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Archive for the ‘Crispr’ Category

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

-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

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

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 use of CRISPR in aquaculture – The Fish Site

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.

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The use of CRISPR in aquaculture - The Fish Site

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

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

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

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

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

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

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

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

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

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.

Wed love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at tips@freethink.com.

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

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

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

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)

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There are 15 Chapters to display theCRISPR-PE Technology.

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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;

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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;

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

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

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.

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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)

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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...

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

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.

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

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

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

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

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...

CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global…

INTRODUCTION Clustered regularly interspaced short palindromic repeats (CRISPR) are a family of DNA sequences, which constitute a primitive immune system that is responsible for protecting prokaryotic cells from phage infections.

New York, Aug. 24, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global Forecasts, 2021-2030" - https://www.reportlinker.com/p06130494/?utm_source=GNW It was first described in 1987, however, its potential as a gene editing tool was not realized until 2012. Since then, the CRISPR revolution has not shown any signs of slowing down and has been responsible for significant advances in molecular biology and therapy development. Fundamentally, the CRISPR/Cas system involves specific palindromic DNA sequences which work in tandem with a family of caspase enzymes (Cas9, Cas12), in order to excise gene fragments with high precision. Compared to the other targeted nuclease-based systems, CRISPR is relatively faster, and cost-efficient; as a result, the demand for this gene editing tool is very high. The relatively recent discovery / development of novel accompanying nucleases, namely Cas12a, Cas13, Cas14 and dCas9, has significantly improved the precision of this technology. Presently, there are several companies using different variants of the CRISPR/Cas technology for basic research, and the development of gene editing solutions. However, the therapeutic use of this versatile genetic manipulation tool is only being investigated by a select few stakeholders in the pharmaceutical industry. The aforementioned scenario is attributed to the surrogate licensing model, which has granted exclusive control of the associated intellectual property (IP) to three leading players, namely Editas Medicine, CRISPR Therapeutics and Intellia Therapeutics, in the contemporary market.

Clinical trials of CRISPR based therapeutics are currently focused mainly on oncological and hematological disorders; however, several product candidates against certain neurological disorders and infectious diseases, specifically targeting recurrent conditions, are under investigation. Post 2014, the overall interest in this technology has grown exponentially, with several start-ups entering the market and 6 of the top 10 pharmaceutical companies restructuring their efforts in this direction. Over time, a substantial body of evidence has also been generated validating the therapeutic applications of this technology, which has, in turn, prompted the establishment of numerous strategic partnerships (focused on therapy development and clinical research) and has caused investors to put in significant capital into innovator companies involved in this domain, over the last two years alone. In fact, the three leading companies in this industry segment together have combined market capitalization of more than USD 10 billion, and have raised more than USD 2.8 billion in various funding rounds. Despite the possibly limitless potential of the CRISPR/Cas technology, further investigation, probing its safety and therapeutic efficacy in large diverse populations, is required. Key impediments to approval and other existing challenges that are being addressed by stakeholders, include off-target toxicity-related concerns and complexities related to the delivery of CRISPR components into target cells. Concerning delivery, innovators in this field have reported notable success using different types of platforms for facilitating the intracellular administration of CRISPR components; examples of successful delivery methods include electroporation, AAV vectors and lipid nanoparticles (LNPs). A few companies are also evaluating bacteriophages as a potential delivery system for such products. Promising clinical results, and ongoing technical developments, coupled to the growing interest of biopharmaceutical developers, are anticipated to push pipeline products to higher phases and on to commercialization. We believe that the market is likely to evolve at a commendable pace over the next decade.

SCOPE OF THE REPORT The CRISPR Based Therapeutics Market, 2021-2030 report features an extensive study of the current market landscape and future opportunity for the players involved in the development of CRISPR based therapeutics for the treatment of a variety of disease conditions. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographies. Amongst other elements, the report includes: A review of the CRISPR based therapeutics that are currently in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as target therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others), phase of development (discovery, preclinical and clinical), approach of therapy (ex vivo and in vivo), cell source (autologous and allogeneic), type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), and the type of technology used. It also includes information on the completed, ongoing and planned clinical trials for CRISPR based therapeutics, sponsored by various industry players. Elaborate profiles of key players in this domain. Each company profile features a brief overview of the company, its financial information (if available), a brief description of its therapeutic candidates, recent developments, and an informed future outlook. An in-depth analysis of around 2,000 patents related to CRISPR technology that have been filed / granted, since 2015, highlighting the key trends associated with these patents, across type of patent, publication year and application year, regional applicability, IPCR symbols, emerging focus areas, inventor information, leading patent assignees (in terms of number of patents filed / granted), patent benchmarking and valuation. An analysis of the partnerships that have been inked by various stakeholders engaged in the development of CRISPR based therapeutics, during the period 2014-2020, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. An analysis of the investments made at various stages of development of the companies engaged in this field, covering instances of seed financing, venture capital financing, grants / awards, capital raised from IPOs and subsequent offerings. An analysis of the start-ups (established in the time period between 2013-2020 and have less than 200 employees) engaged in the development of CRISPR based therapeutics, based on several parameters, such as number of candidates in discovery, preclinical and clinical phase of development, therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

One of the key objectives of the report was to estimate the future growth potential of CRISPR based therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2021-2030. For this purpose, we have segmented the future opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL), [D] key geographical regions (North America, Europe and Asia-Pacific). To account for uncertainties and to add robustness to our model, we have provided three market forecast scenarios, portraying the conservative, base and optimistic tracks of the anticipated industrys growth.

KEY QUESTIONS ANSWERED Who are the leading players engaged in the development of CRISPR based therapeutics? Which key clinical conditions can be treated by CRISPR based drugs? What are the investment trends in this industry? Which partnership models are commonly adopted by stakeholders engaged in this domain? How has the intellectual property landscape in this market evolved over the years? Which factors are likely to influence the evolution of this market? How is the current and future market opportunity likely to be distributed across key market segments?

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been validated from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views

While the focus has been on forecasting the market till 2030, the report also provides our independent views on various non-commercial trends emerging in this industry. This opinion is solely based on our knowledge, research and understanding of the relevant market trends gathered from various secondary and primary sources of information.

CHAPTER OUTLINES Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the CRISPR therapeutics market and its likely evolution in the short-mid to long term.

Chapter 3 provides a general introduction to CRISPR/Cas system. In addition, we have briefly described the components of CRISPR/Cas system, its mechanism of action and vehicles to deliver CRISPR/Cas components in to the target cells. The chapter lays emphasis on the applications of CRISPR technology. It also includes a discussion on the challenges associated with the use of CRISPR based therapeutics.

Chapter 4 presents a detailed assessment of the current market landscape of CRISPR based therapeutics, along with information on type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), approach of therapy (in vivo and ex vivo), cell source (autologous and allogeneic), phase of development (discovery, preclinical and clinical), type of delivery vehicle used (viral vector, electroporation, LNPs, bacteriophage and others), target disease indication and therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others). In addition, it provides an overview of the CRISPR based therapeutics developer landscape, highlighting the players that are active in this domain. It includes information on their year of establishment, company size (in terms of number of employees) and location of headquarters of the drug developers. We have presented a logo landscape, highlighting the distribution of the drug developers based on company size and location of headquarters. Further, it presents an analysis on the initiatives of the big pharma companies in this domain.

Chapter 5 includes profiles of the key players engaged in the development of CRISPR based therapeutics (shortlisted based on strength of product portfolio). Each profile features a brief overview of the company, its financial information (if available), brief details of gene editing technology, therapeutic pipeline, recent developments and an informed future outlook.

Chapter 6 provides an in-depth analysis of the patents filed / granted for CRISPR technology since 2015. The analysis also highlights the key trends associated with these patents, including type of patent (granted patent, patent application and others), publication year, application year, geographical location / patent jurisdiction (North America, Europe, Asia-Pacific and Rest of the World), IPCR symbols, key inventors and leading industry / non-industry players. In addition, it includes a detailed patent benchmarking analysis of leading players and patent valuation analysis, which evaluates the qualitative and quantitative aspects of these patents.

Chapter 7 features a detailed analysis of the partnerships and collaborations that have been inked in this domain since 2014, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. The chapter includes analysis based on year of partnership, type of partnership model, purpose of licensing deal, and most active player(s) (in terms of number of partnerships inked). In addition, the chapter features a discussion on the surrogate licensing practice in the CRISPR based therapeutics market.

Chapter 8 provides an analysis of the investments made since 2014 at various stages of development of companies engaged in this domain, based on the year of investment, number of funding instances, amount invested and type of funding, highlighting most active players (in terms of number of funding instances and amount raised) and most active investors (in terms of number of funding instances).

Chapter 9 presents an analysis of the start-ups (established after 2012 and having less than 200 employees) engaged in the development of CRISPR based therapeutics based on the parameters, such as number of candidates in discovery, preclinical and clinical phase of development, target therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

Chapter 10 features an elaborate discussion on the future market potential of various CRISPR based therapeutics. The chapter provides insights on the likely distribution of the current and forecasted opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL) and [D] key geographical regions (North America, Europe and Asia-Pacific).

Chapter 11 is a summary of the overall report, highlighting the key facts and figures related to the research and analysis presented in the previous chapters.

Chapter 12 is a collection of interview transcripts of discussions held with representatives of renowned organizations engaged in the CRISPR technology domain. In this chapter, we have presented the insights on our conversation with Harrison Wong (Public Relations, Burns McClellan, for eGenesis).

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix, which consists the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06130494/?utm_source=GNW

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CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global...

Applications of CRISPR as a potential therapeutic – DocWire News

This article was originally published here

Life Sci. 2021 Aug 25:119908. doi: 10.1016/j.lfs.2021.119908. Online ahead of print.

ABSTRACT

Genetic disorders and congenital abnormalities are present in 2-5% of births all over the world and can cause up to 50% of all early childhood deaths. The establishment of sophisticated and controlled techniques for customizing DNA manipulation is significant for the therapeutic role in such disorders and further research on them. One such technique is CRISPR that is significant towards optimizing genome editing and therapies, metabolic fluxes as well as artificial genetic systems. CRISPR-Cas9 is a molecular appliance that is applied in the areas of genetic and protein engineering. The CRISPR-CAS system is an integral element of prokaryotic adaptive immunity that allows prokaryotic cells to identify and kill any foreign DNA. The Gene editing property of CRISPR finds various applications like diagnostics and therapeutics in cancer, neurodegenerative disorders, genetic diseases, blindness, etc. This review discusses applications of CRISPR as a therapeutic in various disorders including several genetic diseases (including sickle cell anemia, blindness, thalassemia, cystic fibrosis, hereditary tyrosinemia type I, duchenne muscular dystrophy, mitochondrial disorders), Cancer, Huntingtons disease and viral infections (like HIV, COVID, etc.) along with the prospects concerning them. CRISPR-based therapy is also being researched and defined for COVID-19. The related mechanism of CRISPR has been discussed alongside highlighting challenges involved in therapeutic applications of CRISPR.

PMID:34453943 | DOI:10.1016/j.lfs.2021.119908

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Applications of CRISPR as a potential therapeutic - DocWire News

[PDF] CRISPR and CAS Gene Market Reflect Steady to Explore New Dimensions of Genome Engineering UNLV The Rebel Yell – UNLV The Rebel Yell

CRISPR Clinopathomics is an emerging science with applications in biotechnology and cellular biology. It is a genome-wide, single-gene-based technology to which various pharmacological agents can be added to promote disease resistance. It has great potential to address all life sciences challenges with an approach that allows for the production of multiple proteins that can attack many of lifes common challenges. This technology can potentially address most life-threatening diseases such as cancer, infections, inflammation, inherited disorders, diabetes, aging, neurological and other disorders, and chronic conditions, and possibly more.

FREE | Request Sample is Available @https://www.coherentmarketinsights.com/insight/request-sample/2598

*The Sample only consist ofTable of Content (ToC).Research Frameworkof the actual report.Research Methodologyadopted for it.

Major Company Profiles Covered in This Report:Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Technological advancements in the field of genome editing are expected to drive growth of the global CRISPR and CAS gene market during the forecast period. Key biopharmaceutical companies across the globe are focused on research and development activities, in order to innovate novel technologies in genome editing. For instance, in September 2020, Intellia Therapeutics Inc. presented new data demonstrating the efforts of In Vivo CRISPR/CAS9 edits to reduce a disease-causing protein or restore a functional protein.

Furthermore, in December 2020, Editas Medicine Inc., a genome editing company, submitted an investigational New Drug (IND) to the U.S. Food and Drug Administration (FDA), indicated for sickle cell disease. Such advancements and R&D activities have increased the demand for genome editing. Hence, these factors are expected to drive growth of the global CRISPR and CAS gene market during the forecast period. Moreover, increasing research in plant genome editing programs is expected to boost the global CRISPR and CAS gene market growth over the forecast period.

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However, off-target effects associated with CRISPR technology such as deletions, unintended point mutations, and translocations are expected to hamper the global CRISPR and CAS gene market growth over the forecast period. Among regions, North America is expected to witness significant growth in the global CRISPR and CAS gene market during the forecast period. This is owing to various companies involved in gene editing and the development of novel therapeutics across the region. Moreover, Asia Pacific is expected to register a robust growth rate over the forecast period, owing to increasing popularity of CRISPR technology in the region.

Key companies involved in the global CRISPR and CAS gene market are CRISPR Therapeutics, Inscripta, Inc., AstraZeneca, Mammoth Biosciences, Addgene, Synthego, Caribou Biosciences, Inc., Takara Bio, Inc., Cellectis, New England BioLabs, Editas Medicine, Inc., Merck KGaA, F. Hoffmann-La Roche Ltd., Intellia Therapeutics, Inc., and Danaher Corporation.

For instance, in November 2019, Caribou Biosciences Inc., a CRISPR genome editing company, announced the results of a new study demonstrating human genome engineering with TYPE I CRISPR-CAS systems.

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Some Notable Offerings by Coherent Market Insights Report on CRISPR and CAS Gene market:

We will provide you an analysis of the extent to which this CRISPR and CAS Gene market research report acquires commercial characteristics along with examples or instances of information that helps you to understand it better.

We will also help to identify customary/ standard terms and conditions, as offers, worthiness, warranty, and others.

Also, this report will help you to identify any trends to forecast growth rates.

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The data provided in the CRISPR and CAS Gene market report offers comprehensive analysis of important industry trends. Industry players can use this data to strategize their potential business moves and gain remarkable revenues in the upcoming period.

The report covers the price trend analysis and value chain analysis along with analysis of diverse offering by market players. The main motive of this report is to assist enterprises to make data-driven decisions and strategize their business moves.

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[PDF] CRISPR and CAS Gene Market Reflect Steady to Explore New Dimensions of Genome Engineering UNLV The Rebel Yell - UNLV The Rebel Yell

MRNA’s Next Chapter Has Nothing to Do With COVID-19 Vaccines – TIME

Its safe to say that before the development of the Pfizer-BioNTech and Moderna COVID-19 vaccines, most people hadnt thought about messenger RNA, or mRNA, since high school science classif ever. The molecule plays a pivotal role in the body, carrying the recipes for making various proteins to the parts of cells that produce them. But mRNA wasnt exactly a common phrase until Pfizer-BioNTech and Moderna harnessed the genetic materials power to teach the body to make a piece of a protein found on the COVID-19 virus surface, thus training it to fight the real thing, were it to attack.

The tremendous efficacy of mRNA-based COVID-19 vaccines has generated plenty of excitement about its potential use in vaccines for other diseases. And vaccines may be just the beginning. Last month, researchers used mRNA to deliver CRISPR gene-editing technology that could permanently treat a rare genetic disease in humansan advance that experts say has implications far beyond the treatment of a single condition.

Medical science research utilizing CRISPRa system that allows scientists to add, remove or change specific genetic information within the bodyhad already been advancing rapidly in recent years. Researchers have shown its potential for reversing blindness and sickle cell anemia, and to treat genetic diseases in animals. But new work described in the New England Journal of Medicine in June marks what researchers are calling the first time CRISPR has been shown to treat a genetic disorder when directly administered to human patients.

In this case, the technology was applied towards a therapy for transthyretin amyloidosis, a genetic disease that causes sufferers livers to produce a protein that eventually builds up to toxic levels. The diseases prevalence varies depending on patient demographicsit affects about one in 100,000 Americans of European descent, but as many as one in every 538 people in northern Portugal, for exampleand can be passed down to future generations. While there are drugs that can help patients manage the disease, the goal of the new research was to stop the problem at its source.

To imagine using [CRISPR] as a therapy for people, you need to figure out how to get these editing tools into the cells youre trying to fix. Thats where messenger RNA comes in, explains Daniel Anderson, a professor of chemical engineering at the Massachusetts Institute of Technology and a co-founder of CRISPR Therapeutics, which uses CRISPR technology to develop medications. Anderson was not involved in the research.

The research team, led by Dr. Julian Gillmore, an amyloidosis expert at the U.K.s Royal Free Hospital, programmed mRNA to deliver gene-editing instructions to the liver, shutting down the part responsible for producing the toxic protein. After a one-time injection of the drug, three of the six people in the trial saw an almost complete drop-off in protein production; the remaining three, who received a smaller dose, saw less dramatic results. It will take a few months to see if that accomplishment translates to symptom relief, but the early findings are promising. (The work was funded by pharmaceutical companies Intellia Therapeutics and Regeneron, which produce the injectable CRISPR drug.)

As Dr. John Leonard, Intellias president and CEO, puts it: mRNA is a way to make CRISPR gene editing come alive. CRISPR is the workhorse; mRNA encodes it.

In theory, the same general technology could be used to treat conditions beyond transthyretin amyloidosis. There are a host of diseases in the liver where this might work in an analogous manner, says Dr. Kenneth Chien, a senior professor of cardiology research at Swedens Karolinska Institutet and a co-founder of Moderna Therapeutics, who was not involved in the research. The most important aspect of this is the implications that the technology can be repurposed.

Chien has believed in mRNAs drug-development potential for more than a decade. When Moderna was founded in 2010, in fact, its chief goal was to develop mRNA-based drugs, not vaccines. (Chien no longer works at Moderna and is now an advisor to the pharmaceutical giant AstraZeneca.) He continues to work on an mRNA-based drug he hopes could eventually treat heart conditions.

The tricky part, Leonard says, is figuring out how to get a drug into different tissues, since the strategy for delivering CRISPR-based therapeutics varies depending on its target. The new research offers a blueprint for liver-based conditions, and Leonard believes similar approaches could be used in the near-term for bone-marrow, nervous-system and muscle diseases. The list theoretically grows from there, so long as researchers can fine-tune delivery.

COVID [vaccines are] a big success for mRNA, and if it does nothing else, its been great, Chien says. However, I think youre going to see the next chapter of mRNA is going to be as exciting, if not more so, than the story of mRNA vaccines.

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Write to Jamie Ducharme at jamie.ducharme@time.com.

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MRNA's Next Chapter Has Nothing to Do With COVID-19 Vaccines - TIME

CRISPR Therapeutics Provides Business Update and Reports – GlobeNewswire

- More than 45 patients have been dosed with CTX001 across CLIMB-Thal-111 and CLIMB-SCD-121 to date; completion of enrollment in both trials is expected in 2021-

-Received Orphan Drug Designation (ODD) for Phase 1 clinical trial of CTX130 for the treatment of T-cell lymphoma-

-Enrollment ongoing in CTX110, CTX120 and CTX130 clinical trials-

ZUG, Switzerland and CAMBRIDGE, Mass., July 29, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the second quarter ended June 30, 2021.

We concluded an important quarter in which we reported notable data from our hemoglobinopathies program while rapidly advancing our entire clinical and pre-clinical portfolio and our capabilities, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. Updated clinical data on CTX001 presented at EHA demonstrate consistency and durability, further validating the promise of a functional cure for sickle cell disease and beta thalassemia. We expect to report clinical data from our immuno-oncology programs later this year, and to file multiple INDs for our regenerative medicine and in vivo programs in the next 18 to 24 months.In addition, we continue to expand our portfolio and access best-in-class capabilities through collaborations, such as those recently announced with Capsida Biotherapeutics and Nkarta Therapeutics.

Recent Highlights and Outlook

Second Quarter 2021 Financial Results

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About the CRISPR-Vertex CollaborationVertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About CTX110CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.

About CARBONThe ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.

About CTX120CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma. CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.

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

CRISPR THERAPEUTICS word mark and design logo, CTX001, CTX110, CTX120, and CTX130 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics various clinical programs, including CTX001, CTX110, CTX120 and CTX130; (ii) the status of clinical trials and preclinical studies (including, without limitation, the expected timing of data releases and development, as well as initiation and completion of clinical trials) and development timelines for CRISPR Therapeutics product candidates; (iii) the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials or to support regulatory filings, including expectations regarding the CTX001 data; (iv) the actual or potential benefits of regulatory designations; (v) the potential benefits of CRISPR Therapeutics collaborations and strategic partnerships; (vi) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (vii) the sufficiency of CRISPR Therapeutics cash resources; and (viii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates (including, without limitation, availability and timing of results and whether such results will be predictive of future results of the future trials); uncertainties about regulatory approvals to conduct trials or to market products; the potential impacts due to the coronavirus pandemic such as (x) delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; (y) the timing and progress of clinical trials, preclinical studies and other research and development activities; and (z) the overall impact of the coronavirus pandemic on its business, financial condition and results of operations; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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

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

CRISPR Therapeutics AGCondensed Consolidated Statements of Operations(Unaudited, In thousands except share data and per share data)

CRISPR Therapeutics AGCondensed Consolidated Balance Sheets Data(Unaudited, in thousands)

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CRISPR Therapeutics Provides Business Update and Reports - GlobeNewswire

CRISPR Therapeutics AG (CRSP) Tops Q2 Earnings and Revenue Estimates – Yahoo Finance

CRISPR Therapeutics AG (CRSP) came out with quarterly earnings of $9.44 per share, beating the Zacks Consensus Estimate of $4.19 per share. This compares to loss of $1.30 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of 125.30%. A quarter ago, it was expected that this company would post a loss of $1.45 per share when it actually produced a loss of $1.51, delivering a surprise of -4.14%.

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

CRISPR Therapeutics AG, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $900.7 million for the quarter ended June 2021, surpassing the Zacks Consensus Estimate by 32.84%. This compares to year-ago revenues of $0.04 million. The company has topped consensus revenue estimates just once over the last four quarters.

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

CRISPR Therapeutics AG shares have lost about 21.8% since the beginning of the year versus the S&P 500's gain of 17.2%.

What's Next for CRISPR Therapeutics AG?

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

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

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

Story continues

Ahead of this earnings release, the estimate revisions trend for CRISPR Therapeutics AG was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #4 (Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.38 on $4.29 million in revenues for the coming quarter and $0.66 on $457.55 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the bottom 21% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

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

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CRISPR Therapeutics AG (CRSP) Tops Q2 Earnings and Revenue Estimates - Yahoo Finance

UMD Research Aims To Develop A Genome Editing Pipeline In Carrots, Optimize Crop Production – Bay Net

COLLEGE PARK, Md. -- With two new funding sources, Yiping Qi, associate professor in the Department of Plant Science & Landscape Architecture (PSLA) at the University of Maryland (UMD), continues his work to expand the reach and utility of CRISPR gene editing technologies. Funded by the United States Department of Agricultures National Institute of Food and Agriculture (USDA-NIFA), Qi and his team will be testing new delivery technologies for CRISPR-Cas12a to develop a pipeline for genome editing in carrots. This could lead to the production of more nutritious and hypoallergenic carrot varieties that can be quickly introduced into the marketplace. Additionally, Qi will continue his search for novel CRISPR-Cas12a variants with funding from the Maryland Innovation Initiative (MII), with the ultimate goal of finding more CRISPR tools that are optimized for crop production.

With this new USDA funding, we are excited to showcase CRISPR-Cas12a gene editing technologies that my lab has been working on in carrots as an important vegetable, says Qi. The major innovations in this work are really in the delivery mechanisms that can make targeted and precise edits without the production of a highly regulated GMO [genetically modified organism]. But we also want to explore more CRISPR-Cas12a variants that can be useful for crop production, so the MII grant provides that support.

As Qi explains, most current methods for targeted mutations or precise genome edits rely on the use of transgenes (transferring a gene from one organism to another) that would then be regulated as GMOs. This is typically done by delivering genetic material with agrobacteria used to infect the plants and transfer the desired trait. However, for carrots, Qi is testing different delivery methods that use readily available proteins and guiding molecules to deliver the same material without using agrobacteria and transgenes. This method is new to carrots, and would allow new varieties to make it to market much faster without the need for GMO regulations.

Hopefully, we can take advantage of these technologies to develop some interesting and useful plants that have consumer benefits, says Qi. For example, we are targeting a gene to help the carrot accumulate more beta carotene, which can boost nutritional value. We are also targeting two genes that can potentially be knocked out to create a hypoallergenic carrot that could be eaten by those with certain allergies.

These varieties could not only be useful in and of themselves, but the work will establish a process for genome editing in carrots that hasnt been previously developed. This will save substantial time over traditional breeding, and Qi hopes will inspire many researchers and breeders to consider the possibilities of this technology in crops like carrots.

The whole project is to develop new technology for genome editing in more niche or minor crops that can have major impacts, stresses Qi. Not much work has been previously done in carrots, and I hope this will open up a lot of doors for gene editing in other root vegetables and more.

In addition to this work with the known variants of CRISPR-Cas12a, Qi is continuing his search for novel variants that are optimal for crop genome editing. Qis recent work contributing six novel variants of CRISPR-Cas12a (never before proven in plants) was named UMD Life Sciences Innovation of the Year. These patent-pending tools widen the scope of what CRISPR-Cas12a can do in plants, which can help to produce food more effectively to fight hunger.

With new funding from MII, Qi will continue to explore new patentable CRISPR variants, hoping to find more tools that work efficiently at lower temperatures. For work in human cells, gene editing is happening at the temperature of the human body, which is almost 100 degrees Fahrenheit, says Qi. This is the optimal temperature for most CRISPR systems, but this isnt the best temperature for doing work in plants. All that work needs to be done around room temperature where plants can comfortably grow. So finding tools that are optimized for this lower temperature application is important for advancing genome editing in crops.

Qis team has established a proprietary pipeline for identifying new candidate CRISPR variants, and he will first test these candidates in rice and tomatoes to expand the scope of gene editing in crops.

This work is funded by the United States Department of Agricultures National Institute of Food and Agriculture (USDA-NIFA), Award #2021-67013-34554, and the Maryland Innovation Initiative (MII).

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UMD Research Aims To Develop A Genome Editing Pipeline In Carrots, Optimize Crop Production - Bay Net

University Of Maryland Professor Gets New Funds To Continue Research To Enhance Crop Production – CBS Baltimore

COLLEGE PARK, Md. (WJZ) A professor at the University of Maryland in College Park has received new funding from two sources to continue research into CRISPRgenomeeditingtechnologies, withthegoal of enhancing crop productionand feeding a growing global population, according to a university statement.

Yiping Qi, associate professor in the Department of Plant Science & Landscape Architecture, received the funds from the U.S. Department of Agricultures National Institute of Food and Agriculture to develop a pipelineforgenomeediting in carrots that could lead to more nutritious and hypoallergenic carrot varieties in stores. He also received funding from theMaryland Innovation Initiative to continue his search for novel CRISPR-Cas12 variants. That goal is to find more CRISPR tools that are optimized for crop production.

Qi is testing delivery methods that use readily available proteins and guiding molecules to deliver the same material most current methods for targeted mutations use, transferring a gene from organisms to another that would then be regulated as genetically modified organisms.

This method is new to carrots and would allow new varieties to make it to market more quickly without the need for GMO regulations, according to the statement.

With the MII funding, he will continue exploring new patentable CRISPR variants, hoping to find more tools that work efficiently at lower temperatures.

Qis team has established a proprietary pipeline for identifying new candidate CRISPR variants, and he will first test these candidates in rice and tomatoes to expand the scope of gene editing in crops.

Read this article:
University Of Maryland Professor Gets New Funds To Continue Research To Enhance Crop Production - CBS Baltimore

A revolution against cancer is unfolding and were just getting started – ZME Science

Its hard to put just one label on Aaron Ciechanover. He was awarded the Nobel Prize in Chemistry for characterizing the method that cells use to degrade and recycle proteins usingubiquitin, but his background stems from biology, and he was also trained as a medical doctor and a surgeon. When it comes to understanding the intricacies around human health, few people on Earth can claim the broad view that Ciechanover has.

Which is why, when he says hes excited about whats to come in medicine, its hard not to share his excitement.

The future of medicine is going to be revolutionary, Ciechanover said at the 2021 Lindau Nobel meeting, which took place online this year due to the pandemic. The meetings bring together Nobel laureates and young scientists to foster scientific exchange.

Back in the days when Ciechanover was studying medicine, he recalls, things were very different.

Lets say, if a patient had a tumor, we were not interested in the molecular mechanism that underlies the tumor development, because we did not have the tools to study it, he says.

The procedure was simplistic and straightforward. Doctors would look at the imaging facilities they had access to at the time (either X-ray, CT Scan, or MRI) and decide whether the tumor could be operated on. Surgery was generally the preferred procedure because the tumor mass could be extracted. If the tumor was too big or was touching essential organs, then doctors would try to decrease its size using chemotherapy or radiation, and then try surgery.

But these were (and still are) very harsh measures, with harsh side effects.

They are like shooting a fly with a cannon. They are not discriminating between the healthy tissue and the sick tissue, they are very difficult to direct, Ciechanover explains.

Then, at the turn of the century, a revolution started unfolding. In 2000, a landmark paper published in the journal Nature opened the floodgates of genetic discovery.

I remember it very well, this exciting day when Nature magazine came out with the first human genome. The first human genome gave us the information, the library of what we are made of. This was really the very beginning, but the last 20 years have seen enormous progress. We are now able to diagnose the disease much better [..] and we are able to analyze tumors or any other disease at the molecular level.

Heres a sense of how much things have progressed. The price of whole human genome sequencing was around $2.7 billion in 2003. Today, its under $100. Advancements in technology and decrease in price has made genetic and molecular analyses more widely available, and its not about to stop.

We are developing dedicated tools to stop the tumor or the disease at large, with a very gentle tool directing a bullet direction at the underlying mechanism, Ciechanover adds.

Even with conventional medicine, healthcare has benefited tremendously. Things like imaging, antibiotics, vaccines, operating procedures, and so on, have made a tremendous difference in how we treat patients. But now we are into a much bigger revolution, Ciechanover believes. He foresees a future where the very definition of medicine will change. Finally, we will start treating patients, not diseases, and patients will receive individualized treatments.

Tasuku Honjo is also optimistic. He believes that while cancer wont be eradicated anytime soon, theres a good chance well be able to keep most cancers in check.

Honjo should know. He and his colleagues discovered a molecule called programmed cell death protein 1 (PD-1). They also showed that this molecule functions as a sort of braking system for acquired immunity making sure that your immune system doesnt go into overdrive and cause autoimmune disease. But too much PD-1, and the immune system would not do its job properly.

For instance, several tumors produce something similar to PD-1, which helps the tumors escape the immune system. But if PD-1 could be suppressed in cancer patients, then we could use peoples own immune systems to fight cancer. This is exactly what Honjo says can help keep cancers in check.

Honjo and colleagues found that blocking PD-1 in mice can cure tumors by reactivating acquired immunity in 2002. Then, in a landmark moment in 2014, the treatment of cancer in humans by PD-1 blockade was approved by regulatory bodies in Japan and the USA. Now, there are over 1,000 clinical trials happening in the world, and PD-1 treatments seem to be effective against a wide variety of cancers, with long-lasting positive effects.

Another Nobel-winning discovery that could help our fight against cancer is CRISPR/Cas9.

CRISPR is becoming a mainstream methodology used in many cancer biology studies because of the convenience of the technique, says Jerry Li of NCIsDivision of Cancer Biology.

CRISPR is a relatively simple but very powerful and accurate way to edit genes. It was inspired by nature, from a defense mechanism some bacteria use to protect themselves against viral invasions. The bacterium captures snippets of any virus intruders DNA and stores it as segments called CRISPRs. If the same virus returns and tries to attack again, the bacterium searches its DNA library and releases an enzyme called Cas to slice up the invaders DNA.

Gene editing is not new, ProfessorEmmanuelle Charpentier, one of the pioneers behind CRISPR explained at the Lindau Nobel meeting. But thanks to the work of Charpentier and Jennifer Doudna, who were awarded the 2020 Nobel Prize, we have access to unprecedented tools.

The first CRISPR cancer therapy was launched in 2019. The goal of the study is to edit patients own immune cells to better detect and kill cancer. The treatment is safe, and early results are encouraging but CRISPR is still just getting warmed up.

This [trial] was really a proof-of-principle, feasibility, and safety thing that now opens up the whole world of CRISPR editing and other techniques of [gene] editing to hopefully make the next generation of therapies, said Edward Stadtmauer, M.D., of the University of Pennsylvania, who is involved with the research.

Weve come a long way in our fight against cancer in the past half-century, but despite improving diagnosis and treatments, theres still more work to be done if we want to keep cancer in check. But the tools we need to do so are now coming in.

With approaches like CRISPR or PD-1, researchers can develop customized, efficient treatments with few side effects. Thanks to the likes of Honjo, Charpentier, and Ciechanover, we are witnessing a new revolution of medicine, and its hard not to share their enthusiasm for whats to come.

Its still early days and there are plenty of hurdles to be overcome, but the science is progressing in leaps and bounds. It may not be today or tomorrow, but were gathering the weapons to fight cancer and its shaping up to be a big arsenal.

Read the original:
A revolution against cancer is unfolding and were just getting started - ZME Science

Bear of the Day: CRISPR Therapeutics (CRSP) – Yahoo Finance

CRISPR Therapeutics (CRSP) is one of my favorite biotech companies as the big leader among gene-editing pioneers.

But I had to let the stock go -- right before shares launched into the December ASH meeting (American Society of Hematology) -- because analysts were so bearish on the outlook for when the R&D pipeline would produce any revenues, much less profits.

Of course, talking about revenues and profits for world-changing, early-stage medical science is almost always a non sequitur.

Still in late summer, I let go of my CRSP shares for a 71% gain. But as I describe in this September video and article, it was not the first, nor the last, of great trading gains in the greatest of CRISPR companies...

CRISPR Stocks: Buy or Trade?

In fact, here's the actual trading record from my Healthcare Innovators portfolio of my previous 3 CRSP trades...

So why did CRSP launch from $110 to $210 in December and January?

It was mostly about investors recognizing that the company's early data in treating debilitating illnesses like Sickle Cell Disease (SCD) could indeed become world-changing for millions afflicted with the genetic impairment to their red blood cells.

SCD comprises a group of disorders that cause red blood cells to become misshapen and break down. Red blood cells contort into a sickle shape, and die early, leaving a shortage of healthy red blood cells (sickle cell anemia), and can block blood flow causing pain (sickle cell crisis). Infections, pain, and fatigue are symptoms of sickle cell disease. Current treatments include frequent medications, blood transfusions and, in extreme cases, a bone-marrow transplant -- but no cures.

As Antonio Regalado wrote in the MIT Technology Review wrote last week, "The burden of sickle-cell, an inherited disease that shortens lives by decades (or, in poor regions, kills during childhood), falls most heavily on Black people in equatorial Africa, Brazil, and the US. HIV has also become a lingering scourge: about two-thirds of people living with the virus, or dying from it, are in Africa."

I explained some of this potential in this vlog on December 10...

CRISPR Gene Editing: Owning the Future of Medicine

A secondary "igniter" of all CRISPR stocks launching higher in Dec-Jan (besides my video commentaries linked above) was the investment activity of Cathie Wood and her revolutionary ETF firm ARK Invest.

I produced a video and article about her one-woman investor revolution in early January with her monster ETFs ARK Innovation (ARKK) and ARK Genomics (ARKG)...

How Cathie Put the Wood to Wall Street: TSLA, SQ, ROKU, CRSP, BIDU

There will be a time to buy CRSP again. But as with all emerging Biotechs, you sometimes have to wait for the next clinical data catalyst -- or an M&A one.

I'm betting the latter is the sooner driver of the next move from a $10 billion market cap to a $20 billion one.

Meanwhile, I own the smaller pair of Editas Medicine (EDIT) and Intellia Therapeutics (NTLA) near $5 billion.

Stay CRSPy!

Cooker

Kevin Cook is a Senior Stock Strategist for Zacks Investment Research where he runs the Healthcare Innovators portfolio.

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Bear of the Day: CRISPR Therapeutics (CRSP) - Yahoo Finance

Meridian Bioscience, CRISPR Therapeutics, Tilray and Uber highlighted as Zacks Bull and Bear of the Day – Yahoo Finance

For Immediate Release

Chicago, IL February 11, 2021 Zacks Equity Research Shares of Meridian Bioscience, Inc. VIVO as the Bull of the Day, CRISPR Therapeutics AG CRSP as the Bear of the Day. In addition, Zacks Equity Research provides analysis on Tilray, Inc. TLRY and Uber Technologies, Inc. UBER.

Here is a synopsis of all four stocks:

Meridian Bioscienceis a $1 billion provider of diagnostic test kits for gastrointestinal and respiratory infectious diseases. The company is expected to grow sales 32% this year to $335 million.

And after reporting a strong beat-and-raise quarter last week, analysts had to boost their EPS estimates over 30% from $1.24 to $1.63. So VIVO is back to a Zacks #1 Rank, sporting a projected 52% rise in profits.

I wrote about VIVO in early January and said it wasn't too late for investors to grab hold of this profit rocket near $20 per share...

"Bottom line on VIVO: I always pay attention to small companies growing their sales rapidly as they could become acquisition targets by larger biopharma or MedTech players. Buying VIVO near $20 offers excellent risk/reward, with or without an M&A suitor."

And then last week, right before the company report, I produced a video and article where I talked about why the COVID-19 testing stocks were under-appreciated given their fantastic growth...

Biotech Bonanza: COVID Launches Science at Warp Speed

Well here we are as VIVO pushed to 13-year highs above $30 with the earnings surprise and strong upside guidance.

Following the quarterly report, Piper Sandler analyst Steven Mah, a consistently bullish VIVO fan, raised his price target on Meridian Bioscience to $34 from $26 and reiterated an Overweight rating. Mah believes the company guidance was once again "very conservative" citing management's "pragmatic approach given the limited visibility on the durability of COVID-19 tailwinds."

Mah explained his increased confidence in Meridian's longer-term COVID-19 tailwinds given the Biden Administration's testing stance, slower than expected vaccine rollout, and new strain emergence.

Story continues

Meridian Bioscience develops, manufactures, distributes, and sells diagnostic test kits primarily for gastrointestinal and respiratory infectious diseases, and elevated blood lead levels worldwide. The company operates through Diagnostics and Life Science segments. They describe their mission as helping providers make better diagnostic decisions with a focus on gastrointestinal, neonatal, pediatrics, and respiratory conditions.

The Diagnostics segment offers testing platforms, including real-time PCR (polymerase chain reaction) amplification under the Revogene brand; isothermal DNA amplification under the Alethia brand; lateral flow immunoassay using fluorescent chemistry under the Curian brand; rapid immunoassay under the ImmunoCard and ImmunoCard STAT! brands; enzyme-linked immunoassays under the PREMIER brand; anodic stripping voltammetry under the LeadCare and PediaStat brands; and urea breath testing for H. pylori under the BreathID brand.

I have written often in the past few months of specialized diagnostic companies likeQuidelandHologicas they build new revenue streams from SARS-CoV-2 testing. These revenue streams are likely sustainable as the virus mutates and requires modified tests.

And I recently bought shares of VIVO for the Zacks Healthcare Innovators portfolio because I liked the growth outlook for this small player in a rapidly expanding market for rapid diagnostics -- including coronavirus testing which will continue to be part of our lives for years to come, even with vaccines.

While Meridian Bioscience is a David among diagnostic Goliaths, its long and fascinating history surprised me. From the company website...

In 1977, Bill Motto founded Meridian Bioscience on a $500 investment in his Cincinnati homes basement. Meridians first product was distributing a rapid fungal test developed by the University of Kentucky. While calling on his hospital and research customers, Bill noticed there was no easy, clean way to transport patient samples. He developed the innovative Para-Pak stool transport system to meet this need.

As the product line grew, so did Meridians research and development, leading to a breakthrough in 1982 with a 10-minute rapid test for strep throat. Before the Meridian test, doctors would have to wait for two to three days for a culture result. Innovation continued as the company brought several cutting edge diagnostic technologies to market, including a DNA testing platform and first-of-their-kind tests for C. difficile, E. coli, H. pylori amongst others.

The new bottom line on VIVO:I continue to hold the shares and would recommend new positions between $25 and $27 looking for new bull market highs above $30 by June.

Disclosure: I own shares of QDEL, HOLX, and VIVO for the Zacks Healthcare Innovators portfolio.

CRISPR Therapeuticsis one of my favorite biotech companies as the big leader among gene-editing pioneers.

But I had to let the stock go -- right before shares launched into the December ASH meeting (American Society of Hematology) -- because analysts were so bearish on the outlook for when the R&D pipeline would produce any revenues, much less profits.

Of course, talking about revenues and profits for world-changing, early-stage medical science is almost always a non sequitur.

Still in late summer, I let go of my CRSP shares for a 71% gain. But as I describe in this September video and article, it was not the first, nor the last, of great trading gains in the greatest of CRISPR companies...

CRISPR Stocks: Buy or Trade?

So why did CRSP launch from $110 to $210 in December and January?

It was mostly about investors recognizing that the company's early data in treating debilitating illnesses like Sickle Cell Disease (SCD) could indeed become world-changing for millions afflicted with the genetic impairment to their red blood cells.

SCD comprises a group of disorders that cause red blood cells to become misshapen and break down. Red blood cells contort into a sickle shape, and die early, leaving a shortage of healthy red blood cells (sickle cell anemia), and can block blood flow causing pain (sickle cell crisis). Infections, pain, and fatigue are symptoms of sickle cell disease. Current treatments include frequent medications, blood transfusions and, in extreme cases, a bone-marrow transplant -- but no cures.

As Antonio Regalado wrote in the MIT Technology Review wrote last week, "The burden of sickle-cell, an inherited disease that shortens lives by decades (or, in poor regions, kills during childhood), falls most heavily on Black people in equatorial Africa, Brazil, and the US. HIV has also become a lingering scourge: about two-thirds of people living with the virus, or dying from it, are in Africa."

I explained some of this potential in this vlog on December 10...

CRISPR Gene Editing: Owning the Future of Medicine

A secondary "igniter" of all CRISPR stocks launching higher in Dec-Jan (besides my video commentaries linked above) was the investment activity of Cathie Wood and her revolutionary ETF firm ARK Invest.

I produced a video and article about her one-woman investor revolution in early January with her monster ETFsARK Innovation:

How Cathie Put the Wood to Wall Street: TSLA, SQ, ROKU, CRSP, BIDU

There will be a time to buy CRSP again. But as with all emerging Biotechs, you sometimes have to wait for the next clinical data catalyst -- or an M&A one.

I'm betting the latter is the sooner driver of the next move from a $10 billion market cap to a $20 billion one.

Stay CRSPy!

Cooker

Kevin Cook is a Senior Stock Strategist for Zacks Investment Research where he runs the Healthcare Innovators portfolio.

Markets continued to hover close to the zero-line as of Wednesdays close, with just the Dow finishing in the green among major indexes. The blue-chips rose 0.20% for a new all-time high, while the Nasdaq, S&P 500 and Russell 2000 all took a breather: -0.25%, -0.03% and -0.72% on the day.

One of the major pot stocks we discussed yesterday in this space,Tilray, continues its big run in what looks more like the latest short-squeeze stock, with Reddit groups now piling into the marijuana-based pharmaceutical company of late. Shares shot up another 51% Wednesday, following a +44% performance yesterday; Tilray is now up 400% in just the last month alone.

For sure, increased acceptance in U.S. states and countries around the world are a reason for the stock to do well. However, its market cap has more than doubled and the company has no P/E because it is forecast for negative earnings both in the upcoming quarterly report and full fiscal year. Tilray is up another 10% in late trading, up near $71 per share. This stock was trading at $19 per share on February 1st.

Uber followed a nice 6% bump in regular-day Wednesday trading with a worse-than-expected fiscal Q3 report, missing on the bottom line by a penny to -54 cents per share (though still better than the -64 cents the company reported in the year-ago quarter). Revenues grew $3.17 billion in the quarter, well off the expected pace of $3.55 billion in the Zacks consensus. Yet shares are only selling off minimally in the after-market; the company still says its on track to its profitability goal in 2021.

Though Uber posted a net loss per year of $6.8 billion on ride-sharing revenues down 52% year over year, its Uber Eats delivery service grew 224% in its fiscal Q3. Monthly active platform consumers gained a million more than predicted in the quarter, +93 million. And when one figures in the ride-sharing comeback seemingly inevitable as the Covid-19 pandemic is finally beaten back with vaccinations, we see that Uber looks to have weathered its worst-possible storm and survived.For more on UBER's earnings, click here.

Speaking of the coronavirus, nearly 45 million vaccination doses have now been administered, and the post-holiday season peak now looks to have been successfully scaled. We are now back to 7-day case rates back where they were in October and, importantly, pointed in the right direction. More than 27 million Americans have reportedly contracted Covid-19, leading to more than 466K fatalities. More good news: the death rate is now flat for those whove gotten the worst of the disease.

Questions or comments about this article and/or its author? Click here>>

Experts extracted 7 stocks from the list of 220 Zacks Rank #1 Strong Buys that have beaten the market more than 2X over with a stunning average gain of +24.9% per year.

These 7 were selected because of their superior potential for immediate breakout.

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