Archive for October, 2019

Since the 1970s, genetic screening has been standard practice during pregnancy. The goal of this kind of genetic testing has historically been to understand the likelihood an embryo carries a heritable disease, the classic example being the recessive genes for autosomal recessive disorders like cystic fibrosis, sickle cell anemia, and Tay Sachs, or tests that determine developmental disorders like Down Syndrome. This allows parents to make informed choices about ending pregnancies and planning ahead for potential issues. But as genetic technologies develop and gene editing becomes more widely practiced the possibilities are not limited to observation. As Dr. Robert Klitzsman writes inDesigning Babies: How Technology is Changing the Way We Create Children, genetic testing in the context of IVF is already giving us a glimpse of the ways in which the proliferation of new technologies could affect the genome of future children and future generations. And its not all good news.

Klitzman, Director ofColumbia Universitys Masters of Bioethics Program, suggests that progress has not been accompanied by adequate oversight or regulation. What are the limits of genetic screening, genetic testing, and gene editing technologies? The answer has as much to do with what risks are deemed acceptable and what risks are not. Designer babies are no longer the stuff of science fiction, but would-be parents arent exactly in a DNA Build-a-Bear. A transition is underway and Klitzman believes that parents need to understand the bleeding edge of whats possible to have the necessary context to understand the technologies they are using even in the process of basic screenings.

Fatherly spoke to Dr. Klitzman about the current state of genetic testing and the potential risks of emerging technologies.

Expecting parents likely understand one side of genetic testing, screening for heritable diseases. But new technologies and new discoveries mean that we can do more with genetic information and with genes themselves than ever before. So what are the limits of the current technologies?

A few years ago people thought wed find the cancer gene or the fat gene. But now we know that for most common diseases and most complex traits there are many genes involved. Sure there are certain genes that increase the risk of cancer from, say, 5 or 10 percent. But in genetic screening for diseases people should realize that, for a lot of diseases, the world is more complicated than just screening an embryo.

So its the old debate: nature or nurture? The answer is both. For a lot of traits, genetics explains the part but not all of the risk of the disease. So you may undergo genetic testing or you may screen embryos and the child may still get certain diseases. So its not always foolproof.

But its better than nothing.

Its important for parents to get tested to see if they have recessive conditions particularly if its in their family. If anyone has cystic fibrosis in their family, they should be tested to know. If theyre a carrier, they should see if their spouse is a carrier. If anyone has breast cancer in their family, they should be tested to see if they have that mutation. I think people with sickle cell disease should be tested for that. I think any woman who is over 35 should have the embryo tested for down syndrome and other chromosomal abnormalities. So I think there are certain diseases.

But, through evolution, most diseases for which there is a very predictive genetic test tend to be rare. If there was a terrible gene that was wiping out people, it wouldnt be getting passed on. The only genes that get passed on are not going to be from really terrible mutations because they would kill the people and by and large they wouldnt have any kid.

Its interesting that you point out the limits of genetic testing technologies because youre also a strong advocate for increased access.

I think insurance should pay for genetic testing. If a couple is concerned because their cousin has cystic fibrosis or someone in their family has sickle cell disease and wants to get tested, that should be covered. It may not be covered now so I think thats another set of policies that need to change. And part of that I think there needs to be more genetic counseling, which insurance also doesnt cover. The laws havent kept up with the technology. Our technologies advanced way ahead of our legal system and our duty to understand and figure out what to do with that of ethical legal and social questions involved.

A lot of the thornier issues you write about involve preimplantation genetic diagnosis, which is genetic testing post-conception and prior to pregnancy in the context of IVF. How are the decisions made by would-be parents undergoing PGD different than the decisions being made in the context of normal genetic testing?

Right now, we genetically screen embryos. When a couple undergoes IVF, lets say they create eight embryos. Doctors could say, These four are the girls, these four are the boys. Now, lets say a family has a history of breast cancer or the mother has the BRCA gene which carries breast cancer. The doctors could say, These three embryos have breast cancer gene these five dont. And the couple can choose the ones that dont.

Also, increasingly couples can say, Well, I just want a boy. And that creates a number of ethical challenges as opposed to letting Mother Nature do whatever it would do.

So theres potential in that specific context to take action in a way thats not possible in typical screenings. How much of that action is embryo selection versus actual gene manipulation?

We can take genes out. Theres a gene associated with Huntingtons disease or the BRCA breast cancer gene. We now have the technology to take them out.But these technologies are still in an experimental phase. And Im concerned that theyll soon be made fairly widely available even though there still may be risks involved and people may or may not fully appreciate those risks.

A few years ago, a doctor in China used CRISPR to edit the genes of twin girls. That opened brought a lot of criticism but also raised awareness of what can be done with this technology.

Thats right. So what Dr. He Jiankui did is that he worked with fathers who had HIV. There was a concern that the father could potentially pass HIV onto the child. And so he took the embryo and disabled the CCR5 gene that is involved in letting HIV get into a cell. The problem is that when you disable that gene, the risk of getting HIV goes down but the risk of getting influenza getting in goes up as do other risks.

DNA consists of three billion molecules. Each of us is a shelf of books in an office that has three billion letters in them. Well, if you go in and rip out some letters, you want to make sure you rip out the right ones. And so it looks like Dr. He didnt do it so precisely. So in fact what he said he took out wasnt exactly what he took out. In other words, if a child is born and missing part of the DNA, that part might be the next gene thats involved for, say, brain development or something like that.

You need to be very, very careful.

Presumably, the ethical questions get more complicated when gene editing becomes a more widely available procedure.

Until 60 years ago, we didnt even know what DNA did. We now have the ability to identify genes and were increasingly finding genes that are associated with not only various diseases but also human traits those associated with blonde hair and blue eyes, those associated with height and perfect pitch.

I think CRISPR probably will be used for people wanting or not wanting certain socially desirable or undesirable traits in their children.

A Gattaca situation.

Yes, exactly.

This interview has been condensed and edited for clarity.

Original post:
Gene Editing Is Creating a New World of Designer Babies. Are We Ready For It? - Fatherly

U.S. Attorney's Office in the Eastern District of Louisiana issued the following announcement on Oct. 9.

The Justice Department announced today that UTC Laboratories, Inc. (RenRX) has agreed to pay $41.6 million, and its three principals, Tarun Jolly, M.D., Patrick Ridgeway, and Barry Griffith, have agreed to pay $1 million to resolve allegations that they violated the False Claims Act by paying kickbacks in exchange for laboratory referrals for pharmacogenetic testing and for furnishing and billing for tests that were not medically necessary. RenRX, a laboratory company headquartered in New Orleans, Louisiana, also agreed to a twenty-five year period of exclusion from participation in any federal health care program.

The payment of kickbacks in exchange for medical referrals undermines the integrity of our healthcare system. Todays settlement reflects the Department of Justices commitment to ensuring that taxpayer monies are well spent and not wasted on unnecessary medical testing, said Assistant Attorney General Jody Hunt of the Department of Justices Civil Division.

Healthcare fraud, in any incarnation, hurts patients, honest medical practitioners, and all of the nations taxpayers, said United States Attorney for the Eastern District of Louisiana Peter G. Strasser. The favorable resolution of this False Claims Act matter illustrates the collaborative efforts and firm commitment by our federal partners to use all available remedies, both civil and criminal, to address signs of waste and abuse by providers in our healthcare markets.

The government alleged that between 2013 and 2017, UTC and its principals offered and paid remuneration to physicians to induce the ordering of pharmacogenetic tests, purportedly in return for their participation in a clinical trial known as the Diagnosing Adverse Drug Reactions Registry (DART), clinical trial identifier NCT01970709. The government also alleged that UTC and its principals offered and paid remuneration, including sales commissions, to entities and individuals as part of the scheme, and furnished pharmacogenetic tests that were not medically necessary and billed the Medicare program.

Original source can be found here.

Read more from the original source:
ATTORNEY'S OFFICE OF LOUISIANA: Genetic Testing Company and Three Principals Agree To Pay $42.6 Million to Resolve Kickback and Medical Necessity...

Depressive disorders are among the most common conditions that disrupt lives. Fortunately, medications, psychotherapies, and lifestyle changes are usually successful in treating depression and related disorders, even if symptoms are not entirely eliminated. Sometimes people dont gain sufficient relief from treatment, or must try several medications before finding one that works well. In an age of exciting advances, including brain imaging and genetic testing, many doctors and patients reasonably hope that new technologies will offer answers. And in fact, for antidepressant choice, several companies sell genetic testing as a means to guide treatment. But do these tests work?

Genes determine some of our risk for depression and some of our response to treatment. However, no single gene or small number of genes determines much of either in the general population. And the few genes used in the current commercial test panels do not appear to be the key genes determining risk or response. Some of the genes tested are related to drug metabolism. These genes can affect drug levels in the blood, but generally dont predict clinical response. Other factors, including age, diet, hormonal state, gut bacteria, and any other concurrently taken drugs, are far more important in determining how a person metabolizes a drug and responds to treatment.

Most people with depression improve with careful evaluation of all of these factors, appropriate antidepressant choice and dosing according to expert guidelines, as well as follow-up care to monitor treatment response and address any side effects. Currently, there is no scientific evidence that gene tests are needed or would be helpful as part of those assessments.

A dozen studies focusing on patients with depressive disorders have reported outcomes from using commercially available gene test panels to guide antidepressant choice. Most studies were completely unblinded that is, doctors and patients knew a special test was given. Even with that bias, the use of gene results showed no evidence of effectiveness. A few studies were partially blinded, but doctors and patients still knew some patients got a special test. In these studies, too, the tests failed to show value on their key measures of efficacy.

Notably, many patients had not responded well before entering a study because they were receiving inappropriate treatments. They improved when switched to more standard treatments. However, the same changes would have been made without guidance from the test if the treating clinicians had simply followed good practice, rather than getting an unproven and expensive genetic test. And our ongoing review of newer studies on these tests suggests similar flaws and no further evidence favoring their use.

Against this background, experts with no financial interest in genetic testing have repeatedly recommended that genetic tests should not be used in choosing treatments for depression (see here and here). The American Psychiatric Association convened a task force that reviewed the evidence and agreed: the tests should not be ordered.

Recently, the FDA advised that the tests had no proven value and should not be used. Then they went two steps further, stating that use of the tests could lead to inappropriate treatment choices that might harm patients. Additionally, the FDA sent a warning letter to one company and has contacted others selling the tests, advising them that they cannot legally make specific recommendations to clinicians or patients based on their test results.

Genetic testing is appealing, both to vulnerable patients and time-constrained doctors. And it is vigorously marketed to both parties by the companies that sell it: through news reports, websites, television, and magazines, and to doctors in their offices. There are few restraints that hold that marketing to the facts, yet the facts are clear in evidence summarized by numerous experts and agencies. Currently available genetic test panels have no proven value for choosing antidepressant treatment, and their use risks providing inappropriate care. So, while gene testing can be very useful for some other conditions, notably some cancer treatments, that success does not yet apply in treating depression. Perhaps this will change with more research, but appropriate tests are years away.

In the meantime, there are many good and effective actions to take if treatment is not working well. You and your doctor can

When a medication change is needed, the clinician treating you should follow available guidelines (such as these) or help you obtain a consult from a mental health professional who is more knowledgeable about psychiatric medications. Psychiatry consultations are available at most hospitals and clinics; some hospitals offer these consults by phone or through their websites.

See the article here:
Gene testing to guide antidepressant treatment: Has its time arrived? - Harvard Health Blog - Harvard Health

When Angelina Jolie broke the news about her genetic testing results, a new day dawned for cancer treatment. By opening up about her medical decisions to prevent cancer before it started, she shone a bright light on the positives and negatives of genetic testing for cancer.

So why did she do it?

Research has shown that up to 10% of cancers are caused by factors passed from one generation to the next. These syndromes are known as hereditary cancers, and genetic tests can determine a persons risk for developing these cancers.

Medical professionals get concerned when we see red flags for hereditary cancers that include family members with:

A diagnosis of cancer at a young age.

Several family members with cancer.

Relatives with more than one type of cancer.

A history of different cancers in the same person.

Rare or unusual cancers, such as ovarian cancer, pancreatic cancer, or male breast cancers.

Ashkenazi or eastern European ancestry.

There are many benefits to getting tested, regardless of the eventual result. If one of your family members had cancer, there is a chance that you inherited a gene mutation that not only increases your personal risk of cancer, but also could be passed to the next generation. Those who are carriers of hereditary cancer gene mutations could be at risk of getting cancer earlier in life than the general population. The sooner genetic testing is done, the more likely it is that the risk can be managed appropriately.

If you have had cancer at a young age, a rare cancer, or if cancer occurs frequently in your family, genetic testing may be an important first step for you. If a greater-than-average risk of cancer is found, there are a number of things you and your health-care professional can do to manage that risk.

You might be advised to have more frequent screening to help detect cancer at an earlier, more treatable stage and improve cancer survival. Your health-care professional may recommend preventive strategies, including risk-reducing medications or surgeries, that may reduce your risk of developing cancer. You and your health-care professional can make more informed decisions on your treatment options.

Test results can help your relatives learn more about the inherited risk and how it may affect them.

In addition, family members who do not carry mutations that increase their cancer risk may avoid unnecessary medical interventions. These results also provide valuable information for use in customizing medical management plans, can help your health-care professional make a timely and accurate diagnosis and assist your health-care professional in making important decisions about the management of your disease.

Genetic testing is not for everyone, but if you or your family members have any of the red flags mentioned above, call patient navigators Catherine Gaines or Windy Christy at Gibson Prevention Center to schedule an appointment for further evaluation at 910-671-5357.

Christy

Windy Christy is a physician assistant and patient navigator with Gibson Prevention Center at SeHealths Gibson Cancer Center.

Link:
A genetic cancer risk test is a valuable preventative tool - The Robesonian

Verily Life Sciences, Alphabet's health research arm and sister company to Google Health, announced it'spartnering with genetics and health technology firm Color to supply participants of Verily's Project Baseline research platform with genetic information. Business Insider Intelligence

Project Baseline began in 2017 with goals of making clinical research more accessible to participants and arriving at a quantifiable "baseline" for good health. The research project has since launched several clinical research projects in partnership with some of the largest names in healthcare, including: Pfizer, Mayo Clinic, Novartis, the American Heart Association, and Stanford Medicine.

The partnership could enable Verily to incorporate data on genetic risk factors into its various clinical studies, leading to a more in-depth and holistic understanding of health.It's unclear exactly how information gleaned from Color's genetic tests will be leveraged in Project Baseline studies, but it's possible that future research initiatives may choose to examine how genetic risk factors affect health outcomes alongside patients' behavior and medical history.

And allowing Project Baseline members access to genetic testing and personalized health advice may improve participant engagement with the particular research program they're involved in and with the Project Baseline platform overall, which is critical given that86%of clinical trials fail to hit their participation goals.

We caught up with Color CEO Othman Laraki to discuss how a Verily-Color tie up furthers Color's goals for a genetic future of healthcare below are some key takeaways from our conversation:

We think Color will benefit from the exposure that comes when partnering with a Google-affiliated business, and more users should facilitate stronger population health insights.In the last two months, Color has scored massive partnerships with NIH and its All of Us program and now Verily: two major names in healthcare. And each new project raises not just Color's profile, but that of the genetic testing field as a whole, according to Laraki.

Laraki pointed out that the idea of every home having a personal computer was once considered crazy and that one day it may be the case that genetic data is as commonplace in healthcare as computers are in the home. But this might only be possible if far-reaching research programs like All of Us and Project Baseline can successfully attract participants and deliver actionable results.

Color has become a standout player in genetic testing by focusing on large-scale population health projects which I (Zach) think is a smart business model in the face of apotential slowdownin the direct-to-consumer genetic testing market."In some ways, using a doctor's time to measure your height and have them listen to your heartbeat is almost more expensive now than getting a complete genomic profile," says Laraki.

And the fact that genetic testing is becoming cheaper for consumers could be part of why we're seeing so much interest from providers and research firms in population-level genetic health research: MIT Technology Review now estimates that over100 millionpeople globally will have taken an at-home genetic test by 2021, up from the 26 million consumers at the beginning of 2019, for example.

With industry leaders like Illumina expressing concerns around a potential slowdown for the direct-to-consumer genetic testing market, a model that's given rise to 23andMe and Ancestry the two biggest names in genetic testing today I think that Color's model will conversely gain traction as providers are increasingly becominginterestedin moving beyond individual patient results and searching for the root cause of conditions affecting their communities.

Want to read more stories like this one? Here's how to get access:

See the original post here:
Alphabet-backed Verily partners with Color to bring genetic insights to its research - Business Insider Nordic

WALTHAM, Mass.--(BUSINESS WIRE)--PerkinElmer, Inc., a global leader committed to innovating for a healthier world, today introduced its PG-Seq Rapid Non-Invasive Preimplantation Genetic Testing for Aneuploidy (PGT-A) kit. This solution tests spent embryo culture media for chromosomal abnormalities during in vitro fertilization (IVF) treatment.

PGT-A is used to identify viable embryos, so the transfer or storage of embryos with an incorrect number of chromosomes can be avoided, as those typically lead to failed IVF cycles. Traditionally, PGT-A requires a biopsy of a developing embryo by creating an opening in the outer coating prior to removal and testing of a few cells. However, recent studies have shown that an embryo releases small amounts of DNA into the culture media in which it is growing, allowing the surrounding fluid to be genetically tested instead.

PerkinElmers PG-Seq Rapid Non-Invasive PGT-A kit is specifically designed for this type of sample, which enables embryos to remain fully intact. Leveraging the science behind PerkinElmers biopsy-based PG-Seq kit 2.0, the new non-invasive kit tests the spent embryo culture media to accurately detect aneuploidies, as well as structural rearrangements, including unbalanced translocations and segmental errors.

The kit is a modified version of the new PG-Seq Rapid kit, a three-hour sample preparation workflowless than half of the sample preparation time compared to the PG-Seq kit 2.0 workflow.

Data from a global network of 15 laboratories who have provided samples, shows it is possible to achieve more than 90% correlation between results of biopsied embryo and spent embryo culture media with the PG-Seq Rapid Non-Invasive PGT kit, said Masoud Toloue, Ph.D., vice president, Diagnostics, PerkinElmer. By eliminating the risks associated with performing a cell biopsy, PGT-A becomes more broadly accessible. IVF providers can significantly increase the likelihood of successful embryo transfers and reduce time to pregnancy.

From what weve observed so far, the results look excellent, and we are looking forward to further developing our non-invasive PGT program, said Manuel Viotti, PhD, senior scientist, Zouves Foundation for Reproductive Medicine, Zouves Fertility Center.

PerkinElmer will showcase the PG-Seq Rapid Non-Invasive PGT-A kit and other next-generation workflow solutions at the 75th annual American Society for Reproductive Medicine (ASRM) Scientific Congress, October 12-16, in Philadelphia.

For more information, please visit booth #901 at ASRM or click here.

About PerkinElmerPerkinElmer enables scientists, researchers and clinicians to address their most critical challenges across science and healthcare. With a mission focused on innovating for a healthier world, we deliver unique solutions to serve the diagnostics, life sciences, food and applied markets. We strategically partner with customers to enable earlier and more accurate insights supported by deep market knowledge and technical expertise. Our dedicated team of about 13,000 employees worldwide is passionate about helping customers work to create healthier families, improve the quality of life, and sustain the wellbeing and longevity of people globally. The Company reported revenue of approximately $2.8 billion in 2018, serves customers in more than 180 countries, and is a component of the S&P 500 index. Additional information is available through 1-877-PKI-NYSE, or at http://www.perkinelmer.com.

View post:
PerkinElmer Launches PG-Seq Rapid Non-Invasive Preimplantation Genetic Testing Kit as Alternative to IVF Embryo Biopsies - Business Wire

Updated: May 25, 2018:

JD Supra is a legal publishing service that connects experts and their content with broader audiences of professionals, journalists and associations.

This Privacy Policy describes how JD Supra, LLC ("JD Supra" or "we," "us," or "our") collects, uses and shares personal data collected from visitors to our website (located at http://www.jdsupra.com) (our "Website") who view only publicly-available content as well as subscribers to our services (such as our email digests or author tools)(our "Services"). By using our Website and registering for one of our Services, you are agreeing to the terms of this Privacy Policy.

Please note that if you subscribe to one of our Services, you can make choices about how we collect, use and share your information through our Privacy Center under the "My Account" dashboard (available if you are logged into your JD Supra account).

Registration Information. When you register with JD Supra for our Website and Services, either as an author or as a subscriber, you will be asked to provide identifying information to create your JD Supra account ("Registration Data"), such as your:

Other Information: We also collect other information you may voluntarily provide. This may include content you provide for publication. We may also receive your communications with others through our Website and Services (such as contacting an author through our Website) or communications directly with us (such as through email, feedback or other forms or social media). If you are a subscribed user, we will also collect your user preferences, such as the types of articles you would like to read.

Information from third parties (such as, from your employer or LinkedIn): We may also receive information about you from third party sources. For example, your employer may provide your information to us, such as in connection with an article submitted by your employer for publication. If you choose to use LinkedIn to subscribe to our Website and Services, we also collect information related to your LinkedIn account and profile.

Your interactions with our Website and Services: As is true of most websites, we gather certain information automatically. This information includes IP addresses, browser type, Internet service provider (ISP), referring/exit pages, operating system, date/time stamp and clickstream data. We use this information to analyze trends, to administer the Website and our Services, to improve the content and performance of our Website and Services, and to track users' movements around the site. We may also link this automatically-collected data to personal information, for example, to inform authors about who has read their articles. Some of this data is collected through information sent by your web browser. We also use cookies and other tracking technologies to collect this information. To learn more about cookies and other tracking technologies that JD Supra may use on our Website and Services please see our "Cookies Guide" page.

We use the information and data we collect principally in order to provide our Website and Services. More specifically, we may use your personal information to:

JD Supra takes reasonable and appropriate precautions to insure that user information is protected from loss, misuse and unauthorized access, disclosure, alteration and destruction. We restrict access to user information to those individuals who reasonably need access to perform their job functions, such as our third party email service, customer service personnel and technical staff. You should keep in mind that no Internet transmission is ever 100% secure or error-free. Where you use log-in credentials (usernames, passwords) on our Website, please remember that it is your responsibility to safeguard them. If you believe that your log-in credentials have been compromised, please contact us at privacy@jdsupra.com.

Our Website and Services are not directed at children under the age of 16 and we do not knowingly collect personal information from children under the age of 16 through our Website and/or Services. If you have reason to believe that a child under the age of 16 has provided personal information to us, please contact us, and we will endeavor to delete that information from our databases.

Our Website and Services may contain links to other websites. The operators of such other websites may collect information about you, including through cookies or other technologies. If you are using our Website or Services and click a link to another site, you will leave our Website and this Policy will not apply to your use of and activity on those other sites. We encourage you to read the legal notices posted on those sites, including their privacy policies. We are not responsible for the data collection and use practices of such other sites. This Policy applies solely to the information collected in connection with your use of our Website and Services and does not apply to any practices conducted offline or in connection with any other websites.

JD Supra's principal place of business is in the United States. By subscribing to our website, you expressly consent to your information being processed in the United States.

You can make a request to exercise any of these rights by emailing us at privacy@jdsupra.com or by writing to us at:

You can also manage your profile and subscriptions through our Privacy Center under the "My Account" dashboard.

We will make all practical efforts to respect your wishes. There may be times, however, where we are not able to fulfill your request, for example, if applicable law prohibits our compliance. Please note that JD Supra does not use "automatic decision making" or "profiling" as those terms are defined in the GDPR.

Pursuant to Section 1798.83 of the California Civil Code, our customers who are California residents have the right to request certain information regarding our disclosure of personal information to third parties for their direct marketing purposes.

You can make a request for this information by emailing us at privacy@jdsupra.com or by writing to us at:

Some browsers have incorporated a Do Not Track (DNT) feature. These features, when turned on, send a signal that you prefer that the website you are visiting not collect and use data regarding your online searching and browsing activities. As there is not yet a common understanding on how to interpret the DNT signal, we currently do not respond to DNT signals on our site.

For non-EU/Swiss residents, if you would like to know what personal information we have about you, you can send an e-mail to privacy@jdsupra.com. We will be in contact with you (by mail or otherwise) to verify your identity and provide you the information you request. We will respond within 30 days to your request for access to your personal information. In some cases, we may not be able to remove your personal information, in which case we will let you know if we are unable to do so and why. If you would like to correct or update your personal information, you can manage your profile and subscriptions through our Privacy Center under the "My Account" dashboard. If you would like to delete your account or remove your information from our Website and Services, send an e-mail to privacy@jdsupra.com.

We reserve the right to change this Privacy Policy at any time. Please refer to the date at the top of this page to determine when this Policy was last revised. Any changes to our Privacy Policy will become effective upon posting of the revised policy on the Website. By continuing to use our Website and Services following such changes, you will be deemed to have agreed to such changes.

If you have any questions about this Privacy Policy, the practices of this site, your dealings with our Website or Services, or if you would like to change any of the information you have provided to us, please contact us at: privacy@jdsupra.com.

As with many websites, JD Supra's website (located at http://www.jdsupra.com) (our "Website") and our services (such as our email article digests)(our "Services") use a standard technology called a "cookie" and other similar technologies (such as, pixels and web beacons), which are small data files that are transferred to your computer when you use our Website and Services. These technologies automatically identify your browser whenever you interact with our Website and Services.

We use cookies and other tracking technologies to:

There are different types of cookies and other technologies used our Website, notably:

JD Supra Cookies. We place our own cookies on your computer to track certain information about you while you are using our Website and Services. For example, we place a session cookie on your computer each time you visit our Website. We use these cookies to allow you to log-in to your subscriber account. In addition, through these cookies we are able to collect information about how you use the Website, including what browser you may be using, your IP address, and the URL address you came from upon visiting our Website and the URL you next visit (even if those URLs are not on our Website). We also utilize email web beacons to monitor whether our emails are being delivered and read. We also use these tools to help deliver reader analytics to our authors to give them insight into their readership and help them to improve their content, so that it is most useful for our users.

Analytics/Performance Cookies. JD Supra also uses the following analytic tools to help us analyze the performance of our Website and Services as well as how visitors use our Website and Services:

Facebook, Twitter and other Social Network Cookies. Our content pages allow you to share content appearing on our Website and Services to your social media accounts through the "Like," "Tweet," or similar buttons displayed on such pages. To accomplish this Service, we embed code that such third party social networks provide and that we do not control. These buttons know that you are logged in to your social network account and therefore such social networks could also know that you are viewing the JD Supra Website.

If you would like to change how a browser uses cookies, including blocking or deleting cookies from the JD Supra Website and Services you can do so by changing the settings in your web browser. To control cookies, most browsers allow you to either accept or reject all cookies, only accept certain types of cookies, or prompt you every time a site wishes to save a cookie. It's also easy to delete cookies that are already saved on your device by a browser.

The processes for controlling and deleting cookies vary depending on which browser you use. To find out how to do so with a particular browser, you can use your browser's "Help" function or alternatively, you can visit http://www.aboutcookies.org which explains, step-by-step, how to control and delete cookies in most browsers.

We may update this cookie policy and our Privacy Policy from time-to-time, particularly as technology changes. You can always check this page for the latest version. We may also notify you of changes to our privacy policy by email.

If you have any questions about how we use cookies and other tracking technologies, please contact us at: privacy@jdsupra.com.

See the original post here:
Investigations Newsletter: Genetic Testing Company and Three Principals Settle FCA Allegations for $42.6 Million - JD Supra

The child was critically ill. The treating team at Childrens National Hospital in Washington, DC, was stumped and worried that time was running out. Every test was coming back negative.

Genetics was called in to look for chromosomal mutations that might suggest the source of the problems. The geneticist recommended whole-exome sequencing, which tells a story based not only on all of the childs genes, but on two additional sources as well: the mothers and the fathers genes.

They found something they werent looking for. The father, the worried man in the waiting room who raised this child, wasnt the biological father. In genomics its called an incidental finding, and it raises huge ethical questions: Do you reveal this to the parents? Only to the mother? Or, if the results dont affect the childs care, do you even tell anyone?

In this case, the team called on the hospitals ethics committee for help.

I think withholding information can feel paternalistic, [genetic counselor Monisha Samanta] Kisling says. We dont want to say, Hey, I dont think you can handle this information. Thats not necessarily our judgment call to make. Overall, its just a really, really tough decision.

Read full, original post: Youre Not the Father: A Moral Dilemma in Genetic Testing

Read more:
'Really tough decision': What should doctors do when genetic testing reveals that dad isn't the child's biological father? - Genetic Literacy Project

Since the 1970s, genetic screening has been standard practice during pregnancy. The goal of this kind of genetic testing has historically been to understand the likelihood an embryo carries a heritable disease, the classic example being the recessive genes for autosomal recessive disorders like cystic fibrosis, sickle cell anemia, and Tay Sachs, or tests that determine developmental disorders like Down Syndrome. This allows parents to make informed choices about ending pregnancies and planning ahead for potential issues. But as genetic technologies develop and gene editing becomes more widely practiced the possibilities are not limited to observation. As Dr. Robert Klitzsman writes inDesigning Babies: How Technology is Changing the Way We Create Children, genetic testing in the context of IVF is already giving us a glimpse of the ways in which the proliferation of new technologies could affect the genome of future children and future generations. And its not all good news.

Klitzman, Director ofColumbia Universitys Masters of Bioethics Program, suggests that progress has not been accompanied by adequate oversight or regulation. What are the limits of genetic screening, genetic testing, and gene editing technologies? The answer has as much to do with what risks are deemed acceptable and what risks are not. Designer babies are no longer the stuff of science fiction, but would-be parents arent exactly in a DNA Build-a-Bear. A transition is underway and Klitzman believes that parents need to understand the bleeding edge of whats possible to have the necessary context to understand the technologies they are using even in the process of basic screenings.

Fatherly spoke to Dr. Klitzman about the current state of genetic testing and the potential risks of emerging technologies.

Expecting parents likely understand one side of genetic testing, screening for heritable diseases. But new technologies and new discoveries mean that we can do more with genetic information and with genes themselves than ever before. So what are the limits of the current technologies?

A few years ago people thought wed find the cancer gene or the fat gene. But now we know that for most common diseases and most complex traits there are many genes involved. Sure there are certain genes that increase the risk of cancer from, say, 5 or 10 percent. But in genetic screening for diseases people should realize that, for a lot of diseases, the world is more complicated than just screening an embryo.

So its the old debate: nature or nurture? The answer is both. For a lot of traits, genetics explains the part but not all of the risk of the disease. So you may undergo genetic testing or you may screen embryos and the child may still get certain diseases. So its not always foolproof.

But its better than nothing.

Its important for parents to get tested to see if they have recessive conditions particularly if its in their family. If anyone has cystic fibrosis in their family, they should be tested to know. If theyre a carrier, they should see if their spouse is a carrier. If anyone has breast cancer in their family, they should be tested to see if they have that mutation. I think people with sickle cell disease should be tested for that. I think any woman who is over 35 should have the embryo tested for down syndrome and other chromosomal abnormalities. So I think there are certain diseases.

But, through evolution, most diseases for which there is a very predictive genetic test tend to be rare. If there was a terrible gene that was wiping out people, it wouldnt be getting passed on. The only genes that get passed on are not going to be from really terrible mutations because they would kill the people and by and large they wouldnt have any kid.

Its interesting that you point out the limits of genetic testing technologies because youre also a strong advocate for increased access.

I think insurance should pay for genetic testing. If a couple is concerned because their cousin has cystic fibrosis or someone in their family has sickle cell disease and wants to get tested, that should be covered. It may not be covered now so I think thats another set of policies that need to change. And part of that I think there needs to be more genetic counseling, which insurance also doesnt cover. The laws havent kept up with the technology. Our technologies advanced way ahead of our legal system and our duty to understand and figure out what to do with that of ethical legal and social questions involved.

A lot of the thornier issues you write about involve preimplantation genetic diagnosis, which is genetic testing post-conception and prior to pregnancy in the context of IVF. How are the decisions made by would-be parents undergoing PGD different than the decisions being made in the context of normal genetic testing?

Right now, we genetically screen embryos. When a couple undergoes IVF, lets say they create eight embryos. Doctors could say, These four are the girls, these four are the boys. Now, lets say a family has a history of breast cancer or the mother has the BRCA gene which carries breast cancer. The doctors could say, These three embryos have breast cancer gene these five dont. And the couple can choose the ones that dont.

Also, increasingly couples can say, Well, I just want a boy. And that creates a number of ethical challenges as opposed to letting Mother Nature do whatever it would do.

So theres potential in that specific context to take action in a way thats not possible in typical screenings. How much of that action is embryo selection versus actual gene manipulation?

We can take genes out. Theres a gene associated with Huntingtons disease or the BRCA breast cancer gene. We now have the technology to take them out.But these technologies are still in an experimental phase. And Im concerned that theyll soon be made fairly widely available even though there still may be risks involved and people may or may not fully appreciate those risks.

A few years ago, a doctor in China used CRISPR to edit the genes of twin girls. That opened brought a lot of criticism but also raised awareness of what can be done with this technology.

Thats right. So what Dr. He Jiankui did is that he worked with fathers who had HIV. There was a concern that the father could potentially pass HIV onto the child. And so he took the embryo and disabled the CCR5 gene that is involved in letting HIV get into a cell. The problem is that when you disable that gene, the risk of getting HIV goes down but the risk of getting influenza getting in goes up as do other risks.

DNA consists of three billion molecules. Each of us is a shelf of books in an office that has three billion letters in them. Well, if you go in and rip out some letters, you want to make sure you rip out the right ones. And so it looks like Dr. He didnt do it so precisely. So in fact what he said he took out wasnt exactly what he took out. In other words, if a child is born and missing part of the DNA, that part might be the next gene thats involved for, say, brain development or something like that.

You need to be very, very careful.

Presumably, the ethical questions get more complicated when gene editing becomes a more widely available procedure.

Until 60 years ago, we didnt even know what DNA did. We now have the ability to identify genes and were increasingly finding genes that are associated with not only various diseases but also human traits those associated with blonde hair and blue eyes, those associated with height and perfect pitch.

I think CRISPR probably will be used for people wanting or not wanting certain socially desirable or undesirable traits in their children.

A Gattaca situation.

Yes, exactly.

This interview has been condensed and edited for clarity.

The post Gene Editing Is Creating a New World of Designer Babies. Are We Ready For It? appeared first on Fatherly.

Read the rest here:
Gene Editing Is Creating a New World of Designer Babies. Are We Ready? - Yahoo Lifestyle

Genetic testing has become a hot topic with patients, families, health care providers and many others within the inherited retinal disease (IRD) community. Eye Want 2 Know brought to you by Spark Therapeutics, is pleased to bring you a special educational evening featuring genetic testing stories and experiences as told from the patient, advocate and health care professional perspectives.

Join the IRD community at this limited-seating event on World Sight Day, October 10 in San Francisco to hear first-hand genetic testing accounts, including:

Danelle Umstead, blind Paralympian and former Dancing with the Stars contestant

Elias Traboulsi, MD, M.Ed., Head of the Department of Pediatric Ophthalmology, Director of the Center for Genetic Eye Diseases at Cleveland Clinic's Cole Eye Institute

Chris Moen, MD, Chief Medical Officer of the Choroideremia Research Foundation

Meghan DeBenedictis, MS, LGC, M.Ed., Licensed, Certified Genetic Counselor and Coordinator at Cleveland Clinic's Cole Eye Institute

WHEN: Thursday, October 10, 5:00PM-7:00PM

WHERE: Town Hall at Galvanize, San Francisco

Genetic testing has become a hot topic with patients, families, health care providers and many others within the inherited retinal disease (IRD) community. Eye Want 2 Know brought to you by Spark Therapeutics, is pleased to bring you a special educational evening featuring genetic testing stories and experiences as told from the patient, advocate and health care professional perspectives.

Join the IRD community at this limited-seating event on World Sight Day, October 10 in San Francisco to hear first-hand genetic testing accounts, including:

Danelle Umstead, blind Paralympian and former Dancing with the Stars contestant

Elias Traboulsi, MD, M.Ed., Head of the Department of Pediatric Ophthalmology, Director of the Center for Genetic Eye Diseases at Cleveland Clinic's Cole Eye Institute

Chris Moen, MD, Chief Medical Officer of the Choroideremia Research Foundation

Meghan DeBenedictis, MS, LGC, M.Ed., Licensed, Certified Genetic Counselor and Coordinator at Cleveland Clinic's Cole Eye Institute

WHEN: Thursday, October 10, 5:00PM-7:00PM

WHERE: Town Hall at Galvanize, San Francisco

Read more here:
Beyond the Science: Perspectives on Genetic Testing for IRDs - NBC Bay Area

New guidelines for genetic testing should help more women and their relatives take steps to improve their odds against cancer that might run in the family.

All women who have had cancer of the breast, ovary, Fallopian tubes or peritoneum tissue in the walls of the abdomen should be offered a screening tool to determine their risk of mutation, new U.S. Preventive Services Task Force guidelines say.

The new recommendation removes a wording that limited testing to those diagnosed at younger ages.

Thats a pretty big thing, genetic counselor Kimberly Knapp said at Joyce Murtha Breast Care Center in Windber.

In the past, screening for breast cancer susceptibility genes BRCA1 and BRCA2 was recommended for women who developed breast or ovarian cancer before age 50 and others who had certain types of cancer before age 60.

Now, we dont have to age discriminate, Knapp said.

Anyone with a significant family history of breast or ovarian cancer should also consider genetic screening, which begins with a thorough review of family cancer history with a trained counselor, Knapp said.

Those women can contact breast cancer programs at several of the regions hospitals to find out more about their risk.

Genetic counselors will be able to help determine if they are eligible for genetic testing for the BRCA1 or BRCA2 gene mutations.

BRCA is shorthand for breast cancer, but the mutations are also associated with an increased risk of other cancers.

Genetic testing at Joyce Murtha Breast Care Center at Chan Soon-Shiong Medical Center at Windber tests for the BRCA gene, Knapp said.

Because researchers continually find more genetic links, Windber offers a genetic screening for a panel of almost 50 different mutations.

The new guidelines expand the pool of patients who are eligible for insurance coverage of the test, she said.

At Indiana Regional Medical Center, breast surgeon and genetic counselor Dr. Dan Clark works with families to screen for 24 gene mutations.

Screening is not just a yes or no. You get a report with four pages of results, he said at the hospital.

The report may recommend additional screenings for hereditary cancers, including breast, ovarian and colon cancer.

We know there are a lot of other cancers associated with genetics, he said.

But most cancer is not hereditary, he warned.

The American Cancer Society estimates between 5 percent and 10 percent of all cancers come from gene mutations passed on through families.

Clark fears heightened attention on genetic risk may cause women with breast cancer in their families to think they are not at risk, noting most breast cancer patients have no family history of the disease.

Link:
Genetic testing expanded to help those with disease in their families - Oskaloosa Herald

Von Willebrand disease (VWD) is the most common hereditary bleeding disorder but one of the most difficult to diagnose, especially type 1 VWD. Recurrent challenges include the need to complete several assays of von Willebrand factor (VWF) activity and lack of consensus surrounding the acceptable standard for diagnosis. Consequently, improving current diagnostic techniques, as well as implementing new methods, is essential to ensure patients are provided optimal care.

In a review article published in Current Opinion in Hematology, Veronica H Flood, MD, of the department of pediatrics at the Medical College of Wisconsin in Milwaukee, and colleagues summarized the current literature surrounding the diagnosis of type 1 VWD. They also reviewed new advances in genetic testing for VWF, which could serve as a potential alternative to conventional laboratory methods.

Overview of Genetic Dysfunction

In contrast to type 2 VWD, type 1 VWD may include genetic defects in the coding region of the VWF gene. These mutations vary from insertions and deletions to point mutations that produce missense or nonsense mutations. With conventional sequencing methods, insertions and deletions can be missed, which has historically precluded the clinical use of genetic-based diagnostic techniques. These limitations are not typically seen in type 2 VWD as genetic defects are usually present in the DNA region specific to the impacted functional region.

Because of the high degree of polymorphism seen in the VWF gene, entire genome or exome sequencing may be required for diagnosis; in other instances, the VWF gene may be analyzed directly if a particular coagulation defect is suspected. In type 1 VWD, certain high frequency variants have been linked to disease etiology, but recent data have highlighted the potential role of novel variants in type 1 VWD. The high degree of variability seen in the VWF gene is certainly a key contributor to the disease phenotype, but not all defects will ultimately lead to VWD.

Modifier Genes and Diagnosis

In addition to defects in VWF, several genes independent of the VWF locus have been shown to affect VWF levels. The most described modifier gene is ABO, though others such as CLEC4M, STAB2, and STXBP5 also exist. Blood group O levels of less than 50 IU/dL are routinely used to establish a diagnosis of VWD, but some individuals with blood type O also meet this criteria despite being healthy. Some experts have proposed that low VWF may be more suitably described as a risk factor for bleeding instead of as the basis for bleeding.

Read more:
Advances in the Diagnosis of Type 1 von Willebrand Disease: Genetic Testing - Hematology Advisor

The globalmarket for testosterone replacement therapyis characterized by the presence of a large number of small and large scale manufacturers. All of the manufacturers have been steadfast in filling the meagre market gap in order to enhance their prospects of growth. Furthermore, research and development has been the central characteristic of al the market players operating in the global market.

In 2015, it was found that 80% of the total market share was held by the top five market vendors with AbbVie Inc. taking the lead. The large scale vendors are focusing on establishing an iconic brand for their product by resorting to rigorous marketing and advertising tactics. The smaller companies are expected to concentrate on capturing the local and regional markets to sustain themselves in the current scenario of stiff competition.

A negative implication for the leading market players in recent times has been the loss of patents for their products. This has not only plundered them of revenues but has also affected the workflow of these companies. The market players are expected to launch awareness campaigns about testosterone replacement therapies in order to educate and inform the consumers. Hence, the market for testosterone replacement therapies is expected to witness the emergence of several new trends and opportunities over the forthcoming years. Some of the key players in the global testosterone replacement therapy market include Bayer AG, Endo Pharmaceuticals, Inc., Novartis AG, and Allergen plc.

Get Sample with Latest Research @https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=1097

The CAGR for theglobal testosterone replacement therapy marketis estimated to be -4.20% over the period between 2016 and 2024. The negative growth rate of the global market is expected to take the market value from US2.0 bn in 2015 to a decreased value of US$1.3 bn by 2024-end.

High Incidence of Hypogonadism to Drive Market Demand

Research studies suggest that around 30% of all men suffer from testosterone deficiency, which has driven demand within the global market for testosterone replacement therapy. Furthermore, the population demographic of men in the age range of 40-79 years is more likely to suffer from testosterone deficiency. The need for mutation or having an offspring amongst men in the aforementioned age range has driven demand within the global market. Moreover, the geriatric population has been on a rise, which underhandedly contributes to market growth. Several campaigns aimed at educating people about the benefits of testosterone replacement therapy have been an important propeller of demand within the global market. It is anticipated that more people suffering from testosterone deficiency would resort to these therapies over the coming years.

Enquiry for Discount on this Report @https://www.transparencymarketresearch.com/sample/sample.php?flag=D&rep_id=1097

Side Effects of Testosterone Replacement Therapy Could Obstruct Market Growth

Despite the rising awareness amongst the masses about the advantages of testosterone replacement therapies, the market growth is hindered by the apprehension of the people. The chances of developing metabolic disorders are higher in men who undergo testosterone replacement therapies. Furthermore, the risk of developing cardiovascular diseases also discourages people from resorting to testosterone replacement therapies. The FDA has also cautioned people about the use of such therapies by issuing strict warnings, which has further obstructed the growth of the global market.

See original here:
Testosterone Replacement Therapy Market: An Insight on the Important Factors and Trends Influencing the Market - Online News Guru

An individuals experiences during early life can influence their risk for disease as an adult. Dr. David Barker first postulated this in the 1980s following his observations that infants born with low birth weight were at significantly increased risk for developing cardiovascular disease as adults. This idea was controversial at the time because it challenged the conventional thinking that unhealthy lifestyle was the primary cause of cardiovascular disease. However, subsequent epidemiologic studies confirmed Barkers findings and the concept that early-life experiences are an important determinant of adult disease risk became known as the Barker hypothesis.

While the Barker hypothesis was derived from evidence linking foetal malnutrition to cardiovascular disease risk in adulthood, the concept has since expanded to include a broader spectrum of risk factors, diverse disease states targeting different organ systems, and developmental periods extending beyond gestation. In recognition of this paradigm shift, the Barker hypothesis was renamed the developmental origins of health and disease (DOHaD) hypothesis. Several decades of DOHaD research have identified at least four categories of early-life risk factors: the nutritional status of the pregnant woman and young child, psychosocial stress, immunological stress, particularly infection during pregnancy, and chemical exposures. It is now also appreciated that the risk of most, if not all, adult-onset diseases are influenced by early-life stress, but only if exposures occur during specific periods of development. These high-risk periods are referred to as critical periods or critical windows of development, and these vary depending on the early-life insult and the target organ. For many organ systems, however, the foetal period is the primary critical window.

The nervous system is particularly vulnerable to early-life stressors, perhaps because human brain development continues after birth, throughout early childhood and into adolescence. To date, research supports an association between early-life environmental insults and increased risk of neuropsychiatric disorders like schizophrenia, demyelinating diseases, such as multiple sclerosis (MS), and neurodegenerative diseases, such as Alzheimers and Parkinsons diseases (AD and PD, respectively). Whether an early-life stressor increases an individuals risk for neuropsychiatric or neurologic disease depends on numerous factors. What is the type and magnitude of the stressor? Is the insult continuous, intermittent, or a single isolated incident? What is the timing of exposure relative to critical windows of development, which vary across different brain regions? Does the affected individual carry genes that confer increased susceptibility or increased resistance to the adverse effects of the stressor? Sex also influences the impacts of early-life stressors, perhaps because gonadal sex hormones influence many of the organisational aspects of neurodevelopment. Examining the sex-specificity of early-life stressors may be important for understanding why many neurological and neuropsychiatric diseases exhibit a sex bias in their incidence and clinical profile.

Maternal infection during pregnancy is strongly linked to increased risk of schizophrenia, though it does not appear that any specific pathogen or class of pathogens is responsible. While there is disagreement as to whether a specific trimester of pregnancy is most vulnerable, the pro-inflammatory environment created by maternal infection is thought to alter the pattern of cellular connections made in the foetal brain, laying the groundwork for altered behavior in adulthood. Emerging evidence also suggests that early-life exposure to the lead (Pb) may increase risk for schizophrenia, particularly in individuals that express a mutation in the disrupted in schizophrenia 1 (DISC-1) gene, a gene that is strongly associated with schizophrenia and related mental disorders. AD is the most common cause of progressive dementia in elderly adults and it is rapidly increasing in global incidence; PD is the second most common neurodegenerative disease after AD. Only a small percentage of cases of either disease can be attributed to solely genetic causes, supporting a role for environmental factors in determining individual risk. Recent studies have identified early-life risk factors for AD and PD, including low birth weight, premature birth, living in a rural area, low socioeconomic status during childhood, and prenatal or early childhood exposures to environmental pollutants, including heavy metals, pesticides, and air pollution. Air pollution has also been linked to an increased risk of MS.

A key question in the field is how do early-life events influence risk of adult-onset disease? It is believed that environmental stressors disrupt the organisational patterning and/or function of the developing brain by altering cell numbers or interfering with the differentiation of neurons or glial cells. So why do these changes not manifest as functional deficits or disease until adulthood? One explanation is that the affected cells are not functional until later in life. For example, exposure of the developing brain to high concentrations of the food additive monosodium glutamate (MSG) causes excessive death of neurons in the hypothalamus by triggering apoptosis, a form of programmed cell death. However, functional deficits associated with the foetal loss of these hypothalamic neurons (hypogonadism and infertility) become evident only in adolescence when the neuroendocrine function of these neurons is normally activated. Alternatively, the adverse impacts of the early-life stressor are masked or initially attenuated due to compensatory mechanisms or plasticity of the brain. However, these developmental perturbations predispose the individual to neural deficits following subsequent insults, such as chemical exposure, disease, or aging due to decreased brain reserve capacity. This phenomenon has been demonstrated in both animal models and humans following developmental exposures to methyl mercury or pesticides.

In summary, the experimental evidence indicates that early-life insults can fundamentally change the trajectory of brain development, thereby diminishing the ability of the brain to protect against subsequent insults, which increases susceptibility to disease in adulthood. A significant challenge in the field is to identify early-life stressors that increase adult-onset disease in humans. Detecting effects in the human population is difficult because the effects do not manifest until well after the developmental exposure. However, the effort to identify these associations merits investment of research dollars because preventing disease by identifying and reducing or eliminating risk factors is more effective than treating disease in terms of both individual and societal costs.

Please note: This is a commercial profile

Editor's Recommended Articles

Read more here:
Environmental exposures during early life influence adult disease risk - Open Access Government

The report provides a basic overview of the Androgen Replacement Therapy industry including its definition, applications and manufacturing technology.with leading business players.

Androgen replacement therapy, often referred to as testosterone replacement therapy, is a form of hormone therapy in which androgens, often testosterone, are replaced. ART is often prescribed to counter the effects of male hypogonadism. Rising aging male population with disease and increasing awareness about disease and therapy benefits dominating the market.

Augmented demand for the global Androgen Replacement Therapy market has been increased in the last few years. This informative research report has been scrutinized by using primary and secondary research.

Get a Sample Copy of Global Androgen Replacement Therapy Market Report @ https://inforgrowth.com/sample-request/4914309/androgen-replacement-therapy-market

Top Key Players: AbbVie, Endo International, Eli lilly, Pfizer, Actavis (Allergan), Bayer, Novartis, Teva, Mylan, Upsher-Smith, Ferring Pharmaceuticals, Kyowa Kirin, Acerus Pharmaceuticals

The Global Androgen Replacement Therapy Market Report represents highly detailed data including recent trends, Market demands, supply and distribution chain management strategies which will help to identify the workflow of Global Androgen Replacement Therapy Market Industry.

Global Androgen Replacement Therapy Market Report provides critical and detailed data for investment plans with research and development budgets, raw material budgets, labor cost, and other funds. Global Androgen Replacement Therapy Market industry is large enough to build a sustainable business, so this report helps you to identify the opportunities in Global Androgen Replacement Therapy Market by region: North America, Europe, Asia-Pacific, North America and Middle East and Africa.

Any Other Query, Ask Our Experts @ https://inforgrowth.com/report/4914309/androgen-replacement-therapy-market#speakAnalyst

Androgen Replacement Therapy Market by Type: Gels, Injections, Patches, Others

Androgen Replacement Therapy Market by Application: Hospitals, Clinics, Others

Major highlights of the global research report:

Finally, the researchers throw light on the different dynamics of the market such as drivers, restraints, and opportunities. Additionally, it offers exhaustive information about new products, developments, and investment.

Key Content of Chapters

1:Market Overview, Development, and Segment by Type, Application and Region2:Company information, Sales, Cost, Margin etc.3:Global Market by company, Type, Application and Geography4:Asia-Pacific Market by Type, Application and Geography5:Europe Market by Type, Application and Geography6:North America Market by Type, Application and Geography7:South America Market by Type, Application and Geography8:Middle East and Africa Market by Type, Application and Geography9:Market Features10:Investment Opportunity11:Conclusion

If you have any special requirements, please let us know and we will offer you the report as you want.https://inforgrowth.com/customization/4914309/androgen-replacement-therapy-market

About InForGrowth:

We are a market-intelligence company formed with the objective of providing clients access to the most relevant and accurate research content for their growth needs. At InForGrowth, we understand Research requirements and help a client in taking informed business critical decisions. Given the complexities and interdependencies of market-intelligence, there is always more than one source to explore and arrive at the right answer. Through our smart search feature and our reliable andamp; trusted publishing partners, we are paving way for a more simplified and relevant research.

For all your Research needs, reach out to us at:

Address: 6400 Village Pkwy suite # 104, Dublin, CA 94568, USA

Contact Person: Rohan

Email:[emailprotected]

Phone: +1-909-329-2808

Press Release Distributed by The Express Wire

To view the original version on The Express Wire visit Androgen Replacement Therapy Industry Analysis, Business Opportunities and Future Forecast 2024 by Top Key Players

Original post:
Androgen Replacement Therapy Industry Analysis, Business Opportunities and Future Forecast 2024 by Top Key Players - Infonews Box

Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of agene therapy to prevent heart failure linked to Duchenne (DMD) and Becker muscular dystrophy (BMD), and another to create better measures of treatment responses in DMD clinical trials.

PPMD, a nonprofit organization leading the fight against DMD, awarded $1 million to the professor and researcher H. Lee Sweeney, PhD, and his team at theUniversity of Florida to continue developing gene therapies to address the causes of heart failure in Duchenne and Becker MD.

The grant is part of PPMDs Cardiac Initiative, which draws on contributions from theDuchenne community, as well as the support of other Duchenne families and foundations.

For people with Duchenne, cardiac disease is a great concern. The absence of dystrophin contributes to a progressive deterioration of the hearts muscle, leading to dilated cardiomyopathy (DCM).

In this disease, the muscle and chambers of the heart begin to dilate and can reach a point where the muscle cannot contract enough to pump blood well. As heart muscle weakens, heart failurecan occur.

The funding will support the development of a heart-specific gene therapy using viral vectors (AAV vectors) to deliver two genes engineered to correct calcium handling and prevent the malfunction of mitochondria (the cells energy powerhouses) in the heart of Duchenne and Becker patients.

Sweeney and his team have been studying what happens in the heart during DMD, and found there is a calcium overload straining the organ. They have been treating animal models with the potential gene therapy, so far with positive results.

If successful, the therapy could treat the heart in a way that is independent of, or complementary to, micro-dystrophin based gene therapy.

Heart issues dont just affect some people with Duchenne; they affect ALL people with Duchenne. And while we have improved cardiac care in Duchenne, we still need treatments that repair our childrens hearts, PPMDs president and CEO, Pat Furlong said in a press release. Furlong lost both of her sons to heart failure as a result from DMD.

Chris and Patrick died of heart failure, so the heart is at the center of Duchenne for me, Furlong added. Thats why I am extremely proud to announce this $1 millioninvestment into a gene therapy with the potential to heal the hearts of our loved ones. I am grateful to Dr. Sweeney and the amazing team atUniversity of Florida, as well as the families in our community who believe in our mission and gave generously to help fund the fight to end Duchenne.

PPMD is also partnering with Duchenne UK to fund a project to create a set of highly sensitive and validated patient-reported outcomes (PROs) for use in Europe and the U.S.

The$200,000 grant was awarded to Chad Heatwole, MD, MS, the projects leader and a neuromuscular clinician at theUniversity of Rochester. Its goalis to develop outcome measures for clinical trials that are able to better capture treatment benefits from a patient and caregiver perspective.

Building on their experience in developing health indices for other diseases, Heatwole and his team will conduct a series of interviews with patients from Europe and the U.S. to identify relevant symptoms, and develop a comprehensive set of patient-reported outcomes according to regulatory guidelines from the FDA (in the U.S.) and EMA (in Europe).

We believe that incorporating the voice of the patient through PROs is an extremely powerful tool to support and accelerate drug development. We are grateful to be working with the Duchenne UK team to develop tools that ensure patients are heard, Abby Bronson, PPMDs senior vice president for Research Strategy, said in a press release.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 307

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

Read more from the original source:
PPMD Grants to Promote Gene Therapy for Heart Disease and Patient Outcomes Research - Muscular Dystrophy News

DUBLIN--(BUSINESS WIRE)--The "Growth Opportunities in the Neurodegenerative Disorder Therapeutics Market, Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

As one lives longer, one needs to increasingly battle age-related disorders. Neurodegenerative disorders are the most worrisome because they impact millions of people around the world and lack any curative therapy. While companies continue their search, they are also battling declining revenues from marketed products used to manage symptoms. Heavy genericization, followed by price erosion, reliance on patient-reported data for diagnosis and measuring outcomes, and elusive R&D success, has put unprecedented pressure on pharma companies and prompted several to shed their assets mid-way.

This research service, in addition to quantifying the market, provides details of future products and the expected revenue generation from them. While the therapy market is marred by high rates of pipeline attrition, there are several parallel areas of growth. For instance, the study covers regenerative medicine, which has grown by leaps and bounds and offers the promise of curative therapies.

The study also dives deep into different technological advancements, geographical trends, and potential partnership opportunities for Alzheimer's and Parkinson's diseases. The study covers opportunities in prevention facilitated by digital solutions integration, in conjunction with an understanding of disease diagnosis and progression, expedited drug development, and, most importantly, delivery of the required outcome to the patient.

The study captures the competitive landscape and the different strategies employed by companies to stay ahead of the curve and identifies the game-changing companies leading innovation from the front. Acknowledging the high cost of failure, the study investigates the need and growing acceptance of open innovation to curate data, ask better questions, extract meaningful insights, and create more accurate and predictable solutions.

In addition to collaboration with peers, companies will seek partnership with digital partners. Given the growing availability of data, technologies and tools, and research expertise there are several companies seeking clinically validated solutions, which have been profiled in the study. The study lays down strategic imperatives for companies to recalibrate their business models based on collaboration and be future-ready.

Information is also provided on some of the leading M&A activities impacting the market, as well as unconventional collaboration agreements laying the foundation for propelling innovations toward licensure and delivery. Furthermore, present and future market trends such as regulatory support, focus on wellness, and value chain convergence, which would shape the market, are discussed.

Key Issues Addressed

Key Topics Covered:

1. Executive Dashboard

2. Market Overview and Dynamics

3. Forecast and Trends - Total Neurodegenerative Disorder Therapeutics Market

4. Alzheimer's Disease Segment Analysis

5. Parkinson's Disease Segment Analysis

6. Competitive Landscape - Total Neurodegenerative Disorder Therapeutics Market

7. Visioning Scenarios

8. Growth Opportunities

9. The Last Word

10. Appendix

Companies Mentioned

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

Continue reading here:
Global Neurodegenerative Disorder Therapeutics Market 2019-2024: Opportunities in Digital Biomarkers, Microbiome Therapeutics, Cell and Gene Therapy,...

Recently developed gene therapy seems to be a promising alternative for treating one of the most common causes of blindness, wet age-related macular degeneration (AMD), claims a study.

Data presented at the 123rd Annual Meeting of the American Academy of Ophthalmology showed that six patients with wet age-related macular degeneration (AMD) went at least six months without the need for continued injections to control a disease that typically requires treatment every four to six weeks.

Researchers said the hope is that gene therapy will free patients from nearly monthly eye injections by offering a potential "one-and-done" treatment. It's not just about convenience; a more consistent treatment may also help people keep more of their vision.

"This is potentially paradigm-shifting," said lead researcher, Szilard Kiss, M.D., director of Clinical Research and chief of the Retina Service in the Department of Ophthalmology at Weill Cornell Medical College in New York City.

"It's the next evolutionary leap in treating AMD. When you think about what science fiction is and what science reality is; gene therapy for AMD is becoming a clinical reality," added Dr Kiss.

AMD is a degenerative eye disease that happens when part of the retina is damaged. The damage happens when new, weak blood vessels form behind the retina at the back of the eye. These abnormal vessels leak, causing scarring and killing off the cells that allow us to see.

One main reason why is that patients are undertreated. This is because most people with AMD must go to the ophthalmologist's office every four to eight weeks for an injection directly into their eye (oftentimes in both eyes).

This can be a difficult schedule to maintain for many elderly patients struggling with other maladies and reliant on others to get them to their ophthalmologist visits. It's also unsustainable for the health care system.

Last year alone, ophthalmologists performed more than 8 million anti-VEGF injections in the United States.

Researchers have been searching for a better alternative to monthly injections almost from the moment anti-VEGFs were introduced. Gene therapy is emerging as one of the more promising alternatives to long-term anti-VEGF treatment.

The goal of Dr Kiss' research is to develop a gene therapy that allows the eye to make its own anti-VEGF medicine. The ideal gene therapy would be administered not through a surgical procedure in an operating room, but through an injection into the eye that can be done in the doctor's office, just like routine anti-VEGF treatment is done today.

To do this, Dr Kiss and colleagues have developed a next-generation vector that can insert into the cells of the eye, the genetic material that makes a molecule similar to a widely used anti-VEGF medicine called aflibercept. Once inside the cells, the DNA sequence begins making the aflibercept protein.

Go here to see the original:
Gene therapy effective for treating wet age-related macular degeneration: Study - DNA India

Using its brain mapping platform, Inscopix will team up with researchers at the Broad Institute of MIT and Harvard to investigate how changes in brain activity alter the functioning of nerve cells. The goal is to identify new therapeutic targets for Parkinsons disease.

Thecollaboration will be led by Evan Macosko, MD, PhD, of the Broad Institutes Macosko Lab, an expert in single-cell transcriptomics a next-generation sequencing approach that assesses how gene activity changes in a single cell.

This research builds on a previous study that used Inscopixs miniature microscope, called nVoke, which allows simultaneous imaging and manipulation of nerve cells circuit dynamics in real time. The technology allows researchers to image cell activity for months with single-cell resolution in a living animal.

The previous study, Diametric neural ensemble dynamics in parkinsonian and dyskinetic states, published in Nature, used nVoke to identify alterations in neural activity patterns in brain circuits that regulate movement in a Parkinsons mouse model. This model mimics the human disease by gradually losing dopaminergic neurons a hallmark of Parkinsons.

Dopaminergic neurons release the neurotransmitter dopamine a chemical substance produced in response to nerve signals that allow nerve cells to communicate. In Parkinsons disease, dopamine-producing neurons are mainly lost in a brain region known as the substantia nigra, which plays a key role in reward and movement.

Now, the researchers will investigate whether the observed changes in neuronal activity in the Parkinsons mouse model can be correlated to changes in the expression of genes detected in single cells, which may have occurred as a result of the loss of dopamine. Gene expression is the process by which information in a gene is synthesized to create a working product, like a protein.

By analyzing neuronal activity and gene activity, the researchers hope to gain further insights into the mechanisms of Parkinsons disease. This may allow the identification of new, cell-type specific therapeutic targets.

According to Inscopix, real-time mapping of neural activity in brain circuits has been shown to more accurately predict the efficacy of a therapy to work on the brain, when compared with analyzing animal behavior.

Single-cell transcriptomics and brain mapping have each demonstrated the potential to increase our understanding of neurological conditions, such as Parkinsons disease, and bringing them together could help us make even greater strides forward, Kunal Ghosh, CEO of Inscopix, said in a press release.

We look forward to expanding our research into PD [Parkinsons disease] with the Macosko Lab, with the goal of paving the way for therapeutic programs that can be de-risked at earlier stages of development, Ghosh added.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 208

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

See the rest here:
Real-time Brain Mapping Used to Search for Therapeutic Targets in PD - Parkinson's News Today

NEW YORK, Oct. 14, 2019 /PRNewswire/ -- According to the current analysis of Reports and Data, the Global Gene Expression market is expected to reach USD 11.37 billion by the year 2026, in terms of value at a CAGR of 8.1% from 2019-2026. Gene expression promises to tap into a previously unexplored segment in the vast and burgeoning genetic engineering industry. Gene expression is the process by which the genetic code - the nucleotide sequence - of a gene is used to direct protein synthesis and produce the structures of a cell. It is the process by which instructions in the DNA are converted into a functional product like protein. The commercial applications of gene expression have been studied and researched upon extensively in recent years. Many diverse and wide ranging applications have been found for this novel technique. With the increased availability and lowering costs of DNA technologies, gene expression has become a more readily used tool indispensable in drug discovery and development.

Increase in investments in the market, which are supporting the technological advancements, and rise in healthcare expenditure are estimated to shape the growth of the gene expression market. Drug discovery & development and increase in demand for personalized medicine in chronic diseases such as cancer will be observed as the most lucrative applications for gene expression analysis in the forecast period. Application of gene expression in clinical diagnostics, on the other hand, will reflect a moderate growth throughout the analysis period. Moreover, the falling costs of sequencing have facilitated the integration of genomic sequencing into medicine. With the increased availability and lowering costs of DNA technologies, gene expression has become a more readily used tool indispensable in drug discovery and development. Many companies and educational institutions are collaborating to make gene expression publicly accessible through databases such as the Connectivity Map (CMap), Library of Integrated Network-based Cellular Signatures (LINCS) and the Tox 21 project.

New product development has been the consistent strategy undertaken by majority of the players to expand their product portfolio for serving a larger consumer base. For example, in September 2019, Qiagen N.V., launched the newly enhanced GeneGlobe Design & Analysis Hub, which integrates the company's manually curated knowledge base on over 10,000 biological entities with the industry's most comprehensive portfolio of tools for next-generation sequencing (NGS), polymerase chain reaction (PCR) and functional analysis. Other companies like Thermo Fisher Scientific and Illumina Inc. have launched new products in the last few months which are being used in the gene expression market.

Request free sample of this research report at: https://www.reportsanddata.com/sample-enquiry-form/1474

Further key findings from the report suggest

To identify the key trends in the industry, click on the link below: https://www.reportsanddata.com/report-detail/gene-expression-market

Segments covered in the report:

For the purpose of the study, this Reports and Data has segmented the Gene Expression Market on the basis of product type, platform type, prescription mode, end user and the regional outlook

Product and Services (Revenue, USD Million; 20162026)

Capacity (Revenue, USD Million; 20162026)

Application (Revenue, USD Million; 20162026)

Order Now: https://www.reportsanddata.com/checkout-form/1474

Regional Outlook: (Revenue, USD Million; 20162026)

Browse more similar reports on Biotechnology category by Reports And Data

About Reports and Data

Reports and Data is a market research and consulting company that provides syndicated research reports, customized research reports, and consulting services. Our solutions purely focus on your purpose to locate, target and analyze consumer behavior shifts across demographics, across industries and help client's make a smarter business decision. We offer market intelligence studies ensuring relevant and fact-based research across a multiple industries including Healthcare, Technology, Chemicals, Power, and Energy. We consistently update our research offerings to ensure our clients are aware about the latest trends existent in the market. Reports and Data has a strong base of experienced analysts from varied areas of expertise.

Contact: John W Head of Business Development Reports And Data | Web: http://www.reportsanddata.com Direct Line: +1-212-710-1370 E-mail: sales@reportsanddata.com

SOURCE Reports And Data

Read the original post:
Gene Expression Market to Reach USD 11.37 Billion by 2026 | Reports and Data - PRNewswire

Mart Research new study, Global Gene Therapy MarketReport cover definite aggressive standpoint including the piece of the overall industry & profiles of the key members working in the worldwide market.

The global Gene Therapy market will reach Volume Million USD in 2019 and with a CAGR xx% between 2020-2026.

Gene Therapy Product Type Coverage (Market Size & Forecast, Major Company of Product Type etc.):

Ex vivo

In vivo

Get a free sample report: https://martresearch.com/contact/request-sample/1/83572

Gene Therapy Demand Coverage (Market Size & Forecast, Consumer Distribution):

Cancer Diseases

Monogenic Diseases

Infectious Diseases

Cardiovascular Diseases

Others

Gene Therapy Company Coverage (Sales data, Main Products & Services etc.):

Sangamo

Spark Therapeutics

Dimension Therapeutics

Avalanche Bio

Celladon

Vical

Advantagene

Gene Therapy Major Region Market

North America

Europe

Asia-Pacific

South America

Middle East & Africa

Place the Order of Global Gene Therapy Market Research Report: https://martresearch.com/paymentform/1/83572/Single_User

Some Points from Table of Contents:

Chapter 1 Industry Overview

1.1 Gene Therapy Industry

1.1.1 Overview

1.1.2 Products of Major Companies

1.2 Market Segment

1.2.1 Industry Chain

1.2.2 Consumer Distribution

1.3 Price & Cost Overview

Chapter 2 Gene Therapy Market by Type

2.1 By Type

2.1.1 Ex vivo

2.1.2 In vivo

2.2 Market Size by Type

2.3 Market Forecast by Type

Chapter 3 Global Market Demand

3.1 Segment Overview

3.1.1 Cancer Diseases

3.1.2 Monogenic Diseases

3.1.3 Infectious Diseases

3.1.4 Cardiovascular Diseases

3.1.5 Others

3.2 Market Size by Demand

3.3 Market Forecast by Demand

Chapter 4 Major Region Market

4.1 Global Market Overview

4.1.1 Market Size & Growth

4.1.2 Market Forecast

4.2 Major Region

4.2.1 Market Size & Growth

4.2.2 Market Forecast

Chapter 5 Major Companies List

5.1 Sangamo (Company Profile, Sales Data etc.)

5.2 Spark Therapeutics (Company Profile, Sales Data etc.)

5.3 Dimension Therapeutics (Company Profile, Sales Data etc.)

5.4 Avalanche Bio (Company Profile, Sales Data etc.)

5.5 Celladon (Company Profile, Sales Data etc.)

5.6 Vical (Company Profile, Sales Data etc.)

5.7 Advantagene (Company Profile, Sales Data etc.)

Chapter 6 Conclusion

For more Information or Any Query Visit: https://martresearch.com/contact/enquiry/1/83572

List of Table

Table Global Gene Therapy Market 2016-2019, by Type, in USD Million

Table Global Gene Therapy Market 2016-2019, by Type, in Volume

Table Global Gene Therapy Market Forecast 2020-2026, by Type, in USD Million

Table Global Gene Therapy Market Forecast 2020-2026, by Type, in Volume

Table Global Gene Therapy Demand 2016-2019, in USD Million

Table Global Gene Therapy Demand 2016-2019, in Volume

Table Global Gene Therapy Demand Forecast 2020-2026, in USD Million

Table Global Gene Therapy Demand Forecast 2020-2026, in Volume

Table Global Gene Therapy Market Size & Growth 2016-2019, in USD Million

Table Global Gene Therapy Market Size & Growth 2016-2019, in Volume

Table Global Gene Therapy Market Forecast 2020-2026, in USD Million

Table Global Gene Therapy Market Forecast 2020-2026, in Volume

Table Global Gene Therapy Market 2016-2019, by Region, in USD Million

Table Global Gene Therapy Market 2016-2019, by Region, in Volume

Table Global Gene Therapy Market Forecast 2020-2026, by Region, in USD Million

Table Global Gene Therapy Market Forecast 2020-2026, by Region, in Volume

Table Sangamo Overview List

The key insights of the report:

About us:

Research is and will always be the key to success and growth for any industry. Most organizations invest a major chunk of their resources viz. time, money and manpower in research to achieve new breakthroughs in their businesses. The outcome might not always be as expected thereby arising the need for precise, factual and high-quality data backing your research. This is where MART RESEARCH steps in and caters its expertise in the domain of market research reports to industries across varied sectors.

For More Details Email Us: sales@martresearch.com

Continued here:
Global Gene Therapy Market Size, Share, Growth Rate, Revenue, Applications, Industry Demand & Forecast to 2026 - Galus Australis

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

One of those companies, Mogrify, just raised $16 million in Series A financing to bring its overall funding to over $20 million since its February launch. Led by CEO Darrin Disley, the funding will help expand their new base in Cambridge to a 60-strong staff and push forward their direct-conversion approach to cell therapy through research and licensing. Investors include Parkwalk Advisors and Ahren Innovation Capital.

They list potential applications as treatments for musculoskeletal and auto-immune disorders, cancer immunotherapy, and therapies for ocular and respiratory diseases. For example, you could use it regenerate cartilage in arthritis patients.

If you could take a cell from one part of the body and turn it into any other cell at any other stage of development for another part of the body, you effectively have the Holy Grail of regenerative medicine, Disley told Labiotech.eu in April.

Mogrifys advantage over the Yamanaka method called induced pluripotent stem cells (iPS), is that in theory it can be more scalable and avoid the problems associated with iPS. These include instabilities arising from the induced immature state and an increased risk of cancer if any pluripotent cells remain in the body.

The concept behind Mogrify actually predates, by nearly 19 years, Yamanakas discovery, which fast won him the 2012 Nobel Prize in Medicine. A 2017 Nature study on transdifferentiation, as the process is called, of fibroblasts into cardiac tissue traced the idea to a 1987 findingthat a master gene regulator could convert mice fibroblasts into skeletal muscle.

The problem though, according to Mogrify, is that most current efforts rely on an exhausting guess-and-check process. With hundreds of cell types and an even greater number of transcription factors the program that recodes the cell finding the right factor for the right cell can be like a custodian with a jangling, unmarked key ring trying to get into a building with thousands of locks.

Mogrifys key tech is a computer model they say can predict the right combination. The scientists behind the platform published a 2016 study in Nature applying the model to 173 human cell types and 134 tissues.

Before Mogrify, Disley led the Cambridge-based gene-editing company Horizon Discovery.

Continued here:
Cell therapy startup raises $16 million to fund its quest for the Holy Grail in regenerative medicine - Endpoints News

GlaxoSmithKline announced a five-year collaboration agreement with Lyell Immunopharma, a San Francisco, US-based company, working on methods to prevent inhibition of T cells by tumors and relapses due to loss of T cell functionality.

The agreement will see the two companies working on the advancement of GSKs cell therapy pipeline, specifically on GSK3377794, a potential treatment for multiple cancer types currently in Phase II clinical development, which targets the NY-ESO-1 antigen.

According to GSK, although the first two chimeric antigen receptor (CAR)-T cell therapies, Yescarta (axicabtagene ciloleucel) and Kymriah (tisagenlecleucel), have now reached the market, engineered T cells have not yet delivered strong clinical activity in common solid tumours.

Improving the fitness of T cells and delaying the onset of T cell exhaustion could help engineered T cell therapies become more effective, the company stated.

Further than GSKs cell therapy candidate, the research partnership will look to advance Lyells approach of enhancing initial T cell response against solid tumours into a platform technology for future cell and gene therapies development projects to treat rare types of cancer.

Lyells technology, according to Rick Klausner, the companys CEO, looks to tackle three barriers to T cell efficacy in solid tumours.

We are redefining the ways we prepare patient cells to be made into therapies, modulating cells functionality so that they maintain activity in the tumour microenvironment, and establishing methods of control to achieve specificity and safety for solid tumour-directed cell therapies, Klausner explained.

Go here to read the rest:
GSK partners with Lyell on cell therapies development - BioPharma-Reporter.com

Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a group of muscle diseases that includes limb-girdle muscular dystrophy (LGMD) type 2B.

Edasalonexent, formerly CAT-1004, is a small molecule designed to block the NF-kB cell signaling route a potential driver of muscle dystrophy, with a key role in skeletal and cardiac muscle breakdown. Through this mechanism, edasalonexent is thought to help preserve muscle function and slow disease progression. The experimental therapy is currently in Phase 3 clinical testing in boys with Duchenne muscular dystrophy (DMD).

Dysferlinopathy, which also includes Miyoshi myopathy, is a group of rare muscle disorders characterized by slowly progressive muscle weakness and wasting. The disorders are caused by mutations in the gene DYSF, which provides instructions for making dysferlin, a protein that normally works to provide structure to muscle fibers, and protect them.

In people with dysferlinopathy, muscles lack that key dysferlin protein, which results in chronic activation of the NF-kappa B pathway, muscle fiber damage, and failure to repair injured fibers.

Through the collaboration of Catabasis with the Jain Foundation, a non-profit dedicated to finding a cure for muscular dystrophy caused by dysferlin deficiency, researchers will conduct a preclinical study to evaluate the therapeutic potential of edasalonexent in dysferlinopathy.

The study will measure disease progression in dysferlin-deficient mice treated with edasalonexent, and use magnetic resonance imaging (MRI) and magnetic resonance spectroscopy to assess muscle volume, fat accumulation, and other changes. The initial results are expected in the first half of 2020.

We look forward to working with Catabasis to advance research for Dysferlinopathy, Laura Rufibach, PhD, and Doug Albrecht, PhD, co-presidents of the Jain Foundation, said in a press release.

Patients with dysferlinopathy (LGMD2B / Miyoshi myopathy) experience a progressive and debilitating decline in muscle function which significantly impacts their lives, Rufibach and Albrecht said. We are excited to explore the potential of edasalonexent to benefit those living with this disease.

In Duchenne research, results of the MoveDMD Phase 1/2 trial and its open-label extension (NCT02439216) showed that treating boys ages 4-7 with edasalonexent slowed disease progression and preserved muscle function compared with an off-treatment period. Biomarkers of muscle health and inflammation also showed significant improvements.

Evidence of NF-kB activation in both dysferlinopathy and DMD suggests a similar disease mechanism and opportunity for intervention, said Andrew Nichols, PhD, Catabasis chief scientific officer.

Through this collaboration, we look forward to learning more about the potential of edasalonexent in dysferlinopathy, he added.

The treatments efficiency and safety are being evaluated in children with Duchenne in the Phase 3 trial PolarisDMD (NCT03703882). Enrollment was just completed and exceeded the target number of participants, Catabis said. A total of 130 boys, ages 4 to 7, with any mutation type, have been enrolled in the U.S., Canada, Europe, Israel, and Australia.

We are thrilled to reach this important milestone. The interest and feedback from families and trial sites has been overwhelmingly positive, Joanne Donovan, MD, PhD, Catabasis chief medical officer, said in another press release.

Edasalonexent has the potential to be a foundational therapy, providing benefit to boys, regardless of their underlying mutation, with the potential to benefit muscle function, as well as cardiac function and bone health, she added.

Participants in PolarisDMD will be randomly assigned to receive either edasalonexent capsules three times per day (dose of 100 mg per kg per day) or a placebo, for 52 weeks, after which all boys and their eligible siblings may continue through an open-label extension study called GalaxyDMD.

Top-line results from PolarisDMD are expected in the fourth quarter of 2020. The findings are anticipated to support aNew Drug Application filing with the U.S. Food and Drug Administration in 2021.

We look forward to completing the trial next year and are working diligently toward the goal of making edasalonexent available to patients, Donovan said.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 42

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

Continued here:
Catabasis and Jain Foundation Study Edasalonexent for Dysferlinopathy - Muscular Dystrophy News

Zacks Investment Research upgraded shares of Axovant Gene Therapies (NASDAQ:AXGT) from a hold rating to a buy rating in a research report released on Wednesday morning, Zacks.com reports. The brokerage currently has $6.50 target price on the stock.

According to Zacks, Axovant Sciences Ltd. is a biopharmaceutical company which focuses on the acquisition, development and commercialization of therapeutics for the treatment of neurodegenerative disorders. Its product candidate includes RVT-101 which is in different clinical trial for the treatment of Alzheimers disease and other forms of dementia. Axovant Sciences Ltd. is based in Hamilton, Bermuda.

Other equities analysts also recently issued reports about the stock. ValuEngine raised shares of Axovant Gene Therapies from a sell rating to a hold rating in a research note on Thursday, August 1st. Leerink Swann assumed coverage on shares of Axovant Gene Therapies in a research note on Friday, June 21st. They issued an outperform rating and a $5.79 price target for the company. Svb Leerink assumed coverage on shares of Axovant Gene Therapies in a research note on Friday, June 21st. They issued an outperform rating and a $18.00 price target for the company. Robert W. Baird raised shares of Axovant Gene Therapies from a neutral rating to an outperform rating and decreased their price target for the stock from $16.00 to $13.00 in a research note on Monday, August 12th. Finally, Cowen reissued a hold rating on shares of Axovant Gene Therapies in a research note on Tuesday, July 9th. Two research analysts have rated the stock with a hold rating and nine have given a buy rating to the company. Axovant Gene Therapies has a consensus rating of Buy and a consensus price target of $26.91.

Shares of NASDAQ:AXGT opened at $5.72 on Wednesday. Axovant Gene Therapies has a 12 month low of $3.81 and a 12 month high of $20.80. The company has a 50-day moving average price of $6.84 and a 200-day moving average price of $5.49. The company has a quick ratio of 1.70, a current ratio of 1.70 and a debt-to-equity ratio of 0.60.

Axovant Gene Therapies (NASDAQ:AXGT) last released its quarterly earnings results on Friday, August 9th. The company reported ($1.23) earnings per share (EPS) for the quarter, topping the Zacks consensus estimate of ($1.34) by $0.11. Equities analysts expect that Axovant Gene Therapies will post -4.25 earnings per share for the current year.

A number of institutional investors and hedge funds have recently made changes to their positions in AXGT. Sphera Funds Management LTD. purchased a new stake in Axovant Gene Therapies in the first quarter worth about $6,794,000. Primecap Management Co. CA purchased a new stake in Axovant Gene Therapies in the first quarter worth about $1,400,000. BlackRock Inc. purchased a new stake in Axovant Gene Therapies in the second quarter worth about $1,482,000. Marshall Wace LLP purchased a new stake in Axovant Gene Therapies in the first quarter worth about $272,000. Finally, Jane Street Group LLC grew its holdings in Axovant Gene Therapies by 28.8% in the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after purchasing an additional 10,375 shares during the period. Institutional investors and hedge funds own 13.48% of the companys stock.

About Axovant Gene Therapies

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

Recommended Story: What is the formula for the cash asset ratio?

Get a free copy of the Zacks research report on Axovant Gene Therapies (AXGT)

For more information about research offerings from Zacks Investment Research, visit Zacks.com

Receive News & Ratings for Axovant Gene Therapies Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Axovant Gene Therapies and related companies with MarketBeat.com's FREE daily email newsletter.

View original post here:
Axovant Gene Therapies (NASDAQ:AXGT) Lifted to Buy at Zacks Investment Research - Riverton Roll