Archive for the ‘Crispr’ Category

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.

CLICK HERE !! Connect with our Analyst To Know What Is The Impact Of COVID 19 On CRISPR and Cas Genes Market and be Smart in Redefining Business Strategies

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.

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

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.

For Access Complete TOC, Please Click Here @https://marketresearch.biz/report/crispr-and-cas-genes-market/#toc

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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.

Request Brochure of CRISPR and CRISPR-Associated (Cas) Genes Market Report for more Industry Insights @CLICK HERE NOW

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

The rest is here:
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

Crispr And Crispr Associated Genes Market has recently been added to its extensive repository by Market Research Intellect. This intelligence report includes research based on current scenarios, historical records and future forecasts. In this research report, specific data on various aspects such as type, size, application and end user were checked. It offers a 360-degree overview of the competitive landscape of industries. The SWOT analysis was used to understand the strengths, weaknesses, opportunities and threats faced by companies. This helps companies understand the threats and challenges ahead. The Crispr And Crispr Associated Genes market is growing steadily and the CAGR is expected to improve over the forecast period.

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**Our FREE SAMPLE COPY of the report gives a brief introduction of the Crispr And Crispr Associated Genes market, Detailed TOC, key players of the market, list of tables and figures and comprising key countries regions.**

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The report analyzes the key opportunities, CAGR, and Y-o-Y growth rates to allow readers to understand all the qualitative and quantitative aspects of the Crispr And Crispr Associated Genes market. A competition analysis is imperative in the Crispr And Crispr Associated Genes market and the competition landscape serves this objective. A wide company overview, financials, recent developments, and long and short-term strategies adopted are par for the course. Various parameters have been taken into account while estimating market size. The revenue generated by the leading industry participants in the sales of Crispr And Crispr Associated Genes across the world has been calculated through primary and secondary research. The Crispr And Crispr Associated Genes Market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status.

By Regions:

* North America (The US, Canada, and Mexico)

* Europe (Germany, France, the UK, and Rest of the World)

* Asia Pacific (China, Japan, India, and Rest of Asia Pacific)

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* Middle East & Africa (Saudi Arabia, the UAE, , South Africa, and Rest of Middle East & Africa)

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Chapter 1: Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global Crispr And Crispr Associated Genes market

Chapter 2: Evaluating the leading manufacturers of the global Crispr And Crispr Associated Genes market which consists of its revenue, sales, and price of the products

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

1. FDA OKs first-ever use of gene-editing tool CRISPR to tackle Covid-19  Boston Business Journal
2. Feng Zhang's Sherlock gets first-ever CRISPR nod as FDA green-lights Covid-19 test kit  MedCity News
3. FDA Authorizes First-Ever Crispr Application For COVID-19 Coronavirus Test  Forbes
4. A COVID-19 diagnostic that uses CRISPR gets a nod from the FDA  Chemical & Engineering News
5. FDA authorizes CRISPR-based test for COVID-19  The Verge
6. View Full Coverage on Google News

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FDA OKs first-ever use of gene-editing tool CRISPR to tackle Covid-19 - Boston Business Journal

ZUG, Switzerland and CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, is scheduled to present at the virtual Bank of America Securities 2020 Health Care Conference on Thursday, May 14, 2020, at 11:40 a.m. ET.

A live webcast of the fireside chat will be available on the "Events & Presentations" page in the Investors section of the Company's website at https://crisprtx.com/events. A replay of the webcast will be archived on the Company's website for 14 days following the presentation.

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.

Investor Contact:Susan Kimsusan.kim@crisprtx.com

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

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CRISPR Therapeutics to Present at the Bank of America Securities 2020 Health Care Conference - Yahoo Finance UK

A team of researchers at the McGovern Institute for Brain Research at MIT, the Broad Institute of MIT and Harvard, the Ragon Institute, and the Howard Hughes Medical Institute (HHMI) has developed a new diagnostics platform called STOP (SHERLOCK Testing in One Pot) COVID. The test can be run in an hour as a single-step reaction with minimal handling, advancing the CRISPR-based SHERLOCK diagnostic technology closer to a point-of-care or at-home testing tool. The test has not been reviewed or approved by the FDA and is currently for research purposes only.

The team began developing tests for COVID-19 in January after learning about the emergence of a new virus which has challenged the healthcare system in China. The first version of the teams SHERLOCK-based COVID-19 diagnostics system is already being used in hospitals in Thailand to help screen patients for COVID-19 infection.

The new test is named STOPCovid and is based on the STOP platform. In research it has been shown to enable rapid, accurate, and highly sensitive detection of the COVID-19 virus SARS-CoV-2 with a simple protocol that requires minimal training and uses simple, readily-available equipment, such as test tubes and water baths.

STOPCovid has been validated in research settings using nasopharyngeal swabs from patients diagnosed with COVID-19. It has also been tested successfully in saliva samples to which SARS-CoV-2 RNA has been added as a proof-of-principle.

The team is posting the open protocol today on a new website, STOPCovid.science. It is being made openly available in line with the COVID-19 Technology Access Framework organized by Harvard, MIT, and Stanford. The Framework sets a model by which critically important technologies that may help prevent, diagnose, or treat COVID-19 infections may be deployed for the greatest public benefit without delay.

There is an urgent need for widespread, accurate COVID-19 testing to rapidly detect new cases, ideally without the need for specialized lab equipment. Such testing would enable early detection of new infections and drive effective test-trace-isolate measures to quickly contain new outbreaks. However, current testing capacity is limited by a combination of requirements for complex procedures and laboratory instrumentation and dependence on limited supplies. STOPCovid can be performed without RNA extraction, and while all patient tests have been performed with samples from nasopharyngeal swabs, preliminary experiments suggest that eventually swabs may not be necessary. Removing these barriers could help enable broad distribution.

The ability to test for COVID-19 at home, or even in pharmacies or places of employment, could be a game-changer for getting people safely back to work and into their communities, says Feng Zhang, a co-inventor of the CRISPR genome editing technology, an Investigator at the McGovern Institute for Brain Research and HHMI, and a Core Institute member at the Broad Institute. Creating a point-of-care tool is a critically important goal to allow timely decisions for protecting patients and those around them.

To meet this need, Zhang, McGovern Fellows Omar Abudayyeh and Jonathan Gootenberg, and colleagues initiated a push to develop STOPCovid. They are sharing their findings and packaging reagents so other research teams can rapidly follow up with additional testing or development. The group is also sharing data on the StopCOVID.science website and via a submitted preprint. The website is also a hub where the public can find the latest information on the teams developments.

STOPCovid is not yet approved by the FDA and is currently being used for research purposes.

How it works

The STOPCovid test combines CRISPR enzymes, programmed to recognize signatures of the SARS-CoV-2 virus, with complementary amplification reagents. This combination allows detection of as few as 100 copies of SARS-CoV-2 virus in a sample. As a result, the STOPCovid test allows for rapid, accurate, and highly sensitive detection of COVID-19 that can be conducted outside clinical laboratory settings.

STOPCovid has been tested on patient nasopharyngeal swab in parallel with clinically-validated tests. In these head-to-head comparisons, STOPCovid detected infection with 97% sensitivity and 100% specificity. Results appear on an easy-to-read strip that is akin to a pregnancy test, in the absence of any expensive or specialized lab equipment. Moreover, the researchers spiked mock SARS-CoV-2 genomes into healthy saliva samples and showed that STOPCovid is capable of sensitive detection from saliva, which would obviate the need for swabs in short supply and potentially make sampling much easier.

The test aims to ultimately be simple enough that anyone can operate it in low-resource settings, including in clinics, pharmacies, or workplaces, and it could potentially even be put into a turn-key format for use at home, says Omar Abudayyeh, McGovern Fellow at the McGovern Institute for Brain Research at MIT.

McGovern Fellow Jonathan Gootenberg adds, Since STOPCovid can work in less than an hour and does not require any specialized equipment, and if our preliminary results from testing synthetic virus in saliva bear out in patient samples, it could address the need for scalable testing to reopen our society.

Importantly, the full test both the viral genome amplification and subsequent detection can be completed in a single reaction, as outlined on the website, from swabs or saliva. To engineer this, the team tested a number of CRISPR enzymes to find one that works well at the same temperature needed by the enzymes that perform the amplification. Zhang, Abudayyeh, Gootenberg and their teams, including graduate students Julia Joung and Alim Ladha, settled on a protein called AapCas12b, a CRISPR protein from the bacterium Alicyclobacillus acidophilus, responsible for the off taste associated with spoiled orange juice. With AapCas12b, the team was able to develop a test that can be performed at a constant temperature and does not require opening tubes midway through the process, a step that often leads to contamination and unreliable test results.

Information sharing and next steps

The team has prepared reagents for 10,000 tests to share with scientists and clinical collaborators for free around the world who want to evaluate the STOPCovid test for potential diagnostic use, and they have set up the website STOPCovid.science to share the latest data and updates with the scientific and clinical community.

To learn more about the STOPCovid technology and point of care nucleic acid testing, visit STOPCovid.science. Kits and reagents can also be requested via a form on the website.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

*This article is based on research findings that are yet to be peer-reviewed. Results are therefore regarded as preliminary and should be interpreted as such. Find out about the role of the peer review process in researchhere. For further information, please contact the cited source.

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One-step Test Could Provide Rapid and Sensitive COVID-19 Detection - Technology Networks

Something's been brewing in the health care sector, the second-largest group in the S&P 500. Since the start of the second quarter, the Health Care Select Sector SPDR XLV, -0.01% is one of the top asset-gathering exchange traded funds.

The iShares Nasdaq Biotechnology ETF IBB, +0.13%, thanks to plenty of help from Gilead Sciences GILD, +0.18%, is up almost 14% over the past month. Much of the recent biotechnology ebullience is attributable to progress on the coronavirus treatment and vaccine front.

IBB is the largest biotechnology ETF by assets, but it's not the only one delivering impressive returns in recent weeks. Here's a trio of biotech ETFs that are knocking the cover off the ball.

Virtus LifeSci Biotech Clinical Trials ETF (BBC)

The Virtus LifeSci Biotech Clinical Trials ETF BBC, -0.05% tracks the LifeSci Biotechnology Clinical Trials Index. That benchmark is a collection of companies with drugs and therapies in clinical trials, which means BBC is at the right place at the right time in the battle to quash coronavirus.

BBC's holdings are basically equally weighted, but that doesn't distract from the fact that Moderna MRNA, +8.66% is the fund's top holding. Overall, BBC holds about 90 stocks, most of which dwell at the lower end of large-cap territory or are mid- or small-cap names.

In addition to Moderna, several other BBC components have coronavirus treatment exposure and roughly a dozen are credible takeover targets. That's enough to have BBC up nearly 27% over the past month.

ARK Genomic Revolution ETF (ARKG)

In the health care space, the ARK Genomic Revolution ETF (cboe:ARKG) has been one of the best-performing funds for several years, trouncing traditional health care and biotechnology ETFs for several years, speaking to the capabilities of the fund's active managers.

ARKG typically holds 30 to 50 stocks and currently holds 34, several of which are coronavirus plays and that doesn't even begin to underscore the fund's virus detection exposure, which highlights ARKG's deep CRISPR exposure.

As government officials re-open the US, testing for the COVID-19 virus will be a critical step. A new CRISPR-based test called DNA Endonuclease-Targeted CRISPR Trans Reporter (DETECTR) could help speed the process along, ARK analyst Ali Aurman saidin a recent note.

ARKG is higher by nearly 32% over the past month.

Principal Healthcare Innovators Index ETF (BTEC)

The Principal Healthcare Innovators Index ETF BTEC, +0.45% is a departure from the other funds mentioned here because it's not drug/therapy-centric. Rather, BTEC tilts more toward medical device, equipment and life sciences firms.

The fund invests in companies that are leading the charge toward innovative solutions, rather than spending money on marketing and distribution, according to Principal.

BTEC's nearly 210 holdings are considered research and development-intensive companies and the fund's methodology screens out companies with negative or inconsistent earnings. The fund is higher by almost 25% over the past month.

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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3 Biotech ETFs Up 10% Or More Over The Last Month - MarketWatch

Yield10 Bioscience develops new technologies to increase crop yield in support of a more sustainable food system.

Founded in 1992, the companys focus is metabolic engineering technologies using predictive models to facilitate gene discovery. The aim is to enhance photosynthesis and improve seed and crop yield. The ultimate goal is to make major food crops more productive.

The company is working with a strong pipeline of genetic traits and has achieved initial encouraging yield improvement results in canola, soybean, rice and corn.

According to CEO Dr Oliver Peoples, the biggest gains to be made through CRISPR genome-editing lie in staple food and feed crops.

CRISPR genome-editing can benefit the entire agriculture industry when applied to large acreage of staple food and feed crops such as corn, soybean, canola, rice, wheat, potato and more. Provided the regulatory processes are based on science, genome editing will also be useful in fresh produce crops and for other types of food staples, like cassava and millet, which are important in less developed countries.

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are the hallmarks of the bacterial defence system, which forms the basis of CRISPR-Cas genome editing technology.

CRISPR can be used as an advanced plant-breeding tool. It facilitates crop breeding by making cuts at specific locations in a plant genome. Subsequent repair of the cut by the cells endogenous repair mechanism can introduce precise changes.Unlike GMOs, the system works with the native characteristics in the crop and does not introduce new genes. Supporters argue that this means that the biotechnology poses fewer risk factors than GMOs and the process is frequently compared to traditional crop breeding techniques.

Dr Peoples believes that CRISPR will need to be used alongside traditional breeding and GMOs to support food system sustainability. We need traditional breeding, CRISPR and GMO technologies to ensure sustainable global food security, he told FoodNavigator.

In order to feed the estimated 10 billion people by 2050 using the existing land and resources we have available; the agriculture industry needs to incorporate CRISPR genome-editing to enhance crop efficiency.

The key to improving crop genetics has been to use genetic diversity available within a plant species through breeding. By crossing two different plants we are transferring DNA or genes from one plant into the other and then screening to see if that creates a beneficial trait. CRISPR is just a new tool for creating targeted genetic diversity in a crop.

Dr Peoples places these developments in the context of the changing global climate, which brings increased extreme weather events, and the growing population. These factors mean that there is a pressing need to develop new crop varieties that can withstand challenging weather and produce a greater yield on the same amount of land.

The challenges with sustainable food production caused by climate change are due to the unpredictable and variable seasonal weather extremes that farmers are now experiencing. These include drought, flooding, heat and late or early frosts, all of which can have different impacts across even a single growing season. As the average temperature increases, farmers crops also face more pressure from disease and insects.

Using CRISPR genome-edited traits can help make crops more resilient to these weather extremes and disease pressures. The types of genetic traits can be grouped into two key categories; yield traits and stress tolerance traits.

Yield traits focus on photosynthetic efficiency of a crop during the growing season to either protect seed yield under poor growing conditions or to increase the seed yield of a crop, the latter being crucially important to meet the needs of the growing global population.

Stress tolerance traits help increase a crops resiliency such that yields are not reduced by environmental stressors often associated with climate change such as heat, cold, drought and flooding. Each of these types of traits have a common goal and that is to protect food production so incorporating both, not just one or the other, will be key to overcoming challenging growing seasons stemming from climate change and meeting food security demands.

Traditional plant breeding techniques have been used for thousands of years in crop cultivation. Gene editing offers two key advantages: speed and precision.

Plant breeding for new or improved traits like drought tolerance requires access to new genetic diversity in the form of different DNA sequences. Breeders have used many techniques to create that genetic diversity including radiation and chemical mutagenesis in their breeding programs.

CRISPR genome-editing is a precise plant breeding technique and accelerates the trait development process by activating and suppressing very specific genes in the plant DNA in a highly precise and targeted way. This means its a faster, more efficient approach to create crops with ideal traits such as increased yield and resilience.

Dr Peoples argued that CRISPR and indeed genetic engineering are more precise and even safer than some traditional plant breeding methods, such as radiation mutagenesis.

GMOs have been poorly received by European regulators and significantly consumers. Unlike CRISPR, genetic modification works by introducing new DNA to plants.

Dr Peoples believes that GMOs are an important piece of the toolbox for delivering food security and, again, he compares the techniques used to more orthodox agricultural methods. Take for example the use of pest resistance genes from bacteria, which were introduced into crops to make them insect resistant. This is basically the equivalent to vaccinating the plant, so it has immunity to the pest. The same genes and resistance proteins are used extensively in organic farming by spraying them onto the plants. GMO is simply a more efficient process for accomplishing the same goal which is to protect the crop from insects.

Nevertheless, European regulators take a relatively tough stance on controlling GMOs, the definition of which includes CRISPR techniques.

Dr Peoples said he believes this stance to be politically and economically driven rather than based on the scientific facts. First, there is not a single crop grown in the EU that has not been developed using some form of genetic engineering. Crossing different varieties of plant is, after all, the basis of plant breeding and the purpose is to take DNA from one variety and introduce it into the other using sexual crossing.

The anti-GMO stance of the EU is driven primarily by misinformation to the public from NGOs and market protectionism to sustain the EUs common agricultural policy. The EU imports large quantities of GMO soybean meal from both North and South America for animal feed, so where they have a need, they adapt and follow the science.

He also took aim at Europes controversial decision to lump CRISPR gene editing into its definition of GMOs. I dont believe theres a clear misunderstanding of what CRISPR genome-editing is and how it differs from genetic modification. The EU regulatory bodies have been educated about these differences. In fact, the EU regulatory body initially took the position that CRISPR genome editing is different from genetic modification before pressures from anti-GMO groups drove a 2019 EU court ruling blocking this position.

Dr Peoples suggested that little can be done to win over these anti-GMO lobbyists. I doubt that it matters to these groups which form of genetic engineering is used. Their business model centers around a firm anti-genetic engineering or GMO stance, so the scientific facts dont matter as much as staying on message.

However, he said that the public mood towards gene editing appears to be shifting: Thanks to new advancements and education around this technology, more everyday consumers are embracing CRISPR genome editing. Since this technique uses a plants existing DNA, it feels closer to the traditional breeding techniques, appears safer and doesnt warrant the same backlash as GM.

In order to support this shift, the scientific community is pushing back against the anti-GMO rhetoric to spotlight the economic, health, social and environmental benefits of CRISPR genome-editing.

In the EU, EASAC, the voice of independent science advice, has called for radical reform of EU legislation around GMOs noting that current regulations are no longer fit for purpose.

Efforts to communicate and educate are vital to gaining support, Dr Peoples suggested. Progress has been made but in order to create a substantial impact, the scientific community needs to continue working with various governments and regulatory bodies to educate consumers and legislators about the benefits these breeding techniques can offer.

Dr Peoples also suggested that attitudes towards the use of science in food are being shaped by external events. Necessity, it would seem, not only proves the mother of invention but also of acceptance.

Minimizing the effects of climate change and creating a healthier, more sustainable lifestyle are two key factors weighing heavily on genetically engineered crop acceptance. For example, the plant-based meat trend has been advertised as a healthier, more environmentally friendly alternative to traditional meat, and some of these plant-based substitutes have been developed using GMO products.

For millennials, climate change and sustainability are their key decision drivers and they are more open to technology in general. Many are not vested in the anti-GMO falsehoods which sustained the entire careers of anti GMO activists before evolving into a negative marketing tool. Were living in a time where consumer decisions are driven by their social, economic, environmental and health interests.

The current coronavirus crisis the first global pandemic since the 1918 Spanish flu has led to people feeling threatened physically and economically by the COVID-19 virus. This, too, is making people more open to science-based solutions.

Now more than ever we are looking to science for solutions and the specific scientific method or tool for creating that solution simply does not matter. The third-generation vaccine developed by the Canadian Government and Merck for Ebola is a great example of the power of genetic engineering tools and the benefits to humanity of a GMO product. Work is underway at the University of Oxford in the UK and probably elsewhere to develop a COVID-19 vaccine using a similar approach.

However, Dr Peoples added, there are limits: For the first time in many decades the availability of food is once more a question. The availability of an effective vaccine will not change the minds of many anti-vaxers just like the use of new genetic engineering tools wont change the minds of anti-GMO activists.

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CRISPR genome editing to address food security and climate change: 'Now more than ever we are looking to science for solutions' - FoodNavigator.com

ZUG, Switzerland and CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, is scheduled to present at the virtual Bank of America Securities 2020 Health Care Conference on Thursday, May 14, 2020, at 11:40 a.m. ET.

A live webcast of the fireside chat will be available on the "Events & Presentations" page in the Investors section of the Company's website at https://crisprtx.com/events. A replay of the webcast will be archived on the Company's website for 14 days following the presentation.

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.

Investor Contact:Susan Kimsusan.kim@crisprtx.com

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

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CRISPR Therapeutics to Present at the Bank of America Securities 2020 Health Care Conference - Yahoo Finance

Wide-spread testing has long been the Achilles' heel of the U.S. response efforts. Shortages, false negatives, and inaccurate kits have plagued efforts to provide quick and easy results to Americans. But scientists hope a new test can be the answer to that problem. In other testing news: antibody tests, false negatives and states' ongoing efforts to ramp up screenings.

The New York Times:With Crispr, A Possible Quick Test For The CoronavirusA team of scientists has developed an experimental prototype for a fairly quick, cheap test to diagnose the coronavirus that gives results as simply as a pregnancy test does. The test is based on a gene-editing technology known as Crispr, and the researchers estimated that the materials for each test would cost about $6. Were excited that this could be a solution that people wont have to rely on a sophisticated and expensive laboratory to run, said Feng Zhang, a researcher at the Broad Institute in Cambridge, Mass., and one of the pioneers of Crispr technology. (Zimmer, 5/5)

ABC News:Roche CEO Severin Schwan Says Producing New Antibody Test Part Of Busiest Time In His CareerSeverin Schwan, the CEO of Swiss drugmaker Roche, says employees from across the company are coming together to contribute to meeting the aggressive manufacturing targets the company has promised after receiving emergency Food and Drug Administration approval for its COVID-19 antibody test on Sunday. "I mean, even in the packaging lines, we have our employees who come from all over the place, who raised their hand and who say ... 'Can I help out in the warehouse?'" Schwan said in an interview with ABC News. "The engagement is -- and the commitment and the passion is -- really enormous. I can't tell you how proud I am of the organization and how they excel during this difficult, unprecedented time." (Bolton, 5/5)

Boston Globe:Walthams PerkinElmer Gets Emergency Approval For COVID-19 Antibodies TestPerkinElmer, the Waltham-based diagnostics maker, said Tuesday that a subsidiary has obtained an "emergency use authorization from the Food and Drug Administration for a test that detects antibodies in the blood of people who fought off COVID-19. The test made by EUROIMMUN, a subsidiary based in Lubeck, Germany, is one of a dozen made by different companies that have won such approvals, according to the FDA website. (Saltzman, 5/5)

Cincinnati Enquirer:COVID-19: Who's At Fault When A Negative Test Is False? Maybe NobodyWhen a Hyde Park woman was tested for COVID-19 and the test came back negative despite her symptoms, she was perplexed, disappointed and anxious. She was clearly sick and doctors ultimately decided to treat her. So who was at fault? Maybe nobody. That's because even a good test can end in a bad or faulty result. Improving test reliability is important, as Ohio, Kentucky and other states start to loosen restrictions on business and rely on tests to find people who need treatment. (Mitchell, 5/5)

Politico:Bubble Bursts On Cuomos Hope Of 'Immunity' TestingAs his state was being ravaged by the worst coronavirus outbreak in the country in early April, Gov. Andrew Cuomo offered a glimpse of hope to those looking for a quick restart of New Yorks economy: An antibody test, developed in a state laboratory, may be able to confirm immunity for a vast number of New Yorkers. By last week, Cuomo and other officials had retreated from the approach, pointing instead to other metrics that may indicate the best way to avoid future waves of the virus. Cuomo is now describing the immunity idea as little more than a theory. (Gronewold and Young, 5/5)

WBUR:Florida Boosts COVID-19 Testing, Sending New-Case Rate To A Record LowFlorida Gov. Ron DeSantis says his state has hit a new record low in the rate of positive test results for COVID-19, as only around 2.5% of nearly 24,000 tests confirmed an infection on Monday. The test results were the most Florida has received in a single day. The governor celebrated the good news, which emerged one day after Florida relaxed shutdown restrictions on restaurants and retail stores in much of the state. (Chappell, 5/5)

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Utilizing CRISPR, Scientists Develop Prototype For Coronavirus Test That Could Be As Quick, Easy As A Pregnancy One - Kaiser Health News

New Delhi: Scientists from Delhi based-Institute of Genomics and Integrative Biology (IGIB) would work together with Tata Sons to develop Indias first paper-strip test for rapid diagnosis of Covid-19.

The premier CSIR laboratory has signed a Memorandum of Understanding (MoU) with Tata Sons for licensing of know-how and scaling up the technology in the form a kit which can be deployed for Covid-19 testing on the ground by the end of May.

The testing approach was developed by a team led by scientists, Dr Souvik Maiti and Dr Debjyoti Chakraborty from CSIR-IGIB in early April and named Feluda - FNCAS9 Editor Linked Uniform Detection Assay (Feluda), after a famous fictional character that appeared in short stories written by noted filmaker Satyajit Ray.

The test uses cutting-edge, futuristic gene-editing tool- Crispr-Cas9 to target and identify the genomic sequences of the novel coronavirus in the samples of suspected individuals.

The technology was conceived and developed at CSIR IGIB under sickle cell mission and utilizes an indigenously developed cutting edge CRISPR Cas9. A combination of CRISPR biology and paper-strip chemistry leads to a visible signal readout on a paper strip that can be rapidly assessed for confirming the presence of viral infection in a sample," said Dr Anurag Aggarwal, Director, IGIB, New Delhi.

Unlike the real time PCR test currently used for diagnosis of Covid-19 in India, costing about 4500, this test would be affordable, since it does not depend on expensive real-time PCR machines which are already in limited supply.

We are happy to enter into a partnership with IGIB for further development and commercialization of this CRISPR based technology for COVID-19 detection. It uses a test protocol that is simple to administer and easy to interpret enabling results to be made available to the medical fraternity in relatively lesser time, as compared to other test protocols," said Banmali Agrawala, President - Infrastructure and Defence and Aerospace, Tata Sons.

The kit is similar to a portable paper-strip test used to confirm pregnancy and does not require any specialized tools or different skills to operate.

Unlike most rapid tests which require dedicated machinery, it can be performed using simple standard equipments available in any pathological laboratory.

It is also the first such indigeneous testing kit based on Crisper technology to be developed in India.

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Covid-19: CSIR-IGIB partner with TATA Sons to make Indias first paper-strip kit - Livemint

Within the last decades, new genetic engineering tools for manipulating genetic material in plants, animals and microorganisms are getting large attention from the international community, bringing new challenges and possibilities. While genetically modified organisms (GMO) have been known and used for quite a while now, gene drive organisms (GDO) are yet at the consideration and evaluation stage.

The difference between these two technologies, where both are meant to replace certain characters in animals or plants with ones that are more favourable for the human population, is that, even though in GDO there is also foreign "synthetic" DNA being introduced, the inheritance mode differs. In GDO, the genome's original base arrangements are changed, using CRISPR/Cas-9 genome editing. Once the genome is changed, its alterations are carried down the organism's offspring and subsequent generations.

In their study, published in the open-access journal BioRisk, an international group of scientists led by Marion Dolezel from the Environment Agency Austria, discuss the potential risks and impacts on the environment.

The research team also points to current regulations addressing invasive alien species and biocontrol agents, and finds that the GMO regulations are, in principle, also a useful starting point for GDO.

There are three main areas suggested to benefit from gene drive systems: public health (e.g. vector control of human pathogens), agriculture (e.g. weed and pest control), environmental protection and nature conservation (e.g. control of harmful non-native species).

In recent years, a range of studies have shown the feasibility of synthetic CRISPR-based gene drives in different organisms, such as yeast, the common fruit fly, mosquitoes and partly in mammals.

Given the results of previous research, the gene drive approach can even be used as prevention for some zoonotic diseases and, hence, possible future pandemics. For example, laboratory tests showed that the release of genetically modified mosquitoes can drastically reduce the number of malaria vectors. Nevertheless, potential environment and health implications, related to the release of GDO, remain unclear. Only a few potential applications have so far progressed to the research and development stage.

"The potential of GDOs for unlimited spread throughout wild populations, once released, and the apparently inexhaustible possibilities of multiple and rapid modifications of the genome in a vast variety of organisms, including higher organisms such as vertebrates, pose specific challenges for the application of adequate risk assessment methodologies," shares the lead researcher Mrs. Dolezel.

In the sense of genetic engineering being a fastly developing science, every novel feature must be taken into account, while preparing evaluations and guidance, and each of them provides extra challenges.

Today, the scientists present three key differences of gene drives compared to the classical GMO:

1. Introducing novel modifications to wild populations instead of "familiar" crop species, which is a major difference between "classic" GMOs and GDOs.

"The goal of gene drive applications is to introduce a permanent change in the ecosystem, either by introducing a phenotypic change or by drastically reducing or eradicating a local population or a species. This is a fundamental difference to GM crops for which each single generation of hybrid seed is genetically modified, released and removed from the environment after a relatively short period," shares Dolezel.

2. Intentional and potentially unlimited spread of synthetic genes in wild populations and natural ecosystems.

Gene flow of synthetic genes to wild organisms can have adverse ecological impact on the genetic diversity of the targeted population. It could change the weediness or invasiveness of certain plants, but also threaten with extinction the species in the wild.

Possibility for long-term risks to populations and ecosystems.

Key and unique features of GDOs are the potential long-term changes in populations and large-scale spread across generations.

In summary, the research team points out that, most of all, gene drive organisms must be handled extremely carefully, and that the environmental risks related to their release must be assessed under rigorous scrutiny. The standard requirements before the release of GDOs need to also include close post-release monitoring and risk management measures.

It is still hard to assess with certainty the potential risks and impact of gene drive applications on the environment, human and animal health. That's why highly important questions need to be addressed, and the key one is whether genetically driven organisms are to be deliberately released into the environment in the European Union. The High Level Group of the European Commission's Scientific Advice Mechanism highlights that within the current regulatory frameworks those risks may not be covered.

The research group recommends the institutions to evaluate whether the regulatory oversight of GMOs in the EU is accomodate to cover the novel risks and challenges posed by gene drive applications.

"The final decision to release GDOs into the environment will, however, not be a purely scientific question, but will need some form ofbroader stakeholder engagement and the commitment to specific protection goals for human health and the environment", concludes Dolezel.

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The EU not ready for the release of Gene drive organisms into the environment - Science Codex

The science of changing the traits of plants in order to give rise to a desired trait is called plant breeding. CRISPR refers to clustered regularly interspaced short palindromic repeat and case 9 associated protein system is the plant breeding innovation that uses site-directed nucleases used to target and transform DNA with accuracy. It can be applied in a wide range of uses such as biological research, development od crops & animals and human health.

Increasing importance for sustainable crop production drives the growth of plant breeding and the CRISPR plants market. Besides this, a fall in the cost of genomic solutions also drives the growth. However, high costs involved in modern breeding techniques as compared to conventional breeding techniques restricts the growth. A uprurging investments from seed companies and supportive regulations for molecular breeding is expected to boost the growth of plant breeding and CRISPR plants market.

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Plant Breeding and CRISPR Plants Market Summary Comprises Key Regions, Growth and Segmentation Till 2027 - Jewish Life News

Something's been brewing in the health care sector, the second-largest group in the S&P 500. Since the start of the second quarter, the Health Care Select Sector SPDR (NYSE: XLV) is one of the top asset-gathering exchange traded funds.

The iShares Nasdaq Biotechnology ETF (NASDAQ: IBB), thanks to plenty of help from Gilead Sciences (NASDAQ: GILD), is up almost 14% over the past month. Much of the recent biotechnology ebullience is attributable to progress on the coronavirus treatment and vaccine front.

IBB is the largest biotechnology ETF by assets, but it's not the only one delivering impressive returns in recent weeks. Here's a trio of biotech ETFs that are knocking the cover off the ball.

Virtus LifeSci Biotech Clinical Trials ETF (BBC)

The Virtus LifeSci Biotech Clinical Trials ETF (NYSE: BBC) tracks the LifeSci Biotechnology Clinical Trials Index. That benchmark is a collection of companies with drugs and therapies in clinical trials, which means BBC is at the right place at the right time in the battle to quash coronavirus.

BBC's holdings are basically equally weighted, but that doesn't distract from the fact that Moderna (NASDAQ: MRNA) is the fund's top holding. Overall, BBC holds about 90 stocks, most of which dwell at the lower end of large-cap territory or are mid- or small-cap names.

In addition to Moderna, several other BBC components have coronavirus treatment exposure and roughly a dozen are credible takeover targets. That's enough to have BBC up nearly 27% over the past month.

ARK Genomic Revolution ETF (ARKG)

In the health care space, the ARK Genomic Revolution ETF (CBOE: ARKG) has been one of the best-performing funds for several years, trouncing traditional health care and biotechnology ETFs for several years, speaking to the capabilities of the fund's active managers.

ARKG typically holds 30 to 50 stocks and currently holds 34, several of which are coronavirus plays and that doesn't even begin to underscore the fund's virus detection exposure, which highlights ARKG's deep CRISPR exposure.

As government officials re-open the US, testing for the COVID-19 virus will be a critical step. A new CRISPR-based test called DNA Endonuclease-Targeted CRISPR Trans Reporter (DETECTR) could help speed the process along, ARK analyst Ali Aurman saidin a recent note.

ARKG is higher by nearly 32% over the past month.

Principal Healthcare Innovators Index ETF (BTEC)

The Principal Healthcare Innovators Index ETF (NASDAQ: BTEC) is a departure from the other funds mentioned here because it's not drug/therapy-centric. Rather, BTEC tilts more toward medical device, equipment and life sciences firms.

The fund invests in companies that are leading the charge toward innovative solutions, rather than spending money on marketing and distribution, according to Principal.

BTEC's nearly 210 holdings are considered research and development-intensive companies and the fund's methodology screens out companies with negative or inconsistent earnings. The fund is higher by almost 25% over the past month.

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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3 Biotech ETFs Up 10% Or More Over The Last Month - Benzinga

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

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

-Began treating patients in clinical trial of CTX120, targeting B-cell maturation antigen (BCMA)-

-IND and CTA approved for CTX130, wholly-owned allogeneic CAR-T cell therapy targeting CD70-

-Received $25 million milestone payment from DMD/DM1 collaboration with Vertex in April 2020-

ZUG, Switzerland and CAMBRIDGE, Mass., April 28, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2020.

"We made substantial progress in the last quarter despite the challenges posed by COVID-19. We are now progressing five cell therapy clinical trials in parallel targeting hemoglobinopathies and various cancers," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Our CTX001 and CTX110 programs continue to enroll patients, and we expect to report data for these programs this year. We are pleased to have begun treating patients in our CTX120 trial for the treatment of relapsed or refractory multiple myeloma and we expect to begin treating patients in our CTX130 trial in renal cell carcinoma and T-cell and B-cell hematologic malignancies in the second half of this year. Despite these unprecedented times, we continue to execute on our programs and we remain focused on our commitment to patients and their families."

Recent Highlights and Outlook

Title: Dual Guide CRISPR/Cas9 Editing of the CCR5 Gene Provides Complete Protection Against HIV in Humanized Mouse Models (abstract #1046)Session Title: Gene Targeting and Gene CorrectionDate and Time: Thursday, May 14, 2020; 5:30 PM - 6:30 PM

Title: Multiplexing of Up to 10 Gene Edits Using CRISPR/Cas9 to Generate CAR-T Cells with Improved Function (abstract #1151)Session Title: Cancer - Immunotherapy, Cancer VaccinesDate and Time: Thursday, May 14, 2020; 5:30 PM - 6:30 PM

First Quarter 2020 Financial Results

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 patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.

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

About CTX110CTX110 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19, or CD19, for the treatment of CD19+ malignancies. A wholly-owned asset of CRISPR Therapeutics, CTX110 is being investigated in a clinical trial designed to assess the safety and efficacy of CTX110 for the treatment of relapsed or refractory B-cell malignancies. The multi-center, open-label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110.

About CTX120CTX120 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting B-cell maturation antigen, or BCMA. A wholly-owned asset of CRISPR Therapeutics, CTX120 is being investigated in a clinical trial designed to assess the safety and efficacy of CTX120 for the treatment of relapsed or refractory multiple myeloma. The multi-center, open-label clinical trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

About CTX130CTX130 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. A wholly-owned asset ofCRISPR Therapeutics, CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies.

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

CRISPR Forward-Looking StatementThis press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics' expectations about any or all of the following: (i) potential impacts due to the coronavirus pandemic; (ii) the safety, efficacy and clinical progress of CRISPR Therapeutics' various clinical programs including CTX001, CTX110, CTX120 and CTX130; (iii) the status of clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof, the timing of commencement of clinical trials and the expected timing of data releases), development timelines and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators; (iv) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; (v) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (vi) the sufficiency of CRISPR Therapeutics' cash resources; (vii) the expected benefits of CRISPR Therapeutics' collaborations; and (viii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words "believes," "anticipates," "plans," "expects" and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential impacts due to the coronavirus pandemic such as (x) delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; (y) the timing and progress of clinical trials, preclinical studies and other research and development activities; and (z) the overall impact of the coronavirus pandemic on its business, financial condition and results of operations; the potential for initial and preliminary data from any clinical trial (including CTX001, CTX110, CTX120 and CTX130) not to be indicative of final trial results; the risk that the initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of results from the full planned study population; the outcomes for each CRISPR Therapeutics' planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics' internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics' product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics' product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics' technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics' most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact:Susan Kimsusan.kim@crisprtx.com

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

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CRISPR Therapeutics Provides Business Update and Reports First Quarter 2020 Financial Results - Benzinga

The first manned experiments were carried out in China with CRISPR gene editing technology. In a study with cancer patients, the treatment was shown to be safe and permanent.

Scientists and researchers continue to work on CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a gene editing technology. The first manned experiments with this gene editing technology were completed.

In these first experiments using CRISPR technology, quite promising developments took place. In the experiment, it was revealed by the researchers that the treatment was safe and permanent.

Cancer treatment with CRISPR:A team led by You Lu from West China Hospital applied CRISPR to take the immune cells of people with lung cancer to neutralize a gene called PD-1. The PD-1 protein usually sends signals to the immune cells to hold the attacks from the bodys own tissues, but the active PD-1 also extends the gates to spread the cancer.

The team then injected each participant with a modified version of their own immune cells. The participants experienced quite mild side effects. In addition, potentially dangerous gene mutations from gene regulation, the greatest fear of researchers, were very limited.

These cells, regulated by CRISPR, remained in the blood for at least four weeks, and this also showed that the effects of the strategy could be permanent, but this experiment in China was conducted with only 12 people with cancer. The authors of the article called for more comprehensive studies with newer gene editing systems. Many CRISPR based experiments guided this study, some of which have already been reported.

Read this article:
The Treatments Carried Out With CRISPR Have Been Proved To Be Safe For People - Somag News

BAILARD, INC. bought a fresh place in CRISPR Therapeutics AG (NASDAQ:CRSP). The institutional investor bought 53.0 thousand shares of the stock in a transaction took place on 3/31/2020. In another most recent transaction, which held on 3/31/2020, FRONTIER CAPITAL MANAGEMENT CO. bought approximately 38.5 thousand shares of CRISPR Therapeutics AG In a separate transaction which took place on 3/31/2020, the institutional investor, IMA WEALTH, INC. bought 38.3 thousand shares of the companys stock. The total Institutional investors and hedge funds own 54.30% of the companys stock.

In the most recent purchasing and selling session, CRISPR Therapeutics AG (CRSP)s share price decreased by -0.99 percent to ratify at $52.00. A sum of 1315673 shares traded at recent session and its average exchanging volume remained at 993.63K shares. The 52-week price high and low points are important variables to concentrate on when assessing the current and prospective worth of a stock. CRISPR Therapeutics AG (CRSP) shares are taking a pay cut of -29.73% from the high point of 52 weeks and flying high of 60.99% from the low figure of 52 weeks.

CRISPR Therapeutics AG (CRSP) shares reached a high of $53.615 and dropped to a low of $49.33 until finishing in the latest session at $53.25. Traders and investors may also choose to study the ATR or Average True Range when concentrating on technical inventory assessment. Currently at 3.32 is the 14-day ATR for CRISPR Therapeutics AG (CRSP). The highest level of 52-weeks price has $74.00 and $32.30 for 52 weeks lowest level. After the recent changes in the price, the firm captured the enterprise value of $2.36B, with the price to earnings ratio of 53.55. The liquidity ratios which the firm has won as a quick ratio of 17.30, a current ratio of 17.30 and a debt-to-equity ratio of 0.00.

Having a look at past record, were going to look at various forwards or backwards shifting developments regarding CRSP. The firms shares fell -0.76 percent in the past five business days and grew 17.41 percent in the past thirty business days. In the previous quarter, the stock fell -4.06 percent at some point. The output of the stock increased 15.94 percent within the six-month closing period, while general annual output gained 29.39 percent. The companys performance is now negative at -14.62% from the beginning of the calendar year.

According to WSJ, CRISPR Therapeutics AG (CRSP) obtained an estimated Overweight proposal from the 17 brokerage firms currently keeping a deep eye on the stock performance as compares to its rivals. 2 equity research analysts rated the shares with a selling strategy, 4 gave a hold approach, 11 gave a purchase tip, 0 gave the firm a overweight advice and 0 put the stock under the underweight category. The average price goal of one year between several banks and credit unions that last year discussed the stock is $74.25.

CMS Energy Corporation (CMS) shares on Wednesdays trading session, dropped -3.29 percent to see the stock exchange hands at $57.26 per unit. Lets a quick look at companys past reported and future predictions of growth using the EPS Growth. EPS growth is a percentage change in standardized earnings per share over the trailing-twelve-month period to the current year-end. The company posted a value of $2.28 as earning-per-share over the last full year, while a chance, will post $2.85 for the coming year. The current EPS Growth rate for the company during the year is 3.00% and predicted to reach at 8.21% for the coming year. In-depth, if we analyze for the long-term EPS Growth, the out-come was 5.60% for the past five years and the scenario is totally different as the current prediction is 7.30% for the next five year.

The last trading period has seen CMS Energy Corporation (CMS) move -17.22% and 24.40% from the stocks 52-week high and 52-week low prices respectively. The daily trading volume for CMS Energy Corporation (NYSE:CMS) over the last session is 2.64 million shares. CMS has attracted considerable attention from traders and investors, a scenario that has seen its volume drop -5.36% compared to the previous one.

Investors focus on the profitability proportions of the company that how the company performs at profitability side. Return on equity ratio or ROE is a significant indicator for prospective investors as they would like to see just how effectively a business is using their cash to produce net earnings. As a return on equity, CMS Energy Corporation (NYSE:CMS) produces 13.80%. Because it would be easy and highly flexible, ROI measurement is among the most popular investment ratios. Executives could use it to evaluate the levels of performance on acquisitions of capital equipment whereas investors can determine that how the stock investment is better. The ROI entry for CMSs scenario is at 5.80%. Another main metric of a profitability ratio is the return on assets ratio or ROA that analyses how effectively a business can handle its assets to generate earnings over a duration of time. CMS Energy Corporation (CMS) generated 2.60% ROA for the trading twelve-month.

Volatility is just a proportion of the anticipated day by day value extendthe range where an informal investor works. Greater instability implies more noteworthy benefit or misfortune. After an ongoing check, CMS Energy Corporation (CMS) stock is found to be 3.27% volatile for the week, while 4.28% volatility is recorded for the month. The outstanding shares have been calculated 287.81M. Based on a recent bid, its distance from 20 days simple moving average is -3.57%, and its distance from 50 days simple moving average is -6.24% while it has a distance of -7.97% from the 200 days simple moving average.

The Williams Percent Range or Williams %R is a well-known specialized pointer made by Larry Williams to help recognize overbought and oversold circumstances. CMS Energy Corporation (NYSE:CMS)s Williams Percent Range or Williams %R at the time of writing to be seated at 97.98% for 9-Day. It is also calculated for different time spans. Currently for this organization, Williams %R is stood at 98.20% for 14-Day, 67.39% for 20-Day, 51.47% for 50-Day and to be seated 51.47% for 100-Day. Relative Strength Index, or RSI(14), which is a technical analysis gauge, also used to measure momentum on a scale of zero to 100 for overbought and oversold. In the case of CMS Energy Corporation, the RSI reading has hit 45.34 for 14-Day.

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Solid Income Opportunity With CRISPR Therapeutics AG (CRSP) and CMS Energy Corporation (CMS) - BOV News

CAMBRIDGE, Mass., April 30, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, will present its first quarter 2020 financial results and operational highlights in a conference call on May 7, 2020 at 8 a.m. ET.

To join the call:

A replay of the call will be available through the Events and Presentations page of the Investors & Media section of the companys website at http://www.intelliatx.com, beginning on May 7, 2020 at 12 p.m. ET.

About Intellia Therapeutics

Intellia Therapeutics is 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 about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

Intellia Contacts:

Investor Contact:Lina LiAssociate Director, Investor Relations+1 857-706-1612lina.li@intelliatx.com

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

Continued here:
Intellia Therapeutics to Hold Conference Call to Discuss First Quarter 2020 Earnings and Company Updates - Yahoo Finance