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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

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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

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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.

How ICON, Lotus, and Bioforum are Improving Study Efficiency with a Modern EDC

CROs are often at the forefront of adopting new technologies to make clinical trials more efficient. Hear how ICON, Lotus Clinical Research, and Bioforum are speeding database builds and automating reporting tasks for data management.

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

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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

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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

Recommendation and review posted by Bethany Smith

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

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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

Recommendation and review posted by Bethany Smith

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...

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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

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

Recommendation and review posted by Bethany Smith

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

Recommendation and review posted by Bethany Smith

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

Recommendation and review posted by Bethany Smith

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

Recommendation and review posted by Bethany Smith

A Once-a-Month Birth Control Pill Is ComingHere’s How It Works – WIRED

In the US, more than 15 million Americans will spend a few seconds every day locating and swallowing a birth control pill. If the tableta cordial of contraceptive hormones that trick the body into thinking its pregnantis swallowed during the same three-hour window each day, the method is pretty close to ironclad. But get off-schedule or miss a day, and the pills 99 percent pregnancy prevention rate starts to dip. And most people inevitably falter. In the US, nine out of every 100 people using oral birth control become pregnant in any given year.

IUDs and other forms of contraception are more reliable. But so many people still use the pill because its cheap, easy, and often available over the counter, rather than requiring a trip to a doctor to have some hormone-emitting device implanted under your skin or inserted into your uterus. For a long time, researchers have been trying to merge the benefits of botha longer-lasting supply of hormones that are as simple to take as swallowing. But the stomach has proven a worthy nemesis.

A dark, slimy accordion of an organ, it continuously contracts and grinds a corrosive slurry of gastric juices, sending waves of whatever youve swallowed crashing over a tiny fissure called the pylorus that leads into the deeper recesses of the gut. If youre on any sort of oral medicationfor high cholesterol or HIV or to maintain your reproductive independencethis angry acid sea is the reason you have to take a pill every single day. Drugs just dont last long in such a hostile environment.

Unless, that is, you embed them in a flexible silicon ninja star that folds up neatly into pill form.

Thats the solution a team led by scientists at Brigham and Womens Hospital and MIT came up with about five years ago. Back then they were building slow-release pills designed to deliver treatments for malaria, tuberculosis, and HIV. But in a scientific first, theyve now demonstrated that the same invention can also deliver a steady drip of contraceptive hormones in the body of a pig for up to 29 days.

From an engineering aspect, the key novelty is the ability to deliver a drug for a month after a single ingestion event, says Giovanni Traverso, a gastroenterologist and biomedical engineer at Brigham and Womens and MIT, who co-authored the new study, published today in Science Translational Medicine. The proof-of-concept experiments were conducted late last year. Since then, the long-lasting contraceptive has begun to be commercially developed by a Boston-area company called Lyndra Therapeutics, which Traverso cofounded with MIT bioengineer Robert Langer in 2015. In July, the startup received $13 million from the Gates Foundation to advance the monthly pill to human trials, with a focus on bringing it to low- and middle-income countries.

To picture how this works, Traverso suggests imagining a six-armed starfish that has folded itself up into a cylinder. Except each arm is made of a body-friendly silicone polymer bonded with levonorgestrelthe hormone used in IUDs like Mirena. The arms are cut with little repeating windows that allow the drug to gradually detach itself from the surrounding polymer matrix. They connect to each other via an elastic core, and when the whole thing is folded up, it fits inside a standard-size capsule. Once swallowed, the capsule dissolves, allowing the arms to spring back into rigid starfish shape.

This is key, because once popped back open, those arms span wider than two centimetersthe diameter of the human pylorus, the gateway to the intestines. Trapped inside the stomach, the silicone starfish slowly seeps the medication through its small pores, allowing the hormone to pass through the gut and into the bloodstream to do its work.

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A Once-a-Month Birth Control Pill Is ComingHere's How It Works - WIRED

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The First ‘Bespoke’ TRT Clinic in the UK – News Anyway

The Mens Health Clinic, founded by Dr Robert Stevens MBChB MRCGP Dip.FIPT, is the first clinic of its kind in the UK. From very humble beginnings they now have their very own private medical clinic that attracts people from all over the world. The clinic specialises in the diagnosis and treatment of Testosterone Deficiency. Their clinic offers a uniquely progressive approach to patient care, understanding the need for a personalised approach, not a one-size-fits all model.

Testosterone Deficiency is a medical condition characterised by the negative symptoms associated with low testosterone, along with confirmatory blood test results. The British Society for Sexual Medicine has set out national guidance to support doctors in making the diagnosis. Prominent symptoms of Testosterone Deficiency include an element of low mood, anxiety, and-or depersonalisation. Men often describe a brain fog, fatigue and low libido. Other symptoms can include erectile dysfunction, weight gain, loss of lean muscle mass and a decrease in bone mineral density. As there can be several possible diagnoses for each symptom, it is important to have a comprehensive assessment by a clinician who specialises in the area, before commencing any treatment. There can also be potential reversible causes for low testosterone and so these should always be addressed before committing to, what should be considered, a lifelong therapy.

From the outside, TRT can appear straightforward; if you have a deficiency you restore the testosterone level using exogenous testosterone. Unfortunately, this is a simplistic perspective. Exogenous testosterone suppresses the release of Lutenising Hormone (LH) and Follicle Stimulating Hormone (FSH) from the pituitary gland. As there are LH receptors in numerous organs, most notably the testes and the brain, traditional testosterone monotherapy often causes azoospermia and male infertility due to the suppression of LH. TRT should therefore be addressed as hormone replacement therapy. The aim of TRT and hormone replacement therapy should be to maintain function, in addition to restoring male androgen levels using exogenous testosterone.

Human Chorionic Gonadotropin (HCG) has been used by the NHS to treat male infertility for many years. HCG mimics LH and so The Mens Health Clinic utilises HCG alongside testosterone in testosterone therapy to help preserve fertility and testicular size. Dr Stevens reported nineteen pregnancies in his patients so far with the use of concurrent TRT and HCG. In addition to the fertility aspect, men often report a qualitative improvement in their sense of well-being and libido with the use of HCG alongside their testosterone treatment.

In 2019, The Mens Health Clinic was delighted to be the first clinic in the UK to be granted a licence by the MHRA for the import of Testosterone Cypionate, their gold standard TRT option. Dr Stevens is a proponent of daily injections of Testosterone Cypionate to help mimic the diurnal variation of endogenous testosterone that occurs naturally within the body. He also reports more stable male androgen levels using this methodology. Dr Stevens is also a proponent of subcutaneous injections, over shallow intramuscular ones, as they are less painful and result in less aromatisation of testosterone to oestrogen. Less side effects mean happier, more content patients.

Hormonal balance is fundamental to overall health and well-being. You need a clinician who understands that all hormones are dependent, whether that be direct or indirect. The ratio of testosterone : oestradiol : dihydrotestosterone, is as important as the absolute numbers. The purpose of TRT is normalisation of your male androgen levels to within normal physiological parameters. We aim to reverse the negative symptoms of low testosterone and help you maintain a sustainable improvement in your long-term physical and psychological well-being.

CONTACT:

The Mens Health Clinic9-10 Longham Business Park168 Ringwood RoadFERNDOWNDorsetBH22 9BUTel: 01202 091593Email: info@themenshealthclinic.co.uk

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The First 'Bespoke' TRT Clinic in the UK - News Anyway

Recommendation and review posted by Bethany Smith

Molecular phenotyping and image-guided surgical treatment of melanoma using spectrally distinct ultrasmall core-shell silica nanoparticles – Science…

Supplementary material for this article is available at http://advances.sciencemag.org/cgi/content/full/5/12/eaax5208/DC1

Fig. S1. Clinical trial SLN mapping case in a patient with head and neck melanoma using cRGDY-Cy5.5-PEG-C dots (NCT02106598).

Fig. S2. Development of 700- and 800-nm emitting NIR fluorescent C dots.

Fig. S3. Morphology and purity characterization of MSH-PEG-Cy5.5-C dots and cRGDY-PEG-CW800-C dots.

Fig. S4. The relationship between probe concentration and signal intensity of different probes.

Fig. S5. The bleed-through of MSH-PEG-Cy5.5-C dot probes to the 800-nm channel under the excitation of a 700-nm laser (particle concentration range, 0 to 125 nM).

Fig. S6. The bleed-through of cRGDY-PEG-CW800-C dot probes to the 700-nm channel under the excitation of a 800-nm laser (concentration range, 0 to 250 nM).

Fig. S7. The bleed-through of cocktail C dots (MSH-PEG-Cy5.5-C dot probes and cRGDY-PEG-CW800-C dot) probes to the 700- or 800-nm channels under the combined excitation of 700- and 800-nm lasers (concentration range, 0 to 250 nM).

Fig. S8. Summary of MIP PET images of all seven spontaneous melanoma miniswines (the related MIP short videos are also provided as movies S1 to S7).

Fig. S9. The marking of the expected SLN locations.

Fig. S10. A representative example of multiplexing optical data analysis using the software (Architector Image Viewer, version 1.9.0) from Quest Spectrum.

Fig. S11. Whole-body PET/CT imaging of pig #1.

Fig. S12. Whole-body PET/CT imaging and histological analysis of pig #2.

Fig. S13. Whole-body PET/CT imaging and histological analysis of pig #3.

Fig. S14. Whole-body PET/CT imaging and histological analysis of pig #4.

Fig. S15. Whole-body PET/CT imaging and histological analysis of pig #6.

Fig. S16. Whole-body PET/CT imaging and histological analysis of pig #7.

Fig. S17. IVIS imaging to identify nanoparticles at the tumor site.

Fig. S18. Average body weight of all male mice from the microdose toxicology study.

Fig. S19. Average body weight of all female mice from the microdose toxicology study.

Table S1. Optimization of 800-nm emitted NIR fluorescent C dots.

Table S2. In vivo signal intensity and bleed-through percentage of cocktail C dots excited with a 700-nm laser.

Table S3. Summary of the injection information of all seven spontaneous melanoma miniswines.

Table S4. Summary of PET SUV numbers of all nodes from seven spontaneous melanoma miniswines (~0.5-mm tumor burden was highlighted in green; NA means tumor burden diameter was not available).

Table S5. Microdose toxicology study animal group.

Table S6. Mortality and morbidity summary table of the microdose toxicology study.

Table S7. Hematology parameters in the microdose toxicology study.

Table S8. Clinical chemistry parameters in the microdose toxicology study.

Table S9. Tissues examined microscopically in the microdose toxicology study.

Table S10. Hematology parameters, group 5, male, tumor, vehicle control (saline), day 2.

Table S11. Hematology parameters, group 6, female, tumor, vehicle control (saline), day 2.

Table S12. Hematology parameters, group 7, male, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S13. Hematology parameters, group 8, female, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S14. Hematology parameters, group 9, male, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S15. Hematology parameters, group 10, female, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S16. Hematology parameters, group 11, male, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S17. Hematology parameters, group 12, female, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S18. Hematology parameters, group 13, male, no tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S19. Hematology parameters, group 14, female, no tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S20. Hematology parameters, group 15, male, tumor, vehicle control (saline), day 14.

Table S21. Hematology parameters, group 16, female, tumor, vehicle control (saline), day 14.

Table S22. Hematology parameters, group 17, male, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S23. Hematology parameters, group 18, female, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S24. Hematology parameters, group 19, male, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 14.

Table S25. Hematology parameters, group 20, female, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 14.

Table S26. Hematology parameters, group 21, male, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S27. Hematology parameters, group 22, female, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S28. Hematology parameters, group 23, male, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S29. Hematology parameters, group 24, female, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S30. Hematology parameters, group 5, male, tumor, vehicle control (saline), day 2.

Table S31. Hematology parameters, group 6, female, tumor, vehicle control (saline), day 2.

Table S32. Hematology parameters, group 7, male, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S33. Hematology parameters, group 8, female, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S34. Hematology parameters, group 9, male, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S35. Hematology parameters, group 10, female, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S36. Hematology parameters, group 11, male, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S37. Hematology parameters, group 12, female, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 2.

Table S38. Hematology parameters, group 13, male, no tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S39. Hematology parameters, group 14, female, no tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 2.

Table S40. Hematology parameters, group 15, male, tumor, vehicle control (saline), day 14.

Table S41. Hematology parameters, group 16, female, tumor, vehicle control (saline), day 14.

Table S42. Hematology parameters, group 17, male, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S43. Hematology parameters, group 18, female, tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S44. Hematology parameters, group 19, male, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 14.

Table S45. Hematology parameters, group 20, female, tumor, test article cRGDY-PEG-cw800-C dots at 1.2 nmol, day 14.

Table S46. Hematology parameters, group 21, male, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S47. Hematology parameters, group 22, female, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S48. Hematology parameters, group 23, male, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Table S49. Hematology parameters, group 24, female, no tumor, test article MSH-PEG-Cy5.5-C dots at 1.2 nmol, day 14.

Movie S1. MIP video of pig #1.

Movie S2. MIP video of pig #2.

Movie S3. MIP video of pig #3.

Movie S4. MIP video of pig #4.

Movie S5. MIP video of pig #5.

Movie S6. MIP video of pig #6.

Movie S7. MIP video of pig #7.

Movie S8. Image-guided multiplexing, pig #5.

Movie S9. Image-guided multiplexing, pig #3.

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Molecular phenotyping and image-guided surgical treatment of melanoma using spectrally distinct ultrasmall core-shell silica nanoparticles - Science...

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A matter of life and death: insulin emergency/affordability bill… – MSR News Online

Headline after headline has been writtenabout legislators efforts to help Minnesotans suffering because they cannotafford their insulin medication. Yet no tangible compromise has been reached ona medical issue that affects so many Minnesotans.

Many suffering from diabetes simply cannotwait for a more affordable option. Some people have literally died whilewaiting for economic relief that would allow them to purchase a drug that costsas much as $300 for a 10-day supply.

The 30-day deadline has gone and passed,an exasperated Nicole Smith-Holt said. At this point, it feels like itis all PR just making it look like they care. They need to do this to savelives, she stressed.

State Representative Micheal Howardagreed. He has been working to get a law passed that would make insulinavailable to those who either have an emergency or just cannot afford theinsulin they need to survive. We need to show more urgency; there really is noreason why we shouldnt be able to come together and get something done, hesaid.

This is a bill I have been working on formore than a year. Its been like working at the kitchen table trying to figureout how do we create a safety net so that nobody loses their life because theycannot afford their drugs? explained Howard. I understand the frustration foradvocates who have been pushing for this for more than a year. The crisis isright in front of us; the solution is right in front of us. We need all sidesto show that sense of urgency, the legislator said, alluding to what some havecalled foot-dragging and grandstanding on the part of Republicans.

Everybody knows a family member that isaffected, he added.

Medical experts have described diabetes asa disease that occurs when a persons blood glucose, also referred to as bloodsugar, is too high. And having too much glucose in ones blood can cause healthproblems.

Blood glucose is our main source of energyand comes from the food we eat. Insulin, a hormone made by the pancreas, helpsglucose from food get into your cells to be used for energy. Sometimes our bodydoesnt make enoughor anyinsulin or doesnt use insulin well. Glucose then staysin our blood and doesnt reach our cells.

Diabetes leads to heart disease, visionloss, kidney disease, a stroke or even amputation.

According to the American DiabetesAssociation, diabetes is a major health problem in the U.S. The fallout fromthe disease puts a tremendous strain on the health care industry. Studies haveshown that nearly one-third of the total U.S. population is affected by someform of diabetes, either Type 1, Type 2 or prediabetic.

And the risk of diabetes is higher inAfrican American adults. Latest studies showed that about 4.9 million or 18.7%of all African Americans 20 years of age and older have diagnosed orundiagnosed diabetes, compared to 7.1% of non-Hispanic White Americans. Therisk of diabetes is 77% higher among African Americans than non-Hispanic WhiteAmericans.

Holt has expressed disappointment thatafter all of the talk, no compromise has been reached. She said with theexception of a few, there has been little communication with those affected,especially with the families that have been pushing for something to be done.They cant write the bill without us, she said.

Holt has credited Governor Tim Walz withbeing a great ally but said that something needs to be done soon. There havebeen two deaths this year involving young people who simply could not affordtheir insulin.

Holts son Alec Holt died in 2017 when heattempted to ration his insulin because it was no longer affordable on hissalary. He had been on his mothers health insurance plan until he turned 26.His mother said that he searched for an insurance plan that was affordable onhis restaurant salary. But the best option was one that required a $7,000deductible, so he eventually chose to pay for his medical expensesout-of-pocket while he continued to search for a plan that was affordable.

Alec only survived another month. He wentfrom paying two to three hundred for his insulin to a thousand dollars, whichwas out of reach on his salary. His effort to stretch his meds cost him hislife.

According to Health Care Cost Institute, patients with Type 1 diabetes spent an average of $5,705 in 2016, nearly double what they paid in 2015.

Dr. Victor Montori, an endocrinologist atthe Mayo Clinic in Rochester, accused the health care system of corrupting itsmission for the sake of profits. The consequences are human lives,he said. Its a cruelty we can no longer accept.

The Alec Smith Emergency Insulin Actpassed by the Minnesota House of Representatives last spring was designed tocreate a statewide insulin assistance program to help Minnesotans who struggleto afford the insulin they desperately need. The bill would be funded throughan Insulin Manufacturer Fee, that would be collected from the threelargest insulin manufacturers, Eli Lilly, French company Sanofi and the Danishfirm Novo Nordisk, that control more than 90% of the market.

Since the House passed its version of anemergency bill, two young Minnesota men have died. A 28-year-old whose familyrequested that he remain anonymous died as a result of rationing his insulinsupply. Jesimya Scherer-Radcliff, 21, who had Type 1 diabetes, died in July.His parents said he was on their health insurance, but he still could notafford the insulin he needed to survive. He reportedly had been rationing his insulinsupply. One of his relatives testified at a legislative hearing on the cost ofinsulin that, Jesy didnt die from insulin rationing, he died from theprohibitive cost of insulin.

The pharmaceutical companies absolutelyneed to be a big participant in this because they caused this problem,explainedHoward. They should have a role in solving it. There are onlythree insulin producers that control the entire market and they have ratchetedthe price up.

According to Rep. Howard, both billsrequire contributions from the insulin manufacturers. The House bill includes amanufacturers fee, while the Senate bill requires companies to provide freeinsulin. The other difference between the bills is the Senate bill allows forlong-term assistance, but leaves out the emergency option plan the Houseprefers as a safety net.

What we do produce is going to be a newbill that will take elements of the House proposal and the Senate proposal,which will create a safety net and provide longer-term situations.

The global insulin market is dominated bythree companies: Eli Lilly, the French company Sanofi and the Danish firm NovoNordisk. All three have raised list prices to similar levels. According to IBMWatson Health data, documented that Sanofis popular insulin brand Lantus was$35 a vial when it was introduced in 2001; its now $270. Novo NordisksNovolog was priced at $40 in 2001, and as of July 2018, it was priced at $289.

Rep. Howard said he holds out hope thatsomething that can be done before the next session in February. It does nothave to be either or it should be yes and. We should try to address theemergency issue first to prevent more loss of life but also look at solutionsfor long term affordability. There is a way to marry the two and accomplishboth goals, a compromise that leaves the door open for public funding as well,he said.

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A matter of life and death: insulin emergency/affordability bill... - MSR News Online

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Hormone Substitute Treatment Marketplace International Research and Forecast File 2018 to 2028 – Markets Gazette

In a contemporary marketplace learn about revealed via Truth.MR, the present and long term possibilities of the Hormone Substitute Treatment Marketplace are totally analyzed. Additional, the document contains an in-depth research of the quite a lot of components which are prone to keep an eye on the expansion trajectory of the Hormone Substitute Treatment Marketplace within the upcoming years.

As well as, a qualitative and quantitative evaluate of the quite a lot of marketplace segments is enclosed within the document and correctly represented within the type of tables, graphs, and figures.

The document signifies that the Hormone Substitute Treatment Marketplace is anticipated to sign in a CAGR expansion of ~XX% over the forecast length and succeed in a worth of ~US$XX. The document elaborates at the micro and macro-economic components which are projected to persuade the expansion of the Hormone Substitute Treatment Marketplace within the upcoming years 2018 to 2028.

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Crucial Findings of the File

Necessary questions referring to the Hormone Substitute Treatment Marketplace catered to within the document:

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Aggressive Panorama

The document on hormone alternative treatment marketplace gives intelligence on key individuals within the hormone alternative treatment marketplace. The important thing firms concerned within the components of hormone alternative treatment merchandise are totally assessed and profiled within the document. More than a few sides of pageant together with SWOT research, product portfolio research, drug tendencies and inventions, methods and key financials are coated. Key firms profiled within the document come with Novartis AG, Novo Nordisk A/S, Teva Prescription drugs, Mylan N.V and Pfizer Inc.

Primary firms within the hormone replacement therapy market are targeting increasing their present product portfolio. New hormone alternative treatment formulations being the core center of attention, firms are sporting out trials and acquire FDA approvals. As an example, Novartis AGs analysis and building division delivered 6 crucial FDA step forward treatment designations and 16 submissions in 2017. In Would possibly 2017, Novartis AG introduced U.S. FDAs popularity of its hormone alternative treatment pills for hormone receptor sure and metastatic breast most cancers remedy in postmenopausal ladies.

In February 2018, Novo Nordisk A/S concluded major section of REAL 1 the pivotal section three trial along side somapacitan, a long-acting expansion hormone to regard adults with expansion hormone deficiency.

Teva Prescription drugs Industries Ltd. presented Vagifem in 2017 for the remedy of atrophic vaginitis. In January 2018, the corporate introduced Estrace cream to regard reasonable and serious signs of vaginal and vulgar atrophy going on because of menopause. Likewise, Mylan N.V expanded its US portfolio within the ladiess healthcare vary with U.S. FDA popularity of Estradiol Vaginal Cream to regard vaginal atrophy.

Non-public fairness companies are fascinated about obtaining rising individuals which are excited by drug supply methods within the hormone alternative treatment area. As an example, Riverside Corporate has got DoseLogix in a bid to offer leading edge dosing dispensers for making sure correct dosing drugs of hormone alternative treatment and different prerequisites.

Click on to understand extra on competitive scenario within the hormone alternative treatment marketplace to know key methods of marketplace individuals

Definition

Hormone alternative treatment, additionally known as menopausal hormone treatment, is used to regard quite a lot of signs associated with menopause amongst ladies. Hormone alternative treatment replaces hormones which are low in stage as ladies close to menopause. There are quite a lot of kinds of hormone alternative treatment akin to estrogen hormone alternative treatment, thyroid hormone alternative treatment and expansion hormone alternative treatment.

Concerning the File

The document on hormone alternative treatment marketplace supplies incisive insights on all sides influencing expansion in call for for hormone alternative treatment international. The document supplies a radical research on call for of hormone alternative treatment throughout key areas within the globe along side gross sales of quite a lot of hormone alternative treatment merchandise.

Key drivers, demanding situations, tendencies and alternatives shaping the expansion of the hormone alternative treatment marketplace also are coated within the hormone alternative treatment marketplace document. The hormone alternative treatment marketplace document supplies historic knowledge evaluate on use of hormone alternative treatment, present hormone alternative treatment situation and long term call for of hormone alternative treatment. The forecast projections equipped duvet a timeline of 10 years (2018-2028).

Marketplace Construction

The hormone alternative treatment marketplace is segmented intimately to hide each attitude of the hormone alternative treatment area. The hormone alternative treatment marketplace has been segmented at the foundation of product sort, via dosage shape, via indication, via distribution channel and via area.

More than a few hormone alternative treatment merchandise akin to estrogen hormone alternative treatment, thyroid hormone alternative treatment and expansion hormone alternative treatment is roofed. Via dosage shape, hormone alternative treatment marketplace is segmented into pills, patches, injections, implants and lotions. Via indications, hormone alternative treatment marketplace is categorised into menopause, osteoporosis, thyroid and expansion hormone deficiency. Via distribution channel, its segmented via clinic pharmacies, clinics, retail pharmacies and on-line pharmacies.

The hormone alternative treatment marketplace is classified throughout key areas akin to North The united states, Latin The united states, Europe, Asia Pacific with the exception of Japan (APEJ), Center East and Africa (MEA) and Japan.

Further Questions Spoke back

Except key findings discussed above, the hormone alternative treatment marketplace document additionally solutions further questions akin to:

Analysis Method

The hormone alternative treatment marketplace is drafted the usage of a singular analysis technique comprising of a mixture of secondary and number one analysis methodologies. The knowledge gleaned from number one and secondary analysis is classified along side knowledge from exterior assets. All of the statistics are compiled the usage of triangulation way to acquire extremely correct projections on hormone alternative treatment marketplace.

Request methodology on hormone alternative treatment marketplace document.

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About Us

Truth.MR is an impartial, natural play marketplace intelligence company included with an purpose to ship prime quality, custom designed marketplace analysis answers that lend a hand our purchasers effectively cross to the marketplace supplied with actionable insights in a position to impacting the most important trade choices.

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Hormone Substitute Treatment Marketplace International Research and Forecast File 2018 to 2028 - Markets Gazette

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Acromegaly Treatment Industry 2019 Global Market Size, Demand, Growth Prospects, Key Insights, Top Companies and Forecast till 2025 – VaporBlash

The global Acromegaly Treatment market is driven by the growing prevalence of the genetic disease, changing lifestyle. Also, factors such as rising incidence of hormonal diseases, such as hypopituitarism and endocrine diseases, and high demand for the advanced treatment is expected to increase the demand for Acromegaly treatment market.

Factors, such as unavailability of precise treatment and high cost of the surgery can restrain the market growth.

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Some of the key players operating in this market include Pfizer Inc., Chiasma Inc., Novartis AG, Ipsen Biopharmaceuticals Inc., Wockhardt Ltd., Troikaa Pharmaceuticals Limited, GlaxoSmithKline plc, Aegis Therapeutics LLC, Crinetics Pharmaceuticals Inc, Daewoong Pharmaceutical Co Ltd, Peptron Inc, among others.

Increasing government support, favorable government insurance policies and schemes for the patients and rapid developments in technology will offer lucrative opportunities.

Based on Application, the Acromegaly Treatment market is segmented into Hospitals, Clinics, and others.

Based on Disease Types, the Acromegaly Treatment market is segmented into Ectopic Acromegaly, Pseudo Acromegaly.

Regionally, North America was the largest revenue generator in the Acromegaly Treatment market in 2017, because of high investments in research and development activities to investigate the applications of Acromegaly Treatment market.

Key Benefits of the Report:

* Global, Regional, Country, Application, and Disease Types Market Size and Forecast from 2014-2025

* Detailed market dynamics, industry outlook with market specific PESTLE, Value Chain, Supply Chain, and SWOT Analysis to better understand the market and build strategies

* Identification of key companies that can influence this market on a global and regional scale

* Expert interviews and their insights on market shift, current and future outlook and factors impacting vendors short term and long term strategies

* Detailed insights on emerging regions, Application& Disease Types, and competitive landscape with qualitative and quantitative information and facts.

Global Acromegaly Treatment Industry 2019 Market Research Report is spread across 121 pages and provides exclusive vital statistics, data, information, trends and competitive landscape details in this niche sector.

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Target Audience:

* Acromegaly Treatment providers

* Traders, Importer and Exporter

* Raw material suppliers and distributors

* Research and consulting firms

* Government and research organizations

* Associations and industry bodies.

Research Methodology

The Market is derived through extensive use of secondary, primary, in-house research followed by expert validation and third party perspective like analyst report of investment banks. The secondary research forms the base of our study where we conducted extensive data mining, referring to verified data sources such as government and regulatory published materials, technical journals, trade magazines, and paid data sources.

For forecasting, regional demand & supply factor, investment, Market dynamics including technical scenario, consumer behavior, and end use industry trends and dynamics , capacity Production, spending were taken into consideration.

We have assigned weights to these parameters and quantified their Market impacts using the weighted average analysis to derive the expected Market growth rate.

The Market estimates and forecasts have been verified through exhaustive primary research with the Key Industry Participants (KIPs) which typically include:

* Original Manufacturer,

* Application Supplier,

* Distributors,

* Government Body & Associations, and

* Research Institute.

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Table Of Content

1 Executive Summary

2 Methodology And Market Scope

3 Acromegaly Treatment Market Industry Outlook

4 Acromegaly Treatment Market Type Outlook

5 Acromegaly Treatment Market Application Outlook

6 Acromegaly Treatment Market Regional Outlook

7 Competitive Landscape

End Of The Report

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Acromegaly Treatment Industry 2019 Global Market Size, Demand, Growth Prospects, Key Insights, Top Companies and Forecast till 2025 - VaporBlash

Recommendation and review posted by Bethany Smith

What Is Genetic Testing? The Complete WIRED Guide – WIRED

As these clinical tests became more common, scientists were also busy trying to drill deeper into the substance of DNA, the chemical structure of which had only been deciphered in 1953 by James Watson, Francis Crick, and Rosalind Franklin. Over the next few decades, scientists would come to understand that its helix-shaped pattern of paired basesadenine, thymine, cytosine, and guaninefunctioned like letters, spelling out words that a cell would decode into amino acids, the building blocks of proteins. They would also begin to realize that most of the human genomeabout 98 percentdoesnt actually code for proteins. In the '70s, junk DNA became the popularized term for these nonfunctional sections.

Not long after, in 1984, a British geneticist named Alec Jeffreys stumbled upon a use for all that so-called junk DNA: crime-fighting. In these regions of the genome, the DNA molecule tends to duplicate itself, like its stuttering over the same word over and over again. Scientists can capture and count these stutters, known as short tandem repeats. And because the number of STRs a person has at various locations is unique to them, they can be used to build a personally identifiable profile, or DNA fingerprint.

Genetic Testing Glossary

GenotypingTesting technology, often chip-based, that generates a partial list of your unique genetic differences.

Whole-genome sequencingA method used to determine the exact sequence of your entire genome, all 6.4 billion letters.

Whole-exome sequencingA method used to determine the exact sequence of the protein-coding portion of your genome, comprising about 22,000 genes.

Coverage/DepthA measure of how many times a DNA sequence has been proofread. 30X average depth of coverage is the benchmark of a high-quality sequence.

VariantA generic term referring to places in someones genome that differ from a reference genome

Single Nucleotide Polymorphism (SNP)A variant defined by a single letter change

Polygenic Score (PGS)An algorithm that adds up the effects of multiple variants to predict the likelihood of a physical or behavioral trait based on your DNA.

Preimplantation Genetic Diagnosis (PGD)A method for testing IVF embryos for genetic defects prior to starting a pregnancy.

Non-Invasive Prenatal TestingA method for screening a fetus for certain genetic disorders by testing the mothers blood. Confirming a diagnosis requires more invasive procedures.

Carrier ScreenA test used to find out if you carry any genes for disorders that you could pass on to your children.

Short Tandem Repeat (STR)A pattern of repeating sequences in the noncoding part of your genome used in forensic DNA testing

CODISA national database of genetic profiles collected from criminals and crime scenes, maintained by the US government.

In 1987, this technique was used for the first time in a police investigation, leading to the arrest and conviction of Colin Pitchfork for the rape and murder of two young women in the UK. That same year, Tommie Lee Andrews, who raped and stabbed to death a woman in Florida, became the first person in the US to be convicted as a result of DNA evidence. Since then, forensic DNA testing has put millions of criminals behind bars. In 1994, Congress signed the DNA Identification Act, giving the US Federal Bureau of Investigation authority to maintain a national database of genetic profiles collected from criminal offenders. As of September 2019, this database, known as CODIS, contains DNA from nearly 14 million people convicted of crimes, as well as 3.7 million arrestees, and 973,000 samples gathered at crime scenes.

Throughout the '80s and '90s, while cops were rushing to use DNA to catch rapists and murderers, geneticists were slowly doing detective work of their own. By linking health records, family pedigrees, disease registries, and STR locations and lengths, scientific sleuths painstakingly began to map traits onto chromosomes, eventually identifying the genes responsible for a number of inherited conditions, including Huntingtons disease, cystic fibrosis, and sickle-cell anemia. These diseases linked to single genes, so-called monogenic conditions, are basically binaryif you have the genetic mutation youre almost certain to develop the disease. And once the sequences for these faulty genes were revealed, it wasnt too hard to test for their presence. All you had to do was design a probea single strand of DNA attached to a signal molecule, that would send out a fluorescent burst or some other chemical flare when it found its matching sequence.

As the new millennium approached, companies were beginning to pilot such tests in various clinical settings, i.e. with a doctors order. That included testing amniotic fluid as part of prenatal screening, testing the blood of prospective parents (whats known as carrier screening), and testing the cells of embryos created by in vitro fertilization, in a process called pre-implantation diagnosis. These tests were expensive and targeted only at people with family histories of so-called monogenic diseases. Developing tests to assess a healthy persons risk of developing more complex conditions caused by the interaction of multiple genesthings like heart disease, diabetes, and cancerwould require a more detailed map of human DNA than the fragmented picture scientists had so far decoded. Luckily, that was just around the corner.

In 2000, a rough draft of the human genome sequence was made freely available online, followed three years later by a more complete, high-resolution version. With it, scientists and engineers now had enough information to load up chips with not one or two DNA probes but thousands, even hundreds of thousands. These microarrays made it possible to simultaneously scan a persons genome for thousands of SNPs, or single nucleotide polymorphismssingle changes in the arrangement of DNA letters that make people unique. These SNPs, or variants as theyre alternatively known, can be tallied up to rank a persons susceptibility to various illnesses.

And because this SNP snapshot technology, known as genotyping, could be done much cheaper than full sequencingin 2006 it cost $1,000 as opposed to $1 million for a full-genome scanit launched not only a new wave of research but a new industry: direct-to-consumer DNA testing.

Starting in the mid-2000s, dozens of companies began selling people a new genetic experience that didnt have to take place in a doctors office. They would take a sample of your DNAa few laboriously salivated milliliters of drool sent through the mailscan it, and peer into your ancestral past as well as forecast your genetic future. In the early days, these tests could provide only a limited amount of information. And many companies went under while waiting for researchers to amass more knowledge about the links between certain genes and human traits. But one deep-pocketed Silicon Valley startup weathered the creeping adoption curve (and a spat with the US Food and Drug Administration) to become synonymous with the retail genomics business: 23andMe.

Today though, as costs sink even further and the internet makes the exchange of cheek cells for genetic insights virtually frictionless, 23andMe again has plenty of competition. A recent study identified nearly 250 companies offering DNA tests that people can buy online. Most of these are tests for disease predisposition, ancestry, and paternity. But others offer biological inheritance as infotainmenttests offering matchmaking services, predicting childrens talents, recommending the right diet, or even identifying wines you might be genetically inclined to enjoy.

Customers should be aware though, that many of these recreational tests offer results with little relationship to realitythe science is still just too premature to be truly predictive for most complicated traits. They might be fun, but dont take them too seriously. (And if you care about genetic privacy, dont take them at all!) Even the more medically focused tests, like 23andMes health reports, should be taken with a grain of salt. Its testing formula for breast cancer risk, for example, is built around just three genetic variants in the BRCA genes, common in Ashkenazi Jewish populations and known to be associated with cancer. But there are thousands of other variants in those genes that can also raise your risk of breast cancer. Its just that 23andMes DNA chip isnt set up to capture them. In other words, a clean bill of health from 23andMe shouldnt be taken as definitive. The company emphasizes that its tests are probability readings, theyre not meant to be diagnostic. So if anything does come up, you still have to go see a doctor for confirmatory clinical testing.

Link:
What Is Genetic Testing? The Complete WIRED Guide - WIRED

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Preventive health care at the forefront of genetic testing at CHRISTUS Ochsner Wellness Screening and Genetics – KPLC

Breast cancer, cervical cancer, ovarian cancer, lung, throat, stomach and pancreatic," Manard said. "All immediate family members so, you know, seeing all that and helping them go through all that and watching them go through that I thought if I have a chance to prevent it, Im going to go with it.

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Preventive health care at the forefront of genetic testing at CHRISTUS Ochsner Wellness Screening and Genetics - KPLC

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The thrill of genetic genealogical discoveries should be tempered by ethical concerns – The Jewish News of Northern California

In these times of alarming disregard for scientific data (were talking to you, climate change deniers), lets hear it for science, specifically the astonishing gains made in the field of genetics and genetic testing.

Our three-part Past Lives series highlights the extraordinary resources now readily available to anyone curious about their family ancestry. Easy access to family records on the internet and the mapping of the human genome allows us to peer into our genetic past to learn more about who we are. And we can do both kinds of research from the comfort of our own homes.

For Jews, this has been a blessing for the most part. Unlike those Americans descended from Western European populations who can turn to comprehensive written archives, such as baptismal and marriage records, most Ashkenazi Jews like African Americans and Hispanic Americans lack the paper trail to trace their ancestry back further than a few generations.

Now, with the evolution of genetic testing, we can pinpoint to a remarkable degree of precision the composition of our ethnicity and where we came from. And all it takes is a simple cheek swab.

For some, discovering Jewish roots opens the door to new connections and layers of spiritual meaning.

As our stories show, this technology is about more than percentages and places on the map. For some, discovering the very existence of Jewish roots is a personal marvel, opening the door to new connections and layers of spiritual meaning.

However, as with any technology, ethical concerns run rampant.

Are we now as a global kehillah to rely on DNA test results as a proving ground for belonging to the Jewish people? What about those who convert to Judaism and might hail from different backgrounds? When their DNA pie chart comes back with zero percent Jewishness, does that mean they are any less Jewish?

Though matrilineal descent long ago enshrined a genetic aspect to Judaism, have we not seen enough of eugenics, racism, white nationalism and hate-fueled violence to check a rush to embrace anything that smacks of genetic purity?

These concerns have come to the forefront in Israel, where for the past two years the Chief Rabbinate has been using genetic testing to confirm the Jewishness of immigrants from the former Soviet Union seeking marriage licenses, in cases where the applicants dont have sufficient documentation of their status. Dozens of young couples, and their close relatives, have been humiliated in this way, and the practice is now being challenged before Israels High Court, brought there by the largely immigrant Yisrael Beiteinu party.

Scientific discoveries often involve thorny ethical questions. They must be faced openly.

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The thrill of genetic genealogical discoveries should be tempered by ethical concerns - The Jewish News of Northern California

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Caris Life Sciences and Ambry Genetics Partner to Advance Cancer Care – BioSpace

IRVING, Texas and ALISO VIEJO, Calif., Dec. 4, 2019 /PRNewswire/ --Caris Life Sciences, a leading innovator in molecular science focused on fulfilling the promise of precision medicine, and Ambry Genetics(Ambry), a leading clinical genetic testing company, today announced that Caris will begin offering Ambry's 67-gene CancerNext-Expanded panel to evaluate the hereditary risks for cancer. Combined with Caris' somatic (tumor) tests that analyze a cancer's detailed molecular makeup, Caris will provide patients and their healthcare providers unparalleled information to more accurately diagnose and treat cancer. This will be the most comprehensive, clinically relevant molecular and genetic offering on the market today to guide treatment and management of cancer.

"We are committed to providing clinicians with high-quality information they can use to inform treatment decisions," said David D. Halbert, Caris Life Sciences Chairman, Chief Executive Officer and Founder. "By partnering with Ambry Genetics to better inform patient care, we are able to provide clinicians a greater ability to learn about a cancer's molecular composition."

Caris currently offers clinicians Caris Molecular Intelligence, a proprietary, comprehensive tumor profiling approach that assesses DNA, RNA, and proteins unique to an individual's cancer to reveal a molecular blueprint in order to guide more precise and individualized treatment decisions.

Through the partnership, Caris will now offer Ambry's CancerNext-Expanded hereditary cancer panel. This panel analyzes 67 genes associated with an increased hereditary risk of cancer, including brain, breast, colon, ovarian, pancreatic, prostate, renal, uterine, and many other cancers. Its comprehensive testing identifies inherited risks for cancer in order for clinicians to accurately diagnose, treat, and manage cancer risks for each patient's needs.

"To best diagnose and treat cancer, clinicians must understand whether patients have mutations in genes associated with an increased risk for hereditary cancer," said Aaron Elliott, Chief Executive Officer of Ambry. "Caris' molecular tests combined with Ambry's germline genetic testing, give clinicians the most comprehensive, clinically relevant molecular profile on the market to guide treatment and management."

The combined Caris and Ambry testing is now available nationwide.

"Being able to simultaneously conduct comprehensive tumor genomic testing and multi-gene germline sequencing is invaluable, especially for sick patients at the beginning of their cancer journey," said Michael J. Hall, M.D., M.S., Chair, Department of Clinical Genetics at Fox Chase Cancer Center. "This is information I can immediately begin using for my patients to more accurately diagnose them and to better individualize their treatments."

About Caris Life Sciences Caris Life Sciences is a leading innovator in molecular science focused on fulfilling the promise of precision medicine through quality and innovation. The company's suite of market-leading molecular profiling offerings assesses DNA, RNA and proteins to reveal a molecular blueprint that helps physicians and cancer patients make more precise and personalized treatment decisions.

Caris is also advancing precision medicine with Next Generation Profiling that combines its innovative service offerings, Caris Molecular Intelligence and ADAPT Biotargeting System, with its proprietary artificial intelligence analytics engine, DEAN, to analyze the whole exome, whole transcriptome and complete cancer proteome. This information, coupled with mature clinical outcomes on thousands of patients, provides unmatched molecular solutions for patients, physicians, payers and biopharmaceutical organizations.

Whole transcriptome sequencing with MI Transcriptome provides the most comprehensive and unique RNA analysis available on the market and covers all 22,000 genes, with an average of 60 million reads per patient, to deliver extremely broad coverage and high resolution into the dynamic nature of the transcriptome. Assessing the whole transcriptome allows us to dig deeper into the RNA universe to uncover and detect fusions, splice variants, and expression changes that provide oncologists with more insight and actionable information when determining treatment plans for patients.

Caris Pharmatech, a pioneer of the original Just-In-Time research system with the largest research-ready oncology network, is changing the paradigm from the traditional physician outreach model to a real-time approach where patient identification is completed at the lab and the physician is informed so that the patient can be enrolled days earlier, and remain in the local physician's care, without having to travel to a large central trial site. This fundamentally redefines how pharmaceutical and biotechnology companies identify and rapidly enroll patients in precision oncology trials by combining Caris' highest quality industry leading large-scale molecular profiling services with Pharmatech's on-demand site activation and patient enrollment system.

Headquartered in Irving, Texas, Caris Life Sciences offers services throughout the U.S., Europe, Asia and other international markets. To learn more, please visitwww.CarisLifeSciences.comor follow us on Twitter (@CarisLS).

About Ambry GeneticsAmbry Genetics, as part of Konica Minolta Precision Medicine, excels at translating scientific research into clinically actionable test results based upon a deep understanding of the human genome and the biology behind genetic disease. Our unparalleled track record of discoveries over 20 years, and growing database that continues to expand in collaboration with academic, corporate and pharmaceutical partners, means we are first to market with innovative products and comprehensive analysis that enable clinicians to confidently inform patient health decisions. We care about what happens to real people, their families, and the people they love, and remain dedicated to providing them and their clinicians with deeper knowledge and fresh insights, so together they can make informed, potentially life-altering healthcare decisions. For more information, please visitambrygen.com.

Caris Company Contact & Media:Srikant RamaswamiVice President, Chief Communications Officersramaswami@carisls.com +1-214-769-5510

Ambry Genetics Media Contact:Liz Squirepress@ambrygen.com (202) 617-4662

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Caris Life Sciences and Ambry Genetics Partner to Advance Cancer Care - BioSpace

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Global Hematologic Malignancies Testing Market 2019-2025: Increasing Adoption of Inorganic Growth Strategies in the Market – P&T Community

DUBLIN, Dec. 4, 2019 /PRNewswire/ -- The "Global Hematologic Malignancies Testing Market: Focus on Product, Disease, Technology, End User, Region/Country Data and Competitive Landscape - Analysis and Forecast, 2019-2025" report has been added to ResearchAndMarkets.com's offering.

The market to grow at a significant CAGR of 15.24% during the forecast period, 2019-2025.

Growth in this market is driven by the rising incidence of hematologic malignancies, increasing adoption of inorganic growth strategies in the market, favorable reimbursement scenario in the global hematologic malignancies testing market, and increasing funding in hematologic malignancies testing market. The massive scope in the emerging economies and technological advancements in the field of molecular diagnostics are also expected to provide growth opportunities for players in the market.

The molecular discovery of disease-causing genes in blood cancers has led to the global phenomenon known as precision diagnostics in hematologic malignancies. By understanding the underlying molecular mechanisms of diseases and genetic composition and regulation in humans, molecular diagnostics have empowered physicians to move away from broadly classifying patients according to diseases, shifting toward individualized treatment, with the adoption of specialized kits, and services aimed at an early screening of disease.

The global hematologic malignancies testing market has witnessed significant growth, attributing to the increasing demand for multiple biomarker based-molecular diagnostics. The developments have been instrumental in improving the quality of life and reducing the cost and time of treatment, boosting the growth of the overall healthcare diagnostics market. Further, the increasing awareness and adoption of the precision diagnostic approach in developed as well as developing countries is the key driver for the growth of the global hematologic malignancies testing market.

The evolution of new technologies is enabling the acquisition of more information for more exhaustive characterization of the disease state, the most suitable treatment alternatives for a patient, or in the case of blood cancer, the rapid identification of a specific gene that could be responsible for a particular carcinoma.

Approaches that have the potential to address this requirement for highly increasing amounts of data include multi-marker or multiplexed approaches (methods that can test for multiple biomarkers in a single assay) and gene sequencing that enable the detection of multiple defects that are associated with a particular subset of patients with a disease. The practical application of these methods can be challenging owing to the complexity of the involved assays.

Key Questions Answered in this Report:

Key Topics Covered:

Executive Summary

1 Market Overview1.1 Introduction1.2 Historical Perspective1.3 Types of Hematologic Malignancy Diagnostic Tests1.4 Global Footprint1.5 Trends and Future Potential

2 Market Dynamics2.1 Market Drivers2.1.1 Rising Incidence of Hematologic Malignancies2.1.2 Increasing Adoption of Inorganic Growth Strategies in the Market2.1.3 Favorable Reimbursement Scenario in the Global Hematologic Malignancies Testing Market2.1.4 Increase in Funding in Hematologic Malignancies Testing Market2.2 Restraints2.2.1 High Pricing Pressure2.2.2 Lack of Trained Professionals2.2.3 Issues Pertaining to the Analytical Validity of Genetic Testing for Cancers2.3 Market Opportunities2.3.1 Opportunities in the Emerging Economies2.3.2 Technological Advancements in the Field of Molecular Diagnostics

3 Competitive Landscape3.1 Key Strategies and Developments3.2 Product Scenario3.3 Funding Scenario3.4 Market Share Analysis3.5 Growth Share Analysis (Opportunity Mapping)3.6 Comprehensive Competitive Index

4 Industry Insights4.1 Regulatory Framework4.1.1 Legal Requirements and Framework in the U.S.4.1.2 Legal Requirements and Framework in Europe4.1.3 Legal Requirements and Framework in Asia-Pacific4.1.3.1 Japan4.1.3.2 China4.2 Reimbursement Scenario4.3 Physicians' Perceptions

5 Global Hematologic Malignancies Testing Market (by Product)5.1 Services5.2 Kits5.2.1 Gene Panels5.2.2 Molecular Clonality Testing5.2.3 Translocation Testing5.2.4 Mutation Testing5.2.5 Minimal Residual Disease (MRD) Testing

6 Global Hematologic Malignancies Testing Market (by Disease)6.1 Leukemia6.2 Lymphoma6.3 Multiple Myeloma6.4 Myeloproliferative Neoplasms6.5 Myelodysplastic Syndromes

7 Global Hematologic Malignancies Testing Market (by Technology)7.1 Next-Generation Sequencing (NGS)7.2 Polymerase Chain Reaction (PCR)7.3 Fluorescence in Situ Hybridization (FISH)7.4 Immunohistochemistry (IHC)7.5 Flow Cytometry7.6 Other Technologies

8 Global Hematologic Malignancies Testing Market (by End User)8.1 Specialty Clinics and Hospitals8.2 Diagnostic Laboratories8.3 Reference Laboratories8.4 Research Institutions

9 Global Hematologic Malignancies Testing Market (by Region)

10 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/r9y3z8

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Hematologic Malignancies Testing Market 2019-2025: Increasing Adoption of Inorganic Growth Strategies in the Market - P&T Community

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Global Hematologic Malignancies Testing Market to 2025: Focus on Product, Disease, Technology, End User, Region/Country Data and Competitive Landscape…

DUBLIN--(BUSINESS WIRE)--

The "Global Hematologic Malignancies Testing Market: Focus on Product, Disease, Technology, End User, Region/Country Data and Competitive Landscape - Analysis and Forecast, 2019-2025" report has been added to ResearchAndMarkets.com's offering.

Key Questions Answered in this Report:

Global Hematologic Malignancies Testing Market Forecast, 2019-2025

The hematologic malignancies testing market growth has been primarily attributed to the major drivers in this market such as rising incidence of hematologic malignancies, increasing adoption of inorganic growth strategies in the market, favorable reimbursement scenario in the global hematologic malignancies testing market, and increasing funding in hematologic malignancies testing market. However, there are significant challenges that are restraining the market growth. These challenges include high pricing pressure, lack of trained professionals, and issues pertaining to the analytical validity of genetic testing for cancers.

Scope of the Market Intelligence on Hematologic Malignancies Testing Market

The hematologic malignancies testing market report provides a holistic view of the market in terms of various factors influencing it, including product optimization, and technological advancements.

The scope of this report is centered upon conducting a detailed study of the products and manufacturers allied with the market. In addition, the study also includes exhaustive information on the unmet needs, perception of the new products, competitive landscape, market share of leading manufacturers, the growth potential of each underlying sub-segment, and company, as well as other vital information with respect to global hematologic malignancies testing market.

Key Topics Covered:

Executive Summary

1 Market Overview

1.1 Introduction

1.2 Historical Perspective

1.3 Types of Hematologic Malignancy Diagnostic Tests

1.4 Global Footprint

1.5 Trends and Future Potential

2 Market Dynamics

2.1 Market Drivers

2.1.1 Rising Incidence of Hematologic Malignancies

2.1.2 Increasing Adoption of Inorganic Growth Strategies in the Market

2.1.3 Favorable Reimbursement Scenario in the Global Hematologic Malignancies Testing Market

2.1.4 Increase in Funding in Hematologic Malignancies Testing Market

2.2 Restraints

2.2.1 High Pricing Pressure

2.2.2 Lack of Trained Professionals

2.2.3 Issues Pertaining to the Analytical Validity of Genetic Testing for Cancers

2.3 Market Opportunities

2.3.1 Opportunities in the Emerging Economies

2.3.2 Technological Advancements in the Field of Molecular Diagnostics

3 Competitive Landscape

3.1 Key Strategies and Developments

3.2 Product Scenario

3.3 Funding Scenario

3.4 Market Share Analysis

3.5 Growth Share Analysis (Opportunity Mapping)

3.6 Comprehensive Competitive Index

4 Industry Insights

4.1 Regulatory Framework

4.1.1 Legal Requirements and Framework in the U.S.

4.1.2 Legal Requirements and Framework in Europe

4.1.3 Legal Requirements and Framework in Asia-Pacific

4.1.3.1 Japan

4.1.3.2 China

4.2 Reimbursement Scenario

4.3 Physicians' Perceptions

5 Global Hematologic Malignancies Testing Market (by Product)

5.1 Services

5.2 Kits

5.2.1 Gene Panels

5.2.2 Molecular Clonality Testing

5.2.3 Translocation Testing

5.2.4 Mutation Testing

5.2.5 Minimal Residual Disease (MRD) Testing

6 Global Hematologic Malignancies Testing Market (by Disease)

6.1 Leukemia

6.2 Lymphoma

6.3 Multiple Myeloma

6.4 Myeloproliferative Neoplasms

6.5 Myelodysplastic Syndromes

7 Global Hematologic Malignancies Testing Market (by Technology)

7.1 Next-Generation Sequencing (NGS)

7.2 Polymerase Chain Reaction (PCR)

7.3 Fluorescence in Situ Hybridization (FISH)

7.4 Immunohistochemistry (IHC)

7.5 Flow Cytometry

7.6 Other Technologies

8 Global Hematologic Malignancies Testing Market (by End User)

8.1 Specialty Clinics and Hospitals

8.2 Diagnostic Laboratories

8.3 Reference Laboratories

8.4 Research Institutions

9 Global Hematologic Malignancies Testing Market (by Region)

10 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/polq31

View source version on businesswire.com: https://www.businesswire.com/news/home/20191204005431/en/

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Global Hematologic Malignancies Testing Market to 2025: Focus on Product, Disease, Technology, End User, Region/Country Data and Competitive Landscape...

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Stem Cell Therapy Market Size, Share, Trends, and Opportunity Analysis by 2017 – 2025 – The Market Expedition

Global Stem Cell Therapy Market Analysis

The recent report published by TMRR on the global Stem Cell Therapy market is an in-depth analysis of the overall prospects of the Stem Cell Therapy market in the upcoming years. The data collected from credible primary and secondary sources is accurately represented in the report backed up by relevant figures, graphs, and tables. The report includes a quantitative and qualitative analysis of the various aspects of the market by collecting data from the key participants in the Stem Cell Therapy market value chain.

The report reveals that the global Stem Cell Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and surpass the value of ~US$XX by the end of 2029. The presented study also includes a thorough analysis of the micro and macroeconomic factors, regulatory framework, and current trends that are expected to influence the growth of the Stem Cell Therapy market during the assessment period.

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Stem Cell Therapy Market Segmentation

The market study put forward by TMRR segments the global Stem Cell Therapy market to offer a microscopic understanding of the various aspects of the Stem Cell Therapy market. The Stem Cell Therapy market is segmented on the basis of region, product type, end-user, and more.

The study offers a Y-o-Y growth projection of each market segment and sub-segment over the stipulated timeframe of the study.

Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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