Archive for the ‘Crispr’ Category

First reported consecutive in vivo gene knockout and insertion achieves therapeutically relevant results in an alpha-1 antitrypsin deficiency mouse model

Inserted highly active WT1-TCR into the endogenous TCR locus for potential improved treatments for hematological and solid malignancies

CAMBRIDGE, Mass., Oct. 24, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology is presenting one oral presentation and four poster presentations at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) meeting taking place October 22-25, 2019, in Barcelona, Spain.

We are excited to share progress across Intellias in vivo and ex vivo programs at this important scientific venue, said Laura Sepp-Lorenzino, Ph.D., chief scientific officer, Intellia Therapeutics. Our data shows the complexity of the edits we are able to make with CRISPR/Cas9, while achieving important therapeutically relevant results. We are building on the success of our modular platform now having demonstrated consecutive targeted knockout and insertion genome edits in preclinical studies. Additionally, we presented data from our engineered cell therapy program, which continues to demonstrate the use of CRISPR/Cas9 for combined knockout and targeted integration in human T cells.

Intellia Demonstrates Consecutive In Vivo Genome Editing in Alpha-1 Antitrypsin Deficiency Mouse Model

Intellias oral presentation highlights its alpha-1 antitrypsin deficiency (AATD) study showing that consecutive dosing of two distinct lipid nanoparticle (LNP) formulations, in adultmice, achieves two targeted genome editing events, resulting in knocking out the faulty gene and restoring therapeutic levels of normal alpha-1 antitrypsin protein (hAAT). Intellias approach for AATD uses a modular hybrid delivery system combining a non-viral LNP which encapsulates CRISPR/Cas9 with an adeno-associated virus (AAV) carrying donor DNA template. Compared to traditional viral-based delivery of gene editing components, Intellias LNP delivery system can overcome the inherent limitations of immunogenicity to facilitate multiple in vivo gene editing events.

In a mouse model harboring the human PiZ allele, the most severe genetic defect in AATD patients, Intellia first reduced expression of the defective protein using gene knockout. Three weeks following the PiZ allele knockout, Intellia inserted the normal human alpha-1 antitrypsin gene, resulting in stable (throughout 12 weeks of observation), therapeutically relevant circulating protein levels. In the study, a sustained reduction of the circulating PiZ protein levels of >98% was observed for over 15 weeks. This is the first in vivo demonstration of a non-viral delivery platform, enabling a consecutive dosing approach for achieving multiple genome edits in the same tissue of the same animal. Intellias oral presentation, titled In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression, will be given by Anthony Forget, Ph.D., on October 25, 2019. This presentationwill be available on Intellias website at http://www.intelliatx.com.

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Intellias Poster Presentations

WT1-Specific TCR Engineered Cell Therapy Studies

Intellia presented new in vitro data showing that CRISPR/Cas9-mediated genome editing for in locus insertion, combined with endogenous T Cell Receptor (TCR) knockout, leads to significant reduction in mispairing of endogenous and transferred TCR chains. This approach is expected to generate transgenic-TCR (tg-TCR) T cell therapies for hematological cancers and solid tumors. Results demonstrate a highly efficient reduction of >98% in endogenous TCR and chains while reaching >70% insertion rates of tg-TCRs without further purification. The poster titled Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In Locus Insertion Combined with Endogenous TCR Knockout, was presented on October 24, 2019, by Birgit Schultes, Ph.D.

Researchers also presented in vitro data showing that a library of WT1-specific TCRs were generated, several of which Intellia is currently evaluating as part of its lead engineered cell therapy program targeting Acute Myeloid Leukemia (AML). This presentation, Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia, was presented on October 23, 2019 by Intellias collaborator, Erica Carnevale, Ph.D., IRCCS Ospedale San Raffaele.

Primary Hyperoxaluria Study

Intellia showed the continued progression of its modular platform capability using CRISPR/Cas9 to knockout either hydroxyacid oxidase 1 (Hao1) or lactate dehydrogenase A (Ldha), leading to a dose-dependent and persistent reduction of urinary oxalate levels in a Primary Hyperoxaluria Type 1 (PH1) mouse model. Data shows Ldha gene disruption also decreased LDH enzyme activity in the liver and did not impair the disposition of lactate in either wild type or renally-impaired mice. These results highlight the potential of editing genes in the glyoxylate detoxification pathway using a non-viral delivery approach as a one-time treatment option for PH1. These data were presented as a poster, titled CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria, by Sean Burns, M.D., on October 24, 2019.

Off-Target Screening Platform

Intellia demonstrated its approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Results from targeted off-target sequencing in edited cells showed that biochemical off-target discovery approaches were the most sensitive and accurate. These data were presented as a poster on October 23, 2019, titled In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics, by Dan OConnell, Ph.D.

About Intellia Therapeutics

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

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms such as its AATD program; develop our proprietary LNP-AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

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

Intellia Contacts:

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

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

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Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) - Yahoo...

Ninety-seven percent of potential new cancer drugs never make it to market, dropping out of clinical trials when they dont meet measures of safety or efficacy.

Why that is, we dont really know. But I think that this extremely high failure rate suggests that there are some fundamental issues in how new drug targets are studied and how new drugs are characterized, said molecular biologist Jason Sheltzer, PhD, an Independent Fellow at the Cold Spring Harbor Laboratory on Long Island, NY.

He decided to investigate, and uncovered the potential power of publishing negative evidence. The work fits in with Open Access week here at Public Library of Science.

CRISPR Improves Precision

The team reports inScience Translational Medicineon using the gene editing tool CRISPR-Cas9 to test whether 10 experimental cancer drugs work exactly how their developers predicted they would. And they found a tunnel vision in the way that drugs are targeted that might explain why certain patients do not respond as hoped.

Like an arrow that hits a tree rather than the bullseye, some cancer drugs may not actually reach their targets but many studies werent designed to reveal this. And so when results look promising, the drug candidate progresses through the FDA labyrinth.

Sheltzers strategy was straightforward: use CRISPR to remove the purported target, and if the drug still works, then the target isnt really the target. Perhaps preclinical research that identified a molecule as the drug target was halted, the scientists concluding success, when something fit the bill.

The researchers tested drugs that are either in clinical trials or once were, or are in preclinical studies (animals or human cells) not cancer drugs currently on the market. The experiments were done on standard cell lines from cancer patients.

The idea for many of these drugs is that they block the function of a certain protein in cancer cells. We showed that most of these drugs dont work by blocking the function of the protein that they were reported to block, Sheltzer explained.

Using CRISPR provided greater precision in interrogating potential drug targets than the older method, RNA interference. RNAi knocks down gene expression rather than snipping out a gene like CRISPR can.

Might a small molecule bind more than one type of target, like shooting arrows that hit trees and bushes as well as the bullseye? And sometimes what seems to be a valid drug target in vitro isnt exactly what happens in a body.

But a drug can make it to market without anyone knowing exactly how it works. Thats the case for selective serotonin reuptake inhibitor (SSRI) anti-depressants. The cartoons in ads depict neuromuscular junctions with the drug keeping serotonin in synapses longer by binding the reuptake proteins, presumably offsetting a deficit behind the symptoms. But googling SSRIsreturns the exact mechanism of actionof SSRIs is unknown.

From Slash-and-Burn to Hitting Targets

The new cancer drugs work in a few ways. Some of them zero in on molecules specific to cancer cells. These include:

These targeted drugs offer an alternative or adjunct to traditional drugs that broadly kill many types of rapidly-dividing cells, not just the cancer cells.

The targeted drugs began with Herceptinin 1998, its inventors recently honored with a Lasker award. The FDA approved another hugely successful targeted cancer drug, Gleevec, in just a few months in 2001. Today melodramatic ads pitch the new arsenal of cancer treatments: Zelboraf, Tafinlar, Keytruda, Opdivo.

But targeted drugs can fail if a new mutation alters the target or cancer cells find an alternate pathway that hikes cell division rate.

The researchers took a dual experimental approach based on logic:

Part of the confusion, I think, is semantic. Sometimes we deem a chemical interaction off-target if it doesnt do what we designed it to. Maybe our expectations were wrong. To be more unbiased, some researchers alter the language, calling the reliance of a cancer cell on a particular protein an addiction and investigating to seek druggable cancer dependencies.

The teams work indicates that what was deemed on-target may really be off-target, and vice versa. Perhaps its time to retire those terms.

The First Drug Tested

Earlier, Sheltzer investigated a protein called MELK, to which a company, OncoTherapy Science, is developing an inhibitor, called OTS167. Because MELK (maternal embryonic leucine zipper kinase) is abundant in many tumor types, it was presumed to be essential for their growth and therefore a drug target. But when CRISPR removed the gene that encodes MELK protein, nothing happened.

To our great surprise, when we eliminated these proteins from the cancer cells, they didnt die. The cancer cells continued to grow just fine, in spite of what had previously been published. They just didnt care about MELK, Sheltzer said.

The group published the findings on MELK in 2017, in eLIFE, raising the possibility that OTS167 is perhaps barking up the wrong tree. The drug candidate is in a phase 1(safety) trial for solid tumors and is recruiting for a phase 1trial for triple negative and metastatic breast cancer.

The MELK story inspired the group to use their genetic target-deconvolution strategy to see whether 10 other drugs were actually hitting their supposed targets. About a thousand cancer patients in total are taking one of these drugs in clinical trials.

Another Misguided Drug Reveals a Novel Target

In the new paper, the investigators question another drug, OTS964, being developed to treat certain lung and breast cancers. In the process, theyve discovered a new druggable cancer target.

RNAi had indicated that OTS964 targets a protein called PBK. But CRISPR told a different story cells with PBK gone still succumbed to the drug. It turns out that the interaction with PBK has nothing to do with how the drug actually kills cancer cells, Sheltzer said.

To find out how the PBK-targeting drug works, the researchers applied huge amounts of it to cancer cells and then gave the cells time to acquire mutations that would enable them to resist the drug. Cancer genomes are inherently unstable, mutating often. When a mutation renders a cell resistant to a drug, that cell then has an advantage and soon takes over the tumor.

Discovering how a cell circumvents a drug is priceless information.

The resistance experiments revealed that the cancer cell vulnerability that candidate drug OTS964 taps into isnt PBK after all, but a gene that encodes the protein CDK11. Its a cyclin-dependent kinase, an enzyme that is part of a pathway that leads to cell division.

The FDA has already approved CDK4/6 inhibitors, starting with Ibrance, in February 2015, to treat certain types of breast cancer. CDK11 is a brand new target. And thats potentially huge.

Whats Next?

At a news conference the researchers addressed concerns that their findings will affect people already taking targeted cancer drugs but they maintained that their work did not discover any approved drugs that were hitting trees instead of bullseyes.

But what about ongoing clinical trials for cancer drugs?

Sheltzer tried to alert folks running the trials. I filed a FOIA with the FDA to try to get additional information on the safety and efficacy of these drugs. The FDA declined to share that data, and said that it was a trade secret up until the point that these drugs received FDA approval.

He contacted companies sponsoring clinical trials too, but they wouldnt disclose any information either.

I think that the secrecy and the opacity in this drug development process really hurt scientific progress. A lot of drugs tested in cancer patients tragically dont help cancer patients. If this kind of evidence was routinely collected before drugs entered clinical trials, we might be able to do a better job assigning patients to therapies that are most likely to provide some benefit. With this knowledge, I believe we can better fulfill the promise of precision medicine, Sheltzer said.

The drug companies would do well to pay more attention to basic scientists who figure out how things work or dont work like Sheltzer. Using CRISPR can enable researchers to do a better job finding cancers central genes and a better job validating a drugs on-target mechanism of action. We think that that kind of preclinical foundation will help clinicians design better clinical trials to decrease the failure rate of new drugs, Sheltzer concluded.

Originally posted here:
When the Target Isn't Really the Target: One Way Cancer Drugs Fall Out of Clinical Trials | DNA Science Blog - PLoS Blogs

[He Jiankuis CRISPR babies] brought to the surface common misunderstandings even among scientists and ethicists thatreproductive usesof this genome-modifying tool have therapeutic value, will treat people with genetic disorders, will save lives, and will eradicate disease. None of those are true.

Imagine an individual or couple at high risk for creating a child with a serious genetic disease. They have the following simplified range of options:

Create a genetically related child in the time-honored fashion who will be at high risk for the genetic disease.

Create a genetically related child using CRISPR who will be at very low risk for the genetic disease.

Create no genetically related child.

The existence of option C undermines the claim that rCRISPR applications are lifesaving or curative.

Individuals have a choice in the matter of creating children at high risk of genetic disease: They can choose option C. Here is a different way of seeing the point that rCRISPR is not morally urgent because it does not involve a child whose existence, or illness, is inevitable.

Read full, original post: Using CRISPR to edit eggs, sperm, or embryos does not save lives

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Viewpoint: Why CRISPR embryo editing is not 'morally urgent': No one has to have a child - Genetic Literacy Project

Phenotypic screening is gaining new momentum in drug discovery with the hope that this approach will improve the success rate of drug approval.1 In this article we look at some of the latest screening tools and their applications.

This is illustrated by their recent study with Dr Ayman Zen where the team developed a high-content imaging screen using the endothelial tube formation assay, miniaturized to a 384-well plate format. Screening with an annotated chemical library of 1,280 bioactive small molecules identified a retinoid agonist, Tazarotene, that enhanced in vitro angiogenesis and wound healing in vivo. This high content screen identified an already FDA-approved small molecule that could be potentially exploited in regenerative medicine.3

Immuno-oncology: Pushing the Frontier of Discovery Through Advanced High Throughput Flow Cytometry

Immuno-oncology encompasses a number of approaches with one common thread: they harness the bodys own immune system against cancer.

Download this article to learn how advanced throughput flow cytometry overcomes these challenges to drive forward innovation in the immuno-oncology field.

Ebner is currently working in collaboration with recent Nobel-Prize winner Peter Ratcliffe, alongside scientists at Edinburgh University and MIT, to model hypoxia in glioblastoma. Hypoxia is a problem with some glioblastomas as it protects cells from radiotherapy treatment. Our aim is to use Peters expertise to help us set up an assay that mimics real tumor hypoxia. Then if we can identify small compounds that alter that hypoxic condition we can make the glioma cells more susceptible to either radiotherapy or temozolomide or some other treatment combination.

The labs main readout is high-content imaging, using fluorescent microscopy that can take many thousands of pictures. This approach utilizes different labels and harnesses software that automates the image analysis. The image analysis is set by the biologists but then it's applied across the entire screen. Its lower throughput than plate-based readout, but you get a lot more information out of the images, says Ebner. Increasingly, high content imaging is moving towards using AI and deep learning where you're trying to draw out even more information than the primary phenotype that you were looking at.

Indeed, a recent study using CRISPR-Cas9 mutagenesis showed that the proteins targeted by many cancer drugs currently in clinical development are non-essential for tumor growth, despite evidence to the contrary from previous studies using RNAi and small molecule inhibitors.4 In addition, the efficacy of the drugs tested was unaffected when CRISPR was used to knockout its assumed target suggesting that many are eliciting their anticancer activity through off-target effects.

The other benefit of CRISPR is that its extremely flexible, says Pettitt. This means you can expand the range of cell line models, for example, that you can screen in. The key reason why RNAi was such a popular technology, and now CRISPR is, is that you can basically knock out a gene by synthesizing just a short piece of RNA, he explains. CRISPR guides are very easy to synthesize, you can do it in a very high throughput setting, and you can design customized libraries to knock out every gene in the genome or a particular set of genes. As long as you can get the CRISPR machinery into your cells, it works very reliably.

The classic CRISPR (CRISPR-Cas9) system comprises a nuclease called Cas9 which you can program with a short RNA (20 nucleotides). The RNA will direct the nuclease to a certain site in the genome that matches and the nuclease will cleave the genome at that point. Repair of that double-strand break results in small insertions and deletions that result in knock out of a gene. But theres now more evolved applications of the technology emerging.

I think it's possible to be very creative with CRISPR in a way that it isnt with RNAi, says Pettitt. With RNAi you can really only shut genes off, but with CRISPR as well as making random mutations to knock out genes - you can also precisely edit genes if you provide a template region with a mutation with it. This can be incorporated into the target site for CRISPR so you can introduce the specific mutation youre interested in.

One such example is the problem with BRCA1 mutations: its important to be able to functionally classify whether these mutations are benign or pathogenic. A recent study used CRISPR to test 96.5% of all possible single-nucleotide variants (SNVs) in exons that encode functionally critical domains of BRCA1 and found over 400 non-functional missense SNVs were identified, as well as around 300 SNVs that disrupt expression. This knowledge will immediately aid clinical interpretation of BRCA1 genetic test results.5 In another study,6 Pettitt and colleagues used genome-wide CRISPR-Cas9 mutagenesis screens to identify the mutated forms of PARP that cause in vitro and in vivo PARP inhibitor resistance, and found that these mutations are also tolerated in cells with a pathogenic BRCA1 mutation resulting in a different profile of sensitivity to chemotherapy drugs compared with other types of PARP inhibitor resistance.

You couldnt screen at that level of detail using RNAi, where you design custom CRISPR that targets many different regions of the same gene and you can figure out which domains of the protein are important for your phenotype of interest, says Pettitt.

There are other evolutions of CRISPR now being developed as screens. For example, if you mutate the nuclease activity of Cas9, it still retains its ability to localize to the target site, so you can fuse Cas9 to transcriptional activators or repressors, and screen for transcriptional repression with CRISPR, as well as knock-out screens, says Pettitt. Theres also a whole range of CRISPR tools being developed that will edit bases by causing missense mutations rather than insertions or deletions, or causing methylation of DNA, or bringing in fluorescent proteins so you can visualize where the DNA sequences in the cells are. Its a measure of how flexible and useful CRISPR is in comparison to RNAi.

So will CRISPR be the one technology that everyone turns to for phenotypic screening in future? Im a firm believer that no technology answers every question, says Ebner. CRISPR is amazing, its use as a therapeutic or biologic is the stuff of science fiction. But as a tool for target identification, it comes with one important caveat. CRISPR knockout means exactly that it removes the potential protein that would otherwise be in the mix. Thats very different from a small compound inhibiting a protein that is still able to form a complex or that is just not active. Its the perfect example of a brilliant technology that is transformative, but it's not perfect. No technology is perfect.

References

1. Zheng W, Thorne N and McKew JC. Phenotypic screens as a renewed approach for drug discovery. Drug Discov. Today 2013; 18: 1067-1073.

2. Horvath P, Aulner N, Bickle M, et al. Screening out irrelevant cell-based models of disease. Nat Rev Drug Discov. 2016 Nov;15(11):751-769. doi: 10.1038/nrd.2016.175. Epub 2016 Sep 12.

3. Al Haj Zen A, Nawrot DA, Howarth A, et al. The Retinoid Agonist Tazarotene Promotes Angiogenesis and Wound Healing. Mol Ther. 2016 Oct;24(10):1745-1759. doi: 10.1038/mt.2016.153.

4.Lin et al. Off-target toxicity is a common mechanism of action of cancer drugs undergoing clinical trials. Science Translat Med. 2019; 11: (509). doi: 10.1126/scitranslmed.aaw8412

5.Findlay GM, Daza RM, Martin B et al. Accurate classification of BRCA1 variants with saturation genome editing. Nature. 2018 Oct; 562(7726): 217222. doi: 10.1038/s41586-018-0461-z

6.Pettitt et al. Genome-wide and high-density CRISPRCas9 screens identify point mutations in PARP1 causing PARP inhibitor resistance. Nat Commun. 2018 May 10;9(1):1849. doi: 10.1038/s41467-018-03917-2.

The rest is here:
Phenotypic Screening Advances in Technologies and Techniques - Technology Networks

You probably know from experience that there is not as much information on small-cap companies as there is on large companies. Of course, this makes it really hard and difficult for individual investors to make proper and accurate analysis of certain small-cap companies. However, well-known and successful hedge fund managers like Jeff Ubben, George Soros and Seth Klarman hold the necessary resources and abilities to conduct an extensive stock analysis on small-cap stocks, which enable them to make millions of dollars by identifying potential winners within the small-cap galaxy of stocks. This represents the main reason why Insider Monkey takes notice of the hedge fund activity in these overlooked stocks.

CRISPR Therapeutics AG (NASDAQ:CRSP) was in 13 hedge funds' portfolios at the end of June. CRSP shareholders have witnessed a decrease in support from the world's most elite money managers of late. There were 14 hedge funds in our database with CRSP positions at the end of the previous quarter. Our calculations also showed that CRSP isn't among the 30 most popular stocks among hedge funds(view the video below). Video: Click the image to watch our video about the top 5 most popular hedge fund stocks.

5 Most Popular Stocks Among Hedge Funds

So, why do we pay attention to hedge fund sentiment before making any investment decisions? Our research has shown that hedge funds' small-cap stock picks managed to beat the market by double digits annually between 1999 and 2016, but the margin of outperformance has been declining in recent years. Nevertheless, we were still able to identify in advance a select group of hedge fund holdings that outperformed the market by 40 percentage points since May 2014 through May 30, 2019 (see the details here). We were also able to identify in advance a select group of hedge fund holdings that underperformed the market by 10 percentage points annually between 2006 and 2017. Interestingly the margin of underperformance of these stocks has been increasing in recent years. Investors who are long the market and short these stocks would have returned more than 27% annually between 2015 and 2017. We have been tracking and sharing the list of these stocks since February 2017 in our quarterly newsletter. Even if you aren't comfortable with shorting stocks, you should at least avoid initiating long positions in our short portfolio.

Oleg Nodelman EcoR1 Capital

Unlike former hedge manager, Dr. Steve Sjuggerud, who is convinced Dow will soar past 40000, our long-short investment strategy doesn't rely on bull markets to deliver double digit returns. We only rely on hedge fund buy/sell signals. We're going to take a look at the recent hedge fund action surrounding CRISPR Therapeutics AG (NASDAQ:CRSP).

Heading into the third quarter of 2019, a total of 13 of the hedge funds tracked by Insider Monkey held long positions in this stock, a change of -7% from the first quarter of 2019. By comparison, 17 hedge funds held shares or bullish call options in CRSP a year ago. With hedge funds' sentiment swirling, there exists a few noteworthy hedge fund managers who were boosting their holdings considerably (or already accumulated large positions).

No of Hedge Funds with CRSP Positions

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Among these funds, EcoR1 Capital held the most valuable stake in CRISPR Therapeutics AG (NASDAQ:CRSP), which was worth $75.8 million at the end of the second quarter. On the second spot was Cormorant Asset Management which amassed $33 million worth of shares. Moreover, Farallon Capital, Clough Capital Partners, and Valiant Capital were also bullish on CRISPR Therapeutics AG (NASDAQ:CRSP), allocating a large percentage of their portfolios to this stock.

Since CRISPR Therapeutics AG (NASDAQ:CRSP) has experienced declining sentiment from hedge fund managers, we can see that there exists a select few hedge funds that elected to cut their entire stakes in the second quarter. It's worth mentioning that Steven Boyd's Armistice Capital said goodbye to the biggest investment of the 750 funds monitored by Insider Monkey, comprising about $2.9 million in stock. Noam Gottesman's fund, GLG Partners, also said goodbye to its stock, about $1.8 million worth. These bearish behaviors are intriguing to say the least, as aggregate hedge fund interest fell by 1 funds in the second quarter.

Let's also examine hedge fund activity in other stocks similar to CRISPR Therapeutics AG (NASDAQ:CRSP). We will take a look at Box, Inc. (NYSE:BOX), MGE Energy, Inc. (NASDAQ:MGEE), Independent Bank Corp (NASDAQ:INDB), and AMN Healthcare Services Inc (NYSE:AMN). This group of stocks' market values are closest to CRSP's market value.

[table] Ticker, No of HFs with positions, Total Value of HF Positions (x1000), Change in HF Position BOX,27,395491,-5 MGEE,13,53798,4 INDB,11,16780,5 AMN,14,109685,3 Average,16.25,143939,1.75 [/table]

View table hereif you experience formatting issues.

As you can see these stocks had an average of 16.25 hedge funds with bullish positions and the average amount invested in these stocks was $144 million. That figure was $180 million in CRSP's case. Box, Inc. (NYSE:BOX) is the most popular stock in this table. On the other hand Independent Bank Corp (NASDAQ:INDB) is the least popular one with only 11 bullish hedge fund positions. CRISPR Therapeutics AG (NASDAQ:CRSP) is not the least popular stock in this group but hedge fund interest is still below average. This is a slightly negative signal and we'd rather spend our time researching stocks that hedge funds are piling on. Our calculations showed that top 20 most popular stocks among hedge funds returned 24.4% in 2019 through September 30th and outperformed the S&P 500 ETF (SPY) by 4 percentage points. Unfortunately CRSP wasn't nearly as popular as these 20 stocks (hedge fund sentiment was quite bearish); CRSP investors were disappointed as the stock returned -13% during the third quarter and underperformed the market. If you are interested in investing in large cap stocks with huge upside potential, you should check out the top 20 most popular stocks among hedge funds as many of these stocks already outperformed the market so far in 2019.

Disclosure: None. This article was originally published at Insider Monkey.

Related Content

Original post:
Is CRISPR Therapeutics AG (CRSP) Going To Burn These Hedge Funds ? - Yahoo Finance

23 Oct 2019

A multi-institutional group, including members of the Tau Consortium, unveiled a stem cell tool kit for scientists studying primary tauopathies. In the November 12 issue of Stem Cell Reports, researchers co-led by Celeste Karch ofWashington University, St. Louis, and Alison Goate and Sally Temple of Icahn School of Medicine in New York, describe a collection of fibroblasts, induced pluripotent stem cells, and neural precursor cells. The cells come from 140 skin samples, some given by donors with richly documented clinical histories who carry pathogenic MAPT mutations or risk variants. Others come from noncarrier family members, patients with a sporadic tauopathy, and cognitively normal controls. The set includes induced pluripotent stem cell lines from 31 donors and 21 CRISPR-engineered isogenic lines. The cells are available to other researchers for study.

These types of high-quality repositories are becoming increasingly important for the scientific community, Clive Svendsen of the Cedars-Sinai Medical Center in Los Angeles wrote to Alzforum.

This is the way the field is going, agreed Lawrence Golbe of CurePSP, New York. Golbes organization funds research into progressive nuclear palsy (PSP) and related disorders, and collaborates with the Tau Consortium on other projects. Enthusiastic about the resources potential, Golbe hopes CurePSP grantees will get an automatic pass to use the cells.

Choice Mutations. Cells in the new iPSC collection carry some of the most common MAPT mutations, covering a wide range of clinical and neuropathological phenotypes of frontotemporal lobe dementia (FTLD)-Tau. [Courtesy of Karch et al., 2019.]

Tauopathies have proven difficult to study in animal models, in part because unlike other neuropathologies, they seem to afflict only humans (Heuer et al., 2012). Moreover, while adult human brains express approximately equal amounts of the tau spliced isoforms 3R and 4R, rodents produce almost exclusively 4R (Trabzuni et al., 2012). This is problematic. For example, leading proposals to explain how tau mutations cause disease point to abnormalities in splicing and microtubule binding, which differ between isoforms. The models we had been focusing on were not capturing the complexity of MAPT in human cells, said first author Karch. As a result, human induced pluripotent stem cells (iPSCs) have been gaining popularity in the field. The NINDS Human Cell and Data Repository is helping meet the demand by offering iPSC lines derived from 10 patients harboring MAPT mutations.

However, Karch and her collaborators think the field could benefit from a larger and more diverse collection of human cells, including isogenic iPSC lines. To accomplish this, they collected skin samples from 140 people carrying MAPT pathogenic mutations or risk variants, non-mutation carriers, and patients with sporadic PSP or corticobasal syndrome (CBS), most with comprehensive clinical histories. Although a few cells came from the NINDS repository, most came from patients participating in longitudinal studies at the Memory and Aging Center at the University of California, San Francisco, and the Knight Alzheimer Disease Research Center at WashU. The clinical records of most of these patients include detailed neurological and neuropathological workups, as well as fluid biomarkers and neuroimaging data collected from MRI, A-PET, and tau-PET studies.

To capture a broad range of phenotypes associated with some of the most common MAPT mutations, the authors created 36 fibroblast lines and 29 iPSC lines from individuals carrying the P301L, S305I,IVS10+16, V337M, G389R, and R406W mutations, as well as from carriers of the A152T variant, which increases the risk for both PSP and CBS (image above). The latter could be particularly useful for dissecting the mechanisms that underlie the phenotypic differences between the two diseases. The researchers also obtained iPSC lines from two noncarrier family members, and two people who suffered from autopsy-confirmed sporadic PSP. In addition, they stored fibroblast lines from 12 patients with sporadic PSP, five with CBS, 10 with a mixed PSP/CBS presentation, and 69 cognitively normal controls.

Biopsies are available for 27 of the 31 patients whose cells were used to generate iPSCs, and autopsy data for seven, including the two cases of sporadic PSP.

Importantly, the researchers edited 21 iPSC lines using CRISPR/Cas 9. They corrected cells with these mutations: MAPT IVS10+16,P301L, S305I, R406W, and V337M. Conversely, they inserted into control iPSCs these mutations: R5H, P301L,G389R, S305I, or S305S.

The authors also created a stem cell line carrying MAPT P301S,a mutation commonly overexpressed in tauopathy mouse models but not present in the available donors, by editing the P301L line. Isogenic lines are so powerful, particularly in these diseases which are so variable in their onset and progression, even within the same family, said Karch. Gnter Hglinger and Tabea Strauss at the German Center for Neurodegenerative Disease (DZNE) in Munich agreed. Having a pool of cell lines with different disease-linked mutations and risk variants from several individuals and their isogenic control cells is an excellent resource for the research community to enlighten disease mechanisms, they wrote (full comment below).

Several of the reported lines have already starred in recent studies of tauopathy mechanisms and candidate therapies (e.g., Sep 2019 conference news; Nakamura et al., 2019; Hernandez et al., 2019; Silva et al., 2019).

Karch and colleagues have partially differentiated some of the iPSCs and stored them as neural progenitor cells (NPCs), so that researchers can relatively easily thaw, expand, and differentiate them into neurons. These NPCs have proved useful for large-scale functional-genomics studies, proteomics, and genetic modifier screens (e.g., Cheng et al., 2017; Boselli et al., 2017;Tian et al., 2019).

In addition, the authors inserted a neurogenin-2 transgene into two healthy controls and two MAPT mutant stem cells, P301L and R406W. Neurogenin-2 enables low-cost, large-scale differentiation of stem cells into homogenous excitatory neurons. These transgenic cells are particularly useful for high-throughput drug screens (Wang et al., 2017; Sohn et al., 2019).

Researchers can request all the reported cells online at http://neuralsci.org/tau. They must provide a summary of experimental plans, an institutional material transfer agreement, and a nominal fee to cover maintenance and distribution costs. Karch said the process resembles that of the Coriell Institute and the NINDS repository. Our goal is to share with as few hurdles as possible, she said.

While the authors are still reprogramming fibroblasts they have already collected, they also plan to add more causative mutations, generate more isogenic lines, and obtain more cells from members of the same families to help shed light on phenotypic variability. In addition, Karch said, she hopes repository users will resubmit lines with new modifications they generate.

Jeffrey Rothstein, Johns Hopkins University, Baltimore, welcomed the new resource. I think it is great they have assembled this collection, he said. Rothstein founded and co-directs the Answer ALS research project, which has amassed 600 iPSC lines from controls and patients with amyotrophic lateral sclerosis (ALS).

Rothstein suggested the tauopathy collection may want to prioritize adding cells from donors with the most common form of disease, that is, sporadic. His group aims to generate 1,000 iPSC lines, with a large fraction representing sporadic diseasealso the most common form of ALSto identify the most prevalent disease subtypes. One strategy that has helped his group build their collection, he said, is using peripheral blood mononuclear cells instead of fibroblasts to create iPSCs. More donors are willing to donate blood than have a piece of skin punched out. In addition, iPSCs derived from blood cells are genetically more stable, he noted.

Rothstein emphasized the importance of assembling a large collection of healthy controls. Although isogenic controls are of great value, he cautioned they can be subject to artifacts. One problem is that the cell population can change due to selective pressures during CRISPR editing (Budde et al., 2017). To address this, Karch and colleagues are collecting not only modified iPSC clones, but also control clones that have gone through the editing pipeline but remain unmodified.

Stem-cell users studying tauopathies face another challenge: iPSC-derived neurons express primarily the fetal isoform of tau, 3R0N. However, citing a study that shows three-dimensional neuronal cultures switch to the adult profile relatively quickly (Miguel et al., 2019), Hglinger and Strauss wrote, [It] allows us to be optimistic that current challenges of this model system can be overcome in the future.Marina Chicurel

Read more:
Introducing: iPSC Collection from Tauopathy Patients - Alzforum

LEVERKUSEN, Germany and ZUG, Switzerland and CAMBRIDGE, Mass., Oct. 21, 2019 /PRNewswire/ -- CRISPR Therapeuticsand Bayer today announced proposed plans whereby Casebia Therapeutics, a joint venture between CRISPR Therapeutics and Bayer, would operate under the direct management of CRISPR Therapeutics. Upon closing of the transaction, Casebia Therapeutics would focus on the development of its lead programs in hemophilia, ophthalmology and autoimmune diseases, with Bayer having opt-in rights for two products at IND submission.

"The standalone Casebia entity combined the capabilities of CRISPR Therapeutics and Bayer to significantly advance the CRISPR/Cas9 gene-editing platform," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "As Casebia's programs have advanced beyond the discovery stage, we are evolving the operating model to leverage the manufacturing and clinical expertise of CRISPR Therapeutics to further accelerate these programs."

"We remain excited about the potential of cutting-edge CRISPR/Cas9 based therapies, which have the potential to create a whole new class of medicines," said Kemal Malik, Bayer board member for Innovation. "CRISPR Therapeutics has built the capabilities and expertise necessary to advance the Casebia programs to the next phase of development, and we look forward to continuing our collaboration with them."

The transaction is subject to negotiation and execution of definitive agreements as well as certain customary conditions. The companies anticipate the transaction will close in the fourth quarter of 2019.

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

About Bayer and Leaps by BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Bayer's products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, Bayer aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2018, the Bayer global group employed around 117,000 people and had sales of 39.6 billion euros. Capital expenditures amounted to 2.6 billion euros, R&D expenses to 5.2 billion euros. For more information, go towww.bayer.com.

Leaps by Bayer, a unit of Bayer is investing into solutions to some of today's biggest problems. Previous Leaps investments into potentially breakthrough technologies include BlueRock Therapeutics (iPSC technology to cure cardiovascular and CNS diseases), Joyn Bio (probiotics for plants to enable for chemical fertilizer-free farming), Khloris (iPSC as cancer vaccination agents for potential prevention or cure), Century Therapeutics (iPSCs for allogeneic cell therapy of cancer), and Pyxis Oncology (antibody-based immunotherapies targeting the tumor microenvironment).

CRISPR Forward-Looking StatementThis press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics' expectations about any or all of the following: (i) the proposed transaction involving Casebia Therapeutics; (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas-9 gene editing technologies and therapies, including in hemophilia,ophthalmology and for autoimmune diseases; and (iii) CRISPR Therapeutics' ability to leverage manufacturing and clinical expertise to meaningfully advance certain Casebia Therapeutics programs. Without limiting the foregoing, the words "believes," "anticipates," "plans," "expects" and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: uncertainties inherent in corporate restructuring, including the expected timing for completion of such restructuring and the possibility that the parties will be unable to consummate any proposed transaction; the possibility that the expected synergies from CRISPR Therapeutics' manufacturing and clinical expertise will not be realized, or will not be realized within the expected time period; the risk that the businesses will not be integrated successfully; the initiation and completion of preclinical studies for CRISPR Therapeutics' and/or Casebia Therapeutics' product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics' technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics' most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Therapeutics Investor Contact:Susan Kimsusan.kim@crisprtx.com

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

Bayer Media Contact:Chris Loder(201) 396-4325Christopher.loder@bayer.com

SOURCE Bayer

http://www.bayer.com

The rest is here:
CRISPR Therapeutics and Bayer Announce an Update on Casebia Therapeutics - PRNewswire

Mon, Oct 21, 2019 - 5:50 AM

New York

AS investors await results from the first US clinical trials of the gene-editing system known as Crispr, scientists are focused on finding ways to administer it directly into humans, according to the technology's co-inventor, Jennifer Doudna.

Right now, in studies using Crispr that have treated patients, researchers have had to extract their cells to be able to make edits to faulty DNA before infusing them back into the body for treatment.

Being able to do precise edits directly inside humans, animals or plants could open the door to new applications, Ms Doudna said.

"With advances and delivery techniques, it may be possible to do that kind of very highly efficient targeted genome editing in the patient, without having to remove cells, but actually to just do a treatment in the patient where the delivery vehicle takes the editing molecule to the right cells," she said in an interview before the Welch Foundation Conference on chemical research this week.

"Sounds fantastical today, but I think that's coming."

In essence, Crispr is a gene-editing system that can splice away parts of human DNA that make people susceptible to disease or defects. While it can be used in plants and animals, scientists are working on therapeutic applications that can offer a one-time cure for certain diseases.

Crispr Therapeutics AG was the first company to start a human trial back in February, and is due to report initial results by year-end.

Editas Medicine Inc is leading efforts in "in-vivo", or inside the body, testing and initiated a clinical study in July. Intellia Therapeutics Inc is expected to follow with its own study next year.

A safe delivery of Crispr directly into humans would shorten manufacturing times and offer new opportunities for the companies.

The biggest challenge is to find a way to deliver gene-editing molecules into specific cell types safely and efficiently, Ms Doudna said.

"That's kind of the next frontier," she added. "If we figure that out, it really does open the way to many, many more kinds of applications in genome editing than are possible today."

Crispr and Intellia Therapeutics have licensed their technology from the University of California at Berkeley, Ms Doudna's academic home, while Editas is using inventions from the Broad Institute in Massachusetts.

The two institutions are fighting over who was first to invent breakthrough gene-editing technology. Ms Doudna is a co-founder of Editas and other Crispr startups and is a scientific board member at Intellia.

The gene-editing field, which only recently entered human testing and has been plagued by research raising safety concerns, recently got some encouraging news.

Chinese researchers safely treated a man with leukemia and HIV using gene-edited stem cells, according to a report in the New England Journal of Medicine. While the attempt to cure his HIV failed, his cancer is in remission 19 months after the treatment, and the modified cells integrated into his body.

The case, which is the first detailed report in a major academic journal of how doctors are using Crispr in living patients, is an "important milestone" and suggests that gene editing will be "a safe technology and that the challenge now is to have it be really effective in different disease settings", Ms Doudna noted. BLOOMBERG

Read the original:
Crispr's next frontier is in-human treatment, says co-inventor - The Business Times

A joint venture between gene-editing startup CRISPR Therapeutics and German pharmaceutical company Bayer will come under the control of CRISPR Therapeutics, according to the pairs proposed plans.

In an announcement published yesterday, October 21, CRISPR Therapeutics and Bayer proposed that the joint venture, Casebia Therapeutics, would focus on the development of its lead programmes in haemophilia, ophthalmology and autoimmune diseases.

The companies agreed to form Cambridge, Massachusetts-based Casebia Therapeutics in December 2015, with the aim of discovering, developing and commercialising new breakthrough therapeutics to cure blood disorders, blindness, and congenital heart diseases.

Samarth Kulkarni, CEO of CRISPR Therapeutics, said: As Casebia's programs have advanced beyond the discovery stage, we are evolving the operating model to leverage the manufacturing and clinical expertise of CRISPR Therapeutics to further accelerate these programmes.

Bayer will have opt-in rights for two products at investigational new drug application submissions.

Kemal Malik, Bayer board member for innovation, added: We remain excited about the potential of cutting-edge CRISPR/Cas9 based therapies, which have the potential to create a whole new class of medicines.

The transaction is expected to close in the fourth quarter of 2019.

In September, an alliance of companies that use gene-editing technologies (including CRISPR Therapeutics) released a bioethical framework, as controversy over gene-editing rages on.

The principles agree that the developers do not support germline gene editing (the process by which the genome of an individual is changed so that the change is heritable) in human clinical trials or for human implantation.

Last week, CRISPR Therapeutics announced that it had entered a licence agreement with biotech KSQ Therapeutics, gaining access to KSQs IP for editing certain novel gene targets in its allogeneic oncology cell therapy programmes.

KSQ gained access to CRISPR Therapeutics IP for editing novel gene targets identified by KSQ as part of its current and future cell programmes.

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Bayer, CRISPR Therapeutics, joint venture, CRISPR, gene-editing, haemophilia, ophthalmology, autoimmune diseases

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CRISPR Therapeutics to buy JV from Bayer - Life Sciences Intellectual Property Review

Russian scientist Denis Rebrikov recently revealed that he has started a gene-editing process that might eventually enable couples carrying the genetic mutation that causes deafness to give birth to children who can hear. The news was shared with Nature on 17 October via an e-mail.

According to Nature, the scientist mentioned in the e-mail that he will soon publish the results of his experiments, which involves testing the ability of CRISPR to repair the gene causing deafness GJB2 in cells taken from people that have the mutation. Rebrikov believes that the result will help to lay the groundwork for the clinical work. Rebrikov also added that he wants to help couples with unimpaired hearing to have a child such as these to have a child with the same mutation.

Rebrikov also mentioned that he has local review board's permission to do the research but it does not allow the transfer of the gene-edited eggs into the womb and pregnancy. The scientist also emphasized that he will not be going ahead without the approval from the Ministry of Health of the Russian Federation. "I will definitely not transfer an edited embryo without the permission of the regulator," he confirmed.

However, the chances of this happening seem to be really low as last week, the ministry released a statement where it mentioned that the production of gene-edited babies is premature. Rebrikov, however, is not ready to lose hope. He says, "it is hard to predict" when he'll get permission for it so till then all the necessary safety checks need to be undertaken.

Rebrikov has previously also announced that he intends to use the CRISPR tool for gene-edited babies resistant to HIV. At that time, the news came as a shock to all international researchers as people feared that he is following the Chinese scientist He Jiankui who previously announced the controversial birth of the world's first gene-edited babies twin girls.

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CRISPR scientist wants to edit genes that cause deafness but falls short of permission - International Business Times, Singapore Edition

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

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

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

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

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

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

CRISPR Therapeutics AG (CRSP):

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

CRISPR Therapeutics AG (CRSP) stock Trading Summary:

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

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

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

CRISPR Therapeutics AG (CRSP) Stock Past Performance

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

Volatility in Focus:

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

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

Overbought and Oversold levels

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Read full, original post: Biotech Top Ten

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Earnings & Valuation

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

Profitability

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

Liquidity

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

Analyst Ratings

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

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

Institutional & Insider Ownership

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

Performance

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

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

Summary

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Such data permitted a revelation for Mojica in his work.

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

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

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

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

Genome-editing came as a "wonderful surprise"

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

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

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

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

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

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

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

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

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

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

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

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

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

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

References:

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

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

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

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

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

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

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

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

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

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This report studies the global CRISPR And CRISPR-Associated (Cas) Genes Market analyses and researches the CRISPR And CRISPR-Associated (Cas) Genes development status and forecast in the United States, EU, Japan, China, India, and Southeast Asia. This report focuses on the top players in the global market.

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

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

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

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

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

With tables and figures helping analyze worldwide Global CRISPR And CRISPR-Associated (Cas) Genes market, this research provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

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

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

By theproduct type, the market is primarily split into:

Look into Table of Content of CRISPR And CRISPR-Associated (Cas) Genes Market Report at @https://www.360marketupdates.com/TOC/13714540#TOC

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

The study objectives of this report are:

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

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

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

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

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

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

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

About KSQ Therapeutics

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

About CRISPR Therapeutics

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

CRISPR Therapeutics Forward-Looking Statement

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

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

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

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

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

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

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

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

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain.

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

Oral Presentation:

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

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

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

Poster Presentations:

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

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

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

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

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

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

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

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

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

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria

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

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

About Intellia Therapeutics

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

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms; develop our proprietary LNP/AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

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

Intellia Contacts:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

So where did Nielsen and his colleagues go wrong?

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

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

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

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

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

You can see the now-retracted paper here.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Image Credit: Shutterstock.com

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