Archive for the ‘Crispr’ Category

Cyrus Biotechnology has teamed up with the Broad Institute to optimize CRISPR for use in humans. Feng Zhang, who had a hand in developing CRISPR, will serve as the Broads principal investigator for the collaboration.

One concern with using CRISPR-Cas9 to perform in vivo genome editing stems from the risk that the body will mount an immune response against the system. Those concerns have grown as researchers have shown that many people have antibodies against Cas9, reflecting the fact that the homologs of the protein used in genome editing systems are derived from bacteria that commonly infect people.

Cyrus, which lists Johnson & Johnson among its customers, thinks its technology can mitigate the risk of an immune reaction. That confidence reflects Cyrus experience of using software to identify and work around the epitopes in protein therapeutics that cause immunogenicity.

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We have validated our computational deimmunization platform in a variety of systems, and now seek to apply it where it can make a major impact. Given the extensive therapeutic possibilities of CRISPR systems, and the leading position the Broad Institute and Dr. Zhang hold, we are very excited to work in partnership with them to make these molecules more amenable for use in humans with maximal efficacy and minimal side effects, Cyrus CEO Dr. Lucas Nivn said in a statement.

Partnering with the Broad will allow Cyrus to combine its experience of deimmunization with the skills of researchers who helped put CRISPR on the map. Zhang, the Broads lead on the project, was at the forefront of efforts to optimize Cas9 for use in human cells.

The partners plan to publish their research and make the fruits of their collaboration available to the nonprofit and academic research community for free.

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Cyrus, the Broad team up to make in vivo CRISPR use safer - FierceBiotech

Washington (AFP)

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

- Cures -

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

- Bioterrorism -

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

2019 AFP

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2019: the year gene therapy came of age - FRANCE 24

What happened

Shares of Editas Medicine (NASDAQ:EDIT) rose more than 45% last month, according to data fromS&P Global Market Intelligence. The gene editing pioneer rose for reasons both internal and external.

The business announced an amended collaboration with Celgene (NASDAQ:CELG) for developing engineered immune cells and will receive an upfront payment of $70 million as a result of the new agreement. The company also enjoyed a bump from peer CRISPR Therapeutics, which reported promising results for the first two patients dosed with its lead drug candidate, CTX001. Investors took that as evidence that CRISPR-based medicines might be the real deal, although that's a mighty big leap.

The gene editing company also reported a business update and operating results for the third quarter of 2019, but there wasn't much to report for the pre-commercial entity.

Image source: Getty Images.

Editas Medicine started working with Juno Therapeutics, now owned by Celgene, in 2015. The idea was to combine the gene-editing platform of the former with the immunotherapy leadership of the latter. That's still the case, but the amended agreement scales back the specific types of engineered T cells that will be developed in the collaboration. It's a subtle, but potentially important, detail with (beneficial) ramifications for the long-term future of Editas Medicine.

It appears that the $70 million upfront payment was made in part to compensate Editas Medicine for the difference. After all, the company had already received $70 million in upfront, milestone, and execution payments under the original collaboration agreement. It's not immediately clear how the financial terms have changed, if they did at all, but the gene editing pioneer originally stood to receive up to $920 million in milestone payments.

Beyond that, there were several other updates provided in November:

The gene-editing landscape is still in the earliest stages of development. While CRISPR Therapeutics has taken an early lead as the top gene editing company, Editas Medicine is hoping to prove that its direct delivery approach will prove equally effective. The trial results the company will present in the coming years will become crucial tests for the future of CRISPR-gene editing, especially with competing techniques on the horizon.

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Here's Why Editas Medicine Jumped 45.3% in November - The Motley Fool

Oakland, CA, Dec. 03, 2019 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc., a biotechnology company developing CRISPR-based therapies to cure viral infectious diseases, today announced that veteran pharmaceutical executive William H. Carson, M.D. has joined the Board of Directors as an Independent Director.

Dr. Carson is the President & CEO of Otsuka Pharmaceutical Development & Commercialization, Inc. In this position, he led the companys development efforts in neuroscience, cardio-renal, and oncology, and was instrumental in the development and registration ofABILIFY MAINTENA(aripiprazole) as well as SAMSCA(tolvaptan). Dr. Carson joined Otsuka in 2002 as a board-certified psychiatrist and served as OPDCs Senior Vice President, Global Clinical Development, overseeing the development of all Otsuka-discovered compounds. During his career at Otsuka and earlier at Bristol-Myers Squibb (BMS), he was one of the key drivers in the development and commercialization ofABILIFY(aripiprazole). Dr. Carson received an A.B. degree in history and science from Harvard University and an M.D. degree from Case Western Reserve University. Dr. Carson plans to retire from Otsuka at the end of 2019.

Bill is an invaluable addition to Excisions Board of Directors, said Daniel Dornbusch, Excisions CEO. His extensive and highly regarded experience building successful companies as well as guiding products through early stage development, through clinical trials and to successful commercialization will accelerate Excisions activities throughout the organization. We are delighted that he will bring his insight and acumen to further Excisions growth.

I am honored to join Excisions Board of Directors at this key moment in the companys development, said Dr. Carson Their unique approach to developing cures for viral infectious diseases such as HIV, hepatitis B, JC virus, HSV and others has great potential to fulfill a key area of global health needs. Ive spent over 20 years helping companies grow successfully within the biopharmaceutical industry and look forward to leveraging my expertise to assist Excision during this transformative time.

About Excision BioTherapeutics

Excision BioTherapeutics, Inc., a biotechnology company developing CRISPR-based therapies to cure viral infectious diseases. Excision is focused on improving the lives of chronically ill patients by eliminating viral genomes from infected individuals. By using CRISPR in unique ways, the company has already demonstrated the first functional cure for HIV in animals. Excision is developing technologies and IP developed at Temple University and U.C. Berkeley. Excision is located in Oakland, California and is supported by Artis Ventures, Norwest Venture Partners, SilverRidge Venture Partners, Oakhouse Ventures, and Gaingels. For more information, please visitwww.excisionbio.com.

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CRISPR-focused Excision BioTherapeutics Strengthens Board of Directors Appointing Veteran Pharmaceutical Executive Bill Carson, MD - GlobeNewswire

Global CRISPR Technology Market is estimated to be over US$ 550.0 Million in 2018. It is anticipated to grow at a 24.0% CAGR from 2019 to 2030 and is expected to grow at a double digit CAGR during the forecasted period.The global CRISPR Technology market is segmented by product & services, application, end user, and region.

CRISPR Technology Market Overview and Introduction

GlobalCRISPR Technology Marketis estimated to be over US$ 550.0 Million in 2018. It is anticipated to grow at a 24.0% CAGR from 2019 to 2030and is expected to grow at a double digit CAGR during the forecasted period.The global CRISPR Technology market is segmented by product & services, application, end user, and region.

CRISPR Technology is relatively new technology used in genome editing or gene editing; CRISPR-CAS-9 is cluster of palindromic repeats and is found naturally in bacteria. These sequence enable the bacteria to protection them from virus by producing RNA segment or enzyme that cleaves the virus DNA and deactivates the virus. This ability of CRISPR-CAS9 has allowed scientist to make DNA and RNA libraries as per their need and applications. CRISPR-CAS9 technology have potential applications in the field of treating human diseases, creating gene libraries, and manipulating cell functions like metabolism.

The global CRISPR technology market is driven by growing focus of market players in CRISPR-CAS9 technology, availability of government and private funding and rising incidences related to genetic disorders are major factors driving the market. However, ethical issue, stringent regulatory policy and lack of skilled professionals are likely to restrain the market to certain extent.

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CRISPR Technology Market by Product & ServicesOn the basis of product, the CRISPR technology market is segmented into CRISPR Kits, Enzymes and Services. The CRISPR services are further sub-segmented into design & vector construction, cell line engineering, screening services and other services.CRISPR Technology Market by Application

Based on application, the market is segmented into biological & biomedical applications, agricultural applications, industrial applications and other applications.CRISPR Technology Market by End UserOn the basis of end user, the CRISPR technology market is segmented into academic institutes & research centers, contract research organizations (CROs), pharmaceutical and biotechnology companies and other end users.

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CRISPR Technology Market by RegionsThe global CRISPR technology industry can be segmented into North America, Europe, Asia Pacific, and Rest of World (ROW). North America dominated the market of CRISPR technology, followed by Europe and Asia Pacific. North America will continue to dominate the global CRISPR technology market in the forecast period owing to factors such as growing research in the field of CRISPR technology and adoption of CRISPR technology. Moreover However, Asia Pacific is expected to witness the highest CAGR, with the growth in this market centered at China, India, and Japan. Factors such as the government support are driving the growth of the CRISPR technology market in this region.

CRISPRTechnology Market Prominent Players

The prominent players in the global CRISPR Technology market are Thermo Fisher Scientific, Inc., GenScript, Merck KGaA, GeneCopoeia, Inc., Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Agilent Technologies, and Applied StemCell, Inc., among others.

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CRISPR Technology Market - Industry Growth, Analysis, Business Trends, Competitive Landscape, Regional Forecast to 2030 - Media Releases - CSO...

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.

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.

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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 Genes to 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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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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CRISPR and Cas Genes Market to Reach a Value of US$ 7234.5 Mn by the End of 2026 - News Description

Intraday Trading:OnMonday, Shares ofCRISPR Therapeutics AGmakes a change of -4.23% and now trading at $68.62 The EPS of CRSP stock is strolling at -0.46, measuring its EPS increase this year at -101.60%.

EPS is the part of a companys interest allotted to each outstanding share of natural accretion. EPS works as a gauge of a companys profitability. EPS is usually thought to be the and no-one else various important variable in circumscribing a shares price.

Snapshot: CRISPR Therapeutics AG,belongs tothe Healthcaresector andBiotechnologyindustry.

As an end, the firm has an EPS growth of -1828.00% for the coming year. Companys EPS for the past five years is considered at 0.00%, directing it to an EPS value of 0.00% for the next five years. Given the significance of distinguishing organizations that will guarantee income per share at a high rate, we later fixation to umpire how to recognize which organizations will accomplish high hoarding rates. One evident flaunting to distinguish high profit per part tally together organizations are to find organizations that have shown such develop past the p.s. 5 to 10 years. We cant have enough support the once will consistently mirror the troublesome, however coherently stocks that have developed profit per remittance unequivocally in the consequent to are a fine wagered to keep on producing results, therefore.

The firm has a complete market capitalization of 3.92B and a total of Outstanding outstanding shares.

Trading volume recorded for this company was about 1567613 shares as contrast to its average volume of 821.51K shares.

Technical Analysis of CRISPR Therapeutics AG in the Limelight:

ATR stands at 4.38 whileBetafactor of the stock stands at 0.00. A beta element is used to measure the volatility of the stock. Beta is a measurement unit of the volatility, or managed chance, of a security or a portfolio in contrast with the market in general. Beta is utilized in the capital resource valuing model (CAPM), which calculates the expected return of an asset based on its beta and expects market returns. Beta is also known as the beta coefficient. The stock remained 6.22% volatile for the week and 7.14% for the month.

Performance Review:The stock has shown the weekly performance of 10.32%, and monthly performance stands at 36.23%. The year-to-date (YTD) performance reflected a 140.18%, during the past three months the stock performs 53.10%, bringing six-month performance to 84.17%.

Analysts meantarget pricefor the company is $77.50whileanalysts mean suggestionis 2.10. A final price is the projected price level of a financial security stated by an investment analyst or advisor. It symbolizes a securitys price that, if achieved, results in a trader recognizing the best possible outcome for his investment. This is the price at which the trader or investor wants to exit his current position so he can realize the most reward.

Investigating theproductivity proportionsof CRSP stock, the speculator will discover its ROE, ROA, ROI remaining at -2.60%, -2.00%, and -40.70%, individually.

CRSPinstitutional ownership is held at 46.80% while insider ownership was 0.40%. As of now,CRISPR Therapeutics AGhas a P/S, P/E and P/B values of 18.42, 0.00 and 6.36 respectively. Its P/Cash is valued at 6.22.

Relative Strength Index (RSI):Therelative strength indexof the stock stands 67.30. The relative quality file (RSI) is a specific pointer utilized in the examination of budgetary markets. It is proposed to outline the present and recorded quality or shortcoming of a stock or market dependent on the end costs of an ongoing exchanging period. The pointer ought not to be mistaken for relative quality.

The RSI is most generally utilized on a 14-day time allotment, estimated on a measuring scale from zero (0) to 100, with high and low levels set apart at 70 and 30, individually. Shorter or longer periods are utilized for, on the other hand, shorter or longer standpoints. Progressively extraordinary high and low levels80 and 20, or 90 and 10happen less often yet demonstrate more grounded energy.

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Still Spinning In The Sand: CRISPR Therapeutics AG, (NASDAQ: CRSP) - Curious Coins

CAMBRIDGE, Mass. & MONTREAL Repare Therapeutics, a precision oncology company pioneering synthetic lethality to develop novel therapeutics that target specific vulnerabilities of tumors in genetically defined patient populations, announced today that it has made two additions to its leadership team and Board of Directors. Steve Forte is appointed as Executive Vice President & Chief Financial Officer and leads the Companys financial, capital markets and administrative operations. Samarth Kulkarni, PhD, CEO of CRISPR Therapeutics (NASDAQ:CRSP), has been appointed to Repares Board of Directors as an independent director.

We are thrilled to make these key additions of Steve and Sam to our executive team and board leadership respectively, said Lloyd M. Segal, President & Chief Executive Officer of Repare Therapeutics. Steve Forte will be based in Repares Montreal, QC, offices. Katina Dorton, who had previously served as Repares CFO, has departed to pursue other interests, and will continue to serve as an advisor to the Company.

Steve Forte

Steve is a senior financial leader who has managed in complex, large-scale healthcare financial environments. Until its recent sale to Ipsen for US$1.3 billion, he was CFO of Clementia Pharmaceuticals (NASDAQ:CMTA), a leading biotechnology innovator in treatments for rare diseases. His experience includes nearly a decade at Aptalis Pharma Inc., where he was responsible for the overall corporate controllership function of a multinational pharmaceutical company with approximately $700 million in annual revenue in six global operating entities. Steve led SEC reporting including the preparation of an SEC S-1 registration statement for a U.S. IPO prior to the sale of the company to Forest Labs for $2.9 billion. Prior to Clementia, Steve was CFO of Thinking Capital Financial Corporation, a leading Canadian financial technology firm sold to Purpose Investments in 2018.

Steve received his Bachelor of Commerce in Accountancy from Concordia University and is a CPA/CMA.

Samarth Kulkarni

Sam has served as CEO of CRISPR Therapeutics since 2017. He has significant expertise in strategy and operations in biotechnology and a wide range of related cutting-edge therapeutic technologies. He joined CRISPR in 2015 in the early stages of the company as Chief Business Officer, and then served as President and Chief Business Officer. Prior to joining CRISPR, Sam was a Partner at McKinsey & Company, where he had a leading role in the Pharmaceutical and Medical products practice. While at McKinsey, he co-led the biotech practice, where he focused on topics ranging from strategy to operations and led initiatives in areas such as personalized medicine and immunotherapy. Sam also serves as the Chairman of the Board of Directors of Casebia Therapeutics, a joint subsidiary formed by CRISPR Therapeutics and Bayer.

He received a Ph.D. in Bioengineering and Nanotechnology from the University of Washington and a B. Tech. from the Indian Institute of Technology. While at the University of Washington, he conducted research in the delivery of biological drugs and in the field of molecular diagnostics. He has authored several publications in leading scientific and business journals.

About Repare Therapeutics

Repare Therapeutics is pioneering synthetic lethality to develop novel therapeutics that target specific vulnerabilities of tumors in genetically defined patient populations. The companys initial focus is on novel targeted therapies in cancer types harboring defective DNA-damage response (DDR)- or genome instability-related functions. Repares SNIPRx platform combines a proprietary, high throughput, CRISPRenabled gene editing target discovery technology with highresolution protein crystallography, computational biology, medicinal chemistry and clinical informatics to rapidly generate small molecules for clinical investigation. The company is backed by leading global healthcare investors including Versant Ventures and MPM Capital. For additional information, please visit http://www.reparerx.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191203005090/en/

Contacts

Steve Forte Chief Financial Officer Repare Therapeutics Inc. info@reparerx.com

Originally posted here:
Repare Therapeutics Appoints Steve Forte as CFO and Samarth Kulkarni to Its Board of Directors - Financial Post

In recent times, Today on Tuesday, December 03, 2019, by making a change of 1.18% with the Gain (), the Healthcare stock (CRISPR Therapeutics AG) created a change of 2.27% from opening and finally closed its business at 69.43.

Earnings for each Share (EPS) are the part of a companys profit allocated to respectively outstanding share of common stock. EPS serves as a pointer to a companys profitability/success. EPS is considered to be the only most crucial variable in determining a shares price.

Eye Catching Stocks: CRISPR Therapeutics AG

Intraday Trading of the CRISPR Therapeutics AG:CRISPR Therapeutics AG, a Switzerland based Company, belongs to Healthcare sector and Biotechnology industry.

Trading volume, or volume, is the number of shares or contracts that point towards the full activity of a security or stock market for a given period. The company exchanged hands with 273826 shares contrast to its average daily volume of 843.13K shares. Relative Volume (or RVOL) is a volume indicator, meaning it assists measure shareholder interest in a stock. RVOL compares a stocks current volume to its previous amount over a specific period.

Performance Review:

Technical Analysis of CRISPR Therapeutics AG: Looking into the profitability ratios of CRSP stock, the shareholder will find its ROE, ROA and ROI standing at -2.6%, -2% and -40.7%, respectively. A profitability ratio is an estimate of profitability, which is a way to measure a companys performance. Profitability merely is the capacity to make a profit, and a gain is what is left over from income earned after you have deducted all costs and expenses related to obtaining the income.

The RSI most typically used on a 14-day timeframe, measured on a scale from 0-100, with high and low levels marked at between 70 and 30, respectively. Shorter or longer timeframes used for alternately shorter or longer outlooks. More supreme high and low levels80 and 20, or 90 and 10occur less frequently but indicate stronger momentum. The RSI provides signals that tell investors to buy when the currency oversold and to sell when it is overbought. The present relative strength index (RSI) reading is 67.93.

What do you mean by simple moving average (SMA)?

A simple moving average (SMA) is an arithmetic moving average calculated by adding the closing price of the security for some time periods and then dividing this total by the number of time periods. Its distance from 20-days simple moving average is 18.26%, and its distance from 50 days simple moving average is 43.63% while it has a distance of 57.9% from the 200 days simple moving average. The companys distance from 52-week high price is -6.18% and while the current price is 212.46% from 52-week low price.

As of now, CRISPR Therapeutics AG has a P/S, P/E and P/B values of 18.42, 0 and 6.36 respectively.

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Current Trend:: CRISPR Therapeutics AG, (NASDAQ: CRSP) - Ws News Alerts

DUBLIN, Nov. 28, 2019 /PRNewswire/ -- The "Genome Editing Services Market-Focus on CRISPR 2019-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape of CRISPR-based genome editing service providers. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographical regions.

Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The biopharmaceutical pipeline includes close to 500 gene therapies, several of which are being developed based on the CRISPR technology.

Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. However, successful gene manipulation efforts involve complex experimental protocols and advanced molecular biology centered infrastructure. Therefore, many biopharmaceutical researchers and developers have demonstrated a preference to outsource such operations to capable contract service providers.

Consequently, the genome editing contract services market was established and has grown to become an indispensable segment of the modern healthcare industry, offering a range of services, such as gRNA design and construction, cell line development (involving gene knockout, gene knockin, tagging and others) and transgenic animal model generation (such as knockout mice). Additionally, there are several players focused on developing advanced technology platforms that are intended to improve/augment existing gene editing tools, especially the CRISPR-based genome editing processes.

Given the rising interest in personalized medicine, a number of strategic investors are presently willing to back genetic engineering focused initiatives. Prevalent trends indicate that the market for CRISPR-based genome editing services is likely to grow at a significant pace in the foreseen future.

Report Scope

One of the key objectives of the report was to evaluate the current opportunity and the future potential of CRISPR-based genome editing services market. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2019-2030.

In addition, we have segmented the future opportunity across [A] type of services offered (gRNA construction, cell line engineering and animal model generation), [B] type of cell line used (mammalian, microbial, insect and others) and [C] different geographical regions (North America, Europe, Asia Pacific and rest of the world).

To account for the uncertainties associated with the CRISPR-based genome editing services market and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market's evolution.

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. Overview of Genome Editing3.3. History of Genome Editing3.4. Applications of Genome Editing3.5. Genome Editing Techniques3.5.1. Mutagenesis3.5.2 Conventional Homologous Recombination3.5.3 Single Stranded Oligo DNA Nucleotides Homologous Recombination3.5.4. Homing Endonuclease Systems (Adeno Associated Virus System)3.5.5. Protein-based Nuclease Systems3.5.5.1. Meganucleases3.5.5.2. Zinc Finger Nucleases3.5.5.3. Transcription Activator-like Effector Nucleases3.5.6. DNA Guided Systems3.5.6.1. Peptide Nucleic Acids3.5.6.2. Triplex Forming Oligonucleotides3.5.6.3. Structure Guided Endonucleases3.5.7. RNA Guided Systems3.5.7.1. CRISPR-Cas93.5.7.2. Targetrons3.6. CRISPR-based Genome Editing3.6.1. Role of CRISPR-Cas in Adaptive Immunity in Bacteria3.6.2. Key CRISPR-Cas Systems3.6.3. Components of CRISPR-Cas System3.6.4. Protocol for CRISPR-based Genome Editing3.7. Applications of CRISPR3.7.1. Development of Therapeutic Interventions3.7.2. Augmentation of Artificial Fertilization Techniques3.7.3. Development of Genetically Modified Organisms3.7.4. Production of Biofuels3.7.5. Other Bioengineering Applications3.8. Key Challenges and Future Perspectives

4. CRISPR-BASED GENOME EDITING SERVICE PROVIDERS: CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. CRISPR-based Genome Editing Service Providers: Overall Market Landscape4.2.3. Analysis by Type of Service Offering4.2.4. Analysis by Type of gRNA Format4.2.5. Analysis by Type of Endonuclease4.2.6. Analysis by Type of Cas9 Format4.2.7. Analysis by Type of Cell Line Engineering Offering4.2.8. Analysis by Type of Animal Model Generation Offering4.2.9. Analysis by Availability of CRISPR Libraries4.2.10. Analysis by Year of Establishment4.2.11. Analysis by Company Size4.2.12. Analysis by Geographical Location4.2.13. Logo Landscape: Distribution by Company Size and Location of Headquarters

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. CRISPR-based Genome Editing Service Providers: Competitive Landscape5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. COMPANY PROFILES6.1. Chapter Overview6.2. Applied StemCell6.2.1. Company Overview6.2.2. Service Portfolio6.2.3. Recent Developments and Future Outlook6.3. BioCat6.4. Biotools6.5. Charles River Laboratories6.6. Cobo Scientific6.7. Creative Biogene6.8. Cyagen Biosciences6.9. GeneCopoeia6.10. Horizon Discovery6.11. NemaMetrix6.12. Synbio Technologies6.13. Thermo Fisher Scientific

7. PATENT ANALYSIS7.1. Chapter Overview7.2. Scope and Methodology7.3. CRISPR-based Genome Editing: Patent Analysis7.3.1. Analysis by Application Year and Publication Year7.3.2. Analysis by Geography7.3.3. Analysis by CPC Symbols7.3.4. Emerging Focus Areas7.3.5. Leading Players: Analysis by Number of Patents7.4. CRISPR-based Genome Editing: Patent Benchmarking Analysis7.4.1. Analysis by Patent Characteristics7.5. Patent Valuation Analysis

8. ACADEMIC GRANT ANALYSIS8.1. Chapter Overview8.2. Scope and Methodology8.3. Grants Awarded by the National Institutes of Health for CRISPR-based8.3.1. Year-wise Trend of Grant Award8.3.2. Analysis by Amount Awarded8.3.3. Analysis by Administering Institutes8.3.4. Analysis by Support Period8.3.5. Analysis by Funding Mechanism8.3.6. Analysis by Type of Grant Application8.3.7. Analysis by Grant Activity8.3.8. Analysis by Recipient Organization8.3.9. Regional Distribution of Grant Recipient Organization8.3.10. Prominent Project Leaders: Analysis by Number of Grants8.3.11. Emerging Focus Areas8.3.12. Grant Attractiveness Analysis

9. CASE STUDY: ADVANCED CRISPR-BASED TECHNOLOGIES/SYSTEMS AND TOOLS9.1. Chapter Overview9.2. CRISPR-based Technology Providers9.2.1. Analysis by Year of Establishment and Company Size9.2.2. Analysis by Geographical Location and Company Expertise9.2.3. Analysis by Focus Area9.2.4. Key Technology Providers: Company Snapshots9.2.4.1. APSIS Therapeutics9.2.4.2. Beam Therapeutics9.2.4.3. CRISPR Therapeutics9.2.4.4. Editas Medicine9.2.4.5. Intellia Therapeutics9.2.4.6. Jenthera Therapeutics9.2.4.7. KSQ Therapeutics9.2.4.8. Locus Biosciences9.2.4.9. Refuge Biotechnologies9.2.4.10. Repare Therapeutics9.2.4.11. SNIPR BIOME9.2.5. Key Technology Providers: Summary of Venture Capital Investments9.3. List of CRISPR Kit Providers9.4. List of CRISPR Design Tool Providers

10. POTENTIAL STRATEGIC PARTNERS10.1. Chapter Overview10.2. Scope and Methodology10.3. Potential Strategic Partners for Genome Editing Service Providers10.3.1. Key Industry Partners10.3.1.1. Most Likely Partners10.3.1.2. Likely Partners10.3.1.3. Less Likely Partners10.3.2. Key Non-Industry/Academic Partners10.3.2.1. Most Likely Partners10.3.2.2. Likely Partners10.3.2.3. Less Likely Partners

11. MARKET FORECAST11.1. Chapter Overview11.2. Forecast Methodology and Key Assumptions11.3. Overall CRISPR-based Genome Editing Services Market, 2019-203011.4. CRISPR-based Genome Editing Services Market: Distribution by Regions, 2019-203011.4.1. CRISPR-based Genome Editing Services Market in North America, 2019-203011.4.2. CRISPR-based Genome Editing Services Market in Europe, 2019-203011.4.3. CRISPR-based Genome Editing Services Market in Asia Pacific, 2019-203011.4.4. CRISPR-based Genome Editing Services Market in Rest of the World, 2019-203011.5. CRISPR-based Genome Editing Services Market: Distribution by Type of Services, 2019-203011.5.1. CRISPR-based Genome Editing Services Market for gRNA Construction, 2019-203011.5.2. CRISPR-based Genome Editing Services Market for Cell Line Engineering, 2019-203011.5.3. CRISPR-based Genome Editing Services Market for Animal Model Generation, 2019-203011.6. CRISPR-based Genome Editing Services Market: Distribution by Type of Cell Line, 2019-203011.6.1. CRISPR-based Genome Editing Services Market for Mammalian Cell Lines, 2019-203011.6.2. CRISPR-based Genome Editing Services Market for Microbial Cell Lines, 2019-203011.6.3. CRISPR-based Genome Editing Services Market for Other Cell Lines, 2019-2030

12. SWOT ANALYSIS12.1. Chapter Overview12.2. SWOT Analysis12.2.1. Strengths12.2.2. Weaknesses12.2.3. Opportunities12.2.4. Threats12.2.5. Concluding Remarks

13. EXECUTIVE INSIGHTS

14. APPENDIX 1: TABULATED DATA

15. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

Companies Mentioned

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Genome Editing Services, World Markets to 2030: Focus on CRISPR - The Most Popular Genome Manipulation Technology Tool - PRNewswire

Dec. 2, 2019 10:30 UTC

CAMBRIDGE, Mass. & SEATTLE--(BUSINESS WIRE)-- Cyrus Biotechnology, Inc., and the Broad Institute of MIT and Harvard have embarked on a scientific collaboration to optimize CRISPR for use in developing novel human therapeutics.

CRISPR allows for the highly specific and rapid modification of DNA in a genome, which can dramatically accelerate the drug discovery process.

Feng Zhang will be the principal investigator for the Broad for the collaboration. He is also an investigator of the Howard Hughes Medical Institute (HHMI).

Together, researchers from Cyrus and Broad will work together to mitigate the possibility of the body mounting an immune response against CRISPR. The teams are committed to making the results of their collaboration broadly available for research to help ensure that therapeutic development bringing this technology to the clinic has the best chance of success, while also considering important ethical and safety concerns. The teams have also committed to publishing their results in peer reviewed journals and to make this work freely available to the non-profit and academic scientific community.

Issi Rozen, chief business officer at the Broad Institute, said, Broad researchers and their collaborators have pioneered the development and sharing of new genome editing tools, such as CRISPR-Cas9, which are revolutionizing and accelerating nearly every aspect of disease research and drug discovery around the world. With this collaboration, scientists will continue to improve the technology towards new tools and therapeutics, important to benefiting patients in the long term.

Cyrus CEO Dr. Lucas Nivn added, We have validated our computational deimmunization platform in a variety of systems, and now seek to apply it where it can make a major impact. Given the extensive therapeutic possibilities of CRISPR systems, and the leading position the Broad Institute and Dr. Zhang hold, we are very excited to work in partnership with them to make these molecules more amenable for use in humans with maximal efficacy and minimal side effects.

Cyrus provides commercial and partnered access to Rosetta, which is the worlds leading protein modeling and design software platform. Rosetta has been used to direct the computational design of multiple biologic molecules that have advanced to both pre-clinical and clinical development. Among these are drugs being developed by companies including PVP Biologics, Tocagen, Lyell and others.

About Cyrus Biotechnology

Cyrus Biotechnology, Inc. is a privately-held Seattle-based biotechnology software company offering software and partnerships for protein engineering to accelerate discovery of biologics and small molecules for the Biotechnology, Pharmaceutical, Chemical, Consumer Products and Synthetic Biology industries. Cyrus methods are based on the Rosetta software from Prof. David Bakers laboratory at the University of Washington and HHMI, the most powerful protein engineering software available. Cyrus customers include 13 of the top 20 Global Pharmaceutical firms and is financed by leading investors in both Technology and Biotechnology, including Trinity Ventures, Orbimed, Springrock Ventures, Alexandria Venture Investments, and W Fund.

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Cyrus Biotechnology and the Broad Institute of MIT and Harvard Launch Multi-Target Collaboration to Develop Optimized CRISPR Gene Editing Technology -...

Global CRISPR in Agriculture Market valued approximately USD XX million in 2016 is anticipated tgrow with a healthy growth rate of more than XX% over the forecast period 2017-2025. Increasing demand in drug discovery, late pregnancies leading tbirth disorders, synthetic genes leading the way; aging genetic disorders and investment in path breaking research technology are the drivers for CRISPR Market. Drug discovery technology market plays a dominant role in boosting the CRISPR market. Genome editing has been revolutionized with the discovery of the CRISPR-CAS9 system from streptococcus pyogenes.

Request a Sample Copy of this[emailprotected]https://www.orbisresearch.com/contacts/request-sample/2129000

The objective of the study is tdefine market sizes of different segments & countries in recent years and tforecast the values tthe coming eight years. The report is designed tincorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report alscaters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall alsincorporate available opportunities in micrmarkets for stakeholders tinvest along with the detailed analysis of competitive landscape and product offerings of key players.

The detailed segments and sub-segment of the market are explained below:

By Crop Type:Staple CropsFruits & VegetablesOrnamentalsOthers

By Regions:North AmericaU.S.CanadaEuropeUKGermanyAsia PacificChinaIndiaJapanLatin AmericaBrazilMexicoRest of the World

To make an enquiry on[emailprotected]https://www.orbisresearch.com/contacts/enquiry-before-buying/2129000

Furthermore, years considered for the study are as follows:Historical year 2015Base year 2016Forecast period 2017 t2025

Some of the key manufacturers involved in the market are:

DuPont, Cibus, Monsanto, Bayer AG. Acquisitions and effective mergers are some of the strategies adopted by the key manufacturers. New product launches and continuous technological innovations are the key strategies adopted by the major players.

Browse full[emailprotected]https://www.orbisresearch.com/reports/index/global-crispr-in-agriculture-market-forecasts-2017-2025

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CRISPR in Agriculture Market 2019 by Services, Application, Key Players, Size, Trends and Forecast 2025 - Downey Magazine

Traditional gene therapy has seen numerous challenges during its decades of development, but scientists seem to have finally figured out how to get the treatment to work with regulatory approvals forNovartis' (NYSE:NVS) Zolgensma and bluebird bio's (NASDAQ:BLUE) Zynteglo this year. The process involves inserting genes into diseased cells to express missing or mutated proteins.

Storming onto the scene over the past few years, CRISPR/Cas9, championed by CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT) and Intellia Therapeutics (NASDAQ:NTLA), offered hope for more precise gene editing. At the very least, the process can insert the gene into a precise location in the genome. More impressive -- and something that traditional gene therapy can't readily do -- CRISPR/Cas9 offers the possibility of deleting problematic genes or making specific changes to mutated genes to restore their functions.

Image source: Getty Images.

CRISPR/Cas9 appeared to be working well in preclinical models, and last week, investors got a first look at how the therapy is working in humans with CRISPR Therapeutics and its development Vertex Pharmaceuticals (NASDAQ:VRTX) announcing results for the first two patients treated with CTX001.

One patient with a blood disorder called transfusion-dependent beta thalassemia (TDT) required 16.5 transfusions per year over the two years before being treated with CTX001, but nine months after treatment, the patient was transfusion independent with high expression of fetal hemoglobin, the gene inserted into the patients' cells.

The other patient had sickle cell disease (SCD) with an average of seven vaso-occlusive crises (VOCs) per year over the two years before the study started. Four months after being treated with CTX001, the patient was free of VOCs, which are caused by sickle-shaped red blood cells that block blood vessels. Like the beta thalassemia patient, the SCD patient had expression of fetal hemoglobin.

The results from the first two patients look comparable to Bluebird's Zynteglo, which also treats TDT and SCD by increasing hemoglobin levels. But this was data from just two patients, and investors should still have plenty of questions as we get additional data:

Consistency: One patient in each disease doesn't say much about how well the treatment works in the average patient. What will the efficacy look like after the treatment of a few dozen patients?

Durability: Gene editing and gene therapy are designed to be cures. Do both last forever?

Manufacturing: Bluebird had to adjust its manufacturing procedure to increase expression to treat patients requiring higher expression. Will the initial CRISPR/Cas9 manufacturing procedure work for all patients?

In vivo/ex vivo: That's Latin for in or outside of a living thing -- in this case a human being. CTX001 and Zynteglo are ex vivo treatments because cells are taken from the patient, manipulated to express the gene of interest, and put back into the patient. Novartis has shown that gene therapy can work in vivo with Zolgensma delivered via an injection of a viral vector. Can CRISPR/Cas9 work in vivo in humans? Editas Medicine hopes so, but the company still hasn't advanced a treatment into the clinic.

Last week's data release offers plenty of hope for investors in CRISPR/Cas9 and traditional gene therapy companies should certainly be looking in the rearview mirror at the technology coming up from behind, but it's still way too early to pick a winner between traditional gene therapy and CRISPR/Cas9.

The right answer for investors in biotech companies might end up being to buy both. The upside potential for curing diseases may end up outweighing the downside if one technology doesn't end up working out.

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CRISPR vs. Gene Therapy Round 1: What Investors Need to Know - The Motley Fool

CRISPR is a tool used by researchers to precisely edit genes and has shown potential for treating genetic diseases. This article delves into some recent developments and explores what the future holds for CRISPR.

CRISPR genome editing is a promising field that enables researchers to precisely delete, replace or edit genes.

CRISPR-Cas is a prokaryotic defence system whereby bacteria use RNA molecules and CRISPR-associated (Cas) proteins to target and destroy the DNA of invading viruses. This molecular machinery has been repurposed by researchers to target and edit specific sections of any DNA, whether bacterial or human.

Despite the success of CRISPR, the technique is far from refined. In certain situations, the editing process can result in off-target DNA being changed, causing unwanted effects. Also, CRISPR-Cas9 is a large molecular complex, with both the Cas9 nuclease and an engineered single-guide RNA (sgRNA) that helps the nuclease locate its target. This can make its delivery into the nucleus of the cell, where CRISPR needs to access DNA, difficult.

Consequently, many researchers have sought improvements to CRISPR with the gene editing method expected to continue development well into the future.

Here, three researcher groups who have contributed to recent CRISPR developments explain their work and predict how CRISPR may evolve.

In an attempt to multiplex CRISPR systems to target lots of genes, researchers at ETH Zurich in Switzerland swapped the Cas9 enzyme for Cas12a. Using this plasmid allowed the researchers to simultaneously edit genes in 25 target sites. The team predicts that dozens or even hundreds more sites could be modified using this method.

Genes and proteins in cells interact in many different ways. Each dot represents a gene; the lines are their interactions. For the first time, the new method uses biotechnology to influence entire gene networks in one single step (credit: ETH Zurich/Carlo Cosimo Campa).

Cas12a enabled the researchers to attach shorter sgRNA address molecules than when using Cas9. The shorter length molecules mean that more can fit onto the plasmid, which is a circular DNA molecule that acts as the blueprint of the Cas enzyme, thus enabling CRISPR to edit many genes in a short space of time.

Professor Randall Platt, who led the research, explained that his teams technique is conditional, inducible and orthogonal.

This development offers an improvement on traditional CRISPR technology, which only enables one gene to be edited at a time. This technique therefore speeds the process up, allowing CRISPR to edit many genes simultaneously. It also means that the expression of some genes can upregulated while others can be downregulated.

Platt says that their technique is drastically better, at targeting multiple genes and it afforded the researchers sophisticated control over cellular genomes and transcriptomes.

Another development for CRISPR technologies came from researchers at Duke University in the US. The team successfully used Class 1 CRISPR systems for the first time to edit the epigenome of human cells. Conventional CRISPR-Cas9 methods are categorised as Class 2 systems.

The Class 1 technique makes use of multiple proteins in a process called CRISPR-associated complex for antiviral defence (Cascade). This complex binds with high accuracy to the correct sites. After binding, Cascade utilises a Cas3 protein to target and edit the DNA. They were also able to both activate and repress target gene expression.

Illustrations representing the components of the common dCas9 system (top) and the Cascade system (bottom) (credit: Gersbach Lab).

The team says that this research contributes to an enhancement of CRISPR technologies as it provides a potential alternative for CRISPR-Cas9 when there are complications such as immune responses to Cas proteins. It can also recruit various modifiers of gene regulation, including activators and repressors, to a gene.

Associate ProfessorCharles Gersbach, one of the lead researchers, says that the team will continue to explore CRISPR biology and how the Class 1 method can be developed for gene editing.

It will be exciting to explore other types of effector domains, such as modifiers of DNA methylation, base editors, etc, attached toCascade, Gersbach says.

A further CRISPR development has come from a collaboration between Tufts University in the US and the Chinese Academy of Sciences. These researchers used a biodegradable synthetic lipid nanoparticle to deliver their CRISPR editing tools into the cell to precisely alter the cells genetic code.

According to the team, their method resulted in up to 90 percent efficacy in gene editing. The lipid nanoparticles encapsulate messenger RNA (mRNA) encoding Cas9. Once the contents of the nanoparticles including the sgRNA are released into the cell, the cells protein-making machinery takes over and creates Cas9 from the mRNA template.

A unique feature of the nanoparticles is made of synthetic lipids comprising disulfide bonds in the fatty chain. When the particles enter the cell, the environment within the cell breaks open the disulfide bond to disassemble the nanoparticles and the contents are quickly and efficiently released into the cell.

Once the contents of the nanoparticles are released into the cell, the cells protein-making machinery takes over and creates Cas9

The researchers highlighted that their delivery system refines CRISPR technologies; as Cas9 is a large complex it is difficult to deposit directly into the nucleus of the cell. Other research teams have used viruses, polymers and other kinds of nanoparticles to deliver CRISPR-Cas9, but the low efficiency of transfer limits its success. As their delivery system builds the Cas9 enzyme later in the process, it has high levels of transfer and efficacy.

Professor Qiaobing Xu, a co-corresponding author of the study, highlighted that the synthetic lipid could be made with low-toxicity. Furthermore, he explained, as there is no limit in terms of cargo size, the lipid is an improvement upon viral delivery.

He also emphasised that with viral delivery, there is always a concern about the immune response against the viral particle, but a non-viral delivery method does not have this disadvantage.

These developments in the CRISPR technique indicate how the technology is set to improve and develop in the future. However, research is far from over.

Platt believes that CRISPR processes are still in their infancy, as the current tools are effective at cutting DNA but can result in random repair. He believes that the future of genome editing is going to require new tools to enable more precise changes to the genome.

Eliminating random output would ensure success of the technology for therapeutic effect. Making precise changes is therefore the direction that CRISPR will evolve to, allowing more complex challenges to be tackled.

Gersbach remarks that his teams study will likely stimulate more research into Class 1 systems, which could lead to numerous applications and provide more biological insights into its potential therapeutic use.

Although there is more work to be done with regard to Class 1 CRISPR systems, its unique attributes make it worth investigating, he says.

Xu also comments that CRISPR is a young field compared with other technologies. He highlights the many areas of CRISPR developments: better editors; larger animal or in vitro models; and more precise analytical methods to detect gene editing.

He believes that CRISPR holds tremendous potential to treat disease, which is absolutely ground-breaking. If specific, targeted genes in the body can be controlled, then almost every condition could potentially be treated.

In conclusion, CRISPR can be a highly useful tool for editing genes and to potentially treat complex diseases. However, it still must be refined as a technique. This has caused researchers to strive for improvements in this area, to make the process more precise and effective.

These recent studies demonstrate that improvements are possible and serve to highlight the enormous potential that CRISPR offers.

According to the researchers, CRISPR technologies have progressed and will continue to improve. They all agree that CRISPR could one day be an effective way to treat genetic diseases.

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How will CRISPR change and evolve in the future? - Drug Target Review

"Paired nickases represent a significant step in increasing specificity through a highly flexible and efficient approach to reduce off target effects in gene editing," said Udit Batra, CEO, MilliporeSigma. "MilliporeSigma's technology improves CRISPR's ability to fix diseased genes while not affecting healthy ones, therefore improving the accuracy of potential gene therapy treatments."

These patents cover a foundational CRISPR strategy in which two CRISPR nickases are targeted to a common gene target and work together by nicking or cleaving opposite strands of a chromosomal sequence to create a double-stranded break. This process can optionally include an exogenous or donor sequence for insertion in the same manner as MilliporeSigma's patented CRISPR integration technology. The requirement of two CRISPR binding events greatly reduces the chances of off-target cutting at other locations in the genome.

In addition to Japan and Singapore, MilliporeSigma has CRISPR-related patents in the following regions: Australia, Canada, China, Europe, Israel, South Korea and the U.S. MilliporeSigma was awarded its first foundational patent in Australia covering CRISPR integration in 2017, and its first U.S. CRISPR patent for proxy-CRISPR in 2019.

MilliporeSigma has been at the forefront of innovation in the field for 15 years, with experience spanning from discovery to manufacturing.MilliporeSigma supports research with genome editing under careful consideration of ethical and legal standards. MilliporeSigma's parent company, Merck KGaA, Darmstadt, Germany, established an independent, external Bioethics Advisory Panelto provide guidance for research in which its businesses are involved, including research on or using genome editing. The company has also defined a clear operational position considering scientific and societal issues to inform promising therapeutic approaches for use in research and applications.

Follow MilliporeSigma on Twitter @MilliporeSigma, on Facebook @MilliporeSigma and on LinkedIn.

All Merck KGaA, Darmstadt, Germany news releases are distributed by email at the same time they become available on the EMD Group website. In case you are a resident of the U.S. or Canada please go to http://www.emdgroup.com/subscribe to register again for your online subscription of this service as our newly introduced geo-targeting requires new links in the email. You may later change your selection or discontinue this service.

About the Life Science Business of Merck KGaA, Darmstadt, GermanyThe Life Science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has some 21,000 employees and 59 manufacturing sites worldwide, with a portfolio of more than 300,000 products focused on scientific discovery, biomanufacturing and testing services. Udit Batra is the global chief executive officer of MilliporeSigma.

Merck KGaA, Darmstadt, Germany completed its $17 billion acquisition of Sigma-Aldrich in November 2015, creating a leader in the $125 billion global life science industry.

Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and performance materials. Around 56,000 employees work to make a positive difference to millions of people's lives every day by creating more joyful and sustainable ways to live. From advancing gene-editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices the company is everywhere. In 2018, Merck KGaA, Darmstadt, Germany generated sales of 14.8 billion in 66 countries.

The company holds the global rights to the name and trademark "Merck" internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Performance Materials. Since its founding 1668, scientific exploration and responsible entrepreneurship have been key to the company's technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. For more information about Merck, KGaA, Darmstadt, Germany, visit http://www.emdgroup.com.

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Japan and Singapore Grant CRISPR Patents to MilliporeSigma - PRNewswire

GlobalCRISPR and Cas Genes MarketResearch Report represents an extensive analysis of global CRISPR and Cas Genes industry by delivering evaluation of present forthcoming trends, competitive forces, customers expectations, technological advancements, and working capital in the market. The report also renders a thorough analysis of geographical regions and circumstances, product/service types, Key applications, consumption, revenue, and sales of CRISPR and Cas Genes.

The Research report Delivers a summary of the impact of the key drivers, restraints, and popular trends in the CRISPR and Cas Genes market. [To Know More -Request Sample Report] These factors are studied on regional as well as the global front, for varying levels of depth of market research. Overall review of the factors affecting various decisions in the global market is presented and examined by policies in the market, regulatory scenario of the market, with the help of details of key principles, directions, plans, and strategies in the market. The report includes the detailed analytical account of the markets competitive landscape, with the help of detailed business profiles, SWOT analysis, project feasibility analysis, and several other details about the key companies operating in the CRISPR and Cas Genes market. The report also presents an outline of the impact of recent developments on the markets future growth forecast.

For Better Understanding, Download Free Sample PDF Brochure of CRISPR and Cas Genes Market Research Report @https://marketresearch.biz/report/crispr-and-cas-genes-market/request-sample

Top Companies in Worldwide CRISPR and Cas Genes Market are as follows:-

Addgene Inc, AstraZeneca Plc., Bio-Rad Laboratories Inc, Caribou Biosciences Inc, Cellectis S.A., Cibus Global Ltd, CRISPR Therapeutics AG, Editas Medicine Inc, eGenesis Bio, GE Healthcare, GenScript Corporation And More

Global CRISPR and Cas Genes Market: Segmentation Analysis

Segmentation on the basis of product:

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

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

Biotechnology & Pharmaceutical CompaniesAcademic & Government Research InstitutesContract Research Organizations

Global CRISPR and Cas Genes Market: Regional Analysis

North America

Europe

Asia Pacific

Latin America

Middle East & Africa

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Key questions answered in the CRISPR and Cas Genes Market report:

What are the key market trends impacting the growth of the CRISPR and Cas Genes market?

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Who are the global key manufacturers of CRISPR and Cas Genes Industry: Company Introduction, and Major Types, Sales Market Performance, Product Specification, Contact Information, Production Market Performance.

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What are the upstream raw materials and manufacturing equipment of CRISPR and Cas Genes? UpStream Industries Analysis, Equipment, and Suppliers, Raw Material and Suppliers, Manufacturing Analysis, Manufacturing Plants Distribution Analysis, Manufacturing Cost Structure, Manufacturing Process, Industry Chain Structure Analysis.

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New Research: CRISPR and Cas Genes Market Trends And Top Key Companies Profile || [Addgene Inc, AstraZeneca Plc., Bio-Rad Laboratories Inc] - Industry...

MarketResearchNest.com adds Global CRISPR Market Report 2019 Market Size, Share, Price, Trend and Forecast new report to its research database. The report spread across 102 with table and figures in it.

The global market size of CRISPR is $- million in 2018 with CAGR from 2014 to 2018, and it is expected to reach $- million by the end of 2024 with a CAGR of -% from 2019 to 2024.

Global CRISPR Market Report 2019 Market Size, Share, Price, Trend and Forecast is a professional and in-depth study on the current state of the global CRISPR industry.

This report studies the CRISPR Market with many aspects of the industry like the market size, market status, market trends and forecast, the report also provides brief information of the competitors and the specific growth opportunities with key market drivers. Find the complete CRISPR market analysis segmented by companies, region, type and applications in the report.

Request a sample copy @

https://www.marketresearchnest.com/report/requestsample/770598

The key insights of the report:

There are 4 key segments covered in this report: competitor segment, product type segment, end use/application segment and geography segment.

For competitor segment, the report includes global key players of CRISPR as well as some small players.

At least 9 companies are included:

For complete companies list, please ask for sample pages.

The information for each competitor includes:

For product type segment, this report listed main product type of CRISPR market

For end use/application segment, this report focuses on the status and outlook for key applications. End users are also listed.

Browse full table of contents and data tables @

https://www.marketresearchnest.com/Global-CRISPR-Market-Report-2019Market-Size-Share-Price-Trend-and-Forecast.html

For geography segment, regional supply, application-wise and type-wise demand, major players, price is presented from 2013 to 2023. This report covers following regions:

The key countries in each region are taken into consideration as well, such as United States, China, Japan, India, Korea, ASEAN, Germany, France, UK, Italy, Spain, CIS, and Brazil etc.

Highlights of the Global CRISPR report:

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We also can offer customized report to fulfill special requirements of our clients. Regional and Countries report can be provided as well.

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Growth of CRISPR Market in Global Industry: Overview, Size and Share 2019-2024 - Markets Gazette 24

Rendered Cas9

Results from clinical trials released Tuesday indicate that two patients, one with beta thalassemia and one with sickle cell disease, have potentially been cured of their diseases. The two trials, which involved using Crispr to edit the genes of the patients in question, were jointly conducted by Vertex Pharmaceuticals and CRISPR Therapeutics.

This is the first clinical evidence to demonstrate that Crispr/Cas9 can be used to cure or potentially cure serious genetic illnesses, Jeffery Leiden, CEO of Vertex, told Forbes. It's a remarkable scientific and medical milestone.

Vertex Pharmaceuticals CEO Jeffery Leiden

Crispr/Cas9 is a gene-editing system popular for its ability to snip, repair or insert genes into DNA. The therapies tested in the clinical trials work by extracting bone marrow stem cells from the patients, editing these stem cells to fix the genetic mutations that cause the diseases, and then infusing the cells back into the patients. The patients body then takes over and is able to produce new, healthy cells. Engineering of the cells is done ex vivo (outside of the patients body). This allows the researchers to make sure the correct changes are made and there are no improper edits to the genome.

CTX001, the gene-editing therapy used in these trials, is very surgical in how it makes the change, says David Altshuler, Vertexs chief scientific officer.

It has been nine months since the patient with beta thalassemia received the one-time-only treatment and over four months for the patient with sickle cell disease. In that time, both of their conditions have improved tremendously, Leiden says. The patient with beta thalassemia, who used to undergo more than 16 blood transfusions each year, hasnt needed an infusion since the treatment. The patient with sickle cell disease experienced an average ofseven excruciating health crises per year before the treatment, and since the treatment hasnt experienced any.

Despite the fact that these results have only been seen in two patients, says Samarth Kulkarni, CEO of CRISPR Therapeutics, the effect is so dramatic in these patients that we cant help but think this brings a lot of promise.

CRISPR Therapeutics CEO Sam Kulkarni

Both patients suffered side effects during the treatment, but doctors concluded they were caused by the bone marrow preparation, not the Crispr treatment itself. In order to infuse healthy stem cells, both patients had to undergo intensive chemotherapy to destroy their old bone marrow cells. This treatment, also common for bone cancer patients, can cause nausea, hair loss and organ damage.

Precision medicine is known for its hefty price tag, and this treatment is the zenith of precision medicine, Kulkarni says. Yet when asked about potential cost of the treatment, Kulkarni says that they are still focusing on clinical development and it is too early to contemplate any sort of pricing discussions." Zolgensma, the first FDA approved gene-therapy medication, was priced at $2.1 million last May.

The applications of Crispr seem limitless, but the field has encountered several ethical controversies. Last year, Chinese scientist He Jiankui shocked the medical community by announcing that he had altered the genes of two human children. One of the main worries that researchers have about Crispr is that scientists might alter genes to be inherited, a practice called germline engineering. In a recent article on the anniversary of Hes revelation, Crispr pioneer Jennifer Doudna called for stricter regulations for using Crispr in heritable human genome editing.

But germline editing isnt a concern in these trials, where only somatic, or non-reproductive cells, were altered. People are much more concerned about intentional changes to a persons DNA that could be passed down to their descendants, says Henry Greely, a Stanford law professor and chairman of the California Advisory Committee on Human Stem Cell Research. When it comes to somatic cells, they die with the person," he says.

In addition to following these initial patients for the next two years to see if their diseases reoccur, Leiden says theyre enrolling multiple patients with both diseases for the next phase of the clinical trial and will be starting treatments for those patients in the near future. While they dont yet have a timeline on when the treatment will be commercially available, we want to get this to patients as soon as possible, he says.

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New Data From First Human Crispr Trials Shows Promising Results - Forbes

A University of California professor and co-originator of genome editing technology Clustered Regularly Interspaced Short Palindromic Repeats said researchers plan to expand the technology in order to increase human applications at a campus lecture series Thursday.

Jennifer Doudna, a UC Berkeley biochemistry professor, engaged students and the greater UCLA science community during the quarterly Donald J. Cram Distinguished Lecture series.

The Cram lecture series, a quarterly departmental event, invites prominent academics in the field of chemistry to speak about their research. The series is dedicated to Donald J. Cram, who was a Nobel laureate and a chemistry professor at UCLA for over 50 years.

This fall, the series was hosted by UCLA chemistry professor and Cram Chair Patrick Harran.

Scientists use CRISPR technology, formally known as CRISPR-Cas9, to modify DNA sequences and gene functions. Cas9 is a protein that can cut the strands of DNA-like molecular scissors.

CRISPR is studied and used by students, scientists and researchers to advance progress in the field of gene editing, in medicine and the life sciences.

The UC holds the largest CRISPR patent portfolio in the nation with 16 total patents, according to a UC Berkeley press release.

The United States Patent and Trademark Office granted the UC, along with the University of Vienna and Emmanuelle Charpentier, the director of the Max Planck Institute for Infection Biology, its 16th patent in October.

Doudnas involvement in CRISPR technology began around 2005, when a professor at UC Berkeley, Jill Banfield, invited Doudna to help her with research into the mechanism. From there, Doudna teamed up with Charpentier, who was working with a CRISPR system and its associated protein, Cas9, in 2011.

Doudna is one of the creators of the CRISPR utility for the permanent excision of harmful genes. Doudna said that she developed the idea for the CRISPR technology in 2011 in collaboration with Charpentier.

During the lecture, Doudna detailed how scientists regulate CRISPR enzymes to modify DNA.

CRISPR is a portion of the bacterial genomic sequence that acts as an adaptive immune system, Doudna said.

Bacteria encode the CRISPR system through viral infections, which allows its genome to recognize foreign DNA insertions. These DNA sequences incorporate themselves into the bacterial genome at the CRISPR locus, a genetic database of past infections.

Doudna said this locus was of unique interest to her.

Those sequences, called CRISPR, are transcribed in RNA molecules that provide the zip codes for Cas proteins, allowing them to recognize foreign DNA and cut it up, Doudna said.

Doudna and Charpentier, with the assistance of their team, were able to realize that CRISPR RNA is a 20-nucleotide sequence, which interacts with DNA in a complementary fashion.

This complementarity allows the protein to form a double-stranded break in DNA, necessitating a second RNA tracrRNA to form this functional unit, Doudna said.

And it was (biochemist) Martin Jinek in our lab who figured out that you could combine these two RNAs into a single guide RNA, Doudna said.

From this experiment, Jinek found that single guide RNAs were used by Cas9 to excise DNA at specific sites in a plasmid, a circular piece of bacterial DNA. The revelation from this was that, upon excision, DNA would repair itself in animals and plants, Doudna said.

Doudna said at the end of her talk that the system is becoming increasingly important in the field of medicine, and is currently being used at UCLA, by Donald Kohn, a professor of microbiology, immunology and molecular genetics.

Were within about five years, maybe less, from being able to make, essentially, any change to any genome in any type of cell, Doudna said.

Doudna stressed that this ability to make changes in the genome comes with bioethical responsibility for genome editing in humans.

Fourth-year biochemistry student Jeremy Shek, who attended the event, said although he had done a project that was an offshoot of CRISPR, he had not heard of the progress Doudna discussed.

It is important to be informed on advancements and progress in the field, he added.

Fourth-year bioengineering student Timothy Yu said he came to the lecture to see Doudna in person and get a more solid grasp on the methodology of CRISPR.

Lexi Omholt, a fourth-year microbiology, immunology and molecular genetics student, said that she came to the talk to understand the basis of CRISPR technology.

Jennifer Doudna was one of the reasons I chose my major, Omholt said. At that time, CRISPR came into popular knowledge, and the knockout tool was just coming into use. I am involved in a cancer lab, the Soragni Lab, that uses CRISPR-Cas9 on a regular basis.

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Co-creator of CRISPR lectures about future applications of genome editing technology - Daily Bruin

18 November 2019

Researchers claim thatCRISPR could be used to detect dangerous viruses in a drop of blood within an hour.

The team at Case Western Reserve University in Cleveland, Ohio, harnessed CRISPR's precision to identify and quantify viruses in human samples, including serum from blood. Their technique, named E-CRISPR, could offer a robust, accurate and cost-effective way to enable faster diagnoses of infections such as parvovirus which can cause miscarriages and human papillomavirus (HPV) which is associated with some cancers.

'This could someday become a simple, accurate and cost-effective point-of-care device for identifying different nucleic acid viruses, such as HPV or parvo from a single droplet of a blood sample,' said first author Yifan Dai, a PhD candidate at Case Western. 'And it would also be extremely fast.'

HPV and parvovirus are both DNA viruses, and their genomic material will be present in the blood of an infected person. E-CRISPR uses a CRISPR RNA strand to bind target sequences which are unique to the virus, and when attached, the associated Cas12a (also known as Cpf1) enzyme cuts the viral DNA in the same way as it would in CRISPR/Cas9 genome editing.

However, unlike Cas9, Cas12a is known to indiscriminately cut single-stranded DNA (ssDNA) once activated by the double-stranded target in this case the viral sequence. The researchers usedssDNA strands, tethered at one end to a sensor and with an electrochemical tag molecule at the other to detect if this cut has been made. If Cas12a is activated, these ssDNA strands are cut, detaching the tag molecules and the electrochemical current detectable through the sensor drops, giving an observable result.

'The CRISPR technique works so that it cuts all of the non-specified single-strand DNA around it once the target is recognised, so we program to electrochemically probe this activity,' said Dai. 'No virus no cutting, it's that simple. And the opposite is true: If CRISPR starts to cut, we know the virus is present.'

The researchers hope that this method could be further developed to create a new 'universal biosensing' device able to accurately detect viruses quickly at the point-of-care, similar to existing blood-glucose sensors. Currently, systems that detect HPV and parvovirus take days to process.

The study was published in Angewandte Chemie, a journal of the German Chemical Society.

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E-CRISPR could be used to rapidly detect viruses - BioNews

-Two patients treated with CTX001 successfully engrafted and demonstrated an initial safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant-

-Beta thalassemia: Patient is transfusion independent with total hemoglobin level of 11.9 g/dL and 10.1 g/dL fetal hemoglobin at nine months after CTX001 infusion-

-Sickle cell disease: Patient is free of vaso-occlusive crises with total hemoglobin level of 11.3 g/dL and 46.6% fetal hemoglobin at four months after CTX001 infusion-

-CRISPR Therapeutics will host a conference call today at 8:00 a.m. ET to review these data-

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 19, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. One patient with transfusion-dependent beta thalassemia (TDT) received CTX001 in the first quarter of 2019 and data for this patient reflect nine months of safety and efficacy follow-up. One patient with severe sickle cell disease (SCD) received CTX001 in mid-2019 and data for this patient reflect four months of safety and efficacy follow-up. These studies are ongoing and patients will be followed for approximately two years following infusion. Several additional patients have been enrolled and have had drug product manufactured across the two studies.

Transfusion-Dependent Beta Thalassemia The patient with TDT has the 0/IVS-I-110 genotype and required 16.5 transfusions per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after 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. At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells (erythrocytes expressing fetal hemoglobin).

Sickle Cell Disease The patient with SCD experienced seven vaso-occlusive crises (VOCs) per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days 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 of which resolved. At four months after CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells (erythrocytes expressing fetal hemoglobin).

We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.

The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia, said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. While the data are exciting, we are still in the early phase of this clinical program. We look forward to continuing to work with physicians, patients, caregivers and families over the coming months and years to bring forward the best possible therapy for these two serious diseases and to continue to accelerate our gene-editing programs for other serious diseases such as Duchenne muscular dystrophy and myotonic dystrophy type 1.

About the Phase 1/2 Study in Transfusion-Dependent Beta ThalassemiaThe 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 study 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 study. Enrollment is ongoing at six clinical trial sites in the United States, Canada and Europe.

About the Phase 1/2 Study in Sickle Cell DiseaseThe 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 study 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 study. Enrollment is ongoing at 12 clinical trial sites in the United States, Canada and Europe.

About the Gene-Editing Process in These TrialsPatients who enroll in these studies will have 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.

CRISPR Therapeutics Conference Call and WebcastCRISPR Therapeutics will host a conference call and webcast today at8:00 a.m. ET. The webcast and presentation will be made available on the CRISPR Therapeutics website at https://crisprtx.gcs-web.com/events in the Investors section under Events and Presentations. Following the live audio webcast, a replay will be available on the Company's website for approximately 30 days.

Dial-In InformationLive (U.S. / Canada): (800) 895-3361Live (International): (785) 424-1062Conference ID: 87198237

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 and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

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 AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics CTX001 clinical program; (ii) the status and scope of ongoing and potential future clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development byCRISPR Therapeuticsand its collaborators; (iii) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; v(iv) the intellectual property coverage and positions ofCRISPR Therapeutics, its licensors and third parties; (v) the sufficiency of CRISPR Therapeutics cash resources; and (vi) 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. AlthoughCRISPR Therapeuticsbelieves that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others:the potential for initial and preliminary data from any clinical trial (including CTX001) not to be indicative of final trial results; the risk that the initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of results from the full planned study population;the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and 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 byCRISPR Therapeuticswith theU.S. Securities and Exchange Commission, which are available on theSEC'swebsite atwww.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 Therapeuticsdisclaims 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 four 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 disease. 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.

(VRTX-GEN)

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, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program. 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 factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include that the development of CTX001 may not proceed due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with theSecurities and Exchange Commissionand available through the company's website atwww.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

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

CRISPR Therapeutics Media Contact:Jennifer PaganelliWCG on behalf of CRISPR+1 347-658-8290jpaganelli@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orLeah Gibson, +1 617-961-1507

Media: mediainfo@vrtx.com orNorth America:Heather Nichols, +1 617-341-6992Heather_Nichols@vrtx.com

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CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9...

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The S&P 500 and the Dow Jones indexes retreated from record levels on Tuesday as dour forecasts from Home Depot and Kohl's eroded confidence that the U.S. consumer will support the economy. .N

At 12:23 p.m. ET, the Dow Jones Industrial Average .DJI was down 0.28% at 27,957.31. The S&P 500 .SPX was unchanged at 3,121.96 and the Nasdaq Composite .IXIC was up 0.34% at 8,578.962. The top three S&P 500 .PG.INX percentage gainers: ** Broadcom Inc AVGO.O, up 3 % ** Constellation Brands Inc STZ.N, up 2.5 % ** Biogen Inc BIIB.O, up 2.3 % The top three S&P 500 .PL.INX percentage losers: ** Kohl's Corp KSS.N, down 18.1 % ** Macy's Inc M.N, down 9.6 % ** Home Depot Inc HD.N, down 5.2 % The top three NYSE .PG.N percentage gainers: ** Myovant Sciences Ltd MYOV.N, up 84.8 % ** JinkoSolar Holding Co Ltd JKS.N, up 13 % ** 58.Com Inc WUBA.N, up 12.8 % The top three NYSE .PL.N percentage losers: ** Intelsat SA I.N, down 21.8 % ** Kohl's Corp KSS.N, down 18.1 % ** AeroCentury Corp ACY.N, down 12.9 % The top three Nasdaq .PG.O percentage gainers: ** SAExploration SAEX.O, up 78.5 % ** Karuna Therapeutics Inc KRTX.O, up 39.2 % ** Athenex Inc ATNX.O, up 33.2 % The top three Nasdaq .PL.O percentage losers: ** KLX Energy Services Holdings Inc KLXE.O, down 25.3 % ** Pioneer Power Solutions Inc PPSI.O, down 22.3 % ** My Size Inc MYSZ.O, down 19.8 % ** Home Depot Inc HD.N: down 5.2% ** Kohl's Corp KSS.N: down 18.1% ** Macy's Inc M.N: down 9.6% ** Nordstrom Inc JWN.N: down 5.2% ** Lowe's Companies Inc LOW.N: down 0.8% BUZZ-Home Depot: Falls after FY sales forecast cut, drags Lowe's BUZZ-Kohl's: Falls on FY profit forecast cut, Q3 same-store sales miss BUZZ-Retail stocks tumble on Home Depot, Kohl's dour forecasts

** CRISPR Therapeutics AG CRSP.O: up 17.9% ** Vertex Pharmaceuticals Inc VRTX.O: up 2.0% ** Editas Medicine Inc EDIT.O: up 11.2% ** Intellia Therapeutics Inc NTLA.O: up 12.3% ** Sangamo Therapeutics Inc SGMO.O: up 4.8%

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BUZZ-U.S. STOCKS ON THE MOVE-Retail stocks, CRISPR Therapeutics, Slack Technologies - Nasdaq

Imagine if you had a technology that could give you superpowers. No, we are not talking about gamma rays or radioactive spiders but something pretty similar. If youve watched Marvels Luke Cage series, you may have heard of a gene-editing tool called CRISPR. The protagonist gets super strength and unbreakable skin when a mollusc DNA was fused into his DNA.

As bizarre as it may sound, CRISPR-Cas9 (the shorter and more colloquial name is CRISPR) is a real thing and it can selectively cut and paste DNA pieces from and to your DNA. Though the plan for this tool does not include creating super soldiers. Instead, scientists believe that CRISPR-Cas9 could be a ray of hope for those suffering from inherited genetic disorders like down syndrome, cystic fibrosis and thalassemia.

Representational image. Image source: Getty Images.

The technology isnt just theoretical anymore. A study, to be presented next month at a meeting of the American Society of Hematology, conducted on three cancer patients to test the safety of the tool has shown promising results. And it isnt the only one - more studies are recruiting and being conducted to bring gene editing to life.

CRISPR (pronounced Crisper) is an acronym for Clusters of Regularly Interspaced Short Palindromic Repeats. The Cas9 stands for a CRISPR-associated protein 9.

Nature has quipped bacteria with an ability to protect itself from invading viruses. When a virus attacks a bacterium, viral DNA gets embedded in the bacterium's DNA. This creates a new element within the bacterial DNA called CRISPR. If the same virus then attacks the bacterium again, it uses CRISPR to identify the virus.

CRISPR does this by creating RNA - a molecule that carries messages out from the DNA to the rest of the cell. The RNA is the one that identifies the viral DNA invading the cell (or bacteria).

Then, the Cas9 present in the bacteria chops off the invading virus' DNA, thus neutralising the threat.

The new gene-editing tool for humans is based on this same concept. This innovative tool was, rather unimaginatively, then named CRISPR-Cas9.

The gene-editing tool CRISPR-Cas9 is a bit different from its bacterial version. It contains a small chain of RNA along with Cas9, instead of DNA. This cuts out one step of the bacterial process.

CRISPR-Cas9 could be really helpful for those suffering from genetic diseases. A genetic disease happens due to glitches in the DNA sequence. They usually begin inside the fetus when all your body cells are still dividing. Every time a cell divides, its DNA divides along with it to send a copy of the genome into the new cell. But, a fault can sometimes happen in the copying process. This can alter the complete genetic code, creating a mutation - which leads to genetic disease.

This is where CRISPR-Cas9 comes in. The RNA in the tool identifies the "error" in the genetic code. It then binds with the faulty part of the DNA. Cas9 then cuts off that faulty piece.

The original version of the CRISPR-Cas9 tool would only find and cut out the error in the DNA. This creates a gap in the DNA code, which needs to be 'repaired'. The original tool couldn't do so, and would thus necessarily leave the repair process up to the cell. The cell would either join the broken ends of the DNA or insert a new piece to fill the void. Clearly, while this gets rid of the anomaly in the cell, what happens to the genetic code afterwards wasn't in the tool's control. This made the outcome uncertain.

With the new version of the CRISPR-Cas9 tool, scientists can now take control of the repair process as well. We can now add an entirely new DNA sequence of our choice in the removed region and not just fix an anomaly but even introduce new traits into the organism. Think of this as a find-replace tool in your word editor, as opposed to just the find(-delete) tool.

Currently, there is no cure for genetic diseases. CRISPR-Cas9 is a promising step forward. Scientists have already used it to treat some diseases in plants and animals.

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. For more information, please read our article onHemophilia, a genetic condition which prevents blood from clotting.

Updated Date: Nov 15, 2019 14:21:41 IST

Tags : CRISPR, CRISPR-cas9, Gene Slicing, Gene-Editing, Genetic Diseases, Genetic Disorders, NewsTracker

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Another step closer to curing cancer and genetic diseases: All you need to know about CRISPR-Cas9 - Firstpost

Researchers strive to address societal health issues through gene editing

In October, three researchers at UC Davis were awarded a $1.5 million grant to fund their project which attempts to demonstrate the effectiveness of gene editing through use of CRISPR, a powerful technology that allows alteration of DNA sequences to change gene function.

This kind of design can help enhance personalized medicine, said R. Holland Cheng, a professor of molecular and cellular biology in the College of Biological Sciences. Specific patients with specific illnesses can be treated in specific ways.

Cheng, along with Kit Lam, a distinguished professor and chair of the Department of Biochemistry and Molecular Medicine in the School of Medicine, and David Segal, a professor in the Department of Biochemistry and Molecular Medicine, were awarded this highly competitive and sought-after grant from the National Institute of Health (NIH).

UC Davis is part of the NIHs Somatic Cell Genome Editing (SCGE) consortium which has awarded grants to 45 other research institutes across the nation so they can begin groundbreaking work on gene editing. Through this consortium, the NIH hopes to find an efficient and safe way to conduct gene editing. Research programs are investigating the best delivery mechanism as well as the most dynamic gene editing tool.

The major problem with gene editing currently is the inability of cells to be edited within a living organism. It has become fairly easy and efficient to edit genes in a cell culture outside of the body but extremely difficult to do the same processes inside the body. Cheng, Lam and Segal are focused on changing this.

The question is how to do it inside of an animal and eventually a human, Lam said.

They are answering this question by utilizing Chengs work in engineering a non-toxic nanoparticle that they hope can transport the gene editing tool CRISPR into the cells of a living organism. Cheng has been able to create a Hepatitis E viral nanoparticle (HEVNP) that when manipulated could be a delivery system for CRISPR. They plan to take this nanoparticle and encase CRISPR inside of it, producing a mechanism for delivery of CRISPR.

The Hepatitis E nanoparticle has the capacity to be a highly efficient way to deliver gene editing to cells in the body due to its unique nature. HEVNP is resistant to the gastric acid environment of the intestines and stomach, enabling it to survive once its entered the body. Given its resistant abilities, HEVNP can be taken orally, making it a useful form of medicine. If able to successfully get HEVNP to the target cells in the body and deploy CRISPR, gene editing abilities could drastically change.

The addition of a cell-type specific targeting ligand to the HEVNP would code the nanoparticle to deliver CRISPR to a specific cell. The abilities of this method to be precise and safe will determine its success.

With five years of funding from the NIH, these three researchers are eager to begin work on this project and see the strides that can be made in gene editing. They have impressive goals for this research, as it has the capacity to reshape medicine.

This will redefine precision medicine as currently there is broad medicine that can cause side effects to people and not be effective, yet by making it specialized it is becoming more precise and effective, Cheng said.

As more effective and safe tools to cure illnesses are being tested and created, the benefits to society could be expansive. With so much potential to help improve the health of society, the NIH is dedicated to coming to new solutions at a quick rate. All programs that received grants will be required to share and utilize the research occurring at other funded programs. The NIH is hoping to eliminate the private nature of research through enforcing the sharing of ideas, as scientists are often constrained by the institutions they work for. It is their hope that by having communication between the programs, positive results will arise faster.

I think this is great because scientists inherently want to work with each other but have real world concerns especially with money, Segal said.

The research results, when groundbreaking, can provide incredible monetary gains and credibility to the institutions that made the discovery. Ultimately, scientists collaborating with one another will serve society as people are able to benefit earlier from this innovative research.

We want the public to know that we are working in their best interest, Segal said.

The NIH grant is competitive and still the third research program to join the consortium at UC Davis. Innovation has never been more prevalent than in this field at UC Davis. With three different programs researching gene editing, UC Davis stands out as a hotspot for this field of research.

Written by: Alma Meckler-Pacheco science@theaggie.org

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UC Davis leads in innovative gene editing research with NIH grants - The Aggie

Crispr Therapeutics AG (NASDAQ:CRSP) Stock analysts at Oppenheimer issued their Q1 2020 EPS estimates for Crispr Therapeutics in a note issued to investors on Tuesday, November 12th. Oppenheimer analyst S. Tuerkcan forecasts that the company will post earnings per share of ($0.87) for the quarter. Oppenheimer has a Outperform rating and a $65.00 price target on the stock. Oppenheimer also issued estimates for Crispr Therapeutics FY2021 earnings at ($4.29) EPS, FY2022 earnings at ($4.70) EPS and FY2023 earnings at ($1.07) EPS.

CRSP has been the subject of a number of other reports. Piper Jaffray Companies restated an overweight rating on shares of Crispr Therapeutics in a research note on Monday, October 21st. BTIG Research upped their target price on Crispr Therapeutics from $51.00 to $59.00 and gave the company a positive rating in a research note on Tuesday, July 30th. Roth Capital upped their target price on Crispr Therapeutics from $50.00 to $65.00 in a research note on Tuesday, July 30th. Canaccord Genuity initiated coverage on Crispr Therapeutics in a research note on Friday, July 26th. They issued a buy rating and a $72.00 target price for the company. Finally, BidaskClub upgraded Crispr Therapeutics from a hold rating to a buy rating in a research note on Friday. Two research analysts have rated the stock with a sell rating, three have given a hold rating and twelve have given a buy rating to the company. The stock has a consensus rating of Buy and an average price target of $57.95.

CRSP opened at $56.87 on Friday. The business has a 50 day moving average price of $43.31 and a 200 day moving average price of $44.57. The company has a debt-to-equity ratio of 0.06, a quick ratio of 8.32 and a current ratio of 8.32. The firm has a market cap of $3.04 billion, a P/E ratio of -16.53 and a beta of 3.15. Crispr Therapeutics has a twelve month low of $22.22 and a twelve month high of $57.40.

Crispr Therapeutics (NASDAQ:CRSP) last announced its quarterly earnings results on Monday, October 28th. The company reported $2.40 EPS for the quarter, beating the Thomson Reuters consensus estimate of ($0.95) by $3.35. Crispr Therapeutics had a negative return on equity of 2.60% and a negative net margin of 5.30%. The business had revenue of $211.93 million during the quarter, compared to analysts expectations of $6.32 million.

A number of large investors have recently made changes to their positions in CRSP. NEXT Financial Group Inc grew its position in Crispr Therapeutics by 915.0% in the third quarter. NEXT Financial Group Inc now owns 609 shares of the companys stock valued at $25,000 after acquiring an additional 549 shares during the period. Benjamin Edwards Inc. grew its position in Crispr Therapeutics by 96.4% in the second quarter. Benjamin Edwards Inc. now owns 546 shares of the companys stock valued at $26,000 after acquiring an additional 268 shares during the period. Coastal Investment Advisors Inc. bought a new stake in Crispr Therapeutics in the third quarter valued at $26,000. US Bancorp DE grew its position in Crispr Therapeutics by 553.7% in the second quarter. US Bancorp DE now owns 621 shares of the companys stock valued at $29,000 after acquiring an additional 526 shares during the period. Finally, BSW Wealth Partners bought a new stake in Crispr Therapeutics in the second quarter valued at $39,000. Hedge funds and other institutional investors own 51.09% of the companys stock.

In other Crispr Therapeutics news, Director Pablo J. Cagnoni sold 7,500 shares of the companys stock in a transaction on Tuesday, November 12th. The stock was sold at an average price of $55.00, for a total value of $412,500.00. Following the sale, the director now directly owns 7,500 shares in the company, valued at $412,500. The sale was disclosed in a document filed with the Securities & Exchange Commission, which is accessible through this hyperlink. Corporate insiders own 21.40% of the companys stock.

Crispr Therapeutics Company Profile

CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9) gene-editing platform in Switzerland. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells.

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Oppenheimer Weighs in on Crispr Therapeutics AG's Q1 2020 Earnings (NASDAQ:CRSP) - DFS Caller