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Archive for the ‘Crispr’ Category

CRISPR and CAS Gene Market to Score Past US$ 7603.8 Million Valuation by 2027: CMI KSU | The Sentinel Newspaper – KSU | The Sentinel Newspaper

Global CRISPR and CAS GeneMarket, By Product Type (Vector-based Cas and DNA-free Cas), By Application (Genome Engineering, Disease models, Functional Genomics, Knockdown/activation, and Other Applications), By End User (Biotechnology and Pharmaceutical Companies,Academic Government Research Institutes, and Contract Research Organizations), and By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) was valued at US$ 1,388.1 million in 2017, and is projected to exhibit a CAGR of 20.8% over the forecast period (2018 2026).

Manufacturers in the CRISPR and CAS gene are collaborating with many companies for sponsoring clinical trials. Editas Medicine has licensed CRISPR and other gene editing patent rights from the Broad Institute, the Massachusetts Institute of Technology (MIT), Harvard University, and others. In March 2017, Editas reportedly entered into an agreement with Irish pharmaceutical company Allergan under, which Editas was to receive a US$ 90 million up-front payment for an option to license up to five preclinical programs targeting eye disease. Moreover, various organizations are also focusing on new clinical trials for the CRISPR and CAS gene for cancer treatment. In 2018, CRISPR Therapeutics and Vertex launched the first in-human clinical trial of CRISPR genome editing technology sponsored by U.S. companies. The trial is testing an experimental therapy for the blood disorder -thalassemia in Regensburg, Germany.

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Increasing research and studies regarding the CRISPR and CAS gene technology is majorly driving the growth of CRISPR and CAS gene market. In 2017, Editas partnered with Juno Therapeutics for cancer-related research using CRISPR. Under the terms of the agreement, Juno had to pay Editas an initial payment of US$ 25 million, in which up to US$ 22 million will be used in research support for three programs over five years. Editas has also engaged in a three-year research and development (R&D) collaboration deal with San Raffaele Telethon Institute for Gene Therapy to research and develop next generation stem cell and T-cell therapies for the treatment of rare diseases.

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CRISPR and CAS Gene Market to Score Past US$ 7603.8 Million Valuation by 2027: CMI KSU | The Sentinel Newspaper - KSU | The Sentinel Newspaper

Diseases once thought incurable are now on the cusp of treatments. It’s because of CRISPR. Here’s a primer – Genetic Literacy Project

Like many other advances in science and medicine, CRISPR was inspired by nature. In this case, the idea was borrowed from a simple defense mechanism found in some microbes, such as bacteria.

To protect themselves against invaders like viruses, these microbes capture snippets of the intruders DNA and store them away as segments called CRISPRs, or clustered regularly interspersed short palindromic repeats. If the same germ tries to attack again, those DNA segments (turned into short pieces of RNA) help an enzyme called Cas find and slice up the invaders DNA.

After this defense system was discovered, scientists realized that it had the makings of a versatile gene-editing tool. Within a handful of years, multiple groups had successfully adapted the system to edit virtually any section of DNA, first in the cells of other microbes, and then eventually in human cells.

There are still a lot of questions about all the ways that CRISPR might beput to use in cancer researchand treatment. But one thing is for certain: The field is moving incredibly fast and new applications of the technology are constantly popping up.

People are still improving CRISPR methods, Dr. [Jerry] Li said. Its quite an active area of research and development. Im sure that CRISPR will have even broader applications in the future.

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Diseases once thought incurable are now on the cusp of treatments. It's because of CRISPR. Here's a primer - Genetic Literacy Project

Experts Predict the Hottest Life Science Tech in 2021 and Beyond – The Scientist

Through the social and economic disruption that COVID-19 caused in 2020, the biomedical research community rose to the challenge and accomplished unprecedented feats of scientific acumen. With a new year ahead of us, even as the pandemic grinds on, we at The Scientist thought it was an opportune time to ask what might be on the life science innovation radar for 2021 and beyond. We tapped three members of the independent judging panel that helped name our Top 10 Innovations of 2020 to share their thoughts (via email) on the year ahead.

Paul Blainey: Value is shifting from the impact of individual technologies (mass spectrometry, cloning, sequencing, PCR, induced pluripotent stem cells, next generation sequencing, genome editing, etc.) to impact across technologies. In 2021, I think researchers will increasingly leverage multiple technologies together in order to generate new insights, as well as become more technology-agnostic as multiple technologies present plausible paths toward research goals.

Kim Kamdar: Partially in reaction to the COVID-19 pandemic, one 2021 headline will be the continued innovation focused on consumerization of healthcare, which is redefining how consumers engage with providers across each stage of care. Consumers are even selective about their healthcare choices now, and the retail powerhouses like CVS and Walmart have and will continue to develop solutions to meet the needs of their customers. While this was already underway prior to the pandemic, the crisis has spurred on this activity with the goal of making healthcare more accessible and affordable and ultimately delivering on better health outcomes for all Americans.

Robert Meagher: I think this is easymRNA delivery. This is something that has been in development for years for numerous applications, but the successful development and FDA emergency use authorization of two COVID-19 vaccines based on this technology shines a very bright spotlight on this technology. The vaccine trials and now widespread use of the vaccines will give developers a lot of data about the technology, and sets a baseline for understanding safety and side effects when considering future therapeutic applications outside of infectious disease.

PB:Single-cell technology is here to stay, although its use will continue to change. One analogy to be drawn is the shift we saw from the popularity ofde novo genome sequencing (during the human genome project and the early part of the NGS [next-generation sequencing] era to the rich array of re-sequencing applications practiced today. I expect new ways to use single-cell technology will continue to be discovered for some time to come.

KK: Innovation in single-cell technology has the potential to transform biological research driving to a level of resolution that provides a more nuanced picture of complex biology. Cost has been a key barrier for broader adoption of single-cell analysis. As better technology is developed, cost will be reduced and there will be an explosion in single-cell research. This dynamic will also allow for broader adoption of single-cell technology from translational research to clinical applications particularly in oncology and immunology.

RM: Yesthere is continuing innovation in this space, and room for continued innovation. One area that we have seen development recently, and I see it continuing, is to study single cells not just in isolation, but coupled with spatial information: understanding single cells and their interactions with their neighbors. I also wonder if the COVID-19 pandemic will spur increased interest in applying single-cell techniques to problems in infectious disease, immunology, and microbiology. A lot of the existing methods for single-cell RNA analysis (for example) work well for human or mammalian cells, but dont work for bacteria or viruses.

PB: The promises of CRISPR and gene editing are extraordinary. I cant wait to see how that field continues to develop.

KK: Much of the CRISPR technology focus since it was unveiled in 2012 has been on its utility to modify genes in human cells with the goal of treating genetic disease. More recently, scientists have shown the potential of using the CRISPR gene-editing technology for treatment of viral disease (essentially a programmable anti-viral that could be used to treat diseases like HIV, HBV, SARS, etc. . . .). These findings, published in Nature Communications, showed that CRISPR can be used to eliminate simian immunodeficiency virus (SIV) in rhesus macaque monkeys. If replicated in humans, in studies that will be initiated this year, CRISPR could be utilized to address HIV/AIDS and potentially make a major impact by moving a chronic disease to one with a functional cure.

PB: New therapeutic modalities that expand the addressable set of diseases are particularly exciting. Cell-based therapies offer versatile platforms for biological engineering that leverage the power of human biology. It is also encouraging to see somatic cell genome editing technology advance toward the clinic for the treatment of serious diseases.

The level of innovation that occurred in 2020 to combat COVID-19 will provide a more rapid, focused, and actionable reaction to future pandemics.

Kim Kamdar, Domain Associates

RM: Besides the great success with mRNA-based vaccines that sets the stage for other clinical technologies based on mRNA delivery, the other area that is really in the spotlight this year is diagnostics. There are a lot of labs and companies, both small and large, that have some really innovative products and ideas for portable and point-of-care diagnostics. For a long time, this was often thought of in terms of a problem for the developing world, or resource-limited locations: think, for example, of diagnostics for neglected tropical diseases. But the COVID-19 pandemic and the associated need for diagnostic testing on a massive scale has caused us to rethink what resource-limited means, and to understand the challenge posed by bottlenecks in supply chains, skilled personnel, and high-complexity laboratory facility. There has been a lot of foundational research over the past couple of decades in rapid, portable, easy-to-use diagnostics, but translating these to clinically useful products often seemed to stall, I suspect for lack of a lucrative market for such tests. But we are now starting to see FDA [emergency use authorization for] home-based tests and other novel diagnostic technologies to address needs with the COVID-19 pandemic, and I suspect that this paves the way for these technologies to start being applied to other diagnostic testing needs.

PB: Seeing the suffering and destruction wrought by COVID-19, it is obvious that we need to be prepared with more extensive, equitable, and better-coordinated response plans going forward. While rapid vaccine development and testing were two bright spots last year, there are so many important areas that demand progress. As we learn about how important details become in a crisisno matter how small or mundanediagnostic technologies and the calibration of public health measures are two areas that merit major focus.

KK: The life science community response to the COVID-19 pandemic has already proven to be light-years ahead of previous responses particularly in areas such as vaccine development and diagnostics. It took more than a year to sequence the genome of the SARS virus in 2002. The COVID-19 genome was sequenced in under a month from the first case being identified. Scientists and clinicians were able to turn that initial information to multiple approved vaccines at a blazing speed. Utilizing messenger RNA (mRNA) as a new therapeutic modality for vaccine development has now been validated. Vaccine science has been forever changed. The pandemic has also focused a much-needed level of attention to diagnostics, forcing a rethink of how to increase access, affordability, and actionability of diagnostic testing. The level of innovation that occurred in 2020 to combat COVID-19 will provide a more rapid, focused, and actionable reaction to future pandemics. In addition, the elevation of a science advisor (Dr. Eric Lander) to a cabinet level position in the Biden administration bodes well for our future ability to ground in data and as President Biden himself framed, refresh and reinvigorate our national science and technology strategy to set us on a strong course for the next 75 years, so that our children and grandchildren may inhabit a healthier, safer, more just, peaceful, and prosperous world.

RM: One thing that really kick-started research to address COVID-19 was the early availability of the complete genome sequence of the SARS-CoV-2 virus, and the ongoing timely deposition of new sequences in nearreal-time as isolates were sequenced. This is in contrast to cases where deposition of large number of sequences may lag an outbreak by months or even years. I foresee the nearreal-time sharing of sequence information to become the new standard. Making the virus itself widely and inexpensively available, in inactivated form, as well as well-characterized synthetic viral RNA standards and proteins also helped spur research.

A trend Im less fond of is the rapid publication of nonpeer reviewed results as preprints online. Theres a great benefit to getting new information out to the community ASAP, but unfortunately I think the rush to get preprints up in some cases results in spreading misleading information. This problem is compounded with uncritical, breathless press releases accompanying the posting of preprints, as opposed to waiting for peer-review acceptance of a manuscript to issue a press release. I think the solution may lie in journals considering innovative approaches to speeding up peer review, or a way to at least perform a basic check for rigor prior to posting a preliminary version of the manuscript. Right now the extremes are: post an unreviewed preprint, or wait months or even years with multiple rounds of peer review including extensive additional experiments to satisfy the curiosity of multiple reviewers for high impact publications. Is there a way to prevent manuscripts from being published as preprints with obvious methodological errors or errors in statistical analysis, while also enabling interesting, well-done yet not fully polished manuscripts to be available to the community?

Paul Blaineyis an associate professor of biological engineering at MIT and a core member of the Broad Institute of MIT and Harvard University. The Blainey lab integrates new microfluidic, optical, molecular, and computational tools for application in biology and medicine. The group emphasizes quantitative single-cell and single-molecule approaches, aiming to enable studies that generate data with the power to reveal the workings of natural and engineered biological systems across a range of scales. Blainey has a financial interest in several companies that develop and/or apply life science technologies: 10X Genomics, GALT, Celsius Therapeutics, Next Generation Diagnostics, Cache DNA, and Concerto Biosciences.

Kim Kamdaris managing partner at Domain Associates, a healthcare-focused venture fund creating and investing in biopharma, device, and diagnostic companies. She began her career as a scientist and pursued drug-discovery research at Novartis/Syngenta for nine years.

Robert Meagheris a principal member of Technical Staff at Sandia National Laboratories. His main research interest is the development of novel techniques and devices for nucleic acid analysis, particularly applied to problems in infectious disease, biodefense, and microbial communities. Most recently this has led to approaches for simplified molecular diagnostics for emerging viral pathogens that are suitable for use at the point of need or in the developing world. Meaghers comments represent his professional opinion but do not necessarily represent the views of the US Department of Energy or the United States government.

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Experts Predict the Hottest Life Science Tech in 2021 and Beyond - The Scientist

Got $5,000 and 5 Years to Wait? Buy These 5 Hot Biotech Stocks Now – Motley Fool

Are you the type of investor who patiently plays the long game, or are you the more impulsive type? While it's easy to give in to the temptation to buy and sell your stocks based on short-term price movements, you'll see the largest gains when you buckle up for the long haul. Five years might seem like a long time to wait for your purchases to pay off, but when it comes to biotech stocks, practiced patience can pay off big.

All of the stocks below have recently met key milestones in their drug development efforts. More importantly, each is planning on making even bigger breakthroughs on the five-year horizon, so they're worth buying and holding until then. Given that these companies are still relatively early-stage and the risks of failure remain high, it'll be prudent for investors to diversify across all five rather than going all-in on any single stock.

Image source: Getty Images.

Late last year, CRISPR Therapeutics (NASDAQ:CRSP) reported favorable results from early-stage clinical trials of its CTX001 gene-editing therapy for beta thalassemia and sickle cell disease. That's great news, as CTX001 is the company's most advanced program. Over the next five years, CTX001 will likely continue to move forward in clinical trials.

The same may be true for four of its immuno-oncology programs that are currently in clinical development. Elsewhere in the pipeline, its regenerative gene therapy for diabetes mellitus will enter its phase 1/2 clinical trials in 2021, and potentially conclude them in the following years. It needs that time to prove that its products are safe and effective.

Lexicon Pharmaceuticals (NASDAQ:LXRX) makes sotagliflozin, which is currently approved to treat type 1 diabetes in the EU. Soon, it will likely conclude its registration with U.S.-based regulators and initiate sales shortly thereafter. The company is also conducting phase 3 clinical trials for the drug to see if it's effective at treating heart failure. If successful, Lexicon will have a steady stream of revenue, and it could reach profitability in the next few years. Its project for diabetic peripheral neuropathic pain in phase 2 clinical trials could also drive shareholder returns even further, assuming it too gets approved.

Fate Therapeutics (NASDAQ:FATE) may not have any products approved for sale, but its collection of immuno-oncology treatments derived from pluripotent stem cells has already made its stock a high performer over the last year. While the stock is doubtlessly a bit expensive at the moment considering how far away the company is from recurring revenue, the company is establishing itself as a leader in the stem cell-derived therapeutics space, which will pay off down the line.

Fate currently has seven projects that are currently in phase 1 clinical trials, which means that it'll likely have at least one of those advancing into the final stages of the process by 2026.

CRSP data by YCharts

Jounce Therapeutics (NASDAQ:JNCE) focuses on developing cancer immunotherapies that address underserved patient populations, and it's been a great stock to own over the last year. It'll report two sets of data from a pair of its clinical trials in the second half of 2021, which could bolster a higher stock price.

Through the next few years, Jounce plans to submit an Investigational New Drug (IND) filing to regulators every 12 to 18 months, paving the way for new early-stage clinical trials. With innovation at such a rapid pace, investors will have plenty of growth catalysts to look forward to, assuming Jounce's projects continue to mature.

Corcept Therapeutics (NASDAQ:CORT) is special because it's already profitable, and it has a larger and more advanced pipeline than the other companies listed here. It makes the drug Korlym, a treatment for Cushing syndrome, and it's also exploring in phase 3 trials another drug for the same indication. The company also has a late-stage pancreatic cancer therapy in development, not to mention a handful of other mid-stage oncology and metabolic disease projects.

If you're a bit worried that the other biotechs on this list might not pay off, you can take heart in that Corcept is considerably safer. Though there's still no guarantee that its pipeline projects will turn into new revenue streams anytime soon, its quarterly revenue is growing by 6% year over year. And it barely has any debt, with a scant $3.03 million owed. In other words, this company's story over the next five years will be about using its proceeds from drug sales to invest heavily in its future opportunities for growth. For a biotech investor, there's no better position to be in.

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Got $5,000 and 5 Years to Wait? Buy These 5 Hot Biotech Stocks Now - Motley Fool

Bystander Killing Could Be Key Factor in CAR-T Success in Non-Hodgkin Lymphoma – Cancer Therapy Advisor

Even after a decade of treating patients with hematologic malignancies with chimeric antigen receptor (CAR) T-cell (CAR-T) therapies, researchers are still trying to understand why most patients eventually relapse. Equally puzzling to some scientists is the question of these cellular interventions cause lasting remission at all in many patients.

Given that typical cancers consist of diverse cells, including those that do not express the antigens targeted by CAR-T cells, one would expect relapses through antigen escape to be much more common than currently observed in practice, explained Joshua Brody, MD, director of the Lymphoma Immunotherapy Program at The Tisch Cancer Institute at Mount Sinai in New York, New York. In CAR-T cells, maybe [only] 40% of patients have this antigen escape problem. Its weird to us that its not 100%, he said.

For instance, even though most patients with B-cell acute lymphocytic leukemia (ALL) possess over 1% of CD19-negative cells at diagnosis, the incidence of relapse after CD19-targeted therapy only approximates 20%.1,2 And, in patients with diffuse large B-cell lymphoma (DLBCL), similar response rates to CAR-T cell therapy have been observed regardless of tumoral expression of CD19.3 To Brody and colleagues, such observations suggest that CAR-T cells use mechanisms independent of antigen targeting to eliminate tumor cells.

A study published by Brodys team and collaborators at Kite Pharma in Cancer Discovery in December 2020 offered one explanation.4 Experiments in animal models suggested that CAR-T cells can kill off-target cells that are in the vicinity of the cells theyre designed to target, offering the first in vivo proof of localized bystander killing. This off-target effect is mediated by the interaction between the protein Fasa cell death receptor expressed on many cellular surfacesand its ligand, which is present on T cells. In fact, tumoral expression of Fas was predictive of survival in patients with DLBCL who were treated with anti-CD19 CAR-T cell therapy in the phase 1/2 ZUMA-1 trial (ClinicalTrials.gov identifier:NCT02348216).

I think its becoming more and more [clear] that the CAR-Ts, in addition to their CAR interaction with the tumor antigen, rely on the Fas-Fas ligandinteraction to exert their killing. The next question is, how do we manipulate these pathways safely to make CARs more potent? said Saad J. Kenderian, MB, ChB, a consultant in the division of hematology in the department of internal medicine at the Mayo Clinic in Rochester, Minnesota, who was not involved in the study.

The new research was the result of a serendipitous observation made during a clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) screen exploring the genes that tumor cells use to either resist or facilitate cytotoxic T cell killing. The team noticed in cell culture that T cells engineered to target a specific protein would not only kill the on-target lymphoma cells expressing that protein, but also the cells that did not.

Further experiments pointed to Fas as a mediator of this process. When the researchers experimentally removed the gene encoding Fas from cultured lymphoma cells, they noticed that this protected both on-target and off-target cells. This was the case even as T-cells emitted the cell death-inducing molecules granzyme and perforin, which are thought to represent the main method of killing.

T cells require an interaction with their target antigen to kill a cell via the perforin and granzyme mechanism, but as long as off-target cells are in the direct vicinity of on-target cells, Brodys results suggest that T-cells can eliminate them via the Fas-dependent mechanism. Essentially, you kill the target cell youre going after and just to be safe, you [also] kill the cell next door, he said.

Further cell culture and mouse experiments bolstered this hypothesis. In one experiment, mice treated with murine CD19 CD3-CD28 CAR-T cells had similar rates of survival irrespective of whether their lymphoma tumors consisted of mixed antigen-positive and negative cells or only of antigen-expressing cells. However, ifwe gave Fas ligand-blocking antibody, then those mice died sooner, Brody added.

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Bystander Killing Could Be Key Factor in CAR-T Success in Non-Hodgkin Lymphoma - Cancer Therapy Advisor

Study on plant genome editing with new variant of CRISPR-Cas9 – hortidaily.com

Alongside Dennis vanEngelsdorp, associate professor at the University of Maryland (UMD) in Entomology named for the fifth year in a row for his work in honey bee and pollinator health, Yiping Qi, associate professor in Plant Science, represented the College of Agriculture & Natural Resources on the Web of Science 2020 list of Highly Cited Researchers for the first time. Qi is already making waves in 2021 with a new high-profile publication in Nature Plants introducing SpRY, a newly engineered variant of the famed gene editing tool CRISPR-Cas9. SpRY essentially removes the barriers of what can and can't be targeted for gene editing, making it possible for the first time to target nearly any genomic sequence in plants for potential mutation.

"It is an honor, an encouragement, and a recognition of my contribution to the science community," says Qi of his distinction as a 2020 Web of Science Highly Cited Researcher. "But we are not just making contributions to the academic literature. In my lab, we are constantly pushing new tools for improved gene editing out to scientists to make an impact."

With SpRY, Qi is especially excited for the limitless possibilities it opens up for genome editing in plants and crops. "We have largely overcome the major bottleneck in plant genome editing, which is the targeting scope restrictions associated with CRISPR-Cas9. With this new toolbox, we pretty much removed this restriction, and we can target almost anywhere in the plant genome."

The original CRISPR-Cas9 tool that kicked off the gene editing craze was tied to targeting a specific short sequence of DNA known as a PAM sequence. The short sequence is what the CRISPR systems typically use to identify where to make their molecular cuts in DNA. However, the new SpRY variant introduced by Qi can move beyond these traditional PAM sequences in ways that was never possible before.

"This unleashes the full potential of CRISPR-Cas9 genome editing for plant genetics and crop improvement," says an excited Qi. "Researchers will now be able to edit anywhere within their favorable genes, without questioning whether the sites are editable or not. The new tools make genome editing more powerful, more accessible, and more versatile so that many of the editing outcomes which were previously hard to achieve can now be all realized."

According to Qi, this will have a major impact on translational research in the gene editing field, as well as on crop breeding as a whole. "This new CRISPR-Cas9 technology will play an important role in food security, nutrition, and safety. CRISPR tools are already widely used for introducing tailored mutations into crops for enhanced yield, nutrition, biotic and abiotic stress resistance, and more. With this new tool in the toolbox, we can speed up evolution and the agricultural revolution. I expect many plant biologists and breeders will use the toolbox in different crops. The list of potential applications of this new toolbox is endless."

Read the complete research at http://www.sciencedaily.com.

University of Maryland. "Plant genome editing expanded with newly engineered variant of CRISPR-Cas9: New study introduces SpRY to enable the mutation of nearly any genomic sequence in plants." ScienceDaily.

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Study on plant genome editing with new variant of CRISPR-Cas9 - hortidaily.com

Using CRISPR Genetic Technology to Catch Cancer in the Act – SciTechDaily

Phylogenetic trees, starting with an individual cancer cell. Each color represents a different location in the body. A very colorful tree shows a highly metastatic phenotype, where a cells descendants jumped many times between different tissues. A tree that is primarily one color represents a less metastatic cell. Credit: Jeffrey Quinn/Whitehead Institute

Using CRISPR technology, researchers are tracking the lineage of individual cancer cells as they proliferate and metastasize in real-time.

When cancer is confined to one spot in the body, doctors can often treat it with surgery or other therapies. Much of the mortality associated with cancer, however, is due to its tendency to metastasize, sending out seeds of itself that may take root throughout the body. The exact moment of metastasis is fleeting, lost in the millions of divisions that take place in a tumor. These events are typically impossible to monitor in real time, says Jonathan Weissman, MIT professor of biology and Whitehead Institute for Biomedical Research member.

Now, researchers led by Weissman, who is also an investigator with the Howard Hughes Medical Institute, have turned a CRISPR tool into a way to do just that. In a paper published on January 21, 2021, in Science, Weissmans lab, in collaboration with Nir Yosef, a computer scientist at the University of California at Berkeley, and Trever Bivona, a cancer biologist at the University of California at San Francisco, treats cancer cells the way evolutionary biologists might look at species, mapping out an intricately detailed family tree. By examining the branches, they can track the cells lineage to find when a single tumor cell went rogue, spreading its progeny to the rest of the body.

With this method, you can ask questions like, How frequently is this tumor metastasizing? Where did the metastases come from? Where do they go? Weissman says. By being able to follow the history of the tumor in vivo, you reveal differences in the biology of the tumor that were otherwise invisible.

Scientists have tracked the lineages of cancer cells in the past by comparing shared mutations and other variations in their DNA blueprints. These methods, however, depend to a certain extent on there being enough naturally occurring mutations or other markers to accurately show relationships between cells.

Thats where Weissman and co-first authors Jeffrey Quinn, then a postdoc in Weissmans lab, and Matthew Jones, a graduate student in Weissmans lab, saw an opportunity to use CRISPR technology specifically, a method developed by Weissman Lab member Michelle Chan to track embryo development to facilitate tracking.

Instead of simply hoping that a cancer lineage contained enough lineage-specific markers to track, the researchers decided to use Chans method to add in markers themselves. Basically, the idea is to engineer a cell that has a genomic scratchpad of DNA, that then can be written on using CRISPR, Weissman says. This writing in the genome is done in such a way that it becomes heritable, meaning a cells grand-offspring would have the writing of its parent cells and grandparent cells recorded in its genome.

To create these special scratchpad cells, Weissman engineered human cancer cells with added genes: one for the bacterial protein Cas9 the famed molecular scissors used in CRISPR genome editing methods others for glowing proteins for microscopy, and a few sequences that would serve as targets for the CRISPR technology.

They then implanted thousands of the modified human cancer cells into mice, mimicking a lung tumor (a model developed by collaborator Bivona). Mice with human lung tumors often exhibit aggressive metastases, so the researchers reasoned they would provide a good model for tracking cancer progression in real time.

As the cells began to divide, Cas9 made small cuts at these target sites. When the cell repaired the cuts, it patched in or deleted a few random nucleotides, leading to a unique repair sequence called an indel. This cutting and repairing happened randomly in nearly every generation, creating a map of cell divisions that Weissman and the team could then track using special computer models that they created by working with Yosef, a computer scientist.

Tracking cells this way yielded some interesting results. For one thing, individual tumor cells were much different from each other than the researchers expected. The cells the researchers used were from an established human lung cancer cell line called A549. Youd think they would be relatively homogeneous, Weissman says. But in fact, we saw dramatic differences in the propensity of different tumors to metastasize even in the same mouse. Some had a very small number of metastatic events, and others were really rapidly jumping around.

To find out where this heterogeneity was coming from, the team implanted two clones of the same cell in different mice. As the cells proliferated, the researchers found that their descendants metastasized at a remarkably similar rate. This was not the case with the offspring of different cells from the same cell line the original cells had apparently evolved different metastatic potentials as the cell line was maintained over many generations.

The scientists next wondered what genes were responsible for this variability between cancer cells from the same cell line. So they began to look for genes that were expressed differently between nonmetastatic, weakly metastatic, and highly metastatic tumors.

Many genes stood out, some of which were previously known to be associated with metastasis although it was not clear whether they were driving the metastasis or simply a side effect of it. One of them, the gene that codes for the protein Keratin 17, is much more strongly expressed in low metastatic tumors than in highly metastatic tumors. When we knocked down or overexpressed Keratin 17, we showed that this gene was actually controlling the tumors invasiveness, Weissman says.

Being able to identify metastasis-associated genes this way could help researchers answer questions about how tumors evolve and adapt. Its an entirely new way to look at the behavior and evolution of a tumor, Weissman says. We think it can be applied to many different problems in cancer biology.

Weissmans CRISPR method also allowed the researchers to track with more detail where metastasizing cells went in the body, and when. For example, the progeny of one implanted cancer cell underwent metastasis five separate times, spreading each time from the left lung to other tissues such as the right lung and liver. Other cells made a jump to a different area, and then metastasized again from there.

These movements can be mapped neatly in phylogenetic trees (see image), where each color represents a different location in the body. A very colorful tree shows a highly metastatic phenotype, where a cells descendants jumped many times between different tissues. A tree that is primarily one color represents a less metastatic cell.

Mapping tumor progression in this way allowed Weissman and his team to make a few interesting observations about the mechanics of metastasis. For example, some clones seeded in a textbook way, traveling from the left lung, where they started, to distinct areas of the body. Others seeded more erratically, moving first to other tissues before metastasizing again from there.

One such tissue, the mediastinal lymph tissue that sits between the lungs, appears to be a hub of sorts, says co-first author Jeffrey Quinn. It serves as a way station that connects the cancer cells to all of this fertile ground that they can then go and colonize, he says.

Therapeutically, the discovery of metastasis hubs like this could be extremely useful. If you focus cancer therapies on those places, you could then slow down metastasis or prevent it in the first place, Weissman says.

In the future, Weissman hopes to move beyond simply observing the cells and begin to predict their behavior. Its like with Newtonian mechanics if you know the velocity and position and all the forces acting on a ball, you can figure out where the ball is going to go at any time in the future, Weissman says. Were hoping to do the same thing with cells. We want to construct essentially a function of what is driving differentiation of a tumor, and then be able to measure where they are at any given time, and predict where theyre going to be in the future.

The researchers are optimistic that being able to track the family trees of individual cells in real time will prove useful in other settings as well. I think that its going to unlock a whole new dimension to what we think about as a measurable quantity in biology, says co-first author Matthew Jones. Thats whats really cool about this field in general is that were redefining whats invisible and what is visible.

Reference: Single-cell lineages reveal the rates, routes, and drivers of metastasis in cancer xenografts by Jeffrey J. Quinn, Matthew G. Jones, Ross A. Okimoto, Shigeki Nanjo, Michelle M. Chan, Nir Yosef, Trever G. Bivona and Jonathan S. Weissman, 21 January 2021, Science.DOI: 10.1126/science.abc1944

Original post:
Using CRISPR Genetic Technology to Catch Cancer in the Act - SciTechDaily

Two Gene Therapies Fix Fault in Sickle Cell Disease and -thalassemia – MD Magazine

Two different gene therapies have been used to mitigate a mechanism underlying development of sickle cell disease (SCD) and transfusion-dependent -thalassemia (TDT), and both have demonstrated clinical success in separate, concurrently published trials.

The hemoglobinopathies manifest after fetal hemoglobin synthesis is replaced by adult hemoglobin in individuals who have inherited a mutation in the hemoglobin subunit gene (HBB).Identifying factors in the conversion from fetal to adult hemoglobin synthesis, however, has provided potential targets for therapeutic intervention.

Gene therapy that can safely arrest or reduce the conversion offers the potential for a one-time treatment to obviate the need for lifetime transfusions and iron chelation for patients with TDT, and the pain management, transfusions and hydroxyurea administration for those with SCD.

Two groups of investigators have now reported in The New England Journal of Medicine that, using different gene therapy techniques that target the transcription factor, BCL11a, involved in the globin switching, they have improved clinical outcomes in patients with TDT and with SCD.

In an editorial in the issue featuring the 2 studies, Mark Walters, MD, Blood and Marrow Transplant Program, University of California, San Francisco-Benioff Children's Hospital, welcomed the breakthroughs.

"These trials herald a new generation of broadly applicable curative treatments for hemoglobinopathies," Walters wrote.

In one clinical trial with 2 patients, one with TDT and the other with SCD, Haydar Frangoul, MD, MS, Medical Director, Pediatric Hematology/Oncology, Sarah Cannon Center for Blood Cancer at the Children's Hospital at Tristar Centennial, and colleagues administered CRISPR-Cas9 gene edited hematopoietic stem and progenitor cells (HSPCs) with reduced BCL11A expression in the erythroid lineage.

The product, CTX001, had been shown in preclinical study to restore -globulin synthesis and reactivate production of fetal hemoglobin. Both patients underwent busulfan-induced myeloablation prior to receiving the treatment.

The investigators suggested that the CRISPR-Cas9-based gene-edited product could change the paradigm for patients with these conditions, if it was found to successfully and durably graft, produce no "off-target" editing products, and, importantly, improve clinical course.

"Recently approved therapies, including luspatercept and crizanlizumab, have reduced transfusion requirements in patients with TDT and the incidence of vaso-occlusive episodes in those with SCD, respectively, but neither treatment addressed the underlying cause of the disease nor fully ameliorates disease manifestations," Frangoul and colleagues wrote.

The investigators reported that both patients had "early, substantial, and sustained increases" in pancellularly distributed fetal hemoglobin levels during the 12-month study period. Further, the patients no longer required transfusions, and the patient with SCD no longer experienced vaso-occlusive episodes after the treatment.

In commentary accompanying the report, Harry Malech, MD, Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Disease (NIAID), National Institutes of Health (NIH), Bethesda, MD, described the investigators' application of the gene-editing technology as a "remarkable level of functional correction of the disease phenotype."

"With tangible results for their patients, Frangoul et al have provided a proof of principle of the emerging clinical potential for gene-editing treatments to ameliorate the burden of human disease," Malech pronounced.

In the other published trial, with 6 patients with SCD, Erica Esrick MD, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, and colleagues described results with infusion of gene-modified cells derived from lentivirus insertion of a gene that knocks down BCL11a by encoding an erythroid-specific, inhibitory short-hairpin RNA (shRNA).

The severity of SCD that qualified patients for enrollment included history of stroke (n = 3), frequent vaso-occlusive events (n = 2) and frequent episodes of priapism (1).Patients were followed for 2 years, and offered enrollment in a 13-year long-term follow-up study.The infusion of the experimental drug BCH-BB694, from the short hairpin RNA embedded within an endogeonous micro RNA scaffold (termed a shmiR vector), was initiated after myeloablation with busulfan.

Esrick and colleagues reported that, at median follow-up of 18 months (range, 7-29), all patients had engraftment and a robust and stable HbF induction broadly distributed in red cells.Clinical manifestations of SCD were reduced or absent during the follow-up period; with no patient having a vaso-occlusive crisis, acute chest syndrome, or stoke subsequent to the gene therapy infusion.Adverse events were consistent with effects of the preparative chemotherapy.

"The field of autologous gene therapies for hemoglobinopathies is advancing rapidly," Esrick and colleagues reported, "including lentiviral trials of gene addition in which the nonsickling hemoglobin is formed from an exogenous -globin or modified -globin gene."

Walters agreed that gene therapy is rapidly progressing, but expressed concern about the large gap that looms between laboratory bench and clinical bedside, particularly for this affected population.

"Access to and delivery of these highly technical therapies in patients with sickle cell disease will be challenging and probably limited to resource-rich nations, at least in the short term," Walters commented.

The studies, CRISPR-Cas9 Gene Editing for Sickle Cell Disease and -Thalassemia, as well as, Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease, were published online in The New England Journal of Medicine.

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Two Gene Therapies Fix Fault in Sickle Cell Disease and -thalassemia - MD Magazine

Comprehensive Report on Crispr And Crispr-Associated (Cas) Genes Market 2021 | Trends, Growth Demand, Opportunities & Forecast To 2027 | Intellia…

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Crispr And Crispr-Associated (Cas) Genes Market is growing at a High CAGR during the forecast period 2021-2027. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

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Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Crispr And Crispr-Associated (Cas) Genes market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Crispr And Crispr-Associated (Cas) Genes markets trajectory between forecast periods.

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Genome EditingGenetic engineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem Cells

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Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

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Regions Covered in the Global Crispr And Crispr-Associated (Cas) Genes Market Report 2021:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The cost analysis of the Global Crispr And Crispr-Associated (Cas) Genes Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

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Global Crispr And Crispr-Associated (Cas) Genes Market Research Report 2021 2027

Chapter 1 Crispr And Crispr-Associated (Cas) Genes Market Overview

Chapter 2 Global Economic Impact on Industry

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Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

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Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Crispr And Crispr-Associated (Cas) Genes Market Forecast

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Comprehensive Report on Crispr And Crispr-Associated (Cas) Genes Market 2021 | Trends, Growth Demand, Opportunities & Forecast To 2027 | Intellia...

Natural CRISPR’s Safety Feature Could Become Genetic Dimmer Switch – Genetic Engineering & Biotechnology News

The CRISPR systems inside bacteria serve as adaptive immune systems, but they also threaten to unleash autoimmune reactions. Fortunately, for bacteria such as Streptococcus pyogenes, these systems have a built-in safety feature: a long-form transactivating CRISPR RNA (tracrRNA). Unlike the short-form tracrRNA, which together with CRISPR RNA (crRNA) complexes with the CRISPR-Cas9 enzyme and guides it to DNA sites where it executes cuts, the long-form tracrRNA guides the enzyme to the enzymes own genetic promoter.

The long-form tracrRNA that complexes CRISPR-Cas9 enzyme doesnt need to bind to crRNA, and it doesnt cut. Instead, it merely lingers in place, preventing gene expression.

Essentially, long-form tracrRNA acts as a safety feature, dialing down a bacteriums immune system to prevent it from attacking the bacterium itself rather than foreign DNA. This self-protection function for long-form tracrRNA was uncovered by researchers at Johns Hopkins University. The researchers, led by Joshua W. Modell, PhD, also explored whether long-form tracrRNA could be reprogrammed to guide CRISPR-Cas9 to DNA sites other than the CRISPR-Cas9 promoter.

The researchers findings appeared in the journal Cell, in an article titled, A natural single-guide RNA repurposes Cas9 to autoregulate CRISPR-Cas expression. According to the researchers, long-form tracrRNA could serve as a programmable genetic dimmer switch, one that could be used to inhibit the expression of designated genes in research applications.

We show that in the S. pyogenes CRISPR-Cas system, a long-form transactivating CRISPR RNA folds into a natural single guide that directs Cas9 to transcriptionally repress its own promoter (Pcas), the articles authors wrote. Further, we demonstrate that Pcas serves as a critical regulatory node.

Scientists have long worked to unravel the precise steps of CRISPR-Cas9s mechanism and how its activity in bacteria is dialed up or down. Looking for genes that ignite or inhibit the CRISPR-Cas9 gene-cutting system for the common, strep-throat causing bacterium S. pyogenes, the Johns Hopkins scientists found a clue regarding how that aspect of the system works.

Specifically, the scientists found a gene in the CRISPR-Cas9 system that, when deactivated, led to a dramatic increase in the activity of the system in bacteria. The product of this gene appeared to re-program Cas9 to act as a brake, rather than as a scissor, to dial down the CRISPR system.

From an immunity perspective, bacteria need to ramp up CRISPR-Cas9 activity to identify and rid the cell of threats, but they also need to dial it down to avoid autoimmunitywhen the immune system mistakenly attacks components of the bacteria themselves, said graduate student Rachael Workman, a bacteriologist working in Modells laboratory.

To further nail down the particulars of the brake, the teams next step was to better understand the product of the deactivated gene, a tracrRNA. tracrRNAs belong to a unique family of RNAs that do not make proteins. Instead, they act as a kind of scaffold that allows the Cas9 enzyme to carry the guide RNA that contains a mug shot of previously encountered phage DNA. The mug shot allows Cas9 to cut matching DNA sequences in newly invading viruses.

tracrRNA comes in two sizes: long and short. Most of the modern gene-cutting CRISPR-Cas9 tools use the short form. However, the research team found that the deactivated gene product was the long-form of tracrRNA, the function of which has been entirely unknown.

In bacteria, DNA-cutting CRISPR-Cas9 complexes typically consist of a Cas9 enzyme and a guide RNA. The guide RNA consists of a short-form transactivating CRISPR RNA (tracrRNA) scaffold and a DNA-sequence-specific CRISPR (crRNA). Long-form tracrRNA, however, can complex with and guide Cas9 without crRNA. When long-form tracrRNA does so, it guides the Cas9 enzyme to a Cas9 promoter. The promoter is not cut, but expression is repressed. Left: A schematic of the long-form of the tracrRNA used by the CRISPR-Cas9 system in bacteria. Right: the standard guide RNA used by many scientists as part of the gene-cutting CRISPR-Cas9 system. (Often, the guide RNA is a single synthetic molecule, rather than a combination of tracrRNA and crRNA.) [Joshua Modell and Rachael Workman, Johns Hopkins Medicine]The long and short forms of tracrRNA are similar in structure and have in common the ability to bind to Cas9. The short-form tracrRNA also binds to the guide RNA. However, the long-form tracrRNA doesnt need to bind to the crRNA, because it contains a segment that mimics the crRNA. Essentially, long-form tracrRNAs have combined the function of the short-form tracrRNA and crRNA, explained Modell, assistant professor of molecular biology and genetics at the Johns Hopkins University School of Medicine.

The researchers used genetic engineering to alter the length of a certain region in long-form tracrRNA to make the tracrRNA appear more like a guide RNA. They found that with the altered long-form tracrRNA, Cas9 once again behaved more like a scissor.

Other experiments showed that in lab-grown bacteria with a plentiful amount of long-form tracrRNA, levels of all CRISPR-related genes were very low. When the long-form tracrRNA was removed from bacteria, however, expression of CRISPR-Cas9 genes increased a hundredfold.

Bacterial cells lacking the long-form tracrRNA were cultured in the laboratory for three days and compared with similarly cultured cells containing the long-form tracrRNA. By the end of the experiment, bacteria without the long-form tracrRNA had completely died off. De-repression causes a dramatic 3,000-fold increase in immunization rates against viruses, the articles authors noted. However, heightened immunity comes at the cost of increased autoimmune toxicity.

These findings suggest that long-form tracrRNA normally protects cells from the sickness and death that happen when CRISPR-Cas9 activity is very high. We started to get the idea that the long form was repressing but not eliminating its own CRISPR-related activity, recalled Workman.

To see if the long-form tracrRNA could be re-programmed to repress other bacterial genes, the research team altered the long-form tracrRNAs spacer region to let it sit on a gene that produces green fluorescence. Bacteria with this mutated version of long-form tracrRNA glowed less green than bacteria containing the normal long-form tracrRNA, suggesting that the long-form tracrRNA can be genetically engineered to dial down other bacterial genes.

Another research team, from Emory University, found that in the parasitic bacteria Francisella novicida, Cas9 behaves as a dimmer switch for a gene outside the CRISPR-Cas9 region. The CRISPR-Cas9 system in the Johns Hopkins study is more widely used by scientists as a gene-cutting tool, and the Johns Hopkins teams findings provide evidence that the dimmer action controls the CRISPR-Cas9 system in addition to other genes.

Using bioinformatic analyses, we provide evidence that tracrRNA-mediated autoregulation is widespread in type II-A CRISPR-Cas systems, the Johns Hopkins scientists added. Collectively, we unveil a new paradigm for the intrinsic regulation of CRISPR-Cas systems by natural single guides, which may facilitate the frequent horizontal transfer of these systems into new hosts that have not yet evolved their own regulatory strategies.

The researchers also found the genetic components of long-form tracrRNA in about 40% of the Streptococcus group of bacteria. Further study of bacterial strains that dont have the long-form tracrRNA, said Workman, will potentially reveal whether their CRISPR-Cas9 systems are intact, and other ways that bacteria may dial back the CRISPR-Cas9 system.

The dimmer capability that the experiments uncovered offers opportunities to design new or better CRISPR-Cas9 tools aimed at regulating gene activity for research purposes. In a gene editing scenario, Modell suggested, a researcher may want to cut a specific gene, in addition to using the long-form tracrRNA to inhibit gene activity.

Excerpt from:
Natural CRISPR's Safety Feature Could Become Genetic Dimmer Switch - Genetic Engineering & Biotechnology News

CRISPR (CRSP) Up More Than 80% in Past 3 Months: Here’s Why – Yahoo Finance

TipRanks

Big Tech has been in the news lately, and not necessarily for the right reasons. Accusations of corporate censorship have hit the headlines in recent weeks. While serious, this may have a salutary effect the public discussion of Big Techs role in our digital lives is long overdue. And that discussion will get underway just as the Q4 and full-year 2020 financial numbers start coming in. Of the FAANG stocks, Netflix has already reported; the other four will release results in the next two weeks. So, the upcoming earnings will garner well-deserved attention, and Wall Streets best analysts are already publishing their views on some of the markets most important components. Using TipRanks database, we pulled up the details on two members of the FAANG club to find out how the Street thinks each will fare when they publish their fourth quarter numbers. According to the platform, both have received plenty of love from the analysts, earning a Strong Buy consensus rating. Facebook (FB) Lets start with Facebook, the social media giant that has redefined our online interactions. Along with Google, Facebook has also brought us targeted digital marketing and advertising, and the mass monetization of the internet. Its been a profitable strategy for the company. Facebooks market cap is up to $786 billion, and in the third quarter of 2020, the company reported $21.5 billion at the top line. Looking ahead to the Q4 report, due out on January 27, analysts are forecasting revenues at or near $26.2 billion. This would be in-line with the companys pattern, of rising quarterly performance from Q1 to Q4. At the predicted sum, revenues would rise 24% year-over-year, roughly congruent with the 22% yoy gain already seen in Q3. The key metric to watch out for will be the growth in daily active users; this metric slipped slightly from Q2 to Q3, and further decline will be taken as an ominous sign for the companys future. As it stands now, Facebooks daily average user number is 1.82 billion. Ahead of the print, Oppenheimer analyst Jason Helfstein boosted his price target to $345 (from $300), while reiterating an Outperform (i.e. Buy) rating. Investors stand to pocket ~26% gain should the analyst's thesis play out. (To watch Helfsteins track record, click here) The 5-star analyst commented, "[We] anticipate 4Q advertising revenue will handily top Street estimates. We now forecast 4Q advertising revenue +30% y/y vs. Street's +25% estimate based on a regression of US Standard Media Index Data (r-squared 0.95) and accelerating global CPM data from Gupta Media (4Q +35% y/y vs. 3Q's -12%). Additionally, we are very bullish on FB's eCommerce opportunity following conversations with our checks and our initial work conservatively estimating Shops is a $2550B opportunity vs. current $85B revs. We believe shares currently trading at 7.1x EV/NTM sales offers the most favorable risk/ reward in internet large cap." Overall, the social media empire remains a Wall Street darling, as TipRanks analytics showcasing FB as a Strong Buy. This is based on 34 recent reviews, which break down to 30 Buy ratings, 3 Holds, and 1 Sell. Shares are priced at $276.10 and the average price target of $327.42 suggests a one-year upside of ~19%. (See FB stock analysis on TipRanks) Amazon (AMZN) Turning to e-commerce, we cant avoid Amazon. The retail giant has a market cap of $1.65 trillion, making it one of just four publicly traded companies valued over the trillion-dollar mark. The companys famously price is famously high, and has grown 74% since this time last year, far outpacing the broader markets. Amazons growth has been supported by increased online sales activity during the corona year. Globally, online retail has grew 27% in 2020, while total retail slipped 3%. Amazon, which dominates the online retail sector, is projected to end 2020 with $380 billion in total revenue, or 34% year-over-year growth, outpacing the global e-commerce gains. Cowen analyst John Blackledge, rating 5-stars by TipRanks, covers Amazon and is bullish on the companys prospects ahead of the earnings release. Blackledge rates the stock Outperform (i.e. Buy), and his price target, at $4,350, indicates confidence in a 31% upside on the one-year time horizon. (To watch Blackledges track record, click here) We forecast 4Q20 reported revenue of $120.8BN, +38.2% y/y vs. +37.4% y/y in 3Q20 led by AWS, advertising, subscription and 3P sales [..] We estimate US Prime sub growth accelerated in 4Q20 (reaching 76MM subs in Dec '20 and ~74MM on avg in 4Q20), helped by pandemic demand, Prime Day in Oct, & elongated shopping period, as well as 1 Day delivery [...] In '21, we expect strong top-line growth to continue driven by eCommerce (helped by COVID pull forward in Grocery), adv., AWS & sub businesses," Blackledge opined. That Wall Street generally is bullish on Amazon is no secret; the company has 33 reviews on record, and 32 of them are Buys, versus 1 Hold. Shares are priced at $3,301.26 and the average price target of $3,826 implies that it will grow another 16% this year. (See AMZN stock analysis on TipRanks) To find good ideas for stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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CRISPR (CRSP) Up More Than 80% in Past 3 Months: Here's Why - Yahoo Finance

The Zacks Analyst Blog Highlights: CRISPR Therapeutics, Vertex Pharmaceuticals and Intellia Therapeutics – Yahoo Finance

For Immediate Release

Chicago, IL January 20, 2021 Zacks.com announces the list of stocks featured in the Analyst Blog. Every day the Zacks Equity Research analysts discuss the latest news and events impacting stocks and the financial markets. Stocks recently featured in the blog include: CRISPR Therapeutics AG CRSP, Vertex Pharmaceuticals Incorporated VRTX and Intellia Therapeutics, Inc. NTLA.

Shares ofCRISPR Therapeutics have rallied 87.3% in the past three months compared with theindustry'sincrease of 15.4%.

The company has made rapid progress with the development of its lead pipeline candidate, CTX001. The candidate is an investigational ex vivo CRISPR gene-edited therapy, which is currently being developed for treating sickle cell disease ("SCD") and transfusion-dependent beta thalassemia ("TDT") in partnership withVertex Pharmaceuticals.

In December 2020, the companiesannouncedpromising additional data on CTX001, which demonstrated a consistent and sustained response in treating patients with SCD and TDT. Treatment with CTX001 showed that all seven patients with TDT remained transfusion independent until the last follow-up, while all three patients with SCD were free of vaso-occlusive crises through the last follow-up.

Both diseases have a significant unmet medical need, and if successfully developed and commercialized, the candidate can lend a huge boost to CRISPR Therapeutics' prospects.

Notably, CTX001 has been granted Regenerative Medicine Advanced Therapy, Fast Track, and Orphan Drug designations by the FDA for both TDT and SCD. The European Commission has granted Orphan Drug Designation to the gene therapy candidate for both indications.

Genomic editing to repair a defective genetic material that causes diseases using CRISPR technology is probably one of the most promising and exciting healthcare innovations seen in decades. The technology has the potential to change how diseases, especially those caused by genetic mutations, are treated.

Story continues

Though the market holds great potential, competition remains stiff in the space. Other companies likeIntellia Therapeutics are also engaged in developing candidates to address different indications using CRISPR/Cas9 gene-editing technology.

CRISPR Therapeutics is also developing three gene-edited allogeneic cell therapy programs, chimeric antigen receptor T cell (CAR-T) candidates, CTX110, CTX120 and CTX130 for the treatment of hematological and solid tumor cancers. Several data readouts on the above candidates are expected in the ongoing year and a positive outcome might drive the stock further up in the days ahead.

CRISPR Therapeutics currently carries a Zacks Rank #3 (Hold). You can seethe complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

Each was hand-picked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2020. Each comes from a different sector and has unique qualities and catalysts that could fuel exceptional growth.

Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.

Today, See These 5 Potential Home Runs >>

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Past performance is no guarantee of future results. Inherent in any investment is the potential for loss. This material is being provided for informational purposes only and nothing herein constitutes investment, legal, accounting or tax advice, or a recommendation to buy, sell or hold a security. No recommendation or advice is being given as to whether any investment is suitable for a particular investor. It should not be assumedthat any investments in securities, companies, sectors or markets identified and described were or will be profitable. All information is current as of the date of herein andis subject to change without notice. Any views or opinions expressed may not reflect those of the firm as a whole. Zacks Investment Research does not engage in investment banking, market making or asset management activities of any securities. These returns are from hypothetical portfolios consisting of stocks with Zacks Rank = 1 that were rebalanced monthly with zero transaction costs. These are not the returns of actual portfolios of stocks. The S&P 500 is an unmanaged index. Visit https://www.zacks.com/performance for information about the performance numbers displayed in this press release.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportVertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis ReportIntellia Therapeutics, Inc. (NTLA) : Free Stock Analysis ReportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research

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The Zacks Analyst Blog Highlights: CRISPR Therapeutics, Vertex Pharmaceuticals and Intellia Therapeutics - Yahoo Finance

Investors Who Bought CRISPR Therapeutics (NASDAQ:CRSP) Shares Three Years Ago Are Now Up 411% – Simply Wall St

Generally speaking, investors are inspired to be stock pickers by the potential to find the big winners. Not every pick can be a winner, but when you pick the right stock, you can win big. One such superstar is CRISPR Therapeutics AG (NASDAQ:CRSP), which saw its share price soar 411% in three years. On top of that, the share price is up 115% in about a quarter.

Check out our latest analysis for CRISPR Therapeutics

CRISPR Therapeutics wasn't profitable in the last twelve months, it is unlikely we'll see a strong correlation between its share price and its earnings per share (EPS). Arguably revenue is our next best option. Generally speaking, companies without profits are expected to grow revenue every year, and at a good clip. Some companies are willing to postpone profitability to grow revenue faster, but in that case one does expect good top-line growth.

CRISPR Therapeutics' revenue trended up 81% each year over three years. That's much better than most loss-making companies. In light of this attractive revenue growth, it seems somewhat appropriate that the share price has been rocketing, boasting a gain of 72% per year, over the same period. Despite the strong run, top performers like CRISPR Therapeutics have been known to go on winning for decades. In fact, it might be time to put it on your watchlist, if you're not already familiar with the stock.

The company's revenue and earnings (over time) are depicted in the image below (click to see the exact numbers).

CRISPR Therapeutics is a well known stock, with plenty of analyst coverage, suggesting some visibility into future growth. So it makes a lot of sense to check out what analysts think CRISPR Therapeutics will earn in the future (free analyst consensus estimates)

Pleasingly, CRISPR Therapeutics' total shareholder return last year was 241%. So this year's TSR was actually better than the three-year TSR (annualized) of 72%. The improving returns to shareholders suggests the stock is becoming more popular with time. While it is well worth considering the different impacts that market conditions can have on the share price, there are other factors that are even more important. Case in point: We've spotted 3 warning signs for CRISPR Therapeutics you should be aware of.

If you would prefer to check out another company -- one with potentially superior financials -- then do not miss this free list of companies that have proven they can grow earnings.

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

PromotedIf you decide to trade CRISPR Therapeutics, use the lowest-cost* platform that is rated #1 Overall by Barrons, Interactive Brokers. Trade stocks, options, futures, forex, bonds and funds on 135 markets, all from a single integrated account.

This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. *Interactive Brokers Rated Lowest Cost Broker by StockBrokers.com Annual Online Review 2020

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Investors Who Bought CRISPR Therapeutics (NASDAQ:CRSP) Shares Three Years Ago Are Now Up 411% - Simply Wall St

We may have a CRISPR cure for red blood diseases sickle cell anemia and beta thalassemia – Genetic Literacy Project

Sickle cell anemia and thalassemia are genetic diseases that result in the production of anomalous hemoglobin (protein that carries oxygen) and deformed red blood cells. There is no cure for these ailments, but ten patients who have had their genes edited are on their way to get rid of them, thanks to the Clustered Regularly Interspaced Short Palindromic Repeats technique or CRISPR.

It is possible to edit human cells and safely infuse them in patients; this treatment has totally changed their lives, said haematologist Haydar Frangoul of the Sarah Cannon Research Institute. He is the doctor accompanying the studys first volunteer, the housewife and mother of three children Victoria Gray.

The work consisted of activating the generation of fetal hemoglobin, which is still produced in the womb and which results in healthy red blood cells. When the baby is born, the gene turns off and, in patients with thalassemia and sickle cell anemia, the result is the production of anomalous hemoglobin.

To receive the stem cells edited by CRISPR, patients first had to go through a painful stage: numerous rounds of chemotherapyAfter all the stem cells that produced the anomalous hemoglobin were destroyed, those edited were infused into the patients to reproduce and manufacture fetal hemoglobin.

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We may have a CRISPR cure for red blood diseases sickle cell anemia and beta thalassemia - Genetic Literacy Project

The ARKG ETF: Join the Genomics Revolution – ETF Trends

The genomics space is rapidly innovating. The ARK Genomic Revolution Multi-Sector Fund (CBOE: ARKG) makes accessing innovation much easier.

For investors, ARKGs active management and utility are vital because the fund is flexible and able to capitalize on genomics advancements more rapidly than index-based rivals. Those advancements include gene editing.

Crispr-Cas9 is the second generation of technologies that seek to repair thousands of inherited genetic disorders and battle cancer in new ways. Gene editing is advancing so quickly that next-generation technologies are already on the heels of Crispr-Cas9, including a more-precise tool called base editing, reports Bill Alpert for Barrons.

The CRISPR technology may also be under the spotlight as another disease fighting tool, with the world refocusing on the need for improved healthcare solutions.

The ARK Genomic Revolution ETF tracks the convergence of tech and healthcare. The underlying components are expected to substantially benefit from extending and enhancing the quality of human and other life by incorporating technological and scientific developments and advancements in genomics into their business.

While gene-editing start-ups will lose money during years of clinical trials, its hard to say the stocks are overvalued. If their one-time interventions can cure diseases that otherwise require chronic treatmentor lack any treatment at allthen the stocks will fly, according to Barrons.

That speaks to a big advantage with ARKG: investors dont have to stock pick in the gene editing arena.

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

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

Because gene editing permanently changes the genome, it doesnt appear to suffer from these issues. Nature evolved many tools to cut DNA at specific spots in the genome, addsBarrons.

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

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

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The ARKG ETF: Join the Genomics Revolution - ETF Trends

Gene-edited crops are now a reality but will the public be on board? – The Conversation UK

Once the UK left the EU, it would be free to invest in gene editing of crops and livestock to feed the world. Thats what the prime minister, Boris Johnson, told the House of Commons in 2019. And following the UKs formal departure from the EU in January 2021, the government quickly launched a public consultation on the issue.

Yet media reporting might cause plant scientists to have unpleasant flashbacks to the 1990s, when genetically modified (or GM) crops were first being commercialised in Europe. Some of the language used to report on the consultation is eerily similar: the Daily Mail asks its readers whether Frankenstein food is about to hit UK plates. Two decades ago, GM crops were also labelled Frankenfood.

Whereas GM crops typically contain the DNA of two different species, gene editing is more precise and allows scientists to tweak the DNA of a single species by itself. Today, many plant scientists see a clear difference between first-generation genetic modifications and the new plant breeding techniques of gene editing. These include tools like CRISPR, which can be used like genetic scissors to make changes to a plant that mimic natural variation.

In the US and Canada, for example, a non-browning mushroom has found a quick path to market thanks to breeders ability to knock-out the gene that controls the browning enzyme, improving shelf-life and potentially minimising food waste.

Although this was done in a laboratory, natural processes at the genetic level and in response to environmental conditions turn genes on and off in a similar fashion. These tools have health applications, too. CRISPR is being used to treat cancer and has the potential for many more medical applications.

Because gene-edited plants can be indistinguishable from their conventional cousins unlike GM crops countries around the world are grappling with how they should be regulated. In the European Union, a landmark 2018 ruling by the Court of Justice said that new gene-edited crops should be governed by existing legislation that was developed in response to first-generation GM crops and said that if you breed something that could not occur in nature, it counts as genetically-modified.

However, this does not mean as was widely reported that gene-edited crops are automatically GM crops, which by definition could not occur in nature. The EU, like the UK, is now revisiting this issue through a consultation.

As recipients of European plant science funding, we have seen that scientists and the public often talk past one another on the issue of biotechnology. Scientists, for their part, tend to view it in terms of risk (or lack thereof) and invoke humanitys long history of modifying plants for our own purposes. But we need to move beyond this framework and instead take account of the questions and concerns that the general public has about who benefits from this technology, who owns it and what impacts it will have.

First-generation genetic modification tended to focus on farm productivity. Protecting crops from pests was the top priority. Gene-edited crops could contribute to a wider variety of sustainability and health goals in future though, such as by improving nutrition or using resources more efficiently. In fact, a whole raft of technologies could be about to revolutionise the way we make food.

However, as we learned with GM crops, technologies are most effective when the wider public and key stakeholders, such as farmers, are actively included in their development.

There is greater and greater recognition among researchers and policymakers of the need to ensure that new technology meets the needs, expectations and values of the public. We have seen that the involvement of patients can make new health technologies more relevant and effective. Already, there is more talk of democratising new genomic tools like CRISPR.

So although plant scientists will hope to avoid repeating the same debates about biotechnology that they had two decades ago, there is still opportunity to gain public trust in these technologies through active and open dialogue. We must ask ourselves whether the gene editing consultation goes far enough to gain that trust, particularly for those that see this as Frankenstein-like technology.

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Gene-edited crops are now a reality but will the public be on board? - The Conversation UK

‘Incredible’ gene-editing result in mice inspires plans to treat premature-aging syndrome in children – Science Magazine

A 4-year-old with progeria, a syndrome with features of premature aging that stems from a mutated gene

By Jocelyn KaiserJan. 6, 2021 , 11:00 AM

One mouse is hunched over, graying, and barely moves at 7 months old. Others, at 11 months, have sleek black coats and run around. The videos and other results from a new study have inspired hope for treating children born with progeria, a rare, fatal, genetic disease that causes symptoms much like early aging. In mice with a progeria-causing mutation, a cousin of the celebrated genome editor known as CRISPR corrected the DNA mistake, preventing the heart damage typical of the disease, a research team reports today in Nature. Treated mice lived about 500 days, more than twice as long as untreated animals.

The outcome is incredible, says gene-therapy researcher Guangping Gao of the University of Massachusetts, who was not involved with the study.

Although the developers of the progeria therapy aim to improve it, they are also taking steps toward testing the current version in affected children, and some other scientists endorse a rush. The mouse results are beyond anyones wildest expectations, says Fyodor Urnov, a gene-editing researcher at the University of California, Berkeley. The new data are an imperative to treat a child with progeria and do so in the next 3 years.

About 400 people in the world are estimated to have Hutchinson-Gilford progeria syndrome, which results from a single-base change in the gene for a protein called lamin A that helps support the membrane forming the nucleus in cells. The resulting abnormal protein, called progerin, disrupts the nuclear membrane and is toxic to cells in many tissues. Toddlers soon become bald and have stunted growth, body fat loss, stiff joints, wrinkled skin, osteoporosis, and atherosclerosis. People with progeria die on average around age 14 from a heart attack or stroke.

Researchers have previously used CRISPR to disrupt activity of the mutated gene for lamin A in progeria mice. But their health improved only modestly, and disabling a persons good copy of the gene could cause harm. So David Liu of Harvard University and the Broad Institute turned to base editing, a DNA-changing method originally inspired by CRISPR and developed in his lab. Unlike CRISPR, which makes double-strand cuts in DNA, the base editor used in the progeria study nicks just one strand and swaps out a single base. Base editors have treated liver, eye, ear, blood, and brain disorders in mice, and Liu wanted to try one on an infamous and devastating disease that involves multiple organs or tissues.

Lius group teamed up with Vanderbilt University cardiologist Jonathan Brown and Francis Collins, director of the National Institutes of Health, whose group was one of two that identified the progeria mutation in 2003. The team first tested the base-editing approach in cultured cells from two progeria patients, finding that it corrected the mutation while making few unwanted changes elsewhere in the genome. They then packaged DNA encoding the base editor into adeno-associated viruses (AAVs), a standard delivery vehicle for gene therapies, and injected these into young mice with the progeria mutation.

The results were far better than we had dared to hope, Collins says. When the mice were examined 6 months later,between 20% and 60% of their bone, skeletal muscle, liver, heart, and aorta carried the DNA fix. Progerin levels fell and lamin A levels rose in several tissues. Even though the mice were already 2 weeks old when treated, or about age 5 in human years, their aortas months later bore virtually no signs of the fibrous tissue growth or loss of smooth muscle cells seen in mice and children with progeria. It hits home the potential of this technology, says gene-editing researcher Charles Gersbach of Duke University.

Some of the rodents eventually developed liver tumors, a problem seen before in mice receiving high-dose AAV gene therapy. No people have been shown to have developed liver tumors as a result of such treatments. Still, lowering the AAV dose to improve safety is a goal, Liu says. He and Collins are evaluating more efficient base editors to that end.

Study co-author Leslie Gordon, a Brown University physician whose son died from progeria and who co-founded the Progeria Research Foundation, doesnt want to wait for the next iteration before developing plans and raising money to test the treatment in children. Her foundation is talking to companies, including Beam Therapeutics, which Liu co-founded, in hopes of launching a clinical trial. We will find a way to get this done for these kids, Gordon says.

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'Incredible' gene-editing result in mice inspires plans to treat premature-aging syndrome in children - Science Magazine

CRISPR Therapeutics Is Still Looking Strong – RealMoney – RealMoney

During Tuesday's Mad Money program Jim Cramer cautioned viewers against the CRISPR stocks hoping that human genome editing will usher in the cure for cancer and other ailments. Genomics is indeed a hot science, he said, but it's also incredibly risky. Let's take a look at the charts of CRISPR Therapeutics AG (CRSP) again.

On Dec. 22 we looked at CRSP and concluded that, "if you are still long CRSP then you are way smarter than me. Longs should risk to $145. The round number of $200 and then the Point and Figure target of $230 are the price objectives for now."

In this updated daily bar chart of CRSP, below, we can see that prices remain in a strong uptrend above the rising 50-day moving average line and the rising 200-day moving average line. The On-Balance-Volume (OBV) line has moved higher to confirm the price gains while the Moving Average Convergence Divergence (MACD) is above the zero line and poised for a new buy signal.

In this weekly bar chart of CRSP, below, we can see a 2-1/2-year base pattern in place before the strong gains of this year. Prices are in an uptrend above the rising 40-week moving average line. The weekly OBV line is strong and so is the MACD oscillator.

In this daily Point and Figure chart of CRSP, below, we can see a price target of $200.

In this second Point and Figure chart of CRSP, below, we used weekly close-only price data with a five-box reversal filter. Here our $230 price target is confirmed.

Bottom-line strategy:Continue to hold longs risking to $149 now. Our price targets are $200 and $230 for now.

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CRISPR Therapeutics Is Still Looking Strong - RealMoney - RealMoney

Global CRISPR Technology Market Report 2020: COVID-19 Growth and Change – Market is Expected to Recover to Reach $1.55 Billion in 2023 – Forecast to…

DUBLIN, Jan. 6, 2021 /PRNewswire/ -- The "CRISPR Technology Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

CRISPR Technology Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global crispr technology market.

Major players in the CRISPR technology market are Thermo Fisher Scientific, GenScript Biotech Corporation, CRISPR Therapeutics AG, Editas Medicine, Horizon Discovery Plc., Integrated DNA Technologies, Inc. (Danaher), Origene Technologies, Inc., Transposagenbio Biopharmaceuticals (Hera Biolabs), Intellia Therapeutics Inc., and GeneCopoeia, Inc.

The global CRISPR technology market is expected to increase from $0.76 billion in 2019 to $0.92 billion in 2020 at a compound annual growth rate (CAGR) of 20.91%. The exponential growth is mainly due to the COVID-19 outbreak that has led to the research for drugs for COVID-19 with gene-editing using CRISPR technology. The market is expected to reach $1.55 billion in 2023 at a CAGR of 19.13%.

The CRISPR technology market consists of sales of CRISPR technology products and services which is a gene-editing technology that allows researchers to alter DNA sequences and modify gene function. The revenue generated by the market includes the sales of products such as design tools, plasmid & vector, Cas9 & gRNA, libraries & delivery system products and services that include design & vector construction, screening and cell line engineering.

These products and services are used in genome editing/genetic engineering, genetically modifying organisms, agricultural biotechnology and others which include gRNA database/gene library, CRISPR plasmid, human stem cell & cell line engineering by end-users. The end-users include pharmaceutical & biopharmaceutical companies, biotechnology companies, academic & research institutes and contract research organizations.

North America was the largest region in the CRISPR technology market in 2019. Europe was the second-largest region in the CRISPR technology market in 2019.

In 2019, Cardea Bio Inc., a US-based biotechnology infrastructure company that manufactures biology-gated transistors (Cardean transistors) that utilizes biocompatible graphene instead of silicon and replaces optical signal observations with direct electrical molecular signal analysis, merged with Nanosens Innovations, Inc. The merger is aimed at accelerating the development of the genome sensor that combines Nanosens' CRISPR-Chip technology with Cardea's graphene biosensor infrastructure and is the first DNA search engine globally that runs on CRISPR-Chip technology. Nanosens will be operating as a subsidiary of Cardea Bio. Nanosens Innovations, Inc. is a US-based biotechnology company that develops CRISPR-Chip and FEB technology.

The CRISPR technology market covered in this report is segmented by product type into design tools; plasmid and vector; CAS9 and G-RNA; delivery system products. It is also segmented by application into genome editing/ genetic engineering; genetically modified organisms; agricultural biotechnology; others and by end-user into industrial biotech; biological research; agricultural research; therapeutics and drug discovery.

Stringent government regulations are expected to retard the growth of the CRISPR technology market during the period. There is no existence of internationally agreed regulatory framework for gene editing products and countries are in the process of evaluating whether and to what extent current regulations are adequate for research conducted with gene editing and applications and products related to gene editing. In July 2018, the Court of Justice of the European Union ruled that it would treat gene-edited crops as genetically modified organisms, subject to stringent regulation.

In April 2019, the Australian government stated that the Office of the Gene Technology Regulator (OGTR) will regulate only the gene-editing technologies that use a template, or that insert other genetic material into the cell. According to an article of 2020, in India, as per the National Guidelines for Stem Cell Research, genome modification including gene-editing by CRISPR-Cas9 technology of stem cells, germ-line stem cells or gamete and human embryos is restricted only to in-vitro studies. Thus, strict regulations by the government present a threat to the growth of the market.

Several advancements in CRISPR technology are trending in the market during the period. Advancements in technology will help in reducing errors, limiting unintended effects, improving the accuracy of the tool, widening its applications, developing gene therapies and more. In 2019, a study published in Springer Nature stated the development of an advanced super-precise new CRISPR tool that allows researchers more control over DNA changes. This tool seems to have the capability of providing a wider variety of gene edits which might potentially open up conditions that have challenged gene-editors.

Also, in 2020, another study in Springer Nature stated that researchers have used enzyme engineering to boost the accuracy of the technique of error-prone CRISPR-Cas9 system to precisely target DNA without introducing as many unwanted mutations. The advancements in CRISPR technology will result in better tools that are capable of providing better outcomes.

The application of CRISPR technology as a diagnostic tool is expected to boost the market during the period. The Sherlock CRISPR SARS-CoV-2 kit is the first diagnostic kit based on CRISPR technology for infectious diseases caused due to COVID-19. In May 2020, FDA announced the emergency use authorization to the Sherlock BioSciences Inc's Sherlock CRISPR SARS-CoV-2 kit which is a CRISPR-based SHERLOCK (Specific High-sensitivity Enzymatic Reporter unLOCKing) diagnostic test.

This test helps in specifically targeting RNA or DNA sequences of the SARS-CoV-2 virus from specimens or samples such as nasal swabs from the upper respiratory tract and fluid in the lungs from bronchoalveolar lavage specimens. This diagnostic kit has high specificity and sensitivity and does not provide false negative or positive results. Widening the application of CRISPR technology for the diagnosis of infectious diseases will increase the demand for CRISPR technology products and services.

Key Topics Covered:

1. Executive Summary

2. CRISPR Technology Market Characteristics

3. CRISPR Technology Market Size And Growth

3.1. Global CRISPR Technology Historic Market, 2015 - 2019, $ Billion

3.1.1. Drivers Of The Market

3.1.2. Restraints On The Market

3.2. Global CRISPR Technology Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion

3.2.1. Drivers Of The Market

3.2.2. Restraints On the Market

4. CRISPR Technology Market Segmentation

4.1. Global CRISPR Technology Market, Segmentation By Product Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global CRISPR Technology Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global CRISPR Technology Market, Segmentation By End-User, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. CRISPR Technology Market Regional And Country Analysis 5.1. Global CRISPR Technology Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global CRISPR Technology Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

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

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Global CRISPR Technology Market Report 2020: COVID-19 Growth and Change - Market is Expected to Recover to Reach $1.55 Billion in 2023 - Forecast to...

What is the Market’s View on Crispr Therapeutics AG (CRSP) Stock’s Price and Volume Trends – InvestorsObserver

Crispr Therapeutics AG (CRSP) stock has gained 35.58% over the past week and gets a Bullish rating from InvestorsObserver's Sentiment Indicator.

In investing, sentiment generally means whether or not a given security is in favor with investors. It is typically a pretty short-term metric that relies entirely on technical analysis. That means it doesnt incorporate anything to do with the health or profitability of the underlying company.

Recent trends are a good indicator of current market sentiments. In its most basic form, stocks that are trending up are desirable by investors while stocks currently falling must be unattractive.

InvestorsObserver's Sentimental Indicator tracks both changes in price and volume to analyze the most recent trends. Typically an increase in volume indicates ongoing trends are getting stronger, while a decrease in volume usually signals an end to the current trend.

Available options can also represent current sentiments for a given stock. Since investors are able to bet on future trends of stocks using options, we consider the ratio of calls to puts when analyzing market sentiments .

Crispr Therapeutics AG (CRSP) stock is trading at $207.58 as of 10:53 AM on Friday, Jan 8, an increase of $13.15, or 6.76% from the previous closing price of $194.43. The stock has traded between $196.11 and $210.39 so far today. Volume today is below average. So far 1,482,585 shares have traded compared to average volume of 2,079,604 shares.

To see InvestorsObserver's Sentiment Score for Crispr Therapeutics AG click here.

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

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

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What is the Market's View on Crispr Therapeutics AG (CRSP) Stock's Price and Volume Trends - InvestorsObserver

5 questions facing gene therapy in 2021 – BioPharma Dive

Three years ago, the Food and Drug Administration granted a landmark approval to the first gene therapy for an inherited disease, clearing a blindness treatment called Luxturna.

Since then, the regulator has approved one more gene therapy,the spinal muscular atrophy treatment Zolgensma, and given a green light for dozens of biotech and pharmaceutical companies to start clinical testing on others. Genetic medicines for a range of diseases, including hemophilia, sickle cell and several muscular dystrophies, appear in reach, and new science is galvanizing research.

But, entering 2021, the gene therapy field faces major questions after a series of regulatory and clinical setbacks have shaded optimism. "The ups and downs of adolescence are on full display" analysts at Piper Sandler wrote in September, summing up the state of gene therapy research.

Here are five questions facing scientists, drugmakers and investors this year. How they're answered will matter greatly to the patients and families holding out hope for one-time disease treatments.

The FDA was widely expected last year to approve a closely watched gene therapy for hemophilia A, the more common type of the blood disease. Instead, the agency in August surprisingly rejected the treatment, called Roctavian, and asked its developer, BioMarin Pharmaceutical, to gather more data.

The next day, Audentes Therapeutics reported news came a third clinical trial participant had died after receiving the biotech's experimental gene therapy for a rare neuromuscular disease. The tragedy brought flashbacks to past safety scares in gene therapy, although the current wave of treatments being tested have generally appeared safe.

A little less than five months later, the gene therapy field is grappling with two more setbacks. UniQure is exploring whether a study volunteer's liver cancer was caused by its gene therapy for hemophilia B. And Sarepta, one of the sector's top developers, faces significant doubts about its top treatment for Duchenne muscular dystrophy after disclosing a key study missed one of its main goals.

In each case, the drugmakers involved offered explanations and reasons for optimism. BioMarin still expects to obtain an approval; Audentes' trial is now cleared by the FDA to resume testing; UniQure thinks it's unlikely the cancer case is linked to treatment; and Sarepta argued its negative data were the product of unlucky study design.

But taken together, the developments are powerful reminders of both the stakes and uncertainty still facing gene therapy.

All four events also highlighted lingering worries about one-time genetic treatment. In rejecting Roctavian, for example, the FDA seemed to be concerned the impressive benefit hemophilia patients initially experienced may wane over time. The deaths in Audentes' study, meanwhile,renewed warnings about extremely high doses of gene therapy. Researchers have long watched for evidence that replacing or altering genes may cause cancer to develop in rare instances, particularly after four infants developed leukemia in a gene therapy study in the early 2000s.And Sarepta's negative findings were surprising because early signs of dramatic biological benefit that didn't seem to translate into clear-cut functional gains for all patients.

Experts are still confident gene therapy can deliver on its promise. Bu recent events suggest getting there may take a bit longer than some expected.

"The process is the product," is an often-used cliche about gene therapy, which are complex treatments with exacting manufacturing standards.

Most of the roughly 60,000 pages in Spark Therapeutics' application for approval of Luxturna, for instance, involved what's known in the industry as "chemistry, manufacturing and controls."

The therapeutic basis for gene therapy, by contrast, is much clearer for many of the rare, monogenic diseases that developers are targeting. If mutations in a single gene lead to disease, replacing or otherwise fixing that gene should have a large benefit.

"Genetic medicine is not industrialized serendipity," said Gbola Amusa, an analyst at Chardan, contrasting gene therapy with chemical-based drugs."It often is an engineering question."

In 2020, the FDA gave ample notice that it's watching gene (and cell) therapy manufacturing closely.Sarepta,Voyager Therapeutics,Iovance Biotherapeuticsand Bluebird biowere all forced to revise their development timelines after the agency asked for new details about production processes.

"The FDA is saying to companies that you've got to up your standards," Amusa added.

For their part, FDA officials have indicated the spate of data requests are a product of the sharply higher numbers of companies advancing through clinical testing.

While setbacks have piled up for therapies that seek to replace genes, 2020 was a "transformative year" for therapies designed to edit them, according to Geulah Livshits, an analyst at Chardan.

CRISPR gene editing, already widely recognized as a scientific breakthrough, gained further prestige with the awarding of the Nobel Prize in Chemistry to two early pioneers, Jennifer Doudna and Emmanuelle Charpentier.

But the year also brought important progress from early biotech adopters.Editas Medicine and Intellia Therapeutics, for example, notched CRISPR firsts with use of the editing technology inside the human body.

And CRISPR Therapeutics and partner Vertex showed their experimental therapy, which uses CRISPR to edit stem cells, worked exceptionally well in the first 10 patients with either sickle cell disease or beta thalassemia treated in two early studies.

The data are the most concrete sign yet that CRISPR's clinical use can live up to its laboratory promise. While all three companies' therapies are still in early stages, their advances have ginned up substantial investor enthusiasm.

Together, the market value of CRISPR Therapeutics, Editas and Intellia totals nearly $25 billion. Beam Therapeutics, a startup that uses a more precise form of gene editing, is worth nearly $6 billion.

"Gene therapy will have a big role to play," said John Evans, Beam's CEO. "But I do think in the last year or so there's a growing realization that, when possible, you'd probably rather edit than add an extra gene."

Clinical tests will prove that out but, until then, the large upswing in share price for gene editing companies may not be sustainable as valuations creep higher and higher. Some of the recent run-up, for instance,appears driven by money flowing from generalist investors through exchange-trade funds, rather than from investors experienced in handicapping preclinical- or early clinical-stage companies.

"They're overdue for some type of rationalization," predicted Brad Loncar, CEO of Loncar Investments, adding that many companies are targeting similar diseases, most commonly sickle cell and beta thalassemia.

Tasked with replacing faulty genes with functional ones, scientists for the most part have turned to two types of viruses to safely shuttle genetic instructions into cells. Adeno-associated viruses, or AAVs,are typically used for infused treatments, while researchers working on cells extracted from patients generally opt for lentiviruses.

Each virus class has advantages, but also notable drawbacks. AAVs, for instance, can trigger pre-existing immune defenses in some people, making those individuals ineligible or poor candidates for gene therapy. Lentiviruses, by contrast, are known to integrate their DNA directly into the genomes of cells they infect a useful attribute in some regards but limiting in others.

Over decades of gene therapy research, scientists have found ways to tweak and modify these viral vectors to better suit their needs, but the basic tools are the same. Jim Wilson, a gene therapy pioneer who ran the study that led to the death of teenager Jesse Gelsinger in 1999, told attendees at a STAT conference last fall that he's "somewhat disappointed" by slow progress in viral vector research.

And as more and more gene therapies enter clinical testing, the limitations of current viral vectors have become more apparent.

The pace of research might be picking up, however. Recently, a number of companies aiming to build better delivery tools have launched, including Harvard University spinout Dyno Therapeutics and 4D Molecular Therapeutics, which recently raised $222 million in an initial public offering.

Larger companies are interested, too. Roche, Sarepta and Novartis have all partnered with Dyno, for example.

In gene editing, meanwhile, researchers are developing new ways to cut DNA, while Beam and others are advancing different editing approaches altogether.

Billions of dollars have flowed from pharmaceutical companies into gene therapy over the past few years, leaving few large multinational drugmakers without a research presence.

2020 was no different, with sizable acquisitions inked by Bayer and Eli Lilly, as well as an array of smaller investments from Pfizer, Novartis, Johnson & Johnson, Biogen,and UCB. And CSL Behring, best known for its blood plasma products, spent nearly half a billion dollars to buy UniQure's most advanced gene therapy, a treatment for hemophilia B.

Over the past three years, there's been at least $30 billion spent on biotechs involved in gene or cell therapy. (Four deals account for the majority of that value.)

All of that dealmaking, while following promising and compelling science, is ultimately a bet that one-time genetic treatments can be scaled up and commercialized into a lucrative business.

Many of the acquired companies are working on therapies for very rare disorders affecting hundreds or thousands of people. A handful, however, are taking aim at more prevalent conditions, starting with still relatively uncommon diseases like hemophilia to ones affecting millions of people like Parkinson's.

"For gene therapy to meet our lofty expectations not just for investors, but for society it has to make the leap from these ultra-rare diseases," said Loncar.

Commercially, the track record for the few therapies on the market in the U.S. is mixed.Luxturna, now owned by Roche, is a niche product.Zolgensma has broader use and earned Novartis about $1 billion in the year and a half it's been commercially available.

Two cell therapies from Novartis and Gilead, meanwhile, have struggled to gain traction.

Gene therapy's biggest commercial test yet was supposed to come this year, with the expected approval of BioMarin's Roctavian in hemophilia A. The FDA's surprise rejection could mean a yearslong delay in the U.S., but the challenges of pricing, reimbursement and patient access in gene therapy remain dauntingly large.

Read this article:
5 questions facing gene therapy in 2021 - BioPharma Dive

CRISPR-based strategies in infectious disease diagnosis and therapy – DocWire News

This article was originally published here

Infection. 2021 Jan 3. doi: 10.1007/s15010-020-01554-w. Online ahead of print.

ABSTRACT

PURPOSE: CRISPR gene-editing technology has the potential to transform the diagnosis and treatment of infectious diseases, but most clinicians are unaware of its broad applicability. Derived from an ancient microbial defence system, these so-called molecular scissors enable precise gene editing with a low error rate. However, CRISPR systems can also be targeted against pathogenic DNA or RNA sequences. This potential is being combined with innovative delivery systems to develop new therapeutic approaches to infectious diseases.

METHODS: We searched Pubmed and Google Scholar for CRISPR-based strategies in the diagnosis and treatment of infectious diseases. Reference lists were reviewed and synthesized for narrative review.

RESULTS: CRISPR-based strategies represent a novel approach to many challenging infectious diseases. CRISPR technologies can be harnessed to create rapid, low-cost diagnostic systems, as well as to identify drug-resistance genes. Therapeutic strategies, such as CRISPR systems that cleave integrated viral genomes or that target resistant bacteria, are in development. CRISPR-based therapies for emerging viruses, such as SARS-CoV-2, have also been proposed. Finally, CRISPR systems can be used to reprogram human B cells to produce neutralizing antibodies. The risks of CRISPR-based therapies include off-target and on-target modifications. Strategies to control these risks are being developed and a phase 1 clinical trials of CRISPR-based therapies for cancer and monogenic diseases are already underway.

CONCLUSIONS: CRISPR systems have broad applicability in the field of infectious diseases and may offer solutions to many of the most challenging human infections.

PMID:33393066 | DOI:10.1007/s15010-020-01554-w

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Plant Breeding And CRISPR Plants Market Is Expected To Reach USD 21.78 Billion By 2027 | Top Companies- Bayer AG, KWS SAAT SE & Co. KGaA, DuPont,…

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Summary of the Report

The market would gain significant growth rate during the forecast period of 2020 to 2027 reaching a substantial market size by 2027. The market has been analyzed taking into considerations the different factors which includes the market drivers, restraints, opportunities, key competitor landscape, trend analysis, outlook, estimate and forecast factors.

Plant breeding and CRISPR plants market is expected to reach USD 21.78 billion by 2027 growing with the growth rate of 13.70% in the forecast period 2020 to 2027. Rising importance for sustainable crop production drives the growth of plant breeding and CRISPR plants market in the forecast period of 2020- 2027.

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Major Key Players of the Plant Breeding and CRISPR Plants Market

Bayer AG, KWS SAAT SE & Co. KGaA, DuPont, SGS SA, DLF Seeds Ltd, BioConsortia Inc., Hudson River Biotechnology., Pacific Biosciences of California, Inc, Eurofins Scientific, Syngenta, SGS SA, Land OLakes, Inc, Advanta Seeds Pty Ltd, J.R. Simplot Company among other domestic and global players.

Market Scope, Segments and Forecast of the Plant Breeding and CRISPR Plants Market

The Plant Breeding and CRISPR Plants Market is witnessing high demand due to the rise in demand of the product across different end-use areas. On the basis of product, geography and application the market is bi-furcated into different sub-segments as per the feasibility check and market estimation from 2020 to 2027 have been provided for these segments.

Market Overview, Key Trends Market Dynamics

The market would gain significant growth rate during the forecast period, reaching a substantial market size by 2020. The market has been analyzed taking into considerations the different factors which includes the market drivers, restraints, opportunities, key competitor landscape, trend analysis, outlook, estimate and forecast factors. The impact of COVID -19 could be seen on the market; however, the Plant Breeding and CRISPR Plants Market would recover from this pandemic by end of the next year. We have also mentioned the key trends of the market that would impact the growth of the market at present and in the coming years as well.

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Geographical Coverage of Plant Breeding and CRISPR Plants Market

Table of Contents

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Where Does Crispr Therapeutics AG (CRSP) Stock Fall in the Biotechnology Field? – InvestorsObserver

A rating of 80 puts Crispr Therapeutics AG (CRSP) near the top of the Biotechnology industry according to InvestorsObserver. Crispr Therapeutics AG's score of 80 means it scores higher than 80% of stocks in the industry. Crispr Therapeutics AG also received an overall rating of 64, putting it above 64% of all stocks. Biotechnology is ranked 30 out of the 148 industries.

Searching for the best stocks to invest in can be difficult. There are thousands of options and it can be confusing on what actually constitutes a great value. Investors Observer allows you to choose from eight unique metrics to view the top industries and the best performing stocks in that industry. A score of 64 would rank higher than 64 percent of all stocks.

These scores are not only easy to understand, but it is easy to compare stocks to each other. You can find the best stock in an industry, or look for the sector that has the highest average score. The overall score is a combination of technical and fundamental factors that serves as a good starting point when analyzing a stock. Traders and investors with different goals may have different goals and will want to consider other factors than just the headline number before making any investment decisions.

Crispr Therapeutics AG (CRSP) stock is trading at $180.75 as of 10:23 AM on Thursday, Jan 7, a rise of $16.90, or 10.32% from the previous closing price of $163.85. The stock has traded between $169.39 and $185.43 so far today. Volume today is light. So far 888,487 shares have traded compared to average volume of 1,989,285 shares.

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

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Where Does Crispr Therapeutics AG (CRSP) Stock Fall in the Biotechnology Field? - InvestorsObserver

CRISPR and CAS Gene Market 2021-2028 shooting revenue at US$ 7,603.8 Million with CRISPR Therapeutics, Mirus Bio, Caribou Biosciences, Editas…

CRISPR and CAS Gene Market witness to garner US$ 7,603.8 Million at a booming CAGR of +20% by the term of 2021-28.

CRISPR-Cas9 is a unique technology that enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence. It is currently the simplest, most versatile and precise method of genetic manipulation and is therefore causing a buzz in the science world.

CRISPR is a tool that can be used to edit genes and, as such, will likely change the world. The essence of CRISPR is simple: its a way of finding a specific bit of DNA inside a cell. After that, the next step in CRISPR gene editing is usually to alter that piece of DNA.

When viruses infect bacteria, bacteria will produce this type of DNA and bind to virus DNAs; with working with one nuclease, called Cas, the Cas enzyme will cut the invaded DNA into pieces. Thus, CRISPR/Cas is a type of acquired immune defense mechanism for prokaryotes against virus.

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The CRISPR and CAS Gene Market report gives the 360 degree perspective on the fundamentals of market, definitions, groupings, applications and industry chain review, industry arrangements and plans, item details, forms, cost structures and afterward on. At that point it examine the worlds primary district and economic situations, including the product value, benefit, limit, creation, limit usage, request and development pace of industry.

The investigator likewise centers around monetary and ecological variables, which impacts on the development of the business. Essential and auxiliary exploration methods have been utilized by analysts to get appropriate experiences into business. Requesting patterns and mechanical headways have been introduced in the examination report.

Key Players:

Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Highlights of the report:

The report covers the major driving factors influencing the revenue scale of the CRISPR and CAS Gene Market and details about the surging demand in this area. The report then highlights the latest trends and challenges that leading industry contenders could face. The significant applications and potential business areas are also added to this report. CRISPR and CAS Gene market research is provided for international markets as well as development trends, competitive landscape analysis and development status of key regions. Development policies and plans are discussed as well as manufacturing processes and analysis of cost structures. This report also shows import/export consumption, supply and demand, costs, prices, revenues and gross margins.

CRISPR and CAS Gene Market Report Segment: by product

CRISPR and CAS Gene Market Report Segment: by application

CRISPR and CAS Gene Market Report Segment: by end-user

CRISPR and CAS Gene Market Report Segment: by region

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The report provides major statistics of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market. CRISPR and CAS Gene is explained in detail in various regions and various segments of the industry.

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