The research study encompasses nitty-gritty of the Genetic Testing Market, from future prospects to the competitive scenario, comprehensively. With DROT analysis and Porters Five Forces analysis, the authors have presented a platter of factors influencing the growth of Genetic Testing Market. Further, the Genetic Testing market has been broken down into various segments, regions, end-uses and players to provide a clear picture of the present market situation to the readers.

PMR, in its recently published market research report, provides a detailed analysis of the various factors that are projected to impact the growth of the Genetic Testing market during the forecast period 2020. According to the study, the Genetic Testing market is set to register a CAGR growth of XX% through the assessment period and attain a market value of ~US$XX by the end of 2029.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/3175

The Genetic Testing Market research covers an exhaustive analysis of the following data:

The Genetic Testing Market research addresses critical questions, such as

And many more

The Genetic Testing Market study encloses the projection size of the market both in terms of value (Mn/Bn US$) and volume (x units). With bottom-up and top-down approaches, the report predicts the viewpoint of various domestic vendors in the whole market and offers the market size of the Genetic Testing Market. The analysts of the report have performed extensive primary and secondary research to scrutinize the key players and their market share. Further, different trusted sources were roped in to gather numbers, subdivisions, revenue and shares.

Get Access To TOC Covering 200+ Topics athttps://www.persistencemarketresearch.co/toc/3175

Some of the major companies dealing in genetic testing market are Abbott Laboratories, BioRad Laboratories, Abbott Molecular Inc., AutoGenomics Inc., Celera Group, PerkinElmer Inc., Quest Diagnostics Inc., ELITech Group, Roche Diagnostics Corp., Applied Biosystems Inc., Roche Molecular Diagnostics Inc., Transgenomic Inc. and others.

Key points covered in the report

In order to get a strategic overview of the market,Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/3175

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PMR is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

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Genetic Testing Market : Future Innovation Strategies, Growth & Profit Analysis, Forecast by 2020 - Jewish Life News

Recommendation and review posted by Bethany Smith

Global Genetic Testing Devices Industry Market provides holistic and comprehensive Genetic Testing Devices insights with competitive landscape analysis, revenue share and market status from 2019-2026. The complete business profiles of top Genetic Testing Devices players, product portfolio, specifications, regional and country level analysis. The production capacity, pricing structures, SWOT analysis, products and variety of Genetic Testing Devices Industry applications are covered. The present mergers & acquisitions by key Genetic Testing Devices players, industry plans & policies, product launches and development aspects are mentioned. The geographical segmentation of Genetic Testing Devices Market conducts analysis of regions namely North America, Europe, Asia-Pacific, MEA, South America and rest of the globe.

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The Genetic Testing Devices Market value, volume analysis with future prospect is conducted based on technological advancements and latest developments in this industry. This report studies the sales, production, import/export status, demand, supply and gross margin. The statistical and analytical analysis of Genetic Testing Devices Market is conducted to provide qualitative and quantitative market insights. The profiling of top Genetic Testing Devices Players is covered to estimate market size, growth, sales data and forecast study. The company profiles, sales data, product portfolio, regional presence and Genetic Testing Devices market competition is stated.

Top Market Vendors Analysed Are As Follows:

LuminexQuest DiagnosticsElitechPerkinElmerQiagenEKF DiagnosticsBiocartisMyriad GeneticsInterpace DiagnosticsElitechSeegeneWaferGen BiosystemsRoche DiagnosticsCepheidBioRad LaboratoriesBioMerieuxAbbott LaboratoriesIntegraGen

Market Segment By Product Types Are As Follows:

Type 1Type 2Type 3Type 4Type 5

Market Applications Are Mentioned Below:

Application 1Application 2Application 3Application 4Application 5

Our latest study will provide valuable insights on Genetic Testing Devices Market and detailed analysis with financial data, production process and pricing analysis. The marketing strategies adopted by leading Genetic Testing Devices Industry players, sales volume, value and market share is stated. The future outlook and prospects in Genetic Testing Devices Market, macroeconomic policies, industry development trends, sales channel and market dynamics is covered. The cost analysis, major downstream buyers, traders, distributors and dealers are stated. The consumption ratio, market status, volume, and emerging Genetic Testing Devices players analysis is studied.The market size estimation, data sources, research findings, data triangulation and consumer needs are studied deeply.

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The strategic assessment of Genetic Testing Devices Market, trading policies, CAGR value, raw material study, distribution channel is represented in graphical format. The growth drivers, challenges, investment opportunities, and regional analysis is stated. The market players, trends, scope is explained. The market size in US $ Mn and Y-o-Y growth rate which opportunity analysis is explained. The end use details, historical analysis, price trends, revenue and market share Y-o-Y growth of key players is explained. The strategic recommendations on Genetic Testing Devices Industry, forecast growth areas, product analysis, and downstream buyers are analysed.

The key objectives of the study are:

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Genetic Testing Devices Market Size ,Growth Opportunities & Regional Growth Forecast by Types, Applications - Jewish Life News

Recommendation and review posted by Bethany Smith

New Jersey, United States, The report titled, Preimplantation Genetic Testing Market Size and Forecast 2026 in Verified Market Research offers its latest report on the global Preimplantation Genetic Testing market that includes comprehensive analysis on a range of subjects like competition, segmentation, regional expansion, and market dynamics. The report sheds light on future trends, key opportunities, top regions, leading segments, the competitive landscape, and several other aspects of the Preimplantation Genetic Testing market. Get access to crucial market information. Market players can use the report back to peep into the longer term of the worldwide Preimplantation Genetic Testing market and convey important changes to their operating style and marketing tactics to realize sustained growth.

Global Preimplantation Genetic Testing Market was valued at USD 306.16 million in 2016 and is projected to reach USD 701.49 million by 2025, growing at a CAGR of 9.65% from 2017 to 2025.

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Top 10 Companies in the Global Preimplantation Genetic Testing Market Research Report:

Global Preimplantation Genetic Testing Market: Competitive Landscape

Competitive landscape of a market explains strategies incorporated by key players of the market. Key developments and shift in management in the recent years by players has been explained through company profiling. This helps readers to understand the trends that will accelerate the growth of market. It also includes investment strategies, marketing strategies, and product development plans adopted by major players of the market. The market forecast will help readers make better investments.

Global Preimplantation Genetic Testing Market: Drivers and Restrains

This section of the report discusses various drivers and restrains that have shaped the global market. The detailed study of numerous drivers of the market enable readers to get a clear perspective of the market, which includes market environment, government policies, product innovations, breakthroughs, and market risks.

The research report also points out the myriad opportunities, challenges, and market barriers present in the Global Preimplantation Genetic Testing Market. The comprehensive nature of the information will help the reader determine and plan strategies to benefit from. Restrains, challenges, and market barriers also help the reader to understand how the company can prevent itself from facing downfall.

Global Preimplantation Genetic Testing Market: Segment Analysis

This section of the report includes segmentation such as application, product type, and end user. These segmentations aid in determining parts of market that will progress more than others. The segmentation analysis provides information about the key elements that are thriving the specific segments better than others. It helps readers to understand strategies to make sound investments. The Global Preimplantation Genetic Testing Market is segmented on the basis of product type, applications, and its end users.

Global Preimplantation Genetic Testing Market: Regional Analysis

This part of the report includes detailed information of the market in different regions. Each region offers different scope to the market as each region has different government policy and other factors. The regions included in the report are North America, South America, Europe, Asia Pacific, and the Middle East. Information about different region helps the reader to understand global market better.

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Table of Content

1 Introduction of Preimplantation Genetic Testing Market

1.1 Overview of the Market 1.2 Scope of Report 1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining 3.2 Validation 3.3 Primary Interviews 3.4 List of Data Sources

4 Preimplantation Genetic Testing Market Outlook

4.1 Overview 4.2 Market Dynamics 4.2.1 Drivers 4.2.2 Restraints 4.2.3 Opportunities 4.3 Porters Five Force Model 4.4 Value Chain Analysis

5 Preimplantation Genetic Testing Market, By Deployment Model

5.1 Overview

6 Preimplantation Genetic Testing Market, By Solution

6.1 Overview

7 Preimplantation Genetic Testing Market, By Vertical

7.1 Overview

8 Preimplantation Genetic Testing Market, By Geography

8.1 Overview 8.2 North America 8.2.1 U.S. 8.2.2 Canada 8.2.3 Mexico 8.3 Europe 8.3.1 Germany 8.3.2 U.K. 8.3.3 France 8.3.4 Rest of Europe 8.4 Asia Pacific 8.4.1 China 8.4.2 Japan 8.4.3 India 8.4.4 Rest of Asia Pacific 8.5 Rest of the World 8.5.1 Latin America 8.5.2 Middle East

9 Preimplantation Genetic Testing Market Competitive Landscape

9.1 Overview 9.2 Company Market Ranking 9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview 10.1.2 Financial Performance 10.1.3 Product Outlook 10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Highlights of Report

About Us:

Verified market research partners with clients to provide insight into strategic and growth analytics; data that help achieve business goals and targets. Our core values include trust, integrity, and authenticity for our clients.

Analysts with high expertise in data gathering and governance utilize industry techniques to collate and examine data at all stages. Our analysts are trained to combine modern data collection techniques, superior research methodology, subject expertise and years of collective experience to produce informative and accurate research reports.

Contact Us:

Mr. Edwyne Fernandes Call: +1 (650) 781 4080 Email: [emailprotected]

TAGS: Preimplantation Genetic Testing Market Size, Preimplantation Genetic Testing Market Growth, Preimplantation Genetic Testing Market Forecast, Preimplantation Genetic Testing Market Analysis, Preimplantation Genetic Testing Market Trends, Preimplantation Genetic Testing Market

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Preimplantation Genetic Testing Market 2020 Booming by Size, Revenue, Trend and Top Companies 2026 - Instant Tech News

Recommendation and review posted by Bethany Smith

Spotting the next opportunity before it breaks out has become less of a hobby and more of a total obsession as Ive become a more experienced investor. Theres always money to be made in growth sectors I just wish I had more capital on hand to take advantage of the good companies I research!

Caveat emptor, of course. With fewer daily trades in AIM-listed companies than on the FTSE 100 or FTSE 250, it can be more difficult to sell up if you want out in a hurry.

But having picked apart their finances I think these smaller UK-listed companies will be among the best performers of 2020.

53m market cap Shearwater Group (LSE:SWG) is an AIM-listed cybersecurity, infosec, and IT risk management business with 400 customers across FTSE 350 firms, government departments, and US Fortune 500 companies.

One of the few British independent businesses in the cybersecurity field, Shearwater has been plugging away nicely in recent years, buying up profitable companies to add to their group and cross-selling products between them to generate better revenue, which I like.

The board, now led by CEO Phil Higgins, has focused on a buy, focus, grow strategy, adding penetration and threat testing firm Pentest to its stable in April 2019 for 7.4m.

Behind the scenes there is a lot of product development going on, such as a scanning-as-a-service product that uses machine learning to audit sensitive data on external drives, in cloud applications like Office 365, and in cloud storage, such as Google Drive.

The second half of 2019 represented the maiden profit period for SWG, with revenue swinging up 11% to 16.3m, earnings of 1m and adjusted earnings per share of 2.23p.

News has gone a little quiet in recent weeks, so Id expect the next major update to send the share price soaring.

Manchester genetic testing firm YourGene Health (LSE:YGEN) develops non-invasive products for male fertility, and pre-natal screening for cystic fibrosis and other genetic disorders.

What caught my eye about the 88.2m market cap business is their planned expansion to extend genetic testing into cancer detection and prevention. Acquiring Elucigene in April 2019 means the company has been able to expand into the US market and launch its first oncology product.

Like Shearwater, YGEN has just posted its first positive earnings. Revenue is growing strongly, up from 0.1m to 8.8m over the last five years, and directors in the company continue to buy more stock to raise their personal stakes, which bodes well.

CEO Lyn Rees highlighted the firms strong prospects in recent half year results to 30 September 2019. Revenue was up 97% with excellent organic growth of 56%, gross profits 141% higher, and net cash significantly improved to 3.6m compared to net debt of 12.8m over the previous half.

I usually seek positive language in regulatory updates, backed by strong financial fundamentals, and YGEN has this in spades. I remain convinced we have a very significant opportunity ahead of us, Rees noted, adding, we are confident in our outlook for the year ahead and very excited about the prospects for further growth over the following years.

Savvy investors like you wont want to miss out on this timely opportunity

Heres your chance to discover exactly what has got our MotleyFoolUK analyst all fired up about this pure-play online business.

Not only does this company enjoy a dominant market-leading position

But its capital-light, highly scalable business model has been helping it deliver consistently high sales, astounding near-70% margins, and rising shareholder returns in fact, in 2019 alone it returned a whopping 151.1m to shareholders in dividends and buybacks!

And heres the really exciting part

We think now could be the perfect time for you to start building your own stake in this exceptional businessespecially given the two potentially lucrative expansion opportunities on the horizon that our analyst has highlighted.

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Tom Rodgers has no current position in the shares mentioned. The Motley Fool UK has no position in any of the shares mentioned. Views expressed on the companies mentioned in this article are those of the writer and therefore may differ from the official recommendations we make in our subscription services such as Share Advisor, Hidden Winners and Pro. Here at The Motley Fool we believe that considering a diverse range of insights makes us better investors.

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I'd buy these 2 micro-caps before they soar in 2020 - Motley Fool UK

Recommendation and review posted by Bethany Smith

New Jersey, United States, The report titled, Direct-To-Consumer (DTC) Genetic Testing Market Size and Forecast 2026 in Verified Market Research offers its latest report on the global Direct-To-Consumer (DTC) Genetic Testing market that includes comprehensive analysis on a range of subjects like competition, segmentation, regional expansion, and market dynamics. The report sheds light on future trends, key opportunities, top regions, leading segments, the competitive landscape, and several other aspects of the Direct-To-Consumer (DTC) Genetic Testing market. Get access to crucial market information. Market players can use the report back to peep into the longer term of the worldwide Direct-To-Consumer (DTC) Genetic Testing market and convey important changes to their operating style and marketing tactics to realize sustained growth.

Direct-to-Consumer (DTC) Genetic Testing Market was valued at USD 789.92 Million in 2018 and is projected to reach USD 2,361.12 Billion by 2026, growing at a CAGR of 14.59% from 2019 to 2026.

Get | Download Sample Copy @https://www.verifiedmarketresearch.com/download-sample/?rid=25584&utm_source=ITN&utm_medium=002

Top 10 Companies in the Global Direct-To-Consumer (DTC) Genetic Testing Market Research Report:

Global Direct-To-Consumer (DTC) Genetic Testing Market: Competitive Landscape

Competitive landscape of a market explains strategies incorporated by key players of the market. Key developments and shift in management in the recent years by players has been explained through company profiling. This helps readers to understand the trends that will accelerate the growth of market. It also includes investment strategies, marketing strategies, and product development plans adopted by major players of the market. The market forecast will help readers make better investments.

Global Direct-To-Consumer (DTC) Genetic Testing Market: Drivers and Restrains

This section of the report discusses various drivers and restrains that have shaped the global market. The detailed study of numerous drivers of the market enable readers to get a clear perspective of the market, which includes market environment, government policies, product innovations, breakthroughs, and market risks.

The research report also points out the myriad opportunities, challenges, and market barriers present in the Global Direct-To-Consumer (DTC) Genetic Testing Market. The comprehensive nature of the information will help the reader determine and plan strategies to benefit from. Restrains, challenges, and market barriers also help the reader to understand how the company can prevent itself from facing downfall.

Global Direct-To-Consumer (DTC) Genetic Testing Market: Segment Analysis

This section of the report includes segmentation such as application, product type, and end user. These segmentations aid in determining parts of market that will progress more than others. The segmentation analysis provides information about the key elements that are thriving the specific segments better than others. It helps readers to understand strategies to make sound investments. The Global Direct-To-Consumer (DTC) Genetic Testing Market is segmented on the basis of product type, applications, and its end users.

Global Direct-To-Consumer (DTC) Genetic Testing Market: Regional Analysis

This part of the report includes detailed information of the market in different regions. Each region offers different scope to the market as each region has different government policy and other factors. The regions included in the report are North America, South America, Europe, Asia Pacific, and the Middle East. Information about different region helps the reader to understand global market better.

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Table of Content

1 Introduction of Direct-To-Consumer (DTC) Genetic Testing Market

1.1 Overview of the Market 1.2 Scope of Report 1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining 3.2 Validation 3.3 Primary Interviews 3.4 List of Data Sources

4 Direct-To-Consumer (DTC) Genetic Testing Market Outlook

4.1 Overview 4.2 Market Dynamics 4.2.1 Drivers 4.2.2 Restraints 4.2.3 Opportunities 4.3 Porters Five Force Model 4.4 Value Chain Analysis

5 Direct-To-Consumer (DTC) Genetic Testing Market, By Deployment Model

5.1 Overview

6 Direct-To-Consumer (DTC) Genetic Testing Market, By Solution

6.1 Overview

7 Direct-To-Consumer (DTC) Genetic Testing Market, By Vertical

7.1 Overview

8 Direct-To-Consumer (DTC) Genetic Testing Market, By Geography

8.1 Overview 8.2 North America 8.2.1 U.S. 8.2.2 Canada 8.2.3 Mexico 8.3 Europe 8.3.1 Germany 8.3.2 U.K. 8.3.3 France 8.3.4 Rest of Europe 8.4 Asia Pacific 8.4.1 China 8.4.2 Japan 8.4.3 India 8.4.4 Rest of Asia Pacific 8.5 Rest of the World 8.5.1 Latin America 8.5.2 Middle East

9 Direct-To-Consumer (DTC) Genetic Testing Market Competitive Landscape

9.1 Overview 9.2 Company Market Ranking 9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview 10.1.2 Financial Performance 10.1.3 Product Outlook 10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Highlights of Report

About Us:

Verified market research partners with clients to provide insight into strategic and growth analytics; data that help achieve business goals and targets. Our core values include trust, integrity, and authenticity for our clients.

Analysts with high expertise in data gathering and governance utilize industry techniques to collate and examine data at all stages. Our analysts are trained to combine modern data collection techniques, superior research methodology, subject expertise and years of collective experience to produce informative and accurate research reports.

Contact Us:

Mr. Edwyne Fernandes Call: +1 (650) 781 4080 Email: [emailprotected]

TAGS: Direct-To-Consumer (DTC) Genetic Testing Market Size, Direct-To-Consumer (DTC) Genetic Testing Market Growth, Direct-To-Consumer (DTC) Genetic Testing Market Forecast, Direct-To-Consumer (DTC) Genetic Testing Market Analysis, Direct-To-Consumer (DTC) Genetic Testing Market Trends, Direct-To-Consumer (DTC) Genetic Testing Market

Original post:
Direct-To-Consumer (DTC) Genetic Testing Market 2020 Booming by Size, Revenue, Trend and Top Companies 2026 - Instant Tech News

Recommendation and review posted by Bethany Smith

Ample Market Research added by latest report demonstrates that the global Genetic Testing Market will showcase a steady CAGR in the coming years. The research report includes a thorough analysis of market drivers, restraints, threats, and opportunities. It addresses the lucrative investment options for the players in the coming years. Analysts have offered market estimates at a global and regional level

This report is based on synthesis, analysis, and interpretation of information gathered regarding the target market from various sources. Our analysts have analyzed the information and data and gained insights using a mix of primary and secondary research efforts with the primary objective to provide a holistic view of the market. In addition, an in-house study has been made of the global economic conditions and other economic indicators and factors to assess their respective impact on the market historically, as well as the current impact in order to make informed forecasts about the scenarios in future.

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Top Key Players Present in Genetic Testing Market Are : iagen N.V., Abbott Laboratories, F. Hoffmann-La Roche Ltd, Quest Diagnostics, Illumina, Inc., Natera, Inc., bioMerieux S.A., Myriad Genetics, Luminex Corporation, Bio-Rad Laboratories, Laboratory Corporations of America Holdings, Cepheid (Danaher Corporation), and niche players

Different leading key players have been profiled in this research report to get a clear idea of successful strategies carried out by top-level companies. On the basis of geographical segmentation, the Genetic Testing Market has been fragmented across several regions. This Market research report highlights those leading players who are planning to expand opportunities in the global market.

Genetic Testing Market is presented to the readers as a holistic snapshot of the competitive landscape within the given forecast period. It presents a comparative detailed analysis of the all regional and player segments, offering readers a better knowledge of where areas in which they can place their existing resources and gauging the priority of a particular region in order to boost their standing in the global market.

The Genetic Testing Market is gaining pace and businesses have started understanding the benefits of analytics in the present day highly dynamic business environment. The market has witnessed several important developments over the past few years, with mounting volumes of business data and the shift from traditional data analysis platforms to self-service business analytics being some of the most prominent ones.

For the future period, sound forecasts on market value and volume are offered for each type and application. In the same period, the report also provides a detailed analysis of market value and consumption for each region. These insights are helpful in devising strategies for the future and take necessary steps. New project investment feasibility analysis and SWOT analysis are offered along with insights on industry barriers. Research findings and conclusions are mentioned at the end.

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Reasons to Purchase this Report:

1) To gain insightful analyses of the Genetic Testing Market and have a comprehensive understanding of the global market and its commercial landscape.

2) To assess the production processes, major issues, and solutions to mitigate the development liability.

3) To understand the most affecting driving and restraining forces in the Genetic Testing Market and its impact on the global market.

4) Learn about the market policies that are being adopted by prominent organizations.

5) It provides detailed analysis of changing market trends, current and future technologies used, and various strategies adopted by leading players of the Genetic Testing market.

6)To conclude, the Genetic Testing Market report will provide the clients with a high-yielding market analysis assisting them to understand the market status and come up with new market avenues to capture hold of the market share.

If you have any special requirements, please let us know and we will offer you the report at customized price.

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Table of Content:

Chapter 1 Market Overview

Chapter 2 Industry Chain

Chapter 3 Environmental Analysis

Chapter 4 Market Segmentation by Type

Chapter 5 Market Segmentation by Application

Chapter 6 Market Segmentation by Region

Chapter 7 Market Competitive

Chapter 8 Major Vendors

Chapter 9 Conclusion

You can also get individual chapter wise section or region wise report version like North America, Europe or Asia or Country like US, UK, china and other.

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Know the Future Opportunities of Genetic Testing Market and its detail analysis by Focusing Top Companies like iagen NV, Abbott Laboratories, F....

Recommendation and review posted by Bethany Smith

NEW YORK, Feb. 13, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") announced today that presentations from its targeted conditioning portfolio have been accepted for presentation at the 2020 Transplantation & Cellular Therapy (TCT) Meetings, which brings together thousands of transplant professionals from over 500 transplant centers worldwide. TCT is being held February 19-23, 2020 at the Marriott World Center in Orlando, Florida. Notably, data from the pivotal Phase 3 SIERRA trial of Iomab-B have been selected for an oral presentation.

"We are excited that Iomab-B and the SIERRA trial have once again been selected as an oral presentation at TCT," said Dr. Mark Berger, Chief Medical Officer of Actinium. "We look forward to highlighting the potential benefit that Iomab-B can provide to a patient population with active disease who are otherwise ineligible for BMT. We are confident these findings will be received with great enthusiasm. TCT, which assembles leading transplant physicians from top centers in the United States and worldwide, is the ideal venue to showcase the extremely encouraging findings from the SIERRA trial thus far. In addition, our other conference activities are expected to provide significant exposure for this important trial and invaluable interactions with BMT thought leaders. Through the SIERRA trial, we aspire to change the treatment paradigm for older patients with relapsed or refractory AML to make potentially curative BMT via Iomab-B the standard of care for this patient population that continues to have poor outcomes."

Actinium's TCT Presentations:

Late Breaking Oral Presentation:

Poster Presentation:

About the SIERRA TrialThe SIERRA trial (Study ofIomab-B inElderlyRelapse/RefractoryAcute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. Iomab-B is an ARC (Antibody Radiation-Conjugate) comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume. For more information, visit http://www.sierratrial.com.

About Transplantation & Cellular Therapy Meetings (TCT) TCT, formerly known as the BMT Tandem Meetings, are the combined annual meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).Each year the conference brings together several thousand investigators, clinicians, researchers, nurses and other allied health professionals from over 500 transplant centers from over 50 countries around a full scientific program focused on bone marrow transplant and cellular therapies.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3Study ofIomab-B inElderlyRelapsed orRefractoryAcute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans Vitzthum LifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com/

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Actinium to Highlight Targeted Conditioning Portfolio at 2020 Transplantation & Cellular Therapy Annual Meeting; Phase 3 SIERRA Trial Preliminary...

Recommendation and review posted by Bethany Smith

The calving season is in full swing for us, and our diary is filling up with numerous projects on the horizon.

e had veterinary students from UCD with us for a few weeks in January and there will be more visiting us every weekend in February to try their hands at calving cows, with more booked in for a few weeks in March.

We are all continuously learning so it's always a pleasure helping others to educate themselves.

In early January we were in Glasgow as I was asked to speak at the 30th annual Semex International Dairy Conference.

Semex is one of the leading global genetics companies, with over 1,100 bulls at stud selling in excess of 12m doses of semen globally every year. I was humbled by the response to my presentation, and myself and Paula left the event inspired.

The theme of the conference was 'Be Extraordinary' and the list of speakers lived up to that billing.

Market analyst Chris Walkland explained in great detail, backed up by 10 years of data, why 2020 will be a very positive year for global dairy markets.

Other speakers addressed why it is vital we as farmers communicate more with the public to counteract the anti-agriculture agenda.

I was particularly impressed by Dr Steven Larmer (genomics programme manager) and Jordan Leak (chief operations officer of Double A Dairy and Twin Ridge genetics).

Jordan farms 20,000 dairy cows in the US state of Idaho - Holstein, Jersey and crossbreds with a replacement rate at 28pc.

He also carries 20,000 replacement dairy stock, with all stock being genotyped using that information to make strategic breeding decisions.

It certainly seems to work for him as last year he sold 6,000 in-calf heifers to two clients in Asia, who demanded that all stock were genotyped and above a certain index.

Dr Larmer spoke about the information available to us through genomics. Fertility, milk production, health traits are all more accurately confirmed, but what really grabbed my attention was when he discussed the unknown.

Genotyping creates a wealth of information and currently what is holding it back is computer power to process the volume of data.

Steven was very adamant that within a short space of time computer power will become sufficient to process all the data.

This will unlock the genetic 'black box' to show us which cows produce less methane, require less antibiotics and ultimately ensure every genetic trait required for efficiency is considered.

The end goal is completely tailor-made breeding programmes for every dairy herd. Closing the conference Paul Larmer, CEO of Semex, summed up the last 30 years of genetics, also discussing the next decade ahead.

He said genotyped embryos will become the norm along with beef embryos being used on dairy cows, adding that GM dairy genetics is on the horizon. It's merely a case of which countries will accept GM genetics.

On the plane home, I found myself thinking why are more farmers not embracing genotyping?

We have been genotyping all females born on farm for five years now and when we look at the latest EBI proofs it certainly highlighted how it has helped us make more informed breeding decisions.

There is also a good return on investment - researchers say the payback is three to one with the cost of genotyping at a mere 22 per female being lower in Ireland than anywhere else in the world.

An estimated 15pc of genotyped stock have incorrect sires - in effect there are at least 250,000 dairy cows in Ireland with potentially incorrect parentage.

The reliability has increased further, and for those considering buying or selling dairy stock, it's surely a must-have piece of information.

Yet there are only 80,000 dairy females genotyped in Ireland. If we really want the Irish dairy herd to be highly efficient, we need to gather all available data.

It's back to calving for me now. For those of you on social media keep an eye out for the #FutureofFarming campaign over the next few weeks. Its something myself, Paula and the girls are delighted to be involved in for the year ahead.

Hopefully the spring will be kind to us all and we'll see maximum days at grass to keep the cows content.

Peter Hynes farms with his wife Paula in Aherla, Co Cork

Indo Farming

Continued here:
Peter Hynes: How a new wave of genetic data is set to transform dairy farming in the next decade - Farm Ireland

Recommendation and review posted by Bethany Smith

You know that awkward feeling when someone references a TV show or person you don't know but you smile and nod along because you want to seem in the loop? Yeah, that totally human experience happens in fitness too. Especially around relatively obscure terms like VO2 max, which you may have overheard in a locker room or name dropped by a trainer recentlyit's become a bit of a buzzword as of late. But what is VO2 max, you ask?

Basically, its the maximum amount of oxygen your body can take in and use during exercise at your 100-percent intensity, says Stacy Sims, PhD, exercise physiologist, and author of Roar: How to Match Your Food and Fitness to Your Female Physiology for Optimum Performance, Great Health, and a Strong, Lean Body for Life.

Its often considered the best measurement of someones cardiorespiratory fitness, and Sims notes that its a reflection of how fast your body can regenerate ATP (an energy-carrying molecule burned off as fuel during exercise) in your cells.

That's kind of heady, to be honest, so another way to think about VO2 max is that it's like your PR when it comes to consuming oxygen. In theory, the more O2 you can take in, the more energy you can expend for a longer period of time. It's why athletes are hyper-focused on increasing their own in an effort to optimize their performance.

Even if you're not a fitness pro, though, you can still benefit from training to boost your VO2 max for similar reasonsbigger, better gains! Keep reading for everything you need to know about VO2 max like how to figure out your own, how to improve it, and what's a good benchmark to aim for in the first place.

There's no "perfect score" for VO2 max. Sims says a desirable number really depends on your sport or main fitness activity. "When we look at elite values, top-end cross-country women sit around 65 to 70 ml/kg/min; runners are about 60 to 65ml/kg/min; cyclists are around 55 to 60 ml/kg/min."

Generally speaking, though, this VO2 max chart notes where optimal scores should fall depending on your age:

Jewelyn Butron

Testing VO2 max is where things get a little tricky. The gold standard is direct measurement in a lab setting, says Sims. This is known as a Cardiopulmonary Exercise Testing (CPET), and it involves wearing a mask and heart rate monitor thats hooked up to a treadmill or stationary bike. The mask is connected to a machine that measures the amount of oxygen you inhale, and the amount of carbon dioxide you exhale. During the test, you continue upping the intensity of your bike or tread until your oxygen consumption value reaches a steady state, even as exercise intensity increases, explains Sims. Thats your max. The final measurement is recorded in mL/kg/min. If you're really interested in finding your true VO2 max, see if any gyms or fitness studios near you offer the service.

This, of course, is not realistic for most people, which is why an easier (albeit not as precise) way to get an approximation of you VO2 max is by investing in a fitness tracker or smartwatch that'll estimate it for you based on your heart rate and exercise intensity after collecting your data for a period of time. For the average active adult, this is the best option. Some, like the FitBit Ionic, refer to VO2 max as your "cardio fitness score," while others, like the Apple Watch, list it simply as VO2 maxyou can find yours in the Apple Health app when you click through to all health data, fyi.

Garmin vvosport, Fitness/Activity Tracker with GPS and Heart Rate Monitoring, Slate

$115.19

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Your cardio fitness level isn't the only thing that plays a role. Sims notes that there are actually a number of internal and external factors that can have a major impact on your VO2 max:

If you have the ability to track your VO2 max regularly, it can be one way to determine your fitness level and improvement. The best way to give it a boost is through high-intensity interval training (HIIT), which helps work you body at high levels for a period of time, in order to help build up your aerobic capacity, says Sims.

Try this four-move HIIT workout video to start improving yours:

That said, while VO2 max is used in scientific research and with elite athletes, Sims doesn't recommend getting too hung up on it as a go-to performance or training marker. Instead, focus on challenging your body little by little in cardio workouts to get fitter and faster.

Original post:
What Is VO2 Max? What To Know, According To An Exercise Physiologist - Women's Health

Recommendation and review posted by Bethany Smith

The research report presents a detailed competitive analysis of the Non-Melanoma Skin Cancer Market 2019 market Share, Size, and Future scope 2026. This research report classifies the market by manufacturers, region, type, and applications.

The data presented in the graphical format gives a thorough understanding of the major players of Non-Melanoma Skin Cancer . The restraints and growth, industry plans, innovations, mergers, and acquisitions are covered in this report. The market is segmented based on key industry verticals like the product type, applications, and geographical regions.

Get a Sample Copy of the Report @ https://www.reportspedia.com/report/life-sciences/global-non-melanoma-skin-cancer-market-report-2019,-competitive-landscape,-trends-and-opportunities/28562 #request_sample

Key Players of Non-Melanoma Skin Cancer Report are:

OncothyreonVarian Medical SystemsLEO PharmaAqua PharmaceuticalsMedaIRX TherapeuticsMoberg PharmaEli Lilly and Co.Cannabis ScienceMylan PharmaceuticalMerck & Co.Boehringer IngelheimCellceutix Corp.Bristol Myers Squibb Co.BiofronteraElektaICADValeant PharmaceuticalsSun Pharma IndustriesGaldermaAlmirallGENEXTRAF. Hoffmann-la RocheNovartis International

Short Description of Non-Melanoma Skin Cancer Market 2019-2026:

The Non-Melanoma Skin Cancer market was valued t XX Million US$ in 2019 and is projected to reach XX Million US$ by 2026, at a CAGR of XX% during 2019-2026. The research report gives historic report from 2013-2018.

The market is segmented into below points:

Market by Type/Products:

Type 1Type 2Type 3

Market by Application/End-Use:

Application 1Application 2Application 3

Enquire or share your questions if any before the purchasing this report @ https://www.reportspedia.com/report/life-sciences/global-non-melanoma-skin-cancer-market-report-2019,-competitive-landscape,-trends-and-opportunities/28562 #inquiry_before_buying

Outline of the data covered in this study:

The market study covers the forecast Non-Melanoma Skin Cancer information from 2019-2026 and key questions answered by this report include:

In this study, the years considered to estimate the market size of Non-Melanoma Skin Cancer are as follows:

Historic Period: 2015-2019.

Base Year: 2019.

Estimated Year: 2020.

Forecast Year 2020 to 2026.

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

Table of contents:

For More TOC Content Continued,

Get A Sample Pdf Copy Of Table Of Content Describing Current Value And Volume Of The Market With All Other Essential Information @ https://www.reportspedia.com/report/life-sciences/global-non-melanoma-skin-cancer-market-report-2019,-competitive-landscape,-trends-and-opportunities/28562 #table_of_contents

Thanks A Million For Reading! You Can Also Request Custom Information Like Chapter-Wise Or Specific Region-Wise Study As Per The Given Specifications.

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Global Stem Cells Types, Technologies And Therapeutics Market Is Estimated To Expand At a Healthy CAGR in Upcoming year 2020-2026 - Jewish Life News

Recommendation and review posted by Bethany Smith

Penn State researchers have received a $2.8 million grant to investigate 3D bioprinting tissue for facial reconstructions, according to a Penn State news release.

The grant, from the National Institutes of Healths National Institute of Dental and Craniofacial Research, funds five years of research exploring methods for bioprinting face, mouth and skull tissue directly into patients during surgery, with the ultimate goal of developing a bioprinting technology, according to the release.

Craniomaxillofacial reconstruction currently presents challenges for doctors because it requires precisely stacking several different types of tissue. Penn States researchers hope to solve this problem by bioprinting the tissue directly into the subject, according to the release. Researchers will also be investigating the use of stem cells, biomaterials and differentiation factors in this process.

The team of researchers that received the grant includes professors of plastic surgery, biomedical engineering, and orthopedics and rehabilitation.

The researchers plan to investigate printing each type of tissue necessary for craniomaxillofacial reconstruction bone, fat and skin tissue individually, then study composite tissues that include all three of these layers. They hope that this will help them better understand how vascularization occurs in each type of tissue.

Ultimately, researchers hope to learn how different types of tissue interact and how bioprinting tissue directly into subjects will affect the facial reconstruction process.

If you're interested in submitting a Letter to the Editor, click here.

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Penn State receives grant to study bioprinting tissue for facial reconstructions - The Daily Collegian Online

Recommendation and review posted by Bethany Smith

PHILADELPHIA, Feb. 13, 2020 /PRNewswire/ --Researchers at the University of Pittsburgh and the University of Delaware have been awarded funding and support to accelerate their search for new medicines through an ongoing partnership between global biotechnology leader CSL Behring and the University City Science Center.

CSL Behring awarded Cecelia Yates, Ph.D., from the University of Pittsburgh, and Eleftherios (Terry) Papoutsakis, Ph.D., from the University of Delaware, $250,000 each and an opportunity to work alongside the plasma-based biotech's own experts in an effort to help transform their ideas into groundbreaking therapies to improve patients' health.

CSL Behring, a global leader in treating rare and serious diseases which has its global operational headquarters in King of Prussia, PA, identified the medical researchers utilizing the Science Center's, sourcing innovation framework for technology commercialization, support and infrastructure to efficiently evaluate technologies from multiple institutions.

"Congratulations Drs. Yates and Papoutsakis on being the first recipients of the CSL Behring-Science Center Research Acceleration Initiative," said Bill Mezzanotte, MD, Executive Vice President, Head of Research and Development for CSL Behring. "This initiative is another example of the strength of our partnership with the Philadelphia-based University City Science Center as we look in our 'backyard' for innovative scientific advancements that have the potential to help rare disease patients lead full lives. Our growing R&D organization looks forward to working with Dr. Yates and Dr. Papoutsakis in the years ahead to advance their scientific research."

"The Science Center couldn't be more excited about facilitating the introduction between these talented investigators and CSL Behring," says John Younger, MD, Vice President of Science & Technology at the Science Center. "Our network of universities, biotech, and pharmaceutical companies was built exactly for making these connections not just possible but easy. Supporting the development of new biologics, and new drug and gene delivery systems like those developed by Drs. Papoutsakis and Yates will continue to be an important focus of our team."

The investigators and technologies selected in this inaugural round of the program include:

Cecelia Yates, Ph.D., University of Pittsburgh, for the use of FibroKine biomimetic peptides as potential targeted therapeutic treatment of pulmonary fibrosis.

Fibrotic diseases constitute a significant health problem in the US with the ability to impact any organ that is scarred from chronic disease, including the heart, lung, liver, arteries, and skin. In some cases, progressive and life-threatening diseases follow, but effective therapies don't yet exist. In response, Dr. Yates has developed FibroKine, a chemokine-based class of biomimetic peptides that are potential therapeutic agents for the targeted treatment of tissue fibrosis. Support from CSL Behring will allow the Yates group to test FibroKine peptide ability to effectively treat and halt the progression of pulmonary fibrosis.

Eleftherios (Terry) Papoutsakis, Ph.D., University of Delaware, for exploring the use of cell derived micro-particles and vesicles (MkMPs) for the treatment of thrombocytopenias and in stem-cell targeted gene therapies

Gene delivery to or editing of Hematopoietic (blood) Stem and Progenitor Cells (HSPCs) can provide therapeutic benefit to patients for a variety of genetic hematological disorders, ranging from low platelet count diseases to primary immune deficiencies like Wiskott-Aldrich syndrome. With the support of CSL Behring, Dr. Papoutsakis will investigate the use of human MkMPs: 1) to promote in vivo platelet biogenesis as a potential treatment for thrombocytopenias, and 2) for the in vivo delivery of DNA, RNA, and proteins to HSPCs in gene therapy applications.

In October 2018, the Science Center and CSL Behring joined forces to identify promising technologies and support the commercialization pathways of potential new discoveries. Researchers at academic and research institutions throughout the region were invited to submit proposals for projects with a focus on therapeutics that fit within CSL Behring's five therapeutic areas of expertise: immunology and neurology; hematology and thrombosis; respiratory; cardiovascular and metabolic; and transplant.

Following the success of the initial pilot, the CSL Behring Science Center Research Initiative has expanded and is currently accepting applicationsfrom researchers at 28 institutions across six states with awardees to receive up to $400,000 each.

About CSL BehringCSL Behringis a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited(ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs more than 25,000 people, and delivers its life-saving therapies to people in more than 70 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vitaand follow us on Twitter.com/CSLBehring.

About the Science CenterLocated in the heart ofuCitySquare, the Science Center is a mission-driven nonprofit that commercializes promising technology, cultivates talent and convenes people to inspire action. For over 50 years, the Science Center has supported startups, research, and economic development across the emerging technology sectors. As a result, Science Center-supported companies account for one out of every 100 jobs in the Greater Philadelphia region and drive $13 billion in economic activity in the region annually. By providing the right help at the right time, the Science Center is turning bright ideas into businesses and nurturing a workforce to support our 21st century economy. For more information about the Science Center, go towww.sciencecenter.org

SOURCE CSL Behring

http://www.cslbehring.com

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University City Science Center partnership with CSL Behring accelerates the search for new biotherapies at the University of Pittsburgh and the...

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XconomyNational

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

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

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

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

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

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

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

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

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

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

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

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

8) Pharma and biotech inhouse manufacturing

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

The year saw positive signs that change is happening.

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

This article first appeared in Bioprocess Insider on January 27.

Image: iStock/PashaIgnatov

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

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

Recommendation and review posted by Bethany Smith

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

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

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

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

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

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

About CTX001TMCTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.

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

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

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

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

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

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

Investor Contact:Susan Kimsusan.kim@crisprtx.com

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

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

Recommendation and review posted by Bethany Smith

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

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

Check out our latest analysis for CRISPR Therapeutics

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Recommendation and review posted by Bethany Smith

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

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

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

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

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

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

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

FDA stands its ground

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

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

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

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

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

Experts unconvinced

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

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

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

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

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

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

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

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

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

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

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

Recommendation and review posted by Bethany Smith

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

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

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

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

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

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

Read the original post (Behind paywall)

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

Recommendation and review posted by Bethany Smith

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

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

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

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

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

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

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

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

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

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

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

Recommendation and review posted by Bethany Smith

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

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

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

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

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

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

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

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

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

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

Request Sample Report @ https://www.researchmoz.com/enquiry.php?type=S&repid=2602918&source=atm

Important Key questions answered in CRISPR Genome Editing market report:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Recommendation and review posted by Bethany Smith

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

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

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

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

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

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

To screen for more stocks like CRSP click here.

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

Recommendation and review posted by Bethany Smith

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

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

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

New Data from Intellias Engineered Cell Therapy Development Program for AML

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

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

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

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

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

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

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

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

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

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

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

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

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

About Intellia Therapeutics

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

Forward-Looking Statements

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

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

Intellia Contacts:

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

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

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

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

Recommendation and review posted by Bethany Smith

There are two different types of stem cell transplants:

To understand these treatments, it helps to know about bone marrow and stem cells.

Bone marrow is part of our immune system which protects us from infection and disease. It is found inside our bones, mainly in the hip bone and the breast bone. The bone marrow is where stem cells are made.

Stem cells are blood cells at the earliest stage of development. All our blood cells develop from stem cells in the bone marrow. Stem cells stay inside the bone marrow and when they are fully developed they go into the bloodstream.

Blood cells do not live long. The bone marrow normally makes millions of new blood cells every day to replace blood cells as they are needed.

There are three main types of blood cells:

There are two main types of white blood cell. These are called neutrophils and lymphocytes. Neutrophils are the most common. You will hear your doctor or nurse talk about your neutrophil count during your treatment.

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What are stem cells and bone marrow? - Macmillan Cancer ...

Recommendation and review posted by Bethany Smith

NEW YORK, Feb. 11, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc., (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company recently held a high level meeting with the U.S. Food and Drug Administration (FDA) to discuss potential NurOwn regulatory pathways for approval in ALS. Repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) is currently being evaluated in a fully enrolled Phase 3 pivotal trial in ALS (NCT03280056).

In the planned meeting with senior Center for Biologics Evaluation and Research (CBER) leadership and several leading U.S. ALS experts, the FDA confirmed that the fully enrolled Phase 3 ALS trial is collecting relevant data critical to the assessment of NurOwn efficacy. The FDA indicated that they will look at the "totality of the evidence" in the expected Phase 3 clinical trial data. Furthermore, based on their detailed data assessment, they are committed to work collaboratively with BrainStorm to identify a regulatory pathway forward, including opportunities to expedite statistical review of data from the Phase 3 trial.

Both the FDA and BrainStorm acknowledged the urgent unmet need and the shared goal of moving much needed therapies for ALS forward as quickly as possible.

This is a key turning point in ourworktowardprovidingALSpatientswith a potential new therapy,said ChaimLebovits, President and CEO ofBrainStorm. We commend the FDA foritscommitmentto the ALS communityandtofacilitating the development, and we ultimately hope, the approvalofNurOwn.The entire BrainStorm team is grateful for the ongoing and conscientious collaboration in the quest to beat ALS.

Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, stated, The entire team at BrainStorm has collectively worked to ensure that we conduct the finest, science-based clinical trials. We had the opportunity to communicate with Senior Leadership at the FDA and discuss how we can work together to navigate the approval process forward along a novel pathway. We appreciate their willingness and receptiveness to consider innovative approaches as we all seek to better serve the urgent unmet medical needs of the ALS community.

Brian Wallach, Co-Founder of I AM ALS stated: There is nothing more important to those living with ALS than having access to therapies that effectively combat this fatal disease. We have been working with BrainStorm for months now because we believe that NurOwn is a potentially transformative therapy in this fight. We were privileged to represent the patient voice at this meeting and are truly grateful to the company and the FDA for this critical agreement. This is a truly important moment of hope and we look forward to seeing both the Phase III data and the hopeful approval of NurOwn as soon as is possible.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwnCellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S.FDAapproval of autologous MSC-NTF cells in ALS. BrainStorm received U.S.FDAclearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) inDecember 2018and has been enrolling clinical trial participants sinceMarch 2019. For more information, visit the company'swebsite.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

Or

Katie Gallagher | Account Director, PR and MarketingLaVoieHealthScience Strategic CommunicationsO: 617-374-8800 x109M: 617-792-3937kgallagher@lavoiehealthscience.com

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BrainStorm Cell Therapeutics and FDA Agree to Potential NurOwn Regulatory Pathway for Approval in ALS - GlobeNewswire

Recommendation and review posted by Bethany Smith

Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

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Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.

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Bone Marrow Processing Systems Market Status and Trend: Top Key Players, Industry Dynamics, And Regional Scope 2025 - Med News Ledger

Recommendation and review posted by Bethany Smith

As with The Bell Curve, we will have to wait for peer reviews to carefully sift through the science. Early indications might indicate some trouble for Murray. Last month, the psychologists Michelle N. Meyer, Patrick Turley and Daniel J. Benjamin issued a sharp rebuke to his use of their research on polygenic scores in his piece for The Wall Street Journal teasing the new book. He characterized polygenic scores as providing decisive insight into I.Q. that was impervious to racism and other forms of prejudice. In fact, the psychologists assert in response, polygenic scores can and do reflect racism, sexism or other prejudices, as well as more benign environmental factors.

Murray serenely rolls out his propositions, assuring us on occasion that it is all consensus, securely known. And yet several claims are plainly contentious, even to the lay reader. Take Murrays description of male brains as systemizers and female brains as empathizers, drawing on work of the psychologist Simon Baron-Cohen. Men are drawn to things, in other words, and women to people. (Youll recognize this terminology from James Damores diversity letter to Google.) This rubric becomes an organizing principle in the book, explaining the typically gendered vocations for men and women (Things Jobs and People Jobs). What Murray avoids discussing are the profound questions surrounding one of the studies that scaffold his thinking.

In 2000, Baron-Cohen and colleagues published a study of day-old babies that found that boys looked at mobiles longer (hence systemizers) and girls at faces (empathizers). This study has never been replicated, not even by Baron-Cohen. It was also poorly designed: for one, some of the newborns were propped up; their gaze might have been mediated by how they were held. Not to mention the core question, as posed by the psychologist Cordelia Fine, who has written extensively about bias in research on sex differences in the brain: Why think that what a newborn prefers to look at provides any kind of window, however grimy, into their future abilities and interests?

Or consider Murrays interpretation of why women havent branched into more male-dominated fields over the last 30 years. Once again, he finds an explanation in the female preoccupation with people and emotion as opposed to the male orientation toward things and abstract thought. Sexism cannot be the culprit, he claims. Now that outright prohibition of women entering male-dominated fields has ended, any vestigial opposition ought to have abated in a matter of years. Never mind the wealth of research showing the very real persisting impediments that Murray dismisses. To name just one well-known example: In a study at Yale University, over 100 scientists reviewed a rsum submitted for an open position. The rsums were identical, although half were submitted under mens names and half womens names. The womens rsums were ranked significantly lower than the mens by both female and male faculty.

Why doesnt Murray attend more thoroughly to the role of the environment, to history even if to decisively repudiate their impact? On genetics, too, he dismisses aspects that might dilute the strength of his argument that outside interventions are limited in their effects on personality and social behavior. Developments in epigenetics, for example outside mechanisms that effectively turn genes on or off are waved away as hype.

Stranger still are the inconsistencies. Race is a construct is among the tenets Murray seeks to dismantle. Yet tucked midway through the book is the bland assertion that his evidence does not deny the many ways in which race is a social construct. There is no genetic basis for race. It is a social and legal definition a young, crude one at that, overlaid on the tangled realities of ancestral heredity. Ancestral populations might be more apt, he concedes. Not 40 pages later, however, hes back to huffing at the elite wisdom that race is a social construct. Murray appears to want it both ways: to gesture at a more nuanced and precise formulation but also to harness, when he chooses, the raw rhetorical power of railing against woke dogmas about race.

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Charles Murray Returns, Nodding to Caution but Still Courting Controversy - The New York Times

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