Archive for the ‘Crispr’ Category

New Jersey, NJ, Nov. 13, 2019 (GLOBE NEWSWIRE) -- The key contributing factors for the market growth are increasing demand for enhanced crop varieties using modern breeding techniques and exponential reduction in the cost of genomic solutions. Theglobal plant breeding and CRISPR plants market is expected to grow from USD 6.3 Million in 2017 to USD 21.2 Million by 2025 at a CAGR of 16.4% during the forecast period 2018-2025, according to the new report published by Fior Markets.

The CRISPR-Cas9 system is defined as a plant breeding innovation that uses site-directed nucleases to target and transform DNA with great accuracy. It was developed in 2012 by scientists from the University of California, Berkeley, and has received a lot of focus in recent years due to its wide range of uses, including biological research, breeding and development of crops and animals, and human health applications. It also includes gene silencing, DNA-free CRISPR-Cas9 gene editing, homology-directed repair (HDR), and transient gene silencing or transcriptional repression (CRISPR).

Increasing demand for enhanced crop varieties using modern breeding techniques is a major factor driving the market. Also, exponential reduction in the cost of genomic solutions and advancements in technology ensure strong market growth. High cost associated with modern breeding methods as compared to conventional breeding, poor laboratory infrastructure and lack of validated markers hampers the growth of the market. However, rising investments from seed companies and favourable regulations for molecular breeding may boost the market in the coming years.

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Key players in the plant breeding and CRISPR plants market are Bayer, Syngenta, KWS, DowDuPont, Eurofins, SGS, Advanta Seeds, Benson Hill Biosystems, Bioconsortia, DLF, Equinom, Evogene, Groupe Limagrain, Hudson River Biotechnology, Land Olakes, Pacific Biosciences, SGS, and Syngenta among others. Key players active in the market are involved in collaborative agreements and expansion to bolster the growth of the market.

The hybridization segment held the largest market share of 45.70% in 2017

The process segment is classified into selection, hybridization and mutation breeding. The hybridization segment is dominated the Plant Breeding and CRISPR Plants Market in 2017 with a market share of 45.70%. The most successful applications of hybridization breeding are the utilization of heterosis and generation of seedless horticultural crops, such as watermelon, by employing diploid and tetraploid parents.

Biotechnological method segment valued around USD 3.99 Million in 2017

The type segment includes conventional breeding and biotechnological method. Biotechnological method segment valued around USD 3.99 Million & dominated the market in 2017. The increasing implementation of hybrid and molecular breeding techniques in developing countries and the rising cultivation of GM crops in the Americas are the factors contributing to its high growth.

The herbicide tolerance segment held the largest market share of 36.90% in 2017

Trait segment is divided into segments such as herbicide tolerance, disease resistance, yield improvement and other traits. The herbicide tolerance segment dominated the market in 2017 with a market share of 36.90%. Rising regulations on the use of chemical pesticides and increasing instances of pest attacks during the early germination phase have risen considerably due to the need for pesticide-tolerant seeds.

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The cereals & grains segment valued around USD 2.40 Million in 2017

The application segment includes cereals & grains, oilseeds & pulses, fruits & vegetables and other crop types. The cereals & grains segment valued around USD 2.40 Million and dominated the market in 2017. Corn, wheat, and rice are the major cereals bred with advanced technologies such as molecular breeding and genetic techniques. The availability of germplasm for these crops encourages the adoption of advanced breeding techniques.

Regional Segment Analysis of the Plant Breeding and CRISPR Plants Market

Asia Pacific region dominated the global plant breeding and CRISPR plants market with USD 2.72 Million in 2017. The Asia Pacific region is a major manufacturing hub owing to the ever-increasing demand for commercial seeds in the Asian market aligned with the growing economic growth conditions. Also, seed producers such as Bayer, Monsanto, and Syngenta have been showing increasing interest in tapping this potential market, wherein the companies have been expanding their R&D centres across the Asia Pacific. North America is the second fastest-growing region due to the increasing industrial value for corn and soybean in the US which is encouraging breeders to adopt advanced technologies for better yield, owing to which the adoption rate for genetics in this country remains high.

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The global plant breeding and CRISPR plants market is analysed on the basis of value (USD Million). All the segments have been analysed on global, regional and country basis. The study includes the analysis of more than 30 countries for each segment. The report offers in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining the key insight of the market. The study includes porters five forces model, attractiveness analysis, raw material analysis, supply, demand analysis, competitor position grid analysis, distribution and marketing channels analysis.

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Global Plant Breeding and CRISPR Plants Market is Expected to Reach USD 21.2 Million by 2025 : Fior Markets - GlobeNewswire

Upon meeting for the first time at a dinner at Stanford earlier this year, Fei-Fei Li and Jennifer Doudna couldnt help but note the remarkable parallels in their experiences as scientists.

Stanfords Fei-Fei Li and Jennifer Doudna of UC Berkeley will discuss the ethics of artificial intelligence and CRISPR technology. (Image credit: Getty Images)

Both women helped kickstart twin revolutions that are profoundly reshaping society in the 21st century Li in the field of artificial intelligence (AI) and Doudna in the life sciences. Both revolutions can be traced back to 2012, the year that computer scientists collectively recognized the power of Lis approach to training computer vision algorithms and that Doudna drew attention to a new gene-editing tool known as CRISPR-Cas9 (CRISPR for short). Both pioneering scientists are also driven by a growing urgency to raise awareness about the ethical dangers of the technologies they helped create.

It was just incredible to hear how similar our stories were. Not just the timing of our scientific discoveries, but also our sense of responsibility for the ethics of the science are just so similar, said Li, who is a professor of computer science at Stanfords School of Engineering and co-director of the Stanford Institute for Human-Centered Artificial Intelligence (HAI).

The ethical angle to what we were doing was not something that either of us anticipated but that we found ourselves quickly drawn to, said Doudna, who is a professor of chemistry and of molecular and cell biology at the University of California, Berkeley.

The echoes between Li and Doudnas lives were also not lost on the dinner host that night, Stanford political science professor Rob Reich, who invited the pair to resume their conversation in public. Their talk, titled CRISPR, AI, and the Ethics of Scientific Discovery, will take place at Stanford on Nov. 19 and will be moderated by Stanford bioengineering professor Russ Altman(livestream will be available here).

The event is organized by the Stanford McCoy Family Center for Ethics in Society and HAI and is part of the Ethics, Society & Technology Integrative Hub that arose from the universitys Long-Range Vision.

The subject of the lecture hits the sweet spot of what the Integrative Hubs work is about, which is to cultivate and support the large community of faculty and students who work at the intersection of ethics, society and technology, said Reich, who directs the Center for Ethics in Society and co-directs the Integrative Hub.

I cant think of two better people to engage in a conversation and to really take seriously these questions of how, as you discover the effects of what youve created, do you bring ethical implications and societal consequences into the discussion? said Margaret Levi, a professor of political science at Stanfords School of Humanities and Sciences. Levi is also the Sara Miller McCune Director of the Center for Advanced Study in the Behavioral Sciences and co-director of the Integrative Hub.

Fei-Fei Li is a professor of computer science and co-director of Stanfords Institute for Human-Centered Artificial Intelligence. (Image credit: L.A. Cicero)

In 2006, Li wondered if computers could be taught to see the same way that children do through early exposure to countless objects and scenes, from which they could deduce visual rules and relationships. Her idea ran counter to the approach taken by most AI researchers at the time, which was to create increasingly customized computer algorithms for identifying specific objects in images.

Lis insight culminated in the creation of ImageNet, a massive dataset consisting of millions of training images, and an international computer vision competition of the same name. In 2012, the winner of the ImageNet contest beat competitors by a wide margin by training a type of AI known as a deep neural network on Lis dataset.

Li immediately understood that an important milestone in her field had just been reached, and despite being on maternity leave at Stanford, flew to Florence, Italy, to attend the award ceremony in person. I bought a last-minute ticket, Li said. I was literally on the ground for about 18 hours before flying back.

Computer vision and image recognition are largely responsible for AIs rapid ascent in recent years. They enable self-driving cars to detect objects, Facebook to tag people in photos and shopping apps to identify real-world objects using a phones camera.

Within a year or so of when the ImageNet result was announced, there was an exponential growth of interest and investment into this technology from the private industry, Li said. We recognized that AI had gone through a phase shift, from being a niche scientific field to a potential transformative force of our industry.

The field of biology underwent its own phase shift in the summer of 2012 when Doudna and her colleagues published a groundbreaking paper in the journal Science that described how components of an ancient antiviral defense system in microbes could be programmed to cut and splice DNA in any living organism, including humans, with surgical precision. CRISPR made genomes as malleable as a piece of literary prose at the mercy of an editors red pen, Doudna would later write.

CRISPR could one day enable scientists to cure myriad genetic diseases, eradicate mosquito-borne illnesses, create pest-resistant plants and resurrect extinct species. But it also raises the specter of customizable designer babies and lasting changes to the human genetic code through so-called germline editing, or edits made to reproductive cells that are transmitted to future generations.

This bioethics nightmare scenario was realized last fall when a Chinese researcher declared that he had used CRISPR to edit the genomes of twin girls in order to make them resistant to HIV. Doudna decried the act but allows that her own views on germline editing are still evolving.

Ive gone from thinking never, ever to thinking that there could be circumstances that would warrant that kind of genome editing, she said. But it would have to be under circumstances where there was a clear medical need that was unmet by any other means and the technology would have to be safe.

Both Li and Doudna fervently believe in the potential of their technologies to benefit society. But they also fear CRISPR and AI could be abused to fuel discrimination and exacerbate social inequalities.

The details are different for CRISPR and AI, but I think those concerns really apply to both, Doudna said.

Rather than just leaving such concerns to others to work out, both scientists have stepped outside of the comfort of their labs and taken actions to help ensure their worst fears dont come to pass. I almost feel that at this point of history I need to do this, not that its my natural tendency, Li said. It really is about our collective future due to technology.

Both scientists have testified before Congress about the possibilities and perils of their technologies. Li also co-launched a nonprofit called AI4All to increase inclusion and diversity among computer engineers and she co-directs Stanford HAI, which aims to develop human-centered AI technologies and applications. Doudna spends significant time talking to colleagues, students and the public about CRISPR. In 2015 she organized the first conference to discuss the safety and ethics of CRISPR genome editing.

Because we were involved in the origins of CRISPR, I felt it was especially important for my colleagues and me to be part of that discussion and really help to lead it, Doudna said. I asked myself, If I dont do it, who will?

To read all stories about Stanford science, subscribe to the biweekly Stanford Science Digest.

Altman is the Kenneth Fong Professor of Bioengineering, Genetics, Medicine, Biomedical Data Science and host of the Stanford Engineering radio show The Future of Everything. Levi is a member of Stanford Bio-X, the Wu Tsai Neurosciences Institute, and the Stanford Woods Institute for the Environment. Li is the Sequoia Capital Professor at Stanford and a member of Stanford Bio-X and the Wu-Tsai Neurosciences Institute.

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AI and gene-editing pioneers to discuss ethics - Stanford University News

1. CRISPR: the movie  Nature.com
2. CRISPR: More than just for gene editing?  Phys.org
3. Penn Med study on CRISPR cancer therapy indicates technique is safe in humans  The Daily Pennsylvanian
4. Controversial CRISPR gene-editing tool could be used to detect viruses  Daily Mail
5. Crispr Takes Its First Steps in Editing Genes to Fight Cancer  Seattle Times
6. View full coverage on Google News

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CRISPR: the movie - Nature.com

Amended Celgene collaboration to focus on engineered alpha-beta T cell medicines with a $70 million payment to Editas Medicine

Appointed Judith R. Abrams, M.D., as Chief Medical Officer

EDIT-101 (AGN-151587) for LCA10 first patient dosing expected by early 2020

EDIT-301 for hemoglobinopathies in vivo pre-clinical data to be presented at ASH

CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today reported business highlights and financial results for the third quarter of 2019.

"Our momentum in 2019 remains strong in advancing our pipeline of in vivo CRISPR and engineered cell medicines," said Cynthia Collins, Chief Executive Officer of Editas Medicine. We announced this morning an amended agreement with Celgene to further expand and accelerate our oncology pipeline. In hemoglobinopathies, we look forward to presenting in vivo pre-clinical data for EDIT-301 at ASH that supports its potential as a best-in-class medicine. Finally, we eagerly anticipate first patient dosing with EDIT-101 for LCA10 in the coming months.

Recent Achievements and OutlookIn VivoCRISPR Medicines

Engineered Cell Medicines

Corporate

Upcoming Events

Editas Medicine will participate in the following investor events:

Editas Medicine will present pre-clinical data for EDIT-301 to address sickle cell disease and beta-thalassemia in at the 61st American Society of Hematology Annual Meeting & Exposition. Details are as follows:

Abstract Number: 4636Title: EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCDPresenter: Edouard De Dreuzy, Ph.D.Session: 801. Gene Therapy and Transfer: Poster III Time: Monday, December 9, 2019: 6:00 PM-8:00 PMLocation: Hall B, Orange County Convention Center, Orlando, FL

Third Quarter 2019 Financial Results

Cash, cash equivalents, and marketable securities at September 30, 2019, were $332.6 million, compared to $369.0 million at December 31, 2018. The $36.4 million decrease was primarily attributable to operating and capital expenses related to our on-going preclinical and clinical activities, patent costs and license fees, and employee-related costs, partially offset by $42.1 million in proceeds from financing activities.

For the three months ended September 30, 2019, net loss was $32.9 million, or $0.66 per share, compared to $15.2 million, or $0.32 per share, for the same period in 2018.

Conference Call

The Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the third quarter of 2019. To access the call, please dial 844-348-3801 (domestic) or 213-358-0955 (international) and provide the passcode 6577216. A live webcast of the call will be available on the Investors & Media section of the Editas Medicine website at http://www.editasmedicine.com and a replay will be available approximately two hours after its completion.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

About EDIT-101 (AGN-151587)EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About Leber Congenital AmaurosisLeber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 2030 percent of all LCA patients.

About the Editas Medicine-Allergan AllianceIn March 2017, Editas Medicine and Allergan Pharmaceuticals International Limited (Allergan) entered a strategic alliance and option agreement under which Allergan received exclusive access and the option to license up to five of Editas Medicines genome editing programs for ocular diseases, including EDIT-101 (AGN-151587). Under the terms of the agreement, Allergan is responsible for development and commercialization of optioned products, subject to Editas Medicines option to co-develop and share equally in the profits and losses of two optioned products in the United States. In August 2018, Allergan exercised its option to develop and commercialize EDIT-101 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from EDIT-101 in the United States. Editas Medicine is also eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicines unparalleled CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a).

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companys plans with respect to the Brilliance Phase 1/2 clinical trial for EDIT-101 (AGN-151587), including the Companys expectations regarding the timing of dosing a patient by early 2020. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

Investor ContactMark Mullikin(617) 401-9083mark.mullikin@editasmed.com

Media ContactCristi Barnett(617) 401-0113cristi.barnett@editasmed.com

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Editas Medicine Announces Third Quarter 2019 Results and Update - GlobeNewswire

Q&A

Penn's Kiran Musunuru talks to us about the technology that has been both praised and criticized for its ability to alter human DNA and potentially cure disease.

Kiran Musunuru is an associate professor of medicine in genetics in the Perelman School of Medicine at the University of Pennsylvania. / Courtesy

CRISPR, the technology being used to edit genes in humans, remains polarizing. On one end, detractors argue that using the technology for certain purposes, like performing gene editing on embryos, is not only dangerous but unethical. On the other end, proponents say CRISPR has the potential to revolutionize human health, and early data shows they might be right. Despite a medical community that is still split on the issue, researchers in the U.S. are kicking tests of the technology into high gear. Several clinical trials have launched in the U.S. testing CRISPRs ability to treat various diseases.

NextHealth PHL spoke with Kiran Musunuru, an associate professor of medicine in genetics at the Perelman School of Medicine at the University of Pennsylvania about the true potential of CRISPR technology and how we can expect it to evolve in the future.

NextHealth PHL: What exactly is CRISPR?Musunru: CRISPR is sort of a catch-all term that covers a variety of technologies. If youre saying CRISPR, youre referring to a broad set of tools that may do it in different ways but are all intended to do a form of gene editing or genome editing.

How do basic CRISPR technologies work?The simplest form of CRISPR, what I call version 1.0, is the original standard CRISPR that most laboratories and companies interested in developing new therapies use. It is a two-component system. There is a protein and an RNA molecule thats about 100 bases in length. The protein and the RNA molecule come together to create what well call a molecular machine and the purpose of this molecular machine is to scan across any DNA molecule it encounters. So if you put the CRISPR-Cas9 into the nucleus of a human cell, this molecular machine will scan the entire genome.

The machine has two key functions built into it; the first is a GPS function. When you change the first 20 bases in a DNA length (the first 20 bases is basically the address) to whatever address you want, the GPS function makes the machine go through the entire genome and find the sequence that matches the address. The second function of this machine is to protect the genome, like a search-and-destroy function. You put in the address, it goes to that matching place in the genome and then it makes a cut in the DNA.

Cutting the DNA is actually a bad thing but the cells have ways to try to fix that break, and the actual editing is a result of the cell trying to fix that break in the DNA, not from CRISPR itself, interestingly enough.

How does CRISPR turn a break in someones DNA into a good thing?There are a few ways this can happen. The safest thing you can do is to break a gene or turn off a gene. The metaphor I like to use is to think of the whole genome as a book, and each chromosome in the genome is a chapter in the book, and each gene is a paragraph in the chapter. Together, it all has a meaning. But lets say you had to turn off a gene, the equivalent of making that break in the DNA would be like tearing the page through that paragraph. So, the simplest thing the cell can do and will try to do is to simply tape that tear back up. But as you can imagine, sometimes you tape it back up and its fine, the paragraph is still legible and the meaning is still there, and it eventually heals and functions like it did before. But in this case, thats actually not what you want. The outcome that you want with CRISPR is that you actually want to turn off the gene, not to rip it and make it the way it was before.

What has to happen is when you make the tear, the tear is so rough, you get those jagged edges and you try to tape it up but it doesnt quite fit, the letters dont quite match up. You tape it up as best as you can but its illegible, some letters are lost, and the meaning of the paragraph is lost. Thats exactly what happens with gene editing, the cell tries to repair that break in the DNA, doesnt get it quite right, and loses some bases and that messes up the gene and turns it off.

However, in this scenario, you cant really control what happens. All you can hope for is that that tear you make is going to mess up the gene and thats okay if all youre trying to do is turn it off. Most of the trials underway now are about turning off the gene, and theyre all taking advantage of the fact that its relatively easy to mess up genes and turn off genes. Just like tearing a page its crude, but its effective.

Theres CRISPR 1.0, this first generation of the technology thats not very precise and is a bit arduous. What are the newest forms of CRISPR and how are they better than earlier versions of the technology? There is a newer form of the technology called base editing that keeps the GPS function intact but removes the cutting function. In place of the cutting function, it attaches another machine onto CRISPR and makes chemical modifications in certain areas. This version of CRISPR is more like a search and replace. CRISPR provides the search but then another machine attached to it is doing the replacing. With base editing you can make more precise changes, but only rarely will it make exactly the type of change you want.

The latest form of CRISPR is called prime editing, and we still dont have a good sense of how well it works because its so new. Whats tantalizing is that it looks like it can turn CRISPR into a precise word processor or an eraser that allows you to erase a letter and put in a new letter. CRISPR is very much a wave of technology, and as it gets better, its going to allow us to do more and more powerful things.

There are some extreme ideas about what CRISPR can do. Some believe scientists can use the technology to alter hair or eye color or give patients superhuman athletic or intellectual abilities. Is any of this possible with CRISPR?It depends on what traits youre talking about changing. Since eye color and hair color are controlled by single genes, you could possibly make a single gene change with CRISPR. The problem is, how do you get CRISPR to go where it needs to go to change your hair or eye color? How do you get it into all your hair follicles or through all the cells in your eye? It might be a simpler change to make, but it might not be easy to do in a live adult. Scientists have now edited human embryos, resulting in live-born people. Theres been a lot of ethical debate about whether thats a good thing. If you want to change something like hair color in a single cell embryo made through in-vitro fertilization, thats a bit different and might not be as difficult.

There are some very complicated things, like intelligence or athletic ability, that are not going to be easy to change. Youd probably have to change hundreds of genes, and thats not going to happen anytime soon. With CRISPR as it is now, maybe you can change one gene; maybe if you really work at it you can change two genes, but hundreds of genes? Youre not going to be able to do that with CRISPR anytime soon.

What has CRISPR been used to treat so far and what could it be used for in the future?There are multiple trials underway to treat rare liver disorders. More recently CRISPR has been used in clinical trials at Penn where at least three patients have been dosed using CAR T immunotherapy. In this case, theyre trying to make patients cells more effective at fighting cancer. But again, that editing is being done outside the body.

There are some things that seem like they would be difficult to treat, but if its the right type of disease and you can get CRISPR to where you need it to go, it might work. One example is in sickle cell disease. The cells that you need to fix in sickle cell disease are in the bone marrow. Fortunately, bone marrow is relatively straight forward to work with. You take the cells out and edit them with some form of CRISPR outside of the body and then put them back in.

Something like cystic fibrosis would be much harder because it affects the entire surface of potentially multiple organs inside the body. Its much harder to deliver CRISPR to all of those places in the body.

There are two other clinical trials that have started in the U.S. One is from a company called CRISPR Therapeutics to treat sickle cell disease and similar blood disorders. Theres another trial underway to treat a genetic form of blindness and this editing would actually happen inside the body.

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Q&A: Everything You Need to Know About the Future of CRISPR-Cas9 - Philadelphia magazine

Professor Glenn Cohen is a Professor of Law at Harvard Law School. He is also the director of Harvard Law School's Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics, and one of the world's leading experts on the intersections of bioethics and the law. Cohen's current projects relate to big data, health information technologies, reproduction/ reproductive technology, research ethics, organ rationing in law and medicine, health policy, FDA law, translation medicine, and medical tourism. The utilization of CRISPR technology as a gene editing tool has spurred significant debate across the globe. In this interview, we gain insight of Cohen's perspectives on the "CRISPR revolution" and learn about the basic ethical issues surrounding the manipulation of the genome for enhancement.

Molly Campbell (MC): You are one of the leading experts on the intersection of bioethics and the law. Please can you tell us more about this field and the types of cases it addresses?Glenn Cohen (GC): Wherever law, medicine, and ethics intersect, thats where the field and I are. Whether it is the ethics of research, reproductive technologies, genetics, end of life decision-making, mental health, neuroscience, rationing, AI, clinical practice, etc. It is a robust and very exciting field.

MC: Currently, what restrictions apply to the use of CRISPR technology in different cell types and organisms? What applications are scientists not allowed to use CRISPR or other gene-editing technologies for? GC: In lay terms, in the United States an appropriations rider prohibits FDA from considering the use of germline gene editing in human beings. Thus, it is not possible to do a clinical trial or the like of this. Many (perhaps all, it is not clear everywhere) other countries across the world also prohibit in one way or another, but not all regulatory regimes may be as effective.

MC: The work of Jiankui He arguably startled the scientific community. In your opinion, do you think the publication of He's work prompted authorities to address regulating CRISPR technology? Or was there already a conversation taking place?GC: There was very robust conversation long before Dr. Hes terrible (and in my view completely unethical) experiments. For example, this report from the National Academies. While CRISPR is relatively new in terms of technology, in fact bioethicists have been talking about the basic issues surrounding manipulating the genome for enhancement for at least 40 years if not longer.

MC: There are concerns that the CRISPR tool could be used for enhancement purposes. In recent opinion article you say, "Anyone who has a position on enhancement has not thought deeply enough on the question." Please can you expand on what you mean by this?

GC: My claim is that enhancement is not a single monolithic thing, so it is hard to have a single position on it. Some enhancements would be wonderful and perhaps the state should subsidize them. Others would be terrible and perhaps the state should prohibit. Only when we think about it with some specificity can we know what we think the answer should be. In the article you mention I draw the following distinctions, for example, though others are possible:

1. Biological vs. Non-Biological Enhancement

a. Genetic enhancements vs. non-genetic biological enhancements

2. Choosing for Ourselves vs. Choosing for Others Who Cannot Choose for Themselvesa. Enhancing after birth vs. enhancing before birthi. Enhancing by selection vs. enhancing by manipulation of already fertilized embryos or implanted fetuses

3. Enhancements Compatible with Expanding Life Plans vs. Enhancements That Will Limit Options

4. Reversible vs. Irreversible Enhancement

5. Some would distinguish enhancement from treatment (though others are skeptical about this distinction)a. Enhancements to the upper bounds of what people already have vs. enhancements that add beyond human nature as it now stands

6. Enhancements for Absolute vs. Positional Goods

MC: A novel community of gene-editing "biohackers" has emerged in the rise of CRISPR technology. What are your opinions of biohackers conducting gene-editing experiments from their homes, from a legal and ethical perspective?GC:I think the community is very interesting. I am a huge fan of open science and the building of intellectual communities. I think the key question is whether/when the work undertaken by this community could pose significant externalities for others. Thats probably where I would start to get concerned.

MC: How do we approach implementing a global legal and ethical framework for using gene-editing technologies? What progress has been made thus far?GC: The WHO has chartered an advisory committee which has recommended a registry of all those doing gene editing work and has advised that it is irresponsible at this time for anyone to proceed with clinical applications of human germline genome editing." I think the existence of this committee (alongside the NASEM, Nuffield Council) and others working on these issues is a great step.

My own view is that we ought to be looking for a responsible translational pathway that might allow some clinical work to be reviewed and approved by regulators like the FDA in the future, but certainly there is nothing there yet. The international aspect makes this very, very difficult. Some have suggested we ought to go for an international treaty, like what we have on landmines and chemical weapons but also recognition of adoption, while others think this is infeasible.

MC: What challenges exist when looking to create laws surrounding a novel scientific technology?GC: There are quite a few. The first is uncertainty whenever you move to first-in-humans, whatever pre-clinical work you have done, there is always open questions. The same was true with IVF. The second is the politicization of science and the reduction of difficult and nuanced questions to talking points. The third is deep philosophical disagreement on some key points (for example, some take quite literally the idea of "man created in Gods image" and view altering the human genome as a rejection of that. If thats what someone believes for religious reasons then it is very hard to talk about these issues at a more policy level). Fourth, is the importance but difficult of public engagement. The UK in its public consultation on mitochondrial replacement therapy (that ultimately paved the way for permitting that technology to be used in a limited way) was a very good recent model, but quite difficult and expensive. Moreover, some felt it didnt go far enough in the direction of deliberative democracy. The hope is we will see more such initiatives for gene editing and other novel technologies.

Professor Glenn Cohen, Haravard Law School, was speaking with Molly Campbell, Science Writer, Technology Networks.

Catch up on the previous instalment of Technolology Networks Explores the CRISPR Revolution, an interview with Professor George Church, here.

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Technology Networks Explores the CRISPR Revolution: An Interview With Professor Glenn Cohen, World-leading Expert on Bioethics - Technology Networks

The first attempt in the United States to use a gene editing tool called CRISPR against cancer seems safe in the three patients who have had it so far, but its too soon to know if it will improve survival, doctors reported Wednesday.

The doctors were able to take immune system cells from the patients blood and alter them genetically to help them recognize and fight cancer, with minimal and manageable side effects.

The treatment deletes three genes that might have been hindering these cells ability to attack the disease, and adds a new, fourth feature to help them do the job.

Its the most complicated genetic, cellular engineering thats been attempted so far, said the study leader, Dr. Edward Stadtmauer of the University of Pennsylvania in Philadelphia. This is proof that we can safely do gene editing of these cells.

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After two to three months, one patients cancer continued to worsen and another was stable. The third patient was treated too recently to know how shell fare. The plan is to treat 15 more patients and assess safety and how well it works.

Its very early, but Im incredibly encouraged by this, said one independent expert, Dr. Aaron Gerds, a Cleveland Clinic cancer specialist.

Other cell therapies for some blood cancers have been a huge hit, taking diseases that are uncurable and curing them, and the gene editing may give a way to improve on those, he said.

Gene editing is a way to permanently change DNA to attack the root causes of a disease. CRISPR is a tool to cut DNA at a specific spot. Its long been used in the lab and is being tried for other diseases.

This study is not aimed at changing DNA within a persons body. Instead it seeks to remove, alter and give back to the patient cells that are super-powered to fight their cancer a form of immunotherapy.

Chinese scientists reportedly have tried this for cancer patients, but this is the first such study outside that country. Its so novel that it took more than two years to get approval from U.S. government regulators to try it.

The early results were released by the American Society of Hematology; details will be given at its annual conference in December.

The study is sponsored by the University of Pennsylvania, the Parker Institute for Cancer Immunotherapy in San Francisco, and a biotech company, Tmunity Therapeutics. Several study leaders and the university have a financial stake in the company and may benefit from patents and licenses on the technology.

Two of the patients have multiple myeloma, a blood cancer, and the third has a sarcoma, cancer that forms in connective or soft tissue. All had failed multiple standard treatments and were out of good options.

Their blood was filtered to remove immune system soldiers called T cells, which were modified in the lab and then returned to the patients through an IV. Its intended as a one-time treatment. The cells should multiply into an army within the body and act as a living drug.

So far, the cells have survived and have been multiplying as intended, Stadtmauer said.

This is a brand new therapy so not its not clear how soon any anti-cancer effects will be seen. Following these patients longer, and testing more of them, will tell, he said.

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Doctors try CRISPR gene editing for cancer, a 1st in the US - NBCNews.com

A team of scientists from City University of Hong Kong (CityU) and the Karolinska Institute has created a novel protein that can increase the target accuracy in genome editing. Their findings are published in the journal Proceedings of the National Academy of Sciences (PNAS).Meet CRISPRThe gene editing technology Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9 looks set to revolutionize modern medicine, agriculture, and synthetic biology.The ability to edit the genome in vivo offers the potential to develop novel gene therapies for diseases that currently lack viable treatment options. Several clinical trials are underway exploring the utility of CRISPR technology in treating specific cancers, blood disorders and eye diseases.CRISPR-Cas9 as a gene editing tool is superior over other techniques due to its ease of use. In traditional gene therapy, additional copies of the "normal" gene are introduced into cells. Using CRISPR technology, this isnt necessary; CRISPR-Cas9 enters the cell and "repairs" the problematic gene by removing it or correcting it to restore normal physiological function.

There are different components to the CRISPR mechanism. Cas9 is the enzyme that flags and locates the problematic DNA throughout the genome, acting in a "hunting" fashion. However, the precision of Cas9 cannot always be established, and occasionally modifications of DNA at unintended places can occur. If CRISPR is to be utilized to repair faulty genes in patients, potential off-target genome editing could have serious adverse effects.

There are currently two versions of the Cas9 enzyme commonly adopted in CRISPR research: SpCas9 (Cas9 nuclease from the bacteria Streptococcus pyogenes) and SaCas9 (Cas9 nuclease from Staphylococcus aureus). Both of these enzymes are limited in that they possess a certain level of imprecision.

Thus, scientists have endeavored to develop variants of both enzymes, with the aim being to increase their precision and reduce off-target effects. The issue with SpCas9 is that the modified variants are often too large to "fit" in the delivery system adopted for inserting gene therapies into patients, known as adeno-associated viral (AAV) vectors.SaCas9 is advantageous over SpCas9 in that it can be easily packaged into the AAV vectors for delivering gene-editing contents in vivo. However, at present, there is no SaCas9 variant that possesses high accuracy in genome-wide editing. Until now.Now meet SaCas9-HFIn the new study published in Proceedings of the National Academy of Sciences (PNAS), a research team led by Zheng Zongli, Assistant Professor of Department of Biomedical Sciences at CityU and the Ming Wai Lau Centre for Reparative Medicine of the Karolinska Institute in Hong Kong, and Shi Jiahai, Assistant Professor of Department of Biomedical Sciences at CityU, has successfully engineered SaCas9-HF, a CRISPR Cas9 variant which has demonstrated high accuracy in genome-wide targeting in human cells without compromising on-target efficiency.In the study, the scientists conducted an extensive evaluation of 24 targeted human genetic locations comparing the original (known as wild-type) SaCas9, and the new variant, SaCas9-HF. They discovered that for targets with highly similar sequences in the genome (and therefore often disposed to off-target editing by wild-type Cas9), SaCas9-HF decreased the off-target activity by ~90%. When assessing targets that had relatively less off-targeting editing by wild-type SaCas9, the SaCas9-HF enzyme produced little to no detectable off-target effects.

"Our development of this new SaCas9 provides an alternative to the wild-type Cas9 toolbox, where highly precise genome editing is needed. It will be particularly useful for future gene therapy using AAV vectors to deliver genome editing 'drug' in vivo and would be compatible with the latest 'prime editing' CRISPR platform, which can 'search-and-replace' the targeted genes," said Dr Zheng.Reference: Tan et al. 2019. Rationally engineered Staphylococcus aureus Cas9 nucleases with high genome-wide specificity. Proceedings of the National Aacademy of Sciences (PNAS). DOI: https://doi.org/10.1073/pnas.1906843116

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A Highly Precise Cas9 Enzyme, SaCas9-HF, Is Added to the CRISPR Toolbox - Technology Networks

All right, lets do this one last time. My name is CRISPR. I was made from a bacterial defense system, and for years Ive been the one and only gene editing wunderkind. Im pretty sure you know the rest. Im relatively cheap to make, easy to wield, and snip out genes pretty on target. Im going into clinical trials. Im reviving the entire field of gene therapy. Theres only one CRISPR. And youre looking at it.

Well, just as Spider-Man was way off, so is the idea of a single CRISPR to rule them all. This month, Dr. David Liu at the Broad Institute of MIT and Harvard in Cambridge, MA, introduced an upgrade that in theory may correct nearly 90 percent of all disease-causing genetic variations. Rather than simply deactivating a gene, CRISPR-based prime editing is a true search-and-replace editor for the human genome. With a single version, it can change individual DNA letters, delete letters, or insert blocks of new letters into the genome, with minimal damage to the DNA strand.

For now, prime editing has only been tested in cultured cells. But its efficacy is off the charts. Early experiments found it could correct single-letter misspellings in sickle cell disease, snip out four superfluous letters that underlie Tay-Sachs, and insert three missing letters to correct a genomic typo that leads to cystic fibrosis. In all, the tool worked remarkably well in over 175 edits in both human and mouse cells.

The excitement has been palpable, said Dr. Fyodor Urnov at the University of California, Berkeley, who was not involved in the research. I cant overstate the significance of this.

Given all of the existing CRISPR upgrades, why are scientists head over heels about prime editing?

CRISPR 1.0 generally refers to the classic version, which snips open the double helix to get rid of a certain gene. But as a tool, todays CRISPR is less like genetic scissors and more similar to a Swiss Army knife, one that scientists keep on improving. There are variants that, rather than destroying a gene, insert one or change one genetic letter to another, or ones that can target thousands of genetic spots at the same time. There are also spin-offs that hunt down RNAthe messenger that carries DNAs genetic code to the greater cellular universe, rather than the genetic code itself. Its truly a CRISPR multiverse out there.

Yet for all of CRISPRs upgrades, the tool has serious issues. For one, its very rough on the genome. Cas9, the protein scissor component of CRISPR, doesnt surgically cut out a gene. Rather, editing is in fact the cell detecting damage to the double helix, and trying its best to patch the broken strands back up. Just as scars form on our skin, this process can often introduce errors in the repairing processadding or missing a letter or two. Scientists often take advantage of this botched repair to destroy a gene that causes disease, or sneak in some additional code.

The problem? This process is basically genome vandalism, said Dr. George Church, a CRISPR pioneer at Harvard who wasnt involved in the new work. Its great when the repair goes according to plan; when it doesnt, the repair can introduce unwantedor downright dangerousmutations.

Lius idea for prime editing grew from his work on base editors. Here, the CRISPR machinery doesnt chop up the double helix. Rather, it uses the blood hound guide RNA to shuttle a new protein component to the target DNA sequence. This component then performs a single letter swap: C to T, or G to A.

Although considered much safer than traditional cut-and-glue CRISPR, base editors are limited in the number of genetic diseases they can treat. Its like editing on a broken keyboardsome misspellings just cant be fixed.

Prime editing circumvents these problems by heavily upgrading both components. The altered Cas9, for example, only snips a single strand of the double helix, rather than chomping through both. The new guide, pegRNA, both tethers the entire machinery to the target site, and encodes the desired edit.

Then comes the third component that magically ties everything together: a protein dubbed reverse transcriptase, which can make DNA sequences based on the blueprint in pegRNA, to insert into the nicked target site.

Still confused? Picture the DNA double helix as a laddertwo strands with connecting rungs in the middle. Prime editing cuts one strand using its neutered Cas9. This creates an opening for the other two components to insert a new gene into the severed spot; meanwhile, the original DNA sequence is snipped off. Now, rather than the original X, X (for example), the cell has X, Y.

The prime editor then performs a second snip at the opposing, non-edited strand. This alerts the cell of DNA damage, which it then tries to fixusing the new gene as a template. The end result is the cell goes from disease-causing X, X to normal, healthy Y, Y.

Several reasons.

One, because it doesnt cut both DNA strands, it doesnt immediately activate the cells repair system that is prone to errors. This means that scientists have far better control over the type of edit they want, and its no longer left to chance.

Two, prime is remarkably multi-purpose. Previously, the consensus among genome scientists was that a separate CRISPR tool was required for each specific type of edit: delete a gene, insert new DNA code, or DNA letter substitutions. In contrast, prime can achieve all three functions without additional modification. For experiments, it means less setup. For development into gene therapy, it means less overhead investment.

Three, prime editing can swap any of the DNA letters into any other, meaning it can now target an enormous amount of inherited diseases. For example, sickle cell disease, which causes oxygen-carrying blood cells to deform into sharp sickle-like shapes, requires changing a T into an A at a precise spot. Base editors cant do that. Prime editing can. Thats about 7,000 genetic disorders now amenable to gene therapy.

Four, prime editing also works in cells that no longer divide to renew themselves, such as neurons and muscle cells. Because these cells cant pass on their therapeutic DNA edit to daughter cells, to fix genetic deficits scientists have to be able to efficiently correct mutations in a large population. With prime editing, thats now possible.

Finally, prime editing can remove an exact number of letters from a given spot on the genome, at least up to 80. This allows scientists to precisely dictate the DNA sequences they want out, rather than relying on chance.

Early experiments with prime editing in cells show the tool is incredibly accurate. Off-target nicks were below 10 percent, and less than one-tenth of edited cells had unwanted changes to their genome, compared to up to 90 percent for first-gen CRISPR systems.

Nevertheless, the tool will have to go through rigorous testing before its widely accepted. Working in a few types of human cells is one thing; having it perform equally well inside a living body is something else completely. Most of primes tricks so far can be replicated using CRISPR 1.0, though at lower efficacy and with higher chances of off-target failures. Unlike prime editing, however, the original version has years of experience and plenty of clinical trials underwaycongenital blindness, sickle cell diseaseto back it up.

Whats more, prime is massive in terms of molecular tools. Getting it into cells will be a struggle. Getting it to the brain, which is protected by a dense wall of cells, will be even harder. To get the editor to their target, scientists will likely rely on gene therapy, itself a budding industry.

If CRISPR is like scissors, base editors are like a pencil. Then you can think of prime editors like a word processor, capable of precise search and replace, said Liu. All will have rolesThis is the beginning rather than the end.

Image Credit:petarg/Shutterstock.com

More:
Everything You Need to Know About Superstar CRISPR Prime Editing - Singularity Hub

What happened

Shares of Crispr Therapeutics (NASDAQ:CRSP) jumped nearly 23% last month, according to data provided by S&P Global Market Intelligence. The biopharmaceutical company announced a quarterly update demonstrating multiple areas of progress. The lead drug candidate, CTX001, is enrolling patients at six global sites for a phase 1/2 trial in transfusion-dependent beta thalassemia (TDT) and at 10 global sites for a phase 1/2 trial in sickle cell disease. Preliminary results are expected to be released before the end of 2019, which could have a profound effect on the pharma stock.

The gene-editing pioneer ended September with nearly $630 million in cash, which will come in handy next year when the company expects to have up to five clinical trials ongoing simultaneously. That includes plans to initiate the first clinical trial of CTX120 as a treatment for multiple myeloma in the first half of 2020, which should be followed by multiple trials involving CTX130 in solid tumors and white blood cell cancers.

Image source: Getty Images.

It's certainly difficult for investors to argue against the continuous execution of Crispr Therapeutics. The company was the first using CRISPR gene-editing technology to enter clinical trials, continues to advance multiple assets through the rigors of preclinical work and closer to the clinic, and partners with outside companies to augment its own capabilities. There's the highly visible partnership with Vertex Pharmaceuticals for CTX001 and other pipeline assets, but that's far from the only collaboration.

Crispr Therapeutics previously created a joint venture with Bayer, called Casebia Therapeutics, although control of the start-up will revert to Crispr before the end of 2019. Casebia will focus on programs in hemophilia, eye disorders, and autoimmune diseases. Bayer will have opt-in rights for multiple drug candidates.

Crispr is also collaborating with ViaCyte to develop a cellular medicine for treating type 1 diabetes, and with KSQ Therapeutics to develop CAR-T drug candidates with enhanced allogeneicity (read: grown from a single cell line and able to be used in any individual, in contrast to the strict donor matching required for current CAR-T medicines).

There's a long way to go before the company proves CRISPR gene editing can live up to the hype in human therapeutics, but the pioneer is the best positioned among its peer group. While there are major flaws with CRISPR gene editing that could keep the initial tools from ever being commercialized, Crispr Therapeutics is taking a "softer" approach with ex vivo engineering of white blood cells in blood disorders and for immuno-oncology. Whether the approach yields success remains to be seen, but investors will get their first glimpse of the potential when preliminary results from CTX001 trials are announced in the coming months.

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Here's Why Crispr Therapeutics Gained 22.8% in October - Motley Fool

REPORTING FOR 2019-11-08 | WCX19.ORG: We have done an in-depth analysis of how YGYI has been trading over the last 2 weeks and the past day especially. On its latest session, Youngevity International, Inc. ($YGYI) opened at 4.17, reaching a high of 4.3645 and a low of 4.17 before closing at a price of 4.28. There was a total volume of 40813.

VOLUME INDICATORS FOR YOUNGEVITY INTERNATIONAL, INC. ($YGYI): We saw an accumulation-distribution index of 48.46941, an on-balance volume of -4.21, chaikin money flow of 1.0 and a force index of 0.01053. There was an ease of movement rating of -0.00099, a volume-price trend of -0.48511 and a negative volume index of 1000.0.

VOLATILITY INDICATORS FOR YOUNGEVITY INTERNATIONAL, INC. ($YGYI): We noted an average true range of 0.19856, bolinger bands of 4.18124, an upper bollinger band of 4.14646, lower bollinger band of 4.17, a bollinger high band indicator of 1.0, bollinger low band indicator of 1.0, a central keltner channel of 4.23483, high band keltner channel of 4.04033, low band keltner channel of 4.42933, a high band keltner channel indicator of 1.0 and a low band keltner channel indicator of 1.0. There was a donchian channel high band of 4.17, a donchian channel low band of 4.17, a donchian channel high band indicator of 1.0, and a donchian channel low band indicator of 1.0.

TREND INDICATORS FOR YOUNGEVITY INTERNATIONAL, INC. ($YGYI): We calculated a Moving Average Convergence Divergence (MACD) of -0.00028, a MACD signal of -0.00015, a MACD difference of -0.00012, a fast Exponential Moving Average (EMA) indicator of 4.17, a slow Exponential Moving Average (EMA) indicator of 4.17, an Average Directional Movement Index (ADX) of unknown, an ADX positive of 20.0, an ADX negative of 20.0, a positive Vortex Indicator (VI) of 1.0, a negative VI of 1.0, a trend vortex difference of 0.1596, a trix of -7.37562, a Mass Index (MI) of 1.0, a Commodity Channel Index (CCI) of 66.66667, a Detrended Price Oscillator (DPO) of 0.55384, a KST Oscillator (KST) of -117.24453 and a KST Oscillator (KST Signal) of -117.24453 (leaving a KST difference of -0.65095). We also found an Ichimoku rating of 4.26725, an Ichimoku B rating of 4.26725, a Ichimoku visual trend A of 4.84938, an Ichimoku visual trend B of 4.712, an Aroon Indicator (AI) up of 4.0 and an AI indicator down of 4.0. That left a difference of -4.0.

MOMENTUM INDICATORS FOR YOUNGEVITY INTERNATIONAL, INC. ($YGYI): We found a Relative Strength Index (RSI) of 50.0, a Money Flow Index (MFI) of 34.96838, a True Strength Index (TSI) of -100.0, an ultimate oscillator of -54.12626, a stochastic oscillator of 100.0, a stochastic oscillator signal of 100.0, a Williams %R rating of 1326.2069 and an awesome oscillator of 0.04847.

RETURNS FOR YOUNGEVITY INTERNATIONAL, INC. ($YGYI): There was a daily return of -11.72445, a daily log return of -0.2954 and a cumulative return of -0.29496.

What the heck does all of this mean? If you are new to technical analysis, the above may be gibberish to you, and thats OK (though we do advise learning these things). The bottom line is that AS OF 2019-11-08 (if you are reading this later, the analysis will be out of date), here is what our deep analysis of technical indicators are telling us for Youngevity International, Inc. ($YGYI)

DISCLAIMER: We are not registered investment advisers and the above analysis should be taken at face value only. We strongly advise against buying or selling Youngevity International, Inc. ($YGYI) based solely on our analysis above, and are not responsible for any losses that you may incur if you choose make any investment decisions based on the above.

Continued here:
CRISPR Therapeutics AG ($CRSP): Caution Is Advised (2019-11-08) - WCX19

CRISPR has been making waves with its success in fighting rare genetic diseases, but could it also help turn bacteriums machinery against itself? In the era of superbugs, scientists are hopeful the technology can be a game-changer. Meanwhile, GSK has announced a late-stage study for its new antibiotic to fight urinary tract infections and gonorrhea.

The New York Times:Is Crispr The Next Antibiotic?For decades, scientists and doctors have treated common bacterial and viral infections with fairly blunt therapies. If you developed a sinus infection or a stomach bug, you would likely be given a broad-spectrum antibiotic that would clear out many different types of bacteria. Antiviral drugs help treat viral illnesses in much the same way, by hindering the pathogens ability to reproduce and spread in the body. (Sheikh, 10/28)

Reuters:GlaxoSmithKline Starts Late-Stage Trial For Experimental AntibioticGlaxoSmithKline Plc said on Monday it has begun a late-stage study testing its experimental antibiotic in patients with urinary tract infection and gonorrhoea, a type of sexually transmitted infection. The antibiotic, gepotidacin, is the first of a new class of drugs and is expected to treat the two common infections caused by bacteria - identified as antibiotic resistant threats by U.S. health regulators. (10/28)

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Could CRISPR Technology Rise As A Hero In The Era Of Antibiotic Resistance? - Kaiser Health News

Recently, there has been a shift in societys view of genetic modification and its potential applications in the fight against climate change. This has led to a call for changes in our current policies from farmers and MPs alike. However, due to the Green Partys current stance on this topic, New Zealand is unable to utilise genetic modification for anything that is not laboratory-based.

I am a member of the Emerging Scientists for Climate Action society, which involves students from universities all over New Zealand. We are writing an open letter to the Greens to encourage them to review their stance on genetic modification and the current laws and regulations around genetic engineering. Our overarching goal to tackle climate change aligns with the Greens, and they are in a position to make positive change. We have 155 signatures from emerging scientists (aged under 30) in support.

[Editors note: Deborah Paull is studying for a Masters of Science in Microbiology at the University of Canterbury.]

Genetic modification is a controversial topic, and there is much misunderstanding about its techniques and applications. Genetic modification (aka genetic engineering) uses gene editing technologies and knowledge of genetics to make changes in an organism for a specific outcome. For example, a plant could be genetically modified to grow bigger to produce a higher yield.

Read full, original article: Time to break the stigma on genetic modification, for the sake of the climate

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Young scientists urge New Zealand's Green Party to embrace CRISPR for 'sake of the climate' - Genetic Literacy Project

CRISPR Therapeutics AG (CRSP) came out with quarterly earnings of $2.40 per share, beating the Zacks Consensus Estimate of a loss of $0.95 per share. This compares to loss of $1.07 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of 352.63%. A quarter ago, it was expected that this company would post a loss of $0.81 per share when it actually produced a loss of $1.01, delivering a surprise of -24.69%.

Over the last four quarters, the company has surpassed consensus EPS estimates just once.

CRISPR Therapeutics AG, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $211.93 million for the quarter ended September 2019, surpassing the Zacks Consensus Estimate by 3,252.23%. This compares to year-ago revenues of $0.56 million. The company has topped consensus revenue estimates just once over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

CRISPR Therapeutics AG shares have added about 39.5% since the beginning of the year versus the S&P 500's gain of 20.6%.

What's Next for CRISPR Therapeutics AG?

While CRISPR Therapeutics AG has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for CRISPR Therapeutics AG was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.98 on $6.58 million in revenues for the coming quarter and -$3.85 on $13.83 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 29% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research

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CRISPR Therapeutics AG (CRSP) Q3 Earnings and Revenues Surpass Estimates - Yahoo Finance

CAMBRIDGE, Mass.--(BUSINESS WIRE)--KSQ Therapeutics, a biotechnology company using CRISPR technology to enable the companys powerful discovery engine to achieve higher probabilities of success in drug development, today announced two upcoming presentations at leading scientific immuno-oncology congresses. The data demonstrate the capabilities of the companys proprietary CRISPRomics discovery engine, which allows genome-scale, in vivo validated, unbiased drug discovery.

There is a significant need for next-generation immuno-oncology therapies as the majority of cancer patients today experience an insufficient response to PD-1/PD-L1 therapies. The data we will be sharing demonstrate the potential of our CRISPRomics discovery platform to systematically identify and validate new cancer therapies for patients with PD-1 refractory solid tumors, said Frank Stegmeier, Ph.D., Chief Scientific Officer at KSQ Therapeutics. KSQ was founded on the premise that CRISPR-enabled functional genomics can improve on current approaches to drug discovery and, taken together, these poster presentations describing the output of our genome-scale in vivo T-cell screens show that our platform can do this with a high degree of precision and quality, pointing the direction towards promising avenues of drug development.

Presentations include:

About KSQ Therapeutics

KSQ Therapeutics is using CRISPR technology to enable the companys powerful discovery engine to achieve higher probabilities of success in drug development. The company is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer, across multiple drug modalities including targeted therapies, adoptive cell therapies and immuno-therapies. KSQs proprietary CRISPRomics discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies, and the company is located in Cambridge, Massachusetts. For more information, please visit the companys website at http://www.ksqtx.com.

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KSQ Therapeutics to Present First Data from its Proprietary CRISPRomics Discovery Engine - Business Wire

First reported consecutive in vivo gene knockout and insertion achieves therapeutically relevant results in an alpha-1 antitrypsin deficiency mouse model

Inserted highly active WT1-TCR into the endogenous TCR locus for potential improved treatments for hematological and solid malignancies

CAMBRIDGE, Mass., Oct. 24, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology is presenting one oral presentation and four poster presentations at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) meeting taking place October 22-25, 2019, in Barcelona, Spain.

We are excited to share progress across Intellias in vivo and ex vivo programs at this important scientific venue, said Laura Sepp-Lorenzino, Ph.D., chief scientific officer, Intellia Therapeutics. Our data shows the complexity of the edits we are able to make with CRISPR/Cas9, while achieving important therapeutically relevant results. We are building on the success of our modular platform now having demonstrated consecutive targeted knockout and insertion genome edits in preclinical studies. Additionally, we presented data from our engineered cell therapy program, which continues to demonstrate the use of CRISPR/Cas9 for combined knockout and targeted integration in human T cells.

Intellia Demonstrates Consecutive In Vivo Genome Editing in Alpha-1 Antitrypsin Deficiency Mouse Model

Intellias oral presentation highlights its alpha-1 antitrypsin deficiency (AATD) study showing that consecutive dosing of two distinct lipid nanoparticle (LNP) formulations, in adultmice, achieves two targeted genome editing events, resulting in knocking out the faulty gene and restoring therapeutic levels of normal alpha-1 antitrypsin protein (hAAT). Intellias approach for AATD uses a modular hybrid delivery system combining a non-viral LNP which encapsulates CRISPR/Cas9 with an adeno-associated virus (AAV) carrying donor DNA template. Compared to traditional viral-based delivery of gene editing components, Intellias LNP delivery system can overcome the inherent limitations of immunogenicity to facilitate multiple in vivo gene editing events.

In a mouse model harboring the human PiZ allele, the most severe genetic defect in AATD patients, Intellia first reduced expression of the defective protein using gene knockout. Three weeks following the PiZ allele knockout, Intellia inserted the normal human alpha-1 antitrypsin gene, resulting in stable (throughout 12 weeks of observation), therapeutically relevant circulating protein levels. In the study, a sustained reduction of the circulating PiZ protein levels of >98% was observed for over 15 weeks. This is the first in vivo demonstration of a non-viral delivery platform, enabling a consecutive dosing approach for achieving multiple genome edits in the same tissue of the same animal. Intellias oral presentation, titled In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression, will be given by Anthony Forget, Ph.D., on October 25, 2019. This presentationwill be available on Intellias website at http://www.intelliatx.com.

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Intellias Poster Presentations

WT1-Specific TCR Engineered Cell Therapy Studies

Intellia presented new in vitro data showing that CRISPR/Cas9-mediated genome editing for in locus insertion, combined with endogenous T Cell Receptor (TCR) knockout, leads to significant reduction in mispairing of endogenous and transferred TCR chains. This approach is expected to generate transgenic-TCR (tg-TCR) T cell therapies for hematological cancers and solid tumors. Results demonstrate a highly efficient reduction of >98% in endogenous TCR and chains while reaching >70% insertion rates of tg-TCRs without further purification. The poster titled Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In Locus Insertion Combined with Endogenous TCR Knockout, was presented on October 24, 2019, by Birgit Schultes, Ph.D.

Researchers also presented in vitro data showing that a library of WT1-specific TCRs were generated, several of which Intellia is currently evaluating as part of its lead engineered cell therapy program targeting Acute Myeloid Leukemia (AML). This presentation, Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia, was presented on October 23, 2019 by Intellias collaborator, Erica Carnevale, Ph.D., IRCCS Ospedale San Raffaele.

Primary Hyperoxaluria Study

Intellia showed the continued progression of its modular platform capability using CRISPR/Cas9 to knockout either hydroxyacid oxidase 1 (Hao1) or lactate dehydrogenase A (Ldha), leading to a dose-dependent and persistent reduction of urinary oxalate levels in a Primary Hyperoxaluria Type 1 (PH1) mouse model. Data shows Ldha gene disruption also decreased LDH enzyme activity in the liver and did not impair the disposition of lactate in either wild type or renally-impaired mice. These results highlight the potential of editing genes in the glyoxylate detoxification pathway using a non-viral delivery approach as a one-time treatment option for PH1. These data were presented as a poster, titled CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria, by Sean Burns, M.D., on October 24, 2019.

Off-Target Screening Platform

Intellia demonstrated its approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Results from targeted off-target sequencing in edited cells showed that biochemical off-target discovery approaches were the most sensitive and accurate. These data were presented as a poster on October 23, 2019, titled In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics, by Dan OConnell, Ph.D.

About Intellia Therapeutics

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

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms such as its AATD program; develop our proprietary LNP-AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

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

Intellia Contacts:

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

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

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Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) - Yahoo...

Ninety-seven percent of potential new cancer drugs never make it to market, dropping out of clinical trials when they dont meet measures of safety or efficacy.

Why that is, we dont really know. But I think that this extremely high failure rate suggests that there are some fundamental issues in how new drug targets are studied and how new drugs are characterized, said molecular biologist Jason Sheltzer, PhD, an Independent Fellow at the Cold Spring Harbor Laboratory on Long Island, NY.

He decided to investigate, and uncovered the potential power of publishing negative evidence. The work fits in with Open Access week here at Public Library of Science.

CRISPR Improves Precision

The team reports inScience Translational Medicineon using the gene editing tool CRISPR-Cas9 to test whether 10 experimental cancer drugs work exactly how their developers predicted they would. And they found a tunnel vision in the way that drugs are targeted that might explain why certain patients do not respond as hoped.

Like an arrow that hits a tree rather than the bullseye, some cancer drugs may not actually reach their targets but many studies werent designed to reveal this. And so when results look promising, the drug candidate progresses through the FDA labyrinth.

Sheltzers strategy was straightforward: use CRISPR to remove the purported target, and if the drug still works, then the target isnt really the target. Perhaps preclinical research that identified a molecule as the drug target was halted, the scientists concluding success, when something fit the bill.

The researchers tested drugs that are either in clinical trials or once were, or are in preclinical studies (animals or human cells) not cancer drugs currently on the market. The experiments were done on standard cell lines from cancer patients.

The idea for many of these drugs is that they block the function of a certain protein in cancer cells. We showed that most of these drugs dont work by blocking the function of the protein that they were reported to block, Sheltzer explained.

Using CRISPR provided greater precision in interrogating potential drug targets than the older method, RNA interference. RNAi knocks down gene expression rather than snipping out a gene like CRISPR can.

Might a small molecule bind more than one type of target, like shooting arrows that hit trees and bushes as well as the bullseye? And sometimes what seems to be a valid drug target in vitro isnt exactly what happens in a body.

But a drug can make it to market without anyone knowing exactly how it works. Thats the case for selective serotonin reuptake inhibitor (SSRI) anti-depressants. The cartoons in ads depict neuromuscular junctions with the drug keeping serotonin in synapses longer by binding the reuptake proteins, presumably offsetting a deficit behind the symptoms. But googling SSRIsreturns the exact mechanism of actionof SSRIs is unknown.

From Slash-and-Burn to Hitting Targets

The new cancer drugs work in a few ways. Some of them zero in on molecules specific to cancer cells. These include:

These targeted drugs offer an alternative or adjunct to traditional drugs that broadly kill many types of rapidly-dividing cells, not just the cancer cells.

The targeted drugs began with Herceptinin 1998, its inventors recently honored with a Lasker award. The FDA approved another hugely successful targeted cancer drug, Gleevec, in just a few months in 2001. Today melodramatic ads pitch the new arsenal of cancer treatments: Zelboraf, Tafinlar, Keytruda, Opdivo.

But targeted drugs can fail if a new mutation alters the target or cancer cells find an alternate pathway that hikes cell division rate.

The researchers took a dual experimental approach based on logic:

Part of the confusion, I think, is semantic. Sometimes we deem a chemical interaction off-target if it doesnt do what we designed it to. Maybe our expectations were wrong. To be more unbiased, some researchers alter the language, calling the reliance of a cancer cell on a particular protein an addiction and investigating to seek druggable cancer dependencies.

The teams work indicates that what was deemed on-target may really be off-target, and vice versa. Perhaps its time to retire those terms.

The First Drug Tested

Earlier, Sheltzer investigated a protein called MELK, to which a company, OncoTherapy Science, is developing an inhibitor, called OTS167. Because MELK (maternal embryonic leucine zipper kinase) is abundant in many tumor types, it was presumed to be essential for their growth and therefore a drug target. But when CRISPR removed the gene that encodes MELK protein, nothing happened.

To our great surprise, when we eliminated these proteins from the cancer cells, they didnt die. The cancer cells continued to grow just fine, in spite of what had previously been published. They just didnt care about MELK, Sheltzer said.

The group published the findings on MELK in 2017, in eLIFE, raising the possibility that OTS167 is perhaps barking up the wrong tree. The drug candidate is in a phase 1(safety) trial for solid tumors and is recruiting for a phase 1trial for triple negative and metastatic breast cancer.

The MELK story inspired the group to use their genetic target-deconvolution strategy to see whether 10 other drugs were actually hitting their supposed targets. About a thousand cancer patients in total are taking one of these drugs in clinical trials.

Another Misguided Drug Reveals a Novel Target

In the new paper, the investigators question another drug, OTS964, being developed to treat certain lung and breast cancers. In the process, theyve discovered a new druggable cancer target.

RNAi had indicated that OTS964 targets a protein called PBK. But CRISPR told a different story cells with PBK gone still succumbed to the drug. It turns out that the interaction with PBK has nothing to do with how the drug actually kills cancer cells, Sheltzer said.

To find out how the PBK-targeting drug works, the researchers applied huge amounts of it to cancer cells and then gave the cells time to acquire mutations that would enable them to resist the drug. Cancer genomes are inherently unstable, mutating often. When a mutation renders a cell resistant to a drug, that cell then has an advantage and soon takes over the tumor.

Discovering how a cell circumvents a drug is priceless information.

The resistance experiments revealed that the cancer cell vulnerability that candidate drug OTS964 taps into isnt PBK after all, but a gene that encodes the protein CDK11. Its a cyclin-dependent kinase, an enzyme that is part of a pathway that leads to cell division.

The FDA has already approved CDK4/6 inhibitors, starting with Ibrance, in February 2015, to treat certain types of breast cancer. CDK11 is a brand new target. And thats potentially huge.

Whats Next?

At a news conference the researchers addressed concerns that their findings will affect people already taking targeted cancer drugs but they maintained that their work did not discover any approved drugs that were hitting trees instead of bullseyes.

But what about ongoing clinical trials for cancer drugs?

Sheltzer tried to alert folks running the trials. I filed a FOIA with the FDA to try to get additional information on the safety and efficacy of these drugs. The FDA declined to share that data, and said that it was a trade secret up until the point that these drugs received FDA approval.

He contacted companies sponsoring clinical trials too, but they wouldnt disclose any information either.

I think that the secrecy and the opacity in this drug development process really hurt scientific progress. A lot of drugs tested in cancer patients tragically dont help cancer patients. If this kind of evidence was routinely collected before drugs entered clinical trials, we might be able to do a better job assigning patients to therapies that are most likely to provide some benefit. With this knowledge, I believe we can better fulfill the promise of precision medicine, Sheltzer said.

The drug companies would do well to pay more attention to basic scientists who figure out how things work or dont work like Sheltzer. Using CRISPR can enable researchers to do a better job finding cancers central genes and a better job validating a drugs on-target mechanism of action. We think that that kind of preclinical foundation will help clinicians design better clinical trials to decrease the failure rate of new drugs, Sheltzer concluded.

Originally posted here:
When the Target Isn't Really the Target: One Way Cancer Drugs Fall Out of Clinical Trials | DNA Science Blog - PLoS Blogs

Phenotypic screening is gaining new momentum in drug discovery with the hope that this approach will improve the success rate of drug approval.1 In this article we look at some of the latest screening tools and their applications.

This is illustrated by their recent study with Dr Ayman Zen where the team developed a high-content imaging screen using the endothelial tube formation assay, miniaturized to a 384-well plate format. Screening with an annotated chemical library of 1,280 bioactive small molecules identified a retinoid agonist, Tazarotene, that enhanced in vitro angiogenesis and wound healing in vivo. This high content screen identified an already FDA-approved small molecule that could be potentially exploited in regenerative medicine.3

Immuno-oncology: Pushing the Frontier of Discovery Through Advanced High Throughput Flow Cytometry

Immuno-oncology encompasses a number of approaches with one common thread: they harness the bodys own immune system against cancer.

Download this article to learn how advanced throughput flow cytometry overcomes these challenges to drive forward innovation in the immuno-oncology field.

Ebner is currently working in collaboration with recent Nobel-Prize winner Peter Ratcliffe, alongside scientists at Edinburgh University and MIT, to model hypoxia in glioblastoma. Hypoxia is a problem with some glioblastomas as it protects cells from radiotherapy treatment. Our aim is to use Peters expertise to help us set up an assay that mimics real tumor hypoxia. Then if we can identify small compounds that alter that hypoxic condition we can make the glioma cells more susceptible to either radiotherapy or temozolomide or some other treatment combination.

The labs main readout is high-content imaging, using fluorescent microscopy that can take many thousands of pictures. This approach utilizes different labels and harnesses software that automates the image analysis. The image analysis is set by the biologists but then it's applied across the entire screen. Its lower throughput than plate-based readout, but you get a lot more information out of the images, says Ebner. Increasingly, high content imaging is moving towards using AI and deep learning where you're trying to draw out even more information than the primary phenotype that you were looking at.

Indeed, a recent study using CRISPR-Cas9 mutagenesis showed that the proteins targeted by many cancer drugs currently in clinical development are non-essential for tumor growth, despite evidence to the contrary from previous studies using RNAi and small molecule inhibitors.4 In addition, the efficacy of the drugs tested was unaffected when CRISPR was used to knockout its assumed target suggesting that many are eliciting their anticancer activity through off-target effects.

The other benefit of CRISPR is that its extremely flexible, says Pettitt. This means you can expand the range of cell line models, for example, that you can screen in. The key reason why RNAi was such a popular technology, and now CRISPR is, is that you can basically knock out a gene by synthesizing just a short piece of RNA, he explains. CRISPR guides are very easy to synthesize, you can do it in a very high throughput setting, and you can design customized libraries to knock out every gene in the genome or a particular set of genes. As long as you can get the CRISPR machinery into your cells, it works very reliably.

The classic CRISPR (CRISPR-Cas9) system comprises a nuclease called Cas9 which you can program with a short RNA (20 nucleotides). The RNA will direct the nuclease to a certain site in the genome that matches and the nuclease will cleave the genome at that point. Repair of that double-strand break results in small insertions and deletions that result in knock out of a gene. But theres now more evolved applications of the technology emerging.

I think it's possible to be very creative with CRISPR in a way that it isnt with RNAi, says Pettitt. With RNAi you can really only shut genes off, but with CRISPR as well as making random mutations to knock out genes - you can also precisely edit genes if you provide a template region with a mutation with it. This can be incorporated into the target site for CRISPR so you can introduce the specific mutation youre interested in.

One such example is the problem with BRCA1 mutations: its important to be able to functionally classify whether these mutations are benign or pathogenic. A recent study used CRISPR to test 96.5% of all possible single-nucleotide variants (SNVs) in exons that encode functionally critical domains of BRCA1 and found over 400 non-functional missense SNVs were identified, as well as around 300 SNVs that disrupt expression. This knowledge will immediately aid clinical interpretation of BRCA1 genetic test results.5 In another study,6 Pettitt and colleagues used genome-wide CRISPR-Cas9 mutagenesis screens to identify the mutated forms of PARP that cause in vitro and in vivo PARP inhibitor resistance, and found that these mutations are also tolerated in cells with a pathogenic BRCA1 mutation resulting in a different profile of sensitivity to chemotherapy drugs compared with other types of PARP inhibitor resistance.

You couldnt screen at that level of detail using RNAi, where you design custom CRISPR that targets many different regions of the same gene and you can figure out which domains of the protein are important for your phenotype of interest, says Pettitt.

There are other evolutions of CRISPR now being developed as screens. For example, if you mutate the nuclease activity of Cas9, it still retains its ability to localize to the target site, so you can fuse Cas9 to transcriptional activators or repressors, and screen for transcriptional repression with CRISPR, as well as knock-out screens, says Pettitt. Theres also a whole range of CRISPR tools being developed that will edit bases by causing missense mutations rather than insertions or deletions, or causing methylation of DNA, or bringing in fluorescent proteins so you can visualize where the DNA sequences in the cells are. Its a measure of how flexible and useful CRISPR is in comparison to RNAi.

So will CRISPR be the one technology that everyone turns to for phenotypic screening in future? Im a firm believer that no technology answers every question, says Ebner. CRISPR is amazing, its use as a therapeutic or biologic is the stuff of science fiction. But as a tool for target identification, it comes with one important caveat. CRISPR knockout means exactly that it removes the potential protein that would otherwise be in the mix. Thats very different from a small compound inhibiting a protein that is still able to form a complex or that is just not active. Its the perfect example of a brilliant technology that is transformative, but it's not perfect. No technology is perfect.

References

1. Zheng W, Thorne N and McKew JC. Phenotypic screens as a renewed approach for drug discovery. Drug Discov. Today 2013; 18: 1067-1073.

2. Horvath P, Aulner N, Bickle M, et al. Screening out irrelevant cell-based models of disease. Nat Rev Drug Discov. 2016 Nov;15(11):751-769. doi: 10.1038/nrd.2016.175. Epub 2016 Sep 12.

3. Al Haj Zen A, Nawrot DA, Howarth A, et al. The Retinoid Agonist Tazarotene Promotes Angiogenesis and Wound Healing. Mol Ther. 2016 Oct;24(10):1745-1759. doi: 10.1038/mt.2016.153.

4.Lin et al. Off-target toxicity is a common mechanism of action of cancer drugs undergoing clinical trials. Science Translat Med. 2019; 11: (509). doi: 10.1126/scitranslmed.aaw8412

5.Findlay GM, Daza RM, Martin B et al. Accurate classification of BRCA1 variants with saturation genome editing. Nature. 2018 Oct; 562(7726): 217222. doi: 10.1038/s41586-018-0461-z

6.Pettitt et al. Genome-wide and high-density CRISPRCas9 screens identify point mutations in PARP1 causing PARP inhibitor resistance. Nat Commun. 2018 May 10;9(1):1849. doi: 10.1038/s41467-018-03917-2.

The rest is here:
Phenotypic Screening Advances in Technologies and Techniques - Technology Networks

[He Jiankuis CRISPR babies] brought to the surface common misunderstandings even among scientists and ethicists thatreproductive usesof this genome-modifying tool have therapeutic value, will treat people with genetic disorders, will save lives, and will eradicate disease. None of those are true.

Imagine an individual or couple at high risk for creating a child with a serious genetic disease. They have the following simplified range of options:

Create a genetically related child in the time-honored fashion who will be at high risk for the genetic disease.

Create a genetically related child using CRISPR who will be at very low risk for the genetic disease.

Create no genetically related child.

The existence of option C undermines the claim that rCRISPR applications are lifesaving or curative.

Individuals have a choice in the matter of creating children at high risk of genetic disease: They can choose option C. Here is a different way of seeing the point that rCRISPR is not morally urgent because it does not involve a child whose existence, or illness, is inevitable.

Read full, original post: Using CRISPR to edit eggs, sperm, or embryos does not save lives

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Viewpoint: Why CRISPR embryo editing is not 'morally urgent': No one has to have a child - Genetic Literacy Project

You probably know from experience that there is not as much information on small-cap companies as there is on large companies. Of course, this makes it really hard and difficult for individual investors to make proper and accurate analysis of certain small-cap companies. However, well-known and successful hedge fund managers like Jeff Ubben, George Soros and Seth Klarman hold the necessary resources and abilities to conduct an extensive stock analysis on small-cap stocks, which enable them to make millions of dollars by identifying potential winners within the small-cap galaxy of stocks. This represents the main reason why Insider Monkey takes notice of the hedge fund activity in these overlooked stocks.

CRISPR Therapeutics AG (NASDAQ:CRSP) was in 13 hedge funds' portfolios at the end of June. CRSP shareholders have witnessed a decrease in support from the world's most elite money managers of late. There were 14 hedge funds in our database with CRSP positions at the end of the previous quarter. Our calculations also showed that CRSP isn't among the 30 most popular stocks among hedge funds(view the video below). Video: Click the image to watch our video about the top 5 most popular hedge fund stocks.

5 Most Popular Stocks Among Hedge Funds

So, why do we pay attention to hedge fund sentiment before making any investment decisions? Our research has shown that hedge funds' small-cap stock picks managed to beat the market by double digits annually between 1999 and 2016, but the margin of outperformance has been declining in recent years. Nevertheless, we were still able to identify in advance a select group of hedge fund holdings that outperformed the market by 40 percentage points since May 2014 through May 30, 2019 (see the details here). We were also able to identify in advance a select group of hedge fund holdings that underperformed the market by 10 percentage points annually between 2006 and 2017. Interestingly the margin of underperformance of these stocks has been increasing in recent years. Investors who are long the market and short these stocks would have returned more than 27% annually between 2015 and 2017. We have been tracking and sharing the list of these stocks since February 2017 in our quarterly newsletter. Even if you aren't comfortable with shorting stocks, you should at least avoid initiating long positions in our short portfolio.

Oleg Nodelman EcoR1 Capital

Unlike former hedge manager, Dr. Steve Sjuggerud, who is convinced Dow will soar past 40000, our long-short investment strategy doesn't rely on bull markets to deliver double digit returns. We only rely on hedge fund buy/sell signals. We're going to take a look at the recent hedge fund action surrounding CRISPR Therapeutics AG (NASDAQ:CRSP).

Heading into the third quarter of 2019, a total of 13 of the hedge funds tracked by Insider Monkey held long positions in this stock, a change of -7% from the first quarter of 2019. By comparison, 17 hedge funds held shares or bullish call options in CRSP a year ago. With hedge funds' sentiment swirling, there exists a few noteworthy hedge fund managers who were boosting their holdings considerably (or already accumulated large positions).

No of Hedge Funds with CRSP Positions

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Among these funds, EcoR1 Capital held the most valuable stake in CRISPR Therapeutics AG (NASDAQ:CRSP), which was worth $75.8 million at the end of the second quarter. On the second spot was Cormorant Asset Management which amassed $33 million worth of shares. Moreover, Farallon Capital, Clough Capital Partners, and Valiant Capital were also bullish on CRISPR Therapeutics AG (NASDAQ:CRSP), allocating a large percentage of their portfolios to this stock.

Since CRISPR Therapeutics AG (NASDAQ:CRSP) has experienced declining sentiment from hedge fund managers, we can see that there exists a select few hedge funds that elected to cut their entire stakes in the second quarter. It's worth mentioning that Steven Boyd's Armistice Capital said goodbye to the biggest investment of the 750 funds monitored by Insider Monkey, comprising about $2.9 million in stock. Noam Gottesman's fund, GLG Partners, also said goodbye to its stock, about $1.8 million worth. These bearish behaviors are intriguing to say the least, as aggregate hedge fund interest fell by 1 funds in the second quarter.

Let's also examine hedge fund activity in other stocks similar to CRISPR Therapeutics AG (NASDAQ:CRSP). We will take a look at Box, Inc. (NYSE:BOX), MGE Energy, Inc. (NASDAQ:MGEE), Independent Bank Corp (NASDAQ:INDB), and AMN Healthcare Services Inc (NYSE:AMN). This group of stocks' market values are closest to CRSP's market value.

[table] Ticker, No of HFs with positions, Total Value of HF Positions (x1000), Change in HF Position BOX,27,395491,-5 MGEE,13,53798,4 INDB,11,16780,5 AMN,14,109685,3 Average,16.25,143939,1.75 [/table]

View table hereif you experience formatting issues.

As you can see these stocks had an average of 16.25 hedge funds with bullish positions and the average amount invested in these stocks was $144 million. That figure was $180 million in CRSP's case. Box, Inc. (NYSE:BOX) is the most popular stock in this table. On the other hand Independent Bank Corp (NASDAQ:INDB) is the least popular one with only 11 bullish hedge fund positions. CRISPR Therapeutics AG (NASDAQ:CRSP) is not the least popular stock in this group but hedge fund interest is still below average. This is a slightly negative signal and we'd rather spend our time researching stocks that hedge funds are piling on. Our calculations showed that top 20 most popular stocks among hedge funds returned 24.4% in 2019 through September 30th and outperformed the S&P 500 ETF (SPY) by 4 percentage points. Unfortunately CRSP wasn't nearly as popular as these 20 stocks (hedge fund sentiment was quite bearish); CRSP investors were disappointed as the stock returned -13% during the third quarter and underperformed the market. If you are interested in investing in large cap stocks with huge upside potential, you should check out the top 20 most popular stocks among hedge funds as many of these stocks already outperformed the market so far in 2019.

Disclosure: None. This article was originally published at Insider Monkey.

Related Content

Original post:
Is CRISPR Therapeutics AG (CRSP) Going To Burn These Hedge Funds ? - Yahoo Finance

23 Oct 2019

A multi-institutional group, including members of the Tau Consortium, unveiled a stem cell tool kit for scientists studying primary tauopathies. In the November 12 issue of Stem Cell Reports, researchers co-led by Celeste Karch ofWashington University, St. Louis, and Alison Goate and Sally Temple of Icahn School of Medicine in New York, describe a collection of fibroblasts, induced pluripotent stem cells, and neural precursor cells. The cells come from 140 skin samples, some given by donors with richly documented clinical histories who carry pathogenic MAPT mutations or risk variants. Others come from noncarrier family members, patients with a sporadic tauopathy, and cognitively normal controls. The set includes induced pluripotent stem cell lines from 31 donors and 21 CRISPR-engineered isogenic lines. The cells are available to other researchers for study.

These types of high-quality repositories are becoming increasingly important for the scientific community, Clive Svendsen of the Cedars-Sinai Medical Center in Los Angeles wrote to Alzforum.

This is the way the field is going, agreed Lawrence Golbe of CurePSP, New York. Golbes organization funds research into progressive nuclear palsy (PSP) and related disorders, and collaborates with the Tau Consortium on other projects. Enthusiastic about the resources potential, Golbe hopes CurePSP grantees will get an automatic pass to use the cells.

Choice Mutations. Cells in the new iPSC collection carry some of the most common MAPT mutations, covering a wide range of clinical and neuropathological phenotypes of frontotemporal lobe dementia (FTLD)-Tau. [Courtesy of Karch et al., 2019.]

Tauopathies have proven difficult to study in animal models, in part because unlike other neuropathologies, they seem to afflict only humans (Heuer et al., 2012). Moreover, while adult human brains express approximately equal amounts of the tau spliced isoforms 3R and 4R, rodents produce almost exclusively 4R (Trabzuni et al., 2012). This is problematic. For example, leading proposals to explain how tau mutations cause disease point to abnormalities in splicing and microtubule binding, which differ between isoforms. The models we had been focusing on were not capturing the complexity of MAPT in human cells, said first author Karch. As a result, human induced pluripotent stem cells (iPSCs) have been gaining popularity in the field. The NINDS Human Cell and Data Repository is helping meet the demand by offering iPSC lines derived from 10 patients harboring MAPT mutations.

However, Karch and her collaborators think the field could benefit from a larger and more diverse collection of human cells, including isogenic iPSC lines. To accomplish this, they collected skin samples from 140 people carrying MAPT pathogenic mutations or risk variants, non-mutation carriers, and patients with sporadic PSP or corticobasal syndrome (CBS), most with comprehensive clinical histories. Although a few cells came from the NINDS repository, most came from patients participating in longitudinal studies at the Memory and Aging Center at the University of California, San Francisco, and the Knight Alzheimer Disease Research Center at WashU. The clinical records of most of these patients include detailed neurological and neuropathological workups, as well as fluid biomarkers and neuroimaging data collected from MRI, A-PET, and tau-PET studies.

To capture a broad range of phenotypes associated with some of the most common MAPT mutations, the authors created 36 fibroblast lines and 29 iPSC lines from individuals carrying the P301L, S305I,IVS10+16, V337M, G389R, and R406W mutations, as well as from carriers of the A152T variant, which increases the risk for both PSP and CBS (image above). The latter could be particularly useful for dissecting the mechanisms that underlie the phenotypic differences between the two diseases. The researchers also obtained iPSC lines from two noncarrier family members, and two people who suffered from autopsy-confirmed sporadic PSP. In addition, they stored fibroblast lines from 12 patients with sporadic PSP, five with CBS, 10 with a mixed PSP/CBS presentation, and 69 cognitively normal controls.

Biopsies are available for 27 of the 31 patients whose cells were used to generate iPSCs, and autopsy data for seven, including the two cases of sporadic PSP.

Importantly, the researchers edited 21 iPSC lines using CRISPR/Cas 9. They corrected cells with these mutations: MAPT IVS10+16,P301L, S305I, R406W, and V337M. Conversely, they inserted into control iPSCs these mutations: R5H, P301L,G389R, S305I, or S305S.

The authors also created a stem cell line carrying MAPT P301S,a mutation commonly overexpressed in tauopathy mouse models but not present in the available donors, by editing the P301L line. Isogenic lines are so powerful, particularly in these diseases which are so variable in their onset and progression, even within the same family, said Karch. Gnter Hglinger and Tabea Strauss at the German Center for Neurodegenerative Disease (DZNE) in Munich agreed. Having a pool of cell lines with different disease-linked mutations and risk variants from several individuals and their isogenic control cells is an excellent resource for the research community to enlighten disease mechanisms, they wrote (full comment below).

Several of the reported lines have already starred in recent studies of tauopathy mechanisms and candidate therapies (e.g., Sep 2019 conference news; Nakamura et al., 2019; Hernandez et al., 2019; Silva et al., 2019).

Karch and colleagues have partially differentiated some of the iPSCs and stored them as neural progenitor cells (NPCs), so that researchers can relatively easily thaw, expand, and differentiate them into neurons. These NPCs have proved useful for large-scale functional-genomics studies, proteomics, and genetic modifier screens (e.g., Cheng et al., 2017; Boselli et al., 2017;Tian et al., 2019).

In addition, the authors inserted a neurogenin-2 transgene into two healthy controls and two MAPT mutant stem cells, P301L and R406W. Neurogenin-2 enables low-cost, large-scale differentiation of stem cells into homogenous excitatory neurons. These transgenic cells are particularly useful for high-throughput drug screens (Wang et al., 2017; Sohn et al., 2019).

Researchers can request all the reported cells online at http://neuralsci.org/tau. They must provide a summary of experimental plans, an institutional material transfer agreement, and a nominal fee to cover maintenance and distribution costs. Karch said the process resembles that of the Coriell Institute and the NINDS repository. Our goal is to share with as few hurdles as possible, she said.

While the authors are still reprogramming fibroblasts they have already collected, they also plan to add more causative mutations, generate more isogenic lines, and obtain more cells from members of the same families to help shed light on phenotypic variability. In addition, Karch said, she hopes repository users will resubmit lines with new modifications they generate.

Jeffrey Rothstein, Johns Hopkins University, Baltimore, welcomed the new resource. I think it is great they have assembled this collection, he said. Rothstein founded and co-directs the Answer ALS research project, which has amassed 600 iPSC lines from controls and patients with amyotrophic lateral sclerosis (ALS).

Rothstein suggested the tauopathy collection may want to prioritize adding cells from donors with the most common form of disease, that is, sporadic. His group aims to generate 1,000 iPSC lines, with a large fraction representing sporadic diseasealso the most common form of ALSto identify the most prevalent disease subtypes. One strategy that has helped his group build their collection, he said, is using peripheral blood mononuclear cells instead of fibroblasts to create iPSCs. More donors are willing to donate blood than have a piece of skin punched out. In addition, iPSCs derived from blood cells are genetically more stable, he noted.

Rothstein emphasized the importance of assembling a large collection of healthy controls. Although isogenic controls are of great value, he cautioned they can be subject to artifacts. One problem is that the cell population can change due to selective pressures during CRISPR editing (Budde et al., 2017). To address this, Karch and colleagues are collecting not only modified iPSC clones, but also control clones that have gone through the editing pipeline but remain unmodified.

Stem-cell users studying tauopathies face another challenge: iPSC-derived neurons express primarily the fetal isoform of tau, 3R0N. However, citing a study that shows three-dimensional neuronal cultures switch to the adult profile relatively quickly (Miguel et al., 2019), Hglinger and Strauss wrote, [It] allows us to be optimistic that current challenges of this model system can be overcome in the future.Marina Chicurel

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Introducing: iPSC Collection from Tauopathy Patients - Alzforum

LEVERKUSEN, Germany and ZUG, Switzerland and CAMBRIDGE, Mass., Oct. 21, 2019 /PRNewswire/ -- CRISPR Therapeuticsand Bayer today announced proposed plans whereby Casebia Therapeutics, a joint venture between CRISPR Therapeutics and Bayer, would operate under the direct management of CRISPR Therapeutics. Upon closing of the transaction, Casebia Therapeutics would focus on the development of its lead programs in hemophilia, ophthalmology and autoimmune diseases, with Bayer having opt-in rights for two products at IND submission.

"The standalone Casebia entity combined the capabilities of CRISPR Therapeutics and Bayer to significantly advance the CRISPR/Cas9 gene-editing platform," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "As Casebia's programs have advanced beyond the discovery stage, we are evolving the operating model to leverage the manufacturing and clinical expertise of CRISPR Therapeutics to further accelerate these programs."

"We remain excited about the potential of cutting-edge CRISPR/Cas9 based therapies, which have the potential to create a whole new class of medicines," said Kemal Malik, Bayer board member for Innovation. "CRISPR Therapeutics has built the capabilities and expertise necessary to advance the Casebia programs to the next phase of development, and we look forward to continuing our collaboration with them."

The transaction is subject to negotiation and execution of definitive agreements as well as certain customary conditions. The companies anticipate the transaction will close in the fourth quarter of 2019.

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.

About Bayer and Leaps by BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Bayer's products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, Bayer aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2018, the Bayer global group employed around 117,000 people and had sales of 39.6 billion euros. Capital expenditures amounted to 2.6 billion euros, R&D expenses to 5.2 billion euros. For more information, go towww.bayer.com.

Leaps by Bayer, a unit of Bayer is investing into solutions to some of today's biggest problems. Previous Leaps investments into potentially breakthrough technologies include BlueRock Therapeutics (iPSC technology to cure cardiovascular and CNS diseases), Joyn Bio (probiotics for plants to enable for chemical fertilizer-free farming), Khloris (iPSC as cancer vaccination agents for potential prevention or cure), Century Therapeutics (iPSCs for allogeneic cell therapy of cancer), and Pyxis Oncology (antibody-based immunotherapies targeting the tumor microenvironment).

CRISPR Forward-Looking StatementThis press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics' expectations about any or all of the following: (i) the proposed transaction involving Casebia Therapeutics; (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas-9 gene editing technologies and therapies, including in hemophilia,ophthalmology and for autoimmune diseases; and (iii) CRISPR Therapeutics' ability to leverage manufacturing and clinical expertise to meaningfully advance certain Casebia Therapeutics programs. Without limiting the foregoing, the words "believes," "anticipates," "plans," "expects" and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: uncertainties inherent in corporate restructuring, including the expected timing for completion of such restructuring and the possibility that the parties will be unable to consummate any proposed transaction; the possibility that the expected synergies from CRISPR Therapeutics' manufacturing and clinical expertise will not be realized, or will not be realized within the expected time period; the risk that the businesses will not be integrated successfully; the initiation and completion of preclinical studies for CRISPR Therapeutics' and/or Casebia Therapeutics' product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics' technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics' most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Therapeutics Investor Contact:Susan Kimsusan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Jennifer PaganelliWCG on behalf of CRISPR347-658-8290jpaganelli@wcgworld.com

Bayer Media Contact:Chris Loder(201) 396-4325Christopher.loder@bayer.com

SOURCE Bayer

http://www.bayer.com

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CRISPR Therapeutics and Bayer Announce an Update on Casebia Therapeutics - PRNewswire

Mon, Oct 21, 2019 - 5:50 AM

New York

AS investors await results from the first US clinical trials of the gene-editing system known as Crispr, scientists are focused on finding ways to administer it directly into humans, according to the technology's co-inventor, Jennifer Doudna.

Right now, in studies using Crispr that have treated patients, researchers have had to extract their cells to be able to make edits to faulty DNA before infusing them back into the body for treatment.

Being able to do precise edits directly inside humans, animals or plants could open the door to new applications, Ms Doudna said.

"With advances and delivery techniques, it may be possible to do that kind of very highly efficient targeted genome editing in the patient, without having to remove cells, but actually to just do a treatment in the patient where the delivery vehicle takes the editing molecule to the right cells," she said in an interview before the Welch Foundation Conference on chemical research this week.

"Sounds fantastical today, but I think that's coming."

In essence, Crispr is a gene-editing system that can splice away parts of human DNA that make people susceptible to disease or defects. While it can be used in plants and animals, scientists are working on therapeutic applications that can offer a one-time cure for certain diseases.

Crispr Therapeutics AG was the first company to start a human trial back in February, and is due to report initial results by year-end.

Editas Medicine Inc is leading efforts in "in-vivo", or inside the body, testing and initiated a clinical study in July. Intellia Therapeutics Inc is expected to follow with its own study next year.

A safe delivery of Crispr directly into humans would shorten manufacturing times and offer new opportunities for the companies.

The biggest challenge is to find a way to deliver gene-editing molecules into specific cell types safely and efficiently, Ms Doudna said.

"That's kind of the next frontier," she added. "If we figure that out, it really does open the way to many, many more kinds of applications in genome editing than are possible today."

Crispr and Intellia Therapeutics have licensed their technology from the University of California at Berkeley, Ms Doudna's academic home, while Editas is using inventions from the Broad Institute in Massachusetts.

The two institutions are fighting over who was first to invent breakthrough gene-editing technology. Ms Doudna is a co-founder of Editas and other Crispr startups and is a scientific board member at Intellia.

The gene-editing field, which only recently entered human testing and has been plagued by research raising safety concerns, recently got some encouraging news.

Chinese researchers safely treated a man with leukemia and HIV using gene-edited stem cells, according to a report in the New England Journal of Medicine. While the attempt to cure his HIV failed, his cancer is in remission 19 months after the treatment, and the modified cells integrated into his body.

The case, which is the first detailed report in a major academic journal of how doctors are using Crispr in living patients, is an "important milestone" and suggests that gene editing will be "a safe technology and that the challenge now is to have it be really effective in different disease settings", Ms Doudna noted. BLOOMBERG

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Crispr's next frontier is in-human treatment, says co-inventor - The Business Times

A joint venture between gene-editing startup CRISPR Therapeutics and German pharmaceutical company Bayer will come under the control of CRISPR Therapeutics, according to the pairs proposed plans.

In an announcement published yesterday, October 21, CRISPR Therapeutics and Bayer proposed that the joint venture, Casebia Therapeutics, would focus on the development of its lead programmes in haemophilia, ophthalmology and autoimmune diseases.

The companies agreed to form Cambridge, Massachusetts-based Casebia Therapeutics in December 2015, with the aim of discovering, developing and commercialising new breakthrough therapeutics to cure blood disorders, blindness, and congenital heart diseases.

Samarth Kulkarni, CEO of CRISPR Therapeutics, said: As Casebia's programs have advanced beyond the discovery stage, we are evolving the operating model to leverage the manufacturing and clinical expertise of CRISPR Therapeutics to further accelerate these programmes.

Bayer will have opt-in rights for two products at investigational new drug application submissions.

Kemal Malik, Bayer board member for innovation, added: We remain excited about the potential of cutting-edge CRISPR/Cas9 based therapies, which have the potential to create a whole new class of medicines.

The transaction is expected to close in the fourth quarter of 2019.

In September, an alliance of companies that use gene-editing technologies (including CRISPR Therapeutics) released a bioethical framework, as controversy over gene-editing rages on.

The principles agree that the developers do not support germline gene editing (the process by which the genome of an individual is changed so that the change is heritable) in human clinical trials or for human implantation.

Last week, CRISPR Therapeutics announced that it had entered a licence agreement with biotech KSQ Therapeutics, gaining access to KSQs IP for editing certain novel gene targets in its allogeneic oncology cell therapy programmes.

KSQ gained access to CRISPR Therapeutics IP for editing novel gene targets identified by KSQ as part of its current and future cell programmes.

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Bayer, CRISPR Therapeutics, joint venture, CRISPR, gene-editing, haemophilia, ophthalmology, autoimmune diseases

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CRISPR Therapeutics to buy JV from Bayer - Life Sciences Intellectual Property Review

Russian scientist Denis Rebrikov recently revealed that he has started a gene-editing process that might eventually enable couples carrying the genetic mutation that causes deafness to give birth to children who can hear. The news was shared with Nature on 17 October via an e-mail.

According to Nature, the scientist mentioned in the e-mail that he will soon publish the results of his experiments, which involves testing the ability of CRISPR to repair the gene causing deafness GJB2 in cells taken from people that have the mutation. Rebrikov believes that the result will help to lay the groundwork for the clinical work. Rebrikov also added that he wants to help couples with unimpaired hearing to have a child such as these to have a child with the same mutation.

Rebrikov also mentioned that he has local review board's permission to do the research but it does not allow the transfer of the gene-edited eggs into the womb and pregnancy. The scientist also emphasized that he will not be going ahead without the approval from the Ministry of Health of the Russian Federation. "I will definitely not transfer an edited embryo without the permission of the regulator," he confirmed.

However, the chances of this happening seem to be really low as last week, the ministry released a statement where it mentioned that the production of gene-edited babies is premature. Rebrikov, however, is not ready to lose hope. He says, "it is hard to predict" when he'll get permission for it so till then all the necessary safety checks need to be undertaken.

Rebrikov has previously also announced that he intends to use the CRISPR tool for gene-edited babies resistant to HIV. At that time, the news came as a shock to all international researchers as people feared that he is following the Chinese scientist He Jiankui who previously announced the controversial birth of the world's first gene-edited babies twin girls.

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CRISPR scientist wants to edit genes that cause deafness but falls short of permission - International Business Times, Singapore Edition