Archive for the ‘Crispr’ Category

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

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.

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

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.

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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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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.

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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."

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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

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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.

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

Franoise BaylisHarvard University Press2019297 pp.Purchase this item now

With Altered Inheritance, bioethicist Franoise Baylis has authored a vivid call to action that aims to bridge the divides between theory, science, politics, and practice in response to increased public awareness and scientific applications of CRISPR/Cas9 technology. She achieves her aim in this timely and important book.

Baylis calls for broad societal consensus and shared responsibility to guide heritable gene editing toward the common good, which she defines as that which is essential for survival and well-being, to which the market, property, and liberty are subordinate. Her call demands an urgent response, as she notes that humanity sits precipitously on the verge of either a new beginning or the beginning of the end.

A prologue and epilogue bookend the 10 chapters that comprise Altered Inheritance. The back matter includes an index to make the material accessible for scientifically curious general readers, scientists interested in the ethical dimensions of gene editing, and bioethicists seeking an innovative framework to direct theorizing and practice in the era of CRISPR.

Baylis seamlessly threads definitional and conceptual content into the first three chapters, which include critical information for readers who do not have a strong background in the terms and technology of gene editing. The first chapter analyzes Huntingtons disease as a case study for targeting a single gene. Chapter two distinguishes somatic from germline editingthe former being an edit that affects an individual, the latter being one that will be passed on to the individuals descendants. Heritable gene editing is the main focus of the book.

In chapter three, Baylis constructs a brief history of gene editing, using the case of designer babies as a central example. (Incidentally, the term designer baby was first coined to capture marketing-influenced, brand-heavy consumer behavior and only later came to be applied to gene editing.) She concludes this chapter with an expert philosophical discussion that shows that the demarcation between health-related editing and nonhealth-related editing is not as clear as our intuitions may lead us to believe. Cognitive enhancement through traditional means, including reading and education, may only be a difference of degree, not of kind, from gene editing that selects for particular cognitive traits.

Baylis continues this important discussion into chapter four, in which she argues that all medical treatments are a form of enhancementimproving or returning an aberrant gene to its normal status. However, not all enhancements, she argues, are treatments. If one accepts this premise, the pertinent ethical question is not whether treatments should be allowed and nonmedical enhancements prohibited but whether heritable gene editing of any stripe may be structured to promote equality, access, and fairness.

In chapter six, Baylis continues her discussion of the possible harms and benefits of germline editing through the lens of potential moral wrongs. She worries, for example, about the opportunity costs that could occur when science and technology funding is diverted toward gene editing research, the benefits of which may then only be accessible to those with economic means. Baylis encourages the public and scientists alike to ask first, how gene editing science will improve the human condition, and second, what kind of world they want to live in.

Baylis introduces the idea of impact ethics in chapter nine. Following a heuristic articulated in feminist ethics, in which one sets aside a traditional notion of individual autonomy, impact ethics advances a relational understanding of autonomy, viewing people as interdependent. Here, Baylis also resists the notion that experts have privileged access to the truth.

Decisions about the use of genetic technology are too important to be left to scientists, writes Baylis in chapter 10. This provocative claim is not intended as a negative account of scientists involvement in the gene editing debate nor as a prohibition on their ongoing work. Rather, it is meant as a call for bidirectional engagement between scientists and the public. Public empowerment in these integral policy discussions is something all of us may and should participate in.

Commitments to justice, responsibility, accountability, and consensus-building are features of a socially just science and bioethics. Toward this end, Altered Inheritance is a foundational tool in the path ahead.

The reviewer is a philosopher of science and freelance writer based in Indianapolis, IN, USA.

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A new book offers an introduction to the ethical dimensions of germline gene editing - Science Magazine

The race to build the cutting edge banana is on. The Colombian government affirmed a month ago that a banana-killing organism has attacked the Americas the wellspring of a great part of the worlds banana supply. The intrusion has given new earnestness to endeavors to make organic product that can withstand the scourge.

Researchers are utilizing a blend of ways to deal with spare the banana. A group in Australia has embedded a quality from wild bananas into the top business assortment known as the Cavendish and are right now testing these adjusted bananas in field preliminaries. Scientists are additionally going to the amazing, exact quality altering device CRISPR to support the Cavendishs flexibility against the growth, known as Fusarium wither tropical race 4 (TR4).

Reproducing TR4 opposition into the Cavendish utilizing ordinary techniques is absurd on the grounds that the assortment is sterile and proliferated by cloning. So the best way to spare the Cavendish might be to change its genome, says Randy Ploetz, a plant pathologist at the University of Florida in Homestead. The assortment represents 99% of worldwide banana shipments.

James Dale, a biotechnologist at Queensland University of Technology in Brisbane, Australia, began getting enquiries about his hereditarily altered (GM) bananas in July, as the primary gossipy tidbits surfaced that TR4 had arrived at Colombia. At that point Colombia pronounced a national crisis, Dale says, and now the measure of intrigue is through the rooftop.

This isnt the first occasion when that a business banana assortment has confronted eradication. In the main portion of the 1900s, another strain of the Fusarium parasite got TR1 almost cleared out the times head honcho, the Gros Michel. Be that as it may, ranchers had reinforcement in the Cavendish, which was impervious to TR1, intense enough to withstand taking care of during export and had an extensively adequate surface and taste. By the 1960s, enormous banana cultivators, for example, Chiquita, presently situated in Fort Lauderdale, Florida, were changing to the Cavendish.

Theres no simple elective this time. Rodomiro Ortiz, a plant geneticist at the Swedish University of Agricultural Sciences in Alnarp, says that no normally happening banana species has the characteristics that have made the Cavendish so well-known and the capacity to oppose TR4.

The U.S. Patent and Trademark Office (USPTO) has granted another patent to the University of California (UC), University of Vienna, and Dr. Emmanuelle Charpentier covering techniques for delivering a hereditarily altered cell through the presentation of the Cas9 protein, or

As the world restlessly screens the episode of Ebola in Democratic Republic of the Congo, wellbeing authorities note that a measles flare-up announced a month ago in the nation has killed more individualsfor the most part kidsand quicker. Since January

Among the numerous worldwide issues today, the battle against antimicrobial opposition (AMR) urgently needs a comparable leap forward responsibility. For promoters, AMR's appearance on the G20's plan a year ago, at the gathering's summit in Hangzhou, China, spoke to a

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CRISPR is the only hope against deadly funguses - BPhrm Dv

What if CRISPR could be used not just to edit genes, but also to alter the epigenomethe vast network of chemicals and proteins that orchestrates the actions of genes? A study from biomedical engineers at Duke University describes a new CRISPR technology that could allow scientists to do just that.

The most commonly used gene editing technology, CRISPR-Cas9, uses just one Cas protein to cut DNA. In the CRISPR field, this is known as a class 2 system. Class 1 systems, by contrast, are more complicated because they rely on multiple proteins to bind to DNA and then recruit a Cas3 protein to cut it. That network of proteins is called Cascade (CRISPR-associated complex for antiviral defense).

The Duke team used a class 1 CRISPR system to edit the epigenome in cells. In a study published in Nature Biotechnology, they reported that they were able to attach gene activators to the Cascade complex and regulate levels of gene expression in cells. They also connected a repressor to Cascade to turn genes off altogether.

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"We have found Cascade's structure to be remarkably modular, allowing for a variety of sites to attach activators or repressors, which are great tools for altering gene expression in human cells," said Adrian Oliver, Ph.D., a postdoctoral fellow and lead author of the study, in a statement.

RELATED: A better CRISPR? RNA 'hairpins' could improve gene editing

The potential of CRISPR-Cas3 is already generating some excitement in the biopharma world. In January, Locus Biosciences inked a deal with Johnson & Johnson to develop its platform for using CRISPR-Cas3 to solve the problem of antibiotics resistance. The deal could be worth up to $818 million for Locus, a 2019 Fierce 15 company.

Meanwhile, several academic groups are investigating various ideas for improving CRISPR, including another Duke team thats focused on improving the Cas9 enzyme. In April, it described a technique for adding a short tail to the guide RNA thats used in CRISPR systems to improve the accuracy of gene cuts.

Researchers at Columbia University are hoping to sidestep DNA cutting altogether. Theyre using a transposon, or jumping gene, in a system designed to insert DNA in exact locations in the genome without cutting.

The Duke researchers working onclass 1 CRISPR technology are planning further studies to determine whether it might help solve some of the shortcomings of CRISPR-Cas9 in addressing human disease, including the risk of dangerous immune responses. They are also investigating whether the tool could be used to perform many different genome engineering tasks simultaneously.

"We know CRISPR could have a big impact on human health," said Charles Gersbach, Ph.D., Duke professor of biomedical engineering. "But we're still at the very beginning of understanding how CRISPR is going to be used, what it can do, and what systems are available to us.

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New CRISPR approach could improve gene and cell therapies - FierceBiotech

HIV treatment has come a long way over the years, due in large part to antiretroviral drugs that stop the HIV virus from replicating in the body. This gives the immune system a chance to repair itself and stop further damage. Thanks to these amazing advances, HIV is no longer the death sentence that it was in previous decades.

However, antiretrovirals only keep HIV at bay for as long as theyre taken. Defaulting on the drugs means that the HIV virus comes back. Even worse, it can cause patients to build up resistance to the antiretrovirals so that they do not work so effectively in the future.

In other words, theres still room for improvement when it comes to treatment. Fortunately, researchers from the University of California San Diego School of Medicine are poised to provide help, courtesy of a new genetic-sequencing approach that could possibly provide a kill switch to clear out dormant HIV reservoirs inside cells.

The most exciting part of this discovery has not been seen before, Tariq Rana, professor of pediatrics and genetics at UC San Diego School of Medicine, said in a statement. By genetically modifying a long non-coding RNA, we prevent HIV recurrence in T cells and microglia upon cessation of antiretroviral treatment, suggesting that we have a potential therapeutic target to eradicate HIV and AIDS.

The work is based on the discovery of a recently emerged gene that appears to regulate HIV replication in immune cells, including macrophages, microglia, and T cells. The team refers to this as HIV-1 Enchanced LncRNA (HEAL), and it is elevated in people with HIV. By using CRISPR-Cas9 gene editing, their work suggests that it could stop HIV from recurring in the event that antiretroviral treatment is stopped.

This has the potential for [being a] cure but, [well] have to wait for animal studies, Rana told Digital Trends. As for the next steps, Rana said that future studies will determine if turning this regulator HEAL off can remove viral reservoirs, which are the key source for viral rebound when therapies are discontinued.

A paper describing the work was recently published in the journal mBio.

See the article here:
CRISPR-Cas9 gene editing could one day turn off HIV virus in the body - Digital Trends

[Tweaking an embryos DNA can] help save someone who is already alive.

Take the case of Jessica and Keith, a couple in the Bay Area with a 2 1/2-year-old daughter with Fanconi anemia, a genetic disease that leads to the failure of bone marrow to produce red and white blood cells and carries an increased risk of a number of cancers. The best treatment is a stem cell transplant from a sibling, and Jessica and Keith, who asked that their last name not be used, are now in the process of trying to have another child through IVF who can serve as a donor whats known as a savior sibling.

But making an embryo thats both healthy and a suitable donor match for the older sibling is an exercise in long odds. Its theoretically possible that altering an embryos DNA with the genome-editor CRISPR could improve the process.

He and Jessica understand its too soon to use CRISPR in such cases. The technology is not advanced or precise enough yet and might never be.

But its another example of the ways in which genome-editing could help patients where other reproductive technologies cannot.

Read full, original post: Could editing the DNA of embryos with CRISPR help save people who are already alive?

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CRISPR could save the lives of sick children by tweaking the embryos of their siblings - Genetic Literacy Project

BODY LANGUAGE

Cutting-edge medical advancements are changing the way we think about having children. From gene therapy and pre-implantation genetic testing to women carrying children in transplanted uteruses, these medical advancements each come with an array of issues.

Perhaps the most prominent advancement featuring in the debate is that of gene-editing using CRISPR-Cas9 technology.Gene editing has already been used to create tomatoes that can sit in the pantry and ripen slowly for months without rotting.

It has been used to modify mosquitos so that they are unable to transmit malaria, and create ultra-muscular dogs to be used by law enforcement authorities. Gene editing has even allowed us to create cows without horns.

The most controversial application for gene editing which has been considered, is the genetic modification of the human species. Jennifer Doudna, the American biochemist who is credited for developing the CRISPR-Cas9 system, has been quoted as saying: The power to control our species genetic future is awesome and terrifying. Deciding how to handle it may be the biggest challenge we have ever faced.

Beverley Townsend and Donrich Thaldar(Gene editing is risky, but worth it, Mail & Guardian, September 13), suggest that the blanket moratorium called for by the international community blocks scientific progress and, with it, the opportunity to act responsibly.

They offer two specific examples of gene editing of human embryos: that of Chinese scientist He Jiankuis covert gene editing of twin girls in 2018, and the planned gene editing of embryos by Russian molecular biologist Denis Rebrikov.

Townsend and Thaldar provide an explanation of these two examples, but after brief mention of the gene-editing of the twin girls, who were subsequently born alive and are reportedly healthy, they shift their focus to Rebrikovs plans to remove the genetic sequence for inheritable deafness from embryos created using the gametes of a deaf couple.

Rebrikov is not the best example of the considerations in the debate, because the proposed gene editing itself is problematic. The Chinese scientist planned to use gene editing to make the children born resistant to HIV infection. This was clearly a therapeutic aim. But are Rebrikovs plans to remove inheritable deafness from embryos therapeutic? It is arguable whether deafness is a disease or a disability. In fact, a whole section of society does not believe that deafness is a disability, and that such proposed treatment amounts to discrimination against the deaf population.

The implications of what He has done is crucial to the discussion. Rebrikovs expression of his intention provides us with the opportunity to identify and debate issues regarding gene editing, and the control which we are prepared to allow ourselves to exercise over our genetic heritage.

But, the Chinese incident clearly demonstrates exactly why a moratorium on the gene editing of human embryos must be the first words in the conversation.

He Jiankui used CRISPR-Cas9 to delete part the CCR5 gene that allows HIV to enter cells. The world had a mixed reaction to the sudden announcement of the birth of the children, and some hailed it as the medical miracle that signalled the end of HIV. But, there were many who did not reciprocate this sentiment.

For many doctors and scientists, He had acted in haste without any consideration of what was safe or ethical. At the outset, the ethical implications were clear: he had fated these children to be prized cattle in a society already ravaged with the HIV epidemic.

The news that broke in June this year was more devastating, and proves that we must adopt a cautious, conservative approach to gene editing.

According to a study published in Nature Medicine, He may have inadvertently shortened the twins life expectancy. The study showed that people with two disabled copies of the CCR5 gene are 21% more likely to die before the age of 76 than are people with at least one copy of the gene. The reason for the discrepancy is not yet known.

There are useful measures that may help us to determine whether particular uses of gene editing are justified. We can ask whether the proposed gene editing amounts to enhancement of our biological systems, because therapeutic interventions are always ethically easier to justify over enhancements which offer no therapeutic benefit apart from expressing what we want to see in our future children. Consideration of what is in the best interests of the future child is also important.

The best interests of the child principle is firmly entrenched in law and asks us to consider what action would serve a childs interests best. If the proposed gene editing would place a child at a disadvantage, it would not be appropriate to proceed.

In 2008, a deaf British couple requested permission to genetically test their embryos to ensure that the child was deaf. The couple stated that being deaf is not about being disabled, or medically incomplete its about being part of a linguistic minority. Were proud, not of the medical aspect of deafness, but of the language we use and the community we live in.

A public outcry followed because many people viewed the intention to ensure that a child was hearing-impaired was against the best interests of the child.

The Human Fertilisation and Embryology Authority, which oversees the fertility industry in the United Kingdom, refused to permit the selection.

We should also consider whether the proposed gene editing may cause harm to the child which may be born. There is also the bioethical norm of beneficence, which asks us to consider whether we are doing good by our actions.

Townsend and Thaldar correctly state that we are not aware of the risks of gene editing on human embryos, but they conclude that this cannot be a reason to prohibit the treatment.

They correctly identify that there are risks with any therapeutic procedure. But we subject ourselves to therapeutic procedures after at least some of the inherent risks have been identified and measured. We would not do so if there were no studies regarding the efficacy and safety of such procedures.

This is not a risk we would take for ourselves, let alone the earliest and most vulnerable forms of human life.

A fundamental question is: Who gets to decide these issues? The public and legal and scientific communities need to be involved, and this cannot happen overnight.

Moratoria provide us with the opportunity to pause, debate and conduct critical research, which will allow us to safely navigate the course towards clinical treatment. This is responsible scientific progress.

There may be grave consequences of gene editing humans that we do not yet know. When we exercise control over genes, we are determining what people of the future will be like. This cannot be taken lightly, and we cannot allow cowboy scientists to proceed with gene editing of human embryos until the risks are better understood.

The published study comes too late for the two little girls. Perhaps they will live long, healthy lives. But, there is now evidence against that possibility. Gene editing is definitely worth it, but we must proceed with caution.

Sheetal Soni is an academic at the School of Law at the University of Kwa-Zulu-Natal

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Yes, edit our genes but do it cautiously - Mail and Guardian

Integration of CRISPR-case9 technology to accelerate the discovery of innovative antibiotics

DEINOVE (Euronext Growth Paris: ALDEI), a French biotechnology company that relies on a radical innovation approach to develop innovative antibiotics and bio-sourced active ingredients for cosmetics and nutrition, announces that it has expanded its technological platform with an advanced genetic tool, the CRISPR-cas9 system, to enhance its ability to optimize various microorganisms.

In the last few years, DEINOVE has set up a high throughput genetic engineering platform specifically dedicated to rare microorganisms and thus demonstrated its ability to adapt genetic tools to poorly described organisms. Thus, the exploitation of Deinococci as microbial plants has allowed the large-scale production of pure high value-added compounds such as carotenoids. It should be recalled that Deinococci are extremophilic microorganisms whose biological and molecular specificities have so far been little studied and therefore unexploited.

After developing a platform dedicated to the identification of novel antibiotic structures produced by rare bacteria (AGIR Program), DEINOVE strengthens its expertise in genetic engineering with the integration of a cutting-edge tool, the CRISPR-cas9 technology, known as molecular scissors, which has revolutionized genetic engineering in recent years.

The objective for DEINOVE is to be able to directly manipulate the strains producing antimicrobial activities or to transfer these activities into phylogenetically close frames. This has been successfully achieved by the Company which has made the Streptomyces chassis an effective producer of a pharmaceutical intermediate initially produced by Microbacterium arobescens (proof of concept DNB101/102).

Genome editing occurs at two levels. First, highlights the cluster of genes at the origin of the antibiotic activity of interest. To optimize the spectrum of activity and eliminate any potential cytotoxicity, the structure of a natural molecule can then be modified by directly, finely and precisely editing the genes responsible for this activity.

This technology opens up many opportunities in the identification and optimized production of new antibiotic structures.

"Our expertise in the genetic engineering of a variety of microorganisms, unusual for some, is unique, and the integration of CRISPR-cas9 extends the possibilities of our platform," says Georges GAUDRIAULT, Scientific Director of DEINOVE. "We continue to structure the various technological bricks of the AGIR platform to be able to drastically accelerate the identification and optimization of new antibiotic structures. This technology is an additional asset in our race against the clock in the face of rising antimicrobial resistance."

ABOUT DEINOVE

DEINOVE is a French biotechnology company, a leader in disruptive innovation, which aims to help meet the challenges of antibiotic resistance and the transition to a sustainable production model for the cosmetics and nutrition industries.

DEINOVE has developed a unique and comprehensive expertise in the field of rare bacteria that it can decipher, culture, and optimize to disclose unsuspected possibilities and induce them to produce biobased molecules with activities of interest on an industrial scale. To do so, DEINOVE has been building and documenting since its creation an unparalleled biodiversity bank that it exploits thanks to a unique technological platform in Europe.

DEINOVE is organized around two areas of expertise:

Within the Euromedecine science park located in Montpellier, DEINOVE employs 60 employees, mainly researchers, engineers, and technicians, and has filed more than 350 patent applications internationally. The Company has been listed on EURONEXT GROWTH since April 2010.

CONTACTS

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Integration of CRISPR-case9 technology to accelerate the discovery of innovative antibiotics - GlobeNewswire

We are delighted to be recognized by DTRA for the advanced state of our technology and development efforts. This support will further our mission to harness the power of CRISPR and synthetic biology to develop diagnostic tools that enable effective decision making in any environment at any time, said Rahul K. Dhanda, Sherlocks co-founder, president and CEO. This funding will enable our team to continue to make important progress advancing our platform, while also addressing an urgent need to rapidly identify pathogenic agents and other biothreats.

The multiyear grant will accelerate the companys development of infectious disease diagnostic tests for decentralized settings, such as the battlefield. Further, the support will help the company refine its deep learning and bioinformatics tools for rapid definition and deployment of molecular diagnostic tests.

When we published the call for a new class of rapid diagnostics, we were optimistic that we would identify both a technology and an organization that could deliver a significant leap forward in testing for biothreat and battlefield settings through initiatives driven by the Defense Innovation Unit, said Charles Hong, a science and technology manager for the Detection and Diagnostics Division at DTRA. We are pleased to support Sherlock in its efforts to create novel CRISPR- and synthetic biology-based diagnostic tests that will be sensitive, fast and easily deployable in any setting.

The Defense Innovation Unit identifies commercial industry solutions to support the U.S. military, and through this process, the organization facilitated finding a new class of rapid diagnostic solutions on behalf of DTRA.

This funding provides important validation for the versatility of Sherlocks platform, and our ability to develop simple, impactful diagnostic tools that can be applied in field-based settings, said William Blake, Ph.D., Sherlocks chief technology officer. We are grateful to DTRA for this award and look forward to working with the agency in support of their mission to enable rapid responses to potential threats and crises.

About Sherlock Biosciences

Sherlock Biosciences is dedicated to making molecular diagnostics better, faster and more affordable through Engineering Biology platforms. The company is developing applications of SHERLOCK, a CRISPR-based method to detect and quantify specific genetic sequences, and INSPECTR, a Synthetic Biology-based molecular diagnostics platform that is instrument free. SHERLOCK and INSPECTR can be used in virtually any setting without complex instrumentation, opening up a wide range of potential applications in areas including precision oncology, infection identification, food safety, at-home tests, and disease detection in the field. For more information visit Sherlock.bio.

About DTRA

The Defense Threat Reduction Agency (DTRA), an agency within the United States Department of Defense (DoD), is the official Combat Support Agency for countering weapons of mass destruction (chemical, biological, radiological, nuclear, and high explosives). The Defense Threat Reduction Agency enables DoD, the U.S. government, and international partners to counter and deter weapons of mass destruction and improvised threat networks. Under the auspice of the Chemical and Biological Defense Program, DTRA has the responsibility to manage and integrate the DoD chemical and biological defense science and technology programs. DTRAs continued effort to enhance the combat support mission also advances public health services by developing innovative technologies that protect against biological threats. For more information, visit http://www.dtra.mil.

About Defense Innovation Unit

DIU strengthens our national security by increasing the adoption of commercial technology throughout the military and growing the national security innovation base. Learn more about DIU at http://www.diu.mil.

Continued here:
Sherlock Biosciences Awarded Contract from US Defense Threat Reduction Agency to Support the Development of Ultra-Fast, Ultra-Sensitive Diagnostics -...

CAMBRIDGE, Mass., Sept. 26, 2019 (GLOBE NEWSWIRE) Intellia Therapeutics, Inc. (NASDAQ: NTLA) announced today that an arbitration panel issued an Interim Award confirming that certain structural and chemical guide RNA modification technologies were exclusively licensed to Intellia by Caribou Biosciences under the parties July 2014 agreement. This Interim Award is subject to additional negotiations between the parties and potentially further arbitration proceedings before it becomes final.

After concluding that the chemical modification technology was within the scope of Intellias exclusive license from Caribou, the arbitration panel noted that its decision could delay or otherwise adversely impact the continued development of these modified guide RNAs as human therapeutics. It also noted that Intellia currently is not using these modified guide RNAs in any of its active programs. For this reason, the panel stated it will declare that Caribou has leaseback rights, which it described as exclusive, perpetual and worldwide, to the chemically modified guide RNAs. This leaseback only applies to the chemically modified guides and will be subject to terms, including Caribous future payments to Intellia, to be negotiated by the parties or, if unsuccessful, to additional arbitration proceedings.

The leaseback will not include the structural guide modifications intellectual property at issue in the arbitration, any other intellectual property exclusively licensed or sublicensed by Caribou to Intellia under the Caribou license (including but not limited to the foundational CRISPR/Cas9 intellectual property co-owned by University of California, University of Vienna and Dr. Emmanuelle Charpentier), or any other Intellia intellectual property.

Upon, and subject to the terms of, a final award, which will follow negotiations between the parties and potential further legal proceedings, Caribou would be able to use the modified guide RNAs at issue for human therapeutics. Intellia or Caribou may challenge the arbitration panels decisions under limited circumstances.

The Interim Award has no impact on any of Intellias current programs. Additionally, the Interim Award has no effect on any other Intellia rights or Caribou obligations under their agreement.

Background on Intellias License from Caribou BiosciencesIn July 2014, Intellia Therapeutics, Inc. licensed from Caribou Biosciences, Inc. certain intellectual property. On October 17, 2018, the Company initiated an arbitration proceeding with the Judicial Arbitration and Mediation Services (JAMS) against Caribou asserting that Caribouis violating the terms and conditions of theCaribou license, as well as other contractual and legal rights, by using and seeking to license to third parties technology covered by two patent families (described in, for instance, PCT No. PCT/US2016/015145 and PCT No. PCT/US2016/064860, and related patents and applications) relating to specific structural or chemical modifications of guide RNAs.

AboutIntellia TherapeuticsIntellia 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 StatementsThis 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 ability to advance and expand its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain, protect and expand its related intellectual property portfolio; its plans to negotiate, and ability to agree to, a leaseback with Caribou, including the scope of such arrangement and the timing and amount of payment under any such arrangement; the potential to initiate additional arbitration or legal proceedings if negotiations are not successful; the potential implications and impact the Interim Award may have on Intellias current programs or on any other intellectual property rights and changes in any of the foregoing in connection with the issuance of a final award; its ability to develop otherin vivoorex vivocell therapeutics of all types; and the impact of any of the foregoing on its collaborations and licensing arrangements.

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 as a result of the Interim Award or the finalization of any award; risks related to Intellias relationship with third parties, including our licensors; or risks related to our ability, or the ability of our licensors, to protect and maintain their intellectual property position. 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:Investors:Glenn GoddardExecutive Vice President, Chief Financial Officer+1 857-706-1056glenn.goddard@intelliatx.com

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

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Arbitration Decision Affirms Intellia Therapeutics Interpretation of Licensing Agreement with Caribou Biosciences on the CRISPR/Cas9 Technology -...

A Recent Research By MarketResearch.Biz On CRISPR and Cas Genes Market Provides A Professional, In-depth, Comprehensive, and Well-designed Insights that can help you to make better business decisions

The globalCRISPR and Cas Genes Marketis studied and analyzed with the help of a complete primary and secondary market research. The report provides broad industry data on the CRISPR and Cas Genes market, such as size, forecasted growth, profitability, key players, market share and market trends will give you a high-level overview of CRISPR and Cas Genes market opportunities. This report studies the global market size of CRISPR and Cas Genes, especially focuses on the key regions like North America, Europe and Asia-Pacific, South America, Middle East, and Africa

The market size section gives the CRISPR and Cas Genes market revenues, covering historic growth of the market as well as forecasting the future. Moreover, the report covers a host of company profiles, who are making a mark in the industry or have the potential to do so. The profiling of the players includes their market size, key product launches, information regarding the strategies they employ, and others.

The Leading key players covered in this study:Addgene Inc, AstraZeneca Plc., Bio-Rad Laboratories Inc, Caribou Biosciences Inc, Cellectis S.A., Cibus Global Ltd, CRISPR Therapeutics AG, Editas Medicine Inc, eGenesis Bio, GE Healthcare, GenScript Corporation

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Vector-based CasDNA-free CasSegmentation on the basis of application:

Genome EngineeringDisease ModelsFunctional GenomicsKnockdown/ActivationSegmentation on the basis of end user:

Biotechnology & Pharmaceutical CompaniesAcademic & Government Research InstitutesContract Research Organizations

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Global CRISPR and Cas Genes Market 2019 | Complete Research Study on the Current State of the Global Market with a Focus on the Regional Market - Biz...

Biomedical engineers at Duke University have used a previously unexplored CRISPR technology to accurately regulate and edit genomes in human cells.

With this new approach, the researchers hope to dramatically expand the CRISPR-based tools available to biomedical engineers, opening up a new and diverse frontier of genome engineering technologies.

In a study appearing on Sept. 23 in Nature Biotechnology, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke, and Adrian Oliver, a post-doctoral fellow in the Gersbach lab who led the project, describe how they successfully harnessed Class 1 CRISPR systems to turn target genes on and off and edit the epigenome in human cells for the first time.

CRISPR-Cas is a defense system in which bacteria use RNA molecules and CRISPR-associated (Cas) proteins to target and destroy the DNA of invading viruses. The discovery of this phenomenon and the repurposing of the molecular machinery set off a genome-editing revolution as researchers learned how to wield the tool to specifically target and edit DNA in human cells.

CRISPR-Cas9, the most commonly used genome editing tool today, is categorized as a Class 2 CRISPR system. Class 2 systems are less common in the bacterial world, but they are theoretically simpler to work with, as they rely on only one Cas protein to target and cleave DNA.

Class 1 systems are not so simple, relying on multiple proteins working together in a complex called Cascade (CRISPR-associated complex for antiviral defense) to target DNA. After binding, Cascade recruits a Cas3 protein that cuts the DNA.

"If you were to look at the individual CRISPR systems of all the bacteria in the world, nearly 90 percent are Class 1 systems," said Gersbach. "CRISPR-Cas biology is an incredible source for biotechnology tools, but until recently everyone has only been looking at a small slice of the pie."

To demonstrate the capabilities of the Class 1 system, Oliver attached gene activators to specific sites along a type I E. coli Cascade complex and targeted the system to bind gene promoters, which regulate gene expression levels. Because she did not include the Cas3 protein in the experiment, there was no cutting of the DNA and no change to underlying DNA sequence. The experiment showed that the Cascade activator not only binds to the correct site and can turn up the levels of the target gene, but does so with accuracy and specificity comparable to CRISPR/Cas9.

Oliver repeated the process using type I Cascade complexes from an additional bacterial strain that was particularly robust in working at a variety of target sites. She also showed that the activator domain could be swapped for a repressor to turn target genes off. Again, the researchers noted accuracy and specificity comparable to CRISPR/Cas9 methods.

"We have found Cascade's structure to be remarkably modular, allowing for a variety of sites to attach activators or repressors, which are great tools for altering gene expression in human cells," Oliver said. "The flexible nature of Cascade makes it a promising genome engineering technology."

Gersbach and Oliver were encouraged to investigate the more complicated Class 1 CRISPR systems by their collaborators at nearby North Carolina State University, Professors Rodolphe Barrangou and Chase Beisel, who is now at the Helmholtz Centre for Infection Research in Germany. Barrangou is a microbiologist who has studied the natural biology of diverse CRISPR defense mechanisms for nearly two decades, and Beisel is a chemical engineer who has worked with Barrangou on engineering microorganisms with Class 1 CRISPR systems. They were both curious whether Gersbach's lab could use these systems in human cells similar to their work with Cas9.

"This work and the resulting technologies are a fantastic example of how collaboration across disciplines and across universities in the North Carolina Research Triangle can be highly innovative and productive" says Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University.

Now, the team is optimistic that their study, and the related work of others in the field, will incentivize new research into Class 1 CRISPR systems.

"The purpose of this project was to explore the diversity of CRISPR systems," said Gersbach. "There have been thousands of papers about CRISPR-Cas9 in the last decade, and yet we're constantly learning new things about it. With this study we're applying that mindset to the other 90% of what's out there."

So far, the team has shown that these Class 1 systems are comparable to to CRISPR-Cas9 in terms of accuracy and application. As they consider future directions, they are curious to explore how these systems differ from their Class 2 counterparts, and how these differences could prove useful for biotechnology applications.

The team is also interested in studying how Class 1 systems could address general challenges for CRISPR-Cas research, especially issues that complicate potential therapeutic applications, like immune responses to Cas proteins and concurrently using multiple types of CRISPR for different genome engineering functions.

"We know CRISPR could have a big impact on human health," said Gersbach. "But we're still at the very beginning of understanding how CRISPR is going to be used, what it can do, and what systems are available to us. We expect that this new tool will enable new areas of genome engineering."

Reference: Pickar-Oliver. 2019.Targeted transcriptional modulation with type I CRISPRCas systems in human cells. Nature Biotechnology. DOI: https://doi.org/10.1038/s41587-019-0235-7.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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CRISPR Tool Opens Up a New Frontier of Genome Engineering Technologies - Technology Networks

The U.S. Patent and Trademark Office (USPTO) today granted the University of California (UC) and its partners, the University of Vienna and Emmanuelle Charpentier, a new CRISPR-Cas9 patent, bringing the teams continually expanding patent portfolio to 15.

Jennifer Doudna, co-inventor of the CRISPR-Cas9 gene-editing tool, in 2014, with a model of the complex on her computer screen. (UC Berkeley photo courtesy of Cailey Cotner)

U.S. Patent 10,421,980 covers compositions of certain DNA-targeting RNAs that contain RNA duplexes of defined lengths that hybridize with Cas9 and target a desired DNA sequence. The patent also covers methods of targeting and binding a target DNA, modifying a target DNA, or modulating transcription from a target DNA wherein the method comprises contacting a target DNA with a complex that includes a Cas9 protein and a DNA-targeting RNA.

In the coming months, based on applications allowed by the USPTO, UCs CRISPR-Cas9 patent portfolio will increase to 18. Together, these patents cover compositions and methods for CRISPR-Cas9 gene-editing, including targeting and editing genes and modulating transcription in any setting, such as within plant, animal and human cells.

With every patent that issues, UC strengthens its position as the leader in CRISPR-Cas9 intellectual property in the United States, said Eldora Ellison, the lead patent strategist on CRISPR-Cas9 matters for UC and a director at Sterne, Kessler, Goldstein & Fox. We are steadfast in our commitment to developing a comprehensive patent portfolio that protects the groundbreaking work of the Doudna-Charpentier team on CRISPR-Cas9.

The team that invented the CRISPR-Cas9 DNA-targeting technology included Doudna and Martin Jinek at UC Berkeley; Charpentier, then at Umea University in Sweden and now director of the Max Planck Institute for Infection Biology in Germany; and Krzysztof Chylinski of the University of Vienna. The methods covered by todays patent, as well as the other methods claimed in UCs 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.

Doudna is a UC Berkeley professor of molecular and cell biology and of chemistry, a Howard Hughes Medical Institute investigator and holder of the Li Ka Shing Chancellors Chair in Biomedical and Health Sciences. She also is executive director of the Innovative Genomics Institute, a faculty scientist at Lawrence Berkeley National Laboratory and a senior investigator at the Gladstone Institutes in San Francisco.

Additional CRISPR-Cas9 patents in this teams 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; and 10,415,061. These patents are not a part of the PTABs recently declared interference between 14 UC patent applications and multiple previously issued Broad Institute patents and one application, which jeopardizes essentially all of the Broads CRISPR patents involving eukaryotic cells.

International patent offices have also recognized the pioneering innovations of the Doudna-Charpentier team, in addition to the 15 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.

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University of California awarded 15th U.S. CRISPR-Cas9 patent - UC Berkeley

On one level, Malakkar Vohryzek always knew what was wrong with him. For as long as he can remember hes now 43 the sun has been his enemy, making angry-looking moles pop up on his white-as-a-fish-belly skin like toxic mushrooms after a downpour.

At age 9, he bit one off. Since his teens he has had moles removed as regularly as other kids got haircuts, hoping to catch the growths before they became malignant. Because of his skins extreme sensitivity to sunlight, he takes every UV-blocking precaution, from SPF 60 sunscreen and hats and other cover-ups to, as a 19-year-old, working the graveyard shift as a waiter at Dennys so he could commute in darkness.

But there is no name for what Vohryzek has, and no cure. There is no known inherited genetic mutation that might explain why just a few ultraviolet rays make his skin cells proliferate wildly, forming moles. One of these days, Vohryzek is convinced, hell overlook one, or wait too long before seeing a dermatologist, and hell wake up with malignant melanoma. That cancer, if it metastasizes, is usually fatal.

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Vohryzek, a legal researcher and IT consultant in southern California, has therefore taken matters into his own hands. For the last few months, he has blitzed scientists, biotechnology companies, and biohackers as far away as Sendai, Japan, with email pleas to please CRISPR him. I just want to live, Vohryzek told STAT in an interview.

It was perhaps inevitable that the campaign to give patients the right to try experimental drugs before FDA approval enshrined in a 2018 law would combine with unbounded optimism about the potential curative power of genome-editing technology to send someone like Vohryzek on this quest. In California, lawmakers were so concerned about people biohacking themselves or others with unproven therapies that they passed a law this summer banning it.

Id expect to see more people like Vohryzek, said bioethicist Alison Bateman-House of New York University, an expert on avenues for patients to access experimental therapies. Although parents are increasingly asking scientists for access to experimental compounds that have never even been tested in animals, in order to save their children from a devastating disease, there are likely to be even more such pleas for genetic technologies.

Its so intuitively simple: replace or correct a gene thats not functioning properly, Bateman-House said. There is so much hype, more and more people will think, I want that.

In his early 30s, Vohryzek became increasingly frantic about UV-induced DNA damage, which he fears is worsening with age. Hed spent much of his 20s in federal prison for distributing LSD, after which he legally changed his first name to a bastardization of the Arabic (malak al qur) and Hebrew for Angel of Truth and took his mothers last name. After his release, he found that moles were erupting with frightening frequency, especially on his arms.

In 2017, he read that biohacker Josiah Zayner, who sells genetic engineering kits and lessons through his company The Odin, injected himself with a purportedly muscle-boosting CRISPR cocktail onstage at a biotechnology conference. That inspired me, Vohryzek said though Zayners stunt didnt work and he began combing genetics papers for research on radiation protection.

Earlier this year, he happened on a gene that he believes will save him, one from the tiny, rotund, eight-legged water bear aka moss piglet, aka tardigrade which protects it from the damaging effects of radiation. A study from Japan reported that scientists had sequenced the genome of Ramazzottius varieornatus, a species of the famously resilient tardigrade, and identified a previously unknown gene. It turned out to code for a protein they called Dsup (for damage suppressor).

What caught Vohryzeks eye was what happened when Takuma Hashimoto of the University of Tokyo and his colleagues slipped the tardigrade gene into human cells growing in lab dishes and then bombarded them with X-rays. Photon for photon, X-rays are hundreds of times more powerful than the suns ultraviolet rays. Human cells genetically engineered to express tardigrade Dsup withstood 40% more radiation than regular human cells.

Dsup works by minimizing the harm to genes, apparently, by encasing cells DNA, much like a lead shield in a nuclear reactor. As a result, radiation doesnt break the strands of the double helix a breach that can trigger cancer. Dsup, Vohryzek thought, could protect him from solar UV and therefore melanoma.

For the last few months, he has been asking scientists and companies if theyll give him the biological supplies he would need he isnt always clear on what those might be to receive the tardigrade gene, using CRISPR or some other technology to slip it into his cells.

Hashimotos experiment, Vohryzek told STAT, demonstrates that Im not proposing something insane. I want to participate in [the] use of CRISPR on full genome gene insertion.

In July, he emailed his request to Hashimoto, explaining that he will die soon from skin cancer unless he receives the Dsup gene. If you know a team that can [use] CRISPR to insert the Dsup production into my genome, Vohryzek promised, he would sign an agreement not to hold them responsible for any mishaps. If the experiment killed him, he said, he would donate his body to science so researchers could figure out what happened.

In fact, genome editing technologies such as CRISPR only tweak what already exists in a genome. It can alter a DNA letter, or nucleotide, to transform the gene from a disease-causing form to a healthy one. It can snip out regions from the former, disabling them and leaving only the healthy version (people inherit two copies of every gene, one from mom and one from dad). It can insert a few nucleotides in place of a misspelled, disease-causing segment.

But it cannot insert a completely novel gene. Thats called genetic engineering. Although there are now two approved gene therapies in the U.S., for a form of blindness and for spinal muscular atrophy, this intervention is considered less precise and more prone to problems than genome editing.

None of that has dampened Vohryzeks interest. Although his chief motivation is avoiding melanoma I just know that eventually the roll of the genetic dice will come up snake eyes, and I will die, he said he also believes that becoming a human guinea pig would advance science.

CRISPR science has the potential to save billions of lives, and end misery for billions more, he said. I have hundreds of reasons to willingly contribute my own body for furthering its research, and no reason at all not to.

He has received almost no replies to his requests, and the ones hes gotten have hardly been encouraging. While technically feasible there are many ethical and legal implications to attempting this, wrote an executive at the genetics supply company Atum. Im not sure what sort of help we can give you with this project. To be honest, it seems more like a science fiction project than a commercially viable product. We deal mostly with the latter.

It seems unlikely that any academic or biotech scientists will grant Vohryzeks wish. On the other hand, the birth of CRISPR babiesalso seemed unlikely, until scientist He Jiankui produced two of them in China last year. Just as that bombshell sent tremors through legitimate developers of CRISPR therapies, so could a rogue researcher putting a water bear gene into Vohryzek, said NYUs Bateman-House: Im very worried about systemic ramifications, including shutting down gene therapy everywhere.

None of the biohacker collectives contacted by STAT said it had been asked by patients for help with do-it-yourself genome editing, but Bateman-House suspects that it is just a matter of time. Earlier this month, Vohryzek asked a friend who was attending a biohackers gathering in Las Vegas to see whether any of them might be willing to give him the Dsup gene, though he said he would prefer the professionals to a garage DIYer. In November, he plans to attend a meeting of a DIY collective in Seattle to see if my experimental treatment is feasible for them.

If I die of melanoma, it wont help anyone, he said. If I die because of an experimental treatment, it will at least help science.

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California man pleads with scientists to 'CRISPR me' - STAT - STAT

Sleek CRISPR systems get almost all the attention. They rely on single-protein nucleases instead of multiunit effectors, which are, presumably, too unwieldy for gene engineering applications. Yet CRISPR jumbles have been given a tumble by scientists at Duke University. Led by Charles Gersbach, PhD, the Rooney Family associate professor of biomedical engineering and Adrian Oliver, PhD, a postdoctoral fellow, these scientists used a multiunit effector system to turn target genes on and off in human cells.

Specifically, the scientists used a class 1 CRISPR-Cas system called Cascade (CRISPR-associated complex for antiviral defense). And as if it wasnt clunky enough already, the scientists tacked on a couple of extrasactivation and repression domains. The system, however, omitted the Cas enzyme that would have ordinarily been present.

In this case, the Cas enzyme would have been Cas3, a sort of molecular shredder. Leaving it out seemed a good idea, since the scientists were experimenting with gene regulation, something rather more delicate than shredding.

Using modified versions of Cascade systems from Escherichia coli and Listeria monocytogenes, the scientists achieved both DNA targeting and transcriptional control. They presented their findings in a paper (Targeted transcriptional modulation with type I CRISPRCas systems in human cells) that appeared September 23 in Nature Biotechnology.

We validate Cascade expression, complex formation, and nuclear localization in human cells, and demonstrate programmable CRISPR RNA (crRNA)-mediated targeting of specific loci in the human genome, the articles authors wrote. By tethering activation and repression domains to Cascade, we modulate the expression of targeted endogenous genes in human cells.

Class 1 CRISPR systems, which include the type I CRISPR-Cas system in the current study, represent about 90% of all CRISPR systems in nature. Yet these systems remain largely unexplored for genome engineering applications. By demonstrating the potential of repurposed type I CRISPR-Cas systems, the Duke scientists hope to open a new and diverse frontier of genome engineering technology.

We have found Cascades structure to be remarkably modular, allowing for a variety of sites to attach activators or repressors, which are great tools for altering gene expression in human cells, said Oliver, the lead author of the study. The flexible nature of Cascade makes it a promising genome engineering technology.

[Our purpose] was to explore the diversity of CRISPR systems, added Gersbach, the studys senior author. There have been thousands of papers about CRISPR-Cas9 in the last decade, and yet were constantly learning new things about it. With this study, were applying that mindset to the other 90% of whats out there.

So far, the Duke team has shown that Class 1 systems are comparable to CRISPR-Cas9 in terms of accuracy and application. Going forward, the team intends to explore how these systems differ from their Class 2 counterparts, and how these differences could prove useful for biotechnology applications.

The team is also interested in studying how Class 1 systems could address general challenges for CRISPR-Cas research, especially issues that complicate potential therapeutic applications, like immune responses to Cas proteins and concurrently using multiple types of CRISPR for different genome engineering functions.

We know CRISPR could have a big impact on human health, noted Gersbach. But were still at the very beginning of understanding how CRISPR is going to be used, what it can do, and what systems are available to us. We expect that this new tool will enable new areas of genome engineering.

See the article here:
Clunky CRISPR Flaunts Smooth Moves in Gene Regulation - Genetic Engineering & Biotechnology News

In the volatile world of biotech, investors can find a number of promising markets that could see significant growth in the years to come. Gene editing is one of these markets that has tremendous potential for growth, with some research reports showing the sector could reach $9.66 billion by 2025. Considering that in 2018, the gene-editing market was worth only $3.7 billion; this works out to an impressive 14.7% annual growth rate.

While few biotech stocks are developing gene-editing therapies, the complexity of the subject matter can make it difficult for investors to categorize where an individual company stands in relation to its competition.CRISPR Therapeutics (NASDAQ:CRSP)has a head start. Having developed the CRISPR Cas9 gene-editing technology, CRISPR has quickly made a name for itself in the biotech world. Since then, CRISPR Therapeutics has earned a market cap of $2.65 billion despite only having a handful of early clinical trials to show at the moment.

Amid this wave of excitement, shares of CRISPR have risen by 61.8% so far in 2019. While CRISPR will have to clear a few hurdles before its gene-editing drugs can hit the markets, there's a strong case to be made for this stock becoming a leader in the space. Let's take a look at some of the main therapies CRISPR is developing at the moment and how exactly it compares to its competition.

IMAGE SOURCE: GETTY IMAGES.

CRISPR's main strategy with its gene-editing technology has been to target major genetically based disorders. Its flagship drug, an anti-sickle cell medication known as CTX001, is undergoing a phase 1 clinical trial that, if successful, would be highly lucrative for the company. Approximately 100,000 Americans are diagnosed with sickle cell disease currently, with children being especially vulnerable to the condition.

At the moment, there's no cure for the disease, and researchers are currently exploring the possibility of bone marrow transplants as a therapeutic option. A gene-editing drug that could treat sickle cell anemia would be a home run for CRISPR if it were approved for human use.

Earlier this year, CRISPR announced the first sickle-cell patient had been treated with CTX001 in its clinical trial, and the research team is waiting to see if there are any long-term effects of the treatment. Although CRISPR has said that the clinical trial could be concluded in 2022 and a potential rollout could quickly follow, in the world of clinical trials, that's still plenty of time for things to change.

CRISPR also has a number of early-stage clinical trials underway for its cancer immunotherapies. CTX110 is a drug that is being tested as a potential treatment against cancerous tumors, while CTX120 and CTX130 are other candidate drugs for various cancer types. Cancer immunology is an already-massive market that's estimated to grow to $126.9 billion by 2026, representing a 9.6% compound annual growth rate. However, there's already a fair bit of competition in this space.

Specifically, CTX110 is what's referred to as an allogeneic chimeric antigen receptor T-cell (CAR-T) therapy. Healthcare giantsGilead (NASDAQ:GILD) and Novartis (NYSE:NVS) have their own CAR-T therapies, but the problem they face is that this treatment is extremely expensive for patients, not to mention being quite slow.

CAR-T therapies require scientists to remove immune cells from a patient, teach those cells to better fight cancer cells, and then reintroduce them into the patient's system. Understandably, this is both time-consuming and expensive, with treatments costing hundreds of thousands of dollars per patient.

One solution to these problems is to develop CAR-T drugs from donor cells. This process, referred to as allogeneic, could lead to complications from patients whose immune systems react against or even reject the donor cells. This is where CRISPR comes in, as its gene-editing technology can significantly minimize the chance of rejection.

Ananalyst at Needham named Alan Carr said,"An off-the-shelf allogeneic product presents several advantages and we believe CRISPR is uniquely positioned to succeed in the space, given rights are wholly owned and the simplicity and flexibility of CRISPR technology to make multiple simultaneous edits."

While the market for gene-editing technology is excellent, the last question worth asking is where CRISPR Therapeutics stands in comparison to its rivals. CRISPR's potential competitors include Intellia Therapeutics (NASDAQ:NTLA) and Editas Medicine (NASDAQ:EDIT), both companies working in the gene-editing field. The latter is developing its own sickle cell treatment to rival CTX001. However, Editas is also working on a gene-editing drug called EDIT-101 that would help babies with protein deficiencies in their retinas leading to blindness. So while there is some overlap between the two companies, there are also areas in which the two aren't in direct competition.

CRISPR also enjoys a cushier financial position than the other two companies, with $427.9 million in cash reserves compared to Editas' $317.9 million and Intellia's $275.8 million. However, CRISPR Therapeutics doesn't have as strong an intellectual-property portfolio as some of its competitors; Editas owns more than 70 issued patents and 600 pending patent applications, not to mention the rights to technology owned by institutions such as MIT and Harvard.

Overall, it's hard to say which gene-editing company will end up reigning supreme in the years to come. I would take a shotgun approach, investing a little in most of the promising gene-editing biotech stocks in the market right now while acknowledging that many will likely fail. CRISPR Therapeutics definitely makes the list of gene-editing stocks worth buying, although only for investors willing to jump into a high-risk, high-reward type of investment.

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Is CRISPR Therapeutics a Buy? - Motley Fool