Archive for the ‘Crispr’ Category

Dec. 2, 2019 10:30 UTC

CAMBRIDGE, Mass. & SEATTLE--(BUSINESS WIRE)-- Cyrus Biotechnology, Inc., and the Broad Institute of MIT and Harvard have embarked on a scientific collaboration to optimize CRISPR for use in developing novel human therapeutics.

CRISPR allows for the highly specific and rapid modification of DNA in a genome, which can dramatically accelerate the drug discovery process.

Feng Zhang will be the principal investigator for the Broad for the collaboration. He is also an investigator of the Howard Hughes Medical Institute (HHMI).

Together, researchers from Cyrus and Broad will work together to mitigate the possibility of the body mounting an immune response against CRISPR. The teams are committed to making the results of their collaboration broadly available for research to help ensure that therapeutic development bringing this technology to the clinic has the best chance of success, while also considering important ethical and safety concerns. The teams have also committed to publishing their results in peer reviewed journals and to make this work freely available to the non-profit and academic scientific community.

Issi Rozen, chief business officer at the Broad Institute, said, Broad researchers and their collaborators have pioneered the development and sharing of new genome editing tools, such as CRISPR-Cas9, which are revolutionizing and accelerating nearly every aspect of disease research and drug discovery around the world. With this collaboration, scientists will continue to improve the technology towards new tools and therapeutics, important to benefiting patients in the long term.

Cyrus CEO Dr. Lucas Nivn added, We have validated our computational deimmunization platform in a variety of systems, and now seek to apply it where it can make a major impact. Given the extensive therapeutic possibilities of CRISPR systems, and the leading position the Broad Institute and Dr. Zhang hold, we are very excited to work in partnership with them to make these molecules more amenable for use in humans with maximal efficacy and minimal side effects.

Cyrus provides commercial and partnered access to Rosetta, which is the worlds leading protein modeling and design software platform. Rosetta has been used to direct the computational design of multiple biologic molecules that have advanced to both pre-clinical and clinical development. Among these are drugs being developed by companies including PVP Biologics, Tocagen, Lyell and others.

About Cyrus Biotechnology

Cyrus Biotechnology, Inc. is a privately-held Seattle-based biotechnology software company offering software and partnerships for protein engineering to accelerate discovery of biologics and small molecules for the Biotechnology, Pharmaceutical, Chemical, Consumer Products and Synthetic Biology industries. Cyrus methods are based on the Rosetta software from Prof. David Bakers laboratory at the University of Washington and HHMI, the most powerful protein engineering software available. Cyrus customers include 13 of the top 20 Global Pharmaceutical firms and is financed by leading investors in both Technology and Biotechnology, including Trinity Ventures, Orbimed, Springrock Ventures, Alexandria Venture Investments, and W Fund.

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Cyrus Biotechnology and the Broad Institute of MIT and Harvard Launch Multi-Target Collaboration to Develop Optimized CRISPR Gene Editing Technology -...

Global CRISPR in Agriculture Market valued approximately USD XX million in 2016 is anticipated tgrow with a healthy growth rate of more than XX% over the forecast period 2017-2025. Increasing demand in drug discovery, late pregnancies leading tbirth disorders, synthetic genes leading the way; aging genetic disorders and investment in path breaking research technology are the drivers for CRISPR Market. Drug discovery technology market plays a dominant role in boosting the CRISPR market. Genome editing has been revolutionized with the discovery of the CRISPR-CAS9 system from streptococcus pyogenes.

Request a Sample Copy of this[emailprotected]https://www.orbisresearch.com/contacts/request-sample/2129000

The objective of the study is tdefine market sizes of different segments & countries in recent years and tforecast the values tthe coming eight years. The report is designed tincorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report alscaters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall alsincorporate available opportunities in micrmarkets for stakeholders tinvest along with the detailed analysis of competitive landscape and product offerings of key players.

The detailed segments and sub-segment of the market are explained below:

By Crop Type:Staple CropsFruits & VegetablesOrnamentalsOthers

By Regions:North AmericaU.S.CanadaEuropeUKGermanyAsia PacificChinaIndiaJapanLatin AmericaBrazilMexicoRest of the World

To make an enquiry on[emailprotected]https://www.orbisresearch.com/contacts/enquiry-before-buying/2129000

Furthermore, years considered for the study are as follows:Historical year 2015Base year 2016Forecast period 2017 t2025

Some of the key manufacturers involved in the market are:

DuPont, Cibus, Monsanto, Bayer AG. Acquisitions and effective mergers are some of the strategies adopted by the key manufacturers. New product launches and continuous technological innovations are the key strategies adopted by the major players.

Browse full[emailprotected]https://www.orbisresearch.com/reports/index/global-crispr-in-agriculture-market-forecasts-2017-2025

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CRISPR in Agriculture Market 2019 by Services, Application, Key Players, Size, Trends and Forecast 2025 - Downey Magazine

Traditional gene therapy has seen numerous challenges during its decades of development, but scientists seem to have finally figured out how to get the treatment to work with regulatory approvals forNovartis' (NYSE:NVS) Zolgensma and bluebird bio's (NASDAQ:BLUE) Zynteglo this year. The process involves inserting genes into diseased cells to express missing or mutated proteins.

Storming onto the scene over the past few years, CRISPR/Cas9, championed by CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT) and Intellia Therapeutics (NASDAQ:NTLA), offered hope for more precise gene editing. At the very least, the process can insert the gene into a precise location in the genome. More impressive -- and something that traditional gene therapy can't readily do -- CRISPR/Cas9 offers the possibility of deleting problematic genes or making specific changes to mutated genes to restore their functions.

Image source: Getty Images.

CRISPR/Cas9 appeared to be working well in preclinical models, and last week, investors got a first look at how the therapy is working in humans with CRISPR Therapeutics and its development Vertex Pharmaceuticals (NASDAQ:VRTX) announcing results for the first two patients treated with CTX001.

One patient with a blood disorder called transfusion-dependent beta thalassemia (TDT) required 16.5 transfusions per year over the two years before being treated with CTX001, but nine months after treatment, the patient was transfusion independent with high expression of fetal hemoglobin, the gene inserted into the patients' cells.

The other patient had sickle cell disease (SCD) with an average of seven vaso-occlusive crises (VOCs) per year over the two years before the study started. Four months after being treated with CTX001, the patient was free of VOCs, which are caused by sickle-shaped red blood cells that block blood vessels. Like the beta thalassemia patient, the SCD patient had expression of fetal hemoglobin.

The results from the first two patients look comparable to Bluebird's Zynteglo, which also treats TDT and SCD by increasing hemoglobin levels. But this was data from just two patients, and investors should still have plenty of questions as we get additional data:

Consistency: One patient in each disease doesn't say much about how well the treatment works in the average patient. What will the efficacy look like after the treatment of a few dozen patients?

Durability: Gene editing and gene therapy are designed to be cures. Do both last forever?

Manufacturing: Bluebird had to adjust its manufacturing procedure to increase expression to treat patients requiring higher expression. Will the initial CRISPR/Cas9 manufacturing procedure work for all patients?

In vivo/ex vivo: That's Latin for in or outside of a living thing -- in this case a human being. CTX001 and Zynteglo are ex vivo treatments because cells are taken from the patient, manipulated to express the gene of interest, and put back into the patient. Novartis has shown that gene therapy can work in vivo with Zolgensma delivered via an injection of a viral vector. Can CRISPR/Cas9 work in vivo in humans? Editas Medicine hopes so, but the company still hasn't advanced a treatment into the clinic.

Last week's data release offers plenty of hope for investors in CRISPR/Cas9 and traditional gene therapy companies should certainly be looking in the rearview mirror at the technology coming up from behind, but it's still way too early to pick a winner between traditional gene therapy and CRISPR/Cas9.

The right answer for investors in biotech companies might end up being to buy both. The upside potential for curing diseases may end up outweighing the downside if one technology doesn't end up working out.

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CRISPR vs. Gene Therapy Round 1: What Investors Need to Know - The Motley Fool

CRISPR is a tool used by researchers to precisely edit genes and has shown potential for treating genetic diseases. This article delves into some recent developments and explores what the future holds for CRISPR.

CRISPR genome editing is a promising field that enables researchers to precisely delete, replace or edit genes.

CRISPR-Cas is a prokaryotic defence system whereby bacteria use RNA molecules and CRISPR-associated (Cas) proteins to target and destroy the DNA of invading viruses. This molecular machinery has been repurposed by researchers to target and edit specific sections of any DNA, whether bacterial or human.

Despite the success of CRISPR, the technique is far from refined. In certain situations, the editing process can result in off-target DNA being changed, causing unwanted effects. Also, CRISPR-Cas9 is a large molecular complex, with both the Cas9 nuclease and an engineered single-guide RNA (sgRNA) that helps the nuclease locate its target. This can make its delivery into the nucleus of the cell, where CRISPR needs to access DNA, difficult.

Consequently, many researchers have sought improvements to CRISPR with the gene editing method expected to continue development well into the future.

Here, three researcher groups who have contributed to recent CRISPR developments explain their work and predict how CRISPR may evolve.

In an attempt to multiplex CRISPR systems to target lots of genes, researchers at ETH Zurich in Switzerland swapped the Cas9 enzyme for Cas12a. Using this plasmid allowed the researchers to simultaneously edit genes in 25 target sites. The team predicts that dozens or even hundreds more sites could be modified using this method.

Genes and proteins in cells interact in many different ways. Each dot represents a gene; the lines are their interactions. For the first time, the new method uses biotechnology to influence entire gene networks in one single step (credit: ETH Zurich/Carlo Cosimo Campa).

Cas12a enabled the researchers to attach shorter sgRNA address molecules than when using Cas9. The shorter length molecules mean that more can fit onto the plasmid, which is a circular DNA molecule that acts as the blueprint of the Cas enzyme, thus enabling CRISPR to edit many genes in a short space of time.

Professor Randall Platt, who led the research, explained that his teams technique is conditional, inducible and orthogonal.

This development offers an improvement on traditional CRISPR technology, which only enables one gene to be edited at a time. This technique therefore speeds the process up, allowing CRISPR to edit many genes simultaneously. It also means that the expression of some genes can upregulated while others can be downregulated.

Platt says that their technique is drastically better, at targeting multiple genes and it afforded the researchers sophisticated control over cellular genomes and transcriptomes.

Another development for CRISPR technologies came from researchers at Duke University in the US. The team successfully used Class 1 CRISPR systems for the first time to edit the epigenome of human cells. Conventional CRISPR-Cas9 methods are categorised as Class 2 systems.

The Class 1 technique makes use of multiple proteins in a process called CRISPR-associated complex for antiviral defence (Cascade). This complex binds with high accuracy to the correct sites. After binding, Cascade utilises a Cas3 protein to target and edit the DNA. They were also able to both activate and repress target gene expression.

Illustrations representing the components of the common dCas9 system (top) and the Cascade system (bottom) (credit: Gersbach Lab).

The team says that this research contributes to an enhancement of CRISPR technologies as it provides a potential alternative for CRISPR-Cas9 when there are complications such as immune responses to Cas proteins. It can also recruit various modifiers of gene regulation, including activators and repressors, to a gene.

Associate ProfessorCharles Gersbach, one of the lead researchers, says that the team will continue to explore CRISPR biology and how the Class 1 method can be developed for gene editing.

It will be exciting to explore other types of effector domains, such as modifiers of DNA methylation, base editors, etc, attached toCascade, Gersbach says.

A further CRISPR development has come from a collaboration between Tufts University in the US and the Chinese Academy of Sciences. These researchers used a biodegradable synthetic lipid nanoparticle to deliver their CRISPR editing tools into the cell to precisely alter the cells genetic code.

According to the team, their method resulted in up to 90 percent efficacy in gene editing. The lipid nanoparticles encapsulate messenger RNA (mRNA) encoding Cas9. Once the contents of the nanoparticles including the sgRNA are released into the cell, the cells protein-making machinery takes over and creates Cas9 from the mRNA template.

A unique feature of the nanoparticles is made of synthetic lipids comprising disulfide bonds in the fatty chain. When the particles enter the cell, the environment within the cell breaks open the disulfide bond to disassemble the nanoparticles and the contents are quickly and efficiently released into the cell.

Once the contents of the nanoparticles are released into the cell, the cells protein-making machinery takes over and creates Cas9

The researchers highlighted that their delivery system refines CRISPR technologies; as Cas9 is a large complex it is difficult to deposit directly into the nucleus of the cell. Other research teams have used viruses, polymers and other kinds of nanoparticles to deliver CRISPR-Cas9, but the low efficiency of transfer limits its success. As their delivery system builds the Cas9 enzyme later in the process, it has high levels of transfer and efficacy.

Professor Qiaobing Xu, a co-corresponding author of the study, highlighted that the synthetic lipid could be made with low-toxicity. Furthermore, he explained, as there is no limit in terms of cargo size, the lipid is an improvement upon viral delivery.

He also emphasised that with viral delivery, there is always a concern about the immune response against the viral particle, but a non-viral delivery method does not have this disadvantage.

These developments in the CRISPR technique indicate how the technology is set to improve and develop in the future. However, research is far from over.

Platt believes that CRISPR processes are still in their infancy, as the current tools are effective at cutting DNA but can result in random repair. He believes that the future of genome editing is going to require new tools to enable more precise changes to the genome.

Eliminating random output would ensure success of the technology for therapeutic effect. Making precise changes is therefore the direction that CRISPR will evolve to, allowing more complex challenges to be tackled.

Gersbach remarks that his teams study will likely stimulate more research into Class 1 systems, which could lead to numerous applications and provide more biological insights into its potential therapeutic use.

Although there is more work to be done with regard to Class 1 CRISPR systems, its unique attributes make it worth investigating, he says.

Xu also comments that CRISPR is a young field compared with other technologies. He highlights the many areas of CRISPR developments: better editors; larger animal or in vitro models; and more precise analytical methods to detect gene editing.

He believes that CRISPR holds tremendous potential to treat disease, which is absolutely ground-breaking. If specific, targeted genes in the body can be controlled, then almost every condition could potentially be treated.

In conclusion, CRISPR can be a highly useful tool for editing genes and to potentially treat complex diseases. However, it still must be refined as a technique. This has caused researchers to strive for improvements in this area, to make the process more precise and effective.

These recent studies demonstrate that improvements are possible and serve to highlight the enormous potential that CRISPR offers.

According to the researchers, CRISPR technologies have progressed and will continue to improve. They all agree that CRISPR could one day be an effective way to treat genetic diseases.

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How will CRISPR change and evolve in the future? - Drug Target Review

"Paired nickases represent a significant step in increasing specificity through a highly flexible and efficient approach to reduce off target effects in gene editing," said Udit Batra, CEO, MilliporeSigma. "MilliporeSigma's technology improves CRISPR's ability to fix diseased genes while not affecting healthy ones, therefore improving the accuracy of potential gene therapy treatments."

These patents cover a foundational CRISPR strategy in which two CRISPR nickases are targeted to a common gene target and work together by nicking or cleaving opposite strands of a chromosomal sequence to create a double-stranded break. This process can optionally include an exogenous or donor sequence for insertion in the same manner as MilliporeSigma's patented CRISPR integration technology. The requirement of two CRISPR binding events greatly reduces the chances of off-target cutting at other locations in the genome.

In addition to Japan and Singapore, MilliporeSigma has CRISPR-related patents in the following regions: Australia, Canada, China, Europe, Israel, South Korea and the U.S. MilliporeSigma was awarded its first foundational patent in Australia covering CRISPR integration in 2017, and its first U.S. CRISPR patent for proxy-CRISPR in 2019.

MilliporeSigma has been at the forefront of innovation in the field for 15 years, with experience spanning from discovery to manufacturing.MilliporeSigma supports research with genome editing under careful consideration of ethical and legal standards. MilliporeSigma's parent company, Merck KGaA, Darmstadt, Germany, established an independent, external Bioethics Advisory Panelto provide guidance for research in which its businesses are involved, including research on or using genome editing. The company has also defined a clear operational position considering scientific and societal issues to inform promising therapeutic approaches for use in research and applications.

Follow MilliporeSigma on Twitter @MilliporeSigma, on Facebook @MilliporeSigma and on LinkedIn.

All Merck KGaA, Darmstadt, Germany news releases are distributed by email at the same time they become available on the EMD Group website. In case you are a resident of the U.S. or Canada please go to http://www.emdgroup.com/subscribe to register again for your online subscription of this service as our newly introduced geo-targeting requires new links in the email. You may later change your selection or discontinue this service.

About the Life Science Business of Merck KGaA, Darmstadt, GermanyThe Life Science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has some 21,000 employees and 59 manufacturing sites worldwide, with a portfolio of more than 300,000 products focused on scientific discovery, biomanufacturing and testing services. Udit Batra is the global chief executive officer of MilliporeSigma.

Merck KGaA, Darmstadt, Germany completed its $17 billion acquisition of Sigma-Aldrich in November 2015, creating a leader in the $125 billion global life science industry.

Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and performance materials. Around 56,000 employees work to make a positive difference to millions of people's lives every day by creating more joyful and sustainable ways to live. From advancing gene-editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices the company is everywhere. In 2018, Merck KGaA, Darmstadt, Germany generated sales of 14.8 billion in 66 countries.

The company holds the global rights to the name and trademark "Merck" internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Performance Materials. Since its founding 1668, scientific exploration and responsible entrepreneurship have been key to the company's technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. For more information about Merck, KGaA, Darmstadt, Germany, visit http://www.emdgroup.com.

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Japan and Singapore Grant CRISPR Patents to MilliporeSigma - PRNewswire

GlobalCRISPR and Cas Genes MarketResearch Report represents an extensive analysis of global CRISPR and Cas Genes industry by delivering evaluation of present forthcoming trends, competitive forces, customers expectations, technological advancements, and working capital in the market. The report also renders a thorough analysis of geographical regions and circumstances, product/service types, Key applications, consumption, revenue, and sales of CRISPR and Cas Genes.

The Research report Delivers a summary of the impact of the key drivers, restraints, and popular trends in the CRISPR and Cas Genes market. [To Know More -Request Sample Report] These factors are studied on regional as well as the global front, for varying levels of depth of market research. Overall review of the factors affecting various decisions in the global market is presented and examined by policies in the market, regulatory scenario of the market, with the help of details of key principles, directions, plans, and strategies in the market. The report includes the detailed analytical account of the markets competitive landscape, with the help of detailed business profiles, SWOT analysis, project feasibility analysis, and several other details about the key companies operating in the CRISPR and Cas Genes market. The report also presents an outline of the impact of recent developments on the markets future growth forecast.

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

Top Companies in Worldwide CRISPR and Cas Genes Market are as follows:-

Addgene Inc, AstraZeneca Plc., Bio-Rad Laboratories Inc, Caribou Biosciences Inc, Cellectis S.A., Cibus Global Ltd, CRISPR Therapeutics AG, Editas Medicine Inc, eGenesis Bio, GE Healthcare, GenScript Corporation And More

Global CRISPR and Cas Genes Market: Segmentation Analysis

Segmentation on the basis of product:

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

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

Biotechnology & Pharmaceutical CompaniesAcademic & Government Research InstitutesContract Research Organizations

Global CRISPR and Cas Genes Market: Regional Analysis

North America

Europe

Asia Pacific

Latin America

Middle East & Africa

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Key questions answered in the CRISPR and Cas Genes Market report:

What are the key market trends impacting the growth of the CRISPR and Cas Genes market?

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Who are the global key manufacturers of CRISPR and Cas Genes Industry: Company Introduction, and Major Types, Sales Market Performance, Product Specification, Contact Information, Production Market Performance.

What are the Product types and applications of CRISPR and Cas Genes?

What are the upstream raw materials and manufacturing equipment of CRISPR and Cas Genes? UpStream Industries Analysis, Equipment, and Suppliers, Raw Material and Suppliers, Manufacturing Analysis, Manufacturing Plants Distribution Analysis, Manufacturing Cost Structure, Manufacturing Process, Industry Chain Structure Analysis.

What is the global (North America, Africa, South America, Asia, China, Europe, Middle East, Japan) production, consumption, consumption value, production value, import and export of CRISPR and Cas Genes?

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New Research: CRISPR and Cas Genes Market Trends And Top Key Companies Profile || [Addgene Inc, AstraZeneca Plc., Bio-Rad Laboratories Inc] - Industry...

MarketResearchNest.com adds Global CRISPR Market Report 2019 Market Size, Share, Price, Trend and Forecast new report to its research database. The report spread across 102 with table and figures in it.

The global market size of CRISPR is $- million in 2018 with CAGR from 2014 to 2018, and it is expected to reach $- million by the end of 2024 with a CAGR of -% from 2019 to 2024.

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

This report studies the CRISPR Market with many aspects of the industry like the market size, market status, market trends and forecast, the report also provides brief information of the competitors and the specific growth opportunities with key market drivers. Find the complete CRISPR market analysis segmented by companies, region, type and applications in the report.

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The key insights of the report:

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

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

At least 9 companies are included:

For complete companies list, please ask for sample pages.

The information for each competitor includes:

For product type segment, this report listed main product type of CRISPR market

For end use/application segment, this report focuses on the status and outlook for key applications. End users are also listed.

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For geography segment, regional supply, application-wise and type-wise demand, major players, price is presented from 2013 to 2023. This report covers following regions:

The key countries in each region are taken into consideration as well, such as United States, China, Japan, India, Korea, ASEAN, Germany, France, UK, Italy, Spain, CIS, and Brazil etc.

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Growth of CRISPR Market in Global Industry: Overview, Size and Share 2019-2024 - Markets Gazette 24

Rendered Cas9

Results from clinical trials released Tuesday indicate that two patients, one with beta thalassemia and one with sickle cell disease, have potentially been cured of their diseases. The two trials, which involved using Crispr to edit the genes of the patients in question, were jointly conducted by Vertex Pharmaceuticals and CRISPR Therapeutics.

This is the first clinical evidence to demonstrate that Crispr/Cas9 can be used to cure or potentially cure serious genetic illnesses, Jeffery Leiden, CEO of Vertex, told Forbes. It's a remarkable scientific and medical milestone.

Vertex Pharmaceuticals CEO Jeffery Leiden

Crispr/Cas9 is a gene-editing system popular for its ability to snip, repair or insert genes into DNA. The therapies tested in the clinical trials work by extracting bone marrow stem cells from the patients, editing these stem cells to fix the genetic mutations that cause the diseases, and then infusing the cells back into the patients. The patients body then takes over and is able to produce new, healthy cells. Engineering of the cells is done ex vivo (outside of the patients body). This allows the researchers to make sure the correct changes are made and there are no improper edits to the genome.

CTX001, the gene-editing therapy used in these trials, is very surgical in how it makes the change, says David Altshuler, Vertexs chief scientific officer.

It has been nine months since the patient with beta thalassemia received the one-time-only treatment and over four months for the patient with sickle cell disease. In that time, both of their conditions have improved tremendously, Leiden says. The patient with beta thalassemia, who used to undergo more than 16 blood transfusions each year, hasnt needed an infusion since the treatment. The patient with sickle cell disease experienced an average ofseven excruciating health crises per year before the treatment, and since the treatment hasnt experienced any.

Despite the fact that these results have only been seen in two patients, says Samarth Kulkarni, CEO of CRISPR Therapeutics, the effect is so dramatic in these patients that we cant help but think this brings a lot of promise.

CRISPR Therapeutics CEO Sam Kulkarni

Both patients suffered side effects during the treatment, but doctors concluded they were caused by the bone marrow preparation, not the Crispr treatment itself. In order to infuse healthy stem cells, both patients had to undergo intensive chemotherapy to destroy their old bone marrow cells. This treatment, also common for bone cancer patients, can cause nausea, hair loss and organ damage.

Precision medicine is known for its hefty price tag, and this treatment is the zenith of precision medicine, Kulkarni says. Yet when asked about potential cost of the treatment, Kulkarni says that they are still focusing on clinical development and it is too early to contemplate any sort of pricing discussions." Zolgensma, the first FDA approved gene-therapy medication, was priced at $2.1 million last May.

The applications of Crispr seem limitless, but the field has encountered several ethical controversies. Last year, Chinese scientist He Jiankui shocked the medical community by announcing that he had altered the genes of two human children. One of the main worries that researchers have about Crispr is that scientists might alter genes to be inherited, a practice called germline engineering. In a recent article on the anniversary of Hes revelation, Crispr pioneer Jennifer Doudna called for stricter regulations for using Crispr in heritable human genome editing.

But germline editing isnt a concern in these trials, where only somatic, or non-reproductive cells, were altered. People are much more concerned about intentional changes to a persons DNA that could be passed down to their descendants, says Henry Greely, a Stanford law professor and chairman of the California Advisory Committee on Human Stem Cell Research. When it comes to somatic cells, they die with the person," he says.

In addition to following these initial patients for the next two years to see if their diseases reoccur, Leiden says theyre enrolling multiple patients with both diseases for the next phase of the clinical trial and will be starting treatments for those patients in the near future. While they dont yet have a timeline on when the treatment will be commercially available, we want to get this to patients as soon as possible, he says.

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New Data From First Human Crispr Trials Shows Promising Results - Forbes

A University of California professor and co-originator of genome editing technology Clustered Regularly Interspaced Short Palindromic Repeats said researchers plan to expand the technology in order to increase human applications at a campus lecture series Thursday.

Jennifer Doudna, a UC Berkeley biochemistry professor, engaged students and the greater UCLA science community during the quarterly Donald J. Cram Distinguished Lecture series.

The Cram lecture series, a quarterly departmental event, invites prominent academics in the field of chemistry to speak about their research. The series is dedicated to Donald J. Cram, who was a Nobel laureate and a chemistry professor at UCLA for over 50 years.

This fall, the series was hosted by UCLA chemistry professor and Cram Chair Patrick Harran.

Scientists use CRISPR technology, formally known as CRISPR-Cas9, to modify DNA sequences and gene functions. Cas9 is a protein that can cut the strands of DNA-like molecular scissors.

CRISPR is studied and used by students, scientists and researchers to advance progress in the field of gene editing, in medicine and the life sciences.

The UC holds the largest CRISPR patent portfolio in the nation with 16 total patents, according to a UC Berkeley press release.

The United States Patent and Trademark Office granted the UC, along with the University of Vienna and Emmanuelle Charpentier, the director of the Max Planck Institute for Infection Biology, its 16th patent in October.

Doudnas involvement in CRISPR technology began around 2005, when a professor at UC Berkeley, Jill Banfield, invited Doudna to help her with research into the mechanism. From there, Doudna teamed up with Charpentier, who was working with a CRISPR system and its associated protein, Cas9, in 2011.

Doudna is one of the creators of the CRISPR utility for the permanent excision of harmful genes. Doudna said that she developed the idea for the CRISPR technology in 2011 in collaboration with Charpentier.

During the lecture, Doudna detailed how scientists regulate CRISPR enzymes to modify DNA.

CRISPR is a portion of the bacterial genomic sequence that acts as an adaptive immune system, Doudna said.

Bacteria encode the CRISPR system through viral infections, which allows its genome to recognize foreign DNA insertions. These DNA sequences incorporate themselves into the bacterial genome at the CRISPR locus, a genetic database of past infections.

Doudna said this locus was of unique interest to her.

Those sequences, called CRISPR, are transcribed in RNA molecules that provide the zip codes for Cas proteins, allowing them to recognize foreign DNA and cut it up, Doudna said.

Doudna and Charpentier, with the assistance of their team, were able to realize that CRISPR RNA is a 20-nucleotide sequence, which interacts with DNA in a complementary fashion.

This complementarity allows the protein to form a double-stranded break in DNA, necessitating a second RNA tracrRNA to form this functional unit, Doudna said.

And it was (biochemist) Martin Jinek in our lab who figured out that you could combine these two RNAs into a single guide RNA, Doudna said.

From this experiment, Jinek found that single guide RNAs were used by Cas9 to excise DNA at specific sites in a plasmid, a circular piece of bacterial DNA. The revelation from this was that, upon excision, DNA would repair itself in animals and plants, Doudna said.

Doudna said at the end of her talk that the system is becoming increasingly important in the field of medicine, and is currently being used at UCLA, by Donald Kohn, a professor of microbiology, immunology and molecular genetics.

Were within about five years, maybe less, from being able to make, essentially, any change to any genome in any type of cell, Doudna said.

Doudna stressed that this ability to make changes in the genome comes with bioethical responsibility for genome editing in humans.

Fourth-year biochemistry student Jeremy Shek, who attended the event, said although he had done a project that was an offshoot of CRISPR, he had not heard of the progress Doudna discussed.

It is important to be informed on advancements and progress in the field, he added.

Fourth-year bioengineering student Timothy Yu said he came to the lecture to see Doudna in person and get a more solid grasp on the methodology of CRISPR.

Lexi Omholt, a fourth-year microbiology, immunology and molecular genetics student, said that she came to the talk to understand the basis of CRISPR technology.

Jennifer Doudna was one of the reasons I chose my major, Omholt said. At that time, CRISPR came into popular knowledge, and the knockout tool was just coming into use. I am involved in a cancer lab, the Soragni Lab, that uses CRISPR-Cas9 on a regular basis.

Read more from the original source:
Co-creator of CRISPR lectures about future applications of genome editing technology - Daily Bruin

18 November 2019

Researchers claim thatCRISPR could be used to detect dangerous viruses in a drop of blood within an hour.

The team at Case Western Reserve University in Cleveland, Ohio, harnessed CRISPR's precision to identify and quantify viruses in human samples, including serum from blood. Their technique, named E-CRISPR, could offer a robust, accurate and cost-effective way to enable faster diagnoses of infections such as parvovirus which can cause miscarriages and human papillomavirus (HPV) which is associated with some cancers.

'This could someday become a simple, accurate and cost-effective point-of-care device for identifying different nucleic acid viruses, such as HPV or parvo from a single droplet of a blood sample,' said first author Yifan Dai, a PhD candidate at Case Western. 'And it would also be extremely fast.'

HPV and parvovirus are both DNA viruses, and their genomic material will be present in the blood of an infected person. E-CRISPR uses a CRISPR RNA strand to bind target sequences which are unique to the virus, and when attached, the associated Cas12a (also known as Cpf1) enzyme cuts the viral DNA in the same way as it would in CRISPR/Cas9 genome editing.

However, unlike Cas9, Cas12a is known to indiscriminately cut single-stranded DNA (ssDNA) once activated by the double-stranded target in this case the viral sequence. The researchers usedssDNA strands, tethered at one end to a sensor and with an electrochemical tag molecule at the other to detect if this cut has been made. If Cas12a is activated, these ssDNA strands are cut, detaching the tag molecules and the electrochemical current detectable through the sensor drops, giving an observable result.

'The CRISPR technique works so that it cuts all of the non-specified single-strand DNA around it once the target is recognised, so we program to electrochemically probe this activity,' said Dai. 'No virus no cutting, it's that simple. And the opposite is true: If CRISPR starts to cut, we know the virus is present.'

The researchers hope that this method could be further developed to create a new 'universal biosensing' device able to accurately detect viruses quickly at the point-of-care, similar to existing blood-glucose sensors. Currently, systems that detect HPV and parvovirus take days to process.

The study was published in Angewandte Chemie, a journal of the German Chemical Society.

Read the original here:
E-CRISPR could be used to rapidly detect viruses - BioNews

-Two patients treated with CTX001 successfully engrafted and demonstrated an initial safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant-

-Beta thalassemia: Patient is transfusion independent with total hemoglobin level of 11.9 g/dL and 10.1 g/dL fetal hemoglobin at nine months after CTX001 infusion-

-Sickle cell disease: Patient is free of vaso-occlusive crises with total hemoglobin level of 11.3 g/dL and 46.6% fetal hemoglobin at four months after CTX001 infusion-

-CRISPR Therapeutics will host a conference call today at 8:00 a.m. ET to review these data-

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 19, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. One patient with transfusion-dependent beta thalassemia (TDT) received CTX001 in the first quarter of 2019 and data for this patient reflect nine months of safety and efficacy follow-up. One patient with severe sickle cell disease (SCD) received CTX001 in mid-2019 and data for this patient reflect four months of safety and efficacy follow-up. These studies are ongoing and patients will be followed for approximately two years following infusion. Several additional patients have been enrolled and have had drug product manufactured across the two studies.

Transfusion-Dependent Beta Thalassemia The patient with TDT has the 0/IVS-I-110 genotype and required 16.5 transfusions per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. Two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells (erythrocytes expressing fetal hemoglobin).

Sickle Cell Disease The patient with SCD experienced seven vaso-occlusive crises (VOCs) per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. Three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain, all of which resolved. At four months after CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells (erythrocytes expressing fetal hemoglobin).

We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.

The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia, said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. While the data are exciting, we are still in the early phase of this clinical program. We look forward to continuing to work with physicians, patients, caregivers and families over the coming months and years to bring forward the best possible therapy for these two serious diseases and to continue to accelerate our gene-editing programs for other serious diseases such as Duchenne muscular dystrophy and myotonic dystrophy type 1.

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

About the Phase 1/2 Study in Sickle Cell DiseaseThe ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study. Enrollment is ongoing at 12 clinical trial sites in the United States, Canada and Europe.

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

CRISPR Therapeutics Conference Call and WebcastCRISPR Therapeutics will host a conference call and webcast today at8:00 a.m. ET. The webcast and presentation will be made available on the CRISPR Therapeutics website at https://crisprtx.gcs-web.com/events in the Investors section under Events and Presentations. Following the live audio webcast, a replay will be available on the Company's website for approximately 30 days.

Dial-In InformationLive (U.S. / Canada): (800) 895-3361Live (International): (785) 424-1062Conference ID: 87198237

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

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

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

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

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

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

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

(VRTX-GEN)

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

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

CRISPR Therapeutics Media Contact:Jennifer PaganelliWCG on behalf of CRISPR+1 347-658-8290jpaganelli@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orLeah Gibson, +1 617-961-1507

Media: mediainfo@vrtx.com orNorth America:Heather Nichols, +1 617-341-6992Heather_Nichols@vrtx.com

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CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9...

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The S&P 500 and the Dow Jones indexes retreated from record levels on Tuesday as dour forecasts from Home Depot and Kohl's eroded confidence that the U.S. consumer will support the economy. .N

At 12:23 p.m. ET, the Dow Jones Industrial Average .DJI was down 0.28% at 27,957.31. The S&P 500 .SPX was unchanged at 3,121.96 and the Nasdaq Composite .IXIC was up 0.34% at 8,578.962. The top three S&P 500 .PG.INX percentage gainers: ** Broadcom Inc AVGO.O, up 3 % ** Constellation Brands Inc STZ.N, up 2.5 % ** Biogen Inc BIIB.O, up 2.3 % The top three S&P 500 .PL.INX percentage losers: ** Kohl's Corp KSS.N, down 18.1 % ** Macy's Inc M.N, down 9.6 % ** Home Depot Inc HD.N, down 5.2 % The top three NYSE .PG.N percentage gainers: ** Myovant Sciences Ltd MYOV.N, up 84.8 % ** JinkoSolar Holding Co Ltd JKS.N, up 13 % ** 58.Com Inc WUBA.N, up 12.8 % The top three NYSE .PL.N percentage losers: ** Intelsat SA I.N, down 21.8 % ** Kohl's Corp KSS.N, down 18.1 % ** AeroCentury Corp ACY.N, down 12.9 % The top three Nasdaq .PG.O percentage gainers: ** SAExploration SAEX.O, up 78.5 % ** Karuna Therapeutics Inc KRTX.O, up 39.2 % ** Athenex Inc ATNX.O, up 33.2 % The top three Nasdaq .PL.O percentage losers: ** KLX Energy Services Holdings Inc KLXE.O, down 25.3 % ** Pioneer Power Solutions Inc PPSI.O, down 22.3 % ** My Size Inc MYSZ.O, down 19.8 % ** Home Depot Inc HD.N: down 5.2% ** Kohl's Corp KSS.N: down 18.1% ** Macy's Inc M.N: down 9.6% ** Nordstrom Inc JWN.N: down 5.2% ** Lowe's Companies Inc LOW.N: down 0.8% BUZZ-Home Depot: Falls after FY sales forecast cut, drags Lowe's BUZZ-Kohl's: Falls on FY profit forecast cut, Q3 same-store sales miss BUZZ-Retail stocks tumble on Home Depot, Kohl's dour forecasts

** CRISPR Therapeutics AG CRSP.O: up 17.9% ** Vertex Pharmaceuticals Inc VRTX.O: up 2.0% ** Editas Medicine Inc EDIT.O: up 11.2% ** Intellia Therapeutics Inc NTLA.O: up 12.3% ** Sangamo Therapeutics Inc SGMO.O: up 4.8%

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BUZZ-AT&T Inc: Slips as Moffett Nathason doubts 2020 revenue target achievability ** Personalis Inc PSNL.O: up 2.6%

BUZZ-Personalis gains on tie-up with Germany's Merck over cancer therapies ** TJX Companies Inc TJX.N: up 2.3%

BUZZ-TJX a bright spot in gloomy day for retail ** CNS Pharmaceuticals Inc CNSP.O: up 7.5%

BUZZ-CNS Pharma up on acquiring rights to Berubicin for brain cancer treatment ** Progyny Inc PGNY.O: up 7.8%

BUZZ-Fertility benefits manager Progyny blooms as IPO banks bullish ** ConocoPhillips COP.N: up 1.1%

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The 11 major S&P 500 sectors:

Communication Services

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Energy

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Financial

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up 0.49%

Industrial

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up 0.37%

Materials

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down 0.33%

Real Estate

.SPLRCR

up 0.09%

Utilities

.SPLRCU

down 0.09%

(Compiled by Akanksha Rana in Bengaluru)

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BUZZ-U.S. STOCKS ON THE MOVE-Retail stocks, CRISPR Therapeutics, Slack Technologies - Nasdaq

Imagine if you had a technology that could give you superpowers. No, we are not talking about gamma rays or radioactive spiders but something pretty similar. If youve watched Marvels Luke Cage series, you may have heard of a gene-editing tool called CRISPR. The protagonist gets super strength and unbreakable skin when a mollusc DNA was fused into his DNA.

As bizarre as it may sound, CRISPR-Cas9 (the shorter and more colloquial name is CRISPR) is a real thing and it can selectively cut and paste DNA pieces from and to your DNA. Though the plan for this tool does not include creating super soldiers. Instead, scientists believe that CRISPR-Cas9 could be a ray of hope for those suffering from inherited genetic disorders like down syndrome, cystic fibrosis and thalassemia.

Representational image. Image source: Getty Images.

The technology isnt just theoretical anymore. A study, to be presented next month at a meeting of the American Society of Hematology, conducted on three cancer patients to test the safety of the tool has shown promising results. And it isnt the only one - more studies are recruiting and being conducted to bring gene editing to life.

CRISPR (pronounced Crisper) is an acronym for Clusters of Regularly Interspaced Short Palindromic Repeats. The Cas9 stands for a CRISPR-associated protein 9.

Nature has quipped bacteria with an ability to protect itself from invading viruses. When a virus attacks a bacterium, viral DNA gets embedded in the bacterium's DNA. This creates a new element within the bacterial DNA called CRISPR. If the same virus then attacks the bacterium again, it uses CRISPR to identify the virus.

CRISPR does this by creating RNA - a molecule that carries messages out from the DNA to the rest of the cell. The RNA is the one that identifies the viral DNA invading the cell (or bacteria).

Then, the Cas9 present in the bacteria chops off the invading virus' DNA, thus neutralising the threat.

The new gene-editing tool for humans is based on this same concept. This innovative tool was, rather unimaginatively, then named CRISPR-Cas9.

The gene-editing tool CRISPR-Cas9 is a bit different from its bacterial version. It contains a small chain of RNA along with Cas9, instead of DNA. This cuts out one step of the bacterial process.

CRISPR-Cas9 could be really helpful for those suffering from genetic diseases. A genetic disease happens due to glitches in the DNA sequence. They usually begin inside the fetus when all your body cells are still dividing. Every time a cell divides, its DNA divides along with it to send a copy of the genome into the new cell. But, a fault can sometimes happen in the copying process. This can alter the complete genetic code, creating a mutation - which leads to genetic disease.

This is where CRISPR-Cas9 comes in. The RNA in the tool identifies the "error" in the genetic code. It then binds with the faulty part of the DNA. Cas9 then cuts off that faulty piece.

The original version of the CRISPR-Cas9 tool would only find and cut out the error in the DNA. This creates a gap in the DNA code, which needs to be 'repaired'. The original tool couldn't do so, and would thus necessarily leave the repair process up to the cell. The cell would either join the broken ends of the DNA or insert a new piece to fill the void. Clearly, while this gets rid of the anomaly in the cell, what happens to the genetic code afterwards wasn't in the tool's control. This made the outcome uncertain.

With the new version of the CRISPR-Cas9 tool, scientists can now take control of the repair process as well. We can now add an entirely new DNA sequence of our choice in the removed region and not just fix an anomaly but even introduce new traits into the organism. Think of this as a find-replace tool in your word editor, as opposed to just the find(-delete) tool.

Currently, there is no cure for genetic diseases. CRISPR-Cas9 is a promising step forward. Scientists have already used it to treat some diseases in plants and animals.

Health articles in Firstpost are written by myUpchar.com, Indias first and biggest resource for verified medical information. At myUpchar, researchers and journalists work with doctors to bring you information on all things health. For more information, please read our article onHemophilia, a genetic condition which prevents blood from clotting.

Updated Date: Nov 15, 2019 14:21:41 IST

Tags : CRISPR, CRISPR-cas9, Gene Slicing, Gene-Editing, Genetic Diseases, Genetic Disorders, NewsTracker

Here is the original post:
Another step closer to curing cancer and genetic diseases: All you need to know about CRISPR-Cas9 - Firstpost

Researchers strive to address societal health issues through gene editing

In October, three researchers at UC Davis were awarded a $1.5 million grant to fund their project which attempts to demonstrate the effectiveness of gene editing through use of CRISPR, a powerful technology that allows alteration of DNA sequences to change gene function.

This kind of design can help enhance personalized medicine, said R. Holland Cheng, a professor of molecular and cellular biology in the College of Biological Sciences. Specific patients with specific illnesses can be treated in specific ways.

Cheng, along with Kit Lam, a distinguished professor and chair of the Department of Biochemistry and Molecular Medicine in the School of Medicine, and David Segal, a professor in the Department of Biochemistry and Molecular Medicine, were awarded this highly competitive and sought-after grant from the National Institute of Health (NIH).

UC Davis is part of the NIHs Somatic Cell Genome Editing (SCGE) consortium which has awarded grants to 45 other research institutes across the nation so they can begin groundbreaking work on gene editing. Through this consortium, the NIH hopes to find an efficient and safe way to conduct gene editing. Research programs are investigating the best delivery mechanism as well as the most dynamic gene editing tool.

The major problem with gene editing currently is the inability of cells to be edited within a living organism. It has become fairly easy and efficient to edit genes in a cell culture outside of the body but extremely difficult to do the same processes inside the body. Cheng, Lam and Segal are focused on changing this.

The question is how to do it inside of an animal and eventually a human, Lam said.

They are answering this question by utilizing Chengs work in engineering a non-toxic nanoparticle that they hope can transport the gene editing tool CRISPR into the cells of a living organism. Cheng has been able to create a Hepatitis E viral nanoparticle (HEVNP) that when manipulated could be a delivery system for CRISPR. They plan to take this nanoparticle and encase CRISPR inside of it, producing a mechanism for delivery of CRISPR.

The Hepatitis E nanoparticle has the capacity to be a highly efficient way to deliver gene editing to cells in the body due to its unique nature. HEVNP is resistant to the gastric acid environment of the intestines and stomach, enabling it to survive once its entered the body. Given its resistant abilities, HEVNP can be taken orally, making it a useful form of medicine. If able to successfully get HEVNP to the target cells in the body and deploy CRISPR, gene editing abilities could drastically change.

The addition of a cell-type specific targeting ligand to the HEVNP would code the nanoparticle to deliver CRISPR to a specific cell. The abilities of this method to be precise and safe will determine its success.

With five years of funding from the NIH, these three researchers are eager to begin work on this project and see the strides that can be made in gene editing. They have impressive goals for this research, as it has the capacity to reshape medicine.

This will redefine precision medicine as currently there is broad medicine that can cause side effects to people and not be effective, yet by making it specialized it is becoming more precise and effective, Cheng said.

As more effective and safe tools to cure illnesses are being tested and created, the benefits to society could be expansive. With so much potential to help improve the health of society, the NIH is dedicated to coming to new solutions at a quick rate. All programs that received grants will be required to share and utilize the research occurring at other funded programs. The NIH is hoping to eliminate the private nature of research through enforcing the sharing of ideas, as scientists are often constrained by the institutions they work for. It is their hope that by having communication between the programs, positive results will arise faster.

I think this is great because scientists inherently want to work with each other but have real world concerns especially with money, Segal said.

The research results, when groundbreaking, can provide incredible monetary gains and credibility to the institutions that made the discovery. Ultimately, scientists collaborating with one another will serve society as people are able to benefit earlier from this innovative research.

We want the public to know that we are working in their best interest, Segal said.

The NIH grant is competitive and still the third research program to join the consortium at UC Davis. Innovation has never been more prevalent than in this field at UC Davis. With three different programs researching gene editing, UC Davis stands out as a hotspot for this field of research.

Written by: Alma Meckler-Pacheco science@theaggie.org

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UC Davis leads in innovative gene editing research with NIH grants - The Aggie

Crispr Therapeutics AG (NASDAQ:CRSP) Stock analysts at Oppenheimer issued their Q1 2020 EPS estimates for Crispr Therapeutics in a note issued to investors on Tuesday, November 12th. Oppenheimer analyst S. Tuerkcan forecasts that the company will post earnings per share of ($0.87) for the quarter. Oppenheimer has a Outperform rating and a $65.00 price target on the stock. Oppenheimer also issued estimates for Crispr Therapeutics FY2021 earnings at ($4.29) EPS, FY2022 earnings at ($4.70) EPS and FY2023 earnings at ($1.07) EPS.

CRSP has been the subject of a number of other reports. Piper Jaffray Companies restated an overweight rating on shares of Crispr Therapeutics in a research note on Monday, October 21st. BTIG Research upped their target price on Crispr Therapeutics from $51.00 to $59.00 and gave the company a positive rating in a research note on Tuesday, July 30th. Roth Capital upped their target price on Crispr Therapeutics from $50.00 to $65.00 in a research note on Tuesday, July 30th. Canaccord Genuity initiated coverage on Crispr Therapeutics in a research note on Friday, July 26th. They issued a buy rating and a $72.00 target price for the company. Finally, BidaskClub upgraded Crispr Therapeutics from a hold rating to a buy rating in a research note on Friday. Two research analysts have rated the stock with a sell rating, three have given a hold rating and twelve have given a buy rating to the company. The stock has a consensus rating of Buy and an average price target of $57.95.

CRSP opened at $56.87 on Friday. The business has a 50 day moving average price of $43.31 and a 200 day moving average price of $44.57. The company has a debt-to-equity ratio of 0.06, a quick ratio of 8.32 and a current ratio of 8.32. The firm has a market cap of $3.04 billion, a P/E ratio of -16.53 and a beta of 3.15. Crispr Therapeutics has a twelve month low of $22.22 and a twelve month high of $57.40.

Crispr Therapeutics (NASDAQ:CRSP) last announced its quarterly earnings results on Monday, October 28th. The company reported $2.40 EPS for the quarter, beating the Thomson Reuters consensus estimate of ($0.95) by $3.35. Crispr Therapeutics had a negative return on equity of 2.60% and a negative net margin of 5.30%. The business had revenue of $211.93 million during the quarter, compared to analysts expectations of $6.32 million.

A number of large investors have recently made changes to their positions in CRSP. NEXT Financial Group Inc grew its position in Crispr Therapeutics by 915.0% in the third quarter. NEXT Financial Group Inc now owns 609 shares of the companys stock valued at $25,000 after acquiring an additional 549 shares during the period. Benjamin Edwards Inc. grew its position in Crispr Therapeutics by 96.4% in the second quarter. Benjamin Edwards Inc. now owns 546 shares of the companys stock valued at $26,000 after acquiring an additional 268 shares during the period. Coastal Investment Advisors Inc. bought a new stake in Crispr Therapeutics in the third quarter valued at $26,000. US Bancorp DE grew its position in Crispr Therapeutics by 553.7% in the second quarter. US Bancorp DE now owns 621 shares of the companys stock valued at $29,000 after acquiring an additional 526 shares during the period. Finally, BSW Wealth Partners bought a new stake in Crispr Therapeutics in the second quarter valued at $39,000. Hedge funds and other institutional investors own 51.09% of the companys stock.

In other Crispr Therapeutics news, Director Pablo J. Cagnoni sold 7,500 shares of the companys stock in a transaction on Tuesday, November 12th. The stock was sold at an average price of $55.00, for a total value of $412,500.00. Following the sale, the director now directly owns 7,500 shares in the company, valued at $412,500. The sale was disclosed in a document filed with the Securities & Exchange Commission, which is accessible through this hyperlink. Corporate insiders own 21.40% of the companys stock.

Crispr Therapeutics Company Profile

CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9) gene-editing platform in Switzerland. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells.

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Oppenheimer Weighs in on Crispr Therapeutics AG's Q1 2020 Earnings (NASDAQ:CRSP) - DFS Caller

Doctors at the University of Pennsylvania Abramson Cancer Centre say it could hail a new era of potential treatments

A DNA editing tool used to snip defective genes in unborn children is being tested in the United States to fight cancer.

For the first time outside of China, tests on three patients with advanced cancer were conducted to see how effective DNA-snipping tool Crispr is at fighting the disease.

Doctors at the University of Pennsylvania Abramson Cancer Centre used the technology on patients in their 60s whose cancer had progressed despite undergoing regular treatments such as chemotherapy, radiation and surgery.

Its the most complicated genetic, cellular engineering thats been attempted so far, said study leader Dr Edward Stadtmauer, the centres section chief of hematologic malignancies.

This is proof that we can safely do gene editing of these cells.

The technique extracts immune cells from the patients blood and genetically alters them to recognise and fight cancer cells.

Experts said early tests proved to be safe, and that a breakthrough could hail a new era of potential cancer treatments.

Two of the patients had blood cancer and the other had a rarer form of sarcoma, cancer of the bone or soft tissue.

Although yet to be published in a peer-reviewed medical journal, the findings will be presented at the American Society of Hematology in December.

Researchers said the exercise at this stage was focused on whether the technology is safe and feasible, rather than improving survival rates. It is too early to say whether the treatment will improve survival rates.

The use of Crispr technology in China to edit the genes of couples experiencing fertility problems has been controversial.

The editing tool alters a defective gene in IVF embryos to eliminate life-limiting or chronic illnesses such as sickle cell disease, which starves the body of oxygen.

Some doctors have criticised the early use of the molecular scissors in fertility clinics as the long term effects are not yet known, with potential damage caused to other genes during the treatment.

Updated: November 17, 2019 11:28 AM

Originally posted here:
US cancer scientists on the verge of gene editing breakthrough to treat cancer - The National

CRISPR, the revolutionary ability to snip out and alter genes with scissor-like precision, has exploded in popularity over the last few years and is generally seen as the standalone wizard of modern gene-editing. However, its not a perfect system, sometimes cutting at the wrong place, not working as intended and leaving scientists scratching their heads. Well, now theres a new, more exacting upgrade to CRISPR called Prime, with the ability to, in theory, snip out more than 90% of all genetic diseases.

Just what is this new method and how does it work? We turned to IEEE fellow, biomedical researcher and dean of graduate education at Tuft Universitys school of engineering Karen Panetta for an explanation.

CRISPR is a powerful genome editor. It utilizes an enzyme called Cas9 that uses an RNA molecule as a guide to navigate to its target DNA. It then edits or modifies the DNA, which can deactivate genes or insert a desired sequence to achieve a behavior. Currently, we are most familiar with the application of genetically modified crops that are resistant to disease.

However, its most promising application is to genetically modify cells to overcome genetic defects or its potential to conquer diseases like cancer.

Some applications of genome editing technology include:

Of course, as with every technology, CRISPR isnt perfect. It works by cutting the double-stranded DNA at precise locations in the genome. When the cells natural repair process takes over, it can cause damage or, in the case where the modified DNA is inserted at the cut site, it can create unwanted off-target mutations.

Some genetic disorders are known to mutate specific DNA bases, so having the ability to edit these bases would be enormously beneficial in terms of overcoming many genetic disorders. However, CRISPR is not well suited for intentionally introducing specific DNA bases, the As, Cs, Ts and Gs that make up the double helix.

Prime editing was intended to overcome this disadvantage, as well as other limitations of CRISPR.

Prime editing can do multi-letter base-editing, which could tackle fatal genetic disorders such as Tay-Sachs, which is caused by a mutation of four DNA letters.

Its also more precise. I view this as analogous to the precision lasers brought to surgery versus using a hand-held scalpel. It minimized damage, so the healing process was more efficient.

Prime editing can insert, modify or delete individual DNA letters; it also can insert a sequence of multiple letters into a genome with minimal damage to DNA strands.

Imagine being able to prevent cancer and/or hereditary diseases, like breast cancer, from ever occurring by editing out the genes that are makers for cancer. Cancer treatments are usually long, debilitating processes that physically and emotionally drain patients. It also devastates patients loved ones who must endure watching helpless on the sidelines as the patient battles to survive.

Editing out genetic disorders and/or hereditary diseases to prevent them from ever coming to fruition could also have an enormous impact on reducing the costs of healthcare, effectively helping redefine methods of medical treatment.

It could change lives so that long-term disability care for diseases like Alzheimers and special needs education costs could be significantly reduced or never needed.

How did the scientific community get to this point where did CRISPR/prime editing come from?

Scientists recognized CRISPRs ability to prevent bacteria from infecting more cells and the natural repair mechanism that it initiates after damage occurs, thus having the capacity to halt bacterial infections via genome editing. Essentially, it showed adaptive immunity capabilities.

Its already out there! It has been used for treating sickle-cell anemia and in human embryos to prevent HIV infections from being transmitted to offspring of HIV parents.

IEEE engineers, like myself, are always seeking to take the fundamental science and expand it beyond the petri dish to benefit humanity.

In the short term, I think that Prime editing will help generate the type of fetal like cells that are needed to help patients recover and heal as well as developing new vaccines against deadly diseases. It will also allow researchers new, lower cost alternatives and access to Alzheimers like cells without obtaining them post-mortem.

Also, AI and deep learning is modeled after human neural networks, so the process of genome editing could potentially help inform and influence new computer algorithms for self-diagnosis and repair, which will become an important aspect of future autonomous systems.

Continued here:
Youve heard of CRISPR, now meet its newer, savvier cousin CRISPR Prime - TechCrunch

PHOTO: BARBARA RIES FOR UCSF

There are key moments in the history of every disruptive technology that can make or break its public perception and acceptance. For CRISPR-based genome editing, such a moment occurred 1 year agoan unsettling push into an era that will test how society decides to use this revolutionary technology.

In November 2018, at the Second International Summit on Human Genome Editing in Hong Kong, scientist He Jiankui announced that he had broken the basic medical mantra of do no harm by using CRISPR-Cas9 to edit the genomes of two human embryos in the hope of protecting the twin girls from HIV. His risky and medically unnecessary work stunned the world and defied prior calls by my colleagues and me, and by the U.S. National Academies of Sciences and of Medicine, for an effective moratorium on human germline editing. It was a shocking reminder of the scientific and ethical challenges raised by this powerful technology. Once the details of He's work were revealed, it became clear that although human embryo editing is relatively easy to achieve, it is difficult to do well and with responsibility for lifelong health outcomes.

It is encouraging that scientists around the globe responded by opening a deeper public conversation about how to establish stronger safeguards and build a viable path toward transparency and responsible use of CRISPR technology. In the year since He's announcement, some scientists have called for a global but temporary moratorium on heritable human genome editing. However, I believe that moratoria are no longer strong enough countermeasures and instead, stakeholders must engage in thoughtfully crafting regulations of the technology without stifling it. In this vein, the World Health Organization (WHO) is pushing government regulators to engage, lead, and act. In July, WHO issued a statement requesting that regulatory agencies in all countries disallow any human germline editing experiments in the clinic and in August, announced the first steps in establishing a registry for future such studies. These directives from a global health authority now make it difficult for anyone to claim that they did not know or were somehow operating within published guidelines. On the heels of WHO, an International Commission on the Clinical Use of Human Germline Genome Editing convened its first meeting to identify the scientific, medical, and ethical requirements to consider when assessing potential clinical applications of human germline genome editing. The U.S. National Academy of Medicine, the U.S. National Academy of Sciences, and the Royal Society of the United Kingdom lead this commission, with the participation of science and medical academies from around the world. Already this week, the commission held a follow-up meeting, reflecting the urgent nature of their mission.

Where is CRISPR technology headed? Since 2012, it has transformed basic research, drug development, diagnostics, agriculture, and synthetic biology. Future CRISPR-based discoveries will depend on increased knowledge of genomes and safe and effective methods of CRISPR delivery into cells. There needs to be more discussion about prioritizing where the technology will have the most impact as well as equitable, affordable access to its products. As for medical breakthroughs, clinical trials using CRISPR are already underway for patients with cancer, sickle cell disease, and eye diseases. These and many other future uses of genome editing will involve somatic changes in individuals, not heritable changes that are transmissible. But the rapidly advancing genome editing toolbox will soon make it possible to introduce virtually any change to any genome with precision, and the temptation to tinker with the human germ line is not going away.

The CRISPR babies saga should motivate active discussion and debate about human germline editing. With a new such study under consideration in Russia, appropriate regulation is urgently needed. Consequences for defying established restrictions should include, at a minimum, loss of funding and publication privileges. Ensuring responsible use of genome editing will enable CRISPR technology to improve the well-being of millions of people and fulfill its revolutionary potential.

* J.D. is a cofounder of Caribou Biosciences, Editas Medicine, Scribe Therapeutics, and Mammoth Biosciences; scientific advisory board member of Caribou Biosciences, Intellia Therapeutics, eFFECTOR Therapeutics, Scribe Therapeutics, Mammoth Biosciences, Synthego, and Inari; and director at Johnson & Johnson. Her lab has research projects sponsored by Biogen and Pfizer.

The rest is here:
CRISPR's unwanted anniversary - Science Magazine

Susanna M. Hamilton/Broad Communications

Susanna M. Hamilton/Broad Communications

It's not easy to treat viral infections. Just ask anyone with a bad cold or a case of the flu.

But scientists in Massachusetts think they may have a new way to stop viruses from making people sick by using what amounts to a pair of molecular scissors, known as CRISPR.

It's a gene editing tool based on a molecule that occurs naturally in microorganisms.

CRISPR comes in many "flavors" that perform a variety of functions inside cells. The Cas9 flavor has been widely used as a tool for editing DNA inside cells. It's already shown promise for medical therapies such as treating sickle cell disease.

What's different is that the antiviral approach researchers at the Broad Institute in Cambridge are using involves a form of CRISPR called Cas13 that targets specific regions of RNA, not DNA.

RNA is a chemical cousin of DNA. Many viruses, including flu and Zika, package their genetic instructions in RNA instead of DNA.

When a virus infects a cell in our bodies, it hijacks the cell's molecular machinery to make copies of itself. Those new viruses can go on to spread the infection through your body.

So for therapy, "we need to be able to cut the virus at a fast enough rate to slow down replication or to stop replication from happening," says Cameron Myhrvold, a postdoc at the Broad Institute.

Finding the right target is key. There's a lot of RNA inside cells that is necessary for the cell to survive, so it's important to find an RNA target that's unique to the virus you're trying to control.

Myhrvold says RNA viruses are particularly difficult to control because they are a bit like shape-shifters: They tend to change their genetic sequences when you try to pin them down. That's one of the reasons people need a new flu vaccine each year.

Understanding how the virus changes in response to Cas13 treatment should be informative.

"That could potentially teach us about what parts of the virus are particularly important for its function," says Catherine Freije, a doctoral student at the Broad Institute. And that in turn will show the best places to target the virus in order to disable it.

So far, Freije and Myhrvold say they've only shown their antiviral treatment works in cells.

But Pardis Sabeti, head of the lab they work in, is bullish about using the CRISPR Cas13 system to treat viral infections in people.

"There's still a bunch of things we want to work out, but we feel pretty confident that this will work as a therapy if it can be delivered in the right way," Sabeti says.

By delivering, she means getting the CRISPR Cas13 tool into the right cells inside an infected patient.

Since CRSIPR Cas13 specifically targets RNA, it will only be useful for illnesses caused by RNA.

Janice Chen says researchers are now finding a variety of CRISPRs with different properties. Chen is chief research officer at Mammoth Biosciences, a company that hopes to capitalize on CRISPR technology.

"Having a broader CRISPR toolbox is really important to figure out what is the specific need for any given application," Chen says.

Progress in building that toolbox has proceeded quite quickly. After all, it's only been six years since scientists first became aware of how powerful a tool CRISPR could be.

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CRISPR Could Stop Replication Of Viruses That Cause Illness, Researchers Say : Shots - Health News - NPR

These three health and technology stocks are on the move and pointing higher.

Advanced Micro Devices Inc. AMD, +0.55% jumped 81 cents to $37.52 on 67 million shares traded Wednesday. On Tuesday, the chip maker announced Tencent TCEHY, +0.20% will use its latest server processors. The stock has been in a steep rising channel since its recent low below $28 in early October. Next target is the rising channel top near $40.

Amarin Corp. PLC AMRN, +11.77% followed through on Wednesday, up 55 cents to $21.49 after popping 23% on Tuesday. An FDA advisory committee is scheduled to meet today to help decide the fate of the companys fish-derived cardiovascular drug. While trading has been halted this morning in advance of the meeting, the chart points to a test of the July high in the $23.50-$24.00 zone next.

Crispr Therapeutics AG CRSP, +3.29% rose $1.42 to $55 on 1 million shares Wednesday. The move, on no news from the company, continued the gene-editings stocks month-long rally from around $36. It also followed through on Tuesdays breakout of a mini-wedge. Watch for a move to $58 next.

See Harrys video-chart analysis on these stocks.

The writer has no holdings in any securities mentioned.

Harry Boxer is founder of TheTechTrader.com, a live trading room featuring his stock picks, technical market analysis and live chart presentations.

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AMD, Amarin and Crispr are three stocks to watch for potentially higher prices - MarketWatch

In Europe, we dont do things the way the Americans do

Oral proceedings in the opposition against UC Berkeleys (UCs) main European patent EP2800811 are scheduled for all of three (!) days in February of 2020 at the European Patent Office (EPO). The opposition divisions (ODs) preliminary and non-binding opinion, provided on the 30th of August 2019 in preparation for the hearing, is favorable to the patentee. In its opinion, the OD sides with UC Berkeley and dismisses the main arguments of the seven opponents. Arguments relating to minor issues of added subject matter have been accepted by the OD, however UC is likely to be able to overcome these. Thus, there is a chance that UC Berkeley will keep their strong hold on rights to general platform Crispr technology in Europe.

UC Berkeleys patent claims priority from four provisional US applications. The question of whether the priority from the first provisional application P1 is valid or not lies at heart of the case.

According to European practice, G2/98, the requirements of claiming priority of the same invention in the meaning of Art 87(1) EPC mean that priority can only be acknowledged if the skilled person can derive the subject matter directly and unambiquously, using common general knowledge (CGK), from the previous application as a whole. In addition, the priority document must provide an enabling disclosure, in other words, all essential elements needed to carry out the invention must be disclosed in the priority document.

The opponents argue that P1 fails to provide an enabling disclosure, as it does not disclose or exemplify elements which are essential for the workability of the Crispr-Cas9 system in eukaryotic cells. At the heart of this issue is the protospacer adjacent motif (PAM), a 2-6 base pair DNA sequence which immediately follows the target DNA sequence and is an essential targeting component. The opponents argue that without knowledge of a PAM sequence, a person skilled in the art was not in a position to design an appropriate guide RNA and would therefore not have been able to achieve cleavage of target DNA (as the Cas9 endonuclease will not recognize target DNA without the PAM).

UC replies that the requirement of a PAM to be located downstream of the target DNA sequence was CGK at the date of filing of P1 and therefore the omission of any reference to PAM is of no detriment to the disclosure of P1. UC states that the skilled persons understanding of this was confirmed by the sequences disclosed in P1, wherein the amino acid sequences corresponding to PAMs are present immediately downstream of the target DNA.

If the priority claim from P1 is found to be invalid, the effective date of the patent at hand would be after UCs scientific paper on Crispr-Cas9 was published in Science. This would be detrimental to the patentability of at least some of the claims.

However, UC was successful during the examination before the EPO, as well as in the corresponding UK cases, in arguing that PAM was part of CGK. By accepting in their preliminary opinion that PAM was part of the CGK at the time of filing of P1, the OD has provisionally concluded that the disclosure of P1 is enabling over the whole claim scope, encompassing eukaryotic applications.

Concerning what actually was CGK at the time, UC argues that CGK was represented by review and research articles in the fast-evolving new technology area. UC holds that such articles confirm that the requirement for PAM in the target DNA was CGK. Although full of references to CGK and the skilled person, the preliminary opinion does not dwell on the identity of the skilled person. Establishing the identity of the skilled person is likely to be important during the oral proceedings. Not limited to PAM, the opponents argue that several lines of technical information are missing in P1 and that a skilled person operating within the limits of what is explicitly defined in P1 would be confronted with an inacceptable degree of failure.

The OD has also come to the preliminary view that the claims are novel and exhibit inventive step. The inventive step analysis is based on the problem-solution approach starting from a prior art document from the TALEN field of gene editing, and not from the Crispr-field, based on the purpose of the UC invention. The technical problem solved by the invention is considered to be to provide a more versatile gene editing system. The OD adds that the Examples in the patent show that UCs invention achieves this, or at least renders the achievement credible, also for eukaryotic cells.

UCs position is that P1 not only claimed a new class of endonucleases, but also provided ample guidance on how to use the endonuclease complex for example in eukaryotic systems as, amongst other things, P1 disclosed expression systems including vectors suitable for eukaryotic expression. They point to the fact that several groups in the scientific community quickly, upon publication of the Science paper, confirmed that the Crisp-Cas9 system could be used for gene editing in eukaryotic cells.

Interestingly, the OD takes no notice of UC inventors Doudnas and Charpentiers public statements, made upon publishing of the Science paper, about unpredictability and technical challenges of adapting the Crispr-Cas9 system to eukaryotic gene editing. In fact, the OD underlines the difference between the question of obviousness in the US interference proceedings by exclaiming under US law! in the opinion and the question of plausibility in the present case. Although plausibility is not a term used in the European Patent Convention, it is increasingly more discussed. According to case law, however, the question of plausibility only comes into play if experimental data is lacking. The OD states in its opinion that this does not apply to present case, because the disclosure does contain experimental data.

Nevertheless, plausibility, as well as the identity of the skilled person, are likely to be discussed during the oral proceedings. Is the skilled person going to be someone from the TALEN field? If so, would they be expected to know all the details and nuances of the Crispr-Cas9 field or not? The identity of the skilled person may have implications on several aspects of the case.

For now, it appears that the differences between the European and US patent landscape in the Crispr-Cas9 field may remain, at least as indicated by the non-binding and preliminary opinion of the OD.

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The Crispr-Cas9 patent tussle continues: The case of UC Berkeley at the EPO - Lexology

Over the last few years, thousands of studies have employed CRISPR/Cas systems to edit, or transcriptionally regulate, individual genetic targets. But a new study has taken CRISPR to soaring heights.

CRISPR/Cas is remarkably simple in principle: a protein, usually Cas9, can bind to an RNA molecule, such as a single guide RNA (sgRNA), which has a sequence complementary to a target site in the genome. When the Cas9:sgRNA complex binds to its target site, it cleaves the target DNA. By mutating specific amino acids in Cas9, DNA cleavage activity is abolished, thus converting it into a transcriptional repressor (called dCas9).

Though many research groups have explored methods to increase the number of sgRNAs that can be expressed at once in vivo, it has been a difficult challenge, in part, because sgRNAs have very repetitive elements. One part of the sgRNA, called the handle, is a 42-nucleotide strand of RNA that physically associates with Cas9. Unfortunately, most DNA synthesis manufacturers are unable to synthesize these repetitive elements, thus limiting the number of sgRNAs that can be assembled and expressed in living organisms.

In a new study, published in Nature Biotechnology, researchers from Penn State University have devised a method that enables 22 distinct sgRNAs to be expressed at once in bacterial cells. The solution? Design and characterize hundreds of non-repetitive genetic parts, including new sgRNA handles, that maintain their function but can actually be synthesized by DNA manufacturers.

I sat down with Alex Reis and Sean Halper (joint first authors) and Howard Salis (corresponding author and Associate Professor at Penn State University) to learn more about multiplexed CRISPR, how nonrepetitive parts are designed, and their plans for the future.

This interview with Alex Reis, Sean Halper and Professor Howard Salis on Simultaneous repression of multiple bacterial genes using nonrepetitive extra-long sgRNA arrays, published in Nature Biotechnology, has been edited for clarity. Words in parentheses are my own.

***

Niko McCarty: Can you tell me a bit about the inspiration behind this study? What was the impetus that made you look at the CRISPR multiplexing field and say, I bet we can improve the number of sgRNAs expressed at once in living cells?

Alex Reis: Well, five years ago, Sean Halper (co-first author) and I took a graduate level course with Professor Howard Salis, and we were exploring different ideas for scalable genetic circuit design. We kept going back to CRISPR because it is a scalable system; all you need to do to build complex CRISPR-based genetic circuits is express one protein regulator (Cas9), and a whole bunch of single-guide RNA regulators (sgRNAs). That was a very powerful idea to us, and we really wanted to scale that up. So this project was motivated from an application side, the desire to build larger genetic circuits.

Professor Howard Salis: When looking at this long DNA sequence or genetic circuit that we had designed for this class project, we basically saw that there were quite a few long regions of repetitive DNA. And if you were to copy-paste that sequence into an order form for any gene synthesis provider, it would immediately tell you, We cant make this. Its too long, too repetitive. So we knew that this was going to be a challenge for the CRISPR field as groups try to multiplex sgRNAs. If you redesign the whole system so that there is no more repetitive DNA, you would be able to build it easier, assemble it faster, and you would be able to express a lot more CRISPR regulators simultaneously.

Niko: Can you walk me through the key advancements from the paper, especially the things that you set out to do and what you accomplished?

Alex: After we identified that repetitive DNA was going to be a key bottleneck in cloning sgRNA arrays, we decided that the first step would be to identify and characterize non-repetitive parts for both genetic expression and the sgRNA handles themselves. The first thing we did was to design and characterize non-repetitive promoters and non-repetitive terminators. But a key challenge that that we faced was to identify non-repetitive handle sequence for sgRNAs. What sequences will enable handle sequence variants to still bind to the Cas9 or dCas9 protein?

To design these non-repetitive sgRNA handles, we carried out multiple rounds of a design, build, and test cycles and imposed specific constraints. In the first design round, the constraint was purely structural we told our algorithm that the sgRNA had to fold into a structure that could be recognized by the Cas9 structure. After that round, we applied a machine learning technique called linear discriminant analysis to identify which mutations would cause handle failure. With that, we identified two nucleotides in the sgRNA handle, G43 and G52 that, when mutated, would abolish handle function. After iterating through these processes a few times, we ultimately characterized 28 highly functional, non-repetitive handle variants. And these handles work equally well for Cas9 and dCas9.

Grace Vezeau, another author on the paper, ran a bunch of cleavage assays to verify, measure and quantify how well these different non-repetitive sgRNA handles were able to load up into Cas9 and cleave DNA.

Niko: After you verified these non-repetitive sgRNA handles, you then used them for three different engineering applications. Can you walk me through those?

Sean Halper: We built three different ELSAs (extra long sgRNA arrays), the longest of which contained 22 distinct sgRNAs. We wanted to come up with some applications that would show the power of scaling up the number of sgRNAs using nonrepetitive handles in E. coli. Our first proof-of-concept was to aerobically produce succinate using a knockdown of six different genes. At first, when we targeted these six genes, it didnt work. We troubleshooted the problem, and found that we had to increase the expression of dCas9, after which we saw a 1000-fold knockdown on some of the genes that we were targeting. This incidentally also showed that, once you start expressing many sgRNAs at once, you need to have enough Cas9 or dCas9 to handle that many simultaneous RNA regulators.

In a second example, we used an ELSA to target different amino acid biosynthesis pathways. We really wanted to see if we could use CRISPRi knockdowns to impose auxotrophy-like behavior. For the third example, we knocked down different stress response genes to explore how a broad spectrum perturbation would affect the behavior and response of E. coli.

Howard: Part of this effort was also to develop algorithms that allow us to design DNA sequences that can be readily synthesized by commercial service providers. Some of these ELSAs have over 20 promoters, 20 terminators, and so forth. Terminators can form hairpins and may contain palindromic sequences, however, so if you ask a gene synthesis provider to synthesize any old DNA with lots and lots of hairpins, theyre going to balk at you. But if you design the system correctly, if you draw from a large enough pool or toolbox of genetic parts, and you arrange those genetic parts just right, you can meet your target metrics for what can be synthesized. As long as your DNA sequence is within those target metrics, then these companies can actually deliver the DNA fragments to you. By the end of this project, we were able to synthesize 33 DNA fragments up to 3 kilobases each, all containing ELSAs, with about a 90% success rate and turnaround time, which is about five days.

Niko: Do you have any plans for designing non-repetitive ribozymes or cleavage sites, which may enable you to express many sgRNAs from a single promoter?

Howard: Let me just start off by saying that we started this project four or five years ago, and we have made some important advancements since then. Another graduate student in our group, Ayaan Hossain, developed an algorithm called the Non-Repetitive Parts Calculator, which formalizes how you can go about designing very large toolboxes of non-repetitive genetic parts. With this algorithm, weve been able to design, construct and characterize huge toolboxes of non-repetitive parts, including 4300 non-repetitive E. coli promoters, 1917 non-repetitive yeast promoters, at least 600 non-repetitive ribozymes with near wildtype cleavage activities, and about 2000 non-repetitive Cas9 handles.

So, is it possible to design many more non repetitive parts? It is absolutely possible. We know that for sure. Theoretically, there are about 100,000 non-repetitive sgRNA handles out there for Cas9. We clearly havent characterized 100,000 yet, weve only characterized 2000, but that kind of gives you an order of magnitude for the possibilities. Now, it should be possible to arrange all these genetic parts in an array and build ELSAs that are about 500,000 bases long, which is smaller than many yeast chromosomes that labs have already built. So its possible to build these very long sgRNA arrays, and there are many applications for them across industrial metabolic engineering and in the biomedical space.

Niko: And what about the different authors on the paper? Were there specific skillsets brought by individuals?

Alex: Sean, myself and Phillip Clauer, a former undergraduate, did the bulk of the cloning and characterization of the parts, but most of the lab pitched in and helped out. Daniel Cetnar helped with RNA level characterization, including a lot of the early RT-qPCR on the CRISPRi knockdowns.

Sean: Ayaan Hossain was really helpful in terms of helping us expand our non-repetitive part libraries for the promoters and terminators especially, as well as helping with some of the machine learning analysis. But it was definitely a collaborative effort over the last five years.

Niko: What are your plans for after graduation?

Sean: I actually defended my PhD just a couple of weeks ago. Im part of the SMART Scholarship for Service program, which is a fellowship with the Department of Defense. Once I wrap up here, I plan on beginning work soon with my sponsoring facility, the Army Research Lab in Adelphi, Maryland.

Alex: Im wrapping up a project or two and then will hopefully graduate and move on to the next thing. I love synthetic biology, so I am looking at postdocs along those lines. Im also thinking about some entrepreneurial aspects that I could pursue.

Niko: This study is so appealing to me, in part, because of its collaborative nature. It seems like most people in the group helped out can you tell me a bit about that?

Howard: Well, we have a very relaxed environment. While some people in the synthetic biology field have groups with 30-40 people, our group has less than 10. This means that everyone knows everyone else, and we all help each other. I intentionally set up my lab so that new people come in, and they receive training not just from myself, but from other graduate students and postdocs. Because of this, many students feel the obligation to pay it forward and help out other people. If youre really good at something, and you can carry out some set of experiments quickly, then you should help out your colleagues in the lab. In our group, a lot of sharing goes on, and thats what makes work like this possible.

***

Biographies:

Howard Salis is an Associate Professor of Biological and Chemical Engineering and Synthetic Biology at Penn State University. Research in the Salis laboratory focuses on the development of rational design methods for engineering synthetic biological systems metabolic pathways, genetic circuits, and genomes.

Sean Halper is a graduate student at Penn State University, and co-first author on this study. He recently defended his PhD in Chemical Engineering.

Alex Reis is a graduate student at Penn State University, and co-first author on this study.

The rest is here:
Science Behind the Scenes: Multiplexed CRISPR and sgRNA Arrays with the Howard Salis Lab | PLOS Synthetic Biology Community - PLoS Blogs

The research report provides valuable insights into demand drivers, geographical outlook, and competitive landscape of the CRISPR and Cas Genes Market over the forecast period. Further, it throws light on restraints as well discusses opportunities at length that are likely to come to the fore over the forecast period. The analysis thus provided helps market stakeholders with business planning and to gauge scope of expansion in the CRISPR and Cas Genes Market over the forecast period.

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CRISPR and Cas Genes Market Estimated to Rise at a Lucrative CAGR of 20.1% During 2018-2026 - TheFinanceTime

The CRISPR gene-editing tool can be used to silence an important hepatitisB virus gene, a proof-of-concept invitro study suggests.

"It's the first time we've seen CRISPR editing done in a hepatitisB model," said Douglas Dieterich, MD, director of the Institute of Liver Medicine and professor of medicine at the Icahn School of Medicine at Mount Sinai in New York City.

HepatitisB can lead to liver disease and is the primary cause of hepatocellular carcinoma. In 2015, more than 250million people around the world were infected with the virus, according to the World Health Organization.

For their study, investigator Hao Zhou, from The First Hospital of Jilin University in China and the Department of Medicine at the University of Minnesota in Minneapolis, and colleagues targeted the Sgene. Zhou presented the findings at the Liver Meeting 2019 in Boston.

The Sgene gives rise to the hepatitisB surface antigen, the presence of which indicates that a person is infected with the virus. "The question is whether it's the right target," Dieterich told Medscape Medical News.

Reducing the amount of the hepatitisB surface antigen is a "good idea" because that's what is believed to inhibit the immune system from clearing the virus. Doing so might help the immune system recover and clear the virus, "with a little help from some antivirals," explained Dieterich, who was not involved in the study.

However, "the surface is not the only DNA that's integrated into the host genome," he pointed out. "I think maybe a broader application might be necessary to actually get the hepatitisB genome out of the hepatocytes."

Zhou's team used a newer CRISPR approach, called CRISPR-STOP, for their gene-editing procedure.

"The idea is that CRISPR-STOP can be as efficient as standard CRISPR editing, but it's safer," said Kiran Musunuru, MD, PhD, associate professor of cardiovascular medicine and genetics at Penn Medicine in Philadelphia, who was not involved in the study. Musunuru is cofounder of and senior scientific advisor at Verve Therapeutics, a company using gene editing to prevent cardiovascular disease.

The standard CRISPR-Cas9 approach requires a double-strand break in the genome, and the problem with that is it introduces the possibility for "mischief," he explained. "If you have more than one double-strand break occurring in the human genome at the same time, you have the potential for different parts of different chromosomes coming together in the wrong ways and then causing problems."

Instead of creating a double-strand break, CRISPR-STOP uses a base editor to chemically modify the DNA base from one base to another and introduce a stop codon into the target gene sequence, effectively hamstringing the ability of the target gene to produce a functional protein.

This is a very nice, clean way to turn off a gene effectively.

"This is a very nice, clean way to turn off a gene effectively," Musunuru told Medscape Medical News.

For their CRISPR-STOP procedure, Zhou's team first transduced liver cells infected with the hepatitisB virus using a base editor called AncBE4max. Next, to activate the base editor so that gene editing could begin, they transduced the cells with one of two lentivectors: one encoded for single-guide RNA that targets the Sgene; and an empty one, which served as the control.

With the gene-editing approach, 71% of the liver cells that expressed the base editor gained the desired stop codon in the target gene.

"That's a very robust number," said Musunuru.

In addition, hepatitisB surface antigen secretion was reduced by 92% with the gene-editing approach.

The investigators report a high degree of conservativity for hepatitisB genotypesB, C, F, and H. Specifically, 94% of the Sgene sequence was conserved for genotypeB, 92% for genotypeC, 91% for genotypeF, and 71% for genotypeH.

The Liver Meeting 2019: American Association for the Study of Liver Diseases (AASLD): Abstract86. Presented November10, 2019.

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Link:
CRISPR Used to Silence Crucial Hepatitis B Gene - Medscape

New cell experiments show more effective genetic cuts that could one day become the foundation of more effective gene therapies.

Researchers may have found a way to sharpen the CRISPR-Cas9 technique so it can more successfully cut out undesirable genetic information and could one day fast-track potential therapies for HIV, sickle cell disease and, potentially, other immune conditions.

This is the first time scientists have systematically gone through the guide RNA sequence to change it and improve CRISPR-Cas9 technology, said Tristan Scott, PhD, lead author of the study and a staff research scientist at City of Hopes Center for Gene Therapy.

This could lead to more clean results in cell and mouse model experiments aimed at developing new therapies because the target that was knocked out was more successfully removed. More pronounced results could quicken new the development of therapies. In theory, the therapeutic product should have more successful cuts, which could translate into an improved therapy.

The researchers experimented on cells by making changes to the trans-activating CRISPR RNA (or tracrRNA), which is derived from Streptococcus pyogenes bacteria and is a part of the components used to guide the genetic scissors (Cas9) to the right gene sequence.

They found that the modified tracrRNA improved the silencing of certain genes by increasing desirable mutations in the genetic material. In this study, the target was an essential component of HIVs lifecycle, the protein CCR5 on immune CD4+ T cells. The modified tracrRNA improved cutting at this site and inactivation of CCR5, and hopefully that will translate into better protection for the immune system.

The new design was also better at improving activity at the HBB gene and the BCL11A site, both of which are tied to sickle cell disease and are being targeted in order to develop therapies for the currently incurable blood disease that causes intense pain and premature death.

If this line of research remains consistent and we can dependably sharpen the genetic scissor, the result could eventually be new or improved genetic therapies, Scott said.

The study was published inScientific Reports.

Read more here:
Modified CRISPR gene editing tool could improve therapies - Drug Target Review