Archive for the ‘Crispr’ Category

Los Angeles, United State: Complete study of the global CRISPR And CRISPR-Associated (Cas) Genes market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global CRISPR And CRISPR-Associated (Cas) Genes industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on CRISPR And CRISPR-Associated (Cas) Genes production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

The research study includes great insights about critical market dynamics, including drivers, restraints, trends, and opportunities. It also includes various types of market analysis such as competitive analysis, manufacturing cost analysis, manufacturing process analysis, price analysis, and analysis of market influence factors. It is a complete study on the global CRISPR And CRISPR-Associated (Cas) Genes market that can be used as a set of effective guidelines for ensuring strong growth in the coming years. It caters to all types of interested parties, viz. stakeholders, market participants, investors, market researchers, and other individuals associated with the CRISPR And CRISPR-Associated (Cas) Genes business.

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

Key Players Mentioned in the Global CRISPR And CRISPR-Associated (Cas) Genes Market Research Report: , Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, GE Healthcare Dharmacon

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

Global CRISPR And CRISPR-Associated (Cas) Genes Market Segmentation by Application: , Biotechnology Companies, Pharmaceutical Companies, Academic Institutes, Research and Development Institutes

The report has classified the global CRISPR And CRISPR-Associated (Cas) Genes industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the CRISPR And CRISPR-Associated (Cas) Genes manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall CRISPR And CRISPR-Associated (Cas) Genes industry.

Additionally, the industry analysts have studied key regions including North America, Europe, Asia Pacific, Latin America, and Middle East and Africa, along with their respective countries. Here, they have given a clear-cut understanding of the present and future situations of the global CRISPR And CRISPR-Associated (Cas) Genes industry in key regions. This will help the key players to focus on the lucrative regional markets.

Key questions answered in the report:

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Table of Content

Table of Contents Executive Summary 1 CRISPR And CRISPR-Associated (Cas) Genes Market Overview1.1 Product Overview and Scope of CRISPR And CRISPR-Associated (Cas) Genes1.2 CRISPR And CRISPR-Associated (Cas) Genes Segment by Type1.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate Comparison by Type (2014-2025)1.2.2 Genome Editing1.2.3 Genetic engineering1.2.4 gRNA Database/Gene Librar1.2.5 CRISPR Plasmid1.2.6 Human Stem Cells1.2.7 Genetically Modified Organisms/Crops1.2.8 Cell Line Engineering1.3 CRISPR And CRISPR-Associated (Cas) Genes Segment by Application1.3.1 CRISPR And CRISPR-Associated (Cas) Genes Consumption Comparison by Application (2014-2025)1.3.2 Biotechnology Companies1.3.3 Pharmaceutical Companies1.3.4 Academic Institutes1.3.5 Research and Development Institutes1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Market by Region1.3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size Region1.3.2 North America Status and Prospect (2014-2025)1.3.3 Europe Status and Prospect (2014-2025)1.3.4 China Status and Prospect (2014-2025)1.3.5 Japan Status and Prospect (2014-2025)1.3.6 Southeast Asia Status and Prospect (2014-2025)1.3.7 India Status and Prospect (2014-2025)1.4 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size1.4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue (2014-2025)1.4.2 Global CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2025) 2 Global CRISPR And CRISPR-Associated (Cas) Genes Market Competition by Manufacturers2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Manufacturers (2014-2019)2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Share by Manufacturers (2014-2019)2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Average Price by Manufacturers (2014-2019)2.4 Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Production Sites, Area Served, Product Types2.5 CRISPR And CRISPR-Associated (Cas) Genes Market Competitive Situation and Trends2.5.1 CRISPR And CRISPR-Associated (Cas) Genes Market Concentration Rate2.5.2 CRISPR And CRISPR-Associated (Cas) Genes Market Share of Top 3 and Top 5 Manufacturers2.5.3 Mergers & Acquisitions, Expansion 3 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Regions3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Regions3.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Regions (2014-2019)3.3 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.4 North America CRISPR And CRISPR-Associated (Cas) Genes Production3.4.1 North America CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.5 Europe CRISPR And CRISPR-Associated (Cas) Genes Production3.5.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.5.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.6 China CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2019)3.6.1 China CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.6.2 China CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.7 Japan CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2019)3.7.1 Japan CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.7.2 Japan CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019) 4 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Regions4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Regions4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.3 Europe CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.4 China CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.5 Japan CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019) 5 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price Trend by Type5.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Type (2014-2019)5.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Type (2014-2019)5.3 Global CRISPR And CRISPR-Associated (Cas) Genes Price by Type (2014-2019)5.4 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth by Type (2014-2019) 6 Global CRISPR And CRISPR-Associated (Cas) Genes Market Analysis by Applications6.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Market Share by Application (2014-2019)6.2 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Growth Rate by Application (2014-2019) 7 Company Profiles and Key Figures in CRISPR And CRISPR-Associated (Cas) Genes Business7.1 Caribou Biosciences7.1.1 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.1.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.1.3 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.1.4 Main Business and Markets Served7.2 Addgene7.2.1 Addgene CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.2.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.2.3 Addgene CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.2.4 Main Business and Markets Served7.3 CRISPR THERAPEUTICS7.3.1 CRISPR THERAPEUTICS CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.3.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.3.3 CRISPR THERAPEUTICS CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.3.4 Main Business and Markets Served7.4 Merck KGaA7.4.1 Merck KGaA CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.4.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.4.3 Merck KGaA CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.4.4 Main Business and Markets Served7.5 Mirus Bio LLC7.5.1 Mirus Bio LLC CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.5.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.5.3 Mirus Bio LLC CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.5.4 Main Business and Markets Served7.6 Editas Medicine7.6.1 Editas Medicine CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.6.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.6.3 Editas Medicine CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.6.4 Main Business and Markets Served7.7 Takara Bio USA7.7.1 Takara Bio USA CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.7.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.7.3 Takara Bio USA CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.7.4 Main Business and Markets Served7.8 Thermo Fisher Scientific7.8.1 Thermo Fisher Scientific CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.8.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.8.3 Thermo Fisher Scientific CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.8.4 Main Business and Markets Served7.9 Horizon Discovery Group7.9.1 Horizon Discovery Group CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.9.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.9.3 Horizon Discovery Group CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.9.4 Main Business and Markets Served7.10 Intellia Therapeutics7.10.1 Intellia Therapeutics CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.10.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.10.3 Intellia Therapeutics CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.10.4 Main Business and Markets Served7.11 GE Healthcare Dharmacon 8 CRISPR And CRISPR-Associated (Cas) Genes Manufacturing Cost Analysis8.1 CRISPR And CRISPR-Associated (Cas) Genes Key Raw Materials Analysis8.1.1 Key Raw Materials8.1.2 Price Trend of Key Raw Materials8.1.3 Key Suppliers of Raw Materials8.2 Proportion of Manufacturing Cost Structure8.3 Manufacturing Process Analysis of CRISPR And CRISPR-Associated (Cas) Genes8.4 CRISPR And CRISPR-Associated (Cas) Genes Industrial Chain Analysis 9 Marketing Channel, Distributors and Customers9.1 Marketing Channel9.1.1 Direct Marketing9.1.2 Indirect Marketing9.2 CRISPR And CRISPR-Associated (Cas) Genes Distributors List9.3 CRISPR And CRISPR-Associated (Cas) Genes Customers 10 Market Dynamics10.1 Market Trends10.2 Opportunities10.3 Market Drivers10.4 Challenges10.5 Influence Factors 11 Global CRISPR And CRISPR-Associated (Cas) Genes Market Forecast11.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast11.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate Forecast (2019-2025)11.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue and Growth Rate Forecast (2019-2025)11.1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Price and Trend Forecast (2019-2025)11.2 Global CRISPR And CRISPR-Associated (Cas) Genes Production Forecast by Regions (2019-2025)11.2.1 North America CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.3 China CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.4 Japan CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.3 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast by Regions (2019-2025)11.3.1 North America CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.3 China CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.4 Japan CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.4 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue and Price Forecast by Type (2019-2025)11.5 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast by Application (2019-2025) 12 Research Findings and Conclusion 13 Methodology and Data Source13.1 Methodology/Research Approach13.1.1 Research Programs/Design13.1.2 Market Size Estimation13.1.3 Market Breakdown and Data Triangulation13.2 Data Source13.2.1 Secondary Sources13.2.2 Primary Sources13.3 Author List13.4 Disclaimer

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CRISPR And CRISPR-Associated (Cas) Genes Market Size, Key Trends, Challenges and Standardization, Research, Key Players, Economic Impact and Forecast...

The report on the CRISPR Genome Editing market provides a birds eye view of the current proceeding within the CRISPR Genome Editing market. Further, the report also takes into account the impact of the novel COVID-19 pandemic on the CRISPR Genome Editing market and offers a clear assessment of the projected market fluctuations during the forecast period. The different factors that are likely to impact the overall dynamics of the CRISPR Genome Editing market over the forecast period (2019-2029) including the current trends, growth opportunities, restraining factors, and more are discussed in detail in the market study.

For top companies in United States, European Union and China, this report investigates and analyzes the production, value, price, market share and growth rate for the top manufacturers, key data from 2019 to 2025.

The CRISPR Genome Editing market report firstly introduced the basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the CRISPR Genome Editing market report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

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The major players profiled in this CRISPR Genome Editing market report include:

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

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

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

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

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

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Globally Leading Manufacturers of CRISPR Genome Editing product Scale up Production to Meet Sharp Spike in Demand Fueled by COVID-78 - Personal Injury...

This high-end research comprehension on the Global CRISPR Technology Market renders major impetus on detailed growth facets, in terms of product section, payment and transaction platforms, further incorporating service portfolio, applications, as well as a specific compilation on technological interventions that facilitate ideal growth potential of the market.

The report is so designed as to direct concrete headways in identifying and deciphering each of the market dimensions to evaluate logical derivatives which have the potential to set the growth course in the aforementioned CRISPR Technology market. Besides presenting notable insights on market factors comprising above determinants, this specific, innately crafted research report offering further in its subsequent sections states information on regional segmentation, as well as thoughtful perspectives on specific understanding comprising region specific developments as well as leading market players objectives to trigger maximum revenue generation and profits.

This study covers following key players:Thermo Fisher ScientificMerck KGaAGenScriptIntegrated DNA Technologies (IDT)Horizon Discovery GroupAgilent TechnologiesCellectaGeneCopoeiaNew England BiolabsOrigene TechnologiesSynthego CorporationToolgen

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This illustrative research report on the CRISPR Technology market is an all-in-one, ready to use handbook of market dynamics that upon mindful inference lends valuable insights on market developments, growth trajectory, dominant trends as well as technological sophistication as well as segment expansion and competition spectrum that have a strong bearing on the growth probabilities of the CRISPR Technology market.

This particular section of the CRISPR Technology market report specifically stresses upon various indigenous tactical discretion that eventually contributed towards soliciting heralding market consolidation, impeccable stability and sustainable revenue pools, the ultimate touchstone to judge the potency of the CRISPR Technology market.

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Market segment by Type, the product can be split into EnzymesKitsgRNALibrariesDesign Tools

Market segment by Application, split into BiomedicalAgricultural

The report further unveils pertinent details about segment contribution in coining ample revenue flow, sustainability and long term growth in global CRISPR Technology market. A thorough knowledge base of market facets remains integral and indispensable to decode CRISPR Technology market prognosis. This recent research compilation on the CRISPR Technology market presents a deep analytical review and a concise presentation of ongoing market trends that collectively inculcate a strong influence on the growth trajectory of the aforementioned CRISPR Technology market.

The report sheds light on the particular segment that sets revenue maximization, rolling, thus incurring steady growth in revenues and contributing towards steady sustenance of the CRISPR Technology market. This well versedreport is thoughtfully crafted to arm report readers with convincing market insights on the mettle of all aforementioned factors that propel relentless growth despite significant bottlenecks in the CRISPR Technology market.

Some Major TOC Points:1 Report Overview2 Global Growth Trends3 Market Share by Key Players4 Breakdown Data by Type and ApplicationContinued

In addition to all of the above stated inputs, discussed at length in the report, the report sheds tangible light on dynamic segmentation based on which the market has been systematically split into prominent segments inclusive of type, end use technology, as well as region specific diversification of the CRISPR Technology market to encourage highly remunerative business discretion.

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Global CRISPR Technology Market Expanding Rapidly with Forecast 2025 and Top Players : Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA...

BERKELEY, Calif. Great partnerships depend on a rare combination of shared vision, mutual trust, and complementary skillsets. Scientists from Stanford University and Berkeley Labs Molecular Foundry may have found just that in a newly formed collaboration. Since March, research teams from these two institutions have been developing a new therapeutic that uses CRISPR technology in combination with a novel lipitoid delivery system to fight COVID-19 and other RNA viruses.

Last year, Stanley Qi, an assistant professor in the departments of bioengineering, and chemical and systems biology at Stanford, along with his team pioneered a new technology called prophylactic antiviral CRISPR in human cells, or PAC-MAN. Their efforts were initially focused on developing PAC-MAN as a strategy for targeting and destroying influenzain humans. When the COVID-19 pandemic hit in January, they abruptly pivoted their research to adapt the technology for SARS-CoV-2.

PAC-MAN is based on the popular gene-editing tool, CRISPR, which has revolutionized modern medicine. The technology allows for unprecedented control over gene expression in many species, including humans. At its core, CRISPR is a technique for finding a specific sequence of DNA inside a cell and then altering or deleting it.

The process involves two components: an enzyme (Cas protein) that cuts through genetic material; and a piece of RNA that is used as a guide to direct Cas protein to the targeted gene sequence. PAC-MAN uses a specific version of the Cas protein, called Cas13, which slices through RNA such as that which makes up the genomes of viruses like influenza and SARS-CoV-2.

In a paper published in the journalCell, Qi and his team demonstrate that their PAC-MAN strategy can effectively degrade RNA from SARS-CoV-2 sequences and live influenza A virus in human lung cells. Notably, the authors perform bioinformatics analyses that suggested a group of only six guide RNAs can target more than 90% of all coronaviruses, giving PAC-MAN the potential to become a pan-coronavirus inhibition strategy.

There was one problem. For PAC-MAN to be effective in patients, particularly as an anti-COVID-19 therapy, it needs to be able to be delivered into lung cells. But my lab doesnt work on delivery methods, Qi explains in a media release.

Enter Michael Connolly, a principal scientific engineer associate in the Biological Nanostructures Facility at the Molecular Foundry. His work on synthetic molecules called lipitoidsprovides a possible solution to the delivery issue with PAC-MAN. Lipitoids were first developed 20 years ago by Connollys mentor, Ron Zuckermann, and have since been widely studied for potential therapeutic applications. They are nontoxic to humans and can facilitate entry of RNA or DNA into cells by encapsulating them into tiny nanoparticles, a seemingly perfect delivery system for the PAC-MAN technology.

Berkeley Labs Molecular Foundry has provided us with a molecular treasure that transformed our research, Qi says.

In late April, the PAC-MAN/lipitoid system had its first test run in a sample of human lung cells. According to Qi, this approach reduced the amount of synthetic SARS-CoV-2 by greater than 90%. As an immediate next step to validate their technique, the team is collaborating with researchers at New York University and Karolinska Institute in Sweden. There, researchers are conducting animal trials with live SARS-CoV-2 infection. If successful, they hope to further develop the PAC-MAN/lipitoid system as a therapy against other viruses.

An effective lipitoid delivery, coupled with CRISPR targeting, could enable a very powerful strategy for fighting viral disease not only against COVID-19, but possibly against newly viral strains with pandemic potential, says Connolly.

One of the silver linings to emerge from the COVID-19 pandemic is how the scientific community has banded together to focus urgently on one topic. The joint effort of researchers from Stanford University and Berkeley Labs Molecular Foundry is an archetype of that collaborative spirit. As Qi notes, Its very rewarding to combine expertise and test new ideas across institutions in these difficult times.

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PAC-MAN to the Rescue: CRISPR-Based Tech on Cutting Edge of COVID-19 Therapeutics - Study Finds

Rising government funding and increase in the number of genomic projects, especially in the area of rare diseases, and a growing application horizon are all expected to drive the growth of the genome editing/genome engineering market.

New York, June 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Genome Editing/Genome Engineering Market by Technology, Product & Service, Application, End-User and Region - Global Forecast to 2025" - https://www.reportlinker.com/p05220258/?utm_source=GNW

The global genome editing/genome engineering market is projected to reach USD 11.2 billion in 2025 from USD 5.1 billion in 2020, at a CAGR of 17.0 % during the forecast period. Market growth is largely driven by factors such as the rise in government funding, growth in the number of genomics projects, high prevalence of infectious diseases & cancer, technological advancements, increasing production of genetically modified crops, and growing application areas of genomics. However, the high cost of genomic equipment will restrain the growth of this market.

CRISPR commanded the largest share of the market in 2019.Based on technology, the market is segmented into CRISPR, TALEN, ZFN, antisense, and other technologies.CRISPR accounted for the largest share of the genome editing/genome engineering market in 2019.

The large share of this segment can be attributed to the ease of use associated with the CRISPR technology and its ability to multiplex.

Pharmaceutical companies commanded the largest share of the genome editing/genome engineering market in 2019.By end user, the genome editing/genome engineering market is segmented into pharmaceutical companies, biotechnology companies, and academic & government research institutes.The pharmaceutical companies segment accounted for the largest share of the market.

The pharmaceutical companies segment accounted for the largest share of the genome editing/genome engineering market in 2019. This is due to the increasing prevalence of infectious diseases and cancer, which is driving research in the pharma sector for drug development.

The Asia Pacific region will register the highest growth in the global genome editing/genome engineering market during the forecast period.The Asia Pacific is estimated to grow at the highest CAGR during the forecast period. Factors such as the rapid growth in the pharmaceutical and biopharmaceutical industry, the rising number of genomic projects, and the presence of a genetically diverse population have supported the regions high growth rate.

In-depth interviews were conducted with chief executive officers (CEOs), marketing directors, other directors, and executives from various key organizations operating in the genome editing/genome engineering market. By Respondent Type: Supply Side (80%) and Demand Side (20%) By Designation: D-level (55%), C-level (20%), and Others (25%) By Region: North America (50%), Europe (20%), Asia Pacific (20%), and RoW (10%)

The major companies in the genome editing/genome engineering market include Thermo Fisher Scientific (US), Merck (Germany), Horizon Discovery Limited (UK), Lonza (Switzerland), GenScript (US), Eurofins Scientific (Luxembourg), and Sangamo Therapeutics (US).The study includes an in-depth competitive analysis of these key players in the genome editing/genome engineering market, along with their company profiles, recent developments, and key market strategies.

Research Coverage:The market study covers the genome editing/genome engineering market across various segments.It aims at estimating the market size and the growth potential of this market across different segments, such as products, technology, and region.

The study also includes an in-depth competitive analysis of the key players in the market, along with their company profiles, key observations related to their product and business offerings, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants in this market and provide information on the closest approximations of the revenue numbers for the overall genome editing/genome engineering market and its subsegments.This report will help stakeholders to understand the competitive landscape, gain insights to better position their businesses, and plan suitable go-to-market strategies.

The report will also help stakeholders to understand the pulse of the market and provide information on the key market drivers, restraints, opportunities, and challenges.Read the full report: https://www.reportlinker.com/p05220258/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global genome editing/genome engineering market is projected to reach USD 11.2 billion in 2025 from USD 5.1 billion in 2020, at a CAGR of 17.0 % -...

CRISPR Technology Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost and revenue during the forecast period from 2020 to 2027. A CRISPR Technology market that includes Future Trends, Current Growth Factors, Meticulous Opinions, Facts, Historical Data and Statistically Supported And Industry-Validated Market Data.

This CRISPR Technology market research provides a clear explanation of how this market will make a growth impression during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

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Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT), Horizon Discovery Group, Agilent Technologies, Cellecta Inc., GeneCopoeia Inc., New England Biolabs, Origene Technologies Inc are some of the major organizations dominating the global market.

(*Note: Other Players Can be Added per Request)

Key players in the CRISPR Technology market were identified through a second survey, and market share was determined through a first and second survey. All measurement sharing, splitting and analysis were solved using a secondary source and a validated primary source. The CRISPR Technology market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure. The combination of these two factors will help key players meet the market reach and help to understand offered characteristics and customer needs.

The report also makes some important suggestions for the new CRISPR Technology market project before evaluating its feasibility. Overall, this report covers CRISPR Technology market Sales, Price, Sales, Gross Profit, Historical Growth and Future Prospects. It provides facts related to mergers, acquisitions, partnerships and joint venture activities prevalent in the market.

This report includes market size estimates of value (million US $) and volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the CRISPR Technology market, estimating the size of various other submarkets in the overall market. Major players in the market were identified through secondary studies, and market share was determined through primary and secondary studies. All ratio sharing, splitting and analysis were determined using the secondary source and the identified primary source.

What CRISPR Technology Market report offers:

Regions Covered in This Report

Complete knowledge of the CRISPR Technology market is based on the latest industry news, opportunities and trends in the expected region. The CRISPR Technology market research report provides clear insights into the influential factors expected to change the global market in the near future.

Remarkable Attributes of CRISPR Technology Market Report:

About WMR

Worldwide Market Reports is your one-stop repository of detailed and in-depth market research reports compiled by an extensive list of publishers from across the globe. We offer reports across virtually all domains and an exhaustive list of sub-domains under the sun. The in-depth market analysis by some of the most vastly experienced analysts provide our diverse range of clients from across all industries with vital decision making insights to plan and align their market strategies in line with current market trends.

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Know About CRISPR Technology Market: How COVID 19 is Reshaping the Industry: Thermo Fisher Scientific, Merck KGaA, GenScript - Cole of Duty

Gene Therapies Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost and revenue during the forecast period from 2020 to 2027. A Gene Therapies market that includes Future Trends, Current Growth Factors, Meticulous Opinions, Facts, Historical Data and Statistically Supported And Industry-Validated Market Data.

This Gene Therapies market research provides a clear explanation of how this market will make a growth impression during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

RequestSample PDF of Gene Therapies Market Report https://www.worldwidemarketreports.com/sample/179400

Adaptimmune Therapeutics Plc., Celgene Corporation, Crispr Therapeutics Ag, Glaxosmithkline Plc, Intellia Therapeutics Inc., Merck & Co. Inc., Novartis Ag, Regenxbio Inc., Voyager Therapeutics Inc., Abeona Therapeutics Inc. are some of the major organizations dominating the global market.

(*Note: Other Players Can be Added per Request)

Key players in the Gene Therapies market were identified through a second survey, and market share was determined through a first and second survey. All measurement sharing, splitting and analysis were solved using a secondary source and a validated primary source. The Gene Therapies market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure. The combination of these two factors will help key players meet the market reach and help to understand offered characteristics and customer needs.

The report also makes some important suggestions for the new Gene Therapies market project before evaluating its feasibility. Overall, this report covers Gene Therapies market Sales, Price, Sales, Gross Profit, Historical Growth and Future Prospects. It provides facts related to mergers, acquisitions, partnerships and joint venture activities prevalent in the market.

This report includes market size estimates of value (million US $) and volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the Gene Therapies market, estimating the size of various other submarkets in the overall market. Major players in the market were identified through secondary studies, and market share was determined through primary and secondary studies. All ratio sharing, splitting and analysis were determined using the secondary source and the identified primary source.

What Gene Therapies Market report offers:

Regions Covered in This Report

Complete knowledge of the Gene Therapies market is based on the latest industry news, opportunities and trends in the expected region. The Gene Therapies market research report provides clear insights into the influential factors expected to change the global market in the near future.

Remarkable Attributes of Gene Therapies Market Report:

About WMR

Worldwide Market Reports is your one-stop repository of detailed and in-depth market research reports compiled by an extensive list of publishers from across the globe. We offer reports across virtually all domains and an exhaustive list of sub-domains under the sun. The in-depth market analysis by some of the most vastly experienced analysts provide our diverse range of clients from across all industries with vital decision making insights to plan and align their market strategies in line with current market trends.

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Gene Therapies Market Expansion of Industry Creat New Opportunities for Key Players: Adaptimmune Therapeutics Plc., Celgene Corporation, Crispr...

From sophisticated techniques to easy-to-use strips, the world has deployed all kinds of tools to fightnovel coronavirus

Diagnostic tests are important to identify the sick,trace contacts tracing and ensure the disease does not spread. While the standard tests available for COVID-19 have been used for this purpose so far, researchers have been trying to find new methods for testing which are faster, cheaper and more reliable. The aim is also to develop tests that do not require infrastructure and can be used off-site like in rural areas.

Down To Earth lists the available test and also those that are in the pipeline:

RT-PCR

Medical professionals vouch for the accuracy of Reverse Transcription Polymerase Chain Reaction when it comes to diagnosis of infectious diseases. The test is based on the PCR technique, which replicates genetic material of the pathogen and helps identify it.

But PCR is customised to replicating DNA and for pathogens where the genetic material is RNA, as is the case of SARS-CoV-2 virus, an additional step of converting the RNA into DNA is added. This step is Reverse Transcription.

After a sample of mucous is collected from the nasopharynx using a swab, it is kept in a buffer solution which also helps in extraction of the virus. Then, using RT-PCR, its genetic material is replicated many times over usually 40 times.

During the replication process, primers (which are specific to the viral genetic material), enzymes, nucleotides and fluorescent probes are added.

The fluorescent probes offers a visual signal as soon as a strand is successfully copied. The test is thus also called as real-time RT-PCR (rRT-PCR or qRT-PCR, where q stands for quantitative).

Looks for viral genes Takessix hoursConducted in labs with high biosafety levels, BSL-2 &bill

Automated RT-PCR

RT-PCR test can also be automated. India has so far allowed two such tests which run on proprietary machines: TrueNat, by Molbio Diagnostics of India, and Xpert Xpress, by Cepheid of USA.

Both were originally used to test for TB. TrueNat was approved for COVID-19 by ICMR on April 4. Its battery-operated kits are the size of a telephone and can be taken out into the field to test as many as 15 individuals a day.

The Indian Council of Medical Research (ICMR) allowed the use of Xpert Xpress on April 19 under emergency use authorisation. The test is fully automated and provides the results in 45 minutes.

Four tests can be performed simultaneously. Both tests require positive results to be confirmed by RT-PCR.

Looks for viral genes Takes 35-50 minutes Conducted in BSL-2 labs, portable Positive results have to be confirmed by RT-PCR

Antibody tests

These can be manual or automated immunoassays. They identify the IgM and IgG antibodies developed against the SARS-CoV-2. For the test, whole blood, serum and plasma can be used and if antibodies are present in the sample, these bind to the antigen immobilised on the test strip and give a coloured reaction.

Such test kits are easy to use, provides quick results and are also effective in identifying asymptomatic patients. There is a risk of getting false positives and results need to be confirmed using a more advanced test.

In case of ELISA, (enzyme-linked immunosorbent assay) antibody test, as many as 90 tests can be run simultaneously. The results are more reliable than strip tests and can also provide quantitative data. The test provides results in around 2.5 hours.

Looks for antibodies Takes 20 minutes to 2.5hoursTo be conducted under doctors supervision Useful only for experimental or surveillance purposes

Antigen rapid tests

Antigen is a foreign substance produced by a pathogen, which induces an immune response in the body, especially the production of antibodies. The technique thus detects the presence of antigen or viral RNA in the sample.

The test is being developed by E25Bio, a Massachusetts-based biotech startup. The test relies on a patients nasopharyngeal swab. It resembles an over-the-counter pregnancy test and provides visual results within 15 minutes by detecting the presence of the virus in the sample.

The company is in the process of obtaining clearance under USFDAs emergency use authorisation model. Another company, OraSure Technologies Inc, has also received funds to develop a kit based on this technique.

Looks for viral genes In development stage

CRISPR

This test identifies the virus using the CRISPR genome editor, which uses tags that carry enzymes to the target DNA and cut it. For identifying the virus, a special tag has been developed whose enzyme, instead of cutting the gene, gives off a signal that it has reached the target.

A diagnostic kit based on CRISPR received emergency use authorisation by USFDA on May 7. Sherlock CRISPR SARS-CoV-2 kit is based on the specific high sensitivity enzymatic reporter unlocking technique.

The genetic material is extracted from the patients sample, amplified and incubated and detected with Cas13 enzyme. A commercially available paper dipstick is then used to confirm the presence of the virus with the naked eye.

A similar kit, DETECTR, is being developed by Mammoth Biosciences and UC San Francisco.

Looks for viral genes Takes 30 minutes As accurate as RT-PCR

Dogs

This experiment involves training dogs to sniff out people suffering from COVID-19.

The three-month trials would be led by the London School of Hygiene & Tropical Medicine and are backed with 500,000 pounds of government funding.

The UK hopes these dogs would be part of the countrys strategy to test people.

Each dog is expected to screen as many as 250 people every hour. The dogs would include breeds like labradors and cocker spaniels.

Dogs are already used to detect a host of diseases such as cancers and malaria.

The dogs use specific odour that a sick person gives out. Samples of breath and body odour can come from a number of sources like used face masks.

Looks for odour Under trial Can test 250 people per hour

This was first published in Down To Earths print edition (dated 1-16 June, 2020)

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Battle-ready for COVID-19: A look at the arsenal of various tests - Down To Earth Magazine

Gene editing Cas9 protein.

Applied computational biology discoveries vastly expand the range of CRISPRs access to DNA sequences.

In nature, bacteria use CRISPR as an adaptive immune system to protect themselves against viruses. Over the past decade, scientists have been able to successfully build upon that natural phenomenon with the discovery of CRISPR proteins found in bacteria the most widely used of which is the Cas9 enzyme. In combination with a guide RNA, Cas9 is able to target, cut, and degrade specific DNA sequences.

With applications ranging from the treatment of genetic diseases to the nutritional potency of agricultural crops, CRISPR has emerged as one of the most promising tools for genome editing. Cas9 enzymes, however, rely on specific DNA ZIP codes to pinpoint where to cut and edit. The most widely-used Cas9 fromStreptococcus pyogenes bacteria, SpCas9, requires two G nucleotides beside target sites. Less than 10 percent of DNA sequences meet this requirement.

In research published this month in both Nature Biotechnology andNature Communications,a team of computational biologists in the Media Labs Molecular Machines group and the MIT Center for Bits and Atoms have successfully engineered new proteins with enhanced genome editing capabilities, significantly broadening the spectrum of DNA sequences that can be accurately and effectively accessed.

This work was led by Pranam Chatterjee who recently completed his PhD in media arts and sciences; Noah Jakimo PhD 19, a Media Lab affiliate; and Media Lab Associate Professor Joseph Jacobson, in collaboration with lab members and researchers at the University of Massachusetts Medical School.

These new findings stem from the groups earlier breakthrough work in the computational discovery of Cas9 proteins. The teamidentified and experimentally characterized the Cas9 fromStreptococcus canisbacteria (ScCas9), which, while similar to SpCas9, had the ability to target a much broader range of target DNA sequences. That discovery expanded the number of locations that Cas9 enzymes could target from the original 10 percent of sites on the genome to nearly 50 percent. The team first reported those findings in 2018 inScience Advances.

To improve ScCas9 as a genome editing tool, the scientists computationally identified unique parts from similar Cas9 proteins to engineer an optimized version of ScCas9, which the team has named Sc++.Sc++ is the first known enzyme to simultaneously exhibit the three properties deemed essential for effective genome editing: broad targeting capability; robust cutting activity; and minimal errors due to off-targeting, notes Chatterjee.

Concurrently, the team successfully used their previous SPAMALOT algorithm to discoverStreptococcus macacaeCas9 (SmacCas9) that required two A nucleotides, rather than two Gs. Through domain swapping and further engineering, the team presents the newiSpyMac enzyme as one of the first known Cas9 editors not requiring a G, enabling targeting of an additional 20 percent of the genome that was previously inaccessible.To engineer iSpyMac, we simultaneously made hundreds of changes to SpCas9, knowing even a single change can break it,says Jakimo, the senior author on this second study. Our success is a testament to the wealth of microbial genomic data that can provide helpful clues about protein function with tools like SPAMALOT.

Erik Sontheimer, professor and vice chair of the RNA Therapeutics Institute at the University of Massachusetts Medical School, and a collaborator on the research, notes the significance of this work. The fewer targeting limitations we encounter, and the fewer compromises and trade-offs that have to be made between activity and accuracy, the greater the impact that CRISPR genome editing can have on biotechnology and human health. This is why Sc++ and iSpyMac provide such valuable new additions to the CRISPR editing arsenal.

As labs around the world have already begun to use the enzymes to successfully edit the genomes of various organisms, from rice to rabbits, the next goal for this research will be to develop tools to reach the remaining 30 percent of genome sequences. Chatterjee, in collaboration with the University of Zurich, is looking to unlock the final advances that will allow scientists to access any genomic sequence, and to address any type of gene mutation in the treatment of genetic diseases.

For now, however, as in many labs across the MIT campus, work has pivoted to address the Covid-19 pandemic. By applying computational design principles to engineer proteins that can target and bind to the invading SARS-CoV-2 virus, Chatterjee and the research team at the Media Lab are seeking to create enzymes to rapidly halt the virus, and enable cell recovery.

We engineer proteins differently, Chatterjee adds. Our ability to integrate computation and experimentation enables us to refine our algorithms and build impactful tools for a host of applications, from addressing genetic diseases to Covid-19, and beyond.

References:

A Cas9 with PAM recognition for adenine dinucleotides by Pranam Chatterjee, Jooyoung Lee, Lisa Nip, Sabrina R. T. Koseki, Emma Tysinger, Erik J. Sontheimer, Joseph M. Jacobson and Noah Jakimo, 18 May 2020, Nature Communications.DOI: 10.1038/s41467-020-16117-8

An engineered ScCas9 with broad PAM range and high specificity and activity by Pranam Chatterjee, Noah Jakimo, Jooyoung Lee, Nadia Amrani, Toms Rodrguez, Sabrina R. T. Koseki, Emma Tysinger, Rui Qing, Shilei Hao, Erik J. Sontheimer and Joseph Jacobson, 11 May 2020, Nature Biotechnology.DOI: 10.1038/s41587-020-0517-0

Minimal PAM specificity of a highly similar SpCas9 ortholog by Pranam Chatterjee, Noah Jakimo and Joseph M. Jacobson, 24 October 2018, Science Advances.DOI: 10.1126/sciadv.aau0766

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Nature-Inspired CRISPR Enzyme Discoveries Vastly Expand Genome Editing - SciTechDaily

-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia-

-Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-

-Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted-

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany.

CLIMB-111 Trial in Transfusion-Dependent Beta Thalassemia Updated ResultsData presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRISPR Therapeutics and Vertex announced initial data for the first TDT patient in November of 2019.

Patient 1 with TDT has the 0/IVS-I-110 genotype, which is associated with a severe phenotype similar to 0/0, and had a transfusion requirement of 34 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented today show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 100.0%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year.

Patient 2 with TDT has the 0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.4%.

Hemoglobin data over time are presented for Patient 1 and Patient 2 below.

Figure 1accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/35581299-d683-44b0-a75e-7a1a9b9fe9eb

CLIMB-121 Trial in Severe Sickle Cell Disease Updated Results Data presented today at EHA reflect longer-duration follow-up data for the first patient with SCD treated with CTX001. CRISPR Therapeutics and Vertex announced initial data for this first SCD patient in November of 2019.

Patient 1 with SCD experienced seven vaso-occlusive crises (VOCs) and five packed red blood cell transfusions per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. After CTX001 infusion, three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain; all subsequently resolved. New data presented today show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. Bone marrow allelic editing was 81.4% at 6 months. Figure 2 presents the hemoglobin data over time for this patient.

Figure 2 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7610c5bd-25c8-4f5b-be86-8bc16ed57eb1

With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as consistency of the therapeutic effect across patients, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. These highly encouraging early data represent one more step toward delivering on the promise and potential of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases.

The data announced today are remarkable, including the demonstration of clinical proof-of-concept in TDT, said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. While these are still early days, these data mark another important milestone for this program and for the field of gene editing. The results presented at this medical conference add to results previously shared demonstrating that CRISPR/Cas9 gene editing has the potential to be a curative therapy for severe genetic diseases like sickle cell and beta thalassemia.

In my 25 years of caring for children and young adults facing both sickle cell disease and beta thalassemia, I have seen how these diseases can adversely affect patients lives in very significant ways, said Dr. Haydar Frangoul, Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center and senior author of the abstract presented at the EHA virtual congress. I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease.

Recent Progress in the Phase 1/2 Clinical TrialsCLIMB-111 for TDT has dosed a total of 5 patients, and all patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of the first two patients. Additionally, CLIMB-111 has been expanded to allow enrollment of 0/0 patients and is in the process of being expanded to allow enrollment of pediatric patients ages 12 years or older.

CLIMB-121 for SCD has dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients.

The initial safety profile in these trials appears to be consistent with myeloablative busulfan conditioning and an autologous hematopoietic stem cell transplant.

In March 2020, clinical trial sites in the U.S. and Europe temporarily paused their elective hematopoietic stem cell transplant programs due to the COVID-19 pandemic, and as a result, CRISPR and Vertex temporarily paused conditioning and dosing in these trials. Enrollment, mobilization and drug product manufacturing in each trial remains ongoing. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites. The CLIMB-111 and CLIMB-121 clinical trials are ongoing, and patients will be followed for 2 years following CTX001 infusion. The companies expect to provide additional data in the second half of 2020.

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

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT) from the U.S. FDA, Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), and Fast Track Designation from the FDA for both SCD and TDT.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.

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

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

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

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

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, 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 Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases and activities at clinical trial sites) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is being presented at the European Hematology Associations virtual congress; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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

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

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, and statements regarding our plans and expectations for our clinical trials and clinical trial sites, and our expectations regarding future data announcements. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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

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

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

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

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CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies at the 25th...

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The report offers a systematic presentation of the existing trends, growth opportunities, market dynamics that are expected to shape the growth of the CRISPR and CAS Gene market. The various research methods and tools were involved in the market analysis, to uncover crucial information about the market such as current & future trends, opportunities, business strategies and more, which in turn will aid the business decision-makers to make the right decision in future.

This Report Covers Leading Companies Associated in Worldwide CRISPR and CAS Gene Market: Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

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The report begins with a brief introduction and market overview of the CRISPR and CAS Gene industry followed by its market scope and size. Next, the report provides an overview of market segmentation such as type, application, and region. The drivers, limitations, and opportunities for the market are also listed along with current trends and policies in the industry.

The key players profiled in this report include: Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Regions included:

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

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

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

o South America (Brazil, Argentina, Colombia)

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

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Key Benefits:

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

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

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

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

The research study can answer the following Key questions:

Table of Contents

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

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

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

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

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Impact of COVID-19 on CRISPR and CAS Gene Market Potential Growth and Forecast Period 2020-2027 | By Leading Players Caribou Biosciences Inc., CRISPR...

Global Gene Editing Tools market report gives a top to bottom investigation of the general market over a period from 2020-2027. The Gene Editing Tools Market report additionally gives the market sway and new open doors made because of the COVID19 fiasco.

As a training, this research report consolidating the effect of COVID19 available in each report as a worth included segment. Report incorporate income Impact investigation, interruptions and new open doors in the gracefully chain, overhauled merchant scene blend, new open doors mapping, and then some. Report resolved to rise triumphant against this COVID-19 pandemic and to guarantee that customers have zero to least disturbance in business results.

The global Gene Editing Tools market report covers major market players such as

Thermofisher ScientificCRISPR TherapeuticsEditas MedicineNHGRIIntellia TherapeuticsMerck KGaAHorizon

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The Gene Editing Tools market report next half moreover sheds investigate on the hole among flexibly and utilization. Aside from the referenced business development pace of market in 2027 is additionally clarified. Moreover, type and application insightful utilization tables and figures of Gene Editing Tools market are given.

Execution examination of Gene Editing Tools industry 2020 featuring ongoing business sector development, patterns and advancement 2027 figure report. The global Gene Editing Tools market report additionally contemplates the assembling cost structure and presents the different subtleties, for example, crude material, the general creation process, and the business chain structure.

Global Gene Editing Tools Market Segmentation By Type:

Zinc finger nucleases (ZFNs)Transcription Activator-Like Effector-based Nucleases (TALENs)CRISPR-Cas system

Global Gene Editing Tools Market Segmentation By Applications:

Sickle Cell DiseaseHeart DiseaseDiabetesAlzheimers DiseaseObesityOthers

Global Gene Editing Tools Market Segmentation By Regions:

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Furthermore, the Gene Editing Tools report consists of the important information related to the growth rate, top players of the parent market, product development, and others also by considering the past and present values of the market report predicting the forecasting values of the Gene Editing Tools market. Along with these things report selected the appropriate SWOT analysis which guides the various opportunities and growth scope for the Gene Editing Tools market.

The research report Gene Editing Tools market consist the in-depth information about the data analysis by using the figures, graphs, pie charts, tables and bar graphs. With the help of these users easily understand the analyzed data in a better and easy way. Also, the report provides the different business challenges which are impacting market growth in a positive and negative direction.

There are 13 Chapters to display the Global Gene Editing Tools market:

Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation overviewChapter 2: Market Competition by ManufacturersChapter 3: Production by RegionsChapter 4: Consumption by RegionsChapter 5: Production, By Types, Revenue and Market share by TypesChapter 6: Consumption, By Applications, Market share (%) and Growth Rate by ApplicationsChapter 7: Complete profiling and analysis of ManufacturersChapter 8: Manufacturing cost analysis, Raw materials analysis, Region-wise manufacturing expenses.Chapter 9: Industrial Chain, Sourcing Strategy and Downstream BuyersChapter 10: Marketing Strategy Analysis, Distributors/TradersChapter 11: Market Effect Factors AnalysisChapter 12: Market ForecastChapter 13: Gene Editing Tools Research Findings and Conclusion, Appendix, methodology and data source.

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Global Gene Editing Tools Market 2020 Impact of COVID-19, Future Growth Analysis and Challenges | Thermofisher Scientific, CRISPR Therapeutics,...

Also known as genome editing with engineered nucleases (GEEN), genome editing is a method of altering DNA within a cell in a safe manner. The technique is also used for removing, adding, or modifying DNA in the genome. By thus editing the genome, it is possible to change the primary characteristic features of an organism or a cell.

The global genome editing market can be segmented on the basis of delivery method, technology, application, and geography. By technology, the global genome editing market can be segmented into Flp-In, CRISPR, PiggyBac, and ZFN. Based on delivery method, in vivo and ex vivo can be the two broad segments of the global genome editing market. By application, the global genome editing market can be categorized into medicine, academic research, and biotechnology.

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Global Genome Editing Market: Key Trends

Since genome editing is gaining rising adoption in the domain of scientific research for attaining a better understanding of biological aspects of organisms and how they work, the global genome editing market is likely to promise considerable growth over the forthcoming years. More importantly, genome editing is being used by medical technologies, where it can be used for modifying human blood cells which can then be placed back in the body for treating conditions such as AIDS and leukemia. The technology can also be potentially utilized to combat infections such as MRSA as well as simple genetic disorders including hemophilia and muscular dystrophy.

Global Genome Editing Market: Market Potential

As more easy-to-use and flexible genome technologies are being developed, greater potential of genome editing is being recognized across bioprocessing and treatment modalities. For instance, in May 2017, MilliporeSigma announced that it successfully developed a novel genome editing tool which can make the CRISPR system more productive, specific, and flexible. The researchers thus have a more number of experimental options along with faster results.

All this can lead to a growing rate of drug development, enabling access to more advanced therapies. Proxy-CRISPR, the new technique, makes access to earlier inaccessible aspects of the genome possible. As most of the existing CRISPR systems cannot manage without re-engineering of human cells, the new method is expected to gain more popularity by virtue of the elimination of the need for re-engineering, simplifying the procedures.

Several other market players are focusing on clinical studies with a view to produce effective treatments for different health conditions. For example, another major genome editing firm, Editas Medicine, Inc. announced the results of its pre-clinical study displaying the success of the CEP290 gene present in the retina of primates in the same month. With the positive results of the study, the companys belief in the vast potential of its candidate in the treatment of a genetically inherited retinal degenerative disease, Leber congenital amaurosis type 10, affecting childrens eyesight has been reinforced.

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Global Genome Editing Market: Regional Outlook

By geography, the global genome editing market can be segmented into Latin America, Europe, Asia Pacific, the Middle East and Africa, and North America. North America registered the highest growth in the past, and has been claiming the largest portion of the global genome editing market presently. The extraordinary growth of this region can be attributed to greater adoption of cutting edge technologies across several research organizations. The U.S., being the hub of research activities, is expected to emerge as the leading contributor. Asia Pacific is also likely to witness tremendous demand for genome editing over the forthcoming period, assisting the expansion of the global genome editing market.

Global Genome Editing Market: Competitive Analysis

CRISPR THERAPEUTICS, Caribou Biosciences, Inc., Sigma Aldrich Corporation, Sangamo, Intellia Therapeutics, Inc., Editas Medicine, Thermo Fisher Scientific, Inc., and Recombinetics, Inc are some of the key firms operating in the global genome editing market.

Read more from the original source:
Genome Editing Market Predicted to Accelerate the Growth by 2017-2025 - Owned

The Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market report offers users the detailed study of the market and its main aspects. There are different marketing strategies that every marketer looks up to in order to ace the competition in the Global market. Some of the primary marketing strategies that is needed for every business to be successful are Passion, Focus, Watching the Data, Communicating the value To Your Customers, Your Understanding of Your Target Market. There is a target set in market that every marketing strategy has to reach. Some of the important aspects analyzed in the report includes market share, production, key regions, revenue rate as well as key players. This Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology report also provides the readers with detailed figures at which the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market was valued in the historical year and its expected growth in upcoming years. Besides, analysis also forecasts the CAGR at which the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology is expected to mount and major factors driving markets growth.

This study covers following key players:Thermo Fisher ScientificMerckGenScriptIntegrated DNA TechnologiesHorizon Discovery GroupAgilent TechnologiesCellectaGeneCopoeiaNew England BiolabsOrigene TechnologiesSynthego CorporationToolgen

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A significant development has been recorded by the market of Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology, in past few years. It is also for it to grow further. To analyze the Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market, the analysis methods used are SWOT analysis and PESTEL analysis. To identify what makes the business stand out and to take the chance to gain advantage from these findings, SWOT analysis is used by marketers. Whereas PESTEL analysis is the study concerning Economic, Technological, legal political, social, environmental matters. For the analysis of market on the terms of research strategies, these techniques are helpful. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry.

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Market segment by Type, the product can be split intoProductsServices

Market segment by Application, split intoBiomedical ApplicationsAgricultural ApplicationsIndustrial ApplicationsBiological Research

A systematized methodology is used to make a Report on the Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market. For the analysis of market on the terms of research strategies, these techniques are helpful. All the information about the Products, manufacturers, vendors, customers and much more is covered in research reports. The market tends to be highly competitive in nature as the number of vendors present in the market is too high.

The Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market has its impact all over the globe. On Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology industry is segmented on the basis of product type, applications, and regions. It also focusses on market dynamics, Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology growth drivers, developing market segments and the market growth curve is offered based on past, present and future market data. The industry plans, news, and policies are presented at a Global and regional level.

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Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market 2020 By Size, Share, Trend, Production, High Demand,...

As businesses and schools seek to reopen, most public health experts agree that Covid-19 testing is needed more than ever.

The team behind Ginkgo Bioworks, a genetic engineering start-up, is going all in.

Using equipment from Illumina, a maker of DNA sequencing machines, the company which made CNBC's 2020 Disruptor 50 list is working on technology to run a half million tests per day, said Jason Kelly, Ginkgo's co-founder and CEO.The technology, if approved by federal regulators, will be saliva-based, which in theory would make it easier for consumers to get tested than using the nasal swabs most tests employ today.

Jason Kelly, Founder, Ginkgo Bioworks

Scott Mlyn | CNBC

"We didn't initially have enough tests, but now we've ramped up to about 400,000 per day," he said. "That's enough for our clinical diagnostics needs. ... However, we are now entering phase 2 of this thing."

Ginkgo, a darling of the burgeoning synthetic biology sector, got its start in 2009 when a group of MIT scientists got together to develop biotechnology tools for industries including agriculture, pharmaceuticals and cosmetics. In essence, it develops custom microorganisms that aim to replace technology with biology. Think of it as a way to program cells, a bit like you'd program computers.

"We program DNA and cells to make them do new things," said Kelly, who describes the company as the largest designers of "printing DNA" in the world.

The Boston-based company has raised close to $1 billion to date, as investors clamor to throw money into companies at the intersection of health care and technology. Kelly maintains that enthusiasm is warranted. The cost of sequencing DNA data is coming down faster than the cost of processing data on computers,outpacingMoore's law.

As Kelly puts it, "the chips aren't getting that much faster," but in biology things are "exponentially improving."

Right now Kelly believes Ginkgo can best apply its technology to help ramp up coronavirus testing in the U.S.

As the CEO explained, the initial crop of tests were primarily used to determine if people experiencing Covid-19 symptoms did, in fact, have the virus. But now, as people are going back to their lives, there will be an increasing need for regular testing of people who don't have symptoms.

For instance, Amazon plans to test its fulfillment center workers every two weeks, as well as monitor outbreaks in the community.

That means the country is going to need a lot more coronavirus tests.

Ginkgo started surveyingthe various techniques to scale up testing back in the spring, including antigen (a technology that looks for viral surface proteins), CRISPR-based (a genome editing technique) and next-generation sequencing approaches. Companies have only recently been granted emergency-use authorizations from the U.S. Food and Drug Administration for these types of tests. All of them, if ramped up, could theoretically augment the polymerase chain reaction tests that are currently the gold standard for Covid-19.

Ginkgo has decided to focus on next-generation sequencing with Illumina, which has already been granted an emergency-use approvalfor its Covid-19 test that is designed to sequence the full genome of the virus.

"Beyond diagnostic testing, Illumina and a number of our customers are exploring NGS-based workflows to enable high-volume screening to support a return to work and school," Illumina CEO Francis deSouza said in a statement.

In May Ginkgo announced it had raised another $70 million, including from Illumina, to fund its expansion in the diagnostics field. It is also using the money tobuild out its own testing facility in its highly automated Boston Seaport labs.

More from Disruptor 50:Moderna CEO sees success with Covid-19 vaccineThe technology that will dominate daily life on the other side of coronavirusCLEAR poised to lead in biometric screening for Covid

Kelly can't predict exactly when the company will be rolling out its tests, but it hopes to get FDA approval this summer. He said the company is already starting to work with businesses to help advise them as they determine how to safely get employees back to work. Many are concerned about a potential second shutdown if there's another outbreak in their area.

He believes that testing and contact tracing, where government officials track down and warn people who might have been exposed to Covid-19, are key to reopening the economy.

"I'm also sensing that a lot of people don't have a ton of hope," Kelly said. "Now we have to really try. If we try, we can win."

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Ginkgo Bioworks CEO on scaling up Covid-19 testing: 'If we try, we can win' - CNBC

CRISPR Therapeutics and Vertex have presented updated data on the first patients treated with their CRISPR/Cas9 gene-editing therapy. The partners now have evidence that one-time treatment with CTX001 improves outcomes in sickle cell disease and beta thalassemia patients for up to 15 months.

In November, CRISPR and Vertex moved the gene-editing field beyond an early milestone by linking the use of CTX001 to sustained improvements in the health and biomarkers of two patients with the severe hemoglobinopathies sickle cell disease and transfusion-dependent beta thalassemia. Late last week, the partners used the European Hematology Association virtual congress to share an update on the studies.

CRISPR and Vertex now have data on two beta thalassemia patients. The first patient, who had nine months of follow up as of last years update, has now been tracked out to 15 months after treatment with CTX001. The patients levels of total hemoglobin, fetal hemoglobin and erythrocytes expressing fetal hemoglobin (F-cells) increased between the two updates.

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Submit your entry to demonstrate innovative technologies and services that have the potential to make the greatest impact for biotech and pharma companies.

The partners also have five-month data on a second beta thalassemia patient. The hemoglobin and F-cell levels of the second patient are close to or above those of the first patient at the nine-month mark. Both patients experienced two serious adverse events, none of which the investigator considered to be related to CTX001.

CRISPR and Vertex also used the virtual event to share a nine-month update on a sickle cell patient. Compared to the four-month readout, total hemoglobin and F-cells are up. Fetal hemoglobin is down slightly, but still well above the level likely needed to be efficacious.

The improved biomarker data are supported by evidence the drug is making a meaningful difference to the lives of the patients. The beta thalassemia patients are transfusion independent, having required 34 and 61 units of packed red blood cells a year previously. The subject who required 34 blood units underwent 33 transfusions over the two years before consenting to join the study.

In the sickle cell trial, the patient used to suffer seven vaso-occlusive crises a year, on average. Over the nine months in the study, the patient has been free from vaso-occlusive crises.

The data, which the author of the abstract said demonstrate a functional cure, support the further assessment of CTX001. Efforts to gather more data were hindered by COVID-19, which led to the temporary cessation of elective hematopoietic stem cell transplants at sites in the U.S. and Europe. Some sites are now gearing up to reinitiate dosing.

Even if the restart progresses slowly, CRISPR and Vertex will still be in a position to share more data later in the year. Investigators dosed another three beta thalassemia patients and one sickle cell patient before COVID-19 hit, setting them up to share updates as those subjects pass follow-up milestones.

The updates should also feature longer-term data on the first three patients treated in the trials. In posting long-term data, CRISPR and Vertex will begin to show whether CTX001 has the durability to be a true one-time treatment and, in doing so, carve out a space in a market fought over by rivals such as bluebird bio.

Originally posted here:
CRISPR and Vertex show durability of gene-editing therapy, hoping for one and done treatment - FierceBiotech

New Jersey, United States,- A detailed research study on CRISPR and Cas Genes Market recently published by Verified Market Research. This is the latest report, which covers the time COVID-19 impact on the market. Pandemic Coronavirus (COVID-19) has affected every aspect of global life. This has brought some changes in market conditions. Rapidly changing market scenario and the initial assessment and the future of this effect is included in the report. Reports put together a brief analysis of the factors affecting the growth of the current business scenarios in various areas. Important information relating to the size of the industry analysis, sharing, application, and statistics summed up in the report to present the ensemble prediction. In addition, this report includes an accurate competitive analysis of major market players and their strategies during the projection period.

This report includes market size estimates for the value (million USD) and volume (K Units). Both top-down and bottom-up approach has been used to estimate the size of the market and validate the Market of CRISPR and Cas Genes, to estimate the size of the various submarkets more dependent on the overall market. Key players in the market have been identified through secondary research and their market share has been determined through primary and secondary research. All the shares percentage, split, and the damage have been determined using secondary sources and primary sources verified.

Leading CRISPR and Cas Genes manufacturers/companies operating at both regional and global levels:

CRISPR and Cas Genes Market Competitive Landscape & Company Profiles

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the CRISPR and Cas Genes market.

Segmental Analysis

Both developed and emerging regions are deeply studied by the authors of the report. The regional analysis section of the report offers a comprehensive analysis of the global CRISPR and Cas Genes market on the basis of region. Each region is exhaustively researched about so that players can use the analysis to tap into unexplored markets and plan powerful strategies to gain a foothold in lucrative markets.

Regions Covered in these Report:

Asia Pacific (China, Japan, India, and Rest of Asia Pacific)Europe (Germany, the UK, France, and Rest of Europe)North America (the US, Mexico, and Canada)Latin America (Brazil and Rest of Latin America)Middle East & Africa (GCC Countries and Rest of Middle East & Africa)

To get Incredible Discounts on this Premium Report, Click Here @ https://www.verifiedmarketresearch.com/ask-for-discount/?rid=40530&utm_source=COD&utm_medium=007

CRISPR and Cas Genes Market Research Methodology

The research methodology adopted for the analysis of the market involves the consolidation of various research considerations such as subject matter expert advice, primary and secondary research. Primary research involves the extraction of information through various aspects such as numerous telephonic interviews, industry experts, questionnaires and in some cases face-to-face interactions. Primary interviews are usually carried out on a continuous basis with industry experts in order to acquire a topical understanding of the market as well as to be able to substantiate the existing analysis of the data.

Subject matter expertise involves the validation of the key research findings that were attained from primary and secondary research. The subject matter experts that are consulted have extensive experience in the market research industry and the specific requirements of the clients are reviewed by the experts to check for completion of the market study. Secondary research used for the CRISPR and Cas Genes market report includes sources such as press releases, company annual reports, and research papers that are related to the industry. Other sources can include government websites, industry magazines and associations for gathering more meticulous data. These multiple channels of research help to find as well as substantiate research findings.

Table of Content

1 Introduction of CRISPR and Cas Genes Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 CRISPR and Cas Genes Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 CRISPR and Cas Genes Market, By Deployment Model

5.1 Overview

6 CRISPR and Cas Genes Market, By Solution

6.1 Overview

7 CRISPR and Cas Genes Market, By Vertical

7.1 Overview

8 CRISPR and Cas Genes Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 CRISPR and Cas Genes Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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CRISPR and Cas Genes Market Analysis, Trends, Top Manufacturers, Growth, Statistics, Opportunities and Forecast To 2026 - Cole of Duty

For us, stock picking is in large part the hunt for the truly magnificent stocks. But when you hold the right stock for the right time period, the rewards can be truly huge. One such superstar is CRISPR Therapeutics AG (NASDAQ:CRSP), which saw its share price soar 305% in three years. On top of that, the share price is up 62% in about a quarter. But this move may well have been assisted by the reasonably buoyant market (up 30% in 90 days).

View our latest analysis for CRISPR Therapeutics

While the efficient markets hypothesis continues to be taught by some, it has been proven that markets are over-reactive dynamic systems, and investors are not always rational. One flawed but reasonable way to assess how sentiment around a company has changed is to compare the earnings per share (EPS) with the share price.

CRISPR Therapeutics became profitable within the last three years. That kind of transition can be an inflection point that justifies a strong share price gain, just as we have seen here.

You can see below how EPS has changed over time (discover the exact values by clicking on the image).

We know that CRISPR Therapeutics has improved its bottom line lately, but is it going to grow revenue? This free report showing analyst revenue forecasts should help you figure out if the EPS growth can be sustained.

Were pleased to report that CRISPR Therapeutics rewarded shareholders with a total shareholder return of 27% over the last year. But the three year TSR of 59% per year is even better. While it is well worth considering the different impacts that market conditions can have on the share price, there are other factors that are even more important. Take risks, for example CRISPR Therapeutics has 3 warning signs we think you should be aware of.

For those who like to find winning investments this free list of growing companies with recent insider purchasing, could be just the ticket.

Please note, the market returns quoted in this article reflect the market weighted average returns of stocks that currently trade on US exchanges.

Love or hate this article? Concerned about the content? Get in touch with us directly. Alternatively, email 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. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Thank you for reading.

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DUBLIN--(BUSINESS WIRE)--The "Global Molecular Diagnostics Market - Technologies, Products, Applications and End-Use Sectors" report has been added to ResearchAndMarkets.com's offering.

Amid the COVID-19 outbreak, the global market for Molecular Diagnostics has created mixed opportunities for manufacturers all over the world. While some of the application segments, such as infectious diseases, is projected to record the highest growth during the pandemic period of 2019-2022, other applications such as oncology, etc. are estimated to face a reduction in demand during the same period.

The Global Molecular Diagnostics market has generated a revenue of US$10.85 billion in 2019 and is estimated to be just over US$15 billion in 2020. During 2019 to 2022 period, which is said to be influenced by the Covid-19 pandemic, the Infectious Diseases segment is anticipated to gain rapid demand, growing at a robust CAGR of 14.9%. The Infectious Diseases segment, in which Covid-19 testing is a part, is estimated to generate an extra revenue of US$3.8 billion globally in 2020 compared to 2019.

Research Findings & Coverage

Key Topics Covered:

PART A: GLOBAL MARKET PERSPECTIVE

1. INTRODUCTION

2. KEY MARKET TRENDS

2.1 Speedier Results Promised by New Test for COVID-19

2.2 COVID-19 Pandemic Takes Korean Testing Kits Demand to New Heights

2.3 New CRISPR-Based Technology Developed for COVID-19 Identification

2.4 Tracing Incidences of Salmonella Food-Poisoning to be Enhanced through New DNA Test

2.5 Molecular Diagnostics of Infectious Diseases Undergo Dramatic Advancements

2.6 Monitoring Treatment of Residual Disease in High-Risk Neuroblastoma Patients Facilitated by Innovative Method

2.7 Time for Diagnosing Sepsis Shortened Significantly Via Novel Next-Generation Sequencing Technique

2.8 Monitoring Antimalarial Drug Resistance Made Easier by Blood Drop Assay

2.9 Silicon Based-Nanostructured Microfluidics - A Novel Molecular Diagnostic Technology for Fetal Aneuploidy

2.10 Creation of Novel and Powerful Adaptive PCR Technique for Rapid Genetic Analysis

2.11 Web Tool Foretells Phenotypes Using little DNA Sample

2.12 Emergence of CRISPR as a Novel Molecular Diagnostic Tool

3. KEY GLOBAL PLAYERS

4. KEY BUSINESS TRENDS

5. GLOBAL MARKET OVERVIEW

PART B: REGIONAL MARKET PERSPECTIVE

REGIONAL MARKET OVERVIEW

6. NORTH AMERICA

7. EUROPE

8. ASIA-PACIFIC

9. REST OF WORLD

PART C: GUIDE TO THE INDUSTRY

PART D: ANNEXURE

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/7q2nqy

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Molecular Diagnostics Market - Technologies, Products, Applications and End-Use Sectors to 2022 - ResearchAndMarkets.com - Business Wire

(Editors note: This article about a breakthrough technology bootstrapped with a loan from NCBiotech originally appeared Friday, June 12, in the Duke University Medical School online publicationMagnify. Used with permission.)

DURHAM Life scientists love antibodies, not only because these little proteins help protect us all from pathogens, but because antibodies are also a very handy laboratory tool for identifying and marking proteins of interest in their research.

When youre trying to find something very tiny, you need an itty bitty flag to mark it. Thats an antibody.

Like most life science researchers, Duke cell biology chairScott Soderlinghas been reliant on custom antibodies, molecules made-to-order by hundreds of different supply labs that help scientists find and mark specific proteins in cell cultures and living organisms.

But theres a problem, he explains in the small conference room adjacent to his Nanaline Duke office. Fifty percent of the antibodies on the market are junk. Theyre not specific. They might bind what you think they bind, but then they bind to other things you dont know about, or they dont even bind what you want to bind to at all.

Worse than that, one batch of bespoke antibodies may not be the same as the last one. Say you have a perfect antibody that binds exactly what you want and nothing else. And then you order the next lot and theres a different preparation from a different animal, and youre back to square one. It doesnt work.

Scott Soderling. Les Todd photo

Its thought that these bad antibodies lead to a large fraction of the irreproducible results, Soderling says. So it costs money, it costs time and it costs credibility. This is a huge problem for science, both academic and industry. In part, the problem stems from the fact that custom antibody manufacturing techniques date to the 1970s, he says.

But Soderling has founded a Duke spinout company he hopes will solve the reliability problem.CasTag BioSciencesis based on a technology developed in his lab that marks proteins of interest in an entirely new way, using the genome-editing tool CRISPR.

One major thrust of Soderlings research has been identifying proteins in the synapses of the brain, the tiny gaps between nerve cells where signals are transmitted and received. All that signaling is regulated by specific proteins. But identifying all of those proteins in the synapse and interpreting what theyre saying to the cell is a huge problem in a very tiny space. Antibodies are a key tool, but the work has been frustrating and slow, in part because of the difficulty of working with custom antibodies.

About three years ago, as news of the new gene-editing technology called CRISPR spread, Soderling and his team wanted to see if it might give them a better way to label and visualize the hundreds and even thousands of proteins they were detecting in the tiny synapse between neurons.

We had this idea that CRISPR could be a really amazing tool to address the pressing problem of trying to identify and label these hundreds of proteins, Soderling says. What we developed was a new modular method for basically taking the labeling problem and flipping it on its head.

Theyre using CRISPR to edit short sequences into a gene so that every protein it produces carries a tag they have created that is detected by a known, reliable and well-characterized antibody, rather than a shot-in-the-dark custom antibody.

Based on CRISPR gene editing technology, Homology-independentUniversal Genome Engineering, or HiUGE, uses adeno-associatedvirusesto deliver multiple plug and play gene sequencesto a varietyof cellsin a lab dish or a living organism. (The colored neurons in thisimage are in a mouse brain.)

These antibodies recognize a small segment of amino acid sequences, Soderling explains. So we just take the DNA encoding those amino acids the handle and we plop that handle right into the gene in vivo, or in the cell, Soderling says.

After the proof-of-concept experiments produced beautiful protein labeling in the mouse brain, Soderling looked at the images and said, Okay its huge.

Indeed, they dubbed their new system HiUGE (homology-independent universal genome engineering), and it might just be huge indeed.

Theyve taken to calling it plug and play biology, because with just a few of their tags, they can address hundreds of unknown proteins, and they can even put multiple tags into a gene at the same time. Soderling says the system is modular and easy to use, which will enable semi-automated, high-throughput approaches to labeling proteins.

By way of analogy, think of a delivery truck driver going slowly down the block after dark in a downpour looking for house number 2345. What Soderling and his team have done is put a bright sign on every house numbered 2345 that says Hey UPS! Over here!

The HiUGE system is delivered to living cells, either in a dish or in an organism, by a pair of adeno-associated viruses working as a team. One virus carries guide RNA which will mark the spot at which CRISPR should cut the DNA and insert a new piece of code. The second adeno-associated virus carries the payload, a tag or tags theyve devised that will now be built into every protein that gene subsequently produces.

The vectors, including a synthetic guide RNA and HiUGE tags, are agnostic, or homology-independent, as the name implies. They dont care what gene is around them. We designed this guide RNA so that it specifically doesnt recognize anything in the mouse, human, monkey, cat or donkey genomes, Soderling says.

Its a clever way to explore the unknown.

Not only does this approach advance their own work, Soderling began to realize that a fast, flexible, more accurate way to tag proteins might also be a business opportunity. With a little research, he figured out that custom antibodies are a $2.4 billion market again, with products that only work as advertised half the time.

He reached out to Dukes Office of Licensing and Ventures (OLV) to begin the patenting process and to get some advice on starting a company. Then I had to find a way to run the business, because I already have a great day job. In fact, he had also just been named chair of cell biology at about the same time.

At OLVs recommendation, Soderling visited Biolabs North Carolina, a shared workspace in the Chesterfield Building in downtown Durham which leases individual wet-lab benches on a month-to-month basis and provides all the basic equipment a startup would need, including refrigeration, gene-copying PCR machines, centrifuges, etc. He pitched his idea to Biolabs and had a look around.

The next day, BioLabs NC president Ed Field called Soderling and asked if hed like some help running the business. Field, a startup veteran, is now the CEO of CasTag. The firm has raised enough money with a loan from the North Carolina Biotechnology Center to hire a recent Fuqua Business School graduate as the business development lead and a former postdoc for Soderling to run the lab part-time while he looks for a job in industry.

Weve got a website. Weve got orders. Weve got customers. Its up and running, Soderling says, with a measure of wonder in his voice. His conference talks about HiUGE and a July 1, 2019 paper in Neuron attracted some attention. Then the paper was republished as one of the journals best of 2018-2019, drawing still more notice.

And now they also have ideas for new products. Im hoping that this will expand and become even bigger than just tagging proteins, Soderling says.

You know, North Carolina was a manufacturing state back in the day, says Soderling, a soft-spoken native Tennessean. I would love to wake up some day and drive into downtown Durham and see one of the former manufacturing warehouses humming away with people making these reagents to ship out around the world. Thats the dream.

Durham academic research services companyResearch Squarehas producedthis 3 1/2-minute Vimeo videoexplaining the CasTag BioSciences technology.

(c) North Carolina Biotechnology Center

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Duke spinout CasTag BioSciences builds a better protein trap with boost from NCBiotech - WRAL Tech Wire

[269 Pages Report] Mice Model Market report categorizes the Global market by Type (Inbred, Knockout, Hybrid), Technology (CRISPR, Microinjection), Application (Oncology, Diabetes), Service (Breeding, Quarantine, Genetic testing) & Care Products (Bedding, Feed) & Geography. COVID-19 impact on Mice Model Industry.

MarketsandMarkets forecaststhemice model marketto grow from USD 1.11 billion in 2016 to USD 1.59 billion by 2021, at a Compound Annual Growth Rate (CAGR) of 7.5%during the forecast period. The growth of the market can be attributed to ongoing innovations in mice models, continuous support in the form of investments and grants, and growing demand for humanized mice models.

By Technology, the CRISPR technology segment to account for the largest share of the global mice model market in 2016

Based on technology, the market is segmented into CRISPR/Cas9, microinjection, embryonic stem cell injection, nuclear transfer, and other technologies. The CRISPR technology accounts for the largest share of the global mice model market in 2016.

The large share of this segment can primarily be attributed to the fact that CRISPR is the most widely used technique due to the various advantages associated with it, such as ease of design, high efficiency, and relatively low cost.

Emergence of CRISPR as a powerful tool in the field of biomedical research

CRISPR (clustered, regularly interspaced, short palindromic repeat) is seen as a revolutionary technology for gene editing. The use of Cas9 enzyme differentiates CRISPR from other forms of genetic modification.

This technology edits and rearranges genes by cutting out damaged or unwanted parts of the DNA, allowing the remaining DNA to be rearranged in a new way.

Moreover, this fast, precise, and easy-to-use technology is considered as a revolutionary tool in research, and there is an intense interest to validate its therapeutic usage in humans.

CRISPR was first shown to work in mouse and human cells less than three years ago and has already been applied to a range of biological systems and disease areas. CRISPR is used in the development of genetically modified mice strains, making the process not only quicker but also less expensive.

Thus, the emergence of CRISPR as a popular technology is expected to offer potential growth opportunities in the coming years.

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North America to account for the largest market size during the forecast period.

North America is accounted for the largest share of the market. North Americas leadership in the market can be attributed to the increased focus on biomedical research in the U.S., rising demands for monoclonal antibody production, nexus between CROs and pharmaceutical companies, continued and responsible use of animals ensured by animal care organizations, rising preclinical activities by CROs and pharmaceutical companies, and growing stem cell research in Canada.

Asia Pacific is the third-largest market for mice model market and is slated to register the highest CAGR of 7.9% during the forecast period. The high growth in the region can be attributed to less stringent regulations on the use of animal models for research in the region, international alliances for R&D activities in China, growth in regenerative medicine, biomedical, and medical research in Japan, growing presence of global players, development of bioclusters to boost the biotechnology industry in India, ongoing biomedical research activities in Australia, and rising pharmaceutical & biotechnology R&D activities.

Key Market Players

Charles River Laboratories International, Inc. (U.S.), The Jackson Laboratory (U.S.), Taconic Biosciences, Inc.(U.S.), TRANS GENIC Inc. (Japan), Horizon Discovery Group plc (U.S.), Envigo (U.K.), Laboratory Corporation of America Holdings (U.S.), and genOway (France).

Charles River Laboratories is a leading player in the global mice models and services market. The mice models offered by the company include inbred, outbred, hybrid, immunodeficient, congenic, and genetically engineered mice.

The company also offers mice modeling services such as surgical services for rodents, genetic testing services, colony management services, and health monitoring services. The strong portfolio enables the company to increase collaboration with clientsfrom early lead generation to candidate selection.

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Explore the Mice Model Market: CRISPR As a Powerful Tool in the Field of BioMedical Research - WhaTech Technology and Markets News

Biotech companies from across the globe are posting strong data at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress. Below is a roundup of some of the news coming out of the virtual conference.

bluebird bio Data from a Phase I/II study of Cambridge, Mass.-based bluebird bios gene therapy treatment LentiGlobin in adult patients with sickle cell disease has shown a show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS), the company said. VOCs are life-threatening episodes that are the primary manifestation of sickle cell disease. A nearly complete elimination of this demonstrates the potential of LentiGlobin in this indication, David Davidson, the chief medical officer at bluebird said in a statement. Data from the Phase I/II study showed a 99.5% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome in patients. Two years after those patients remained free from VOCs and ACS, bluebird said.

These results illustrate the type of outcomes we believe are needed to provide truly meaningful improvements for people living with sickle cell disease. In addition, the improvement of laboratory measures of hemolysis and red cell physiology, with nearly pan-cellular distribution of the anti-sickling HbAT87Q, suggest LentiGlobin for SCD may substantially modify the causative pathophysiology of SCD, he said.

Based on these results, bluebird said it plans to seek an accelerated approval from the U.S. Food and Drug Administration for LentiGlobin in this indication.

Additionally, bluebird released data from its Phase III study of betibeglogene autotemcel (formerly LentiGlobin gene therapy for -thalassemia) in transfusion-dependent -thalassemia. Data showed the majority of patients achieved transfusion independence and were able to maintain it with near-normal hemoglobin levels, bluebird said.

CRISPR Therapeutics Working with its partner Vertex Pharmaceuticals, CRISPR Therapeutics presented new clinical data for CTX001, an investigational CRISPR/Cas9 gene therapy, from the CLIMB-111 and CLIMB-121 Phase I/II trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease, respectively. In the CLIMB-111 trial, data demonstrated clinical proof-of-concept for CTX001 in TDT based on the first patient dosed. Updated data presented at the conference showed that 15 months following treatment with CTX001, the patient was transfusion independent. Data from the second patient treated with CTX001 was also promising. After five months, the patient was transfusion independent. Both patients experienced serious adverse events, but they were determined to not be related to the treatment.

In the CLIMB-121 trial, the data showed that nine months after CTX001 infusion, the patient was free of VOCs, transfusion independent and had improved hemoglobin levels. The patient that formed the basis for this early study also experienced SAEs, but they were determined to not be related to the treatment.

ADC Therapeutics Less than a month after it closed on a $267 million IPO, ADC Therapeutics announced updated data from its pivotal Phase II study of the companys lead candidate loncastuximab tesirine (Lonca) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) at the conference. The company also presented interim results from its Phase I/II trial of Lonca in combination with ibrutinib. In the Phase II LOTIS 2 study, ADC said Lonca demonstrated anti-tumor activity and durability in a broad population of difficult-to-treat patients with relapsed or refractory DLBCL. Data showed that Lonca provided an overall response rate of 48.3% and a complete response rate of 24.1%. The study also showed a median duration of response of 10.25 months. The company said Lonca had a manageable toxicity profile. ADC Therapeutics is on track to submit a BLA to the FDA for Lonca in the second half of this year and, if approved, the company plans to launch the product sometime in mid-2021.

ADC also noted at the conference that interim results from the Phase I/II LOTIS 3 study of a combination of Lonca and Janssens Imbruvica (ibrutinib) demonstrate the potential of Lonca to be used for earlier lines of therapy in combination with other therapies in patients with relapsed or refractory DLBCL or mantle cell lymphoma. Interim data showed an ORR of 66.7% and a CRR of 50%, ADC noted.

Apellis Pharmaceuticals Waltham, Mass.-based Apellis presented results from its Phase III PEGASUS study at the conference that showed detailed data from the results first released in January, which showed superiority for pegcetacoplan over eculizumab in improving hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH). New data from the pivotal study showed that pegcetacoplans effect was seen consistently across the study population, both in patients who had low or no transfusion requirements and high transfusion requirements. Pegcetacoplan also demonstrated a robust response across several key hematologic and clinical measures for PNH. The study showed that 71% of pegcetacoplan-treated patients achieved LDH normalization in comparison to 15% of eculizumab-treated patients. Additionally, 73% of pegcetacoplan-treated patients achieved a clinically meaningful improvement in FACIT-fatigue score in comparison to 0% of eculizumab-treated patients, the company said.

With the strong results in hand, Apellis plans to submit a New Drug Application to the FDA and a Marketing Authorization Agreement to the European Medicines Agency for pegcetacoplan for the treatment of PNH in the second half of 2020.

Takeda Takeda Oncologyannouncedpositivedata from two studies of Ninlaro,an oral proteasome inhibitorthatis being studied across the continuum of multiple myeloma treatment settings. Phase III data from theTOURMALINE-MM4trialdemonstrate thattreatment with NINLARO resultedin a statistically significant and clinically meaningful improvement inprogression-free survival (PFS).This corresponds to a 34% reduction in the risk of progression or death in patients treated with Ninlaro.

Additionally, Takeda reported that real-world data from theUS MM-6studyrevealed thein-class transition from treatment with parenternal bortexomibto an oralNinlaro-based treatment resulted in an increase in overall response rate from 62% to 70% and an increase in complete response from 4% to 26%.These data suggest promising efficacy without impacting patients quality of life, with treatment taken by patients at home, the company said.

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European Hematology Association Meeting Kicks Off with Strong Data Presentations - BioSpace

The cultivation of grapes in Europe, whose acreage represents 3% of the total cultivable acreage, accounts for up to 65% of the pesticides used by EU growers, given the high incidence of powdery mildew and mildew in the productions. However, this percentage could be drastically reduced if the EU opted for the most advanced plant reproduction technologies, such as CRISPR, which would make it possible to obtain grape varieties resistant to both fungi.

Thus, research has been carried out in this field for several years in order to improve European grape varieties. In the case of Italy, in 2015, ten genetically edited grape varieties were registered in the National Variety Catalog, and in 2018, the first field harvests were carried out. Although still in the pre-commercial phase, the results so far have been positive in terms of resistance to diseases.

The researchers hope that the regulatory uncertainty of CRISPR technologies will be resolved (they are subject to the same regulations as transgenics, despite not being the same). They also hope that both producers and consumers will learn about the potential of these techniques to tackle the agro-food and environmental challenges that humanity is facing.

Source: agronewscastillayleon.com

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Genetic editing of grapes by CRISPR could reduce the use of pesticides in Europe - FreshPlaza.com

There is new hope for cat lovers who are allergic to their pets. Rather than desensitizing the human, researchers are working to eliminate the Feld1 protein, the primary allergen, from the cat, using CRISPR-Cas9 gene-editing technology.

"One of the benefits of CRISPR, compared to other methods of tackling this problem, is that you can permanently remove Feld1, compared with other techniques that only reduce the allergen," said Nicole Brackett, PhD, from Indoor Biotechnologies.

Previous attempts to remove the allergen have included feeding cats a specially formulated food that reduces Feld1 in the saliva, so less ends up on the dander when they lick themselves, as reported by Medscape Medical News.

"We hope to get to a point where we can offer an injection, or a series of injections, you would get at the vet, which would make the cat allergen-free," said Brackett, who presented the research in a poster at the European Academy of Allergy and Clinical Immunology 2020 Digital Congress.

When you're using this kind of technology, you are taking on a tremendous amount of responsibility.

About 10% of humans are allergic to cats, and we see the ones who are affected by their own cats, said Dean Mitchell, MD, an allergist and immunologist from Mitchell Medical Group in New York City.

"This research is interesting, but when you're using this kind of technology, you are taking on a tremendous amount of responsibility," he told Medscape Medical News.

"It's really an exciting technology, but I think it's scary, altering genes," he said. "You never know what you're going to change. Maybe we should use it to cure COVID first."

For their study, Brackett and her colleagues used discarded tissue samples from 50 spayed and neutered cats to collect genomic DNA from the Feld1 chains1 and 2.

The first goal was to see how similar genes were between cats, she explained. "We wanted to target a region in the gene that is well conserved something you would see in all cats not a random mutation."

The researchers were able to sequence a panel of 10 guide RNAs and use CRISPR Cas-9 to edit the genes. "We now have proof of principle in a cat cell line," Brackett told Medscape Medical News.

We still have a long way to go, but should have something we can test in a cat in a couple of years. But, she acknowledged, "we still don't know the role of the protein in the cat."

Feld1 expression differs from cat to cat, Brackett pointed out. "Some cats have an abundance and some have very little. The expression can vary, even within one cat."

Speculation on the function of Feld1 also varies. Because it's produced in the sebaceous gland, "it may serve as a way to coat, or protect, the skin. Or maybe it has something to do with chemical communication, maybe to communicate with other cats," she said. "But the fact that we see so much Feld1 variability with no obvious correlating behaviors makes us think it's not essential. One of the benefits of our study is we may figure that out."

Her lab is also looking at Feld1 expression in wild cats to determine its origin from an evolutionary standpoint. "We are curious to see how this allergen has evolved in different species of cats," she explained.

Cat allergies "rank number two in frequency and seriousness of allergies after food allergies, causing people to need to be on steroids or medications," Mitchell told Medscape Medical News. "Fifty percent of my immunotherapy practice is cat or dog allergy; it's a significant problem."

Young kids can't play at their friend's houses, relationships are affected, and families have to choose between their beloved cat and a healthy family member, he said.

Sublingual immunotherapy of Feld1 has been proven effective in clinical trials and is common in Europe, but "only about 100 allergists in the United States offer it," Mitchell said. "It's been a very underappreciated therapy, and I really don't know why; maybe because it's not patentable by a drug company."

Sublingual drops are not covered by insurance, and the therapy runs about $120 per month in the United States. "I've helped hundreds of patients with it," said Mitchell, and usually the therapy significantly improves patient quality of life.

The fact is, people make major life choices based on their cats, Mitchell explained.

One of his pollen-allergic patients who loved both her cat and her cat-allergic boyfriend told him that her boyfriend wouldn't sleep over. "Can you help him?" she asked.

"The first day I see him, he's wheezing and can't breathe." Mitchell recalled. He treated the boyfriend with sublingual Feld1 immunotherapy. "After 4 or 5 months, he could go over to her house using inhalers on weekends sometimes. A year later, they moved in together and got married. The cat even slept on the bed at the end.

Mitchell discharged the boyfriend after 3 years of treatment. He ran into him on a New York sidewalk a couple of years later and asked how he was doing. It turned out the couple had gotten divorced.

"That's too bad, I told him. But at least you don't have to deal with the cat anymore," Mitchell recalled.

"Oh no, I liked the cat too much, he stayed with me," his patient told him.

Mitchell laughed. "You just can't make this stuff up!"

Brackett works for Indoor Biotechnologies as a scientist. Mitchell has disclosed no relevant financial relationships.

European Academy of Allergy and Clinical Immunology (EAACI) 2020 Digital Congress

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More Than One Way to Fix Cat Allergy - Medscape

Join us on Friday, June 19, to find out how massively parallel microbial strain engineering may lead to the development of novel therapeutics to combat the most difficult antibiotic-resistant pathogens

Massively parallel microbial strain engineering on a CRISPR-based benchtop platform has enabled the exploration of the genetic dependencies of antibiotic function in unprecedented scale and detail. The ability to design and deliver precisely determined edits throughout the entire E. coli genome has resulted in an unparalleled opportunity to query a diverse population of strain variants for their growth responses to antibiotics from multiple different functional classes.

Find out more in this expert webinar as Dr. Dan Held, Director of Synthetic Biology, Microbial Applications Development, Inscripta, outlines how the knowledge garnered through the use of this strain engineering technology has significant potential to lead to the development of novel therapeutics against our most difficult antibiotic-resistant pathogens.

Key learning objectives

Who should attend?

This webinar will run on Friday, June 19, 2020, at:

Register to watch the full webinar here>>

SelectScience runs 3-4 webinars a month across various scientific topics,discover more of our upcoming webinars>>

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Expert Insight: Discover the genetic dependencies of antibiotic function - SelectScience