represent yet another valuable research and analysis points that can help you decide whether to invest in CRSP or pass.

CRISPR Therapeutics AG (CRSP) is based in the Switzerland and it represents one of the well-known company operating with Healthcare sector. If you wish to compare CRSP shares with other companies under Electronic Equipment and Consumer Goods, a factor to note is the P/E value of for CRISPR Therapeutics AG, while the value can represent an indicator in the future growth of the company in terms of investors expectations. The later value should have a steady growth rate, increasing and growing gradually, which serves the purpose of reliably showcasing the progress of the company. The value -3.93 is supported by the yearly ESP growth of -101.60%.

Besides from looking into the fundamentals, you should also note the number of people inside the company owning the shares, as the values should be in line with the expectations of investors. In that spirit, the present ownership of stocks inside the company is set at 2.00%, which can provide you with an insight of how involved executives are in owning shares of the company. In oppose to the executives share, the institutional ownership counts 51.10% of shares, carrying an equal significance as an indicator of value, as the presence of large investors may signal a strong company.

It appears that more than several institutional investors and hedge funds decided to increase stakes in CRSP in the recent period. That is how ARK Investment Management LLC now has an increase position in CRSP by 34.67% in the first quarter, owning 2.72 million shares of CRSP stocks, with the value of $111.67 million after the purchase of an additional 701,332 shares during the last quarter. In the meanwhile, Nikko Asset Management Americas, also increased their stake in CRSP shares changed 324.31% in the first quarter, which means that the company now owns 1.87 million shares of company, all valued at $76.71 million after the acquisition of additional 1,430,364 shares during the last quarter.

Waddell & Reed Investment Managem acquired a new position in CRISPR Therapeutics AG during the first quarter, with the value of $41.07 million, and Federated Global Investment Manag increased their stake in the companys shares by 8.82% in the first quarter, now owning 67,400 shares valued at $34.09 million after the acquisition of the additional 831663 shares during the last quarter. In the end, Bellevue Asset Management AG increased their position by during the first quarter, now owning 810462 CRSP shares, now holding the value of $33.22 million in CRSP with the purchase of the additional 810,462 shares during the period of the last quarter. At the present, 51.10% of CRSP shares are in the ownership of institutional investors.

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At Current Price, Is It Too Late To Buy CRISPR Therapeutics AG (CRSP)? - US Post News

As biologys first big science collaboration, the international Human Genome Project mapped and sequenced the entire human genome, paving the way for unparalleled innovations in medicine, biotech and life sciences.

More than 8 million people can now trace their origins to this scientific breakthrough, which began with a single in vitro fertilization (IVF) birth four decades ago. By 2100, IVF could be responsible for 3.5 percent of the global population.

This relatively simple gene-editing technique carries world-changing implications: By allowing scientists to precisely change an organisms DNA on the spot, CRISPR could eradicate inherited diseases or cure existing ones. Since its inception in 2012, CRISPR has fueled much controversy too, as teams look to modify everything from crops to mosquitos. That discussion reached a fever pitch this year after a scientist in China claimed to have created the worlds first babies genetically edited with CRISPR.

Identifying individuals based on hair, blood or other biological samples may seem a given now. But its only possible because of this breakthrough sciencewhich also has led to new findings in cancer and genetic conditions.

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Top 10 biotech innovations of all time, including CRISPR, IVF babies - Genetic Literacy Project

Humankind is on the verge of a genetic revolution that holds great promise and potential. It will change the ways food is grown, medicine is produced, animals are altered and will give rise to new ways of producing plastics, biofuels and chemicals.

Many object to the genetic revolution, insisting we should not be playing God by tinkering with the building blocks of life; we should leave the genie in the bottle. This is the view held by many opponents of GMO foods. But few transformative scientific advances are widely embraced at first. Once a discovery has been made and its impact widely felt it is impossible to stop despite the pleas of doubters and critics concerned about potential unintended consequences. Otherwise, science would not have experienced great leaps throughout historyand we would still be living a primitive existence.

[Editors note: This is the first in a four-part series examining genetic engineerings impact on our lives. The second installment examines regulatory obstacles blunting the potential of genetically engineered animals;the third looks at the role of gene editing in medicine; and the final segment looks at synthetic biology and other novel applications.]

Gene editing of humans and plantsa revolutionary technique developed just a few years ago that makes genetic tinkering dramatically easier, safer and less expensivehas begun to accelerate this revolution. University of California-Berkeley biochemistJennifer Doudna, one of the co-inventors of the CRISPR technique:

Within the next few years, this new biotechnology will give us higher-yielding crops, healthier livestock, and more nutritious foods. Within a few decades, we might well have genetically engineered pigs that can serve as human organ donorswe are on the cusp of a new era in the history of life on earthan age in which humans exercise an unprecedented level of control over the genetic composition of the species that co-inhabit our planet. It wont be long before CRISPR allows us to bend nature to our will in the way that humans have dreamed of since prehistory.

The four articles in this series will examine the dramatic changes that gene editing and other forms of genetic engineering will usher in.

Despite the best efforts of opponents, GE crops have become so embedded and pervasive in the food systemseven in Europe which has bans in place on growing GMOs in most countriesthat it is impossible to dislodge them without doing serious damage to the agricultural sector and boosting food costs for consumers.

Even countries which ban the growing of GMOs or who have such strict labeling laws that few foods with GE ingredients are sold in supermarkets are huge consumers of GE products.

Europe is one of the largest importers of GMO feed in the world. Most of the meat we consume from cattle, sheep, goats, chickens, turkeys, pigs and fish farms are fed genetically modified corn, soybeans and alfalfa.

And the overwhelming majority of cheeses are made with an enzyme produced by GM microbes and some beers and wines are made with genetically engineered yeast.

North America, much of South America and Australia are major consumers of foods grown from GE seeds. Much of the corn oil, cotton seed oil, soybean oil and canola oil used for frying and cooking, and in salad dressings and mayonnaise is genetically modified. GM soybeans are used to make tofu, miso, soybean meal, soy ice cream, soy flour and soy milk. GM corn is processed into corn starch and corn syrup and is used to make whiskey. Much of our sugar is derived from GM sugar beets and GE sugarcane is on the horizon. Over 90 percent of the papaya grown in Hawaii has been genetically modified to make it resistant to the ringspot virus. Some of the squash eaten in the US is made from GM disease-resistant seeds and developing countries are field testing GM disease-resistant cassava.

Many critics of GE in agriculture focus on the fact that by volume most crops are used in commodity food manufacturing, specifically corn and soybeans. One reason for that is the high cost of getting new traits approved. Indeed, research continues on commodity crops, although many of the scientists work for academia and independent research institutes.

For example, in November 2016, researchers in the UK were granted the authority to begin trials of a genetically engineered wheat that has the potential to increase yields by 40 percent. The wheat, altered to produce a higher level of an enzyme critical for turning sunlight and carbon dioxide into plant fuel, was developed in part by Christine Raines, the Head of the School of Biological Sciences at the University of Essex.

A new generation of foods are now on the horizon, some as the result of new breeding techniques (NBTs), such as gene editing. Many of these foods will be nutritionally fortified, which will be critical to boosting the health of many of the poorest people in developing nations and increase yields.

Golden rice is a prime example of such a nutrition-enhanced crop. It is genetically engineered to have high levels of beta carotene, a precursor of Vitamin A. This is particularly important as many people in developing countries suffer from Vitamin A deficiency which leads to blindness and even death. Bangladesh is expected to begin cultivation of golden rice in 2018. The Philippines may also be close to growing it.

A strain of golden rice that includes not only high levels of beta carotene but also high levels of zinc and iron could be commercialized within 5 years. Our results demonstrate that it is possible to combine several essential micronutrients iron, zinc and beta carotene in a single rice plant for healthy nutrition, said Navreet Bhullar, senior scientist at ETH Zurich, which developed the rice.

The Science in the News group at Harvard University discussed some of the next generation foods.

Looking beyond Golden Rice, there are a large number of biofortified staple crops in development. Many of these crops are designed to supply other micronutrients, notably vitamin E in corn, canola and soybeansProtein content is also a key focus; protein-energy malnutrition affects 25% of children because many staple crops have low levels ofessential amino acids. Essential amino acids are building blocks of proteins and must be taken in through the diet or supplements. So far, corn, canola, and soybeans have been engineered to contain higher amounts of the essential amino acid lysine. Crops like corn, potatoes and sugar beets have also been modified to contain more dietary fiber, a component with multiple positive health benefits.

Other vitamin-enhanced crops have been developed though they have yet to be commercialized. Australian scientists created a GE Vitamin A enriched banana, scientists in Kenya developed GE Vitamin A enhanced sorghum and plant scientists in Switzerland developed a GE Vitamin B6 enhanced cassava plant.

Scientists genetically engineered canola, a type of rapeseed, to produce additional omega-3 fatty acids. Research is being conducted on developing GM gluten free wheat and vegetables with higher levels of Vitamin E to fight heart disease.

Other more consumer-focused genetically-engineered crops that do not use transgenics, and have sailed through the approval system include:

Other products are in development that fight viruses and disease. Scientists have used genetic engineering to develop disease-resistant rice. A new plum variety resists the plum pox virus. It has not yet been commercialized. GE solutions may be the only answer to save the orange industry from citrus greening, which is devastating orange groves in Florida. GE might be utilized to curb the damage caused by stem rust fungus in wheat and diseases effecting the coffee crop.

In Africa, GE solutions could be used to combat the ravages of banana wilt and cassava brown streak disease and diseases that impact cocoa trees and potatoes. A GE bean has been developed in Brazil that is resistant to the golden mosaic virus. Researchers at the University of Florida, the University of California-Berkeley and the 2Blades Foundation have developed a disease resistant GM tomato.

Scientists at the John Innes Center in the UK are attempting to create a strain of barley capable of making its own ammonium fertilizer from nitrogen in the soil. This would be particularly beneficial to farmers who grow crops in poor soil conditions or who lack the financial resources to buy synthetic fertilizers.

Peggy Ozias-Akins, a horticulture expert at the University of Georgia has developed and tested genetically-engineered peanuts that do not produce two proteins linked to intense allergens.

New gene editing techniques (NBTs) such as CRISPR offer great potential and face lower approval hurdles, at least for now.

In June 2017, the EPA approved a new first of its kind GE corn known as SmartStaxPro, in which the plants genes are tweaked without transgenics to produce a natural toxin designed to kill western corn rootworm larvae. It also produces a piece of RNA that shuts down a specific gene in the larvae, thereby killing them. The new GE corn is expected to be commercialized by the end of the decade.

What could slowor even stopthis revolution? In an opinion piece for Nature Biology, Richard B. Flavell, a British molecular biologist and former director of the John Innes Center in the UK, which conducts research in plant science, genetics and microbiology, warned about the dangers of vilifying and hindering new GE technologies:

The consequences of simply sustaining the chaotic status quoin which GMOs and other innovative plant products are summarily demonized by activists and the organic lobbyare frightening when one considers mounting challenges to food production, balanced nutrition and poverty alleviation across the world. Those who seek to fuel the GMO versus the non-GMO debate are perpetuating irresolvable difference of opinion. Those who seek to perpetuate the GMO controversy and actively prevent use of new technology to crop breeding are not only on the wrong side of the debate, they are on the wrong side of the evidence. If they continue to uphold beliefs against evidence, they will find themselves on the wrong side of history.

A version of this article previously ran on the GLP on January 24, 2018.

Steven E. Cerier is a freelance international economist and a frequent contributor to the Genetic Literacy Project

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Genetic engineering, CRISPR and food: What the 'revolution' will bring in the near future - Genetic Literacy Project

[Sickle cell] diseaseis caused by a genetic defect that turns red blood cells into hard, sticky, sickle-shaped cells that dont carry oxygen well, clog the bloodstream, damage organs and cause torturous bouts of pain.

The pain is excruciating. Its like being in a car accident and having lightning in your chest. Its a pain that makes a grown woman like me scream, Gray says. Its an overwhelming pain.

Like many sickle cell patients, Victoria had to drop out of school, quit work and spend weeks in the hospital away from her family. Since many sickle cell patients dont survive past their 40s, Gray worries whether shell live to see her children grow up. She just turned 34.

But Gray has hope now, because in July doctors infused billions of her own bone marrow cells back into her body, after editing them with CRISPR.

Scientists used CRISPR to modify a gene in the cells to make them produce fetal hemoglobin, a protein that babies usually stop making shortly after birth. The hope is that the protein produced through the gene-editing treatment will give sickle cell patients like Gray healthy red blood cells.

Read full, original post: A Patient Hopes Gene-Editing Can Help With Pain Of Sickle Cell Disease

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CRISPR gene editing raises hopes of easing the pain associated with sickle cell disease - Genetic Literacy Project

New York City, NY: October 19, 2019 Published via (Wired Release) Global CRISPR Technology Market Research Reportrepresents the proficient analysis of CRISPR Technology industry providing a competitive study of leading market players, market growth, consumption(sales) volume, key drivers and limiting factors, future projections for the new-comer to plan their strategies for business. Further, the report contains the study of CRISPR Technology market ups and downs of the past few years and forecasts sales investment data from 2019 to 2028.

The CRISPR Technology Report outlining the vitals details which are based on manufacturing region, top players, type, applications and so on will gives the transparent view of Industry. The important presence of different regional and local players of CRISPR Technology market is tremendously competitive. The CRISPR Technology Report is beneficial to recognize the annual revenue of key players, business strategies, key company profiles and their benefaction to the market share.

Download Free Sample Copy of CRISPR Technology Market Report:https://marketresearch.biz/report/crispr-technology-market/request-sample

Top Manufacturers Are Covered in This Report:Thermo Fisher Scientific Inc, Merck KGaA, GenScript Corporation, Integrated DNA Technologies Inc, Horizon Discovery Group, Agilent Technologies Inc, Cellecta Inc, GeneCopoeia Inc, New England Biolabs Inc, Origene Technologies Inc

This research report contains a pictorial representation of important data in the form of graphs, figures, diagrams and tables to make simplified for the users to understand the CRISPR Technology market new trends clearly.

Geographically, report on CRISPR Technology is based on several regions with repect to CRISPR Technology export-import ratio of the region, production and sales volume, share of CRISPR Technology market and growth rate of the industry. Major regions included while preparing the report areNorth America, Latin America, Europe, Middle East, Africa, and Asia Pacific.

The leading players in CRISPR Technology industry are estimated to ahead on these opportunities to invade the global market. CRISPR Technology market size and revenue of key players is assessed using the Bottom-up way.

Reasons for Buying Global CRISPR Technology Market Report

* Report provides in-depth study on changing CRISPR Technology market dynamics.

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* Estimated CRISPR Technology market growth depending on the study of historical and the present size of the industry.

Customize Report AndInquiry For The CRISPR Technology Market Report:https://marketresearch.biz/report/crispr-technology-market/#inquiry

Report Table of Content Gives Exact Idea about Global CRISPR Technology Market Report:

Chapter 1explains CRISPR Technology report necessary market surveillance, product price structure, and study, market scope and size forecast from 2019 to 2028. Although, CRISPR Technology market activity, factors impacting the growth of business also complete analysis of current market holders.

Chapter 2offers detailing of top manufacturers of CRISPR Technology market with their share, sales, and revenue.

Chapters 3, 4, 5studies CRISPR Technology report competitive study based on the type of product, their regional sales and import-export study, the annual growth ratio of the market and the coming years study from 2019 to 2028.

Chapter 6offers a detailed analysis of CRISPR Technology business channels, CRISPR Technology market investors, vendors, CRISPR Technology suppliers, dealers, CRISPR Technology market opportunities and threats.

This content has been distributed via WiredRelease press release distribution service. For press release service inquiry, please reach us at[emailprotected]

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Global CRISPR Technology Market Revenue And Value Chain 2019-2028 - The State News - BBState

We are contrasting Seelos Therapeutics Inc. (NASDAQ:SEEL) and CRISPR Therapeutics AG (NASDAQ:CRSP) on their institutional ownership, analyst recommendations, profitability, risk, dividends, earnings and valuation. They both are Biotechnology companies, competing one another.

Earnings & Valuation

Table 1 demonstrates Seelos Therapeutics Inc. and CRISPR Therapeutics AGs top-line revenue, earnings per share (EPS) and valuation.

Profitability

Table 2 hightlights the return on equity, net margins and return on assets of the two companies.

Liquidity

The Current Ratio of Seelos Therapeutics Inc. is 2.5 while its Quick Ratio stands at 2.5. The Current Ratio of rival CRISPR Therapeutics AG is 15.8 and its Quick Ratio is has 15.8. CRISPR Therapeutics AG is better equipped to clear short and long-term obligations than Seelos Therapeutics Inc.

Analyst Ratings

Seelos Therapeutics Inc. and CRISPR Therapeutics AG Ratings and Recommendations are available on the next table.

Meanwhile, CRISPR Therapeutics AGs consensus target price is $62, while its potential upside is 63.55%.

Institutional & Insider Ownership

Institutional investors held 10.4% of Seelos Therapeutics Inc. shares and 50% of CRISPR Therapeutics AG shares. 18.48% are Seelos Therapeutics Inc.s share held by insiders. Insiders Comparatively, held 2% of CRISPR Therapeutics AG shares.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Seelos Therapeutics Inc. had bearish trend while CRISPR Therapeutics AG had bullish trend.

Summary

CRISPR Therapeutics AG beats on 7 of the 10 factors Seelos Therapeutics Inc.

CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its proprietary clustered, regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9)gene-editing platform in Switzerland. The CRISPR/Cas9 technology allows for changes to genomic DNA. It has a collaboration agreement with Vertex Pharmaceuticals, Incorporated to develop, manufacture, commercialize, sell, and use therapeutics; a license agreement with Anagenesis Biotechnologies SAS; and a service agreement with MaSTherCell SA to develop and manufacture allogeneic CAR-T therapies. The company also has research collaboration agreements with Neon Therapeutics and Massachusetts General Hospital Cancer Center to develop novel T cell therapies for cancer. CRISPR Therapeutics AG is headquartered in Basel, Switzerland.

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Reviewing Seelos Therapeutics Inc. (SEEL)'s and CRISPR Therapeutics AG (NASDAQ:CRSP)'s results - MS Wkly

As the co-inventors of CRISPR/Cas9 gene-editing technology, Jennifer Doudna, Emmanuelle Charpentier and Feng Zhang are typically the first names that spring to mind when CRISPR is being discussed. What you may not know, however, is that the CRISPR mechanism was originally discovered back in the 90s by a particularly humble microbiologist, Francisco Mojica, Professor at the University of Alicante.

Kicking offTechnology Networks Explores the CRISPR Revolution, Professor Mojica, or "Francis", takes us on a journey back to the original research that, despite being deemed "crazy" by members of the scientific community at the time, led to the CRISPR revolution that is anticipated to edit evolution forever.Impossible to anticipate

The greatest finds in scientific discovery are typically unanticipated when a researcher embarks on a study. The discovery of CRISPR is no exception: "It was absolutely impossible to anticipate the huge revolution that we are enjoying nowadays," Mojica tells me as he laughs, still seemingly astounded by it.

In 1992, Mojica was working on his thesis at the University of Alicante. A keen microbiologist, he was studying microorganisms belonging to the Archaea family, a group of prokaryotes that he brands "quite peculiar". These microorganisms are halophiles, meaning they require high-salt conditions to survive. Mojica and colleagues were interested in understanding how Archaea are able to grow in high salinity and, when required, adapt to changes in such salinity.

They opted to sequence their DNA to look for clues in the genome. In a pre-Human Genome Project era, this was quite the task. The scientists didn't have the luxury of sequencing data being at their disposal.

Nevertheless, Mojica and the team's efforts were successful. They discovered that the halophiles' DNA possessed a series of regularly spaced repeats, which they labelled tandem repeats (or TREPS). "We saw that these repeated regions in the halophiles were transcribed, meaning they were active. The cell was reading this information in each of the growing conditions that we tested, and so we knew that they had to be important for the cell," Mojica tells me.

The researchers scoured the literature for evidence of previous work outlining the existence of TREPS. They struck gold in the form of a paper published in 1987 by Ishino and colleagues (a huge feat considering that PubMed was yet to be invented). The Japanese researchers had too stumbled across these TREPS in E. coli.1 A "crazy" hypothesis, and a backlash from the scientific community

Mojica's curiosity was sparked. What function did these repeats serve? They existed in bacteria and archaea thus, surely their origin was ancestral?

"In 1995 we published a paper where we suggested a hypothesis which, nowadays, may sound crazy. We hypothesized that the repeats were involved in the segregation of the chromosomes in cell division," Mojica tells me.2 "It was completely wrong," he laughs, "but it fit with our data at the time."

Mojica's efforts to explore the functional role played by the repeats were greeted with an initial backlash from the scientific community.

"When we didn't have any idea about the role these systems played, we applied for financial support from the Spanish government for our research," he explains. "The government received complaints about the application and, subsequently, I was unable to get any financial support for many years." It immediately occurs to me that the culprits behind the complaints are probably kicking themselves now.

I pause for a second, slightly bewildered, before asking him why. "Initially they said, "You want to explore the role played by some repeats in very peculiar and strange organisms. Maybe you discover the role, maybe you don't. But, in any case, your findings will only apply to these strange organisms."" They doubted the relevance of the research. "That was the first criticism. Next I was implored by the reviewers of the grant to move to a model organism such as E. coli, which I did." Mojica continues, "It was a huge mistake."

Whilst the repeats are transcribed in halophilic archaea, in E. coli, they are repressed unless you create mutations. "I spent many years trying to understand the function of these repeats in a model organism in which the system was not working. I could not get any results," he tells me, surprisingly maintaining an upbeat, positive tone to his voice at all times during this recollection. Unphased by his lack of results, Mojica persisted. In 2000, TREPS received a rebranding after he discovered that the repeats existed in many other organisms that were hardly close on the evolutionary tree. Going forward, the repeats were to be known as short regularly spaced repeats, or SRSRs.3 CRISPR enters scientific literature

By 2001, both Mojica and Ruud Jansen, of Utrecht University, were searching for the repeats in various prokaryotic organisms. Jansen reached out to Mojica to inform him that his research team had discovered genes next to the repeats and wanted to agree on common terminology for the repeats.

Several names were proposed before an agreement was made. "I thought about a few alternatives, of which I just remember RISR and CRISPR," Mojica says. "I introduced them to Ruud, explaining their meaning and the pros and cons of each. CRISPR was the one that considered all the features of the repeats. We agreed to use it in our future publications." In 2002, the first mention of clustered regularly interspaced short palindromic repeats, or CRISPR, appeared in the scientific literature.4A dramatic revelation courtesy of the genomic era

In the early 00s, science entered a "genomics era" in which genome sequencing technologies rapidly advanced, paralleled by increased sequencing data being made available to scientists in public databases.

Such data permitted a revelation for Mojica in his work.

When sequencing one particular strain of E. coli, Mojica discovered that there were sequences between the repeats known as "the spacer regions" of CRISPRs that matched the sequence of a particular virus. Further exploration of sequencing data revealed that this was the case in many other, extremely different organisms. These DNA sequences protected the prokaryotes from being infected by viruses carrying the same sequence in its genome; the virus simply couldn't infect the cell. And so, he realized: "This is an immune system. This is an adaptive immune system!"

As he sits with a wide grin on his face recalling the fine details, it is very clear to me that the sheer thrill of making this discovery remains with Mojica to this day. "It was a very nice surprise," he says.

Unfortunately, the scientist was once again greeted by criticism when he endeavoured to publish his research findings. "The paper was rejected by four different journals for many different reasons. One journal said to us that it wasn't interesting enough and another said we needed more experimental proof. We almost considered not publishing the paper." One of those papers was the journal Nature. He adds: "I guess it was a very new idea. We presented our findings at a conference in Spain and some of my colleagues came to me and suggested that what we were doing was very pretentious and 'overgrown'." Mojica laughs.

The study was eventually published in the Journal of Molecular Evolution in 2005.5

Genome-editing came as a "wonderful surprise"

Following the publication of their discovery in 2005, Mojica and colleagues anticipated that their research findings would have a large impact on the biotechnology, biopharmaceutical and clinical science sectors.

And so, in 2012, when Doudna and Charpentier demonstrated that they had reprogrammed the CRISPR mechanism to function as a gene-editing tool in vitro, Mojica was "wonderfully surprised, and very, very impressed."

Since the 2012 publication, a wide variety of research groups have further developed and manipulated the CRISPR mechanism for an array of purposes, ranging from agriculture, diagnostics, drug development, cancer research the list goes on, and will be explored throughout the series.

I am intrigued to know what Mojica deems his favourite application of CRISPR thus far. "My goodness, every single one of them has been astonishing," he continues, "I cannot choose one. I must choose two! And they are the two papers published back to back in Science in 2013." Mojica is referring to a paper published by Feng Zhang that was followed by a second paper in the same journal by George Church.6,7Both research groups outlined their novel use of the CRISPR tool to edit the genome of mice and human cells, igniting the CRISPR genome-editing "revolution".

Filing a patent? I never thought about it An ongoing patent dispute lurks behind the excitement and flurry surrounding CRISPR technology, which will be explored in a later instalment of the series. Interestingly, Mojica is one of few scientists involved in the discovery of the CRISPR mechanism and its applications that has not filed a patent.

"Some people ask me why I didn't file a patent 10 years ago," he pauses. "I have to confess, I never thought about doing that. In my lab, we aim to understand biology. Filing patents probably should be one of the goals," he laughs before adding, "But it is not."

He then goes on to express his anxieties regarding the impact the patent dispute may have on the progress of CRISPR research and applications. "I'm pretty sure the patent dispute could be slowing down the transfer of experiments and research from the lab to the clinic. I'm not absolutely sure, but I fear that could be a problem, and that isn't fair." He adds "It's quite difficult for me to understand why there is such a long-lasting dispute on getting money from research."

Mojica strikes me as a passionate scientist who truly thrives on the quest for novel discovery and is modest in doing so. When asked in a previous interview how he would react to being awarded a Nobel Prize for his work in the CRISPR field, he admitted, "I will disappear from the planet. I need to rest and relax, and I need time to get back to what motivates me and return to the lab." Looking to the future of CRISPR

Mojica's work in this the field of CRISPR is certainly far from finished. He tells me, "We are still interested in understanding how the CRISPR mechanisms work in nature; particularly how these systems develop the memory of past infections. There is a huge diversity of CRISPR Cas mechanisms, and different systems work differently in a variety of organisms." "We are using metagenomics high-throughput sequencing to identify more CRISPR Cas systems and variants that are different to those we know of currently. We hope that either our group, or other groups across the globe can look to identify further applications for these systems or improve the current CRISPR tools we have now."

I ask Mojica what he envisions the CRISPR research field to look like in 10 years' time.

"I am a microbiologist, I'm not a specialist in genome editing but I do read a lot!" he laughs. " It's risky to predict any situation, but I wish that in 10 years' time CRISPR will already be in the clinic, and some patients will have been cured from diseases that currently have limited treatment options. Who knows exactly how many diseases could be tackled by CRISPR."

"A reality right now is CRISPR's application in agriculture. I do anticipate that in some countries, we will soon be eating food that is derived from CRISPR-edited crops. But it's risky to predict any situation."

As our interview comes to a close, I express my sheer gratitude to Mojica for lending his time to me and for sharing his CRISPR story. He replies, humble as ever, "The pleasure is all mine."

ProfessorFrancisco Mojica was speaking with Molly Campbell, Science Writer, Technology Networks.

References:

1. Ishino, Shinagawa, Makino, Amemura, and Nakata. 1987. Nucleotide Sequence of the iap Gene, Responsible for Alkaline Phosphatase Isozyme Conversion in Escherichia coli, and Identification of the Gene Product. Journal of Bacteriology. DOI: 10.1128/jb.169.12.5429-5433.1987.

2. Mojica, Ferrer, Juez and Rodrguez-Valera. 1995. Long stretches of short tandem repeats are present in the largest replicons of the Archaea Haloferax mediterranei and Haloferax volcanii and could be involved in replicon partitioning. Molecular Microbiology. DOI: 10.1111/j.1365-2958.1995.mmi_17010085.x

3. Mojica, Dez-Villaseor, Soria and Juez. 2000. Biological significance of a family of regularly spaced repeats in the genomes of Archaea, Bacteria and mitochondria. Molecular Microbiology. DOI: 10.1046/j.1365-2958.2000.01838.x

4. Jansen, Embden, Gaastra and Schouls. 2002. Identification of genes that are associated with DNA repeats in prokaryotes. Molecular Microbiology. DOI: 10.1046/j.1365-2958.2002.02839.x

5. Mojica, Dez-Villaseor, Garca-Martnez and Soria. 2005. Intervening Sequences of Regularly Spaced Prokaryotic Repeats Derive from Foreign Genetic Elements. Journal of Molecular Evolution. https://doi.org/10.1007/s00239-004-0046-3.

6. Cong et al. 2012. Multiplex genome engineering using CRISPR/Cas systems. Science. DOI: doi: 10.1126/science.1231143.

7. Mali et al. 2012. RNA-Guided Human Genome Engineering via Cas9. Science. DOI: 10.1126/science.1232033.

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Francis Mojica: The Modest Microbiologist Who Discovered and Named CRISPR - Technology Networks

CRISPR And CRISPR-Associated (Cas) Genes Market Report provides a relevant source of perceptive data for investors. CRISPR And CRISPR-Associated (Cas) Genes Market Report also examines global CRISPR And CRISPR-Associated (Cas) Genes Industry growth analysis, the past and innovative cost, demand and supply information, and revenue.

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Clustered regularly interspaced short palindromic repeats (CRISPR) are segments of prokaryotic DNA containing short repetitions of base sequences. Each repetition is followed by short segments of spacer DNA from previous exposures to a bacteriophage virus or plasmid.

The CRISPR/Cas system is a prokaryotic immune system that confers resistance to foreign genetic elements such as those present within plasmids and phages, and provides a form of acquired immunity. CRISPR associated proteins (Cas) use the CRISPR spacers to recognize and cut these exogenous genetic elements in a manner analogous to RNA interference in eukaryotic organisms. CRISPRs are found in approximately 40% of sequenced bacterial genomes and 90% of sequenced archaea.

, CRISPR And CRISPR-Associated (Cas) Genes industry is relatively concentrated, manufacturers are mostly in the Europe and North America. Among them, North America region accounted for more than 45.70% of the total market of global CRISPR And CRISPR-Associated (Cas) Genes.

Although this market has great potential for future development, we do not recommend entering the market for investors who do not have strong capital or do not have key technology.

TheGlobal CRISPR And CRISPR-Associated (Cas) Genes market is valued at 350 million US$ in 2018 and will reach 5220 million US$ by the end of 2025, growing at a CAGR of 40.0% during 2019-2025. The objectives of this study are to define, segment, and project the size ofThe CRISPR And CRISPR-Associated (Cas) Genes market based on company, product type, end user and key regions.

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List of Major CRISPR And CRISPR-Associated (Cas) Genes marketcompetition by top manufacturers, with production, price, and revenue (value) and market share for each manufactures:

The report also focuses on global major leading industry players of Global CRISPR And CRISPR-Associated (Cas) Genes market providing information such as company profiles, product picture, and specification, capacity, production, price, cost, revenue and contact information. Upstream raw materials and equipment and downstream demand analysis are also carried out. The Global CRISPR And CRISPR-Associated (Cas) Genes market development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and overall research conclusions offered.

By theproduct type, the market is primarily split into:

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By theend users/application,this report covers the following segments:

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Global CRISPR And CRISPR-Associated (Cas) Genes Market Boosting the Growth Worldwide: 2019 Market Key Dynamics, Recent and Future Demand, Trends,...

CRISPR Therapeutics to receive non-exclusive access to certain KSQ IP for its allogeneic CAR-T programs

KSQ Therapeutics to receive non-exclusive access to certain CRISPR IP for its autologous cell therapies, including its existing eTILTM cell franchise

ZUG, Switzerland & CAMBRIDGE, Mass.(BUSINESS WIRE)CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and KSQ Therapeutics, a biotechnology company using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development, today announced a license agreement whereby CRISPR Therapeutics will gain access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ will gain access to CRISPR Therapeutics IP for editing novel gene targets identified by KSQ as part of its current and future eTILTM (engineered tumor infiltrating lymphocyte) cell programs. The financial terms of the agreement are not being disclosed.

We are thrilled to gain access to CRISPR Therapeutics foundational IP estate through this agreement, said David Meeker, M.D., Chief Executive Officer at KSQ Therapeutics. Our eTILTM programs involve editing gene targets in human TILs that were discovered at KSQ by applying our proprietary CRISPRomics approach to immune cells in multiple in vivo models. This agreement clears an important path for us to be able to bring these programs through development and commercialization, leveraging CRISPR Therapeutics proprietary editing technology.

The gene targets within the scope of the license agreement were identified using KSQs proprietary CRISPRomics drug discovery engine, which allows genome-scale, in vivo validated, unbiased drug discovery. These specific targets were uncovered in screens to identify genetic edits that could enhance the functionality and quality of adoptive cell therapies in oncology.

KSQ has built an industry-leading platform to screen for novel gene targets using its technology, and has identified a group of targets that could help unlock the full potential of adoptive cell therapy in oncology, said Samarth Kulkarni, Ph.D., Chief Executive Officer at CRISPR Therapeutics. As a result of this license agreement, CRISPR Therapeutics will have the opportunity to bring these novel targets into our leading allogeneic CAR-T development platform to further strengthen our future programs in this important therapeutic area.

About KSQ Therapeutics

KSQ Therapeutics is using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development. The company is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer, across multiple drug modalities including targeted therapies, adoptive cell therapies and immuno-therapies. KSQs proprietary CRISPRomics drug discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies, and the company is located in Cambridge, Massachusetts. For more information, please visit the companys website at http://www.ksqtx.com.

About CRISPR Therapeutics

CRISPR 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.

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SECs website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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CRISPR Therapeutics and KSQ Therapeutics Announce License Agreement to Advance Companies' Respective Cell Therapy Programs in Oncology - SynBioBeta

Scientists have successfully grown tomatoes, leeks, potatoes and several other crops in simulated Lunar and Martian soil, meaning that humans could survive off a closed-loop agriculture ecosystem on Mars. They also emphasized that cellular agriculture and insect farming would be instrumental to feeding a future Martian population.Read more at Modern Farmer

CRISPR technology is a precise form of gene-editing that genetically modifies foods in a faster and cheaper way than ever beforeand its been subtly present in the dairy aisle for quite some time. But while this tool could transform agriculture as we know it, there is much scientists still dont know about the ramifications of using it on the environment and human health.Read more at The Atlantic

Emerging data shows that the boom in consumer desire for convenient, ready-to-cook frozen fare is sweeping the frozen fish and seafood section. Seafood marketers and producers should pay special attention to childless, single adults over 35 and senior couplestheyre driving the dollar growth in this category.Read more at Nielsen

In a statement published yesterday the Federal Drug Administration solidified its position on the use of cannabidiol (CBD) by pregnant women. While there is no comprehensive research on the effects of the popular ingredient on fetuses, the administration argues that the potential for contamination in CBD products is too high to ignore and cites a study wherein high doses of CBD in pregnant test animals adversely affected developing male fetuses. Read more at FDA.gov

Small farmers that use responsible and regenerative growing practices are few and far between now that industrialized, multinational corporations have a stronghold on the U.S. food system. Just 43% of farms are profitable according to USDA, and federal policies are in place that work to uphold the soil-depleting practices of larger corporations. Whats clear is that the government immediately must act to improve crop subsidies, increase biodiversity, promote soil health, use integrated pest management practices and manage energy use via regenerative actions to prevent an agricultural crisis.Read more at Quartz

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5@5: Crops on Mars? | The future of CRISPR - New Hope Network

CAMBRIDGE, Mass., Oct. 16, 2019 (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, announced one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain.

Intellias data includes important updates about the companys programs and platform development activities:

Oral Presentation:

In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellias gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellias similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.Abstract number: OR48Session 5b: New delivery systems and technologiesPresentation date/time: Friday, October 25, 2019, 11:30 a.m. 1:30 p.m. CETLocation: Room 113-115

Poster Presentations:

In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics

This poster presentation will highlight Intellias approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel OConnell, Ph.D.Poster ID Number: P655Date: Wednesday, October 23, 2019

Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia

This poster presentation focuses on Intellias ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San RaffaelePoster ID Number: P111Date: Wednesday, October 23, 2019

Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout

This poster presentation focuses on the companys T cell engineering technology, which is being applied in its Wilms Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.Poster ID Number: P162Date: Thursday, October 24, 2019

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.Poster ID Number: P552Date: Thursday, October 24, 2019

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing 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 aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia 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 IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms; develop our proprietary LNP/AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; 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 and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half 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, including through our arbitration proceedings against Caribou; 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; the risk that the results of preclinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivocollaborations will not continue or will not be successful. 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 theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

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

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

Excerpt from:
Intellia Therapeutics Announces Presentations at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) - BioSpace

As word spread in 2018 about the birth of the world's first genetically altered babies, concerns over their future health mounted, with one study even raising the tragic possibility of shortened lives for the newborns. That risk now seems far less likely.

The alarming paper published in Nature last June has now been retracted by the authors themselves, who in the wake of criticism admit the way they searched for signs of a mutated gene in a data sample left too much room for doubt.

It's an important lesson not only in how science values self-correction, but how researchers need to tread lightly as they trawl through population-sized databanks in search of new discoveries.

"I feel I have a responsibility to put the record straight for the public," University of California population geneticist, Rasmus Nielsen, told Ewen Callaway at Nature.

The gene at the centre of the research serves as a template for a receptor on white blood cells.

Called CCR5, its usual job is to detect chemical signals used in immune responses. Unfortunately the deadly human immunodeficiency virus (HIV) evolved to use it as a window to gain entry into the cells.

Ever since the receptor's role in HIV infection was discovered, researchers have wondered just how important this receptor really is. Would we really miss it if it was gone?

Luckily an answer might be found among a percentage of people of European descent with a naturally occurring 'broken' version of CCR5 called delta-32. Those who carry a single copy of the delta-32 variant seem to be less susceptible to HIV than the rest of the population.

In November 2018 a Chinese geneticist named He Jiankui claimed to have used the engineering technology CRISPR-Cas 9 to alter the CCR5 genes in human embryos to artificially give them resistance.

He Jiankui's initial announcement suggested at least one of the twins was carrying two altered CCR5 genes. While they don't appear to match the delta-32 variants, it was enough to invite speculation over what kind of lives the children might have.

HIV resistance is no doubt a good thing in a world where the disease it causes is still destroying too many lives. But those benefits to any one individual might not be so great if a low quality CCR5 receptor raises the risks of developing other health problems.

Nielsen and his colleagues intended to answer this question by looking for similarly altered versions of the CCR5 gene in the UK Biobank's giant genetics database.

They estimated about 1 percent of the records in the database came from individuals with two delta-32 variant copies of the gene. Importantly, they calculated that this tiny fraction was 21 percent more likely to die before their 76th birthday, compared with those at least one 'normal' copy of the gene.

Thankfully, as happens in science, big claims often attract sceptical inquiries. Others quickly dived into the statistics in search of similar correlations using both the UK Biobank and other nation's datasets, coming up empty handed.

So where did Nielsen and his colleagues go wrong?

The cause of the discrepancy could lie in how the data was collected in the first place.

One way to work out whether a person has a specific gene is to simply use a template that sticks to a target sequence. These probes don't always work perfectly, meaning some people will incorrectly appear as negative in the database.

By potentially undercounting the number of people with the CCR5 delta-32 receptor, Nielsen risked masking the true impact of the mutation, making it look like there is a difference in mortality statistics. Which is why he asked for the paper to be retracted.

For researchers, huge banks of genetic and medical data collected from across a population provide the necessary quantities of information needed to spot subtle patterns that demarcate healthy from unhealthy bodies.

Yet as potentially useful as those statistics are, there's dangers in forgetting they come with plenty of assumptions.

You can see the now-retracted paper here.

Read more:
Researchers Admit They Were Wrong to Predict Early Death of The Famed CRISPR Babies - ScienceAlert

Theres broad consensus that genetically modifying humans isnt a good idea, at least not anytime in the near future. But it seems Russia has less qualms about the idea, which could leave it to determine the future of the technology.

After Chinese geneticist He Jiankui announced he had used CRISPR to genetically edit two human embryos there was widespread outrage from both the scientific community and authorities at home and abroad. But it took less than a year for Russian scientist Denis Rebrikov to announce his desire to carry out similar experiments that edit germline DNA, which refers to changes that will be passed on to future generations.

Condemnation from the international community was again swift, but it appears Rebrikov may be finding a more receptive audience at home. Bloomberg reports that a secret meeting of top Russian geneticists and health officials was convened over the summer to discuss the proposals.

And the meeting had a significant guest: Maria Vorontsova, an endocrinologist and daughter of the man likely to make the final call on Russias position on gene-editing President Vladimir Putin.

Bloomberg reports there was a back and forth between opponents and proponents of the idea, but Vorontsova said scientific progress cant be stopped and suggested such research should be controlled by state-run institutions to ensure oversight.

While thats a long way from an official endorsement, the Russian governments response to Rebrikovs plans has certainly been tepid compared to those in the US, where politicians recently renewed a ban on germline editing, and in China, where Hes work quickly led to a tightening of regulations around human gene editing.

Rebrikovs proposal potentially has more merit than Hes. Rebrikov initially planned to target the same gene as He, which is believed to determine susceptibility to HIV. Switching this gene off was criticized for being an unnecessarily complicated and dangerous way of ensuring the disease wasnt passed from parent to child.

Now he plans to use CRISPR to switch off a rare gene that leads to deafness. He is working with couples who are both deaf due to the condition, but dont want to pass it on to their children. Theres still very little understanding of what the potential side effects of this kind of intervention could be, which has led many to call for a moratorium on the technology.

Both the World Health Organization and an international commission set up by the US national academies and the UKs Royal Society are trying to develop guidelines for human gene editing technology, but scientists leading these efforts admit theres little they can do to prevent this kind of research at present.

And while Rebrikovs proposals may sound fairly benign, the way he talks about the technology should give serious cause for concern. In the Bloomberg article he openly discusses starting small and the prospect of parents genetically enhancing their children, while seeming to invoke the Soviet Unions pursuit of nuclear weapons as a justification for developing a technology that can be used for both good and evil.

So far, most of the discussion around germline editing has been focused on safety. But writing in Scientific American Mildred Solomon, president of bioethics institute The Hastings Center, says we need to start tackling questions that go beyond safety before its too late.

That will inevitably include discussions around the ethics of genetic enhancement, but its becoming increasingly clear that there also needs to be consideration of the geopolitical ramifications of the technology.

Putin has already voiced his concerns about genetically-engineered soldiers, and in todays hostile international climate its easy to see the worlds great powers worrying about being left behind by their adversaries. Rebrikov alluded to this train of thought in his comments to Bloomberg, saying hes sure embryo gene-editing is happening in clandestine dark sites.

Despite Chinas forceful public response to Hes research, theres evidence the government was actually funding it, and bioethicist James Giordano told National Defense that its highly unlikely the scientist was a rogue actor in a country where government, academia, and industry are so deeply entwined.

Were still a long way from the kind of capabilities required for doomsday scenarios like super-soldiers or genetically-targeted biological weapons, but recent developments suggest theres a real danger of a genetic arms race developing. Exactly what can be done to stop it remains far from clear, but there needs to be a major push to ensure the fundamental basis of our humanity doesnt end up being governed by realpolitik.

Image Credit: Shutterstock.com

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Russia Could Take the Lead on Human Gene Editing - Singularity Hub

Human Nature is a documentary created by Emmy award-winning producer Adam Bolt with the help of NC State professors that explains what CRISPR is and the impact it could have on society.

CRISPR is a gene-editing tool that allows humans to modify the genetic code of organisms. With this technology, it is possible to cure diseases, create new foods or redefine human life. How can such a thing be possible? Can we as humans be trusted with this technology? These are the questions the documentary answers.

Rodolphe Barrangou, assistant professor of food science at NC State and Todd R. Klaenhammer Distinguished Scholar in probiotics research* and an active participant in the creation of the documentary, spoke about the project over email.

In my opinion, the documentary captures the high potential of genome editing for the benefits of humankind, and also brings up critical questions about the ethical issues that must be assessed, and the importance to capture the many voices of all involved and impacted, Barrangou said.

Barrangou was one of the first people from NC State to be part of the filming process and is considered the driving force behind getting NC State in the documentary. He worked with the film crew to help select the people to feature and the early design of the story.

We spent 11 days on campus and in RTP to feature work underway at NC State in CALS and CVM and also at Syngenta, Barrangou said in the email.

Another example of a voice that can be found in the documentary is that of Jorge Piedrahita, a professor and the director of the Comparative Medicine Institute at NC State. His lab created genetically modified pigs for biomedical research, specifically to carry organs for human use. Piedrahita spoke about the fun experience of working with a professional film crew and how important he believed the CRISPR technology is.

You need to be aware of it because the more you understand it, the more you understand the benefits, the more you understand the risks," Piedrahita said. "You start to understand that the benefits vastly outweigh the risks.

One of the topics Piedrahita discussed was the democratization of the technology and the regulations that would follow it. This would ensure that it wouldnt just be billion-dollar biotech companies with state-of-the-art labs working with CRISPR. The technology would be available to governments and labs worldwide.

CRISPR lies within an ethical gray area, and a large part of the documentary looks objectively at the good and the bad the technology can do. In the documentary trailer, there is a video of Vladimir Putin describing how the technology could be used to create soldiers that would fight without fear or pain. Then, within the same minute, it shows a parent saying, Anything that will stop my child from suffering, Im for.

Piedrahita spoke about the growing importance of CRISPR and its possible impact.

I think it will be crazy for someone not to understand CRISPR, because it will be such a big part of our lives, every single facet of our lives, in the next ten years, Piedrahita said.

From the food people eat, to the way disease is treated, to even the future generations of people, CRISPR will impact everything, which is what makes Human Nature so impactful. The film premiered at the 2019 SXSW Film Festival, and its general release will be on Nov. 7.

* Editor's Note, Oct. 8, 2019: [and Todd R. Klaenhammer Distinguished Scholar in probiotics research] was rephrased for clarity.

See the article here:
'Human Nature': A Film on the Technology Defining the Future - N.C. State University Technician Online

Whencoauthors Ann Lin and Christopher Giuliano, then at Stony Brook University in NewYork, saw their lab results, they were worried. We were both undergrads at thetime, said Lin. It was our first CRISPR experiment, and we were like, is thisour fault, or is this real?

Using CRISPR, Lin and Giuliano had knocked out the gene for MELK, a kinase reported to be essential in multiple cancer types, and of particular interest in triple-negative breast cancer. Surprisingly, they found that breast cancer cells grew happily even without MELK. Even more strangely, the cells lacking MELK remained vulnerable to OTS167, a MELK inhbitor.1Their advisor, Jason Sheltzer, PhDwho is a fellow at Cold Spring Harbor Laboratorywasnt inclined to blame the odd results on undergraduate incompetence. They began pursuing the hypothesis that the drug must exert its killing activity through other proteins or through some other mechanism.

Fourclinical trials are currently underway testing OTS167 in human cancers yet thedrugs mechanism of action may be misunderstood. Its a real problem: targetedtherapies for cancer overwhelmingly fail clinical trials, according to a recentanalysis,2 with only some 3% to 4% of candidates earning approvalfrom the US Food and Drug administration (FDA).

Thisstatistic startled the researchers and prompted them to broaden theirinvestigation. They tested 10 cancer drugs that targeted 6 different proteins,looking to confirm the published mechanisms of action. The target proteins wereHDAC6, MAPK14/p38, PAK4, PBK, PIM1, and CASP3/caspase-3.

Mostof the evidence implicating these proteins as essential for cancer growth camefrom RNA-interference (RNAi) screens, in which short RNA molecules designed tosilence the gene successfully impaired cancer cell growth. In each case, asmall-molecule inhibitor targeting the protein exists, with demonstratedcancer-killing ability. They intentionally selected drug-target pairs that hadno published resistance-granting mutations, which would unequivocally validatethe mechanism of action.

Whenthey knocked out the genes with CRISPR, Lin and Giuliano found that in everycase, inactivating the gene did not diminish the cancer cells survival. Upontesting 4 of the original RNAi constructs that had been used to identify theproteins as essential, the constructs still hampered the cells growth evenwhen the targeted gene of interest had been knocked out.

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Some Targeted Therapies May Miss the Mark - Cancer Therapy Advisor

Researchers have developed a new method to use the gene editing tool, CRISPR, to target specific bacteria and kill them -- an advance that may lead to new techniques for treating bacterial infections, and for customising the gut microbe composition of individuals. The study, published in the journal Nature Communications, increases the possibility of using CRISPR technology to alter the makeup of the human microbiome -- the community of microbes that live in and on us -- in a way that could be personalized for each individual.

The researchers from the University of Western Ontario in Canada said that CRISPR could be programmed to target specific stretches of genetic code, and to edit DNA at precise locations, helping researchers permanently modify genes in living cells and organisms, and also to kill bacteria.

But until now, the researchers said that there wasn't a way to efficiently kill specific bacterial strains.

While the idea of using CRISPR to kill cells and organisms is not new, the researchers noted that the main hurdle was in getting the gene editing tool to target specific cells.

"This technology could also be used to help 'good' bacteria produce compounds to treat diseases caused by protein deficiencies," Karas said.

See the original post here:
Researchers develop gene editing method that may alter microbiome makeup - Gadgets Now

An 11-year-old with OCA2, a form of albinism. Human Nature delves into whether conditions like this should be edited out of human genomes before birth.

Wonder Collaborative

Bottles of nucleic acids at Synthego, a company which synthesizes the key components of CRISPR at an industrial scale.

Wonder Collaborative

When you see something unusual, you automatically assume its interesting, says a microbiologist in Human Nature, a documentary on the science and ethics of genetic editing and engineering. Thats just how science works.

It may be how science works, but its not how filmmaking works. So, while the first chunk of Adam Bolts Human Nature will be catnip for the biochemists, the rank and file Science for Dummies people might find the DNA-coding tutorial DOA. Still, the soundtrack is charismatic and the talking heads are the fun chemistry-teacher types, not the lab-coat introverts.

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David Sanchez, a teenager with sickle-cell disease, looks at a tube containing the CRISPR gene editing machinery.

Wonder Collaborative

A science lesson on the eureka-level technology called CRISPR eventually sets up a lively discussion on the ethics of designer babies and building better humans. An interesting voice belongs to David Sanchez, an upbeat boy with sickle cell anemia who believes his condition gave him an evolved sense of patience and positivity.

I dont think Id be me, if I didnt have have sickle cell, he says. The who dares to play God? discussion is nothing new Aldous Huxleys 1932 dystopian novel Brave New World involved genetically modified citizens but now science fiction has turned into science fact.

In 1993s Jurassic Park, Jeff Goldblums prudent doctor character worried that genetic scientists were too preoccupied with could we? and not enough with should we? With Human Nature, director Bolt offers balance and nuance to the arguments.

Human Nature opens in Toronto and Victoria on Oct. 4, before expanding theatrically across Canada.

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Original post:
Review: DNA-dissecting documentary Human Nature is catnip for scientists and ethicists - The Globe and Mail

The future of genetically modified babies may lie in the hands of Russian president Vladimir Putin,Bloomberg reported over the weekend.

Secret summit:According to Bloomberg, top Russian geneticists held a secret meeting this summer with government health officials in Moscow to debate a bid by a scientist there, Denis Rebrikov, to create babies genetically modified with the gene-editing technology CRISPR.

The first such children were born in China last year as part of a project to make HIV-resistant humans. That undertaking was halted amid pointed criticism of its ethical failings and a criminal investigation.

Putins choice:The question now is whether Russia will grab the CRISPR baton where China dropped it. Dmitry Peskov, the spokesman for Russias leader, declined to give Bloomberg a position, saying gene editing is not a presidential issue.

Putin has already made some comments about gene editing, likening the technology to a nuclear bomb and citing the possibility ofcreating soldiers who feel no pain. According to Bloomberg, Putin last year directed $2 billion to be spent on genetic research that he said will determine the future of the whole world.

Read full, original post: Putin could decide for the world on CRISPR babies

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Will the future of CRISPR babies be decided by Russian president Vladimir Putin? - Genetic Literacy Project

Illinois State University researchers are part of a team thats creating a new crop that could help both the environment and farmers' bottom lines.

TheU.S. Department of Agricultureis givingthem $10 million for researchthat will lead to the planting and harvesting of pennycress, a penny-shaped weed that grows in the spring, as a winter cover crop, which processors can then convert into fuel.

Pennycress has a number of natural attributes that makes it perfect for being a crop, Illinois State University genetics professor John Sedbrook said on WGLT's Sound Ideas. It has extreme cold tolerance, it soaks up the nitrogen so it keeps nitrogen from running into the streams to keep streams clean, and its related to canola.

Cover crops tend to have more environmental than monetary value and provides soil health and natural benefits. Sedbrook and the research team are looking to change that.

To achieve the goal of the project, researchers are using gene editing, called CRISPR, to change the very nature of the original plant.

With CRISPRthis is game changing and going to improve our lives in a lot of different ways, not just crop improvement but treating human diseases. Weve been able to apply CRISPR to rapidly improve pennycress genetically, Sedbrook said.

With just two genetic changes, the team has been able to make pennycress oil and meal edible.

Not only can we use it for food, Sedbrook said, we can also use it for making biodiesel or jet fuel. Its really quite versatile. Another change we made was reducing the fiber content in the seed so the meal has the same nutritional value as canola adding value to pennycress along with the breeding program to get the yields higher where now its economical.

That means farmers can benefit off the cover crop, putting more money in their pockets during what Sedbrook calls a challenging time in farming.

Sedbrook also said converting the oil to biodiesel is not that difficult. With current technology and modern techniques a simple plant like pennycress can be converted to even jet fuel.

But what does it mean to domesticate a plant?

In the past it took thousands of years for cavemen to wait for the right genetic change to come along, Sedbrook said. For wheat there is a handful of changes and for corn there are six changes they have identified that changed the weed teosinte into what we know as corn.

It takes hundreds of thousands of years for evolutionary changes in plants. But with genetic science, changes can be made rapidly.

Sedbrook said the demand for the new strain of pennycresscalledCoverCressis already there.

Theres the old saying build it and theyll come," he said. "There are people just waiting for us to produce this oil and theyre ready to process it."

People like you value experienced, knowledgeable and award-winning journalism that covers meaningful stories in Bloomington-Normal. To support more stories and interviews like this one, please consider making a contribution.

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From Weed to Cash: Researchers Genetically Engineer Pennycress - WGLT News

BERKELEY, Calif., Oct. 1, 2019 /PRNewswire/ --Today, the U.S. Patent and Trademark Office (USPTO) granted a new CRISPR-Cas9 patent to the University of California (UC), University of Vienna, and Dr. Emmanuelle Charpentier covering new methods of the gene-editing technology in prokaryotic cells. The new patent (U.S. Patent No. 10,428,352) covers methods of targeting and binding or methods of cleaving a target DNA in a prokaryotic cell using Cas9 protein and single molecule DNA targeting RNAs. This patent also specifically covers these methods in bacterial cells.

This is the fifth consecutive week that the USPTO has awarded a CRISPR-Cas9 patent to UC, which has immensely increased the compositions and methods covered in the portfolio. The university's total portfolio to-date includes 16 patents, marking the largest CRISPR-Cas9 patent portfolio in the country, and will rise to 18 in the coming weeks, once other applications that the USPTO has allowed are issued as patents. The extensive portfolio covers compositions and methods for the CRISPR-Cas9 gene-editing technology, including targeting and editing genes and modulating transcription in any setting, such as within plant, animal, and human cells.

"The continuous issuance of CRISPR-Cas9 patents to UC adds significant new compositions and methods to our burgeoning portfolio that has quickly become the widest-ranging for the technology," said Eldora L. Ellison, Ph.D., lead patent strategist on CRISPR-Cas9 matters for UC and a Director at Sterne, Kessler, Goldstein & Fox. "We are pleased by the USPTO's ongoing recognition of the Doudna-Charpentier team's leadership related to CRISPR-Cas9."

The Doudna-Charpentier team that invented the CRISPR-Cas9 DNA-targeting technology included Jennifer Doudna and Martin Jinek at the University of California, Berkeley; Emmanuelle Charpentier (then of Umea University); and Krzysztof Chylinski at the University of Vienna. The methods covered by today's patent, as well as the other compositions and methods claimed in UC's previously issued patents and those set to issue, were included among the CRISPR-Cas9 gene editing technology work disclosed first by the Doudna-Charpentier team in its May 25, 2012 priority patent application.

Additional CRISPR-Cas9 patents in this team's portfolio include 10,000,772; 10,113,167; 10,227,611; 10,266,850; 10,301,651; 10,308,961; 10,337,029; 10,351,878; 10,358,658; 10,358,659; 10,385,360; 10,400,253; 10,407,697; 10,415,061; and 10,421,980. These patents are not a part of the PTAB's recently declared interference between 14 UC patent applications and multiple previously issued Broad Institute patents and one application, which jeopardizes essentially all of the Broad's CRISPR patents involving eukaryotic cells.

International patent offices have also recognized the pioneering innovations of the Doudna-Charpentier team, in addition to the 16 patents granted in the U.S. so far. The European Patent Office (representing more than 30 countries), as well as patent offices in the United Kingdom, China, Japan, Australia, New Zealand, Mexico, and other countries, have issued patents for the use of CRISPR-Cas9 gene editing in all types of cells.

University of California has a long-standing commitment to develop and apply its patented technologies, including CRISPR-Cas9, for the betterment of humankind. Consistent with its open-licensing policies, UC allows nonprofit institutions, including academic institutions, to use the technology for non-commercial educational and research purposes.

In the case of CRISPR-Cas9, UC has also encouraged widespread commercialization of the technology through its exclusive license with Caribou Biosciences, Inc. of Berkeley, California. Caribou has sublicensed this patent family to numerous companies worldwide, including Intellia Therapeutics, Inc. for certain human therapeutic applications. Additionally, Dr. Charpentier has licensed the technology to CRISPR Therapeutics AG and ERS Genomics Limited.

SOURCE University of California Office of the President

Original post:
University of California expands US CRISPR-Cas9 patent portfolio with issuance of new patent - PRNewswire

A scientific studypublished this past spring came with damning implications for Chinese scientist He Jiankui, who created the worlds first gene-edited babies: People with the rare genetic variants that Hetried to engineer into embryos, the study asserted, had an increaseddeath rate.

On [September 27], the papers senior author said his study was wrong.

The study centers around the effects of a variant of the gene known as CCR5, called 32, which is best known for protecting against infection with HIV, the virus that causes AIDS.

[Author Rasmus] Nielsen told STAT that the error stemmed from the specific single nucleotide polymorphism, or genetic marker, that he and [collaborator Xinzhu] Wei looked at. In the U.K. Biobank data, the marker they chose to work with had systematic errors related to genotype calling at that site in the DNA; thats the process by which the genotype is determined for each individual in the sample at each site.

The way the genotypes were being called caused certain genotypes to show up less frequently than they should have, Nielsen said, apparently generating the erroneous signal around increased mortality.

Read full, original post: Major error undermines study suggesting change introduced in the CRISPR babies experiment shortens lives

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Researcher backtracks on study suggesting He Jiankui's controversial CRISPR babies will have shorter lifespans - Genetic Literacy Project

XconomyBoston

A number of gene-editing companies have joined the public markets in recent years. Beam Therapeutics, which is developing a CRISPR-based technology intended to offer even more precise genomic edits, aims to become the latest one.

Cambridge, MA-based Beam filed its IPO paperwork with securities regulators late Friday. The company set a preliminary $100 million target for the offering. It has applied for a Nasdaq listing under the stock symbol BEAM.

CRISPR-Cas9 gene editing cuts the genome at specific locations in order to remove or add a piece of DNA. But Beam contends theres room to make CRISPR editing even more precise. If you picture the double helix structure of DNA as a ladder, each rung is made up of a base pair, which consists of two bases. Many genomic mutations occur in a single base. Beams technology, called base editing, is being developed to target these single base errors, which are called point mutations.

If existing gene editing approaches are scissors for the genome, our base editors are pencils, erasing and rewriting one letter in the gene, Beam says in its IPO prospectus.

Beam faces plenty of competition in the gene editing space. Other companies using CRISPR-Cas9 technology to develop new therapies include Caribou Biosciences, Editas Medicine (NASDAQ: EDIT), CRISPR Therapeutics (NASDAQ: CRSP), and Intellia Therapeutics (NASDAQ: NTLA). But the ability to edit point mutations could make the Beam technology applicable to a broader range of genetic diseases. The company says point mutations represent 58 percent of all known genetic errors associated with disease.

Beam was founded in 2017. Until now, Beam has kept quiet about which diseases it aims to treat. The companys filing lists 12 programs, including the blood disorders beta thalassemia and sickle cell disease, and the blood cancers acute lymphoblastic leukemia and acute myeloid leukemia. The pipeline also includes potential treatments for liver diseases, as well as disorders of the eye and the central nervous system.

All of Beams programs are preclinical. For most of them, the company says it has demonstrated therapeutically relevant base editing of cells in the lab. Next year, Beam aims to show that it can base edit genes in animals, tests that are slated for next year. If all goes well, Beam says it could start filing for clearance to begin human testing for multiple programs in 2021.

Beam has raised more than $223 million; its most recent financing was a $135 million Series B round in April. The company still has plenty of money in its coffers: As of June 30, Beam reported $126.8 million in cash holdings. The company says it will use the IPO proceeds to continue research and development of its base editing programs. According to the IPO filing, ARCH Venture partners is Beams largest shareholder with a 23 percent stake followed by F-Prime Capital Partners Healthcare Fund, which owns 19.4 percent of the company.

Heres more on the origins of Beam, which is based on research from Harvard University, the Broad Institute, and Massachusetts General Hospital.

Photo by Flickr user marco.savia a Creative Commons license

Frank Vinluan is editor of Xconomy Raleigh-Durham, based in Research Triangle Park. You can reach him at fvinluan [at] xconomy.com

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Beam Therapeutics Preps IPO and Sheds Light on Its Gene-Editing Drugs - Xconomy

CRISPR Therapeutics AG (CRSP):

If you are considering getting into the day trading or penny stock market, its a legitimate and profitable method for making a living. Every good investor knows that in order to make money on any investment, you must first understand all aspects of it, so lets look at daily change, stock price movement in some particular time frame, volatility update, performance indicators and technical analysis and analyst rating. Picking a stock is very difficult job. There are many factors to consider before choosing a right stock to invest in it. If picking stock was easy, everyone would be rich right? This piece of financial article provides a short snap of CRISPR Therapeutics AG (CRSP) regarding Monday trading session and presents some other indicators that can help you to support yours research about CRISPR Therapeutics AG (CRSP).

CRISPR Therapeutics AG (CRSP) stock Trading Summary:

CRISPR Therapeutics AG (CRSP) stock changed position at -3.23% to closing price of $40.99 in recent trading session. The last closing price represents the price at which the last trade occurred. The last price is also the price on which most charts are based; the chart updates with each change of the last price. The stock registered Monday volume of 435486 shares. Daily volume is the number of shares that are traded during one trading day. High volume is an indication that a stock is actively traded, and low volume is an indication that a stock is less actively traded. Some stocks tend always to have high volume, as they are popular among day traders and investors alike. Other stocks tend always to have low volume, and arent of particular interest to short-term traders. The stock average trading capacity stands with 459.2K shares and relative volume is now at 0.9.

CRISPR Therapeutics AG (CRSP) Stock Price Movement in past 50 Days period and 52-Week period

CRISPR Therapeutics AG (CRSP) stock demonstrated 84.47% move opposition to 12-month low and unveiled a move of -23.95% versus to 12-month high. The recent trading activity has given its price a change of -23.95% to its 50 Day High and -0.02% move versus to its 50 Day Low. Prices of commodities, securities and stocks fluctuate frequently, recording highest and lowest figures at different points of time in the market. A figure recorded as the highest/lowest price of the security, bond or stock over the period of past 52 weeks is generally referred to as its 52-week high/ low. It is an important parameter for investors (as they compare the current trading price of the stocks and bonds to the highest/lowest prices they have reached in the past 52 weeks) in making investment decisions. It also plays an important role in determination of the predicted future prices of the stock.

CRISPR Therapeutics AG (CRSP) Stock Past Performance

CRISPR Therapeutics AG (CRSP) stock revealed -12.30% return for the recent month and disclosed -14.09% return in 3-month period. The stock grabbed 15.63% return over last 6-months and -11.74% return in yearly time period. To measure stock performance since start of the year, it resulted a change of 43.47%. Past performance shows you the funds track record, but do remember that past performance is not an indication of future performance. Read the historical performance of the stock critically and make sure to take into account both long- and short-term performance. Past performance is just one piece of the puzzle when evaluating investments. Understanding how performance fits in with your overall investing strategy and what else should be considered can keep you from developing tunnel vision.

Volatility in Focus:

The stock unfolded volatility at 5.66% during a week and it has been swapped around 4.43% over a month. Volatility is a rate at which the price of a security increases or decreases for a given set of returns. Volatility is measured by calculating the standard deviation of the annualized returns over a given period of time. It shows the range to which the price of a security may increase or decrease. Volatility measures the risk of a security. It is used in option pricing formula to gauge the fluctuations in the returns of the underlying assets. Volatility indicates the pricing behavior of the security and helps estimate the fluctuations that may happen in a short period of time. If the prices of a security fluctuate rapidly in a short time span, it is termed to have high volatility. If the prices of a security fluctuate slowly in a longer time span, it is termed to have low volatility.

The average true range is a volatility indicator. This stocks Average True Range (ATR) is currently standing at 2.04.

Overbought and Oversold levels

The stock has RSI reading of 29.88. RSI gives an indication of the impending reversals or reaction in price of a security. RSI moves in the range of 0 and 100. So an RSI of 0 means that the stock price has fallen in all of the 14 trading days. Similarly, an RSI of 100 means that the stock price has risen in all of the 14 trading days. In technical analysis, an RSI of above 70 is considered an overbought area while an RSI of less than 30 is considered as an oversold area. RSI can be used as a leading indicator as it normally tops and bottoms ahead of the market, thereby indicating an imminent correction in the price of a security. It is pertinent to note that the levels of 70 and 30 needs to be adjusted according to the inherent volatility of the security in question.

Analyst Watch: Analysts have assigned their consensus opinion on this stock with rating of 2.3 on scale of 1 to 5. 1 or 2 =>Buy view 4 or 5 => Sell opinion. 3 =>Hold. Analysts recommendations are the fountainhead of equity research reports and should be used in tangent with proprietary research and investment methodologies in order to make investment decisions.

Christopher Jones

Category Finance

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Email: [emailprotected]

Zip code: 97534

Contact # 541-479-1890

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Stock In Active Zone:: CRISPR Therapeutics AG (CRSP) - WEB NEWS OBSERVER