StemCell Therapy

Already containing health records, surveys, and measurements from more than 200,000 people from all walks of life, the All of Us Research Programs Researcher Workbench is a treasure trove waiting to be mined.

An immigrant from Peru, Dr. Ral Montaez-Valverde was surprised to encounter perplexing research showing that Latinos in the United States are at lower risk of heart disease than their white counterpartsdespite a generally lower socioeconomic status and access to health care.

It prompted me to think about why Hispanics could have better outcomes, given all the challenges, said Montaez-Valverde. I was very curious about this question.

So curious that, despite his intense schedule as a second-year internal medicine resident at Jackson Memorial Hospital, Montaez-Valverde jumped at the chance to use the All of Us Research Programs Researcher Workbench to investigate the long-debated validity of whats known as the Latino Epidemiological Paradox. Now open to investigators from all disciplines and career stages across the University of Miami, the Researcher Workbench contains the de-identified electronic health records of more than 200,000 people across the United Statesincluding 10,000 from Greater Miamiwho have enrolled in the most inclusive study ever undertaken by the National Institutes of Health.

Launched in 2018, the 10-year, $2 billion-plus All of Us Research Program (AoURP) is building one of the worlds largest and most diverse health datasets by collecting lifestyle, health, and genetic information from 1 million people of all races, ethnicities, backgrounds, and gender identities living in the U.S. The ultimate goal is to advance personalized medicine by helping researchers and physicians like Montaez-Valverde understand why different people are more vulnerable to different diseases and conditions and tailor prevention, treatment, and care approaches specifically for them.

But neither the AoURP nor the Universitys Miller School of Medicine, which is leading the AoURPs effort to recruit some 80,000 of the 1 million participants from Florida and Georgia, are waiting for the enrollment process to conclude before making the data available to researchers. The University has signed a data-use agreement with the NIH allowing any faculty members, research assistants, students, residents, or other trainees with an NIH eRA Commons account to begin mining the treasure trove, which will grow in both value and volume as more people discover its riches.

The Researcher Workbench is a major milestone in fulfilling the promise of the All of Us program, but for now it may be one of the best kept secrets in biomedical research, said Stephan Zchner, professor and chair of the Dr. John T. Macdonald Foundation Department of Human Genetics and the lead principal investigator for the AoURPs Southeast Enrollment Center (SEEC), which also includes the University of Florida, Emory University, and Morehouse School of Medicine.

At the end of the day, continued Zchner, who also co-directs the John P. Hussman Institute for Human Genomics, we want to work with data to create new knowledge and insights into medicine, and the workbench is a major tool for that. Whats exciting is that it opens biomedical data access to many qualified investigators, including people in the social sciences, basic sciences, sports, even the arts. The possibilities are endless, and there will be a lot more of them as the data get richer and larger over time.

For the time being, the cloud-based research platform, which requires proficiency with the R or Python programming languages, does not include the genetic information that most interests researchers like Zchner. But as he noted, the AoURP is currently sequencing the genomes of the first 100,000 participants and plans to do the same for all 1 million participantsall of which eventually will make its way into the database and the hands of those who shared their DNA.

Yet even in its infancy, the workbench already contains four types of data. In addition to the electronic health records of roughly 203,000 people, the database includes survey data from more than 315,000 people who answered questions about their medical history, lifestyle, access to care and, more recently, experiences with COVID-19, including the pandemics impact on their mental and financial health. It also contains physical measurementsincluding blood pressure, heart rate, and body mass indexfrom more than 260,000 people, and data collected by the Fitbit wearable devices of more than 8,000 people.

But for Dr. Olveen Carrasquillo, an expert in health disparities who serves as the SEECs participant engagement lead, the most exciting aspect of the AoURP is its success in recruiting minorities who have long been overlooked by medical research. According to the AoURP, about half of the participants whose data is in the Researcher Workbench are people of color.

One of my biggest concerns was that this project would be like everything else, and minorities would be left out, but weve seen really robust and good efforts at assuring they are included, said Carrasquillo, professor of public health sciences, chief of the Division of General Internal Medicine, and a co-principal investigator for the AoURP. And by minorities, I mean that in the full sense, not just race and ethnicity, but by income, education, gender identity. So, with lots of data on minorities, this humongous data source will be a very powerful tool for people who want to reduce health disparities and improve health equity.

To get an idea of its power, Carrasquillo enlisted Montaez-Valverde, the resident he happened to meet on the Metrorail after leaving Jackson Memorial one night, to become one of the Universitys first workbench users. At the time, Montaez-Valverde, who plans to specialize in cardiology, wasnt familiar with the AoURP, or the Latino paradox. But he shared Carrasquillos skepticism that, given their higher rates of diabetes and uncontrolled blood pressure, Latinos would have better cardiovascular health than their white counterparts, as other studies have shown.

Montaez-Valverde was amazed to learn he would have the electronic records of more than 200,000 people to analyze, a powerful tool that helped him conclude that Latinos in the AoURP dataset actually have a higher, not lower, or similar prevalence of cardiovascular disease, than whites. He was just invited to present those findings at theAmerican College of Cardiologys 70th annual scientific session in May.

What we saw clearly does not support the Latino paradox, Carrasquillo said. But thats only in this database, so for now were just throwing more fire on a debate thats been raging for 25 years.

But not subject to much debate will be the growing value of using the AoURP Research Workbench for research and discovery. As the data grow, the research is going to be a lot more powerful, meaningful, and useful, he said.

To learn more about the workbench, visit the All of Us Research Hub or listen to an overview by Drs. Zchner and Carrasquillo presented by the Clinical and Translational Science Institute. For more information about or to enroll in the study, visit the All of Us Research Program.

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National health database opens to University researchers - University of Miami

Method utilizes electronic sensor that instantly and accurately detects concentrations of the antipsychotic drug, clozapine, in the blood via a finger prick, allowing maximal therapeutic benefit while minimizing side effects

BEER-SHEVA, Israel, Feb. 16, 2021 /PRNewswire/ -- Researchers at Ben-Gurion University of the Negev (BGU) have developed a novel method for instantly and accurately monitoring blood levels of the antipsychotic drug, clozapine, using a blood drop from a finger prick. The method, developed by Dr. Hadar Ben-Yoav, Department of Biomedical Engineering and Ilse Katz Institute for Nanoscale Science and Technology, BGU, is based on an electrochemical microsensor which enables, for the first time, clozapine detection in one drop of finger-pricked whole blood samples of schizophrenia patients without using any pretreatment steps.

Clozapine is considered the most effective antipsychotic medication for schizophrenia and the only antipsychotic currently approved for treatment-resistant schizophrenia but is also associated with harsh side effect. Both its efficacy and its side effects are strongly correlated with blood concentration levels, which can differ up to 20-fold between individuals prescribed identical doses, and can be greatly affected by age, gender, drug interactions and other parameters.

Despite the importance of monitoring clozapine blood levels, its current monitoring scheme is burdensome and involves frequent invasive blood draws, leading to sub-optimal treatment efficacy due to the poor ability to titrate its dose for maximal therapeutic benefit while minimizing side effects. As a result, clozapine is still one of the most underutilized evidence-based treatments in the field of mental health.

Dr. Ben-Yoav's team has invented a miniaturized microelectrode sensor that is able to accurately and immediately detect clozapine levels in a microliter-sample of whole blood such as obtained by a simple finger prick. A recent study carried out in collaboration with Prof. Deanna L. Kelly, Maryland Psychiatric Research Center (MPRC), University of Maryland, School of Medicine, showed good correlation between clozapine blood concentrations measured by the device compared to standard laboratory blood tests in schizophrenia patients[i].

Dr. Ben-Yoav, said, "We were excited to see the promising initial results of our novel device, that can supply people with schizophrenia and their caretakers with instantaneous, accurate results of their blood clozapine levels. Clozapine plasma levels are helpful in improving response rates and minimizing unnecessary side effects. Our device can be the basis of rapid, accurate point-of-care monitoring of patients that will enable personalized medicine through close monitoring and adjustment of the dose of this important drug."

"We hope that this innovative invention will help increase patient compliance and facilitate the use of clozapine for people living with schizophrenia," said Josh Peleg, CEO of BGN Technologies. "The medical research field is investing considerable efforts in simplifying and miniaturizing various blood tests, enabling patients to receive medical results immediately and at home, and the device being developed by the team of Dr. Ben-Yoav is an important contribution to this trend. Importantly, the technology underlying this novel clozapine sensor can be used as a platform for the detection of additional substances. After filing for patent protection, BGN Technologies is currently seeking a strategic partner for further developing and commercializing this device."

The novel sensor can be used as a platform for detecting other redox (reducing-oxidizing) chemicals in small quantities of untreated, whole blood samples. Redox molecules are involved in multiple significant chemical reactions, such as synthesis of various substances, biochemical processes in living organisms, diagnostics and medical procedures. Redox agents can be monitored by specific electrodes, but currently available methods of detection require pretreatment of the blood sample in order to separate the desired molecules from other, interfering substances. The sensor developed by Dr. Ben-Yoav's team can detect minute quantities of various redox molecules in untreated blood samples, thus paving the way for developing miniaturized, point-of-care devices that will be able to monitor various targets.

In August 2020, Dr. Ben-Yoav was one of the recipients of the Brain & Behavior Research Foundation's 2020 Klerman and Freedman Prizes, recognizing exceptional clinical and basic research in mental illness. The prizes are awarded annually to honor outstanding scientists working to advance the prevention, diagnosis and treatment of psychiatric illness. Dr. Ben-Yoav received the prize for his development of "novel biosensors to detect unique diagnostic electrical fingerprints from blood samples of schizophrenia patients that can provide crucial information about their treatment management."

References:

[i] Shukla et al. (2020) An integrated electrochemical microsystem for real-time treatment monitoring of clozapine in microliter volume samples from schizophrenia patients. Electrochemistry Communications 120 (2020) 106850; https://doi.org/10.1016/j.elecom.2020.106850

About BGN Technologies

BGN Technologies is the technology transfer company of Ben-Gurion University, the third largest university in Israel. BGN Technologies brings technological innovations from the lab to the market and fosters research collaborations and entrepreneurship among researchers and students. To date, BGN Technologies has established over 100 startup companies in the fields of biotech, hi-tech, and cleantech, and has initiated leading technology hubs, incubators, and accelerators. Over the past decade, BGN Technologies has focused on creating long-term partnerships with multinational corporations such as Deutsche Telekom, Dell-EMC, PayPal, and Lockheed Martin, securing value and growth for Ben-Gurion University as well as the Negev region. For more information, visit the BGN Technologies website.

Media Contact: Tsipi HaitovskyGlobal Media LiaisonBGN TechnologiesTel: +972-52-598-9892E-mail: tsipihai5@gmail.com

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SOURCE BGN Technologies

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Ben-Gurion University Researchers Develop Novel Method for Personalizing Dose of Schizophrenia Drug Clozapine - BioSpace

Introduction

Low-density lipoprotein (LDL) cholesterol has been identified as the causative factor for atherosclerotic cardiovascular disease (ASCVD) based on a variety of evidence obtained from epidemiology,1 human pathology,2 human genetics,3 and clinical trials.47 Familial hypercholesterolemia (FH), an inherited hyper-LDL cholesterolemia, has often been associated with tendon and cutaneous xanthomas and premature ASCVD.8 This disorder has been regarded as a Mendelian autosomal dominant disease caused by rare genetic mutation(s) in the LDL receptor or its associated genes. Theoretically, this disease can be diagnosed at the early stages of life, even as early as pregnancy. Currently, two major methods have been proposed for the screening of FH worldwide.9 The first approach involves cascade screening where a diagnosis of FH in new cases, typically younger relatives, is triggered by the diagnosis of the index cases. The second approach involves universal screening where LDL cholesterol measurements are conducted universally at a certain age, after which detailed assessments, including genetic analyses, are subsequently performed to confirm their diagnoses. However, diagnosing FH in children and adolescents is often quite difficult given that physical xanthomas and family histories are usually obscure and/or difficult to obtain among such young patients.

Apart from the difficulties in diagnosing FH among children and adolescents, debates regarding when, how, and who to treat at this early stage of life have remained ongoing. In this regard, recent advancements in human genetics have revealed that personalized medicine can also be applicable to FH, where patients with deleterious genetic mutations and/or signs of premature atherosclerosis development should be treated earlier and more aggressively.10 On the other hand, children and adolescents with FH who had started early treatment exhibited excellent prognosis even under mild treatment,11 suggesting the importance of earlier treatment in the management of FH.

The current review outlines the current status of clinical and genetic diagnosis of FH in children and adolescents while also providing useful management strategies for FH in children and adolescents based on currently available clinical evidence.

FH is characterized by the clinical triad of primary hyper-LDL cholesterolemia, tendon xanthomas, and premature ASCVD.12 The first documentation of FH dates back as far as 1873,13 during which it had been described as xanthomatous disease. By the 1930s, FH had started to be considered as an inherited disease,14 with Prof. Brown and Goldstein later discovering genetic abnormalities in the LDL receptor as the principal cause of this condition by the 1970s.15 Subsequently, other genes, including apolipoprotein B (APOB)16 and proprotein convertase subtilisin/kexin type 9 (PCSK9)17 genes, have also been identified to cause this disease. Since its initial documentation, FH has long been described to have a prevalence of 1 in 500 individuals among the general population. In 2011, our group found that FH had a prevalence of 1 in 208 based on genetic epidemiology of homozygous FH in the Hokuriku district of Japan.18 Following our report, similar estimates have been obtained in the United States and Europe.19,20 Currently, FH is considered to have a prevalence of 1 in ~300 individuals among the general population.21 By such estimates, only one monogenic mutation causes the critical phenotype. Monogenic FH is considered the standard form of FH, wherein the mutation status of affected genes is associated with increased likelihood of developing ASCVD, independent of LDL cholesterol values.22 This disease appears to account for at least a portion of patients with ASCVD (estimated to be ~1 in 31 individuals), especially those with premature ASCVD (estimated to be ~1 in 15 individuals). No other single disorder can be responsible for such proportions of ASCVD, which has been identified as the leading cause of mortality worldwide.23 Moreover, timely diagnosis and treatment of children and adolescents with FH have been shown to promote a favorable prognosis.11 Accordingly, children and adolescents with require better awareness and more attempts at diagnosing FH compared to adults.

As stated previously, timely diagnosis and treatment has been shown to prevent ASCVD events in patients with FH. As such, identifying patients with FH at a younger age is of particular importance given that this leads to prompt treatment initiation and prevention of premature ASCVD. However, diagnosing FH in younger individuals is somewhat difficult considering that they typically do not exhibit increased Achilles tendon thickness, which has been used as one of the major diagnostic criteria for adult FH worldwide. One proposed screening method for FH is universal screening at an age when FH can be effectively identified.24,25 On the other hand, opportunistic screening, utilizing every opportunity to screen patients for FH, is also effective to find FH.2628 For example, measurement of LDL cholesterol is common practice, and we can find patients with FH when LDL cholesterol level is over a threshold irrespective of the primary aim of its measurements. Another effective screening method for FH is cascade screening, which has been recommended by many organizations around the world. Indeed, countries where dedicated cascade screening programs have been implemented have identified a notably higher number of patients with FHs. For instance, the Netherlands and Norway have diagnosed 71% and 43% of FH cases, respectively.29 In addition, we had demonstrated that cascade screening is significantly associated with better prognoses among patients with FH30 One of the major factors contributing to our results is the notion that an earlier diagnosis promotes better outcomes, which is especially true for patients with FH. As such, although numerous studies have shown the efficacy of LDL-lowering therapies among patients with FH, the magnitude of the benefits obtained from such therapies appear to vary according to the timing of therapy commencement, with far greater benefits having been observed among children than among adults in secondary prevention settings.11,30,31 Accordingly, we firmly believe that earlier diagnosis either via cascade or universal screening and timely LDL-lowering therapies could be beneficial for patients with FH. Alternative way of screening for FH is reverse cascade screening where the index case is a child, and then parents are diagnosed as FH.32,33 It is usually associated with universal screening. However, studying the parents first has a high diagnostic yield.34

Several different types of clinical diagnostic criteria have been established for FH globally, including the Dutch Lipid Clinical Network (DLCN),35 Make Early Diagnosis to Prevent Early Deaths (MEDPED) diagnostic criteria,36 Japan Atherosclerosis Society (JAS) FH diagnostic criteria,37 and Simon Broome diagnostic criteria for FH.35 Each of the aforementioned diagnostic criteria for pediatric FH has specific cutoffs for LDL cholesterol (Boxes 1 and 2; Tables 1 and 2) given the considerable variability in its levels within this group, especially among adolescents.38 Moreover, children and adolescents with FH barely exhibit physical xanthomas, which is one of the major clinical diagnostic criteria for adults. Nonetheless, care should be exercised when using lower LDL cholesterol thresholds for screening young patients with FH, with family history being much more important in pediatric than in adult cases. In this regard, clinical diagnostic criteria for pediatric FH by JAS appears to be quite useful. Because it is quite simple to use (there are only 2 elements), and it really put weight on their family history (of parents). In order to diagnose them as FH, (reverse) cascade screening for FH will be conducted, and then at least 2, or perhaps even more patients with FH can be identified.

Box 1 Diagnosis of Familial Hypercholesterolemia in Children and Adolescents (EAS)

Box 2 Pediatric Familial Hypercholesterolemia Diagnostic Criteria (JAS)

Table 1 Diagnosis of Familial Hypercholesterolemia (MEDPED)

Table 2 Simon Broome Diagnostic Criteria for FH

To establish a diagnosis of FH in children and adolescents, genetic testing may be quite useful, although ethical aspects should be carefully considered.39 However, we also need to be careful for what is FH. A few years ago, an useful classification has been proposed regarding the classification of FH. According to this, FH can be classified into heterozygous FH (caused by a deleterious mutation in FH-gene), homozygous FH (caused by double deleterious mutations in FH-gene), polygenic FH (caused by LDL-associated common genetic variations), and polygenic FH plus hypertriglyceridemia (caused by LDL-, and TG-associated common genetic variations).40 In terms of genetic diagnosis, it is still quite difficult to diagnose polygenic state of FH. Accordingly, genetic diagnosis of FH is usually referring to genetic testing for rare genetic variations of FH-genes. In addition, it is also important to think differently between heterozygous FH and homozygous FH irrespective of ages. As stated above, the prevalence of heterozygous FH is 1 in ~300 among general population, which is a common disorder, and difficult to diagnose them as FH in children adolescents because of reasons stated previously. On the other hand, homozygous FH is a rare condition, the prevalence of which is estimated to 1 in 160,000 among general population. However, it is of note that state of homozygous FH is an emergent condition, where cardiovascular complications are observed in their adolescence.8 There are several special treatments for homozygous FH, including microsomal triglyceride transfer protein (MTTP) inhibitor, LDL apheresis, and liver transplantation.4145 Genetic diagnosis for homozygous FH is very important not just because of their diagnosis, but can be useful for their phenotyping. It has been shown that PCSK9 inhibitor, which is quite useful for heterozygous FH, has minimal effect to reduce LDL cholesterol level among the patients with homozygous FH caused by null-type of mutations of LDLR.46 Other useful points for this matter include assessment of responsiveness to dietary intervention, although dietary interventions typically have minimal influence on LDL cholesterol levels among pediatric patients with FH.47 Moreover, an important differential diagnosis worth considering in pediatric FH includes sitosterolemia,48,49 a disease found to be a phenocopy of homozygous FH. Although patients with sitosterolemia usually exhibit physical xanthomas associated with elevated LDL cholesterol, sitosterolemia is a recessive disorder, with dietary interventions being quite useful for reducing LDL cholesterol levels.50 Sitosterolemia can be distinguished from FH based on the mentioned important clinical manifestations.

FH has been considered one of the major causes of premature ASCVD, with carotid ultrasound being one of the most popular and non-invasive methods for assessing atherosclerosis among pediatric patients with FH. Carotid intima-media thickness (IMT) is often used as a surrogate marker for systemic atherosclerosis among not only the general population but also pediatric patients with FH.51,52 Moreover, coronary and/or aortic calcium scores have been used to assess early subclinical atherosclerosis,53 apart from actual plaque accumulation in the coronary artery.54 Furthermore, arterial stiffness assessed through brachial-ankle pulse wave velocity had been found to be significantly associated with the presence of ASCVD in patients with FH.55 According to accumulated evidence obtained thus far, the development of ASCVD among patients with FH appears to start during adolescence. These findings have motivated us to consider initiating LDL cholesterol-lowering treatments at an earlier stage of life.

Lifestyle interventions should be the fundamental strategy for managing FH in children and adolescents at any age. Statins can be introduced according to guidelines or recommendations. For instance, pitavastatin can be used for Japanese pediatric patients with FH (age 10 years) whose LDL cholesterol levels remain 180 mg/dL under lifestyle interventions, with the optimal target being set at <140 mg/dL, especially among those with diabetes or a family history of premature ASCVD (Figure 1).37 There are many studies showing the efficacy and safety regarding the use of statins for children and adolescents, and a meta-analysis and a systemic review are suggesting that it is true.56,57 Adherence should be closely monitored among those with poor response to statins before increasing the dose. Adolescent girls should be counseled to suspend statin therapy when contemplating pregnancy. Other medications, such as ezetimibe and resin, can be considered when needed. Notably, the European Atherosclerosis Society had proposed a similar strategy in Europe where high-risk pediatric patients with FH aged 810 years are recommended to start statins to reduce LDL cholesterol (Figure 2).10 Moreover, the National Lipid Association expert panel on FH had recommended similar management approaches (Box 3).58 In addition, resin, and ezetimibe are also shown to effectively reduce LDL cholesterol among the pediatric FH patients.59,60 More recently, it has been shown that evolocumab reduced the LDL cholesterol level and other lipid variables among them.61 Notably, all of the mentioned recommendations have acknowledged the need for actively attempting to diagnose/identify FH in children and adolescents and considering lowering LDL cholesterol levels through lifestyle intervention and statins.62

Figure 1 Strategies for the management of pediatric familial hypercholesterolemia (FH) (JAS). Green arrows indicate Yes; blue arrows indicate No. The essential message is that the pediatric patients with FH aged 10 or greater who have low-density lipoprotein cholesterol levels 180 mg/dL under appropriate lifestyle intervention may be treated using statins. Reproduced from Harada-Shiba M, Ohta T, Ohtake A, et al. Joint Working Group by Japan Pediatric Society and Japan Atherosclerosis Society for Making Guidance of Pediatric Familial Hypercholesterolemia.Guidance for Pediatric Familial Hypercholesterolemia 2017.J Atheroscler Thromb. 2018;25(6):539553.37

Figure 2 Strategies for the diagnosis and management of familial hypercholesterolemia (FH) in children and adolescents (EAS). Premature coronary heart disease is defined as a coronary event before age 55 and 60 years in men and women, respectively. Definite FH is defined as genetic confirmation of at least one FH-causing genetic mutation. Close relative is defined as 1st or 2nd degree relatives. Highly probable FH is based on clinical presentation (ie, phenotypic FH): either an elevated low-density lipoprotein cholesterol (LDL-C) level 5 mmol/L in a child after dietary intervention or a LDL-C level 4 mmol/L in a child with a family history of premature coronary heart disease in close relatives and/or high baseline cholesterol in one parent. Cascade screening from an index case with a FH-causing mutation may identify a child with elevated LDL-C levels 3.5 mmol/L. Reproduced with permission from Wiegman A, Gidding SS, Watts GF, et al. European atherosclerosis society consensus panel. Familial hypercholesterolaemia in children and adolescents: gaining decades of life by optimizing detection and treatment. Eur Heart J. 2015;36(36):24252437.10

On the other hand, there are other strategies, including MTTP inhibitor (lomitapide), APOB inhibitor (mipomersen), LDL apheresis, and liver transplantation for the cases with homozygous FH. Another potential medical therapy is Angiopoietin-like 3 (ANGPTL3) inhibitor, whose efficacy and safety for adult patients with homozygous FH has been shown.63

Box 3 Summary Recommendations from the National Lipid Association Expert Panel on Treatments for Pediatric Familial Hypercholesterolemia

Regardless of its definition, genetic analyses for patients with FH should have clear indications beyond clinical diagnosis. As such, we herein highlight the several advantages of genetic background analysis in FH. First, quite a few patients with hypercholesterolemia have obscure or marginal clinical diagnoses of FH. Moreover, their family history is sometimes quite challenging to obtain. Genetic analyses can definitively identify patients with FH, especially those with traditional monogenic FH. Second, genetic analysis allows us to determine whether patients are heterozygous or homozygous. Several special medical therapies, such as MTTP inhibitors and LDL apheresis, have typically only been used for homozygous FH in many parts of the world. Moreover, homozygous FH is one of the designated intractable diseases where all medical costs can be covered by the Japanese government, thereby increasing the importance of diagnosis. Third, a portion of patients with FH may have been misdiagnosed as such and actually have another diagnosis, such as sitosterolemia. In such cases, ezetimibe instead of statins is recommended. Fourth, the genetic status of patients with FH has been found to be associated with increased risk for ASCVD. Accordingly, determining the genetic status can lead to better risk stratification. Fifth, genetic status determination can lead to better cascade screening and consequently better prognoses. Panel sequencing covering FH genes appears to be the current gold standard for determining the genetic status.64 However, determining the pathogenicity of the identified genetic variations has remained challenging for us.

We currently face an important dilemma regarding the definition of FH. If we adhere to clinical manifestations, such as tendon xanthomas, we believe that it is too late considering that earlier interventions based on earlier diagnosis have already been proposed. We believe that at least two different types of diagnostic criteria can be established. The first criteria, which would aim to diagnose definite FH, can be rather strict and have high diagnostic specificity, whereas the second one, which would aim to diagnose potential FH, can have high diagnostic sensitivity. Nonetheless, a diagnosis of FH needs to be ultimately established and adequately treated as early as possible before pediatric patients grow into adults.

Data science and personalized medicine are two major keywords describing medical innovations in the coming 10 years. In the management of FH, genetic analyses involving genes associated with not only LDL cholesterol itself but also ASCVD will become standard. Moreover, target, timing, and LDL cholesterol-lowering therapies will become quite individualized based on genotype, lifestyle, environmental factors, and belief systems. For the earlier identification of patients with FH, nation-wide mass screening, similar to that currently conducted for several other inherited metabolic diseases, such as newborn screening, phenylketonuria, and homocystinuria, will become standard. Furthermore, family history will be automatically assessed to accumulate a huge dataset over the years, which will help us minimize the risk of overlooking children and adolescents with FH (and other inherited diseases).

Given that FH is an inherited disease, early diagnosis and intervention can lead to excellent prognosis. Cascade and universal screening appear to be practical strategies for the early identification of patients with FH; however, we need to consider that certain clinical approaches can promote better identification of children and adolescents with FH. Clinical practices and genetic analyses will certainly help improve not only diagnostic accuracy but also risk stratification for personalized medicine.

The authors report no conflicts of interest in this work.

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19. Benn M, Watts GF, Tybjrg-Hansen A, Nordestgaard BG. Mutations causative of familial hypercholesterolaemia: screening of 98 098 individuals from the Copenhagen general population study estimated a prevalence of 1 in 217. Eur Heart J. 2016;37(17):13841394. doi:10.1093/eurheartj/ehw028

20. de Ferranti SD, Rodday AM, Mendelson MM, et al. Prevalence of familial hypercholesterolemia in the 1999 to 2012 United States national health and nutrition examination surveys (NHANES). Circulation. 2016;133(11):10671072. doi:10.1161/CIRCULATIONAHA.115.018791

21. Beheshti SO, Madsen CM, Varbo A, Nordestgaard BG. Worldwide prevalence of familial hypercholesterolemia: meta-analyses of 11 million subjects. J Am Coll Cardiol. 2020;75(20):25532566. doi:10.1016/j.jacc.2020.03.057

22. Tada H, Kawashiri MA, Nohara A, Inazu A, Mabuchi H, Yamagishi M. Impact of clinical signs and genetic diagnosis of familial hypercholesterolaemia on the prevalence of coronary artery disease in patients with severe hypercholesterolaemia. Eur Heart J. 2017;38(20):15731579. doi:10.1093/eurheartj/ehx004

23. Virani SS, Alonso A, Benjamin EJ, et al. American heart association council on epidemiology and prevention statistics committee and stroke statistics subcommittee. Heart disease and stroke statistics-2020 update: a report from the American heart association. Circulation. 2020;141:e139e596.

24. Lozano P, Henrikson NB, Dunn J, et al. Lipid screening in childhood and adolescence for detection of familial hypercholesterolemia: evidence report and systematic review for the US preventive services task force. JAMA. 2016;316(6):645655. doi:10.1001/jama.2016.6176

25. Groselj U, Kovac J, Sustar U, et al. Universal screening for familial hypercholesterolemia in children: the Slovenian model and literature review. Atherosclerosis. 2018;277:383391. doi:10.1016/j.atherosclerosis.2018.06.858

26. Bell DA, Hooper AJ, Bender R, et al. Opportunistic screening for familial hypercholesterolaemia via a community laboratory. Ann Clin Biochem. 2012;49(6):534537. doi:10.1258/acb.2012.012002

27. Scicali R, Di Pino A, Platania R, et al. Detecting familial hypercholesterolemia by serum lipid profile screening in a hospital setting: clinical, genetic and atherosclerotic burden profile. Nutr Metab Cardiovasc Dis. 2018;28(1):3543. doi:10.1016/j.numecd.2017.07.003

28. Mirzaee S, Choy KW, Doery JCG, Zaman S, Cameron JD, Nasis A. The tertiary hospital laboratory; a novel avenue of opportunistic screening of familial hypercholesterolemia. Int J Cardiol Heart Vasc. 2019;23:100354. doi:10.1016/j.ijcha.2019.100354

29. Besseling J, Sjouke B, Kastelein JJ. Screening and treatment of familial hypercholesterolemia - lessons from the past and opportunities for the future (based on the anitschkow lecture 2014). Atherosclerosis. 2015;241(2):597606. doi:10.1016/j.atherosclerosis.2015.06.011

30. Tada H, Okada H, Nomura A, et al. Prognostic impact of cascade screening for familial hypercholesterolemia on cardiovascular events. J Clin Lipidol. 2021. doi:10.1016/j.jacl.2020.12.012

31. Rodenburg J, Vissers MN, Wiegman A, et al. Statin treatment in children with familial hypercholesterolemia: the younger, the better. Circulation. 2007;116(6):664668. doi:10.1161/CIRCULATIONAHA.106.671016

32. Wu X, Pang J, Wang X, et al. Reverse cascade screening for familial hypercholesterolemia in high-risk Chinese families. Clin Cardiol. 2017;40(11):11691173. doi:10.1002/clc.22809

33. Vinson A, Guerra L, Hamilton L, Wilson DP. Reverse cascade screening for familial hypercholesterolemia. J Pediatr Nurs. 2019;44:5055. doi:10.1016/j.pedn.2018.09.011

34. Ibarretxe D, Rodrguez-Borjabad C, Feliu A, Bilbao J, Masana L, Plana N. Detecting familial hypercholesterolemia earlier in life by actively searching for affected children: the DECOPIN project. Atherosclerosis. 2018;278:210216. doi:10.1016/j.atherosclerosis.2018.09.039

35. Austin MA, Hutter CM, Zimmern RL, Humphries SE. Genetic causes of monogenic heterozygous familial hypercholesterolemia: a HuGE prevalence review. Am J Epidemiol. 2004;160(5):407420. doi:10.1093/aje/kwh236

36. Williams RR, Hunt SC, Schumacher MC, et al. Diagnosing heterozygous familial hypercholesterolemia using new practical criteria validated by molecular genetics. Am J Cardiol. 1993;72(2):171176. doi:10.1016/0002-9149(93)90155-6

37. Harada-Shiba M, Ohta T, Ohtake A, et al. Joint Working Group by Japan Pediatric Society and Japan Atherosclerosis Society for Making Guidance of Pediatric Familial Hypercholesterolemia.Guidance for Pediatric Familial Hypercholesterolemia 2017.J Atheroscler Thromb. 2018 ;25(6):539553.

38. Eissa MA, Mihalopoulos NL, Holubkov R, Dai S, Labarthe DR. Changes in fasting lipids during puberty. J Pediatr. 2016;170:199205. doi:10.1016/j.jpeds.2015.11.018

39. Tada H, Nohara A, Kawashiri MA. Monogenic, polygenic, and oligogenic familial hypercholesterolemia. Curr Opin Lipidol. 2019;30(4):300306. doi:10.1097/MOL.0000000000000609

40. Masana L, Ibarretxe D, Rodrguez-Borjabad C, et al. Expert group from the Spanish arteriosclerosis society. Toward a new clinical classification of patients with familial hypercholesterolemia: one perspective from Spain. Atherosclerosis. 2019;287:8992. doi:10.1016/j.atherosclerosis.2019.06.905

41. France M. Homozygous familial hypercholesterolaemia: update on management. Paediatr Int Child Health. 2016;36(4):243247. doi:10.1080/20469047.2016.1246640

42. Raal FJ, Hovingh GK, Catapano AL. Familial hypercholesterolemia treatments: guidelines and new therapies. Atherosclerosis. 2018;277:483492. doi:10.1016/j.atherosclerosis.2018.06.859

43. Pottle A, Thompson G, Barbir M, et al. Lipoprotein apheresis efficacy, challenges and outcomes: a descriptive analysis from the UK lipoprotein apheresis registry, 19892017. Atherosclerosis. 2019;290:4451. doi:10.1016/j.atherosclerosis.2019.09.006

44. Kawagishi N, Satoh K, Akamatsu Y, et al. Long-term outcome after living donor liver transplantation for two cases of homozygous familial hypercholesterolemia from a heterozygous donor. J Atheroscler Thromb. 2007;14(2):9498. doi:10.5551/jat.14.94

45. Mlinaric M, Bratanic N, Dragos V, et al. Case report: liver transplantation in homozygous familial hypercholesterolemia (HoFH)-long-term follow-up of a patient and literature review. Front Pediatr. 2020;8:567895. doi:10.3389/fped.2020.567895

46. Santos RD, Stein EA, Hovingh GK, et al. Long-term evolocumab in patients with familial hypercholesterolemia. J Am Coll Cardiol. 2020;75(6):565574. doi:10.1016/j.jacc.2019.12.020

47. Malhotra A, Shafiq N, Arora A, Singh M, Kumar R, Malhotra S. Dietary interventions (plant sterols, stanols, omega-3 fatty acids, soy protein and dietary fibers) for familial hypercholesterolaemia. Cochrane Database Syst Rev. 1975;2014;2014(3):CD001918. doi:10.1152/ajplegacy.1975.229.3.570

48. Tada H, Nohara A, Inazu A, Sakuma N, Mabuchi H, Kawashiri M-A. Sitosterolemia, hypercholesterolemia, and coronary artery disease. J Atheroscler Thromb. 2018;25(9):783789. doi:10.5551/jat.RV17024

49. Tada H, Okada H, Nomura A, et al. Rare and deleterious mutations in ABCG5/ABCG8 genes contribute to mimicking and worsening of familial hypercholesterolemia phenotype. Circ J. 2019;83(9):19171924. doi:10.1253/circj.CJ-19-0317

50. Tada H, Kawashiri MA, Takata M, et al. Infantile cases of sitosterolaemia with novel mutations in the ABCG5 gene: extreme hypercholesterolaemia is exacerbated by breastfeeding. JIMD Rep. 2015;21:115122.

51. Tada H, Kawashiri MA, Okada H, et al. Assessments of carotid artery plaque burden in patients with familial hypercholesterolemia. Am J Cardiol. 2017;120(11):19551960. doi:10.1016/j.amjcard.2017.08.012

52. Tada H, Nakagawa T, Okada H, et al. Clinical impact of carotid plaque score rather than carotid intima-media thickness on recurrence of atherosclerotic cardiovascular disease events. J Atheroscler Thromb. 2020;27(1):3846. doi:10.5551/jat.49551

53. Okada H, Tada H, Hayashi K, et al. Aortic root calcification score as an independent factor for predicting major adverse cardiac events in familial hypercholesterolemia. J Atheroscler Thromb. 2018;25(7):634642. doi:10.5551/jat.42705

54. Tada H, Kawashiri MA, Okada H, et al. Assessment of coronary atherosclerosis in patients with familial hypercholesterolemia by coronary computed tomography angiography. Am J Cardiol. 2015;115(6):724729. doi:10.1016/j.amjcard.2014.12.034

55. Tada H, Kawashiri MA, Nohara A, Inazu A, Mabuchi H, Yamagishi M. Assessment of arterial stiffness in patients with familial hypercholesterolemia. J Clin Lipidol. 2018;12(2):397402. doi:10.1016/j.jacl.2017.12.002

56. Dombalis S, Nash A. The effect of statins in children and adolescents with familial hypercholesterolemia: a systematic review. J Pediatr Health Care. 2020;S0891-5245(20)303084. doi:10.1016/j.pedhc.2020.11.007

57. Anagnostis P, Vaitsi K, Kleitsioti P, et al. Efficacy and safety of statin use in children and adolescents with familial hypercholesterolaemia: a systematic review and meta-analysis of randomized-controlled trials. Endocrine. 2020;69(2):249261. doi:10.1007/s12020-020-02302-8

58. Goldberg AC, Hopkins PN, Toth PP, et al. Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients: clinical guidance from the national lipid association expert panel on familial hypercholesterolemia. J Clin Lipidol. 2011;5(3):133140. doi:10.1016/j.jacl.2011.03.001

59. Glueck CJ, Mellies MJ, Dine M, Perry T, Laskarzewski P. Safety and efficacy of long-term diet and diet plus bile acid-binding resin cholesterol-lowering therapy in 73 children heterozygous for familial hypercholesterolemia. Pediatrics. 1986;78(2):338348.

60. van der Graaf A, Cuffie-Jackson C, Vissers MN, et al. Efficacy and safety of coadministration of ezetimibe and simvastatin in adolescents with heterozygous familial hypercholesterolemia. J Am Coll Cardiol. 2008;52(17):14211429. doi:10.1016/j.jacc.2008.09.002

61. Santos RD, Ruzza A, Hovingh GK, HAUSER-RCT Investigators, et al.. Evolocumab in pediatric heterozygous familial hypercholesterolemia. N Engl J Med. 2020;383(14):13171327. doi:10.1056/NEJMoa2019910

62. Ramaswami U, Futema M, Bogsrud MP, Holven KB. Comparison of the characteristics at diagnosis and treatment of children with heterozygous familial hypercholesterolaemia (FH) from eight European countries. Atherosclerosis. 2020;292:178187. doi:10.1016/j.atherosclerosis.2019.11.012

63. Raal FJ, Rosenson RS, Reeskamp LF, ELIPSE HoFH Investigators, et al.. Evinacumab for homozygous familial hypercholesterolemia. N Engl J Med. 2020;383(8):711720. doi:10.1056/NEJMoa2004215

64. Tada H, Kawashiri MA, Nomura A, et al. Oligogenic familial hypercholesterolemia, LDL cholesterol, and coronary artery disease. J Clin Lipidol. 2018;12(6):14361444. doi:10.1016/j.jacl.2018.08.006

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[Full text] Familial Hypercholesterolemia: A Narrative Review on Diagnosis and Man | VHRM - Dove Medical Press

Article by Karen B. Roberts Photo illustration by David Barczak February 15, 2021

According to Hungarian biochemist Albert Szent-Gyrgyi, who won the Nobel Prize in physiology or medicine in 1937 for his study of vitamin c and cell respiration, Innovation is seeing what everybody has seen and thinking what nobody has thought.

Most of the time, innovators do not know if their ideas will pan out. A lot of the time they dont. When failure occurs, inventors step back, reconsider and regroup, then keep pursuing their ideas, incorporating lessons learned along the way in order to pivot or start anew.

As we celebrate National Innovation Day on Tuesday, Feb. 16, UDaily asked several University of Delaware researchers who are fellows of the National Academy of Inventors to share their successes, stumbling blocks and suggestions on what it takes to innovate, invent and inspire new solutions to challenges facing society and the world.

Eleftherios (Terry) Papoutsakis is the Unidel Eugene Du Pont Chair ofChemical and Biomolecular Engineering. He was selected for NAI fellow status in December 2020 for translational biotechnology contributions that have profoundly impacted sustainable manufacturing and human health. One technology that Papoutsakis said has proven particularly useful and successful is his teams development of a method to engineer microparticles that deliver gene-regulating material to hematopoietic stem and progenitor cells that live deep in our bone marrow, where they direct the formation of blood cells. The technology could be useful in treatment for inherited blood disorders, such as sickle cell anemia, or to improve personalized medicine. The discovery, he said, was completely unexpected, but it is currently generating a lot of interest from companies.

Q: Were there inventors that you looked up to as a kid or other people or events that inspired your inventiveness?

Papoutsakis: As a child, I did not know what inventors do but I was amazed by the ability of airplanes to fly, thediscovery of plastics, fertilizers and pesticides (my dad hadan orchard and could tell how important they were) and theconcept of the vaccine. Mygeneration first experienced the benefits of the polio vaccine andvaccines for otherdevastatingdiseases. That iswhy I decided tobecome achemical engineer. I did not know at the time how broad thefield was, but I had a cousin-in-law who wasforward-looking, and he explained to me the potential of thefield and its breadth.

Q: What are some of the stumbling blocks youve encountered as an inventor? How did you overcome them?

Papoutsakis: Two things come to mind. First, I wish I had taken a course inpatent law and patent writing. I amstill learning as I go along; however, I cant help but think about what might have been different if Id had training. I missed several opportunities to protect my researchwork.

Second, Iwish I understood better how tosell (commercialize) myinventions and be good at it. It takes the right personality and athick skin to swallowwithoutpain therejections (and I lack both), plus a lot of time to keeppushing.

Q: Are the best innovators also subject-matter experts? Or do great innovations just as often or more often flow from an idea from someone who does not know how to bring that idea to life, but gets connected with someone who does?

Papoutsakis: Not necessarily, intuition and imagination are more important, I think. In terms of which is better, subject-matter expertise or connections, I think the latter is as potent anavenue as the subject-matter expertwho has intuition and imagination, orthe rightpeople towork with.

Q: What are the critical innovations we need now?

Papoutsakis: We havedone wellwith theeasy thingsthat make a lot of money like social media and the Googles and the Amazons of the world. We needthese things, and the folks that developed them aregeniuses. But we still need tosolvereallybig problems inenergy, the environment, global warming, sustainablemanufacturing and transportation. Then there is the problem of affordable and adaptable health care. The pandemic is just a reminder and anadvance notice asto whathumanity might beup against as wemoveforward.

Q: Are there ways to develop/nurture an innovative mind and keep that spark alive?

Papoutsakis: Patents are a key part of invention. I think it is important to engage both undergrads and graduate students in all aspects of the patent process early on. From patent applications to writing provisional patents and, later, work with lawyers to file the utility patents or even just to read them. It is so different from reading scientific papers. Having this knowledge and background early in ones academic or industrial career would be beneficial for an individual and for future inventors working with that individual to keep the spark alive.

Q: Is there anything you would tell your younger inventor self if you could?

Papoutsakis: At the risk of repeating myself, I would tell my younger self to take a course in patent writing and entrepreneurship, to work with a master in my field and tothink outside of the box. The best ideas are not necessarily based on expensive science.

Kristi Kiick, Blue and Gold Distinguished Professor of Materials Science and Engineering, was named a fellow of the National Academy of Inventors in 2019. Her research involves developing biomaterials to advance medicine, from healing wounds faster and improving chemotherapies, to treating heart and musculoskeletal diseases.Kiicks proudest moment of invention occurred as a graduate student at the California Institute of Technology when she discovered that the natural protein-synthesis machinery of E. coli can be tuned to use novel chemical groups not normally used by nature in protein synthesis. Specific enzymes that normally control what amino acids are included in proteins can simply be produced at higher levels in the bacterial cell. This change alone can permit an enormous range of chemically reactive proteins to be produced. Other scientists have built on Kiicks original approach to create applications that now help scientists learn about processes inside of cells in order to better understand development, disease and drug treatments.

Q: Were there inventors that you looked up to as a kid or other people or events that inspired your inventiveness?

Kiick: My exposure to and interest in invention occurred while I was a research scientist at Kimberly Clark Corporation. I was inspired by many of my co-workers, who each approached innovation and invention differently.Some people saw research articles and applied those findings to technical advances we were trying to make in our laboratories, and others found inspiration from the fundamental principles of the world around them.It was inspiring and a little bit intimidating for me to watch how these colleagues generated and implemented ideas.It definitely changed how I looked at science and its application in solving technical challenges.

Q: What are some of the stumbling blocks youve encountered as an inventor? How did you overcome them?

Kiick: Honestly, the biggest stumbling block for me was trusting my scientific intuition as a young scientist.It took me a long time to understand that my ideas could be novel and that what might appear as an experimental failure could actually be a new discovery.The thoughtful and supportive mentoring by my graduate adviser was pivotal in my making this transition.

Q: Are the best innovators also subject-matter experts? Or do great innovations just as often or more often flow from an idea from someone who does not know how to bring that idea to life, but gets connected with someone who does?

Kiick: The best innovations dont necessarily come from subject matter experts. Having a fresh look at a question or an idea can spark innovation. The implementation of many technical innovations is often best accomplished by a diverse team, where deep technical knowledge can be applied in a new way because someone has thought to look at the idea differently.

Q: What are the critical innovations we need now?

Kiick: I think there are still critical innovations to be made in how we apply massive amounts of data to create new technologies and social systems that allow us to be good stewards of our planet, our communities and ourselves.

Q: Are there ways to develop/nurture an innovative mind and keep that spark alive?

Kiick: As Walt Whitman said, Be curious, not judgmental.

Q: Is there anything you would tell your younger inventor self if you could?

Kiick: I just laughed out loud.I would say surround yourself with supportive people who are trying to make a positive difference. Say yes, and and not no, but. Travel more.Enjoy the journey.

Yushan Yan, Henry B. du Pont Chair in Chemical and Biomolecular Engineering, was named a fellow of the National Academy of Inventors in 2018. He is a co-inventor on more than 20 patents. Among his teams most recent inventions is a new class of ionically conducting polymers that have the potential to drastically reduce the cost of green hydrogen and fuel cells and to help deeply decarbonize all sectors of our economy. In 2019, Yan launched a startup called W7energy, now known as Versogen, alongside UD students and alumni to commercialize this new class of polymers and membranes. Hes proud to report that the company has grown rapidly over the last two years.

Q: Were there inventors that you looked up to as a kid or other people or events that inspired your inventiveness?

Yan: When I was a kid, I did not understand the concept of invention, per se, but I did like tinkering with my hands. For example, I enjoyed making my own primitive telescope or modifying my kerosene lamp to make it burn cleaner. Years later I would learn that what I did to the lamp was to turn the diffusion flame (where the fuel and oxidizer are separate prior to the reaction) into a premixed flame (where the fuel and oxidizer are mixed) like those found in a Bunsen burner.

Q: What are some of the stumbling blocks youve encountered as an inventor? How did you overcome them?

Yan: Coming up with an invention that is useful is not difficult, but developing a good sense of what kind of invention can be commercialized and have a measurable societal impact took some time.

Q: What are the critical innovations we need now?

Yan: As a society we still need many critical innovations in all kinds of fields. For myself, being able to reduce the cost of hydrogen and fuel cells to help deeply decarbonize our economy is a very high priority.

Q: Are there ways to develop/nurture an innovative mind and keep that spark alive?

Yan: I think it is important to instill curiosity into our children and to convince them that everyone has the potential to change what is possible.

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Inspiring innovation | UDaily - UDaily

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G-CON PODs Successfully Delivered for Cell Therapy Manufacturing on an Accelerated Timeline - PRNewswire

Clinical trial digitization allows the processing in different forms of voluminous patient-related data. Such data are being used by pharmaceutical companies to improve the effectiveness of trial execution.

Growing demand for quality data is expected to drive the market growth. Some of the other factors such as increasing demand for personalized drugs, increasing adoption of new technology in clinical research, growing research & development promoting outsourcing and increasing diseases prevalence will drive the market in the forecast period of 2020 to 2027

Competitive Landscape and Pharma Clinical Trial Digitization Market Share Analysis

Pharma clinical trial digitization market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to pharma clinical trial digitization market.

Pharma Clinical Trial Digitization Market Scope

The pharma clinical trial digitization market is segmented on the basis of countries into U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

All country based analysis of pharma clinical trial digitization market is further analyzed based on maximum granularity into further segmentation. On the basis of services, the pharma clinical trial digitization market is segmented into drug dose adjustment, drug impact monitoring, medical prescription system, bioprinting, preventive therapy, and individualized drug printing. Based on application, the market is segmented into clinical data management, trial monitoring, patient recruitment and enrollment. The pharma clinical trial digitization market on the basis of theme is segmented into digital continuity acrossclinical trial IT systems, patient-centric remote and virtual trial design and direct-to-patient home services.

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Key Pointers Covered in the Pharma Clinical Trial Digitization Market Industry Trends and Forecast to 2027

Pharma Clinical Trial Digitization Market Scenario

According to Data Bridge Market Research the market for pharma clinical trial digitization is increasing owing to the penetration of technology in the clinical research. The adoption of patient-centric remote and virtual trial design and direct-to-patient home services is helping the pharma clinical trial digitization to expand. Germination of health problems day by day is catering a good demand of research and technology, which on the whole is basic and keen parameter aiding to pharma clinical trial digitization market growth during the forecast period of 2020 to 2027.

Now the question is which are the other regions intuitive is targeting? Data Bridge Market Research has forecasted a large growth in theNorth America, owing to the advanced healthcare infrastructure. On the contrary Asia-Pacific (APAC) is expected to bounce the market growth exponentially due to surging players penetration and government initiatives taken.

Table of Contents-Snapshot Executive SummaryChapter 1 Industry OverviewChapter 2 Industry Competition by ManufacturersChapter 3 Industry Production Market Share by RegionsChapter 4 Industry Consumption by RegionsChapter 5 Industry Production, Revenue, Price Trend by TypeChapter 6 Industry Analysis by ApplicationsChapter 7 Company Profiles and Key Figures in Industry BusinessChapter 8 Industry Manufacturing Cost AnalysisChapter 9 Marketing Channel, Distributors and CustomersChapter 10 Market DynamicsChapter 11 Industry ForecastChapter 12 Research Findings and ConclusionChapter 13 Methodology and Data Source

For More Insights Get Detailed TOC @https://www.databridgemarketresearch.com/toc/?dbmr=global-pharma-clinical-trial-digitization-market&pm

Global Pharma Clinical Trial Digitization Market Scope and Market Size

Pharma clinical trial digitizationmarket is segmented of the basis of services, application and themes. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

On the basis of services, the pharma clinical trial digitization market is segmented into drug dose adjustment, drug impact monitoring, medical prescription system, bioprinting, preventive therapy, and individualized drug printing.Based on application, the market is segmented into clinical data management, trial monitoring, patient recruitment and enrollment.The pharma clinical trial digitization market on the basis of theme is segmented into digital continuity across clinical trial it systems, patient-centric remote and virtual trial design and direct-to-patient home services.

Pharma Clinical Trial Digitization Market Country Level Analysis

Pharma clinical trial digitization market is analysed and market size insights and trends are provided by services, application and themes as referenced above.

The countries covered in the pharma clinical trial digitization market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

The country section of the pharma clinical trial digitization market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Pharma clinical trial digitization market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipments, installed base of different kind of products for pharma clinical trial digitization market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the pharma clinical trial digitization market. The data is available for historic period 2010 to 2018.

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Corporatesales@databridgemarketresearch.com

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Pharma Clinical Trial Digitization Market 2021 Rising Demand and Grow Opportunities Antidote Technologies, Aparito, Clinerion, CliniOps, Inc., KSU |...

DUBLIN--(BUSINESS WIRE)--The "Global Point of Care (PoC) Molecular Diagnostics Market 2020-2030 by Product (Assays, Instruments, Software), Technology (PCR, INAAT, Microarray), Application, End User, and Region: Trend Forecast and Growth Opportunity" report has been added to ResearchAndMarkets.com's offering.

The global PoC molecular diagnostics market will reach $5,665.3 million by 2030, growing by 13.5% annually over 2020-2030 driven by increase in adoption of personalized medicine and surge in usage of PoC molecular diagnostics for drug discovery and development amid COVID-19 pandemic.

This report is based on a comprehensive research of the entire global PoC molecular diagnostics market and all its sub-segments through extensively detailed classifications. Profound analysis and assessment are generated from premium primary and secondary information sources with inputs derived from industry professionals across the value chain. The report is based on studies on 2015-2019 and provides forecast from 2020 till 2030 with 2019 as the base year.

In-depth qualitative analyses include identification and investigation of the following aspects:

The trend and outlook of global market is forecast in optimistic, balanced, and conservative view by taking into account of COVID-19. The balanced (most likely) projection is used to quantify global PoC molecular diagnostics market in every aspect of the classification from perspectives of Product, Technology, Application, End User, and Region.

Key Players:

Key Topics Covered:

1 Introduction

1.1 Industry Definition and Research Scope

1.1.1 Industry Definition

1.1.2 Research Scope

1.2 Research Methodology

1.2.1 Overview of Market Research Methodology

1.2.2 Market Assumption

1.2.3 Secondary Data

1.2.4 Primary Data

1.2.5 Data Filtration and Model Design

1.2.6 Market Size/Share Estimation

1.2.7 Research Limitations

1.3 Executive Summary

2 Market Overview and Dynamics

2.1 Market Size and Forecast

2.1.1 Impact of COVID-19 on World Economy

2.1.2 Impact of COVID-19 on the Market

2.2 Major Growth Drivers

2.3 Market Restraints and Challenges

2.4 Emerging Opportunities and Market Trends

2.5 Porter's Five Forces Analysis

3 Segmentation of Global Market by Product

3.1 Market Overview by Product

3.2 Kits & Assays

3.3 Analyzers & Instruments

3.4 Software & Services

4 Segmentation of Global Market by Technology

4.1 Market Overview by Technology

4.2 Polymerase Chain Reaction (PCR)

4.2.1 Real-Time PCR (rt-PCR)

4.2.2 Digital PCR (d-PCR)

4.3 Isothermal Nucleic Acid Amplification Technology (INAAT)

4.4 Genetic Sequencing-based Technology

4.5 Microarray-based Technology

4.6 Other Technologies

5 Segmentation of Global Market by Application

5.1 Market Overview by Application

5.2 Infectious Disease

5.2.1 Respiratory Infections

5.2.2 Hospital Acquired Infections

5.2.3 Sexually Transmitted Infections

5.3 Gastrointestinal Infections

5.4 Oncology

5.5 Hepatitis

5.6 Prenatal/Neonatal Testing

5.7 Other Applications

6 Segmentation of Global Market by End User

6.1 Market Overview by End User

6.2 Hospitals

6.3 Clinics & Diagnostic Centers

6.4 Research and Academic Institutes

6.5 Other End Users

7 Segmentation of Global Market by Region

7.1 Geographic Market Overview 2019-2030

7.2 North America Market 2019-2030 by Country

7.2.1 Overview of North America Market

7.2.2 U.S.

7.2.3 Canada

7.2.4 Mexico

7.3 European Market 2019-2030 by Country

7.3.1 Overview of European Market

7.3.2 Germany

7.3.3 UK

7.3.4 France

7.3.5 Spain

7.3.6 Italy

7.3.7 Russia

7.3.8 Rest of European Market

7.4 Asia-Pacific Market 2019-2030 by Country

7.4.1 Overview of Asia-Pacific Market

7.4.2 Japan

7.4.3 China

7.4.4 Australia

7.4.5 India

7.4.6 South Korea

7.4.7 Rest of APAC Region

7.5 South America Market 2019-2030 by Country

7.5.1 Argentina

7.5.2 Brazil

7.5.3 Chile

7.5.4 Rest of South America Market

7.6 MEA Market 2019-2030 by Country

7.6.1 UAE

7.6.2 Saudi Arabia

7.6.3 South Africa

7.6.4 Other National Markets

8 Competitive Landscape

8.1 Overview of Key Vendors

8.2 New Product Launch, Partnership, Investment, and M&A

8.3 Company Profiles

9 Investing in Global Market: Risk Assessment and Management

9.1 Risk Evaluation of Global Market

9.2 Critical Success Factors (CSFs)

Originally posted here:
Worldwide Point of Care (PoC) Molecular Diagnostics Industry to 2030 - Impact Analysis of COVID-19 - ResearchAndMarkets.com - Business Wire

A New Business Research Report released by DBMR with title Global Pharma Clinical Trial Digitization Market Study Forecast till 2027. This Report presents detailed competitive analysis including the market share, size, growth, trends, demand, revenue, cost structure, segment and future scope 2027. This study categorizes the global Health and Safety Products breakdown data by manufacturers, region, type and applications, also analyzes the market drivers, opportunities and challenges. This Global Pharma Clinical Trial Digitization market report brings data for the estimated year 2021 and forecasted till 2027 in terms of both, value (US$ MN) and volume (MT). The report also consists of forecast factors, macroeconomic factors, and a market outlook of the Pharma Clinical Trial Digitization market. The study is conducted by applying both top-down and bottom-up approaches and further iterative methods used to validate and size market estimation and trends of the Global Pharma Clinical Trial Digitization market. Pharma Clinical Trial Digitization Market Report will add the analysis of the impact of COVID-19 on this industry.

Get Exclusive Sample (350 Pages PDF) Report: To Know the Impact of COVID-19 on this Industry@ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-pharma-clinical-trial-digitization-market&aB

This upgradation of healthcare IT infrastructure and technological assistance being provided on vast scale is driving the market growth at the rate of 5.70% annual for the mentioned seven years. This momentum will be maintained by rising practice of individualized drug printing.

Data Bridge Market Research analyses the market to grow at a CAGR of 5.7% in the above-mentioned forecast period. Growing demand for personalized medicine is expected to create new opportunity for the pharma clinical trial digitization mark

Pharma Clinical Trial Digitization Market Overview:

Clinical trial digitization allows the processing in different forms of voluminous patient-related data. Such data are being used by pharmaceutical companies to improve the effectiveness of trial execution.

Growing demand for quality data is expected to drive the market growth. Some of the other factors such as increasing demand for personalized drugs, increasing adoption of new technology in clinical research, growing research & development promoting outsourcing and increasing diseases prevalence will drive the market in the forecast period of 2020 to 2027

Global Pharma Clinical Trial Digitization Market 2020 Reportencompasses an infinite knowledge and information on what the markets definition, classifications, applications, and engagements are and also explains the drivers and restraints of the market which is obtained from SWOT analysis. By applying market intelligence for this Pharma Clinical Trial Digitization Market report, industry expert measure strategic options, summarize successful action plans and support companies with critical bottom-line decisions. Additionally, the data, facts and figures collected to generate this market report are obtained forms the trustworthy sources such as websites, journals, mergers, newspapers and other authentic sources. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, price, cost, revenue and gross margins.

According to this report Global Pharma Clinical Trial Digitization Market will rise from Covid-19 crisis at moderate growth rate during 2020 to 2027. Pharma Clinical Trial Digitization Market includes comprehensive information derived from depth study on Pharma Clinical Trial Digitization Industry historical and forecast market data. Global Pharma Clinical Trial Digitization Market Size To Expand moderately as the new developments in Pharma Clinical Trial Digitization and Impact of COVID19 over the forecast period 2020 to 2027.

TO UNDERSTAND HOW COVID-19 IMPACT IS COVERED IN THIS REPORT, GET FREE COVID-19 SAMPLE @https://www.databridgemarketresearch.com/covid-19-impact/global-pharma-clinical-trial-digitization-market?ab

Pharma Clinical Trial Digitization Market report provides depth analysis of the market impact and new opportunities created by the COVID19/CORONA Virus pandemic. Report covers Pharma Clinical Trial Digitization Market report is helpful for strategists, marketers and senior management, And Key Players in Pharma Clinical Trial Digitization Industry.

Pharma Clinical Trial Digitization Market Segments Outlook:

By Services (Drug Dose Adjustment, Drug Impact Monitoring, Medical Prescription System, Bioprinting, Preventive Therapy, Individualized Drug Printing)

By Application (Clinical Data Management, Trial Monitoring, Patient Recruitment and Enrollment)

By Themes (Digital Continuity Across Clinical Trial IT Systems, Patient-centric Remote and Virtual Trial Design, Direct-to-patient Home Services)

List of Companies Profiled in the Pharma Clinical Trial Digitization Market Report are:

Antidote Technologies, Inc.AparitoClinerion Ltd.CliniOps, Inc.ConsilxDeep 6 AIKoneksa Health Inc.Medidata SolutionsOraclePatientsLikeMeTrialbeeTriNetX, Inc.Veeva SystemsComplete Report is Available (Including Full TOC, List of Tables & Figures, Graphs, and Chart) @https://www.databridgemarketresearch.com/toc/?dbmr=global-pharma-clinical-trial-digitization-market&Ab

Pharma Clinical Trial Digitization Reportdisplays data on key players, majorcollaborations, merger & acquisitions along with trending innovation and business policies. The report highlights current and future market trends and carries out analysis of the effect of buyers, substitutes, new entrants, competitors, and suppliers on the market. The key topics that have been explained in this Pharma Clinical Trial Digitization market report include market definition, market segmentation, key developments, competitive analysis and research methodology. To accomplish maximum return on investment (ROI), its very essential to be acquainted with market parameters such as brand awareness, market landscape, possible future issues, industry trends and customer behavior where this Pharma Clinical Trial Digitization report comes into play.

Global Pharma Clinical Trial Digitization Market Scope and Market Size

On the basis of services, the pharma clinical trial digitization market is segmented into drug dose adjustment, drug impact monitoring, medical prescription system, bioprinting, preventive therapy, and individualized drug printing.

Based on application, the market is segmented into clinical data management, trial monitoring, patient recruitment and enrollment.

The pharma clinical trial digitization market on the basis of theme is segmented into digital continuity across clinical trial it systems, patient-centric remote and virtual trial design and direct-to-patient home services.

Market Size Segmentation by Region & Countries (Customizable):

North America (Canada, United States & Mexico)Europe (Germany, the United Kingdom, Benelux, France, Russia & Italy)Asia-Pacific (Japan, South Korea, China, India & Southeast Asia)South America (Argentina, Brazil, Peru, Colombia, Etc.)Middle East & Africa (United Arab Emirates, Egypt, Saudi Arabia, Nigeria & South Africa)Key Questions Answered

What impact does COVID-19 have made on Global Pharma Clinical Trial Digitization Market Growth & Sizing?

Who are the Leading key players and what are their Key Business plans in the Global Pharma Clinical Trial Digitization market?

What are the key concerns of the five forces analysis of the Global Pharma Clinical Trial Digitization market?

What are different prospects and threats faced by the dealers in the Global Pharma Clinical Trial Digitization market?

What are the strengths and weaknesses of the key vendors?

Major Key Contents Covered in Pharma Clinical Trial Digitization Market:

Introduction of Pharma Clinical Trial Digitization with development and status.

Manufacturing Technology of Pharma Clinical Trial Digitization with analysis and trends.

Analysis of Global Pharma Clinical Trial Digitization market Key Manufacturers with Company Profile, Product Information, Production Information, and Contact Information.

Analysis of Global Pharma Clinical Trial Digitization market Capacity, Production, Production Value, Cost and Profit

Analysis Pharma Clinical Trial Digitization Market with Comparison, Supply, Consumption, and Import and Export.

Pharma Clinical Trial Digitization market Analysis with Market Status and Market Competition by Companies and Countries.

2020-2027 Market Forecast of Global Pharma Clinical Trial Digitization Market with Cost, Profit, Market Shares, Supply, Demands, Import and Export

Trending factors influencing the market shares of APAC, Europe, North America, and ROW?

Pharma Clinical Trial Digitization Market Analysis of Industry Chain Structure, Upstream Raw Materials, Downstream Industry

Strategic Points Covered in Table of Content of Global Pharma Clinical Trial Digitization Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the Pharma Clinical Trial Digitization market

Chapter 2: Exclusive Summary the basic information of the Pharma Clinical Trial Digitization Market.

Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges of the Pharma Clinical Trial Digitization

Chapter 4: Presenting the Pharma Clinical Trial Digitization Market Factor Analysis Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.

Chapter 5: Displaying market size by Type, End User and Region 2010-2019

Chapter 6: Evaluating the leading manufacturers of the Pharma Clinical Trial Digitization market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile

Chapter 7: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries (2020-2027).

Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source

Finally, Pharma Clinical Trial Digitization Market is a valuable source of guidance for individuals and companies in decision framework.

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

Contact Us:

Data Bridge Market Research

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Pharma Clinical Trial Digitization Industry 2021 With Covid-19 Impact on Market Research by Size, Top Leading Countries, Companies, Consumption,...

Personalized Medicine at FDA: The Scope & Significance of Progress in 2020describes how newly approved tests and therapies will help transform care for molecularly selected subsets of patients with cancer, rare diseases, and common/infectious diseases. Seven of the personalized therapies listed in the report are designed to address the root causes of devastating rare genetic diseases for which patients had few treatment options before. The report also notes how expanded indications will extend the unprecedented benefits of cancer immunotherapies to patients with solid tumors of all types that are tumor mutational burden-high (TMB-H). Perhaps most significantly, it explains how the first FDA-approved blood-based biomarker tests for cancer will help guide targeted treatment strategies for patients who are unable to undergo invasive operations to obtain tissue biopsies. The emergence of blood-based biomarker testing anticipates a new era in which cancers are detected at earlier stages, when they are easier and less expensive to treat.

"Personalized Medicine at FDA: The Scope & Significance of Progress in 2020 documents FDA's unwavering commitment to expanding the frontiers of personalized medicine while also demonstrating industry's commitment to developing innovative and groundbreaking products that serve patients and make health systems more efficient," said PMC President Edward Abrahams.

About the Personalized Medicine Coalition:The Personalized Medicine Coalition, a 501(c)3 organization comprised of 14 distinct stakeholder groups within health care, promotes the understanding and adoption of personalized medicine concepts, services and products to benefit patients and the health system. For more information, please visit http://www.personalizedmedicinecoalition.org.

Contact: Christopher J. WellsPersonalized Medicine Coalition[emailprotected]202-580-9780

SOURCE Personalized Medicine Coalition

http://www.personalizedmedicinecoalition.org

Read the original post:
Personalized Medicines Top One-Third of All New Drug Approvals in 2020 for Third Time in Last Four Years - PRNewswire

February 11, 2021 -A team from Cleveland Clinic has developed a precision medicine platform designed to accelerate cancer gene therapies and genome-informed drug discovery.

In a study published in Nature Genetics, researchers describe the My Personal Mutanome (MPM) platform. The platform features an interactive database that offers insight into the role of somatic mutations in cancer acquired mutations that cant be passed to offspring and prioritizes mutations that may be responsive to drug therapy.

Although advances in sequencing technology have bestowed a wealth of cancer genomic data, the capabilities to bridge the translational gap between large-scale genomic studies and clinical decision making were lacking, said Feixiong Cheng, PhD, assistant staff in theGenomic Medicine Institute, and the studys lead author.

MPM is a powerful tool that will aid in the identification of novel functional mutations/genes, drug targets and biomarkers for cancer, thus accelerating the progress towards cancer precision medicine.

The team used clinical data to integrate nearly 500,000 mutations from over 10,800 tumor exomes the protein-coding part of the genome across 33 cancer types into the platform. The team then systematically mapped the mutations to over 94,500 protein-protein interactions (PPIs) and over 311,000 functional protein sites where proteins physically bind with one another. Researchers then incorporated patient survival and drug response data.

The platform analyzes the relationships between genetic mutations, proteins, PPIs, protein functional sites, and drugs to help users easily search for clinically actionable mutations. The MPM database includes three interactive visualization tools that offer two- and three-dimensional views of somatic mutations and their associated survival and drug responses.

According to the researchers, previous studies have linked disease pathogenesis and progression to mutations and variations that disturb the human interactome, the complex network of proteins and PPIs that impact cellular function. Mutations can disrupt the network by directly changing the normal function of a protein, known as nodetic effect, or by altering PPIs, known as edgetic effect.

Additionally, in a separate, previous study, a team of researchers found that somatic mutations were highly enriched where PPIs occurred. The group also demonstrated that PPI-perturbing mutations were significantly correlated with drug sensitivity or resistance as well as poor survival rate in cancer patients.

The results from another study published inNature Genetics, which was a collaboration between Cleveland Clinic and several other institutions, motivated us to develop the mutanome platform, said Cheng.

OurNature Geneticsfindings, along with previous research, provide proof-of-concept of both nodetic and edgetic effects of somatic mutations in cancer. What we learned from that study inspired us to develop a systems biology tool that, by mapping mutations to PPI interfaces and protein functional sites and integrating survival and drug response data, identifies cancer-driving and actionable mutations to guide personalized treatment and drug discovery.

Researchers expect that MPM will lead to a better understanding of mutations at the human interactome network level. This could lead to new insights in cancer genomics and treatments, ultimately achieving the goal of cancer precision medicine.

The team will continue to update MPM annually in order to provide researchers and physicians with the most comprehensive, complete data available. Researchers also plan to apply advanced analytics technologies to their insights to improve treatment development for other conditions.

OurNature Geneticsstudy also demonstrates the nodetic and edgetic effects of mutations/variations in other diseases, said Cheng.

As a next step, we are developing new artificial intelligence algorithms to translate these genomic medicine findings into human genome-informed drug target identification and precision medicine drug discovery (i.e., protein-protein inhibitors) for other complex diseases, including heart disease and Alzheimers disease.

Read this article:
Precision Medicine Platform Aims to Advance Cancer Gene Therapies - HealthITAnalytics.com

SALT LAKE CITY, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc.. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, announced today additional data further validating the prognostic power of its Prolaris test and its ability to help accurately predict which men with more aggressive prostate cancer will benefit from intensification of therapy and which patients may safely avoid such treatments. This second validation study was presented during an oral presentation at the American Society of Clinical Oncology Genitourinary Cancer Symposium (ASCO-GU) by Jonathan Tward M.D., Ph.D, associate professor in the Department of Radiation Oncology at the University of Utah.

According to estimates by the American Cancer Society, 248,530 new cases of prostate cancer are expected to be diagnosed this year in the U.S. While early screening tests have helped reduce the mortality rate, they can often result in overdiagnosis and overtreatment of a disease that is clinically insignificant. The Prolaris test can more accurately predict the aggressiveness of the cancer allowing for more precise treatment and avoidance of more intense therapies with a patients parallel morbidities.

There are many viable treatment paths for men with prostate cancer, said Dr. Tward. This new data helps distinguish the most appropriate personalized treatment path for each patient based on how their specific tumor is behaving. For some men, this means being able to avoid overtreating patients with therapies including hormone treatment that can momentously impact their quality of life, while still appropriately treating their prostate cancer.

The new data comes from a second study following previous data, recently published in Clinical Genitourinary Cancer in January 2021, that incorporated men treated surgically or with radiation therapy. This new study combined a Prolaris molecular risk score threshold with a clinical model for predicting a patients benefit from androgen deprivation therapy. Prolaris determined that about one of every two men with unfavorable intermediate-risk and one of every five men with high-risk prostate cancer are below the proposed threshold associated with aggressive disease and can therefore safely be treated with less intense therapy while maintaining the benefits of treatment. Additional key findings revealed that the Prolaris test was an accurate predictor of progression to metastatic disease.

Myriad Genetics was the first company to offer a test that directly measures the molecular biology of an individual patients prostate cancer, said Todd D. Cohen, M.D., vice president of Medical Affairs for Urology at Myriad Genetics. This study by Dr. Tward and his team is another strong validation of the prognostic power of the Prolaris test and our ongoing commitment to providing healthcare professionals with the tools needed to determine the most effective treatments and monitoring strategies for each patient.

In March 2020, the National Comprehensive Cancer Network updated its professional guidelines to include biomarker testing for unfavorable intermediate and high-risk patients with prostate cancer. With the updated guidelines, Prolaris was one of only two prognostic tests to be considered for those expanded indications. Approximately 60% of men with prostate cancer currently have insurance or Medicare access to Prolaris, and Myriad continues to work toward expanding access so that every man who is facing difficult treatment decisions will be able to utilize the full benefits of the test.

About ProlarisProlaris is a genetic test developed by Myriad Genetics that directly measures tumor cell growth. The Prolaris test paired with other clinical and pathologic variables provides the level of aggressiveness of a patients individual prostate cancer and assesses risk of death or the development of metastatic disease from prostate cancer. For more information visit: http://www.prolaris.com.

About Myriad GeneticsMyriad Genetics Inc., is a leading genetic testing and precision medicine company dedicated to transforming patient lives worldwide. Myriad discovers and commercializes genetic tests that determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across medical specialties where critical genetic insights can significantly improve patient care and lower healthcare costs. For more information, visit the Company's website:www.myriad.com.

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

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to the validation study presented during at ASCO-GU by Jonathan Tward M.D., Ph.D; expanding access so that every man who is facing difficult treatment decisions will be able to utilize the full benefits of the Prolaris test; and the Companys strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Assn for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Intl, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2020, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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New Study from Leading University of Utah Radiation Oncologist Validates Ability of Myriad Genetics' Prolaris test to Guide Treatment for Prostate...

IRVINE, Calif.--(BUSINESS WIRE)--GATC Health, an innovative technology company focused on whole genome testing and Predictive Multiomics, today announced the completion of its Viral Immunity Platform (VIP), a prediction tool for personal immune response analysis. VIP can predict the likelihood of infection and severity of outcomes for SARS-CoV-2 and other viral infections.

VIP runs on the companys Multiomic Advanced Technology (MAT), which combines machine learning, neural networks, deep sequencing and bioinformatics in a comprehensive platform. MAT integrates personal metadata to accurately predict individual immunity response to agents compromising the immune system. MAT also combines advanced AI systems and quantitative genomics, proteomics and microbiomics to decipher billions of datapoints, delivering extremely accurate results.

VIP provides personalized medical analysis that assesses risk of infection, predicts immune response, identifies potential complications, and could even provide therapeutic guidance. The results enable patients and physicians to make informed decisions about individual immunity and safety, including who can return to work, who should attend social gatherings, who may have a moderate illness, and who is at high risk for a serious infection or prone to complications.

In our current climate, its more important than ever to have accurate information about how we will react to COVID or other viral infections so that we can make well-educated decisions, said John Stroh, Interim CEO of GATC Health. Our new VIP technology platform has the ability to provide predictive, actionable information about how an individual will respond when exposed to a virus. Armed with this knowledge, physicians and patients can make decisions to reduce their risk of infection, determine the most likely course of an infection if it does occur, and plan the appropriate treatment.

GATC Healths Viral Immunity Platform will provide customers with a personalized report outlining predicted viral infection risk, immunity response, and potential complications. VIP is expected to be available for consumers and physicians in Q2 2021.

About GATC Health

GATC Health is a pioneering technology company using Predictive Multiomics to advance drug discovery and improve human health. GATC Healths proprietary Multiomic Advanced Technology (MAT) sequences an individuals DNA, reading the entire genome and analyzing the full data set of omics, including genomics, proteomics, and microbiomics. A complete understanding of a persons genetics combined with the evaluation of other omics yields the most comprehensive individual health analysis available, which can lead to fast-tracking drug development, pre-screening clinical trial participants, identifying new drugs and repurposing existing treatments. GATC Healths innovative technology can help take healthcare from reactive to predictiveit is truly personalized medicine. For more information about GATC Health, visit http://www.GATCHealth.com.

Disclaimer

The information set forth herein is presented for informational purposes only and should not be deemed an offer to sell securities. This presentation contains information which the Company believes to be correct, including certain financial information and projections, but the Company does not guarantee as to the accuracy or completeness of such information. The Company reserves the right to modify or amend the information contained herein.

Here is the original post:
GATC Health Introduces Personalized Immunity Platform to Predict Individual Response to SARS-CoV-2 and Other Viral Infections - Business Wire

Dublin, Feb. 12, 2021 (GLOBE NEWSWIRE) -- The "Biobanking Market by Specimen Type, Type of Biobank, Ownership, and Application: Global Opportunity Analysis and Industry Forecast, 2020-2027" report has been added to ResearchAndMarkets.com's offering.

The global biobanking market was valued at $37. 93 billion in 2019, and is estimated to reach $57. 67 billion by 2027, growing at a CAGR of 4. 6% from 2020 to 2027.

Biobank is a biorepository meant to collect and preserve biological materials that are used for diagnosis, biodiversity studies, and research. In addition, it is used to support the research of most common types of genetic disorders to develop personalized medicines and to maintain and update database of diseases related to age.

The rise in funding by private & government organizations for biobanking and increase in application areas of bio banked samples are the major factors that drive the growth of the global biobanking market. Moreover, upsurge in incidence of diseases such as cancer significantly contributes toward the market growth, owing to the fact that biobanked specimens are used in the treatment of several chronic diseases. However, ethical issues related to biobanking and lack of awareness about it are expected to impede the market growth. On the contrary, advancements in stem cell research and unmet medical needs in emerging nations are anticipated to provide lucrative opportunity for the market expansion.

The rise in genomic research activities acts as a key driving force of the global market. Furthermore, government is taking multiple initiatives to support regenerative medicine research, which is expected to boost the market growth. Moreover, increase in demand for cost-effective drug delivery & development accelerates the market growth. Furthermore, usage of human biospecimens is increasing in cohort studies. Number of registered clinical studies with the National Institutes of Health (NIH) increased from 181,305 in 2014 to 262,445 in 2017. A substantial number of large ongoing cohort studies related to precision medicine have fomented the establishment of population-based banks.

The global biobanking market is segmented into specimen type, type of biobank, ownership, application, and region. Depending on specimen type, the market is categorized into blood products, solid tissue, cell lines, nucleic acid, and others. On the basis of ownership, it is divided into national/regional agencies, nonprofit organization, universities, and private organization. The applications covered in the study include therapeutic and research. By type of biobank, the market is bifurcated into population-based biobank and disease-oriented biobank. Region wise, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

Some of the major companies that operate in the global market for biobanks are BioIVT & Elevating Science, Geneticist Inc, Firalis S. A., AMS biotechnology Ltd. (AMSBIO LLC), and US Biolab Corporation, Inc., ProteoGenex, Inc., Cureline, Inc., Bay Biosciences LLC, CTI Biotech, and Boca Biolistics.

Key Benefits

Key Topics Covered:

Chapter 1: Introduction1.1. Report Description1.2. Key Benefits for Stakeholders1.3. Key Market Segments1.1. Research Methodology1.1.1. Secondary Research1.1.2. Primary Research1.1.3. Analyst Tools and Models

Chapter 2: Executive Summary2.1. Key Findings of the Study2.2. Cxo Perspective

Chapter 3: Market Overview3.1. Market Definition and Scope3.2. Key Findings3.2.1. Top Investment Pockets3.2.2. Top Player Positioning3.3. Porter'S Five Forces Analysis3.4. Market Dynamics3.4.1. Drivers, Restraints, and Opportunity3.4.2. Drivers3.4.2.1. The Rise in Funds for Biobanks3.4.2.2. Increase in R&D Activities for Application of Biobank Specimens3.4.2.3. Surge in Prevalence of Fatal Chronic Diseases3.4.3. Restraints3.4.3.1. Legal and Ethical Issues of Biobanking3.4.3.2. Lack of Awareness Toward Biobanking3.4.4. Opportunity3.4.4.1. High Growth Potential in Developing Countries3.5. Impact of Covid-19 on Biobanking Market3.5.1. Overview3.5.2. Impact Analysis3.5.3. List of 10 Largest Biobanks Across the Globe

Chapter 4: Biobanking Market, by Specimen Type4.1. Overview4.1.1. Market Size and Forecast4.2. Blood Products4.2.1. Key Market Trends and Opportunities4.2.2. Market Size and Forecast, by Region4.2.3. Market Analysis, by Country4.3. Solid Tissues4.3.1. Key Market Trends and Opportunities4.3.2. Market Size and Forecast, by Region4.3.3. Market Analysis, by Country4.4. Cell Lines4.4.1. Key Market Trends and Opportunities4.4.2. Market Size and Forecast, by Region4.4.3. Market Analysis, by Country4.5. Nucleic Acid4.5.1. Key Market Trends and Opportunities4.5.2. Market Size and Forecast, by Region4.5.3. Market Analysis, by Country4.6. Others4.6.1. Key Market Trends and Opportunities4.6.2. Market Size and Forecast, by Region4.6.3. Market Analysis, by Country

Chapter 5: Biobanking Market, by Type of Biobank5.1. Overview5.1.1. Market Size and Forecast5.2. Population-Based Biobanks5.2.1. Key Market Trends and Opportunities5.2.2. Market Size and Forecast, by Region5.2.3. Market Analysis, by Country5.3. Disease-Oriented Biobanks5.3.1. Key Market Trends and Opportunities5.3.2. Market Size and Forecast, by Region5.3.3. Market Analysis, by Country

Chapter 6: Biobanking Market, by Ownership6.1. Overview6.1.1. Market Size and Forecast6.2. National/Regional Agencies6.2.1. Key Market Trends and Opportunities6.2.2. Market Size and Forecast, by Region6.2.3. Market Analysis, by Country6.3. Nonprofit Organization6.3.1. Key Market Trends and Opportunities6.3.2. Market Size and Forecast, by Region6.3.3. Market Analysis, by Country6.4. Universities6.4.1. Key Market Trends and Opportunities6.4.2. Market Size and Forecast, by Region6.4.3. Market Analysis, by Country6.5. Private Organization6.5.1. Key Market Trends and Opportunities6.5.2. Market Size and Forecast, by Region6.5.3. Market Analysis, by Country

Chapter 7: Biobanking Market, by Application7.1. Overview7.1.1. Market Size and Forecast7.2. Therapeutic7.2.1. Key Market Trends and Opportunities7.2.2. Market Size and Forecast, by Region7.2.3. Market Analysis, by Country7.3. Research7.3.1. Key Market Trends and Opportunities7.3.2. Market Size and Forecast, by Region7.3.3. Market Analysis, by Country

Chapter 8: Biobanking Market, by Region8.1. Overview8.2. North America8.3. Europe8.4. Asia-Pacific8.5. LAMEA

Chapter 9: Company Profiles9.1. Ams Biotechnology Ltd. (Amsbio LLC)9.1.1. Company Overview9.1.2. Company Snapshot9.1.3. Operating Business Segments9.1.4. Product Portfolio9.2. Bay Biosciences LLC9.2.1. Company Overview9.2.2. Company Snapshot9.2.3. Operating Business Segments9.2.4. Product Portfolio9.3. Bioivt & Elevating Science9.3.1. Company Overview9.3.2. Company Snapshot9.3.3. Operating Business Segments9.3.4. Product Portfolio9.3.5. Key Strategic Moves and Developments9.4. Boca Biolistics9.4.1. Company Overview9.4.2. Company Snapshot9.4.3. Operating Business Segments9.4.4. Product Portfolio9.5. Cti Biotech9.5.1. Company Overview9.5.2. Company Snapshot9.5.3. Product Portfolio9.6. Cureline, Inc.9.6.1. Company Overview9.6.2. Company Snapshot9.6.3. Operating Business Segments9.6.4. Product Portfolio9.7. Firalis S. A.9.7.1. Company Overview9.7.2. Company Snapshot9.7.3. Operating Business Segments9.7.4. Product Portfolio9.8. Geneticist Inc.9.8.1. Company Overview9.8.2. Company Snapshot9.8.3. Operating Business Segments9.8.4. Product Portfolio9.9. Proteogenex, Inc.9.9.1. Company Overview9.9.2. Company Snapshot9.9.3. Operating Business Segments9.9.4. Product Portfolio9.10. Us Biolab Corporation, Inc.9.10.1. Company Overview9.10.2. Company Snapshot9.10.3. Operating Business Segments9.10.4. Product Portfolio

For more information about this report visit https://www.researchandmarkets.com/r/5gm10t

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Outlook on the Biobanking Global Market to 2027 - Opportunity Analysis and Industry Forecasts - GlobeNewswire

Chinese pharmaceutical and 3D printing technology firm Triastek has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for its first 3D printed drug product, T19.

T19 has been developed in-house and is designed to treat rheumatoid arthritis, which is an autoimmune disease where the bodys immune system attacks the cells that line joints by mistake, making them stiff and swollen.

Triastek 3D printed the drug product using its Melt Extrusion Deposition (MED) technology platform and has global intellectual property (IP) rights to its 3D printed formulation.

Triastek is committed to improving the efficiency of formulation development, enhancing the effects of drug products, and ensuring the quality of drug delivered to patients by using the 3D printing technology platform, said Dr. Senping Cheng, Co-founder and CEO of Triastek. The FDA IND clearance of T19 is an important milestone in the development and application of MED 3D printing technology.

MED 3D printing platform

Triasteks MED 3D printing technology platform uses digital pharmaceutical dosage form design and automated intelligent manufacturing to enable the construction of tablets with sophisticated shapes and internal geometric structures. These structures allow the onset time, duration, and mode of drug delivery to be closely controlled and adjusted, which in turn provides more predictable and reproducible drug delivery results.

By enabling greater customization of the printed drugs, the MED system can help to enhance the therapeutic effects of medicines, while lowering side effects and improving compliance with patients. Triasteks novel pharmaceutical product development method, 3D formulation by Design (3DFbD) can be used in tandem with MED 3D printing technology to reduce the need for trial and error often required during traditional formulation development processes. 3DFbD is designed to improve both the efficiency and success rate of Triasteks drug product development in order to accelerate development time and lower costs.

The firm has also integrated real-time Process Analytical Technology (PAT) into the MED system that can continually monitor the 3D printing process to ensure product quality and make regulatory monitoring more convenient.

In April last year, the MED platform was accepted into the FDAs Emerging Technology Program (ETP) which recognized the 3D printing technology as a fully automated process for the manufacture of modified release solid oral dosage forms.

We believe that the MED 3D printing technology will be the enabler for digital pharmaceutical product development and intelligent drug manufacturing, said Dr. Xiaoling Li, Triasteks Co-founder and CSO. Triastek will work with any interested parties to take advantage of the platform technology for developing pharmaceutical products with better clinical value and higher product quality.

A chronotherapeutic drug delivery system

According to Triastek, deploying 3D printing for T19s novel design permits it to function as a chronotherapeutic drug delivery system. Chronotherapy treatment is based on the idea that administering medicine at different times of the circadian cycle the bodys natural internal process that regulates sleep over a 24 hour period will maximize a drugs therapeutic impact while minimizing side effects.

The T19 tablet is 3D printed on Triasteks MED system in a specific shape and with a precise internal geometric structure that facilitates the precise control of the drugs release to achieve the desired uptake by the patient. Taken at bedtime, T19s design means the drug will be released in a delayed manner so that blood concentration peaks when symptoms of pain and joint stiffness are most acute, such as in the early hours of the morning.

Having received IND clearance from the FDA for T19, Triastek is planning to apply for the same approval in China later this year, followed by applications in Japan and Europe. The firm expects to file a New Drug Application (NDA) for T19 to the FDA in 2023, and announced it has also developed a 505(b)(2) product portfolio using MED 3D printing technology to improve the outcomes of drug therapy.

3D printing and drug delivery

Personalized medication for individual patients is a fast-growing area of interest within the 3D printed pharmaceutical field. 3D printing enables the size and geometries of tablets and drug delivery devices to be modified to control their release and dosages depending on clinical need.

In 2015, Aprecia Pharmaceuticalsreceived FDA approvalfor its 3D printed Spiritam medication, making it the first 3D printed pharmaceutical to be approved by the FDA. The medication is designed to treat seizures in people with epilepsy and was approved for commercial manufacturing operations later that year. Aprecia recently announced a long-term collaboration with R&D firm Battelle to expand its capabilities within 3D printed pharmaceuticals and advance its 3D printing equipment from clinical supply to commercial scale. The firm has also previously partnered with Cambridge-basedCycle Pharmaceuticalsto produce orphan drugs for thetreatment of rare medical conditionsusing its ZipDose 3D printing process.

3D printed pharmaceuticals firm FabRx has also been active on the printed drug development front, having produced personalized medicine for children with the rare metabolic disorder maple syrup urine disease (MSUD), and printed its chewable Printlet tablets with Braille and Moon patterns on the surface to aid medicine taking for patients with visual impairment. The company launched its M3DIMAKER 3D printer in April 2020, designed specifically for the manufacture of personalized drug delivery devices.

Elsewhere, global pharmaceutical firm Merck has announced plans to work with EOS Group company ACMC to produce 3D printed tablets first for clinical trials, then later for commercial manufacturing. Meanwhile, in the research realm 3D printing has been used to optimize the controllable dosage of antibiotic tablets, and semi-solid extrusion 3D printing has been explored as a coating technology for customizing the release rate of patient-specific drugs.

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Featured image showsTriastek has gained IND clearance from the FDA for its T19 drug for rheumatoid arthritis. Image via Triastek.

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Triastek receives FDA IND clearance for 3D printed drug to treat rheumatoid arthritis - 3D Printing Industry

Newswise George Mason UniversitysCollege of Scienceannounced an exciting $1.33 million collaboration withRutgers Cancer Institute of New Jerseyand Masons Center for Applied Proteomics and Molecular Medicine (CAPMM) which taps cancer research diagnostics and treatment strengths within the medical facilities and CAP/CLIA laboratory respectively of the two regional powerhouses.

The effort will use a new way to measure biomarkers of the HER2 protein, which is expressed on breast cancer cells. Women who have this protein get specific therapies, now part of the standard of care in both early stage and metastatic settings of breast cancer.

The projects overall goal is clinical validation, in two independent patient cohorts from the Rutgers Cancer Institute, of a new way for measuring HER2 based on its activation (phosphorylation) and predicting, using only the patients diagnostic biopsy, whether or not that patient will respond favorably to the treatment.

The Mason-developed test uses a new Fluidigm laser capture microdissection (LCM), which enables researchers to capture cellular regions of interest from frozen and FFPE tissue sections for downstream analysis using DNA and RNA sequencing, real-time PCR or mass spectrometry analysis. The test is designed for patients with HER2 positive breast tumors to predict if each patient will achieve a complete remission of the tumor so that the tumor is completely absent and cannot even be found on pathologic tissue examination.

We aim to emerge from this short-term, high-impact effort with established cut-points, reagents, and CLIA/CAP SOPs developed and validated for our assay that would be published for general use, saidLance Liotta, MD, PhD, study PI, and CAPMM co-founder and co-director.

During the first year of the partnership, researchers will measure HER2 activation retrospectively on previously collected cancer specimens and cross reference it with treatment outcomes. During the second year, those findings will help inform the design of prospective trials enabling real-time decision making influencing the selection of future therapy options offered to patients at Rutgers Cancer Institute and RWJBarnabas Health.

We believe the test can have great benefit for individualizing therapies, added, Dr. Emanuel Petricoin, PhD, Co-PI of the study and also co-founder and co-director of Mason CAPMM. Clinical studies have supported the recent approval of two escalation strategies, unfortunately, these escalations are associated with considerable increased costs and additional risks of toxicity, Petricoin explained.

Therefore, careful and accurate patient risk stratification up-front, to identify which patients will benefit most from treatment, is essential to minimize risk and maximize benefit, said Rutgers Cancer Institutes principal investigator,Shridar Ganesan, MD, PhD, chief of molecular oncology, associate director for translational research, and Omar Boraie Chair in Genomic Science.

If validated, this predictor may help lead to development for modified or, de-escalated regimens for patients who score at low risk for recurrence or high chance of complete pathologic response Similarly, patients who score at high risk of recurrence or low chance of complete pathologic response from current standard therapy may benefit from trials exploring other treatment approaches.

The tests will be conducted on the pretreatment biopsy tissue we have collected and directly evaluate the molecular target of the major classes of HER2 neoadjuvant therapies, said Ganesan, a professor of medicine and pharmacology at Rutgers Robert Wood Johnson Medical School who had previously collaborated with Liotta while he was at the NIH.

This partnership allows us to continue to provide the most advanced treatment options possible for our patients, Ganesan added.

The teams plan also includes a rapid deployment of the testing if it proves conclusive.

Weve partnered with Theralink Diagnostics, Inc. who will be responsible for the assay commercialization and expanded clinical assessment to provide an accelerated adoption and clinical use by medical oncologists, saidEmanuel Petricoin, CAPMM co-director, and a scientific advisor to the Company. We have set up the infrastructure to quickly deploy the information and technology; if this holds true, we have a commercial partner ready to take it to the bedside as part of a patient clinical care plan and treatment decision support tool, Petricoin explained.

This is a smart, strategic partnershipboth Rutgers and Mason each bring invaluable expertise and infrastructure to minimize time to market and maximize research impact, said Mason College of Science dean, Fernando Miralles-Wilhelm. Mason scientists created the protein measurement test and offer CAP/CLIA facilities that dovetail well with the existing biobank of tissue samples and preeminent therapeutic cancer treatments options offered to patients at Rutgers Cancer Institute of New Jersey, Miralles-Wilhelm added.

The College of Science at George Mason University

Mason's College of Science is a leader in scientific discovery and a creator of innovative solutions for the rapidly-changing needs of today's world. The college prides itself in being home to a diverse population of students and researchers serving as a magnet for all scientific minds. With new discoveries, our scientists continue to grow Mason's portfolio of patents, licenses, partnerships, and spin off companies.

The college blends traditional science education with sought-after programs at all levels to challenge and engage in disciplines including personalized medicine, infectious diseases, drug discovery, climate dynamics, environmental justice and conservation, materials science, astronomy, forensic science, computational science, and applied mathematics. Mason's College of Science offers enhanced undergraduate and graduate academic and research opportunities, including innovative minors, certificates, and graduate degrees, that allow professionals to upskill or change careers. Learn more atscience.gmu.edu

About George Mason University

George Mason University is Virginia's largest public research university. Located near Washington, D.C., Mason enrolls more than 39,000 students from 130 countries and all 50 states. Mason has grown rapidly over the last half-century and is recognized for its innovation and entrepreneurship, remarkable diversity, and commitment to accessibility. Visithttp://www.gmu.edu

About Rutgers Cancer Institute of New Jersey

As New Jersey's only National Cancer Institute-designated Comprehensive Cancer Center, Rutgers Cancer Institute, together with RWJBarnabas Health, offers the most advanced cancer treatment options including bone marrow transplantation, proton therapy, CAR T-cell therapy and complex surgical procedures. Along with clinical trials and novel therapeutics such as precision medicine and immunotherapy - many of which are not widely available - patients have access to these cutting-edge therapies at Rutgers Cancer Institute of New Jersey in New Brunswick, Rutgers Cancer Institute of New Jersey at University Hospital in Newark, as well as through RWJBarnabas Health facilities. Learn more atcinj.org

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Mason Scientists and Rutgers Cancer Institute of New Jersey Collaboration Receives U.S. Armys Breakthrough Award to Better Inform HER2 Breast Cancer...

DUBLIN--(BUSINESS WIRE)--The "Companion Diagnostic Markets - the Future of Diagnostics, by Funding Source and Application with Customized Forecasting/Analysis, COVID-19 Updates, and Executive and Consultant Guides 2021-2025" report has been added to ResearchAndMarkets.com's offering.

Will Personalized Companion Diagnostics become the norm for diagnostics?

Companion Diagnostics are poised to revolutionize the diagnostics industry. The market is finally moving out of the lab and into the clinic. Oncology, especially immune-oncology is leading the way. And the FDA is holding the door open for this diagnostic technology of the future. But COVID-19 is impacting healthcare treatment everywhere and lowering demand for specialized cancer testing. Find out the latest outlook for this important market.

Learn all about how diagnostic players are jockeying for position with their pharmaceutical counterparts and creating new and significant business opportunities. And some players are already taking the lead. It is a dynamic market situation with enormous opportunity. Diagnostic companies are trying to back the right horse. The science is racing forward. And the cost of molecular diagnostics continues to fall.

Key Topics Covered:

Companion Diagnostic Market - Strategic Situation Analysis

1. Introduction and Market Definition

1.1 What are Companion Diagnostics?

1.2 The Personalized Medicine Revolution

1.3 Market Definition

1.4 Methodology

1.5 A Spending Perspective on Clinical Laboratory Testing

2. Market Overview

2.1 Players in a Dynamic Market

2.1.1 Academic Research Lab

2.1.2 Diagnostic Test Developer

2.1.3 Instrumentation Supplier

2.1.4 Distributor and Reagent Supplier

2.1.5 Independent Testing Lab

2.1.6 Public National/regional lab

2.1.7 Hospital lab

2.1.8 Physician Office Labs

2.1.9 Audit Body

2.1.10 Certification Body

2.2 Personalized Medicine and Companion Diagnostics

2.2.1 Basics

2.2.2 Method

2.2.3 Disease risk assessment

2.2.4 Applications

2.2.5 Diagnosis and intervention

2.2.5.1 Companion Diagnostics

2.2.6 Drug development and usage

2.2.7 Respiratory proteomics

2.2.8 Cancer genomics

2.2.9 Population screening

2.2.10 Challenges

2.2.11 Regulatory oversight

2.2.12 Intellectual property rights

2.2.13 Reimbursement policies

2.2.14 Patient privacy and confidentiality

2.3 Chromosomes, Genes and Epigenetics

2.3.1 Chromosomes

2.3.2 Genes

2.3.3 Epigenetics

2.4 Cancer Genes

2.4.1 Germline vs Somatic

2.4.2 Changing Clinical Role

2.5 Structure of Industry Plays a Part

2.5.1 New Pharmaceutical Funding Market

2.5.2 Economies of Scale

2.5.2.1 Hospital vs. Central Lab

2.5.3 Physician Office Labs

2.5.4 Physicians and POCT

3. Market Trends

3.1 Factors Driving Growth

3.1.1 Level of Care

3.1.2 Immuno-oncology

3.1.3 Liability

3.1.4 Aging Population

3.2 Factors Limiting Growth

3.2.1 State of knowledge

3.2.2 Genetic Blizzard.

3.2.3 Protocol Resistance

3.2.4 Regulation and coverage

3.3 Instrumentation and Automation

3.3.1 Instruments Key to Market Share

3.3.2 Bioinformatics Plays a Role

3.4 Diagnostic Technology Development

3.4.1 Next Generation Sequencing Fuels a Revolution.

3.4.2 Single Cell Genomics Changes the Picture

3.4.3 Pharmacogenomics Blurs Diagnosis and Treatment

3.4.4 CGES Testing, A Brave New World

3.4.5 Biochips/Giant magneto resistance based assay

4. Companion Diagnostics Recent Developments

4.1 Recent Developments - Importance and How to Use This Section

4.1.1 Importance of These Developments

4.1.2 How to Use This Section

5. Profiles of Key Players

6. The Global Market for Companion Diagnostics

6.1 Global Market Overview by Country

6.2 Global Market by Application - Overview

6.3 Global Market Funding Source - Overview

7. Global Companion Diagnostic Markets - By Application

7.1 Oncology

7.2 Neurology

7.3 Cardiology

7.4 Other Application

8. Global Companion Diagnostic Markets - Funding Source

8.1 Global Market Pharmaceutical

8.2 Global Market Venture

8.3 Global Market Clinical

8.4 Global Market Other Funding

For more information about this report visit https://www.researchandmarkets.com/r/f07ek

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Global Companion Diagnostic Markets Report 2021: A Steep Growth Curve Interrupted by COVID-19 - Forecast to 2025 - ResearchAndMarkets.com - Business...

WALTHAM, Mass.--(BUSINESS WIRE)--BostonGene Corporation, a biomedical software company committed to defining optimal precision medicine-based therapies for cancer patients, and Weill Cornell Medicine, today announced a publication in Cancer Discovery, a journal of the American Association for Cancer Research. The manuscript Clinical and biological subtypes of B-cell lymphoma revealed by microenvironmental signatures highlights the tumor microenvironment as a critical component of B-cell lymphoma biology and the effects of different microenvironments on diffuse large B-cell lymphoma (DLBCL) clinical behavior, establishing a significant opportunity for the development of novel and personalized therapeutic strategies for this disease.

In this research study, the microenvironment subtypes of over 4,600 DLBCL patients were classified using curated and refined transcriptional signatures encompassing key microenvironment and cancer cells activities and processes. This analysis revealed four distinct DLBCL microenvironments (LMEs), each with its own set of unique biological and clinical properties. The LMEs were also found to correlate with different clinical outcomes and prognoses, and downstream preclinical mechanistic studies demonstrated that the LMEs could be applied in clinical decision-making for DLBCL patients.

This study was designed to evaluate the role of the tumor microenvironment in DLBCL biology, said Leandro Cerchietti, M.D., Associate Professor of Medicine and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine. The results revealed distinct DLBCL microenvironments with unique therapeutic vulnerabilities that can be utilized for optimization of DLBCL treatment strategies.

The data demonstrate that this novel classification platform provides a roadmap for the therapeutic exploitation of the tumor microenvironment in DLBCL patients, said Nathan Fowler, MD, Chief Medical Officer at BostonGene. Together with Weill Cornell Medicine, we look forward to identifying new treatment strategies to ultimately improve the clinical outcomes of these patients.

About BostonGene Corporation

BostonGene Corporation is pioneering the use of biomedical software for advanced patient analysis and personalized therapy decision making in the fight against cancer. BostonGenes unique solution performs sophisticated analytics to aid clinicians in their evaluation of viable treatment options for each patient's individual genetics, tumor and tumor microenvironment, clinical characteristics and disease profile. BostonGenes mission is to enable physicians to provide every patient with the highest probability of survival through optimal cancer treatments using advanced, personalized therapies. For more information, visit BostonGene at http://www.BostonGene.com.

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BostonGene and Weill Cornell Medicine Announce Publication in Cancer Discovery Revealing the Role of the Tumor Microenvironment in the Clinical...

MINNEAPOLIS, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Predictive Oncology (NASDAQ: POAI), a knowledge-driven company focused on applying artificial intelligence (AI) to personalized medicine and drug discovery, announced today that it will start an in-house drug repurposing project, focused on ovarian cancer, using its proprietary AI-driven, patient centric discovery platform PeDAL. The project will use PeDAL to rapidly and cost-effectively profile panel of existing drugs against hundreds of patient cell lines. This process will generate data on which compounds are active against which specific patient profile(s), delivering both proof data for the PeDAL approach and valuable Intellectual Property (IP) for the company.

With our Helomics divisions deep experience in clinical profiling of patient tumor drug response, plus our unique PeDAL platform, we have all the components to perform in-house drug repositioning and even full drug discovery. We are very excited to kick-off this first project focused on ovarian cancer. Our expectations are that the project will have significant value by generating proof data for our PeDAL approach, which will be useful in our commercial discussions with Pharmaceutical companies. In addition, the project will also demonstrate the long-term value of Helomics unique data, explained Dr. Carl Schwartz, CEO of Predictive Oncology.

Looking forward, we strongly believe successful execution of this project will demonstrate that Predictive Oncology is emerging as a leader in AI-driven drug discovery, commented Dr. Schwartz.

About Predictive Oncology Inc.

Predictive Oncology (NASDAQ: POAI) operates through three segments (Skyline, Helomics and Soluble Biotech), which contain four subsidiaries: Helomics, TumorGenesis, Skyline Medical and Soluble Biotech.

Helomics applies artificial intelligence to its rich data gathered from patient tumors to both personalize cancer therapies for patients and drive the development of new targeted therapies in collaborations with pharmaceutical companies. TumorGenesis Inc. specializes in media that help cancer cells grow and retain their DNA/RNA and proteomic signatures, providing researchers with a tool to expand and study cancer cell types found in tumors of the blood and organ systems of all mammals, including humans. Skyline Medical markets its patented and FDA cleared STREAMWAY System, which automates the collection, measurement, and disposal of waste fluid, including blood, irrigation fluid and others, within a medical facility, through both domestic and international divisions. Soluble Biotech is a provider of soluble and stable formulations for proteins including vaccines, antibodies, large and small proteins, and protein complexes.

Forward-Looking Statements

Certain matters discussed in this release contain forward-looking statements. These forward-looking statements reflect our current expectations and projections about future events and are subject to substantial risks, uncertainties and assumptions about our operations and the investments we make. All statements, other than statements of historical facts, included in this press release regarding our strategy, future operations, future financial position, future revenue and financial performance, projected costs, prospects, plans and objectives of management are forward-looking statements. The words anticipate, believe, estimate, expect, intend, may, plan, would, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Our actual future performance may materially differ from that contemplated by the forward-looking statements because of a variety of factors including, among other things, factors discussed under the heading Risk Factors in our filings with the SEC. Except as expressly required by law, the Company disclaims any intent or obligation to update these forward-looking statements.

Investor Relations Contact:

Landon Capital Keith Pinder (404) 995-6671kpinder@landoncapital.net

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Predictive Oncology's Wholly Owned Subsidiary Helomics announces the start of its drug repurposing project using its ground-breaking PeDAL platform...

The collaboration with the Spanish Lung Cancer Group (Grupo Espaol de Cncer de Pulmn GECP), a Spanish cooperative group for the research on lung cancer, is to show the potential of SOPHiA Radiomics a groundbreaking application that analyzes medical images for research use put to use in an additional retrospective analysis of the data from the phase 2 NADIM clinical trial (NCT03081689) (the NADIM trial).

The NADIM trial, funded by Bristol-Myers Squibb and part of the European Union's Horizon 2020 research and innovation program, aimed to assess the antitumor activity and safety of neoadjuvant chemoimmunotherapy for resectable stage IIIA NSCLC. The important results recently published in TheLancet Oncology supported the addition of neoadjuvant nivolumab to platinum-based chemotherapy in patients with resectable stage IIIA NSCLC. This very aggressive type of cancer is unfortunately terminal in most patients with locally advanced staged disease; these results could therefore support a change of perception of locally advanced lung cancer as a potentially lethal disease to one that is curable.

Additional data are expected to be generated through the analysis of the radiology images of NADIM patients through the SOPHiA Radiomics Platform. These data will then be combined with clinical, biological, and genomics data, and multimodal machine learning models will be developed to predict response to neoadjuvant treatment, using baseline and pre-surgery data. The predictive analysis will also aim to stratify patient cohorts with regard to progression-free and overall survival.

"We are very happy to collaborate in this innovative and revolutionary project that opens the door to a new precision medicine. Certainly, this partnership will improve the knowledge relating to the treatment for this group of patients and will allow to approach the best prospects for curing early-stage non-small cell lung cancer (NSCLC)," said Dr. Mariano Provencio, Head of the Medical Oncology Department at Puerta de Hierro University Hospital in Madrid and lead investigator of the NADIM trial.

"We are very excited to apply our radiomics and multimodal analytics capabilities to such an important clinical question," said Prof. Thierry Colin, Vice-President of Radiomics Research at SOPHiA GENETICS. "In the Spanish Lung Cancer Group, we have found visionary partners that clearly see the promise of next-generation health data such as radiomics being married with tech-enabled solutions in artificial intelligence to generate entirely novel clinical insights for the benefit of oncology patients."

"Unlocking the synergistic potential of multimodal health data through artificial intelligence holds revolutionary promise for the future of personalized medicine in oncology and many other health conditions. We are inspired by the potential to positively impact on patients by supporting their care providers in predicting the best course of treatment," said Dr. Philippe Menu, Chief Medical Officer at SOPHiA GENETICS.

The NADIM trial involved 18 centers from the Spanish Lung Cancer Network that will contribute their data to the joint project.

Results from the additional analysis of NADIM trial data are expected to be available later this year.

About SOPHiA GENETICS

SOPHiA GENETICS is a health tech company democratizing Data-Driven Medicine to improve health outcomes and economics worldwide. By unlocking the power of new-generation health data for cancer and rare diseases management, the universal SOPHiA Platform allows clinical researchers to act with precision and confidence. The company's innovative approach enables an ever-expanding community of over 1,000 institutions to benefit from knowledge sharing, fostering a new era in healthcare. SOPHiA's achievement is recognized by the MIT Technology Review's "50 Smartest Companies".

More info: SOPHiAGENETICS.COM, follow @SOPHiAGENETICS on Twitter.

About the Spanish Lung Cancer Group

The SLCG is an independent, non-profit, cooperative group founded in 1991 to promote the study and research of lung cancer. The group is composed of 525 medical professionalsoncologists, thoracic surgery specialists, radiotherapists and basic researchersand unites 177 public and private centers across Spain.The group has treated over 37,000 patients in different studies and clinical trials, the results of which have been presented and published at national and international congresses and in leading medical journals.

More info: gecp.com, follow @gecp_org on Twitter.

SOPHIA GENETICS Media ContactSophie ReymondPR & Communications Manager [emailprotected]+41 79 863 11 10

SLCG ContactRita Perales [emailprotected] +34 670 24 70 50

SOURCE SOPHiA GENETICS

https://www.sophiagenetics.com/

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SOPHiA GENETICS and the Spanish Lung Cancer Group Team Up to Explore the Predictive Potential of Multimodal Health Data in Resectable Stage IIIA...

In recent years, the idea of the microbiome has gone from being an esoteric term used in scientific circles, to a mainstream concept employed in adverts to sell microbiome-boosting health drinks and supplements. The increase in public interest has been fed by a series of headline-grabbing research breakthroughs, and the fact that the microbiome has a key role to play in the development of precision medicine.The trillions of microbes contained in the human body are a key element of a personalized approach to treatment; the microbiome influences endocrinology, physiology, and even neurology, and has a crucial role in disease progression. The growing awareness of the various ways in which microbiota affects each of us individually in sickness and in health is also leading to an increase in research. An area in which this interest is growing particularly quickly is oncology.

Multiple publications implicate microbiota in the onset and progression of cancers, as well as toxicity and the response rate of cancer treatments. An analysis of 12 million full-text publications, 29 million abstracts and 521 thousand grant applications for semantic relations between cancers and microbiota is shown in figure 1. The data show a considerable increase in the number of articles linking cancers to microbiota for five cancer types with the highest number of reports overall.

Figure 1.Trend of reports linking cancers to microbiota 20082019. Credit: Graph generated using Elsevier Text Mining and Scopus.

With overall cancer rates set to increase worldwide, the current interest in the microbiome and its role in precision medicine is likely to continue because it offers new hope of treatments. Evidence suggests the importance of looking for predictors of therapeutic response beyond the tumor by focusing on host factors, such as microbiota and host genomics.1 Importantly, the microbiota is a modifiable factor, and potentially can become not just a predictive marker but also a potential target in order to improve outcomes for patients.

Progress is also being made in clinical trials looking at the microbiome and melanoma. Since 2018, four clinical trials that aim to study and modulate the gut microbiomes impact on response to immunotherapy of melanoma have been registered at clinicaltrials.gov. Dr Marc Hurlbert, Chief Science Officer for the Melanoma Research Alliance, commented on the findings: As noted in the report, there has been an explosion of knowledge about melanoma with an ever-increasing list of protein targets. Also noted, the role of the microbiome in melanoma and in response to immunotherapy is of increasing interest in the field.

To further develop targeted precision therapies, further research is now required. Firstly, to map genetic variants; secondly, to determine which variant is clinically significant; thirdly, to understand the impact of variant on gene function, and whether variation activates or inhibits the gene. This is particularly important for increased understanding of specific, precision medicine and to enhance therapeutic efficacy.

For non-hereditary (sporadic) melanoma, the analysis showed that there are 752 genes genetically linked to sporadic melanomas and its subtypes, and 449 genetic variants genetically linked to sporadic melanoma and its subtypes. Out of the 449 genetic variants, 395 are from 78 genes that are genetically linked to melanoma. The remaining missing 54 variants are not currently genetically linked in the platform to any known melanoma gene; this could therefore be a potential area for further research.

Understanding whether specific genetic variants exist and/or contribute to melanomas severity and prevalence in populations will help the research and development (R&D) industry to develop more effective and profitable therapeutics. These types of data will provide the R&D community with a greater depth of understanding and of the increased likelihood of hitting the target. Through our analysis we found an increased incidence of drugs targeting genetic mutations over the last decade, particularly targeting protein kinases and growth factor receptors.

It is an attractive future research avenue to recognize how a patients microorganisms genome, both symbiotic and pathogenic, can dramatically effect treatment plans and outcomes. Positively influencing the microbiome in patients needs further study that could lead to exciting opportunities for patients and for drug discovery. For the therapeutic pipeline it would be beneficial to understand these host-microbiota interactions and ways to positively tip the balance towards improving treatment outcomes.

One other interesting future consideration during drug development for all cancers is the influence of the microbiome on treatment-induced adverse events, and whether clinical and post-clinical adverse events are related to a patients microbial composition. It adds a level of complexity as to the efficacy of therapeutics that may not readily be considered, and potentially may be something to consider during future clinical trials.

Moreover, in the current COVID-19 era, in-person and patient interactions are reduced and many research labs are still unable to operate at full capacity. The ability to conduct research, take samples and study real patients is limited at present, so looking at detailed existing literature and data is a vital avenue to support R&D. It will keep R&D functions going and help them to direct efforts to the areas of greatest potential. 2021 will be a year of reduced R&D budgets globally this type of data insight will be vital to empowering future R&D.

Tom is the Life Sciences Group Manager of Project Management, Knowledge Manager, and Research Scientist. He has extensive experience as an academic researcher in neurodegeneration and Alzheimers disease. He is also skilled in biophysical chemistry, dementia disorders, and biochemistry. He is the author of many publications in the field of protein-membrane interactions, protein misfolding, and Alzheimers disease. At Elsevier he delivers and implements information solutions for customers.

Tom discusses the study and unmet needs in melanoma R&D in detail, here, alongside Marc Hurlbert, Ph.D. Chief Science Officer, Melanoma Research Alliance.

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Exploring the Relationship Between the Microbiome, Precision Medicine and Cancer - Technology Networks