Archive for the ‘Crispr’ Category

Researchers are racing against time to find ways to treat and prevent COVID-19. There is currently no treatment for the disease, and the World Health Organisation has created Solidarity, a global clinical trial which is testing four drugs as possible treatment. There are also more than 90 vaccine trials being undertaken worldwide, but it may take more than a year before a vaccine is developed. And there is currently a global shortage of COVID-19 testing kits.

One of the methods researchers are exploring to combat COVID-19 is gene editing. Gene editing could potentially be used on the genome of the virus that causes COVID-19, to make it harmless. It could be used to develop better testing kits, and could even be used to edit the human genome to prevent people from being infected by the virus.

But gene editing is associated with a range of ethical issues such as safety, equal access and consent. Bioethicists and researchers believe that gene editing in humans must be proven to be safe before it can be offered as a treatment option. There is also the issue of equal access to treatment, which must be considered.

To ensure that an ethical approach to research for a cure for COVID-19 is taken, the International Bioethics Committee and the World Commission on the Ethics of Scientific Knowledge and Technology issued a joint statement calling for an interdisciplinary dialogue among scientific, ethical and political stakeholders. The joint statement does not describe specific treatment options, but it calls on the research community to work together to find a cure using a bioethics and ethics of science and technology perspective which is rooted in human rights.

Scientists have considered the possibility of CRISPR (or clustered regularly interspaced short palindromic repeats) technology being used to address the COVID-19 pandemic. CRISPR is a mechanism that arose in bacteria millions of years ago to fight off disease. The CRISPR protein can be used to target specific sequences of DNA, which it then cuts like a pair of scissors. The cut DNA strand can then repair itself, or a new DNA sequence can be inserted. It has now been turned into a biological tool for editing genomes of biological organisms in order to modify them or target disease. It therefore has a number of different uses, from improving crop quality to correcting genetic conditions.

There are three potential ways that CRISPR may help fight COVID-19:

CRISPR has the potential to disable the virus that causes COVID-19 by editing its genome so that it is, in effect, made harmless. Using an approach called PAC-MAN (Prophylactic Antiviral Crispr in huMAN cells), researchers at Stanford University have shown that CRISPR has the ability to attack the SARS-CoV-2 genetic makeup and reduce the amount of virus in a test solution by 90%. Research is ongoing, but its thought that this approach is so effective, it might have the potential to stop the disease in people. There would be no barrier to this research as long as researchers abide by the ethical and legal guidelines that apply to their institution and country.

Gene editing tools have the potential to improve testing rates and could be an answer to the global shortage of COVID-19 tests. Apart from being a gene editing tool, CRISPR is also a diagnostic tool, and can be used to detect infection in cells. Scientists are hopeful that CRISPR based testing will alleviate the global testing burden. While many of these tests are still in the development stage, the Food and Drug Administration approved a CRISPR-based COVID-19 diagnostic test by a Cambridge biotech start-up on 8 May 2020. The test can provide results within an hour, and the company making it claims that more than 1 million tests can be performed in a week. In order for these tests to be legally made available for use, they would need to be approved by the appropriate regulatory authority, such as the Food and Drug Administration in the US, or South Africas Health Products Regulatory Authority.

CRISPR creates the potential to edit peoples genes to make them resistant to infection. So, if we cant stop the virus, can we stop ourselves from getting infected? Gene editing in humans takes one of two forms: somatic cell editing and germline editing.

Somatic cell editing affects a persons body cells, while germline editing involves editing the DNA in sperm, eggs or embryos, resulting in genetic changes in an individuals descendants. There are a number of somatic cell CRISPR clinical trials being undertaken and some treatments have been successful. But germline editing is more controversial and over 40 countries prohibit it in their law.

When the Chinese scientist He Jiankui used CRISPR to edit the genomes of two children, he was criticised as acting unethically, since the safety and efficacy of germline editing has not been established. Scientists around the world called for a five-year moratorium on it. He Jiankui was sentenced to three years in prison in 2019.

There are also laws which will obstruct this potential use of CRISPR. Article 3 of the Oviedo Convention states that an intervention seeking to modify the human genome may only be undertaken for preventive, diagnostic, or therapeutic purposes and only if its aim is not to introduce any modification in the genome of any descendants. This has been interpreted as expressly forbidding germline gene editing.

National law can also prohibit this. Section 57 of South Africas National Health Act states that a person may not manipulate any genetic material, including genetic material of human gametes, zygotes or embryos. Section 57 is enacted to prohibit human reproductive cloning. But this law was enacted before CRISPR even existed and it mimics the approach taken in international law against genetic manipulation of gametes and embryos. There are licensed somatic cell CRISPR therapies available. But there are potential legal barriers to the lasting protection which germline CRISPR intervention would give us.

There is pressure on researchers to develop safe and effective treatment and vaccines. CRISPR technology has been used in a variety of ways, but it raises a series of ethical and legal issues with regard to its potential use in humans.

So far, scientists have been cautious about putting CRISPR technology to use in humans. But should CRISPR be considered as a legitimate weapon in the fight against the pandemic, knowing that time is of the essence? While we all act together in the fight against COVID-19, it cannot be at the expense of ethical and legal standards.

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COVID-19 and gene editing: ethical and legal considerations - The Conversation Africa

DUBLIN--(BUSINESS WIRE)--The "Global Genome Editing Market By Technique (CRISPR, Zinc Finger Nucleases, TALENs, Restriction enzymes, Others), By Applications (Synthetic Biology, Engineering Cell Line and Organisms, Others), By Source, By End-User, By Region, Forecast & Opportunities, 2025" report has been added to ResearchAndMarkets.com's offering.

The Global Genome Editing Market is expected to grow at a brisk rate during the forecast period owing to growing number of research activities for treatment of various chronic diseases using this technology. Further, increased government funding for genomics technology around the globe, growing preference for personalized medicine and increase in R&D expenditure are fueling the market growth of genome editing.

Genome editing is a way of making specific changes to the DNA of a cell or organism. It could be used to edit the genome of any organism. It uses a type of enzyme called an engineered nuclease' which cuts the genome in a specific place. After cutting the DNA in a specific place, the cell naturally repairs the cut. It finds application in large number of areas, such as mutation, therapeutics, and agriculture biotechnology. Moreover, rise in the number of chronic and infectious diseases is likely to fuel the market for genome editing in the coming years.

The Global Genome Editing market is segmented based on technique, applications, source, end-user and region. Based on applications, the market is segmented into synthetic biology, engineering cell line & organisms, therapeutic genome editing and others. Among them, the cell line engineering is expected to witness the highest growth rate in the coming years due to increase in the number of people suffering with genetic disorders and rising government funding for stem cell research.

Based on end-user, the Global Genome Editing Market is segmented into pharmaceutical & biotechnology companies, clinical research organization and research institutes. Pharmaceutical & biotechnology companies contribute to the largest share of revenue generation for the Global Genome Editing Market. Growing establishments of biotech and pharma companies in emerging economies and growing usage of gene editing technique in research activities undertaken by them to manufacture and develop drugs for rare diseases anticipated to fuel the market across the globe.

Companies Mentioned

Objective of the Study:

Key Topics Covered:

1. Product Overview

2. Research Methodology

3. Executive Summary

4. Global Genome Editing Market Outlook

4.1. Market Size & Forecast

4.2. Market Share & Forecast

4.3. Market Attractiveness Index

5. Asia-Pacific Genome Editing Market Outlook

5.1. Market Size & Forecast

5.2. Market Share & Forecast

5.3. Market Attractiveness Index

5.4. Asia-Pacific: Country Analysis

6. Europe Genome Editing Market Outlook

6.1. Market Size & Forecast

6.2. Market Share & Forecast

6.3. Market Attractiveness Index

6.4. Europe: Country Analysis

7. North America Genome Editing Market Outlook

7.1. Market Size & Forecast

7.2. Market Share & Forecast

7.3. Market Attractiveness Index

7.4. North America: Country Analysis

8. South America Genome Editing Market Outlook

8.1. Market Size & Forecast

8.2. Market Share & Forecast

8.3. Market Attractiveness Index

8.4. South America: Country Analysis

9. Middle East and Africa Genome Editing Market Outlook

9.1. Market Size & Forecast

9.2. Market Share & Forecast

9.3. Market Attractiveness Index

9.4. MEA: Country Analysis

10. Market Dynamics

10.1. Drivers

10.2. Challenges

11. Market Trends & Developments

12. Competitive Landscape

12.1. Competition Outlook

12.2. Players Profiled (Leading Companies)

13. Strategic Recommendations

14. About Us & Disclaimer

For more information about this report visit https://www.researchandmarkets.com/r/tgb83z

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Outlook on the Worldwide Genome Editing Industry to 2025 - Featuring Pfizer, Bayer Crop Science & Editas Medicine Among Others -...

Global CRISPR Technology Market 2020 offers detailed research and analysis of the COVID-19 impact provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global CRISPR Technology market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global CRISPR Technology market is carefully analyzed and researched about by the market analysts.

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Top Companies in the Global CRISPR Technology Market: Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT), Horizon Discovery Group, Agilent Technologies, Cellecta, GeneCopoeia, New England Biolabs, Origene Technologies, Synthego Corporation, Toolgen

CRISPR technology is a relatively new technology used in genome editing or gene editing; CRISPR-CAS-9 is a palindromic repeats cluster and is found naturally in bacteria. This sequence allows the bacteria to protect them from the virus by producing segments enzyme that cuts RNA or DNA virus and inactivate the virus. This ability of the CRISPR-CAS9 has allowed scientists to make DNA and RNA library as needed and their applications. CRISPR-CAS9 technology has potential applications in treating human disease, creating a gene library, and manipulate cell functions such as metabolism.

Market Segmented by Types:

Enzymes

Kits

gRNA

Libraries

Design Tools

Market Segmented by Applications:

Biomedical

Agricultural

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Regional Analysis:

For comprehensive understanding of market dynamics, the global CRISPR Technology Market is analyzed across key geographies namely: United States, China, Europe, Japan, South-east Asia, India and others. Each of these regions is analyzed on basis of market findings across major countries in these regions for a macro-level understanding of the market.

Important Features that are under Offering and Key Highlights of the Reports:

Detailed overview of Market

Changing market dynamics of the industry

In-depth market segmentation by Type, Application etc.

Historical, current and projected market size in terms of volume and value

Recent industry trends and developments

Competitive landscape of Market

Strategies of key players and product offerings

Potential and niche segments/regions exhibiting promising growth

Take a look at some of the important sections of the report:

Market Overview:It starts with product overview and scope of the global CRISPR Technology market and later gives consumption and production growth rate comparisons by application and product respectively. In addition, it provides statistics related to market size, revenue, and production.

Production Market Share by Region:Apart from the production share of regional markets analyzed in the report, readers are informed about their gross margin, price, revenue, and production growth rate here.

Company Profiles and Key Figures:Each company profiling of leading players operating in the market growth keeping in view vital factors markets served, production sites, price, gross margin, revenue, production, product application, product specification, production sites and product introduction.

Manufacturing Cost Analysis: Readers are provided with detailed manufacturing process analysis, industrial chain analysis, manufacturing cost structure analysis, and raw materials analysis.

Market Dynamics:The analysts explore critical influence factors, market drivers, challenges, risk factors, opportunities, and market trends.

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Finally, CRISPR Technology Market report is the believable source for gaining the Market research that will exponentially accelerate your business. The report gives the principle locale, economic situations with the item value, benefit, limit, generation, supply, request and Market development rate and figure and so on. This report additionally Present new task SWOT examination, speculation attainability investigation, and venture return investigation.

Note: All the reports that we list have been tracking the impact of COVID-19. Both upstream and downstream of the entire supply chain has been accounted for while doing this. Also, where possible, we will provide an additional COVID-19 update supplement/report to the report in Q3, please check for with the sales team.

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CRISPR Technology Market Technology Advancements, Overview and Developments in Medical Industry 2020 to 2021 - Cole of Duty

The global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market study encloses the projection size of the market both in terms of value (Mn/Bn US$) and volume (x units). With bottom-up and top-down approaches, the report predicts the viewpoint of various domestic vendors in the whole market and offers the market size of the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market. The analysts of the report have performed in-depth primary and secondary research to analyze the key players and their market share. Further, different trusted sources were roped in to gather numbers, subdivisions, revenue and shares.

The research study encompasses fundamental points of the global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market, from future prospects to the competitive scenario, extensively. The DROT and Porters Five Forces analyses provides a deep explanation of the factors affecting the growth of Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market. The Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market has been broken down into various segments, regions, end-uses and players to provide a clear picture of the present market situation to the readers. In addition, the macro- and microeconomic aspects are also included in the research.

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The key players covered in this studyThermo Fisher ScientificMerckGenScriptIntegrated DNA TechnologiesHorizon Discovery GroupAgilent TechnologiesCellectaGeneCopoeiaNew England BiolabsOrigene TechnologiesSynthego CorporationToolgen

Market segment by Type, the product can be split intoProductsServicesMarket segment by Application, split intoBiomedical ApplicationsAgricultural ApplicationsIndustrial ApplicationsBiological Research

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

The study objectives of this report are:To analyze global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology status, future forecast, growth opportunity, key market and key players.To present the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology 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 Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology 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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The Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market research covers an exhaustive analysis of the following data:

The Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market research addresses critical questions, such as

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The global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market research considers region 1 (Country 1, country 2), region 2 (Country 1, country 2) and region 3 (Country 1, country 2) as the important segments. All the recent trends, such as changing consumers demand, ecological conservation, and regulatory standards across different regions are covered in the report.

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COVID-19: Potential impact on Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market by Application Analysis 2019-2029 -...

Vertex Pharmaceuticals (VRTX) is the $75 billion champion of cystic fibrosis (CF) who is expected to grow sales 37% this year to $5.7 billion -- after a 37% topline advance last year.

Since 2012, Vertex has developed a suite of drug treatments for CF, including the "triple threat" combo Trikafta, which was approved by the FDA in October 2019.

Cystic fibrosis is a hereditary disease that affects the lungs and digestive system. The body produces thick and sticky mucus that can clog the lungs and obstruct the pancreas. CF can be life-threatening, and people with the condition tend to have a shorter-than-normal life span, with many adults not making it to their 30th birthday.

Vertexs lead marketed products are Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko/Symkevi (tezacaftor in combination with ivacaftor), Orkambi (lumacaftor in combination with ivacaftor) and Kalydeco (ivacaftor), which are collectively approved to treat around 60% of the 75,000 CF patients in North America, Europe and Australia.

Trikafta, approved in people aged 12 years and older who have at least one F508del mutation, is under review in Europe and is also being evaluated in younger patients in the United States. With approval of Trikafta, Vertex can address a significantly larger CF patient population almost 90% of patients with CF in the future.

Q1 Quarter and Outlook

Despite COVID-19 related uncertainty, Vertexs sales in 2020 are being driven by rapid uptake of Trikafta and higher international revenues due to reimbursement arrangements in key ex-U.S. countries. Trikaftas early approval and launch was a significant milestone for Vertex.

On April 29, Vertex reported Q1 results and beat estimates for earnings and sales. The 2020 outlook sparked analysts to raise EPS estimates significantly with this year getting boosted 15.8% from $7.60 to $8.80, representing 65% growth.

The company recorded total revenues of $4.16 billion in 2019, up 37%. Orkambi accounted for 29.4% of the companys total product revenues, Kalydeco accounted for 24.7%, Symdeko accounted for 35.4% and Trikafta comprised 10.5% of the same.

Vertexs dependence on the CF franchise for growth is a concern, especially as competitors would only erode market share. But Vertexs non-CF pipeline is progressing rapidly with data in multiple disease arenas expected in 2020: sickle cell disease, thalassemia and pain management.

Vertex + CRISPR = Potential Knockout Punch for Blood Disorders

Vertex is co-developing a gene editing treatment, CTX001 in partnership with CRISPR Therapeutics (CRSP) in two devastating diseases sickle cell disease and thalassemia. Phase I/II studies of CTX001 in adult transfusion-dependent b-thalassemia in Europe and sickle cell disease in the United States are ongoing.

In June 2019, Vertex announced expansion of its collaboration with CRISPR Therapeutics and acquisition of privately held Exonics Therapeutics to boost its gene editing capabilities to develop novel therapies for Duchenne muscular dystrophy (DMD) and Myotonic dystrophy type 1 (DM1).

In the April 29 update, Vertex and partner CRISPR Therapeutics said they remain on track to provide additional data from the two ongoing Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with transfusion-dependent beta thalassemia and in patients with severe sickle cell disease in 2020.

Bottom line for VRTX: The COVID-19 crisis has put the Biotech sector in the spotlight and dozens of companies are responding with resourceful R&D and robust adaptations to clinical trial interruptions. Vertex is a strong leader here and should be part of any growth-oriented healthcare-focused portfolio.

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Bull of the Day: Vertex Pharma (VRTX) - Yahoo Finance

More than three months into the coronavirus pandemic, widely acknowledged shortcomings with testing continue to hamper the nations recovery. So when a team of microbiologists headed by Dr. Feng Zhang of the McGovern Institute at MIT and the Broad Institute reported this month that they had ironed out a protocol for a simple, cheap, point-of-care test that uses CRISPR to detect the virus, it was hailed as one of the most important contributions to fighting COVID-19.

Efforts to build and scale up a diagnostic have been beset by a number of snags, from a scarcity of chemicals called reagents and equipment to slow return of results. As officials debate how to safely reopen the country, those weaknesses would need to be rectified in a way where relaxing stay-at-home orders doesnt set off a viral rebound.

According to the Harvard Global Health Institute, in order to safely reopen the country and keep it open, we must ramp our testing rate from about a million per week to a million per day. But that remains a stretch by conventional means.

Enter CRISPR, the precision genome-editing technique that is anything but conventional.

Dr. Zhang and his colleagues harnessed a new type of CRISPR to build a test able to rapidly detect as few as 100 coronavirus particles in a swab or saliva sample, according to instructions for the new test, called STOPCovid, that they posted online. The teams focus has now shifted to proving that the test is safe and effective on a mass scale.

Like any diagnostic chemistry, we need to demonstrate that STOPCovid is accurate on a large enough cohort of patient samples to provide benefit, wrote Drs. Jonathan Gootenberg and Omar Abudayyeh, both of the McGovern Institute, in response to emailed questions. In addition, the current pandemic has made clear the need to scale to thousands or millions of tests, and solutions for that are necessary as well.

As to when the new diagnostic may be pressed into service against the novel coronavirus, Its hard to predict what the timeframe would be for a point-of-care or at-home test, the two noted. But given the need for these diagnostics, we would hope at latest by the end of the year.

That timeframe is possible because a prototype for a quick, easy, cheap and precise CRISPR-based diagnostic test had already existed. In 2017 Dr. Zhang and bioengineer Dr. James Collins published research showing that CRISPR could be trained to detect extremely low amounts of genetic material and was suitable for use during disease outbreaks. They dubbed this system Sherlock, for specific high-sensitivity enzymatic reporter unlocking.

Its from that earlier prototype that the new SARS-CoV-2 test takes its cue. STOP stands for Sherlock Testing in One Pot think multiple steps reduced to a single reaction in a tube. Its similar to DNA-targeting CRISPRs, like the well-known Cas9 system for DNA-editing of the human genome. Only this system has been reformulated to target strands of RNA, which are the building blocks of viruses.

Once a guide RNA molecule brings the Cas12 enzymes to the area of interest, the enzymes cut it in such a way that it generates a fluorescent readout. That readout is detectable, in much the same way home pregnancy tests pick up on pregnancy-related hormones.

Broadscale testing of the type afforded by STOPCovid would be integral to reopening the economy again, said Dr. Neville Sanjana, a genome engineer who was not involved in the CRISPR diagnostic research but whose lab, based at the New York Genome Center and NYU, is involved in several efforts using CRISPR, both COVID-related and non.

The CRISPR-based diagnostics do present a path forward not just to scaling up but to longitudinal sampling, continual sampling, said Dr. Sanjana, who, in addition to being a faculty member at the New York Genome Center, is also assistant professor of biology at New York University and of neuroscience and physiology at the NYU School of Medicine. Were only going to feel comfortable going back to work if not only do you know that your coworker was Covid-negative yesterday, but you know your coworker was Covid-negative last week and Covid-negative today if you have as many assurances as possible.

To fully appreciate why a CRISPR-based test might be a good fit in this situation requires a comparison to the standard COVID-19 test, which is based on a technology called qPCR. The qPCR tests work by detecting nucleic acids, like RNA, and amplifying them. In that sense, theyre similar to CRISPR-based tests.

However, thats where the similarities end.

The qPCR tests require a specialized piece of equipment combining a thermocycler (a machine that automatically cycles through multiple temperature changes) and a microscope. These machines are typically only found in labs and normally cost a few thousand dollars apiece. The other big limitation of qPCR is that the reagents needed to perform the test require cold storage.

CRISPR-based diagnostics, on the other hand, can assay the presence or absence of a nucleic acid at just one temperature. In addition, its been demonstrated also by Dr. Zhang and colleagues that its possible to freeze-dry CRISPR enzymes, obviating the need for cumbersome cold storage.

You dont need the microbiology lab. Instead, you maybe need a stove, said Sanjana.

One of the unique aspects of CRISPR is that it enables sensitive detection without requiring fancy equipment or refrigerated storage.

Reading the results is relatively simple, too. Whereas qPCR and PCR-based test results must be read out either using a microscope or something called agarose gel electrophoresis another lab mainstay the CRISPR-based assay for SARS-CoV-2 uses a lateral flow strip for readout, akin to a home pregnancy test.

A test strip is placed in a tube, and the presence of two lines indicates SARS-CoV-2. Results come in about an hour, the researchers say, with no special handling needed. A mobile phone app can analyze images captured by the phone camera to readout test results, they noted in their white paper.

That [kind of straightforward readout] would be an enabling technology, said Sanjana, even in places located close to a traditional lab.

Whats more, STOPCovid tested on a nasopharyngeal swab bested sensitivity and specificity rates of qPCR, according to Zhangs team (its been shown to work in saliva, too). His group has prepared reagents for 10,000 tests to make freely available to other researchers who want to evaluate its diagnostic use.

At least two other research groups are studying CRISPR technologys promise in diagnosing coronavirus. The FDA was suitably impressed with the test coming out of the Broad group, which is being commercialized through a company aptly named Sherlock, that the agency handed it an emergency use authorization this month. Meanwhile, Zhang is reportedly talking to would-be commercial partners about designing a device based on a disposable, single-use test cartridge, similar to a pregnancy test.

Thats the kind of thing we want, said Dr. Sanjana. If I had to think about what would be the ideal test, it would be something that would be dirt cheap, you could do it every single day, you could test yourself and your whole family, and youd get results instantly or close to instantly. This is what we need to go back to work safely.

Not to say that the CRISPR-based testing is the end-all-be-all, he added, but I think it can fill a very unique niche, even in places where you might have access to what you need to do qPCR-based tests. But the convenience would be greater and thus the adoption would be better.

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A CRISPR-based, at-home COVID-19 test is set to arrive this year. Here's what you need to know - News - MM&M - Medical Marketing and Media

The recent outbreak of the COVID-19 (Coronavirus) pandemic has built and broken many value-grab opportunities for companies in the CRISPR and Cas Genes market. Gain full access on our latest analysis about COVID-19 and how companies in the CRISPR and Cas Genes market are capitalizing on new strategies to maintain stable revenue income. Look into our resourceful insights highlighting the impact of COVID-19 caused on the global market landscape.

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The report on the global CRISPR and Cas Genes market published by Market Research Reports Search Engine(MRRSE) provides a clear understanding of the flight of the CRISPR and Cas Genes market over the forecast period (20XX-20XX). The study introspects the various factors that are tipped to influence the growth of the CRISPR and Cas Genes market in the upcoming years. The current trends, growth opportunities, restraints, and major challenges faced by market players in the CRISPR and Cas Genes market are analyzed in the report.

The study reveals that the global CRISPR and Cas Genes market is projected to reach a market value of ~US$XX by the end of 20XX and grow at a CAGR of ~XX% during the assessment period. Further, a qualitative and quantitative analysis of the CRISPR and Cas Genes market based on data collected from various credible sources in the market value chain is included in the report along with relevant tables, graphs, and figures.

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Key Takeaways of the Report:

CRISPR and Cas Genes Market Segmentation

By Region

The presented study throws light on the current and future prospects of the CRISPR and Cas Genes market in various geographies such as:

By Product Type

The report highlights the product adoption pattern of various products in the CRISPR and Cas Genes market and provides intricate insights such as the consumption volume, supply-demand ratio, and pricing models of the following products:

Companies Mentioned in the Report

The report also profiles the major players in the market in terms of various attributes such as company overview, financial overview, product portfolio, business strategies, and recent developments. Key players operating in the global CRISPR and Cas genes market include Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell Inc. These players are adopting organic and in-organic growth strategies to expand product offerings, strengthen geographical reach, increase customer base, and market share.

The global CRISPR and Cas genes market has been segmented as follows:

Global CRISPR and Cas Genes Market, by Product

Global CRISPR and Cas Genes Market, by Application

Global CRISPR and Cas Genes Market, by End-user

Global CRISPR and Cas Genes Market, by Region

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The report addresses the following doubts related to the CRISPR and Cas Genes market:

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The impact of the coronavirus on the CRISPR and Cas Genes Market 2020:Key Insights, Drivers and Restraints, Opportunities and Challenges, Sales and...

After William Blair and Piper Sandler gave Crispr Therapeutics AG (NASDAQ: CRSP) a Buy rating last month, the company received another Buy, this time from Oppenheimer. Analyst Jay Olson maintained a Buy rating on Crispr Therapeutics AG yesterday and set a price target of $80.00. The companys shares closed last Friday at $61.09.

According to TipRanks.com, Olson is currently ranked with 0 stars on a 0-5 stars ranking scale, with an average return of -7.8% and a 39.0% success rate. Olson covers the Healthcare sector, focusing on stocks such as Madrigal Pharmaceuticals, ACADIA Pharmaceuticals, and Enanta Pharmaceuticals.

Currently, the analyst consensus on Crispr Therapeutics AG is a Moderate Buy with an average price target of $71.83, a 28.8% upside from current levels. In a report issued on May 6, Chardan Capital also reiterated a Buy rating on the stock.

See todays analyst top recommended stocks >>

The company has a one-year high of $74.00 and a one-year low of $32.30. Currently, Crispr Therapeutics AG has an average volume of 967.6K.

TipRanks has tracked 36,000 company insiders and found that a few of them are better than others when it comes to timing their transactions. See which 3 stocks are most likely to make moves following their insider activities.

CRISPR Therapeutics AG engages in the development and commercialization of therapies derived from genome-editing technology. Its proprietary platform CRISPR/Cas9-based therapeutics allows for precise and directed changes to genomic DNA. The company was founded by Rodger Novak, Emmanuelle Charpentier, Shaun Patrick Foy, Matthew Porteus, Daniel Anderson, Chad Cowan and Craig Mellow in 2014 and is headquartered in Zug, Switzerland.

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Crispr Therapeutics AG (CRSP) Received its Third Buy in a Row - Smarter Analyst

A new business intelligence report released by CMI with title Global CRISPR and CAS Gene Market Research Report 2020-2027 is designed covering micro level of analysis by manufacturers and key business segments. The Global CRISPR and CAS Gene Market survey analysis offers energetic visions to conclude and study market size, market hopes, and competitive surroundings. The research is derived through primary and secondary statistics sources and it comprises both qualitative and quantitative detailing. Some of the key players profiled in the study are Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Whats keeping Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc. Ahead in the Market? Benchmark yourself with the strategic moves and findings recently released by CMI

Get Summery Of this Report:https://www.coherentmarketinsights.com/market-insight/crispr-and-cas-gene-market-2598

Market Overview of Global CRISPR and CAS Gene

If you are involved in the Global CRISPR and CAS Gene industry or aim to be, then this study will provide you inclusive point of view. Its vital you keep your market knowledge up to date segmented by Applications and major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports we can provide customization according to your requirement.

This study mainly helps understand which market segments or Region or Country they should focus in coming years to channelize their efforts and investments to maximize growth and profitability. The report presents the market competitive landscape and a consistent in depth analysis of the major vendor/key players in the market.

Detailed Segmentation:

By Product Type:Vector-based CasDNA-free CasGlobal CRISPR and CAS Gene Market, By Application:Genome EngineeringDisease modelsFunctional GenomicsKnockdown/activationOther Applications

Regions included:

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

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

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

o South America (Brazil, Argentina, Colombia)

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

Key Benefits:

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

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

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

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

The PDF Research only provides Table of Contents (ToC), scope of the report and research framework of the report.

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The research study can answer the following Key questions:

Table of Contents

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

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

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

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

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Note- This report sample includes:

Brief Introduction to the research report.

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Top players in the market

Research framework (presentation)

Research methodology adopted by CMI

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An Overview of the Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

National governments and international bodies are focused on adopting collaborative efforts to encourage financial institutions to meet the financial needs of customers and members affected by the coronavirus. However, there are some sectors that have remained unscathed from the impact of the pandemic and there are some that are hit the hardest.

Reasons to Purchase CRISPR and CAS Gene report is:

Gives a complete understanding of the CRISPR and CAS Gene Market to express competitor information, analysis, and insights to formulate effective RD strategies.

Collect data of the developing participants having the potentially profitable portfolio in this space and create effective counter-strategies to gain competitive benefits.

Note: Access insightful study with over 150+ pages, list of tables & figures, profiling 20+ companies.

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Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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CRISPR and CAS Gene Market to Witness Huge Growth by 2027 | Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio...

The shares of CRISPR Therapeutics AG (NASDAQ:CRSP) has been pegged with a rating of Hold by Stifel in its latest research note that was published on March 05, 2020. The Healthcare company has also assigned a $52 price target. Stifel wasnt the only research firm that published a report of CRISPR Therapeutics AG, with other equities research analysts also giving their opinion on the stock. Evercore ISI advised investors in its research note published on February 03, 2020, to In-line the CRSP stock while also putting a $52 price target. The stock had earned Outperform rating from William Blair Markets when it published its report on November 19, 2019. The stock was given Outperform rating by Oppenheimer in its report released on November 12, 2019, the day when the price target on the stock was placed at 65. Jefferies was of a view that CRSP is Buy in its latest report on August 01, 2019. Canaccord Genuity thinks that CRSP is worth Buy rating. This was contained in the firms report on July 26, 2019 in which the stocks price target was also moved to 72.

Amongst the analysts that rated the stock, 2 have recommended investors to sell it, 4 believe it has the potential for further growth, thus rating it as Hold while 11 advised investors to purchase the stock. The price of the stock the last time has raised by 89.13% from its 52-Week high price while it is -17.45% than its 52-Week low price. A look at the stocks other technical shows that its 14-day RSI now stands at 66.89.

The shares of the company added by 9.32% during the trading session on Friday, reaching a low of $54.86 while ending the day at $61.09. During the trading session, a total of 1.03 million shares were traded which represents a -6.22% decline from the average session volume which is 0.97 million shares. CRSP had ended its last session trading at $55.88. CRISPR Therapeutics AG debt-to-equity ratio currently stands at 0.00, while its quick ratio hovers at 16.90 CRSP 52-week low price stands at $32.30 while its 52-week high price is $74.00.

The company in its last quarterly report recorded -$1.15 earnings per share which is below the predicted by most analysts. The CRISPR Therapeutics AG generated 889.71 million in revenue during the last quarter. In the second quarter last year, the firm recorded $0.51 earnings per share. Compared to the same quarter last year, the firms revenue was up by 12.17%. CRISPR Therapeutics AG has the potential to record -4.51 EPS for the current fiscal year, according to equities analysts.

Investment analysts at BofA/Merrill published a research note on May 15, 2020 where it informed investors and clients that Canada Goose Holdings Inc. (NYSE:GOOS) is now rated as Underperform. Even though the stock has been trading at $20.70/share, analysts expect it to down by -7.15% to reach $32.90/share. It started the day trading at $19.74 and traded between $18.52 and $19.22 throughout the trading session.

A look at its technical shows that GOOSs 50-day SMA is 20.58 while its 200-day SMA stands at 33.10. The stock has a high of $51.71 for the year while the low is $12.94. The stock, however, witnessed a rise in its short on 04/30/20. Compared to previous close which recorded 10.25 M shorted shares, the short percentage went lower by -6.05%, as 9.63M CRSP shares were shorted. At the moment, only 16.46% of Canada Goose Holdings Inc. shares were sold short. The companys P/E ratio currently sits at 28.14, while the P/B ratio is 9.99. The companys average trading volume currently stands at 2.23M shares, which means that the short-interest ratio is just 4.32 days. Over the past seven days, the company moved, with its shift of -15.74%. Looking further, the stock has dropped -38.00% over the past 90 days while it lost -44.79% over the last six months.

Morgan Stanley Asia Ltd. (Investm meanwhile bought more GOOS shares in the recently filed quarter, changing its stake to $162,992,259 worth of shares.

Following these latest developments, around 0.71% of Canada Goose Holdings Inc. stocks are owned by institutional investors and hedge funds.

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Mixed Analyst Attention: CRISPR Therapeutics AG (NASDAQ:CRSP), Canada Goose Holdings Inc. (NYSE:GOOS) - State Reviewer

ZUG, Switzerland and CAMBRIDGE, Mass., May 15, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that four abstracts have been accepted for poster presentation at the American Association for Cancer Research (AACR) Virtual Annual Meeting II, which will take place from June 22 to 24, 2020.

Session information is available online via the Annual Meeting Itinerary Planner through the AACR website at http://www.aacr.org.

Title: Functional and single-cell assessment of CRISPR-modified CAR-T cells from NSCLC patients and healthy donors Session Title: Adoptive Cell Therapy 1 E-Poster Number: 879 Abstract Number: 3338

Title: Allogeneic CAR-T cell products containing 10 gene edits using CRISPR/Cas9 can retain full functionality in vivo and in vitro Session Title: Adoptive Cell Therapy 1 E-Poster Number: 880 Abstract Number: 4647

Title: Allogeneic anti-PTK7 CAR-T cells for the treatment of solid tumors Session Title: Adoptive Cell Therapy 3 E-Poster Number: 3243 Abstract Number: 6231

Title: Targeting T cell lymphomas with CRISPR/Cas9-generated anti-CD70 allogeneic CAR-T cells Session Title: Adoptive Cell Therapy 5 E-Poster Number: 6595 Abstract Number: 3308

About CRISPR Therapeutics CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic 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 Investor Contact: Susan Kim +1 617-307-7503 susan.kim@crisprtx.com

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

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CRISPR Therapeutics Announces Presentations at the American Association for Cancer Research 2020 Annual Meeting - Stockhouse

Los Angeles, United StatesThe report offers an all-inclusive and accurate research study on the global CRISPR And CRISPR-Associated (Cas) Genes market while chiefly focusing on current and historical market scenarios. Stakeholders, market players, investors, and other market participants can significantly benefit from the thorough market analysis provided in the report. The authors of the report have compiled a detailed study on crucial market dynamics, including growth drivers, restraints, and opportunities. This study will help market participants to get a good understanding of future development of the global CRISPR And CRISPR-Associated (Cas) Genes market. The report also focuses on market taxonomy, regional analysis, opportunity assessment, and vendor analysis to help with comprehensive evaluation of the global CRISPR And CRISPR-Associated (Cas) Genes market.

Key companies operating in the global CRISPR And CRISPR-Associated (Cas) Genes market include : , Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, GE Healthcare Dharmacon CRISPR And CRISPR-Associated (Cas) Genes

Get PDF Sample Copy of the Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) :

https://www.qyresearch.com/sample-form/form/1704011/covid-19-impact-on-global-crispr-and-crispr-associated-cas-genes-market

Segment Analysis

The segmental analysis will help companies to focus on high-growth areas of the global CRISPR And CRISPR-Associated (Cas) Genes market. In order to broaden the overall understanding of the global CRISPR And CRISPR-Associated (Cas) Genes industry, the report has segregated the global CRISPR And CRISPR-Associated (Cas) Genes business into varied segments comprising product type, application, and end user. This examination has been carried out based on parameters like size, CAGR, share, production, and consumption. Also, region-wise assessment, wherein lucrative prospects that a region or country is likely to offer has been explored.

Global CRISPR And CRISPR-Associated (Cas) Genes Market Segment By Type:

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

Global CRISPR And CRISPR-Associated (Cas) Genes Market Segment By Application:

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

Competitive Landscape:

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

Key companies operating in the global CRISPR And CRISPR-Associated (Cas) Genes market include : , Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, GE Healthcare Dharmacon CRISPR And CRISPR-Associated (Cas) Genes

Key Questions Answered

Enquire for Customization in this Report: https://www.qyresearch.com/customize-request/form/1704011/covid-19-impact-on-global-crispr-and-crispr-associated-cas-genes-market

Table of Contents

1 Study Coverage1.1 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction1.2 Market Segments1.3 Key CRISPR And CRISPR-Associated (Cas) Genes Manufacturers Covered: Ranking by Revenue1.4 Market by Type1.4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size Growth Rate by Type1.4.2 Genome Editing1.4.3 Genetic engineering1.4.4 gRNA Database/Gene Librar1.4.5 CRISPR Plasmid1.4.6 Human Stem Cells1.4.7 Genetically Modified Organisms/Crops1.4.8 Cell Line Engineering1.5 Market by Application1.5.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size Growth Rate by Application1.5.2 Biotechnology Companies1.5.3 Pharmaceutical Companies1.5.4 Academic Institutes1.5.5 Research and Development Institutes1.6 Coronavirus Disease 2019 (Covid-19): CRISPR And CRISPR-Associated (Cas) Genes Industry Impact1.6.1 How the Covid-19 is Affecting the CRISPR And CRISPR-Associated (Cas) Genes Industry

1.6.1.1 CRISPR And CRISPR-Associated (Cas) Genes Business Impact Assessment Covid-19

1.6.1.2 Supply Chain Challenges

1.6.1.3 COVID-19s Impact On Crude Oil and Refined Products1.6.2 Market Trends and CRISPR And CRISPR-Associated (Cas) Genes Potential Opportunities in the COVID-19 Landscape1.6.3 Measures / Proposal against Covid-19

1.6.3.1 Government Measures to Combat Covid-19 Impact

1.6.3.2 Proposal for CRISPR And CRISPR-Associated (Cas) Genes Players to Combat Covid-19 Impact1.7 Study Objectives1.8 Years Considered 2 Executive Summary2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size Estimates and Forecasts2.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue 2015-20262.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales 2015-20262.2 CRISPR And CRISPR-Associated (Cas) Genes Market Size by Region: 2020 Versus 20262.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Retrospective Market Scenario in Sales by Region: 2015-20202.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Retrospective Market Scenario in Revenue by Region: 2015-2020 3 Global CRISPR And CRISPR-Associated (Cas) Genes Competitor Landscape by Players3.1 CRISPR And CRISPR-Associated (Cas) Genes Sales by Manufacturers3.1.1 CRISPR And CRISPR-Associated (Cas) Genes Sales by Manufacturers (2015-2020)3.1.2 CRISPR And CRISPR-Associated (Cas) Genes Sales Market Share by Manufacturers (2015-2020)3.2 CRISPR And CRISPR-Associated (Cas) Genes Revenue by Manufacturers3.2.1 CRISPR And CRISPR-Associated (Cas) Genes Revenue by Manufacturers (2015-2020)3.2.2 CRISPR And CRISPR-Associated (Cas) Genes Revenue Share by Manufacturers (2015-2020)3.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Market Concentration Ratio (CR5 and HHI) (2015-2020)3.2.4 Global Top 10 and Top 5 Companies by CRISPR And CRISPR-Associated (Cas) Genes Revenue in 20193.2.5 Global CRISPR And CRISPR-Associated (Cas) Genes Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.3 CRISPR And CRISPR-Associated (Cas) Genes Price by Manufacturers3.4 CRISPR And CRISPR-Associated (Cas) Genes Manufacturing Base Distribution, Product Types3.4.1 CRISPR And CRISPR-Associated (Cas) Genes Manufacturers Manufacturing Base Distribution, Headquarters3.4.2 Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Product Type3.4.3 Date of International Manufacturers Enter into CRISPR And CRISPR-Associated (Cas) Genes Market3.5 Manufacturers Mergers & Acquisitions, Expansion Plans 4 Breakdown Data by Type (2015-2026)4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size by Type (2015-2020)4.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales by Type (2015-2020)4.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue by Type (2015-2020)4.1.3 CRISPR And CRISPR-Associated (Cas) Genes Average Selling Price (ASP) by Type (2015-2026)4.2 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast by Type (2021-2026)4.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast by Type (2021-2026)4.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast by Type (2021-2026)4.2.3 CRISPR And CRISPR-Associated (Cas) Genes Average Selling Price (ASP) Forecast by Type (2021-2026)4.3 Global CRISPR And CRISPR-Associated (Cas) Genes Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End 5 Breakdown Data by Application (2015-2026)5.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size by Application (2015-2020)5.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales by Application (2015-2020)5.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue by Application (2015-2020)5.1.3 CRISPR And CRISPR-Associated (Cas) Genes Price by Application (2015-2020)5.2 CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast by Application (2021-2026)5.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast by Application (2021-2026)5.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast by Application (2021-2026)5.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Price Forecast by Application (2021-2026) 6 North America6.1 North America CRISPR And CRISPR-Associated (Cas) Genes by Country6.1.1 North America CRISPR And CRISPR-Associated (Cas) Genes Sales by Country6.1.2 North America CRISPR And CRISPR-Associated (Cas) Genes Revenue by Country6.1.3 U.S.6.1.4 Canada6.2 North America CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Type6.3 North America CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Application 7 Europe7.1 Europe CRISPR And CRISPR-Associated (Cas) Genes by Country7.1.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Sales by Country7.1.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Revenue by Country7.1.3 Germany7.1.4 France7.1.5 U.K.7.1.6 Italy7.1.7 Russia7.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Type7.3 Europe CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Application 8 Asia Pacific8.1 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes by Region8.1.1 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Sales by Region8.1.2 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Revenue by Region8.1.3 China8.1.4 Japan8.1.5 South Korea8.1.6 India8.1.7 Australia8.1.8 Taiwan8.1.9 Indonesia8.1.10 Thailand8.1.11 Malaysia8.1.12 Philippines8.1.13 Vietnam8.2 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Type8.3 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Application 9 Latin America9.1 Latin America CRISPR And CRISPR-Associated (Cas) Genes by Country9.1.1 Latin America CRISPR And CRISPR-Associated (Cas) Genes Sales by Country9.1.2 Latin America CRISPR And CRISPR-Associated (Cas) Genes Revenue by Country9.1.3 Mexico9.1.4 Brazil9.1.5 Argentina9.2 Central & South America CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Type9.3 Central & South America CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Application 10 Middle East and Africa10.1 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes by Country10.1.1 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Sales by Country10.1.2 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Revenue by Country10.1.3 Turkey10.1.4 Saudi Arabia10.1.5 UAE10.2 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Type10.3 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Market Facts & Figures by Application 11 Company Profiles11.1 Caribou Biosciences11.1.1 Caribou Biosciences Corporation Information11.1.2 Caribou Biosciences Description, Business Overview and Total Revenue11.1.3 Caribou Biosciences Sales, Revenue and Gross Margin (2015-2020)11.1.4 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.1.5 Caribou Biosciences Recent Development11.2 Addgene11.2.1 Addgene Corporation Information11.2.2 Addgene Description, Business Overview and Total Revenue11.2.3 Addgene Sales, Revenue and Gross Margin (2015-2020)11.2.4 Addgene CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.2.5 Addgene Recent Development11.3 CRISPR THERAPEUTICS11.3.1 CRISPR THERAPEUTICS Corporation Information11.3.2 CRISPR THERAPEUTICS Description, Business Overview and Total Revenue11.3.3 CRISPR THERAPEUTICS Sales, Revenue and Gross Margin (2015-2020)11.3.4 CRISPR THERAPEUTICS CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.3.5 CRISPR THERAPEUTICS Recent Development11.4 Merck KGaA11.4.1 Merck KGaA Corporation Information11.4.2 Merck KGaA Description, Business Overview and Total Revenue11.4.3 Merck KGaA Sales, Revenue and Gross Margin (2015-2020)11.4.4 Merck KGaA CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.4.5 Merck KGaA Recent Development11.5 Mirus Bio LLC11.5.1 Mirus Bio LLC Corporation Information11.5.2 Mirus Bio LLC Description, Business Overview and Total Revenue11.5.3 Mirus Bio LLC Sales, Revenue and Gross Margin (2015-2020)11.5.4 Mirus Bio LLC CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.5.5 Mirus Bio LLC Recent Development11.6 Editas Medicine11.6.1 Editas Medicine Corporation Information11.6.2 Editas Medicine Description, Business Overview and Total Revenue11.6.3 Editas Medicine Sales, Revenue and Gross Margin (2015-2020)11.6.4 Editas Medicine CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.6.5 Editas Medicine Recent Development11.7 Takara Bio USA11.7.1 Takara Bio USA Corporation Information11.7.2 Takara Bio USA Description, Business Overview and Total Revenue11.7.3 Takara Bio USA Sales, Revenue and Gross Margin (2015-2020)11.7.4 Takara Bio USA CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.7.5 Takara Bio USA Recent Development11.8 Thermo Fisher Scientific11.8.1 Thermo Fisher Scientific Corporation Information11.8.2 Thermo Fisher Scientific Description, Business Overview and Total Revenue11.8.3 Thermo Fisher Scientific Sales, Revenue and Gross Margin (2015-2020)11.8.4 Thermo Fisher Scientific CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.8.5 Thermo Fisher Scientific Recent Development11.9 Horizon Discovery Group11.9.1 Horizon Discovery Group Corporation Information11.9.2 Horizon Discovery Group Description, Business Overview and Total Revenue11.9.3 Horizon Discovery Group Sales, Revenue and Gross Margin (2015-2020)11.9.4 Horizon Discovery Group CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.9.5 Horizon Discovery Group Recent Development11.10 Intellia Therapeutics11.10.1 Intellia Therapeutics Corporation Information11.10.2 Intellia Therapeutics Description, Business Overview and Total Revenue11.10.3 Intellia Therapeutics Sales, Revenue and Gross Margin (2015-2020)11.10.4 Intellia Therapeutics CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.10.5 Intellia Therapeutics Recent Development11.1 Caribou Biosciences11.1.1 Caribou Biosciences Corporation Information11.1.2 Caribou Biosciences Description, Business Overview and Total Revenue11.1.3 Caribou Biosciences Sales, Revenue and Gross Margin (2015-2020)11.1.4 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Products Offered11.1.5 Caribou Biosciences Recent Development 12 Future Forecast by Regions (Countries) (2021-2026)12.1 CRISPR And CRISPR-Associated (Cas) Genes Market Estimates and Projections by Region12.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast by Regions 2021-202612.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast by Regions 2021-202612.2 North America CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast (2021-2026)12.2.1 North America: CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast (2021-2026)12.2.2 North America: CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast (2021-2026)12.2.3 North America: CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast by Country (2021-2026)12.3 Europe CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast (2021-2026)12.3.1 Europe: CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast (2021-2026)12.3.2 Europe: CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast (2021-2026)12.3.3 Europe: CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast by Country (2021-2026)12.4 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast (2021-2026)12.4.1 Asia Pacific: CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast (2021-2026)12.4.2 Asia Pacific: CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast (2021-2026)12.4.3 Asia Pacific: CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast by Region (2021-2026)12.5 Latin America CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast (2021-2026)12.5.1 Latin America: CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast (2021-2026)12.5.2 Latin America: CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast (2021-2026)12.5.3 Latin America: CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast by Country (2021-2026)12.6 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast (2021-2026)12.6.1 Middle East and Africa: CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast (2021-2026)12.6.2 Middle East and Africa: CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast (2021-2026)12.6.3 Middle East and Africa: CRISPR And CRISPR-Associated (Cas) Genes Market Size Forecast by Country (2021-2026) 13 Market Opportunities, Challenges, Risks and Influences Factors Analysis13.1 Market Opportunities and Drivers13.2 Market Challenges13.3 Market Risks/Restraints13.4 Porters Five Forces Analysis13.5 Primary Interviews with Key CRISPR And CRISPR-Associated (Cas) Genes Players (Opinion Leaders) 14 Value Chain and Sales Channels Analysis14.1 Value Chain Analysis14.2 CRISPR And CRISPR-Associated (Cas) Genes Customers14.3 Sales Channels Analysis14.3.1 Sales Channels14.3.2 Distributors 15 Research Findings and Conclusion 16 Appendix16.1 Research Methodology16.1.1 Methodology/Research Approach16.1.2 Data Source16.2 Author Details

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Scientists at the Council of Scientific & Industrial Research-Institute of Genomics and Integrative Biology have come up with a low-cost coronavirus test that will not require any expensive machines for detection of the pathogen.

The test can detect the coronavirus disease (Covid-19) within one hour and is expected to be available for the first phase of testing in four weeks.

Named after ''Feluda'', the detective character in legendary filmmaker Satyajit Ray's stories, although it is also an acronym for FNCAS9 Editor-Linked Uniform Detection Assay.

The test uses CRISPR gene-editing technology to identify and target the genetic material of Sars-CoV2, the virus that causes Covid-19. The test has been developed by Debojyoti Chakraborty and Souvik Maiti as a simpler way of detecting SARS-coV2 presence in clinical samples.

The CRISPR-based Feluda testing works by combining CRISPR biology and paper strip chemistry. Briefly, Cas9 protein, a component of the CRISPR system, is barcoded to interact specifically with the Sars-CoV2 sequence in the patients genetic material.

The complex of Cas9 with Sars-CoV2 is then applied to a paper strip, where using two lines (one control, one test) make it possible to determine if the test sample was infected with Covid-19.

Using the innovative chemistry on a paper strip, the CRISPR complex, bound to that specific sequence, can be visualised as a positive band, like one sees in simple pregnancy tests. The entire diagnostic process takes about one hour, starting from RNA to giving a visual readout on the strip.

Most labs are working with PCR(polymerase chain reaction)-based technology, which is costly and needs a lab set-up. The paper strip does not require (biosafety) Level-2 or Level-3 lab for testing and can be done in any path lab.

Unlike other CRISPRtests that use CAS12 and CAS13 proteins to detect Sars-CoV2, the CSIR-IGIB kit technology uses CAS9 protein (CRISPR-associated protein 9) to identify and bind to the target sequence.

CRISPR, which is short for Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technology that can be used to detect a specific snippet of DNA from a sequence. It can also be used to turn genes on or off without altering their sequence.

Feluda is not limited to Covid-19. The team has been working on Feluda for the past two years to develop an assay that can work on detecting any DNA-RNA or their mutations.

This is the only Covid-19 testing kit that has been developed using CRISPR-based technology in India. Feluda has been licenced to Tata Sons, which will commercialise the technology for Covid-19 detection.

Each Feluda test costs Rs 500 in the lab, and is expected to bring the cost of testing down from the Rs 4,500 per test for the real-time polymerase chain reaction test (RT-PCR), which is the only available test for detecting current Covid-19 infection.

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This Test Named After Satyajit Ray's Detective Character Can Reportedly Detect The Virus In An Hour - ScoopWhoop

Covid-19 has changed life as we know it. It has also accelerated already rapid trends in innovation and collaboration across the scientific community.

As the pandemic spreads across the globe, researchers are racing to develop diagnostics, vaccines and treatments. In the pursuit of new solutions to tackle SARS-CoV-2, the novel coronavirus that causes Covid-19, researchers have been turning to machine learning, AI and high-throughput experimental automation that aid in development. Another powerful tool they are using to accelerate the process is CRISPR. This gene-targeting and gene-editing technology, based on the mechanism that bacteria naturally use to fight viruses, is already proving useful in our joint fight against this new virus.

CRISPR Advances Covid-19 TestingWe know early detection of SARS-CoV-2 is essential to isolating infected patients and managing appropriate healthcare responses. Recently, researchers at MIT published a rapid CRISPR-Cas13-based COVID-19 detection assay protocol.Since CRISPR can be modified to target nearly any genetic sequence, it can be used to detect SARS-CoV-2 RNA in a patient sample. This assay utilizes an RNA-targeting CRISPR nuclease to help scientists detect the SARS-CoV-2 RNA from patient samples within 60 minutes. More recently, an improved assay was developed by researchers at MIT that was shown to provide faster and more robust results.

Utilizing another CRISPR nuclease that is thermostable, they developed a test that in one step copies the viral RNA in a patient sample, such as saliva, into the more stable DNA and then specifically identifies a SARS-CoV-2 gene sequence. Performing this point-of-care assay requires minimal lab equipment and resources, as it only needs a few reagents and a heat source, delivering results in as little as 40 minutes. Supplementing existing tests with new CRISPR-based approaches can broaden accessibility to Covid-19 testing, a key strategy for stopping the spread through track and trace efforts, as outlined by the World Health Organization.

CRISPR Helps Engineer Future TreatmentsPreviously, the genome-engineering power of CRISPR has been directed at fighting genetic diseases. But more recently, its also being harnessed to fight infectious diseases, now including the new coronavirus.

Understanding how a pathogenic disease operates at the host-pathogen interface is critical to developing new treatments. CRISPR-based genome engineering enables researchers to study how SARS-CoV-2 interacts with human cells and generate the appropriate cell models that could lead to faster discovery of a potential new treatment or an existing drug combination that may provide a treatment solution. Once a potential treatment is identified, CRISPR makes the next step drug target screening more efficient, advancing us more quickly to a viable treatment option.

As an example of this approach in action, researchers are exploring if CRISPR can be used to verify the functional relevance of human genes recently identified to interact with SARS-CoV-2 proteins. The investigation of the molecular mechanisms of the novel virus can ultimately help identify drug combinations that have the best potential to treat those infected.

Current Fight for the Future of Human HealthGenome engineering has been rapidly harnessed by academic and non-profit institutions, the biopharma industry, and scientific pioneers to develop Covid-19 testing and treatment solutions. CRISPR-based genome engineering enables researchers to study how SARS-CoV-2 interacts with human cells and generate the appropriate cell models that could lead to faster discovery of a potential new treatment or an existing drug combination that may provide a treatment solution.

Beyond this, the unprecedented innovation taking place in response to the Covid-19 pandemic will provide a foundation for improving human health in the future. Additionally, as technologies and understanding mature, new approaches, such as engineered cell therapies, will become part of the toolkit in future responses to global health challenges.

The current scientific response is representative of the future of life sciences a future where we integrate multiple technologies and disciplines including high throughput experimental automation, machine learning and agile, programmable tools such as CRISPR to fundamentally change our approach to research and development. We are seeing a new bar being set on the speed of science as the research community comes together, leveraging these technologies to respond to the Covid-19 pandemic at unprecedented velocity. Once the public health crisis subsides and the research halted by the pandemic resumes, the need for these transformative tools, technologies and approaches to life science research and development will be greater than ever.

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How CRISPR can help us win the fight against the pandemic - MedCity News

CRISPR-Cas9 gene-editing is one of the most powerful tools available to modern science, but genetically-modified organisms (GMOs) in food are subject to some tight regulations. Now, researchers at North Carolina State University have created a new version of CRISPR that lets scientists edit crops without introducing new DNA, meaning they technically arent GMOs.

CRISPR-Cas9 allows for precise cut-n-paste edits to DNA in living cells. An RNA guide sequence directs the system to the target section of the genome. Once there, an enzyme, usually Cas9, snips out the sequence then deletes it or replaces it with something else. In this way, scientists can cut out problem genes, such as those that cause disease, or add new beneficial ones, such as giving crops better pest resistance.

For the new study, the researchers tweaked the process to make a cleaner edit in plants. It uses a process known as lipofection, where positively-charged lipids are used to build a kind of bubble around the Cas9 and RNA mechanisms. When injected into the organism, this bubble binds to and fuses with the cellular membrane, which pushes the CRISPR system into the cell itself. The method also uses a Cas9 protein itself, rather than the Cas9 DNA sequence.

The team tested the method by introducing fluorescent proteins into tobacco plants. And sure enough, after 48 hours the edited plants were glowing, indicating it had worked.

Wusheng Liu/NC State University

The new method has a few advantages over existing ones, the team says. Its easier to target the desired genetic sequence, and opens up new crops that couldnt be edited with existing methods. Plus, the protein only lasts for a few days before degrading, which reduces off-target edits.

But the most important advantage is that the resulting crops arent considered GMOs. Since the new method doesnt use Cas9 DNA, it doesnt introduce foreign DNA into the plant, which is an important distinction.

This was the first time anyone has come up with a method to deliver the Cas9 protein through lipofection into plant cells, says Wusheng Liu, lead author of the study. Our major achievement was to make that happen. Also, since many consumers prefer non-GMO specialty crops, this method delivers the Cas9 protein in a non-GMO manner.

As useful as genetic engineering can be, the term GMO has negative connotations for many people, who believe there are health concerns with eating these crops or meats. Other problems include the chance of modified plants or animals escaping into the wild, where they can spread their new genes to the native population, affecting ecosystems.

As such, the US Department of Agriculture (USDA) and the Food and Drug Administration (FDA) have regulations on which edited crops and animals are allowed in food. And theyve decided that the line is drawn at introducing foreign genes into an organism.

It makes sense. Humans have been genetically-engineering plants and animals for millennia, through selective breeding. Many of our most widely-eaten crops are bigger, tastier, and easier to eat or grow, to the point that they hardly resemble their wild counterparts anymore.

CRISPR and other gene-editing tools can be the next generation of this process. By removing problematic genes or ensuring that specific ones are turned on or off, scientists arent really creating anything new. Some individuals naturally have mutations that do the same thing all the scientists are really doing is removing the element of chance, genetically.

In 2015, a new type of salmon became the first genetically engineered animal approved by the FDA for human consumption. In 2016, a Swedish scientist grew, harvested and served up CRISPR cabbage after approval by the Swedish Board of Agriculture. In both cases, the products were allowed because they were functionally identical to wild-type organisms the scientists had just chosen beneficial genes from an existing natural pool, without introducing foreign DNA.

That said, the rules aren't the same everywhere. In 2018 the Court of Justice of the European Union somewhat controversially ruled that tough GMO laws applied to crops that had been edited even if new DNA hadn't been inserted. The issue will likely remain fragmented, but for the NC State team at least, their crops aren't GMOs according to their own country's regulations.

However, there are still some hurdles to overcome before the new method becomes viable. The team says that lipofection can only be done if the outer wall of the plant cell is removed first. This kind of plant cell, known as a protoplast, allows scientists to more easily tweak the genes, but it isnt possible in all types of crops, and even when it does work, its a complex process.

Instead, the researchers are exploring other options that dont require removing the cell wall at all. One such alternative is to use CRISPR to introduce the Cas9 protein into pollen grains, which can then go on to fertilize another plant. Some of the offspring will have the required genetic edits from day one.

The researchers plan to investigate this latter method in tomatoes and hemp first, before moving onto others.

The new study was published in the journal Plant Cell Reports.

Source: NC State University

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New CRISPR method edits crops without technically making them GMOs - New Atlas

ZUG, Switzerland and CAMBRIDGE, Mass., May 15, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that four abstracts have been accepted for poster presentation at the American Association for Cancer Research (AACR) Virtual Annual Meeting II, which will take place from June 22 to 24, 2020.

Session information is available online via the Annual Meeting Itinerary Planner through the AACR website at http://www.aacr.org.

Title: Functional and single-cell assessment of CRISPR-modified CAR-T cells from NSCLC patients and healthy donors Session Title: Adoptive Cell Therapy 1E-Poster Number: 879Abstract Number: 3338

Title: Allogeneic CAR-T cell products containing 10 gene edits using CRISPR/Cas9 can retain full functionality in vivo and in vitro Session Title: Adoptive Cell Therapy 1E-Poster Number: 880Abstract Number: 4647

Title: Allogeneic anti-PTK7 CAR-T cells for the treatment of solid tumors Session Title: Adoptive Cell Therapy 3E-Poster Number: 3243Abstract Number: 6231

Title: Targeting T cell lymphomas with CRISPR/Cas9-generated anti-CD70 allogeneic CAR-T cellsSession Title: Adoptive Cell Therapy 5E-Poster Number: 6595Abstract Number: 3308

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

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

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CRISPR Therapeutics Announces Presentations at the American Association for Cancer Research 2020 Annual Meeting - GlobeNewswire

Researchers at ChristianaCare are looking to see how a newly approved coronavirus test using CRISPR could be used in Delaware, as they hope to learn how a patients genetic makeup can affect how the patient reacts to COVID-19.

Theres been a wide variance in the severity of COVID-19 symptoms.Many have required hospitalization and died from the disease while many others have experienced no symptoms at all. Others still have had much less common immune reactions to the virus like the rare inflammatory syndrome recently found in some children.

Dr. Eric Kmiec is Director of the Gene Editing Institute at ChristianaCare's Helen F. Graham Cancer Center & Research Institute. He, and others, believe this could be due to the genetic makeup of the patients.

It is possible for us to predict what types of people will be more susceptible? asked Kmiec. We know age and preconditions are absolute, and that is true, but now were seeing incredible variance among young people, millennials, even babies, and most of them dont have preexisting conditions that we can define. We might be able to define a genetic preexisting condition that may not even be apparent to the patient.

Kmiec says his team will be starting a research project in partnership with Stanford University and a few biotech companies to learn the relationship between an individuals genetic profile and how they respond to the virus.

He says the project will utilize CRISPR gene-editing technology developed at ChristianaCare to recreate genetic information on synthetic pieces of DNA on a chip.

This announcement comes as a new coronavirus test using CRISPR was fast-tracked through the approval process and okd by the Food and Drug Administration (FDA) just last week.

The so-called Sherlock test utilizes the gene-editing technology to detect the genome of the virus, and yields results in a couple hours.

Kmiec says the folks at MIT recently sent the test over.

Well be, sort of, working on it in the lab to see if theres a place for it in the future of what we call the long arch of testing here in Delaware, he said. The test has the ability to get to rural communities and hospitals that may not have such sophisticated instrumentation to carry out the tests that are currently on the market.

Delaware Medical Director Dr. Rick Hong couldnt say for sure if the state will be investing in the Sherlock test. But he says Delaware is looking into that as well as other testing technology for potential future purchase.

We appreciate our partners looking into other types of modalities, because I think we all need to work together on that, said Hong.

Kmiec says thatthough the price is subject to changeit appears the Sherlock test is less costly and requires less equipment than the qPCR tests currently being widely used. Be he adds Sherlock is rumored to be more sensitive and may run the risk of yielding more false positive results.

CRISPR technology in still in the early stages of clinical use. The technology raised controversy when Chinese scientists used it to change the genetic makeup of babies in 2018. It was used in the U.S. to treat a patient with a genetic disorder for the first time last year.

Kmiec says the Sherlock test was likely streamlined through the FDA process because of CRISPRs perceived future role in diagnostics.

The application here is for diagnostics, said Kmiec. So in terms of the rest of our concerns about off-site changes and its use to create mutations in patients, or something like that, that is off the table here.

Kmiec says ChristianaCare was already looking to move towards using CRISPR as tool to combat infectious diseases, but was pushed in that direction more quickly by the coronavirus outbreak.

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Gene editing tech CRISPR could be used to test for coronavirus and find who is most at-risk - Delaware First Media

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals Incorporated (VRTX) today announced that new data from two ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies have been accepted for an oral presentation at the EHA Congress, which will take place virtually from June 11-14, 2020.

An abstract posted online today includes 12 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and 6 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Updated data will be presented at EHA, including longer duration follow-up data for the first two patients treated in these trials and initial data for the second patient treated in the CLIMB-111 trial.

The accepted abstract is now available on the EHA conference website: https://ehaweb.org/congress/eha25/key-information-2/.

Abstract Title: Initial Safety and Efficacy Results With a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Hematopoietic Stem and Progenitor Cells in Transfusion-Dependent -Thalassemia and Sickle Cell DiseaseSession Title: Immunotherapy - ClinicalAbstract Code: S280

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

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

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

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

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

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic 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.

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

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

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

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, information regarding the data that is expected to be presented at the European Hematology Association (EHA)s upcoming Congress. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with theSecurities and Exchange Commissionand available through the company's website atwww.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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

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

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

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

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New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001 for Severe Hemoglobinopathies Accepted for Oral Presentation at the 25th European...

New Research Study On GlobalCRISPR and Cas Genes marketgives in-depth information on Market shares, growth opportunities, Industry Analysis, and Growth Aspects on competitive landscapes. The report helps readers to clearly understand the current and future status of the CRISPR and Cas Genes market from 2020 to 2029. This is the latest report covering the current market impact of COVID-19. The Coronavirus pandemic (COVID-19) has infected every aspect of life worldwide. This has brought with it numerous shifts in business conditions.

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The CRISPR and Cas Genes Market research report covers market features, volume, and growth, segmentation, geographical breakdowns, market shares, trends, and plans for this business. It allows you to identify the products/services and end-users that drive revenue growth and profitability. The CRISPR and Cas Genes industry report lists the leading competitors and provides the game-changing strategic analysis of the key factors driving the market. The report includes the forecasts by 2020-2029, analysis by 2014-2019, and discussion of important industry trends, market size, market share predictions, and profiles of the top CRISPR and Cas Genes industry players: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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CRISPR and Cas Genes Market Segmentation based on product, application, end user, and region-

Segmentation on the basis of product:

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

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

Biotechnology & Pharmaceutical CompaniesAcademic & Government Research InstitutesContract Research Organizations

The Report Covers Global regions North America, South America, Europe, and the Middle-East,& Africa and Asia-Pacific.

Research Study Offers a detailed analysis of this report:

Marketing strategy study and growth trends.

CRISPR and Cas Genes Market growth driven factor analysis.

Emerging recess segments and region-wise CRISPR and Cas Genes Markets.

An overall framework study, consisting of an evaluation of the parent market.

An empirical evaluation of the curve of the CRISPR and Cas Genes Market.

Major variations in CRISPR and Cas Genes Market dynamics.

Latest, Historical, and Expected size of the market from both prospect value and volume.

The report offers exclusive graphics and illustrative SWOT analysis of CRISPR and Cas Genes Market segments.

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Some Business Questions Answered in this Report:

1. What will the market size be in 2029?

2. What are the key factors driving the CRISPR and Cas Genes market?

3. Who are the key players in the market?

4. What are the challenges of market growth?

5. What are the market opportunities and threats facing key players?

6. What will be the growth rate in 2029?

7. Which strategies are used by top players in the CRISPR and Cas Genes market?

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Table of Contents:

1. Overview of the CRISPR and Cas Genes Industry.

2. Global CRISPR and Cas Genes Market Competitive aspects.

3. A share of Global CRISPR and Cas Genes Market.

4. CRISPR and Cas Genes Supply Chain Study.

5. Leading CRISPR and Cas Genes Company Profiles.

6. CRISPR and Cas Genes Globalization & Trade.

7. CRISPR and Cas Genes Suppliers and Buyers.

8. Import/Export scenario, Consumption by CRISPR and Cas Genes Major Countries.

9. Global CRISPR and Cas Genes Industry Forecast to 2029.

10. Key Growth factors and CRISPR and Cas Genes Market Outlook.

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CRISPR and Cas Genes Market Detailed Analysis 2020 : Impact Of COVID-19 and How Market Will Grow In The Upcoming Period 2020-2029? 3w Market News...

Global CRISPR and CRISPR-Associated (Cas) Genes Market: Snapshot

Over the years, biomedical researchers have increasingly focused on developing efficient and reliable methods for precise and targeted changes to virtually any point of genome of any living cell. Recent advances in the genome engineering has triggered several biological researches and translational applications. Economical manipulation and modification of genomic sequences enable molecular biologists identify and characterize key genetic determinants to facilitate the investigation of various biological processes.

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Genome editing via clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated (Cas) is considered as an innovative technique in programmable and high-throughput functional genomics. CRISPR-Cas system consists of pattern of repetitive sequences in the DNA of certain bacteria, who used it as an adaptive immune system to find a protection mechanism against invading foreign DNA.

In less than a decade, a host of novel targeted techniques and genomic engineering tools have been developed that facilitates precise and diverse genomic modifications in a variety of organisms and tissues. The recent tool having enormous potential in biomedical researches is the clustered regularly interspaced short palindromic repeats associated Cas9/sgRNA system, also called Cas9/sgRNA. Cas9 protein is an RNA guided endonuclease. Along with its variants it has generated considerable excitement versatile genomic engineering tool in the development of genetically edited (GE) crops. Primary areas research for this include examining gene function, understanding the regulatory signaling networks, and rewiring sgRNA for advance loss-of-function screening. This will help in combating biotic and abiotic stresses, thereby leading to the development of climate resilient crops and sustainable agriculture practices in the coming years.

Global CRISPR and CRISPR-Associated (Cas) Genes Market: Overview

In the past few years research and development of CRISPR or clustered regularly interspaced short palindromic repeats has allowed molecular biologists to designs solutions for repairing cells by genome editing. This method allows a change to a specific genome by the introduction of a new function or by correction of a mutation. The exceptional fidelity, simplicity of construction, and low cost has triggered a monumental demand for the several solutions offered by the global CRISPR and CRISPR-associated (Cas) genes market. The market is riding a wave of success as these factors have augmented the uptake of this method in several molecular biology laboratories.

The well-documented research report presents a fair case study of the global CRISPR and CRISPR-associated (Cas) genes market. The report includes a SWOT analysis and Porters five forces analysis, which help in understanding several facets of the global market in greater depth. Furthermore, analysts have used primary and secondary research methodologies, which ensure the authenticity of the facts. This information in the report has also been seconded by market experts with comments and recommendations about the subject matter. The comprehensive research report is aimed at guiding each of its readers to make well-informed business decisions.

Global CRISPR and CRISPR-Associated (Cas) Genes Market: Trends and Drivers

The products available in the global CRISPR and CRISPR-associated (Cas) genes market are DNA-free Cas and vector-based Cas. The widening applications of these are expected offer several lucrative opportunities to the global market. Out of various applications, genome engineering is expected to be a key contributor to the soaring revenue of the overall market in the near future. This trend will be attributable to eh increasing uptake of genome editing method for the therapeutic development and germline modifications. The report indicates that advancements in plant genome engineering will result in positive impact on the global market.

Analysts predict that CRISPR could be the next biotechnology treatment that has the ability to gradually replace the present single-antibody drugs. Genome engineering is anticipated to pick up a phenomenal pace in the coming years as it is being developed to build an immune response for targeting cancer. The widening application of these methods in the field of oncology is likely to change the game for the global market in the coming years.

Global CRISPR and CRISPR-Associated (Cas) Genes Market: Regional Outlook

In terms of geography, the global market is segmented into North America, Asia Pacific, Latin America, the Middle East and Africa, and Europe. North America is estimated to lead the global CRISPR and CRISPR-associated (Cas) genes market as the U.S. has shown a keen interest in developing effective therapeutics. Asia Pacific is also expected to offer several growth opportunities to the overall market as the region is facing a challenge of mounting unmet medical needs.

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Key Players Mentioned in the Report are:

The report has identified the following as the key operating players in the globalCRISPR and CRISPR-associated (Cas) genes market: Thermo Fisher Scientific, Inc., Caribou Biosciences, Inc., CRISPR THERAPEUTICS, Addgene, Mirus Bio LLC, Merck KGaA, Editas Medicine, GE Healthcare Dharmacon Inc., Takara Bio USA, Horizon Discovery Group plc, and Intellia Therapeutics, Inc.Analysts predict that these companies will focus on making strategic collaborations to ahead of the competition present in the overall market.

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CRISPR and CRISPR-Associated (Cas) Genes Market Competitive Landscape Analysis with Forecast by 2025 - News Distinct

CAMBRIDGE, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology bothin vivoandex vivo,is presenting three oral presentations and two poster presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place virtually from May 12-15, 2020. Intellia researchers are presenting new data in support of NTLA-5001, the companys engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML). Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE).

At Intellia, we are applying our CRISPR/Cas9 technology to develop new processes that can produce enhanced engineered cell therapies to treat severe cancers, such as AML, that traditional approaches cannot address. Our proprietary platform provides a powerful tool to generate more potent TCR-directed cells, that can treat blood cancers initially and potentially solid tumors. The data being presented today validate Intellias approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year, said Intellia President and CEO John Leonard, M.D. We are also pleased to present data that support our recently announced HAE development candidate, NTLA-2002, Intellias second systemic therapy employing our in vivo knockout approach and modular delivery platform.

Data Presentations on Intellias First Engineered Cell Therapy Development Candidate, NTLA-5001 for the Treatment of AML, and Proprietary Cell Engineering Process

NTLA-5001 is Intellias first engineered T cell receptor (TCR) T cell therapy development candidate, which targets the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001 is being developed in collaboration with Chiara Boninis team at IRCCS Ospedale San Raffaele to treat AML patients regardless of the genetic subtype of a patients leukemia. AML is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment and is the most common type of acute leukemia in adults(Source:NIH SEER Cancer Stat Facts: Leukemia AML).

Intellias proprietary process is a significant improvement over standard engineering processes commonly used to introduce nucleic acids into cells. Intellias process enabled multiple gene edits using CRISPR/Cas9, while maintaining cell products with high expansion potential and minimal undesirable chromosomal translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR in locus, while eliminating the expression of the endogenous TCRs, with the goal of producing homogeneous T cell therapies like NTLA-5001.

Intellias novel approach with NTLA-5001 can overcome the challenges of standard T cell therapy, including risks of reduced specificity associated with mixed expression and mispairing of endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease (GvHD) risks, which could lead to an attack on the patients healthy cells; and reduced efficacy tied to lower tgTCR expression per T cell. Intellias unprecedented process is expected to streamline cell engineering and manufacturing, yielding a homogenous product comprising WT1-targeted T cells with high anti-tumor activity. Data highlights from todays presentation include the following:

Intellias cell engineering efforts are focused on its initial clinical investigation of NLTA-5001 on AML, while continuing preclinical studies exploring the potential for targeting WT1 in solid tumors. The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021.

The presentation titled, Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering, will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell Therapy, and can be found here, on the Scientific Publications & Presentations page of Intellias website. These data were a follow-on to the study presented at Keystone Symposias Engineering the Genome Conference from this past February.

In Vivo Data Supports Intellias Novel TCR Candidate

A second presentation on engineered cell therapy progress, in collaboration with IRCCS Ospedale San Raffaele, showed in vivo data demonstrating the potential of TCR-edited T cells to effectively target WT1 tumor cells in AML. In addition to the previously disclosed results of effective in vitro recognition of primary AML tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells), new data indicate that the selected TCR also enables T helper cells (CD4 T cells) to react to WT1-expressing tumor cells, providing cytokine support. This distinguishes Intellias TCR from other therapeutic TCR candidates, which either exclusively activate toxic CD8 T cells or require the co-transfection of CD8 into CD4 T cells to render them functional.

Using a mouse model carrying disseminated human primary AML, researchers observed a significant therapeutic effect, including decreased AML tumor burden. In addition, no signs of GvHD were observed in mice treated with the WT1-specific T cells. The data show that tgTCR-engineered cells have targeted anti-cancer activity in a challenging model of systemic AML, demonstrating the therapeutic potential of Intellias engineered TCR T cell approach.

The presentation titled, Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia, will be given today by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy. Notably, ASGCT meeting organizers selected this presentation as one of six to receive the ASGCT Excellence in Research Award this year.

Continued Progress on Intellias Second In Vivo Development Candidate, NTLA-2002 for the Treatment of HAE

Intellia is presenting development data updates on its potential HAE therapy, NTLA-2002, which utilizes the companys systemic in vivo knockout approach, including its proprietary lipid nanoparticle (LNP) system. 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. NTLA-2002 aims to prevent unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.

The KLKB1 gene knockout in an ongoing non-human primate (NHP) study resulted in a sustained 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 six months, demonstrating the same high level of efficacy and durability seen in earlier rodent studies.

The short talk titled, CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema, will be given by Jessica Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when it will be made available here, on the Scientific Publications & Presentations page of Intellias website. The presented data include results from ongoing collaborations with researchers at Regeneron, and the program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent to initiate a Phase 1 trial for NTLA-2002 in the second half of 2021.

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.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia 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 or similar clinical trial application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020 and its planned dosing of first patients in the second half of 2020; 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; plans to submit an IND or similar clinical trial application for its hereditary angioedema (HAE) program in the second half of 2021; plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program and HAE programs, and other animal studies supporting other in vivo and ex vivo programs, including its AML program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion; further development of its proprietary cell engineering process for multiple sequential editing; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; 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; 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; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis or Regeneron Pharmaceuticals, Inc., and Regenerons ability to enter into a co-development and co-promotion agreement for the HAE program; statements regarding the timing of regulatory filings regarding its development programs.

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 its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Novartis or Regeneron or its other ex vivo collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities 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:Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917 lynnea.olivarez@intelliatx.com

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

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Intellia Therapeutics Reports Progress on CRISPR/Cas9 AML Cancer Therapy Using Proprietary Cell Engineering Process at the 23rd Annual Meeting of the...

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.

The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

In addition to RMAT designation, CTX001 has received Orphan Drug Designation from the U.S. FDA for TDT and from the European Commission for TDT and SCD. CTX001 also has Fast Track Designation from the U.S. FDA for both TDT and SCD.

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

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

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

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic 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 status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of RMAT designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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

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

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program and related global regulatory approvals, and expectations regarding the RMAT designation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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

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

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

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-961-0534orInternational: +44 20 3204 5275

More:
CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001 for the...

1. First CRISPR test for the coronavirus approved in the United States  Nature.com
2. FDA approves first coronavirus diagnostic test that uses CRISPR technology  ModernHealthcare.com
3. A CRISPR Way  GenomeWeb
4. FDA Approves CRISPR-Based Coronavirus Test  ExtremeTech
5. First gene-editing CRISPR test for coronavirus approved by the FDA, here is how it works  Times Now
6. View Full Coverage on Google News

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First CRISPR test for the coronavirus approved in the United States - Nature.com

The agency's guidelines would help companies who are making kits that allow Americans to swab themselves and send the sample into a lab all from the safety of their own home. Meanwhile, CRISPR technology could help cut testing time down to an hour. Meanwhile, as states grapple with testing questions, the porn industry could offer a template for moving forward.

The New York Times:F.D.A. Paves Way For Home Testing Of CoronavirusIn a move that could significantly expand the nations testing capacity, the Food and Drug Administration has posted new guidelines that could pave the way for millions of people to test themselves for the coronavirus at home. The guidelines allow companies to develop and market testing kits with the tools to swab their noses and mail the specimens to any lab in the country. (Jacobs, 5/7)

The Washington Post:FDA Gives Emergency Authorization For CRISPR-Based Diagnostic Tool For CoronavirusThe Food and Drug Administration on Thursday approved a new diagnostic tool that employs the revolutionary CRISPR gene-editing technology to determine in just one hour if someone is infected with the novel coronavirus. The FDAs emergency use authorization allows only high-complexity laboratories to use the test kit, developed by researchers at the Massachusetts Institute of Technology, Harvard University, the Ragon Institute and the Howard Hughes Medical Institute and marketed by Sherlock Biosciences of Cambridge, Mass. (Achenbach and McGinley, 5/7)

Boston Globe:Cambridge Biotechs Virus Test Using CRISPR Gene Editing OKd For CrisisA Cambridge biotech startup received emergency clearance Thursday for a faster and radically different laboratory test to diagnose COVID-19, a kit the company says can give results within an hour. The Food and Drug Administration provided an emergency use authorization for the test made by Sherlock Biosciences. It relies on the revolutionary genome-editing tool CRISPR, which edits DNA and has the potential to treat a vast array of diseases but had yet to win FDA approval for any product. The tool was repurposed to create a diagnostic test. (Saltzman, 5/7)

Stat:Could The Porn Industry Offer A Model For Reopening Amid Covid-19?As states and employers furiously develop plans to safely reopen workplaces in the midst of the coronavirus pandemic, theyre grappling with what seems like an endless list of questions: where to test, who to test, and how often to test for the virus? Further complicating matters are issues of workers privacy, geography, politics, science, and cost. Its a difficult mandate. But there is one place to look for guidance the adult film industry. (McFarling, 5/8)

CNN:Coronavirus: Rollout Of Antibody Tests Met With Confusion, Little OversightPublic health experts, including members of the White House Coronavirus Task Force, have argued accurate Covid-19 antibody tests can support efforts to get Americans back to work by determining who may have overcome the virus. But the rollout of millions of antibody tests in the US has created frustration and division among state health departments due to a mix of questionable tests, shifting federal rules and a hodgepodge of different methods for tracking results. (Devine, Bronstein and Griffin, 5/7)

Kaiser Health News:Trying Out LAs New Coronavirus Testing RegimeLast week, after Mayor Eric Garcetti announced that Los Angeles was offering COVID-19 tests to all city and county residents, I decided to get one myself and test Garcettis bold new promise in the bargain. I was surprised how easily I was able to log on to L.A.s testing website. I answered a few questions about myself, including whether I had any symptoms of the disease the answer was no and within three minutes, I had a same-day appointment at one of eight city-run testing sites. (Wolfson, 5/8)

San Francisco Chronicle:California Clinics, Counties Get $97 Million From Feds To Expand TestingThe U.S. Department of Health and Human Services has awarded $97.3 million to 179 California health centers to expand coronavirus testing in low-income communities, the federal agency said Thursday. The grants are part of $583 million the federal government is distributing to 1,385 health centers that receive funding from the Health Resources and Services Administration, a unit of HHS that seeks to improve health care access for uninsured and vulnerable Americans. The money comes from the federal Paycheck Protection Program and Health Care Enhancement Act, which provides funding for small businesses hurt by the pandemic and economic support for health care providers and testing efforts. (Ho, 5/7)

Atlanta Journal-Constitution:Governor Urges All Georgians To Get Testing For CoronavirusGov. Brian Kemp urged all Georgians to schedule an appointment for coronavirus screening regardless of whether they have symptoms, as the state continues to expand testing for the disease even as the rapid growth has exposed new strains. With the state no longer facing crippling shortages of key supplies, Kemp said Thursday that the capacity for testing now outstrips the publics demand in the weeks after he began to reopen parts of the economy. That has stressed area labs, however, struggling to keep up with record numbers of tests. (Bluestein and Hallerman, 5/7)

Atlanta Journal-Constitution:Ga. Heath Centers To Receive More Than $12.2 Million For COVID TestingNearly three dozen health centers in Georgia will share more than $12.2 million in federal dollars to expand testing forCOVID-19. This infusion of funding is part of nearly $583 million awarded across the U.S. and its territories by the U.S. Department of Health and Human Services to boost testing capacity. The money is going to centers funded by its Health Resources and Services Administration, which provide health care services to populations that have limited access to health care services. (Mariano, 5/7)

Boston Globe:Maine To Triple Testing Capacity For CoronavirusMaine Governor Janet Mills announced a partnership with IDEXX Laboratories, a local manufacturer, which will allow the state to more than triple its testing capacity in the coming weeks and remove testing criteria for those who believe they may have the virus. The public-private partnership will introduce a new diagnostic testing system to the states health department and bolster the states testing 2,000 tests per week to 7,000 tests per week for the foreseeable future, Mills said at a press conference Thursday afternoon. IDEXX is also lending 3,500 test kits to the states health department. (Berg, 5/7)

More here:
FDA Cutting Red Tape To Speed Development Of In-Home Tests; CRISPR Technology Gets Green Light From Agency - Kaiser Health News

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Crispr And Crispr Associated Genes Market 2020 | Growth Drivers, Challenges, Trends, Market Dynamics and Forecast to 2026 - Cole of Duty