Archive for the ‘Crispr’ Category

CRISPR Therapeutics (NASDAQ:CRSP) reported its fourth-quarter financial results on Wednesday afternoon, substantially beating revenue expectations and impressing both analysts and investors alike.

Fourth-quarter revenue grew to $77 million, a substantial increase from the mere $100,000 reported in Q4 2018. Total annual revenue came in at $289.6 million, while last year's income came in at a much smaller $3.1 million. While this surge is mainly due to collaboration agreements with Vertex Pharmaceuticals as opposed to product sales, it's still an impressive increase considering analysts had expected just $45.2 million for the quarter.

Image source: Getty Images.

Other financial metrics, such as CRISPR's cash position, have improved as well. By Dec. 31, cash and cash equivalents grew to $943.8 million, a 106.7% increase from the $456.6 million reported last year. The biotech company is also now reporting a net profit thanks to this revenue hike. Net income came in at $30.5 million, whereas last year CRISPR reported a net loss of $47.6 million in its 2018 fourth quarter.

CRISPR is developing four main treatments. CTX001 is a treatment for patients with sickle cell disease and transfusion-dependent beta thalassemia. Both of these are genetic blood disorders that impact the blood's ability to transport oxygen throughout the body.

The company's three other drugs, CTX110, CTX120, and CTX130, are types of cancer treatments known as CAR-T therapies. While other healthcare companies are developing their own CAR-T drugs, these types of therapies tend to be quite expensive. CRISPR's technology could make the development process for these types of drugs much cheaper than their competition.

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CRISPR Therapeutics Reports Q4 Earnings, Beats Revenue Expectations - The Motley Fool

CRISPR Therapeutics AG (CRSP) came out with quarterly earnings of $0.51 per share, beating the Zacks Consensus Estimate of $0.04 per share. This compares to loss of $0.92 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of 1,175%. A quarter ago, it was expected that this company would post a loss of $0.95 per share when it actually produced earnings of $2.40, delivering a surprise of 352.63%.

Over the last four quarters, the company has surpassed consensus EPS estimates two times.

CRISPR Therapeutics AG, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $77.02 million for the quarter ended December 2019, surpassing the Zacks Consensus Estimate by 3.42%. This compares to year-ago revenues of $0.12 million. The company has topped consensus revenue estimates two times over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

CRISPR Therapeutics AG shares have lost about 6.4% since the beginning of the year versus the S&P 500's gain of 3.9%.

What's Next for CRISPR Therapeutics AG?

While CRISPR Therapeutics AG has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for CRISPR Therapeutics AG was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #4 (Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is -$1.08 on $7.50 million in revenues for the coming quarter and -$4.65 on $30.04 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 28% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportTo read this article on Zacks.com click here.Zacks Investment Research

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CRISPR Therapeutics AG (CRSP) Beats Q4 Earnings and Revenue Estimates - Yahoo Finance

PHILADELPHIA - Three patients with advanced cancer suffered no serious side effects from being treated at the University of Pennsylvania in the first U.S. clinical study of cells edited with CRISPR, the gene editing technology.

But neither did they benefit, according to results published this month in Science. One patient with a bone marrow cancer called multiple myeloma has died and another has progressed. A patient with sarcoma, a soft tissue cancer, also progressed.

Pilot clinical trials are designed to assess safety, not effectiveness. And the inaugural U.S. test of CRISPR-edited cells in humans was so ethically and scientifically fraught that Penn spent more than two years getting necessary approvals for the January 2018 launch.

Still, the experiment was intended to combine and improve on two revolutionary immune-boosting approaches that showed startling effectiveness even in early trials. One approach, which cuts a natural brake on the immune system, has led to a class of cancer drugs called checkpoint inhibitors. The other approach genetically engineers immune soldiers called T cells to recognize and attack cancer cells; the first approved T-cell therapy, Novartis' Kymriah, was pioneered at Penn and Children's Hospital of Pennsylvania.

Although the CRISPR-edited cells did not melt away tumors or stop cancer progression, the cells did survive and grow in patients for up to nine months. In a previous Penn clinical trial that used engineered T cells to attack multiple myeloma, half the cells were dead in a week.

Penn T-cell researcher Carl June, principal leader of the new study and previous groundbreaking work, sees the CRISPR trial as another incremental step in a medical odyssey. CRISPR technology was invented just eight years ago, yet the version used by Penn is already so outmoded that the trial has been discontinued.

"We learned what we wanted to learn," June said of the study, on which Stanford University collaborated. "It opens up the door for a lot of new approaches."

What they learned was mostly reassuring.

The difficult, many-step manufacturing process was feasible. CRISPR was used on T cells to cut out two genes, one that codes for the immune system brake, and another that could hamper the T cells' ability to latch onto cancer cells. CRISPR also inserted a gene that enabled the T cells to recognize and target a protein found on the cancer cells but not healthy cells.

These edits accidentally caused some unusual rearrangements of DNA in a small fraction of T cells - one of the biggest worries with CRISPR. But as the T cells took hold and multiplied in patients, these rearrangements steadily decreased, "suggesting that they conferred no growth advantage," the researchers wrote.

The edited T cells did not trigger any of the dangerous toxicities related to revving up the immune system that are common with checkpoint inhibitors and engineered T cells.

However, the researchers noted that a longer trial with more patients and higher doses will be needed "to fully assess the safety of this approach."

A problem that has occurred with engineered T-cell therapies also showed up in the CRISPR trial: one patient's cancer cells stopped making the protein, called NY-ESO-1, that the T cells had been edited to target.

In future trials, "we would not just use NY-ESO-1 because we've learned the tumor can live without it," June said. "We'd want to use multiple targets."

Renier J. Brentjens, an oncologist and cell therapy researcher at Memorial Sloan-Kettering Cancer Center in New York City, called the paper an important "proof of principle."

"The T cells persisted and found the tumors. It would have been nice to see remissions or tumor regression, but it doesn't necessarily mean the approach is flawed," Brentjens said. "It may be that the target they used is not sufficient."

(c)2020 The Philadelphia Inquirer

Distributed by Tribune Content Agency, LLC.

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Study suggests editing human genes to fight cancer is safe. But does it work? - PostBulletin.com

The CRISPR Technology market report [5 Years Forecast 2020-2025] focuses on Major Leading Industry Players, providing info like market competitive situation, product scope, market overview, opportunities, driving force and market risks. Profile the top manufacturers of CRISPR Technology, with sales, revenue and global market share of CRISPR Technology are analyzed emphatically by landscape contrast and speak to info. Upstream raw materials and instrumentation and downstream demand analysis is additionally administrated. The CRISPR Technology market business development trends and selling channels square measure analyzed. From a global perspective, It also represents overall industry size by analyzing qualitative insights and historical data.

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CRISPR Technology Market 2020 Trends, Emerging Technologies and Growth Analysis BY Forecast to 2025 - Nyse Nasdaq Live

EPS growth is an important number as it gives a suggestion of the future prospects of a company. It is usually expressed as a percentage and is then referred to as the EPS growth rate. Growth in EPS is an important measure of administration performance because it shows how much money the company is making for its investors or shareholders, not only because of changes in profit, but also after all the effects of issuance of new shares (this is especially important when the growth comes as a result of acquisition).

CRISPR Therapeutics AG, belongs to Healthcare sector and Biotechnology industry. The companys Market capitalization is $3.48B with the total Outstanding Shares of 437. On 14-02-2020 (Friday), CRSP stock construct a change of -3.99 in a total of its share price and finished its trading at 55.8.

Profitability Ratios (ROE, ROA, ROI):

Looking into the profitability ratios of CRSP stock, an investor will find its ROE, ROA, ROI standing at -2.6%, -2% and -40.7%, respectively. Return on assets (ROA) is a financial ratio that shows the percentage of profit a company earns about its overall resources. A performance measure used to estimate the efficiency of an investment or to compare the ability of some different investments. ROI measures the amount of return on an investment relative to the investments cost.

Earnings per Share Details of CRISPR Therapeutics AG:

The EPS of CRSP is strolling at -0.46, measuring its EPS growth this year at -101.6%. As a result, the company has an EPS growth of -1824% for the approaching year.

Given the significance of identifying companies that will make sure earnings per share at a tall rate, we later obsession to umpire how to identify which companies will achieve high amassing rates. One obvious showing off to identify high earnings per portion count together companies are to locate companies that have demonstrated such build up beyond the p.s. 5 to 10 years.

We cant have sufficient maintenance the once will always replicate the difficult, but logically stocks that have grown earnings per allowance strongly in the subsequent to are a fine bet to keep on to take effect as a result.

Analysts mean target price for the company is $74.96 while analysts mean suggestion is 2.2.

A beta factor is used to measure the volatility of the stock. The stock remained 6.36% volatile for the week and 4.49% for the month.

Historical Performance In The News:

Taking a look at the performance of CRISPR Therapeutics AG stock, an investor will come to know that the weekly performance for this stock is valued at 6.65%, resulting in a performance for the month at -6.31%.

Therefore, the stated figure displays a quarterly performance of 1.45%, bringing six-month performance to 21.04% and year to date performance of -8.38%.

P/S, P/E, P/C and P/B/ SMA50, SMA 200:

The price-to-sales is a valuation ratio that relates a companys stock price to its revenues. The price-to-sales ratio is a symbol of the value placed on each dollar of a companys sales or taxes. As of now, CRSP has a P/S, P/E and P/B values of 16.37, 0 and 5.17 respectively. P/E and P/B ratios both are used on a regular basis by the investor to measure the value of the company and to get the right amount of the share.

Its P/Cash valued at 5.53. The price-to-cash-flow ratio is a stock valuation indicator that measures the value of a stocks price to its cash flow per share

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 0.5%, and its distance from 50 days simple moving average is -8.64% while it has a distance of 11.83% from the 200 days simple moving average. The companys distance from 52-week high price is 437% and while the current price is 437% from 52-week low price.

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Current Technicals:: CRISPR Therapeutics AG, (NASDAQ: CRSP) - NasdaqNewsFeed

Crispr Therapeutics AG (NASDAQ:CRSP) Equities researchers at Oppenheimer lowered their Q1 2020 earnings per share estimates for Crispr Therapeutics in a research report issued on Wednesday, February 12th. Oppenheimer analyst S. Tuerkcan now anticipates that the company will earn ($1.00) per share for the quarter, down from their previous forecast of ($0.89). Oppenheimer has a Outperform rating and a $80.00 price objective on the stock. Oppenheimer also issued estimates for Crispr Therapeutics Q2 2020 earnings at ($1.07) EPS, Q3 2020 earnings at ($1.16) EPS, Q4 2020 earnings at ($1.14) EPS, FY2020 earnings at ($4.38) EPS, FY2021 earnings at ($4.80) EPS, FY2022 earnings at ($5.90) EPS, FY2023 earnings at ($3.52) EPS and FY2024 earnings at $1.11 EPS.

Crispr Therapeutics (NASDAQ:CRSP) last announced its earnings results on Wednesday, February 12th. The company reported $0.51 earnings per share for the quarter, topping the consensus estimate of ($0.68) by $1.19. The business had revenue of $77.00 million for the quarter, compared to analysts expectations of $39.08 million. During the same period in the previous year, the company earned ($0.92) earnings per share. Crispr Therapeuticss quarterly revenue was up 76900.0% on a year-over-year basis.

Shares of CRSP opened at $55.80 on Monday. Crispr Therapeutics has a fifty-two week low of $30.75 and a fifty-two week high of $74.00. The firms 50 day moving average price is $57.63 and its 200 day moving average price is $53.03.

Several large investors have recently made changes to their positions in the business. Captrust Financial Advisors lifted its stake in shares of Crispr Therapeutics by 1,373.9% in the 4th quarter. Captrust Financial Advisors now owns 14,739 shares of the companys stock worth $874,000 after acquiring an additional 13,739 shares during the period. Cubist Systematic Strategies LLC lifted its stake in shares of Crispr Therapeutics by 672.8% in the 4th quarter. Cubist Systematic Strategies LLC now owns 47,140 shares of the companys stock worth $2,871,000 after acquiring an additional 41,040 shares during the period. First Republic Investment Management Inc. acquired a new stake in shares of Crispr Therapeutics in the 4th quarter worth $294,000. Marshall Wace LLP acquired a new stake in shares of Crispr Therapeutics in the 4th quarter worth $145,000. Finally, Bank of New York Mellon Corp lifted its stake in shares of Crispr Therapeutics by 17.1% in the 4th quarter. Bank of New York Mellon Corp now owns 46,167 shares of the companys stock worth $2,812,000 after acquiring an additional 6,736 shares during the period. 50.01% of the stock is currently owned by institutional investors.

In other Crispr Therapeutics news, Director Pablo J. Cagnoni sold 7,500 shares of the stock in a transaction that occurred on Tuesday, November 19th. The shares were sold at an average price of $62.00, for a total value of $465,000.00. Following the sale, the director now directly owns 7,500 shares of the companys stock, valued at approximately $465,000. The sale was disclosed in a legal filing with the SEC, which is available at the SEC website. Also, President Rodger Novak sold 33,618 shares of the stock in a transaction that occurred on Tuesday, November 19th. The shares were sold at an average price of $70.00, for a total transaction of $2,353,260.00. Following the completion of the sale, the president now directly owns 33,618 shares in the company, valued at $2,353,260. The disclosure for this sale can be found here. Corporate insiders own 21.40% of the companys stock.

About Crispr Therapeutics

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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Brokers Offer Predictions for Crispr Therapeutics AGs Q1 2020 Earnings (NASDAQ:CRSP) - Enterprise Echo

Defining life is hard. Plants, animals, and amoebas certainly fit, butwhat about virusesorprions? For those who put viruses on the non-living side of that division, things just got a bit awkward with the discovery of hundreds of viruses whose genome resembles bacteria.

Bacteriophages are bacteria-infecting viruses. Some have been usedin place of antibiotics, but their diversity is so great we've barely scratched the surface. Now, a study published inNaturehas turned up 351 bacteriophage species with genomes at least four times as large as most members of their class, almost four times the number known when the study began. These viruses have characteristics usually associated with living organisms, blurring the lines between life and non-life.

One genomic giant has 735,000 base-pairs, 15 times as many as a typical phage, beating the previous 596,000 record and more than many bacteria. The paper notes many of these jumbophages and megaphages are related, suggesting their genome size is an evolutionary stable trait, rather than something that occurred recently.

Large genomes are sometimes full of junk DNA, but these viruses are different. These enormous genomes carry parts of the CRISPR system. Now made famous as a method by which humans can edit our own genes withexceptional precision and power, CRISPR originated as a way for bacteria to fight off viral attacks. In the microbial worlds, where genesget shuffledbetween species, it seems these bacteriophages got hold of CRISPR and turned it to their own ends. Co-first author UC Berkeley graduate studentBasem Al-Shayebthinks the bacteriophages deploy CRISPR to defeat other viruses they battle for control of host bacteria.

These phages inhabit an astonishing variety of locations, from human guts and mouths to soils, hot springs and freshwater lakes, sometimes with closely related jumbophages found in very different environments.

"We are exploring Earth's microbiomes, and sometimes unexpected things turn up. These viruses of bacteria are a part of biology, of replicating entities, that we know very little about," Professor Jill Banfield said in astatement. There definitely seem to be successful strategies of existence that are hybrids between what we think of as traditional viruses and traditional living organisms."

Combined with this week's announcement of a virus containingmostly genes we've never seen beforewe're learning how little we know about the genetics of viruses that don't infect us.

The team suspects understanding bacteriophages' CRISPR adaptations will provide pointers for how we can use it ourselves.

Although phages don't directly infect humans, they can affect our health because they accelerate the transfer of genes between bacteria, including spreading those for antibiotic resistance. Banfield noted that larger genomes are more able to facilitate such gene transfer, so her discoveries may be particularly dangerous, making it important we learn to understand them better.

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Hundreds Of New Viruses With Enormous Genomes Blur The Boundaries Of Life - IFLScience

XconomyNational

CRISPR, capacity, and consolidation powered the cell and gene therapy space in 2019, but a proactive focus on patient access topped Falcon Therapeutics CEO Susan Nichols annual roundup.

In what has become one of the most anticipated presentations at the Phacilitate Leaders World Conference, Susan Nichols, CEO of private North Carolina-based cell therapy firm Falcon Therapeutics, laid out the top 10 events of the previous year that shaped the regenerative medicine space, driving conversation, investment, and innovation.

The top spot in 2019 focused on efforts to increase patient access to the life-changing therapies entering the market, withMays approval of Novartis/AveXis Zolgensma(onasemnogene abeparvovec) and its unprecedented $2.1 million (1.9 million) price tag being the catalyst for change.

Nichols number one spot in 2018 centered around reimbursement conversations. Sparks approval of gene therapy Luxturna (voretigene neparvovec) shook 2017, while Europes approval ofex-vivostem cell gene therapy Strimvelis significantly advanced the sector in 2016.

For further context,check out theTop 10 cell and gene therapy milestones of 2018here, but below in reverse order is the full list of the top 10 key events of the previous 12 months, as presented at the conference in Miami, FL:

In December, a jury found Kite Pharma owned by Gilead Sciences (NASDAQ: GILD) guilty of infringing a patent exclusively licensed by Juno Therapeutics owned by Bristol-Myers Squibb (NYSE: BMS) from researchers at the Memorial Sloan Kettering Cancer Center.

The 190 patentrelates to technology used in Kite/Gileads chimeric antigen receptor (CAR) T-cell therapy Yescarta (axicabtagene ciloleucel).

The jurys decision left Gilead to pay $585 million plus 27.6% in royalties, totaling $752 million, to Bristol and Sloan Kettering, resolving a case filed a day after Yescarta won approval in October 2017.

But in a post-script that could well feature on Nichols 2020 list,it has been suggested the emboldening of Bristol has led the firm to file a motion last month to include punitive damages that would raise Gileads penalty to $1.5 billion.

Vertex (NASDAQ: VRTX) and CRISPR Therapeutics (NASDQ: CRSP) opened clinical trials in b-thalassemia and sickle cell disease to replace the defective genes that case these disorders andin November, the firms announced positive efficacy data from the first two patients treated with the investigational therapy CTX001.

Meanwhile, Editas Medicine (NASDAQ: EDIT) and Allergan initiated clinical trials for their CRISPR-based candidate AGN-151587 (EDIT-101), aimed at treating Leber congenital amaurosis 10 (LCA10), an inherited form of blindness.

The significance is CRISPR therapies have finally arrived in the clinic, Nichols said.

8) Pharma and biotech inhouse manufacturing

With a lack of third-party capacity especially for viral vector production, 2019 saw numerous investments by major cell and gene therapy players to grow their internal networks. Some of the examples Nichols pointed out include:

Susan Nichols, CEO of Falcon Therapeutics, spoke at Phacilitate in Miami, Florida in January

Positive data from Decembers American Association of Hematology (ASH) meeting in San Diego, CA was a further boon for the sector, said Nichols.

Johnson & Johnsons (NYSE: JNJ) JNJ-4528, a CAR-T Cell Therapy Directed Against B-Cell Maturation Antigen (BCMA), reported a 100% remission rate and response from its Phase Ib/II CARTITUDE-1 trial. 69% of patients showed complete remission or better.

The candidate licensed fromNanjing Legend in a $350 million deal will move into a full Phase II study this year.

ASH also brought positive news from bluebird and Bristol-Myers Squibb, which saw a 73.4% overall response rate in a Phase II KarMMa trial of its BCMA-targeted CAR-T candidate idecabtagene vicleucel.

The $950 million deal,announced in September, adds Semma Therapeutics a firm focusing on using stem-cell derived human islets as a possible cure for type 1 diabetes to Vertex growing regenerative medicine portfolio.

For Vertex, the deal represented its entry into the cell therapy space, complementing its move into gene editing just months prior with theacquisition of Exonics and a research expansion with CRISPR Therapeutics.

But for the industry, the investment in a company developing a cell therapy for a large indication other than cancer is of major significance, said Nichols.

As mentioned before, Astellas acquired Audentes for $3 billion, but the Japanese pharma firm also bought South San Francisco-basedCAR technology developer Xyphos Biosciencesas part of an end-of-year buying spree.

According to Nichols, these deals by Astellas are a signifier that medium pharma may be using advanced therapies to grow and expand.

With a wealth of therapies moving through the clinic, capacity is at a premium and 2019 saw contract development and manufacturing organizations (CDMOs) scrabbling to secure capabilities.

Thermo Fisher Scientific (NYSE: TMO) acquiredBrammer Bio for $1.7 billion, then Catalent (NYSE: CTLT) paid $1.2 billion toadd Paragon Bioservicesto its CDMO offering. Both marked the first move into gene therapy services by the two large contract manufacturers. Nichols noted the size of the deals as being somewhat impressive.

In other signs of CDMO consolidation, Hitachi Chemical Advanced Therapeutics Solutions (HCATS) entered Europe byacquiring German cell therapy manufacturing firm apceth Biopharma, and Tennessee-based cell therapy firm Cognateacquired Swedish DNA and viral vector manufacturer Cobra Biologics.

2019 also saw a flood of licensing deals with large upfront payments.

Roche (OTCGX: RHHBY) is paying more than $1 billion upfrontfor the rights to Sarepta Therapeutics (NASDAQ: SRPT) Duchenne muscular dystrophy (DMD) gene therapy outside of the US.

Genentech entered a $300 million with Adaptive Bio (NASDAQ: ADPT) for access to its T-receptor discovery and immune profiling platform, though the deal could be worth up to $2 billion.

And Vertex, as previously mentioned, inked a $175 million deal with CRISPR Therapeutics for its gene therapy pipeline.

We saw medium pharma grow. We saw major licensing deals. We saw CDMO consolidation. But we also saw Big Pharmas buying power with the sector making a significant impact on the cell and gene therapy space in 2019.

The biggest deal sawBristol buy Celgenefor a whopping $74 billion, bringing with it several CAR-T programs.

But Roches $4.8 billionacquisition of Spark Therapeutics which has already seen commercial success with Luxturna was also significant, as was Biogens (NASDAQ: BIIB) $877 million purchase ofNightstar Therapeutics, Pfizers stake-in and optionto buy out Vivet, and Bayers acquisition of the remaining shares ofBlueRock Therapeutics.

These signal that Big Pharma is optimistic to M&A in the advance therapy space and the value that these therapies can bring, said Nichols.

The business model for this new breed of curative medicines is significantly different to that of traditional pharma and biologics, and patient access poses a challenge. With the arrival of Zolgensma and its $2.1 million price tag, the conversations have changed, and all elements of the industry have been forced to address how to manage patient access.

Zolgensma represents a life or death drug for 68% of pediatric patients with SMA1. The patients must be dosed before the age of two, yet only around ten states offer screening before this age.

We need to work as an industry to ensure reimbursement and access is in sync with approvals, said Nichols. However, she added, we must move the conversation to state level and bring state Medicaid and insurance companies to the core of the conversation.

The year saw positive signs that change is happening.

Nichols noted that patient advocacy voices are loud across all disease indications pushing for access to these next-generation medicines. Meanwhile Novartis suggested lottery-style free drug program despitesome criticism demonstrates industry itself is looking for innovative ways to improve access.

This article first appeared in Bioprocess Insider on January 27.

Image: iStock/PashaIgnatov

Dan Stanton is Xconomy's managing editor and is based in France. You can reach him at dan.stanton@knect365.com.

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CRISPR, CAR-T, Consolidation: Top Advanced Therapy Milestones of 2019 - Xconomy

-Enrollment ongoing in clinical trials of CTX001 for patients with severe hemoglobinopathies-

-Enrollment ongoing in clinical trial of CTX110, targeting CD19+ malignancies-

-Enrollment has begun in clinical trial of CTX120, targeting B-cell maturation antigen (BCMA)-

ZUG, Switzerland and CAMBRIDGE, Mass., Feb. 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the fourth quarter and full year ended December 31, 2019.

In 2019, CRISPR Therapeutics achieved important milestones and momentum across key programs. We announced positive interim safety and efficacy data from the first two patients in our ongoing CTX001 clinical trials, one patient with beta thalassemia and one patient with sickle cell disease. These preliminary data support our belief in the potential of CTX001 to have meaningful benefit for patients following a one-time intervention, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. In addition, we advanced our first allogeneic CAR-T cell therapy, CTX110, targeting CD19+ malignancies and, building on this progress, today announced that we have begun enrolling patients in a clinical trial for our second allogeneic CAR-T therapy, CTX120, targeting BCMA for the treatment of relapsed or refractory multiple myeloma.

Dr. Kulkarni added: 2020 has the potential to be a pivotal year in our companys growth. We expect to conduct clinical trials in five indications, and we anticipate new data from our immuno-oncology and hemoglobinopathies programs. Our continued progress brings us closer to potentially providing transformative therapies to patients with serious diseases.

About CTX001TMCTX001 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 CTX110TMCTX110 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19, or CD19, for the treatment of CD19+ malignancies. A wholly-owned asset of CRISPR Therapeutics, CTX110 is in a clinical trial designed to assess the safety and efficacy of CTX110 in relapsed or refractory B-cell malignancies. The multi-center, open-label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110.

About CTX120TMCTX120 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting B-cell maturation antigen, or BCMA. A wholly-owned asset of CRISPR Therapeutics, CTX120 is in a clinical trial designed to assess the safety and efficacy of CTX120 in relapsed or refractory multiple myeloma. The multi-center, open-label clinical trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

About CTX130TMCTX130 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting CD70, an antigen expressed on hematologic cancers. A wholly-owned asset of CRISPR Therapeutics, CTX130 is in development for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies.

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

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics various clinical programs including CTX001, CTX110, CTX120 and CTX130; (ii) the status of 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 by CRISPR Therapeutics and 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; (iv) the integration of Casebia Therapeutics; (v) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (vi) the sufficiency of CRISPR Therapeutics cash resources; ; (vii) the expected benefits of CRISPR Therapeutics collaborations, including those with Bayer, KSQ, ProBioGen, StrideBio and Vertex; and (viii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial (including CTX001, CTX110, CTX120 and CTX130) not to be indicative of final trial results; the risk that the initial data from a limited number of patients 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; 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; the risk that the CRISPR Therapeutics business and Casebia Therapeutics business will not be integrated successfully; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact:Susan Kimsusan.kim@crisprtx.com

Media Contact:Rachel EidesWCG on behalf of CRISPR617-337-4167 reides@wcgworld.com

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CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2019 Financial Results - GlobeNewswire

A look at the shareholders of CRISPR Therapeutics AG (NASDAQ:CRSP) can tell us which group is most powerful. Institutions often own shares in more established companies, while its not unusual to see insiders own a fair bit of smaller companies. Companies that have been privatized tend to have low insider ownership.

CRISPR Therapeutics has a market capitalization of US$3.4b, so its too big to fly under the radar. Wed expect to see both institutions and retail investors owning a portion of the company. Taking a look at our data on the ownership groups (below), its seems that institutions own shares in the company. We can zoom in on the different ownership groups, to learn more about CRISPR Therapeutics.

Check out our latest analysis for CRISPR Therapeutics

Institutions typically measure themselves against a benchmark when reporting to their own investors, so they often become more enthusiastic about a stock once its included in a major index. We would expect most companies to have some institutions on the register, especially if they are growing.

We can see that CRISPR Therapeutics does have institutional investors; and they hold 41% of the stock. This suggests some credibility amongst professional investors. But we cant rely on that fact alone, since institutions make bad investments sometimes, just like everyone does. If multiple institutions change their view on a stock at the same time, you could see the share price drop fast. Its therefore worth looking at CRISPR Therapeuticss earnings history, below. Of course, the future is what really matters.

Hedge funds dont have many shares in CRISPR Therapeutics. The companys largest shareholder is Vertex Pharmaceuticals Incorporated, with ownership of 9.1%, With 7.1% and 5.5% of the shares outstanding respectively, Versant Venture Management, LLC and S.R. One, Limited are the second and third largest shareholders.

A closer look at our ownership figures suggests that the top 11 shareholders have a combined ownership of 50% implying that no one share holder has a majority.

Researching institutional ownership is a good way to gauge and filter a stocks expected performance. The same can be achieved by studying analyst sentiments. Quite a few analysts cover the stock, so you could look into forecast growth quite easily.

While the precise definition of an insider can be subjective, almost everyone considers board members to be insiders. The company management answer to the board; and the latter should represent the interests of shareholders. Notably, sometimes top-level managers are on the board, themselves.

Most consider insider ownership a positive because it can indicate the board is well aligned with other shareholders. However, on some occasions too much power is concentrated within this group.

Our most recent data indicates that insiders own some shares in CRISPR Therapeutics AG. The insiders have a meaningful stake worth US$61m. Most would see this as a real positive. It is good to see this level of investment by insiders. You can check here to see if those insiders have been buying recently.

The general public holds a 26% stake in CRSP. While this group cant necessarily call the shots, it can certainly have a real influence on how the company is run.

Private equity firms hold a 13% stake in CRSP. This suggests they can be influential in key policy decisions. Some investors might be encouraged by this, since private equity are sometimes able to encourage strategies that help the market see the value in the company. Alternatively, those holders might be exiting the investment after taking it public.

It appears to us that public companies own 19% of CRSP. This may be a strategic interest and the two companies may have related business interests. It could be that they have de-merged. This holding is probably worth investigating further.

I find it very interesting to look at who exactly owns a company. But to truly gain insight, we need to consider other information, too. Take risks, for example CRISPR Therapeutics has 4 warning signs (and 1 which is potentially serious) we think you should know about.

But ultimately it is the future, not the past, that will determine how well the owners of this business will do. Therefore we think it advisable to take a look at this free report showing whether analysts are predicting a brighter future.

NB: Figures in this article are calculated using data from the last twelve months, which refer to the 12-month period ending on the last date of the month the financial statement is dated. This may not be consistent with full year annual report figures.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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What Kind Of Shareholders Own CRISPR Therapeutics AG (NASDAQ:CRSP)? - Simply Wall St

A heated battle over the future of animal gene-editing in the US has intensified following the publication of dueling articles from the Food and Drug Administration (FDA) and the editorial board of the journal Nature Biotechnology.

In an analysis of genome-edited cattle and an accompanying letter to the editor, the FDA defended its proposal to regulate gene-edited animals as veterinary drugs, arguing the regulation would help prevent unintended consequences that could arise from the use of new breeding techniques such as CRISPR-Cas9. Nature responded in a sharply wordededitorial claiming the agencys proposal is at odds with the evidence, and saying it appears primarily designed to avoid legal battles with litigious anti-GMO groups.

In January 2020, Genetic Literacy Project released its Global gene-editing regulation tracker and index, evaluating nations based on how their laws encourage or hinder innovation. These tools illustrate that the US is a pioneer in agricultural gene-editing research, ranking seventh in the world by maintaining sensible rules that protect public health while developing novel food crops that are addressing important nutritional and environmental challenges.

The one exception in US policy is animal biotechnology. Brazil, Argentina, Japan, Canada and Australia have thus far taken a much more progressive stance on this issue. There are currently no gene-edited animals approved for human consumption in the US.

Under the FDAs oversight, only one genetically engineered animal, the transgenic (GMO) fast-growing AquAdvantage salmon developed by AquaBounty has received regulatory approval, which took roughly 20 years. Because of political wrangling, the salmon is not yet sold in the US, but salmon eggs imported from Canada are being raised at an AquAdvantage fish farm in Indiana. In May 2016, the Canadian Food Inspection Agency approved the sale of the GM fish, and AquaAdvantage salmon fillets became available to customers in Canada.

The FDA is now poised to classify gene-edited animals as veterinary drugs, which would put them in the same regulatory bucket as GMO animals, such as AquaBountys salmon, which is engineered in part using genes from another fish species.

Critics contend this proposed policy would trigger extensive, costly premarket regulatory review that would severely limit the ability of scientists to breed gene-edited animals, though the FDA says it will balance its guidelines to protect public health without hampering innovation. The agencys proposal has been panned by animal biotech scientists, some of whom have moved their breeding programs to countries with less restrictive regulations.

FDA stands its ground

Writing in Nature Biotechnology on February 7, FDA Center for Veterinary Medicine (CVM) Director Steven Solomon explained the rationale guiding the agencys proposal. Citing the examples of a gene-edited bull whose genome was unintentionally altered to contain bacterial DNA and a conventionally bred animal from the 1950s, Solomon argued that:

The intended genome edit sought to introduce the Celtic POLLED allele into Holstein cattle. This allele exists in some other cattle breeds and results in the animals lacking horns (or being polled) . [H]owever, the editing also produced unintended modifications . Unintended alterations can have unexpected and deleterious consequences no matter the size of the alteration or how it was produced . There is a particularly compelling example of the risks of occult genomic alterations in cattle produced by traditional breeding techniques: a high incidence of bovine leukocyte adhesion deficiency (BLAD) syndrome, a lethal autosomal recessive disease, in Holstein calves.

The selection of a particular Holstein bull for superior milk production genetics resulted in wide dissemination of carrier bulls semen for breeding beginning in the 1950s and 1960s. It turned out that the selected bull was a carrier of a naturally occurring single point mutation that causes BLAD when two copies are present. The extensive use of carrier bulls semen led to an eventual 23% BLAD carrier frequency in Holstein calves in the United States. It took a decade to effectively breed the genetic mutation that causes BLAD out of dairy cattle genetics.

. We recognize that there is tremendous excitement over quickly embracing and bringing to market the fruits of genome editing technology, as well as the critical importance of adequately identifying potential risks, efficiently evaluating whether the risks do in fact exist, and determining whether the risks pose an actual safety hazard in a timely manner.

It is the FDAs role to balance these competing imperatives. We want to support the timely development of beneficial products, but not at the expense of abdicating our critical role in protecting consumer and animal health. We have a public health obligation to protect consumer and animal health that we must balance with the potential of this innovative technology to enhance human and animal health and food production.

Experts unconvinced

Nature Biotechnologys editorial board found Solomons case unpersuasive. If anything, they wrote, his examples undermine the justification for strict FDA oversight of gene-edited animals:

BLAD is not a justification for mandatory regulation of all gene-edited animals . [I]t illustrates that conventional breeding might warrant tighter FDA oversight, especially when rapid breeding expansion programs thrust particular genetic profiles to the fore . [I]f the BLAD case history tells us anything, it is that the origin of a DNA arrangement (conventional breeding, recombinant DNA or gene editing) makes little difference to an animal.

While the editorial authors conceded that the presence of extraneous donor plasmid in the gene-edited POLLED Holstein DNA was unexpected and initially missed, they said the unintended edit should be properly contextualized. The genomes of domesticated cattle contain north of 86 million mutationsinsertions, deletions and single nucleotide variantsand none of these changes is linked to negative health outcomes in humans who consume milk or meat from cows. Amidst this background of innocuous variation, how can the presence of one identifiable variant justify the costs and delays of mandatory FDA oversight? the authors asked.

The answer, they continued, has more to do with politics than science. Anti-GMO groups have utilized every tool at their disposal to restrict the progress of agricultural biotechnology. One of their most potent weapons has been a constant stream of litigation aimed at the USDA, FDA and EPA, the three federal agencies tasked with oversight of biotechnology. Activist litigation was a primary factor in the delayed approval of AquaBountys salmon, for example. The groups behind the mass of lawsuits have also successfully stoked consumer concern about the alleged risks of genetically engineered crops and animals. Meanwhile, the relatively small animal biotechnology industry doesnt have anywhere near the same the public relations and legal resources. As the editorial explained:

[T]here are very few companies in this sector to argue the case for genetically engineered (including gene-edited) animals . On the other hand, there is a powerful and litigious anti-GMO/pro-organic lobby that repeatedly challenges the legitimacy of regulatory rulings and attempts to block market access following approval.

Life-saving biotech drugs gain wide support in Washington. But there is little political capital invested in backing gene-edited animals. Food remains plentiful and, if anything, the public mood is shifting to vegetarianism and animal-substitute products. Scientists and breeders want to use new technology in agriculture, but public sentiment nostalgically harks back to Victorian farming practices in a way that it doesnt for Victorian medical practices.

A cautious, process-based regulatory route keeps the FDA out of trouble and lowers litigation risks for CVMs lawyers.

The authors ended with a proposal of their own, urging the FDA to regulate gene-edited animals based on the risks they may pose to human health, not the process that was used to breed them:

Mandatory oversight could be phased out to a system whereby the agency exercises discretion over which gene-edited animals are regulated according to the hazard represented by the introduced trait. This would be consistent with USDA policy and longstanding US regulatory policy. It would give the animal biotech sector a chance to bloom. And it would counter the narrative of fearmongers who would taint all gene-edited animals as hazardous to public health and injurious to animal welfare.

Cameron J. English is the GLPs senior agricultural genetics and special projects editor. He is a science writer and podcast host. BIO. Follow him on Twitter @camjenglish

Originally posted here:
FDA defends CRISPR-edited animal rules likely to block most uses: Is the agency trying to avoid litigation from anti-GMO groups? - Genetic Literacy...

Recent gene editing technologies advances, such as CRISPR/Cas9, will continue to shape the future of agriculture and genetically engineered crops. Using a representative survey of a North American Midwestern state, we examine the relative weights of specific risks and benefits associated with GMO foods in impacting potential rejection of the technology.

Controlling for established predictors, we find perceptions of specific risks and benefits of the technology have a significant and substantial impact on GMO rejection, with risk aspects playing a relatively greater role. Two risks, viewing GMOs as benefiting food manufacturers and causing allergies and illness, are among the strongest predictors of GMO food rejection, suggesting social dimensions are important to consider and present in the public mind.

Supplementing this, people also consider aspects related to health and nature. We discuss implications for communication efforts about GE foods and crops, and for the future of gene editing in food production.

Together, our results emphasize the interconnectivity of media coverage of GMOs and public rejection, in this case through the coverage and subsequent salience of various aspects of GM foods. Although we nd that the eects of media attention are limited with the addition of perceptions of risks and benets associated with GM food into the model (suggesting a potential overlap in explanatory power), media attention remains a strong predictor of rejection. Further, previous research has suggested that another predictor, perceived familiarity, is likely connected to media attention, again underscoring the importance of the media in GM food rejection.

We suggest that some specic potential risks and benets may be more salient for those who pay attention to news about GM foods based on the aspects of the technology that are frequently discussed. Our results also indicate that those who pay more attention to GMO-specic news rate the importance of GMO-free foods more highly. A brief consideration of the ongoing media coverage on GMO-related issues, such as mandatory labeling legislation or concerns with the ethics of biotechnology companies, supports this nding.

Additionally, although viewing GM foods as unnatural, an indication of moral concern with the technology, is associated with increased rejection, we nd that other aspects of the technology are of greater weight in explaining rejection. This nding pushes against recent claims on the pervasive appeal of GMO opposition as an emotion-based response to the unnaturalness of GE foods. Rather, the perhaps less instinctually-based views of GM foods as only beneting food manufacturers and causing allergies and illness elicited stronger responses.

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Consumers who reject CRISPR fear that gene-edited crops cause allergies and only benefit food companies, survey shows - Genetic Literacy Project

FIDELITY MANAGEMENT & RESEARCH C bought a fresh place in CRISPR Therapeutics AG (NASDAQ:CRSP). The institutional investor bought 368.6 thousand shares of the stock in a transaction took place on 12/31/2019. In another most recent transaction, which held on 12/31/2019, NIKKO ASSET MANAGEMENT AMERICAS, bought approximately 272.1 thousand shares of CRISPR Therapeutics AG In a separate transaction which took place on 12/31/2019, the institutional investor, ARK INVESTMENT MANAGEMENT LLC bought 174.5 thousand shares of the companys stock. The total Institutional investors and hedge funds own 47.60% of the companys stock.

In the most recent purchasing and selling session, CRISPR Therapeutics AG (CRSP)s share price decreased by -3.70 percent to ratify at $52.32. A sum of 1031772 shares traded at recent session and its average exchanging volume remained at 1.21M shares. The 52-week price high and low points are important variables to concentrate on when assessing the current and prospective worth of a stock. CRISPR Therapeutics AG (CRSP) shares are taking a pay cut of -29.30% from the high point of 52 weeks and flying high of 78.32% from the low figure of 52 weeks.

CRISPR Therapeutics AG (CRSP) shares reached a high of $54.70 and dropped to a low of $51.92 until finishing in the latest session at $54.45. Traders and investors may also choose to study the ATR or Average True Range when concentrating on technical inventory assessment. Currently at 2.41 is the 14-day ATR for CRISPR Therapeutics AG (CRSP). The highest level of 52-weeks price has $74.00 and $29.34 for 52 weeks lowest level. After the recent changes in the price, the firm captured the enterprise value of $2.3B. The liquidity ratios which the firm has won as a quick ratio of 8.30, a current ratio of 8.30 and a debt-to-equity ratio of 0.00.

Having a look at past record, were going to look at various forwards or backwards shifting developments regarding CRSP. The firms shares rose 0.71 percent in the past five business days and shrunk -16.33 percent in the past thirty business days. In the previous quarter, the stock rose 7.99 percent at some point. The output of the stock increased 6.67 percent within the six-month closing period, while general annual output gained 75.69 percent. The companys performance is now negative at -14.10% from the beginning of the calendar year.

According to WSJ, CRISPR Therapeutics AG (CRSP) obtained an estimated Overweight proposal from the 16 brokerage firms currently keeping a deep eye on the stock performance as compares to its rivals. 2 equity research analysts rated the shares with a selling strategy, 3 gave a hold approach, 11 gave a purchase tip, 0 gave the firm a overweight advice and 0 put the stock under the underweight category. The average price goal of one year between several banks and credit unions that last year discussed the stock is $77.50.

Schlumberger Limited (SLB) shares on Fridays trading session, dropped -0.26 percent to see the stock exchange hands at $34.42 per unit. Lets a quick look at companys past reported and future predictions of growth using the EPS Growth. EPS growth is a percentage change in standardized earnings per share over the trailing-twelve-month period to the current year-end. The company posted a value of -$7.32 as earning-per-share over the last full year, while a chance, will post $1.97 for the coming year. The current EPS Growth rate for the company during the year is -576.90% and predicted to reach at 23.29% for the coming year. In-depth, if we analyze for the long-term EPS Growth, the out-come was -29.90% for the past five years and the scenario is totally different as the current prediction is 11.03% for the next five year.

The last trading period has seen Schlumberger Limited (SLB) move -29.58% and 12.30% from the stocks 52-week high and 52-week low prices respectively. The daily trading volume for Schlumberger Limited (NYSE:SLB) over the last session is 8.52 million shares. SLB has attracted considerable attention from traders and investors, a scenario that has seen its volume drop -20.34% compared to the previous one.

Investors focus on the profitability proportions of the company that how the company performs at profitability side. Return on equity ratio or ROE is a significant indicator for prospective investors as they would like to see just how effectively a business is using their cash to produce net earnings. As a return on equity, Schlumberger Limited (NYSE:SLB) produces -33.90%. Because it would be easy and highly flexible, ROI measurement is among the most popular investment ratios. Executives could use it to evaluate the levels of performance on acquisitions of capital equipment whereas investors can determine that how the stock investment is better. The ROI entry for SLBs scenario is at -24.50%. Another main metric of a profitability ratio is the return on assets ratio or ROA that analyses how effectively a business can handle its assets to generate earnings over a duration of time. Schlumberger Limited (SLB) generated -15.90% ROA for the trading twelve-month.

Volatility is just a proportion of the anticipated day by day value extendthe range where an informal investor works. Greater instability implies more noteworthy benefit or misfortune. After an ongoing check, Schlumberger Limited (SLB) stock is found to be 2.29% volatile for the week, while 2.52% volatility is recorded for the month. The outstanding shares have been calculated 1.41B. Based on a recent bid, its distance from 20 days simple moving average is -5.19%, and its distance from 50 days simple moving average is -8.70% while it has a distance of -6.51% from the 200 days simple moving average.

The Williams Percent Range or Williams %R is a well-known specialized pointer made by Larry Williams to help recognize overbought and oversold circumstances. Schlumberger Limited (NYSE:SLB)s Williams Percent Range or Williams %R at the time of writing to be seated at 46.62% for 9-Day. It is also calculated for different time spans. Currently for this organization, Williams %R is stood at 69.98% for 14-Day, 79.97% for 20-Day, 82.56% for 50-Day and to be seated 64.06% for 100-Day. Relative Strength Index, or RSI(14), which is a technical analysis gauge, also used to measure momentum on a scale of zero to 100 for overbought and oversold. In the case of Schlumberger Limited, the RSI reading has hit 37.46 for 14-Day.

Continued here:
Try CRISPR Therapeutics AG (CRSP) and Schlumberger Limited (SLB) which never ever heard from Anyone - BOV News

Global CRISPR Genome Editing 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 Genome Editing industry.

The report also covers segment data, including: type segment, industry segment, channel segment etc. cover different segment market size, both volume and value. Also cover different industries clients information, which is very important for the manufacturers.

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

Make An EnquiryAbout This Report @ https://www.researchmoz.com/enquiry.php?type=E&repid=2602918&source=atm

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

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

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

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

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

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

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Important Key questions answered in CRISPR Genome Editing market report:

What will the market growth rate, Overview, and Analysis by Type of CRISPR Genome Editing in 2024?

What are the key factors affecting market dynamics? What are the drivers, challenges, and business risks in CRISPR Genome Editing market?

What is Dynamics, This Overview Includes Analysis of Scope and price analysis of top Manufacturers Profiles?

Who Are Opportunities, Risk and Driving Force of CRISPR Genome Editing market? Knows Upstream Raw Materials Sourcing and Downstream Buyers.

Who are the key manufacturers in space? Business Overview by Type, Applications, Gross Margin, and Market Share

What are the opportunities and threats faced by manufacturers in the global market?

You can Buy This Report from Here @ https://www.researchmoz.com/checkout?rep_id=2602918&licType=S&source=atm

The content of the study subjects, includes a total of 15 chapters:

Chapter 1, to describe CRISPR Genome Editing product scope, market overview, market opportunities, market driving force and market risks.

Chapter 2, to profile the top manufacturers of CRISPR Genome Editing , with price, sales, revenue and global market share of CRISPR Genome Editing in 2019 and 2015.

Chapter 3, the CRISPR Genome Editing competitive situation, sales, revenue and global market share of top manufacturers are analyzed emphatically by landscape contrast.

Chapter 4, the CRISPR Genome Editing breakdown data are shown at the regional level, to show the sales, revenue and growth by regions, from 2019 to 2025.

Chapter 5, 6, 7, 8 and 9, to break the sales data at the country level, with sales, revenue and market share for key countries in the world, from 2019 to 2025.

Chapter 10 and 11, to segment the sales by type and application, with sales market share and growth rate by type, application, from 2019 to 2025.

Chapter 12, CRISPR Genome Editing market forecast, by regions, type and application, with sales and revenue, from 2019 to 2025.

Chapter 13, 14 and 15, to describe CRISPR Genome Editing sales channel, distributors, customers, research findings and conclusion, appendix and data source.

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CRISPR Genome Editing Market Globally Expected to Drive Growth Through 2019-2026 - Jewish Life News

The 68 rating InvestorsObserver gives to Crispr Therapeutics AG (CRSP) stock puts it near the top of the Healthcare sector.

In addition to scoring higher than 86 percent of stocks in the Healthcare sector, CRSPs 68 overall rating means the stock scores better than 68 of all stocks.

Finding the best stocks can be tricky. It isnt easy to compare companies across industries. Even companies in the healthcare sector can be tricky to compare sometimes. InvestorsObservers tools allow a top-down approach that lets you pick a metric, find the top sector and industry and then find the best stocks in that sector.

Not only are these scores easy to understand, but it is easy to compare stocks to each other. You can find the best stock in healthcare or look for the sector that has the highest average score.

The overall score is a combination of technical and fundamental factors that serves as a good starting point when analyzing a stock. Traders and investors with different goals may have different goals and will want to consider other factors than just the headline number before making any investment decisions.)

Crispr Therapeutics AG (CRSP) stock has fallen -0.69% while the S&P 500 has gained 0.69% as of 10:33 AM on Tuesday, Feb 11. CRSP is down -$0.39 from the previous closing price of $56.62 on volume of 375,983 shares. Over the past year the S&P 500 is up 24.56% while CRSP has gained 80.63%. CRSP lost -$0.46 per share the over the last 12 months.

To screen for more stocks like CRSP click here.

Read more from the original source:
Healthcare Sector: Is Crispr Therapeutics AG (CRSP) A Winner? - InvestorsObserver

CAMBRIDGE, Mass., Feb. 10, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, is presenting new data from two of its development programs at Keystone Symposias Engineering the Genome Conference, a joint meeting with the Emerging Cellular Therapies: Cancer and Beyond Conference, taking place Feb. 8-12, 2020, in Banff, Canada. Intellia researchers are presenting data in support of the companys lead engineered cell therapy development candidate, NTLA-5001 for the treatment of the hematological cancer, acute myeloid leukemia (AML). Intellia also is sharing preclinical results for its hereditary angioedema (HAE) program, which is Intellias third CRISPR/Cas9 development program, announced in January 2020.

Intellia continues to demonstrate strong progress across both our engineered cell therapy and in vivo pipelines, said Intellia President and Chief Executive Officer John Leonard, M.D. We are observing very favorable preclinical data with our engineered T cells, and we are moving ahead with IND-enabling studies and manufacturing for NTLA-5001, to enable a regulatory submission in the first half of 2021.

On the in vivo side, the data from our HAE development program reinforce the modularity of Intellia's non-viral delivery genome editing platform and how it is enabling independent, single-dose therapies for multiple monogenic diseases. For HAE, we expect to nominate a development candidate in the first half of this year, continued Dr. Leonard.

New Data from Intellias Engineered Cell Therapy Development Program for AML

NTLA-5001, which is Intellias first engineered T cell therapy development candidate and is wholly owned, utilizes a T cell receptor (TCR)-directed approach to target the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. The companys WT1-TCR T cell approach aims to develop a broadly applicable treatment for AML patients, regardless of mutational background of a patients leukemia.

The company is presenting data demonstrating that the selection of a natural, high-affinity TCR, in combination with CRISPR-enabled engineering and targeted insertion, results in an engineered T cell capable of specific and potent killing of primary AML blasts. Todays presentation at Keystone builds on data previously presented last fall at the Annual Congress of the European Society of Gene and Cell Therapy (ESGCT).

The data being presented at the Keystone conference substantiate the advantages that a homogeneous T cell product developed through CRISPR engineering, like NTLA-5001, may have over traditional T cell engineering approaches. In particular, traditional T cell engineering methods typically result in a T cell product that carries two different TCRs, one endogenous and one transferred, which can pair in various combinations of alpha and beta chains and form mixed TCRs with unknown specificities. Intellia researchers are sharing today that the precise replacement of the endogenous TCR with the transgenic TCR (tgTCR) resulted in T cells with improved tgTCR expression levels and in 95% of edited T cells carrying exclusively the desired pairs of the tgTCR alpha and beta chains. This therapeutic TCR profile is expected to yield improved T cell product homogeneity, as researchers showed that Intellias T cell editing approach results in superior function of the engineered T cells toward WT1-positive targets in vitro. This therapeutic TCR profile is also expected to result in lower reactivity against unwanted targets on normal tissues that could lead to toxicities, including graft-versus-host disease (GvHD).

Researchers identified that the selected lead WT1 TCR exhibits high avidity (in the nM range) to its target epitope and shows tight epitope specificity. Being a natural TCR isolated from a healthy donor, it may have a lower cross-reactivity risk than many affinity-matured TCRs. Cells engineered with Intellia's lead WT1 TCR also demonstrated no detectable cytotoxicity toward bone marrow CD34+ cells, which express WT1 at low levels. This is an advantage over current CAR-T cell approaches targeting CD33 or CD123 in AML, which have been shown to induce severe bone marrow toxicity.

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Furthermore, the data demonstrate that specific and potent killing of WT1-positive primary AML blasts result from T cells expressing Intellias lead WT1 TCR when cocultured in vitro. This outcome was observed across multiple patient samples that harbor the frequent HLA-A*02:01 allele and that express different WT1 levels as well as AML characteristics. These data validate that the epitope targeted by the lead WT1 TCR, which is distinct from a previously evaluated RMF epitope, is presented efficiently and broadly by AML tumor cells that carry the correct human leukocyte antigen (HLA) restriction. Intellias lead WT1 TCR also has the potential to target WT1-positive solid tumors, such as ovarian cancer, glioblastoma, lung cancer and mesothelioma.

The company plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the first half of 2021 for NTLA-5001 for the treatment of AML. Details on todays presentations on WT1 TCR T cells, including data from ongoing collaborations with researchers at IRCCS Ospedale San Raffaele, Milan, at Keystone are as follows:

First Data Presented on Potential CRISPR/Cas9-Based Therapy for HAE, Intellias Third Development Program

Researchers presented yesterday at the Keystone conference the companys first dataset in support of Intellias development program for HAE. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. The disease is caused by increased levels of the bradykinin protein. Most patients with HAE have a C1 esterase inhibitor (C1-INH) protein deficiency, which normally prevents the unregulated release and buildup of bradykinin.

Intellias HAE treatment hypothesis involves knocking out the kallikrein B1 (KLKB1) gene to reduce kallikrein activity, which is involved in the biological pathway for release of bradykinin. Intellia expects this reduction to correlate with a decrease in bradykinin activity, thus, preventing the activation of endothelial cells that causes vascular leakage and angioedema in HAE patients. The data presented at the Keystone conference showed that the knockout of KLKB1 produces in non-human primates (NHPs) a 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates (Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least five months in an ongoing NHP study, in a highly reproducible manner observed across both rodent and NHP studies.

Similar to its lead in vivo program, for the treatment of transthyretin amyloidosis (ATTR), Intellias potential HAE therapy utilizes the companys modular non-viral lipid nanoparticle (LNP) system to deliver CRISPR/Cas9. Intellias proprietary LNP-based delivery system includes two basic components: Cas9 messenger RNA (mRNA) and a guide RNA (gRNA). The gRNA is the only variable portion of the LNP delivery system and is the sole component that needs to be changed from the LNP-based delivery system that forms the foundation of NTLA-2001, Intellias development candidate for the treatment of ATTR for which the company intends to submit an IND application in mid-2020.

Intellia continues to evaluate several potential guide RNAs and expects to nominate a development candidate for HAE in the first half of 2020. Intellias KLKB1 HAE program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party.

Yesterdays short talk, titled In Vivo Delivery of CRISPR/Cas9 to the Liver Using Lipid Nanoparticles Enables Gene Knockout Across Multiple Targets in Rodent and Non-Human Primates, was made by Jessica Seitzer, director, genomics, Intellia. These data included results from ongoing collaborations with researchers at Regeneron.

All of Intellias presentations can be found here, on the Scientific Publications & Presentations page of Intellias website.

About Intellia Therapeutics

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

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020; its plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; its plans to nominate a development candidate for its hereditary angioedema (HAE) program in the first half of 2020; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; its presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML and HAE programs, in any future studies, including human clinical trials; its ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; its business plans and objectives for its preclinical studies and clinical trials, including the therapeutic potential and clinical benefits thereof, as well as the potential patient populations that may be addressed by its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc.(Regeneron) and Regenerons ability to enter into a Co/Co agreement for the HAE program; statements regarding the timing of regulatory filings for its development programs; its use of capital, including expenses, future accumulated deficit and other financial results during 2019 or in the future; and the ability to fund operations through the end of 2021.

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

Intellia Contacts:

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

Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917lynnea.olivarez@intelliatx.com

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

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Intellia Therapeutics Presents New Data From Its Engineered Cell Therapy and In Vivo Programs at Keystone Symposias Engineering the Genome Conference...

Biogen $BIIB just won one of the biggest fights its ever faced.

The big biotech beat Mylans challenge on the patents that guard their cash cow, Tecfidera, the multiple sclerosis drug that drove the companys comeback under George Scangos and sustains his successor Michel Vounatsos as they search for new drugs.

In the inter partes review ruling, the Patent Trial and Appeal Board or PTAB determined:Having considered all the evidence, petitioner has not demonstrated by a preponderance of the evidence the unpatentability of claims 1-20 of the 514 patent.The news, a closely watched catalyst that had analysts on high alert, immediately triggered a huge 29% spike in their share price. Tecfidera earned $3.3 billion in 2019, almost half its revenue for the year.

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Kymriah pioneer Carl June is back, with first US human evidence for CRISPR use in cancer - Endpoints News

A company developing means to edit genes at the single-letter level has raised nearly $200 million in its initial public offering.

Cambridge, Massachusetts-based Beam Therapeutics said Wednesday evening that it had priced its IPO at $180 million, or $17 per share. The company began trading Thursday on the Nasdaq under the ticker symbol BEAM.

The company had filed to go public in September, aiming at a $100 million IPO. J.P. Morgan, Jeffereies and Barclays acted as joint book runners.

Its last major financing before the IPO was a Series B funding round that it raised in March worth $135 million. The company launched with an $87 million Series A round in May 2018 after operating in stealth mode for a year.

Beams work focuses on base editing, a technology that enables editing of the genome letter by letter, as opposed to the cutting technique usually involved in CRISPR/Cas9 gene editing. Each letter of the genome corresponds with a specific nucleobase: A for adenine, G for guanine, T for thymine and C for cytosine. In other words, base editing would enable changing C to T, G to A and so forth. The company has a licensing agreement with the Massachusetts Institute of Technology and Harvard Universitys Broad Institute for co-founder and CRISPR/Cas9 pioneer Feng Zhangs RNA base-editing technology.

Its most recent presentation of data was in April 2019, when it showed preclinical data from its base editing platform at the American Society of Gene and Cell Therapys annual meeting in Washington. For the study, Beam used the base editor BE4 for multiplex base editing of engineered CAR-T cells, showing the ability to knock out expression of three cell surface targets TRAC, B2M and PD-1 in 95%, 95% and 88% of cells, respectively. That technique, the company said, may be able to create CAR-T cells with improved therapeutic properties.

Gene editing is a key technology used for the creation of CAR-T cells derived from donors rather than from patients own T cells, also known as allogeneic or off-the-shelf CAR-Ts, with both CRISPR/Cas9 and TALEN gene-editing technology being used. Without such modifications, off-the-shelf CAR-T cells would be rejected by the body.

Photo: Spencer Platt, Getty Images

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Beam Therapeutics raises $180M in IPO - MedCity News

MarketResearch. Biz provides in-depth market analyzes, including refined forecasts, growth factors, birds eye view of competitive landscape, and key market insights to help companies make strategic decisions. One of the report we provide is CRISPR and Cas Genes Market 2020 Analyzes Current Market Size and Upcoming 10 years Growth of this industry.

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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

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Segmentation on the basis of product:

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

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Global CRISPR and Cas Genes Market 2020 : An Incredibly Research Insight That Works For All - TheLoop21

By Michael Le Page

Vchal/Getty Images

CRISPR gene-edited immune cells have been injected into threepeople with advanced cancer without any serious side effects, the first trial of its kind in the US. It is also the first CRISPR cancer trial in the world to publish its findings, and the encouraging results will pave the way for many more trials.

Its an important milestone, says Waseem Qasim at theUCLGreat Ormond Street Institute of Child Health in the UK, who is carrying out a similar trial there.

The US trial was intended only to assess safety. The threeparticipants had tumours that hadnt responded to other treatments, and were given only one dose of gene-edited cells.

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Is it safe and feasible? says team member Edward Stadtmauer at the University of Pennsylvania. I think thats what we demonstrated.

Many cancers involving blood cells are now treated by removing immune cells from individuals, adding a gene that makes them target cancer cells and putting them back in the body.

But this treatment doesnt work for everyone, says Stadtmauer. And in some, it works at first but they later relapse.

The hope is that using gene editing to delete genes in addition to adding the targeting gene will make this approach even more effective. For instance, immune cells have a safety switch, called PD-1, that other cells can flip to say dont hurt me. Many cancers exploit this to avoid immune attacks.

In this trial, the team removed immune cells from three peoplewho hadtumours with the same protein on their surface. A virus was used to add a gene to make the immune cells target this protein.

Next, three genes, including PD-1, were deleted using CRISPR. After six weeks, the cells were put back in the individuals, where they survived for at least 9 months.

There were two big safety concerns. Firstly, CRISPR can cause unintended changes to genomes that could turn cells cancerous. Deleting three genes means cutting around each one in three spots in the genome, for instance, and the wrong ends can be joined up. This did happen in some cells, but there was no sign of any turning cancerous.

The other worry was that lingering traces of the CRISPR protein used for gene editing might trigger an immune reaction, since it is a bacterial protein. There was no sign of this.

The trial wont continue because its 2016 gene-editing technology is already outdated, says Carl June, also at the University of Pennsylvania. In particular, a new form of CRISPR called base editing can be used to inactivate genes without cutting DNA, which should reduce the cancer risk even further. We are very attracted to that, says June.

There are also many other ways to edit immune cells to make them more effective, says Stadtmauer. The possibilities are limitless based on our imagination and scientific focus.

In particular, Stadtmauer wants to create off-the-shelf cells that could be given to any patient, rather than modifying each patients own cells. This would speed up treatments and greatly reduce costs.

Qasims team has already saved lives in an ongoing trial at Great Ormond Street using off-the-shelf cells created by an older form of gene editing called TALEN. But these cells have to be given as part of a drastic treatment, which is followed by a bone marrow transplant that kills off the edited cells. Stadtmauer wants to create cells that can survive indefinitely in peoples bodies.

The risk of edited cells turning cancerous or starting to attack healthy cells would be higher if they survive longer. But it is also possible to add a self-destruct mechanism triggered by a specific drug to kill them off if necessary.

There have been a number of trials of CRISPR-edited immune cells for treating cancer in China, but no results have yet been published.

Journal reference: Science, DOI: 10.1126/science.aba7365

More on these topics:

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CRISPR cancer trial finds that gene-edited immune cells are safe - New Scientist News

Spurred by promising clinical results in an important trial, each of the three major CRISPR stocks had a great performance in the second half of 2019. Unfortunately, they didn't keep the momentum going in the first month of 2020.

Shares of Intellia Therapeutics (NASDAQ:NTLA) fell 18.8% in January, according to data provided by S&P Global Market Intelligence. That was followed by a 14.7% loss for shares of CRISPR Therapeutics (NASDAQ:CRSP) and a 10.7% tumble for shares of Editas Medicine (NASDAQ:EDIT).

While each has recovered some ground in the first week of February, this trio of pharma stocks is no stranger to volatility. Investors should probably expect that to continue as clinical programs advance in 2020.

Image source: Getty Images.

In November, CRISPR Therapeutics reported data for the first two individuals in the trial, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), treated with its lead drug candidate CTX001. Both enjoyed significant benefits in their standard of living, which investors interpreted as a sign that CRISPR gene editing might actually live up to the hype.

That fueled annual gains of 113% for CRISPR Therapeutics last year. While Editas Medicine and Intellia Therapeutics gained only 30% and 7%, respectively, each had been sitting at a year-to-date loss in October.

What relevance does that have for the tumbles taken in January? First, it's not unusual for stocks to regress to the mean. Stocks that are red hot eventually cool off, while those that tumble without good reason eventually recover some ground.

Second, and the more important consideration for investors, is that the early stage results for CTX001 mean relatively little for the industry's pipeline of CRISPR-based gene editing drug candidates.

Consider that CTX001 is an ex vivo tool. Researchers harvest bone marrow from patients, extract specific types of stem cells, and engineer those with CTX001. The engineered stem cells are then grown in the lab before being reinjected into the patient.

Many other CRISPR-based drug candidates are designed as in vivo tools. That means the gene editing payloads are designed to engineer a patient's DNA while inside the body. An in vivo approach is inherently more complex and will be more difficult to control compared to an ex vivo approach.

Put another way, investors cannot take the promising, early stage results from CTX001 and extrapolate it broadly across all first-generation CRISPR tools. Wall Street certainly isn't, if the correlation between technical approach and stock performance is any guide.

Consider that the two most advanced drug candidates from CRISPR Therapeutics rely on ex vivo engineering. By contrast, the lead drug candidate from Editas Medicine relies on in vivo methods.

The lead pipeline asset from Intellia Therapeutics is also an in vivo tool, though unlike the lead assets from its peers, it has yet to advance to clinical trials.

Investors should expect 2020 to be a busy year for these CRISPR stocks. CRISPR Therapeutics will have more clinical data from CTX001 and the first set of data for its lead oncology asset CTX110.

Similarly, Editas Medicine should have results for EDIT101 and progress additional assets, while Intellia Therapeutics is preparing to finally enter the clinic with NTLA-2001 in the second half of the year.

Investors cannot know if the next batch of results will be as rosy as the initial data for CTX001, but they can probably expect another year of volatile stock movements.

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Here's Why CRISPR Stocks Fell in January - The Motley Fool

The first targets of trials in humans are serious diseases we know are caused by a single gene, but as we understand more about combinations of genes increasing risk factors for other diseases, Crispr could be used more widely

BL PREMIUM

01 February 2020 - 06:17 Hannah Kuchler

Jennifer Doudna wears her responsibility lightly. The scientist who co-discovered Crispr (clustered regularly interspaced short palindromic repeats) does not appear to be weighed down by the burden of her creation: the revolutionary gene-editing technology that promises to empower humans to control our own genome. She waves and smiles as she bypasses the hostess stand at Gather, an organic, farm-to-table restaurant in Berkeley. Its the sort of place that wears its principles on its sleeve a fitting venue for a discussion of the ethical conundrums that Crispr has unleashed.

Doudna appears to have thrown on her blazer in a rush, squashing down one side of her shirt collar. Since news of her scientific breakthrough was published in 2012, she has learnt to toggle between the white coat of her lab work, building on that initial discovery, and her suited-and-booted role pushing politicians and lawmakers to contemplate the consequences of changing the human genome.

Continued here:
LUNCH WITH THE FT: Jennifer Doudna, scientist and gene editor at the heart of the Crispr maelstrom - Business Day

The recent performance of Fastly (NYSE:FSLY) stock in the market spoke loud and clear to investors as FSLY saw more than 1.51M shares in trading volumes in the last trading session, way higher than the average trading volume of 1.51M shares by far recorded in the movement of Fastly (FSLY). At the time the stock opened at the value of $22.60, making it a high for the given period, the value of the stock jumped by 1.43%. After the increase, FSLY touched a low price of $22.10, calling it a day with a closing price of $22.43, which means that the price of FSLY went 22.75 below the opening price on the mentioned day.

Given the most recent momentum in the market in the price movement of FSLY stock, some strong opinions on the matter of investing in the companys stock started to take shape, which is how analysts are predicting an estimated price of $26.11 for FSLY within consensus. The estimated price would demand a set of gains in total of 8.5%, which goes higher than the most recent closing price, indicating that the stock is in for bullish trends. Other indicators are hinting that the stock could reach an outstanding figure in the market share, which is currently set at 38.62M in the public float and 2.21B US dollars in market capitalization.

When it comes to the technical analysis of FSLY stock, there are more than several important indicators on the companys success in the market, one of those being the Relative Strength Indicator (RSI), which can show, just as Stochastic measures, what is going on with the value of the stock beneath the data. This value may also indicate that the stock will go sideways rather than up or down, also indicating that the price could stay where it is for quite some time. When it comes to Stochastic reading, FSLY stock are showing 19.29% in results, indicating that the stock is neither overbought or oversold at the moment, providing it with a neutral within Stochastic reading as well. Additionally, FSLY with the present state of 200 MA appear to be indicating bullish trends within the movement of the stock in the market. While other metrics within the technical analysis are due to provide an outline into the value of FSLY, the general sentiment in the market is inclined toward positive trends.

With the previous 100-day trading volume average of 964502 shares, CRISPR Therapeutics AG (CRSP) recorded a trading volume of 902880 shares, as the stock started the trading session at the value of $52.71, in the end touching the price of $53.48 after jumping by 1.46%.

CRSP stock seem to be going ahead the lowest price in the last 52 weeks with the latest change of 82.28%.Then price of CRSP also went backward in oppose to its average movements recorded in the previous 20 days. The price volatility of CRSP stock during the period of the last months recorded 4.09%, whilst it changed for the week, now showing 3.21% of volatility in the last seven days. The trading distance for this period is set at -6.90% and is presently away from its moving average by -14.88% in the last 50 days. During the period of the last 5 days, CRSP stock lost around -3.36% of its value, now recording a sink by 8.63% reaching an average $49.32 in the period of the last 200 days.During the period of the last 12 months, CRISPR Therapeutics AG (CRSP) dropped by -12.19%.

According to the Barcharts scale, the companys consensus rating fall to 4.27 from 4.40, showing an overall improvement during the course of a single month. Based on the latest results, analysts are suggesting that the target price for CRSP stock should be $53.48 per share in the course of the next 12 months. To achieve the target price as suggested by analysts, CRSP should have a spike by 0% in oppose to its present value in the market. Additionally, the current price showcases a discount of 48.58% when compared to the high consensus price target predicted by analysts.

CRSP shares recorded a trading volume of 857794 shares, compared to the volume of 1.21M shares before the last close, presented as its trading average. With the approaching 3.21% during the last seven days, the volatility of CRSP stock remained at 4.09%. During the last trading session, the lost value that CRSP stock recorded was set at the price of $53.48, while the lowest value in the last 52 weeks was set at $29.34. The recovery of the stock in the market has notably added 82.28% of gains since its low value, also recording -10.03% in the period of the last 1 month.

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Stocks That Are Finally Turning The Corner: Fastly (FSLY), CRISPR Therapeutics AG (CRSP) - US Post News

The UK must break away from Europes restrictive agricultural gene-editing rules, science writer Matt Ridley told the UKs House of Lords on January 30. A vocal advocate for technological innovation, Ridely argued that Britain should embrace CRISPR and other new breeding techniques, or risk falling behind enterprising nations, including the US, Canada and Argentina, that have recognized the technologys benefits.

While critics warn that gene-edited crops and animals could pose a risk to human health and the environment, Ridely countered that no data support such concerns and offered several examples of how Britain stands to gain by taking an evidence-based position on CRISPR:

There is no clearer case of a technology in which we could and should take the lead, but in which we are and will be held back if we do not break free from the EU approach. That would not be a race to the bottom but the very opposite: a race to the top.

For example, if we allowed the genome-edited blight-resistant potatoes developed at the Sainsbury Laboratory to be grown here in the UK, we would be able to greatly reduce the spraying of fungicides on potato fields, which happens up to 15 times a year, harming biodiversity and causing lots of emissions from tractors. That would be an improvement, not a regression, in environmental terms.

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Video: House of Lords member and science writer Matt Ridely urges UK to 'break free' of Europe's restrictive CRISPR crop rules - Genetic Literacy...

SEOUL, South Korea, Feb. 3, 2020 /PRNewswire/ --Scientists at Dongguk University successfully treated Alzheimer's disease in mice using the gene editing tool CRISPR-Cas9. They used the tool to edit out the Alzheimer's causing gene in the mice, thereby improving their memory and other cognitive functions. While this is only a first step and much research remains to be done, these promising results put gene therapy on the map of possible treatments for Alzheimer's.

As Alzheimer's disease continues to elude the efforts of modern medicine, scientists turn to gene editing technology to treat, or 'manage' it. Alzheimer's is commonly associated with the BACE 1 gene, which drives the production of amyloid-proteins in the brain. In the brains of Alzheimer's patients, this protein accumulates on the outsides of neurons or nerve cells as "plaque," and is thought to be one of the main causes of the disease. Using "DNA scissors" called CRISPR-Cas9, which can cut out a specified part of a DNA sequence, Dr. Jongpil Kim and his colleagues from Korea (Dongguk University) and the USA attempted to edit out the BACE 1 gene in the neurons of the adult mouse brain.

Theirs is the first in vivo attempt (using cells inside living mice) to test the effectiveness of CRISPR-Cas9 in stopping Alzheimer's progression. Their ultimate ambitionlike that of many others in the fieldis to develop treatments for different forms of dementia, which would dramatically improve patients' quality of life. "We aimed to see whether CRISPR-Cas9, one of the latest developments in biotechnology, can open up a new direction for treating dementia, which is, at present, considered an incurable disease," Dr. Kim says.

Their results indeed look promising. Within 8 to 12 weeks of treatment with the CRISPR-Cas9 technology, the plaque surrounding affected neurons in the mice dramatically decreased and their cognitive functions improved substantially. The mice also showed better associative learning and spatial working memory. Further, there was no evidence of increasing mutations in other, non-targeted parts of the genome, suggesting minimal side-effects.

All this indicates the huge potential of gene editing technologies to treat neurodegenerative diseases: by removing faulty genes at their source, disease progression can be halted, or even reversed, in just a few weeks.

The authors advise caution, however, as gene editing cannot be undone. In Dr. Kim's words, one must ensure that "no detrimental, and potentially very rare, genomic alterations are caused". Thus, this technique requires much more research before it can be applied to human subjects.

Nonetheless, as another remark by Dr. Kim goes, "this study shows how CRISPR-Cas9 can be applied to the treatment of neurodegenerative diseases as well."

Reference

Author:

Jongpil Kim

Title of original paper:

In vivo neuronal gene editing via CRISPRCas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease

Journal:

Nature Neuroscience

DOI:

10.1038/s41593-019-0352-0

Affiliations:

Departments of Chemistry and Biomedical Engineering, Dongguk University

Media contact:Jongpil Kim 233458@email4pr.com+82-10-4013-3685

SOURCE Dongguk University

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DNA "Scissors" Could Cut Out the Alzheimer's Causing Gene in Mice - PRNewswire