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Archive for the ‘Crispr’ Category

Here’s Why CRISPR Therapeutics Jumped 42.2% in November – The Motley Fool

What happened

Shares of CRISPR Therapeutics (NASDAQ:CRSP) gained over 42% last month, according to data provided by S&P Global Market Intelligence. The company provided the first glimpse of CTX001, a therapy based on CRISPR gene editing, in sickle cell disease and transfusion-dependent beta-thalassemia (TDT). While the preliminary results were from only two patients -- one in each indication -- they demonstrated promising potential for the approach.

Investors cheered the update, which was the first from any company developing CRISPR-based tools. The news sent several gene-editing stocks higher. Shares of Editas Medicine and Intellia Therapeutics rose as well, although super-early success for CTX001 means next to nothing for industry peers. More meaningful might be the implications for Vertex Pharmaceuticals (NASDAQ:VRTX), the collaboration partner of CRISPR Therapeutics for CTX001, which could be handsomely rewarded for its early bet on the gene-editing technique.

Image source: Getty Images.

CRISPR Therapeutics reported that after one dose of CTX001, the individual with TDT was transfusion independent through the nine-month mark. The patient had averaged 16.5 blood transfusions per year in the two years prior to the clinical trial.

Similarly, after one dose of CTX001, the individual with sickle cell disease was free of vaso-occlusive crises (painful blockages of small blood vessels caused by abnormally shaped blood cells) at the four-month mark. The patient had averaged seven such events in the two years prior to the study. Both patients achieved promising results for an important biomarker as well.

How excited are investors? Well, CRISPR Therapeutics announced a public stock offering days after providing the clinical update, but after briefly tumbling, shares actually shook off the dilution concerns and continued ascending through the end of the month. The business ended September with over $629 million in cash and raised up to $315 million from the November offering.

Simply put, the early update from CTX001 is about as good as investors could have hoped for, although the results are obviously very preliminary. The studies are designed to enroll dozens of patients and track them for two years. Will the results prove durable? Will adverse events or other safety issues crop up as the studies mature? Investors will have to remain patient, but CRISPR Therapeutics remains the top gene-editing stock.

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Here's Why CRISPR Therapeutics Jumped 42.2% in November - The Motley Fool

Chinas CRISPR babies: Read exclusive excerpts from the unseen original research – MIT Technology Review

Earlier this year a source sent us a copy of an unpublished manuscript describing the creation of the first gene-edited babies, born last year in China. Today, we are making excerpts of that manuscript public for the first time.

Titled Birth of Twins After Genome Editing for HIV Resistance, and 4,699 words long, the still unpublished paper was authored by He Jiankui, the Chinese biophysicist who created the edited twin girls. A second manuscript we also received discusses laboratory research on human and animal embryos.

The metadata in the files we were sent indicate that the two draft papers were edited by He in late November 2018 and appear to be what he initially submitted for publication. Other versions, including a combined manuscript, may also exist. After consideration by at least two prestigious journals, Nature and JAMA, his research remains unpublished.

The text of the twins paper is replete with expansive claims of a medical breakthrough that can control the HIV epidemic. It claims successa word used more than oncein using a novel therapy to render the girls resistant to HIV. Yet surprisingly, it makes little attempt to prove that the twins really are resistant to the virus. And the text largely ignores data elsewhere in the paper suggesting that the editing went wrong.

We shared the unpublished manuscripts with four expertsa legal scholar, an IVF doctor, an embryologist, and a gene-editing specialistand asked them for their reactions. Their views were damning. Among them: key claims that He and his team made are not supported by the data; the babies parents may have been under pressure to agree to join the experiment; the supposed medical benefits are dubious at best; and the researchers moved forward with creating living human beings before they fully understood the effects of the edits they had made.

Because these documents relate to one of the most important public interest issues of all timethe ability to change human heredity using technologywe here present excerpts from the twins manuscript, together with some of the experts comments, and explain the questions they raise. The excerpts are in the order in which they appear in the paper.

To understand why the manuscripts have remained unpublished up to now, read the accompanying article on He's attempts to get them into scientific journals. For the case for making their content public, read the op-ed by Kiran Musunuru, a gene-editing specialist at the University of Pennsylvania, who argues the Chinese data shows that gene-editing for reproduction is unsafe and premature.

1. Why arent the doctors among the papers authors?

The manuscript begins with a list of the authors10 of them, mostly from He Jiankuis lab at the Southern University of Science and Technology, but also including Hua Bai, director of an AIDS support network, who helped recruit couples, and Michael Deem, an American biophysicist whose role is under review by Rice University.

Its a small number of people for such a significant project, and one reason is that some names are missingnotably, the fertility doctors who treated the patients and the obstetrician who delivered the babies. Concealing them may be an attempt to obscure the identities of the patients. However, it also leaves unclear whether or not these doctors understood they were helping to create the first gene-edited babies.

To some, the question of whether the manuscript is trustworthy arises immediately.

Hank Greely, professor of law, Stanford University: We have no, or almost no, independent evidence for anything reported in this paper. Although I believe that the babies probably were DNA-edited and were born, theres very little evidence for that. Given the circumstances of this case, I am not willing to grant He Jiankui the usual presumption of honesty.

2. The researchers own data dont support their main claims

The abstract, or summary, lays out the aim of the projectto generate humans resistant to HIVand the main results. It states that the team was successfully able to reproduce a known mutation in a gene called CCR5. The small percentage of people born naturally with this mutation, known as CCR5 delta 32, can be immune to infection by HIV.

But the summary goes well beyond what the data in the paper can back up. Specifically, as well see later, the team didnt actually reproduce the known mutation. Rather, they created new mutations, which might lead to HIV resistance but might not. They never checked to see, according to the paper.

Fyodor Urnov, genome-editing scientist, Innovative Genomics Institute, University of California, Berkeley: The claim they have reproduced the prevalent CCR5 variant is a blatant misrepresentation of the actual data and can only be described by one term: a deliberate falsehood. The study shows that the research team instead failed to reproduce the prevalent CCR5 variant. The statement that embryo editing will help millions is equal parts delusional and outrageous, and is akin to saying that the 1969 moonwalk brings hopes to millions of human beings seeking to live on the moon.

Rita Vassena, scientific director, Eugin Group: Approaching this document, I was hoping to see a reflective and mindful approach to gene editing in human embryos. Unfortunately, it reads more like an experiment in search of a purpose, an attempt to find a defensible reason to use CRISPR/Cas9 technology in human embryos at all costs, rather than a conscientious, carefully thought through, stepwise approach to editing the human genome for generations to come. As the current scientific consensus indicates, the use of CRISPR/Cas9 in human embryos destined to give rise to a pregnancy is, at this stage, unjustified and unnecessary, and should not be pursued.

3. Gene-editing embryos wont bring HIV under control, especially in the worst-affected countries

The end of the abstract and beginning of the main text is where the authors justify their research. They suggest that gene-editing babies could save millions of people from HIV infection. Our commenters call this claim preposterous and ludicrous, and point out that even if the CRISPR method works to create people who are HIV resistant, its unlikely to be practical in places where HIV is rampant, such as in the southern part of Africa.

Rita Vassena: This work offers little justification for the editing and subsequent transfer of human embryos to generate a pregnancy. The idea that editing-derived embryos may one day be able to control the HIV epidemic, as the authors claim, is preposterous. Public health initiatives, education, and widespread access to antiviral drugs have been shown to control the HIV epidemic.

Hank Greely: That this is a plausible way to control the HIV epidemic seems ludicrous. If every baby in the world were given this variation (beyond unlikely), it would begin to affect HIV infection substantially in 20 to 30 years, by which time we should have much better methods of stemming the epidemicas well as existing methods that have substantially, if not yet sufficiently, slowed it. The 64% increase in infections in China (if true) is from a very low base. China has a substantially lower rate of HIV infection than Western countries. The situation in some developing countries remains more serious. But that this high-tech response is likely to be helpful in those countries is not plausible.

4. The parents might have wanted to take part for the wrong reasons

Contrary to some interpretations, the point of using CRISPR on the babies DNA wasnt to prevent them from catching HIV from their father, who was infected. As the paper describes, this was achieved by sperm washing, a well-established technique. Instead, the purpose of the editing was to give the children immunity to HIV later in life. Thus, the experiment didnt provide clear, immediate medical benefits to either the parents or the children. Why did the couple agree? One reason may have been to access fertility treatment at all.

Rita Vassena: I find it worrying that the husband in the couple offered this experimental genome editing was positive to HIV infection, as one can imagine the unnecessary emotional pressure on the couple to consent to a procedure offering no improvement to the patient and their childrens health, but carrying a potential risk of negative consequences. It is worth remembering that HIV infection is not passed on through generations like a genetic disease; the embryo needs to catch the infection. For this reason, preventive measures such as controlling the viral load of the patient with appropriate drugs, and careful handling of the gametes during IVF, can avoid contagion very efficiently. Current assisted reproductive techniques ensure safe procreation for HIV-positive men and women, avoiding both horizontal (between partners) and vertical (between parent and embryo/fetus) transmission, making the editing of embryos in these cases unnecessary. In fact, the couple in the experiment did undergo such ART procedures, consisting in this case of an extended wash of semen to remove all seminal fluid, which may harbor HIV. Extended sperm washing has been used for almost two decades in IVF laboratories worldwide and in thousands of patients; in ours and others experience, it is safe for both parents and their future children and does not entail invasive manipulation of embryos.

Jeanne OBrien, reproductive endocrinologist, Shady Grove Fertility: Being HIV-positive in China carries a significant social stigma. In spite of intense familial and societal obligations to have a child, HIV-positive patients have no access to treatment for infertility. The social context in which the clinical study was carried out is problematic and it targeted a vulnerable patient group. Did the study provide a genetic treatment for a social problem? Was this couple free from undue coercion?

5. The gene edits werent the same as the mutations that confer natural HIV resistance

Here, the researchers describe the changes CRISPR actually made to the twins. They removed a few cells from the IVF embryos to look at their DNA, and found that edits intended to disable the CCR5 gene had indeed taken hold.

But while they expect these edits to confer HIV resistance by nullifying the activity of the gene, they cant know for sure, because the edits are similar but not identical to CCR5 delta 32, the mutation that occurs in nature. Moreover, only one of the embryos had edits to both copies of the CCR5 gene (one from each parent); the other had only one edited, giving partial HIV resistance at best.

Hank Greely: Successfully is iffy here. None of the embryos got the 32-base-pair deletion to CCR5 that is known in millions of humans. Instead, the embryos/eventual babies got novel variations, whose effects are not clear. As well, what does partial resistance to HIV mean? How partial? And was that enough to justify transferring the embryo, with a CCR5 gene never before seen in humans, to a uterus for possible birth?

6. There could have been other, unwanted CRISPR edits

CRISPR isnt a perfect tool. Trying to edit one gene can sometimes create other, unintended changes elsewhere in the genome. Here the team discusses their search for such unwanted edits, called off-target mutations, and say they found just one.

The search was incomplete, however, and the manuscript also glosses over a key point: any cells the researchers took from the early-stage embryos to test didnt, therefore, actually contribute to the twins bodies. The remaining cells, the ones that would multiply and grow to become the twins, could have harbored off-target effects too, but there would have been no way to know that in advance of starting the pregnancy.

Fyodor Urnov: An egregious misrepresentation of the actual data that can, again, only be described as a blatant falsehood. It is technically impossible to determine whether an edited embryo did not show any off-target mutations without destroying that embryo by inspecting every one of its cells. This is a key problem for the entirety of the embryo-editing field, one that the authors sweep under the rug here.

7. The doctors treating the couple may not have known what was going on

Reporting by a variety of news outlets, including the Wall Street Journal, has charged that Hes team tricked doctors by switching blood samples and that not all of them knew they were involved in creating gene-edited children. If true, thats a problem, since its the duty of doctors to do what is in the best interest of the patient.

Jeanne OBrien: The IVF procedure described follows the same steps and time line whether or not CRISPR is used for genome editing. The Chinese physicians who performed the IVF may have been unaware of the fathers HIV status or that the embryos were genetically modified. He Jiankui would have only needed a willing embryologist to inject CRISPR at the time of insemination. Hes comments make it appear as if the physicians who performed the IVF were not involved in the subsequent decision regarding which embryos to select for transfer. This is a wake-up call to physicians involved in IVF: the science and technology will continue to progress, and desperate couples with infertility may overlook the unknowns or believe the technology is proven safe. Once we, the infertility physicians, knowingly transfer an embryo with germline editing, we are in essence confirming the safety of the modification to the parents and the future child. Is it ever possible to know that?

8. The manuscript misrepresents when the babies were born

By now, several media reports and people familiar with the research have established that the twins were born in October, not November. Why did Hes team include a false date? It may have been to protect the anonymity of the patients and their twins. In a country the size of China, there could be more than ten thousand sets of twins born each month. The falsified date may have been an attempt to make their reidentification even more difficult.

9. Its not clear if there was a proper ethics review

The paper includes an exceptionally brief discussion of ethics. It says the research plan was registered with the China Clinical Trial Registry, but in fact the public registration occurred only after the twins were born.

Hank Greely: Registered when? The answer is on November 8, 2018, after the births and very shortly before they were announced, and probably in order to increase publication potential. This was not a normal registration. Maybe there was an ethics approvalthough that hospital has denied it. Who is telling the truth? Not sure well ever know. The phrase we were told about a comprehensive ethics review is not very powerful evidence. The article also does not discuss the Chinese ban on assisted reproductive services for HIV-positive parents. It has been reported that He had other men pretend to be the intended fathers for purpose of the required HIV tests. The article doesnt say this. It seems to me likely to be trueand damning. If true, it means He defrauded the Chinese regulatory process.

10. The researchers didnt test whether the HIV immunity worked before creating living human beings

Here the Chinese team outlines their plan to collect blood from the twins to see if their edited cells really resist HIV. That is something they could have tried to learn ahead of time, before creating the girls. Before transferring the embryos, they could have kept them frozen while they made identical edits in laboratory cells and tested the effects of HIV on those cells.

Fyodor Urnov: This statement proves that the research team placed their interests above those of the couple who donated the embryos and of their prospective children. There is zero evidence in the manuscript supporting the essential expectation that the new forms of CCR5 would be HIV-protective. It was essential to have determined that before the embryos were implanted. They could have done so using a known assay: introduce the same edits into immune system cells in the laboratory and then infect them with HIV. Only the cells that have HIV-protective variants of CCR5 survive. The research team chose not to do that assay. Instead, they made children out of embryos that had forms of CCR5 of entirely uncertain functional impact. Were the researchers in a rush? Did they simply not care? Whatever the explanation, this egregious violation of elementary norms of ethics and of research borders on the criminal.

11. An American Nobelist may have helped He justify his experiment

The articles conclusion contains an unexpected digression that puts forth an entirely new justification for the research, one that connects the project to the heart of the HIV epidemic in Africa. Its that many uninfected children of African mothers with HIV suffer a syndrome called HEU that makes them more susceptible to a variety of childhood illnesses. The authors say genome editing could be a novel strategy against HEU.

There isnt any evidence for this idea, but there are some clues about where He got it. In an email he sent on November 22 to Craig Mello, a biologist at the University of Massachusetts who at the time was an advisor to one of his companies, He thanked Mello for suggestions on the topic and enclosed in his email the same paragraph above.

Does that mean Mello, a winner of the 2006 Nobel Prize for medicine, contributed a key idea to the paper? Mello was told about the twins project early on but, through a spokesman, says he never gave He advice on how to write the paper. According to Hes email, however, any such interaction was meant to remain unacknowledged. Again, I wont tell people you know what is happening here, he wrote to Mello.

12. The project had other supporters, but some key information is missing

The manuscript concludes by thanking a list of people who, according to He, gave him direct feedback on draft versions of the text or other advice. In an acknowledgement for editing the text, he names Mark Dewitt, a researcher at the University of California. Dewitt didnt reply to emails but earlier gave a description of his role, saying he had warned against the project. William Hurlbut, an ethicist at Stanford, says he gave ethics advice to He but didnt know that the Chinese scientist had created children.

He also thanks W.R. Twink Allen, an equine reproduction specialist in the United Kingdom, and Allens onetime student Jin Zhang, also known as John Zhang, who is now head of New Hope Fertility Center in New York, one of the largest in the US. According to reports, Zhang was planning with He late last year to open a medical tourism business for gene-edited babies.

Of these names, only Allens has not previously been cited in connection with the CRISPR-baby research. Allen did not reply to attempts to contact him by email. Zhang, who has not been forthcoming about his role, told us he was not familiar with the manuscript. I have never seen it, he told us in October.

The version of the twins manuscript we have is missing two critically important disclosures usually present in scientific papers. First, it gives no information about who funded the project or what financial interests the authors have in the outcome. Also missing is a section in which each authors scientific contribution is detailed. This means the text does not explicitly describe the role of the single non-Chinese author, Michael Deem of Rice University in Texas. The nature of Deems roleparticularly any hands-on involvement with the patientscould determine penalties that Deem, or his university, could face. Deems lawyers did not answer questions, including a request for copies of his past statements, which sought to minimize his role in the research. Rice says its investigation is ongoing.

13. The researchers ignored evidence that the gene edits werent uniform

In data attached to the paper, in the so-called supplementary material, are tables that He previously showed publicly. It shows chromatograms, or the readout of the DNA sequences found in the embryos and birth tissues of the twins (the umbilical cord and placenta) when his team tried to measure what editing had happened to the CCR5 gene.

Some observers, including Musunuru in our accompanying op-ed, say these data show clearly that the embryos are mosaic, meaning that different cells in the embryo were edited differently. He says presence of multiple edits is visible in the chromatograms, where several distinct readings are registered in overlapping signals at a given DNA position.

The implication of the data is that the twins bodies could be composites of cells edited in different ways, or not at all. That, Musunuru points out, means only some of their cells might have the HIV-resistant gene edit; it also means some might have undetected "off-target" edits, which could potentially cause health problems. The problem of mosaicism was well known to He from his experiments on animal embryos. One of the mysteries of the research project is why He chose to proceed with embryos if they were flawed in this way.

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In his manuscript, He doesnt resolve the mystery. It says only, The CCR5 gene was deep sequenced for all samples to examine the mosaicism of gene editing. Theres no interpretation of what was found, and no acknowledgement that the data seem to show mosaicism or that its a problem.

Fyodor Urnov: They should have worked and worked and worked until they reduced mosaicism to as close to zero as possible. This failed completely. They forged ahead anyway.

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Chinas CRISPR babies: Read exclusive excerpts from the unseen original research - MIT Technology Review

Opinion: We need to know what happened to CRISPR twins Lulu and Nana – MIT Technology Review

Its been a year since He Jiankui announced that hed made the worlds first gene-edited human babies, twin girls with the pseudonyms Lulu and Nana. Widespread condemnation of his actions followed the announcement. But the facts of the case remain unclear, because he has not been transparent about his work.

In his single public appearance following his announcement, at the Second International Summit on Human Genome Editing in Hong Kong in November 2018, He presented his work by racing through about 60 slides in just 20 minutes. Although he showed data about what he had done to the twins genes, it was blink-and-youll-miss-it, and not enough to convince anyone of his claim that hed safely edited the genomes of the human IVF embryos that became Lulu and Nana.

At the summit, He did say hed just submitted a manuscript describing this work to a scientific journal. Twelve months later, however, the manuscript has remained unpublished and its contents mysterious.

He was asked at the summit why he hadnt posted his manuscript to a preprint server such as bioRxiv or on a public websitesomething scientists frequently do to invite feedback on early drafts. He claimed that hed intended to do so, but colleagues had advised him to allow the manuscript to go through peer review by other scientists before posting it. (Normally, formal peer review takes place only when an academic journal is considering publishing a paper.)

By deciding not to release his manuscript right away, He has made it difficult for other scientists to figure out exactly what he did and how he did it. We already know that there were profound ethical problems with Hes work in germline gene editing, which refers to genetic alterations to embryosor to egg or sperm cellsthat can be passed down through the generations. But its scientific merit, and especially its safety, have remained in question.

When I first had the opportunity to look through a complete manuscript from He last November, I immediately realized there were problems.

The most serious was rampant mosaicism. This means that the gene edits He made to the embryos didnt take effect uniformly: different cells showed different changes. Evidence of mosaicism is present in both Lulus and Nanas embryos, as well as in Lulus placenta, making it likely the twins themselves are mosaic. Some parts of their bodies may contain the specific edits He said he made, other parts may contain other edits he didnt highlight, and yet other parts may contain no edits at all. This would mean that the purported benefit of Hes editing HIV resistancemay not extend to the twins entire bodies, and they could still be fully vulnerable to HIV.

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When judging whether the embryos had edits, He took a few cells from the 200 to 300 present in an IVF embryo and analyzed their DNA. But it was the remaining cells went on to multiply to make up the full body. So it is possible that some parts of the twins bodies have edits that He didnt intend to make (off-target edits) and never had a chance to see. Such off-target edits could cause problems such as cancer and heart disease, and could be passed on to Lulus and Nanas future children.

He apparently didnt realize that his own data revealed extensive mosaicism in the embryos, since he made no note of it in the manuscript I saw. Some have wondered if the CRISPR twins were actually a hoax, but to me, the flaws evident in the data make it clear that they werent. Rather, Hes work was a graphic demonstration of attempted gene editing gone awry. Two living human beings, and potentially their descendants too, will bear the consequences.

You shouldnt have to take my word for any of this. You should be able to judge for yourself, or at least hear what other scientists have to say about it.

However, it seems increasingly unlikely that He will be publishing in a peer-reviewed journal. For one thing, I doubt that any respectable journal would seriously consider publishing research with such ethical problems. And even if one did, and sent the manuscript for peer review, He would be in no position to respond to any technical criticisms with further experimental work. He has been under house arrest, and his laboratory was shut down shortly after his announcement about the twins last year.

The only reason to continue keeping Hes work under wraps would be to preserve his ability to publish it someday in a peer-reviewed journal and earn the imprimatur of scientific quality. The community is under no obligation to grant him this privilege. Indeed, it owes him no professional courtesy at all, any more than it would have owed such courtesy to the doctors responsible for the medical experiments in Nazi Germany or the American scientists in charge of the Tuskegee syphilis study.

Rather, in light of the egregious scientific and ethical lapses inherent in Hes cavalier and secretive efforts to make the worlds first gene-edited babies, it is he who owes all of us a full accounting of his actions. Since he has shirked his responsibility to make his work public, its up to others to step in.

Why must the information be public? Its because Hes work reveals serious, unresolved safety concerns. Its not clear that any effort to directly edit human embryos, even if done ethically and with full social approval, can reliably avoid these problems.

International committees convened by the World Health Organization, the US National Academies of Medicine and Sciences, and the Royal Society are currently working to propose regulatory frameworks for doing clinical germline gene editing safely, if it is to be done at all. How can the committees properly do their work without fully understanding all the scientific problems with the single real-world application of clinical germline gene editing thats been attempted to date?

Most worrying is that scientists like Denis Rebrikov in Russia aspire to follow in Hes footsteps. Rebrikov has said hell be able to edit the human germline safely. But how can Rebrikov credibly claim to be able to do better than He if the nature of the problems with Hes work arent widely known? How can the Russian authorities properly evaluate the safety of his proposals without being able to refer to Hes work for guidance?

Its time for the scientific community to fully understand what happened with Lulu and Nana, and to avoid stumbling down a path toward further ill-starred experiments with clinical germline gene editing.

Kiran Musunuru is an associate professor of cardiovascular medicine and genetics at the Perelman School of Medicine at the University of Pennsylvania and the author of The CRISPR Generation, a book about the history of gene editing and the Chinese twins.

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Opinion: We need to know what happened to CRISPR twins Lulu and Nana - MIT Technology Review

Cyrus, the Broad team up to make in vivo CRISPR use safer – FierceBiotech

Cyrus Biotechnology has teamed up with the Broad Institute to optimize CRISPR for use in humans. Feng Zhang, who had a hand in developing CRISPR, will serve as the Broads principal investigator for the collaboration.

One concern with using CRISPR-Cas9 to perform in vivo genome editing stems from the risk that the body will mount an immune response against the system. Those concerns have grown as researchers have shown that many people have antibodies against Cas9, reflecting the fact that the homologs of the protein used in genome editing systems are derived from bacteria that commonly infect people.

Cyrus, which lists Johnson & Johnson among its customers, thinks its technology can mitigate the risk of an immune reaction. That confidence reflects Cyrus experience of using software to identify and work around the epitopes in protein therapeutics that cause immunogenicity.

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

Partnering with the Broad will allow Cyrus to combine its experience of deimmunization with the skills of researchers who helped put CRISPR on the map. Zhang, the Broads lead on the project, was at the forefront of efforts to optimize Cas9 for use in human cells.

The partners plan to publish their research and make the fruits of their collaboration available to the nonprofit and academic research community for free.

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Cyrus, the Broad team up to make in vivo CRISPR use safer - FierceBiotech

2019: the year gene therapy came of age – FRANCE 24

Washington (AFP)

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

- Cures -

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

- Bioterrorism -

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

2019 AFP

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2019: the year gene therapy came of age - FRANCE 24

Here’s Why Editas Medicine Jumped 45.3% in November – The Motley Fool

What happened

Shares of Editas Medicine (NASDAQ:EDIT) rose more than 45% last month, according to data fromS&P Global Market Intelligence. The gene editing pioneer rose for reasons both internal and external.

The business announced an amended collaboration with Celgene (NASDAQ:CELG) for developing engineered immune cells and will receive an upfront payment of $70 million as a result of the new agreement. The company also enjoyed a bump from peer CRISPR Therapeutics, which reported promising results for the first two patients dosed with its lead drug candidate, CTX001. Investors took that as evidence that CRISPR-based medicines might be the real deal, although that's a mighty big leap.

The gene editing company also reported a business update and operating results for the third quarter of 2019, but there wasn't much to report for the pre-commercial entity.

Image source: Getty Images.

Editas Medicine started working with Juno Therapeutics, now owned by Celgene, in 2015. The idea was to combine the gene-editing platform of the former with the immunotherapy leadership of the latter. That's still the case, but the amended agreement scales back the specific types of engineered T cells that will be developed in the collaboration. It's a subtle, but potentially important, detail with (beneficial) ramifications for the long-term future of Editas Medicine.

It appears that the $70 million upfront payment was made in part to compensate Editas Medicine for the difference. After all, the company had already received $70 million in upfront, milestone, and execution payments under the original collaboration agreement. It's not immediately clear how the financial terms have changed, if they did at all, but the gene editing pioneer originally stood to receive up to $920 million in milestone payments.

Beyond that, there were several other updates provided in November:

The gene-editing landscape is still in the earliest stages of development. While CRISPR Therapeutics has taken an early lead as the top gene editing company, Editas Medicine is hoping to prove that its direct delivery approach will prove equally effective. The trial results the company will present in the coming years will become crucial tests for the future of CRISPR-gene editing, especially with competing techniques on the horizon.

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Here's Why Editas Medicine Jumped 45.3% in November - The Motley Fool

CRISPR-focused Excision BioTherapeutics Strengthens Board of Directors Appointing Veteran Pharmaceutical Executive Bill Carson, MD – GlobeNewswire

Oakland, CA, Dec. 03, 2019 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc., a biotechnology company developing CRISPR-based therapies to cure viral infectious diseases, today announced that veteran pharmaceutical executive William H. Carson, M.D. has joined the Board of Directors as an Independent Director.

Dr. Carson is the President & CEO of Otsuka Pharmaceutical Development & Commercialization, Inc. In this position, he led the companys development efforts in neuroscience, cardio-renal, and oncology, and was instrumental in the development and registration ofABILIFY MAINTENA(aripiprazole) as well as SAMSCA(tolvaptan). Dr. Carson joined Otsuka in 2002 as a board-certified psychiatrist and served as OPDCs Senior Vice President, Global Clinical Development, overseeing the development of all Otsuka-discovered compounds. During his career at Otsuka and earlier at Bristol-Myers Squibb (BMS), he was one of the key drivers in the development and commercialization ofABILIFY(aripiprazole). Dr. Carson received an A.B. degree in history and science from Harvard University and an M.D. degree from Case Western Reserve University. Dr. Carson plans to retire from Otsuka at the end of 2019.

Bill is an invaluable addition to Excisions Board of Directors, said Daniel Dornbusch, Excisions CEO. His extensive and highly regarded experience building successful companies as well as guiding products through early stage development, through clinical trials and to successful commercialization will accelerate Excisions activities throughout the organization. We are delighted that he will bring his insight and acumen to further Excisions growth.

I am honored to join Excisions Board of Directors at this key moment in the companys development, said Dr. Carson Their unique approach to developing cures for viral infectious diseases such as HIV, hepatitis B, JC virus, HSV and others has great potential to fulfill a key area of global health needs. Ive spent over 20 years helping companies grow successfully within the biopharmaceutical industry and look forward to leveraging my expertise to assist Excision during this transformative time.

About Excision BioTherapeutics

Excision BioTherapeutics, Inc., a biotechnology company developing CRISPR-based therapies to cure viral infectious diseases. Excision is focused on improving the lives of chronically ill patients by eliminating viral genomes from infected individuals. By using CRISPR in unique ways, the company has already demonstrated the first functional cure for HIV in animals. Excision is developing technologies and IP developed at Temple University and U.C. Berkeley. Excision is located in Oakland, California and is supported by Artis Ventures, Norwest Venture Partners, SilverRidge Venture Partners, Oakhouse Ventures, and Gaingels. For more information, please visitwww.excisionbio.com.

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CRISPR-focused Excision BioTherapeutics Strengthens Board of Directors Appointing Veteran Pharmaceutical Executive Bill Carson, MD - GlobeNewswire

CRISPR Technology Market – Industry Growth, Analysis, Business Trends, Competitive Landscape, Regional Forecast to 2030 – Media Releases – CSO…

Global CRISPR Technology Market is estimated to be over US$ 550.0 Million in 2018. It is anticipated to grow at a 24.0% CAGR from 2019 to 2030 and is expected to grow at a double digit CAGR during the forecasted period.The global CRISPR Technology market is segmented by product & services, application, end user, and region.

CRISPR Technology Market Overview and Introduction

GlobalCRISPR Technology Marketis estimated to be over US$ 550.0 Million in 2018. It is anticipated to grow at a 24.0% CAGR from 2019 to 2030and is expected to grow at a double digit CAGR during the forecasted period.The global CRISPR Technology market is segmented by product & services, application, end user, and region.

CRISPR Technology is relatively new technology used in genome editing or gene editing; CRISPR-CAS-9 is cluster of palindromic repeats and is found naturally in bacteria. These sequence enable the bacteria to protection them from virus by producing RNA segment or enzyme that cleaves the virus DNA and deactivates the virus. This ability of CRISPR-CAS9 has allowed scientist to make DNA and RNA libraries as per their need and applications. CRISPR-CAS9 technology have potential applications in the field of treating human diseases, creating gene libraries, and manipulating cell functions like metabolism.

The global CRISPR technology market is driven by growing focus of market players in CRISPR-CAS9 technology, availability of government and private funding and rising incidences related to genetic disorders are major factors driving the market. However, ethical issue, stringent regulatory policy and lack of skilled professionals are likely to restrain the market to certain extent.

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CRISPR Technology Market by Product & ServicesOn the basis of product, the CRISPR technology market is segmented into CRISPR Kits, Enzymes and Services. The CRISPR services are further sub-segmented into design & vector construction, cell line engineering, screening services and other services.CRISPR Technology Market by Application

Based on application, the market is segmented into biological & biomedical applications, agricultural applications, industrial applications and other applications.CRISPR Technology Market by End UserOn the basis of end user, the CRISPR technology market is segmented into academic institutes & research centers, contract research organizations (CROs), pharmaceutical and biotechnology companies and other end users.

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CRISPR Technology Market by RegionsThe global CRISPR technology industry can be segmented into North America, Europe, Asia Pacific, and Rest of World (ROW). North America dominated the market of CRISPR technology, followed by Europe and Asia Pacific. North America will continue to dominate the global CRISPR technology market in the forecast period owing to factors such as growing research in the field of CRISPR technology and adoption of CRISPR technology. Moreover However, Asia Pacific is expected to witness the highest CAGR, with the growth in this market centered at China, India, and Japan. Factors such as the government support are driving the growth of the CRISPR technology market in this region.

CRISPRTechnology Market Prominent Players

The prominent players in the global CRISPR Technology market are Thermo Fisher Scientific, Inc., GenScript, Merck KGaA, GeneCopoeia, Inc., Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Agilent Technologies, and Applied StemCell, Inc., among others.

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CRISPR Technology Market - Industry Growth, Analysis, Business Trends, Competitive Landscape, Regional Forecast to 2030 - Media Releases - CSO...

CRISPR and Cas Genes Market to Reach a Value of US$ 7234.5 Mn by the End of 2026 – News Description

Increase in applications of CRISPR and Cas gene editing technology in bacteria and usage of gene editing technology for prevention of various diseases are the major factors anticipated to drive the market from 2018 to 2026. Rise in need of alternative medicine for chronic diseases and increase in investments by key players in Asia Pacific are projected to propel the market during the forecast period.

The report also provides profiles of leading players operating in the global CRISPR and Cas market such as 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.

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Increase in Usage of DNA-free Cas

DNA-free Cas9 is most commonly used with synthetic crRNA tracrRNA and chosen by researchers who strive to avoid unwanted vector DNA integration into their genomic DNA. CRISPR-Cas9 utilizing mRNA or protein is ideal for applications such as knocking of a fluorescent reporter using HDR or knockout cell line generation. Advantages such as gene editing with DNA-free CRISPR-Cas9 components to reduce potential off-targets and potential usage of CRISPR-Cas9 gene editing to find correlations with human diseases in model systems drive the segment.

Rise in Incidence of Genetic Disorders and Increase in Applications of CRISPR and Cas Genes to Propel Market

Genetic diseases are generally termed as rare diseases. According to NCBI, prevalence of these rare diseases is approximately 5 in 10,000. There are 6,000 to 8,000 rare diseases, with 250 to 280 new diseases diagnosed every year. Hence, 6% to 8% of the global population is projected to be affected by rare diseases i.e., genetic diseases in the near future. Researchers are developing treatments for these diseases with applications of new technologies such as CRISPR. The applications of CRISPR technology are expanding in other industrial sectors. This is expected to drive the market during the forecast period.

Usage of CRISPR/Cas9 technology in plant research has enabled the investigation of plant biology in detail which has helped to create innovative applications in crop breeding. Site-directed mutagenesis and site-specific integration of a gene, which is also called knock-in, are important in precision crop breeding. Cas9/gRNA-mediated site-directed mutagenesis and knock-in is widely used in rice and Arabidopsis protoplasts. CRISPR/Cas9 provides a simple method to generate a DSB at a target site to trigger HDR repair.

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Asia Pacific Market to Witness Exponential Growth

In terms of revenue, the CRISPR and Cas genes market in Asia Pacific is expected to expand at a CAGR of 22.0% during the forecast period. Growth of the market in the region can be attributed to increase in incidence of chronic diseases such as cancer and the need of development of genetic engineered treatment options. According to the report, Call for Action: Expanding Cancer Care for Women in India, 2017, an estimated 0.7 million women in India are suffering from cancer. China dominated the CRISPR and Cas genes market in Asia Pacific. In 2016, scientists based in China launched the first known human trials of CRISPR, the genomic tech that involves slicing and dicing the bodys very source code to fight cancer. Japan was the second largest market for CRISPR and Cas genes in Asia Pacific.

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CRISPR and Cas Genes Market to Reach a Value of US$ 7234.5 Mn by the End of 2026 - News Description

Still Spinning In The Sand: CRISPR Therapeutics AG, (NASDAQ: CRSP) – Curious Coins

Intraday Trading:OnMonday, Shares ofCRISPR Therapeutics AGmakes a change of -4.23% and now trading at $68.62 The EPS of CRSP stock is strolling at -0.46, measuring its EPS increase this year at -101.60%.

EPS is the part of a companys interest allotted to each outstanding share of natural accretion. EPS works as a gauge of a companys profitability. EPS is usually thought to be the and no-one else various important variable in circumscribing a shares price.

Snapshot: CRISPR Therapeutics AG,belongs tothe Healthcaresector andBiotechnologyindustry.

As an end, the firm has an EPS growth of -1828.00% for the coming year. Companys EPS for the past five years is considered at 0.00%, directing it to an EPS value of 0.00% for the next five years. Given the significance of distinguishing organizations that will guarantee income per share at a high rate, we later fixation to umpire how to recognize which organizations will accomplish high hoarding rates. One evident flaunting to distinguish high profit per part tally together organizations are to find organizations that have shown such develop past the p.s. 5 to 10 years. We cant have enough support the once will consistently mirror the troublesome, however coherently stocks that have developed profit per remittance unequivocally in the consequent to are a fine wagered to keep on producing results, therefore.

The firm has a complete market capitalization of 3.92B and a total of Outstanding outstanding shares.

Trading volume recorded for this company was about 1567613 shares as contrast to its average volume of 821.51K shares.

Technical Analysis of CRISPR Therapeutics AG in the Limelight:

ATR stands at 4.38 whileBetafactor of the stock stands at 0.00. A beta element is used to measure the volatility of the stock. Beta is a measurement unit of the volatility, or managed chance, of a security or a portfolio in contrast with the market in general. Beta is utilized in the capital resource valuing model (CAPM), which calculates the expected return of an asset based on its beta and expects market returns. Beta is also known as the beta coefficient. The stock remained 6.22% volatile for the week and 7.14% for the month.

Performance Review:The stock has shown the weekly performance of 10.32%, and monthly performance stands at 36.23%. The year-to-date (YTD) performance reflected a 140.18%, during the past three months the stock performs 53.10%, bringing six-month performance to 84.17%.

Analysts meantarget pricefor the company is $77.50whileanalysts mean suggestionis 2.10. A final price is the projected price level of a financial security stated by an investment analyst or advisor. It symbolizes a securitys price that, if achieved, results in a trader recognizing the best possible outcome for his investment. This is the price at which the trader or investor wants to exit his current position so he can realize the most reward.

Investigating theproductivity proportionsof CRSP stock, the speculator will discover its ROE, ROA, ROI remaining at -2.60%, -2.00%, and -40.70%, individually.

CRSPinstitutional ownership is held at 46.80% while insider ownership was 0.40%. As of now,CRISPR Therapeutics AGhas a P/S, P/E and P/B values of 18.42, 0.00 and 6.36 respectively. Its P/Cash is valued at 6.22.

Relative Strength Index (RSI):Therelative strength indexof the stock stands 67.30. The relative quality file (RSI) is a specific pointer utilized in the examination of budgetary markets. It is proposed to outline the present and recorded quality or shortcoming of a stock or market dependent on the end costs of an ongoing exchanging period. The pointer ought not to be mistaken for relative quality.

The RSI is most generally utilized on a 14-day time allotment, estimated on a measuring scale from zero (0) to 100, with high and low levels set apart at 70 and 30, individually. Shorter or longer periods are utilized for, on the other hand, shorter or longer standpoints. Progressively extraordinary high and low levels80 and 20, or 90 and 10happen less often yet demonstrate more grounded energy.

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Still Spinning In The Sand: CRISPR Therapeutics AG, (NASDAQ: CRSP) - Curious Coins

Repare Therapeutics Appoints Steve Forte as CFO and Samarth Kulkarni to Its Board of Directors – Financial Post

CAMBRIDGE, Mass. & MONTREAL Repare Therapeutics, a precision oncology company pioneering synthetic lethality to develop novel therapeutics that target specific vulnerabilities of tumors in genetically defined patient populations, announced today that it has made two additions to its leadership team and Board of Directors. Steve Forte is appointed as Executive Vice President & Chief Financial Officer and leads the Companys financial, capital markets and administrative operations. Samarth Kulkarni, PhD, CEO of CRISPR Therapeutics (NASDAQ:CRSP), has been appointed to Repares Board of Directors as an independent director.

We are thrilled to make these key additions of Steve and Sam to our executive team and board leadership respectively, said Lloyd M. Segal, President & Chief Executive Officer of Repare Therapeutics. Steve Forte will be based in Repares Montreal, QC, offices. Katina Dorton, who had previously served as Repares CFO, has departed to pursue other interests, and will continue to serve as an advisor to the Company.

Steve Forte

Steve is a senior financial leader who has managed in complex, large-scale healthcare financial environments. Until its recent sale to Ipsen for US$1.3 billion, he was CFO of Clementia Pharmaceuticals (NASDAQ:CMTA), a leading biotechnology innovator in treatments for rare diseases. His experience includes nearly a decade at Aptalis Pharma Inc., where he was responsible for the overall corporate controllership function of a multinational pharmaceutical company with approximately $700 million in annual revenue in six global operating entities. Steve led SEC reporting including the preparation of an SEC S-1 registration statement for a U.S. IPO prior to the sale of the company to Forest Labs for $2.9 billion. Prior to Clementia, Steve was CFO of Thinking Capital Financial Corporation, a leading Canadian financial technology firm sold to Purpose Investments in 2018.

Steve received his Bachelor of Commerce in Accountancy from Concordia University and is a CPA/CMA.

Samarth Kulkarni

Sam has served as CEO of CRISPR Therapeutics since 2017. He has significant expertise in strategy and operations in biotechnology and a wide range of related cutting-edge therapeutic technologies. He joined CRISPR in 2015 in the early stages of the company as Chief Business Officer, and then served as President and Chief Business Officer. Prior to joining CRISPR, Sam was a Partner at McKinsey & Company, where he had a leading role in the Pharmaceutical and Medical products practice. While at McKinsey, he co-led the biotech practice, where he focused on topics ranging from strategy to operations and led initiatives in areas such as personalized medicine and immunotherapy. Sam also serves as the Chairman of the Board of Directors of Casebia Therapeutics, a joint subsidiary formed by CRISPR Therapeutics and Bayer.

He received a Ph.D. in Bioengineering and Nanotechnology from the University of Washington and a B. Tech. from the Indian Institute of Technology. While at the University of Washington, he conducted research in the delivery of biological drugs and in the field of molecular diagnostics. He has authored several publications in leading scientific and business journals.

About Repare Therapeutics

Repare Therapeutics is pioneering synthetic lethality to develop novel therapeutics that target specific vulnerabilities of tumors in genetically defined patient populations. The companys initial focus is on novel targeted therapies in cancer types harboring defective DNA-damage response (DDR)- or genome instability-related functions. Repares SNIPRx platform combines a proprietary, high throughput, CRISPRenabled gene editing target discovery technology with highresolution protein crystallography, computational biology, medicinal chemistry and clinical informatics to rapidly generate small molecules for clinical investigation. The company is backed by leading global healthcare investors including Versant Ventures and MPM Capital. For additional information, please visit http://www.reparerx.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191203005090/en/

Contacts

Steve Forte Chief Financial Officer Repare Therapeutics Inc. info@reparerx.com

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Repare Therapeutics Appoints Steve Forte as CFO and Samarth Kulkarni to Its Board of Directors - Financial Post

Current Trend:: CRISPR Therapeutics AG, (NASDAQ: CRSP) – Ws News Alerts

In recent times, Today on Tuesday, December 03, 2019, by making a change of 1.18% with the Gain (), the Healthcare stock (CRISPR Therapeutics AG) created a change of 2.27% from opening and finally closed its business at 69.43.

Earnings for each Share (EPS) are the part of a companys profit allocated to respectively outstanding share of common stock. EPS serves as a pointer to a companys profitability/success. EPS is considered to be the only most crucial variable in determining a shares price.

Eye Catching Stocks: CRISPR Therapeutics AG

Intraday Trading of the CRISPR Therapeutics AG:CRISPR Therapeutics AG, a Switzerland based Company, belongs to Healthcare sector and Biotechnology industry.

Trading volume, or volume, is the number of shares or contracts that point towards the full activity of a security or stock market for a given period. The company exchanged hands with 273826 shares contrast to its average daily volume of 843.13K shares. Relative Volume (or RVOL) is a volume indicator, meaning it assists measure shareholder interest in a stock. RVOL compares a stocks current volume to its previous amount over a specific period.

Performance Review:

Technical Analysis of CRISPR Therapeutics AG: Looking into the profitability ratios of CRSP stock, the shareholder will find its ROE, ROA and ROI standing at -2.6%, -2% and -40.7%, respectively. A profitability ratio is an estimate of profitability, which is a way to measure a companys performance. Profitability merely is the capacity to make a profit, and a gain is what is left over from income earned after you have deducted all costs and expenses related to obtaining the income.

The RSI most typically used on a 14-day timeframe, measured on a scale from 0-100, with high and low levels marked at between 70 and 30, respectively. Shorter or longer timeframes used for alternately shorter or longer outlooks. More supreme high and low levels80 and 20, or 90 and 10occur less frequently but indicate stronger momentum. The RSI provides signals that tell investors to buy when the currency oversold and to sell when it is overbought. The present relative strength index (RSI) reading is 67.93.

What do you mean by simple moving average (SMA)?

A simple moving average (SMA) is an arithmetic moving average calculated by adding the closing price of the security for some time periods and then dividing this total by the number of time periods. Its distance from 20-days simple moving average is 18.26%, and its distance from 50 days simple moving average is 43.63% while it has a distance of 57.9% from the 200 days simple moving average. The companys distance from 52-week high price is -6.18% and while the current price is 212.46% from 52-week low price.

As of now, CRISPR Therapeutics AG has a P/S, P/E and P/B values of 18.42, 0 and 6.36 respectively.

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Current Trend:: CRISPR Therapeutics AG, (NASDAQ: CRSP) - Ws News Alerts

Genome Editing Services, World Markets to 2030: Focus on CRISPR – The Most Popular Genome Manipulation Technology Tool – PRNewswire

DUBLIN, Nov. 28, 2019 /PRNewswire/ -- The "Genome Editing Services Market-Focus on CRISPR 2019-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape of CRISPR-based genome editing service providers. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographical regions.

Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The biopharmaceutical pipeline includes close to 500 gene therapies, several of which are being developed based on the CRISPR technology.

Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. However, successful gene manipulation efforts involve complex experimental protocols and advanced molecular biology centered infrastructure. Therefore, many biopharmaceutical researchers and developers have demonstrated a preference to outsource such operations to capable contract service providers.

Consequently, the genome editing contract services market was established and has grown to become an indispensable segment of the modern healthcare industry, offering a range of services, such as gRNA design and construction, cell line development (involving gene knockout, gene knockin, tagging and others) and transgenic animal model generation (such as knockout mice). Additionally, there are several players focused on developing advanced technology platforms that are intended to improve/augment existing gene editing tools, especially the CRISPR-based genome editing processes.

Given the rising interest in personalized medicine, a number of strategic investors are presently willing to back genetic engineering focused initiatives. Prevalent trends indicate that the market for CRISPR-based genome editing services is likely to grow at a significant pace in the foreseen future.

Report Scope

One of the key objectives of the report was to evaluate the current opportunity and the future potential of CRISPR-based genome editing services market. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2019-2030.

In addition, we have segmented the future opportunity across [A] type of services offered (gRNA construction, cell line engineering and animal model generation), [B] type of cell line used (mammalian, microbial, insect and others) and [C] different geographical regions (North America, Europe, Asia Pacific and rest of the world).

To account for the uncertainties associated with the CRISPR-based genome editing services market and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market's evolution.

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. Overview of Genome Editing3.3. History of Genome Editing3.4. Applications of Genome Editing3.5. Genome Editing Techniques3.5.1. Mutagenesis3.5.2 Conventional Homologous Recombination3.5.3 Single Stranded Oligo DNA Nucleotides Homologous Recombination3.5.4. Homing Endonuclease Systems (Adeno Associated Virus System)3.5.5. Protein-based Nuclease Systems3.5.5.1. Meganucleases3.5.5.2. Zinc Finger Nucleases3.5.5.3. Transcription Activator-like Effector Nucleases3.5.6. DNA Guided Systems3.5.6.1. Peptide Nucleic Acids3.5.6.2. Triplex Forming Oligonucleotides3.5.6.3. Structure Guided Endonucleases3.5.7. RNA Guided Systems3.5.7.1. CRISPR-Cas93.5.7.2. Targetrons3.6. CRISPR-based Genome Editing3.6.1. Role of CRISPR-Cas in Adaptive Immunity in Bacteria3.6.2. Key CRISPR-Cas Systems3.6.3. Components of CRISPR-Cas System3.6.4. Protocol for CRISPR-based Genome Editing3.7. Applications of CRISPR3.7.1. Development of Therapeutic Interventions3.7.2. Augmentation of Artificial Fertilization Techniques3.7.3. Development of Genetically Modified Organisms3.7.4. Production of Biofuels3.7.5. Other Bioengineering Applications3.8. Key Challenges and Future Perspectives

4. CRISPR-BASED GENOME EDITING SERVICE PROVIDERS: CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. CRISPR-based Genome Editing Service Providers: Overall Market Landscape4.2.3. Analysis by Type of Service Offering4.2.4. Analysis by Type of gRNA Format4.2.5. Analysis by Type of Endonuclease4.2.6. Analysis by Type of Cas9 Format4.2.7. Analysis by Type of Cell Line Engineering Offering4.2.8. Analysis by Type of Animal Model Generation Offering4.2.9. Analysis by Availability of CRISPR Libraries4.2.10. Analysis by Year of Establishment4.2.11. Analysis by Company Size4.2.12. Analysis by Geographical Location4.2.13. Logo Landscape: Distribution by Company Size and Location of Headquarters

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. CRISPR-based Genome Editing Service Providers: Competitive Landscape5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. COMPANY PROFILES6.1. Chapter Overview6.2. Applied StemCell6.2.1. Company Overview6.2.2. Service Portfolio6.2.3. Recent Developments and Future Outlook6.3. BioCat6.4. Biotools6.5. Charles River Laboratories6.6. Cobo Scientific6.7. Creative Biogene6.8. Cyagen Biosciences6.9. GeneCopoeia6.10. Horizon Discovery6.11. NemaMetrix6.12. Synbio Technologies6.13. Thermo Fisher Scientific

7. PATENT ANALYSIS7.1. Chapter Overview7.2. Scope and Methodology7.3. CRISPR-based Genome Editing: Patent Analysis7.3.1. Analysis by Application Year and Publication Year7.3.2. Analysis by Geography7.3.3. Analysis by CPC Symbols7.3.4. Emerging Focus Areas7.3.5. Leading Players: Analysis by Number of Patents7.4. CRISPR-based Genome Editing: Patent Benchmarking Analysis7.4.1. Analysis by Patent Characteristics7.5. Patent Valuation Analysis

8. ACADEMIC GRANT ANALYSIS8.1. Chapter Overview8.2. Scope and Methodology8.3. Grants Awarded by the National Institutes of Health for CRISPR-based8.3.1. Year-wise Trend of Grant Award8.3.2. Analysis by Amount Awarded8.3.3. Analysis by Administering Institutes8.3.4. Analysis by Support Period8.3.5. Analysis by Funding Mechanism8.3.6. Analysis by Type of Grant Application8.3.7. Analysis by Grant Activity8.3.8. Analysis by Recipient Organization8.3.9. Regional Distribution of Grant Recipient Organization8.3.10. Prominent Project Leaders: Analysis by Number of Grants8.3.11. Emerging Focus Areas8.3.12. Grant Attractiveness Analysis

9. CASE STUDY: ADVANCED CRISPR-BASED TECHNOLOGIES/SYSTEMS AND TOOLS9.1. Chapter Overview9.2. CRISPR-based Technology Providers9.2.1. Analysis by Year of Establishment and Company Size9.2.2. Analysis by Geographical Location and Company Expertise9.2.3. Analysis by Focus Area9.2.4. Key Technology Providers: Company Snapshots9.2.4.1. APSIS Therapeutics9.2.4.2. Beam Therapeutics9.2.4.3. CRISPR Therapeutics9.2.4.4. Editas Medicine9.2.4.5. Intellia Therapeutics9.2.4.6. Jenthera Therapeutics9.2.4.7. KSQ Therapeutics9.2.4.8. Locus Biosciences9.2.4.9. Refuge Biotechnologies9.2.4.10. Repare Therapeutics9.2.4.11. SNIPR BIOME9.2.5. Key Technology Providers: Summary of Venture Capital Investments9.3. List of CRISPR Kit Providers9.4. List of CRISPR Design Tool Providers

10. POTENTIAL STRATEGIC PARTNERS10.1. Chapter Overview10.2. Scope and Methodology10.3. Potential Strategic Partners for Genome Editing Service Providers10.3.1. Key Industry Partners10.3.1.1. Most Likely Partners10.3.1.2. Likely Partners10.3.1.3. Less Likely Partners10.3.2. Key Non-Industry/Academic Partners10.3.2.1. Most Likely Partners10.3.2.2. Likely Partners10.3.2.3. Less Likely Partners

11. MARKET FORECAST11.1. Chapter Overview11.2. Forecast Methodology and Key Assumptions11.3. Overall CRISPR-based Genome Editing Services Market, 2019-203011.4. CRISPR-based Genome Editing Services Market: Distribution by Regions, 2019-203011.4.1. CRISPR-based Genome Editing Services Market in North America, 2019-203011.4.2. CRISPR-based Genome Editing Services Market in Europe, 2019-203011.4.3. CRISPR-based Genome Editing Services Market in Asia Pacific, 2019-203011.4.4. CRISPR-based Genome Editing Services Market in Rest of the World, 2019-203011.5. CRISPR-based Genome Editing Services Market: Distribution by Type of Services, 2019-203011.5.1. CRISPR-based Genome Editing Services Market for gRNA Construction, 2019-203011.5.2. CRISPR-based Genome Editing Services Market for Cell Line Engineering, 2019-203011.5.3. CRISPR-based Genome Editing Services Market for Animal Model Generation, 2019-203011.6. CRISPR-based Genome Editing Services Market: Distribution by Type of Cell Line, 2019-203011.6.1. CRISPR-based Genome Editing Services Market for Mammalian Cell Lines, 2019-203011.6.2. CRISPR-based Genome Editing Services Market for Microbial Cell Lines, 2019-203011.6.3. CRISPR-based Genome Editing Services Market for Other Cell Lines, 2019-2030

12. SWOT ANALYSIS12.1. Chapter Overview12.2. SWOT Analysis12.2.1. Strengths12.2.2. Weaknesses12.2.3. Opportunities12.2.4. Threats12.2.5. Concluding Remarks

13. EXECUTIVE INSIGHTS

14. APPENDIX 1: TABULATED DATA

15. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

Companies Mentioned

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Genome Editing Services, World Markets to 2030: Focus on CRISPR - The Most Popular Genome Manipulation Technology Tool - PRNewswire

Cyrus Biotechnology and CRISPR pioneers team up to boost gene-editing therapies – GeekWire

MIT researcher Feng Zhang will be the principal investigator for the Broad Institutes collaboration with Cyrus Biotechnology. (HHMI Photo)

Seattle-based Cyrus Biotechnology says itll collaborate with the Broad Institute of MIT and Harvard on ways to optimize CRISPR gene-editing techniques for use in developing novel human therapeutics.

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Cyrus Biotechnology and CRISPR pioneers team up to boost gene-editing therapies - GeekWire

Human Nature film review: Telling the CRISPR story with wit and verve – New Scientist

By Simon Ings

David Sanchez eyes a CRISP future for his sickle-cell anaemia

Human Nature

Directed by Adam Bolt

UK cinemas, 6 December

Advertisement

MATURE and intelligent, Human Nature shows us how gene editing works, explores its implications and in a field awash with alarmist rhetoric and cheap dystopianism explains which concerns are worth losing sleep over.

This gripping documentary covers a lot of ground, but also works as a primer on CRISPR, the spectacular technology that enables us to cut and paste genetic information with something like the ease with which we manipulate

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Human Nature film review: Telling the CRISPR story with wit and verve - New Scientist

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

Dec. 2, 2019 10:30 UTC

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

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

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

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

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

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

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

About Cyrus Biotechnology

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

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

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

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

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

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

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

By Crop Type:Staple CropsFruits & VegetablesOrnamentalsOthers

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

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

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

Some of the key manufacturers involved in the market are:

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

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

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

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

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

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

Image source: Getty Images.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Japan and Singapore Grant CRISPR Patents to MilliporeSigma – PRNewswire

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

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

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

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

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

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

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

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

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

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

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Japan and Singapore Grant CRISPR Patents to MilliporeSigma - PRNewswire

CRISPR Therapeutics to Present at Upcoming Investor Conferences – StreetInsider.com

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

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

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

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

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

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

Global CRISPR and Cas Genes Market: Segmentation Analysis

Segmentation on the basis of product:

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

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

Biotechnology & Pharmaceutical CompaniesAcademic & Government Research InstitutesContract Research Organizations

Global CRISPR and Cas Genes Market: Regional Analysis

North America

Europe

Asia Pacific

Latin America

Middle East & Africa

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CRISPR and Cas Genes Market Research report will be favorable for:

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Propose investors and Private Equity Enterprises

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End-Use Industries

And Much More

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

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

What will the CRISPR and Cas Genes market size and the growth rate be in 2028?

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

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

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

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

What are the CRISPR and Cas Genes market opportunities and threats faced by the vendors in the global Industry?

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

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

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

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

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

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

Request a sample copy @

https://www.marketresearchnest.com/report/requestsample/770598

The key insights of the report:

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

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

At least 9 companies are included:

For complete companies list, please ask for sample pages.

The information for each competitor includes:

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

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

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

Rendered Cas9

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

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

Vertex Pharmaceuticals CEO Jeffery Leiden

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

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

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

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

CRISPR Therapeutics CEO Sam Kulkarni

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

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

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

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

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

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

BRIEF-CRISPR Therapeutics And Vertex Announce Data From Two Patients Treated With Gene-Editing Therapy CTX001 – Reuters

Nov 19 (Reuters) - CRISPR Therapeutics AG:

* CRISPR THERAPEUTICS AND VERTEX ANNOUNCE POSITIVE SAFETY AND EFFICACY DATA FROM FIRST TWO PATIENTS TREATED WITH INVESTIGATIONAL CRISPR/CAS9 GENE-EDITING THERAPY CTX001 FOR SEVERE HEMOGLOBINOPATHIES

* CRISPR THERAPEUTICS - 2 SERIOUS ADVERSE EVENTS OCCURRED, NEITHER OF WHICH THE PRINCIPAL INVESTIGATOR CONSIDERED RELATED TO CTX001 Source text for Eikon: Further company coverage:

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BRIEF-CRISPR Therapeutics And Vertex Announce Data From Two Patients Treated With Gene-Editing Therapy CTX001 - Reuters

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