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New generation CRISPR technology could pave the way for therapeutics to treat inherited retina diseases, researchers report.

In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function, says Krzysztof Palczewski, chair and a professor in the Gavin Herbert Eye Institutes ophthalmology department at the University of California, Irvine School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, says first author Susie Suh, assistant specialist in the ophthalmology department.

We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, says co-first author Elliot Choi, also an assistant specialist in the ophthalmology department.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the researchers successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases.

Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast, and spatial and temporal frequency, says Palczewski.

These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases.

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status, and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy.

Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

The new paper appears in Nature Biomedical Engineering.

Support for the research came from the National Institutes of Health; the Research to Prevent Blindness Stein Innovation Award; Fight for Sight; the Eye and Tissue Bank Foundation (Finland); the Finnish Cultural Foundation; the Orion Research Foundation; the Helen Hay Whitney Foundation; US Department of Veterans Affairs; and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

Source: UC Irvine

The rest is here:
Therapy restores vision in mice with retina disease - Futurity: Research News

Here are some events that caught our eye, either because of their nod to current events or their unique nature. But really, you'll want to peruse the schedule yourself as the options are seemingly endless and you can customize your search to your interests and age range. Also, you must register (it's free!) to see full details of each event.

Events That Take You Outside

Interactive Bird Scavenger Hunt

If you've ever wanted to get into birding but find the Sibley guides overwhelming, this session is for you. Download the scavenger hunt card and join other aspiring ornithologists in observing local birds and their behavior.

Share the Night Sky

What do you get when you mix a radio show and a planetary show? This show features San Franciscos Urban Astronomer Paul Salazar and KPOO's DJ Marilynn. Tune in for a guided tour of the night sky in real time.

Events for Big Thinkers

Astronomy Talks: Quantum Mechanics vs General Relativity: Clash of the Titans

Join a discussion about unifying the two theories and creating "a single theory that describes the entire Universe." Doesn't get much bigger picture than that, now, does it?

CRISPR, Sickle Cell Disease, and Society: A VR Explainer and Ethics Discussion

Explore how CRISPR genome editing may be used to repair the sickle cell mutation. See a virtual reality tour of the human body, and a talk on health disparities in the U.S. and the ethics of CRISPR.

N ~ 1: Alone in the Milky Way

Dr. Pascal Lee, a planetary scientist who works at the Mars Institute, the SETI Institute and the Haughton-Mars Project at the NASA Ames Research Center, makes the case that "we might be it, in the vastness of our galaxy."

Events for the Foodies (or the Science-Reluctant)

The Science of Sundaes

You had me at ice cream scientist. Dr. Maya Warren will teach families how to make their own sweet treats at home and discuss her career as a "real-life ice cream scientist"!

The Science Behind Hand-Pulled Noodles

Members of the Stanford Polymer Collective will make a basic dough and explain how additives like oil, water and salt change the dough's mechanical properties. Participants are encouraged to follow along at home and decide which dough makes the best hand-pulled noodles.

Events for Climate Activists

Bay Area Youth Climate Activism Panel

Get inspired and learn from the young people in the Bay Area who are trying to save our ecosystems.

Wetland Protectors: Our First Line of Defense

Save the Bay will demonstrate why tidal marshlands and native plants are key to flood protection for bayside communities threatened by climate change.

Events at the Frontline of the Pandemic

Behind the Scenes at a COVID-19 Diagnostic Testing Center

You ever wonder what happens after the nose and throat swabs? We'll go inside a lab that's working with UCSF on COVID-19 testing. "Follow a sample from its arrival at the lab, through RNA extraction and amplification, to the end result to reveal if it is positive or negative for the virus."

Exploring Connections Between Cancer and COVID-19

Talk to researchers from the UCSF Cancer Cell Map Initiative (CCMI) and Quantitative Biology Institute Coronavirus Research Group (QBI-QCRG) about the "connections between the mechanisms of cancer and COVID-19."

Events at the Intersection of Race and Science

Cultural Tax: The Cost of Being the Only or the Few

Tyrone Poster, a principal investigator at Boston University, shares insights on the "silent burden" that often accompanies being a Black STEM student and professional.

A Conversation with the Creators of The Nocturnists

The Nocturnists is a medical storytelling community founded by two UCSF physicians. Their audio documentary series "Stories from a Pandemic" and Black Voices in Healthcare" provide unique first-person accounts from health care workers.

Events for the Creature-Curious

Catch that Critter!

Ever wonder who or what is going bump in the night in your backyard? Here's your chance! Learn how to set up a "wildlife monitoring system and hear about the critters caught on camera at the Cal State East Bay Concord Campus and cameras set up in the Diablo hills. This first event will be a how-two and the second event will give participants the chance to share what they discovered.

A Very Spine-Tingling Spider Screening with KQEDs Deep Look

Yes, this one is a shameless plug! The team behind KQED's own Deep Look video series will present three of their popular spider episodes. Deep Look producers and cinematographers will reveal how they captured the spiders behavior on camera and spider experts will be on hand to answer all your arachnid-related questions.

And there are hundreds more events on all variety of subjects: storytelling and COVID, composting, wildfires. Check out the BASF website to see them all.

Editor's Note: KQED is a media sponsor of the Bay Area Science Festival.

Follow this link:
Bay Area Science Festival 2020: From Black Holes to Sourdough, It's All Virtual - KQED

Indias first indigenously developed test for coronavirus disease (Covid-19) based on the genetic editing tool CRISPR/Cas-9 is likely to be available in the market by the end of October. Heres everything that you need to know about the test:

Why is the test named Feluda?

Feluda is the acronym for FNCAS9 Editor Linked Uniform Detection Assay but the test is named after a fictional private detective from West Bengal created by the renowned writer and filmmaker Satyajit Ray.

Who has developed the test?

The test was developed by a research team led by Debojyoti Chakraborty and Souvik Maiti of the Council of Scientific and Industrial Research (CSIR) and researchers from the Institute of Genomics and Integrative Biology. It will be marketed by Tata Sons and has been approved by the Drug Controller General of India last month.

What is the effectiveness of the test?

It has a sensitivity of 96 per cent and specificity of 98 per cent which means that the test can detect positive and negative cases both up to 96 or 98 per cent of the time. The test matches the accuracy levels of RT- PCR tests which are widely being used for Covid- 19 diagnosis.

How does the test work?

With quicker result time and less expensive equipment, the test uses indigenously developed CRISPR gene-editing technology. It is the worlds first diagnostic test that uses specially adapted Cas9 protein to successfully detect the virus. It requires a nasal swab to be collected and sent to a lab. The Cas9 protein is barcoded to interact with the SARS-CoV2 sequence in the patients genetic material. The Cas9-SARS-CoV2 complex is then put on the paper strip, where using two lines (one control, one test), the test determines whether the sample was infected with Covid-19.

What is the CRISPR technology?

CRISPR, short form for Clustered Regularly Interspaced Short Palindromic Repeats, is a genome editing technology used in correcting genetic defects and treating and preventing the spread of diseases. The CRISPR technology that won the Nobel Prize for chemistry this year can detect specific sequences of DNA within a gene using an enzyme functioning as molecular scissors to snip it. It also allows researchers to easily alter DNA sequences and modify gene function.

What is the cost of the test?

The Feluda test is likely to cost about Rs 500 but the final cost will only be known once the test is commercially available. In contrast, the RT-PCR test now costs anywhere between Rs 1,600 to Rs 2,000.

Read the rest here:
What is the Feluda test that will be commercially available by October 31? - Hindustan Times

This report also researches and evaluates the impact of Covid-19 outbreak on the CRISPR & Cas Genes industry, involving potential opportunity and challenges, drivers and risks. We present the impact assessment of Covid-19 effects on CRISPR & Cas Genes and market growth forecast based on different scenario (optimistic, pessimistic, very optimistic, most likely etc.).

Global CRISPR & Cas Genes Market Overview:

The research report, titled [Global CRISPR & Cas Genes Market 2020 by Company, Regions, Type and Application, Forecast to 2025], presents a detailed analysis of the drivers and restraints impacting the overall market. Analysts have studied the key trends defining the trajectory of the market. The research report also includes an assessment of the achievements made by the players in the global CRISPR & Cas Genes market so far. It also notes the key trends in the market that are likely to be lucrative. The research report aims to provide an unbiased and a comprehensive outlook of the global CRISPR & Cas Genes market to the readers.

Get PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) @ https://www.marketresearchhub.com/enquiry.php?type=S&repid=2696251&source=atm

Global CRISPR & Cas Genes Market: Segmentation

For clearer understanding of the global CRISPR & Cas Genes market, analysts have segmented the market. The segmentation has been done on the basis of application, technology, and users. Each segment has been further explained with the help of graphs figures. This breakdown of the market gives the readers an objective view of the global CRISPR & Cas Genes market, which is essential to make sound investments.

The major players profiled in this report include:CRISPR TherapeuticsAstraZenecaAddgeneCaribou Biosciences, Inc.CellectisEditas Medicine, Inc.EgenesisF. Hoffmann-La Roche Ltd.Horizon Discovery Group PlcGenscripDanaher CorporationIntellia Therapeutics, Inc.LonzaMerck KGaANew England BioLabsTakara Bio, Inc.

To understand the changing political scenario, analysts have regionally segmented the market. This gives an overview of the political and socio-economic status of the regions that is expected to impact the market dynamic.

Global CRISPR & Cas Genes Market: Research Methodology

To begin with, the analysis has been put together using primary and secondary research methodologies. The information has been authenticated by market expert through valuable commentary. Research analysts have also conducted exhaustive interviews with market-relevant questions to collate this research report.

Do You Have Any Query Or Specific Requirement? Ask to Our Industry [emailprotected] https://www.marketresearchhub.com/enquiry.php?type=E&repid=2696251&source=atm

Global CRISPR & Cas Genes Market: Competitive Rivalry

The research report also studied the key players operating in the global CRISPR & Cas Genes market. It has evaluated and elucidated the research and development statuses of these companies, their financial outlooks, and their expansion plans for the forecast period. In addition, the research report also includes the list of strategic initiatives that clearly explain the achievements of the companies in the recent past.

The end users/applications and product categories analysis:On the basis of product, this report displays the sales volume, revenue (Million USD), product price, market share and growth rate of each type, primarily split into-General Type

On the basis on the end users/applications, this report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate of CRISPR & Cas Genes for each application, including-Biomedical

You can Buy This Report from Here @ https://www.marketresearchhub.com/checkout?rep_id=2696251&licType=S&source=atm

Strategic Points Covered in TOC:

Chapter 1: Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global CRISPR & Cas Genes market

Chapter 2: Evaluating the leading manufacturers of the global CRISPR & Cas Genes market which consists of its revenue, sales, and price of the products

Chapter 3: Displaying the competitive nature among key manufacturers, with market share, revenue, and sales

Chapter 4: Presenting global CRISPR & Cas Genes market by regions, market share and with revenue and sales for the projected period

Chapter 5, 6, 7, 8 and 9: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions

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CRISPR & Cas Genes Market: Analysis and In-depth study on market Size Trends, Emerging Growth Factors and Regional Forecast to 2025 - Eurowire

MarketsandResearch.biz has released a recent industry research report titled Global CRISPR and Cas Genes Market 2020 by Company, Regions, Type and Application, Forecast to 2025 that spotlights on the study of past, present, and future look of the industry. The study conducted is inclusive of the industry trends and competitive and regional analysis covering the period 2020-2025. The report provides an actual industry viewpoint on market trends, dynamics for market growth rate, trading, growth rate, and revenue, in terms of demand and supply, cost structure, barriers, and challenges, product type, key market players, regions, and applications. Studying the market in terms of growth and expansion, the report covers the crucial factors influencing the global CRISPR and Cas Genes market.

Competitive Landscape:

The report analysts have identified direct or indirect market competitors, as well as comprehend their mission, vision, values, niche market, strengths, and weaknesses. The report provides Porters five forces including the threat of substitute products or services, established rivals, new entrants, and two others such as the bargaining power of suppliers and customers. Prominent players joined with their market share are highlighted in the report. The well-established players in the global CRISPR and Cas Genes market are: Synthego, OriGene Technologies, Inc., Addgene, Thermo Fisher Scientific, Inc., Transposagen Biopharmaceuticals, Inc., GenScript, Horizon Discovery Group Co., Integrated DNA Technologies, Inc., Merck, New England Biolabs, Cellecta, Inc., Agilent Technologies, Applied StemCell, Inc.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/107687

NOTE: Our report highlights the major issues and hazards that companies might come across due to the unprecedented outbreak of COVID-19.

Geographical provincial information will help you in focusing on all the best-performing locales. The regions are extensively analyzed with respect to every parameter of the geographies in question, comprising, North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

In market segmentation by types, the report covers: Vector-based Cas, DNA-free Cas

In market segmentation by applications, the report covers the following uses: Genome Engineering, Disease Models, Others

The report evaluates production, consumption, and product segmentation highlights the current trend in the global market, and projects the revenue and potential developments of key players. Further, the report has analyzed the market concerning the regional landscape which incorporates extensive details about the types and application spectrums of this business. The research report tracks competitive growths such as joint ventures, tactical alliances, mergers and achievements, new product developments, and research and developments in the global CRISPR and Cas Genes market.

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/107687/global-crispr-and-cas-genes-market-2020-by-company-regions-type-and-application-forecast-to-2025

Consumer Behavior Analysis:

Furthermore, the report analyzes the behavior of the CRISPR and Cas Genes consumers in the marketplace and looks at motives for those behavioral trends. Later, personal, and social consumer behavior is studied through focus groups, surveys, and tracking sales history. Our consumer behavior study helps businesses to understand consumers value. Not all consumers value the same benefits, so its important for businesses to segment their consumer base. Using the latest technology and analysis on the demand-side, key players are getting into consumer behavior and their changing preferences.

Customization of the Report:

This report can be customized to meet the clients requirements. Please connect with our sales team (sales@marketsandresearch.biz), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

Contact UsMark StoneHead of Business DevelopmentPhone: +1-201-465-4211Email: sales@marketsandresearch.bizWeb: http://www.marketsandresearch.biz

Read more:
Global CRISPR and Cas Genes Market 2020 Trending Technologies, Developments, Key Players and End-use Industry to 2025 - Illadel Graff Supply

The latest adventure in food enhancement is CRISPR (e.g., clustered regularly interspaced short palindromic repeats/Cas9) gene-editing technology. It potentially has many major implications for enhanced global agriculture and much needed improvements in food security. CRISPR and gene editing tools simultaneously represents an extensive legal and regulatory challenge and additionally, a monumental scientific opportunity for the global food industry. With the development of genome editing technologies, the possibility of directly targeting and subsequently modifying genomic sequences in plants is intriguing. Genome editing can extend our ability to develop an extraordinary potential in applied biotechnology and its effects on increased world food production.

An important concept to the understanding of CRISPR/Cas came from scientific observations that the prokaryote repeat cluster (a family of DNA sequences found in the genomes ofprokaryoticorganisms such as bacteria and archaea) was incorporated into a set of homologous genes (having the same relation/structure or relative position) that make up CRISPR-associated systems orCasgenes.

TheCasproteins show nuclease and helicase motifs (a linkage that is found in a great many other DNA processing enzymes) which suggests an important role of the CRISPR loci. The development of programmable nucleases (e.g., clustered regularly interspaced short palindromic repeat (CRISPR)Cas-associated nucleases, zinc-finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs)) has expedited the ability to improve the field of gene editing and subsequently potentially improve food production. CRISPR/Cas gene editing technology can potentially increase plant/crop yields and quality, plant drought resistance, herbicide and insecticide resistance, improved food safety and security, enhance the removal of antibiotic resistance (AMR), improve product shelf life and it can potentially accelerate the process of plant domestication.

Read more at Michigan State University (Scott Haskell)

Read more:
CRISPR and our food supply: What's next in feeding the world? - hortidaily.com

We think that its fair to say that the possibility of finding fantastic multi-year winners is what motivates many investors. Mistakes are inevitable, but a single top stock pick can cover any losses, and so much more. Take, for example, the CRISPR Therapeutics AG (NASDAQ:CRSP) share price, which skyrocketed 424% over three years. In more good news, the share price has risen 1.7% in thirty days.

View our latest analysis for CRISPR Therapeutics

We dont think that CRISPR Therapeutics modest trailing twelve month profit has the markets full attention at the moment. We think revenue is probably a better guide. As a general rule, we think this kind of company is more comparable to loss-making stocks, since the actual profit is so low. For shareholders to have confidence a company will grow profits significantly, it must grow revenue.

Over the last three years CRISPR Therapeutics has grown its revenue at 101% annually. Thats well above most pre-profit companies. And its not just the revenue that is taking off. The share price is up 74% per year in that time. Despite the strong run, top performers like CRISPR Therapeutics have been known to go on winning for decades. So wed recommend you take a closer look at this one, or even put it on your watchlist.

You can see below how earnings and revenue have changed over time (discover the exact values by clicking on the image).

CRISPR Therapeutics is well known by investors, and plenty of clever analysts have tried to predict the future profit levels. So it makes a lot of sense to check out what analysts think CRISPR Therapeutics will earn in the future (free analyst consensus estimates)

Pleasingly, CRISPR Therapeutics total shareholder return last year was 163%. So this years TSR was actually better than the three-year TSR (annualized) of 74%. The improving returns to shareholders suggests the stock is becoming more popular with time. 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. For example, weve discovered 2 warning signs for CRISPR Therapeutics that you should be aware of before investing here.

But note: CRISPR Therapeutics may not be the best stock to buy. So take a peek at this free list of interesting companies with past earnings growth (and further growth forecast).

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

PromotedIf youre looking to trade CRISPR Therapeutics, open an account with the lowest-cost* platform trusted by professionals, Interactive Brokers. Their clients from over 200 countries and territories trade stocks, options, futures, forex, bonds and funds worldwide from a single integrated account.

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. *Interactive Brokers Rated Lowest Cost Broker by StockBrokers.com Annual Online Review 2020

Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team@simplywallst.com.

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Introducing CRISPR Therapeutics (NASDAQ:CRSP), The Stock That Soared 424% In The Last Three Years - Simply Wall St

LOS ANGELES, United States:The report titledGlobal CRISPR And CRISPR-Associated (Cas) Genes Marketis one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global CRISPR And CRISPR-Associated (Cas) Genes market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global CRISPR And CRISPR-Associated (Cas) Genes market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global CRISPR And CRISPR-Associated (Cas) Genes market is carefully analyzed and researched about by the market analysts.The market analysts and researchers have done extensive analysis of the global CRISPR And CRISPR-Associated (Cas) Genes market with the help of research methodologies such as PESTLE and Porters Five Forces analysis. They have provided accurate and reliable market data and useful recommendations with an aim to help the players gain an insight into the overall present and future market scenario. The CRISPR And CRISPR-Associated (Cas) Genes report comprises in-depth study of the potential segments including product type, application, and end user and their contribution to the overall market size.

In addition, market revenues based on region and country are provided in the CRISPR And CRISPR-Associated (Cas) Genes report. The authors of the report have also shed light on the common business tactics adopted by players. The leading players of the global CRISPR And CRISPR-Associated (Cas) Genes market and their complete profiles are included in the report. Besides that, investment opportunities, recommendations, and trends that are trending at present in the global CRISPR And CRISPR-Associated (Cas) Genes market are mapped by the report. With the help of this report, the key players of the global CRISPR And CRISPR-Associated (Cas) Genes market will be able to make sound decisions and plan their strategies accordingly to stay ahead of the curve.

Competitive landscape is a critical aspect every key player needs to be familiar with. The report throws light on the competitive scenario of the global CRISPR And CRISPR-Associated (Cas) Genes market to know the competition at both the domestic and global levels. Market experts have also offered the outline of every leading player of the global CRISPR And CRISPR-Associated (Cas) Genes market, considering the key aspects such as areas of operation, production, and product portfolio. Additionally, companies in the report are studied based on the key factors such as company size, market share, market growth, revenue, production volume, and profits.

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

Get Full PDF Sample Copy of Report: (Including Full TOC, List of Tables & Figures, Chart)

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Segmental Analysis

The report has classified the globalCRISPR And CRISPR-Associated (Cas) Genesindustry 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 theCRISPR And CRISPR-Associated (Cas) Genes manufacturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overallCRISPR And CRISPR-Associated (Cas) Genesindustry.

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

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

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

Biotechnology Companies, Pharmaceutical Companies, Academic Institutes, Research and Development Institutes

The CRISPR And CRISPR-Associated (Cas) Genes Market report has been segregated based on distinct categories, such as product type, application, end user, and region. Each and every segment is evaluated on the basis of CAGR, share, and growth potential. In the regional analysis, the report highlights the prospective region, which is estimated to generate opportunities in the global CRISPR And CRISPR-Associated (Cas) Genes market in the forthcoming years. This segmental analysis will surely turn out to be a useful tool for the readers, stakeholders, and market participants to get a complete picture of the global CRISPR And CRISPR-Associated (Cas) Genes market and its potential to grow in the years to come.

Key questions answered in the report:

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Table of Contents:

Table of Contents 1 Report Overview1.1 Research Scope1.2 Top CRISPR And CRISPR-Associated (Cas) Genes Manufacturers Covered: Ranking by Revenue1.3 Market Segment by Type

1.3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size by Type: 2015 VS 2020 VS 2026 (US$ Million)

1.3.2 Genome Editing

1.3.3 Genetic engineering

1.3.4 gRNA Database/Gene Librar

1.3.5 CRISPR Plasmid

1.3.6 Human Stem Cells

1.3.7 Genetically Modified Organisms/Crops

1.3.8 Cell Line Engineering1.4 Market Segment by Application

1.4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Application: 2015 VS 2020 VS 2026

1.4.2 Biotechnology Companies

1.4.3 Pharmaceutical Companies

1.4.4 Academic Institutes

1.4.5 Research and Development Institutes1.5 Study Objectives1.6 Years Considered 2 Global Market Perspective2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue (2015-2026)

2.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue (2015-2026)

2.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales (2015-2026)2.2 CRISPR And CRISPR-Associated (Cas) Genes Market Size across Key Geographies Worldwide: 2015 VS 2020 VS 2026

2.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales by Regions (2015-2020)

2.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue by Regions (2015-2020)2.3 Global Top CRISPR And CRISPR-Associated (Cas) Genes Regions (Countries) Ranking by Market Size2.4 CRISPR And CRISPR-Associated (Cas) Genes Industry Trends

2.4.1 CRISPR And CRISPR-Associated (Cas) Genes Market Top Trends

2.4.2 Market Drivers

2.4.3 CRISPR And CRISPR-Associated (Cas) Genes Market Challenges 2.4.4 Porters Five Forces Analysis

2.4.5 Primary Interviews with Key CRISPR And CRISPR-Associated (Cas) Genes Players: Views for Future 3 Competitive Landscape by Manufacturers3.1 Global Top CRISPR And CRISPR-Associated (Cas) Genes Manufacturers by Sales (2015-2020)

3.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales by Manufacturers (2015-2020)

3.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Market Share by Manufacturers (2015-2020)

3.1.3 Global 5 and 10 Largest Manufacturers by CRISPR And CRISPR-Associated (Cas) Genes Sales in 20193.2 Global Top Manufacturers CRISPR And CRISPR-Associated (Cas) Genes by Revenue

3.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue by Manufacturers (2015-2020)

3.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Share by Manufacturers (2015-2020)

3.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Market Concentration Ratio (CR5 and HHI)3.3 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in CRISPR And CRISPR-Associated (Cas) Genes as of 2019)3.4 Global CRISPR And CRISPR-Associated (Cas) Genes Average Selling Price (ASP) by Manufacturers3.5 Key Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Plants/Factories Distribution and Area Served3.6 Date of Key Manufacturers Enter into CRISPR And CRISPR-Associated (Cas) Genes Market3.7 Key Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Product Offered 3.8 Mergers & Acquisitions, Expansion Plans 4 Market Size by Type4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Historic Market Review by Type (2015-2020)

4.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Market Share by Type (2015-2020)

4.1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Type (2015-2020)

4.1.4 CRISPR And CRISPR-Associated (Cas) Genes Price by Type (2015-2020)4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Estimates and Forecasts by Type (2021-2026)

4.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast by Type (2021-2026)

4.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast by Type (2021-2026)

4.2.4 CRISPR And CRISPR-Associated (Cas) Genes Price Forecast by Type (2021-2026) 5 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size by Application5.1 Global CRISPR And CRISPR-Associated (Cas) Genes Historic Market Review by Application (2015-2020)

5.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Market Share by Application (2015-2020)

5.1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Application (2015-2020)

5.1.4 CRISPR And CRISPR-Associated (Cas) Genes Price by Application (2015-2020)5.2 Global CRISPR And CRISPR-Associated (Cas) Genes Market Estimates and Forecasts by Application (2021-2026)

5.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast by Application (2021-2026)

5.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast by Application (2021-2026)

5.2.4 CRISPR And CRISPR-Associated (Cas) Genes Price Forecast by Application (2021-2026) 6 North America6.1 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company6.2 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type6.3 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application6.4 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

6.4.1 North America CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

6.4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

6.4.3 U.S.

6.4.4 Canada 7 Europe7.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company7.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type7.3 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application7.4 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

7.4.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

7.4.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

7.4.3 Germany

7.4.4 France

7.4.5 U.K.

7.4.6 Italy

7.4.7 Russia 8 Asia Pacific8.1 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company8.2 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type8.3 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application8.4 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Regions

8.4.1 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Sales by Regions

8.4.2 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Revenue by Regions

8.4.3 China

8.4.4 Japan

8.4.5 South Korea

8.4.6 India

8.4.7 Australia

8.4.8 Taiwan

8.4.9 Indonesia

8.4.10 Thailand

8.4.11 Malaysia

8.4.12 Philippines

8.4.13 Vietnam 9 Latin America9.1 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company9.2 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type9.3 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application9.4 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

9.4.1 Latin America CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

9.4.2 Latin America CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

9.4.3 Mexico

9.4.4 Brazil

9.4.5 Argentina 10 Middle East and Africa10.1 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type10.2 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application10.3 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

10.3.1 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

10.3.2 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

10.3.3 Turkey

10.3.4 Saudi Arabia

10.3.5 U.A.E 11 Company Profiles11.1 Caribou Biosciences

11.1.1 Caribou Biosciences Corporation Information

11.1.2 Caribou Biosciences Business Overview and Total Revenue (2019 VS 2018)

11.1.3 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)

11.1.4 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Products and Services

11.1.5 Caribou Biosciences SWOT Analysis

11.1.6 Caribou Biosciences Recent Developments11.2 Addgene

11.2.1 Addgene Corporation Information

11.2.2 Addgene Business Overview and Total Revenue (2019 VS 2018)

11.2.3 Addgene CRISPR And CRISPR-Associated (Cas) Genes Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)

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CRISPR And CRISPR-Associated (Cas) Genes Market Break Down by Top Companies, Countries, Applications, Challenges, Opportunities and Forecast by...

CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) have announced that the European Medicines Agency has granted their experimental, autologous, ex vivo CRISPR/Cas9 gene-edited therapy, CTX001 Priority Medicines designation. The therapy has been granted PRIME designation for severe sickle cell disease treatment.

The PRIME designation is a regulatory mechanism that offers early and proactive support to promising medicines developers to enhance development plans. Also, it accelerates the evaluation of medicines under development to reach patients as soon as possible. PRIMEs objective is to ensure patients benefit fasters from new proprietary therapies that have shown the potential of addressing a significant unmet medical need. The companies received the PRIME designation based on data from their on-going phase 1/2 study of CTX001 in treating severe sickle cell disease patients.

CTX001 is currently being investigated to treat patients with severe SCD or transfusion-dependent beta-thalassemia, whereby hematopoietic stem cells are purposed to produce high fetal hemoglobin levels in red cells. HbF is an oxygen-carrying hemoglobin form present naturally at birth, which later switches to adult hemoglobin form. HbF elevation by CTX001 can potentially alleviate transfusion requirements for TDT patients and minimize painful and devastating sickle crises in patients with SCD.

So far, CTX001 has received Regenerati9ve Medicine Advanced Therapy (RMAT), Orphan Drug Designation, and Fast Track designation from the FDA. Also, CTX001 has Orphan Drug Designation from the European Commission for SCD and TDT.

CRISPR Therapeutics and Vertex are developing CTX001 under a co-development and co-commercialization agreement. So far, CTX001 is the most progressive gene editing therapy under development for SCD and TDT. The companies entered a strategic collaboration agreement in 2015 intending to use CRISPR/Cas9 to develop and discover novel therapies for the treatment of underlying genetic causes of diseases. CTX001 is the first joint treatment to emerge from the collaboration, and all development costs and profits will be shared.

Read this article:
CRISPR Therapeutics (NASDAQ:CRSP) and Vertex (NASDAQ:VRTX) Receive Priority Medicines (PRIME) Designation From EMA For CTX001 In SCD - BP Journal

Emmanuelle Charpentier and Jennifer Doudna have been given the 2020 Nobel Prize in Chemistry for their discovery and development of CRISPR-Cas9 genome editing.

The Royal Swedish Academy of Sciences has decided to award the Nobel Prize in Chemistry 2020 to Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens, Germany, and Jennifer Doudna of the University of California, Berkeley, US. The Nobel Prize is for the development of CRISPR-Cas9, a method for genome editing.

According to the Royal Swedish Academy of Sciences, Emmanuelle Charpentier and Jennifer Doudna discovered the CRISPR-Cas9 genetic scissors. Using these, researchers can change the DNA of animals, plants and microorganisms with extremely high precision. This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may aid in curing inherited diseases.

Also, using the CRISPR-Cas9 genetic scissors, it is now possible to change DNA over the course of a few weeks. This used to be time-consuming, difficult and sometimes impossible work.

There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments, said Claes Gustafsson, chair of the Nobel Committee for Chemistry.

The discovery of CRISPR-Cas9 was made during Emmanuelle Charpentiers studies of Streptococcus pyogenes, one of the bacteria that cause the most harm to humanity. She discovered a previously unknown molecule, tracrRNA. Her work showed that tracrRNA is part of bacterias ancient immune system, CRISPR-Cas, that disarms viruses by cleaving their DNA.Charpentier published her discovery in 2011. The same year, she initiated a collaboration with Jennifer Doudna, an experienced biochemist with vast knowledge of RNA. Together, they succeeded in recreating the bacterias genetic scissors in a test tube and simplifying the scissors molecular components so they were easier to use.

In another experiment, they then reprogrammed the genetic scissors. In their natural form, the scissors recognise DNA from viruses, but Charpentier and Doudna proved that they could be controlled so that they can cut any DNA molecule at a predetermined site.

The academy highlights that since Charpentier and Doudna discovered the CRISPR-Cas9 genetic scissors in 2012 their use has exploded. This tool has contributed to many important discoveries in basic research and clinical trials of new cancer therapies are underway.

Continued here:
Nobel Prize in Chemistry awarded to discoverers of CRISPR-Cas9 - Drug Target Review

Graphic Source: CRISPR Therapeutics, Inc.

CRISPR Therapeutics (NASDAQ:CRSP) is a gene-editing company focused on the development and versatile application of CRISPR/Cas9 therapeutics, a special brand of therapeutics used for precision genome editing by applying a viral defense mechanism from bacteria to regulate, disrupt, or correct genes related to key diseases. CRSP is currently targeting disease areas, including hemoglobinopathies, oncology, and regenerative medicines.

Founded in 2013 in Switzerland, CRSP has since grown to over 304 employees producing relatively inconsistent revenues ranging from $3M in 2018 to $290M in 2019 with expectations for 2020 at $6.7M. Their lead candidate is CTX001, an investigational autologous gene-edited hematopoietic stem cell therapy developed in partnership with Vertex Pharmaceuticals (NASDAQ:VRTX) for treating transfusion-dependent beta-thalassemia ("TDT") and severe sickle cell disease ("SCD").

Products: CRSP's pipeline consists of 9 therapeutics: 4 in the clinical phase and 5 in the research phase. Of the 4 clinical phase therapeutics, the first targets TDT and SCD (mentioned above: CTX001), while the 3 others fall into immuno-oncology covering: CD19+ malignancies (Product: CTX110), multiple myeloma (CTX120) and solid tumors and hematologic malignancies (CTX130). All immuno-oncology therapeutics are allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapies wholly owned by CRISPR Therapeutics with data updates typically every 6 months.

Customers/market: For CRSP's clinical phase pipeline, the total estimated 2022 global market potential is $220B with an average market size for each disease of $36.7B growing at an average 15.2% CAGR (median market: $13.3B | CAGR 10.9%). The largest market is Solid Tumors, at a 2022 estimated size of $145B (8.1% CAGR), and the highest CAGR market CAR T/CD19+ market at a 34.5% CAGR. For CTX001, the lead candidate, the target market can be broken down into the TDT market at very roughly $1.8B with a 10.8% CAGR and the SCD market at $4.1B with an 11% CAGR by 2022.

Management: Many are now familiar with co-founder Dr. Emmanuelle Charpentier, who in 2020 under much-debated circumstances co-received the Nobel Prize in Chemistry for her work with developing the CRISPR/Cas9 genetic scissors, the foundation of CRSP's therapeutics today. In addition to her role now as Scientific Advisory Board Member, CRSP has a variety of other accomplished leaders.

CEO: Dr. Samarth Kulkarni has served as CEO (covering long-term strategy) since December of 2017 when he was promoted from President and Chief Business Officer. Before CRSP, he was a Partner at McKinsey & Company (MCK) co-leading the biotech practice. His specialties are in strategy and operations, and he has a Ph.D. in Bioengineering and Nanotechnology.

Share Price Change under his leadership (Dec. 2017 - Present): 499% | CAGR: ca 71%.

President/Chairman: Dr. Rodger Novak, a co-founder with Charpentier and Shaun Foy in 2013, has served as CEO until 2017 and since, as President (day-to-day operations) and Chairman. Rodger is an experienced biotech/pharma executive having served in leadership positions (primarily covering infectious diseases and related) at Sanofi and Novartis. He co-also founded Nabriva Therapeutics (NASDAQ:NBRV). His specialty is in translating scientific technologies into pharmaceutical products. Before all else, he was a professor of Microbiology at the Vienna BioCenter (Austria).

Other management updates:

Strategy: In terms of strategy, CRSP stated they intend to use their scientific expertise, together with their unique platform to bring about a new class of highly active and potentially curative therapies for specialty patients to which biopharmaceutical approaches have limited exposure. CRSP has been investing heavily in its long-term platform.

Additionally, CRSP seems to be taking the lead in most of its partnerships, particularly the ViaCyte partnership evidenced by the structure of their collaboration agreements and payments due to ViaCyte. Investors also saw last year CRSP buying Bayer's (OTCPK:BAYZF) control of Casebia and expanding their internal clinical pipeline with internal funds (hence the cash stockpile). CRSP is acting as the all-or-nothing winner. It is a unique approach that expresses internal confidence in their technology and financial capabilities.

Financial position: CRSP received new upfront payments from Vertex in 2019 boosting revenues to $290M, which are not expected to be repeat. The estimated decline in 2020 brings revenue to $6.7M (-98%). 2019 was the only year in the company's history to achieve net income ($67M), whereas the 3-year average net loss is -$56M. For 1H 2020, net losses are already -$149M. CRSP operates with a strong cash cushion of $945M at 1H 2020, enough to cover the -$42M 3-year average CFO+CAPEX expenditures for 22+ years. Total debt as of 1H 2020 is a manageable $50M ($40M in capital leases). Accounts payable are a small $13M.

Investment thesis: Although most of CRSP's products are years away from revenue-generating outside of milestone payments, the constant updates from clinical trials offer a compelling progress report for the long position. Investors must be willing to pay for the premium that exists currently, but with CRISPR being in the public spotlight, investor interest may increase. The real question remains if they are truly the most advanced CRISPR position, though clearly not discounted. Operational strategy is a key selling point with the new McKinsey-inherited leadership, but science is still the core of any biotech investment. Below will be an expounded analysis of what their therapeutics are, but it seems to be that CRSP has a compelling niche for the long-term investor, given their 7+ years of experience with this new biotechnology. Therefore, with the Vertex partnership, enough cash to keep steady progress, and stable operational-based leadership, CRSP is a "buy".

CRSP's pipeline consists of 9 therapeutics: 4 clinical phases and 5 in the research phase. CRSP is supported by 1 strong partnership and 1 weaker partnership including:

Vertex partnership (est. 2015) for clinical analysis of TDT and SCD with a research phase target of cystic fibrosis ("CF") is based around co-developing CTX001 (since Dec. 2017). The partnership did expand in June 2019 for Duchenne muscular dystrophy ("DMD") and myotonic dystrophy type 1, which adds further upside potential for CRSP.

In 2015, CRSP received a $75M upfront payment and in 2017 received $7M and, thereafter, a low-seven-digit milestone payment for second patient dosing related to TDT and SCD. Looking forward, CRSP is eligible for up to $420M for further milestones and product-sales royalties.

In 2019, after the new collaboration agreement was established for DMD and myotonic dystrophy type 1 ("DM1"), CRSP has received an upfront payment of $175M with eligible milestone payments up to $825M. Tiered royalties on product sales are also available. The DMD program makes Vertex responsible for R&D, manufacturing, and commercialization. For DM1, CRSP will cover RNA research with Vertex responsible for all other costs. After DM1 IND filing, CRSP retains the option to co-develop/co-commercialize all DM1 products but must forgo milestone payments/royalties and cover 50% of R&D costs incurred by Vertex. Similar amendments were made to the 2015 agreement. In Oct. 2019, Vertex accepted the right to exclusively license the three remaining options granted under their 2015 agreement, resulting in CRSP receiving a $30M 4Q 2019 payment. CRSP also received a milestone $25M payment in 2Q 2020. At 1H 2020, CRSP had $11.8M of non-current deferred revenue related to Vertex.

ViaCyte partnership (est. Sep 2018) for diabetes through gene-edited allogeneic stem cell therapies. A key aspect of this partnership is ViaCytes stem cell capabilities and CRSP's gene-editing capabilities to enable beta-cell replacement without the need for immune system suppression. After successful completion of the studies proving verification of developing the immune-evasive stem cell line, CRSP and ViaCyte will jointly be responsible for further development and commercialization, globally. The partnership entitled ViaCyte to $16.2M in payments for participating which CRSP recognized as $15M in R&D expense and $1.2M in other expenses. The expected partnership term is in force for 5.5+ years and obligates both parties to jointly develop the research plan with each party responsible for their research costs.

Bayer partnership (JV est. 2015) was terminated in 4Q 2019 which included Casebia Therapeutics and focused on treating genetic causes related to bleeding disorders and autoimmune diseases, amongst others. CRSP retained full-ownership of Casebia and Bayer retained the right to co-develop two therapeutics related to autoimmune disorders, eye disorders, and hemophilia A disorders with exclusive licenses; termed the "2019 Option Agreements".

The clinical phase therapeutics of CRSP will be outlined below.

Graphic Source: CRISPR Therapeutics Investor Presentation (Sep 2020)

Therapeutic 1: CTX001 is CRSP's lead candidate targeting TDT and severe SCD through an investigational autologous gene-edited hematopoietic stem cell therapy. It is being co-developed by Vertex Pharmaceuticals.

TDT: CTX001 is currently in a Phase 1/2 open-label clinical trial (CLIMB THAL-111) for transfusion-dependent -thalassemia. The study aims to assess safety and efficacy for single dosages of CTX001 in a 12-35-year-old population with TDT. In 4Q 2019, Vertex & CRSP expanded the TDT patient population to include beta 0/beta 0 subtypes with the first two severe patients indicating successful dosing and engraftment. The study is designed for up 45 patients and aims to follow them for a duration of two years post-infusion with 6-month investor updates. CTX001 has received the Regenerative Medicine Advanced Therapy ("RMAT"), fast-track, and orphan drug designations (+European Commission) by the FDA for treating TDT. Preliminary clinical data was released in 4Q 2019, and in June 2020, their 15 months of follow-up data were also released in the ongoing study.

SCD: CTX001 is also currently in a Phase 1/2 open-label clinical trial (CLIMB SCD-121) for severe sickle cell disease testing safety and efficacy for single dosages of CTX001 in an older patient population than TDT (18-35). Similar to CLIMB THAL-111, the first two patients were treated sequentially then expanded for up to 45 concurrent patients for a 2-year following. CTX001 for SCD has also received the same designations as for TDT with the same data publication dates but in June releasing only 9 months of follow-up data.

Competition Notes:

Therapeutic 2: CTX110 is CRSP's lead candidate amongst their wholly-owned CAR-T therapies, which is a gene-edited allogeneic CAR-T therapy targeting CD19 in CD19+ malignancies cases. It's currently in a Phase 1 study focused on safety and efficacy ("S&E") in the treatment of relapsed/refractory B-cell malignancies. The study is designed for up to 131 patients on a multi-dose level.

Therapeutic 3: CTX120 is another gene-edited allogeneic CAR-T therapy but targeting B-cell maturation antigen. CTX120 is in a Phase 1 S&E study for treating relapsed/refractory("R&R") multiple myeloma. It is also a multi-center open-label trial but designed for up to 88 patients also on a multi-dose level investigation.

Therapeutic 4: CTX130 is CRSP's other CAR-T therapy with a target of CD70, the antigen expressed on solid-tumors and hematologic malignancies (study's target). The treatment is developed around solid-tumors (e.g. renal cell carcinoma) and T/B-cell hematologic malignancies. CRSP is currently running two independent Phase 1 S&E studies for CTX130 treating R&R renal cell carcinoma and other types of lymphoma. The first Phase 1 study, focused on R&R renal cell carcinoma, is a multi-center open-label investigation with an enrollment of up to 95 patients on a multi-dose level, and the second study for various lymphomas is designed for up to 46 patients.

For further analysis of the science and clinical trial updates, see clinical.gov studies linked above, the September 2020 Investor presentation, or the Chardan Conference Call (Sep 2020).

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Revenue/cash flow: Financially, CRSP is far from stable on any operating metric (other than cash resources), but survived COVID with 80-90% productivity. Revenue is earned from collaboration agreements and their associated milestones. This swayed investors in 2019 when $289M (99.6% of 2019 Revenue) was recognized from the Vertex partnership expansion. This comprised revenue from the DMD and DM1 licenses worth $202M and from Vertex exercising their "Collaboration Target Options" worth $76.7M and a $6.7M payment for Vertex waving their fourth exclusive license. Neither CRSP nor the author expects this to continue in the next 1-2 years on any metric unless new partnerships are formed and upfront payments are made.

CRSP does have $12.6M in unearned revenue accounted for, but only 1M as current. Analysts do expect another $6.5M in revenue 2H 2020, but that only brings the tangible revenue benchmark to $6.7M for the year; however, the focus is on the viability of the therapeutics and not revenue benchmarks. It seems investors in CRSP think the same. New and old investors should focus on the results of clinical trials for the foundation of any investment thesis as accessing the Vertex milestone payments worth up $1.25B is directly the result of the Phase 1/2 CTX001 results and the Pre-IND research phase targeting DMD, DM1, and CF.

Balance sheet composition:

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Regarding the balance sheet, what stands out for investors is the high cash balance accumulated ($945M), particularly from Vertex, which is sufficient to finance the existing pipeline of 4-core products (3 internal, 1 external) and a few promising research phase therapeutics. The low-unearned revenue ($13M) enlightens investors towards the short term, but with little debt ($50M) and capital leases making up the majority portion ($44M), there isn't a heavy draw on cash that shouldn't produce an advancement towards reaching clinical phases. What is worrisome though is the desire for only 2 external partners (1-lead and 1-sub) which may be attributed to the McKinsey style leadership that in the author's opinion does not foster the right developmental environment and deviates from the biotech norm. This may be a benefit in some investors' eyes as the rewards will be substantial in 3-7 years, but it is a long-term position an investor must make.

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Firstly, any valuation on CRSP at this point is highly speculative, given the inconsistent milestone marks being met and their highly divergent payments with relatively new technology (though bluebird and Novartis are setting precedents). A revenue basis seems the most closely tied to reality and by using 11-14 analyst forecasts, an approximate valuation can be made as above with base-case (+2% upside) being far too conservative due to the reactions investors take upon clinical trial announcements and certainly not enough to lure any biotech investor.

An upside of 311% in an optimistic scenario seems more than compensatory to the risk, given the large cash cushion. However, a premium of 894x Sales is remarkable, but outlandishly inaccurate due to the fluctuations of the revenue position which does not compensate for the high potential of gaining market share in the $220B market their combined therapeutics target. In summary, the author will say that an upside potential exists but is uncertain beyond 30% in the short term (1-2 years).

Data by YCharts

Upcoming Catalysts (1-12 months):

In summary, CRSP is in a very unique position with such a large $945M cash cushion and a promising advancement on therapeutic development with cutting-edge technology and 7+years of experience in it, far greater than most enterprising biotechs looking into genome-editing. The partnership with Vertex, a leader in its sphere, does provide substantiation to the technology CRSP is applying, but with only two partnerships, it seems CRSP is either too early to the game or is not moving fast enough. CRSP does face competition, such as bluebird's TDT gene-therapy; however, what CRSP is building should surpass competition if it can swiftly make it to market compensating investors that must withstand early-clinical phase results risk. CRSP has stated their platforms are worthy of reaching a $100B company status, but the author adds that investors must be patient. There are few companies that the author truly feels uncertain regarding valuing, and CRSP is one of them. Any realistic valuation would greatly underestimate the potential of CRSP, but by averaging the downside and the upside potential, a reasonable price target can be surmised from analyst expectations. Therefore, the author projects CRISPR Therapeutics as a "buy" for the long-term investor with an uncertain 2-year stock price target at $204.63 (+113% upside).

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Crispr Therapeutics: Waiting On Early Data In 2H 2020, But The Clinical Pipeline Shows Promise (113% Upside) - Seeking Alpha

Researchers in the US led by the 2020 Nobel laureate for chemistry Jennifer Doudna have used a CRISPR gene-editing technology to develop a rapid, portable, accurate, and low-cost mobile-based test that can detect the pandemic coronavirus (Sars-CoV2) in five minutes.

This new CRISPR diagnostic method doesnt amplify coronavirus RNA but uses multiple guide RNAs that work in tandem to increase the sensitivity of the test, said the research team in the yet to be peer-reviewed study published in the pre-print server medRxiv. The test does not require expensive lab equipment, and can be deployed for rapid point-of-care testing at doctors offices, schools, and office buildings.

The diagnostic gold standard for coronavirus disease diagnosis is the quantitative reverse transcription-polymerase chain reaction (RT-qPCR) test, which takes five to six hours to produce result. CRISPR-based diagnostics that utilises RNA and DNA-targeting enzymes can augment gold-standard PCR-based testing if they can be made rapid, portable and accurate, said Doudnas team.

The assay achieved ~100 copies/L sensitivity in under 30 minutes and accurately detected a set of positive clinical samples in under 5 minutes. We combined crRNAs targeting SARS-CoV-2 RNA to improve sensitivity and specificity, and we directly quantified viral load using enzyme kinetics. Combined with mobile phone-based quantification, this assay can provide rapid, low-cost, point-of-care screening to aid in the control of SARS-CoV-2, write researchers.

The test also quantifies the amount of virus in a sample, with the strength of the fluorescent signal proportional to the amount of virus in a sample. This cannot be done in standard Covid-19 tests that amplify the virus genetic material to detect it. Detecting a patients viral load can guide treatment decisions. The test needs validation before it is commercially available.

CRISPR diagnosis work by identifying a sequence of RNAabout 20 RNA bases longthat is unique to SARS-CoV-2. They do so by creating a guide RNA that is complementary to the target RNA sequence so it binds to it in solution. The binding turns on the CRISPR tools Cas13 scissors enzyme that cuts single-stranded RNA to release a separately introduced fluorescent particle in the test solution. These fluorescent particles light up to when hit with laser light, signaling the presence of the virus.

CRISPR diagnostics is already being used for Sars-CoV-2 detection, but the new test is the fastest CRISPR-based diagnostic yet.

I think this is an interesting new approach that is faster because it doesnt have an amplification step. But, because it doesnt have the amplification step, it cannot easily detect low viral loads unlike qRT-PCR or FELUDA. The five minutes result is only when starting from RNA with high amount of virus. For usual samples it will be more than 30 minutes. The device requirement is not zero but low - a constant temperature holder and a laser illumination optical box. I think CRISPR based tests will see a lot of innovation and it is a good sign for the fight against COVID-19, said Dr Anurag Agrawal, director, Council of Scientific & Industrial Research-Institute of Genomics and Integrative Biology (CSIR-IGIB), New Delhi.

Scientist from CSIR-IGIB have also developed a precise and cost-effective strip test named after a popular Satyajit Ray detective FELUDA to detect Covid-19 in one hour, starting from RNA to giving a visual readout on the strip.

FELUDA, which is an acronym for FNCAS9 Editor-Linked Uniform Detection Assay, uses CRISPR gene-editing technology to identify and target the genetic material of Sars-CoV2, the virus that causes Covid-19. It has been developed by CSIR-IGIB senior scientists Dr Debojyoti Chakraborty and Dr Souvik Maiti.

FELUDA tests work by combining CRISPR biology and paper strip chemistry. A Cas9 protein, a component of the CRISPR system, is barcoded to interact specifically with the Sars-CoV2 sequence in the patients genetic material.

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

Going forward, CRISPR-based tests have the potential for modification to detect the next emerging virus and rapidly scale up testing, if needed.

The rest is here:
Covid detection with CRISPR, phones in offing - Hindustan Times

With the vision of linking computers up to the LIVE signals of biology, Cardea now adds an advisory team of Key Opinion Leaders covering all the cross disciplinary efforts being done.

SAN DIEGO (PRWEB) October 13, 2020

Cardea Bio Inc., who is using graphene-based Biology-gated Transistors (Cardean Transistors) to directly link the live signals that run biology up to electronics and computers, today announced the Cardea Innovation Council. The Council will serve as an advisory body to guide and help the Cardea team of talents to continue the breakthroughs being made via Cardea's core technology, Cardean Transistors. The council members will also participate in the Company's Innovation Partnership Program on relevant projects. The Council consists of a body of Key Opinion Leaders and experts from diverse science and technical fields and will bring a depth of knowledge to aid Cardea in building the most complete Tech+Bio Communication Chipsets and Infrastructure available for current and future generations.

Cardea is pleased to welcome the Council Members to its team:

Professor Susan Wessler

Distinguished Professor of Genetics and the Neil and Rochelle Campbell Chair for Innovation in Science Education at the University of California Riverside. In 2011 she was elected Home Secretary of the U.S. National Academy of Sciences (NAS), the first woman to hold this position in its 150-year history. She is a plant molecular geneticist known for her contributions to the field of transposon biology and plant genome evolution.

Professor Virginijus Siksnys

Among the very first to discover and characterize the CRISPR-Cas9 complex and recognize its editing potential for "DNA surgery" in many life science applications. His pioneer work was recognized with several international awards including the Kavli Prize. Since CRISPR-Chip is an important chipset type for Cardea, his expertise regarding CRISPR is important in improving fast and precise (amplification-free) DNA and RNA detection.

Dr. Kurt Petersen

Founder and CTO of numerous MEMS (Micro-ElectroMechanical System) companies, including NovaSensor, Verreon and molecular testing company Cepheid. Kurt was recently awarded the IEEE Medal of Honor for his contribution to the field of MEMS. Cardean Transistors are very similar to MEMS in many regards and Kurt's experience with MEMS as it applies to biotechnology will elevate Cardea's chip designs, scalability and capabilities.

Dr. Phil Cotter

Fellow of the American College of Medical Genetics and Genomics as well as founder and Principal of ResearchDx. Dr. Cotter has been a leader in developing sequencing as a clinical diagnostics tool as the Director of Illumina Clinical Services Laboratory, and through ResearchDx, a leader in development of companion diagnostics based on DNA, RNA and protein biomarkers.

Dr. Lauge Farnaes

As Head of Medical Affairs at IDbyDNA, and Medical Doctor & Researcher formally at Rady Children's Genomic Institute with expertise in genetic disorders, infectious disease detection, and nucleic acid-based diagnostics, Dr. Farnaes has among other things helped pioneer the use of Rapid Whole Genome Sequencing to diagnose rare genetic disorders in children.

Dr. Elia Stupka

A visionary Key Opinion Leader in bringing the most advanced forms of big data and analytics to healthcare. Dr. Stupka's work has contributed to the understanding of the human genome, transcriptome, and the development of gene-therapeutics. His understanding of data and its role in enhancing the value of new technology will help steer Cardea's data management capabilities and add value to every Innovation Partner application.

Dr. Paul Grint

Chairman of the Innovation Council and Chairman of the Board of Directors of Cardea. Dr. Grint has served on the Boards of Illumina, AmpliPhi Biosciences, and as the CEO of several companies. Dr. Grint's ability to see the early potential in technology served Illumina well when Next Generation Sequencing was in its infancy as it will Cardea with its Biology-gated Transistors.

"Cardea's core technology is really the convergence of many highly complex technical fields such as life science, data analytics, and semiconductor technology." says Dr. Grint. "In order for Cardea to be successful in its mission to elevate the world's ability to gain new insight into biology, we need the best from every field. The Innovation Council will elevate Cardea's capabilities across all of these fields."

The news of Cardea's Innovation Council comes only weeks after the company announced the first close of their A2 round and first commercial "Powered by Cardea" product launch. This is a major move for Cardea on its mission to continue developing even more products together with their Innovation Partners. To learn more about the Council, visit Cardea's website.

About Cardea Bio

Cardea is linking biology directly up to computers for the very first time by building a Tech+Bio Infrastructure and offering chipsets based on proprietary Biology-gated Transistors, or Cardean Transistors. These transistors leverage graphene, a nanomaterial that in contrast to the common semiconductor material silicon, is biocompatible and a near perfect conductor due to only being one atom thick. It that way replaces optical static observations with interactive live-streams of multi-omics signal analysis, representing a new life science observation paradigm where multi-omics data-streams will be the new norm instead of most of the current standard technologies that are single-omics frozen-in-time datasets. Together with their Innovation Partners, Cardea can link biology directly to compute power and convert real-time biological signals to digital information, allowing for immediate biological insight and a new generation of applications Linking up to Life.

Contacts

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Rob Lozuk

Chief Business Officer

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Amanda Zimmer

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CRISPR Pioneer, Home Secretary of the U.S. National Academy of Sciences, IEEE Medal of Honor Recipient, and other experts joins Cardea Bio's...

Scribe Therapeutics, a biotech firm focused on developing next-generation gene-editing technology, has raised $20 million in its first major round of financing, backed by Andreessen Horowitz. The firm separately unveiled a deal with Biogen to develop CRISPR-based treatments for amyotrophic lateral sclerosis (ALS).

Scribe was cofounded in 2018 by several University of California, Berkeley, scientists, including gene-editing pioneer Jennifer Doudna and protein engineer Benjamin Oakes, who at the time was an entrepreneurial fellow at the Innovative Genomics Institute, where Doudna is president. Their goal was to engineer a newly discovered class of Cas proteins to make them behave better as therapies than the original CRISPR-Cas9 gene-editing system.

The original system was found in bacteria, which use it to recognize and chop up DNA from invading pathogens. Scientists, including Doudna, quickly realized the system could be co-opted to make precise cuts to human DNA. The tool set off a race among companies trying to use it to address the genetic mutations underlying many diseases.

But even with its promise, the CRISPR-Cas9 system comes with evolutionary baggage, Oakes, who is now CEO of Scribe, says. Those systems arent designed to work within the context of the human cell or even the human genome, he says, complicating efforts to turn the technology into drugs.

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Scribe Therapeutics launches to explore next-generation CRISPR technology - Chemical & Engineering News

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CRISPR Industry Size 2019, Market Opportunities, Share Analysis up to 2026 - The Research Process

Several years ago, a brand new method of genetic engineering called CRISPR was invented, and it was based on discoveries made about the rudimentary "immune system" possessed by bacteria. Essentially, bacteria have a way of "remembering" which viruses had infected them previously, and they possess a molecular system that destroys viral DNA that matches that of a prior infection.

The molecular system consists of a DNA-cutting protein called Cas9. (See infographic from Business Insider below.) When equipped with a special guide RNA, Cas9 can be used to cut specific DNA sequences, for instance, a mutated gene that is causing a health problem. Because a broken DNA molecule is dangerous, the cell will attempt to repair it. If a DNA segment is snuck into the cell before the repair occurs, the cell can insert the new (and usually improved) DNA piece, providing a method to "edit" DNA.

The implications for such a technology are obvious. Such a method could be used, for example, to cure a person of a genetic disease or more easily produce genetically enhanced crops for farmers. But there are even cleverer uses. Because the CRISPR-Cas9 system can be designed to be self-propagating, it can be used to force a gene into a population of animals, such as mosquitoes. If this system targets genes that are important for survival or reproduction, then once released, this "gene drive" would rapidly spread through the population, killing off mosquitoes. (See infographic from The Economist.)

Now, a team of researchers writing in the journal Nature Communications has shown that a gene drive can be used to suppress infection with cytomegalovirus, a type of herpesvirus. The underlying molecular mechanism of the gene drive is similar to others before it: A self-propagating chunk of DNA inserts itself into a gene that is important to the virus. In this case, the gene is UL23, which is needed for cytomegalovirus to avoid the human immune response.

The researchers showed that when a cell is infected by both the normal virus (called "wildtype" or "WT") and the modified virus carrying a gene drive ("GD"), the gene drive was able to quickly and efficiently spread through the entire population, representing up to 95% of the final proportion of viruses. The end result is the suppression of viral infection (in cell culture, not in an animal model) because the gene drive virus lacks the important UL23 gene, which is needed for the virus to avoid a potent immune molecule known as interferon gamma(IFN-), which the authors added to the cell culture.

Could such a system work to treat viral infections in humans? Possibly. The authors note that a different gene (other than UL23) might need to be targeted, since lack of this gene is only fatal to the virus if IFN- is added to the cell culture. There are also concerns that a gene drive system could cause the viruses to mutate in various ways and may have unforeseen consequences.

Still, the technology is powerful and should be researched further. The coronavirus pandemic reminds us that we want to have multiple weapons in the public health arsenal should we be confronted with another life-threatening microbe.

Source: Walter, M., Verdin, E. Viral gene drive in herpesviruses. Nat Commun 11, 4884 (2020). Published: 28-Sept-2020. DOI: 10.1038/s41467-020-18678-0

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Gene Drives Could Kill Mosquitoes and Suppress Herpesvirus Infections - American Council on Science and Health

One summer day almost 20 years ago, a group of protestors arrived at a plot of genetically modified corn growing near the town of Montelimar in southern France.

They were led by Jos Bov, a left-wing activist famous for his skirmishes with the law and his tremendous moustache. Using machetes and shears, the protestors uprooted the crops and dumped the debris outside the offices of the regional government.

I thought about Bov this week as I read a new report on the next generation of genetic food technology. The techniques in the report make the processes that Bov opposed look clunky.

The GMOs he destroyed were created by inserting genes from other organisms say a stretch of DNA that confers resistance to a particular herbicide into a plants genome. This brute force approach is time-consuming and hard to control. Now scientists are using a new suite of gene-editing techniques, including a process known as CRISPR, to rapidly and precisely control the behavior of specific plant genes.

Gene-edited crops already exist. Scientists at the biotech firm Corteva, for example, have developed a high-yield strain of a variety of corn used in food additives and adhesives. Yet these initial advances belie the technologys potential.

Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones?

The power of gene editing can be wielded to modify plants and, among other things, achieve significant sustainability wins.

Here are a few potential outcomes explored in the new report, published by the Information Technology & Innovation Foundation, a pro-technology think tank:

This potential is thrilling, and there are signs that it will arrive soon. In China, where the government has made a big bet on gene-editing technology, numerous labs are working on crop strains that require less pesticides, herbicides and water. In the United States, a small but growing group of gene-editing startups is bringing new varieties to market, including an oilseed plant that can be used as a carbon-sequestering cover crop during the winter.

Yet when I read the ITIF report, I thought of Bov. Not because I agree with everything he said. Twenty years and many studies later, we know that the anti-GMO activists were wrong to say that modified crops posed a threat to human health. (The demonization of GMOs had profound consequences nonetheless: Fears about the risks posed by the crops are one reason why the crops are highly restricted in Europe and viewed warily by some consumers on both sides of the Atlantic.)

The reason I thought of Bov is that, at one level, he and other activists were pushing society to take a broader view of GMOs. They wanted people to ask who and what the crops were for, because they believed, rightly, that the crops were produced mainly with the profits of ag companies in mind.

Thats not to say its a bad thing for ag companies to be profitable. But our food systems affect so many aspects of our lives from the composition of the atmosphere to the prevalence of disease. When GMOs first began to be planted, there hadnt been enough debate about how the technology might affect these things. No wonder people were angry.

Thats a lesson I hope we can remember as gene editing shapes agriculture. Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones? If they can, we might end up with crops that everyone wants.

This article was adapted from the GreenBiz Food Weekly newsletter. Sign up here to receive your own free subscription.

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Get ready for the next wave of GMOs | Greenbiz - GreenBiz

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CRISPR in Agriculture Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

The mistake Nobel Prize prognosticators yours truly included make is to look through the greatest hits of biochemistry, biology, and medicine (the areas STAT covers) nuclear hormone receptors! microRNAs! and figure (as last years prediction story did) one of those is due and deserving. The trouble is, as MITs Phillip Sharp, who shared the 1993 medicine Nobel, told me, There is just a lot of good science that will never get recognized.

So focusing on the greatest hits to forecast the science winners who will be announced next week is too simplistic. Theyre all contenders, but the smart money looks for other criteria. Like toggling between discoveries of what cells and molecules do and inventions of techniques that reveal what they do, or between disciplines, or (for medicine) between something that directly cures patients and something about the wonders of living cells.

By that criteria, it might be a techniques turn, since the last such winner in medicine was for turning adult cells into stem cells, in 2012. Could this be the year for optogenetics, which allows brain scientists to control genetically modified neurons with light? I dont think optogenetics has made a big enough impact outside of neuroscience yet, said cancer biologist Jason Sheltzer of Cold Spring Harbor Laboratory, who dabbles in Nobel predictions, but who knows.

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The last Nobel for DNA sequencing was way back in 1980, he pointed out, and since then we have seen the complete sequencing of the human genome, one of humanitys towering achievements. (Sheltzer correctly predicted 2018s medicine Nobel for immuno-oncology pioneer James Allison. The Human Genome Project could win it for the officials who led it, like Francis Collins of the National Institutes of Health and Eric Lander of the Broad Institute. Would Craig Venter, who led a competing private effort, make it to Stockholm, too? Let the betting commence!

Just to be clear, science Nobels arent chosen all that, well, scientifically. For medicine, a five-member Nobel Committee for Physiology or Medicine at Swedens Karolinska Institute sifts nominations and selects candidates. The 50-member Nobel Assembly votes, this year on Oct. 5. So you can get head-scratchers from, say, 20-18-12 or similarly split votes if, say, genetics fanciers split their votes among two contenders. (If you want to know if that happened, hang on until 2070: Nobel records are secret and sealed for 50 years.) For chemistry, chosen on Oct. 7 this year, the five-member Nobel Committee of the Royal Swedish Academy of Sciences likewise sifts nominations and recommends finalists to the academy for a vote.

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Besides invention and discovery switching off in the medicine Nobel, there certainly seems to be periodicity in terms of disciplines taking turns, said David Pendlebury of data company Clarivate Analytics. He has made 54 correct Nobel predictions (usually in the wrong year, but in 29 cases within just two) since 2002 by analyzing how often a scientists key papers are cited by peers and awarded predictive prizes like the Lasker or Gairdner awards.

Neuroscience won the medicine Nobel in 2000, 2004, 2014, and 2017, immunology in 2008, 2011, and 2018, for instance. Infectious disease and cancer win every decade or two, and so are probably also-rans for 2020. Thats why STAT said last year that the 2018 medicine award for immuno-oncology made cancer an unlikely 2019 winner. Yet William Kaelin, Peter Ratcliffe, and Gregg Semenza won for discovering how cells sense and adapt to oxygen availability, through gene regulation, which is tangentially related to cancer. Go figure.

For the medicine prize, periodicity also applies to toggling between super-basic molecular biology and stuff that actually cures people (not year by year, but generally). Last years award for how cells sense changing oxygen levels was pretty abstruse and might shape this years choice.

Prizes with a more clinical focus have been 2003 (MRI), 2005 (H. pylori and ulcers), 2008 (HIV), 2015 (roundworm and malaria therapy), and 2018 (immuno-oncology), [so] maybe a clinical type of prize this year, [such as] hepatitis C treatment, brain stimulation for Parkinsons, cochlear implant, statins Pendlebury said. We wouldnt be surprised at a hep C win for Charles Rice of Rockefeller University and Ralf Bartenschlager of Heidelberg University (2016 Lasker winners) for the super-basic discoveries that led to drugs that cure the viral disease.

Like Pendlebury, Sheltzer believes in predictive prizes. I looked back at the last 20 years of Nobel Prizes in medicine/physiology, he said. Eighty-three percent of them had won at least one of three prizes before the Nobel: the Lasker, the Gairdner, or the Horwitz Prize. Of the five people who have recently won all three, only one works in a field so far ignored by the Nobel committees, he said: Yale School of Medicines Arthur Horwich, a pioneer of protein folding and chaperone proteins. In addition to the Gairdner in 2004, Horwitz in 2008, and Lasker in 2011, he received the $3 million Breakthrough Prize in 2019. So thats guess #1, Sheltzer said.

Unless Weve had a few [medicine] awards that you could classify as cell biology recently oxygen sensing in 2019, autophagy in 2016, even immune regulation is kinda cell biological, Sheltzer acknowledged. So I think a genetics award is more likely than one to Horwich, whose discoveries about how cells fold the proteins they synthesize are central to the understanding of life. STATs nickel says look no further than the 2015 Lasker Basic Medical Research Award: It honored Evelyn Witkin of Rutgers and Stephen Elledge of Harvard for discovering how DNA repairs itself after being damaged.

Might David Allis of Rockefeller and Michael Grunstein of UCLA finally get the call to Stockholm? They discovered one way genes are activated (through proteins called histones). Theyve shared a 2018 Lasker and a 2016 Gruber Prize in Genetics, and basically launched the hot field of epigenetics. I think a prize related to epigenetic control of transcription by DNA and histone modifications could be in order, Kaelin told STAT.

For physiology or medicine, Pendlebury likes Pamela Bjorkman of Caltech and Jack Strominger of Harvard for determining the structure and function of major histocompatibility complex (MHC) proteins, a landmark discovery that has contributed to drug and vaccine development, as well as Yusuke Nakamura of the University of Tokyo for genome-wide association studies that led to personalized approaches to cancer treatment (personally, we doubt this is cancers year again), and Huda Zoghbi of Baylor College of Medicine for work on the origin of neurological disorders.

In chemistry, Pendlebury likes Moungi Bawendi of MIT, Christopher Murray of the University of Pennsylvania, and Taeghwan Hyeon of Seoul National University for synthesizing nanocrystals, a cool new way to deliver drugs, and Makoto Fujita of the University of Tokyo for discovering supramolecular chemistry, in which lab-made molecules self-assemble by emulating how nature makes them. That has some overlap with Frances Arnolds 2018 Nobel for chemistry, so were skeptical, but who knows?

Lets address the elephant in the Nobel anteroom, and the chatter that the revolutionary genome editing technique CRISPR will win for chemistry. (Its value in medicine is still TBD, but its stellar biochemistry.)

The discovery of the CRISPR-Cas9 system is certainly worthy of a Nobel Prize, Kaelin said. I suspect the challenge here will be to get the attribution right. Perhaps there could be a chemistry prize for the basic mechanism and a medicine prize for application to somatic gene editing in human cells.

By attribution, he means, who gets CRISPR credit? Only three people can share a Nobel. But CRISPR has more mothers and fathers than that. Jennifer Doudna of the University of California, Berkeley, and her collaborator Emmanuelle Charpentier have won a slew of predictive prizes for their work turning a bacterial immune system into a DNA editor, but dark horse Virginijus iknys of Vilnius University shared the 2018 $1 million Kavli Prize in nanoscience for his CRISPR work. And Feng Zhang of the Broad Institute is more widely cited than the above three, Pendlebury said, a marker of what colleagues think.

CRISPR citations built up more to Feng Zheng et al. than to Doudna and Charpentier, but I dont think that matters as much as judgments about priority claim, Pendlebury said. There are more than three to credit and I do think that is problematic. Bad feelings are not something the Nobel Assembly wants to generate, I am sure.

CRISPR will win, said CSHLs Sheltzer. Its a question of when, not if. Zhang/Doudna/Charpentier/Horvath/Barrangou shared the Gairdner. Pick 2 or 3 of them?

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Dust off the crystal ball: It's time for STAT's 2020 Nobel Prize predictions - STAT

CRISPR & Cas Genes Market size 2020-2025 report, added by Market Study Report, unveils the current & future growth trends of this business sphere in addition to outlining details regarding the myriad geographies that form a part of the regional spectrum of CRISPR & Cas Genes market. Intricate details about the supply & demand analysis, contributions by the top players, and market share growth statistics of the industry are also elucidated in the report.

The research study on the CRISPR & Cas Genes market projects this industry to garner substantial proceeds by the end of the projected duration, with a commendable growth rate liable to be registered over the estimated timeframe. Elucidating a pivotal overview of this business space, the report includes information pertaining to the remuneration presently held by this industry, in tandem with a meticulous illustration of the CRISPR & Cas Genes market segmentation and the growth opportunities prevailing across this vertical.

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A brief run-through of the industry segmentation encompassed in the CRISPR & Cas Genes market report:

Competitive landscape:

Companies involved: CRISPR Therapeutics, Genscrip, AstraZeneca, Caribou Biosciences, Inc., Cellectis, Addgene, F. Hoffmann-La Roche Ltd., Editas Medicine, Inc., Horizon Discovery Group Plc, Egenesis, Takara Bio, Inc., Mammoth Biosciences, Synthego, Danaher Corporation, Merck KGaA, Lonza, Cibus, New England BioLabs, Intellia Therapeutics, Inc., Inscripta and Inc

Vital pointers enumerated:

The CRISPR & Cas Genes market report provides an outline of the vendor landscape that includes companies such as CRISPR Therapeutics, Genscrip, AstraZeneca, Caribou Biosciences, Inc., Cellectis, Addgene, F. Hoffmann-La Roche Ltd., Editas Medicine, Inc., Horizon Discovery Group Plc, Egenesis, Takara Bio, Inc., Mammoth Biosciences, Synthego, Danaher Corporation, Merck KGaA, Lonza, Cibus, New England BioLabs, Intellia Therapeutics, Inc., Inscripta and Inc. Parameters such as the distribution and sales area, alongside other pivotal details such as the firm profiling and overview have also been mentioned.

The study mentions the products manufactured by these esteemed companies as well the product price prototypes, profit margins, valuation accrued, and product sales.

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Geographical landscape:

Regions involved: USA, Europe, Japan, China, India, South East Asia

Vital pointers enumerated:

Segmented into USA, Europe, Japan, China, India, South East Asia, as per the regional spectrum, the CRISPR & Cas Genes market apparently covers most of the pivotal geographies, claims the report, which compiles a highly comprehensive analysis of the geographical arena, including details about the product consumption patterns, revenue procured, as well as the market share that each zone holds.

The study presents details regrading the consumption market share and product consumption growth rate of the regions in question, in tandem with the geographical consumption rate with regards to the products and the applications.

Product landscape

Product types involved: Vector-based Cas, DNA-free Cas and Cell Line Engineering

Vital pointers enumerated:

The CRISPR & Cas Genes market report enumerates information with respect to every product type among CRISPR Therapeutics, Genscrip, AstraZeneca, Caribou Biosciences, Inc., Cellectis, Addgene, F. Hoffmann-La Roche Ltd., Editas Medicine, Inc., Horizon Discovery Group Plc, Egenesis, Takara Bio, Inc., Mammoth Biosciences, Synthego, Danaher Corporation, Merck KGaA, Lonza, Cibus, New England BioLabs, Intellia Therapeutics, Inc., Inscripta and Inc, elaborating on the market share accrued, projected remuneration of each type, and the consumption rate of each product.

Application landscape:

Application sectors involved: Biotechnology and Pharmaceutical Companies, Academics and Government Research Institutes and Contract Research Organizations (CROs

Vital pointers enumerated:

The CRISPR & Cas Genes market report, with respect to the application spectrum, splits the industry into Biotechnology and Pharmaceutical Companies, Academics and Government Research Institutes and Contract Research Organizations (CROs, while enumerating details regarding the market share held by each application and the projected value of every segment by the end of the forecast duration.

The CRISPR & Cas Genes market report also includes substantial information about the driving forces impacting the commercialization landscape of the industry as well as the latest trends prevailing in the market. Also included in the study is a list of the challenges that this industry will portray over the forecast period.

Other parameters like the market concentration ratio, enumerated with reference to numerous concentration classes over the projected timeline, have been presented as well, in the report.

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CRISPR & Cas Genes Market Analysis with Key Players, Applications, Trends and Forecasts to 2025 - The Daily Chronicle

DUBLIN--(BUSINESS WIRE)--The "Global Mice Model Market by Mice Type (Inbred, Knockout), Technology (CRISPR, TALEN, ZFN), Application (Oncology, Diabetes, Immunology), Service (Breeding, Cryopreservation, Genetic Testing), Care Products (Cages, Bedding, Feed), and Region - Forecast to 2025" report has been added to ResearchAndMarkets.com's offering.

The global mice model market size is projected to reach USD 1.9 billion by 2025 from USD 1.4 billion in 2020, at a CAGR of 6.4% during the forecast period.

The growth of this market is driven mainly by ongoing innovations in mice models, growing demand for personalized medicine, continuous support in the form of grants and investments, growth in the number of pharmaceutical R&D activities, and increasing focus of associations on the development of embryonic stem cells as well as knockout and mutant mice. Moreover, the popularity of humanized mice models and emerging technologies such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) will present lucrative opportunities for the market in the coming years.

By mice type, the genetically engineered mice accounted for the fastest-growing segment of the mice model market

Genetically engineered mice segment is anticipated to be the fastest-growing due to the widespread use of these mice in diverse research areas, the emerging CRISPR technology, increasing focus on personalized medicine with the continuous introduction of new models

By service, the breeding segment accounted for the largest share of the mice model market

The breeding segment is expected to account for the largest market share in 2020, with the highest growth rate as well. This can primarily be attributed to the increasing demand for mice models for drug discovery and development and the subsequent increase in the demand for personalized medicines.

Oncology segment expected to grow at the fastest growth rate during the forecast period

Based on application, the mice model market has been segmented into oncology studies, immunology and inflammation studies, endocrine metabolic studies, cardiovascular studies, central nervous system studies (CNS), genetic studies, infectious disease studies, and other disease studies. The endocrine disease studies segment is further segmented into diabetes and other endocrine metabolic disease. The oncology segment is expected to account for the largest market share in 2020, with the highest growth rate as well. This can primarily be attributed to the increasing number of patients who have cancer and the subsequent increase in the demand for cancer therapies.

By technology type, CRISPR/Cas9 accounted for the largest share of the mice model market

CRISPR is the most widely used technology in the mice model market and contributed to the largest share of the mice model market in 2020. Ease of design, high efficiency, and relatively lower cost have increased the demand for CRISPR-customized mice models.

By mice care product, cages segment accounted for the largest share of the mice model market

Based on mice care products, the mice model market has been segmented into cages, feed, bedding, and other products (gnotobiotic equipment, water systems, and accessories). The cages segment accounted for the largest share of the mice model market. This can be attributed to the availability of a wide range of cages designed for specific research needs and the higher cost of cages compared to other care products.

Asia Pacific: The fastest-growing region in the mice model market

The Asia Pacific market is projected to grow at the highest CAGR during the forecast period. Several global pharmaceutical firms have entered the APAC market to tap the significant growth opportunities in emerging Asian countries and lower their production costs by shifting their drug discovery R&D operations and manufacturing to the region. A large number of qualified researchers and low-cost operations in APAC countries, such as India and China, are some of the major factors supporting this trend.

North America: The largest share of the drug discovery services market

North America, which includes the US and Canada, accounted for the largest share of the mice model market. The large share of the North America region can be attributed to the presence of major players operating in the mice model market in the US, growing biomedical research in the US, and rising preclinical activities by CROs and pharmaceutical companies in the region.

Companies Mentioned

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$1.9 Billion Mice Model Market by Mice Type, Technology, Application, Service, Care Products - Global Forecast to 2025 - ResearchAndMarkets.com -...

Timothy Ray Brown, who became the first HIV patient to be cured of the infection, died September 29 of leukemiathe very disease that led to the fortuitous eradication of the virus from his body. He was 54.

Until he disclosed his identity, Brown was known as the Berlin patient, whose HIV infection was eliminated in 2007 after undergoing a stem cell transplant to treat acute myeloid leukemia. The bone marrow donor was selected to have a naturally occurring genetic variant that blocked HIV from entering cells. The treatment workedboth for his cancer, and his viral infection.

Timothy symbolized that it is possible, under special circumstances to cure HIV, Gero Htter, the doctor who performed the stem cell transplant, tells theAssociated Press.

Until2016, Brown remained the only person in the world to have been cured of AIDS using this approach and his unique experience motivated him to advocate for AIDS research. As he toldThe Scientist in 2015, I didnt want to be the only one in my club.

Brown was born in 1966 and grew up in Seattle. He was living in Berlin when he received the diagnosis of leukemia and sought treatment from Htter. The doctor had previously read about individuals with variants in the CCR5 gene, which codes for a receptor on cell surfaces, that gives themnatural immunity to HIV. Upon finding out that Brown was HIV-positive, Htter decided to look for a bone marrow donor who might have this variant. As Htter explained to The Scientist in 2015, he screened dozens of donors until he found one with the so-called delta32 mutation.

Within months of the transplant, the virus was gone from Browns cells, although his recovery was difficult and he required a second transplant to treat the leukemia.

In 2012, Brown and activist Dave Purdy started the Cure for AIDS Coalition to raise awareness of HIV research. According to aFacebook post by Browns partner, Tim Hoeffgen, Tim committed his lifes work to telling his story about his HIV cure and became an ambassador of hope. Tim also gave numerous blood and tissue samples to researchers after his cure.

The invasiveness of the bone marrow transplant precludes it from being applied more widely to HIV patients, but the insights gained from Browns successful cure have inspired further work on CCR5. For instance, in 2017, researchers used CRISPR to disrupt the gene in human hematopoietic stem cells anddemonstrated that these cells could ward off HIV infection in mice transplanted with them. More recently, andcontroversially, the gene was a target of CRISPR-based editing in human embryos to make them resistant to HIV.

Brown never again tested positive for HIV. His leukemia, however, relapsed five months ago.

Timothy was a champion and advocate for keeping an HIV cure on the political and scientific agenda, Sharon Lewin, the director of the Doherty Institute in Melbourne, Australia, tells theBBC. It is the hope of the scientific community that one day we can honour his legacy with a safe, cost-effective and widely accessible strategy to achieve HIV remission and cure using gene editing or techniques that boost immune control.

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Timothy Ray Brown, First Person to Be Cured of HIV, Dies - The Scientist

The comprehensive research report on theCRISPR and Cas Genes Marketinfluences iterative and comprehensive research methodology to offer insights into the existing market scenario over the forecast timeframe. The report also delivers in-depth details about the growth and development trends that will have a major impact on the behavior of the CRISPR and Cas Genes market in the approaching years. Furthermore, the report touches upon other key pointers such as the regional aspects and policies overriding the industry. The report suggests that the global CRISPR and Cas Genes market Demand is expected to witness a considerable CAGR growth of % during the forecast period and surpass the value of ~US$ by 2026.

Report Covers Impacts of COVID-19 to the market.

The on-going pandemic has overhauled various facets of the market. This research report provides the financial impacts and market disturbance on the CRISPR and Cas Genes market. It also includes analysis on the potential lucrative opportunities and challenges in the foreseeable future. Fact.MR has interviewed various delegates of the industry and got involved in the primary and secondary research to confer the clients with information and strategies to fight against the market challenges amidst and after COVID-19 pandemic.

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Market Segmentation:

Few of the companies that are covered in the report,

Note: Additional companies can be included in the list upon the request.

On the basis of product,

On the basis of end use,

On the basis of region, the CRISPR and Cas Genes market study contains:

The research report provides a detailed analysis of the prominent player in the market, products, applications, and regional analysis which also include impacts of government policies in the market. Moreover, you can sign up for the yearly updates on the CRISPR and Cas Genes market.

Key Questions Answered in this Report on the CRISPR and Cas Genes Market:

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In the Patent Trial and Appeal Board's decision on motions issued September 10th in Interference No. 106,115 (see"PTAB Decides Parties' Motions in CRISPR Interference")between Senior Party The Broad Institute, Harvard University, and the Massachusetts Institute of Technology (collectively, "Broad") and Junior Party the University of California/Berkeley, the University of Vienna, and Emmanuelle Charpentier (collectively, "CVC") the Board denied Broad's Motion No. 2 to substitute the Count.

To recap, the Count in the '115 interference as declared recited in the alternative either claim 18 of the Broad's U.S. Patent No. 8,697,359 (dependent on claim 15), which taken together recites the following invention:

An engineered, programmable, non-naturally occurring Type II CRISPR-Cas system comprising a Cas9 protein and at least one guide RNA that targets and hybridizes to a target sequence of a DNA molecule in a eukaryotic cell, wherein the DNA molecule encodes and the eukaryotic cell expresses at least one gene product and the Cas9 protein cleaves the DNA molecules, whereby expression of the at least one gene product is altered; and, wherein the Cas9 protein and the guide RNA do not naturally occur together,wherein the guide RNAs comprise a guide sequence fused to a tracr sequence.

(where the underlined portion recites the relevant language from claim 18), or Claim 156 of Berkeley's U.S. Patent Application No. 15/981,807:

A eukaryotic cell comprising a target DNA molecule and an engineered and/or non-naturally occurring Type II Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)- CRISPR associated (Cas) (CRISPR-Cas) system comprisinga) a Cas9 protein, or a nucleic acid comprising a nucleotide sequence encoding said Cas9 protein; andb) a single molecule DNA-targeting RNA, or a nucleic acid comprising a nucleotide sequence encoding said single molecule DNA-targeting RNA; wherein the single molecule DNA-targeting RNA comprises: i) a targeter-RNA that is capable of hybridizing with a target sequence in the target DNA molecule, and ii) an activator-RNA that is capable of hybridizing with the targeter-RNA to form a double-stranded RNA duplex of a protein- binding segment,wherein the activator-RNA and the targeter-RNA are covalently linked to one another with intervening nucleotides; andwherein the single molecule DNA-targeting RNA is capable of forming a complex with the Cas9 protein, thereby targeting the Cas9 protein to the target DNA molecule, whereby said system is capable of cleaving or editing the target DNA molecule or modulating transcription of at least one gene encoded by the target DNA molecule.

Broad's Motion No. 2 requested that the Board substitute proposed Count 2:

A method, in a eukaryotic cell, of cleaving or editing a target DNA molecule or modulating transcription of at least one gene encoded by the target DNA molecule, the method comprising:contacting, in a eukaryotic cell, a target DNA molecule having a target sequence with an engineered and/or non-naturally-occurring Type II Clustered Regularly lnterspaced Short Palindromic Repeats (CRISPR)-CRISPR associated Cas) (CRISPR-Cas) system comprising: a) a Cas9 protein, and b) RNA comprising i) a targeter-RNA that is capable of hybridizing with the target sequence of the DNA molecule or a first RNA comprising (A) a first sequence capable of hybridizing with the target sequence of the DNA molecule and (B) a second sequence; and ii) an activator-RNA that is capable of hybridizing to the targeter-RNA to form an RNA duplex in the eukaryotic cell or a second RNA comprising a tracr sequence that is capable of hybridizing to the second sequence to form an RNA duplex in the eukaryotic cell,wherein, in the eukaryotic cell, the targeter-RNA or the first sequence directs the Cas9 protein to the target sequence and the DNA molecule is cleaved or edited or at least one product of the DNA molecule is altered.

The distinction Broad made was between embodiments of CRISPR methods that are limited to "single-molecule guide RNA" (aka "fused" or "covalently linked" species), versus embodiments that encompass single-molecule and "dual molecule" species (wherein in the latter versions, the "targeter-RNA" and "activator-RNA" as recited in the proposed Count are not covalently linked). Broad argued that its Proposed Count 2 should be adopted by the Board because it "properly describes the full scope of the interfering subject matter between the parties because both parties have involved claims that are generic, non-limited RNA claims." The brief also argued that Proposed Count 2 "sets the correct scope of admissible proofs [i.e., their own] for the breakthrough invention described by the generic claims at issue in these proceedingsthe successful adaption of CRISPR-Cas9 systems for use in eukaryotic environments," which Broad contended current Court 1 (in either alternative) does not.

The Board denied this motion for the simple reason that, in its opinion, "Broad fails to provide a sufficient reason why the count should be changed." Citing Louis v. Okada, 59 U.S.P.Q.2d 1073, 1076 (BPAI 2001) (relied upon in opposition by CVC), the Board notes that it will only change the Count when reasons for doing so are "compelling." Broad's motion argued that their claims (and CVC's) were directed to eukaryotic embodiments of CRISPR that were not limited to either single- or dual-molecule RNA species, but that the phrase "guide RNA" was generic. Based on the claim construction, the Board rejected this construction, limiting the claims to single-molecule RNA embodiments.

The Decision also states that "Broad's argument for broadening the scope of the count to be generic as to RNA configuration is unpersuasive." According to the Decision, CVC convinced the Board that there were other differences between Count 1 (as declared in the interference) and Broad's proposed Count 2. These include that Count 2 is directed to a method whereas Count 1 recites system or eukaryotic cell. This is enough, the Board states, for the PTAB to deny Broad's Motion No. 2 simply on these grounds. The Board also was persuaded by CVC's argument that all of the Broad's claims are directed to "guide RNA" or "chimeric RNA" and thus to single-RNA molecule eukaryotic CRISPR embodiments. Further, the Board faulted the Broad for not specifically identifying all the claims it contends recite generic eukaryotic CRISPR embodiments with regard to its RNA components. Continuing, the Decision asserts that Broad also failed to convince the Board that the few claims that expressly recited "fused" RNA embodiments were sufficient under the doctrine of claim differentiation to construe the independent claims as encompassing both single- and dual-RNA molecule eukaryotic CRISPR embodiments.

As is its wont, the Board identified formal deficiencies in some Broad arguments that were sufficient to deny the relief requested under the rubric set forth in 37 C.F.R. 41.121(b) that "the party filing the motion has the burden of proof to establish that it is entitled to the requested relief." These include instances where the Broad's brief cited a footnote that does not stand for the cited proposition, and hence that "CVC did not have notice of arguments regarding claim 15 or of any other claim Broad asserts is directed to a generic RNA configuration without using the term 'guide RNA'". Accordingly, the Board concluded that "[b]ecause Broad did not provide arguments about the interpretation of specific claims in its Motion 2 we are not persuaded by its argument that the scope of the 'vast majority' of its claims requires a broader count."

The Board's Decision also turns on its head the Broad's argument (recited throughout its briefing) that this interference is unfair to Broad due to "CVC's strategic decisions" in earlier Interference No. 105,048 between the parties. The Board notes that the outcome in that interference, that there was no interference-in-fact, "achiev[ed] Broad's desired remedyending the interference." "Had Broad wished to remain in a priority contest with CVC under the count in that interference, it could have chosen not to file the motion for no interference-in-fact," according to the decision, and thus the Board saw "no unfairness in Broad not having had a chance to present its best proofs in a priority contest with CVC in the '048 interference under these circumstances."

This portion of the decision concludes by denying Broad's alternative remedy of redeclaring the interference with both Counts, the Board stating its reasoning that "Broad fails to explain why this would be an appropriate remedy, given that we are not persuaded that a majority, or even a significant number, of its claims are drawn to a generic RNA configuration."

The remainder of the Board's Decision will be discussed in future posts.

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PTAB Denies Broad Motion No. 2 to Substitute the Interference Count - JD Supra

SAN FRANCISCO, Sept. 28, 2020 /PRNewswire/ -- SynBioBeta, the leading community of biological engineers, investors, innovators, and entrepreneurs to build a betterworld with biology, announced the schedule for its 2020 Global Synthetic Biology Summit.

The Summit will feature such luminaries as Tristan Harris (Center for Humane Technology), George Church (Harvard), Jennifer Holmgren (LanzaTech), Christina Smolke (Antheia), Sylvia Wolf (AquaBounty), Ed Boyden (MIT), and Timothy Lu (MIT).

Despite the economic slowdown of COVID, synthetic biology startups raised arecord-setting $3.0 billion in the first half of 2020. While funding is strong for tools and technologies companies -- the engine of thebioeconomy-- there is increasing investment in synthetic biology-enabled companies in consumer products, food, agriculture, medicine, chemicals, materials, and other manufacturing sectors, signaling the impact techand biology is poised tohave on every industry.

"This year, the pandemic has brought previously unimaginable challenges to ourcommunity, not just how we meet and work, but more importantly, how we respondto the society's urgent needs," said John Cumbers, founder and CEO of SynBioBeta,which earlier in the year hosted a series of events on synthetic biology and thepandemic. "Synthetic biology is ready to turn today's industry on its head and revolutionize the way we do business. In the same way that every company today isin some wayan Internet company, every company will one day be a biology company. SynBioBeta 2020 is the place to get ahead of the curve."

This year's conference will explore how engineered biology will disrupt consumer products, food, agriculture, medicine, chemicals, materials, and more. Sessions include:

Each year, SynBioBeta is honored to recognize synthetic biology leaders whoembody the best of this industry and the aims it seeks to achieve. This year'swinners are an exceptional group of innovators who have helped the communitygrow while making profound contributions to society:

About SynBioBeta 2020SynBioBeta 2020 is the Global Synthetic Biology Conference that unites leadingbiological engineers, investors, innovators, and entrepreneurs who are building the future with biology. This year's digital offering gives you even more ways to connect,including our annual conference, new events and grand challenges, access to online contentand groups, and AI-powered networking. Learn the latest technologies, hear the big announcements in the field, make new partnerships, meet investors, and discover new companies. Learn more and register here.

About SynBioBetaSynBioBeta is the leading community of innovators, investors, engineers, andthinkers who share a passion for using synthetic biology to build a better, more sustainable universe. We create and energize innovation communities to make theimpossible possible via unparalleled opportunities for growth, networking, storytelling, and learning.

SynBioBeta offers a weekly industry digest, The Bioeconomy Hub membershipprogram, the SynBioBeta Podcast, Good Genes magazine, and educationalcourses in addition to providing our world-class industry partners with opportunities for advertising, partnership, trade show exhibition, strategic consultation, and promotion.

For more information, visit http://www.synbiobeta.com.

Contact: Amanda Prieto, [emailprotected], (707) 344-8279

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Synthetic Biology Industry Gathers at SynBioBeta 2020 Global Summit to Grow the Bioeconomy, Fight the Pandemic, and Honor CRISPR Pioneer Jennifer...