Archive for the ‘Crispr’ Category

The most recent research will provide you with an overview of the global Plant Breeding and CRISPR Plants Market in general, as well as factors that may influence future growth, or lack thereof, as well as potential opportunities and current trends. This study examines the global markets structure, as well as market segmentation, growth rates, and revenue share comparisons. The market study reports research findings aid in the assessment of a range of essential variables, such as investment in a developing market, product success, and market share expansion.

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A revenue market size analysis, as well as market drivers, restraints, and opportunities, are all included in the study. The report also includes a picture of the industrys main competitors competitive landscape, as well as the top businesses percentage market share. This research looks into the Plant Breeding and CRISPR Plants Market in great detail. The research reports market estimates and predictions are based on extensive secondary research, primary interviews, and in-house expert opinions. The impact of different political, social, and economic factors, as well as current market conditions, on market growth, is examined in these market projections and estimates.

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The segmentation of the global Plant Breeding and CRISPR Plants Market in terms of the various regions and countries involved, as well as a breakdown of revenues, market shares, and possible expansion prospects, are all covered in this section. This research looks at revenue growth at the global, regional, and country levels, as well as recent industry trends in each sub-segment.

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COVID-19 Impact Analysis

The influence of COVID-19 on the Plant Breeding and CRISPR Plants market at the global and country levels is examined in this research report. The demand and supply side effects of the target market are considered in this study. Primary and secondary research, as well as private databases and a paid data source, were used in this study. Market players will benefit from the COVID-19 impact study as they implement pandemic mitigation strategy.

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The Plant Breeding and CRISPR Plants market study contains a chapter on major global market participants, which includes a review of the companys business, financial statements, product description, and strategic aims. The companies covered in the report can be customized to meet the needs of a client. This chapter will go through each of the major industry competitors and their present market position in detail.

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Plant Breeding and CRISPR Plants Market Analysis By Industry Size, Share, Revenue Growth and Demand Forecast To 2027 Energy Siren - Energy Siren

This week, were pleased to bring you a different version of Futurism, containing stories from the horizon of hedonism. Welcome toThe Science of Pleasure.In collaboration with our friends over atMEL Magazine, this week, well be bringing you stories from both publications about the pleasures of tomorrow, today.

Bonobos are a species of primate believed to be humankinds second-closest living ancestor. Thats intriguing, because zoologists have long observed that these distant cousins of ours are driven by a syrupy mixture of empathy and hedonism, lounging in simian cuddle puddles, caressing one another, kissing with tongue, and engaging in an awe-inspiring variety of bisexual encounters, sometimes with multiple partners, and often while screaming in pleasure.

Sounds pretty fun, doesnt it? Sure, party drugs like MDMA can temporarily flood your brain with oceans of empathy and euphoria, but they can be terrible for you if you take them too much, and the hangovers can be brutal. In other words, theres no drug thats gonna give you bonobo-brain safely and long term.

Relentless progress in the science of genetic engineering raises an interesting alternative, though: what if scientists gene-hacked humans, who already share around 98 percent of their genetic material with bonobos, so that wed be more affectionate, amorous and generally happy? What if you could take a CRISPR-like brain injection that permanently altered your cognition so that you could experience bonobo-like bliss?

If such a thing is theoretically possible, geneticists told us, its far beyond todays biotech capabilities. But eventually, if research continues to barrel ahead, those types of profound alterations to the human mind may well become a reality and if so, theyre likely to raise profound questions about what it means to be human.

I like to think that everything is in the realm of scientific possibility, said Esmerelda Casas-Silva, a biomedical researcher at the National Institutes of Health who cautioned that her musings on our strange question did not reflect the views of her employer. Its really just a matter of us figuring out how to do it.

Overall, she said, she believes that the idea would be tricky yet certainly possible.

If so, what a life it would be. A greater genetic propensity for peace and promiscuity, like that of the bonobos, doesnt sound so bad.

Famed scientist Carl Sagan and his spouse, Cosmos cocreator Ann Duyan, wrote extensively about bonobos in the 1993 book Shadows of Forgotten Ancestors: A Search for Who We Are.

Bonobos use sexual stimulation in everyday life for many purposes besides mere satisfaction of the erotic impulse, they wrote, including in exchange for food, as a way to resolve conflicts among same sex adults, and as a generic, all-purpose approach to social bonding and community organization.

Were we humans to become more like bonobos, militarism and excess aggression could become obsolete, as less authoritarian social structures characteristic of bonobo societies came to predominate. Socioeconomically, a bonobo way of life could embody the ethic of solidarity and cooperative decision-making over competition, domination or exploitation.

The kind of pleasures that bonobos enjoy are not decadent, said Susan Block, a sex therapist and the author of the 2014 book The Bonobo Way: The Evolution of Peace Through Pleasure. They dont spend any money. Maybe a few bananas, but they share those bananas.

Whether gene hacking ourselves to be as guileless and empathic as bonobos would be ethical, of course, is a different question entirely.

For one thing, the whole hypothetical smacks of eugenics. For another, even though the goal of gene therapy to make humans more like bonobos might nominally be to encourage a more equitable society, the ironic reality is that our actual world is rife with inequality, hierarchies, and unfair institutional arrangements.

In other words, itd probably be the wealthy and powerful who would seize the treatment for themselves or, chillingly, foist it on others in order to create a more obedient workforce, sort of similar to how in the 2018 satire film Sorry to Bother You, a parody of contemporary entrepreneurs named Steve Lift tries to use gene-modifying powder to create Equisapien human-horse hybrids capable of laboring harder and longer than people already do today.

We really shouldnt be in a hurry to make designer babies right now, Casas-Silva said. We have very incomplete information, and even if we do know exactly whats going to happen, whos to say what we should decide and what characteristics are best or not?

That point is larger than just the bonobo thought experiment, of course, and its also something the world is likely going to need to confront soon. A Chinese scientist named He Jiankui sparked a global controversy in 2018 when he announced that he had altered the genetic makeup of twin girls, using CRISPR to attempt to make them immune to HIV while they were still embryos and it later emerged that he had been involved in discussions to open a designer baby clinic as well.

If those types of clinics become a reality, itll likely only be affluent parents who can afford them, deepening existing social and economic divides.

Are we going to create even more disparities in health and different populations that are suffering because they dont have access to the same technologies as, you know, other people who might have enough funds to make superhuman babies? Casas-Silva asked. And we could make very dangerous, in my opinion, very dangerous future humans.

And, on a practical level, our anxious human brains keep the power plants running and the industrial agriculture machinery feeding everybody. If we spent all day lounging around and engaging in bacchanalia, that could easily fall apart, for better or worse.

In fact, Block points out that bonobos are a good case study in why giving yourself over to pleasure entirely might not be a great survival strategy.

Block told Futurism about an incident involving bonobos and common chimpanzees our closest living ancestors, for those keeping score in a German zoo during the bombing of Dresden. The zoo wasnt bombed, but the apes could hear the detonations and possibly felt some of the impact.

All the bonobos died of heart attacks, and all the common chimps didnt care, the sexologist said, adding that the zoo kept the former separated in captivity, meaning they werent together to calm each other down.

When youre empathetic, thats a double edged sword, isnt it? she said. Empathy can feel so good, but it also means that when you hear bombs dropping, even if theyre not dropping on you, you freak out. Its like its happening to you. Or if you see someone being hurt, you freak out.

If we do go wild with CRISPR, or whatever advanced tool it takes, to turn human beings into overly peaceable and amorous creatures, Casas-Silva cautions that we could end up falling in love incessantly, with everyone or everything. It might even preclude the intense and exclusionary monogamous love thats often the social norm.

I mean, that could be pretty devastating, Casas-Silva said. Theres a reason that our bodies and our minds decrease our feelings and our memories. Thats a safety measure for anyone whos fallen in love and had it not work out. You know, its a blessing that we can diminish those feelings of hurt and anxiety and everything that comes with a breakup over time.

But, even if we refrain from hacking away at our genes in the hopes of bonoboizing ourselves, Block maintains we still have a lot to learn from our sex positive comrades, including lessons in revaluing our natural bodies and pursuing mutual pleasure without guilt.

The idea that our natural self is Satan is killing us, she said. [The belief] that we are somehow superior, that we are angels, that we are not part of nature, that we are not part of this earth, that we can fly away, like angels maybe a couple people can, you know? Captain Kirk went up there, right? Ninety years old, okay. But most of us are here, and Im looking to improve the lives of those of us that are here on this beautiful, wild sexual planet Earth.

More on CRISPR:WHO Says Gene-Hacking Superhumans Should Be Illegal

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Excerpt from:
Could We Gene Hack Ourselves to Be Blissed Out Sex Maniacs, Like Our Distant Cousins the Bonobos? - Futurism

Vertex Pharmaceuticals will develop in vivo gene-editing therapies for two genetic diseases using Mammoth Biosciences CRISPR systems through a collaboration that could generate more than $695 million for Mammoth, the companies said today.

The companies is not disclosing which diseases they will target.

We are focused on developing in vivo gene-editing therapies in two indications for specific serious and/or life-threatening diseases with the Vertex team,Peter Nell, Mammoths Chief Business Officer and Head of Therapeutics Strategy, told GEN.

Mammoth and Vertex did say, however, that they will apply Mammoths CRISPR platform consisting of a proprietary toolbox of novel Cas enzymeswhat the company calls the largest toolbox of CRISPR proteins on earth.

These include Cas12, which targets double-stranded DNA;Cas13, which targets and recognizes single-stranded RNA; Cas14, which targets single-stranded DNA; and Cas, which is encoded exclusively in the genomes of huge bacteriophages.

Mammoth has exclusively licensed foundational IP around novel CRISPR Cas12, Cas13, Cas14, and Cas systems from the University of California, Berkeley, where, they were discovered in the lab of Nobel laureate and Berkeley-based researcher Jennifer Doudna, PhD.

Doudna is a co-founder of Mammoth Biosciencesalong with CEO Trevor Martin, PhD; Janice Chen, PhD, the companys CTO, who discussed her companys technology last year onGENs monthly GEN Live program; Lucas Harrington, Mammoths CSO; and Ashley Tehranchi, PhD, who served as CTO until May 2019.

Cas14 and Cas are the smallest known CRISPR systems. Their sizes530 amino acids for Cas14a and 757 amino acids for Cas-2are less than half those of commonly used variants of Cas9 [1368 amino acids for SpCas9] and Cas12 [1,300 amino acids for FnCas12], offering numerous potential advantages for the therapies Mammoth plans to develop, Martin told GEN Edge last month, after the company announced the completion of $195 million in new financing completed over the past year.

The additional financingconsisting of $150 million in Series D financing and a $45-million Series C round whose investors included Amazonbrought Mammoths total capital raised from investors to more than $255 million., propelling the company to a unicorn valuation of more than $1 billion.

In addition, Mammoth said, it is building out its IP portfolio by discovering novel CRISPR systems within and beyond the foundational work. The company has yet to disclose those systems or other Cas enzymes under development.

The combination of Mammoths unique technology with Vertexs unmatched experience in serious disease research and development will only accelerate programs with the goal of reaching patients with high unmet medical need, Nell added. We believe our novel ultra-small CRISPR systems have the potential to be game-changers when it comes to systemic and targeted delivery of in vivo gene-editing therapies.

CRISPR-edited therapies have been an area of focus for Vertex. Late last year, the company and CRISPR Therapeutics reported positive data from a pair of Phase I/II trials for their CRISPR-Cas9 gene-edited therapy CTX001 showing consistent and sustained positive response in 10 patients treated for a pair of blood disorders, sickle cell disease (SCD) and beta thalassemia.

The companies in April amended their collaboration agreement to give Vertex leadership in global development, manufacturing, and commercialization of CTX001 with support from CRISPR Therapeutics, in return for CRISPR receiving a $900 million upfront payment and a potential additional $200 million milestone payment upon CTX001 regulatory approval. Two months later, during the Joint European Hematology Association-American Society of Hematology (EHA-ASH) Symposium, researchers presented additional clinical data showing CTX001 to have delivered a consistent and sustained response to treatment in 22 patients in two ongoing Phase I/II trials.

We see tremendous potential for CTX001, Stuart A. Arbuckle, Vertexs executive vice president and chief commercial and operations officer, told analysts July 29 on the companys quarterly earnings call following release of second-quarter results. He cited an estimate of more than 150,000 patients in the United States and Europe, who have beta thalassemia, or sickle cell disease, approximately 32,000 of whom have severe disease; plus another 25,000 severe sickle cell disease patients, the vast majority of which were in the United States.

We believe that a gene-editing approach which holds the potential for a one-time curative treatment will be highly valued by patients, physicians, and payers, Arbuckle said. Consistent with our own internal market research, published physician surveys in the United States consistently indicate that they expect a quarter to a third of their sickle cell disease patients would be good candidates for a one-time curative approach using the current conditioning regimen, which is in line with the estimates of the numbers of severe patients.

With gentler conditioning regimens in the future, Arbuckle added, we expect CTX001 to be an attractive option for a much larger proportion of the 150,000 beta thalassemia and sickle cell disease patients.

To launch its collaboration with Mammoth, Vertex has agreed to pay the Brisbane, CA,-based company $41 million upfront, including an investment in the form of a convertible note, and up to $650 million in potential future payments tied to achieving research, development, and commercial milestones across two potential programs.

Mammoth is also eligible for tiered royalties from Boston-based Vertex on future net sales on any products that may result from the collaboration, the first one announced by Mammoth for the development of gene-edited therapies.

Vertex and Mammoth share the same commitment to developing therapies that have the potential to be transformative for people with serious diseases, stated David Altshuler, MD, PhD, Vertexs CSO. We look forward to expanding our cell and genetic therapies capabilities with the addition of Mammoths ultra-small CRISPR systems for in vivo genome editing, which will provide us with another set of tools to tackle many of the diseases were interested in.

Mammoth is also developing CRISPR-based diagnostics, having applied Cas12 in itsCOVID-19diagnostic effort which culminated in the SARS-CoV-2 RNA DETECTR Assay, a COVID-19 diagnostic for whichUCSF Health Clinical Laboratorieswasgranted an FDA Emergency Use Authorization (EUA)in August 2020.

The 45-minute test is designed to detect nucleic acid from SARS-CoV-2 in upper respiratory specimens. The test extracts, isolates, and purifies SARS-CoV-2 nucleic acid for simultaneous reverse transcription into cDNA, followed by amplification using loop-mediated amplification (RT-LAMP).

The SARS-CoV-2 RNA DETECTR Assay was co-developed by Mammoth through itspartnership with UCSF professor Charles Chiu, MD, PhD, who is also director of the UCSF-Abbott Viral Diagnostics and Discovery Center, and a member of the companys Scientific Advisory Board. Mammothin 2019exclusively licensed two U.S. patents granted to the regents of the University of California that cover CRISPR collateral cleavage diagnostic systems.

In July 2020, Mammoth won funding for its development of a scalable COVID-19 test, when the company wasawarded $23.1 millionof $248.7 million in contracts to the first seven lab-based and point-of-care tests diagnostics developersfunded through the NIHs Rapid Acceleration of Diagnostics (RADx) initiative. The testing system can be adapted to detect for other viruses, though Mammoth has not made public which ones.

Two months earlier in May 2020, Mammoth launched a collaboration with GlaxoSmithKlines GSK Consumer Healthcare to develop a handheld test designed to apply the DETECTR platform at point of need. Mammoth has disclosed few details since the initial announcement, with Martin saying last month: I cant say too much about it, but definitely weve made huge strides.

Original post:
Vertex, Mammoth Launch Up-to-$695M CRISPR Gene-Editing Collaboration - Genetic Engineering & Biotechnology News

Life goes on as gene-edited foods begin to hit the market. Japanese consumers have recently startedbuying tomatoes that fight high blood pressure, and Americans have been consuming soy engineered to produce high amounts of heart-healthy oils for a little over two years. Few people noticed these developments because, as scientists have said for a long time, the safety profile of a crop is not dictated by the breeding method that produced it. For all intents and purposes, it seems that food-safety regulators have done a reasonablejob of safeguarding public health against whatever hypothetical risks gene editing may pose.

But this has not stopped critics of genetic engineering from advocating for more federal oversight of CRISPR and othertechniquesused to make discrete changes to the genomes of plants, animals and other organisms we use for food or medicine. Over at The Conversation, a team of scientists recently made the case for tighter rules in Calling the latest gene technologies natural is a semantic distraction they must still be regulated.

Many scientists have defended gene editing, in part, by arguing that it simply mimics nature. A mutation that boosts the nutrient content of rice, for example, is the same whether it was induced by a plant breeder or some natural phenomenon. Indeed, the DNA of plants and animals we eat contains untold numbers of harmless, naturally occurringmutations. But The Conversation authors will have none of this:

Unfortunately, the risks from technology dont disappear by calling it natural... Proponents of deregulation of gene technology use the naturalness argument to make their case. But we argue this is not a good basis for deciding whether a technology should be regulated.

They have written a very long peer-reviewed article outlining a regulatory framework based on "scale of use."The ideais that the more widely a technology is implemented, the greater risk it may pose to human health and the environment, which necessitates regulatory "control points" to ensure its safe use. It's an interesting proposal, but it's plagued by several serious flaws.

Where's the data?

The most significant issue with a scale-based regulatory approachis that it's a reaction to risks that have never materialized. This isn't to say that a potentially harmful genetically engineered organism will never be commercialized. But if we're going to upend our biotechnology regulatory framework, we need to do so based on real-world evidence. Some experts have actually argued, based on decades of safety data, that the US over-regulates biotech products. As biologist and ACSHadvisorDr. Henry Miller and legal scholar John Cohrssen wrote recently in Nature:

After 35 years of real-world experience with genetically engineered plants and microorganisms, and countless risk-assessment experiments, it is past time to reevaluate the rationale for, and the costs and benefits of, the case-by-case reviews of genetically engineered products now required by the US Environmental Protection Agency (EPA), US Department of Agriculture (USDA) and US Food and Drug Administration (FDA).

The problem with scale

Real-world data aside for the moment, there are some theoretical problems with the scalabilitymodel as well. Theargument assumes thatrisks associated with gene editing proliferate as use of the technology expands, because each gene edit carries a certain level of risk. This is a false assumption, as plant geneticist Kevin Folta pointed out on a recent episode of the podcast we co-host (21 minute mark).

Scientists have a variety of tools with which to monitor and limit the effects of specific gene edits. For example, proteins known as anti-CRISPRs can be utilized to halt the gene-editing machinery so it makes only the changes we want it to. University of Toronto biochemist Karen Maxwell has explained how this could work in practice:

In genome editing applications, anti-CRISPRs may provide a valuable 'off switch for Cas9 activity for therapeutic uses and gene drives. One concern of CRISPR-Cas gene editing technology is the limited ability to control its activity after it has been delivered to the cell . which can lead to off-target mutations. Anti-CRISPRs can potentially be exploited to target Cas9 activity to particular tissues or organs, to particular points of the cell cycle, or to limit the amount of time it is active

Suffice it to say that these and other safeguards significantly alter the risk equation and weaken concerns about a gene-edits-gone-wild scenario. Parenthetically, scientists design these sorts of preventative measures as they develop more genetic engineering applications for widespread use. This is why the wide variety of cars in production today have safety features that would have been unheard of in years past.

Absurdity alert: The A-Bomb analogy

To bolster their argument, The Conversation authors made the following analogy:

Imagine if other technologies with the capacity to harm were governed by resemblance to nature. Should we deregulate nuclear bombs because the natural decay chain of uranium-238 also produces heat, gamma radiation and alpha and beta particles? We inherently recognize the fallacy of this logic. The technology risk equation is more complicated than a supercilious 'its just like nature' argument

If someone has to resort to this kind of rhetoric, the chances are excellent that their argument is weak. Fat Man and Little Boy, the bombs dropped on Japan in 1945, didn't destroy two cities because a nuclear physicist in New Mexico made a technical mistake. These weapons are designed to wreak havoc. Tomatoes bred to produce more of an amino acid, in contrast, are not.

The point of arguing that gene-editing techniques mimic natural processes isn't to assert that natural stuff is good; therefore, gene editing is also good. Instead, the point is to illustrate that inducing mutations in the genomes of plants and animals is not novel or uniquely risky. Even the overpriced products marketed as all-natural have been improved by mutations resulting from many years of plant breeding.

Nonetheless, some scientists have argued that reframing the gene-editing conversation in terms of risk vs benefit would be a smarter approach than making comparisons to nature. I agree with them, so let's start now. The benefits of employing gene editing to improve our food supply and treat disease far outweigh the potential risks, which we can mitigate. Very little about modern life is naturaland it's time we all got over it.

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CRISPR Revolution: Do We Need Tighter Gene-Editing Regulations? No - American Council on Science and Health

Published: Oct. 20, 2021 at 9:05 PM EDT

DAEJEON, South Korea, Oct. 20, 2021 /PRNewswire/ -- On September 2, 2021, a scientific paper from GenKOre on the new hypercompact CRISPR system called CRISPR/Cas12f-GE was published in an online issue of Nature Biotechnology (IF 54.9), one of the world's top-tier biotechnology journals.

For effective gene therapy, it is absolutely essential that the genetic payload be delivered to the desired location inside a patient's body. The best method for accomplishing this is to use the Adeno-Associated Virus (AAV) as a vector. AAV is a non-enveloped single-stranded DNA virus that penetrates cells that either divide or do not divide. AAV replicates only when a helper virus is present and thus it is non-pathogenic to humans. Because of these traits, AAV is an effective and practical method to deliver genes into various types of cells and is used as a vector in gene therapy. However, with the CRISPR/Cas9 technology, the size of the scissor gene is large and it is difficult to transport the gene inside the human body using the virus (AAV) carrier. For this reason, the clinical use of CRISPR/Cas9 as a gene therapy is very limited.

Because the size of the gene in the 'CRISPR/Cas12f-GE' system developed by GenKOre is one-third that of Cas9, it is an ideal payload for AAV delivery. GenKOre demonstrated its potential utility as the best gene therapy by improving the editing efficiency. In addition to high editing efficiency, it has proven to be effective and safe as gene scissor technology with respect to the off-target issue, a chronic weakness of CRISPR gene scissors.

The hypercompact CRISPR system Cas12f-GE has revealed its value as a therapeutic agent that can be widely used for developing medical treatments for patients who chronically suffer from life-long illnesses or diseases due to the lack of fundamental remedies.

GenKOre, which successfully developed this new technology, is a spin-off company of the Korea Research Institute of Bioscience & Biotechnology (KRIBB), a research institute funded by the South Korean government.

Dr. Yong-Sam Kim, CEO of GenKOre remarked, "I hope that our achievements based on our research will bring about a revolution in gene therapies utilizing this genome-editing tool. Our technology can be seen as a breakthrough by resolving the major obstacle linked to the original CRISPR technology, and I am optimistic that in tandem with existing gene scissors, our technology will contribute to the human health and welfare."

With the successful development of CRISPR/Cas12f-GE, GenKOre plans to step up its development of gene therapy and new products through its unique gene scissor technology and collaborate with other companies in becoming a market leader in the realm of gene therapy and new plant biotechnology

For more content on GenKOre's paper published online by Nature Biotechnology, refer to https://www.nature.com/articles/s41587-021-01009-z.

The website of GenKOre is http://www.genkore.com

View original content to download multimedia:

SOURCE GenKOre

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

Original post:
A Hypercompact CRISPR Technology Developed by GenKOre is deemed a Potential Game Changer in Gene Therapy. - WKYT

CAMBRIDGE, Mass., Oct. 21, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis. This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body. NTLA-2001 has the potential to be the first single-dose treatment for ATTR amyloidosis as it may be able to halt and reverse the devastating complications of this disease. ATTR amyloidosis is a rare condition that can impact a number of organs and tissues within the body through the accumulation of misfolded transthyretin (TTR) protein deposits.

Orphan drug designation underscores the FDAs recognition of NTLA-2001s potential promise as a single-dose, novel therapy for the treatment of ATTR amyloidosis, said Intellia President and Chief Executive Officer John Leonard, M.D. At Intellia, we are committed to advancing our modular genome editing platform to develop potentially curative treatment options for life-threatening diseases, and we look forward to working with the ATTR amyloidosis community and the FDA to bring a much-needed treatment option to patients.

NTLA-2001 is currently being studied in a Phase 1 trial in adults with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). In June 2021, Intellia and its collaborator Regeneron announced positive interim clinical results from the first two cohorts of this study. These results, which were published in the New England Journal of Medicine, represented the first-ever clinical data supporting the safety and efficacy of in vivo CRISPR genome editing in humans.

The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval. The decision by the FDA follows a March 2021 decision by the European Commission (EC) to also grant NTLA-2001 orphan drug designation for the treatment of ATTR amyloidosis.

Story continues

About Transthyretin (ATTR) Amyloidosis Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid deposits in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without any genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart.

About NTLA-2001Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first curative treatment for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or intravenously, to edit genes inside the human body. Intellias proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001s potential as a single-administration therapeutic. Interim Phase 1 clinical data released in June 2021 confirm substantial, dose-dependent reduction of TTR protein following a single dose of NTLA-2001. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron.

About Intellia TherapeuticsIntellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, Intellia is pursuing two primary approaches. The companys in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellias ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellias deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatweets.

Forward-Looking StatementsThis press release contains forward-looking statements of Intellia 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: being able to complete clinical studies for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis pursuant to its clinical trial applications (CTA), including submitting additional regulatory applications in other countries; ability to demonstrate effectiveness of NTLA-2001 in treating or reversing ATTR amyloidosis in patients; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; expectations of the potential impact of the coronavirus disease 2019 pandemic on strategy, future operations and timing of its clinical trials or IND submissions; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Regeneron, including its co-development programs for ATTR amyloidosis; and statements regarding the timing of regulatory filings regarding its development programs.

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 its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its 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 or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Regeneron or its other collaborations 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 the Securities and Exchange Commission (SEC). All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:Ian KarpSenior Vice President, Investor Relations and Corporate Communications+1-857-449-4175ian.karp@intelliatx.com

Lina LiDirector, Investor Relations+1-857-706-1612lina.li@intelliatx.com

Media:Lisa QuTen Bridge Communications+1-678-662-9166media@intelliatx.com lqu@tenbridgecommunications.com

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Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin...

Latest Study on Industrial Growth of Global CRISPR-Based Therapeutics Market 2021-2027. A detailed study accumulated to offer Latest insights about acute features of the CRISPR-Based Therapeutics market. The report contains different market predictions related to revenue size, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary and SWOT analysis.

The Major Players Covered in this Report: Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics & GE Healthcare Dharmacon

CRISPR-Based Therapeutics Market Study guarantees you to remain / stay advised higher than your competition. With Structured tables and figures examining the CRISPR-Based Therapeutics, the research document provides you a leading product, submarkets, revenue size and forecast to 2027. Comparatively is also classifies emerging as well as leaders in the industry.Click To get SAMPLE PDF of CRISPR-Based Therapeutics Market (Including Full TOC, Table & Figures) @https://www.htfmarketreport.com/sample-report/3176523-global-crispr-based-therapeutics-market-growth

This study also covers company profiling, specifications and product picture, sales, market share and contact information of various regional, international and local vendors of Global CRISPR-Based Therapeutics Market. The market proposition is frequently developing ahead with the rise in scientific innovation and M&A activities in the industry. Additionally, many local and regional vendors are offering specific application products for varied end-users. The new merchant applicants in the market are finding it hard to compete with the international vendors based on reliability, quality and modernism in technology.

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The titled segments and sub-section of the market are illuminated below:In-depth analysis of Global CRISPR-Based Therapeutics market segments by Types: , Genome Editing, Genetic Engineering, gRNA Database/Gene Librar, CRISPR Plasmid, Human Stem Cells, Genetically Modified Organisms/Crops & Cell Line EngineeringDetailed analysis of Global CRISPR-Based Therapeutics market segments by Applications: Biotechnology Companies, Pharmaceutical Companies, Academic Institutes & Research and Development Institutes

Major Key Players of the Market:Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics & GE Healthcare Dharmacon

Regional Analysis for Global CRISPR-Based Therapeutics Market: APAC (Japan, China, South Korea, Australia, India, and Rest of APAC; Rest of APAC is further segmented into Malaysia, Singapore, Indonesia, Thailand, New Zealand, Vietnam, and Sri Lanka) Europe (Germany, UK, France, Spain, Italy, Russia, Rest of Europe; Rest of Europe is further segmented into Belgium, Denmark, Austria, Norway, Sweden, The Netherlands, Poland, Czech Republic, Slovakia, Hungary, and Romania) North America (U.S., Canada, and Mexico) South America (Brazil, Chile, Argentina, Rest of South America) MEA (Saudi Arabia, UAE, South Africa)

Furthermore, the years considered for the study are as follows:Historical year 2015-2020Base year 2020Forecast period** 2021 to 2027 [** unless otherwise stated]

**Moreover, it will also include the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product services of key players.

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Key takeaways from the Global CRISPR-Based Therapeutics market report: Detailed considerate of CRISPR-Based Therapeutics market-particular drivers, Trends, constraints, Restraints, Opportunities and major micro markets. Comprehensive valuation of all prospects and threat in the In depth study of industry strategies for growth of the CRISPR-Based Therapeutics market-leading players. CRISPR-Based Therapeutics market latest innovations and major procedures. Favorable dip inside Vigorous high-tech and market latest trends remarkable the Market. Conclusive study about the growth conspiracy of CRISPR-Based Therapeutics market for forthcoming years.

What to Expect from this Report On CRISPR-Based Therapeutics Market:1. A comprehensive summary of several area distributions and the summary types of popular products in the CRISPR-Based Therapeutics Market.2. You can fix up the growing databases for your industry when you have info on the cost of the production, cost of the products, and cost of the production for the next future years.3. Thorough Evaluation the break-in for new companies who want to enter the CRISPR-Based Therapeutics Market.4. Exactly how do the most important companies and mid-level companies make income within the Market?5. Complete research on the overall development within the CRISPR-Based Therapeutics Market that helps you elect the product launch and overhaul growths.

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Detailed TOC of CRISPR-Based Therapeutics Market Research Report-

CRISPR-Based Therapeutics Introduction and Market Overview CRISPR-Based Therapeutics Market, by Application [Biotechnology Companies, Pharmaceutical Companies, Academic Institutes & Research and Development Institutes] CRISPR-Based Therapeutics Industry Chain Analysis CRISPR-Based Therapeutics Market, by Type [, Genome Editing, Genetic Engineering, gRNA Database/Gene Librar, CRISPR Plasmid, Human Stem Cells, Genetically Modified Organisms/Crops & Cell Line Engineering] Industry Manufacture, Consumption, Export, Import by Regions (2015-2020) Industry Value ($) by Region (2015-2020) CRISPR-Based Therapeutics Market Status and SWOT Analysis by Regions Major Region of CRISPR-Based Therapeutics Marketi) Global CRISPR-Based Therapeutics Salesii) Global CRISPR-Based Therapeutics Revenue & market share Major Companies List Conclusion

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, MINT, BRICS, G7, Western / Eastern Europe or Southeast Asia. Also, we can serve you with customize research services as HTF MI holds a database repository that includes public organizations and Millions of Privately held companies with expertise across various Industry domains.

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CRISPR-Based Therapeutics Market Analysis, Size, Strategic Assessment, Market Growth and Forecasts to 2027 Puck77 - Puck77

How successful of an investor is Cathie Wood? Consider that over the last five years, the two best-performing non-leveraged exchange-traded funds (ETFs) were her ARK Next Generation Internet ETF and ARK Innovation ETF. And her ARK Genomic Revolution ETF came in at No. 5.

The kinds of stocks that Wood likes aren't for everyone. They're often high-risk, potentially high-reward plays that are suitable only for aggressive investors. If you don't mind taking on considerable risk, here are three stocks Wood is buying that you might want to consider too.

Image source: Getty Images.

Both the ARK Innovation ETF and ARK Genomic Revolution ETF have scooped up more shares ofBeam Therapeutics (NASDAQ:BEAM) in October. The biotech stock is only one spot away from jumping into the top 10 holdings for the ARKG ETF.

Beam specialized in base editing. It's a type of gene editing that enables pinpoint rewriting of a specific letter in the genome. And Beam's approach could prove to be more important over the long run than other types of gene editing.

The company doesn't have any candidates in clinical testing yet. That could change relatively soon, though, with Beam on track to submit an Investigational New Drug (IND) application within the next couple of months for BEAM-101 in treating rare blood disorders beta-thalassemia and sickle cell disease.

Beam's market cap stands above $6 billion. That's a quite lofty valuation for a preclinical-stage biotech. However, if the company's base editing works in one indication, it could potentially be applied in many other indications. Wood obviously thinks the chances that will happen are worth placing a major bet on Beam Therapeutics.

Beam Therapeutics isn't the only gene-editing stock that Wood likes these days. Her ARK Innovation ETF and ARK Genomic Revolution ETF have also added to their positions in CRISPR Therapeutics (NASDAQ:CRSP) in recent weeks.

CRISPR Therapeutics could become the first biotech focused on CRISPR gene editing to have a shot at getting a product on the market. The company and its partner Vertex Pharmaceuticalshope to file for regulatory approvals of CTX001 in 2023. CTX001 is a CRISPR gene-editing therapy targeting beta-thalassemia and sickle cell disease.

Some investors were disappointed with the durability of response in CRISPR Therapeutics' phase 1 data for its off-the-shelf chimeric antigen receptor T cell (CAR-T) therapy CTX110. However, the day after those results were announced, Wood chose to buy more shares of the stock at a discount.

CRISPR Therapeutics' market cap of over $7 billion might be hard to swallow for cautious investors. But with the prospects of potentially launching its first product in 2024 and a still-promising pipeline of gene-editing therapies, the company could become a big player in healthcare over the long run.

There's no question thatTeladoc Health (NYSE:TDOC) remains one of Wood's favorite stocks. So far in October, three of her ARK ETFs have bought additional Teladoc shares. Teladoc ranks as the top holding of the ARK Genomic Revolution ETF. It's the second-largest position in the ARK Innovation ETF. The telehealth stock is even in the top 10 for the ARK Fintech Innovation ETF.

Some investors have soured on Teladoc this year due to concerns about slowing membership growth. However, the company continues to perform well overall. Visits have risen. Revenue per member per month is up. Utilization rates are climbing.

On the other hand, Wall Street hasn't given up on the stock. Analysts think that Teladoc could soar close to 40% over the next 12 months. This optimism about Teladoc from both Wood and Wall Street centers on the company's long-term prospects.

The virtual care market remains only in its early stages. Teladoc is the clear leader in the market. It also continues to launch new products and services that provide further competitive advantages. Of these three stocks being bought by Wood recently, Teladoc is the least risky while still offering strong growth prospects.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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3 Stocks Cathie Wood Is Buying That You Might Want to Consider Too - Motley Fool

CRISPR in agriculture market includes biological techniques that have been utilized in a wide variety of plant species to improve agricultural traits. Due to the deterioration of air quality, the health of soils, and adverse climate change CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) has been implemented to improve crop yields.

The agriculture sector is one of the potential sectors that utilize CRISPR technology to increase the production of crops. The CRISPR processes in which specified enzymes have been modified can remove DNA from a genome that is different from the genetically modified organisms (GMOs).

According to FSA (Federation of American Scientists), NIH had sanctioned funds with the value of approximately $1,155.4 million in 2020 respectively for the CRISPR related research activities and projects.

Report Consultant has published an innovative statistical data, titled as CRISPR in Agriculture market. This report has been aggregated with different market segments, such as applications, end-users and revenue. It focuses on the analysis of the existing market and upcoming innovations, to provide better insights for the businesses. This study includes the elaborative description of CRISPR in Agriculture market along with the different perspectives from various industry experts.

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Top Key Players:Bayer AG, Benson Hill Biosystems, Inc., Caribou Bioscience Inc., CRISPR Therapeutics, Horizon Discovery Group PLC, Intellia Therapeutics Inc., Yield10 Biosciences Inc., and so on.CRISPR in Agriculture Market By End-UserAcademic & Research InstitutesBiotech Companies

CRISPR in Agriculture Market By Target TypeCropsLivestockAquaculture

The genetic editing technology in CRISPR has brought multiple benefits to the food and agricultural industry that improves the productivity of fermentation processes. The development of abiotic stress-tolerant crop plants in the tropics by using the technology of CRISPR such as drought, soil salinity, heat stress that significantly limit the yield of crops across the globe. The CRISPR increases the demand of genome-editing for emerging diseases and pests in tropical regions that has opened a new opportunity for rapid development of disease resistant crop varieties by deletion of susceptibility.

Different regions, such as North America, Latin America, Japan, China, Asia Pacific, and India are considered to examine the facts of the leading key players. Through quantitative and qualitative analysis, this report has summarized about the significant pillars that can boost the performance of different industries.Get a Sample Copy of this CRISPR in Agriculture Market report now! @https://www.reportconsultant.com/request_sample.php?id=77685

CRISPR in Agriculture Market Research objectives1. To study and analyze the CRISPR in Agriculture market size by key regions/countries, product type and application, history data from 2016 to 2020, and forecast to 2028.2. To understand the structure of the CRISPR in Agriculture market by identifying its various sub-segments.3. To analyze the CRISPR in Agriculture concerning individual growth trends, prospects, and their contribution to the total market.4. To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).

5. To project the size of CRISPR in Agriculture sub-markets, concerning key regions (along with their respective key countries).6. To analyze competitive developments such as expansions, agreements, new product launches and acquisitions in the market.7. To strategically profile the key players and comprehensively analyze their growth strategies.

A substantial study of the market strategies, challenges, prospects for advancements along with a detailed introspection of prominent aspects affecting the market is included in this report. It also sheds light upon the ongoing developments in this field as well as prevailing strategies to thrive in such a continually evolving market. To conclude with, this report covers all the necessary financial, economic and social factors relevant to the industry, thus enhancing a readers ability to understand the information required to make an informed decision.Customization Service of the Report:Report Consultant provides customization of reports as per your need. This report can be personalized to meet your requirements. Get in touch with our sales team, who will guarantee you to get a report that suits your necessities.Contact us:Rebecca Parker(Report Consultant)Contact No: +81-368444299[emailprotected]www.reportconsultant.comAbout Report Consultant:Report Consultant A global leader in analytics, research and advisory that can assist you to renovate your business and modify your approach. With us, you will learn to take decisions intrepidly. We make sense of drawbacks, opportunities, circumstances, estimations and information using our experienced skills and verified methodologies.Our research reports will give you an exceptional experience of innovative solutions and outcomes. We have effectively steered businesses all over the world with our market research reports and are outstandingly positioned to lead digital transformations. Thus, we craft greater value for clients by presenting advanced opportunities in the global market.

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CRISPR in Agriculture Market to Exhibit Robust Growth in Forthcoming Period 2021- 2028 With Prominent Players: Bayer AG, Benson Hill Biosystems, Inc.,...

Detailed study and analysis of the Global CRISPR Technology Market highlights new trends in the CRISPR Technology industry and provides companies with trading insights. This study helps manufacturers, suppliers and investors, CEOs to identify opportunities and business optimization strategies to improve their value in the global CRISPR Technology market. Provides important information for well-known companies that are one of the top performing companies. The report provides comprehensive coverage of existing and potential markets as well as an assessment of competitiveness in changing market scenarios.

The report also presents data in the form of charts, tables and figures together with contact details and sales contact information for the major market players in the global market. There is a detailed overview of the competitive landscape of the global CRISPR Technology industry, with all the information gathered and deepened with the SWOT analysis. Opportunities for potential industrial growth have been discovered and the competition risks involved have also been structured.

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The segmentation chapters enable readers to understand aspects of the market such as its products, available technology and applications. These chapters are written to describe their development over the years and the course they are likely to take in the coming years. The research report also provides detailed information on new trends that may define the development of these segments in the coming years.

CRISPR Technology Market Segmentation:

CRISPR Technology Market, By Application (2016-2027)

CRISPR Technology Market, By Product (2016-2027)

Major Players Operating in the CRISPR Technology Market:

Company Profiles This is a very important section of the report that contains accurate and detailed profiles for the major players in the global CRISPR Technology market. It provides information on the main business, markets, gross margin, revenue, price, production and other factors that define the market development of the players studied in the CRISPR Technology market report.

Global CRISPR Technology Market: Regional Segments

The different section on regional segmentation gives the regional aspects of the worldwide CRISPR Technology market. This chapter describes the regulatory structure that is likely to impact the complete market. It highlights the political landscape in the market and predicts its influence on the CRISPR Technology market globally.

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The Study Objectives are:

This report includes the estimation of market size for value (million USD) and volume (K Units). Both top-down and bottom-up approaches have been used to estimate and validate the market size of CRISPR Technology market, to estimate the size of various other dependent submarkets in the overall market. Key players in the market have been identified through secondary research, and their market shares have been determined through primary and secondary research. All percentage shares, splits, and breakdowns have been determined using secondary sources and verified primary sources.

Some Major Points from Table of Contents:

Chapter 1. Research Methodology & Data Sources

Chapter 2. Executive Summary

Chapter 3. CRISPR Technology Market: Industry Analysis

Chapter 4. CRISPR Technology Market: Product Insights

Chapter 5. CRISPR Technology Market: Application Insights

Chapter 6. CRISPR Technology Market: Regional Insights

Chapter 7. CRISPR Technology Market: Competitive Landscape

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CRISPR Technology Market Size 2021 Key Strategies, Applications, Trends and Opportunities | Top Brands: Thermo Fisher Scientific, Merck KGaA,...

Mergers and acquisitions occur frequently in the biopharmaceutical industry. And there are often rumors of other deals that never materialize. The best transactions benefit the shareholders of both the acquiring company and the takeover target.In this Motley Fool Live video recorded on Oct. 13, Fool contributors Keith Speights and Brian Orelli discuss potential biotech buyouts that would be great for investors.

Keith Speights: All right, Brian. Now, let's take a speculative twist in our discussion about acquisitions. We've just talked about two deals that have been announced this week. But Brian, is there a biotech buyout that you would really like to see? Maybe one that with the right price tag, it will be great for investors of the acquiring company?

Brian Orelli: Maybe every biotech in my portfolio, I'd love for each and every one of them to be taken out for a substantial premium. I don't know. There's some people who say that they're disappointed because a company got taken out because they'd rather own the company and profit from the growth of that company individually.

But the way I see it, there's plenty of biotechs in the sea to reinvest those profits. If you're going to give me a quick up-front profit, I'm definitely going to take it and I'm not going to complain about it.

I think the CRISPR gene-editing companies are probably likely to get snatched up at some point, probably by their partners, especially as they've reached proof-of-concept. I'm thinking Intellia (NASDAQ:NTLA) potentially being acquired by Regeneron (NASDAQ:REGN), and CRISPR Therapeutics (NASDAQ:CRSP) potentially being acquired by Vertex Pharmaceuticals (NASDAQ:VRTX).

I think these would be great for the investors, mostly because I think maybe their valuations are a little inflated. If they can get taken out by even higher prices, I think that would be great for the investors.

I own Vertex so I certainly don't want Vertex overpaying for CRISPR Therapeutics. Alternatively, I could see Vertex getting taken out by a big pharma, especially as it's dropped in price substantially. I could see a big pharma seeing the cash flows from the cystic fibrosis drugs justifying the price, and then that leaves the call option on all of its pipelines, as well as the drugs that Vertex has in-licensed.

Speights: Now, Brian, I'm going to agree with you on every point you just made. No. 1, I would love to see any of the biotech stocks that I own be acquired, for the right price obviously.

No. 2, I agree with you that the CRISPR gene-editing companies are probably near the top of the list of potential acquisition targets over the next few years. I also agree with you that Vertex, if there's going to be one of those massive big pharma mergers like we've seen over the last 20 years, some of those massive deals, I think Vertex would be an excellent candidate to be acquired by a bigger company.

I'm not sure. We'll see if that happens, but I think it would be one of the top megamerger acquisition candidates that are on the market right now.

Orelli: I think a pharma that wanted a lot of cash flow, but also wanted to boost up their pipeline, Vertex probably has more external deals than it has internal candidates. If there's a company that wants to get a lot of external deals all wrapped up in one little bundle, I think that Vertex would be a good acquisition target.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Biotech Buyouts That Would Be Great for Investors - Motley Fool

-thalassemia is one of the most common autosomal recessive diseases in the world and researchers are seeking to produce a gene therapy to treat the condition. Scientists are applying a variety of strategies and techniques to correct the underlying genetic imbalance that causes -thalassemia, including using CRISPR to correct the mutated gene. The approach is promising, but scientists are still working to improve CRISPRs editing efficiency, which is still an open question. Consequently, CRISPRs ability to successfully correct mutations associated with -thalassemia is still uncertain. Therefore, researchers need to pair CRISPR editing protocols with a quality control tool such as droplet digital PCR (ddPCR) that accurately detects the presence of successful CRISPR edits.A promising yet complex gene editing approachRoughly 1.5% of the global population carries mutations associated with -thalassemia, with more than 60,000 new cases diagnosed every year. Unfortunately, scientists do not have a straightforward path towards addressing this condition at the genetic level. Adult hemoglobin is composed of two pairs of globin subunits, -globin and -globin, which must be expressed in equal numbers for hemoglobin to develop normally. People with -thalassemia harbor genetic mutations in the gene for -globin, HBB, that lead to downregulation of the gene. Free -globin, then forms toxic precipitates that impair red blood cell development and kill mature red blood cells. As a result, patients experience a wide range of severe symptoms, and the condition often leads to early death.Some research suggests that deleting the -globin gene, HBA, may improve outcomes. Introducing a healthy HBB gene via a lentiviral vector improves patients' clinical outcomes, but only if these patients already express some -globin. A research group based in France and Italy recently combined these two approaches: they used CRISPR to delete HBA and replace it with HBB in hopes of restoring the balance between the two hemoglobin subunits.Performing such an edit is a complex task. First, after designing a guide RNA (gRNA) that locates the gene that needs to be edited, scientists need to introduce it to ones cells using a viral vector. Then, the gRNA must identify the correct cutting sites flanking the HBA gene, while Cas9 must perform the cuts. The same gRNA must facilitate the insertion of the HBB gene at the same locus. This dual edit approach will not work if CRISPR does not successfully remove HBA and introduce the HBB gene in the same spot. Such a multifaceted edit requires rigorous quality control to ensure CRISPR performs the correct edits in the correct locations. This is where ddPCR technology comes in.Advantages of ddPCR assaysddPCR is a highly sensitive tool designed to detect and quantify rare genetic variants, and it can be used to detect outcomes of CRISPR editing. For example, ddPCR assays can detect CRISPR edits via both homology-directed repair (HDR) and nonhomologous end joining (NHEJ). It can also detect excisions and inversions independent of sequence length.ddPCR technology works by partitioning a sample into approximately 20,000 nL-sized droplets and running a separate PCR reaction in each one. Each droplet contains one or a few nucleic acid strands. If a droplet contains a strand featuring the target genetic sequence, that DNA will amplify, and the droplet will release a strong fluorescent signal. If a droplet does not contain the target sequence, the droplet will only emit weak fluorescence. By counting the strongly vs weakly fluorescent droplets, one can detect specific sequences with great sensitivity and measure the concentration of the target sequence in the original sample with great accuracy.Compared to next-generation sequencing, ddPCR is fast, inexpensive, and not labor-intensive, and it can detect rare events without being limited by read depth. ddPCR is also more sensitive and accurate than quantitative PCR (qPCR). While researchers must use a standard curve to interpret qPCR results, exposing the data to amplification bias, ddPCR quantifies genetic variants directly, without a standard curve, and thereby provides an absolute count.The abovementioned European group that developed the dual editing approach for treating -thalassemia used ddPCR assays to assess the success of their edits. The researchers first used ddPCR technology to quantify HBA copy number, which correlates with -thalassemia severity. They also used it to detect the successful insertion of HBB. In human umbilical cord blood-derived erythroid progenitor (HUDEP-2) cells, the team showed robust insertion of the HBB gene; the team confirmed on-target integration of the gene at 0.8 copies per cell.The team could not have detected this integration using qPCR. Because of the inherent variability in how qPCR results are measured, the technique cannot detect gene copies at concentrations lower than two or three copies per cell. Without ddPCR, these researchers would not have been able to show that their CRISPR strategy has potential for future clinical testing.

Future CRISPR applicationsApproximately30clinicaltrialsare in planning or underwayto study whetherCRISPRcan be used to treat genetic diseases, and regulatory agencies might approve the first CRISPR-based gene therapy in less than a decade. But given the continued challenge of developing a reliable CRISPR editing protocol, biopharmaceutical companies developing CRISPR therapies must take extra care to ensure their therapies are safe and effective. ddPCR technology can provide the confidence they need.For example, ddPCR assays can be designed to detect any CRISPR edits by using probes that span the junction between the native genome and the donor sequence. Researchers and biomanufacturers can screen out cell lines containing unsuccessful edits before they even reach patients by analyzing cell lines for specific CRISPR edits. This, in turn, will increase the chance of clinical success for CRISPR-based gene therapies and open the door to a new generation of treatments for difficult-to-treat genetic diseases like -thalassemia.

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CRISPR Gene Therapies: Assessing the Success of Gene Edits Using ddPCR - Technology Networks

Simple tasks like riding a bike down the street or driving to the grocery store are a no-go for Carlene Knight. Afflicted with a rare genetic disease called Leber congenital amaurosis type 10 (LCA10), Knight, 55, has been legally blind since birth. She has no peripheral vision. Its an extreme tunnel vision, she told The Daily Beast. I kind of liken it to looking through a window with a tiny hole in it and trying to find something like a building outside. Simple tasks like walking through a crowded room were arduous trials to avoid bumping into something and potentially injuring herself. At her office where she works at a call center, if she tried to walk around without her cane, she was constantly running into cubicles and tables and other objects all the time.

But the Happy Valley, Oregon resident has found her world opening up, ever since doctors literally fixed the DNA in the cells of her eyes.

By now youve probably heard of CRISPR, the gene-editing tool thats taken the biomedicine industry by storm. CRISPR basically allows scientists to find a specific sequence of DNA inside a cell, and alter it. That opens up the possibility of treating and potentially curing a slew of illnesses and disorders caused by genetic mutations, like LCA10, which impairs the function of retinal cells. These kinds of cells cant simply be removed, fixed, and plugged back into the eye. If theyre going to be fixed, it has to be in the body itself.

What happened to Knight is a huge step forward for physicians and researchers looking into CRISPR-related treatments. Up until now, the biggest breakthroughs in the space have revolved around taking unhealthy cells in patients, using CRISPR in the lab to modify them, and then putting them back into the patient. In this instance, CRISPR was used to directly edit the cellular DNA still inside of Knight and the others who participated in the trial.

With any kind of new therapy being used on the human body for the first time, you always have to be cautious, Mark Pennesi, an ophthalmologist at the Casey Eye Institute at the Oregon Health & Science University who led the experiment, told The Daily Beast. There's the things you might know, and then there are the things you don't know. You have to always take a cautious approach.

The trial was conducted jointly by the university and biotech company Editas Medicine, which specializes in gene editing. Preliminary trials on mice and non-human primates were safe and encouraging, so a clinical trial on humans with LCA10 was organized. The initial findings published Wednesday report the results for five participants (the other two having been treated only very recently). Two were given low doses of the new therapy, and three were given mid-range doses.

When I was told about the trial, I was really excited because I wanted to help children whose lives could be enhanced with vision, said Knight. The hope is that if they have the procedure early enough, theyll have a lot more vision later on life, while their neural pathways are developing.

Knight, who received a mid-range dose, and another participant who received the low dose both found their vision significantly improving. Neither has normal vision, but Knight said shes been amazed how much easier it is to do mundane things like find doorways, locate objects on the ground, and simply move around without having to surmount a myriad of hurdles. Colors are brighter and easier to seeto celebrate, shes even dyed her hair green, her favorite color.

It is amazing how the simple things can be so nice when you get them back, she said.

Two out of five success stories is not the ideal outcome Pennesi was hoping for, and his team doesnt have a clear explanation as to why not everyone who was treated saw improved vision. It could be the amount of dose, or factors specific to someones biology. And it might also be that patients need more time before the treatment works. Even if the editing works, the brain kind of has to rewire itself to even recognize the improved cellular function, he said. That could take many more months for some people.

The fact that none of the participants experienced any severe side effects is also a major milestone. Editas has started recruiting participants for higher dose trials, including children with LCA10. And the findings will likely be used as an encouraging sign for groups working on using CRISPR to treat other diseases where cells must be modified directly in the body, like Alzheimers, Huntingtons and Parkinsons.

Knights vision continues to improve bit by bit since the procedure. Its going to be nice if I can see my granddaughter play and ride her bike and stuff like that, she said. I hope I could one day read a childrens book to her, with the large print. That would really be nice.

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Gene Editing Gave This Blind Woman Some of Her Vision Back - The Daily Beast

The gene editing tool CRISPR is now being used to generate a range of traits in a variety of farmed aquatic species including salmonids, crustaceans and carp but there is still a way to go before it is likely to be financially and regulatorily viable in commercial aquaculture.

CRISPR is a modern, high-tech method of editing genes, but its based on a simple natural defence system found in many bacteria. Like higher organisms, bacteria also have to cope with a number of viruses and plasmids that can invade and take over their cells. The bacteria maintain a sort of genetic library within their DNA, containing key bits of gene sequences from past encounters with pathogens they might normally have to contend with. CRISPR refers to Clustered Regularly Interspaced Short Palindromic Repeats, which is basically what the bacterial libraries use to catalogue various viral and plasmid threats. These sequences, in conjunction with special enzymes, can serve as templates for precise molecular scissors that hunt down and cut DNA or RNA to destroy intracellular invaders before they can replicate. The trick to avoid any self-inflicted damage to the genetic sequence on file in the bacteriums library is that, in addition to the specific viral genetic sequence, the scissors also look for what is called protospacer adjacent motif (PAM), which is found in the invaders genetic material but not in the bacteriums.

Once scientists learned how to programme the molecular scissors of CRISPR using their own custom-designed genetic sequences they could target specific points on DNA strands to turn off, or knock out certain genes in almost any organism.

Once scientists learned how to programme the molecular scissors of CRISPR using their own custom-designed genetic sequences rather than those from a bacteriums library, they could target specific points on DNA strands to turn off, or knock out certain genes in almost any organism. This type of whack-a-mole research still continues in many aquatic species, especially with regard to health and immunity questions. But, after a brief period of somewhat exploratory research based on a knock stuff out and see what happens approach, a number of maybe we can turn stuff on applications began to emerge.

If modifications from gene editing are to be heritable, however, they usually must take place very early in the development of an animal or plant (ideally at the one-cell stage) so that they can be incorporated in its gametes once it matures. With this caveat comes the need for specialised methods like microinjection to deliver the CRISPR constructs and enzymes on a microscopic scale. An annoying side-effect of this approach is that the CRISPR effect can continue within the developing embryo well beyond the one-cell stage, often resulting in mosaicism (when various cells display different genetic makeups).

To date, most CRISPR research in farmed aquatic organisms reflects the same emphases as more traditional genetic improvement initiatives, namely growth, disease resistance and sterility, although some interesting work has also been done with regard to colouration patterns in various fishes.

A review of the science and the step-by-step details of how CRISPR has advanced are beyond the scope of this article (and the expertise of this author, for that matter), but suffice it to say that the potential applications for the technology now seem limited only by the imaginations of the scientists that have mastered it. Some examples include altering mosquitos so they cannot find human targets, breeding coffee beans with all the flavour and none of the caffeine, developing hangover-free wine with health benefits, creating tomatoes with the same spice producing genes as chili peppers (the genes are already present, just not expressed), molecular sleuthing for pathogens in natural or man-made environments and even bringing back the extinct wooly mammoth (well, actually creating a sort of mammoth/elephant hybrid, depending how one looks at it).

To date, most CRISPR research in farmed aquatic organisms reflects the same emphases as more traditional genetic improvement initiatives, namely growth, disease resistance and sterility, although some interesting work has also been done with regard to colouration patterns in various fishes. Problems with applying the technology to complex traits such as growth and disease resistance remain, since a number of genes are involved and many of them might require editing to attain desired outcomes. Fortunately advances in genomic selection continue in many aquatic species and this may provide shortcuts for the application of CRISPR, but significant work will be required to determine which versions of which genes should be targeted through editing. Editing to insert artificial alleles or genetic sequences from other species is now possible, but it will most likely face the same resistance observed toward the commercialisation of transgenic aquatic organisms.

Her keynote address on the subject at Aquaculture Europe in Dubrovnik in 2017 attracted over 500 delegates. Rob Fletcher

In salmonids, much of the work to date has focused on Atlantic salmon. While CRISPR can be used to produce sterile salmon by targeting the dead end gene, more effort will be required to apply this approach commercially because by default it cannot be transmitted to subsequent generations. Nonetheless, prior work with laboratory species (zebrafish and medaka) suggests that a method to restore fertility for breeding stocks might be possible. In other research, the dead end gene has been used to produce sterile fish which can then be implanted with germ cells from closely related donor fish (either endangered or more commercially valuable species). One example in Japan involves using edited grass puffer fish (Takifugu alboplumbeus) to serve as surrogate broodstock for the more valuable tiger puffer (T. rubripes).

Enhancing resistance to various pathogens may seem like fertile ground for the application of CRISPR in aquaculture, but disease resistance is a complex phenomenon involving the interaction of host species with both pathogens and the production environment, and many genes are usually involved. One research focus that shows commercial promise involves identifying genes that confer enhanced sea lice resistance in Pacific salmon species and then establishing similar attributes in Atlantic salmon through editing.

Japanese researchers reported in 2018 on the use of CRISPR techniques for the development of a line of red sea bream with enhanced muscle mass and a relatively short overall body length. Mutations were created by deletions in the Pm-mstn myostatin gene, and no exogenous genetic constructs were used. Compared to non-edited bream, the line exhibited a 16 percent increase in edible muscle tissue, and within two years a stable breeding population was established. As is the case in many other efforts with aquaculture species, this work built on prior research in laboratory populations of medaka and zebrafish. Two years later, Chinese investigators reported on a strain of yellow catfish (Pelteobagrus fulvidraco) that was also produced through editing of the myostatin gene. At 210 days post-fertilisation, fish from the edited line were 37 percent heavier than regular P. fulvidraco, with increased muscle mass.

Dr Masato Kinoshita, Kyoto-University and Dr Keitaro Kato, Kindai University

Editing myostatin genes has also been reported to significantly increase muscle mass in common carp, olive flounder, blunt snout bream, Sea bream, mud loach and channel catfish over the past five years, and scientists are now beginning to look at other genes in efforts to enhance growth rates in aquatic species. In the past several months, another group of researchers from China reported on the use of CRISPR to knock out the PI3K gene in Gibel carp. Disruption of this gene improves insulin sensitivity in mammals, but edited carp exhibited no alteration of plasma and hepatic glucose levels or uptake. They did, however, have improved somatic growth and feed conversion efficiency. Another recent study in China established that deletion of the t1r1 gene significantly improved acceptance of plant proteins in zebrafish.

Colouration and colour patterns are also important considerations in some aquaculture species. In China, researchers used CRISPR to disrupt carotenoid transport genes to alter red and white colour patterns in ornamental common carp. Another group used the technology to alter two agouti signalling protein (ASIP) genes to eliminate black patches in Oujiang common carp. Also in China, in a study to be published shortly, researchers used CRISPR to knock out the tyrosinase gene in a line of red tilapia. Once a true breeding population was established, continuous production of uniformly red fish with no black pigment was possible. And Israeli scientists recently published results (in The CRIPSR Journal) detailing the use of CRISPR to produce true albino Nile tilapia (pink eyes and all). They disrupted the slc45a2 gene, which mediates melanin biosynthesis, to produce zygotes with up to 99 percent albinism, including lack of melanin in the eyes.

Dr Jakob Biran

Use of CRISPR technology has also been demonstrated in crustaceans. Researchers from China reported in 2016 on the first genome editing of a decapod, the ridgetail white prawn Exopalaemon carinicauda. The primary objective of the work was to clarify the function of the chitinase compound EcChi4, by knocking out the gene responsible for its production. Since this species carries the fertilised eggs prior to hatching, one-cell stage embryos were collected from newly spawned females, stored in sterilised seawater at 4 C to halt further development and then microinjected. After 15 days of artificial incubation at room temperature, the shrimp hatched and were raised through the juvenile phase. Genomic DNA was extracted from both mysis larvae and juveniles to determine whether CRISPR methods resulted in mutation of the target gene. Results indicated different mutations were induced in the target gene, and that mosaic shrimp had been produced. The rate of mutation was calculated at 7.3 percent, and when individuals that were heterozygous for the induced mutation were crossed with normal shrimp, Mendelian inheritance (with a simple pattern where genes segregate into gametes at equal frequencies) was observed. When heterozygous offspring of the mutant shrimp were crossed, roughly one quarter of their offspring were homozygous for the mutation. In the case of EcChi4 in E. carinicauda, the induced mutation was heritable and did not influence survival or growth. Other crustaceans that have successfully been modified using CRISPR include Daphnia magna and the amphipod Parhyale hawaiensis.

To approach a CRISPR-based strategy, some knowledge of an organisms genome is required. While few and far between, some bivalve genomes are tentatively available for this type of research, including those of the Pacific oyster, eastern oyster, pearl oyster, Sydney rock oyster, Mediterranean mussel, Philippine horse mussel, king scallop and Yesso scallop. Nonetheless, work on bivalve gene editing has been quite difficult to date. Researchers have reported on the use of CRISPR targeting the myostatin gene in the Pacific oyster, with some success using microinjection methods.

For the time being, applying CRISPR to aquatic species will continue to be a pursuit of academic and research institutions. Its one thing for a university to develop the molecular technology and expertise required, but for private concerns to establish such high-tech capabilities a tremendous investment in equipment and staff will be required. Nevertheless, CRISPR is cheaper and more precise than other gene editing alternatives and usually provides better results.

Future applications and implications for CRISPR-related research in aquaculture species are currently debated, sometimes passionately, in many scientific and social contexts. One thing we can be certain of is the steady progress in our understanding of the genomes and complex physiological interactions of many important aquatic species, which in turn will allow more precisely targeted gene editing to improve production characteristics. And all with the potential to actually minimise genetic impacts on wild fisheries.

His career has included experience with numerous aquatic species in a number of countries. Dr Lutz is also the author of the book Practical Genetics for Aquaculture.

Original post:
The use of CRISPR in aquaculture - The Fish Site

The first CRISPR gene-edited food has gone on sale in Japan recently, in the form of a tomato packed with an alleged increase innutritionalcontent. TheSicilian Rouge High GABA tomato, created bystartup SanatechSeed,sold gene-edited seedlings to any farmers that wanted them earlier in the year, and 4,200 farmers tookup the offer. Now, the tomatoes are ripe for sale.

As far asSanatechSeed and media outlets cantell, this marks the first-ever CRISPR-edited food on sale to the public.

According to the company, the original plan was to sell the puree to begin with, but due to many requests they have begun selling tomatoes ahead of schedule.However, the tomatoes are just the beginning of the edited array of fruit and vegetables, with many more variants to come in the future.

As a seedling development company that utilizes genome editing technology, we are pleased with consumers and producers. We will continue to develop varieties that can be enjoyed, said SanatechSeed in their announcement.

The tomatoes in question are modified to have reduced levels of an enzyme that breaks down GABA, an inhibitory neurotransmitter that blocks signals between nerve connections. As a result, the tomatoes have around five times as much GABA in them, whichsome research suggests has a calming effect on the body and may improve stress and sleep. This research is debated, with many such studies having a conflict of interest, butso far evidence suggests supplemental GABA provides a limited effect on improvements in this area.

While gene editing may sound scary and is often used as a buzzword for those against genetically modified organisms, most produce we consume today has gone through gene alteration in some way.Modern bananas, for example, are a result of centuries of hybridization with other varieties, with wild bananas being filled with large seeds. Throughout this process, the farmers are altering characteristicsasthey wish via selective breeding CRISPR simply gives scientists far more control over which genes are introduced, silenced, or activated.

Japan does not consider these tomatoes as genetically modified, due to the fact that similar changes can occur naturally, and so they are available for purchase now.

TheSicilian Rouge High GABA tomato is almost definitely not the last consumers will see of CRISPR-modified food.The UK is currently undergoing alaw rework in the wake of their exit from the European Union, in which they are expected torelax gene editing lawsfor food. Should this go forward, plant biologists based in the UK have announced plans for a genetically-edited wheat plant, which should produce lower amounts of a possible carcinogen when toasted or fried.

[H/T:New Scientist]

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Tomato In Japan Is First CRISPR-Edited Food In The World To Go On Sale - IFLScience

ZUG, Switzerland and CAMBRIDGE, Mass., Oct. 01, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced two poster presentations at the Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting, to be held both virtually and at the Walter E. Washington Convention Center in Washington, D.C., from November 10 to 14, 2021. The Company also announced an oral presentation at the SITC 2021 Pre-Conference Program, The Evolution of Immunotherapy: An Exploration of Immunity Beyond T cells, CAR T in Solid Tumors and Novel Combinations, which will be held from 2:00 p.m. 6:00 p.m. ET on November 10, 2021.

CRISPR Therapeutics presentation:

Title: CRISPR/Cas9 gene-edited allogeneic CAR-T cells targeting CD33 show high preclinical efficacy against AML without long-term hematopoietic toxicityAbstract Number and Type: 133, posterDate and Time: Friday, November 12, 2021, 7:00 a.m. 8:30 p.m. ETLocation: Walter E. Washington Convention Center, Hall E, or https://www.sitcancer.org/2021/home

Presented jointly with Nkarta:

Title: A combined strategy of CD70 CAR co-expression with membrane-bound IL-15 and CISH knockout results in enhanced NK cytotoxicity and persistence Abstract Number and Type: 16439, oralDate and Time: Wednesday, November 10, 2021, 2:40 p.m. ETLocation: Walter E. Washington Convention Center, or https://www.sitcancer.org/2021/program/pre-conference-programs/industryprogram

Title: CISH gene-knockout anti-CD70-CAR NK cells demonstrate potent anti-tumor activity against solid tumor cell lines and provide partial resistance to tumor microenvironment inhibition Abstract Number and Type: 113, posterDate and Time: Friday, November 12, 2021, 7:00 a.m. 8:30 p.m. ETLocation: Walter E. Washington Convention Center, Hall E, or https://www.sitcancer.org/2021/home

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

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CRISPR THERAPEUTICS word mark and design logo are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

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

Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

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CRISPR Therapeutics to Present Preclinical Data at the Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting - Yahoo Finance

The idea of gene editing was once a thing of the future but today, it soon could be saving the lives of people all over the world. CRISPR is a gene editing system that bacteria have been using for possibly a few billion years. When a virus attacks a bacterium, this system takes a mug shot and keeps a snippet of the viruss DNA in order to remember it. If the same virus ever attacks again, the bacterium can fight off the virus before it causes harm.

Accompanied with 21st century tech, thats no longer all CRISPR can do. Scientists have since learned how to repurpose this system to cut our own DNA, wherever we tell it to, in order to edit our genes.

Is gene editing something we need to worry about? Some scientists think so. If we move too fast and over-indulge in this technology, its possible that we could be making permanent changes to the human species. But as of today, CRISPR is being used for good editing genes in people living with chronic diseases, helping patients around the world live healthy, normal lives.

WALTER ISAACSON: Early on, I thought, I'm gonna write a book about the great adventure of understanding gene editing. You know, I've written about the physics revolution that dominated the first half of the 20th century. And then of course I was deeply immersed in the digital revolution, which was the second half of the 20th century. But what happened in the past few years is we've found easy to reprogram tools that will allow us to edit our genes. Man, that's going to be 10 times more impactful than the digital revolution was.

So whenever you have a wonderful tale of adventure, it's always good to have one central character that helps bring the narrative along. And for me, Jennifer Doudna was perfect for that. When she was a young scientist and graduate student in the 1990s, all the men in science and biology, they were all running after the soccer ball, focusing on DNA and the human genome project. But she became fascinated with RNA. And it turns out that's a molecule that actually does more work. She was able to discover how RNA could replicate itself, which gets to one of the big questions in life. Which is, how did life begin on the planet? Then she discovered how to take this tool that bacteria use to fight viruses, called CRISPR, and repurpose it by reprogramming the RNA to edit our own human genes.

So all of these things come out of Jennifer's work in understanding the structure of RNA. CRISPR is a system that bacteria have been using for a billion years. And they learned a simple trick. If a virus attacks them, they take a mugshot, and they wrap it into their own bacterial code. If the virus ever attacks them again, they got that mugshot, and they take a guide, and take a pair of scissors known as an enzyme, and they chop up the virus. But what Jennifer Doudna

and Emmanuelle Charpentier and others did, was figure out, we can repurpose this so that the guide doesn't just chop up the viruses attacking bacteria, we'll reprogram it so that it cuts our own DNA wherever we tell it to. And thus, it becomes a tool to edit our genes.

Right after Jennifer invented this technology, she had a nightmare. And it's somebody who wanted to learn how to use the technology. She walks into the room, and in the nightmare, it's Hitler. So she starts gathering scientists to answer your question, which is, what are the perils

we need to worry about? Now, the perils to me, are that we go too fast down the road and make inheritable edits in the human genome in a way that affects our whole species. And I think that's a ethical line we have to pause and be very careful before we cross. We know ways to use this

in individual patients for deeply important medical needs, like sickle cell, cystic fibrosis, Huntington's, Tay-Sachs, muscular dystrophy. I think we should focus on those, and be careful about doing things that would allow rich people to buy better genes for their children. Because if people could go to a genetic supermarket, and say, what color eyes, what color hair, what height, I think we would harm the human species.

You know, we think of these as futuristic technologies, but we've already had CRISPR

be used for a real person, Victoria Gray. They use CRISPR technology to take her stem cells, edit them, reinsert them into her body, so that she is now makinG healthy blood cells. We're already using this to help the human species. So all these things are about the unbelievable excitement of the journey of science. And that open inquiry, that ability to approach things with an open mind, we sometimes lose that. We go into our ideological corners and we have knee jerk reactions to things without saying, "Show me the evidence." So one of the things I wish

people would think about, is it's not just about science, it's about the scientific method. Which means you're open to changing your mind.

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CRISPR: The future or undoing of humanity? - Big Think

Dedicated pre-seed and seed firm NFX has closed on fund 3 at $450 million, its largest seed fund to date.

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We spoke with the team to understand its evolution as a fund over the last four years.

We are singularly focused on being the best and first institutional investor, said Pete Flint, one of the firms founding partners. Given how early NFX invests were very comfortable with a great team and a rough concept, he said.

The firm, based in San Francisco and Israel, raised its first seed fund in 2017 at $150 million. Its second fund, raised in 2019, was $275 million, close to double the first fund. And fund 3 is even larger at $450 million, tripling its first fund size.

We count over 190 portfolio companies it has invested in to date, per Crunchbase data.

The three founding partners James Currier, Pete Flint and Gigi Levy-Weiss added a fourth general partner, Morgan Beller, over a year ago. Beller, who worked on Diem (previously Libra) at Facebook, will lead investments in crypto, amongst other sectors. Omri Amirav-Drory, promoted to general partner as part of that announcement, signals the firms increased interest in tech-bio.

NFX plans to invest in 70-plus additional companies with this new fund, with around 50 percent of it reserved for pro rata follow-on funding.

What gets me really excited about fund 3, and the fact that were bringing on Omri to do bio, [is that] were [also] doing proptech and crypto and fintech and gaming, and they actually all overlap, said Beller on the firms wide enough aperture, to not miss the best founders who might be playing around in weird places.

On what it takes to be successful at seed, Beller said You need to get into the best deals, and then you need to do a good job so that the best founders tell their friends to go back to you.

To support companies at seed, NFX has 45 staff members, with a platform team that it has built to help with recruiting, marketing, growth strategies, financial and legal support as well as a community platform to connect founders to share best practices.

We spoke with Amirav-Drory about the firms wide-ranging tech-bio practice, including therapeutics, diagnostics, food-ag, chemicals, materials and energy. The firm likes to invest in platform technologies and the intersection of tech and bio, said Amirav-Drory.

The firm is a seed investor in Mammoth Biosciences, recently valued at over $1 billion in a Series D funding. Mammoth has a diagnostic DNA sequencing platform as well as unique IP for small CRISPR proteins, he said.

NFX is also an investor in drug delivery companies, namely Nano Carry, which can deliver antibodies to the brain. Antibodies are an important modality for curing diseases, but 98 percent of antibodies dont reach the brain, said Amirav-Drory.

Another portfolio company, EdiTy Therapeutics is modifying T-cells as a CRISPR delivery platform. Delivery is one of the most important problems to solve for CRISPR, he said.

C2i Genomics, a fund 2 investment that recently raised a $100 million Series B, detects cancer through a liquid biopsy. Every cancer patient has its own unique biomarker that you can track, said Amirav-Drory. So instead of waiting months to see if a different intervention actually works, you can see every two weeks.

In 2021, multistage venture firms are raising large dedicated seed funds. Andreessen Horowitz announced a $400 million fund in August 2021, and Greylock announced a $500 million dedicated seed fund a month later. Sequoia Capital closed on its fourth dedicated seed fund of $195 million in early 2021 targeting U.S. and European startups.

With larger seed funds announced this year, we expect seed to become more competitive.

Photo courtesy of NFX: General Partners at NFX from left, Omri Amirav-Drory, Morgan Beller, James Currier, Gigi Levy-Weiss and Pete Flint.

Stay up to date with recent funding rounds, acquisitions, and more with the Crunchbase Daily.

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NFX Closes On Pre-seed And Seed Fund 3 At $450M - Crunchbase News

A Japanese startup is now selling the first food edited using CRISPR a gene-edited tomato that may be able to lower your blood pressure.

GABA boost: Gamma aminobutyric acid (GABA) is a compound produced naturally in our brains. Because its been linked to reduced feelings of anxiety, some people take supplements or eat foods with high levels of GABA to relieve stress, sleep better, and lower blood pressure.

Some plants are even cultivated to have higher-than-normal GABA levels, but the farming techniques used to do that can be labor intensive and affect crop yields.

The gene-edited tomato contains four to five times as much GABA as its unedited counterpart.

Gene-edited tomato: Now, Japanese researchers have used CRISPR to create a tomato that produces less of an enzyme that breaks down the plants natural GABA. The resulting gene-edited tomato contains four to five times as much GABA as its unedited counterpart.

Because the researchers didnt add anything to the tomatoes genome, Japanese authorities decided in December that the fruits neednt be regulated as genetically modified crops, saving the team from a long and expensive approval process.

In early 2021, the researchers started giving away Sicilian Rouge High GABA seedlings, and on September 17, they announced plans to begin selling the tomatoes themselves through the Sanatech Seed startup.

A 6.6 pound box of the gene-edited tomatoes costs about $68.

The tomatoes arent considered genetically modified because nothing was added to their genomes.

The bigger picture: The tomatoes appear to be the first CRISPR-edited food to be sold commercially anywhere in the world (a CRISPR-edited fish is being sold on a trial basis through a Japanese crowdfunding campaign).

However, the gene-edited tomato isnt the first edited food to hit consumers plates a cooking oil made from soybeans, edited using a different tech, went on sale in the U.S. in 2019.

CRISPR is lauded for its precision and ease of use compared to other gene-editing technologies, though, and now that one food developed using the technology is out in the world, we could see many more follow suit.

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This gene-edited tomato may help lower your blood pressure - Freethink

The pressure to produce results quickly during the drug discovery and development process continues to increase as does the role of genetically engineered custom mouse models. However, even the fastest custom mouse model generation projects take about 6 months to reach the stage when a few F1 heterozygous mice are available for experiments or breeding. This timeline increases if more than a few heterozygotes or homozygotes are needed. Taconics ExpressMODEL portfolio of products shifts the deliverable of a model generation project from a few heterozygous F1 mice to a much larger cohort of 10-100 mice while reducing the timeline to obtaining data, adding predictability, all without compromising essential quality control steps. By applying innovative thinking and leveraging our ability to seamlessly integrate custom model generation, embryology and colony management services, the ExpressMODEL portfolio achieves the industry's fastest timelines to study cohort with no compromise in quality for models generated using embryonic stem cell (ESC), CRISPR, or random integration transgenic (RITg) methodology.

Regardless of the methodology used to generate the founder animals, ExpressMODEL is built around the concept that using in vitro fertilization (IVF) rather than conventional breeding to generate the F1 mice from founders has a number of distinct advantages including:

However, ExpressMODEL is more than simply using IVF to produce F1 mice because the quality of the male founders needs to be high in order to fully realize the advantages IVF provides. High quality means that founder males need to have both a high percentage of the desired genetically-modified gene and a high fertility rate. Thus, the candidate founder males need to be well-characterized. Because the different methodologies used for model generation produce founders with different characteristics, we have developed unique founder analysis protocols to fit the three different methodologies. The founders produced from injection of ESCs into blastocyst-stage embryos are called chimeras because they are derived from two different populations of genetically distinct cells that originated from different embryos. The founders produced by introducing CRISPR reagents or transgenic DNA into one-cell embryos (zygotes) are mosaics meaning they are composed of two or more different populations of genetically distinct cells that originated from the same embryo.

ExpressMODEL: Embryonic stem cell (ESC)

ESC-mediated mouse model generation remains the gold standard and best choice for complex projects such as genomic replacement humanizations. Using ExpressMODEL: ESC, the timeline for a typical project that would take 66 weeks to deliver a homozygous study-size cohort is reduced to 54 weeks, saving at least 3 months. The key components of ExpressMODEL: ESC are:

The data from these analyses facilitate the choice of founder male(s) to be utilized for the IVF to produce an F1 heterozygous cohort that is sized to meet the customers downstream goals and timeline requirements. It is important to note that all quality control steps in vector construction and ES cell targeting are preserved.

ExpressMODEL: CRISPR

Two great advantages of CRISPR methodology are the speed at which a genetically engineered model can be generated and the ability to modify a wide range of genetic backgrounds, including existing genetically-engineered models. Using ExpressMODEL: CRISPR, the timeline for a typical project that would take 48 weeks to deliver a homozygous study-size cohort is reduced to 36 weeks, saving at least 3 months. ExpressMODEL: CRISPR combines our ability to produce founders with a low degree of mosaicism and to accurately estimate the degree of mosaicism of each founder male. The key components of ExpressMODEL: CRISPR are:

ExpressMODEL: Random Integration Transgenic (RITg)

More than 30 years since the first RITg model was generated, the method continues to be a favored path to quickly generate gain of function models that express an ectopic gene. However, because genomic integration of the transgene is random in each injected embryo, the resulting founder line are unique and may or may not perform to the desired specifications. Additionally, each founder often has transgene insertions at multiple sites and the configuration and copy number of those insertions differs. Thus, RITg founders can be more genetically complex than CRISPR founders. As a result, common practice is to separately propagate multiple lines to generate offspring for extensive transgene expression studies. These data are then used to determine which founder line(s) to propagate. The cost and time of this downstream breeding and characterization of multiple founder lines greatly exceeds the original cost to generate the lines and takes significant additional time. Because transgene expression is assessed in founder animals, ExpressMODEL: RITg takes the guesswork out of the process and allows one to avoid the cost of breeding and characterizing multiple founder lines. Moreover, it reduces project timeline by at least 12 weeks and potentially up to 24 weeks or more. The key components of ExpressMODEL: RITg are:

Additional customizable options are available including the provision of tissue lysates and fixed tissue for protein expression analyses, and transgene mapping analysis to accurately determine the transgene integration site and configuration.

Taconics ExpressMODEL suite of technologies is designed to reduce the custom model generation timeline from project conception to study cohort without taking any shortcuts that compromise quality. Taconic provides a seamless end-to-end solution incorporating industry leading model generation, embryology, and colony management capabilities that allows your project to travel in the express lane.

Interested in learning more about custom animal model generation? Visit Taconic's website at http://www.taconic.com.

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The Role of Quality and Speed in Custom Model Generation - FierceBiotech

North America, July 2021, The CRISPR-PE TechnologyMarket research report includes an in-sight study of the keyGlobal CRISPR-PE Technology Marketprominent players along with the company profiles and planning adopted by them. This helps the buyer of the CRISPR-PE Technologyreport to gain a clear view of the competitive landscape and accordingly plan CRISPR-PE Technology market strategies. An isolated section with top key players is provided in the report, which provides a complete analysis of price, gross, revenue(Mn), CRISPR-PE Technology specifications, and company profiles. The CRISPR-PE Technology study is segmented by Module Type, Test Type, And Region.

The CRISPR-PE Technology market size section gives theCRISPR-PE Technologymarket revenue, covering both the historic growth of the market and the forecasting of the future. Moreover, the report covers a host of company profiles, who are making a mark in the industry or have the potential to do so. The profiling of the players includes their market size, key product launches, information regarding the strategies they employ, and others. The report identifies the total market sales generated by a particular firm over a period of time. Industry experts calculate share by taking into account the product sales over a period and then dividing it by the overall sales of theCRISPR-PE Technologyindustry over a defined period.

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The CRISPR-PE Technology research covers the current market size of theGlobal CRISPR-PE Technology Marketand its growth rates based on 5 year history data. It also covers various types of segmentation such as by geography North America, Europe, Asia-Pacific etc., by product type CRISPR-PE Technology, by applications CRISPR-PE Technologyin overall market. The in-depth information by segments of CRISPR-PE Technologymarket helps monitor performance & make critical decisions for growth and profitability. It provides information on trends and developments, focuses on markets and materials, capacities, technologies, CAPEX cycle and the changing structure of the Global CRISPR-PE Technology Market.

This CRISPR-PE Technology study also contains company profiling, product picture and specifications, sales, market share and contact information of various international, regional, and local vendors of CRISPR-PE Technology. The CRISPR-PE Technology market competition is constantly growing higher with the rise in technological innovation and M&A activities in the industry. Moreover, many local and regional vendors are offering specific CRISPR-PE Technology application products for varied end-users. The new vendor entrants in the CRISPR-PE Technology market are finding it hard to compete with the international vendors based on quality, reliability, and innovations in technology.

Global CRISPR-PE Technology(Thousands Units) and Revenue (Million USD) Market Split by variousapplication & types:-

Segment by Type Cell Line Engineering Genome Regulation

Segment by Application Biomedical Research Agricultural Research Others

The research study is segmented by Application such as Laboratory, Industrial Use, Public Services & Others with historical and projected market share and compounded annual growth rate.GlobalCRISPR-PE Technology(Thousands Units) by Regions (2021-2029)

Geographically,this CRISPR-PE Technology report is segmented into several key Regions, with production, consumption, revenue (million USD), and market share and growth rate ofCRISPR-PE Technologyin these regions, from 2012 to 2028(forecast), covering

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There are 15 Chapters to display theCRISPR-PE Technology.

Chapter 1, to describe Definition, Specifications and Classification ofCRISPR-PE Technology, Applications ofCRISPR-PE Technology, Market Segment by Regions;

Chapter 2, to analyze the CRISPR-PE Technology Manufacturing Cost Structure, CRISPR-PE Technology Raw Material and Suppliers, CRISPR-PE Technology Manufacturing Process, CRISPR-PE Technology Industry Chain Structure;

Chapter 3, to display the CRISPR-PE Technology Technical Data and Manufacturing Plants Analysis ofCRISPR-PE Technology industry, CRISPR-PE Technology Capacity and Commercial Production Date, CRISPR-PE Technology Manufacturing Plants Distribution, CRISPR-PE Technology R&D Status and Technology Source, CRISPR-PE Technology Raw Materials Sources Analysis;

Chapter 4, to show the Overall CRISPR-PE Technology Market Analysis, CRISPR-PE Technology Capacity Analysis (Company Segment), CRISPR-PE Technology Sales Analysis (Company Segment), CRISPR-PE Technology Sales Price Analysis by Beam Therapeutics, CRISPR Therapeutics, GenScript Biotech, Horizon Discovery, Integrated DNA Technologies (IDT, Intellia Therapeutics Inc, Inscripta, Precision Bioscience, Sangoma Therapeutics, Synthego Corporation;

Chapter 5 and 6, to show the CRISPR-PE Technology Regional Market Analysis that includes North America, Europe, Asia-Pacific etc.,CRISPR-PE TechnologySegment Market Analysis by Types;

Chapter 7 and 8, to analyze theCRISPR-PE TechnologySegment Market Analysis (by Application) Major Manufacturers Analysis ofCRISPR-PE Technology;Beam Therapeutics, CRISPR Therapeutics, GenScript Biotech, Horizon Discovery, Integrated DNA Technologies (IDT, Intellia Therapeutics Inc, Inscripta, Precision Bioscience, Sangoma Therapeutics, Synthego Corporation

Chapter 9, CRISPR-PE Technology Market Trend Analysis, CRISPR-PE Technology Regional Market Trend, CRISPR-PE Technology Market Trend by Product Types , CRISPR-PE Technology Market Trend by Applications;

Chapter 10, CRISPR-PE Technology Regional Marketing Type Analysis, CRISPR-PE Technology International Trade Type Analysis, CRISPR-PE Technology Supply Chain Analysis;

Chapter 11, to analyze the Consumers Analysis ofCRISPR-PE Technology;

Chapter 12, to describeCRISPR-PE TechnologyResearch Findings and Conclusion, CRISPR-PE Technology Appendix, CRISPR-PE Technology methodology and CRISPR-PE Technology various data source;

Chapter 13, 14 and 15, to describeCRISPR-PE Technologysales channel, CRISPR-PE Technology distributors, CRISPR-PE Technology traders, CRISPR-PE Technology dealers, CRISPR-PE Technology Research Findings and CRISPR-PE Technology Conclusion, appendix and data source.

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Find more research reports onCRISPR-PE Technology Industry.By JC Market Research.

Thanks for reading this article; you can also get individual CRISPR-PE Technology chapter wise section or region wise report version like North America, Europe or Asia.

About Author:JCMR global research and market intelligence consulting organization is uniquely positioned to not only identify growth opportunities but to also empower and inspire you to create visionary growth strategies for futures, enabled by our extraordinary depth and breadth of thought leadership, research, tools, events and experience that assist you for making goals into a reality. Our understanding of the interplay between industry convergence, Mega Trends, technologies and market trends provides our clients with new business models and expansion opportunities. We are focused on identifying the Accurate Forecast in every industry we cover so our clients can reap the benefits of being early market entrants and can accomplish their Goals & Objectives.

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CRISPR-PE Technology Market Impressive Gains including key players Beam Therapeutics, CRISPR Therapeutics, GenScript Biotech, Horizon Discovery Bulk...

The CRISPR-Based Therapeutics Market analysis outline by Reports Intellect is an exhaustive study of the latest trends leading to this vertical trend in various regions. The report also calculated the market size, revenue, price, revenue, gross profit margin and market share, cost structure, and growth rate. The report will help stakeholders understand the competitive landscape and gain insight to better position their businesses.

Get a Sample PDF copy of the report @ https://www.reportsintellect.com/sample-request/1071136

This report provides a comprehensive view of the competitive environment of the CRISPR-Based Therapeutics market and includes an extensive description of the performance of the major global players completed in the market. This market research report should be used to gain valuable insight into the market in a cost-effective manner. The CRISPR-Based Therapeutics global report is created taking into account all of the business requirements that are essential for successful business growth.

CRISPR-Based Therapeutics Market competition by top manufacturers as follow: Caribou Biosciences, Intellia Therapeutics, Addgene, Merck KGaA, Mirus Bio LLC, CRISPR THERAPEUTICS, Thermo Fisher Scientific, Editas Medicine, Horizon Discovery Group, Takara Bio USA, GE Healthcare Dharmacon.

Segmentation by type:

Genome EditingGenetic EngineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem CellsGenetically Modified Organisms/CropsCell Line Engineering

Segmentation by application:

Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

Market segmentation, by regions:North America (United States, Canada)Europe (Germany, France, UK, Italy, Russia, Spain, Netherlands, Switzerland, Belgium)Asia Pacific (China, Japan, Korea, India, Australia, Indonesia, Thailand, Philippines, Vietnam)Middle East & Africa (Turkey, Saudi Arabia, United Arab Emirates, South Africa, Israel, Egypt, Nigeria)Latin America (Brazil, Mexico, Argentina, Colombia, Chile, Peru)

Questions Answered within the CRISPR-Based Therapeutics Market Report:

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What youll expect From Our Report:

About Us:-Reports Intellect marketing research, an exploration and business firm providing syndicated likewise as custom reports with precise analysis and future outlook. We tend to at Reports Intellect marketing research believe client satisfaction and propose they take strategic decisions relating to the current and future endeavors. So, whether or not its the newest report from the researchers or a custom demand, our team is here to assist you within the very best method.

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CRISPR-Based Therapeutics Market Predicted to Grow in Future by Focusing on Top Players: Caribou Biosciences, Intellia Therapeutics, Addgene, Merck...

The Global CRISPR Gene Editing Tools Market business is anticipated to grow quickly from 2021 to 2027, according to a recent study by MarketandResearch.biz. The record anticipates a market share evaluation in terms of quantities for the projection period. The research focuses on past and current market trends, which serve as a foundation for predicting the markets future. The research is based on an in-depth examination of a number of factors, including challenges, market dynamics, competitive analyses, market size, issues, and the agencies involved.

The study tackles the essential aspects and difficulties of geographical areas while adhering to the framework of global CRISPR Gene Editing Tools market competency research. The market research examines provincial and national market sizes, division market growth deals, opportunities, international market players, current events, exchange guidelines, and important business development research.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketandresearch.biz/sample-request/175842

Product type segmentation:

Use application segmentation as a guide:

The CRISPR Gene Editing Tools analysis identifies the following major market players:

The following key nations are included in the market research:

ACCESS FULL REPORT: https://www.marketandresearch.biz/report/175842/global-crispr-gene-editing-tools-market-2021-by-company-regions-type-and-application-forecast-to-2026

This section includes information on the market size, volume, and value of each region for the forecast period to aid our clients in attaining a stronger position in the global market. The competitive landscape section includes in-depth case studies on how to overcome challenges in the CRISPR Gene Editing Tools market as well as top market competitors strategies.

Customization of the Report:

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

Contact UsMark StoneHead of Business DevelopmentPhone: +1-201-465-4211Email: sales@marketandresearch.biz

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Global CRISPR Gene Editing Tools Market 2021 Growth Opportunities and Competitive Landscape 2027 Abcam, Inc., Agilent Technologies, Inc., CRISPR...

SAN FRANCISCO--(BUSINESS WIRE)--Latch Bio, a company building data infrastructure for the biocomputing revolution, today announced the closing of a $5 million seed funding round led by Lux Capital with participation from General Catalyst, Haystack, Fifty Years, and Asimov co-founder and CEO, Alec Nielsen, Ph.D. The company has also announced the launch of its first-of-a-kind web-based platform which enables any biologist to analyze CRISPR data without any code or cloud infrastructure setup.

Like genomics before it, the CRISPR community is facing a tsunami of data and a dearth of computational tools required for their analyses. As a result, skilled bioinformaticians are in high demand, and researchers are waiting days for what can be completed in a couple hours, said Brandon Reeves, Partner, Lux Capital. The Latch team has made it possible for any researcher in any lab to open a web browser, upload data and execute a powerful computational pipeline without having to enter a single line of code or build any sort of cloud infrastructure. By empowering the researcher, Latch is helping remove a significant bottleneck that is currently slowing down the whole CRISPR research cycle.

Latch Bio was founded in 2021 by Alfredo Andere, Kyle Giffin, and Kenny Workman who met as undergraduates at the University of California, Berkeley. Bringing together their backgrounds in engineering and computer science, they formed Latch Bio to build infrastructure for scientists who need ready-to-run solutions to advance their research. The Latch platform is a web-first solution that addresses some of the primary challenges facing CRISPR researchers, namely access to bioinformatics experts and the implementation of cloud resources.

Since 2015, the ChristianaCare Gene Editing Institute has been at the forefront of innovation in advancing the use of gene editing to support improved human health, said Pawel Bialk, M.S., principal investigator at the Gene Editing Institute. Access to the right tools is essential to our success. The Latch platform, which includes ChristianaCares DECODR program, will provide immediate access to a wide range of popular CRISPR workflows that will further accelerate our discovery and translational research programs.

Using the Latch platform, any researcher can now create a centralized one-stop-shop for storing, transforming and visualizing their data without writing any code. Through the Latch plugins, users can import files from their existing data stack including Amazon S3, Benchling, and Illuminas BaseSpace. Biologists have access to dozens of popular workflows including CRISPResso, MAGeCK, CasTLE, Cas.py, MultiQC, and CasOffinder, among others. After performing a specific workflow, users can further interrogate the results using the built-in genomic visualizer and quality controls. The Latch platform is offered free to academic research users.

With the public launch of the Latch platform, we have officially begun our campaign to build and disseminate the data infrastructure for the biocomputing revolution, said Kenny Workman, co-founder and Chief Technology Officer.

CRISPR scientists who are committed to changing the world deserve the best software, and at Latch we actually listen to them, then build what they want. We want to be their champion and build solutions that make their job easier, said Kyle Giffin, co-founder and Chief Operating Officer.

We look forward to working closely with our users, who are each revolutionizing the capabilities of gene-editing and will be invaluable partners as we continue to improve the Latch platform, said Alfredo Andere, co-founder and Chief Executive Officer. We are at the very beginning of realizing our vision and look forward to working closely with our advisors and growing base of users to further expand the platform's capabilities.

To learn more about access to the Latch platform, please visit http://www.Latch.bio.

About Latch Bio

Founded in 2021, Latch Bio is on a mission to build and disseminate the data infrastructure for the biocomputing revolution. The companys cloud-based platform offers no-code bioinformatics for CRISPR researchers seeking to store, transform and visualize their data. Through any web browser, the global CRISPR community can quickly access popular bioinformatics pipelines and data visualization tools. The company's investors include Lux Capital, General Catalyst, Haystack, and Fifty Years. Latch Bio is based in San Francisco, California and can be found online at http://www.LatchBio.com.

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Latch Bio Emerges from Stealth with Seed Funding Round Led by Lux Capital - Business Wire

DUBLIN, Oct. 4, 2021 /PRNewswire/ -- The "Cell Based Assay & High Content Screening Markets" report has been added to ResearchAndMarkets.com's offering.

Research and Markets Logo

Cell-Based Assays are a mainstay of drug development and scientific research, but growth is now accelerating as the race for a COVID-19 cure gains speed.

On top of this, new technology is allowing Cell-Based Assays to be used to measure any aspect of cell function. This market just keeps on growing with no end in sight. The workhorse of the pharmaceutical industry is becoming a central player in biotechnology.

This is a complex area but this readable report will bring the entire management team up to speed, on both the technology and the opportunity.

The technology is moving faster than the market. Genomics and Immunology are playing a role too. Find the opportunities and pitfalls. Understand growth expectations and the ultimate potential market size.

Industry Overview

Players in a Dynamic Market

Academic Research Lab

Contract Research Organization

Genomic Instrumentation Supplier

Cell Line and Reagent Supplier

Pharmaceutical Company

Audit Body

Certification Body

Market Trends

Factors Driving Growth

Candidate Growth

Immuno-oncology

Genomic Blizzard

Technology Convergence

The Insurance Effect

Factors Limiting Growth

Technology Development

Cell Based Assays Recent Developments

Axxam and FUJIFILM Cellular Dynamics Announce Strategic Alliance

Cancer Genetics to Acquire Organoid Startup Stemonix

Curi Bio Acquires Artificial Intelligence Firm Dana Solutions

CRISPR Screens Uncover Novel Cancer Therapy Targets

ERS Genomics Licenses CRISPR-Cas9 Patents to Axxam

New Transcriptomics Assay Facilitates Compound Screens

Carta Biosciences Betting on Gene Interaction Mapping

High-throughput Identifies cancer drug candidates

Velabs Therapeutics partners with Alytas Therapeutics to develop a novel immune-based therapy for obesity

InSphero platform selected to test Cyclerion's sGC stimulator technology

OcellO to provide in vitro research services to Merus

Charles River Laboratories to acquire Citoxlab

Reaction Biology Corporation Purchases ProQinase GmbH

Cisbio extends its assay portfolio for immuno-oncology drug discovery

STEMCELL Technologies Launches Next-Generation Culture System

Abcam Acquires Calico Biolabs

Evotec announces achievement in Celgene alliance utilizing IPSC screening

Fujifilm Cellular Dynamics Inc. launches iCELL Microglia

Cisbio and Excellerate Bioscience partner

Horizon Discovery extends CRISPR Screening Service to primary human T cells

Profiles of Key Cell Based Assay Companies

Story continues

Abcam

Agilent

Aurora Instruments Ltd

Axxam

Beckman Coulter Diagnostics

Becton, Dickinson and Company

BioIVT

Bio-Rad Laboratories, Inc.

BioTek Instruments

BioVision, Inc.

BMG Labtech

Cell Biolabs, Inc

Cell Signaling Technology, Inc.

Charles River Laboratories

Cisbio Bioassays

Corning, Inc

Cytovale

Enzo Life Sciences, Inc

Eurofins DiscoverX Corporation

Evotec AG

Excellerate Bioscience

Fujifilm Cellular Dynamics International

Genedata

Hemogenix

Horizon Discovery

Invivogen

Leica Biosystems

Lonza Group Ltd.

Luminex Corp

Merck & Co., Inc

Miltenyi Biotec

Molecular Devices

Nanion

Ncardia

New England Biolabs, Inc

Olympus

Origene Technologies

Perkin Elmer

Promega

Qiagen Gmbh

Reaction Biology

Recursion Pharma

Roche Diagnostics

Sartorius

Sartorius-ForteBio

Sartorius-IntelliCyt

Sony Biotechnology

SPT Labtech

Stemcells Technologies Canada Inc

Tecan

Thermo Fisher Scientific Inc.

Vitro Biopharma

For more information about this report visit https://www.researchandmarkets.com/r/olr3vr

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Cell Based Assay & High Content Screening Market Report 2021: The Workhorse of the Pharmaceutical Industry is Becoming a Central Player in...