Archive for the ‘Crispr’ Category

Depression is a dark horse.

The disease often goes unnoticed, but affects work performance, social interaction and the ability to take pleasure in everyday life. According to theNational Center for Biotechnology Information, antidepressants only help around 50 percent of those who struggle with depression and anxiety and, even when they are effective, scientists have yet to understand how they work in the brain.

MSU associate professor of physiology A.J. Robison and his lab used new CRISPR-based technology to uncover pathways of depression-like behavior in the mouse brain. Credit: College of Natural Science

But groundbreaking research in the lab of Michigan State University scientistA.J. Robison, associate professor in theDepartment of Physiologyand MSUsNeuroscience Program, is directing some new rays of light onto the molecular, cellular and circuit-level mechanisms underlying depression-like diseases.

Theresultswere recently published inNature Communications.

In this paper, we perform the first ever CRISPR-based gene editing [a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified] in a single circuit between two areas of the mouse brain, explained Robison about the culmination of five years of research funded by the National Institutes of Mental Health. We can reach into the mouse brain and manipulate specific genes in a circuit involved in depression and anxiety-like behaviors a critical advance on the road to genetic medicine for psychiatric diseases.

Scientists estimate there are roughly 80-100 billion neurons connecting regions of the brain. To accomplish the feat of locating and manipulating a single gene in a single circuit required new and sophisticated technology. With the expertise of co-author Rachael Neve, director of theGene Transfer Core at Massachusetts General Hospital, they developed it.

The key advance is that we designed a dual-vector system to manipulate a specific gene in the connections between two brain areas, and that has never been done before, Robison said.

Cross section of a mouse brain. The projections of the cells between the vHPC and NAc, shown here in neon green, are manipulated by the new CRISPR viral vector-based technology developed by Rachael Neve and the Robison Lab. Credit: Andrew Eagle

The neurons that Robison and his team zeroed in on originate in the ventral hippocampus (vHPC), a deep-seated structure that projects to regions in the brain important in stress susceptibility, mood and social avoidance. Neurons rooted in the vHPC reach out with branch-like structures called axons to connect with the nucleus accumbens, or NAc. The completed circuit is regulated by the star of the pioneering paper, the transcription factor known as DFosB.

Using the viral vector technology specifically designed and packaged by Neve, the team split the CRISPR system in half. Half of the system, inert on its own, was an enzyme that can mutate DNA in the vHPC. The other half, a guide RNA, was sent to all cells that project to the NAc and tells the enzyme where to bind and the specific gene to mutate. Only those cells specific to the circuit from the vHPC to the NAc got both halves, triggering the enzyme to bind with and turn off a single gene: FosB.

When the FosB gene was turned off in the neurons, we were able to get a circuit-specific behavioral effect relevant to a disease like depression, said Robison about the landmark discovery. When we put it back, or rescued it within the circuit, the effect was erased.

Claire Manning was a key contributor to the groundbreaking study and is now a postdoctoral researcher at Stanford University. Credit: Ken Moon

One of the most exciting findings from our investigations was the circuit-specific role of the FosB protein in conferring resilience to stress, Eagle said. We also discovered that FosB altered the excitability of hippocampal circuit neurons and may be affecting long-term downstream changes that lead to changes in the activity of this circuit. But removing DFosB permanently altered the expression of a suite of genes, in effect removing the conductor from the orchestra. To that end, the paper goes on to report in-depth experiments on DFosB largely done by the members of theRobison Labincluding co-first authorsClaire Manning, a 2019 neuroscience graduate, now a postdoc at Stanford University; andAndrew Eagle, a former postdoctoral researcher, now an assistant professor in the MSU Department of Physiology.

Andrew Eagle, shown here imaging a mouse brain, played a major role in conducting experiments to further probe the function of DFosB. Credit: Research@MSU.

Based on the findings in the paper, the Robison Lab will continue to develop highly collaborative and cutting-edge techniques, accelerated by MSUs newly completed Interdisciplinary Science and Technology Building. This work is important because it elucidates a potential mechanism, namely FosB, for how stress may contribute to depression, Eagle continued. Future clinical work may find ways to directly manipulate FosB, or more likely one of its gene targets, to provide resilience to stress and decrease the incidence of depression in vulnerable people.

The end of this paper, which shows us measuring the changes of expression in hundreds of genes when we remove DFosB, is only the beginning of years of work for our lab, Robison said. Which genes are important and what are they doing in the brain? This is the challenge of a lifetime for me and my lab.

This article is repurposed content originally featured on the College of Natural Sciences website.

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Illuminating the opaque pathways of depression - MSUToday

The CRISPR and Cas genes market is projected to reflect stupendous growth with a 21.2% CAGR between the years 2020 and 2026. A Fact.MR study has found that that the coronavirus outbreak has generated key lucrative opportunities to participants in the market in the short term. Application of CRISPR technologies diagnosing covid-19 cases, and potential for a cure has is likely to contribute to market growth. Wide ranging field of applications will sustain the high growth for years to come.

CRISPR and Cas gene systems have attracted significant attention among researchers owing to cheaper, faster, and accurate, results in comparison to other existing processes for genome editing. In addition, rising investments in the field by the biotech companies will contribute substantially to the growth of the industry through the forecast period, says the FACT.MR report.

Request a sample of the report to gain more market insights at:https://www.factmr.com/connectus/sample?flag=S&rep_id=4823

CRISPR and Cas Genes Market- Key Takeaways

CRISPR and Cas Genes Market- Driving Factors

CRISPR and Cas Genes Market- Major Restraints

COVID-19 Impact on CRISPR and Cas Genes Market

CRISPR and CAS gene technology developers are likely to benefit from the coronavirus pandemic. Researchers have been studying these technologies as for potential in covid-19 diagnostic applications. In addition, CRISPR is also providing opportunities in terms of a cure through destroying the RNA structure of the virus. The market is expected to continue growing exponentially even in the post-pandemic era owing to applications in plant gene editing and drug development applications for the foreseeable future.

Explore the global CRISPR and Cas Genes market with 88 figures, 24 data tables, along with the table of contents of the report. You can also find detailed segmentation on:https://www.factmr.com/connectus/sample?flag=RM&rep_id=4823

Competitive Landscape

Thermo Fisher Scientific, Addgene, and Integrated DNA Technologies are some of the major players in the CRISPR and Cas gene market.

CRISPR and Cas gene researchers have been investing increasingly in tech innovations and diversification of potential applications for a growing number of end use verticals, supporting long term prospects.

For instance, Thermo Fisher Scientific has collaborated with BioEnergy Science Center to use CRISPR/Cas9 protein delivery for a non-GMO process to edit plant genes. Integrated DNA Technologies has engineered a new high-fidelity Cas9 nuclease through unbiased bacterial screen. Beam Therapeutics is collaborating with Addgene for the commercialization of base editing tools for laboratory use in the United States.

About the Report

This study offers readers a comprehensive market forecast of the CRISPR and Cas gene market. Global, regional and country-level analysis of the top industry trends impacting the CRISPR and Cas gene market is covered in this FACT.MR study. The report offers insights on the CRISPR and Cas gene market on the basis of product (Vector-based Cas and DNA-free Cas), application (genome engineering, disease models, functional genomes, knockdown/activation, and others), and end user (biotechnology & pharmaceutical companies, academic research institutes, and contract research organizations) across five regions (North America, Latin America, Europe, Asia Pacific, and MEA).

Media Release: https://www.factmr.com/media-release/1591/global-crispr-and-cas-genes-market

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The Vector-Based Systems to be Highly Lucrative in CRISPR and Cas Genes Market, Fact.MR Study - The Cloud Tribune

Intellia Therapeutics Inc. [NASDAQ: NTLA] gained 8.59% or 1.66 points to close at $20.92 with a heavy trading volume of 1102470 shares. The company report on September 2, 2020 that Intellia Therapeutics to Present at Bairds 2020 Virtual Global Healthcare Conference.

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, will present at Bairds 2020 Virtual Global Healthcare Conference on Wednesday, September 9, 2020 at 10:50 a.m. ET.

A live webcast of Intellias presentation will be accessible through the Events and Presentations page of the Investors & Media section of the companys website at http://www.intelliatx.com. To access the webcast, please log on approximately 15 minutes prior to the start time, to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Intellias website for approximately 14 days following the live event. About Intellia TherapeuticsIntellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

It opened the trading session at $19.78, the shares rose to $21.44 and dropped to $19.78, the range by which the price of stock traded the whole day. The daily chart for NTLA points out that the company has recorded 121.56% gains over the past six months. However, it is still -127.89% lower than its most recent low trading price.

If we look at the average trading volume of 784.64K shares, NTLA reached to a volume of 1102470 in the most recent trading day, which is why market watchdogs consider the stock to be active.

Based on careful and fact-backed analyses by Wall Street experts, the current consensus on the target price for NTLA shares is $30.15 per share. Analysis on target price and performance of stocks is usually carefully studied by market experts, and the current Wall Street consensus on NTLA stock is a recommendation set at 2.10. This rating represents a strong Buy recommendation, on the scale from 1 to 5, where 5 would mean strong sell, 4 represents Sell, 3 is Hold, and 2 indicates Buy.

Oppenheimer have made an estimate for Intellia Therapeutics Inc. shares, keeping their opinion on the stock as Outperform, with their previous recommendation back on February 28, 2020. The new note on the price target was released on February 14, 2020, representing the official price target for Intellia Therapeutics Inc. stock.

The Average True Range (ATR) for Intellia Therapeutics Inc. is set at 1.32, with the Price to Sales ratio for NTLA stock in the period of the last 12 months amounting to 23.83. The Price to Book ratio for the last quarter was 3.17, with the Price to Cash per share for the same quarter was set at 7.56.

Intellia Therapeutics Inc. [NTLA] gain into the green zone at the end of the last week, gaining into a positive trend and gaining by 15.42. With this latest performance, NTLA shares gained by 1.97% in over the last four-week period, additionally plugging by 121.56% over the last 6 months not to mention a rise of 46.77% in the past year of trading.

Overbought and oversold stocks can be easily traced with the Relative Strength Index (RSI), where an RSI result of over 70 would be overbought, and any rate below 30 would indicate oversold conditions. An RSI rate of 50 would represent a neutral market momentum. The current RSI for NTLA stock in for the last two-week period is set at 54.55, with the RSI for the last a single of trading hit 59.58, and the three-weeks RSI is set at 53.42 for Intellia Therapeutics Inc. [NTLA]. The present Moving Average for the last 50 days of trading for this stock 20.56, while it was recorded at 19.08 for the last single week of trading, and 16.77 for the last 200 days.

Operating Margin for any stock indicates how profitable investing would be, and Intellia Therapeutics Inc. [NTLA] shares currently have an operating margin of -246.78. Intellia Therapeutics Inc.s Net Margin is presently recorded at -230.92.

Return on Total Capital for NTLA is now -37.57, given the latest momentum, and Return on Invested Capital for the company is -35.52. Return on Equity for this stock declined to -36.34, with Return on Assets sitting at -29.21. When it comes to the capital structure of this company, Intellia Therapeutics Inc. [NTLA] has a Total Debt to Total Equity ratio set at 6.81. Additionally, NTLA Total Debt to Total Capital is recorded at 6.37, with Total Debt to Total Assets ending up at 5.50. Long-Term Debt to Equity for the company is recorded at 4.68, with the Long-Term Debt to Total Capital now at 4.38.

Reflecting on the efficiency of the workforce at the company, Intellia Therapeutics Inc. [NTLA] managed to generate an average of -$368,641 per employee. Receivables Turnover for the company is 7.09 with a Total Asset Turnover recorded at a value of 0.13.Intellia Therapeutics Inc.s liquidity data is similarly interesting compelling, with a Quick Ratio of 7.50 and a Current Ratio set at 7.50.

With the latest financial reports released by the company, Intellia Therapeutics Inc. posted -0.49/share EPS, while the average EPS was predicted by analysts to be reported at -0.62/share. When compared, the two values demonstrate that the company surpassed the estimates by a Surprise Factor of 21.00%. The progress of the company may be observed through the prism of EPS growth rate, while Wall Street analysts are focusing on predicting the 5-year EPS growth rate for NTLA. When it comes to the mentioned value, analysts are expecting to see the 5-year EPS growth rate for Intellia Therapeutics Inc. go to 30.00%.

There are presently around $1,130 million, or 83.00% of NTLA stock, in the hands of institutional investors. The top three institutional holders of NTLA stocks are: ARK INVESTMENT MANAGEMENT LLC with ownership of 11,403,379, which is approximately 11.369% of the companys market cap and around 0.90% of the total institutional ownership; SUMITOMO MITSUI TRUST HOLDINGS, INC., holding 6,195,189 shares of the stock with an approximate value of $129.57 million in NTLA stocks shares; and NIKKO ASSET MANAGEMENT AMERICAS, INC., currently with $129.33 million in NTLA stock with ownership of nearly -12.8% of the companys market capitalization.

Positions in Intellia Therapeutics Inc. stocks held by institutional investors increased at the end of August and at the time of the August reporting period, where 100 institutional holders increased their position in Intellia Therapeutics Inc. [NASDAQ:NTLA] by around 10,206,052 shares. Additionally, 50 investors decreased positions by around 2,748,793 shares, while 30 investors held positions by with 41,050,254 shares. The mentioned changes placed institutional holdings at 54,005,099 shares, according to the latest SEC report filing. NTLA stock had 42 new institutional investments in for a total of 4,196,261 shares, while 18 institutional investors sold positions of 408,199 shares during the same period.

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Intellia Therapeutics Inc. [NTLA] gain 42.57% so far this year. What now? - The DBT News

Spiderman and CRISPR-Taming Chemist Amit Choudhary both tell us that "with great power comes great responsibility". In Amit's case, he speaks of power and responsibility with CRISPR gene editing technology.

Today, the gene editing tool CRISPR-Cas9 serves as a "genetic 'find and replace' function," that allows one to search and cut a DNA sequence at its precise location.

Yet, this powerful tool must be used with great responsibility, given that a misstep could have irreversible consequences. Amit has pioneered precision tools - small molecule inhibitors, activators, and shredders - to control Cas9 activity. Having successfully fine-tuned CRISPR activity with first generation technologies, Amit is eager to pave the way for everyone to benefit from this gene editing technology safely and responsibly.

Amit's journey with precision control tools for CRISPR-Cas9 demonstrates that responsible use of technology allows us to build solutions that are both effective and finely tuned.

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The Power of CRISPR Cas9 Comes With Great Responsibility - Technology Networks

In November 2018, at a gene-editing summit hosted by scientific societies from the U.S., the U.K., and Hong Kong, a Chinese researcherannouncedthat he had created the worlds first genetically modified babies.He Jiankuifully expected to be celebrated for a scientific breakthrough; hementionedthe Nobel Prize. Instead, he was almost universally condemned.

Key figures associated with theU.S. National AcademiesandU.K. Royal Societyjoined in thecriticismbut did not reject heritable genome editing. Instead, they objected to the Chinese researchers timing. It was too soon, they said. It hadnt been done as they thought it should have been. But according to the researcher now being called a rogue, it was theNational Academies 2017 reportthat had given him the green light for his experiments.

In the aftermath of this headline-grabbing debacle, the scientific societies decided on a do-over. They declared it time to define a rigorous, responsible translational pathway toward clinical use of heritable genome editing. Theyset upa carefully selectedinternational commissionwith themandateto map the scientific details ofhowdesigner-baby technology could be brought to the fertility clinic.

This mandate was flawed from the start. The idea that now is the time to set aside the deeply controversial question ofwhetherheritable genome editing should be done at all so that a small group of experts can settle the nitty-gritty details ofhowit should take place is entirely backward. It flies in the face of the widely shared acknowledgment that scientists alone cannot make this decision; that we must have wide-ranging and inclusive public discussions aimed at buildingbroad societal consensus. It undermines policies in some70 countriesaround the world that prohibit heritable genome editing. And its a slap in the face to the manyscientists,biotech executives,human rights and social justice advocates, and others who support a moratorium or ban on altering the human germline.

The commissions 225-pagereport, released on Sept. 3, does have some strong points. It is more cautious than the previous report, recommending that heritable genome editing should initially be allowed only in the exceedingly rare cases where embryo screening for severe genetic conditions would not be an option. And it paints a vivid picture of the significant technical hurdles facing those eager to pursue heritable human genome editing: shortfalls in the editing tools, in the technologies necessary to test safety and efficacy, even in our understanding of the genetics underlying most heritable diseases.

These findings ought tolay to restthe unfounded assumption that engineering the genomes of human embryos will soon be safe and effective. But even the most cautious considerations of technical safety cant stand-in for the fundamental point that the decision about whether to allow heritable genome editing should be driven by our values, not settled by the science.

The commission claims they are not endorsing heritable genome editing, merely constructing maps of the technological path in case a country should wish to use them. At best, this puts the cart before the horse and sends both horse and cart down a one-way road.

Heritable genome editing cant be separated from its real-world consequences. There are already clear signs that legalizingit would lead to reproductive tourism, jurisdiction shopping, andmission creep. As an example, the U.K.sapprovalof so-called mitochondrial donation for a small number of women with certain mitochondrial DNA diseases wasquickly followedby fertility clinics inUkraine,Spain, and Greeceoffering this high-risk technique, with no evidence of effectiveness, for general and age-related infertility.

A similar trajectory is all too easy to foresee if heritable genome editing is approved, even for limited circumstances. Especially where fertility services are offered on a for-profit basis, its unlikely that any boundaries would hold. We could soon see fertility clinics marketing genetically upgraded embryos, tempting parents-to-be with ads about giving their child the best start in life. From there, a normalized system of market-based eugenics could emerge, exacerbating already existing discrimination, inequality, and conflict.

Amid our multiple ongoing crises, it would be easy to overlook another report on still speculative biotechnology. But this one represents a profoundly consequential step, one that tries to settle in advance the coming decision about whether to engineer genes and traits passed on to future children and generations. Its another attempt to focus discussion on the science, while minimizingthe complex social realities in which scientific and technological developments unfold.

KatieHassonis program director on genetic justice andMarcyDarnovskyis executive director of theCenter for Genetics and Society,a non-profit organization based in Berkeley, California that works to encourage responsible uses and effective governance of human genetic and assisted reproductive technologies.

Excerpt from:
Are we mapping a path to CRISPR babies? | TheHill - The Hill

The genome editor CRISPR, whose invention is at the heart of a fierce patent battle, typically uses an RNA molecule (red) to guide a DNA-cutting enzyme such as Cas9 (orange) to a DNA sequence (blue) targeted for cutting.

By Jon CohenSep. 11, 2020 , 6:40 PM

The long-running patent battle over CRISPR, the genome editor that may bring a Nobel prize and many millions of dollars to whoever is credited with its invention, has taken a new twist that vastly complicates the claims made by a team led by the University of California.

The Patent Trial and Appeal Board (PTAB) ruled 10 September that a group led by the Broad Institute has priority in its already granted patents for uses of the original CRISPR system in eukaryotic cells, which covers potentially lucrative applications in lab-grown human cells or in people directly. But the ruling also gives the University of California group, which the court refers to as CVC because it includes the University of Vienna and scientist Emmanuelle Charpentier, a leg up on the invention of one critical component of the CRISPR tool kit.

This is a major decision by the PTAB, says Jacob Sherkow, a patent attorney at the University of Illinois at Urbana-Champaign who has followed the case closely but is not involved. Theres some language in the opinion from today thats going to cast a long shadow over the ability of the [CVC] patents going forward.

Jennifer Doudna, a biochemist at the University of California at Berkeley, and Charpentier, now with the Max Planck Institute, first published evidence that the bacteria-derived CRISPR system could cut targeted DNA in June 2012, seven months before the Broad team led by Feng Zhang published its own evidence it could be a genome editor. But the CVC team did not show in its initial paper that CRISPR worked inside eukaryotic cells as Zhangs team did in its report, even though the original CVC patent application broadly attempted to cover any use of the technology. The U.S. Patent and Trademark Office issued several CRISPR-related patents to Broad beginning in 2014, sparking a legal a battle in 2016 based on CVC claims of patent interference. That led to a first PTAB trial, which seemed to deliver a mixed verdict, ruling that the eukaryotic CRISPR and other uses of the genome editor were separate inventions, patentable by Broad and CVC respectively. Unsatisfied, CVC took the issue to a federal court, which denied its appeal.

CVC subsequently filed new claims that led PTAB to declare a second interference. The board this time did a more direct comparison of which group had the best evidence for the first demonstration that CRISPR worked in eukaryotic cells. The PTAB ruling did not accept CVC arguments that it crossed this line first, giving the priority edge to the Broad.

This doesnt settle the dispute, however, but instead requires that CVC provide more evidence at a future hearing that it was first. The interference [hearing] is going ahead all the way this time to determine who was the first to invent, says Catherine Coombs, a patent attorney at the U.K legal firm Murgitroyd who has not been involved in the case but handled other CRISPR litigation in Europe. Coombs notes that theres a large gap between the CRISPR patent environment in the United States and Europe, where CVC has won the upper hand in the European Union patent office.

Sherkow anticipates the PTAB will face a tough, complex decision. Its going to need to subpoena Doudna and subpoena Zhang and subpoena a bunch of graduate students and put a bunch of eight-year-old lab notebooks in evidence, says Sherkow.

CRISPR, which typically comprises a DNA-cutting enzyme known as Cas9 and a molecule that guides it to a specific DNA sequence, often is compared to molecular scissors. A key dispute in the patent battle focuses on the guide component. Zhangs first description of CRISPR working in eukaryotic cells used a guide that combined two RNA molecules while CVCs use relied on a single RNA to do the same thing. This single molecule guide RNA now is the standard tool in the field.

A statement from a University of California spokesperson says it is "pleased" with the new ruling, noting that it denied several of the Broads motions. The PTAB "has ruled in our favor in most instances and will continue with the interference proceeding to determine which party was the first to invent CRISPR in eukaryotes, the statement says. [W]e remain confident that the PTAB will ultimately recognize that the Doudna and Charpentier team was first to invent the CRISPR-Cas9 technology in eukaryotic cells.

A statement issued by the Broad calls for something akin to a peace treaty. Although we are prepared to engage in the process before the PTAB and are confident these patents have been properly issued to Broad, we continue to believe it is time for all institutions to move beyond litigation and instead work together to ensure wide, open access to this transformative technology, the statement says. The best thing, for the entire field, is for the parties to reach a resolution and for the field to focus on using CRISPR technology to solve todays real-world problems.

Many observers of the patent battle long have hoped that the Broad and CVC will reach a settlement, but Sherkow thinks its less likely now. Almost every outcome is stacked in Broads favor, he says. If the CVC wins, he says, they will have the patent for the single molecule guide, but the Broad will not lose its eukaryotic patent and, at worst, will have to share it. If CVC loses, theyre toast, they come away empty, says Sherkow. But Ive been wrong about settlement before so theres every expectation that Ill be wrong again.

The PTAB ruling does not specify a date for its next hearing.

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The latest round in the CRISPR patent battle has an apparent victor, but the fight continues - Science Magazine

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia, and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the currently available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

[Read: These tech trends defined 2020 so far, according to 5 founders]

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene-editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts as a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up our genome. This means that researchers like me can leverage CRISPR technology to revolutionize gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for the immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

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CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the number of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibodies against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually, the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology, provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

This article is republished from The Conversation by Samira Kiani, Associate Professor of Pathology, University of Pittsburghunder a Creative Commons license. Read the original article.

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How CRISPR is tackling the troubling immune response thats plagued gene therapy until now - TNW

AUSTIN, Texas One of the biggest scientific advances of the last decade is getting better thanks to researchers at The University of Texas at Austin; the University of California, Berkeley; and Korea University. The team has developed a new tool to help scientists choose the best available gene-editing option for a given job, making the technology called CRISPR safer, cheaper and more efficient. The tool is outlined in a paper out today in Nature Biotechnology.

The CRISPR gene-editing technique holds tremendous potential to improve human health, agriculture and the future of people on the planet, but the challenge lies in the delicate nature of gene editing there is almost no room for error.

To edit genes, scientists use dozens of different enzymes from a naturally occurring system called CRISPR. Researchers locate a problematic DNA sequence and use these specialized enzymes to snip it as if using a pair of scissors, allowing genetic material to be added, removed or altered. But these scissors are not perfect. Accuracy and effectiveness vary by the CRISPR enzyme and the project. The new tool guides users, so they can pick the best CRISPR enzyme for their high-stakes gene edit.

We designed a new method that tests the specificity of these different CRISPR enzymes how precise they are robustly against any changes to the DNA sequence that could misdirect them, and in a cleaner way than has ever been done before, said Steve Jones, a UT research scientist who co-wrote the paper with Ilya Finkelstein, an associate professor of molecular biosciences.

Problems can occur when a CRISPR enzyme targets the wrong sections of DNA. Each CRISPR enzyme has strengths and weaknesses in editing different sequences, so the researchers set out to create a tool to help scientists compare the different enzymes and find the best one for a given job.

CRISPR wasnt designed in a lab. It wasnt made by humans for humans. It was made by bacteria to defend against viruses, said John Hawkins, a Ph.D. alumnus who was recently with UTs Oden Institute for Computational Engineering and Sciences. There is incredible potential for its use in medicine, but the first rule of medicine is do no harm. Our work is trying to make CRISPR safer.

The team of researchers developed a library of DNA sequences and measured how accurate each CRISPR enzyme was, how long it took the enzyme to edit the sequences and how precisely they edited the sequence. For some tasks the commonly used enzyme CRISPR-Cas9 worked best; in others, different enzymes performed much better.

Its like a standardized test, Hawkins said. Every student gets the same test, and now you have a benchmark to compare them.

The tool allows scientists to choose the best enzyme for editing on the first try, so the process becomes more efficient and cheaper. Additionally, it gives scientists information about where mistakes are most likely to occur for each enzyme, saving time.

This technique gives us a new way to reduce risk, Jones said. It allows gene edits to be more predictable.

Nicole V. Johnson, Kuang Hu, James R. Rybarski, William H. Press and Ilya J. Finkelstein of The University of Texas at Austin; Cheulhee Jung of Korea University; and Janice S. Chen and Jennifer A. Doudna of University of California, Berkeley, contributed to the research.

The research was funded by a College of Natural Sciences Catalyst Grant, The Welch Foundation and the National Institutes of Health.

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Matching CRISPR to the Job Improves the Safety, Efficiency of the Gene-Editing Tool - UT News | The University of Texas at Austin

The fight against the novel coronavirus is putting plenty of healthcare ETFs in the spotlight this year, including the ARK Genomic Revolution Multi-Sector Fund (CBOE: ARKG), but theres much more to the ARKG story.

In fact, ARKG was one of the best-performing healthcare ETFs prior to COVID-19 becoming part of the daily lexicon. Thats a trajectory the actively managed ARK fund can continue when the virus is defeated due to its exposure to disruptive themes, including CRISPR.

Looking ahead, CRISPR-based innovations to accelerate given the technologys ease of use, cost-efficacy, growing body of research surrounding its safety, and AI-powered CRISPR nuclease selection tools. CRISPR could also be utilized to address some of the most prominent healthcare problems, which opens up a significant investment opportunity in monogenic diseases.

In a recent Harvard study, CRISPR gene editing transformed white fat into brown fat that burns energy and can contribute to weight loss, ARK analyst Ali Urman wrote in a recent report. The engineered cells helped mice avoid weight gain and potentially diabetes, despite a high fat diet.

CRISPR can cut DNA/RNA at a single point or in stretches; insert DNA/RNA and create novel gene sequences; activate and silence genes without making permanent changes; regulate protein expression levels epigenetically; record and timestamp biological events; track the movement of specific biological molecules; identify the presence of specific cancer mutations and bacteria; locate molecules without making changes; target and destroy specific viral and bacterial DNA and RNA; interrogate gene function multiplexed, and activate drug release at a specified trigger.

As the aforementioned Harvard study notes, theres a big opportunity for CRISPR as it pertains to fighting to obesity and that could be meaningful for long-term ARKG investors.

This study could have profound implications for CRISPRs total available market. In the United States, more than 34 million people have diabetes and the medical costs and loss of work wages associated with it are valued at $327 billion annually, notes ARKs Urman. If CRISPR were to be effective in increasing insulin sensitivity, CRISPRs total available market would expand significantly, if not exponentially.

ARKG includes companies that merge healthcare with technology and capitalize on the revolution in genomic sequencing. These companies try to better understand how biological information is collected, processed, and applied by reducing guesswork and enhancing precision; restructuring health care, agriculture, pharmaceuticals, and enhancing our quality of life.

For more on disruptive technologies, visit our Disruptive Technology Channel.

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

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CRISPR Benefits Available in This Genomics ETF - ETF Trends

Broadcast Date: October 6, 2020Time: 8:00 am PT, 11:00 am ET, 17:00 CET

Next Spring marks the tenth anniversary of two key moments in the annals of CRISPR. In San Juan, Puerto Rico, Jennifer Doudna, PhD and Emmanuelle Charpentier, PhD discussed a collaboration that would culminate in an immortal paper that repurposed CRISPR-Cas9 into a programmable gene targeting technology. Meanwhile, Feng Zhang heard about CRISPR for the first time, setting him on course to become one of the first investigators to demonstrate genome editing in mammalian cells.

Since the publication of those classic reports in 201213, CRISPR has catalyzed a revolution in genome editing, empowering basic researchers around the world, spawning a multibillion-dollar biotech industry, and showing signs of genuine promise in the clinic. But CRISPR has also been misapplied, notably in the creation of CRISPR babies in 2018, prompting calls for a moratorium on germline editing.

Kevin Davies, PhD, founding executive editor of The CRISPR Journal, has chronicled these events in his new book, Editing Humanity: The CRISPR Revolution and the New Era of Genome Editing. This GEN keynote webinar takes place on the official publication day of the book. Davies will share highlights and insights from his reporting over the past few years and consider some of the exciting new directions that CRISPR will empower in the years ahead.

A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelists.

About GEN KEYNOTE webinars:

GEN invites renowned experts to lecture on topics of broad interest to the biotechnology and biomedical community. Look for more GEN KEYNOTE webinars in 2020!

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Heroes and Villains: Reflections on the CRISPR Revolution - Genetic Engineering & Biotechnology News

CRISPR represents a new frontier in gene editing. In comparison to other currently available technologies, it is a less expensive, more specific and simpler-to-use gene editing tool but is not without its criticism or concerns.

Photo: Unsplash

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Gene editing is often discussed and presented in the context of revolutionizing treatment and diagnostics. In 2012, Jennifer Doudna and Emmanuelle Charpentier demonstrated the potential of CRISPR, which made the promise of gene editing therapies more tangible.

However, COVID-19 has brought to the fore additional applications, most notably exploring how CRISPR can be used as a mechanism to develop non-gene based therapies. Once the process is refined, it could represent another notable step forward for the healthcare sector, although several regulatory hurdles still remain before CRISPR manufactured non-gene editing therapies are widespread.

The CRISPR technology works by coding for a specific gene sequence using guide RNA. When the appropriate DNA sequence is found, the Cas9 protein, working as a pair of scissors, cuts the DNA at the desired location. Once the cut has been made, the gene can be disabled, or missing genetic information can be inserted or replaced.

Due to its characteristics, CRISPR represents a new frontier in gene editing; in comparison to other currently available technologies, it is a less expensive, more specific and simpler-to-use gene editing tool.

The technology is not without its criticism or concerns. Most notably, there is the possibility of off-target mutations, whereby cuts are made in unintended DNA sequences. Additionally, ethical questions related to gene editing as a technology overall remain pervasive among policy makers and the public alike. Fortunately, increased study and greater exposure to the technology are lessening some of these concerns, and it continues to represent a potential and long-awaited therapeutic option for many genetic, and often rare, diseases, for many of which treatments have remained elusive.

COVID-19 and the Ebola epidemic in the mid-2010s illustrated the lengthy time lag of developing preventative therapies. CRISPR technology has the potential to overcome this challenge by significantly accelerating the development of vaccines or therapeutic options to respond to pandemics. This is because CRISPR technology is based on a naturally occurring gene editing system that is found in bacteria, which can be used to fight viruses.

Increased urbanization and contact between different world regions is likely to lead to an increase in the frequency of epidemics, and this new reality has spurred increased interest in CRISPR as a means to quickly respond to disease outbreaks, or even unpredictable seasonal infections. A quick response can help protect healthcare professionals in the short term, and embrace the promise of prevention is better than treatment.

There are a range of different applications for using CRISPR in the development of therapeutic solutions.

Traditional vaccines typically consist of a weakened or dead strand of the virus that it is meant to inoculate patients from viruses. When developing a vaccine, the manufacturer must select viral strands, which are then either grown and incubated in hen eggs or cells.

CRISPR can be used to modify the incubator to increase viral products, which reduces the number of eggs or cells needed in the manufacturing process.

CRISPR technology can be used to engineer B cells, a white blood cell that produces the antibodies that in turn combat pathogens. Using CRISPR, B cells can be injected into patients and provide them with the antibodies to an infection, such as COVID-19, without ever being exposed to the disease itself.

This CRISPR technique effectively skips the step in traditional vaccines of introducing a version of the virus to effectively stimulate the development of an antibody. If the platform, rather than a specific vaccine, can receive authorization, it will allow companies to respond to viral outbreaks in a matter of months, as opposed to years, by simply engineering the appropriate B cells to respond to the viral threat.

The major drawback from this approach is that in the limited studies conducted thus far, it has only offered protection for a very short period of time.

Our increased understanding of genes has given rise to a new class of vaccine known as mRNA vaccines. The concept has become increasingly popular over the last few years, especially during COVID-19, and is the basis of CureVacs approach. The vaccines work by triggering cells to develop antibodies to a specific virus, e.g. SARS-CoV-2,without needing to introduce the virus itself.

Finally, beyond vaccination, there are plenty of other potential uses of CRISPR to combat COVID-19 and viral pandemics in the future. A recent Stanford study used CRISPR to directly target COVID-19 virus (SARS-CoV-2) and disable disease at an RNA level.

Beyond the current pandemic, CRISPR has potential applications to combat other viruses that humanity has been struggling with, ranging from the seasonal flu to HIV. Currently, influenza vaccinations are developed based on hypothesizing about next years viral strain. Due to the manufacturing process, companies cannot quickly respond to a change in the external environment. However, CRISPR introduces a more agile and cheaper manufacturing process, either by improving and reducing the incubation process or aiming to leverage the human body to produce its own antibodies,

Policy has already begun to respond to the potential implications of gene editing as part of a healthcare systems pandemic response mechanism. In a bold step, suggesting a softening of European Union regulations, the European Parliament and Council of the European Union adopted a regulation introducing a temporary derogation to the GMO regulation for clinical trials on vaccines for COVID-19 utilizing gene editing technologies.

This decision could have wider implications for the future use of gene editing for therapeutic use in the EU, especially as the EU looks to review its pharmaceutical strategy for the coming years, where personalized and gene therapies will be a significant component.

COVID-19 has brought the potential of CRISPR-based therapies to combat viral infections further to the foreground and encouraged research in the field. COVID-19 has also played an important role in helping to demystify gene editing to a broader audience. These developments are surely to lead to increased interest and investment in gene editing as a therapeutic solution or manufacturing mechanism outside of the rare and genetic disease space.

The EUs decision to provide an exemption to the GMO regulation could be the first step in a renewed understanding of CRISPR, or it could lead to political backlash. The coming months and years are likely to shape the future of CRISPR and gene editing as a therapeutic solution.

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Could CRISPR Create a COVID-19 Vaccine? BRINK News and Insights on Global Risk - BRINK

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the current available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts like a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up or genome. This means that researchers like me can leverage CRISPR technology to revolutionise gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the quantity of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibody against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology,provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

Samira Kiani, Associate Professor of Pathology, University of Pittsburgh

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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CRISPR Can Help Combat the Troubling Immune Response Against Gene Therapy - Gizmodo Australia

Korro Bio has got off a meaty series A funding round, nabbing $91.5 million to run its platform of single-base RNA edits to treat a range of diseases.

The Cambridge, Massachusetts-based biotech will use the cash haul toward getting its preclinical program into the clinic, while also establishing a broad portfolio of innovative RNA editing therapies.

The financing was led by Wu Capital with help from current investors Atlas Venture and New Enterprise Associates. Additional new investors include Qiming Venture Partners USA, Surveyor Capital (a Citadel company), Cormorant Asset Management, MP Healthcare Venture Management and Alexandria Venture Investments.

Accelerate Clinical Operations Across Sponsors, CROs, and Partners

The most advanced life sciences organizations know that digital innovation and multi-platform integrations are essential for enabling product development. New platforms are providing the life sciences industry with an opportunity to improve the efficiency of clinical trials and reduce costs while remaining compliant and reducing risk.

Korro's platform, known as OPERA (Oligonucleotide Promoted Editing of RNA), harnesses the bodys natural base editing system.

OPERA can repair disease-causing mutations at the RNA level, in addition to creating therapeutically beneficial versions of proteins to improve patient outcomes, the biotech said in a statement. This method is based upon the work of Josh Rosenthal, Ph.D., of the Marine Biological Laboratory in Massachusetts, an affiliate of the University of Chicago.

The company, launched last year, is helmed by Nessan Bermingham, Ph.D., who three years back, handed the reins of gene editing biotech Intellia Therapeutics over to John Leonard, M.D., the R&D chief who had built the company alongside him.

After a break, he landed at Atlas Venture, the VC firm that helped launch Intellia in 2014 and that debuted Korro Bio, Berminghams latest project, last year.

Korros approach relies on nucleotide deamination, an endogenous RNA-modifying process that already happens in cells. Specifically, it takes advantage of a family of RNA-editing enzymes called adenosine deaminases acting on RNA (ADARs).

This group of enzymes deaminates the nucleotide adenosine to make inosine, which is read as guanosine (G) inside cells.

Korros ADAR approach doesnt permanently edit the genome by making a double-stranded cut in the DNA, like CRISPR systems do. With CRISPR-Cas9 systems, the delivery vehicle has to carry the actual gene-cutting mechanism. With Korros approach, that RNA-editing protein is already in the cell.

Korro is still not disclosing indications, but Duchenne muscular dystrophy and Parkinsons disease are two areas in which G-to-A mutations play a role.

This technology holds tremendous potential to usher in a new era of RNA editing therapies, said Bermingham.

We are leveraging a natural cellular system that has evolved over millions of years to base edit RNA. By co-opting these endogenous enzymes, we can create highly targeted, titratable and reversible therapeutics that are straightforward to design, manufacture and deliver. We are grateful for the continued support of our existing investors and look forward to working with our new investors to advance a new generation of transformational therapies to the clinic.

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Korro Bio hits the high notes with $91.5M series A to run its OPERA RNA platform - FierceBiotech

Though it is a member of the European Union, Finland has long avoided becoming entangled in the Wests military alliances, such as NATOa choice informed both by history and by the porous 830-mile-long border the country shares with Russia. This deliberate nonalignment has made the Finnish president someone pretty unique: a world leader whos well received in both Moscow and Washington.

Meanwhile, as the coronavirus continues to wreak havoc around the globe, theres good reason to fear that the next viral pandemic could be genetically engineered by humans, perhaps even using amateur tools purchased on Amazon.

And a checklist of all the ways U.S. President Donald Trump is maneuvering his way into autocracy, so you can keep track of his abuses in real time.

Here are Foreign Policys top weekend reads.

Much of the world has been captivated by Finlands young, female prime minister and her swift response to the coronavirus pandemic. But Finlands ceremonial president, Sauli Niinistoa cool-headed realistmay prove the more effective geopolitical asset, Gordon F. Sander writes.

Developed only a few years ago, CRISPR gene-editing technology is so user-friendly and accessible that scientific feats once plausible only in secretive government laboratories have quickly become kitchen table ubiquities. But this sort of democratization could doom us all, Vivek Wadhwa writes.

When Donald Trump was elected U.S. president in 2016, optimists suggested his authoritarian impulses would be reined in by the norms and seasoned Washingtonians around him. Four years later, these prognoses have proved pathetically naive, Stephen M. Walt writes.

The cool, blue Eastern Mediterranean has become heated with tension in recent months, as Greece, Turkey, and Cyprus vie for natural gas riches. The only way to prevent a full-scale conflict? Introduce a moratorium on drilling, which could save the peaceand, conveniently, the climate, tooPaul Hockenos writes.

Many of Chinas regional foes have grown comfortable with Trumps tough approach toward Chinese President Xi Jinping. Now, officials in capitals across Asia are privately voicing concern about the potential geostrategic consequences of a more Beijing-balanced Biden presidency, James Crabtree writes.

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Finland's President Can Hold His Own With Both Putin and Trump, and Other Top Weekend Reads - Foreign Policy

The Supreme Court has blocked the Nebraska Medical Bill Initiative from making it to the November ballots of 2020. This was done following a legal challenge filed by the Sheriff of Lancaster County, Nebraska, Terry Wagner.

Sheriff Terry Wagner had implied that the legalization proposal had violated the states single-subject rule required for ballot initiatives. Under this rule, any voter initiative or proposed legislative change cannot contain more than one focal point for voters approval.

According to attorneys of the plaintiff, the initiative is making provisions for a number of issues, which automatically disqualifies it from contesting for the ballots.

The initiative includes provisions on patient access, retail and distribution of medical marijuana, which expands the number of subjects to almost eight. This number is patently well beyond the legal limit implied by the single-subject rule of the Nebraskan legislation.

Although the state had rejected this opposition by Sheriff Terry Wagner, the Supreme Court has announced its verdict in favor of this dissent. If voters are to intelligently adopt a State policy with regard to medicinal cannabis use, they must first be allowed to decide that issue alone, unencumbered by other subjects, the justices wrote.

The Nebraska Medical Marijuana Initiative had proposed a number of constitutional amendments for the state.

Under the provisions, the purchase and use of medical marijuana usage for adults 18 years or older was to become legal. Patients below this age could still gain access to the drug by approval from licensed physicians or nurses. Legal guardians or parents of underage minor patients could also administer the drug to them after the physicians permission.

In addition to this, the bill had also proposed state legislatures to pass laws that would ensure safety of medical marijuana users. Laws for ensuring compliance with the legislation were also proposed to be devised by regulatory bodies.

Before getting challenged in the Supreme court, the bill had secured about 200,000 signatures for getting listed in the November ballots. This amount was well beyond the 121,699 signatures that were required to qualify it for the initiative.

It is important to note that these signatures were collected amidst the coronavirus pandemic. This signature drive included a truly impressive feat of grassroots mobilization. We would not have crossed the finish line without the tireless efforts of advocates, patients, families, volunteers, and hardworking Nebraskans who believe in this cause, said Senator Anna Wishart after resuming the signature collection drive which was halted temporarily due to the pandemic.

States like Oklahoma had to stop their legalization petitions midway due to a difficulty in gathering enough signatures. By crossing this huge obstruction, Nebraskans had surely proven their enthusiasm for the approval of the bill.

A marijuana advocacy group named Nebraskans for Medical Marijuana expressed disappointment at the removal of the initiative from the ballot.

Senator Adam Morfeld and Anna Wishart were also among those who were dismayed by the Supreme Courts verdict. But the duo still expressed commitment to their cause by stating a come back with new legislation and provisions.

Supporters of Nebraska Medical Bill Initiative argued that with this ruling of the Supreme Court, medical marijuana patients will suffer. They will be classified as criminals under current laws as a consequence of consuming marijuana to get relief from their respective ailments.

Governor Pete Ricketts has never shied away from expressing his opposition towards marijuana reforms.In January, he addressed Nebraskans in an article and detailed the drawbacks of the drug.

Listing the social costs of marijuana legalization, he had expressed concerns for the safety and well-being of the people. As Governor, I have a duty to promote public safety. I want Nebraskans to be informed of the dangers of marijuana and to know where I stand on the issue, he wrote.

Smart Approaches to Marijuana has their affiliate in Nebraska. The national anti-legalization group congratulated their counterpart on the Supreme Courts verdict. President of SAM, Kevin Sabet is in favor of a cannabis research bill calle HR 3797.

According to him, legalization of medical marijuana should wait before there is enough research to back its benefits. He believes that medicine should be kept away from politics and voting.

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Gene Editing Tool CRISPR Used To Modify Cannabis Seeds - Cannabis Health Insider

CRISPR-Cas9 has taken the world by storm with the promise of making gene editing much easier and faster than ever before. But how does CRISPR actually work? How can biology research benefit from it? What will happen when we start using it to edit human DNA? And whats the fight between its developers all about?

CRISPR-Cas9 is one of the biggest discoveries of the 21st century. Since it was developed in 2012, this gene editing tool has revolutionized biology research, making it easier to study disease and faster to discover drugs. The technology is also significantly impacting the development of crops, foods, and industrial fermentation processes.

But the one application that has made it famous is the modification of the human genome, which brings the promise of using CRISPR to cure disease. The first clinical trials testing CRISPR-Cas9 in people are already underway in China, Europe, and the US. So while scientists start venturing into tweaking our own DNA, it is worth taking the time to fully understand what CRISPR is, and what the actual benefits and risks of using the technology are.

CRISPR is short for clustered regularly interspaced short palindromic repeats. The term makes reference to a series of repetitive patterns in the DNA of bacteria and archaea that were discovered by Spanish scientist Francis Mojica in the 90s.

These patterns are the basis of a primitive immune system that bacteria use to remember the DNA of viral invaders by incorporating the DNA sequence of the virus within the CRISPR patterns. The Cas9 protein is then able to recognize the DNA sequence stored within CRISPR patterns and cut any DNA molecules with a matching sequence.

But it wasnt until 2012 that Jennifer Doudna and Emmanuelle Charpentier at the University of California, Berkeley in the US took the discovery a step further. They published a scientific paper showing what happened when the CRISPR-Cas9 system was taken out of bacteria and introduced in eukaryotic cells the ones that make up plants or animals.

When you cut the DNA of a bacterium, you kill it. But in eukaryotes, when you cut DNA you activate a repair mechanism that opens the possibility to rewrite DNA, Mojica told me. Jennifer and Emmanuelle did it in vitro and it worked wonderfully.

Another two papers published just a few months later by Feng Zhang and George Church from the Broad Institute also reported some early uses of CRISPR as a gene editing tool.

It is important to note that CRISPR is by far not the first system that allows us to edit DNA in all sorts of organisms. Other technologies used extensively before are TALEN and zinc-finger nucleases (ZFNs). In fact, some experts point out that these tools, which have been in use enough time to become quite refined, are more accurate than CRISPR-Cas9.

But CRISPR brings an important advantage over these other techniques: it is much easier and faster to use. Most previous technologies required creating a gene editing protein from scratch for each specific DNA modification. With CRISPR, the same Cas9 molecule can be directed to any sequence just by providing it with a guide RNA molecule, which is much easier to synthesize. Companies like Synthego in the US have spotted a good business opportunity producing these guide molecules for researchers.

In theory, CRISPR gene editing could be used to make any modification to the DNA of virtually any living being. In biotech and pharma companies, CRISPR is becoming the go-to tool for drug discovery. In academic research labs, the gene editing tool is being used to modify the genome of all sorts of organisms to study the function of any gene of interest, whether it is one that causes disease or one that makes a crop grow faster or survive harsh conditions.

In agriculture, CRISPR could be used to produce crops with better yields or that resist drought, much faster than is possible with traditional breeding techniques. It can also be used to add new features, such as making tomatoes spicy, or remove others for example making gluten-free wheat or decaf coffee beans.

However, regulations can limit the use of these technologies. While the US has already seen the launch of CRISPR-modified crops, the European Union decided to set strict GMO regulations that scientists believe are hindering the potential of the technology.

But right now, most of the money seems to be in using CRISPR-Cas9 to engineer human DNA. With over 10,000 diseases caused by mutations in a single human gene, CRISPR offers hope to cure all of them by repairing any genetic error behind them.

There are two main approaches to using CRISPR as a therapy. The first is called ex vivo gene editing. It involves extracting human cells, engineering them in the lab, and reinjecting them into the patient. This method is similar to that used for most gene therapies already on the market. However, it can become quite expensive given each patient requires an individual manufacturing process for their therapy.

The second method is called in vivo gene editing and involves delivering CRISPR-Cas9 into the patients body to edit the DNA directly from within the cells. CRISPR could be delivered inside nanoparticles or encoded into DNA and be cleared out of the body once it has completed its mission. This method has only started human testing in 2020, and there are some concerns that there is a risk of CRISPR making off-target modifications.

Those are the big questions right now. Especially after CRISPR gene editing was controversially used to create the worlds first gene-edited babies in 2018. These CRISPR babies carry a mutation intended to protect them against HIV infection. The experiment resulted in a strong pushback as scientists around the world questioned the ethics of altering human DNA without fully understanding the possible consequences. Indeed, there was a study suggesting that people carrying these mutations might be at risk of catching certain infections and dying younger.

Despite all the controversy around CRISPR therapy and the large amounts of money invested in it, we are still in the very early stages of clinical trials. Scientists are wary of repeating the same mistakes as when gene therapy was first tested in humans back in the 90s, resulting in the death of 18-year old Jesse Gelsinger and causing years of delay in the development of gene therapy.

Now, even if CRISPR proves to be safe in humans, is it ethical to modify the human genome? The first applications of the technology, aimed at curing genetic diseases, seem quite straightforward. But where should the line be drawn? At what point does a therapy become a tool for eugenics?

Although the point in time when we are able to modify all sorts of human features at will is far ahead in the future, it is never too early to start thinking about how the technology should be regulated. Many scientists, including Jennifer Doudna, seem to agree that we should stay away from germline editing that is, any modifications that children will inherit. At least for now.

Since the first publications showcasing CRISPR-Cas9 as a gene editing tool back in 2012, a number of companies have been set up by the developers of the technology. Based in Switzerland and the US, there is CRISPR Therapeutics, co-founded by Emmanuelle Charpentier. Working in partnership with Vertex Pharmaceuticals, the company already has preliminary results showing promise for the treatment of the blood disorders -thalassemia and sickle cell disease. The company is using an ex vivo approach where the bone marrow stem cells of the patient are genetically engineered outside the body.

In the US, the first CRISPR clinical trial started in early 2019, run by scientists at the University of Pennsylvania. On top of academic efforts, the US counts also with the firm Intellia Therapeutics, co-founded by Jennifer Doudna; its first target will be an in vivo treatment for a rare neurological disease called transthyretin amyloidosis.

Founded by Doudna and Charpentiers competitor Feng Zhang, there is also Editas Medicine, working in therapies for genetic blindness and cancer, among others. Doudna originally co-founded Editas along with Zhang, but stopped all involvement with it just a few weeks after Zhang was granted his CRISPR patent and issues concerning intellectual property began to appear.

The intellectual property in this space is pretty complex, to put it nicely, said Rodger Novak, co-founder and previous CEO of CRISPR Therapeutics. Everyone knows there are conflicting claims.

The team of Doudna and Charpentier at UC Berkeley filed a first patent application for CRISPR in May 2012, a few months before their paper was published. Zhang and the Broad Institute filed theirs in December that year, but they paid the US patent office to fast-track the review process. This resulted in Zhangs patents being issued before there was a decision on his competitors.

UC Berkeley then initiated a process to invalidate the Broads patent on the basis that Doudna and Charpentier had developed the technology and applied for a CRISPR patent earlier. The US patent office ended up ruling in favor of the Broad Institute, after both parties combined had already spent over $20M (16M) in legal fees.

It reminds me of reading about really unhappy rich people, said George Church about the patent fight. They have such a big blank check that they just make each other miserable.

Everything here is very exaggerated because this is one of those unique cases of a technology that people can really pick up easily, and its changing researchers lives. Things are happening fast, maybe a bit too fast, commented Charpentier. I am very confident that the future will clarify the situation. And I would like to believe the story is going to end up well.

Indeed, the situation is quite favorable for Charpentier and Doudna across the Atlantic. In Europe, they have secured broad patents on CRISPR while the Broad Institute saw its patents revoked earlier this year. While the Broad can still enforce its intellectual property in Europe with narrower claims, its licensing and royalty fees may be lower.

The situation is paralyzing small companies. They are afraid of being held liable for patent infringement so theyd rather not use the technology, said Ulrich Storz, Senior Partner at Michalski Htterman Patent Attorneys. This situation is not very common in biotech. We werent prepared, and thats why there have been so many problems with this technology, with this patent challenge.

With its potential already demonstrated in research applications, the next big milestone for CRISPR will be proving to be safe and effective as a treatment. But there are still many other applications underway.

For example, the US company eGenesis, co-founded by George Church, is using CRISPR to modify the pig genome so that their organs can be transplanted into humans without rejection. Another is the use of CRISPR as a diagnostics tool; some researchers have already developed a method to detect Covid-19 using CRISPR.

CRISPR might still surprise us as new variants are developed. Swiss scientists have developed a method to simultaneously edit up to 25 genes using CRISPR. And another version of the gene editing tool called CRISPR-Cpf1 that makes it easier to replace one DNA sequence by another is already being used by big brands such as BASF. You can imagine that many labs including our own are busily looking at other variants and how they work, Doudna said. So stay tuned.

The gene editing tool is also becoming very popular between DIY scientists and biohackers. Some believe that the relatively simple methods that this technique requires might help democratize science and bring it closer to people outside the lab. However, some cases of biohackers injecting themselves with experimental treatments have alarmed the public and it remains to be seen how these uses will be regulated.

In any case, the impact of CRISPR in biology is already tangible and will undoubtedly go down in history as a big discovery. The cherry on the cake will come when the technology wins the Nobel prize, which many have been unsuccessfully predicting will go to CRISPR for years.

It is possible they are waiting for CRISPR to demonstrate all the potential that is expected, but it would be unfair, CRISPR discoverer Francis Mojica told me. What CRISPR has already achieved is much more than what other tools that have received the Nobel have achieved. The prize has gone to tools used to cut and copy DNA in the test tube. CRISPR can be used to edit genomes, change expression levels, visualize DNA, kill bacteria, develop diagnostics, and many more applications, even to store a movie within DNA.

I am convinced it will get it. When? I dont know.

Images via Soleil Nordic /Shutterstock; The Conversation; Knaw /Flickr CC2.0; Science

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CRISPR-Cas9: The Gene Editing Tool Changing the... - Labiotech.eu

1. 'CRISPR babies' are still too risky, says influential panel  Nature.com
2. Panel Lays Out Guidelines for CRISPR-Edited Human Embryos  The Scientist
3. Panel Lays Out Gene Editing Guidelines, Condemns Risk of Creating 'CRISPR' Babies  BioSpace
4. Closely-watched international CRISPR ethics panel leaves door ajar for germline editing one day  Endpoints News
5. CRISPR: Still too soon to try altering human embryo DNA, experts say  Hindustan Times
6. View Full Coverage on Google News

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'CRISPR babies' are still too risky, says influential panel - Nature.com

ZUG, Switzerland and CAMBRIDGE, Mass., Sept. 03, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, is scheduled to present at the Wells Fargo 2020 Virtual Healthcare Conference on Thursday, September 10, 2020, at 11:20 a.m. ET.

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

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

Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

Media Contact:Rachel Eides WCG on behalf of CRISPR+1-617-337-4167reides@wcgworld.com

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CRISPR Therapeutics to Present at the Wells Fargo 2020 Virtual Healthcare Conference - Yahoo Finance

Global CRISPR Technology industry report about In-depth Research, estimates Revenue, and forecasts Growth Details in segments, regional, and research scope, historical data, Key Player and Growth Value.

The Global CRISPR Technology Market 2020 analysis provides a basic summary of the trade as well as definitions, classifications, applications and business chain structure. The worldwide CRISPR Technology marketing research is provided for the international markets together with development trends, competitive landscape analysis, and key regions development standing. Development policies and plans are mentioned similarly as producing processes and value structures are analyzed. This report additionally states import/export consumption, supply and demand Figures, cost, price, revenue, and gross margins.

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Major Classifications of CRISPR Technology Market:

Major Key players covered in this report:

By Product Type:

By Applications:

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Impact of COVID-19:CRISPR Technology Market report analyses the impact of Coronavirus (COVID-19) on the CRISPR Technology industry. Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the CRISPR Technology market in 2020.

The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; emergency declared in many countries; massive slowing of the supply chain; stock market unpredictability; falling business assurance, growing panic among the population, and uncertainty about future.

COVID-19 can affect the global economy in 3 main ways: by directly affecting production and demand, by creating supply chain and market disturbance, and by its financial impact on firms and financial markets.

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This Market Study covers the CRISPR Technology Market Size across segments. It aims at estimating the market size and the growth potential of the market across segments by component, data type, deployment type, organization size, vertical, and region. This CRISPR Technology study also includes an in-depth competitive analysis of the key market players, along with their company profiles, key observations related to product and business offerings, recent developments, and key market strategies.

Attributes such as new development in CRISPR Technology market, Total Revenue, sales, annual production, government norm, and trade barriers in some countries are also mentioned in detail in the report. CRISPR Technology Report discusses about recent product innovations and gives an overview of potential regional market shares.

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CRISPR Technology Market 2020: Potential Growth, Challenges, and Know the Companies List Could Potentially Benefit or Loose out From the Impact of...

The report offers a far-reaching analysis of CRISPR And CRISPR-Associated (Cas) Genes industry, standing on the readers perspective, conveying definite market information, and adding valuable & knowledgeable data. It surveys the effect of the mechanical advancements, changes in speculation propensities, and top to the bottom outline of Product Specification.

Competitive Landscape:Geographically, the global CRISPR And CRISPR-Associated (Cas) Genes market is analysed by top manufacturers (production, price, revenue (value), and market share). The competitive analysis of leading market players is another notable feature of this report; it identifies direct or indirect competitors in the market are Caribou Biosciences, Addgene, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, CRISPR THERAPEUTICS, GE Healthcare Dharmacon

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In the primary research process, various sources from both the supply and demand sides were interviewed to obtain qualitative and quantitative information for this CRISPR And CRISPR-Associated (Cas) Genes Market report. The primary sources from the supply side include product manufacturers (and their competitors), opinion leaders, industry experts, research institutions, distributors, dealers, and traders, as well as the raw materials suppliers, and producers, etc.

The primary sources from the demand side include CRISPR And CRISPR-Associated (Cas) Genes industry experts such as business leaders, marketing and sales directors, technology and innovation directors, supply chain executive, End-User (product buyers), and related key executives from various key companies and organizations operating in the global CRISPR And CRISPR-Associated (Cas) Genes market.

Primary Types of the industry are Type 1, Type 2

Primary Applications of the industry are Application 1, Application 2

This report is based on the synthesis, analysis, and interpretation of information collected on the CRISPR And CRISPR-Associated (Cas) Genes market from various sources. Our analysts have analysed the information & data and gained insights using a mix of primary and secondary research efforts with the primary objective to provide a holistic view of the CRISPR And CRISPR-Associated (Cas) Genes Industry.

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The following market parameters were considered to estimate market value:

Market Overview

The report includes overviews market introduction, market drivers & influencing factors, restraints & challenges, and potential growth opportunities of CRISPR And CRISPR-Associated (Cas) Genes market. The report consists of market evaluation tools such as Porters five forces, PESTLE Analysis, and value chain analysis.

Table of Content

Chapter 1 About the CRISPR And CRISPR-Associated (Cas) Genes Industry1.1 Industry Definition and Types1.2 Main Market Activities1.3 Similar Industries1.4 Industry at a Glance

Chapter 2 World Market Competition Landscape2.1 CRISPR And CRISPR-Associated (Cas) Genes Markets by Regions2.1.1 USAMarket Revenue (M USD) and Growth Rate 2014-2019Sales and Growth Rate 2014-2019Major Players2.1.2 EuropeMarket Revenue (M USD) and Growth Rate 2014-2019Sales and Growth Rate 2014-2019Major Players2.1.3 ChinaMarket Revenue (M USD) and Growth Rate 2014-2019Sales and Growth Rate 2014-2019Major Players2.1.4 IndiaMarket Revenue (M USD) and Growth Rate 2014-2019Sales and Growth Rate 2014-2019Major Players2.1.5 JapanMarket Revenue (M USD) and Growth Rate 2014-2019Sales and Growth Rate 2014-2019Major Players2.1.6 South East AsiaMarket Revenue (M USD) and Growth Rate 2014-2019Sales and Growth Rate 2014-2019Major Players2.2 World CRISPR And CRISPR-Associated (Cas) Genes Market by Types2.3 World CRISPR And CRISPR-Associated (Cas) Genes Market by Applications2.4 World CRISPR And CRISPR-Associated (Cas) Genes Market Analysis2.4.1 World CRISPR And CRISPR-Associated (Cas) Genes Market Revenue and Growth Rate 2014-20192.4.2 World CRISPR And CRISPR-Associated (Cas) Genes Market Consumption and Growth rate 2014-20192.4.3 World CRISPR And CRISPR-Associated (Cas) Genes Market Price Analysis 2014-2019

Chapter 3 World CRISPR And CRISPR-Associated (Cas) Genes Market share3.1 Major Production Market share by Players3.2 Major Revenue (M USD) Market share by Players3.3 Major Production Market share by Regions in 2019, Through 20243.4 Major Revenue (M USD) Market share By Regions in 2019, Through 2024

Chapter 4 Supply Chain Analysis4.1 Industry Supply chain Analysis4.2 Raw material Market Analysis4.2.1 Raw material Prices Analysis 2014-20194.2.2 Raw material Supply Market Analysis4.2 Manufacturing Equipment Suppliers Analysis4.3 Production Process Analysis4.4 Production Cost Structure Benchmarks4.5 End users Market Analysis

Chapter 5 Company Profiles5.1 Caribou Biosciences5.1.1 Company Details (Foundation Year, Employee Strength and etc)5.1.2 Product Information (Picture, Specifications and Applications)5.1.3 Revenue (M USD), Price and Operating Profits5.2 Addgene5.2.1 Company Details (Foundation Year, Employee Strength and etc)5.2.2 Product Information (Picture, Specifications and Applications)5.2.3 Revenue (M USD), Price and Operating Profits5.3 Merck KGaA5.3.1 Company Details (Foundation Year, Employee Strength and etc)5.3.2 Product Information (Picture, Specifications and Applications)5.3.3 Revenue (M USD), Price and Operating Profits5.4 Mirus Bio LLC5.4.1 Company Details (Foundation Year, Employee Strength and etc)5.4.2 Product Information (Picture, Specifications and Applications)5.4.3 Revenue (M USD), Price and Operating Profits5.5 Editas Medicine5.5.1 Company Details (Foundation Year, Employee Strength and etc)5.5.2 Product Information (Picture, Specifications and Applications)5.5.3 Revenue (M USD), Price and Operating Profits5.6 Takara Bio USA5.6.1 Company Details (Foundation Year, Employee Strength and etc)5.6.2 Product Information (Picture, Specifications and Applications)5.6.3 Revenue (M USD), Price and Operating Profits5.7 Thermo Fisher Scientific5.7.1 Company Details (Foundation Year, Employee Strength and etc)5.7.2 Product Information (Picture, Specifications and Applications)5.7.3 Revenue (M USD), Price and Operating Profits5.8 Horizon Discovery Group5.8.1 Company Details (Foundation Year, Employee Strength and etc)5.8.2 Product Information (Picture, Specifications and Applications)5.8.3 Revenue (M USD), Price and Operating Profits5.9 Intellia Therapeutics5.9.1 Company Details (Foundation Year, Employee Strength and etc)5.9.2 Product Information (Picture, Specifications and Applications)5.9.3 Revenue (M USD), Price and Operating Profits5.10 CRISPR THERAPEUTICS5.10.1 Company Details (Foundation Year, Employee Strength and etc)5.10.2 Product Information (Picture, Specifications and Applications)5.10.3 Revenue (M USD), Price and Operating Profits5.11 GE Healthcare Dharmacon5.11.1 Company Details (Foundation Year, Employee Strength and etc)5.11.2 Product Information (Picture, Specifications and Applications)5.11.3 Revenue (M USD), Price and Operating Profits

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CRISPR And CRISPR-Associated (Cas) Genes Market Analysis Focusing on the Key Issues Surrounding the Growth of the Industry and Further Develop...

Overall market sentiment has been down on Crispr Therapeutics AG (CRSP) stock lately. CRSP receives a Bearish rating from InvestorsObserver's Stock Sentiment Indicator.

Sentiment uses short term technical analysis to gauge whether a stock is desired by investors. As a technical indicator, it focuses on recent trends as opposed to the long term health of the underlying company. Updates for the company such as a earnings release can move the stock away from current trends.

Changes in price are generally the best indicator of sentiment for a particular stock. At its core, a stock's trend indicates whether current market sentiment is bullish or bearish. Investors must be bullish if a stock is trending upward, and are bearish if a stock is moving down.

InvestorsObserver's Sentiment Indicator factors in both price changes and variations in volume. An increase in volume usually means a current trend is stengthening, while a drop in volume tends to signal a reversal to the ongoing trend.

Our system also uses the options market in order to receive additional signals on current sentiments. We take into account the ratio of calls and puts for a stock since options allow an investor to bet on future changes in price.

Crispr Therapeutics AG (CRSP) stock is trading at $83.11 as of 3:27 PM on Friday, Sep 4, a drop of -$2.58, or -3.01% from the previous closing price of $85.69. The stock has traded between $76.71 and $87.00 so far today. Volume today is elevated. So far 1,357,043 shares have traded compared to average volume of 880,219 shares.

To screen for more stocks like Crispr Therapeutics AG click here.

CRISPR Therapeutics AG is a gene-editing company. It is engaged in the development of CRISPR/Cas9-based therapeutics. CRISPR/Cas9 is a technology that allows for precise, directed changes to genomic DNA. The company advanced programs target beta-thalassemia and sickle cell disease, two hemoglobinopathies that have a high unmet medical need.

Click Here to get the full Stock Score Report on Crispr Therapeutics AG (CRSP) Stock.

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Is it Time to Dump Crispr Therapeutics AG (CRSP) Stock After it Is Lower By 8.38% in a Week? - InvestorsObserver

Cell Theraputics Market

Los Angeles, United State, ,-The research study presented here is a brilliant compilation of different types of analysis of critical aspects of the global Cell Theraputics market. It sheds light on how the global Cell Theraputics market is expected to grow during the course of the forecast period. With SWOT analysis and Porters Five Forces analysis, it gives a deep explanation of the strengths and weaknesses of the global Cell Theraputics market and different players operating therein. The authors of the report have also provided qualitative and quantitative analyses of several microeconomic and macroeconomic factors impacting the global Cell Theraputics market. In addition, the research study helps to understand the changes in the industry supply chain, manufacturing process and cost, sales scenarios, and dynamics of the global Cell Theraputics market.

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Each player studied in the report is profiled while taking into account its production, market value, sales, gross margin, market share, recent developments, and marketing and business strategies. Besides giving a broad study of the drivers, restraints, trends, and opportunities of the global Cell Theraputics market, the report offers an individual, detailed analysis of important regions such as North America, Europe, and Asia Pacific. Furthermore, important segments of the global Cell Theraputics market are studied in great detail with key focus on their market share, CAGR, and other vital factors.

Key Players:

Bristol-Myers Squibb Company (Celgene), Bluebird Bio, Crispr Therapeutics, Roche (Spark Therapeutics), PTC Therapeutics, Moderna Therapeutics, Quanterix, Brainstorm Cell Therapeutics Inc., Lineage Cell Therapeutics, Cti Biopharma, Atara Biotherapeutics, Inc, Adaptimmune

Type Segments:

Stem Cells, Immunocyte

Application Segments:

Allogeneic Cell Therapy, Autologous Cell Therapy, Xenogeneic Cell Therapy

Regional Segments

Table of Contents

1 Market Overview of Cell Theraputics1.1 Cell Theraputics Market Overview1.1.1 Cell Theraputics Product Scope1.1.2 Market Status and Outlook1.2 Global Cell Theraputics Market Size Overview by Region 2015 VS 2020 VS 20261.3 Global Cell Theraputics Market Size by Region (2015-2026)1.4 Global Cell Theraputics Historic Market Size by Region (2015-2020)1.5 Global Cell Theraputics Market Size Forecast by Region (2021-2026)1.6 Key Regions Cell Theraputics Market Size YoY Growth (2015-2026)1.6.1 North America Cell Theraputics Market Size YoY Growth (2015-2026)1.6.2 Europe Cell Theraputics Market Size YoY Growth (2015-2026)1.6.3 China Cell Theraputics Market Size YoY Growth (2015-2026)1.6.4 Rest of Asia Pacific Cell Theraputics Market Size YoY Growth (2015-2026)1.6.5 Latin America Cell Theraputics Market Size YoY Growth (2015-2026)1.6.6 Middle East & Africa Cell Theraputics Market Size YoY Growth (2015-2026)1.7 Coronavirus Disease 2019 (Covid-19): Cell Theraputics Industry Impact1.7.1 How the Covid-19 is Affecting the Cell Theraputics Industry

1.7.1.1 Cell Theraputics Business Impact Assessment Covid-19

1.7.1.2 Supply Chain Challenges

1.7.1.3 COVID-19s Impact On Crude Oil and Refined Products1.7.2 Market Trends and Cell Theraputics Potential Opportunities in the COVID-19 Landscape1.7.3 Measures / Proposal against Covid-19

1.7.3.1 Government Measures to Combat Covid-19 Impact

1.7.3.2 Proposal for Cell Theraputics Players to Combat Covid-19 Impact 2 Cell Theraputics Market Overview by Type2.1 Global Cell Theraputics Market Size by Type: 2015 VS 2020 VS 20262.2 Global Cell Theraputics Historic Market Size by Type (2015-2020)2.3 Global Cell Theraputics Forecasted Market Size by Type (2021-2026)2.4 Stem Cells2.5 Immunocyte 3 Cell Theraputics Market Overview by Type3.1 Global Cell Theraputics Market Size by Application: 2015 VS 2020 VS 20263.2 Global Cell Theraputics Historic Market Size by Application (2015-2020)3.3 Global Cell Theraputics Forecasted Market Size by Application (2021-2026)3.4 Allogeneic Cell Therapy3.5 Autologous Cell Therapy3.6 Xenogeneic Cell Therapy 4 Global Cell Theraputics Competition Analysis by Players4.1 Global Cell Theraputics Market Size (Million US$) by Players (2015-2020)4.2 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in Cell Theraputics as of 2019)4.3 Date of Key Manufacturers Enter into Cell Theraputics Market4.4 Global Top Players Cell Theraputics Headquarters and Area Served4.5 Key Players Cell Theraputics Product Solution and Service4.6 Competitive Status4.6.1 Cell Theraputics Market Concentration Rate4.6.2 Mergers & Acquisitions, Expansion Plans 5 Company (Top Players) Profiles and Key Data5.1 Bristol-Myers Squibb Company (Celgene)5.1.1 Bristol-Myers Squibb Company (Celgene) Profile5.1.2 Bristol-Myers Squibb Company (Celgene) Main Business and Companys Total Revenue5.1.3 Bristol-Myers Squibb Company (Celgene) Products, Services and Solutions5.1.4 Bristol-Myers Squibb Company (Celgene) Revenue (US$ Million) (2015-2020)5.1.5 Bristol-Myers Squibb Company (Celgene) Recent Developments5.2 Bluebird Bio5.2.1 Bluebird Bio Profile5.2.2 Bluebird Bio Main Business and Companys Total Revenue5.2.3 Bluebird Bio Products, Services and Solutions5.2.4 Bluebird Bio Revenue (US$ Million) (2015-2020)5.2.5 Bluebird Bio Recent Developments5.3 Crispr Therapeutics5.5.1 Crispr Therapeutics Profile5.3.2 Crispr Therapeutics Main Business and Companys Total Revenue5.3.3 Crispr Therapeutics Products, Services and Solutions5.3.4 Crispr Therapeutics Revenue (US$ Million) (2015-2020)5.3.5 Roche (Spark Therapeutics) Recent Developments5.4 Roche (Spark Therapeutics)5.4.1 Roche (Spark Therapeutics) Profile5.4.2 Roche (Spark Therapeutics) Main Business and Companys Total Revenue5.4.3 Roche (Spark Therapeutics) Products, Services and Solutions5.4.4 Roche (Spark Therapeutics) Revenue (US$ Million) (2015-2020)5.4.5 Roche (Spark Therapeutics) Recent Developments5.5 PTC Therapeutics5.5.1 PTC Therapeutics Profile5.5.2 PTC Therapeutics Main Business and Companys Total Revenue5.5.3 PTC Therapeutics Products, Services and Solutions5.5.4 PTC Therapeutics Revenue (US$ Million) (2015-2020)5.5.5 PTC Therapeutics Recent Developments5.6 Moderna Therapeutics5.6.1 Moderna Therapeutics Profile5.6.2 Moderna Therapeutics Main Business and Companys Total Revenue5.6.3 Moderna Therapeutics Products, Services and Solutions5.6.4 Moderna Therapeutics Revenue (US$ Million) (2015-2020)5.6.5 Moderna Therapeutics Recent Developments5.7 Quanterix5.7.1 Quanterix Profile5.7.2 Quanterix Main Business and Companys Total Revenue5.7.3 Quanterix Products, Services and Solutions5.7.4 Quanterix Revenue (US$ Million) (2015-2020)5.7.5 Quanterix Recent Developments5.8 Brainstorm Cell Therapeutics Inc.5.8.1 Brainstorm Cell Therapeutics Inc. Profile5.8.2 Brainstorm Cell Therapeutics Inc. Main Business and Companys Total Revenue5.8.3 Brainstorm Cell Therapeutics Inc. Products, Services and Solutions5.8.4 Brainstorm Cell Therapeutics Inc. Revenue (US$ Million) (2015-2020)5.8.5 Brainstorm Cell Therapeutics Inc. Recent Developments5.9 Lineage Cell Therapeutics5.9.1 Lineage Cell Therapeutics Profile5.9.2 Lineage Cell Therapeutics Main Business and Companys Total Revenue5.9.3 Lineage Cell Therapeutics Products, Services and Solutions5.9.4 Lineage Cell Therapeutics Revenue (US$ Million) (2015-2020)5.9.5 Lineage Cell Therapeutics Recent Developments5.10 Cti Biopharma5.10.1 Cti Biopharma Profile5.10.2 Cti Biopharma Main Business and Companys Total Revenue5.10.3 Cti Biopharma Products, Services and Solutions5.10.4 Cti Biopharma Revenue (US$ Million) (2015-2020)5.10.5 Cti Biopharma Recent Developments5.11 Atara Biotherapeutics, Inc5.11.1 Atara Biotherapeutics, Inc Profile5.11.2 Atara Biotherapeutics, Inc Main Business and Companys Total Revenue5.11.3 Atara Biotherapeutics, Inc Products, Services and Solutions5.11.4 Atara Biotherapeutics, Inc Revenue (US$ Million) (2015-2020)5.11.5 Atara Biotherapeutics, Inc Recent Developments5.12 Adaptimmune5.12.1 Adaptimmune Profile5.12.2 Adaptimmune Main Business and Companys Total Revenue5.12.3 Adaptimmune Products, Services and Solutions5.12.4 Adaptimmune Revenue (US$ Million) (2015-2020)5.12.5 Adaptimmune Recent Developments 6 North America Cell Theraputics by Players and by Application6.1 North America Cell Theraputics Market Size and Market Share by Players (2015-2020)6.2 North America Cell Theraputics Market Size by Application (2015-2020) 7 Europe Cell Theraputics by Players and by Application7.1 Europe Cell Theraputics Market Size and Market Share by Players (2015-2020)7.2 Europe Cell Theraputics Market Size by Application (2015-2020) 8 China Cell Theraputics by Players and by Application8.1 China Cell Theraputics Market Size and Market Share by Players (2015-2020)8.2 China Cell Theraputics Market Size by Application (2015-2020) 9 Rest of Asia Pacific Cell Theraputics by Players and by Application9.1 Rest of Asia Pacific Cell Theraputics Market Size and Market Share by Players (2015-2020)9.2 Rest of Asia Pacific Cell Theraputics Market Size by Application (2015-2020) 10 Latin America Cell Theraputics by Players and by Application10.1 Latin America Cell Theraputics Market Size and Market Share by Players (2015-2020)10.2 Latin America Cell Theraputics Market Size by Application (2015-2020) 11 Middle East & Africa Cell Theraputics by Players and by Application11.1 Middle East & Africa Cell Theraputics Market Size and Market Share by Players (2015-2020)11.2 Middle East & Africa Cell Theraputics Market Size by Application (2015-2020) 12 Cell Theraputics Market Dynamics12.1 Industry Trends12.2 Market Drivers12.3 Market Challenges12.4 Porters Five Forces Analysis 13 Research Finding /Conclusion 14 Methodology and Data Source 14.1 Methodology/Research Approach14.1.1 Research Programs/Design14.1.2 Market Size Estimation14.1.3 Market Breakdown and Data Triangulation14.2 Data Source14.2.1 Secondary Sources14.2.2 Primary Sources14.3 Disclaimer14.4 Author List

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Cell Theraputics Market Status, Players, Types, Applications, and Forecast 2020-2026-Crispr Therapeutics, Roche (Spark Therapeutics), PTC Therapeutics...

Bloomberg

(Bloomberg) -- As Covid-19 cases surge on U.S. campuses, a New York state university sent students home for the semester and Indiana University warned of uncontrolled spread at fraternities and sororities.Australias hotspot Victoria state recorded 81 new virus cases overnight, after warning against easing lockdown measures too quickly.Brazil passed 4 million cases. Globally, the Latin American nation now lags only the U.S. in both cases and deaths from the disease.Key Developments:Global Tracker: Cases surpass 26 million; deaths exceed 866,000Mexico sees more than 500 new deaths from coronavirusChina reports 25 new cases, says all are importedDozens of new infections in Australias Victoria stateBrazil passes 4 million cases; now trails only U.S.Frontrunning Covid vaccines will soon have their moment of truthVaccine tracker: Where are we in the race for protection?Subscribe to a daily update on the virus from Bloombergs Prognosis team here. Click CVID on the terminal for global data on coronavirus cases and deaths.Mexico Reports Hundreds More Deaths (8:12 a.m.)The hard-hit country reported 5,937 more virus cases -- bringing its total to 616,894 -- according to data released by the Health Ministry Thursday night. There were 513 new deaths.Outbreak Continues in North and South Dakota (7:10 a.m. HK)The surge of cases in North and South Dakota showed no sign of easing, as each state reported a daily increase of cases with numbers significantly higher than two months ago.North Dakota reported 360 cases, up from 267 the day before and the second highest on record. South Dakota reported 334 cases, up from 254 the day before and more than triple the worst day in July.South Dakota reported Thursday that 118 people in the state were infected because of the Sturgis motorcycle rally Aug. 7-16, the Rapid City Journal reported. They are among several hundred cases reported in about a dozen other states. On Wednesday, Minnesota reported the first virus death from a person who attended the rally.Brazil Passes Four Million Cases (6:10 a.m. HK)Brazil reached the mark of 4 million confirmed coronavirus cases, doubling the infection count in two months as large parts of the Latin American nation emerge from isolation.The country reported 43,773 new cases on Thursday, pushing the toll to 4,041,638. Deaths rose by 834 to 124,614, according to data from the Health Ministry.Indiana University Raises Warning on Frats (5:20 p.m. NY)Indiana University urged students at fraternities and sororities to re-evaluate living there after outbreaks in Greek housing showed positive-test rates as high as 87%.IUs team of public health experts is extremely concerned that Greek houses are seeing uncontrolled spread of Covid-19, the university said in a statement. This poses a significant risk to the nearly 2,600 students currently living in Greek or other communal housing organizations, as well as to the other 42,000 IU Bloomington students.Fraternities and sororities are privately owned and the university does not have the authority to close them, the statement said.Texas Double Counted Inmate Cases as Hurricane Loomed (5:14 p.m. NY)Texas double counted almost 300 prisoners with the virus after they were transferred to another county as Hurricane Laura approached last week.When the 281 inmates were shuttled 140 miles (225 kilometers) inland from coastal Jefferson County to Walker County, they were entered into the Texas database as new Walker County diagnoses, the state health department said in a footnote on its website on Thursday. They have since been deleted from Walker Countys tally.Statewide, Texas added 3,899 new cases on Thursday, bringing the cumulative total to 625,347, health department data showed. Hospitalizations continued their downward trend, dipping to 4,075, the lowest since June 22.Low Chance of Vaccine by November: U.S. Official (4:33 p.m. NY)Authorization of a Covid-19 vaccine by Nov. 1 when U.S. health officials have told states to be prepared to distribute shots is extremely unlikely but not impossible, Moncef Slaoui, chief scientific adviser to Operation Warp Speed told NPRs All Things Considered on Thursday.There is a very, very low chance that the trials that are running as we speak could read before the end of October, Slaoui said, referring to when data on a vaccines safety and effectiveness could be available.He also said there will likely be about 15 million to 20 million vaccine doses available by the end of the year and enough to immunize the U.S. population by the middle of 2021.U.S. Cases Rise 0.7% (4 p.m. NY)Coronavirus cases in the U.S. increased 0.7% as compared with the same time Wednesday to 6.13 million, according to data collected by Johns Hopkins University and Bloomberg News. The increase matched the average daily gain over the past week. Deaths rose by 0.8% to 186,293.Florida reported 637,013 cases, up 0.6% from a day earlier, in line with the average increase in the previous seven days. Deaths reached 11,650, an increase of 149, or 1.3%.Arizona reported 1,091 new cases, the biggest one-day tally since Aug. 13. The 0.5% spike, bringing the states total cases to 203,953. Deaths rose by 65 for a total of 5,130.Hawaii experienced a 3.9% increase in the number of cases from the same time yesterday, bringing the total to 8,991, according to the Johns Hopkins and Bloomberg News data.SUNY Oneonta Sends Students Home (2:30 p.m. NY)The State University of New York at Oneonta plans to send on-campus students home and cease all in-person classes for the rest of the fall semester after a Covid-19 outbreak. The college in upstate New York had 389 confirmed cases since the start of the semester Aug. 24.The college had begun a two-week pause period on Aug. 30 in order to focus on testing while limiting the spread of COVID-19.California Cases Up; Hospitalizations Drop (2:30 p.m. NY)California reported 5,125 new cases, a 0.7% increase and roughly in line with its 14-day average. The state recorded 164 new deaths, exceeding the two-week average of 117, for a total of 13,327 virus fatalities.Still, there were more signs of improvement in the states outbreak, with hospitalizations dropping 4.5% to 3,604 patients, the lowest in almost 11 weeks.French Cases Continue to Surge (1:30 p.m. NY)France registered 7,157 new cases over 24 hours, the third time in a week that its reported more than 7,000 new cases. The seven-day rolling average has been climbing for more than two weeks, rising to the highest since the start of the outbreak.The virus is spreading exponentially in France, and increased testing doesnt explain the surge in infections, the Health Ministry said. While testing has more than doubled since early July, new cases have increased by a factor of 12. Its spreading in particular among young adults, who arent sticking to preventive measures as well as the elderly have.Pentagon Picks AstraZeneca Trial Sites (12:50 p.m. NY)The U.S. Department of Defense said that five sites have been selected for Phase 3 trials of a vaccine candidate under from AstraZeneca as part of Operation Warp Speed.Now that vaccines have passed the first phases of testing for safety, dosing and response, we are ready to move into the next phase where volunteers are needed to join large clinical studies, Assistant Secretrary of Defense for Health Affairs Tom McCaffery said in a statement. The sites include Naval Medical Center San Diego; Joint Base San Antonio Brooke Army Medical Center; Wilford Hall Ambulatory Surgical Center; Walter Reed Medical Center; and Fort Belvoir Community Hospital.For more articles like this, please visit us at bloomberg.comSubscribe now to stay ahead with the most trusted business news source.2020 Bloomberg L.P.

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The mission of MIT Technology Review is to make technology a greater force for good by bringing about better-informed, more conscious technology decisions through authoritative, influential, and trustworthy journalism.

On September 9, the science series NOVA, produced by GBH/Boston, will premiere the documentary Human Nature on PBS, which explores the science, history, and ethics of this revolutionary gene-editing technology. CRISPRs ability to genetically modify DNA offers amazing possibilities to treat disease but comes with the incredible responsibility of altering human life.

Jennifer Doudna was the first biologist to propose the genetic programming ability of CRISPR. Well ask Jennifer about the current state of CRISPR, its short and long-term possibilities and the incredible responsibilities of altering human DNA. Well also meet Adam Bolt, director of Human Nature, and discuss how he brought this ethically and technically complex story to life.

MIT Technology Reviews Spotlight On sheds light on the technologies and trends of the moment, such as gaming, CRISPR, advanced manufacturing, and cybersecurity. For an audience that craves unbiased, hype-free insight, Spotlight On invites recognized insiders to answer the essential questions of new tech: What is this? and Why should I care?

Jennifer DoudnaBiochemist and co-inventor of CRISPR genome editing technology

Adam BoltWriter and director, Human Nature

Hosted by Antonio RegaladoSenior editor of biomedicine,MIT Technology Review

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Spotlight on CRISPR - MIT Technology Review

In late January, as the COVID-19 pandemic was gaining steam, Charles Chiu, a researcher at the University of California, San Francisco, reached out to colleagues at San Franciscobased biotech Mammoth Biosciences. Chius group and the team at Mammoth had already collaborated on developing a CRISPR-based diagnostic test for Lyme disease, which they thought would easily translate to SARS-CoV-2 detection, he says.

The CRISPR system allows you to target pathogens very precisely, Chiu, who is part of Mammoths scientific advisory board, tells The Scientist. Within two to three weeks, we were able to go from just designing the assay to actually getting it to work and demonstrating that we can use it to rapidly identify or potentially diagnose SARS-CoV-2 from clinical samples.

The gold standard for detecting a virus such as SARS-CoV-2 is reverse transcription PCR, which requires isolation of RNA from a sample followed by the conversion of RNA to DNA and subsequent PCR to amplify any viral nucleic acids. While sensitive, this type of test requires collection of a specimen, such as saliva or a nasal swab, and the transport of that sample to a clinical lab for processing. Earlier in the year, reagents for this type of test were in short supply.

The new assay developed by Chiu and colleagues is called SARS-CoV-2 DETECTR (for SARS-CoV-2 DNA endonuclease-targeted CRISPR trans reporter); researchers published details about how it works in Nature Biotechnology on April 16. The test is fast: after RNA extraction, it takes less than an hour to get a result. And it uses loop-mediated amplification (LAMP), which takes place at just one temperature, rather than the cycling temperatures necessary for PCR, meaning an expensive thermocycler is not required.

The enzyme most commonly used for CRISPR genetic modification is Cas9, which, guided by a short RNA sequence, finds and cleaves a nucleic acid target. DETECTR uses a different enzyme, Cas12, which also follows a guide RNA to its targetin this case, SARS-CoV-2 sequencesbut cleaves more than just its target; it also starts chopping up nearby nucleic acids. The assay includes a reporter molecule that, when it gets cleaved by Cas12 during the cutting frenzy precipitated by SARS-CoV-2 nucleic acid recognition, can be visualized on a test strip, similar to a home pregnancy test.

In their paper, the authors reported that the sensitivity of DETECTR was comparable to a reverse transcription PCR assay run in a clinical lab on about 80 patient samples. On July 9, the assay was given emergency use authorization (EUA) by the US Food and Drug Administration (FDA), only for use at the University of California, San Franciscos clinical lab. Since then, the developers have continued to focus on making the test more accessible and easier to use.

In the iteration that was approved for EUA, theres still an RNA extraction step, Chiu explains. Thats a possible issue because there was a shortage of extraction reagents just a month or two ago, and its also limited by the fact that extractions typically take anywhere from a half an hour to an hour, so . . . we ended up modifying this test so that we can actually run the LAMP reaction directly from [an] original sample.

On May 20, Mammoth Biosciences established a partnership with pharmaceutical company GlaxoSmithKline Consumer Healthcare to further develop DETECTR into a handheld, disposable device that would be appropriate for home use and be about as expensive as an at-home pregnancy test.

The way point-of-need and at-home diagnostics will work is if theyre truly all-in-one, says Trevor Martin, Mammoth Biosciencess CEO. It needs to be as easy to use as a pregnancy test, and were also very much believers that it needs to give you results that are as trusted and accurate as something you would get in the lab.

Since the COVID-19 pandemic started to take off in the US in March, testing has largely focused on people who already have symptoms, partly because resources are limited. The trouble is, it seems as though the virus is most contagious around the time of symptom onset, meaning that if people are waiting to quarantine or take other protective measures until they get a positive result, they may have already infected others.

Testing at the earliest stage of symptoms is useful from an epidemiological perspective to understand how COVID impacts patients, says Rahul Dhanda, the CEO of Sherlock Biosciences, a Cambridge, Massachusettsbased biotech that develops diagnostics and is now focusing on SARS-CoV-2. But in terms of prevention and tracking the history of disease, what you want to do is test people who are asymptomatic.

Toward the goal of catching more cases before they spread and avoiding supply chain issues, a number of biotechs and academic labs are devising new strategies and exploiting existing technology to make testing simpler and available in settings other than doctors offices and clinical labs.

In a study published in PLOS Pathogenson August 27, for instance, a group based in China described a test that relies on RNA extraction that they call CRISPR-COVID. Their assay leverages Cas13a, an enzyme that behaves similarly to Cas12 to cleave a single-stranded reporter that fluoresces when SARS-CoV-2 RNA is present, and another type of isothermal (constant temperature) amplification, reverse transcription recombinase polymerase amplification. In the study, the authors estimate the cost of one CRISPR-COVID test at about $3.50, but predict it could be as low as $0.70 if the reagents were purchased in bulk. The current version is most appropriate for the lab, as it requires an RNA extraction step and specialized equipment to pick up fluorescent signals.

The fact that you can do a molecular diagnostic test in the palm of your hands is something that has never been possible before.

Rahul Dhanda, the CEO of Sherlock Biosciences

In a study published August 31 in PNAS,researchers developed a test that uses LAMP directly on patient samples and detects SARS-CoV-2 with a microfluidic cartridge and portable smartphone-based reader. Because it does not require RNA extraction or PCR amplification, this approach could enable the scalable deployment of COVID-19 diagnostics without laboratory-grade infrastructure and resources, especially in settings where diagnosis is required at the point of collection, such as schools, facilities that care for the elderly or disabled, or sporting events, the authors write.

The current pandemic is not the first time LAMP has been developed for diagnosing viral infections. Because of its portability, its been deployed in Ebola and Zika outbreaks. But the technology is not without drawbacks. There are a couple of problems with isothermal amplification, cautions Max Wilson, a biologist at the University of California, Santa Barbara (UCSB). Wilson has developed a COVID-19 test in use at UCSB that uses CRISPR for detection and PCR for amplification. In our hands . . . [LAMP] is less sensitive than other amplification methods, he explains.

Another problem is that if everyone starts using LAMP, the reagents, which arent made at volumes supportive of pandemic-scale testing, could become harder to get, Wilson says. The production has not been streamlined and optimized.

Despite these and other issues, there are a lot of really promising and complementary testing frameworks out there, he adds. All of the various approachesat-home testing, lab-based testing of samples taken at healthcare facilities, and assays that could be used to test thousands of people daily, such as in public health departments or at big universitiesdont even compete for resources or manufacturing capability, Wilson says, and theyre all necessary.

Theres a huge amount of unmet need when it comes to COVID-19 testing, Martin agrees. We just need all hands on deck.

Starting in mid-March, Dhanda and his team at Sherlock Biosciences pivoted their entire research program to focus on developing a CRISPR-based COVID-19 test kit using their platform SHERLOCK, which stands for specific high sensitivity enzymatic reporter unlocking. On May 6, that kit granted EUA and is now distributed in the US through a collaboration with Integrated DNA Technologies.

Because that test still includes a lot of steps, most of which are best performed in a lab, the researchers are now also working on a room-temperature, instrument-free test, the output of which can be visualized on a paper strip. That technology, which is called INSPECTR for internal splint-pairing expression cassette translation reaction, was originally developed at the Wyss Institute at Harvard University and relies on hybridization of a sample, such as saliva, to freeze-dried synthetic DNA complementary to SARS-CoV-2 RNA. If the sample contains viral RNA, a reporter protein is activated and then can be visualized with the naked eye, making the test a great option for eventual home use.

A design concept of INSPECTR

Wyss Institute at Harvard University

The fact that you can do a molecular diagnostic test in the palm of your hands is something that has never been possible before, says Dhanda, who adds that they are planning to apply for an EUA once development is finished, probably early next year. How much it will cost hasnt been disclosed, but a company representative tells The Scientistthat Sherlock is committed to making the test accessible.

In a study published in Science Advances on August 25, a group of researchers led by University of Albany biomedical engineer Ken Halvorsen also used a synthetic DNA approach to detect viral RNA, both from Zika virus and from SARS-CoV-2. The authors created DNA nanoswitches, small bits of DNA that change their shape in response to binding a target sequence. Because of the shape change, samples that contain viral RNA look different when run during gel electrophoresis than do samples without virus.

We can use this approach to detect viruses at levels that are relevant for health, Halvorsen says. The group is now trying to figure out how we can do a workflow that will allow us to do the whole thing outside of a lab. Because the test can be performed without enzymes, it already lends itself to streamlining, and they plan to simplify the readout, possibly using some type of cartridge that could be preconstructed, portable, and disposable. Were hoping to get the whole process down to under an hour, he tells The Scientist.

As for competition, I dont think that the testing for coronavirus is going to be a winner-take-all situation, Halvorsen says. There really need to be lots of different options. And it may turn out that there are many different testing types that all work in different situations, he adds. This may not be a short-term problem. We may be testing for years.

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Toward COVID-19 Testing Any Time, Anywhere - The Scientist