Archive for the ‘Crispr’ Category

Transparency Market Research (TMR) has published a new report titled, CRISPR and Cas GenesMarket Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026. According to the report, theglobal CRISPR and Cas genes marketwas valued at US$ 7,234.5 Mn by 2026, expanding at a CAGR of around 20.1% from 2018 to 2026. Increase in applications of CRISPR and Cas gene editing technology in bacteria and usage of gene editing technology for prevention of various diseases are the major factors anticipated to drive the market from 2018 to 2026. Rise in need of alternative medicine for chronic diseases and increase in investments by key players in Asia Pacific are projected to propel the market during the forecast period.

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Increase in Usage of DNA-free Cas

DNA-free Cas9 is most commonly used with synthetic crRNA tracrRNA and chosen by researchers who strive to avoid unwanted vector DNA integration into their genomic DNA. CRISPR-Cas9 utilizing mRNA or protein is ideal for applications such as knocking of a fluorescent reporter using HDR or knockout cell line generation. Advantages such as gene editing with DNA-free CRISPR-Cas9 components to reduce potential off-targets and potential usage of CRISPR-Cas9 gene editing to find correlations with human diseases in model systems drive the segment.

Rise in Incidence of Genetic Disorders and Increase in Applications of CRISPR and Cas Genesto Propel Market

Genetic diseases are generally termed as rare diseases. According to NCBI, prevalence of these rare diseases is approximately 5 in 10,000. There are 6,000 to 8,000 rare diseases, with 250 to 280 new diseases diagnosed every year. Hence, 6% to 8% of the global population is projected to be affected by rare diseases i.e., genetic diseases in the near future. Researchers are developing treatments for these diseases with applications of new technologies such as CRISPR. The applications of CRISPR technology are expanding in other industrial sectors. This is expected to drive the market during the forecast period. Usage of CRISPR/Cas9 technology in plant research has enabled the investigation of plant biology in detail which has helped to create innovative applications in crop breeding. Site-directed mutagenesis and site-specific integration of a gene, which is also called knock-in, are important in precision crop breeding. Cas9/gRNA-mediated site-directed mutagenesis and knock-in is widely used in rice and Arabidopsis protoplasts. CRISPR/Cas9 provides a simple method to generate a DSB at a target site to trigger HDR repair.

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Ethical Issues Concerning Gene Editing Technology to Restrain Market

Any changes in the human genome are made in the germline, and hence are expected to be passed on to the future generations. The safety and efficacy concerns with genome editing have gained momentum with the discovery of CRISPR, as it has the potential to make accurate use of genome editing technologies. Major ethical issues concerning the human genome editing technology are disturbance of the ecological equilibrium, patent regulations concerning CRISPR/Cas9 gene technology, non-therapeutic interventions using human genome, and formation of chimera which has the risk of violation of the order of nature and giving rise to moral confusion for treating organisms.

Asia Pacific Market to Witness Exponential Growth

In terms of revenue, the CRISPR and Cas genes market in Asia Pacific is expected to expand at a CAGR of 22.0% during the forecast period. Growth of the market in the region can be attributed to increase in incidence of chronic diseases such as cancer and the need of development of genetic engineered treatment options. According to the report, Call for Action: Expanding Cancer Care for Women in India, 2017, an estimated 0.7 million women in India are suffering from cancer. China dominated the CRISPR and Cas genes market in Asia Pacific. In 2016, scientists based in China launched the first known human trials of CRISPR, the genomic tech that involves slicing and dicing the bodys very source code to fight cancer. Japan was the second largest market for CRISPR and Cas genes in Asia Pacific.

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Addgene, Thermo Fisher Scientific, Inc., and Integrated DNA Technologies, Inc. to Lead Market

The report also provides profiles of leading players operating in the global CRISPR and Cas market such as 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.

More Trending Reports by Transparency Market Research 1.https://www.prnewswire.com/news-releases/global-radiation-therapy-market-to-reach-us-8-6-bn-by-2026product-approvals-to-drive-growth-transparency-market-research-300998453.html

2.https://www.biospace.com/article/cholesterol-lowering-drugs-market-increase-in-prevalence-of-coronary-artery-disease-to-drive-market/

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CRISPR and Cas Genes Market Share is Thriving Worldwide By Size, Revenue, Emerging Trends and Top Growing Companies 2026 - Science Examiner

The Global Gene Therapies Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost, and revenue during the forecast period from 2020 to 2027. The report provides a full assessment. Gene Therapies market with future trends, current growth factors, meticulous opinions, facts, historical data and statistically supported and industry-validated market data.

This Gene Therapies market research provides a clear explanation of how this market will impress growth during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

To Learn More About This Report, Request a Sample Copy:https://www.worldwidemarketreports.com/sample/179400* The sample copy includes: Report Summary, Table of Contents, Segmentation, Competitive Landscape, Report Structure, Methodology.

Adaptimmune Therapeutics Plc., Celgene Corporation, Crispr Therapeutics Ag, Glaxosmithkline Plc, Intellia Therapeutics Inc., Merck & Co. Inc., Novartis Ag, Regenxbio Inc., Voyager Therapeutics Inc., Abeona Therapeutics Inc. are some of the major organizations dominating the global market.(*Note: Other Players Can be Added per Request)

Key players in the Gene Therapies market were identified through a second survey, and their market share was determined through a primary and second survey. All measurement sharing, splitting, and analysis were solved using a secondary source and a validated primary source. The Gene Therapies market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure, and when used together, how key players can meet market coverage, offered characteristics, and customer needs It helps to understand.

The report also makes some important suggestions for new Gene Therapies market projects before evaluating their feasibility. Overall, this report covers Gene Therapies market Sales, Price, Sales, Gross Profit, Historical Growth,and Future Prospects. It provides facts related to the widespread merger, acquisition, partnership, and joint venture activities on the market.

This report includes market size estimates of value (million US $) and trading volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the Gene Therapies market, estimating the size of various other subordinate markets in the overall market. All ratio sharing, splitting, and analysis were determined using the secondary source and the identified primary source.

What Gene Therapies Market report offers:

Remarkable Attributes of Gene Therapies Market Report:

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Worldwide Market Reports is your one-stop repository of detailed and in-depth market research reports compiled by an extensive list of publishers from across the globe. We offer reports across virtually all domains and an exhaustive list of sub-domains under the sun. The in-depth market analysis by some of the most vastly experienced analysts provide our diverse range of clients from across all industries with vital decision making insights to plan and align their market strategies in line with current market trends.

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Gene Therapies Market: Future Opportunities, Revenue Growth, Rating, and Profit (2020 2027): Adaptimmune Therapeutics Plc., Celgene Corporation,...

On 27 June, Verve therapeutics, a US-based biotechnology firm, in a press release,announced that they have demonstrated, in primate models, the successful use of base-editing to switch off two genes in the liver to reduce blood LDL (low-density lipoproteins) and triglyceride levels. These two factors increase the risk of coronary atherosclerosis.

Coronary atherosclerosis or coronary artery disease refers to the development of plaque inside the coronary arteries (arteries that supply blood to the heart). This plaque narrows down the arteries, reducing blood flow. This leads to chest pain, lightheadedness and shortness of breath and increase in the risk of a heart attack.

Base-editors are CRISPR-based tools that can specifically make point mutations in the DNA of an organism. Mutations refer to changes in the DNA sequence. A mutation can switch on or off a gene or it can increase or decrease its functioning.

Representational image. Reuters

CRISPR-Cas9 system consists of two things - CRISPR, which are short palindromic sequences of DNA/RNA that help identify the target sequences and Cas 9, which is an enzyme that cuts the sequences that CRISPR identifies.

The results of the study were presented at the 2020 Virtual Annual Meeting of the International Society for Stem Cell Research (ISSCR).

The study

For the study, the researchers at Verve targetted and turned off (by giving an intravenous drug) one of the two genes in a group of 14 monkeys - PCSK9 (proprotein convertase subtilisin/Kexin type 9) or ANGPTL3 (angiopoietin-like protein 3). The former produces a protein that increases LDL levels in the blood while the latter produces a protein that increases blood-triglyceride rich lipoprotein levels.

Results were noted two weeks after delivering the drug. Here is what was found:

The future prospects

According to the World Health Organisation, cardiovascular diseases, including coronary artery disease, are the number one cause of death in the world, claiming about 17.9 million lives each year.

The condition is currently treated through a combination of lifestyle modifications and medications to control the symptoms and keep the disease from worsening. Surgery is often needed to remove the plaque.

Praising their new tool, Dr Andrew Bellinger, Chief Scientific Officer at Verve Therapeutics said in the press release that the fact that PCSK9 and ANGPTL3 can be silenced safely and effectively in non-human primates and that blood LDL and triglyceride levels can be reduced is very exciting.

He added that one of the most important things is that no other non-target gene was affected in the study and that this just proves that Verves once-and-done gene editing treatments may actually be effective in treating coronary artery disease in adults.

Since the genes are silenced, the person will not need to be on treatment for long. Verve is set to start the clinical phase of their study by 2023.

For more information, read our article on Heart Disease.

Health articles in Firstpost are written by myUpchar.com, Indias first and biggest resource for verified medical information. At myUpchar, researchers and journalists work with doctors to bring you information on all things health.

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Research on a gene-editing technique to treat heart disease shows promising results in animal trials - Firstpost

ZUG, Switzerland and CAMBRIDGE, Mass., June 29, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq:CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, today announced that it is commencing an underwritten public offering of $325,000,000 of common shares. In addition, the underwriters will have a 30-day option to purchase up to an additional $48,750,000 of common shares at the public offering price less the underwriting discount.

Goldman Sachs & Co. LLC, BofA Securities and Jefferies are acting as joint book-running managers for the offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed or as to the actual size or terms of the offering.

The common shares will be offered and sold pursuant to the Companys previously filed automatically effective shelf registration statement on FormS-3(FileNo.333-227427)filed with the U.S. Securities and Exchange Commission (the SEC) on September19, 2018. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The offering will be made only by means of a prospectus. A copy of the prospectus supplement relating to the offering will be filed with the SEC and may be obtained, when available, from Goldman Sachs & Co. LLC by mail at 200 West Street, New York, NY 10282, Attention: Prospectus Department, by telephone at (866) 471-2526, or by email at prospectus-ny@ny.email.gs.com; from BofA Securities by mail at NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at dg.prospectus_requests@bofa.com; or from Jefferies, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 547-6340, or by email at prospectus_department@jefferies.com.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding CRISPR Therapeutics anticipated public offering. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release, such as the intended offering terms, are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, uncertainties related to market conditions and the completion of the public offering on the anticipated terms or at all. These and other risks and uncertainties are described in greater detail in the section entitled Risk Factors in CRISPR Therapeutics Annual Report on Form 10-K for the year ended December 31, 2019, as filed with the SEC on February 12, 2020, the prospectus supplement related to the public offering and other filings that CRISPR Therapeutics may make with the SEC in the future. Any forward-looking statements contained in this press release represent CRISPR Therapeutics views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. CRISPR Therapeutics explicitly disclaims any obligation to update any forward-looking statements.

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

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

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CRISPR Therapeutics Announces Proposed Offering of Common Shares - GlobeNewswire

In recent months, even as our attention has been focused on the coronavirus outbreak, there have been a slew of scientific breakthroughs in treating diseases that cause blindness.

Researchers at U.S.-based Editas Medicine and Ireland-based Allergan have administered CRISPR for the first time to a person with a genetic disease. This landmark treatment uses the CRISPR approach to a specific mutation in a gene linked to childhood blindness. The mutation affects the functioning of the light-sensing compartment of the eye, called the retina, and leads to loss of the light-sensing cells.

According to the World Health Organization, at least 2.2 billion people in the world have some form of visual impairment. In the United States, approximately 200,000 people suffer from inherited forms of retinal disease for which there is no cure. But things have started to change for good. We can now see light at the end of the tunnel.

I am an ophthalmology and visual sciences researcher, and am particularly interested in these advances because my laboratory is focusing on designing new and improved gene therapy approaches to treat inherited forms of blindness.

Gene therapy involves inserting the correct copy of a gene into cells that have a mistake in the genetic sequence of that gene, recovering the normal function of the protein in the cell. The eye is an ideal organ for testing new therapeutic approaches, including CRISPR. That is because the eye is the most exposed part of our brain and thus is easily accessible.

The second reason is that retinal tissue in the eye is shielded from the bodys defense mechanism, which would otherwise consider the injected material used in gene therapy as foreign and mount a defensive attack response. Such a response would destroy the benefits associated with the treatment.

In recent years, breakthrough gene therapy studies paved the way to the first ever Food and Drug Administration-approved gene therapy drug, Luxturna TM, for a devastating childhood blindness disease, Leber congenital amaurosis Type 2.

This form of Leber congenital amaurosis is caused by mutations in a gene that codes for a protein called RPE65. The protein participates in chemical reactions that are needed to detect light. The mutations lessen or eliminate the function of RPE65, which leads to our inability to detect light blindness.

The treatment method developed simultaneously by groups at University of Pennsylvania and at University College London and Moorefields Eye Hospital involved inserting a healthy copy of the mutated gene directly into the space between the retina and the retinal pigmented epithelium, the tissue located behind the retina where the chemical reactions takes place. This gene helped the retinal pigmented epithelium cell produce the missing protein that is dysfunctional in patients.

Although the treated eyes showed vision improvement, as measured by the patients ability to navigate an obstacle course at differing light levels, it is not a permanent fix. This is due to the lack of technologies that can fix the mutated genetic code in the DNA of the cells of the patient.

Lately, scientists have been developing a powerful new tool that is shifting biology and genetic engineering into the next phase. This breakthrough gene editing technology, which is called CRISPR, enables researchers to directly edit the genetic code of cells in the eye and correct the mutation causing the disease.

Children suffering from the disease Leber congenital amaurosis Type 10 endure progressive vision loss beginning as early as one year old. This specific form of Leber congenital amaurosis is caused by a change to the DNA that affects the ability of the gene called CEP290 to make the complete protein. The loss of the CEP290 protein affects the survival and function of our light-sensing cells, called photoreceptors.

One treatment strategy is to deliver the full form of the CEP290 gene using a virus as the delivery vehicle. But the CEP290 gene is too big to be cargo for viruses. So another approach was needed. One strategy was to fix the mutation by using CRISPR.

The scientists at Editas Medicine first showed safety and proof of the concept of the CRISPR strategy in cells extracted from patient skin biopsy and in nonhuman primate animals.

These studies led to the formulation of the first ever in human CRISPR gene therapeutic clinical trial. This Phase 1 and Phase 2 trial will eventually assess the safety and efficacy of the CRISPR therapy in 18 Leber congenital amaurosis Type 10 patients. The patients receive a dose of the therapy while under anesthesia when the retina surgeon uses a scope, needle and syringe to inject the CRISPR enzyme and nucleic acids into the back of the eye near the photoreceptors.

To make sure that the experiment is working and safe for the patients, the clinical trial has recruited people with late-stage disease and no hope of recovering their vision. The doctors are also injecting the CRISPR editing tools into only one eye.

An ongoing project in my laboratory focuses on designing a gene therapy approach for the same gene CEP290. Contrary to the CRISPR approach, which can target only a specific mutation at one time, my team is developing an approach that would work for all CEP290 mutations in Leber congenital amaurosis Type 10.

This approach involves using shorter yet functional forms of the CEP290 protein that can be delivered to the photoreceptors using the viruses approved for clinical use.

Gene therapy that involves CRISPR promises a permanent fix and a significantly reduced recovery period. A downside of the CRISPR approach is the possibility of an off-target effect in which another region of the cells DNA is edited, which could cause undesirable side effects, such as cancer. However, new and improved strategies have made such likelihood very low.

Although the CRISPR study is for a specific mutation in CEP290, I believe the use of CRISPR technology in the body to be exciting and a giant leap. I know this treatment is in an early phase, but it shows clear promise. In my mind, as well as the minds of many other scientists, CRISPR-mediated therapeutic innovation absolutely holds immense promise.

In another study just reported in the journal Science, German and Swiss scientists have developed a revolutionary technology, which enables mice and human retinas to detect infrared radiation. This ability could be useful for patients suffering from loss of photoreceptors and sight.

The researchers demonstrated this approach, inspired by the ability of snakes and bats to see heat, by endowing mice and postmortem human retinas with a protein that becomes active in response to heat. Infrared light is light emitted by warm objects that is beyond the visible spectrum.

The heat warms a specially engineered gold particle that the researchers introduced into the retina. This particle binds to the protein and helps it convert the heat signal into electrical signals that are then sent to the brain.

In the future, more research is needed to tweak the ability of the infrared sensitive proteins to different wave lengths of light that will also enhance the remaining vision.

This approach is still being tested in animals and in retinal tissue in the lab. But all approaches suggest that it might be possible to either restore, enhance or provide patients with forms of vision used by other species.

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Gene therapy and CRISPR strategies for curing blindness (Yes, you read that right) - The Conversation US

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The biotech firm CRISPR Therapeutics is one of Piper Sandler analyst Ted Tenthoffs top picks for 2020, he reaffirmed in a note out Monday.

CRISPR Therapeutics stock (ticker: CRSP) was up 0.5% Monday morning after rising as much as 3.9% in pre-market trading. Shares fell 2.6% on Friday. CRISPR Therapeutics, which is developing therapeutic genome-editing technology, announced last Thursday that it would build a new manufacturing facility in Massachusetts to produce its experimental cell therapies.

In his Monday note, Tenthoff wrote that the company is moving forward with its CRISPR-based allogenic CAR-T cell therapies, a set of experimental cancer treatments, with data expected by the end of the year.

CRISPR [Therapeutics] remains a top pick for 2020, Tenthoff wrote. He maintained his Overweight rating on this stock, and his $104 price target. The stock closed at $74.04 on Friday and traded as high as $76.98 in pre-market trading Monday. CRISPR Therapeutics stock was recently trading up 2.25% to $75.71.

Shares of CRISPR Therapeutics are up about 22% so far this year. Of the 17 analysts tracked by FactSet who cover the stock, 11 assign it a Buy rating. The analysts have a mean target price of $75.75.

In his note on Monday, Tenthoff highlighted recent data showing promising early results in two transfusion-dependent beta-thalassemia patients who had received CTX001, a treatment CRISPR Therapeutics is developing in partnership with Vertex Pharmaceuticals (VRTX).

He also highlighted developments in the companys CAR-T cancer programs, a handful of which are beginning or about to begin Phase I/II studies. We see the potential for CRISPR to garner significant value from its wholly-owned CAR-T pipeline, Tenthoff wrote.

CRISPR Therapeutics has a market capitalization of $4.5 billion. It has outperformed the iShares Nasdaq Biotechnology ETF (IBB) so far this year, which is up 11.4%, and the S&P 500, which is down 6.9%.

Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com

Excerpt from:
CRISPR Therapeutics Is a Top Pick for Piper Sandler. Heres Why. - Barron's

What happened

CRISPR Therapeutics (NASDAQ:CRSP) stock is hot this week, after the biotech presented four research posters at the American Association for Cancer Research's Virtual Meeting II this week, offering preclinical data on its gene-editing cancer research and sparking a flurry of supportive notes from Wall Street.

CRISPR stock is up a solid 4.5% as of 2:55 p.m. EDT today and, added to yesterday's gains, has risen more than 9% over the past two days.

Image source: Getty Images.

CRISPR's CAR T-cell technology modifies a patient's immune system T cells and tailors them to fight specific forms of cancer. In this week's posters, the company is said to be reporting data showing that its CTX130 treatment is both specific and potent in fighting both hematologic and solid tumors.

Specific news on the findings is hard to come by, but whatever CRISPR is saying, analysts seem to like it -- a lot. In less than just the last 24 hours, no fewer than three separate Wall Street firms have raised or reiterated their price targets on CRISPR stock. TheFly.com is reporting that Oppenheimer & Co. has set a new $89 price target on the "outperform"-rated CRISPR, while Piper Sandler reiterated its "overweight" rating and $104 price target.

StreetInsider.com adds that Stifel Nicolaus, too, is optimistic, and has raised its price target to $61.

That last rating isn't quite as bullish as the first two. After their two-day run-up, CRISPR shares already sell for more than $75, after all -- and accordingly, Stifel only gives the stock a "hold" rating. Still, investors seem to think that two out of three isn't bad, and are focusing on the more positive valuation estimates from Oppenheimer and Piper Sandler today.

Speaking of valuation, investors looking at CRISPR for the first time in light of these analyst endorsements may be encouraged to see that this start-up biotech already seems to be profitable and free-cash-flow positive -- but don't be fooled. CRISPR's flush with cash currently because it just received a big milestone payment from partner Vertex Pharmaceuticals. Analysts expect the company will end this year with a loss, however, and will continue losing money for at least the next three years before becoming profitable again.

Investing in CRISPR still requires keeping an eye on the big picture, and monitoring the company's steady progress toward long-term, sustained sales, revenue, and profits -- all of which are years in the future. In the near term, the company's numbers are bound to resume looking pretty ugly, and sooner rather than later.

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Why CRISPR Therapeutics Stock Just Popped Another 5% - Motley Fool

The first patient to be treated for Sickle Cell Disease with the revolutionary gene-editing program CRISPER had a transfusion of billions of modified cells last year, and now a year later her treatment is still working perfectly.

Before Victoria Gray underwent the treatment in mid-2019 she had been experiencing frequent bouts of physical pain. Patients like her are often forced to get blood transfusionsan average of seven per year in Grays casein order to ensure that their body has enough healthy red blood cells to carry oxygen throughout the body.

But now, alleviation of almost all of her symptoms has allowed her to stay out of Mississippi hospitals and support her three kids while her husband, a national guardsman, was temporarily deployed out of town.

The era of COVID-19 is hardly the time to be popping into a hospital for a blood transfusion, so the improvement for Gray has been substantialand feels like a miracle.

Since my treatment Ive been able to do everything for myself, everything for my kids, she told NPR. And its been a joy not only for me but for the people around me.

RELATED: Worlds Second Person Cured of HIV: 40-Year-old Man is Confirmed to Be 30 Months Virus-Free

An artifact of the strange genetic history of humanity, sickle-cell genes evolved in Africans as a strong defense against malaria, but they can also increase the chance of dying relatively young from the blood disorder.

However, her case, along with those of two others who were treated in the same way, is producing only good news a year later. Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tennessee, who is treating Gray, described it as extremely thrilling to see and extremely exciting.

Frangoul and other scientists using CRISPER presented the results of their latest testing of the three cases to the European Hematology Association on June 12th. The two other subjects had a related condition, beta thalassemia, and were treated in Germany with a similar method and have now been able to live without blood transfusions for 15 months.

RELATED: After Her Years of Research, a Cambridge Scientist Could Be on the Verge of Curing Multiple Sclerosis

I think this is a huge leap for the medical field, Frangoul told NPR in an interview, asserting that Gray will be, hopefully, the first of many thousands more of patients who will be able to see things they never imagined possible.

High school graduations, college graduations, weddings, grandkids I thought I wouldnt see none of that, Gray said.

SHARE the Good News on Social Media (Photo by Libertas Academica, CC license on Foter)

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First Sickle Cell Patient Treated with CRISPR Gene Editing is Now Thriving One Year Later, And Able to Care For Her Kids - Good News Network

Latest Report on CRISPR and Cas Genes Market Size | Industry Segment by Applications (Genome Engineering, Disease Models and Others), by Type (Vector-based Cas and DNA-free Cas), Regional Outlook, Market Demand, Latest Trends, CRISPR and Cas Genes Industry Share & Revenue by Manufacturers, Company Profiles, Growth Forecasts 2025.

The research report on CRISPR and Cas Genes market provides a broad perspective of this business vertical and comprises of substantial details such as market size, revenue estimation, industry remuneration, and market valuation over the study period.

The study assesses the key factors positively impacting the overall industry landscape on the basis of market growth and sales acceleration. Moreover, it delivers information regarding the major market trends and their impact on the business space.

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Major parameters of CRISPR and Cas Genes market report:

Regional study of CRISPR and Cas Genes market:

CRISPR and Cas Genes Market Segmentation: Americas, APAC, Europe, Middle East & Africa.

An overview of the geographical landscape of CRISPR and Cas Genes market:

Product spectrum and application scope of CRISPR and Cas Genes market:

Product landscape:

Product types: Vector-based Cas and DNA-free Cas

Key factors encompassed in the report:

Application Landscape:

Application segmentation: Genome Engineering, Disease Models and Others

Data delivered by the study:

Other details specified in the report:

Competitive space of the CRISPR and Cas Genes market:

Leading players in the CRISPR and Cas Genes market: Synthego, Horizon Discovery Group Co., Thermo Fisher Scientific, Inc., Addgene, Merck, GenScript, OriGene Technologies, Inc., Integrated DNA Technologies, Inc., New England Biolabs, Transposagen Biopharmaceuticals, Inc., Cellecta, Inc., Applied StemCell, Inc. and Agilent Technologies

Key aspects listed in the report:

Key questions answered in this report:

What will the market size be in 2025 and what will the growth rate be?

What are the key market trends?

What is driving this market?

What are the challenges to market growth?

Who are the key vendors in this market space?

What are the market opportunities and threats faced by the key vendors?

What are the strengths and weaknesses of the key vendors?

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CRISPR and Cas Genes Market with Future Prospects, Key Player SWOT Analysis and - News by aeresearch

Some stocks have just one problem: Once you've bought them, you never want to sell them.That's the case with Vertex Pharmaceuticals (NASDAQ:VRTX), a biotech company with a leading position in the area of cystic fibrosis.

Vertex's shares have climbed 35% this year to trade near a record high. As the coronavirus outbreak has weighed on business across industries, the impact on this company has been limited to slowdowns in certain clinical trials. Vertex actually revised its cystic fibrosis revenue estimate for the year -- upward.

Image source: Getty Images.

Beyond this winning performance, here are three reasons to grab Vertex now and hang on for the long term.

Vertex was already a leader in cystic fibrosis with three marketed products when a fourth, Trikafta, was approved in October 2019. In the third quarter of 2019, just before Trikafta's launch, the company's cystic fibrosis drugs generated a combined $950 million, a 21% increase from the year-earlier period. Trikafta immediately leaped ahead. In its first full quarter on the market, the drug's $895 million in sales represented 60% of product revenue. In Vertex's most recent earnings call, the company predicted that Trikafta will drive revenue growth in the coming years.

What makes Trikafta such a winning product? This combination therapy, which includes elexacaftor, tezacaftor, and ivacaftor, may treat up to 90% of cystic fibrosis patients worldwide, Vertex says. Trikafta addresses the underlying cause of the disease in patients with the most common genetic mutation.

Trikafta is clearly a blockbuster in the making. Along with Vertex's other cystic fibrosis drugs, it's creating a long-term growth story. Vertex's cystic fibrosis treatments may generate more than $10 billion in sales by 2028, according to Morningstar. That's compared to $4 billion last year.

Gene and cell therapies involve introducing genetic material or living cells into the body to produce a therapeutic effect. Last year, Vertex took steps to solidify its presence in this promising area. The company announced a research center dedicated to the program, acquired gene-editing specialist Exonics Therapeutics, and expanded its partnership with CRISPR Therapeutics (NASDAQ:CRSP).

Using expertise from Exonics and CRISPR, Vertex is boosting its gene-editing program for the treatment of Duchenne muscular dystrophy and myotonic dystrophy type 1. In gene editing, DNA is changed -- or literally "edited" -- in order to treat disease. It's wise of Vertex to pursue this therapeutic technique now. At a 25% compound annual growth rate, the global gene-editing market is expected to reach $4.4 billion by 2023, according to BCC Research. Grand View Research forecasts that the market will hit $8 billion by 2025.

Most recently, Vertex and CRISPR announced encouraging results from phase 1/2 clinical trials of the CTX001 gene-editing therapy in two blood disorders: transfusion-dependent beta-thalassemia and severe sickle cell disease. Early patients were transfusion-independent months after treatment, and the data demonstrated clinical proof of concept. The companies will provide more data later this year.

Vertex's annual revenue has increased steadily since 2015. As for earnings, they have soared past analysts' estimates in the past four quarters. For instance, in the most recent quarter, earnings per share came in at $2.56 compared with the average forecast of $1.84.

History isn't always indicative of the future. One small issue facing Vertex is that its up-and-coming therapies are all in phase 2 studies, or in earlier review stages. So we can't expect new drug approvals in the near term.

That said, Vertex's cystic fibrosis platform is strong enough to power the company along until earlier-stage programs bear fruit. Considering Vertex's recent approval of Trikafta, and the drug's hugely successful debut, it's reasonable to expect revenue to climb moving forward.

Vertex is trading at 51 times trailing-12-month earnings, and the shares have surpassed Wall Street's average price target by about 3%. By comparison, other profitable biotech companies like Amgen (NASDAQ:AMGN) and Gilead Sciences (NASDAQ:GILD) trade at just under 20 times earnings. So, Vertex isn't cheap. Still, for the three reasons above, I think this biotech stockhas plenty of room to move higher over the long term.

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3 Reasons Why Vertex Is a Great Stock to Buy and Hold for the Long Term - Motley Fool

Los Angeles, United State: Complete study of the global CRISPR And CRISPR-Associated (Cas) Genes market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global CRISPR And CRISPR-Associated (Cas) Genes industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on CRISPR And CRISPR-Associated (Cas) Genes production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

The research study includes great insights about critical market dynamics, including drivers, restraints, trends, and opportunities. It also includes various types of market analysis such as competitive analysis, manufacturing cost analysis, manufacturing process analysis, price analysis, and analysis of market influence factors. It is a complete study on the global CRISPR And CRISPR-Associated (Cas) Genes market that can be used as a set of effective guidelines for ensuring strong growth in the coming years. It caters to all types of interested parties, viz. stakeholders, market participants, investors, market researchers, and other individuals associated with the CRISPR And CRISPR-Associated (Cas) Genes business.

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It is important for every market participant to be familiar with the competitive scenario in the global CRISPR And CRISPR-Associated (Cas) Genes industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key Players Mentioned in the Global CRISPR And CRISPR-Associated (Cas) Genes Market Research Report: , Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, GE Healthcare Dharmacon

Global CRISPR And CRISPR-Associated (Cas) Genes Market Segmentation by Product: Genome Editing, Genetic engineering, gRNA Database/Gene Librar, CRISPR Plasmid, Human Stem Cells, Genetically Modified Organisms/Crops, Cell Line Engineering

Global CRISPR And CRISPR-Associated (Cas) Genes Market Segmentation by Application: , Biotechnology Companies, Pharmaceutical Companies, Academic Institutes, Research and Development Institutes

The report has classified the global CRISPR And CRISPR-Associated (Cas) Genes industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the CRISPR And CRISPR-Associated (Cas) Genes manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall CRISPR And CRISPR-Associated (Cas) Genes industry.

Additionally, the industry analysts have studied key regions including North America, Europe, Asia Pacific, Latin America, and Middle East and Africa, along with their respective countries. Here, they have given a clear-cut understanding of the present and future situations of the global CRISPR And CRISPR-Associated (Cas) Genes industry in key regions. This will help the key players to focus on the lucrative regional markets.

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Table of Content

Table of Contents Executive Summary 1 CRISPR And CRISPR-Associated (Cas) Genes Market Overview1.1 Product Overview and Scope of CRISPR And CRISPR-Associated (Cas) Genes1.2 CRISPR And CRISPR-Associated (Cas) Genes Segment by Type1.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate Comparison by Type (2014-2025)1.2.2 Genome Editing1.2.3 Genetic engineering1.2.4 gRNA Database/Gene Librar1.2.5 CRISPR Plasmid1.2.6 Human Stem Cells1.2.7 Genetically Modified Organisms/Crops1.2.8 Cell Line Engineering1.3 CRISPR And CRISPR-Associated (Cas) Genes Segment by Application1.3.1 CRISPR And CRISPR-Associated (Cas) Genes Consumption Comparison by Application (2014-2025)1.3.2 Biotechnology Companies1.3.3 Pharmaceutical Companies1.3.4 Academic Institutes1.3.5 Research and Development Institutes1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Market by Region1.3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size Region1.3.2 North America Status and Prospect (2014-2025)1.3.3 Europe Status and Prospect (2014-2025)1.3.4 China Status and Prospect (2014-2025)1.3.5 Japan Status and Prospect (2014-2025)1.3.6 Southeast Asia Status and Prospect (2014-2025)1.3.7 India Status and Prospect (2014-2025)1.4 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size1.4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue (2014-2025)1.4.2 Global CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2025) 2 Global CRISPR And CRISPR-Associated (Cas) Genes Market Competition by Manufacturers2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Manufacturers (2014-2019)2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Share by Manufacturers (2014-2019)2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Average Price by Manufacturers (2014-2019)2.4 Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Production Sites, Area Served, Product Types2.5 CRISPR And CRISPR-Associated (Cas) Genes Market Competitive Situation and Trends2.5.1 CRISPR And CRISPR-Associated (Cas) Genes Market Concentration Rate2.5.2 CRISPR And CRISPR-Associated (Cas) Genes Market Share of Top 3 and Top 5 Manufacturers2.5.3 Mergers & Acquisitions, Expansion 3 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Regions3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Regions3.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Regions (2014-2019)3.3 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.4 North America CRISPR And CRISPR-Associated (Cas) Genes Production3.4.1 North America CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.5 Europe CRISPR And CRISPR-Associated (Cas) Genes Production3.5.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.5.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.6 China CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2019)3.6.1 China CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.6.2 China CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.7 Japan CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2019)3.7.1 Japan CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.7.2 Japan CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019) 4 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Regions4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Regions4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.3 Europe CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.4 China CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.5 Japan CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019) 5 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price Trend by Type5.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Type (2014-2019)5.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Type (2014-2019)5.3 Global CRISPR And CRISPR-Associated (Cas) Genes Price by Type (2014-2019)5.4 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth by Type (2014-2019) 6 Global CRISPR And CRISPR-Associated (Cas) Genes Market Analysis by Applications6.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Market Share by Application (2014-2019)6.2 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Growth Rate by Application (2014-2019) 7 Company Profiles and Key Figures in CRISPR And CRISPR-Associated (Cas) Genes Business7.1 Caribou Biosciences7.1.1 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.1.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.1.3 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.1.4 Main Business and Markets Served7.2 Addgene7.2.1 Addgene CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.2.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.2.3 Addgene CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.2.4 Main Business and Markets Served7.3 CRISPR THERAPEUTICS7.3.1 CRISPR THERAPEUTICS CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.3.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.3.3 CRISPR THERAPEUTICS CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.3.4 Main Business and Markets Served7.4 Merck KGaA7.4.1 Merck KGaA CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.4.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.4.3 Merck KGaA CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.4.4 Main Business and Markets Served7.5 Mirus Bio LLC7.5.1 Mirus Bio LLC CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.5.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.5.3 Mirus Bio LLC CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.5.4 Main Business and Markets Served7.6 Editas Medicine7.6.1 Editas Medicine CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.6.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.6.3 Editas Medicine CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.6.4 Main Business and Markets Served7.7 Takara Bio USA7.7.1 Takara Bio USA CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.7.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.7.3 Takara Bio USA CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.7.4 Main Business and Markets Served7.8 Thermo Fisher Scientific7.8.1 Thermo Fisher Scientific CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.8.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.8.3 Thermo Fisher Scientific CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.8.4 Main Business and Markets Served7.9 Horizon Discovery Group7.9.1 Horizon Discovery Group CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.9.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.9.3 Horizon Discovery Group CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.9.4 Main Business and Markets Served7.10 Intellia Therapeutics7.10.1 Intellia Therapeutics CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.10.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.10.3 Intellia Therapeutics CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.10.4 Main Business and Markets Served7.11 GE Healthcare Dharmacon 8 CRISPR And CRISPR-Associated (Cas) Genes Manufacturing Cost Analysis8.1 CRISPR And CRISPR-Associated (Cas) Genes Key Raw Materials Analysis8.1.1 Key Raw Materials8.1.2 Price Trend of Key Raw Materials8.1.3 Key Suppliers of Raw Materials8.2 Proportion of Manufacturing Cost Structure8.3 Manufacturing Process Analysis of CRISPR And CRISPR-Associated (Cas) Genes8.4 CRISPR And CRISPR-Associated (Cas) Genes Industrial Chain Analysis 9 Marketing Channel, Distributors and Customers9.1 Marketing Channel9.1.1 Direct Marketing9.1.2 Indirect Marketing9.2 CRISPR And CRISPR-Associated (Cas) Genes Distributors List9.3 CRISPR And CRISPR-Associated (Cas) Genes Customers 10 Market Dynamics10.1 Market Trends10.2 Opportunities10.3 Market Drivers10.4 Challenges10.5 Influence Factors 11 Global CRISPR And CRISPR-Associated (Cas) Genes Market Forecast11.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast11.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate Forecast (2019-2025)11.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue and Growth Rate Forecast (2019-2025)11.1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Price and Trend Forecast (2019-2025)11.2 Global CRISPR And CRISPR-Associated (Cas) Genes Production Forecast by Regions (2019-2025)11.2.1 North America CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.3 China CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.4 Japan CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.3 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast by Regions (2019-2025)11.3.1 North America CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.3 China CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.4 Japan CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.4 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue and Price Forecast by Type (2019-2025)11.5 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast by Application (2019-2025) 12 Research Findings and Conclusion 13 Methodology and Data Source13.1 Methodology/Research Approach13.1.1 Research Programs/Design13.1.2 Market Size Estimation13.1.3 Market Breakdown and Data Triangulation13.2 Data Source13.2.1 Secondary Sources13.2.2 Primary Sources13.3 Author List13.4 Disclaimer

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CRISPR And CRISPR-Associated (Cas) Genes Market Size, Key Trends, Challenges and Standardization, Research, Key Players, Economic Impact and Forecast...

The report on the CRISPR Genome Editing market provides a birds eye view of the current proceeding within the CRISPR Genome Editing market. Further, the report also takes into account the impact of the novel COVID-19 pandemic on the CRISPR Genome Editing market and offers a clear assessment of the projected market fluctuations during the forecast period. The different factors that are likely to impact the overall dynamics of the CRISPR Genome Editing market over the forecast period (2019-2029) including the current trends, growth opportunities, restraining factors, and more are discussed in detail in the market study.

For top companies in United States, European Union and China, this report investigates and analyzes the production, value, price, market share and growth rate for the top manufacturers, key data from 2019 to 2025.

The CRISPR Genome Editing market report firstly introduced the basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the CRISPR Genome Editing market report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

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The major players profiled in this CRISPR Genome Editing market report include:

The key players covered in this studyEditas MedicineCRISPR TherapeuticsHorizon DiscoverySigma-AldrichGenscriptSangamo BiosciencesLonza GroupIntegrated DNA TechnologiesNew England BiolabsOrigene TechnologiesTransposagen BiopharmaceuticalsThermo Fisher ScientificCaribou BiosciencesPrecision BiosciencesCellectisIntellia Therapeutics

Market segment by Type, the product can be split intoGenetic EngineeringGene LibraryHuman Stem CellsOthersMarket segment by Application, split intoBiotechnology CompaniesPharmaceutical CompaniesOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

The study objectives of this report are:To analyze global CRISPR Genome Editing status, future forecast, growth opportunity, key market and key players.To present the CRISPR Genome Editing development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by type, market and key regions.

In this study, the years considered to estimate the market size of CRISPR Genome Editing are as follows:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year 2020 to 2026For the data information by region, company, type and application, 2019 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Globally Leading Manufacturers of CRISPR Genome Editing product Scale up Production to Meet Sharp Spike in Demand Fueled by COVID-78 - Personal Injury...

This high-end research comprehension on the Global CRISPR Technology Market renders major impetus on detailed growth facets, in terms of product section, payment and transaction platforms, further incorporating service portfolio, applications, as well as a specific compilation on technological interventions that facilitate ideal growth potential of the market.

The report is so designed as to direct concrete headways in identifying and deciphering each of the market dimensions to evaluate logical derivatives which have the potential to set the growth course in the aforementioned CRISPR Technology market. Besides presenting notable insights on market factors comprising above determinants, this specific, innately crafted research report offering further in its subsequent sections states information on regional segmentation, as well as thoughtful perspectives on specific understanding comprising region specific developments as well as leading market players objectives to trigger maximum revenue generation and profits.

This study covers following key players:Thermo Fisher ScientificMerck KGaAGenScriptIntegrated DNA Technologies (IDT)Horizon Discovery GroupAgilent TechnologiesCellectaGeneCopoeiaNew England BiolabsOrigene TechnologiesSynthego CorporationToolgen

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This illustrative research report on the CRISPR Technology market is an all-in-one, ready to use handbook of market dynamics that upon mindful inference lends valuable insights on market developments, growth trajectory, dominant trends as well as technological sophistication as well as segment expansion and competition spectrum that have a strong bearing on the growth probabilities of the CRISPR Technology market.

This particular section of the CRISPR Technology market report specifically stresses upon various indigenous tactical discretion that eventually contributed towards soliciting heralding market consolidation, impeccable stability and sustainable revenue pools, the ultimate touchstone to judge the potency of the CRISPR Technology market.

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Market segment by Type, the product can be split into EnzymesKitsgRNALibrariesDesign Tools

Market segment by Application, split into BiomedicalAgricultural

The report further unveils pertinent details about segment contribution in coining ample revenue flow, sustainability and long term growth in global CRISPR Technology market. A thorough knowledge base of market facets remains integral and indispensable to decode CRISPR Technology market prognosis. This recent research compilation on the CRISPR Technology market presents a deep analytical review and a concise presentation of ongoing market trends that collectively inculcate a strong influence on the growth trajectory of the aforementioned CRISPR Technology market.

The report sheds light on the particular segment that sets revenue maximization, rolling, thus incurring steady growth in revenues and contributing towards steady sustenance of the CRISPR Technology market. This well versedreport is thoughtfully crafted to arm report readers with convincing market insights on the mettle of all aforementioned factors that propel relentless growth despite significant bottlenecks in the CRISPR Technology market.

Some Major TOC Points:1 Report Overview2 Global Growth Trends3 Market Share by Key Players4 Breakdown Data by Type and ApplicationContinued

In addition to all of the above stated inputs, discussed at length in the report, the report sheds tangible light on dynamic segmentation based on which the market has been systematically split into prominent segments inclusive of type, end use technology, as well as region specific diversification of the CRISPR Technology market to encourage highly remunerative business discretion.

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Global CRISPR Technology Market Expanding Rapidly with Forecast 2025 and Top Players : Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA...

BERKELEY, Calif. Great partnerships depend on a rare combination of shared vision, mutual trust, and complementary skillsets. Scientists from Stanford University and Berkeley Labs Molecular Foundry may have found just that in a newly formed collaboration. Since March, research teams from these two institutions have been developing a new therapeutic that uses CRISPR technology in combination with a novel lipitoid delivery system to fight COVID-19 and other RNA viruses.

Last year, Stanley Qi, an assistant professor in the departments of bioengineering, and chemical and systems biology at Stanford, along with his team pioneered a new technology called prophylactic antiviral CRISPR in human cells, or PAC-MAN. Their efforts were initially focused on developing PAC-MAN as a strategy for targeting and destroying influenzain humans. When the COVID-19 pandemic hit in January, they abruptly pivoted their research to adapt the technology for SARS-CoV-2.

PAC-MAN is based on the popular gene-editing tool, CRISPR, which has revolutionized modern medicine. The technology allows for unprecedented control over gene expression in many species, including humans. At its core, CRISPR is a technique for finding a specific sequence of DNA inside a cell and then altering or deleting it.

The process involves two components: an enzyme (Cas protein) that cuts through genetic material; and a piece of RNA that is used as a guide to direct Cas protein to the targeted gene sequence. PAC-MAN uses a specific version of the Cas protein, called Cas13, which slices through RNA such as that which makes up the genomes of viruses like influenza and SARS-CoV-2.

In a paper published in the journalCell, Qi and his team demonstrate that their PAC-MAN strategy can effectively degrade RNA from SARS-CoV-2 sequences and live influenza A virus in human lung cells. Notably, the authors perform bioinformatics analyses that suggested a group of only six guide RNAs can target more than 90% of all coronaviruses, giving PAC-MAN the potential to become a pan-coronavirus inhibition strategy.

There was one problem. For PAC-MAN to be effective in patients, particularly as an anti-COVID-19 therapy, it needs to be able to be delivered into lung cells. But my lab doesnt work on delivery methods, Qi explains in a media release.

Enter Michael Connolly, a principal scientific engineer associate in the Biological Nanostructures Facility at the Molecular Foundry. His work on synthetic molecules called lipitoidsprovides a possible solution to the delivery issue with PAC-MAN. Lipitoids were first developed 20 years ago by Connollys mentor, Ron Zuckermann, and have since been widely studied for potential therapeutic applications. They are nontoxic to humans and can facilitate entry of RNA or DNA into cells by encapsulating them into tiny nanoparticles, a seemingly perfect delivery system for the PAC-MAN technology.

Berkeley Labs Molecular Foundry has provided us with a molecular treasure that transformed our research, Qi says.

In late April, the PAC-MAN/lipitoid system had its first test run in a sample of human lung cells. According to Qi, this approach reduced the amount of synthetic SARS-CoV-2 by greater than 90%. As an immediate next step to validate their technique, the team is collaborating with researchers at New York University and Karolinska Institute in Sweden. There, researchers are conducting animal trials with live SARS-CoV-2 infection. If successful, they hope to further develop the PAC-MAN/lipitoid system as a therapy against other viruses.

An effective lipitoid delivery, coupled with CRISPR targeting, could enable a very powerful strategy for fighting viral disease not only against COVID-19, but possibly against newly viral strains with pandemic potential, says Connolly.

One of the silver linings to emerge from the COVID-19 pandemic is how the scientific community has banded together to focus urgently on one topic. The joint effort of researchers from Stanford University and Berkeley Labs Molecular Foundry is an archetype of that collaborative spirit. As Qi notes, Its very rewarding to combine expertise and test new ideas across institutions in these difficult times.

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PAC-MAN to the Rescue: CRISPR-Based Tech on Cutting Edge of COVID-19 Therapeutics - Study Finds

Rising government funding and increase in the number of genomic projects, especially in the area of rare diseases, and a growing application horizon are all expected to drive the growth of the genome editing/genome engineering market.

New York, June 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Genome Editing/Genome Engineering Market by Technology, Product & Service, Application, End-User and Region - Global Forecast to 2025" - https://www.reportlinker.com/p05220258/?utm_source=GNW

The global genome editing/genome engineering market is projected to reach USD 11.2 billion in 2025 from USD 5.1 billion in 2020, at a CAGR of 17.0 % during the forecast period. Market growth is largely driven by factors such as the rise in government funding, growth in the number of genomics projects, high prevalence of infectious diseases & cancer, technological advancements, increasing production of genetically modified crops, and growing application areas of genomics. However, the high cost of genomic equipment will restrain the growth of this market.

CRISPR commanded the largest share of the market in 2019.Based on technology, the market is segmented into CRISPR, TALEN, ZFN, antisense, and other technologies.CRISPR accounted for the largest share of the genome editing/genome engineering market in 2019.

The large share of this segment can be attributed to the ease of use associated with the CRISPR technology and its ability to multiplex.

Pharmaceutical companies commanded the largest share of the genome editing/genome engineering market in 2019.By end user, the genome editing/genome engineering market is segmented into pharmaceutical companies, biotechnology companies, and academic & government research institutes.The pharmaceutical companies segment accounted for the largest share of the market.

The pharmaceutical companies segment accounted for the largest share of the genome editing/genome engineering market in 2019. This is due to the increasing prevalence of infectious diseases and cancer, which is driving research in the pharma sector for drug development.

The Asia Pacific region will register the highest growth in the global genome editing/genome engineering market during the forecast period.The Asia Pacific is estimated to grow at the highest CAGR during the forecast period. Factors such as the rapid growth in the pharmaceutical and biopharmaceutical industry, the rising number of genomic projects, and the presence of a genetically diverse population have supported the regions high growth rate.

In-depth interviews were conducted with chief executive officers (CEOs), marketing directors, other directors, and executives from various key organizations operating in the genome editing/genome engineering market. By Respondent Type: Supply Side (80%) and Demand Side (20%) By Designation: D-level (55%), C-level (20%), and Others (25%) By Region: North America (50%), Europe (20%), Asia Pacific (20%), and RoW (10%)

The major companies in the genome editing/genome engineering market include Thermo Fisher Scientific (US), Merck (Germany), Horizon Discovery Limited (UK), Lonza (Switzerland), GenScript (US), Eurofins Scientific (Luxembourg), and Sangamo Therapeutics (US).The study includes an in-depth competitive analysis of these key players in the genome editing/genome engineering market, along with their company profiles, recent developments, and key market strategies.

Research Coverage:The market study covers the genome editing/genome engineering market across various segments.It aims at estimating the market size and the growth potential of this market across different segments, such as products, technology, and region.

The study also includes an in-depth competitive analysis of the key players in the market, along with their company profiles, key observations related to their product and business offerings, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants in this market and provide information on the closest approximations of the revenue numbers for the overall genome editing/genome engineering market and its subsegments.This report will help stakeholders to understand the competitive landscape, gain insights to better position their businesses, and plan suitable go-to-market strategies.

The report will also help stakeholders to understand the pulse of the market and provide information on the key market drivers, restraints, opportunities, and challenges.Read the full report: https://www.reportlinker.com/p05220258/?utm_source=GNW

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The global genome editing/genome engineering market is projected to reach USD 11.2 billion in 2025 from USD 5.1 billion in 2020, at a CAGR of 17.0 % -...

CRISPR Technology Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost and revenue during the forecast period from 2020 to 2027. A CRISPR Technology market that includes Future Trends, Current Growth Factors, Meticulous Opinions, Facts, Historical Data and Statistically Supported And Industry-Validated Market Data.

This CRISPR Technology market research provides a clear explanation of how this market will make a growth impression during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

RequestSample PDF of CRISPR Technology Market Report https://www.worldwidemarketreports.com/sample/254147

Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT), Horizon Discovery Group, Agilent Technologies, Cellecta Inc., GeneCopoeia Inc., New England Biolabs, Origene Technologies Inc are some of the major organizations dominating the global market.

(*Note: Other Players Can be Added per Request)

Key players in the CRISPR Technology market were identified through a second survey, and market share was determined through a first and second survey. All measurement sharing, splitting and analysis were solved using a secondary source and a validated primary source. The CRISPR Technology market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure. The combination of these two factors will help key players meet the market reach and help to understand offered characteristics and customer needs.

The report also makes some important suggestions for the new CRISPR Technology market project before evaluating its feasibility. Overall, this report covers CRISPR Technology market Sales, Price, Sales, Gross Profit, Historical Growth and Future Prospects. It provides facts related to mergers, acquisitions, partnerships and joint venture activities prevalent in the market.

This report includes market size estimates of value (million US $) and volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the CRISPR Technology market, estimating the size of various other submarkets in the overall market. Major players in the market were identified through secondary studies, and market share was determined through primary and secondary studies. All ratio sharing, splitting and analysis were determined using the secondary source and the identified primary source.

What CRISPR Technology Market report offers:

Regions Covered in This Report

Complete knowledge of the CRISPR Technology market is based on the latest industry news, opportunities and trends in the expected region. The CRISPR Technology market research report provides clear insights into the influential factors expected to change the global market in the near future.

Remarkable Attributes of CRISPR Technology Market Report:

About WMR

Worldwide Market Reports is your one-stop repository of detailed and in-depth market research reports compiled by an extensive list of publishers from across the globe. We offer reports across virtually all domains and an exhaustive list of sub-domains under the sun. The in-depth market analysis by some of the most vastly experienced analysts provide our diverse range of clients from across all industries with vital decision making insights to plan and align their market strategies in line with current market trends.

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Know About CRISPR Technology Market: How COVID 19 is Reshaping the Industry: Thermo Fisher Scientific, Merck KGaA, GenScript - Cole of Duty

Gene Therapies Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost and revenue during the forecast period from 2020 to 2027. A Gene Therapies market that includes Future Trends, Current Growth Factors, Meticulous Opinions, Facts, Historical Data and Statistically Supported And Industry-Validated Market Data.

This Gene Therapies market research provides a clear explanation of how this market will make a growth impression during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

RequestSample PDF of Gene Therapies Market Report https://www.worldwidemarketreports.com/sample/179400

Adaptimmune Therapeutics Plc., Celgene Corporation, Crispr Therapeutics Ag, Glaxosmithkline Plc, Intellia Therapeutics Inc., Merck & Co. Inc., Novartis Ag, Regenxbio Inc., Voyager Therapeutics Inc., Abeona Therapeutics Inc. are some of the major organizations dominating the global market.

(*Note: Other Players Can be Added per Request)

Key players in the Gene Therapies market were identified through a second survey, and market share was determined through a first and second survey. All measurement sharing, splitting and analysis were solved using a secondary source and a validated primary source. The Gene Therapies market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure. The combination of these two factors will help key players meet the market reach and help to understand offered characteristics and customer needs.

The report also makes some important suggestions for the new Gene Therapies market project before evaluating its feasibility. Overall, this report covers Gene Therapies market Sales, Price, Sales, Gross Profit, Historical Growth and Future Prospects. It provides facts related to mergers, acquisitions, partnerships and joint venture activities prevalent in the market.

This report includes market size estimates of value (million US $) and volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the Gene Therapies market, estimating the size of various other submarkets in the overall market. Major players in the market were identified through secondary studies, and market share was determined through primary and secondary studies. All ratio sharing, splitting and analysis were determined using the secondary source and the identified primary source.

What Gene Therapies Market report offers:

Regions Covered in This Report

Complete knowledge of the Gene Therapies market is based on the latest industry news, opportunities and trends in the expected region. The Gene Therapies market research report provides clear insights into the influential factors expected to change the global market in the near future.

Remarkable Attributes of Gene Therapies Market Report:

About WMR

Worldwide Market Reports is your one-stop repository of detailed and in-depth market research reports compiled by an extensive list of publishers from across the globe. We offer reports across virtually all domains and an exhaustive list of sub-domains under the sun. The in-depth market analysis by some of the most vastly experienced analysts provide our diverse range of clients from across all industries with vital decision making insights to plan and align their market strategies in line with current market trends.

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Gene Therapies Market Expansion of Industry Creat New Opportunities for Key Players: Adaptimmune Therapeutics Plc., Celgene Corporation, Crispr...

From sophisticated techniques to easy-to-use strips, the world has deployed all kinds of tools to fightnovel coronavirus

Diagnostic tests are important to identify the sick,trace contacts tracing and ensure the disease does not spread. While the standard tests available for COVID-19 have been used for this purpose so far, researchers have been trying to find new methods for testing which are faster, cheaper and more reliable. The aim is also to develop tests that do not require infrastructure and can be used off-site like in rural areas.

Down To Earth lists the available test and also those that are in the pipeline:

RT-PCR

Medical professionals vouch for the accuracy of Reverse Transcription Polymerase Chain Reaction when it comes to diagnosis of infectious diseases. The test is based on the PCR technique, which replicates genetic material of the pathogen and helps identify it.

But PCR is customised to replicating DNA and for pathogens where the genetic material is RNA, as is the case of SARS-CoV-2 virus, an additional step of converting the RNA into DNA is added. This step is Reverse Transcription.

After a sample of mucous is collected from the nasopharynx using a swab, it is kept in a buffer solution which also helps in extraction of the virus. Then, using RT-PCR, its genetic material is replicated many times over usually 40 times.

During the replication process, primers (which are specific to the viral genetic material), enzymes, nucleotides and fluorescent probes are added.

The fluorescent probes offers a visual signal as soon as a strand is successfully copied. The test is thus also called as real-time RT-PCR (rRT-PCR or qRT-PCR, where q stands for quantitative).

Looks for viral genes Takessix hoursConducted in labs with high biosafety levels, BSL-2 &bill

Automated RT-PCR

RT-PCR test can also be automated. India has so far allowed two such tests which run on proprietary machines: TrueNat, by Molbio Diagnostics of India, and Xpert Xpress, by Cepheid of USA.

Both were originally used to test for TB. TrueNat was approved for COVID-19 by ICMR on April 4. Its battery-operated kits are the size of a telephone and can be taken out into the field to test as many as 15 individuals a day.

The Indian Council of Medical Research (ICMR) allowed the use of Xpert Xpress on April 19 under emergency use authorisation. The test is fully automated and provides the results in 45 minutes.

Four tests can be performed simultaneously. Both tests require positive results to be confirmed by RT-PCR.

Looks for viral genes Takes 35-50 minutes Conducted in BSL-2 labs, portable Positive results have to be confirmed by RT-PCR

Antibody tests

These can be manual or automated immunoassays. They identify the IgM and IgG antibodies developed against the SARS-CoV-2. For the test, whole blood, serum and plasma can be used and if antibodies are present in the sample, these bind to the antigen immobilised on the test strip and give a coloured reaction.

Such test kits are easy to use, provides quick results and are also effective in identifying asymptomatic patients. There is a risk of getting false positives and results need to be confirmed using a more advanced test.

In case of ELISA, (enzyme-linked immunosorbent assay) antibody test, as many as 90 tests can be run simultaneously. The results are more reliable than strip tests and can also provide quantitative data. The test provides results in around 2.5 hours.

Looks for antibodies Takes 20 minutes to 2.5hoursTo be conducted under doctors supervision Useful only for experimental or surveillance purposes

Antigen rapid tests

Antigen is a foreign substance produced by a pathogen, which induces an immune response in the body, especially the production of antibodies. The technique thus detects the presence of antigen or viral RNA in the sample.

The test is being developed by E25Bio, a Massachusetts-based biotech startup. The test relies on a patients nasopharyngeal swab. It resembles an over-the-counter pregnancy test and provides visual results within 15 minutes by detecting the presence of the virus in the sample.

The company is in the process of obtaining clearance under USFDAs emergency use authorisation model. Another company, OraSure Technologies Inc, has also received funds to develop a kit based on this technique.

Looks for viral genes In development stage

CRISPR

This test identifies the virus using the CRISPR genome editor, which uses tags that carry enzymes to the target DNA and cut it. For identifying the virus, a special tag has been developed whose enzyme, instead of cutting the gene, gives off a signal that it has reached the target.

A diagnostic kit based on CRISPR received emergency use authorisation by USFDA on May 7. Sherlock CRISPR SARS-CoV-2 kit is based on the specific high sensitivity enzymatic reporter unlocking technique.

The genetic material is extracted from the patients sample, amplified and incubated and detected with Cas13 enzyme. A commercially available paper dipstick is then used to confirm the presence of the virus with the naked eye.

A similar kit, DETECTR, is being developed by Mammoth Biosciences and UC San Francisco.

Looks for viral genes Takes 30 minutes As accurate as RT-PCR

Dogs

This experiment involves training dogs to sniff out people suffering from COVID-19.

The three-month trials would be led by the London School of Hygiene & Tropical Medicine and are backed with 500,000 pounds of government funding.

The UK hopes these dogs would be part of the countrys strategy to test people.

Each dog is expected to screen as many as 250 people every hour. The dogs would include breeds like labradors and cocker spaniels.

Dogs are already used to detect a host of diseases such as cancers and malaria.

The dogs use specific odour that a sick person gives out. Samples of breath and body odour can come from a number of sources like used face masks.

Looks for odour Under trial Can test 250 people per hour

This was first published in Down To Earths print edition (dated 1-16 June, 2020)

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Battle-ready for COVID-19: A look at the arsenal of various tests - Down To Earth Magazine

Gene editing Cas9 protein.

Applied computational biology discoveries vastly expand the range of CRISPRs access to DNA sequences.

In nature, bacteria use CRISPR as an adaptive immune system to protect themselves against viruses. Over the past decade, scientists have been able to successfully build upon that natural phenomenon with the discovery of CRISPR proteins found in bacteria the most widely used of which is the Cas9 enzyme. In combination with a guide RNA, Cas9 is able to target, cut, and degrade specific DNA sequences.

With applications ranging from the treatment of genetic diseases to the nutritional potency of agricultural crops, CRISPR has emerged as one of the most promising tools for genome editing. Cas9 enzymes, however, rely on specific DNA ZIP codes to pinpoint where to cut and edit. The most widely-used Cas9 fromStreptococcus pyogenes bacteria, SpCas9, requires two G nucleotides beside target sites. Less than 10 percent of DNA sequences meet this requirement.

In research published this month in both Nature Biotechnology andNature Communications,a team of computational biologists in the Media Labs Molecular Machines group and the MIT Center for Bits and Atoms have successfully engineered new proteins with enhanced genome editing capabilities, significantly broadening the spectrum of DNA sequences that can be accurately and effectively accessed.

This work was led by Pranam Chatterjee who recently completed his PhD in media arts and sciences; Noah Jakimo PhD 19, a Media Lab affiliate; and Media Lab Associate Professor Joseph Jacobson, in collaboration with lab members and researchers at the University of Massachusetts Medical School.

These new findings stem from the groups earlier breakthrough work in the computational discovery of Cas9 proteins. The teamidentified and experimentally characterized the Cas9 fromStreptococcus canisbacteria (ScCas9), which, while similar to SpCas9, had the ability to target a much broader range of target DNA sequences. That discovery expanded the number of locations that Cas9 enzymes could target from the original 10 percent of sites on the genome to nearly 50 percent. The team first reported those findings in 2018 inScience Advances.

To improve ScCas9 as a genome editing tool, the scientists computationally identified unique parts from similar Cas9 proteins to engineer an optimized version of ScCas9, which the team has named Sc++.Sc++ is the first known enzyme to simultaneously exhibit the three properties deemed essential for effective genome editing: broad targeting capability; robust cutting activity; and minimal errors due to off-targeting, notes Chatterjee.

Concurrently, the team successfully used their previous SPAMALOT algorithm to discoverStreptococcus macacaeCas9 (SmacCas9) that required two A nucleotides, rather than two Gs. Through domain swapping and further engineering, the team presents the newiSpyMac enzyme as one of the first known Cas9 editors not requiring a G, enabling targeting of an additional 20 percent of the genome that was previously inaccessible.To engineer iSpyMac, we simultaneously made hundreds of changes to SpCas9, knowing even a single change can break it,says Jakimo, the senior author on this second study. Our success is a testament to the wealth of microbial genomic data that can provide helpful clues about protein function with tools like SPAMALOT.

Erik Sontheimer, professor and vice chair of the RNA Therapeutics Institute at the University of Massachusetts Medical School, and a collaborator on the research, notes the significance of this work. The fewer targeting limitations we encounter, and the fewer compromises and trade-offs that have to be made between activity and accuracy, the greater the impact that CRISPR genome editing can have on biotechnology and human health. This is why Sc++ and iSpyMac provide such valuable new additions to the CRISPR editing arsenal.

As labs around the world have already begun to use the enzymes to successfully edit the genomes of various organisms, from rice to rabbits, the next goal for this research will be to develop tools to reach the remaining 30 percent of genome sequences. Chatterjee, in collaboration with the University of Zurich, is looking to unlock the final advances that will allow scientists to access any genomic sequence, and to address any type of gene mutation in the treatment of genetic diseases.

For now, however, as in many labs across the MIT campus, work has pivoted to address the Covid-19 pandemic. By applying computational design principles to engineer proteins that can target and bind to the invading SARS-CoV-2 virus, Chatterjee and the research team at the Media Lab are seeking to create enzymes to rapidly halt the virus, and enable cell recovery.

We engineer proteins differently, Chatterjee adds. Our ability to integrate computation and experimentation enables us to refine our algorithms and build impactful tools for a host of applications, from addressing genetic diseases to Covid-19, and beyond.

References:

A Cas9 with PAM recognition for adenine dinucleotides by Pranam Chatterjee, Jooyoung Lee, Lisa Nip, Sabrina R. T. Koseki, Emma Tysinger, Erik J. Sontheimer, Joseph M. Jacobson and Noah Jakimo, 18 May 2020, Nature Communications.DOI: 10.1038/s41467-020-16117-8

An engineered ScCas9 with broad PAM range and high specificity and activity by Pranam Chatterjee, Noah Jakimo, Jooyoung Lee, Nadia Amrani, Toms Rodrguez, Sabrina R. T. Koseki, Emma Tysinger, Rui Qing, Shilei Hao, Erik J. Sontheimer and Joseph Jacobson, 11 May 2020, Nature Biotechnology.DOI: 10.1038/s41587-020-0517-0

Minimal PAM specificity of a highly similar SpCas9 ortholog by Pranam Chatterjee, Noah Jakimo and Joseph M. Jacobson, 24 October 2018, Science Advances.DOI: 10.1126/sciadv.aau0766

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Nature-Inspired CRISPR Enzyme Discoveries Vastly Expand Genome Editing - SciTechDaily

-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia-

-Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-

-Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted-

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany.

CLIMB-111 Trial in Transfusion-Dependent Beta Thalassemia Updated ResultsData presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRISPR Therapeutics and Vertex announced initial data for the first TDT patient in November of 2019.

Patient 1 with TDT has the 0/IVS-I-110 genotype, which is associated with a severe phenotype similar to 0/0, and had a transfusion requirement of 34 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented today show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 100.0%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year.

Patient 2 with TDT has the 0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.4%.

Hemoglobin data over time are presented for Patient 1 and Patient 2 below.

Figure 1accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/35581299-d683-44b0-a75e-7a1a9b9fe9eb

CLIMB-121 Trial in Severe Sickle Cell Disease Updated Results Data presented today at EHA reflect longer-duration follow-up data for the first patient with SCD treated with CTX001. CRISPR Therapeutics and Vertex announced initial data for this first SCD patient in November of 2019.

Patient 1 with SCD experienced seven vaso-occlusive crises (VOCs) and five packed red blood cell transfusions per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. After CTX001 infusion, three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain; all subsequently resolved. New data presented today show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. Bone marrow allelic editing was 81.4% at 6 months. Figure 2 presents the hemoglobin data over time for this patient.

Figure 2 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7610c5bd-25c8-4f5b-be86-8bc16ed57eb1

With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as consistency of the therapeutic effect across patients, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. These highly encouraging early data represent one more step toward delivering on the promise and potential of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases.

The data announced today are remarkable, including the demonstration of clinical proof-of-concept in TDT, said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. While these are still early days, these data mark another important milestone for this program and for the field of gene editing. The results presented at this medical conference add to results previously shared demonstrating that CRISPR/Cas9 gene editing has the potential to be a curative therapy for severe genetic diseases like sickle cell and beta thalassemia.

In my 25 years of caring for children and young adults facing both sickle cell disease and beta thalassemia, I have seen how these diseases can adversely affect patients lives in very significant ways, said Dr. Haydar Frangoul, Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center and senior author of the abstract presented at the EHA virtual congress. I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease.

Recent Progress in the Phase 1/2 Clinical TrialsCLIMB-111 for TDT has dosed a total of 5 patients, and all patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of the first two patients. Additionally, CLIMB-111 has been expanded to allow enrollment of 0/0 patients and is in the process of being expanded to allow enrollment of pediatric patients ages 12 years or older.

CLIMB-121 for SCD has dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients.

The initial safety profile in these trials appears to be consistent with myeloablative busulfan conditioning and an autologous hematopoietic stem cell transplant.

In March 2020, clinical trial sites in the U.S. and Europe temporarily paused their elective hematopoietic stem cell transplant programs due to the COVID-19 pandemic, and as a result, CRISPR and Vertex temporarily paused conditioning and dosing in these trials. Enrollment, mobilization and drug product manufacturing in each trial remains ongoing. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites. The CLIMB-111 and CLIMB-121 clinical trials are ongoing, and patients will be followed for 2 years following CTX001 infusion. The companies expect to provide additional data in the second half of 2020.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT) from the U.S. FDA, Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), and Fast Track Designation from the FDA for both SCD and TDT.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex Collaboration CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

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

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases and activities at clinical trial sites) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is being presented at the European Hematology Associations virtual congress; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, and statements regarding our plans and expectations for our clinical trials and clinical trial sites, and our expectations regarding future data announcements. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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

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

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies at the 25th...

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The report offers a systematic presentation of the existing trends, growth opportunities, market dynamics that are expected to shape the growth of the CRISPR and CAS Gene market. The various research methods and tools were involved in the market analysis, to uncover crucial information about the market such as current & future trends, opportunities, business strategies and more, which in turn will aid the business decision-makers to make the right decision in future.

This Report Covers Leading Companies Associated in Worldwide CRISPR and CAS Gene Market: 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.

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The report begins with a brief introduction and market overview of the CRISPR and CAS Gene industry followed by its market scope and size. Next, the report provides an overview of market segmentation such as type, application, and region. The drivers, limitations, and opportunities for the market are also listed along with current trends and policies in the industry.

The key players profiled in this report include: 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.

Regions included:

o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

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Key Benefits:

o This study gives a detailed analysis of drivers and factors limiting the market expansion of CRISPR and CAS Gene

o The micro-level analysis is conducted based on its product types, end-user applications, and geographies

o Porters five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players

o By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this CRISPR and CAS Gene market

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Table of Contents

Report Overview: It includes the CRISPR and CAS Gene market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary: This section of the report gives information about CRISPR and CAS Gene market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players: Here, key players of the CRISPR and CAS Gene market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study: All of the regions and countries analyzed in the CRISPR and CAS Gene market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

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Impact of COVID-19 on CRISPR and CAS Gene Market Potential Growth and Forecast Period 2020-2027 | By Leading Players Caribou Biosciences Inc., CRISPR...

Global Gene Editing Tools market report gives a top to bottom investigation of the general market over a period from 2020-2027. The Gene Editing Tools Market report additionally gives the market sway and new open doors made because of the COVID19 fiasco.

As a training, this research report consolidating the effect of COVID19 available in each report as a worth included segment. Report incorporate income Impact investigation, interruptions and new open doors in the gracefully chain, overhauled merchant scene blend, new open doors mapping, and then some. Report resolved to rise triumphant against this COVID-19 pandemic and to guarantee that customers have zero to least disturbance in business results.

The global Gene Editing Tools market report covers major market players such as

Thermofisher ScientificCRISPR TherapeuticsEditas MedicineNHGRIIntellia TherapeuticsMerck KGaAHorizon

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The Gene Editing Tools market report next half moreover sheds investigate on the hole among flexibly and utilization. Aside from the referenced business development pace of market in 2027 is additionally clarified. Moreover, type and application insightful utilization tables and figures of Gene Editing Tools market are given.

Execution examination of Gene Editing Tools industry 2020 featuring ongoing business sector development, patterns and advancement 2027 figure report. The global Gene Editing Tools market report additionally contemplates the assembling cost structure and presents the different subtleties, for example, crude material, the general creation process, and the business chain structure.

Global Gene Editing Tools Market Segmentation By Type:

Zinc finger nucleases (ZFNs)Transcription Activator-Like Effector-based Nucleases (TALENs)CRISPR-Cas system

Global Gene Editing Tools Market Segmentation By Applications:

Sickle Cell DiseaseHeart DiseaseDiabetesAlzheimers DiseaseObesityOthers

Global Gene Editing Tools Market Segmentation By Regions:

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Furthermore, the Gene Editing Tools report consists of the important information related to the growth rate, top players of the parent market, product development, and others also by considering the past and present values of the market report predicting the forecasting values of the Gene Editing Tools market. Along with these things report selected the appropriate SWOT analysis which guides the various opportunities and growth scope for the Gene Editing Tools market.

The research report Gene Editing Tools market consist the in-depth information about the data analysis by using the figures, graphs, pie charts, tables and bar graphs. With the help of these users easily understand the analyzed data in a better and easy way. Also, the report provides the different business challenges which are impacting market growth in a positive and negative direction.

There are 13 Chapters to display the Global Gene Editing Tools market:

Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation overviewChapter 2: Market Competition by ManufacturersChapter 3: Production by RegionsChapter 4: Consumption by RegionsChapter 5: Production, By Types, Revenue and Market share by TypesChapter 6: Consumption, By Applications, Market share (%) and Growth Rate by ApplicationsChapter 7: Complete profiling and analysis of ManufacturersChapter 8: Manufacturing cost analysis, Raw materials analysis, Region-wise manufacturing expenses.Chapter 9: Industrial Chain, Sourcing Strategy and Downstream BuyersChapter 10: Marketing Strategy Analysis, Distributors/TradersChapter 11: Market Effect Factors AnalysisChapter 12: Market ForecastChapter 13: Gene Editing Tools Research Findings and Conclusion, Appendix, methodology and data source.

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Global Gene Editing Tools Market 2020 Impact of COVID-19, Future Growth Analysis and Challenges | Thermofisher Scientific, CRISPR Therapeutics,...

Also known as genome editing with engineered nucleases (GEEN), genome editing is a method of altering DNA within a cell in a safe manner. The technique is also used for removing, adding, or modifying DNA in the genome. By thus editing the genome, it is possible to change the primary characteristic features of an organism or a cell.

The global genome editing market can be segmented on the basis of delivery method, technology, application, and geography. By technology, the global genome editing market can be segmented into Flp-In, CRISPR, PiggyBac, and ZFN. Based on delivery method, in vivo and ex vivo can be the two broad segments of the global genome editing market. By application, the global genome editing market can be categorized into medicine, academic research, and biotechnology.

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Global Genome Editing Market: Key Trends

Since genome editing is gaining rising adoption in the domain of scientific research for attaining a better understanding of biological aspects of organisms and how they work, the global genome editing market is likely to promise considerable growth over the forthcoming years. More importantly, genome editing is being used by medical technologies, where it can be used for modifying human blood cells which can then be placed back in the body for treating conditions such as AIDS and leukemia. The technology can also be potentially utilized to combat infections such as MRSA as well as simple genetic disorders including hemophilia and muscular dystrophy.

Global Genome Editing Market: Market Potential

As more easy-to-use and flexible genome technologies are being developed, greater potential of genome editing is being recognized across bioprocessing and treatment modalities. For instance, in May 2017, MilliporeSigma announced that it successfully developed a novel genome editing tool which can make the CRISPR system more productive, specific, and flexible. The researchers thus have a more number of experimental options along with faster results.

All this can lead to a growing rate of drug development, enabling access to more advanced therapies. Proxy-CRISPR, the new technique, makes access to earlier inaccessible aspects of the genome possible. As most of the existing CRISPR systems cannot manage without re-engineering of human cells, the new method is expected to gain more popularity by virtue of the elimination of the need for re-engineering, simplifying the procedures.

Several other market players are focusing on clinical studies with a view to produce effective treatments for different health conditions. For example, another major genome editing firm, Editas Medicine, Inc. announced the results of its pre-clinical study displaying the success of the CEP290 gene present in the retina of primates in the same month. With the positive results of the study, the companys belief in the vast potential of its candidate in the treatment of a genetically inherited retinal degenerative disease, Leber congenital amaurosis type 10, affecting childrens eyesight has been reinforced.

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Global Genome Editing Market: Regional Outlook

By geography, the global genome editing market can be segmented into Latin America, Europe, Asia Pacific, the Middle East and Africa, and North America. North America registered the highest growth in the past, and has been claiming the largest portion of the global genome editing market presently. The extraordinary growth of this region can be attributed to greater adoption of cutting edge technologies across several research organizations. The U.S., being the hub of research activities, is expected to emerge as the leading contributor. Asia Pacific is also likely to witness tremendous demand for genome editing over the forthcoming period, assisting the expansion of the global genome editing market.

Global Genome Editing Market: Competitive Analysis

CRISPR THERAPEUTICS, Caribou Biosciences, Inc., Sigma Aldrich Corporation, Sangamo, Intellia Therapeutics, Inc., Editas Medicine, Thermo Fisher Scientific, Inc., and Recombinetics, Inc are some of the key firms operating in the global genome editing market.

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Genome Editing Market Predicted to Accelerate the Growth by 2017-2025 - Owned

The Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market report offers users the detailed study of the market and its main aspects. There are different marketing strategies that every marketer looks up to in order to ace the competition in the Global market. Some of the primary marketing strategies that is needed for every business to be successful are Passion, Focus, Watching the Data, Communicating the value To Your Customers, Your Understanding of Your Target Market. There is a target set in market that every marketing strategy has to reach. Some of the important aspects analyzed in the report includes market share, production, key regions, revenue rate as well as key players. This Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology report also provides the readers with detailed figures at which the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market was valued in the historical year and its expected growth in upcoming years. Besides, analysis also forecasts the CAGR at which the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology is expected to mount and major factors driving markets growth.

This study covers following key players:Thermo Fisher ScientificMerckGenScriptIntegrated DNA TechnologiesHorizon Discovery GroupAgilent TechnologiesCellectaGeneCopoeiaNew England BiolabsOrigene TechnologiesSynthego CorporationToolgen

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A significant development has been recorded by the market of Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology, in past few years. It is also for it to grow further. To analyze the Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market, the analysis methods used are SWOT analysis and PESTEL analysis. To identify what makes the business stand out and to take the chance to gain advantage from these findings, SWOT analysis is used by marketers. Whereas PESTEL analysis is the study concerning Economic, Technological, legal political, social, environmental matters. For the analysis of market on the terms of research strategies, these techniques are helpful. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry.

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Market segment by Type, the product can be split intoProductsServices

Market segment by Application, split intoBiomedical ApplicationsAgricultural ApplicationsIndustrial ApplicationsBiological Research

A systematized methodology is used to make a Report on the Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market. For the analysis of market on the terms of research strategies, these techniques are helpful. All the information about the Products, manufacturers, vendors, customers and much more is covered in research reports. The market tends to be highly competitive in nature as the number of vendors present in the market is too high.

The Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market has its impact all over the globe. On Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology industry is segmented on the basis of product type, applications, and regions. It also focusses on market dynamics, Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology growth drivers, developing market segments and the market growth curve is offered based on past, present and future market data. The industry plans, news, and policies are presented at a Global and regional level.

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Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market 2020 By Size, Share, Trend, Production, High Demand,...

As businesses and schools seek to reopen, most public health experts agree that Covid-19 testing is needed more than ever.

The team behind Ginkgo Bioworks, a genetic engineering start-up, is going all in.

Using equipment from Illumina, a maker of DNA sequencing machines, the company which made CNBC's 2020 Disruptor 50 list is working on technology to run a half million tests per day, said Jason Kelly, Ginkgo's co-founder and CEO.The technology, if approved by federal regulators, will be saliva-based, which in theory would make it easier for consumers to get tested than using the nasal swabs most tests employ today.

Jason Kelly, Founder, Ginkgo Bioworks

Scott Mlyn | CNBC

"We didn't initially have enough tests, but now we've ramped up to about 400,000 per day," he said. "That's enough for our clinical diagnostics needs. ... However, we are now entering phase 2 of this thing."

Ginkgo, a darling of the burgeoning synthetic biology sector, got its start in 2009 when a group of MIT scientists got together to develop biotechnology tools for industries including agriculture, pharmaceuticals and cosmetics. In essence, it develops custom microorganisms that aim to replace technology with biology. Think of it as a way to program cells, a bit like you'd program computers.

"We program DNA and cells to make them do new things," said Kelly, who describes the company as the largest designers of "printing DNA" in the world.

The Boston-based company has raised close to $1 billion to date, as investors clamor to throw money into companies at the intersection of health care and technology. Kelly maintains that enthusiasm is warranted. The cost of sequencing DNA data is coming down faster than the cost of processing data on computers,outpacingMoore's law.

As Kelly puts it, "the chips aren't getting that much faster," but in biology things are "exponentially improving."

Right now Kelly believes Ginkgo can best apply its technology to help ramp up coronavirus testing in the U.S.

As the CEO explained, the initial crop of tests were primarily used to determine if people experiencing Covid-19 symptoms did, in fact, have the virus. But now, as people are going back to their lives, there will be an increasing need for regular testing of people who don't have symptoms.

For instance, Amazon plans to test its fulfillment center workers every two weeks, as well as monitor outbreaks in the community.

That means the country is going to need a lot more coronavirus tests.

Ginkgo started surveyingthe various techniques to scale up testing back in the spring, including antigen (a technology that looks for viral surface proteins), CRISPR-based (a genome editing technique) and next-generation sequencing approaches. Companies have only recently been granted emergency-use authorizations from the U.S. Food and Drug Administration for these types of tests. All of them, if ramped up, could theoretically augment the polymerase chain reaction tests that are currently the gold standard for Covid-19.

Ginkgo has decided to focus on next-generation sequencing with Illumina, which has already been granted an emergency-use approvalfor its Covid-19 test that is designed to sequence the full genome of the virus.

"Beyond diagnostic testing, Illumina and a number of our customers are exploring NGS-based workflows to enable high-volume screening to support a return to work and school," Illumina CEO Francis deSouza said in a statement.

In May Ginkgo announced it had raised another $70 million, including from Illumina, to fund its expansion in the diagnostics field. It is also using the money tobuild out its own testing facility in its highly automated Boston Seaport labs.

More from Disruptor 50:Moderna CEO sees success with Covid-19 vaccineThe technology that will dominate daily life on the other side of coronavirusCLEAR poised to lead in biometric screening for Covid

Kelly can't predict exactly when the company will be rolling out its tests, but it hopes to get FDA approval this summer. He said the company is already starting to work with businesses to help advise them as they determine how to safely get employees back to work. Many are concerned about a potential second shutdown if there's another outbreak in their area.

He believes that testing and contact tracing, where government officials track down and warn people who might have been exposed to Covid-19, are key to reopening the economy.

"I'm also sensing that a lot of people don't have a ton of hope," Kelly said. "Now we have to really try. If we try, we can win."

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Ginkgo Bioworks CEO on scaling up Covid-19 testing: 'If we try, we can win' - CNBC