There could be a link between marijuana use and heart health – Medical News Today

New research found that 2.3% of reported marijuana users also have some form of cardiovascular disease.

The study appeared in the Journal of the American College of Cardiology.

The paper also looked at the possible interactions marijuana may have with drugs typically prescribed for cardiovascular issues, and the way marijuana chemicals may affect the heart at a molecular level.

According to the Centres for Disease Control and Prevention (CDC), heart disease a type of cardiovascular disease (CVD) is the leading cause of death in the United States.

In 2015, heart disease caused 23.4 % of all deaths in the U.S.

According to the American Heart Association (AHA), nearly half of all U.S. adults have some form of CVD. They cite an article in the journal Circulation that sets this figure at 121.5 million U.S. adults, which is approximately 48% of the population.

In contrast, the reported 2.3% of reported marijuana users who also have CVD is relatively low compared to the broader U.S. population.

This does not mean that marijuana use and CVD have no links. However, there is insufficient evidence to prove any connection, and what is available is of poor quality.

In the U.S., there are significant restrictions on researching marijuana. This means that scientists cannot carry out randomized clinical trials to understand its effects on health.

Researchers typically focus their research on observational studies, and the authors of the study claim that these show a possible link between marijuana use and cardiovascular events.

However, according to an article in the journal Annals of Internal Medicine, observational studies that have taken place are either inconclusive or have problems with their methodologies. This means that it is difficult for scientists to draw any conclusions from them.

The authors of the article in the Journal of the American College of Cardiology looked at data from the National Health and Nutrition Examination Survey (NHANES) to estimate how many marijuana users have CVD.

They found that 2 million of the 89.6 million U.S. adults who reported using marijuana also had CVD. This equates to 2.3% of reported marijuana users.

Dr. Ersilia M. DeFilippis, a former internal medicine resident at Brigham and Womens Hospital, Boston, MA, is the lead author. She is now a cardiology fellow at Columbia University Irving Medical Center.

She says, Marijuana use, both recreational and medical, is increasing nationally, yet many of its cardiovascular effects remain poorly understood. In our NHANES query, we estimated that 2 million adults with marijuana use had CVD in 20152016.

Since that time, additional states have passed legislature related to marijuana, so its use may have increased even further.

Notably, many of our cardiology patients are on medications that can interact with marijuana in unpredictable ways, depending on the formulation. This highlights that we need more data so that we can better counsel providers as well as patients.

Given the increase in marijuana use in the U.S., the authors of the study urge doctors to ask patients whether they use marijuana and to review their medication if necessary.

Current evidence is limited, and what little there is does not suggest a clear link between marijuana use and cardiovascular events. Researchers need to do more high quality research to determine precisely how and why marijuana affects cardiovascular health if it does at all.

According to the corresponding author Dr. Muthiah Vaduganathan, who is a cardiologist at Brigham, In the clinic, patients often ask us about the safety of marijuana use, and were pressed to offer the best scientific evidence.

Our current approach is that patients who are at high risk of cardiovascular events should be counseled to avoid or at least minimize marijuana use and that rigorous scientific research should be conducted to further inform recommendations for patient care.

Dr. Muthiah Vaduganathan

Before this can happen, however, authorities will need to lift the restrictions on marijuana research.

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There could be a link between marijuana use and heart health - Medical News Today

UVA Honors Its Leading Researchers at Boar’s Head Banquet – University of Virginia

The University of Virginias top leaders gathered Wednesday evening at the Boars Head Resort to honor faculty members from across Grounds for their outstanding contributions to their fields of study and societal impact through their research and scholarly activities.

University of Virginia President Jim Ryan presented the 2019 Research Achievement Awards to 13 UVA faculty members at the dinner event.

This is the first year of the Research Achievement Awards, Vice President for Research Melur Ram Ramasubramanian said. We believe that as a university, we are what we celebrate. We want to acknowledge the talented UVA faculty who are leaders in their fields and are impacting the world in positive ways.

Provost Elizabeth Liz Magill said, Were delighted to have a chance to celebrate the accomplishments and achievements of our faculty. From education policy to precision medicine to police-community relations, there are many different fields and individuals being honored by these awards.

Im awed and immensely grateful for the contributions the award winners have made to their respective fields and to the University of Virginia, Ryan said. Our strategic plan focuses a good deal of attention on supporting research. ... Our ultimate goal is to make it possible for researchers at UVA to do their very best work.

The awards covered excellence in research, collaboration, mentorship, public impact and innovation.

Pompano arrived at UVA in 2014 and assembled a robust research team in her lab. Pursuing new technologies and new questions, she is developing new approaches to study immunity. In the areas of immunoanalysis and immunoenineering, she is working to map out cellular activity in live tissues. Her group was recently awarded a large grant from the National Institutes of Health to develop an artificial lymph node on a microfluidic chip.

Dr. Pompano chose the road less travelled by pursuing entirely new technologies and questions, rather than the safer route of building on the experiences from her Ph.D. or postdoc work, Jill Venton, chair of the Department of Chemistry, said. This strategy required spending the first 2.5 years of her professorship laying new groundwork. Dr. Pompano is a research leader in the fields of analytical chemistry and immunoengineering.

Bassoks work is in early childhood education, and her focus has been to find a way for it to both meet high standards and make a difference in the lives of young children. To do this, she has partnered with policymakers and school districts in Virginia and Louisiana to study how early childhood education opportunities can happen at scale.

In the past four years, her work has accelerated. She has published 16 articles and received more than $6 million in grant funding. In 2017, Bassok was honored with the Presidential Early Career Award for Scientists and Engineers.

Daphna Bassok has raised the bar for the field and will motivate other scholars to do more insightful and rigorous work, said Katherine Magnuson, director of the University of Wisconsins Institute for Research on Poverty.

Alhusens research focuses on improving maternal and early infant health outcomes for disabled women and women living in poverty. Her research has been funded by the National Institutes of Health, the Health Resources and Services Administration and numerous foundations, and the goal of her work is to provide higher quality care to vulnerable populations.

She has received numerous awards for her work, including the Southern Nursing Research Society Early Science Investigator Award; the Association of Womens Health, Obstetric, and Neonatal Nurses Award for Excellence in Research; and School of Nursings Faculty Research Mentor Award.

Walsh is Lockhart B. McGuire Professor of Internal Medicine and directs the School of Medicines Hematovascular Biology Center. His research is focused on clonal hematopoiesis of indeterminate potential, or CHIP.

In his lab, he is looking at how mutations in blood cells lead to chronic diseases like heart attack and stroke. Through precision medicine, he is identifying and combatting the out-of-control multiplying process in these mutations to fight age-related diseases, as well as blood cancers like leukemia.

Walsh has published more than 350 scientific articles and he is the recipient of multiple research grants from the National Institutes of Health, including a MERIT Award. In 2011, the American Heart Association designated him a Distinguished Scientist by for his contributions to cardiovascular research.

Throughout his career, Scullys research, scholarship and teaching have focused on the science of how corrosion occurs and the engineering required to prevent it. He has conducted research and collaborated with scientists around the world in numerous industries such as energy, transportation, infrastructure, aerospace, maritime and microelectronics.

His projects include two U.S. Department of Energy Energy Frontier Research centers, two Department of Defense multi-university research initiatives, as well as grants from the National Science Foundation, PPG Industries and Axalta (formerly DuPont), and the U.S. Office of the Undersecretary of Defense.

Scully, the Charles Henderson Chaired Professor and chair of the Department of Materials Science and Engineering, also co-directs the Center for Electrochemical Science and Engineering, one of the leading centers in the world focusing on materials degradation. The center has generated more than $30 million in research funding in the last 10 years and graduates on average four to five Ph.D. students per year.

Scully is technical editor in chief of CORROSION, The Journal of Science and Engineering, the premier international research journal for the field. He serves in several capacities as an ambassador for the materials-corrosion field, including several meetings to debrief the U.S. Congress on materials degradation issues of national importance.

John Scullys contributions to corrosion can be characterized by quality, quantity and longevity, said Gerald S. Frankel, Ohio State University distinguished professor in materials science and engineering and a member of CORROSIONs editorial board. It is clear that he is a world leader, if not the world leader, in metal passivity, passivity breakdown and localized corrosion, and stress corrosion cracking.

In more his more than 20 years at UVA, Lambert has advanced the science of risk analysis and systems engineering. He has led more than 60 projects related to advanced logistics systems for a total of approximately $25 million in research funding.

Lambert, a professor in the Department of Engineering Systems and Environment, has focused on the disruption of system priorities by emergent and future conditions, including technologies, regulations, markets, environments, behaviors and missions. His work has been applied to disaster resilience, energy infrastructure, coastal protection, economic development, transportation, biofuels and Olympics planning, among other challenges.

His research has been cited more than 5,000 times across more than 200 publications. In 2019, he chaired the Fifth World Congress on Risk, convening more than 300 scientists in Cape Town, South Africa.

Professor Lambert is among the most accomplished and respected scientists of systems engineering and risk analysis in the world today, said Igor Linkov, Risk and Decision Science Team Lead for the U.S. Army Engineer Research and Development Center. Lambert in his research invented the application of scenario-based preferences in risk analysis.

Connelly, Morris and Grossman worked together on a multi-disciplinary project to examine how early life experiences affect the brain and social behaviors. The team studies the brain, as well as social and cognitive development, during the first two years of life, focusing on oxytocin and its role in social behavior. Their research has helped to illuminate gaps in our knowledge about behavioral development in humans, and helps us better understand healthy and atypical development.

They received a National Science Foundation Research Award in 2017 for their cutting-edge approach in combining epigenetic, neuroscience and behavioral methods across their three labs, and their work has led to several publications.

Moore is a busy physician-scientist with his own lab, and has recently become the division chief of Pediatric Gastroenterology, Hepatology, & Nutrition at UVA. He also co-wrote the application for a Trans-University Microbiome Initiative grant, which was funded last year by the Universitys Strategic Investment Fund in an effort to make UVA a center for microbiome research. But that has not stopped him from repeatedly aiding his colleagues and providing them with key resources when they needed them.

Three colleagues joined forces to nominate Moore for the mentorship award, mentioning his critical support, his generous sponsorship and advice, and his guidance as they dealt with grant applications and the logistics of their first accepted grants. Moore went above and beyond, donating lab space and reaching out to his networks to help them make the connections and give them a leg up in their careers.

Williams only arrived at Batten two years ago, but after the violent Unite the Right rally in Charlottesville in August 2017 he was able to immediately show the value of his research and public service engagement to the University community.

Starting before he came to the University, he has spent two decades doing research in the field on police-community relations around racial profiling, community policing and the need for law enforcement officers to work with their community on issues of public safety. In all his work, he strives to make an impact on communities by building relationships and tackling problems wherever they crop up.

Dr. Williams consistently uses his knowledge, experience and passion for the good of our city, Mindy Goodall, executive director of the Charlottesville Police Foundation, said. Charlottesville is fortunate to have gained him as a citizen and champion of police and community reconciliation.

The award for Innovator of the Year was presented to Dillingham and Ingersoll for their creation of PositiveLinks, an application designed to improve health outcomes and care for people living with HIV. They will give deliver a keynote lecture Feb. 18 in the Rotunda Dome Room.

Other researchers (in alphabetical order by school) were honored for being the top 25 in sponsored funding, top cited, national award winners, named to a national academy, or named as an outstanding researcher for their school:

Timothy Beatley, PlanningBarbara Brown Wilson, PlanningMona El Khafif, Urban & Environmental Planning

Jessica Connelly, PsychologyRita F. Dove, EnglishKevin Everson, ArtTobias Grossman, PsychologyL. Ilse Cleeves, AstronomyNitya Kallivayalil, AstronomyLee M. Lockwood, EconomicsJames P. Morris, PsychologyKen Ono, MathematicsRebecca R. Pompano, ChemistryMarilyne Stains, ChemistryAlan S. Taylor, History

Christopher Barrett, Director

David G. Mick, Marketing

Derrick P. Alridge, Leadership, Foundations and PolicyDaphna Bassok, Leadership, Foundations and PolicyRobert Q. Berry, Instruction and Special EducationCatherine Bradshaw, Human ServicesBenjamin L. Castleman, Leadership, Foundations and PolicyNancy L. Deutsch, Youth-NexJason Downer, Human ServicesSara E. Rimm-Kaufman, Leadership, Foundations and PolicyWilliam J. Therrien, Instruction and Special EducationArt Weltman, KinesiologyJoanna Lee Williams, Leadership, Foundations and PolicyAmada P. Williford, Human Services

Thomas H. Barker, Biomedical EngineeringHilary Bart-Smith, Mechanical and Aerospace EngineeringCraig H. Benson, Environmental EngineeringSteven M. Bowers, Electrical and Computer EngineeringJames T. Burns, Materials ScienceBenton H. Calhoun, Electrical and Computer EngineeringJoe C. Campbell, Electrical and Computer EngineeringGeorge J. Christ, Biomedical EngineeringJason L. Forman, Center for Applied BiomechanicsJeffery W. Holmes, Biomedical EngineeringPatrick E. Hopkins, Mechanical and Aerospace EngineeringKevin A. Janes, Biomedical EngineeringJames H. Lambert, Systems and EnvironmentXiaodong (Chris) Li, Mechanical and Aerospace EngineeringPamela M. Norris, Mechanical and Aerospace EngineeringElizabeth J. Opila, Materials ScienceMatthew B. Panzer, Mechanical and Aerospace EngineeringJohn R. Scully, Materials ScienceKevin Skadron, Computer ScienceMary Lou Soffa, Computer ScienceJohn A. Stankovic, Computer ScienceMalathi Veeraraghavan, Electrical and Computer Engineering

Brian N. Williams, Public PolicyJay Shimshack, Research Dean

Jayakrishna Ambati, OphthalmologyRuth Bernheim, Public Health SciencesAlison K. Criss, Microbiology /GIDIRebecca Dillingham, Infectious DiseasesLinda R. Duska, Obstetrics/Gynecology OncologyAnindya Dutta, Biochemistry/Molecular GeneticsW. Jeff Elias, NeurosurgeryEdward H. Egelman, Biochemistry/Molecular GeneticsRobin A. Felder, Clinical PathologyEric R. Houpt, Infectious DiseasesKaren Ingersoll, Psychiatry and Neurobehavioral SciencesKaren C. Johnston, NeurologyJaideep Kapur, NeurologyAnne K. Kenworthy, Molecular Physics and BiophysicsJonathan Kipnis, NeuroscienceRobert C. Klesges, Public Health SciencesBoris P. Kovatchev, Psychiatry and Neurobehavioral SciencesThomas P. Loughran, Oncology and MedicineColeen A. McNamara, Internal and Cardiovascular MedicineWladek Minor, Molecular Physics and BiophysicsSean R. Moore, PediatricsJames P. Nataro, PediatricsImre Noth, Internal and Pulmonary MedicineMark D. Okusa, NephrologyGary K. Owens, Cardiovascular Research, Molecular Physiology and Biological PhysicsKevin A. Pelphrey, NeurologyWilliam A. Petri, Internal Medicine and Infectious DiseasesKodi S. Ravichandran, MicrobiologyPatricio E. Ray, PediatricsStephen S. Rich, Public Health SciencesLukas K. Tamm, Molecular Physics and BiophysicsGregory C. Townsend, Internal Medicine and Infectious DiseasesKenneth Walsh, Internal and Cardiovascular MedicineKatharine Hsu Wibberly, Public Health SciencesMichael C. Wiener, Molecular Physics and BiophysicsMark Yeager, Molecular Physics and BiophysicsJames C. Zimring, Pathology

Jeanne L. Alhusen, Nursing

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UVA Honors Its Leading Researchers at Boar's Head Banquet - University of Virginia

Meet the Ex-Dallas Doctor Now Playing a Doctor on TV During the Super Bowl – D Magazine

When you briefly glimpse Calvin Peters in a Verizon commercial during Super Bowl LIV on Sunday, his face might seem familiar.

Maybe you remember him from his performances during the audition stage of American Idol. Perhaps you recognize him from guest appearances in television dramas such as 9-1-1 or Snowfall. Or possibly he was your doctor during an appointment at UT Southwestern Medical Center.

The diverse resume for the former Dallas physician now finds him splitting much of his time between treating patients and auditioning for roles on stage and screen.

The art caters to my right brain, and the medicine and science is challenging and fostering my left brain, Peters said. I love helping people. Every time I look in a patients eyes and theyre hurting, that tugs at my heart in a way that nothing else can. But then I love to be on stage and perform.

Appropriately enough, Peters plays a surgeon in the Verizon spot that will debut Sunday. Its a continuation of the companys ongoing campaign saluting first responders and was directed by Hollywood filmmaker Peter Berg (Friday Night Lights).

Hes a visionary. It was so encouraging and motivating to see this guy work, Peters said. I want to continue to grow to where Im working with all kinds of creative talent.

Peters developed a passion for medicine and for performing while growing up in the Stop Six neighborhood in Fort Worth. He acted in his first play at age 9, and later graduated from North Side High School.

At Hampton University in Virginia, Peters was involved in musical theater before graduating with a degree in molecular biology in 2006.

He returned to Texas to attend medical school at UT Southwestern, then stayed to complete his residency in 2014, specializing in physical medicine and rehabilitation.

During his residency, he impulsively auditioned for American Idol, eventually making the show in 2013. Later that year, he appeared on an episode of The Dr. Oz Show spotlighting the Most Eligible Doctors. He was hooked.

I was still going to work every day, and everybody at the hospital was really supportive, but I still felt empty, he said.

So Peters took some classes and hired an agent in Dallas, and began acting in local commercials and short films. In 2015, he relocated to Los Angeles and set up his practice primarily as a therapist for nursing home patients, which allowed him to have a flexible schedule.

As his exposure grew, Peters refined his craft with improvisation classes, voice classes, and dance classes. He hired a manager and began booking jobs, including an episode of Snowfall that aired in August and a small role on 9-1-1 in November. Peters also plays a waiter in the low-budget romantic comedy What Love Looks Like, which began streaming on Amazon Prime earlier this month.

Juggling his two careers has become more of a challenge. Peters recently had an opportunity to play Harpo in a traveling stage production of The Color Purple, but couldnt commit to the full year away from his practice.

His circumstances mean Peters isnt be as desperate as other up-and-coming actors in California, but he said hes just as driven. He spends 30-40 hours per week reading scripts and rehearsing.

My bills are all paid. Im still saving money and have health insurance. I have an assistant. I live in Beverly Hills. My life is not bad, Peters said. But when I go into an audition, Im still going hard. Im giving it my all. Im putting my foot in the water just as fast and hard as somebody whos waiting tables.

Eventually, he would like to give back to the rough neighborhood in eastern Fort Worth where he was raised, and where his family has been victimized by a pervasive cycle of drugs and violence.

Ill always be connected to that community. Im never going to act like Im too good for them. Fort Worth will always be in my heart, he said. Sometimes its so difficult to go be around some of the situations because the energy and the vibes are not right. The way I grew up was not ideal, but I feel so fortunate.

As his success in show business grows and he plans for the long-term future, Peters said he hasnt thought about abandoning his career in medicine.

I dont think you can ever stop somebody from being a doctor, Peters said. I try not to think about that right now, because I dont have to make that choice yet. If the situation got big enough, I probably wouldnt be able to go into a hospital and practice, but theres always a way to stay connected to healthcare.

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Meet the Ex-Dallas Doctor Now Playing a Doctor on TV During the Super Bowl - D Magazine

Realizing The Full Potential Of Precision Medicine In Oncology – Contract Pharma

Precision medicine promises a new paradigm in oncology where every patient receives truly personalized treatment. This approach to disease diagnosis, treatment and prevention utilizes a holistic view of the patientfrom their genes and their environment to their lifestyleto make more accurate decisions.

Growing at a rate of 10.7 percent, the precision medicine market is expected to exceed $96 billion by 2024.1 Bioinformatics represent a significant share of the market, as bioinformatics tools enable the data mining necessary for rapid identification of new drug targets and repurposing of existing treatments for new indications.1 (Reuters) The oncology segment of the precision market is expected to experience an 11.1 percent compounded annual growth rate (CAGR) leading up to 2024 due to the success of recent targeted therapies and subsequent high demand.

Still, precision medicine is in its infancy, and making personalized treatment a reality for all patients requires a transformation in how novel therapies are developed and delivered. New regulatory, technical, clinical and economic frameworks are needed to ensure that the right patients are able to access the right therapy at the right time. In this article, we review the current state of precision medicine in oncology and explore some of the challenges that must be addressed for precision medicine to reach its full potential.

Great strides toward precision medicine are being made in the area of cancer immunotherapy, which is designed to boost a patients own immunity to combat tumor cells. The introduction of immune checkpoint inhibitors (PD-1/PD-L1 and CTLA-4 inhibitors) revolutionized treatment for certain hematologic malignancies and solid tumors. To date, immune checkpoint inhibitors have been approved by the U.S. Food and Drug Administration (FDA) for more than 15 cancer indications, but their widespread use has been hampered by unpredictable response rates and immune-related adverse events.

The approvals of the first chimeric antigen receptor (CAR)-T cell (CAR-T) therapies in 2017 were the next leap forward in precision medicine. These immunotherapies demonstrated that it was possible to take out a patients own T-cells, genetically modify them, and then put them back in to target cancer cells. With complete remission rates as high as 83 percent within three months of treatment, CAR-T therapies represent a seismic shift in our approach to cancer, bringing the elusive possibility of a cure one step closer. However, longer-term follow-up has shown that these remissions may not be durable2 and prevention of relapse must still be studied.

Ultimately, the goal of cancer immunotherapy is to stimulate the suppressed immune system of a patient with cancer so that it can launch a sustained attack against tumor cells.3 This is complicated, as the interactions between tumors and immune systemsometimes called the Cancer-Immunity Cycle (see Figure 1 in the slider above)4are complex and dynamic. The Cancer-Immunity Cycle manages the delicate balance between the immune systems ability to recognize non-self and the development of autoimmunity.

In some cases, the immune system may fail to recognize tumor cells as non-self and may develop a tolerance to them. Moreover, tumors have an armamentarium of methods for evading the immune system. Given this elaborate interplay between cancer and immunity, there is a wide range of potential cancer immunotherapy approaches:

The immune response to cancer involves a series of carefully regulated events that are optimally addressed as a group, rather than individually.4 The complexity of the immune response to cancer provides a strong rationale for combination therapies, for instance:

Increasingly, the development and deployment of immunotherapy relies on harnessing genomic data to identify the patients most likely to respond to immunotherapy and to customize immunotherapy for a given patient.6 Thus, molecular profiling technologies, such as next-generation sequencing, have become integral to drug development and patient selection. At the same time, researchers are focusing on identifying molecular alterations in tumors that may be linked to response.7 The molecular fingerprints of a tumor can be quite complex and heterogeneous, not only across tumors, but also within a single patient. Consequently, molecular tumor characterization requires both multidimensional data from laboratory and imaging tests and advanced software and computational methods for analyzing these data.8 This emergence of computational precision oncology is associated with both opportunities and challenges, from validation and translation to regulatory oversight and reimbursement.

The regulatory landscape is evolving to keep pace with technological advances in cell engineering and gene editing. Since 2013, the FDA has published four guidance documents on cellular and gene therapy products, as well as two guidance documents providing recommendations on regenerative medicine advanced therapies (RMATs). Specifically, their Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, published in November 2017, provides guidance on the expedited development and review of regenerative medicine therapies for serious or life-threatening diseases and conditions. This document also provides information on the use of the accelerated approval pathway for therapies that have been granted the RMAT designation.9

In the EU, the European Medicines Agency (EMA) published a draft revision of its Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells in July 2018.10 This draft revision includes current thinking on the requirements for nonclinical and clinical studies, as well as specific sections on the scientific principles and clinical aspects of CAR-T products.

Precision medicines such as CAR-T therapies require manufacturers to transform a complex, individualized treatment into a commercial product. In conventional manufacturing, the entire manufacturing process occurs within the confines of the manufacturing facility. With cell therapies, however, the process begins with the collection of cells from the patient and ends with administration of the final product (see Figure 2 in the slider above). In between, the cells are handed off multiple times for the process of genetic modification, creating a complex supply chain that blends manufacturing and administration.11

Moreover, in contrast to traditional manufacturing where the starting materials are standardized or well-defined, the starting materials for cell therapies are derived from patients and, thus, highly variable.

As evidenced by the manufacturing challenges that plagued the launch of Kymriah (tisagenlecleucel), even pharmaceutical giants have struggled with meeting label specifications for commercial use.13 To help address its manufacturing hurdles, Novartis acquired CellforCure, a contract development manufacturing organization, and plans to transform by focusing on data and digital technologies.14,15 What this means for sponsors is that robust, scalable manufacturing must be incorporated into clinical developing planning at its earliest stages.

The high price tags associated with CAR-T therapies illustrate how expensive targeted therapies are in comparison to their traditional counterparts.16 Existing health insurance models have not been structured to reimburse for costly treatments that offer the potential for long-term benefit or even cure. The pricing model for CAR-T therapies may be especially challenging for private insurance companies, which have higher turnover and shorter coverage windows than national health insurance programs. For sponsors of precision medicine therapies, one way to address the challenge of reimbursement is to create innovative, value- or outcomes-based pricing models, rather than focusing on sales volume. The success of these new pricing models will rely on patient selection. To demonstrate value and optimizing outcomes, sponsors will need to develop profiles of patients who are most likely to respond and provide tools for identifying these patients.8

Of note, on August 7, 2019, the Centers for Medicare & Medicaid Services (CMS) finalized the decision to cover FDA-approved CAR-T therapies when provided in healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies (REMS) for FDA-approved indications. Medicare will also cover FDA-approved CAR-T treatments for off-label uses that are recommended by CMS-approved compendia.17

Beyond the pharmaceutical companies that are working to develop personalized treatments, the precision medicine ecosystem has a number of other key stakeholdersregulators, payers, diagnostic companies, healthcare technology companies, healthcare providers and, of course, patients. Pharmaceutical companies need to engage with each of these stakeholders by providing education or developing partnerships that help demonstrate the need for high-quality data collection, the value of precision medicine, and the process for identifying the right patients.

Sponsors may also benefit from engaging with patient advocacy groups as these groups play a critical role in connecting patients and caregivers with scientific and healthcare experts to learn about how new immunotherapy breakthroughs are changing the standard of care.

Empowered patients pushing for the latest innovations are propelling precision medicine forward, but we still have a way to go before the full potential of precision medicine is realized. In its maturity, precision medicine will not only enable the personalization of treatments for individual patients, but also inform public health at a population level as insights from the genetic and molecular data collected are used to advance our understanding of disease. Robust data collection and analysis, along with standardization, are required for building this foundation of precision medicine, and multi-stakeholder buy-in is necessary for addressing issues around data integration and privacy.

While significant challenges remain, the opportunity to transform patient outcomes and population health with precision medicine is tantalizing. Increasingly, we are seeing advanced technologiessuch as artificial intelligence and machine learningbeing incorporated into the drug discovery and development process. This underscores the critical need for a multidisciplinary approach to precision medicine, from discovery at the bench all the way through to delivery at the bedside, to help ensure that more patients can access the right therapy at the right time, and the right price.

References

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Realizing The Full Potential Of Precision Medicine In Oncology - Contract Pharma

Triple negative breast cancer might not actually be ‘breast cancer’ – Clinical OMICs News

A new study from New York might completely change how breast cancer is classified and treated. Researchers have discovered, in a study of triple negative breast cancer in black women, that the molecular mechanisms involved are more closely related to non-breast cancers, and two specific gene mutations may be responsible for the tumor development.

This study would signal a potential treatment breakthrough for patients with this form of breast cancer, which disproportionately affects and tends to develop more aggressively in black women. Triple negative breast cancer is a form of breast cancer that is not hormone driven, and it does not express the estrogen receptor, progesterone receptor, or HER2/neu genesunlike other forms of the disease, which rely on hormone-driven therapies to control the cancer.

The paper, authored by researchers at The Graduate Center of The City University of New York, Hunter College, Memorial Sloan Kettering Cancer Center, and the University of Chicago, details findings about the interaction between two specific genes during DNA replication that appear to drive growth of malignant cells in patients with triple negative breast cancer.

The research team found the presence of a mutated p53 (a tumor suppressor gene whose mutation often leads to cancer development) and a mutated PARP (a gene that maintains DNA integrity) in a large majority of patients with triple negative breast cancer. Specifically, they found that p53 would interact with replicating DNA and PARP, driving cell growth and division.

This information would suggest that if drugs could specifically target these two mutated genes and the proteins they produce, it would be possible to suppress their actions and slow or stop tumor growth, so the researchers tested this in lab, using drugs developed for other types of cancer.This was indeed the case.

Our new findings suggest that the presence of both [p53] and PARP could serve as a good identifier of breast cancers that would respond to combined treatment with talazoparib a PARP inhibitor that was developed to treat breast cancers with the BRCA mutation and temozolomide a chemotherapy agent that is used to treat some brain cancers, said Professor Jill Bargonetti, whose lab conducted the research. This is an exciting finding because it could lead to the first targeted therapy for triple negative breast cancer, enabling more precise and effective treatment of a very aggressive form of the disease.

To conduct this study, researchers investigated a variety of breast cancer cell lines, patient-derived xenographs, tissue microarray samples, and data from The Cancer Genome Atlas to tease out the association and interaction between p53 and PARP in triple negative breast cancer.

Our findings that mutant p53 and PARP participate in the DNA replication pathway will provide mechanism-derived dual biomarkers that aid in the diagnosis and treatment of these therapeutically elusive subsets of breast cancer, said Gu Xiao, a research associate with Bargonettis lab and the papers first author.

The scientists had discovered that high levels of these proteins are present on replicating DNA in these types of tumors, suggesting that the unusual presence and levels of these proteins may drive tumor growth. When these researchers reviewed the therapeutic mechanisms of existing cancer drugs, it led the research team to identify talazoparib and temozolomide as existing drugs that could potentially be combined to create a targeted therapy that could effectively suppress the growth of triple negative breast cancer tumors.

Looking to the future, this group plans to test if this combination of drugs can successfully block replication of triple negative breast cancer cells in xenographed animal models. If the therapy can prove to be successful, it would very likely lead to the reclassification of triple negative breast cancer to a category of cancers called mutant p53/PARP1 positive cancers, which are treatable with a combination PARP inhibitor therapies.

With so little in positive news for this particular subset of breast cancer, and many previous clinical trials being disappointing, these new findings are both welcome news to the scientific community, as well as a good explanation as to why this disease has been so hard to treat compared to other forms of breast cancer.

This study gives doctors two new biomarkers to test for in patients, and many new potentials in clinical trials, which can be fast tracked since so many drugs already exist that target these specific genes. While this study only tested out two drugs, there are many other drugs that specifically target p53 and PARP, and many more which can be developed, which may give even better results. Hopefully, there will be many new, positive developments for this disease following this discovery.

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Triple negative breast cancer might not actually be 'breast cancer' - Clinical OMICs News

Is Predictive Technology Group Inc (OTCMKTS:PRED) Ready for Another Run? – The Oracle Dispatch

Predictive Technology Group Inc (OTCMKTS:PRED) just announced that it and Atrin Pharmaceuticals LLC areentering into a collaboration agreement to develop molecular diagnostic toolsto facilitate improved selection of cancer patients who would most benefit fromtreatment with DNA Damage and Response (DDR) inhibitors, including Atrins andother small molecule ATR inhibitors.

According to the release, Atrinand Predictive will jointly utilize Predictive Laboratories state-of-the-artsequencing capabilities and genomics expertise to identify cancer patients withspecific molecular markers that predict the level of clinical response toAtrins, and other, targeted therapies. This is intended to improve patientoutcomes as well as improve Atrins ability to successfully progress itsproduct pipeline, and upon commercialization, improve on the treatments forwomen with cancer.

Predictive Technology GroupInc (OTCMKTS:PRED) promulgates itself as a company that, together with itssubsidiaries, develops and commercializes discoveries and technologies involvedin novel molecular diagnostic and pharmaceutical therapeutic/human cells,tissues, and human cellular and tissue-based products (HCT/Ps).

The company operates throughtwo segments, Regenerative Medicine Products and HCT/Ps, and Diagnostics andTherapeutics. It offers ARTguide, a genetic diagnostic and prognostic test forwomen experiencing infertility as a result of endometriosis and other healthconcerns; and regenerative medicine products, including AmnioCyteT, AmnioCytePlusT, PolyCyteT, and CoreCyteT.

The company was formerly knownas Global Enterprises Group, Inc. and changed its name to Predictive TechnologyGroup, Inc. in July 2015. Predictive Technology Group, Inc. was founded in 2005and is headquartered in Salt Lake City, Utah.

According to companymaterials, Predictive Technology Group aims to revolutionize patient carethrough predictive data analytics, novel gene-based diagnostics and companiontherapeutics through its subsidiaries Predictive Therapeutics, PredictiveBiotech, and Predictive Laboratories. These subsidiaries are focused onendometriosis, scoliosis, degenerative disc disease and human cell and tissueproducts. The subsidiaries use genetic and other information as cornerstones inthe development of new diagnostics that assess a persons risk of illness andtherapeutic products designed to identify, prevent and treat diseases moreeffectively.

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As noted above, PRED just announced that it and Atrin Pharmaceuticals LLC are entering into a collaboration agreement to develop molecular diagnostic tools to facilitate improved selection of cancer patients who would most benefit from treatment with DNA Damage and Response (DDR) inhibitors, including Atrins and other small molecule ATR inhibitors.

Weve witnessed 36% piled onfor shareholders of the listing during the trailing month. In addition, thename has witnessed a pop in interest, as transaction volume levels haverecently pushed 11% above the average volume levels in play in this stock overthe longer term.

We are very pleased to workwith Atrin Pharmaceuticals, a recognized leader in the development ofanti-cancer therapeutics targeting DDR, said Bradley Robinson, president andchief executive officer of Predictive Technology Group. We see an opportunityto develop a precision medicine approach to address unmet medical needs bycombining our state-of-the-art sequencing capabilities, genomics expertise andcompanion diagnostics with Atrins targeted therapeutics. This collaboration isconsistent with our vision of building a leading womens health platform, andwe look forward to working together on this important initiative.

Currently trading at a marketcapitalization of $276M, PRED hasa chunk ($842K) of cash on the books, which stands against about $8.6M in totalcurrent liabilities. PRED is pulling in trailing 12-month revenues of $43.7M.In addition, the company is seeing major top-line growth, with y/y quarterlyrevenues growing at 2.4%. You can bet we will update this one again as newinformation comes into view. Sign-up forcontinuing coverage on shares of $PRED stock, as well as other hot stock picks,get our free newsletter today and get our next breakout pick!

Disclosure: we hold no position in $PRED, either long orshort, and we have not been compensated for this article.

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OpGen (NASDAQ:OPGN) and Avant Diagnostics (NASDAQ:AVDX) Head-To-Head Review – Riverton Roll

OpGen (NASDAQ:OPGN) and Avant Diagnostics (OTCMKTS:AVDX) are both small-cap medical companies, but which is the better stock? We will contrast the two businesses based on the strength of their profitability, valuation, risk, dividends, analyst recommendations, institutional ownership and earnings.

Profitability

This table compares OpGen and Avant Diagnostics net margins, return on equity and return on assets.

Volatility and Risk

OpGen has a beta of 0.64, suggesting that its stock price is 36% less volatile than the S&P 500. Comparatively, Avant Diagnostics has a beta of 9.42, suggesting that its stock price is 842% more volatile than the S&P 500.

Earnings & Valuation

This table compares OpGen and Avant Diagnostics revenue, earnings per share and valuation.

Avant Diagnostics has lower revenue, but higher earnings than OpGen.

Analyst Recommendations

This is a breakdown of current ratings and target prices for OpGen and Avant Diagnostics, as reported by MarketBeat.com.

OpGen presently has a consensus target price of $7.93, suggesting a potential upside of 405.31%. Given OpGens higher probable upside, equities research analysts plainly believe OpGen is more favorable than Avant Diagnostics.

Insider & Institutional Ownership

0.3% of OpGen shares are held by institutional investors. Comparatively, 0.0% of Avant Diagnostics shares are held by institutional investors. 5.0% of OpGen shares are held by insiders. Comparatively, 38.0% of Avant Diagnostics shares are held by insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a stock will outperform the market over the long term.

Summary

Avant Diagnostics beats OpGen on 7 of the 11 factors compared between the two stocks.

About OpGen

OpGen, Inc., a precision medicine company, engages in developing molecular information products and services to combat infectious diseases in the United States and internationally. The company utilizes molecular diagnostics and informatics to help combat infectious diseases. It also helps clinicians with information about life threatening infections, enhance patient outcomes, and decrease the spread of infections caused by multidrug-resistant microorganisms. The company's products include Acuitas AMR Gene Panel, a vitro diagnostic test for the detection and identification of various bacterial nucleic acids and genetic determinants of antimicrobial resistance in urine specimens or bacterial colonies isolated from urine and other body sites; and QuickFISH and PNA FISH products, which are FDA-cleared and CE-marked diagnostic test designed to detect antimicrobial- resistant pathogens. In addition, it offers Acuitas Lighthouse informatics systems, a cloud-based HIPAA compliant informatics offerings, which combine clinical lab test results with patient and hospital information, and provide analytics and insights to enable manage MDROs in the hospital and patient care environment. The company was incorporated in 2001 and is headquartered in Gaithersburg, Maryland.

About Avant Diagnostics

Avant Diagnostics, Inc., a commercial-stage molecular data-generating company, focuses on the development and commercialization of proprietary data-generating assays that provide information for physicians and patients in the areas of cancers. It owns license and distribution right for OvaDx, a noninvasive proteomics diagnostic screening test for the early detection of ovarian cancer. The company was founded in 2009 and is based in Washington, District of Columbia.

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OpGen (NASDAQ:OPGN) and Avant Diagnostics (NASDAQ:AVDX) Head-To-Head Review - Riverton Roll

The Global Market for Sample Preparation in Genomics, Proteomics and Epigenomics is Projected to Reach $7.7 Billion by 2021, Growing at a CAGR of 6.5%…

DUBLIN--(BUSINESS WIRE)--The "Sample Preparation in Genomics, Proteomics, and Epigenomics: Global Markets" report has been added to ResearchAndMarkets.com's offering.

This research report categorizes the market for sample preparation in genomics, proteomics and epigenomics by product segment. The major product segments are instruments, consumables, accessories and sample preparation kits. The instruments segment is subdivided into workstations, liquid handling systems, extraction systems and other instruments. Consumables are divided into columns, filters, tubes, plates and other consumables. Sample preparation kits are segmented into purification kits, isolation kits, extraction kits and others.

The global market for sample preparation is segmented by end user into research centers, academic institutes, and government institutes, hospitals and clinics, pharmaceutical and biotechnology companies and others. The markets in North America, Europe, Asia-Pacific and rest of the world are covered. The market is also segmented by application into genomics, proteomics and epigenomics.

The Report Includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market Overview

Chapter 4 Global Market for Sample Preparation in Genomics, Proteomics and Epigenomics by Product Segment

Chapter 5 Global Market for Sample Preparation in Genomics, Proteomics and Epigenomics by Application Segment

Chapter 6 Global Market for Sample Preparation in Genomics, Proteomics and Epigenomics by End User

Chapter 7 Global Market for Sample Preparation in Genomics, Proteomics and Epigenomics by Region

Chapter 8 Company Profiles

Chapter 9 Competitive Landscape

For more information about this report visit https://www.researchandmarkets.com/r/j4pqkx

Excerpt from:

The Global Market for Sample Preparation in Genomics, Proteomics and Epigenomics is Projected to Reach $7.7 Billion by 2021, Growing at a CAGR of 6.5%...

Australia Day Honours list celebrates staff and alumni – News – The University of Sydney

StaffCompanion (AC) in the General Division of the Order of Australia

Professor Bruce Gregory Robinson AM (MBBS 80 MSc 83 MD 91)For eminent service to medical research, and to national healthcare, through policy development and reform, and to tertiary education.

Professor Peter Joseph McCluskeyFor distinguished service to ophthalmology, and to medical education, to eye health organisations, and to the community.

Emeritus Professor Jules Mitchell Guss (BSc 67 PhD 70)For distinguished service to education and scientific research in the field of molecular bioscience, and to professional organisations.

Dr Stephen Bourke For significant service to the international community of Jordan through archaeologicalprojects.

Professor Pierre Henri ChapuisFor significant service to medical education, and to colorectal surgery.

Professor Richard de DearFor significant service to education, particularly the design of the built environment.

Associate Professor Nicholas John EvansFor service to medicine in the fields of gastroenterology and hepatology.

Dr Meng Chong NguFor service to medicine in the fields of gastroenterology and hepatology.

Her Excellency the Honourable Margaret Joan Beazley AO QC (LLB 74 LLD 08)For eminent service to the people of New South Wales, particularly through leadership roles in the judiciary, and as a mentor of young women lawyers.

Professor Margaret Elaine Gardner AO (BEc 76 PhD 84)For eminent service to tertiary education through leadership and innovation in teaching and learning, research and financial sustainability.

Ms Ilana Rachel Atlas (LLM 87)For distinguished service to the financial and manufacturing sectors, to education, and to the arts.

Dr John Michael Bennett AM (LLM 64 BA 66 LLB 69 LLD 90 Dlitt 07)For distinguished service to the law through prolific authorship of biographies of eminent members of the legal profession.

Professor Robert Graham Cumming (MD 17)For distinguished service to medical education and research, particularly to ageing and age-related diseases.

Mr Kevin McCann AM (BA 61 LLB 64)For distinguished service to business, to corporate governance, and as an advocate for gender equity.

Professor John Reginald Piggot (BA 70)For distinguished service to education, to population ageing research, and to public finance policy development.

Professor Robert (John) Simes (Bsc (Med) 74 MBBS 76 MD 88)For distinguished service to education, and to medicine, in the field of cancer research and clinical trials.

Dr Robyn Williams AM (DSc 88 D.Sc(Honoris Causa) 88)For distinguished service to science as a journalist, radio presenter and author, and to education.

Mr Anthony Abrahams (BA 56 LLB 59)For significant service to Australia-France relations, and to the law.

Emeritus Professor Thomas (John) Boulton (MMedHum 10)For significant service to medical education, and to paediatric medicine.

Emeritus Professor Richard Laurence Broome (PhD 75)For significant service to education in the field of history, and to historical groups.

The Honourable Dr Meredith Anne Burgmann (BA 69 MA 74)For significant service to the people and Parliament of New South Wales.

Dr Graham John Faichney (MAgr 63)For significant service to science in the fields of animal nutrition and physiology.

Emeritus Professor John Joseph Fitzgerald (BA 80)For significant service to higher education, particularly in the field of Chinese studies.

Dr Robyn Rae Iredale (BA 66 DipEd 67)For significant service to people with an intellectual disability, and to education.

Mr David Harley Jacobs (BEc 74)For significant service to Australia-Japan relations, and to business.

Professor Margaret Anne Jolly (BA 70 PhD 80)For significant service to education, particularly to gender and Pacific studies.

Dr David Ronald Leece PSM RFD ED (BScAgr 64 MAgr 68)For significant service to the environment, and to defence and security studies.

Dr Qazi Ashfaq Ahmad (PhD 76)For service to the Muslim community, and to interfaith relations.

Mrs Diana Joy Alexander (Med 99)For service to education, and to the community of Lockhart.

Dr Ian Melvyn Chung (MBBS 63)For service to the law, to medicine, and to the community.

Mrs Robyn Madelon Claydon (MA 79)For service to education, and to the Anglican Church of Australia.

Dr Phillip Seldon Cocks (MBBS 73)For service to medicine, and to medical associations.

Ms Marie Ann Ficarra (BSc 76)For service to the people and Parliament of New South Wales.

Mr Alan Claude Locke (BE(Mech) 63)For service to the community through charitable organisations.

Mr Robert Peter Selinger (BA 69 Med 75)For service to the community, and to education.

Mr John Stewart Stoddart (BArch 59)For service to the performing arts as a designer.

Dr Mark Tredinnick (BA 84 LLB 86)For service to literature, and to education.

Dr Treve Williams (BVSc 65)For service to veterinary science.

Ms Vicki Telfer (MPA 02 MAdminLawPol 10)For outstanding public service to industrial relations policy and reform in New South Wales.

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Australia Day Honours list celebrates staff and alumni - News - The University of Sydney

Current research: 2020 Latest Report on Exosome Diagnostics Market Report Technologies, Analyze the Pipeline Landscape and Key Companies – WhaTech…

Exosome Diagnostics Market Report analysis including industry Overview, Country Analysis, Key Trends, Key Retail Innovations, Competitive Landscape and Sector Analysis for upcoming years.

ReportsnReports added a new report on The Exosome Diagnostics Market Technologies report delivers the clean elaborated structure of the Report comprising each and every business related information of the market at a global level. The complete range of information related to the Exosome Diagnostics Market Technologies is obtained through various sources and this obtained the bulk of the information is arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis to generate a whole set of trade based study regarding the Exosome Diagnostics Market Technologies.

Download a Free PDF Sample of Exosome Diagnostics Market Technologies Research Report at:

http://www.reportsnreports.com/contactme=1781607

Top Companies mentioned in this report are Capricor Therapeutics Inc, Evox Therapeutics Ltd, ReNeuron Group Plc, Stem Cell Medicine Ltd, Tavec Inc, Codiak Biosciences Inc, Therapeutic Solutions International Inc, ArunA Biomedical Inc, Ciloa 85.

This latest report is on Exosome Diagnostics Market Technologies which explores the application of exosome technologies within the pharmaceutical and healthcare industries. Exosomes are small cell-derived vesicles that are abundant in bodily fluids, including blood, urine and cerebrospinal fluid as well as in in vitro cell culture.

These vesicles are being used in a variety of therapeutic applications, including as therapeutic biomarkers, drug delivery systems and therapies in their own right. Research within this area remains in the nascent stages, although a number of clinical trials have been registered within the field.

Exosomes have several diverse therapeutic applications, largely centering on stem cell and gene therapy.

Exosomes have been identified as endogenous carriers of RNA within the body, allowing for the intracellular transportation of genetic material to target cells.

As such, developers have worked to engineer exosomes for the delivery of therapeutic miRNA and siRNA-based gene therapies. As RNA is highly unstable within the body, a number of different biological vector systems have been developed to enhance their transport within the circulation, including viruses and liposomes.

Similarly, exosomes derived from stem cells have also been identified for their therapeutic applications, particularly in the treatment of cancer and cardiovascular disease. Exosome technologies offer several advantages over existing biologic-based drug delivery systems.

Reasons to buy this Report:

Develop a comprehensive understanding of exosome technologies and their potential for use within the healthcare sector, Analyze the pipeline landscape and gain insight into the key companies investing in exosomes technologies, Identify trends in interventional and observational clinical trials relevant to exosomes.

Get this Report @ http://www.reportsnreports.com/purchasme=1781607

Scope of this Report:

What are the features of the exosome lifecycle?,How are therapeutic exosomes prepared?,How do exosome therapies in development differ in terms of stage of development, molecule type and therapy area?,Which companies are investing in exosome technologies?,How many clinical trials investigate exosomes as biomarkers, therapeutics and vectors?

Table of contents for Exosome Diagnostics Market Technologies:

1 Table of Contents 4

1.1 List of Tables 6

1.2 List of Figures 7

2 Exosomes in Healthcare 8

2.1 Overview of Exosomes 8

2.2 Drug Delivery Systems 9

2.2.1 Modified Release Drug Delivery Systems 9

2.2.2 Targeted Drug Delivery Systems 10

2.2.3 Liposomes 12

2.2.4 Viruses 14

2.2.5 Exosomes 17

2.3 The Exosome Lifecycle 18

2.4 Exosomes in Biology 18

2.5 Exosomes in Medicine 19

2.5.1 Biomarkers 19

2.5.2 Vaccines 20

2.6 Exosomes as a Therapeutic Target 20

2.7 Exosomes as Drug Delivery Vehicles 21

2.8 Therapeutic Preparation of Exosomes 21

2.8.1 Isolation and Purification 22

2.8.2 Drug Loading 22

2.8.3 Characterization 23

2.8.4 Bioengineering 23

2.8.5 Biodistribution and In Vivo Studies 23

2.8.6 Advantages of Exosome Therapies 24

2.8.7 Disadvantages of Exosome Therapies 24

2.9 Exosomes in Therapeutic Research 25

2.9.1 Exosome Gene Therapies 25

2.9.2 Exosome in Stem Cell Therapy 26

2.10 Exosomes in Oncology 27

2.10.1 Immunotherapy 27

2.10.2 Gene Therapy 28

2.10.3 Drug Delivery 29

2.10.4 Biomarkers 30

2.11 Exosomes in CNS Disease 30

2.11.1 Tackling the Blood-Brain Barrier 30

2.11.2 Exosomes in CNS Drug Delivery 31

2.11.3 Gene Therapy 32

2.12 Exosomes in Other Diseases 33

2.12.1 Cardiovascular Disease 33

2.12.2 Metabolic Disease 33

3 Assessment of Pipeline Product Innovation 36

3.1 Overview 36

3.2 Exosome Pipeline by Stage of Development and Molecule Type 36

3.3 Pipeline by Molecular Target 37

3.4 Pipeline by Therapy Area and Indication 38

3.5 Pipeline Product Profiles 38

3.5.1 AB-126 - ArunA Biomedical Inc. 38

3.5.2 ALX-029 and ALX-102 - Alxerion Biotech 39

3.5.3 Biologics for Autism - Stem Cell Medicine Ltd 39

3.5.4 Biologic for Breast Cancer - Exovita Biosciences Inc. 39

3.5.5 Biologics for Idiopathic Pulmonary Fibrosis and Non-alcoholic Steatohepatitis - Regenasome Pty 39

3.5.6 Biologic for Lysosomal Storage Disorder - Exerkine 39

3.5.7 Biologics for Prostate Cancer - Cells for Cells 40

3.5.8 CAP-2003 - Capricor Therapeutics Inc. 40

3.5.9 CAP-1002 - Capricor Therapeutics Inc. 41

3.5.10 CIL-15001 and CIL-15002 - Ciloa 42

3.5.11 ExoPr0 - ReNeuron Group Plc 42

3.5.12 MVAX-001 - MolecuVax Inc. 43

3.5.13 Oligonucleotides to Activate miR124 for Acute Ischemic Stroke - Isfahan University of Medical Sciences 44

3.5.14 Oligonucleotides to Inhibit KRAS for Pancreatic Cancer - Codiak BioSciences Inc. 44

3.5.15 Proteins for Neurology and Proteins for CNS Disorders and Oligonucleotides for Neurology - Evox Therapeutics Ltd 44

3.5.16 TVC-201 and TVC-300 - Tavec Inc. 45

4 Assessment of Clinical Trial Landscape 48

4.1 Interventional Clinical Trials 48

4.1.1 Clinical Trials by Therapy Type 48

4.1.2 Clinical Trials by Therapy Area 49

4.1.3 Clinical Trials by Stage of Development 50

4.1.4 Clinical Trials by Start Date and Status 50

4.2 Observational Clinical Trials 51

4.2.1 Clinical Trials by Therapy Type 51

4.2.2 Clinical Trials by Therapy Area 51

4.2.3 Clinical Trials by Stage of Development 52

4.2.4 Clinical Trials by Start Date and Status 53

4.2.5 List of All Clinical Trials 54

5 Company Analysis and Positioning 67

5.1 Company Profiles 67

5.1.1 Capricor Therapeutics Inc. 67

5.1.2 Evox Therapeutics Ltd 72

5.1.3 ReNeuron Group Plc 73

5.1.4 Stem Cell Medicine Ltd 77

5.1.5 Tavec Inc. 78

5.1.6 Codiak Biosciences Inc. 80

Originally posted here:

Current research: 2020 Latest Report on Exosome Diagnostics Market Report Technologies, Analyze the Pipeline Landscape and Key Companies - WhaTech...

Vizgen Launches with $14M Series A Financing Led by ARCH Venture Partners and Northpond Ventures – Yahoo Finance

Vizgen leverages validated, proprietary technology to push the boundaries of spatially resolved, single-cell transcriptomics, yielding unprecedented insight into molecular and cellular organization of both heathy and pathological tissues.

CAMBRIDGE, Mass., Jan. 30, 2020 /PRNewswire/ --Vizgen, a privately-held biotech company developing solutions for next-generation spatially resolved, single-cell transcriptomics, announced the closing of a $14 million Series A financing led by ARCH Venture Partners and Northpond Ventures. The investors have come together to accelerate productization of Vizgen's patented MERFISH (multiplexed error-robust fluorescence in situ hybridization)platform with the goal of empowering and commercializing this technology for research and development and eventual clinical use.

MERFISH is a quantitative and genome-scale multiplexed imaging technology for identifying nucleic acids in their native cellular and tissue environment [1, 2, 3]. MERFISH leverages error-correcting barcoding schemes and combinatorial labeling and imaging for near 100% detection efficiency and low false-positive rates at high throughput and low cost [1, 2, 3,4,5]. Deep profiling of RNAs in single cells in their native context by MERFISH reveals cell type, state, organization, interactions, and function within the tissue [6], providing an unprecedented window into health and disease.

The MERFISH technology was developed in the laboratory of Dr. Xiaowei Zhuang, a Howard Hughes Medical Institute Investigator and David B. Arnold Professor of Science at Harvard University. Dr. Zhuang was also the inventor of STORM, one of the first and most widely used super-resolution microscopy methods responsible for uncovering numerous novel cellular structures.

In addition to Dr. Zhuang, the co-founders of Vizgen also include David Walt, Ph.D., Core Faculty at the Wyss Institute at Harvard University, Professor of Pathology at Brigham and Women's Hospital, and Hansjrg Wyss Professor of Biologically Inspired Engineering at Harvard Medical School and Dr. Jeffrey Moffitt, Ph.D., Investigator at the Program in Cellular and Molecular Medicine at Boston Children's Hospital and an Assistant Professor in the Department of Microbiology at Harvard Medical School. Dr. Walt is a pioneer of single-molecule detection and analysis techniques and the scientific founder of Illumina Inc, Quanterix Corp, and several other cutting-edge life science companies. Dr. Walt will serve on Vizgen's Board of Directors. Dr. Moffitt co-invented MERFISH while a postdoctoral fellow in the laboratory of Dr. Zhuang and is a leader in the field of spatially resolved single-cell transcriptomics.

"MERFISH is a game-changing technology in single-cell genomics and has broad applications in both fundamental biology and medicine," said Dr. Walt. "Error detection and correction enable researchers to get the right answer. Vizgen's patented technology is critical for powering this unprecedented massively multiplexed single molecule detection capability. We are excited that this funding will help us make the technology broadly accessible to the community, greatly amplifying its impact."

"We are proud to join proven innovators like Drs. Walt and Zhuang in bringing this breakthrough technology to market," said Keith Crandell, Co-founder and Managing Director at ARCH Venture Partners. "The deep information unlocked by MERFISH exposes biology at the network level, driving insights that will help us build the future of human health."

About VizgenVizgen is a privately held biotech company developing the next generation of spatially resolved, single-cell transcriptomics technology and toolbox. The company's patented MERFISH technology enables massively multiplexed, genome-scale nucleic acid imaging with high accuracy and detection efficiency at subcellular resolution. The high throughput and resolution and the low cost per cell achieved by the technology will enable a wide range of tissue-scale basic research and development and will be instrumental to efforts to discover and map cell types and states in a range of tissues and organisms. Tools employing MERFISH will advance data-driven drug discovery and development and enable new insights for clinical pathology and diagnostics. For more information, please visit http://www.vizgen.com.

Story continues

About ARCHARCH Venture Partners is one of the largest early stage technology venture firms in the U.S. ARCH has co-founded and provided initial investments for more than 250 companies organized around innovations from research universities, national laboratories, corporate research groups and entrepreneurs. By focusing on leading scientific innovators, ARCH has been at the forefront of investing in major areas of innovation in the life sciences and physical sciences, and has helped to catalyze revolutionary advances in genomics, nanotechnology, industrial biotechnology and major disease treatments. ARCH has raised ten primary funds with combined committed capital at the time raised of approximately $4.5 billion. For more information please visit http://www.archventure.com.

About NorthpondThe round was co-led by Northpond Ventures,a global venture capital firm dedicated to science and technology.Northpond'sSharon Kedar,the Co-Founder and Partnerat the firm, will join the Vizgen team as a Board member.

Contact:Mary ConwayMConway@MKCStrategies.com516-606-6545

View original content:http://www.prnewswire.com/news-releases/vizgen-launches-with-14m-series-a-financing-led-by-arch-venture-partners-and-northpond-ventures-300996237.html

SOURCE Vizgen

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Vizgen Launches with $14M Series A Financing Led by ARCH Venture Partners and Northpond Ventures - Yahoo Finance

Global Molecular Diagnostics Market is Likely to Surpass US$ 22.5 Billion by the End of Year 2025 – ResearchAndMarkets.com – Associated Press

DUBLIN--(BUSINESS WIRE)--Jan 30, 2020--

The Molecular Diagnostics Market Share & Global Forecast, By Application, Technology, End User, Regions, Companies report has been added to ResearchAndMarkets.coms offering.

Increasing prevalence of Infectious diseases such as Influenza, HPV, Hepatitis, HIV and Tuberculosis despite rise in sanitation practices globally. In the past, antimicrobials medicines were used to fight powerful infectious disease but slowly in todays time antimicrobial agent is not able to give the desired results because the problem of drug resistant occurs in many people across the world.

Nowadays, a new diagnostic procedure is being followed to fight infectious disease like molecular diagnostic test is very effective which is quite fast and precise. The number of cancer patients is increasing very fast, so it is believed that in the coming time the molecular diagnostic test market will be growing at rapid pace. Global Molecular Diagnostics Market is likely to surpass US$ 22.5 Billion by the end of year 2025.

There are various reasons that will propel the market growth in forecast year; rising incidence rate of infectious disease, increasing incidence rate of cancer of all type, increasing people awareness regarding molecular diagnostic, rapid technological growth, widely acceptance of personalized medicine, rising healthcare infrastructure, increasing healthcare per capita expenditure across the developed and developing nation, accuracy of diagnosis, growing population of cardiovascular and neurological disorder etc. In addition, increasing prevalence of genetic disorder will further boost the market in forecast period of time.

The report titled Molecular Diagnostics Market Share & Forecast, By Application (Infectious Diseases, Blood Screening, Oncology, Genetic Testing, HLA (Tissue Typing), Microbiology, Cardiovascular Diseases, Neurological Diseases, Pharmacogenomics and Others), By Technology (PCR, Transcription-Mediated Amplification (TMA), Hybridiazation (In-situ Hybridiazation & FISH), DNA Sequencing & NGS, Microarray and Others), By End User (Hospitals & Academic Laboratories, Clinics and Commercial Laboratories, Others), By Regions [United States, Europe (Expect Russia), India, China, Japan, Brazil, South Korea, Mexico, Russia and ROW], Companies (Roche, Abbott, Myriad Genetics, Qiagen, BioMrieux and Others) provides a complete analysis of Molecular Diagnostics Market.

Market Insight by Application

The report provides comprehensive analysis of molecular diagnostic test market by application into ten parts: Infectious Diseases, Genetic Testing, Blood Screening, Oncology, HLA (Tissue Typing), Microbiology, Neurological Diseases, Pharmacogenomics, Cardiovascular Diseases, and Others. This report also provides key opportunities market and specific factors are given by each application market.

Market Insight by Technology

Here the market is fragmented into six parts; PCR, Transcription-Mediated Amplification (TMA), Hybridiazation (In-situ Hybridiazation & FISH), DNA Sequencing & NGS, Microarray and Others. Besides, many factors are analyzed that influence the growth, challenges and opportunities of market in technological context.

Market Insight by End User

The report provides complete insight of market by End User segments: Hospitals & Academic Laboratories, Clinics & Commercial Laboratories and Others. According to the publisher, Hospitals & Academic Laboratories will hold the largest market in global molecular diagnostic test market in forecast period of time.

Market Insight by Regions

This report covers the complete regional profile by 10 geographical market; United States, Europe, India, China, Japan, Brazil, South Korea, Mexico, Russia and Rest of World (ROW).

Key Topics Covered:

1. Executive Summary

2. Global Molecular Diagnostic Market

3. Market Share - Global Molecular Diagnostics

3.1 By Application

3.2 By Technology

3.3 By Countries

3.4 By Companies

4. Application - Molecular Diagnostics Market

4.1 Infectious Diseases

4.1.1 Hospital Acquired Infections (HAI)

4.1.2 HIV / HCV Testing

4.1.3 STD Testing

4.1.4 HPV Testing

4.2 Blood Screening

4.3 Oncology / Cancer

4.3.1 Breast

4.3.2 Colorectal

4.3.3 Prostate

4.3.4 Others

4.4 Genetic Testing

4.5 HLA (Tissue Typing)

4.6 Microbiology

4.7 Cardiovascular Diseases

4.8 Neurological Diseases

4.9 Pharmacogenomics

4.10 Others

5. Technology - Molecular Diagnostics Market

5.1 PCR

5.2 Transcription-Mediated Amplification (TMA)

5.3 Hybridiazation (In-situ Hybridiazation & FISH)

5.4 DNA Sequencing & NGS

5.5 Microarray

5.6 Others

6. Region - Molecular Diagnostics Market

6.1 United States

6.2 Europe

6.3 India

6.4 China

6.5 Japan

6.6 Brazil

6.7 South Korea

6.8 Mexico

6.9 Russia

6.10 Rest of World (ROW)

7. End Users - Molecular Diagnostics Market

7.1 Hospitals & Academic Laboratories

7.2 Clinics and Commercial Laboratories

7.3 Others

8. Roche Diagnostics - Company Analysis

8.1 Merger & Acquisitions

8.2 Sales Analysis

9. Abbott Laboratories - Company Analysis

9.1 Merger & Acquisitions

9.2 Sales Analysis

10. Myriad Genetics - Company Analysis

10.1 Merger & Acquisitions

10.2 Sales Analysis

11. Qiagen - Company Analysis

11.1 Merger & Acquisitions

11.2 Sales Analysis

12. BioMrieuxs Inc - Company Analysis

12.1 Merger & Acquisitions

12.2 Sales Analysis

13. Market Drivers

13.1 Various Developments in the Molecular Diagnostics Landscape

13.2 Integral to Traditional Labs

13.3 Improved Assay / Test Efficiencies

13.4 Targeting Antibiotic Resistance

13.5 Next Generation Ultrasensitive Molecular Diagnostics

13.6 Increasing Investment in Genomics & Proteomics Research

13.7 Technological Advances in Molecular Diagnostics

13.8 Increasing Acceptance of the Personalized Medicine

13.9 Growing Molecular Diagnostics for Food Safety

14. Challenges

14.1 Dearth of Trained Professionals

14.2 Regulatory Issues

14.3 Various Factors Slowing Growth of Molecular Diagnostics

14.4 Reimbursement Capabilities

14.5 Quality Checkpoints, Awareness & Acceptance

For more information about this report visit https://www.researchandmarkets.com/r/j3on5s

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CONTACT: ResearchAndMarkets.com

Laura Wood, Senior Press Manager

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Global Molecular Diagnostics Market is Likely to Surpass US$ 22.5 Billion by the End of Year 2025 - ResearchAndMarkets.com - Associated Press

Precision Medicine Leaps Ahead with Data Science: Bio-IT World 2020 to Highlight Data-Driven Approaches to Discovery – Yahoo Finance

3,200 Life Science and IT Leaders to Unite for Conference and Expo, April 21-23 in Boston

Bio-IT World 2020, the leading conference and expo uniting life science, data, informatics and IT leaders, today announced an expanded focus on the Data Science innovations that are moving precision medicine into new frontiers.

More than 3,200 such experts from pharmaceutical, biotech, healthcare and technology organizations, government and academia will converge for the 19th annual event, taking place April 21-23, 2020 in Boston.

"Data science is foundational to life science companieschanging the competencies required to compete. The opportunities to push our industry to the edge of discovery are thrilling for those organizations that embrace a collaborative, data-driven approach," said Allison Proffitt, Editorial Director, Bio-IT World.

From a plenary keynote presentation on the National Institutes of Healths (NIH) Strategic Vision for Data Science, to a new Data Science and Analytics Technologies track with Bristol-Myers Squibb, Takeda and more, the conference will highlight effective strategies, analytics and tools.

Altogether, 16 conference tracks will cover AI for Drug Discovery, Bioinformatics, Data Storage and Transport, Pharmaceutical R&D Informatics, Cancer Informatics, Genome Informatics, Clinical Research and Translational Informatics, Data and Metadata Management, Data Visualization Tools, Emerging AI Technologies, AI: Business Value Outcomes, Software Applications and Services, Data Security and Compliance, Cloud Computing, and Open Access and Collaborations, in addition to Data Science and Analytics Technologies.

Other highlights include 250+ presentations, 160+ exhibitors, Plenary Keynotes, pre-conference workshops, a Hackathon, awards, poster sessions and networking. See details at bio-itworldexpo.com.

About Cambridge Healthtech InstituteCambridge Healthtech Institute (CHI), a division of Cambridge Innovation Institute, is the preeminent life science network for leading researchers and business experts from top pharmaceutical, biotech, CROs, academia, and niche service providers. CHI is renowned for its vast conference portfolio held worldwide including PepTalk, Molecular Medicine Tri-Conference, SCOPE Summit, Bio-IT World Conference & Expo, PEGS Summit, Drug Discovery Chemistry, Biomarker World Congress, World Pharma Week, The Bioprocessing Summit, Next Generation Dx Summit, Immuno-Oncology Summit, and Discovery on Target. CHI's portfolio of products include Cambridge Healthtech Institute Conferences, Barnett International, Insight Pharma Reports, Bio-IT World, Clinical Research News and Diagnostics World.

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Contacts

Dawn Ringel781-449-8456 or dawn@ringelpr.com

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Precision Medicine Leaps Ahead with Data Science: Bio-IT World 2020 to Highlight Data-Driven Approaches to Discovery - Yahoo Finance

Strategies for Increasing the Effectiveness of Aromatase Inhibitors in | CMAR – Dove Medical Press

Giulia Grizzi,1 Michele Ghidini,2 Andrea Botticelli,3,4 Gianluca Tomasello,5 Antonio Ghidini,6 Francesco Grossi,2 Nicola Fusco,7,8 Mary Cabiddu,9 Tommaso Savio,10 Fausto Petrelli9

1Oncology Unit, Oncology Department, ASST of Cremona, Cremona, Italy; 2Oncology Unit, Internal Medicine Department, Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, Milan, Italy; 3Medical Oncology Department, SantAndrea Hospital, Rome, Italy; 4Department of Clinical and Molecular Medicine, Sapienza University of Rome, Rome, Italy; 5Oncology Unit, Niguarda Cancer Center, Grande Ospedale Metropolitano Niguarda, Milan, Italy; 6Medical Oncology Unit, Casa Di Cura Igea, Milan, Italy; 7Division of Pathology, Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, Milan, Italy; 8Department of Biomedical, Surgical and Dental Sciences, University of Milan, Milan, Italy; 9Oncology Unit, Medical Sciences Department, ASST of Bergamo Ovest, Treviglio, Italy; 10Breast Unit, ASST of Bergamo Ovest, Treviglio, Italy

Correspondence: Fausto PetrelliOncology Unit, Medical Sciences Department, ASST of Bergamo Ovest, Piazzale Ospedale 1, Bergamo 24047, Treviglio, ItalyTel +39 03 6342 4420Fax +39 03 6342 4380Email faupe@libero.it

Abstract: Neoadjuvant hormonal therapy (NEO-HT) is a possible treatment option for breast cancer (BC) patient with estrogen receptor positive (ER+) and HER2 negative (HER2-) disease. The absence of solid data on the type of drugs to be used and duration of treatment as well as lack of clear evidence of effectiveness of NEO-HT compared to chemotherapy (CT) reserve its use for patients with old age or frail conditions. However, the low pathologic complete response rate (pCR) obtained with tamoxifen or aromatase inhibitors (AIs) alone does not make NEO-HT as a suitable option for the neoadjuvant treatment of HR+ HER2-. The use of the cyclin-dependent kinase 4 and 6 (CDK 4/6) inhibitors palbociclib, ribociclib and abemaciclib of the mammalian target of rapamycin (mTOR) inhibitor everolimus and of the phosphoinositide 3 kinase (PI3K) inhibitor taselisib together with endocrine therapy (ET) has become a standard in advanced breast cancer, showing clinical effectiveness and significantly prolonging median progression-free survival compared to ET only. In the early phase disease, the use of ET together with CDK 4/6, mTOR and PI3K inhibitors is still investigational. Data from recent studies are promising even though less impressive than in metastatic setting. In this context, the use of genomic-transcriptomic tools (such as ONCOTYPE, PAM50) and the identification of novel biomarkers (ESR1, PI3Kca, PDGF-R) on tissue or with liquid biopsy could help to select patient prone to respond to endocrine-combined therapy and able to achieve pCR. With our review, we aimed at evaluating the current state of the art in the treatment of locally advanced breast cancer with NEO-HT.

Keywords: neoadjuvant endocrine therapy, breast cancer, CDK 4/6 inhibitors, mTOR inhibitors, PI3K inhibitors, aromatase inhibitors

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Strategies for Increasing the Effectiveness of Aromatase Inhibitors in | CMAR - Dove Medical Press

Gene fragment could explain link between autism and cognitive difficulties: U of T study – News@UofT

Autism is associated with brilliance as well as cognitive difficulty, but how either scenario plays out in the brain is not clear. Now a study by University of Toronto researchers has found that a tiny gene fragment impacts the brain in a way that could explain swathes of autism cases that come with mental health challenges.

Researchers led byBenjamin Blencowe, a professor of molecular genetics in the Donnelly Centre for Cellular and Biomolecular Research and Faculty of Medicine, andSabine Cordes, a senior investigator at Sinai Health Systems Lunenfeld-Tanenbaum Research Institute (LTRI), have identified a short gene segment that is crucial for brain development and information processing. Writing in the journalMolecular Cell, the researchersdescribe how an absence of this segment is sufficient to induce altered social behaviour a hallmark of autism in mice, as well as learning and memory deficits, which are seen in a subset of autism cases.

Best known for causing difficulties in social interaction and communication, autism is thought to arise from mishaps in brain wiring during development. It can strike in various ways. Those who experience it can have superior mental ability or need full-time care. Where on the autism spectrum a person falls depends in large part on their genetics, but most cases are idiopathic, or of unknown genetic origin.

Its very important to understand the mechanisms that underlie autism, especially in idiopathic forms where it is not clear what the underlying causes are, saysThomas Gonatopoulos-Pournatzis, a research associate in Blencowes lab and lead author of the study. Not only have we identified a new mechanism that contributes to this disorder, but our work may also offer a more rational development of therapeutic strategies.

Blencowes team had previously uncovered a link between autism and short gene segments, known as microexons, that are predominantly expressed in the brain. Through a process known as alternative splicing, microexons are either spliced in or left out from the final gene transcript before it is translated into a protein. Although small, microexons can have dramatic effects by impacting a proteins ability to bind its partners as required during brain development. However, how individual microexons contribute to autism is not clear.

The team focused on a specific microexon located in a gene known as eIF4G, which is critical for protein synthesis in the cell. They found that this microexon is overwhelmingly excluded from eIF4G gene transcripts in the brains of autistic individuals.

Hippocampal neurons from a normal mouse (above) and a mouse bred to lack the eIF4G microexon (below). The latter contains fewer particles that represent paused protein synthesis machineries. In these mice, higher levels of protein synthesis in neurons lead to disrupted brain waves and autistic-like behaviors as well as cognitive deficits down the line.

To test if the eIF4G microexon is important for brain function, Gonatopoulos-Pournatzis, together with Cordess team, bred mice that lack it. These mice showed social behaviour deficits, such as avoiding social interaction with other mice, establishing a link between the eIFG4 microexon and autistic-like behaviours.

A surprise came when the researchers found that these mice also performed poorly in a learning and memory test, which measures the animals ability to associate an environment with a stimulus.

We could not have imagined that a single microexon would have such an important impact not only on social behaviour but also on learning and memory, says Gonatopoulos-Pournatzis.

Further analysis revealed that the microexon encodes a part of eIF4G that allows it to associate with the Fragile X mental retardation protein, or FMRP, which is missing from people affected with Fragile X syndrome, a type of intellectual disability. About a third of individuals with Fragile X have features of autism but the link between the two remained unclear until now.

FMRPandeIF4G work together to act as a brake to hold off protein synthesis until new experience comes along, as the brake is removed by neural activity, the researchers also found.

Its important to control brain responses to experience, says Gonatopoulos-Pournatzis. This brake in protein synthesis is removed upon experience and we think it allows formation of new memories.

Without the microexon, however, this brake is weakened and what follows is increased protein production. The newly made proteins, identified in experiments performed withAnne-Claude Gingras, a senior investigator at LTRI and a professor in the department of molecular genetics, form ion channels, receptors and other signaling molecules needed to build synapses and for them to function properly.

However, making too many of these proteins is not a good thing because it leads to the disruption of the type of brain waves involved in synaptic plasticity and memory formation. This is revealed by electrode recordings of mouse brain slicesin experiments performed by the teams ofGraham Collingridge, a senior investigator at LTRI and a professor in the department of physiology, andMelanie Woodin, a professor of cell and systems biology at U of T and the dean of the Faculty of Arts & Science.

Moreover, an excess of similar kinds of proteins occurs in the absence of FMRP, suggesting a common molecular mechanism for Fragile X and idiopathic autism.

Researchers believe that their findings could help explain a substantial proportion of autism cases for which no other genetic clues are known. The findings also open the door to the development of new therapeutic approaches. One possibility is to increase the splicing of the eIF4G microexon in affected individuals using small molecules as a way to improve their social and cognitive deficits, Blencowe said.

The study would not have been possible without a close collaboration among multiple teams contributing diverse expertise. Blencowe and Gonatopoulos-Pournatzis also worked closely withJulie Forman-Kay, a professor of biochemistry and program head and senior scientist in the molecular medicine program at the Hospital for Sick Children, andNahum Sonenberg, a professor of biochemistry at McGill University.

The research was made possible by grants from the Canadian Institutes of Health Research, Simons Foundation and theCanada First Research Excellence Fund Medicine by Design program, among others.

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Gene fragment could explain link between autism and cognitive difficulties: U of T study - News@UofT

Link Between Autism and Cognitive Impairment Identified May Lead to New Treatments – SciTechDaily

Depicted are hippocampal neurons from a normal mouse (above) and a mouse bred to lack the eIF4G microexon (below). In the latter, there are fewer particles representing paused protein synthesis machineries. In these mice, higher levels of protein synthesis in neurons lead to disrupted brain waves and autistic behaviors as well as cognitive deficits down the line. Credit: Thomas Gonatopoulos-Pournatzis

Mouse study identifies a brain mechanism underlying social deficits and mental disability in a finding that could lead to new treatments.

Autism can bestow brilliance as well as cognitive difficulty, but how either scenario plays out in the brain is not clear. Now a study by University of Toronto researchers has found that a tiny gene fragment impacts the brain in a way that could explain swathes of autism cases that come with mental disability.

Researchers led by Benjamin Blencowe, a professor of molecular genetics in theDonnelly Centre for Cellular and Biomolecular Research, and Sabine Cordes, a senior investigator at Sinai Health Systems Lunenfeld-Tanenbaum Research Institute (LTRI), have identified a short gene segment that is crucial for brain development and information processing.Writing in the journalMolecular Cell, the researchers describe how an absence of this segment is sufficient to induce altered social behavior a hallmark of autism in mice, as well as learning and memory deficits, which are seen in a subset of autism cases.

Best known for difficulties in social interaction and communication, autism is thought to arise from mishaps in brain wiring during development. It can strike in various ways those who suffer from it can have superior mental ability or need full time care. Where on the autism spectrum a person falls depends in large part on their genetics, but most cases are idiopathic, or of unknown genetic origin.

Its very important to understand the mechanisms that underlie autism, especially in idiopathic forms where it is not clear what the underlying causes are, says Thomas Gonatopoulos-Pournatzis, a research associate in Blencowes lab and lead author of the study. Not only have we identified a new mechanism that contributes to this disorder, but our work may also offer a more rational development of therapeutic strategies.

Blencowes team had previously uncovered a link between autism and short gene segments, known as microexons, that are predominantly expressed in the brain. Through a process known as alternative splicing, microexons are either spliced in or left out from the final gene transcript before it is translated into a protein. Although small, microexons can have dramatic effects by impacting a proteins ability to bind its partners as required during brain development. However, how individual microexons contribute to autism is not clear.

The team focused on a specific microexon located in a gene known as eIF4G, which is critical for protein synthesis in the cell. They found that this microexon is overwhelmingly excluded from eIF4G gene transcripts in the brains of autistic individuals.

To test if the eIF4G microexon is important for brain function, Gonatopoulos-Pournatzis together with Cordes team bred mice that lack it. These mice showed social behaviour deficits, such as avoiding social interaction with other mice, establishing a link between the eIFG4 microexon and autistic-like behaviors.

A surprise came when the researchers found that these mice also performed poorly in a learning and memory test, which measures the animals ability to associate an environment with a stimulus.

We could not have imagined that a single microexon would have such an important impact not only on social behavior but also on learning and memory, says Gonatopoulos-Pournatzis.

Further analysis revealed that the microexon encodes a part of eIF4G that allows it to associate with the Fragile X mental retardation protein, or FMRP, which is missing from people affected with Fragile X syndrome, a type of intellectual disability. About a third of individuals with Fragile X have features of autism but the link between the two remained unclear until now.

eIF4G and FMRP associate in a complex that acts as a brake to hold off protein synthesis until new experience comes along, as the break is removed by neural activity, the researchers also found.

Its important to control brain responses to experience, says Gonatopoulos-Pournatzis. This brake in protein synthesis is removed upon experience and we think it allows formation of new memories.

Without the microexon, however, this brake is weakened and what follows is increased protein production. The newly made proteins, identified in experiments performed with Anne-Claude Gingras, Senior Investigator at LTRI, form ion channels, receptors and other signaling molecules needed to build synapses and for them to function properly.

But, making too many of these proteins is not a good thing because this leads to the disruption of the type of brain waves involved in synaptic plasticity and memory formation, as revealed by electrode recordings of mouse brain slices, in experiments performed with the teams of Graham Collingridge, Senior Investigator at LTRI, and Melanie Woodin, a professor of cell and systems biology at U of T.

Moreover, an excess of similar kinds of proteins occurs in the absence of FMRP, suggesting a common molecular mechanism for Fragile X and idiopathic autism.

Researchers believe that their findings could help explain a substantial proportion of autism cases for which no other genetic clues are known. The findings also open the door to the development of new therapeutic approaches. One possibility is to increase the splicing of the eIF4G microexon in affected individuals using small molecules as a way to improve their social and cognitive deficits, Blencowe said.

The study would not have been possible without a close collaboration among multiple teams contributing diverse expertise. Blencowe and Gonatopoulos-Pournatzis also worked closely with Julie Forman-Kay, a professor of biochemistry and Program Head and Senior Scientist in the Molecular Medicine Program at the Hospital for Sick Children, and Nahum Sonenberg, a professor of biochemistry at McGill University.

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Link Between Autism and Cognitive Impairment Identified May Lead to New Treatments - SciTechDaily

Myriad to Announce Fiscal Second-Quarter 2020 Financial Results on February 6, 2020 – Yahoo Finance

SALT LAKE CITY, Jan. 30, 2020 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN), a leader in molecular diagnostics and precision medicine, announced that it will hold its fiscal second-quarter 2020 sales and earnings conference call with investors and analysts at 4:30 p.m. ET on Thursday, February 6, 2020. During the call, Mark C. Capone, president and CEO, and Bryan Riggsbee, CFO, will provide an overview of Myriads financial performance for the fiscal second-quarter and provide a business update.

To listen to the earnings call, interested parties in the United States may dial 800-757-5680 or +1 212-231-2938 for international callers. All callers will be asked to reference reservation number 21950986. The conference call also will be available through a live webcast and a slide presentation pertaining to the earnings call also will be available under the investor section of our website at http://www.myriad.com. A replay of the call will be available two hours after the end of the call for seven days and may be accessed by dialing 800-633-8284 within the United States or +1 402-977-9140 for international callers and entering reservation number 21950986.

About Myriad GeneticsMyriad Genetics Inc., is a leading molecular diagnostic and precision medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on five strategic imperatives: building upon a solid hereditary cancer foundation, growing new product volume, expanding reimbursement coverage for new products, increasing RNA kit revenue internationally and improving profitability with Elevate 2020. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice HRD, EndoPredict, Vectra, GeneSight, riskScore, Prolaris, Foresight and Prequel are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

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Myriad to Announce Fiscal Second-Quarter 2020 Financial Results on February 6, 2020 - Yahoo Finance

NantHealth (NASDAQ:NH) Stock Rating Lowered by ValuEngine – Riverton Roll

NantHealth (NASDAQ:NH) was downgraded by analysts at ValuEngine from a sell rating to a strong sell rating in a report issued on Tuesday, January 14th, ValuEngine reports.

Separately, Zacks Investment Research downgraded shares of NantHealth from a buy rating to a hold rating in a research note on Thursday, December 19th.

Shares of NantHealth stock opened at $1.83 on Tuesday. The firm has a market cap of $204.65 million, a P/E ratio of -2.01 and a beta of 1.52. NantHealth has a twelve month low of $0.45 and a twelve month high of $2.75. The firms 50-day simple moving average is $1.17 and its 200-day simple moving average is $0.83.

NantHealth (NASDAQ:NH) last released its quarterly earnings results on Thursday, November 7th. The company reported ($0.15) EPS for the quarter, missing the Zacks consensus estimate of ($0.07) by ($0.08). The firm had revenue of $22.36 million during the quarter, compared to analyst estimates of $23.40 million. NantHealth had a negative net margin of 105.82% and a negative return on equity of 10,106.10%. Sell-side analysts predict that NantHealth will post -0.46 earnings per share for the current year.

Institutional investors and hedge funds have recently bought and sold shares of the stock. Paloma Partners Management Co grew its holdings in NantHealth by 436.1% during the second quarter. Paloma Partners Management Co now owns 91,100 shares of the companys stock valued at $48,000 after purchasing an additional 74,108 shares during the period. Tower Research Capital LLC TRC grew its holdings in NantHealth by 778.8% during the third quarter. Tower Research Capital LLC TRC now owns 45,988 shares of the companys stock valued at $33,000 after purchasing an additional 40,755 shares during the period. Finally, Paragon Wealth Strategies LLC acquired a new position in NantHealth during the fourth quarter valued at approximately $38,000. 2.81% of the stock is owned by institutional investors and hedge funds.

About NantHealth

NantHealth, Inc, together with its subsidiaries, operates as a healthcare technology company in the United States and internationally. The company engages in converging science and technology through an integrated clinical platform to provide health information at the point of care. It develops NantHealth solutions, including molecular profiling solutions, software, and hardware systems infrastructure, which integrates patient data management, bioinformatics, and molecular medicine to enable value-based care and evidence-based clinical practice.

Read More: What does cost of debt say about a companys financial health?

To view ValuEngines full report, visit ValuEngines official website.

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NantHealth (NASDAQ:NH) Stock Rating Lowered by ValuEngine - Riverton Roll

Seattle-based genome scientist gets to play with DNA – Seattle Times

Meet Lea Starita, a Seattle-based genome scientist who is working to understand how our individual genes impact our health. Here, she answers some questions about her work.

What do you do? I am a research assistant professor of genome sciences at the University of Washington School of Medicine, and I co-direct the advanced technology lab at the Brotman Baty Institute for Precision Medicine (or the BAT-lab).

One of the main goals for precision medicine is to be able to practice genome-guided medicine. However, as a field, we are really good at reading DNA sequences of people, but we are really bad at understanding the health risks or benefits associated with any given DNA change. At the BAT-lab, we are developing production-scale molecular-profiling technologies that we hope will accelerate our understanding of the impact of genetic variation on human development and human health, and in treating disease.

The BAT-lab team also helped to build the worlds premier respiratory pathogen surveillance platform as part of the Seattle Flu Study.

How did you get started in this specialty?Ive been on this trajectory since I took an awesome molecular biology lab class in college. They handed us the New England Biolabs catalog to use instead of a textbook. To this day, that catalog contains nearly any enzyme you could need for cutting and pasting pieces of DNA together. I fell in love with the puzzle posed by molecular biology.

Whats a typical day like?I am lucky enough to get to spend the day with my colleagues, collaborators, staff and trainees, who are all brilliant and creative scientists. My favorite times are when someone is at the white board drawing up a new idea or an improvement on an old idea. On the best days, I actually get in the lab to do some molecular biology myself. I love to play with DNA.

Whats the best part of the job?Dreaming up new technologies to answer tough biological questions with students, staff and collaborators. We try to answer questions like these: What is unique about each of the cell types in a human or animal? How do we understand the effect of a small change in a human or viral genome on health and disease? Although, I even find small process improvements exciting.

What surprises people about what you do? They are surprised when we talk about how important creativity and communication are in being successful as a scientist. I think people think we are these nerdy automatons, and that is totally not true. Well, the automaton part isnt true, anyway.

Do you have a cool job or know someone in the Seattle area who does? Email Michelle Archer with your recommendations for people to feature in Cool Job.

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Seattle-based genome scientist gets to play with DNA - Seattle Times

Precaution only weapon to fight against emerging threat of coronavirus – The Nation

Karachi - Prof. Dr. M. Iqbal Choudhary, Director of International Center for Chemical and Biological Sciences (ICCBS), University of Karachi has said that precaution is the only weapon to fight against the emerging threat of the 2019 novel coronavirus (2019-nCoV). There is no pathological laboratory performing a diagnostic test in Pakistan for the confirmation of coronavirus infections, he lamented.

He was speaking at a meeting held at the National Institute of Virology, which works under the Dr. Panjwani Center for Molecular Medicine and Drug Research (PCMD), University of Karachi, on Tuesday. The meeting was also attended by Dr. Muhammad Rashid, senior research officer of the institute of virology, and other scientists.

Prof. Iqbal Choudhary expressed serious concerns over the media reports of the cases of corona viruses in Pakistan, pointing out that a recent cluster of pneumonia cases in Wuhan province of China was caused by the 2019 novel coronavirus.

He said that there was a need to monitor and control all immigrants and visitors, livestock and goods at entry points. People at all international airports should be scanned for temperature and suspected individuals should be examined in isolation, he said.

He said that the National Institute of Virology was working to produce quality researches and researchers who could play their due role in the area of research and development in the country.

Dr. Rashid, in the meeting, said that there were only seven coronaviruses known to infect human, the well-known examples of human coronaviruses were SARS and MERS both viral outbreak had caused significant life losses at their time of outbreaks.

Coronaviruses are group of viruses that generally exist and maintain their life cycle in animals like camels, bats, cats, snakes and other wild animals, he said, adding that. SARS has caused 774 deaths in southern China in 2002-3 and MERS was first identified in 2012 in Saudi Arabia has caused 858 deaths. The newly emerged coronavirus named as 2019-nCoV is the third highly-virulent entry into the humans, has already caused more than 100 deaths and the death toll rises every hour in China, he said.

The 2019-nCoV infection may cause mild to severe respiratory disease, initial clinically presentation include fever, dry cough, myalgia (muscle pain) and fatigue and gradually progress into sever productive cough (a cough that produces phlegm), episodes of headache, hemoptysis (coughing up blood) and occasional diarrhea, he said.

He said that in case of suspected invasion the person should be kept in isolation and treated within the confined premises. The nCoV is highly infectious it usually infects through mouth and nose, the use of a surgical mask can minimize the risk, he maintained.

He said that there was no vaccine available against nCoV neither any anti-viral drug found effective to fight against the viral infection.

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Precaution only weapon to fight against emerging threat of coronavirus - The Nation