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Gianficaro: Rush Limbaugh’s cancer reveal brings out the darkest side of humanity – Bucks County Courier Times

February 7th, 2020 3:45 am

Two women celebrated the cancer diagnoses affecting the others political opponent, revealing the cold, cruel side to our humanity.

In the kingdom of glass, everything is transparent, and there is no place to hide a dark heart. Vera Nazarian, author

What is it that prompts the human heart to venture into the deepest corners of darkness? To descend there willingly and zealously? To encourage death to snatch life from a foe over little more than crudeness and differing points of view? To celebrate a pending death proudly, to hope for it quickly? To rationalize it as justice being the executioner?

Just what causes humanity to become so incredibly twisted and strangled by unyielding cords of hate?

On the surface, the two women Tuesday morning appeared much like any youd see sharing a table in a coffee shop. It was only when they began to speak did their words and feelings reveal the worst of who we sometimes are.

One day earlier, right-wing radio talk show host Rush Limbaugh revealed he had been diagnosed with advanced, or metastatic, lung cancer. Last month, Rep. Jerry Nadler, a Democrat and a lead House impeachment manager urging the removal of Donald Trump from office for abuse of power and obstruction of Congress, announced he would step away from the impeachment trial to be with his wife, who has metastatic pancreatic cancer.

The women, clearly on opposite ends of the political spectrum, discussed what both men are facing in their lives. My coffee went down with ease. Their conversation did not.

Limbaughs getting what he deserves, one woman said of the often caustic, occasionally unsympathetic radio host. Hes a rotten, evil man. I hate that anyone gets cancer, but theres some justice here with him. I remember he mocked Ruth Ginsburg when she got cancer. I bet its not so funny to him now.

Getting what he deserves. Getting cancer. Celebrating a mans possible, probable death. Unfathomable. As I found the distasteful opinion hard to swallow on World Cancer Day, of all days, I thought about my 88-year-old mom battling cancer today, and how the disease and the treatment are beating her up, and how I wouldnt wish the disease on anyone, especially because they wear a red tie and I wear a blue one.

Yet into the darkness the women tread. Zealously.

And then her friend followed her into the abyss. Eagerly.

Yeah, well, that Nadler is getting his, too, the other woman said. You dont survive pancreatic cancer; look at Alex Trebek. (Nadler) was part of the whole witch hunt on the president, and now this. I guess throwing Trump out of office isnt so important to him now.

Two women drinking hot coffee and revealing frozen hearts. I was hoping for better. The thaw never came.

Across the internet and on social media, cold hearts thumped out a drum beat of inhumanity, mostly against Limbaugh.

A 2006 video was recirculated of Limbaugh accusing actor Michael J. Fox of faking his symptoms of Parkinsons disease in a video ad endorsing a Democratic candidate for Senate, Claire McCaskill, who supported embryonic stem-cell research. Limbaugh also mocked Fox on air by impersonating tremors associated with the disease and charging that Fox was exaggerating them for effect. Insensitivity does not get much lower than that.

The intended message by those recirculating Limbaughs inhumanity shortly after he disclosed his cancer diagnosis was unmistakable: Paybacks are hell.

Religious scholar Reza Aslan also attacked Limbaugh, tweeting this Monday: Ask yourself this simple question: Is the world a better place or a worse place with Rush Limbaugh in it? Azlans answer was not a mystery.

I approached the two women with cold hearts. I introduced myself, told them Id overheard their conversation, and would like to talk to them about their casual discussion of cancer and death. They chided me for eavesdropping, then, predictably, refused.

While the cold hearts revealed by those women was likely an extreme perspective, it was, without question, reflective of the widening political divide in America, one in which disagreement doesnt represent the absolute ground floor. For some, it goes much deeper.

And darker.

Columnist Phil Gianficaro can be reached at 215-345-3078, pgianficaro@theintell.com, and @philgianficaro on Twitter.

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The biology, function, and biomedical applications of exosomes – Science Magazine

February 7th, 2020 3:45 am

Clinical uses of cellular communication

Exosomes are a type of extracellular vesicle that contain constituents (protein, DNA, and RNA) of the cells that secrete them. They are taken up by distant cells, where they can affect cell function and behavior. Intercellular communication through exosomes seems to be involved in the pathogenesis of various disorders, including cancer, neurodegeneration, and inflammatory diseases. In a Review, Kalluri and LeBleu discuss the biogenesis and function of exosomes in disease, highlighting areas where more research is needed. They also discuss the potential clinical applications of exosome profiling for diagnostics and exosome-mediated delivery of therapeutics to target disease cells.

Science, this issue p. eaau6977

All cells, prokaryotes and eukaryotes, release extracellular vesicles (EVs) as part of their normal physiology and during acquired abnormalities. EVs can be broadly divided into two categories, ectosomes and exosomes. Ectosomes are vesicles that pinch off the surface of the plasma membrane via outward budding, and include microvesicles, microparticles, and large vesicles in the size range of ~50 nm to 1 m in diameter. Exosomes are EVs with a size range of ~40 to 160 nm (average ~100 nm) in diameter with an endosomal origin. Sequential invagination of the plasma membrane ultimately results in the formation of multivesicular bodies, which can intersect with other intracellular vesicles and organelles, contributing to diversity in the constituents of exosomes. Depending on the cell of origin, EVs, including exosomes, can contain many constituents of a cell, including DNA, RNA, lipids, metabolites, and cytosolic and cell-surface proteins. The physiological purpose of generating exosomes remains largely unknown and needs investigation. One speculated role is that exosomes likely remove excess and/or unnecessary constituents from cells to maintain cellular homeostasis. Recent studies reviewed here also indicate a functional, targeted, mechanism-driven accumulation of specific cellular components in exosomes, suggesting that they have a role in regulating intercellular communication.

Exosomes are associated with immune responses, viral pathogenicity, pregnancy, cardiovascular diseases, central nervous systemrelated diseases, and cancer progression. Proteins, metabolites, and nucleic acids delivered by exosomes into recipient cells effectively alter their biological response. Such exosome-mediated responses can be disease promoting or restraining. The intrinsic properties of exosomes in regulating complex intracellular pathways has advanced their potential utility in the therapeutic control of many diseases, including neurodegenerative conditions and cancer. Exosomes can be engineered to deliver diverse therapeutic payloads, including short interfering RNAs, antisense oligonucleotides, chemotherapeutic agents, and immune modulators, with an ability to direct their delivery to a desired target. The lipid and protein composition of exosomes can affect their pharmacokinetic properties, and their natural constituents may play a role in enhanced bioavailability and in minimizing adverse reactions. In addition to their therapeutic potential, exosomes also have the potential to aid in disease diagnosis. They have been reported in all biological fluids, and the composition of the complex cargo of exosomes is readily accessible via sampling of biological fluids (liquid biopsies). Exosome-based liquid biopsy highlights their potential utility in diagnosis and determining the prognosis of patients with cancer and other diseases. Disease progression and response to therapy may also be ascertained by a multicomponent analysis of exosomes.

The study of exosomes is an active area of research. Ongoing technological and experimental advances are likely to yield valuable information regarding their heterogeneity and biological function(s), as well as enhance our ability to harness their therapeutic and diagnostic potential. As we develop more standardized purification and analytical procedures for the study of exosomes, this will likely reveal their functional heterogeneity. Nonetheless, functional readouts using EVs enriched for exosomes have already provided new insights into their contribution to various diseases. New genetic mouse models with the ability for de novo or induced generation of cell-specific exosomes in health and disease will likely show the causal role of exosomes in cell-to-cell communication locally and between organs. Whether exosome generation and content change with age needs investigation, and such information could offer new insights into tissue senescence, organ deterioration, and programmed or premature aging. Whether EVs and/or exosomes preceded the first emergence of the single-cell organism on the planet is tempting to speculate, and focused bioelectric and biochemical experiments in the future could reveal their cell-independent biological functions. Single-exosome identification and isolation and cryoelectron microscopy analyses have the potential to substantially improve our understanding of the basic biology of exosomes and their use in applied science and technology. Such knowledge will inform the therapeutic potential of exosomes for various diseases, including cancer and neurodegenerative diseases.

Exosomes are extracellular vesicles generated by all cells and they carry nucleic acids, proteins, lipids, and metabolites. They are mediators of near and long-distance intercellular communication in health and disease and affect various aspects of cell biology.

The study of extracellular vesicles (EVs) has the potential to identify unknown cellular and molecular mechanisms in intercellular communication and in organ homeostasis and disease. Exosomes, with an average diameter of ~100 nanometers, are a subset of EVs. The biogenesis of exosomes involves their origin in endosomes, and subsequent interactions with other intracellular vesicles and organelles generate the final content of the exosomes. Their diverse constituents include nucleic acids, proteins, lipids, amino acids, and metabolites, which can reflect their cell of origin. In various diseases, exosomes offer a window into altered cellular or tissue states, and their detection in biological fluids potentially offers a multicomponent diagnostic readout. The efficient exchange of cellular components through exosomes can inform their applied use in designing exosome-based therapeutics.

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Maybe Memorizing the Krebs Cycle Was Worthwhile After All – Medscape

February 7th, 2020 3:44 am

Like most medical students, I struggled to memorize the Krebs cycle, the complex energy-producing process that takes place in the body's mitochondria. Rote learning of Sir Hans Krebs' eponymous cascade of reactions persists and has been cited as a waste of time in modern medical education. However, it looks like that specialized knowledge about mitochondrial structure and function may finally come in handy in the clinic.

Advances in genetics have contributed to improved diagnostic accuracy of a diverse spectrum of mitochondrial disorders. Respiratory chain, nuclear gene, and mitochondrial proteome mutations can lead to multisystem or organ-specific dysfunction.

A new potential treatment for mitochondrial disorders, elamipretide, has received orphan drug designation from the US Food and Drug Administration (FDA) and is in clinical trials sponsored by Stealth Biotherapeutics. [Dr Wilner has consulted for Stealth Biotherapeutics.] Recently I had the opportunity to interview Hilary Vernon, MD, PhD, associate professor of genetic medicine at Johns Hopkins University, Baltimore, Maryland, and an expert on mitochondrial disorders. Dr Vernon discussed her research on elamipretide as a treatment for Barth syndrome, a rare form of mitochondrial disease.

I am the director of the Mitochondrial Medicine Center at Johns Hopkins Hospital. I work with individuals from infancy through adulthood who have mitochondrial conditions. I became interested in this particular area when I was early in my pediatrics/genetics residency at Johns Hopkins and saw the toll that mitochondrial disorders took on patients' lives and the limited effective therapies. At that point, I decided to focus on patient care and research in this area.

Mitochondrial disorders can be difficult to recognize because of their inherent multisystem nature and variable presentations (even between affected members of the same family). However, there are several considerations that should raise a clinician's suspicion for a mitochondrial condition. Ascertaining a family history of disease inheritance through the maternal line can raise the suspicion for a mitochondrial DNA disorder. Identification of a combination of medical issues in different organ systems that are seemingly unrelated in an individual (ie, optic atrophy and muscle weakness or diabetes and hearing loss) can also raise suspicion for a mitochondrial condition.

Due to the nature of mitochondria as the major energy producers of the cells, high-energy-requiring tissues such as the brain and the muscles are often affected. Perhaps the best known mitochondrial diseases to neurologists are MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke) as well as MERFF (myoclonic epilepsy with ragged red fibers). There is a nice body of literature on the effects of arginine and citrulline in modifying stroke-like episodes in MELAS, and this is a therapy that is in current practice.

Mitochondria are complex organelles whose structure and function are encoded in hundreds of genes originating from both the nucleus of the cell and the mitochondria themselves. Mitochondria have many key roles in cellular function, including energy production through the respiratory chain, coordination of apoptosis, nitrogen metabolism, fatty acid oxidation, and much more.

Various cofactors and vitamins can be employed to improve mitochondrial function for different reasons. For example, if a specific enzyme is dysfunctional, supplying the cofactor for that enzyme may improve its function (ie, pyruvate dehydrogenase and thiamine). Antioxidants have also been considered to help reduce the oxidant load that could potentially cause ongoing damage to the mitochondrial membrane resulting from respiratory chain dysfunction (ie, coenzyme Q-10).

It is important to remember that the highest number of individual mitochondrial disorders result from mutations in genes located in the nuclear DNA. For example, the TAZ gene that is abnormal in Barth syndrome is a nuclear gene located on the X chromosome. These genes are amenable to the "regular" approaches to gene therapy.

Targeting mitochondrial DNA for gene therapy requires a different set of approaches because the gene delivery has to overcome the barrier of the mitochondrial membranes. However, research is ongoing to overcome these obstacles.

Barth syndrome is a very rare genetic X-linked disorder that usually only affects males. The genetic defect leads to an abnormal composition of cardiolipin on the inner mitochondrial membrane. Cardiolipin is an important phospholipid involved in many mitochondrial functions, including organization of inner mitochondrial membrane cristae, involvement in apoptosis, and organization of the respiratory chain (which is responsible for producing ATP via the process of oxidative phosphorylation), and many of these functions are abnormal in Barth syndrome. Individuals with Barth syndrome typically have early-onset cardiomyopathy, myopathy, intermittent neutropenia, fatigue, poor early growth, among other health concerns.

Early in my post-residency career, I followed several patients with Barth syndrome and was quickly welcomed into the Barth syndrome community by the families and the Barth Syndrome Foundation. From there, I founded the only interdisciplinary Barth syndrome clinic in the US and began to focus a significant amount of my clinical and laboratory research on this condition.

Most commonly, these individuals come to medical attention because of cardiomyopathy, but a minority of patients do come to attention due to repeated infections and neutropenia. Patients were identified for study participation through the Barth Syndrome Foundation or because they were already patients of my study team.

All participants were known to have Barth syndrome prior to study entry, and all had confirmatory genetic testing showing a pathogenic mutation in the TAZ gene.

By binding to cardiolipin in the inner mitochondrial membrane, elamipretide is believed to stabilize cristae architecture and electron transport chain structure during oxidative stress. I thought it would be great if this could help to stabilize the abnormal cardiolipin components on the inner mitochondrial membrane in Barth syndrome.

We observed improvements in several areas across the study population in the open-label extension part of the study. This includes a significant improvement in exercise performance (as measured by the 6-minute walk test, with an average improvement of 95.9 meters at 36 weeks) and a significant improvement in muscle strength. We also observed a potential improvement in cardiac stroke volume. Most of the adverse events were local injection-site reactions and were mild to moderate in nature.

The TAZPOWER trial has an ongoing open-label extension with the same endpoints as the placebo-controlled portion evaluated on an ongoing basis. In addition, in my laboratory, we are using induced pluripotent stem cells to learn more about how cardiolipin abnormalities affect different cell types in an effort to understand the tissue specificity of disease. This will help us to understand whether different aspects of Barth syndrome would necessitate individual management or clinical monitoring strategies.

Mitochondrial inner membrane dysfunction is increasingly recognized as a major aspect of the pathology of a wide range of mitochondrial conditions. Therefore, based on the role of stabilizing mitochondrial membrane components, elamipretide has a potential role in many disorders of the mitochondria.

Yes, this is what we would call "secondary mitochondrial dysfunction" (meant to differentiate from "primary mitochondrial disease," which is caused by defects in genes that encode for mitochondrial structure and function). Approaches intended to protect the mitochondria from further damage, such as antioxidants or strategies that can bypass the mitochondria for ATP production, could overlap as treatment for primary mitochondrial disease and secondary mitochondrial dysfunction.

This is something that is much discussed as a newer consideration for families who are affected by disorders of the mitochondrial DNA, but not something I have experience with firsthand.

Yes. The United Mitochondrial Disease Foundation and the Mitochondrial Medicine Society collaborated to develop the Mito Care Network, with 19 sites identified as Mitochondrial Medicine Centers across the US.

Andrew Wilner is an associate professor of neurology at the University of Tennessee Health Science Center in Memphis, a health journalist, and an avid SCUBA diver. His latest book is The Locum Life: A Physician's Guide to Locum Tenens.

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AI, 5G, and IoT can help deliver the promise of precision medicine – VentureBeat

February 7th, 2020 3:44 am

This article is part of the Technology Insight series, made possible with funding from Intel.

When my son was a toddler, he went to his pediatrician for a routine CAT scan. Easy stuff. Just a little shot to subdue him for a few minutes. Hed be awake and finished in a jiffy.

Except my son didnt wake up. He lay there on the clinic table, unresponsive, his vitals slowly falling. The clinic had no ability to diagnose his condition. Five minutes later, he was in the back of an ambulance. My wife and I were powerless to do anything but look on, frantic with worry for our boys life.

It turned out that hed had a bad reaction to a common hydrochloride sedative. Once that was figured out, doctors quickly brought him back around, and he was fine.

But what if, through groundbreaking mixtures of compute, database, and AI technologies, a quick round of analyses on his blood and genome could have revealed his potential for such a reaction before it became a critical issue?

What if it were possible to devise a course of treatment specific to him and his bodys unique conditions, rather than accepting a cookie-cutter approach and dealing with the ill effects immediately after?

And what if that could be done with small, even portable medical devices equipped with high-bandwidth connectivity to larger resources?

In short, what if, through the power of superior computing and next-generation wireless connectivity, millions of people like my son could be quickly, accurately treated on-site rather than endure the cost and trauma of legacy medical methods?

These questions I asked about my son are at the heart of todays efforts in precision medicine. Its the practice of crafting treatments tailored to individuals based on their characteristics. Precision medicine spans an increasing number of fields, including oncology, immunology, psychiatry, and respiratory disorders, and its back end is filled with big data analytics.

Key Points:

Pairing drugs to gene characteristics only covers a fraction of the types of data that can be pooled to target specific patient care.

Consider the Montefiore Health System in the Bronx. It has deployed a semantic data lake, an architecture for collecting large, disparate volumes of data and collating them into usable forms with the help of AI. Besides the wide range of data specific to patients collected onsite (including from a host of medical sensors and devices), Montefiore healthcare professionals also collate data from sources as needed, including PharmGKB databank (genetic variations and drug responses), the National Institute of Healths Unified Medical Language System (UMLS), and the Online Mendelian Inheritance in Man (human genomic data).

Long story short, the Intel/Cloudera/Franz-based solution proved able to accurately create risk scores for patients, predict whether they would have a critical respiratory event, and advise doctors on what actions to take.

We are using information for the most critically ill patients in the institution to try and identify those at risk of developing respiratory failure (so) we can change the trajectory, noted Dr. Andrew Racine, Montefiores system SVP and chief medical officer.

Now that institutions like Montefiore can perform AI-driven analytics across many databases, the next step may be to integrate off-site communications via 5G networking. Doing so will enable physicians to contribute data from the field, from emergency sites to routine in-home visits, and receive real-time advice on how to proceed. Not only can this enable healthcare professionals to deliver faster, more accurate diagnoses, it may permit general physicians to offer specialized advice tailored to a specific patients individual needs. Enabling caregivers like this with guidance from afar is critical in a world that, researchers say, faces a shortage 15 million healthcare workers by 2030.

Enabling services like these is not trivial in any way. Consider the millions of people who might need to be genetically sequenced in order to arrive at a broad enough sample population for such diagnostics. Thats only the beginning. Different databases must be combined, often over immense distances via the cloud, without sacrificing patients rights or privacy. Despite the clear need for this, according to the Wall Street Journal, only 4% of U.S. cancer patients in clinical trials have their genomic data made available for research, leaving most treatment outcomes unknown to the research and diagnostic communities. New methods of preserving patient anonymity and data security across systems and databases should go a long way toward remedying this.

One promising example: using the processing efficiencies of Intel Xeon platforms in handling the transparent data encryption (TDE) of Epic EHR patient information with Oracle Database. Advocates say the more encryption and trusted execution technologies, such as SGX, can be integrated from medical edge devices to core data centers, the more the public will learn to allow its data to be collected and used.

Beyond security, precision medicine demands exceptional compute power. Molecular modeling and simulations must be run to assess how a drug interacts with particular patient groups, and then perhaps run again to see how that drug performs the same actions in the presence of other drugs. Such testing is why it can take billions of dollars and over a decade to bring a single drug to market.

Fortunately, many groups are employing new technologies to radically accelerate this process. Artificial intelligence plays a key role in accelerating and improving the repetitive, rote operations involved in many healthcare and life sciences tasks.

Pharmaceuticals titan Novartis, for example, uses deep neural network (DNN) technology to accelerate high-content screening, which is the analysis of cellular-level images to determine how they would react when exposed to varying genetic or chemical interactions. By updating the processing platform to the latest Xeon generation, parallelizing the workload, and using tools like the Intel Data Analytics Acceleration Library (DAAL) and Intel Caffe, Novartis realized nearly a 22x performance improvement compared to the prior configuration. These are the sorts of benefits healthcare organizations can expect from updating legacy processes with platforms optimized for acceleration through AI and high levels of parallelization.

Interestingly, such order-of-magnitude leaps in capability, while essential for taming the torrents of data flowing into medical databases, can also be applied to medical IoT devices. Think about X-ray machines. Theyre basically cameras that require human specialists (radiologists) to review images and look for patterns of health or malady before passing findings to doctors. According to GE Healthcare, hospitals now generate 50 petabytes of data annually. A staggering 90% comes from medical imaging, GE says, with more than 97% unanalyzed or unused. Beyond working to use AI to help reduce the massive volume of reject images, and thus cut reduce on multiple fronts, GE Healthcare teamed with Intel to create an X-ray system able to capture images and detect a collapsed lung (pneumothorax) within seconds.

Simply being able to detect pneumothorax incidents with AI represents a huge leap. However, part of the projects objective was to deliver accurate results more quickly and so help to automate part of the diagnostic workload jamming up so many radiology departments. Intel helped to integrate its OpenVINO toolkit, which enables development of applications that emulate human vision and visual pattern recognition. Those workloads can then be adapted for processing across CPUs, GPU, AI-specific accelerators and other processors.

With the optimization, the GE X-ray system performed inferences (image assessments) 3.3x faster than without. Completion time was less than one second per image dramatically faster than highly trained radiologists. And, as shown in the image above, GEs Optima XR240amx X-ray system is portable. So this IoT device can deliver results from a wide range of places and send results directly to doctors devices in real time over fast connections, such as 5G. A future version could feed analyzed X-rays straight into patient records. There, they become another factor in the multivariate pool that constitutes the patients dataset, which in turn, enables personalized recommendations by doctors.

By now, you see the problem/solution pattern:

The U.S. provides a solid illustration of the impact of population in this progression. According to the U.S. Centers for Disease Control (CDC), even though the rate of new cancer incidents has flattened in the last several years, the countrys rising population pushed the number of new cases diagnosed from 1.5 million in 2010 to 1.9 million in 2020, driven in part by rising rates in overweight, obesity, and infections.

The white paper Accelerating Clinical Genomics to Transform Cancer Care (below) paints a stark picture of the durations involved in traditional approaches to handling new cancer cases from initial patient visit to data-driven treatment.

At each step, delays plague the process extending patient anxiety, increasing pain, even leading to unnecessary death.

Intel created an initiative called All in One Day to create a goal for the medical industry: take a patient from initial scan(s) to precision medicine-based actions for remediation in only 24 hours. This includes genetic sequencing, analysis that yields insights into the cellular- and molecular-level pathways involved in the cancer, and identification of gene-targeted drugs able to deliver the safest, most effective remedy possible.

To make All in One Day possible, the industry will require secure, broadly trusted methods for regularly exchanging petabytes of data. (Intel notes that a typical genetic sequence creates roughly 1TB of data. Now, multiply that across the thousands of genome sequences involved in many genomic analysis operations.) The crunching of these giant data sets calls for AI and computational horsepower beyond what todays massively parallel accelerators can do. But the performance is coming.

As doctors will have to service ever-larger patient populations, expect them to need data results and visualizations delivered to wherever they may be, including in forms animated or rendered in virtual reality. This will require 5G-type wireless connectivity to facilitate sufficient data bandwidth to whatever medical IoT devices are being used.

If successful, more people will get more personalized help and relief than ever possible. The medical IoT and 5G dovetail with other trends now reshaping modern medicine and making these visions everyday reality. A 2018 Intel survey showed that 37% of healthcare industry respondents already use AI; the number should rise to 54% by 2023. Promising new products and approaches appear daily. A few recent examples are here, here and here.

As AI adoption continues and pairs with faster hardware, more diverse medical devices, and faster connectivity, perhaps we will soon reach a time when no parent ever has to watch an unresponsive child whisked away by ambulance because of adverse reactions that might have been avoided through precision medicine and next-gen technology.

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Tampa health care expert unveils health care plan using artificial intelligence, genetics, medical case management to save taxpayers $1.4 trillion per…

February 7th, 2020 3:44 am

A Tampa health care expert goes public with a plan to fix America's broken health care system.

TAMPA, Fla. (PRWEB) February 05, 2020

TAMPA, Fla. Local health care expert and medical case management consultant Paul Roberts has spent two decades working to develop more efficient ways to improve healthcare, and now the 47-year-old is attempting his most ambitious task to reform America's health care system.

"There's no question about it, our healthcare system is broken and that really is something that all Americans can get behind, regardless of their political ideologies," said Roberts. "It affects all of us and we need to address it before costs skyrocket even more."

According to Roberts, his alternative plan will save tax payers an estimated 40 percent over Medicare for All, while enhancing the overall quality of care.

Roberts calls his plan Coordinated Care for All, and while it is modeled somewhat like a single-payer government-managed healthcare system, there would be no mandate to participate. Private sector competition would continue by allowing people to opt out. The plan could also be easily adopted for use in the private sector as well.

Roberts's plan focuses on several key areas including prediction of risk, education, prevention, cost containment, efficiency, and medical case management, combining all of these target areas of focus with a centralized computer system, while utilizing artificial intelligence and voluntary genetic input to predict risk.

Coordinated Care for All would combine technology, artificial intelligence and genetics to identify and target the following:

According to Roberts, while all of this can be applied to a model for a single-payer source (Universal Healthcare), the same ideas can also be also applied to the private sector. Additionally, the allowance of pre-existing conditions and competition within the private healthcare sector are inclusive in the plan.

To promote his plan, Roberts has taken big steps. Originally, he tried working with lawmakers, but when that process resulted in slow results, he decided to invest more than $250,000 into the production of a feature-length documentary film called "Diagnosing Healthcare," which is set to be released this spring.

Since beginning his push, Roberts has been gaining momentum. The Case Management Society of America published an article focusing on his plan in their latest issue of their flagship magazine, CMSA Today. Additionally, Roberts was recently interviewed about Coordinated Care for All on American Medicine Today, and next month Roberts is scheduled to speak at the Synapse Innovation Summit at Amalie Arena in Tampa Feb 11-12.

"I realized that in order to be taken seriously, I needed to take my plan directly to the American public, so that's what I am doing," said Roberts. "At the end of the day, the focus of my plan is on improving cost-effectiveness, while also improving the quality of care delivered."

For more information on Coordinated Care for All, visit http://www.robertsccm.com.

For the original version on PRWeb visit: https://www.prweb.com/releases/tampa_health_care_expert_unveils_health_care_plan_using_artificial_intelligence_genetics_medical_case_management_to_save_taxpayers_1_4_trillion_per_year/prweb16890739.htm

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Dyne Therapeutics Expands Leadership Team with Key Hires – Business Wire

February 7th, 2020 3:44 am

WALTHAM, Mass.--(BUSINESS WIRE)--Dyne Therapeutics, a biotechnology company pioneering targeted therapies for patients with serious muscle diseases, today announced the addition of three key members to its leadership team: Oxana Beskrovnaya, Ph.D., senior vice president and head of research; Chris Mix, M.D., senior vice president, clinical development; and John Najim, vice president, chemistry, manufacturing and controls (CMC).

Dyne is establishing a leadership position in muscle disease therapeutics by combining transformative science with an organizational passion for changing the lives of patients, said Joshua Brumm, president and chief executive officer of Dyne. We are thrilled to welcome Oxana, Chris and John to our growing team. Leveraging their collective experience in the discovery and development of novel medicines, we are poised to rapidly advance our programs toward the clinic and are fully focused on execution.

Dr. Beskrovnaya is an accomplished R&D leader with a strong track record of discovering and developing first-in-class therapeutics for rare genetic diseases. Prior to joining Dyne, she served as head of musculoskeletal and renal research in Sanofis rare disease and neurological unit, where she advanced a pipeline of drug candidates using multiple therapeutic modalities, including nucleic acids, proteins and small molecules. Dr. Beskrovnaya is the author of numerous patents, invited reviews, editorials, book chapters and original research articles in major scientific journals. She received her Ph.D. in genetics from Moscow Genetics Institute, followed by postdoctoral fellowship training in neuromuscular diseases at the Howard Hughes Medical Institute at the University of Iowa.

Dr. Mix brings extensive clinical development experience to Dyne, most recently serving as vice president of rare genetic disease clinical development at Agios Pharmaceuticals, where he oversaw development across several hemolytic anemia indications. In his previous role as vice president of clinical development at Sarepta Therapeutics, he focused on advancing candidate therapies for rare neuromuscular disease. Dr. Mix received his B.A. in chemistry from Haverford College and his M.D. from the University of Massachusetts Medical School. He completed his residency in internal medicine at Tufts Medical Center, a fellowship in nephrology at the Beth Israel Deaconess Medical Center in Boston and an M.S. in clinical care research at the Tufts School of Biomedical Sciences.

Mr. Najim brings a wealth of CMC biopharmaceutical development and cGMP manufacturing experience across multiple biologic expression systems and small molecules. Mr. Najim previously held roles of increasing responsibility at Proteon Therapeutics, including most recently as vice president of manufacturing and process development, and also served as associate director of manufacturing at Dyax Corporation. He received his B.S. in biochemistry from Merrimack College and his MBA from Bentley University.

About Dyne TherapeuticsDyne Therapeutics is pioneering life-transforming therapies for patients with serious muscle diseases. The companys FORCE platform delivers oligonucleotides and other molecules to skeletal, cardiac and smooth muscle with unprecedented precision to restore muscle health. Dyne is advancing treatments for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Dyne was founded by Atlas Venture and is headquartered in Waltham, Mass. For more information, please visit http://www.dyne-tx.com, and follow us on Twitter and LinkedIn.

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Nigerias most prominent geneticists and medical research professionals are coming together to promote genomics research and make new drug discoveries…

February 7th, 2020 3:44 am

54gene has announced the launch of the African Centre for Translational Genomics (ACTG), an initiative established to facilitate translational genomics research by African scientists.

This initiative is the coming together of leading scientists as a welcome development for leveraging talent from across Nigeria to promote genomics research scholarship.

The establishment will also re-invest in the health ecosystem by empowering the next generation of African genomic scientists through the provision and implementation of grants, fellowships, internships, and training for medical researchers, trainees, and students, the company said in a statement released on Tuesday, February 4th, 2020.

According to a 2018 National Center for Biotechnology Information (NCBI) report, Nigeria has few medical geneticists and genetic counselors in Nigeria with most genetic disorders patients seen by pediatricians.

With this first concerted effort from genetic scientists and researchers, the ACTG will be funding its first study under the Non-Communicable Diseases - Genetic Heritage Study (NCD-GHS) Consortium. The consortium will see over 100,000 Nigerians participate in the eponymous study which will seek to understand the genetic basis of the highly prevalent non-Communicable Diseases (NCDs) in Nigeria such as cancers, diabetes, Alzheimers, chronic kidney, sickle cell disease, among others.

ALSO READ: Business Insider chats with the CEO of 54gene, the company building the world's first pan-African biobank

The consortium will have a steering committee co-led by the Director-General of Nigeria Institute of Medical Research [NIMR], Prof. Babatunde Lawal Salako, the Director of National Biotechnology Development Agencys Centre for Genomics Research and Innovation [NABDA-CGRI], Prof Oyekanmi Nash, the CEO of 54gene, Dr. Abasi Ene-Obong, Dr. Segun Fatumo, Assistant Professor, London School of Hygiene and Tropical Medicine [LSHTM], and Dr. Omolola Salako, Consultant Oncologist, College of Medicine, University of Lagos [CMUL].

Speaking on the consortium launch, Dr. Abasi Ene-Obong, said, In continuation with our belief at 54gene that genetic research in Africa should be ethical and beneficial to the communities we serve, and that African scientists be at the forefront of new drug discoveries that benefit Africans and the world at large; we have set up the ACTG to harness translational genetic research across Africa. The NCD-GHS study is our pilot effort under the ACTG that has the potential to rewrite the playbook of genomics research, where African scientists will be placed at the forefront of new drug discoveries for conditions that affect the health of not only Nigerians but greater Africa and the world.

Our collaboration with scientists at NIMR and NABDA-CGRI, as a consortium, is a highly welcome initiative which we believe will be a rewarding and mutually beneficial experience for all parties. For 54gene specifically, the opportunity for us to contribute to a broader national agenda for genomics research is both inspiring and humbling, and we are committed to ensuring its success.

Joining Dr. Ene-Obong at the launch of the NCD-GHS Consortium included many of Nigerias most prominent geneticists and medical research professionals.

Professor. Babatunde Lawal Salako, Director General of the Nigeria Institute of Medical Research (NIMR), added that the consortium will engage senior scientists that have made their mark in the field of cardiometabolic research in teaching hospitals across the country to ensure representativeness and quality.

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Engineering Bugs, Resurrecting Species: The Wild World of Synthetic Biology for Conservation – Singularity Hub

February 7th, 2020 3:43 am

Imagine a world where a mosquito bite is just an itchy annoyance. No malaria. No dengue fever.

Last month, scientists announced they had taken one more step toward that vision. A paper in the journal PLOS Pathogens described how they synthetically engineered mosquitoes to stop the spread of dengue fever, a viral tropical disease that sickens as many as 100 million people each year.

Now imagine genetically tweaking an invasive species of mosquito to save native Hawaiian birds from extinction, or transferring genes from one species of endangered chestnut tree to another to help the latter resist blight. Employing the same sort of genetic engineering used to make a plant-based burger bleed, scientists are beginning to explore the ways synthetic biology could help protect biodiversity and conserve species.

Synthetic biology, or synbio, employs the latest and greatest gene-editing tools, such as the cut-and-paste technology known as CRISPR-Cas9. Combined with new techniques to digitize and automate the design and modeling of various genetic elements, scientists can now engineer organisms to produce novel food ingredients or to rewire the switches that express genes that control certain functions.

In the case of those dengue-carrying mosquitoes, scientists genetically tweaked members of the Aedes aegypti species by transferring genes from the human immune system that create an antibody to suppress dengue fever into the blood-sucking insect. The antibody is activated and expressed once the female mosquito draws blood. In effect, the mosquito is cured of dengue fever before it can transmit the disease.

The next step would be to propagate the new genetic element to confer dengue immunity through a population. Thats where a gene drive comes in. Gene drive systems, which can be natural or synthetically engineered, skew inheritance of a certain genetic element so that it will spread more quickly through generations.

The idea is to bypass normal inheritance rulesthat classic Darwinian concept that inheritance is driven by genetic variations that improve an organisms ability to compete in a dog-eat-dog worldso that re-engineered traits become dominant.

In terms of conservation, synbio could potentially address several areas of concern, such as curbing invasive species, reducing pressures from wildlife trade, improving resistance to disease, and even bringing a species back from the brink of extinction.

Biologists at the University of California San Diego (UCSD), who also led the team that wrote the PLOS Pathogens paper on mosquitoes, developed a novel gene drive system for manipulating genetic inheritance in Drosophila suzukii, a fruit fly with the common name spotted-wing drosophila.

This particular pest, native to Japan and first discovered in the US in 2008, injects its eggs into soft ripening fruit like berries. Current practices to defend against spotted-wing drosophila rely on either heavy insecticide use or early harvesting. Its estimated the pest costs the US economy as much as $700 million each year in losses.

The engineered gene drive from UCSD, dubbed Medea after the character in Greek mythology that killed her offspring, uses a synthetic toxin and a corresponding antidote function to achieve 100 percent inheritance bias in less than 20 generations.

This genetic Trojan Horse could then be used to spread elements that confer susceptibility to certain environmental factors, such as triggering the death of the modified fruit flies at a certain temperature.

UC San Diego associate professor Omar Akbari told Singularity Hub that his team is getting close to field testing some of our technologies. The furthest along for our group would be the use of [precision guided sterile insect technique] to control wild populations of D. Suzuki.

A number of companies are turning to synbio to create ingredients where the natural product is expensive, rare, or threatened. Take the well-known example of vanilla. Most products on the market use a synthetic version of vanillas main ingredient, vanillin, made from petrochemicals.

Swiss company Evolva has developed a genetically modified yeast to produce vanillin in a manner similar to brewing beer. Modern Meadow also uses DNA editing tools to engineer specialized collagen-producing yeast cells for making leather products.

In a case more directly related to wildlife conservation, Singaporean scientists engineered a synthetic replacement for horseshoe crab blood cells, which have been used in biomedical applications for decades. All four species of horseshoe crabs are considered imperiled by the International Union for Conservation of Nature (IUCN).

However, while a replacement product for horseshoe crab blood has been commercially available for more than 15 years, it has yet to be broadly adopted for various reasons. Thats finally changing, as new studies have confirmed that available synthetics are just as reliable as horseshoe crab blood for detecting endotoxins in biomedical manufacturing.

The long-lived American chestnut was once one of the dominant tree species of forests in the eastern US. A blight from Asia introduced in the late 1800s has all but wiped them out. Efforts to breed American chestnuts with disease-resistant chestnut trees in China have had limited success, as its not easy to propagate the desired traits from several genes through succeeding generations.

A project led by the College of Environmental Science and Forestry in Syracuse, New York is using synbio to produce a blight-resistant American chestnut without even harming the fungus.

The researchers have copied a single gene from wheat and transferred it into American chestnuts. The gene produces an enzyme called oxalate oxidase that doesnt kill the fungus. Instead, it breaks down the fungus toxin that attacks the trees tissue properties.

The bonus is that the fungus itself is left untouched, so the blight remains dormant and doesnt evolve resistance over time.

While bringing the dead back to life is one trick that will likely elude scientists in our lifetime, synbio researchers have been actively working to resurrect the woolly mammoth and other extinct species such as the passenger pigeon, which disappeared for good more than a century ago.

These projects arent strictly creating pure examples of these long-gone species. Rather, scientists are inserting sections of ancient DNA code into modern relatives. In the case of the woolly mammoth, researchers are attempting to create a mammoth-elephant hybrid using the Asian elephant.

Proponents of this sort of resurrection science say its less about trying to revive extinct species than about saving those that are currently at risk of disappearing. The Asian elephant (Elephas maximus) is on the IUCN Red List of Threatened Species.

A team led by George Church out of Harvard University hopes that by transferring genes in the mammoth genome to the Asian elephant it will be able to survive in the Arctic; relevant genes might include those that code for extra fat and dense hair. That would extend the animals range into regions that are already changing due to a warming climate.

Like geoengineeringmanipulating the environment to stave off the effects of climate changebioengineering has its critics and detractors. Some react viscerally to the idea of altering natural systems in any way.

One of the main arguments revolves around the concern that introducing a genetically modified species could have unintended consequences. While no one expects a Jurassic Park scenario where genetically enhanced monsters chase Jeff Goldblum through the jungle, there is a chance that genetically tweaked traits could jump species or otherwise go off script.

Kent Redford believes fostering a conversation about the possible advantages and disadvantages of the role of synbio in conservation is important regardless of where one stands on the divide.

My mission is to make sure that the conservation community knows about these technologies and has taken a considered and informed opinion on them, and tried to influence [these] technologies for the good of biodiversityto minimize harm and to increase positive outcomes, he told Singularity Hub during a phone interview.

A conservation expert who has served at the The Nature Conservancy and Wildlife Conservation Society, Redford is the chair of an IUCN task force on synthetic biology and biodiversity conservation. He was the lead editor on an assessment report, Genetic Frontiers for Conservation, which will be presented this summer at the IUCN World Conservation Congress in France.

The opinion of the IUCN matters. Its 1,300 member organizations include governments, non-governmental organizations, business associations, and scientific and academic institutions.

Redford declined to speculate as to what sort of recommendations may come out of the IUCN meeting. He did note that the intersection of synbio and conservation remains on the periphery for many in the conservation community.

Most of my colleagues dont see why they should be paying much attention to this, he said. Some of those who are aware of these emerging technologies consider them to be relevant tools to help solve some of the intractable problems in conservation. Others believe these genetic techniques have the potential to completely ruin the natural world and the lives of poor people.

Akbari agreed that the biggest challenge for synbio in conservation isnt the technology but securing regulatory approvals and public support. I think we need time, he said. As more technologies are developed and tested with positive outcomesI believe the resistance will lessen.

While the scientific community debates the potential and the pitfalls of synbio, biodiversity will continue to decline.

A report last year by the United Nations Intergovernmental Science-Policy Platform on Biodiversity and Ecosystem Services issued a number of disturbing statistics. For example, the average abundance of native species in most major land-based habitats has fallen by at least 20 percent, mainly since 1900. And nearly 10 percent of all domesticated breeds of mammals humans have used for food and agriculture throughout history were extinct by 2016, with at least 1,000 more breeds still threatened.

I think the natural world is in serious trouble, Redford said. Whether synbio can be part of the answer to that problem remains a big question.

Image Credit: Image by RayNight from Pixabay

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Have humans evolved beyond nature and do we even need it? – Bywire News

February 7th, 2020 3:43 am

Manuel Berdoy, University of Oxford

UK (The Conversation) - Such is the extent of our dominion on Earth, that the answer to questions around whether we are still part of nature and whether we even need some of it rely on an understanding of what we want as Homo sapiens. And to know what we want, we need to grasp what we are.

It is a huge question but they are the best. And as a biologist, here is my humble suggestion to address it, and a personal conclusion. You may have a different one, but what matters is that we reflect on it.

Perhaps the best place to start is to consider what makes us human in the first place, which is not as obvious as it may seem.

This article is part of Lifes Big Questions The Conversations new series, co-published with BBC Future, seeks to answer our readers nagging questions about life, love, death and the universe. We work with professional researchers who have dedicated their lives to uncovering new perspectives on the questions that shape our lives.

Many years ago, a novel written by Vercors called Les Animaux dnaturs (Denatured Animals) told the story of a group of primitive hominids, the Tropis, found in an unexplored jungle in New Guinea, who seem to constitute a missing link.

However, the prospect that this fictional group may be used as slave labour by an entrepreneurial businessman named Vancruysen forces society to decide whether the Tropis are simply sophisticated animals or whether they should be given human rights. And herein lies the difficulty.

Human status had hitherto seemed so obvious that the book describes how it is soon discovered that there is no definition of what a human actually is. Certainly, the string of experts consulted anthropologists, primatologists, psychologists, lawyers and clergymen could not agree. Perhaps prophetically, it is a layperson who suggested a possible way forward.

She asked whether some of the hominids habits could be described as the early signs of a spiritual or religious mind. In short, were there signs that, like us, the Tropis were no longer at one with nature, but had separated from it, and were now looking at it from the outside with some fear.

It is a telling perspective. Our status as altered or denatured animals creatures who have arguably separated from the natural world is perhaps both the source of our humanity and the cause of many of our troubles. In the words of the books author:

All mans troubles arise from the fact that we do not know what we are and do not agree on what we want to be.

We will probably never know the timing of our gradual separation from nature although cave paintings perhaps contain some clues. But a key recent event in our relationship with the world around us is as well documented as it was abrupt. It happened on a sunny Monday morning, at 8.15am precisely.

The atomic bomb that rocked Hiroshima on August 6 1945, was a wake-up call so loud that it still resonates in our consciousness many decades later.

The day the sun rose twice was not only a forceful demonstration of the new era that we had entered, it was a reminder of how paradoxically primitive we remained: differential calculus, advanced electronics and almost godlike insights into the laws of the universe helped build, well a very big stick. Modern Homo sapiens seemingly had developed the powers of gods, while keeping the psyche of a stereotypical Stone Age killer.

We were no longer fearful of nature, but of what we would do to it, and ourselves. In short, we still did not know where we came from, but began panicking about where we were going.

We now know a lot more about our origins but we remain unsure about what we want to be in the future or, increasingly, as the climate crisis accelerates, whether we even have one.

Arguably, the greater choices granted by our technological advances make it even more difficult to decide which of the many paths to take. This is the cost of freedom.

I am not arguing against our dominion over nature nor, even as a biologist, do I feel a need to preserve the status quo. Big changes are part of our evolution. After all, oxygen was first a poison which threatened the very existence of early life, yet it is now the fuel vital to our existence.

Similarly, we may have to accept that what we do, even our unprecedented dominion, is a natural consequence of what we have evolved into, and by a process nothing less natural than natural selection itself. If artificial birth control is unnatural, so is reduced infant mortality.

I am also not convinced by the argument against genetic engineering on the basis that it is unnatural. By artificially selecting specific strains of wheat or dogs, we had been tinkering more or less blindly with genomes for centuries before the genetic revolution. Even our choice of romantic partner is a form of genetic engineering. Sex is natures way of producing new genetic combinations quickly.

Even nature, it seems, can be impatient with itself.

Advances in genomics, however, have opened the door to another key turning point. Perhaps we can avoid blowing up the world, and instead change it and ourselves slowly, perhaps beyond recognition.

The development of genetically modified crops in the 1980s quickly moved from early aspirations to improve the taste of food to a more efficient way of destroying undesirable weeds or pests.

In what some saw as the genetic equivalent of the atomic bomb, our early forays into a new technology became once again largely about killing, coupled with worries about contamination. Not that everything was rosy before that. Artificial selection, intensive farming and our exploding population growth were long destroying species quicker than we could record them.

The increasing silent springs of the 1950s and 60s caused by the destruction of farmland birds and, consequently, their song was only the tip of a deeper and more sinister iceberg. There is, in principle, nothing unnatural about extinction, which has been a recurring pattern (of sometimes massive proportions) in the evolution of our planet long before we came on the scene. But is it really what we want?

The arguments for maintaining biodiversity are usually based on survival, economics or ethics. In addition to preserving obvious key environments essential to our ecosystem and global survival, the economic argument highlights the possibility that a hitherto insignificant lichen, bacteria or reptile might hold the key to the cure of a future disease. We simply cannot afford to destroy what we do not know.

But attaching an economic value to life makes it subject to the fluctuation of markets. It is reasonable to expect that, in time, most biological solutions will be able to be synthesised, and as the market worth of many lifeforms falls, we need to scrutinise the significance of the ethical argument. Do we need nature because of its inherent value?

Perhaps the answer may come from peering over the horizon. It is somewhat of an irony that as the third millennium coincided with decrypting the human genome, perhaps the start of the fourth may be about whether it has become redundant.

Just as genetic modification may one day lead to the end of Homo sapiens naturalis (that is, humans untouched by genetic engineering), we may one day wave goodbye to the last specimen of Homo sapiens genetica. That is the last fully genetically based human living in a world increasingly less burdened by our biological form minds in a machine.

If the essence of a human, including our memories, desires and values, is somehow reflected in the pattern of the delicate neuronal connections of our brain (and why should it not?) our minds may also one day be changeable like never before.

And this brings us to the essential question that surely we must ask ourselves now: if, or rather when, we have the power to change anything, what would we not change?

After all, we may be able to transform ourselves into more rational, more efficient and stronger individuals. We may venture out further, have greater dominion over greater areas of space, and inject enough insight to bridge the gap between the issues brought about by our cultural evolution and the abilities of a brain evolved to deal with much simpler problems. We might even decide to move into a bodiless intelligence: in the end, even the pleasures of the body are located in the brain.

And then what? When the secrets of the universe are no longer hidden, what makes it worth being part of it? Where is the fun?

Gossip and sex, of course! some might say. And in effect, I would agree (although I might put it differently), as it conveys to me the fundamental need that we have to reach out and connect with others. I believe that the attributes that define our worth in this vast and changing universe are simple: empathy and love. Not power or technology, which occupy so many of our thoughts but which are merely (almost boringly) related to the age of a civilisation.

Like many a traveller, Homo sapiens may need a goal. But from the strengths that come with attaining it, one realises that ones worth (whether as an individual or a species) ultimately lies elsewhere. So I believe that the extent of our ability for empathy and love will be the yardstick by which our civilisation is judged. It may well be an important benchmark by which we will judge other civilisations that we may encounter, or indeed be judged by them.

There is something of true wonder at the basis of it all. The fact that chemicals can arise from the austere confines of an ancient molecular soup, and through the cold laws of evolution, combine into organisms that care for other lifeforms (that is, other bags of chemicals) is the true miracle.

Some ancients believed that God made us in his image. Perhaps they were right in a sense, as empathy and love are truly godlike features, at least among the benevolent gods.

Cherish those traits and use them now, Poppy, as they hold the solution to our ethical dilemma. It is those very attributes that should compel us to improve the wellbeing of our fellow humans without lowering the condition of what surrounds us.

Anything less will pervert (our) nature.

To get all of lifes big answers, join the hundreds of thousands of people who value evidence-based news by subscribing to our newsletter. You can send us your big questions by email at bigquestions@theconversation.com and well try to get a researcher or expert on the case.

More Lifes Big Questions:

Manuel Berdoy, Biologist, University of Oxford

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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In small study, hints of promise for ‘natural killer’ cell therapy – BioPharma Dive

February 7th, 2020 3:43 am

A new type of cancer cell therapy could avoid some of the serious side effects commonly associated with CAR-T treatments, and possibly offer an easier path to developing "off-the-shelf" treatments, suggest findings from a small study led by researchers at the MD Anderson Cancer Center in Houston, Texas.

The results, which were published Wednesday in the New England Journal of Medicine, are from just 11 patients. Other factors, such as the use of postremission therapy, limit what conclusions can be drawn about the researchers' approach, which relies on "natural killer" cells rather than the T cells used in cellular drugs like Novartis' Kymriah.

Still, the data offer a glimpse into why Japanese drugmaker Takedaagreed last November to license the CAR NK cell therapy from MD Anderson, part of the company's broader push into cell and gene treatments. Some of the data published Wednesday was previously disclosed by the pharma.

The success of cancer immunotherapy, of which CAR-T treatments are a major part, has put T cells at the center of a now decade-long research revival in oncology.

But T cells are only one component of the body's immune system, and scientists in academia and in biotech are exploring whether other cellular defenders could be similarly recruited.

Researchers at MD Anderson have turned to natural killer cells, which by design recognize and attack cancers or other invaders. Such cells have been tested as an anti-cancer treatment before,but using genetic engineering to improve their tumor-killing properties, which the MD Anderson team has done, is a newer innovation.

"To my knowledge, this is the largest body of evidence on the use of CAR NK cells in patients with cancer," said Katayoun Rezvani, the study's corresponding author and a professor of stem cell transplantation and cellular therapy at MD Anderson, in an interview.

Using NK cells derived from cord blood, Rezvani and her colleagues engineered the cells to express a receptor for a protein called CD19, commonly found on the surface of B-cell malignancies like leukemia and lymphoma. They also added a gene for interleukin-15 to boost the expansion and persistence of the infused NK cells, which without engineering would typically disappear after about two weeks.

While the CAR-T treatments Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) also target CD19, they are made from a patient's own T cells, which are extracted and then engineered outside the body. The personalized process is time-consuming and laborious, hampering the commercial uptake of both Kymriahand Yescarta.

By using cord blood, Rezvani and her team are pursuing an allogeneic, or "off-the-shelf," approach to cell treatment something many consider to be the next step for the field.

Initial data look promising. Seven of the 11 treated patients, who had either chronic lymphocytic leukemia or non-Hodgkin lymphoma, responded to treatment, with the cancers of three going into remission.Most notably, none experienced cytokine release syndrome or neurotoxicity, two severe side effects that commonly occur in patients treated with CAR-T therapy.

"The lack of toxicity is very exciting here," wrote Stephan Grupp, an oncologist at Children's Hospital of Philadelphia and a leader in the CAR-T field, in comments emailed to BioPharma Dive. He was not involved with the MD Anderson study.

"We really think that this is something inherent to the biology of the natural killer cells, which means their profile of toxicity is different than that of T cells,"Rezvanisaid.

Study participants did have blood toxicities that researchers associated with the chemotherapy given prior to infusion of the CAR NK cells.

While positive, the results are limited by several factors which make drawing broader conclusions about the ultimate potential of the treatment difficult.

Five of the seven responding patients received postremission treatment, including stem cell transplants, Rituxan (rituximab) and Revlimid (lenalidomide), so researchers did not assess the duration of response to CAR NK therapy.

Additionally, a fresh CAR NK cell product was manufactured for each patient in this study, rather than using the cord blood to produce multiple therapies as would be envisioned for a true off-the-shelf product.

"I think the potential for this approach to be 'off-the-shelf' is also a little speculative at this time," wrote Grupp.

"We would need to see multiple patients treated from the same expanded product with no HLAmatching to know if 'off-the-shelf' is going to be part of the story here," he added, referring to the process by which patients are matched to donor cells.

If cord blood-derived CAR NK cells were able to be given without matching to a patient's HLA genotype, any resulting treatment could be used more widely. Nine patients were partially matched in the MD Anderson study, while the last two were treated without consideration of HLA type.

The MD Anderson researchers plan to continue enrolling patients in the study and are working with Takeda to design a larger, multi-center trial.

The drugmaker is planning to advance the treatment, which it licensed and now calls TAK-007, into pivotal studies in two types of lymphoma and CLL by 2021, with a potential filing for approval in 2023.

"Targeting CD19 was a proof of concept and now that we've demonstrated that this CAR NK approach can work and is safe we want to use this platform to target other types of cancers," said Rezvani, indicating interest in multiple myeloma and acute myeloid leukemia.

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Sonoma Biotherapeutics launches with $40 million in Series A funding to advance regulatory T cell therapy in autoimmune and degenerative diseases -…

February 7th, 2020 3:43 am

Company founded by four pioneers of Treg cell biology and cell therapy and financed by a syndicate of leading biotech investors

SOUTH SAN FRANCISCO, Calif. and SEATTLE, Feb. 6, 2020 /PRNewswire/ -- Sonoma Biotherapeutics, a privately held company developing regulatory T cell (Treg) therapies for autoimmune and degenerative diseases, launched today in South San Francisco, CA and Seattle, WA with $40 million in its Series A financing. Sonoma brings together next-generation research, development and manufacturing capabilities in cell therapy and genetic engineering with an accomplished team of executives, scientists, board members and investors with extensive experience in the fields of cell therapy and drug discovery.

"With this team and our assembled expertise and technologies, we are in an ideal position to move adoptive cell therapy beyond cancer, to establish safe, effective and long-lasting treatments for a range of conditions where current drugs and biologics are simply not good enough," said founder and CEO Jeffrey Bluestone, PhD. "As the immune system's master regulators of protecting the body against self-destruction, Treg cell therapy is perhaps the ideal means to shut down unwanted immune reactions and provide meaningful treatment for patients."

The financing involves an investor syndicate that includes Lyell Immunopharma, ARCH Venture Partners, Milky Way Ventures and 8VC. "Treg therapies have the potential to transform the treatment of autoimmune and degenerative diseases," said Robert Nelsen, managing partner and co-founder of ARCH Ventures Partners. "Sonoma Biotherapeutics has assembled the team and capabilities required to make this vision a reality for patients and their families."

The goal of Treg therapy is to restore a state of self-tolerance by halting harmful inflammatory responses in autoimmune diseases such as rheumatoid arthritis, inflammatory bowel disease and multiple sclerosis, along with degenerative diseases including amyotrophic lateral sclerosis (ALS) and Alzheimer's. Over 50 million Americans currently live with an autoimmune disease, and millions more with some form of degenerative diseases. For many, existing therapies are ineffective at controlling their disease.

Tregs have a clear role in many of these conditions. These cells' natural ability to migrate to inflamed tissues and control harmful immune responses make them ideal for treating a range of conditions. In addition, the ability to engineer Treg cells to target specific disease-causing antigens reduces the potential for unwanted systemic effects. The role of Tregs in tissue maintenance and repair offers the potential for effective, durable and restorative treatments.

Sonoma Biotherapeutics is co-founded by four of the foundational scientists in the Treg field:

Collectively, the founding team brings expertise and proprietary methodologies across the Treg drug discovery and development process, including selection, manipulation, editing, regulation and translation for clinical use. Together, Drs. Bluestone and Tang have pioneered adoptive Treg cell therapy in some of its first clinical uses in type 1 diabetes, lupus and organ transplantation. Drs. Rudensky and Ramsdell co-discovered FOXP3, a critical transcription factor for Treg development and function, and in 2017 were awarded the Crafoord Prize by the Royal Swedish Academy of Sciences for their landmark studies. They are complemented by an experienced senior management team and seasoned board of directors.

"The Sonoma Biotherapeutics leadership are responsible for a significant portion of our understanding of the nature of Treg cells, their role in disease and their potential for use as a cell therapy," said Dr. Rick Klausner, CEO of Lyell Immunopharma and newly appointed Chair of the Sonoma Biotherapeutics Board of Directors. "Perhaps more importantly, they understand the requirements of a successful cell therapeutic and the corresponding challenges in defining the pathway to market. We look forward to a strong partnership between Lyell and Sonoma Biotherapeutics."

In this regard, Sonoma Biotherapeutics has entered into a strategic partnership with Lyell that provides both parties with access to technologies and know-how to enhance the durability, stability and specificity of cell therapies in their respective indications of focus. This partnership will further enable Sonoma's rapid translation of Treg therapies from target identification and discovery, through preclinical and clinical development.

Senior Management Team

Jeffrey Bluestone, PhD, Founder, CEO & PresidentFred Ramsdell, PhD, Founder & CSOPeter DiLaura, Chief Business & Strategy OfficerJoshua Beilke, MBA, PhD, VP Translational Development

Board of Directors

Rick Klausner, MD (Chair) Founder & CEO, Lyell Immunopharma, Inc.Maggie Wilderotter CEO, Grand Reserve Inn; former board member, Juno TherapeuticsToni Hoover, PhD Director, Strategy, Planning and Management for Global Health, Bill & Melinda Gates FoundationTerry Rosen, PhD CEO, Arcus BiosciencesDavid Moskowitz, PhD Principal, 8VC (observer)Jeffrey Bluestone, PhD, CEO & President, Sonoma Biotherapeutics

About Sonoma Biotherapeutics

Sonoma Biotherapeutics is a privately held, San Francisco and Seattle-based company leading the development of adoptive Treg therapies cell for autoimmune and degenerative diseases. Using next generation genome editing and target-specific cell therapy, Sonoma is focused on developing its best-in-class platform across the entire spectrum of Treg cell therapeutic capabilities. Founded by pioneers in Treg biology and cell therapy, the company brings together leading expertise and proprietary methodologies for the discovery and development of disease modifying and curative therapies.

Contact: media@sonomabio.com

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Arab Writers: The Coronavirus Is Part Of Biological Warfare Waged By The U.S. Against China – Middle East Media Research Institute

February 7th, 2020 3:43 am

Following the spread of the coronavirus in China and other countries, several writers in the Arab press wrote that this virus and others, such as the SARS and swine flu viruses, were deliberately created and spread by the U.S. in order to make a profit by selling vaccines against these diseases. Others wrote that the virus was part of an economic and psychological war waged by the U.S. against China with the aim of weakening it and presenting it as a backward country and a source of diseases.

Coronavirus sparks war between the U.S. and China (source: baladnaelyoum.com, February 2, 2020)

The following are translated excerpts from some of these articles:

Saudi Writer: It's No Coincidence That The Coronavirus Has Skipped Over Israel And The U.S.

In Saudi daily Al-Watan, writer Sa'ud Al-Shehry claimed that the coronavirus was a plot by American and Israeli drug companies aimed at increasing their profits. He wrote: "A 'wonder' virus was discovered yesterday in China; tomorrow it will be discovered in Egypt, but it will not be discovered either today, tomorrow or the day after tomorrow in the U.S. or Israel, nor in poor countries such as Burundi or the Comoro Islands

"The corona[virus] is a known virus, and we know that it was discovered in 1960 and that it causes ordinary respiratory diseases. Its symptoms are like those of any other virus: coughing, congestion, and perhaps also diarrhea and fever. [Therefore,] it is strange to hear that the World Health Organization is saying that 'this is a virus first discovered in 2012 in Saudi Arabia, in a camel...'

"And here is something else that's strange: As soon as Egypt announced, a few years ago, that it would rely on poultry [raised in the country], and that it would even export [poultry] abroad that is, that it no longer needed poultry from the U.S., France, and so on [suddenly] there appeared, from underneath the ground, the avian flu virus with the aim of nipping [Egypt's economic] awakening in the bud. Helpless, the world searched for a serum [i.e. vaccine] for this miserable avian [flu] virus. Out of the blue, like a miracle, Merck Sharp appeared like an innocent lamb, with the longed-for medicine in its hand, as if it knew nothing and as if one of its managers, Donald Rumsfeld, knew nothing and thought that the world too knew nothing. And maybe [the world] really did not know that this Donald Rumsfeld had served as [U.S.] secretary of defense for five years, into 2006. This secret member of the army brought the 'hidden' serum in the form of [the antiviral medication] Tamiflu, and thus he and his company raked in tens of billions of dollars from this miserable swine flu. The question is, what is the [U.S.] Department of Defense's connection to medical treatments?!

"Even before this, the same thing was done in China when in 2003 [the country] announced that it had [the [world's] largest dollar reserves [and] they [the Americans] introduced coronavirus' cousin, SARS, into [the country] [along with] the [vaccine] serum, [saying] 'We are the only ones who have this and you'll pay for it.' There was also the anthrax experiment, with the same company, Merck Sharp, and the same fraud and roundabout methods and it happened also with the swine flu, when Novartis and many other companies made $6 billion from this.

"Dear reader, when you read these scenarios, you will surely agree that behind the [outbreak of] corona[virus] there is a plan of deceit aimed at making a profit, and nothing more. The whole thing is a virus industry, a world of tiny creatures viruses and genetic engineering that culminate in the manufacture of a virus that is transferred to wealthy countries that can buy the [vaccine] serum. It is transferred through food, beverages, animals, the air, or perhaps via cosmetics and other means that don't come to mind. At the same time, the appropriate [vaccine] serum is being prepared for this virus, and it is held until the people need it badly because of the severity of the disease [caused by] this virus, which is genetically engineered. Then the patient grasps at any straw and pays all his money to buy this artificial treatment that was created at the same time as the virus [itself].

"And perhaps, dear reader, you will look at the statistics on the rate of contagion with the corona[virus] worldwide, and you will learn that the Gulf states hold the first places [in this list], followed by European countries, and you will never find [in these statistics] [either] the U.S. or Israel. This is a question mark that I leave for you to hypothesize about. You will also not find [the virus] in a poor country. I will solve the riddle [of why this is so], but don't tell a soul it is because [a poor country] cannot pay the price of the serum.

"Finally, rest assured that your country will pay a high price. Rest assured [also] that this is an 'ordinary' disease and not highly contagious only when [people] gather in large crowds. Long live Saudi Arabia and be strong and healthy."[1]

Syrian Writer: The Coronavirus Epidemic Is An Artificial Crisis Intended ToUndermine China's Economy

Hussein Saqer, a columnist for the Syrian daily Al-Thawra, made similar claims in a February 3, 2020 column, saying that the coronavirus was part of a commercial-biological-psychological war waged by the U.S. against China. He wrote: "From Ebola, zika, SARS, avian flu and swine flu, through anthrax and mad cow disease to the corona[virus] [all these] deadly viruses were manufactured by the U.S. and threaten to annihilate the peoples of the world. [The U.S.] has turned biological warfare into a new type of war, by means of which it intends to change the rules of play and shift the conflict with the peoples [of the world] away from the conventional path. What was reportedly said recently by the Finnish Minister of Health and Social Affairs was not fake news of the kind that features in counter-propaganda and in the tabloids. It was an authentic video with sound and image...[2] [The Finnish minister] said that the U.S. was acting to reduce the population of the world by two thirds in a way that would not cause it any losses. In fact, [the U.S.] would earn billions after forcing the World Health Organization to designate these diseases as deadly plagues so that [getting] the vaccine would be obligatory rather than voluntary, especially for the most vulnerable populations that constitute the next generation: pregnant women and children.

"The announcement of the Finnish minister firmly proves that the U.S. has a schedule for manufacturing viruses of this kind, and that the coronavirus is [another] link in the chain of deadly biological [agents] that it means to use, after mad cow disease, avian flu and the other diseases mentioned above. It embarked on this path of war after losing the commercial and financial competition, so as to punish and crush the economies of the countries that surpass it [economically],and after acting to strengthen the pharmaceutical companies owned by [its] Congressmen and ministers and placing [these companies] at the service of the vaccine industry. The World Health Organization, for its part, is willing to market the disease and the treatment together, according to the instructions of the White House, using the so-called 'good news' about new vaccines discovered for these diseases.

"The discourse, then, currently revolves around an artificial crisis of a new sort, which was created by the U.S., just like the many [other] crises it invents for its own benefit. After American economic advisors began to fear [that the U.S. would be unable] to compete with China or even match it, they came up with the virus, so as to preoccupy the Chinese officials on the one hand, and market [American] medicines and increase the panic among the Chinese people, on the other. This is therefore a war that has commercial, biological and psychological [aspects] simultaneously, and it is far removed from the conventional kind of confrontation."[3]

Egyptian Writer: The U.S. Spread The Virus To Harm China's Economy And Reputation

On the Egyptian news website Vetogate.com, Egyptian journalist Ahmad Rif'at explained why the U.S. chose the Chinese city of Wuhan as the epicenter of the disease: "American factories are the first to manufacture every kind of virus and bacteria, from the virulent smallpox virus and the bubonic plague virus to all the viruses we saw in the recent years, such as mad cow disease and swine flu. Wuhan, the city that has now been struck by the corona [disease], is an industrial town, but it is nevertheless the eighth-richest city in China, after Shanghai, Guangzhou

Guangzhou, Beijing, Tianjin and Hong Kong, which are the country's major cities. Its place at the bottom of the list [of China's major cities] is what makes it a suitable [site] for an American crime... for it is not a focus of attention, and the level of healthcare there is surely lower than in the larger and more important cities.

"All that is needed in order to let a virus spread quickly is to release it from some bag, using an ordinary syringe or in any other way. But the really interesting fact is the large number of Americans who were staying in Wuhan and decided to leave it immediately and quickly, [as was shown] on American news channels, among them a CNN reporter, even though none of them contracted [the disease]! We don't know what that [CNN reporter] was doing there. Did he come to report on the events? If so, why did he leave so dramatically? Did he come there before [the outbreak of the epidemic]? [If so,] what caused him to go there before the coronavirus crisis began?...

"This war is not only intended to worry China, trouble it and cause it to spend billions of dollars on emergency measures and medicines which, by the way, will be manufactured by an Israeli company... The U.S. wants to inform the world, and especially China itself, as part of a propaganda war targeting [China's] prestige and status, that [China] is still a backward country whose citizens eat bat soup and which exports diseases and epidemics to the rest of the world!"[4]

[1] Al-Watan (Saudi Arabia), February 2, 2020.

[2] The reference is apparently to Finnish physician and conspiracy theorist Rauni-Leena Luukanen-Kilde, who claimed that the swine flu epidemic was a hoax created by Big Pharma in order to market the vaccine, which is actually poisonous and threatens to depopulate the world.

[3] Al-Thawra (Syria), February 3, 2020.

[4] Vetogate.com, January 27, 2020.

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DNA Synthesis Market Report with Executive Summary, Introduction, Sizing, Analysis and Forecast to 2027 – Press Release – Digital Journal

February 7th, 2020 3:43 am

Future Market Insights has announced the addition of the "DNA Synthesis Market: Global Industry Analysis 2012 - 2016 and Opportunity Assessment; 2017 - 2027" report to their offering

This press release was orginally distributed by SBWire

Valley Cottage, NY -- (SBWIRE) -- 02/06/2020 -- DNA synthesis is the natural creation of nucleic acid strands through the process of DNA replication. Artificially, they are synthesized using genetic engineering and enzyme chemistry in the laboratory to be used for various applications such as therapeutic, diagnostics as well as academic and industrial research. DNA synthesis services provided by different companies varies greatly by the cost of per base pair, error rates, lengths, throughput, etc. DNA synthesis market further includes the oligonucleotide synthesis and gene synthesis which has various end users such as agricultural science, food science, antibody discovery, immunology, cancer research, infectious disease, synthetic biology. Market for the therapeutic applications is mostly distributed only among the biopharmaceutical industries which is driven by their continuous research in the respective domain.

The market for DNA synthesis got the surge form Human Genome Project leading to several advancements in the technological processes for production and reduces the time of production which made possible the synthesis of high throughput custom nucleotides. Nowadays, it is possible to do the customizations and get the required sequence online and at required time. The commercial availability of DNA synthesis machines has also a great impact in the synthesis services market.

Download Sample Copy@ https://www.futuremarketinsights.com/reports/sample/rep-gb-5900

DNA Synthesis Market: Drivers & Restraints

Growing number of research & developments in the field of genomics and next generation sequencing supports the market growth of DNA synthesis services over the globe. The growing numbers of mergers and collaborations by the market players also strengthening the market growth. Along with this, the developments in the synthetic biology segment promotes the market progression of DNA synthesis services. A robust growth in the oligonucleotide therapeutic segment as antisense oligos, siRNAs, miRNA inhibitors and mimics also supports the market growth of DNA synthesis for the commercial end. However, cuts in the federal fundings for the research purpose, stringent regulatory requirements in the therapeutic applications for DNA also limits the market to expand across the globe.

DNA Synthesis Market: Segmentation -

Segmentation by Service Type:

-Oligonucleotide Synthesis -Gene Synthesis

Segmentation by Application:

-Research and Development -Diagnosis-Therapeutics

Segmentation by End User

-Biopharmaceutical Companies-Academic and Research Institutes-Contract Research Organizations

Download Table of Contents@ https://www.futuremarketinsights.com/toc/rep-gb-5900

DNA Synthesis Market: Overview

Companies involved in the DNA synthesis services market are involved in continuous updation of their manufacturing technologies for high throughput synthesis with cost control. Recently, Twist biosciences also gathered $82 million investment from Illumina for developing a new technology platform for synthesizing DNA on silicon. These market players are also focusing on their brand improvement and market penetration by focusing on their sales force, geographical expansion as well as expansion of manufacturing facilities.

Moreover, synthetic DNA costs are anticipated to decrease owing to the introduction of advanced technology. Intensifying competition in the synthetic biology services also leads to price reduction per base pair. However, the multi-billion dollar PCR industry constantly supports the market growth of DNA synthesis services. With the increasing outsourcing services for the life science research activities, the market has huge potential of growth opportunities. The availability of research funds also had a great impact in the DNA synthesis market size and growth rate in different regions over the globe.

DNA Synthesis Market: Region-wise Outlook

Geographically, North America leads the market for DNA synthesis services owing to the high requirement in the academic research as well as biopharmaceutical industries for research and therapeutic production. This is followed by the Western Europe region supported by the high availability of research fundings in universities and commercial availability of therapeutic drugs made of DNA active pharmaceutical ingredients. Eastern Europe region shares a low market share and slow growth rate comparatively to other regions over the forecast period. Asia Pacific region represents the significant growth rate in the DNA synthesis market with highest market growth in research applications. Recent trends shows China to be leading the market in the region in terms of market size as well as growth rate. Latin America and Middle East & Africa has been observed the least market share over the forecast period.

Download Segment-wise Analysis@ https://www.futuremarketinsights.com/checkout/5900

DNA Synthesis Market Treatment Market: Key Players

Some of the players in the DNA Synthesis market includes -

-Bioneer Corporation-IBA GmbH-Eurofins Scientific-Integrated DNA Technologies Inc.-LGC Biosearch Technologies-Eton Bioscience Inc.-GenScript Biotech Corporation-Eurogentec-Thermo Fisher Scientific Inc.-Quintara Biosciences

For more information on this press release visit: http://www.sbwire.com/press-releases/dna-synthesis-market-report-with-executive-summary-introduction-sizing-analysis-and-forecast-to-2027-1274866.htm

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The global gene therapy market was valued at $393.35 million in 2018, and is estimated to reach $6205.85 million by 2026, registering a CAGR of 34.8%…

February 7th, 2020 3:41 am

NEW YORK, Feb. 6, 2020 /PRNewswire/ --

Gene Therapy Market by Vector Type (Viral Vector and Non-viral Vector), Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others), and Application (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Disease, and Other Diseases): Global Opportunity Analysis and Industry Forecast, 20192026

Read the full report: https://www.reportlinker.com/p05844072/?utm_source=PRN

The global gene therapy market was valued at $393.35 million in 2018, and is estimated to reach $6,205.85 million by 2026, registering a CAGR of 34.8% from 2019 to 2026.Gene therapy is a technique that involves the delivery of nucleic acid polymers into a patient's cells as a drug to treat diseases. It fixes a genetic problem at its source. The process involves modifying the protein either to change the genetic expression or to correct a mutation. The emergence of this technology meets the rise in needs for better diagnostics and targeted therapy tools. For instance, genetic engineering can be used to modify physical appearance, metabolism, physical capabilities, and mental abilities such as memory and intelligence. In addition, it is also used for infertility treatment. Gene therapy offers a ray of hope for patients, who either have no treatment options or show no benefits with drugs currently available. The ongoing success has strongly supported upcoming researches and has carved ways for enhancement of gene therapy.Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is on an increase, despite the limited number of products that have successfully reached the market. In addition, benefits of gene therapy over conventional cancer therapies and increase in government support fuel the growth of the gene therapy market.The gene therapy market is a widely expanding field in the pharmaceutical industry with new opportunities. This has piqued the interests of venture capitalists to explore this market and its commercial potential. Major factors that drive the growth of this market include high demands for DNA vaccines to treat genetic diseases, targeted drug delivery, and high incidence of genetic disorders. However, the stringent regulatory approval process for gene therapy and the high costs of gene therapy drugs are expected to hinder the growth of the market. On the contrary, increase in the pipeline developments for gene therapy market are expected to provide lucrative opportunity during the forecast period.The global gene therapy market is segmented based on vector type, gene type, application, and geography. Based on vector type, it is categorized into viral vector and non-viral vector. Viral vector is further segmented into retroviruses, lentiviruses, adenoviruses, adeno associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others. Non-viral vector is further categorized into naked/plasmid vectors, gene gun, electroporation, lipofection, and others. Based on gene type, the market is classified into antigen, cytokine, tumor suppressor, suicide, deficiency, growth factors, receptors, and others. Based on application, the market is divided into oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious disease, and other diseases. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

KEY MARKET BENEFITS FOR STAKEHOLDERS This report offers a detailed quantitative analysis of the current market trends from 2018 to 2026 to identify the prevailing opportunities. The market estimations provided in this report are based on comprehensive analysis of the key developments in the industry. In-depth analysis based on geography facilitates in analyzing the regional market to assist in strategic business planning. The development strategies adopted by key manufacturers are enlisted in the report to understand the competitive scenario of the market.

KEY MARKET SEGMENTS

By Vector Type Viral vectoro Retroviruseso Lentiviruseso Adenoviruseso Adeno Associated Viruso Herpes Simplex Viruso Poxviruso Vaccinia Viruso Others Non-viral vectoro Naked/Plasmid Vectorso Gene Guno Electroporationo Lipofectiono Others

By Gene Type Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Others

By Application Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

By Region North Americao U.S.o Canadao Mexico Europeo Germanyo UKo Franceo Spaino Italyo Rest of Europe Asia-Pacifico Japano Chinao Australiao Indiao South Koreao Rest of Asia-Pacific LAMEAo Brazilo South Africao Saudi Arabiao Rest of LAMEA

KEY PLAYERS PROFILED Adaptimmune Therapeutics Plc. Anchiano Therapeutics Ltd. Achieve Life Sciences, Inc. Adverum Biotechnologies, Inc. Abeona Therapeutics Inc. Applied Genetic Technologies Corporation Arbutus Biopharma Corporation, Audentes Therapeutics, Inc. AveXis, Inc. Bluebird Bio, Inc. Celgene Corporation CRISPR Therapeutics AG Editas Medicine, Inc. Editas Medicine, Inc. GlaxoSmithKline Plc. Intellia Therapeutics, Inc. Merck & Co., Inc. Novartis AG REGENXBIO Inc. Spark Therapeutics, Inc. Sangamo Therapeutics, Inc. Uniqure N. V.Voyager Therapeutics, Inc

The other players of the gene therapy market include (companies not profiled in the report): Amgen Epeius Biotechnologies Sanofi Juno Therapeutics Advantagene

Read the full report: https://www.reportlinker.com/p05844072/?utm_source=PRN

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The global gene therapy market was valued at $393.35 million in 2018, and is estimated to reach $6205.85 million by 2026, registering a CAGR of 34.8%...

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How Can We Tackle the Bottleneck in Plasmid Production for Gene… – Labiotech.eu

February 7th, 2020 3:41 am

The cell and gene therapy sector is booming. At the end of 2019, there were 1,069 registered clinical trials in the field. By 2024, the cell and gene therapy market is estimated to reach revenues of $6.6B (5.9B). But there is a problem. As a result of the rapid growth of gene and cell therapies, the demand for plasmids is skyrocketing. And the industry is struggling to meet it.

Plasmids are the key building blocks needed to manufacture viral vectors, which are the most common strategy to develop gene therapies, explains Stefano Baila, Director of Operations and Business Development at Anemocyte. Plasmids are not only crucial for the development of gene therapies, but also for therapies that involve the genetic modification of cells, such as CAR-T. This has greatly increased the demand for plasmids and I would say that the industry was probably not ready to address this demand.

While the industry is struggling with the lack of plasmids on the market, another challenge is quality. Those providers that can meet the high-quality requirements for plasmids have long waiting lists of up to 12 months, says Baila. For companies moving into phase 3 or commercial production, it becomes crucial that the good manufacturing practice (GMP) requirements are met.

But another result of the rapidly evolving gene and cell therapy space is the fact that the regulatory requirements are not quite up to scratch. The regulatory framework around plasmid production is very confusing for the industry at the moment, says Baila. The main guidelines refer to the quality of the product, but the level of quality remains open to interpretation. More clarity would definitely help once and for all to define the exact quality levels required at different stages of drug development.

Consequently, plasmid providers have to be able to address all quality levels required at each stage of drug development. As the first biotech manufacturing organization (BMO) worldwide, Italian company Anemocyte has met this challenge by focusing greatly on the industrys needs. Their keyword is flexibility.

Before starting their work on plasmid manufacturing, the team spent several months interviewing companies about their difficulties and needs regarding the bottleneck in plasmid production and regulatory issues.

For us, it was key to understand the needs of the industry and find a possible solution, Baila explains. Our research resulted in a brand new facility, which is designed with adaptable manufacturing spaces that enable flexible time management. This ensures that the manufacturing process continues to roll without creating a bottleneck.

As a next step, the Anemocyte team had to decide whether to use a classified cleanroom or just a regular lab for the manufacturing process. We decided to keep the bar pretty high, so we are working with a cleanroom facility where we apply the GMP standard, says Baila. We maintain a high quality and also address the time issues that all companies seem to share as their main challenge in cell and gene therapy development.

The flexibility of its manufacturing facility allows the Anemocyte team to easily adapt to its customers needs. Our customers have control over what we do, explains Marco Ferrari, CEO of Anemocyte. They have the opportunity to be involved in the process, and decisions and actions are discussed and shared with them to ensure their product is produced at the high standard they expect.

Moreover, Anemocyte pays attention to new technologies and innovations. The fast evolution of the cell and gene therapy industry greatly increases the demand for new solutions, Ferrari explains. Staying on top of innovation is therefore mandatory today. Our approach is to stay ahead of the trends and be capable of deploying useful solutions for our customers.

Anemocytes manufacturing facility is built in such a modular way that it can be replicated and adapted to meet the rising demand for plasmids. This, as Ferrari puts it, ensures that the Anemocyte team will not be caught off guard when more companies come knocking at our door. This is an advantage for long-term customers because the manufacturing facility can be duplicated and built according to the customers needs.

As the worlds first BMO, Anemocyte pays specific attention to innovation and the ongoing trends in the industry. One of the emerging trends is the use of nonviral vectors for the development of gene therapies.

Even in the nonviral approach, plasmids play a key role, explains Baila. A part of the nonviral strategy is the transfer of plasmids into cells via mechanical or chemical methods. So, in one way or another, plasmids will always be needed.

Our investment in the nonviral gene modification space is an example of how we are tackling potential future trends that are still under the radar or explored at an academic level, adds Ferrari.

Dont sit on a waiting list to get your plasmids produced! Get in touch with the team at Anemocyte or learn more about the company and the development of plasmids for cell and gene therapies here!

Images via Shutterstock.com

Author: Larissa Warneck, Science Journalist at Labiotech.eu

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GE Healthcare joins CMRI to optimize gene therapy manufacturing – BSA bureau

February 7th, 2020 3:41 am

The collaboration with Childrens Medical Research Institute will boost the efficiency of AAV purification, leading to increased access to the viral vectors needed to manufacture gene therapies

GE Healthcare Life SciencesandChildrens Medical Research Institutewill jointly drive the development of new affinity ligands for the purification of adeno-associated viral (AAV) vectors used in gene therapies. The focus of the collaboration is to bring to market-specific ligands for multiple AAV types, enhancing the chromatographic separation of AAV-based vectors. This will improve the manufacturing efficiency and scalability of gene therapies, enabling the availability of viral vectors on a global scale.

With more than 800 gene therapies currently in clinical trials, there is an increasing demand for the raw materials needed in the manufacturing process of viral vectors. AAVs are viral vectors used in more than 70% of the in vivo gene therapy clinical trials. According to GlobalData, the 2025 gene therapy in vivo therapeutic market is expected to reach USD 32 billion with an estimated CAGR of 105% between 2019-2025.

The collaboration combines the expertise from the latest available research on AAVs with application testing, advancing a comprehensive understanding of the clinical functionality and the commercial opportunities of AAV-based gene therapies. Childrens Medical Research Institute will share with GE Healthcare Life Sciences AAV capsid variants targeting different tissues. GE Healthcare Life Sciences will then design and test ligand prototypes, which Childrens Medical Research Institute will assess. Based on the performance results, GE Healthcare Life Sciences will manufacture and commercialize novel improved AAV affinity ligands.

Dr Leszek Lisowski, the lead gene therapy scientist at Childrens Medical Research Institute, says: Bringing the fruits of our work to the patients requires a joint effort between academia and the industry. The collaboration with GE Healthcare Life Sciences will allow us to expedite the development of novel clinical options at a lower cost.

Olivier Loeillot, General Manager, Bioprocess at GE Healthcare Life Sciences, says: The industry needs better and more personalized technologies to speed biopharmaceuticals through clinical trials and bring them to market. Our long biomanufacturing expertise combined with Childrens Medical Research Institutes pioneering research will lead to purification technologies that will streamline the production of gene therapies.

Catarina Flyborg, General Manager, Cell and Gene Therapy at GE Healthcare Life Sciences, says: Collaborations with organizations such as Childrens Medical Research Institute are critical to developing the technologies needed to move the industry forward. By working directly with world-class researchers, GE Healthcare Life Sciences can develop the purification technologies that will contribute to increasing the availability of viral vectors globally.

Childrens Medical Research Institute in Australia is globally recognized for its work on microsurgery, cancer research, neurobiology, embryology and gene therapy. The AAV affinity ligands resulting from this collaboration will be compatible with GE Healthcare Life Sciences resin-based chromatography portfolio used in the purification of most FDA-approved biopharmaceuticals.

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FDA guidance on gene therapies development and manufacturing – BioPharma-Reporter.com

February 7th, 2020 3:41 am

With more than 900 investigational new drug (IND) applications for ongoing clinical studies related to gene therapies, and with the number of advanced therapy medicinal products at clinical stage worldwide exceeding 1,000, the US Food and Drug Administration (FDA) this week released a number of policies.

The policies, addressed to developers and manufacturers, include six final guidance documents on gene therapy manufacturing and clinical development of products, following up to respective draft guidance documents released in 2018, and a draft guidance related to orphan drug designations for therapeutic candidates.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review, commented Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, adding The framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field.

Regarding the draft guidanceInterpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, the agency explained that it focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease.

The final guidance titled Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) aims to inform sponsors on how to provide sufficient CMC information, in order to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product and to be able to claim market authorization from the regulatory body.

Addressed to developers and manufacturers of retroviral vector-based human gene therapy products, the second document titled Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up determines testing for RCR during manufacture, as well as the regulations for follow-up monitoring of patients who have received such treatments.

Titled Long-Term Follow-Up After Administration of Human Gene Therapy Products, the third document includes recommendations by the FDA regarding the design of long-term follow-up studies for the collection of data on delayed adverse events.

Specifically, the FDA suggests that, as a result of long-term exposure to an investigational gene therapy, patients may be at increased risk of undesirable and unpredictable outcomes, and therefore they may be monitored for an extended period of time past the active follow-up period. The document outlines several factors based on which a risk assessment should be performed to determine the necessity of long-term monitoring for each product.

Another guidance of the FDA is focused on Human Gene Therapy for Hemophilia, and it provides recommendations regarding the clinical trial design for such therapies, as well as addressing discrepancies between Hemophilia A and B coagulation factors activity assays.

Focusing on Human Gene Therapy for Retinal Disorders, the fourth FDA guidance includes recommendations related to product development, preclinical testing, and clinical trial design for such gene therapy products.

Finally, the guidance on Human Gene Therapy for Rare Diseases, with suggestions on the clinical design for such products, is needed, according to the FDA, due to the limited study population size and potential feasibility and safety issues. Moreover, the FDA cites issues related to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the product.

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Nationwide Children’s Seeks City Tax Breaks On For-Profit Gene Therapy Facility – WOSU

February 7th, 2020 3:41 am

Nationwide Childrens Hospital is asking for tax abatements on a new for-profit gene therapy center. If granted, it would exempt the company from paying money to Columbus City Schools for 15 years.

Andelyn Biosciences has been proposed as a for-profit company that would manufacture gene therapy products.

Those products would be developed and distributed to companies that sell them for medical treatment, explains Libbey Hoang, vice president of planning and business development with Nationwide Childrens.

"Our focus will be to bring rare genetic disorder treatment for children to the market," Hoang says. "We believe that theres a great opportunity for Nationwide Childrens to be the leader in that because of our transformational science that has occurred here."

The center would be part of Ohio State Universitys West Innovation Campus, at the corner of Lane and Carmac. Hoang says it will be specifically designed for biologics manufacturing.

That combined with having to hire an expected 150 employees before they can turn a profit is why theyre asking for a 15-year, 100% tax abatement.

"The facility cost us about $64 million in improvements to construct," Hoang says. "Because the company will take approximately four years to actually then produce treatment, we will have nearly $30 million in taxable payroll with very limited income, so thats the major reason were seeking the tax abatement."

However, Nationwide Children's ask is drawing criticism.

"Our stance has always been that we oppose tax abatements for corporations that dont need them," says John Coniglio, president of the Columbus Education Association.

The teachers union has long opposed the city forfeiting tax dollars to spur development. He points to Nationwide Childrens hospitals profit margin an average of about $327 million per year.

"The first thing that comes to my mind is: Does Childrens Hospital really need this tax abatement?" he says.

The hospital runs several programs in partnership with Columbus City Schools, including STEM programs and primary care services. Nationwide Childrens is also offering an estimated $53 million in health care services in the schools during the 15-year abatement period.

"Childrens Hospital does do good things in Columbus City Schools," Coniglio says. "But my question would be: Are you doing this just because you dont want to take the risk that regular individuals have to take every time they want to open a business or do something new?"

Michael Stevens, interim director of development for the City of Columbus, says the land proposed for Andelyn Biosciences is owned by the state, so its not currently producing any tax revenue.

"Without this incentive there would not be the project," Stevens says. "And at this point this parcel does not generate any revenue for the schools or the city, and as a result of incentivizing and making this investment, then were going to see revenue coming into the income tax for the city that then we share with the schools over the 15-year period."

Stevens says the city and the schools will split land and income taxes from Andelyn Biosciences projected to be about $2.5 million each. That price is still significantly less than would have been collected if the project went through without the abatement.

To receive the tax abatement, Nationwide Childrens needs approval from both the Columbus Board of Education and Columbus City Council.

The proposal hasnt been presented to City Council, yet so member Elizabeth Brown declined to make a judgement. But Brown says its important to consider the motivation behind Andelyn Biosciences.

"The genetic disorders are so narrowly presented in the average population that pharmaceutical companies cant make money on developing those things," she says. "Which is why Childrens Hospital is going after it, to my understanding."

The school board plans to take up the abatements on Tuesday. In a statement, board president Jennifer Adair says Nationwide Children's has a strong partnership with the district, and they are considering the abatement. Council will have the final say.

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Nationwide Children's Seeks City Tax Breaks On For-Profit Gene Therapy Facility - WOSU

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Novartis and Gilead provide funding to Vineti – BioPharma-Reporter.com

February 7th, 2020 3:41 am

The two big pharma companies joined, among others, the investing group for Vineti, the developer of a digital platform for personalized therapeutics enabling the distribution of regenerative medicine, both at clinical and commercial scale.

More specifically, the cloud-based digital platform connects healthcare providers, suppliers, and biopharmaceutical developers by providing them with real-time access to data.

Focusing on personalized medicine, the platform is designed to provide simplicity to end users. Vinetis CEO, Amy DuRoss explained to us why simplicity is important for such treatments:Cell and gene therapies have many, many different types of people involved in the manufacturing and delivery process, including healthcare providers who arent typically part of manufacturing supply chains.

These unfamiliar, highly regulated processes need to be as simple for healthcare providers and other stakeholders as possible, so that these therapies can be produced for patients quickly and without error, she added.

According to the company, Vineti is the only platform of record currently enabling both clinical and commercial personalized therapeutics, such as chimeric antigen receptor (CAR) T-cell therapies and allogeneic cell therapies, into the supply chain.

Both Gilead and Novartis hold assets at commercial stage, the marketing of which can benefit from the use of the platform. Such assets are Novartis gene therapy Zolgensma (onasemnogene abeparvovec-xioi) and Kymriah (tisagenlecleucel), as well as Gileads Yescarta (axicabtagene ciloleucel).

Vinetis Series C financing round closed at $35m (31.8m), with the software developer announcing that it will use the funding for the expansion of its business, including new software product development and commercial expansion in Europe and Asia-Pacific.

The financing was led by Cardinal Health, with participation from Novartis and Gilead, through its subsidiary, Kite, as well as existing investors. Following the completion of the funding, representatives of Novartis and Kite will join Vinetis leadership as board observers.

Kites global head of technical operations, Charles Calderaro, stated that Kite played a foundational role as Vineti's first biopharma partner, when the company prepared for the launch of Yescarta.

Asked about how the platform will be expanded after the funding, DuRoss explained that it will offer improved configurability, enabling users to add new or updated features and adjust them at will, ultimately saving them cost and time.

In biopharma, its all about speed to market, and our configuration approach supports that, DuRoss told us.

Moreover, the platform will position the company towards global expansion, by including more languages as well as support for more regional regulations and standards.

According to DuRoss, the companys partnersincreasingly operate worldwide, in international medical centers and manufacturing sites, and many patient-based drug products cross borders on their journey to becoming a therapy.

Additionally, the company plans to add to the platform more of the standards and integrations required to industrialize cell and gene therapies. Vinetis CEO told us that It may sound contradictory to standardize such deeply personalized drug products, but for scale, standardization is absolutely essential.

DuRoss claimed that even though its still early days in cell and gene therapy, some standard approaches are emerging, as are pre-built integrations that connect critical technology stacks.

We want to help our partners focus on the uniqueness of their science, and not have to reinvent the wheel on supply chain management with every new drug product, she added.

Finally, the company plans to expand the range of therapies that the platform supports to include other high-value therapeutics. Well keep our focus on cell therapy, gene therapy, and personalized cancer vaccines, but expand into other therapeutic areas requiring precision coordination, said DuRoss.

Read more:
Novartis and Gilead provide funding to Vineti - BioPharma-Reporter.com

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Save the Date: Alliance for Cancer Gene Therapy 2020 Cancer Summit to be Held in New York City – Yahoo Finance

February 7th, 2020 3:41 am

Thought leaders in cancer cell and gene therapy research will share the latest advances, address the greatest challenges and showcase the most innovative programs in progress today.

Alliance for Cancer Gene Therapy (ACGT) will host its inaugural ACGT 2020 Cancer Summit on April 16, 2020 at the Alexandria Center for Life Science in New York City. The ACGT 2020 Cancer Summit which launches ACGTs 20th anniversary will bring together researchers, companies, investors and advocates in cancer cell and gene therapy to discuss the latest advances, with a focus on combating solid tumors.

A partial list of ACGT 2020 Cancer Summit speakers includes:

Presenting sponsors include Alexandria Real Estate Equities, Inc./Alexandria Venture Investment. Additionally, STAT will be a media partner for the ACGT 2020 Cancer Summit. For more sponsorship information and early bird registration, please visit https://summit2020.acgtfoundation.org

Media Registration

Media registration is free of charge for all valid press card holders or via provision of formal journalist credentials. Register early by contacting ACGT.pr@HDMZ.com to begin receiving advance meeting materials, media alerts, and access to meeting presenters.

About Alliance for Cancer Gene Therapy

For nearly 20 years, Alliance for Cancer Gene Therapy (ACGT) has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Founded by Barbara and Edward Netter after their daughter-in-laws death from breast cancer, ACGT funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. One hundred percent of all public funds raised by ACGT directly support program and research, thanks to separate funding to support administrative expenses.

For more information, visit acgtfoundation.org, call 203-358-5055, or join the ACGT community on Facebook, Twitter, Instagram and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200203005084/en/

Contacts

Media Inquiries: Emily Maxwell 312-506-5220emily.maxwell@hdmz.com

Continued here:
Save the Date: Alliance for Cancer Gene Therapy 2020 Cancer Summit to be Held in New York City - Yahoo Finance

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